And Ethical? Can you not treat a control group that may have a known thrombotic risk? Standard treatment for those who have already had a thrombotic event is warfarin, used long term, or perhaps throughout life. APS in pregnancy is routinely treated with heparin, and for those with only aPL detected—it depends who you ask. “One of the things we’re seeing is that people don’t necessarily need to receive a medication if they have antibody only. I believe that ethically considered control groups are entirely possible.” says Dr. Lockshin.
Explore this issueMarch 2011
For any trial, what Dr. Lockshin most wants to see is progress towards moving upstream: elucidation of the Novel. He wants to see the mechanistic studies that will point the way toward disease prevention. “What we’re doing right now is at the very end of the stream, anticoagulating. It’s preventing damage, but if we could get in much before that, before there’s any clotting occurring at all…” That’s the ACTION to take.
In a field that has few if any corporate sponsors (there are no anti-APS agents in any pipeline) funding is key, and in regards to APS ACTION, funding is personal, which helps answer the question: Why now? “A benefactor came along,” says Munther Khamashta, MD, PhD, director of the lupus research unit at King’s College London School of Medicine at St. Thomas’ Hospital in London, and APS ACTION Executive Committee Member. “For the last ten years we’ve been talking about international collaboration but we never had the clear opportunity to do it until this benefactor appeared—this person who had a family member who suffers from this disease—and what she wanted to know was this: Why don’t international experts talk to each other?” APS ACTION was the answer, and was supported by this funding.
Dr. Khamashta, based in the U.K. and having been in the APS field since its inception, can appreciate the obstacles to this international endeavor. Foremost among these is assay standardization. “This is a huge hurdle,” he says, going on to bluntly explain the reason: the inventor of the aPL test failed to get a patent. “Huge mistake. So, because it’s not a patented test, anyone can copy the recipe. This is why we have 50 different kits available in the market today without any kind of standards control.” Therefore, the first job of this international initiative, Dr. Khamashta says, has to be a standardization of the test. “Think about it, what is positive for me in the U.K. could be negative in New York, and borderline in Sydney.” That’s no way to treat, let alone do clinical research.