Home » Locatelli: “The revolution of advanced therapies must be made accessible to patients”

Locatelli: “The revolution of advanced therapies must be made accessible to patients”

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Thousands of patients today without the possibility of a cure in the coming years could find an answer to their health needs. It is the promise of advanced therapies, innovative treatments that work on genes and cells, which could however remain unfulfilled. In fact, drugs of this kind manage to modify the history of serious illnesses but they are very expensive, and the Health Service is faced with both a financial and an ethical problem. Yes, because within 10 years at least 60 advanced therapy products will hit the market, tripling today’s offer. “There is no doubt that advanced therapies are revolutionizing the treatment of some diseases, both genetically determined and acquired. But we must ensure that research funding is constant, that companies make commitments to guarantee drugs once they are developed and that the Health Service makes them accessible”, explains Franco Locatelli, director of the Onco-Hematology and Cell Therapy department and Genica of the Bambino Gesù Hospital in Rome, which today spoke at the conference “Unlocking Innovation: How to prepare the health system for the challenge of Advanced Therapies”, organized by the American Embassy and Vertex Pharmaceuticals.

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Upcoming therapies

At the forefront of the therapies of the future are the so-called CarT, therapies that exploit the action of cells of the immune system which, suitably modified, are able to strike and eliminate the tumour. “Their effectiveness has already been demonstrated for various blood cancers, so much so that 6 products are already approved. In the coming years, the indications for these treatments will certainly be expanded to include other hematological conditions and also solid tumors”, explains Locatelli. “We are obtaining some very interesting data in pediatric neuroblastoma and are about to start studies in young adults affected by this and other central nervous system tumors.” Researchers are also obtaining promising results in the case of some acute leukemias and, still on the front of acquired diseases, the results obtained in the treatment of systemic lupus erythematosus should be noted. “Also in this case the determining pathogenic component is that of the B cells which therefore constitute the target of the therapy. Studies demonstrate both clinical and marker remission,” Locatelli points out. Reassuring data also comes from trials on progressive systemic sclerosis.

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On the front of genetically determined diseases, however, the technologies used make it possible to insert one or more healthy copies of defective genes or to correct the production of substances which, due to genetic alterations, are not expressed as much as they should. It is possible to cure diseases which therefore depend on the defect of a single gene, as demonstrated by the research carried out by Telethon or as in the case of spinal muscular atrophy. Or even pathologies caused by the low production of hemoglobin: Locatelli’s team has developed a genetic manipulation therapy that manages to increase it, so as to allow patients with beta thalassemia to stop transfusions in 90% of cases. An important perspective for the more than 7,000 thalassemic patients living in Italy for now studied only in young adults but which researchers will soon begin to test in children as well.

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Access to treatments

Faced with these opportunities, how should the National Health Service be organised? On the one hand, ensuring that research goes ahead – preserving Italian excellence – on the other, building a network of centers in the area that can guarantee access to these therapies. As regards the first point, Italian research can count on the funds of the Pnrr, which provide for investment lines dedicated to gene therapy in tumors and hereditary diseases, and then on the complementary funds made available by the Ministry of Health for the among other things, of the Life Science Hub operating in the Advanced Therapies sector (LSH-TA), whose objective is to create a network of technological and service platforms. “But then it will be necessary to identify the centers that can apply the gene therapies that will be validated and approved by the regulatory agencies, as was done for the Carts”, says Locatelli. “Centres that meet criteria of quality and excellence, but that are also distributed throughout the territory”.

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Sustainability also represents a further challenge on which the institutions will have to work. And here the analyzes and studies of pharmacoeconomics and the virtuous relationship with the pharmaceutical companies, which develop the therapies, come in handy. So that cases such as those of Bluebirdbio, a company that involved European research centres, primarily Italian ones, in the development of a gene therapy which it later decided not to make available in the Old Continent do not repeat themselves. Involving private individuals as well and imagining access as well as development strategies with them is the objective of the National Center for the development of gene therapy and drugs with RNA technology, set up with funds from the Pnrr. But it is clear that different public finance mechanisms will also have to intervene, with the implementation of innovative repayment models. Considering spending on advanced therapies as an investment rather than just a cost. Only in this way will it be possible to maintain the universality of the Health Service and at the same time its sustainability.

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