From Wall Street Journal. by ERIC PFANNER
For Otsuka Drug, U.S. Says No and Japan Says Yes
Japanese pharmaceutical regulators have approved a treatment for a rare kidney disease, only a few months after the U.S. Food and Drug Administration rejected the use of the drug for the same purpose.
The affliction, called autosomal dominant polycystic kidney disease, causes a painful swelling of the kidneys, resulting in renal failure in one-half of those afflicted by the age of 70, according to Otsuka Pharmaceutical Co., which developed the drug, called Samsca. ADPKD, a genetic disease, has been diagnosed in 30,000 people in Japan, 120,000 in the United States and 200,000 in Europe, according to the company.
Otsuka, which is based in Tokushima, Japan, says Samsca is the first drug that has been shown to slow the progression of the disease. The company said clinical trials in 15 countries, involving more than 1,400 patients, demonstrated that the treatment significantly delayed the development of cysts that cause the kidneys to swell.
While the Ministry of Health, Labor and Welfare approved the drug for the treatment of ADPKD this week, the FDA said last year that the studies were inconclusive. The FDA also raised concerns about possible buildup of harmful enzymes in the liver, stemming from treatment with Samsca. Otsuka said it would put in place a system to monitor this effect.
Despite the FDA’s rejection of the drug to treat the kidney disease, Samsca is already sold in a number of countries, including the U.S., to treat a symptom of heart failure. In this situation, there is less risk of side effects in the liver because the drug is only prescribed for a short term, the company said.
Kidney News
Higher protein intake in middle age is associated with greater declines in kidney function over time, researchers reported online ahead of print in Nephrology Dialysis Transplantation.
Massimo Cirillo, MD, of the University of Salerno in Salerno, Italy, and colleagues collected data on overnight urinary urea, serum creatinine, estimated glomerular filtration rate (eGFR), and other variables in 1,522 men and women aged 45-64 years. Subjects were participants in the Gubbio study, an ongoing population-based investigation in Gubbio, Italy. The investigators re-assessed serum creatinine, eGFR, and other variables in 1,144 of 1,425 survivors after 12 years of follow-up.
At baseline, the study population had a mean eGFR of 84 mL/min/1.73 m2 as calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation and protein intake of 1.34 g/day per kilogram of ideal weight as assessed by measurements of overnight urine excretion of urea nitrogen.
Cross-sectional analyses showed that each 1 g/day increment in protein intake was associated with a 4.7 mL/min/1.73 m2 increment in eGFR. Longitudinal results demonstrated that, after 12 years of follow-up, study subjects had experienced a mean 11.6 mL/min/1.73 m2 decline in eGFR. Each 1 g/day increment in protein intake was associated with a 4.1 mL/min/1.73 m2 decline in eGFR and a 78% increased likelihood of having an eGFR below 60 mL/min/1.73 m2.
“The study reports the first evidence that, in a sample of the middle-age general population, higher protein intake is associated cross-sectionally with higher eGFR and longitudinally with greater eGFR decline over time,” the investigators staged. “Findings were similar for both sexes, independent of several variables, and consistent in subgroup analyses.”
The study's findings agree with clinical studies demonstrating short-term GFR stimulation by protein intake and unfavorable effects of high protein diet in CKD, Dr. Cirillo's team said. [Read more]
From TechPageOne.Dell.com, By Barbara Bermudez
About 430,000 Americans receive dialysis, a treatment that takes hours at a time.
A new wearable artificial kidney may soon allow patients with end-stage kidney failure to avoid hooking up to a bulky dialysis machine, where patients can be tethered for hours a day.
The battery-operated device is light enough to be worn on a tool belt around the waist, allowing patients to go about their lives working, sleeping and taking showers with it on.
Known as a WAK, the 10-pound device is being tested in Seattle at the Kidney Research Institute at the University of Washington, in collaboration with the Food and Drug Administration.
Researchers will be looking not only at the technical operations of the device — its safety and effectiveness — but how it may improve the quality of life of dialysis patients. Nationwide, 430,000 Americans receive dialysis, working around a regimented schedule of being hooked up to a machine that is often located in hospitals or dialysis centers.
“Quality of life issues will be embedded in the trial design,” said Dr. Jonathan Himmelfarb, director of the institute. “We’ll probably be asking patients, ‘Can you move with ease? How do you feel? How does the device or the treatment affect your daily life? Can you go to work with it on, or go out with your family and friends?’ We will be looking at key health outcomes as well as health economics.”
Taxing on patients’ time
An estimated 31 million Americans, about 10 percent of the population, have chronic kidney disease. It is the eighth leading cause of death, and every day 14 people die waiting for a kidney.
Dialysis prolongs life by keeping the body in balance. It removes waste, salt and extra water to prevent them from building up in the body. It maintains a safe level of chemicals in the blood such as potassium, sodium and bicarbonate while also helping to control blood pressure. Patients typically undergo dialysis about four hours a day, three times a week. But research has shown that patients who have dialysis daily fare better than those who do it three times a week.
“The problem is there is not enough money to do daily dialysis, not enough machines and nurses, and patients hate to be on dialysis,” said Victor Gura, an associate clinical professor at the David Geffen School of Medicine at the University of California, who developed and patented the WAK. “Treatment of dialysis patients is one of the most expensive items on the Medicare budget.”
Costly treatment
Approximately $16 million is spent annually on dialysis treatment in the United States, with about 80 percent of those costs paid for by Medicare, Medicaid or other government programs.
Today’s dialysis machines are bulky, power-hungry and rival the size of washing machines. If successful, the WAK would be the equivalent to today’s electronic tablets or laptops, revolutionized from the first massive computers that stood as tall as refrigerators.
Gura hopes to make the device commercially available at a cost similar to the expense incurred for conventional dialysis and supplies. [Read more]
Kidney Donors
With March being National Kidney Month, the Northeast Kidney Foundation is making sure people are aware of the signs of kidney disease and how they can help save lives. As part of their mission to improve the quality of life of those affected by kidney disease through early identification, intervention, prevention and support services, the foundation educates the community at health fairs, schools and worksites.
Kidney disease occurs when the organs become damaged, causing an excess buildup of waste products and fluids. Taking good care of your body and knowing the risk factors of kidney disease are critical to staying healthy.
“Know your risk factors and getting your kidneys checked is really important,” said Carol LaFleur, executive director of the foundation, which began in 1974 by Edwin and Rhoda Sperber, after Edwin donated a kidney to their daughter.
Risk factors include diabetes, high blood pressure, obesity and high cholesterol. The five stages of kidney disease can be based off of a person’s glomerular filtration rate (GFR), which is the best way to determine kidney function. A normal GFR is 90 or above, however a person with a normal GFR can still develop kidney disease due to risk factors. The possibility is even higher for African Americans and the older the person.
Andrea Hanny, a Schenectady resident diagnosed at stage three almost 20 years ago, took precautions to keep herself healthy. It wasn’t until late 2008 that her kidney function dropped below 10 percent. “This was an extremely stressful time for me because I realized that I was no longer in control of what was happening to my body,” said Hanny.
Hanny received a new kidney in November 2011, keeping it healthy by visiting a transplant surgeon every three months, taking the required medication and avoiding foods that could affect the medications. “All I am certain of is that someone continues to live on in my body and we have a symbiotic relationship,” said Hanny, who remains unsure of the exact identity of the deceased donor.
In an effort to increase organ donation, the foundation works with the Center for Donation & Transplant and the New York Alliance for Donation, and will soon partner with Kidney Connection.
The human body has eight transplantable organs: the heart, lungs, liver, kidneys, intestine and pancreas. On average, 18 people die daily in the United States waiting for an organ, said Becky Daniels, Hospital & Community Services specialist for the Center for Donation & Transplant.
“There are almost 11,000 people on the waiting list for organ transplants in New York state alone and almost 9,000 of them are waiting for kidneys,” said Tricia Shapiro, a Troy native and current Guilderland resident. Shapiro’s husband, Ed Shapiro, received a kidney transplant in 1988 after being diagnosed with Polycystic Kidney Disease.
When the kidney began to fail in 2007, Tricia spoke with a transplant doctor about donating a new kidney to Ed. Although above the normal age considered for donation, Tricia was healthy. On May 19, 2008, at age 65, she became one of the oldest living kidney donors at AMC. He died in 2011, two months after being diagnosed with pancreatic cancer. “I would do it again in a heartbeat,” said Tricia “I often tell people that donating that kidney to him was the best thing I had ever done for myself.”
For Sherril and Peter Bindelglass, who made the decision to donate the organs of Amy Bindelglass, their 2-year-old daughter who died in a car accident in Jan. 2000; the choice was obvious, they say.
Amy was bright for her age, she knew the entire alphabet and could even write a few words, said Sherril. Amy adored her older brother, Adam Bindelglass, who sustained serious injuries from the accident. Adam, now a freshman at Northeastern University, is an active supporter of organ donation.
“Organ donation saves many lives and gives others a more enjoyable life,” said Adam. “Each person who donates their organs can have an effect on multiple people.” Sherril, Peter and Adam are all registered organ donors, while the two youngest members of their family, Andy and Abby Bindelglass, await their 18th birthday to join the registry.
“Only about 22 percent of New Yorkers are registered to be organ donors,” said Daniels. While you can register to be an organ donor at your local DMV, enrollment can also be done online by visiting www.donatelifecdt.com or mailing forms to the New York State Department of Health, said Daniels.
“Organ donation saves many lives and gives others a more enjoyable life,” said Adam. “Each person who donates their organs can have an effect on multiple people.” Sherril, Peter and Adam are all registered organ donors, while the two youngest members of their family, Andy and Abby Bindelglass, await their 18th birthday to join the registry.
“Only about 22 percent of New Yorkers are registered to be organ donors,” said Daniels. While you can register to be an organ donor at your local DMV, enrollment can also be done online by visiting www.donatelifecdt.com or mailing forms to the New York State Department of Health, said Daniels.
From WetherbyNews, Wetherby, Yorkshire, England
Railwayman Dennis Roberts was left fighting for his life after being diagnosed with a crippling genetic disease.
Until, that is, his wife Jacqueline proved a match and gave him a kidney transplant.
“When I woke up after my operation, it was as if I had jump leads attached,” said the Knaresborough signalman, who lives in Ripon.
“I felt revitalised. I thought at first it was the morphine. But I haven’t come down yet.”
Dennis, now 61, was diagnosed with polycystic kidney disease at the age of 33. It meant that cysts were growing on his kidneys, causing them slowly to fail.
Checking his family medical history, it came as a sharp shock to realise the condition was genetic - and almost everyone in his family was affected.
His brother died of the illness. Another is on dialysis as he waits on the transplant list. His mother and grandmother both suffered.
And he has passed it on to his two sons, aged 24 and 30.
“It’s the most common hereditary disease there is,” he said. “But it doesn’t usually show itself until you’re in your 50s.”
Dennis was given support and advice from the polycystic kidney charity and referred to consultants at York Teaching Hospital, who said they would carry on as normal until his kidney function dropped to 12 per cent. Which it did in 2009.
“’Was there anybody in my family that could give me a kidney?’”, he said. “There was only Jacqueline left. She said yes, straight away.
“And the tests on her came back amazing. We were a match - a good match.”
The couple were booked in for surgery at St James’ hospital in Leeds just weeks later.
“It was strange really, knowing we would both be going in together,” said Jacqueline, 55.
“We decided to catch the 36 bus to Leeds. We knew we weren’t going to be able to drive home, so we tried to keep it normal.
“It was actually quite fun. We were sat there giggling at the thought of what everyone else on the bus would say if they knew what we were doing.”
The operation was on a Friday morning in October, 2009.
“Jacqueline went down first,” said Dennis. “Then, they said, they would have ‘a spot of lunch’ before I went down to the transplant room as well.
“They didn’t remove the old kidneys, so I ended up with three and Jacqueline had one.”
The couple even have nicknames for their kidneys. [Read more]
Lots of people get nicknames and it looks like Ken Wilkie’s is going to stick.
Wilkie, 48, who caught attention across the country after advertising for a kidney on Kijiji back in November, is still being recognized months later.
"It's insane — people still come up to me all the time and say, 'Hey, you’re the kidney guy,'" he said laughing.
Wilkie is hoping for life-changing news soon and already has a three-day celebration planned.
“I’ve been planning a kayak trip from one end of the Bras d’Or Lake to the other, so I’ll be heading out when I finish,” he said.
“I can’t wait, I spent 30 some years dreading the day I’d have to start dialysis; now I’m looking forward to the day they tell me I won’t have to anymore.”
Wilkie said several people are in various stages of testing as possible kidney donors for him, and he expects word in six to eight weeks.
"Right now they're telling me a girl from Bay St. Lawrence I was in school with up to Grade 3 is probably going to be my best match."
Wilkie was diagnosed with polycystic kidney disease — a genetic condition —at age 15. [Read more]
Kidney Dialysis
From MarketWatch.com, Press Release
Rockwell Medical, Inc. RMTI -0.41% , a fully-integrated biopharmaceutical company targeting end-stage renal disease (ESRD) and chronic kidney disease (CKD) with innovative products and services for the treatment of iron replacement, secondary hyperparathyroidism and hemodialysis, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking approval for the marketing and sale of Triferic (soluble ferric pyrophosphate citrate), the Company's late-stage investigational iron-replacement drug for treating iron deficiency in chronic kidney disease patients receiving hemodialysis. The NDA submission is based primarily on the datasets derived from the Triferic Phase 3 registration study program, and includes efficacy and safety data from additional studies...
About Triferic
Triferic is a unique iron compound that is delivered to the hemodialysis patient via dialysate, replacing the 5-7 mg of iron that is lost during every dialysis treatment. Triferic is introduced into the sodium bicarbonate concentrate on-site at the dialysis clinic, which is subsequently mixed into dialysate. Once in the dialysate, Triferic crosses the dialyzer membrane and enters the blood where it immediately binds to apo-transferrin and is taken to the bone marrow, similar to how dietary iron is processed in the human body. In completed clinical trials to date, Triferic has demonstrated that it can safely and effectively deliver sufficient iron to the bone marrow, maintain hemoglobin and not increase iron stores (ferritin), while significantly reducing ESA dose. {Read more]
From RadioAustralia.net , Samoa
While the number of people undergoing dialysis at the foundation's centre in Apia has increased more than ten fold over the last nine years, its budget has remained stuck on five million dollars a year.
When the centre opened in 2005 it had six patients, now it has 75, and the queue is getting longer as more people join the waiting list for dialysis.
Presenter: Richard Ewart
Speaker: Mulipola Roger Hazelman, General Manager, National Kidney Foundation, Samoa
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