Thursday, 11 August 2011
Leukemia, MS and promises of miraculous treatments - who gets hurt?
Two headline stories overnight speak to a key issues in health services.
A 2nd Canadian dies after vein surgery for multiple sclerosis.
Gene therapy for leukemia treatment successful.
Multiple sclerosis is a devasting disease on individuals and their families. Despite years of research, minimal progress has been made on effectively altering the course of the disease.
Leukemia strikes fear into most of our hearts. Someone we have known has likely suffered from the disease and we are all too familiar with the tragic consequences for some. Unlike multiple sclerosis tremendous gains have been made in treatment options and survival rates over the past 4-5 decades. While incidence of childhood leukemia stable, 90% survival rates and improvements in mortality over the past 30 years from 50% mortality. However one needs to read the fine print of the article, the treatment is only for persons with chronic lymphocytic leukemia (CLL).
There are four major classes of leukemia, CLL is almost exclusive an illness afflicting aging person and rarely seen before age 40. It had a 75% 5-year survival rate, with as many afflicting succumbing to other disease of age as to the disease itself. It afflicts about 1800 Canadians a year. About 50% more common in males than females, it has been slightly increasing over the past couple of decades with rates up about 50%. Unlike most leukemias – there are the majority of suffers have genetic anomalies.
The treatment being utilized is exciting from a technical perspective and no doubt likely something of considerable expense. Its application to other forms of leukemia or cancers will be limited, although the technology perhaps will form the basis for treatment of other genetic anomaly disorders. The cost of developing the techniques will be millions of dollars to help a very small subset of sufferers. The good news, is that the procedure is following the accepted process for scientific scrutiny. The researchers do not attempt to overstate the value, it is the media spin that may elevate some people’s hopes.
The second story is an example of a therapy that has not received scientific scrutiny and yet public expectation have been raised. As with any new therapy, there is hope that the final result will demonstrate a significant benefit and judgement on its effectiveness should be reserved until it has undergone the level of rigorous testing required to ensure it works, and ensure it is safe. That an increasing number of private facilities offer the service on a user pay basis reflects the challenge of living with MS. There are about 85000 Canadians living with MS. Canada is well known for its high incidence of MS – one of the highest in the world. As such, the potential for a dramatic breakthrough in treatment would be very welcomed.
The big BUT, the vein cleaning surgery for chronic cerebro-spinal venous insufficiency (CCSVI) has not undergone the types of testing that medical procedures would normally be subjected to. Its origins in an Italian clinic in 2008 that has not undertaken formal research on the procedure has incited controversy across the world. Governments are pouring dollars into researching the procedure and to date the findings are not nearly as promising as the hype. In the meantime, hundreds of Canadians have traveled out of country for the procedure and disease specific societies have advocated for inclusion of the procedure as funded as well as research into its value. Objectivity in the debate is easily lost. Millions upon millions of dollars will be spent to ultimately determine the value of the procedure. As a rule of thumb it costs twice as much to prove something doesn’t work as it does to prove it does. The onus has now been placed on the public to fund this work.
True “breakthrough” therapies in medicine are rare. Breakthroughs are large improvements in outcomes over existing therapy. Most improvements occur through incremental improvements over existing therapy. Research is expensive, and the “system” is unlikely to fund research into unproven therapies that might lead to breakthroughs – most likely because they are so rare and uncommon. However, there are innumerable vulnerable populations ripe to respond to overzealous claims of benefit, and ready to dig deep into their personal pockets to buy hope.
The damage done by excessively raising public expectations, by siphoning research dollars away from activities that will benefit larger numbers or persons, and the personal toll taken by individual pocketbooks or through lives of those that are unwittingly being subjected to unproven therapies is discouraging in the least. My sympathies to those caught in the middle between the need for hope and the desire for something that works.
If it sounds too good to be true – it probably isn’t.