This is a step closer to a cure in humans.
Experimental gene therapy has shown promising results in reversing heart failure in laboratory pigs, suggesting the potential for significant advancements in treating human heart disease. The therapy not only prevented the worsening of heart failure in four pigs, but also helped their hearts recover and grow stronger, improving heart function. Researchers observed that despite ongoing stress on the heart, the treated pigs experienced a “reverse remodeling,” meaning their hearts became more stable and regained a more normal structure.
The gene therapy targets cardiac bridging integrator 1 (cBIN1), a crucial protein in heart muscle cells. Previous studies have linked lower levels of cBIN1 with severe heart problems, and the therapy aimed to restore these levels to improve heart function. By delivering an extra copy of the cBIN1 gene using a modified virus, the researchers successfully introduced the gene into the pigs’ heart cells. Pigs are often used in medical research due to their organs’ similarities to human organs, making the results particularly relevant.
Typically, animals with the level of heart failure present in these pigs would not survive for long, but all four pigs in the study lived for the full six months. Their heart function showed significant improvement, with pumping ability increasing by 30%, a remarkable gain compared to the 5%-10% improvement seen with current heart failure treatments. Additionally, the treated pigs’ hearts started resembling those of healthy animals, a reversal of the damage usually associated with heart failure.
The gene therapy also had an impact at the cellular level, helping heart cells and proteins organize better. This breakthrough in heart failure research is considered unprecedented, with the improvements in heart function far exceeding previous treatments. The research team, in collaboration with TikkunLev Therapeutics, plans to move forward with preparations for human clinical trials, aiming for FDA approval by the fall of 2025. The success of this gene therapy offers hope for curing heart failure, a condition that affects millions of people worldwide.
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