FDA Awards Research Grants for the Study of Rare Cancers
The U.S. Food and Drug Administration (FDA) recently awarded 12 new clinical trial research grants totaling more than $15 million over the next 4 years, many of them focusing on cancer. Also, the FDA awarded two new research grants for natural history studies in rare diseases, totaling more than $4.1 million over the next 4 years, one of which focuses on medually thyroid carcinoma. These grants are intended to enhance the development of medical products for patients with rare cancers and diseases.
Janet Maynard, MD
“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,” said Janet Maynard, MD, Director of the FDA’s Office of Orphan Products Development.
Clinical Research Grants
The principal investigators, research topics, and approximate funding amounts for the clinical trial research grants follow:
- Maryam Fouladi, MD, MSc, FRCPC, Cincinnati Children’s Hospital Medical Center: Phase I study of PTC596 for the treatment of diffuse intrinsic pontine glioma and high-grade gliomas ($750,000 over 3 years)
- Parinda Mehta, MD, Cincinnati Children’s Hospital Medical Center: Phase II study of quercetin chemoprevention for the treatment of squamous cell carcinoma in patients with Fanconi anemia ($1.7 million over 4 years)
- Sara Pai, MD, PhD, Massachusetts General Hospital, Boston: Phase II study of anti–PD-1 therapy for HPV-associated recurrent respiratory papillomatosis ($1 million over 3 years)
- Manijeh Goldberg, PhD, MBA, Privo Technologies, Peabody, Massachusetts: Phase I/II study of cisplatin patch (PRV111) for the treatment of oral cancer ($2 million over 4 years)
- Ricardo Carvalho, PhD, Targeted Therapy Technologies, Somerset, New Jersey: Phase I study of episcleral topotecan for the treatment of retinoblastoma ($660,000 over 3 years)
- Gregory Friedman, MD, University of Alabama at Birmingham: Phase I study of oncolytic engineered herpes simplex virus therapy for pediatric malignant cerebellar brain tumors ($750,000 over 3 years)
- Jason Sicklick, MD, FACS, University of California San Diego: Phase II study of temozolomide for the treatment of gastrointestinal stromal tumor ($1.5 million over 3 years)
- Michael Andreeff, MD, PhD, The University of Texas MD Anderson Cancer Center, Houston: Phase I/II study of imipridone (ONC201) for the treatment of acute myeloid leukemia ($1 million over 4 years).
Grant for Natural History Studies
Information from natural history studies can facilitate the design of efficient clinical trials to test future treatments. The FDA 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, including representatives from academia, patient groups, the National Institutes of Health, and the FDA.
The principal investigators research topics, and approximate funding amounts for the natural history study grant follow:
- Elizabeth Grubbs, MD, The University of Texas MD Anderson Cancer Center, Houston: Prospective study in medullary thyroid carcinoma (approximately $1.7 million over 4 years). The study seeks to leverage a multi-institutional registry to characterize disease variables and patient perspectives that inform decisions regarding the initiation of and adherence to chemotherapy in medullary thyroid cancer. Such critical knowledge can be incorporated into the design of clinical trials of emerging therapies for this disease. ■