First Approved Lupus Drug in 50 Years: What Promise Might it Hold?

The FDA’s approval of Benlysta (belimumab) may only offer modest benefit for some patients, but it’s the first new lupus drug to be endorsed in more than 50 years. We tapped rheumatologist and lupus scientist Jennifer Anolik, M.D., Ph.D., for insight into the drug, its promise, and its importance for patients with the systemic autoimmune disease. The disease affects between one and a half to two million people nationwide; 90 percent of are women, who are most often stricken in their childbearing years with symptoms waxing and waning.

Connection: Does this drug approval mark a pretty momentous occasion for you as a lupus researcher?

Anolik: Yes, for a couple of reasons. First, it’s an improvement over our current medications, like steroids, which work by suppressing the immune system in broad fashion – sometimes resulting in infections, bone thinning, even heart disease. Belimumab is different; it’s a biologic medication administered by IV (patients would likely receive one infusion a month, each infusion lasting about an hour), and represents a new wave of “targeted” therapies that home in on a specific molecule and pathway thought to help faulty B-cells survive. Thanks to its targeted approach, belimumab helps keep side-effects to a minimum.  Second, the success of the belimumab trials validates the utility of B-cell targeted therapies, a type of treatment approach that we’ve been studying extensively at the University of Rochester for several years.

Still, though we’re clearly excited, we must rein-in our enthusiasm. Belimumab isn’t a cure; in fact, 30 percent of placebo recipients also reported disease improvement after one year, compared with 40 to 50 percent in the belimumab group. So, while the benefits are real, they work for some subsets of patients studied, but not all, and effects are modest. On top of this, certain types of lupus patients (e.g., those with significant kidney or central nervous system disease) weren’t included in the trials at all. So, we have yet to see how the drug functions in these specific patient populations. 

Connection: What do we know about the drug’s safety?

Anolik: Overall, the drug seems remarkably safe. More than 2,000 patients have participated in trials, some for more than two years. While some patients did experience infections while using belimumab, lupus patients are already at increased risk for infections just by nature of their disease and the other medications they take. So, it’s not clear from the data that belimumab increases one’s risk for infection.

Connection: Why have new lupus drugs been so few and far between?

Anolik: We face a number of challenges while studying lupus. First, the disease itself is incredibly heterogeneous; we often say that “no two patients present alike.” To complicate matters more, the natural history of disease is episodic; symptoms come and go.  So, it’s hard to pinpoint if a drug is really working, or if symptoms are just easing up naturally. Finally, up to this point, we’ve lacked the well-defined outcome measures necessary to objectively say “this drug works, this patient is better.” This is perhaps one of the most exciting things to come out of the big, phase 3 studies that led up to belimumab’s approval; for the first time, they set forth objective measures to prove a lupus drug’s efficacy. What’s more, the FDA has deemed these acceptable measures to use going forward, paving the way for future lupus trials.

Anolik is an associate professor of Medicine and of Pathology and Laboratory Medicine at URMC. She and fellow rheumatologists Iñaki Sanz, M.D., and R. John Looney, M.D., treat lupus patients throughout Western New York and are in hot pursuit of some of the nation’s most promising treatments.

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