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The Era of "Change": New Pathways for Research in Parkinson's

By Robin Elliott

The sense of change that began with the US elections of last November has grown as the new Administration wrestles with the most serious economic crisis in almost a century.  Part of this change is a growing acceptance of the necessary role of government in resolving the current crisis and setting the stage for a stable and hopeful future.  What does this change mean for medical research, and specifically, for our hopes for new therapies for Par­kinson’s disease (PD)?

Strengthening Funding for Research

First, some good news: Congress has voted additional funding, albeit temporary, for medical research.  The economic stimulus plan initiated by President Obama will pour $10 billion into the National Institutes of Health (NIH) — a whopping one-third of the agency’s annual budget.  After several years of flat funding, this initiative will be a welcome boost for all areas of research — Parkinson’s included.

The money, which must be spent over two years so as to maximize its impact on the economy, will enable NIH to fund new researchers, supplement funding for existing researchers, and invite new requests for support.

The problem with a short-term stimulus of this kind is that its benefits are likely to be temporary.  Moreover, the stimulus itself will not benefit the private pharmaceutical industry, which traditionally provides the next step of action in the treatment development process.  At a recent Board meeting of the American Society for Experimental Neurotherapeutics (ASENT), of which I am a member, a venture capitalist drew groans from the audience when he predicted that by the end of 2009, one-half of all the biotechnology firms in the United States will be out of business. 

The Challenge of Translational Research

To many, the underlying challenge with Parkinson’s disease is not about money alone, but about bridging the gap between the discoveries of the research lab and the products that appear in the neighborhood pharmacy.  This gap, often referred to as “the valley of death,” reflects the fact that many discoveries found promising in early stages of research are not explored further, but are instead left on laboratory shelves.   

Bridging this gap is the challenge of a research stage often referred to as “translational.”  For Parkinson’s, unfortunately, the challenge of translational research is especially tough, in part because the brain is by far the most complex and difficult to access organ of the body.  In addition, the National Institute of Neurological Disorders and Stroke (NINDS), the government agency that addresses PD — unlike the institutes that address such disease areas as cancer or heart disease — has a portfolio of more than 600 diseases to worry about, just one of which is Parkinson’s disease.

Seeing Signs of Change at the NIH 

Certainly, there are some hopeful signs of change and new attention being paid to the importance of translational research, beginning with the NIH itself.  At the recent annual Forum of the Parkinson’s Action Network (PAN), the public policy arm of the PD community, Story Landis, Ph.D., Director of NINDS, observed that “five years ago, we used to think that the business of translational research would be done by the pharmaceutical industry … but this didn’t happen!”  She acknowledged that NIH in the past had paid insufficient attention to the research initiatives on specific diseases like PD, but that this has begun to change. 

One reflection of this change of attitude is to be found in the strategic planning process of NINDS itself.  The strengthening of translational research at NINDS is one of the main objectives of this process, and it is being led by Howard J. Federoff, M.D., Ph.D., a prominent neuroscientist who currently serves as Executive Vice President for Health Sciences for Georgetown University and Executive Dean of the University’s School of Medicine.  

Signs of change are also apparent within the structure of NINDS.  Dr. Landis is creating an Office of Translational Research, charged with identifying promising ideas that come out of basic research and providing the funding to take them to the next phase.  She is currently searching for a permanent director for this new office.

Lastly, we see signs of NINDS’ commitment to translational and clinical research by looking at its very own funding.  One example is the agency’s support of the same Dr. Federoff mentioned above, who will soon be undertaking an early-stage human trial of GDNF, a so-called “growth factor” that was put on the shelf in 2004 following a failed clinical trial.  Dr. Federoff has told PDF News & Review that his new project, using a gene therapy technique, will apply lessons learned from the earlier trial — and those from another, more recent, failed trial of neurturin, another growth factor. 

Another interesting angle on the role of the NIH in translational research appears in the form of a provocative report recently released by Faster Cures, a Washington-based think-tank.  The report calls attention to the part of NIH that is known as the “Intramural Program,” in which research is performed by basic and clinical researchers who work within the NIH itself.  Among other things, the report calls on NIH to improve translational research by making better use of the impressive but under-used research hospital system based at its Bethesda, MD campus.  (The GDNF trial mentioned above will in fact be led from the NIH campus.)

Improving Our Understanding of How Parkinson’s Disease Progresses … and Searching for Biomarkers 

It is well known that Parkinson’s develops differently in every person, but to date there exist no scientific mechanisms to measure the pace or direction of this development.  These measures, called biomarkers — usually chemical tests or imaging procedures — are used to track the di­re­c­tion and pace at which a disease develops, and can take years off the process of clinical trials.

One initiative that may have application to biomarkers development is LABS-PD, a long-term project led by scientists from the Parkinson Study Group and supported by PDF, involving potentially thousands of people with Parkinson’s.  The uniqueness of the project lies in the manner in which the participants are selected — namely, as “alumni” of earlier clinical trials, all of whom have been carefully characterized and have signed up to continue in the service of PD. (See the "Spotlight on Research" article from this issue to learn more about LABS-PD.)

Another initiative is being led by the newly-created Coalition Against Major Diseases (CAMD), an outgrowth of Critical Path, a project of the US Food and Drug Administration.  CAMD brings together industry leaders with government regulators, clinical trial specialists and patient-advocacy groups with the aim of improving the process upon which the decision to approve new treatments is made.  The initial diseases to be studied are Alzheimer’s disease (AD) and Parkinson’s, and there will be three major projects: creating new systems of electronic data, including medical records, to accelerate data-sharing across trials; developing bio­markers to help diagnose and track disease progression; and understanding the natural history of how AD and PD develop, to aid in creating new approaches to clinical trials design.  PDF, along with the Parkinson’s Action Network (PAN), are founding members of this coalition. 

The Way Forward

Certainly the general mood in the Parkinson’s community is more sober today than it was, say, a decade ago when the pipeline of treatments development was bulging, and budgets at the NIH and in the biotechnology industry were rising sharply.  But there is room for hope, in new attitudes and initiatives at the National Institutes of Health, and in other pathways.  The work goes on, and PDF will be doing all it can to contribute to it.