Racing against time to save their children’s lives, Niclas and Jessica of Sweden weren’t going to let a worldwide pandemic nor international travel restrictions get in their way. Just one hour before the borders closed to foreign visitors, the couple and their three children – all of whom have a fatal, neurodegenerative disease – cleared immigration in New York to head to their ultimate destination: The Children’s Inn and the National Institutes of Health.
Niclas and Jessica’s world forever changed when they learned their son, Hampus, 5, had GM1 gangliosidosis, an ultrarare, neurodegenerative disease for which no treatment exists. Five months later, the heartbroken family learned their twin girls, Julia and Isabella, 3, also had the disease.
They found hope when Jessica came across an experimental gene therapy online. Getting their children admitted to the study became their single focus. After finally arriving in Bethesda, Maryland, undergoing two weeks of isolation and multiple coronavirus tests, the children were cleared to prepare for the trial.
In late April, the National Human Genome Research Institute (NHGRI) at the NIH Clinical Center administered the experimental IV injection that provided all three children with healthy copies of their damaged gene. The hope is to coax their bodies into producing a crucial enzyme the children lack. If successful, the treatment could halt or possibly even reverse the children’s progressive nerve cell death.
Jessica and Niclas hold their children, from left, Hampus, Julia and Isabella prior to undergoing gene therapy. The family’s remarkable story of determination and hope was recently featured in the Washington Post. Read it here.
A week after receiving gene therapy, the family is back at The Children’s Inn, where they can finally relax. Their children love the indoor playroom, but when the weather is nice, they gravitate to The Inn’s large outdoor playground. They also enjoy the catered meals. But most of all, they love spending time together as a family. Read a thank-you letter, shown below, Niclas and Jessica wrote to you for your unwavering support.
Hi to all of you!
We’re a Swedish family who is staying at The Children’s Inn with our three young children. Hampus is the older brother, and he is turning 6 after the summer; Julia and Isabella are his younger twin sisters, and they are turning 4 in a few weeks.
All of them have an ultrarare genetic disease called GM1 gangliosidosis, which is a fatal disease that affects the brain and nervous system, but we came here to have them all participate in a clinical trial that might stop their disease progression. They all received gene therapy through this trial a few weeks ago, and we are very hopeful this treatment can save their lives. They are all still on immunosuppressants for another six months, so our plan is to stay at The Children’s Inn until it’s safe to travel back home again, considering both the state of their immune systems and the global pandemic.
When we first came here, we had to leave home in a hurry because the borders were suddenly closing down due to the Covid-19 pandemic. Our trip here ended up taking about 30 hours from door to door, and we walked up to the front desk at The Children’s Inn around 6 a.m. on a Saturday morning in the middle of March.
We had seen a few pictures of The Children’s Inn before, but we were struck by how beautiful the building and surroundings are when you see it in person for the first time. We came here the same day as the daily screenings were introduced and the pandemic had already changed a lot of things all around. One thing was that there were not many families staying here at the time, and the activities in the building were kept at a minimum. We are yet to experience The Children’s Inn in its full glory when everything is normal, but still, it’s such a great place for both the children and us parents.
We are very happy how all of the staff takes such a good care of us. To be greeted with a smile and a, ”Good morning,” or a friendly, ”Hello!” can really make a difference when you’re having an otherwise difficult day. Hampus, Julia and Isabella only know a few English word, like ”Hello,” ”Thank You” and “Bye-bye,” but they get very happy when you talk to them. We also appreciate how everyone at The Children’s Inn understands the importance of keeping things clean, so that the patients who are staying here can stay safe from catching other infections or diseases.
The children just love spending time outside at the playground and they have so much fun there. They are also very fascinated by all the different animals that we usually don’t see at home; we haven’t seen this many squirrels or deer before, and they also spotted their first live fox since we came here. Hampus, Julia and Isabella have been so brave through this hole roller coaster ride of a journey.
They’ve grown a lot in these last two months, and now they even dare going down the biggest slide on the playground.
Some people have told us this has been the coldest spring in years, but coming from Sweden, we think the weather suits us and the children just fine right now, and we also get to experience how everything is starting to bloom, which is very nice.
Whenever we need something or the children wants some new things to play with, you seem to find just the right thing for us. We really appreciate all the things you do for us and our children. It helps making living here feel a little bit more like home for us.
Niclas, Jessica and Family
Although nobody can fully predict how exactly treatment outcomes will affect the children’s health, for Niclas and Jessica, the gift of additional time with their children is what matters most. The family thanks you for your support of The Children’s Inn and NIH clinical research for giving them hope for the future.
Your support allows families like this one to stay free of charge at The Children’s Inn so they can focus on what matters most – their children and the medical care they need. Thanks to caring donors like you, our families never have to worry about paying for lodging, food or finding activities to lift their spirits and occupy their time. Thanks for helping make childhood possible today and a treatment or cure possible tomorrow.