Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial -

Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial
Information type: Randomised controlled trials
Source: Lancet Public Health
Specialities: Cardiovascular system disorders
Summary
CUPID 2, the largest gene transfer study in patients with heart failure (HF) to date (n=250), found that despite promising results from previous studies, AAV1/SERCA2a at dose tested did not improve clinical course (vs. placebo) of patients with HF and reduced ejection fraction.
UKMi comment
A commentary notes the researchers had reported that inadequate gene delivery was a potential cause of the negative trial. It discusses why, after 20 years, this issue still exists, and notes that insufficient investigation of basic gene delivery approaches and underlying variables, together with non-existent funding for methods development, are substantial contributors to this issue.
Related links: Comment