Lutate provides a breakthrough in treating rare cancer but treatment is struggling for finance Hope: Erin Potter received treatment for her tumour that extended her life expectancy. It is a dark shadow, eating away at your insides. It starts from nothing, really - well nothing anyone can explain. A hot flush, tummy upsets, asthma symptoms. In and out of doctors' surgeries for years. Some suggest it is all in your head. And then, when the answer finally comes, it is the words we fear most: "You have cancer. We didn't pick it up early enough ... it's spread ... you have months, a couple of years if you're lucky. This is the reality for the majority of Australians diagnosed with neuroendocrine tumours, the cancer that killed Apple founder Steve Jobs. Unusual case: Steve Jobs. But an increasing body of evidence is showing that for many of those for whom surgery, chemotherapy and hormone treatment has failed, a new treatment, Lutate, provides hope. Sydney mother Erin Potter knows just how much hope. When her little boy started kindergarten she learnt she had a tumour in her pancreas. By the time he was in year 2, they told her she had between one and three years to live. But this week, more than five years later and after treatment with Lutate, she watched him finish primary school. "Then you get selfish and you think, 'I want more, I want to see him get married,"' she says. Like many people with rare cancers, neuroendocrine patients face huge hurdles in a system set up to develop, assess and finance treatments for the masses. John Leyden's sister was in her early 30s when she was told she had neuroendocrine tumours that had spread to her liver and bone, and had six months to live. After Lutate, she lived for four years. Leyden, an anaesthetist, has since dedicated his life to the cancers through his charity, the Unicorn Foundation, and has spent the past seven years trying to get steady funding for Lutate, from state and federal health departments. Lutate is unusual. It does not even really fit our definitions of what a "drug" is: it is known as a "radiopharmaceutical." Radioactive particles hitch a ride into the tumour, and as Leyden puts it, "nuke them from the inside out." Rare Cancers Australia's Richard Vines says rare cancers are ignored by industry and governments. He says when two people, one with a rare cancer and the other a common one, are recommended the same treatment by their doctor, often the person with the rare cancer will have to pay from their own pocket because it has only been "proven" for treating the common cancer. "If people really understood how the system works, they wouldn't accept it," he says, adding federal programs such as the Lifesaving Drugs Program have never been used for a cancer drug. It is little wonder doctors and patients found it virtually impossible to get Lutate funded. But they had another option. The Agency for Clinical Innovation was set up to ensure promising treatments favoured by doctors could be adopted in NSW. The 2008 Special Commission of Inquiry into the health system - the Garling Reforms - recommended the agency be given the highest level powers to make this happen. John Dwyer is a doctor who helped develop the idea of doctor-driven reform in NSW. He finds it concerning that its expert group could spend years on its plan, only to have it rejected. "It's the process that worries me," he says. No one is interested in these patients as much as the clinicians treating them, and that's one of the reasons Garling recommended this group be set up. One of those clinicians is Rod Hicks. It is not often cancer doctors are willing to use terms such as "miracle," but that is how Hicks describes Lutate's effects on some. "We see patients when often they have been given no hope by their medical oncologist," he says. But they are getting back up, paying taxes and going back to work. Hicks says the need is not for large randomised controlled trials, but going smaller, more fine-grained: finding out what differentiates tumours that respond from those that do not, and what time in the disease progression is best to start it. Our drug regulation system was set up to prevent widespread prescribing disasters, such as the thalidomide tragedy that saw thousands of children born without limbs because their mothers took a morning sickness drug they did not know would cause birth defects. But rapid developments in research are beginning to overtake it. Ever-expanding genomic information creates advances of which we have only dared to dream, but at the same time more subgroups of what are, essentially, rare cancers that need targeted treatment. "What the government needs to do with rare diseases is create a new paradigm," the chairman of the Medical Oncology Group of Australia, Gary Richardson, says. It's impossible to use the standard paradigm of approval for any of these agents because recruiting enough patients into a trial to show clinically meaningful benefit is becoming a more significant problem. Associate professor Richardson says cancer doctors have become increasingly concerned that Australia is lagging behind when it comes to new cancer drugs. "The system overall has become ... probably too rigid," he says. He sees a future on "adaptive licensing" - allowing drugs onto the market earlier than usual, but then carefully monitoring every patient who takes them. If the drug does not perform as it did in the early trials, it can be removed, or the price paid for it cut. But there is one serious problem that would remain: cost. When someone's life is at risk, doctors and patients often wish to try products that are, simply, potentially over-priced. "If you wanted to buy a house worth $100,000 but someone offered to sell it to you for $10 million, you wouldn't buy it," Richardson says. "These drugs are massively expensive, and to some degree you do sit there and go, 'Would I pay $120,000 for an extra two months?"' And Australia has very little power do fix this problem. "We are only 1 per cent of the drug market," he says. What we really need is the US to say 'these drugs are too expensive', and as soon as they do that the price will come down. Richardson says federal regulators have made some promising moves, and he is hopeful we will see change. But for the patients stuck waiting for treatments, that change cannot come soon enough.