UAMS, Columbia ALS Study To Focus on Adult Stem Cells


Hynek Wichterle, Ph.D., a Columbia University associate professor of pathology and cell biology, neurosciene and neurology, speaks about his research of ALS using adult stem cells.

Thomas May, founder of the Katherine and J. Thomas May Center for ALS Research at UAMS, left; Mahmoud Kiaei, Ph.D., assistant professor in the UAMS College of Medicine's Department of Neurobiology and Developmental Sciences; and Hynek Wichterle.

March 20, 2013 | A Columbia University professor March 19 at the University of Arkansas for Medical Sciences (UAMS) explained his research into adult stem cells as part of the search for a treatment or cure for ALS — unofficially inaugurating the beginning of a research collaboration between the two universities.

Hynek Wichterle, Ph.D., an associate professor of pathology and cell biology, neuroscience and neurology at Columbia University, spoke on “ALS in a Dish: From Motor Neuron to Disease Modeling” as part of the UAMS Center for Translational Neuroscience’s Distinguished Lecture Series.

Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease, is a fatal degenerative disorder of the nerve cells in the brain and spinal cord that control voluntary muscle movement. ALS patients progressively lose the ability to function and care for themselves.

Mahmoud Kiaei, Ph.D., assistant professor in the UAMS College of Medicine’s Department of Neurobiology and Developmental Sciences, will help lead the UAMS research team that will work with the Columbia researchers. He introduced Wichterle before the lecture.

Finding a drug to treat ALS has been difficult because drugs and compounds tested successfully in mice for their effectiveness against ALS have not worked in humans.  Wichterle, working with researchers at Harvard University, has been able to take adult skin fibroblasts from ALS patients and convert them to stem cells then program them to grow into neurons that can be used to test potential ALS treatments.

Motor neurons fall into subtype categories. For example, some help control motor function in the eye while another type works in a part of the spinal cord.

Wichterle explained it is possible now to generate motor neurons with that kind of specialization.

Human motor neurons in ALS patients are difficult to access. They also are difficult to model in a lab because the disease in humans can take decades to manifest itself. Wichterle’s research team was able to find compounds that would accelerate the development of ALS in the neurons so drugs could be tested on them to see if they were effective in reversing the disease.

Oculomotor neurons, which control eye movement, are resistant to ALS. Having those available for research will facilitate more in-depth research as to what it is that makes them resistant and what might make other types of neurons more vulnerable.

Being able to grow human motor neurons affected by ALS in an adequate supply means researchers can conduct many tests of drugs and drug combinations at once, speeding up the research and testing process.

“We are living in a time when many different fields are converging and enabling us to move forward in understanding neuro-degenerative diseases,” Wichterle said. “In the case of ALS, the research is being helped by the fact that we can generate motor neurons with such high efficiency.”

UAMS has acquired some of those cells for research here. Kiaei said the number of tests that now can be done make the overall job of finding more effective drug treatments for ALS too big for any one lab.

“Our lab here at UAMS will be further strengthened by the expertise they have at Columbia,” Kiaei said. “We can complement their work, move the field faster and have effective therapy to help patients as soon as possible. Everyone will contribute their own ideas.”

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