Is there any evidence of successfully using organoids as working as mini organs for drug testing? I know the thought is there but is anyone using this successfully?


There is growing interest in using organoids in drug screening and the pharmaceutical industry is now looking to use the platform as well. However, there are still many challenges to overcome to make this technology routine for drug-screening. The first successes were documented in a subset of cystic fibrosis patients who carry a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)-gene. Vries team created organoids from rectal epithelium of patients with a host of CFTR mutations and measured organoid swelling in reaction to two drugs, ivacaftor and lumacaftor made by Vertex Pharmaceuticals. They found organoids from 2 patients carrying rare CFTR mutations responded to the drugs. Based on the in vitro success, these drugs were administered to the patients who responded favorably. This was the first success where events in the organoid-response corelated to physiological responses in the patient (Science Transl. Med. 2016. Vol 8, Issue 344:344ra84; Nature Rev. Drug Discov. 2017. 16: 6-7).