The true forefront in medicine today is a broad offensive where medical and research professionals are now pulling out all the stops in a never-ending war against broad-spectrum degenerative diseases like cancer or degenerative diseases of specific tissues, such as Parkinson’s and Alzheimer’s, which severely cripple a patient’s central nervous system (CNS). Unfortunately, there is very little in the way of truly therapeutic options for patients with degenerative CNS diseases.
In the case of Parkinson’s, dopamine-generating neurons in the midbrain (substantia nigra) progressively die off, resulting in a variety of motor control issues (dyskinesia) at first, with dementia, insomnia, and severe depression or emotional problems typically following in later stages. There is no currently known cure for Parkinson’s and the standard of care consists primarily of medications designed to manage and/or provide relief from the symptoms.
The main family of drugs used to offset Parkinson’s symptoms is Levodopa (L DOPA, which metabolizes into dopamine), but MAOIs (monoamine oxidase inhibitors) and dopamine agonists have seen a significant increase of use in recent years as a first choice, in order to prolong the start of L DOPA treatment. For you see, prolonged use of L DOPA typically results in dyskinesia that is equivalent to the long-term effects of Parkinson’s itself.
Because less than 10 percent of L-DOPA actually makes it through the blood-brain barrier, the vast majority of it is metabolized elsewhere in the body, resulting in numerous side effects like nausea and joint stiffness, in addition to the aforementioned Parkinson’s-like motor control problems. MAOIs, historically already in wide usage as a treatment for atypical depression, are pretty effective at delimiting the primary monoamine oxidase that degrades dopamine, MAO-B, and thus are able to somewhat offset the lack of dopamine that is being caused by neuronal loss.
As you can see, the only solutions for Parkinson’s patients which are currently available aren’t really solutions at all, and carry with them the looming inevitability of a lost battle against this degenerative disease. A truly disheartening reality for patients and their families. Long-term options for Parkinson’s patients and their families are severely limited as well and include invasive surgery, or palliative care designed merely to improve quality for end of life patients. Reasonable extrapolations from official Parkinson’s Disease Foundation data indicates that the number of people on earth currently suffering from the disease is likely close to, or over 10 million. Some 60,000 or more people in the U.S. alone are diagnosed with Parkinson’s each year, meaning the real number is likely much higher, after factoring in all the cases that go undiagnosed, and unreported.
Hence the undisputable potential value of the proprietary, scalable and ethical human parthenogenetic (asexual reproduction from unfertilized egg) stem cell (hpSC) technology currently being developed by International Stem Cell Corp. (OTCQB: ISCO). Because hpSCs are self-renewing multipotent cells, they represent an as-yet essentially untapped goldmine of therapeutic developments which could provide solutions for countless degenerative diseases, and do so across multiple tissue types. The company’s hpSC platform for chemically stimulating eggs to reproduce, which uses a series of different activation techniques in order to create sizeable batches of healthy adult cells that are HLA/immune-matched (human leukocyte antigen) either to the individual or to the general population, has led to an exciting novel therapeutic cellular product consisting of human parthenogenetic neural stem cells (hPNSCs).
Because hPNSCs have been shown to be able to actually differentiate into dopaminergic neurons, therapy using these injected cells represents a wholly-new approach to the problem of Parkinson’s, wherein the root cause of the disease is addressed directly. Moreover, transplanted hPNSCs have been shown to express powerful brain-protecting neurotrophic factors in pre-clinical animal model studies, meaning that not only does this product hold the potential to simply grow new dopamine-producing cells, it can also help shield the remaining healthy cells from degeneration and/or death. ISCO’s recent announcement that the company is now moving full steam ahead towards phase I/IIa human clinical trials in Australia, subsequent to a meeting with the Australian Therapeutics Goods Administration and signage of an LOI with the conducting facility, Royal Melbourne Hospital, is a major milestone for the company. A milestone that puts ISCO squarely in the pole position for developing the first true Parkinson’s therapy.
TGA approval for the phase I/IIa clinical trials is expected sometime this month, with enrollment commencing shortly after, and ISCO could have a real winner on its hands depending on whether the results jog with those generated by the preceding nine-month safety GLP study of 300 rodents, which showed zero tumor growth in any of the subjects receiving transplanted cells. ISCO seems to have overcome the two major stumbling blocks that have hindered other developers in this field: immune-related tissue rejection and tumor formation.
The chemically close-to-nature methodology whereby the company generates its hpSCs is likely a main reason its therapies have had such preclinical successes, and one need look no further than the results for the other candidates (such as those for metabolic liver and degenerative eye diseases) in ISCO’s therapeutic pipeline in order to get a good idea of where the Parkinson’s therapy is headed. A savvy observer will note that the probability of success for ISCO with its hPNSC phase I/IIa clinical trials is telegraphed readily by the demonstrated versatility of the platform in allowing for a robust pipeline of several promising indications. The hpSC platform looks solid and ISCO could have one or two disruptive commercial breakthroughs on its hands in the near future.
Unlike many preclinical biopharma developers, ISCO has a cash pipeline already in place to help fund the expensive work of drug trials, with two wholly-owned subsidiaries that benefit from the company’s hpSC platform: Lifeline Cell Technology and Lifeline Skin Care. Respectively engaged in the sale of human cell culture products/reagents, as well as cosmeceuticals based on a proprietary extract derived from hpSCs, these two profitable subsidiaries not only help feed the R&D machine that is ISCO, they represent promising long-term opportunities in and of themselves. Quarterly financial data out as of November 16 from ISCO shows that Lifeline Cell Technology sales were up handsomely in Q3 (ended September 30), climbing 22 percent compared to the same quarter last year, alongside a nine percent jump in the company’s total consolidated revenue over the same period. Having wound down its multiple preclinical studies during the first six months of 2015, ISCO has managed to slash its cash burn rate and the company is now eager to see the fruits of its labor emerge from human clinical trials of hPNSCs in Parkinson’s.
The ability to grow functional, immune-matched adult human stem cells without the need to fertilize an egg is as ground-breaking a revolution in medicine as it sounds. And ISCO is basically the tip of the spear here too, alongside a tiny handful of other companies, many of whom lack the crucial IP and pre-clinical success story to deliver on a platform solution that could eventually hit hard and fast across the gamut of degenerative and similar diseases.
To find out what the buzz is all about, visit www.internationalstemcell.com