Some organoids only model a snapshot of the organ during a specific time during development. Some lack certain types of cells, or do not replicate the complexity of actual organs. Already these less-than-perfect systems are providing great platforms for experiments and facilitating breakthroughs that current animal models would not have allowed for.
In this study, a research team led by Jeffrey Beekman, Associate Professor at University Medical Center Utrecht, used rectal biopsies from 71 cystic fibrosis patients to grow individual gut organoids for each patient. Those organoids' reactions to drugs targeting CFTR in vitro were closely correlated with data from clinical trials of the same drugs. The video below, from the Lancet, is about the CFTR channel.
In cystic fibrosis, mutations in the CFTR gene cause corresponding dysfunction in the CFTR protein. As a result, thick mucous builds up and clogs the lungs, pancreas and other organs of those that suffer from it. However, different gene mutations manifest differently in patients, making personalized medicine very important for these patients.
In this work, scientists followed up on their laboratory findings to take action in the clinic. The investigators selected two patients with a rare and uncharacterized mutation in their CFTR genes for a specific treatment. Following that drug therapy, both people had improved lung function and displayed fewer symptoms of disease.
The following video talks a bit about growing organoids in culture.
Sources: AAAS, Nature, Science