Gene Editing (CRISPR) Opens the Door to New Therapies for Rare Childhood Cancers

Research conducted by a team from the Carlos III Health Institute (ISCIII) concludes that a strategy based on CRISPR gene editing technology has demonstrated positive results in preclinical models of Ewing sarcoma, a type of childhood cancer considered a rare disease, for which there have been few therapeutic advances in recent years.

The article revealing the results of the research on CRISPR therapy as been published in the journal Cancer Gene Therapy by a team from the Rare Diseases Research Institute (IIER) of the ISCIII, led by Javier Alonso. The authors of the paper also belong to the Rare Diseases Area (CIBERER) of the Center for Biomedical Research Network ( CIBER -ISCIII).

The study on CRISPR now published is a continuation of the work published in 2021 by the same ISCIII team, which laid the groundwork for proof of concept in cellular models of this therapeutic strategy

On this occasion, the authors have gone further and demonstrated the efficacy of this gene therapy for the treatment of Ewing’s sarcoma in preclinical models of the disease, incorporating a significant novelty: thanks to a specific promoter, CRISPR/Cas9 technology was expressed exclusively in Ewing’s sarcoma cells, thus eliminating the potential side effects of gene editing.

Furthermore, according to IIER-ISCIII research professor Javier Alonso, it has also been demonstrated that it is possible to use adenoviruses, which are already used in other clinical approaches, to deliver the therapy directly to tumor cells, which opens the door to a medium-term clinical application of this CRISPR therapy against Ewing sarcoma.

The advances in this (CRISPR) strategy, explained Alonso, “represent a qualitative leap of great significance, since we use an ‘Ewing-specific’ promoter that can open up a huge range of opportunities for the treatment of this disease:” In fact, his group is working on using this promoter to express other therapeutic proteins and thus advance toward the development of new, highly specific therapies that can be evaluated in clinical trials with patients .

Saint T. Cervera, a predoctoral researcher at the IIER-ISCIII and the first author of the article, also explains that thanks to the use of this “Ewing-specific” promoter, the possibility of applying this type of strategy in clinical practice is closer:

“We are currently evaluating other therapeutic genes that in some cases are yielding better results than those shown in this study. Most importantly, we have seen that this strategy (CRISPR) is highly specific, meaning that the therapy only affects tumor cells, significantly reducing the likelihood of side effects. We hope that these new approaches can soon be evaluated in clinical trials in patients with this disease.”

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(Featured image by Sangharsh Lohakare 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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