Stem cell therapy for Parkinson’s disease approved for Phase 1 trials
XS-411 uses iPSCs, which can be developed into nearly any other cell type
The U.S. Food and Drug Administration (FDA) has accepted an investigational new drug (IND) application for XS-411, a stem cell-based therapy developed by Xellsmart for Parkinson’s disease, clearing the way for a Phase 1 clinical trial.
The company also received FDA approval for a similar stem cell therapy to enter clinical trials for amyotrophic lateral sclerosis (ALS), another neurodegenerative disease.
These regulatory steps “[pave] the way for these therapies to advance into clinical trials in the United States and [lay] the foundation for future commercialization in the global market,” Xellsmart’s CEO and founder Xiang Li, PhD, said in a company press release.
An ongoing clinical trial of XS-411 in Parkinson’s patients in China, where Xellsmart is based, has so far reported a reduction of Parkinson’s symptoms and no adverse events related to the treatment, according to the company.
In Parkinson’s disease, the loss of nerve cells that produce dopamine, a chemical transmitter essential for motor function, leads to tremors, slowed movement, and a range of both motor and nonmotor symptoms. XS-411 is designed to replace these damaged cells, potentially alleviating symptoms.
What is XS-411 and how does it work?
The therapy utilizes induced pluripotent stem cells (iPSCs), a versatile type of cell that can be developed into nearly any other cell type in the lab. Researchers can differentiate iPSCs into dopaminergic neurons, which are then injected into the brain. If successfully integrated, these new dopamine-producing neurons may help restore some of the function lost as the disease progresses.
XS-411 uses allogeneic iPSCs derived from third-party donors, rather than from the patients themselves. This approach allows for the mass production of stem cells and the development of an “off-the-shelf” product that can be readily available for multiple patients. While autologous iPSCs, that is, those derived from the patient, can help minimize the risk of immune rejection, allogeneic iPSCs offer the advantage of scalability and the potential for more accessible, standardized treatments.
XS-411 has shown initial safety and efficacy in a clinical trial in China, according to Xellsmart. In the study, cells were injected into the striatal putamen, a brain area affected by the loss of dopaminergic neurons, in patients with moderate or moderate to severe Parkinson’s.
In six to 12 months of follow-up, the participants haven’t reported complications related to the therapy, according to the company. All those treated showed significant improvements in key measures, such as increased on time, which is when symptoms are adequately controlled, and reduced motor and nonmotor symptoms, as reflected by lower scores in the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale.
The company believes the results support XS-411’s further development. “XellSmart remains firmly committed to providing allogeneic, off-the-shelf, iPSC-derived regenerative cell therapies to patients with [central nervous system] diseases worldwide,” Li said.
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