Alzheimer's disease - huge unmet need, signs of hope

Jeff Elliott's picture

I recently attended the American Academy of Neurology (AAN) annual meeting in Washington, where advances in the development of drugs for debilitating diseases such as Alzheimer’s disease (AD) and Multiple Sclerosis (MS) were presented. Although still in the early- to middle-innings of development, there are encouraging signs of hope from a number of companies. These could ultimately represent huge steps forward in the management of some very difficult-to-treat diseases. I came away incrementally positive on AD drugs from Biogen Idec and Roche’s new MS treatment.

In my view, one of the most exciting areas of current development is in drugs for the treatment of AD. Historically a “graveyard” for new drugs, there have recently been significant advances in understanding the root cause of disease progression and, even more excitingly, potential treatments that address the underlying pathology. Despite billions of dollars spent in R&D and decades of work, all of the currently available medications for AD treat some of the symptoms, but do not alter the underlying cause of the disease.

Recent trials by companies such as Biogen Idec, Roche, and Eli Lilly give us hope that new treatments that could potentially alter the course of certain diseases could finally be on the horizon. These drugs target the removal of amyloid plaque, a toxic protein that builds up in the brains of patients with AD. At the AAN meeting,  Biogen Idec presented details of its Phase 1 trial (the first stage of trials in humans) for its novel antibody (BIIB-037) that demonstrate that the drug can reduce amyloid plaque levels as well as slow the decline in cognitive function, in a dose and time-dependent manner, a key hallmark of an effective drug. Excitement amongst neurologists is palpable, with hope that a disease modifying treatment is potentially now in sight.

Although BIIB-037 is still several years away from the market and is not a “cure” (the drug appears to slow but not reverse decline), the unmet need in AD is huge, with approximately 44 million patients affected globally and an estimated US$605 billion spent per year on treatment. The potential of a disease-altering AD treatment is significant both commercially (likely on the order of $10-20 billion per year), and clinically, representing the first drug that could actually slow progression of a devastating illness with a huge societal burden.

We currently own shares of Roche and Biogen Idec in many of our Signature funds and believe that focusing on companies at the forefront of transformative science will generate outsized returns in the long-term.

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