Cell & Gene Therapy FAQ

What is gene therapy?

Gene therapy is the introduction or alteration of genetic material within a cell or organism with the intention of curing or treating a disease. Approaches to cancer gene therapy include three main strategies: replacing a mutated gene that causes disease with a healthy copy of the gene, genetic modification to silence a mutated gene, and genetic approaches to directly kill the cancer cells.

What is cell therapy?

Cell therapy is the transfer of cellular material to a patient for treatment or prevention of disease.

How are gene therapy and cell therapy related?

Both therapies have the potential to alleviate the underlying cause of genetic and acquired diseases by replacing the missing proteins or cells causing the symptoms, suppressing expression of proteins that are toxic to cells, or eliminating cancerous cells.

What are clinical trials?

A clinical trial is designed to answer specific questions about vaccines, new therapies, or new ways of using known treatments. Clinical trials invite human volunteers to determine whether new drugs or treatments are both safe and effective.

Clinical trials are conducted in four phases:
Phase I tests a new drug or treatment in a small group for safety;
Phase II expands the study to a larger group of people for safety and effectiveness;
Phase III groups are as large as several thousand to confirm results; and
Phase IV defines the drug or treatment risks and benefits for government approval.

Every clinical study is led by a principal investigator, who is often a medical doctor. Clinical studies also have a research team that may include doctors, nurses, social workers, and other health care professionals.

Clinical studies can be sponsored, or funded, by pharmaceutical companies, academic medical centers, voluntary groups, and other organizations, in addition to Federal agencies such as the National Institutes of Health, U.S. Department of Defense, and U.S. Department of Veterans Affairs. Physicians, health care providers, and other individuals can also sponsor clinical research.

How can I participate in a clinical trial?

To view a list of clinical trials in the U.S. for cell and gene therapy for a specific disease, see Clinical trials, “I agree” and then click on a specific cancer type. The list of clinical trials indicates whether each clinical trial is pending, recruiting (looking for patients), ongoing (have enrolled sufficient patients), or completed.

What is the difference between gene therapy and cell therapy?

Gene therapy involves the transfer of genetic material and the uptake of the gene into the appropriate cells of the body; a set of strategies that modify the expression of an individual’s genes or that correct abnormal genes. Cell therapy is the transfer/administration of live whole cells or maturation of a specific cell population in a patient for the treatment of a disease. Each strategy involves the administration of a specific DNA (or RNA).

What is Immunotherapy?

Immunotherapy stimulates or restores the ability of the immune system to fight infection and disease. Research hopes to harness immune mechanisms against tumor cells, by using the patient’s own immune system or by transferring of antibodies or T-cells from an outside source.

What is Anti-angiogenesis?

Gene medications strangulate cancer cells by cutting off their blood supply.

How are new healthy genes delivered to patients?

Healthy genes are often delivered using a vector. Vector is a gene therapy delivery vehicle or carrier that encapsulates therapeutic genes for delivery to cells. Viral vectors [disabled, not live virus] transfer tumor antigens (proteins found on a tumor cell) which should stimulate an anti-tumor immune response in the body. Viral vectors may also be used to carry genes that can differentiate cancer cells back to normal cells. Viruses can act as a Trojan horse, delivering the therapeutic DNA into the host cell.  Deactivated viruses which are currently being used for human gene therapy, include: adenovirus, HIV, lentivirus, herpes simplex virus and adeno-associated virus.

In vivo gene therapy is when a gene is transferred to cells inside the patient’s body by a vector carrying the therapeutic genetic material. This form of gene therapy is in vivo because the gene is transferred to cells inside the patient’s body. Ex vivo gene therapy is the process when cells from a patient are removed, cultivated in the laboratory, and then injected back into the patient. This is called ex vivo because the cells are studied outside the patient’s body.

Oncolytic viruses (man-made) are engineered to replicate only or predominantly in cancer cells and not in normal human cells. These viruses grow in cancer cells and cause the cancer cells to burst, releasing more oncolytic viruses to infect surrounding cancer cells. These viruses can also carry therapeutic genes to increase toxicity to tumor cells, stimulate the immune system or inhibit angiogenesis of the tumor.