Methods of treatment in diseases treated with therapeutic proteins

Unmet Need

The challenge of antibody formation against protein replacement therapies (PRT) significantly impacts the efficacy of treatments as patients often develop anti-drug antibodies, particularly those who are CRIM-negative, leading to adverse outcomes and limiting the effectiveness of enzyme replacement therapy. Current methods for managing these antibody responses are inadequate, necessitating improved strategies to enhance patient outcomes.

Description of the Technology

Duke inventors have developed a method to reduce or prevent antibody titers in patients undergoing protein replacement therapy or gene therapy. The method utilizes a proteasome inhibitor that effectively decreases antibody titers by eliminating both short- and long-lived plasma cells, which are the primary sources of antibody production. This approach aims to facilitate the efficacy of protein therapies and improve clinical outcomes in patients receiving enzyme replacement or gene therapy.

Advantages

• Reduces antibody titers, enhancing the effectiveness of PRT and gene therapy.
• Targets both short- and long-lived plasma cells, addressing the root cause of antibody formation.
• Offers a potential solution for CRIM-negative patients at high risk of developing adverse antibody responses.
• Can be combined with other therapeutic modalities for improved treatment outcomes.
• May provide a favorable risk-benefit ratio in managing severe antibody responses.
• Supports improved quality of life for patients by increasing the efficacy of existing therapies.