New York Scientists Win Prize for Treatment of disease that can cause fatal brain hemorrhages in unborn babies and newborn children

By Dave Andrusko

Dr. Richard Berkowitz, left, and Dr. James Bussel

Two longtime colleagues and collaborators have received the King Faisal International Prize for Medicine for pioneering treatment  of alloimmune thrombocytopenia, an auto-immune disease which can cause fatal brain hemorrhages in unborn babies and newborn children.

“The cause is unclear,” wrote Clem Richardson of the New York Daily News, “but the illness causes the mother’s immune system to attack the fetus as if it were a foreign body or disease.”

“In this disease, the mother’s body makes antibodies to the platelets in the baby’s blood,” co-winner Prof. James Bussel explained. “The baby’s blood becomes anti-coagulated in utero, which can cause spontaneous bleeding in the fetus, and the place the fetus bleeds most is in the brain.”

The diagnosis of alloimmune thrombocytopenia typically takes place after birth—after the mother has lost her baby or the baby has bleeding in the brain. The aliment is found around the world.

“The only way to know a woman is at risk of having it is if she had a sister who had it, or if it were a factor in a previous pregnancy,” Prof. Richard Berkowitz said. “For almost 30 years we have been working on treating fetuses that we know are at risk for this disease.”

Giving the mother high doses of steroids is part of the treatment. Although they are looking for alternatives, given the cost and the fact that people tolerate steroids differently.

Berkowitz and Bussel have treated at least 500 mothers since that first time they encountered the disease 28 years, and have been consultants on hundreds more.

“We serve as a kind of national referral center on alloimmune thrombocytopenia,” Berkowitz told Richardson. “If a doctor is treating a patient and has a question about treatment, they call one or the other of us to make sure they’re doing the right thing.”

In the U.S. alone Berkowitz and Bussel estimate about 1,000 babies are born each year with a severe form of the disorder. “Without treatment, almost a third will have serious bleeding in the brain — like a severe stroke — or die from it,” Richardson writes.

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