Date: November 28, 2001
Subject: Pediatric Brain Foundation’s (formerly CNS Foundation) 1st Annual Scientific Advisory Board Think Tank
Location: Boston, MA
- Mark D. Noble, University of Rochester
- Jeffrey D. Macklis; Professor of Stem Cell and Regenerative Biology, Harvard University, and Professor of Neurology [Neuroscience] and of Surgery [Neurosurgery], Harvard Medical School; Department of Stem Cell and Regenerative Biology, Center for Brain Science, and Harvard Stem Cell Institute, Harvard University
Attendees: Parents of children affected with neurological disorders and members of the Pediatric Brain Foundation Scientific Advisory Board, including Mark Noble, Evan Snyder, Steve Goldman, Jeff Macklis, Angelo Vescovi, and Harley Kornblum.
Recap: Meeting via teleconference in November, the parents and scientists of the Pediatric Brain Foundation debated different strategies to move the science forward. Members agreed that a significant way the foundation can accelerate biomedical discovery and speed the translation of scientific insights into effective treatments and cures is to orchestrate collaboration among investigators. It became clear that by helping coordinate individual scientific efforts, the roadblocks to progress could be more readily overcome.
Pediatric Brain Foundation FINDS ITS RHYTHM AS RESEARCHERS DISCUSS WAYS TO MOVE FORWARD
By: Science Journalist, Mary Jane Friedrich
Co-Principal Investigators: Mark D. Noble, University of Rochester &
Jeffrey D. Macklis, Harvard Medical School
It started off simply, like the first notes of a song. But in no time, a harmony emerged as the members of the Pediatric Brain Foundation began to discuss ways to harness the power of scientific knowledge to improve the lives of children with neurological disabilities. The music sounded new and fresh, as if a novel arrangement of an old score was being played for the first time.
Meeting via teleconference in November, the parents and scientists of the Pediatric Brain Foundation debated different strategies to move the science forward. Members agreed that a significant way the foundation can accelerate biomedical discovery and speed the translation of scientific insights into effective treatments and cures is to orchestrate collaboration among investigators. It became clear that by helping coordinate individual scientific efforts, the roadblocks to progress could be more readily overcome.
Excitement about collaboration and enthusiasm for helping children were palpable among the members of the scientific advisory board (SAB). Describing his own reasons for working with Pediatric Brain Foundation, Mark David Noble, Ph.D., professor of genetics at the University of Rochester School of Medicine, NY, expressed the commitment shared by all Pediatric Brain Foundation scientific advisors, “I’m committed to Pediatric Brain Foundation because this is why I do science, to have an impact on medical problems.”
Another appeal of the new foundation, said SAB co-chair Evan Snyder, M.D., Ph.D., assistant professor of neurology at Harvard Medical School, MA, is “that working to correct neurological problems of childhood can literally change an entire lifetime, making a difference for the next 70 or so years for someone.”
During a roundtable discussion, SAB participants discussed different approaches the foundation should follow to make strides in understanding and correcting the neurological problems that beset approximately 15 million children in the United States.
FUNDAMENTAL ISSUES AND HOW TO ADDRESS THEM
Kicking off the dialogue, Noble suggested that SAB members collaborate on resolving a fundamental question in the field by looking at one particular model of a childhood neurological disorder. He explained, for example, that one of the big problems facing neuroscience researchers interested in using the emerging information of stem cell biology in the design of new therapies is the fact that they do not know what stage of differentiation a cell should be in to achieve the best result in repair of central nervous system damage.
Researchers currently are studying between five and seven different types of precursor cells to understand their potential to achieve repair, he said, but little is known about the comparative utility of different precursor cells in any repair setting. These different cells may actually play different roles in repair; the problem is that no one has systematically examined this question.
Noble suggested that SAB members could do just that: pool their resources and expertise to methodically determine what type of precursor cell would be most effective in repairing a specific neurological defect.
This suggestion raised the question of whether the foundation should direct its efforts in this way toward a particular disease or research priority. Since this approach is followed by the National Institutes of Health and large-disease oriented foundations, a small organization such as Pediatric Brain Foundation might do better to fill in general gaps in basic science knowledge that are not currently being addressed in research, said Steve Goldman, M.D., Ph.D., Professor of Neurology and Neuroscience at Cornell University Medical College, NY.
Goldman explained that such information gaps slow down the translation of basic science. Research in these areas is not always well funded, particularly if the questions are not defined in terms of their relevance in disease. The Pediatric Brain Foundation, he said, might be able to abrogate these knowledge gaps by funding research groups that study important basic science questions with an eye toward understanding how these processes operate in disease.
This approach would leverage the funding being made available by other foundations and other sources in the most efficacious manner, said Goldman.
Jeff Macklis, M.D., Ph.D., associate professor of neurology at Harvard Medical School and SAB co-chair, suggested that one concrete way of addressing the issue of how best to facilitate the exchange of knowledge would be for Pediatric Brain Foundation to fund a postdoctoral employee who could actually “float” among the laboratories working on a collaborative project. “I think the collaborative inter-lab approach may be a unique way to facilitate things and make them go more quickly,” he said.
OF MICE AND MEN
SAB members also discussed the need to keep an open mind about funding projects that use stem cells of various species. At the same time, it is important to recognize that what is discovered in the mouse system may not always translate directly to human systems, said Angelo Vescovi, Ph.D., co-director of the Institute for Stem Cell Research, Milan.
In some cases, however, using mouse cells and mouse model systems is the best way to explore a question, explained Macklis. For some neurological problems, mouse models contribute “dramatically” to the eventual use of human cells; in other disease models, human cells may be closer to therapeutic application. “Deciding which species system to use will depend on the questions being asked,” added Snyder. “As is the case with any grant application process, applicants for Pediatric Brain Foundation funding will need to justify their technique, model, cell type, molecule, etc.,” Snyder advised. “If there is a reasonable rationale for using one system over another, “we’ll respect that.”
COLLABORATION AS CATALYST
Harley Kornblum, M.D., PhD., assistant professor of pediatrics at UCLA School of Medicine, CA, reprised an earlier refrain that SAB members identify a particular neurological problem on which to collaborate. Once a problem is agreed on, members could pool their talents to determine the fundamental scientific questions that need to be answered in order to close in on a clinical solution.
Pediatric Brain Foundation might fund such a project in its first year, setting the tone for future projects the organization would support.
Hashing out these fundamental issues in neuroscience, remarked Snyder, will be important not only for the foundation but for the field in general.
It is clear that Pediatric Brain Foundation will need to consider a diversity of approaches as it establishes its scientific priorities. On one point, however, there was unanimous agreement the opportunities for Pediatric Brain Foundation to play a significant role in encouraging and fostering research on developmental brain disorders are limited only by its financial resources.
Emphasizing the commitment exhibited by all members, Noble remarked, “I think it’s a privilege to take part in these kinds of processes, a privilege to work with the people on the board, and it’s a responsibility we have to try to figure out ways to do something that’s different for children with neurological disabilities.”
The meeting drew to a close on this harmonious note, as strains of a new and exciting melody lingered in the air, inspiring plans for future meetings, endeavors, and achievements.”