New Drug Class for Treating Multiple Sclerosis

Tech ID: 30293 / UC Case 2017-382-0

Summary

UCLA researchers from the Department of Molecular & Medical Pharmacology have developed a novel drug class for the treatment of multiple sclerosis.

Background

Multiple sclerosis is a debilitating autoimmune disease that results in the demyelination of neurons in the central nervous system (CNS). γ-aminobutyric acid (GABA) is a widely used neurotransmitter in the vertebrate CNS. Although neuronal GABA-receptors have been studied for decades, only recently has it become appreciated that immune cells also express GABA-receptors that can modulate immune cell function and replication. Specifically, it has been shown that GABA administration can inhibit autoreactive Th1 and CD8+ T cell responses through the activation of immune cells. This suggest that GABA-receptor agonists may be a suitable target for suppressing the autoimmune causes of MS.

Innovation

UCLA researchers have developed a novel drug class for the treatment of multiple sclerosis targeting immune cell GABA-receptors. GABA-receptor agonists show promising immunosuppressive effects without debilitating immune system function. Orally administered GABA has little ability to pass through the blood brain barrier (BBB). Homotaurine, a natural amino acid found in algae has GABA receptor agonist properties, passes through the BBB, and was found to be safe in long-term phase III clinical trials for another indication. UCLA researchers demonstrated that oral homotaurine can induce suppressor T cells known as a CD4+ and CD8+ Tregs and can inhibit inflammatory Th17 and Th1 responses. Treatment with homotaurine suppressed the development of multiple sclerosis in mouse models even after the clinical onset of the disease. This therapeutic strategy may also be used in the treatment of other CNS inflammatory disorders.

Applications

  • Treatment of multiple sclerosis and other CNS inflammatory disorders

Advantages

  • Passes through the blood-brain-barrier 
  • Previously demonstrated safety profile 
  • Higher GABA-receptor affinity 
  • Induces CD4+ and CD8+ Treg suppressor cells
  • Suppresses Th17 and Th1 responses

State Of Development

Demonstrated preclinical success in treating two different mouse models of multiple sclerosis.

Patent Status

Patent Pending

Contact

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Inventors

  • Kaufman, Daniel L.

Other Information

Keywords

multiple sSclerosis, autoimmune, central nervous system, CNS, inflammatory disease, GABA, immune modulator, homotaurine

Categorized As

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