Abstract: INFO14-FR
Bempedoic Acid Therapy for Polycystic Kidney Disease (BEAT-PKD) Randomized Clinical Trial
Session Information
- Informational Posters - 2
October 23, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- No subcategory defined
Authors
- Pastor-Soler, Nuria M., University of Vermont, Burlington, Vermont, United States
- Seliger, Stephen L., University of Maryland Baltimore School of Medicine, Baltimore, Maryland, United States
- Miskulin, Dana, Tufts Medical Center, Boston, Massachusetts, United States
- Abebe, Kaleab, University of Pittsburgh, Pittsburgh, Pennsylvania, United States
- Duddalwar, Vinay, University of Southern California, Los Angeles, California, United States
- Gordon, Craig E., Tufts Medical Center, Boston, Massachusetts, United States
- Perrone, Ronald D., Tufts Medical Center, Boston, Massachusetts, United States
- Solomon, Richard J., University of Vermont, Burlington, Vermont, United States
- Watnick, Terry J., University of Maryland Baltimore School of Medicine, Baltimore, Maryland, United States
- Webb, Kimberly, University of Pittsburgh, Pittsburgh, Pennsylvania, United States
- Lalama, Christina M., University of Pittsburgh, Pittsburgh, Pennsylvania, United States
- Diggs, Charalett E., Tufts Medical Center, Boston, Massachusetts, United States
- Fairhurst, Penny A., University of Vermont, Burlington, Vermont, United States
- Gotwals, Kira, University of Vermont, Burlington, Vermont, United States
- Morgan, Madison, University of Pittsburgh, Pittsburgh, Pennsylvania, United States
- Mohammed, Shuaib, Tufts Medical Center, Boston, Massachusetts, United States
- Copelli, Susan J., University of Pittsburgh, Pittsburgh, Pennsylvania, United States
- Hallows, Kenneth R., University of Vermont, Burlington, Vermont, United States
Group or Team Name
- BEAT-PKD Randomized Clinical Trial Investigators
Description
Autosomal dominant PKD (ADPKD), the most commonly inherited kidney disease, is characterized by the development of cysts in the kidney that impair function. Of those affected, half will progress to end-stage kidney disease by age 60, requiring dialysis or kidney transplant. To date, only a single medication – tolvaptan – is approved by the FDA for ADPKD treatment. However, the effect of tolvaptan in slowing kidney function decline is modest, and adverse drug effects including frequent urination and thirst and drug-related liver injury restrict its use. Our goal, therefore, is to develop new strategies to treat ADPKD that are safe, tolerable, and effective. The development of cysts in individuals affected by ADPKD results from two main cellular processes. The first is cell growth with an increase in the number of kidney cells that make up the outer surface of the cyst. The second is an increase in fluid secretion into the cysts. We have shown that an enzyme, AMP-activated protein kinase (AMPK), when activated can inhibit both of those processes. Moreover, other researchers showed that the genetic mutation that causes ADPKD alters the energy metabolism of the cell, which in turn inhibits AMPK activity. Bempedoic acid, a safe medication that is FDA-approved for the treatment of hypercholesterolemia, activates AMPK. In addition to activating AMPK, bempedoic acid inhibits a second enzyme called ATP-citrate lyase (ACLY), which is involved in cholesterol synthesis. ACLY has received growing attention as a novel target for cancer treatment. ACLY inhibition blocks increases of cell numbers by inhibiting the lipid synthesis that is required for new cell membranes. In our preclinical mouse studies bempedoic acid reduced kidney size and improved kidney function by itself and there were additional beneficial effects on kidney size and function when bempedoic acid was combined with tolvaptan. Recruitment sites: U. Vermont, U. Maryland and Tufts Medical Center. Data Coordinating Center: U. Pittsburgh; Imaging center: U. Southern California.
In the BEAT-PKD randomized clinical trial funded by the U.S. Department of Defense, we will test whether treatment with bempedoic acid is safe and tolerable and will help reverse dysfunctional metabolism and slow disease progression in individuals with ADPKD, including those treated with tolvaptan. NCT07282821.
Acknowledgment
With the participation of Dr. Christina Clise and Dr. Todd Conner from the Department of Veterans Affairs, Office of Research & Development, Cooperative Studies Program, Clinical Research Pharmacy Coordinating Center.
Funding
- US Department of Defense HT94252510293