The Middletown Press (Middletown, CT)
‘An exciting time for us’
Ridgefield boy with genetic condition may get therapy to prolong his life
RIDGEFIELD — Jessica Curran said she recalls the day eight years ago, sitting in a doctor’s office, when her entire life came crashing down.
That was the moment she and her husband, Christopher Curran, were told their son, Conner Curran, now 12, has Duchenne muscular dystrophy, or DMD.
The genetic condition affects mostly young boys and is marked by the progressive loss of muscle as the body fails to make dystrophin, a muscle-building protein. It also deteriorates the skeletal, cardiac and pulmonary muscles.
“They handed me a thick packet. They said, ‘Take him home. Love him. There’s nothing you can do. Enjoy him while you can. And there’s no treatment,’ ” said Jessica Curran, 46, of Ridgefield.
The Currans haven’t given up hope for their son. Through their charity, Kindness Over Muscular Dystrophy, nearly $2 million has been raised for research into DMD.
Today, Conner is exceeding the limitations nearly all DMD patients of his age, due to a gene therapy he received five years ago as part of an ongoing clinical trial.
Many children with DMD begin using a wheelchair by age 12, according to the Muscular Dystrophy Association.
“Here’s a miracle. Conner is almost 13 and he’s still walking. That is unheard of,” said Christopher Curran.
But time is running out and the effects of the gene therapy are beginning to fade, he said.
Conner has been selected for a new gene trial that will determine if he’s eligible to receive the gene therapy again — which could give him more years of mobility.
“This is an exciting time for us,” said Christopher Curran, 43.
Dr. Jude Samulski, a professor at the University of North Carolina in the department of pharmacology, created the drug Conner used in the first trial.
“(With the prior gene therapy dose), we bought Conner time so that he could be a candidate for the next wave of the gene therapy technology,” Samulski said.
Conner said he hopes that gene therapy gets approved for wider use “so
that all kids with Duchenne can get help like I did. I hope to be able to get the gene therapy again one day.”
Boys with DMD often did not live much beyond their teen years, but today patients have survived into their 30s thanks to advances in care, according to the Muscular Dystrophy Association.
Gene trials
In 2018, Conner, now a sixth grader at Scotts Ridge Middle School, became the first patient in the gene therapy trial offered by Pfizer for boys with DMD.
His family saw results within two months.
“It was incredible,” said Christopher Curran, adding Conner greatly improved his walking ability and speed.
“The therapy bought Conner five years of life,” said Christopher Curran, who works in the maintenance department for the town of Lewisboro, New York. Jessica Curran teaches first grade for the Carmel, New York, Central School District. Conner has a twin brother, Kyle, and a younger brother, William, 10.
Since Conner already received the gene therapy, however, he can’t be given another dose — at least, not at the moment.
“Because it’s a virus, it’s a one-time treatment. If (doctors) tried to give it to Conner again, his body would have a massive immune response and would attack it,” Christopher Curran said. “But we’re working to change that.”
In a new trial, beginning most likely early next year, Conner’s doctors will see if they can lower his antibodies to the gene therapy, so he can safely get another dose.
At the beginning of
2024, Conner will participate in the trial at the University of Florida in Gainesville, along with about a dozen other children with DMD.
“(Conner) is actually an ongoing experiment. And as a result, it’s clear that Conner and others boys like him will need a second dose as they continue to grow and develop new muscle,” Samulski said.
Scientists from multiple drug companies are on the cusp of finding a treatment to slow the degenerative effects of Duchenne.
Last week, the first Food and Drug Administration Advisory Committee was expected to discuss one such type of treatment.
An approval would be for kids who can walk and haven’t received the therapy, Christoper Curran said. “Hopefully, this is FDA approved so that every kid can get that (extra) five years that Conner got,” he said.
Spreading awareness
Conner was selected for the original gene trial due to “persistence,” Christopher Curran said.
“It was persistence and nonstop calling and begging and pleading and going to all the gene therapy presentations at conferences,
and meeting all the top docs and scientists and Ph.D.s,” he said.
To help spread awareness of DMD, the family forged relationships with state leaders, including U.S. Rep. Jim Himes, D-4, and U.S. Sens. Richard Blumenthal and Chris Murphy, D-Conn.
“The most important point for me has been the superhuman courage, resilience and resolve of this extraordinary family — most specially Conner,” Blumenthal told Hearst Connecticut Media.
Additionally, the Ridgefield community has been supportive of the Currans in their efforts to spread awareness of Conner’s fight, Christopher Curran said.
Conner was made honorary captain of the Ridgefield Lacrosse Team, and Ridgefield residents have attended and contributed to many fundraising events of the Currans’ foundation.
“The Ridgefield community has buoyed us and lifted us up,” Christopher Curran said. “The most important thing to us is to ultimately to save our son’s life and awareness can help us get there,” Christopher Curran said. “This is our life’s mission.”