Gene therapy shows promise for reversing heart failure

Good news!

"... The new gene therapy ... prevented heart failure (HF) from worsening and even improved some key measures of heart function in pig models. ..."

"A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model, according to new research ... The therapy increases the amount of blood the heart can pump and dramatically improves survival, in what the study calls “an unprecedented recovery of cardiac function.”

Currently, heart failure is irreversible. In the absence of a heart transplant, most medical treatments aim to reduce the stress on the heart and slow the progression of the often-deadly disease. But if the gene therapy shows similar results in future clinical trials, it could help heal the hearts of the one in four people alive today who will eventually develop heart failure. ..."

From the abstract:

"Heart failure (HF) is a major cause of mortality and morbidity worldwide, yet with limited therapeutic options. Cardiac bridging integrator 1 (cBIN1), a cardiomyocyte transverse-tubule (t-tubule) scaffolding protein which organizes the calcium handling machinery, is transcriptionally reduced in HF and can be recovered for functional rescue in mice.

Here we report that in human patients with HF with reduced ejection fraction (HFrEF), left ventricular cBIN1 levels linearly correlate with organ-level ventricular remodeling such as diastolic diameter. Using a minipig model of right ventricular tachypacing-induced non-ischemic dilated cardiomyopathy and chronic HFrEF, we identified that a single intravenous low dose (6 × 1011 vg/kg) of adeno associated virus 9 (AAV9)-packaged cBIN1 improves ventricular remodeling and performance, reduces pulmonary and systemic fluid retention, and increases survival in HFrEF minipigs. In cardiomyocytes, AAV9-cBIN1 restores t-tubule organization and ultrastructure in failing cardiomyocytes. In conclusion, AAV9-based cBIN1 gene therapy rescues non-ischemic HFrEF with reduced mortality in minipigs."

Gene therapy shows promise for reversing heart failure

New gene therapy reverses heart failure in large animal model (original news release)

Cardiac bridging integrator 1 gene therapy rescues chronic non-ischemic heart failure in minipigs (open access)

Fig. 2: AAV9 transduced cBIN1 in myocardium rescues survival rates in minipigs with RVP-induced non-ischemic HFrEF.