A detailed analysis of 13 case histories has found intravenous injections of stem cells lead to substantial motor function improvements in patients suffering spinal cord injury. The research also suggests the novel therapy is safe, with no major adverse events recorded, but larger clinical trials are needed to further affirm efficacy.
A number of different stem cell therapies targeting spinal cord injuries have been developed in recent years. Many animal tests have suggested these therapies could help repair damaged spinal cords, however, it is still early days for testing safety and efficacy in humans.
Mesenchymal stem cells (MSCs) derived from bone marrow have shown promising results in preliminary human trials. Prior studies have administered MSCs by invasive injections directly into the spinal cord, but this new research looked at how safe and effective the therapy is when administered more innocuously by intravenous infusion.
The study, published in the journal Clinical Neurology and Neurosurgery, closely details 13 case studies trialing the experimental therapy that led to a controversial approval of the therapy for clinical use in Japan back in 2019.
In 2014, Japan introduced a fast-track approval system allowing for conditional and time-limited use of certain experimental regenerative medicines as long as initial safety data has been supplied.
Called Stemirac, this stem cell therapy’s fast-track approval in Japan was criticized by international experts at the time. This new study is the first clinical data to be published from the preliminary test cases that resulted in the Japanese fast-track approval, and it certainly offers promising signs the still-experimental stem cell therapy may be effective in repairing spinal cord injuries. However, robust clinical trials are still lacking.
The 13 case studies described in the research detail rapid and significant functional improvements in motor function for more than half of the cohort. No substantial adverse effects were noted in any of the cases.
“Similar results with stem cells in patients with stroke increases our confidence that this approach may be clinically useful,” says co-senior author Jeffrey Kocsis. “This clinical study is the culmination of extensive preclinical laboratory work using MSCs between Yale and Sapporo colleagues over many years.”
Stephen Waxman, co-senior author on the study, shares Kocsis’ view that much more work is needed to understand how effective this therapy actually is in human patients. They are both certainly optimistic but stress it may be years before clear efficacy data would lead to clinical approvals for the therapy in other countries.
“The idea that we may be able to restore function after injury to the brain and spinal cord using the patient’s own stem cells has intrigued us for years,” says Waxman. “Now we have a hint, in humans, that it may be possible.”
The new study was published in the journal Clinical Neurology and Neurosurgery.
Source: Yale University
