SECTION: CAPITOL HILL HEARING TESTIMONY

LENGTH: 1933 words

COMMITTEE: SENATE APPROPRIATIONS

SUBCOMMITTEE: LABOR, HEALTH AND HUMAN SERVICES, EDUCATION

HEADLINE: PARKINSON'S DISEASE

TESTIMONY-BY: MICHAEL J. FOX, FOUNDER

AFFILIATION: THE MICHAEL J. FOX FOUNDATION FOR PARKINSON'S RESEARCH

BODY:
Statement of Michael J. Fox Founder The Michael J. Fox Foundation for Parkinson's Research

Subcommittee on Labor, Health and Human Services, Education Committee on Senate Appropriations

Parkinson's Disease

May 22, 2002

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 it is a tribute to you that 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 indeed all Americans, should be grateful for your leadership on issues of health, including your continuing efforts to make biomedical research a higher nation priority. This Subcommittee - along with other individuals in this room - have spearheaded a historic effort that will soon succeed in doubling the budget for the National Institutes of Health over five years. You have accomplished this feat through difficult budget times, through changing administrations, and even through changes in majority control - an achievement that 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. None of us here has any interest in becoming another of this city's self-perpetuating cottage industries. Our appeals to you are part of our larger effort to accelerate the cure for Parkinson's disease. Much progress is being made, but there is no question that a well-funded and coordinated effort by the federal government would hasten the pace. And as you have already heard, time lost to Parkinson's inevitably means that lives are lost as well.

You've also heard from Dr. Isacson about the wide array of promising research opportunities relating to Parkinson's. He and dozens of other senior investigators make clear the inevitability of a breakthrough. Taken together, their message is unmistakable: curing Parkinson's is not a question of "if." It is a question of "when."

Back in 1999, I testified that Parkinson's research was far ahead of the money. Joan, Dr. Bill Langston, and I all testified 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, we saw there was more we could do ourselves. With their help and advice, and together with a group of like-minded people, 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 lay people 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 toward the 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 in keeping with the higher-risk, higher-reward nature of our mission we've streamlined the NIH model and added our own wrinkles.

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 the development of a cell line specifically for the study and treatment of Parkinson's.

One month after the meeting that made the recommendation we launched a $2.2 million initiative to develop cell lines with characteristics deemed relevant to Parkinson's. 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 allowed 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 - 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 good 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 there is a paradigm shift underway. Understanding of the brain and of neurological disorders has advancing at a rapid pace, moving from definition of the disease to treatment to the possibility of repairing the brain and restore lost function.

Not too long ago it was an anathema to think that the brain has any capacity to regenerate and repair itself. But in recent year many scientists have embraced this recently revolutionary concept and run with it. Dr. Isacson, for example, is so convinced in the potential of the science that he has named his lab the "Neuroregeneration Laboratory." NIH recognizes this shift and has taken some appropriate steps to respond. Nonetheless, they still trail behind the scientists out on the cutting edge - those whose experience increases our confidence that a cure is within reach. Our own experience shows the type of efficient funding process that is possible and the level of interest there is in doing the necessary work. All of this is tremendous progress, but it also increases impatience and sense of frustration over what is NOT getting done.

To meet the opportunity, I encourage the new NIH Director to fill open NINDS Director position, and to do so with someone committed to using all available tools - including the Director?s discretionary budget authority - in the FY02 and FY03 budgets 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.

I want the Subcommittee to know that we in the private sector hope to engage in greater collaboration with NIH when tapping Parkinson's researchers for advisory and planning meetings. The goal is to reduce the number and replication of meetings and allow more time for the best scientists to work in their labs.

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, I might add.

The other thing given to every person diagnosed - particularly the growing number of young-onset cases - is a reason to hope.

We are told that scientists are making great progress and that with the proper funding there may be a cure in five or ten years. We hear that there is no shortage of good ideas, just 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, support 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 have got to 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 challenge. We ought to act as surely as we act in response to other challenges to our health, our lives, and our society.

I would not take this coveted time before the Subcommittee to argue for something than 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 "tipping point," the moment of critical mass when the momentum towards the cure becomes irresistible and the only remaining question is whether the federal government will be helping lead the process, or will it be trailing along behind'

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. And we're 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.

Thank you.



LOAD-DATE: May 29, 2002