Woodrow Miller, a 21-month-old toddler, has now received his first infusion of Adrabetadex, an experimental medication for Niemann-Pick Type C disease.
ABC15 has been in touch with Woodrow's family every day since they left for Chicago's Rush University Medical Center last week, to start Woodrow's treatment. His family tells us Woodrow took his first spinal infusion like a champ.
The experimental medication gives hope to so many families who have children suffering from Niemann-Pick Type C, a rare and fatal disease that eventually takes away a child's ability to walk, talk, swallow, and breathe. It has been likened to a childhood form of Alzheimer's.
"This medication is going to prolong his life is going to give him a longer quality of life. There's kids that are in their early 20s that are still driving on this medication, and they live on their own and they have jobs, and they live normally," said Denise Miller.
She added that prolonging a child's life would give families time to wait, as there was so much research being done on Niemann-Pick disease. There was hope that new treatments could help cure or at the very least help children manage the disease better as science progressed, thus giving families more quality time with their children.
Woodrow will have to travel to Chicago every other week to get treatment. The Miller's said it could take four to five treatments before they started seeing any changes in their son. They were hopeful the treatment could continue at Phoenix Children's Hospital in the near future, so they would not have to travel so much.
ABC15 has also learned that a bi-partisan effort between Arizona Congresswoman Debbie Lesko, and Arizona state Senators Mark Kelly and Kyrsten Sinema helped Woodrow get access to this drug.
Senator Mark Kelly's office released a joint statement with Senator Sinema and Congresswoman Lesko today:
“I've lived through the experience of having to be an advocate for a loved one facing a serious medical condition,” said Senator Mark Kelly in a statement. “When I spoke with Woodrow’s mom, we talked about the challenges of searching for and accessing the best care just as their family is doing now. No family should have to jump through hoops to get their child access to the care they need. I’m so glad that we were able to get Woodrow access to this critical treatment he needs.”
“Arizona families like the Millers are in heart-breaking situations, and like any parents, are seeking solutions to help access treatments their child needs,” said Senator Kyrsten Sinema in a statement. “We are pleased the companies and stakeholders like the FDA, at our urging, were able to find a solution to ensure that the Millers will be able to access lifesaving treatment for Woodrow, and we’ll continue supporting Arizona families asking for access to similar lifesaving care.”
“After speaking with Woodrow’s mother, Denise, I sent a letter to the FDA advocating for access to this lifesaving drug. I also spoke directly with Secretary of Health and Human Services Xavier Becerra about Woodrow’s condition and the importance of him gaining access,” said Congresswoman Debbie Lesko in a statement. “While I am so encouraged that Woodrow is currently receiving this treatment, the fight is not over. I will continue to do what I can to encourage the parties involved to continue providing this lifesaving treatment to Woodrow and other children affected by this horrible disease.”
Read the full letter sent by U.S. Senators Mark Kelly and Kyrsten Sinema and Congresswoman Debbie Lesko to Acting Commissioner of the Food and Drug Administration Janet Woodcock below: