CORRECTION | A story yesterday about an experimental drug used to treat Robert Lord, a 9-year-old boy paralyzed from the neck down in an accident, incorrectly said the family included five children. There are six children, including Christine, 14. The San Diego Union-Tribune regrets the error. (980321, A-2)
20-Mar-1998 Friday
There isn't much more that the Lord family can do but wait -- and pray --
that an experimental drug they learned about on the Internet will save
their 9-year-old son from a lifetime of paralysis.
Robert was climbing a rope ladder hanging from the old acacia tree in his
Encinitas yard Sunday when the accident happened.
The rain-soaked ground let loose of the roots, upending the tree, and it
came crashing down, Robert and all. His spine was fractured and dislocated
in the fall and ligaments in his neck snapped like a branch from that very
same tree.
The student at St. James Academy was flown by helicopter to Children's
Hospital in Kearny Mesa where "doctors said they'd never seen such severe
damage in a child this young," said Robert's father, Stephen.
He was diagnosed as being quadriplegic with an injury just above the base
of his neck.
Although Robert can talk, think and eat, and breathe on his own, he cannot
move his arms or legs and has extremely limited sensation below the chest.
He lies with his head back and facing the ceiling, to keep the bones in
just the right position.
"They told us, `Don't give up hope,' " Lord said. "But then they let the
other shoe drop, that my son might regain only 15 to 20 percent of his
function. And that he was actually lucky being so young, he might be able,
someday, to feed himself.
"I got desperate."
A friend suggested that he look to the Internet for help.
Lord, a chemical engineer, typed the words "spinal cord injury" and found
what he called "a litany of horrors that laid out in excruciating detail
what could happen to my son."
There were articles on how to prevent bedsores from progressing to cancer,
how to deal with muscles shriveling and bones becoming brittle, and a lot
of high tech articles out of reach to the average person.
"My heart just broke," Lord said, his voice quivering.
But in the midst of it all, he found a 12th-grader's English class essay
that briefly referenced a drug, GM-1 ganglioside, which, if given within 72
hours of an injury, might dramatically improve recovery in spinal cord
patients. A doctor named Geisler was the principal investigator.
With another Web search, a friend found Dr. Fred Geisler practicing in
Chicago, and further research found his 1991 report in the prestigious New
England Journal of Medicine.
In a study of 34 patients with injuries similar to Robert's, those given
ganglioside had significantly better improvement than patients given a
placebo.
Dr. Roberto Fiorentini, president of Fidia Pharmaceutical, the drug's
manufacturer, suggested the drug has two ways of working on spinal cord
injuries. It might stimulate nerve cells to grow more than they otherwise
would, and it could slow the normal process in which nerve cells die after
injury.
But the study was too small to be definitive.
"It could have been a fluke," Fiorentini said. In 1993, another study was
designed to take a scientific look at the drug's performance in a group of
800 spinal cord patients, half of whom would get a placebo.
When Lord called Geisler in Chicago, he learned from a secretary that the
results are in from the larger trial and are being analyzed.
The secretary suggested that he call Fidia Pharmaceutical and see if he
could get some of the drug. A Fidia representative said yes, but use of
drugs not yet approved by the federal government requires special
permission.
Lord was told to have Robert's neurosurgeon at Children's Hospital, Dr. Hal
Meltzer, request permission for "compassionate use" from the U.S. Food and
Drug Administration.
The infrequent requests are made on behalf of patients for whom there are
no other treatments, and for whom the prognosis is otherwise not good, an
FDA spokesman said. They are not always granted, but in this case it was,
and within hours, Meltzer said. "The FDA moved us along extremely quickly,"
he said.
By Wednesday, Fidia had wrapped up a one-month supply of the drug and a
friend of Lord's in Washington, D.C., drove it to the airport. In San
Diego, Lord picked up the package at Lindbergh Field and drove it to the
hospital, just about 76 hours after the accident.
Some physicians familiar with ganglioside worry that while there are no
guarantees that it will work in Robert, there's also a chance it could do
unknown harm.
"Giving this drug makes the fundamental assumption that something is better
than nothing," said Dr. Larry Marshall, UCSD Medical Center neurosurgery
chief who directs the trial of ganglioside here.
He cautioned that the drug may react badly with a steroid drug that is
standard treatment after such injuries. Robert received the steroid hours
after he was brought to Children's Hospital.
New York Jets player Dennis Byrd, who received a paralyzing neck injury,
and actor Christopher Reeve, a spinal cord injury victim, both received the
drug. It is not known whether it helped them, Fiorentini said.
Meltzer said that for now, everyone is refusing to give up hope.
"Children have a much better chance of recovering than adults, and because
of that, I never say never," Meltzer said. "He is a very, very brave boy."
The Lords are hoping to see some improvement in a few days, but any benefit
might take six months to a year.
Robert's mother, Michele Tutoli, thinks he will be fine. They have four
other children, including 17-year-old Stephanie, a freshman at UCSD who
wants to be a pediatrician.
"Robert said to us the other day, when Stephanie was in the room, that he
knows Stephanie will be a doctor when she grows up. She always helps him
when he has a cut on his leg," Stephen Lord said.
"She just doesn't know how to fix this problem just yet."