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Cyclosporine and Traumatic Brain Injury


Cyclosporine was first discovered in 1969 and was isolated from a fungus. The drug was immediately recognized to have tremendous immuno-suppressive properties and was put to work treating organ transplant patients facing tissue rejection. Since that time it has had numerous other clinical applications.

There has been continued interest in Cyclosporine as a potential treatment in traumatic brain injury (TBI). The second stage of brain trauma after the initial injury has been shown to be inflammatory and has been the subject of previous blogs. After an initial event, millions of brain cells continue to be lost because of the release of numerous chemicals in the brain from damaged cells. Thus, research has been directed at stopping these secondary effects.

Unfortunately, numerous TBI pharmaceuticals have been introduced into advanced clinical trials and all have failed to meet FDA (Federal Drug Agency) requirements for efficacy. These drugs don’t work.

Mitochondria are small organelles within each cell important to cellular function. Traumatic brain injury causes loss of mitochondria, and is probably an important path of secondary cell damage. Cyclosporine protects mitochondria from injury in many tissues (including the heart) and thus has shown to have promise in protecting the brain.

A unique formulation of Cyclosporine called NeuroSTAT has shown favorable results in TBI patients. In a collaboration, CHIC (Copenhagen Head Injury Cyclosporine) from Denmark and the University of Pennsylvania, observed a decreased volume of brain damage (approx. 35%) after brain MRI scans in experimental TBI studies with NeuroSTAT. In addition, the drug appears to have a beneficial impact on mitochondrial respiratory function.

So what lies ahead for such promising interventions? Larger scale studies involving hundreds of patients must confirm protective findings before a drug gains formal approval. As stated, many molecules look promising in the beginning but fall short in larger trials.

A Brief and Probably Unrelated Look at Cyclosporine and the Eye

Cyclosporine eye drops (brand name: Restasis) has been approved for treatment of dry eyes for many years. It works by reducing inflammation on the surface of the eye. The brain and the eye are quite similar. In any case, since TBI is here to stay, so effective drug therapies would be an important discovery.

Steven H. Rauchman, M.D. is an eye physician and surgeon who has been in private practice for 30 years. He has served as a Traumatic Brain Injury (TBI) medical/legal expert for the last 6 years specializing in the area of personal injury and related traumatic brain injuries.

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