Annette Adkins doesn’t think of herself as a medical trailblazer.
Yet, that’s exactly what she may be. As one of the first two participants in the country to undergo a new type of brain cell therapy procedure to treat an intractable form of epilepsy, the 59-year-old Portlander has been almost seizure-free since she underwent the experimental procedure on Oct. 26 at Oregon Health & Science University.
“This is wonderful,” she said. “I’m able to do things without being concerned that a seizure is going to happen while I’m out and about. I’m going back to a more normal life.”
Previously, she endured about 14 seizures a month with no relief from medication.
The procedure involves injecting inhibitory nerve cells, called interneurons, into a precisely defined focal point in the brain to quell seizures. Known as NRTX-1001 neuronal cell therapy, the surgeon injects high-purity inhibitory neurons — derived from human stem cells — into the brain.
Her surgeon, Kim Burchiel, M.D., professor of neurological surgery in the OHSU School of Medicine, is accustomed to pioneering advancements in health care. Three decades ago, he was the first surgeon to bring deep brain stimulation to the United States for the treatment of tremor associated with Parkinson’s disease.
This new intervention was first established in animal models, including nonhuman primates at OHSU’s Oregon National Primate Research Center. Burchiel said it will take more time and more patients to know whether the procedure is truly effective, but in the meantime, he is gratified that Adkins has found some relief.
“She deserves an enormous amount of credit to do something that effectively has never been done before,” Burchiel said. “We’re just at the beginning. Even though the animal research looks very robust and durable, eventually someone has to be the first patient. That requires a lot of bravery and optimism on the part of the patient.”
Adkins is one of two patients to undergo the procedure; the first was last year at SUNY Upstate Medical University in New York. Neurona Therapeutics, a San Francisco-based biotherapeutics company, developed NRTX-1001 and is organizing the multisite trial.
During the American Academy of Neurology annual meeting last month, the research team reported that both participants had undergone the procedure safely, and both had a greater than 90% reduction in seizure frequency.
“Annette is doing much better than we might have guessed,” David Spencer, M.D., professor of neurology in the OHSU School of Medicine and lead investigator for the clinical trial at OHSU. “It’s encouraging. The more time that goes by, the more data we have to understand whether this works.”
Most people with epilepsy are able to reduce or eliminate seizures through the use of medication. However, medication is insufficient for about a third of the estimated 50 million people worldwide living with epilepsy.
For people with drug-resistant epilepsy, surgical intervention can include implanting an electrical stimulator to stop seizures or removing a small portion of the brain where seizures emanate.
In contrast, the new procedure is considered a regenerative alternative.
“This is a novel, non-destructive approach to treating certain forms of seizures, which I do believe is clearly the future,” Burchiel said. “The objective is not to remove things, but to implant these cells that control the seizures without having to remove brain tissue.”
Neurocognitive tests conducted on the first two participants in the ongoing clinical trial reveal no cognitive impairments and improved memory scores so far.
The procedure involves a one-time infusion of interneurons that produce a type of neurotransmitter known as GABA — gamma-aminobutyric acid — that blocks overactive impulses between nerve cells in the brain. It’s done while the patient is under general anesthesia, with a small incision and a relatively quick recovery.
“She went home the next day,” Burchiel said.
Today, Adkins is nearly seizure-free and said she is continuing to improve with other symptoms such as fatigue — which her physicians believe may be related to immunosuppressant medications that are necessary while the new cells in her brain form long-term connections, but can be discontinued after a year. Before her epilepsy diagnosis, Adkins was accustomed to backpacking and walking up to 10 miles a day; she’s currently walking up to three miles daily.
“I’m one of these people who can’t sit still,” she said. “I’m able to hike for many more miles now.”
As a trained pharmacist, Adkins had to step back from her career due to unpredictable seizures that began in 2014, following a viral infection. With her continued recovery, she has recently enjoyed getting to see her 1-year-old granddaughter in person. She and her husband, Brad Adkins, met as teenagers growing up in Central Oregon, and the couple are already making plans that would not have been possible with the seizures.
Brad, a retired nurse, is working on a tiny home on wheels, complete with exquisite wooden and copper railing; an interior stairway stowed on a pulley system; composting toilet; troughs and pipes to capture rainwater; and elaborate detailing inside and out of 360 square feet of living space. Eventually, the couple plans to situate the home on a large property and use it as a base of operations while they travel the country.
“This is our dream,” she said.
Thanks to her courage as a trailblazer willing to embark on a new medical intervention at OHSU, that dream may soon become a reality.
The clinical trial is funded in part by the California Institute for Regenerative Medicine.