Even Miracles Take a Little Time

It takes a dedicated team of researchers and several funding sources to get a treatment/cure to human clinical trial. Miracle For Madison and Friends Fund at The Ohio State University has been supporting Dr. Arthur Burghes's SMA research lab since 1998 and is used to pioneer SMA research work that is highly innovative and very risky. This type of research, is not funded by NIH, but when successful and is shown that it works, the researchers can then seek additional funding from NIH or the various SMA organizations to move the research forward.

When we first heard about a novel approach to noninvasive gene delivery in 2006, we became very excited and hopeful about the potential outcome this could have and wanted to help. http://researchnews.osu.edu/archive/newvect.htm -published 2006
The Kaspar Lab at The Research Institute at Nationwide Children's Hospital discovered that a single injection of self complimentary AAV9 vector (scAAV9) with green fluorescent protein (GFP) crossed the blood brain barrier (BBB) in neonatal mice and targeted expression in motor neurons. In regards to SMA, increased expression of the missing survival motor neuron gene (SMN) in lower motor neurons (LMNs) may have therapeutic benefits. Their results suggested that with a single injection, they may be able to effectively restore SMN expression levels in LMNs.
http://www.miracleformadison.org/archive/foustetalnaturebiotech.pdf  -study published 2008

This discovery led to the development and collaboration of the SMA Gene Therapy program that now exists between the Burghes and Kaspar labs. Miracle For Madison and Friends began funding this innovative and risky idea through the Burghes lab at OSU with additional support from NIH to both labs. Dr. Brian Kaspar was invited to our annual fundraiser Madison's Angels at the End Zone in 2007 and  2008 to update families and donors about this groundbreaking research. The Delta 7 SMA mouse model created in the Burghes lab several years earlier was used for the next phase. The SMN1 gene was injected via the scAAV9 viral vector into the SMA mice at one day of age and resulted in complete "rescue" or reversal of the disease SMA. The treated mice showed no signs of SMA, gained weight and lived longer than any other drug or treatment being developed. Testing was also done on a larger and older animal, a non human primate and the "window of time" was extended to show the viral vector could still cross the BBB and target motor neurons giving hope to older patients living with the disease.
http://researchnews.osu.edu/archive/smnrescue.htm -published 2010

The data was quite impressive! The proof of concept was already there. The next 2 years would involve the required safety and toxicology studies needed before FDA would give approval to move on to human clinical trial. Additional funding came in for these studies from Families of SMA, FightSMA, Gwendolyn Strong Foundation and Sophias Cure Foundation and their partners along with the Pepsi Refresh grant which provided the cost for production of sufficient amounts of scAAV9. A project such as this is very expensive and never fully funded. There are always additional questions & other critical studies being done. Miracle For Madison & Friends support extended to both labs in helping with addressing FDA questions in the mice, creating various mouse and pig models of various types of SMA, understanding the complexity of the SMN protein and it's role throughout the body and additional avenues such as intrathecal or CNS delivery.
http://m.hmg.oxfordjournals.org/content/19/20/3895.full

With the preclinical studies now complete that brings this program to step 1 of 3 in gaining approval to move systemic delivery of scAAV9-SMN forward to human clinical trial. On December 4th, 2012, Dr. Jerry Mendell, Dr. Brian Kaspar, Dr. Arthur Burghes and Dr. John Kissel presented their data and proposal to the RAC- Recombinant DNA Advisory Committee at NIH. The RAC was very impressed and gave a positive unanimous decision and approval to move forward with a submission to FDA. Each institution has it's own review board so IRB approval will also need to be obtained before moving into the clinic. This video is the entire Day 1 of the RAC meeting and SMA begins around 98 minutes in from the start. Learn more about SMA and see this amazing data along with personal stories from several incredible SMA families:
http://videocast.nih.gov/summary.asp?Live=12202&bhcp=1

Many more exciting new therapies are being developed in both labs with the hope of moving into the clinic. The goal is to find an effective treatment for those of all ages and types of SMA. As you can see it takes many individuals, families, groups and organizations (big and small) along with the researchers to come together to make a MIRACLE happen. Keep on raising funds and spreading awareness. Continue to click on those links to vote. Share to the world that there is HOPE and someday soon no child will have to suffer loss of mobility or life because of SMA.

We THANK YOU and everyone involved for your part in helping a MIRACLE!
Always BELIEVE! 

                                                 
                                               http://miracleformadison.blogspot.com/

Children's Champions

THIS SUNDAY October 22, 2102, WE NEED YOUR HELP & SUPPORT! 

Would you help if you knew you could save lives? Here is your chance to bring a MIRACLE to LIFE! 

Madison's aunts, cousins and even second cousins are running in the Nationwide Children's Hospital Marathon and they need a BIG PUSH to get to the finish line! They've got inspiration from Madison and all the SMA kids but they need that extra pixie dust to help their efforts save these kids!

Praying we can reach our goal of $10,000! All money goes DIRECT to Dr. Kaspar who is waiting on FDA approval to bring his SMA Gene Replacement Therapy to these children! DONATE NOW: http://www.nationwidechildrens.org.kintera.org/faf/search/searchTeamPart.asp?ievent=1011247&lis=1&kntae1011247=E42D38520DFE4173A3080D1594C8C5E0&supId=0&team=5101628&cj=Y

JOIN THE TEAM! BE A CHAMPION! PLEASE SPREAD THE WORD!

                                     
                                             http://miracleformadison.blogspot.com/

Disney Princess 1/2 Marathon Feb. 2012 UPDATE

To CELEBRATE Madison's Super Miraculous Achievement; turning 15 years old, we traveled to the happiest place on earth- Walt Disney World! Madison had so much fun and didn't want to come back home. Her next big adventure is attending another Big Time Rush concert on July 5th and meeting her favorite boys for the 4th time. Please continue your prayers for her health to remain stable so she can attend this concert.

Madison's Aunt Michelle ran in the Disney Princess Half Marathon while we were there. Please read below Michelle's own account of her training and experience:

"This past Feb. 26 2012, I entered the Disney Princess Half Marathon to Run 4 A Miracle - Cure SMA.  Words can not describe the feeling or feelings I had as I ran and walked 13.1 miles from Epcot to the Magic Kingdom and back! It was truly magical as I felt the wings of So Many Angels carry me to the finish line!

 I trained for 14 months - doing the Galloway beginner half marathon training program I think 3 times and group core and strength training one day a week with my guy Rick at Premier. I mixed up two other days with a light bike workout and kettlebell program.   At first I really did not get the run/walk, so I made up run walks just to get me to 6 miles.  Then by accident or fate - fate for sure- I came across a video of the Galloway run/walk -- ok I got it now!!!  So I finally got comfortable doing the run/walk - 4/1 was my goal.  It finally all clicked into place the last 3 months when 80% of my training runs felt good to great.  You know what they say about the other 20% - they are the ones that either hurt or make you want to just puke and leave that psychic mark in your mind - why am I doing this?

I did no short races like a 5k or even a 10K - my goal was to make this as tough as possible and to be in the unknown -- just like SMA.  Total cold turkey.  My last big run was the Sunday before the race.  We were leaving Tuesday for the two day road trip down.  On Thursday morning I needed to get at least a 30 minute run in and the weather was already up in the 70's.  I walked to the small fitness center at the rental house resort we stayed in - did a 30 minute motor memory run treadmill run - then ran back to the house.  I felt pretty good about things especially since the next three days we would be walking Disney theme parks for a good 2- 3 hours.  I was praying for cool weather for Sunday, because the three days before were in the 70's, and it happened -- it was 55 and cloudy with a touch of misty rain.  Thank you Lord.

Wake up was at 2:30 am  leaving the house with Fran at 3 am.  You had to be at the park no later than 4 am if you were driving because they begin to close the roads leading into Epcot and the Magic Kingdom.  I think all the moons were aligned - we ended up with a sweet parking space, walk to the staging area was short and sweet.  Once there, we checked out a few places especially the CHEAR fan area.  I had to be in my corral no later than 5 am - so I headed on over to it around 4:30 am.  Wow - I've  never seen so many port-a-pottys in my life!  The walk was a good 20 minutes, due to trying to get around those standing in line to take the last pee or poop to the crowd of over 20,000 heading to their corral.

I was in the second to the last corral G.  I found a spot and waited for the big show.  They had a big screen TV so you could hear ( not well ) but see as all the corrals were started by the Fairy Godmother and a round of - yep you guessed it - FIREWORKS.  So as each corral in front of me left I moved up until I was in the start area.  Yep - the Fairy Godmother did the countdown and the Fireworks went off -- and so did my corral.  I started out walking -on the right side like you are suppose to - then I saw the family and friends on the left side so i decided to JOG and look to see if I could see Fran -- no Fran and if she was there I could not see her because it was too dark.  So I was still jogging to the first water station 1.5 mile - goal was to walk all water stations -- got through that one so I began to jog again not really thinking. I just did that to the next water station 1.5 mile again.  I walked through the station again and began to jog a little then looked at my watch and saw it was not 7 am yet ( oh my corral was to leave at 6:21am but I think we left a little later).  I realized I just ran- ok- jogged but I think it was a run cuz I did each mile in like 10 something.  Which I did not plan on so I decided I was going to do my run walk the rest of the way.  As I slowed to walk - I was next to a lady who looked to be by herself so I said hi and how are you doing?  Turns out she was by herself as her friend had a sprained ankle and dropped out.  She was beginning to get a blister on her foot -so i asked her if she wanted any company.  So we walked ran the rest of the race about 8 miles.
My goal was to finish so I was not in a hurry to make some record time and then be dead the next day.  And the time went faster with a new friend. I felt great the whole time - as if i could have JOGGED the whole thing ( which I am glad I did not).  Or even did my run walk - 4/1.

We passed through the Magic Kingdom toll booth heading to the Contemporary then the back way to Main Street USA and the Castle is in front of you.  How cool is that!!! Way cool!!!!  Right turn to Tomorrowland past Space Mountain, The Tea Cups and around the corner to the back of the Castle.  The Trumpeter tooted a royal toot as you ran through the Castle and out the front and down to Liberty Bridge.  Then I saw Jan at the 6 mile area - I gave her a sweaty hug and off to Frontierland.  I was hoping the turkey leg stand was open but no luck there.  Good thing I had one the night before.  We then pass Splash Mountain heading towards the back lot of Adventureland which takes us to the road to the Grand Floridian and back to World Drive.  At the 8 mile mark we get GU -- yummo!!  I milked my three GUs for the next mile.  Next thing you know it we are at mile 10-11 the big clover leaf exit ramp back to Epcot.  WOW what a ramp -- it was banked and it was elevated - the most elevation you can get on the mostly flat course.  Your left buttock cheek was feeling it now.  Then there is mile 12 - Epcot, the golf ball, the water fountain to showcase bridge- then turn around and see it all again.  Now you are getting to the finish line which is again taking us to the back cove of Disney productions ( only where the eyes of cast member are to see) - AND THEN there it is the finish line-1 mile to go!

It took me 3 hours & 24 minutes to do but it was the best 3+ hours of my life!  I  crossed the finish line, got my princess medal, took a photo op with it, walked around and was thinking -- I did it -- after 14 months of training and a 3 hour tour of Disney World - I did it and felt great!!!

I am glad I did no runs before this and I am glad I trained the way I did.  I do not think I would have felt a great accomplishment if I had done a few races before this.  Defeating the unknown is the most rewarding feeling ever. Oh - and staying with keeping it as hard as possible - I did the Princess in shoes I did not train in!! I bought a pair of Brooks two weeks before the race.  However, I tried them on 4 times before buying them. The UNKNOWN!!! Just like SMA!

In doing this run - it has made me want to continue a journey to Run to help Cure SMA.
So because of the Princess Run I am going to BE A CHAMPION and run in The Nationwide Children's Hospital Half Marathon on Oct 21, 2012 -- RUN 4 A MIRACLE CURE SMA.

Remember I did my first ever road race at the Disney Princess Half Marathon Feb. 26 2012. After that unbelievable experience - I have decided to add more to my list - so before the Columbus Half Marathon - I will be doing the Color Run, Dublin Emerald Run, Columbus Ruckus and the Hell Run (going for the SuperWomans Cape).

 If you plan on doing the Nationwide's Children's Columbus Marathon and DO NOT HAVE A CAUSE to run 4 then join my team and RUN 4 Madison's Angels Cure4SMA.
Please DONATE to SMA Gene Therapy Research at Dr. Brian Kaspar's Lab  Madison's Angels Cure4SMA

YOU CAN JOIN MY TEAM- all you have to do is go to Madison's Angels Cure4SMA  and hit the link JOIN MY TEAM. You will raise awareness and funds for SMA Research. You can do this the REAL way by entering the Columbus Marathon and joining me on the streets of downtown Columbus or the VIRTUAL way by running or walking in YOUR city (if not Columbus) or running or walking in your Columbus neighborhood -- mileage of your choice.

Remember SMA or SPINAL MUSCULAR ATROPHY is the #1 genetic killer of children under 2.
7 million Americans or 1:35 people UNKNOWINGLY carry the SMA gene.
SMA is untreatable, under funded, incurable and FATAL.  

Columbus HAS the TOP 2 SMA Research Teams in the world at The Ohio State University and The Research Institute at Nationwide Children's Hospital."- Michelle Worrellia

                                             
                                               http://miracleformadison.blogspot.com/

SMA Awareness Angel Doll & Run4a Miracle

Tattered Rags doll maker Jodi Cain has created this one of a kind handmade SMA Awareness Angel doll in honor of one of our SMA Friends in the Fight- Miss Skyler McAdams. You have an opportunity to WIN this beautiful doll, spread SMA awareness and help fund a MIRACLE! 3 chances to win with every $5 donation. 100% will go towards funding the SMA Gene Replacement Program at Nationwide Children's Research Institute and The Ohio State University.
 Skyler will be drawing the winning name on March 7th.

Make your donation and chance to win here

The Disney Princess 1/2 marathon which takes place on February 26th, 2012 is the kick off for Run4aMiracle. Michelle Worrellia- AKA Madison's Aunt Shell, will set the pace for Team Miracle and release her inner princess when she runs for Madison and all those with SMA.

Please consider making a $15 donation in honor of Madison's 15th Special Miraculous Achievement and help Michelle carry all the SMA Princesses and Princes over the finish line.

Donate here




If you would like to mail in your donation for the raffle or the run please make your check payable to:
Dr. Brian Kaspar SMA Gene Research
and mail to:
 MFM&F
6166 Enke Ct.
Dublin, Ohio 43017

THANK YOU so much to all those who have sent Madison birthday cards, gifts and donations! She has been through a very difficult time this past year with the loss of some movement and weakness. Your support really helps lift her spirits and helps her to keep fighting. Please continue your prayers and support. It's the only way for us to get through this.

"Many nights we pray
With no proof anyone could hear
Though hope is frail
It's hard to kill
Who knows what MIRACLES you can achieve
When you BELIEVE..."
- When You Believe by Whitney Houston

                                                      http://miracleformadison.blogspot.com/

Madison turns 15- Run4aMiracle

15 years ago we were blessed with a beautiful gift from God! 8 months later we found out that this special gift was an Angel and that she would only be with us for a very short time. Our little Angel just turned 15 years old- a Super Miraculous Achievement!

Please help us celebrate and put an end to SMA so that no other family has to experience living with SMA! 

Consider donating at least $15  ($1 for every year of Madison’s life) to Run4a Miracle!

https://giving.nationwidechildrens.org/miracle4madison

We have joined together with several other SMA families and SMA organizations to help accelerate forward the very promising SMA Gene Replacement Therapy at Nationwide Children’s Research Institute. They are right on track to bring this miracle to human clinical trial this year. 
Right here in Columbus, Ohio we have the opportunity to help eradicate SMA! 

HAPPY 15th BIRTHDAY MADISON ROSE REED!

We LOVE you!

http://miracleformadison.blogspot.com/