He and Jessica Lindqvist had been planning to travel to the United States weeks later with their three children, 5-year-old Hampus and 3-year-old twins Isabella and Julia. But when that call came, they knew they had little choice but to start packing.
Their children all have a rare and fatal disease. Getting to NIH’s Maryland campus offered them their only hope of slowing its progression.
Before the coronavirus caused a pandemic, before cases even started showing up in China, the three siblings had been picked to be among the first participants in a human gene therapy trial for children with GM1 gangliosidosis, a rare disorder that progressively destroys nerve cells in the brain and spinal cord. The trial is the first of its kind, so even the doctors and scientists who have spent 10 years preparing for it can’t say yet whether it will be successful, and if it is, what that success will look like for the children who participate.
But the couple knew what would happen if their children didn’t participate. When Hampus was first diagnosed at the age of 3, doctors told his parents he might not live past his 7th birthday.
“They told us it’s a cruel disease for those around him, but he will develop dementia, so he won’t know he’s sick,” Flysjo says. “They told us to just go home and love your kids and try to make beautiful memories.”
Months later, the couple’s daughters, who were 2 at the time, received the same diagnosis.
“It was as if everything had been taken from us,” Flysjo says.
“Since the diagnosis, you grieve all the time,” Lindqvist says. “We are grieving all the time.”
The couple tell me this over FaceTime on a recent afternoon. A small pink teepee stands in a corner behind them, and a coloring book sits in front of them. Their children are napping.
I asked them to share their story with me, so I could share it with you, because what has happened to the family since that phone call is a testament to parental instincts, the country’s scientific capabilities and the hurdle-jumping power of a last hope. Theirs is not just a story of survival. It is a story of survival amid a pandemic.
Right now, at a time when we are all questioning the strength of our immune systems, all three of their children are taking immunosuppressant medication that has weakened their defenses against the coronavirus. It is a situation that is as nerve-racking as it sounds. It is also not the most stressful one the family has faced lately.
To get into the trial, Flysjo and Lindqvist knew they would have to be among the first to apply when the posting went live on a website. They worried that might happen while they were sleeping, or away from a computer. It happened while Flysjo was at work, walking to the restroom. He says he rushed back to his desk and called the number listed. No one answered, so he started sending emails and then he began calling again.
The three siblings are among seven children who were accepted into the first phase of the trial. Those children will be the first humans to receive the gene therapy, which has previously proved successful in cats.
“I know it’s a trial and it’s not certain to work, but I’ve read through all the research and it’s promising,” Flysjo says. “It’s the only thing we can do for them regarding this disease.”
“We’ve done everything we can,” Lindqvist says.
But before their children could receive that gene therapy, the couple had to get them to Maryland, and they had to do that in the narrow two-day window between when Trump announced he was implementing the ban and when it went into effect.
After the phone call, Flysjo says the family made plans to take a train to Denmark and fly directly to a Washington-area airport. But, he says, hurricane conditions caused the train to stop running.
The family then got tickets flying out of Frankfurt, Germany, the next day, but just hours before they were supposed to leave, they learned that flight path was no longer a possibility.
“You have to find a new way out,” Flysjo recalls being told by the person at NIH who was trying to help the family.
The couple found a flight from London to New York and booked it. The plane was crowded and several passengers coughed from takeoff to landing, but the plane left and arrived on time.
The family got to the airport around 9 p.m. The ban went into effect at midnight.
To get from New York to Maryland, they decided to rent a car. Flysjo says they struggled to find one. They then spent an hour packing their suitcases and crying children into one car, only to learn as they were driving out of the garage that it was due for maintenance. They ended up renting the only other vehicle that was available and could fit three car seats and their luggage: a shuttle van.
Flysjo was driving that van when he pulled into the parking lot of the Children’s Inn at NIH. The nonprofit, which is located on NIH’s Bethesda campus, provides free housing, food and much-needed distractions to families while they participate in clinical trials and studies.
Once in Maryland, the family spent two weeks in isolation as a precaution. The children were then tested twice for the coronavirus before being given the immunosuppressant drugs, which are needed to make their bodies more receptive to the gene therapy.
On Monday, they will get their fourth dose of the drugs.
On Tuesday, they will receive the gene therapy.
“We call it a birthday,” says Cynthia Tifft, a geneticist at NIH’s National Human Genome Research Institute.
On that day, signs are decorated and cakes are cut. It is a celebratory atmosphere, because even if it’s too early to know the result of the trial, there is reason to revel.
The trial offers the families their first chance to do anything about the disease. For the past decade, Tifft has been part of a team that has followed the progression of the disorder in 25 children who participated in a natural history study, knowing they likely wouldn’t get accepted into the gene therapy trial. A requirement of the trial is that a child’s condition not be so far deteriorated that progress can’t be measured.
One child who was accepted into the trial has an older sibling who didn’t qualify for that reason, Tifft says. She describes it as just one of the many difficult decisions that have been made. When the application process opened, she says, they heard from families in South Korea, Australia, China, the Philippines and all across Europe.
I ask Tifft what success would look like for the children who participate, and she says it is too early in the process to know.
“Success can be they are completely cured and they are normal 25-year-olds who go to college and get professional degrees,” she says. “That’s probably unlikely, but to be honest, we don’t know. Now, they are on this downhill trajectory. They could not get any worse. They could get a little better, but not a lot better. They could just get worse more slowly.”
Already there are indications, she says, that the first child who received it is “gaining skills.”
Three children, so far, have received the gene therapy.
Hampus, Isabella and Julia are next in line.
Tifft says now that she has evaluated the siblings, and realizes how long concerns over the coronavirus might last, she feels even more convinced that if the family hadn’t made that trip when they did, the children wouldn’t have qualified for the study.
The siblings don’t say many phrases, but they know the signs for “ice cream” and “cookies.” They also love to dance, and they are one another’s favorite playmates.
More than losing them, Flysjo says, he has been dreading watching them lose one another.
“What happens when they one by one leave each other?” he says. “What happens when one is left behind?”
“That’s the fear,” Lindqvist says.
But a moment later, the couple is speaking about the gene therapy trial and possibilities they’ve never been able to consider before.
All around them, in Maryland and across the country and world, the virus is causing unexpected and excruciating losses. But, at this moment, they are discussing the lives their children might have.
They are talking about their hope that those children might outlive them.
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