DES MOINES, Iowa -- An eastern Iowa mother is running out of options to help save her only child from a rare and fatal brain disease. A cure could be right around the corner, in the hands of a team of doctors from out of state.
Meagan Rockwell had a normal pregnancy but says she knew something was wrong as soon as she gave birth to her daughter, Tobin Grace.
"I had no idea what was going on. She was three months. She hadn`t missed many of her milestones but she kinda had that disconnect. She didn`t really make eye contact. She wasn`t looking at you, she was looking through you," says Rockwell.
After weeks of tests, scans, pokes and prods the bad news came. Tobin was born with what doctors describe as an ultra-rare brain disease called Canavan's disease.
"It's kind of like PacMan. He eats his white dots and gets rid of them. Tobin is missing a PacMan that gets rid of the acids in her brain," Rockwell explains. At almost two-years-old, Tobin can't walk or talk and now the disease is slowly robbing her ability to swallow. " I call her my 28-pound newborn baby because she's that of a baby because she can't support herself at all."
Doctors say Tobin is only expected to live until her 10th birthday. There is currently no treatment, no cure but there is hope. Chicago neurosurgeon Dr. Christopher Jansen thinks he's close to a medical breakthrough. He's spent years researching and developing a gene therapy to treat the disease.
"In the absence of any other treatments, this is a shot of potentially a cure or at least making the kids better, showing signs of improvement both cognitive and motor skills," says Jansen. He believes just one injection of gene therapy into the brain could be enough to reverse or at least slow the progression of the disease but it comes at a cost. Researchers need $3.4 million to treat about 10 kids. So far, through community fundraisers, Tobin's family has raised only $67,000, but they don't have to do it alone.
"This is going to be parent funded from the eight families because no big company wants to sign up to fund this because it could be saving these children's lives but there isn't much money to be made in it," Rockwell says.
Money isn't the family's only concern but whether the therapy will even work. Dr. Jansen is optimistic, "I think this gene therapy does hold the potential to help all these kids and improving their condition...that`s my hope."
No matter the outcome, it's a risk Tobin's family is willing to take in the race against time.
"We really don`t know how much time we have with her and her second birthday is coming up in September and knowing this is another year gone, and another year this disease has progressed. We need to get this going as fast as possible."
Dr. Jansen and his team say it could be as early as the first of the year to begin treating the first child with gene therapy, that's if all money is raised.
To find out how you can help Tobin's family meet their fundraising goal click here or send a check to Grace United Methodist Church in Brooklyn at P.O Box 27 Brooklyn, Iowa 52211. Checks can be made payable to Meagan Rockwell. You can also follow along with Tobin's progress on "Tobin's Journey" Facebook page.