Little Hassan, & baby butterfly & # 39; edited by award winner Lombardy is research & 39; (AdnKronos Salute photo)
Published: 11/08/2018 6:23 pm
Epidermolysis bullosa it is a disease that destroys the quality of life of affected boys. In its most severe form, 40% do not reach puberty. When we are treated, with gene therapy ex-vivo tuning for small forms of junctional epidermolysis bullous Hassan "," baby butterfly "arrived from Syria to Germany, in the special center of Bochum, "it was all new, after a few days of surgery we returned for the first time to the hospital where we were. We removed the gauze with vaselin that covered it and we saw pink, normal, dense skin had grown. That was truly the strongest emotion of my scientific career"This is a story about Michele De Luca, director and gene therapy coordinator of 'Stefano Ferrari & # 39; Center for Regenerative Medicine (CMR) from the Universities of Modena and Reggio Emilia.
Scientists who accept today at the Teatro alla Scala in Milan that Lombard Nobel, together with coworkers Graziella Pellegrini, CM cell therapy coordinator, and German surgeon Tobias Hirsch, retracing the steps of the research that has gotten the page & # 39; and who have assured "unanimously" a 15-judge jury, chaired by Giuseppe Remuzzi, who has the task of identifying the winners of the second edition Award 'Lombardy is research & # 39;. "This is a long journey, nothing comes from nothing, this continues", observes Pellegrini.
While De Luca returned to an important moment, when a research found its clinical application. In this case the recipient of therapy is a refugee child who is welcomed in Germany with his family. The disease, junctional Eb, mercilessly went berserk at Hassan. "It's always worse. And at some point he almost died. When he was 7 years old, he had lost almost all of his skin, "recalled Hirsch, who was 10 years old and" a truly normal life, "said De Luca." He was completely covered in skin. He no longer makes lesions, bubbles ", typical symptoms of the disease."See him walking and going to school, cycling and traveling with his family, it is a dream come true. We work for this, to treat patients ".
It was experienced and Hassan came back to life. This is a research lesson, said Remuzzi, who invited researchers to "not work only for impact factors". The boy looked smiling in a video on the screen installed at La Scala when he kicked the ball, "even if it's not like Ronaldo", De Luca's joke. For scientists, "the joy of having received Hassan's visit to Cmr of Modena" even surpassed the conquered telephone call received from Alam, which conveyed to him the publication of the results of experiments carried out with his team. First world, born in the sector – research – that has no limits. And he brought the German Hirsch, looking for opportunities for his small patients, to the center of Italy where De Luca and Pellegrini did their studies.
"On our way we started from afar, from very serious burns – retraces Pellegrini – We learn a lot of things. In the midst of scientific experience that leads us to corneal reconstruction and special drug registration: the first product based on a stem that is certified as a drug by the European Agency, which accepts the validity of the study and has made it available to all European patients. "After Hassan, the adventure continues: from the damage and suffering experienced by a child, we understand more about the mechanisms that govern the maintenance of human skin:" That's amazing. – said the researcher – understand that stem cells that we genetically modify remain in basically normal conditions and are capable of carrying out their ongoing renewal work. Hassan really changed his skin at least 36 times"
Hirsh, who said he was "respected" for the recognition given by the Lombardy Region, met small patients regularly. "This is a miracle for us, it will be important to see how it lasted for years, in follow-up studies." Scientists move forward with research. "Hassan is a compassionate case – observing De Luca – We now want to continue with phase I-II clinical trials on junctional forms to confirm the results obtained in 3 patients (including Hassan), and then we will immediately register someone else".
For Lombard Nobel, it is expected 70% of funds assigned "dedicated to developing activities in the region worked with the Lombardy research system"This is remembered by the Regional Vice President and the Council for Research, Innovation, Universities, Exports and Internationalization Fabrizio Sala, who spoke of "a significant response that we will have both in terms of competitiveness, but also work". And award-winning scientists confirm the commitment: "We will use it in collaboration with researchers and laboratories in Lombardy to develop together several aspects of gene therapy for epidermolysis bulosa (Eb)", explained Michele De Luca, who was awarded with his colleagues Graziella Pellegrini and Tobias Hirsch.
"We think – he continued – We might involve at least some Lombard centers, for example we have the possibility to use this loan to provide scholarships, research grants, doctoral scholarships to Lombard researchers. There are still many who will be dedicated to human resources"Patients with this disease, underlining Pellegrini," are very complex, have many problems in various districts of the body. This certainly makes us have a broad field of collaboration, because we should not just focus on pieces of skin. We have become an important step because we have replaced the largest organ of the human body, but to cure patients there are 360 degrees of intervention that must be done ".
There are several components, added De Luca, "clinics, immunology, etc. In addition we want to use money to give contracts to researchers, now everything must be built". The next step? "There are two aspects – the list – one of the basic studies, which will continue to look for ways to attack other forms of epidermolysis bullosa because they are not all the same and we have focused on current junctional forms. Then we will start new clinical trials that must trying to expand the field of action, it will take time – acknowledging De Luca – but we will work hard for this: we want to attack this disease in all its forms"
And precisely the hope expressed by this patient's family, present today at the Scala ceremony. "For us all this represents something beautiful – he told AdnKronos Salute Rosalba Loprevite, mother of Alexander, 18, & # 39; butterfly & # 39; – This is the starting point for future therapy. Hope is so great and this is one day to see how to improve the quality of life of our children. Living with bullous epidermolysis is very damaging. Every day we have to poke bubbles that form on the skin of boys, we have to bow them to long and painful clothes, making miles of bandages. "
Those who suffer, continue their mothers, "have difficulty even walking or eating, because the problem is a lot, even the esophagus, for example. We must rediscover new life. First you touch the bottom. Then from the boy himself out so much love and strength. I have an 18 year old son, and I can say that we continue with all the difficulties. "From the award-winning team & Lombardy, the research is ##, Rosalba expects that" we will soon find a way to achieve therapy. genes for all patients affected by various forms of disease. I dreamed later that these people were fully accepted. This is a clear and frightening disease. I dream of a better future for a new generation, dreaming that tomorrow's parents should not suffer as we see their children cry and suffer ".
"We have a series of ideas and previous experiences – think of Pellegrini – but to reach the goal it serves critical mass. "And they need funds.
"We feel strongly supported by the Lombardy Region at this time and thank you for taking care of this research, and we have felt supported for years by the Emilia Romagna Region", Pellegrini and De Luca reply to those who ask him if there is support currently adequate for research by national institutions.
The winner of the first edition will testify about what they transformed and what contribution they made to the funds allocated with the Lombard prize. Giacomo Rizzolatti (University of Parma), father of & # 39; mirror neuron & # 39; Scientists decided to invest them to donate to experts from the epilepsy surgery center at the Niguarda hospital in Milan, with whom he conducted research, a machine for intraoperative monitoring. "Help is valuable" for successful interventions, minimizing the risk of motor and language deficits for patients.
"25% of the surgical procedures performed at the facility have benefited from the use of this machine," Rizzolatti explained. "And then there are scientific results that are permitted to be published in magazines like Brain & # 39; and # 39; Pnas & # 39;. This award is also used to guarantee research grants to neurologists and surgeons, good for Niguarda" .
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