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Failing Fast: Innovation in the Search for a Cure to Alzheimer’s

December 13, 2016
For those of us who have watched family members suffer the decline that results from Alzheimer’s and other neurodegenerative diseases, the holidays in particular can be a tough time of the year.  They bring back memories sweet and sorrowful—and yet even those memories are a blessing denied to those who no longer share in them.

Today, more than 5 million Americans live with Alzheimer’s disease.  The financial burden on families and on our nation is significant: $200 billion in 2015, out of which $154 billion came from Medicaid and Medicare.  But as those impacted by Alzheimer’s know, the burden on families and communities only begins with the financial costs.  Family members and friends become caretakers, not only forgoing opportunities for work, but also struggling with the emotional trials that accompany this kind of care.

So how do we help?  What relief awaits those suffering from Alzheimer’s?  What respite lies in the shadows for caretakers, yet to be realized?  Currently, dozens of new Alzheimer’s medications and diagnostics are under development.  But in the last 16 years, 123 medicines have been tested and only four of those have been approved.  Four.  That’s about a three percent rate of approval, where getting to an approved drug can take well over $1 billion dollars spent on research, development, and testing.

And while the reason to continue the search for a cure is clear for those affected, it can help to remember the benefits to society, the ways in which we all stand to gain from an effective treatment for Alzheimer’s disease.

In a report produced by the Alzheimer’s Association, we see that with just one new treatment that delays the onset of Alzheimer’s by five years, the number of Alzheimer’s patients would drop by nearly half.  That, in turn, would save more than $367 billion in healthcare costs.  Those are dollars that stay in the pockets of patients and communities, reducing the strains on our health care system.  Imagine, too, how that $367 billion could be unleashed to find cures for other diseases or support education budgets or rebuild our roads and bridges.  For perspective, that $367 billion is more than a third of the president-elect’s plan for infrastructure spending.

So how do we get there?  Innovate.  And then accelerate the innovation.  Perhaps the cures, the effective treatments, will come from those already in the pipeline, awaiting results from drug trials.  But what if we could speed up the part of the process that gets new drugs to the trials?  One approach that a few companies have been experimenting with is developing better animal models for the diseases.

A common refrain in the innovation hub that is Silicon Valley is to “fail fast.”  Animal models for human disease enable medical researchers to do just that. Currently, over 90% of the drug candidates that pass the preclinical, animal testing stage of drug development fail during human clinical trials.  Using genetically accurate animal models of human disease to test new drugs will enable researchers to fail quicker, saving developers hundreds of millions of dollars for each new drug candidate.   Faster failure, then, leads to success sooner.

What is a “genetically accurate animal model of human disease?”  In essence, researchers first identify the gene or genes they think contribute to human diseases.  They then alter the animal’s DNA to mimic the genetic basis of the human disease.  This provides an opportunity not only for improved efficacy and safety testing in preclinical tests, but also for basic research into gene function.

Recombinetics, for example, specializes in developing swine models of human diseases. Why pigs?  Their similarity to humans anatomically, genetically, and physiologically make for better models than other animals. They also develop the disease symptoms much earlier.  The hope is that these advantages will provide useful information to researchers and drug developers as they look to discover, test, and commercialize new Alzheimer’s treatments—or even a cure.  In fact, the National Institutes of Health just awarded BIO member Recombinetics a Small Business Innovative Research grant for precisely this purpose.

Of course, the path to a cure encompasses a broad spectrum of research and development needs and requires continued investment.  Just last week, the 21st Century Cures Act passed with strong bipartisan support in both houses of Congress.  If signed by the president, the act would include funding for the Brain Initiative, a Presidential initiative aimed at revolutionizing our understanding of the human brain.  While this serves as but a part of the ongoing work in this area, it does provide critical support for the tools that will help us better understand Alzheimer’s and other similar diseases.

A couple months ago, I attended an event hosted by a group committed to ending Alzheimer’s.  The organizers brought together drug manufacturers working on treatments, individuals with perspectives from the agencies that buy those drugs, caretakers sharing their stories, and some of the patients themselves, bravely standing up to talk about their experience living with the disease.  The takeaway, for me at least, was clear: to find a solution, we need to keep alive the sense of urgency to find a cure.

Scott Fahrenkrug, the executive chairman of Recombinetics, gets it: “with millions of lives at stake, there’s a critical need to speed up discoveries that lead to better outcomes for patients.”

Innovation saves—saves lives, saves dollars.  With a continued commitment to innovation, we look to fail fast in a race toward successful treatments for challenging diseases.  And along that route, we all share in the benefits of better health and better lives.