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Promising new path for refractory rheumatoid arthritis patients?

Genovese - croppedI blogged last week about some important new findings concerning the underlying causes of rheumatoid arthritis. As I mentioned then, it takes a long time - it can easily be a decade or more, if ever - to turn such basic-research results, riveting though they may be, into effective drugs people can actually use.

So it comes as a welcome development that, in a pivotal, large-scale, multicenter phase-3 trial led by Stanford rheumatologist and immunologist Mark Genovese, MD, baricitinib - a new pill being tested for rheumatoid arthritis - substantially relieved symptoms and improved daily physical functioning in patients for whom other therapies had failed. That trial's results are reported in this study in the New England Journal of Medicine.

If you have rheumatoid arthritis or know someone who does, you know how debilitating it can be: pain, stiffness, swelling and eventual destruction of multiple joints, typically smaller ones such as in the hands and feet. (About three of every four people with the disease are, for reasons that remain mysterious, women.)

Over the past 15 to 20 years, there's been a torrent of progress in treating this disorder. Three of the eight top-selling drugs in the United States in terms of dollar sales - adalimumab (Humira), etanercept (Remicade) and infliximab (Embrel) - are prescribed for rheumatoid arthritis. All three drugs work by blocking the action of a substance called tumor necrosis factor or TNF, secreted by various immune cells, that potently stimulates the immune system and spurs inflammation. A number of other drugs also prescribed for rheumatoid arthritis act through different immune-modulating mechanisms.

Here's the problem, as I noted in my news release about the study:

The success of the plethora of drugs now used for treating rheumatoid arthritis carries a downside: Increasing numbers of patients become refractory. The drugs they’re taking no longer provide sufficient benefit, or they produce unacceptable side effects, or both. As a result, Genovese estimates, some 15 to 20 percent of rheumatoid arthritis patients find themselves in the position of having exhausted the current inventory of available medications.

“It’s an ever-growing population,” Genovese told me.

It was precisely these refractory patients who were the focus of the new trial. Drug companies often, understandably, fret about underwriting clinical trials involving such hard cases, for fear of seeing a trial costing many hundreds of millions of dollars, or more, crash and burn. But that's what Eli Lilly and Co., the pharmaceutical giant that sponsored this trial, did. As a result, people who have run out of therapeutic options might, in the not too distant future, find that they've got a new one.

Baricitinib has already been successful in a few other large-scale, late-stage clinical trials in which it performed well in previously untreated patients and compared favorably against both the front-line treatment for rheumatoid arthritis, methotrexate, and the world's top-selling pharmaceutical drug, Humira.

If all goes well, it may not be long before this drug gets licensed by the federal Food and Drug Administration and becomes commercially available.

Previously: Unmasked: Molecular mechanism of rheumatoid arthritis revealed, Is osteoarthritis an inflammatory disorder? New thinking gets clinical test, Rheumatoid arthritis patients fare better than 20 years ago, study reports and important metabolic defect identified in immune cells of rheumatoid arthritis patients
Photo of Mark Genovese by Norbert von der Groeben

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