New Sickle Cell Treatment Gets Thumbs Up in the UK

Big news on the horizon for all my fellow warriors dealing with sickle cell disease, as the news broke this week of a new sickle cell treatment called CASGEVY™. Let’s dive in together and unpack what this could mean for us!

From Pain to Promise

Living with sickle cell is like riding a rollercoaster of pain, and we’re always looking for something to make the ride smoother. CASGEVY aka Exa-cel, this new sickle cell treatment, just might be that something! It’s got the official thumbs-up in the UK for patients 12 and up who:

1. Live with sickle cell disease (SCD) and have recurrent vaso-occlusive crises (VOCs).

2. Are transfusion-dependent living with beta thalassemia (TDT) and can’t get a matched donor for a stem cell transplant.

The Nitty Gritty Of This New Sickle Cell Treatment:

Ok so, Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics have announced the ‘conditional‘ marketing authorization of CASGEVY™ in the UK. This will be the first regulatory approval for a CRISPR-based gene-editing therapy in the world. It is currently under review by the European Medicines Agency, the Saudi Food and Drug Authority, and the U.S. Food and Drug Administration.

For the UK, Vertex said it will potentially open the treatment up to 2,000 SCD patients. However, due to its steep price (estimated over £1 million) there’s no saying when or if it will ever be commercialised in England?

Can We Call It A Cure?

When news like this breaks, the world media love to throw the ‘C’ word around, which makes me want to throw a different one right back to them!

I would not call this a cure (yet), but clinical trials show some real promise. In trials, 28 out of 29 sickle cell patients were free of severe pain and 39 of 42 beta thalassemia patients no longer needed blood transfusions for at least a year:

“I said yes without hesitation, knowing that I would be the first person but this was my opportunity to fight… I now work full time and I contribute to my household and my community.”

Victoria Gray, a 38-year-old woman who was the first patient to be treated with exa-cel.

“Gene therapy has given me the ability to take full control of my life… In a world where the deck was stacked against me, gene therapy has been a winning hand. While I recognize gene editing won’t be the solution for everyone I strongly recommend [sickle cell] warriors to consider this one-time therapy.”

Jimi Olaghere, who participated in the exa-cel study about three years ago.

Are There Any Question Marks ??

One thing living with sickle cell has taught me is the sometimes un-even balance of treatment vs side-effect. So, I did a deep dive of the published FDA meeting discussion as well as the official UK press release and here are some of the questions raised about this new treatment for sickle cell:

Off-Target Edits: Concerns have been raised about the potential for off-target edits, where CRISPR makes unintended cuts to DNA other than the intended target. This could disrupt gene expression and have unintended consequences.

Long-Term Safety: While short-term safety seems acceptable, long-term safety remains a concern. Vertex has proposed 15-year safety studies to track the long-term safety of the treatment and aim to address uncertainties about potential delayed effects or complications that may arise over time.

A Warriors Hopeful Heart

As someone sailing the ship for sickle cell, news like this fills me with hope. It’s not just about me; it’s about all of us living this journey. Wouldn’t it be great if this new sickle cell treatment could be the wind in our sails, steering us toward a painless new way of life?

For those of us used to battling the pain, poor care and unpredictability of sickle cell, I definitely think this new treatment could be a game-changer. The only real question is, will this new treatment be able to permanently change the constant struggles of SCD?

While I wont be throwing a “Cure Party” just yet, this new treatment for sickle cell getting approval is like a mini celebration. It’s progress in our fight against SCD, and you all know I love to celebrate progress. 🌟