GAINESVILLE — A proof-of-concept clinical study led by University of Florida scientist Joe G.N. Garcia found that the monoclonal antibody ALT-100 improved ventilator-free days and reduced organ failure scores in patients with acute respiratory distress syndrome (ARDS). The findings were published in the American Journal of Respiratory and Critical Care Medicine.

The trial enrolled 15 patients at academic medical centers in six U.S. cities, including UF Health Shands Hospital in Gainesville. Participants diagnosed with ARDS were randomly assigned to receive either ALT-100, a monoclonal antibody targeting extracellular NAMPT, or a saline solution placebo.

Over a 28-day period, the group receiving ALT-100 experienced an average of 21 ventilator-free days, compared to 14 ventilator-free days for the placebo group. The ALT-100 group also showed reduced inflammatory markers and lower organ failure scores. The treatment's safety profile was comparable to that of the placebo group.

Garcia stated, "Particularly the ARDS data showing that the monoclonal antibody improved both ventilator-free days and organ failure scores, because organ failure is the main cause of ARDS-related mortality." He also said, "Even though we were only able to enroll 15 patients, the data we are getting is simply incredible."

ARDS occurs when infection or injury triggers an inflammatory cascade, leading to fluid leaking into the lungs. Nearly 40% of individuals affected by ARDS die. An estimated 500,000 people are diagnosed with ARDS annually in the United States, and 2 million people are affected globally. Garcia's lab discovered NAMPT as a factor in the inflammatory response. In patients with mutations that impair NAMPT breakdown, unremitting inflammation can result, which may lead to organ failure and death.

"There are no FDA-approved therapies to give these patients, and given the unacceptable mortality and pervasiveness of the disease, ARDS treatment remains one of the greatest unmet needs in medicine," Garcia said.