Cancer crowdfunding is something that crops up in my professional world from time to time - where patients choose to raise funds to get experimental treatments generally abroad. I've met a handful of patients who have chosen that path, like in this video article of a young woman's family who sought crowdfunded care in Germany.
To understand my uncertainties about cancer crowdfunding, it's possibly helpful to explain how any drug comes to being available to a patient in the first place. Clinical trials are how we get drugs from concept-in-the-lab to standard of care treatment. We generally divide it up into a number of chunks (image from Cancer Institute NSW, 2018)
Pre-clinical - The lab bit. So whenever I've rambled on about lab research on this blog (got to pick this entry from near the end of my MSc entitled 'This is NOT panicking' - a title which for me, at least, epitomises lab research!) - that's pre-clinical research. One could pick this apart further, but essentially this is where you test things in a lab.
Phase 1 - The 'first in humans' bit. Patients involved in phase 1 trials are, in my opinion, absolute heroes. This is where studies will test new treatments in humans for the first time, gradually increasing the dose from patient to patient to understand more about the drug and its side effects. The main purpose is to work out if it is worth proceeding to phase 2 and at what dose. Often they are given alongside established drugs - for example, chemotherapy plus new drug - so even in a trial setting they are getting previously approved anti-cancer therapy.
Phase 2 - The 'Is it safe and does it work on a small scale' bit. This is where the team says 'Ok - this is safe. Now let's give it to more people at the dose we sussed out in phase 1 and work out how to give it (how often, where, when), what the side effects are like and start to get a sense of whether it works or not'.
Phase 3 - The 'Does it work' bit. These are big studies to find out if the new treatment works. Usually patients are divided into those receiving the current gold standard treatment in a particular situation (or placebo if there is none) and the proposed new treatment regimen. To be a good study, this division should be random, and 'blinded', which means not knowing which treatment you're on. If the new treatment comes out on top, the researchers will argue the case for the new treatment becoming the new standard of care.
Phase 4 - The Afterparty - The drug is 'Out There' - and phase 4 is basically where toxicity and efficacy continue to be evaluated.
Phew. So as you can see - it's difficult. Very few drugs which start out looking promising make it out the other side. It's really, really expensive. At every stage, everyone involved has to remain as unbiased as possible to avoid pushing on with a treatment that ultimately doesn't work. That's harder than it sounds when everyone wants a miracle.
My worry with cancer crowdfunding is that the above steps are bypassed. During the tragic case of Charlie Gard, the people in my lab and I would discuss and debate, as I think many people were, the lengths that are worth going to in search of medical miracles. I know some people round the table felt there should, and could, be no limit. I'm less sure. Perhaps I am inevitably biased by my job and the fact I cannot say I've ever seen a 'miracle cure'. I've seen people promised what I know to be scientifically impossible or illogical, and hope being used to persuade them to proceed in spite of this. Most people that have sought outside-the-box options spend inordinate amounts of money and 'feeling well' time, with no extra time gained. Indeed, some studies demonstrate good early palliative care can actually buy patients the most time. When people say that they think people should do anything to find a treatment, I find it hard to forget the many patients whose care I've been involved with in their final days of life. I wonder what those patients would say. I also wonder what patients and families who want to be allowed to let go feel under the pressure to seek treatment at all costs.
We should be encouraging clinical trials, because it's the only truly rigorous way of working out the best novel and innovative ways of treating people, and often allows patients who are out of standard treatment options access to the most experimental agents out there.
That's why I feel some anxieties about the cancer crowdfunding thing - proceed with caution.
To understand my uncertainties about cancer crowdfunding, it's possibly helpful to explain how any drug comes to being available to a patient in the first place. Clinical trials are how we get drugs from concept-in-the-lab to standard of care treatment. We generally divide it up into a number of chunks (image from Cancer Institute NSW, 2018)
Pre-clinical - The lab bit. So whenever I've rambled on about lab research on this blog (got to pick this entry from near the end of my MSc entitled 'This is NOT panicking' - a title which for me, at least, epitomises lab research!) - that's pre-clinical research. One could pick this apart further, but essentially this is where you test things in a lab.
Phase 1 - The 'first in humans' bit. Patients involved in phase 1 trials are, in my opinion, absolute heroes. This is where studies will test new treatments in humans for the first time, gradually increasing the dose from patient to patient to understand more about the drug and its side effects. The main purpose is to work out if it is worth proceeding to phase 2 and at what dose. Often they are given alongside established drugs - for example, chemotherapy plus new drug - so even in a trial setting they are getting previously approved anti-cancer therapy.
Phase 2 - The 'Is it safe and does it work on a small scale' bit. This is where the team says 'Ok - this is safe. Now let's give it to more people at the dose we sussed out in phase 1 and work out how to give it (how often, where, when), what the side effects are like and start to get a sense of whether it works or not'.
Phase 3 - The 'Does it work' bit. These are big studies to find out if the new treatment works. Usually patients are divided into those receiving the current gold standard treatment in a particular situation (or placebo if there is none) and the proposed new treatment regimen. To be a good study, this division should be random, and 'blinded', which means not knowing which treatment you're on. If the new treatment comes out on top, the researchers will argue the case for the new treatment becoming the new standard of care.
Phase 4 - The Afterparty - The drug is 'Out There' - and phase 4 is basically where toxicity and efficacy continue to be evaluated.
Phew. So as you can see - it's difficult. Very few drugs which start out looking promising make it out the other side. It's really, really expensive. At every stage, everyone involved has to remain as unbiased as possible to avoid pushing on with a treatment that ultimately doesn't work. That's harder than it sounds when everyone wants a miracle.
My worry with cancer crowdfunding is that the above steps are bypassed. During the tragic case of Charlie Gard, the people in my lab and I would discuss and debate, as I think many people were, the lengths that are worth going to in search of medical miracles. I know some people round the table felt there should, and could, be no limit. I'm less sure. Perhaps I am inevitably biased by my job and the fact I cannot say I've ever seen a 'miracle cure'. I've seen people promised what I know to be scientifically impossible or illogical, and hope being used to persuade them to proceed in spite of this. Most people that have sought outside-the-box options spend inordinate amounts of money and 'feeling well' time, with no extra time gained. Indeed, some studies demonstrate good early palliative care can actually buy patients the most time. When people say that they think people should do anything to find a treatment, I find it hard to forget the many patients whose care I've been involved with in their final days of life. I wonder what those patients would say. I also wonder what patients and families who want to be allowed to let go feel under the pressure to seek treatment at all costs.
We should be encouraging clinical trials, because it's the only truly rigorous way of working out the best novel and innovative ways of treating people, and often allows patients who are out of standard treatment options access to the most experimental agents out there.
That's why I feel some anxieties about the cancer crowdfunding thing - proceed with caution.
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