Finding A Cure For Parkinson's

by: Jonathan Verenger

Parkinson's - which afflicts about a million Americans, including boxing legend Muhammad Ali and Michael J Fox - is a progressive neurological disorder that mostly strikes people over 50, causing tremors, slow movement and other problems.

It occurs when nerve cells in the brain that produce dopamine, a chemical that helps control muscle movement, are slowly destroyed.

Here is a list of companies attempting to develop a cure:

Living Cell Technologies Limited (OTCQX:LVCLY)

Living Cell Technologies Limited (OTCQX:LVCLY) has received ethical approval for its NTCELL. In a controlled trial for NTCELL, a Parkinson's-like condition was produced in non-human primates using drugs which selectively deplete dopamine-producing neurons in the brain. Reduction of dopamine levels in the brain is a hallmark of Parkinson's disease in humans. The non- human primates developed movement disorders and neurological deficits similar to Parkinson's patients. NTCELL was then implanted into the affected brain areas and within two weeks, improvements in both motor abnormalities and neurological function were observed in the NTCELL treated subjects compared to controls. These improvements increased and persisted for at least six months beyond the NTCELL implant, the end-point of the study. Microscopic analysis of both treated and untreated brain regions clearly showed an increase in the number of dopamine producing neurons in the NTCELL treated subjects compared to controls. In addition, there was no cellular or pathological evidence of inflammation or other adverse event.

These results from February 2012 were consistent with all Living Cell Technologies' prior pre-clinical NTCELL studies in other small animal models of Parkinson's.

Sanofi-Aventis (SNY)

The drug company Sanofi recently entered into a collaboration with the Michael J Fox Foundation - MJFF - to conduct a clinical trial to assess the safety of a new drug, a type of inhibitor called phosphodiesterase (coded AVE8112). Todd Sherer, Ph.D., Chief Executive Officer of MJFF, said that the drug had: "Shown promising pro-cognitive activity in preclinical models that could be of interest to the under-addressed cognitive aspects of Parkinson's disease, an area of unmet need where a new treatment could make a tangible difference in patients' lives."

Amicus Therapeutics, Inc. (FOLD)

Amicus Therapeutics is pursuing orally administered drugs called pharmacological chaperones to treat a broad range of diseases of neuro-degeneration. Amicus is targeting the GBA1 mutation that has been identified as the most common genetic factor of Parkinson's.

Amicus is currently undergoing preclinical studies to evaluate two of these chaperones, AT2101 and AT3375, in an effort to target an enzyme that is at the root cause of the disease. Amicus received a grant from the Michael J Fox Foundation to pursue this potential cure.

Impact Laboratories (IPXL)

Last month the FDA extended its review of Impax Labs Parkinson's drug, Rytary, by three months until Jan. 21. The primary endpoint of the Rytary (IPX066) trial in 2011 was the percentage of "off time"--when patients' medication effect has worn off and there is a return of Parkinson's symptoms--during waking hours. Rytary demonstrated a 37 percent improvement from baseline compared to a 17 percent improvement from baseline for the standard treatment.

"Consistent with our Phase II findings, these data show IPX066 provides a robust level of efficacy across a range of PD clinical and QOL measures, which represents a potentially significant improvement over existing treatment options," said Impax CSO Dr. Suneel Gupta.

Amarantus BioSciences (OTCQB:AMBS)

Amarantus' proprietary anti-apoptosis therapeutic protein MANF, recently announced that the Company has received positive behavioural efficacy data for MANF in a neurorestoration 6-hydroxydopamine (6-OHDA) rat model of Parkinson's disease. The data demonstrated that when MANF is delivered directly to the primary brain region associated with Parkinson's called the substantia nigra, MANF significantly reduced behavioural deficits in the model, whereas GDNF (another therapeutic protein in Phase II trials) did not.

Dr. Joseph Rubinfeld, co-founder of Amgen, stated "I have reviewed a great number of technologies in my 45 year career in the biopharmaceutical field, and I believe that MANF could be one of the biggest successes that I have ever seen. The data, while early, demonstrates very clearly at the cellular level and in animals that MANF reduces apoptosis, improves cellular function, and restores behavioural deficits in a number of disease models, including Parkinson's, Stroke, Myocardial Infarction and Traumatic Brain Injury. These are all indications with very large markets and clear unmet medical need. I believe that if we are able to further de-risk MANF with positive toxicology studies and early clinical data, the Company's new orphan drug strategy could get MANF to market rather expeditiously. MANF has the commercial potential to become a blockbuster drug."

Amarantus is also a recipient of a grant from the Michael J Fox Foundation.


Please note that both Living Cell and Amarantus are penny stocks and are subject to wild swings in their prices. Both are startup companies and may lack the resources to bring their drugs to market, therefore potentially subjecting shareholders to dilution.

Disclosure: I have no positions in any stocks mentioned, but may initiate a long position in SNY, FOLD over the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.