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: There was a high incidence of induction deaths related to infection and high percentages of very early/early relapses, with high mortalities and low 5-year overall survival rates. These findings suggest the urgent need for modification of chemotherapy regimens to be suitable for the local conditions, including implementation of supportive care and infection control policies. There is also a requirement for antimicrobial prophylaxis during induction period combined with the necessary increase in government healthcare spending to improve the survival of acute lymphoblastic leukemia in Egyptian children. PMID...
Source: Jornal de Pediatria - Category: Pediatrics Authors: Tags: J Pediatr (Rio J) Source Type: research
Acute Lymphoblastic Leukemia (ALL) is the most common childhood cancer, representing approximately 30% of all malignant pediatric neoplasia [1,2]. In about 85% of children with ALL, the leukemia starts in the B cells (B-cell ALL). Recent advances in the chemotherapy of childhood ALL, based on a cocktail of chemotherapeutic drugs that includes inhibitors of tyrosine kinases, have resulted in survival rates of>90% [3]. Despite the clinical success of this treatment, around 20% of the children present serious toxicological complications that require a reduction in the dosage or even the complete interruption of the treatment [4].
Source: Leukemia Research - Category: Hematology Authors: Source Type: research
Pediatric Blood&Cancer, EarlyView.
Source: Pediatric Blood and Cancer - Category: Cancer & Oncology Authors: Source Type: research
We report here, for the first time, that the expression of G9a (a histone methyltransferase related with gene silencing) correlates with the expression of the integrin subunit α4 in children with ALL. We have demonstrated that G9a depletion or its inhibition with BIX01294 abrogated the ability of ALL cells to migrate through an endothelial monolayer. Moreover, G9a-depleted and BIX01294-treated cells presented bigger nuclei and more adherent phenotype than control cells on endothelial monolayers. Blocking G9a did not affect the cell cytoskeleton or integrin expression of ALL cell lines, and only its depletion redu...
Source: Cancers - Category: Cancer & Oncology Authors: Tags: Article Source Type: research
(Mary Ann Liebert, Inc./Genetic Engineering News) A new study provides novel insights into the cognitive effects of childhood acute lymphoblastic leukemia (ALL) and of chemotherapeutic treatment in long-term survivors of ALL.
Source: EurekAlert! - Cancer - Category: Cancer & Oncology Source Type: news
Authors: Shafik RE, Abd El Wahab N, Senoun SA, Ebeid E, El Taweel MA Abstract Background: MicroRNAs (miRNAs) play important roles in the pathogenesis of leukemia and their altered expression is associated with many types of solid and hematological malignancies. Methods: The study was performed on 70 consecutive newly diagnosed pediatric acute lymphoblastic leukemia (ALL) patients, of which 56 were evaluated for both bone marrow miR-128 and let-7b (all 70 for let-7b) by real-time quantitative reverse transcriptase polymerase chain reaction (RT-qPCR). In addition, seven age and sex matched healthy controls were asses...
Source: Asian Pacific Journal of Cancer Prevention - Category: Cancer & Oncology Tags: Asian Pac J Cancer Prev Source Type: research
Philadelphia chromosome–like acute lymphoblastic leukemia (Ph-like ALL; BCR-ABL1–like ALL) in children with National Cancer Institute (NCI) intermediate- or high-risk (HR) ALL is associated with poor outcome. Ph-like ALL is characterized by genetic alterations that activate cytokine receptor and kinase signaling and may be amenable to treatment with tyrosine kinase inhibitors. The prevalence, outcome, and potential for targeted therapy of Ph-like ALL in standard-risk (SR) ALL is less clear. We retrospectively analyzed a cohort of 1023 SR childhood B-ALL consecutively enrolled in the Children's Oncology Group AA...
Source: Blood - Category: Hematology Authors: Tags: Pediatric Hematology, Lymphoid Neoplasia, Clinical Trials and Observations Source Type: research
Authors: Skah S, Richartz N, Duthil E, Gilljam KM, Bindesbøll C, Naderi EH, Eriksen AB, Ruud E, Dirdal MM, Simonsen A, Blomhoff HK Abstract Autophagy is important in regulating the balance between cell death and survival, with the tumor suppressor p53 as one of the key components in this interplay. We have previously utilized an in vitro model of the most common form of childhood cancer, B cell precursor acute lymphoblastic leukemia (BCP-ALL), to show that activation of the cAMP signaling pathway inhibits p53-mediated apoptosis in response to DNA damage in both cell lines and primary leukemic cells. The pres...
Source: Oncotarget - Category: Cancer & Oncology Tags: Oncotarget Source Type: research
Pediatric Blood&Cancer, EarlyView.
Source: Pediatric Blood and Cancer - Category: Cancer & Oncology Authors: Source Type: research
Conclusion: Silymarin decreased early Doxorubicin induced left ventricular systolic function disturbances and can be recommended as adjuvant drug in patients with ALL under doxorubicin therapy. RECOMMENDATION: Multicenter studies on large number of patients with longer duration of follow up to prove the protective effects of silymarin in early and late Doxorubicin induced cardiotoxicity. PMID: 30073931 [PubMed - as supplied by publisher]
Source: Infectious Disorders Drug Targets - Category: Infectious Diseases Authors: Tags: Infect Disord Drug Targets Source Type: research
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