What’s Worth Fighting For?

What lengths would you go to save your child?

Anyone that has a child with a serious life-threatening illness understands how valuable time is and anyone that doesn’t can empathize. During my latest challenge with trying to get my son on the trial for the bio-agent Selumetinib, the mek inhibitor that shows huge promise with pediatric brain tumors, I discovered another story in the process. I failed to get Jimmy on the trial, but I learned. It came down to too much red tape and not enough compassion in the world. I won’t give up though, I’ll keep fighting.

I do know everything happens for a reason. I was meant to learn of this little boy named Gabe and his story in the process, so I am grateful for the experience. I always say the cliché, “oh things could always be worse or harder,” and for that little boy named Gabe, he is literally running out of time. This story inspired me so much that I needed to share it with you. Who knows if I will ever be in those shoes, fighting desperately for a drug approval to save my son’s life. I hope not, but if I am, I would hope people would write and inspire as many as they can to help.

Gabe has a rare genetic condition called Duchenne Muscular Dystrophy, which is a genetic condition that leads to complete loss of mobility and premature death. Complications from the associated cardiac and respiratory muscle weakness steal life from children as young as ten years of age. The drug, Eleplirsen, is designed to stop the progression of the condition by inducing the production of a vital protein called dystrophin that helps muscles recover.

Everything about Gabe’s story hits home, mostly because my son is fighting for his life, he has an optic pathway glioma brain tumor. We need to buy as much time as possible as well. As far as time, I have a slight idea of what Gabe’s family is up against. With great emphasize on slight.

Imagine knowing that your son was going to die and there was a drug in trial that has shown huge promise in slowing the illness down, but an ancient dinosaur with big sharp teeth, called the FDA was standing in your way. Why ancient dinosaur? Because the amount of red tape, the important, yet lengthy procedures and the amount of time it takes to get a drug into a desperate, very sick patients hands is beyond ridiculous. That is what Gabe and his family are facing along with the disease itself. I read a quote in one of the articles called “Congress acts to help Alabama family save their son who ‘doesn’t know he’s dying’” from yellowhammernews.com saying that,

“Through it all, the Griffins remain hopeful.” “Gabe doesn’t know that he’s dying, and one day I’m going to have to tell him that,” his father said. “I pray everyday that that day doesn’t come.”

One can argue FDA regulations and procedures for days, or how important protocols are, but until you’re holding your little boy in your arms and knowing there is a drug that can help him and you may not have the “power” to get it for him, that’s when you’ll truly understand what’s worth fighting for.

I emailed Dr. Janet Woodcock, the director of the FDA encouraging her to “open the floodgates for Duchenne” and allow the drug Eleplirsen to get full approval through the FDA’s Expedited Programs for Serious Conditions -Drugs and Biologics. If you were interested in encouraging her as well, her email is listed here http://directory.psc.gov/hhsdir/eeKey.asp?Key=30511&Format=Table. Every word, every email helps. Who knows, something you say could inspire her.

I’ve included links below if you want to learn more about Gabe and the giant strides his family has made in the state of Alabama. Including “Gabe’s Right to Try Law.”

This family is making progress and paving way for all families that are interested in advancing promising therapies to terminally ill patients with rare conditions. His dad bicycled across the United States to build awareness, what an incredible father. http://www.ride4gabe.com/

This story is something to make note of. It’s inspiration in its purest form.

Thank you Griffin Family you are making a difference, I noticed and I am grateful to all of you.

This is what is worth fighting for!

http://governor.alabama.gov/newsroom/2015/06/governor-bentley-signs-gabes-right-try-act-law/

http://www.washingtontimes.com/news/2015/jun/7/family-behind-new-ala-right-to-try-looks-beyond-th/?page=all

http://birminghammedicalnews.blogspot.com/2016/05/first-disease-modifying-treatment.html

http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf

http://media.wix.com/ugd/653359_27d7b196c26042e3b3496ea4be574277.pdf

http://www.bizjournals.com/boston/blog/bioflash/2016/04/fda-chief-lays-out-rationale-for-duchenne-drug-to.html

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  • Thank you for sharing. Gabes Mom is one of my first childhood friends. They’re doing amazing work to support/fight for/share information on this horrible disease. Thank you, too, for your helpful in doing so. Bless you and your family, too, with your son. Sincerely, kelly

  • Lacie – I am Gabe’s dad and your article touched our heart! It means the world to me to hear that we have impacted you. This came back up on my Facebook news feed and I realized I did not reply earlier. As a parent we are responsible for “fixing” all of the bad things that happen to our children. If one of my 4 children fall down and cut their leg I bandage and kiss it and make it better. I cannot make this better by myself. When your child is dying you will do ANYTHING for them and you pray everyone you come across takes this story to heart and helps in some little way. As a result of your post, many folks have reached out to us to help. They have informed me of letters, emails, and phone calls made to the FDA asking them to approve the drug Eteplirsen and all of this is a direct result of your post. Please know that YOU are making a difference not only in your own family’s life but in our life as well. Yesterday evening it was announced that Dr. Farkas who was with the FDA is no longer with the agency. He was the main FDA panel member that was blocking the approval of the drug. The stock of the company soared this morning with this announcement yesterday and my hope is this is a sign that approval is coming very soon. I wanted to give you an update and to let you know that like you, as long as there is breath in my lungs I will continue to fight for my son…I just pray we win the fight! May God Bless you and your family and thank you for your post!