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Cancer Research UK takes investigational multi-cancer drug from laboratory into unique trial

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by Cancer Research UK | News

18 January 2013

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Cancer Research UK’s Drug Development Office (DDO), in partnership with AstraZeneca, has opened a unique three-armed study of a new investigational drug called AZD0424 that is being tested for the treatment of a range of cancers.

This first-ever phase I trial of the AstraZeneca-owned drug is led from the Oxford Cancer Research UK Cancer Centre based at the Churchill Hospital. 

It will recruit up to 30 patients, initially across all solid tumour types. Later the design of the trial will be adapted, enabling the study to separate into three separate ‘personalised’ arms. 

Each of these future arms will test AZD0424 in different combinations alongside standard or other experimental treatments in specific patient populations. The Edinburgh and Belfast Cancer Research UK Centres are also involved as clinical sites, carrying out research to guide the drugs to be used in combination with AZD0424 and the specific patient groups involved in the trial.

This is the first time the DDO has undertaken an adaptive design trial of this type – one where the planned combination treatment to be given to patients is modified as the trial progresses.

Study leader, Professor Adrian Harris, Cancer Research UK clinician at the University of Oxford, said: “This unique study design means that for the first time, we’re able to monitor the data we receive in the first phase of the trial and feed this back into the study to adapt it as it’s happening – rather than having to wait until it ends – which could be several years.

This exciting approach will hopefully accelerate development to give us a better chance of identifying the most effective ways to use the drug either alone or in combination in specific patient groups.”   

AZD0424 works by blocking two proteins called Src and ABL1 – found in high levels in cancer cells. The proteins have an important role in cell growth and blood vessel development. Laboratory studies have shown that AZD0424 blocks these proteins, preventing delivery of nutrients via blood vessels to cancer cells – stopping their growth.

Cancer Research UK has carried out the preclinical development work of AZD0424 through the charity’s Clinical Development Partnerships (CDP) scheme. CDP is a joint initiative between the DDO and the charity’s commercial arm, Cancer Research Technology, to put drugs that otherwise would not be developed by pharmaceutical companies through early phase clinical trials. 

The scheme lets companies keep the background rights to their programmes while enabling Cancer Research UK to take on early development work, to see if there is a benefit to cancer patients.

Graham Richmond, AZD0424 project leader at AstraZeneca, said: “Collaborations such as this between AstraZeneca and Cancer Research UK are critical in the fight against cancer. By joining forces and combining our assets with external expertise, it means we can bring forward a wider range of experimental compounds than we could do simply using our own resources and – as a result – patients get access to a trial of a potential new cancer treatment earlier.

“AstraZeneca was the first pharmaceutical company to have a strategic alliance with Cancer Research UK focused on developing novel combinations of experimental cancer drugs through early phase clinical trials.”

The trial is also supported by the National Institute for Health Research Oxford Biomedical Research Centre, and the Cancer Research UK and National Institute for Health Research (NIHR) Experimental Cancer Medicine Centre Network.

Dr Vicky John, head of clinical partnerships, at Cancer Research UK’s DDO, said: “We’re delighted that our unique CDP scheme has made it possible to launch the first trial of this promising drug.

“We’re enormously proud of our successes so far from the initiative, which allows us to work alongside industry to take on and develop deprioritised potential cancer treatments – which otherwise may not have reached patients for many years.

“There are now eight drugs in our CDP portfolio – including a multipeptide vaccine, a monoclonal antibody and other molecularly targeted drugs. Four treatments have already successfully entered trials** with others scheduled to open early 2013. This demonstrates how our strong collaborations with international pharmaceutical and biotechnology companies have provided new experimental drugs for patients on trials, for whom existing drugs no longer work.  

“We intend to continue to seek future partnerships, to develop more potential drugs and ultimately achieve our goal to save more lives from cancer.”


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