Source: University of Chicago Medicine
I was excited to read an article today about a possible cure for Sickle Cell Anemia. Now my son is heterozygous for Sickle Cell so mild to no problems from it. However, I have a friend who does have sickle cell anemia and it can be life threatening.
For those of you who don't know, red blood cells should be a nice round shape so they go nicely through blood vessels, arteries and veins. However, some people have a genetic abnormality which turns nice round blood cells into crescent shaped blood cells. With a crescent shape they get stuck, cause clots and can lead to severe pain, clots, strokes and a short life span.
There is one small benefit of having sickle cell anemia. Those people with sickle cell anemia are less likely to get malaria. That small benefit means that the majority of people who have this problem are in Africa (yes other countries have it too but I'm showing a point).
Now sickle cell anemia hasn't had a cure just a treatment that while not great was better than nothing. Folic acid to help with anemia and chemo drugs like hydroxyurea to help promote healthy blood production. However, even with these medications there will be painful bouts of clots and there will be problems--just fewer--we hope.
I was reading an article about a new treatment which can reverse the problems for years or longer. It is a gene therapy where the bad blood and bad marrow is taken out, it is generically altered and then replaced. That's the very simple idea behind a long, complicated treatment. It requires many months of very sophisticated treatment but for those lucky few to get it their lives can be changed.
Would you consider that a medical breakthrough?
But is it really a breakthrough?
My first thought was, awesome, a possible treatment for those with sickle cell anemia. Then I kept reading some more.
Those people who need it the most are those people who are least able to afford it. I read about people in Tanzania who can't even afford the $1 HBD/month for Folic acid let along the more expensive Hydroxyurea. Even those in Africa with a little better means may find the current treatment and transfusions expensive.
Then I read a little further. I was curious. How expensive is this new treatment?
Two to Three MILLION USD
I shook my head at this cruel joke.
Millions of people suffer from the disease. Scientists come up with something that can treat the disease but... it will leave millions of people still suffering from the disease. It's theoretically possible to treat but in reality nothing will change. Where would you find the CRISPR technology, the cryogenic freezers and specialist medical equipment in a place where people live on less than Americans pay for a nice dinner?
I'm torn on this advancement
While I was initially disgusted at the situation I've been doing some thinking. If scientists don't do the research then things can't change in the future. Right now its important to note that advancement has been made. It is just theoretical right now as the price means its not available for pretty much anyone except the super rich. However, time and practice tend to make things cheaper.
I remember marvelling at the first computers that were being sold in the Radio Shack catalogue (where I first worked). Computers back in the last 70s / early 80s were over $10,000 (which would be roughly $40K in today's dollars). However, prices have come down with sophistication and experience. I hope that is the same for treatments like this gene therapy advancement.
So what is the value of this research
What does this medical advancement give to people suffering from sickle cell anemia? Well, maybe a little bit of hope. Hope that in the future prices will come down and their suffering can end (in a good way). It is small comfort for those suffering now but sometimes a little light and a little hope can make just enough difference to keep someone soldiering on.
Just my thoughts for the day.
Thanks for reading.