Nervous System Diseases Program

Program Leaders: Paola Arlotta, Ph.D., Golub Family Professor of Stem Cell and Regenerative Biology, Harvard Department of Stem Cell and Regenerative Biology; and Clifford Woolf, M.B., B.Ch., Ph.D., Director, F.M. Kirby Neurobiology Center and Neurobiology Program, and Professor of Neurology and Neurobiology, Harvard Medical School

Scientists in the HSCI Nervous System Diseases Program investigate neurodegenerative and traumatic diseases of the brain and spinal cord. Their goal is to understand exactly which neurons degenerate and why, and how to interfere with the degenerative process to provide a therapy.

Researchers in this collaborative, inter-institutional research program study the underlying biology of many diseases, including:

  • Amyotrophic Lateral Sclerosis (ALS)
  • autism
  • Alzheimer’s disease
  • corticobasal degeneration
  • hearing loss
  • Huntington's disease
  • narcolepsy
  • pain
  • Parkinson’s disease
  • retinal degeneration
  • spinal cord injury

How we approach our work

HSCI researchers use many different approaches to discover which stem cell populations are present in the nervous system and, if so, to understand what drives them to differentiate and maintain the breadth of neuronal cell types.

One approach is the cultivation of “organoids” – cells in a dish that hold are valuable tools for modeling human neurological disease in new ways.

These tools allow scientists to explore how stem cell differentiation can be harnessed to develop neurons, which could be used to replace diseased or injured cells. They also facilitate the search for small molecules that could be useful in new therapies, for example by directing stem cells to differentiate into ‘desirable’ neurons.

What we have achieved so far

Researchers in the HSCI Nervous System Diseases Program have:

  • Used in vitro models of ALS patient derived neurons to identify drugs and have brought a former anti-epileptic agent to the clinical trial stage for ALS treatment.
  • Identified compounds that activate glial cells - cells that are directly important in multiple sclerosis and cerebral palsy and that promote the survival of other neuronal types, such as motor neurons.
  • Identified several different pathways that regulate levels of a protein that is defective in spinal muscular atrophy patients.
  • Generated pain neurons to serve as a platform for discovering new pain medications and explore the mechanisms of and treatments for peripheral neuropathy.
  • Found small molecules that effectively turn embryonic stem cells into dopaminergic neuron progenitor cells and into medium spiny neuron progenitor cells – cells that are critical in Parkinson’s disease and Huntington’s disease.
  • Developed new methods to create three dimensional, “organoid”, based models of complex tissues such as the brain.  This, in turn, will allow the study of conditions and diseases which have been poorly understood to date.

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