Gene therapy shows clinical benefit in patients with a debilitating disorder caused by HPV
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In patients with recurrent respiratory papillomatosis, multiple benign tumors (red) can form in the respiratory tract. An experimental gene therapy has been shown to shrink these tumors.
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.
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