Intestinal organoids and personalized medicine in cystic fibrosis: A successful patient-oriented research collaboration

Current Opinion in Pulmonary Medicine

Volumn 22, Issue 6, 2016, Pages 610-616

  Noordhoek, Jacquelien ^a   Gulmans, Vincent ^a   Van Der Ent, Kors ^b   Beekman, Jeffrey M ^b,c  

^a Dutch Cystic Fibrosis Foundation (NCFS)   (Netherlands)

^b UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^c UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

Author keywords

Cystic fibrosis transmembrane conductance regulator modulators; Intestinal organoids; Patient organization; Personalized medicine

Indexed keywords

CHLORIDE CHANNEL STIMULATING AGENT; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR MODULATOR; UNCLASSIFIED DRUG;

BIOBANK; CYSTIC FIBROSIS; DRUG EFFICACY; DRUG RESPONSE; HEALTH CARE ORGANIZATION; HUMAN; IN VIVO STUDY; INTESTINAL ORGANOID; INTESTINE; NONHUMAN; PATIENT CARE; PERSONALIZED MEDICINE; PHASE 2 CLINICAL TRIAL (TOPIC); PRECLINICAL STUDY; PRIMARY CELL CULTURE; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; STEM CELL CULTURE; GENOTYPE; INTERSECTORAL COLLABORATION; MUTATION; ORGANOID;

CYSTIC FIBROSIS; GENOTYPE; HUMANS; INTERSECTORAL COLLABORATION; MUTATION; ORGANOIDS; PRECISION MEDICINE;

EID: 84987924535     PISSN: 10705287     EISSN: 15316971     Source Type: Journal    
DOI: 10.1097/MCP.0000000000000315     Document Type: Review

References (28)

1. De Boeck K, Amaral MD. Progress in therapies for cystic fibrosis. Lancet Respir Med 2016; 4:662-674.
2. De Boeck K, Wilschanski M, Castellani C, et al.; Diagnostic Working Group. Cystic fibrosis: terminology and diagnostic algorithms. Thorax 2006; 61:627-635.
3. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011; 365:1663-1672.
4. Boyle MP, Bell SC, Konstan MW, et al. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial. Lancet Respir Med 2014; 2:527-538.
5. Wainwright CE, Elborn JS, Ramsey BW, et al. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med 2015; 373:220-231.
6. Accurso FJ, Rowe SM, Clancy JP, et al. Effect of VX-770 in persons with cystic fibrosisandtheG551D-CFTRmutation. N Engl JMed 2010;363:1991-2003.
7. Flume PA, Liou TG, Borowitz DS, et al. Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation ivacaftor in homozygous F508del-CFTR mutation. Chest 2012; 142:718.
8. Clancy JP, Rowe SM, Accurso FJ, et al. Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax 2012; 67:12-18.
9. De Boeck K, Munck A, Walker S, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros 2014; 13:674-680.
10. Van Goor F, Hadida S, Grootenhuis PD, et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci USA 2011; 108:18843-18848.
11. Van Goor F, Hadida S, Grootenhuis PD, et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci USA 2009; 106:18825-18830.
12. Yu H, Burton B, Huang C-J, et al. Ivacaftor potentiation of multiple CFTR channels with gating mutations. J Cyst Fibros 2012; 11:237-245.
13. Dekkers JF, Wiegerinck CL, De Jonge HR, et al. A functional CFTR assay using primary cystic fibrosis intestinal organoids. Nat Med 2013; 19:939-945.
14. Dekkers JF, Berkers G, Kruisselbrink E, et al. Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis. Sci Transl Med 2016; 8:344ra84.
15. Sato T, Vries RG, Snippert HJ, et al. Single Lgr5 stem cells build crypt-villus structures in vitro without a mesenchymal niche. Nature 2009; 459:262-265.
16. Sato T, Clevers H. Growing self-organizing mini-guts from a single intestinal stem cell: mechanism and applications. Science 2013; 340:1190-1194.
17. Sato T, Stange DE, Ferrante M, et al. Long-term expansion of epithelial organoids from human colon, adenoma, adenocarcinoma, and Barrett's epithelium. Gastroenterology 2011; 141:1762-1772.
18. Servidoni MF, Sousa M, Vinagre AM, et al. Rectal forceps biopsy procedure in cystic fibrosis: technical aspects and patients perspective for clinical trials feasibility. BMC Gastroenterol 2013; 13:91.
19. Dekkers JF, der Ent van CK, Beekman JM. Novel opportunities for CFTR-targeting drug development using organoids. Rare Dis 2014; 1:e27112.
20. Zomer-Van Ommen DD, Vijftigschild LA, Kruisselbrink E, et al. Limited premature termination codon suppression by read-through agents in cystic fibrosis intestinal organoids. J Cyst Fibros 2016; 15:158-162.
21. Dekkers JF, Gogorza Gondra RA, Kruisselbrink E, et al. Optimal correction of distinct CFTR folding mutants in rectal cystic fibrosis organoids. Eur Resp J 2016; 48:451-458.
22. Dekkers JF, Van Mourik P, Vonk AM, et al. Potentiator synergy in rectal organoids carrying S1251N, G551D, or F508del CFTR mutations. J Cyst Fibros 2016. [Epub ahead of print]
23. Mall M, Hirtz S, Gonska T, et al. Assessment of CFTR function in rectal biopsies for the diagnosis of cystic fibrosis. J Cyst Fibros 2004; 3 (Suppl 2): 165-169.
24. De Jonge HR, Ballmann M, Veeze H, et al. Ex vivo CF diagnosis by intestinal current measurements (ICM) in small aperture, circulating Ussing chambers. J Cyst Fibros 2004; 3 (Suppl 2):159-163.
25. Harris RC. Review of selected bacterial enterotoxins and their role in gastroenteritis. Ann Clin Lab Sci 1988; 18:102-108.
26. Arets HG, Brackel HJ, van der Ent CK. Applicability of interrupter resistance measurements using the MicroRint in daily practice. Respir Med 2003; 97:366-374.
27. Dekkers R, Vijftigschild LA, Vonk AM, et al. A bioassay using intestinal organoids to measure CFTR modulators in human plasma. J Cyst Fibros 2015; 14:178-181.
28. 2-adrenergic receptor agonists activate CFTR in intestinal organoids and subjects with cystic fibrosis. Eur Resp J 2016. [Epub ahead of print]