The first human organoid transplantation research yields positive results

The research team from Tokyo Medical and Dental University (TMDU) announced on July 7 that the world’s first clinical transplantation of a “mini organ (also called Organoid)” into a patient with “Ulcerative Colitis (UC)” was successful. UC causes ulcers and inflammation in the digestive tract. It can be incapacitating and occasionally result in life-threatening complications. UC is a member of the group of conditions known as “Inflammatory Bowel Disease” (IBD). It is estimated that the number of patients in Japan and throughout the world is rising to between 220,000 and 5,000,000. In severe cases, the entire colon may be removed. The standard treatment for colitis is anti-inflammatory drugs.

If our first-in-human research using organoid transplantation yields positive results, we anticipate the advancement of organoid medicine for intractable digestive tract diseases such as Crohn’s disease.”

Dr. Ryuichi Okamoto, a professor at the Graduate School of Medical and Dental Sciences’ Department of Gastroenterology and Hepatology, stated, “We embarked on the path of developing new methods for treating incurable diseases. This treatment should quickly establish its efficacy and safety and be administered to patients.” If the team’s efforts are successful, the mucous membrane could regenerate, leading to a complete cure for UC.

The clinical research began by collecting healthy colonic mucosa from the patient’s vicinity and cultivating it for approximately one month to form spherical organoids with a diameter of approximately 0.1 to 0.2 mm. Using a colonoscopy, an organoid was transplanted into the colon of the same patient on July 5. The patient recovered and was released on July 6.

In previous experiments with mouse models, the team confirmed that when the cells were cultured in organoids and then transplanted, the mucous membranes regenerated within a month and the clinical course improved, whereas the stem cells alone did not transplant because they were incapable of in vitro culture.

Since the patient’s own cells are utilised in this clinical study, there is no risk of transplant rejection. In addition, since colonoscopy is used for collection and transplantation, laparotomy is unnecessary and minimally invasive treatment is possible.

After this transplant, a medical examination will be conducted four and eight weeks later. To confirm safety and efficacy, the patient will be observed for up to a year. An additional transplantation of organoids will be performed on up to eight patients.


Journal Reference

Sağraç D, Şişli HB, Şenkal S, Hayal TB, Şahin F, Doğan A. Organoids in Tissue Transplantation. Adv Exp Med Biol. 2021;1347:45-64. doi: 10.1007/5584_2021_647. PMID: 34164796.

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