相关临床试验
438
67 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
1962
进行中（未招募）
59
13.5%
Available
1
0.2%
已完成
239
54.6%
No Longer Available
1
0.2%
尚未招募
8
1.8%
招募中
90
20.6%
暂停
2
0.5%
终止
32
7.3%
撤回
6
1.4%
暂无批准数据
- Scientists at St. Jude Children's Research Hospital found that deleting the Regnase-1 gene from CAR T cells improves their ability to control osteosarcoma and prevent lung metastasis in preclinical models. - Nearly all mice treated with Regnase-1 knockout CAR T cells survived, whereas untreated mice and those receiving conventional B7-H3-targeting CAR T cells succumbed to disease. - The modified CAR T cells remodeled the tumor microenvironment, activating other immune cells while reducing immunosuppressive signaling, supporting a broad anticancer immune response. - The St. Jude team is developing an early-phase clinical trial through the Center of Excellence for Pediatric Immuno-Oncology (CEPIO) based on these findings.
- Antisense oligonucleotides (ASOs) target messenger RNA to alter how genetic information is processed before a harmful protein is made, offering a flexible therapeutic approach for rare inherited diseases. - A single gene may be linked to one condition, but many different mutations within that gene can produce the same disease, complicating treatment strategies that ASOs are uniquely positioned to address. - Design principles established for one ASO mutation can help guide development for other variants in the same gene, accelerating the path toward personalized therapies. - Only 14 ASOs have received FDA approval to date, and St. Jude Children's Research Hospital is leading efforts to apply this technology to rare inherited pediatric diseases where conventional approaches have fallen short.
- Researchers identified YAP/TAZ transcriptional co-activators as essential regulators of bone marrow niche recovery following radiation and chemotherapy-induced injury, with knockout in MSCs and ECs significantly impairing hematopoietic regeneration. - A small molecule called GA-003 that inhibits LATS1/2 kinase and activates YAP/TAZ was shown to accelerate BM niche recovery, enhance hematopoietic regeneration, and promote engraftment after HSC transplantation in mouse models. - GA-003 acted synergistically with granulocyte colony-stimulating factor (G-CSF) to further enhance white blood cell recovery, suggesting combination potential for managing neutropenia. - The study, published in Blood on June 22, 2026, introduces a new therapeutic concept targeting the bone marrow microenvironment rather than hematopoietic cells themselves, with potential applications across chemotherapy, radiotherapy, and HSCT.
- St. Jude Children's Research Hospital's Total Therapy approach raised childhood ALL survival rates from below 20% to above 80%, now reaching 94%. - The Total Therapy 5 trial in the late 1960s combined chemotherapy with targeted cranial radiotherapy, curing roughly half of the 35 enrolled children. - The ongoing INITIALL clinical trial uses decades of genetic and outcomes data to match pediatric leukemia patients with the most effective treatments. - Long-term survivor Heidi Griffin, treated in 1967, exemplifies the enduring impact of these trials and continues participating in survivorship research.
- A St. Jude associate scientist is designing a first-in-human clinical trial for EPHA2-targeted CAR T cells in pediatric brain tumors including medulloblastoma. - The EPHA2 protein is abnormally exposed on cancer cell surfaces while largely hidden in normal tissues, offering a potentially selective therapeutic target. - Preclinical models of medulloblastoma have shown encouraging signs of activity, with safety evaluations underway before clinical translation. - The researcher transitioned from a PhD in chemistry studying cobalt ions to immunotherapy, completing the Clinical Investigations Master's Program at St. Jude Graduate School.
- Li-Fraumeni syndrome, caused by TP53 gene mutations, confers nearly a 100% lifetime cancer risk with a 50% chance before age 30. - Annual whole-body MRI screening in an Australian clinical trial is being investigated for early tumor detection in TP53 mutation carriers. - Genetic testing through programs like St. Jude's Cancer Predisposition Program can identify inherited cancer risk and guide preventive care decisions. - Patients with Li-Fraumeni syndrome undergo intensive surveillance including annual MRIs, skin checks, blood tests, and regular endoscopies and colonoscopies.
- St. Jude Children's Research Hospital has been redesignated as the WHO Collaborating Centre for Childhood Cancer, reaffirming its role as the first and only WHO collaborating centre dedicated to pediatric cancer. - The expanded work plan now includes contributions to policy and systems science, palliative care, and management of other pediatric catastrophic diseases such as sickle cell disease. - The partnership supports the WHO Global Initiative for Childhood Cancer's vision to achieve at least 60% survival for children with cancer by 2030, particularly in countries where survival is currently below 30%. - The St. Jude Global Alliance now encompasses more than 400 institutions and foundations across over 90 countries, creating a powerful implementation network for transforming pediatric cancer outcomes globally.
- Tumor-agnostic drugs and basket trials are expanding clinical trial access for people with rare cancers who historically lacked standard treatment roadmaps and trial options. - In a basket trial spanning 17 cancer types, the NTRK inhibitor larotrectinib shrank tumors in 75% of patients and prevented progression for at least one year in about 55%. - Larotrectinib became the second tumor-agnostic targeted therapy approved by the FDA based on basket trial results, offering children with CIFS a way to potentially avoid harsh chemotherapy and surgery. - The American Cancer Society's ACS ACTS program uses artificial intelligence to match patients with trials and address barriers such as low enrollment among Black, Hispanic, rural, and older patients.
- The BABY HUG study demonstrated that hydroxyurea safely reduces hospitalizations, pain crises, and organ damage in infants and young children with sickle cell disease. - St. Jude's SCCRIP longitudinal study follows over 1,800 participants to understand why disease severity varies and to guide personalized treatment decisions. - Researchers are using AI and computational approaches to identify patterns that may predict severe disease and match patients to optimal therapies, including emerging gene-based treatments. - Insights from long-term data could enable transformative therapies to be administered before cumulative organ damage increases treatment risks.
- A landmark study of 619 patients reveals that blood stem cells in aplastic anemia independently acquire protective mutations to evade autoimmune attack, with a median of three independent clones per patient. - HLA-loss clones and CHIP mutations almost never co-occur in the same cells, suggesting HLA loss alone provides sufficient proliferative advantage without selecting for pre-leukemic mutations. - The findings challenge prior assumptions by showing these protective clones can arise years before diagnosis and are associated with long-lasting remission rather than increased cancer risk. - Researchers identified CD34 enrichment as a potential biomarker for long-lasting recovery, with rescued clones showing higher expression of this surface marker.
