Timeline: Our Key Moments in ALS

• Our ALS program launches. The overarching goal? Develop ALS therapies.
• We investigate the potential of insulin-like growth factor I for slowing death of motor neurons (those involved in ALS).

• We test noninvasive ventilation to help ALS patients breathe with greater ease.

• We test another candidate, vascular endothelial growth factor, which slows the degeneration of motor neurons.

• We launch preclinical studies of stem cells as a potential ALS therapy, testing feasibility in animal models.

• We report on our phase I trial of stem cells in ALS, our first findings in humans, which deemed the stem cells safe. Plans are set in motion for a phase II trial.
• We initiate studies of the so-called “epigenome” in ALS, that is, regulatory elements that control the expression of genes. In this instance, we look at DNA methylation.

• We conduct an imaging study of ALS patients revealing changes in neurotransmitters (molecules that aid nerve conduction).

We launch our first investigations into the impact of environmental pollutants on ALS risk. We find that pesticides and fertilizers are linked to ALS onset.

• We discover that insulin-like growth factor I expression enhances the potential of stem cells as an ALS therapeutic.

A busy year!

• We publish findings of our phase II trial of stem cells for ALS. Again, our results find the treatment is well-tolerated.
• We embark on our inquires into the involvement of the immune system on ALS status.
• We expand our studies on environmental pollutants in ALS, finding a link between persistent organic pollutants and disease risk.
• We extend our study of the ALS “epigenome” by investigating regulatory elements called microRNAs.

• We discover that distinct types of immune cells correlate with ALS progression.

• We publish a perspective piece in JAMA Neurology advocating more research on the impact of environmental pollutants on ALS risk.
• We initiate our studies of the ALS metabolome in patient plasma, that is, the cumulative of all metabolites. This is linked to biological processes occurring in ALS.
• We find that early exposure to specific metals is linked to ALS risk.

• We publish a perspective piece in JAMA Neurology advocating more research on the impact of environmental pollutants on ALS risk.
• We initiate our studies of the ALS metabolome in patient plasma, that is, the cumulative of all metabolites. This is linked to biological processes occurring in ALS.
• We find that early exposure to specific metals is linked to ALS risk.

• We launch preclinical studies of an immune-targeting drug, tofacitinib, as a potential ALS therapeutic.
• We report the first metabolome replication study in ALS. Lipid pathways are recurrent.
• We are invited to write two reviews on recent advances in ALS in the top neurology journal, The Lancet Neurology.