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Research Overview The Consortium began operations in July 2000 and receives $3 - $5 million annually from the state to support the recruitment of leading researchers in the field of gene therapy, to establish core technology labs at all three institutions, and to develop gene therapy technologies for clinical applications. To date, more than 33 researchers have been assembled who are being supported by more than $60 million in research grants. Current gene therapy research includes both cell-based and vector-based
technologies focusing on interventions for cancer, cystic fibrosis,
asthma, spinal cord repair, heart disease, alcoholism, and other
diseases or genetic conditions. The Tulane Center for Gene Therapy is the first
research facility in the country wholly dedicated to using adult
(non-fetal) stem cells. Researchers have developed techniques that
allow them to grow large numbers of stem cells from a small sample
extracted from a patient’s
bone marrow. In some of the most groundbreaking research, Tulane’s
Gene Therapy Center is using adult stem cells to treat genetic abnormalities.
Based on Tulane center’s research, five patients at St. Jude
Children’s Research Hospital in Memphis were successfully treated
with stem cells from their healthy siblings. The new stem cell treatment
is now ready for large-scale clinical trials, and the researchers
are seeking corporate partners and venture capital to continue the
development. At LSUHSC-New Orleans, the scope of research in the Gene Therapy
Program ranges from the development of gene-based strategies for
prevention or therapy of intractable infectious diseases and different
cancers, to the restoration of missing function in genetic disease.
These translational programs are underpinned by strong fundamental
research in vector development, gene discovery, and clarification
of underlying disease mechanisms. Program scientists, led by Dr Alistair
Ramsay, aim to develop novel preventives and therapies for several
common diseases and conditions, including HIV/AIDS, tuberculosis,
cystic fibrosis, neurological disease, and asthma, as well as cancers
of the breast, lung and prostate. At LSUHSC-Shreveport, gene therapy researchers are also working
with a variety of vectors. Dr. Mike Mathis has assembled a team of
seven researchers across multiple departments all focused on treatments
of different types of cancer. One novel approach utilizes a targeted
delivery system to stop the growth of certain types of cancer tumors.
Shreveport has also built out a clinical research facility to facilitate
implementation of clinical trials. As research progresses and gene
therapy techniques are perfected, these treatments must be tested
in clinical trials. The FDA closely monitors clinical trials in humans,
including the manufacture of both stem cells and gene vector systems. |
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