A starter guide for patients
Understanding gene therapy for sickle cell disease
Today, gene therapy for sickle cell disease is considered experimental and only available to you if you participate in a clinical trial. While there are a number of clinical trials for sickle cell gene therapy, the general process for this journey is similar across all the trials. This can be a long process and it’s important to understand what happens at each phase. Discussion with your physician, clinical trial team or support system should take place before, during and after each step.
If you are referred to a certain trial, you should learn about:
Your eligibility to participate in a trial may be dependent on:
You may also be required to have:
Gene therapy requires stem cells to be collected from your bone marrow or blood, taken to a lab for modification, then returned to you.
Prior to collection, you may:
The stem cell collection process is called apheresis. Each round occurs over two to three days.
To collect your stem cells, you will:
During collection, stem cells flow into a bag, and the rest of your blood returns to your body.
You may require multiple rounds of the preparation and collection phases to provide enough stem cells. There are usually four weeks between rounds.
Make sure you know about potential side effects, including pain crisis.
Your collected stem cells will be taken to a special laboratory where they will be modified, or manufactured and processed. This process can take many weeks after stem cell collection.
The process differs based on the type of gene therapy approach.
The process does not always work.
If the gene-modified stem cells meet the criteria to be returned to your body, the clinical trial team will contact you to schedule your appointment for the next process.
To receive your modified stem cells, you will:
Make sure you know about potential side effects.
Inpatient: 1 month
Because the conditioning process will lower your immune system, you will be moved to a protective environment in the hospital to reduce your risk of infection.
Medical staff will monitor you closely through:
Outpatient: 2–3 months
Once your clinical trial physician determines that your bone marrow and immune system have recovered, you may leave the hospital and move to outpatient care.
Make sure you know about potential complications.
The Democratizing Education Project welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or questions to DemocratizingEd@mail.nih.gov.
These educational materials are for informational purposes only. They are meant to promote your general understanding of gene therapy for sickle cell disease. We encourage you to use these educational materials to talk with your healthcare provider or a clinical trial team.
Sickle Cell Reproductive Health Education Directive (SC RED) advocates for high quality sexual and reproductive health care for individuals living with all types of sickle cell disease
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