Regenerative medicine researchers explore innovative ways to treat childhood conditions

About 3 percent of the babies born in the United States have a birth defect. Children without a birth defect are also susceptible to injury or disease. At The University of Texas Health Science Center at Houston (UTHealth), regenerative medicine researchers are exploring innovative ways to treat these conditions.

The Department of Pediatric Surgery at the UTHealth Medical School operates a research program devoted to childhood conditions that is seeking to harness the body's regenerative powers to repair malformed organs and to mitigate injury from illness or trauma. It is called the Children's Regenerative Medicine Program.

Kevin Lally, M.D., chairman of the Department of Pediatric Surgery at the UTHealth Medical School and surgeon-in-chief at Children's Memorial Hermann Hospital, announced the recruitment of four stem cell scientists to the program. The researchers are associate professor Yong Li, M.D., Ph.D., and assistant professors Scott Olson, Ph.D., Fabio Triolo, M.Phil., D.d.R., Ph.D., and Pamela Wenzel, Ph.D.

"We were able to recruit outstanding investigators thanks in part to the generous support of Mrs. Clare Glassell and Mrs. Evelyn Griffin," said Lally, who is the A.G. McNeese Chair in Pediatric Surgery and the Richard Andrassy, M.D., Endowed Distinguished Professor at UTHealth. "Our program in regenerative medicine is committed to a collaborative environment in which clinicians work with basic researchers."

Lally has a special interest in the treatment of a potentially life-threatening condition known as congenital diaphragmatic hernia that occurs in as many as one in every 2,500 live births and is often treated surgically. The diaphragm is a muscle separating the chest cavity and belly and is important for breathing.

"The core fundamental problem is infants born with structural problems that need to be repaired," said Lally, who believes doctors may be able to use stem cells to replace the diaphragm or repair defects in the abdominal wall.

Stem cells may also help children with traumatic brain injuries. Charles Cox Jr., M.D., professor of pediatric surgery, director of the Pediatric Trauma Program at the UTHealth Medical School/Children's Memorial Hermann Hospital and The Children's Fund Inc. Distinguished Professor in Pediatric Surgery, is leading clinical research on the use of these versatile cells to address this leading cause of death and lifelong disability among children.

Yong Li, M.D., Ph.D.

Li previously served as a tenure track assistant professor in the Stem Cell Research Center at Children's Hospital of Pittsburgh, where his laboratory studied the regenerative power of newts and salamanders in the hope of applying this information to the care of people.

Newts are able to regenerate missing body parts including limbs, spine and heart. Li has studied growth factors that facilitate this regeneration process in some amphibians and how they could be used for stem cell population and tissue engineering.

At UTHealth, Li's laboratory is centered on engineering tissue that could potentially be used to repair defects in the diaphragm, heart and abdominal wall. He hopes to do this by turning back the clock on various stem cells so that they can be used to create specialized cells such as those in the diaphragm, heart and musculoskeletal tissues.

He said, "Regenerative medicine has developed dramatically in the last decade. The advantage in pediatric regenerative medicine is that the young patients heal robustly and their cells are more amenable to tissue engineering. There will also certainly be challenges with children, since you have to design the engineered tissue application to allow for growth."

Scott Olson, Ph.D.

Olson came to UTHealth from the University of California at Davis Institute of Regenerative Cures, where he worked in a laboratory that used stem cells to deliver a therapy to target a neurodegenerative disorder known as Huntington's disease.

He said the technology could be used to address other genetic disorders. "We are trying to make new and more effective cellular therapies," said Olson, whose research is focused on an adaptable adult cell known as a mesenchymal stem cell.

Olson's research interests also include traumatic brain injury.

Stem cells have been called "paramedic cells" and "reparative cells," according to Olson, and could speed the healing process.

At UTHealth, Olson's lab is focused on better understanding the role of mesenchymal stem cells in traumatic brain injury.

Fabio Triolo, M.Phil., D.d.R., Ph.D.

Triolo, an expert in clinical cell therapy manufacturing, previously served in Italy at the Mediterranean Institute for Transplantation and Advanced Specialized Therapies of the University of Pittsburgh Medical Center, where he designed and directed a state-of-the-art facility in which clinical-grade cells were produced for clinical applications of regenerative medicine.

There is a chronic shortage of organ donors worldwide. Regenerative medicine could help reduce the gap between need and availability of organs for transplantation, he said. "Instead of substituting failing organs, regenerative medicine promises to 'fix' them through regeneration provided by transplanted cells, stimulation of endogenous repair mechanisms or implantation of bioengineered tissue," he said.

Triolo added, "Tissue-engineered urethras and bladders have already been successfully used to treat children with complex anatomical defects or damage. Mesenchymal stem cells used in clinical trials were recently proven to play various therapeutic roles in diseases where conventional therapy is still ineffective in pediatric patients."

At UTHealth, Triolo runs the Judith R. Hoffberger translational laboratory and the Evelyn H. Griffin Stem Cell Therapeutics Research Laboratory and his role is to "translate, scale-up and validate promising new therapeutic technologies developed by our scientists at a preclinical level into clinical-grade processes that can be used to manufacture cell-based and/or tissue engineered products for clinical applications."

Pamela Wenzel, Ph.D.

Wenzel, a stem cell biologist, joins UTHealth from Children's Hospital Boston and Harvard Medical School, where she worked to develop ways to improve cellular therapies for genetic blood disorders and cancer.

She has conducted research on the relationship between hemodynamic forces produced by blood flow in arteries such as friction, pressure, and stretching, and the 'potency' of stem cells.

"Frictional force produced by blood flow in the circulatory system of the embryo or fetus produces a cascade and tells the body to produce more high-quality blood-forming stem cells," said Wenzel.

"We want to use this knowledge to generate and expand stem cell sources in the lab that can be used clinically for patients in need of hematopoietic cell transplantation. We are also now refining some of these same principles to evaluate a role for mechanical force in treatment of traumatic brain and spinal cord injuries," she said.

Li and Wenzel are on the faculty of the Center for Stem Cell and Regenerative Medicine at The Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases at UTHealth.

"The successful recruitment of these four faculty members to the Department of Pediatric Surgery significantly increases the strength of the regenerative medicine program here at UTHealth. We are particularly delighted to have Drs. Li and Wenzel as active faculty members in our Center for Stem Cell and Regenerative Medicine -- with their laboratories on the sixth floor of the Sarofim Research Building," said Brian R. Davis, Ph.D., interim director of the Center for Stem Cell and Regenerative Medicine.