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Avitzur, Orly, MD, MBA


In October 2004, the AAN released a position paper regarding the use of embryonic and adult human stem cells in biomedical research. It can be accessed at:

Next month's In Practice column will continue with a discussion of neurologists who work in the biotechnology and pharmaceutical sectors.

The August 2001 executive order by President Bush limiting embryonic stem cell research to 60 stem cell lines already in existence has not provided the last word on what investigators can – or cannot do – in the US. Indeed, the November passage of Proposition 71 in California – an initiative authorizing low-interest, tax free bonds providing up to $3 billion in funds over 10 years to fund stem cell research in the state – is causing a stir in the academic ranks of neurology.

In what is being dubbed “the stem cell gold rush,” neurologists, neuroscientists, and other investigators are heading westward in pursuit of research unencumbered by current federal restrictions. Moreover, there is now an obligatory race for states to provide money for stem cell research – lest they lose investigators and biotech firms to California.



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Consider, for example, the academic odyssey of Arnold R. Kriegstein, MD, PhD. He became Director of the Developmental and Stem Cell Biology Program at UCSF in August 2004 when he left Columbia University where he had served as Director of the Neural Stem Cell Center. The program at UCSF, established in August of 2002, was launched with a $5 million matching grant from Andy Grove, Chairman of Intel; last winter it increased its funding to more than $11 million.

“The prohibition covers NIH funds, not private industry” noted Dr. Kriegstein, “and with Proposition 71, there are definite opportunities in California that don't currently exist anywhere else; only an investigator in California can apply to the California Institute for Regenerative Medicine for funds.”

Dr. Kriegstein's research focuses on early stages of brain development. “I believe that to recreate the process that starts with an undifferentiated stem cell and turns it into a more specialized cell type, we need to understand the steps in the normal process,” he said.

“Anyone who is interested in working in translational aspects of stem cell research – applications intended to discover targets for therapeutic intervention in disease – is more likely to want to use human cell lines than animal model cell lines,” he continued. “When you get closer to testing in patients, and bring it to the point where it can be used therapeutically, no question you want to do it in California.”

He added: “The other advantage of funding in California is that the ten-year timetable allows a young investigator to proceed with the security that there will be sufficient time to set up a lab, attain funding, and establish a reputation.”

In fact, since Proposition 71, Dr. Kriegstein has seen an enormous increase in unsolicited CVs and inquiries from neurologists and scientists, as well as a tremendous response to ads.

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John A. Kessler, MD, Chair of Neurology at Northwestern University Medical School, has been working in the field of stem cell research for the past 12 years. “I think that as time goes by, federal legislation related to embryonic stem cell research will undoubtedly exert an effect on where research is performed. NIH funding is getting more difficult to attain, and although I am less concerned about established investigators, young investigators faced with the thought of huge restrictions will be attracted by the availability of funds in California. Young scientists know that their careers are at risk if they are unable to attain grant funding.”



Although Dr. Kessler had worked in neuroscience research his entire career, his work became personal when his daughter, Alison, was injured in a skiing accident and developed a spinal cord injury. Since that time, spinal cord injury has become the focus of his clinical research.

His laboratory is studying the question of what directs stem cells to become specific populations of neurons and glia; he is also working to combine nanotechnology with stem cell biology in order to promote regeneration of cells following injury. He is optimistic about the future. Dr. Kessler said, “Obviously predicting when successful treatment of spinal cord injuries will be achieved is unknowable, but I would be very surprised if we do not achieve viable therapies in the next six or seven years.”

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Douglas Kerr, MD, PhD, Assistant Professor, Neurology and Molecular Microbiology and Immunology, and Director of the Johns Hopkins Transverse Myelitis Center, has been working in the field of stem cell research for the past four years. He is studying the potential of stem cells to restore function in paralyzed animals and hopes to ultimately use this knowledge in human paralyzing disorders such as ALS and spinal cord injuries.

“We have learned how to efficiently and specifically generate embryonic stem cells into motor neurons, we have begun to learn how to guide them to extend axons into muscle,” he said, “and we are trying to develop a set of strategies to guide axonal growth to the correct muscle.”

“We are currently using mouse embryonic stem cells for which there are fewer restrictions than human embryonic stem cells. In part, we are not using the human stem cells due to limitations of funding.”

Dr. Kerr believes Proposition 71 makes California a very attractive state in which to conduct this type of research. “It may indeed cause some geographic redistribution of scientists in this field,” he said. “Hopefully, the offshoot may be that this legislative precedent helps other states with similar initiatives to succeed so that a ‘brain drain’ does not occur.”

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Dawn McGuire, MD, was trained in neurology at the University of California-San Francisco Medical Center, completed an NIH-fund program in experimental therapeutics, and served as fulltime faculty in the Department of Neurology. She is currently Chief Medical Officer at Avigen, a publicly held biotech company in Alameda, CA, and heads their human clinical research programs in gene therapy and other experimental therapeutics in neurological disease. She believes that one important impact of Proposition 71 will be that fewer neurologists and scientists working in embryonic stem cell research will be tempted to leave the US in order to work in a less constrained environment.

“The federal restrictions – limiting the number of cell lines ‘blessed’ and supported by federal funds – really amounts to research sanctions,” she said. “Over the past few years, some academic research centers have lost gifted stem cell researchers to the UK, for example, where the environment for regenerative medicine, including tissue re-engineering, is more welcoming. We may be able to fend of the threat of a ‘brain drain’ in stem cell research, if the funds made available by Proposition 71 are managed well.”

Dr. Kerr has also seen good neuroscientists go abroad. “It is difficult to navigate through the limitations in the US and many lines of human embryonic stem cell research are simply not available to US researchers,” he said. “We need to be able to extend these lines to US researchers.”

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In fact, many other countries are rapidly advancing their stem cell research programs. Doros Platika, MD, President and CEO of the Pittsburgh Life Sciences Greenhouse, a public-private partnership of funds and resources which includes an incubator, investor network, and venture capital network for the support and development of new life science companies, practiced traditional academic neurology for 12 years before founding and managing several successful biotechnology companies.



“I was involved with a number of stem cell companies in the past, but the current legislation poses several significant challenges that deter investors,” he said. “In addition to the technology risk inherent in any biotech venture, there is a legal risk that makes investors shy away from this field. For example, if there is a breakthrough in the treatment of Parkinson disease through the application of human embryonic stem cell research, would Medicare or the other payers cover such treatment in this politico-ethical environment?”

Tamir Ben-Hur, MD, PhD, Chief Physician and Associate Professor in Neurology in the Hadassah Hebrew University Medical Center and Medical School, divides his time between clinical neurology and basic research in a more supportive regulatory environment.

He is currently researching transplantation of stem cells in experimental autoimmune encephalomyelitis – the animal model of multiple sclerosis – and the mechanisms by which transplanted cells improve the clinical signs of disease. “We are also working with Dr. Ben Reubinoff, Director of The Hadassah Embryonic Stem Cell Research Center, to develop the use of human embryonic cells for dopaminergic neurons in an animal model of Parkinson disease,” he said.

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What about laws and policies in Israel? “Stem cell research was brought to public and parliament discussion resulting in a law that specifically allows it,” said Dr. Ben-Hur. “The legislation, which requires re-evaluation and renewal every five years, permits research and cloning human embryonic stem cell lines for therapeutic purposes and prohibits human cloning. The government supports the collaboration between academic researchers and biotech companies in stem cell research by matching funds.”

Jewish scholars in the US, including the Orthodox Union, have come out largely in support of embryonic stem cell research. Dr. Ben-Hur explained, “According to all sectors of Judaism, the embryo is not considered alive until 40 days of gestation and provided that it is in the uterus. Therefore, even the Orthodox religious sector supports embryonic stem cell research with its potential to cure diseases.

Collection of ovum and IVF is permitted in Israel strictly for the sake of reproduction, however, not for research or for economic rewards. Frozen embryos (blastulas) are kept for five years after which they can be discarded. Under these circumstances, and with the couple's specific consent to donate the material to research – thus losing any rights over the outcome of that embryo –, the embryo can serve to produce a new human embryonic stem cell line. By this law, these lines can be made within the ethical standards of both religious and secular sectors in our society and prevent the formation of a black market.”

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Dr. Kerr recently spoke at the Christopher Reeves Paralysis Foundation in Washington DC in favor of a public action strategy for paralysis “It is an incredibly exciting time to be a neurologist and neuroscientist,” he said. “We are at the brink of finding dramatic cures for patients and any neurologist starting his or her career will soon see amazing improvements in how we treat disease. We will no longer be considered the specialty for which little can be done for the patient … all that is changing … and part of it will be due to stem cell research.”

Dr. Kessler has testified before Congress in support of lifting restrictions on stem cell research and has been active in advocating for the passage of Illinois' stem cell research bill. He advises young people who are interested in science: “Stem cell research is an extraordinarily exciting field that will change the way that we practice neurology.”

But Dr. Platika laments, “We now risk being surpassed in a field that we in the US pioneered. The restrictions imposed on stem cell embryonic stem cell research provide an opening for other countries that are more welcoming in terms of such research. Singapore, for example, has built an entire city, Biopolis, to support facilities for stem cell and other biotech research. The most effective research scientists are bound to go to where the environment is most receptive.”

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The laws restricting stem cell research vary widely from state to state. Some states are following California's lead and introducing more favorable legislation in an attempt to prevent the loss of researchers and scientists. New Jersey is the second state to pass a law specifically allowing embryonic stem cell research. Earlier this year, New Jersey Governor Richard J. Codey announced a $380 million initiative to build the Stem Cell Institute of New Jersey and to finance promising stem cell research.

New York and Connecticut are also considering proposals to encourage researchers in this field or to provide them with economic incentives.

In January, New York State Democratic leaders proposed that $1 billion be used over a ten year period for the creation of a stem cell institute and Connecticut Governor Jodi Rell proposed committing $20 million to fund stem cell research after state legislature failed to pass a favorable bill in 2004.

For more information on your state's legislation:

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  • ✓ Stem cell investigators discuss how the funding environment for stem cell research – particularly, in California where more money is available – is affecting job growth in certain parts of the country.
© 2005 American Academy of Neurology