Expert: Pharmacists Guide the Future of Gene Therapy in Oncology and Hematology

In an interview with Pharmacy Times®, Varshini Kanukollu, PharmD, BCOP, pediatric oncology clinical pharmacy specialist at Memorial Sloan Kettering, discussed how gene therapy offers a new treatment approach by modifying a patient’s cells to address the root cause of disease, providing a potentially curative alternative to traditional chemotherapy or supportive care. She noted that pharmacists play an essential role throughout the process, from patient selection and education to preparation, administration, and supportive care management. Kanukollu emphasized that while long-term monitoring guidelines for some gene therapies are still evolving, pharmacists remain central in ensuring safe treatment and follow-up.

Pharmacy Times: What are the key differences between traditional chemotherapy/targeted therapies and gene therapies?

Varshini Kanukollu, PharmD, BCOP: Gene therapy offers a promising new treatment method for many disease states by allowing us to genetically modify a patient’s cells and target the underlying problem. We can now enhance or modify the body’s immune system to help immune cells fight against disease.

For oncologic disease states like leukemias and lymphomas, traditional chemotherapies rely on targeting rapidly dividing cells, including cancer cells, but they are associated with many adverse effects that can significantly affect a patient’s quality of life. For hematologic diseases such as sickle cell disease and transfusion-dependent beta thalassemia, current therapies include either supportive care or disease-modifying therapies that reduce disease severity without correcting the underlying cause.

Historically, hematopoietic stem cell transplant has been considered a curative option, but the search for an appropriate donor and the risks of complications limit its use. Gene therapies now offer another potentially curative option by modifying a patient’s genome and addressing the underlying cause. Beyond these examples, gene therapy has shown benefit and has been approved in treatment areas such as hemophilia, leukemia, lymphoma, spinal muscular atrophy, and other rare genetic conditions.

Pharmacy Times: What is the pharmacist's role in the process of gene therapies, from patient selection and pre-infusion care to long-term monitoring and follow-up?

Kanukollu: Pharmacists play a key role in the complex and lengthy gene therapy process, from interprofessional collaboration and patient education to supportive care management.

In the early phases of patient selection, multidisciplinary collaboration is essential to determine treatment options and whether a patient qualifies for gene therapy. Once a patient is approved, pharmacists play a large role in education, particularly when reviewing the different types of gene therapy, such as CRISPR technology or viral vector technology.

There are also many operational and financial considerations, including insurance approval, staff education on preparation and administration, and review by the pharmacy and therapeutics committee. Once a product is identified and approved, significant preparation is required before administration. For example, in my presentation on sickle cell disease and transfusion-dependent beta thalassemia, I reviewed preparative requirements such as discontinuing disease-modifying and iron chelation therapies at specific time points.

Most of the gene therapy products I discussed require patients to undergo a myeloablative conditioning regimen with busulfan before administration. For many patients, this is their first and only exposure to chemotherapy—and at high doses. Pharmacists can play a critical role here by providing patient education on chemotherapy, managing chemotherapy side effects, and overseeing supportive care such as infection prophylaxis, nausea and vomiting management, and pain management.

In terms of long-term monitoring and follow-up, this area is still evolving for gene therapies. For certain cellular therapies, like CAR T-cell therapies in hematologic malignancies, we already have a strong understanding of post-treatment monitoring. However, for gene therapies in conditions such as sickle cell disease and beta thalassemia, much of the long-term data is still being developed.