Sunday, July 29
Opening Plenary Session
Imatinib as a Paradigm of Targeted Cancer Therapies
Brian Druker, MD
Director, Knight Cancer Institute, Oregon Health & Science University
JELD-WEN Chair of Leukemia Research
Investigator, Howard Hughes Medical Institute
This session shows how to translate knowledge of the molecular pathogenesis of cancer into specific therapies and investigate the optimal use of these molecularly targeted agents. Dr. Druker revolutionized the treatment of cancer through research that resulted in the first drug to target the molecular defect of a cancer while leaving healthy cells unharmed. Imatinib (marketed as Gleevec) turned a once-fatal cancer, chronic myeloid leukemia, into a manageable condition. Imatinib received Food and Drug Administration approval in record time and established Dr. Druker as a pioneer in the field of precision medicine. Most importantly, his discovery became a new proof of principal for targeted therapies, spurring the development of more than 50 similar precision therapies for other cancers.
Monday, July 30
Genetic Defects in Bile Acid Synthesis Causing Liver Disease - Diagnosis and Treatment - Translational Medicine from Mass Spectrometry Discovery to the Bedside
Kenneth Setchell, PhD
Director, Mass Spectrometry Lab
Professor, University of Cincinnati Department of Pediatrics
Cincinnati Children’s Hospital Medical Center
This session highlights how mass spectrometry was successfully applied to define new genetic defects in the bile acid biosynthetic pathway. Bile acid synthesis disorders caused by single enzyme defects often present in infancy or early childhood with a progressive cholestatic hepatitis that, if unchecked, leads to cirrhosis, liver failure, and death. Prior to the seminal work of Dr. Setchell and colleagues that identified six genetic diseases as discrete entities and conceived of an effective therapy, children with these autosomal recessive diseases either underwent liver transplantation, or more commonly, were given supportive care until they died of liver failure of unknown origin. The session describes the use of mass spectrometry techniques that led to the elucidation of the biochemical basis of these diseases, the development of an international screening program, and the evaluation of therapeutic responses that served to ultimately gain regulatory approval from the Food and Drug Administration for a life-saving therapy based on oral administration of cholic acid. This application of mass spectrometry to clinical chemistry is a noteworthy example of the transition from bench to bedside.
Tuesday, July 31
HPV Associated Cancers and the HPV Vaccine
Denise Galloway, PhD
Director, Pathogen-Associated Malignancies Integrated Research Center
Fred Hutchinson Cancer Research Center
This session highlights the discovery that human papillomaviruses (HPVs) cause cervical and other cancers. In just 25 years, this discovery led to the development of HPV vaccines (Gardasil 9 and Cervarix). Dr. Galloway will review the history of HPV vaccine development, especially the work needed to meet Food and Drug Administration regulations and the importance of achieving herd immunity. Future work includes improving efficacy, assessing the adequacy of initial vaccination, vaccinating males, assessing the need for boosters, reducing cost, and improving international availability.
Wednesday, August 1
Nucleic Acid Detection Using CRISPR-Dx
James Collins, PhD
Termeer Professor of Medical Engineering & Science
Professor of Biological Engineering
Massachusetts Institute of Technology
Founding Core Faculty & Lead, Living Cellular Devices
Wyss Institute at Harvard University
This session reports on the discovery that CRISPR-Cas13a/C2c2 can be used for the rapid, reliable, inexpensive detection of nucleic acid sequences. This technique achieves single-base specificity in detection of specific RNA or DNA variants. The proof of concept experiment used fragments of the Zika virus genome spliced into a lentivirus and achieved detection down to 1,000 copies per mL (2 attomolar). Dr. Collins and his colleagues have coined the technique SHERLOCK (Specific High-sensitivity Enzymatic Reporter unLOCKing).
Thursday, August 2
Essential Diagnostics: Meeting the Needs of a Global Population
Timothy Amukele, MD, PhD
Medical Director, Makerere University-Johns Hopkins University Research Collaboration Core Laboratory
Assistant Professor of Pathology
Johns Hopkins School of Medicine
Lee Schroeder, MD, PhD
Director of Point-of-Care Testing
Associate Director of Chemical Pathology
University of Michigan
While medicines treat disease, diagnostics find disease. Yet, in global health initiatives, diagnostics receive much less attention. The World Health Organization’s Model List of Essential Medicines has been critical to the efficient delivery of medicines. This session will describe how a Model List of Essential Diagnostics will help strengthen laboratory capacity in resource-poor settings.