A young boy suffering from what has been described as a combination of Alzheimer’s and Parkinson’s is hoping for a miracle after clinical trials of a drug to treat his condition were disrupted.
6-year-old Leo Vokler suffers from infantile neuroaxonal dystrophy (INAD), a disease caused by an enzyme deficiency or dangerous accumulation of iron in the body. He was diagnosed at the age of two.
The rare diagnosis affects only about 150 children worldwide, Walker’s parents told Insider, and its rarity also means treatment options are few.
An option appeared for the youngster, and he signed up for a trial of a drug believed to be able to cure his rare disease. Unfortunately, the company conducting the trial went bankrupt and cut it short. His condition was too advanced for him to re-enter school later.
Waukler’s parents, mother Deborah and father Antoine and sister Eva, are hoping something will be found to treat him before he dies within the next six years, with little left.
6-year-old Leo Vokler (pictured) has infantile neuroaxonal dystrophy (INAD).
Lion’s disease (pictured) is fatal within 5 to 10 years of diagnosis in a child
“We try not to say ‘poor baby’ in front of Eva,” Deborah Voeckler, 41, told Insider.
“But we say it often because his life is unimaginable.”
The family is from New York but currently lives in France. Deborah says that Eva has become her brother’s helper, but she doesn’t know exactly what kind of situation her brother is in.
“If he’s down, he’ll kiss him and help me… he doesn’t understand because he’s only 3, but he loves being with her,” she explained.
He first started showing symptoms when he was 14 months old. His parents noted that he had poor balance and missed some important milestones in speech development.
Care by therapists and routine testing did not identify the condition. In 2018, she underwent genetic testing and was diagnosed with INAD.
Leo’s (left) parents say his 2-year-old younger sister, Eva (right), helped him because they weren’t sure why his brother was sick.
Leo’s family (pictured) enrolled him in a drug trial for a potential INAD treatment candidate, but that was cut short after the company filed for bankruptcy in 2021.
The National Institutes of Health describes the condition as a rare neurological disorder that affects the axons of the brain.
It gradually causes the sufferer to lose sight, mental ability and muscle control – which is why it has been compared to Alzheimer’s and Parkinson’s.
The disease usually appears in the first two years of a child’s life. Its main reasons are unknown.
There is no treatment or cure that can slow its progression over time.
“We thought, ‘How could this be? How could our child, or any child, be given this life?’
When Leo was diagnosed with INAD, Deborah was pregnant with Eva and feared their unborn child would also develop the condition – doctors estimated a 25 percent chance.
“I was a total wreck… I couldn’t sleep. I couldn’t eat, I was always nauseous from nervousness,” he describes.
Tests before Eva was born revealed she was likely to be healthy, but it was a relief for the family.
The family teamed up with the INADcure Foundation to try to find a cure for the disease.
The family has set up a foundation for Leo, hoping to raise funds to develop a gene therapy that could halt the progression of INAD.
Leo’s mother, Deborah (centre), fears that even if a treatment is developed, it will be too late to save her son
After that, the Bisous for Leo campaign was launched. In the months following his diagnosis, the youngster was also enrolled in a drug trial for a promising treatment candidate for the disease.
In 2021, the company conducting the trial faced financial difficulties and declared bankruptcy, suspending the trial.
When his family tried to get Leo into a follow-up trial, they were told that his condition had deteriorated to the point where the medication might be doing him more harm than good.
His family says he is now barely aware of his surroundings. He requires a feeding tube and uses a molded chair that supports his body and keeps him upright.
The state of the foundation needs about $7 million to start a new gene therapy, but even if the money comes, it may be too late for Leo.
‘They say it will be ready in 18-24 months…is he eligible for trial? Does he have another 18-24 months to live?’ Deborah said.