Gayatri R. Rao, MD, JD
Eighteen new research grants, totaling more than $19 million, have been awarded by the FDA for product development in rare diseases such as HPV-related head and neck cancer and recurrent glioblastoma, the regulatory agency recently announced.
“The FDA is in a unique position to help those who suffer from rare diseases by offering important incentives to promote the development of products, one of which is our grants program,” said Gayatri R. Rao, MD, JD, director of the FDA’s Office of Orphan Product Development, in a statement. “The grants awarded this year support much-needed research [for several] rare diseases, many of which have little, or no, available treatment options.”
Baylor College of Medicine was awarded more than $1.1 million over 3 years to support its ongoing phase II trial of the immunotherapy axalimogene filolisbac (ADXS11-001) for the treatment of HPV-associated oropharynx cancer. ADXS11-001 has been shown to generate T cells directed against a cancer antigen and neutralize suppressor regulatory T cells (Tregs) and myeloid-derived suppressor cells that protect the tumor microenvironment from an immunologic attack and contribute to tumor growth.
“Applications to the Orphan Products Grants Program undergo rigorous review for scientific and technical merit by a panel of experts to compete for funding,” said Daniel J. O’Connor, president and chief executive officer of Advaxis, the company manufacturing ADXS11-001, in a statement. “We are encouraged to see axalimogene filolisbac recognized as viable by being awarded a grant for its clinical research that could ultimately contribute to market approval.”
The trial is aimed to evaluate the efficacy of ADXS11-001 as a neoadjuvant treatment prior to robotic surgery in patients with HPV-associated oropharyngeal cancer.
“We hope to improve outcomes in HPV-associated head and neck cancer by exploring axalimogene filolisbac in this indication, where existing treatment options are associated with risk of long-term morbidity,” said lead study author Andrew G. Sikora, MD, associate professor of Otolaryngology at Baylor College of Medicine, and co-director of the Head and Neck Cancer Program at Dan L. Duncan Cancer Center, in a statement. “If successful, this trial could pave the way for immunotherapy to become a standard treatment for HPV-associated cancers.”
A $1.6 million grant over 4 years was also awarded to the University of California San Diego for a phase II study of G-202, a vascular-targeted prodrug, for the treatment of recurrent glioblastoma.
The FDA grants, which come from the Orphan Products Grants Program, were awarded to principal investigators in 10 states whose research covers domestic and international clinical sites.
Additional sites that received funding have studies related to sickle disease, acute kidney injury, AL amyloidosis, X-linked hypohidrotic ectodermal dysplasia, neurofibromatosis, autosomal dominant polycystic kidney disease, familial dysautonomia, mesenteric panniculitis, Prader-Willi Syndrome, epidermolysis bullosa, desmoid tumors or aggressive fibromatosis, pachyonychia congenita, sporadic inclusion body myositis, and parenteral nutrition-associated cholestasis. One grant will help fund a phase II study for a selective cytopheretic device for the treatment of pediatric patients with acute kidney injury.
More than $350 million has been awarded since the program’s creation in 1983 to fund more than 570 new clinical trials and support marketing approval of over 50 products. The program receives approximately 100 applications annually.
Rare diseases affect nearly 30 million Americans. HPV-related throat cancer is currently the fastest-growing type of head and neck cancer, with an incidence of approximately 15,500 new cases of HPV-associated oropharynx cancer in the United States each year.
ADXS11-001 is being examined in other tumor types, including cervical cancer. In a phase II study recently reported by the Gynecologic Oncology Group, patients with recurrent/refractory cervical cancer who received the vaccine either alone or combined with chemotherapy demonstrated prolonged survival, objective tumor responses, and a manageable safety profile.