Researchers begin clinical trials of new, inhaled gene therapy to treat pulmonary hypertension

By Liz Meszaros, MDLinx
Published July 14, 2017

Key Takeaways

If clinical trials in human subjects succeed, a new, investigational airway-delivered gene therapy may provide the first means to reverse the damage caused by pulmonary hypertension (PH), according to researchers from Mount Sinai, New York City, NY, who have collaborated with Theragene Pharmaceuticals Inc.

Although rare (15-50 cases per million), PH carries a 5-year mortality rate of approximately 50%, and primarily affects young adults (58% of cases) and women (72%). Currently, there is no effective cure available.

“This is a devastating disease, and our work in collaboration with many laboratories across the country has allowed us to identify a specific molecular target and use gene therapy to improve cardiovascular and lung parameters in experimental models of PH. We look forward to starting first in-human studies using this approach in affected patients,” said senior author of these studies, Roger J. Hajjar, MD, professor of medicine and director, Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York City, NY.

This investigational gene therapy technique is based on Dr. Hajjar’s research, and has currently been demonstrated to be effective in preclinical rodent and pig models.

Delivered via inhaled aerosolized spray, this new gene therapy technique works to increase the expression of sarcoplasmic reticulum calcium ATPase pump (SERCA2a) protein. SERCA2a protein serves to regulate intracellular calcium within the vascular cells to prevent proliferation within the vessel wall. Previous research has shown calcium cycling abnormalities to play a vital role in the pathophysiology of PH, as do deficiencies in the SERCA2a protein.

Dr. Hajjar added that the clinical trials in humans will commence in the next 2 years, and commercial availability of this product may take several years.

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