Jaap Jan Boelens
MD, PhD
Interview by Hannah W. Song, PhD and Shyam Bhakta, MD, MBA
Cell and gene therapies (CGTs) offer a new paradigm of medicine. The scientific community must now work to ensure they reach all patients.
CGTs use a patient’s own cells – often immune cells or stem cells – and genetically engineer them to address the disease directly. Examples include T-cells for blood cancers like leukaemia and lymphoma, as well as stem cells for sickle cell disease. When the therapy works, the results can be transformative, even lifesaving.
Barriers to access
However, costs of CGTs are high, and logistics for delivering them are complex. These barriers, on top of a fragmented healthcare system, mean that most patients can’t reliably access CGTs.
For example, of the 18 commercial engineered T-cells available for blood cancers, half are ineligible for insurance reimbursement, and ultimately only three of ten eligible patients receive them1,2 – despite patients achieving long-term remission after therapy.
To overcome this, the International Society for Cell and Gene Therapy (ISCT) unites the world’s CGT community to work together on solutions spanning healthcare, laboratory and pharmaceutical industries.
We’re still going to face questions of access,
side effects and efficacy. It’s only the beginning
Finding gaps, identifying solutions
Dr Jaap Jan Boelens, Chief, Pediatric Transplantation and Cellular Therapies Service, Memorial Sloan Kettering and Chief Medical Officer of ISCT, recognises the need for providers and clinicians to bring their unique experience to the table alongside scientists and regulators.
“How can we learn from real-world data? The needs in Europe may be different from those in South America or Asia, but there’s significant common ground,” says Boelens.
Boelens points to progress made by regional hubs, such as Spain’s ARI Program and India’s ImmunoACT, offering homegrown CGTs priced at a fraction of those from traditional pharmaceutical companies. So far, these therapies seem to deliver similar benefits as their commercial counterparts.
The future of CGTs
The field has made substantial progress, and it continues: the same engineered T-cell therapies developed for blood cancers have also shown early success in reversing autoimmune disorders in highly treatment-refractory patients.
While the list of approved CGTs grows, according to Boelens, “We’re still going to face questions of access, side effects and efficacy. It’s only the beginning.”
For more information, visit www.ISCTglobal.org.
[1] Ge, A.Y. et al. (2026) Global access to commercial CAR T-cell therapies: a cross-sectional study of health technology assessment across the G20 countries. Blood.
[2] Canales Albendea, M. et al. (2023). Comparative analysis of CAR T-cell therapy access for DLBCL patients: associated challenges and solutions in the four largest EU countries. Front Med (Lausanne).
