Nervous System Diseases Program

The HSCI Nervous System Diseases Program focuses on neurodegenerative and traumatic diseases of the brain and spinal cord. In particular this collaborative, inter-institutional research program studies amyotrophic lateral sclerosis, often referred to as "Lou Gehrig's Disease”; spinal cord injury; Alzheimer’s disease; narcolepsy, and--by extension--corticobasal degeneration; Huntington's disease; and Parkinson’s disease. The aim is to understand exactly which neurons degenerate and why, and how to interfere with the degenerative process to provide a therapy.

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Key Research Questions

The HSCI Nervous System Diseases Program is pursuing cell-based and chemical-based approaches that answer the following questions:

  • Are adult stem cells present in the nervous system and can we identify the molecules and genes that control the differentiation of these cells?
  • How can the differentiation of adult progenitors and/or embryonic stem cells be directed into the specific types of neurons that can replace diseased or injured cells?
  • Are there chemicals that could direct the differentiation of desired cells among the many types of neurons, and chemicals that could be used therapeutically in neurodegenerative diseases?

Key Scientific Results

Scientific findings from the HSCI Nervous System Diseases Program include:

  • The derivation of ALS disease-specific human embryonic stem cell lines; discovery of a hyper excitability phenotype in these and repurposing of an anti-epileptic agent for testing a clinical trial on ALS patients. 
  • The identification of compounds, through chemical screening, that: activate oligodendrocyte progenitors important in multiple sclerosis and cerebral palsy; and promote survival of amyotrophic lateral sclerosis embryonic stem cell-derived motor neurons.
  • The identification of several different pathways involved in spinal muscular atrophy that regulate the levels of the protein defective in the disease.
  • The identification of small molecule signaling modulators that effectively turn mouse embryonic stem cells into dopaminergic neuron progenitor cells (important in Parkinson’s disease) and into medium spiny neuron progenitor cells (affected in Huntington’s disease).
  • The generation of pain neurons from human fibroblasts embryonic stem cells as a platform for discovering new pain medications and exploring the mechanisms of and treatments for peripheral neuropathy.