Program Overview

Development of the Program

The Need for the Program

The Immediate Goal of the Program

The Opportunity to help the Program

Program Overview

New York Medical College began the basic science component of a gene therapy program in early 1997, anticipating the potential of this new technique for treating disease into the 21st century. Gene therapies for chronic mylogenic leukemia (CML), for certain solid tumors and for Gaucher's disease will be available. Other genes available through the program are those that modulate the immune system. These are needed to prevent the rejection of organ transplants, such as kidney and liver. Retrovirus genes containing the thymidine kinase (HSTK) gene have been shown to be effective in such clinical situations. Adenoviral heme oxygenase is currently under preclinical study to evaluate its role as an important modulator in hypoxia-mediated cardiovascular diseases, such as hypertension and atherosclerosis.

The laboratory's centralized location in Westchester County affords convenient transport of gene therapy to clinical sites throughout the tri-state area. There are no gene therapy programs of comparable breadth and scope anywhere else in the region.

Trials for gene therapy against AIDS, prostate and breast cancer are in development. The backbone of the retrovirus for u.-interferon has been approved by the FDA and will form the basis for other studies that are expected to be available by the end of 1997. In addition, a retrovirus interferon gene construct is in Phase I clinical trial in France as antiviral therapy for AIDS. Similar trials for gene therapy utilizing interferon against AIDS, prostate and breast cancer are underway in our institution.

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Development of the Program 

New York Medical College's Department of Pharmacology, under the leadership of the distinguished scientist John C. McGiff, M.D., has been building toward the establishment of the Gene Therapy Program for the past several years. Dr. McGiff, an NIH MERIT award recipient, is internationally known for his work with certain hormones that contribute, to high blood pressure, congestive heart failure and kidney disease. On the strength of the ongoing research in the Department of Pharmacology, Dr. McGiff was able to attract Dr. Nader G. Abraham to head the Gene Therapy Program at the College.

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The Need

With the arrival of Dr. Nader Abraham, supported by a team of investigators who are expert in adenovirus and retrovirus gene transfer, the Gene Therapy Program at New York Medical College has taken a quantum leap forward. Expertise can now be brought to bear on a variety of basic science and clinical problems for the ultimate benefit of patients in the tri-state area.

In order for the Gene Therapy Program to move beyond basic science to the treatment of patients, start-up funds are critically needed. While some supporting equipment is available, including PCR, the university is actively seeking $2.0 million in philanthropic underwriting to make the program fully operational and prepare for clinical trials. These funds will be used to renovate existing laboratory space, to purchase equipment such as a flow cytometer and to support three research associates, an administrative office and other expenses. The program will function as the core of all university activities in gene therapy.

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Immediate Goal

The university's leadership is committed to ensuring the success of this program and will maximize every opportunity to advance its progress. There is an equally strong commitment to basic and clinical research within the College's Department of Pharmacology. Here basic scientists work hand in hand with the university's clinical departments. Faculty clinicians, in turn, are active throughout the College's extensive network of affiliated hospitals, which includes two academic medical centers, and in collaboration with hospitals throughout the region.

The immediate goal of the Gene Therapy Program is to transfect deficient cells with a new gene and then to transport these cells to participating clinical sites in the tri-state area. Ultimately, however, the program's gene therapy protocols will be available to clinical sites nationally and internationally Four international centers, in Berlin and Restock, Germany, in Rome, Italy, and in Tokyo, Japan, are currently collaborating with Dr. Abraham to provide such service.

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Opportunity

A significant investment is required for the Gene Therapy Program. The program provides a number of recognition and naming opportunities for interested benefactors. New York Medical College would be pleased to discuss how these opportunities can create broad awareness for this exciting program throughout the region and across the nation.

The College invites exploration of this opportunity with any interested party. Please call Dr. John McGiff or Dr. Nader Abraham at 914/594-4132 to discuss any of the issues central to gene therapy Dr. Abraham's assistant can also be reached via e-mail at: Jennifer Brown@NYMC.edu

Contact Rev. Msgr. Harry C. Barrett, D.Min., M.P.H., President and Chief Executive Officer of the university, at 914/594-4600 to obtain further information or to discuss how to support this endeavor.

We welcome your participation in this important gene therapy venture.

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