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Neuroblastoma

[/cs_text][cs_text]Neuroblastoma is a solid cancerous tumor that begins in the nerve cells outside the brain of infants and young children. It can start in the nerve tissue near the spine in the neck, chest, abdomen, or pelvis, but it most often begins in the adrenal glands. The adrenal glands are located on top of both kidneys. These glands make hormones that help control body functions, such as heart rate and blood pressure.

Neuroblasts are immature nerve cells found in unborn babies. Normal neuroblasts mature into nerve cells or adrenal medulla cells, which are cells found in the center of the adrenal gland. Neuroblastoma forms when neuroblasts don’t mature properly. Sometimes, babies are born with small clusters of neuroblasts that eventually mature into nerve cells and do not become cancer. A neuroblast that does not mature can continue to grow, forming a mass called a tumor.

Neuroblastoma develops most often in infants and children younger than 5. It can form before the baby is born and can sometimes be found during a prenatal (before birth) ultrasound. Most often, however, neuroblastoma is found after the cancer has spread to other parts of the body, such as the lymph nodes, which are tiny, bean-shaped organs that help fight infection, liver, lungs, bones, and bone marrow, the spongy, red tissue in the inner part of large bones.[/cs_text][/cs_column][/cs_row][/cs_section][cs_section parallax=”false” separator_top_type=”none” separator_top_height=”50px” separator_top_inset=”0px” separator_top_angle_point=”50″ separator_bottom_type=”none” separator_bottom_height=”50px” separator_bottom_inset=”0px” separator_bottom_angle_point=”50″ style=”margin: 0px;padding: 0 0px 20px;”][cs_section parallax=”false” separator_top_type=”none” separator_top_height=”50px” separator_top_inset=”0px” separator_top_angle_point=”50″ separator_bottom_type=”none” separator_bottom_height=”50px” separator_bottom_inset=”0px” separator_bottom_angle_point=”50″ style=”margin: 0px;padding: 0 0px 30px;”][cs_row inner_container=”true” marginless_columns=”false” style=”margin: 0px auto;padding: 0px;”][cs_column fade=”false” fade_animation=”in” fade_animation_offset=”45px” fade_duration=”750″ type=”1/1″ style=”padding: 0px;”][cs_text class=”cs-ta-center”]

Neuroblastoma Trials

[/cs_text][x_prompt type=”left” title=”NMTRC012″ message=”Pediatric Precision Laboratory Advanced Neuroblastoma Therapy (PEDS-PLAN) A prospective open label, multicenter study to evaluate the feasibility and acute toxicity of using molecularly guided therapy in combination with standard therapy followed by maintenance therapy with DFMO in subjects with newly diagnosed high risk neuroblastoma. OPEN” button_text=”ClinicalTrials.gov” button_icon=”chain” circle=”false” href=”https://clinicaltrials.gov/ct2/show/NCT02559778″ href_title=”” target=”blank”][x_prompt type=”left” title=”NMTRC014″ message=”Neuroblastoma Maintenance Therapy Trial (NMTT) Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 500 to 1000 mg/m2 BID on each day of study. This study will focus on the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to prevent recurrence. OPEN” button_text=”ClinicalTrials.gov” button_icon=”chain” circle=”false” href=”https://clinicaltrials.gov/ct2/show/NCT02679144″ href_title=”” target=”blank”][x_prompt type=”left” title=”NMTRC010″ message=”Study of DFMO in Combination With Bortezomib for Relapsed or Refractory Neuroblastoma The purpose of this research study is to evaluate an investigational drug (DFMO) in combination with bortezomib, for relapsed and refractory neuroblastoma. DFMO is an investigational drug because it has not been approved by the U.S. Food and Drug Administration (FDA). This study will look at the safety and tolerability of DFMO in combination with bortezomib as well as the tumors response to this study drug. OPEN” button_text=”ClinicalTrials.gov” button_icon=”chain” circle=”false” href=”https://clinicaltrials.gov/ct2/show/NCT02139397″ href_title=”” target=”blank”][x_prompt type=”left” title=”NMTRC003B” message=”Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission The purpose of this research study is to evaluate a new investigational drug to prevent reoccurrence of neuroblastoma that is in remission. This study drug is called DFMO. The objectives of this study will be to monitor for safety and look at efficacy of DFMO.

The safety of the proposed dosing regimen in this trial will be tested by an on-going risk/benefit assessment during the study. A patient benefiting from treatment, not progressing on therapy, and in the absence of any safety issues associated with DFMO may continue on treatment up to 27 cycles with the expectation that there will be an overall clinical benefit. CLOSED” button_text=”ClinicalTrials.gov” button_icon=”chain” circle=”false” href=”https://clinicaltrials.gov/ct2/show/NCT02395666″ href_title=”” target=”blank”][x_prompt type=”left” title=”NMTRC011″ message=”A Phase I Trial of Tolcapone and Oxaliplatin in Patients with Relapsed or Refractory Neuroblastoma OPEN” button_text=”ClinicalTrials.gov” button_icon=”chain” circle=”false” href=”https://clinicaltrials.gov/ct2/show/NCT02630043?term=NCT02630043&rank=1″ href_title=”” target=”blank”][x_prompt type=”left” title=”NMTRC009″ message=”Molecular-guided therapy for the treatment of patients with relapsed and refractory childhood cancers CLOSED” button_text=”ClinicalTrials.gov” button_icon=”chain” circle=”false” href=”https://clinicaltrials.gov/ct2/show/NCT02162732″ href_title=”” target=”blank”][x_prompt type=”left” title=”ONC-403-011″ message=”A Two-Part Study of TB-403 in Pediatric Subjects with Relapsed or Refractory Medulloblastoma, Neuroblastoma, Ewing Sarcoma, or Alveolar Rhabdomyosarcoma. OPEN” button_text=”ClinicalTrials.gov” button_icon=”chain” circle=”false” href=”https://clinicaltrials.gov/ct2/show/NCT02748135″ href_title=”” target=”blank”][x_prompt type=”left” title=”V0706″ message=”A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma OPEN TO THE FOLLOWING GROUPS: RELAPSE NEUROBLASTOMA MEDULLOBLASTOMA” button_text=”ClinicalTrials.gov” button_icon=”chain” circle=”false” href=”https://clinicaltrials.gov/ct2/show/NCT00601003″ href_title=”” target=”blank”][x_prompt type=”left” title=”NMTRC002″ message=”A Phase I Trial of DFMO Targeting Polyamine Addiction in Patients with Relapsed/Refractory Neuroblastoma Neuroblastoma (NB) is the most common cancer in infancy and most frequent cause of death from extracranial solid tumors in children. Ornithine decarboxylase (ODC) expression is an independent indicator of poor prognosis in NB patients. This study investigated safety, response, pharmacokinetics, genetic and metabolic factors associated with ODC in a clinical trial of the ODC inhibitor difluoromethylornithine (DFMO) ± etoposide for patients with relapsed or refractory NB. CLOSED” button_text=”ClinicalTrials.gov” button_icon=”chain” circle=”false” href=”https://clinicaltrials.gov/ct2/show/NCT01059071?term=NCT01059071″ href_title=”” target=”blank”][/cs_column][/cs_row][/cs_section][cs_section parallax=”false” separator_top_type=”none” separator_top_height=”50px” separator_top_inset=”0px” separator_top_angle_point=”50″ separator_bottom_type=”none” separator_bottom_height=”50px” separator_bottom_inset=”0px” separator_bottom_angle_point=”50″ style=”margin: 0px;padding: 0 0px 10px;”][cs_row inner_container=”true” marginless_columns=”false” style=”margin: 0px auto;padding: 0px;”][cs_column fade=”false” fade_animation=”in” fade_animation_offset=”45px” fade_duration=”750″ type=”1/1″ style=”padding: 0px;”][cs_text class=”cs-ta-center”]

Abstracts and Publications

[/cs_text][/cs_column][/cs_row][/cs_section][cs_section parallax=”false” separator_top_type=”none” separator_top_height=”50px” separator_top_inset=”0px” separator_top_angle_point=”50″ separator_bottom_type=”none” separator_bottom_height=”50px” separator_bottom_inset=”0px” separator_bottom_angle_point=”50″ style=”margin: 0px;padding: 0 0px 20px;”][cs_row inner_container=”true” marginless_columns=”false” style=”margin: 0px auto;padding: 0px;”][cs_column fade=”false” fade_animation=”in” fade_animation_offset=”45px” fade_duration=”750″ type=”1/3″ style=”padding: 0px;”][x_feature_box title=”PLOS Phase 1 Published Paper” title_color=”” text_color=”” graphic=”icon” graphic_size=”60px” graphic_shape=”square” graphic_color=”#ffffff” graphic_bg_color=”#2ecc71″ align_h=”center” align_v=”top” side_graphic_spacing=”20px” max_width=”none” link_text=”Download PDF.” href=”https://beatnb.org/wp-content/uploads/2016/12/Plos-One-DFMO-Phase-I-Article.pdf” href_title=”” href_target=”blank” link_color=”” graphic_icon=”file-pdf-o”]A Phase I Trial of DFMO Targeting Polyamine Addiction in Patients with Relapsed/ Refractory Neuroblastoma.[/x_feature_box][/cs_column][cs_column fade=”false” fade_animation=”in” fade_animation_offset=”45px” fade_duration=”750″ type=”1/3″ style=”padding: 0px;”][x_feature_box title=”Oncotarget LIN28/Let 7 Published Paper” title_color=”” text_color=”” graphic=”icon” graphic_size=”60px” graphic_shape=”square” graphic_color=”#ffffff” graphic_bg_color=”#2ecc71″ align_h=”center” align_v=”top” side_graphic_spacing=”20px” max_width=”none” link_text=”Download PDF.” href=”https://beatnb.org/wp-content/uploads/2016/12/Oncotarget-LIN28-Let-7-NB-Sholler.pdf” href_title=”” href_target=”blank” link_color=”” graphic_icon=”file-pdf-o”]Targeting ornithine decarboxylase reverses the LIN28/Let-7 axis and inhibits glycolytic metabolism in neuroblastoma.[/x_feature_box][/cs_column][cs_column fade=”false” fade_animation=”in” fade_animation_offset=”45px” fade_duration=”750″ type=”1/3″ style=”padding: 0px;”][x_feature_box title=”ANR 2016 Abstract Book” title_color=”” text_color=”” graphic=”icon” graphic_size=”60px” graphic_shape=”square” graphic_color=”#ffffff” graphic_bg_color=”#2ecc71″ align_h=”center” align_v=”top” side_graphic_spacing=”20px” max_width=”none” link_text=”PDF.” href=”https://beatnb.org/wp-content/uploads/2016/12/ANR_2016_RINT_READY_v5_2016-10-06.pdf” href_title=”” href_target=”blank” link_color=”” graphic_icon=”file-pdf-o”]DFMO maintains remission and increases overall survival in high risk neuroblastoma: results of a phase II prevention trial.[/x_feature_box][/cs_column][/cs_row][/cs_section][/cs_content]