4.6 Review

Regenerative therapies in autoimmune Addison's disease

Journal

EUROPEAN JOURNAL OF ENDOCRINOLOGY
Volume 176, Issue 3, Pages R123-R135

Publisher

BIOSCIENTIFICA LTD
DOI: 10.1530/EJE-16-0581

Keywords

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Funding

  1. Medical Research Council: The Translational Stem Cell Committee [G0900001]
  2. Experimental Medicine-2 panel [G07017632]
  3. MRC [MR/J002526/1, G0900001, MC_PC_14101, G0701632] Funding Source: UKRI
  4. Medical Research Council [G0701632, MR/J002526/1, G0900001] Funding Source: researchfish
  5. National Institute for Health Research [CL-2014-01-002] Funding Source: researchfish

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The treatment for autoimmune Addison's disease (AAD) has remained virtually unchanged in the last 60 years. Most patients have symptoms that are relatively well controlled with exogenous steroid replacement, but there may be persistent symptoms, recurrent adrenal crisis and poor quality of life, despite good compliance with optimal current treatments. Treatment with conventional exogenous steroid therapy is also associated with premature mortality, increased cardiovascular risk and complications related to excessive steroid replacement. Hence, novel therapeutic approaches have emerged in the last decade attempting to improve the long-term outcome and quality of life of patients with AAD. This review discusses the recent developments in treatment innovations for AAD, including the novel exogenous steroid formulations with the intention of mimicking the physiological biorhythm of cortisol secretion. Our group has also carried out a few studies attempting to restore endogenous glucocorticoid production via immunomodulatory and regenerative medicine approaches. The recent advances in the understanding of adrenocortical stem cell biology, and adrenal plasticity will also be discussed to help comprehend the science behind the therapeutic approaches adopted.

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