FDA clears cell therapy XS-228 for ALS Phase 1 clinical trial
Therapy uses allogenic cells, allowing for large-scale production
The U.S. Food and Drug Administration (FDA) has given Xellsmart Biopharmaceutical the go-ahead to start a Phase 1 clinical trial testing its stem cell-based therapy XS-228 in people with amyotrophic lateral sclerosis (ALS).
This milestone, reflecting the approval of Xellsmart’s investigational new drug (IND) application, follows promising results from early clinical studies performed last year in China, where the company is based. It positions XS-228 as the first experimental ALS therapy of its kind to reach this stage, according to Xellsmart.
The FDA granted XS-228 orphan drug designation to treat ALS, a status that supports and speeds the development of promising treatments by offering incentives like market exclusivity, faster review, and additional regulatory support.
Xellsmart also received FDA clearance to begin Phase 1 trial testing of XS-411, its cell therapy candidate for Parkinson’s disease.
“The FDA’s unconditional approval of the INDs for Xellsmart’s XS411 and XS228 products, targeting Parkinson’s disease and ALS, paves the way for these therapies to advance into clinical trials in the United States and lays the foundation for future commercialization in the global market,” Xiang Li, PhD, Xellsmart’s founder and CEO, said in a company press release.
Allogenic cells allow for large-scale production
ALS is caused by the progressive dysfunction and death of motor neurons, the nerve cells that control voluntary movements. As these neurons die, patients experience ALS symptoms such as muscle weakness, paralysis, and ultimately respiratory failure.
Current ALS treatment options are limited and largely focused on symptom management rather than targeting the underlying neuronal loss. Stem cell-based therapies like XS-228, which aim to replace the nerve cells lost to disease progression, are emerging as a promising new approach for ALS.
XS-228 uses induced pluripotent stem cells (iPSCs), which are adult cells reprogrammed in a lab into a versatile, embryonic-like state with the potential to develop into almost any cell type.
These iPSCs are then matured into motor neuron precursors and transplanted into the brains of ALS patients, where they are expected to integrate neural circuits and restore neural function.
XS-228, and more generally Xellsmart’s cell therapies, leverage the use of allogeneic iPSCs, meaning they are derived from healthy third-party donors. This strategy allows for the mass production of stem cells, meaning that XS-228 can be manufactured in large, standardized quantities and stored until needed.
This off-the-shelf approach offers rapid treatment accessibility for multiple patients and ensures consistent quality. This is in contrast to therapies based on autologous iPSCs, or those derived from patients themselves, involving a time-consuming, costly process that also suffers from high variability among patients.
Early clinical studies, conducted in China last year after clearance by China’s National Health Commission, demonstrated that XS-228 was safe and could slow disease progression relative to standard treatment, according to the company.
“Xellsmart remains firmly committed to providing allogeneic, off-the-shelf, iPSC-derived regenerative cell therapies to patients with CNS [central nervous system] diseases worldwide,” Li said. The CNS comprises the brain and spinal cord.
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