Jonathan Watkins – How the 伯明翰大学 Supports Researchers Repurposing Drugs.


COVID-19大流行迅速改变了医疗保健的提供. 它促进了医疗诊断和设备的快速创新, 制造业也做出了灵活的回应, 需求导向的呼吸机和个人防护装备制造.

But the first round of clinical trials for COVID featured drugs that were already on the market, 目的是将它们重新用于一种新的疾病. 为什么是这个?

乔纳森·沃特金斯博士, 伯明翰企业大学知识产权负责人, explains why drug repurposing is the route of choice where speed is of the essence, and how it has worked for a Birmingham researcher in a very different clinical setting.

A white plastic cylindrical medical pill bottle with the grooves of a screw top at the neck of the vessel lies on its side. Between 10 and 20 oblong orange and white pill capsules are arranged as if they've spilled out of the open bottle. Bottle and pills alike are on a light blue background which takes up the entire frame of the photo


“While the discovery and development of new drugs remains at the heart of the work we do, the interest in repurposed drugs is growing both inside and outside the University. 这其中有明显的原因. Healthcare practitioners have around thousand licensed drugs to that they can use. However, of these, only around three to four hundred are used in routine medical care. 这种常规使用意味着我们对这些药物了解很多——它们的安全概况, 我们在人体中使用的剂量范围, 我们可以预见到副作用. It is also the quickest way of bringing effective therapies to patients who have few, 或没有, 治疗方案.”


辛克莱教授亚历山德拉, who is now a Clinician Scientist and Professor of Neurology at the 伯明翰大学, 注意到艾塞那肽的使用之间的相关性, 一种目前被批准用于治疗糖尿病的药物, and a reduction in brain pressure in patients with Idiopathic Intracranial Hypertension (IIH).

辛克莱教授当时正在临床环境中工作, running a neurology clinic specifically for people with this rare and debilitating condition.

Dr Watkins describes the way she first observed the correlation: “This was not a standard drug discovery. Usually scientists explore a disease with a particular drug candidate in the lab first to see what happens before the drug candidate goes anywhere near a patient. 在这种情况下, the starting point was a direct clinical observation from use in patients and then going in to the lab to understand it further and optimising the use of the drug for this condition.”

Professor Sinclair was preparing to present these observations at a conference. 但在展示结果之前, she decided to run the results past the IP team at the 伯明翰大学 to see whether her ideas about using Exenatide in IIH could be the basis for a marketable innovation.

Watkins and his team concluded that it was worth developing these ideas further and protecting the IP via a patent application. The team worked to file the patent application and also helped secure a mixture of external grant money and internal university funding to run a ‘human clinicial study’.

Watkins stresses that this type of study differs from a clinical trial for a new medicine. 这种药物已经被认为是安全的, so the study aimed to confirm the initial observation that had shown the potentially positive impact of Exenatide on people with IIH.


研究结果令人鼓舞, 再加上其他评估的数据, 他们提出了增加资金的有力理由.

Leveraging funds is a crucial part of the Technology Transfer role, and in drug repurposing. 但大学是第三部门机构, 虽然他们有很好的设备, 和创新研究, 他们无权进行金融投机.


The 伯明翰大学 assigned the rights to the IP to Australian biopharmaceutical company Invex Therapeutics. 同时, Invex在澳大利亚证券交易所上市, 为二期临床试验筹集资金. 该试验显示了疗效的明确统计证据, demonstrating Exenatide could deliver both an immediate reduction in brain pressure and a strong and sustained clinical benefit for patients at 12 weeks.

该公司目前正计划在IIH进行三期临床试验. The successful completion of this trial will help the researchers and the company to get the drug licensed for use with IIH and also to look at whether the drug can provide relief in other conditions marked, 或造成, 包括中风在内的脑部高压, 以及创伤性脑损伤.

It has taken nearly six years to get from the initial observations to this trial. This is substantially quicker than the amount of time it would have taken to develop a new drug from the ground up – and as we’ve seen recently, 当情况紧迫时,改变用途甚至可以更快.