Author Interviews, Leukemia / 04.02.2026

Chronic lymphocytic leukemia, or CLL, and small lymphocytic lymphoma, or SLL, are chronic B cell cancers that often need long term treatment. Many patients are older at diagnosis or have other medical conditions, so the choice of first line therapy is important. Over time the goal is not only to control disease but also to preserve quality of life while keeping side effects manageable. Targeted therapies have changed how CLL and SLL are treated. Bruton tyrosine kinase (BTK) inhibitors are now commonly used because they offer oral treatment and avoid traditional chemotherapy. Zanubrutinib is a covalent BTK inhibitor that has been studied a lot in the frontline setting. Long term results from the SEQUOIA zanubrutinib study phase 3 PFS data give useful insight into how this approach performs over time compared with chemoimmunotherapy.
Author Interviews, Duke, Leukemia, Nature / 12.12.2025

MedicalResearch.com Interview with: [caption id="attachment_71759" align="alignleft" width="200"]Dr. Hirschey Dr. Matthew Hirschey[/caption] Matthew Hirschey Ph.D. Associate Professor of Medicine Associate Professor of Cell Biology Associate Professor in Pharmacology and Cancer Biology Member of the Duke Cancer Institute Member of Sarah W. Stedman Nutrition and Metabolism Center Hirschey Lab in the Duke Molecular Physiology Institute, Duke University MedicalResearch.com: What is the background for this study? Would you briefly describe AML and why new therapeutic approaches are needed? Response: Acute myeloid leukemia (AML) is an aggressive blood cancer that begins in the bone marrow and progresses rapidly. While recent advances, particularly the BCL-2 inhibitor venetoclax combined with other agents, have improved outcomes for some patients, many still relapse or don't respond to treatment. The five-year survival rate remains below 30% overall, highlighting an urgent need for new therapeutic strategies. We know that cancer cells rewire their metabolism to fuel rapid growth, and the mitochondria (the cell's powerhouses) play a central role. However, understanding exactly how different metabolic pathways connect and depend on each other has been challenging. We wanted to develop better tools to map these connections and identify new vulnerabilities we could potentially target.
Author Interviews, Genetic Research, Leukemia, Personalized Medicine / 12.11.2024

MedicalResearch.com Interview with: [caption id="attachment_64714" align="alignleft" width="125"]Professor Ong Sin TiongCancer & Stem Cell Biology Signature Research Programme Duke-NUS Medical School, Singapore Prof. Ong Sin Tiong[/caption] Professor Ong Sin Tiong Cancer & Stem Cell Biology Signature Research Programme Duke-NUS Medical School, Singapore [caption id="attachment_64743" align="alignleft" width="125"]Dr. Yu Mengge Dr. Yu Mengge[/caption] Dr Yu Mengge Research Fellow, Cancer & Stem Cell Biology Signature Research Programme Duke-NUS Medical School MedicalResearch.com: What is the background for this study? Response: The background of this study is rooted in the observation that certain genetic variations among East Asian populations, notably the BIM deletion polymorphism (BDP), impact treatment outcomes in chronic myeloid leukaemia (CML). Patients with the BDP show resistance to conventional treatments, specifically tyrosine kinase inhibitors like imatinib. This resistance stems from the variant's role in promoting cancer cell survival, which leads to more aggressive disease progression.
Author Interviews, Cancer Research, Leukemia, Stem Cells / 01.11.2024

MedicalResearch.com Interview with: [caption id="attachment_64480" align="alignleft" width="130"]Eirini Papapetrou, MD, PhDProfessor of Oncological Sciences Professor of Medicine, Hematology and Medical Oncology Director, Center for Advancement of Blood Cancer Therapies Co-Director, Stem Cell Engineering Core Icahn School of Medicine at Mount Sinai New York, NY 10029 Dr. Papapetrou[/caption] Eirini Papapetrou, MD, PhD Professor of Oncological Sciences Professor of Medicine, Hematology and Medical Oncology Director, Center for Advancement of Blood Cancer Therapies Co-Director, Stem Cell Engineering Core Icahn School of Medicine at Mount Sinai New York, NY 10029   MedicalResearch.com: What is the background for this study? What are the main findings? Response: RAS in the most commonly mutated oncogene in human cancers. Particularly in acute myeloid leukemia, about one third of cases have RAS mutations. We set out to understand the role of these mutations in the development of leukemia and in response to treatment. We found that RAS mutations happen late in the course of the disease as progression mutations because they are acquired by more mature leukemic cells coming from preexisting leukemia stem cells (LSCs). Importantly, these more mature cells, upon acquisition of RAS mutations, become leukemia stem cells (LSCs) with different properties than the previous LSCs. Most critically, they develop resistance to a recently FDA-approved drug for the treatment of leukemia, venetoclax (VEN). In addition, these RAS-mutated LSCs give rise to leukemia cells with monocytic differentiation. Both RAS mutations and monocytic differentiation of AML have previously been associated with VEN resistance in clinical studies. We show that it is the RAS mutations that cause both the monocytic differentiation and the VEN resistance. Thus, poor patient outcomes after VEN therapy are driven by RAS mutations and not by monocytic disease. 
Hematology, Leukemia / 02.07.2024

bone-marrow-aspirateBone marrow aspiration is a medical procedure where a small amount of the liquid part of your bone marrow is removed for examination. A bone marrow aspirate can be essential in diagnosing and monitoring various conditions affecting your blood cells, such as anemia, leukemia, and infections. Understanding this procedure can provide insight into why your doctor may recommend it and what to expect. During a bone marrow aspiration, your healthcare provider will take a sample using a needle inserted into a larger bone, typically the hip. The process is usually performed alongside a bone marrow biopsy, which involves taking a small amount of solid bone marrow tissue. These procedures can yield valuable information about the health and functionality of your bone marrow. Preparation for bone marrow aspiration involves a few straightforward steps to ensure your comfort and safety. Knowing the potential risks and benefits can help you feel more at ease and prepared for this critical diagnostic tool.
Author Interviews, Cancer Research, Hematology, Leukemia / 15.06.2023

Medical Research Interview: [caption id="attachment_60524" align="alignleft" width="195"]Dr. Daniel Thomas Dr. Thomas[/caption] Dr. Daniel Thomas MD PhD FRACP FRCPA Program Leader, Blood Cancer Precision Medicine Theme at the South Australia Health Medical Research Institute Clinical Hematologist, Royal Adelaide Hospital Associate Professor, Adelaide Medical School, The University of Adelaide MedicalResearch.com: What is the background for this study? Would you briefly describe the condition of CMML? Response: Chronic myelomonocytic leukemia (CMML) is a rare, but increasingly frequent, clonal stem cell disorder that results in hyperproliferation of inflammatory monocytes, a form of white blood cells. It features both myelodysplasia and myeloproliferation. CMML is most often found in older adults and leads to anemia, decreased quality of life, and an increased risk of acute myeloid leukemia (AML). Granulocyte-macrophage colony-stimulating factor (GM-CSF) is a cytokine that stimulates production, growth, differentiation, activation, and function of myeloid cells (monocytes, neutrophils, and eosinophils). In the presence of RAS-pathway mutations, a greater sensitivity to GM-CSF contributes to the hyperproliferation of myelocytes in myelodysplastic leukemias such as CMML, juvenile myelomonocytic leukemia (JMML), and acute myeloid leukemia (AML).  In CMML, greater sensitivity to GM-CSF stimulates excessive monocytic precursor proliferation. The PREACH-M Trial, which stands for PREcision Approach to CHronic Myelomonocytic Leukemia, assesses the efficacy of lenzilumab in addition to azacitidine in treatment-naïve CMML participants with RAS-pathway mutations (KRAS, NRAS, CBL) and separately high dose ascorbate in participants with TET2 mutations who do not have RAS-pathway mutations. The study is currently underway and actively enrolling.  It is being conducted and funded by the South Australian Health and Medical Research Institute (SAHMRI). 
Author Interviews, Leukemia, Stem Cells, Technology / 11.02.2021

MedicalResearch.com Interview with: [caption id="attachment_56645" align="alignleft" width="130"]Eirini Papapetrou, MD, PhD Associate Professor Department of Oncological Sciences Icahn School of Medicine at Mount Sinai New York, NY 10029 Dr. Papapetrou[/caption] Eirini Papapetrou, MD, PhD Associate Professor Department of Oncological Sciences Icahn School of Medicine at Mount Sinai New York, NY 10029 MedicalResearch.com: What is the background for this study? Would you tell us a little about acute myeloid leukemia? Response: Acute myeloid leukemia is a form of cancer of the blood. It is typically very aggressive and lethal without treatment. The main treatment is high-dose chemotherapy and it has not changed very much in decades. Some more recent "targeted" therapies that are less toxic help somewhat but still do not result in cures. We believe a reason for this might be that both chemotherapy and newer "targeted" therapies target the cells at the later stages of the disease and spare the earlier ones, which can then give rise to disease resistance and relapse. 
Author Interviews, Cancer Research, COVID -19 Coronavirus, Leukemia / 06.12.2020

MedicalResearch.com Interview with: William Wood, MD Associate Professor of Medicine, Division of Hematology UNC School of MedicineWilliam Wood, MD Associate Professor of Medicine, Division of Hematology UNC School of Medicine MedicalResearch.com: What is the background for this study? What are the main findings? Response: In the earliest days of the COVID-19 pandemic, there were concerns that individuals with cancer, and especially those with hematologic malignancies, could be at higher risk for adverse outcomes following COVID-19 infection than the general population. For this reason the ASH Research Collaborative developed a voluntary data collection registry in which providers or sites caring for patients with hematologic malignancies and COVID-19 infection provided de-identified data via an online data collection platform. We believe that our findings – that 20% of patients with blood cancers who had COVID-19 infection died, including 33% of those who required hospital or ICU level-care – indicate that patients with blood cancers are a medically vulnerable group when it comes to COVID-19. The risk of dying was highest in patients who were older, had more severe infection, opted to forego more intensive treatment, and/or had poorer prognosis before their COVID-19 infection, as determined by their treating clinicians
Author Interviews, Cancer Research, Leukemia / 16.06.2020

MedicalResearch.com Interview with: [caption id="attachment_54594" align="alignleft" width="140"]Courtney D. DiNardo, M.D., MSCE Department of Leukemia, Division of Cancer Medicine The University of Texas MD Anderson Cancer Center Dr. DiNardo[/caption] Courtney D. DiNardo, M.D., MSCE Department of Leukemia, Division of Cancer Medicine The University of Texas MD Anderson Cancer Center MedicalResearch.com: What is the background for this study? Response: At this year’s virtual European Hematology Association (EHA) meeting, we are presenting late-breaking data from the Phase 3 VIALE-A trial. The randomized double-blind, placebo-controlled trial evaluated venetoclax in combination with azacitidine in previously-untreated patients with acute myeloid leukemia (AML) who are ineligible for standard induction therapy compared to azacitidine plus placebo. 
ASCO, Author Interviews, Cancer Research, Leukemia, UC Davis / 28.05.2020

MedicalResearch.com Interview with: [caption id="attachment_54355" align="alignleft" width="150"]Dr-Brian A. Jonas Dr. Jonas[/caption] Brian A. Jonas, M.D., Ph.D. UC Davis Health System MedicalResearch.com: What is the background for this study? At this year’s American Society of Clinical Oncology (ASCO) and European Hematology Association (EHA) virtual meetings, we presented data on the rapidity and likelihood of response to venetoclax treatments, and its associated characteristics, in older patients with newly diagnosed acute myeloid leukemia (AML). We evaluated data from two clinical trials of venetoclax in combination with azacitidine, or decitabine (M14-358), or low-dose cytarabine (LDAC) (M14-387) in this patient population.
Author Interviews, Cancer Research, Leukemia / 11.12.2019

MedicalResearch.com Interview with: [caption id="attachment_52430" align="alignleft" width="167"]Bridget Marcellino, MD Icahn School of Medicine at Mount Sinai Mount Sinai Hospital Dr. Marcellino[/caption] Bridget Marcellino, MD Icahn School of Medicine at Mount Sinai Mount Sinai Hospital MedicalResearch.com: What is the background for this study? Response: Our work focuses on elucidating the mechanisms that drive the pathogenesis and progression of myeloproliferative neoplasms (MPN). Dysregulation of the TP53 pathway is associated with MPN progression evidenced by the association of TP53 loss of heterozygosity with transformation to acute myeloid leukemia (AML) and the presence of inactivating mutations of TP53 found in a proportion of MPN-related AML patients.   Studies have shown that TP53 mutations, TP53 deletions and overexpression of the negative regulator of TP53, Murine Double Minute 2 (MDM2) all contribute to TP53 downregulation in MPNs and we therefore are interested in exploring other potential means by which TP53 is downregulated. Protein Phosphatase, Mg2+/Mn2+ Dependent 1D (PPM1D) is another negative regulator of the TP53 pathway and activating mutations in this gene are present in myeloid malignancies including MPNS. We therefore hypothesized that genomic alterations in PPM1D and/or overexpression of PPM1D would be found in the hematopoietic cells of MPN patients.
Author Interviews, Chemotherapy, Leukemia / 18.06.2019

MedicalResearch.com Interview with: [caption id="attachment_49825" align="alignleft" width="133"]Dr Carlos Buesa Dr Buesa[/caption] Dr. Carlos Buesa PhD Chief Executive Officer Oryzon Genomics  MedicalResearch.com: What is the background for this study? Response: Lysine specific demethylase 1 (LSD1, also known as KDM1A) is a histone modifying enzyme that removes methyl groups, thereby regulates the expression of many genes important in cancer progression and cell proliferation. Aberrant expression of LSD1 has been shown in many types of cancers. In particular, LSD1 expression is upregulated in bladder, small cell lung (SCLC), and colorectal clinical cancer tissues when compared with the corresponding nonneoplastic tissues. LSD1 has also been shown to be overexpressed in some breast cancers and may function as a biomarker of the aggressiveness of the disease. However, the function of LSD1 is most understood in acute leukemia, particularly Acute Myeloid Leukemia and Acute Lymphoblastic leukemia. LSD1 is crucial for the function and maintenance of the leukemic stem cells, a subset of malignant cells that is believed to be the ultimate reason for relapse in these patients. LSD1 inhibition might be a therapeutic solution to avoid those relapses.
ASCO, Author Interviews, Bayer, Leukemia, Pediatrics / 05.06.2019

MedicalResearch.com Interview with: Joseph Germino, M.D., PhD Vice President US Medical Affairs Oncology Bayer Healthcare Pharmaceuticals Whippany, N.J. 07981 MedicalResearch.com: What is the background for this study? Response: Sorafenib (Nexavar®) is an oral anticancer therapy approved in more than 100 countries worldwide. It is approved for the treatment of patients with advanced hepatocellular carcinoma (HCC); advanced renal cell carcinoma (RCC) who have failed prior interferon-alpha or interleukin-2 based therapy or are considered unsuitable for such therapy; progressive, locally advanced or metastatic differentiated thyroid carcinoma (papillary/follicular/Hürthle cell), that is refractory to radioactive iodine (RAI). The AAML 1031 is a recently completed Phase III clinical trial evaluating the use of bortezomib and sorafenib in patients 30 years or younger with newly diagnosed acute myeloid leukemia (AML). At the 2019 ASCO Annual meeting, results of a report from the AAML1031 trial, which assessed whether intensification of Induction II chemotherapy (ADE or AraC/ Mitoxantrone) and liberalized stem cell transplant (SCT) donor source criteria improved clinical outcomes in patients with residual AML. 
ASCO, Author Interviews, Cancer Research, J&J-Janssen, Leukemia / 03.06.2019

MedicalResearch.com Interview with: [caption id="attachment_49526" align="alignleft" width="144"]Paul M. Barr, M.D. Associate Professor of Medicine and Director of the Clinical Trials Office Director of the Clinical Trials Office Wilmot Cancer Institute Dr. Barr[/caption] Paul M. Barr, M.D. Associate Professor of Medicine and Director of the Clinical Trials Office Director of the Clinical Trials Office Wilmot Cancer Institute  MedicalResearch.com: What is the background for this study?   Response: When the study was designed, chronic lymphocytic leukemia (CLL)  treatment options were largely limited to chemotherapy and monoclonal antibodies. Ibrutinib had shown promise in early studies. The intent was to compare ibrutinib to a standard of care treatment option at that time, of atumumab, in patients with relapsed or refractory disease. The goal of the current analysis is to evaluate the durability of ibrutinib and report the long-term safety results.
Author Interviews, Cancer Research, Genentech, Leukemia / 25.03.2019

MedicalResearch.com Interview with: [caption id="attachment_48117" align="alignleft" width="200"]Nancy Valente, M.D.VP of Global Hematology DevelopmentGenentech Dr. Valente[/caption] Nancy Valente, M.D. VP of Global Hematology Development Genentech Dr. Valenta discusses the announcement of the submission by Genentech of a supplemental New Drug Application to the FDA for Venclexta plus Gazyva for people with previously untreated chronic lymphocytic leukemia  with co-existing medical conditions. MedicalResearch.com: What is the background for this study? What are the main findings of the Phase III CLL14 study?  Response: We completed the submission of a supplemental New Drug Application (sNDA) to the FDA for Venclexta® (venetoclax) in combination with Gazyva® (obinutuzumab) in people with previously untreated chronic lymphocytic leukemia (CLL) and co-existing medical conditions. CLL is the most common form of adult leukemia and more than 20,000 new cases will be diagnosed in the U.S. this year. The sNDA is based on data from the Phase III CLL14 study, which evaluated fixed-duration Venclexta in combination with Gazyva in people with previously untreated CLL. Results showed this chemotherapy-free combination can help people with previously untreated CLL live significantly longer without their disease worsening (progression-free survival; PFS) compared to standard-of-care Gazyva plus chlorambucil. The FDA is reviewing our application under the Real-Time Oncology Review (RTOR) pilot program, which is exploring a more efficient review process to ensure safe and effective treatments are available to patients as early as possible. 
Author Interviews, Breast Cancer, Cancer Research, JAMA, Leukemia / 21.01.2019

MedicalResearch.com Interview with: medicalresearch.comDr. Marie Joelle Jabagi, PharmD, MPH University of Paris Sud, Paris-Saclay University, Paris Health Product Epidemiology Department French National Agency for Medicines and Health Products Safety Saint-Denis, France MedicalResearch.com: What is the background for this study? Response: Secondary hematologic malignant neoplasms that develop months or years after the diagnosis of breast cancer may be a consequence of genetic predisposition, environmental factors, previous cancer treatments or a combination of all those factors. These secondary malignant neoplasms are increasingly becoming a concern given that the population of breast cancer survivors is growing substantially. However, their frequency in real life has been poorly investigated to date. The aims of our research were to estimate the frequency of various types of hematologic malignant neoplasm following a diagnosis of primary breast cancer among women aged 20 to 85 years in France during the past decade, and to compare it to the corresponding frequency in women of the French general population.
Author Interviews, Hematology, Leukemia, Pediatrics / 05.12.2018

MedicalResearch.com Interview with: [caption id="attachment_46330" align="alignleft" width="200"]Charles G. Mullighan, MBBS (Hons), MSc, MD Member, St. Jude Faculty Co-Leader, Hematological Malignancies Program Medical Director, St. Jude Biorepository William E. Evans Endowed Chair St. Judes Children’s Research Hospital Memphis, TN Dr. Mullighan[/caption] Charles G. Mullighan, MBBS (Hons), MSc, MD Member, St. Jude Faculty Co-Leader, Hematological Malignancies Program Medical Director, St. Jude Biorepository William E. Evans Endowed Chair St. Judes Children’s Research Hospital Memphis, TN MedicalResearch.com: What is the background for this study?   Response: B-lineage acute lymphoblastic leukemia (B-ALL) is the commonest form of ALL, and the commonest childhood tumor. It is a leading cause of childhood cancer death. It consists of multiple subtypes defined by genetic alterations. These are often chromosomal translocations that deregulate oncogenes or form fusion proteins. These alterations are disease initiating events and are associated with distinct patterns of leukemic cell gene expression. Most subtypes also have additional mutations that are important for cells to become fully leukemic. Identifying these initiating genetic changes is very important to identify patients that are likely to respond or do poorly with conventional therapy (multiagent chemotherapy). Also, some identify new opportunities for targeted therapy. However, using standard genetic testing approaches such as chromosomal cytogenetics, about 30% of B-ALL patients don’t have a subtype classifying alteration.
Anemia, Author Interviews, Cancer Research, Hematology, Leukemia / 03.12.2018

MedicalResearch.com Interview with: [caption id="attachment_46334" align="alignleft" width="150"]Dr. Alan List MD President and Chief Executive Officer Moffitt Cancer Center Tampa, FL Dr. List[/caption] Dr. Alan List MD President and Chief Executive Officer Moffitt Cancer Center Tampa, FL MedicalResearch.com: What is the background for this study?   Response: In patients with lower risk Myelodysplastic Syndromes (MDS), which accounts for the vast majority of patients with MDS overall, the most common symptomatic cytopenia is anemia. These patients, overtime, become dependent upon red blood cell transfusions and with that, they face a risk of iron loading as well as complications that occur with it. The standard first line therapy that we consider for these patients is erythropoietin-stimulating agents (ESAs). Patients who are transfusion dependent have a low response rate to ESAs, and responses are of short duration. There limited effective limited treatment options for those patients unresponsive or lose response to ESAs. For years, we’ve known that the transforming growth factor (TGF)-β pathway play an important pathogenetic role in suppressing red cell maturation and cell survival. Luspatercept is an agent that acts as an erythroid maturation agent by inhibiting the TGF-β signaling pathway by neutralizing a select group of TGF-β superfamily ligands. 
Author Interviews, Leukemia / 11.09.2018

MedicalResearch.com Interview with: [caption id="attachment_44407" align="alignleft" width="172"]Hans van Eenennaam, Ph.D. Executive vice president antibody research and site head Aduro Biotech Europe Dr. van Eenennaam[/caption] Hans van Eenennaam, Ph.D. Executive vice president antibody research and site head Aduro Biotech Europe MedicalResearch.com: What is the background for this study? Response: A PRoliferation Inducing Ligand (APRIL) is a member of the tumor necrosis factor superfamily and has been shown to stimulate the proliferation and survival of healthy B-lymphocytes. Malignant B-lymphocytes, such as multiple myeloma, use APRIL primarily in the bone marrow to support its proliferation and survival. Studies have shown that APRIL is overproduced in patients with multiple myeloma and mediates primarily via binding to B-cell maturation antigen (BCMA) to stimulate a wide variety of responses that promote multiple myeloma growth and survival, as well as suppressing the immune system so that the tumor cells are protected and sustained in the bone marrow and can escape current available treatments.
Abuse and Neglect, Dermatology, Leukemia, Lymphoma, Melanoma / 15.08.2018

MedicalResearch.com Interview with: [caption id="attachment_43885" align="alignleft" width="160"]Clive S. Zent MD Professor of Medicine Director of Lymphoma/CLL Program Wilmot Cancer Institute University of Rochester Medical Center Rochester NY Dr. Zent[/caption] Clive S. Zent MD Professor of Medicine Director of Lymphoma/CLL Program Wilmot Cancer Institute University of Rochester Medical Center Rochester NY MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL) have an increased risk of all skin cancers including malignant melanoma. This study in a stable population of CLL patients managed by a regional referral center confirmed that melanoma was over 6 times more common in than in an age and sexed matched general population. Because of the proactive skin screening program at the University of Rochester Medical Center’s Wilmot Cancer Center, most melanomas (77%) were detected at earlier stages and were treated surgically with curative intent. One patient with CLL and metastatic melanoma had a sustained remission of both diseases on treatment with ibrutinib and pembrolizumab.
Author Interviews, Cancer Research, JAMA, Leukemia, Transplantation / 30.07.2018

MedicalResearch.com Interview with: [caption id="attachment_43481" align="alignleft" width="156"]Smita Bhatia, MD, MPH Gay and Bew White Endowed Chair in Pediatric Oncology Professor, Pediatric Oncology Vice Chair for Outcomes Research, Dept of Pediatrics Director, Institute for Cancer Outcomes and Survivorship School of Medicine University of Alabama at Birmingham Associate Director for Outcomes Research UAB Comprehensive Cancer Center  Dr. Bhatia[/caption] Smita Bhatia, MD, MPH Gay and Bew White Endowed Chair in Pediatric Oncology Professor, Pediatric Oncology Vice Chair for Outcomes Research, Dept of Pediatrics Director, Institute for Cancer Outcomes and Survivorship School of Medicine University of Alabama at Birmingham Associate Director for Outcomes Research UAB Comprehensive Cancer Center  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Allogeneic bone marrow transplantation BMT is used with a curative intent for life-threatening malignant and non-malignant diseases of childhood. In this observational study, we describe the late mortality experienced by children undergoing BMT over the past 3 decades. Our cohort included 1388 BMT recipients who had undergone allogeneic BMT between 1974 and 2010 and survived 2 or more years. We found that, conditional on surviving the first 2 years after bone marrow transplantation, the probability of surviving an additional 20 years approached 80%. Risk of dying from non-relapse-related causes exceeded the risk of dying from relapse-related causes. The leading non-relapse-related causes of death were infection (with or without graft vs. host disease) and new cancers. Overall, the cohort was at a 14-fold greater risk of dying as compared with the general population (of similar age and sex). Further, this excess risk remained elevated even among those who had survived 25 years. On a positive note, the risk of late mortality has continued to decline over the past 3 decades. 
Abbvie, ASCO, Author Interviews, Cancer Research, Leukemia / 03.06.2018

MedicalResearch.com Interview with: [caption id="attachment_42144" align="alignleft" width="200"]Dr. Danelle James, Dr. James[/caption] Dr. Danelle James, M.D., M.A.S. Head of Clinical Science Pharmacyclics, an AbbVie Company MedicalResearch.com: What is the background for this study? What are the main findings? Response: CAPTIVATE is a Phase 2 study investigating IMBRUVICA (ibrutinib) plus VENCLEXTA (venetoclax) for the treatment of Chronic Lymphocytic Leukemia (CLL) in the first-line setting. It was designed to evaluate if remission with undetectable minimal residual disease (MRD) can provide treatment-naïve CLL/SLL patients with treatment holidays (a period of time when a patient is able to stop therapy). The study enrolled 164 patients with previously untreated CLL or SLL. In preclinical and ongoing clinical studies, we’ve seen complementary activities with this combination. The combination has also previously shown potential for deeper remissions, as well as potential for lower risk of tumor lysis syndrome with ibrutinib as the lead-in therapy. Early data from CAPTIVATE show promising activity for the combination in this patient population, with 77 percent of the first 30 patients achieving responses with no detectable MRD in the blood after only six cycles of the combination therapy. Approximately nine out of 10 of the first patients achieved undetectable MRD after 12 cycles of combination therapy (which were preceded by three cycles of single agent ibrutinib, for a total of 15 cycles of therapy). Specifically, 86 percent of the first 14 patients achieved undetectable MRD in the marrow and 93 percent in the peripheral blood.
Author Interviews, Cancer Research, Leukemia, Nature, University of Pennsylvania / 01.06.2018

MedicalResearch.com Interview with: [caption id="attachment_42093" align="alignleft" width="148"]Dr. J Joseph Melenhorst, PhD Director, Product Development & Correlative Sciences laboratories (PDCS) Adjunct Associate Professor Penn Medicine Center for Cellular Immunotherapies University of Pennsylvania Dr. Melenhorst[/caption] Dr. J Joseph Melenhorst, PhD Director, Product Development & Correlative Sciences laboratories (PDCS) Adjunct Associate Professor Penn Medicine Center for Cellular Immunotherapies University of Pennsylvania MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by CLL and CAR T cells?  Response: We started treating patients with a form of blood cancer called CLL (chronic lymphocytic leukemia) using a form of gene therapy wherein we engineer the patient’s own immune cells – T cells – with a tumor targeting molecule: The CAR, which stands for chimeric antigen receptor. When we engineer the patient’s immune cells we use a vehicle, in this case virus, that inserts the payload – the CAR – into the patient’s DNA. The virus disappears, and the CAR stays. Where this CAR inserts itself is unpredictable, but we always get stably engineered cells. 
Author Interviews, Global Health, JAMA, Leukemia / 21.05.2018

MedicalResearch.com Interview with: [caption id="attachment_41916" align="alignleft" width="125"]Andrew J. Cowan, MD Seattle Cancer Care Alliance Division of Medical Oncology University of Washington, Seattle Dr. Cowan[/caption] Andrew J. Cowan, MD Seattle Cancer Care Alliance Division of Medical Oncology University of Washington, Seattle MedicalResearch.com: What is the background for this study? What are the main findings?   Response: Multiple myeloma (MM) is a plasma cell neoplasm with substantial morbidity and mortality. A comprehensive description of the global burden of multiple myeloma is needed to help direct health policy, resource allocation, research, and patient care. Myeloma cases and deaths increased from 1990 to 2016, with middle-income countries contributing the most to this increase. Treatment availability is very limited in countries with low socioeconomic development.
Author Interviews, Infections, Leukemia, MD Anderson, Transplantation / 20.05.2018

MedicalResearch.com Interview with: [caption id="attachment_41862" align="alignleft" width="125"] Dr-Roy F. Chemaly Dr. Chemaly[/caption] Roy F. Chemaly, MD, MPH F.A.C.P., F.I.D.S.A. Department of Infectious Diseases Infection Control and Employee Health Division of Internal Medicine MD Anderson Center MedicalResearch.com: What is the background for this study? What are the main findings? Response: CytomegalovirusCMV infection is a common cause of morbidity and mortality in allo-HCT recipients. Evidence suggests that CMV infection has not only an enormous clinical burden, but a substantial economic burden as well. We conducted this study at MD Anderson to determine the economic and clinical burden of preemptive therapy (PET) for CMV infection. Between 2012 and 2015, 100 consecutive patients hospitalized at our institution for allo-HCT who experienced reactivation of CMV and were treated pre-emptively, were enrolled. The majority of patients were men (55%), who had underlying leukemia (73%), and underwent matched unrelated donor transplant (59%). At the time of hospitalization, most patients had acute GvHD (62%), and were on steroids (58%) within 30 days of CMV reactivation which occurred at a median of 32 days post-HCT (2 -174). A total of 192 episodes of PET occurred in the 100 allo-HCT recipients within 1 year post-HCT. PET consisted of ganciclovir (41%), foscarnet (40%), and valganciclovir (38%). IVIG was also used as adjunct therapy in 20% of episodes. Progression to Cytomegalovirus disease occurred in 4 patients (4%) and mainly affected the GI tract. Mean length of stay for patients treated with ganciclovir or foscarnet was 32 days (2-141) and 41 days (1-177), respectively. The average direct cost per patient admitted for PET was $126,038 ($7,866-$641,841) and the mean cost of CMV antiviral drug per hospitalization was $6,096 for IVIG, $2,410 for foscarnet, $836 for ganciclovir, and $780 for valganciclovir. Serious side effects from PET were observed in 35% of patients on ganciclovir and 12% of patients on foscarnet. Total direct cost per encounter was significantly higher in patients who had serious side effects from foscarnet. All-cause mortality was 59% at 1 year post-transplant.
Author Interviews, Cancer Research, J&J-Janssen, Leukemia / 17.05.2018

MedicalResearch.com Interview with: [caption id="attachment_41804" align="alignleft" width="132"]Andrzej Jakubowiak, MD, PhD Professor of Medicine Director, Myeloma Program University of Chicago Dr. Jakubowiak[/caption] Andrzej Jakubowiak, MD, PhD Professor of Medicine Director, Myeloma Program University of Chicago MedicalResearch.com: What is the background for this announcement? Would you briefly explain what is meant by multiple myeloma?   Response: DARZALEX (daratumumab) in combination with VELCADE (bortezomib), melphalan and prednisone - VMP - received U.S. FDA approval for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT). With this most recent approval, DARZALEX is now the first monoclonal antibody approved for newly diagnosed patients with this disease. Multiple myeloma is an incurable blood cancer that occurs when malignant plasma cells grow uncontrollably in the bone marrow. Despite the introduction of new medicines over the last decade, which has led to significant improvements in outcomes for patients with multiple myeloma, multiple myeloma remains an incurable disease. In 2018, it is estimated that 30,700 people will be diagnosed and 12,770 will die from the disease in the United States.
Author Interviews, Brigham & Women's - Harvard, Infections, Leukemia, Merck, Transplantation / 12.12.2017

MedicalResearch.com Interview with: [caption id="attachment_38873" align="alignleft" width="120"]Francisco M. Marty, M.D Associate Professor, Harvard Medical School Dana–Farber Cancer Institute and Brigham and Women’s Hospital Dr. Marty[/caption] Francisco M. Marty, M.D Associate Professor, Harvard Medical School Dana–Farber Cancer Institute and Brigham and Women’s Hospital MedicalResearch.com: What is the background for this study? What are the main findings? Response: Cytomegalovirus (CMV) is the most common infection in patients who undergo allogeneic hematopoietic-cell transplantation (bone marrow transplantation with cells from donors different than the patient). Up until now, we had no antiviral agent that could be used for prophylaxis (prevention) of CMV post-transplant because of the side effects of drugs available to date (ganciclovir, valganciclovir, foscarnet, cidofovir). This trial confirmed that letermovir was highly effective in preventing CMV infection when used in the first 100 days after allogeneic HCT, was associated with minimal side effects of concern and was also associated with lower all-cause mortality by Week 24 post-HCT.
Author Interviews, JAMA, Leukemia, Transplantation / 19.09.2017

MedicalResearch.com Interview with: Huisheng Ai, MD, Director Department of Hematology and Transplantation, Affiliated Hospital of the Academy  of Military Medical Sciences, Beijing, China  MedicalResearch.com: Which of these results did you find most interesting or surprising? Response: First, we must stress that microtransplant dramatically improved the outcome of older patients with AML. As we know, older AML patients often possess unfavorable prognostic factors, organ dysfunction, and slow post-chemotherapy hematopoietic recovery. Therefore, the general treatment outcome is unsatisfactory even though the incidence is increasing by age with low complete remission (CR) rates (34% to 65%) and poor short-term survival (Two years overall survival was about 11% to 25%). This study involved cases from multiple centers of China, USA and Spain, and found that microtransplant could not only significantly improve complete remission rate in older AML patients among all age groups from 60 to 85, but also improve 1-year and 2-year overall survival and disease free survival especially in patients aged 60 to 75. Second, microtransplant completely overcomes the restriction of HLA typing. The donor could be the patient’s haploidentical family member, or unrelated and fully mismatched one. The incidence of graft-versus-host disease (GVHD) was only 1.1%, even if no any GVHD prevention was given. Other treatment related complications and mortality were also decreased. These results are much better than those of traditional chemotherapy, myeloablative and non-myeloablative transplant, which provides a more safe and effective treatment choice. We are looking forward to seeing the revision of NCCN guideline for older AML to make microtransplant benefit more older patients.
Author Interviews, Leukemia / 03.05.2017

MedicalResearch.com Interview with: [caption id="attachment_34329" align="alignleft" width="160"]Martin J.S. Dyer, D.Phil MA FRCP FRCPath Ernest and Helen Scott Haematological Research Institute University of Leicester, UK Prof. Dyer[/caption] Martin J.S. Dyer, D.Phil MA FRCP FRCPath Ernest and Helen Scott Haematological Research Institute University of Leicester, UK MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study follows on from a world-first clinical trial of a new drug to treat particular blood cancers. Results of that international clinical trial were published in the journal Blood in November 2015 and looked at the efficacy of a new inhibitor, ONO/GS-4059, in the treatment of CLL and Non-Hodgkin Lymphoma patients, refractory or resistant to current chemotherapies. ONO/GS-4059 targets BTK, a protein essential for the survival and proliferation of the tumour cells. The study opened in January 2012 and 90 patients were enrolled in different centres in the UK and in France, with 28 coming from Leicester. Patients with CLL showed the best response and most of them were still on the study after 3 years, and remarkably without notable toxicities. In the new paper, we are reporting the long-term follow-up results. This work, published in the journal Blood, was funded by the Ernest and Helen Scott Haematological Research Institute, ONO Pharmaceuticals, Gilead Pharmaceuticals and the Cancer Research UK Leicester Experimental Cancer Medicine Centre. Local charity Hope Against Cancer fund the Clinical Trials Facility based at the Leicester Royal Infirmary. This current paper describes the long term follow up and shows that in patients with CLL the remissions are durable and associated with no new toxicities. Furthermore, in collaboration with Sistemas Genomicos, a company in Valencia, we have shown that mutations associated with aggressive disease respond well to treatment with ONO/GS-4059.
Author Interviews, Leukemia, Nature, Pediatrics, UT Southwestern, Weight Research / 13.12.2016

MedicalResearch.com Interview with: [caption id="attachment_30486" align="alignleft" width="148"]Chengcheng (Alec) Zhang, Ph.D. Associate Professor Hortense L. and Morton H. Sanger Professorship in Oncology Michael L. Rosenberg Scholar for Medical Research Department of Physiology UT Southwestern Medical Center Dr. Alec Zhang[/caption] Chengcheng (Alec) Zhang, Ph.D. Associate Professor Hortense L. and Morton H. Sanger Professorship in Oncology Michael L. Rosenberg Scholar for Medical Research Department of Physiology UT Southwestern Medical Center MedicalResearch.com: What is the background for this study? Response: New therapeutic targets and approaches are needed to effectively treat leukemia. Acute myeloid leukemia (AML) is the most common form of adult acute leukemia whereas acute lymphoblastic leukemia (ALL) is the most common form of cancer in children; ALL also occurs in adults. Although treatment of pediatric ALL is highly effective, a sizeable number of patients are non-responders who succumb to this disease. The outcome of ALL in adults is significantly worse than for pediatric ALL. Additionally, some types of ALL have a much poorer prognosis than others. Dietary restriction, including fasting, delays aging and has prolonged effects in a wide range of organisms and has been considered for cancer prevention. In certain types of solid tumor,_ENREF_1 dietary restriction regimens are able to promote T cell-mediated tumor cytotoxicity and enhance anticancer immunosurveillance, and coordinate with chemotherapy to promote the anti-cancer effects. However, the responsiveness of hematopoietic malignancies to dietary restriction, including fasting, remains unknown. Furthermore, whether dietary restriction alone can inhibit cancer development is not clear.
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