Update on the Important Work of the Celiac Sprue Research Foundation

Celiac.com 02/11/2023 - The Celiac Sprue Research Foundation has opened its research laboratory in Sunnyvale, California at the dawn of the new year. Foundation Scientific Director, Gary M. Gray, M.D., is working closely with founder Chaitan Khosla, Ph.D., in establishing the Foundation’s drug development programs and priorities. Dr. Gray also supervises two Research Associates, Qing Li, Ph.D., and Thomas Marti, Ph.D., and Clinical Associate, Gail G. Pyle, M.D. Blair W. Stewart is Vice President and General Counsel of the Foundation.

Dr. Li received her Ph.D. in Biophysical Chemistry from Stanford University in 2002 after studying at Nanjing University and North Carolina State University. She has authored papers in protein chemistry and structural biology. Dr. Marti received his Ph.D. in Organic Chemistry from the Swiss Technical Institute (ETH) in Zurich, and performed postdoctoral research at Stanford University between 1998 and 2000. From 2000 through 2002 he worked as a management consultant at McKinsey & Co. in their Zurich offices. A Celiac herself, Dr. Pyle received her M.D. from Stanford School of Medicine. The mother of two young children, she is a Board Certified Internist, and practiced Primary Care Internal Medicine in the Stanford area for several years before joining the Foundation.

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Promoting the development of a safe and effective therapeutic alternative to a lifelong gluten-free diet is the number one goal of the Celiac Sprue Research Foundation. Toward this end the Foundation’s current efforts are predominantly focused on laying the groundwork for a proof-of-principle clinical trial that will test whether dietary gluten can be detoxified via treatment with a prolyl endopeptidase enzyme. This work builds on recent studies by Drs. Gray, Khosla and their collaborators from Stanford University and the University of Oslo, published last year (Science, 297, 2275-2279, 2002).

A key prerequisite for this trial is the production of a recombinant enzyme, produced in bacteria. To do so Drs. Li, Marti, and Khosla recently installed two fermentors at the Foundation’s laboratory. These fermentors, on loan from Kosan Biosciences, Inc., a Hayward, California-based biotechnology company, will enable CSRF to produce intermediate amounts of the enzyme for research using advanced recombinant biotechnology methods. Later this year, production of the enzyme will be scaled up to the larger quantities needed for the proof-of-principle clinical trial.

The bacteria are grown in the fermentor in a water-based environment that requires careful control of such variables as temperature, oxygen level, and nutrients. Drs. Li, Marti, and Khosla worked long into the night setting up and calibrating pumps, instruments, air and water supplies and an automatic control system. Actual fermentation began in early March and samples of the active enzyme have been produced in the Foundation laboratory.

Dr. Marti has also installed a state-of-art High Performance Liquid Chromatography system, which is being used to develop a reproducible large-scale procedure for preparing enzyme-treated gluten for clinical trials. The scientific protocols for production and testing of the enzyme are being designed for reproducibility from batch to batch and to allow scaling up production to the larger batches of material needed for the clinical trial.

In parallel with these laboratory efforts, Drs. Gray and Pyle are developing the protocols and consent forms for the clinical trial, and making arrangements with a local clinic for performing this trial. Resources permitting, the trial will be completed by the end of the year. If successful, it will provide a firm basis for initiating further clinical studies on whether an orally administered enzyme pill can effectively counter the toxic effects of dietary gluten in a Celiac Sprue patient.

To ramp up its enzyme therapy project and also initiate a second drug development project, the Celiac Sprue Research Foundation is recruiting additional Ph.D. level scientists with strong backgrounds in the life sciences. Of particular interest are outstanding immunologists, cell biologists, molecular biologists and chemists, who are interested in a career opportunity at the interface between academia and industry that allows them to gain a unique perspective on preclinical and clinical To ramp up its enzyme therapy project and also initiate a second drug development project, the Celiac Sprue Research Foundation is recruiting additional Ph.D. level scientists with strong backgrounds in the life sciences. Of particular interest are outstanding immunologists, cell biologists, molecular biologists and chemists, who are interested in a career opportunity at the interface between academia and industry that allows them to gain a unique perspective on preclinical and clinical aspects of drug development.

The Celiac Sprue Research Foundation is a public charity that relies upon donations from individuals to support its activities. As an Internal Revenue Code Section 501(c)(3) non-profit organization, your donations are tax deductible to the extent permitted by law.