Is This The Cure For Sickle Cell Disease?

Sickled Cells in a patient with Sickle Cell Disease

It’s all over the place. Everywhere you turn there is this buzz. Relief, one is happy to say, from the relentless reports of wars, conflicts and disgraceful tweets. The thing that is causing the buzz is the story about a new cure for sickle cell, a disease that affects hundreds of thousands, if not millions throughout the world.

There is nothing unusual about new cures. Breakthroughs for this or that are regularly announced, both in the regular media and in fringe outlets. And we humans seriously take them on board together with their hope and their hype, especially for diseases that stubbornly resist the best efforts of our medicine men and women, scientific or shamanistic. The cure for cancer, especially (as if cancer is just one disease, like appendicitis), is one that regularly grabs the imagination. But one must always be cognizant of the fact that however relevant these “cures” might be, their impacts are mostly incremental and, in any case, require time to mature into full-fledged remedies.

The cure that was announced last week must, as in other similar examples, be the subject of a reality check. Nevertheless, this is one announcement that should be taken very seriously because the procedure directly targets the engine that drives the terrible clinical consequences of the disease. In the report, French researchers describe how they harvested blood stem cells from a 13-year- old boy suffering from the disease and inserted normal haemoglobin genes into them. They then returned the modified stem cells back into the boy, who subsequently began to make normal red blood cells. Fifteen months later he is free of all the symptoms of the disease. Pretty good, when you consider that for most of his young life he had suffered severe symptoms. Everybody is now talking excitedly about how this treatment can be rolled out to all those hundreds of thousands of patients throughout the world suffering from sickle cell.

Conceptually, the procedure is simple enough. But in practical terms, it could be devilishly complicated as demonstrated by the huge number of researchers that pulled off the treatment. Indeed, when I looked at the report, I had to give up counting the named individuals and institutions that collaborated in the work. I can say, though, that their number, combined, might well exceed the entire scientific and medical capital in some countries where sickle cell is prevalent.

Another point: This is just one case. That of a13-year-old located in a well-resourced setting and is a long way from signalling that the treatment can be extended everywhere, even to other rich countries, never mind into the swamp-like conditions that prevail in poor ones. To show that the treatment can be applied elsewhere calls for a clinical trial that would involve many patients preferably from more than one big institution. Undertaking such would require time and money. We’re probably looking at, may be, five years, if not longer, not to mention the necessary amount of cash.

In the mean time we do have other measures which, though not curative provide much relief for patients. These are the things we should focus on at present. But even here, there is an urgent need for peoples and governments to step up and do their duty.

Tell Fren Tru