Testimony of Michael J. Fox Founder, The Michael J. Fox Foundation for Parkinson's Research Mr. Chairman, Senator Specter, and members of the Subcommittee, thank you for this opportunity to testify. Is it just me, or were you sitting in different seats the last time I was here? I apologize if that is a sore subject for some members of the Subcommittee, but I bring it up only to make the point that is a tribute to each of you. Irrespective of the musical chairs of electoral politics, and who sits in which seats on the dais, this Subcommittee's commitment to biomedical research funding remains constant. Mr. Chairman, all Iowans, and all Americans, should be grateful for your leadership on issues of health, including your continuing efforts to make biomedical research a higher national priority. You and Senator Specter have spearheaded the historic effort that will soon succeed in doubling the budget for the National Institutes of Health over five years. The fact that you've done so despite difficult budgetary times, changing administrations, and changes in majority control underscores the notion that illness and injury truly are non-partisan issues needing bipartisan solutions. I am grateful for the Subcommittee's invitation, but I would not have come back again if I did not feel I have something constructive to add. Although we appreciate this forum, none of us here today has any interest in becoming another of this city's self-perpetuating cottage industries. Back in 1999, I testified that Parkinson's research was far ahead of the money, that high quality and high-impact projects were being slowed down or stalled completely by the lack of available support. In addition to appealing to you and the NIH, it was clear that there was more we could do ourselves. It didn't take long to find a group of like-minded people, and together we launched a foundation with the single purpose of stimulating and supporting research, strategic thinking, and collaborations to accelerate the cure for Parkinson's. We are committed to enabling the work of scientists, and to do so we focus on the process of identifying, funding, and tracking research. We try to target where we can have the biggest impact and to employ the best methods to shorten the funding cycle, share the outcomes of research, and stimulate a coordinated effort to translate promising findings into a cure. Some of our programs support investigator-initiated grants ? the bread and butter of the NIH system and an indispensable mechanism for supporting new ideas ? but with our emphasis on higher-risk, higher-reward projects we've streamlined the NIH model and applied some of our own innovative thinking. We've also enlisted scientists to identify the highest-priority areas of research and recommend proactive steps we can take to move the field forward in meaningful ways. Such assessments have lead to several specific funding initiatives, including a search for a conclusive diagnostic test ? or biomarker ? for Parkinson's and the development of a cell line specifically for the study and treatment of Parkinson's. Last fall we launched a $2.2 million initiative to develop cell lines with characteristics deemed relevant to Parkinson's. As with our other programs, the scientific response was overwhelming. We received applications from a veritable who's who of cellular biologists worldwide. The number and quality of the proposals compelled us to double the program's budget to $4.4 million, which made it possible for us to support a portfolio of projects exploring all the promising techniques for creating cell lines from adult, fetal and embryonic cells. The number and diversity of these programs will allow for a meaningful comparison of these exciting technologies. In our request for applications we made it clear that we valued results over technique or cell source. Our program is to develop an effective tool to study and treat Parkinson's, not to support new technologies for their own sake or to pick favorites among emerging therapies - as any patient will tell you, that their favorite therapy is the one that works. This is an obvious and logical approach if your goal is to cure a disease, but often the political debate can lead to arbitrary decisions or otherwise obscure the fact that the goal of the research is to treat, heal, and cure. I want to commend you Senators Harkin and Specter, along with Senators Feinstein, Hatch and all your other bipartisan colleagues for supporting the "Human Cloning Prohibition Act of 2002," which strikes the necessary balance between development of potentially life-saving research and inappropriate applications of this powerful technology. It is important to make clear that the debate is not about promoting one type of research over others, it is about protecting researchers from being demonized or criminalized so they can go about their work exploring new opportunities to treat illness and disease. Development of such promising new therapies puts us on the threshold of a new era of medicine. Today a neurologist may be able to do little more than tell you the name of the disease that's taking away your life, or in some cases he or she may be able to give you a prescription or two to ease the symptoms for a few years. It's not a great proposition; but fortunately there is a paradigm shift underway. Understanding of the brain and of neurological disorders is advancing at a staggering pace, moving from definition of the disease to treatment to the possibility of repairing the brain and restoring lost function. The time has come where the brain is no longer just a place for research; it is a place for cures. The NIH recognizes this shift and has taken some steps to respond. Unfortunately, vacant leadership positions have prevented the bold action we need. Our foundation has succeeded thus far mostly by tapping into the enormous backlog of promising yet unfunded science. We didn't create this opportunity, we're simply responding to it with whatever resources we can muster. As exciting and gratifying as it is, seeing the possibilities only increases our impatience and sense of frustration over what is NOT getting done. NIH has the resources and the infrastructure to do so much more. To meet the opportunity, I encourage the new NIH Director to immediately fill the open NINDS Director position, and to do so with someone committed to using all available tools ? including the Director's discretionary budget authority ? to direct significantly more funding toward implementation of PD Research Agenda. I believe the NIH should responsibly pursue all available regenerative therapies for Parkinson's and other diseases, and adopt an aggressive, proactive "Bunsen burner to bedside" approach to pursuing cures, not just research. When I first appeared before this Subcommittee I spoke about my experience with Parkinson's disease. I did so in very personal terms because that is what I know. I know my own Parkinson's, which is different than Muhammad's, or Joan's, or Don's, or Milly Kondracke's. Every person who is diagnosed with Parkinson's is given their own custom version of the disease ? and no operating instructions or "OFF" switch, I might add. The other thing given to every person diagnosed ? particularly the growing number of young-onset cases ? is a reason to hope, to believe that their disease can be turned off forever. We are told time and time again that scientists are making great progress and that with the proper funding there may be a cure in five or ten years. We've heard, and indeed witnessed for ourselves, that there is no shortage of meritorious science. Far from it, there is simply a shortage of research money. More recently we have been told that more money is on the way. The heroic efforts of the grassroots advocacy community are having an impact and Congress is taking steps to ensure more Parkinson's research funding. Congress passed Parkinson's-specific legislation, asked NIH to develop a Parkinson's Research Agenda, and last year adopted strong report language urging more funding and full implementation of the research agenda. These have each been significant accomplishments, and we are all grateful to this Subcommittee and your colleagues in the Senate and House for your support. And yet despite these legislative achievements, funding of Parkinson's research has failed to keep pace with the overall growth in NIH's budget, it has not meet the goals of the NIH Research Agenda, and it falls far short of the scientific opportunity. Is it that this system is not designed to systematically and aggressively study, treat, and cure a disease? If that's true, we must reinvent the system. NIH has a vital roll funding basic research and supporting scientific explorations. But when there are opportunities to reduce human suffering and societal costs by curing a disease like Parkinson's, then I think it is appropriate for the National Institutes of Health to commit a fraction of it's resources to actually treating the nation's health. Parkinson's disease is both an individual and national crisis. We ought to act as surely as we act in response to other threats to our health, our lives, and our society. I would not take this coveted time before the Subcommittee to argue for something that cannot be done. Our experience is evidence that it can. And we'll keep at it, because unlike some other reports you may have heard, we have yet to determine any shortage of interest in Parkinson's research or in high-quality, high-impact projects that await funding. Don't let anyone tell you that everything that can be done is being done or that the scientific community has reached its capacity for Parkinson's research. What's more, I believe we are at the moment in time critical mass when the cure is inevitable. I know of a recently published book that provides and insightful and inspirational description of this phenomenon. It is one of those books that you can't put down, and once finished, you find yourself turning to again and again. Perhaps you've heard of it. It's called The Tipping Point (How Little Things Can Make the Difference), by Malcolm Gladwell. Parkinson's has reached its tipping point. My question is: "Will the federal government help lead the process?" In describing our efforts we often make analogies to great achievements like the "Moon Shot". But I am here to tell you that administering a successful research program is not rocket science. It is mostly common sense and the will to get things done. Mr. Chairman, we are going to get this done. This Subcommittee, the Congress, and the NIH have the opportunity to make it happen in time for many more people living today with Parkinson's disease. Thank you.