- Evaluation and Preparation: Before beginning treatment, patients undergo a comprehensive evaluation to ensure that gene therapy can be administered safely and effectively. Testing may include blood work, imaging studies, organ function assessments, infectious disease screening, and other specialized evaluations. These assessments help determine eligibility for treatment and establish a personalized care plan. Before treatment begins, patients identify a dedicated live-in caregiver, typically a family member or close friend, who will provide essential support throughout treatment and recovery.
- Stem cell mobilization and collection: Your body makes and stores stem cells in your bone marrow. To collect these cells, patients receive medications that encourage stem cells to move from the bone marrow into the bloodstream. The stem cells are then collected through a process called apheresis. During apheresis, blood is circulated through a machine that separates and collects stem cells while returning the remaining blood components to the body. Apheresis takes approximately 5 hours per day and may require multiple days. Your collected stem cells are then sent to a specialized laboratory to undergo manufacturing. Collection may require admission to the hospital, so you can get your mobilization medications at the appropriate time before each day of collection.
- Gene Therapy Manufacturing: After collection, the stem cells are transported to a specialized manufacturing facility where they undergo genetic modification. Depending on the therapy, this may involve adding a functional gene or editing an existing gene to restore healthy blood cell production. The manufacturing process typically takes several weeks and is one of the most sophisticated forms of personalized medicine available today.
- Conditioning Chemotherapy: This refers to high-dose chemotherapy that you will receive to clear out faulty stem cells in the bone marrow and make room for the genetically modified stem cells to be given back to you.
- Gene Therapy Infusion: The genetically modified stem cells are infused through an intravenous line in a process like a blood transfusion. After infusion, cells travel naturally to the bone marrow, where they begin rebuilding healthy blood production.
- Engraftment and Blood Cell Recovery: Following infusion, the genetically modified stem cells establish themselves within the bone marrow and begin producing new blood cells carrying the desired genetic change. This process, known as engraftment, occurs gradually over several weeks and is monitored closely by the care team.
- Recovery and Monitoring: Recovery from gene therapy takes time and requires close monitoring and follow-up. The recovery period typically extends over several weeks to months. During this time, many patients require assistance with daily activities, including transportation, household responsibilities, and attending medical appointments. For this reason, a dedicated live-in caregiver is required following discharge to provide ongoing support. During this time, patients are monitored closely for infections, treatment-related side effects, blood count recovery, and successful engraftment of the genetically modified stem cells. Frequent clinic visits, laboratory testing, and follow-up evaluations are an essential part of the recovery process and help ensure your safety and treatment success. Maintaining good nutrition, engaging in physical activity as tolerated, and getting adequate rest can help promote recovery and overall well-being.
Support and Follow-up Care
A successful gene therapy journey requires close collaboration between patients, caregivers, and the healthcare team. Before treatment begins, patients identify a dedicated live-in caregiver, typically a family member or close friend, who will provide essential support throughout treatment and recovery. Patients receiving gene therapy often remain hospitalized for approximately four to six weeks, although the exact duration varies depending on individual circumstances. After discharge, ongoing follow-up care is critical to monitor recovery, assess how well the gene-modified stem cells are functioning, and identify any potential complications.
At Smilow Cancer Hospital we remain committed to our patients long after treatment is complete, providing expert care and lifelong partnership throughout their journey.
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