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Multiple Sclerosis: How Scientists Developed a Breakthrough Drug

The new drug ocrelizumab is helping lots of people with multiple sclerosis.

Imagine waking up to a world that blurs in front of you. Heading to the kitchen for breakfast, you’re hit with a wave of exhaustion, and your arms and legs feel like lead. This is the reality for the more than 400,000 people living with multiple sclerosis (MS), one of the most common neurological conditions in the United States. MS causes progressive nerve and brain damage as the body attacks the protective covering around each of its own nerve cells (called the myelin sheath). It causes vision loss, weakness and numbness in legs, and eventual disability. There are two types: relapsing-remitting and primary-progressive. In relapsing-remitting MS (the more common form), patients experience episodes of symptoms, followed by periods of recovery. In primary-progressive MS, there are no periods of relapse and the patient’s condition steadily declines.

While records of this condition date back hundreds of years, it is only in the past 25 years that the first drugs with modest efficacy were approved to treat MS. Still, there hasn’t really been a good way to control it—until last year. In 2017, the Food and Drug Administration (FDA) approved ocrelizumab, a medication that has been shown to significantly slow down the progression of the disease, and the first approved to treat primary-progressive MS. It was hailed in headlines as a breakthrough drug.

But why didn’t this drug come sooner?

Contrary to popular belief, most drug discoveries are not the product of one eureka! moment, but the result of decades of research, trial and error, and persistence woven together by scientists and doctors from around the world, each learning from others’ findings and mistakes.

“Drug discovery can be a random walk,” says David A. Hafler, MD, chair of neurology at Yale Medicine. “There are myriad experiments a researcher could choose to do. The one that ultimately leads to the cure can be based largely on chance.”

Dr. Hafler would know: With 402 published papers, he is one of the most widely cited living neurologists and has decades of first-hand experience with the complexity of medicine. He also played a major role in identifying the biological processes underlying multiple sclerosis. “Ocrelizumab is a significant milestone in treating MS,” says Dr. Hafler. “What’s exciting about this drug is that it gives us insight into its cause.”

Here’s how it all came together.

Multiple sclerosis is a mysterious disease

In 1868, Jean-Marie Charcot, known as the “father of modern neurology,” dissected the brain of a deceased patient with (what we now think of as) multiple sclerosis and found hardened patches, indicating nerve damage, scattered throughout the white matter of the spinal cord, brain stem and brain. That was when the scientific community began to suspect that the condition, which had baffled physicians because of its constellation of symptoms with no apparent root cause, was a disorder affecting the nervous system. “It was also the first time anyone identified multiple sclerosis as a distinct disease,” says Dr. Hafler, who has a bust of Dr. Charcot sitting in his office.

Read on: Multiple Sclerosis: How Scientists Developed a Breakthrough Drug

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