Researchers at St. Jude Children's Research Hospital have identified a new mechanism behind the rapid growth of retinoblastoma, a rare childhood eye tumor. The study found that epigenetic factors play a crucial role in cancer progression, linking an abnormal gene to aggressive tumor growth.
Researchers at St. Jude Children's Research Hospital and University College London have achieved early success with a gene therapy developed to treat hemophilia B. The treatment increased Factor IX levels in adults with the disorder, reducing the need for clotting factor injections to prevent bleeding episodes.
Researchers found a gene, Prox1, essential for normal development of the lymphatic system and proper formation of one-way valves controlling fluid movement between systems. The study provides new insights into valve defects affecting children and adults.
Researchers at St. Jude Children's Research Hospital identified a chemical known as an acetyl group that serves as a key to mediate protein interactions, which are essential for cell function. The discovery has implications for drug discovery and understanding basic mechanisms governing protein interactions.
Scientists developed a small molecule, SJ-AK, that selectively blocks a thyroid hormone signaling pathway. This approach could lead to more tailored drugs with fewer side effects, offering new hope for treating conditions like leukemia and autoimmune disorders.
A newly identified DNA repair defect may leave some young leukemia patients less likely to benefit from chemotherapy, increasing the risk of relapse. The study found low levels of a key protein and missing regulatory genes in leukemia cells, suggesting a potential new marker for high-risk patients.
Researchers found over half of diffuse intrinsic pontine glioma (DIPG) tumors carried extra copies of specific cancer-linked genes, offering potential new treatments. The study, led by St. Jude Children's Research Hospital scientists, analyzed 43 DIPG samples and identified two key regulatory mechanisms disrupted in the tumor.
Researchers used a pioneering approach to speed up drug development for ependymoma, a rare tumor with few treatment options. Dozens of potential new treatments were identified, including 5-fluorouracil (5-FU) and other chemotherapy agents.
Researchers at St. Jude Children's Research Hospital identified a hybrid cell as the origin of childhood eye tumor retinoblastoma, which has been linked to multiple developmental pathways being turned on simultaneously. The study also found that blocking certain chemicals reduced growth in human retinoblastoma cells, providing a potent...
The research identified phosphatase MKP-1 as a crucial regulator of immune balance and a bridge between innate and adaptive immune responses. The findings suggest that MKP-1 controls the production of specialized T cells, including Th1, Th17, and regulatory T cells.
Researchers at St. Jude Children's Research Hospital identified Tsc1 as a key regulator of the immune system, maintaining a balanced immune response by inhibiting effector T cells and naïve T cells. Loss of Tsc1 leads to premature activation and depletion of immune cells, compromising bacterial infection resistance.
Recent studies have shown that bone marrow transplant survival has significantly increased for young, high-risk leukemia patients, particularly those without matched donors. The results are attributed to advances in treatment and donor selection.
A study led by St. Jude Children's Research Hospital found that about 10% of adult childhood cancer survivors develop new tumors unrelated to their original cancers. Regular cancer screenings are crucial for this growing population.
Scientists at St. Jude Children's Research Hospital have developed a new algorithm called CREST to identify chromosomal aberrations in tumors, which are likely to advance our understanding of cancer. Using CREST, researchers found 89 new structural differences in cancer genomes and 50 new variations in melanoma cells.
A new study shows hydroxyurea reduces acute pain episodes, pneumonia-like illness, hospitalization time, and other symptoms in young patients with sickle cell anemia. The treatment is safe and effective for infant and toddler patients, raising hopes for improving patient quality of life.
Research from the Childhood Cancer Survivor Study reveals that adults who survived childhood cancer are particularly vulnerable to impaired memory, emotional control, and related skills due to fatigue and sleep problems. The study suggests that periodic screenings for fatigue and sleep disturbances may be beneficial for helping adult c...
Dr. Ching-Hon Pui, a world-renowned leukemia physician and researcher at St. Jude Children's Research Hospital, is the recipient of the 2011 AACR Joseph H. Burchenal Memorial Award for Outstanding Achievement in Clinical Research. He has made exceptional contributions to childhood cancer research and treatment.
New research from St. Jude Children's Research Hospital scientists shows that mutations in three pathways cooperate to launch glioblastoma, an aggressive brain tumor. Tumors develop in multiple regions of the brain, highlighting the complexity and cooperation of genetic changes driving cancer.
St. Jude researchers found that 18.3% of relapsed childhood acute lymphoblastic leukemia (ALL) patients had alterations in the CREBBP gene, compared to just 1% in non-relapsed patients. The study suggests that CREBBP mutations may be a potential indicator of relapse risk.
Researchers at St. Jude Children's Research Hospital have identified a protein called FLIP as playing a pivotal role in controlling programmed cell death pathways, offering new potential targets in the fight against cancer and virus-infected cells.
A new study found that Native American ancestry is associated with a greater risk of relapse in young leukemia patients. Administering an extra phase of chemotherapy can help eliminate this risk, according to the research published in Nature Genetics.
Research by St. Jude Children's Research Hospital found that intensive chemotherapy significantly increased the cure rate to nearly 88% for older adolescents with acute lymphoblastic leukemia, narrowing the survival gap between older and younger patients.
Researchers have identified distinct subtypes of medulloblastoma brain tumors originating from unique cells, leading to different treatment approaches. The study's findings support the development of targeted therapies and aid in searching for combinations of cells and mutations that may lead to other cancers.
Research at St. Jude Children's Research Hospital investigates managing iron overload associated with stroke prevention in young sickle cell patients. Genetic predictors of stroke risk are also being studied, with five common genetic variations validated as markers of increased risk.
A Phase I/II gene therapy trial has demonstrated no evidence of toxicity in four patients with hemophilia B. The trial uses a vector developed at St. Jude Children's Research Hospital and UCL to correct the inherited bleeding disorder.
Scientists at St. Jude Children's Research Hospital identified a signaling pathway regulating neuronal departure from the brain niche, crucial for brain architecture formation and associated with epilepsy and mental retardation. The findings also provide insight into mechanisms in epithelial tissues.
Researchers discover normal protein function, not cell buildup, causes muscle weakness and other symptoms of a rare neurodegenerative disease. The finding offers hope for treatment and a new model for understanding the mechanism at work in common nervous system disorders like Parkinson's and Alzheimer's diseases.
Researchers at St. Jude Children's Research Hospital have identified a new pathway that helps control the immune balance through reciprocal regulation of specialized T lymphocytes. The mechanism offers a promising target for new drugs against autoimmune disorders and could lead to new anti-rejection drugs.
The Six3 gene plays a pivotal role in the developing nervous system as a key regulator of the Wnt family of signaling proteins. Inhibiting Six3 disrupts the normal formation of the retina, highlighting its essential function.
Researchers identified a novel structure in cells that serves as a control switch in the body's system for eliminating damaged cells, offering new therapeutic potential. Small molecules can regulate p53 protein production and protect healthy tissue from damage caused by radiation, chemotherapy, or other factors.
A multicenter trial found that childhood cancer survivors who took methylphenidate showed improved sustained attention and social skills, with benefits lasting a year after treatment. The study offers hope and reassurance for survivors and their families.
A St. Jude researcher has received a $8.6 million NIH grant to study how genes affect responses to medicines in leukemia, with the goal of developing personalized treatment plans for kids. The project will partner with the Children's Oncology Group and move pharmacogenetic testing into real patient care.
Researchers discovered that the Prox1 gene plays a crucial role in developing and maintaining cells vital for making new memories throughout life. The study revealed a novel feedback mechanism regulating differentiation of adult neural stem cells.
Researchers discover unique combinations of cells and mutations that drive the formation of different cancer subtypes, revealing a potential breakthrough in understanding and treating ependymomas. The study also identifies the first gene linked to ependymoma and creates an accurate laboratory model of this disease.
A new experimental drug targeting the Hedgehog pathway has shown early signs of efficacy in pediatric brain tumor patients, with some still on treatment a year after diagnosis. The study's findings have given the green light for larger Phase II studies to advance targeted therapies against medulloblastomas.
An international team identified over 1,100 new compounds with confirmed activity against the malaria parasite, including two dozen families of molecules that could be candidates for drug development. The researchers launched a public database to share findings and accelerate anti-malarial drug development.
A study led by St. Jude Children's Research Hospital identified a cancer gene unusually active in some childhood brain tumors, providing a new treatment target for high-grade gliomas (HGGs). The research found significant differences between genetic defects in children and adults with HGG.
A new study from St. Jude Children's Research Hospital finds that individualized therapy and supportive care significantly improve the survival rate of young acute myeloid leukemia (AML) patients to 71 percent three years after diagnosis. This is 20 percent better than previously reported US rates.
Researchers at St. Jude Children's Research Hospital found that individuals who received the 1976 vaccine mounted a stronger immune response against both the 2009 pandemic H1N1 virus and a different H1N1 strain. The study suggests that routine vaccination against seasonal flu might confer broader-than-realized protection.
Researchers identified childhood cancer survivors at increased risk for deteriorating lung health due to bone marrow transplants. Long-term monitoring and early intervention, as well as educating high-risk patients on avoiding tobacco smoke and air pollution, are recommended to prevent respiratory problems.
Researchers at St. Jude Children's Research Hospital have identified a new class of compounds targeting the MDMX protein, implicated in promoting various cancers, including retinoblastoma. The compound, SJ-172550, has shown promise in killing cancer cells by binding to the protein and allowing p53 to function normally.
St. Jude Children's Research Hospital investigators have identified a protein that plays a pivotal role in protecting the intestinal tract from colitis. The Nlrp3 protein helps maintain healthy colon function by triggering production of epithelial cells to compensate for damage caused by colitis.
A multicenter trial found that pegfilgrastim and filgrastim were similarly safe and effective at restoring neutrophils following chemotherapy, requiring fewer injections. The study's results may transform treatment for all childhood cancer patients, easing the burdens of therapy for them.
Statins may protect individuals from serious illness following bacterial infection, including pneumococcal infections that threaten those with sickle cell disease. The cholesterol-lowering drugs employ methods to dampen inflammation and block bacterial toxins, prolonging the lives of mice with sickle cell disease.
Scientists discover GM1-ganglioside builds up in brain cells of patients with GM1-gangliosidosis, disrupting calcium balance and leading to programmed cell death. The study suggests a two-step process and identifies key proteins involved in the disease.
Arthur Nienhuis, M.D., of St. Jude Children's Research Hospital, received the 2009 ASH Mentor Award for his dedication to mentoring trainees and colleagues. His expertise in bone marrow transplantation and gene therapy paved the way for many advances in hematological disorders.
A new study aims to prevent brain complications in children with sickle cell disease by comparing two treatments: standard therapy of monthly blood transfusions and chelation vs. a daily dose of hydroxyurea and monthly phlebotomy. The goal is to reduce the risk of first strokes in high-risk patients with sickle cell anemia.
Researchers have discovered a new chromosomal abnormality in acute lymphoblastic leukemia (ALL) that is particularly common in children with Down syndrome. The finding has led to the development of new diagnostic tests and potential treatments, including an experimental medication targeting one of the altered genes.
Researchers discovered that T cells recognize a specific marker on insulin-producing cells to gain access to the islets, triggering inflammation and destruction. The findings suggest new approaches to halt or prevent type I diabetes by blocking T cell entry into the islets.
St. Jude Children's Research Hospital scientists identify inherited genes ARID5B and IKZF1 associated with childhood ALL, including a gene that may predict drug response. The study offers the first proof-based evidence of inheritance playing a role in childhood ALL.