Stem cell technology’s real untilled and still extremely fertile soil is the therapeutics market, where injected cells could help repair damaged tissue systems and thus effectively treat many conditions, including degenerative ones, for which the current standard of care is often palliative at best. Dealing with the underlying cause of a given condition, through the gradual replacement and/or reinforcement of an impaired tissue system or organ using injected stem cells, represents a true paradigm shift in how we think about treating many of today’s most debilitating diseases. However, to tap into the rich spoils of this as yet largely undiscovered country, requires a mix of technologies and logistical capabilities, of which tragically few companies today can seriously boast.
One company that doesn’t need to boast, as it has clearly achieved both the capacity to proprietarily differentiate adult human cells by chemically stimulating unfertilized donor eggs, as well as provide long-term, storable, high-quality stem cell product for real-world, commercial-scale therapeutic applications, is International Stem Cell Corp. At the core of the company’s pipeline of rapidly emerging therapeutics is its parthenogenesis technology, whereby the company can create large batches of pluripotent (able to develop into any type of cell), human parthenogenetic stem cells (hpSCs), and do so in a completely ethical fashion. The company’s proprietary stimulation and differentiation technology also helps resolve the one major stumbling block facing stem cell therapies, immune rejection by the host. The company’s ability to make large batches of adult human cells from stem cells that are either specifically immune-matched to the donor, or immune-matched to the general population, gives ISCO an open road to therapy development, and the company has the testing to back up its technology too.
ISCO’s nine-month, 300 subject rodent model safety study, testing the tumorigenicity (propensity to promote tumor formation/growth) of its human neural stem cells (hpNSCs), which have been developed for the treatment of Parkinson’s disease (PD), was submitted back in July as part of the build up to phase 1/2a human clinical trials in Australia. This study followed up on earlier results by ISCO with its hpNSCs, showing no tumors in any of the animals receiving neural stem cell transplants. The demonstrated ability of injected hpNSCs to create new neurons that produce the primary neurotransmitter, dopamine (with whose falloff PD is causally associated), as well as exhibit a neuroprotectant effect on surviving neurons makes this technology a potential gold mine if human clinical trials yield good results.
Given that PD is a progressive, degenerative disorder, and that the current standard of care, oral levodopa (L-dopa), is associated with progressively worse periods where symptoms manifest themselves fully, called “off” periods, there hasn’t really been a good long-term solution for addressing the unmet needs of a majority of PD patients. This majority of PD patients, for whom the current standard of care so often feels like they are just postponing the inevitable succumbing to the disease’s impairments, are plagued by the frequency and intensity of relapse periods increasing as the disease progresses using L-dopa. ISCO isn’t trying to mask the problem chemically with synthetic dopamine, the company has developed injectable replacement neurons to treat the underlying brain tissue degeneration, and hopefully actually restore full functionality to the patient.
According to a new study out by research and consulting firm, GlobalData, the PD treatment market for just the U.S., Brazil, Japan, and the major EU member states is on track to hit upwards of $4.7 billion by 2022. However, newly FDA-approved market entrants, such as Xadago (safinamide) – a drug which blocks the primary enzyme which break down dopamine, from Zambon and its SIX Swiss Exchange-traded partner Newron Pharmaceuticals (SWX: NWRN) – or Impax Laboratories’ (NASDAQ: IPXL) RYTARY, an extended-release oral capsule formulation of carbidopa-levodopa – still do not address the underlying causes of the disease. It is up to a company like ISCO to bring forth a real therapeutic treatment option here, with ethical stem cell technology that can actually regenerate the damaged tissue system(s). With PD, given its progressive nature, it is not unthinkable that periodic injections, or simply a thorough course of therapeutic treatments with hpNSCs, could restore the patient to full functionality.
A good indicator of this potential is ISCO’s parallel hpNSC therapy pipeline for stroke, where preclinical data indicates that injections administered even several weeks after the stroke could actually reverse the typically associated functional deficits completely. The alternative to such a therapy in the case of a stroke is often grueling physical therapy, required in order to get back or re-learn lost motor function. But with ISCO’s treatment option on the table, stroke could be handled in the future with a simple injection regimen, even well after the initial stroke hospitalization. The tremendous potential of hpNSC therapy in strokes speaks volumes about what that same therapy can do for PD patients, given that both are maladies of the CNS, and that the therapy functions by replacing damaged neurological tissue.
To dig deeper, visit www.internationalstemcell.com
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