PROOF OF CONCEPT – IMPROVING ON A PROVEN ALS THERAPY
At Brain Trust Bio, we are singularly focused on reformulating and improving the best drugs on the market, based on our successful implementation of riluzole administered by intrathecal infusion (ITRil) for the treatment of amyotrophic central nervous system diseases like lateral sclerosis (ALS).
In 1995, the U.S. Food and Drug Administration (FDA) approved riluzole (marketed under the brand names Rilutek and Tiglutik) for the treatment of ALS. The drug is taken orally (as a tablet or a liquid formulation) and is considered to increase survival of ALS patients by a modest two to three months. However, it does not halt or reverse disease progression, and the oral doses cannot be augmented because of the drug’s hepatic toxicity. In addition, oral riluzole can impair cognitive function and induce asthenia, which may lead to treatment discontinuation in patients who poorly tolerate these central side effects.
Brain Trust Bio hypothesized that continuous intrathecal (IT) delivery of low daily doses of riluzole could elevate spinal cord (SC) concentrations and improve the drug’s survival benefit, without increasing the risk of systemic toxicity or asthenia and without the impaired cognitive function caused by oral therapy. Due to riluzole’s low aqueous solubility, Brain Trust Bio developed and patented a novel IT formulation, thereby taking a known working drug and optimizing its complete potential.