Towards A Genetic Future

Better focused research and advanced genetics are helping healthcare companies develop the next generation of drugs. Sean Ottewell reports.

Spend more than a few moments studying a graph of investment against the number of new drugs launched and you might well need a bit of intensive healthcare yourself.

The main healthcare companies are currently investing over US$40bn every year in the hunt for new drugs. This is double the investment made in 1997 and nearly three times the 1992 figure. So far so good you might think.

But the problem comes when the figures for new drug launches are included. In the USA, only 24 arrived on the market in 2001. This dropped to 17 the following year. It’s the same story in Europe where the European Medicines Evaluation Agency received 31 new drug applications in 2002, down from 60 the year before.

To add insult to injury, it takes more than twice as long for a new drug to reach the market in the 21st century as it took in the 1970s.

One of the main causes of this has been the hugely increased costs associated with today’s stringent regulation of the healthcare industry. And even if the case can be made for taking a drug all the way from development and testing to the pharmacy shelf, there still remains the challenge of getting it adopted by the different health schemes run in the major markets of Europe and the USA.

Despite this, the healthcare industry does make healthy profits from its products and notable recent multi billion dollar successes such as Viagra show how valuable the prize can be.

Today the healthcare industry is developing new strategies to ensure that its resources are better focused on the likely success stories. One of the most important of these involves outsourcing technology and drug discovery to biotech companies.

Being smaller and faster moving, such companies are often better at managing the drug discovery process. Outsourcing takes the form of alliances, research agreements and collaboration.

A study by international market research company Frost & Sullivan (F&S;) has found that outsourcing is particularly common when developing drugs for the key market sectors of oncology, chemistry, broad-based screening, autoimmunity and inflammation. These alone account for nearly US$5bn worth of outsourcing deals today. Help is also at hand from the regulators.

The US Food and Drug Administration (FDA), which is responsible for licensing all drugs sold in the US, is acutely aware that ill people might not be getting the high-quality healthcare products they need as quickly as possible because of its lengthy licensing procedures.

As a result, the FDA has launched a two-year “smart regulation” initiative aimed at simplifying and speeding up the whole process.

“Using state-of-the-art approaches in the FDA’s many critical review and inspection activities will encourage innovation and continuous improvement in drug manufacturing to minimise production problems, and that will make it easier to get safe, high quality medications to patients who need them,” said Mark B McClellan, Commissioner of Food and Drugs. But what will the FDA be licensing in years to come?

A gene genie

The future is genetic – and controversial – with gene therapy and stem cells likely to produce the new blockbusters. Gene therapy involves inserting a working gene into a person whose own gene is either damaged or missing.

It sounds straightforward enough, but the technical challenges are immense. Early trials in the 1990s were at best inconclusive and at worst fatal.

One of the main challenges is deciding how best to deliver the gene to its target cells. Typically a virus is used, but one which has been genetically disabled. This sounds straightforward too.

After all, most people today are routinely vaccinated with weakened or disabled viruses at some point during their lives. However, in one recent gene therapy trial, the virus involved switched on a cancer-causing gene.

So, although the introduced gene was working well and overcoming the original ailment, the patient ended up with a mild form of leukaemia.

In theory, however, a gene for just about any protein could be inserted into a patient’s cell – massively increasing the potential for treating illness.

It is no surprise, therefore, that the first gene therapies are expected to hit the market in the next 2-3 years. By the end of the decade, sales could be worth US$6bn.

Looking further ahead, perhaps 20-30 years, stem cells could be used to cure a range of diseases, produce new organs and even rejuvenate almost any part of the human body.

Stem cells are found in human embryos, first appearing about a week after fertilisation, and go on to make all the cells of the adult body.

In animal experiments, stem cells placed inside damaged parts of the body have turned themselves into new cells of the appropriate type. Consequently, their potential to treat a vast range of human diseases – including Parkinson’s, diabetes and paralysis – is attracting much interest from scientists.

In theory, everyone could have a cloned embryo used to provide them with stem cells if needed as a result of accident or illness.

Again in theory, because such cells are clones of a person’s existing cells, there should be no chance of them being rejected by an immune system. It is regarded by life science companies as one of the most promising and potentially profitable areas of biotechnology – and one in which European companies have a world lead.

For the moment, however, such activities attract huge controversy. In late 2003, members of the European Parliament voted to allow EU money to be used to fund research on embryonic stem cells.

Although not binding, the vote will signal to healthcare companies across the continent that they don’t want the European lead in stem research to go to countries in the Far East

The author, Sean Ottewell, is a freelance writer and editor specialising in engineering and life sciences topics. As well as writing for many magazines and journals he also works with a number of international companies, helping them to prepare technical reports, in-house newsletters and speeches.