Arizona toddler with rare disease gets ‘miracle drug’
PHOENIX – An Arizona boy with a devastating disease received the first dose of an experimental drug Friday after USA TODAY Network The Arizona Republic brought national attention to his family’s struggle to gain access to it.
Denise and Rickey Miller have fought for months for their 22-month-old son, Woodrow, to receive adrabetadex, a “miracle drug” some say allows children with Niemann-Pick Disease Type C to lead longer, more normal lives.
Few children with the extremely rare disorder survive past their teenage years. Over time, it damages memory, hearing and sight, while rendering them unable to walk, speak and swallow. Denise feared Woodrow, without treatment, could die by 3 years old.
Mallinckrodt Pharmaceuticals and the Federal Drug Administration halted new access to adrabetadex in January after a clinical trial showed disappointing results. But families and physicians argued the trial failed to incorporate data that could show the medicine is effective.
As soon as the Millers heard this week a deal had been struck to allow Woodrow access, they jumped on a plane to Chicago, where his physician administered the 15-minute treatment. Afterward, the blue-eyed boy smiled with a toothy grin. “My super heroes wear scrubs,” his T-shirt said.
“It was like a 1,000-pound weight being lifted off our shoulders,” said Denise, 35, a pediatric nurse. “The suffering that Woodrow would have experienced without adrabetadex would have been astronomical. … We have gained so many years with Woodrow that will be spent running and jumping and not hooked to machines and tubes.”
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The newspaper coverage gave hope to parents like Denise who have been lobbying the federal government and pharmaceutical companies for a solution, she said. Members of Congress from Arizona and other states helped keep up the pressure.
“The Republic lit the flame for Woodrow to get access,” Denise said. “It helped immensely. It allowed me to have something to back up what I was telling all these parties was, in fact, true.”
A mother who wouldn’t stop
Born seven weeks premature, Woodrow’s decline has been rapid.
He turned yellow from severe liver dysfunction as an infant until doctors tried adrabetadex intravenously. His liver returned to normal.
Once Woodrow displayed neurological problems, like struggling to walk and talk, physicians recommended a more invasive procedure, adrabetadex by spinal tap.
In preparation, the Millers moved from Utah to Arizona to be closer to a program at Phoenix Children’s Hospital.
But by the time the neurological symptoms cropped up, Mallinckrodt announced it would block access for a handful of new patients like Woodrow and end manufacturing of the drug in October for more than 40 existing patients.
The decision was crushing.
“We uprooted our entire lives for this medication,” Denise said.
Without adrabetadex, Woodrow’s deterioration continued. He began to lose his balance and babble less and less. He started to choke while eating and drinking.
On Mother’s Day, his arms gave out while he was crawling around the house, Denise said. He smashed into the tile floor, leaving him with a bloody face.
“He is suffering when he does not have to suffer,” Denise emailed FDA and Mallinckrodt officials. “What are you trying to accomplish? Are you not a physician yourself and took an oath to do no harm?”
She worked with other parents to raise more than $200,000, hire consultants and raise awareness on Capitol Hill. She contacted other drug companies and posted appeals on social media.
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“It was endless hours of writing and sending emails, late-night Zoom calls, sending the same email over and over again until I finally got a response,” Denise said. “It was because of these efforts that all of the people at FDA and Mallinckrodt knew Woodrow by name and that he needed access and that me, as his mother, would not stop until it happened.”
A different Arizona boy flourishes with the drug
On the other side of the Valley, another boy with Niemann-Pick has thrived on adrabetadex.
Parker Miller, no relation to Woodrow, nearly died of liver dysfunction as a baby, which cleared up with the IV form of the medicine.
When standing on one foot, walking in a straight line and jumping to a certain height became a challenge, doctors added the spinal tap to his twice-monthly hospital visits.
Since then, 4-year-old Parker has flourished, his mother Michelle Miller of Mesa, Ariz., said.
On an April afternoon, he jumped on a trampoline, zipped around in a toy Jeep and jumped off the diving board into the pool with his brother, Roman, shouting and laughing the whole time.
“Dad! I did a cannonball!” he exclaimed.
Parker’s improvements on adrabetadex are real, Michelle said.
“We’re not imagining it. We spend every day with our kids. We see the difference,” Michelle said. “It’s an absolute miracle drug.”
She braced for bad news this week when she saw an email update to parents about adrabetadex. Instead, Michelle learned the drug would be sold to a different pharmaceutical company, Mandos, with plans to continue production for up to two years.
“Instantly it felt like a small group of people can actually make a difference, and that was a good feeling,” Michelle said.
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Mallinckrodt said it halted the drug after a comprehensive analysis of clinical data failed to show clear evidence of benefit for Niemann-Pick patients, but the company acknowledged some parents and physicians have observed positive effects in children treated with adrabetadex.
“We are hopeful Mandos will further advance both the understanding of the disease and the potential to develop the drug as a future treatment option for NPC patients,” Mallinckrodt Chief Scientific Officer Steven Romano said.
The fight for a cure continues
While parents are grateful to the FDA and drug companies for the reprieve, their advocacy is not over.
There is no promise that Mandos will deliver adrabetadex indefinitely, and the drug is not a cure.
Parents say they also hope the FDA rethinks some of its requirements for approving medicines for rare diseases.
“When you have such a small group of people with such a terrible disease like this, you can’t expect the same type of study as when it affects thousands or hundreds of thousands,” Michelle said. “This is a step in the right direction, that a company is willing to take a chance with us. I know (Mandos) won’t be disappointed (in the results of clinical research) if there’s a realistic timeline set and they look at all the data.”
For now, families say they are relieved.
Denise and her husband are “full of joy, hope, happiness, excitement all rolled into one,” she said, because “Woodrow will now be able to grow and thrive and learn.”
The outcome is a reminder of what’s important — and what’s possible, Michelle said.
“It shows what parents can do if they fight for what their kids really need,” she said.
How to support children with Niemann-Pick Type C
- Share posts from the parents’ Facebook group: www.facebook.com/dontgiveuponnpc
- Donate to the parents’ fundraising group, Don’t Give Up On NPC: www.gofundme.com/f/saveourmedicine
- Donate to the National Niemann-Pick Disease Foundation: nnpdf.org
- Donate to the Ara Parseghian Medical Research Fund at Notre Dame: parseghianfund.nd.edu
Follow Rebekah L. Sanders on Twitter: @RebekahLSanders.