Gene Therapies: Revolutionizing Disease Treatment

Gene therapies represent one of the most significant advances in modern medicine. These innovative techniques allow for the modification of genes within a patient's cells to treat or prevent diseases, directly addressing the underlying causes of genetic conditions.

By Sermes CRO

Gene therapies involve the introduction, removal, or modification of genetic material within a patient’s cells to correct genetic defects, replace mutated genes, or introduce new genes that help combat diseases. This approach has the potential to offer long-lasting and even curative treatments for previously untreatable diseases. According to a study published in Gene Therapy, gene therapies have demonstrated high therapeutic value, especially in the treatment of rare and genetic diseases.

Initially, these therapies were developed for rare genetic disorders, such as Duchenne muscular dystrophy or hemophilia, because their primary focus is to correct or replace defective genes. However, advances in biotechnology have allowed gene therapy to also transform the treatment of more common diseases, such as certain types of cancer (e.g., CAR-T therapies for leukemias and lymphomas) and neurodegenerative diseases (such as Parkinson’s).

Therefore, gene therapies are revolutionizing the treatment of genetic diseases and, more broadly, other diseases with genetic or cellular components.

Advances in Gene Therapies in Spain

In Spain, hospitals have been at the forefront of research and application of gene therapies. Since the implementation of the National Health System’s Advanced Therapies Approach Plan in 2018, numerous advances have been made in this field. This plan has allowed the integration of advanced therapies into the public health system, facilitating access to innovative treatments for patients. In this article, we provide some examples of gene therapies applied in Spanish hospitals.

CAR-T Therapy

One of the most notable examples of gene therapies in Spain is CAR-T therapy. This technique involves the genetic modification of a patient’s T lymphocytes to identify and destroy cancer cells. Since 2018, Spanish hospitals have made more than 1,859 requests for CAR-T treatments, with an approval rate of 85.5% by the Ministry of Health. These treatments have been primarily used to combat hematological cancers such as leukemia and lymphomas. A study published in the Spanish Journal of Cardiology highlights the efficacy of these therapies in treating hematological diseases.

Gene Therapy for Hemophilia

Another significant advance has been in the treatment of hemophilia, a genetic disease that affects blood clotting. At the La Paz University Hospital in Madrid, a gene therapy has been implemented that introduces a functional gene for the missing clotting factor in patients. This treatment has shown promising results, significantly improving patients’ quality of life and reducing the need for continuous treatments. An article in Haemophilia details the positive results of these clinical trials.

Gene Therapy for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a genetic disease that causes progressive muscle weakness. At the Sant Joan de Déu Hospital in Barcelona, a gene therapy has been developed that introduces a functional gene for dystrophin, an essential protein for muscle function. Preliminary results have been encouraging, showing improvements in patients’ strength and mobility. A study in The Lancet supports these findings, highlighting the safety and efficacy of the therapy.

The Road Ahead for Gene Therapies

Despite the advances, gene therapies face several challenges. One of the main ones is the high cost of these treatments, which can limit their accessibility for some patients. Additionally, there are ethical considerations regarding genetic modification and potential long-term side effects. It is crucial that these treatments are carefully evaluated and regulated to ensure their safety and efficacy.

The future of gene therapies in Spain is promising. Personalized medicine, where treatments are specifically tailored to each patient’s genetic needs, will be a key area of development.

It is a reality that gene therapies are revolutionizing the treatment of genetic diseases, offering new hope to patients in Spain and around the world. With concrete examples such as CAR-T therapy, advances in the treatment of hemophilia, and Duchenne muscular dystrophy, Spanish hospitals are leading the way in the application of these innovative techniques. As we continue to advance in this field, it is essential to address the challenges and ethical considerations to ensure that all patients can benefit from these transformative treatme.

Catalá, E., Iacoboni, G., & Barba, P. (2022). Therapy with T lymphocytes with chimeric antigen receptor (CAR-T) in patients with aggressive B-cell lymphoma. Current perspective after a decade of treatment. Clinical Medicine, 158(7), 327-332. https://doi.org/10.1016/j.medcli.2021.10.005
Puerto, G. (2023, July 5). A new Spanish CAR-T therapy achieves complete remission in 67% of patients with multiple myeloma. Responsible Medicine. https://medicinaresponsable.com/enfermedades/terapia-cart-contra-mieloma-multiple
U.S. Food and Drug Administration. (2023, 22 de junio). FDA approves the first gene therapy for the treatment of certain patients with Duchenne muscular dystrophy. https://www.fda.gov/news-events/press-announcements/la-fda-aprueba-la-primera-terapia-genica-para-el-tratamiento-de-ciertos-pacientes-con-distrofia