A new potential drug that could treat polycystic kidney disease was found. The disease is incurable, and it often happens to lead to end-stage kidney failure.
The study, published in Nature Communications, on Feb. 16, was conducted by researchers at the UT Southwestern Medical Center, who collaborated with a California-based biotech company.
Polycystic Kidney Disease, Potential Treatment Found
The new medicine that could potentially cure polycystic kidney disease is called RGLS4326, and it is currently in preclinical animal testing. According to assistant professor Vishal Patel, senior author of the study, an investigational drug associated with this finding should be available later in 2017.
The disease manifests through an abnormal growth of the kidney due to cysts filled with fluid, which keep growing in size until they eventually prevent the organ for serving its functions. Due to this, the kidney loses its capacity to remove bodily waste and filter blood.
According to the researchers, the patients can have this disease for decades until the first symptoms show up, such as blood in the urine. At the moment, there is no available drug on the market to treat this disease, and the only two available options once the kidney reaches failure are dialysis or a transplant.
Back in 2009, Dr. Patel started to look for microRNAs (MiRs) that could provide a better understanding on the disease. These MiRs are very small pieces of RNA that can interfere with normal gene expression.
Researchers discovered the role of MiRs back in the 1990's, and the scientific interest in finding a viable drug to treat diseases caused by these small RNA fragments increased rapidly. The reason for this scientific interest is that, once the fragment is found, the rest of the scientific process can evolve rapidly.
Because of the fragments' small dimensions, researchers can easily create drugs to annihilate their actions. Once the researchers identify the RNA fragment, the only thing left to do is preparing an anti-MiR with the very opposite sequence.
As part of a 2013 research, the scientists focused their efforts on finding MiR clusters in the attempt to identify potential viable targets. As a result to that research they published a paper with a potential RNA sequence that they found promoted kidney cyst growth, called the 17~92 sequence.
In the current study, the researchers employed mouse models, in which they inhibited the microRNA sequence, finding that the genetic deletion of the 17~92 sequence slowed cyst growth. Additionally, the lifespan of the tested mice increased more than twice its initial value.
"In support of this conclusion, we show that genetic deletion of miR-17∼92 attenuates disease progression in ADPKD mouse models irrespective of the mutated gene, the type of mutation (null or hypomorphic) or the dynamics of cyst growth (rapidly fatal, aggressive but long-lived or slowly progressing)," noted the study.
Polycystic Kidney Disease In The United States
Polycystic kidney disease is a genetic disorder that affects approximately 600,000 people in the United States, about half of those affected with the disease experience kidney failure by the age of 60. The cysts cause high blood pressure, as well as problems with blood vessels in the heart and brain, according to the National Institute of Diabetes and Digestive and Kidney Diseases.
For the patients who are currently suffering from this disease, it is highly recommended to regulate a healthy diet, in order to have control on blood pressure.
