Degenerative disorders, which often strike our increasingly elderly populations, like the roughly 10k Baby Boomers in the U.S. hitting their 60s and retiring each day, are on the rise all over the planet, affecting more than 45 million people worldwide. Characterized by nerve cells progressively deteriorating to the point of cell death, a process which leads to a host of maladies ranging from Alzheimer’s and Parkinson’s disease, to ALS (amyotrophic lateral sclerosis) and Huntington’s disease, degenerative disorders currently have few, if any, real medical solutions available for patients.
In China, Europe and Japan, the same population and disease phenomena is occurring. Populations where people are living longer and where increased environmental exposure to various bio-accumulating toxins is causing a continued rise in the volume of a variety of neurological diseases and disorders, for which modern medicine remains largely incapable of providing substantial cures, and in some cases, even palliative treatments. This is why the regenerative medicine technology, based on an entirely new class of ethically manufactured stem cells, which is being aggressively developed toward full commercialization by International Stem Cell Corporation (OTC: ISCO), is so exciting.
Parthenogenesis is not a term most people today are familiar with, but the times they are a-changin’ – and they are changing fast. Degenerative disorders once thought incurable are now within striking distance of being effectively combated through the injection of human parthenogenetic neural stem cells (hpNSCs), which not only have all the best characteristics of other types of stem cell technologies, but lack their drawbacks as well. Thanks to the revolutionary work being done by ISCO with pluripotent stem cells (which that have the ability to mature into several different types of cells), chemically stimulating oocytes through a process where no embryo is ever created or destroyed, the dawn of ethical, high-volume, commercial stem cell technology that could be used to treat a wide variety of diseases, is at hand.
Derived from unfertilized human oocytes, the company’s stem cell lines can be made, via their proprietary process, into HLA (human leukocyte antigen) heterozygous “matched” and therefore histocompatible cells that won’t be rejected by the donor, giving this technology a serious advantage in a field still plagued by donor cell rejection. HLA homozygous hpSCs can also be produced which have been shown to exhibit histocompatibility across massive segments of the global human population, meaning that ISCO’s stem cell bank approach to the space could put the company at the forefront of supplying much-needed, large supplies of compatible donor cells to global markets, just as commercialization of this revolutionary technology dawns worldwide.
The upper limit on therapies involving stem cells is quite extraordinary, as these cells function like an atomized organ or other tissue transplant, repairing existing tissue systems by developing into healthy new cells that replace the damaged ones. In degenerative neurological disorders, this is truly a quantum leap, as no effective means exists to otherwise replace brain and other CNS tissue. The idea of injecting Parkinson’s disease (PD) patients with cultured neural stem cells (hpNSCs) that actually grow into new brain cells is extraordinary, and this same approach can be used to treat other neurological diseases as well, for many of which modern medical science can offer essentially only palliative care.
The current standard of care in PD is with dopamine agonists and drugs like oral levodopa (L-dopa), which has significant dosing issues that often lead to episodes where the symptoms of PD reemerge with a vengeance, particularly in patients who have been on an L-dopa regimen for over five years or more. In fact, 90 percent of young-onset sufferers treated for more than five years with L-dopa experience such episodes. L-dopa, while exhibiting fewer than other antiparkinsonian agents, has numerous adverse effects, including psychiatric ones, as well as the potential for the patient to develop dopamine dysregulation syndrome, and even eventual drug resistance. Needless to say, existing approaches are by no means effective solutions and the advent of an actual therapy for restoring the underlying tissue systems would be a paradigm shift of unprecedented magnitude. A shift whose shockwaves would likely propagate into multiple other degenerative disorders, especially considering the removal of ethical considerations about where the stem cells come from, given ISCO’s parthenogenesis production techniques.
The company’s hpNSCs help to repair the brain in several key ways, like responding to damaged cell signals and releasing anti-inflammatory molecules that help speed the recovery process. Directly addressing inflammation, which is one of the major concomitant symptomatological factors in PD, as well as forming new tissue by developing into new cells, are powerful properties that could rocket hpNSCs to the front of the line in PD therapy. Being able to give someone their life back through the application of this technology could change the way we think about degenerative disorders forever. The idea that we could take cases where a loved one has become effectively disabled – representing not just a serious loss for the individual and economy, but typically creating a significant burden for their families and the healthcare system – and actually restore them to nearly 100 percent functionality, represents one of the most exciting frontiers in biomedicine today.
ISCO’s sizeable preclinical GLP and non-GLP study data on hpNSCs, including brain transplant safety studies conducted in healthy and induced Parkinson’s disease animals, showed that not only were the hpNSC injections well-tolerated, with no sign of tumor or other abnormal growth formation at even high dosages, but that a distinct improvement to motor function resulted. These same studies further showed the neuroprotectant and recovery assistance functionality of hpNSCs, as well as a significant increase in dopamine levels, the primary agent which allows brain cells involved in movement to communicate with one another. National Parkinson Foundation data indicates that even in the roughly 15 percent of patients who develop young-onset Parkinson’s disease before the age of 50, the same loss of some 80 percent of dopamine-producing cells is observed before motor function impairment arises, something which clearly indicates the potential of ISCO’s technology.
ISCO is barreling towards their 1/2a clinical study in Parkinson’s disease over in Australia, which should kick off within the coming handful of months, as the company has already submitted the requisite CTX (Clinical Trial Exemption) application needed to commence this landmark study. The high safety levels of this technology and emerging knowledge of the causes and symptomatology of degenerative disorders, could even lead to developing hpSCs into frontline preventative treatments in patients who are identified as at risk or in the early stages of a given disease.
At any rate, ISCO has their eye on the prize and has already completed manufacturing a large supply of over 2.6 million clinical-grade hpNSCs for the Australia Parkinson’s study, enough to easily handle any foreseeable trial requirements, and further validating their UniStemCell bank approach to the market. Paired up with their proprietary parthenogenesis process for manufacturing cells, a regionalized stem cell bank franchising architecture could be a significant source of future revenues for the company, serving scientists and populations across the country and around the world with an essential stem cell line banking framework. Such a framework, while helping to further validate a growing library of hpSC lines with many people working on and developing them, could also develop into a serious royalty-generating engine fueled by diverse, emergent cellular therapeutics.
ISCO has a strong IP position with numerous patents and filings under their belt too, spanning everything from specific pluripotent hpSC lines and production methods, to cell differentiation and research methods, as well as therapeutic and commercial uses. Like the 16 issued and 91 pending patent applications across 15 different patent families, as well as 8 patents pending across 4 different families, covering their stem cell-based Lifeline Skin Care (http://www.lifelineskincare.com/) products.
For more information, please visit www.internationalstemcell.com
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