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A Phase 2 trial will explore effect of AM1476 on skin thickness, lung function by Andrea Lobo, PhD | February 6, 2024  AM1476, AnaMar’s investigational anti-fibrotic medication, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for treating scleroderma.
To get this designation, a medication must be intended to treat a life-threatening rare disease that affects fewer than five in 100,000 people in Europe or under 200,000 people in the U.S. The designation provides certain incentives, including regulatory fee reductions and the potential for extensive market exclusivity — seven years in the U.S. and 10 in the European Union — if approved. The company plans a Phase 2 clinical trial to evaluate the treatment in people with scleroderma and interstitial lung disease (ILD), a group of diseases marked by lung scarring. The study will evaluate AM1476’s effects on skin thickness and lung function in 60 patients with diffuse cutaneous scleroderma and ILD over a year. “This is a significant milestone and underscores the significant unmet need for novel medicines to prevent, heal, and slow organ scarring from fibrotic diseases, which are often progressive and can have a poor prognosis,” Ulf Ljungberg, PhD, AnaMar’s CEO, said in a company press release. Scleroderma, also known as systemic sclerosis, is marked by inflammation and fibrosis, or uncontrolled tissue hardening and scarring, in the skin and even the heart, kidneys, and lungs. Up to 80% of people with SSc may develop ILD, which leads to reduced lung function and breathing problems. Spike promotes infiltration of pro-inflammatory cells in mouse model by Andrea Lobo, PhD | January 30, 2024  SARS-CoV-2, the virus that causes COVID-19, may accelerate the development and progression of systemic sclerosis (SSc) through fibrosis (scarring), inflammation, autoantibody production, and blood vessel damage.
That’s according to a study in South Korea that shows the SARS-CoV-2 spike protein that’s required for the virus to infect human cells promotes the infiltration of pro-inflammatory cells in the skin and lung tissue of an SSc mouse model. The study, “SARS-CoV-2 spike protein accelerates systemic sclerosis by increasing inflammatory cytokines, Th17 cells, and fibrosis,” was published in the Journal of Inflammation. SSc is an autoimmune disease that features scar tissue accumulating in the skin and potentially the internal organs, including the heart, kidney, lungs, and digestive tract. The disease is thought to develop due to excessive scarring, autoantibodies being produced that mistakenly attack healthy tissues, and damage to small blood vessels. COVID-19 induces the overactivation of immune cells, inflammation, autoantibody production, and bleeding disorders, similar to mechanisms in SSc and other autoimmune diseases. “While there is a single case report suggesting an association between COVID-19 and SSc, the effects of COVID-19 on SSc are not yet fully understood,” wrote the researchers who used human embryonic kidney cells that were manipulated to produce the SARS-CoV-2 spike protein and the ACE2 receptor, a protein to which the virus binds to enter cells. The spike protein is also known to induce fibrosis, the investigators said. Its presence significantly increased the activity of proteins associated with fibrosis, namely alpha-SMA and Col1a1. Patients in interview study talk of the various life changes they have to confront by Marisa Wexler, MS | January 23, 2024  Living with scleroderma requires adapting to changes in a woman’s sense of self, but practicing gratitude and accepting change can help patients reclaim themselves, a study aiming for a “grounded theory” of identify management reports.
Its scientists said these findings may lay the groundwork for future studies aiming to develop interventions to help people with scleroderma hold on to their sense of self while navigating life with the progressive disease. The study, “Process of Maintaining Self in Individuals Living With Systemic Sclerosis: A Grounded Theory Study of American Women,” was published in the Western Journal of Nursing Research. Flow-mediated dilation 'helpful tool in the overall assessment of vascular injury' by Lindsey Shapiro, PhD | January 16, 2024  A non-invasive technique called flow-mediated dilation (FMD) could be useful for monitoring blood vessel alterations in people with systemic sclerosis (SSc) and predicting the likelihood of certain blood flow (vascular) complications, a study has found.
The test findings show distinct differences between SSc patients and healthy people, and were more altered in SSc patients who had been living with the disease longer and had certain vascular symptoms, including pulmonary arterial hypertension (PAH). This correlated well with the degree of damage seen in other tests of vascular damage as well as blood biomarkers of the endothelial cell dysfunction thought to drive blood vessel changes in SSc. The study, “Flow Mediated Dilation in Systemic Sclerosis: Association with clinical findings, capillaroscopic patterns and endothelial circulating markers,” was published in Vascular Pharmacology. Endothelial dysfunction drives blood vessel damage, blood flow changes Alterations to the endothelial cells that line blood vessels — called endothelial dysfunction — drive blood vessel damage and blood flow changes in SSc. As a result, patients may experience certain vascular complications such as Raynaud’s phenomenon, PAH, or digital ulcers. Small blood vessels (i.e. capillaries) as well as larger vessels (i.e. veins and arteries) in patients may be affected, known as microvascular and macrovascular damage, respectively. Microvascular damage is more common and better characterized. These changes can be assessed using a technique called nailfold video-capillaroscopy (NVC), where the capillaries under the skin near the fingernail beds are visualized under a microscope. A method for detecting macrovascular damage is not as well established. FMD, which looks at an artery’s diameter in response to blood flow restriction, could be one way to do this, but it is not yet widely used in clinical practice. |
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