Can an AI-Based Drug Treat ALS?

FB1006, a novel drug that was discovered and developed using artificial intelligence (AI), may be a potential treatment for amyotrophic lateral sclerosis (ALS).

ALS, also known as Lou Gehrig disease, is a fatal motor neuron disorder characterized by progressive degeneration of nerve cells in the spinal cord and brain. It causes muscle weakness and paralysis, which lead to the eventual inability to walk, speak, swallow, and breathe. There is no cure for ALS, and the average life expectancy after diagnosis is 2 to 5 years.

FB1006, developed by 4B Technologies, is being tested for the treatment of ALS in an investigator-initiated clinical trial comprising 64 patients. The trial is being conducted at the Third Hospital of Peking University and is expected to complete double-blind dosing in August 2024, followed by 1-year clinical observation in February 2025.

AI technology was utilized in multiple stages of the drug’s discovery and development, including target identification, patient enrollment, and efficacy assessment. The use of AI enabled the investigative team to significantly shorten the patient screening and complete enrollment within 1 year, as well as maximize the use of the clinical data.

“The development of ALS medications calls for innovative approaches to accelerate clinical research,” said Dongsheng Fan, MD, PhD, a renowned ALS expert at Third Hospital and the trial’s principal investigator, via a press release. “In the clinical study of FB1006, we successfully adopted the innovative approach of a trial-ready cohort (TRC), which is established through datasets comprising patient history, blood biochemistry results, EEG/EMG, imaging, genome, and more. With the help of the ALS TRC database, we could accurately enroll patients upon clinical trial initiation, based on target and drug properties, clinical effects, and side effects, thus shortening the time and reducing the cost of patient enrollment, while increasing the success rate of the investigator-initiated trial. We look forward to the promising results of FB1006.”

Bai Lu, PhD, founder of 4B Technologies and a professor at Tsinghua University, added, “We are pleased to see that 4B Technologies completed the whole development process of FB1006, from target identification and compound screening to patient enrollment, in less than 2 years with the joint efforts. We expect that these explorations into innovative concepts, technologies, and modes will help overcome the challenges of ALS and benefit patients worldwide.”

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