Forgotten tumors Targeted therapies against forgotten cancers
Oncology Why childhood cancer is the great forgotten of the pharmaceutical industry
Little Sofía turned two years old on August 21 and she did so with all her happiness and health, after having spent half her life with hardly any muscle strength to crawl, without big smiles and with a nasogastric tube to eat. According to his father, Raimundo Hurtado, shortly after his first birthday, "he was diagnosed with a very rare type of leukemia, with one of the worst prognoses and one that is less investigated." If it weren't for the work of the Fundación CRIS Contra el Cáncer, "Sofía would not have succeeded."
The reason for this 'sentence':
Juvenile myelomonocytic leukemia
. In the words of Antonio Pérez, director of the CRIS Unit for Research and Advanced Therapies in Childhood Cancer at Hospital La Paz, "there are no more than 30 cases in Spain and a five-year survival rate is below 50%. a very rare disease due to its prevalence and a very high mortality rate ".
The bad news came as a result of the symptoms of Covid-19 in Sofia's mother. Both parents had tested positive and, while waiting at home, they observed "hoarse breathing" in their little girl. After an analysis, the doctors who treated her in Córdoba detected certain anomalies for which they began to perform a battery of tests with which they finally detected a genetic mutation responsible for her rare leukemia. An immediate bone marrow transplant had to be performed.
However,
hopes were minimal
: "We cried Sofia's death every day", so the family began looking for other options until they found a research project of the CRIS Foundation against cancer, in Madrid. They contacted the doctor who was taking him, Antonio Pérez. "The mutation of this type of leukemia conditions a very bad prognosis, with many relapses, and that is why we started the investigation", explains the doctor. "We have seen that these patients benefit from a treatment based on a demethylating drug so that the disease is controlled before transplantation. These drugs are not authorized in children (less than 20% of those we use in children with cancer are authorized)" .
With this eight-session treatment, they
managed to stabilize Sofía and then transplant her with a non-identical donor
and here is another of the keys to her current state of health. "The less identical the donor, the less likely there is to relapse (although the transplant is more difficult)." This was the choice for the little girl and after the intervention, "we did cell therapy procedures, that is, we started putting memory lymphocytes against her leukemia and later, we put the demethylating treatment back on her."
For Raimundo and his wife, the result has been "incredible."
As the specialist explains, "we have been in treatment for seven months, with a very good clinical situation and, although it is too early to talk about a cure, the scenario
has gone from practically imminent death to having a normal life
."
Since May 5 "we are at home. The girl is phenomenal. She is the girl she was before. She laughs again, crawls, eats normally, plays ... She has begun to speak and also to give her first steps. He gets up and falls asleep with energy. Incredible. "
They only have to return to Madrid once a month, to the University Hospital of La Paz, to perform periodic infusions of CAR-T cells.
Research, the key
Sofia's "is a clear example of how research impacts rare diseases with high mortality," says Pérez. An idea that underlines coinciding that today is the International Day of Research against Cancer. "Research is still a luxury, a situation that society still does not value adequately, despite the pandemic. Independent research, academic, in universities, hospitals ... is done with very little support, with very little funds, with scholarship salaries, without job stability, with great voluntariness always [...]
It is necessary to have a plan that goes beyond what private organizations such
as the CRIS Against Cancer Foundation can do ".
There are more frequent types of cancer where progress is being made with pharmaceutical research, but in others, such as Sofia's leukemia, which affects only around 30 children in Spain, "we continue as we have been for more than 20 years and there is no interest in pharmaceutical companies ". For these cases, it
would be convenient to put three, four or five centers with twice the resources
, to make a research plan exclusively in pediatric cancer and with the purpose of curing this disease in the next decade ".
With rare diseases with high mortality, the strategy should be to "focus attention and knowledge in a few centers to have more experience, to be able to do more research, move faster and improve the prognosis."
It should be remembered that cancer in children cannot be prevented.
There is no preventive strategy
.
In adults, yes.
Exposure to tobacco, alcohol or sedentary lifestyle plays a role. "Somehow," the administration should make a powerful research plan. "
Along the same lines, Raimundo recognizes and appreciates the work of the Foundation and the doctors who have cared for Sofía because "if it weren't for them, our daughter would not have gotten ahead. They gave us life."
Cancer with low survivals
Sofia's type of leukemia is not the only cancer whose survival is well below average. That of tumors of the pancreas, lung, esophagus and liver does not reach 20%. For this reason, cancer professionals and researchers demand a national, public and private investment.
Ramón Reyes, president of the AECC and of the AECC Scientific Foundation, assures that "it
is necessary to correct the inequity in research
that happens, among other factors, by investigating all tumors and because everyone has access to research results There are more than 100,000 patients that each year develop cancers with low or stagnant survivals who have less chance of survival than other patients whose tumors are more investigated. "
There is evidence that, in general, those cancers with better survival data also have more research.
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