Saving Belle & Abby
Belle & Abby Andrews
Belle & Abby Andrews are two sisters living in Austin, Texas, diagnosed with Neimann Pick Type C1 (NPC1) in March of 2016. Belle (7yo) is big sister to Abby (2 1/2yo).
In 2014, parents Pam and Chris Andrews noticed their oldest daughter, Belle, was showing some developmental delays in her gross and fine motor skills. Because she was only 3 years old, the delays were subtle and it was hard to know what was normal development and what was cause for concern. But Pam and Chris sensed that something was wrong with their daughter. They sought answers from a multitude of specialists, including speech therapists, vision therapists, physical therapists, neurologists, oncologists, ENTs, nephrologists, endocrinologists, among others. Their search culminated with Drs. Amy White and Mary Elizabeth Parker, who advised them to have Belle’s genome analyzed by Whole Exome Sequencing in order look for potential genetic diseases.
The family was crushed when the test results revealed that 5 year-old Belle had Niemann-Pick Disease Type C1, a genetic disease so rare that Pam and Chris had never even heard of it. There are only 500 diagnosed cases of NPC1 in the world and only 100 in the US.
Shortly after receiving Belle’s results, their geneticist Dr. James Gibson suggested that the Andrews have their two-year-old daughter, Abby, tested. They were devastated again when Dr. Gibson confirmed that Abby also had NPC1.
NPC1 is a progressive, neurodegenerative disease where the lysosomes of all cells of the body accumulate toxic amounts of lipids, causing progressive damage to the brain, spleen, liver, and other tissues. If left untreated, Belle and Abby will likely eventually suffer from seizures, dementia, ataxia, and cataplexy. As the disease progresses, children diagnosed with NPC1 require feeding tubes because of difficulty swallowing, are confined to wheelchairs, are unable to speak or recognize loved ones, and die in adolescence.
Finding hope and living with determination
After pouring over the research and talking with other NPC1 families, the Andrews family found a bit of solace in a growing community of fellow NPC1 families who were able to share their journey. They also learned of a clinical trial being conducted by Vtesse, a rare disease pharmaceutical company, and the National Institute of Health (NIH), to evaluate a drug called VTS-270 for its effectiveness in treating NPC1. There is currently no FDA-approved drug to slow the progression of NPC1.
Through tireless efforts by Pam and Chris, Belle was admitted into the clinical trial and Abby was granted compassionate use of the drug. This meant the family traveled to Chicago every two weeks so the girls could receive treatment, which has taken an emotional, physical and financial toll on the entire family.
Because the Andrews live in Austin, they reached out to everyone they knew who might have connections with Seton Family Healthcare, a part of Assencion Healthcare and Dell Children’s Medical Center (DCMC), a research hospital in Austin they felt would be a prime candidate for helping to administer VTS-270 for the Vtesse trial. Through their vigilant efforts, DCMC heeded the call of these vulnerable children faster than any other hospital participating in this clinical trial in the world, and implemented a VTS-270 trial for all affected children in their region.
In January 2017, just eight months after their initial diagnoses, Belle and Abby began receiving treatments of VTS-270 at DCMC, just a twenty minute drive from home.
In the meantime, the Andrews family continues to hope this clinical trial results in the FDA approval of VTS-270, so their daughters and other afflicted children can begin to get some relief from this insidious disease.
We want to save our daughters and we need your help to do so.
NPC1 is an ultra-rare genetic disease and the medical expenses of caring for two children with such a rare disease have proven to be something unimaginable.
While Belle and Abby receive treatments of VTS-270 at Dell Children's in Austin now, the family still has considerable medical and travel expenses – up to six different therapies a week for Belle, including a full-time shadow at her school, as well as making trips to Chicago for assessments and to the NIH for additional assessments.
While Pam and Chris both continue to work full time, the Andrews family is humbly asking for help. They need any kind of support you can offer. This effort will take a village and they will not be able to do this alone. Pam and Chris are desperate to help save their daughters from this cruel and hideous disease. They are equally determined to help find a cure for this disease and are working tirelessly to help raise awareness and find a cure for this disease.
Thank you in advance for your kindness, your love, your prayers, and your generosity.
A message from Daddy
There are pivot days. Days that change your life. Some days you mourn. Some days you celebrate. Of course, March 10th, 2016 is the day we began this journey. Two years ago today. Another pivot date is February 28th, 2017. That is the day we launched the Firefly Fund. That’s the day we officially started to fight back.
Today, on the anniversary of Belle’s diagnoses, we reaffirm dedication to saving all children, even those yet to be born, from NPC. As long as there is breath in our bodies, Pam and I will fight to end this disease. Nothing. Absolutely nothing will stop us. We will not rely on hope. We will not count on miracles. We will do it. It is that simple. We will do it.
There is one thing that frustrates me. When I tell people our story and tell them about them about our journey and mission in our life and that of The Firefly Fund.…I can see it their eyes. They certainly have sympathy, and that is appreciated. They pray for us, and that means everything to us. But I’m not sure that anyone really believes that we can cure this disease. Why should they? Chances are they have never seen anyone cure a disease. I wish they could feel what I feel. I wish they could know what I know. I wish they could see the world through my eyes.
The truth is we have multiple potential treatments on the horizon. We have brilliant minds working on this disease from around the world. We have dedicated parents, from all over the country, willing to do whatever it takes to save their children. We are funding and doing work that will cure this disease. One or all, or some combination the things we are funding could end this disease, minimally making it a chronic disease. It is not hope. It is not wishful thinking. It is real. It is not someday. It is now.
The hardest challenge is just beginning. The only way our mission will be accomplished is if we can continue to fund it. We, all of us, our tribe, must do whatever we can do. There can be no half-efforts. It’s time to go “all in”.
Pam and I have a dream. Someday a child will be born. A doctor will walk into the hospital room to address two new parents. Then, for the first time in history, She will say, “This might be upsetting but it will be alright. Because of newborn screening, we have discovered that your child has a rare disease called Niemann Pick type C. But don’t worry, We have cure. “That’s the day that we are fighting for. That is the day that when we will exhale... Wherever that hospital room is, we will take planes, trains, and automobiles to get to that child. We will walk into that hospital room, we will hug the parents, we will pick up that child, and kiss him or her on their innocent head. We are fighting for that child, and the many that will come after.
Thank you for your unending support and for staying with us on our journey. We have come a long way in two years and we have a very long way to go.
It's been a while since I’ve made the time to update our wonderful community – our tribe, as my husband likes to call all of YOU. We’ve been busy with school. Busy with treatment at Dell Children’s Medical Center. Busy with Belle’s 7 different therapies every week. Busy with the NPC community and SOAR. Busy with work. Very busy with the foundation, The Firefly Fund. Belle and Abby stay busy living their lives, pushing themselves beyond their physical and various other medical challenges. They are deeply in love with their school and the community that they have built and that lovingly surrounds them at school every day. A place where Belle has been since she was 18 months old. And, with all of the business, Chris and I also stay busy just watching and enjoying them live their life to their fullest. Absorbing and relishing in the joy and purity that each of them bring to our lives. Each. And. Every. Second. Living and breathing in the here and now. Isn’t that all any of us have? The here and the now.
Don’t get me wrong. There are many times throughout each day where I feel like the air I breathe has teeny tiny shards of broken glass in it. There are times when I think about the enormity of what we are facing and my heart stops. There are times I am paralyzed with the disbelief that this is happening to my precious daughters… to my family. There are many times that I wake up in the middle of the night thinking about the expense of caring for two little girls with a progressive neurodegenerative disease and I can’t sleep. There is pain in watching Belle struggle to walk, to talk, to eat and to be one with her community. There is fear of whether or not Abby will have these same struggles. We live every day in the unknown. We are traveling a road much less traveled. But honestly, I have to believe that this is our life’s work. I believe and am clear now that my purpose in this life is to help these children. All of them.
We WILL cure this disease. Not just for Belle and Abby, but for each and every innocent life that has been afflicted by such a horrific condition.
When we are out and about and run in to people we know we are usually asked the same three questions.
1. How are the girls doing?
2. Is the medicine working?
3. How are things going with the foundation?
I can tell you that the girls are doing pretty well, all things considered. They are happy and Chris and I work hard to keep a tight circle of people around them that love and support them unconditionally and without judgment. Belle is struggling to walk. She has a full time aide at school that helps keep her safe from falling. And boy has she become buddies with her aide, Corrine. Abby is asymptomatic for the most part. She is a feisty 3 ½ year old and blazes through each day and is Belle’s biggest fan. Abby loves balls, trucks, trains, dinosaurs and anything she can build with her hands. She loves to hold Belle’s hand when they are walking and she loves to tell Belle “take a deep breath and count to five” when Belle gets frustrated with a task that is difficult for her.
When people ask me if the medicine is working I usually stumble around trying to answer… Belle was much more progressed when she started receiving the VTS-270 than Abby was. So you can’t compare the two, really. I definitely feel as if Belle is stable and we haven’t seen any progression of the disease in Abby. But I want to be clear. The medicine is slowing the progression. It is not a cure. Which leads me to the foundation that was inspired by Belle and Abby’s diagnosis.
The Firefly Fund has become our third child. As with our children, Chris and I pour our hearts and soul in to its success. I’m happy to report that we’ve just launched a drug development campaign called Join the Flight, which you may have seen on social media. – Facebook, Instagram, Twitter and You Tube. If not, please visit our website at www.firefly.fund to learn more about it, AND to JOIN THE FLIGHT! Our goal with this campaign is for every person in our community to donate $10, $20, or $30 dollars a month to The Firefly Fund. Really, what we want is for you to come up with an amount that is invisible to your monthly expenses. I promise you that while it might be invisible to you, it could save the lives of these precious children thought the drug development work the foundation is doing.
You are also welcome to e-mail or call us directly if you want to learn more about the various therapies we are funding through the foundation. We are also working tirelessly and in collaboration with many stakeholders’ to create a path forward for newborn screening for future children born with NPC. My prayer is that no family will ever go through what our family went through on our diagnostic odyssey.
During this holiday season Chris and I hope that you will hold your loved one very close and enjoy being in the here and now.
Thank you for continuing to reach out to us. We remember and hold dear each and every message of love and support. We will be adding a button on the Firefly Fund website that will take you to a blog where I will post updates about how Belle & Abby are doing. So, if you haven’t already, be sure to sign up for updates from the Firefly Fund.
From our family to yours, we hope you have a tremendous and memorable holiday season with your family and friends.
With Love and Gratitude,
Pam & Chris Andrews
Proud parents of Belle & Abby Andrews
It’s funny the stories that stick in your head over the years. The urban legends. Folklore. Greek mythology. One of my favorite stories is the one about the collage philosophy final exam. The story goes that “a group of three hundred college freshman file into a large lecture hall to take their final philosophy exam. The exam is passed out. The students open the exam booklet and find only one question: WHY? "
There was a collective, audible gasp, followed by panic. Then 299 students began to write furiously. They called upon every school of thought they could think of: objectivism, nihilism, Platonism, and so forth.
The answers were poorly structured, circular, and frankly, absurd.
One student, in the front row, wrote nothing. He just sat there, with his arms folded across his chest, staring quizzically at the professor. After about 5 minutes, the student smiled, picked up his pen, wrote a two word answer, turned in this exam and walked out of the lecture hall. That student received the highest grade in the class. His answer: “Why Not?”
Like most children, Belle (7yo) and Abby (3yo) have a favorite question. “Why?” Every parent is familiar with this line of questioning.
“Time to go bed.”
“Because you need your sleep”
“Because sleep is good for you”
What follows in an exhaustive string of “why” questions until the exasperated parent, attempting to put the matter to rest, responds with the age-old, and certainly ineffective, “Because I said so”.
I, thinking myself witty and clever, will sometimes respond to Belle and Abby with the philosophical “why not?” It's my hope that this whimsical reply will act like a flash-bang grenade, temporarily rendering them disoriented, allowing me to make my escape. It doesn’t usually work.
Not all “why” questions are so comical. In March of 2016, I sat with my face buried in my hands, and screamed “WHY?” Why were our girls being taken away from us? What kind of cosmic joke was being played on us? The universe had opened up and God had blessed us with two of the most beautiful little girls I had ever known, showing us the true, deep and unconditional meaning of love. Then, in the next breath, it seemed that the universe conspired to rip them from our arms. Not only would we lose our entire family, but it would be done slowly, in the most brutal and painful way possible. Why? Why my girls? Why us? Why my family? What kind of benevolent God would wish this on any of His children?
Belle has started to ask questions about her condition. Instinctually, she knows something isn’t right, and she has a lot of frustration that she that things are “hard for her.” She asks why we have to go to the hospital every other week for medicine. She asks why does she have to get a shot in her back. She asks why does Abby have to get one too. She asks why she have to do countless therapies every week (PT (2x), OT (3x), Speech (1x), Reading, Vision (1x), etc.). She asks why does she have to wear hearing aids. She asks why she can’t walk and dance and do gymnastics like she used to. She asks why does she have use a wheelchair sometimes. But the most painful question of all... sometimes she’ll ask, why she doesn’t have friends anymore and why she can't see the friends she used to have.
This is the stark reality of our circumstances. Endless expenses trying to save our beautiful daughters for, what many tell us, is a hopeless cause. And one that will no doubt bankrupt our family in the process.
So, why fight for this (supposedly) hopeless cause? My answer is simple. WHY NOT? There is too much at stake.
I will be direct. And I ask that you read this carefully and mark my words. I have said this before and I will continue to say it. We do not live in “hope”. I actually hate the word “hope”. I have asked my wife to stop using the word HOPE. Why? Because “hope” leaves room for doubt. In history, nothing great was ever accomplished by "hoping". We have a different mindset. We live in certainty. We know, without a shadow of a doubt, that WE WILL CURE THIS DISEASE. We are building an army of scientists. doctors, researchers, and patient advocates with one mission in mind... curing NPC. We will not rest. We will not stop. We will give no quarter. We are relentless. We are stalwart. So help me God, we will save all the children that are affiliated with this horrid disease.
Once again, we ask that you spread the word and help us raise awareness. We ask that you join the fight. We ask that you become part of our village, part of our tribe, and we ask you to ask your friends and their friends to do the same. We ask that you share our determination. We ask that you never give up. We ask that you pray for these children, all over the world. And. we ask that you continue to pray for our Belle and Abby.
Pam and I thank you for your continued support.
With love from the deepest place in my heart,
Chris Andrews, Daddy to Belle & Abby Andrews
As we fly home to our babies my head is spinning.... Spinning with excitement to wake them up when we get home and hug them and squeeze them and tell them how much I've missed them. Spinning with so much gratitude to all of the families that have come before us, paving the way and quite literally giving us the hope that we have today because without them we wouldn't have the experimental medication (VTS-270) that Belle & Abby are taking. This I know for sure. My head is spinning with anxiety over how much more work we have to do and it seems the more I understand about this disease and rare diseases in general, the more I understand just how much work needs to be done. My head is spinning with fear... Fear because with each passing day my beloved Belle and Abby are getting one day older and Father-time doesn't slow down for anything or anyone, as we all know. We have to work, hard, fast, smart, and efficiently to get additional therapies, and I pray a CURE, so that Belle & Abby can enjoy the quality of life they deserve and we so desperately want for them, and all of these precious NPC kiddos. Mostly, as I sit here on this plane and look down at the clouds and think about the road ahead for my family, my head is spinning with a lot of love. My heart is full of love for my daughters, love for the good in this world and the long list of good people that work day in and day out to find a cure for this disease. Most of all, love for our large village of supporters -- all of you that have shown our family so much love and grace over this past year. Thank you for the love.
Thank you, too, to Ann Wyatt Little and Fox News 7 for continuing to cover our journey with NPC and the difference we are committed to making for NPC families everywhere.
Hi Pam. Thank you so much for these updates. I think about you and Chris, your girls and your mom so often. I have commented to others on the irony that the work you did as the committee clerk for the House Committee on Public Health was largely responsible for the CHIP legislation that got passed, making health insurance available to more than 600,000 Texas children. only to now have your own children face such a catastrophic need. I just want you to know that I keep you close in my heart with love, and I offer daily prayers for your strength and comfort.
Hello - I am Bea Ann Smith's daughter-in-law and wanted to pass along some information to you, having previously worked for Southwest Airlines. (You may already be aware of this program - and others like it - but just in case.) Southwest Airlines partners with RUSH via a Medical Transportation Grant Program. They provide a certain number of complimentary tickets to the hospitals with whom they partner, who then delegate the tickets, as they see fit. Worth looking into, considering the degree to which your family will need to travel. If you have any questions, do let me know. I am happy to help you find answers. https://www.southwest.com/html/southwest-difference/community-involvement/charities/medical_transportation.html#il You have beautiful, beautiful girls. Warmest Regards, Beth Anne Smith
Good evening. My name is LINDSEY and I own Love Song . Your family has been nominated 14 times for our help. If you are not aware of what I do I will attach some information. Please contact me with any questions. We would love to help your family! Www.facebook.com/lovesongeventsandphotography Our Mission: Impacting lives and raising awareness worldwide through the lens of love. Lindsey prides herself on taking people through a journey with her photography and video diaries. Journeys that allow her clients, their loved ones and the public to remember the smallest details long after the memories fade. The Love Song Foundation is an organization that truly makes an impact in the lives of our clients. Love Song captures priceless moments through the art of photography and storytelling. Documenting many controversial and highly emotional stories and topics makes Love Song unique. We raise awareness by documenting all aspects of life...The good times, the bad times and everything in between. Lindsey and the stories of her clients have been featured in Forbes, CNN, People Magazine, Huffington Post, Yahoo News, The Daily Mail and many, many more. Lindsey's social media following is among the highest for any photographer on Facebook with videos viewed by millions...Bringing awareness and support to causes such as: ALS, childhood and adult cancers, stillbirths, Alzheimer's and numerous others. Love Song caters to the individual and specific needs of each client...No two clients are the same. Love Song is global and has featured stories from around the world. We don’t view our client as just another documentation. We focus on creating personal and lasting relationships. We go above and beyond by documenting a little something extra, granting of wishes, recording a heartbeat or taking a video during someone’s last breath. Love Song...Putting our heart and soul into everything that we do because that is what our clients deserve...Love Song...The Difference. Our goal is to capture significant periods in the lives of our clients. Oftentimes we are meeting clients for the very first time during their most trying or darkest hours- whether it be photographing a still birth, featuring an amazing journey, or capturing someone’s final moments here on earth. We don’t just photograph our clients...We share their stories. We give others a glimpse into our client’s lives when we share their most sacred moments. Love Song assists clients by helping to coordinate fundraising through awareness. From July of 2014 to September 2016 Love Song raised $611,000 for families in need by sharing their stories through social media. Those numbers have continued to rise daily. Often, our stories are featured in the news, through local media and even internationally. “Our client relationship does not stop with a photo shoot, we walk along with them on their journey because it becomes personal. We do our best to grant wishes, put on memorable events, and even help making Christmases and weddings special. In November of 2014 Love Song was able to donate an $86,000 wedding to one of our clients with terminal cancer" Please message us via facebook if you are interested.
You and your husband and those amazing girls of yours are so, so incredible. Thank you so much for continuing to share your story. What a great point, hoping this field is tackled at the new medical school. I would definitely join any effort to make that happen.
Thank you so much for your continued updates! We have yet to meet, but your girls have found their way into my dreams (I dreamt that I picked them up from the airport and drove them to your home, LOL! That's what happens when I read updates before going to sleep...). I do think about your family's fight so often, and am so happy to read that your amazing girls are getting such good care, and will continue to hope (and help make it happen if at all possible??) that the treatments will one day happen in Austin! We are friends of Heather, Darren and Harper, please do let us know if there's anything we can do for y'all, they know how to find us!
Prayers up for these sweet baby girls and to you all as we are praying for the best results and answers in this journey! We will support in any way we can but first and foremost know we love you and think about you all the time. XO and Holla Holla-Meals
Praying for these sweet babies! I have mini strokes daily and have been reading about the use of hemp oil to help the brain continue to function as normally as possible for as long as possible even for Alzheimer patients. Not sure you are interested in the Charlotte's Web (doesn't have as much THC) hemp oil but reading about it wouldn't hurt.
This is the prayer for today in the Divine Mercy novena. I'm praying it over and over for your precious girls.: Here are the prayers for today: Day 6 - The Divine Mercy Novena (Audio/Video Version) "Today bring to Me THE MEEK AND HUMBLE SOULS AND THE SOULS OF LITTLE CHILDREN, and immerse them in My mercy. These souls most closely resemble My Heart. They strengthened Me during My bitter agony. I saw them as earthly Angels, who will keep vigil at My altars. I pour out upon them whole torrents of grace. Only the humble soul is capable of receiving My grace. I favor humble souls with My confidence." Most Merciful Jesus, You yourself have said, "Learn from Me for I am meek and humble of heart." Receive into the abode of Your Most Compassionate Heart all meek and humble souls and the souls of little children. These souls send all heaven into ecstasy and they are the heavenly Father's favorites. They are a sweet-smelling bouquet before the throne of God; God Himself takes delight in their fragrance. These souls have a permanent abode in Your Most Compassionate Heart, O Jesus, and they unceasingly sing out a hymn of love and mercy. Eternal Father, turn Your merciful gaze upon meek souls, upon humble souls, and upon little children who are enfolded in the abode which is the Most Compassionate Heart of Jesus. These souls bear the closest resemblance to Your Son. Their fragrance rises from the earth and reaches Your very throne. Father of mercy and of all goodness, I beg You by the love You bear these souls and by the delight You take in them: Bless the whole world, that all souls together may sing out the praises of Your mercy for endless ages. Amen.
Thanks for the update. I love your message. We continue to pray for you all daily. Much love from the Toomey's!
So sorry to hear that you are facing this terrible challenge. Best of luck for your family and I sincerely hope the treatment is successful. They are beautiful girls.