A phase 1 clinical trial recently launched in mass general Brigham examines the safety and feasibility of a revolutionary treatment approach for Parkinson’s disease in which a patient’s stem cells are reprogrammed to replace dopamine cells in the brain damaged by the disease. The first test of its kind of a transplant of autologous stem cells, based on research and technologies invented and validated preclinically at the Neuroregeneration Institute (NRI) of McLean Hospital, registered and treated three patients in Brigham and Women’s hospital.
In total, six participants will be included in the phase 1 trial which will follow the patients for 12 months and beyond to determine the safety of the procedure and monitor for any improvement in Parkinson’s disease. After the first 6 patients transplanted in the phase 1 study, researchers hope to extend and recruit more patients as part of the 2A phase study.
This new therapeutic approach to treat Parkinson’s disease incorporates the use of stem cells derived from the blood of a patient who had been converted into induced pluripotent stem cells (IPSC). These cells are then reprogrammed to transform into dopaminergic neurons of the specific medium brain ready for transplantation. The autologous transplantation approach to the use of a person’s own cells bypassing the requirement of immunosuppressive treatments, which are necessary when cells other donors are used.
The replacement of Parkinson’s disease cells replaces the dopaminergic neurons lost in degeneration and can restore the dopaminergic function in the brain, offering a completely new treatment modality compared to the currently available treatments. The founding director of the NRI, Ole Isacson, Dr Med SCI, who is also a professor of neurology (neuroscience) at the Harvard Medical School and in mass general Brigham, has launched cell therapy for Parkinson in the last 30 years and has laid the basics of this clinical trial.
“Seeing this new transformational replacement of patient cells from its own dopaminergic neurons materialize – very fundamental scientific breakthroughs of our laboratory to be completely translated into a clinical application for the patient’s suffering from Parkinson’s disease – is very rewarding,” said Isacson. “We believe that this approach could open a new treatment paradigm and lead to the development of many additional cellular therapies to restore damaged brain systems and replace degenerate brain cells in other diseases.”
Under the leadership of Isacson, the NRI of McLean has developed and patented an autologous cell restoration based on cells in Parkinson’s disease with a pioneering preclinical publication in 2002 using stem cells and the first preclinical demonstration of dopamine using human cells effective in 2010. The psychiatry teacher at the Harvard Medical School, provided the first Long -term safety evidence and advantages of autologous stem cell therapy in an animal model of non -human primate of Parkinson’s disease.
The NRI received the official authorization of the US Food and Drug Administration (FDA) on August 23, 2023, approving its investigation application on new drugs (IND) for a clinical trial of phase 1 to test this cell therapy of neuron dopamine unique.
Following this FDA approval for the Phase 1 clinical trial, the innovative preclinical work of the NRI was translated into a clinic with the first patient treated on September 9, 2024. This collaboration includes trial) departments in Brigham and Women’s Hospital. Isacson is not directly involved in the clinical trial because he is the holder of the innovative technology patent and also co-founder of Oryon Cell Therapies, which has the license of this technology. The test is led by Hallett and his colleagues from Mass General Brigham Healthcare System and his institutions affiliated with the Harvard Medical School.
“It is extraordinary to see that the investigators of our establishment can bring new treatments to patients throughout the laboratory process” bench at bedside “, and this inspires many investigators to pursue their scientific and medical ideas in order to reach patients in need”
The phase 1 open clinical trial will be the first trial of this type to test dopaminergic neurons derived from IPSC derived from blood in patients with Parkinson’s disease and is funded by the National Institute of Health Institute of Neurological Disorders and AVC (NINDS). The Ninds have awarded highly competitive cooperative research to activate and advance the subsidy of translational companies for organic (organic creation) for this work in 2020.
People looking for more information in the trial can send an email: @bwh.harvard.edu “title =” mailto: neurosurgergcrc@bwh.harvard.edu “> neurosurgiecrc@bwh.harvard.edu
For more information:
- The McLean Hospital receives a nih coveted subsidy to clinically study therapy of autologous stem cells for Parkinson’s disease
- Patient derived stem cells could improve research on drugs for parkinson
Funding: The study was supported by an organic Create Ninds subsidy (U01NS109463).
The content is only the responsibility of the authors and does not necessarily represent the official views of the NIH.
Disclosure:Isacson co-founded a company (ORYON CELL THERAPIES) which authorized patents and know-how to develop autologous cellular therapies for Parkinson’s disease. Isacson’s interests have been examined and are managed by McLean Hospital and Mass General Brigham in accordance with their conflict of interest.
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About the mass general Brigham
The mass general Brigham is an integrated academic health care system, uniting great minds to solve the most difficult problems in medicine for our communities and in the world. The Brigham mass general connects a complete continuum of care in a system of academic medical centers, community and specialized hospitals, a health insurance plan, doctors, community health centers, home care and long -term care services. The mass general Brigham is a non -profit organization engaged in patient care, research, education and community services. In addition, the mass general Brigham is one of the country’s main biomedical research organizations with several Harvard Medical School teaching hospitals. For more information, please visit Massgeneralbrigham.org.
(tagstotranslate) stem cells; Parkinson