After a 50-year drought of new drugs and a string of disappointing failures of potential treatments for lupus, researchers said Monday they have found an experimental drug that can ameliorate the symptoms of the life-threatening autoimmune disorder that afflicts as many as 1.5 million Americans.
In unpublished results released by the company, a team from Human Genome Sciences said the experimental drug Benlysta significantly reduced lupus symptoms in a randomized trial of more than 850 patients, reducing their need for debilitating steroids and improving quality of life.
In recent years, at least seven companies or coalitions of companies have reported failed or unimpressive results with potential lupus treatments. Results have been so universally discouraging that, as recently as a week or two ago, market analysts were predicting that Human Genome Sciences’ drug also would fail.
“We have had many disappointments, so for (a drug) to make it through the door with a very significant success rate is an extraordinary accomplishment,” said Margaret G. Dowd, president of the Lupus Research Institute, who was not involved in the research.
Not only does the trial offer lupus patients the hope of a new treatment with fewer side effects, she said, but it also should offer encouragement to other companies with lupus drugs in the pipeline.
“Everybody was getting a little discouraged,” added Dr. Betty Diamond, chief of the autoimmune disease center at North Shore-LIJ Health System’s Feinstein Institute for Medical Research in Manhasset, N.Y., who also was not involved in the research.
“It’s very exciting to finally have a trial in lupus showing efficacy,” said Diamond, who is an adviser to the Lupus Research Institute. “We’re going to have to work with it longer to understand when it should be used and exactly who is going to benefit from it … but we are very pleased to have a drug that looks as though it really did do something.”
Experts cautioned, however, that the results have not been peer-reviewed or published in a journal, so it is difficult to assess their significance. Human Genome Sciences said the results would be presented at a meeting later this year and published after the second study is completed. A spokesman said the robust nature of the data and the 50-year drought in drugs justified releasing the data now.