Lou Gehrig's Disease is the colloquial name for Amyotrophic Lateral Sclerosis (ALS), a neurodegenerative disease that inhibits proper messaging between the brain and muscles, eventually leading to complete paralysis in those who suffer from it.
Named after baseball great Lou Gehrig, who was among the first high profile individuals to fall victim to the condition, ALS has no known cure, although there are treatments that may marginally extend the lifespan of those who suffer from it and others that treat specific symptoms with the aim of offering comfort to the afflicted.
The most widely administered treatment for ALS is already FDA approved and widely marketed by French biopharm giant Sanofi-Aventis (SNY), a company with a market cap just shy of $114 billion whose stock yields 3.93% annually and sports a price/earnings multiple of just 13.5.
The company's treatment, Riluzole, has proven that it can slow the process of deterioration associated with ALS, but cannot stop it. It's a far from perfect solution to a devastating condition, but as of today, it's the only commercially available product that's been tested, and to its credit, it does do something. For the record, there have been cases of riluzole causing liver damage. Its administration, therefore, has to be monitored closely.
The other drug that's regularly administered to ALS patients isNuedexta, a treatment developed by Avinir Pharmaceuticals Inc. (AVNR), a small cap development company that's known principally for its cold sore remedies, but whose ALS drug helps reduce the emotional instability that accompanies the disease.
All in all, for those who have ALS, it's not a tremendously optimistic situation.
Up and Comers
Of course the game's not over, by any stretch. There are a host companies currently working on stem cell-based treatments for ALS and other related conditions. Among the most prominent of these areBrainstorm Cell Therapeutics Inc. [BCLI.OB], an Israeli company; Neuralstem Inc. (CUR), whose treatment pipeline includes a tremendous array of stem cell-based clinical hopefuls; Stemcells Inc.(STEM), a public company now traded for over two decades; and Neostem Inc. (NBS), which also offers collection and storage services for healthy adults interested in storing stem cells for later therapeutic use.
Let's have a brief look at each.
We start with Brainstorm, whose initial trials of their NurOwn treatment have apparently restored one patient's ability to both walk and speak without difficulty after having earlier being confined to a wheelchair and losing his ability to communicate.
While the company is quick to assert that they're still at the outset of their clinical testing phase, all agree that initial indications are positive. However, for those awaiting treatment, it will be some time before Brainstorm's product is available for commercial release in the U.S., as they've only this summer jumped on the FDA approval track.
NeuralStem Works all Angles
NeuralStem may be the most exciting of the above listed crop of startups, for the simple reason that it has so much on its plate. Along with being first to the plate with an FDA-approved neural stem cell trial for the treatment of ALS, the company is also developing treatments for stroke victims and for those who suffer from major depressive disorders.
Most recent results have shown positive for the company. Its ALS treatment is now in advanced Phase I trials and should wrap up just after New Year's, while expansion trials and phase II trials are already in process in Mexico (see diagram below).
The company's stroke remedy has also shown strong results in rats, restoring both motor and neurological function after stem cell transplantation therapy.
FDA approval was received this month to treat a second cohort in its major depressive disorder application.
Below is an overview of the company's FDA clinical targets for the next twelve months.
NeuralStem is ambitiously targeting treatments for a variety of conditions directly after its ALS remedy is finalized. They include Alzheimer's, Post Traumatic Stress Disorder, Huntington's Disease, Multiple Sclerosis, Parkinson's and Cerebral Palsy, to name just a few.
The company also possesses 9 U.S. patents which have been reaffirmed worldwide and an exclusive worldwide license for its own intraspinal stem cell delivery platform.
New Capital Approved For StemCells Inc.
StemCells Inc. has been around since the early '90s, researching and developing commercially viable technologies for academia and industry in the stem cell field. Last month it was awarded $20 million from the California Institute for Regenerative Medicine to fund preclinical costs associated with its Alzheimer's treatment - a treatment the company believes may have applications to a number of other conditions, including ALS.
Neostem and the Old Startup Story - Cash Burn
NeoStem has undergone some significant corporate changes in the last couple of years, merging with Progenitor Cell Therapy LLC in January 2011, then acquiring Amorcyte Inc. the following October.
Yet despite the company's best efforts, what's most notable for this writer is NeoStem's stock chart, which looks like this for the last few years:
If that's not distressing enough, the company's shares have lost exactly 99.68% of their value since the IPO on January 14th, 2000. To make matters worse, NeoStem now faces litigation, in which shareholders are alleging inappropriate compensation was given to the company's operators.
Granted, it's not easy keeping a publicly traded biotech developer afloat in general, let alone one involved in the stem cell research game.
There have been a number of notable flops in this sector, including much hyped industry forerunner, Geron (GERN), whose fifteen year history in the field hit an historic milestone exactly one year ago, when its CEO admitted its research in stem cells had yielded no benefit to patients with spinal cord damage. The company has since refocused itself on developing therapies for cancer.
Generally, investors know that development stage companies are hard to profit from, as their cash burn rate almost always necessitates new share issuance and reverse stock splits that are punitive to initial investors.
Raising capital is of the essence of the business for the first few years at least, until FDA approvals are near complete or larger biotech concerns pony up cash for the privilege of sharing in the later rewards.
Companies like Neostem may be successful at acquiring successive new rounds of financing, but it hasn't yet translated into shareholder value.
A chart of Neuralstem's stock for the last five years shows a company that doesn't appear to be burning through its cash at as fast a rate.
That should offer longer term investors some hope.
Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this article.