Gene therapy real drug will now begin to translate beyond clinical trials

Gene therapy has received a new boost this year with the approval of Kymriah from Novartis by the United States regulatory agency, the FDA. The treatment is indicated for acute lymphoblastic leukemia in children and young people (see HERE). It consists of the infusion of chimeric T lymphocytes (T CAR), extracted from the patient him – or herself and modified to express antigen receptors against the tumor cells. “The FDA’s decision is so meaningful because it shows that the perception towards these kinds of medicines is finally catching up to the science. It is not just a research, these are real drugs that will now begin to translate beyond clinical trials“, said Alessandra Biffi, director of the Dana Farber/Boston Children’s Gene Therapy program.

Some other advances in approval of different gene therapy treatments

This groundbreaking approval comes after the European Medicines Agency gave the green light to GSK’s Strimvelis, a therapy based on autologous CD34+ cells transduced to express adenosine deaminase (ADA) to treat severe combined immunodeficiency due to ADA deficiency (an inherited disorder that damages the immune system and causes severe combined immunodeficiency), and it joins other recent advances that confirm the healthy state of genetic treatments. Notable examples include reconstruction of the skin of child with junctional epidermolysis bullosa by Italian scientists at the University of Modena using the patient’s keratocytes, genetically modified and grown in the laboratory, and two recent studies published in the NEJM: one conducted in children with adrenoleukodystrophy, also using genetically modified autologous cells, halted disease progression, while another carried out on spinal muscular atrophy type 1 (which also shows the efficacy of a new type of viral vector) has prolonged survival and quality of life in children with this fatal disease.

Indeed, that new vector, adeno-associated virus serotype 9 (AAV9), which has the ability to cross the blood-brain barrier, is being used in a trial in patients with Sanfilippo A syndrome in the United States and Australia, with the participation of Hospital Clínico de Santiago de Compostela in Spain as the only European site. To all this, we must add the remission achieved with gene therapy in hemophilia B, and the promising results for type A, which have just been announced at the American Society of Hematology conference, to cite some other studies.

Our statement

We all know the difficulties that gene therapy has had for clinical use, such that a moratorium on its use was even requested. However, it subsequently began to be used in clinical medicine, which is corroborated in the news discussed here.

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