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Melissa Stevens
Executive Director, Center for Strategic Philanthropy
Health and Medical Research and Philanthropy
Melissa Stevens is the executive director of the Milken Institute's Center for Strategic Philanthropy. The Center for Strategic Philanthropy works to maximize return on philanthropic investment by ensuring that innovation used to address one social issue is translated to another, best practices and metrics guide new and existing giving programs,...
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Giving Smarter to Fight Alzheimer’s Disease

By: Melissa Stevens
May 21, 2015
   
   

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Alzheimer’s disease is quite possibly the greatest health challenge of our time. It erases a person’s biography and is not part of the normal course of aging. It is estimated that 5.3 million Americans and 20 million people worldwide are living with Alzheimer’s. If nothing is done to change the trajectory of the disease, by 2050, nearly 14 million Americans and 100 million people worldwide will develop the disease.

In addition to the widespread devastation this disease will pose for patients and their families, it will have significant economic implications. In 2010, it was estimated that dementia (Alzheimer’s disease is the majority of dementia cases) cost the global economymore than $600 billion, or about 1 percent of global GDP. To put the enormity of this disease into perspective, if dementia were its own country, just based on 2010 data, it would have been the 18thlargest economy in the world – situated between Turkey and Indonesia. If dementia were its own company, it could be the largest in the world by revenue, larger than Apple, Exxon Mobil, or Walmart.

The bottom line is that there is no cure. There are just three approved therapies, which only help to manage symptoms. None of them modify the underlying disease. And for those three approvals, we’ve seen more than 100 failures, some of them very late-stage clinical trials that have had huge opportunity costs in terms of financial resources, time, and patients.

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Identifying barriers and opportunities

Because of this urgency, the Philanthropy Advisory Service undertook an effort to identify the barriers holding us back from developing new treatments, and the opportunities for philanthropy to play a role in getting closer to a cure. With expert input from a scientific advisory board chaired by Jeffrey Cummings of the Cleveland Clinic Lou Ruvo Center for Brain Health and Rudolph Tanzi of Harvard Medical School and Massachusetts General Hospital, we identified opportunities where philanthropy can help to drive the field closer to cures.

Last month, we brought together an all-star set of clinicians, researchers, investors, and philanthropists to discuss the results of this report as well as other opportunities for philanthropy and impact investment to help accelerate our search for Alzheimer’s treatments and cures. In addition to Cummings, our panel included Francis Collins of the National Institutes of Health (NIH), David Dolby of Dolby Family Ventures, and Trish Vradenburg of USAgainstAlzheimer’s.

Taking the risks needed for progress

Strategic philanthropy can play an important role in advancing treatments because this capital is nimble, plays the long game, and has a big appetite for risk. In fact, its willingness to take on risk is perhaps its highest and best use – to take the risk that investors won’t take. Drug development is all about risk and reward. And philanthropy, when allocated strategically, can be extremely catalytic and take risk out of the science and out of the research system. Most likely a new therapy isn’t going to make it to the market funded 100 percent by philanthropy, but it can be used to make it more attractive for other funders like the government, venture capital, or industry to join in.

First, philanthropy can help to de-risk the science. We need to understand more about the underlying biology of the disease. Basic science is typically funded by the NIH; however, the NIH is facing significant funding constraints across the board that is exacerbated in Alzheimer’s disease. It is funding about $600 million in Alzheimer’s disease research, but that’s only one-fifth of what is allocated for HIV and one-third of cancer funding. But since $1 in NIH funding yields $25 or more in follow-on funding, increasing the NIH budget for Alzheimer’s disease research could add significant scientific and economic value to the field.

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For years the field has followed two major scientific theories around amyloid plaques and tau tangles. We need more early-stage research to diversify therapeutic strategies and identify new drug targets. Drug developers typically talk about shots on the goal, but with Alzheimer’s disease, it is more like shots in the dark.

Additionally, because of limited NIH funding, it has never been harder for academic researchers to secure grants. We are at risk of losing a generation of scientists – precious human capital that is needed to gain ground in the battle against this disease.

Second, philanthropy can help to build better research tools that will also de-risk the R&D process. We need better preclinical models to understand what might work in a human - a mouse can only tell us so much. There is an exciting opportunity for philanthropy to support an innovative model – “Alzheimer’s in a Dish.” We can take an individual’s stem cells, grow his or her neurons in a dish, and then test experimental therapies and see if there is any signal, before we would put them in humans. In fact, we could use this technology to test the thousands of already approved compounds (whether they are for cancer or toenail fungus) and see if they have any effect on neurons with Alzheimer’s disease.

We also need tools (biomarkers) that can help us to diagnose patients and understand progression of disease. It’s pretty hard for the Food and Drug Administration to approve a new therapy if we don’t have a good way of knowing if a drug is slowing down, stopping, or reversing the disease. This is particularly challenging with Alzheimer’s disease as we are coming to understand that we need to be intervening very, very early in the onset of the disease. We need more sensitive measurements to understand who has the disease and the impact of new therapies. There are new imaging technologies in development that will allow us to use protein load in brains as biomarkers. There are also companies working on developing online, mobile-enabled technologies to understand cognitive impairment and establish that as a reliable biomarker.

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Third, if we have potentially commercially viable technology, philanthropy can be used tosupport early-stage companies and de-risk their work to make them more attractive for follow-on investors. We’ve seen a paucity of venture capital funding put toward addressing Alzheimer’s disease – only $400 million between 2009 and 2013. (In fact, there was three times as much venture capital funding going to reformulate already approved pain medications than Alzheimer’s disease in that same time period). Philanthropy or impact investing dollars can help to support young companies with innovative technologies (replicate science, help them increase data sets, or conduct “killer” experiments) until they are primed for follow-on investment.

Moving closer to a cure

There are many other ways that philanthropy can help to advance treatments for Alzheimer’s disease: funding research on creating new regulatory pathways, mobilizing patients into clinical trials, engaging the public and raising awareness about the issue, and more.

So there are numerous exciting, transformative, and meaningful ways that strategic philanthropy can play a role in moving us closer to cures for Alzheimer’s and dementia. These diseases in particular are very personal. We will all be touched if a loved one is diagnosed or because we will feel the economic ramifications of this disease. Philanthropy likely won’t develop a therapy on its own, but we will surely get there faster with it.


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