HD research news - medical research into treatment & prevention

HDF workshop finds stem cells in the brain offer hopeful new paths for research

Ethan Signer, Ph.D., Executive Director, Cure HD Initiative

At a recent Hereditary Disease Foundation Workshop held in New York City on July 18 and 19, researchers focused on the striking and unexpected finding of "stem cells" in the adult mammalian brain, a finding with valuable implications for exploring previously inaccessible features of the disease process.

Stem cells are basic, undifferentiated cells that can generate not only more stem cells but a host of different adult cell types as well. Each stem cell is like a baby, with the potential to develop in many different directions. For many years, neurobiologists believed that cells of the adult brain, which normally don't proliferate further, had permanently lost that special ability. They believed there were no stem cells in the brain. It has recently become clear, however, that a small number of brain cells are, in fact, stem cells. In the proper conditions, these stem cells can become activated, begin to proliferate, and eventually produce what is essentially the entire range of differentiated brain cells.

Because Huntington's disease is marked by the loss of neurons in specific brain regions, the striatum and cortex, this finding raises the possibility that stem cells might provide a way of replacing the neurons lost to disease. Although this possibility is quite likely far off, it is certainly real and promising.

More immediately, though, stem cells provide an exceedingly valuable research tool for learning about features of the disease process that as yet we have no other way of approaching. With that in mind, the HDF organised a workshop entitled "Neural Progenitor Cells and Novel Regenerative Strategies for Neurodegenerative Diseases" which was held in mid-July in NYC. Seventeen scientists sat down to discuss how best to exploit the possibilities of stem cells in the brain, using the new transgenic Huntington's disease mouse models developed in the last three years, most of them with Hereditary Disease Foundation support.

One approach is to graft not only normal cells into diseased brain, but also stem cells carrying the Huntington's disease gene into normal brain, in order to study how the cells interact. Another approach is to uncover the molecular signals that can activate stem cells, so that chemical compounds able to generate such signals can be provided from outside, with drugs for example.

The level of excitement and enthusiasm among the participants was unusually high, and the coming months will likely see more and more stem cell research bearing on Huntington's disease. This should in turn soon begin to reveal details of disease initiation and progression - formation of intranuclear inclusions (clumps of protein in the nucleus), establishment of connections among neurons, behavioural manifestations and the like - that could prove vital for eventual therapy.