ALS Research Update 2008. Richard A. Lewis MD Director, Hiller ALS Clinic and Research Center Wayne State University School of Medicine Detroit, Michigan. Current Research Areas (as shown on ALSA website). Axonal Transport . Neurofilaments are proteins that
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Richard A. Lewis MD
Director, Hiller ALS Clinic and Research Center
Wayne State University School of Medicine
Neurofilaments are proteins that
maintain axonal structure. Defects
cause motor neuron disorders
Mutations in Tubulins, Dynein
and Dynactin, all involved in axonal transport, have been shown to cause motor neuron disease
NMJ is the connection between the
nerve and the muscle. The site of
attack in Myasthenia Gravis.
In ALS the NMJ appears to degenerate
before the motor neuron degenerates.
This suggests that there is loss of the
nutrients and energy to this structure.
Dr. Loeb’s laboratory at WSU is looking
at Neuregulin, a protein important to the
development of both the NMJ and
Techniques now available to study transport
Dr. Jun Li at WSU is currently looking at transport of mitochondria and other structures in new genetic forms of MNDMitochondrial Transport
Impulsive, emotional behavior without memory disturbance
70% of patients with ALS may have FTD
TDP-43, effects mRNA in constructing neurofilaments
May be common thread between ALS and FTD
A way of tracking the progression of a disease and monitoring the effect of a treatment
Serum or Spinal fluid substance
Physiologic parameters- MUNE
The spread of disease may be prevented by targeting treatments to these cells
Using viruses and other vectors which can invade cells, researchers can introduce new genes, trophic factors and other substrates into neurons or glia.
Anti-sense molecules can attach to RNA and prevent protein formation
Short RNAi placed in cells will induce the cell to destroy the corresponding gene
SOD mutations produce an abnormal protein
RNAi could be targeted to the mutant SOD gene to prevent the production of the protein
A potentially powerful tool for a number of diseases
Mean survival increased by 36% (110 to 148 days)
Increased disease duration from 9 to 38 days
Delayed onset of paralysis
Compound or Trial