Cedars-Sinai receives $7.3 million grant to test safety of novel cell-based therapy in treating PAH

Researchers from the Cedars-Sinai Heart Institute and the Cedars-Sinai Department of Medicine are expanding their ongoing evaluation of a novel cell-based therapeutic candidate into the area of pulmonary arterial hypertension (PAH). This work will be supported by a recently-awarded $7.3 million grant from the California Institute for Regenerative Medicine.

Pulmonary arterial hypertension is a chronic disease that is very different from, and much more dangerous than, regular hypertension. Caused by high blood pressure in the large arteries leading from the heart to the lungs, the condition affects 200,000 patients in the U.S. every year and can lead to heart failure and premature death. Symptoms include shortness of breath, dizziness and chest pain.

The new clinical study will test the safety and effectiveness of treating PAH with cells known as cardiosphere-derived cells (CDCs). CDCs consist of a single type of cardiac progenitor cell, and are currently being studied in clinical trials at Cedars-Sinai for other types of heart disease and for Duchenne muscular dystrophy. The CDCs to be used in the upcoming study are manufactured by Capricor Therapeutics, Inc. (NASDAQ: CAPR), which is developing this technology as its therapeutic product candidate CAP-1002.

"We have an exciting opportunity to try something new in these patients who currently have limited treatment options," said Eduardo Marbán, MD, PhD, director of the Cedars-Sinai Heart Institute and the researcher who invented and developed the CDC technology. "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 clinical study in PAH will be led by Michael I. Lewis, MD, director of Respiratory Therapy at Cedars-Sinai, and could begin enrolling patients as early as mid-2017.

In 2009, a team led by Marbán completed the world's first clinical trial of CDCs. The results, which were published in The Lancet in 2012, showed a medical first: evidence that healthy heart muscle could be therapeutically regenerated in a heart damaged by a heart attack. Since then, Marbán's research has led to several clinical trials in which heart disease patients undergo a catheter-based procedure during which they receive an infusion of millions of CDCs.

"The primary goal of our study in pulmonary arterial hypertension is to verify safety," Lewis said. "However, 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."

"This award is a reflection of the continued excellence of our heart institute in leading the field and maintaining Cedars-Sinai as a pioneer in successfully and safely bringing cellular therapies to treat our patients with serious cardiovascular disorders," said Shlomo Melmed, MD, Cedars-Sinai executive vice president for Academic Affairs, dean of the medical faculty and the Helene A. and Philip E. Hixon Distinguished Chair in Investigative Medicine.

It is the second time in 2016 that Marbán's research resulted in a major grant to fund a clinical trial for patients with an incurable condition. Earlier this year, the Department of Defense awarded Cedars-Sinai a $10 million grant to fund a cell therapy trial for patients diagnosed with a common but difficult-to-treat form of heart failure called heart failure with preserved ejection fraction.

The process to grow CDCs was developed by Marbán when he was on the faculty of Johns Hopkins University and further developed at Cedars-Sinai. Capricor has licensed the process from Johns Hopkins and from Cedars-Sinai for clinical and commercial development. Capricor has licensed additional intellectual property from Cedars-Sinai and the University of Rome. Cedars-Sinai and Marbán have financial interests in Capricor.