Moser Center for Leukodystrophies Launched
Patients with a group of rare degenerative brain diseases known as leukodystrophies have few places to turn for expertise in their medical care. Neurologist Dr. Ali Fatemi and his colleagues hope to change that with the new Moser Center for Leukodystrophies, launched this past December.
Following in the footsteps of the late Kennedy Krieger neurologist Hugo W. Moser, one of the world’s foremost leaders in the field of leukodystrophies, the new center carrying his name offers comprehensive treatment, research, and hope for families struggling with these disorders.
Leukodystrophies, which destroy the protective myelin sheath surrounding the brain’s nerve cells, can affect multiple organ systems, leading to an array of debilitating symptoms such as blindness, deafness, seizures, loss of muscle control, and cognitive decline.
Some leukodystrophies strike in childhood, and many children die within one to ten years of onset of symptoms. Other leukodystrophies have an adult onset. All are progressive diseases, meaning they worsen over time, and there is no cure.
“We want families to know that just because there is no cure doesn’t mean there is no care,” says Dr. S. Ali Fatemi, who heads the Moser Center. “These patients have a neurological disease, but it also often involves other organ systems,” he says. “We recognize that we need to address all aspects of their care.”
To accomplish this, the center has a team of interdisciplinary specialists—including neurologists, physical therapists, social workers, physical medicine and rehabilitation physicians, urologists, and endocrinologists. Genetic counselors are also on hand to counsel patients and their families about inheritance, prognosis, family planning, and other questions they may have about the disorder. For patients with unknown leukodystrophies, the center also offers an individualized medicine approach using advanced genomic techniques, in addition to the standard tests and MRIs.
Investigators at the Moser Center are actively researching new therapies and drugs aimed at preventing and treating leukodystrophies.
Scientists are exploring cell therapy and stem cell research, brain imaging, and treatment models in mice to search for drugs that reduce very long chain fatty acids, the biomarker for leukodystrophies. If the drugs show improvement in mouse models, permission for clinical trials is sought.
The Center works collaboratively with other leukodystrophy centers across the country and around the world. Working together, leukodystrophy experts can share information that will help patients get the treatment they need and seek funding for the most promising treatments to help those in the leukodystrophy community.
The Lorenzo’s Oil trial, championed by Dr. Moser and made famous by the 1992 movie “Lorenzo’s Oil,” was an encouraging clinical research study conducted at Kennedy Krieger. This trial showed that three-quarters of the presymptomatic boys with adrenoleukodystrophy (ALD) treated with the oil did not develop the devastating neurological symptoms of this disease during childhood.
Scientist Ann Moser, Hugo Moser’s widow, has focused much of her research over the past several years on refining newborn screening that can identify babies with X-linked ALD, which mainly affects boys, and implementing newborn screening for ALD nationally. “The only way to save these boys is to identify them early, before they have the first symptoms of brain disease,” she says.
This past December, the state of New York added ALD newborn screening, resulting in the identification of two positive screens in the first weeks of implementation. With the help of lobbying efforts by parent ALD groups,
Moser is hopeful that other states will follow suit.
Asked what her late husband would think of the new center, Moser replied, “I think he would be pleased. He believed strongly not only in providing care to people with leukodystrophies, but in developing new therapies and using the best treatments available for these patients.”
To learn more about the Moser Center for Leukodystrophies at Kennedy Krieger Institute, visit kennedykrieger.org/leukodystrophy.