Industry Leaders Detail Transformational Strategies to Mainstream CAR-T Care

The future of chimeric antigen receptor T cell (CAR-T) therapy hinges on overcoming systemic barriers, ensuring quality, and scaling manufacturing rapidly to meet unprecedented demand, according to a panel discussion at the 2025 Cell and Gene Meeting on the Mesa session, "The Evolution of CAR-T from Niche Treatment to Mainstream Care" (1). While CAR-T offers curative potential, a healthcare professional gap persists, leaving an estimated 80% of eligible patients currently not receiving the therapy (2). This challenge forces the cell and gene therapy (CGT) sector and the broader biopharmaceutical industry to rethink policy, logistics, and delivery models, the industry leaders said during the panel (1).

Addressing this access gap is crucial for realizing the commercial promise of these groundbreaking therapies. Joe DePinto, head of Cell, Gene and Advanced Therapies at McKesson, noted that incremental improvements are insufficient, advocating for "transformational progress for these patients" (1).

Why is the current healthcare system not ready for CAR-T scale?

Logistical and financial constraints are preventing widespread access, even for approved therapies, and the best care is local care, according to the panelists, yet patients and their families often must travel significant distances to reach the limited number of qualified treatment centers. Navneet Majhail, MD, FASTCT, physician-in-chief of Blood Cancers at Sarah Cannon Cancer Network, underscored this complexity, stating, “I think we [should] make this simple … but this is so complicated.”

Majhail noted that a major factor complicating the system is the payer mix; between 30% and 50% of the US patient population relies on Medicaid/Medicare coverage that is highly sensitive to changes in government payment procedures and health exchange cutbacks. Gustavo Mahler, PhD, CEO of Cellistic, concurred that the regulatory environment is volatile, emphasizing that the situation is a “mixed bag sometimes.”

The logistical difficulty extends to the patient experience, where complexities in obtaining treatment approvals have been compared with the speed of bespoke gene editing. “How often do people listen to patients, or how often do we talk about all these other barriers, is [something] we need to ask ourselves often,” said George Eastwood, executive director of the Emily Whitehead Foundation, emphasizing that the patient experience must be central to policy changes.

How are industry leaders redefining quality and access standards?

To expand access, stakeholders are working to decentralize care, moving beyond academic centers into community and outpatient settings, the panelists explained. This shift requires reforming quality standards. Historically, accreditation by organizations like the Foundation for the Accreditation of Cellular Therapy (FACT) was tied to the complex requirements of the transplant setting, Majhail said. He explained that FACT is now working on "fit-for-purpose" CAR-T standards, removing requirements—such as certain nurse-patient ratios or on-site adoption—not necessary for community outpatient sites.

Standardization across sites is critical, and the delivery of high-quality care must not be compromised by expansion, especially when managing toxicities like cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome, according to Majhail. New care models, such as those partnering full-service community sites with experienced adverse event hospitals, are emerging, the panelists noted.

Rebecca Poehnelt, PhD, vice-president, Global Commercial Strategy Lymphoma, Leukemia and Cell Therapy, Johnson & Johnson Innovative Medicine, summarized the aspirational goal for the industry, saying, “I look forward to the day, and maybe having some significant progress or milestones, on when [treatment is] a clinical decision between the physician and the patient, and it no longer involves any logistical challenges, whether it be on the [treatment administration] side or the patient side.”

What does the expansion into autoimmune diseases mean for manufacturing?

The biggest impending change for the broader biopharmaceutical industry is the expansion of CAR-T into autoimmune diseases, such as stiff person syndrome or myasthenia gravis. According to Warner Biddle, executive CEO of Kyverna Therapeutics, Kyverna is aiming to be the first CAR-T company to launch in the autoimmune disease space. He emphasized that this disease space represents an unprecedented patient population far exceeding hematological oncology (1).

Scaling to treat hundreds of thousands of patients will likely necessitate a major technological pivot from current autologous processes to allogeneic-based cell therapy (I.e., off-the-shelf), Mahler pointed out. This industrial scaling is vital for reducing the cost of goods dramatically, targeting between $10,000 and $20,000 per dose, to make treatment affordable for the healthcare system, he noted. He also explained that, while allogeneic solutions address volume, autologous durability (five years plus in hematology, two years off immunosuppressants in autoimmunity) offers a potent "one-and-done" value proposition.

As these therapies become routine, education for both referring physicians and patients is key. By changing the narrative from "science fiction" to routine care, the industry can improve referral rates and access. DePinto concluded that normalizing these breakthroughs is essential. “The entire ecosystem plays a role in that education … that this isn't ‘voodoo’, this is science and high science—and exciting breakthrough science,” he stated.

Meanwhile, Kyverna’s move into autoimmunity is expected to be a proving ground for the industry’s readiness to scale. "Being in a position to be the first CAR-T company to launch in autoimmune spaces may help fulfill some of the goals that [this panel] have touched on,” Biddle said.

The Cell and Gene Meeting on the Mesa occurred Oct. 6–8 in Phoenix, Ariz.

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References

1. DePinto, J.; Biddle, W.; Eastwood, G.; et al. The Evolution of CAR-T from Niche Treatment to Mainstream Care. Presentation at Cell and Gene Meeting on the Mesa. Oct. 6, 2025. https://meetingonthemesa.com/agenda/
2. Nze, C.; Flowers, C. R. Barriers to Accessing Cellular Therapy for Patients Receiving Care in Community Practices. Hematology Am Soc Hematol Educ Program. 2023, 2023 (1), 382–385. DOI: 10.1182/hematology.2023000518