Research into vitiligo first started over 2000 years ago, but sadly over the past 50 years. there have not been even a handful of clinical trials involving vitiligo. Thankfully this is now changing. Due to the availability of more advanced research equipment, a greater interest from Pharmaceutical companies, and the work undertaken by vitiligo specialty clinics around the world (such as The Vitiligo Centre Australia), the interest and pace of research is increasing. As a result of global collaboration between vitiligo scientists and Dermatologists, the next 5-10 years promises to be the most rewarding in the search for better treatment and potentially finding a cure for vitiligo
Vitiligo research is undertaken in three ways:
- Basic research is where one tries to find out what is happening at the molecular or cellular level in vitiligo. Basic research takes place outside of the human body (in-vitro research) with cells in a dish or on animal models, such as mice. Basic science is the most time-consuming of the types of research, as elucidating an important vitiligo cell component or disease mechanism can take years even decades.
- Translational research then aims to take the findings of basic research (that the scientists discovered) and translate it into something that might eventually be used in a clinical (real life) setting. A lot of translational research will also take place in animal models which can provide a valuable tool for testing different therapies before moving into humans. Translational research may also be undertaken with humans where it involves taking blood and skin samples for analysis and testing.
- Clinical research is the last step, in which you validate a new drug or therapy in real vitiligo patients, looking at effectiveness and safety, often comparing the results to other existing treatments. Clinical research can take place within a specialist centre (such as The Vitiligo Centre Australia) or take place globally in specially designed Clinical Trials involving multiple specialised centres.
Clinical Trials are comprised of three phases. In a phase 1 clinical trial, the tolerability and safety of the new drug is studied, usually in a small number of healthy volunteers. Phase 2 is aimed at determining the drug’s efficacy and optimal dosing regimen in a small number of vitiligo patients. After phase 2, the drug is entered into the next phase of testing referred to as a Phase 3 trial. In a phase 3 trial, the main focus is to demonstrate and confirm the preliminary evidence gathered in the previous trials that the drug is, a safe, beneficial and effective treatment for vitiligo. Phase 3 is the last phase of testing to be completed before the drug can be considered by a countries regulatory authorities for approval and release. Phase 3 trials are often multicentre worldwide trials involving up to 3,000 participants.
New clinical trials for vitiligo are in progress (Phase I and II) and more are in the pipeline. To understand what current clinical trials are trying to achieve, firstly imagine the immune system as a huge tree. Now picture the part of the immune system responsible for vitiligo as being a small branch at one end of that tree. Ideally, we want to switch off vitiligo by finding a drug that cuts (or trims) only that small branch at the end of the tree responsible for causing vitiligo, instead of using a drug that may damage/cut the trunk of the tree which would suppress the vitiligo patient’s entire immune system. Thus, the search is on for a drug that works precisely on the problem area (the tree branch) without the unwanted side effects associated with suppressing the entire immune system (the tree trunk)
Vitiligo patients can find out about clinical trials at https://clinicaltrials.gov/
The Vitiligo Centre Australia looks forward to being involved in future Phase 3 clinical trials and will keep our vitiligo patients and their families updated on future research developments.