Life-saving treatment for Cedar Rapids girl receives initial FDA support

CEDAR RAPIDS, Iowa (CBS2/FOX28) — Meagan Rockwell has received amazing news just in time for Mother's Day. She now has learned that her baby girl and only child has a spot in a clinical trial to treat the fatal Canavan Disease.

Donate to Canavan Research Illinois here or Tobin's Time here.

"It's mind-blowing," said Rockwell. "It's what we've been waiting for for so long."

Last December, CBS2 news brought you the story of Tobin Grace Hansen, a now 17-month-old who was born with Canavan Disease. Canavan is a rare, progressive disorder that destroys the brain, limiting a child's simple movement functions, including the ability to hold onto objects or hold her head up. Most children with the disease do not live past age 10.

Tobin's parents, Meagan Rockwell and Heath Hansen had been holding out hope for Tobin's spot on a clinical trial for a gene therapy treatment to treat the disorder, headed by Dr. Paola Leone.

Dr. Leone is the Director of the Cell and Gene Therapy Center at Rowan University School of Osteopathic Medicine and has spent 25 years researching Canavan Disease and developing a gene therapy to treat it. She tells CBS2 news a recent meeting with the FDA to discuss the drug was met with enthusiastic support.

"We walked out of the meeting walking on clouds," said Dr. Leone. She says the new therapy has tested exceedingly well in animals.

"It's designed to replace the gene exactly where it's inactive in the cells that absolutely need it to form white matter," said Dr. Leone. "White matter is the one that is necessary for voluntary movement, for function."

She says the treatment would stop the progression of the disease, and depending on how early a child receives the treatment, he or she may have a typical life span.

Leone says that eight children are currently on the list to receive the drug, including Tobin. Several others are on a waiting list. She says that while the meeting with the FDA went well and they received positive feedback, they still await investigational new drug approval, which can only be granted once the protocol is filed.

Children who are part of the initial trial may be able to receive the drug as early as January, depending on the time it takes to manufacture. The timeline is highly dependent on how much funding they receive, according to Dr. Leone. She wasn't able to put an exact price on the treatment, but all said and done, for each child, she says it will be well over a million dollars.

She emphasized, however, that while donations from foundations and private donors are appreciated, the families are not ultimately responsible for paying for their child's life-saving treatment.

"It is not 'pay-to-play,'" she said.

Still, Meagan Rockwell says she and her family are doing what they can to raise funds. They've seen a huge outpouring of support for Tobin. Friends and family have been active in fundraising through benefits, clothing sales, donation jars, and even rummage sales.

Tax-deductible donations can be made here at Canavan Research Illinois. These donations will help fund all children to eventually receive treatment.

To donate directly to Meagan's fundraiser to help cover family costs associated with travel, appointments, and other clinical expenses, donate here.