In early October, the U.S. Food and Drug Administration awarded 12 new clinical trial research grants to advance treatments for rare diseases. One of the trials is a phase 2 study of quercetin chemoprevention for the treatment of squamous cell carcinoma in patients with Fanconi anemia. The trial, led by Dr. Parinda Mehta at Cincinnati Children’s Hospital Medical Center, will receive $1.7 million over four years.
Initial funding to support a portion of the phase 2 trial was provided by donors of the FA Research Fund in 2018. “We are very thankful to FARF for providing the seed money for this study,” said Dr. Mehta. “That funding covered the cost of study initiation and participation of the first few patients and eventually led to additional extramural funding to cover the cost of the entire study.”
New approaches are needed for both prevention and treatment of squamous cell carcinoma (SCC) in patients with FA - approaches that have fewer and less severe side effects than chemotherapy and radiation therapy. The purpose of this Phase 2 study is to use quercetin (a naturally occurring anti-oxidant) treatment for two years to prevent or delay development of SCC in 45 post-transplant and 10 pre-transplant patients with FA. Clinical and laboratory tests will determine whether quercetin is effective at reducing oxidative stress and DNA damage. This could lead to a new prevention strategy for SCC in post-transplant patients with FA that could eliminate or at least delay the development of SCC.