Sunday, December 25, 2016

PKD: What Medicare Pays, PKD Research: 3D Map of PC2, PKD Fundraising: Philly, Gift of Life: Longview WA, DownUnder

Living with PKD

From National Public Radio NPR, by Richard Harris (On Morning Edition)

Medicare Pays For A Kidney Transplant, But Not The Drugs To Keep It Viable


The federal government will pay more than $100,000 to give someone a kidney transplant, but after three years, the government will often stop paying for the drugs needed to keep that transplanted kidney alive.

Constance Creasey is one of the thousands of people who find themselves caught up by this peculiar feature of the federal kidney program.

Creasey started kidney dialysis about 12 years ago after her kidneys failed. That meant going to a dialysis center three times a week, for three hours per session. (A typical patient undergoes three to five hours of dialysis per session).

"The first three years of dialysis was hard. I walked around with this dark cloud. I didn't want to live, I really didn't," she says.

Being dependent on these blood-cleansing machines was physically and emotionally draining. But she stuck it out for 11 years. Medicare pays for dialysis, even for people under the age of 65. It also pays for kidney transplants for people with end-stage renal disease.

"Finally, a year and a half ago, transplant came. I was a little apprehensive but I said OK. And I call her Sleeping Beauty, that's my kidney's name."

Creasey, a 60-year-old resident of Washington, D.C., no longer needs to spend her days at a dialysis center. She has enough energy for a part-time job at a home furnishing store and time to enjoy life's simple pleasures.

"I was able to do my favorite thing — go to the pool — and I was just loving it because it's like I had no restrictions now," she says.

But there is still a dark cloud on Creasey's horizon. Medicare's kidney program currently pays for a large share of the expensive drugs she needs to take twice a day to prevent her body from rejecting the transplanted kidney. But under federal rules, that coverage will disappear three years after the date of her transplant.

"I have a year and a half to prepare, or save," she says. "How am I going to do this?"

She's already paying copays, premiums and past medical bills. She says she sleeps on the floor because she considers buying a bed a luxury she can't afford.

She has no idea what kind of insurance she'll be able to get after her Medicare coverage runs out. And she was shocked to discover how big the bills could be. One day she went into the pharmacy to pick up her drugs, and the Medicare payment hadn't been applied.

The pharmacist told her she'd need to pay a $600 copay for the one-month supply. "And I'm like are you kidding me? Six hundred? What am I going to do? I can't pay that!"

A social worker at MedStar Georgetown University Hospital in Washington, D.C., where Creasey got her transplant, sorted that out. But it's not a permanent solution.

The three-year cutoff for Medicare payments is a common problem, says Dr. Matthew Cooper, who runs the kidney transplant program at the hospital. That's especially so since many people with serious kidney disease have low incomes in the first place.

"It's probably about 30 percent of people who find themselves in a troublesome spot at this 36-month mark," he says.

Some people end up trying to stretch out their drug supplies by not taking them as often as they need to, he says. "We see that a lot."

But this isn't like skipping a pain pill and bearing the consequences. People lose their transplanted kidneys through organ rejection if they don't take their medicine religiously.

Rita Alloway, a clinical pharmacist at the University of Cincinnati, says she also encounters this false economy. "If we were telling them to take four pills twice a day, they may start taking three pills twice a day without telling us, to extend their coverage that they had for the prescriptions they had," she says. [Read more]



PKD Research

From PR.com

3D Map of PC2 to Jump-Start Drug Discovery Efforts in Polycystic Kidney Disease

Boston, MA, December 21, 2016 --(PR.com)-- Scientists from the Structural Genomics Consortium (SGC) and Oxford University report a three-dimensional view of the Polycystin-2 (PC2), a protein that causes autosomal dominant polycystic kidney disease (ADPKD) when mutated, in an article published this week in Nature Structural and Molecular Biology.

ADPKD is one of the most common life-threatening genetic diseases, affecting ~1 in 800 individuals at birth. The disease is characterized by the formation of fluid-filled cysts that compromise kidney function and cause substantial pain for patients. 50% of patients with ADPKD eventually develop end-stage kidney disease and require dialysis or a transplant to survive.

Mutations in the genes that encode polycystin-1 (PC1) and PC2 cause the disease, but the mechanisms by which these mutations disrupt the function of these proteins and lead to disease are not known. PC1 and PC2 proteins are embedded in multiple membranes of kidney cells, regulate the movement of positively charged ions into these cells or into specialized compartments within the cells, and impact cellular signaling.

Scientists led by Profs Liz Carpenter and Juha Huiskonen at the University of Oxford employed a technique called cryo electron microscopy (cryoEM) to provide a three-dimensional view of PC2. The work revealed a novel substructure called the Tetragonal Opening for Polycystins or TOP domain, which caps the protein. At least 27 of the known ADPKD-causing mutations map to this region of the protein, and this structure provides important insight into how the TOP domain impacts the ability of PC2 to bind to and control the movement of ions.

“There is a significant need for scientists to provide insight into the biology of PC1 and PC2 so we can develop medicines to help patients with ADPKD. These structural insights will help scientists begin to understand how mutations in PC2 cause disease, and more importantly, generate ideas for how the disease can be treated,” said Chas Bountra, Director of SGC Oxford.

SGC scientists now plan to identify small molecules that stabilize PC2, with the hope of supporting its function in kidney cells and inhibiting cyst formation.

The paper, Structure of the polycystic kidney disease TRP channel Polycystin-2 (PC2), was published December 19th online on Nature Structural and Molecular Biology’s website.




PKD Fundraising

From Digital Journal

Chorus Communications Helps Raise $10,000 for Polycystic Kidney Disease Patient and Donor



Philadelphia, PA -- (SBWIRE) -- 12/23/2016 -- Chorus Communications, a leading customized business solutions consultant, held a fundraiser last month for Brian Servis, a polycystic kidney disease (PKD) patient currently undergoing dialysis. The fundraiser occurred to support Servis and his sister-in-law, Amy Servis, Chorus Communications' Channel Manager, and kidney donor to Brian.

"The outpour of support for Brian and I has been a true inspiration, and made this holiday season so special for our entire family," said Amy Servis.

As soon as she discovered that she and Brian share the same blood type, Amy Servis, a member of the coveted Women in the Channel organization, quickly jumped at the opportunity to donate a kidney to her brother-in-law. PKD is a hereditary disorder that causes cysts to grow inside a person's kidneys, which leads to their enlargement and subsequent failure. As of now, Brian is on dialysis, and in need of a kidney transplant as soon as possible.

Chorus Communications would like to give a special thank you to Telecom For Change, for their generosity in contributing $2,500 to Amy and Brian to help supplement their recovery time post-surgery.

The fundraiser took place at Chambers 19 Bistro & Bar in Doylestown, Pennsylvania on November 30, and saw an enormous and supportive turnout. As well as the restaurant donating 20 percent of their profits for the day to Brian and his family, donors were able to raise roughly $10,000 through a Hair O' The Dog ticket raffle, gift basket sales, a 50-50 raffle and a GoFundMe campaign.

Those interested in contributing to Brian Servis' GoFundMe campaign can click here. To learn more about Chorus Communications, a leading business internet consultant in Bucks County, Pennsylvania and Greater Philadelphia, please visit their website.

About Chorus Communications
Chorus Communications is an award-winning Technology Consulting Firm that has built an unmatched reputation with its sales-partners, suppliers, and customers. Founded in 1995, Chorus is highly regarded as a leading Master Agency and has an extensive portfolio of cutting edge products and services to meet today's ever-evolving world of technology. The experts at Chorus provide knowledgeable recommendations on your needs from complex cloud designs to global wide-area network deployments and everything in between.

Chorus is dedicated to helping sales partners and customers in making knowledgeable choices about services, technology, and cost-efficient communications.

For more information on this press release visit: http://www.sbwire.com/press-releases/chorus-communications-helps-raise-10000-for-polycystic-kidney-disease-patient-and-donor-754164.htm





Gift of Life

From The Daily News, Longview, WA, by Denver Pratt


kidney donor


Dana Clayton had waited more than 20 years for this moment. It was the day her body was free of the disease that had caused her extreme pain, exhaustion and shortness of breath, had yellowed her skin, and was about to force her into dialysis treatments to stay alive.

In early December, just four months after her best friend’s husband donated her one of his kidneys, Dana had her own, diseased kidneys removed. She’s showing no signs of organ rejection and, for the first time in more than 20 years, she is healthy and free of polycystic kidney disease.

“It’s going to change my life and our lives now that I’m going to be able to be healthy and do things again,” Dana said in an interview last week.


Dana’s story and that of her donor, Cowlitz 2 firefighter Brad Yoder, have been told before. But even now that this unlikely story is coming to a close, Dana, Brad, and their spouses are still amazed at how fast the process from matching to donation to recovery took. And while there are tinges of guilt and some struggles with recuperation, the bond between them and their families has never been stronger.

A perfect match

In 1994, Dana, of Kalama, was diagnosed with polycystic kidney disease, which causes cysts to form on kidneys and eventually causes them to fail.

In November last year, Dana, 44, was placed on the transplant list when her kidney function dropped below 10 percent. Her kidneys were the length of a ruler; they should have been the size of a fist. Dana and her husband, Brian Clayton, prepared for a long wait and dialysis treatment, thinking it would take years to find a matching donor. But, to their surprise, this wasn’t their future.

“Everything went incredibly faster than what they anticipated,” Brian said. “Everything has worked out. It’s pretty amazing.”

When Dana was placed on the transplant list over a year ago, her mother sent an email about it to close friends and family. The message listed statistics on wait times for organs, information about Dana’s disease and a link to sign up to become a donor. Among the recipients of the email was Laura Yoder of Kelso. She’s been Dana’s best friend since junior high school.

Knowing he had O-negative blood, the type to be a universal donor, Brad Yoder, Laura’s husband, clicked on the link taking him to the organ donor website. Forty-eight hours after he read the email, he signed up to see if he was a match.

A week later, a transplant team contacted him. They had reviewed his application to become a donor for Dana. They had discarded every single applicant but him. When his wife found out, she said she wasn’t surprised her husband, 44, was willing to donate a kidney to Dana.

He started the testing process immediately. Normally, the matching process takes four to six months, but Brad was determined to get it done quicker. He didn’t want Dana to go through dialysis, he said.

The testing process was rigorous. But after six weeks, he had confirmation: Not only was Brad a match for Dana, but he was so close of a match that doctors typically only see those results in siblings or relatives. Brad was so excited about the news that he showed up to Dana’s workplace to tell her in person.

On Aug. 15, the pair underwent the transplant surgery at Legacy Good Samaritan Hospital in Portland, removing one of Brad’s kidneys and implanting it in Dana. They left her two diseased kidneys in place.

Following the surgery, there were high-fives, hugs and excitement shared among family, friends and coworkers in the waiting room. After Dana’s surgeon came out to say her operation had gone well and that the donated kidney was in and functioning, Laura cried tears of happiness.

“Having been (Dana’s) friend for so long and knowing that you were going to go to her surgery someday, I knew I would be there in a form of support role somehow,” Laura said. “But, realizing that both of my best friends, literally my best friend — my husband — and my best friend of childhood ended up there … you already knew you were going to walk those steps. I just didn’t quite realize how big of a shoe it was going to be.”

Recovery

It’s been a little over four months since the transplant, and just a few weeks since Dana had her diseased kidneys removed. Doctors had waited to remove them to give the donated kidney every fighting chance, she said.

One thing the group noticed after the surgery was how healthy Dana looked. They had not realized how sick she had been.

“I noticed it 24 hours after the surgery,” Brad said. “During pre-op surgery we’re side by side, we’re across the room from each other, and I started taking things in and just kind of noticing … how Dana looked. It didn’t really hit me until the next day.”

Dana improved dramatically right away.

“The day before it was like a black and white picture and then you see that same picture with full color and it is a trip. Her skin looked tighter. It was glowing and there was color and just vibrance, and that hit me,” Brad said. “I don’t think any of us paid that much attention to it until we just sat back and noticed it. I mean that’s how long it’s been, I’ve known her for seven years, and you just didn’t see it.”




From News.com.au by Brad Crouch

Organ donor officials say they now see cases where a healthy person donates one of their two kidneys to a stranger as a gift of life

Erin Taylor with her aunt Eleanor Taylor, who donated her kidney to her niece.

Erin Taylor with her aunt Eleanor Taylor, who donated her kidney to her niece.Source:News Limited


WHILE some girls hope for an iPad or perfume for Christmas, Erin Taylor, 12, is hoping for a kidney.

It may come from the true spirit of humanity — although rare, organ donor officials say they now see cases where a healthy person donates one of their two kidneys to a stranger as a gift of life.

Erin will spend Christmas with her Aunty Eleanor in Torquay enjoying their special bond.

Four years ago, Eleanor Taylor donated one of her kidneys to her niece but, in a sad twist, rejection of the organ means another transplant will be needed.

The North Brighton family’s emotional ordeal began in the lead-up to Christmas 2011 when Erin, then aged seven, was taken to Flinders Medical Centre on December 7 with what the family thought was gastro. She was immediately transferred to the Women’s and Children’s Hospital intensive care unit with kidney failure.

She made it home for Christmas Day but within four months her under-formed kidneys had been removed and she was being kept alive by dialysis.

Her parents, Andrew and Sarah, were not suitable donor matches but Andrew’s sister Eleanor selflessly stepped in to volunteer one of her own healthy organs for her niece.

On September 17, 2012, Erin received the donated kidney.

Unfortunately there have been problems since the transplant operation, Erin’s mother said.

“For the last three years her body has been rejecting the kidney and a biopsy a couple of weeks ago showed it is getting severely scarred,” she said.

“We’ve been told she has probably got one or two years left with it — she will need another transplant.”

Mrs Taylor is considering joining a donor pool where she would donate a kidney to another family in a similar position in return for a suitable kidney for Erin.

Erin is on growth hormones due to the treatment slowing her growth but is full of enthusiasm for life — she swims three times a week and plays sport, including volleyball and softball.

“I feel fine,” she said. “I’m really looking forward to going to Torquay for Christmas with Aunty Eleanor.”

In cases where a family member is not a suitable match, people needing a kidney transplant go on the waiting list for a donor organ which usually comes from a deceased person who made the decision to donate.

According to the Australian and New Zealand Organ Donor Registry, in 2015 in Australia 435 deceased organ donors donated to 1241 transplant recipients which included 703 kidneys.

However there are rare cases of a gift that even Santa can’t match — a healthy person giving a total stranger a kidney.

In the past 12 months several selfless people have stepped forward with a life-changing gift by making an altruistic donation of an organ.

Kidney Health Australia spokeswoman Professor Carol Pollock said it was now not uncommon for friends as well as family members to volunteer.

“Sometimes there are cases where a person who was getting ready to donate a kidney to a friend — and who had been through all the tests and was a good candidate to be a donor — no longer has to give one because a suitable donor kidney became available,” Prof Pollock said.

“We are now seeing cases where those people decide to continue on and become an altruistic donor — they give a kidney which goes into the pool to help someone they do not even know.

“In the past 12 months there have been several cases like this that I am aware of.

“You can function perfectly well with one kidney and it makes a huge difference to the recipient.”

Sunday, December 18, 2016

PKD Life: Donating Old Kidneys, More New Donation Ideas, PKD Research: Clinical Trials, Venture Capitalists

PKD Life

From CNN, By Susan Scutti


Transplant centers come up short more often than not when treating patients in need of a kidney, as there is a shortage of donors. Yet kidneys donated by people 65 or older can still function for many years after transplantation, according to a study published Thursday in the Clinical Journal of the American Society of Nephrology. The results suggest that a new source of quality kidneys could be available to patients in need of life-saving transplants.

"Kidneys from an old donor may be favorably used, particularly in an age-matched patient," said Dr. Luigi Biancone, lead author of the study and associate professor of nephrology at the University of Torino in Italy.

Biancone and his colleagues found similar survival rates for transplanted organs from donors in the age ranges of 50 to 59 years old, 60 to 69 years old, 70 to 79 years old and even 80 years or older.
"Age is not the sole criteria by which the outcomes of an organ or the quality of an organ should be judged," said Dr. David Klassen, chief medical officer of the United Network for Organ Sharing(UNOS), which operates the Organ Procurement and Transplantation Network -- the nation's transplant system -- under a contract with the federal government.

"To exclude an organ just based on age is probably not appropriate," said Klassen, who was not involved in the new study. He said the new results are "consistent with previous studies suggesting the outcomes for older donors can be quite good."

An extensive wait list

The kidneys, a pair of bean-shaped organs about 4½ inches long, remove waste from the body and help regulate blood pressure, blood volume and blood composition. Kidney failure, also called renal failure, occurs when they are unable to remove waste and maintain the blood's chemical balance. Chronic failure can cause fatigue, sluggishness, anemia, decreased urine and complications such as congestive heart failure. According to the UNOS website, if treatment fails, dialysis and transplantation become necessary.

The organ shortage is dramatic, and the numbers tell the story.

Currently, 99,101 people are waiting for kidneys in the United States, according to UNOS, which updates its numbers in real time. Yet only 12,245 transplant operations using kidneys from 8,306 deceased donors have been performed this year, as of November 30.

Although there are nearly "100,000 people waiting for kidney transplants, the other organ waiting lists are not nearly as large," observed Klassen. This is because people on dialysis can wait for years for a transplant -- and often do -- but people who need heart, lung or liver transplants cannot wait, because of both the nature of their disease and "the ability of medical science to sustain people."
"There's no equivalent of dialysis for lung recipient candidates or liver recipient candidates," Klassen said.

However, a very common reason for discarding a donated kidney is the older age of a donor.
"There are no restrictions, per se," Klassen said. The organ network has never established a policy or "mandated restriction that you cannot procure organs from a recipient a certain age. That's never been the case."

A potential new source of donor organs

Troubled by the small number of available kidneys and the large number of people waiting, Biancone and his colleagues investigated how the age of a kidney donor affects the long-term functioning of the transplanted organ.

Generally, survival rates after kidney transplants are good: over 95% for patients and around 95% or slightly lower for the kidney itself, according to data from the Organ Procurement and Transplantation Network.

Biancone and his research team analyzed data from kidney transplants performed at the Turin University Renal Transplant Center between 2003 and 2013.

They identified a total of 647 transplants in which the donors were either between 50 and 59 years old with certain risk factors, or older than 60. Patient and kidney survival rates were comparable after an average follow-up period of 4.9 years among all the age groups studied: 50 to 59 years, 60 to 69 years, 70 to 79 years and 80 years and older.

After five years, patient survival rates ranged from 87.8% to 90.1%, while kidney survival rates ranged from 65.9% to 75.2%.

In conclusion, Biancone and his co-authors said their results support the use of older donors -- even those older than 80 -- if they've been accurately selected and managed.

Meanwhile, other researchers continue to explore new ways to expand the donor pool.

New ideas in donation

One area of increased donation has been substantial, if unanticipated.
"There's the opioid epidemic that's sweeping across the country, and in fact, many people dying of overdoses have become donors," Klassen said. "There are parts of the country where the percentage of donors who have died from overdoses is approaching 25% of the donor population, so that has been quite a change."

Another new avenue that has resulted in more transplants is the HIV Organ Policy Equity, or HOPE, Act, which allows HIV-positive donors for HIV-positive recipients. At least six hospitals in the United States are able to perform such transplants. Johns Hopkins performed the first in March with a kidney and liver transplant.

There are also efforts underway to change the way available organs are allocated based on geography. The proposal for liver transplants "would create eight liver allocation districts nationwide, with district-wide distribution of livers for candidates in the most urgent medical condition ... before local matching of less sick candidates," according to UNOS.

Researchers are hard at work for solutions as well. 

Engineering synthetic organs is making progress in laboratories, but that's long-term research, not a capability that will be available anytime soon. "That's on the far horizon, not the near horizon," said Klassen, who is hopeful nevertheless.

"There's a lot of research these days and actually clinical trials on techniques of organ profusion and organ preservation," he said. These technologies are changing the way organ preservation is done, allowing for "resuscitation, if you will, of donor organs."

Researchers are primarily applying these methods to lungs, hearts and livers because these organs are really limited by how long they can be out of the body, Klassen explained.

"A kidney can be out of the body and in cold storage for up to 48 hours. That's not a rare event," he said. "With hearts and lungs, we're talking four, six, eight hours."

Though there may be a more pressing need to explore this technique on other organs, Oxford University researchers have begun to study the effects of a process called normothermic perfusion on kidneys.

Before transplantation, scientists flush the donated kidney with oxygenated blood to revive the organ and repair any damage caused by cold storage. Additionally, the kidney is pumped with drugs, including anti-inflammatory agents, to prime it for surgery. Normothermic profusion also enables a surgeon to test kidney function, ultimately lowering the discard rate.

By contrast, other researchers are investigating new ways to preserve kidney function in patients with polycystic kidney disease, which causes small cysts to form in the kidneys. Over time, patients with this genetic disease may develop kidney scarring, which can lead to organ failure, requiring a transplant.

One researcher at University College London Medical School has begun to explore the possibility of blocking an enzyme that contributes to kidney scarring with drugs. This would keep the kidneys working and so prevent the need for a transplant.
[Read more]



PKD Research

From Taipei Times, By Wu Po-hsuan and Jake Chung


LONG-TERM STUDY:A team working for 13 years on possible treatments for chronic renal injuries is to file for a patent and is hoping to begin clinical trials soon

A research project by National Taiwan Normal University (NTNU), National Applied Research Laboratories and National Cheng Kung University (NCKU) might provide a critical first step to cures for polycystic kidney disease or similar conditions, researchers said.

The team said that the overexpression of exogenous neutrophil gelatinase-associated lipocalin (NGAL), a biomarker for acute and chronic renal injuries, had the potential to help reduce cystic progression and prolong the lifespan of people with such diseases.

NGAL is considered one of the most promising novel biomarkers to help doctors detect acute kidney injuries and chronic kidney diseases early enough to offer preventive measures, they said.

The team said that tests on mice showed that animals with kidney conditions that had an overexpression of exogenous kidney-specific NGAL also had reduced cystic progression and prolonged lifespans compared with mice with endogenous NGAL or NGAL deficiencies.

The biomarker is also associated with reductions in interstitial fibrosis and proliferation, and augmented apoptosis, the team said.

The project has been going for more than 13 years, NTNU said, adding that the team is filing for a multinational patent for its discoveries and is looking to begin clinical trials soon.

The results have also been reported by Kidney International, the official journal of the International Society of Nephrology.

NTNU said that autosomal dominant polycystic kidney disease is the most common inherited kidney disorder, affecting an estimated 12.5 million people globally, and that the condition does not have any effective treatment.

According to National Health Insurance Administration statistics, people with the condition comprised roughly 5 to 10 percent of all patients on dialysis, costing the nation roughly NT$3.4 billion (US$107 million) per year.



From Fierce BioTech

Third Rock launches Goldfinch Bio, with $55M for kidney disease work

Perennial early-stage biotech investor Third Rock Ventures has launched Goldfinch Bio with a $55 million Series A pot.

The upstart will work on discovering and developing precision therapies for patients with kidney disease.

Peter Mundel, formerly on the faculty at Harvard Medical School, joins Goldfinch as the SVP of biology, while clinical geneticist and Third Rock venture partner Phil Reilly will serve as the company’s interim CMO.

Dr. Abbie Celniker, a partner at Third Rock Ventures and 30-year industry veteran, becomes temporary CEO until a full-time head is found.

“Current treatment options for patients with progressive kidney disease are often off-label, non-specific, and carry significant toxicities,” Dr. Celniker said.

“By building on recent breakthroughs from our founders to better understand the molecular processes of kidney disease, we see a unique opportunity to spur a renaissance in the discovery and development of new therapies to treat the underlying causes of disease. To accomplish this, Goldfinch has brought together the leading scientists, clinicians, and drug hunters behind a common goal to create the first precision therapies for patients with kidney disease.”

Goldfinch will use an inclusive patient registry designed to collect and mix in genomic as well as phenotypic data, all of which is designed to help with the discovery of molecular drivers of kidney disease.

“Using state of the art biological tools, Goldfinch will focus on actionable targets for intervention, and develop precision therapies for genetically stratified patient groups,” the company said.

Goldfinch will start off working on areas where there is a known genetic component, including focal segmental glomerulosclerosis (FSGS) and polycystic kidney disease (PKD).

Sunday, December 11, 2016

PKD Life: Understanding Dialysis, Understanding Kidney Transplants, PKD Research: Regulus ADPKD Trial, Gift of Life: Sacramento, Greenville, MI, Wales

Understanding Dialysis 

From HJNews.com by Caroline Shugart

Unraveling the mysteries of kidneys, dialysis


To many people, kidneys are a complete mystery, except to produce urine. Do you understand the physiology or anatomy of your kidneys? So what happens when your kidneys fail?

Imagine this scene. You are sitting in your physician’s office, waiting for the results of a number of exploratory tests. You hear the words, “I have some bad news for you.” You hold your breath, and your physician explains, “You’re in kidney failure.” Your anxiety level is off the charts. The conversation includes words like potassium, uremia, edema, transplant, and irreversible.

Then you are told, “You need to start dialysis.” You ask, “Dialysis? What is dialysis?” Little do you realize how this will change your life. I know. I’ve worked in a dialysis clinic for over 15 years.

Currently, over 468,000 people in the United States are on dialysis. Most patients come to a clinic, typically three times a week, and are hooked up to a blood-filtering machine for an average of three to four hours each day. Adding travel and the process of being connected and disconnected to a machine, the time spent in a center can be the major part of your day.

The average life expectancy on dialysis is between five and 10 years, although patients occasionally live over 15 years on dialysis, depending on other health issues and the management of meals, physical activity, and medications.

The cost of one year of dialysis is about $80,000. With 468,000 people on dialysis, that’s a lot of money! Most patients can’t even begin to cover the cost of treatments, or other needed surgeries, medications, and emergent issues, so Medicare is available for people of all ages with permanent kidney failure requiring dialysis. Medicare ends up spending about $31 billion yearly just on dialysis treatment.

Dialysis is an unusual procedure. Nurses or certified technicians access a person’s blood supply, either through a fistula or a central line. A central line catheter is usually placed into the vena cava, in the chest, which then dumps directly into the heart. A better access is called a fistula, which is created by connecting an artery to a vein. Over about six weeks, the high pressure system of the artery develops and improves the strength of the vein. Then needles can be used to connect the patient’s blood supply in this vein, through tubing, to a dialysis machine.

A dialysis filter, or artificial kidney, is comprised of thousands of tiny straws. Each straw is a semi-permeable membrane. On the one side of the membrane is the blood supply and on the other side of the membrane is a fluid called the dialysate. Using the principle of diffusion, small solutes such as sodium and potassium, and waste products such as nitrogen and urea, pass from the blood into the solution, going from a higher concentration to a lower concentration. Excess water is pulled out of the blood using a vacuum pressure pump. The process needs to be monitored closely throughout the treatment cycle.

Your kidneys work 24/7, filtering about 50 gallons of blood daily, excreting about a quart of urine, and reabsorbing the rest. However, the kidneys don’t just regulate fluid balance and get rid of waste products. Kidneys also have important functions to make red blood cells, regulate blood pressure, protect your bones, and keep your blood from becoming too acidic. When your kidneys fail, dialysis only does about 5 to 10 percent of what your kidneys normally do for you. It’s a lot more than just producing pee. That’s why losing kidney function really impacts your health.

One third of all kidney failure is caused by diabetes and uncontrolled blood sugar levels. Circulating sugar molecules act like shards of glass, destroying delicate kidney cells called nephrons. Twenty-nine million Americans currently have diabetes, and seven million of them don’t know it. Millions more have a pre-diabetic condition, which generally advances to full-on diabetes (unless preventive measures are taken).

The second major cause of kidney disease is hypertension. Thirty-two percent of all American adults have hypertension and, unfortunately, half of them don’t know it. However, that excess pressure causes irreversible damage to the nephrons. And your kidneys work really, really hard. Some people can lose 90 percent of their kidney function without expressing any signs or symptoms. That’s why it is often a surprise when you are diagnosed with kidney failure. Your kidneys were working until they just couldn’t function anymore.

Unfortunately, the future health of Americans is a dim landscape. Of children born today, it is estimated that one out of every three children will get diabetes in their lifetime. Diabetes directly follows the obesity epidemic.

If I can leave you with one message, it is the power of knowledge. Protect your health NOW. Here is what you can do. You can maintain a healthy body weight by eating a plant-based, whole foods meal plan. You can drink water instead of sugary soda and juice. You can exercise every single day, as if your life depended on it (because it does). And you can know your numbers: your blood pressure, your blood sugar, and your kidney function.

Protect your health by protecting your kidneys. Live Well to Be Well.




Understanding Kidney Transplants

From ZeeNews, India, By Salome Phelamei

Kidney transplant: Who can have it? Procedure, risks and complications!



External Affairs Minister Sushma Swaraj, who is currently undergoing treatment for kidney failure at AIIMS, is likely to recieve a transplant next week.

According to hospital sources, the process required for pretransplant has been completed with the organ being harvested from a living unrelated donor.

Here are some key facts you need to know about kidney transplantation or renal transplantation.

What is a kidney transplant?

A kidney transplant is a surgical procedure in which a healthy kidney from a live or deceased person is transferred to someone whose kidneys no longer function. In other words, it is the organ transplant of a kidney into a patient with end-stage renal disease.
What are the common causes of end-stage renal disease?

Diabetes, high blood pressure, chronic glomerulonephritis - an inflammation either of the glomeruli or of the small blood vessels in the kidneys - polycystic kidney disease, are some common causes of end-stage renal disease.

Who can have a kidney transplant?

People with chronic kidney disease who meet certain criteria of kidney function and those on dialysis can have one, regardless of their age. Doctors will also carry out some evaluation to determine whether the transplant would be safe and beneficial for the patients. The transplant team will also check you for other serious conditions, cardiovascular disease, including chronic infections and cancer.

Different types of kidney transplants

There are two types of kidney transplantations depending on the source of the donor organ. They are-

Living-donor transplantation -- a patient gets a kidney from someone who is still alive. A living donor may be someone in your immediate or extended family, but occasionally a stranger.

Deceased or non-living-donor transplant – a person with kidney failure gets a kidney from someone who is dead but has chosen to donate his/her organs upon death.

What are the risks involved in transplantation?

There are a number of risks associated with kidney transplantations although rates of serious complications have fallen sharply in last few decades. The risks of a kidney transplant include:

Bleeding
Blood clots
Failure of the donated kidney
Rejection of the donated kidney
Infection
Heart attack
Stroke
Death

Perhaps, following a healthy lifestyle - such as eating a healthy diet, not smoking, keeping body weight in check, taking steps to reduce infections - after a kidney transplant is critical for minimising the risk of complications.

Although a kidney transplant is the most successful treatment for kidney failure, there are no 100% guarantees. Since kidney transplantation is a life-extending procedure, a person who receives a transplant may live 10 to 15 years longer than if kept on dialysis.




PKD Research

From Fierce BioTech, by Phil Taylor

Regulus adds two more projects to pipeline as it works to get lead hep C drug back on track

MicroRNA specialist Regulus has bulked up its pipeline with two new clinical candidates for liver and kidney diseases that could start human trials next year.

The two new micro RNA (miRNA) drugs—Regulus' specialty—are a drug to treat impaired bile flow (cholestasis) codenamed RGLS5040 and RGLS4326 for autosomal dominant polycystic kidney disease (ADPKD), both of which are expected to start trials before the end of 2017.

The company is keen to show that it has pipeline depth beyond RG-101, its hepatitis C virus candidate currently subject to an FDA-mandated clinical hold after serious adverse events were seen in a phase II trial. The two new drugs were highlighted at the firm's R&D event for investors yesterday.

Cholestasis therapy RGLS5040 will target diseases like primary biliary cholangitis (PBC)—which has two approved treatments—as well as primary sclerosis cholangitis and congenital disorders like Alagille syndrome which have no current therapies.

Cholestasis typically leads to serious liver damage, and for years the only treatment was ursodeoxycholic acid, which has limited efficacy. In May Intercept picked up an FDA approval for Ocaliva (obeticholic acid), which has been tipped as a blockbuster in PBC alone, with even more potential if its use can be expanded into bigger indications such as NASH, or fatty liver disease.

RGLS5040 has a different mechanism of action from the current drugs, targeting a microRNA called miR-27, and has potential both as a monotherapy and in combination with approved therapies, according to Regulus' director of biology Zhi-Liang Chu.

ADPKD candidate RGLS4326 is also targeting an underserved market with no approved drug in the U.S. and just one therapy—Otsuka's Samsca (tolvaptan)—available for use in the EU, Japan and Canada. Regulus' drug targets miR-17 and seems to reduce cell proliferation and cyst growth in a mouse model of the disease as well as an in vitro model based on human tissue.

The disease is life-threatening and affects around 12.5 million people worldwide. Around 50% of all patients go on to develop end-stage renal disease, putting them on a path towards needing dialysis and potentially a kidney transplant.

The start of trials for the two new candidates will give Regulus five candidates in the clinic alongside RG-101, AstraZeneca-partnered RG-125 for NASH and RG-012 for Alport syndrome, which is being developed with Sanofi's rare disease subsidiary Genzyme. Both RG-012 and RG-125 are in Phase II.

Updating on progress with RG-101, Regulus' CEO Peter Grint said that the company is on track to file its responses to the FDA on RG-101 by the end of the year and is still expecting a verdict from the regulator in the first quarter of 2017.

"Today's announcement of two new drug development candidates underlines the confidence we have in our rigorous targeting and validation process, which we believe should enable us to identify at least one new drug development candidate per year moving forward," he said.




Gift of Life

From The Sacramento Bee, BY ELLEN GARRISON

Their daughter died five years ago. Now they meet the woman whose life she saved.

On New Years’ Eve, two months before she died, Kaylee West told her family that if anything were to happen to her, she wanted to be an organ donor. On Feb. 6, 2011, the 24-year-old El Dorado Hills resident had a brain aneurysm in her sleep.


Linda West broke down as she embraced Jeanne Schreiber at the Golden 1 Center on Tuesday, meeting for the first time the woman whose life was saved by her only daughter’s kidney.

“I guess I should let go now,” she said, holding tight to Schreiber who also was teary eyed.

On New Years Eve, two months before she died, Kaylee West told her family that if anything were to happen to her, she wanted to be an organ donor. On Feb. 6, 2011, the 24-year-old El Dorado Hills resident had a brain aneurysm in her sleep and died.

Her kidneys and liver saved three lives, her corneas gave sight to two people and her tissue went to countless others.

The meeting between Ken and Linda West and Schreiber took place on what would have been Kaylee’s 30th birthday, at the moment she was born – 3:38 p.m. Arranged by Sierra Donor Services, the Wests met Schreiber at the Golden 1 Center because they’re huge Sacramento Kings fans and Kaylee’s favorite color was purple.

“Meeting her today, it just closes the circle,” Ken West said. “We feel like we’ve fulfilled Kaylee’s last wishes. And it’s awesome. It’s bittersweet, it’s not sad. It feels right.”

Schreiber, 60, has polycystic kidney disease, which causes numerous cysts to grow in the kidneys. While on the transplant list, she had two false alerts about available kidneys before Kaylee’s kidney was offered to her.

“(Meeting a donor family) is something you don’t know how to prepare for, it doesn’t happen every day,” she said. “I’m thrilled to meet them.”

They also got to put finishing touches on a floral portrait – a “florigraph” – of their daughter that will adorn a float in the 2017 Rose Bowl parade. Kaylee’s parents, her brothers, Kenny and Alex, and Schreiber carefully added the eyebrows to the portrait.

Kaylee’s parents have spent about 100 hours encouraging people to join the “Donate Life” registry at various events around California. The Wests stressed the importance of having the conversation about organ donation with loved ones. Kaylee didn’t have the pink donor circle on her driver’s license, so her parents wouldn’t have known her wishes if she hadn’t spoken to them about it.

“It’s just to help tell the story – not just our personal story – but the hundreds of thousands of people who are on transplant lists who never get the opportunity,” Ken West said. “That’s the reason I do it. …There’s people out there that the only way they have a chance is by somebody’s decision to be a donor. I think that’s priceless.”

As of a couple weeks ago, there were 2,632 people on the transplant list in the Sacramento region, said Deanna Santana with Sierra Donor Services. [Read more]


Read more here: http://www.sacbee.com/news/local/health-and-medicine/article119320968.html#storylink=cpy


Read more here: http://www.sacbee.com/news/local/health-and-medicine/article119320968.html#storylink=c

From Greenville Daily News, Michigan, By Emilee Nielsen


Patty Crawford and Diane Kuhn are cousins with a bond that goes beyond family into the realm of friendship and deeper than blood.

The two have been lifelong friends as well as relatives and have been there for each other through the years. When Diane needed a change in her life, she moved to California where Patty opened her home and welcomed her.

When Patty needed a kidney transplant in order to survive, Diane had herself tested for transplant compatibility without being asked.

Patty suffers from polycystic kidney disease (PKD), a disorder which causes fluid-filled cysts to grow on the kidneys. If the cysts grow too large or too many, kidney function can start to decline and eventually fail altogether. PKD is a genetic disease that afflicts Patty’s three brothers as well as her father.

Thirteen years ago, Patty found out she had PKD which came as a shock to her. She was tested along with the rest of her family in the 70s and she and her sister were the only ones found not to have the disease at that time.


A SOBERING REALIZATION

Last year, she began to feel nauseated and fatigued. Having the energy to go on about her day-to-day life was difficult and she said it got so bad at one point she had to nap at her desk on her lunch break at work.

“I was always nauseous. I threw up every morning. I had no appetite; lost weight,” Patty said. “Your kidneys do a lot of stuff…that my kidneys weren’t doing. I was anemic because my kidneys weren’t producing red blood cells. And… the cysts can burst. When they burst, that causes havoc in your abdomen area…which is very uncomfortable. It was a lot of things.”

Though it was evident that Patty needed a kidney transplant, she didn’t reach out to her family to see if anyone would undergo blood and tissue typing tests for her. It was her younger brother who made the call to Diane to see is she would be tested.

The typing tests were nothing new to Diane. She had undergone the same ones several years earlier on behalf of Patty’s younger brother; the same brother who reached out on Patty’s behalf last year. There was a problem several years ago and it didn’t work out. For Patty, though, Diane was a match.

“It’s always been about Patty. It’s always been for her. I think it was just meant to be,” Diane said.




From WalesOnLine, BYJAMES MCCARTHY

These people's lives were changed forever through organ donation


Thanks to a law change in Wales, dozens of extra donated organs have become available - and the benefits have been huge

Since an opt-out organ donation system was created in Wales a year ago, dozens more have become available to save lives.

It means adults automatically become donors after death unless they’ve specifically chosen not to in life.

Wales is the only part of the UK to have such a scheme.

The lives of these three people have been changed as a result of kidney donation - that's why they all support Wales' opt-out organ donation system.

Halima Yousuf and her brother Mohammed Yousuf


Cardiff Imam, Mohammed Yousuf, 40, was on a family holiday when he suffered kidney failure. It left him on dialysis three times a week for two years and needing a transplant.

Mohammed’s sister, Halima Yousuf, immediately offered to donate a kidney to her brother.

“When my brother ended up in hospital on holiday to Turkey, he thought it was a bout of food poisoning,” she said.

“He never imagined it to be kidney failure that would leave him on daily dialysis treatment three times a week for the next two years and in need of a transplant operation.

“I’ve seen how transplants can transform patients’ lives through my job as a hospital operation department practitioner.

“So when my brother found himself on the kidney transplant waiting list I immediately put myself forward as a potential living donor.

“I tested positive as a match and we had our operations at the beginning of October 2016.

“Mohammed’s health had deteriorated drastically in the lead up to the operation and he almost completely lost his eyesight.

“Now, thanks to the successful kidney transplant, he is reading books again for the first time in almost a year and we’re both recovering well.

“My brother Mohammed is both a local Cardiff Imam and Cardiff’s first Muslim Somali to receive a transplant.

“He’s now keen to encourage others in our community to opt in as organ donors.

“Organ donation is fully permitted in Islam, as with other major religions.

“There’s a huge need for more donors from the black, Asian and ethnic minority communities in Wales to come forward to fill the shortage and we’d encourage everyone to make time to share their organ donation decision with those closest to them.

“It really does save lives.”

Robin Simpson

Robin Simpson, 58, from Cardiff, was diagnosed with a hereditary kidney disease in 2001.

He had always avoided the subject of donation.

But when he was told a transplant was the only way to keep him healthy, the issue could no longer be dodged.

“I was diagnosed in my 40s with polycystic kidney disease,” he said.

“It took 12 years for my kidneys to deteriorate to such an extent until I was on dialysis four times a day, and the only option left for me was a kidney transplant.

“I was in work in February 2016, and about to go out for lunch, when I got the call to say a suitable match had been found and I had the transplant operation at seven o’clock that same evening.

“The recovery process wasn’t easy. I came down with an infection 10 days after the operation, and spent the next two weeks in isolation. On the last day of isolation however, I woke up feeling better than I had in years.

“Six weeks after the operation, my kidney function was dropping and showing signs of rejection.

“I immediately began treatment through a steroid drip, which was successful, and I was able to continue the recovery process.

“My transplant continues to improve, and I was able to begin a phased return to work in May, after ten weeks off.

[Read more]

Sunday, December 4, 2016

PKD Life: Potential Heart Damage during Dialysis, Donate Kidney, then Run Marathon; Save Ralph: Donor Search, PKD Report

PKD Life

From EurekaAlert

World first MRI study sheds light on heart damage during kidney dialysis


Experts in magnetic resonance imaging (MRI) and kidney disease have carried out the first ever scans to study the heart function of kidney patients while they are having dialysis treatment.

People with kidney failure need regular dialysis to remove fluid and waste products from their blood, but this process can cause falls in blood pressure and reduced blood flow to the heart. Over time this can cause long-term damage to the heart.

Research at The University of Nottingham was undertaken to investigate stress on the heart during kidney dialysis and to compare two different types of dialysis in this regard: standard haemodialysis (HD) and hemodiafiltration (HDF), a process that removes more fluid during treatment but with additional replacement fluid being given to the patient.

Experts from the University's Sir Peter Mansfield Imaging Centre (SPMIC) and the Centre for Kidney Research and Innovation (CKRI) carried out MRI scans on 12 kidney dialysis patients who were each allocated to receive both standard haemodialysis (HD) and HDF in a random order.

The study found significant cardiovascular effects with both standard haemodialysis and HDF, but no differences between the two. Results demonstrate that cardiac MRI can be a vital tool for evaluating future improvements to dialysis treatment.

Professor of Physics, Sue Francis, said: "This is the first time that MRI has been used to look at heart function while the kidney patient is actually undergoing dialysis. There were several hurdles to overcome to achieve this. We had to set up a dialysis machine in our MRI research centre, change the metal needles that go into the patient to non-magnetic silicone ones, extend and insulate the lines that connect the patient to the dialysis machine and position the equipment using our knowledge of the magnetic fields in the MRI unit."

Professor of Medicine (Nephrology), Maarten Taal, said: "Using this unique set-up we were able to monitor multiple cardiovascular measurements while dialysis was taking place in the patients. We measured how many litres of blood were pumped per minute by the left ventricle of the heart, how well the heart muscle was able to contract, blood flow in the coronary artery which supplies the heart muscle and myocardial perfusion to check the efficiency of blood flow to the capillaries or tiny blood vessels in the heart muscle."

"Interestingly, we found all measures of systolic contractile function fell during both standard haemodialysis and haemodiafiltration with partial recovery after dialysis. All patients showed some degree of left ventricular dysfunction and blood flow to the small capillaries in the heart muscle decreased significantly during both types of treatment. Our observations confirm the need for interventions to reduce the negative impact of dialysis on the heart."

Having successfully tested this method, the research team is now aiming to test the effects of other dialysis treatments using MRI.

Intradialytic Cardiac Magnetic Resonance Imaging to Assess Cardiovascular Responses in a Short-Term Trial of Hemodiafiltration and Hemodialysis is published in the Journal of the American Society of Nephrology (JASN).



From WBUR.com, by Karen Given

5 Months After Donating Kidney, Steve Russolillo Ran His Best Marathon

Steven Russolillo visits with his mother-in-law, Batya Banjo, after the surgery. (Courtesy)

Steven Russolillo is a daily columnist for the Wall Street Journal. A few years back, he met his now-wife, Shelly Banjo — she was working at the Wall Street Journal, too — and she told him about polycystic kidney disease. It runs in her family.

"It wasn’t on a third date, but you know, it was — I would say within the first year of dating this came up," Steve says. "Shelly’s mom would ultimately need a transplant at some point. The timing was unknown, It could be in a year, it could be in five years, it could be 10 years, who knows? But it was — this was just something that was on the horizon."

Steve says he just tried to do the things that good companions do: He listened, he comforted, he hoped for the best. Life went on. The disease progressed. Shelly’s mom, Batya, got sicker. Steve and Shelly’s relationship progressed, which led to happier developments.

"I proposed to Shelly in March, end of March, 2015. Really, the first thought that came to mind was, 'It has to be running related, somehow,'" Steve says. "It was a Sunday morning in Central Park, and I tried to make it as normal of a run as possible, like, 'Hey, Shell, let’s go running this morning.' 'OK, fine.'"

"How’d you hide a ring in running clothes?" I ask.

"So, I had it in the box and I put the box in this running jacket that I was wearing," Steve says with a laugh. "And I thought that I caught her glancing at it. And in my head I kind of freaked out: 'Oh, my God, she knows something’s up.'"

Shelly didn’t know. After running for about a mile, Steve stopped, got on one knee and proposed. Shelly’s sister was there to take pictures.

The Search For A Donor

But Batya Banjo was still getting sicker. Doctors told her it was time for a kidney transplant. The family had already started the search for a living donor.

"We put a flyer out. I mean, literally, we made this flyer, and Shelly posted it and posted it on Twitter," Steve says. "We actually had 11 people raise their hand and went and got tested."

Eleven people stepped up. Eleven people were medically disqualified.

"Each one, one after the other, you get your hopes up and you get really excited, and then, you get the bad news," Steve says. "I’m watching very interesting people step up. I’m watching complete strangers from the internet. I’m watching close friends. I watched my own aunt go and get tested. I’m sitting here thinking to myself, 'If all these people can raise their hands, why can’t I?'"

Why not, Steve? I can think of one reason. Shelly might need a kidney someday. The disease runs in her family. But Steve and Shelly decided to focus on the current need rather than worry about a potential need later. So, Steve went in for testing. At first, no one knew but Shelly. They didn’t want to get the family’s hopes up.

Besides, Steve had some tough questions for the doctors.

"At the time I had ran five marathons, 15 to 20 half marathons — I mean this was really important to me," he says. "It was the first thing I asked them, I said, 'Will I be able to run?' and if they had said no — oh, God. I just asked myself a difficult question. I don’t know how I would’ve responded to that. But, thank God, they said yes. So I don’t have to answer that question now. But I wanted to know what the sacrifices were ahead of time, and this would’ve been a really, really huge one for me."




From WCPO, Channel 9 Cincinnati, by Jordan Burgess

Giving thanks: Man with polycystic kidney disease waits, grateful for those who may help him one day



For Ralph Beach, even Thanksgiving doesn't bring a holiday from the treatment keeping him alive.

He spends 10 hours a day on dialysis while he waits for a possible kidney transplant. A normal, healthy kidney is about the size of a fist; Beach's doctors said his are the size of footballs. He has polycystic kidney disease, a hereditary condition.


"It causes cysts to grow in your kidneys, and over time, as the cysts grow bigger and bigger, your kidneys shut down and no longer function," he said.

The wait list for a kidney donation is at least five years, time Beach isn't sure he'll have. He's gotten more than 90 responses from possible donors since putting up yard signs asking for help, but he hasn't found a match yet.




PKD Report

From MedGadget

Polycystic Kidney Disease illuminated by New Report


Global Markets Direct’s latest Pharmaceutical and Healthcare disease pipeline guide Polycystic Kidney Disease Pipeline Review, H2 2016, provides an overview of the Polycystic Kidney Disease (Genetic Disorders) pipeline landscape.

Polycystic kidney disease (PKD) is a disorder in which clusters of cysts develop primarily within kidneys. Polycystic kidney disease symptoms may include high blood pressure, back or side pain, headache, blood in urine, frequent urination and kidney failure. The predisposing factors include age and family history. Treatment includes antihypertensive drugs and diuretics.

Report Highlights

Global Markets Direct’s Pharmaceutical and Healthcare latest pipeline guide Polycystic Kidney Disease Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Polycystic Kidney Disease (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Polycystic Kidney Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Polycystic Kidney Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 2, 1, 11 and 2 respectively for Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 2 molecules, respectively for Polycystic Kidney Disease.

Polycystic Kidney Disease (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Directs proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Download Sample Copy of This Report at: http://www.marketresearchreports.biz/sample/sample/862986

Sunday, November 27, 2016

PKD Research: Bioartifical Kidney; Stem Cells, Living with PKD: Mandy in Huddersfield, UK

PKD Research

From iTech Post, by Christie Abagon


Dutch scientists and engineers have taken a big step towards developing a bionic kidney that could one day potentially replace the need for dialysis or transplantation. They have tested a "living membrane" made with human cells that would be at the heart of a functional artificial kidney implant.


The Device Filtered Out Waste, The Same Way As A Real Kidney

The team presented the advancement at at ASN Kidney Week 2016 November 15-20 at McCormick Place in Chicago, IL. They demonstrated this activity by attaching human renal proximal tubular epithelial cells on the surfaces of artificial hollow structures. These cultured cells indeed function as a living membrane and are able to actively remove the waste products.

"This study shows the successful development of a living membrane consisting of a reproducible ciPTEC monolayer on hollow fiber membranes, an important step towards the development of a bioartificial kidney device," said Professor Dimitrios Stamatialis from University of Twente in Netherlands.


The Bioartificial Kidney Could Replace The Painful Process Of A Dialysis

In the U.S., kidney is one of the commonly transplanted organs, wherein thousands of patients are put on a waiting list for a suitable kidney donor. Another treatment for kidney disease is Dialysis, where it takes over the job of kidney if the organ fails to filter harmful wastes, salt and excessive fluids from the blood. The treatment itself does not hurt, but the needles going in can be painful.

The goal of the Bioartificial kidney is to eliminate the need of dialysis or transplant in millions of people with kidney failure, and hopefully pave the way to similar developments for bioartificial devices for other organs as well.

"The strategies and methods of this work could be relevant to development of other bioartifical organs, such as bioartificial liver or bioartificial pancreas and organs on chips - such as kidney on chip, a lung on chip or a liver on chip," Stamatialis said.




From MedicalResearch.com

Kidney Organoids Derived from Human Pluripotent Stem Cells Model Polycystic Disease

MedicalResearch.com Interview with:

Dr. Ryuji Morizane MD, PhD
Associate Biologist, Renal Division
Brigham and Women’s Hospital
Affiliated Faculty, Harvard Stem Cell Institute
Instructor, Harvard Medical School

MedicalResearch.com: What is the background for this study?

Response: Polycystic kidney disease (PKD) accounts for 10% of end-stage kidney disease (ESKD), and there is currently no curable treatment available for patients with PKD.

The adult onset form of PKD, the most common type of PKD, takes 30 years to form cysts in humans; therefore, it is difficult to study mechanisms of PKD to find novel therapeutics for patients.


MedicalResearch.com: What are the main findings?

Response: Last year, we developed a novel tool, a kidney organoid to study kidney diseases in culture plates. The kidney organoid is a 3-dimensional kidney tissue converted from human skin cells. This time, we have generated kidney organoids from patients with Polycystic kidney disease. The kidney organoids generated from PKD patients exhibited severe cyst formation with clinically relevant characteristics in 4 weeks of experiments.


MedicalResearch.com: What should readers take away from your report?

Response: These results are encouraging, as they indicate that the kidney organoid can be used to study PKD in a realistic time course of experiments to elucidate mechanisms of the disease and to find novel therapeutics in the future.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: These results indicated that the kidney organoid can be used as a novel tool to study PKD. We recommend that mechanisms of cyst formation in the kidney organoid continue to be investigated ultimately to find new therapeutic approaches for patients with PKD.

MedicalResearch.com: Thank you for your contribution to the MedicalResearch.comcommunity.

Citation:

November 2016ASN Abstract 2139

Kidney Organoids Derived from Human Pluripotent Stem Cells Contain Multiple Kidney Compartments and Model Polycystic Kidney Disease




Living with PKD

From BBC.com/News/magazine By Claire Bates

My kidneys have failed, and I know my kids face the same fate


Mandy was born with a disease that caused both her kidneys to fail

After years of pain and dialysis, she has just had a kidney transplant

Her mother, brother and two sisters have the same condition

Mandy found out her two youngest children have inherited the disease

Mandy Littlewood



Mandy Littlewood is sitting on a park bench with her husband John, their two small dogs are chasing each other up and down the sloping field.

"The kids love to come down and play here as soon as they get home from school," Mandy says.

"It's so convenient as it's just down the road from our house."

But she knows the days of playing carefree in the park are numbered. Millie, 10 and Zac, nine, have inherited a life-threatening disease from her.

Mandy has polycystic kidney disease. It causes fluid-filled cysts to grow in the kidneys over several years. Symptoms, such as chronic pain in the abdomen and urinary tract infections, usually appear between the ages of 30 and 40 but can appear as early as childhood. Sufferers are placed on dialysis and eventually need an organ transplant.

The disease is hereditary and affects Mandy's mother, her sister and two or her aunts and uncles. Her grandmother died from the disease. Someone affected by the condition has a 50% chance of passing it on to their children.

"I've never felt angry about having it as it isn't anyone's fault - it's just something that happens."

Mandy didn't realise she could pass the condition on when she had her first son, Callum, at 19. However she and her husband John knew of the risk before having Millie and Zac.

"John and I had discussed the possibility of passing on the condition but we knew we wanted kids together. I had hoped they would have been spared like my youngest sister. I felt horrendous and so guilty when they were diagnosed.

"I'm glad we got them tested though because now we know to watch their blood pressure and be extra careful if they get infections."

Mandy says the condition only really started to impact on her life six years ago when she was 33.

"I had sharp stabbing pains in my back and side almost constantly. It was too much to cope with and I was prescribed anti-depressants to help with my mood."

She had her first kidney removed in 2011 and was placed on the transplant list in 2012. A year later she was placed on dialysis, which left her feeling exhausted.

"It's taken over my life," she says.

"I had to stop working as a learning disability nurse in 2013 and then stop driving at the start of this year. I didn't like to make plans to go out, in case I had to cancel because I felt rubbish."

Mandy and her family live in Huddersfield - two taxis and a train ride from specialist kidney services in Leeds. It's an issue many people who live far from a larger teaching hospital face.

"John gave up his job as a floorer to take care of me and the kids so we have struggled financially. It costs £30 in taxis and train fare for John to come with me to a hospital appointment."

The NHS first provided patient transport to take Mandy to Leeds for dialysis as it was the closest place with a spot available. She later received dialysis at Wakefield.

"The travel was always a nightmare," says Mandy.

"The patient transport turned the four-hour session into a day-long ordeal. I would be picked up hours before my appointment and then have to wait around after it had finished. As I went three times a week I felt like I was spending my life in hospital."

After two years a spot opened up at the local satellite dialysis unit in Huddersfield. It was a great relief to Mandy. Dialysis left her feeling sick and she was given meal replacements after losing a lot of weight.

"Everything tasted metallic. The kids would come in for their tea and I would have forgotten about it because I wasn't hungry."

This summer there were 141 people in West Yorkshire and 5,066 in the UK waiting for a kidney-only transplant. Waiting times vary due to clinical need and matching between patient and donor. The average waiting time for a kidney-only transplant is 944 days.

In July this year, Mandy finally got the call that there was a possible match.

"They rang early in the morning. I had to wake John and the kids and called my mum to come and take care of them while we rushed to Leeds General Hospital."

The mother-of-three was told she would have a long wait when she arrived at hospital because it was a 'heart-beating' donor. She was then left for hours in a small TV room in St James' Hospital in Leeds.

"I know they were busy but I found that very upsetting. I later found out a lot about my donor's health problems in case I didn't want the organ. It made me feel very sad for him. He had given me such a gift."  [Read more]


What is polycystic kidney disease?

About 70,000 adults and children in the UK have Polycystic Kidney Disease

It causes fluid-filled cysts to grow and multiply in both kidneys, eventually causing them to fail

More than half of sufferers will have kidney failure by the time they are 60 years old

High-blood pressure, pain and urinary tract infections are common symptoms

The form Mandy has is the world's most common inherited life-threatening condition

For more information visit the PKD charity website

Sunday, November 20, 2016

PKD Research: Treating ADPKD with Tesevatinib, PKD Pipeline Review, PKD Mechanisms, New Kidney Organoids Methods, Pasireotide LAR: Slow ADPKD Kidney Volume Growth

PKD Research

From Business Wire

Kadmon to Present Additional Data Demonstrating Tesevatinib Safety for the Treatment of Polycystic Kidney Disease at ASN Kidney Week 2016

Kadmon Holdings, Inc. (NYSE:KDMN) (“Kadmon” or the “Company”) today announced additional data from its ongoing Phase 2a clinical study demonstrating the safety of tesevatinib, the Company’s oral tyrosine kinase inhibitor, for the treatment of autosomal dominant polycystic kidney disease (ADPKD). The data will be presented in a poster session on Saturday, November 19 at the American Society of Nephrology (ASN) Kidney Week 2016 in Chicago, IL.

Recent findings from Kadmon’s ongoing Phase 2a study in patients with ADPKD have demonstrated that tesevatinib is well tolerated and have also identified tesevatinib 50 mg once daily (QD) as the optimal dose to treat ADPKD.

New data reported in the poster indicate that tesevatinib is a new member of a group of drugs known as MATE 1/2-K transporter inhibitors, such as cimetidine, which mildly increase levels of serum creatinine. Normally, an increase in serum creatinine can be used to indicate kidney damage, but in the case of MATE 1/2-K inhibitors, these serum creatinine increases occur without clinically meaningful alterations in renal function.

Specifically, new data demonstrated that serum creatinine levels in tesevatinib patients increased by 10% to 14% during the first 28 days of treatment and reversed upon treatment discontinuation. Importantly, levels of cystatin C, another measure of renal function, were relatively unchanged during the same period. In vitro studies demonstrated that tesevatinib potently inhibits MATE1/2-K transporters at tesevatinib concentrations achieved with the 50 mg QD dose. Therefore, MATE inhibition by tesevatinib may explain mild serum creatinine increases associated with tesevatinib treatment.

“Kadmon has identified a contradiction in PKD drug development: MATE transporters such as tesevatinib increase levels of creatinine, the standard measure of PKD drug efficacy, but without clinically relevant effects on kidney function,” said James Tonra, Ph.D., Senior Vice President, Nonclinical Development at Kadmon and first author of the poster. “These findings further characterize the safety of tesevatinib while highlighting the need for alternative methods to evaluate kidney function in PKD drug development.”

“We believe these findings support the continued development of tesevatinib for PKD,” said John Ryan, M.D., Ph.D., Executive Vice President and Chief Medical Officer at Kadmon. “Kadmon has had discussions with the FDA to develop innovative methods to demonstrate drug safety and efficacy in PKD, a major unmet medical need. These discussions will have important implications for clinical trial designs for tesevatinib as well as other therapies in development for renal diseases, especially MATE inhibitors.”




From WhaTech

Recent report on polycystic kidney disease - pipeline review, H2 2016 just published

Polycystic Kidney Disease - Pipeline Review, H2 2016 is a new market research publication announced by Reportstack. This report provides an overview of the Polycystic Kidney Disease (Genetic Disorders) pipeline landscape.

Polycystic kidney disease (PKD) is a disorder in which clusters of cysts develop primarily within kidneys. Polycystic kidney disease symptoms may include high blood pressure, back or side pain, headache, blood in urine, frequent urination and kidney failure.

The predisposing factors include age and family history. Treatment includes antihypertensive drugs and diuretics.

Report Highlights

This report Pharmaceutical and Healthcare latest pipeline guide Polycystic Kidney Disease - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Polycystic Kidney Disease (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Polycystic Kidney Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Polycystic Kidney Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 2, 1, 11 and 2 respectively for Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 2 molecules, respectively for Polycystic Kidney Disease.

Polycystic Kidney Disease (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from a proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources.

Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.




From Nephrology News

Method to create kidney organoids from patient cells provides insights into polycystic kidney disease

Scientists have developed a method to coax human pluripotent stem cells to mature into cells that go on to form the functional units of the kidney and demonstrated how the method can be used to study human kidney diseases

A new method to create kidney organoids from patient cells may provide insights into how kidney diseases arise and how they should be treated. The research is presented at ASN Kidney Week 2016 November 15-20 in Chicago.

Previously, Ryuji Morizane, MD, PhD, from Brigham and Women’s Hospital, and his colleagues developed a method to coax human pluripotent stem cells (hPSCs) to mature into cells that go on to form the functional units of the kidney.

In their latest work, they show how their method can be used to study human kidney diseases. Researchers used hPSCs derived from patients with autosomal recessive polycystic kidney disease (ARPKD) to generate kidney organoids that possessed tubules with large cysts like those seen in patients with the disease.

“Establishment of a novel platform to model ARPKD using human kidney organoids will facilitate studies on mechanisms of cyst formation and contribute to the development of chemical screening systems to find potential therapeutic agents for polycystic kidney disease,” said Morizane. “Also, our organoid system enables in vitro studies of kidney pathophysiology, nephrotoxicity assays, and disease modeling, and ultimately will lead to development of bioengineered kidneys for regenerative medicine.”

Study: “Kidney organoids derived from human pluripotent stem cells contain multiple kidney compartments and model polycystic kidney disease” (Abstract 2139)




From Northwestern Medicine, BY SARAH PLUMRIDGE


A new study published in the journal Cell provides insight into potential mechanisms for the activation of a mutated gene in autosomal dominant polycystic kidney disease (ADPKD), a life-threatening genetic disorder that afflicts more than 600,000 Americans and more than 12 million people worldwide.

The gene, named polycystic kidney disease 2 (PKD2), codes for a protein that is a part of a large ion channel and sits on the membrane of cells. A team of scientists, including co-author Paul DeCaen, PhD, assistant professor of Pharmacology, used a type of microscopy called single-particle electron cryo-microscopy combined with nanodisc technology to determine the structure of the PKD2 ion channel.

The structure described in the paper establishes the molecular basis for the majority of disease-causing mutations in PKD2-related ADPKD. They show how the extracellular domain (outside of the cell) of PKD2 — a hotspot for mutations — contributes to channel assembly and may serve as a location for extracellular stimuli to bind to and affect the function of the channel’s gates, which monitor ion flow.

They further uncovered details of the channel’s current and regulation of the flow of ions, or the permeation pathway, using electrophysiology methods.

“Although we know the PKD2 dysfunction leads to ADPKD, we don’t know how to treat patients,” DeCaen said. “This work provides a structural template for drug design, which is an exciting possibility since none currently exist.”

Currently, there are no treatments for ADPKD; right now, treatment can only alleviate some symptoms of the disease. A better understanding of PKD2 structure and function could further the development of better treatments, according to the authors.

The study was supported by the National Institutes of Health NIDDK Pathway to Independence (PI) Award (K99/R00), Howard Hughes Medical Institute, and National Institutes of Health grant R01 DK110575-01A1.




From EMPR.com, Boston, MA

Pasireotide LAR Slows Liver, Kidney Volume Increase in PLD, ADPKD

The investigative agent, pasireotide LAR (SOM230) was found to delay the progressive increase in liver volume and total kidney volume in patients with polycystic liver disease (PLD) and autosomal dominant polycystic kidney disease (ADPKD), in a study presented at The Liver Meeting® 2016.

Octreotide LAR, a somatostatin receptor analog, has been reported to reduce liver volume, improve quality of life in symptomatic PLD and slow glomerular filtration rate (GFR) decline in ADPKD. Maria V. Irazabal, MD, and colleagues from the Mayo Clinic, Rochester, MN, aimed to assess the safety and efficacy of pasireotide LAR in severe PLD and ADPKD. Pasireotide, a novel multi-receptor ligand somatostatin analog, possesses a "broader binding profile and higher affinity to known somatostatin receptors with potential for greater efficacy."

In the 1-year, double-blind randomized trial, 48 study patients were assigned 2:1 to receive pasireotide LAR 60mg or placebo every 28 days; patients were stratified by ADPKD and ADPLD.

"Primary endpoint was change in liver volume; secondary endpoints were change in kidney volume, estimated GFR [eGFR], and quality of life [QOL]," explained Dr. Irazabal. Forty patients completed liver volume measurements at 12 months.

The researchers reported a 3.3% decrease in annualized change in liver volume (4271 ± 2373mL to 4104 ± 2265mL) in the pasireotide LAR group vs. a 6.3% increase in liver volume (4047 ± 1298mL to 4294 ± 1314mL) in the placebo group (P=0.001).

Regarding kidney volume, researchers reported a 1.1% decrease in the pasireotide LAR group vs. a 3.9% increase in the placebo group (P=0.024).

"Changes in eGFR were not different between groups," noted Dr. Irazabal.

Pasireotide LAR the target dose, was reached in 87.5% of patients within the first year. Patients in the pasireotide LAR group, however, experienced a higher incidence of adverse events such as hyperglycemia (73% vs. 20%; P=0.003) and diabetes (30% vs. 0%; P=0.042) compared to the placebo group.

Overall, pasireotide LAR slowed the progressive growth in both liver and total kidney volume in patients with PLD and ADPKD. Bigger studies are warranted to determine the impact of somatostatin receptor analogs on eGFR decline, concluded study authors.




From News-Medical


The tiny fruit fly can help humans investigate the genetic and neural bases of detecting painful or harmful cold stimuli and offer intriguing, potential implications for human health, according to a new study.

A team of researchers led by Dr. Daniel N. Cox, associate professor of neuroscience at Georgia State University, has discovered that fruit flies have cold-sensing neurons that when activated drive specific, aversive behaviors to damaging cold, which requires the function of evolutionarily conserved ion channels known as Transient Receptor Potential (TRP) channels.

In the journal Current Biology, the researchers establish the fruit fly, Drosophila melanogaster, as a powerful genetic and behavioral model for unraveling questions about the cellular and molecular bases of damaging cold perception, which have not been well understood.

The study explores the concept of nociception, the peripheral and central nervous systems' perception of painful or potentially tissue damaging stimuli, which is generated by activating sensory nerve cells called nociceptors. This evolutionarily conserved process is critically important for survival.

Nociception, coupled with pain sensation, alerts an organism to possible environmental dangers and allows it to execute behavioral responses to protect against incipient damage. Acute and chronic pain can manifest as altered nociception in neuropathic pain states.

The study found that one of the implicated TRP channel genes called Pkd2 has been causally linked to autosomal dominant polycystic kidney disease (PKD), the most common monogenic disease in humans. Pkd2 ion channels appear to function as cold sensors and misexpression of Pkd2 can confer cold sensitivity to normally insensitive neurons. While it is not yet known if PKD patients have cold nociception defects, these new findings suggest this merits further investigation as a potential non-invasive diagnostic.

These same cold-sensing neurons also function as mechanosensors for touch, revealing that they, as well as the TRP channels identified in this study, are multimodal and raising the question of how neurons and ion channels distinguish between harmless and harmful stimuli to drive specific behavioral responses. Using sophisticated optical assays of neural activation by touch versus cold stimuli, the researchers demonstrate that these sensory neurons have different activation thresholds, with touch having a low threshold and cold having a high threshold, that ultimately determine the appropriate behavioral response.

"This new model sets the stage for uncovering evolutionarily conserved molecular control of nociception," said Cox. "It also provides a powerful genetic platform for unraveling the neural circuitry and molecular mechanisms that integrate multimodal sensory input to produce specific behaviors in response to diverse environmental stimuli."

The research team included Kevin Armengol, Atit A. Patel, Nathaniel J. Himmel, Luis Sullivan, Dr. Srividya C. Iyer and Dr. Eswar P.R. Iyer of the Cox Lab at Georgia State's Neuroscience Institute and Center for Behavioral Neuroscience, and collaborators Heather N. Turner and Michael J. Galko from MD Anderson Cancer Center.

The next steps will be to dissect the neural circuitry, additional molecular players and synaptic mechanisms that modulate cold nociception and multimodal sensory processing.