FDA Awards Research Grants for 12 Rare Diseases, Including GIST, AML, and Brain Tumors

The FDA has awarded 12 new research grants that together total more than $15 million, to enhance the development of medical products for patients with rare diseases, including acute myeloid leukemia (AML) and gastrointestinal stromal tumor (GIST), among others.¹

The grants are awarded through the Congress-funded Orphan Products Clinical Trials Grants Program, which is designed to encourage clinical development of drugs, biologics, medical devices, and medical foods for the treatment of patients with rare diseases. Additionally, the grants are planned to contribute to marketing approval of products for these rare diseases or provide the necessary clinical data that are required for development of such products, the agency stated in a press release.

“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,” Amy Abernethy, MD, PhD, FDA principal deputy commissioner, stated in the press 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.”

Eighty-nine clinical trial grant applications were reviewed by more than 100 rare disease experts, which also includes those of academia. The FDA added in their statement that 75% of the new awards fund trials that are enrolling children, some of whom are ≥1 month old.

Cincinnati Children’s Hospital Medical Center was awarded $750,000 over 3 years to support its phase I trial (NCT02404480) of the small molecule PTC596 for the treatment of patients with diffuse intrinsic pontine glioma and high-grade gliomas. Phase I data that were presented at the 2017 ASCO Annual Meeting showed that PTC596, which is designed to target cancer stem cells, is tolerable with manageable gastrointestinal adverse events.²

Additionally, Cincinnati Children’s Hospital Medical Center also received $1.7 million over 4 years to support the institution’s phase II study (NCT03476330) of quercetin chemoprevention for the treatment of squamous cell carcinoma in patients with Fanconi Anemia. Investigators hypothesize that the use of quercetin could prevent or delay the development of squamous cell carcinoma in this patient population.

Two million dollars was granted to Privo Technologies, LLC, for their phase I/II study (NCT03502148) of the cisplatin transmucosal system PRV111 as a potential treatment for patients with oral cancer. In the trial, following therapy with PRV111, patients are monitored for local and systemic safety, tumor response to treatment, and systemic drug exposure.

A $1.5 million grant over 3 years was also awarded to the University of California San Diego for a phase II study (NCT03556384) of temozolomide for the treatment of patients with advanced or metastatic GIST, specifically those who have SDH mutations or deficiencies.

At the University of Alabama at Birmingham, a phase I trial (NCT03911388) of oncolytic engineered herpes simplex virus therapy for the treatment of pediatric malignant cerebellar brain tumors was awarded $750,000 over 3 years. The goal of the early-phase trial is to determine the safety of the investigational therapy.

Moreover, the agency awarded $1 million over 4 years to The University of Texas MD Anderson Cancer Center to support a phase I/II trial (NCT02392572) of imipridone (ONC201) as a potential therapy for patients with AML. The study is looking at the highest-tolerated dose of imipridone alone and in combination with low-dose cytarabine that can be administered to patients with relapsed/refractory AML, acute lymphoblastic leukemia, or myelodysplastic syndrome.

Further sites that received funding have studies related to complement 3 glomerulopathy, daily vitamin D for the treatment of patients with sickle-cell respiratory complications, cardiomyopathy associated with Duchenne muscular dystrophy, HPV-associated recurrent respiratory papillomatosis, refractory viral infections and T-cell immunodeficiency, and retinoblastoma.

“The majority of rare diseases still do not have approved therapies and the FDA is committed to fostering product development in areas of unmet need. The Orphan Products Grants Program is one of several ways that the FDA supports the development of products for rare diseases. Since its creation in 1983, the program has provided more than $400 million to fund more than 600 new clinical studies,” Janet Maynard, MD, director of the FDA’s Office of Orphan Products Development, stated in the press release.

“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.”

Past studies funded by this program that have resulted in or contributed to recent approvals, include fish oil triglycerides as a source of calories and fatty acids in children with parenteral nutrition-associated cholestasis, and tafamidis meglumine and tafamidis for patients with heart disease that is caused by transthyretin mediated amyloidosis.

References

1. FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases. FDA. Published October 8, 2019. https://bit.ly/31YzAUd. Accessed October 8, 2019.
2. Infante JR, Bedard PL, Shapiro G, et al. Phase 1 results of PTC596, a novel small molecule targeting cancer stem cells (CSCs) by reducing levels of BMI1 protein. J Clin Oncol. 2017;35(suppl; abstr 2574). doi: 10.1200/JCO.2017.35.15_suppl.2574.