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Sam's News — health — 2026-09-12

Pharmaceuticals

8.5 Scholar Rock Wins FDA Approval for First SMA Muscle Loss Treatment

Scholar Rock received FDA approval for Isembyld, the first therapy targeting muscle loss in spinal muscular atrophy for patients ages 2 and older already on SMN2-targeting drugs. Clinical trial data showed Isembyld combined with SMN2-targeting therapy improved motor skills in young patients after one year, while placebo recipients declined—a statistically significant difference.

  • Isembyld approved for SMA patients age 2+ currently on SMN2-targeting therapies
  • First drug targeting muscle loss in SMA after decades of failed myostatin inhibition efforts
  • Trial showed motor skill improvement vs. decline in placebo group (statistically significant)

Sources: STAT News AI Web Searched

Health

7.5 AstraZeneca breast cancer pill fails in pivotal trial

AstraZeneca's camizestrant (Etcamah) failed its pivotal SERENA-4 trial, showing no improvement in progression-free survival compared to standard treatment for advanced ER-positive, HER2-negative breast cancer. The drug had received accelerated FDA approval in early September 2026 for resistant tumors but cannot now expand into the larger first-line market.

  • Camizestrant failed SERENA-4 pivotal trial on September 12, 2026
  • No improvement in progression-free survival vs. standard treatment
  • Drug received accelerated FDA approval weeks earlier (early September 2026)
  • Company continuing early-stage disease testing despite first-line market setback

Sources: STAT News AI Web Searched

7.5 Semaglutide shown to slow aging and extend lifespan in mice

Semaglutide, the active ingredient in Ozempic and Wegovy, extended lifespan by nearly 100 days in older mice while improving memory, muscle function, and reducing aging markers, according to an NIH-funded UC Berkeley study. The drug outperformed calorie restriction in memory and blood-sugar control, suggesting it acts through biological mechanisms of aging independent of appetite suppression.

  • Median lifespan extended by nearly 100 days in treated mice
  • Improved memory, muscle function, blood sugar control, and reduced inflammation
  • Outperformed calorie restriction in exploratory behavior and spatial memory
  • Metabolic rate remained unchanged (unlike calorie restriction)
  • Suggests separate aging pathway beyond appetite suppression

Sources: ScienceDaily AI Web Searched, ScienceDaily — Health RSS, ScienceDaily — Nutrition RSS

7.5 Scientists Identify Method to Breach Pancreatic Cancer's Immune Shield

Researchers at University of Miami's Sylvester Comprehensive Cancer Center found that blocking IL1RAP, an inflammatory receptor, can disrupt pancreatic tumors' protective immune shield and enhance treatment effectiveness. The team is planning a first-of-its-kind neoadjuvant clinical trial combining IL1RAP targeted therapy with chemoimmunotherapy in operable pancreatic cancer patients.

  • IL1RAP inhibition reduced tumor-protecting immune-suppressive cells and decreased fibrosis
  • Increased T cell activity in preclinical experiments
  • IL1RAP functions as shared control point linking tumor cells, immune cells, and fibroblasts
  • First-of-its-kind neoadjuvant clinical trial planned with chemoimmunotherapy
  • Targets pancreatic tumors' paradox: highly inflamed yet immune-suppressive

Sources: ScienceDaily AI Web Searched, ScienceDaily — Health RSS

6.5 Long-lived bats' genetic strategies may reveal human longevity secrets

Researchers studying long-lived bat species have identified genetic mechanisms combining powerful immune defenses with cell-damage elimination that could inform strategies to extend human healthspan.

Sources: ScienceDaily RSS, ScienceDaily — Health RSS

5 Heart calcium scans add limited value for most patients despite popularity

A 10-year study of over 6,000 adults found that coronary calcium scans contribute little beyond standard risk assessment for most patients, though they may help those with borderline risk.

Sources: ScienceDaily — Health RSS

Policy

5.5 Pharmaceutical Industry Wins EU Challenge Over Wastewater Treatment Cost Burden

An EU Advocate General recommended annulling a directive requiring drugmakers to finance 80% of wastewater treatment costs.

Sources: STAT News RSS