CUPID: First-in-man gene therapy for advanced heart failure promising in small study

From The

May 31, 2010 | Steve Stiles

Berlin, Germany – In the first clinical test of a gene therapy for heart failure, administration of a gene that upregulates an enzyme involved in myocardial contraction and relaxation appeared to improve symptoms, functional status, and ventricular volumes in patients with severe systolic heart failure. The small dose-finding study, reported here at the Heart Failure Congress 2010, sponsored by the Heart Failure Association of the European Society of Cardiology, is something of milestone and, its researchers say, paves the way for studying the novel treatment in larger clinical-outcomes trials.

But an unconventional broad-based primary end point in the randomized, placebo-controlled study, called Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID), along with seemingly inconsistent effects at different dosage levels, left some observers scratching their heads about what the trial actually shows.

Continue reading