Alex starts another Clinical Trial…

It’s been a while since I last wrote an update on how things are going, so thought I would update you all on what’s happening and why we’ve been busy little bees lately. The news about Alex’s Trial, his 13th Birthday and how he is getting on.

After the news that Alex’s trial stopped last year, as the drug wasn’t working, we’ve been in regular contact with the amazing doctors and nurses up in Newcastle to see if there is anything Alex might be able to take part in. And the good news is Alex has been visiting Newcastle a lot recently for many tests to see if we could get him onto a trial they’ve been running for a little while.

This trial is to test a therapeutic drug called Givinostat which has the potential to be an anti-inflammatory, antifibrotic with pro-regenerative effects – which essentially means that it could have the capability of stabilising boys with Duchenne – for a while at least.  Alex was accepted and he started the new drug a few weeks ago!  He’ll be on the trial for 19 months – but we have no way of knowing whether he’s taking the drug or on the placebo (sugar pill) but we’re prepared to give it a go and see what happens. When you have no treatments available, you will try anything – believe me having a child with Duchenne is complex, they have extra needs and we need to be vigilant and try whatever we can to help stabilise his condition and stop his muscles from wasting away as much as we possibly can.

Whilst Alex looks okay from the outside, inside his muscles are mainly made up of fatty scar tissue, and any inflammatory should help flush out the toxins and keep what muscles he does still have left as healthy as possible. If you would like to read more about this trial, you can here: https://dmdhub.org/trials/givinostat/

A few weeks back, Alex reached his 13^th birthday, it was so lovely to see him out and about on his own with his friends having loads of fun. He’s still ambulant, meaning he’s still able to walk – which is a blessing – we never thought Alex would still be walking into his teenage years. We really do put this down to the proactive management of his condition (as well as the fact that he might have a slightly less aggressive strain – but that we are not sure about) and doing everything we possibly can for him. His medical team at Newcastle and Leicester have been amazing and we have them to thank them for all their support and advice.

We hope he continues walking for some time yet (keeping our fingers and toes crossed), we’re planning on a trip to Alton Towers next week and Alex is so excited, he loves nothing more than crazy thrill-seeking rides. The one good thing about having this disability (his words not mine) is that he gets a disability badge so he doesn’t have to queue at the rides – we’ll take any plus points!  But, we know that so many families out there living with Duchenne are not so lucky. Duchenne is a devastating condition, and often boys cannot do the things they love to do from such an early age – because their muscle weakness stops them. Often in wheelchairs before they even reach 10 years old – and watch their friends from the side lines. This breaks my heart. It’s such an unfair and cruel disease – all we ever want for our children is for them to be happy and to do what they love doing.

All the money we raise is for them, we hope it’s going to benefit Alex in the long-run and are keeping optimistic – but we do what we do because we care about every single child and young adult affected and that keeps us going.

We’ve had a few ups and downs lately with Alex, he went through a time where he was falling over lots, because he has muscle weakness in his hips, he walks differently to other boys with a very pronounced arched back, which causes discomfort when walking, especially up and down hills.  As a result Alex often has to take breaks and this can also stop him from being as active as he wants. Sometimes his knee will go inwards causing him to fall without any warning. Now he’s getting a bit older he’s very conscious of it and doesn’t want to bring attention to himself. If I help, he just shouts and says “leave me I’m fine, let me get up on my own”. I totally understand, I know whenever I’ve fallen over in public it’s embarrassing and everyone rushes over to check you are okay.  Alex knows this is something he must live with and is such a fighter – he certainly won’t let the falls stop him – not for now anyway. He’s just had a scan of his back, something we need to do now every year to check for signs of scoliosis (arching of the back) and for tiny fractures which can go unmissed – these are often caused by the falls. Fingers crossed he’s all okay.

Alex has started using walking sticks when we go out for family walks, and they help massively and give him the security of having something to hold onto to help lessen the amount of times he falls over.

Both Alex and I did the Zipwire in Wales recently to raise money for Alex’s Wish, it was such a brilliant experience – we loved it and was so appreciative to see so many of our supporters join us. Alex is planning on doing some more experiences to help raise vital funds as he loves to join in and help out where he can.

We continue working incredibly hard to raise much-needed funds. The past two months has been hectic, but we are raising money and its making a huge amount of difference.  All the money we raise goes directly to where it needs to go to help bring new treatments that will help delay the devastating muscle wasting, as well as invest in a potential cure.

Thanks again to all our amazing and wonderful supporters, we cannot do what we do without you. Thank you for staying with us, this is a long-term fight – but a fight we are prepared to take.  Being charity partners to Duchenne UK means we are getting there sooner; we know that we cannot fight this on our own.  We know that the time is now for Duchenne and we are excited about the future.

Sending you lots of love and hope the summer months brings us all lots of sunshine.

Take care all,

Em (Alex’s mum) X

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