Valley child receives first dose of investigational drug that could help prolong his life

Woodrow Miller, a 21 month old child, has now received his first infusion of Adrabetadex, an investigational drug for Niemann-Pick type C disease.

ABC15 has been in contact with Woodrow’s family every day since they left Rush University Medical Center in Chicago last week to begin treatment for Woodrow. His family tell us that Woodrow took his first spinal infusion like a champ.

The investigational drug gives hope to so many families who have children with Niemann-Pick Type C, a rare and fatal disease that ultimately takes away a child’s ability to walk, talk, swallow and breathe. It has been compared to a childhood form of Alzheimer’s disease.

“This drug will prolong her life will give her a better quality of life. There are kids who are in their early twenties who still drive on this drug, and they live alone and they have a job, and they live normally, ”said Denise Miller.

She added that extending the life of a child would give families time to wait, as there was so much research going on with Niemann-Pick disease. It was hoped 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 two weeks for treatment. The Millers said it would take four to five treatments before they began to see changes in their son. They were hoping that treatment could continue at the Phoenix Children’s Hospital in the near future, so that they wouldn’t have to travel so much.

ABC15 also learned that a bipartisan effort between Arizona Congresswoman Debbie Lesko and Arizona State Senators Mark Kelly and Kyrsten Sinema helped Woodrow gain access to this drug.

Senator Mark Kelly’s office today released a joint statement with Senator Sinema and MP Lesko:

“I have had the experience of having to advocate for a loved one facing serious illness,” Senator Mark Kelly said in a statement. “When I spoke with Woodrow’s mom, we talked about the challenges of finding and accessing the best care, as their families are currently doing. No family should have to overcome barriers to ensure that their child has access to the care they need. I’m so glad we were able to get Woodrow’s access to this critical treatment he needs.

“Arizona families like the Millers are in heartbreaking situations and, like all parents, are looking for solutions to help access the treatment their children need,” Senator Kyrsten Sinema said in a statement. “We are pleased that companies and stakeholders like the FDA, at our request, were able to find a solution to ensure the Millers can access life-saving treatment for Woodrow, and we will continue to support Arizona families who request access to similar treatments. vital care. “

“After speaking with Woodrow’s mother, Denise, I sent a letter to the FDA pleading for access to this life-saving drug. I also spoke directly with Health and Human Services Secretary Xavier Becerra about the state of Woodrow and how important it is to him to have access, ”Congresswoman Debbie Lesko said 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 those involved to continue to provide this life-saving treatment to Woodrow and the 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 Food and Drug Administration Commissioner Janet Woodcock below:

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