Jun 21, 2021
patient story
gene therapy provides hope
Benny and Josh Landsman
In a year filled with uncertainty, hope lives on at Dayton Children’s for the Landsman family from Brooklyn, New York. For the past three and a half years, Jennie and Gary Landsman have been on a non-stop mission to save their two sons, Benny, 4 and Josh, 3 from Canavan disease, a rare and fatal neurological disease.
When Benny was 6-months-old Jennie and Gary noticed that he wasn’t hitting the developmental milestones typical for his age. For example, he was having a hard time sitting up on his own. Until that point, Benny was hitting milestones like any other baby. At 7-months-old, Jennie took Benny to a neurologist for an evaluation. The neurologist didn’t have major concerns with Benny’s development but recommended he receive physical and occupational therapy. At 8-months, he still wasn’t rolling over, sitting up or attempting to crawl. He hadn’t reached any new milestones since he turned 6-months-old.
After seven months of physical and occupational therapy, and second opinions from multiple specialists, Benny was referred to have genetic testing done.
At Benny’s genetic appointment, Jennie also had 2-week-old Josh with her. The genetic specialist looked at Josh and suggested both boys have the testing done. Two weeks later, on August 14, 2017, Benny and Josh were diagnosed with Canavan disease.
Life after diagnosis
Canavan disease is an inherited, fatal, neurological disease, characterized by the spongy degeneration of the white matter in the brain, which begins at infancy and destroys a child’s vision, speech and motor function. Currently, there is no cure for Canavan disease.
The doctors told Jennie and Gary that there is nothing they could do but go home and make the boys comfortable. After letting the diagnosis of Canavan disease settle in for a few weeks, Jennie and Gary began researching and connecting with other families.
One thing they learned was that the milestones Benny had met prior to 6-months of age, he wasn’t going to lose. This information gave Jennie and Gary the drive to help Josh develop milestones more quickly so he would be able to keep them once the degeneration started.
Through the process of connecting with other families, the Landsmans found Paola Leone, PhD. Dr. Paola Leone is a leading researcher of Canavan disease from The Canavan Research Foundation. Dr. Leone has been a longtime Canavan researcher and conducted two previous clinical trials in 1998 and 2001. In 2001, Christopher Janson, MD from Premier Health’s Clinical Neuroscience Institute collaborated with Dr. Leone on a clinical trial for the disease. While their 2001 clinical trial provided patients with fewer seizures and some benefit, they discovered that targeting the cells that promote white matter was more beneficial than targeting gray matter.
Gene therapy offers hope
By 2017, Dr. Leone and Dr. Janson had developed a new gene therapy using a modified recombinant Adeno-Associated – Viral Vector. This lab-produced gene targets the cells that are significant for white-matter development.
Around the same time the new gene therapy was developed, Jennie reached out to Dr. Leone for help. Dr. Leone estimated it would cost $1.5 million to bring this newly developed gene therapy from the lab to clinical trials. Raising $1.5 million seemed nearly impossible, and Jennie and Gary weren’t sure how they would do it, but that didn’t stop them. The family set up a GoFundMe campaign, called ‘Save Benny and Josh’. In no time at all, friends, family, and strangers from around the world rallied together helping the family raise $1.5 million. Unfortunately, due to updated FDA requirements the cost of the trial was raised to $5 million. Between additional funding needed and other obstacles coming into play, the family’s original timeline to begin the clinical trial in April 2018 was pushed back.
Once enough funding was raised, and FDA approval was received, the clinical trial was ready to move forward. However, there was still one piece missing. The clinical team needed a neurosurgeon to perform the implantation of the gene therapy. Through connections in Dayton and the Living Biobank, Dr. Janson knew of the perfect neurosurgeon for the job. When asked to join the clinical trial, Dayton Children’s pediatric neurosurgeon, Dr. Robert Lober was up for the challenge!
“For the last year in our hospital windows, we’ve featured ‘hope lives in Dayton,’ and that couldn’t be truer than it is today,” said Dr. Lober. “This work is giving hope to so many families and will open the door for additional research at Dayton Children’s, allowing us to provide life-changing care for kids within our own community and around the world.”
On April 8, Benny became the first patient in the world to receive gene therapy for Canavan disease, using a newly modified recombinant Adeno-Associated – Viral Vector (rAAV) at Dayton Children’s Hospital. Friends and family from around the world followed along on Jennie’s Facebook page as she provided frequent updates from the operating room. At 4:30 pm, followers let out a sigh of relief when Jennie posted that “Benny is resting comfortable in the ICU. His vitals are strong.”
A week following his surgery, Benny had his first follow-up appointment with Dr. Lober, where he was cleared to return home to New York. Benny will continue to travel from Brooklyn to Dayton over the course of the next year for follow-up appointments with Dr. Lober. Alongside his brother, Josh will also travel to Dayton for follow-up appointments. On June 3, Josh was the second patient to participate in the gene therapy clinical trial.
Both boys received four doses of the vector therapy directly into the cerebral fluid in their brains. Dr. Lober and the research team are hopeful that the gene therapy will lead to a decreased amount of spongy white matter, leading to less degeneration. If the gene therapy works as they hope, Benny, Josh and the other children who receive the therapy may be able to relearn some skills and learn new ones. It can take up to 12 months before they see any clinical benefits from the gene therapy. The Landsman family is cautiously optimistic that this treatment will provide Benny and Josh with a new quality of life.
“There is reason for optimism and hope, but we try to temper that just a little bit,” said Gary. “I dream of running with my two sons who currently can’t sit up, walk or do much of anything. To have the expectation that we will be running through a field together a year or two after this treatment probably isn’t realistic. But at the same time, we don’t know what to expect. At the very least we hope this treatment extends their life expectancy and provides them more comfort. On the other end of the spectrum, we want the world for them. We want to watch them put their backpacks on, walk out the door and get on the school bus."
For more information on this clinical trial visit, clinicaltrials.gov
