"We are undoubtedly slowing down the MND [motor neuron disease] supertanker, and can start to see how we might turn it around for the first tim," said Martin Turner, senior clinician scientist at the University of Oxford (United Kingdom), on research being reported on at the 24th International Symposium on ALS/MND.
The symposium, held Dec. 6-8, 2013, in Milan, Italy, included more than 950 people from 34 countries, who gathered to hear the latest findings from scientific and clinical experts in amyotrophic lateral sclerosis (ALS), often referred to as "motor neuron disease" (MND) in the United Kingdom.
Jane Larkindale, MDA's vice president of research, attended the conference and was encouraged by progress in several areas of therapeutic development. "This was an exciting meeting," she says. "There were nearly a thousand ALS experts in one place, talking about little else than what the barriers are to understanding and treating ALS, and how to overcome those barriers."
Four areas covered at the conference were:
Stanley Appel, a meeting participant from Methodist Neurological Institute in Houston and a longtime MDA research grantee, commented that several of the mechanisms found to underlie ALS appear to converge on misregulation of the immune system, an area of great interest to him.
Among the immune system modulation strategies for ALS now in development are:
Combatting toxic proteins and RNA
Meeting participant Jan Grimm, chief scientific officer at the Swiss company Neurimmune, described how mice that received antibodies (proteins made by the immune system) against a misfolded, toxic protein known to cause ALS showed improvements in gait and the distance they walked, as well as longer survival and fewer abnormal protein clumps in their cells.
Additional strategies being pursued to combat toxic proteins and RNA are:
Replacing or repairing damaged cells
Meeting participant Dimitrios Karussis of Hadassah Medical Center in Jerusalem, who is testing the effects of bone marrow-derived, NurOwn stem cells in ALS patients in a clinical trial, said current trials have shown these cells are apparently safe and well-tolerated. Plans are to increase the dosage.
Among the strategies being pursued to support existing nerve cells or supply new ones are:
Improving muscle function
Investigators testing the drug tirasemtiv, which may increase muscle sensitivity to signals from the nervous system, said they hope to have results in spring 2014.