$7.3M Grant to Support Clinical Trial of Stem Cell Therapy in PAH Patients

Researchers at Cedars-Sinai Medical Center received a $7.3 million grant from the California Institute for Regenerative Medicine to pursue the development of a new stem cell therapy for pulmonary arterial hypertension (PAH).

Working with Capricor Therapeutics, the grant will support a clinical trial to test infusions of a heart progenitor cell type called cardiosphere-derived cells (CDCs). Patient enrollment in the planned study could begin as soon as mid-2017.

“We have an exciting opportunity to try something new in these patients who currently have limited treatment options,” Eduardo Marbán, MD, PhD, director of the Cedars-Sinai Heart Institute and the researcher who invented and developed the CDC technology, said in a news release.

“Our theory is that introducing these cells into the arteries leading to the lungs will reduce inflammation and, as a result, prevent permanent damage to the heart by decreasing pulmonary blood pressure.”

The therapy was previously tested in patients with other types of heart disease, and results from the first completed clinical trial were published in the journal The Lancet in 2012, titled “Intracoronary cardiosphere-derived cells for heart regeneration after myocardial infarction (CADUCEUS): a prospective, randomised phase 1 trial.” The trial demonstrated that the cells could regenerate damaged heart muscle in people who had had a heart attack.

Since then, the method has been tested in patients with other types of heart problems, and in people with Duchenne muscular dystrophy — a degenerative muscle disease in which the heart muscle also gets worn down.

Marbán developed the technique while at Johns Hopkins University, and further developed it at Cedars-Sinai. Capricor Therapeutics licensed the procedure from Johns Hopkins and Cedars-Sinai to continue clinical and commercial development.

The PAH trial will first assess the safety of the procedure in patients.

Michael I. Lewis, MD, director of Respiratory Therapy at Cedars-Sinai, who will lead the clinical trial, said “we did see significant improvement in laboratory animal tests that we hope will lead us to innovative and effective treatments for a group of patients who currently face an uphill battle.”