It’s been a long three years for Jennie and Gary Landsman.
The Landsmans are parents to Benny and Josh, two young boys ages 4 and 3. The boys suffer from Canavan disease, a rare, life-threatening progressive brain disorder. Most children with Canavan disease die by age ten. Three years ago the Landsmans were told by doctors that there is nothing they can do to save their boys.
“We were told to just go home and love them,” said Jennie.
Though devastated, Jennie mustered up her strength and began researching the disease online. She connected with other families affected by Canavan disease and learned about Dr. Paola Leone, a renowned neuroscientist, and director of the Cell and Gene Therapy Center at Rowan University’s School of Osteopathic Medicine in New Jersey. As one of the top experts on Canavan disease in the world, Leone and her team have been working on developing cutting edge treatments for neurological diseases for over 20 years.
The Landsmans reached out to Dr. Leone who agreed to devote what would become the next 3 years of her life to creating a treatment for Benny and Josh, taking advantage of all the recent advancements in gene therapies.
Their next challenge was figuring out how to come up with the money to pay for the treatments. Insurance companies don’t cover experimental treatments and drug companies don’t invest in drugs for rare diseases that treat such a small percentage of the population.
The Landsmans set out on a mission to raise the necessary funds and save their sons. In November 2017 they began fundraising online and their story went viral. They were filmed for a segment on Good Morning America, featured in People magazine, and had countless articles written sharing their story with the world.
“We were so incredibly grateful,” said Jennie. “So much money came from everyday people, people who don’t know us. They want to save Benny and Josh. It makes me cry. People have so much love to give. It changed my view of the world.”
Despite the big price tag, raising the funds wasn’t their biggest hurdle. Gaining FDA approval for the treatment was the most difficult part of all.
The FDA required that Dr. Leone and the Landsmans produce the drug at the highest purity and at a pharmaceutical grade level. And they needed to conduct and pay for expensive animal and primate studies before the FDA would approve its use.
“They wanted us to do everything at a pharmaceutical level as if we are a pharmaceutical company,” said Jennie, “but we are just a simple family.”
Though the additional expense to satisfy the FDA requirements meant more fundraising, the Landsmans viewed the FDA meeting as a positive one as they knew the FDA would not stand in their way of treating their boys. The Landsmans also insisted that the FDA allow the treatment to be used on other children as well. “There are other families we’ve met, with children waiting for a chance at life, for me it wasn’t a choice, they need to be treated as well,” said Jennie. The FDA indicated that they would allow up to ten children to be treated in this first trial. “I opened this charity (Cure Canavan Fund), and we’re going to keep it going after Benny and Josh hopefully get treated this summer,” she said. “I’m going to see this through to the end until this is a drug that’s available for the public. No parent should ever have to go through what we have.”
Jennie is on a mission to help her boys and all other suffering children across the globe, “I never, ever want another mom to have to go through what I did. I never want a mom to get the news that there’s nothing doctors can do,” she said. “I want parents to be told, ‘There’s something that you could give them, and they’ll be okay.’ That’s what I want.”
Over the past three years the Landsmans have already paid out over $4.5 million to pay for clinical trial costs, research, development, safety testing, manufacturing, and production.
Benny and Josh are now finally scheduled to receive the cutting-edge gene therapy that will help rid them of this devastating disease. It will help them to become like other children, drawing, playing, eating… and give them a more normal life.
“The hope is that they will lead full, functional lives,” said Dr. Leone, who has carried out successful gene therapy trials with mice & large primate animals.
But they still have one last payment deadline lurking, and the Landsmans do not know where the funds will come from.
They still need to raise $1.2 Million. $650,000 is due May 31st, right after Shabvuos, to pay for the last invoice to the drug manufacturer to release the treatment. Then the next month in June they will need an additional $560,000 to pay for the brain surgery to deliver the cutting edge treatment, and the hospitalization costs for the extended hospital stay for each child.
One of the most rewarding aspects of this journey is that Benny Josh and the other children are not the only ones who will benefit from this treatment. If the therapy works as expected, scientists will then apply the gene therapy treatment to other brain disorders such as Alzheimer’s, Parkinson’s, ALS, and Multiple Sclerosis. It will help a much larger part of the population.
“The entire journey has been miraculous,” said Jennie. “Somehow, I really think it was the hand of God,” she says. “We have a long way to go, but we’re hopeful.”
To help Benny and Josh, visit their Chesed Fund page here.
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