By Sharon Atieno

In a bid to accelerate rare disease research and offer new options for patients suffering from ailments that lack effective treatments, the Rare Genomics Institute (RG), has announced the opening of their annual competition for grants for rare disease research.

The initiative referred to as BeHEARD(Helping Empower and Accelerate Research Discoveries) seeks to overcome the gap in funding to conduct research and clinical trials for diseases that affect a small number of people.

“There are 7,000 known rare diseases affecting over three hundred million people worldwide, but treatments are available for less than 5 percent of those diseases,” explains Danielle Fumagalli, BeHEARD Program Director.

“Often, this is not due to any inherent difficulties in developing successful therapies, but a severe lack of both private and public sector funding to conduct research and clinical trials for diseases that affect a relatively small number of people. The BeHEARD competition seeks to overcome this gap by partnering with biotech companies to put critical resources into the hands of those seeking solutions to rare disease.”

Since the initiative’s start in 2012, BeHEARD has provided over $2.2 million in grants to support rare disease researchers. Every year, between 20-30 research groups encompassing 15-20 rare diseases are supported. So far, it has supported research on Sanfilippo syndrome, giant congenital melanocytic nevus (CMN), Vici syndrome, pulmonary hypertension (PH) or high blood pressure in the lungs, and many others.

“When the research is successful they usually publish their discoveries and obtain more funding to further develop the cure, for example,  using our BeHEARD prize, Jonah’s Just Begun foundation won an NIH grant for $223,102 for the first-ever pre-clinical research for a treatment for Sanfilippo Subtype D,” says Dr. Arvin Gouw, Vice President for Research and Development, RG.

The competition is open globally to researchers, foundations, or anyone whose idea is constrained due to limited resources.

Families with a child afflicted with a rare disease can be connected with a network of academic researchers to develop research proposals tailored to advancing treatments for their child’s disease. Applications are simple and can be accessed on the BeHEARD website.

“Evaluation of the proposals is not based on attractive scientific hypotheses, the key factor we always consider is: Will this research make a real impact to advance rare disease treatments?” says Dr.Gouw.

“Too often, rare disease patients are left undiagnosed and untreated through years of going in and out of hospitals. Our goal is to bring hope of a cure to everyone.”

The grant application will close on April 30,2020 with prizes including more than $85,000 of sponsored cutting-edge technologies and services from top scientific companies, such as vectors from Addgene, multiplex panel design software from DNA Software Inc., antibodies from Novus Biologicals/BioTechne and mouse models from Taconic.