At the avant-garde
of translational research
New treatments derived from human cells cultured in the lab have been developed for medical use. These cells can be modified through genetic techniques (virus therapy, gene therapy). Both types of cells, genetically modified and non-modified cells, are considered a novel group of medications called “Advanced Therapy Medicinal Products” (ATMPs).
The Spanish Medicines Agency (AEMPS) defines ATMPs as medications based on genes (gene therapy), cells (cell therapy) or tissue (tissue engineering). The group includes autologous, allogeneic and xenogeneic products.
Altogether, ATMPs are being incorporated into new treatment strategies. Their further development will help create treatment opportunities for patients with diseases that have currently no cure. These advanced therapies have already shown efficacy in treating several diseases in adult patients.
Dr. Luis Madero and his team are leaders in the field of advanced therapies through the translational research carried out at the Pediatric Hematology and Oncology Department. A summary of the clinical work on advanced therapies performed at Hospital “Niño Jesús” over the last years is presented below:
HEMATOLOGY AND ONCOLOGY
Over the last ten years, our research group has been developing a medication called Celyvir for the treatment of relapsed and metastatic pediatric solid tumors. This medication is based on an oncolytic adenovirus that is transduced into mesenchymal stem cells extracted from the patient’s bone marrow. Our initial clinical experience relied on a program of compassionate use for children with relapsed tumors. Following this, a clinical trial was carried out (EudraCT2008-000364-16; ClinicalTrials ID: NCT01844661).
The first project in this area at Hospital “Niño Jesús” was a program of compassionate use for the treatment of Perthes disease with poor prognosis. The treatment consisted of infusing autologous bone marrow stem cells; the achieved results were not satisfactory. Following this, autologous bone marrow MSCs were applied to leukemia patients that had developed avascular necrosis of the femoral head as a result of dexamethasone treatment. The obtained, very positive results have been recently published. We believe that this therapy can be useful in the management of avascular necrosis and may substitute or delay joint replacement.
The immunomodulatory effects of MSCs are being investigated for the treatment of children with Rasmussen’s syndrome. This disease is a childhood degenerative encephalopathy, with refractory epilepsy that frequently requires surgical treatment. Being our hospital a reference center for epilepsy surgery, we have easy access to this group of patients.
We have recently started designing a clinical trial for children with cerebral palsy, that will use allogeneic umbilical cord mesenchymal stem cells.
Beyond this, Dr. Madero and his team have extended their experience on the use of advanced therapies to other diseases in adult patients:
- Joint arthrosis: About 20 patients with grade III and IV arthrosis in the knee or hip have been treated so far. Before this treatment, the only option for these patients would have been joint replacement. The results have been positive in 90% of the patients, with pain reduction and function improvement. After the treatment, none of the patients required joint replacement.
Lastly, it is worth mentioning that the field of advanced therapies is expanding. New applications for these treatments are soon to be expected; in fact, there are over 5,000 ongoing clinical trials investigating advanced therapies (https://clinicaltrials.gov Searched on 11/06/2017).