With the recent announcement from Australia’s version of the FDA, the Therapeutic Goods Administration, that pluripotent stem cell manufacturing innovator International Stem Cell Corp. (OTCQB: ISCO) has been cleared to start Phase I/IIa dose escalation clinical trials focused on the safety and efficacy of its human parthenogenetic neural stem cells (ISC-hpNSCs), the company has taken another major step toward a real therapeutic solution to Parkinson’s disease. An incurable condition that primarily affects the planet’s growing elderly population, Parkinson’s currently afflicts well over 10 million people worldwide and represents a therapeutics market somewhere in the neighborhood of $2.6 billion.
However, while the sale of extant therapeutics will continue to be dominated by mere dopamine agonists and an increasing use of MAO-B inhibitors (historically used to treat depression), such treatments are palliative at best, and sales will be substantially impacted by the rapid proliferation of generics. Even the newer agents coming down the industry’s pipeline through to 2020, such as a reformulated levodopa from Impax Laboratories (NASDAQ: IPXL) and GlaxoSmithKline (NYSE: GSK), or the MAO-B inhibitor safinamide being developed by Merck (NYSE: MRK), Newron (OTC: NWPHF) and EMD Serono, will be forced to compete with generics.
ISCO, on the other hand, has the inside track in this market with an injectable cellular therapy that is potentially capable of actually replacing dead and dying neurons in the midbrain, while directly offsetting Parkinson’s symptoms. This solution also offers substantial protection to surviving neurons, shielding them from further deterioration. Considerable pre-clinical animal model testing has already shown extremely promising results and the progress ISCO is set to make via the Phase I/IIa clinical trial in humans could propel the company to stardom as the first to develop an actual solution for Parkinson’s sufferers.
This same technology, because it employs high purity functional adult human cells that have been created from unfertilized donor eggs at the company’s state-of-the-art Oceanside, California, facility using an ethical, patented chemical differentiation process, could also evolve into frontline treatments for other CNS (central nervous system) maladies such as Alzheimer’s and stroke. The FDA’s recent IND approval of Stemedica’s allogeneic (same species but genetically dissimilar and generally immunologically incompatible) stem-cell therapy for a Phase IIa clinical study in Alzheimer’s at UC San Diego shows how much potential there is for this kind of technology, and how receptive the FDA has become to stem cell technology.
ISCO’s ability to manufacture commercial-scale batches of both precisely human leukocyte antigen-matched (HLA) and therefore histocompatible human parthenogenetic stem cell lines, as well as HLA-homozygous lines that are suitable for significant segments of the overall population, gives the company a real edge here as well. Stemedica’s allogeneic stem cells, for instance and by contrast, are cultivated from donor tissues, not differentiated from unfertilized eggs. Thus, ISCO’s technology is quite remarkable, because it substantially overcomes one of the main challenges facing stem cell therapeutics as a viable solution to numerous diseases; namely, cell rejection by the patient’s immune system.
International Stem Cell Corporation could be first to market a treatment actually capable of halting the progression of Parkinson’s disease in its tracks, or even reversing the impact of the disease. Results from the company’s landmark human clinical trial should start to become available in the coming months and investors should keep a close eye on ISCO for breaking news.
For more information, visit www.internationalstemcell.com
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