Completed Diabetes, Hormones & Metabolism Lungs & Breathing

Development of a long-acting growth hormone antagonist to address the unmet need for treatment in acromegaly

Funder MRC (part of UKRI)
Recipient organisation University of Sheffield
Funding £2.6M
Period Feb 2015 — Oct 2018

In plain English

AI plain-English summary

Acromegaly patients currently face daily injections or drugs that fail to control their disease. The condition, caused by excess growth hormone, doubles mortality rates and causes heart failure, diabetes, and disfigurement. Surgery fails in half of patients, leaving them reliant on inadequate drug therapies. This project aims to turn a new technology called Profuse into a single, long-acting injection that blocks growth hormone. The drug is designed to control the disease in more than 95% of patients, while being cost-effective enough for routine NHS use. The three-year grant will produce a version ready for first-in-human trials. If successful, the treatment could replace daily injections with a single dose lasting weeks or months. For patients, that means fewer hospital visits, better disease control, and reduced risk of heart failure and early death. For the health system, it offers a cheaper alternative to current therapies that cost thousands per patient per year. The next steps would be clinical trials for market approval.

View original technical description
Acromegaly is a disease of excess growth hormone secretion and if untreated patients suffer from disfigurement, hypertension, diabetes, arthritis and heart failure. Acromegalic patients have twice the mortality of the general population. First line therapy is surgery but in 50% of patients the tumours are inoperable or cannot be fully removed and these patients need drug therapy. Current drug therapy is not satisfactory as it either only controls the disease in a proportion of patients or involves daily injections with a drug that is not cost effective. We have developed a new technology, Profuse, for making a long-acting drug that blocks growth hormone actions and is expected to be cost effective and control the disease in >95% of patients. The DPFS application is for a 3 year grant to deliver a Profuse drug ready for clinical proof-of-concept. It is the first phase required to bring the Profuse drug to market. The next stages are a clinical trial (Phase 1/2a proof of concept trial) in patients, followed by Phase 2 & 3 trials for market authorisation.

View the original record at the funder ↗

Researchers

Ian Robert Wilkinson (Co-Investigator)Jon Sayers (Co-Investigator)R Ross (Principal Investigator)S Dixon (Co-Investigator)

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Original classification

Research Grant

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