Kwang-Soo Kim, PhD, directs the Neurobiology Lab at McLean Hospital, where he has spent most of his career tirelessly trying to track down the elusive cure for Parkinson’s. His research has focused on developing novel therapeutics to treat Parkinson’s disease, in particular via neuroprotective drug development and cell replacement therapy using induced pluripotent stem cells (iPS cells) derived from patients’ own skin biopsy. Based on successful in vivo studies using animal models of PD, he hypothesized this might work in combatting the disease.
In 2017, the FDA approved a “compassionate use” or “expanded access” protocol to implant one Parkinson’s patient with his autologous derived dopamine cells. The selected patient was an internist from the west coast whose symptoms had progressed despite taking the regimen of medications, and he was experiencing diminished quality of life. Two surgeries were performed, each six months apart, one in September 2017 and the other in March 2018. The second surgery was performed by Jeffrey Schweitzer, MD, PhD, together with Bob Carter, MD, PhD, in Neurosurgery at Mass General Hospital.
To date, the implanted patient has shown “modest improvements in his symptoms,” according to Kim, but more recently his improvements have “plateaued.” While Kim continues to gather more data, he remarks that he’s grateful, at the very least, that the sole implanted patient had no adverse effects until now from the surgery. However, the patient’s recovery was only modest, prompting Kim to investigate the underlying cause of the limited survival of implanted dopamine neurons.
“From our rodent studies, we learned that when we injected the dopamine cells, they did not survive the transplant,” said Kim. This he attributes to what he calls “needle trauma,” which is an inflammatory immune response triggered by the insertion of the needle into the brain. Thus, “needle trauma” negatively impacts dopamine cells’ survival.
Kim needed a workaround to mitigate this inflammatory immune response. Would transplanting the dopamine cells with autologous (from the same person) T-regulatory cells work, he postulated? Could that co-transplantation reduce the inflammatory response from the needle trauma? Yes. Dramatic improvements were observed in model animal studies showing greater survival of dopamine cells co-transplanted with autologous T-regulatory cells.
It is hoped that co-transplantation with autologous T-regulatory cells will provide added benefits to the cell therapy. “We can’t cure the disease,” says Kim. “But if the cell therapy works, we can provide a good quality of life.”
Sherene Shenouda, PhD, Director, Business Development & Licensing, worked closely with Kim to move his innovation forward. “Dr. Kim’s pioneering efforts in the field are at the forefront of medical innovation. He has achieved a ground-breaking milestone with the successful transplantation of autologous midbrain dopaminergic progenitor cells into a Parkinson’s patient--a historic achievement.”
With a blast of new funding and patents on his work, Kim hired more lab help and sped up his experiments. He has also founded NurrOn, a clinical-stage biopharmaceutical company dedicated to developing novel, targeted therapeutics for the treatment of Parkinson's disease and other associated incurable diseases. Kim hopes that combined treatment of novel drugs and cell therapy will maximize the benefit to Parkinson’s disease patients in the future.
Three weeks ago, NurrOn and partners, HanAll Biopharma and Daewoong Pharmaceutical, announced they had received approval from the FDA for phase 1 first-in-human clinical trial for HL192, the small molecule associated with the development and maintenance of dopaminergic neurons. Initial results of the trial are expected next year.
“Dr. Kim's personal dedication and passion for his work are truly inspiring. Collaborating with him over the past several years has been an enriching experience. I am eagerly anticipating the continued advancement of his technology and its potential to profoundly enhance the quality of life for individuals afflicted by Parkinson's disease,” says Shenouda.