Current and future medical treatments for patients with acromegaly

ABSTRACT

Introduction: Acromegaly is a relatively rare condition of growth hormone (GH) excess associated with significant morbidity and, when left untreated, high mortality. Therapy for acromegaly is targeted at decreasing GH and insulin-like growth hormone 1 levels, ameliorating patients' symptoms and decreasing any local compressive effects of the pituitary adenoma. The therapeutic options for acromegaly include surgery, medical therapies (such as dopamine agonists, somatostatin receptor ligands and the GH receptor antagonist pegvisomant) and radiotherapy. However, despite all these treatments option, approximately 50% of patients are not adequately controlled.

Areas covered: In this paper, the authors discuss: 1) efficacy and safety of current medical therapy 2) the efficacy and safety of the new multireceptor-targeted somatostatin ligand pasireotide 3) medical treatments currently under clinical investigation (oral octreotide, ITF2984, ATL1103), and 4) preliminary data on the use of new injectable and transdermal/transmucosal formulations of octreotide.

Expert opinion: This expert opinion supports the need for new therapeutic agents and modalities for patients with acromegaly.

KEYWORDS:

• Acromegaly
• antisense molecules
• GH
• IGF-1
• pegvisomant
• somatostatin analogs
• somatostatin receptor subtypes

Article highlights

• Surgery remains the first treatment choice for growth hormone secreting pituitary adenoma, however half of all acromegaly patients require long-term medical treatment.

• Somatostatin receptor ligands are the mainstay in the treatment of acromegaly with beneficial effects on growth hormone hypersecretion and tumor growth.

• In the past few years, research has focused on medical treatments potentially able to obtain better biochemical results, less side-effects, and improved patient’s compliance.

• Multireceptor-targeted somatostatin ligands, such as pasireotide, were shown to be effective in acromegaly.

• New formulations of somatostatin receptor ligands, such as oral and transdermal octreotide, have been developed to improve the patient acceptability for a long-term treatment.

• Antisense technology has provided molecules targeting the growth hormone receptor which may request a novel ‘peripheral’ approach to future treatment of acromegaly.

This box summarizes key points contained in the article

Acknowledgments

We thank Marco Formenti for his precious contribution in Figure drawing.

Declaration of interest

G Mazziotti received consultancy fees from Novartis and Ipsen and received lecture fees from Ipsen. A Giustina is a consultant for Ipsen, Novartis and Pfizer. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.