An experimental stem cell therapy has reduced seizure frequency by almost 95 per cent in the first two epilepsy patients, according to early results of a trial. Developed by San Francisco-based biotech Neurona Therapeutics, the single dose therapy, called NRTX-1001, involves lab-grown neurons which are injected in the patients to silence the seizure activity in the epileptic region of the brain.
The regenerative neural cell therapy, NRTX-1001, was derived from human pluripotent stem cells. The preliminary data demonstrate promising reduction (90 per cent) in seizure frequency in the first and second patients at one year and seven-months post-treatment, respectively. In addition, neuropsychological testing suggests an improvement in memory after NRTX-1001 administration.
"Although our clinical investigation is ongoing in additional patients, it is gratifying to witness the first two patients achieving seizure relief without additional cognitive impairment to date," said Cory Nicholas, the CEO of Neurona Therapeutics, in a statement.
"We are hopeful that these improvements will continue in the ongoing trial," he added. The early findings were presented at the recently held International Society for Stem Cell Research's annual meeting in Boston, US.
The two patients had a history of significant monthly seizure activity that was not controlled by anti-seizure medications. The first patient, treated at SUNY Upstate Medical University, had a seven-year history of seizures, and in the six months prior to the administration of NRTX-1001 experienced an average of 32 seizures per month.
The second patient treated in the trial, at the Oregon Health & Science University, had a nine-year history of seizures and averaged 14 seizures per month in the six months prior to treatment. Both patients continue to report reduced overall seizure counts of more than 90 per cent at one year and seven-months post NRTX-1001 administration, respectively.
No serious adverse events have been reported. "Patient-one has reached the one-year post treatment endpoint and has achieved more than 95 per cent overall monthly seizure reduction, including elimination of all seizure events since the seventh month post-administration of NRTX-1001," said David Blum, Neurona's chief medical officer, in the statement.
"In addition, this patient has shown improved memory performance on cognitive tests," he added.