Gene Therapy may Treat Heart Failure

Novel promising gene therapy to treat heart failure is moving closer to the clinic.

Novel promising gene therapy to treat heart failure is moving closer to the clinic, as per a study at the Baylor College of Medicine, the Texas Heart Institute, and collaborating institutions, published in the journal Science Translational Medicine.

Heart failure is a condition where there is an inability of the heart muscle to pump sufficient blood to the body. It is the leading cause of mortality in the western world, more than all cancers combined.

Congestive Heart Failure (Congestive Cardiac Failure) Current Management
Congestive heart failure (CHF) is a condition in which the heart fails to work adequately as a pump that can deliver oxygen rich blood to the body.

‘Novel promising gene therapy helps in treating heart failure in pig models. This holds a potentially transformational strategy to treat human heart failure using gene therapy through further human clinical trials. ’

A heart transplant is the best current treatment for heart failure. However, the limited number of hearts available for transplant mandates the need for alternative treatments.

"One of the interests of my lab is to develop ways to heal heart muscle by studying pathways involved in heart development and regeneration," says corresponding author Dr. James Martin, professor, and Vivian L. Smith Chair in Regenerative Medicine at Baylor and director of the Cardiomyocyte Renewal Lab at the Texas Heart Institute.

Gene Therapy in Heart Failure

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Earlier studies have shown that there is an increase in the activity of the Hippo signaling pathway (inhibits heart repair) during a heart failure. Turning this pathway off in the mouse model revealed recovery of the heart’s pumping function.

The study team created a pig model to assess the effect of gene therapy by knocking down the Hippo signaling pathway in a heart attack. Since the pig's heart is closely associated with the human heart, the study may yield promising results for gene therapy in treating human heart failure.

Fat Cells Linked to Heart Failure Outcomes
Research in mice suggests that targeting fat cells could help in treating patients with heart failure.

"We were excited by the results. Three months after we treated the pigs, those that received the gene therapy had improved their heart function, showed signs that their cardiomyocytes (heart muscles) were regenerating, had less fibrosis or scarring and had evidence that new blood vessels had formed. The procedure was shown to be safe since the pigs tolerated the therapy very well," says first author Dr. Shijie Liu, a postdoctoral associate in the Martin lab.

Thus these findings hold a potentially transformational strategy to treat human heart failure using gene therapy. The team anticipates next bring the therapy to human clinical trials for further benefits.

Increased Cancer Risk in Heart Failure Patients
Heart failure patients are more likely to develop cancer compared to patients without heart failure condition.

Source-Medindia