“With HIV virus we can cure rare diseases”

Alessandra Biffi heads the Pediatric Onchematic Clinic in Padua, which is at the forefront of treating genetic pathologies: “Today, more frequent leukemias, in less severe forms, make 9 out of 10 children healthy, but that’s the way to bring them home. “.

“The other morning we cried with parents we have known for a long time: we had to say there was nothing more to do for their baby. These children often stay here with us for years. They become our children, they were my children, even before my Guido was born. ”Alessandra Biffi, 49, from Milan, runs the clinic in pediatric onchematology in Padua and teaches pediatrics at Bo ‘. Now that Covid has loosened the grip, the children have occupied the large space where they meet their parents and play. M is seven years old, she has lost all her hair, her eyes are blue like the color of a hospital wall. He made a paper plane that takes a short flight before falling. EI, 12, also hairless, clings to her young mother. He picks up a rubber dinosaur and moves it as in a scene from Jurassic Park: he pretends to be scared, then his mouth gets filled with laughter. Doctor Biffi looks behind the window. “This is a very special place. Here we deal with diagnosing and treating children who have blood diseases, including cancer. All rare diseases. Every day we have 60 patients and a ward that is always filled with 20 hospital beds and 6 beds. she arrived in Padua after an international career as a researcher and an experience as director of the Boston Department of Gene Therapy with a chair at Harvard. In his field he is an authority, he also has a place in Treccani’s book of the year. developed a method and drug for the treatment of a rare childhood disease, Leukodystrophiametachromatic (BN): healthy genes are introduced into the stem cells of patients through the modified HIV virus, which is used as a kind of ram before it is re-injected into the modified strain. sick.

Does the method work, can it also be extended to other diseases?
“Yes, an oar has begun. My research explains how to use these cells with gene therapy, for example also for thalassemia. With a modified approach, it is also valid for the treatment of other cancers: For acute leukemias in children, there are already drugs on the market that are also useful for adults for leukemia and lymphomas. I was the first in Italy who, with a certain feeling, infused a child with a substance of this type. The new challenge now is to do it with new diseases, such as diabetes and multiple sclerosis ».

Was she a child who wanted to be a geneticist when she grew up?
“I lost my father when I was three and a half years old, I grew up with a mother who rolled up her sleeves and grandparents who are very close. Then my mother found a partner who followed me step by step. I had started in Engineering, I was bored, I was scared, so I said to my mother: I want to be happy in the morning to go to work, I change faculty. And I switched to medicine, I wanted to do genetics. Milan, near San Raffaele, had Professor Claudio Bordignon, Professor of Hematology, a pioneer in bone marrow transplantation between adults, founded a unique institute in Europe in collaboration with Telethon for the development of gene therapy. talk about leukemia, that was my topic: he invited me to discuss the thesis with him.From the moment I started dealing with gene therapy, I worked in the department for twenty years, until 2015. Bordignon and, after him, Maria Grazia Roncarolo and Luig in Naldini, they taught me how to go this way “.

And research based on the HIV virus?
“Naldini was the first to develop a type of tool for HIV-based therapeutic gene transfer. Viruses rendered harmless to bring a piece of DNA into the cells and make the cell regain function. If the patient has a genetic disease, that is, that there is a missing gene, we put back the lack of function to cure his disease.We started with the virus, which at the time was so scary, because combined with AIDS it was about bringing the healthy gene into the cells. collected all data for three years and found reagents to treat patients.In 2010 we started treating this rare disease, Mld, which is terrible: children are born normal, but after years the signs start to show, they do not keep their heads up, they stop walking, but if an early diagnosis can be made, if it is treated at the onset of the very first symptoms, even at the age of one year, because the little ones start to go back, then they can have an almost normal life thanks to be this medicine ddel, which has a liberating name: Lib- Mld, free us from disease. The first child treated came from Lebanon: today he is in Italy, he speaks three languages, he is well. His two older brothers died of the same disease when they were five.

You have been to America and led a major research program.
“After working for twenty years in Milan, I received a job offer from the United States in 2015: I thought about it for a year, my husband, a neuroradiologist, convinced me. His name is Letterio, with that name I call him by his last name: Police I went to Boston as an associate professor at Harvard and under the direction of the Gene Therapy program. It was a challenge. In Milan I had always dealt with my niche, I also felt protected, now it was a matter of taking over an already advanced program and “I learned to work in a different way, I gained new scientific and also managerial skills.”

And how did it arrive in Padua?
“In 2018, I heard that Professor Beppe Basso wanted to talk to me, and we met at a restaurant run by Roncarolo, who now teaches at Stanford. He had to retire and would offer me to come to Padua to continue his work. Already in the autumn I was in Padua with my son Guido, who went to second grade.The change was radical, until that moment I had always been concerned with clinical studies and pediatric transplantation, but always with the clinical eye of the researcher. to do much more ».

Why is Padua a special place?
“This is a very special place, we deal with diagnosing and treating children who have blood diseases, all rare diseases, but never rare enough. We are connected to the Tower of the City of Hope, which is a huge reality. We focus primarily on advanced diagnostics to make an accurate map of the patient to target his tumor, not that tumor in general.Then the search to identify a therapy.Samples from all over Italy arrive every day at our diagnostic laboratory, 6000 per year.Padua is in front, here in forty years more than 600 transplants have been performed in children.In the Research Tower there are 55 young researchers and unfortunately many of them are precarious, but they have a great will and a unique enthusiasm, my task is also to find resources to support their salaries.Our research has just received a prestigious award from the European Community, which gives us five years of respite ».

Is it harder to heal a child?
“Diseases in children are always difficult to accept. Cancers in children are on the rise, in the Western world they are among the causes of death in the pediatric age. Leukemias are more frequent and up to 30 years ago very few heal, today nine out of ten heal “in the less severe forms. It is also difficult to get them home after so many months in the hospital. There is a long way to go to get the child back to being a baby.”

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