Shark Diving for PKD

Raising Money for PKD Patients & Research

From Linlithgow Gazette, Linlithgow, Scotland
Diving in to help kidney charity

A caring 11-year-old is swimming in a tank full of sharks to raise money for a charity which means so much to his family.

Calum Graham, of Queensferry, will don a wetsuit and dive into a tank with tiger sharks at Deep Sea World tomorrow (Saturday, August 24) to raise cash for Polycystic Kidney Disease Charity (www.pkdchar
ity.org.uk).

He is raising cash for the charity as his dad Niki suffers from the disease and is currently awaiting a transplant.

Niki has to spend five hours a day, three times a week, hooked to a dialysis machine.

Calum said: ‘‘Even though my dad looks well his body isn’t and he is in real need of a new kidney and has been on the transplant list since November 2011. Raising money for this charity will hopefully raise more awareness about the disease. ”

Niki said: ‘‘Calum is one of the kindest, most thoughtful and bravest kids I have ever known. He has seen me, health wise, at my best and at my very, very worst and has always stayed strong.’’  [Read more]

From Playbill.com, by Carey Purcell

Bryce Pinkham, Kate Rockwell, Amber Iman Are Part of Broadway Sings for PKD at 54 Below Aug. 21

The third annual Broadway Sings for PKDis held Aug. 21 at 11:30 PM at 54 Below.

The concert, which benefits the Polycystic Kidney Disease Foundation, features new musical theatre songs by emerging writers. Composers include Gaby Alter, Landon Braverman, Derek P. Hassler, Timothy Huang, Bob Kelly, Itamar Moses, Kelly Pomeroy and more.

Hosted by Ben Cameron (Wicked), the concert's performers include Ali Ewoldt, (Les Miserables, The Fantasticks), F. Michael Haynie (Wicked, Dogfight),Amber Iman (Soul Doctor), Janet Krupin (Hands on a Hardbody, Bring it On), Bryce Pinkham (A Gentleman's Guide to Love and Murder, Love's Labour's Lost), Kate Rockwell (Rock of Ages, Bring it On), Robb Sapp (Shrek, Wicked), Tally Sessions (Big Fish, Yank) and Melanie Burg.

Rachel Kunstadt produces and directs the event, and Yan Li is the musical director.

Proceeds from the evening go to the Polycystic Kidney Disease Foundation, which "promotes programs of research, advocacy, education, support and awareness in order to discover treatments and a cure for Polycystic Kidney Disease and improve the lives of all it affects." [Read more]

Gift of Life

From Bella Magazine, by Pam Childs

Beauty from the Inside Out: Saving a life brings friends together again

Patti Gaddis was searching for friends on Facebook when she read something that alarmed her.

A childhood friend, Trenia Burkett, indicated in a post that she was seriously ill. One of her children had written, “Momma, it’s going to be OK.”

Patti did some digging and learned that Trenia had polycystic kidney disease (PKG) and needed a kidney. PKG is an inherited disease that causes the kidneys to enlarge and cysts to form on them. Eventually, it leads to end-stage kidney failure.

A mom herself, Patti couldn’t imagine Trenia’s children losing their mom in her early 50s.

“All I needed was to read one of her kid’s comments, and it hit me,” said Patti. “You have two kidneys, you know you’re healthy. Share your health’.’’

Patti called Trenia.  [Read more]

From Fox2Now.com, St. Louis, MO, by Tom O'Neal

One sister receives double organ transplant, other still on waiting list

CRYSTAL CITY, MO (KTVI)– We first introduced you to the Derque family of Crystal City about a year and a half ago. At that time, 7-year old Kate Derque and her 12-year old sister, Abbi both needed double organ transplants. A genetic disease called Polycystic Kidney Disease was destroying their kidneys and liver. Kate was getting dialysis three times a week. Abbie was getting monthly checkups. Abbi had had a recent scare with bleeding in the liver. Both were on the waiting list for a transplant.

Middle child, John, was healthy. Their mother, Kim refused to feel sorry for herself or let her children feel sorry for themselves. She was determined that they not be defined by their disease.

Our followup report found a big change in the family’s situation. In May of this year, Kate now 9, underwent a 10 hour surgery at Cardinal Glennon Children’s Medical Center to receive a new kidney and new liver. Things went well and Kate has had a good recovery.

But the family’s wait is not over. Abbi is still waiting for a similar transplant. Kate was transplanted first because she was higher on the list. Abbi is doing pretty well right now, but the call could come at any time. She says waiting is the hard part. And right now, she can’t really participate in sports and has to stay within three hours of the hospital in case that call comes. Both girls were anxious to start school.

Kim says it’s hard to find words to express her gratitude to the donor family that gave Kate a new lease on life. She does not know the identity of the donor. Now, they take it one day at a time to wait for a transplant that will give Abbi a chance at a normal life.

This Sunday evening, August 25th Mid-America Transplant Services will hold its annual Candlelight Memorial Ceremony. It’s a chance for MTS donor families and recipients to come together and honor the gift of life. Along with other recipients, Kate will be handing out roses to donor families at the event. It will be held at Donor Memorial Monument and Park. That’s 1110 Highlands Plaza Drive East. (In the area where the old Arena was located) The public is invited to share in the event.

For more information you can visit the MTS website at www.mts-stl.org

PKD Research

From Noozhawk.com, Santa Barbara, California, by UCSB Office of Public Affairs

UCSB Professor Joins Grant-Reviewing Body of National Institutes of Health

Thomas Weimbs, one of the world's leading experts on polycystic kidney disease and an associate professor in UC Santa Barbara's Department of Molecular, Cellular, and Developmental Biology and at the campus' Neuroscience Research Institute, has been appointed as a member of the Center for Scientific Review's Kidney Molecular Biology and Genitourinary Organ Development (KMBD) study section.

"It is an honor to be a member of an NIH study section," Weimbs said. "It goes to emphasize that there is a lot of medically relevant research being conducted at UCSB even though we don't have a medical school here."

Research in the Weimbs Lab at UCSB focuses on two related areas of investigation: autosomal-dominant polycystic kidney disease (ADPKD) and epithelial cell polarity and important cellular proteins essential for the transport of membranes inside cells. ADPKD is a very common genetic disease that leads to kidney failure and for which no approved treatment is available.

"Congratulations!" USCB Chancellor Henry Yang wrote in an email to Weimbs. "Your service in this critical NIH position is a reflection of your renowned research expertise and achievements in kidney disease, and will help to further advance national biomedical research."

As a standing member of the KMBD study section, Weimbs will review dozens of NIH grant applications each year of his six-year term. [Read more]

From 4-Traders.com

Research and Markets: Polycystic Kidney Disease - Pipeline Review, H2 2012

This research provides an overview of the indication's therapeutic pipeline. This report provides information on the therapeutic development for polycystic kidney disease, complete with latest updates, and special features on late-stage and discontinued projects. It also reviews key players involved in the therapeutic development for polycystic kidney disease.

The information is built using data and information sourced from proprietary databases, company/university websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources.

Scope

- A snapshot of the global therapeutic scenario for polycystic kidney disease.

- A review of the polycystic kidney disease products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.

- Coverage of products based on various stages of development ranging from discovery till registration stages.

- A feature on pipeline projects on the basis of monotherapy and combined therapeutics.

- Coverage of the polycystic kidney disease pipeline on the basis of route of administration and molecule type.

- Key discontinued pipeline projects.

- Latest news and deals relating to the products. [Read more]

New York, New York! Broadway Sings for PKD

Donations for PKD Research

From Cabaret.BroadwayWorld.com, by BWW News Desk
Ali Ewoldt, Kate Rockwell & More Set for Broadway Sings for PKD at 54 Below

The third Broadway Sings for PKD, a concert benefitting the Polycystic Kidney Disease Foundation, will be offered Wednesday, August 21st at 54 Below in NYC.

The 11:30 PM concert will feature new musical theatre songs by emerging writers, including Gaby Alter, Landon Braverman, Derek P. Hassler, Timothy Huang, Bob Kelly, Itamar Moses, Kelly Pomeroy, and
more.

Performers will include Broadway actors Ali Ewoldt, (Les Miserables, The Fantasticks), F. Michael Haynie (Wicked, Dogfight), Amber Iman (Soul Doctor), Janet Krupin (Hands on a Hardbody, Bring it On), Bryce Pinkham (A Gentleman's Guide to Love and Murder, Love Labour's Lost), Kate Rockwell (Rock of Ages, Bring it On), Robb Sapp (Shrek, Wicked), and Tally Sessions (Big Fish, Yank), and rising star Melanie Burg. Ben Cameron (Wicked) will host.

Broadway Sings for PKD is produced and directed by Rachel Kunstadt, with musical direction by Yan Li.

Proceeds from the evening will go to the Polycystic Kidney Disease Foundation, which "promotes programs of research, advocacy, education, support and awareness in order to discover treatments and a cure for Polycystic Kidney Disease and improve the lives of all it affects."

54 Below is located at 254 W 54th St, Cellar. For tickets, visit www.54below.com. For more information on the Polycystic Kidney Disease Foundation, visit www.pkdcure.org.

[Read more]

Budget Cuts

From PostTrib.SunTimes.com, by Jerry Davich, Crown Point, Indiana

Dialysis patients drained by proposed budget cut

James Myers sat quietly in a large chair as his blood was artificially cleansed of waste by a whirring dialysis machine that beeped every few seconds.

With a blanket draped over his legs and a catheter connected to his chest, Myers sat patiently inside the Fresenius Medical Center in Crown Point. He visits there three times a week for at least four hours each time to undergo hemodialysis, which replaces the function of his failing kidneys.

The 59-year-old Crown Point man suffers from polycystic kidney disease, which took the lives of several family members, including his father. Though Myers was diagnosed in his younger days, his positive lifestyle choices the past three decades only delayed the inevitable.

His genetic fate caught up to him last summer. He’s been on dialysis ever since. [Read more]

Renal Disease

From Philippine Information Agency

Renal disease is top 6 cause of death in Kalinga

TABUK CITY, Kalinga, Aug. 14 (PIA) -- End Stage Renal Disease (ESRD) is ranked as the top six cause of death in Kalinga in 2012 with 51 reported cases at the Provincial Health Office (PHO).

Donalyn Lasmarias, point person on lifestyle diseases at the Kalinga PHO said the renal disease has sustained its fifth to sixth rank for the past five years, making it one of the top three lifestyle diseases of mortality in the province.

For the first quarter this year, some public schools have also conducted a deepstick urine test among students and at least four female ages 5-14 were found to be positive of renal disease.

Rural Health Units have also reported that deep stick conducted among adults ages 20-64 resulted to the finding of at least 12 females and four males to be positive or renal disease. These cases were referred to the municipal health units or the provincial hospital for proper management.

Several patients she said are availing of outside health facilities for their treatment such dialysis and other medical needs considering that the Kalinga Provincial Hospital has yet to establish its own dialysis center.

Based on studies, diabetes and high blood pressure are the most common causes of ESRD, thus it is vital to manage these with your doctor so it would not result to ESRD or kidney failure which is the stage when the kidneys no longer work for a person to live without dialysis or a kidney transplant, Lasmarias said.

Other causes also include auto immune diseases (like lupus, HIV and IgA nephropathy), Genetic diseases like polycystic kidney disease, injuries and some medicines or other drugs. [Read more]

FDA Committee Says No to Tolvaptan

PKD Treatment

From MarketWatch.com, via Business Wire 
U.S. Food and Drug Administration's Cardiovascular and Renal Drugs Advisory Committee Recommends Not to Approve Otsuka Pharmaceutical's Tolvaptan for Use in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

A Press Release.
TOKYO, Aug 05, 2013 (BUSINESS WIRE) -- Otsuka Pharmaceutical Co., Ltd. announced today the U.S. Food and Drug Administration's (FDA) Cardiovascular and Renal Drugs Advisory Committee voted 9 to 6 not to approve tolvaptan for the treatment of ADPKD. The FDA is not bound by the Committee's guidance but takes its advice into consideration...  [Read more]

From MedPageToday.com, by David Pittman, Washington Correspondent, MedPage Today

FDA Panel Vetoes New Tolvaptan Indication

SILVER SPRING, Md. -- An FDA advisory committee has declined to endorse an expanded indication for tolvaptan (Samsca) to treat autosomal dominant polycystic kidney disease (ADPKD).

The agency's Cardiovascular and Renal Drugs Advisory Committee voted 9-6 on Monday against recommending approval of tolvaptan for use against the rare disease that causes a proliferation of kidney cysts, resulting in grossly enlarged kidneys.

Panel members echoed concerns FDA reviewers had expressed in advanced of the meeting,namely, that judging efficacy from the single trial supporting the treatment is difficult because of missing data.

Furthermore, it was tough to balance the drug's various safety issues including liver toxicity with the unmet need that ADPKD patients face. [Read more]

Living With PKD

From Pittsburgh Courier

Three-year old toddler in desperate need of kidney transplant

Jordyn Maddox of Delaware has end-stage kidney failure and is currently at the Children’s Hospital of Philadelphia.

Before Jordyn was born, doctors diagnosed her in-utero with Autosomal Recessive Polycystic Kidney Disease, a rare and potentially deadly condition that causes low-functioning, cyst-filled kidneys. The doctors did not expect her to make it to birth. Jordyn was very sick when she was born and was not expected to live.

“Once she was born, the doctors looked at her and said there was nothing they could do. We were passing her around, saying goodbye to her,” recalled her mother, Taylor Burden.

Jordyn was placed in the hospital’s neonatal intensive care unit where she spent three months fighting to get better. After she was released from the hospital, she made it to her first birthday before she needed serious medical intervention. Shortly after her first birthday, Jordyn had to be placed on peritoneal dialysis, a type of dialysis that uses the lining of the belly to filter the blood. [Read more]

Gift of Life

From The Courant, Hartford, Connecticut, by Jesse Leavenworth

Manchester Police Officer Gives Kidney To Fellow Officer

The joke around the police department is that Det. James Moore will have to change his last name to "Marois."

That would be Officer Adam Marois, Moore's fellow officer and now, something closer to blood kin.

On June 27 at Yale-New Haven Hospital, Marois donated one of his kidneys to Moore, 44, who had been diagnosed with kidney disease. The two knew each other before Marois, 26, came forward for the organ donation, but they were not steady friends.

"It definitely strengthened the bond," Marois said. "There was definitely a bond before, but now it's a little more personal."  [Read more]

From Creston News Advertiser, Creston, Iowa, by Kyle Wilson

Kidney Exchange, Crestonian Barb Coenen donates kidney to stranger so fellow Crestonian Allison Danilovich can receive a new, healthy kidney transplant.

Two Crestonians made Iowa history this week.

Tuesday morning Barb Coenen of Creston laid on a surgical table for three hours at Iowa Methodist Hospital. Surgeons cut her open, removed her kidney and shipped it to a complete stranger in New Jersey....[Read more]

Raising Funds for Life

From Get Hampshire.co, United Kingdom, By Stephen Lloyd

Mum and daughter's reunion walk for great cause

Mother flies to UK from New Zealand to join daughter for fundraising London walk

A mother and daughter who live on the opposite sides of the world have been reunited by a cause close to their hearts.

Rae Nunes Vaz flew from New Zealand to join daughter Rachelle Edwards for Kidney Research UK’s annual London Bridges Walk on July 14.

The pair, who had not seen each other for four years, have so far raised more than £1,000.

Both have polycystic kidney disease, an inherited genetic condition where the kidney is gradually replaced by slow-growing, fluid-filled cysts. The cysts grow at different rates and can cause the kidneys to grow massively in size.

Rachelle, 36, who lives in Hook after moving from her native New Zealand, said: “When we found out the event coincided with mum’s first visit to the UK, it was an opportunity not to be missed. It was great to do the walk together.”

London Bridges Walk is Kidney Research UK’s flagship fundraising event and offers those taking part the chance to pass by many of London’s most spectacular sights, including the London Eye, Big Ben and the Tower of London. [Read more]

From MessengerNewspapers.co, United Kingdom

Proud parents launch fundraising drive

PROUD parents James and Jo Gray are holding a fundraising raffle to raise money for research into polycystic kidney disease - after their son, Isaac was diagnosed with the illness at three-months-old.

Following concerns during pregnancy Isaac spent the first week of his life in intensive care before his condition was identified.

The Ashton on Mersey couple were told that it was a hereditary illness, with a 50 per cent chance that any future children would be affected too.

Now they are doing all they can to raise funds for research into the condition.  [Read more]

PKD Treatment Ups and Downs

Research Surprise

From Nature Genetics, via MedicalXpress.com
Surprising mechanism discovered in polycystic kidney disease

A study by Yale researchers has uncovered a new and unexpected molecular mechanism in the development of polycystic kidney disease, or PKD. The study appears in Nature Genetics.

Cilia are the hair-like structures on the surface of many human cells that can either move things along – dirt out of the lungs, or an egg from the ovary to the uterus – or sense the environment, such as vision in the retina or smell in the nose. Recent research has implicated defects in the sensory cilia—often caused by genetic mutations—in many human diseases, including cancer, cardiac disease, blindness, and kidney disease. In the kidney, disruption of sensory cilia cause kidney cysts.

The polycystin-1 and -2 (also known as PC1 and PC2) proteins are key players in the normal functioning of the kidneys. Earlier research has shown that when they are lost or mutated, cysts grow in the kidneys and cause almost all cases of PKD in humans.

Working in mice, the Yale team found that cysts grew when the cilia were intact but lacked polycystin—but, surprisingly, cysts stopped growing despite the absence of polycystins when the cilia were disrupted or eliminated.

The activity of this pathway, and the timing of the loss of polycystin proteins and the cilia, determined the severity of both early- and adult-onset PKD, the researchers found.

"None of the other pathways discovered so far have proven as universal as the cilia dependent pathway in explaining polycystic kidney disease," said corresponding author Dr. Stefan Somlo, professor of internal medicine (nephrology) and genetics at Yale School of Medicine. "We found to our surprise that elimination of cilia suppresses cyst growth in all of the genetic models of human PKD." [Read more]

PKD Treatment 

From MedPageToday.com, by David Pittman, Washington Correspondent, MedPageToday

FDA Reviewers Pan Tolvaptan Trial Results

WASHINGTON -- FDA reviewers aren't ready to endorse tolvaptan (Samsca) to treat autosomal dominant polycystic kidney disease (ADPKD), citing missing clinical trial data, they said ahead of an advisory committee meeting to consider the drug.

A single phase III trial showed the drug slowed the loss of renal function in patients with relatively healthy kidneys but deemed at high risk of failure, FDA reviewers noted in briefing documents prepared for the agency's Cardiovascular and Renal Drugs Advisory Committee.

"However, because of missing data in a sizeable portion of the study population and particularly so in the tolvaptan arm, the size of the treatment effect is unclear," staff stated in the documents for Monday's meeting. "Treatment effects on other endpoints -- kidney volume and renal pain events requiring medical intervention -- were supportive of the drug's activity."

The committee will meet to discuss the trial results and vote on recommending approval for slowing kidney disease in adults at risk of rapidly progressing ADPKD. [Read more]

Save a Life

From Herald-Mail.com, Fredeirick, Maryland

Kindness of a stranger: Woman donates kidney to her customer

FREDERICK, Md. — It can be called a bank withdrawal and deposit of a different kind for two Frederick women.

Deanna Henderson, assistant branch manager of M&T Bank’s Worman’s Mill Branch in Frederick, recently donated a kidney to a bank customer, Carla Watts of Frederick.

Henderson, of Frederick and a Boonsboro native, said she only knew Watts in passing from coming into the bank. She said they didn’t have any other relationship than a friendly hello when Watts banked. That changed in March 2012.

Living with PKD

From Tele-Management.ca

Will a healthy diet help my kidneys?

I am a 45-year-old male with polycystic kidney disease. I have changed my diet and eat mostly vegetarian. Will this type of diet help my kidneys? Is there anything I can do as far as dieting or exercise that will preserve my kidney function?

...There is no specific treatment for the condition, but measures should be taken to avoid damaging the kidneys further. Contact sports are inadvisable, since trauma can cause bleeding in the cysts. Kidney infections, which usually follow catheterization or cystoscopy, must be avoided. Medications that damage the kidneys, including some antibiotics (not penicillin, cephalosporin or erythromycin), phenacetin (a component of some pain meds), and nonsteroidal anti-inflammatories such as ibuprofen and others, should be avoided.

Hypertension must be aggressively treated. Dehydration and salt restriction should be avoided.

Your letter raises an interesting question regarding eating meat. When someone with polycystic renal disease develops actual kidney failure, then protein must be limited, and meat of course is rich in protein. However, this does not mean that avoiding protein before the development of kidney failure will prevent it. Research in normal laboratory animals seems to indicate that eating a high-protein diet may help cause kidney failure, but I don’t believe that this has been shown in humans, and standard texts do not advise restricting meat in this condition until the kidney failure actually develops. If you are happy with your vegetarian diet, which can be perfectly healthy if it is balanced, then perhaps you may slow the progression to kidney failure, but I am not aware of any strong evidence in favor of this treatment. [Read more] Medical Disclaimer...