I am a physician-scientist who focuses my clinical and research efforts on vitiligo, and therefore I think about this disease a lot – all the time, in fact. Therefore I thought it would be helpful to share my thoughts with others who are interested in vitiligo as well, particularly the patients who suffer from it and their loved ones. I want to make clear that while I am affiliated with many vitiligo organizations, my comments in this blog are my own, and do not reflect the opinions of those organizations. In addition, my research is largely focused on finding new treatments, and ultimately a cure, for vitiligo. This work is supported by a number of sources, including pharmaceutical companies, which by definition creates potential conflicts of interest. In full disclosure, here is a list of our vitiligo research supporters. Please know that, to the best of my ability, all of my comments are unbiased reflections of my understanding of vitiligo as both a physician and scientist. I do not permit advertisements on my website, and do not endorse companies or products that may advertise on other sites that may be referenced here.
I am excited to announce outstanding results of the first large, randomized clinical trial to test a treatment for vitiligo! For some background, we and a few others reported that the JAK inhibitors tofacitinib and ruxolitinib were effective treatments in a small number of patients with vitiligo. The rationale for using these treatments was based on the cytokine signaling pathways responsible for driving vitiligo. Read more...
Ever since the report from Yale published about a vitiligo patient who improved after using Xeljanz (tofacitinib)and then our report about Jakafi (ruxolitinib) in another patient, there has been a lot of buzz around the use of JAK inhibitors in vitiligo. Many have asked me for updates on these new drugs for vitiligo by commenting below the blog posts, or tweeting, or emailing me, or stopping me in the hall, pulling me aside at conferences, etc. There have been no published reports since these first two, and I have to be careful speaking publicly about ongoing studies, discussions with companies, etc. But I will do my best to update you on the potential for these drugs in vitiligo.
We recently published a report about a vitiligo patient who quickly improved after taking ruxolitinib (Jakafi), a drug that inhibits Janus Kinases (JAKs), which are important for signals that tell the immune cells where to go and what to do. This result is really exciting, because it is an oral drug that had a very rapid effect on vitiligo, improving the patient’s facial pigmentation from less than 1% to over 50% in just 4-5 months. We hypothesized that it might be effective for vitiligo because it blocks a pathway in the immune system that we have found to be critical for vitiligo.
You may have already heard about the recent report by Dr. Brett King at Yale University, about one of his patients with vitiligo who responded to treatment with tofacitinib (brand name Xelganz), regaining much of the pigment in her face and hands within a few months. While only a single patient, this report is indeed very exciting, as it suggests that Jak inhibitors may provide a new effective treatment for vitiligo. We are also excited because Dr. King based his rationale for using tofacitinib in vitiligo on our research, which defined the IFN-γ signaling pathway as critical for vitiligo progression and maintenance. It supports our hypothesis that targeting this pathway would be an effective strategy for developing new treatments.
I am a physician-scientist who focuses my clinical and research efforts on vitiligo, and therefore I think about this disease a lot – all the time, in fact. Therefore I thought it would be helpful to share my thoughts with others who are interested in vitiligo as well, particularly the patients who suffer from it and their loved ones. I want to make clear that while I am affiliated with many vitiligo organizations, my comments in this blog are my own, and do not reflect the opinions of those organizations. In addition, my research is largely focused on finding new treatments, and ultimately a cure, for vitiligo. This work is supported by a number of sources, including pharmaceutical companies, which by definition creates potential conflicts of interest. In full disclosure, here is a list of our vitiligo research supporters. Please know that, to the best of my ability, all of my comments are unbiased reflections of my understanding of vitiligo as both a physician and scientist. I do not permit advertisements on my website, and do not endorse companies or products that may advertise on other sites that may be referenced here.