Marta G. V.
- Country
- Spain
- Department
- Pediatric Onco-Hematology
Research Overview
- Safety, Tolerability, and Feasibility of Rectal MaaT013 (Allogeneic Pooled Fecal Microbiota) in Ruxolitinib‑Refractory Pediatric and Adolescent Gastrointestinal Acute Graft‑Versus‑Host Disease
- Phase 2 Multicenter Trial of Mesenchymal Stromal Cells MC0518 vs. Best Available Therapy in Pediatric Steroid-Refractory Acute Graft-Versus-Host Disease
- Evaluation of Belumosudil and Prednisone Versus Placebo and Prednisone in Patients Aged 12 and Older with Newly Diagnosed Chronic Graft-Versus-Host Disease
Data & Insights
Has run more than one trial at 67 of 147 partner sites.
Specializations
Pediatric haematologic malignancies
Clinical research in Pediatric Onco-Hematology centered on acute lymphoblastic leukemia, acute myeloid leukemia, lymphoblastic lymphoma, chronic myeloid leukemia, and high-risk neuroblastoma, with involvement in relapsed, refractory, and newly diagnosed disease settings.
- Acute lymphoblastic leukemia
- Acute myeloid leukemia
- Classical Hodgkin lymphoma
- Neuroblastoma
- Relapsed and refractory lymphoid and myeloid cancers
Work also extends to CD22-positive and FLT3-ITD-positive leukemias, Philadelphia chromosome-positive disease, and other high-risk paediatric malignancies.
Paediatric solid tumours and brain tumours
The investigator is active in trials for solid tumours and central nervous system tumours, including diffuse intrinsic pontine glioma, diffuse midline glioma, medulloblastoma, ependymoma, atypical teratoid/rhabdoid tumour, and rhabdomyosarcoma.
- Diffuse intrinsic pontine glioma
- Diffuse midline glioma
- Medulloblastoma
- Atypical teratoid/rhabdoid tumour
- Ewing sarcoma
Research interests include molecularly defined tumours such as ALK fusion-positive, BRAF-altered, and H3 K27M-mutant cancers.
Immune-mediated and inflammatory diseases
Clinical activity includes paediatric studies in atopic dermatitis, asthma, food allergy, psoriasis, systemic lupus erythematosus, juvenile idiopathic arthritis, uveitis, and inflammatory bowel disease.
- Atopic dermatitis
- Severe asthma
- Food allergy
- Ulcerative colitis
- Crohn's disease
Additional involvement covers systemic juvenile idiopathic arthritis, vitiligo, and chronic graft-versus-host disease.
Rare genetic and metabolic disorders
The investigator also contributes to trials in rare inherited diseases, including Fanconi anemia, pyruvate kinase deficiency, mucopolysaccharidosis type II, osteogenesis imperfecta, cystic fibrosis, and tuberous sclerosis complex.
- Fanconi anemia
- Pyruvate kinase deficiency
- Mucopolysaccharidosis type II
- Osteogenesis imperfecta
- Tuberous sclerosis complex-related epilepsy
Research extends to neurodevelopmental and epileptic encephalopathies, Rett syndrome, and metabolic storage disorders.
Cell and gene therapy
Research involvement includes CAR T-cell and gene therapy programmes across paediatric oncology and inherited haematologic conditions, with attention to long-term outcomes after cellular and genetic interventions.
- CAR T-cell therapy
- Gene-modified T cells
- Lentiviral vector-based gene therapy
- Hematopoietic stem cell gene correction
- Long-term follow-up of advanced cellular therapies
This area also includes autologous cell-based treatments and precision immuno-oncology for children and young adults with refractory disease.
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