Omni Bio Pharmaceutical, Inc. announced the publication of final results from its pilot study of AAT in patients with recent onset Type 1 diabetes (T1D) in the Journal of Clinical Endocrinology and Metabolism. The study, which was conducted under the leadership of Dr. Peter Gottlieb at the Barbara Davis Center for Childhood Diabetes, University of Colorado Denver, concluded that AAT may have a beneficial effect on T1D in recently diagnosed patients through the down-modulation of IL-1 and other pro-inflammatory cytokines in the blood cells of treated patients. IL-1b is known to be harmful to insulin-producing cells and plays a role in T1D.

The authors further concluded that targeting inflammation with inhibitors of the innate immune system, such as AAT, might represent an efficient therapeutic strategy for disease prevention. Twelve patients with recently diagnosed T1D were enrolled in the study with each receiving 8 consecutive weekly infusions of 80 mg/kg of AAT. Patients were evaluated for effects of therapy on C-peptide levels following a mixed meal glucose tolerance test as a measure of insulin-producing islet cell function, and on proinflammatory cytokine measures of innate immunity over an 18 month period post-dosing.

Key findings were as follows: AAT led to increased or unchanged levels of C-peptide responses in four patients during 18 months' follow up; the total content of TLR4-induced cellular IL-1 in monocytes at 12 months among all patients was reduced 3-fold compared to baseline (p < 0.05); Monocyte production of IL-1 was reduced from 82% at baseline to 42% at 12 months; Similar reductions were seen using TLR7/8 and TLR3 agonists in monocytes and myeloid dendritic cells (mDCs); Unexpectedly, the reduction in cellular IL-1 was observed at 9-12 months post-treatment but not in a cohort of untreated diabetics; Improved islet cell function in the four responder patients correlated with the reduced monocytes and mDC production levels of IL-1 (p < 0.04 and p < 0.02, respectively); No significant adverse effects were reported in the study.