Indian Researcher Develops First New Medicine for Refractory Epilepsy

Telugu NRI scientist Dr. Samba Reddy, who works as Regents Professor at Texas A&M University in USA, has developed a new medicine for a type of epilepsy that was previously untreatable. After decades of tireless research and dedication, a groundbreaking new medicine for refractory epilepsy has been clinically approved by the FDA. Developed by Dr. Reddy, a renowned pharma scientist and professor at Texas A&M School of Medicine, ganaxolone (Ztalmy) has been approved for the treatment of CDKL5-deficient epilepsy, a debilitating condition that had previously been resistant to treatment. This is a transformational  step forward in the fight against epilepsy and a true testament to the impact that dedicated researchers can have on improving the lives of millions. Epilepsy is a devastating brain condition with repeated seizures (fits) and it affects 70 million people worldwide, including 7 million in India.

This incredible achievement is the result of Dr. Reddy’s extensive expertise and experience in the field of neurosteroids, a class of compounds synthesized in the brain that deeply influence brain functions and mood. Dr. Reddy is considered to be among the top 2% of scientists worldwide and has published 225 papers, five textbooks, trained 100 students/postdocs, and made 450 presentations. He has received numerous awards and honors for his iconic contributions, including the AAPS Global Leader Award in Pharmaceuticals.

Dr. Reddy and his team spent over a decade researching ganaxolone, uncovering that it binds to two types of receptors in the brain: synaptic receptors and extrasynaptic GABA-A receptors. They also found that the “calming” effect of neurosteroids is more powerful and lasts longer when they bind to the extrasynaptic receptors. With this knowledge, they conducted pilot studies in experimental models, proving that ganaxolone was effective in treating various forms of epilepsy. These results were then used to establish clinical trials in humans, which also proved to be highly successful.

Ganaxolone is not only a major breakthrough for those suffering from refractory type of epilepsy, but it also marks the second FDA-approved therapy based on pioneering studies from Dr. Reddy’s lab. In 2019, brexanolone, an injectable version of the neurosteroid allopregnanolone, became the FDA-approved neurosteroid and made history as the first and only FDA-approved treatment for post-partum depression.

Dr. Reddy’s ultimate goal is to directly see positive outcomes in patients, and with the approval of ganaxolone, that goal has been achieved. He is determined to continue his research, developing even more effective therapies and working on projects that will serve other people affected by treatment-resistant epilepsy, including brain injuries and chemical exposures in civilian and military persons.

An injectable neurosteroid therapy developed by Dr. Reddy and his team has been approved for phase-3 clinical trials for the treatment of status epilepticus, a condition that is often resistant to current medications. He is optimistic that his new research will lead to the development of more effective medicines with fewer side effects.