Progresso em Pesquisa de ELA

Entering 2006 with Renewed Hope: Progress in ALS Research

Roberta Friedman, Ph.D., ALSA Research Department Information Coordinator
The turning of the year brings with it a renewal of hope for progress toward successful treatment for ALS, with numerous major advances during the past twelve months. In particular, 2005 saw the launch of a new initiative that is already bringing lab advances into the clinical arena.
Through its new TREAT ALS initiative, Translational Research Advancing Therapy for ALS, The ALS Association (ALSA) implemented a drug discovery program and clinical trials process that accelerates discovery and testing of candidate clinical drugs for amyotrophic lateral sclerosis (ALS, also called Lou Gehrig’s disease). Two new grants go toward testing innovative therapeutic strategies in ALS patients. A third grant allows investigators to devise a lab test to show whether a candidate treatment about to enter clinical trials is affecting the targeted aspect of the disease process.
In the latter half of 2005, important publications included progress toward candidate biomarkers. These chemical signatures in the body could reveal early signs of ALS and might allow investigators to objectively monitor the progress of the disease to better test investigational treatments. An impressive list of publications this year (www.alsa.org/research/article.cfm?id=866) attests to a better knowledge of how the disease works and ways to target it, for instance, through new ALS models. A lab worm now can show changes due to a mutant protein that is linked to some inherited forms of the disease and should yield therapeutic insights into sporadic ALS as well. A biotech partnership will now screen for new drugs using this worm; meanwhile, this past year saw progress toward yet another model for the disease, the zebrafish.
A stem cell workshop in September brought together experts in a unique push toward effective stem cell therapy for ALS. Discussions at Banbury Center, Cold Spring Harbor Laboratory in New York centered on both the basic biology of stem cells and why the nervous system falters when trying to fix itself. By bringing together different disciplines and introducing so many noted experts to the challenge of ALS, the workshop brought into focus the reality that all the different aspects of replenishing motor neurons need to be carefully understood for design of a successful therapy.
The possible cognitive changes associated with ALS and implications for families trying to cope were addressed by researchers from around the world who convened in May in London, Canada for a workshop on frontotemporal dementia. The varied symptoms of ALS can include motor and cognitive changes, which may merely reflect where damage is being done in the brain and spinal cord, the scientists concluded. Further research into the issue will undoubtedly help steer counseling and new treatment options.
A better knowledge of the disease’s biology is bringing about a shift in viewing the disease process that will aid the effective design of clinical trials in ALS, a theme re-emphasized during the December International Symposium on ALS/MND in Dublin (see www.alsa.org/research/article.cfm?id=864 for more info).
This year also provided a new means for ALS patients and people who care about them to individually further progress toward effective new therapies. In partnership with the National Institutes of Health, ALSA has worked to implement the NINDS ALS repository (see http://www.alsa.org/news/article.cfm?id=867), a collection, storage and distribution system for patient samples that will lead to new discoveries into the cause and treatment of ALS. To uncover yet-unknown gene changes responsible for ALS, researchers need blood samples and some basic health-related information from patients and from people who do not have ALS. Samples to provide DNA that can be stored indefinitely, along with relevant clinical information, will allow distribution from a single location to ALS investigators while maintaining donor anonymity.
Only in a few cases is there a known reason for a person having ALS. Research to identify other responsible genetic factors, made possible through this repository, will lead to a better picture. Those with the disease now can contribute toward that brighter future.
“This year has certainly been a productive one,” said Lucie Bruijn, Ph.D., ALSA science director and vice president, “with many exciting new collaborations between academia and industry and the initiation of our new project, TREAT ALS, to complement our existing efforts and to bring ideas from the lab into clinical applications. It gives us real hope for the new year.”