Gene Therapy Shows Early Promise for Heart Failure

Injecting a protein known to recruit stem cells may help repair damaged heart muscle, researcher says

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But this new approach, Friedman said, would avoid the steps of harvesting, culturing and reintroducing stem cells into patients. And, according to Penn, it should also be less costly.

For now, though, Goldberg said, "there are many, many unanswered questions. How durable will these effects be? Are there any unintended side effects in the long term?"

It's also not certain that the gene therapy did, in fact, recruit patients' stem cells to their damaged hearts. The researchers could not measure that directly.

Even with all the uncertainties, Goldberg said it's exciting to see that this approach could be feasible. "I am cautiously optimistic," he said.

One thing everyone agreed on was that the ultimate hope is to develop heart failure therapies that somehow rehabilitate damaged heart muscle. Right now, heart failure treatment involves drugs and lifestyle changes that relieve symptoms and ease the workload on the heart. But the disease does ultimately progress, until patients eventually need implanted devices or, as a last resort, a heart transplant.

Penn is the founder of Juventas Therapeutics Inc., which is developing SDF-1-based therapies for heart failure and other diseases.

More information

Learn more about heart failure from the U.S. National Heart, Lung, and Blood Institute.

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