Pituitary cells grown from human stem cells in mice may cure dwarfism

  Researchers have successfully used human stem cells to produce a functional pituitary gland that can secrete hormones. The hormones produced are critical to the body's stress response and are equally important to the body's growth and reproductive functions. When functional pituitary glands are transplanted into mice with hypopituitarism—a disease related to dwarfism and premature aging in humans—the pituitary cells grown in the laboratory can promote the release of normal hormones. The research that laid the foundation for future preclinical work appeared in the June 14th issue of the Journal of Stem Cell Reports, a publication of the International Association for Stem Cell Research.

   Bastian Zimmer of the Sloan-Kettering Cancer Institute, as the first study author, said: "The current treatment plan for patients with hypopituitarism, that is, pituitary dysfunction, is far from ideal." "Cell replacement therapy using pluripotent stem cell-derived hormone-secreting cells can provide a longer-lasting treatment option that can be functionally integrated and respond to positive and negative feedback from the body. Accepting such a long-term goal may guide these patients A chance for a potential cure, not just a treatment."

   Recently, scientists have developed a method to generate pituitary cells from human pluripotent stem cells-an unlimited cell source for regenerative medicine-using tissue culture to simulate the three-dimensional organization of the developing pituitary gland. However, this method is inefficient and complicated. It relies on ambiguous cell signals, lacks reproducibility, and is not scalable or suitable for clinically graded cell manufacturing.

  In future studies, the researchers plan to further expand this protocol to generate pure populations of various hormone-releasing cell types, so that the grafts produced can be tailored to the needs of individual patients. They will also test this method in more clinically relevant animal models that suffer from pituitary damage caused by radiation therapy and receive transplants at or near the pituitary gland rather than subcutaneously. Given that hypopituitary function is one of the main reasons for poor quality of life after brain radiotherapy, this research can have an important impact on cancer survivors.

  Zimmer said: "Our discovery represents the first step in the treatment of hypopituitarism, but that does not mean that the disease will be permanently cured in the near future." "However, our research work embodies the hope of human pluripotent stem cells, because it proposes a direct path towards the hope of achieving regenerative medicine to cure certain hormonal disorders."

   Researchers are funded by New York State Stem Cell Science and Starr Foundation. This work was further supported by the National Institutes of Health and the National Cancer Institute.