ENZO SUCCESSFULLY ENGRAFTS CELLs IN BONE MARROW for HIV
patients
Monday October 2, 7:52 am Eastern Time
Press Release
Enzo Biochem Reports New Data On Phase I HIV Test Shows Engraftment of Engineered Cells in Bone Marrow
Company Says Development is Major Biotech Milestone
FARMINGDALE, N.Y.--(BUSINESS WIRE)--Oct. 2, 2000-- Treatment Producing New Enzo Engineered
Differentiated CD4+ Cells to Fight Virus
Enzo Biochem, Inc. (NYSE:ENZ - news) reported today that new data on the first individual treated in the Phase 1 clinical trial of HGTV-43, the Company's HIV-1 gene medicine product, show that after nine-and-one-half-months Enzo engineered cells have successfully engrafted in the patient's bone marrow and were spawning new differentiated CD4+ cells designed to fight the virus.
``This is a dramatic result, one that underscores, in the light of the other achievements we have recorded in this trial, that our gene medicine and technology designed to counter the HIV virus is on the right track,'' said Dean L. Engelhardt, Executive Vice President of Enzo. ``It is also, we believe, a scientific milestone since to our knowledge no other technology has been successful in engrafting cells expressing a cloned gene in the bone marrow of adult subjects without first ablating the patient, a process in which blood cells are destroyed.''
Enzo is currently compiling and analyzing data from the remaining five patients in the trial. The Company said that while the data is limited and must await further study, it is highly encouraged. ``We believe that Enzo has taken an important step to reconstituting an individual's immune system with resistant cells, an exceptional achievement,'' said Dr. Engelhardt.
``These findings are very exciting. This novel approach may be one of the new treatment platforms we desperately need to confront the problem of resistance to the anti-viral cocktails,'' said Marcus A. Conant, MD, a leading physician in the management of HIV-1 infected patients and one of the principal investigators of the trial.
The Company said that it is focusing on preparations for a Phase II trial, and that pending submissions to the FDA, it had already begun negotiations with several hospitals at which the broader study would be conducted.
Enzo said that all the patients tolerated the procedure well. The new data that show that the progeny CD4+ cells are now in circulation indicate that it is possible to effectively reintroduce engineered cells into a patient, have them reestablish themselves and, importantly, reproduce progeny cells having the same engineered characteristics.
HGTV-43, the Company's proprietary gene medicine product, was developed to protect human immune cells against infection by a broad spectrum of strains of HIV-1 including its mutational variants. This medicine incorporates two proprietary technologies, plus additional techniques, all of which were successfully implemented during the trial. One involves insertion of a new type of genetic material into blood cells that has been shown to inhibit the growth of HIV-1. This technology, known as genetic antisense, utilizes a ``mirror image'' of a gene, called an antisense molecule, to block the virus.
The second technology is based on a novel vector designed to deliver the antisense molecule to the cell. This vector is employed to insert new genes into target cells with the objective of integrating the gene into the chromosome of the cell, in a process known as transduction. Enzo stated previously that it has achieved high levels of transduction within 18 hours, thus limiting inconvenience to the patient, and in achieving levels of stable gene transfer to target cells greater than 30%. The cells containing these new genes, when transferred to the patient, replicate and provide the patient with the newly inserted biological function. The Company believes that its proprietary vector system constitutes a technological breakthrough in the development of commercially viable gene medicine.
Effectively, Enzo has succeeded in developing and utilizing its proprietary vector for delivery of its HIV gene medicine into the patient's blood cells. With this vector, Enzo succeeded in achieving high levels of stable gene transfer within one day, as compared to previous procedures that required two or more weeks. These cells have been shown to persist in patients and to produce differentiated CD4+ immune cells. The engraftment of these blood cells expressing Enzo's anti-HIV-1 transgene brings the goal of reconstituting the immune system with virus resistant cells considerably closer to completion.
Enzo Biochem is engaged in the research, development and manufacture of innovative health care products based on molecular biology and genetic engineering techniques, and in providing diagnostic services to the medical community. |
