UF pioneers treatment for disease

Published: Thursday, January 10, 2008 at 6:01 a.m.
Last Modified: Thursday, January 10, 2008 at 12:00 a.m.

Taha Naveed spent most of the first four years of his life constantly weak, with a belly uncomfortably swollen.

His mother, Noreen Naveed, left her job teaching English at a university in Pakistan and spent her days following Taha with a spoon and a bag of cornstarch. Three alarm clocks warned her when to give the next dose, because Taha would have a seizure if he did not get it. Cornstarch was a medicine that worked, and she gave it to him every two hours.

Taha's unstable condition prevented him from going to school. Doctors in Pakistan said Taha could not eat sweets or drink milk, so he spent all his birthdays without a cake. Enter Shands at the University of Florida and its world-regarded program treating glycogen storage disease, which had rendered Taha so ill.

The disease tricks the body into thinking it is starving whenever it is not digesting food, Weinstein said. Thus the body cannot burn stored energy for nourishment between meals.

Some 25 years ago, doctors discovered that cornstarch acts like a medicine for this disease, so it continues to be the "medicine" of choice.

Naveed and Taha traveled from Pakistan and spent three months in the U.S. so Taha could be treated for glycogen storage disease at Shands. They recently returned to Pakistan after completing treatment.

Taha was among the first patients to have his medical expenses provided by the Alyssa's Angel Fund - a fund that those at Shands expect to be used to attract other patients from across the U.S. and around the globe.

The fund was created in September to help people get treatment and manage their disease, said Dr. David A. Weinstein, director of UF's glycogen storage disease program. It was named after Alyssa, a 2-year-old still coping with glycogen storage disease. Alyssa's family started the fund with a $30,000 donation that has since grown.

The goal of the fund is to help three to four children, like Taha, each year, Weinstein said. So far, it has helped five patients get medications and therapy, and Taha was the first patient from a foreign country to be helped by the fund.

One in every 100,000 people is born with glycogen storage disease, Weinstein said. This disease is considered fatal because of its rarity, he said. Many doctors do not even know it exists or how to treat it. But now that doctors are successfully treating the disease, it is no longer considered life-ending, Weinstein said.

There are two centers in the U.S. that research and treat this disease, Weinstein said. Shands is the leader in research funding and capacity to treat patients. Duke University Hospital has the other center.

In 1982, doctors discovered that cornstarch acts like a medicine for this disease, he said. Before this discovery, Weinstein said, patients had to be constantly fed through a tube so the body would always be digesting and receiving nourishment.

Cornstarch can help because it takes a long time to digest and typically will hold those with the disease over between their meals, he said. But if doses of cornstarch are not timed correctly, it can harm the patient.

If the doses are too little, the body will go back into starvation mode and burn its own muscle for energy, Weinstein said. This can be crippling for growing children. And if the patient receives too much, it can build up and make the body sluggish and ill.

When Naveed told her family about her plans to travel with Taha to the U.S., they were resistant, she said. Naveed is the youngest daughter in her family, and her parents did not want her to travel alone. Taha's father could not accompany them because the embassy would not release his passport, Naveed said.

Naveed said she and her husband e-mailed doctors all over the world in search of help for Taha. UF was the first to respond. Not only did Weinstein reply, he provided her with medical advice by e-mail and sent her an application after Alyssa's Fund was established, said Laurie Fiske, the glycogen storage disease program coordinator at Shands.

The family's medical expenses were provided for because they did not have the money for the flights or the treatment. The Association for Glycogen Storage Disease provided their plane tickets, Fiske said.

Now that Taha and his mom have returned to Pakistan, Taha's treatment will be in the hands of local doctors and Weinstein via e-mail.

After working with Weinstein, Naveed said she learned that Taha can have milk products in moderation and he can also eat fruits. She was trained to give Taha 30 grams of cornstarch with meals after every four hours, she said - about half of what she originally gave him. Now he can go to school because classes last for only four hours.

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