Israelis Yulia and Gili Ben Moshe were confronted with every parent’s nightmare when their third child, then three months old, was diagnosed with Canavan disease, a degenerative condition that occurs disproportionately among Ashkenazi Jews.
Canavan patients lack a particular enzyme that breaks down fat in the brain. Over time, the accumulation of fat erases the brain’s ability to function, and sufferers lose their sight, hearing, and motor function. Most children with Canavan don’t survive past age four.
Within 24 hours of Lavi’s diagnosis, the couple, who live in Hadera, found Dr. Paola Leone, a New Jersey expert on the disease. Associate professor of neurosurgery and director of the Cell & Gene Therapy Center at UMDNJ-Robert Wood Johnson Medical School, based in Stratford, she is developing innovative treatments for Canavan patients.
Ten days later, the couple flew to New Jersey to meet with Leone. Lavi was started on pharmacologic therapies, which resulted in delaying his brain deterioration. Today, at 17 months, he is progressing — he can see and hear — defying the diagnosis. Drugs alone, however, will not retard the disease’s progression.
Leone is conducting research on possible treatment involving the implanting of stem cells into the brains of children with Canavan, which, she said she believes, would result in the proliferation of functional brain cells. She intends to begin clinical testing as soon as the efficacy and safety of the treatment are proved. She expects the clinical protocol to be approved within 24 months by the Food and Drug Administration.
“Lavi’s psycho-motor development has been improving greatly since the pharmacological therapy began,” said Leone in a prepared statement. “The therapy is preserving his brain but is not curing the disease. The timing and success of a cure for Canavan Disease fully depends upon the availability of appropriate research funds.
According to Leone, her research is funded only partially by the government and for it to be completed and approved, an estimated $1 million is needed.
After successfully raising funds for Lavi’s treatments, the Ben Moshes have launched an on-line fund-raising campaign for Leone’s research.
“You know, it’s very hard for my wife and me to face this ordeal,” Gili Ben Moshe said in a prepared statement. “But as my wife says, Lavi came into this world with a purpose. We believe it’s our privilege to fight for Lavi’s life and for the lives of other children who have this lethal disease.”