After a 20 year struggle overcoming scientific barriers and commercial sharp practice, scientists at Cambridge University today announce a breakthrough in the treatment of multiple sclerosis which could have a “transformative” effect on sufferers from the debilitating disease.
Two trials of alemtuzumab have shown that it is the most effective treatment yet discovered against the chronic condition and could be suitable for two thirds of all new patients. Multiple sclerosis affects 100,000 people in the UK and millions worldwide.
But the company behind the breakthrough, Genzyme, is under a cloud after it denied access to the drug for existing patients pending the outcome of the trials, triggering a protest by leading neurologists to health secretary Jeremy Hunt, as revealed in The Independent.
One of the scientists who led the trials, published today in The Lancet, said yesterday that three of his own patients had been unable to complete treatment with the drug because of the company’s action.
Genzyme, part of the pharmaceutical giant Sanofi, is also expected to raise the price of alemtuzumab by up to 20 times when it applies for a licence next year, potentially putting it beyond reach for many patients.
The Lancet says in an editorial: “Multiple sclerosis runs a chronic and progressive course eventually disabling many patients. More effective, affordable, evidence-based treatment with long-term benefits are desperately needed. Finding promising treatments such as alemtuzumab is important. But so is keeping alemtuzumab accessible and affordable if its early success proves to be of enduring value.”
Alemtuzumab was developed as a treatment for leukaemia by scientists at Cambridge University in the 1970s but was later discovered to be effective in multiple scleroisis and the first patient to receive it was treated in 1991.
Since then the drug has been owned by 13 different companies, some of whom bought it in order to close down its development, according to Professor Alasdair Coles, a neurologist at the University of Cambridge who led one of the trials.
“Some put a lot of effort into developing it, others were neutral or negative because they owned a competitive drug,” Professor Coles said.
Neurologists have used the drug for the last two decades “off label” – prescribing it on their own initiative even though it was not licensed for multiple sclerosis - in patients with aggressive disease, until earlier this year. Genzyme announced in the summer it was withdrawing the drug for off label use pending the outcome of the trials.
Professor Coles said: “Genzyme have been very supportive and put a lot of money into the trials. But I am disappointed they chose to withdraw it [for off label use]. I don’t see the point. I have personal knowledge of three patients who needed further treatment and we can’t give it to them. They are being given alternative treatments which are not as effective.”
Today’s results show patients on alemtuzumab were nearly half as likely to relapse within two years compared to those on the current most effective treatment, interferon beta 1a. In the second trial of more severely affected patients they were a third less likely to relapse. The drug carries a risk of potentially serious side effects but these can be managed, the researchers said.
The drug is given in two courses a year apart and currently costs around £2,500 a patient but doctors expect the price will rise 15-20 times when it is licensed, based on previous experience. .
Professor Coles said: “Alemtuzumab is the most effective drug that has been seen to date. It is expected to be licensed in the EU in June next year. Everything is in place except the price and that will determine its availability. I wish I could influence it but unfortunately I can’t.”
Professor Alastair Compston, from the University of Cambridge, principal investigator and Chair of the Steering Committee which oversaw the trials, said “Our research shows the transformative effect that alemtuzumab can have for people with MS.”
A spokesperson for Genzyme said the price of alemtuzumab would be established after it was licensed and would be subject to scrutiny by the National Institute for Clinical Excellence (NICE). “We are committed as a company to engaging constructively in this process,” the spokesperson said.