Nerve and Muscle (ALS)

The Challenge

Amyotrophic lateral sclerosis (ALS), or Lou Gherig’s Disease, is one of a group of incurable and fatal neurodegenerative diseases. ALS occurs in 1 of 100,000 people, or about 300 new cases per year in San Diego County.

  • The cause and basic mechanism of neurodegeneration are not understood, with 7 or more competing hypotheses.
  • There is no useful treatment, only supportive care.
  • Diagnosis is difficult as there are no biomarkers, only clinical and electrophysiological criteria.

The Innovation

We plan to leverage highly developed neuroscience research in dementia, basic science, stem cell therapy, and neuro-imaging, as well as other areas such as genomics, to accelerate development of a Nerve and Muscle Research and Treatment Program.

Initially, this program will concentrate on motor neuron diseases such as ALS to begin with. The breadth, depth, and strength of the UCSD Department of Neurosciences, when paired with a strong clinical program, will create a rare multidisciplinary research and treatment program.

Research Opportunities

  • Study the mechanisms of neuronal degeneration in human cells using techniques currently being studied in animal models.
  • Discover biomarkers, e.g. proteomics, to help understand disease mechanisms, to use in early diagnosis and in monitoring of disease progression.
  • Use neuroimaging of upper motor neuron (UMN) pathways as diagnostic tool and to monitor progression.
  • Use functional neuroimaging studies (fMRI, MEG) to correlate anatomy and clinical function.
  • Use neurophysiological studies of UMN and lower motor neuron pathways for diagnosis and disease monitoring.
  • Conduct epidemiological studies to examine risk factors for the disease.
  • Study dementia in ALS using cerebrospinal fluid, imaging, molecular biology, and neuropsychological testing.
  • Collaborate with outside colleagues and industry to conduct therapeutic trials, both biological (e.g. stem cell) and chemical.

While the initial focus will be on ALS, the program could be broadened to include other motor neuron disorders, such as pediatric and adult forms of spinal muscular atrophy, and post-polio syndrome, leading to the development at UCSD of a comprehensive Motor Neuron Study Collaborative.

Related Clinical Information

See Also

Related Faculty

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View a discussion about ALS with faculty members Don Cleveland, John Ravits, Al La Spada and Larry Goldstein.