A cure for sickle cell may be a long time coming, but local researchers
want to offer patients a medicine they say could ease the pain in
the meantime.

   Steven Goodman's studies show that N-Acetyl Cysteine, a prescription
drug already on the market, could help sickle cell patients suffering
from the pain of this genetic blood disease.

   Goodman, who directs the University of South Alabama Comprehensive
Sickle Cell Center, has been studying how this drug changes the
bonds of sickle-shaped cells. After promising research, he and
his team are ready to start patient trials here in late February,
the next step toward the Food and Drug Administration's approval
of the drug as a sickle cell treatment. It is already used as a remedy
for overdoses of a substance commonly found in painkillers.

   It's going to make them feel better,'' Goodman said about
the potential of the drug as a sickle cell treatment.

   The help won't come too soon for sickle cell patients, who suffer
when their elongated  or sickled  cells try to squeeze through blood
vessels designed for round cells. The disease causes organ damage
because the deformed red blood cells cannot do their job of transporting
oxygen.

   Sickle cell disease is a genetic disorder that afflicts about
60,000 African Americans. One in 500 African Americans has the
disease, and one in 12 carries the genetic trait.

   Sickle cell anemia, the most severe form of sickle cell disease,
affects about 2,000 Alabama residents and an estimated 148,000
carry the genetic trait, according to figures from Alabama's Sickle
Cell Disease Association.

   Some scientists propose certain gene therapies for the treatment
of sickle cell disease. Several different scientists have experimented
with drug treatments to increase fetal hemoglobin, a type of hemoglobin
that helps decrease the number of cells that sickle.

   But Goodman said he's the first to study blocking the formation
of the irreversibly sickled cells with drug therapies. His team
has found that red blood cells that become irreversibly deformed
do so because of damage to a protein called beta-actin.

   When this damage occurs, a type of bridge forms, changing the
shape of the actin protein and locking the cellular skeleton into
a sickle shape.

   Some cells remain in this shape even after being treated with
oxygen and even though their hemoglobin appears to have returned
to normal.

   N-Acetyl Cysteine is promising because it breaks the bridge-like
structure by adding hydrogen atoms to the protein. This allows
the cell to return to its normal shape and pass unrestricted through
blood vessels. Although the treatment does not prevent the cells
from sickling again, it does reduce the pain patients experience
and minimize organ damage.

   Goodman and his team of doctors and researchers want to start
off by testing 40 sickle cell sufferers age 15 and older who have
more than two painful sickle-cell crises per year. The patients
feel intense internal pain because their organs can't get enough
oxygen. The sickled cells can't transport oxygen, and they block
normal red blood cells from getting to the organs.

   The next step could be a larger patient trial at centers across
the country. One advantage of N-Acetyl Cysteine is that it already
has been tested on thousands of patients for its other uses, marketed
and proven safe, said Dr. Betty Pace, an assistant professor of
pediatrics and structural and cellular biology at the USA College
of Medicine. The drug's worst known side-effect is nausea and diarrhea
in 1 percent of all patients.

   The biggest thing is that it's a potential new treatment for
sickle cell patients,'' said Pace. It could improve the quality
of life.''

To Get Involved
   The Comprehensive Sickle Cell Center at the University of South
Alabama is looking for sickle cell disease patients to participate
in a drug trial of N-Acetyl Cysteine, a drug that could help prevent
painful sickle-cell crises.

   Participants who are age 15 or older and who have two painful
sickle-cell crises per year are preferred.

   Each patient will be tested to see if he or she has enough dehydrated
cells to participate in the study.

   For more information, call 405-5115 or 460-7334.

   PHOTO     Sickled red blood cells can't transport oxygen to the body's
organs, and they block normal red blood cells from passing through the
vessels. This leads to vessel blockage  called vaso-occlusion in scientific
terms. It also means pain to sickle cell patients who aren't getting enough
oxygen in their organs. In this illustration, some sickle cells are shown in
lighter gray.

   PHOTO   Rather than being round and pliable, a sickle cell is elongated and
hard.

This article reproduced with special permission from the Mobile Register.