Revival of Stem cells in Addison's disease (RoSA)

• Research type

  Research Study

• Full title

  Revival of autochthonous adrenocortical stem cells in autoimmune Addison’s disease (RoSA)

• IRAS ID

  47702

• Contact name

  Simon HS Pearce

• Eudract number

  2009-018074-56

• ISRCTN Number

  Not available

• Research summary

  Autoimmune Addison's disease (AAD) is a rare and debilitating disease in which an autoimmune attack progressively destroys the adrenal cortex. Untreated it is universally fatal and treated people are absolutely dependent upon steroid medications lifelong, with a consequent excess in morbidity and mortality. A key feature of the adrenal cortex is that it is one of the most plastic tissues in the body, because its cells are responsive to changes in circulating adrenocorticotrophic hormone (ACTH) concentration. This study aims to regenerate adrenocortical steroidogenic cell function in patients with established autoimmune Addison??s disease (AAD) by stimulating proliferation and differentiation of their progenitor cells, the adrenocortical stem cells (ACSCs). The autoimmune attack in Addison??s disease is directed at the steroidogenic enzymes, which are not expressed by ACSCs. Therefore, these progenitor cells are likely to be spared the immune-mediated destruction of AAD, however, once steroid replacement treatment for AAD is started, the ACTH drive becomes lessened and these ACSCs become dormant. Using daily subcutaneous ACTH, administered according to two different regimens over 20 weeks, we will investigate whether regeneration of adrenal steroidogenic function through revival of ACSC activity is a realistic possibility. This would be a significant proof of principle, potentially spawning a new field of regenerative adrenal therapies in man.

• REC name

  North East - York Research Ethics Committee

• REC reference

  10/H0903/14

• Date of REC Opinion

  5 May 2010

• REC opinion

  Further Information Favourable Opinion