SILVER SPRING, Md. and BOZEMAN, Mont.—The U.S. Food and Drug Administration (FDA) announced today that it has awarded two new research grants for natural history studies in rare diseases. The FDA will be providing over $4.1 million in funding over the next four years for these studies.
Natural history studies closely look at how specific diseases progress over time. The natural history of a disease is the course a disease takes from its onset, through the presymptomatic and clinical stages to a final outcome in the absence of treatment. Information from natural history studies can facilitate the design of efficient clinical trials to test future treatments.
“The FDA is committed to funding these important studies in order to define how rare diseases develop and progress,” said FDA principal deputy commissioner Amy Abernethy, M.D., Ph.D. “Further, these studies provide important roadmaps for how to conduct subsequent studies. With the natural history of many rare diseases remaining relatively unknown, efficiently developing diagnostics and therapies for patients poses unique challenges. Promoting and conducting work in this area is critical.”
The FDA has received 31 grant applications that were reviewed and evaluated for scientific and technical merit by more than 45 rare disease, natural history, regulatory and statistical experts that included representatives from academia, patient groups, the National Institutes of Health (NIH) and the FDA.
One of the grants was awarded to the University of Texas MD Anderson Cancer Center in Houston, Texas, and Elizabeth Grubbs, M.D., for a prospective study in medullary thyroid carcinoma which will receive approximately $1.7 million over four years.
The study seeks to leverage a multi-institutional registry to characterize disease variables and patient perspectives that inform decisions regarding initiation of and adherence to chemotherapy in medullary thyroid cancer. This knowledge can be incorporated into the design of clinical trials of emerging therapies for this disease.
The second grant was awarded to Vanderbilt University Medical Center in Nashville, Tennessee, and Jonathan Soslow, M.D., M.S.C.I., for a prospective study in cardiac disease in Duchenne muscular dystrophy. The study will receive approximately $2.4 million over four years.
This study aims to focus on cardiomyopathy, which is the leading cause of death in Duchenne muscular dystrophy, and will combine genetic differences with imaging and blood biomarkers to identify surrogate biomarkers that predict the risk of cardiac dysfunction in Duchenne muscular dystrophy and other related diseases. This information has the potential to improve future clinical trial efficiency in these diseases by decreasing their size and cost.
Today also brings news that the NIH has given two HEAL Initiative awards to Montana Molecular. The NIH HEAL Initiative is focused on improving prevention and treatment strategies for opioid misuse and addiction, and enhancing pain management. The awards will support the optimization and validation of preclinical cell-based assays for the development of non-addictive treatments for pain and opioid use disorder (OUD).
Montana Molecular’s platform uses novel genetically-encoded fluorescent biosensors that monitor spatial and temporal information from signals which mediate pain and addiction in living cells. A grant from the National Institute on Drug Abuse will support research and development on unique preclinical assays for quantifying biased agonism, an important aspect of pain physiology. Information from these assays will be used to rank lead compounds for effectiveness in pain relief, while hopefully minimizing the adverse effects of OUD.
“Our platform for monitoring the signals that mediate both pain and addiction in live neuronal cells empower drug discovery teams who need to prioritize lead compounds before advancing to clinical trials,” noted Anne Marie Quinn, MPH, chief executive officer of Montana Molecular, in a press release.
Montana Molecular’s ASPIRE Design Challenge Prize from the National Center for Advancing Translational Sciences at NIH will also support deployment of the company’s unique fluorescent biosensor assays in sensory neurons so that new pain medications can be evaluated in a biologically relevant model for pain before advancing to clinical trials.
“It’s clear that a multi-pronged scientific approach is needed to reduce the risks of opioids, accelerate development of effective non-opioid therapies for pain, and provide more flexible and effective options for treating addiction to opioids,” concluded NIH director Francis S. Collins, M.D., Ph.D., who launched the initiative in early 2018. “This unprecedented investment in the NIH HEAL Initiative demonstrates the commitment to reversing this devastating crisis.”
