FDA Grants Spur Rare Disease Trials

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The U.S. Food and Drug Administration (FDA) has awarded 12 new clinical trial research grants totaling more than $18 million over the next four years to “enhance the development of medical products for patients with rare diseases,” the agency said yesterday (September 24).

FDA also released the names of grant recipients, principal investigators (PIs), and approximate funding amounts:

  • Alkeus Pharmaceuticals, Inc. (Cambridge, Mass.), Leonide Saad, Phase II study of ALK-001 for the treatment of Stargardt disease – $1.75 million over four years
  • Arizona State University-Tempe Campus (Tempe, Ariz.), Keith Lindor, Phase II study of oral vancomycin for the treatment of primary sclerosing cholangitis – $2 million over four years
  • Cedars-Sinai Medical Center (Los Angeles, Calif.), Shlomo Melmed, Phase II study of seliciclib for the treatment of Cushing disease – $2 million over four years
  • Columbia University of New York (New York City), Yvonne Saenger, Phase I study of talimogene laherparepvec for the treatment for advanced pancreatic cancer –  $750,000 over three years
  • Emory University (Atlanta, Ga.), Eric Sorscher, Phase I/II study of Ad/PNP fludarabine for the treatment of head and neck squamous cell carcinoma – $1.5 million over three years
  • Fibrocell Technologies, Inc. (Exton, Pa.), John Maslowski, Phase I/II study of gene-modified ex-vivo autologous fibroblasts for the treatment of dystrophic epidermolysis bullosa – $1.5 million over four years
  • Johns Hopkins University (Baltimore, Md.), Amy Dezern, Phase I/II study of CD8-reduced T cells for the treatment of myelodysplastic syndrome or acute myeloid leukemia – $750,000 over three years
  • Oncolmmune, Inc. (Rockville, Md.) Yang Liu, Phase IIb study of CD24Fc for the prevention of graft versus host disease – $2 million over four years
  • Patagonia Pharmaceuticals, LLC (Woodcliff Lake, N.J.), Zachary Rome, Phase II study of PAT-001 (isotretinoin) for the treatment of congenital ichthyosis – $1.5 million over three years
  • The General Hospital Corporation (Boston, Mass.), Stephanie Seminara, Phase II study of kisspeptin for the treatment of dopamine agonist intolerant hyperprolactinemia – $1.4 million over four years
  • University of Minnesota (Minneapolis, Minn.), Kyriakie Sarafoglou, Phase IIa study of subcutaneous hydrocortisone infusion pump for the treatment of congenital adrenal hyperplasia – $1.4 million over three years
  • University of North Carolina at Chapel Hill (Chapel Hill, N.C.), Matthew Laughon, Phase II study of sildenafil for the prevention of bronchopulmonary dysplasia – $2 million over four years

Four of the new awards hope to accelerate cancer research by enrolling patients with rare forms of cancer, including advanced pancreatic cancer, head and neck squamous cell carcinoma, myelodysplastic syndrome, and acute myeloid leukemia. Three fund studies evaluating drug products for rare endocrine disorders, including Cushing disease.

Five of the grants fund studies which enroll children and adolescents, targeting a variety of rare diseases in children such as Stargardt disease, a juvenile genetic eye disorder that causes progressive vision loss; dystrophic epidermolysis bullosa, a genetic condition that causes the skin to be fragile resulting in painful blisters; and bronchopulmonary dysplasia, a serious lung condition that affects infants.

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

Author: Michael Causey