Here it is.....
Here's the real story and the real loss. Big thumbs down to the six no votes.
BETHESDA, Md. (AP) - Partially paralyzed patients sobbed as they pleaded
with the government Thursday to approve a drug that promised to modestly
slow their Lou Gehrig's disease - but scientists rejected their pleas.
Scientific advisers to the Food and Drug Administration decided on a 6-3
vote that there was not enough evidence that the drug Myotrophin would
help patients with Lou Gehrig's disease.
The problem: One study suggested the drug could postpone patients'
worsening paralysis by three months - and eventually increase life by an
equal time - but a second study showed no benefit at all.
``I'd love to be able to say'' Myotrophin works, panel chairman Dr. Sid
Gilman, a University of Michigan neurologist, said. ``The data are just
not here.''
Lou Gehrig's is so untreatable it ``should be a special case,'' argued
Medical College of Virginia biostatistician Chris Gennings, but she was
outvoted.
Drug developers Cephalon and Chiron Inc. took a gamble in ignoring the
same panel's call last June to do a tie-breaking study. The companies
banked on newly available data that showed the originally tested
patients lived, on average, three extra months after Myotrophin
treatment.
About 30 patients and their relatives erupted into tears as the panel
rejected the drug. The three months of stable health patients believe
Myotrophin offers are vital, they said, and one mother accused the FDA
of whittling away her son's life by not allowing Myotrophin to sell when
the first positive study concluded a year ago.
``If Myotrophin had been cleared ... when you evaluated it last year, he
might still be able to eat, he might still be able to breathe,'' Diane
Whitaker of San Francisco said of her 35-year-old son, Douglas, who
needs a feeding tube and oxygen after being diagnosed with Lou Gehrig's
disease last summer.
Myotrophin offers ``a gift of hope that a cure can be found in those
months,'' Janice Dorfman of Long Island sobbed, her voice paralyzed and
her statement read by a computer translator.
The FDA is not bound by advisory committee decisions, but typically
follows them.
Some 30,000 Americans suffer Lou Gehrig's disease, formally known as
amyotrophic lateral sclerosis or ALS. It kills nerve cells in the brain
and spinal cord, causing creeping paralysis that eventually leaves every
victim unable to eat or even breathe. Patients' life expectancy - just
three to five years - has changed little since the disease killed
Yankees first baseman Lou Gehrig in 1941.
Only one drug, Rilutek, is sold for ALS. It prolongs some patients'
lives by a few months but has not been proved to slow the worsening
symptoms.
Myotrophin, however, is a genetically engineered growth factor called
IGF-1 that promised to improve patients' quality of life by slowing the
paralysis. It is thought to promote the survival of nerve cells and help
them rebound after injury.
But drug tests found Myotrophin's effects modest at best, the FDA panel
concluded. One study in North America found Myotrophin slowed patients'
muscle deterioration by 26 percent - buying them about three months of
better health before paralysis increased.
Then a second study in Europe found no effect. FDA doctors couldn't
agree which study was more believable, so they asked the outside panel
of neurologists for help.
Last June, the same panel agreed to let Myotrophin be given for free to
desperate patients while Cephalon and Chiron got an answer. Only 450
patients won the drug under a special lottery, because supplies are
limited.
The companies argued the future of ALS treatment may be in combining
Rilutek and Myotrophin - much as diseases like AIDS and cancer are
treated with drug combinations - and they planned to study that question
if Myotrophin was allowed for sale. |
