A paradigm shift in the way we treat spinal injuries is now in sight, with the world's first regenerative cell therapy being granted approval for a registrational Phase I clinical trial. It's a historical milestone that could successfully treat what has, until now, been an incurable condition.
This week, the US Food and Drug Administration (FDA) and China’s National Medical Products Administration (NMPA) approved the global clinical trial to treat spinal cord injury (SCI), which is estimated to affect more than 15 million people worldwide. SCI impacts people across demographics, and most often results from traffic accidents, sporting injuries and other trauma, including serious falls and workplace incidents. There's no real cure; treatment is more management, with surgery and rehabilitation to restore some degree of quality of life. Sufferers, however, are often left with paralysis or severe disability for life.
Now, Chinese biotechnology company XellSmart has the potential to change this forever, as its allogeneic induced pluripotent stem cells (iPSC) regenerative therapy has been given the green light by both US and Chinese health administrations to enter a clinical trial. Pluripotent stem cells are, of course, immature stem cells that can develop into specific cells. In this case, the kind to replace the damaged or dead neural cells caused by SCI. The treatment aims to not just repair the injury but provide the foundation to regrow all the cells needed to return function to the damaged region.
"Each year, China and the US report approximately 100,000 and 18,000 new cases of acute or subacute SCI – equivalent to nearly 10 and two new cases every hour, respectively," a statement from XellSmart noted. "Most patients experience permanent disability, severely compromising their quality of life. Due to the limited regenerative capacity of the central nervous system, nerve repair following SCI remains extremely challenging."
The human trial news comes on the back of four years of preclinical research and aims to have broad application that doesn't require harvesting cells from a patient but offer a "one size fits all" off-the-shelf treatment that will work with anyone who has suffered SCI. This means, if it passes through the trial phases, it'll be easy to manufacture and scale for broad access.
And because of the subtyping of cells already being well-researched, the allogeneic cells – ones from other sources, not native to the patient – should have low rejection risk.
The SCI trial is being conducted in partnership with Sun Yat-sen University's Third Affiliated Hospital in China, a specialist in treating and researching these complex spinal injuries.
This trial is the latest for XellSmart, which is currently testing specific cell therapy in the treatment of Parkinson's disease and ALS. If the SCI trial is successful and this novel therapy makes it to market, it has remarkable potential that includes restoring function for individuals with paralysis.
Realistically, this Phase I trial – which tests for safety and efficacy, as well as dose parameters – should be completed by some time next year, and if successful will move to the next stage, involving a larger population. Phase II would then be expected to begin in 2028 at the earliest. But this therapy could be then mass produced and "off the shelf" available within five to seven years.
“We’re moving beyond care and into cure," said a XellSmart spokesperson. "For the first time, we’re offering real hope to millions living with spinal cord injury.”
Source: XellSmart via PR Newswire
