Auteur Topic: Nieuw medicijn voor zeldzame vorm van coeliakie in zicht  (gelezen 1171 keer)

ine

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Nieuw medicijn voor zeldzame vorm van coeliakie in zicht
« Gepost op: mei 29, 2016, 22:04:39 »
Nieuw medicijn voor zeldzame vorm van coeliakie in zicht

Persbericht
13 mei 2016

Onderzoekers van VUmc onderzoeken samen met een Amerikaanse bedrijf een nieuw medicijn voor patiŽnten met een zeldzame vorm van coeliakie. Naar schatting 1% van de Nederlanders krijgt een ontstekingsreactie in de darm door het eten van gluten. Deze komen voor in tarwe, rogge en gerst. Dit veroorzaakt schade aan de dunne darm. Deze aandoening heet coeliakie. Een ernstige en zeldzame vorm van coeliakie kan leiden tot lymfeklierkanker. Het nieuwe medicijn hiervoor wordt nu uitgetest op een internationale patiŽntengroep in Amsterdam.

De meeste coeliakiepatiŽnten herstellen volledig op een levenslang glutenvrij dieet. In zeldzame gevallen herstelt de darm van coeliakiepatiŽnten niet ondanks een goed gevolgd dieet. Deze patiŽnten hebben een verhoogd risico op het ontwikkelen van een agressieve vorm van lymfeklierkanker in de darm. Behandelingen voor deze zogenoemde refractaire coeliakie type II bestonden tot nu toe enkel uit chemotherapie, al dan niet in combinatie met een stamceltransplantatie. Omdat chemotherapie niet altijd werkt en stamceltransplantatie niet altijd uit te voeren is vanwege een slechte conditie of hoge leeftijd van de patiŽnt, is er een sterke behoefte aan een nieuw medicijn.

De afdeling maag-, darm- en leverziekten van VUmc is sinds april 2016 onder leiding van professor Chris Mulder en professor Gerd Bouma gestart met een onderzoek naar een nieuw medicijn tegen refractaire coeliakie. Dit doen zij in samenwerking met het Amerikaanse bedrijf Celimmune. Vanwege de zeldzaamheid van de ziekte komen voor deze studie patiŽnten vanuit diverse landen in Europa naar Amsterdam voor deelname. Naast VUmc doen ook ziekenhuizen in Amerika, Finland, Spanje en Frankrijk mee aan dit onderzoek.

Bron: VUmc



Hoogst waarschijnlijk wordt dit medicijn/artikel bedoeld:
Celimmune and Celiac Disease Foundation Partner for Phase 2 Clinical Drug Study

Celimmune and Celiac Disease Foundation have joined forces to deploy the iCureCeliac patient registry as a screen for phase 2 clinical study for refractory celiac disease type II. This will be the first-ever use of the new and growing iCureCeliac registry to support celiac disease therapeutic treatment development.

Celimmune, LLC is a clinical development-stage immunotherapy company dedicated to developing transformational therapies that will relieve the burden of refractory celiac disease, historically referred to as refractory sprue, and other serious autoimmune conditions.
Studies show that approximately 1 in 200 celiac disease patients suffer from refractory celiac disease, meaning that the damaged villi in the small intestine do not heal from a gluten-free diet, and all other potential causes for this damage have been ruled out.

Currently, there is no medication approved or standard of care treatment for those with refractory celiac disease type II, with average survival falling between 3-10 years post-diagnosis due to chronic diarrhea and/or high-grade lymphomas, or more rarely, metastasis (the spread of a cancer or other disease from one organ or part of the body to another not directly connected with it), such as the skin or lungs. The gluten-free diet alone is not enough to treat someone with refractory celiac disease, leaving the patient without options and at an increased risk for premature death.

For the phase 2 study, data from iCureCeliacô, Celiac Disease Foundationís Patient-Powered Research Network (patient registry), has been analyzed, and patients with refractory celiac disease have been identified. Celiac Disease Foundation will contact these patients to inform them of this trial opportunity, ensuring that their privacy is protected. These patients will then have the option of contacting Celimmune to participate in this ground-breaking clinical trial. By working with Celiac Disease Foundation, Celimmune hopes to more efficiently test a therapy that will allow the damaged villi of people with refractory celiac disease to heal, thus improving their quality of their life.




Persbericht via Celiac Disease Foundation
Celimmune Commences Screening in Phase 2 Clinical Study in Refractory Celiac Disease Type II


Multinational Study to Evaluate the Potential of Anti-IL-15 Antibody, AMG 714, to Treat Rare in situ Small Bowel T cell Lymphoma
Lebanon, NJ and Bethesda, MD, March 31, 2016 --- Celimmune LLC, a clinical development-stage immunotherapy company focused on treating and preventing immune-mediated diseases, announced that it has commenced screening patients in its Phase 2 clinical study for AMG 714, an investigational anti-IL-15 monoclonal antibody for the treatment of refractory celiac disease Type II (RCD-II).

The randomized, double-blind, parallel-group study is expected to enroll 24 patients with a confirmed diagnosis of RCD-II. Study duration is 20 weeks and visits to the site take place every two weeks to receive intravenous infusion of AMG 714 or placebo. Intestinal biopsies will be taken before and after treatment to evaluate the efficacy of AMG 714. Patients enrolled will maintain a strict gluten-free diet during the study.

Francisco Leon, M.D., Ph.D., CEO and Chief Medical Officer of Celimmune, commented, ďWe are pleased to have commenced patient screening in this unprecedented industry-sponsored clinical trial evaluating an experimental therapeutic in refractory celiac disease Type II, an in situ T cell lymphoma that appears in the intestine of certain individuals with celiac disease as a rare complication of years of exposure to gluten.
Refractory celiac disease Type II has been treated with cladribine and bone marrow transplants, but these approaches have a limited response rate and carry their own risks. AMG 714, which neutralizes a molecule believed to be a key driver of this disease, IL-15, is an exciting new experimental approach that could provide a treatment option for patients diagnosed with refractory celiac disease Type II.Ē

Dr. Leon continued, ďIn addition to this trial, we recently started screening patients in another Phase 2 clinical study evaluating AMG 714 as a potential therapeutic for gluten-free diet non-responsive celiac disease, which reflects our commitment towards developing high-potential therapeutics for priority immune-mediated diseases.Ē

AMG 714 is a fully human immunoglobulin monoclonal antibody that binds to IL-15 and neutralizes its effects. IL-15 is considered to have a central role in celiac disease and to be a key driver of the generation of aberrant and malignant intraepithelial lymphocytes (IELs) in RCD-II. In four previous clinical studies, AMG 714 has been well tolerated by healthy volunteers, as well as by patients diagnosed with rheumatoid arthritis or psoriasis.

Refractory celiac disease (RCD), also known as refractory sprue, is a rare but specific complication of celiac disease that is believed to appear after many years of inadvertent or intentional exposure to gluten. Approximately 1 in 200 celiac patients will be diagnosed with RCD-II, which occurs when the proportion of abnormal IELs exceeds 20%. T
he risk of developing enteropathy-associated T cell lymphoma (EATL) increases to greater than 50% in RCD-II patients.
Currently, there is no medication approved or standard of care treatment for those with RCD-II, with average survival falling between 3-10 years post-diagnosis due to intractable diarrhea and/or high-grade lymphomas, or more rarely, metastasis to other parts of the body, such as the skin or lungs.

To learn more about RCD-II, AMG 714 or the Phase 2 clinical study, visit www.celimmune.com or www.clincaltrials.gov, using Identifier NCT02633020.


About Celimmune
Celimmune LLC is a clinical development-stage immunotherapy company focused on treating and preventing immune-mediated diseases and is headquartered in in Lebanon, NJ and Bethesda, Maryland. Celimmune will initially focus its distinctive core competence in translational medicine, immunotherapy clinical development and commercialization on combating celiac disease and other serious immune-mediated diseases. Celimmune has an exclusive licensing agreement with Amgen to develop, manufacture and commercialize AMG 714, a Phase 2-stage anti-IL-15 monoclonal antibody.
For more information on Celimmune, please visit www.celimmune.com.

https://celiac.org/wp/wp-content/uploads/2016/05/Celimmune-Commences-Screening-in-RCD-II-Study-FINAL-033116.pdf