Reframe Daily: FDA approves first cell therapy for severe aplastic anemia, plus big wins in gene and brain trials

Reframe Daily—curated by Christin Chong (neuroscience PhD, Buddhist chaplain, healthtech strategy consultant)—delivers optimistic and credible health research updates you won’t find in most popular news outlets, from sources scientists and healthcare providers read and trust.

Today in one sentence: The FDA cleared the first cell therapy for severe aplastic anemia; a one-time gene treatment into the spinal fluid helped children with spinal muscular atrophy in a big trial; a new flu shot boosted both antibodies and T cells; a daily pill for narcolepsy sharpened thinking; a combo drug plan after liver cancer surgery looked safe enough for larger tests; and home brain-stimulation for people with Alzheimer’s improved memory and daily tasks.

Good news: The FDA just cleared a brand-new cell therapy for a rare, very serious blood disease. That means patients in the US can start getting it (with a doctor’s help).

FDA Approves First Cellular Therapy to Treat Patients with Severe Aplastic Anemia

Market readiness: 🙂🙂🙂🙂🙂 (FDA-approved and available in the US now)

Good news: This study tested a one-time gene therapy given into the spinal fluid for kids with spinal muscular atrophy. A big, phase 3 trial like this is a strong step toward wider use.

Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial

Market readiness: 🙂🙂🙂🙂 (phase 3 randomized trial; could be nearing FDA review, but not broadly available yet)

Good news: Both flu shots in this study boosted protective antibodies a lot within 28 days, and they also triggered helpful T-cell responses. That’s a good sign for stronger protection.

Influenza vaccines promote humoral and cellular immune responses: a randomized, double-blind, phase 3 trial

Market readiness: 🙂🙂🙂 (phase 3 clinical trial, but not necessarily available to US patients yet)

Good news: A new daily pill for narcolepsy helped people think more clearly—like better attention and memory—in a randomized trial.

Effects of Oveporexton, an Orexin Receptor 2–Selective Agonist, on Cognition in Narcolepsy Type 1: A Secondary Analysis of a Randomized Clinical Trial

Market readiness: 🙂🙂🙂 (phase 2 randomized trial; promising, but still needs larger studies before US availability)

Good news: After liver cancer surgery, this combination treatment looked doable and ready to be tested in larger trials—important because stopping the cancer from coming back is the big goal.

Adjuvant benmelstobart plus anlotinib in patients with high-risk recurrence after resection of hepatocellular carcinoma: a phase II study (ALTER-H006)

Market readiness: 🙂🙂🙂 (phase 2 study; not a standard US treatment yet and needs bigger confirmatory trials)

Good news: People with Alzheimer’s used a home brain-stimulation device safely, and the study reports improvements in thinking and daily activities.

Home-Based Gamma Transcranial Alternating Current Stimulation for Alzheimer Disease: A Randomized Clinical Trial

Market readiness: 🙂🙂 (early clinical trial; not an established, widely available US treatment yet)

Thank you for taking the time to take care of yourself and your loved ones.