A new study by Frost & Sullivan, the international market consultancy, believes that the European market will see the first commercial launch of gene therapy in 2005. Despite sustained caution, the first successes are a welcome milestone for gene therapy, which has been under fire since the death of a volunteer during experimental treatment in 1999. Despite an inquiry into the patient's death placing blame on the trial investigators and the patient's suitability for inclusion, the setback has been extremely severe.

Christian Downton, Research Analyst at Frost & Sullivan, reports that a new generation of gene therapy will be tested in human clinical trials in the coming years. Perhaps the most crucial advantage these trials will provide in terms of safety is the ability to target the gene of interest to a specific position on a specific chromosome.

Believed by some to be the Holy Grail of medical research, scientists both in and out of the field, as well as big pharmaceutical companies, are eagerly awaiting success stories in gene therapy. They recognise the promise it offers, particularly in the post-genomic era, and realise that once it has gained "proof of principle", popular interest in gene therapy will be enormous.

Downton explains that establishing "proof of principle" will have a manifold effect on the market. "Firstly, opinion leaders are ready to receive gene therapy products for use in medicine. Secondly, pharmaceutical R&D directors are poised to restart substantial investment in this field, which has waned over the past few years. Thirdly, the current 'baited breath' situation could release substantial market noise on a first launch, propelling other developments into the limelight."

"Technology is also progressing to alter vectors so they can target particular tissue types. This would reduce the need for direct injection of gene therapies and would further minimise the systemic effects where these are not needed," Downton continues.

Gene therapy has already broken away from the initial constraints of monogenic diseases. Monogenics still offer the greatest opportunity to gene therapy in terms of "proof of principle" and prestige but are unlikely to provide the greatest returns on the technology because of the low patient populations.

"Ten to fifteen years into the future, we are likely to see gene therapy applications gaining blockbuster status similar to other biotech products, like Amgen's Epogen (epoetin alfa). These blockbusters, though, are most likely to come from major infectious and chronic diseases instead of niche cancer and associated applications."

Initial revenues will most likely be derived, to an equal degree, from the cancer and vascular disease markets in 2005. Further into the future, it is expected that vascular diseases will take the lead in generating revenues for the gene therapy market as a whole, as new products are launched and penetration of the large available population increases.

The modestly populated cancer indications imply that this will have become a less significant proportion. However, from 2008, further indications and several new products gain importance in cancer gene therapy and produce a predicted 28 per cent of the USD 1.1 billion revenues in 2010.

Advexin (ad5CMV-p53) is expected to be launched by Introgen's as yet unappointed European partner for the indication of advanced head and neck cancer in 2005. The second product, Generx (ad5-FGF4) is expected to be launched by Schering AG in the same year for the indication of coronary artery disease (CAD).

These two products could well provide the "proof of principle" needed for this market in two key areas - cancer and vascular diseases. Monogenic diseases are set to experience revenue growth in 2006. The successful launch of products in each of these areas will pave the way for further gene therapy products to make an impact on their respective markets.

"The gene therapy market is extremely diverse in terms of technology and disease type and is currently typified by more of a collegiate feel rather than a competitive edge. Nevertheless, as the investment community and pharmaceutical companies continue to squeeze greater performance out of companies in the field, and products begin to appear on the market, the levels of competition will increase dramatically," the study adds.

Aventis, Novartis and Schering AG are at the forefront of gene therapy developments and this is likely to catapult them into the lead when gene therapy finally takes off.

Downton concludes: "The general consensus in the industry is that once "proof of principle" has been achieved, the excitement surrounding this field will find its initial impetus once again as gene therapy's future is being reconsidered by its stakeholders."

"However," he stresses, "those companies involved in this wide-reaching field have a responsibility to educate the public and medical professionals that this technology works and is safe. Describing products with euphemisms may limit the medium-term stock price damage, but in the long-term merely confuses the public and does little to settle the gene therapy market as a whole."