Stem Cell Therapy Shows Promise as Treatment for Pituitary Gland Failure

However, this inefficient and complicated approach relied on ill-defined cellular signals, lacked reproducibility, and was not scalable or suitable for clinical-grade cell manufacturing.

To correct these deficiencies, investigators at the Sloan Kettering Institute for Cancer Research (New York, NY, USA) developed a simple and efficient strategy to derive human pituitary lineages using monolayer culture conditions suitable for cell manufacturing.

The method, which was described in detail in the June 14, 2016, online edition of the journal Stem Cell Reports, was based on the precisely timed exposure of hPSCs to a few specific cellular signals that were known to play an important role during embryonic development. In particular, the relative composition of different hormonal cell types could be controlled by exposing hPSCs to different ratios of two proteins: FGF8 (fibroblast growth factor 8) and BMP2 (bone morphogenetic protein 2).

Pituitary cells derived from hPSCs showed basal and stimulus-induced hormone release in vitro and engraftment and hormone release in vivo after transplantation into a rat model of hypopituitarism. The grafted cells secreted adrenocorticotropic hormone, prolactin, and follicle-stimulating hormone, and they also triggered appropriate hormonal responses in the kidneys of the rats.

"The current treatment options for patients suffering from hypopituitarism, a dysfunction of the pituitary gland, are far from optimal," said first author Dr. Bastian Zimmer, a postdoctoral researcher at the Sloan Kettering Institute for Cancer Research. "Cell replacement could offer a more permanent therapeutic option with pluripotent stem cell-derived hormone-producing cells that functionally integrate and respond to positive and negative feedback from the body. Achieving such a long-term goal may lead to a potential cure, not only a treatment, for those patients. Our findings represent a first step in treating hypopituitarism, but that does not mean the disease will be cured permanently within the near future. However, our work illustrates the promise of human pluripotent stem cells as it presents a direct path toward realizing the promise of regenerative medicine for certain hormonal disorders."

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Sloan Kettering Institute for Cancer Research