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.
So, trying to save our own family (through this GoFundMe campaign) and the world (through The Firefly Fund) from NPC1 and other neurodegenerative genetic diseases affecting children has honestly become our life's work. Chris and I believe it is our responsibility and our calling to do everything in our power to help these precious children.
The girls are busy with their School, when they are healthy enough and able to go in between treatments. It's been a brutal cold and flu season, which I know every family in Austin has experienced this year due to the unseasonably warm winter and lack of a hard freeze. Belle is also doing 4-5 therapies a week... OT, PT, ST, Vision Therapy as well as working with a reading specialist and she has a full time shadow at school. She is well loved and adored by many that see her throughout each day. And to say she is a trooper is a gross understatement. Abby lives in the moment of each and every day. She does so with curiosity, excitement, love of her big sister and a great deal of humor. She is hysterical and loves to be our family's entertainment.
In case you missed the 20 minute documentary that People magazine published highlighting our families journey with an ultra rare disease, you can watch it here.
Thank you for the continued prayers (!!!), love, positive thoughts, emails, texts, voice mails, and overall support. We could do what we are doing without each and every one of you.
With love, HOPE, and gratitude,
Pam and Chris Andrews
First, we ended the month of February on the 28th, World Rare Disease Day, with a launch of a 501C3 called the Firefly Fund, which was inspired by Belle & Abby's diagnosis and has at its core mission to fund medical research and education to find and fund additional therapies/treatments and eventually a cure for all children with living with NPC1, as well as all neurodegenerative diseases in children. You can learn more about the Firefly Fund at www.firefly.fund. Chris and I are very proud of this labor of love and accomplishment.
Second, People Magazine covered our family in a 4 page article http://people.com/human-interest/familys-fight-to-save-daughters-who-both-have-childhood-alzheimers and accompanying 20 minute documentary http://people.com/pen/00000158-3295-d6e5-a15b-f79d34930000/0000015a-8250-d8c1-ad7a-837325c50000 in the March 7th issue (the Oscar's issue) of the magazine. Our hope and prayer is that the article and documentary will bring awareness to the NPC1 community and help in our race against time to find a cure and additional therapies/treatments for our precious Belle & Abby and all of these children worldwide living with this horrid disease. We hope that the documentary and article help other families struggling to find answers about what is going on with their children and we hope, as well, that they will bring the attention of this disease to the medical community so that we can further advance the science for NPC1. It feels as if a cure is so close, yet so far, since the clock keeps ticking and ticking and our girls are getting older and older day by day.
Third, on March 10th we recognized that we had been living in our new reality with NPC1 for exactly 1 year. It was a tough day (a tough week) and we were flooded with the memories and emotions of the events that took place on that very same day, March 10th, 2016.
Fourth, however, on March 18th we celebrated Belle's 7th birthday with great delight and joy. Belle had a wonderful time and enjoyed having her family, many friends and classmates over to our house for an outdoor celebration with cupcakes and lots and cheer. See some pics attached.
It's been quite a month! While Chris and I wake up every day with determination to find a cure for this disease that is threatening to take our children from us, we do live with a belief that this can actually happen in Belle & Abby's lifetime.
Thank you for following our journey and continuing to keep Belle & Abby and our entire family in your thoughts and prayers. We are humbled by your generosity, love and support.
With love and gratitude,
Pam and Chris Andrews
While our journey has been heartbreaking and painful, there have been many blessings along the way. The biggest blessing in our journey thus far is our awe-inspiring village of people that has come together for our family. Over the course of the last year, many people have said to us “you guys are so brave.” We appreciate the compliment, but to be honest; it has been very easy to be brave. Why? Because we believe we can cure this disease. Why do we believe we can cure this disease? Because we have been standing on the shoulders of giants for the past year.
People like Cindy and Michael Parseghian, who started this fight against NPC1 when there was literally no hope. And, after losing 3 of their 4 children to NPC1, they have continued to advocate and fight to HELP us move the dial and find therapies and a cure for this disease. Our fellow NPC1 parents, whom we have come to love so much; a community that rallied to our aid, all of whom have embraced us with a hug and said “it will be ok.” Many have flown to Austin, to meet the girls. All of these families have become part of our family.
We are thankful for institutions like the NIH and Rush University Medical Center who have embraced us, along with their wonderful team led by Drs. Elizabeth Berry-Kravis and Denny Porter. Our local physicians and therapists, including Dr. Amy White and Dr. Mary Elizabeth Parker, who have given us so much help and hope, always encouraging us to follow our intuition and have, without a doubt, saved our girls from years of unnecessary neurodegeneration.
Our beloved local hospital, Seton Healthcare Family and Dell Children’s Medical Center (DCMC) who have stepped up and lived up to their motto of treating the most vulnerable. We are grateful for the many doctors at DCMC, who under no obligation, have committed themselves to a complex clinical trial for these children, simply because they saw a need.
And of course, The University of Texas Dell Seton Medical School, which is committed to walking “hand-in-hand” and in partnership with us as we aggressively work to find a cure for this disease.
Words cannot express the gratitude we have for
VTesse led by Dr. Ben Machilse. When most people hear of a horrible disease, they assume that there is some company out there working on it. That’s not the case. This is especially true in the rare disease world. We thank our lucky stars every day for VTesse and all of the people on Dr. Machilse’s team.
Our many friends, who have stood by our sides from that first dark day. They came running to help us and they never left. Many have put their own lives on hold to save ours. They have dropped everything to go to Chicago with us. To hold the girls’ hands. To be there. To let us know we are not alone.
Our Family -- our brothers, sisters, aunts, uncles and extended families that have given us unending support and love and of course, our parents… There are no words to describe the support we have received from our parents.
There are too many people to mention and not enough time. But, to us, they are heros. All of them. All of you.
So, why are we so Brave? It is easy to be brave when you are surrounded by heros.
We can cure this disease. We WILL cure this disease. That is what we believe. That is why we created the Firefly Fund. And that is what the Firefly Fund believes and that is what it stands for. This is our tribe. You are our village.
We can’t do this alone. We need each and every one of you. We need your friends and their friends and their friends. And just to be clear, this is not plea for sympathy. This is a call to action. The cure for this horrid disease is within arms-reach. These children, not just our Belle and Abby, but all over the world are counting on us. They are counting on you.
Belle and Abby are now receiving treatment in our hometown of Austin, TX at Dell Children’s Medical Center. These two little girls have been an immense blessing to our lives and we will continue to fight for them, and all of these precious children, until more therapies and a cure for NPC1 is found. We are “all in.”
From the deepest place in our hearts, thank you for the support you have shown our family over the last year. Please continue with your generosity, your love, your support and your prayers. We have come a long way in a short amount of time. But the truth is, we have a very long road ahead of us.
If you haven’t seen People Magazine’s documentary that features our family’s journey with NPC1 this year, you can view it here, www.people.com/andrewsfamily
If you would like to keep up with the Firefly Fund and the amazing things we have planned for these precious children, please visit us often at www.firefly.fund
With love, HOPE and gratitude,
Pam & Chris Andrews
To learn more about the event, visit the newly launched Firefly Fund website at www.firefly.fund!
For more information on sponsorship opportunities contact firstname.lastname@example.org or 512-465-2162.
Tickets & sponsorships can be purchased on Eventbrite - https://www.eventbrite.com/e/firefly-fund-inaugural-event-tickets-30337161266
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
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.
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.
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.
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.