Letter from America: Buy and cell

Angelina Jolie
Image caption Angelina Jolie opted for a double mastectomy after testing for a gene seen as an indicator of possible breast cancer

The search for a cure to cancer is not one for the impatient, or those short of money.

But at least one small team of Scottish researchers may be getting close to something significant.

They are developing and trialling a drug that may yet prove to increase the life of an elderly patient with acute myeloid leukaemia (AML) - a condition for which the most recent new treatment was approved 44 years ago.

If it does so, by turning the cancer cells behaviour back on themselves, there could be applications far beyond that.

There are only nine in the research team, working in Dundee for a company called Cyclacel. In the 1990s, it spun out from the Dundee University work led by Sir David Lane, finding a gene common to more than half of people who suffer from cancer. He's since departed for a rather more attractive research post in Singapore.

The other nine employees of the company are in a 'corporate park' of impressive office blocks set amid original woodland and manicured lawns a short drive from New York.

While visiting the US, I've just visited them to learn more from president and chief executive Spiro Rombotis. He spent 10 years living in Dundee, building up the company.

But he had to get much closer to the specialists in life science investment within Wall Street's big financial firms.

Nowhere in Europe is there that expertise or the liquidity of life science capital markets to sustain the kind of investment that Cyclacel needs if it's to get through the hugely demanding and hugely expensive process of satisfying America's pharmaceutical regulator, the Food and Drug Administration.

This week, Spiro Rombotis updated investors with news of further progress with the trials. The latest findings indicate that a person aged over 75 with AML may live 9.4 months, while using the drug sapacitabine, whereas those without it live on average for only 3.4 months. They are both harsh figures, but such is the measurement of progress in drug development.

'Persistent and humble'

The staggering bit of this business - at least for anyone who's not in it - is the cost. The first quarter results from Cyclacel, issued this week, showed that losses have gone up slightly, to $3m, or £2m.

By most business standards, that doesn't look good. But in this line of work, that's to be expected. In 2012, there was revenue of only $112,000, but a loss (the measure applicable to shareholders) of $19m (£12.6m).

The annual report looks back to August 1996, with the inception of Cyclacel. It shows that the firm has received research grants making up revenue of $6.8m over 16 years. But in that same time, it's sustained losses totalling nearly $313m. That's more than £200m.

So this requires committed investors, who know what they're doing. It's reckoned that the average drug costs around $1bn to bring to market, and the attrition rate is colossal.

According to Spiro Rombotis: "This is a marathon - you have to be patient, persistent and also extremely humble to take on such a huge challenge."

The chance of success? "Before we enter pre-clinical investigation, less than one in 100. Once we enter clinical trials, maybe 10%. After we got to phase two, 40%. And in phase 3: 60%".

With Cyclacel now a third of the way through the third phase, you can expect to hear more from Mr Rombotis on BBC Radio Scotland's Business Scotland programme later this month.

Faulty gene

But that preview provides a bit of context for a big controversy relating to the news, published in the New York Times on Tuesday, that the actress Angelina Jolie has opted to have a double mastectomy, without having been diagnosed with breast cancer.

A test showed that she carries the same genetic sequence that led to her mother's early death. Whereas most people have a sequence called BRCA1, which helps to suppress tumours, some people are born with a faulty version of it, so tumours go unchecked. That condition gave Ms Jolie (her doctors told her) an 87% chance of contracting the same cancer by the age of 70. It's also believed to lead to a 44% chance of ovarian cancer.

The actress was encouraging other women facing the same predicament to face up to it, and she has been widely praised for doing so. One reckoning is that around 7% of breast cancers are related to the BRCA1 mutation, or the similar BRCA2.

The controversy is that the test costs more than £2,000, and that's because the BRCA1 genetic sequence was discovered by, and is owned by, Myriad Genetics. This is a molecular diagnostics firm, based in Salt Lake City, Utah, with a monopoly of the testing and the ability to put constraints on further research into the sequence.

As Cyclacel has also recently found, this is an expensively litigious business in an expensively litigious country. Myriad is being sued by a group representing academic researchers, claiming it cannot be right that genetic sequences can be owned as intellectual property.

How can you own something which is naturally occurring, and protect it for your own commercial interest, goes one side of the argument? But without intellectual property rights, goes the industry, such research won't be done and cures won't be developed.

The US Supreme Court is due to rule on that later this year, with huge implications for the development of genetically-based personalised medicine.

In the meantime, the Angelina Jolie publicity was not only good for the health of women facing the same genetic condition. It was good for business: Myriad's stock price in New York rose by 4%.

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