What is Precision Medicine?

Precision medicine takes into account the precise information about the variability of each individual’s genes, proteins, environment and life style for the prevention, diagnosis, and treatment of human diseases.  The human body consists of about 37 trillion living cells, each containing about 23,000 genes.  No two humans are genetically identical.  Even monozygotic twins have genetic differences due to mutations, genetic rearrangements and polymorphisms during development.  As we uncover the mechanism on how the genome affects health, disease, and response to drugs, it becomes clear that we must take into account individual differences in each person’s genes, environment and lifestyle for better disease prevention and treatment.  Most of current medical treatments have been designed for the “average patient”.  This “one-size-fits-all” approach inevitably leads to successful treatments for some patients but not for others. Precision medicine, also known as personalized medicine, was proposed to fill in the gap. The advancements in genomic sequencing technology and rapid reduction in sequencing cost are leading to widespread utilization of patient genomic information and paving the way for a new era of precision medicine.  The Precision Medicine Initiative launched by President Obama in his State of Union Address in January 2015 provides another significant push in facilitating research, development, innovation as well as regulatory modernization for precision medicine.

 

The paradigm of cancer treatment has already shifted toward precision oncology.  Patients are selected for therapy using predictive biomarkers, such as oncogenic mutations, rather than using empiric chemotherapy.  Currently, patients with breast cancer, lung cancer, colorectal cancer, melanoma, and leukemia routinely undergo molecular diagnostic testing as part of patient care, enabling doctors to choose more precise treatments that improve chances of survival and reduce adverse effects.  High-throughput genomic technologies and programmatic sequencing efforts, such as NCI/ National Human Genome Research Institute Cancer Genome Atlas Network, and the Sanger Cancer Genome Project have generated the molecular profiles of numerous cancers, and identified actionable targets for therapeutic intervention.  Targeting the oncogenic driver mutations has been a proven effective therapeutic strategy to control tumor growth and disease progression.  Trastuzumab (Herceptin®), the first genotype-guided therapy for the treatment of HER-2 positive metastatic breast cancers was approved in 1998.  Identification of the causative genetic lesion BCL-ABL in chronic myelogenous leukemia (CML) resulted in the breakthrough medicine imatinib (Gleevec®), a tyrosine kinase ABL inhibitor for the treatment of CML in 2001. Cancer patients and oncology community expect to see many more discoveries leading to novel treatments for the unmet medical needs.  With in-depth knowledge, enriched experience and successful track record in the marketed precision oncology medicines (XALKORI® and SUTENT®), our TP Therapeutics (TP) discovery and development team led by Dr. J. Jean Cui are well-positioned to discover and develop more effective targeted therapy drugs and make significant contributions in the new Era of Precision Medicine.