Success of gene drug trials boosts Ark
Britain’s gene therapy industry has taken a step forward with a medicine that may help cure brain cancer moving closer to receiving regulatory approval.
Ark Therapeutics, maker of Cerepro – which helps to stop cancer returning to the brain after a tumour is removed through an operation – yesterday said it had successfully manufactured a batch of the medicine to commercial standards and its facilities have met European regulatory standards.
Europe’s drugs regulator, the EMEA, will decide whether to ask further questions of Ark or to give the green light to marketing Cerepro.
The regulator is working out its policy on medicines made from genes as it goes along because there are no gene-based medicines on the market outside China.
Gene medicine has proved controversial because society has been suspicious of a science which alters human beings’ genetic make-up. There have also been examples of experiments going wrong, for instance when gene medicines have been injected into patients and have affected the whole body, rather than a specific diseased area, leading to severe side-effects including leukaemia.
However, this area is also regarded by scientists as potentially offering some of the most promising answers to illness. On Thursday results were released of a trial conducted by the US National Cancer Institute which showed gene therapy had eradicated skin cancer in two seriously ill men.
While only two out of a trial of 17 responded, the trial has been trumpeted as evidence that manipulating the cells in people’s bodies could be an effective medicine against diseases such as cancer.
Within gene therapy, there is a spectrum of ways in which human cells can be altered. Ark’s method is at the conservative end, and involves modifying a cell but not affecting its nucleus.
With Cerepro, which has been through phase 2 trials, cells are engineered to produce a protein which kills any cancer reappearing after a brain operation. Prevention of this type of recurrence is one of the biggest challenges in fighting this disease.
Nigel Parker, Ark’s chief executive, said: “This review is significant because it gives clarity to the regulatory requirements and standards that need to be met for gene-based medicines.”