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Polycystic kidney disease
January 25, 2006NIH and PKD Foundation launch HALT-PKD treatment trials
The National Institutes of Health (NIH) and the PKD Foundation have launched two treatment trials for autosomal dominant polycystic kidney disease (ADPKD). The common inherited disorder is characterized by cysts in the kidneys and other organs, high blood pressure, and aneurysms (bulges in blood vessels, which may burst) in the brain. Symptoms usually appear between the ages of 30 and 40 and include back and side pain and headaches. About half of ADPKD patients eventually develop kidney failure and require dialysis or a kidney transplant. The first Halt Progression of Polycystic Kidney Disease (HALT-PKD) patient enrolled last week at Emory University in Atlanta, one of seven recruitment sites (www.pkd.wustl.edu/pkd-tn).
"Decades of clinical and basic studies by NIH and others have delivered this exciting opportunity for translational research," says Catherine M. Meyers, M.D., a kidney specialist who directs HALT-PKD at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). "Naturally, we appreciate the PKD Foundation's invaluable guidance and support."
PKD Foundation President and CEO Dan Larson applauded the start of the trials. "PKD families are eager to learn of any potential benefits," says Larson. "Their hope and the hope of the PKD Foundation is that this will be a step toward finding a cure for PKD and improving the care and treatment of those it affects."
Carefully controlling blood pressure and using ACE-inhibitors or ARBs significantly delays or prevents kidney disease and failure from diabetes and other causes by reducing protein in the urine and preventing damage to the small blood vessels in the kidneys. Earlier trials of these treatments in PKD were not definitive, possibly because a small number of patients were involved.
Over the next 2 years, HALT-PKD will recruit more than 1,000 people with ADPKD and treat them for up to 4 years at centers in Atlanta, Boston, Cleveland, Denver, Kansas City, Kansas, and Rochester, Minnesota. The two trials will compare standard therapy using an angiotensin-converting enzyme inhibitor (ACE-inhibitor) to intensive therapy using both an ACE-inhibitor and an angiotensin receptor blocker (ARB). Patients will receive a device for measuring blood pressure at home, clinic visits, lab tests, and study medications at no charge. They will also have their kidney function estimated using a standard blood test measurement called eGFR and other measures to track progression of kidney disease.
HALT-PKD Study A, for people 15 to 49 years of age with early disease (eGFR >60), will also compare standard (120-130/70-80 mm Hg) and low (95-110/60-75 mm Hg) blood pressure targets and measure changes in cyst and kidney size using a Magnetic Resonance Imaging method developed by NIDDK's Consortium for Radiologic Imaging Studies of PKD. Study B, for people 18 to 64 and more advanced disease (eGFR 30-60), will track the time it takes eGFR to drop by 50 percent, the need for kidney failure treatment, and patient deaths.
PKD affects an estimated 500,000 people, about 90 percent of whom have ADPKD. In 2003, 23,000 people with cystic kidney disease (mostly PKD) received dialysis or a kidney transplant, making it this country's fourth leading cause of kidney failure. While genetic testing for ADPKD can help determine whether a family member can safely donate a kidney, testing can't predict onset of symptoms or severity of the disease, which also increases the risk of heart attacks, strokes and early death compared to the general population.
NIH/National Institute of Diabetes and Digestive and Kidney Diseases
PKD Foundation President and CEO Dan Larson applauded the start of the trials. "PKD families are eager to learn of any potential benefits," says Larson. "Their hope and the hope of the PKD Foundation is that this will be a step toward finding a cure for PKD and improving the care and treatment of those it affects."
Carefully controlling blood pressure and using ACE-inhibitors or ARBs significantly delays or prevents kidney disease and failure from diabetes and other causes by reducing protein in the urine and preventing damage to the small blood vessels in the kidneys. Earlier trials of these treatments in PKD were not definitive, possibly because a small number of patients were involved.
Over the next 2 years, HALT-PKD will recruit more than 1,000 people with ADPKD and treat them for up to 4 years at centers in Atlanta, Boston, Cleveland, Denver, Kansas City, Kansas, and Rochester, Minnesota. The two trials will compare standard therapy using an angiotensin-converting enzyme inhibitor (ACE-inhibitor) to intensive therapy using both an ACE-inhibitor and an angiotensin receptor blocker (ARB). Patients will receive a device for measuring blood pressure at home, clinic visits, lab tests, and study medications at no charge. They will also have their kidney function estimated using a standard blood test measurement called eGFR and other measures to track progression of kidney disease.
HALT-PKD Study A, for people 15 to 49 years of age with early disease (eGFR >60), will also compare standard (120-130/70-80 mm Hg) and low (95-110/60-75 mm Hg) blood pressure targets and measure changes in cyst and kidney size using a Magnetic Resonance Imaging method developed by NIDDK's Consortium for Radiologic Imaging Studies of PKD. Study B, for people 18 to 64 and more advanced disease (eGFR 30-60), will track the time it takes eGFR to drop by 50 percent, the need for kidney failure treatment, and patient deaths.
PKD affects an estimated 500,000 people, about 90 percent of whom have ADPKD. In 2003, 23,000 people with cystic kidney disease (mostly PKD) received dialysis or a kidney transplant, making it this country's fourth leading cause of kidney failure. While genetic testing for ADPKD can help determine whether a family member can safely donate a kidney, testing can't predict onset of symptoms or severity of the disease, which also increases the risk of heart attacks, strokes and early death compared to the general population.
NIH/National Institute of Diabetes and Digestive and Kidney Diseases
Polycystic Kidney Disease Current Events and Polycystic Kidney Disease News RSSAustralian study sheds light on kidney repair and disease
A study by Monash University researchers has shed new light on the microscopic antennas in the kidney that are involved in the organ's repair process.
The vasculature emerges as a potential therapeutic target in treating ADPKD liver cysts
As part of an effort to develop effective medical therapies that block the progression of liver cyst growth in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD), researchers at the University of Colorado Anschutz Medical Center have found that the liver cyst walls develop and maintain a vasculature as they grow out from the body of the liver and that factors released by epithelial cells that line the liver cyst wall lumen can drive the proliferation and development of vascular endothelial cells.
Mutations in gene linked to ciliopathies
An international team of scientists, led by researchers at the University of California, San Diego School of Medicine, have discovered a connection between mutations in the INPP5E gene and ciliopathies.
New insight into human ciliopathy
In the September 1st issue of G&D, Dr. Karen Oegema (UCSD) and colleagues identify the molecular basis of the lethal developmental disorder, hydrolethalus syndrome, and reveal that hydrolethalus syndrome actually belongs to the emerging class of human ciliopathy diseases.
New advances in cancer research to be highlighted at University of Leicester
A scientist whose work is key to understanding how cancer cells divide and spread in the body is to present advances in his latest research at a public forum to be held at the University of Leicester.
Proteins from garden pea may help fight high blood pressure, kidney disease
Researchers in Canada are reporting that proteins found in a common garden pea show promise as a natural food additive or new dietary supplement for fighting high blood pressure and chronic kidney disease (CKD). Those potentially life-threatening conditions affect millions of people worldwide.
Researchers disrupt biochemical system involved in cancer, degenerative disease
Screening a chemical library of 200,000 compounds, researchers at UT Southwestern Medical Center have identified two new classes that can be used to study and possibly manipulate a cellular pathway involved in many types of cancer and degenerative diseases.
Can you hear me now? How the inner ear's sensors are made
A UCLA study shows for the first time how microscopic crystals form sound and gravity sensors inside the inner ear. Located at the ends of cilia - tiny cellular hairs in the ear that move and transmit signals - these crystals play an important role in detecting sound, maintaining balance and regulating movement.
NYU scientists identify critical protein complex in formation of cell cilia
An international team led by NYU Cancer Institute have identified a protein complex that regulates the formation of cilia, which are found on virtually all mature human cells and are essential to normal cell function.
1 patient's account of becoming a live kidney donor
Annabel Ferriman, an editor at the BMJ, gives a frank first person account of her journey through the "protracted" and sometimes "frustrating" process of becoming a live kidney donor to her friend, Ray, who had been suffering from polycystic kidney disease for eight years.
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