Experimental Therapy Halts Infant’s Severe Seizures, Dubai Hospital Reports

Rare Daily Staff

An infant who had been experiencing about 110 seizures a day became seizure-free within 30 minutes of receiving an experimental medicine for a rare genetic form of epilepsy, according to King’s College Hospital London–Dubai.

The child, identified as Adnan, received his first dose of an experimental oral treatment ABS-1230 on September 14 after seven previous treatment approaches failed to adequately control his seizures. He was discharged from the hospital a week later.

The hospital said no clinical seizures were observed during follow-up assessments through October 11 and that Adnan showed progress in growth and development. It said he remained seizure-free after that date but did not provide a more recent assessment date.

The reported response offers an encouraging example of precision medicine—using a patient’s genetic diagnosis to help select a treatment aimed at the biological cause of disease. But the account concerns a single child, and the hospital cautioned that the findings cannot be generalized to other patients.

Adnan’s seizures began in the first days after birth and grew more frequent and severe despite treatment. Doctors used whole-genome sequencing to identify a disease-causing genetic variant and investigate treatments that might address its effects. The hospital did not identify the gene or the specific variant in its announcement.

The medical team, led by Ubaid Shah, a consultant pediatric neurologist and epilepsy specialist at King’s College Hospital London–Dubai, identified ABS-1230 as a potential option. The hospital described the drug as targeting abnormal activity in an ion channel associated with Adnan’s form of epilepsy. It said the treatment is being investigated in international clinical trials.

The hospital did not name the drug’s developer, however, Actio Biosciences is developing an experimental therapy called ABS-1230 for KCNT1-related epilepsy.

Actio describes ABS-1230 as a first-in-class, orally administered, selective small-molecule inhibitor of the KCNT1 ion channel. According to the company, preclinical studies showed that ABS-1230 inhibited abnormal channel activity associated with all pathogenic KCNT1 mutations tested, suggesting potential applicability across patients with different disease-causing variants.

“This is an exceptional moment for us, and it highlights what precision medicine can achieve when we are able to identify the genetic cause of a disease and choose a treatment that targets the mechanism linked to it,” Shah said.

According to the hospital, Adnan is the youngest patient and only the second infant worldwide to receive ABS-1230. Doctors obtained the medicine through a compassionate-use pathway that allowed him to receive the experimental treatment with regulatory authorization. The hospital prepared the medical documentation, the Emirates Drug Establishment reviewed and approved the request, and the Dubai Health Authority helped coordinate the process.

The medication arrived in Dubai within weeks of the request, after the necessary medical, ethical and regulatory approvals, according to the hospital. That allowed Adnan to receive treatment locally rather than travel abroad.

Photo: Adnan, an infant with an ultra-rare, genetic epilepsy