For people who suffer from familial dysautonomia (FD), hope recently came in the form of an Israeli chicken egg.
In 2001, a team led by Bar-Ilan University professor Ron Goldstein implanted human embryonic stem cells into chicken embryos to study the early stages of normal cellular development.
Now, Goldstein is using embryonic stem cells to create FD-carrying human nerve cells in an attempt to better understand how the degenerative genetic disease works and to test possible treatments.
“Once we can produce FD-infected nerve tissue in a petri dish, we will have a model to understand what is happening on a molecular level,” Goldstein told the Forward. “And this model could also be used to test new drug therapies for the disease.”
Familial dysautonomia is a degenerative disease of the peripheral nervous system — the nerves and neurons outside the central nervous system — that is found exclusively among Ashkenazic Jews. People who suffer from dysautonomia have difficulty swallowing and regulating such involuntary responses as body temperature and blood pressure.
Embryonic stem cells are particularly useful for advanced FD research, because of their pluripotent nature, which means they can proliferate indefinitely and develop into virtually any cell type. Essentially they are a “blank slate,” Goldstein said. These unique traits also make stem cells an attractive resource for medical researchers studying other, more common degenerative diseases such as Alzheimer’s and Parkinson’s.
But because days-old human embryos are destroyed in this process, various religious and pro-life groups have opposed the research. In July, President Bush vetoed a bill designed to award federal funding for embryonic stem-cell research.
Unlike some branches of Christianity, Judaism places no theological restrictions on research work with embryonic stem cells. While experimental work has slowed in the United States, scientists such as Goldstein have flourished in Israel in recent years.
“According to how Jewish tradition understands the fetus in utero, until the 40th day it is ‘like water,’” explained Rabbi Elliot Dorff, professor at the University of Judaism and author of “Stem Cell Research,” a rabbinic edict approved by The United Synagogue of Conservative Judaism. From the 41st day onward, Jewish tradition considers a fetus “like the thigh of its mother,” and therefore a full human being. Stem cells are harvested from embryos within 14 days of gestation, so there is no theological prohibition against their medical use according to the Conservative movement.
“The Jewish religion would consider it far better to use [embryonic stem cells] for cures than to simply throw them away,” Dorff said, citing the talmudic concept of saving a life in jeopardy, pikuach nefesh.
A wide range of Jewish organizations, including Hadassah, the Orthodox Union and B’nai B’rith International have pushed for increased domestic funding for stem-cell research. The chief officer of medical ethics at the Israeli Health Ministry, Rabbi Mordechai Halperin, said everyone agrees that using stem cells to research potential medical cures is permissible. “If Jews have ever been in complete agreement on anything, then this issue is it,” Dorff said.
With no religious or legal restrictions on using stem cells for scientific purposes, Israel has emerged as a leading center. According to a recent study published in the magazine The Scientist, Israeli researchers, per capita, are the world’s most prolific authors of articles in scientific journals on stem cells. The United States is number six on the list. State research centers such as Jerusalem’s Hadassah Medical Center and Haifa’s Rambam Medical Center have emerged as global leaders in advanced stem- cell research.
“The big difference is, here you can get governmental money,” Goldstein said, noting that the National Institutes of Health would not have approved funding for his earlier stem-cell experiments.
Medical advocacy groups in the United States, like the New York-based Familial Dysautonomia Foundation, are acutely aware of both the promise of Goldstein’s research and the difficulty in supporting such work domestically. “The best work we could have done was in Israel,” said David Brenner, president of the FD Foundation. The foundation gave Goldstein a $200,000 grant.
“U.S. labs couldn’t take our money for work on embryonic stem cells if they also receive money from the government,” Brenner said.
To circumvent these restrictions, however, private groups such as the $100 million Harvard Stem Cell Institute are forming to create an alternative funding stream for stem-cell projects in the United States.
“We [Israelis] had a little bit of head start,” Goldstein acknowledged, but “I think more and more we will lose our edge.”
Goldstein cautioned that more advanced treatments — like using stem cells to grow functioning nerve cells to replace those that did not develop properly in FD patients — are not yet on the medical horizon. “People may think that stem- cell replacement therapy is right around the corner, but it’s not,” Goldstein said. “It may not be five or even 10 years away.”
But Goldstein does hope that his six-person team will have the ability to test human neurons with FD within the next couple of years. “Mostly the issue is raising funds,” he said, echoing a common sentiment in the stem-cell research community.
Dorff, who authored the book “Matters of Life and Death: A Jewish Approach to Modern Medical Ethics,” suggested that it is no accident Israelis have emerged at the forefront of stem-cell research.
“Judaism is unique among the religions of the world in the emphasis it places on medical research,” he said. “Our theology portrays the doctor as the partner and agent of God in the ongoing act of healing.”