Localized scleroderma is a subtype of scleroderma, a chronic, autoimmune skin disease. It is characterized by excessive production of collagen, the body’s main connective-tissue protein. This over-expression results in a thickening of the connective tissue and the skin.
In addition to skin symptoms, patients with scleroderma often experience an exaggerated reaction to cold or emotional distress. This can include numbness, pain, or color alterations in the fingers and toes, as well as acid reflux, other digestive system problems, and impaired functioning of the heart, lungs, and kidneys.
Scleroderma can affect growth and development in children due to lesions in the joints that can be painful, affect motion, and be permanent. In the United States, nearly 200,000 people have localized scleroderma. About 40,000 have a major joint affected by the disease, or experience severe joint pain.
There is no cure for scleroderma. In the case of localized scleroderma, the treatment options available — including corticosteroids applied to the skin or taken orally, ultraviolet light therapy and physical therapy — are focused on relieving the symptoms.
How FCX-013 Works
FCX-013 is an investigational compound classified as an autologous fibroblast cell therapy. Fibrocell is developing it in alliance with Intrexon. The drug modifies patients’ genes in order to increase the expression of a protein that breaks down excess collagen.
Fibroblast cells are the most common cells in skin and connective tissue, and are responsible for the production of collagen. In patients with scleroderma, the cells produce collagen in abnormally elevated amounts because the body assumes there is an injury that needs to be repaired with collagen.
Medical practitioners collect fibroblast cells from a patient, culture them in a lab and genetically modify them to regulate collagen accumulation. They are then injected at the site of the disease.
FCX-013 uses a biologic switch activated by an orally administered compound, Intrexon’s proprietary RheoSwitch Therapeutic System. Medical practitioners use the system to control future protein expression once the initial fibrosis is resolved.
FCX-013 for Scleroderma
FCX-013 is in pre-clinical-trial development for the treatment of linear scleroderma, a form of localized scleroderma. The drug received Orphan Drug designation in April 2016 from the U.S. Food and Drug Administration (FDA). Orphan Drug status helps companies advance the development and testing of a drug. It means that FCX-013 is eligible for specific benefits, including tax credits, technical assistance with the applications necessary for marketing approval, and marketing exclusivity for seven years after receiving marketing approval.
Fibrocell has already completed a proof-of-concept trial on FCX-013. It evaluated the compound’s ability to decrease fibrotic tissue skin thickness. Researchers tested FCX-013 in mice with scleroderma. The drug reduced fibrotic tissue dermal thickness and thickness of muscle below the skin.
Based on the successful completion of the proof-of-concept study, Fibrocell started a pre-clinical-trial dose-ranging study in November 2016, aimed at determining the optimum dose for treatment. Studies of the drug’s ability to move through the body and its toxicity were the next steps in the development agenda.
Fibrocell expects to submit an Investigational New Drug (IND) application for FCX-003 to the FDA in 2017. An IND application is a request that the FDA allow doctors to administer an investigational drug to humans before the agency gives its full approval to the therapy. After pre-clinical development, a drug must undergo clinical trials in humans before the FDA considers it for approval.
Note: Scleroderma News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.