Toddler Battling Rare Disease Like Alzheimer's Receives Experimental Treatment
A 19-month-old toddler battling a rare disease that experts akin to Alzheimer's received an experimental treatment at a hospital in Chicago. The family of Marian McGlocklin from Southern California wanted to share their daughter's story in the hopes of raising awareness for Neimann-Pick Type C (NPC).
Doctors diagnosed the toddler's condition after her mom Sara McGlockin noticed Marian failed to meet usual developmental milestones for babies, as per ABC News. Doctors learned the child had an enlarged spleen and further tests confirmed she had the degenerative disorder, which medical experts also call childhood Alzheimer's.
Childhood Alzheimer's affects one in 150,000 births where the baby's body fail to break down cholesterol and lipids, thus causing a build-up in the organs, including the brain. The condition is progressive and affects a child's development mentally and physically. The effects of the disease vary from child to child.
Some children receive NPC diagnosis between six to 10-years-old when they lose the ability to function normally, as per the National Institutes of Health. The life expectancy of a child with Alzheimer's or NPC is under 20-year-old.
Marian is the youngest person to undergo the experimental treatment for NPC under Dr. Elizabeth Berry-Kravis. The doctor is working on a drug, VTS-270, with a limited approval from the U.S. Food and Drug Administration (FDA). According to CBS Chicago, the treatments should prolong Marian's life but it is not yet a cure that will reverse her condition.
The VTS-270 is injected to Marian like an epidural. "The theory is that the most possible will cross the blood-brain barrier and gets into the brain," Sara said.
The experimental treatment needs to be undertaken every two weeks at Chicago's Rush University Medical Center. To help fund travel costs as well as pursue more research for an NPC cure, the family opened a GoFundMe page for Marian.