The advancement of so-called precision medicine, which means a new model for medicine as a whole, is unstoppable. A good example of this is the growing number of new therapies that are being researched to offer treatment options otherwise unknown until now. According to the latest data from PhRMA, the association of pharmaceutical research and manufacturers in the United States, there are currently 289 gene and cell therapies in the R&D phase for the treatment of a hundred pathologies. More than a third of these new therapies (111) are aimed at treating different types of cancer.

So far, efforts in R&D in the field of gene and cell therapy have culminated in several effective treatments, such as two immunotherapies based on anti-CD19 CAR-T cells to be used against acute lymphoblastic leukaemia and cell B-cell lymphoma, a gene therapy to treat Leber’s amaurosis (congenital disease of the retina), a vaccine against prostate cancer (also based on immunotherapy) and an RNA treatment of intervention against cardiac amyloidosis by transthyretin. The first three treatments have also been approved in Europe.

The existence of this type of advancement is the product of innumerable investigations since, half a century ago, the possibility of directly altering human genes was discovered. Today, the emergence of so-called precision medicine marks the beginning of a new era in the approach to diseases, which has its main players in the development of gene and cell therapies.

In both cases it is a matter of modifying the human genetic material to fight different diseases, but while cell therapy involves the infusion or reintroduction of whole cells in the organism, gene therapy is based on the modification or introduction of previously altered genes through different carriers (mainly viruses). Of course, both techniques can be combined, as in the case of therapies based on CAR-T cells, where cells are extracted from the body, genetically modified outside the body (ex vivo) and reintroduced into the patient for their treatment.

Included in these procedures are others involved in the field of regenerative medicine, such as tissue engineering and the development of biomaterials, which form a completely new context that lays the foundations for medicines of the future.

With regard to new gene and cell therapies in the R&D phase, they aim to provide innovative treatments for a broad group of diseases. In this way, while it is true that different types of cancer are the target of more than a third of these therapies, other diseases have also been the aim of the researchers’ efforts. It deals with ocular pathologies, where there are 28 therapies in development, cardiovascular (24), neurological (22), blood (21), genetic (15) or infectious (13), among others.

The success of high-risk R&D

All these advances would never have been possible without the commitment to a research model led by the pharmaceutical industry, one that is constantly evolving and becoming increasingly open, collaborative and international. In the case of Spain, for example, half of the funds dedicated to R&D by pharmaceutical companies (which exceed 1,100 million euros per year) are invested in projects in collaboration with hospitals and public and private research centres.

In any case, the process of R&D of a new drug is very long (10-12 years on average) and full of obstacles. It represents a huge investment (about 2,400 million euros) and also a very risky business, since only one in ten thousand potential drugs in research will finally reach the patient.

In fact, the great challenge is ensuring that these investigations in new gene and cell therapies become authorised treatments, given the complexity of management they have and the demands of evaluation and manufacturing. Assuming the risks involved in this type of development is part of the raison d’être and commitment of the pharmaceutical industry.