New Progress Against ALS

Lou Gehrig
Lou Gehrig might have considered himself lucky. But he couldn't have imagined that seven decades after his emotional farewell, there would still be no treatment for ALS.

"When I was initially diagnosed, the doctors, three different doctors, told me the same thing: 'There's nothing we can do. Go home and die,' basically," said Philip Carlo, an ALS patient.

But CBS Evening News Anchor Katie Couric reports that science may finally be making the kind of progress that would have made the iron horse proud.

"Much as those Yankees featured some of the greatest teamwork in the history of baseball. We have assembled a dream team of scientists and clinicians to work on the problem," said Valerie Estess, co-founder, Project ALS.

Once competitors, researchers from both Harvard and Columbia are working together - with skin samples from both patients and healthy donors to better understand what causes this disease.

"Well, for the first time we can now have billions of these cells to study in the laboratory and ask why it is that they get sick," says Dr. Kevin Eggan of the Harvard Stem Cell Institute.

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In ALS, nerve cells - called motor neurons - become incapable of sending messages from the brain, to the spinal cord, and on to the muscles - resulting in paralysis and ultimately death.

"You're alive. But you watch yourself die, and you can't do anything about it," said Carlo.

Rather than retrieving the motor neurons from the spinal cord of patients, a procedure that's far too risky, scientists are creating them from scratch.

"Skin cells are very accessible, but they have nothing to do with the disease. So that's why we've had to find out a way of turning the skin cells into motor neurons," said Chris Henderson, Do-Director, Motor Neuron Center, Columbia University.

Couric decided to donate some of her skin to science.

A tiny piece of skin is put it in a Petri dish, where cells then multiply. They are genetically modified to behave like embryonic stem cells - capable of becoming any kind of cell in the body.

"They have all of the characteristics of embryonic stem cells but they don't come from the embryo," said Dr. Eggan.

They're then able to manipulate those very malleable stem cells to become motor neurons - enabling researchers to do what's never been done before: study the progression of ALS under a microscope.

"We want to compare the motor neurons from ALS patients with the motor neurons from healthy individuals. And through that we really do believe that we'll come to a fundamental understanding of what leads to ALS," said Henderson

Which in turn, should help them develop a treatment for the disease.

Carlo said the research is essential, "we have light at the end of the tunnel. And that -you can't ask for more than hope."

Hope, that even the luckiest man on the face of the earth never had.

At the end of his speech, Gehrig said, "I might have been given a bad break, but I've got an awful lot to live for. Thank you."