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Ruth Arnon's failed experiment became a multiple sclerosis drug

Ruth Arnon and her colleagues set out to cause a multiple sclerosis-like disease in animals; their molecule blocked it instead, and after nearly 30 years of work it became Copaxone.

Ruth Arnon
Ruth Arnon · b. 1933
Person
Ruth Arnon
Lived
b. 1933
Field
Immunology
From
Israeli

The story

In 1967 the immunologist Ruth Arnon, her mentor Michael Sela and their doctoral student Dvora Teitelbaum began a project at the Weizmann Institute of Science in Israel. They wanted to understand a laboratory disease called experimental autoimmune encephalomyelitis, which is triggered in animals by injecting a protein from the brain and which mimics multiple sclerosis, the illness in which the immune system attacks the myelin sheath that insulates nerves. Their plan was to build synthetic copies of the brain protein and use them to cause the disease on demand, giving researchers a cleaner tool.

The plan failed. Their best characterized molecule, a random chain of four amino acids they named Copolymer 1, would not cause the disease. Arnon later remembered the mood in her personal account of the drug's history: "Disappointment. Was our hypothesis wrong?" After more than a year of trying, the team asked whether the molecule might be doing the opposite, and in 1971 they found that it was: several of their copolymers strongly suppressed the MS-like disease. "We didn't intend to develop a therapy for multiple sclerosis," Arnon has said. "It just happened in this serendipitous way."

What followed was a test of patience. A first trial in four severely ill patients at Hadassah in Jerusalem in 1977 showed no major side effects. Arnon then spent years on what she called the peddling period, going to almost every MS conference to persuade doctors to run larger studies. Murray Bornstein in New York agreed, and his 50-patient double-blind trial, published in the New England Journal of Medicine in 1987, found that the drug reduced attacks in relapsing-remitting MS. The Weizmann Institute licensed it to Teva Pharmaceutical Industries that year, and after a trial at 11 American medical centers the US Food and Drug Administration approved it as Copaxone, or glatiramer acetate, on December 23, 1996, nearly three decades after the project began.

Copaxone became one of the first treatments able to change the course of MS rather than only ease attacks. The Weizmann Institute has reported that at one point some 40 percent of the world's MS patients were taking it. Since its original patent expired in 2015, cheaper generic versions have been sold, and glatiramer acetate is on the World Health Organization's List of Essential Medicines. Arnon summed up the reward in her own words: the realization that their research had brought relief to somebody, "be it only to a single individual."

Why it matters

Glatiramer acetate, born of an experiment that did not go to plan, is still used around the world to reduce relapses in people with multiple sclerosis, now also in low-cost generic form.

About Ruth Arnon

Tel Aviv-born immunologist who, with Michael Sela and Dvora Teitelbaum, created Copaxone, a medicine that has helped millions of people with multiple sclerosis live with fewer relapses.

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Ruth Arnon
b. 1933 · Immunology
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Sources

Another act of tikkun olam. This is part of tikkun olam, the Jewish idea of repairing the world, and every one of us can add to it.

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