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Graduate Student Sany Hoxha: Captivated by Complexity



Of Note

TSRI Screening Center Helps Advance Drug Candidate to Clinical Trials

The Molecular Screening Center at the California campus of The Scripps Research Institute (TSRI) has played an essential role in advancing to clinical trials a new compound to treat multiple myeloma, the second-most common form of cancer of the blood.

The compound, CB-5083, is produced by Cleave Biosciences, a biotechnology company headquartered in Burlingame, California. CB-5083 is a first-in-class, oral inhibitor of p97, a critical enzyme that controls various aspects of protein homeostasis.

Under the auspices of a National Institutes of Health (NIH) Molecular Libraries Screen Centers Network grant, spearheaded by TSRI Professor Hugh Rosen and in collaboration with Professor Raymond Deshaies of the California Institute of Technology, TSRI scientists screened an NIH network library of small molecules (nearly 220,000 at that time) searching for potential p97 inhibitors. They narrowed the field to the top 50 candidates—cutting years off the research process.

The CB-5083 Phase I clinical trial will evaluate the compound’s safety and efficacy in multiple myeloma patients with relapsed/refractory or refractory disease who have not responded to other forms of therapy. According to the National Cancer Institute, an estimated 70,000 people are living with multiple myeloma in the U.S., and approximately 24,050 new cases will be diagnosed this year. 

Karsten Sauer and Stephanie Rigaud Receive Immunology Award

TSRI Associate Professor Karsten Sauer and Stephanie Rigaud, research associate in the Sauer lab, have received a Careers Immunology Fellowship award from the American Association of Immunologists (AAI), a 101-year-old international organization of scientists dedicated to advancing the knowledge of immunology and its related disciplines. 

The supported project studies signal transduction mechanisms that ensure the generation of a pathogen-reactive but self-tolerant T cell repertoire. Defects in these mechanisms cause immune deficiencies and autoimmune diseases such as rheumatoid arthritis or type 1 diabetes. By elucidating the underlying mechanisms, the Sauer lab hopes to ultimately develop improved therapies for these serious and unpreventable diseases.

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