Despite some amazing advances medicine has made in the past 30 years, the lack of progress has been equally amazing on some fronts. Alzheimer's disease is one of those areas where we haven't seen much progress. That may be about to change though, not because of a new biotechnology, but because of a twist on a very old idea. Investors should take note.
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The Barrier, in More Ways Than One
The cause of Alzheimer's isn't a misunderstood one - it's a buildup of toxic abeta 42 proteins in the brain. The challenge has been in getting drugs to the brain to do the repair work. For years, the barrier between the brain and its blood flow - simply referred to as the blood/brain barrier - hasn't allowed medicine to be delivered to brain cells. This is because drug molecules are bigger than the tiny permeable passages in that barrier.
That changed earlier this year, however, and a handful of public companies are charging ahead with the idea of this so-called "small molecule technology." (For related reading, see Investing In The Healthcare Sector.)
A Lot of Promising Candidates
With the help of Astex Pharmaceuticals (Nasdaq:ASTX), AstraZeneca (NYSE:AZN) is working on AZD-3839 - a drug that blocks the enzyme that may ultimately induce Alzheimer's. AstraZeneca owns the drug; Astex Pharmaceuticals is supplying the technology that allows AstraZeneca to develop it. It's called "Pyramid," and it's not only being used by AstraZeneca to develop this drug, but by several other companies looking to develop other small molecule therapies ... now that they can.
Pink sheet company Intellect Neurosciences (OTCBB:ILNS) has also developed a small molecule drug-development platform (molecule), which other companies are using to develop their own Alzheimer's drugs. Pfizer (NYSE:PFE), for instance, is in Phase III testing of Intellect's ANTISENILIN as a treatment for Alzheimer's.
However, Intellect Neurosciences is also developing a couple of its own Alzheimer's treatments using CONJUMAB-A. They're simply being called RV01 and RV02 for now ('Recall-Vax'). Though both are pre-clinical, the fact that they're both small molecule founded make them worth watching.
Elan Corporation (NYSE:ELN) is working on a drug along those same lines - ELND-005 - a small molecule beta-amyloid anti-aggregation therapy (against abeta 42, specifically), that is expected to act as a treatment for Alzheimer's by preventing the buildup of a toxic protein in the brain.
The common element? Big bucks are being shelled out by some big names to develop small molecules. The Elan development speaks most favorably of the premise, in that the company's Phase II results didn't meet the targeted endpoints, yet the company is moving ahead with its development anyway.
The Bottom Line
The final chapter in the Alzheimer's race is far from written. In fact, the Alzheimer's-treatment story may be back to the beginning again. The technology this time around, however, seems to hold more promise than many other Alzheimer's treatment ideas and approved drugs have of late. Indeed, some consider current therapies ineffective to the point of uselessness.
For the company, or companies, that can get their small molecule platforms to deliver drugs as hoped, a $3 billion Alzheimer's market awaits them. That number, however, assumes the current treatment market is its full size. Remember though, most of the currently- available drugs are ineffective, and therefore, unmarketable. If a truly effective treatment option became available, some experts believe that the Alzheimer's drug market could swell to as much as $20 billion. (For related reading, see Evaluating Pharmaceutical Companies.)
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At the time of writing, James Brumley did not own shares in any of the companies mentioned in this article.