“It is exciting to think that what started as research in cells ... could one day have an impact on patients.”
Oren Levy, M.D., Ph.D.
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Marina Emborg, M.D., Ph.D.
This article originally appeared in the Winter 2009 edition of PDF's quarterly newsletter, News & Review.
Imagine if your doctor was able to treat your Parkinson’s disease (PD) with a therapy made from your very own skin cells!
It may sound futuristic, but it’s exactly the type of treatment, called ‘personalized medicine,’ that is envisioned by Marina Emborg, M.D., Ph.D., and Su-Chun Zhang, M.D., Ph.D., both of the University of Wisconsin, Madison. They were recently awarded $150,000 from the Parkinson’s Disease Foundation (PDF) to study the potential of this concept.
If you are a regular reader of News & Review, Dr. Emborg’s name may be familiar to you. She has twice authored our cover story, both times providing comprehensive updates about new treatments in development for PD. Now we are proud to call her a PDF-funded researcher. Dr. Emborg, along with Dr. Zhang, is conducting a pilot study that examines the use of transformed adult skin cells in the treatment of Parkinson’s.
With new technology, first used successfully in 2007, scientists can take skin cells from a person with PD and coax them to become pluripotent stem cells (iPS). These cells appear to have similar potential to embryonic stem cells (hESCs), but sidestep the ethical and political debates associated with hESCs. The most important similarity between the two is the ability to become any other type of cell in the human body, including dopamine neurons — the cells that are damaged in the brains of people with Parkinson’s. Scientists theorize that transplanting the transformed cells into the brains of people with PD could ease symptoms of the disease.
Dr. Emborg and Dr. Zhang will test this theory in monkeys. Dr. Zhang will transform the skin cells of Rhesus monkeys into iPS cells and subsequently into dopamine neurons. Next, Dr. Emborg will implant the transformed dopamine neurons into the brains of the same animals that donated the skin cells — animals that, through use of the compound MPTP, will develop a condition much like PD. She will observe how well these cells survive and integrate in the brain and will measure the cells’ effect on the health of the monkeys, monitoring the development of tumors, immune reactions and changes in the animals’ parkinsonian signs.
Drs. Zhang and Emborg hope that implanting the transformed cells back into the monkeys from whom they were originally taken will replace the dopamine neurons missing and damaged by PD. Although technical challenges lie ahead with the iPS method, treatments made using the method would derive from the host’s own genetic materials, avoiding issues of immune rejection that can be found with other stem cell therapies.
Dr. Emborg notes that while her investigations are in early stages, the results from this pilot study, “should illuminate the potential of an exciting concept: treating people with Parkinson’s with their very own cells.”
Marina E. Emborg, M.D., Ph.D., is Director of the Preclinical Parkinson’s Research Program at the Wisconsin National Primate Research Center and an Assistant Professor of Medical Physics at the University of Wisconsin-Madison.