Chinese Scientists Achieve First-in-Human In Vivo CAR-T Therapy Using Genetically Modified Virus
核心洞察
Chinese researchers at Union Hospital of Tongji Medical University (搜索) successfully treated four multiple myeloma (搜索) patients using ESO-T01, a genetically modified lentivirus that creates CAR-T (搜索) cells directly inside the body.
Two patients achieved complete remission with tumor lesions disappearing after two months, while the remaining two showed significant tumor shrinkage within 28 days of a single injection.
This breakthrough in vivo CAR-T (搜索) approach reduces treatment time from 3-6 weeks to just 72 hours and could cut costs by more than 80% compared to traditional CAR-T therapy.
Chinese scientists have achieved a groundbreaking milestone in cancer (搜索) immunotherapy by successfully treating multiple myeloma (搜索) patients using a genetically modified virus that creates CAR-T (搜索) cells directly inside the human body. The phase 1 trial, conducted at Union Hospital of Tongji Medical University (搜索) in Wuhan, demonstrated that this revolutionary approach could reduce treatment time from weeks to days while cutting costs by more than 80%.
Revolutionary In Vivo CAR-T Technology
The research team developed ESO-T01, a lentivirus specifically engineered to target T cells (搜索) within the patient's body and program them to attack cancer (搜索) cells. Unlike traditional CAR-T (搜索) therapy, which requires extracting T cells from patients, genetically modifying them in laboratories, and reinfusing them back into the body, this new method eliminates the need for external cell manipulation.
"This is a ready-to-use product, no longer a drug that is individually formulated for each individual," the research team stated in their study published in The Lancet.
The ESO-T01 virus carries genetic code that creates a CAR against BCMA (搜索), a protein commonly found on multiple myeloma (搜索) cells. Preclinical testing in mice demonstrated both efficacy and safety of the approach.
Clinical Trial Results Show Promising Outcomes
The first-in-human trial treated four patients with advanced multiple myeloma (搜索) between November 2024 and January 2025. All patients had disease that had spread outside the bone and had received at least two prior treatments without success. Some patients were severely refractory, having failed previous CAR-T (搜索) therapy.
Each patient received a single intravenous dose of ESO-T01 without prior cell collection or preparatory chemotherapy. The treatment process took just 72 hours, compared to the 3-6 weeks required for traditional CAR-T (搜索) therapy.
After two months of follow-up, the results were remarkable. Two out of four patients achieved strict complete remission with both intra- and extra-skeletal lesions disappearing completely. Patient number 2 achieved this improvement after only 28 days. The remaining two patients experienced significant tumor shrinkage and achieved bone marrow disease-free status (MRD negative) within 28 days.
Safety Profile and Monitoring
All four patients experienced acute inflammatory symptoms following treatment, including chills and fever lasting 6-18 hours. Additional symptoms such as hypotension, hypoxemia, confusion, and headache occurred in subsequent days, ranging from mild to severe. However, all symptoms were closely monitored and promptly controlled without life-threatening complications.
CAR-T (搜索) cells began appearing in patients' blood from day 4, reaching peak levels between days 10-17, according to the research report.
Transformative Potential for Cancer Treatment
Traditional CAR-T (搜索) therapy costs can exceed 1 million yuan (approximately $139,000) for a single treatment in China. This new approach represents what Chinese social media platforms specializing in cell therapy have called a "milestone" in the field, with potential to completely change the current "custom medicine" model.
The breakthrough follows earlier work by Capstan Therapeutics (搜索) in the United States, which successfully tested a gene delivery system to create CAR-T (搜索) cells in vivo in mice. However, China is the first country to apply this technique in humans.
Future Applications and Research Directions
Scientists consider this development a breakthrough that could expand immunotherapy access to more patients, not only for cancer (搜索) treatment but also for chronic diseases such as asthma (搜索) and autoimmune diseases (搜索).
The researchers acknowledge that while these initial results are encouraging, this was a single-group, open-label, uncontrolled study. They emphasize that further studies with larger patient numbers, longer follow-up periods, and randomized controlled designs are needed to confirm long-term efficacy and safety.
The study's evaluation concluded that ESO-T01 demonstrates high therapeutic potential for refractory multiple myeloma (搜索) and represents a promising new technology and breakthrough in cancer (搜索) immunotherapy.