Intriguing Science: genes that increase brain size
The human brain has tripled in size since our species diverged from chimpanzees, with changes in human accelerated regions (HAR) suspected to be key drivers of this increase.
In a recent article in science, Debra Silver and her team from Duke University in Durham, North Carolina, USA, managed to implement in mice a human HARE5 gene. This genetic edition led to a +6.5% brain increase. This was consistent with the observation in a second experiment where, in an organoid model, the human HARE5 lead to more growth than chimpanzee HARE5.
You are asking yourself if these HAR modified mice have grown more intelligent with this change. You will have to wait.
Unveiling brain growth mechanism in a cognitively high performing species / species of interest (a Monday compliment is welcome), has the potential to treat neurodegenerative diseases such as Alzheimer.
European Medicine Agency (EMA)
The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) held its monthly meeting from May 6 to 9, 2025. Here are the key takeaways:
- Ixchiq (live attenuated chikungunya vaccine, Valneva): the PRAC has initiated a safety review following reports of 17 serious adverse events, including two deaths, in elderly individuals. As a precautionary measure during the review, The EMA recommends that Ixchiq should not be used in adults aged 65 years and older.
- Finasteride and Dutasteride: PRAC confirmed that suicidal ideation (suicidal thoughts) is a side effect of finasteride tablets. However, it concluded that the benefits of both finasteride and dutasteride medicines outweigh their risks for all approved indications. Product information for finasteride already includes warnings about mood changes such as depression, low mood, and suicidal ideation. Patients experiencing such symptoms are advised to seek medical attention and discontinue treatment. PRAC meeting highlights.
US Food and Drug Administration (FDA)
The FDA did take the following decisions:
- Welireg (belzutifan, Merck) approval for the treatment of adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). Merck press release.
- Emrelis (telisotuzumab vedotin-tllv, AbbVie) accelerated approval for the treatment of adult patients with locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) with high c-Met protein overexpression who have received a prior systemic therapy. AbbVie press release.
- Zynyz (retifanlimab-dlwr, Incyte) approval in combination with carboplatin and paclitaxel (platinum-based chemotherapy) for the first-line treatment of adult patients with inoperable, locally recurrent, or metastatic squamous cell carcinoma of the anal canal (SCAC). Incyte press release.
- Avmapki Fakzynja Co-pack (avutometinib capsules/defactinib tablets, Verastem Oncology) accelerated approval for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC). Verastem press release.
New data from clinical trials (Phase 2, Phase 3)
Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between May 5 and May 18, 2025:
- GSK and iTeos Therapeutics confirmed they are ending the development program for belrestotug in patients with previously untreated, unresectable, locally advanced or metastatic PD-L1 high non-small cell lung cancer (NSCLC). The decision is based on new interim analyses from two Phase 2 studies, which failed to meet the pre-defined efficacy thresholds. GSK press release.
- Viatris reported positive results from two Phase 3 trials of the novel fast-acting formulation of meloxicam (MR-107A-02) for the treatment of moderate-to-severe acute pain. Viatris press release.
- Gilead Sciences shared positive results from Phase 3 study of bulevirtide in adults living with chronic hepatitis delta virus (HDV). Gilead press release.
- Johnson & Johnson announced that the Phase 3 study investigating icotrokinra (JNJ-2113) in patients with difficult-to-treat scalp and genital psoriasis met its primary endpoint. J&J press release.
- Cognition Therapeutics reported positive results from the Phase 2 trial of zervimesine (CT1812) in adults with geographic atrophy secondary to dry age-related macular degeneration (dry AMD). Cognition Therapeutics press release.
- Vir Biotechnology announced that the Phase 2 study evaluating tobevibart and elebsiran, without or with pegylated interferon alpha (PEG-IFNα), in patients with chronic hepatitis B (CHB) did not meet its primary efficacy endpoint. Vir Biotechnology press release.
- LEO Pharma shared positive results of the Phase 2b trial of temtokibart in adults with moderate-to-severe atopic dermatitis (AD). LEO Pharma press release.
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Cited articles:
- Z. Wu, L. Xu, Y. Xie, A. Sambangi, S. Swaminathan, Z. Pei, W. Ji, Z. Li, Y. Guo, Z. Li, G. Chen, Brain-Wide Neuroregenerative Gene Therapy Improves Cognition in a Mouse Model of Alzheimer’s Disease. Adv. Sci.2025, 12, 2410080. https://doi.org/10.1002/advs.202410080
- doi: https://doi.org/10.1038/d41586-025-01515-z
- Girskis, K. M. et al. Neuron 109, 3239–3251 (2021).
- Pal, A. et al. Cell 188, 1504–1523 (2025).
- Boyd, J. L. et al. Curr. Biol. 25, 772–779 (2015).
