Advancing Sarcoma Treatment: CAR-T Cell Therapy Offers Hope for Rare Tumors

Although sarcomas represent only a small percentage of all malignant tumors, their diagnosis and treatment remain a challenge. Biotechnology is opening up new therapeutic avenues, particularly in the development of advanced cellular immunotherapies tailored to these rare tumors, such as CAR-T therapies.

Sarcomas are malignant tumors originating in the connective tissue of soft tissues, excluding bones and cartilage. According to the Spanish Association Against Cancer (AECC) , this group includes tissues that support or fill the body: adipose tissue, muscle, tendons, blood vessels, nerves, and deep skin tissue.

This is a rare disease, which is why July 13th is International Sarcoma Day, a key date for raising awareness about this type of mesenchymal tumor.

“Within CIBERONC, an ambitious project targeting CAR-T cells is being developed focused on the development of advanced cellular immunotherapies for pediatric sarcomas, particularly Ewing sarcoma,” explains Enrique de Álava, group leader of the Cancer area at CIBER (CIBERONC), a partner consortium of AseBio.

This project brings together leading groups in translational biomedical research with the goal of generating new therapeutic strategies based on CAR-T cells adapted to the biological characteristics of these tumors, which are rare and have a poor prognosis. “The research combines molecular biology, preclinical models, genetic engineering , and immunotherapy, with a strong translational focus,” the researcher added.

Sarcoma in figures and how CAR-T ctherapies could help

The World Health Organization (WHO) has recognized more than 150 varieties of sarcomas. In Spain, approximately 2,600 new cases are diagnosed each year. Although their incidence is low (approximately 1% of malignant tumors in adults and 13-15% in children), they account for approximately 2% of cancer mortality.

According to data from the Spanish Federation of Rare Diseases (FEDER), around 40% of diagnoses are late or incorrect, and one in four patients initially receives treatment for another condition.

Thanks to current biotechnological tools, it is possible to precisely analyze the expression profile of proteins and genes in both tumors and healthy tissues. This allows for the identification of potential therapeutic targets that are tumor-specific and safe, such as CAR-T therapies.

In the case of sarcoma, new proteins have been detected on the surface of tumor cells that could serve as targets for highly selective immune therapies. “These findings are serving as a basis for designing personalized cell therapies that recognize and attack tumor cells exclusively, minimizing side effects,” explained De Álava.

The team is currently working on validating these therapies in preclinical models and on their future clinical translation, including strategies to overcome the barriers of the tumor microenvironment, which frequently limit the efficacy of immunotherapies in solid tumors. These CAR-T therapies are based on novel tumor targets identified through advanced transcriptomic and proteomic analysis.

Barriers to developing new therapies

Clinical translation, funding, and regulation are some of the obstacles facing treatments targeting rare diseases such as sarcomas. Turning an experimental therapy into an available option for patients requires a complex process that includes preclinical validation, GMP production, ethical and regulatory approvals, and adequate hospital infrastructure.

Despite these challenges, CIBERONC is making efforts to shorten development times, as demonstrated by the recent exceptional compassionate use of one of these cell therapies in a pediatric patient with Ewing’s sarcoma with no other therapeutic options.

CAR-T therapies targeting pediatric sarcomas

“Collaboration is key,” said De Álava. “Our project involves clinical centers, academic groups, and biotechnology companies, combining expertise in molecular diagnostics, immunotherapy, preclinical models, and technology transfer.” This synergy allows for resource sharing, accelerated knowledge generation, and progress toward more personalized medicine.

One of the most recent milestones of the consortium, an AseBio partner, has been the development of a new generation of CAR-T therapies targeting pediatric sarcomas, with promising results in preclinical models and already leading to a compassionate use application. This breakthrough has been jointly led by Dr. Enrique de Álava’s group (Institute of Biomedicine of Seville, IBiS-HUVR) and Dr. Pablo Menéndez’s group (Josep Carreras Institute, in the group of Dr. Anna Bigas), within the framework of CIBERONC.

This project, with a strong translational component, has been made possible thanks to the support of the Ministry of Science, Innovation and Universities through the 2022 Strategic Collaboration call, with co-financing from the Recovery, Transformation and Resilience Plan and European NextGenerationEU funds.

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(Featured image by National Cancer Institute via Unsplash)

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First published in diariofarma. A third-party contributor translated and adapted the article from the original. In case of discrepancy, the original will prevail.

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