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Biopharm, Hospital to Partner on Cell Energy Diseases

Children's Hospital of Philadelphia

Children’s Hospital of Philadelphia, main campus (

23 January 2017. A biopharmaceutical company in Sweden and children’s hospital in the U.S. are collaborating on research into deficiencies in cells’ energy components responsible for a number of genetic disorders. Financial details of the project between NeuroVive Pharmaceuticals AB  in Lund, Sweden and Children’s Hospital of Philadelphia were not disclosed.

The agreement calls for a team at Children’s Hospital to evaluate compounds in an experimental drug by NeuroVive to treat mitochondrial disorders, inherited diseases affecting the mitochondria, components in cells that provide energy for the cells at large. The team is led by Marni Falk, director of the hospital’s Mitochondrial Disease Clinical Center.

Mitochondria provide 90 percent of the energy for sustaining human organs and functions, thus disorders in mitochondria result in cell energy and death, particularly in organs requiring high energy, such as the heart, brain, muscles, and lungs. Symptoms can include seizures, strokes, as well as inability to walk, see, talk, or digest food. United Mitochondrial Disease Foundation says mitochondrial disease affects mainly children, but adult onset is occurring more frequently.

Falk and colleagues will review NeuroVive’s experimental drug code-named NVP015 designed to penetrate outer cell membranes and activate inside the cell. The Children’s Hospital team will evaluate NVP015’s ability to affect energy metabolism and disease development in preclinical models of disease resulting from deficiencies in a part of the mitochondria known as Complex I (roman numeral I), the source of most mitochondrial disorders.

Complex I is one of four protein chains in the mitochondria that produce energy, with complex I deficiency resulting in progressive neurological degeneration, and affecting high-energy organs including brain, heart, liver, and skeletal muscles. A number of rare inherited disorders are associated with complex I deficiency, such as Leigh Syndrome and Leber’s hereditary optic neuropathy.

The hospital’s Mitochondrial Disease Clinical Center is both a treatment and research facility, where Falk serves as an attending physician as well as its director. The center conducts studies with simple animal models examining signaling pathways affecting mitochondrial disease, as well as possible treatments, including a form of niacin, or vitamin B3. Its studies also cover protein generation from genes and autophagy, the process of cell maintenance, both of which are disrupted in mitochondrial disorders.

This collaboration is not the first for NeuroVive with an institution in Philadelphia. As reported in Science & Enterprise in April 2016, the company is partnering with University of Pennsylvania’s medical school in preclinical studies of its drug candidate NeuroStat that protects against long term damage caused by traumatic brain injury. NeuroVive develops drugs based on cyclosporine, a common compound used largely to suppress the immune system for preventing rejection in organ transplants. The company’s work extends to another property of cyclosporine, to protect mitochondria in nerve cells following traumatic injuries.

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