Clínic Hospital

Science magazine has named lenacapavir as breakthrough of the year. Lenacapavir is an injectable drug that prevents HIV infection for six months with near 100% efficacy. The publication notes its potential to dramatically reduce infections in high-risk populations, but also reminds that global roll-out will depend on affordability, manufacturing agreements and a robust health infrastructure. Approval of the drug is expected by 2025. 

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 U.S. FDA has approved a new drug—called Cobenfy—to treat the symptoms of schizophrenia. Unlike traditional treatments, which are based on blocking the effects of dopamine, its mechanism of action simulates the effects of another neurotransmitter, acetylcholine. It is the first drug to be approved with a different mechanism of action in over 70 years.

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. 

Five out of ten potential treatments move from animal studies to human studies; four to randomised controlled clinical trials; and one in 20 moves on to approval by regulatory agencies, an analysis estimates. Concordance between positive results in animals and in clinical studies is 86%, according to the study, published in PLoS Biology, which pools the findings of 122 published studies on 54 different human diseases.  

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. 

The Lancet Psychiatry publishes the first meta-analysis of the incidence of antidepressant treatment discontinuation symptoms that includes data from more than 20,000 patients collected from 79 randomised controlled trials and observational studies. The study sought to distinguish between symptoms directly caused by medication discontinuation and other ‘non-specific’ symptoms that may be associated with patient or professional expectations (the nocebo effect). The study concludes that one in six to seven patients will experience one or more symptoms directly caused by stopping medication, and one in 35 are likely to experience severe symptoms.  

The Science group is simultaneously publishing four papers (two in the journal Science, one in Science Immunology and one in Science Translational Medicine) that include advances in a sequential vaccination strategy for an effective HIV vaccine. The methods employed aim to obtain broad-spectrum neutralising antibodies and one of the proposals is already in clinical trials. 

The largest meta-analysis to date that studies the risk of children of people with a mental disorder also suffering from some type of mental disorder during their lifetime has been published, with Spanish participation. According to the study, the risk is more than double that of the rest of the population. To explain the study and resolve any doubts that may arise, the Science Media Centre Spain organised an information session with one of the authors, psychiatrist Joaquim Raduà.

An international team, led by the Hospital Clínic-IDIBAPS in Barcelona together with King's College London (UK) and Dalhousie University (Canada), has published the largest meta-analysis to date studying the risk of children of people with mental disorders developing the same, or another type of disorder. The study included more than three million children of parents with different types of disorders and more than 20 million people as a control population. The results, published in the journal World Psychiatry, indicate that, overall, the former have a 1.5 to 3 times higher risk of developing a disorder in their lifetime. According to the authors, in the case of bipolar, depressive or anxiety disorders, the risk of their offspring suffering from some type of disorder is more than 50%.