BrainStorm Therapeutics fuses AI and brain organoids to tackle neurological disorders

BrainStorm Therapeutics is pioneering a revolutionary approach to treating neurological disorders that affect over a billion people worldwide. The San Diego startup combines AI-powered drug discovery with organoid technology—creating miniature 3D brain cell structures from patient stem cells—to accelerate the development of treatments for conditions ranging from Alzheimer’s to rare neurological diseases. This “lab in the loop” methodology, where AI and laboratory experiments inform each other, represents a significant advancement in addressing the brain’s complex biology and could dramatically reduce the 93% failure rate of neurological drug candidates in clinical trials.

The big picture: BrainStorm Therapeutics is using a hybrid approach combining AI and brain organoids to develop treatments for neurological disorders that affect over a billion people globally.

• The company’s “lab in the loop” method uses tiny 3D bundles of brain cells created from patient stem cells alongside AI models to accelerate drug discovery.
• This approach aims to solve a critical problem in neurological medicine, where over 93% of drug candidates currently fail during clinical trials.

What they’re saying: Scientists behind the venture believe this combination of technologies could finally crack the code of the brain’s complex biology.

• “The brain is the last frontier in modern biology,” explained Robert Fremeau, BrainStorm’s founder and CEO and former scientific director in neuroscience at Amgen.
• “By combining our organoid disease models with the power of generative AI, we now have the ability to start to unravel the underlying complex biology of disease networks.”

How it works: BrainStorm’s technology creates personalized brain cell models that can accurately represent specific neurological conditions while using AI to identify potential treatments.

• The company develops organoids – miniature 3D structures of brain cells – derived from patient stem cells that mimic specific disease states.
• These living models are paired with NVIDIA’s BioNeMo generative AI platform to identify promising drug candidates by analyzing patterns in biological data.
• The iterative process allows experimental results to continuously refine AI predictions, creating a feedback loop that improves accuracy over time.

Why this matters: The approach could revolutionize treatment development for neurological conditions by making it faster and more economically viable to develop therapies for both common and rare disorders.

• Traditional drug development for brain diseases is particularly challenging due to the complexity of the central nervous system and limited access to living brain tissue.
• By identifying therapeutics that can be applied to multiple diseases, BrainStorm could create economically viable treatments for rare conditions that might otherwise be neglected.

Behind the innovation: BrainStorm leverages cutting-edge computational technology to process the complex biological data needed for drug discovery.

• The company uses NVIDIA’s accelerated computing platforms and AI models trained on biological data to identify patterns in disease networks.
• BrainStorm is also working with the CURE5 Foundation, which is developing a resource-sharing platform to help researchers studying rare neurological disorders.