Every day a new and mind-boggling advance in medical science appears, with researchers manipulating genes and stem cells to encourage the human body to heal itself.
Investors in biotech companies may be frustrated that few of the ground-breaking discoveries have led to any financial return on the billions of pounds poured into research, but spare a thought for patients with life-threatening diseases.
Their hopes are continually raised when a new treatment is flagged up, but then dashed when it emerges that it won't be available for several years.
So it was good news this week when Genzyme, a US company which has its UK headquarters at Oxford Science Park, won approval from European regulators of a new drug for patients with two blood cancers, non-Hodgkin’s lymphoma and multiple myeloma. The drug, Mozobil, can be injected into patients who need transplants of blood-forming stem cells so that they can withstand the fierce chemotherapy treatment which will cure their cancer. The stem cells are taken out of the patient's blood stream, and then replaced once the cancer has been killed.
Dr Chris Hatton, lead consultant for lymphoma at Oxford Radcliffe Hospitals Trust, said: “This product enables stem cell mobilisation. With lymphoma, one in five patients will not mobilise, so previously we could not give them stem-cell transplants.”
He said Mozobil was different from other expensive cancer drugs that may only prolong life for a few months. “For patients, it is curative therapy, and Mozobil has been successful in the small number of patients that we have used it for,” he said.
For the past year or so, Oxford has been one of several centres in Europe to be given free ‘compassionate use’ of Mozobil by Genzyme, enabling life-saving transplants on an estimated 250 people nationwide.
But now that it is on the market, Genzyme is hoping to recoup some of the millions of dollars it has spent on research, in particular the £353m cost of buying a Canadian biotech called AnorMed, which developed the drug. The Oxford hospitals, like everyone else, will now have to pay £4,000 per dose for Mozobil.
Dr Hatton said: “The issue for patients in the Oxford area is that as yet we have no statement from anyone about how to fund the drug.”
The National Institute for Health and Clinical Excellence (Nice) which has been the ‘baddie’ in many stories about patients being denied expensive cancer drugs by the NHS, will not rule on the issue, since the number of patients involved is so small.
However, Rory Cameron, Genzyme’s director of oncology, believes that the funding issue will be solved. “We will be talking to various centres about funding, probably until next April. My limited understanding is that a tariff has already been set for the treatment and you now have a new drug.
“Overall, the cost of failure would be larger than the cost of Mozobil — you could save the cost of having a patient on a machine for two days.”
At this week’s launch, cancer doctors were excited about two other potential uses for Mozobil — to transplant another person’s stem cells into patients, and to treat leukaemia by coaxing cancer cells from bone marrow into the blood, so that they can be treated.
An even wider use for the drug was recently highlighted by New Scientist magazine in an article entitled “Healing power of DIY stem cells unleashed”. Sarah Rankin’s team at Imperial College, London, used Mozobil to boost production of stem cells in mice, opening up the possibility of harnessing people’s stem cells to help them heal their own bodies, repairing blood, muscle, broken bone, ligaments, or damaged cardiac tissue following heart attacks.
Genzyme’s Steve Bates takes a cautious view of this. “We think this is an exciting hypothesis, but at the moment what it is useful for is getting stem cells out of the bone marrow into the blood supply.”
Mr Cameron agreed, saying: “We have not launched Mozobil for any of these uses and the Imperial College research is even further into the future.”
He defended Genzyme’s pricing policy, saying the company anticipated UK sales of £1m next year in return for its huge investment. “It’s a one-off treatment and the cost is relatively cheap. It can get you a transplant which can save your life. The key for us is that it’s relatively rare to see product launches and it’s nice for us to have provided compassionate use for so many patients successfully.
“There are so many drugs that never get there. To see a product that makes such a difference to people is amazing. The big impact is on those patients who would have been told that they couldn’t have a transplant, which was their only chance of survival. That must be devastating.”
So perhaps we can take heart that dazzling advances do sometimes result in new treatments. And as for the promise of ‘DIY healing’, Dr Rankin said: “Mozobil is already out there on the market, so it’s feasible that trials could happen in the next five to 10 years.”
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