Throughout 2015, Scleroderma News reported on studies characterizing scleroderma disease subsets, unique features, case reports, potential therapies and strategies for symptom management. As the year comes to an end, here are the top 10 articles that generated the most interest among Scleroderma News readers, as registered by total views. Here are the articles, in ascending order:
Patients with systemic sclerosis that exhibit symptoms associated with at least one other connective tissue disease are diagnosed with systemic sclerosis-overlap syndrome. Researchers from the University of Cologne in Germany showed that systemic sclerosis-overlap syndrome should actually be regarded as a separate subset in systemic sclerosis, as patients with the condition experience a disease course significantly different from patients with limited or diffuse cutaneous systemic sclerosis.
Johns Hopkins University researchers analyzed whether the botulinum toxin could provide relief to people suffering from scleroderma-associated Raynaud’s syndrome, a condition that causes small arteries to narrow, restricting blood circulation. Researchers found that the botulinum toxin can reduce pain and improve blood flow. A Phase 3 clinical trial is currently being conducted to determine whether the botulinum toxin (Botox) can relieve symptoms of Raynaud’s syndrome in scleroderma patients.
Resunab (Corbus Pharmaceuticals), a new potential therapy for scleroderma, received clearance from the FDA as an investigational new drug (IND) for the treatment of diffuse cutaneous systemic sclerosis, the most severe form of scleroderma. Resunab, originally developed as a painkiller, was found to attenuate inflammation. Phase 1 clinical trials revealed that Resunab has a promising safety profile. New trials will determine whether the drug can slow disease progression, improve patients’ quality of life and increase survival.
A study revealed that individuals with systemic sclerosis have a unique bacterial signature in their colon that is different from healthy individuals. Researchers reported that systemic sclerosis patients have a microbiota imbalance, with a marked reduction in bacteria known to supply essential nutrients (commensal bacteria like Bacteroides and Faecalibacterium), and an increase in pathogenic bacteria (Enterobacteriales and Fusobacterium). Scientists believe that investigating this specific microbial signature further could lead to better diagnostic tools and treatment approaches.
A previous trial showed that treatment based on autologous, patient-derived stem cells not only slowed disease progression in systemic sclerosis patients but also reversed it. Furthermore, researchers observed an improvement in lung function parameters and patient quality of life after the treatment. A Phase 3 clinical trial is now underway to evaluate the therapeutic potential of this stem cell-based strategy, especially in terms of changes in skin scores, and lung, kidney, and gastrointestinal functions.
A study reported that patients with systemic sclerosis have reduced respiratory and lower limb muscle strength. Such muscle weakness can decrease exercise performance and patients’ ability to perform daily tasks, leading to decreased functional capacity and interfering with quality of life. Researchers believe that individuals with systemic sclerosis should be encouraged to improve their physical performance, and that the findings may help future interventional studies focused on rehabilitation strategies for these patients.
Although a variety of anti-inflammatory drugs are available to treat systemic sclerosis symptoms, there is currently no cure for the condition. A study suggests the possibility of using human stem cell transplant
ation (HSCT) to treat the disease. The authors reported that scleroderma patients treated with HSCT showed significant improvement in serological markers over 12 months of treatment, a reduction in inflammation and a stabilization of lung function. The authors believe HSCT might represent a promising therapy for systemic sclerosis, although the effects of long-term treatment should be further evaluated.
This study focuses on a new Phase 2a clinical trial enrolling participants to assess the potential therapeutic effects of RayVa™ (Apricus Biosciences) in patients with scleroderma-related Raynaud’s phenomenon. RayVa is a topical, combined formulation of alprostadil, a potent vasodilator, and the company’s patented permeation enhancer, DDAIP.HCl. This on-demand ointment is thought to be able to increase blood flow locally, potentially reducing symptoms of Raynaud’s and subsequent digital injuries.
Similar to article No. 7, this study also reports that patients with systemic sclerosis harbor a unique microbiome in their gut when compared to healthy individuals, which may contribute to their immune dysfunction. Researchers discovered that systemic sclerosis patients’ microbiota are enriched by inflammatory bacteria while protective bacteria were reduced. The team is continuing to study the role of the microbiome in systemic sclerosis in order to understand if the alterations observed are a cause or consequence of the disease. This knowledge will advance the design of potential new therapeutics against systemic sclerosis.
Fatigue can affect a person’s mental and emotional well-being, as well as the ability to work. An online survey revealed that the majority of patients with autoimmune diseases, including scleroderma, experience fatigue and that this symptom is often overlooked by physicians and not properly addressed. Patients reported that fatigue can lead to emotional distress, a sense of isolation, anxiety, and depression. The authors believe that fatigue is a symptom that has been ignored and/or misunderstood by the medical community and the public at large. More research and education are needed to advance understanding on the condition and develop effective treatments.
More encouraging news and discoveries in systemic sclerosis are expected in 2016, especially regarding disease diagnosis, symptom management, and promising therapies such as stem cells. Scleroderma News will continue to report on developments that ultimately contribute to improvements in the lives of patients living with systemic sclerosis.
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