The groundbreaking potential of ATMPs in the treatment of serious diseases has been widely demonstrated by the release of several ATMP drugs. This potentiality has been very well exploited in the treatment of genetic disorders, where these innovative medicines were able to give back a normal life to several pediatric patients affected by tremendous pathologies. Unfortunately, the most important limitation to the spread of Advanced Therapy has already been found in the extremely high cost of production and manufacturing.
In a recent article, published in the Italian language on the website “Osservatorio Terapie Avanzate” this topic has been discussed with Giulio Cossu, Constance Thornley Professor of Regenerative Medicine at the University of Manchester.
The provocative question and main topic of the discussion were: can ATMPs be considered similar to a speculative bubble? Have ATMPs the reliability that really makes them a scientific and economic bet of the present (and of the near future) or if, instead, they are a sector destined to implode on itself?
A significant example is the case of Strimvelis, an innovative ATMP drug developed by the Italian institute San Raffaele. Strimvelis resulted extremely efficient in the treatment of ADA-SCID, a rare genetic disorder usually affecting patients in their childhood, causing severe immunodeficiency and short lifespan. Orchard Therapeutics, the company that is in charge of the commercialization, announced the withdrawal of Stimvelis for commercial reasons. This withdrawal came immediately after another one, the one actuated by Bluebird bio with two ATMP drugs against beta-thalassemia and adrenoleukodystrophy.
According to prof. Cossu, the risk that, globally, “companies will be discouraged from investing more work and money and the entire industry will collapse because of this stagnation” exist and must be taken into account. The institutions and the governments should take care of – at least a part – of the costs of the creation of new GMP facilities and new clinical trials. One of the possible solutions in Europe is the AGORA consortium (Access to Gene therapies fOr RAre disease), which, other than founds clinical research and facilities, will be able to “enhance the awareness of the need to not abandon patients to their fate just because the advanced therapies that could cure them cost too much”.
Another important aspect mentioned by prof. Cossu is that the collaboration for the improvement of ATMPs must be shared by all the EU nations together, and by the media, which have the unique power to sensibilize the public opinion regarding this issue.