Citing Science.org, the U.S. Advanced Research Projects Agency for Health (ARPA-H) recently announced its first program targeting a specific disease, which will focus on osteoarthritis. The degenerative condition affects approximately 32 million people in the United States and causes the breakdown of cartilage in the joints, leading to pain and mobility issues. Patients are typically treated with physical therapy and anti-inflammatory drugs, and metal joint replacements are offered when necessary.
ARPA-H’s new program, Novel Innovations for Tissue Regeneration in Osteoarthritis, aims to use a patient’s own cells to regenerate lost bone and cartilage. The federal funder, launched last year, is charged with taking bold, innovative approaches to health research and is modeled on the applications-focused, outside-the-box science sponsored by the Defense Advanced Research Projects Agency.
In other news, a vaccine designed to protect infants from respiratory syncytial virus (RSV) received unanimous support from a panel advising the U.S. Food and Drug Administration (FDA). RSV is a leading cause of infant hospitalization and can cause severe lung infections. The vaccine, called RSVpreF and branded Abrysvo, is given to pregnant people and causes mothers to produce protective antibodies that their babies acquire during pregnancy. The Pfizer vaccine was found to be 69.4% efficacious in protecting infants younger than 7 months from severe disease.
However, four panel members were not persuaded due to an elevated rate of premature births in the vaccinated group versus the placebo group. The difference did not reach statistical significance, and neonatal deaths did not increase. The FDA is expected to rule in August on whether to license the vaccine. Lower respiratory tract infections from RSV kill an estimated 46,000 babies younger than 7 months every year, with hundreds of them in the United States.
Meanwhile, large clinical trials testing cancer therapies frequently alter their primary endpoint, Citing Science.org. The goal posts are moved to better reflect the latest scientific understanding of the disease and the therapy’s mechanism of action. These changes can lead to delays and additional costs for clinical trials, which can already be prohibitively expensive.
In summary, ARPA-H’s new program targeting osteoarthritis aims to use a patient’s own cells to regenerate lost bone and cartilage. A vaccine designed to protect infants from RSV received unanimous support from an FDA advisory panel, but some members were not persuaded due to an elevated rate of premature births. Finally, large clinical trials testing cancer therapies frequently alter their primary endpoint to better reflect the latest scientific understanding of the disease and the therapy’s mechanism of action.
