Howard Fillit, MD: Funding Clinical Trials for Alzheimer's Disease

DECEMBER 10, 2017
Matt Hoffman
Howard Fillit, MD, the founding executive director and chief scientific officer of the Alzheimer's Drug Discovery Foundation, sat with MD Magazine to discuss the struggles faced by Alzheimer's Disease therapies when it comes to funding.

Fillit discussed the role the Alzheimer's Drug Discovery Foundation plays in helping these ventures get off the ground, as many of them, despite having potentially huge clinical implications, are often risky and expensive to fund.

Howard Fillit, MD, the founding executive director and chief scientific officer of the Alzheimer's Drug Discovery Foundation:
The pharmaceutical companies have big dollars. They can come in phase 3—one phase 3 trial in Alzheimer's costs $300 to $400 million, and you are going to need 2 or 3 of them, maybe 4, to get FDA approval. Biogen is risking up to $3 billion on their aducanumab at this point. That is a big risk. You cannot take many of those bets without going out of business, and a lot of big pharma companies have left not just Alzheimer's, but the whole CNS (Central Nervous System) space because it is so risky. You cannot run a company with such large risks, financial and scientific.

Same thing with venture capital. They have 10-year funds. Well, it takes 12 to 15 years to develop a drug. So they are tending to step in late. They want to see, generally speaking, phase 2 data. There are some VC funds that will step in earlier into the preclinical space, but most of them won't. Again, for the same reason. They have obligations to their investors to make money, and that is a good thing because if a drug is brought to market, everybody wins. But where they can put their money is in a different place than the pipeline.

With venture philanthropy—the role of philanthropy in our society is to take risks, and that's what we (the Alzheimer's Drug Discovery Foundation) do. We are in the space of drug discovery and development, but we're the ones who can take risks.

If somebody comes to me like Hank Kung, who came to me to get this amyloid scan going back in 2000—no one would give him any money. I funded that thing for 4 years at the University of Pennsylvania. Now it is a revolutionary technology. Back then, he could not get any money. So we are doing this all the time. I have funded over 95 biotechnology companies and programs in the last 18 years. There are a lot of companies that are out there now that have really interesting drugs that are in development, and they are having trouble getting investors, so we fund them, and NIH provides some funding to them.

It is not a great way to run a company—to do it on grants—but it is the role of the foundation. We have a really good due diligence, and we are known for that. We are non-profit, so we do not really have any bias (we hope). We know the investor community and we can fund these things. We make investments, these are investments that the foundation is making, and we can move programs forward into the clinic.  Hopefully, with our money—through the Valley of Death—companies can survive to a point where they can pivot and get pharmaceutical deals or venture capital investments, and pull it all the way through to phase 3. 

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