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In sub-Saharan Africa, millions of people suffer from sickle cell disease, and an estimated 50% to 90% of children born with the ailment die before the age of f

In sub-Saharan Africa, millions of people suffer from sickle cell disease, and an estimated 50% to 90% of children born with the ailment die before the age of five. Bill Gates says he has a plan to cure it. Sickle Cell protects against malaria, which is also a horrible disease. The Gates Foundation hopes to do something about Sickle Cell in the next ten years and hopes to eradicate malaria in the next twenty (see below). I hope they decide this needs to be more of a coordinated effort. The Bill & Melinda Gates Foundation has committed nearly $2 billion to antimalaria efforts, and Bill Gates has said he believes the disease can be wiped out by 2040. Malaria today is transmitted in ninety-five countries, accounting for nearly half of the world’s population. Eighty-nine percent of all reported cases are in sub-Saharan Africa https://www.cfr.org/backgrounder/can-malaria-be-eradicated https://www.cfr.org/backgrounder/can-malaria-be-eradicated

HarryHirsch 6y ago
Last year, a Hb polymerization inhibitor came to market: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-voxelotor-sickle-cell-disease https://www.fda.gov/drugs/resources-information-approved-dru... It's a remarkable breakthrough.
- DoreenMichele 6y ago
  Thank you. I searched for an explanation of what it does in layman's terms and found this: None of the currently approved drugs can modify the underlying pathology of the disease. Voxelotor, first of its kind, is an orally administered drug that can alter the underlying disease pathology (by increasing the affinity between Hb and oxygen) and inhibit sickling of red blood cells. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7100618/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7100618/
waterhouse 6y ago
> Sickle Cell protects against malaria I knew being heterozygotic for sickle cell helped. Does having two copies of the gene have a net benefit? ... Wiki seems to say no: "The protective effect of sickle cell trait does not apply to people with sickle cell disease; in fact, they are more vulnerable to malaria, since the most common cause of painful crises in malarial countries is infection with malaria." https://en.wikipedia.org/wiki/Sickle_cell_disease#Malaria_prevention https://en.wikipedia.org/wiki/Sickle_cell_disease#Malaria_pr...
- DoreenMichele 6y ago
  Yes, Sickle Cell trait exists in that area because it is protective. Sickle Cell Disease occurs when someone has two copies of the gene and having two copies goes bad places. Maybe I am missing something and the plan here is to leave Sickle Cell trait alone and only subject people with Sickle Cell Disease to what amounts to genetic experimentation. I find that ethically questionable, but perhaps there are people who think that makes sense. To me, fixing this means getting rid of the mutation. The mutation exists because it is protective. If you remove that protection without first removing the reason it exists, I find that also morally questionable. First, eradicate malaria from the region. Then eradicate the trait. You could reduce incidence of the disease dramatically without coming up with Frankensteinian medical experiments by testing everyone for the trait as a condition of getting a marriage license and trying to get carriers to stop breeding with other carriers. It would be a social intervention, not a medical intervention, and it's completely humane. Of course it wouldn't make headlines as being ooh, shiny new tech and some people would no doubt have a cow about it for some reason or other, like "What right do you have to tell me whom I can love?" But as someone with a different homozygous recessive disorder (who also has a child with the same thing), that was more or less my policy after I was diagnosed and was getting a divorce. I didn't wish to risk getting pregnant by a carrier again, so I did what I could to protect myself against that scenario.
  - ampdepolymerase 6y ago
    You are correct in understanding that the Gates foundation should clarify their timelines. I am not sure why you have been downvoted for this. The economic and health impacts of malaria versus sickle cell diseases ought to be decided by the individual. If the person has no plans to immigrate and if his or her country is stuck in a perpetual resource trap with no ability to combat malaria, then the cure (eradicating sickle cell anemia) may be more harmful than beneficial.
    - DoreenMichele 6y ago
      Most people haven't really thought about the problem space in a comprehensive fashion and don't see the interconnections. The modern view tends to be "What pill/medical treatment fixes X disease" and looking at it in a larger context of how that disease even occurs to begin with flies in the face of those mental models. Throw in words like ethically questionable and some folks will probably hit the downvote button in knee jerk response to perceived "SJW" stuff without trying to understand the point of the comment.

HarryHirsch 6y ago

Last year, a Hb polymerization inhibitor came to market: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-voxelotor-sickle-cell-disease https://www.fda.gov/drugs/resources-information-approved-dru... It's a remarkable breakthrough.

DoreenMichele 6y ago
Thank you. I searched for an explanation of what it does in layman's terms and found this: None of the currently approved drugs can modify the underlying pathology of the disease. Voxelotor, first of its kind, is an orally administered drug that can alter the underlying disease pathology (by increasing the affinity between Hb and oxygen) and inhibit sickling of red blood cells. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7100618/ https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7100618/

waterhouse 6y ago

> Sickle Cell protects against malaria I knew being heterozygotic for sickle cell helped. Does having two copies of the gene have a net benefit? ... Wiki seems to say no: "The protective effect of sickle cell trait does not apply to people with sickle cell disease; in fact, they are more vulnerable to malaria, since the most common cause of painful crises in malarial countries is infection with malaria." https://en.wikipedia.org/wiki/Sickle_cell_disease#Malaria_prevention https://en.wikipedia.org/wiki/Sickle_cell_disease#Malaria_pr...

DoreenMichele 6y ago
Yes, Sickle Cell trait exists in that area because it is protective. Sickle Cell Disease occurs when someone has two copies of the gene and having two copies goes bad places. Maybe I am missing something and the plan here is to leave Sickle Cell trait alone and only subject people with Sickle Cell Disease to what amounts to genetic experimentation. I find that ethically questionable, but perhaps there are people who think that makes sense. To me, fixing this means getting rid of the mutation. The mutation exists because it is protective. If you remove that protection without first removing the reason it exists, I find that also morally questionable. First, eradicate malaria from the region. Then eradicate the trait. You could reduce incidence of the disease dramatically without coming up with Frankensteinian medical experiments by testing everyone for the trait as a condition of getting a marriage license and trying to get carriers to stop breeding with other carriers. It would be a social intervention, not a medical intervention, and it's completely humane. Of course it wouldn't make headlines as being ooh, shiny new tech and some people would no doubt have a cow about it for some reason or other, like "What right do you have to tell me whom I can love?" But as someone with a different homozygous recessive disorder (who also has a child with the same thing), that was more or less my policy after I was diagnosed and was getting a divorce. I didn't wish to risk getting pregnant by a carrier again, so I did what I could to protect myself against that scenario.
- ampdepolymerase 6y ago
  You are correct in understanding that the Gates foundation should clarify their timelines. I am not sure why you have been downvoted for this. The economic and health impacts of malaria versus sickle cell diseases ought to be decided by the individual. If the person has no plans to immigrate and if his or her country is stuck in a perpetual resource trap with no ability to combat malaria, then the cure (eradicating sickle cell anemia) may be more harmful than beneficial.
  - DoreenMichele 6y ago
    Most people haven't really thought about the problem space in a comprehensive fashion and don't see the interconnections. The modern view tends to be "What pill/medical treatment fixes X disease" and looking at it in a larger context of how that disease even occurs to begin with flies in the face of those mental models. Throw in words like ethically questionable and some folks will probably hit the downvote button in knee jerk response to perceived "SJW" stuff without trying to understand the point of the comment.