Drug Discovery and Therapeutics

 

Finding new cures for children with cancer and life-threatening diseases is central to the St. Jude mission. Working closely together, our scientists and clinicians translate laboratory breakthroughs into novel treatment options for patients at St. Jude—and around the world.

   

Small molecule discovery

From high-throughput screening to tailored molecular design, our investigators pursue the full spectrum of approaches to identify small molecules with excellent therapeutic potential. On-campus resources include:

A unique small molecule library with more than 800,000 carefully curated, purified compounds. The library is one of the largest at any academic institution and continues to expand. To date, St. Jude chemists have synthesized more than 50,000 of these compounds. 

One of the world's best “repurposing” collections of FDA-approved drugs. This collection includes the active ingredients from most approved drugs and many developmental therapeutics. These drugs are available to rapidly explore for new uses in treating childhood diseases.

A high-throughput compound screening facility focused solely on generating new treatments for children. The robots—each named by a St. Jude patient—can screen 50,000 compounds for bioactivity per day.

An extraordinary culture of collaboration. The St. Jude campus and culture have been deliberately designed to spark connections between clinicians and scientists to give drug discovery projects the greatest chance of real-world success. 

 

Vaccines, gene therapy and other biologics

Many effective therapies involve biological products. To serve our patients and investigators, St. Jude was the first pediatric cancer research center in the U.S. to open an on-site current Good Manufacturing Practice (GMP) facility. 

The diverse biological products manufactured in the facility include:

  • Novel vaccines
  • Gene therapy products
  • Monoclonal antibodies
  • Recombinant proteins

The GMP facility and expert staff provide St. Jude investigators a key advantage: the ability to develop and produce innovative treatment options for patients that may not find an initial investment by a major pharmaceutical company. 

St. Jude also supports scientific and clinical researchers with a dedicated Vector Development & Production facility, which produces viral vectors for basic research and early development of gene therapy vectors for clinical studies. Gene therapy vectors are transferred to the GMP facility for process development, scale-up and cGMP manufacturing in support of clinical studies.

 
 

Toward precision medicine

Each child is unique, and every cancer is different. At St. Jude, we are pioneering precision medicine for pediatric cancer patients. The cornerstone of this effort is our leading expertise in key fields: next-generation genome sequencing, cancer predisposition and pharmacogenomics. 

For example, through the St. Jude—Washington University Pediatric Cancer Genome Project, St. Jude investigators have successfully pinpointed the genetic factors behind some of the toughest pediatric cancers and revealed novel treatment leads. 

St. Jude is developing a state-of-the-art clinical genomics program to translate these discoveries into the new gold standard of patient care. Our vision is that clinical genomics will one day inform every step along the continuum of care at St. Jude, including:

  • Making more accurate diagnoses 
  • Better predicting how a tumor will respond to a therapy
  • Better predicting side effects
  • Developing targeted therapies aimed at a cancer’s genetic weak spots while sparing healthy tissue
  • Detecting DNA changes early that confer resistance to therapy
  • Predicting inherited susceptibility to cancer before cancer has formed to improve monitoring and prevention
 
 

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