(About Huntington’s disease has been successfully treated for the first time in a groundbreaking clinical trial) This news comes exactly 10 years after I spoke on a panel to the FDA begging for the same type treatment for JHD and Cory’s life. A panel of scientists, doctors who admitted that they learned a lot from the panelists that day, and later denied our request, stating that more research was needed. Granted after the meeting, I came home and told Terry I felt accomplished in a way, and was at peace with whatever happened to Cory. But 3 months later, Cory passed, so this news hits a little differently for me.
it’s a 12 hour brain surgery, that will be so expensive, not covered by insurance, and only will be available to the adults, maybe kids in late teens, in the early stages of the disease. Even if it had been approved when I went to the FDA, or when Cory was diagnosed at 15, he probably wouldn’t have qualified. If he had, I know my Cory’s Crusade team would have done everything in their power to make it happen. But this only significantly slows down the progression of the disease. Yes to the point some may never experience the last stages before their natural death occurs. I refused to let Cory have a brain surgery for a deep brain stimulation device because they only gave it a 50/50 chance or being successful, I had seen it work for maybe 1-2 people, and worse for others, I couldn’t put Cory through that pain with those odds. I am still completely comfortable with the decision I made then.
While I am happy for those that this treatment will help, it is also a slap in the face. I am right back to the day we lost Cory, so I am sad one minute and pissed the next. I appreciate everyone being excited for me, but it’s going to take me some time to get there. To everyone reading, thank you and I do love you. So I am going on a pre planned camping/kayaking trip with friends for my birthday month, and going to disconnect for a few days. I will be fine, just give me time to process
(About Huntington’s disease has been successfully treated for the first time in a groundbreaking clinical trial) Im so happy for the future of HD but unfortunately its too late for warriors like my Emma.
(About Huntington’s disease has been successfully treated for the first time in a groundbreaking clinical trial) YES! I’ve heard about the successful “treatment” (NOT CURE) in Huntington’s Disease! Which, don’t get me wrong, is absolutely amazing and holds hope for the “future” What I think some people need to understand though is this is not a magic capsule everyone with HD can grab and they’re good! AMT-130 will be a preventative treatment. It’s goal is to delay or stop symptoms from starting. It WILL NOT REVERSE/CURE damage already done by the disease! The new TREATMENT is a type of gene therapy given during 12 to 18 hours of delicate brain surgery, data shows the disease was slowed by 75% in patients. The drug WILL NOT be available for everyone due to the highly complex surgery and the anticipated cost! It will also require that EARLY TESTING so many are against as it’s only for those who are PRE-SYMPTOMATIC and those up to stages 2 and 3. NOT for those who are further progressed in the disease!
I had to edit this post to add Jane’s input for those who may miss it in the comments. This is VERY TRUE!
Jane Mervar: It also will not be appropriate for the very young kids with childhood and infancy onset because it also targets the proteins that are essential for brain development and not necessary function for older young people and adults . The JHD study at Uc Davis under kyle fink is does not have the amt of funding as many of these companies fund! So might be a worthwhile place to fundraise for Juvenile HD???
(About Huntington’s disease has been successfully treated for the first time in a groundbreaking clinical trial) Missing Brandon to this 4.5 years ago getting notifications about this “Cure” please read article Is No Cure, won’t help everyone. I’m sharing this because still be generation battling. Having mixed emotions as we all do 1812 when HD was discovered all the funding etc it took this long for this breakthrough let’s get back to reality. It’s a good achievement not 100% We all have opinions this is mine…
Before telling someone who has a loved some dying from Hutington’s Disease “CONGRATS ON YOUR LOVED ONE GETTING A CURE”, PLEASE read the article. I have it posted on my page and will drop it in the comments, as well.
This treatment is not a cure. It is just that: a *ground breaking treatment*. Not a cure. Not an “end all, be all”. It won’t even be available in the US until AT LEAST the end of next year and its a gene therapy brain surgery that costs well over over $80k that will not be covered by insurance at this time because it is so new and still undergoing trials. Furthermore, it is for individuals with adult-onset Huntington’s Disease in the early to mid-stages. Not Juvenile Huntington’s Disease, not 3rd through end stage HD. The criteria one must meet to even qualify for the procedure is also incredibly strict.
So yes, while this could prove to be a major source of excitement and celebration to those who are HD+ in the the future and it is definitely something worth getting excited over for those who have loved ones that are at risk, it is *not* going to cure or help those of us who have loved ones that are actively dying with juvenile and/or adult onset HD. Thanks for coming to my talk. Stepping off my box and retreating back into my hole now.
(About Huntington’s disease has been successfully treated for the first time in a groundbreaking clinical trial) I randomly shared a video of my niece a couple days ago…. and within 24 hours, it was all over Facebook that they’ve successfully treated Huntingtons Disease. I’m sad that it was too late for my family.. but so excited for what it’s going to do for HD families in the future. I haven’t shared much about her lately… so the timing of the two posts really had me thinking. My daughter being born two weeks late.. on the anniversary of my nieces passing, wasn’t just by chance either.
I grew up in the church. I never questioned my faith when I had to watch Huntingtons disease slowly kill my father. Passing the day before my 16th birthday. I didn’t really question my faith in God when I had to watch my sister slowly and painfully be taken from us…. Passing at 24 years old. Leaving her young daughter behind.
I could almost understand adults having to suffer because of their sins….. but I could never understand why my niece had to suffer the way that she did. We had her at a Huntingtons Disease Walk… where she was walking in memory of her mother. We were playing at the park and I was climbing through a little tunnel chasing her… when she had her first seizure. I remember running with her, praying as hard as I could. My faith in God changed that day.
I spent the next 16 months watching her decline. Watching all of my nieces and nephews running and playing… doing everything that a child should but watching her struggle. Band-aide after band-aide… boo-boo buddy’s.. ice packs… while just trying to play and keep up with other kids her age. Until eventually a wheelchair was suggested. This angel was the best human I’ve ever seen in my life. Smiled until the very hour that she passed. I want to have the faith that I once had.. but no one has ever explained this and made it make sense. If she was suddenly taken, I wouldn’t be angry like this. Why the years of absolute SUFFERING?
Car accident 1- “God was really watching out for you and your family. It could have been so much worse!” Car accident 2- a young girl dies. Why was accident 1 worth saving but not accident 2? Stuff like this makes me question everything. I want to believe.. I want to restore my faith… I just think that I’ve watched way too much awful stuff happen without understanding the why. Don’t come at me saying that “you just have to believe that God had a plan” or “you just have to have faith.”
I did had faith.. until I held the hand of an innocent child, while she took her last breath.. a child that suffered for years. And don’t get me started on why we had to deal with that awful neighbor for years on top of this. Help me understand.
(About Huntington’s disease has been successfully treated for the first time in a groundbreaking clinical trial) My first reaction was Not excitement it was like a deer in the headlights, Frozen unable to comprehend or process the news . Then I began to think of my daughters ,will this work ,what will it cost , will one of the two girls qualify ,if so how does that work and how do you have one daughter treated and the other not ,emotionally ethically financially BUT also physically present in two places? Then came the conversations ,neither were even interested ,thats tough because everything in my nature wanted to fight for them BUT their words rang truth ,Im tired of feeling unwell and hurting and I dont want a brain surgery only to cause these things to last even longer . That solves the what if one can have it dilemma .BUT then the conversations about the actual study data whats there and what isnt . Who will be helped potentially and who will be left behind . Dont get me wrong ,I am happy for the advancement happy for the people this might help . I currently am stuck with a feeling of sadness and concern for all the people who have already left and those who are well into their progression and ESPECIALLY the Juvenile population . Im surprised at some peoples reactions ,sending the announcement to a mom whos child has passed saying If only ,or those who say you NEED to fight for this after mom expresses its not for my child or actually most of them. I have appreciated those who have shared this and respected even appreciated that its not for every person .
Those of us it isnt for can be excited for the people who it might help and excited for the advancement in research ,BUT we grieve what isnt for ours. We celebrate what is for those beautiful people we know and love like family . We do this while still feeding medicating assisting caring for….. some pretty special people also . Its overwhelming right now and its overwhelming on the best of days . I seriously cannot help but feel the momentum to raise awareness for the young people, fundraise for the research and to help them have a fighting chance for an exciting breakthrough also needs increased momentum once again .
This advancement may eventually lead to something that will help kids also? BUT one thing Ive noticed is it also targets proteins production THAT tells me its not going to be available to children ,they require those building blocks to grow their brains and nervous system . I dont even know if older teens would ever be in line ? brain isnt developed until what age? 26 ? Not to mention by that age the “normal/familiar ” presentation will already have eliminated most from meeting criteria ,I say most because there is a population of juveniles who were diagnosed with a lower CAG and slower progression ,those young people alot is still unclear about .
I can think of therapies that also indicate they slow the disease if introduced early enough and many are skeptical at the very least about them . I get it there is far more security and trust in what HD specialized Drs prescribe . I personally dont trust anything that Drs cant explain the reasoning behind and are not following my family members with, even natural supplements and vitamins . Anyhow thanks for getting this far with me and please be aware people are grieving and celebrating and its all for legitimate rational reason so try to be mindful of both extremes and know that NO ONE is intentionally saying this isnt for me or ours its not a one size fits all situation .
Had my husband been alive still and eligible imagine that because our girls would not have been ,this caused me to recall ethical decision making in college ! There are possibly people you know who are faced with similar dynamics they need compassion and support !!I can say as a JHD mom we learn an early lesson that life for our kids isnt fair in a way many people might ,hopefully, never know . The news is a reminder to me of this reality .I know there are researchers who are working hard to tip that scale for our children and they are a priceless gift!
(About Huntington’s disease has been successfully treated for the first time in a groundbreaking clinical trial) This is the BEST news! It is hard to not think, ” I wish my daughters, Carissa Krivanek and Judy were alive and healthy enough to have benefited.” That is my selfish thought. My heart is so grateful that others will benefit from this.
(About Huntington’s disease has been successfully treated for the first time in a groundbreaking clinical trial) Yesterday I cried all day. 7 times I got messages cheering that now Greg can be cured! How excited I must be to know he isn’t going to die from JHD. After the 3rd I stopped responding. Because while I know they meant well, they aren’t on this journey. They aren’t a full time caregiver to a 28 yr old 275 lb woolybugger who’s mentally 10 on a good day. They aren’t here watching him panic if I walk over to the neighbors house, they don’t fix his meals, manage his meds, remind him to shower and brush his teeth and change his clothes. These well meaning friends didn’t have to sign DNR, DNI paperwork for their child or have the number for the brain bank in their phone for when their child passes. So while I am hopeful and want a cure more than anything in this world, today I have cried… For our boy with JHD.. for his dad and two uncles who are in mid to late stages of HD.. for his Pepaw and great grandma who lost their battle with HD… And for all the mommas I have been blessed to know who have a child with JHD, or who have lost their babies to this horrible disease….
(About Huntington’s disease has been successfully treated for the first time in a groundbreaking clinical trial) It breaks my heart that it has taken 11 years since I wrote this post below. Heartbreaking for all those amazing, beautiful kids (and adults) with HD and JHD who we have lost. 
But the incredible clinical trial results from Uniqure mean that this enemy, Huntingtons disease, CAN be defeated. And that is a huge win for all of us to celebrate. Research to selectively knock down the mutant Huntingtin RNA and protein are still ongoing with many groups, including ours with Dr. Kyle Fink, and NO one is giving up on the kids. 
-Jan Nolta (Researcher)
JHD Initiative 