McMaster University

Demystifying Medicine
Seminar Series

Clinical Trials for ALS

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This video overviews treatments for amyotrophic lateral sclerosis (ALS) and examines the latest clinical trials being conducted. The two main focuses of ALS clinical testing revolves around using stem cell and gene therapies.

— Video by Tanzim Hoque, Inderpreet Gill, Nikhita Stoimenov and Tawfiq Hasan

References

  • Chio, A., Canosa, A., Gallo, S., Cammarosano, S., Moglia, C., Fuda, G., ... & Mora, G. (2011). ALS clinical trials Do enrolled patients accurately represent the ALS population? Neurology, WNL-0b013e318232ab9b.
  • Dimos, J. T., Rodolfa, K. T., Niakan, K. K., Weisenthal, L. M., Mitsumoto, H., Chung, W., ... & Wichterle, H. (2008). Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons. Science, 321(5893), 1218-1221.
  • Kern, R. Z., & Gothelf, Y. D. (2018, May 25). Safety and Efficacy of Repeated Administrations of NurOwn in ALS Patients. ClinicalTrials.gov. Retrieved November 22, 2018.
  • Moss, A. H., Oppenheimer, E. A., Casey, P., Cazzolli, P. A., Roos, R. P., Stocking, C. B., & Siegler, M. (1996). Patients with amyotrophic lateral sclerosis receiving long-term mechanical ventilation: advance care planning and outcomes. Chest, 110(1), 249-255.
  • Oskarsson, B., Gendron, T. F., & Staff, N. P. (2018, July). Amyotrophic lateral sclerosis: an update for 2018. In Mayo Clinic Proceedings. Elsevier.
  • Petrov, D., Mansfield, C., Moussy, A., & Hermine, O. (2017). ALS clinical trials review: 20 years of failure. Are we any closer to registering a new treatment? Frontiers in Aging Neuroscience, 9, 68.
  • Rothstein, J. D. (2003). Of mice and men: reconciling preclinical ALS mouse studies and human clinical trials. Annals of Neurology: Official Journal of the American Neurological Association and the Child Neurology Society, 53(4), 423-426.
  • Yang, Y. M., Gupta, S. K., Kim, K. J., Powers, B. E., Cerqueira, A., Wainger, B. J., ... & Arvanites, A. C. (2013). A small molecule screen in stem-cell-derived motor neurons identifies a kinase inhibitor as a candidate therapeutic for ALS. Cell stem cell, 12(6), 713-726.