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I n a conference room in La Jolla, California,
Kristiina Vuori, a leading expert in cell adhesion and metastasis, discusses her team’s latest findings with colleagues. Across the country, near Orlando, Florida, robotic arms move at a steady pace, testing hundreds of thousands of compounds for their ability to bind to a protein implicated in cancer. The common link between the two is both are part of basic medical research and drug discovery efforts at Sanford-Burnham
Medical Research Institute, where Vuori serves as president and interim CEO. Sanford-Burnham is an independent, nonprofit institute focused on bridging the gap between academic inquiry and pharmaceutical development, speeding delivery of new treatments to patients.
Sanford-Burnham traces its roots to the La Jolla
Cancer Research Foundation, founded more than
30 years ago and recognized for excellence in basic biomedical research. From these beginnings, the Institute has expanded its mission and undergone a transformation. Thanks to a concerted ten-year effort, Sanford-Burnham now boasts state-of-the-art drug discovery technology, expertise across multiple therapeutic areas, and translational research capabilities.
In addition, the Institute now spans both coasts, employing 1,200 people, 900 of them scientists, many with industry experience.
The Institute’s investments in technology and a “bench-to-bedside” approach advance insights from basic research rapidly into the clinic. According to Sanford-Burnham's vice president of Drug Discovery and Development,
Michael Jackson, "Our focus is to generate firstin-class drugs that will transform the treatment of serious disease. We leverage our expertise and technology platforms to bridge fundamental and translational research." The result is a
‘hybrid’ medical research institute combining the creativity of an academic environment with rigorous industry-standard research, bringing to life the Institute’s motto, “From Research, the
Power to Cure.”
While commercial pharmaceutical companies are under pressure to invest in highly validated targets, Sanford-Burnham aims to lower the barrier for industry partners to embrace new science. The Conrad Prebys Center for Chemical
Genomics’ goal is to ‘de-risk’ candidates by identifying chemical leads that can be used to validate a drug target in animal models. This provides an attractive starting point for commercial development.
“As trail-blazers, we add value by conducting early drug discovery activities on novel drug targets that need additional validation. Although high risk, we aim to accelerate the development of transformational therapies,” said Jackson.
Historically, many drugs have been identified using phenotypic cell-based screens. With that in mind, Prebys Center researchers take a broad approach to identifying candidate drugs, with capabilities in high-content phenotypic screens rather than relying on classic high-throughput screening (HTS) of small-molecule libraries ( see box ).
Jackson began a new chapter in his drug discovery career when he joined Sanford-Burnham three years ago, after a 15-year stint at Johnson
& Johnson, where he led US drug discovery and put dozens of compounds into the clinic.
Similar to industry, Sanford-Burnham scientists manage projects in a milestone-driven process.
They work in cross-functional teams of pharmatrained medicinal chemists and pharmacologists, collaborating to move projects from target identification through early lead optimization, to validation.
According to Jackson, "We build alliances where there is a shared mission and complementary expertise, including pharma, clinical organizations, and academia." Ongoing partnerships include Janssen, Pfizer, and Takeda, as well as clinical centers like Mayo Clinic and Adventist
Health System’s flagship, Florida Hospital.
Johnson & Johnson subsidiary Janssen
Pharmaceutical Research and Development is currently collaborating with Sanford-Burnham in a three-year effort to identify lead compounds targeting novel pathways involved in Alzheimer’s disease and major psychiatric disorders like depression, schizophrenia, and bipolar disorder.
These diseases together affect over seven million Americans and, although treatment options exist, a high unmet medical need remains.
Together with Mayo Clinic, Sanford-Burnham is building a pipeline of therapeutic drugs aimed at a variety of diseases. The collaboration combines Mayo Clinic’s clinically relevant targets with Sanford-Burnham’s drug discovery platform in a translational initiative aimed at advancing a portfolio of novel therapeutics.
Conrad Prebys Center for Chemical
Genomics (Prebys Center)
 Integrated drug discovery capabilities
(assay development, HTS, hit-to-lead and lead optimization), including medicinal chemistry and pharmacology.
 Capacity of 50 HTS campaigns per year using diverse libraries that include
750,000 compounds.
 Assay and algorithm development to perform high-content, phenotypic imaged-based screens.
 Si, ShRNA, and microRNA library screening capabilities.
Stem Cell Research Center
 Generate patient-derived induced pluripotent stem cell (iPSC) lines, for example: muscular dystrophy,
Alzheimer’s, and bipolar disorders.
 Develop disease-in-a-dish assays and drug screens based on patient-derived iPSCs.
 High-throughput screen stem cellderived cultures, for example: neuron and cardiomyocytes for morphological and physiological endpoints.
In collaboration with Takeda and Florida
Hospital, Sanford-Burnham researchers are tackling obesity. The three-way partnership offers a unique model to accelerate early drug discovery, by combining basic and clinical research and pharmaceutical expertise. According to Daniel P.
Kelly, scientific director at Sanford-Burnham at
Lake Nona, “This approach allows us to identify new therapeutic targets and to evaluate clinical utility more efficiently than in the traditional drugdevelopment process.”
Michael Jackson , Ph.D.
Vice President,
Drug Discovery and Development
Sanford-Burnham Medical Research Institute
Tel: +1 858 795 5201
Email: mjackson@sanfordburnham.org
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