Drug intelligence / Profile preview

GABA + GAD-alum

Development stage
Phase 1
Lead developer
University of Alabama at Birmingham
Modality
Vaccines & Immunotherapeutics, Small Molecules, Recombinant Proteins and Enzymes
Administration
Oral, Subcutaneous
01

Overview

GABA + GAD-alum is a combination therapy under investigation for the treatment of type 1 diabetes (T1D), particularly in children and newly diagnosed patients. The combination consists of oral gamma-aminobutyric acid (GABA), a major inhibitory neurotransmitter that also modulates immune responses, and glutamic acid decarboxylase 65 (GAD65) formulated with alum as an adjuvant. Mechanistically, GABA acts on immune cells to dampen proinflammatory Th1 cytokine responses and promote regulatory T cell activity, while also being abundant in pancreatic islets where it may help preserve beta-cell function. GAD65-alum serves as an antigen-specific immunotherapy designed to induce immune tolerance or shift the immune response toward a less inflammatory profile by promoting Th2/Treg-skewed immunity. Clinical trials have shown that this combination can reduce fasting and meal-stimulated serum glucagon levels but has not demonstrated significant preservation of beta-cell function at low doses in pediatric populations. The therapy is considered safe and well-tolerated, with ongoing research into higher or longer-acting formulations for improved efficacy[1][2][3].

Other names
GABA/GAD65-alumgamma-aminobutyric acid plus glutamic acid decarboxylase 65 with alum
02

Targets

GABRR (GABA-A receptor subunit rho)GABA-B (Gamma-aminobutyric acid receptor type B)GAD2 (Glutamic Acid Decarboxylase 65)

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