Weekly Rundown: Gut microbes may help treat painful fibromyalgia

Welcome to the Weekly Rundown! Have suggestions for this new column? Send us an email at: editor@drugdiscoverynews.com

Gut microbes may help treat painful fibromyalgia

In results from a clinical trial published in Neuron, researchers suggested that gut microbes might help treat people with fibromyalgia — a chronic pain disorder that mainly affects women and is not well understood (1). In the study, researchers at McGill University transplanted gut bacteria from fibromyalgia patients or healthy individuals into germ-free mice. Only mice that received bacteria from fibromyalgia patients developed increased pain sensitivity and molecular changes linked to the condition, whereas mice given healthy bacteria did not. When the researchers replaced the microbes from fibromyalgia patients with microbes from the healthy participants, the mice’s symptoms improved. The team then treated 14 women with fibromyalgia with gut bacteria derived from healthy donors. Twelve of the patients reported improvements in pain, anxiety, and sleep — key symptoms of the condition. The findings suggest gut microbiota transplants could offer a viable new approach to treating this prevalent chronic pain syndrome. – Dika Ojiakor

HHS reverses freeze on Vaxart’s COVID-19 vaccine study

According to an SEC filing last Thursday, HHS has lifted its stop-work order on Vaxart’s Phase 2b trial for its oral COVID-19 vaccine. The decision allows the company to resume enrolling participants in the 10,000-person study. In February, HHS instructed Vaxart to unexpectedly halt all trial activity related to the biotech’s development of a COVID-19 vaccine tablet, except for a protocol-required follow-up with 400 participants. The trial aims to compare the company’s oral tablet with an authorized mRNA COVID-19 vaccine. The pause forced Vaxart to lay off ten percent of its workforce. According to reporting from Fierce Biotech, Vaxart will begin “reactivating field sites and screening participants for enrollment” in the study. Meanwhile, other COVID-19 vaccine developers, including GeoVax and Novavax, continue to face regulatory hurdles. – Dika Ojiakor

Highlights from AACR 2025

With more than 20,000 attendees on the ground in Chicago, the 2025 Annual Meeting of the American Association for Cancer Research (AACR) was the place to be this week. The McCormick Place Convention Center was abuzz with cancer researchers from around the world sharing their work. Keep your eye out for more DDN stories from the conference, but for now, here are some of the highlights:

People with mismatch repair deficient tumors may not need to undergo surgery or other interventions if they first receive six-months-long treatment with the PD-1 inhibitor immunotherapy drug, dostarlimab. Researchers from Memorial Sloan Kettering Cancer Center reported results from their Phase 2 trial that showed that treating people with rectal cancer or other solid tumors using the FDA approved drug led to a 100 percent complete clinical response in patients with rectal cancer and a 65 percent complete clinical response in patients with non-rectal solid tumors (2). Avoiding surgery to remove solid tumors in the rectum, stomach, or pancreas, gives patients a much higher quality of life and a survival benefit.
For patients with a KRAS G12D mutation in their non-small cell lung cancer (NSCLC) tumors, there are no approved treatments. In a Phase 1 trial, researchers treated them with zoldonrasib — a molecule developed by Revolution Medicines that locks the Ras oncogene in its active conformation. This action prevents the cancer from developing resistance to the drug by trying to overactivate the Ras signaling pathway. Patients tolerated with drug well, and for the 18 patients with efficacy results so far, 89 percent experienced disease control. Revolution Medicine also reported positive preclinical data of their drug RMC-5127 for NSCLC with KRAS G12V mutations.
Another exciting result in NSCLC came in the context of those cancers with a HER2 mutation. FDA approved HER2 inhibitors can also target the epidermal growth factor receptor, causing side effects. Zongertinib, developed by Boehringer Ingelheim, targets HER2 specifically, and in Phase 1a/1b clinical trial results, the drug proved safe and efficacious in patients with a variety of HER2 mutant NSCLC tumors. The research was also published Monday in the New England Journal of Medicine (3).
Researchers from the National Cancer Institute reported that one dose of the HPV vaccine rather than two may be sufficient for preventing HPV-associated cancers. Just requiring one dose will make the vaccine easier and cheaper to disseminate more widely, which has the potential to save millions of lives around the world.
Bristol Myers Squibb described their discovery of BMS-986449, a molecular glue degrader that targets the transcription factors Helios and Eos. When these transcription factors are overexpressed in regulatory T cells, they lead to immunosuppressive characteristics. The drug is currently in Phase 1/2 trials patients with advanced solid tumors. – Stephanie DeMarco

A new gene therapy for a rare skin disease

On Tuesday, the FDA approved a new cell-based gene therapy from Abeona Therapeutics to treat a rare skin disorder, recessive dystrophic epidermolysis bullosa (RDEB). The condition is caused by mutations in both copies of the collagen type VII alpha 1 chain gene and leads to delicate skin that blisters and forms hard-to-heal wounds all over the body. The approval was based on the results of the Phase 3 VIITAL trial which showed that the therapy improved the healing of 81 percent of wounds after a single surgical treatment compared to 16 percent treated with the standard of care. “Today’s approval of ZEVASKYN represents a pivotal moment in the treatment of RDEB, answering the call of people living with the clinical, economic, and human impact of this devastating disease,” said Vishwas Seshadri, Chief Executive Officer of Abeona in a press release. – Allison Whitten

Novavax’s COVID-19 vaccine struggles continue

Earlier this month, the FDA missed its deadline to decide whether to approve Novavax’s protein-based COVID-19 vaccine or not. It was a surprising delay in the approval process. The vaccine currently has emergency use authorization in the US, but granting it approval like Pfizer and Moderna’s COVID-19 vaccines would allow for the vaccine’s availability in the long term. This week, the Wall Street Journal reported that the FDA requested that Novavax conduct an additional randomized clinical trial to demonstrate the vaccine’s effectiveness. In a press release published on Monday, Novavax said, “We believe our application is approvable upon alignment on the details of the [postmarketing commitment for a clinical trial]. We look forward to continued engagement with the FDA.” However, in a recent X post, FDA Commissioner Martin Makary seemed to indicate that updated COVID-19 vaccines tailored to currently circulating strains might require new clinical trials, putting other vaccine updates in question and setting them up for almost certain delays. – Stephanie DeMarco

How fast a cell divides matters in the development of cancer

In a new paper published Wednesday in Nature, researchers from the Lunenfeld-Tanenbaum Research Institute revealed a key driver of cancer progression (4). By studying oncogenic mutations in mice that either turned into cancerous tissue or remained harmless, they found that the cell cycle duration — how long it takes for the cell to divide into two daughter cells — can predict a cell’s susceptibility to cancer. The team showed that cells that divide too rapidly are more likely to progress to cancer, and this was true across various types of tissues and cancers including retinoblastoma, pituitary cancer, and lung cancer. In a press release, lead study author Rod Bremner said, “Our work suggests that we might be able to intervene in cancer-prone cells to slow them down a little bit with the right therapeutic agents.” – Allison Whitten

References