JUN 29, 2016 10:21 AM PDT

Mini-guts can Personalize Cystic Fibrosis Treatment

WRITTEN BY: Carmen Leitch
Organoids, basically a small in vitro model of an organ, could aid in identifying the best therapies for some people suffering from cystic fibrosis. A study published in Science investigated how mini-guts, simple versions of the human gut, might be used to match patients with the correct drugs, something currently possible but very time-consuming and expensive.
The response to CFTR-targeted drugs measured by swelling of intestinal organoids differs between subjects with cystic fibrosis. This material relates to a paper that appeared in the 22 June 2016, issue of Science Translational Medicine, published by AAAS. Image by Johanna Dekkers/University Medical Center Utrecht
Beginning in 2008, scientists took notice of the ability of stem cells to organize into balls of neurons that automatically organized into layers. Researchers have built on that idea ever since, attempting to grow rudimentary versions of human organs in the lab, to varying degrees of success.

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
About the Author
  • Experienced research scientist and technical expert with authorships on over 30 peer-reviewed publications, traveler to over 70 countries, published photographer and internationally-exhibited painter, volunteer trained in disaster-response, CPR and DV counseling.
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