<![CDATA[FDA accepts AXS-12 NDA for cataplexy in narcolepsy after phase 3 shows fewer attacks, better wakefulness, and cognition; decision due May 2027.]]>
Oligonucleotide–siRNA conjugate for SOD1 amyotrophic lateral sclerosis: a phase 1 trial
Nature Medicine, Published online: 15 July 2026; doi:10.1038/s41591-026-04491-7
In a human clinical trial, repeated administration of an oligonucleotide–siRNA conjugate targeting SOD1 exhibited a favorable safety profile in six patients with SOD1-ALS.
Stem Cell Therapy Advances Toward Clinical Trial for Chronic Spinal Cord Injury
A stem cell therapy designed to restore function by repairing damaged nerve pathways is moving closer to clinical testing for people with chronic spinal cord injury (SCI), according to research presented at the International Society for Stem Cell Research (ISSCR) 2026 Annual Meeting.
The work, presented by Hideyuki Okano, MD, PhD, of Keio University in Japan, builds on more than a decade of research into regenerative therapies for SCI. Okano’s team pioneered the development of induced pluripotent stem cell (iPSC)-derived neural stem/progenitor cells and launched the world’s first clinical study of the approach in patients with subacute SCI.
In March 2025, the researchers announced one-year follow-up results from that four-patient first-in-human study, reporting no serious treatment-related adverse events and neurological improvements in two participants, including one who regained the ability to stand independently and begin gait training. Although those findings have not yet been published in a peer-reviewed journal, they provided the safety foundation for expanding the program to chronic SCI.
The physician-initiated trial, expected to begin recruiting patients in 2027, will enroll individuals with chronic, incomplete SCI who retain inactive but demyelinated nerve fibers. Unlike patients treated soon after injury, these individuals often have surviving nerve pathways that have lost the myelin insulation needed for efficient signal transmission.
“We have already successfully completed a world-first, first-in-human clinical study targeting patients in the subacute phase, which demonstrated a promising safety profile,” Okano said. “Our shift to the chronic phase is the next logical milestone, built upon that solid foundation. Since the cellular environment changes over time, we are evolving our strategy from just establishing safety to actively overcoming the stubborn, long-standing barriers of chronic paralysis.”
The biology of chronic injury also required a different cell therapy. While the earlier program used neural stem/progenitor cells capable of generating neurons, the new approach uses clinical-grade gliogenic neural stem/progenitor cells (gNS/PCs), which preferentially develop into astrocytes and oligodendrocytes—the support cells responsible for maintaining neurons and restoring myelin around surviving nerve fibers. Rather than attempting to regenerate entirely new neural circuits, the therapy aims to repair and restore the function of existing ones. Earlier preclinical studies demonstrated that these gliogenic cells promoted remyelination, preserved host axons, and improved locomotor recovery in animal models, providing the rationale for advancing the therapy toward human testing.
The latest preclinical findings further support that strategy. The researchers reported that the clinical-grade gNS/PCs efficiently differentiated into neurons, astrocytes, and oligodendrocytes in laboratory studies. Following transplantation into animal models of chronic SCI, the cells promoted behavioral recovery without evidence of tumor-like tissue formation while remodeling the injured spinal cord microenvironment.
The post Stem Cell Therapy Advances Toward Clinical Trial for Chronic Spinal Cord Injury appeared first on Inside Precision Medicine.
Why Navigantis thinks it can win the stroke telesurgery race
Navigantis says it has what it takes to win the race to treat stroke patients remotely with its interventional robotic platform VASCO, which is already being used in a clinical trial for neurointerventional procedures. Stroke telesurgery is one of the biggest opportunities in medtech. Major OEMs and startups barely out of stealth mode are racing…
The post Why Navigantis thinks it can win the stroke telesurgery race appeared first on Medical Design and Outsourcing.
An anti-PMEL antibody−drug conjugate with a Gq/11 inhibitor payload in GNAQ/GNA11-mutant melanomas: a phase 1 trial
Nature Medicine, Published online: 13 July 2026; doi:10.1038/s41591-026-04518-z
In a phase 1 trial, treatment with the antibody−drug conjugate DYP688, targeting PMEL and delivering a Gq/11 inhibitor, in patients with GNAQ/GNA11-mutant metastatic uveal melanoma or other GNAQ/GNA11-mutant melanomas, showed acceptable safety and encouraging preliminary efficacy, with supporting biomarker data for proof of mechanism.
STAT+: A breakthrough in pancreatic cancer has experts excited — and braced for what’s to come
LONDON — When researchers started planning a pancreatic cancer conference here, the aim was to simply offer a counterpart to a regular U.S. meeting, where international scientists and doctors could discuss the latest developments in the specialty.
Then came the biggest shake-up in pancreatic cancer care in decades.
“It’s one of those moments,” Talia Golan, an oncologist at Israel’s Sheba Medical Center, said on a panel that conference organizers hastily added to the schedule centered on a new pancreatic cancer drug. She likened the medicine’s recent clinical trial performance to other milestones in cancer treatment such as the arrival of the first checkpoint inhibitors.
<![CDATA[Phase 3 COMP360 psilocybin data show lasting TRD relief; experts explain brain-network reset, therapy support, and what approval could mean.]]>
Effects of an e-Coach Program on the Knowledge, Attitude, and Practice of Patients Self-administering Their First Insulin Injection: Quasi-Experimental Study
Background: The current level of insulin knowledge, attitudes, and practices of patients self-administering their first insulin injection needs to be improved. There is an emerging need to develop a program for patients self-administering their first insulin injection based on the e-coach model derived from the temporal self-regulation theory. Objective: This study aimed to examine the effectiveness of a temporal self-regulation theory–based e-coach program on the knowledge, attitude, and practice of patients self-administering their first insulin injection. Methods: A quasi-experimental research design was used, and the study adhered to the Transparent Reporting of Evaluations with Nonrandomized Designs statement. The study was developed and evaluated in a level IIIA hospital between May 1 and December 1, 2022, in Suzhou, Jiangsu Province, China. The control group received care as usual. The intervention group received a self-regulation program called the e-coach program: the “COACHING” implementation steps. The insulin attitude scores and insulin knowledge-practice scores were compared using repeated-measures ANOVA. In particular, given that baseline blood glucose monitoring frequency differed significantly between the 2 groups, thereby compromising their comparability, a repeated-measures analysis of covariance (ANCOVA) was performed, with monitoring frequency entered as a covariate. This approach was adopted to enhance the scientific rigor of the findings and reduce potential bias. Results: In total, 86 patients were enrolled; of these, 75 patients were followed up, 40 in the experimental group and 35 in the control group. Repeated-measures ANOVA results revealed statistically significant between-group effects (=47.67, <.001), time effects (=5.02, =.02), and interaction effects (=4.75, =.03) for total insulin knowledge scores in both groups. In both groups, statistically significant between-group effects (=16.04, <.001) and interaction effects (=12.52, <.001) were observed for total insulin attitude scores, but time effects (=2.47, =.11) were not statistically significant. In both groups, statistically significant between-group effects (=0.66, =.004) were observed for total insulin practice scores, but time effects (=1.05, =.35) and interaction effects (=2.82, =.08) were not statistically significant. Conclusions: The e-coach program based on the TST was effective in improving insulin knowledge and insulin attitude but was not proven effective for insulin practice. A longer follow-up study is needed to uncover its long-term benefits on clinical outcomes. Trial Registration: Chinese Clinical Trial Registry ChiCTR2200058895; https://tinyurl.com/mtztdvc4
The ECHO study: compassion-focused therapy for young voice hearers and their caregivers. A pilot study
BackgroundVoice hearing can occur in healthy people without a trigger, be caused by a traumatic event, or occur as part of a psychiatric disorder. There are still many gaps in our understanding of voice hearing, and research in this field can be challenging, maybe because it is associated with shame and stigma. Those who experience distress by voice hearing could benefit from a treatment, aimed directly at that. The ECHO manualized treatment is based on the principles of compassion-focused therapy (CFT).MethodsTreatment will take place on several different psychiatric hospitals in the Region of Southern Denmark. Treatment consists of a 10-session, manualized CFT for voice hearing. Caregivers participate in half of the sessions, and a recording of the voice content is made, which the caregivers are encouraged to listen to. The study is designed as a prospective single-arm pre–post intervention study using a mirror-image approach, in which participants serve as their own controls over time. The intervention group consists of (N = 60) voice-hearing adolescents aged 13–18 years. Additionally, a clinical comparison group (who also receive the intervention) of (N = 20) individuals aged 18–20 years with first-episode schizophrenia and voice hearing is included for descriptive and exploratory analyses. Data are collected 3 months prior to CFT treatment, immediately before the start of treatment, immediately after treatment is completed, and at 1 month follow-up. Data will be centered around social cognition, perceived social safeness, social connectedness, general well being, auditory hallucinations and compassion.DiscussionThe ECHO study addresses the wellbeing and thriving of an overlooked group of young people. The results of this study will provide information about who the voice hearers are, what challenges they face, and how they perceive them. The results will also provide information about the feasibility and efficacy of the treatment manual and, thus, its potential further implementability. Caregivers participate in half of the sessions, to facilitate a common language about mental states and voice hearing. To support their understanding, a recording of the voice content is made and the caregivers are encouraged to listen to it. We suggest that when caregivers understand the young voice hearers’ experiences, they can adjust their expectations and approach accordingly. We also suggest that by sharing the voice content, the young voice hearer will feel less isolated.Study protocol registrationhttps://clinicaltrials.gov/study/NCT07314515, identifier NCT07314515.
Anti-LAG-3 with or without anti-PD-1 in recurrent glioblastoma: a phase 1 trial
Nature Medicine, Published online: 10 July 2026; doi:10.1038/s41591-026-04475-7
In a non-randomized phase 1 trial, patients with recurrent glioblastoma received anti-LAG-3 relatlimab with or without anti-PD-1 nivolumab, showing that both monotherapy and combination therapy are safe and result in encouraging preliminary clinical response signals.