Chicago Tribune / Naperville Sun April 2016

Originally published on Chicago Tribune.com  on April 30th 2016.

New therapy gives hope in children’s fight against fatal Batten disease.

“Cautiously optimistic” is how Gordon and Kristen Kaiser Gray describe the new groundbreaking gene therapy their daughters received in recent weeks.

Charlotte and Gwenyth Gray are the first two children in the world to participate in a clinical trial on humans at Nationwide Children’s Hospital in Ohio to evaluate a new investigative treatment for a rare neurodegenerative disorder known as Batten disease CLN6.

The gene therapy is funded by the Charlotte and Gwenyth Gray Foundation — founded by film producer Gordon Gray and his wife, Kristen, formerly of Naperville — after their two young daughters were diagnosed with the fatal childhood disease last spring.

Although it is difficult to judge how the therapy is working because it has not been tested before, the Naperville North alumna said she already has seen slight improvements in 5-year-old Charlotte’s vocabulary.

“We, my husband and I, have noticed she was more connected, more engaged,” she said. “It’s nice to have her back here with us.”

Kristen Gray said even Charlotte’s therapists have mentioned she appears to be moving better and feels stronger.

“I can’t even put in words how excited we are,” she said.

Because Gwenyth, 2, had yet to show signs of Batten’s effects, the medical team will be closely monitoring her behavior and motor and verbal skills.

It was the sheer tenacity of the Grays that brought this treatment to clinical trials so quickly, said Kristen Gray’s best friend Casey McCormick, of Naperville.

“FDA approval can take years. The hoops they had to jump through — they were relentless,” she said. “It’s amazing what you can do when you don’t take ‘no’ for an answer.”

Inspired to support their friend, McCormick and fellow Naperville residents Bianca Morin and Whitney Robbins created the Friends of the Charlotte and Gwenyth Gray Foundation, which raised $440,000 at a gala in Naperville in the fall.

McCormick, who planned to visit her friend this weekend in Ohio while doctors assess Gwenyth’s recent treatment, said she remains optimistic the motor and verbal skills Charlotte lost will be reversed.

“It is still early to tell. I just hope to God this works,” she said. “I hope this is the tip of a huge iceberg.”

Money raised initially went to researchers like Dr. Jill Weimer, a scientist and director of the Children’s Health Research Center at Sanford Research in South Dakota. Weimer and her team developed an animal model and cell-therapy method to treat Batten disease, and she delivered an update on her Batten disease research this week in Vatican City before the Third International Conference on the Progress of Regenerative Medicine and Its Cultural Impact.

Weimer, who lives in the Naperville area and commutes to Sioux Falls, S.D., said the basic concept of the treatment is to take a virus, strip out all nonessential elements and then load it with the CLN6 gene defective in the Gray girls. “The virus is still able to get into cells — in this case into neurons in the brain — but it is unable to reproduce or cause an immune response,” she said.

Based on the brain-based research through animal trials, Weimer told the Grays that within weeks of the gene therapy, the CLN6 expressed inside of the neurons will start to make them healthier. “This will strengthen the synaptic connections of the cells that were sick and maintain the health of the cells that weren’t sick,” she said.

In addition, the glial cells surrounding and providing support to the neurons will themselves start to get healthier and will continue to work on overdrive to clean up any cellular debris that’s left from the cells that had died, according to Weimer.

Months after the treatment, researchers will be watching for synaptic connections starting to reshape or strengthen to compensate for lost neurons, she said.

Her breakthrough research helped lead to the clinical trials at Nationwide Children’s Hospital.

“This is truly incredible. This is a great example of where researchers from different institutions have come together and collaborated, ignoring the typical timeline to get such work done to give children with CLN6-Batten disease a chance to live,” Dr. David Pearce, president of Sanford Research and a Batten disease expert, said in a statement.

Because the life expectancy for children with Batten disease CLN6 is between 6 and 12 years, Kristen and Gordon Gray — armed with funding from the foundation — pushed to accelerate the pace of efficacy studies and FDA approval of clinical trials at Nationwide Children’s Hospital.

“We are working side by side with the Gray Foundation to make this approach available to other children with Batten disease CLN6,” Nationwide clinical team member Dr. Jerry Mendell said in a statement.

“While this is the first step in making treatment a reality for this condition, we want potential partners in this campaign to know that we will do everything we can to make a difference for this disease, including refining these initial efforts if any symptom of the disease remains unresolved.”

Nationwide is searching for other patients with Batten disease CLN6 for clinical trials — one child this summer and three more before the end of the year. Details about the clinical trial are at www.clinicaltrials.gov.

Kristen Gray said the foundation would provide scholarships to assist families with travel and living expenses.

Since it was founded 10 months ago, the foundation has raised $3.5 million, roughly a third of its $10 million goal.

Kristen Gray said she is grateful to the Naperville community that helped her family reach this important milestone just one year after Charlotte’s diagnosis. She said every donation, social media post and other act of support have carried her family to this point.

“We are so thankful, but we’ve just started,” she said.

Annual fundraisers already are in the planning stages for Naperville and California.

If the treatment proves successful for Batten disease CLN6, Kristen Gray said doctors will expand their methods to other forms of Batten disease.

“What could work for one could work for other forms,” she said.

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