A researcher at the Feinstein Institute for Medical Research has been awarded two grants for clinical trials on existing FDA-approved drugs that can be indicated to treat hereditary hemorrhagic telangiectasia.
A researcher at the Feinstein Institute for Medical Research has been awarded two grants for clinical trials on existing U.S. Food and Drug Administration (FDA)-approved drugs that can be indicated to treat hereditary hemorrhagic telangiectasia (HHT), a rare bleeding condition.
Professor Philippe Marambaud, PhD, received a 4-year, $2 million grant from the Department of Defense (DOD), the largest grant ever awarded to research HHT. In addition, Dr. Marambaud has been awarded a 4-year, $1.6 million award from the National Institutes of Health (NIH).
HHT is an inherited disorder in which the blood vessels can cause excessive bleeding. Abnormal blood vessels, called arteriovenous malformations, can develop in several areas of the body, including the brain, lungs, liver or intestines. Once AVMs appear on the skin, they are called telangiectasias.
When bleeding occurs, doctors are able to cauterize the point of bleeding. However, these interventions are unable to be performed in some organs, including the liver. A liver transplant is required in patients who experience bleeding in the organ.
"Currently there is no treatment for hereditary hemorrhagic telangiectasia other than trying to stop the bleeding," said Dr. Marambaud in a press release. "We're extremely grateful for the support from the DOD and the NIH to allow us to continue our search for a treatment for this highly debilitating disease."
Dr. Marambaud and his fellow researchers plan on identifying therapies tailored to correct the molecular defects that cause HHT and reverse the development of abnormal vessels.
In the DOD study, Dr. Marambaud and Professor Lionel Blanc, PhD, a Feinstein institute Associate, will find potential drug candidates that treat HHT from pre-existing FDA approved drugs indicated for the reduction of loss of blood vessel function. The findings will be tested in human clinical trials at Toronto University, the largest HHT care center in North America. The DOD is advocating for more research on vascular pathologies that can be debilitating for military personnel.
Dr. Marambaud and his team of researchers will work to understand the genetic causes of HHT to identify new therapies. Mutations that cause HHT are mostly found in the ALK1 and ENG genes, leading to a loss of function.
The studies Dr. Marambaud will lead have the potential to receive fast-track designation for the first therapies that treat HHT.
Kevin J. Tracey, MD, president & CEO of the Feinstein Institute said: "Dr. Marambaud's research to repurpose FDA-approved medications offers a more efficient path for new therapies that are desperately needed for this devastating illness."