Friday, 14 November 2014

Please help Sam Brown, Harry's classmate and friend.

Please click on the link above and sign this petitician and share with your friends to sign too.

I do not normally do this, but Sam's situation is different to many asking the NHS to fund drugs.

Why? Because Sam has been on a trial for 2.5 years and so the benefits to him have have been measured and quantified. The drug has now been licensed, which is testament that it is beneficial to children with Morquio Syndrome. The drug has to go to a panel for funding to be agreed for Sam to continue having the drug. The petition is about getting the score card system changed with regards how new drugs are scored in relation to those affected by rare diseases, child or adult. With the current scoring system it seems unlikely that drugs for those with rare diseases will receive funding. Signing the petition raises the profile of this situation and hopefully will reverse the scoring system, so all diseases needing new drugs to limit the effects, will get a fare crack at being funded. 

Sam is age 6. He has a rare life limiting disease called Morquio Syndrome, which is a type of MPS - a group of ultra-rare genetically inherited diseases that affect children's development in different ways. Neither Katy nor Simon knew they were carriers of the gene that causes the disease, until they started having tests to get to the bottom of Sam's physical development delays, when Sam was a toddler. 

There are only 105 sufferers in the UK. the disease causes progressive physical disability, shortness of stature and significant health problems in adult life often leading to a significant shortening of life expectancy and reduction in quality of life. Sam has been receiving the drug Vimizim (elosulfasealfa) via a clinical trial of treatment at Royal Manchester Children’s Hospital for two and a half years. The drug received licensing approval in the EU in April 2014 – meaning that it is approved as ethical, safe, and that it delivers the proven clinical benefits. A separate decision will be made on the 16th  December as to whether the NHS will be willing to fund the drug in the UK. If it is not funded, Sam’s treatment would stop unless the drug company approved a short extension of treatment on ethical grounds whilst it fought that decision. The drug is a man made enzyme, replacing the one Sam doesn’t have. Its aim is to slow, and in the best case stop the progression of the disease. However pre-existing impacts of the disease cannot be reversed ... meaning that if treatment stopped even for a short time, there would be irreparable consequences. The drug is having a significantly positive impact on Sam – he has grown, his posture and stature is better, and physically his abilities have not deteriorated at all, in fact they have improved. Health wise he has been less prone to chest infections, and his hearing has been normal for two years. Sam not receiving the drug is unthinkable. The consequences are unbearable.
Sam could end up immobile within a few years, which would mean he would need significant services involved with managing his care needs. The drug Vimizim could hold the degenerative effects of the disease for many years, giving Sam an increased quality of life that previously could only be dreamt about. Vimizim gives Sam hope for a brighter future. 
Being part of the trial has been a huge huge commitment, Sam misses a day a week of school and it causes significant family upheaval. If it didn't work and give Sam significant benefits to the quality of his life, the upheaval would not be worth it. 

Thank you for reading this x 

Wednesday, 12 November 2014

Remembering .........

I have just tried to post this update to Facebook, but it won't let me! It says the content contravenes facebook policies! So here it is, where I can freely post it!

3 years ago today Harry had not one, but 2 ultrasounds on his abdomen at LGI.  2 weeks prior my pleas for an ultrasound had been ignored during a stay at Airedale hospital. He only had an x-ray, which just highlighted constipation!
On this fateful saturday evening I was told on my own (Paul was at home with Callum), in the parents room on ward 51 that Harry had a large tumour sat on his adrenal gland, possible into his kidney too. The tumour stretched right across his abdomen. I heard the words Neuroblastoma cancer for the first time. A lot of the conversation is a blur, but I distinctly remember being told that the treatment plan would involve us having to take time off work and it would last for months (not wrong there!). Wow I thought, money, Callum, our family.....
So we were catapulted into the world of oncology. 
Within 1 week Harry had, had several anaesthetics, scans and one big biopsy, which involved a 9 inch incision. He also started chemotherapy;  had his first blood transfusion; his first NG tube; his first port was inserted during his biopsy, which has been accessed via needles,  so many, many, many times for treatment, blood tests and for the many blood and platelet transfusions that kept him going. 
Our world was turned upside down....
So 2 years ago we were praying that the treatment would be a success and the nightmare would be over, alas not so.
1 year ago, we prayed that Harry would make one more Christmas and have quality of life to enjoy it. Our wish was granted!
This year, we are nursing fractured hearts. Working our way through the mist of grief. Grief is a form of disability, but it is masked, not easily visible to most and unrecognised. A broken heart is what we have. Like having a disability we have good and bad days, we smile,; we laugh; we cry. Each and every day we think of Harry and have numerous private gulping moments. As time passes we get better at putting a mask on. 
The lesson is to learn to fight, carry on and not let cancer consume or define us. 
Life is for living, none of us knows when our journey will end. 

So to mark the first anniversary of Harry's passing, we will be having Harry's ball at The Craiglands Hotel on 25 th April 2015, in aid of Nuzzlet's farm and Candlelighters. The theme will be pirates, a theme very close to Harry's heart. Tickets have now been printed and are available.  Inbox me for more information or how to pay. Cheques should be made payable to Harry's Ball.! 
Live, love, laugh,
because Harry did! X

The picture below of Harry was taken in September 2012, post radiotherapy. He was slipping through our fingers right before our eyes, constantly vomiting from the effects of the radiotherapy on his abdomen. I pleaded yet again for him to be admitted. Eventually at the end of August he was readmitted and commenced on TPN (total parental feed via his port). Fortunately he began to regain strength, but not before one of his lines in his port became infected! The trials and tribulations to just try and seek survival. The amount of machines and fluids that Harry is attached to speaks volumes! The path of childhood cancer is a long and winding one and effects not only the patient and their family, but a community too.