A Future Cure For Sickle Cell?

Recently, an article has gone viral among the sickle cell community regarding a new pioneering treatment which has reversed a teenagers sickle cell disease by changing his DNA. I cannot even count how many posts I have been tagged in and messages I have been sent of this article. If you haven’t seen it, I don’t know where you have been!

Now I am always skeptical of new “cures” and “treatments” when it comes to any health condition in general; because I believe in cause and effect. In my experience, EVERYTHING has a side effect; and sometimes those side effects can be worse than the initial problem you were trying to fix in the first place. For this very reason, I have never been one to go crazy sharing news stories of hope or to voice my views based on 1 or 2 media articles, since I personally do not take everything that is written by the media as fact. So when I was asked by the BBC to do a live radio interview pertaining to this new development in sickle cell, with one of the lead clinicians on the study who was quoted in the article; I was very eager to hear what he had to say first hand.

I knew everyone would have their own opinion on this, and being only 1 of 15000 people living with sickle cell in England; I wanted to be able to give fellow sickle cell warriors and their family / supporters the opportunity to base their opinion on direct information instead of it possibly being distorted by any bias I may have had. With that in mind, I decided it would be a good idea to record the interview so it could be shared for those who were unable to listen live at the time it aired.

The audio is around 16 minutes long, so here are some of the key points mentioned incase you do not have that time on your hands in this moment:

• This is not yet proven as a cure; a lot more time, research and clinical trials are needed before this can be certain.
• There was no mention of infecting the bone marrow with a virus; it is genetically modified using baby (foetal) haemoglobin.
• Since the procedure almost 2 years ago, the teenager in question has had no symptoms of sickle cell and the disease process has stopped; so it does look promising.
• It is an attempt to cure those with full blown sickle cell disease and not an attempt to prevent carriers from passing it on.
• There are currently 7 patients in the US who are being treated with the therapy for sickle cell, however it is too early to comment on the results.
• The same therapy is being used for Beta Thalassaemia and is more advanced with over 20 patients throughout the US, Europe, Australia and Thailand currently in clinical trials.
• At this stage, patients would only be warranted this new gene therapy if there is no compatible donor for a bone marrow transplant.

Aside from feeling self-satisfied at the fact that the lead clinician in the study, professor Philippe Leboulch, complemented me on the way I described sickle cell and the nature of the disorder, I must say I was very impressed with his explanation of the therapy and respected his emphasis on the fact that he did not want to use the word ‘cure’. While the title of this post seems very optimistic (I am, and hope to remain a ‘glass half full’ type of person); I am not here to give false hope. At the same time however, we have to acknowledge the progress that has been made so far, and the fact that a company is actually sponsoring the development of a new gene therapy for sickle cell. I think it is fair to say that we are all hoping for a cure for sickle cell, and this seems like it could be a step in the right direction. The issue now is with them being able to carry out enough trials and research on individuals who are willing to try this new therapy. While there could be some very serious risks involved; of which we do not know the details of, I guess that brave decision would lie with the individual themselves.