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Showing results for tags 'trials'.
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Celiac.com 02/28/2024 - We get a lot of questions about what celiac disease trials and studies are going on, but there are so many, it can be hard to keep track. One easy source is the Mayo Clinic, which is at the forefront of a number of research trials, and studies to better understand, and treat celiac disease. Currently, there are more than a dozen exciting clinical trials underway, each focusing on different aspects of the celiac disease. One study aims to assess the effect of gluten on gut barrier function by using a novel gut permeability test developed by Mayo Clinic researchers. Another trial is evaluating the safety and tolerability of a potential new treatment, KAN-101, for celiac disease patients. Additionally, researchers are investigating the effectiveness and safety of numerous compounds for relieving symptoms in adult celiac disease patients. These trials represent important steps forward in advancing our understanding of celiac disease and developing new therapies to improve the lives of those affected by this condition. Celiac disease trials and studies currently underway at the Mayo Clinic facilities include: Gut Permeability Assessment in Celiac and Gluten Sensitive Children Rochester, MN This study will assess the effect of gluten on gut barrier function. Investigators at the Mayo Clinic have developed a new gut permeability test using rhamnose (sugar & water solution), and are hoping to prove its effectiveness in a clinical setting. A Study to Assess the Safety and Tolerability of Single and Multiple Doses of KAN-101 in Celiac Disease Patients Rochester, MN The purpose of this study is to assess the safety and tolerable of single and multiple doses of KAN-101 in patients with Celiac disease. A Study to Evaluate the Effectiveness and Safety of Larazotide Acetate for the Relief of celiac disease Symptoms Rochester, MN The purpose of this study is to assess the effectiveness and safety of larazotide acetate versus placebo for the relief of persistent symptoms in adult celiac disease patients. Study of the Safety, Tolerability, Pharmacokinetics and Biomarker of DONQ52 in Celiac Disease Patients Rochester, MN The purpose of this study is to evaluate the safety and tolerability of single and multiple doses of DONQ52 in celiac disease patients and to evaluate the safety and tolerability of DONQ52 in celiac disease patients in the presence of gluten after multiple doses of DONQ52. Furthermore, to characterize the DONQ52 PK profile following single and multiple subcutaneous (SC) dose(s) in celiac disease patients and to investigate the immunogenicity of DONQ52. A Study of the Response to and Changes in Intestinal Permeability 4 hours after a Gluten Challenge in Subjects with Celiac Disease and Non Celiac Gluten Sensitivity Rochester, MN The purpose of this study is to identify mediators—innate immune system and intestinal permeability—of acute onset gastrointestinal symptoms after gluten ingestion in subjects with celiac disease and non-celiac gluten sensitivity. Gut Permeability in Environmental Enteropathy Rochester, MN The goal of this project, to develop a simple and sensitive test of small bowel permeability, will improve the scientific and public health understanding of environmental enteropathy and guide development of preventative and treatment modalities such as clean water and sanitation. Mucosal Interactions in Celiac Disease Rochester, MN To obtain biopsies and blood from subjects not previously known to have celiac disease who are undergoing duodenal biopsies for clinical indications. The subjects will have no prior diagnosis of celiac disease and are eating a gluten-containing diet. The biopsies will be treated “in vitro” with enzymatic digests of gluten derived from bread made from different species of wheat, the auto-antigen tTg, cytokines, or other proteins. A Study to Evaluate the Safety, Effectiveness, and Tolerability of Latiglutenase to Treat Patients with Well-Controlled Celiac Disease Rochester, MN The purpose of this study is to demonstrate a positive correlation of histologic protection (biological signature) and symptom protection (clinical outcome) for latiglutenase treatment versus placebo in Celiac Disease (celiac disease) patients undergoing a deliberate gluten challenge. A Study of the Safety, Pharmacodynamics, Effectiveness, and PK of TIMP-GLIA in Subjects with Celiac Disease Rochester, MN The purpose of this study is to evaluate participants for immune responses and histological changes in the small bowel following 2 doses of TIMP-GLIA or placebo and a 14-day oral gluten challenge. Dose-Ranging Study of the Efficacy and Safety of TAK-101 for Prevention of Gluten-Specific T Cell Activation in Participants With Celiac Disease on a Gluten-Free Diet Rochester, MN The purpose of this study is to compare the number of baseline interferon-gamma (IFN-γ) spot forming units (SFUs) to the number of IFN-γ SFUs after a 6-day oral gluten challenge among HLA DQ2–positive subjects treated with TAK-101 versus placebo. A Study of Simvastatin Metabolism as a Test for Celiac Disease Activity Rochester, MN The purpose of the study is to assess the connection between simvastatin metabolism by an enzyme that varies based on the state of the small intestine in treated celiac disease. Antibody Treatment for Advanced Celiac Disease Rochester, MN Celiac disease is a condition where the immune system attacks the cells of the small intestine. The intestine becomes inflamed and cannot digest food properly. The disease most often causes a reaction to foods that contain gluten. Most people can treat celiac disease with a gluten-free diet. However, some people have digestion problems even on a gluten-free diet. Researchers want to try a new antibody therapy for celiac disease. The treatment may block the immune reaction that causes the disease. They will test this antibody in people who have celiac disease that has not responded to a gluten-free diet. Prospective, Randomized, Double-Blind, Placebo-Controlled, Crossover Study in Symptomatic Celiac Disease Patients Rochester, MN This is a phase 2b, multi-center, prospective, randomized, double-blind, placebo-controlled, crossover study in symptomatic celiac disease patients attempting a gluten free diet (GFD) for at least one year prior to screening. A Study of TAK-062 in Treatment of Active Celiac Disease in Participants Attempting a Gluten-Free Diet Rochester, MN; Scottsdale/Phoenix, AZ The purpose of this study is to see how TAK-062 works to reduce celiac-related symptoms and improve small intestinal damage due to gluten exposure, in participants with celiac disease (celiac disease) attempting to maintain a gluten-free diet (GFD) in treated participants versus placebo controls. A Study of the Safety, Effectiveness and Tolerability of Nexvax-2 in Patients with Celiac Disease (celiac disease) Rochester, MN The purpose of this study is to evaluate the human leukocyte antigen (HLA)-DQ 2.5+ in adults with celiac disease (celiac disease). Evaluation of the Efficacy and Safety of ALV003 in Symptomatic in Celiac Disease Patients Scottsdale/Phoenix, AZ; Jacksonville, FL; Rochester, MN To determine the effects of 12 weeks administration of different dose levels of ALV003 on the mucosal lining of the small intestine and symptoms in celiac disease patients. Minimal Risk Registry of Endoscopic Image and Pathology Correlation for Fujiflim Jacksonville, FL; Rochester, MN The purpose of this study is to see if advanced endoscopic imaging may be helpful to accurately distinguish pathological tissue from normal tissue and guide therapy of endoscopically identified pathology. Functional Gastrointestinal Disease, Celiac Disease, and Non-celiac Gluten Sensitivity in an Olmsted County Cohort Rochester, MN The aim of this study is to the number of individuals following a gluten-free diet and possible reasons including symptoms of non-celiac gluten sensitivity and functional gastrointestinal diseases (FGIDs). lso assess Diagnoses and lab values associated with celiac disease, NCGS, and FGIDs will also be assessed, as well as past GI survey data and medical record data available through the Rochester Epidemiology Project. Stay tuned for updates on the progress of these and other related studies! Read more at Mayo.edu
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Celiac.com 07/18/2022 - Currently, a gluten-free diet is the only treatment for people with celiac disease. A number of companies have been attempting to create treatments that reduce or eliminate celiac disease symptoms, mostly for patients on a gluten-free diet. Larazotide, whose clinical trial is dubbed "CedLara," is such a drug. It's designed to reduce persistent celiac disease symptoms for people on a gluten-free diet. In an earlier phase 2 trial, Larazotide was shown to reduce celiac symptoms in patients who had been on a gluten-free diet for at least 12 months. Many were excited to see how it would do in a phase 3 trial. The answer, unless we get some better news from 9 Meters Biopharma, the company that has been developing it, is badly. For the phase 3 trial, 9 Meters Biopharma set out to enroll 525 patients in the phase 3 trial to determine the effect of larazotide on celiac disease severity. To determine the number of people needed to measure a statistically significant effect, the company conducted an analysis with half of the expected patients enrolled. According to a company news release, their analysis showed that the additional number of patients needed to produce a significant clinical outcome between placebo and Larazotide is too large for the company to pursue. Reading between the lines of the news release, it seems as though the the company might need far more test subject than originally estimated to show a statistically significant result. That means that, no matter how effective the drug was for some people, the company can't afford to test in large enough numbers to show that it's genuinely effective. With the failure of Larazotide, 9 Meters Biopharma announced that it will be pivoting to the development of vurolenatide, a repeated injection aimed at increasing nutrient absorption in patients with short bowel syndrome. Phase 2 results should be unveiled soon. The failure of Larazotide marks the latest addition to the growing graveyard of celiac disease drugs. As Larazotide has been touted since 2013, this failure is particularly disappointing. To punctuate the ignoble end for a once hopeful drug, the company's CEO and president, John Temperato, says that financial and human resources from Larazotide will be reassigned to advance vurolenatide and the company’s early-stage product candidates, pending a review. Read more at seekingalpha.com
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FDA Issues New Guidelines for Celiac Drug Trials
Jefferson Adams posted an article in Additional Concerns
Celiac.com 07/04/2022 - The Food and Drug Administration (FDA) has issued a set of new guidelines for companies doing celiac disease drug trials. The agency noted that the guidance is intended only to provide clarity regarding existing requirements, and should be viewed solely as recommendations, unless they mention specific regulations or laws. The FDA directs sponsors of trials for clinical drugs to ensure the following regarding celiac disease patients on a gluten-free diet: Trial population Patients should undergo diagnostic esophagogastroduodenoscopy, with multiple biopsies to confirm celiac diagnosis. The biopsies should include one or two samples of the duodenal bulb and at least four samples of the distal duodenum. To avoid inclusion of patients whose symptoms are not celiac-related, patients should receive esophagogastroduodenoscopy screening with biopsy to ensure they meet histologic eligibility criteria at the time of trial enrollment. Patients should be symptomatic at baseline, based on enrollment criteria, to allow for evaluation of symptom improvement. Prior to trail enrollment and for the full duration, patients should follow to a strict gluten-free diet with input from trained dietitians. Trial design The FDA recommends using randomized, double-blind, placebo-controlled trial design. Before randomization of participants, sponsors should include a screening period to confirm histologic eligibility criteria, document clinical signs and symptoms, and train patients and/or providers in collecting clinical outcome assessment data. Trial duration and outcome assessments should be informed by the therapy goal, expected drug onset of action and the time frame in which clinical benefit is observable. The FDA recommends a placebo-controlled treatment period of at least 52 weeks for drugs intended for chronic use, with continued patient adherence to a gluten-free diet. Efficacy assessments for both clinical and histologic endpoints may be evaluated at week 24, and esophagogastroduodenoscopy with biopsy should be performed at week 52 to assess durability of response. Sponsors should make sure their patients follow a gluten-free diet for the complete treatment period. Efficacy and clinical outcome assessments Trails intended to support market approval should include coprimary endpoints to assess a drug’s effect on clinical signs, symptoms and related underlying mucosal condition. The FDA also recommends a pre-specified secondary endpoint to determine the number of patients who see improvement of clinical signs, symptoms and mucosal inflammation. Trial sponsors should seek FDA input early, when critical milestones are met, and throughout drug development. Sponsors should also identify disease and treatment burdens using patient input. To better assess symptom severity and event-related signs and symptoms, sponsors should ask patients to rate their worst experience and frequency of a specific sign or symptom over a 24-hour period. Statistical and safety considerations To be demonstrate efficacy, trial results should demonstrate statistical significance for both clinical and histological endpoints, and analyses should include all randomized patients. Sponsors should assess gluten-free diet adherence, which could alter efficacy data. Moreover, sponsors should talk with patients about the importance of following a strict gluten-free diet, since the benefit of the drug is still unknown. To assess the safety of drugs intended for long-term use, patients should be follow the intended market dosage for at least one year. The FDA recommends that sponsors include safety analyses to compare risk and confidence intervals in treatment groups. Read the full recommendations at FDA.gov-
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Celiac Disease Vaccine Enters Phase 1 Clinical Trials in U.S.
Gryphon Myers posted an article in Latest Research
Celiac.com 09/10/2012 - Last year, ImmusanT's Nexvax2 celiac disease vaccine passed its phase 1 clinical trials in Australia and New Zealand, causing a stir of hope and anticipation within the celiac disease community. It will still be a while before the vaccine is available to the public, but yesterday ImmusanT announced that it commenced phase 1b clinical trials in New Zealand and Australia and phase 1 clinical trials in the U.S. New Zealand and Australia's phase 1b randomized, placebo-controlled, double-blind study will follow on the prior phase 1 trials and involve approximately 84 celiac disease patients on gluten-free diet at four study sites throughout the two countries. The focus of the study will be on evaluating safety, tolerability and pharmacokinetics (what the body does to the drug), as well as determining appropriate doses for subsequent studies. In the U.S., the phase 1 randomized, placebo-controlled, double-blind study will involve 30 celiac disease patients on gluten-free diet at approximately four test sites throughout the country. This preliminary study will evaluate safety, tolerability, and pharmacokinetics of the drug for American subjects. Patients in both studies will have confirmed diagnosis of celiac disease, as well as the HLA-DQ2 gene (which approximately 90% of celiac disease sufferers carry). Blood tests for gluten-reactive T cells will also be used to select suitable test subjects. The studies will employ an intradermal injection vaccine delivery solution by BD. The Nexvax2 vaccine is intended to restore the body's ability to tolerate gluten. It is hoped that antigen-specific T cells will be the key to allowing patients to consume gluten freely with no adverse effects. If all goes well during this and subsequent clinical trials, we could have a cure for celiac disease in the not-too-distant future. Source: http://www.sacbee.com/2012/09/04/4784050/immusant-initiates-clinical-trials.html- 27 comments
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Celiac.com 02/20/2019 - Pharmaceutical company ImmusanT is developing a celiac disease vaccine called Nexvax 2. Many vaccines provide long-term or permanent protection against disease after just one, or several doses. Because celiac disease is not caused by a virus, like polio, but is a response to the presence of an antigen (similar to an allergen that triggers an allergy), the approach to creating a vaccine like Nexvax 2 is different and, in some ways, easier, than creating a traditional vaccine, like the HPV vaccine. Nexvax 2 is a vaccine in much the same way that allergy shots are, but not in the way the polio vaccine is. Celiac Vaccine is Similar to Allergy Shots Unlike traditional vaccines, such as the polio vaccine, or the measles vaccine, Nexvax 2 does not inject a small dose of dead or weakened virus, or any virus fragment, into the patient to achieve disease immunity. Allergy shots work by desensitizing the body’s reaction by strengthening the immune system, thereby reducing or eliminating reactions to certain allergens. Nexvax 2 would work in a similar manner to allergy shots. It would build tolerance levels until there was little or no immune reaction to gluten exposure. Anyone who’s ever had allergy shots knows that their effectiveness can range from person to person. Some people get minimal relief, though most see good to excellent results. Many experience tremendous relief, and see their symptoms disappear. Nexvax 2 Faces Easier Path to Approval Because Nexvax 2 works less like a traditional vaccine, and more like allergy therapy, the process for testing and approval is potentially easier and shorter; several years, rather than a decade or more. The hope is that, once treated with Nexvax 2, “the immune system, now seeing these fragments of gluten in a different way, might learn to tolerate gluten," said Benjamin Lebwohl, director of clinical research at the Celiac Disease Center at Columbia University. Certainly, the ability to reduce or neutralize the body’s reaction to gluten in people with celiac disease would be a major breakthrough in the treatment of celiac disease. Benefits for celiac patients could include a reduction in severity of gluten contamination symptoms, and potentially an elimination of symptoms entirely. Nexvax 2 treatment, if successful, could allow some people with celiac disease to safely consume wheat. That is potentially huge news. Phase two clinical trials of the Nexvax 2 are slated for completion by the end of 2019. Read more: Promising Celiac Vaccine Nexvax 2 Begins Phase Two Trials
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Celiac.com 07/19/2016 - The world's first vaccine aimed at curing celiac disease is slated to begin full trials later this year, and residents of the Australian state of Victoria will be among the first humans to give it a try against celiac disease. The vaccine, called Nexvax2, was developed by Australian scientist Dr Bob Anderson, and is aimed at giving celiac patients a chance to overcome their immune reaction to the gluten found in products containing wheat, rye and barley. Nexvax2 aims to de-sensitise patients to three peptides contained in gluten that trigger a damaging reaction in their immune system. Previous trials on 150 patients from Melbourne, Perth, Adelaide, Brisbane and Auckland were aimed at finding a safe dosage rather than assessing its ability to beat celiac disease. Results from those favorable earlier trials were released in May, and Dr Anderson says that the larger phase II study, also being undertaken in the US and Europe, will assess how well the vaccine works against celiac disease. Dr Anderson first identified the peptides triggering coeliac disease and began developing the vaccine while working at Melbourne's Walter and Eliza Hall Institute, before travelling to Boston for six weeks as part of a sister city arrangement through the City of Melbourne, where he made contact with ImmusanT to further the discovery. This is certainly exciting news for people with celiac disease, many of whom may benefit from such treatment. Stay tuned for news on the progress of these trials. Source: dailysecrets.press
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Celiac.com 04/04/2016 - Any one eager to try the first approved treatment for celiac disease might not have to wait much longer. Alba Therapeutics has announced that their celiac treatment, larazotide acetate, will enter the first Phase 3 clinical trials ever conducted in a celiac disease drug later this year. Lorazotide acetate works by improving regulation of tight junctions in the bowel. In healthy people, these junctions remain closed except to shed dead cells, but in patients with celiac disease, gluten keeps tight junctions open, triggering an inflammatory reaction that eventually destroys the intestinal villi, tiny, finger-like projections in the small intestine that are essential for nutrient absorption. Early research suggests larazotide acetate helps to keep the tight junctions closed when it's taken before a meal, thus stopping, or reducing the reaction and the resulting inflammation. Larazotide acetate recently completed during phase 2b clinical trials for efficacy, safety and tolerability in 342 patients with celiac disease. Those trials showed larazotide acetate to be safe and effective in a "real world setting" for celiac patients, according to Alba's website. The treatment is now headed to Phase 3 trials in "late 2016", and has received "fast track" designation from the Food and Drug Administration. Alba has announced that Innovate Biopharmaceuticals Inc. has licensed all of Alba Therapeutics' assets related to larazotide acetate, and that larazotide acetate has been renamed INN-202. If approved on schedule, INN-202 will become the first prescription medicine for treating celiac disease. Source: Allergic Living
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Celiac.com 04/18/2015 - Research is underway on a number of new drugs intended to celiac disease treatment beyond a simple gluten-free diet. However, even though several drugs are in Phase 2 trials and results appear promising, discussion around regulatory endpoints is just beginning. A research team recently reviewed endpoints for Phase 2 and 3 trials of new celiac disease drugs currently under development, and detailed their results in a scientific paper. The team included Klaus Gottlieb, Jill Dawson, Fez Hussain and Joseph A. Murray. They are variously affiliated with the department of Immunology and Internal Medicine of Medical Strategy & Science, Quintiles, Durham, NC, USA, with Corporate Communications, Quintiles, Durham, NC, USA, and with the Division of Gastroenterology and Hepatology, Mayo Clinic, Rochester, MN, USA. In the paper, the team discusses celiac drugs currently under development, along with trial endpoints, such as patient-reported outcomes, histology, serology, gene expression analysis and other tests. They outline the differing requirements for proof-of-concept Phase 2 trials and Phase 3 registration trials, with a particular emphasis on current thinking in regulatory agencies. They conclude their paper with recommendations and a glossary of regulatory terms, to enable readers who are less familiar with regulatory language to take maximum advantage of this review. Stay tuned for more news and information on all developments concerning trials of new celiac drug treatments. Source: Gastroenterology Report, Oxford Journals. 10.1093/gastro/gov006
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Celiac.com 11/30/2011 - Researchers have been talking about it for some time, raising the hopes of the celiac community: a drug to help relieve us from the harmful effects of gluten exposure. Celiac patients are closer than ever to having such a drug on the market, as Alvine Pharmaceuticals has announced that their drug ALV003 has shown promise in a clinical trial by reducing gluten-triggered harm in people with celiac disease. Celiac disease is an autoimmune reaction triggered by exposure to gluten, a protein found in wheat, barley, and rye, that causes the immune system to attack the small intestine, interfering with the absorption of nutrients and leading to malnutrition and a variety of other symptoms. The disease currently has only one treatment, which is non-drug: the gluten-free diet. By eliminating gluten completely from the diet, most celiac patients can heal their small intestine. There is currently no other drug on the market that can help relieve the symptoms of celiac disease or the intestinal damage it can cause. Now Alvine Pharmaceuticals, which is focused on developing biopharmaceuticals for autoimmune inflammatory diseases such as celiac disease, has reported favorable results for a trial with their drug ALV003, which was developed to lessen mucosal injury in the intestine caused by gluten exposure in well-controlled celiac patients. A control group study was conducted that collected data from 34 celiac patients. After both the active drug group and placebo group ingested two grams of gluten on a daily basis for six weeks, "The group with the placebo reported higher incidence of 'non-serious adverse events' (code for GI symptoms)," Triumph Dining reported. "They also had significantly more mucosal injury in their small intestines, as measured by biopsy data." ALV003 works by breaking down the gluten molecule into nontoxic parts. (Alvine Pharmaceuticals explains more specifically how the drug works on their website, AlvinePharma.com.) The drug is intended to help alleviate the gastrointestinal and other symptoms associated with cross-contamination, incorrect or misleading "gluten-free" labeling, and exposure to gluten caused by carelessness or imprudence. Even when celiac patients take care to maintain a strict gluten-free, it's difficult to stay completely away from gluten. That's why, according to coordinating investigator of the latest ALV003, Markku Maeki, M.D., chair and professor of pediatrics at the University of Tampere and Tampere University Hospital in Finland, "New non-dietary treatment options that can either eliminate, or meaningfully reduce the gluten present in an attempted gluten-free diet are needed." Currently celiacs have no drug options to help alleviate their symptoms. "These results are groundbreaking," said Professor Maeki, "as they demonstrate for the first time, in a controlled clinical trial, that a drug has the potential to diminish gluten-induced injury in celiac disease patients." According to Triumph Dining, "After Phase 3 trials, so long as results remain promising, ALV003 will enter Phase 2b trials soon; after that come Phase 3 trials and (hopefully) submission to the FDA for approval." The release of ALV003, should results remain favorable, will no doubt bring relief to many members of the celiac community.
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First Ever Celiac Disease Vaccine Trials Underway in Australia
Jefferson Adams posted an article in Latest Research
Celiac.com 04/06/2009 - Celiac sufferers around the globe are anxiously awaiting word from Australia, as the world's first vaccine trials for the treatment of celiac disease get underway in Melbourne. In April, Bob Anderson, of the Walter and Eliza Hall Institute of Medical research, will begin the initial phase of the first-ever trials for a celiac vaccine that, if successful, might just mean the end of gluten-free diets for those with celiac disease. The treatment has been successful in mice and is now ready to be tested on humans. In this initial phase, 40 volunteers with celiac disease will receive doses of the vaccine over an 11-month period to determine that it will cause no harm. Once researchers make sure the vaccine is safe, they will begin phase II trial, wherein they give vaccine doses to trial subjects and evaluate their responses to gluten challenges to determine the efficacy of the vaccine. Evaluation will include an examination of immune response and intestinal condition to determine the level of gluten tolerance. The vaccine therapy involves repeatedly injecting solutions of gluten at increasing concentrations. The goal is to reduce and ultimately eliminate gluten sensitivity slowly, in a manner similar to common allergy desensitization treatments. The road to the development of this treatment has not been easy. Dr. Anderson is that rare combination of medical doctor (gastroenterologist) and PhD scientist who is able to develop practical treatments from bedside observations. After struggling to gain funding throughout his research career, he eventually patented his vaccine and co-founded Nexpep in an effort to develop the vaccine on his own. Because, like common dust and hay fever allergy therapies, this treatment approach may allow people with celiac disease to actually consume the gluten that produces the toxic reaction and reduce or even eliminate that reaction via vaccination. This approach will also serve as a model for a vaccine approach for other immune conditions such as type 1 diabetes, rheumatoid arthritis and multiple sclerosis. Until recently, doctors thought celiac disease was rare. But according to statistics, it is twice as common as type1 diabetes or breast cancer. Celiac disease is now known to strike one per cent of Americans, but although modern blood testing has made early detection accurate and efficient, most people with celiac disease still do not know that they have it. Just 3% of sufferers have been diagnosed, leaving nearly 3 million people undiagnosed, and therefore unable to benefit form simple treatment in the form of a gluten-free diet. Long-term risks for untreated celiac disease include malnutrition, infertility, osteoporotic fractures, liver failure and various cancers. Symptoms can vary between individuals, with some experiencing no symptoms at all, even though damage to the bowel and general health still occurs whether or not symptoms are present. Presently, long-term monitoring of dietary compliance for celiac patients is haphazard at best, and standards for gluten-free products have yet to take effect in the USA and other countries. Geoff Withers, director of pediatric gastroenterology at Brisbane's Royal Children's Hospital, points out that a gluten-free diet is "notoriously difficult. It is expensive and lifelong, and comes at a cost to the individual." Even treatment with a gluten-free disease is no panacea. People on gluten-free diets routinely suffer from a deficiency of certain vitamins, especially B vitamins. Roughly half of those following gluten-free diets have impaired intestinal healing due to compliance issues, and that means they are in danger of associated risks which include cancer. A successful vaccine could have massive consequences for treatment of celiac disease, and might radically improve the lives of those with the condition. -
Celiac.com 05/23/2011 - ImmusanT, Inc., a biotechnology start up based in Cambridge, Massachusetts, is testing a vaccine to desensitize celiac patients to gluten. It is called Nexvax2, and it has already passed Phase I clinical trials, which means that it is safe and tolerable to humans. Nexvax2 is slated to begin Phase II trials, which address efficacy, within the next year. Nexvax2 was developed by Nexpep Pty, Ltd., a company in Melbourne, Australia. It is based on their findings that only three peptides are responsible for eliciting the majority of the T cell response that goes on to destroy the intestines of celiac patients. HLA molecules function to present these toxic peptides to T cells; this presentation is what activates the T cells, instigating the inflammatory response. Thus, this vaccine relies on the HLA type. It is specific for celiacs with the HLA-DQ2 haplotype, accounting for about 90% of celiac patients. Nexvax2 encompasses these three proprietary peptides, presenting them to T cells in the absence of a second, T-cell stimulatory signal. T cell recognition of the HLA-DQ2 bound toxic peptides thus occurs in a non-inflammatory environment, establishing tolerance to dietary gluten. This peptide based approach has been successful in generating tolerance in people with cat-sensitive asthma, and has not been used more broadly because it has been difficult to identify the correct toxic epitopes. Similar efforts are underway to discover and develop peptide-based therapeutic vaccines for other autoimmune diseases, including multiple sclerosis, Type-1 diabetes, and rheumatoid arthritis, but celiac disease is an ideal target for the technology because the HLA types that activate the inflammatory T cells in celiac disease are so well defined. The vaccine consists of a weekly or monthly injection, and would allow those with celiac disease to resume eating "normal" levels of gluten without suffering adverse effects. Other therapies that have proposed to treat celiac disease, such as those promoted by the companies Alva, Alba, and Chemocentryx, did not aim to replace the gluten free diet; they allowed only small, intermittent exposure to gluten. During the Phase I trial of Nexvax2, some people who got the injections containing the highest doses of the toxic peptides suffered gastrointestinal distress; they thus inadvertently acted as a positive control, indicating that the peptides administered are in fact the correct ones. ImmusanT is also partnering with INOVA Diagnostics to use reactivity to these peptides as a diagnostic test both for celiac disease and for those celiac patients who might be good candidates for the Nexvax2 vaccine - i.e. those 90% who are HLA-DQ2 rather than those who are HLA-DQ8. Source: http://www.sciencedaily.com/releases/2011/05/110509091559.htm
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Celiac Disease Vaccine Trials Slated for 2009
Jefferson Adams posted an article in Diagnosis, Testing & Treatment
Celiac.com 12/18/2008 - Celiac disease is a life-long autoimmune disease. When people with celiac disease consume the gluten proteins found in wheat, rye and barley they damage the lining of the gut, which prevents normal digestion and absorption of food. There is currently no cure for the celiac disease. The only treatment is life-long adherence to a strict gluten-free diet. If a gluten-free diet is not followed, the disease can ultimately lead to ill health and life-threatening conditions including malnutrition, osteoporosis, bowel cancer, and may cause infertility problems. The charity group Coeliac UK, recently hosted a conference at the Royal Society of Arts in central London where, among the latest findings in celiac disease research, they announced progress on the development of a possible vaccine for the condition. Dr. Bob Anderson of the Autoimmunity and Transplantation Division of Australia’s Walter and Eliza Hall Institute has led a research team that has isolated the toxic elements of gluten, paving the way for a possible vaccine that will suppress or prevent gluten toxicity. The research indicates that the toxic, autoimmune response in celiac patients exposed to wheat is triggered by just few dominant peptides in the gluten protein. This small number of offending peptides makes it exponentially easier for researchers to develop a vaccine. Dr. Anderson is a joint founder and CEO of Nexpep, an Australian company that is actively working to develop a vaccine to treat celiac disease. Dr. Anderson’s team has created a peptide-based therapeutic vaccine to treat the main problem T-cell epitopes of gluten. The vaccine has the potential to treat at about 80% of people with celiac disease and having the appropriate genetic background. Similar to traditional desensitization therapy for allergies, the peptide-based vaccines are given in multiple small doses over a course of injections in an effort to create immune tolerance not only to the selected gluten fragments, but also lower the toxicity of related toxic gluten molecules. Nexpep is currently raising capital for a clinical trial program for a peptide-based therapeutic vaccine and intends to commence a Phase 1 clinical trial in the first half of 2009. Reference: http://www.medicalnewstoday.com/articles/131745.php- 18 comments
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