Breakthrough Living Drug Offers Hope for Young Cancer Patients in Madrid

A revolutionary living drug developed at a public hospital in Madrid is making waves in the fight against B-cell acute lymphoblastic leukemia, the most common and aggressive childhood cancer. This groundbreaking therapy has already saved the lives of eight young patients who had been declared terminal after enduring multiple relapses and the failure of standard treatments. The innovative treatment involves extracting immune cells from the patient, reengineering them in a laboratory to enhance their cancer-fighting capabilities, and reintroducing them into the body to target and destroy tumors. Pediatrician Antonio Pérez, who leads the research, announced impressive results at La Paz University Hospital: a remarkable 70% survival rate after more than 18 months of follow-up. Among those presenting at the event was 15-year-old Lucía Álvarez from Cádiz, whose story exemplifies the transformative potential of this therapy. Diagnosed with B-cell acute lymphoblastic leukemia at just 17 months old, Lucía's journey through chemotherapy and other treatments was fraught with complications and relapses. However, after exhausting all conventional options, she became one of the first patients to receive this new therapeutic approach, which effectively eradicated her cancer in just a month. This new CART (Chimeric Antigen Receptor T-cell) therapy utilizes a novel strategy that enhances detection of cancer cells by targeting two specific proteins, CD19 and CD22, rather than just one. This dual-action mechanism reduces the risk of tumors evading detection by camouflaging themselves, which is a common challenge in previous therapies. Following the initial treatment, five of Lucía's peers were able to undergo lifesaving bone marrow transplants, and after 20 months of monitoring, eight patients remain alive, showcasing the new therapy's promising prospects. These findings were recently published in the peer-reviewed journal eBioMedicine, inviting cautious optimism about the therapy's future. Famed U.S. immunologist Carl June draws parallels between the success stories of these young patients and biblical resurrections. Lucía and her fellow patients are testament to what is now being referred to as a medical revolution enabling modified cells to save lives. Yet, while the results are remarkable, it is important to note that CART therapies do not always yield success; they effectively save about half of the children with the most aggressive forms of cancer who previously had no treatment options. The journey of these brave young patients has underscored the importance of leveraging public hospital resources to develop such treatments, especially given the substantial costs associated with CART therapies. For instance, Novartis has priced its Kymriah CART therapy at over $300,000 per patient, while the pioneering treatment from the Barcelona Hospital Clínic comes in significantly cheaper at about $90,000. Antonio Pérez continues to advocate for the advancement of new therapies within public healthcare institutions, emphasizing their capability to deliver innovative solutions at a fraction of the price of private alternatives. This is especially critical given the fact that two out of ten children diagnosed with cancer still cannot be treated effectively, despite advances in technology. In addition to the recent successes, the CRIS Unit of Advanced Therapies for Childhood Cancer, led by Pérez at La Paz University Hospital, is now exploring further innovations, including adapting donor white blood cells to better serve patients whose own cells are too compromised for effective treatment. As Lucía Álvarez expressed at the press conference, she feels ecstatic to be alive and eager to help other children access these life-saving therapies, a sentiment shared by many whose lives have been positively impacted. With approximately 400,000 children and adolescents diagnosed with cancer worldwide each year, continued efforts in research and collaboration among medical professionals, public institutions, and non-profit organizations are crucial to fighting this disease and improving survival rates. As advancements in treatments like this living drug unfold, the hope for a cure for pediatric cancers grows ever closer. Related Sources: • Source 1 • Source 2