Two Alzheimer's Breakthroughs

Jason Stutman

Posted March 17, 2014

There are very few things more heartbreaking than the effects of Alzheimer’s disease.

If you’ve had the unfortunate experience of being acquainted with this monster, you know firsthand the emotional toll it can take on everyone involved.

Alzheimer’s doesn’t just destroy the life of a single victim — it consumes entire families as they helplessly watch their loved ones fade into madness.

To me, this is absolutely terrifying. Broken limbs and organs can be replaced, but dementia has so far proven irreversible. Once you lose your mind, it’s nearly impossible to get back.

Because of the irreparable damage that’s involved, early detection has become crucial in developing and implementing treatments for Alzheimer’s. Unfortunately, conventional diagnostic methods have proven too invasive, too expensive, or too time-consuming for widespread adoption.

Patients currently have just three choices: costly MRIs, invasive spinal taps, or a blood test for beta-amyloid protein ratios.

Of these three methods, blood-based biomarkers offer us the best shot at large-scale screening. Blood tests are quick, cheap, and minimally invasive.

Patients are unlikely to ever accept spinal taps as a part of their routine checkups, and medical centers cannot afford the personnel, equipment, or facilities required for high-frequency MRI screening.

Unfortunately, finding a reliable blood-based biomarker for Alzheimer’s has been difficult because blood does not come in direct contact with our brains thanks to protection from the blood-brain barrier (BBB). So while beta-amyloid ratios may accurately correlate with Alzheimer’s after onset of the disease, they are not sensitive enough to effectively indicate pre-clinical cases.

For this reason, the scientific community has been actively seeking a better blood-based biomarker — one that can predict the onset of Alzheimer’s disease — and they may have just found it.

On March 9, Nature Medicine published a study revealing a newly developed blood test that identifies 10 lipids that predicted Alzheimer’s disease up to three years in advance with over 90% accuracy.

If you’re curious, the primary reason this works is that lipids are especially skilled at penetrating through the BBB. This means we can get a fairly accurate idea of what’s going on in the brain by looking at the lipids in our bloodstream. Specifically, these lipids can reveal the breakdown of neural cells years before cognitive impairment.

Now, this is truly incredible news for the medical community, and we can expect further research in this area. But it will likely be some time before lipid-based screening reaches the commercial market.

Until then, we’re looking at 35 million individuals currently suffering from Alzheimer’s disease and 115 million expected cases by 2050. That’s undoubtedly a depressing statistic, but it highlights an incredible market opportunity at the same time.

The unfortunate reality is that the Alzheimer’s market is incredibly underserved right now. There are many treatments available that address symptoms, but there are currently no drugs available to reverse or halt the progression of Alzheimer’s disease.

This fact, along with an aging global population, makes prospective treatments for the disease incredibly valuable. There are currently 20-25 drugs at various stages of the pipeline aimed at meeting this need. The trick, of course, is picking the right ones.

Tick-Tock, Tick-Tock

Of all the Alzheimer’s related drugs currently in development, there is one particular candidate that currently stands out above the rest.

Right now, this drug is in Phase 2 clinical trials, with an FDA decision expected at the end of the month.

The innovative treatment is being developed by an Australian biotech company with potential annual revenues over $5 billion and a market cap of just $295 million. We added this company to our Technology and Opportunity portfolio back in October and are up 170% since.

With an FDA decision expected in mere weeks, we’re looking at even further breakout potential, and we’re feeling pretty good about our prospects.

Unlike the current lot of approved drugs that do not modify the disease, this promising candidate actually treats the underlying structure of Alzheimer’s.

As a quick background, research has shown that certain metals, such as copper, play a significant role in Alzheimer’s disease. These metals interact with naturally occurring proteins in our body (beta-amyloids) to create a toxic material known as oligomers that damage the synapses in our brains.

The problem for developing a treatment is that we need these metals and proteins for other important bodily functions. Copper is a critical component for energy production in our cells, and beta-amyloids work to regulate cholesterol transport. Without these materials, we simply wouldn’t be able to survive.

The solution to this dilemma therefore lies not necessarily in the reduction of these metals or proteins, but in disrupting the interaction between them. This is where our Phase 2 candidate comes in.

The drug, known as a Metal Protein Attenuating Compound (MPAC), works by blocking the interaction between copper and beta-amyloids. This, in turn, prevents the creation of destructive oligomers and helps restore synaptic function in the brain.

In the first stage of its Phase 2 study, the drug was shown to significantly improve the Executive Function in mild Alzheimer’s disease patients within just three months of treatment.

With a longer study at hand, we’re expecting similar results for the final stage and jointly a positive FDA decision by April.

That gives you just two weeks to get in…

Turning progress to profits,

  JS Sig

Jason Stutman

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