It Takes Planning

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by, Ken Aidekman

Parkinson's advocates and community members who helped pass the Morris K. Udall Act in 1997 were spurred on by the goal of increasing Federal funding for research from $25 million to $100 million per year. The National Institutes of Health (NIH), the Department of Defense and the Veterans Administration and other government agencies now spend more than $200 million dollars per year on Parkinson's related research. At this level of funding, research planning and allocation have become more critical to the effort to find a cure.

Before the Udall Act, few advocates and administrators at NIH spent much time contemplating the plan and infrastructure necessary to produce significant results. While higher levels of funding are always desirable, it is appropriate to take stock of our current situation and ask important questions. Are we making the best use of the financial, physical and human resources available to find a cure? Is our research adequately coordinated? Where have we found success? Where have we come up short? How can we apply our collective body of experience to improve PD research in the future?

The agenda of the Fifth Annual Meeting of the Morris K. Udall Centers of Excellence for Parkinson's Research allotted a time and format to explore these difficult questions and also raise additional issues that effect progress in finding a cure. On the second day of the conference, Diane Murphy of the National Institute for Neurological Diseases and Stroke (NINDS) mediated a "town meeting" to solicit comments on progress and recommendations for future areas of concentration. Robin Elliot, of the Parkinson 's Disease Foundation suggested that NINDS along with lay scientific writers draft a document to tell the public what we've learned in the past five years since the Udall Center initiative began.

After only two weeks on the job as Scientific Director of NINDS, Story Landis was faced with the task of reviewing the overall performance of the Udall Center program. She expressed an understanding of the need for an assessment and welcomed the opportunity. At the same time she acknowledged the legitimate concerns scientists and administrators have with "top-down" planning. She referred to a recent article in the New Yorker magazine titled "The Thirty Years War" which relates a number of negative consequences of excessive research management of research at the National Cancer Institute. She reminded attendees that while NINDS focuses on prioritizing lines of research and fostering an atmosphere of cooperation, ultimately, it is still committed to funding the very best scientists in the field.

NIH Director, Elias Zerhouni, has made it clear to NINDS staff that it must continuously develop and update a hierarchy of PD research community goals. Under his guidance NINDS established a matrix to evaluate each individual research goal on the basis of chances for success and expected timeframe for completion. This matrix can be found at www.ninds.nih.gov/parkinsonsweb/matrix.asp. It provides an excellent overview of the standards used to decide upon one area of research over another. The priorities laid out in the matrix are not set in stone but they do provide perspective on where NINDS sees the greatest potential reward for each level of risk. It was designed to be a living agenda that is subject to regular re-evaluation by NINDS staff and the PD community.

On the same page as the matrix is a link to the Parkinson's Disease Research Community Goals - FY2003. This document summarizes the results of serious dialogue between scientists, NINDS staff and volunteer organizations on how best to proceed with research, set investigative standards and maximize efficient use of resources. It lays out an action list that includes initiatives on sharing animal models, brain banks, genetic information and clinical data. It also addresses roadblocks to research such as complex patent issues on compounds and processes and an acute scarcity of investigators in the field that threatens progress.

The best scientists are generally independent and creative people. They do not respond well to having to follow a strict research protocol prepared by a government agency. That said, NINDS has made collaboration between researchers a hallmark of the Udall Centers program. So, collaboration became the number one theme running throughout the presentations at the 2003 annual meeting. Each presenter gave evidence of collaboration with other Udall Center scientists outside their own facility. It was highly apparent that showing an effort to collaborate is an essential requirement for future grant approval.

With so much emphasis on collaboration at the meeting one might wonder whether it is truly essential for success in curing PD or more of a reflexive response to popular pressure. The answer lies with future results, but, for now, it is clear that as research data expands dramatically in both volume and complexity the need for cooperation on data sets between state of the art Udall centers increases. This is especially true in the fields of genetics, protein analysis and large scale clinical reporting. At best, the emphasis on collaboration will enhance productivity, promote shared standards and facilitate communication making possible speedier discoveries and more frequent breakthroughs.

Is there an instance we can point to in which Udall Center interaction has been positive? Ole Isacson at the Mclean Institute cited an example of cooperation that has helped him in his work. As a cell biologist and expert in the transplantation of foreign dopamine cells it is important for him to study results from related protocols. Because of NINDS-led cooperation Isacson has access to brain tissue samples received from every Udall center that has been involved in transplants. It is unlikely he could have benefited from that kind of access without improved cooperation between Udall Centers.

Whether one is working on PD susceptibility genes with micro arrays or high throughput screening of potentially neuroprotective drugs, researchers today are swamped with massive amounts of data. By entering collected information into a properly devised minimum data set, researchers will be able to "mine" that data more effectively now and for years to come. With this in mind NINDS is soliciting proposals from organizations to create a new Data Coordinating Center for the Udall Centers. Genetics labs are at the forefront of working with sophisticated bioinformatics software to cull through mounds of data but Story Landis stressed that NINDS and NIH are seriously looking at the need for new computational tools to handle the increasing load across the board.

NINDS is also actively influencing the direction of PD research by encouraging Udall Centers to set up a clinical component if they do not have one in place. They have increased the cap on funding per institution from $1 million to $1.5 million for centers with approved clinics. While most of the centers do incorporate clinics, this action stresses the importance NINDS places on the need for clinical trials and translational efforts. Even before this recent action, Udall Centers had been attracting clinical work and research from other disciplines within their parent institutions.

For example, Jeff Vance at Duke has seen his Neurogenetics Department staff grow to nineteen people and begin to link up with a clinical department. They are now able to reach out for more DNA blood samples and have had success in increasing the size of their brain bank. Researchers tend to move more easily between genetics and cell biology now because the Neurogenetics Department has a higher profile as a Udall Center. Ted Dawson at Johns Hopkins told of a similar experience. His department has seen an increase in brain donations, the creation of a new clinical component and a growing storehouse of patient information that includes data on behavioral assessments and psychiatric manifestations. The increased integration of fields of studies is having a multiplier effect on attracting assets.

This is only the second year that members of volunteer groups have been invited to the Udall Annual Meeting. Is there any reason for us to participate? Is there anything that PWP's and non-scientists do to push along the process other than supply more money? The answer to both of these questions is "YES".

Some scientists in Parkinson's research have little or no contact with people with PD. Some researchers, and even neurologists, are surprised to learn that when it comes to quality of life motor symptoms like tremor are not necessarily the greatest obstacle. Laura Marsh, a psychiatrist at Johns Hopkins, mentioned that depression has been found to be the most frequent complaint by PWP's in terms of quality of life. What does this suggest to researchers and funders? For one thing, it's time to reassess priorities and give greater recognition to alleviating debilitating non-motor symptoms with available treatment at the same time dramatic cutting edge technology is used to find a cure.

Two days with Udall Center researchers, NINDS staff and other attendees at the Udall Annual Meeting is more than enough time to establish that the Parkinson's community is getting a major commitment to excellence from the professionals involved. So what is left to do for advocates and members of volunteer organizations? It turns out that there are a number of issues that require attention.

Researchers discussed the growing burden of having to satisfy the requirements of regional institutional review boards (IRBs). IRBs were established to protect patients from risky investigational procedures and to protect their personal privacy when it comes to medical information. This can be a serious issue when it comes to studying genetics. If a genetic predisposition to debilitation disease is found in an individual it is understandable that they would not want that information to get into the hands of their employer or medical insurance company.

But, excess paperwork and needless questions can also put a strain on researchers and subjects alike. Consent forms that used to be one or two pages now go on much longer. If the bureaucratic element of a research trial becomes too great it can scare away subjects or make a project too expensive to be practical. In addition IRBs have nearly complete autonomy in which to conduct their business and they can vary greatly from one region to the next.

Scientists are asking for help in this area. They are suggesting that advocates recognize the issue and perhaps even apply to become members of IRBs in order to present their viewpoint. Hopefully, they can push for simpler consent forms and greater uniformity between IRBs nationwide. Representatives from NINDS also mentioned their willingness to intervene on behalf of scientists when they need help with IRBs.

All in all, attending the Udall Centers annual meeting was very encouraging from an advocate's perspective. It's good to hear that NIH, and NINDS in particular, are operating under standards, which call for continuing re-assessment of priorities and progress in the curing of disease. Another indicator of the state of goal-directed research is the fact that NINDS is increasingly willing to partner and coordinate with the other institutes of NIH when it comes to fighting Parkinson's. As Story Landis said at the conference, "Diseases do not respect institute boundaries." Fortunately, the Udall Center team is finding it easier to cross those boundaries with some help from their friends in Washington.


Ken Aidekman
Board Member, The Parkinson Alliance

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