Moyamoya, Deadly And Mysterious Disease In Little People Can Be Treated

Main Category: Pediatrics / Children's Health
Also Included In: Neurology / Neuroscience
Article Date: 19 Sep 2008 - 7:00 PDT

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Neurosurgeon Gary Steinberg, MD, PhD, leads the largest and most successful program in the world for treating Moyamoya, a mysterious vascular time bomb in the brain . Patients from Alabama to Australia have been seeking Steinberg out in a desperate effort to treat a brain disease so rare most neurologists will never see a case.

But after treating over 350 kids and adults worldwide, and with a 95% success rate, Steinberg is wondering about a pattern developing among an unusual subset of patients. With this week's surgery on 14-year-old Drake Taylor of Dunlap, IL, Steinberg has now treated three dwarfs in 2008.

"It's not certain why," said Steinberg, "but there's an association between Moyamoya and Drake's type of dwarfism." That's why Steinberg is partnering with the Potentials Foundation, a non-profit advocacy group, to find the approximately 150 dwarfs worldwide born with Drake's Majewski osteodysplastic primordial dwarfism type II (MOPDII). Currently, the Foundation and its network of support are in touch with 50 of the dwarfs. "The Moyamoya could be lying dormant, waiting to attack. That's why we want this entire community to have MRI/MRAs done annually." Steinberg, who's working as an advisor to the Foundation, is volunteering to read the resulting scans. "Unless you have our program's experience," added Steinberg, "it can be difficult to diagnose."

Steinberg may find more cases like Drake's. "At school last spring, Drake got a blockbuster of a headache," said mom Beverly of her 7th grader who, at 39" and 39 lbs, has maxed out his growth. "Tests showed Moyamoya. My husband Randy and I were worried that Drake's next headache would be a stroke or hemorrhage." The rare disorder, cause unknown, creates a narrowing of the blood vessels to the brain, which reduces blood flow and leads to strokes, hemorrhaging, and other deficits. Steinberg treats patients like Drake through a highly complex and delicate revascularization surgery, "which basically allows us to create a new channel of blood flow to the brain," said Steinberg. "Still, after hundreds of successful surgeries, there are a lot of people who believe Moyamoya is untreatable. We're proving it's not."

Now recovering from surgery, Drake will soon return to cowboy life on his "Little D Ranch" of farm and miniature animals back in Illinois, where he takes breaks by riding horses, catching John Wayne movies, and working on his Little D jewelry. "Drake is 39 inches of the purest love, pride, compassion, and determination you'll ever meet," said Beverly, "and thanks to Dr. Steinberg, his journey can now continue."

For Steinberg and the Potentials Foundation, the outreach to locate dwarfs like Drake has important research implications.

"Certain syndromes are well-recognized as having an association with Moyamoya incidence, such as neurofibromatosis, sickle cell disease and Down syndrome," said Steinberg. "But there's scant literature on the subject and until recently, Moyamoya association with rare diseases like Drake's has been overlooked." Though it may take awhile to find everyone, Steinberg is excited about this research. "I have no doubt this project will better define the link between Moyamoya and this type of dwarfism," said Steinberg. "And as a result, our program can help this community live much healthier lives."

Links

- In 2006, the New York Times profiled the Moyamoya work of Gary Steinberg, MD, PhD, neurosurgeon at Packard Children's and professor and chair of neurosurgery at the Stanford University School of Medicine, in the feature Out of Nowhere, a Devastating Tangle in the Brain.

- Read about "the smallest people in the world" at http://www.primordialdwarfism.com/.

- Drake Taylor's blog.

- Surgery Video Here.

About Lucile Packard Children's Hospital

Ranked as one of the nation's top pediatric hospitals by U.S. News & World Report, Lucile Packard Children's Hospital at Stanford is a 272-bed hospital devoted to the care of children and expectant mothers. Providing pediatric and obstetric medical and surgical services and associated with the Stanford University School of Medicine, Packard Children's offers patients locally, regionally and nationally the full range of health care programs and services, from preventive and routine care to the diagnosis and treatment of serious illness and injury.

Lucile Packard Children's Hospital