Gene Therapy Requires More Than an Infusion: ASTCT Experts Detail Hidden Barriers to Care

At Cohen Children’s Medical Center, a 13-year-old referred for gene therapy required 2 stem cell collection attempts. The first was unsuccessful; after the second, the manufactured product did not meet a required specification. Resolving the problem meant new approvals and approximately 4 additional months before the patient could receive an infusion, Amelia Halac, MD, told attendees at the 2026 American Society for Transplantation and Cellular Therapy (ASTCT) Gene Therapy Summit in Boston.¹

“Those milestones are key and essential, but they don't really tell the whole story,” Halac said of collection, manufacturing, and infusion.¹ The case illustrated a problem shared by 4 presenters: delivering an advanced therapy requires a care program that can adapt when patients, products, and families are not ready on the same schedule.

When Manufacturing Delays Disrupt Treatment

Halac, of Cohen Children’s Medical Center, reported that the program had infused approximately 28 patients after collecting cells from roughly 40, requiring more than 60 collection cycles. These figures reflect the center's experience, not national manufacturing outcomes.¹

Yonatan Diamond, MBBS, FRACP, explained that patients with hemoglobinopathies may need changes to disease-modifying therapy, transfusion schedules, and organ assessments before collection or conditioning. The longer the interval, the greater the potential clinical and practical burden.

“Every week you wait; that's another blood transfusion. It's another exposure to our antigens. It's more iron,” he said.¹

He described delays as routine rather than exceptional: “Waiting is the norm. It's not the exception.” When a cell product is delayed, his team asks whether the patient can safely remain prepared or whether the treatment plan should be reassessed.

“The first thing we ask is, how realistic is this? Like, let's not be optimistic. Let's be realistic for the family.”¹

The 2026 ASTCT and International Society for Cell & Gene Therapy recommendations likewise emphasize coordinated mobilization, apheresis, conditioning, fertility preservation, and product manufacturing for sickle cell gene therapy.² For pharmacy teams, a postponed infusion may require reviewing medication holds, transfusion-related therapies, conditioning orders, and supportive care plans rather than simply shifting a date.

The Financial Burden Families Carry

Stephanie Fooks-Parker, MSW, LSW, OSW-C, described lessons from pediatric CAR T-cell therapy and transplant services at Children’s Hospital of Philadelphia. Travel, lodging, missed work, and care for siblings could make treatment difficult even for families with a planned clinical pathway.

“If you have two caregivers, and one stops working, you lose funds. They have to travel back and forth to their center, also taking care of their home needs as well,” she said.¹

Previously, the program reimbursed qualifying travel costs, but families still needed money upfront. Some could not cover those expenses while awaiting repayment. Fooks-Parker described working with lodging providers, home-center social workers, and outside foundations when financial assistance became more limited.

She also questioned unnecessary repeat visits: “Why can't we consent to collection in the same trip so we reduce the trip by one?”¹

That seemingly small scheduling change can matter when families must travel across the country.

These concerns extend beyond any single center. Researchers have identified substantial geographic, infrastructure, and financial barriers to sickle cell gene therapy.³ In a 2026 survey of 82 evaluable families with children who had sickle cell disease, 60% reported household material hardship, and only 40% reported that they could access a hypothetical curative therapy with their existing resources. The survey measured reported access, not actual uptake of approved gene therapies.⁴

Making Coordination Visible to Patients

At Cohen Children’s, Halac described separate clinical, manufacturing, insurance, and family-readiness workflows that must converge before treatment. Standardized checklists helped identify tasks, but they could not anticipate each patient's circumstances. Her team now reviews changes in clinical status, product availability, and family readiness across multidisciplinary meetings, with named responsibility for follow-through.¹

“It is important for us to give them one point of contact. If that person doesn't know the answer, for sure they're going to find the answer,” Halac said.¹

The approach also requires honest discussion of what patients and families understand about the next step. A published consensus statement on psychosocial readiness for sickle cell gene therapy recommends assessing practical support needs and coping resources to prepare patients for treatment rather than using psychosocial concerns as automatic exclusion criteria.⁵

Pharmacists can contribute by reconciling medication plans after schedule changes, verifying conditioning readiness, coordinating infection prophylaxis, and clarifying medication instructions when care crosses departments. Those are pharmacy practice implications of the speakers' operational concerns, not tasks they assigned exclusively to pharmacists.

Building Follow-Up That Lasts

Ivory White, APRN, FNP-C, of Texas Children’s Hospital, argued that the demands of gene therapy continue long after discharge.

“The current model works clinically, but it just asks the patients to do everything, right?” she said, describing patients juggling hematology, transplant, fertility, pain, and psychological services.¹

Her team is developing a coordinated follow-up model with shared visits, a nurse navigator, and flexible scheduling.

“We want to have one care home, one team, one longitudinal plan, and one goal,” White said.¹

The proposed pathway extends to 15 years, with monitoring tailored to treatment phase and clinical stability, including organ complications, immune recovery, and delayed safety concerns.

FDA guidance recommends risk-based long-term observation for gene therapy products with potential delayed adverse events; not every product carries the same follow-up requirement.⁶ White's model seeks to make surveillance realistic for patients who live far from their treatment centers, not merely available on a schedule.

Programs must therefore assess more than successful infusions. As Halac reminded attendees, “Every decision you make, every change will affect their quality of life.”¹

REFERENCES

1. Fooks-Parker S, Halac A, Diamond Y, White I. Designing a gene therapy program that patients can actually use. Presented at: ASTCT Gene Therapy Summit; November 5, 2026; Boston, MA.

2. Sharma A, Kassim A, Thompson A, et al. Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy. Transplant Cell Ther. 2026;32(7):778-791. doi:10.1016/j.jtct.2026.03.019

3. Sharma A, John TD. Dismantling cost and infrastructure barriers to equitable access to gene therapies for sickle cell disease. Lancet Haematol. 2024;11(8):e556-e559. doi:10.1016/S2352-3026(24)00175-3

4. C. A. Kelly, M. A. Paul, M. Griffin, et al. “ Out of Reach: Understanding Resource Barriers to Gene Therapy for Pediatric Patients With Sickle Cell Disease.” Pediatric Blood & Cancer 73, no. 10 (2026): e70536. https://doi.org/10.1002/1545-5017.70536

5. Hardy SJ, Crosby LE, Porter JS, et al. Assessing Psychosocial Risk and Resilience to Support Readiness for Gene Therapy in Sickle Cell Disease: A Consensus Statement. JAMA Netw Open. 2024;7(8):e2429443. Published 2024 Aug 1. doi:10.1001/jamanetworkopen.2024.29443

6. US Food and Drug Administration. Long term follow-up after administration of human gene therapy products: guidance for industry. Published January 2020. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/long-term-follow-after-administration-human-gene-therapy-products