Stemcell Develops New Mini-guts Line Grown from CF Patients to Test Therapies

Using a cystic fibrosis patient’s tissue to grow mini-guts in a lab has become easier and is   available to more scientists who want to use it both to study the disease and test patients’ response to therapies.

Stemcell Technologies, a Canadian biotechnology company, has released two new lines of organoids for research — an intestinal line and a cerebral line. The company has worked for 20 years on organoid technology, developing new tools to deliver robust and reliable organoids that can model human diseases.

“The development of organoid culture techniques has expanded the limits of what scientists can accomplish in vitro. At Stemcell, we are committed to providing tools for culturing organoids to researchers across the globe,” Allen Eaves, founder and chief executive officer of Stemcell Technologies, said in a press release.

Organoids are grown from adult stem cells in certain tissue, such as intestines. In 2007, a team led by geneticist Hans Clevers in the Netherlands discovered stem cells in the gut. Clevers and his post-doctoral fellow Toshiro Sato found that they could isolate these cells from bits of tissue. Under the right conditions, they would self-assemble into structural and functional mini-intestines — or 3D structures known as intestinal organoids.

Since their discovery, organoids have become extremely useful for studying the mechanisms of human diseases and as a tool to test patients’ responses to therapies.

Patients with CF often develop inflammation and gastrointestinal problems, including poor digestion and absorption of nutrients.

Stemcell has partnered with Clevers and his team of researchers at the Foundation Hubrecht Organoid Technology (HUB) to develop a standardized culture median — called IntestiCult Organoid Growth Medium — to allow more researchers to work with intestine organoids.

Organoids can help identify which patients can benefit the most from certain therapies, like Kalydeco.

The U.S. Food and Drug Administration (FDA) approved Kalydeco (ivacaftor), developed by Vertex Pharmaceuticals, as a therapy for children with CF aged 2 or older who carry any of 38 mutations of the CFTR gene. However, despite the drug being described as highly effective, it doesn’t work for every patient.

Organoids work as personalized mini-guts when grown from a small piece of a CF patient’s tissue. This allows researchers to quickly and cheaply test in a lab how the intestines will respond to Kalydeco and other therapies.

“Organoid technology is now something we can apply to improve patient care and outcomes. We are currently using intestinal organoids to identify appropriate treatments for all 1,500 cystic fibrosis patients in the Netherlands,” said Clevers, founding director of HUB.

The company has also developed a new human cerebral organoid culture system that could become a valuable tool for studying neurological diseases.