FDA Awards 12 New Research Grants for Rare Diseases, Including AML

The U.S. Food and Drug Administration (FDA) has awarded 12 new grants totaling more than $1 million for the development of medical devices, drugs, biologics, and medical foods for patients with rare diseases, including acute myeloid leukemia (AML).

Awarded through the congressionally funded Orphan Products Clinical Trials Grants Program, the grants will contribute to marketing approval for products for rare diseases and provide clinical data necessary for their development.

As part of the selection process, more than 100 rare disease experts reviewed 89 clinical trial grant applications. Several grants researching hematologic disorders were funded. Cincinnati Children’s Hospital Medical Center received $1.7 million over four years to support a phase II study of chemoprevention with quercetin, a plant flavonol, for squamous cell carcinoma in patients with Fanconi anemia. The agency also awarded $1 million over four years to MD Anderson Cancer Center to support a phase I/II trial looking at the highest-tolerated dose of an imipridone (a class of drugs that targets G-protein coupled receptors) alone and combined with low-dose cytarabine for patients with relapsed/refractory AML, acute lymphocytic leukemia, or myelodysplastic syndromes.

Institutions also have received funding for studies related to daily vitamin D for patients with sickle cell disease–related respiratory complications, gastrointestinal stromal tumor, T-cell immunodeficiency, and more.

“We are pleased to continue to support research for a variety of rare diseases that have little, or no, treatment options for patients. By helping to spark research, we hope to speed the development of products for rare diseases, and ultimately, make needed treatments available to those patients who need them most,” said Janet Maynard, MD, director of the FDA’s Office of Orphan Products Development.

Source: FDA news release, October 8, 2019.