Are We Any Closer to Intestinal Gene Therapy in Crohn's Disease?
05/24/22 • 20 min
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Drs Peter Higgins and Judy Cho discuss recent success in sickle cell anemia and whether we're any closer to using intestinal gene therapy in Crohn's disease. Relevant disclosures can be found with the episode show notes on Medscape.com (https://www.medscape.com/viewarticle/967621). The topics and discussions are planned, produced, and reviewed independently of advertiser. This podcast is intended only for US healthcare professionals.
Resources
Sickle Cell Anemia https://emedicine.medscape.com/article/205926-overview
Crohn's Disease https://emedicine.medscape.com/article/172940-overview
Inflammatory Bowel Disease https://emedicine.medscape.com/article/179037-overview
CRISPR-Cas systems: Overview, Innovations and Applications in Human Disease Research and Gene Therapy https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7508700/
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia https://www.nejm.org/doi/full/10.1056/NEJMoa2031054
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease https://www.nejm.org/doi/10.1056/NEJMoa2117175
Causes of Crohn's disease https://medlineplus.gov/genetics/condition/crohn-disease/#causes
IL-10R Polymorphisms Are Associated With Very-Early-Onset Ulcerative Colitis https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3744177/
Characterization of Crohn Disease In X-Linked Inhibitor of Apoptosis-Deficient Male Patients and Female Symptomatic Carriers https://pubmed.ncbi.nlm.nih.gov/24942515/
A Frameshift Mutation in NOD2 Associated With Susceptibility to Crohn's Disease https://www.nature.com/articles/35079114
Extended Haplotype Association Study in Crohn's Disease Identifies a Novel, Ashkenazi Jewish-Specific Missense Mutation in the NF-κB Pathway Gene, HEATR3 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3785105/
Autologous Stem Cell Transplant for Crohn's Disease https://www.mountsinai.org/clinical-trials/autologous-stem-cell-transplant-for-crohns-disease
Stem-Cell Transplantation for Crohn's Disease: Same Authors, Different Conclusions? https://www.thelancet.com/journals/langas/article/PIIS2468-1253(17)30076-6/fulltext#%20
A Myeloid-Stromal Niche and gp130 Rescue in NOD2-Driven Crohn's Disease https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8284870/
Expression of NOD2 in Paneth Cells: A Possible Link to Crohn's Ileitis https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1773866/
Role of ATG16L, NOD2 and IL23R in Crohn's Disease Pathogenesis https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3270503/
Drs Peter Higgins and Judy Cho discuss recent success in sickle cell anemia and whether we're any closer to using intestinal gene therapy in Crohn's disease. Relevant disclosures can be found with the episode show notes on Medscape.com (https://www.medscape.com/viewarticle/967621). The topics and discussions are planned, produced, and reviewed independently of advertiser. This podcast is intended only for US healthcare professionals.
Resources
Sickle Cell Anemia https://emedicine.medscape.com/article/205926-overview
Crohn's Disease https://emedicine.medscape.com/article/172940-overview
Inflammatory Bowel Disease https://emedicine.medscape.com/article/179037-overview
CRISPR-Cas systems: Overview, Innovations and Applications in Human Disease Research and Gene Therapy https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7508700/
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia https://www.nejm.org/doi/full/10.1056/NEJMoa2031054
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease https://www.nejm.org/doi/10.1056/NEJMoa2117175
Causes of Crohn's disease https://medlineplus.gov/genetics/condition/crohn-disease/#causes
IL-10R Polymorphisms Are Associated With Very-Early-Onset Ulcerative Colitis https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3744177/
Characterization of Crohn Disease In X-Linked Inhibitor of Apoptosis-Deficient Male Patients and Female Symptomatic Carriers https://pubmed.ncbi.nlm.nih.gov/24942515/
A Frameshift Mutation in NOD2 Associated With Susceptibility to Crohn's Disease https://www.nature.com/articles/35079114
Extended Haplotype Association Study in Crohn's Disease Identifies a Novel, Ashkenazi Jewish-Specific Missense Mutation in the NF-κB Pathway Gene, HEATR3 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3785105/
Autologous Stem Cell Transplant for Crohn's Disease https://www.mountsinai.org/clinical-trials/autologous-stem-cell-transplant-for-crohns-disease
Stem-Cell Transplantation for Crohn's Disease: Same Authors, Different Conclusions? https://www.thelancet.com/journals/langas/article/PIIS2468-1253(17)30076-6/fulltext#%20
A Myeloid-Stromal Niche and gp130 Rescue in NOD2-Driven Crohn's Disease https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8284870/
Expression of NOD2 in Paneth Cells: A Possible Link to Crohn's Ileitis https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1773866/
Role of ATG16L, NOD2 and IL23R in Crohn's Disease Pathogenesis https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3270503/
Previous Episode
Early Risk Markers, Interventions, and Prevention in Crohn's Disease
1 RecommendationsDrs Peter Higgins and Jean-Frederic Colombel discuss exciting new evidence in Crohn's disease research that may help in identifying those at risk before disease starts and pave the way for a vaccine. Relevant disclosures can be found with the episode show notes on Medscape.com (https://www.medscape.com/viewarticle/967620). The topics and discussions are planned, produced, and reviewed independently of advertiser. This podcast is intended only for US healthcare professionals.
Resources
Inflammatory Bowel Disease https://emedicine.medscape.com/article/179037-overview
Crohn Disease https://emedicine.medscape.com/article/172940-overview
Ulcerative Colitis https://emedicine.medscape.com/article/183084-overview
Standardising the Lactulose Mannitol Test of Gut Permeability to Minimise Error and Promote Comparability https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4047110/
Cohort Profile of the PRoteomic Evaluation and Discovery in an IBD Cohort of Tri-service Subjects (PREDICTS) Study: Rationale, Organization, Design, and Baseline Characteristics https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6446072/
Combined Serological, Genetic, and Inflammatory Markers Differentiate Non-IBD, Crohn's Disease, and Ulcerative Colitis Patients https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3792797/
The GEM PROJECT https://crohnsandcolitis.ca/Research/Funded-research/The-gem-project
Serum Zonulin Measured by Commercial Kit Fails to Correlate With Physiologic Measures of Altered Gut Permeability in First Degree Relatives of Crohn's Disease Patients https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8027468/
Tofacitinib and Newer JAK Inhibitors in Inflammatory Bowel Disease — Where We Are and Where We Are Going https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9007061/
Effects of B-Cell Directed Therapy on the Preclinical Stage of Rheumatoid Arthritis: The PRAIRI Study https://ard.bmj.com/content/annrheumdis/78/2/179.full.pdf
Longitudinal Analysis Reveals High Prevalence of Epstein-Barr Virus Associated With Multiple Sclerosis https://www.science.org/doi/10.1126/science.abj8222
Digestive Disease Week abstracts https://ddw.org/education/abstracts/
Serious Infections in Pediatric Inflammatory Bowel Disease 2002-2017 — A Nationwide Cohort Study https://www.jpeds.com/article/S0022-3476(21)00658-2/fulltext#%20
GM-CSF Autoantibodies: Predictors of Crohn's Disease Development and a Novel Therapeutic Approach https://academic.oup.com/jcag/article/4/Supplement_1/5/6158591
Next Episode
How Physicians Can Help Patients With Crohn's Disease Navigate Insurance Delays and Denials
1 RecommendationsDrs Peter Higgins and Frank Scott discuss the challenges of delays in care for patients with Crohn's disease, how insurance denials and delays harm patients, and what doctors can do to help.
Relevant disclosures can be found with the episode show notes on Medscape.com (https://www.medscape.com/viewarticle/967624). The topics and discussions are planned, produced, and reviewed independently of advertiser. This podcast is intended only for US healthcare professionals.
Resources
Specialty Drug Coverage Varies Across Commercial Health Plans in the US https://www.healthaffairs.org/doi/full/10.1377/hlthaff.2017.1553
2021 AMA Prior Authorization (PA) Physician Survey https://www.ama-assn.org/system/files/prior-authorization-survey.pdf
Corticosteroid Use and Complications in a US Inflammatory Bowel Disease Cohort https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4918923/
Increased Risk of Venous Thromboembolic Events With Corticosteroid vs Biologic Therapy for Inflammatory Bowel Disease https://www.cghjournal.org/article/S1542-3565(14)01045-3/fulltext
Impact of Prior Authorization of Antiepileptic Drugs in Children With Epilepsy https://www.pedneur.com/article/S0887-8994(18)30151-6/fulltext
Prior Authorization Delays Biologic Initiation and Is Associated With a Risk of Asthma Exacerbations https://pubmed.ncbi.nlm.nih.gov/33404389/
Treatment Delays Associated With Prior Authorization for Infusible Medications: A Cohort Study https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7062557/
Delays Related to Prior Authorization in Inflammatory Bowel Disease https://publications.aap.org/pediatrics/article/149/3/e2021052501/184867/Delays-Related-to-Prior-Authorization-in
What Is the Role of Fecal Calprotectin Levels in the Diagnosis of Inflammatory Bowel Disease (IBD)? https://www.medscape.com/answers/179037-54913/what-is-the-role-of-fecal-calprotectin-levels-in-the-diagnosis-of-inflammatory-bowel-disease-ibd
Variation In Use and Content of Prescription Drug Step Therapy Protocols, Within and Across Health Plans https://www.healthaffairs.org/doi/10.1377/hlthaff.2021.00822?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed
What Is Step Therapy/Fail First? https://www.crohnscolitisfoundation.org/get-involved/be-an-advocate/steptherapy
H.R. 2163 – Safe Step Act https://www.congress.gov/bill/117th-congress/house-bill/2163
Step Therapy's Balancing Act — Protecting Patients While Addressing High Drug Prices https://www.nejm.org/doi/10.1056/NEJMp2117582?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed
Battle Heats up Over Bill on "Step Therapy," Drug Rebates https://www.coloradopolitics.com/legislature/battle-heats-up-over-bill-on-step-therapy-drug-rebates/article_0f6e56c4-c0b8-11ec-9ead-47fa6f99ac6e.html
Crohn Disease Pathology https://emedicine.medscape.com/article/1986158-overview#a6
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