Experimental Drug By Chicago Dr. Helps Girl With Childhood Alzheimer's

CHICAGO (CBS) -- The parents of a two year old, diagnosed with a childhood Alzheimer's disease have found hope from an experimental drug, offered to them by a doctor here in Chicago.

Sara McGlocklin's first indication that something was wrong with her daughter came at "Mommy and Me" classes.  Marian wouldn't hold her head up like the other babies did. A pediatrician suggested they see a neurologist. An ultrasound confirmed she had an enlarged spleen and liver, classics signs of a storage disorder. Mirian was identified as Niemann-Pick Type C.  It's is believed to be fatal.

By 18 months of age, McGlocklin says Marian was already starting to lose the little memory she had.

"When we got the diagnosis it was so shocking and devastating. It was like falling through the floor," said McGlocklin.

Marian, diagnosed at age one, is among the youngest to get it, but she's a fighter.

For six months now, Marian's been receiving a drug called VTS-270, giving her family hope. It was given to her every two weeks by a doctor in Chicago.

Now Mirian is walking and talking, learning new words every day, and this weekend, she's back in Chicago, for her six month evaluation.  She turned two in August.

"At 18 months, she couldn't even take a step. She couldn't even stand up without holding onto something," said McGlocklin. "And now she can walk, she can walk fast. She's talking a lot more, and every day she's saying a new word," said McGlocklin.

The family's asking listeners to take the NoPuckerChallenge. To bite a lemon and post it on social media using the hashtag NoPuckerChallenge, donating $10 to find a cure for NPC.