Parents, family rally around sick toddler
By Connie Lyons
Alysa Dye sits on her mother’s lap, banging sharply on the table with a spoon. She has wide-apart eyes, with beautifully arched brows in an enchanting, heart-shaped face. Like any toddler, she is possessive of her mother’s attention.
“Here!,” says the two-and-a-half year old imperiously, handing her the spoon. But Alysa is not like other toddlers. She has a rare neurological disease called Metachromatic Leukodystrophy, and she will likely die before she reaches her fifth birthday.
“Up until last March, she was perfectly normal,” said Alysa's mother, Christine. “Then we noticed something funny going on with her right eye, like it was turning in. Thinking it was a 'lazy eye,' we took her to an eye doctor. He thought it was likely nothing, but ordered an MRI just to be sure. Well, the MRI came back abnormal.”
The neurologist the Dyes consulted told them that, judging from what he saw in the MRI, he had expected her to be really sick, but she wasn’t.
“He didn’t think she had MLD. Then she started dragging her right foot. She walked late ? not till she was 18 months ? but we figured she was just a late walker. But when she started dragging that foot, the neurologist had her admitted to Children’s Hospital in Washington, D.C., where they did a thorough work-up,” said Dye. The work-up involved a spinal tap, another MRI, and blood and urine analyses. All the tests were positive for MLD, and the diagnosis was confirmed at the end of last March.
“Basically, what MLD does is shut the nerves down,” said Dye. “Alysa will go blind. She will have muscle spasms. Those have already started, and they’re really painful, so she has medication for that. She can’t walk without holding on to something. Eventually the spasms will progress to seizures; she will become comatose, and she will die by the age of five or five and a half.”
MLD is a rare genetic disorder. Both parents must be carriers of the disease. The Dye’s sons, Jonathan, 7, and Hunter, 6, are genetically carriers of the disease but are not affected.
It takes three forms: late infantile, which is the one affecting Alyssa; juvenile, and adult. The infantile form of the disease is the most common, and the most rapidly advancing. Symptoms are muscle weakness and wasting, muscular rigidity, developmental delays, progressive loss of vision, convulsions, impaired swallowing, and dementia.
The juvenile form of the disease, which progresses more slowly, manifests itself between the ages of three and 10. It starts with impaired school performance, mental deterioration and dementia and progresses to the same set of symptoms as the infantile form of the disease.
The extremely rare adult form appears after age 16 and starts as a psychiatric disorder or progressive dementia, and it, too, advances far more slowly than the infantile form of the disease. There is no cure at any age, though bone marrow transplants and experimental therapies may slow its progress.
“The muscle spasms began a few months ago. Alysa would begin screaming in the middle of the night, and I could see the muscles tightening up. The medicine she takes has helped a lot with that. Eventually she’ll have seizures,” said Dye.
Every two months, her parents take Alysa to Dr. Adeline Vanderver, a pediatric neurologist in Washington, D.C. as well as to an eye doctor.
“She’s losing some vision, so they prescribed glasses, but I’m having trouble getting her to wear them,” Dye said.
And every three months they make the long journey to Chapel Hill, North Carolina where Alysa is a participant in an experimental study.
“We discussed the possibility of a bone marrow transplant,” said Dye. “But that would only slow the disease down, not cure it, and the chances of her surviving the chemotherapy were one in three. So we decided against it.”
The study Alysa is involved in centers on an enzyme treatment that has been successful in Europe and is newly approved by the FDA.
“They’re having really good results,” said Dye. “But again, it only slows the progress of the disease; it’s not a cure. And they need 10 participants signed up before the study can start, and since the disease is so rare, that’s not easy. Most doctors have never even heard of it. “
Because Alysa has difficulty swallowing, she doesn’t eat well. “Lack of nutrients will cause the disease to speed up,” said Dye, noting that a feeding tube was surgically implanted at Children’s Hospital in Washington, D.C. on Oct. 24. At the same time, the lining of the baby’s stomach was pulled up and fastened to her esophagus to prevent reflux.
“At first it was terribly hard coping with Alysa’s situation,” Dye said. “My husband has his up days and down days, but he’s doing better than he was at first. I had to quit my job because of all the doctor’s appointments. And in order to qualify Alyssa for Social Security disability benefits, my husband had to take a lower paying job. We do have family in the area, and they’ve been wonderful about giving us hands-on help. Financial, as well, when we needed it.”
Medicaid helps to pay for the extensive elaborate equipment Alysa needs ? braces to help her walk, night-time braces to keep her legs straight so they won’t cramp, a special needs stroller, bathtub and walker.
“What would really help, would be for people in the community to spread the word, to know about the disease, about Alysa’s situation,” Dye said. “And donate to the foundation. It’s lack of funds that’s slowing down the research. So help won’t be coming for our baby, but maybe down the road there’ll be help for other children. Then, maybe through Alysa, another child will live.”
Donations to help the Dye family may be sent to P.O. Box 611, Bealeton, VA 22712, or directly deposited into Alysa Dye’s account at the Fauquier Bank. To donate to the MLD foundation, go to its website: www.mldfoundation.org.