Participate in PKD Clinical Studies

PKD Clinical Studies

From PKD Foundation

Clinical Studies

For drugs and therapies to be developed for PKD, clinical studies must be conducted. Patients play a key part. Learn how you can help unlock the secrets of PKD and find a treatment by participating in a study.

Participate in PKD Clinical Studies to Discover Treatments and Improve Your Health

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Types of Clinical Studies

The two types of studies currently underway for PKD patients include observational studies and clinical trials.
Observational studies monitor and record various clinical and/or subjective factors over a period oftime, like blood pressure or quality of life, to better understand how the disease progresses. Involvement includes activities such as having conversations with researchers and physicians, or recording daily events in journals. No drugs are given or other interventions are made to alter the course of the disease. An example of an observational study is the Consortiumfor Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP).
Clinical trials monitor and record various clinical and/or subjective parameters by testing experimental treatments, devices or combinations of drugs that may alter the course of the disease. Participants receive specific interventions which may be medical products such as drugs or devices; procedures; orchanges to participants’ behavior such as diet. The purpose of a clinical trial is to test a new drug or other intervention for safety and effectiveness in treating the disease in question before it can be prescribed for patients. An example of a clinical trial is theTolvaptan TEMPO ¾ Trial.

According to clinicaltrials.gov, an intervention is a processor action that is the focus of a clinical study. This can include giving participants drugs, medical devices, procedures,vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches such as surveys, education and interviews.

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Phases of a Clinical Trial

A new drug to treat PKD must move through each stage before it can be reviewed for approval by the FDA.
Phase I – The new drug is tested for safety and side effects in a small number of healthy volunteers withPKD.

Phase II – The new drug is tested for safety, doseranging and preliminary effectiveness in a small number of volunteers.

Phase III – The new drug is tested in a large number of volunteers with PKD to establish effectiveness, monitor side effects and compare results with currenttreatments. The data collected during the clinical trial is analyzed and then submitted to the FDA for regulatory review, which can take one to three years. If there is an intent to apply for an NDA (new drug application), it is communicated to the FDA and shapes the design of the study. Once it is approved, the new drug can be prescribed by physicians to treat PKD.

Phase IV – These studies are done after the drug has been approved by the FDA and it is in use (considered a post-marketing study).Clinical Trials Awareness Program (CTAP)

Perhaps the single greatest impediment to clinical trial patient participation/recruitment begins with awareness. We believe it is our goal to address this challenge and launched the Clinical Trials Awareness Program, an aggressive program to help create awareness amongst patients and families and speed up clinical trial recruitment. The program is focused on educating patients about current studies so they can make educated decisions about participating. The goal is to simplify the process of finding clinical studies for PKD patients in their geographic area by sending Accelerating Clinical Trials (ACT) Alert emails about studies that are being conducted.

Sign Up to Receive ACT Alerts

Learn more about the Clinical Trials Awareness Program

Participate in a Clinical Trial

To participate in a clinical trial, you will need to have a formal diagnosis of PKD made by a physician. Learn more about the implications of a PKD diagnosis for yourself or your children.

By participating in a clinical trial, you can:

Play a more active role in your own health care.
Have access to new treatments before they are available to the public.
Contribute to the development of treatments for PKD.
Make things better for the next generation of those with PKD.



PKD Research Funding

From Targeted News Service, by MARY GRACE DEJUCOS

$4.4 Million Funding Available for Polycystic Kidney Disease Research and Translation Core Centers

WASHINGTON, July 22 -- The U.S. National Institutes of Health has announced that it expects to award a maximum of four discretionary grants to support research for polycystic kidney disease.

For more information, contact NIH OER Webmaster, FBOWebmaster@od.nih.gov. NewsFocusCode FedGrants NWNAgrants federalgrants TNSFG140721

PKD Fundraising

From Topeka Capital Journal, Topeka, Kansas

Bass tourney to raise funds for kidney transplant

A catch-and-release bass tournament and chili cookoff will be from 8 a.m. to 3 p.m. Aug. 2 in the Coeur d’ Alene Shelter at Melvern Lake. Other activities include face painting and a bake sale.

Proceeds will benefit the Midwest/West Kidney Transplant Fund in honor of Tom Thurston, who has suffered from polycystic kidney disease since birth. Over the past few years, his kidneys have become increasingly enlarged and recently began to shut down. He is awaiting a kidney transplant.

Even with insurance, Thurston’s out-of-pocket costs will be about $2,000 a month for the rest of his life. He is working with the nonprofit HelpHOPELive to raise funds to cover uninsured medical costs associated with the transplant.

Registration fees are $50 for the bass tournament and $20 for the chili cookoff. The event is free for spectators.

Donations to Thurston’s fundraising campaign can be made online at helphopelive.org or by calling (800) 642-8399. Checks can be mailed in honor of Thurston to HelpHOPELive, 2 Radnor Corporate Center, 100 Matsonford Road, Suite 100, Radnor, Pa. 19087.

From TribLive, Fayette, PA

PKD Walk

The fourth annual Walk for PKD in Memory of Chuck Bryner will take place at Yough River Park on Aug. 2. Polycystic Kidney Disease is one of the most common life-threatening genetic diseases. There is no treatment or cure. The Walk for PKD is the PKD Foundation's largest annual fundraising event; these funds make up a third of the group's annual budget for research. Join the walk as a participant, volunteer or donor. Questions or comments can be directed to Janet Bryner at 724-434-4821.

Dialysis Research

From abc30 News, Fresno, CA

NEW HOPE FOR DIALYSIS: BIOENGINEERED BLOOD VESSELS

Nearly 400,000 Americans are on dialysis for kidney failure. The treatment uses a special machine to filter toxins from the blood and often requires a graft to connect an artery to a vein to speed blood flow. But in many patients, synthetic grafts lead to infection and frequent hospitalizations. Now a first-of-its-kind bioengineered blood vessel is changing that.

William Alexander has suffered with kidney failure for 15 years. Dialysis keeps him alive.

"(It's not) like you can't do it," Alexander told ABC30. "You've got to have dialysis to live."

However, his arm tells the story of failed blood vessel grafts used to help clean his blood.

"It's disfiguring," Jeffrey Lawson, MD, PhD, Professor of Vascular Surgery and Pathology at Duke University Medical Center, Durham, N.C., told ABC30.

It's a reality Dr. Lawson says most patients face.

"I tell many of my patients they can expect to have a procedure related to dialysis at least once a year," Dr. Lawson explained.

Now, a new bioengineered blood vessel using donated human cells could change that.

"We'll be able to reduce the number of interventions they have to have," Dr. Lawson said.

At the lab, Dr. Shannon Dahl says donated cells are placed in a bioreactor and cultured for two months.

"So we're growing the cells and we're putting the bioreactor parts together," Shannon L.M. Dahl, PhD, Vice-President, Scientific Operations, cofounder, Humacyte, Inc., Durham, N.C., told ABC30.

Once the vessel is formed, it's cleansed of the donor cells, leaving a collagen structure that the body readily accepts as its own.

"It then becomes your blood vessel as your body grows into it, which is very, very exciting," Dr. Dahl explained.

Alexander had the bioengineered vessel placed in his right arm eight months ago.

"I don't have any trouble and it's doing good, and I'm glad it's doing good," Alexander said.

If you would like more information, please contact:

Sarah Avery
Duke Medicine News and Communications
Office: 919-660-1306
Cell: 919-724-5343
sarah.avery@duke.edu

Real PKD Cheerleader

PKD Cheerleader

From Daily Mail, United Kingdom, By ANNABEL FENWICK ELLIOTT

Mom with incurable kidney disease defies all odds to make NFL cheerleader squad - at age 40

Kriste Lewis joins 35 other women on the New Orleans Saintsations team, some as young as 19, and most in their early to mid-20s.

She is the oldest cheerleader in her team's history, and the second oldest ever to make an NFL squad

A 40-year-old mother-of two-has scored herself the prized position of an NFL cheerleader, beating out competitors half her age to join the New Orleans Saintsations.

Kriste Lewis, from Hattiesburg, Mississippi, was diagnosed with polycystic kidney disease 14 years ago, and as she approached her 40th birthday in April, she decided it was now or never if she wished to tick this lifelong dream off her bucket list.

'Today is the day, not tomorrow,' she told the Hattiesburg American. 'Wear your best shoes. Don’t save that bottle of wine... I know that I will be on dialysis and in need of a transplant, but I could be killed in a car wreck tomorrow.'

The feisty dancer, who appeared on ABC's Good Morning America this morning with her husband Tim, and sons Jake 14, and Rob, 11, trained hard for six months ahead of the try-outs; an experience she would have been happy to settle with as a birthday present to herself on its own.

Mrs Lewis did not disclose her age during the first round of nerve-wracking auditions in April, and she held on to her husband's motto 'focus and fun' to keep her eye on the prize.

She impressed the judges strictly based on her dancing abilities alone, and two rounds later, it was unanimously decided that she should make the team.

'I had honestly forgotten how much I loved to dance,' Mrs Lewis, who works part-time as a fitness instructor, admits. 'I told my husband, "Please remind me after this audition to not stop dancing."'

'It's just amazing to watch her,' Mr Lewis told ABC.  [Read more]

Gift of Life

From Fox31, Denver, Colorado, BY SHAUL TURNER
Kidney donation network helps patients move up transplant list

GREELEY, Colo. – Polycystic kidney disease was causing the pain that Bertina Marie Hofmeister lived with each day.

“I started getting really bad back pain,” she said. “I didn`t know what was going on.”

Bertina needed a kidney transplant and was willing to wait for it rather than ask one of her kids.

“I’m not sure how other mothers feel about it, but it`s your child and you don`t want to see your child go through any kind of unnecessary surgery,” she said.

Her youngest child, Amanda, didn’t agree with that. She tried to donate her kidney without telling her mother, but learned that she wasn’t a positive match. All hope was not lost, though: Doctors at the University Of Colorado Hospital told Amanda and her mother about another option called pair donation.

Through a computer system, mismatched donors and recipients are grouped with others across the country. If there is an incompatible pair in Texas, Los Angeles, Boston or other cities, the system can find compatibility elsewhere.

“The pair donation network is an amazing way for pairs of people who want to actually contribute or donate a kidney to their loved ones but are unable to,” Dr. Alex Wiseman of CU Hospital explained.

Amanda’s donation to a different recipient helped move Bertina’s chance of getting a donor to the top of the list.

“It didn`t matter that my kidney went to a stranger as long as my mom got hers,” Amanda said.

Amanda’s kidney was shipped to Seattle, and Bertina received hers from Georgia. A selfless act that saved not only one life, but two.

It has been a year now since their surgery and both mom and daughter are doing great. Amanda is finishing her police academy training in Greeley and Bertina is expecting her third grandchild any day.

For more information about the national kidney registry pairing program, visit http://www.kidneyregistry.org/compatible_pairs.php?cookie=1

From MLive.com, by Justin P. Hicks

Saving a stranger: Kidney donor discovers 'tremendous need,' helps man he just met

GRAND RAPIDS, MI – John Teeples still can't explain the decision he made last fall shortly after meeting Arthur Bott, a 79-year-old Byron Center man struggling with kidney failure.

“He needed it and I had two,” Teeples, a 57-year-old attorney who practices in Georgetown Township, said of the choice to give Bott a kidney “I’ve never been able to explain why I said 'yes' to a man I basically just met. It was just, I was in a position to help a man and I did.”

Bott knew his health was declining after 20 years of battling polycystic kidney disease. He was preparing for the worst before Teeples offered him a lifeline after they connected through a mutual friend.

Seven months later, the chief executive of Grand Rapids Plastics is back to work and Teeples is no worse for the wear.

“I’m doing fine. I have my health. I have a new kidney,” Bott said. “It’s the darndest thing, but the stranger that knew me (a few) weeks said ‘I just have to save your life.’ Who does these things?”

Related: Grand Rapids Plastics owner who 'flunked retirement' earns lifetime achievement award

Teeples said he heard people talk about donating their organs postmortem, but didn't realize there was such a “tremendous need” for living kidney donors. He hadn’t researched the option much until meeting Bott.

Most kidney donors know their recipient, but living donor coordinator Simie Bredeweg said Mercy Health Saint Mary’s Kidney Transplant Program sees a handful of anonymous donations a year.

Random donors like Teeples help alleviate both Michigan's 2,653-patient wait list that can keep a person hoping for a transplant for five years. The national list is more than 101,000 patients, according to Gift of Life Michigan and the U.S. Department of Health and Human Services.

“The wait list has not gotten shorter (in recent years) and there are definitely not more organs available than there used to be,” Bredeweg said. “There are more people with kidney failure and the resources have not increased to meet the demand.” [Read more]

From ABC7, Denver, Colorado, By Julie Pursley

Woman gives life to fiancé; donates kidney ahead of their wedding

INDIANAPOLIS - An Indiana woman is giving a part of herself to her fiancé to improve his quality of life as their wedding nears.

Jeff Graham and Donell Heberer Walton already have a bond that will connect them for a lifetime.

With Jeff's kidneys shutting down because of polycystic kidney disease, Donell made the decision to give him another chance at life after doctors at St. Vincent Hospital determined she was a match for a successful kidney donation.

"She's actually giving me something to prolong my life, so I'm just very thankful and blessed," Graham said.

"I am one of those people who believes all things happen for a reason," Walton said. "You are where you are meant to be at that moment."

Without a transplant, Graham would have needed dialysis.

"That's selfless. That's noble," said Dr. Alvin Wee." The act that they do is just heroic, if you ask me."

After months of doctor visits and testing the transplant team at St. Vincent removed a healthy kidney from Walton using what they described as a relatively simple laparoscopic procedure.

The delicate surgery was completed just steps away in another operating room when the kidney was placed in Graham. The procedure will allow him to carry on a normal life.

"God works through us to help others, and God works through these donors to change the lives of these recipients," said Dr. Islam Ghoneim.

Walton's gift to Graham gives him new perspective.

"I appreciate life a lot more. You appreciate that and what she has given up to help me and prolong my life," he said. "I will be forever grateful for what she's done."

Since the transplant, Graham can now go on long-distance trips. The couple recently traveled to Hawaii and has bigger plans.

"I guess we'll start saving our pennies maybe for Europe now," he said.

Graham and Walton are telling their story to help raise awareness to the importance of living donors. [Read more]

From ABC13, Houston, Texas, By Natasha Barrett

10 PEOPLE DONATE OR RECEIVE NEW KIDNEYS IN 10 DAYS AT MEMORIAL HERMANN

HOUSTON (KTRK) --Ten people over 10 days all received or donated a new kidney to a loved one at Memorial Hermann-Texas Medical Center.

On the day of their first check-ups, ABC-13 spoke with seven of them.

Patricia Bolar and Debra Garrett-Graves aren't just sisters. Bolar raised her 10 siblings when she was just 18 years old.

Their mother died from polycystic kidney disease, and Bolar was born with it. She was on dialysis for two years before receiving her sister's kidney.

"She was like when you're ready for your kidney. I got it. And, I was like okay," Bolar said. "I love her for doing this for me. I couldn't have asked for a better gift."

Garrett didn't hesitate to step in and help her sister who had given so much to her.

"It makes me feel even better to give back because she sacrificed a lot for me, so why can't I sacrifice for her?" Garrett-Graves said. [Read more]

From ABC6, Knoxville, Tennessee, By LORI TUCKER 

Brother and sister with kidney disease demonstrate why East Tennessee Kidney Foundation is important

KNOXVILLE (WATE) - A brother and sister who know all too well what it's like to have kidney failure are a shining example of why we chose the East Tennessee Kidney Foundation for our 6 On Your Side Day of Service project for July.

"Sometimes it's sort of like a frog. You put it in warm water, and as the water temperatures goes up, the frog doesn't really know what's going on," said Link McGinnis. He is talking about kidney failure caused by polycystic kidney disease.

He and his sister Vicki Geist inherited inherited the disease, in which cysts grow on the kidneys, causing them to slowly shut down over time.

"I took procreate shots that they use for chemo patients to help them get through the day," said Geist.

"Our failure was slow and it took a lot of time. If you saw us from one year to the next, you could see quite a bit of difference," McGinnis said.

All that changed for this brother and sister almost one decade ago when they found out they were going into kidney failure.

"We both started looking for living donors and put the word out, and had a lot of people offer to donate kidneys to us and get tested," said Geist.

It wasn't long before two living donors were found to be a match.

"It's just incredible for someone to see a need and say, 'I can do that,'" said McGinnis.

6 News was at Vanderbilt University Hospital in Nashville in 2005 when McGinnis went through his surgery.

"Probably I wouldn't be standing here if it wasn't for organ donation," he said.

Polycystic kidney disease is an unwanted legacy in the family, first starting with their grandmother who died from it and then passed to their mother.

Their mother Lois had a kidney transplant 20 years ago. Now mother, daughter and son are living full and healthy lives.

"Of course modern science has come a long way, and once you get a new kidney, you really are just as good as anyone else," said Geist.

"My life has completely changed because of my donor," McGinnis said.

This family is not alone. The numbers are staggering. According to Donate Life Tennessee, almost 100,000 people in the United States are living with kidney failure, and of those, only about 17,000 received a transplant last year.

That number in Tennessee is 23,000, with less than 500 transplants last year.

More online: 6 On Your Side Day of Service to benefit East Tennessee Kidney Foundation with blanket drive. [Read more]

PKD Fundraising

From The Daily Observer, Canada, by Sarah Hyatt

Slo-pitch for kidney disease raises $4,000

Right now, two million Canadians are living with kidney disease or are at risk.

Every day, 14 Canadians learn that their kidneys are failing.

And the devastation of a diagnosis can strike anyone, at any time.

About nine years ago, a father of three here, a husband, a friend to many, Don Sutherland was one in 14, and he and his loved ones experienced the devastation of such a diagnosis.

Don was diagnosed with polycystic kidney disease. It’s a genetic disorder that causes multiple cysts to form in the kidneys. High blood pressure, urine and kidney infections, kidney stones and kidney failure often and eventually follow the diagnosis. For the Sutherland family there was and remains no cure.

After the diagnosis, Don and his family learned his kidneys would fail within two to five years. And they did.

Dialysis and/or a transplant are the only hope for someone with a diagnosis like Don’s, Cathy, Don’s wife explained. But neither will cure the disease – both simply prolong one’s life, she continued. And either aren’t without their own set of hardships.

Don went for dialysis three times a week with Cathy in Renfrew after he was first diagnosed. Each dialysis treatment took hours.

Cathy remembers cooking a lot of suppers in advance during that time for those three nights a week at the hospital, she said. Their family dog often accompanied them too for the trips to Renfrew. “We just spent so much time there,” she said. Everything changed for the family.

While Don was going through dialysis treatments, both Cathy and his sister went through months of testing to see if either were a match to donate a kidney to Don. And on Oct. 30, 2008, Don got a new kidney from Cathy.

But there’s still no way of telling how long the transplanted kidney will work for, Cathy said. For each person it’s different, she explained. Sometimes a transplant can work for a day, sometimes 25 years, but it won’t last for the rest of Don’s life, Cathy said. There’s also a lot of medication involved to prevent rejection in the case of a transplant, Cathy added.

However, Don continues to do well to date. “He’s a lot healthier,” Cathy said. “He feels a lot better.”

For Don’s daughter Heather, she’s happy her dad is able experience great quality of life with his transplant.

The battle isn’t really over though for the Sutherland family and others just like them, sharing the same devastation of a diagnosis. And with that, for the past few years the Sutherland family has rallied people together to fight back.

On the weekend, they hosted their second annual slo-pitch tournament to support people affected by kidney disease. [Read more]

PKD Help

From TheSpec.com, Hamilton, Canada

Too Good to Miss

PKD support meeting, July 20, 2 p.m., St. Joseph's Healthcare Hamilton, Classroom B (T2208), 2nd Floor, Juravinski Innovation Tower, 50 Charlton Ave. E. A webinar by Robert Weiss, M.D., Professor of Medicine at UC Davis, and Chief of Nephrology at Sacramento VAMC, will help address some of what causes cardiovascular issues in people with PKD.

New Proposed Medicare Rule Raises Concerns About Dialysis Payments

Kidney Dialysis Issues

From Nephrology News 

Kidney Care Partners expresses concern about proposed rule for ESRD payments

Kidney Care Partners said they are concerned that a combination of flat payments and increased regulatory pressures proposed by the Centers for Medicare & Medicaid Services may negatively impact stability and affect quality of care in Medicare's end-stage renal disease program. KCP is a coalition of kidney patient advocates, clinicians, care professionals, dialysis providers, researchers, and manufacturers.

CMS released a proposed rule July 2 that would increase payments to dialysis facilities by 0.3% to 0.5%, while rural facilities will receive a decrease of 0.5%. CMS projects that ESRD facilities in Puerto Rico and the Virgin Islands will receive a 3.6% decrease in estimated payments, based on the proposed rule.

"On initial review, KCP appreciates the agency's interpretation of the kidney provisions contained in the Protecting Access to Medicare Act of 2014 (PAMA) to stabilize rates through 2015, though we remain concerned that despite these actions, Medicare reimbursement for dialysis care is not keeping pace with the rising costs of delivering quality health care to individuals with kidney disease," said Dr. Edward R. Jones, Chair of KCP. "While the proposed rule implemented Congressional intent by modifying deep cuts planned for 2015, many dialysis facilities continue to face economic hardship."

In recent years, the Medicare ESRD benefit has not received an inflation adjustment from CMS, which when coupled with sequestration cuts and other reductions by CMS, has resulted in Medicare reimbursement that is below the cost of care for most patients, according to KCP. "Without adequate Medicare funding, providers and physicians are forced to make difficult choices regarding staffing, quality improvement interventions, and ultimately whether to keep a center's doors open to patients," KCP said in a statement.

"KCP supports efforts to ensure that payments reflect the actual cost of providing care so that our community can continue delivering high quality dialysis care and sustained quality improvements," said Jones. "We will continue our diligent work with the broader kidney care community to submit detailed comments to the proposed rule during the public comment period."

Jones said that KCP will devote significant time and attention to the portion of the proposed rule pertaining to quality. "The Quality Incentive Program has tremendous implications for care quality and economic stability. We look forward to working with CMS to ensure that the final rule contains policies that continue to elevate the quality of patient care," Jones said.

From Medical Research.com

End Stage Kidney Disease: Does Peritoneal Dialysis Offer A Survival Advantage?

MedicalResearch.com Interview with:
Victoria A. Kumar, M.D.
Internal Medicine/Nephrology
Division of Nephrology
Department of Internal Medicine
Southern California Permanente Medical Group
Los Angeles, California, USA


Medical Research: What are the main findings of the study?

Dr. Kumar: There was over a 2 fold increase in patient survival in incident peritoneal dialysis patients in the first year on dialysis compared to propensity matched incident hemodialysis patients. We excluded any patients who utilized a central dialysis catheter at any point during the first 90 days on hemodialysis in an effort to reduce the mortality bias associated with hemodialysis patients who start with a catheter. All hemodialysis patients had pre-dialysis care by a nephrologist prior to starting dialysis.

The 2+ fold increase in survival among peritoneal dialysis patients resulted in a 2-3 year cumulative survival advantage for peritoneal dialysis patients, using both intent to treat and as-treated analyses.

Medical Research: Were any of the findings unexpected?

Dr. Kumar: Given that a couple of recent reports had attributed the initial 1-2 year survival advantage seen in PD patients in several large studies to the early use of central venous catheters among matched hemodialysis patients, our findings were surprising. Other authors have attributed the initial 1-2 year survival advantage seen in other studies to lack of pre-dialysis care, but all of our study patients were managed by a nephrologist prior to starting PD/HD.

Medical Research: What should clinicians and patients take away from your report?

Dr. Kumar: The survival advantage afforded by peritoneal dialysis in the first couple years on dialysis should be emphasized when patients are in the process of choosing a dialysis modality.

From Science 2.0, Source: American Society of Nephrology

Cost Of Expensive Medication In Dialysis Catheters May Be Offset By Reduced Complications

Using an expensive agent to prevent blood clots in kidney failure patients' dialysis catheters may turn out to be less costly overall due to its ability to reduce medical complications, according to a study appearing in an upcoming issue of the Journal of the American Society of Nephrology (JASN).

Recombinant tissue plasminogen activator (rt-PA) is a medication used to break up blood clots that occur in the vessels of patients having a heart attack. A recent clinical trial revealed that using rt-PA once per week plus the anticlotting agent heparin twice per week was better than using heparin alone 3 times per week for preventing blood clots and infections in dialysis catheters. Its significant expense has limited its use in many dialysis programs, however.

Braden Manns, MD, MSc (University of Calgary, in Alberta, Canada) and his colleagues collected detailed costs within this trial to determine how the use of rt-PA affected overall health care costs over time. The researchers found that the increased cost of rt-PA was partially offset by lower costs for managing complications. Overall, the difference in unadjusted average cost for managing patients with rt-PA/heparin versus heparin alone was $323 Canadian dollars. When the costs were extrapolated over a 1-year time horizon, assuming ongoing rt-PA effectiveness, the overall costs of the strategies in the trial were similar.

"Our study suggests that for patients at high risk of dialysis line blockage, a higher expense rt-PA may provide reasonable value for the money," said Dr. Manns.

PKD Research

From 7th Space

Characterization of the SAM domain of the PKD-related protein ANKS6 and its interaction with ANKS3

Autosomal dominant polycystic kidney disease (ADPKD) is the most common genetic disorder leading to end-stage renal failure in humans. In the PKD/Mhm(cy/+) rat model of ADPKD, the point mutation R823W in the sterile alpha motif (SAM) domain of the protein ANKS6 is responsible for disease.

SAM domains are known protein-protein interaction domains, capable of binding each other to form polymers and heterodimers. Despite its physiological importance, little is known about the function of ANKS6 and how the R823W point mutation leads to PKD.

Recent work has revealed that ANKS6 interacts with a related protein called ANKS3. Both ANKS6 and ANKS3 have a similar domain structure, with ankyrin repeats at the N-terminus and a SAM domain at the C-terminus.

Results: The SAM domain of ANKS3 is identified as a direct binding partner of the ANKS6 SAM domain.

We find that ANKS3-SAM polymerizes and ANKS6-SAM can bind to one end of the polymer. We present crystal structures of both the ANKS3-SAM polymer and the ANKS3-SAM/ANKS6-SAM complex, revealing the molecular details of their association.

We also learn how the R823W mutation disrupts ANKS6 function by dramatically destabilizing the SAM domain such that the interaction with ANKS3-SAM is lost.

Conclusions: ANKS3 is a direct interacting partner of ANKS6. By structurally and biochemically characterizing the interaction between the ANKS3 and ANKS6 SAM domains, our work provides a basis for future investigation of how the interaction between these proteins mediates kidney function.

Author: Catherine N LeettolaMary Jane KnightDuilio CascioSigrid HoffmanJames U Bowie
Credits/Source: BMC Structural Biology 2014, 14:17

PKD in Cats

PKD in Cats

From St. Louis Dispatch, Dr. Fox Q & A

Polycystic kidney disease claims a young cat

Dear Dr. Fox • Please educate us feline lovers on polycystic kidney disease in cats. I just lost my 3-year-old kitty to this horrible disease.

We had her groomed and shaved for summer, and after losing her long coat, we noticed she was thin. She vomited the next week, quit eating and drinking and hid from us. This was not like her personality.

She was the runt of a rescue litter and always small. I took her to the vet, and he thought she had a virus. Antibiotics did not help. Blood work was drawn on the third week, and she was already in the “red zone” for kidney failure and dehydration. She would not drink and ate only a small amount of wet food. She was put on IV fluids, but they did not help.

She continued to lose weight weekly and was finally diagnosed with PKD when her levels had elevated to almost the end of the red zone, renal failure. She became very weak, and I had her euthanized. Please enlighten and educate your readers and me on this terrible disease. She was a rescue black cat, breed unknown, but had moderately long hair. Thank you for your column. —P.K., St. Louis

Dear P.K. • My sincere condolences for your loss. It was indeed a sad ordeal for both you and your young cat.

This condition is a degenerative disease of the kidneys that is a genetic defect prevalent in some breeds, such as Persians, but not uncommon in the more genetically diverse cat population. Some genetic diseases are self-limiting, afflicting animals before they reach breeding age, which prevents transmission to offspring. Regrettably, this is not the case with polycystic kidney disease.

Some veterinarians have done kidney transplants from healthy donor cats from shelters, and with the addition of immunosuppressant drugs to help stop rejection, some cats have had some life extension. In my opinion, aside from the costs, there are ethical considerations for both donor and recipient animals that lead me to not endorse such transplants. For humans, it is a lifesaver, but cats are not good candidates.

Kidney Dialysis 

From Digital Journal, Press Release

NaturaLyte Manufacturer Issues Recall, Citing Unacceptable Bacterial Infection Risk, Reports Baron and Budd

Fresenius Medical Care North America, manufacturer of the dialysis drugs GranuFlo and NaturaLyte, has recalled 56 lots of NaturaLyte due to finding higher bacteria levels than allowed by internal specifications. The company is involved in litigation in a Massachusetts federal court where more than 1,700 lawsuits have been filed in conjunction with a previous recall. (In Re: Fresenius GranuFlo/NaturaLyte Dialysate Litigation, MDL No. 2428)

On May 21, 2014, the U.S. Food and Drug Administration (FDA) issued a safety alert announcing the latest NaturaLyte recall. According to the alert, unacceptable levels of the Halamonas bacteria had been found and the affected drugs should be immediately taken out of use. (fda.gov/safety/medwatch/safetyinformation/safetyalertsforhumanmedicalproducts/ucm398553.htm, May 21, 2014)

GranuFlo and NaturaLyte are both used to neutralize blood acid levels in patients suffering from kidney failure. GranuFlo is a powder and NaturaLyte is a liquid. At the FDA's insistence, Fresenius issued a recall in March 2012 for both products after it was revealed they posed a significant risk of causing cardiac arrest in patients. The New York Times published an article stating that Fresenius circulated an internal memo on Nov. 4, 2011 that more than 900 patients being treated at dialysis centers owned by the company had suffered cardiac arrest the previous year. The two products contain high levels of bicarbonate, which were linked to the higher risk of cardiac arrest. (http://www.nytimes.com/2012/06/15/health/fda-investigates-fresenius-for-failure-to-warn-of-risk.html?_r=0)

According to court documents, plaintiffs' representatives have filed more than 1,700 lawsuits in the U.S. District Court, District of Massachusetts alleging their clients suffered harm due to use of GranuFlo and NaturaLyte. In the lawsuits, plaintiffs allege that Fresenius chose not to alert thousands of dialysis centers not owned by Fresenius about the risks, and that the communications to Fresenius clinics were inadequate. (In Re: Fresenius GranuFlo/NaturaLyte Dialysate Litigation, MDL No. 2428). [Read more]

Gift of Life

From ABC News, by Ely Brown via Nightline

Strangers Donate Kidneys to Keep Country's Longest Donor Chain Going

It’s Sunday afternoon, and Dr. Jayme Locke, director of the Incompatible Kidney Transplant Program at the University of Alabama at Birmingham Medical Center, is preparing for a marathon.

“We are going to be doing 14 operations this week,” she said, checking in on her patients.

Sprinkled among the rooms up on the eighth floor are patients waiting to receive the gift of life -- a new kidney. Also among the patients are the living donors bearing those gifts, people who are willingly giving up one of their two kidneys to help a stranger.

“We are anxious, all of us are, to hear the story of our recipient,” said Pastor Derek Lambert, one of the donors. “I don’t know if this is perhaps a young mother who’s feared leaving her kids, or a young man who is unable to provide for the needs of his family and this would give these types of individuals a new lease on life.”

They are all part of an intricate living donor kidney transplant chain that began last December. By the end of the week, 21 patients will have received kidney transplants making it the longest, on-going, single-institution chain in the country. The catch? In order to receive a kidney from a stranger, each recipient must have someone in their life willing to donate a kidney to a stranger in their honor to keep the chain going.

For donor Courtney McLaughlin, the decision to donate in her cousin’s honor was easy. “She’s been on a waiting list for a deceased donor for years and with no end in sight, and we’ve been on this list for three months and here we are,” she said.

More than 100,000 Americans are currently in need of a kidney transplant. For some, the wait for a kidney from a deceased donor can stretch as long as eight to 10 years. These living donor kidney chains can expand the pool of both donors and recipients and have the possibility of shortening wait times to just months. They also can provide recipients with more compatible matches and younger organs.

A year ago, Katelyn Pickel, an 18-year-old high school student, suddenly became severely ill, wound up on dialysis and required a kidney transplant. Her father Earl was a potential match for her, but by the two of them joining the chain, two things happened: Katelyn received a kidney from a much younger donor, and someone else waiting on the list was able to receive her father’s kidney.

“I have prayed to God that he would send an answer to my child. And He has,” Earl Pickel said. “How can I refuse someone else when somebody stepped up for me?”

Mickey Little had suffered from a rare kidney disease for more than a decade and he had become dependent on dialysis that kept him tied up to a machine for 8 hours every night. A previous transplant that failed after just a few days had left him with a less than one percent chance of finding a match. It took a while, but Dr. Locke was able to find him not only a match, but a perfect one.

“I won’t be restricted because of dialysis,” Little said. “I should be back to a normal life again which is amazing to me.’

“[He] certainly beat the odds,” said Dr. Locke. “He found his one in a million.”

Click on the infographic below explaining UAB's kidney donation chain to see a larger version. Story continues on Page 2 after the jump. [Read more]

From New York Daily News, BY MEG WAGNER

New Mexico college instructor donates kidney to student who wrote essay about health struggle

Last semester, Jennifer Adams asked her community college English class to write personal essays. When one of her students wrote about the day her kidneys failed, the teacher decided to donate one of her own.

Doing her homework paid off.

A Central New Mexico Community College instructor decided to donate a kidney to a student after she learned about the student's health struggles from an essay homework assignment.

In November, Jennifer Adams asked her English class to write personal essays, KRQE reported.

Melina Hernandez wrote hers about her kidneys.

Hernandez explained that she had felt sick for about six months before she finally went to the ER in 2010.

Doctors told her both her kidneys were failing. They put her on dialysis, and she's been waiting for a transplant ever since.

In her essay Hernandez wrote about the shock of learning her health was failing and how she continues to fight to stay alive, mostly for her 7-year-old daughter.

"After I heard the essay, of course, it was emotional hearing about somebody's hardships like that," Adams told the TV station.

The teacher immediately felt a special bond with her student.

"She was a genuine, honest, caring person," she said. "She was a mother, and I'm a mother."

Adams had to help.[Read more]

From Ledger-Enquirer, Columbus, Georgia, BY ALVA JAMES-JOHNSON

Couple raising money for kidney transplant

The love story brewed at Starbucks.

Michael Grant was a shift supervisor at the store on Bradley Park, and Shelby Harrell a barista.

They shared a love for biology, and Harrell was drawn to Grant's life-long battle with kidney failure. One of the first things she noticed was the bulging fistula on his arm, the result of a surgical procedure that prepared his vein for dialysis. Most people thought it was weird, which made Grant self-conscious. But Harrell found it intriguing.

"Right away people were like, 'Look at this, look at his arm,'" Harrell said. "And I was the only one there who was like, 'That's so cool. That's awesome. How does that work?'"

From there, Grant, now 28, and Harrell, 23, became friends, and now they are engaged to be married.

But they're also preoccupied with finding Grant a kidney, and raising $10,000 for the surgery.

Three months ago, the couple started a Go Fund Me web page to tell Grant's story and solicit donations. So far they've raised $2,690. The Georgia Transplant Foundation has set up an account for the donations and will match up to $10,000, Grant said. However, the couple said they'll need more than that to cover their expenses. Grant, who has already had two kidney transplants, said his surgery will cost almost $1 million, not to mention all the trips he'll have to make to Emory for treatment and the cost of medication.

"My insurance company should cover most of it, but with that high of a price tag, covering 80 percent still leaves me with a lot of extra medical bills," he said. "There's a lot of lost income that this will help offset." [Read more]

Read more here: http://www.ledger-enquirer.com/2014/07/04/3185587/couple-raising-money-for-kidney.html#storylink=cpy