Precision medicine is the “future”. What does it consist of? How can it help in pediatric oncology? The answers are provided by Ana Fernández-Teijeiro, head of the Pediatric Onco-Hematology Section at the Virgen de la Macarena Hospital and president of the Spanish Society of Pediatric Hematology and Oncology (SEHOP)
The future of pediatric oncology lies in precision medicine
Precision medicine (MP) was born as a result of all the advances that seek better patient care. This medicine pursues the personalization of diagnosis and treatment to combat the molecular key of the tumor.
“To talk about precision medicine in pediatric oncology, what we need is to design a panel of genetic alterations that must be analyzed in pediatric tumors,” emphasizes Dr. Ana Fernández-Teijeiro.
In order to analyze the possibility of personalized treatment, clinical trials are needed.
On the one hand, there are the ‘umbrella trials’, in which different mutations are identified for the same type of cancer, which are attempted to be attacked with different drugs.
On the other hand, the ‘basket trials’, where there are many cancers and tumors that share a common genetic alteration and with which the same drug is tested to see its efficacy.
“Precision medicine seeks that after DNA analysis a suitable suit can be made for each patient”, defines the doctor; that is, it seeks to adapt the treatment to the molecular alteration of that patient, trying to attack only the tumor and reduce side effects.
To talk about it, the Association of Galician Doctors, Asomega Muller, has drawn up a series of webinars with the support of GSK and PharmaMar.
Current situation of childhood and adolescent cancer
It is calculated that cEach year around 400,000 children and adolescents between 0 and 19 years of age worldwide suffer from cancer., being one of the main causes of mortality in childhood and adolescence.
In high-income countries, more than 80% of children affected by cancer are cured, but in many low- and middle-income countries less than 30% are cured, reports the World Health Organization.
Childhood cancer is characterized by its embryonic origin and develops in the first two decades of life. Its growth is fast, aggressive and invasive and it is rarely associated with environmental carcinogens. They are more chemosensitive and radiosensitive than adult tumors, although they have a higher cure rate.
In Spain, 1,100 new cases are diagnosed each year in children under 14 years of age, leukemias being the most frequent tumors (30%). In second place are tumors of the central nervous system (20%). Finally, lymphomas, followed by neural crest tumors, renal tumors and sarcomas, in addition to others less frequently.
Pediatric oncology survival
“With data from 2021 we know that survival is 81%, of every five children we can cure four at this time,” says the doctor.
The reason for this survival refers to all the advances made in medicine that have allowed there to now be treatments for childhood cancer based on the same strategies used in adults: surgery, radiotherapy, chemotherapy and immunotherapy.
“All this has supported a fundamental support treatment such as nutritional, transfusion, diagnosis and treatment of opportunistic infections. Now all of this constitutes the approach to children and adolescents with cancer, contributing to survival”, emphasizes the specialist.
The expert emphasizes that the importance is not only to cure children but to “cure them better”. About 70% of cancer survivors will have sequelae and 40% of them will suffer severe sequelae, informs the expert.
Precision medicine aims to “reduce the use of unnecessary treatments and reduce prescription errors derived from medications that may not be useful for the patient”.
Conventional medicine vs. Personalized medicine
In traditional medicine, patients receive the same treatment despite being each in a different way. Some may benefit from the treatment while for others it may be null or produce major adverse effects.
Personalized medicine develops therapeutic targets that allow attacking the tumor in its alteration and benefit the patient with targeted therapy.
Diagnoses in precision medicine are expanded compared to traditional medicine. It allows diagnosis at the molecular, genomic or methylation (DNA cover) level, based on family history and external factors in order to define a specific treatment.
“To design molecules in precision medicine we need to analyze DNA and RNA and metabolic analyses, which can be done either in the tumor, in the metastasis or in the liquid biopsy (detection of tumor cells that may be present in the blood)”, reports Dr. Ana Fernández-Teijeiro.
This type of medicine allows radiotherapy Analyze factors that may indicate sensitivity to treatment. In this way, specialists could determine different treatment groups that are radioresistant, more radiosensitive or with a high risk of toxicity. It also allows the patient to be monitored after treatment.
Biological barriers for precision medicine applications
The design of medicines has some difficulties since “we talk about the drug having to reach the intimacy of the cell to be able to alter it”. There are a number of barriers within the patient to access it. For this reason, nanoparticles of different composition are being designed.
“It is the future and it is what the most sophisticated laboratories are working on. It is a way of bringing medicine to the intimacy of the cell”.
Why do we need to implement PM in Spain?
Currently in Spain there are five centers that participate in this consortium for the innovation of new therapies in childhood cancer: the ITCC.
It brings together the Vall d’Hebron (Barcelona), La Fe (Valencia), Niño Jesús (Madrid), San Joan de Deu (Barcelona) and La Paz (Madrid) hospitals.
“We need to improve diagnosis and treatment. Unlike other countries, in Spain we do not currently have a pediatric precision medicine program. What we have are groups with initiative that have selflessly offered to carry out these studies on patients and support their inclusion in some clinical trials”, claims the doctor.
Inside of Spanish Society of Pediatric Hematology and Oncology (SEHOP) there are some centers that have begun to develop clinical trials.
A network of hospitals
Spain has 40 pediatric cancer units, “detecting that 70% are treated in the largest units, but not all patients could benefit from these centers.”
With the help of the Ministry of Health and the Ministries of Health, an organization of pediatric cancer was launched in 2019, with an update in 2021, in such a way that at the level of the autonomous communities, networking is carried out with the most important hospitals.
“What we want is for precision medicine in Spain to become a reality. We hope that the Ministry of Health can define a genomic portfolio in pediatric cancer that is a reference for the entire national health system and makes it possible for any of the reference centers that can do so to facilitate molecular diagnosis and treatment of cancers. patients”.
In this way one of the “great challenges of pediatric oncology for the next 10 years” was born.
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