Interventions: Device: Photobiomodulation
Sponsors: Suez University
Not yet recruiting
Dr. Shinya Yamanaka is recognized for the generation of induced pluripotent stem cells (iPSCs) from fibroblasts by a combination of multiple transcription factors, and he won the Nobel Prize in Physiology or Medicine in 2012 jointly with Sir John B. Gurdon for this discovery. Twenty years after the discovery, the Cell Reports Medicine editorial team discusses with Dr. Yamanaka the scientific, technical, and translational milestones that have shaped the field of regenerative medicine. We also discuss the role of iPSCs in disease modeling and drug discovery, the interplay with genome editing, and ongoing issues that still prevent the widespread clinical application of iPSC-derived therapies.
A deeply phenotyped healthy Hong Kong cohort reveals age- and sex-dependent immune, clinical, and lifestyle variation, highlighting population-specific differences and the need to extend systems immunology frameworks beyond European ancestry.
Bellut et al. show that the NLRP3 inflammasome drives hyperacute inflammation and infarct progression during large vessel occlusion in stroke. Pharmacological inhibition reduces infarct growth in mice. NLRP3-positive cells in human pial blood before thrombectomy correlate with clinical outcome and highlight a potential therapeutic target prior to recanalization.
Hang et al. identify an out-of-frame CBX3::ALK fusion in metastatic melanoma that generates functional ALK isoforms through alternative translation start sites. This study demonstrates that rare noncanonical out-of-frame fusions can be oncogenic and therapeutically actionable, highlighting an analytic blind spot in current gene fusion detection pipelines.
Fei et al. review combination immunotherapy strategies for colorectal cancer, focusing on overcoming resistance in pMMR/MSS/MSI-L tumors. They highlight integrating ICIs with chemotherapy, targeted therapy, and radiotherapy and discuss insights into the tumor microenvironment and biomarkers to improve outcomes.
The Swiss drugmaker Roche on Tuesday presented the latest data for its experimental multiple sclerosis drug, setting the stage for the company to seek approval for a medicine that it believes can cut relapse rates and slow the progressive disability the disease causes.
Now the test is whether the drug, called fenebrutinib, can win the regulatory green light.
While three late-stage trials of the drug have shown it to be effective, analysts have homed in on some potentially worrying liver safety signals, an issue that previously prompted the Food and Drug Administration to reject an MS therapy developed by Sanofi. In data released Tuesday, researchers also disclosed that there were two drug-related deaths among patients who took fenebrutinib.
SAN DIEGO — Revolution Medicines is already cooking up the next iteration of RAS inhibiting drugs.
At the American Association of Cancer Research annual meeting here, the company is the talk of the town for the clinical trial success of daraxonrasib, its next generation targeted therapy, in advanced pancreatic cancer. And while the company presented more data on that drug Tuesday, showing promising first line and combination data on daraxonrasib, scientists also showed in another session intriguing preclinical data on a completely new compound that may represent what comes after the current lineup.
That drug, currently called RM-055, is what RevMed CEO Mark Goldsmith is calling an entirely “novel class of catalytic inhibitors.” These are targeted therapies that not only block the RAS signaling that drives cancer, but molecularly turn the cancer protein off.
A one-time, personalized cell therapy from Kyverna Therapeutics improved mobility and reduced disabilities in patients with stiff person syndrome, a rare, neurological autoimmune disorder, according to study results presented Tuesday.
Kyverna intends to submit the treatment to the Food and Drug Administration by the middle of the year. If approved, it would become the first treatment for stiff person syndrome and the first personalized CAR-T therapy for an autoimmune disease of any kind to reach the market.
Currently, CAR-T treatments are approved only for blood cancers, but using engineered T cells to deplete B cells — essentially performing an immune system reset inside a patient — has pushed a growing number of biotech companies to shift their CAR-T focus to autoimmune diseases.