Tuesday, November 27, 2018
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I’m writing this post in response to multiple exciting and generous requests to help support our research effort to find a cure for vitiligo. I have been touched by the unsolicited outreach by readers all over the world who are just as passionate as we are about improving the lives of tens of millions with vitiligo. In response, we have set up a user-friendly and safe method to donate to support our work. At the end of the blog post there is a link to do just that. I’ll also outline what we have done so far with donor support, and tell you where we’d like to go in the future if we have sufficient resources to do it. You can help!
Over the past 8 years, our team has grown from just 2 scientists (me plus a research technician) to currently more than 15, made up of trainees from all over the world who all are contributing their expertise and passion to finding better treatments for patients with vitiligo. We have attracted the best and brightest from the US, China, Japan, Egypt, Pakistan, and other locations to work with us, and the lab is buzzing with new ideas and excitement every day. They know that their work is important, and they take their task very seriously. At the same time, we have fun and learn from each other, appreciating our differences but also finding camaraderie in our common cause.
We initiated one important project about 4 years ago that was not supported by National Institutes of Health (NIH) or other government funding – a project that was funded by private philanthropy and focused on learning why successfully treated vitiligo spots return to the exact same locations after stopping the treatment. In those studies, we discovered that autoimmune memory T cells form in the skin and reinitiate the spots, and we learned that those cells were dependent on a protein called IL-15 that is expressed in the skin. Long story short, when we blocked that protein with a new treatment we were able to remove those memory cells, and observed long-lasting reversal of the disease in mice with vitiligo. Human studies mirrored our observations in mice, and we are excited to report that we have received NIH funding to initiate a clinical trial to test this treatment in patients starting next summer, 2019. You can read more about that study, its results, and impact here:
https://www.umassmed.edu/vitiligo/blog/blog-posts1/2018/08/science-translational-medicine-journal/
https://theconversation.com/new-treatment-in-the-works-for-disfiguring-skin-disease-vitiligo-100058
We also were fortunate to have private philanthropic support for a new project to discover the immune cells that are present in the skin and either cause the disease or work to suppress it. To do this, we used a very new, but expensive, technology called single cell RNA sequencing, and applied this technology directly to cells that we get from affected patient skin. We recently developed a new method to sample patient skin using suction blisters, which opens up a completely new opportunity for these studies, and we started this project by looking at the skin in just 2-3 vitiligo patients to determine whether this cutting-edge technique would be useful for this purpose. This early data, funded by contributions from supporters, was very exciting and allowed us to secure a $1.25M grant from the NIH to expand our studies into more patients. So far, we have analyzed the skin of many patients with vitiligo and healthy controls, with astounding resolution and exciting results. The “good problem” that we have now is that we have many more ideas to develop improved treatments, and need to redouble our efforts to pursue them, which will require significantly more support and resources to do this efficiently.
We believe that these studies will transform our understanding of how the immune system causes vitiligo, and that the “cure” for this disease may be found in the data that we have already collected. However, we now must conduct many more studies to find the key elements in this data that will lead to the cure. Currently, it is a bit like finding a needle in a haystack, and we really need “all hands-on deck” to make this work. We will eventually accomplish this, either with our current small team of scientists over a long period of time (20-30 years), or with a larger team and expanded resources that will allow us to do this much more efficiently and quickly (5-10 years).
Our next priorities include hiring scientists to join the team who would be tasked with using this existing data and distilling out the most promising targets for therapy. Sufficient support will also allow us to use the best tools available for this work, which are often out of reach on our current budget. If you would like to be a part of what we’re doing, please consider clicking on the link and donating whatever you can to help us. I will write periodically to update you on our progress and hopefully give the good news someday that we have identified a cure. Join us!
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