Tetrahydrobiopterin for Treatment of Cardiac Failure and Remodeling

Tetrahyrdobiopterin (BH4) is an obligate cofactor for the aromatic amino acid hydroxylases (tyrosine, tryptophan, and phenylalanine), as well as for the nitric oxide synthases. It is currently used clinically in a subset of patients born with an inborn error of metabolism known as phenylketonuria (PKU). It has not been previously used as a clinical therapy to target nitric oxide synthase (NOS), but recent studies suggest this may be a very useful approach. When BH4 is oxidized and/or depleted, NOS become functionally ?uncoupled?. Rather than synthesizing nitric oxide, NOS generates radical oxygen species (ROS). Now, JHU scientists have discovered for the first time that NOS uncoupling through BH4 depletion is central to the function and structural integrity of the stressed heart. Using repletion therapy of BH4 in mouse models, scientists have demonstrated that cardiac dilation and the development of heart failure induced by exposure to chronic pressure-overload stress can be both prevented, and reversed by therapeutic administration of BH4. These data support a novel use for BH4 as a therapy for cardiac failure and chamber remodeling. Description (Set) Proposed Use (Set) Heart failure is a major public health problem in the industrialized world and a drain on medical resources. Its prevalence in the general population is estimated at 3-20 per 1000 and rises to 23-130 per 1000 in patient groups over the age of 60. The application of BH4 as a preventative for heart failure is a promising new treatment option.

Inventor(s): Kass, David

Type of Offer: Licensing

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