Benzinga | Sep 15, 2021 04:08PM EDT

Heart Hope: Announcing the Encouraging Results of a Cell-Therapy Clinical Study by Longeveron

Photo by jesse orrico on Unsplash

Researchers in the medical field are constantly working to create medicines to prevent and cure diseases. The development of the multiple COVID-19 vaccines from Pfizer (NYSE:PFE), Johnson & Johnson (NYSE:JNJ) and Moderna (NASDAQ:MRN) only highlights the scientific community's commitment and possible advancements. And although the coronavirus is at the forefront of the world's attention, that doesn't mean other breakthroughs aren't happening all the time.

One example of this effort was recently reported by Longeveron, Inc. (NASDAQ:LGVN) which just concluded and reported results from a Phase 1 trial of a regenerative medicine approach for a life-threatening congenital condition called hypoplastic left heart syndrome (HLHS).

What is Hypoplastic Left Heart Syndrome?

HLHS is a rare heart defect that develops during a fetus's growth process in the womb. Several factors can contribute to the classification of this heart issue, like an underdeveloped left ventricle, a mitral valve or aortic valve that's too small, or a hole between the upper right and left chambers of the heart. No matter the exact defect in the left side of the heart, the result is a problem with proper blood flow.

The job of the left half of the human heart is to pump blood to the rest of the body, so when the organ has damage, the circulatory system can't function. Without intervention, the outcome is death.

But Longeveron, a clinical-stage biotechnology company, created a therapy to address HLHS and completed a Phase 1 trial to test its safety and explore effectiveness in this rare pediatric condition.

Phase 1 Clinical Study Results

The primary purpose of the first trial of Lomecel-B was to test its safety and tolerability of direct injection into the heart in infants with HLHS during surgery. Lomecel-B is a living cell product that is made from medicinal signaling cells (MSCs) isolated from the bone marrow of young, healthy adult donors. The first safety endpoint showed that the infants tolerated the Lomecel-B well with no major adverse cardiac events and no reported treatment-related infections.

The secondary objectives of the trial were to explore the drug's effect on clinical outcomes and the heart's function. Although there was no control arm for comparison, the results of the study showed:

* All infants were alive and heart transplant free up to 3.5 years after injection

* Infants showed normal development and growth during the 1-year follow-up

* Heart function and structure did not significantly deteriorate

The company is continuing its research to determine if this drug could improve cardiac function for people suffering from HLHS.

The Next Step

After the encouraging results from its Phase 1 trial, Lomecel-B is now being tested in a Phase 2 trial. This blinded and controlled study is projected to enroll 38 infants with HLHS from 7 children's hospitals across the United States.

Depending on the Phase 2 results, the company could potentially move into Phase 3, which is the final phase required for FDA approval.

With principal Investigator Sunjay Kaushal, M.D., Ph.D. and the Ann and Robert H. Lurie of the Children's Hospital of Chicago at the helm, and the second phase backed by a grant from the NIH's NHLBI, Longeveron is one step closer to helping bring hope to afflicted babies and their families.