ALS Association Grant to Advance Treatment Aimed at Gut Microbiota

Bloom Science, a biopharmaceutical company, announced it has received a $500,000 grant from The ALS Association to advance a treatment that, by managing bacteria populations in the gut, might restore neurotransmitters in the brain to ease motor symptoms of amyotrophic lateral sclerosis

The investigative treatment, called BL-001, uses Bloom’s GOLD (Genetically Optimized Living Drugs) platform to modulate the gut microbiota — the population of bacteria and other microbes naturally present in a person’s gut.

While the exact causes of ALS are unknown, studies have linked the disease to an imbalance between two neurotransmitters, glutamate and gamma aminobutyric acid (GABA). 

Glutamate is an excitatory neurotransmitter that is responsible for stimulating motor neurons — specialized nerve cells that control muscle function — present in the brain. GABA is an inhibitory neurotransmitter that blocks the action of glutamate.

Glutamate is found at excessive levels in people with ALS, and thought to lead to prolonged nerve cell activation that damages motor neurons.

“Disrupted neurochemical homeostasis [natural balance] has been hypothesized to contribute significantly to a cascade of effects leading to neuronal death in ALS,” Christopher Reyes, PhD,  the CEO for Bloom Science, said in a press release.

In recent years, research has also shown that the gut microbiota can influence the levels of neurotransmitters in the brain via the gut-brain axis — a bidirectional communication system between the brain and gastrointestinal organs made through nerve and immune cells. 

Bloom focuses on developing therapies for people with neurological diseases using its GOLD platform, based on evidence of a connection between gut microbiota and critical disease pathways.

GOLD is used to create genetically engineered microbes, designed to be oral therapies able to influence different neurological and immune system pathways.

“We are witnessing incredible innovation in the field of microbiota-based therapeutics and research,” Reyes said. 

The grant will support further work on Bloom’s BL-001 lead program, which attempts “to modify the microorganisms in the stomach to restore the GABA and glutamate levels that are disrupted in the brains of people living with ALS,” said Kuldip Dave, PhD, vice president of research at The ALS Association.

Riluzole, the first approved ALS treatment, works to block the release of glutamate. It is now available in several forms: Rilutek, an oral tablet; Tiglutik, a liquid suspension; and Exservan, an oral film.

“Bloom’s lead program BL-001 represents a novel therapeutic opportunity; we have demonstrated in animal models an increase [in] endogenous [self-produced] GABA levels to restore a balance in the homeostatic levels of glutamate and GABA,” Reyes said.

The grant was made available through the Association’s Lawrence and Isabelle Barnett Drug Development Program. The program supports preclinical testing and development of ALS treatments with a high probability of reaching the clinic within three years.

“We are grateful to the ALS Association and the ALS community for their support to accelerate our efforts to realize a novel therapy for people living with this disease,” Reyes said.

BL-001 is also being developed to potentially treat drug-resistant epilepsies, Bloom reports on its website.