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Breakthroughs offer hope for vitiligo patients

June 22, 2023 | UCI Health
Dr. Anand K. Ganesan, left, and Dr. Jessica Shiu are UCI HEalth dermatologists whose research efforts are aimed at reversing vitligo, an autoimmune skin disease that causes white patches where it has destroyed pigment-making cells., leaving patches   are conducting research llaborators Jvitiligolabganesanshiulab01264x291
Skin specialists Dr. Anand K. Ganesan and Dr. Jessica Shiu are thrilled that breakthrough vitiligo treatments tested at UCI Health are now available for the disease, which causes white patches where the body's immune system has attacked pigment-making cells. Photo for UCI Health

When Dr. Anand K. Ganesan started a vitiligo specialty practice in 2018 at the UCI Health Beckman Laser Institute & Medical Clinic, it was to find new therapies to reverse the disfiguring skin disorder.

Vitiligo — pronounced vit-ill-EYE-go — causes white patches, often on the face and hands, the result of the immune system destroying the skin’s pigment-producing cells.

“All we had at the time was light therapy and topical steroids, which were minimally effective,” says Ganesan, a UCI Health dermatologist and vitiligo expert who was keenly aware of the emotional and psychological toll the disease had on his patients, many of them vulnerable young children and teens who withdrew from peers rather than risk ridicule or worse.

Today, there has never been more hope for patients with the autoimmune disease, which affects an estimated 70 million people worldwide, at least 25% of them children, says the UCI School of Medicine professor of dermatology and biological chemistry.

New options

A topical cream called ruxolitinib is the first therapy approved by the U.S. Federal Drug Administration (FDA) to restore pigment in vitiligo patients. The FDA also recently gave breakthrough device approval for RECELL®, a one-time therapy using the patient’s healthy cells to stimulate lasting repigmentation in stable vitiligo, the regenerative medicine company Avita Medical announced June 16. Other topical, oral and injectable medications also have shown success at halting progression of the disease in early phase trials.

Ganesan and his team of clinicians and researchers have been directly involved in studying all these therapeutic breakthroughs. In addition, they used genomics and UC Irvine’s powerful microscopy resources for a novel study that reveals how immune cells, pigment-making melanocytes and keratinocytes — cells that give skin its structure — interact to maintain depigmented areas. The study was led by UCI Health dermatologist Dr. Jessica Shiu, who, along with Ganesan, is among a handful of U.S. physician-scientists to conduct National Institutes of Health-funded vitiligo research. 

“We have made a lot of progress, but there is still a lot more work to be done,” says Ganesan, who recently was honored by the American Skin Association for his pioneering research on vitiligo.  “While many of these new therapies work for patients with disease on the face, they don’t work well on other parts of the body, especially the hands, which is equally distressing for patients.”

Raising awareness and support

As international experts, patients and prominent advocates for improved vitiligo care converge in Atlanta this week for a three-day conference to mark the 13th annual World Vitiligo Day on June 25, Ganesan and his team have launched a campaign to raise awareness about the disease, fund a dedicated dermatology fellowship and provide patients with additional resources to cope with their condition.

Learn how to support vitiligo care and research at UCI Health ›

Ganesan first began treating vitiligo patients as a dermatology resident at University of Texas Southwestern Medical School in Dallas under the guidance of internationally regarded vitiligo expert Dr. Amit Pandya. Armed with a PhD in microbiology and molecular genetics as well as a medical degree, the young resident was already interested in melanocytes, cells found in skin and hair that produce pigment.

“Vitiligo is unique,” he says. “In other skin diseases like psoriasis or eczema, you treat the autoimmune component to stop the body from attacking the cells and the disease resolves itself. I was struck by this disconnect. Patients with vitiligo had melanocytes but they weren’t working to repigment the white patches.”

More than a skin disorder

Equally distressing is the disease’s effect on emotional and mental health. “When patients first come to me, many of them are down, dejected because they’ve been told no effective treatments exist for their disease,” he says. “This is why advocacy is so important. Both physicians and patients need to know that viable therapies are available.”

An estimated 30% to 50% of vitiligo patients suffer from depression, which in rare cases may even result in suicide attempts. It’s one of the reasons Ganesan helped launch a UCI Health vitiligo support group, to provide a safe space for patients to share treatment experiences and discuss coping strategies for daily living, such as makeup and clothing tips. To understand the impact of the disease, he recommends viewing the 2016 video “Vitiligo: Truth Hope and Change,” which was filmed in part in Orange County with support from UCI.  

“When people actually start getting better, their perspective changes,” he says of his patients, a number of whom participated in the vitiligo trials and have seen improvement, “It’s the most rewarding thing for me, to see that spark come back in their eyes.”

Hand spots harder to treat

The new topical treatment restores pigment on the face in about 60% of patients, although it isn’t as successful for the rest of the body. “Hands in particular don’t seem to respond to any of the newer therapies, which is very distressing to the patient,” he says.

New oral therapies also show promise in stopping disease progression. These treatments, which target the Janus kinase signaling pathway, suppress the body’s T-cell defense systems and may result in long-term side effects.

“It’s not perfect,” he says of the new oral therapies. “But it’s a start and hopefully we can improve on them with more research.”

Great strides also have been made in skin-grafting techniques and light therapy to stimulate repigmentation. The RECELL treatment involves harvesting the patient’s healthy melanocytes from a small amount of skin, which is processed to create a solution of skin cells that are applied to white patches of skin prepped by laser ablation. The treated area is then covered with a dressing that allows the solution to “stick” to the grafted site.

Studies showed that more than a third of RECELL participants saw greater than 80% repigmentation 24 weeks after treatment, while over half of patients saw greater than 50% repigmentation over the same period.

Making treatment more accessible

The hope is that RECELL will become a procedure any dermatologist could perform with very limited training, Ganesan says. “It does require a laser to ablate the skin, but it’s a laser that most dermatologists already have in their office.”

Light therapy, too, has become more accessible. Treatments that required intensive daily or weekly office visits now can be done at home. “We give patients a light box and teach them how to use it,” he says. “Light therapy treatments are really hard on children because you have to go into the box two or three times a week.

“One patient I’ve been treating since she was about 8 years old is a teenager now. Being able to do the treatment at home has been a godsend!”

With the white patches gone from her face and about 60% to 70% of her body, he says she is now an outgoing student who participates in school and extracurricular activities with confidence.

Ganesan has a lot to share at the next quarterly vitiligo support group meeting, which went on hiatus during the COVID-19 pandemic.“We want to let our community know about the exciting new treatments available, about our research and how we are expanding our research team,” he says.

“One of the most fulfilling things we do is work with our patients to learn how they are affected by the disease. We use that information to design better treatments for them.” 

Anand K. Ganesan, MD, PhD, is a board-certified UCI Health dermatologist who specializes in skin cancer and skin pigmentation disorders, including vitiligo, melasma and albinism.

A prolific physician-scientist, Ganesan explores how melanocytes respond to environmental cues, such as ultraviolet radiation and inflammation, to maintain normal homeostasis and how that homeostasis is disrupted by diseases processes, including melanoma and vitiligo. His work spans the spectrum from basic laboratory studies to clinical trials as he seeks to develop novel approaches to diagnosing and treating disease.

He is a professor of dermatology and biological chemistry at the UCI School of Medicine, where he serves as associate dean for physician-scientist development. He also co-directs the Chao Family Comprehensive Cancer Center’s programs in Biotechnology, Imaging and Drug Development and Molecular Diagnostics and Therapeutics. He is the author or co-author of dozens of peer-reviewed publications, including the recent https://www.ucihealth.org/news/2022/04/uci-researchers-discover-promising-new-molecule-for-cancer-therapy" discovery of a new class of drugs to potentially treat melanoma and other cancers.

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