Despite recent drug-breakthrough news for management of psychiatric symptom management (psychosis) associated with the neurologic degenerative nature of Parkinson’s disease (PD), the cause and potential cure for PD still elude the medical community and patients challenged with this disorder. However, the tide may turn on this bleak prognosis as researchers from McLean Hospital and Massachusetts General Hospital (MGH) have published findings of a study in which they reprogrammed and repurposed cells from the patient’s own brain, representing an advantage over using cells from another individual.
Senior author, Kwang-Soo Kim, PhD, director of the Molecular Neurobiology Laboratory at McLean Hospital, the largest clinical neuroscience and psychiatric affiliate of Harvard Medical School, and colleagues set out to explore opportunities to slow progressive brain cell death using the patient’s own dopaminergic brain cells, believed to be the origin of PD.
The team hypothesized that with the use of the patient’s own cells, the risk of rejection and treatment failure would be minimized, if not entirely avoided. “Because the cells come from the patient, they are readily available and can be reprogrammed in such a way that they are not rejected on implantation. This represents a milestone in 'personalized medicine’ for Parkinson’s,” says Dr. Kim, who added, “We have shown for the first time in this study that these reprogrammed cells are still recognized as self by the patient's immune system and won't be rejected.”
The team embarked on the reprogramming of a 69-year-old subject’s skin cells, and through a number of challenging steps, they conducted an in-vitro differentiation and transformation process that triggered the cells to behave like the dopamine-releasing neurons that are degenerated in PD.
Prior to the surgical implantation, Dr. Kim and colleagues received approval for a single-patient Investigational New Drug application. These processed cells were tested to ensure that the potential for immunologic reaction was minimized (free from immunogenicity). Once confirmed and characterized, the cells were implanted into the left hemisphere, and 6 months later into the right hemisphere of the patient’s brain. The team highlighted that there was no need for induced immunosuppression and that the clinical measures they used to monitor improvement 18 to 24 months after the surgery were notable. Due to the broad impact of the potential for a cure or disease-mitigating intervention, the research received funding Funded from the National Institutes of Health and other institutions.
Co-senior author and chief of Neurosurgery at MGH, Bob Carter, MD, PhD, stated, “This strategy highlights the emerging power of using one's own cells to try and reverse a condition—Parkinson's disease—that has been very challenging to treat. I am very pleased by the extensive collaboration across multiple institutions, scientists, physicians, and surgeons that came together to make this a possibility.”
The authors concluded that their research led to a successful outcome with the patient’s imaging tests confirming that the transplanted cells were alive and functioning as natural dopaminergic neurons in the brain as long as 2 years after the surgical implantation. According to the scientists, this serves as a positive early indicator that this intervention deserves further studies to validate the findings.
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