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Generation of human steroid-producing organoids: a new approach to treat adrenal insufficiency

Periodic Reporting for period 1 - GHSO (Generation of human steroid-producing organoids: a new approach to treat adrenal insufficiency)

Reporting period: 2019-06-01 to 2021-05-31

Adrenal insufficiency is a disorder that occurs when the adrenal glands do not produce enough levels of certain hormones, including cortisol, a hormone essential for life. Current treatments for adrenal insufficiency are based on hormone replacement therapy (HRT), consisting of 2 or 3 daily doses of the missing hormones in a pill format.
The general purpose of this proposal is to find alternative treatments for patients suffering from adrenal insufficiency using cells that can produce hormones, generated in the laboratory.
The overall significance and importance of this technology is the ability to transplant hormone-producing cells that can be properly regulated by the patient’s body, responding with adequate hormone secretion in a time and dose dependent manner. To avoid rejection of the implanted cells by the immune system, we aim to encapsulate (protect) cells into devices developed for this purpose.
The two main milestones achieve during the first two years of the fellowship were: 1) We have been able to generate billions of steroid-producing cells. 2) Steroid-producing cells can produce the human steroid cortisol, one of the main regulators of human physiology.
If animal in vivo experiments proof the success of this technology, patients suffering from adrenal insufficiency can benefit from a significant improvement of their quality of life. Instead of taking life-long medication, which it is far from optimal, patients can benefit of a cellular therapy that can autonomously sense the physiological requirements of the body during time.
The two main milestones achieve during the first two years of the fellowship were: 1) We have been able to generate billions of steroid-producing cells. 2) Steroid-producing cells can produce the human steroid cortisol, one of the main regulators of human physiology.
If animal in vivo experiments proof the success of this technology, patients suffering from adrenal insufficiency can benefit from a significant improvement of their quality of life. Instead of taking life-long medication, which it is far from optimal, patients can benefit of a cellular therapy that can autonomously sense the physiological requirements of the body during time.
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