Scientists create neurons from skin cells of ALS patients

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(Agencies)
Updated: 2008-08-02 15:36

WASHINGTON -- A team of US scientists have for the first time used a new technique to transform an ALS (amyotrophic lateral sclerosis) patient's skin cells into motor neurons, a process that may be used in the future to create tailor-made cells to treat the debilitating disease.

This undated file photo shows the patient specific monitor neurons created in the Eggan Laboratory. [Agencies]

The research, conducted by scientists from Harvard University and Columbia University, was published in the August 1st issue of the journal Science.

This is the first time that skin cells from a chronically-ill patient have been reprogrammed into a stem cell-like state, and then coaxed into the specific cell types that would be needed to understand and treat the disease.

The motor neurons were created by using a new technique that can reprogram human adult skin cells into cells that resemble embryonic stem cells.

The technique, called induced pluripotent stem (iPS) cells, was a major advance in the field that was first reported last November by researchers in Japan and the United States. In their studies, they made use of skin cells from healthy adults. But it remained unknown whether iPS cells could be created with cells from chronically-ill patients and then transformed into neurons.

Researchers from the Columbia-Harvard team, in their paper, proved it was possible using ALS patients' skin cells.

ALS is caused by the degeneration and death of motor neurons, the nerve cells that convey nerve impulses from the spinal cord to each of the body's muscles. The death of motor neurons leads to paralysis of these muscles, including those involved in swallowing and breathing, and ultimately the death of the patient.

Though cell replacement therapies are probably still years away, the new cells will solve a problem that has hindered ALS research for years: the inability to study a patient's motor neurons in the laboratory.

"Up until now, it's been impossible to get access to the neurons affected by ALS," says co-author Chris Henderson at Columbia. "Our paper now shows that we can generate hundreds of millions of motor neurons that are genetically identical to a patient's own neurons. This will be an immense help as we try to uncover the mechanisms behind this disease and screen for drugs that can prolong life."

"Therapeutic use of the cells is probably a long way off," Henderson says. Right now there are safety issues with iPS cells, including a risk of cancer. "We also don't know how to reintroduce cells into a sick adult in a way that will be beneficial. All these hurdles need to be overcome first before we can think about using the cells to treat disease, but we can start immediately to evaluate them as a tool for drug discovery."

Researchers at Columbia and Harvard are already collaborating to investigate the cells with the ultimate goal of determining how they differ from a healthy person's motor neurons.