Cell Therapy: living drugs are here

The use of stem cells to treat diseases creates hope for patients with certain diseases that until now were considered incurable.

Investigadora en el laboratorio

To tell this story we must go back to 1998, to the beginning of the acquisition of stem cells from the human blastocyst. Those of embryonic origin, capable of regenerating any type of tissue, were the first to be isolated. Two years later, some researchers found these cells in bone marrow and called them adult stem cells. In 2006, Japan’s Shinya Yamanaka, who won the Nobel Prize in Medicine in 2012, described how embryonic stem cells could be made from any cell.

Stem cell research is leading to the creation of the first drugs with living components.

The head of the Department of Surgery of the University Hospital of the Jiménez Díaz Foundation, Damián García Olmo recalls, Yamanaka discovered that these cells can have therapeutic effects on their own. These are mesenchymal cells, the aim of which is to control inflammation and facilitate tissue regeneration. "They are currently very efficient from a clinical point of view, because the basis of many human diseases is inflammation,” explains García Olmo. Thus, its good control presupposes the cure of ailments that until now had no treatment or whose management produced many side effects. This scenario arises the possibility of curing with cells, that is cell therapy, and its translation in the clinical application is what is called living medicine. “These cells are used to cure, mainly for signalling purposes," says García Olmo. Until now, humans have been cured with inert substances".

In his opinion, cell therapy contemplates several forms: therapy that uses wild genotype, that is, raw cells, without genetic changes or alterations -mesenchymal cells-, and on the other hand, genetic engineering, that is, the genetic modification of cells in our immune system to fight disease, in this case by means of CAR-T cells.

How do patients get into these therapies? So far, the world’s only live cell-based drug approved by European authorities is the result of a Spanish study for the treatment of Crohn’s fistula led by García Olmo. A few months ago, the Ministry of Health welcomed darvadstrocel, an allogeneic mesenchymal stem cell-based therapy in the development of which the Autonomous University of Madrid (UAM) participated through the development of a patent co-ownership of Tigenix.

Stem cells are obtained from liposuctions of fatty tissue from healthy donors, then they are expanded in the laboratory, injected into the patient’s perianal area, and the cells are responsible for closing the fistula without the need, in many cases, to apply immunosuppressive drugs, which had been the usual technique until now.

This treatment is a new option for patients who do not respond to current therapies and who may have undergone numerous invasive surgical procedures. The development process, as the surgeon recalls, lasted fourteen years, until obtaining approval for clinical use.

The surgeon values the importance of networking: "There is no doubt that clinical-private collaboration is essential in translational research". In his view, this is a model of success. Fidel Rodríguez Batalla, director of the Foundation of the Autonomous University of Madrid, agrees with this, explaining that, beyond the technical part, this research is a clear example of success. "In the end, patients are being treated who are improving their quality of life and giving them a solution to their problem," he says.

Continue reading