Center for Applied Medical Research (CIMA)
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Researcher in the Gene Therapy and Regulation of Gene Expression Programme and Director of Innovation and Transfer at Cima University of Navarra
Professor of Immunology at the University of Navarra, CIMA researcher and co-director of the Department of Immunology and Immunotherapy at the Clínica Universidad de Navarra.
Senior Researcher of the Gene Therapy in Neurodegenerative Diseases Programme at the Centre for Applied Medical Research (CIMA), University of Navarra
Researcher in the Solid Tumours Programme at CIMA and the Clínica Universidad de Navarra
Researcher of the Gene Therapy and Regulation of Gene Expression Programme at Cima (Centre for Applied Medical Research) University of Navarra
CAR-T cell-based treatments have been successful against some blood tumours, but are much less effective for solid tumours. A phase 1 clinical trial has tested their use in 11 children and young adults with diffuse midline glioma, a tumour of the nervous system that is considered incurable. The results, published in the journal Nature, indicate that the treatment improved functional status in nine of the 11 patients. One of the four who showed a strong response is still healthy four years later.
The regulatory agencies for medicines in the United States and Europe have issued statements informing about a possible risk of developing certain types of tumors following CAR-T cell immunotherapy treatment. What do we know so far? What is the real risk? Does the benefit-risk balance still hold? Has anything changed after these alerts? We answer these questions with expert opinions and the data currently available.
CAR-T cell therapies may, in some cases, produce tumours secondary to treatment. A few months ago, the US Food and Drug Administration (FDA) said it was assessing this risk. Now, a study conducted at Stanford University Medical Center (USA) has tracked 724 patients who received this type of treatment since 2016. Of these, 14 developed another blood tumour, but only one was a T-cell lymphoma that could be a direct consequence of the therapy. Further analysis ruled out this link. The results are published in the journal NEJM.
A monoclonal antibody called prasinezumab reduces the worsening of motor symptoms in people with Parkinson's disease who have rapidly progressive disease, according to an analysis of a phase 2 clinical trial published in Nature Medicine. These findings suggest that clinical efficacy of prasinezumab, which works by binding to alpha-synuclein protein aggregates, is seen after one year of treatment in such patients. According to the authors, more research is needed to determine whether the antibody can be effective in people with slower disease progression after longer periods of treatment.
A phase 2 clinical trial in France has examined whether taking an oral anti-diabetic drug called lixisenatide - a GLP1 receptor analogue, similar to those also used for weight loss - also has an effect on the progression of Parkinson's disease. The results indicate that there is a modest but significant decrease in the progression of motor symptoms of the disease, although side effects were also observed. The results are published in the journal NEJM.
A multidisciplinary study involving several Spanish research groups has preclinically tested a new type of immunotherapy for multiple myeloma. Instead of modifying T cells to attack the tumour directly, as CAR-T cells do, they have managed to make them secrete bispecific antibodies, which bind to the tumour on one side and to other T cells on the other, attracting them to the tumour. According to the authors, this cell therapy was more effective than traditional CAR-Ts and could generate less resistance. The results are published in the journal Science Translational Medicine.
The US Food and Drug Administration (FDA) has issued a statement reporting that it has received reports of T-cell tumours in patients who received various CAR-T cell treatments. As quoted in the statement, "although the overall benefits of these products continue to outweigh their potential risks for their approved uses, FDA is investigating the identified risk of T cell malignancy with serious outcomes, including hospitalization and death, and is evaluating the need for regulatory action".
Research by researchers in Shanghai, China, suggests that continued practice of tai chi can slow the progression of Parkinson's disease symptoms and delay the need for increased doses of medication. The study compared 143 people who underwent tai chi training with 187 non-trainees who served as a control group. The results are published in the Journal of Neurology Neurosurgery & Psychiatry.
Parkinson's disease is usually diagnosed when there is already extensive neuronal damage and symptoms are evident. Now, researchers at Cardiff University in the UK have used movement and sleep quality data from wearable accelerometers and concluded that they can help identify the disease early, years before clinical diagnosis. Although there is no effective preventive treatment, the authors propose that the tool can determine people at risk of developing Parkinson's disease and identify participants for clinical trials of neuroprotective treatments. The results are published in the journal Nature Medicine.
A team of scientists led by the Catholic University of America in Washington has designed new artificial vectors based on viruses to improve gene therapy processes. The main novelty is that they are constructed from viruses that infect bacteria. Among other advantages, this would make it possible to avoid the possible memory of our defences against them and have a greater capacity. According to the authors, who publish their results in the journal Nature Communications, these nanoparticles "have the potential to transform gene therapies and personalised medicine".