A proof-of-concept study showed an experimental monoclonal antibody targeting ARDS inflammation increased ventilator-free days and reduced organ failure.
RT’s Three Key Takeaways:
- Increased Ventilator-Free Days: Patients treated with the ALT-100 monoclonal antibody averaged 21 ventilator-free days compared to 14 days for the placebo group.
- Targeted Inflammatory Regulation: The therapy targets NAMPT, a regulator of inflammation, to prevent the “cytokine storm” that causes lung fluid leakage and organ failure.
- Comparable Safety Profile: Early trial data showed that the safety of the experimental treatment was similar to a placebo, while also reducing inflammatory markers in participants.
An experimental monoclonal antibody treatment (ALT-100) may improve outcomes for patients with acute respiratory distress syndrome (ARDS), a condition with a nearly 40% mortality rate and no current USFDA approved therapies, according to a proof-of-concept study, published in the American Journal of Respiratory and Critical Care Medicine.,
The study from University of Florida (UF) researchers evaluated a treatment called ALT-100. In an early phase clinical trial involving 15 patients across six US cities, including UF Health Shands Hospital, participants were randomly assigned to receive either the antibody or a saline placebo.
Over 28 days, the group receiving ALT-100 experienced an average of 21 ventilator-free days, compared to 14 days in the placebo group. The treatment also led to lower organ failure scores and reduced inflammatory markers.
“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,” said Joe GN Garcia, MD, the study’s first author and associate vice president for research at UF Health, in a news release.
ARDS occurs when an inflammatory cascade triggered by infection or injury causes fluid to leak into the lungs, significantly lowering blood oxygen levels. Garcia developed ALT-100 to target NAMPT, a protein that regulates the inflammatory response. In patients with mutations that impair the breakdown of NAMPT, unremitting inflammation known as a “cytokine storm” can develop, leading to organ failure and death.
Unlike similar medications that dampen the body’s ability to fight infection, ALT-100 is designed to limit excessive immune responses.
“This is a Humira-like drug, targeting the innate immune response, which is why it has profound implications for diseases like cancer, organ fibrosis and so on,” said Garcia, who also serves as the director of the center for inflammation science and systems medicine at the Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology, in a news release.
While the study was limited by its small sample size of 15 participants, researchers noted that the data showed the monoclonal antibody improved both ventilator-free days and organ failure scores.
“Even though we were only able to enroll 15 patients, the data we are getting is simply incredible,” said Garcia, founder and CEO of Aqualung Therapeutics, Corp., in a news release. “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.”
Looking ahead, the research team hopes to obtain funding for a larger clinical trial. In addition to ARDS, the FDA has authorized the study of ALT-100 in patients with progressive pulmonary fibrosis, a common complication for ARDS survivors involving the buildup of scar tissue in the lungs.
