Initial funding from PKD Foundation leads to collaboration for potential ADPKD treatment

As the PKD Foundation-funded 2014-2016 research grants concluded, rich new data were uncovered, continuing to move us closer to treatments. Among highlights are outcomes from Dr. Vishal Patel’s study, which focused on the role and function of microRNA clusters in autosomal dominant polycystic kidney disease (ADPKD).

With the initial funding from the PKD Foundation and additional funding from the National Institutes of Health, Dr. Patel’s research has led to a collaboration with a biotech company to investigate a promising treatment for slowing cyst growth in ADPKD patients.

Significance of microRNA for ADPKD treatments

MicroRNA, a type of RNA found in our cells, serves to regulate gene expression, a key factor in many diseases, including ADPKD. According to Dr. Patel’s research results, one specific cluster of microRNAs, miR-17~92, affects kidney cyst growth in mouse models. Overexpression of miR-17~92 produces kidney cysts in mouse models, while inactivation of miR-17~92 slows cyst growth and improves kidney function.

Based on research such as Dr. Patel’s, drugs designed to inactivate these microRNA are emerging as a potential way to treat PKD. Dr. Patel’s ongoing collaboration with the biotech company has resulted in the development of a new, anti-miR-17drug compound for the treatment of ADPKD.

Credit: UT Southwestern Medical Center
Researchers (from left) Andrea Flaten, Matanel Yheskel, Dr. Vishal Patel, Dr. Sachin Hajarnis, and Dr. Ronak Lakhia

“Work funded initially by the PKD Foundation and eventually by the NIH provided the scientific rationale to develop drugs that target the miR-17 cluster” says Dr. Patel. “As we embark on additional preclinical studies, we are excited and optimistic that anti-miR-17 drugs will prove to be safe and effective”.

The biotech company hopes to file an Investigational New Drug (IND) program application with the U.S. Food and Drug Administration in 2017. The IND application is the first step toward initiating clinical trials in humans.

Getting the best minds to work on finding treatments for PKD

The PKD Foundation’s research grant program strives to fund a diverse range of PKD research projects in order to broaden our understanding of the disease and encourage researchers to explore a wide variety of potential treatments. Every two years, we award research grants to 15 outstanding PKD researchers for a total investment of $2.4 million over each two-year period. Each grant recipient, like Dr. Patel, receives $80,000 per year for two years. Projects are selected based on their potential to increase our understanding of the genetic and pathological processes involved in PKD and to accelerate the development of potential therapies for PKD patients.

About Vishal Patel, M.D.

Dr. Vishal Patel is an Assistant Professor of Medicine at the University of Texas Southwestern Medical Center in Dallas, Texas. He obtained his medical degree in India, and then completed his internal medicine residency training at the McGraw Medical Center of Northwestern University in Chicago, and a clinical nephrology fellowship and a basic science fellowship sponsored by the National Institutes of Health at the University of Texas Southwestern Medical Center. Dr. Patel has received numerous awards for academic achievement during medical school and a career development award from the NIH. He was also named a ‘rising star’ in Nephrology by Texas Monthly magazine.

Dr. Patel runs a pre-clinical polycystic kidney disease research laboratory at UT Southwestern. His research is focused on understanding the role of microRNAs in PKD progression and developing microRNA-based drugs for PKD. His laboratory is funded by grants from the NIH and the PKD foundation. In addition to research, Dr. Patel also sees patients at Parkland Hospital in Dallas, Texas. He is planning to launch a new multidisciplinary clinic in the coming months with the goal of providing comprehensive clinical care for PKD patients at UT Southwestern.