Novel Stem Cell Therapies

October 5, 2022 / by Max Sellman

A first look at patient data at ISSCR

I’m always grateful for the chance to hear the latest results from drug developers in the CRISPR space. That’s why it was a huge bonus to have a world class cell and gene therapy conference just fifteen minutes down the road from Aldevron Madison in September!

I was fortunate to be able to attend the ISSCR/ASGCT Joint Conference on Emerging Therapies at the Intersection of Genetic and Cellular Technologies, where one presentation that really stood out was from Neurona Therapeutics, a clinical-stage biotech based in South San Francisco that is making great progress in developing novel stem cell therapies for neurological disorders. Neurona’s Chief Development Officer, Catherine Priest, Ph.D., offered a first look at patient data from their lead clinical candidate for chronic refractory epilepsy, NTRX-1001.

Meisal Temporal Lobe Epilepsy (MTLE) is one of the most common forms of focal epilepsy, affecting more than 3.4 million individuals in the United States. Though there are several approved therapeutics for MTLE, these drugs are not very effective in controlling seizures for somewhere between 25-35% of patients.

This is the population that Neurona is seeking to treat with NTRX-1001 – patients suffering from drug resistant epilepsy. NTRX-1001 is an allogeneic cell therapy composed of MGE-type GABAergic interneurons. These neurons, derived from pluripotent stem cells, are intended to tone down overactive GABA signaling that causes refractory seizures.

Dr. Priest presented three-month follow-up data from the first patient to be treated with NTRX-1001. This patient has drug resistant epilepsy and had the average history of more than 30 seizures per month coming into the trial. The patient received a single dose of NTRX-1001 and, three months out, has experienced a total of four seizures.

So far, the treatments seem to be well tolerated by patients, so this data represented a very encouraging step towards curing drug-resistant MTLE. A big congratulations to the team at Neurona, we look forward to hearing more positive steps in the future to addressing this key unmet medical need.

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