Collaboration in Developing New Cell Therapy

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Bob S. Carter, MD, PhD: In a recent report in The New England Journal Of Medicine, our team of scientists, physicians and surgeons reported on a new approach to treating Parkinson's disease. This personalized cell therapy for Parkinson's, was made reality by a team of physicians and researchers from McLean hospital, Massachusetts General Hospital and Harvard Medical School. I've asked our team members, doctors Jeffrey Schweitzer, Todd Herrington and Kwang-Soo Kim, to describe for us the concepts behind the therapy and implementation for the first Parkinson's disease patient treated with this method.

Kwang-Soo Kim, PhD: The conceptual idea behind this therapy is to replace dopamine cells which have been lost in Parkinson's disease. Because Parkinson's disease is mainly caused by the loss of dopamine neurons in the midbrain area, dopamine cell replacement is a promising and new therapy for Parkinson's disease. So my research goal during the last two decades has been to understand and optimize each aspect of cell-replacement therapy for Parkinson's disease. In the current report, we used an approach which we describe as personalized cell therapy for Parkinson's. This is called personalized, because we actually use the patient's own cells and reprogram them into nerve cells that produce dopamine. Years of effort were required to prove this concept in the laboratory environment. We finally established a platform to do this approach and obtained FDA approval to treat the first patient. First, we biopsied the patient's skin cells and grow them.

Our next critical step was to figure out how to safely and efficiently reprogram these skin cells back into our early stage stem cells, namely Induced Pluripotent Stem Cells, or IPS cells. Then we optimized the way to turn these IPS cells into brain cells that produce the all important missing chemical dopamine. The scientific foundation behind this report builds on the work of many different investigators over many years. One of the special advances made by our team includes developing the right combination of agents, which allow for safe and effective reprogramming of cells. Then we tested these cells in a large number of preclinical studies, to prove that they can correct Parkinson's in animal models, without any tumor formation.

Todd M. Herrington, MD, PhD: The personalized cell therapy approach developed in Dr. Kim's laboratory, involves taking a Parkinson's patient's own cells, transforming them in the laboratory, and then using them to correct a problem at the cellular level in the brain. It is tremendously exciting. However, we also know that many therapies that work in animal or preclinical studies, prove not to be effective in human studies. And we've learned a lot, even from that one patient. We now have a better understanding of how these transplanted cell graphs appear on MRI scans over time, how much dopamine is produced by these graphs. This is also the first test of our hypothesis, that because the original source of the cells came from the patient himself, that the cell graphs would not be rejected and immunosuppressive medications would not be required.

Jeffrey S. Schweitzer, MD, PhD: My role in this project was to bring together the extraordinary scientific achievements of professor Kim's lab, with the prowess of Mass General Neurology and Neurosurgery, and the courageous efforts of the patient to volunteer for this novel therapy. In addition to that, I was involved in designing new and improved ways of delivering the cells into the brain safely, meaning so that both the cells and the brain suffered the minimum possible trauma and had the best possible survival and outcome. To do that we designed special techniques and equipment, which improved the survival of the cells by over 30%. Together, this team and our work here at Mass General, enabled us to ask the right questions, so that we can move on from here with the guidance of the FDA to design a full clinical trial that will allow us to learn what role this type of therapy will play in the care of Parkinson's disease in the future.

Bob S. Carter, MD, PhD: The Harvard Medical School environment, and the collaboration between Massachusetts General Hospital and McLean Hospital, has been the perfect incubator for bringing this new therapy to life. Our goal is to continue to build this approach into a center for cell therapy for Parkinson's, the opportunity to collaborate across the Harvard system, to build a dream team of scientists and surgeons and clinical neurologists, who would all come together with the goal of creating a new therapy for Parkinson's, has been very inspiring.

Of course, more so than anything, we're inspired by our first patient who allowed us to carefully study the effects of the therapy in order to share this information with our colleagues in the medical field. We feel that we have enough information to begin planning a phase one trial with a larger number of patients. This would be done under the auspices of the FDA and our local institutions. We will also continue the cycle of innovation, working with our team of basic scientists and clinical doctors to continue to push forward and improve this therapeutic approach.

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Learn more about the latest updates on Parkinson's Disease Cell Therapy