Drugs Showing Promise in Cancer Trials Down Scarring in Scleroderma

Drugs that have shown promise in cancer trials significantly reduce scarring in the cells of patients with scleroderma, as per the new study.

In a recent study, Michigan Medicine researchers focused on BETs, which are proteins that regulate gene expression by binding to modifications on proteins around which DNA wraps, a process called epigenetic regulation.

‘BRD4, known to play a role in cancer, also affects fibrosis in scleroderma. BRD4 inhibitors stopped scarring in both human-derived cells and in animals.’
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Drugs targeting BETs, specifically an isoform called BRD4, have been developed by various pharmaceutical companies for cancer treatment.

Results published in JCI Insight reveal that drugs that inhibit BRD4, known to play a role in cancer, also affect fibrosis in scleroderma.

Testing on Scleroderma Patients

Researchers tested BRD4 inhibitors on the skin fibroblasts of scleroderma patients and in mouse models of skin fibrosis. They found that the treatment stopped scarring in both human-derived cells and in animals.

The inhibitors used by Michigan Medicine researchers have shown promise for treating various cancers in preclinical studies. Specifically, one drug used in the recent study, called AZD5153, is being tested in Phase I clinical trial for sarcomas and lymphomas.

“Through this study, we have uncovered a new class of epigenetic drugs that can be used in scleroderma fibrosis,” said Pen-Suen Tsou (Eliza), PhD, senior author of the paper and a rheumatology researcher at Michigan Medicine.

“If we can repurpose these drugs and get them through development more quickly, we can provide faster relief for patients who struggle with debilitating symptoms of this autoimmune disease. The process can typically take around 10 years, but our patients cannot wait that long.”

The study is a collaborative effort with Michigan Medicine’s Scleroderma Program. Tsou’s team also found that a calcium signaling protein, called CaMKII, affects fibrosis in scleroderma, which researchers had previously not seen.

“Right now, we are doing some follow-up studies to see if inhibitors of this protein can block scarring for scleroderma,” Tsou said. “This opens up a brand-new direction for us to offer a novel target for this disease.”

Scleroderma - CREST Syndrome - Signs - Symptoms - Diagnosis - Treatment - Prognosis
Scleroderma or CREST syndrome is a chronic, auto immune disease which manifests as thick, dry, fibrous skin. Scleroderma/CREST syndrome is classified as rheumatic and connective tissue disease.

Source-Medindia