ORGANISMS FROM SINGLE-CELLED PROTOZOA to mammoth whales all depend on tiny cellular components called mitochondria for their energy supply. But just as a motor cannot operate if its fuel line is blocked, disorders that disrupt mitochondrial function can be life threatening. Investigator Vamsi Mootha, MD, of the MGH Department of Molecular Biology, focuses on understanding mitochondrial disease and has just received one of 17 Transformative Research Project Awards from the National Institutes of Health (NIH). These five-year grants, which do not have traditional budget caps, are designed to support unconventional projects that have the potential to revolutionize their fields. Three additional MGH investigators – Harald Ott, MD, of the Department of Surgery; Timothy Padera, PhD, of the Department of Radiation Oncology; and Joy Wu, MD, PhD, of the Endocrine Unit – are among 49 new recipients of five-year, $1.5 million New Innovator Awards.
“We're absolutely thrilled to receive this enabling grant,” says Mootha. “We will focus on a group of devastating disorders for which there is no proven therapy. Most involve a blockade in the pipeline of reactions required to produce ATP, the cell’s primary energy source. We’re proposing a totally novel approach and drawing inspiration from nature, specifically from microbes that regularly cope with similar blockages. Our work could lay the foundation for a brand new class of therapies for these neglected disorders.” Collaborating with Mootha will be Stanley Shaw, MD, PhD, of the MGH Division of Cardiology, and Jon Clardy, PhD, of Harvard Medical School.
Ott has developed a technique for assembling transplantable organ grafts by seeding the protein structures of donor organs with living cells. His team will work to expand fundamental animal studies to generate organs suitable for transplantation into human patients. Padera will seek to better understand the process of tumor metastasis, focusing on how the environment inside lymph nodes containing cancer cells contributes to tumor progression and possibly leading to new treatment targets. Wu’s project is directed toward increasing the availability of blood system stem cells, which are responsible for the success of bone marrow transplantation. By using bone-forming cells called osteoblasts to grow greater quantities of those essential cells in culture, her team’s work could expand the number of patients who can benefit from stem cell transplantation.
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