A drug commonly used to treat breast cancer might be able to help people living with hereditary hemorrhagic telangiectasia (HHT) – a rare genetic bleeding condition that causes enlargements and tangles of veins and arteries, as well as other malformations of the blood vessels – according to new research from The Feinstein Institutes for Medical Research. The study, which published in Nature Cardiovascular Research, also shows that HHT could cause certain cells in the endothelium of the blood vessels to multiply via a cancer-like mechanism of abnormal cell growth.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20241101936872/en/

Dr. Philippe Marambaud led the research published in Nature Cardiovascular Research. (Credit: Feinstein Institutes)

Philippe Marambaud, PhD, professor in the Institute of Molecular Medicine at the Feinstein Institutes, led the research that shows a specific process controlled by a protein called CDK6 is making endothelial cells multiply abnormally, directly linking to the blood vessel concerns seen in HHT. Researchers used drugs Palbociclib and Ribociclib, which are known to block the CDK6 protein in multiplying cancer cells and found that it stopped the abnormal cell growth in animal models with HHT who were not following normal growth patterns in their blood vessels.

“Currently, there is no treatment for HHT, a highly debilitating disease that affects more than 1.4 million people worldwide, and little is known about what happens at the molecular level,” said Dr. Marambaud. “However, these findings are a step towards better understanding the cause of HHT and how commonly used drugs could help patients living with it.”

Results from the study suggest that by targeting CDK6 with these drugs, doctors might be able to control the excessive cell growth in blood vessels seen in HHT – which could occur in the brain, lungs, digestive system, skin and other organs.

“Repurposing already approved medications that are proven to be safe and effective for one disease presents an opportunity to develop new therapies needed for vascular disease,” said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes and Karches Family Distinguished Chair in Medical Research. “Dr. Marambaud’s research is a step forward furthering our understanding of HHT and potential therapies.”

This research comes from a $3.6 million grant Dr. Marambaud received in 2018 from the National Institute of Health (NIH) to study repurposing of FDA-approved drugs to treat HHT.

About the Feinstein Institutes:

The Feinstein Institutes for Medical Research is the home of the research institutes of Northwell Health, the largest health care provider and private employer in New York State. Encompassing 50+ research labs, 3,000 clinical research studies and 5,000 researchers and staff, the Feinstein Institutes raises the standard of medical innovation through its six institutes of behavioral science, bioelectronic medicine, cancer, health system science, molecular medicine, and translational research. We are the global scientific leader in bioelectronic medicine – an innovative field of science that has the potential to revolutionize medicine. The Feinstein Institutes publishes two open-access, international peer-reviewed journals Molecular Medicine and Bioelectronic Medicine. Through the Elmezzi Graduate School of Molecular Medicine, we offer an accelerated PhD program. For more information about how we produce knowledge to cure disease, visit http://feinstein.northwell.edu and follow us on LinkedIn.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241101936872/en/