pharmacology

The European Medicines Agency (EMA) has recommended granting marketing authorisation in the European Union for a new therapy for the treatment of adult patients with amyotrophic lateral sclerosis (ALS), a rare and frequently fatal disease that causes muscle weakness and leads to paralysis. Qalsody (tofersen) is indicated for the treatment of adults with ALS who have a mutation in the SOD1 gene. There is currently only one treatment for ALS authorised in the EU (riluzole).

For the treatment of many diseases it is necessary to use large molecules that do not resist the digestion process and must be injected. Now, a group of scientists at the Institute of Chemical Sciences and Engineering in Lausanne (Switzerland) has developed a method for synthesizing a type of protein called cyclic peptides, capable of binding to varied and complex targets, and they have done so in a way that makes them capable of being administered orally. According to the press release accompanying the publication, this opens "a new era in drug development".

A study that has analysed more than three million pregnancies with more than 130,000 miscarriages has concluded that the use of benzodiazepines during pregnancy is associated with an increased risk of miscarriage. The results are published in the journal JAMA Psychiatry.

Biosimilars work in a similar way to biologics - which are made from living cells and are very complex and expensive - such as insulin, growth hormone, vaccines and monoclonal cancer antibodies. We explain what savings biosimilars bring to the healthcare system and what the process is to ensure that their quality, efficacy and safety are equivalent to those of the reference medicine.

A standard chemotherapy drug injures surrounding noncancerous cells, which can awaken dormant cancer cells and promote cancer growth, according to a study published today in the journal PLOS Biology.

The drug lecanemab (Leqembi™) has received full approval as a treatment for early Alzheimer's disease by the US Food and Drug Administration (FDA), after confirming its clinical benefit in a trial involving 1,795 patients. The FDA highlights the warning that in rare cases it can cause serious and potentially life-threatening side effects.

Research led by scientists at Harvard Medical School (USA) has isolated small aggregates of amyloid from the brains of post-mortem Alzheimer's patients. The achievement has made it possible to study the structure of these "clumps", which exist outside plaques and are considered highly toxic, and to test their effect on synapses. In addition, the authors have shown that the drug lecanemab, recently approved by the FDA, is able to bind to them and help neutralise their action. The results are published in the journal Neuron.

A press release from Eli Lilly claims that its antibody treatment, donanemab, significantly slowed cognitive and functional decline in a phase 3 study of early-stage Alzheimer's disease. The results have not yet been published in a scientific journal.

Research published in The BMJ has compared the information on cancer drugs available to regulatory authorities with that officially received by doctors and patients. According to the authors, important information is often omitted. In particular, "none of the package inserts communicated information about the benefits of the drug that patients could expect based on the results of the studies". In addition, doubts raised by European evaluators about the reliability of the evidence "were rarely communicated to physicians, patients or the public," they say.

A meta-analysis analysing 98 published clinical trials highlights the lack of scientific evidence on the efficacy and safety of commonly used analgesic drugs for acute low back pain (such as anti-inflammatory drugs, paracetamol and opioids, among others). The study, published in The BMJ, includes data from more than 15,000 people. It concludes that there is "considerable uncertainty" about the efficacy of these drugs in reducing the severity of non-specific acute low back pain and calls on doctors and patients to use them with caution.