Courtesy: Medical Miracles, from Readers Digest 1981.
November.
From the frontiers of science and the far horizons of
personal courage, these stories of medical triumphs and miracles will reaffirm
your faith in the awesome powers of the human spirit. Dramatic victories and
human triumphs.
Selected and edited by the editors of Readers Digest
Ten year-old Philip had a cold, and his life was in the
balance. In his raw, swollen throat a few tiny blood vassals had broken. In most of us, this common mishap would go
unnoticed. But Philip was a hemophiliac,
a bleeder. His blood would not clot, and
the hemorrhage threatened to choke off his breathing.
To halt the previous bleed, doctors had transfused Philip
with plasma—the clear liquid portion of blood.
But each pint contains only a small amount of the clotting factor his
blood lacked and he had to be transfused day after day. Again and again the doctors had had to stop,
as the massive volume of plasma strained the capacity of the body’s circulatory
system and vital organs. Three times
Philip faced death before they could begin transfusion again. Only after four weeks did the bleeding end.
But this time the treatment was different. Science had found a way to concentrate the
clotting factor from over a gallon of blood [an adult’s entire supply] to a
vial of white powder. This was mixed
with sterile water, and flowed into Philip’s veins. Within hour his throat hemorrhage had
stopped.
“For the world’s hemophiliacs,” said the late Dr. martin
Rosenthal, former medical director of the national Hemophilia Foundation, “the
concentrated promise a new life. Now
each small accident need not mean a desperate struggle to survive.”
To understand this achievement, it helps to know the story
of Dr. Murray Thelin, for, though many researchers were involved, it was on
this young biochemist that the final triumph centered. And for him it had a double meaning: he, too,
was a bleeder. In serious form.
Hemophilia—an inherited disease—of furs in one of every 10,000 males,
prevalence roughly equal to that of polio before the Salk vaccine. But until accident or surgery causes a bleed.
In 1961, Dr. Thelin jointed the highland Division of Baxter
Laboratories in Los Angeles, to search for a concrete of the ant hemophilic
factor—called AHF. He was 34, a rather
small man, pale from recurring blood loss.
His colleagues remember that he arrived with his right arm in a sling,
impinge on his left leg and using a cane.
A few weeks latter they noticed that the sling was on the other arm and
the limp in the other leg. For, like
most bleeders, he suffered badly from joint hemorrhages, cause
deformities. Murray’s legs had bled
repeatedly since his infancy, and for years his main treatment had been bed
rest and painkillers.
Patients of hemophiliacs go to great lengths to protect
their children against injury. Internal
bleeds, not open wounds, are the worst threats, and a minor mishap may break a
blood vessel anywhere in the body, including the brain. Thus much of normal life may be denied to
homothetic youngsters.
But not young Murray Thelin, born in 1972 on a farm in
western Canada/ Murray tried everything.
He became an Eagle Scout, passing every test fairly. When he had to spend weeks in bed waiting for
bleeds to stop, he taught himself to play the saxophone and formed a band; he
became a champion model maker. But above all, he dreamed of doing something for
bleeders.
The Thelins were determined that Murray should have his
chance. When he was 19, they sold their
farm and moved to Seattle, where Murray began a premedical course at the
University of Washington. Getting a job
with a blood bank, he worked his way through.
It took him five years, and at the end there was disappointment; medical
schools refused him, saying he could not take the stress of training. Murray turned to blood chemistry. In three years, still working his way though,
he had his master’s degree and began studying for a doctorate at the University
of North Carolina, under Dr. K.M.Brinkhous, renowned authority in the field of
hematology, and Dr. Robert Sangner, a biochemist.
Brinkhous had given much hope to bleeders, for his work had
made transfusions far more practical as a way to build up the clotting factor
in emergencies. He had discovered that
the AHF was in the plasma, thus eliminating the need for whole blood
transfusions with the attendant risk of red blood cells accumulating in the
patient’s blood in such quantities as to cause dire reactions. Brinkhous learned that plasma with its
lifesaving AHF could be frozen and stored for emergencies. Since plasma doesn’t have to be typed,
bleeders now could be treated without a frantic search for donors. Still, the problem of volume remained. Brinkhous sought a way to concentrate the
AHF, but every known means of removing the fluid also destroyed the delicate
protein.
Then in 1957, Dr. Wagner suggested that an amino acid—a
substance from which proteins are made—might bond to AHF without damaging
it. Some amino acids proved to be gentle
enough agents to separate the AHF from the plasma. The researchers at Chapel Hill tested the AHF
concentrates prepared from human plasma.
Meanwhile, Stanford University’s Dr. judity Pool, working on
a potency test for AHF-bearing plasma, made a strange observation. Knowing how quickly AHF breaks down, she
wondered how much was lost during the 40-odd minutes it took to transfuse a
pint of plasma. She tested a sample of
plasma from the start of a transfusion, and one from the end of the
procedure. To her surprise, the sample
at the end was not weaker but far stronger.
Months passed before she fully understood why. Frozen plasma, thawed for use, was not
completely clear: it had a few white flecks of protein floating in it. In transfusion a filter in the plasma bottle
screened these out.
Then a fussy lab man decided to centrifuge the plasma to
remove the flecks. When Dr. Pool tested
this sample, it was surprisingly weak.
At once she suspected that the precious AHF was in the white sludge.
Clearly, under ordinary circumstances, some AHF dissolved
during the transfusion. But woven them
some remained trapped by the filter. Was
this a road to concentrate? To find out,
highland laboratories hired Murray Thelin.
At first Thelin found only more problems. To preserve the AHF, which loses its potency
at room temperatures, Murray conducted his studies in temperature close to
freezing. The cold did his joints no
good. He had bleeds in his knees,
ankles, hips, nose and tongue, and was in pain more often than not. Until this time, he had kept up his outside
activities—fishing, serving as a scout leader and as a Mormon elder. Now he devoted every hour he was well to the
lab.
By 1964, Murray, working with Dr. Edward Shabrom, medical
director of highland, was close to accomplishing what many believed
impossible. Now, after hundreds of
tests, they found a way to use an amino acid to gather AHF from the protein
sludge.
The result was not pure AHF, but it was highly
concentrated. And Murray managed to dry
this to a powder that dissolved in water.
He was just beginning to test the effect of his powder, when the worst
happened. Murray suffered a brain
hemorrhage.
Rarely, if ever, had a hemophiliac survived much a
hemorrhage. Plasma would take effect too
slowly to save him. Murray’s only hope
was his own untested concentrate. Yet
the concentrate might well cause fever of violent allergic reactions; even
ordinary plasma made Murray break out in a bad rush. Murray and Dr. shanbrom had no choice but to
take the risk. And Murray, though badly
blurred speech, helped give instructions to the lab and laid out a program of
tests on him to observe the drug’s effect.
Then came the first transfusion. Murray’s clotting levels soared. There was no untoward reaction. In ten days he walked out of the hospital,
smiling. And now he speeded his efforts
to learn how to produce the powder in quantity.
Tragedy threatened again.
Murray began to bleed from a peptic ulcer, usually a death warrant for a
hemophiliac. Once more his concentrate
worked, quickly, safely.
Still the experts raised questions. How would repeated doses affect a bleeder
over a period of time? Murray made
himself a guinea pig.
Both he and Dr.Dhanbrom felt that his tests might answer a
bigger question; could regular injections prevent bleeding? Shanbrom administered the powder daily, and
then found that weekly doses were enough.
The results seemed miraculous: weeks passed without a bleed. Yet hemophiliacs, for unknown reasons, go
through cycles of bleeding or not bleeding.
Was the concentrate really working?
The answer came when Murray went through a bad auto crash
without a bleed. Yet when the
concentrate was deliberately withheld, he blew at a slightest provocation. Clearly, the drug was protecting him.
When the concentrate was released to physicians for testing,
success stories rapidly mounted. A
six-year old Chicago boy had bitten his tongue, and for five weeks the bleeding
would not stop. Two vials of the powder
and the episode were over. In New York a
hemophiliac had suffered for months with chromic appendicitis. With the concentrate, his doctors decided to
risk removing the appendix. “The
operation was nearly as simple as if the man had been normal,” they said. In Boston, surgeons at Tufts University have
performed major operations on the spleen and brain of two hemophilic
patients. They both came through in fine
shape.
Then, as the supply of the concentrate began to grow,
doctors tried applying it to such common bleeder problems as tooth
extraction. Most important, because the
concentrate can be used to stop joint bleeds quickly, before permanent tissue
damage can occur, it offers the hope of preventing crippled joints. Says a
doctor at Stanford, “We believe today’s hemophilic youngsters have every chance
of growing up with straight limbs.”
The AHF concentrate is now widely available, and many
hemophiliacs are able to treat themselves at the onset of a bleed.
More recent discoveries offer hope for the few bleeders who
are not helped by the commoner concentrates.
While most hemophiliacs are different in the clotting protein called
Factor VIII, about 15 percent lack Factor IX or X. These factors were concentrated
experimentally by Dr. James Tulles of Boston’s Protein Foundation and New
England Deaconess Hospital, and are now commercially available. Some problems remain to be solved, and
research is focusing on ways to bypass the action of patients’ antibodies,
which sometimes limits the usefulness of the new medications.
Murray Thelin is not directing this project. In November of 1967 his heart suddenly gave
out. He was 39.
A month later Hyland laboratories accepted an award from the
Hemophilia Foundation, for Murray’s work and that of Dr.Shanbrom on the
concentrate. “I wished that Murray had
been there,” Dr.Shabbrom says. “But he
died knowing that in many ways, in his fight against hemophilia, he had won.”
