Meet A Mom Who's Acting FAST to Save Lives

Courtney Davidopoulos is a mom who is part of The FAST Movement committed to help saving the lives of those with Spinal Muscular Atrophy (SMA). FAST stands for Families for Access to Spinal Muscular Atrophy Treatments. She is speaking before the FDA on May 2nd.

Courtney will describe the day-to-day obstacles of living with SMA, and the importance of accelerated approval for SMA treatments. This includes not only the two SMA treatments currently in clinical trials, Ionis’ Nusinersen and AveXis’ AAV9, but also any future SMA treatments that exhibit early efficacy and safety.Courtney is intimately aware of these obstacles, and the need for accelerated access of SMA treatments, because her child, Matthew, is afflicted with the disease. Access to SMA treatments should stop the progression of the deadly disease in children like Matt, and even provide improvement.Courtney is available for an interview before she leaves for the FDA on or before April 30, when she returns from the FDA May 2, or both. Canned footage of the trip to the FDA can be provided for a post story, or a follow up story.

The following is a link to a televised segment.

http://www.whdh.com/story/30021106/wheelchair-bound-football-player-scores-touchdown-for-teamhttp://www.myfoxboston.com/news/westford-pop-warner-helps-matthew-davidopoulos-score-a-touchdown-1/9548413https://www.bostonglobe.com/metro/regionals/north/2013/11/07/team-impact-unites-kids-and-local-college-teams/aVMcP2yIpo8llpi5EalT6L/story.htmlhttp://archive.boston.com/news/local/articles/2011/10/16/to_fully_live_his_life_young_matthew_needs_wheels/http://www.lowellsun.com/news/ci_22880613/were-part-teamhttp://espn.go.com/video/clipTest?id=13660321

The FAST Movement and the Davidopoulos Family has been covered in the media before. The following is a link to an article about the group in The Boston Globe.https://www.bostonglobe.com/business/2015/12/03/biogen-feels-heat-from-families-coping-with-spinal-muscular-atrophy/NLo5Ul8lTZI5ysYTAKLO5I/story.html

The FAST Movement was also indirectly mentioned in an article about the potential of Ionis valuation spiking.

http://seekingalpha.com/article/3924646-ionis-spike-due-breakthrough?li_...

Another in depth story covering The FAST Movement and an SMA child is here. http://www.wndu.com/home/headlines/Three-years-later-Baby-Ella-continues...

The FAST Movement’s web site can be found at TheFastMovement.org

What is The FAST Movement?

Answer: FAST stands for Families for the Acceleration of SMA Treatments. It was started by a core group of moms desperate to access SMA treatments in clinical trials for their children with SMA. Many families throughout the SMA community have joined The FAST Movement’s efforts. FAST presents facts, together with pictures and videos supporting the accelerated approval of SMA treatments by the FDA. The group has a website, as well as Facebook and Twitter accounts.What is Spinal Muscular Atrophy?Answer: SMA is a motor neuron disease like ALS, but it affects babies and children in its deadliest forms. It is the number one genetic killer of babies and children under the age of two. It robs the ability to move, swallow, and eventually breathe. One in forty unknowingly carry the gene responsible for SMA.

Can you tell me about your child with SMA?

Answer: Matthew is a 7 year old boy who loves life. He is very engaging and funny and enjoys all social opportunities. He attends first grade with his typical peers and is at the top of his class in all subjects. He recently celebrated his 7th birthday at a bowling alley with his friends from school and family. His favorite thing is whatever his big brother is doing at the time. Matt has played power soccer, baseball, and recently scored a touchdown on a pop warner football team. Coverage can be seen below.

Describe age at diagnosis & prognosis.

Answer: Matt was diagnosed at 7 months with SMA. The diagnosis was devastating we had never heard of the disease but we heard the doctors say “no treatment, no cure” most children won’t live to see their second birthday and some will never speak, eat or breathe on their own.

Describe what you did to overcome the prognosis.

Answer: We immediately started researching yet it all looked so grim but we eventually met other families and that gave us our first sign of hope. We all got right to work including Matthew and his older brother Noah. He started physical therapy, occupational therapy and aquatherapy. He got started on respiratory equipment that up to this day he only needs at night time to maintain his daytime strength. We made sure he got his power wheelchair as soon as possible to overcome social delays (he was two when he rode away in his first chair). However, one of the most important things we found was proper nutrition which truly made a difference and we had a gtube placed surgically before he lost his swallowing ability to keep his weight on and nutrition ideal. He also is placed in a stander, which does exactly what is implied, as often as possible to help with bone density, circulation, respiration, digestion and more.

Describe a brief daily treatment regime.

Answer: Our regime happens twice a day. Matthew gets put on the Vest, it is designed to mimic chest physical therapy and essentially just shakes him to loosen any mucus build up in his lungs. He also gets a nebulizer treatment like an asthma patient would. Lastly, he uses a cough assist. The weak muscles make a productive cough impossible so a machine with a mask placed on his face does the work for him. Matthew does not mind any of this as he has been doing all of these treatments since he was an infant. If sick with any kind of respiratory illness these protocols happen as often as every two hours and he “gets coughed” as often as needed.

Why are you going to speak before the FDA on May 2nd?

Answer: I’m going to explain to the FDA what it’s like for Matthew to live with SMA on a daily basis. I’m going to highlight some of the problems we encounter, like respiratory illnesses. I want them to understand we fear losing Matthew to a respiratory illness. I’m also going to describe our daily treatment regime to keep Matt healthy. I want to stress that Matt is very happy, and he loves life. I hope to encourage the FDA to consider accelerated access for SMA treatments, to help save the lives of those with SMA like Matthew.

Why is it important for the FDA to grant accelerated approval of SMA treatments?

Answer:The two SMA treatments in clinical trials, Ionis’ Nusinersen and AveXis’ AAV9, trials should effectively stop the progression of SMA in all children. This could also provide improvements. We have seen significant improvements in many of the children in the clinical trials. Both Nusinersen and AAV9 have shown improvements beyond our wildest dreams. However, many of these children were treated at an early age. Still, it’s very important to bring these treatments to the older more affected children as soon as possible. These treatments should improve our children too, but obviously not to the same extent. Our children would still greatly benefit from improved movement and respiratory function. In short, it should give our children a better chance at surviving their fight against SMA.

What is it like for you to see some children receive treatment in clinical trials?

Answer: I’m very happy for the children in the clinical trials. I want to see the deadly effects of this disease stopped in their tracks. To a large extent, it looks like the children in Nusinersen and AAV9 clinical trials will be spared from these deadly effects.However, it’s hard, because Matthew isn’t being treated, and remains afflicted with the deadly effects of SMA. I believe every child’s life is just as valuable as the next, and every child with SMA deserves treatment. I want Matt treated, together with all others with SMA. It’s absolutely heartbreaking for SMA families to know that there are effective treatments out there, and yet they dangle out of our reach.

Courtney Davidopoulos may be reached at cfmccabe@hotmail.com if you have any advice that may help her and families like her, attain accelerated approvial of SMA treatments for her child.