What
A clinical trial led by researchers at the National Institutes of Health (NIH) has demonstrated that an experimental gene therapy called PRGN-2012 may be an effective treatment for patients with recurrent respiratory papillomatosis (RRP). This rare, debilitating condition, caused by chronic infection with human papillomavirus (HPV) types 6 and 11, produces noncancerous tumors, or papillomas, throughout the respiratory tract that affect a person’s voice and breathing. The results were published on January 21, 2025, in The Lancet Respiratory Medicine.
Doctors currently use surgery to manage RRP, but repeated surgeries can eventually damage a person’s vocal cords and airways. PRGN-2012 uses a modified gorilla adenovirus, designed by the biopharma company Precigen, to deliver genes that elicit immune responses directed against cells infected with HPV 6 or HPV 11. In the phase 1/2 trial, 35 adults with RRP were treated with PRGN-2012 following papilloma-removal surgery. Of these patients, 18 did not need any procedures to remove papillomas in the year after treatment. After a median follow-up of 22 months, 15 patients still did not need further procedures. Side effects, which included fatigue and chills, were mild.
The trial was conducted as part of a Cooperative Research and Development Agreement between NIH’s National Cancer Institute and Precigen.
Who
Clint T. Allen, M.D., and Scott M. Norberg, D.O., Center for Cancer Research Center, National Cancer Institute
The Study
“PRGN-2012 gene therapy in adults with recurrent respiratory papillomatosis: a pivotal phase 1/2 clinical trial” appeared January 21, 2025, in The Lancet Respiratory Medicine.
About the National Cancer Institute (NCI): NCI leads the National Cancer Program and NIH’s efforts to dramatically reduce the prevalence of cancer and improve the lives of people with cancer. NCI supports a wide range of cancer research and training extramurally through grants and contracts. NCI’s intramural research program conducts innovative, transdisciplinary basic, translational, clinical, and epidemiological research on the causes of cancer, avenues for prevention, risk prediction, early detection, and treatment, including research at the NIH Clinical Center—the world’s largest research hospital. Learn more about the intramural research done in NCI’s Center for Cancer Research. For more information about cancer, please visit the NCI website at cancer.gov or call NCI’s Cancer Information Service, at 1-800-4-CANCER (1-800-422-6237).
About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit nih.gov.
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