The Players of Regenerative Medicine — Neuron, the Star Quarterback

San Diego Regenerative Medicine Institute and Xcelthera announce the publication of ground-breaking technique on direct conversion of human embryonic stem cells into neuronal cells. Realizing the developmental and therapeutic potential of human embryonic stem cells has been hindered by the inefficiency and instability of generating desired cell types from pluripotent cells through multi-lineage differentiation. The original research, titled “Efficient Derivation of Human Neuronal Progenitors and Neurons from Pluripotent Human Embryonic Stem Cells with Small Molecule Induction” funded by the National Institutes of Health, is the first to show that pluripotent human embryonic stem cells can be uniformly converted into a neuronal lineage by simple provision of small molecules. This technology breakthrough not only provides a large supply of clinical-grade human neuronal therapeutic products for neuron regeneration and replacement therapy against a wide range of neurological disorders, but also offers means for small-molecule-mediated direct control and modulation of the pluripotent fate of human embryonic stem cells when deriving an unlimited supply of clinically-relevant lineages for regenerative medicine. The protocol of this novel technology developed from human embryonic stem cell research was published in JoVE (http://www.jove.com ), the World’s first peer-reviewed scientific video Journal through open access link.

To date, lacking of a clinically-suitable neuronal cell source, of course, has to be from human, has been the major setback in developing cell-based therapies for restoring the damaged or lost nerve in a wide range of neurological disorders, such as Alzheimer’s disease, Parkinson’s disease, Lou Gehrig’s disease, and spinal cord injury. Like our star quarterback, neurons are the key player of the central nervous system and hard to find the replacement. Despite of initial excitement of early 90th to discover that cells in our brain are still growing, we quickly learned that they could not grow into the damaged or lost neurons we need. Against ethics, restriction, immune-rejection, and all odds on using human fetal tissues, transplantation using tissue-derived human neural stem/progenitor cells became unavoidable if a cure was desired. After almost 20 years of battle, the controversy on using human fetal tissue for cell therapy has faded away. However, 20 years of studies have also outdated a field once fought so hard. It has become known that those tissue-derived cells can only regenerate a supporting team to slow down the disease, but not the neurons to provide a cure.
The derivation of human embryonic stem cells proffers cures for a wide range of neurological disorders by supplying the diversity of human neuronal cell types for repair. Therefore, they have been regarded as an ideal source to provide an unlimited supply of human neuronal cells for repair. However, conventional approaches rely on multi-lineage inclination of pluripotent cells through spontaneous germ layer differentiation, which is inefficient and uncontrollable. So far, the cell products generated from conventional multi-lineage differentiation of pluripotent cells remain supportive, but cannot regenerate the key functional player, neurons. It is understatement that our ground breaking study allows human embryonic stem cells fulfill the large need for a clinically-suitable human neuronal cell source for restoring the damaged or lost nerve tissue and function in today’s healthcare industry. The availability of human neuronal cells in high purity and large quantity with adequate neurogenic potential will accelerate the development of effective cell-based therapy against a wide range of neurological disorders, one step closer to a cure.