A cannabis-based medicine which had excited the hopes of many multiple sclerosis patients has been refused a licence.
Many people with the devastating disease have used cannabis illicitly to relieve the symptoms, such as spasticity and nerve pain, so there had been great expectations of the medicine, produced as a nasal spray called Sativex, which had been undergoing trials for several years.
But the Medicines and Healthcare Products Regulatory Authority announced that the licence application to treat spasticity filed by GW Pharmaceuticals, which designed and manufactured the drug, had been turned down on appeal. The MHRA said there was not enough proof that the drug worked.
"We acknowledge that this decision may cause disappointment to MS patients. However, there was insufficient evidence to show that the risk-benefit relationship was favourable," said Kent Woods, the authority's chief executive.
Patient groups were dismayed and the company's chairman, Geoffrey Guy, said he was "very, very frustrated". He said further trials were under way to try to obtain the data that would convince the regulator to grant a licence.
The MHRA is satisfied that the drug is safe and of good quality - but it has fallen at the third hurdle, efficacy.
Dr Guy said his company had won the battle to have patients' reports of their improvement considered, rather than an assessment of how much better they were on a disputed clinical scale. But another trial must now be run to obtain data from larger numbers of patients.
Mike O'Donovan, chief executive of the Multiple Sclerosis Society, said: "This news will be greeted with dismay by many of the 85,000 people in the UK who have MS and suffer from distressing symptoms, including spasticity and pain. How long must they wait? When the society spoke at the Medicines Commission hearing we stressed that the choice of available treatments was limited, and many people did not find them effective. It was emphasised that measurements to assess new treatments for a fluctuating condition like MS do not always detect significant benefits patients feel.
"We believe there is convincing evidence that cannabis-derived drugs can significantly improve their quality of life."
Christine Jones, chief executive of the MS Trust, agreed. "It is extremely frustrating that the prospect of a licensed cannabis-derived medicine is still no closer," she said.
"Unfortunately neither the Medicines Commission nor the MHRA appears to be willing to take account of the very positive and statistically significant feedback from people with MS, who report relief from spasticity."
Sativex has already been given a licence for nerve pain relief in Canada, although this was conditional on more trials being done. Dr Guy said the company had a lot of evidence that the drug was effective in pain relief - but was told to apply for a licence for spasticity by the MHRA, only to be told that the efficacy data was not sufficient.
He said the drug was very helpful to some people in the trials. "The investigators see some patients' lives being totally changed beyond recognition," he said. "There are no other medicines that treat these patients. All our work was done in patients for whom all other treatment possibilities had been exhausted."
There are 400 patients in the UK still taking Sativex because they were in the trials. Other patients may seek to get it from Canada, he said, but they will need a prescription from a doctor who believes the drug can help, approval from the MHRA, and an import licence from the Home Office, because cannabis is a schedule 1 drug.
Dr Guy said he would now push for cannabis to be rescheduled, because therewas proof that it had a medical use, both from Canada and from the UK trials. Schedule 1 drugs are defined as having no therapeutic value.
