Researchers have developed the world’s first congenital pituitary hypoplasia (CPH) model using patient-derived iPS cells. The research group succeeded in using the model to illuminate the mechanisms underlying CPH. The team has been attempting to apply this model to other pituitary diseases and drug discovery.

The results of this study were published in the American Scientific Journal ‘J Clinical Investigation‘ on December 17, 2019.

Research Background

Hypopituitarism caused by CPH is not uncommon and it is sometimes life-threatening. Patients with this disease require lifelong hormone replacement therapy. The causes and underlying mechanisms are not well understood.

Prior research on pituitary diseases has been mainly conducted using animal models, such as knock out mouse (*5). However, sometimes there are differences in the phenotypes between animal and human. This means that human models are necessary in order to fully understand the mechanisms of such diseases.

In recent years, iPS (induced pluripotent stem) cells have been utilized in the development of disease models, regenerative medicine, and drug discovery. In addition, a method using iPS cells to induce differentiation of both the pituitary and hypothalamus in vitro has been developed; however it had yet to be applied to pituitary disease modeling.

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