In the U.S. alone each year somewhere around 60k people are diagnosed with the crippling degenerative central nervous system (CNS) disorder known as Parkinson’s disease, which is brought on by an as-yet poorly understood cascade death of dopamine-generating cells in the midbrain. Roughly 0.3% of the entire population, or nearly one million Americans, currently lives with this life-shattering disorder. That’s more than multiple sclerosis, muscular dystrophy, and Lou Gehrig’s disease combined.
Parkinson’s is the kind of CNS disorder that can destroy an entire family, devastating a family member and rendering them unable to effectively care for themselves, as the disorder is most commonly characterized by severe diminishment of motor control, uncontrollable limb rigidity and shaking, as well as considerable difficulty just walking or moving around. Later on in the progression of Parkinson’s, sensory and emotional problems arise, with sleep deprivation and depression being all too common. Eventually, the patient often develops thoroughly debilitating dementia as well and Parkinson’s has become a dreaded thief that can rob a family of their loved ones, and for which there are currently only extremely limited/costly treatment options.
Worldwide the incident rate is more difficult to get a handle on, but the Parkinson’s Disease Foundation estimates that as many as 10 million people worldwide are currently afflicted and that in the U.S., total direct and indirect costs, including treatment and lost wages, amounts to around $25 billion each year. With medications to help patients cope running around $2.5k a year for most and only somewhat effective therapeutic surgeries running somewhere in the neighborhood of $100k, there is immense demand for a more effective and less invasive solution.
International Stem Cell Corp. (OTCM:ISCO), a small publicly-traded biotech developer based in California with around 47 full-time employees, 23.4% of whom are Phd/MDs, may have just such a solution in the works thanks to their decade-plus pioneering in ethical pluripotent stem cell technology. The company’s proprietary human parthenogenetic stem cell (hpSC) platform technology, which is clearly differentiated from induced pluripotent stem cell (iPS) technology due to zero manipulation of the cellular genome and thus does not bear the safety and regulatory burdens associated with iPS approaches, is now rapidly advancing towards ISCO’s first clinical product, human parthenogenetic neural stem cells (hpNSC).
Recently vindicated in the EU via a final and definitive ruling by the EU’s highest court, the Court of Justice of the European Union, in a decision which roundly confirmed the earlier opinion issued by the Advocate General in 2014, the company’s core technology patent applications are not prohibited, or even covered under the EU’s prohibition on patenting embryonic stem cells. This decision clears the way for commercial rollout in the Eurozone of ISCO’s rapidly advancing hpNSC technology, as parthenogenetic stem cell’s legally patentable status is now clearly differentiated from still-banned embryonic stem cell patenting.
Earlier this month, the company was pleased to announce that they have successfully completed manufacturing the requisite cell bank for their upcoming Phase 1/2a clinical trials in Parkinson’s, having produced in excess of 2.6 billion clinical-grade human neural stem cells, which is more than enough to cover all foreseeable clinical needs, utilizing the company’s patented hpNSC production process. The company’s highly-optimized and chemically-defined differentiation production process was published in a Nature publication in 2013 and ensures that all hpNSCs are manufactured according to strict cGMP-compliant conditions and that these high purity cells are effectively cryopreserved.
This platform and production technology puts ISCO at the forefront of hpSC science today and makes this publicly-traded biotech company a real up-and-comer for investors to keep a close eye on, as their hpSC platform and production architecture’s potential goes well beyond just developing Parkinson’s treatments. The company is also currently developing hpNSCs for ischemic stroke, a leading cause of death in the U.S., which claims over 130k lives each year and which represents over 87% of all stroke cases caused by a clot in a blood vessel that supplies the brain.
Treatment options are very limited for ischemic stroke sufferers and while dissolving the blood clot within two to three hours after the acute phase has shown to be effective, often severe debilitation can occur and extensive rehabilitation is necessary to recover (some, generally not all) cognitive and motor functions. With around 800k cases of stroke each year in the U.S., effectively costing (direct and indirect) over $74 billion, the demand for ISCO’s hpNSC solution is potentially even greater here than it is for Parkinson’s.
With compelling pre-clinical evidence that NSC treatment can actually reverse the debilitation caused by a stroke, even when it is utilized several weeks after the initial acute phase of the stroke, ISCO’s highly purity hpNSC solution could emerge as the de facto treatment option for ischemic strokes and seriously help patients to ably recuperate from an otherwise potentially crippling incident. Looking at the company’s hpNSC clinical development pathway for such CNS issues as Parkinson’s and ischemic stroke, it is pretty easy to see that additional cell therapy treatment options for other CNS diseases and disorders can be developed using this technology.
ISCO is dedicated to developing new treatment options for CNS diseases, as well as those of the liver and eye where such cell therapy has demonstrated efficacy, but where options are currently limited by a lack of readily available, safe human cells. The company’s ability to readily produce high purity stem cell-derived liver cells (CytoHep), as well as human retinal epithelium (RPE) cells and human corneal cells/whole corneal tissue (CytoCor) from human parthenogenetic stem cells, puts their development pipeline potentially within eventual striking distance of treating a vast array of conditions. Conditions like the metabolic liver disease Crigler-Najjar syndrome, which can lead to severe brain and nervous system damage (as well as death), as well as eye conditions like age-related macular degeneration, which causes vision loss and blindness in as many as 50 million people around the world today.
Learn more about the company by visiting www.internationalstemcell.com
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