At Bar-Ilan University in Ramat Gan, near Tel Aviv, professor Ron Goldstein is trying to save lives one stem cell at a time.
He and his colleagues are using stem cells to study and devise treatments for familial dysautonomia, a genetic disease of the peripheral nervous system. Approximately 1 in 27 Ashkenazi Jews is a carrier.
“It used to be you couldn’t survive beyond age 5 to 7” with the disease, says Goldstein, a graduate of Columbia University who grew up in Highland Park, Ill., and immigrated to Israel in 1983.
Although some strides have been made in providing clinical care to those afflicted with dysautonomia — currently estimated at about 300 worldwide — it is nonetheless “a horrible disease, and the families that I’ve met are unbelievable, really saints,” says Goldstein, in San Francisco a while ago for a meeting of the American Society for Developmental Biology.
One of the first signs of the disease is exhibited in children who cry “but don’t shed tears,” he says. From there the nervous system continues to degenerate: Symptoms include loss of pain sensation, difficulty eating and breathing, irregular pulse and blood pressure, even scoliosis.
He calls dysautonomia an “orphaned disease” because “the money [for research] goes elsewhere.”
Goldstein’s lab is trying to create nerve cells, through the use of stem cells, that will be useful in developing and testing drugs for treatment of dysautonomia, and studying how the disease works. Eventually, the researchers hope to be able to transplant new cells into patients to replace nerve cells that have died as a result of the disease.
Like other stem-cell research taking place in Israel, Goldstein’s is not done in a vacuum. He has collaborated with professors Benjamin Reubinoff at Hadassah Hospital and Nissim Benvenisty of Hebrew University of Jerusalem. Back around 2001, the team of scientists extracted stem cells from human embryos and transplanted them into chicken embryos in an effort to learn more about the earliest stages of development.
While that type of procedure is prohibited in stem-cell lines approved by the U.S. National Institutes of Health, it is approved for the lines involved, which came from Australia.
Such restrictions have actually served Israeli scientists favorably, Goldstein suggests: The field of stem-cell research “is less competitive than it could be because of President Bush and because of Catholic countries in Europe … Israel has a very, very big head start in this” because of all the embryonic stem-cell research that has been done.
Goldstein is most interested in the applications of stem-cell research toward pharmacology and the embryonic stages of development, such as the human genome. The ultimate benefit, of course, would be in learning more about certain diseases and abnormalities, and how to treat them.
No stranger to California (Goldstein has a brother in Lafayette and a sister in Los Angeles, and his father lives in Walnut Creek), he was more than a little interested in the passage of Proposition 71, which will pump $3 billion into stem-cell research (although the total bond debt will cost California taxpayers $6 billion).
“It’s great for humanity,” he comments, “but it’s more competition for Israel in terms of stem-cell [research] … So we’re working as fast as we can.”
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