Canavan disease

Jennie Landsman’s 2-year-old son, Benny has always been a laid back little guy.

“He’s a very chill dude,” says Jennie, 34, who owns a yoga and martial arts studio in Brooklyn, New York, where she lives with her family.

He’s so mellow that when he struggled to reach milestones at 6 months that other babies easily met, Jennie figured this was just part of his laid-back personality since he was as social as can be.

“He understood peek-a-boo,” she says. “He would respond with a big smile if you said hello. He just wasn’t sitting up and he wasn’t reaching very well for things.”

But with Benny still unable to sit up, support his head or roll over at 7 and 8 months, Jennie took him to several neurologists before learning in August 2017, that he – and his new baby brother, Josh, who was born a month earlier, suffered from Canavan disease — a rare, progressive and fatal genetic neurological disorder.  Worst of all, the debilitating disorder, which leads to an inability to walk, talk and even swallow, leaves affected children with a lifespan of just 5 to 10 years old.

“Broken is not even enough of a description,” says Jennie about how she felt when a geneticist delivered the horrific news to her and her husband, Gary Landsman, 44.

“It was really dark, really scary, and there were lots of tears,” says Gary, who got the news not long after he opened a new business, Taste Wine Co. in Manhattan’s East Village.

Told by doctors that nothing could be done for the boys except to “go home and love them,” Jennie and Gary at first sank into a deep depression, terrified about the uncertain future ahead.

“Every time I held either one of my babies, every time I rocked them to sleep, I couldn’t stop thinking, ‘How many more times will I hold my baby?’ ” says Jennie. “‘How many more times will I hold their hands?'”

Weeks later, Jennie realized that she and Gary needed to take action themselves. Searching the Internet and asking her growing network of contacts in the Canavan world about treatment options that could somehow help her babies, she hit the jackpot when she found a promising gene therapy that will likely halt – and even reverse the disease’s most damaging symptoms.

The cost? A whopping minimum $1.2 million to allow renowned neuroscientist Dr. Paola Leone, director of the Cell and Gene Therapy Center at Rowan University’s School of Osteopathic Medicine in New Jersey and her team to finish developing the cutting-edge therapy. “The hope is that they will lead full, functional lives,” says Leone, whose gene therapy has already proven successful in mice.

In the potentially life-saving clinical trial, Benny, Josh and other children will undergo a surgical procedure to have a genetically-engineered virus injected into their brains to help them produce normal white matter which they need to regain motor function.

“We have an expectation that the therapy will make a real difference in the lives of these kids,” says renowned neurologist Dr. Christopher Janson of the University of Illinois at Chicago who will direct the team performing the surgeries.

Their state-of-the-art research can also help find a cure for other diseases including ALS, Alzheimer’s and Parkinson’s.

Wanting to do everything they could to give their boys — and other children suffering from Canavan — any chance at life they could, Jennie and Gary set out against all odds to raise the money for the therapy and the clinical trial.

“We had no idea how we were going to do this,” she says.“When you have kids you know you would do anything for (them.)”

So they simply asked for help — in a humble, touching — and heartbreaking video they filmed in their living room – sitting on their couch and holding their boys on their laps – that was shot by their wedding videographer, which they posted on the GoFundMe campaign – called Save Benny and Josh – that they launched in November.

Over the next six months, to come close to their goal by raising $1.15 million with the help of family, friends and strangers alike.

“We are so grateful,” says Jennie, who calls the generosity they were shown a miracle.

“I put up a link and people showed up. It’s unbelievable.”

Besides hundreds of people with huge hearts, she says, “God played a huge role in this.”

Even her 8-year-old son Michael Heiney, from her first marriage, stepped up, reading science fiction books “to help find a cure,” she says.

The family’s fundraising is still not done, though.

Additional costs that have come up – including specialized MRIs the boys will need, plasma, and further studies and testing the FDA is now requesting, have driven the price way up.

To meet these mounting costs, Jennie and Gary have raised their GoFundMe goal to $1.5 million – for now – so that they can cover these costs and help pay for surgeries and MRIs for the other children in the trial. (The money raised by their GoFundMe has already covered the costs for other children to participate in the trial and receive the treatments.)

“We have been given so much that we want to give back, too,” she says.

Adds Gary: “This is part of an ongoing mission that Jennie and I are going to have for the rest of our lives.”

To help Benny and Josh and other children with Canavan, please visit their GoFundMe page.

https://people.com/human-interest/parents-raise-1-1-million-save-boys-fatal-genetic-disease/

Courtesy of my daughter