David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic neuroendocrine cancer (PNET) — the same disease that killed Apple’s founder and CEO Steve Jobs. After three years of progress…
Immunosuppressive therapy followed by an autologous stem cell transplant resulted in significant long-term benefits, including improved overall survival, in adult scleroderma patients compared to immunosuppressive therapy alone, according to the results of a Phase 2/3 trial. Despite a higher rate of treatment-related deaths in the transplant group, researchers argue…
Fibrocell Science has filed an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for FCX-013, its gene therapy candidate for moderate to severe localized scleroderma. An IND is a request for FDA authorization to administer an investigational therapy to humans. It is generally…
Should their doctors agree, breast cancer patients with scleroderma may undergo radiation therapy — despite research suggesting it increases the risk of scarring in the skin, lungs and other organs, a team of American scientists has concluded. The previous studies’ small sample size — fewer than four patients — makes…
This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…
New research establishes a link between immune cells, known as macrophages, and systemic sclerosis progression. The study showed that gene expression in macrophages from systemic sclerosis patients is altered, including higher activity of the susceptibility gene GSDMA. The study “Changes in macrophage transcriptome associate with systemic sclerosis and mediate GSDMA contribution…
A data safety monitoring board (DSMB) has recommended that Inventiva continue its Phase 2b clinical trial assessing lanifibranor (previously known as IVA337) for the treatment of diffuse cutaneous scleroderma, after reviewing data gathered in the study to date. The trial, which is now fully enrolled with 145 patients, compares lanifibranor to a…
Results from a Phase 2 clinical trial shows that stem cell transplants can provide better long-term benefits than the standard treatment for patients with severe scleroderma. Myeloablative autologous hematopoietic stem cell transplant, which uses high doses of chemotherapy and/or radiation before the patient’s own stem cells are transplanted,…
Researchers have discovered that plasmacytoid dendritic cells (pDCs) contribute to the skin scarring in scleroderma, making them a possible target for treating the disease. The study, “Plasmacytoid dendritic cells promote systemic sclerosis with a key role for TLR8,” was published in the journal Science Translational Medicine.
Organ fibrosis in scleroderma patients might be reversed with treatments that allow fibrotic cells to self-destruct, Harvard scientists suggest. Their study, “Targeted apoptosis of myofibroblasts with the BH3 mimetic ABT-263 reverses established fibrosis,” appeared in the journal Science Translational Medicine. It demonstrated that the survival–self-destruction balance of fibrosis-producing myofibroblast cells…
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