Parkinson’s and the President: How Does the $100 Million BRAIN Initiative Announcement Impact our Community?

Dr. Francis Collins, Director of the National Institutes of Health (NIH), introduced him as the "Scientist in Chief."  And he introduced himself as "the kid who had trouble with high-school physics."  But there was no mistaking the passion for his subject of the man who stood in front of us in the East Room of the White House this morning, announcing a major federal initiative in brain science.

It was President Obama at his rhetorical best, mixing easy banter with a deeply serious expression of his commitment to the brain research initiative that he described as the "next great American project."

When he described the pathos of someone watching "a beloved family member slip behind the mask of Parkinson's disease," you could practically feel a shudder of recognition among the normally restrained scientists, government officials and advocacy group leaders like myself who packed the room.  Perhaps "Empathizer in Chief" is more to the point; the guy really seems to mean it.

So what does this mean for Parkinson's?  We won't know for sure at least until the President delivers his budget message to Congress next Wednesday, and possibly not even then.  But the overall strategy of the BRAIN Initiative is clear: to use the resources of the federal government and private sectors to find out more than we know now about how the brain works – specifically, how the various parts connect with one another, and in what patterns.

All of this could have exciting implications for finding the solution to Parkinson's disease.

As Dr. Eric Kandel, the Columbia University scientist who won the Nobel Prize for his work on memory, said to me at a reception following the event, "this is an historic moment!"

We'll see.  There are several big questions to be answered, beginning with money:

• The President is calling for initial spending of $100 million in the year that begins October 1, 2013, but it is far from clear how much of this will be new money and how much will be existing funds repackaged for the new initiative.
• Then there's the matter of who spends it and how between the NIH, the Defense Advanced Research Projects Agency and the National Science Foundation.  And there will be others.
• Lastly, how will patient advocates be involved? PDF certainly supports this initiative’s collaborative nature, utilizing federal research agencies and private scientific organizations.  We urge the President to expand this commitment to include patient advocates as we have done through the Parkinson’s Advocates in Research program.  After all, the people affected each day by Parkinson's disease are critical to our goals of finding better treatments at a faster pace.

But the overall picture is encouraging -- even exciting. This the first time that the President, after years of slogging through the thickets of the nation's fiscal crisis looking for an exit, has made a major statement about medical research, comparing the challenge -- and the potential for its success -- with the moon shot of the 1960s, the development of the computer chip in the 1970s, and the exploration of the human genome of the 1990s.

The common thread through all of these triumphs was what he described as the American "genius for innovation," an outgrowth of a national character of "dreamers and risk-takers."

"How can we afford to do this?" he asked.  Then he answered himself: "How can we afford not to?"

To learn more see PDF's official statement on our website here.

Notes from a Day Spent Listening to Scientists Judging PDF Research Applications

Last Friday, March 16, was the occasion for reviewing applications for PDF’s International Research Grants program and Research Fellowships programs. Before us were some 30 proposals from some of the best young (and not-so-young) scientific investigators in the world.

As I sat there in the meeting as an observer, listening to the members of our scientific review committee as they made their comments and pronounced their judgments, I found myself scribbling notes on what seemed to be the principles on which they were basing their decisions. What they were saying, it seemed to me, said a lot about how we go about choosing the projects we will fund. Here are some of the ideas I picked up:

Relevance to Parkinson’s Disease. Every proposal that we fund – without exception -- must show promise in its potential to advance our understanding of PD, or charting the path to its cure.

New Ideas, New Investigators. To maximize the leverage of the program, successful applications will be one of two kinds. One is the innovative pilot project that shows promise for leading to a larger-scale endeavor that will be eligible for funding from the NIH (drawing on PDF’s “leverage” function). The other is the Parkinson’s-related application from an exemplary scientist whose past contributions have been largely in areas other than Parkinson’s – and who could be inspired by means of the PDF grant to turn his or her attention to PD.

Demonstrating Results. The renewal of an earlier award to the same scientist depends on the investigator’s ability to show “significant progress” since the first grant – that is, you don’t get a second grant if you can’t show that you used the first grant well.

Establishing Credibility. To make it through the grant review process, good ideas aren’t enough; the applicant needs to be able to demonstrate – both in his/her personal accomplishments and in the reputation of the lab in which the work will be done – a stellar track record in producing innovative and useful science.

Including Advocates in the Research Process. The meeting included three members of Parkinson’s Advocates in Research (PAIR), a PDF program in which lay leaders in the PD community are prepared to take on a variety of roles advocating for clinical research. An example of their contribution came early in the day, when one of these advocates raised a question about the validity of animal models in Parkinson’s research, which generated a spirited exchange among the scientist members of the committee.

I hope you are as impressed as I am with this little vignette of PDF’s research culture!

I conclude with a quotation from a memo on the mission of the program from Dr. James Beck, who is our Director of Research Programs at PDF and staffs the grants review committee. In thanking the scientists in the room, he said, in effect: “The two groups that will benefit most from your decisions today are not even here. One is the world of talented young scientists whose work will be made possible through your efforts. The other is the community of almost one million in the U.S. who live with Parkinson’s. In behalf of these two communities, PDF thanks you!”

Amen, James.

NINDS and Translational Research

For years now, many of us at the Parkinson’s Disease Foundation (PDF) and other Parkinson’s disease (PD) organizations have fretted that the brain research funded by the National Institutes of Health (NIH) has been too focused upon the basic processes of neuroscience and too little upon the next stage of research: finding clues to potential new therapies for specific conditions like Parkinson’s.

This next stage of scientific investigation is often called “translational research,” because it is here that the molecules and compounds identified as interesting in the laboratory are “translated” into animal studies to determine their therapeutic potential. Those compounds that make it through this phase may then go on to be tested in human trials, and from there – the hope is – to approved treatments.

So it was with delight that I learned at a recent NIH-sponsored meeting that from now on, university centers that are selected for five-year grants for Parkinson’s research under the prestigious Morris K. Udall program must show a commitment to translational research. The news was announced at a session arranged for representatives of PDF and other PD organizations by Story Landis, Ph.D., Director of the National Institute for Neurological Disorders and Stroke (NINDS), the entity that provides more than $20 million a year to support the 14 Udall centers in the program. She said that the Udall centers would henceforth be moving towards including a more translational focus to their work, and that those academic centers that cannot make the switch will need to seek sources of other support, such as the so-called program project grants that NIH offers.

Specifically, in the language of the request for applications recently issued by NIH, “Responsive applications will demonstrate proven ability or considerable potential to pursue rapid translation of research to clinical practice.” Applicants that are not able to demonstrate this translational component – however strong they may be on the basic science – will simply not be eligible for designation as Udall Centers.

Another encouraging note sounded at our meeting was by Robert Finkelstein, Ph.D., who directs the institute’s extramural grants program that includes the Udall Centers. He reminded us that the new focus on translational research reflects the development of the science of Parkinson’s. He observed that 15 years ago, NIH would not have had so much opportunity to focus on translational proposals because the underlying basic science was simply not there to support them. He also said that he welcomed this new direction because “we at the NINDS have the mission to cure Parkinson’s!”

The reformulation of the Udall Centers is not the only good sign of a stronger commitment to translational science at the NIH these days. Another is the appointment of two dynamic new research administrators to fill the new positions of Director of Translational Research (Bill Matthew, Ph.D. ) and Director of Clinical Research (Petra Kaufman, M.D., M.Sc.).

Dr. Matthew has a strong background in industry -- most recently, as leader of the Partnering and Business Development at UCB, an international biopharmaceutical company based in Brussels. His charge at NINDS will be heading up efforts to translate the results of laboratory research into treatments for neurological disorders.

Dr. Kaufman, who is considered to be among the nation’s leading experts in the design and management of clinical trials for neuromuscular disorders, has served for a decade as an associate professor of neurology at the Neurological Institute at Columbia University.

Another promising development with big implications for translational research at the NIH as a whole is the appointment of the new Director, Francis Collins, M.D., Ph.D.. In his first statement about his dreams for the NIH, he included as one of his five top priorities, “translating basic science into new treatments.”

The times are changing, and in some good directions. At PDF, we will be working with our colleague organizations to make sure that these good times continue to roll.