FDA Funding For Disease Research
FDA recently announced the approval of two grants totaling $4.1 billion for rare disease research. The awards will specifically go to research for medullary thyroid carcinoma and Duchenne muscular dystrophy. It is expected that these subsequent findings will likely help researchers better understand the pathology of several illnesses.
The US FDA approved approximately $1.7 million over four years for research into medullary thyroid carcinoma and roughly 2.4 million dollars over four years into research on Duchenne muscular dystrophy.
Congress appropriates funding to The U.S. Food and Drug Administration to use for study on the disease, but often the most common diseases receive the majority of this funding. This new move will help spread resources to allow scientists to explore potential new treatments for less frequent diseases.
With the natural history of many rare disorders remaining relatively unknown, efficiently developing diagnostics and therapies for patients poses unique challenges.
Amy Abernethy, M.D., Ph.D. FDA Principal Deputy Commissioner, highlighted that FDA is committed to funding these critical studies to define how rare diseases develop and progress. Promoting and conducting work in this area is vital.
Medullary thyroid carcinoma is a rare form of thyroid cancer. It accounts for
only about 3% of all thyroid cancers. This form of thyroid cancer is caused by genetic alterations of cells found within the thyroid gland.FDA Funding For Disease Research- Scientists Who Received The Grants
The grant was awarded to the University of Texas MD Anderson Cancer Center in Houston, Texas, Elizabeth Grubbs’s prospective study.
The study aims to leverage a multi-institutional registry to characterize disease variables and patient perspectives that inform decisions regarding initiation and adherence to chemotherapy in medullary thyroid cancer.
The second grant by the USFDA was awarded to Vanderbilt University Medical Center (Nashville, Tennessee), Jonathan Soslow, for prospective research in cardiac disease in Duchenne muscular dystrophy. Duchenne muscular dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness.
This study aims to focus on cardiomyopathy (heart muscle disease), which is the leading cause of death in Duchenne muscular dystrophy. For individuals with DMD, cardiac disease, caused by the weakening of the heart muscles, is a common cause of death.
The grant money will be used to combine genetic differences with imaging and blood biomarkers, which may allow doctors to predict the risk of cardiac disease in not only DMD but also other related illnesses.
