A living medicine that fits in a spoon saves the lives of eight young people with the most common childhood cancer | Health and well-being

A new living medicine, composed of cells that could fit on a spoon and manufactured in a public hospital in Madrid, has so far saved the lives of eight young people with a very aggressive form of the most common childhood cancer, type B acute lymphoblastic leukemia. The patients, all under 24 years of age, were hopeless, after multiple relapses and the chain failure of all the usual therapies. The sophisticated experimental treatment involves extracting a sample of defensive cells, redesigning them in the laboratory to multiply their ability to destroy cancer cells, and reintroducing them into the body to kill the tumor. The leader of the research, pediatrician Antonio Pérez, presented the results this Thursday at the La Paz University Hospital: 70% survival after more than a year and a half of follow-up.

A happy patient, Lucía Álvarez, 15, from Cádiz, accompanied doctors, politicians and financiers at the event. His name is the latest to be added to a hopeful list of young people who are leading this revolution in medicine. First there was Emily Whitehead, a 6-year-old American girl with apparently lethal leukemia who, in desperation, became the first child patient treated with this experimental therapy, called CAR-T, in 2012. It worked perfectly and her doctors now consider her cured.

Thousands of people have survived since then thanks to their own modified cells. The father of the treatment, the American immunologist Carl June, speaks of resurrections “like that of Lazarus”, the biblical character miraculously revived by Jesus Christ. However, the results are still insufficient. CAR-T treatments don’t always work. They only manage to save half of the children with very aggressive tumors and no alternative.

Lucía Álvarez and another dozen patients received the new therapy when they were an average of 12 years old. It is a very latest generation CAR-T. Usually, the treatment consists of installing a type of radar in the patients’ white blood cells: a synthetic molecule capable of detecting the CD19 protein present in the cancer cells of leukemias and lymphomas. The problem is that the tumor often manages to camouflage itself. The new therapy is, in the words of Antonio Pérez, like “a tandem”, capable of grabbing tumor cells at two points, CD19 and CD22, minimizing the risk of leakage.

Thanks to this sophisticated strategy conceived in La Paz, eight of the 11 patients saw their cancer disappear and become undetectable in just one month. “The treatment lasts a short time, it is a bridging treatment and it must be consolidated with a bone marrow transplant,” Pérez explained. Five of the young people were able to receive this life-saving transplant. After 20 months of follow-up, eight are still alive, which represents a survival of more than 70% in patients who had no option to continue living. The results, which must be evaluated with caution pending more trials, were published in August in the specialized journal eBioMedicine.

“I am very happy to be here,” said the fifteen-year-old, with words with a double meaning that received a standing ovation at the press conference. The case of Lucía Álvarez shows the suffering of these patients and their families. She was diagnosed with type B acute lymphoblastic leukemia when she was 17 months old. Chemotherapy worked very well for him, but when he was 7 years old he had his first relapse, which was contained with more chemo. In the midst of the Covid pandemic, he had a second relapse, with the tumor spreading through his nervous system. A first conventional CAR-T therapy and a bone marrow transplant achieved remission, but everything failed in a chain until, with no options, he received the tandem CAR-T experimentally and a second transplant. Now he leads a normal life, he has been able to return to school and dreams of studying Biology.

There are only eight CAR-T products authorized in the European Union: seven business and one academic, ARI-0001, a treatment developed at the Hospital Clínic of Barcelona that takes its name from Ariana Benedé, an 18-year-old teenager who helped promote research before dying in 2016 from acute lymphoblastic leukemia. The examples of Madrid and Barcelona show that these revolutionary therapies can be developed in public hospitals, for much less money. The Swiss pharmaceutical company Novartis set a price of more than 300,000 euros per patient for its Kymriah treatment, a CAR-T against leukemias and lymphomas. At the same time, Barcelona therapy cost only 90,000 euros.

Pediatrician Antonio Pérez directs the CRIS Unit for Advanced Therapies for Childhood Cancer, located on the eighth floor of the iconic polygonal tower in La Paz, where more than 700,000 Spaniards have been born. However, the researcher has spent the last year working and learning at London’s Great Ormond Street Children’s Hospital, financed with the copyrights of the character Peter Pan since the Scottish novelist James Matthew Barrie donated them in 1929. There Pérez met one of the most legendary patients of the British institution: Alyssa, a teenager with leukemia. incurable that in 2022 she was the first patient to receive cells from another person, but modified with a kind of genetic pencil to avoid rejection.

Pérez’s team is also now working with that genetic pencil, the so-called DNA base editors, to adapt white blood cells from donors, an excellent option when patients have their own cells very deteriorated or there is no time to waste. The CRIS Unit of La Paz does not receive funding for the rights to Peter Pan, but it does receive funding from the CRIS Foundation Against Cancer, a non-profit entity that has contributed more than 10 million euros. In February 2024, the foundation announced the case of another of the 11 patients, Mathías, a 7-year-old boy treated with tandem CAR-T therapy against his aggressive leukemia, which had caused him to spend half his life hospitalized. In less than a month, his tumor went into complete remission.

About 400,000 children and adolescents have cancer each year in the world, according to the World Health Organization. They are very rare diseases—there are barely 1,500 cases annually in Spain—and their survival rate exceeds 80% in rich countries, but that is precisely why Antonio Pérez insists in every act that public hospitals have to take the reins of research into new treatments, given the lack of economic interest for the pharmaceutical industry. There are still two out of ten children whom no one is able to cure. “I am very happy to be able to be here helping so that other children can receive treatment,” Lucía Álvarez continued.