How is gene therapy studied?
Any new treatment needs to go through a process to prove that it works and is safe. It is a long and detailed journey to approval—here is an overview:
- Early on, scientists study and begin to understand the details of the possible treatment in a lab. This research takes place long before a person receives treatment.
- Following the lab studies, the research moves into a process called clinical trials. These clinical trials are broken up into phases. Each phase checks the effects of the treatment in people.
- The final phase of the clinical trial process is to submit to the FDA for approval of the treatment.
- The process doesn’t end when the FDA approves the treatment. Many studies continue long after the treatment receives approval to monitor long-term effects.
Can I join a clinical trial if there is a gene therapy being studied for a disease that I have?
Only people who are eligible can join a clinical trial. Study investigators create a set of guidelines that have to be met to join the trial. Currently, researchers are studying gene therapies for:
- Cancer
- Genetic diseases
- Infectious diseases
Talk to your doctor to find out if there is a clinical trial for a disease you or a loved one may be suffering from.
2015
- The FDA approved the first gene therapy for use in the United States, which was a treatment for
melanoma recurrent after surgery
2017
Inherited retinal disease Acute lymphoblastic leukemia - Certain types of
large B-cell lymphoma
2019
Spinal muscular atrophy
2020
Mantle cell lymphoma (a rare form of non-Hodgkin lymphoma)
2021
- Certain types of
large B-cell lymphoma - Certain types of
multiple myeloma
2022
Beta thalassemia
Gene therapy may offer hope for millions of people affected by genetic diseases.
As the field of gene therapy continues to evolve, talk to your doctor if you have questions.
You’ve now seen a bit about gene therapy and where it’s heading. The next step in the journey is to take a closer look at gene addition, a type of gene therapy.
Learn more
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