The Search for Answers

Malcolm Brenner, Ph.D., M.B.B.Ch., directs the Center for Cell and Gene Therapy, which tackles adult and childhood diseases in the lab and at the bedside.

Type the terms "Cell and Gene Therapy" into Google's search engine and Baylor College of Medicine's Center for Cell and Gene Therapy pops up at the top of the list in just .17 seconds.

That search is easy, but the continuing pursuit of how to save lives using cell and gene therapy at the bedside is a much more complicated quest.

Working together is the best way to try to answer the questions raised by the work, said Malcolm Brenner, Ph.D., M.B.B.Ch., the Center's Director.

"It's exciting when you find collaborative work is more than the sum of its parts," said Brenner, who has been treating patients with cell and gene therapy for 15 years. "That is how we make discoveries, taking things that colleagues have done, add it to another's work and then help them develop something that is more than the sum of the two."

Cell therapy involves the manipulation of cells that are then used to treat people with cancers and other diseases. Bone marrow or stem cell transplants are forms of cell therapy. Gene therapy involves the transfer or alteration of genes to encourage the body to make a protein it lacks or a healthy form of a protein in order to treat an inherited or acquired disease.

Brenner arrived at BCM 10 years ago to develop the Center for Cell and Gene Therapy among three institutions in the Texas Medical Center—Baylor College of Medicine, The Methodist Hospital and Texas Children's Hospital. He came from St. Jude's Children's Research Hospital in Memphis, Tennessee, because BCM offered him the opportunity to treat adults with genetic and autoimmune disorders as well as children with those diseases and cancers. The hope, he said, is to use gene therapy to treat a wide range of diseases such as cancer, cardiovascular disease, diabetes and HIV/AIDS.

The Center combines both basic and clinical research components into a translational base from which it is possible to move results quickly from the laboratory to patient treatment.

The hope, Brenner said, is to use gene therapy to treat a wide range of diseases such as cancer, cardiovascular disease, diabetes and HIV/AIDS.

A dozen basic science researchers in the Center look at the fundamental questions involved in cell and gene therapy. They take those ideas to the laboratory, where they fine-tune them into treatments for patients. Among the areas they study are stem cells and how they differentiate into different kinds of tissue, how to put genes into cells and how to regulate those genes to encourage them to repair a genetic defect, retarget an immune cell to enable it to recognize a cancer cell or to halt the damage caused by tumor cells.

"At Texas Children's Hospital, we see patients with leukemia and childhood malignancies such as neuroblastoma," said Brenner. "What we do is a series of small-scale clinical trials or studies in a few patients."

In these translational studies, the researchers, following strict guidelines for patient research, apply their treatments to patients and determine the outcome. They then take their treatments back to the laboratory for refinement and then try them again.

"We've been pretty successful with lymphomas and treating hematologic malignancies such a leukemias using genetically modified cells," said Brenner.

"And now that we have good results in lymphomas, we want to expand to lung cancer, brain tumors, breast cancer and patients with immune deficiencies and genetic disorders."

Both basic and clinical scientists work with the Center's manufacturing facility to manufacture vectors (the biological tools that take modified genes into cells). In fact, said Brenner, they produce vectors for institutions and laboratories studying gene therapy around the world.

Making novel therapies using cells and modified genes available to physicians and their patients is the goal of the Center for Cell and Gene Therapy, said Brenner. In the next decades, he anticipates that the work will prove even more successful, taking treatments to patients who need
it most.

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Volume 4, Issue 3, Winter 2008


	The Search for Answers by Kimberlee K. Norton Malcolm Brenner, Ph.D., M.B.B.Ch., directs the Center for Cell and Gene Therapy, which tackles adult and childhood diseases in the lab and at the bedside. Type the terms "Cell and Gene Therapy" into Google's search engine and Baylor College of Medicine's Center for Cell and Gene Therapy pops up at the top of the list in just .17 seconds. That search is easy, but the continuing pursuit of how to save lives using cell and gene therapy at the bedside is a much more complicated quest. Working together is the best way to try to answer the questions raised by the work, said Malcolm Brenner, Ph.D., M.B.B.Ch., the Center's Director. "It's exciting when you find collaborative work is more than the sum of its parts," said Brenner, who has been treating patients with cell and gene therapy for 15 years. "That is how we make discoveries, taking things that colleagues have done, add it to another's work and then help them develop something that is more than the sum of the two." Cell therapy involves the manipulation of cells that are then used to treat people with cancers and other diseases. Bone marrow or stem cell transplants are forms of cell therapy. Gene therapy involves the transfer or alteration of genes to encourage the body to make a protein it lacks or a healthy form of a protein in order to treat an inherited or acquired disease. Brenner arrived at BCM 10 years ago to develop the Center for Cell and Gene Therapy among three institutions in the Texas Medical Center—Baylor College of Medicine, The Methodist Hospital and Texas Children's Hospital. He came from St. Jude's Children's Research Hospital in Memphis, Tennessee, because BCM offered him the opportunity to treat adults with genetic and autoimmune disorders as well as children with those diseases and cancers. The hope, he said, is to use gene therapy to treat a wide range of diseases such as cancer, cardiovascular disease, diabetes and HIV/AIDS. The Center combines both basic and clinical research components into a translational base from which it is possible to move results quickly from the laboratory to patient treatment. The hope, Brenner said, is to use gene therapy to treat a wide range of diseases such as cancer, cardiovascular disease, diabetes and HIV/AIDS. A dozen basic science researchers in the Center look at the fundamental questions involved in cell and gene therapy. They take those ideas to the laboratory, where they fine-tune them into treatments for patients. Among the areas they study are stem cells and how they differentiate into different kinds of tissue, how to put genes into cells and how to regulate those genes to encourage them to repair a genetic defect, retarget an immune cell to enable it to recognize a cancer cell or to halt the damage caused by tumor cells. "At Texas Children's Hospital, we see patients with leukemia and childhood malignancies such as neuroblastoma," said Brenner. "What we do is a series of small-scale clinical trials or studies in a few patients." In these translational studies, the researchers, following strict guidelines for patient research, apply their treatments to patients and determine the outcome. They then take their treatments back to the laboratory for refinement and then try them again. "We've been pretty successful with lymphomas and treating hematologic malignancies such a leukemias using genetically modified cells," said Brenner. "And now that we have good results in lymphomas, we want to expand to lung cancer, brain tumors, breast cancer and patients with immune deficiencies and genetic disorders." Both basic and clinical scientists work with the Center's manufacturing facility to manufacture vectors (the biological tools that take modified genes into cells). In fact, said Brenner, they produce vectors for institutions and laboratories studying gene therapy around the world. Making novel therapies using cells and modified genes available to physicians and their patients is the goal of the Center for Cell and Gene Therapy, said Brenner. In the next decades, he anticipates that the work will prove even more successful, taking treatments to patients who need it most.		Search all issues: Features Baylor College of Medicine in Houston: 65 years of Excellence BCM Looks to the Future on the McNair Campus Legacy of Leadership: BCM Icons Set Foundation for the Future 1,000 Genomes Project: Looking for the Differences News O'Malley Receives National Medal of Science Robert Todd Named to Lead BCM's Department of Medicine Roy Huffington Remembered as Bold and Generous Spotlight Following his Passion—One Physician's Journey The Search for Answers When Two Degrees are Better Than One: M.D. - M.P.H. Thomas Street—The Road to Health Perceiving—A new Look at Brain and Behavior Briefs Mind Games Wang Wins NIH Award Genetics Used to Personalize Heart Disease Treatment First Drug for Huntington's Disease Eye Problems from Pain Free Migraines Obesity also Causes Cirrhosis Genetic Insights into Deadly Brain Tumor Made-to-Order Weapon in the Fight Against Childhood Cancer BCM Joins Largest Children's Study Development/Alumni Minds & Medicine Campaign Update: McNair Campus gets First Tenants -- Ophthalmology Philanthropy Provided Key Assistance Gifts Help Restore Sight to Patients with Corneal Damage Alums Start Careers, Life Together at BCM Development Briefs Campaign Reaches Milestone Charitable Gifts Lead to National Recognition Trustee Chuck Watson Makes Unique Gift The Legacy and the Future

	Volume 4, Issue 3, Winter 2008



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