Houston Center To Offer Cell, Gene Therapy

http://ipn.intelihealth.com/ipn/ihtIPNNeuro?c=234652&t=11141

HOUSTON, Jul 27, 1999 (NYT Syndicate) — Three Houston medical
institutions have created the world's first center for cell and gene
therapy, a treatment option that is expected to become the future of
medicine.

The center — a partnership teaming Baylor College of Medicine, Texas
Children's Hospital and Methodist Hospital — will use new knowledge
about the molecular basis of disease to revolutionize therapy for
illnesses from cancer and AIDS to cardiovascular disease and diabetes.
The start-up cost is estimated at about $30 million.

"Centers like this will be at the forefront of a coming shift in the way
disease is treated," said Dr. Malcolm Brenner, director of the center.
"Not tomorrow, not 5 years from now, but 20 years from now, medicine
won't be something you take in a bottle. It will be like surgery was
going to be 50 years ago."

The center was established in January 1998, but formally launched
Monday, a couple weeks before the opening of a state-of-the-art,
25,000-square-foot, 15-bed inpatient adult stem cell transplant unit at
Methodist. Texas Children's Hospital is currently constructing a similar
unit that will be another part of the center.

Cell and gene therapy, simply defined, is the placement of beneficial
genes or cells into patient cells. Much heralded in the early 1990s,
then stalled by scientific setbacks, it is finally starting to live up
to its promise, thanks mostly to the Human Genome Project, the mammoth
effort to locate and sequence all 100,000 genes on the 46 human
chromosomes. Baylor is currently sharing an $80 million grant to
complete the genome project.

Gene therapy is ultimately expected to be used on embryos, first to
prevent disease, later to enhance physical or mental characteristics.
But Brenner said although such controversial work with embryos will
happen, "at this stage" he doesn't plan to pioneer such treatment.

The center will combine basic science and clinical research with
comprehensive pediatric and adult cell-and-gene therapy transplant
units. During the first five years, said Brenner, the focus will be on
cancer therapy — bone marrow transplants — and AIDS protocols. In the
next 10 years, added Brenner, treatment will move on to central nervous
system disorders like Parkinson's and multiple sclerosis, where progress
will likely come more slowly.

Brenner said he envisions cell and gene therapy as treatment options to
complement, but not replace, surgery, pharmaceuticals and radiation. He
said these therapies might initially draw patients who have exhausted
other forms of treatment, but ultimately will be best used as preventive
medicine.

Brenner currently supervises a staff of 20 clinical and research faculty
members. Over the next five years, plans call for adding 30 more faculty
and 300 support staff.

Facilities housed at the International Center for Cell and Gene Therapy
are the Pediatric Bone Marrow and Stem Cell Transplant Unit, the Gene
Vector Laboratory, the Transitional Research Laboratory, the Cell and
Molecular Therapy Laboratories, the Flow Cytometry Core Laboratories and
the Shell Center for Gene Therapy.

Examples of possible cell and gene therapy include research on the
ability of stem cells to regenerate and change function, said Dr. Helen
Heslop, director of the adult stem cell program at Methodist. Given the
right circumstances, she said they may be used for brain or muscle
cells, thus holding promise in the treatment of muscular dystrophy or
degenerative multiple sclerosis.

Heslop said another promising treatment is the development of a gene
that would block HIV at an early stage. Aimed at stopping HIV patients
from getting AIDS disease symptoms, it will likely be brought into
clinical practice in the next couple years, she said.

But Heslop, Brenner and other leaders stressed that the center, like all
of medicine, will advance by tiny steps, not giant breakthroughs. They
also said the flow of steady progress will justify the early 1990s hype.

"I've been thinking of something like this for a long time," said Dr.
Ralph Feigin, president and chief executive officer of Baylor. "I hope
in 20 years it's known not only for treating patients in new ways, but
also for training people who export this knowledge so patients around
the world can benefit."

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