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

开发长效生长激素拮抗剂，以满足肢端肥大症治疗未满足的需求

• 批准号: MR/M015491/1
• 负责人: R Ross
• 金额: $ 333.59万
• 依托单位: University of Sheffield
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2015
• 资助国家: 英国
• 起止时间: 2015 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=MR%2FM015491%2F1
• 关键词: Development; long; acting; growth; hormone

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.

肢端肥大症是一种生长激素分泌过多的疾病，如果不治疗，患者会出现毁容、高血压、糖尿病、关节炎和心力衰竭。肢端肥大症患者的死亡率是普通人群的两倍。一线治疗是手术，但在50%的患者中，肿瘤无法手术或无法完全切除，这些患者需要药物治疗。目前的药物疗法并不令人满意，因为它要么只在一定比例的患者中控制疾病，要么需要每天注射一种成本效益不高的药物。我们已经开发了一种名为FUFUSE的新技术，用于制造一种阻止生长激素作用的长效药物，预计将具有成本效益，并在95%的患者中控制疾病。DPFS的申请是为了提供一种用于临床概念验证的丰富药物的3年赠款。这是将这种储量丰富的药物推向市场所需的第一阶段。下一阶段是在患者身上进行临床试验(第1/2a阶段概念验证试验)，然后是第2和第3阶段的上市授权试验。

项目成果

A long-acting GH receptor antagonist through fusion to GH binding protein.

• DOI: 10.1038/srep35072
• 发表时间: 2016-10-12
• 期刊: Scientific reports
• 影响因子: 4.6
• 作者: Wilkinson IR;Pradhananga SL;Speak R;Artymiuk PJ;Sayers JR;Ross RJ
• 通讯作者: Ross RJ