CF Patients Discuss Their Hopes and Concerns About Organoid Technology

Organoid technology has created hopes of improved, personalized treatment for cystic fibrosis, but has also posed challenges and generated doubts.

Organoids are three-dimensional, engineered cell structures built from stem cells that mimic the architecture and function of human tissue. These mini-organs have the potential to improve our knowledge of disease-related mechanisms and evaluate patients’ responses to therapies.

Dutch scientists wanted a better idea of CF patients’ and caregivers’ perceptions of the technology. The team from University Medical Center Utrecht conducted the project in collaboration with the Dutch Cystic Fibrosis Foundation.

The researchers felt that having the patients’ and caregivers’ perspective could help scientists do a better job of setting the organoid research agenda. This perspective “is necessary for responsible development of this ethically challenging technology,” they wrote.

The study, “Mini-guts in a dish: Perspectives of adult Cystic Fibrosis (CF) patients and parents of young CF patients on organoid technology,” was published in the Journal of Cystic Fibrosis.

Organoids have mainly been used in a research setting. But advances in the technology have a chance of affecting patients’ care and their lives, particularly those with rare mutations. Such technology could be a game changer for patients with CF, because it offers a strikingly accurate and personalized model of the disease.

But the use of organoids has raised several ethical concerns and challenges, many of which have also been debated for other technologies, such as induced pluripotent stem cells (iPSCs).

The Dutch researchers asked 14 adults with CF and 12 parents of young patients about their experiences with organoid technology and their thoughts about it.

The answers fell into four categories:

• Ambivalence toward the technology.
• Hopes, but also concerns, about it.
• Concerns about its use as a treatment.
• Interest in patients being involved in its development and the rights that organoid donors would have if the technology is commercialized.

In general, the respondents supported the technology. The idea that they may benefit from it makes them more inclined to donate organoids built from their own stem cells. But they also expressed concern about how the donations could be used beyond their own treatment.

Another issue was concern that the technology would lead to very expensive treatments. “This fear is not unfounded given the high prices of the latest generation of CF drugs,” the researchers wrote.

A number of the survey participants said organoid donors’ rights must be ensured if the technology is commercialized. Steps they believed need to be taken include initial donor consent, long-term patient engagement in the process, responsible stewardship of the technology and its use, and stringent conditions for its commercial use.

Although the technology holds great promise for personalized CF therapy, “this can only be realized if the perspective of the person in question — i.e., the patient — is taken adequately into account,” the team wrote.