Research Horizons


Two Cincinnati Children’s Scientists Earn FDA Rare Disease Clinical Trial Grants

The U.S. Food and Drug Administration (FDA) announced on Oct. 8, 2019, that it has awarded 12 new clinical trial research grants totaling more than $15 million over the next four years to enhance the development of medical products for patients with rare diseases.

Two of those grants were awarded to scientists at Cincinnati Children’s.

Maryam Fouladi, MD, MSc, Medical Director of the Brain Tumor Center here, was awarded a three-year $750,000 grant to fund a Phase 1 study of PTC596 for the treatment of diffuse intrinsic pontine glioma & high-grade gliomas.

Parinda Mehta, MD, Division of Bone Marrow Transplantation and Immune Deficiency, was awarded a four-year $1.7 million grant to fund a Phase 2 study of quercetin chemoprevention for the treatment of squamous cell carcinoma in patients with Fanconi Anemia.

The FDA awarded the grants through its Orphan Products Clinical Trials Grants Program.

“For more than 35 years, the FDA has been providing much-needed financial support for clinical trials of potentially life-changing treatments for patients with rare diseases. To date, the Orphan Products Clinical Trials Grants Program’s grants have supported research that led to the marketing approval of more than 60 treatments for rare diseases,” said FDA Principal Deputy Commissioner Amy Abernethy, MD, PhD, in a news release. “We are encouraged by the amount of interest we continue to have in the grants program and are committed to working with researchers and industry to facilitate and support the study and development of treatments for patients with rare diseases.”

The 12 grantees were selected from 89 applications.

Read the FDA announcement