While modern science has solved some access issues, others still remain. “If we go back six years, the biggest barrier was the fact that there were not donors to match,” says Ronneberg. “As more patients have a donor, we now are facing issues around referral. Thousands of patients are not getting referred to transplant centers in a timely manner, those that are oftentimes are facing major barriers from a financial perspective.”
Dr. Eneida R. Nemecek, director of Pediatric Cell Therapy and Transplant & Cancer Clinical Research at Oregon Health & Science University, agreed that geography is a big problem for patients who need care. She said it’s a symptom of a bigger problem with the way our health systems have been designed. “When we're designing systems for healthcare, we forget that we're designing the systems for the patient. The system designs the system for itself, and then we try to adjust it to the patient,” Nemecek says.
Because of how specialized CAR-T therapies were designed and tested, there can be natural bottlenecks when it comes to scaling up and disseminating it to a wider audience. Dr. David L. Porter, director of the Center for Cell Therapy and Transplant at Penn Medicine, says that advanced therapies like bone marrow transplants or cellular immunotherapies are developed and largely performed at major academic institutions in big cities, usually as a proof of concept. However, swaths of the population in the country do not live near one of these institutions or have to drive hours to one that has both the technology and the expertise. Now, there are seven FDA-approved commercial CAR-T cells, and the manufacturers have no problem receiving cells from collection centers, manufacturing them, and returning them. “The barrier is actually collecting the cells,” Porter says. “It's a very specialized process.”
Additionally, undergoing collection and treatment causes huge displacement in patients and their caregivers’ lives. The entire process of treatment and testing can take weeks. “We have those discussions all the time that people would accept suboptimal therapy and give up the potential for curative therapy because they can’t do that,” Porter says. “There are an enormous number of initiatives trying to improve, and they move the needle a little bit.” One of these methods is to try to give these therapies in community health centers closer to where the patients are. Porter and his colleagues recently conducted a pilot study at the University of Pennsylvania with two of their community partners to implement high-end CAR-T cell therapy in a community setting. Over the course of about a year, they’ve been able to treat over 60 patients who otherwise largely would not have had access because they would not have traveled to a big city for it.
With science changing fast and treatment outcomes improving, Ronneberg says that it’s more important than ever to “get the education and awareness out to the community,” and provide them with “funds to travel.” As a cancer survivor herself, Ronneberg recalled having a hard time navigating the process even when she had everything at her fingertips—a husband who’s a doctor, resources from her job at the NMDP. The organization’s focus has moved beyond simply finding donors to now getting patients to the right treatment they need at the right locations, whether that’s an academic center or a community center.
To this end, Nemecek and her colleagues have been working on projects like ACCESS Initiative, in partnership with NMDP, in order to start training community centers and equip them with accreditation needed to collect cells. “With valuable telehealth, with use of technology,” Nemecek says, “that’s really where the future of our field is.”