Scientists have long known what causes sickle-cell disease and its devastating effects: a single mutation in one errant gene. But for decades, there has been only modest progress against an inherited condition that mainly afflicts people of African descent.
With advances in gene therapy, that is quickly changing — so much so that scientists have begun to talk of a cure.
In a half-dozen clinical trials planned or underway, researchers are testing strategies for correcting the problem at the genetic level. Already a handful of the enrolled patients, who have endured an illness that causes excruciating bouts of pain, strokes and early death, no longer show signs of the disease.
Among them is Brandon Williams, 21, who lives with his mother in Chicago. Because of his sickle-cell disease, he had suffered four strokes by age 18. The damage makes it hard for him to speak. His older sister died of the disease... (continues)
With advances in gene therapy, that is quickly changing — so much so that scientists have begun to talk of a cure.
In a half-dozen clinical trials planned or underway, researchers are testing strategies for correcting the problem at the genetic level. Already a handful of the enrolled patients, who have endured an illness that causes excruciating bouts of pain, strokes and early death, no longer show signs of the disease.
Among them is Brandon Williams, 21, who lives with his mother in Chicago. Because of his sickle-cell disease, he had suffered four strokes by age 18. The damage makes it hard for him to speak. His older sister died of the disease... (continues)
I hope that this experimental treatment is approved by 2022. It's crazy to think that advancements in sickle cell disease have been barred for financial reasons as well as the skepticism that governments have given gene modifying research. In the chapter Clinical Ethics under the section of regenerative medicine I was shocked to read that governments have been slow to regulate the field thus delaying progression in groundbreaking science.
ReplyDeleteThis article discusses how such advances in gene therapy would be helpful to the many individuals who suffer from Sickle-Cell Disease. It also discusses the cost of treatment and that many impoverished countries have many afflicted by the illness. What is the likelihood that individuals in impoverished countries will receive treatments and how will they offer payments? Would free treatment only be given to the impoverished in the "trial period" where the consequences of the therapy is unknown, only to become available to the wealthy when it is deemed safe for the patient?
ReplyDeleteUnfortunately, I don't think that people from less profitable countries will have ready access to treatment. Just regarding the research, the article explains that an institute director remarked that the studies would bring "significant new money", showing that their motives are still very possibly far-fetched and selfish as opposed to benevolent.
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ReplyDeleteIs Dr. Hoots' motives for the cure of sickle cell anemia pure, or for his own gain?
Once there is a single, established method of treatment, should it be readily accessible for all who need it, or should it be somewhat profiteered?
Is there potential for there to be a single method of treatment, or if all studied options work, will there be an option left up to the patient (i.e., opting out of bone marrow samples)?
I believe healthcare in general should be readily accessible to all who need it as it is our right and obligation to stay in the healthiest condition. I only wish that was possible right now as healthcare is currently orientated like that of a business with the goal of profit.
DeleteWouldn't it be nice if we could figure out how to incentivize the expansion of access to new therapies, drugs? I'd have no objection to profiteering (to an extent) that had such a positive result. The ultimate objective, for that to happen, still has to be the widespread recognition of healthcare as a human right and not a market-driven commodity reserved for wealthy individuals and nations. It is encouraging to see that more young people ("Generation Z"-1995-2010) do think of healthcare as a right, albeit a still largely-unacknowledged one.
ReplyDeleteWhether you are opposed to gene modification or gene therapy or not, it can and should be used in this way. In a way that helps improve people's quality of life and give them happiness when they have had to live with pain and suffering their whole life. I fully hope more research goes into this as it would be an amazing accomplishment.
ReplyDeleteTo add to this, only one half of the genome for the individual would need to be modified. Hemoglobin is a resilient protein, when part of the instructions are intact, a person can be successful.
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