A parent ’s guide to clinical trials

Children with life-threatening conditions, such as cancer, are often candidates for clinical trials. What are they? Which factors should parents weigh in determining whether enrolling in one is a good option for their child? Dr. Steven DuBois, director of the Advancing Childhood Cancer Therapies Clinic at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, answers questions about clinical trials. What are clinical trials? Why are they important? Clinical trials are systematic, scientific investigations of new drugs or therapies for a specific disease. Through clinical trials, we have taken many fatal pediatric cancers and turned them into diseases that now have cure rates of over 90 percent. Although we still want to improve those cure rates, research is now increasingly focused on making treatments more tolerable and reducing late effects of therapy. In other pediatric cancers, we are not doing as well, so our goal is to improve cure rates for patients with a poor prognosis. After decades of focusing on various chemotherapy agents and combinations to improve outcomes, much of today’s  focus is on precision medicine, immunotherapies and other novel approaches that we hope will deliver less toxic and more effective treatment than chemotherapy. What is a Phase 1 trial? Phase 2? Phase 3? When we hear about a new drug or combination that has not yet been tested in children but shows promise in laboratory research or adult trials, we must first determ...
Source: Thrive, Children's Hospital Boston - Category: Pediatrics Authors: Tags: Ask the Expert Research and Innovation clinical trial Dana-Farber/Boston Children's Cancer and Blood Disorders Center Dr. Steven DuBois Source Type: news

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CONCLUSION: Childhood cancer survivors are at an increased risk of hyperthyroidism, potentially resulting in non-endocrine morbidity. PMID: 30585514 [PubMed - as supplied by publisher]
Source: Acta Oncologica - Category: Cancer & Oncology Authors: Tags: Acta Oncol Source Type: research
In conclusion, arginase causes death of lymphoblasts by arginine-depletion induced apoptosis, via mechanism distinct from asparaginase. Therapeutic implications for childhood ALL include: arginase might be used as treatment (but antagonised by dietary arginine and citrulline), chloroquine may enhance efficacy of asparaginase treatment, and partial resistance to arginase and asparaginase may develop by BCL-2 expression. Arginase or asparaginase might potentially be used to treat Burkitt lymphoma.
Source: Apoptosis - Category: Molecular Biology Source Type: research
Publication date: Available online 12 December 2018Source: Cancer GeneticsAuthor(s): Peter Papenhausen, Carla A. Kelly, Zhenxi Zhang, James Tepperberg, Rachel D. Burnside, Stuart SchwartzAbstractT-cell acute lymphoblastic leukemia (T-ALL) is not as frequently reported as the B-cell counterpart (B-ALL), only occurring in about 15% of pediatric cases with a typically heterogeneous etiology. Approximately 8% of childhood T-ALL cases have rearrangements involving the ABL1 tyrosine kinase gene at 9q34.12; although a t(9;22), resulting in a fusion of ABL1 with the BCR gene at 22q11.23 is a common occurrence in B-ALL, it is not a...
Source: Cancer Genetics - Category: Cancer & Oncology Source Type: research
Swaminathan Dhivyasree, Jeevarathnam Dhivyalakshmi, Shuba Sankaranarayanan, Julius Xavier ScottJournal of Cancer Research and Therapeutics 2018 14(12):1244-1246 Hypercalcemia is a rare and unusual complication of childhood malignancies. Acute lymphoblastic leukemia (ALL) presenting with hypercalcemia and lytic bone lesions is very rare in children. Here, we report a case of a 4-year-old girl with ALL who presented with severe hypercalcemia and radiological evidence of osteolytic lesions. Malignancies are the most common parathyroid hormone-independent cause of hypercalcemia. Severe hypercalcemia is a life-threatening emer...
Source: Journal of Cancer Research and Therapeutics - Category: Cancer & Oncology Authors: Source Type: research
ang W, Zapotocka E, Gupta R, Schmiegelow K Abstract Asparaginase-associated pancreatitis is a life-threatening toxicity to childhood acute lymphoblastic leukemia treatment. To elucidate genetic predisposition and asparaginase-associated pancreatitis pathogenesis, ten acute lymphoblastic leukemia trial groups contributed remission samples from patients aged 1.0-17.9 years and treated from 2000-2016. Cases were defined (n=244) by at least two of the following criteria: i) abdominal pain, ii) pancreatic enzymes>3 x upper normal limit, iii) imaging compatible with asparaginase-associated pancreatitis. Controls (n=1...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
PurposeAcute lymphoblastic leukemia (ALL) is the most common cancer in childhood and has a high treatment success rate. However, survivors of childhood ALL have a high prevalence of chronic medical illnesses, such as metabolic syndrome. Dysbiosis in the gut microbiome is associated with metabolic derangement and disease risk. Chemotherapy and antibiotics increase gut microbiome dysbiosis. We tested the hypothesis that ALL treatment can cause alterations in the gut microbiome that persist in survivorship.MethodStool samples were collected on fecal occult blood test cards from 38 survivors between 2-18 years of age who were&...
Source: Blood - Category: Hematology Authors: Tags: 612. Acute Lymphoblastic Leukemia: Clinical Studies: Poster I Source Type: research
Conclusions: Akt2 might serve as a more direct and specific kinase mediating GC resistance through FoxO3a/Bim-signaling pathway in ALL, and targeting Akt2 with CCT128930 may be explored as a promising therapeutic strategy for resistance reversal.Figure.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - Category: Hematology Authors: Tags: 605. Molecular Pharmacology, Drug Resistance-Lymphoid and Other Diseases: Poster III Source Type: research
Recent studies using next-generation sequencing of selected individuals, such as those with familial leukemia or congenital syndromes, have identified rare and highly penetrant germline mutations that predispose to childhood acute lymphoblastic leukemia (ALL). High hyperdiploidy (HD), the most common cytogenetic subtype of childhood ALL, is enriched in children with RASopathies who develop ALL and, similarly, a high proportion of ALL patients with germline ETV6 or IKZF1 mutations presented with the HD subtype. Here, we aimed to identify novel predisposition genes in a selected group of HD-ALL patients.Targeted sequencing o...
Source: Blood - Category: Hematology Authors: Tags: 618. Acute Lymphoblastic Leukemia: Biology, Cytogenetics, and Molecular Markers in Diagnosis and Prognosis: ALL Risk Associated Genes and Leukemia Infiltration Source Type: research
CONCLUSION: Polyene prophylaxis offers effective antifungal activity with improved tolerability compared to older agents. The potential impact of this treatment should be included in current prophylaxis guidelines of antileukemic treatment.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - Category: Hematology Authors: Tags: 612. Acute Lymphoblastic Leukemia: Clinical Studies: Poster III Source Type: research
Children and adolescents with leukemia are potentially at a high risk of developing vitamin D deficiency, due to limited physical activity and sunlight exposure, poor nutrition, chemotherapy, and its complications. The prevalence of vitamin D inadequacy in European pediatric cancer patients has been reported to be high. It is not known how many patients already have vitamin D deficiency at the time of diagnosis and whether vitamin D status at the time of diagnosis influences clinical outcome.We aimed to investigate vitamin D status in children with leukemia at the time of diagnosis and explore possible factors (age, type o...
Source: Blood - Category: Hematology Authors: Tags: 612. Acute Lymphoblastic Leukemia: Clinical Studies: Poster III Source Type: research
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