NC Award Public Service SA Mikhail ChrisJess Curran

April 7, 2021

N.C. Awards Committee
North Carolina Department of Natural and Cultural Resources
109 East Jones Street, Suite 315
Raleigh, North Carolina 27601

Re: Nomination of Sheila Mikhail for the 2021 North Carolina Science Award

Dear Awards Committee:

It is with great pleasure that I write this letter of support for Sheila Mikhail, JD, MBA. The amazing impact that she has had on my son, Conner, cannot be overstated.

My name is Jessica Curran. I am a first-grade teacher and a mother of 3 little boys. I also have been blessed with a loving and supportive husband, Chris. I am a woman of faith and I believe that God works in our lives for good - opening our eyes to miracles even in the worst of times. We just have to be patient and trust Him. I have learned so much of this through life’s journey.

I’d like to tell you a little bit about my relationship with Sheila Mikhail. She is a bright star in the biotech world, but she is also someone our family holds very close to our hearts. She is an award-winning, accomplished attorney, turned entrepreneur, who has worked tirelessly to bring dramatic new drugs to the market. What she has achieved is remarkable, but our family knows her in a much more personal way. Her star literally lit up our world and gave us new hope.

Our son Conner was diagnosed at the age of 4 with Duchenne Muscular Dystrophy. It was a heartbreaking diagnosis for our family, and it was devastating for Conner. It brought us to our knees. Duchenne was slowly and meticulously destroying Conner’s muscles. This horrific disease takes every single muscle away. It robs young boys of their childhoods. Duchenne is a progressive muscle disease. Boys typically go into wheelchairs between the ages of 9 and 12. They lose their arm muscles in their teen years; they soon have to be put on ventilators and they need around the clock care. In the end, their heart and lungs give out and this will eventually take their life somewhere in their mid-20s.

Imagine, the prime of your life being the end and watching your son go through that. Some of you reading this may have children and can imagine them running and playing at recess with their friends – smiling and laughing. It was always a different story for Conner. We knew that Conner was sidelined at recess, he was unable to keep up with his peers, but he put on a brave face when he came home every day and say, “Mommy, it’s ok – I watched my friends play.”

Stairs were almost impossible for him to climb. It was hard for him to walk short distances and he would fall quite often. I will never forget the time that he fell outside the Post Office. He was so excited to help me put the mail in the big mailbox. He lost his balance and he hit his head and his face, and he kept saying, “I’m ok Mommy.” I ran over to him and tears were running down my face and he said, “I’m ok” and he wiped my tears and I thought – he is helping me. Conner always had a big glaring bruise on his forehead. When one healed a new one was sure to take its place right away. I knew the progression of this disease was horrible. It just takes and takes and takes from the child. I knew that he would need a wheelchair in the not-too-distant future, and I knew that he would eventually lose the ability to use his arms, to feed himself, and to hug us. And I knew that by his mid-20s, he would not be able to breathe properly, and his precious little heart would give out. No child should have to bear this, and no parent should have to witness this.

Unbeknownst to us at the time, there was tremendous hope in the form of that bright star, Sheila Mikhail, who’s tenacity and drive was instrumental in partnering with Jude Samulski and forming their new therapeutic company, Bamboo. This formation would have such a profound impact on Conner’s life. You see there was a lab with brilliant scientists, and they had something – they had a drug to help boys like Conner, but they didn’t know how to get it to market, they didn’t know how to get it to patients. It is no easy task getting a drug from concept to trials. Sheila made this happen. She has been able to find a way, through her hard work and her dedication and her magic. And this changed everything for so many boys and Conner is one of those boys.

So, after Bamboo sold to Pfizer, something miraculous happened. Conner was chosen as the first patient to be dosed with the mini-dystrophin drug developed by Jude at Bamboo. He would never have received this life-altering drug if it wasn’t for Sheila.

We happily report that Conner is doing well at 3 years post infusion. His stamina and endurance improved considerably. He is able to walk farther than pre-infusion. His North Star Assessment tests improved dramatically during the first 2 years post-infusion -ranging from a 50% improvement to a 100% improvement depending on the test. Most Duchenne patients only experience decline, and we are incredibly grateful to Shelia for providing Conner these years of muscle stability. It has been a blessing for Conner to be able to maintain so much function for such a long period of time. It has not only helped physically but emotionally as well. These past 3 years Conner has been able to interact with our family and his peers without extreme accommodations and we have cherished each memory that he is able to make. Recently, Conner has experienced some leveling off of his abilities, but we remain hopeful because Sheila is hard at work while scientists develop methods that will enable Conner to be redosed with gene therapy. Currently, there is no method to redose without a catastrophic immune response to the AAV but we have faith that Sheila will come through again for Conner.

He is currently 10 years old and in the 4th grade. He is happy and loves school. I’ll never forget one of his first assignments was to write about the most important thing that happened last year. Many students were writing things like getting a new bookbag or getting new jeans; Conner wrote, “I’m proud that I can run faster.”

We are so grateful to this brilliant woman, Sheila, who formed and willed and persevered in getting this amazing drug to trial. We have always considered Conner to be the trailblazer in the Duchenne community, but really, it is Sheila. She is the true trailblazer who continues to lead the fight to end Duchenne and many rare diseases. Our family will be forever grateful for Sheila, for giving Conner his childhood back and a second chance at life.

Sincerely,

/Jessica and Christopher Curran/