Author Topic: Miracles on the Horizon! (Read 50752 times)

crustpope · « **on:** August 30, 2011, 11:04:40 PM »

First of all, thank you all so much for the prayers that were offered on Micah's behalf at Nationals. While I was unable to be there with you in person, it means so much to me to know that I am part of such a loving and caring community.

Recently, My wife and I have had some Amazing news that we would like to share with all of you

To help you understand what Muscular Dystrophy is, basically it is a degenerative muscle disease where all the muscles of your body (including your heart) slowly deteriorate due to the fact that an MD patient cannot produce (or does not produce enough) Dystrophin. Dystrophin is a protein that protects muscles from deteriorating. In an MD patient any muscle damage is basically muscle lost forever. MD patients usually lose the ability to walk on their own by age 12 and often die by the time they are 20. Usually cases are not caught until the child shows significant weakness around the ages of 5-8. My son's case was caught much early ( he is 1 and a half) because we were having him tested for something else and happened to stumble on this.

As far as Micah's news goes, short version is this: They are in the final stages of a treatment for Micah's version of Muscular Dystrophy. To explain it in detail gets pretty complicated but if you imagine Micah's X chromosome as a computer program, the part of his X chromosome that produces Dystrophin, a protein that protects muscles from deteriorating, is damaged. When the DNA is read, it gets to this point and crashes, producing no dystrophin. This lack of Dystrophin production is what causes his accelerated muscle deterioration.

In the successful treatment, they have inserted an engineered molecular plaster that helps the gene "skip" the damaged portion and still produce Dystrophin. This is called "Exon Skipping." In the first trials individuals had dystrophin production increase from 40 to 100%. while 40 doesnt sound like a lot, you only need about 36% to retain major motor function (read: be able to walk into your elderly years). The great news is that starting next month, the full scale human subject testing on Exon Skipping will begin at Cincinnati Childrens Hospital. While Micah is too young to be a candidate for the test, this test and its (hopefully) successful conclusion will be over long before He shows symptoms. Furthermore, I have it on good authority that given the fact that this is the FIRST treatment for MD, when it goes before the FDA for approval, it will be fast tracked and approved barring no major problems or complications are revealed in testing.

This is absolutely AMAZING News and has the MD community in a tizzy. Of course the medicine will be astronomically expensive but at least there will be a treatment option available instead of the long goodbye that many of those who suffer from MD currently have to look forward to.

I want to encourage all or you to continue praying for a successful testing and that we are all able to find ways of funding and paying for this treatment. I have enclosed some recent pictures of Micah and my family so you can have a face to pray for as you lift him up. Thank you all so much!

crustpope · « **Reply #1 on:** August 30, 2011, 11:05:02 PM »

Pics in this post. Most are from Vacation this year at Holden Beach, NC

Looks like someone has a drool problem

Right now this is my all time favorite picture. Micah goes HIGH! YAY!

This is a great picture of all of us. Obviously not to hard to get him to smile!

Making him a Reds fan Early!

Prof Underwood · « **Reply #2 on:** August 30, 2011, 11:24:14 PM »

Matt,
This is awesome news! I just checked out your link and am fascinated by the science behind all this, but most of all, as your friend I am just excited for the hope that this gives to you and your family!

God answers prayers

TheJaylor · « **Reply #3 on:** August 30, 2011, 11:30:07 PM »

Wow, great news!

Jeremiah 29:11 "For I know the plans I have for you declares the Lord. Plans to prosper you and not to harm you, plans to give you a hope and a future."

This is the verse that ran across my mind when I read this and it make me think about how the Lord answers prayers!

crustpope · « **Reply #4 on:** August 30, 2011, 11:36:08 PM »

Some pictures are up. @ Mark, the science behind this is brilliant. The "medicine" is delivered via a weekly IV drip and from what I understand, the molecular plasters cover up the damaged section of the DNA allowing the gene to be read with a blank spot instead of a damaged spot. This allowes the protein to be made, even if it is slighly less effective or produced at slightly less amounts.

Either way the end result is that MD patients show little to no muscle deterioration and should be able to walk into their advanced years.

Of course there is little stull understood about any possible long term consequenses so that still has to be considered, but from my prospective anything that allows my son to survive into his adult years is a step in the right direction!

Captain Kirk · « **Reply #5 on:** August 31, 2011, 12:30:51 AM »

Praise the Lord Matt! I (along with many others) have been praying for a miracle. It sounds like this may be that miracle we have all been praying for. I will continue praying!

Kirk

lightningninja · « **Reply #6 on:** August 31, 2011, 01:15:35 AM »

Sometimes God doesn't use a "now you're healed miracle," but a more 'natural' way to solve the problem. I will be praying that this is that miracle for your son, and that the miracles do not end. I pray you get the funding you will need to buy this treatment when it is released (and in faith it will be).

Before you asked, God answers your prayers. Sometimes Gabriel is just a little caught up fighting the devil. But I think he'll make it in time.

uthminister [BR] · « **Reply #7 on:** August 31, 2011, 10:14:41 AM »

Great news Matt! God is good!

Maynid · « **Reply #8 on:** August 31, 2011, 10:19:26 AM »

I was so encouraged and uplifted reading your awesome news. You have a beautiful family! May God continue to keep you in His care.

crustpope · « **Reply #9 on:** October 11, 2012, 10:25:31 PM »

I am Zombifying this thread for good reason...please don't hate me but...if you do..

an-e-way...

The news on this front just keeps getting better and better.

First of all about Micah.

My son is still about 6 months behind the rest of his peers, but he is constantly progressing. He is walking, jumping, talking and interacting like a normal 2 1/2 year old. He is a bit small and short....but so was my dad so may be he got the wrong end of those genes

Here we are this summer...with apologies to YMT...I have THE hot wife;)

Here is Micah with his future wife

Micah and the Markers...probably the name of his future rock band...also a great description of this picture

Finally got a pic with him at our brick down at GABP

This was during our church easter egg hunt...yes our church engages in pagan fertility rituals... -_-

Now for the breakthroughs on the Medical Front!

Here is an article that showed up in the latest "Quest" magazine that describes the success of a drug called "Eteplurisen" The short version is that this drug helps promote the creation of Dystrophin which helps protect muscles from the natural destructive processes in the body. This is an extention of an earlier study that showed those on Eteplursin showed a slowdown in the effects of MD. As the study got longer...the effects of MD were reversed and the subjects actually began to IMPROVE in their 6 minute walk test.

Basically, it seems that Eteplursin not only slows the effects of MD, but it can actually reverse the process and allow patients with MD to re-grow muscle tissue and enable them to improve their condition and that these effects seem to be cumulative.

This is a stage 2 test so the next stage is a longer study with many more human subjects, but the belief is that this is the silver bullet for MD. this drug only works for those with deletions before exon 51 because this treatment allows for the skipping of Exon 51.

For those who dont know about exons and gene therapy, here is a blurb that my wife wrote in an email that helps explain it.

Quote from: Kami Archibald

OK, now for the good stuff. I have been extensively researching several therapies and research ever since Micah’s diagnosis. One of the hot subjects right now is called “exon skipping” therapy. The dystrophin gene is a very large gene sequence, and if you zoom in on that particular gene, you will see 79 parts like links in a chain. These links are called “exons.” Micah is missing link #50, which means the body starts to execute the instructions to produce dystrophin, gets to link #49 in the chain and then quits because the chain is broken. So you say why not just link up #49 to #51 and keep on going? Well, that is the idea behind the therapy, but the problem is that linking #49 to #51 is like trying to put a square peg into a round hole. So the way this works is an “AON”, which is the drug, goes in and finds the closest chain link to hook up, in effect, skipping several exons to re-link the chain. So in Micah’s case, he would need to skip exons 50 and 51 to fit the “round peg” of #49 into the “round hole” of #52. This creates a shorter chain of dystrophin, but a little dystropin is better than none! In fact Dr. Wong said that muscles only need 27% dystrophin production to maintain normal muscle motor function! This therapy has been showing anywhere from 40% to 100% dystrophin production in clinical trials, so I’d say they are doing pretty good!! This therapy has been very encouraging, but so far the clinical trials have only been conducted overseas in the Netherlands and surrounding countries. Matt said we could move to the Netherlands and he would trim tulips and make wooden shoes if that means we would have access to this therapy. But behold! It looks like we won’t have to. Micah is currently too young to participate in the trails, but the good news is that if the trials go well, by the time he will need intervention, this therapy will probably be available to him!!! God is working!!! Yay!

So here is the update! Thanks for listening and praying! I continue to covet these prayers!

soul seeker · « **Reply #10 on:** October 11, 2012, 11:33:57 PM »

Great update! It is good to hear about this exciting news Matt!

Captain Kirk · « **Reply #11 on:** October 12, 2012, 12:19:19 AM »

Wow - praise the LORD! I will continue praying.

Kirk

STAMP · « **Reply #12 on:** October 12, 2012, 04:23:54 PM »

I very much appreciate your sharing!

Prof Underwood · « **Reply #13 on:** October 12, 2012, 04:40:11 PM »

This was a great update Matt. I'm so glad that God has been working in this area. It is an answer to a lot of prayer, and I'm glad that it is the answer we have been hoping for

CJSports · « **Reply #14 on:** October 12, 2012, 08:09:00 PM »

All the more reason to attend the fund-raiser everyone. Unfortunately I have former obligations but I hope all goes well.

YourMathTeacher · « **Reply #15 on:** October 13, 2012, 12:11:37 PM »

Quote from: crustpope on October 11, 2012, 10:25:31 PM

Here we are this summer...with apologies to YMT...I have THE hot wife;)

I will let this comment go for now..... for Micah's sake.

Congratulations on the great news! Keep us updated.

RTSmaniac · « **Reply #16 on:** December 03, 2012, 01:57:27 AM »

You are truly blessed Matt. May God continue to keep his hand upon you and your family. I know it's been awhile since we talked and for that I am sorry.

crustpope · « **Reply #17 on:** July 09, 2013, 12:23:38 PM »

Wow it used to be that you zombified a thread if it was 120 days old, then 90, now it is 14? yowzas!

anyway. I know I have not been active on the boards recently. I have been on a redemption hiatus for a while now but I am really itching to come back and play again. I really miss it and the new cards look really tight. I also wanted to continue to add to the good news on this front.

First of all, the super good news. GlaxoSmithKiline has recieved "Breakthrough" therapy designation which means it is on the fast-track to approval by the FDA. Hopefully it will be fully approved for treatment by the FDA soon. GSK is one of the two companies pursuing this type of treatment and both are before the FDA right now. THe good news is at least one of them is being fast tracked for approval!

In other news we are hosting a 5k Benefit run for the MDA on Saturday September 21st here in Trenton, OH. I know it is a long shot but if anyone is interested in running, or if you know anyone who might be interested you can register online at getmeregistered.com/MicahsMonkeyRun. You can also check out our new website www.MicahsMonkeys.com.

I don't have any cool pictures of Micah to share right now because he is sick and I am busy taking care of him but I will try to get on again soon and post some.

Thanks again for all your support!

Prof Underwood · « **Reply #18 on:** July 09, 2013, 05:33:46 PM »

I'm glad to hear that this treatment has had such positive research findings so far and is on the fast track to approval.

I'm also glad to hear that you're ready for some more Redemption. I'm taking a group down to the EC Regionals this weekend. If you can get to here, I could take you the rest of the way

Otherwise, I know of a group carpooling across northern OH on the way to Nats (if you wanted to hook up with them).

crustpope · « **Reply #19 on:** November 18, 2013, 09:51:02 PM »

Update on the FDA approval for the GSK drug. Evidently there was some problems with their petition and it was put on hold. Furthermore, that caused the FDA to re-think whether they should be using similar data from the other drug company (Sarepta) with a similar drug. Basically, GSK is back to square one and Sarepta looks like they may be headed that way too.

Altogether it has been a real crappy last couple of weeks on the MDA front.

Prof Underwood · « **Reply #20 on:** November 19, 2013, 01:05:22 PM »

I hurt for you guys to receive such disappointing news. I suppose the upside is that it would have been worse if things had progressed further before discovering these problems (ie. a later start on the next generation of research) or even if this had ended up becoming an unsafe drug (ie. that could have been deadly).

I'll pray that things will turn around and that some good news will come your way soon.

crustpope · « **Reply #21 on:** November 19, 2013, 10:58:18 PM »

Quote from: Prof Underwood on November 19, 2013, 01:05:22 PM

I suppose the upside is that it would have been worse if things had progressed further before discovering these problems (ie. a later start on the next generation of research) or even if this had ended up becoming an unsafe drug (ie. that could have been deadly).

This is like kissing your sister, it may be a kiss, but come on, does it really count? I mean, yeah it is good if they are not on the right track, but my wife and I believe it is because they are starting with boys that are already too far advanced and Micah would be a great candidate for this type of drug used in a preventative way.

It had no negative side effects beside a higher protein content to the urine as far as I know so that was not a major concern at all.

Anyway, we are back to being concerned again, and my dreams have taken on a much more morose slant recently. Please pray that there is some breakthrough soon, or that God can give me the strength to endure what I cannot imagine at the present.

uthminister [BR] · « **Reply #22 on:** January 18, 2014, 09:42:28 PM »

Praying!

crustpope · « **Reply #23 on:** May 06, 2014, 09:57:47 PM »

Yes I am zombifying this thread...again. Cause I am just awesome.

Hey all, thanks for praying.....your prayers, and mine, have been answered!

Just recently (last couple of weeks) the FDA has reversed its decision and decided to fast track the approval of Sarepta's Drug, Eteplursen for the treatment of DMD. They have shown continued positive results and...they have realized that perhaps it might be better to give the drug to even younger children so they are starting a clinical trial for a group of boys Micah's age...and if Cicninnati or Columbus is chosen, Micah will be an excellent candidate for the study.

Regardless, if all goes well, the drug should be approved for treatment of DMD this time next year.

A word of caution. While it has shown the ability to improve the function of large muscle groups like Quads and such making it possible for DMD patients to continue walking for longer periods of time, it has not shown to be effective in treating the degradation of the heart muscle. Part of this is the fact that it has only been in trials for 4 years, but the other part is that the heart never rests so that may be part of why it does not seem to be as effective.

Regardless, if this even has a slim chance of extending his life...I'll take it. Thanks so much for praying

This is the link to a forbes article on the drug and its chances of approval and what still needs to be done.

AJ · « **Reply #24 on:** May 07, 2014, 06:18:42 AM »

Great news Crustpope! Me and my family are praying.

News:

Author Topic: Miracles on the Horizon! (Read 50752 times)