With the recent approval of multiple curative CAR-T gene therapy treatments for cancers, trials have expanded to test this treatment approach in patients with other life-threatening conditions, including heart failure.
Heart failure impacts 5.7 million adults in the United States, with half of patients dying within 5 years of heart failure development. A gene therapy treatment for heart failure with reduced ejection fraction would radically change the standard of care for these patients.
Currently, phase 3 trials are under way to determine the efficacy of RT-100 (Ad5.hAC6), an investigational gene therapy that repairs the arteries that feed the heart. The treatment is a one-time administration that works by adenovirus vector encoding. The gene transfer is completed via infusion directly into the arteries.
Phase 2 studies of 56 adults with symptomatic heart failure with ejection fraction less than 40% were recently completed. These patients received a one-time intracoronary administration of RT-100 or a placebo.
“The point of this trial is [to see] if through a heart catheterization they [can] release a medicine through an adenovirus to see if patients who have ejection fractions less than 35% but greater than 10% will have improvement of their ejection fraction and heart failure symptoms,” Maria Mountis, DO, of the department of cardiovascular medicine at The Cleveland Clinic told MD Alert in a video interview.
CAR-T Therapy on the Horizon For Heart Failure. A video interview with Dr Mountis.
Results of the Phase 2 study showed that 21 patients had increased ejection fraction at 4 weeks, but not at 12 weeks. Patients in the placebo group had no change in ejection fraction at either 4 or 12 weeks. Further. heart failure symptoms were reduced at 12 weeks in patients who received the CAR-T treatment. There were no changes in symptoms in the placebo group.
Additionally, the hospital admission rate was significantly lower in the intervention group compared to the placebo group, at 9.5% vs 28.6%, respectively.
As a result of these trials, Phase 3 trials are currently underway with 536 patients receiving randomized treatment. Primary data for this clinical trial will be released in summer of 2020. In the meantime, the FDA has granted RT-100 with a Fast Track designation.
“The goal is to avoid hospitalization within 1 year,” Dr Mountis explained. “That would be huge. If that [kind of treatment] is going to be available that would be fantastic to have one more medication or device like this to be able to help improve hospitalization in heart failure patients.”
Watch the video above for more from Dr Mountis on how CAR-T could change heart failure treatment if approved.