While heart disease and cancer may be this nation's top two killers, it doesn't make the prospect of developing Alzheimer's any less terrifying.
Based on statistics from the Alzheimer's Association, more than 5 million people in the United States, most of them seniors, currently have Alzheimer's disease. But, by 2050 nearly 14 million people within the United States are expected to have this terrible disease.
Why Alzheimer's is so tough to treat
Alzheimer's is a particularly difficult disease to treat for two reasons.
First, the blood-brain barrier makes it tough to develop drugs that will effectively target the brain. To date, a number of late-stage attempts to develop an Alzheimer's therapy have failed, including bapineuzumab which was being co-developed by pharmaceutical powerhouses Johnson & Johnson and Pfizer, and solanezumab from Eli Lilly. It should be noted, though, that solanezumab is being studied in a longer-term European trial as it may have benefits in early stages of the disease. However, the point is this: regardless of how deep a pharmaceutical company's pockets have been, we still don't have an Alzheimer's cure.
The other reason Alzheimer's is a tough disease to treat is that researchers aren't entirely certain what triggers it. High risk factors such as age and family history likely play a role, but researchers haven't discovered the secret as to why one person winds up with Alzheimer's yet another person does not. This makes personalizing treatments or targeting drugs at Alzheimer's a challenge for drug developers.
However, this tough-to-treat disease may have moved one step closer to a cure as of last week.
A big breakthrough from "down under"
Based on a study out of the Queensland Brain Institute (QBI) at the University of Queensland in Australia and published in Science Translational Medicine, a method to reverse the effects of Alzheimer's disease may be possible -- and best of all it could be non-invasive!
Using mouse models, researchers at QBI utilized a very specific type of repeated scanning ultrasound treatment to breach the blood-brain barrier and send sound waves into the brain tissue. These sound waves stimulated the microglial cells in the mice which are responsible for removing waste from the brain, which includes both beta amyloid plaques, the protein responsible for sticking between neurons and progressively disrupting cognitive function, and tau proteins, which are responsible for neurofibrillary tangles within neurons that disrupt the transportation of nutrients to your brain.
According to the findings of researchers, after utilizing repeated scanning ultrasound treatments, 75% of the mice tested had their memories restored. This was confirmed by testing the mice in a handful of memory tasks, such as mazes and the recognition of new objects. Best of all, researchers noted no damage to the surrounding brain tissue of the mice, and the ultrasound didn't need to be accompanied by any anti-amyloid drugs.
The next step for researchers will be to move onto larger animal models shortly and potentially even human clinical studies by as early as 2017.
Big changes could be on the way
I'd caution readers not to get too excited considering this is just a mouse model study and the reaction in larger animals and humans could be markedly different. Nonetheless, I can't help but be encouraged by the high percentage of success exhibited by the simply use of repeated (yet precise) ultrasound technology.
Of course, it could also be years before we see the results of human clinical trials and have an approved therapy for physicians to use on Alzheimer's patients. In the meantime, though, we do have a handful of Alzheimer's therapies working their way through clinical trials that may make a more immediate impact on Alzheimer's patient's quality of life.
Perhaps no Alzheimer's drug candidate has received more attention than Biogen's (NASDAQ:BIIB) BIIB037, also known as aducanumab. Earlier this month, Biogen released its full set of data from its phase 1b study on the drug, and it was everything that optimists had hoped for.
In general, the 166-patient study in patients with early to mild forms of Alzheimer's disease showed that the higher dosage of aducanumab administered, the more pronounced the effect of beta amyloid removal. Within the 30-point mental acuity test the placebo group saw a 3.14 point decline over the course of a year compared to a decline of just 0.75 points and 0.58 points, respectively, for those receiving the 3 mg and 10 mg aducanumab dose.
A second test, the 18-point Clinical Dementia Rating, demonstrated a 2.14 point decline for the control group, with the 10 mg aducanumab group achieving statistical significance with a 0.59 point reduction. The 3 mg group did not meet statistical significance in this second study.
In addition to removing beta amyloid plaques and improving cognition, Biogen's drug was deemed safe with very few serious side effects. Aducanumab has been shuffled straight from phase 1b to phase 3 trials, and we could have data as soon as next year on whether or not this drug could offer new hope for Alzheimer's disease patients.
Also, as I noted in February, privately held TauRx Pharmaceuticals is worth keeping a close eye on. TauRx's lead drug is a phase 3 study involving LMTX, a drug that targets misfolded or aggregated tau proteins. As TauRx points out, LMTX is something of a second-generation Alzheimer's therapy for the company, with it having the same active ingredient as previously tested Alzheimer's drug Rember, but with a more favorable safety profile. For those who are unaware, Rember delivered a 90% reduction in the rate of Alzheimer's progression in a prior phase 2 study, so the combination of slower progression and better safety could make for an effective Alzheimer's treatment.
Although investors can't buy into TauRx at the moment, there's a possibility if the drug is approved or it succeeds in late-stage studies that it may choose to go public and raise cash for a product launch.
While the victories have been few and far between for Alzheimer's patients, there appear to be an exciting number of drug-based and even device-based therapies on the horizon. Though change isn't likely to happen overnight, it's nonetheless encouraging to see that progress is being made and that researchers are indeed pushing forward toward a cure.
Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.
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