In milestone, scientists create stem cells matched to ALS patients

By Carolyn Y. Johnson, Globe Staff

Reaching a milestone in stem cell research, scientists at Harvard and Columbia universities reported today that they created the first stem cell lines from sick people, then coaxed these cells to become nerve cells genetically matched to those that had gone bad in the patients’ spinal cords.

In a paper published online in the journal Science, the team claimed success at what researchers have long been racing to do -- create in the laboratory a plentiful supply of cells that have the same genetic makeup as a patient with a particular disease.

The work was done with patients suffering from ALS, or Lou Gehrig’s disease, but the researchers said the same technique could be used to study many other genetic diseases. By comparing diseased cells to normal cells in a Petri dish, scientists hope to better understand what causes disease and search for potential drug targets.

A series of dramatic discoveries over the past two years have pushed stem cell science forward, so this advance was expected. But the work is the first example of scientists reporting complete success at a task that has been a fundamental reason for doing stem cell research.

"Since the cloning of Dolly the sheep and the first derivation of a human embryonic stem cell line by Jamie Thomson some ten years ago, it's been the hope of scientists ... to generate stem cell lines that have the genes of a patient," said Kevin Eggan, a co-author of the paper and a principal faculty member of the Harvard Stem Cell Institute. "This really suggests that it's going to be possible to make these cells from patients suffering from other diseases" -- whether it is Parkinson's disease, diabetes, or genetic cardiac maladies.

To accomplish their task, Eggan and his colleagues took advantage of a new technique first developed two years ago by Japanese stem cell researchers.

By inserting four genes into skin cells taken from 82- and 89-year-old ALS patients, the scientists triggered those cells to revert to an embryonic-like state, capable of developing into any cell. Then, they prompted the stem cells, called IPS cells, to become motor neurons, the type of cells that die off in ALS.

Next, they hope to study the motor neurons derived from the ALS patients, comparing them with normal ones to see whether they can observe differences that would help them better understand how ALS develops. A more distant goal is to fix defects in the cells and transplant them back into patients, but the technique currently used to create the cells requires adding viruses and genes that can cause cancer.

"The value of the work is not for treatment. But as a tool to find treatment it's really great," said Jose Cibelli, a professor of animal biotechnology at Michigan State University. "It's one of the papers that is predictable, but until someone actually shows it works, it's up in the air."