Feb 04, 2009
Promise: 2010 Nervous System Repair Teams Meet, Plan Next Steps Toward Restoring Nerve Function in People with MS
Over 70 members of the four international teams funded through the National MS Society’s Promise: 2010 Campaign met in New York City in January to share progress, forge new collaborations, and plan future steps to speed efforts toward clinical trials of therapies to protect and reverse neurological damage in people with multiple sclerosis.
With the Society’s pledged funding of $15.6 million for five years, the teams have been conducting a broad range of studies aimed at protecting and reversing neurological damage – from basic molecular studies to planning clinical trials.
Leading these four teams are Peter A. Calabresi, MD (Johns Hopkins University), Charles ffrench-Constant, PhD, FRCP (University of Edinburgh and University of Cambridge, UK), Gavin Giovannoni, MBBCh, PhD (Queen Mary University of London, UK) and Ian D. Duncan, BVMS, PhD, FRCPath (University of Wisconsin Madison).
Each team reported impressive progress, and clinical trials of potential nerve-protecting agents are already underway.
Read details of the progress reported at the meeting in the “Research Now” section of the Society’s Momentum magazine (PDF)
Searching for New Targets and Therapies: The search for repair strategies begins with efforts to find the therapeutic targets that end up under study in clinical trials in people with MS. The teams reported exciting search results related to their use of advanced tools to identify molecules that facilitate myelin repair or protect nerve integrity. Several candidates have been identified and are ready to be taken to the next stage of clinical development.
Progress was also reported in identifying cell populations that may be key to strategies involving transplanting cells to stimulate repair. In addition, team members discussed strategies for testing the potential of existing drugs that may be re-purposed for MS.
Tracking Success: Finding noninvasive methods of pinpointing nerve fiber damage – and repair – is crucial to preventing disease progression and for determining whether future repair strategies are working. Teams are testing many types of novel imaging techniques, and are also identifying “biomarkers,” such as the presence of specific proteins in the blood or spinal fluid, that would provide a snapshot of a person’s disease status and the health of their tissues.
Clinical Trials: The ultimate goal of the Society’s repair initiative is to translate these findings into clinical trials of agents that can rebuild damaged tissues in people with MS and/or protect against further injury. The teams are in the process of creating new clinical trial designs that will allow the use of fewer participants and permit faster determinations about the possible benefits of new therapies.
Ancillary to the repair initiative, Dr. Giovannoni reported progress on two ongoing studies focused on neuroprotection in which his team is participating. One involves lamotrigine, a drug used in epilepsy, which is being tested in a phase 2 study to see if it can slow brain tissue loss in people with secondary-progressive MS. Results are expected to be available this year. The other is a large multicenter study is investigating whether the active compound in cannabis, THC (Tetrahydrocannabinol, which has shown benefit in pre-clinical studies), can slow MS progression. Results are expected in late 2011.
In addition, two of the teams are about to launch new, small-scale clinical trials, also with separate funding. One will investigate the safety of treatment with bone marrow (“mesenchymal”) stem cells; the other will attempt injections of neural stem cells. (Both trials are fully enrolled and awaiting further funding and approvals.) It is not clear yet whether either of these cell types will actually serve as replacement cells to restore brain tissue. Research in animal models suggests that these cells may create an environment that stimulates repair by resident cells. More research, which is ongoing, will help bring clarity to these and other questions.
As Dr. Giovannoni commented, “We’re still learning how to do [cell therapy] trials, but we’ve got to start somewhere, and we will learn by doing them, even if their results are initially negative.” Team members discussed further steps that could speed efforts toward clinical trials of repair strategies and the need for guidelines for future trials that will result in the most informative outcomes.
“We’re more than halfway there,” said John Richert, MD, Executive Vice President of the National MS Society’s Research & Clinical Programs Department in his address to workshop participants. “Thanks in large part to your work, we now have tremendous leads for strategies to protect people with MS from nervous system damage and to even reverse that damage.”
Read background information about the Nervous System Repair Initiative
Read about other Society-funded researchers focusing on tissue repair in MS