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Lilia Carolina Leon-Moreno, PhD
Postdoctoral Researcher
CIATEJ
Guadalajara, Mexico

María Ignacia Cádiz, MSc
Production GMP Facility Manager
Cells for Cells - REGENERO
Santiago, Chile

Desiree Kosan, RN, MS, OCN,CDCES
CAR-T Cell Collections Operations Manager, LATAM
Janssen Pharmaceutical Companies of Johnson & Johnson
Raritan, NJ, United States

Tatiana Maron-Gutierrez, PhD
Researcher
Oswaldo Cruz Institute, Fiocruz
Rio de Janeiro, Brazil

Andrés Caicedo, PhD
Professor
Universidad San Francisco de Quito, USFQ
Ecuador

Every ISCT region has its unique and specific necessities and challenges. Despite being a large region where most countries share cultural backgrounds and languages, there is still a lack of communication and cooperation within the more than 20 countries comprising South and Central America (SCA). Regarding cell and gene therapy, there is a critical lag that has dragged us back in the development of these types of therapies. This, in turn, has led to many countries in the region being among the top 10 medical tourism destinations in the world, offering unproven and unethical stem cell treatments. These hurdles can only be fully understood, addressed, and battled through the involvement of the regional stakeholders.

The SCA Regional Executive Committee has been working in the last few years to deliver key global ISCT objectives and overcome the scientific, regulatory, and industry challenges in our region to level the ground with the lead countries in cell and gene therapy development and to fulfill the ISCT mission to drive the clinical translation of these therapies worldwide.

To increase the sense of community and engagement among scientists across these countries and to encourage and strengthen the network created so far, the SCA Regional Executive Committee hosted a Virtual Town Hall last April. In this meeting, specialists from the region and its collaborators exposed and discussed relevant topics for our community regarding regulation. Opportunity areas were identified and described. 

The main objectives of the Town Hall were:

• Show what scientists in this region are working on.
• Help the community to identify labs and specialists to collaborate with and support each other.
• Promote the active participation of ISCT members in the regional challenges.
  

During the Virtual Town Hall, Regional Executive Committee Vice-Presidents Milena Soares and Antonio Carlos Campos de Carvalho disclosed the ISCT’s Mission, Vision and Values, and the pillars on which the society stands: academia, regulation, and commercialization so that new or non-members know and understand the relevance of the ISCT and the benefits they could have for being part of this global community.

Professor Maroun Khoury from the University of Los Andes in Santiago, Chile, participated as a guest speaker and talked about the Regional Advancement in Cell and Gene Therapies, focusing on Extracellular Vesicles and the ongoing Clinical Trials in the region. He explained how the interest of the biotechnology industries has shifted from RNA, chemical, or antibody-based drugs to extracellular vesicles (EV)-based ones due to their advantages compared to cell therapy: this is a ready-to-use product with simpler logistics. However, he specified that the researchers agreed that although this therapy has much potential in the future, there are still many technical, quality, economical, and regulatory challenges to overcome to bring these therapies to the patients.

On SCA, only one company is developing EV-based therapies: Cells for Cells, a spin-off company from the University of Los Andes in Chile. They are generating a lot of preclinical data and validating the use of EVs for different pathologies. Further, they are initiating Clinical Trials with their EVs and looking to develop GMP products with FDA authorization. They also organized hand to hand with IMPACT (another Chilean research center) seminars and courses in Spanish to increase the researchers, MDs, and students’ knowledge in this field. This is an opportunity for all the region’s countries to follow their example, collaborate and bring therapies to life.

Unfortunately, our region is plagued by clinics exploiting preclinical or anecdotal results to promote unproven and unethical treatments and offering online courses to teach how to administer them. Therefore, Dr. Maroun highlighted the ISCTs and their members’ relevance in population education about investigational, proven, and unproven treatments with scientific information. Accessibility to treatments and accurate, evidence-based information is vital in SCA, and we all should participate in fulfilling this necessity.

In this sense, Dr. Fernando Figueroa, the Co-Chair of the ISCT SCA Legal and Regulatory Affairs Committee (SCA LRA), talked about the lack of a regulatory framework in our region and its consequences. More than 70% of the countries in Latin America have yet to have specific regulations for ATMPs. There needs to be more basic and clinical research. Researchers need help translating preclinical work to a patient-ready treatment, and regulation plays an important role. In some cases, like in Chile, regulation is a significant obstacle. Although significant money is invested in biotechnological entrepreneurship, the regulatory process hampers the efforts of the industry to bring treatments to patients. Despite this, the University of Los Andes in Chile has executed many clinical trials over the years by itself and in collaboration with other countries such as Colombia. Researchers in this area are making considerable effort, but the task is enormous, and we must join forces. Communication between agencies and exchanging experiences with the most developed ones is crucial. Given this, we reiterate the role of the ISCT and its members.

As an example for the region, health regulation specialist at the Brazilian Health Regulatory Agency (ANVISA), Renata Miranda Parca, presented the Advanced Therapy Regulatory Model in Brazil, which is aligned with the benchmark agencies worldwide. Brazil is leading the regional efforts to regulate ATPMs with specific technical manufacturing standards based on its known challenges as complex products: high variability in starting material, high cost, absence of a compendium for quality control, and unknown long-term risks. Because of their work, the World Health Organization (WHO) invited ANVISA to participate in the draft of the document of WHO considerations on the Regulatory Convergence of Cell and Gene Therapy Products. As a result of this groundwork of the agency, Brazil has five gene therapy products approved for their clinical use. Many patients have benefited from this. Despite what has already been accomplished, ANVISA is in constant actualization and will launch the new GMP requirements for ATMP manufacturing. Brazil is the representative of regional potential.

Another essential part of the meeting was the announcement of the committee’s initiatives for the near future, presented by our Regional Treasurer: Dr. Hector Mayani, from Mexico. In the past, the Executive Committee has held regional meetings, joint meetings with different national organizations and the North America Committee, and supported training opportunities in Cell and Gene manufacture.

This year, the main objective is to consolidate cell and gene therapy in Latin America as a result of the following actions:

• Promotion of connectivity, interaction, and networking among regional research teams.
• Boosting interaction between basic and clinical research teams. 
• Establishing and consolidating SCA's regulatory, scientific, and medical committees.
• Presenting and promoting more training opportunities inside and outside Latin America. 
  

Also, our Regional Secretary, Dr. Andrés Caicedo from the University of Quito, presented the Regional Early-Stage Professionals (ESP) Subcommittee, its importance in supporting the Executive Committee’s work, and introduced the newest members to the community. ESPs exchanged their experiences and perks of being part of the ISCT to encourage non-members in the region to join the conversation in the global community.

The meeting concluded with the starring participation of Dr. Patricia Rocco, the Global Secretary of ISCT, who coordinated the roundtable of questions and commentaries in which ESP had the opportunity to externalize their specific necessities from the ISCT leadership. 

In conclusion, prioritizing the advancement and involvement of early-stage professionals within ISCT is crucial for the future growth and success of the society. By actively working to increase their numbers and engagement, providing networking opportunities, and offering resources and support, ISCT can create an inclusive environment that fosters loyalty and long-term membership retention. Furthermore, securing corporate funding for the activities of the ESP subgroup will enable the implementation of impactful initiatives and further enhance the opportunities available for early-stage professionals in the field of ISCT.

After this Town Hall, we are confident we will have more collaborative initiatives and increased regional member participation. We encourage SCA members to post any questions or topic suggestions for future Town Hall meetings through our community forum on the ISCT website.