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Simmons Family Foundation awards collaborative research grants

Teams from Rice University, Texas Children's Hospital and The Methodist Hospital Research Institute study leukemia, pediatric hearts, lysosomal disorders, harmful bacteria

By Mike Williams
Rice News staff 

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Leukemia, pediatric heart valves, lysosomal storage disorders and the bacteria that causes strep throat – and worse – are the targets of this year's annual grants supported by the Virginia and L.E. Simmons Family Foundation Collaborative Research Fund.

The fund is a five-year, $3 million initiative to discover new ways to diagnose and treat diseases. Grants go to teams of collaborators from Rice University, Texas Children's Hospital and The Methodist Hospital Research Institute (TMHRI).

Four projects were funded in the program's fourth year. Successful initial findings will ideally lead the researchers to pursue further funding from the National Science Foundation, the National Institutes of Health and other organizations.

The fund was formed to promote novel solutions to difficult medical problems through the combined expertise of Texas Medical Center scientists, engineers and physicians who might not otherwise collaborate.

"The quality of the proposals received this year maintains the high standards we've seen since we began the fund," said L.E. Simmons, a trustee at Rice and Texas Children's Hospital and a board member of TMHRI. "The choices are always difficult, but no task we take on is as satisfying as supporting the groundbreaking work being done by these researchers."

Previous grants have funded research into breast and childhood cancers, hearing loss, flu, tuberculosis, repair of newborns' hearts and elimination of brain tumors.

This year's winners:

Stopping strep and more 

Paul Sumby and Edward Nikonowicz will study the expression of genes that code for a subset of virulance-enhancing proteins that support group A Streptococcus (GAS), which causes strep throat and necrotizing fasciitis, aka the "flesh-eating syndrome," among other diseases.

The researchers hope to lay the foundation for future collaborations on GAS therapeutics.

Sumby is an assistant member of TMHRI, a research scientist at the Center for Molecular and Translational Human Infectious Diseases Research and a member of the Department of Pathology and Genomic Medicine at The Methodist Hospital. Nikonowicz is an associate professor of biochemistry and cell biology at Rice.

Treatment for leukemia 

Zachary Ball, Michele Redell and David Tweardy will work to develop new drugs to treat acute myeloid leukemia (AML), which afflicts thousands of new patients every year, many of them children. Current treatment options, including chemotherapy and bone marrow transplants, are difficult and painful; relapses are common. The researchers indicated that patients in whom AML recurs are often resistant to further treatment.

The team will take a new approach to create drugs that target the STAT3 protein, which has been linked to aggressive growth of AML cells.

Ball is an assistant professor of chemistry at Rice. Redell is a physician and researcher in the Texas Children's Cancer Center. Tweardy is a professor of medicine and chief of the Section of Infectious Diseases at Baylor College of Medicine (BCM) and is affiliated with Texas Children's Hospital.

Fixing children's hearts 

Henri Justino, Jane Grande-Allen and Daniel Harrington are developing an artificial valve to repair infant and juvenile hearts that could be inserted within a stent to avoid painful, complicated open-heart surgeries. The researchers claim that pulmonary valve replacement surgery now accounts for 75 percent of all valve replacement surgeries in children. Because of the poor durability of current replacement valves, repeated surgeries are often necessary.

A stent-based replacement valve could be inserted through a minimally invasive procedure in which a thin catheter would be threaded through a tiny incision in the groin. The team intends to study the chemical and physical properties of the artificial valve to improve its durability.

Justino is a pediatric interventional cardiologist at Texas Children's Hospital and associate professor of pediatrics at BCM. Grande-Allen is an associate professor of bioengineering at Rice. Harrington is a faculty fellow in biochemistry and cell biology at Rice.

Lysosomal disorder-targeted therapy 

Marco Sardiello, Laura Segatori and Karl-Dimiter Bissig are collaborating to find an effective treatment for a range of currently incurable lysosomal storage disorders that include Gaucher's disease, neuronal ceroid lipofuscinoses and Sanfilippo syndrome. A successful strategy could also impact the treatment of Alzheimer's, Parkinson's and Huntington's diseases.

The lysosome is a membrane-enclosed organelle that functions as a cell's waste disposal system. Impairment of the lysosome often leads to the accumulation of undegraded material and to the development of diseases, the most severe of which affect the brain. These are incurable because the blood-brain barrier, which separates brain tissues from the body's bloodstream, limits the ability to deliver therapeutics. The team plans to base a new type of therapy on a nontoxic, brain-accessible molecule that will boost lysosomal function.

Sardiello is an assistant professor in the Department of Molecular and Human Genetics at BCM and affiliated with Texas Children's Hospital. Segatori is the T.N. Law Assistant Professor of Chemical and Biomolecular Engineering at Rice. Bissig is an assistant professor in the Department of Molecular and Cellular Biology at BCM.

The Simmons family 

L.E. Simmons is president and founder of SCF Partners, an investment firm that provides management expertise to energy service companies. Virginia Simmons is vice president of the Simmons Family Foundation, which supports religious, art and cultural organizations, education, and youth and medical associations.

For information on the Virginia and L.E. Simmons Family Foundation Collaborative Research Fund, visit www.collaborativeresearchfund.org.