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
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