World First: First Phase 3 Trial of In Vivo CRISPR Therapy Successfully Completed - CRISPR treatment comes one step closer to reality Researchers from Amsterdam UMC, in collaboration with other hospitals, have successfully completed the first-ever Phase 3 study of an in vivo CRISPR therapy. In this large-scale, double-blind Phase 3 trial, 80 patients with hereditary angioedema were randomized to receive either the CRISPR therapy or a placebo. CRISPR therapy is a medical technique that allows doctors to precisely modify errors in cellular DNA to treat specific hereditary diseases. Danny Cohn, leader of the research, is highly enthusiastic: "The study demonstrates that the therapy is genuinely effective and safe. This confirmation is exactly what regulatory authorities need to approve the very first in vivo CRISPR gene editing treatment for the market."
Amsterdam UMC gives future generations a seat in Parliament "We must create policy and take action today as if the lives of future generations depend on it. Because they do," says Tessa Roseboom. As the Future Generations Commissioner at Amsterdam UMC, her mission is to make those lives and the interests of future generations audible, visible, and tangible. Today, she symbolically grants them a seat in the House of Representatives’ Committee on Health, Welfare and Sport (VWS) by giving each member a chair and a powerful manifesto.
Women with menstrual disorders to receive earlier treatment thanks to major FEMCURE project One in three women experiences menstrual problems due to conditions such as endometriosis, fibroids, or scarring in the uterus. Nevertheless, it often takes 8 to 11 years before they receive a clear diagnosis, and targeted treatments are lacking. The FEMCURE project, led by Professor of Gynaecology Judith Huirne, aims to drastically shorten the time to effective treatment. Researchers will develop self-tests that allow women to assess their symptoms at home. In addition, methods will be devised to help general practitioners and gynaecologists recognize these conditions more quickly. The researchers are also developing targeted therapies, ensuring that each woman receives the treatment most effective for her.
Temporary benefit for immune system in early HIV treatment, but dysregulation returns Despite effective HIV medication, the immune system of people with HIV remains disrupted in the long term. Researchers at Amsterdam UMC investigated whether this dysregulation can be prevented by starting HIV medication immediately after infection – i.e. within a few days. They saw that six months after this early treatment, the immune system did indeed work as in people without HIV. But in the longer term, the immune system was disrupted again. This is shown by research by Amsterdam UMC, published in the journal EBioMedicine.
Transgender women do not have an increased risk of heart attack and stroke Contrary to previous research, transgender women who use the hormone oestradiol for their transition do not have an increased risk of heart attack and stroke compared to men in the general Dutch population. This is shown by a large-scale study from Amsterdam UMC, with data from more than 4,000 transgender individuals, published today in the European Heart Journal. The researchers suspect that the hormone treatment transgender women receive has a protective effect on the heart and blood vessels.
Hunting for the Crohn's Holy Grail Crohn's disease turns the lives of young patients upside down. Work, relationships, children: often all three need to put on hold. Jean-Frédéric Colombel's mission is to give these patients their lives back. Through medication, operations, prevention, but preferably by finding the ultimate cause and cure. He will give an update about his search for this holy grail during the Anatomy Lesson, on 20 November in the Concertgebouw.
Addition of Progesterone Leads to Increased Breast Growth for Transgender Women The addition of the hormone progesterone to gender-affirming hormone therapy leads to increased breast growth for transgender people following feminising hormone therapy. This is demonstrated by an Amsterdam UMC-led trial among 90 participants and these results are presented today at the European Professional Association for Transgender Health (EPATH) annual congress in Hamburg.
Concerns about independence of US guidelines for treatment of childhood obesity The American Academy of Pediatrics (AAP) is not transparent about the creation of its guideline for childhood obesity medication. The scientific substantiation appears to be limited, and the AAP appears to have financial ties with pharmaceutical companies that produce this medication, which were not mentioned in the guidelines. This is the conclusion of researchers from Amsterdam UMC, VU Amsterdam, the University of California and the University of Colorado, which is published today.
International research into local healthcare solutions in Kenya, Ghana and Uganda Building primary care for the chronically ill together with local stakeholders, so that this care fits seamlessly with the environment. That is the idea behind M-CARE, a five-year international research project in Kenya, Ghana and Uganda that starts today, thanks to a Horizon Grant worth four million euros. Project leader Charles Agyemang: "Healthcare providers, patients, administrators: we want to hear all those voices, so that we know exactly what is going on."
Video consultations are faster, cheaper and more sustainable for patients A video consultation between patient and surgeon is just as good as a physical visit to the outpatient clinic to discuss a major operation. This is evident from research by Amsterdam UMC, demonstrated that a video consultation is just as good as a physical consultation in terms of information uptake. Alongside this finding, patients save an average of two hours of travel time and waiting time per consultation, incur no costs for travel and parking, and reduce their ecological footprint. The CO₂ emissions of a video consultation are 99 percent lower than those of a physical hospital visit. The results were published today in The Lancet Digital Health.
