In the ever-evolving field of cancer treatment, two recent stories—one from the front lines of clinical research in China, and another from a patient’s journey in the United States—shine a spotlight on the remarkable progress and persistent challenges in the world of CAR T-cell therapy. These tales, though vastly different in scope and context, illustrate both the scientific ingenuity driving new therapies and the deeply personal impact such innovations have on patients and their families.
Let’s begin in China, where a team led by Zhong Wang, Yuzhang Wu, and Yulun Huang from the Hospital of Soochow University and the Army Medical University in Chongqing has just published a phase I clinical trial in Nature Communications that could change the way doctors approach one of the most aggressive brain cancers: recurrent glioblastoma. According to CMN Intelligence, this investigator-initiated trial (ChiCTR2000028801) enrolled adults whose tumors expressed the IL-13Rα2 antigen in at least 50% of cells after standard therapies—surgery, radiotherapy, and temozolomide—had failed them. The prognosis for these patients is, as the report puts it, "stark": survival is often measured in mere months, and the logistics of creating personalized, autologous CAR-T therapies can’t keep up with the rapid progression of the disease.
The trial’s answer? An off-the-shelf, or universal, CAR-T product called MT026. Instead of relying on each patient’s own cells, MT026 is engineered from donor T cells that have undergone sophisticated gene editing. The TRAC locus is disrupted to silence the endogenous T-cell receptor, and HLA class I α-chains are edited to reduce the risk of host-versus-graft reactions. This approach, the researchers note, is a clever alternative to knocking out β2-microglobulin, as it preserves some class I expression and may help the cells evade destruction by natural killer (NK) cells—a persistent hurdle for allogeneic therapies.
Preclinical assays, detailed by CMN Intelligence, showed that while allogeneic NK cells killed only 20–30% of MT026 cells (compared to 60% lysis of HLA-null controls), the engineered CAR-T cells remained potent against glioma targets. In animal models, local administration of MT026 suppressed tumor signals and modestly extended survival, suggesting the edits didn’t blunt the cells’ anti-tumor punch.
Safety, always a primary concern with gene-edited products, was rigorously assessed. Using four different methods—AID-seq, GUIDE-seq, iGUIDE-seq, and PEM-seq—the team found only one off-target hit in a coding region (MUC4), and it was present at a clone fraction of less than 0.1%. On-target translocations were rare and fell below thresholds of concern. As the authors summarized in Nature Communications: “Collectively, these multilayered assessments demonstrate: 1) sgRNA-specific off-target profiles with limited coding region impact, 2) low structural variation risk at therapeutic thresholds, and 3) precise on-target editing, supporting the safety and clinical translatability of this dual-edited UCAR-T product.”
The trial, which ran from August 2020 to July 2022, enrolled five patients (down from a planned twelve, due to COVID-19 restrictions). These were not healthy volunteers: all carried adverse molecular features (IDH wild-type and unmethylated MGMT promoter), had a mean age of 52, and four had a Karnofsky Performance Score below 70, indicating significant impairment. MT026 was administered monthly by lumbar puncture at doses of 1.0–3.0 × 107 cells, without lymphodepletion. Each patient received four to nine infusions over periods ranging from 2.6 to 7.9 months.
The results? Promising, particularly for such a difficult-to-treat population. All five patients showed radiographic tumor shrinkage; one achieved a complete response and three had partial responses, yielding an objective response rate of 80% (95% CI 37.6–96.4). The mean duration of response was 3.4 months, progression-free survival averaged 4.7 months, and overall survival from recurrence averaged 13.1 months (with one patient reaching 33.2 months). Notably, the study met its primary safety endpoint: “No grade ≥3 AEs, SAEs, immune effector cell–associated neurotoxicity syndrome (ICANS), GvHD, or infections were reported during the study,” the authors emphasized. The side effects—fever, hypoxia, vomiting, and headache—were mild (grade 1–2) and transient. Pharmacokinetic data showed that CAR DNA peaked in the cerebrospinal fluid two to four days after dosing and remained detectable between cycles, but never appeared in the blood. Cytokine analysis revealed highly localized immune activity, with CSF IL-6 concentrations 79- to 647-fold higher than serum.
Yet, as with all early-phase studies, caveats abound. The small cohort size, the lack of a control group, and the emergence of antigen loss as a resistance mechanism all point to the need for larger, more definitive trials. The researchers are already considering next-generation edits—such as augmenting HLA-E or CD47, or knocking out CIITA—to further reduce the risk of immune rejection and enhance efficacy. Still, as they conclude: “The absence of therapy-related serious adverse events and the potential clinical benefit warrant further investigation.”
Across the globe, the impact of CAR T-cell therapy is being felt in deeply personal ways. In a recent interview with CURE published on January 7, 2026, Colleen Mabasa, a patient with multiple myeloma, recounted her own experience with CAR T-cell therapy. After receiving her treatment on January 29, 2025, she underwent a bone marrow biopsy less than a month later, on February 25, which showed she had achieved measurable residual disease (MRD)–negative status—meaning no detectable disease remained. “I never anticipated it would occur that quickly,” she told CURE. “Literally, I hadn’t even gotten home yet; I was still at Sloan when I had the test and they were like, ‘Here you go, it worked.’ I can’t even tell you the mental burden and the space it lifted.”
Colleen’s story is a testament to the transformative power of new therapies—not just in terms of clinical outcomes, but in restoring hope and a sense of normalcy. “It let me just be a mom, which is pretty awesome, to be a mom and not have to worry about all these other roles,” she reflected. Her doctors had predicted a favorable response due to her low disease burden and minimal inflammation, but even they were surprised by the speed and completeness of her remission. Colleen now uses her experience to advocate for others in the myeloma community, urging patients to approach treatment decisions with confidence and optimism.
As the field of CAR T-cell therapy continues to advance, these dual narratives—one of scientific progress in the lab and clinic, the other of hope and resilience in the face of disease—remind us that every breakthrough is both a technical achievement and a profoundly human story. The journey is far from over, but for patients and their families, each step forward brings new reasons to hope.
