Drug Platform for L-Asparaginases for the Treatment of Leukemias

Application

A novel humanized therapeutic L-asparaginase compound to treat acute lymphoblastic leukemia (ALL) and other cancers.

Key Benefits

• A novel drug discovery platform for developing asparaginase-based compounds to treat leukemias and other cancers.
• The derivatives could exhibit less immunogenicity and systemic toxicity than FDA-approved asparaginase medications.
• Potential to improve the treatment of leukemias while reducing the severe and life-threatening toxicities experienced by patients.

Market Summary

Leukemia is a class of malignant transformations and proliferation of blood cells in the bone marrow, blood, and extramedullary sites, with over 60,000 newly diagnosed and 24,000 deaths yearly. The disease is a common malignancy in children and adults and can lead to a devastating disease. Some forms of leukemia are particularly aggressive and lack effective treatment options. For instance, in Acute Lymphocytic Leukemia (ALL), over 6,600 newly diagnosed and 1,500 deaths yearly. Although 80% of ALL occurs in children, it represents the second most common leukemia in adults and results in devastating disease. Several asparaginase therapies are approved for various leukemias but exhibit severe toxicity and hypersensitivity reactions in about 30% of patients.

Technical Summary

Emory researchers have developed a novel in silico drug discovery platform for developing next-generation asparaginase therapies. The approach uses ancestral sequence reconstruction (ASR) to identify novel asparaginase sequences that can be rapidly screened for potential efficacy in humans. Already, the inventors have identified ten ancestral L-asparaginase sequences with a sequence identity ranging from 81% - 98% compared to humans.

Developmental Stage

Preclinical stage of development.