Tofacitinib: A Promising New Drug for SS Patients



In 2001, Amy was just 19 years old when she began exhibiting symptoms of systemic sclerosis (SS), or scleroderma. Her fingers began turning blue, she started to get severe ulcers on her fingertips, and she developed scarring on her lungs. However, being diagnosed with diffuse systemic sclerosis didn’t overtake her life, but redefined it. In her blog on Cloudy With A Chance of Pain, she shares how she’s attended conferences and been part of commercials to spread awareness and gain knowledge about her disease.


Impacting around 100,000 people in the United States each year, scleroderma is “a rare rheumatic disease that affects connective tissue and the vascular system producing excessive collagen that causes fibrosis in internal organs.” There is no known cure, identified cause, and licensed treatments for SS. However, researchers at Michigan Medicine and the University of Pittsburgh, which is where Amy often went to receive treatment, conducted a study funded by Pfizer and have found promising results for a new drug that could aid early SS patients.


Tofacitinib was synthesized as a drug for rheumatoid arthritis, and the researchers wanted to see how tofacitinib would affect SS patients and how the drug would affect skin tissue single-cell RNA transcription. The study had 15 early SS patients, with 10 given Tofacitinib and 5 the placebo. Over 24 weeks, the mRSS (modified Rodnan skin score) and gene expression in fibroblast and keratinocyte subpopulations showed marked improvement for the experimental group, and after the placebo group was given the drug, similar results emerged. Additionally, researchers found that tofacitinib did not inhibit T-cells, which are white blood cells fundamental to the immune system.


As researchers continue to discover more and more about systemic sclerosis, it is important to recognize that these innovations are helping hundreds of thousands of people across the world. Unfortunately, there was no update from Amy’s blog post in 2017, but the conclusions made from this study are a promising look into what licensed treatments for SS could look like and how people like Amy can find relief from SS.


Thank you for reading,

Adeba Mukul


References:

https://www.cloudywithachanceofpain.com/blog/amy-sc-story

https://labblog.uofmhealth.org/lab-report/tofacitinib-shows-promise-scleroderma-patients-researchers-are-optimistic-for-next-phase

https://insight.jci.org/articles/view/159566

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