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State of the Parkinson's World

Robin A. Elliott, Executive Director

Every so often in our work at PDF, we pause to take stock of just where we are in the march against Parkinson's. My own take: that we are going through a bleak patch but there are signs of better times ahead.

The Money Just a few months from now, we will be marking the fifth anniversary of the initiation of the Parkinson's Disease Research Agenda (PDRA), a bold initiative by the National Institute of Neurological Disorders and Stroke that was designed to focus attention - a "first" for this Institute - on the solution of a specific disease. The NIH-named scientists who put the plan together estimated that exploiting the existing scientific opportunities in Parkinson's would require about $1 billion a year by 2006. Today, the government's own estimate is that NIH spending on Parkinson's research in 2005 will be about $250 million - just one-fourth of what the experts said was needed.

And the picture doesn't look as though it will get better anytime soon. After a succession of record annual 15 percent increases that virtually doubled the NIH budget over five years, the pace has slowed to three percent - barely enough to keep up with inflation. Meanwhile, our re-elected President remains faced with the problem of paying for a hugely expensive war and reconstruction, and it is unlikely that this will leave much in the way of additional resources for medical research and other domestic programs.

The Science

At the time NIH was launching the PDRA, leading scientists were speaking publicly about the possibility that a cure for Parkinson's - or at least some major intervention - would be found in 10 years, perhaps even less. Five years on, the therapeutic scorecard suggests that we are way off track towards this goal. The biggest therapeutic news of the past few years was a new form of Parkinson's surgery - deep brain stimulation - that can have dramatic results for many PWPs but clearly is only appropriate for a small percentage of patients.

Other new releases are even more modest. One is simply an ingenious combination of two existing medications. An-other is a dissolvable version of carbidopa/levodopa, that hoary "gold standard" drug that is approaching its 35th birthday. And a third - an undeniably dramatic and needed "rescue" technique for patients who experience serious "off" periods - was painfully slow in coming to the U.S. market, nine years after it began to be successfully used in Europe.

And that's not all. While the pipeline of new therapies has continued to dribble, some of its best hopes have turned out far short of expectations. Three years ago, two expertly managed and widely watched double-blind studies of neural transplantation using human fetal cells turned up few good results and at least one bad one (un-controlled dyskinesias). And just recently, the Amgen company announced that it was halting human trials of GDNF - a so-called "growth factor" that had been seen as a potential breakthrough in neuroprotection - because of disturbing evidence that it created antibodies (see News In Brief story on page 1).

Frustratingly, this pattern of low output peppered by disappointing reversals is taking place at a time when scientists are reporting unprecedented excitement in the basic science of Parkinson's. Our growing knowledge in a dozen or more areas - for example, how cells die in Parkinson's (at Columbia University and elsewhere); how gene therapy can slow or stop the development of parkinsonism in monkeys (Rush University in Chicago); what might be a candidate for an anti-PD vaccine (Columbia again); which environmental toxins may be implicated in PD (Emory University in Atlanta); and the increasing number of genes (10 and counting) that have been found to play a role in at least some cases of PD - helps build the platform for future advances in understanding and remediating the disease. One measure of this energy is that the number of Parkinson's-related articles appearing in refereed scientific journals has risen by one-third in just three years - from 313 in 2000 to 417 in 2003.

The Challenge What do we conclude from all this? That the anti-Parkinson's movement in America, energized in the 1990s by leaders and celebrities such as Joan Samuelson, Muhammad Ali and Michael J. Fox, is somehow running out of gas? Not at all.

What we need to realize is that there is no single route, no one magic bullet or stock formula, for solving the Parkinson's puzzle. For starters, I count four.

1. Sponsor the Best and Most Imaginative Research

This should include not only time-tested strategies of supporting centers and in-vestigator-initiated grants (such as PDF's long-standing and continuing commitment to supporting research at Columbia University) but also novel, "out-of-the-box" re-thinking of how research themes can be identified and supported. Sharon Begley's provocative recent article in the Wall Street Journal pointed out how private foundations in some disease areas have moved from being passive funders of research into active partners with the scientists; the Michael J. Fox Foundation for Parkinson's Research received a well-deserved mention.

One implication of this statement, if my bleak assessment of government funding opportunities is accurate, is that not-for-profit groups like our own are going to have to improve their ability to marshal private resources. Private funding is likely to be the main area of growth for some years to come.

2. Cultivate Grassroots Communities to Get Them Active in Education and Advocacy

We need to do more to harness the unique energy and passion of patient advocates to keep up the pressure on public agencies to build on the platform of exciting Parkinson's knowledge and re-focus some of their own energy from theoretical understandings to disease solutions. The Parkinson's Action Network (PAN) is doing a great job on this and we need to increase our support for PAN so that it can do more. (Note: the PDF Board of Directors has increased its support of PAN from $100,000 to $150,000 a year.)

3. Find New Ways to Speed Up the Pipeline of New Therapies Development

Traditionally, the voluntary PD organizations have focused on basic research. This is fine so far as it goes - all scientists agree that developments in basic science are key to improved therapies and paths to the cure - but if this is all we do, then we are missing a huge opportunity. Patients and families themselves provide a matchless resource for therapies development. Through Advancing Parkinson's Therapies (APT), a community-wide coalition that is being coordinated by PDF - and that has recently unveiled a state-of-the-art website on the subject, www.PDtrials.org - we hope to explore ways in which this patient community can actively participate in the drug development process, whether by joining trials, or becoming active in the institutional review boards of local medical centers, or becoming a member of "research partners" (a kind of "buddy system" for people who participate in trials.)

Much of the credit for these concepts goes to the Parkinson Pipeline Project, an all-volunteer group of talented and devoted PD activists led by Perry Cohen, a Washington-based social scientist who also serves as a consultant with PDF.

4. Continue to Demonstrate our Strength as a Unified Community

The Parkinson's organizations have come a long way since the mid-1990s, when they were mostly engaged in fighting one another. Some or all of us now meet semi-annually for strategy sessions on Washington policy, convened by PAN; participate in a community fast-track research program led by the Michael J. Fox Foundation for Parkinson's Research; and join together in the magnificent Parkinson's Unity Walk, which gets stronger and more profitable each year. I have already mentioned one important community initiative, the APT project; another is the coalition that is assembling to create a first-ever World Parkinson Congress, in Washington, DC, in late February 2006. (A major feature of this conference will be the presentation of healthcare delivery and "quality of life" initiatives, subjects that are too often ignored in major Parkinson's conferences.) This kind of collaboration is important not just because it feels good (though it does) but because unity is a condition of success with the outside world, whether in DC, or with national news media, or with industry or with the philanthropic community.

Let's wish ourselves a better and more productive 2005 and work like hell to make sure we achieve it.