Europe's largest cancer charity pushes new treatments to market

Cancer Research UK is stepping up its efforts to increase the number of cancer treatments that make it to market, with a plan to rescue viable compounds stuck on the shelves of pharma and biotech companies, and the opening of a North American technology transfer office in Boston.

In the charity’s scheme to revive the fortunes of marooned compounds it will “borrow” drugs and put them through the next stage of clinical development free of charge. Companies will retain intellectual property rights to their original molecules and have the first option to view the trial data.

If the owners subsequently take the compounds through to commercialisation the charity will receive royalties. But if they chose not to do so, Cancer Research UK will have the right to find a licensee that will.

Harpal Kumar, chief operating officer of the charity, says clinical development partnerships are part of the charity’s ambition to double its drug development activity over the next five years. “We are seizing an opportunity to seek out new treatments that otherwise might not get developed.”

There are many reasons why effective compounds do not make it to market. For a start there are far more promising candidates around than there are the resources to develop them. With new cancer treatments taking £500 million and eight years to develop, anything that does not look extremely promising and address a large potential market is likely to be left languishing.

In addition, drugs may be left on the shelf as a result of company merger, they may have failed because trials were poorly designed, or they could be in development for other indications when new biological knowledge shows potential utility in cancer.

An example of this appeared in the British Journal of Cancer on Tuesday (16 May). Researchers at the Thoracic Oncology, Surgery Branch of the US National Cancer Institute’s Center for Cancer Research, led by Dao Nguyen, report that a combination of two drugs, including one commonly used to treat epilepsy, has proved effective at killing cancer cells. The journal is published by Cancer Research UK.

The group was researching ways to increase the potency of an emerging new class of cancer drugs based on histone deacetylase inhibitors. They found that valproic acid, a well-known antiepileptic, was in fact, one of these inhibitors. It was tested in combination with a second compound, designated UCN-01, which has been used in clinical trials.

The researchers are continuing to research combinations of valproic acid and other agents and Nguyen said he believes combinations including valproic acid will become useful treatments.

Stuck in pipelines

Kumar believes companies are held back from attempting to capitalise on drugs that are stuck in their pipelines by the embarrassment factor of outlicensing a compound that becomes a commercial success in someone else’s hands. The advantage of letting the charity take compounds forward is that the charity’s interest is not in making money but in generating more treatments.

To date, of all the agents that have completed Phase I trials under the auspices of the charity, more than 5 per cent have subsequently made it to market – a conversion rate that matches that achieved by the pharmaceutical industry.

“The drug companies have these potential treatments trapped in their pipelines and we have the expertise and capacity to release this potential,” said Kumar.

For Cancer Research UK the cost of increasing the number of trials it conducts is marginal because it has a network of 14 trial centres and scientists, with the facilities to carry out Phase I and Phase II trials, already in place.

As yet, no partnerships have been established but Kumar said he has talked to all the large pharmaceutical companies and each has expressed interest in the scheme.

The charity is prepared to take on both small molecules and biopharmaceuticals. It is interested in compounds with novel modes of action and would take on drugs that were developed for another indication, which it is now thought might be relevant to cancer.

In some cases it is expected that compounds will have full preclinical toxicology package, and material available for study. But this is not essential and at a minimum, the charity will want in vivo proof of principle data, and the means to produce more material to carry out the further work required to get a drug into the clinic.

For products that make it to market the share of the revenues will be based on the value each party has added to the project, and will vary depending on the type of agent, the stage of development and the availability of material  

US licensing company

In a further push to increase the number of cancer treatments Cancer Research UK has set up a technology transfer subsidiary in the US to in-license and commercialise research from US institutes.

Cancer Research Technology Inc is based in Boston and headed by Larry Steranka, former executive director of Brandeis University's Office of Technology Licensing, and a former associate director for licensing at Harvard University.

The aim is to accelerate commercialisation of new cancer therapies. To date its UK arm, Cancer Research Technology Ltd (CRT), has been involved in forming 13 companies and has 18 compounds in clinical development, 16 of which are out-licensed.

There are two attractions for US academics of doing deals with Cancer Research Technology Inc rather than home-grown technology transfer organisations. The company stands out from its counterparts because of its sole focus on oncology, and because it has a development laboratory in which it can carry out proof-of-concept studies, adding value and enhancing the potential for commercialisation of any product.

Development fund

Apart from being the largest cancer charity in Europe, Cancer Research UK is the world's largest independent funder of oncology research, and has a development fund through which it can finance proof of concept studies.

The development laboratory has expertise in molecular and cell biology, drug discovery services, early pharmacology and medicinal chemistry. CRT sponsors clinical trials, also.

Keith Blundy, chief operating officer of CRT and chairman of the new Boston-based subsidiary, said the US office, “will reinforce our strategy of oncology-focused development of diagnostics and therapeutics in partnership with leading US academic institutions”.

When CRT was formed from two existing bodies in 2002 its main focus was on out-licensing research sponsored by Cancer Research UK. It then branched out into providing technology transfer services to other publicly funded research institutions. In recent months, CRT has expanded its commercialisation activities yet further, by in-licensing technology for further development from commercial companies, and also carrying out co-development work with companies.

For example, it recently agreed to a development collaboration with Cronos Therapeutics of London to use Cronos' chromatin-based gene inactivation platform to inhibit gene expression of an undisclosed apoptotic target.

And in February CRT signed a deal with Stealthyx Therapeutics to co-develop the London-based company's Prothyx drug delivery platform, which adds functionality to drug molecules, making them preferentially active at sites of diseases, thus minimising side effects and enabling higher doses to be given less often.