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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Isabel Medina-Vera Sergio Juárez-Méndez Droplet digital PCR is the most robust method for absolute nucleic acid quantification. However, RNA is a very versatile molecule and its abundance is tissue-dependent. RNA quantification is dependent on a reference control to estimate the abundance. Additionally, in cancer, many cellular processes are deregulated which consequently affects the gene expression profiles. In this work, we performed microarray data mining of different childhood cancers and healthy controls. We selected four genes that showed no gene expression variations (PSMB6, PGGT1B, UBQLN2 and ...
Source: Genes - Category: Genetics & Stem Cells Authors: Tags: Article Source Type: research
Childhood acute lymphoblastic leukemia (cALL) is the most frequent pediatric cancer. Over the past decades, treatment of cALL has significantly improved, with cure rates close to 90%. However intensive chemoth...
Source: BMC Medical Research Methodology - Category: Research Authors: Tags: Research article Source Type: research
Publication date: Available online 8 May 2019Source: The Lancet HaematologyAuthor(s): Daniel A Mulrooney, Geehong Hyun, Kirsten K Ness, Nickhill Bhakta, Ching-Hon Pui, Matthew J Ehrhardt, Kevin R Krull, Deborah B Crom, Wassim Chemaitilly, Deokumar K Srivastava, Mary V Relling, Sima Jeha, Daniel M Green, Yutaka Yasui, Leslie L Robison, Melissa M HudsonSummaryBackgroundTreatment for childhood acute lymphoblastic leukemia has evolved over the past five decades, with moderation of traditional chemotherapy and radiotherapy and the introduction of targeted immune-based and cellular-based therapies. The affect of these changes on...
Source: The Lancet Haematology - Category: Hematology Source Type: research
Abstract BACKGROUND: Vitamin D (25-OHD) has a role in bone health after treatment for cancer. 25-OHD deficiency has been associated with risk factors for cardiovascular disease, but no data focusing on this topic in childhood cancer survivors have been published. We investigated the 25-OHD status in children treated for acute lymphoblastic leukemia (ALL), and evaluated its influence on vascular function. METHODS: 25-OHD levels were evaluated in 52 ALL survivors and 40 matched healthy controls. Patients were grouped according to 25-OHD level (
Source: World Journal of Pediatrics : WJP - Category: Pediatrics Authors: Tags: World J Pediatr Source Type: research
Rimjhim Sonowal, Vineeta GuptaIndian Journal of Cancer 2019 56(2):180-181 Severe hyperlipidemia (>1000 mg/dL) at initial presentation of acute lymphoblastic leukemia (ALL) is rare. Cases of hyperlipidemia during therapy for childhood ALL where they were secondary to L-asparaginase or steroids have been described. This is a case report of a one-and-half-year-old boy who presented to us with fever, abdominal distension, severe pallor, and hepatosplenomegaly. Although his investigations were suggestive of ALL, the initial blood samples were found to be grossly lipemic. The lipid profile was abnormal, showing severe hyper...
Source: Indian Journal of Cancer - Category: Cancer & Oncology Authors: Source Type: research
Elda Pereira Noronha1†, Luísa Vieira Codeço Marques1†, Francianne Gomes Andrade1, Luiz Claudio Santos Thuler2, Eugênia Terra-Granado1, Maria S. Pombo-de-Oliveira1*† and the Brazilian Collaborative Study Group of Acute Leukemia‡ 1Pediatric Hematology-Oncology Program, Research Center, Instituto Nacional de Câncer, Rio de Janeiro, Brazil 2Clinical Research Division, Research Center, Instituto Nacional de Câncer, Rio de Janeiro, Brazil T-cell acute lymphoblastic leukemia (T-ALL) is a biologically heterogeneous malignancy, which reflects distinctive stages ...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
Although contemporary chemotherapy has improved the cure rate of childhood acute lymphoblastic leukemia (ALL) to nearly 90%, relapsed/refractory ALL is still a leading cause of tumor-related death in children....
Source: Cancer Cell International - Category: Cancer & Oncology Authors: Tags: Primary research Source Type: research
Discussion This case demonstrates successful cure of pre-B-ALL complicating XLA by alloSCT with restoration of B-cell development and functional antibody response. We are aware of only one previous case of pre-B-ALL in an XLA patient (21), which suggests that human BTK deficiency in itself does not predispose to pre-B-ALL. However, there are data to suggest that BTK may act as a tumor suppressor, and BTK deficiency may predispose to tumor development following a “second hit.” Mice with a genetic deficiency in Slp65, a gene encoding an adaptor protein that functions together with BTK, have a block in progenito...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
(Lund University) The most common form of childhood cancer is acute lymphoblastic leukaemia (ALL). Researchers at Lund University in Sweden, in cooperation with Karolinska Institutet, SciLifeLab and the University of Cambridge, have now carried out the most extensive analysis to date of ALL at the protein level, by studying the activity in over 8 000 genes and proteins. The results show aberrant folding in the DNA string, which in turn affects the genes' activity. The study was recently published in Nature Communications.
Source: EurekAlert! - Cancer - Category: Cancer & Oncology Source Type: news
In this study, we evaluated the U.S Food and Drug Administration (FDA) approved ALL drugs according to their physicochemical and pharmaceutical properties, and Nelarabine was found to have the highest bioactivity score. Using the key strategy of bioisosterism commonly accepted by medicinal chemists, we investigated in silico ADME properties, drug-likeness, and biological activity of new designed twenty-four compounds including Nelarabine. The results were evaluated in terms of two classifications: broad spectrum biological activity and filtering of five different drug likeness criteria of the literature including Lipinski'...
Source: European Journal of Pharmaceutical Sciences - Category: Drugs & Pharmacology Authors: Tags: Eur J Pharm Sci Source Type: research
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