Development of new therapies for cancer
We treat large numbers of patients with multiple myeloma and triple negative breast cancer. Clinical trials examining the efficacy of genome directed therapy for patients with triple negative breast cancer are already underway. Some success has been reported in using cell-based immunotherapy to treat refractory myeloma. Establishment of the production facility for the manufacturing (cGMP) of cell-based medicines will permit a more wide spread application of CAR T cell therapy to eligible patients and allow other clinical trials of cell-based immunotherapies and cutting edge cell-based regenerative medicine protocols for patients.
Therapies for childhood genetic diseases
Development of early curative therapies for monogenetic disorders that manifest at birth or in childhood can give our patients a lifetime of health. Members of our pediatric faculty are already planning to lead a human clinical trial of gene therapy for Fanconi anemia (a congenital disorder that causes bone marrow failure with predisposition to cancer development). We aim to use the planned expansion of the cGMP production facilities to focus our expertise for gene therapy on addressing many more commonly found genetic illnesses.
Immunotherapies for Alzheimer’s and neurodegenerative diseases
Increasing prevalence of Alzheimer’s disease (AD) in the aging population makes it an important focus area for programmatic research. One of our commercial partners, Eli Lilly and Company, is interested in developing immunotherapies to AD. Several potential mechanisms of targeting AD, including immunotherapy via infusion of monoclonal antibodies, are currently under investigation. Another research direction is development of a preventive AD vaccine as a practical alternative to high-cost, limited-supply treatment.