Researchers present advanced drug discovery technology at the “Stem Cell Meeting on the Mesa”

By Susan Gammon, Ph.D.
October 15, 2013
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On October 14-16, 2013, two Sanford-Burnham researchers participated in the world-renowned “Stem Cell Meeting on the Mesa” held each year in La Jolla, Calif. The two scientists are presenting Sanford-Burnham’s unique capability of combining induced pluripotent stem cells (iPSCs) with ultra-high-throughput screening capabilities at the Institute’s Conrad Prebys Center for Chemical Genomics to discover molecules that effect cell differentiation and function. The molecules discovered, combined with iPSC technology, represent a promising approach to restoring, repairing, and regenerating damaged tissue in the human body.

In a partnership forum session titled “Cell and Tissue Models in Drug Discovery and Development,” Anne Bang, Ph.D., director of Cell-Based Disease Modeling and Screening in the Prebys Center, presented a case study based on work performed in collaboration with Cellular Dynamics International.

The presentation illustrated how neurons generated from human induced pluripotent stem cells (hiPSCs) were used to screen over 5,000 bioactive compounds to identify molecules that influence neurite formation. Neurites are the projections from neurons that serve to wire up the nervous system.

“The technology presented in this case study has the potential to identify new molecules that affect neurite differentiation. However, the major goal of the project, in support of the Prebys Center’s drug discovery mission, was to develop a technology platform that can be used to perform drug screens on hiPSC-derived neurons from patients with neurological disorders such as Alzheimer’s and Parkinson’s disease,” said Bang.

In the scientific symposium session titled “A Chemical Approach to Regenerative Medicine,” Mark Mercola, Ph.D., professor and director of the Muscle Development and Regeneration Program at Sanford-Burnham, will present the results of a study performed to discover drug targets and RNA therapeutics for heart failure.

The study shows how a high-throughput chemical and functional-genomics screening strategy using iPSC-derived cardiac myocytes—heart muscle cells—identified a number of molecules that modulate cell differentiation and contractility. By inhibiting one of these molecules with antisense RNA, the scientists were able to halt the progression of heart failure and restore cardiac functions in mouse models of heart failure.

“There is an urgent need for therapies that reverse the course of ventricular dysfunction in heart failure, the leading cause of morbidity and mortality in the United States. Using the Prebys Center’s ultra-high-throughput screening technology, our efforts to discover a drug that rescues heart-muscle activity have been productive,” said Mercola.

The Stem Cell Meeting on the Mesa is a world-renowned event examining the scientific, ethical, and translational challenges facing stem-cell research today. The meeting brings companies, investors, research institutes, government agencies, and medical philanthropies together to share knowledge, establish relationships, and form partnerships.

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Susan Gammon, Ph.D.
Susan Gammon, Ph.D.

Susan is an associate director of Communications at Sanford-Burnham.

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