One Small Step in the Fight Against Sickle Cell Disease

Dunstan Nicol-Wilson avatar

by Dunstan Nicol-Wilson |

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The treatment Adakveo (crizanlizumab) has been made available in the U.K. as the first new therapy for sickle cell disease in over two decades.

The aim of Adakveo, which will be available to patients 16 and older in England and Wales, is to reduce the number of sickle cell crises experienced by patients. It will be offered to about 300 patients a year with the hope that more will receive it in the years ahead, according to the U.K.’s National Institute for Health and Care Excellence.

When I first heard the news about this treatment for sickle cell disease patients — which was approved in the U.S. two years ago and in the European Union last year — I was happy because we finally had a new breakthrough in sickle cell treatment here in the U.K.

I work as a research manager, so I understand how long the process can be from an idea to the production of a new drug. I appreciated this news as a small step in the right direction.

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Tempered optimism

Word spread quickly after the announcement, and I was soon inundated by friends and strangers sharing the news. Many contacted me to see if I had seen it or to send me “thinking of you” messages.

Some of the comments I saw were frustrating, though, because they made it seem as though Adakveo would solve all of the problems of the sickle cell community. It will not solve all our problems — not by itself, anyway.

Adakveo initially will be available only to a limited number of eligible patients, as there’s more to learn about its long-term effects. Sickle cell is not a one-size-fits-all disease, and one type of treatment won’t necessarily be effective for every patient. Therefore, it’s essential that research into new treatments for sickle cell disease continues. Demand still exists for every sickle cell patient to have access to a form of treatment.

More steps needed

For most of my life, we hadn’t seen a new treatment for this disease. That’s a harsh reality to come to terms with, because it validates the feelings of neglect that we sickle cell patients feel. It often feels as if we’ve been sent a coded message from healthcare services, researchers, doctors, and government officials indicating that our disease — that we — aren’t significant.

Still, I remain hopeful that this narrative will change, and that the work being done to raise awareness and to improve patients’ quality of life gets recognized. We still need more blood donors, as blood transfusions remain a key treatment for many patients.

So, yes, I am very happy that there is a new treatment in the U.K. that will save a few lives in the sickle cell community. But I will not be truly happy, and I will not stop raising awareness, until there are enough treatments for all sickle cell patients. Adakveo is one small step in the right direction, but many more are needed.

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Note: Sickle Cell Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Sickle Cell Disease News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to sickle cell disease.

Comments

Shannon Bentley avatar

Shannon Bentley

My son has sickle cell SS we are in Maryland US. My son tried Adakveo and it gave him severe joint pain which is one of the side affects. My son had two infusions and both times he was hospitalized for pain. Please do your research before jumping out there.

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Nancy avatar

Nancy

Thanks. For your comments because we are in consultation with the doctors for this medication for my son

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