IMPACT-CGT- ISCT Joint Session: A Roadmap for Accelerating Access to Life-Saving Therapies in South and Central America
Author:
Maroun Khoury, PhD
IMPACT, Center for Interventional Medicine for Precision and Advanced Cellular Therapy
Chile
Editor(s):
Fernando E. Figueroa, MD, FACP
Universidad de los Andes School of Medicine
Chile
Martin Hernán Bonamino, PhD
Instituto Nacional de Câncer
Brazil
The IMPACT Cell and Gene Therapy Meeting brought together leading researchers, physicians, and specialists in cell and gene therapy and immunotherapy to share their experiences and research on cell and gene therapy and immunotherapy, with a particular focus on the development of CAR-T cells and the impact of these new treatments in diseases such as cancer.
In his introductory remarks, Dr. Maroun Khoury, a member of ISCT Strategic Advisory Council and the Executive Director at IMPACT Center, recalled that there were 96 CGT approved worldwide listed in the last ISCT Global Regulatory Report (December 2023) [1].
He asked the audience, "How many of these are available today for patients living on our continent?” and then he added “NONE”.
However, ANVISA reported that there are 6 ATMP approvals from Brazil: 2 gene therapies and 4 cell therapies [2].
The gap analysis, he presented distinguished, 5 important requirements:
- A certified Infrastructure
- An efficient technology transfer/partnership
- Feasible regulatory guidelines
- Clinical research platform and funding
- Reimbursement policy
IMPACT Center of Excellence, a non-profit institution with public and private funding was born to address that crucial gap. It aims to make medical treatments more accessible and affordable in Chile and the region.
Despite acknowledging the great challenge, IMPACT has made significant progress in the two years since its inception, thanks to funding from the National Agency for Research and Development (ANID) and the Universidad de los Andes hospital.
IMPACT has collaborated to support clinical research by creating an academic-based clinical trial coordination unit called CRIO. Additionally, they have secured additional funds for the creation of a manufacturing platform for the clinical grade CGT products. The center is a lab member of the ISCT and has active participation in expert roundtables with regulatory bodies of the country.
Dr Khoury mentions, “We aim to collaborate with regulatory bodies to establish a regulatory framework that enables access to therapies such as CAR-T and other life-saving therapies. Our goal is to follow science-driven approaches to each stage of the development and production process, bolstered by strong quality control tools, regulatory support and efficient trial execution”.
He explains that they are also participating in trade association such as Pro Salud Chile, which advocates for innovation to benefit patients and individuals.
A panel discussion on the regulation of advanced therapies was held during the meeting. This panel was moderated by the Executive Vice President of Pro Salud Chile, Jean-Jacques Duhart, and included the following panelists:
- Dr. Martin Bonamino, ISCT SCA LRA Committee and INCA, Rio de Janeiro;
- Dr. Puig-Saus, an expert in new therapies with TCR and CAR cells, UCLA, USA;
- Dr. Julian Molina, the representative of the Committee of the Mayo Clinic;
- Dr. Julia Palma, the representative of the Chilean National Cancer Institute (CNIO); and
- Carolina Sepulveda, representative of the Chilean national regulatory agency - ISP.
Dr. Cristina Puig-Saus explained that a high percentage of advanced therapies are saving the lives of patients who would not be cured with other treatments, there is no doubt that this treatment needs to reach the patients independent of where they live.
The regulatory framework for advanced therapies is different today, with constant updates to know what is recommended for different trials. This allows for faster progress in research and better clinical outcomes for patients.
Brazil is a good example of a regulatory system for advanced therapies, with a body that approves from a health perspective and the benefits of a certain therapy. Another body defines the price, considering commercial therapies and academic therapies that need approval from the regulatory agency to test them. To sensitize a government or state to the development of advanced therapies, it must be clear that they may not have the capacity to develop all the therapies needed, but perhaps it will be possible to develop therapies for the most prevalent diseases. In Brazil, they have successfully treated patients with lymphoma and leukemia who have not responded to traditional therapies.
Victoria Picanco, ISCT Regional Treasurer and researcher and associate professor at the Ribeirao Blood Centre and the Cell Therapy Centre of the University of Sao Paulo, shared her recipe for progress in the field: "The most important thing is to bring together the regulatory agency, the government, researchers, and doctors."
The Chilean Ministry of Health and the Institute of Public Health, key bodies in the promotion of advanced therapies in the country, also attended the event. Advanced Therapies is a public policy, but it is important to see it in numbers. The National Plan for Paediatric and Adolescent Cancer includes the pillar of treatment regimens, which includes access for patients with high-risk leukemia without response to treatment. CAR-Ts are a possibility in pediatric oncology in Chile, but there are still issues in the resolution phase and temporalities that are not the same as in pediatric oncology. In the private sector, cell therapy initiatives in adults, such as the IMPACT Centre and Alemana Clinic, have made great advances in clinical trials. From the Ministry, it is visualized being able to promote these initiatives in the public adult sector, covering both the public and private sectors.
Chile is aiming to establish an intersectoral working table to promote regional development policies in advanced therapies, like those in the Ibero-American Network of Advanced Therapies (RITA). The advanced therapy department of the national agency-ISP, led by Carolina Sepúlveda, is working to adapt international regulatory bases to local realities without stifling innovation in the health sector. They aim to organize working groups between different actors to generate regulations that apply to Chile, considering manufacturing, quality control, safety, and bioethical aspects to protect research subjects.
The meeting provided a great opportunity to push advanced therapies, revise regulatory models, and capitalize on international experiences while adapting to local realities.
REFERENCES:
[1] International Society for Cell & Gene Therapy (ISCT). Cell and Gene Therapy Global Regulatory Report. December 2023. Accessed from this link https://higherlogicdownload.s3.amazonaws.com/ISCT/15175d29-b676-4bb3-8e1a-f7de41062a62/UploadedImages/2312_Global_Regulatory_Report.pdf on June 6, 2024.
[2] ANVISA. Registro de Produtos de Terapias Avançadas No Brasil. Accessed from this link https://app.powerbi.com/view?r=eyJrIjoiZGRiYTU4ZTItMDY0Ny00M2E2LTlkYWEtNWE2NjU2MDRjOGNkIiwidCI6ImI2N2FmMjNmLWMzZjMtNGQzNS04MGM3LWI3MDg1ZjVlZGQ4MSJ9 on June 6, 2024.
#RegulatoryWatchdog