The AD Challenge: Hitting the Right Mark

by Jeongyeon Shim, FasterCures, Domestic Program Analyst

Last week, I attended "Early Detection of Alzheimer’s Disease: A Virtual Town Hall*,"organized by the Alzheimer Research Forum and the Alzheimer’s Study Group, sponsored by the Geoffrey Beene Foundation Alzheimer’s Initiative. Moderated by Dr. Harold Varmus, the meeting featured presentations from seven eminent Alzheimer’s researchers, highlighting the importance of an antecedent biomarker and providing an update of ongoing efforts, using media such as imaging, blood, or cerebrospinal fluid.

While the importance of biomarkers is well known, the importance of reliable and low-cost antecedent biomarkers for Alzheimer’s disease (AD) is paramount and should be appreciated in the context of the disease. First, AD is an irreversible neurodegenerative disease—once clinical symptoms appear, the neurons that govern the affected functions are permanently lost. This means that even if we had a cure that addresses the cause of the disease, it will not necessarily revive the lost functions for later-stage patients—unless we also find a way of regenerating lost neurons. Early treatment through early detection is not only ideal but critical in reducing the burden of the disease—preventing the clinical form of the disease.

In addition to its limitation in detecting clinical symptoms, the current AD diagnostics are not always accurate nor available. AD diagnosis based on clinical symptoms has an accuracy of over 90 percent when conducted by experienced clinicians. Unfortunately, this is often not the case for mild AD, when distinction with mild cognitive impairment (which does not progress to dementia) may not be as obvious. And, "experienced clinicians" are a scarce resource. Instead, primary care physicians, thrust on the frontlines of AD care, may not necessarily have the tools and training to recognize AD. With these factors, it is more important than ever to develop a reliable and widely-available diagnostic tool

We discussed Dr. Lee Goldstein’s effort to develop an eye exam to diagnose AD a few weeks ago. And last week, I was pleased to see that scientists are pushing forward multiple other efforts. Identification of multiple markers will allow us to choose a marker that best fits the purpose—whether it is for diagnosis, management of risk factors, or measurement treatment effectiveness. Such efforts exactly match FasterCures’ goal—to accelerate the process of discovery and clinical development of new therapies. We look forward to see many more such efforts, not only in AD but also in other diseases.
*The proceedings from the Town Hall will be made available at www.AlzStudyGroup.org in the near future.



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When It Comes to Nonprofits, Does Location Really Matter?

by Jeongyeon Shim, Domestic Program Analyst, FasterCures

Recently, Charity Navigator published its sixth annual Metro Market Study. The study ranks 30 different metropolitan areas based on the financial performance of nonprofit organizations in each area. Based on the aggregate performance of nonprofits for each metropolitan area, the areas are ranked according to CEO compensation, level of financial reserves, volume of funds raised, and other Charity Navigator financial metrics.

Some nonprofit experts and concerned individuals have highlighted, in various venues, the limitations of Charity Navigator’s focus on financial performance in evaluating nonprofit organizations. Financial metrics based on data from tax forms have the advantage of being available for most organizations, but they provide limited insight into an organization’s actual impact or potential. For this reason, FasterCures’ Philanthropy Advisory Service (PAS) program focuses on the qualitative aspects of nonprofit disease research organizations, including their strategies, management policies, governance structures, and results-to-date.

I am concerned that the Metro Market Study’s focus on financial metrics may provide an unbalanced view of the performance of nonprofit organizations and wonder about the consequences of prioritizing geographic location above all other considerations. What insight does this singular focus on location convey?

Some nonprofit organizations, especially those serving local communities, operate on a local level, and for those organizations, location does affect both their missions and financial performance. However, there are also a sizeable number of organizations whose missions are to serve on a national or even global level. Therefore, the importance of location is different across the types and missions of the organizations—which does not seem to be taken into account in the Metro Market Study.

Fundamentally, I question the value of using location as the first criteria to analyze the financial performance or concluding that charities operate according to the general practices of their metropolitan areas. For example, the focus of FasterCures’ PAS program is on nonprofit organizations engaged in medical research and none of the organizations to be studied aim to develop a treatment only for the residents of the community where they are located, rather they aim to find cures for all. This is true not just for the mission of the organizations, but also for its financial aspects. For example, all of the five organizations ranked as raising the most funds through special events, as reported by Charity Navigator’s Special Event Study, conduct their fundraising events across the country, rather than just in the community where they are headquartered.

Another concern I’d like to raise is the ultimate value of the information produced through this study. Should a nonprofit organization based in Detroit, the metropolitan area with the lowest overall score (Charity Navigator’s measure for overall financial health), move to Miami, which has the highest overall score, and would its financial situation improve (or can it even move given its mission)? Probably not. The same goes for donors. Should the donors make their giving decision based on the location of the headquarters or take the location as a sign of financial health? And how will this help donors interested in giving to nonprofit organizations based in their own community?

A comprehensive analysis of other factors, such as the organization’s area of focus, strategy, process, and governance structure, are probably needed and more important than headquarter location in guiding giving decisions and determining the drivers of financial health.

Charity Navigator is one of the largest databases on nonprofit organizations with a wide scope of coverage. These advantages can be used to understand the key drivers of growth and performance in nonprofit organizations and conduct meta-analyses to understand larger trends. Its analysis of the nonprofit marketplace should focus on variables that really matter—I felt the Metro Market Study fell short of living up to its potential.

NIH Asks How to Create a More Effective Clinical Trials System

The National Institutes of Health recently held a town hall meeting to brief patient advocacy groups and other external constituencies about its initiative to remove barriers to more efficient conduct of clinical trials at the Clinical Center on the NIH campus. The Clinical Center is really a national jewel, the largest hospital in the world dedicated exclusively to research (and therefore a major sponsor of clinical trials). It has almost 2000 active protocols in which tens of thousands of patients participate with the goal of advancing our understanding of disease and, one hopes, developing more effective treatments.

But even though the Clinical Center has spectacular resources compared to many clinical trials sites around the country -- starting with its beautiful new building and state-of-the-art lab space in Bethesda -- it experiences all the same frustrations with the process as everyone else. Regulatory burdens increasing. Inconsistent policies and practices among Institutional Review Boards -- even within NIH there is no uniformity, and different Institutes and Centers handle things differently! Flat research funding. Declining numbers of clinical researchers. It hopes through its study of the problems on its own campus to inform the debate outside its walls, in the extramural research community. The goal of the NIH's review is to share best practices in conducting trials across the institutes and centers within NIH, and to identify over-interpretation of applicable regulations. Officials who spoke at the event seemed confident that many of the barriers were not externally imposed but were rather due to differing interpretations of regulations and the sheer volume of changing requirements, with which everyone finds it hard to keep up.

The NIH's Intramural Research Program -- the research conducted on its campus by NIH-employed scientists -- of which the Clinical Center is a part, is a little recognized critical resource in our taxpayer-funded research portfolio. At its best it provides a model of cutting-edge biomedical research for the extramural, mostly academic research world, taking on the highest-impact science and suggesting solutions to systemic challenges. We hope that this effort to plow through the thicket of complaints and paperwork that surround clinical research and clinical trials will be such a model for the rest of us.

-- Kristin Schneeman, Program Director, FasterCures