With more than 20 years’ experience in research and development of immunology and gastroenterology, including celiac disease, Francisco León, MD, PhD, CEO and Chief Medical Officer of Celimmune, reflected on the changing tides within the emerging celiac R&D space. At the forefront in changes are measurement of outcomes and validation of celiac as a treatable disease.
“Something interesting is happening; traditionally, the celiac field relied heavily on histology while the IBD field relied heavily on symptoms for regulatory and even clinical decisions,” León told Healio Gastroenterology. “The FDA has been studying the virtues and drawbacks of these two different extremes, and in the end it seems they have converged into a common pathway for gastrointestinal disorders, where they really want companies to provide both evidence of clinical benefit and objective evidence of decreased inflammation, or at least objective evidence that the disease pathways are ameliorated.”
“In the IBD field, there are now new endpoints under development that meet the current guidelines and guidance from FDA, patient-reported outcomes [PROs], but also there is much greater use of objective endpoints such as endoscopy, and even histology. Conversely, in celiac where we had histology, we now have two custom-made PROs ... that are now being used in clinical trials. And that is a big change.”
“At the GREAT 3 meeting, celiac doctors said, ‘We already have really good tools here. We have histology,’ and the FDA said, ‘We love your histology but we also want you to focus on symptoms.’ And that’s why most trials today use both. That’s the preferred endpoint in celiac disease.”
Yet these increased requirements are not deterring manufacturers from entering the field and trying to find the trigger, whether it be IL-15 — which León’s company is targeting — or another mechanism.
“Now we have more than a dozen companies doing drug development in celiac disease, which indicates there is interest, and many of these companies are partnering or talking with big pharmaceutical companies,” León said.
These tidal changes in celiac disease research point to hope for the patients living with this disease.
Editor’s Note: León represents just one of the many researchers and drug development companies in celiac disease. Healio Gastroenterology appreciates his insight as an established investigator in the field.
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- Francisco León, MD, PhD, can be reached at email@example.com.
Disclosure: León reports financial interests in Celimmune, Biomedal, Alba Therapeutics and Glutenostics.