California toddler being treated for 'childhood Alzheimer's' in Chicago

CHICAGO, Ill. -- A toddler from California is being treated in Chicago for a genetic disorder that can be deadly.

A 19-month-old, Marian McGlocklin, is battling a disease that is often called "childhood Alzheimer's."

Years ago there was nothing that could be done to treat the disease. But now a drug in clinical trials is offering hope to families around the world.

Rush University Medical Center is offering treatment for children not in the trials.

You probably wouldn't notice anything unusual at first about the California girl.

MORE: Family raising funds to find cure for daughter diagnosed with childhood Alzheimer's

Marian is on par with her peers when it comes to language and cognition skills but she isn't walking yet. The reason is a rare genetic disease that affects the brain called Niemann Pick Type C, which some call "childhood Alzheimer's."

It is a fatal disease, but an experimental drug is showing promise in Chicago. Every two weeks, Marian is brought to Chicago for the treatment. Monday was her second visit to Rush University Medical Center.

"I feel really frightened. But extremely, extremely grateful and hopeful," Sara McGlocklin, Marian's mother, said.

A fundraising campaign in Los Angeles is helping her family with travel and medical expenses. Her older sister held a lemonade stand. And there is other support such as the family friend who traveled with Marian and her mom.

"I couldn't imagine not being here. You need all hands on deck," Christine Allen said.

Doctor Elizabeth Berry-Kravis said Marian is the youngest patient treated with VTS-270. She said the drug has slowed the disease's progression in other patients.

"Either we can keep them from progressing long enough that we discover other new things that we can also use or we keep them from progressing for a very long time," Dr. Berry-Kravis said, Rush University Medical Center pediatric neurologist, said.

"I hope that she is going to live a long and healthy and happy life. I just want her to be happy. And I want my girls to have each other as sisters. They love each other so much," McGlocklin said.

In addition to funds raised for the family, colleagues of Sara McGlocklin have donated their vacation days to allow McGlocklin more time to spend with Marian and travel to Chicago appointments.

Eventually, Marian's doctor expects a facility in California will be able to administer the drug.