Four-year-old Willow Hemsley, who has a rare childhood disease, has been on a trial medication that has kept her alive for a year now. The FDA is refusing to approve the medication, causing a life-threatening problem for Hemsley and other children like her.
Hemsley has Niemann-Pick type c, which is a rare inherited disease and a lysosomal storage disorder.
“The body is not able to process lipids, like cholesterol and other fatty lipids,” said Hemsley’s mother, Krystal Samuelson. “These lipids build up inside the body and tissues die. So, a lot of the time it affects the brain, the liver, and the spleen.”
Last year, doctors told Samuelson that her daughter wouldn’t live another year without medication, so Hemsley was given trial medication that comes from Europe.
“The medication works as a heat-shock protein to help the cells be able to break down the fatty substances in Willow’s body,” Samuelson explained.
Samuelson was able to get the trial medication for her daughter in October 2020 through a pharmaceutical company, but because it is not yet FDA approved, the pharmaceutical company will run out of funds to be able to keep producing the medication soon.
Earlier this year, the pharmaceutical company submitted an application to the FDA requesting that the drug, called Arimoclomol be approved, but in June, they received a denial letter.
Since then, the pharmaceutical company has been submitting more data and trying to get the FDA to approve the medication but little progress has been made.
“This trial drug is sustaining Willow’s life and giving her time right now,” said Samuelson. “If that drug goes away, she will lose everything. It’s likely that she will start regressing again and time will be very short. She will pass away.”
Samuelson knew that she couldn’t sit around and wait for possible FDA approval. She had to do something.
Samuelson, along with a group of mothers on Facebook created a petition on change.org, to fight the FDA denial of Arimoclomol and another medication that children with NPC are on, which is in the same position as Arimoclomol with the FDA denial.
“There is another medication that some of the kids with NPC take,” said Samuelson. “They are in the same boat we are in with Willow’s medication.”
It’s hard to get these kinds of medications approved through the FDA, because of the small number of children with NPC and if more placebo groups were requested for testing, children with the disease would die.
“It’s been difficult,” said Samuelson. “Only five percent of all rare diseases have an approved drug.”
Even though things are difficult, the petition has gained over 42,000 signatures and the mothers are going to address Congress soon.
“Here in the next few weeks, we are going to have a briefing with Congress. We want to raise awareness about what NPC is and try to get them to help us in this fight. We pushed hard for this and we have invited 535 Congress leaders,” Samuelson said.
Samuelson is asking members of the community to sign the petition.
“We need as many signatures as possible,” she said. “We are shooting for 50,000 because we don’t want to get our hopes up. The more signatures we can get, the more awareness it’s going to bring.”
The petition is on change.org and can be found by searching “FDA Save Our Medicine” or “NPC.”
A photo on the petition site shows children holding signs that say “FDA please don’t take my medicine. It works. Don’t let us die.”
“All of our children’s lives depend on these medications,” said Samuelson. “If we lose the medicine, we will lose these children.”
To donate to the cause visit www.gofundme.com/saveourmedicine.
To learn more, please visit dontgiveuponnpc.com