At the annual ALS Association ALS Drug Company Working Group, held at the American Academy of Neurology (AAN) Meeting in Vancouver, British Columbia, ALS researchers from academia and industry convened to discuss avenues for advancing ALS research and therapy development. This year’s presenters included the 2016 recipient of the Sheila Essey Award for ALS Research, Ammar Al-Chalabi, Professor of Neurology and Complex Disease Genetics at King’s College London (see April 2016 news), and representatives of Cytokinetics, Biogen and Origen Data Sciences. Al-Chalabi discussed the contribution of genetics to ALS, reflecting on how knowledge of ALS pathophysiology has dramatically changed over the past two decades, and expressed hope that advances in genetics will facilitate therapy development.
A detailed discussion of two ongoing clinical trials in ALS followed, with presentations by Jinsy Andrews, Senior Director of Clinical Research and Development and Head of Neuromuscular Therapeutics of Cytokinetics and Elizabeth McNeil from Biogen. Last July, Cytokinetics began its VITALITY-ALS Phase III clinical trial, which is testing efficacy of tirasemtiv vs. placebo on respiratory function in ALS (see July 2015 news). Biogen’s Elizabeth McNeil presented the Methodology Study of Novel Outcome Measures to Assess Progression of ALS, a clinical trial aimed at identifying more precise and sensitive outcome measures of disease progression than the ALS Functional Rating Scale (ALSFRS) for use in phase II trials (see Feb 2016 news). Last to present was Origent Data Sciences’ Chief Scientific Officer David Ennist, who discussed the importance of developing computational approaches to predict disease progression in ALS patients, both for the individual patients and for clinical trials. In a clinical trial setting, these methods can enable more sensitive assessment of the impact of drug interventions (a webinar on the program is available here), and Origent will now begin testing these algorithms in collaboration with Cytokinetics (see March 2016 news) .
For more details of the Drug Company Working Group meeting, read the press release here.