Brain Trust Bio Receives Approval for Patient Trials Targeting ALS and other CNS Diseases with Continuous Intrathecal Drug Delivery Method

The approval sets the stage for patient trials in Australia designed to enhance treatment efficacy and patient outcomes in the battle against debilitating neurological conditions, and would enable a direct progression to Phase II trials in the U.S. without repetition.

ATLANTA, March 25, 2022 – Brain Trust Bio (BTB), a leader in the development of treatments for central nervous system (CNS) diseases, has received approval to commence Phase I clinical trials in Australia for its Continuous Intrathecal Drug Delivery Method. The equivalence of these Phase I trials to American standards ensures that findings will be directly accepted in the U.S., allowing for an immediate advancement to Phase II without the need for repetition. This milestone paves the way for a pioneering approach in the management of debilitating neurological conditions, including amyotrophic lateral sclerosis (ALS), aiming to transform therapeutic standards and foster significant improvements in patient well-being.

With an unwavering commitment to innovation and patient care, BTB is at the forefront of redefining therapeutic strategies for CNS disorders. By optimizing an existing FDA-approved therapy, BTB’s patented Continuous Intrathecal Drug Delivery Method aims to significantly improve drug efficacy and patient outcomes through direct delivery of IT-Riluzole (the only drug demonstrated to prolong survival in ALS) to the CNS.

The Continuous Intrathecal Drug Delivery Method is designed to deliver therapeutic agents directly to the cerebrospinal fluid (CSF), bypassing the blood-brain barrier and allowing smaller doses to achieve greater (x5) delivery at the target site. This method not only enhances the potential for neuroprotection but also minimizes systemic side effects, offering a promising outlook for the future of CNS treatment.

“The approval to conduct these trials signifies a pivotal moment in advancing our mission, setting the stage for significant breakthroughs in treatment efficacy and patient care,” said Chen Benkler, Ph.D., Co-Founder and CEO of Brain Trust Bio. “Importantly, our proposed treatment offers unprecedented access to CSF sampling, which will enable us to provide CSF biomarker results to an extent never seen before, further enhancing our understanding and ability to treat CNS diseases.”

BTB’s proprietary approach has already demonstrated potential in early applications, showing the ability to safely deliver smaller doses to achieve greater delivery to the cells that need it. Notably, with two patients already treated for over two years without any observed side effects, there are promising indications of the treatment’s efficacy. Compared to existing methods, preliminary estimates suggest it could be approximately five times more effective, potentially extending survival from an additional three months to an additional year, or possibly even a year and a half, thereby significantly enhancing the quality of life for patients. With IT-Riluzole as the focal point of these trials, BTB is set to explore this innovative method’s full potential in altering the course of CNS diseases, including Multiple sclerosis, spinal cord cancer, and other neurodegenerative diseases.

The upcoming trials, set to involve 10 patients over a six-month period across two distinguished Australian sites, benefit from the 43.5% Australian R&D cash rebate. This strategic approach to funding exemplifies BTB’s commitment to scientific innovation and showcases its ability to navigate financial and regulatory environments effectively, propelling the development of transformative treatments. The trials will be conducted under the expertise of leading medical professionals at Flinders University, Adelaide, and Sunshine Coast Hospital, Birtinya, ensuring the highest standards of clinical investigation.

For further information and updates on the Continuous Intrathecal Drug Delivery Method and Brain Trust Bio’s pioneering work, please visit



About Brain Trust Bio
Brain Trust Bio is a next-generation biotechnology company improving upon outdated models to protect patients from avoidable neural damage from central nervous system diseases and disorders by improving the performance of already approved medicines. They do this by harnessing better drug delivery methods and allowing for medicine delivery free from the effects of metabolic differences and other inhibitors of a drug’s beneficial effects. Brain Trust Bio has entered into phase one of IND-enabling studies to secure approval to conduct in-human clinical trials.

Kissy Black
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