In March 2021, the FDA expanded access approval to biotechnology company Longeveron Inc.’s investigational Lomecel-B to a pediatric patient with hypoplastic left heart syndrome (HLHS). According to a press release on the matter, the treatment will be administered during surgery by Dr. Sunjay Kaushal, MD, PhD.

Lomecel-B

So what is Lomecel-B? According to the Longeveron website:

Lomecel-B is made from special living cells called Medicinal Signaling Cells, or MSCs that are isolated from fresh bone marrow tissue that has been donated by adult donors aged 18 to 45. Because the cells come from another individual, Lomecel-B is referred to as an “allogeneic” (donor-derived) product.

Because of the cells’ special culturing and formulation, Lomecel-B does not prompt a harmful or negative immune response. Longeveron believes that its product provides therapeutic benefit in 4 main ways: by forming nanotube bridges, releasing exosomes, promoting cell-to-cell interactions, and releasing proteins and growth factors like anti-inflammatory cytokines to promote cell regeneration.

In a past Phase 1 clinical trial, researchers injected Lomecel-B intramyocardially for patients with HLHS. These researchers determined that the treatment was relatively safe and well-tolerated. Moving forward, Longeveron hopes to enter into additional Phase 1 and Phase 2 clinical trials. Outside of HLHS, Longeveron is also considering Lomecel-B as a potential therapy for patients with Alzheimer’s disease and acute respiratory distress syndrome (ARDS).

In this most recent case, the therapy is being provided via the Expanded Access (or “compassionate use”) program. The program allows patients to be able to use or have access to investigational therapies when patients are seriously ill and no other satisfactory options are available outside of clinical trials. According to the FDA, patients qualify for Expanded Access if:

• The risk justifies the potential benefit/reward.
• A patient is unable to enroll in a clinical trial.
• Patient(s) have a serious and/or life-threatening condition or disease.
• There is no other comparable or satisfactory treatment, diagnosis, or monitoring option.
• Taking the therapy will not interrupt with or derail potential clinical trials.

Hypoplastic Left Heart Syndrome (HLHS)

Hypoplastic left heart syndrome (HLHS) is a severe congenital birth defect which impacts normal blood flow through the heart. It affects an estimated 1,000 American infants each year. Altogether, the cause of HLHS is unknown. However, doctors do know that it occurs when the baby’s heart is developing. As a result, the left side of the heart forms incorrectly. This may include the left ventricle or mitral valves being too small or narrow, or an underdeveloped aorta or aortic valve. Regardless, the heart is unable to pump enough oxygen-rich blood throughout the body.

HLHS is considered to be a critical congenital heart defect (CCHD), as babies need surgery or other health procedures immediately following birth. In the past, the prognosis for HLHS was very poor. Surgical interventions and other therapies have slightly improved the survival rate. Unfortunately, HLHS remains difficult and complex to deal with, with high mortality rates within the first few weeks to years (with treatment). Symptoms include:

• Lethargy
• Cold hands and feet
• Poor feeding
• Cyanosis (a gray/blue skin color)
• Respiratory distress
• Shock
• Seizures
• Weak pulse
• Cool, clammy skin