A promising treatment for an incurable, deadly kidney disease

Kidney Disease

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DALLAS – Sept. 14, 2019 – A potential treatment for polycystic kidney disease – a genetic disorder that causes the kidneys to swell with multiple cysts and can eventually lead to organ failure – has shown promising results in animal testing.

A studyDiscovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease” describing the drug’s development and testing appeared in Nature Communications. The study shows an approximately 50 percent reduction in kidney size in afflicted mice following treatment. The drug is now in early clinical trials in human subjects, said Dr. Vishal Patel, Associate Professor of Internal Medicine at UT Southwestern and senior author of the study. Dr. Vishal Patel is a doctor of Indian origin.

ADPKD
affects about 12 million people worldwide

Autosomal dominant polycystic kidney disease (ADPKD) affects about 12 million people worldwide, with half developing end-stage kidney disease by age 60, according to the study. “Once the kidneys have failed, the only options for survival are dialysis or a kidney transplant,” Dr. Patel said. “A large percentage of ADPKD patients on dialysis die each year while waiting for a donated kidney.”

The only
drug currently approved to treat ADPKD, called Jynarque (generic name tolvaptan),
carries the FDA’s highest warning in its prescribing information, a box
notifying prescribers and users of the possibility of “serious and potentially
fatal liver injury.”

The new
treatment cooperatively developed at UT Southwestern and Regulus Therapeutics
Inc., a biopharmaceutical company based in California, showed no evidence of
toxicity in animals or in human cell tests, according to the study. It is
preferentially delivered to kidneys rather than the liver after being
administered, according to the Nature Communications study.

“We earlier
showed that levels of a tiny RNA fragment called microRNA-17 are increased in
models of ADPKD,” Dr. Patel said. “MicroRNA-17 interferes with the
normal function of other, beneficial RNAs, causing kidney cysts to grow.
RGLS4326, as the new drug is called in development, works by blocking the
harmful microRNA-17.”

Early phase
one clinical trials began last year, conducted by Regulus Therapeutics. The FDA
has asked for additional toxicity information from animal testing before human
trials can move to the next step, Dr. Patel said.

Patents for RGLS4326 are jointly held by UT Southwestern and Regulus, which funded the research, and Dr. Patel has served as a scientific consultant for the company. Dr. Patel’s lab also receives funding from the National Institutes of Health and the Department of Defense.

About UT Southwestern Medical Center

UT Southwestern, one of the premier academic medical centers in the USA., integrates pioneering biomedical research with exceptional clinical care and education.

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