Some breathtaking things are happening in the world of sickle cell anemia (SCA) that could potentially impact the lives of nearly 275,000 babies around the world and nearly 100,000 African Americans in the USA.

A children’s specialty hospital in Paris, France is leading the way (an expensive one) towards a cure by using stem cell transplants. You can read more about it here.

In fact, this hospital has already made the claim that they have cured SCA. The procedure is really rough and is also not without risks, something that should be taken into serious consideration. But the results are quite promising—at least in the short-term results, benefits. The stem cell transplant therapy is consistently showing no evidence of disease—at least by follow-up markers, that is.

Will the benefits of this risky treatment continue to work for these patients after two years? 10 years? Or is it more of a beneficial treatment with only short-term guarantees? And even so, WHEN will an effective treatment be made for adults that doesn’t have a significant amount of risks and or side effects involved?

SCA Background and Challenges:

These are alarming statistics for a common blood disorder, which is genetically passed on from one generation to the next—the children have to inherit one mutated gene from each parent in order to have SCA.

The challenge with SCA is that it’s debilitating and painful. Because the blood cells are mutated into a sickle shape vs. a round shape, hemoglobin cannot circulate throughout the body properly. Their hemoglobin becomes abnormal and as a result, the blood stops up, like a clogged drain. Except in this case, minute blood vessels get restricted from lifesaving blood.

As a result, people experience extreme pain. Although it can vary from person to person, because everyone’s sickle cell anemia is different.

One of the baffling things about this condition is that, with so much variety in how it will affect someone, there is not standard protocol for how to treat it. So sometimes, doctors are left guessing on how to prescribe medicines to relieve discomfort of their patients. I’m sure they mean well…

I think it’s very exciting that clinical evidence is showing promise for SCA. I just continue to be concerned about the cost, making it affordable for people no matter how wealthy they are, or the color of their skin.

What’s your opinion on what these doctors and scientists are doing in Paris? Please post on Patient Worthy. Remember: You are not alone. Your voice matters.