Samba Reddy saw his professional life’s worth of work come to fruition last year.
Reddy, a researcher at Texas A&M University School of Medicine, had studied for almost 25 years on how a drug could treat patients with epilepsy. His team’s medication, ganaxolone, which is sold under the brand name Ztalmy, was approved by the Food and Drug Administration in March 2022 to treat a rare form of refractory epilepsy called CDKL5-deficient epilepsy (CDD) in children above the age of 2. The drug was designated a first-in-class medication since it was the first drug to be approved to treat this condition. The anti-seizure drug hit the market in July 2022.
CDD is a genetic disorder first identified in 2004, according to the Epilepsy Foundation. CDD is rare and occurs in just 1 in 60,000 births and affects four times as many females as males.
“The last 25 years of my life since getting a Ph.D., I’ve been working on these compounds and specifically on this molecule for nearly 18 years,” Reddy said. “We kind of gave up the hope that it would ever reach the market and be helpful to any patients, but I never gave up. A lot of companies and trials gave up, but I continued my work and I’m so thrilled that finally it saw the light and hit the market.”
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Reddy is a regents professor of neuroscience and experimental therapeutics and the founding director of Texas A&M’s Institute of Pharmacology and Neurotherapeutics. He began his research on ganaxolone in 1999 and spent the last 14 years of his research at A&M after he spent time at the U.S. National Institutes of Health (NIH) and North Carolina State University. He works out of A&M’s Health Science Center in Bryan.
The biggest turning point during Reddy’s research was finding out how neurosteroids, which are compounds synthesized within the brain Reddy wrote in an abstract rapidly modulate neuronal excitability. Reddy said he has studied how neurosteriods work in the brain since he was a post-doctoral fellow at the NIH. Once that was proven, Reddy said it was a turning point not just for him, but his entire field and might help pave the way for other neurosteroid products to be approved for use in the future.
A social stigma comes for people with epilepsy, Reddy said. Children with CDD often have seizures within the first few months of life, according to the Epilepsy Foundation. Other effects of CDD include problems with nervous system development, which includes cognitive development.
“For those people, if they can become seizure-free with this medicine or related medicines, that’s the biggest gift that research can give to them,” Reddy said. “They can get independence back.”
Moving forward, Reddy said, he has proposed new testing to extend his research and treatment for a wider range of epilepsy. Reddy’s team received a Biomedical Advanced Research and Development Authority grant worth $51 million and his team is underway with advanced clinical testing to see if ganaxolone can treat status epilepticus. He noted that the drug could become a way to treat people such as soldiers who were exposed to chemicals or traumatic brain injuries and could be a drug used by first responders during emergency seizures.
“It’s going from epilepsy to soldiers to back to the road for day-to-day care,” Reddy said.