'Light is back in his eye': Arizona toddler thrives after mom fights for experimental drug
By Rebekah L. Sanders

Seven months ago, Denise and Rickey Miller's child was declining in front of their eyes.

Woodrow's arms were weakening by the day, collapsing when he tried to crawl. Normally a bubbly kid, he had stopped saying "mama" and "dada." Meal times could be scary; often he would choke.

Today, the giggling 2½-year-old cherub with a rare condition called Niemann-Pick Disease Type C is nearly walking. He's saying new words like his dog's name, "Gadget." And the prospect of a feeding tube is gone.

At least, for now.

"The light is back in his eye," Denise Miller, 36, said at a recent visit to physicians at Phoenix Children's. "It's a night and day difference."

The difference, according to Woodrow's doctors, is an experimental drug known as adrabetadex taken by spinal tap twice a month.

The Millers fought for access to the medicine after a pharmaceutical company announced earlier this year it would phase out adrabetadex for existing patients and deny it to new patients like Woodrow following a disappointing clinical trial.

Parents and physicians across the country decried the decision, saying the drug was a "miracle" for many children and the trial itself was flawed.

Denise, who quit her job as a pediatric nurse to care for Woodrow, spent months calling, emailing and meeting with anyone who could possibly help.

After The Arizona Republic spotlighted the family's pleas, members of Congress questioned the Food and Drug Administration, and a new company agreed to take over the drug. Woodrow started treatment May 21.

Hugging Woodrow tight on her lap, Denise's voice breaks as she reflects on her son's progress since then.

"There’s no word that can describe how it makes me feel," she said. "It’s literally given life back to him."

But the victory is fragile. Parents are wary that decisions by the FDA or the pharmaceutical company at any time could upend the access they have now.

They are continuing to push for permanent solutions.

"The fight will be over when Woodrow has access to all drug therapies used to treat NPC," Denise said. "The fight will be over when those same drug therapies are approved by the FDA."

Uprooting their lives to seek hope

Woodrow has overcome the odds before.

Born seven weeks premature, he weighed about half the average full-term baby and his liver barely functioned.

The diagnosis of Niemann-Pick Disease Type C was devastating. Most children with the disorder, sometimes dubbed "childhood Alzheimer's," die before adulthood. Only a few hundred cases exist in the world.

The Millers uprooted their lives to ensure Woodrow would receive the best care, first flying to St. Louis and Chicago for appointments, then moving from Utah to the West Valley to be closer to Phoenix Children's.

They learned that Woodrow's cells, unable to process important molecules, would deteriorate, particularly in the liver and brain. He would likely struggle to walk, speak and eat while facing challenges with memory, breathing, hearing and sight.

But they also found out about an experimental medicine, adrabetadex, that appeared to help cells in Niemann-Pick patients process molecules like lipids more normally. The drug's main component, cyclodextrin, is used in products as common as air freshener and mayonnaise and even the COVID-19 vaccine.

The results, according to doctors and parents, could be stunning.

A 4-year-old from Mesa with the same disease, Parker Miller (no relation), was born with a liver so damaged his doctors considered a transplant. After several years of treatment, his liver is largely healthy and he runs, swims and jumps on the trampoline with his brother, zooming around the backyard like a rocket.

"We’re not imagining it," his mother, Michelle Parker, said. "It’s an absolute miracle drug."

A rude awakening

Children like Parker gave Woodrow's parents hope for a better life, even if the drug wasn't a cure. And his medical team were determined to help.

Staff at Phoenix Children's navigated federal bureaucracy and an internal research committee to secure approval to administer the medicine to Woodrow, said Dr. Kyrieckos Aleck, a clinical geneticist.

"There are a lot of institutional barriers to getting something like this done. ... (But) our experience with this drug had been very, very good," Aleck recalled. "We said, 'Let’s do this thing.'”

Just as Woodrow's spinal tap infusions were to begin in January 2021, however, Mallinckrodt Pharmaceuticals announced a halt to the drug for new patients and a nine-month phase-out for existing ones.

The company said a clinical trial had demonstrated no evidence of benefit, leaving only negative side effects.

Parents and physicians criticized the trial for excluding younger children, who may see more benefit, and pointed to improvements they saw at home every day.

They also speculated about other reasons for discontinuing the drug: Mallinckrodt, which sold more opioids in the U.S. than any other company during the height of the pill crisis, was consumed with lawsuits and had declared bankruptcy.

The Millers and other families rallied to find a new company to take over adrabetadex and pressed the FDA to support continued access.

They raised more than $200,000 to get the word out, garnered more than 46,000 petition signatures and received attention from "One Tree Hill" actress Jana Kramer.

They also built bipartisan support on Capitol Hill, telling their stories to members such as U.S. Rep. Debbie Lesko, a West Valley Republican, and Democratic U.S. Sens. Mark Kelly and Kyrsten Sinema of Arizona.

Meanwhile, the Millers agonized over Woodrow's decline.

Every morning when Denise went to Woodrow's crib, she worried what abilities he had lost since the day before.

"My child wasn’t going to suffer and lose skills while other kids got to have the drug and live and thrive," she said.

New drug company meets crucial milestones, faces challenges

The families' efforts paid off, with a new pharmaceutical company called Mandos agreeing to take over the drug in May.

Three days later, Woodrow received his first spinal infusion.

"We look forward to engaging with this passionate community of patients, caregivers, clinicians, and regulators over the coming months," company executive Scott Riccio said in a media release. "We care deeply about ensuring access for patients and are committed to supporting efforts to improve the lives of people with NPC1."

So far, Mandos has met crucial milestones.

The company secured approvals to acquire and administer the drug, expanded treatment to 50 patients, solicited feedback from doctors and joined meetings of experts, according to media releases. Mandos' review of trial data is expected to be released early next year.

But challenges remain.

Mandos does not appear yet to have secured a permanent supply of adrabetadex. (The company did not respond to requests for comment.

And parents fear the FDA will require another clinical trial, this time with placebos, randomly choosing children to not receive the drug without their family's knowledge.

Denise calls such an idea "unethical."

"They have completely written off this generation of NPC kids. Even with a placebo-controlled trial, it would take decades to get enough data to prove anything, given (Niemann-Pick's) ultra-rare nature," Denise said. "So in the meantime, kids on the placebo would deteriorate, suffer and die."

An FDA spokesperson declined to comment on whether a placebo-controlled drug trial would be required.

But agency officials have planned a public meeting in January with parents, physicians, pharmaceutical representatives and other stakeholders to discuss how to better evaluate the drug.

"FDA will continue working with the NPC patient community and drug companies to promote and facilitate the development of safe and effective therapies for patients with NPC," agency spokesperson April Grant said. "Patients are at the forefront of everything we do, and FDA remains deeply committed to supporting the NPC patient community to help alleviate the impact and suffering from this serious disease."

'Such a little joy'       

For now, the Millers are soaking up every minute of Woodrow's development, from waving hello to pulling himself up on furniture to getting bright blue glasses.

"He’s getting stronger with each treatment," Denise said.

She believes he would be unable to move and hooked to a feeding tube this Christmas if he hadn't received treatment when he did.

Instead, he's blowing kisses and making his parents laugh.

"He's a jokester," Denise said. "He’s such a little joy."

How to support children with Niemann-Pick C:

• Share posts from the parents' Facebook group: https://www.facebook.com/dontgiveuponnpc

• Sign the parents' petition: https://www.change.org/p/fda-save-our-medicine

• Donate to the parents' fundraising effort: www.gofundme.com/f/saveourmedicine

• Donate to the National Niemann-Pick Disease Foundation: https://nnpdf.org

• Donate to the Ara Parseghian Medical Research Fund at Notre Dame: https://parseghianfund.nd.edu