Daily/Weekly Thoughts
Daily/Weekly Thoughts

December 2017

Canadian Decision Shocks SMA Community

The Canadian Agency for Drugs and Technologies in Health just published a report that  recommends only allowing the miracle drug Spinraza to be made available to a very small subset of Canadians who have Spinal Muscular Atrophy. As you can imagine, this is a heart wrenching decision that has filled many Canadian parents and individuals with SMA with fear and sadness. CADTH doesn't make the decision, that's up to each province, but the provinces widely listen to and often accept CADTH's recommendations. 

We literally do not know what to say.

If you have been following Emmett's Journey, then you know that he was one of 124 infants worldwide who were part of a blind study and that he joined this study in the fall of 2015. In the care of professionals at BC Children's Hospital, we quickly started to see improvements. This was a blind study, but when improvements begin happening in an 8 month old who had lost all movement in his legs, had minimal arm movement, with very limited movement his head, and was being fed by G-Tube, then you quickly notice. Spinal Muscular Atrophy is a degenerative disease. There may be plateaus but evidence demonstrating improvement is simply not there. We saw and still see improvement so we know that this drug, Spinraza, works.

Emmett's parents were concerned about him participating in a medical study. I've shared before that as grandparents we thought it was a crazy idea. I've also shared that we were wrong and that we're so glad that Alisha and Josh agreed to have Emmett participate. He was one of four kids at BC Children's Hospital who were part of the trial for this drug that came to be known as Spinraza. The three other children, sadly, did not survive. You see, Spinal Muscular Atrophy is a genetic disease that takes the life of more babies than any other genetic disease. It literally shuts down their respiratory system. This is after it has shut down their ability to move their limbs. It is a terrifying disease.

That is why we are terrified by this decision. The drug is already horrendously expensive and that is obviously a challenge to families dealing with SMA. Yet this drug, Spinraza, is the absolute first treatment option available to individuals with SMA. Emmett has SMA Type 1. Some little ones are identified even younger than Emmett - who was officially diagnosed at five months. Some have Type 2 which is a bit later onset. Then there's Type 3 and Type 4. This decision, if the provinces act upon it, bars Type 2, Type 3, Type 4 and even a large number of Type 1 kids (and adults) with SMA from having access to this drug.

Now, because Emmett was and is a part of this study he probably fits all of the criteria that CADTH has set out for access to this drug. That, however, is not the point. Obviously, we want Emmett to improve and that is not in question. But this drug works for all SMA types, so why is it being limited? It is life changing for anyone with SMA and their families. Emmett was not in this trial for himself - he was in it for everyone. Think about it, when Emmett got involved in this it was an unproven drug. No one knew what would happen - although there were indications and a lot of hope that it was positive. But Emmett's parents took a leap of faith - hoping beyond hope for a miracle for their son.

Emmett unknowingly worked very hard to get this drug approved. He has benefitted from this drug, Spinraza, which I absolutely call a miracle drug. That it was approved in the USA, Canada, and several other countries is a miracle. It is available now in the U.S.A. for all types of SMA. Now we need another miracle - a Canadian miracle. We need the experts at CADTH that look at drugs to realize that they need to expand their point of view and recommend full access to this drug. The decision they have made is based upon scientific fact but to the narrowest of definitions. As a result there will be more Canadian babies who die of Spinal Muscular Atrophy. Children and parents who have been waiting years for a treatment will be denied access - their hopes dashed.

To those who question the expense of this drug, please realize that I question the expense too. It's literally beyond belief. Then again, creating new pathways for the brain to be able to speak with muscles that control lungs and muscles is absolutely beyond belief.

Please watch this blog - we hope to be able to share ways in which you can be an advocate for wider access to this drug here in Canada.

Links:

https://www.cadth.ca/nusinersen - nusinersen is one of the early names of the drug Spinraza

Cure SMA Canada

 

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Guest Comment 6 months ago
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This is absolutely shocking! I will be watching this for ways we can add our names to those advocating for those in need of this drug. I am so sorry that this news has interrupted your Christmas joy.
Frank Ewald 6 months ago
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I will be posting some photos and hopefully video clips in the next day or so that highlight Emmett's joy of Christmas! He has had a marvellous Christmas!
Guest Comment 6 months ago
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We are getting it for our type 3 son who is 3 years old using our private insurance and we saw drastic changes just under 2 months. He is approved for a year and we don't know what we gonna do after the first year. If our private insurances deny saying the province should cover this then we will have to go again and see him lose all abilities. So hard and sad.
Frank Ewald 6 months ago
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Thank you for that update about your son and the improvement you're seeing. We will hope and pray - and work hard once we know how to approach this - to ensure that Spinraza is available to all who need it.

It Was Christmas!

Merry Christmas, Everyone!

Emmett has been so excited about Christmas for weeks. At night while outside he would look up into the sky and exclaim, "Ho! Ho! Ho!". Inside he would want to have parts of the Nativity scene - to hold and play with, specifically Mary, Joseph, and the baby Jesus. So when he had the chance to meet Santa face to face, well, he did what most every other two year old would do! Cried and tried to get away! Ha Ha Ha! Even though Santa was his dad ... and even though Josh had put on the Santa suit right in front of Emmett ... it was pure childhood terror! Honestly, there must be more adults with Clausophobia (it's real, look it up on Google if you don't believe me!) than with Coulrophobia. Fortunately, it's only if Santa is up close that Emmett has a negative reaction.

Of course, if Santa is at a distance or a small toy figure then Emmett is all over it! Emmett was right into the Christmas spirit. Helping his dad pass out presents! Telling his mom what was in the presents that she was getting! And he was right, too! Josh handed Alisha one of her presents and Emmett piped up, "Tea Pot!".  Then of course there was present time - Emmett loved presents. He would sit and point at the present that he wanted to open next. His expressions were fantastic - especially when the present was clothes! After the first sweater, some disappointment was evident until prompted by mom or dad to express joy and happiness.

This was also a white Christmas in Abbotsford, so Emmett got to head out on his new sled. He handled it incredibly well while it was stationary. Once it started moving, however, he simply couldn't keep his head held upright. He was quite fine and not injured at all - except for his feelings as everyone who witnessed it live or video couldn't help but laugh. 

 

The conundrum of dealing with a medically fragile child. For example, little ones often grab at and sometimes can break necklaces, glasses, and other items often worn by an adult. Of course, with Emmett that has not been an issue. He either couldn't reach or, even if he did, didn't have the strength to do anything. Well, the other night he grabbed my glasses and hauled them over to his face. That was so awesome and I was so proud of him. In fact, I looked through his handprints on my lens all night long rather than pulling a tissue out of my pocket and cleaning them. Then as we were getting him ready for bed, he started grabbing diapers and tossing them onto the floor. Perhaps his mom and dad would have stopped him, but he was having so much fun and I loved watching him grab a diaper with his left hand, pass it to his right hand, and then toss it onto the floor! So I let him continue to do it. (And since his grandma picked them up, I didn't even have to clean up the mess that the two of us made!) This grabbing certainly came in handy with Christmas presents! In the video you'll note that Emmett does a pretty good job of grabbing onto tissue paper and pulling it out of the way. What really made us laugh was his impatience when we were chatting and not passing him presents. You'll hear a very distinct, "Hey, Daddy!" coming out of this little one. His personality is so charming! There may be a little bit of spoiling going on, but that is something that Grandparents are supposed to do.

From here on it is simply pictures! Merry Christmas from Emmett, his mom (Alisha) and dad (Josh), and his grandparents Rhonda and Roger Willms and Frank and Pamela Ewald. Have a Happy New Year too!

 

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In March I Will Be Three

If you didn't realize this already, I think that Emmett is extremely adorable. And smart. And cuddly! This past November in the beautiful Fraser Valley of British Columbia it has rained and rained and rained. Emmett's outside time has been severely curtailed. Yes, there is a giant poncho to cover up Emmett and his power chair but it is still not as great as awesome sunshine. The result - more reading time. "Book! Book! Book!" is Emmett's favourite phrase. And he got lots of reading time this past month. And reading time means cuddle time!

His speech is getting more and more recognizable. And like other toddlers he picks up on the most adorable things - or things that parents absolutely wish hadn't been heard. Recently we were all getting ready to go outside but I couldn't find his hat (it was packed in a container already in the van) so I put one of mine on him. Then perhaps I commented about him having a 'fat head'. It's quite possible someone else said that as it was a long time ago and I'm having trouble recalling the facts. At any rate, Emmett said 'Fat Head'. Someone laughed. I'm pretty sure it wasn't me. But that started it. The chant was even more pronounced than "Book. Book. Book." Except it was "Fat Head! Fat Head! Fat Head!" over and over again. He would finally stop but then if he saw me the chant would start up again. Sigh! I'm working hard to ensure that I don't teach him any more unique phrases.

 

Yesterday Alisha and Emmett surprised me with a question and answer session.

Alisah, "Emmett, how old will you be in March?"

Emmett held up three fingers and exclaimed, "Three!"

"How old are you now was followed with a two and two fingers held up. We are so excited and happy for the speech and the finger control that Emmett has. We pray for more and more!

Emmett is also thrilled that it is Christmas time. His fascination with the Baby Jesus, in Nativity scenes, has carried over from previous Christmas seasons. However, we have to tell you that it is minor when compared to his desire to see "Ho!Ho!Ho!" who, according to Emmett, is visible every evening in the night sky. Emmett was supposed to have been on an excursion with Canuck Place in which Santa Claus was going to be a prime feature - but the cold that he has put a stop to that outing. Emmett loves his Christmas tree and checking out the ornaments. Wendell the cat also likes the Christmas tree and, believe it or not, also likes checking out the ornaments.

Emmett does love his power chair! The speed! The freedom! The flexibility! That is, until he does something like get stuck or jam a wheel. Then it is a catastrophe of the largest order. Even worse, a time out because he ran into someone. That qualifies as an instant time out in which his joystick is deactivated and he has to sit and contemplate his actions. My goodness, the alligator tears are huge when this happens. Fortunately, his control is quite exceptional and he doesn't usually run into people or things. But when he does, it is quite possible that it was intentional! Of course, there are accidents like yesterday, when he was having fun outside and he accidentally rammed a concrete step. This jammed a wheel and brought his fun to a stop. With a bit of help the wheel was freed up and Emmett was rolling again, chasing a ball around, and then showing some neighbours his new headlight and flashing tail lights. He's visible when he's out for late afternoon strolls!
 
On the ninth Emmett was two years and nine months old. The milestones continue!
 
 
 
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I Sneeze Into My Hand!

Why I don't sneeze into my elbow!

And cough too! If I have a tissue that I sneeze into that - obviously that's much preferred. Then I wash and disinfect often. This weekend Emmett is off limits to me. I have a cough. I cannot see him. We can talk on the phone. Maybe a video call. Just not in person. And all of us who care for Emmett sneeze/cough into our hands. You're probably thinking how uncivilized. Haven't we seen the videos and public health presentations about sneezing into your sleeve, the nook of your elbow, to reduce the spread of germs? And of course we have and we all taught ourselves to do that. But think about it - the nook of your elbow is EXACTLY where Emmett's head goes. Every time you pick him up! Every time. So we retrained ourselves back to sneezing into our hands. And then washing or using sanitizer. If that makes us uncivilized, then so be it.

Right now I'm teaching a group of grade two kids so there are a lot of coughs and germs rampant in the classroom. This teaching position sort of dropped into my lap and when it ends on December 22 then I will have more free time. Hopefully less coughing too. Because right now I do have a cold just like many of the seven year olds in this classroom. The teacher that I'm filling in for set up a washroom system whereby the child who heads out to the washroom puts either the girls' or the boys' sanitizer dispenser on their desk. That way you know who's at the washroom plus the kids know if the dispenser is on a desk, then they cannot go to the washroom. (Unless it's an emergency - please run out fast if it's an emergency.) And they've been taught to use it, in addition to washing their hands in the washroom. Some of the boys caught me using the girls' sanitizer dispenser on my hands yesterday. They laughed at me and I told them sanitizer is all the same. Of course, the dispenser is not - and I prefer using the girls' dispenser over the boys. Just because.

When I have a cold, not only do I miss seeing Emmett but I don't see Alisha or Josh. Were I to give my cold to them, then Emmett's main care givers have a serious challenge. They have to stay healthy. So I stay away, because I have a cold. Even when I didn't have a cold - just a few days ago - I did not go to see Emmett until I'd showered and changed clothes. And I would often wear a medical mask. You see, we're germophobic. At school where I'm working (and my entire career has been working in schools) it is not reasonable to expect that a child will not arrive without a cold - schools seem to be a hot bed for germs during some times of the year, thus we need to be germophobic.

In spite of that, Emmett has a cold. Not a hospital stay cold, but a cold that causes him to have more secretions than normal that then require more suctioning than normal and then more bi-pap time than normal. If it wasn't so worrisome, it would have been extremely cute when Emmett's voice went hoarse earlier this week. He actually was laughing at himself, because he thought his voice was hilarious. We had a nervous laugh - worried that this cute voice might result in something scary. Emmett has his voice back but this cold is still there. It's not bad. He's fighting the bug. And that in itself is amazing. Before this amazing drug called Spinraza, this little cold would have been much more challenging for Emmett and most likely would have meant a stay in the ICU. Even now, it is scary. His little lungs simply don't have the strength to handle a bad cold.

If you have a bad cold or some other potentially infectious ailment, steer clear of Emmett and his family. You're a risk that they cannot afford. In fact, steer clear of all medically fragile little ones. While you're at it, steer clear of medically fragile large ones, senior citizens, and ... well ... steer clear of everyone. I know, it's not always possible to steer clear of everyone. I was coughing at school yesterday. Into my hands. And washing them often and using sanitizer. So often, in fact, that the boys caught me using the girl's sanitizer container!

I sneeze into my hand - because Emmett's head is going to be cradled in the nook of my elbow.

This message has been approved by Emmett!

 

 
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