Denise and Rickey Miller are pleading for help, knowing time could be running out for their 21-month-old son Woodrow, who is diagnosed with a rare disease called Niemann-Pick Disease Type-C.
Denise Miller describes it as a rare disease that is fatal 100 percent of the time.
"There are roughly 100 kids in the United States with this disease, 500 in the world," said Denise.
Some believe the medication to help children like Woodrow is out there, but the pharmaceutical company behind the drug Adrabetadex says there is no proof the drug works, so they will no longer be making it available to patients after October 2021.
The Millers say they've heard amazing stories from families who have used this experimental drug and they've been on a mission to allow their son access to the drug hoping it could help prolong his life.
Woodrow was born seven weeks premature. He has bright blue eyes, a big smile, but a liver that is barely functioning.
Niemann-Pick Disease is likened to a form of childhood Alzheimer's by some. The devastating genetic disease eventually takes away a child's ability to walk, talk, swallow, eat, and breathe, as it weakens their muscles, brain, hearing, sight, and motor skills.
Denise quit her full-time job as a nurse to work full time caring for her son.
"Our daily life right now is consumed with doctor's appointments, therapies, speech therapy, occupational therapy," said Denise.
She added that Niemann-Pick Disease is 100% fatal 100% of the time, but treatments like Adrabetadex have shown to slow down the progression of the disease, allowing children to live longer lives.
Some parents ABC15 spoke with did not think their children would survive past the age of three, but they are now well into their teens and doing well. These parents credited Adrabetadex for their children's good health.
That is why the Millers are so desperate to at least try the drug on their child to see if it will make a difference. Rickey said over the last couple of months they have already seen Woodrow's ability to walk and swallow getting weaker. They're worried that without any medication, statistically, he could live for only about one more year.
"Within that year he will lose every skill, every ability he has and go into a vegetative state until the day he dies," said Denise.
There was no cure for Niemann-Pick Disease Type C. Many families feel Adrabetadex provides them with hope of being able to spend more quality time with their children. Some parents have testified that after starting the drug, their children started doing things they never thought possible, such as running and climbing trees, or being able to read, and put together full sentences.
This hope for many families is soon coming to an end, as the company that has been developing the drug has decided to stop production of Adrabetadex, citing clinical trials that did not show promising results. The company is allowing about 40 children who are already using the drug until October 2021 before cutting off access.
Woodrow is not one of those children. Miller has been writing letters to pharmaceutical company officials, FDA officials, and lawmakers begging to allow Woodrow access to this drug, as his body continues to get weaker.
"It's the most hopeless feeling, knowing that there is a medication literally sitting on a shelf across the country that 40 other children are getting access to, and that Woodrow is being denied access to," said Denise.
In a letter to families, officials with Mallinkrodt expressed regret saying the clinical trials they had been conducting with the drug did not go far enough.
ABC15 reached out Mallinkrodt officials to get more information on these clinical trials. A company official sent us this statement:
"Mallinckrodt deeply acknowledges the disappointment the community, families and patients are experiencing as a result of our decision to discontinue the development of Adrabetadex as a potential treatment for Niemann-Pick Type C (NPC) disease. Over the years, we have come to know many of the families and children impacted by NPC, and like them, we sincerely wish the outcome had been different.
Since acquiring the drug in 2018, Mallinckrodt invested substantially in investigating Adrabetadex as a possible treatment for this ultra-rare and devastating disease. Throughout this process, we have worked closely with the U.S. Food and Drug Administration (FDA) and other regulatory authorities, the National Institutes of Health (NIH), study investigators and NPC experts, seeking their input and guidance.
We are aware that some families and their physicians report seeing benefit in children being treated with Adrabetadex. These reports and case studies were the reason we were hopeful the scientific study would also demonstrate evidence of benefit for patients with NPC. However, after exhaustive analysis of clinical data, there is no clear evidence that Adrabetadex changes or improves disease progression across the group of patients that were studied. An independent data and safety monitoring review board evaluated these data and outcomes and agreed with our assessment. This decision was difficult for Mallinckrodt as we recognize the critical need for new treatment options for NPC and know how hopeful the NPC community was that Adrabetadex would become available for widespread use.
As we announced publicly in January of this year, we will continue to provide Adrabetadex through October 2021 to existing eligible patients already receiving treatment in current studies and expanded access programs. This will allow time for physicians and patients/families to develop a plan for when Adrabetadex is no longer available.
We are deeply grateful to the patients, caregivers, clinicians and patient advocacy groups for their participation and support of the Adrabetadex clinical development program. We remain hopeful that new treatment options for NPC are on the horizon."
ABC15 then asked Mallinkrodt officials why all children diagnosed with Niemann-Pick disease could not have access to the drug until October 2021 if they chose to do so.
In a follow-up statement, a spokesman said:
"When Mallinckrodt announced the determination of the negative risk balance of Adrabetadex in January 2021, we committed to continuing access to the drug for eligible patients for treatment for up to nine months, as these patients transitioned to other potential treatment options and/or care. We consulted with the FDA on the decision to discontinue development while still allowing access to the drug for this additional nine-month period. During this consultation, Mallinckrodt and the FDA agreed to very stringent criteria in recognition of the negative benefit/risk profile for the drug. As part of this criteria, it was agreed that no new patients will be able to start Adrabetadex, and we will not be able to initiate any new company-sponsored clinical studies, EAPs or IIR sites."
For the Millers, these statements are a punch in the gut, as they know their baby boy is running out of time.
"Some days are better than others. Some days he's happy go lucky and other days he goes into staring spells, and he won't respond to his name," said Rickey.
"We need it now, if we have to wait, it will be too late for Woodrow," said Denise.
The family continues to plead with FDA officials and are hopeful lawmakers could intervene and get children like Woodrow access to Adrabetadex.
Despite the results of the clinical trials cited by the company, many parents say their children are proof that this drug works.