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The health of every living organism is dependent on metabolism, a basic process of life that captures and releases the energy contained in foods we eat to help fuel the body. Within nearly every cell type in the body are tiny, pill-shaped structures called mitochondria. These are the powerhouses for the cells, allowing proper growth, enabling the organs and muscles of the body to function effectively, and providing us with the energy needed for good health. Defects in mitochondrial function can result in serious, often fatal, diseases.

The Center for BioEnergetics co-directed by Sidney Hecht, PhD and Guy Miller, MD, PhD, will focus on improved diagnoses and treatments for diseases caused by impaired energy metabolism. The majority of these diseases is degenerative and affects children and young adults. They include heart, liver or kidney disease; diabetes; poor growth; loss of muscle function; vision and hearing problems; developmental delays or mental retardation; respiratory and gastrointestinal disorders; and dementia. So in addition to impacting children, impairment to the mitochondria also is implicated as a factor in aging. It is associated with Parkinson's disease, atherosclerotic heart disease, stroke, Alzheimer's disease and cancer. On the other end of the spectrum, optimal mitochondrial function has been linked with peak physical performance, such as that exhibited by top athletes.

Miller brings firsthand clinical expertise and knowledge as a chemist and as an attending physician in medical-surgical critical care at Stanford University School of Medicine's Palo Alto VA Medical Center (PAVAMC). Hecht has more than three decades of experience in academic and industrial research positions as a biological chemist and drug designer, and also serves on the board of several biotech companies. In 2005, they co-founded Edison Pharmaceuticals, which focuses on inherited mitochondrial diseases. Together, they will probe into solving the "energy code" of these inherited mitochondrial deficiencies to help improve the lives of individuals suffering from these conditions.

More about mitochondria:

Mitochondria contain their own unique genetic code made up of 16,000 base pairs encoding 23 proteins. Mitochondrial information contained in this “energy code” is passed on directly from mother to child in a way that is completely separate from the human genome. So, mitochondrial diseases are inherited, but vary greatly in their effect on individuals.

The number of Mitochondria in each cell type varies. Cells that require a lot of energy to perform their function will have particularly high numbers of mitochondria.

Mitochondrial Diseases

Below are some of the approximately 40 types of mitochondrial diseases. Independently, these diseases are considered “rare” (or orphan) diseases, because they affect fewer than 200,000 people in the United States. The Center for BioEnergetics hopes that looking at these disorders as related diseases will lead to improved research funding and progress.

• Kearns-Sayre syndrome (KSS)
  
• Leigh's syndrome
  
• mitochondrial DNA depletion syndrome (MDS)
  
• mitochondrial encephalomyopathy, lactic acidosis and strokelike episodes (MELAS)
  
• myoclonus epilepsy with ragged red fibers (MERRF)
  
• mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)
  
• neuropathy, ataxia and retinitis pigmentosa (NARP)
  
• Pearson syndrome
  
• progressive external ophthalmoplegia (PEO)

    Our research can be broadly
     divided into three primary goals:
• Understanding nature’s biochemical energy lexicon -- the energy code
• Identification of chemical scaffolds to enable therapeutic design
• Design of systems biology models predictive of human function


    Translation aspects of our
     research program include:
• Development of biomarkers to enable optimization and therapeutic evaluation
• Chemical optimization using systems biology screens
• Advance small molecules into therapeutic evaluation