for people ages 18-65 (full criteria)
at San Francisco, California and other locations
study started
completion around
Principal Investigator
by Robert C Knowlton, MD
Headshot of Robert C Knowlton
Robert C Knowlton



This clinical trial is designed to test whether a single stereotactic intracerebral administration of inhibitory nerve cells into subjects with drug-resistant mesial temporal lobe epilepsy is safe (frequency of adverse events) and effective (seizure frequency).

Official Title

A First-In-Human (FIH) Study of Inhibitory Interneurons (NRTX- 1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)


Subjects will undergo a single stereotactic intracerebral administration of neural cells, called interneurons, that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA).

Subjects will then take medicines to partially suppress their immune system (aimed to prevent the body from rejecting the cells) for 1 year. Safety, tolerability, evidence of neural cell viability and local inflammation (using MRI scans of the brain), and effects on epilepsy disease symptoms will be assessed for 2 years post-transplant. Subjects will be followed for an additional 13 years with quarterly phone contact and annual visits.


Mesial Temporal Lobe Epilepsy With Hippocampal Sclerosis, Epilepsy, Temporal Lobe Epilepsy, Hippocampal Sclerosis, NRTX-1001


You can join if…

Open to people ages 18-65

  1. Male or Female, age ≥18 to 65
  2. Focal seizures, clinically defined as TLE
  3. Has failed to achieve seizure control despite adequate trials of at least 2 ASDs at appropriate doses
  4. Currently on stable doses (at least 1 month) of approved ASDs
  5. Single seizure focus confirmed as within one temporal lobe
  6. For subjects entering Stage 1, the seizure focus is either a) in the non-dominant hemisphere, or b) in the dominant hemisphere and the subject has a Rey Auditory Verbal Learning Test (RAVLT) assessed within one year of screening, or at the screening visit, that is at least 1.5 standard deviations below the population mean.
  7. Seizure frequency averages ≥2 per 28-day period over the 6 months prior to screening.

You CAN'T join if...

  1. Epilepsy due to other and/or progressive neurologic disease
  2. Significant other medical condition which would impair safe participation
  3. Primary or secondary immunodeficiency
  4. Suicide attempt in the past year
  5. Severe psychiatric disorders
  6. Chronic indwelling intracranial device
  7. MRI indicating potential malignant lesion
  8. Pregnancy, or currently breastfeeding


  • UCSF accepting new patients
    San Francisco California 94143 United States
  • Stanford University accepting new patients
    Palo Alto California 94304 United States

Lead Scientist at UCSF

  • Robert C Knowlton, MD
    Dr. Robert Knowlton is a neurologist who specializes in diagnosing complex cases of epilepsy, such as those with no clear origin found using standard tests. In his clinical practice, Knowlton combines a variety of advanced diagnostic tests to pinpoint the source of seizures in the brain.


accepting new patients
Start Date
Completion Date
Neurona Therapeutics
Phase 1/2 Epilepsy Research Study
Study Type
Expecting 40 study participants
Last Updated