August 1, 2008

Stem Cells Generated from ALS Patients for the First Time

By Richard Robinson, Science Writer

For the first time, stem cells have been generated from individual patients with ALS. The accomplishment is likely to lead to development of new models of ALS and new understanding of disease mechanisms.

“They will also provide a potential resource for drug discovery and the development of new treatments for ALS,” said Lucie Bruijn, Ph.D., science director and vice president of The ALS Association.

“Model systems to date have focused on the SOD1 mutations linked to 2% of ALS. These findings enable the development of cell lines from ALS patients, even those for which the specific causative genes remain unknown,” Bruijn said. “The ability to generate human motor neurons from ALS patients carrying genes linked to the disease is a very exciting accomplishment building on novel technology and the work of several groups.”  Motor neurons are the nerve cells that die in ALS.

In the present study, researchers at Harvard University and Columbia University took skin cells from patients with a genetic form of ALS, caused by mutation in the superoxide dismutase (SOD1) gene. The skin cells were treated with a small set of genes that scientists have recently learned will reprogram adult cells to become stem cells capable of developing into many cell types.

The researchers showed that the genes “deprogrammed” the skin cells, reverting them to an earlier stage in their development, turning them into stem cells. Stem cells, which are formed normally during human development, have the ability to become many different kinds of cells. The researchers showed the new stem cells could transform into motor neurons.

Parallel Efforts Underway by The ALS Association

One important next step will be to make sure the motor neurons can mature, since it is mature motor neurons that are affected by ALS. Such cells could then be examined to determine what factors make them susceptible to the disease process. These studies will need to be done not only in cells from the patients in the current work, but also in cells derived from other patients, to make sure the results are widely applicable. Another important step will be to generate stem cells from patients with non-genetic (sporadic) forms of the disease. Motor neurons derived from ALS patients will be compared with those derived from people that do not have ALS. These studies are currently underway in parallel efforts funded by The Association.  See http://www.alsa.org/research/grants.cfm?grant_id=192&type_id=&category_id=&state=&country=&title="&institution=&investigator=44&zip=&radius=250&action=detail

Mature motor neurons would likely be useful for drug discovery, looking for compounds that improve their survival. While much publicity has surrounded the possible use of stem cells to replace dying motor neurons in ALS, the potential of such a treatment is unknown and was not attempted in this study.

More information on stem cells in ALS can be found HERE (http://www.alsa.org/research/article.cfm?id=715)