Starting up Science: From Lab to Therapy

John C. Malone Professor of Molecular, Cellular, and Developmental Biology, and Professor of Chemistry, of Pharmacology, and of Management at Yale University, New Haven, Connecticut, USA

Craig M. Crews is a pioneer in the field of controlled protein degradation. His early research, which was focused on blocking the cell’s protein degradation machinery, led to an FDA-approved drug used to treat multiple myeloma. More recently, Craig M. Crews conceived and demonstrated proof-of-concept of an entirely new approach to control protein levels in cells: the so-called PROTAC technology (PROTteolysis TArgeting Chimera). PROTACs activate a cell’s quality control system to target disease-causing proteins for degradation, paving the way for entirely new treatment options for various diseases. The first PROTACs are already in clinical trials by the Yale-based company Arvinas, which Craig M. Crews founded a few years ago. They target the androgen and estrogen receptors in patients with metastatic prostate and breast cancer.

CEO of ActiTrexx GmbH and Department Head of Skin and Lymph Node Sonography at the University Medical Centre of the Johannes Gutenberg University in Mainz, Germany

Andrea Tüttenberg has shown that malignant melanoma can be fought by loading a special type of immune cells from the blood with tumour elements and reintroducing these cells into the body as a vaccine. This enables the immune system to recognize the tumour cells as foreign and kill them. Together with colleagues in Mainz, Andrea Tüttenberg also developed a new strategy for treating graft-versus-host disease (GvHD), which can occur after transplanting blood stem cells in leukemic patients. Her approach exploits the body’s own ability to maintain immunologic tolerance using regulatory T cells and thus avoids the severe side effects of conventional GvHD treatments, such as infections and relapse of leukemia. At ActiTrexx, she is developing this approach further to enable the safe transplantation of blood stem cells and new options to treat autoimmune diseases.