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How much progress has been made on vitiligo research?

 Posted on 15th March 2023  5 minute read

One of the questions we as vitiligo patients often pose ourselves is what the current state of research progress for treatments of the skin condition is – is it garnering the medical attention it deserves and can we expect new developments in the upcoming years. We aim to answer these questions through a dissection of recent research articles which include the history of vitiligo translational research and recent advances , the first FDA-approved pharmacological treatment to address regimentation and other promising treatments on the way.

What is Vitiligo?

Vitiligo is a chronic autoimmune condition that causes white spots on the skin due to a lack of functionality in pigment producing cells called melanocytes. Although vitiligo does not lead to scarring skin injuries or other long-term physical conditions, it can have a profound relevant effect on the psychological condition and social quality of life of the affected people.

Most of the pharmaceutical drugs under development work by interfering with the immunosuppressive process, given since the autoimmune attack against melanocytes in causes this disorder. The main etiology of vitiligo is associated with the failure of immune tolerance but its pathogenies are considered multifactorial and include dysregulation of adaptive immunity, innate immunity, and genetic factors [1] .

Opzelura – an FDA approved treatment for Vitiligo

Targeting the Janus-activated kinase (JAK) seems to be the most promising treatment. JAK inhibitors are normally used to treat chronic inflammatory disorders such as rheumatoid arthritis, psoriatic arthritis and atopic dermatitis. After specific clinical trials where vitiligo patients were recruited ( NCT04052425 and  NCT04057573 ), the first topical treatment for vitiligo is finally available in US.

In both trials, subjects were treated with Opzelura or placebo cream twice daily for 24 weeks, followed by an additional 28 weeks of treatment with Opzelura for all subjects.

At the end of the 24-week treatment period, 30% of Opzelura patients had at least 75% improvement in the facial Vitiligo Area Scoring Index, compared with 10% of placebo patients [2]. Opzelura has since been approved by the FDA in 2022.

Opzelura is a topical Janus kinase (JAK) inhibitor currently approved for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis and approved cream for the treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older.

Can Opzulera be used to treat segmental vitiligo?

We spoke to Dr John Harris, Director of the Vitiligo Clinic and Research Centre at UMass Chan Medical School, who explained:

Segmental [vitiligo] is harder to treat because it turns the hairs white quickly. Clinical trials usually exclude people with segmental vitiligo because of this, they want to have the best chance of showing if their drug works. When a drug is approved it is only approved for the type of disease tested in the clinical trials, so that’s why Opzelura is approved only for nonsegmental vitiligo. Opzelura seems to work well in segmental vitiligo if the hairs are still their normal color, and doesn’t work well in any vitiligo where there isn’t hair growing, or most of the hairs are white.

Other Potential Treatments

Among the new approaches being developed, the strategy of targeting Cytokine has been clinically tested and those inhibitors are considered promising options too. Other emerging therapeutics targeting still under evaluation and not yet clinically tested are MicroRNA-based therapeutics (miRNA) and T-cells (Tregs) that are regulating or suppressing other cells in the immune system [3] . Studies were conducted on the migratory capacity of melanocytes that was altered by the application of microRNAs. While a clear decrease in Treg cells was observed in vitiligo skin within lesional, non-lesional, and perilesional sections, indicating that increasing the number of Tregs with normal function might be an important therapeutic intervention for vitiligo treatment [3] . Other studies were conducted to evaluate the effect of the application of topical 5-fluorouracil after micro-needling excimer light (308 nm) application in the treatment of non-segmental vitiligo. The results of the study were promising and confirmed that the combined therapy is faster in inducing re-pigmentation [4] .

State of Research

The One consideration that can be done made by when looking at the different papers available on vitiligo is that the number of studies grew has grown exponentially over the past 40 years up to the approval in 2022 of the first drug for the treatment of vitiligo (Figure 1).

One of the key assets for vitiligo was the long history of studying vitiligo using translational methods that is an interdisciplinary field that has the aim to fill the gap between clinical and basic research and to develop a more complete picture of the disease pathogenesis by applying an open-minded approach among researchers, pharmacologists, and clinicians. Translational studies have been great assets particularly useful for understanding the mechanisms of disease, which promoted the development of promising new treatments [5] .

2022 brought important news and promising results on the treatment of vitiligo. Our wish for the coming years is that research activities conducted with an open-minded approach will not stop and that effective and safe treatments for this disease will soon be available to support all patients. With this wish in mind, we will aim to monitor ongoing research and provide the latest updates in a digestible format in this article series.


  1. Marchioro HZ, Silva de Castro CC, Fava VM, Sakiyama PH, Dellatorre G, Miot HA. Update on the pathogenesis of vitiligo. An Bras Dermatol. 2022;97(4):478-490. doi:10.1016/j.abd.2021.09.008
  3. Feng Y and Lu Y (2022) Advances in vitiligo: Update on therapeutic targets. Front. Immunol. 13:986918. doi: 10.3389/fimmu.2022.986918
  4. Saad MA, Tawfik KM, Abdelaleem HL. Efficacy and safety of micro-needling combined with topical 5-fluorouracil and excimer light vs.excimer light alone in treatment of non-segmental vitiligo: A comparative study. J Cosmet Dermatol. 2022;00:1-12. doi: 10.1111/jocd.15481
  5. Katz EL and Harris JE (2021) Translational Research in Vitiligo. Front. Immunol. 12:624517. doi: 10.3389/fimmu.2021.624517

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