Claim: Gene therapy shown to destroy leukemia tumors
Verdict: SUPPORTS

Retrieved passages:
title: Chemotherapy
text: of genetic mutations in oncogenes (genes that control the growth rate of cells) and tumor suppressor genes (genes that help to prevent cancer), which gives cancer cells their malignant characteristics, such as uncontrolled growth. In the broad sense, most chemotherapeutic drugs work by impairing mitosis (cell division), effectively targeting fast-dividing cells. As these drugs cause damage to cells, they are termed "cytotoxic". They prevent mitosis by various mechanisms including damaging DNA and inhibition of the cellular machinery involved in cell division. One theory as to why these drugs kill cancer cells is that they induce a programmed form of cell

title: Chronic lymphocytic leukemia
text: patients are the best candidates and which approach is best in different situations. Researchers at the Abramson Cancer Center of the University of Pennsylvania School of Medicine reported preliminary success in the use of gene therapy, through genetically modified T cells, to treat CLL. The findings, which were published in August 2011, were based on data from three patients who had modified T cells injected into their blood. The T cells had been modified to express genes that would allow the cells to proliferate in the body and destroy B cells including those causing the leukemia. Two patients went into

title: Gene therapy
text: repeated hematopoietic stem cell transplantation (see also allogeneic stem cell transplantation, allogeneic bone marrow transplantation, allotransplantation) with double-delta-32 mutation which disables the CCR5 receptor. This cure was accepted by the medical community in 2011. It required complete ablation of existing bone marrow, which is very debilitating. In August two of three subjects of a pilot study were confirmed to have been cured from chronic lymphocytic leukemia (CLL). The therapy used genetically modified T cells to attack cells that expressed the CD19 protein to fight the disease. In 2013, the researchers announced that 26 of 59 patients had achieved complete remission

title: Acute myeloid leukemia
text: and IDH2, which lead to the generation of a novel oncometabolite, "D"-2-hydroxyglutarate, which inhibits the activity of epigenetic enzymes such as TET2. The hypothesis is that such epigenetic mutations lead to the silencing of tumor suppressor genes and/or the activation of proto-oncogenes. First-line treatment of AML consists primarily of chemotherapy, and is divided into two phases: induction and postremission (or consolidation) therapy. The goal of induction therapy is to achieve a complete remission by reducing the number of leukemic cells to an undetectable level; the goal of consolidation therapy is to eliminate any residual undetectable disease and achieve a cure.

title: Chemotherapy
text: out DNA repair. Upregulation of these genes can overcome the DNA damage and prevent the induction of apoptosis. Mutations in genes that produce drug target proteins, such as tubulin, can occur which prevent the drugs from binding to the protein, leading to resistance to these types of drugs. Drugs used in chemotherapy can induce cell stress, which can kill a cancer cell; however, under certain conditions, cells stress can induce changes in gene expression that enables resistance to several types of drugs. Targeted therapies are a relatively new class of cancer drugs that can overcome many of the issues seen

title: Experimental cancer treatment
text: the patient to ensure safety via the avoidance of cytolytic destruction of noncancerous cells infected with the vector. However, new studies focus on adenoviruses that can be permitted to reproduce, and destroy cancerous cells in the process, since the adenoviruses' ability to infect normal cells is substantially impaired, potentially resulting in a far more effective treatment. Another use of gene therapy is the introduction of enzymes into these cells that make them susceptible to particular chemotherapy agents; studies with introducing thymidine kinase in gliomas, making them susceptible to aciclovir, are in their experimental stage. Epigenetics is the study of heritable

title: Artificial cell
text: survivals amongst patients with advanced-stage pancreatic cancer in phase I/II clinical trials, and a tripling in one-year survival rate. In treatment of genetic diseases, gene therapy aims to insert, alter or remove genes within an afflicted individual's cells. The technology relies heavily on viral vectors which raises concerns about insertional mutagenesis and systemic immune response that have led to human deaths and development of leukemia in clinical trials. Circumventing the need for vectors by using naked or plasmid DNA as its own delivery system also encounters problems such as low transduction efficiency and poor tissue targeting when given systemically. Artificial

title: Craig B. Thompson
text: leukemia (CLL). Thompson’s discovery of oncogenic metabolites (succinate, fumarate, and 2-hydroxyglutarate) that can inhibit tumor suppressor function and/or impair cellular differentiation has helped lead to the development of new treatments for leukemia, gliomas, sarcomas, and bladder cancer, currently in clinical trials. In December 2011, The University and its Abramson Family Cancer Research Institute sued Thompson after his move to Memorial Sloan Kettering, charging that he had made use of research conducted at the University of Pennsylvania to start a biotechnology company, Agios Pharmaceuticals that Thompson had co-founded in 2007 while still at the University. While most of the details were

title: Chemotherapy
text: a gene are produced by cancer cells. This overcomes the effect of drugs that reduce the expression of genes involved in replication. With more copies of the gene, the drug can not prevent all expression of the gene and therefore the cell can restore its proliferative ability. Cancer cells can also cause defects in the cellular pathways of apoptosis (programmed cell death). As most chemotherapy drugs kill cancer cells in this manner, defective apoptosis allows survival of these cells, making them resistant. Many chemotherapy drugs also cause DNA damage, which can be repaired by enzymes in the cell that carry

title: Severe combined immunodeficiency
text: can survive in the presence of the injected ADA. In 2000, a gene therapy "success" resulted in SCID patients with a functional immune system. These trials were stopped when it was discovered that two of ten patients in one trial had developed leukemia resulting from the insertion of the gene-carrying retrovirus near an oncogene. In 2007, four of the ten patients have developed leukemias. Work aimed at improving gene therapy is now focusing on modifying the viral vector to reduce the likelihood of oncogenesis and using zinc-finger nucleases to more specifically target gene insertion. No leukemia cases have yet been

title: P53
text: Research has shown that this restoration can lead to regression of certain cancer cells without damaging other cells in the process. The ways by which tumor regression occurs depends mainly on the tumor type. For example, restoration of endogenous p53 function in lymphomas may induce apoptosis, while cell growth may be reduced to normal levels. Thus, pharmacological reactivation of p53 presents itself as a viable cancer treatment option. The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of head and neck squamous cell carcinoma. It delivers a functional copy of the p53 gene using

title: Chronic lymphocytic leukemia
text: remission, while the presence of leukemia in the third patient reduced by 70%. One of the patients had been diagnosed with CLL for 13 years, and his treatment was failing before he participated in the clinical trial. One week after the T cells were injected, the leukemia cells in his blood had disappeared. The T cells were still found in the bloodstream of the patients six months after the procedure, meaning they would be able to fight the disease should leukemia cells return. This was the first time scientists "have used gene therapy to successfully destroy cancer tumors in patients

title: Gene therapy
text: 2009 in a trial with a genetically engineered virus with a rare mutation (CCR5 deficiency) known to protect against HIV with promising results. Clinical trials of gene therapy for sickle cell disease were started in 2014. There is a need for high quality randomised controlled trials assessing the risks and benefits involved with gene therapy for people with sickle cell disease. In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the disease. In March

title: Gene therapy
text: CAR-T cells in around a hundred people with advanced Non-Hodgkin lymphoma. In March, French scientists reported on clinical research of gene therapy to treat sickle-cell disease. In August, the FDA approved tisagenlecleucel for acute lymphoblastic leukemia. Tisagenlecleucel is an adoptive cell transfer therapy for B-cell acute lymphoblastic leukemia; T cells from a person with cancer are removed, genetically engineered to make a specific T-cell receptor (a chimeric T cell receptor, or "CAR-T") that reacts to the cancer, and are administered back to the person. The T cells are engineered to target a protein called CD19 that is common on B

title: Vectors in gene therapy
text: successful application of gene therapy to date. More than twenty patients have been treated in France and Britain, with a high rate of immune system reconstitution observed. Similar trials were restricted or halted in the USA when leukemia was reported in patients treated in the French X-SCID gene therapy trial. To date, four children in the French trial and one in the British trial have developed leukemia as a result of insertional mutagenesis by the retroviral vector. All but one of these children responded well to conventional anti-leukemia treatment. Gene therapy trials to treat SCID due to deficiency of the

title: Leukemia
text: basic research studies are generally less immediately useful to patients with the disease. Treatment through gene therapy is currently being pursued. One such approach used genetically modified T cells to attack cancer cells. In 2011, a year after treatment, two of the three patients with advanced chronic lymphocytic leukemia were reported to be cancer-free and in 2013, three of five subjects who had acute lymphocytic leukemia were reported to be in remission for five months to two years. Identifying stem cells that cause different types of leukaemia is also being researched. Leukemia is rarely associated with pregnancy, affecting only about

title: Roland Mertelsmann
text: followed. Since experiments in murine models demonstrated that local production by gene transduced cells produced a stronger and more specific immune response than a systemic application, e.g. of Interleukin-2, this strategy was also pursued by his group in Phase I clinical trials. These studies contributed to the later, successful clinical implementation of gene therapy for this and other indications. In clinical trials for patients with leukemias and lymphomas at the MSKCC and subsequently in German and European clinical trial groups, new therapeutic concepts were studied including classical chemotherapies as well as novel therapeutic strategies. For some rare, generally lethal cancers,

title: Gene therapy
text: being treated with genetically modified T cells which attacked cells with CD19 genes on their surface, i.e. all B-cells, cancerous or not. The researchers believed that the patients' immune systems would make normal T-cells and B-cells after a couple of months. They were also given bone marrow. One patient relapsed and died and one died of a blood clot unrelated to the disease. Following encouraging Phase 1 trials, in April, researchers announced they were starting Phase 2 clinical trials (called CUPID2 and SERCA-LVAD) on 250 patients at several hospitals to combat heart disease. The therapy was designed to increase the

title: Cancer and Leukemia Group B
text: of minimally invasive surgery in patients receiving state-of-the-art multimodality therapy as well as neoadjuvant therapy prior to surgery. During the 1970s, the CALGB initiated the application of immunologic methods for the study of leukemia and lymphoma. Continuing with progressively more sophisticated studies, interest has expanded to include the application of molecular-genetic techniques to characterize leukemias, lymphomas, and solid tumors at the gene level. Pharmacokinetic data are important for determining the optimal dose or schedule of a drug. The Pharmacology & Experimental Therapeutics Committee has increasingly focused on pharmacogenetics, the study of how genetic variation may impact on the toxicity or

title: X-linked severe combined immunodeficiency
text: such as the retrovirus, that incorporate the gene into the genome result in long-term effects. This, coupled with the bone marrow stem cells, has been successful in treating individuals with X-SCID. In one particular trial by Cavazzana-Calvo et al., ten children were treated with gene therapy at infancy for X-SCID. Nine of the ten were cured of X-SCID. However, about three years after treatment, two of the children developed T-cell leukemia due to insertion of the IL2RG gene near the LMO2 gene and thereby activating the LMO2 gene (a known oncogene). A third child developed leukemia within two years of