Progress Report May 2012
Co-Principal Investigator, Prof. Alan Mackay-Sim reports in mid-May 2012 that the stem cell research project is progressing on schedule. One of the new findings is that HSP neuronal stem cells, as well as having restricted microtubule networks, are only 80% the size of non-affected cells. The significance of this is not as yet known.
As drug screening progresses, Dr. Yongjun Fan is focusing on a class of compounds that are showing promise, as well as refining the methodology for the best way to do the drug screen. As mentioned in a previous update, the tests developed to measure the effectiveness of any potential drug compound have had to be invented from scratch (imagine trying to measure temperature without a thermometer). So there is a natural cycle of improvement and refinement with the tests as more data becomes available.
Gautam Wali is being successful in growing neuron-like cells with one long “arm” or axon so that they are like the elongated nerve cells in the spinal cord. He has also significantly advanced the technique for live imaging the transport and traffic of the tiny cell organelles that carry energy to different parts of the cell, and whose impairment leads to degeneration that is the cause of HSP symptoms.
Simon Weyers has been investigating non-SPG4, adult onset, pure/uncomplicated HSP stem cells that exhibit very similar gene expression to each other, but quite different from SPG4 stem cells. However, so far he is finding that the cellular outcomes are the same as for SPG4. This raises the possibility that if a cure is discovered for SPG4, it may also be a cure for some other types of HSP.