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Miguel Forte, MD, PhD
ISCT President
mC4Tx
Belgium

We are just two months away from our ISCT Scientific Annual Meeting in New Orleans, under the theme of “Driving Global Innovation Towards Patient Access”.

It will be the highlight moment of the year for all of us who are focused on developing cell and gene therapy products for the benefit of patients. Also, a moment to enjoy great science, fantastic company and networking, some fun, and discussions on the hot topics of today, ranging from challenges in the political environment to ethics. A key topic, as stated on the main meeting theme, will be patient access. Medical need is global, but access is limited with only a fraction of approved and eligible patients, about 20%, receiving the benefit of cell and gene therapy products.

In this context, and in a challenging geo-political global environment, cell and gene therapies are crossing a “wintery period” with impacts on investment and funding, innovation, challenges in patient access and overall concerns for the continued development of the field and new products. Together, we must address this daily, jointly as ISCT membership, and together at our ISCT annual meeting! And we will!

Patient access to C&GT products continues to lag behind patients’ needs and stakeholders’ expectations. Multiple factors impact patient access, and it is important to work on all of them to reduce the reality and perception of these hurdles. From a product perspective, general perceptions of lengthy and costly development, manufacturing and logistics are associated with requirements for significant upfront investment and financial barriers to adoption of these products. This calls for the need to expand technological innovation, through new enabling solutions and manufacturing strategies, both central and distributed, together with new models of payment and reimbursement. Significant as well is the limited availability of site infrastructure and workforce training and development in many areas, from research and development to regulatory and clinical contexts. Finally, patients and health care professionals’ education and perception on the risk-benefit balance for treatment is critical for the acceptability of these therapies ⁽¹⁾. Launching these therapies adequately, with all these aspects proactively in mind, will be critical for the adoption success. 

ISCT as a global society is conscious and active in fostering global access. Efforts in various geographies and countries, like India, Brazil and elsewhere, are shaping the future of therapy access with alternative models of product development and production, together with private and public partnerships, leading to interesting opportunities to expand patient access ^{(2, 3)}

Difficulties surrounding C&GT adoption challenge expectations towards return on investment and results in more risk-averse investor behavior and lower willingness to invest, diverting focus to other technologies, even if overall investment in biotech continues to be slow to improve! All these topics will be at the center of many of our discussions at ISCT 2025 New Orleans, in the translational pathway program, the roundtable sessions and the innovation zone.

Another chilling “wintery wind” is negatively impacting biomedical research and translational work, impacting primarily the US, but certainly with global consequences. The new US administration is “freezing” grants and R&D academic work impacting biomedical science ⁽⁴⁾. Other measures include limitations to investment (like the capping of indirect costs at 15%, down from 28-60% previously), and delaying or cancelling decisions (42 out of 47 NIH meetings to review grants have been abruptly cancelled, effectively suspending new grant issuance). These actions are placing academic and public research activities and staff at risk with consequences in the short term and long-term outlook for US leadership in research and, even more importantly, with negative impacts to the translational activities critical to the development of products, cell and gene among them, for the sake of patients’ needs ⁽⁵⁾. Calls for this to be addressed are mounting, and discussion of the implications and consequences and possible solutions will, as well, be part of the relevant topics again at our Annual Scientific Meeting.

With spring's arrival, our Annual Meeting will happen!

We have the responsibility towards the field, and above all, towards the patients in need, to foster the development in cell and gene therapy so that we can challenge the negative environment. We are called upon to address the perceptions and realities on complexity, cost, and levels of adoption of these products. Increasingly, we might focus on the value delivered to unmet medical needs with long term benefits and lasting cures. We must turn winter into spring, improving the current environment through technological innovation, ethical development, and the fostering of regulatory discussions from initial discovery all the way to launch. Along the way, we must continue training the workforce, enabling patient access and, very importantly, increasing the confidence and willingness for investment and public funding to support the activities that result in true long term and curative therapeutic value to patients in need.

The Annual Meeting will be the right place to contribute to this responsibility, sharing and learning about innovation, developments and opportunities in cell and gene therapy and raising the enthusiasm in the field. Let us live up to our joint responsibility and turn the current Winter into Spring in New Orleans and beyond. Let's do our part together to be there and carry back home the knowledge, the connections and the enthusiasm for the value that we are creating for the cell and gene therapy field and our patients.

Looking forward to seeing you there!

1) Navigating Patient Access Challenges When Bringing Your CGT Product To Market; Cell & Gene, February 2025

2) Cutting edge cancer therapy is made in India – at one tenth of the cost, S Mallapaty, Nature vol 627, 709

3)  INCA e Fiocruz iniciam parceria para beneficiar pacientes oncológicos do SUS, INCA gov.br

4)   NIH Research Grants still Frozen Despite Law Suits, M Kozlov, Nature vol 638, 870-871

5)   Safeguarding the future of biomedical science in the United States, T Maniatis, Cell 188, 1-3