Our lead investigational therapeutic candidate is a cellular therapy called Lomecel-B™. Lomecel-B is being evaluated in multiple clinical trials for aging-related chronic diseases and other life-threatening conditions under US FDA-approved Investigational New Drug (IND) Applications.
What is the source for Lomecel-B? 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.
How is Lomecel-B made? Once the MSCs have been isolated from the fresh bone marrow through a careful selection process, the cells are culture-expanded (allowed to replicate under controlled laboratory conditions) into the billions using specialized techniques and processes in our GMP facility in Miami, Florida. After a specific number of expansion cycles called “passages”, the cells are harvested, separated into specific doses (e.g. 50 million cells), and frozen until future use.
Can Lomecel-B be administered to a patient without causing an immune response (tissue rejection)? Yes. These cells have special characteristics that allows them to be transplanted from a donor to host without triggering a harmful immune response in the recipient. The cells can be administered on an out-patient basis in as little as 40 minutes after thawing. These are advantages over autologous cell therapy interventions, which involve removing cells from an individual through an operative procedure (e.g. liposuction), and then reintroducing the cells back into the same person, sometimes after weeks or months of culture expansion. Accordingly, autologous approaches lack economies of scale since they serve only a single patient whereas tissue from a single bone marrow donor can be used to make many doses of Lomecel-B for use in multiple patients.
Because of these characteristics, Lomecel-B is considered an “off-the-shelf” product. In some of our trials such as Alzheimer’s Disease and Aging Frailty, Lomecel-B is administered via peripheral intravenous infusion, while in our Hypoplastic Left Heart Syndrome trial, Lomecel-B is administered via direct injection into the heart tissue.
How does Lomecel-B work? It is becoming increasingly clear that the therapeutic effects of allogeneic MSCs occurs through secretion of bioactive molecules and direct cell-to-cell contact at the site of inflamed and damaged tissue. There are several potential mechanisms of action believed to mediate therapeutic benefits:
Release of growth factors and other proteins, such as anti-inflammatory cytokines. These have the potential to reduce inflammation, and stimulate nearby stem cells and other cells (called paracrine activity) to promote regenerative and repair responses.
Engage in direct cell-cell interactions to induce positive pathways in contacted cells
Release exosomes, which have cargo consisting of RNA, proteins, and other molecules that can be taken up by other cells to provide beneficial effects.
Potential to form nanotube bridges, which can allow the exchange of mitochondria and other cellular contents between cells.