Why We Must Find Treatment for Alzheimer’s Disease

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Alzheimer’s is Rising Worldwide and Will Affect Baby Boomers Most

By Dr. Martin Tolar

While baby boomers have largely been caregivers for their aging parents with Alzheimer’s disease, their generation will now soon become afflicted. Therefore, baby boomers will face the dual challenge of caring for spouses or siblings, while staying cognitively fit enough to continue working to generate sufficient income to fund their own health needs in retirement.

Using therapeutics to reduce the societal disease burden, as has been achieved in  cardiovascular disease or cancer, is not likely to be available in time to help the current generation deal with the looming cost tsunami of Alzheimer’s disease, unless we create a sense of urgency about testing new treatments in time for the baby boomer generation.

Currently about 5.8 million Americans have Alzheimer’s disease, and that number is expected to grow to 8.4 million by 2030, and 14 million by 2050. Ten million of those people will be baby boomers. The disease affects one in eight Americans aged 65 and older. By the age of 85, this becomes one in two. By the age of 80, 75 percent of people with Alzheimer’s end up in a nursing home, compared with only four percent of the overall U.S. population.

Worldwide, an estimated 50 million people have dementia, of which 35 million suffer from Alzheimer’s, and this number will nearly double every 20 years, reaching 75 million people with dementia in 2030 and 131.5 million in 2050.

A majority of people with Alzheimer’s will be in the developing world where nearly 60 percent of people with dementia now live. By 2050 this will rise to nearly 70 percent, with the fastest pace in China and India, the world’s largest populated countries.

What is Alzheimer’s Disease?

Alzheimer’s disease is a progressive dementia. It is characterized by the decline of cognitive function (thinking, understanding, reasoning and learning), memory, communication, and behavioral abilities, ultimately leading to death due to the loss of nerve cells and connections in the brain.

Scientific data suggests that the leading cause of the disease is due to the misfolding and clumping of protein fragments called beta amyloid oligomers, which are toxic and cause neuronal damage and cell death. Their levels build up in nerve cells and can aggregate to form insoluble deposits called amyloid plaques, which can be identified by an image scan and also in post-mortem pathology assessments. Emerging science is showing that, unlike heart disease where coronary plaques block arteries, in Alzheimer’s, the plaques are not toxic; the beta amyloid oligomers are toxic by themselves and create a vicious cycle that kills nerve cells and leads to cognitive decline.

The Cost of Alzheimer’s

Alzheimer’s disease is often thought to be diagnosed late in life and to develop slowly. However, in individuals with a genetic predisposition, the disease can be diagnosed early in a person’s 60s and progress very quickly. Therefore, the economic consequences of the disease are doubly taxing. Since many people must continue to work after 65 years of age to fund their retirement, there is a loss in earning power during key years, as well as the increasing costs of direct care as they lose their own independence or must stop working to care for their spouse or afflicted loved ones.

The average person survives four to six years after a diagnosis. Some people live more than 10 years, but 40 percent of that time is spent in the most severe stage. This makes the cost of Alzheimer’s an economic catastrophe for caregivers and their families. The need for therapeutics, which slow progression to a more severe and costly stage of the disease, particularly for patients at-risk but who are still cognitively fit and living independently, is immense.

According to the Alzheimer’s Association, the yearly cost of care per person ranges from $41,689 to $56,290, which includes nursing home care, out-of-pocket spending, and in-home care.

Nationally, the total direct cost associated with Alzheimer’s is $277 billion, and that does not include unpaid caregiving at an additional estimated cost of $232 billion. These costs are expected to reach $1.2 trillion by 2050 if no drug intervention is developed.

The estimated total global costs of dementia, including direct and informal care costs, were almost $1 trillion in 2016 and growing at approximately 16 percent per year.

On Track for an Effective Treatment

Over the past two decades, scientists have learned a lot about Alzheimer’s – what is causing the disease, who is at risk and when to intervene.

Previous treatments for Alzheimer’s have been focused on trying to stop the build-up of amyloid plaques, because they were thought to kill brain cells. However, large clinical trials have not been successful pursuing this approach, either because of safety concerns or because removing the amyloid plaque didn’t seem to slow the disease progress.

New research from labs around the world are pointing to the real culprit in the disease: toxins called beta amyloid oligomers that can kill nerve cells if they accumulate in the brains of patients. None of the previous therapies were able to inhibit the toxicity of beta amyloid oligomers or block them from forming.

Clinical trials allowed us to fine-tune the therapeutic strategies and focus on the critical issues for Alzheimer’s therapies which are: (1) what toxin must be inhibited or removed to slow the disease progression, (2) does the therapeutic agent effectively enter the brain and engage the target, and (3) what is the optimal study population for confirmation of efficacy.

The failures of clinical trials were related to drug development challenges and continue to support the compelling science of targeting toxic beta amyloid oligomers. We have learned from the failed programs and are applying these lessons towards a more selective approach of addressing the underlying disease process in Alzheimer’s.

Alzheon is Now the Frontrunner in Alzheimer’s Treatment

A number of high-profile failures have been announced within the last year, leaving Alzheon’s drug ALZ-801 as one of the last remaining late stage therapeutic programs with a potential to become a treatment that can slow or stop the course of the disease. The ALZ-801 program has emerged as a frontrunner because of rigorous application of the principles and lessons learned from other programs, and robust support for this novel approach by the basic science as well as clinical data.

Alzheon is poised to deliver the first effective treatment that can slow or stop the course of the disease: a safe oral pill that is designed to protect brain cells by preventing the formation of the disease-causing toxins in the brain, which leads to loss of brain cells and ultimately the onset of symptoms of Alzheimer’s.

For the first time, we have an opportunity to meaningfully slow the progression of the disease with ALZ-801, which prevents: (1) the formation of toxins which drive the disease, and (2) the shrinkage of the hippocampus, the first part of the brain affected by Alzheimer’s, which is responsible for memory formation.

Alzheon scientists have developed a pill that is well tolerated and enters brain at drug levels sufficient to achieve the desired effect. The treatment does not cause side effects like brain swelling or brain hemorrhage associated with some of the other Alzheimer’s medicines being studied.

ALZ-801 program has received Fast Track designation from the U.S. Food and Drug Administration and is ready for a Phase 3 trial in patients who are at high risk for the disease because of their genetic make-up. These are people who will likely develop the disease earlier, when they are otherwise healthy. This is important because the genetic make-up of these patients virtually eliminates the risk of misdiagnosis – which ruined an earlier generation of Alzheimer’s disease trials.

The Phase 3 trial will be an efficient and relatively low-cost study in high risk patient population, applying lessons learned from the precision medicine. We expect to have the study results within three years and the drug could be on the market soon thereafter.

Conclusion

Alzheon’s ALZ-801 program is one of the best opportunities in a generation to solve the problem of bringing an effective treatment to patients with Alzheimer’s. ALZ-801 pill prevents the formation of brain toxins before they build up and cause damage, and the drug has no significant side effects.

An effective treatment can’t come fast enough for the millions of people worldwide who will suffer without it. And it’s a multibillion-dollar opportunity for investors who want to make a difference for patients and societies around the globe.

About Dr. Martin Tolar

Martin Tolar, MD, PhD, is the Founder, President & CEO of Alzheon. An expert in neuroscience and neurodegenerative disorders, for the past 25 years he has practiced medicine, taught at Yale University School of Medicine, held leadership roles at Pfizer and led multiple biotechnology companies. Dr. Tolar serves on many boards, including the Alzheimer’s Foundation and the Alzheimer’s Drug Discovery Foundation. Dr. Tolar was recognized as one of the Top 100 Most Influential People in the world of drug development and manufacture by The Medicine Maker Magazine’s Power Lists for 2016, 2017, 2018 and 2019.

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