gene-therapy

Review
Excerpts from
Q: What is a human gene therapy product?
A: Human gene therapy is the use of genetic material to treat, cure, or prevent a disease or medical condition.

Gene therapy is highly innovative and poses some unique and potentially unknown risks. However, it also presents unique possibilities and the hope to cure and treat diseases in a manner differently, and in some cases potentially better or more safely, than some currently available treatments.

Q: How do these products work?
A: Genetic material can be used to replace defective genes in a person’s body that are responsible for a disease or medical problem. It can also be used to treat disease through the expression of a protein that the gene codes for. Currently, all gene therapy is investigational.

Q: What is the potential impact of these products for consumers?
A: Research in this area has the potential to revolutionize the treatment of diseases that currently are incurable or have inadequate treatments. Gene therapy is being studied to meet unmet medical needs in diverse areas such as genetic diseases, heart disease, cancer, diabetes and stopping the replication of HIV in AIDS patients.

Q: What is FDA’s role in regulating these products?
A: Since the first human gene transfer in the late 1980s, CBER has provided proactive scientific and regulatory guidance in this area of novel product development.

CBER works closely with product sponsors of potential investigational new drug (IND) applications, and encourages early and frequent dialogue to help define the best scientific approaches and clarify FDA regulatory requirements. These meetings between CBER review staff and product sponsors help reduce product development time and risk.

Q: Have any gene therapy products been approved yet?
A: No. FDA has not yet approved for sale any human gene therapy products.

Q: What are the challenges that scientists and government agencies face in making these products widely available?
A: The development of gene therapy products is a relatively new field compared to other biologics and to traditional drugs. It takes time and careful evaluation for medical interventions based on the administration of genetic material to modify or manipulate the expression of a gene or to alter the biological properties of living cells to develop and potentially result in licensed products.

We continue to see broad interest in the development of a number of different gene therapy vectors used to treat a wide variety of diseases and medical conditions.

Source
This article appears on FDA’s Consumer Update page, which features the latest on all FDA-regulated products.

Article Title
Human Gene Therapies: Novel Product Development
Q&A with
Celia M. Witten, Ph.D, M.D.

Date Posted: October 15, 2007
Date accessed: June 22, 2009
URL http://www.fda.gov/ForConsumers/ConsumerUpdates/ucm103331.htm
About Author
Celia M. Witten, Ph.D., M.D.,
is Director of the Office of Cellular, Tissue and Gene Therapy at FDA’s Center for Biologics Evaluation and Research (CBER). She worked for more than 10 years as a practicing physician at the National Rehabilitation Hospital in Washington, D.C., before joining FDA as Division Director of General, Restorative, and Neurological Devices in the Center for Devices and Radiological Health. Before attending medical school at the University of Miami, she received a Ph.D. in Mathematics from Stanford University.