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Swallowed whole; bacteriu-yum
In the lexicon of ‘things that rarely occur’ we have been over-using the ‘hen’s teeth’ simile, when there was an evolutionary biology exemplar available to us. In the 3.5 billion years since life first evolved on Earth, bacteria had merged with other organisms on just three occasions: this is the origin of organelles. Now, a fourth example has been found, a once-independent bacterium has evolved into an organelle providing nitrogen to algal cells (figure). They call this the nitroplast. Optimists out there can let their imaginations run wild with the potential for new and exotic life forms, whereas the PN-reading pessimists can predict a pantheon of pathogens.
Science. 2024; 3846692: 217-222.
ASM > ASD
Topiramate was first licenced by the FDA in 1996, without sufficient scrutiny to know whether it is safe for women to take in pregnancy. Using a population-based cohort of pregnant women and their children in two health care databases in the United States, (2000 – 2020; 4,292,539 eligible pregnancies) researchers looked at developmental outcomes in children exposed to anti-seizure medication in utero. At 8 years 1.9% of all children had an autism diagnosis; this increased to 4.2% for children whose mothers had epilepsy but took no medication in pregnancy (8815 children). When mothers took topiramate this climbed to 6.2% (1030 children), valproate was 10.5% (800 children) and there was no change with lamotrigine (4.1%; 4205 children). This confirms serious concerns raised by Nordic data-linking research; expect a pregnancy prevention programme for topiramate to be coming your way soon.
N Engl J Med. 2024; 390 (12):1069–1079.
Going going GAN?
Gene therapy – big science, big opportunities, big tolerability problems? Giant axonal neuropathy (GAN) is a genetically determined autosomal recessive neurodegenerative disorder. Fourteen people with GAN were given intrathecal scAAV9/JeT-GAN in a dose-escalation study; this is a self-complementary adeno-associated virus–based gene therapy containing the GAN transgene. But I suspected you knew that much already. By 24 months, 6 of the 14 benefited, in so much that sensory-nerve action potential amplitudes increased, stopped declining, or became recordable. However, 48 serious adverse events and 682 adverse events were recorded. How many of these are genuinely treatment associated is debateable when studying a polysymptomatic progressive disorder? The delivery model (intrathecal) remains a barrier to more widespread adoption of such clever targeted therapies.
N Engl J Med. 2024; 390 (12): 1092–1104.
Of MICE and mental health
A Fo Ben has been known to cock a snook at studies that make big claims extrapolating wildly from rodent models, so it is a pleasure to support this MICE study. MICE (Mental health Intervention for Children with Epilepsy) is a UK-based parallel group, multicentre, open-label, randomised controlled trial of people aged 3–18 years. MICE used a modular psychological intervention designed to treat common mental health conditions in children and young people using evidence-based approaches such as cognitive–behavioural therapy and behavioural parenting strategies. 166 participants were randomly assigned to the MICE group and 168 were controls. As early as 6 months the MICE group had fewer difficulties, as measured by SDQ (17·6 (SD 6·3) for MICE, and 19·6 (6·1) for controls. 8% patients in the MICE group experienced at least one serious adverse event vs 14% of controls, but as you can imagine 68% of these events were seizures. The MICE system was delivered by a variety of clinicians, and included children with intellectual disability and autism, which hopefully bodes well for broader applicability in clinical practice.
Lancet. 2024; 403 (10433): 1254–1266
A Braarudosphaera bigelowii cell, with a black arrow showing its nitrogen-fixing organelle. Image: Tyler Coale, University of California, Santa Cruz.
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Footnotes
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests None declared.
Provenance and peer review Commissioned; internally peer reviewed.