Pathway-Specific Medicines: 21st Century Solutions to Unmet Medical Needs

Cancer remains one of the great unmet medical needs with high morbidity and mortality. Older therapies relied upon exceedingly toxic methods, such as chemotherapy and radiation, which had marginal therapeutic indices.

Since the discovery of the oncogene, biochemistry, and cell and molecular biologists have discovered a diversity of deregulated pathways in solid and liquid tumors. These discoveries have ushered in the era of patient- and pathway-specific oncology therapies, including, for example, Herceptin for breast cancer and Gleevec for chronic myelogenous leukemia.

In today’s talk, I will discuss the approaches utilized by three early-stage biotechnology companies who hope to further explore other oncogenic pathways that may be involved in a large number of tumors. Proteolix has developed a modulator of a protein-degradation pathway that has shown efficacy in hematologic tumors. Intellikine has generated an inhibitor of one of the components of the PI3 Kinase pathway, one of the most mutated pathways in both solid and liquid tumors. Finally, Oncomed has developed monoclonal antibody inhibitors of early developmental pathways, such as Notch and Wnt, in order to eliminate the cancer stem cell. Together, these three stories will highlight the newest trend in oncology: patient- and pathway-specific medicines.