Emerging Therapies in Vitiligo

Brett King, MD: Let's move on to segment 4, future directions. This is really an area of a lot of excitement, just because there's so much in development in the vitiligo. And Amit let's start with you. Can you tell us about the drugs that are in development?

Amit Pandya, MD: It's a very exciting time for me. I've been practicing for 30 years, and this is the first time we've had medications that are about to be approved and are being studied for vitiligo up until now we didn't have anything that was approved for vitiligo. Ruxolitinib is the one that's closest to being approved. We've had the completion of the phase 2 in the phase 3 studies, and hopefully, we'll be having FDA approval soon. Ruxolitinib is already approved for atopic dermatitis. It is a JAK 1/2 inhibitor and is very effective in removing the T cells from the skin. In these studies, no phototherapy was given, these patients were simply asked to do their normal daily activities, including driving and walking, and the amount of sun exposure they were getting normally. And amazingly, after 52 weeks of treatment, they got 50 to 75% of the color back on their face. And that's called the FVASI, the facial vitiligo area scoring index. It was very exciting to see that result. It is a study ongoing in which we're going to be combining the topical ruxolitinib with phototherapy, and we expect to see even better results with that one. Ruxolitinib is an oral JAK 3 inhibitor and as a JAK 3 inhibitor, it would have less toxicity than oral JAK 2 inhibitors, which is exciting that has completed its phase 2 studies. And it did show response compared to placebo the response wasn't quite as good as what we saw with the topical ruxolitinib. But that might have had something to do with the type of patients that were enrolled patients with active disease and not patients with stable disease. We're going to have to see what further studies show with ruxolitinib, but that was the first oral JAK inhibitor study that completed the phase 2 study. Upadacitinib is ongoing. It's a JAK1 inhibitor. Again, we think that's going to be safer than other type of JAK inhibitors. And that is right now undergoing its phase 2 studies. We should have the results of that hopefully later this year or next year. And that's exciting to have an oral JAK inhibitor, something that can be ingested rather than something to apply topically. There are other oral JAK inhibitor studies going on, as well as a new pathway, which is the IL-15 pathway. And there's a study with AMD 714, which is a subcutaneous injection, which is given for a certain period in patients with vitiligo. And what it does is it blocks, IL-15 production in the skin, which we think is important for resident memory T cells. When vitiligo occurs and goes away when it comes back, it comes back in the same spot frequently. And that's because resident memory T cells are still there. This strategy by blocking IL-15, it gets rid of the resident memory T cells, we think which will give you a durable remission in that area. An exciting study that's being done, and then future studies are going to be done on IL-15 receptor antibodies. And finally, there's a study going on with a kit. And this kit allows you to isolate melanocytes from a skin sample from the patient's hip, and then apply those melanocytes in drop-like fashion onto an area that you've prepared with dermabrasion or laser, where you can expand the donor-recipient ratio a multiple fold. And that study is ongoing. It has finished enrollment and we should see the results of that later this year or next year. There are a lot of great studies going on with both topical and systemic therapy, and we're going to have a lot more in our armamentarium very soon.

Brett King, MD: It's exciting to think about the idea of we're going to have to become more sophisticated, practitioners, and think much more carefully in a world of multiple choices. David, how do you think about topical versus systemic therapy? What's your algorithm going to look like for that?

David Rosmarin, MD: Currently we don't have a single FDA-approved medication to repigment patients with vitiligo. We have a clear need for both topicals as well as systemics. For those patients who have more limited involvement, a body surface area of maybe of 10% or less will probably be thinking of using our topical options, such as ruxolitinib cream. And for patients who have very widespread disease who may have 30% of their body covered with vitiligo, the oral option may make more sense. Additionally, if a patient has progressive disease, if they're getting new lesions, it's hard to use a topical to chase after new lesions, whereas an oral or a systemic option makes a lot more sense. And a lot of the systemics that we decide and will depend on what is the data that comes out of those studies, the safety, the comorbidities of the patient. And it's certainly a bright future.

Brett King, MD: Amit as you pointed out this is like, just an unbelievably exciting time. Literally, we've gone from having topical corticosteroids, topical calcineurin inhibitors, and phototherapy to a conversation in which we're talking about new topicals that are targeted, new oral agents that are targeted, a subcutaneous treatment that might create a durable remission. It's an unbelievably exciting time. And putting this all together, David, what does the future look like for treating vitiligo?

David Rosmarin, MD: With the first medicines that'll be FDA approved, I'm first hoping that we'll get improved access and recognition from the insurance companies and the payers that this is truly a medical disease not just a cosmetic disease and that patients who want treatment are able to receive it. Furthermore, not all patients have access to phototherapy, or providers who are adept at providing that phototherapy. And being able to give patients topical options pills, whether or not in combination, I think is very promising and to tailor the options to the particular needs of the patient, I think provides us with a lot of hope.

Brett King, MD: I'm glad you brought up the points that you did. We really must and that's part of why we're all here doing this round table is it's really critical that we as providers recognize this disease and recognize that it is a medical disease and treat patients with the respect that they deserve and to try as much as they want to get better, try to make them better. We need to fight as providers for access. We can't just accept the denial. We have to appeal. We have to fight for our patients. And ultimately, in doing this, we can change this disease forever. But ultimately these new treatments coming along are going to create possibility that we've never had before and make the process so much more enjoyable for us as providers and for our patients who are going to get better with these new therapies.

Transcript Edited for Clarity