Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation, today announced that it is funding a clinical trial of curcumin in people with cystic fibrosis (CF). CFFT has formed an alliance with SEER Pharmaceuticals, a privately held early-stage pharmaceutical company, which will conduct the trial through CFFT's Therapeutics Development Network (TDN).
CFFT has been working with SEER on a potential curcuminoid therapy for more than a year, but has now made an award to SEER for a Phase I clinical trial to assess the safety and dosage parameters of curcumin. This award comes concurrently with the publication in the journal Science of a CF Foundation-funded study on curcumin's effect on the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein.
The CFTR protein transports chloride in and out of cells. In people with CF, this function is severely impaired, likely causing the chain reaction of infection and inflammation in CF lungs that eventually leads to respiratory failure. Scientists believe that if CFTR function is restored, CF cells will begin to function normally and the course of the disease should slow dramatically or even come to a halt.
In the study, done jointly at Yale University and the Hospital for Sick Children in Toronto, and led by Michael J. Caplan, M.D., Ph.D., Marie Egan, M.D., and Gergely Lukacs, M.D., Ph.D., curcumin's effects were analyzed in CF mice homozygous for the DeltaF508 mutation of the CF gene. Nearly 90 percent of people with CF carry at least one copy of DeltaF508 CFTR, while 50 percent of people with CF are homozygous for DeltaF508. This study showed that curcumin corrects the DeltaF508 CFTR physiological defect in mice. Clinical benefits also were demonstrated, with CF mice treated with curcumin having a better survival rate than those not treated. It should be noted that CF mice do not have lung disease as people with CF do; instead they have only the intestinal complications of the disease.
"Through our work with these investigators and with SEER over the past year and a half, we have learned that a curcuminoid therapy could have potential in treating CF. Therefore, we feel that it is in the best interests of our patients to move curcumin forward as safely and as quickly as possible," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation and CFFT. "We ask for the patience and support of people with CF and their families while this trial is underway and caution them against taking curcumin on their own until safety trials are completed."