Hi Karen
I just got a copy of the report printed by the Bloomberg News.
See below.
Amgen Arthritis Drug Kineret May Fight a Rare Genetic Disease
2006-08-09 17:00 (New York)
By Emily Brown
Aug. 9 (Bloomberg) -- An arthritis drug sold by Amgen Inc.,
the world's biggest biotechnology company, may help combat a rare
inflammatory disorder that strikes during infancy, a study found.
The drug, Kineret, eliminated rashes and inflammation in the
eyes within three days, researchers report in tomorrow's New
England Journal of Medicine. About 200 children in the U.S. have
the disorder, known as neonatal onset multisystem inflammatory
disease.
New treatments are needed, as the disease kills about 20
percent of its victims before they reach adulthood.
``These findings let us hope that we may be able to prevent
mental retardation, deafness and vision loss as a consequence of
ongoing un-prevented inflammation in the brain, ears and eyes,''
said Raphaela Goldbach-Mansky, lead author and a staff clinician
at the National Institute of Arthritis and Musculoskeletal and
Skin Diseases in Bethesda, Maryland, in an e-mail today.
One child in the study was Seth Durrant, now seven years
old, of San Francisco. He was helped by Kineret.
``It's changed his life,'' said Karen Durrant, 36, his
mother, in a telephone interview today. ``He couldn't go to
preschool because he was screaming in pain and had a rash
everywhere.'' Durrant formed a support group called NOMID
Alliance for families whose children have the malady.
Similar to Arthritis
The disease, which is similar to rheumatoid arthritis,
occurs when a protein called cryopyrin, which regulates
inflammation in the body, isn't working properly. The result is
increased production of cytokine interleukin-1, a molecule that
causes inflammation. Kineret blocks that substance, according to
the study.
The Bethesda, Maryland-based National Institutes of Health
funded the research. Leonard Stein, Joe Cole, Terry Moore and
Richard Vehe, scientists involved in the study, received either
consulting and lecture fees or research support from Amgen,
Genentech Inc. or Abbott Laboratories, companies with interests
in the field.
In the study, 18 patients between the ages of four and 32
years old had been treated ineffectively for the inflammatory
disease before taking Kineret. The amount of drug injected in
each patient depended on their body mass, one to two milligrams
per kilogram of weight, Goldbach-Mansky said.
The treatment improved or stabilized the hearing and vision
in all patients. The drug also eased their pain. Inflammatory
symptoms subsided in eight patients at three months and in 10 at
six months, the researchers said.
Suppressing Inflammation
Current treatments are aimed at suppressing inflammation and
have included high-dose corticosteroids and anti-rheumatic drugs,
researchers said. Inflammation persists in most children who use
these medicines.
``There have been no effective therapies for this
condition,'' Goldbach-Mansky said. ``High doses of steroids are
effective for some disease manifestations, but have considerable
side effects.''
Children taking steroids before adolescence are shorter in
stature because of a premature growth halt and accelerated
puberty changes due to the drug. Other side effects include liver
tumors, cancer and jaundice, a yellow pigmentation of skin,
tissue and body fluids, according to the National Institutes of
Health Web Site.
The neonatal disorder is known in Europe as Chronic
Infantile Neurological, Cutaneous and Articular Syndrome. The
condition affects the central nervous system and leads to
seizures, meningitis and inflammation in blood and joints, which
causes swelling and redness.
Effects of Disease
Children who have the condition usually get a hives-like
rash within six weeks of birth and a bony overgrowth in the
knees.
The only way to contract the disease is through a genetic
mutation that changes a certain inflammation-fighting protein,
the researchers said. Sixty percent of the children diagnosed
with the disorder have this genetic change.
The U.S. Food and Drug Administration approved Kineret to
treat patients 18 and older with rheumatoid arthritis, an
autoimmune disease that causes stiffness, pain and swelling in
the joints. The condition is caused when the body produces too
much of a certain protein which leads to joint damage.
--Editor: Tannenbaum (rls)
Story illustration: For sources of research on health care and
medicine, see {HTGV <GO>} for organizations starting with the
letter A through G and {HMHZ <GO>} for H through W. For Web
sites related to diseases, see {DISE <GO>}.
To contact the reporter on this story:
Emily Brown in Washington at (1) (202) 624-1800 or
ebrown18@....
To contact the editor responsible for this story:
Robert Simison at (1) (202) 624-1812 or
rsimison@....
[TAGINFO]
DNA US <Equity> CN
AMGN US <Equity> CN
ABT US <Equity> CN
NI US
NI COS
NI HEA
NI SCIENCE
NI MEDICAL
NI DRG
NI GEN
NI MD
#<610787.1588400.2006-06-07T09:10:00.25>#
-0- Aug/09/2006 21:00 GMT