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Unapologetically Going for the Cure

A recent article entitled “A perspective on “cure” for Rett syndrome” authored by a British clinician, Angus Clarke and Ana Paula Abdala Sheikh has sparked differing reactions from Rett families as well as several Rett organizations.

Our partners in the UK, Reverse Rett, wrote an open letter to the authors to address certain inaccuracies and omissions. With permission from Reverse Rett, we’ve included the letter below.

Having pondered the article, the open letter and the comments we’ve received from families I decided this is a good time to share my perspective on a few things.

Firstly, RSRT IS ALL IN regarding the search for a cure.  What we are trying to achieve is remarkably difficult and fraught with challenges every step of the way. There is no room for doubts and paying lip service to a cure doesn’t cut it. What is required is unwavering belief, courage, conviction and tenacity.

We surround ourselves with driven and optimistic people. That includes our trustees, our staff, our volunteers, our families, our donors and last but certainly not least…the scientists and clinicians that we fund.

So what will that cure look like for our children? Will it be reserved for newborns, toddlers and young children?  What can older children, teens and adults expect? Although animal data strongly suggests we have reasons to be optimistic, regardless of age, we don’t yet know how that will translate to people.  The upcoming gene therapy trial will offer the first clues.

Many have asked me whether I think there will be a complete cure for Chelsea, who is 21 years old and severely debilitated.  My answer….No, I don’t.  Do I think she can improve dramatically from a therapeutic that attacks Rett at its core? Yes, I do.  Do I think those improvements will magically happen overnight? Of course not, and neither do any of the parents I know. It is a given that our children will need therapies for a very long time, maybe for the rest of their lives, and that their improvements will be hard fought.

When will that cure arrive? I’ve already written a blog on the subject so rather than repeat myself I’ll simply link to it.   Spoiler alert….the answer is “I don’t know”.

Some have cautioned against using the word “cure” as it’s too loaded.  I disagree.  RSRT will continue to use the word because it is, after all, what we are striving for.   We are not hyping, we are not deceiving families, we are not using the word to raise more money. We use the word unapologetically because we believe a cure is possible.  Cure is the reason we exist …and…. the reason we will one day, hopefully soon, cease to exist.


 

From Reverse Rett – Reverse Rett is a UK medical research charity working to speed treatments and a cure for Rett Syndrome:

  • Lina Pradenas

    First of all we congratulate Monica how she has conducted the association, with her mind and heart, explaining us all researches and how she feels about them.
    It seems to me that Angus Clark and Ana Abdala are only interested in prescribing drugs to merely moderate symptoms without “Correcting” the underlyng problem. It´s not because the “cure” may have inintented harzardous effects, be incomplete or contraindicated for some, that it don´thave to be tried.
    Experiments are need to get a “cure”. It´s expected some fail.
    I believe that each patient with Rett Sindrome have to undergo a variety of detailed exames before undergo the treatment.
    About “Perils of Late Cure”, I think they are not correctly evaluated. There are patients that have no smaller head circunference, as my daugther, and also others symptoms of the Rett Syndrome.One patient is very different from another.
    Late cure is much better than no cure. No family get used to a disease and think that is better do mantain a situation instead of trying a treatment.
    My daughter Viviane, is 33 years old and she is relatively good, although Rett Sindrome.
    I have been dedicating all my life to take care of her. But I fell very worried about her future. We both deserve the opportunity of the “cure”. I hope when I have gone, she may be much better for someone to take care of her.
    I agree with Hellen Keller and I belive in the science progress.
    Finally I trust in “Reverserett.org”.

    Lina and Luis Pradenas – São Paulo, Brazil.