"After helplessly watching the disease's debilitating impact on my son's early childhood, and later discovering my wife was also a Celiac, I decided to apply my knowledge of chemistry, biology and drug development to Celiac Sprue," explained Dr. Khosla, Founder and President of the Celiac Sprue Research Foundation.
Dr. Khosla's collaborator Gary M. Gray, M.D., professor of medicine emeritus and former Chief of the Gastroenterology Division, and Director of the NIH-supported Digestive Disease Center at Stanford University Hospital, is equally enthusiastic. "The high frequency of Celiac Sprue is not generally appreciated by physicians in the Untied States, and patients often suffer for many years before the diagnosis is made. Then the requirement for a strict lifelong diet devoid of wheat and most grains becomes a burden for the patient and their families. Thus the potential for taking a supplemental peptidase to break up the gluten peptides may offer an important alternative to the restrictive diet sometime in the near future."
Gluten Is Toxic For Celiac Sprue Patients
Gluten is one of the most prevalent substances in foods, medicines and other consumer products, yet for approximately 1 in 200 individuals around the world who are Celiacs, even an occasional encounter with gluten may make them ill. And the long-term implications of the untreated disease, which include a substantially enhanced risk of malignancies (both cancer and lymphoma) of the small intestine and osteoporosis, are far more serious than the disease itself.
"It is now important to begin the process of translating new scientific knowledge about Celiac Sprue into products and technologies that will help patients," Dr. Gray continued. Dr. Gray co-chairs the Scientific Advisory Board forth Celiac Sprue Research Foundation.
Notwithstanding the need for a Celiac pill to cure the disease, this problem has escaped the attention of the pharmaceutical industry thus far. "This is a bold experiment in which a non-profit organization, rather than a pharmaceutical company, is taking the lead in drug development for a major unmet medical need," said Christopher T. Walsh, a Professor at Harvard Medical School and a former President of the Dana Farber Cancer Center in Boston. Dr. Walsh serves on the Board of Directors of the Celiac Sprue Research Foundation, and is also an advisor to several pharmaceutical and biotechnology companies. He continued that, if successful, "The Foundation could not only help Celiacs, but also set an example for how other overlooked diseases could be attacked."
What is Celiac Sprue?
Celiac Sprue (a.k.a. celiac disease, coeliac disease, or gluten intolerance) is an hereditary disorder characterized by a sensitivity to the toxic effects of gluten in the diet, leading to abnormal small-intestinal structure, malabsorption, and intolerance to gluten, a component of nutritionally important proteins found in common dietary food grains such as wheat, rye and barley.
The disease commonly presents in early childhood with severe symptoms including chronic diarrhea, abdominal distension, and failure to thrive. The general condition of these children is severely impaired. In many patients, symptoms may not develop until later in life when the disease presents with fatigue, diarrhea, and weight loss due to malabsorption of nutrients and vitamins, anemia and neurological symptoms.
This is a lifelong disease, and if untreated, patients have a substantially enhanced risk for the development of complications such as infertility, osteoporosis, intestinal cancer and lymphoma. There is no therapeutic option available to Celiac Sprue patients, the only treatment being a lifelong adherence to a strict gluten-free diet.
How Common Is Celiac Sprue?
Although the disease was considered uncommon until recently, the American Gastroenterological Association's most recent technical review on Celiac Sprue (Gastroenterology 120, 1526-1540, 2001) summarizes the results of several independent studies suggesting that the prevalence of the disease may be as high as 1 in 200 people in most parts of the world. Like other immune disorders such as Type 1 diabetes, rheumatoid arthritis and multiple sclerosis, both genetic and environmental factors play a role in the onset of Celiac Sprue.
Latest Research on Celiac Sprue
Together with collaborators from the University of Oslo in Norway, Stanford researchers Chaitan Khosla and Lu Shan, a graduate student in Dr. Khosla's laboratory, recently released their latest findings about Celiac Sprue, have discovered that a relatively short fragment of the gluten protein is exceptionally toxic to Celiac Sprue patients. This gluten fragment is unusually resistant to breakdown by digestive enzymes in the intestine, where it remains intact to have a destructive effect on the intestinal lining. Using this information, they identified a bacterial enzyme (a peptidase) that can rapidly degrade this and other related toxic fragments from gluten. The entire paper was published this week in the international weekly journal Science and can be found at www.sciencemag.org or by calling 202-326-6417.
The Celiac Sprue Research Foundation Objectives
Founded in 2002 and headquartered in Palo Alto, CA, with scientific advisors worldwide, the Celiac Sprue Research Foundation is a science-driven public charity that seeks to improve the quality of life of Celiac Sprue patients by promoting research and development, and by enhancing awareness of the disease among scientists, healthcare professionals, consumer product manufacturers and the general public. Its primary goal is to translate emerging knowledge about Celiac Sprue pathogenesis into a comprehensive plan for developing a therapeutic alternative to a gluten-free diet. Once the Foundation's initial drug development strategy has been launched, it will use available resources to promote basic research that might lead to fundamentally new insights into the disease, and to improve technologies for detecting new patients of this seriously under-diagnosed disease.