Daily/Weekly Thoughts
Daily/Weekly Thoughts

CTV News Clip - Spinraza for all

 

Emmett loved seeing himself on TV. He is a very bright and alert little kid, but I really cannot say that he knew that he was on the provincial news. He did, however, absolutely want to have the clip reviewed and watch it over again and again. He also recognized some of the other kids in the video - some he's met before while for some others this was the first time. 

Emmett fits the recommendations that are currently being reviewed by local provincial government here in B.C.  This is not merely a disease that affects children. Type 4 is adult onset Spinal Muscular Atrophy. Type 3 may not be identified until into the teen years - although it is usually evident at a much younger age. Then there are Type 2 youngsters who are identified early by the fact that, while they can sit up, they are unable to walk. As you can imagine, there are a range of ability within each of these types. Emmett is Type 1. Not all Type 1 individuals will meet the narrow recommendations.

The teacher (and perfectionist) in me wants to clarify. Kids and adults with SMA have perfectly fine muscles. The problem is that the message between the brain and the muscles is not transmitted. Thus the muscles atrophy. This drug, Spinraza, essentially creates a pathway that allows this message to get to the muscles. This is why this drug is so fantastic. And so fantastically expensive. But when governments, most importantly our Canadian government, around the world start discussions with Biogen, they will be able to get the price to a much more realistic figure.

This is why we need and want you to write your local MPP and your province's Minister of Health. See yesterdays post for that information.

Emmett has a couple of cameo's in this clip. Watch for him! He was asked for an interview but he declined! He just wanted to play!

 

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Here's what you can do!

Baking Up An Angel Food Cake!

Recently we posted that a recommendation was being made that would severely limit who received access to the miracle drug, Spinraza. Provincial government officials will be reviewing these recommendations and then making the decision about this new drug. Right now, we believe that it will be beneficial to write to your local Member of Provincial Parliament, your Provincial Minister of Health, and your Provincial Deputy Minister of Health and express your concern. To assist you, we have drafted a letter that you may want to use - click on the province in which you live. These are in rich text format, which will hopefully work easily with the word processing program of your choice. One is drafted for those living in British Columbia (click here to download) and another is for those living in Ontario (click here to download) - we've used these two provinces as that is where the majority of Emmett's family lives. If you live in another province, we encourage you to write to your provincial leaders. If you need help adapting this letter, please let us know.

What does this young baker have to do with needing a drug called Spinraza? Absolutely everything. Without Spinraza Emmett could not attempt to stir the batter. Without Spinraza he would not be able to even hold something as heavy as this wooden spoon. Without Spinraza Emmett could not hold his head up. Without Spinraza Emmett would be so limited in what he could do. Please take the time to copy one of these letters, fill in your information, and mail or email it one or hopefully all three of the listed names for your province. All Canadian's with Spinal Muscular Atrophy need to have access to this drug.  Our Government needs to talk to Biogen, the company that makes Spinraza, and get the cost of this drug down to a more reasonable level. Patients cannot get pricing changes but the government has the ability and purchasing power to make that happen.

Thank you for your help and support.

 

Don't want to click and download? Here's the text of the letter that you can copy and paste.

Below is a sample letter that may help you develop yours. Items underlined can be changed for the specifics of your letter. At this point we feel the most valuable contacts would be your MLA (link below for BC MLAs), Adrian Dix (BC Minister of Health), and Doug Hughes (BC Deputy Minister). If you don’t live in BC, we would appreciate you taking the time to send to your provincial representatives. Approval of Spinraza is something that will be a challenge all across Canada. Thanks you so much for your support of our family, and all other families affected by SMA!

Sincerely,

Alisha, Josh, and Emmett

 

Your Name

Your Address

Your Phone/Email

 

Date ??, 2018

 

Subject: Approval of Spinraza (Nusinersen) treatment for all patients with Spinal Muscular Atrophy

 

Dear Honorable [MLA]:

 

My name is _______________ and I have resided in the _______________ area for over ______ years. I am a (your job).  My ___(friend, family)________, Emmett Willms was diagnosed with Spinal Muscular Atrophy, Type 1 when he was five months old. SMA is a genetic, life-threatening and seriously debilitating neuromuscular condition for which there is no cure. In December 2016, the US FDA approved a medication called Spinraza and Health Canada followed suit in July 2017 with their approval. This news brought hope to the SMA communities worldwide as this treatment was proving transformative, especially for those starting young. Emmett began a clinical trial just over two years ago (for Spinraza). Patients in the trial achieved milestones such as the ability to move arms, legs, and sit unassisted when they would otherwise be unexpected to do so. They reached these milestones when specialists expected that they would be forever lost, and lifespans are now, rather than terrifyingly short, happily extended. The overall findings of the controlled double blind trials support the effectiveness of Spinraza across the range of SMA patients, and appear to support the early initiation of the treatment.  

 

For the first seven months of Emmett’s life (before starting on Spinraza), he lost the ability to move his legs or any significant arm movement. He lost the ability to swallow. He could not sit, hold his head up, or even sit in a car seat. His physicians doubted that we would ever hear him talk. They prepared us for the fact that he would not survive. We feared for his future.

 

In the past two years since beginning the Spinraza Trial, Emmett has gained strength in so many ways. He has gained very significant core strength and head control. He is gaining strength in his arms and movement is returning to his legs. He can sit in a car seat and, with lower back support, can sit and hold his own head up. He is incredibly bright and eager – chattering like many other two and three year olds. Emmett has had a power wheelchair since he was 16 months old – he enjoys the freedom that this mobility offers him.

 

Without Spinraza, Emmett would never have been able to do these things and, as SMA is a degenerative and terminal disease, his lifespan was drastically shortened. With Spinraza, Emmett is literally so much more physically involved in everything that is around him. Raising a child with SMA is taxing and emotionally and physically draining on caregivers.  Spinraza will ease the burden on families because patients will be stronger and more capable.

 

Spinraza (manufactured by Biogen) will soon be under review at the provincial level and I hereby request that you support the decision to approve this treatment for all those suffering from Spinal Muscular Atrophy.  It is a rare condition that affects 1 in 6,000 to 1 in 10,000 patients, so there are a small number of patients living in British Columbia with the condition.  All of them deserve a better quality of life and access to treatment.  

 

Other countries such as Sweden, France, Israel, and the US have treatment available for all types of SMA regardless of age, the number of backup genes and other symptoms.    

 

For more information about SMA, please visit CureSMA.org or CureSMA.ca.

 

Below is a link to a blog that documents Emmett’s improvements while on the clinical trial.

 

www.emmetts-journey.com

 

If you are unable to use the link above, if you search “Emmett’s Journey SMA” on Google or in YouTube, you will find links to Emmett’s Journey.

 

Thank you in advance for your attention to this very important and urgent matter.  I look forward to hearing from you with regard to this request. Please feel free to contact me at the address and phone number above.

 

Sincerely,

 

[Your name]

 

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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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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 3 weeks ago
Site Visitor
+1
Poor Comment Good Comment
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 3 weeks ago
Site Visitor
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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 2 weeks 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 2 weeks 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.