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| 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
