The Fight for Precision Medicines Continues.

So this week NICE announced its decision NOT to recommend the gene specific medication Orkambi to the NHS in England. Orkambi is the first drug ever to be manufactured to target the root cause of Cystic Fibrosis. Up until now all treatments have been simply treating the symptoms, not actually changing how cells work inside a CF patient’s body. It’s incredible that one pill can attempt to do that and equally incredible that my daughter has been born at a revolutionary time with precision medicines. However, because this drug is one of a kind, it comes with a fairly hefty price tag. This drug is already available and in use in the USA, Australia, France and Germany. Not. Fair.

I often think to myself, ‘well, it’s ok, my daughter is way too young to take anything like this anyway and by the time she’s six an even better version of this pill will be readily accessible for her’. However, what about all the other, older ones with CF who are just sitting waiting, lung function slowly declining? Results with Orkambi have not been amazing. From all the testimonies I have read, it seems to work fantastically for some – completely changing their lives. They can live life unlimited with CF.  On the flip side I have read personal stories of it not doing anything for a patient at all. But how can someone take away the right to give it a go, take away someone’s right to a potential full life? Orkambi has been shown to stabilise lung function (I’ll happily take that) and reduce the need for frequent hospital admissions for IV antibiotics, therefore, saving money.

In a previous post, linked here, you can read just how good this drug has been for a CF patient. Why should some countries have access and some not? Are their lives more valuable than ours? I truly believe that this drug will eventually be approved but at the cost of how many lives?

How can you help? You can click here to email your local MP. It takes all of two minutes. I know lots of people local to me will be bombarding the inbox of our local member of Parliament but what would be great would be people who read this living in different areas bringing this to the attention of their MP. Let’s keep my girl always as happy as she is now!

If you would like to read more about Cystic Fibrosis and find out other ways you can help click here.

An Afternoon Out For Nowt (ish): South Shields

A few weeks ago I decided to try an afternoon out a little closer to home with my two daughters aged 4 and 18 months. Ten years ago an afternoon out in South Shields would have been something totally different and this was an entirely sober affair. South Shields has a great, free museum which I hadn’t been to in years so I thought we would give it a go. It hasn’t changed much since I was a girl really but still looks brilliant. I thought a lot of it wouldn’t be of much interest to my eldest but to my surprise she really enjoyed looking at everything. Upstairs they had some of the ‘Spineless’ exhibition which had been on at The Great North Museum and when I realised I thought she might have been bored seeing as she had already seen it but thankfully not.

There wasn’t as much as at The Great North Museum but still lots to do and occupy little ones. They have some tie-in events coming up too; a rainforest roadshow and a craft afternoon which you can book into over the Easter holidays. Click here for more details. Just along from the Spineless exhibit was a wonderful collection of art which I really enjoyed; kids probably not so much! I also love The Victorian Pantry based in the museum and have often popped in there without going into the museum. Well worth a visit.

Just up the road from the museum is a fairly new place called Stay and Play Tearooms which we tried next. As I had parked near the library I walked up with my youngest in the pushchair so was rather bewildered when I arrived to a full flight of stairs to get into the building. Fine if you’re with another adult but I was on my own and had to take Harriet out, run up the stairs, plonk her in the doorway then run back down for the buggy. This, however, would be my only criticism. The Tearooms are small but the benefit is that for the price of a cuppa, Mams can sit down and keep a close eye on children playing right by them. I often get a little panicky in huge soft plays when my eldest goes off and I can’t see where she is!

Along with the main play area which has a slide, ball pool and heaps of toys there is a lovely little sensory room which my two loved. The Stay and Play Tea Rooms are located on Beach Road in South Shields – click here for more information.

I have been back a few times now to Stay and Play and they kids have always had fun. In fact, my sister has booked it for her daughter’s upcoming birthday party. I would say it is best suited to under 5’s as my four year old looked a little big to be there sometimes! Definitely another one to check out over Easter.

Harriet’s Sixteen Month Update

This last month has been a busy one for Harriet. From birthday parties to clinic appointments to her first holiday abroad! She is well at the moment despite me putting in ANOTHER phone call to the CF nurse this morning about a niggling cough. Sixteen months in and I still have no idea what we are dealing with here. CF is such a complex condition. She has only just finished an extra course of antibiotics for a bad cold and cough, and when I took her for her clinic appointment four days after completing the course I felt she was starting to show symptoms again after only being well for about a week. I was slightly worried as we were due to go away two days afterwards so I was given some powered antibiotics to take with us just in case the suspected cold developed.

At Harriet’s routine clinic the doctor we saw that day informed us that from reading her notes she has only had one month free of any antibiotics so far. Great. I do feel like we have just bounced from one cold and cough to the next but to hear that as fact made me worry. What does it mean? Is CF going to be worse and more severe for my girl? I expressed this concern to the nurse who was lovely and reassured me that they do see lots of children go through the same as Harriet when they are young especially when they have an older sibling. Nancy brings home all sorts of germs from nursery and she too has had her fair share of snotty noses this winter. It’s inevitable she is going to pick up everything. The only plus here is that Harriet has always responded well to the extra antibiotics and has never needed to be admitted for a two week course of IV’s although it has been a close call on a few occasions I think!

There was also a slight concern in that Harriet had only gained 100g which isn’t much since her last appointment. I mentioned that she has been going through a rather fussy stage, only wanting to eat sweets and puddings but the doctor made a note to monitor this closely. So much emphasis is placed on children with CF gaining weight. Because their bodies cannot digest fats properly it can be hard to put weight on despite taking enzymes before eating anything with fat in it. Cystic Fibrosis patients are advised to eat a high fat, high calorie diet which we try to do but now it’s become my mission to fatten her up! She remains on the 75th centile for her weight and 91st for height. In fact she had grown another two centimetres in length which was a positive.

Harriet’s percussion physiotherapy has improved so much lately. She is very complient at the minute thanks to the iPad and Peppa Pig – long may it continue. Our physiotherapist was pleased that we are now able to complete two full twenty minute sessions in each day. What was mentioned, however, was the fact that she is still not walking. She can crawl and sometimes does but her preferred method is the bum shuffle. She is so fast and gets herself all over. She tries to stand up and will hold onto things. Oddly she will push herself along on ride on toys but if you try to get her to walk she takes a few steps then either wants to be picked up of just plonks herself back down! On our recent holiday she became the star of the show at the children’s disco! Every night she bum shuffled herself out into the middle of the dance floor to get involved and the audience would clap and cheer for her! It was like being on holiday with a celebrity as people would see us through the day and not speak, just imitate her shuffle technique! At the minute she is trying so hard to stand but seems to be going about it in the hardest way without holding on to a piece of furniture and just going for it unaided. Anyway our physio is arranging for someone to come out to us to help get her walking in the next few weeks so fingers crossed this will help.

I had hoped that the warmer weather in Lanzarote would have helped with Harriet’s cold but it didn’t do much. She continued to present with cold symptoms for the week but only coughed a lot at night. At the minute I am doing her percussion therapy through the night too as this is when she is at her worst. She isn’t on any other medication at the minute and hopefully won’t be. I still can’t understand why the coughing is only happening in fits and starts. I hate CF! As always though she is her usual smiley, happy self. She is starting to say lots of new words too which is very cute. I am, more than ever, determined to keep on doing as much as I can to raise awareness and funds to give Harriet a brighter future. As always, your support, shares, ideas are all very much appreciated.

If you would like to learn more about cystic fibrosis and ways you could help click here.