What was the specific context that brought the company to life and what is the mission that guides your efforts?

Neurona Therapeutics was founded based on discoveries made by me and three co-founders who are professors of neurology, neurosurgery, and psychiatry at the University of California, San Francisco. Our team was among the first to discover that inhibitory nerve cells that serve to balance the activity of excitatory nerve cells, originate in a transient structure at the base of the brain that is lost in adults, called the medial ganglionic eminence (MGE). These MGE-derived inhibitory cells are essential for regulating neural circuits and are known to be affected in multiple neurological disorders, including epilepsy. We demonstrated in preclinical models that nerve cells from the MGE can be injected into a brain in which the inhibitory cells are damaged and can repair the damage and rebalance the affected activity. We had the idea to develop a unique off-the-shelf cellular therapy that replicated the cells from the MGE and could be used to treat disorders such as epilepsy.  We have been working for several years to develop a process to coax human pluripotent stem cells into these specific inhibitory nerve cells on which we have done extensive preclinical testing. This is the genesis of our NRTX-1001 program which is currently being evaluated in people with drug-resistant focal epilepsy. 

Which portions of the population tend to benefit from the therapies that you are now developing?

Epilepsy is the fourth most common neurologic disease in the world, affecting over three million people in the United States alone.

One-third of people with epilepsy are drug resistant, and while a surgical option to remove or laser ablate a part of their brain can be offered, there can be serious irreversible side effects on the person's memory, mood, or vision. Furthermore, many people are ineligible for such an intervention because the affected part of the brain is not surgically tractable. People with drug-refractory epilepsy who are not inclined or eligible for traditional brain surgery have few effective therapeutic options and could instead be good candidates for a regenerative cell therapy approach like NRTX-1001.

We began the NRTX-1001 trial last year in people with the most common type of focal epilepsy, mesial temporal lobe epilepsy (MTLE) and have already treated two patients in what is the world's first human cell therapy trial for epilepsy. The goal is to implant these inhibitory nerve cells in the region of the brain where seizures begin in order to prevent them from occurring. Early results have shown that we were able to reduce more than 90% of the seizures to date, while completely eliminating the more disabling impaired awareness seizures in one patient. In addition to epilepsy, we believe that the NRTX-1001 cell therapy could be applied to any disease with a neuro-hyperactive cause, ranging from chronic neuropathic pain to neurodegenerative diseases such as Parkinson's or Alzheimer's.

What are the preferred ways to fund Neurona’s programs?

We are a privately held company that has primarily raised capital through private venture capital equity financing, but we have also received funds through government grants. Fortunately, there is a special initiative in California, the California Institute for Regenerative Medicine (CIRM), that financially supports regenerative cell therapy research and development, and we have been fortunate to benefit from this incentive. 

What are some milestones that you are keen to reach in the coming two or three years?

Our plan is to enroll the first cohort of five patients in our ongoing NRTX-1001 clinical trial in 2023, and if all continues to go well, we will then move to a higher dose and enroll another five patients.  We currently only enroll adults into our trial whose epilepsy is focused on one side of the brain, however, it can occur in both sides of the brain, and we are looking to expand our clinical studies into this specific sub-type. Eventually we’d also like to be able to develop our therapy for use in children.

Epilepsy significantly impacts a person’s life and day to day activities, and when seizures go untreated, there is a higher risk of dementia, Alzheimer's, depression, anxiety, and ADHD, so epilepsy is a major public health issue. With this always top of mind, our mission is to continue to work hard to advance NRTX-1001, which offers a regenerative cell therapeutic option that has the potential to significantly improve the quality of life for people with epilepsy and possibly other neurological disorders.