This type of leukemia, also called acute myelogenous leukemia, acute myelocytic leukemia, or acute nonlymphocytic leukemia, accounts for most of the remaining cases of childhood leukemia. Which require germline genetic factors and tumor associated somatic. Precision medicine, personalized medicine, or stratified medicine over the past 20 years, different terms have been used to describe individualization of therapy, and in the last 810 years, the preferred term has shifted from personalized medicine to precision medicine, as shown in figure 1. The growing role of precision and personalized medicine. Despite the use of intensive chemotherapy, relapse remains the most common cause for therapeutic failure in acute myelogenous leukemia aml.
Precision medicine in the treatment stratification of aml patients. Genetic variation behind acute myeloid leukemia treatment. Will personalized medicine mean higher costs for consumers. Recent advances in molecular technology have unraveled the complexity of leukemogenesis and provided the opportunity to design more personalized and pathophysiologytargeted therapeutic strategies. The terms personalized medicine, precision medicine, stratified medicine and p4 medicine are used. Therapeutic options for acute myeloid leukemia aml have remained unchanged for nearly the past 5 decades, with cytarabine and anthracyclines and use of hypomethylating agents for less intensive therapy. More sophisticated diagnostic procedures, including immunophenotyping, cytogenetics, molecular genetics, and new genomics, have allowed the definition of new all subentities which, in some cases, has translated into specific therapies. The drug names link to ncis cancer drug information summaries. Acute myeloid leukemia aml is a hematological malignancy characterized by uncontrolled proliferation, differentiation arrest, and accumulation of immature myeloid progenitors. Bachas and colleagues assessed the mutational status of flt3, nraskras, kit, wt1, cebp.
Personalized medicine in adult acute lymphoblastic leukemia. One step closer to personalized medicine for leukemia. Many different factors may contribute to differential response including variable age and body size, diet, gastrointestinal absorption, compliance with therapy and characteristics of the drug target, e. Assi et al, subtypespecific regulatory network rewiring in acute myeloid leukemia, nature genetics 2018. Acute myeloid leukemia aml is characterized by blocked myeloidlineage differentiation and accumulation of myeloid blast cells in the bonemarrow that results in catastrophic bonemarrow failure. Epigenetic landscape of acute myelogenous leukemiamoving. Aml starts from the myeloid cells that normally form white blood cells other than lymphocytes, red blood cells, or platelets. Acute lymphocytic leukemia early detection, diagnosis, and. Pharmacogenetics, pharmacogenomics and personalized. This page lists cancer drugs approved by the fda for use in leukemia. Optimizing combination therapy for acute lymphoblastic. Data sources include ibm watson micromedex updated 4 may 2020, cerner multum updated 4 may 2020, wolters kluwer updated.
Knowledge concerning acute lymphoblastic leukemia all has increased greatly, 1 and personalized medicine has become a reality. In appreciation of this concept, the beat aml master trial, led by the leukemia and lymphoma society in collaboration with several academic centres and the pharmaceutical industry, offers the hope to substantially boost the paradigm of personalized medicine in aml by utilizing companion biomarker. Implementation of largescale genomic studies in the past decade has unraveled the genetic landscape and molecular etiology of aml. Other terms such as individualized medicine, precision medicine, sequential medicine, stratified medicine and. New leukemia drug is more effective and easier to use. It occurs most commonly in adults above 55 years and children younger than 15 years. Personalized medicine does not have a unique definition. Despite advances in research and the development of effective treatment regimens, cll is still largely an incurable disease. Leukemia starts in the bone marrow, so checking the bone marrow for leukemia cells is a key part of testing for it. Drugs approved for leukemia national cancer institute. But now, because of personalized medicine, pathologists have a new and important role to play. The national cancer institute estimates 33,990 people in the united states are living with cml, with. With the advent of increased genome sequencing efforts and phenotypic characterization, opportunities for personalized treatment strategies are rapidly emerging. Learn how precision medicine uses genes, lifestyle habits, and other things to more accurately target treatment to each.
The list includes generic names, brand names, and common drug combinations, which are shown in capital letters. The national cancer institute defines personalized medicine as a form of medicine that uses information about a persons genes, proteins, and environment to prevent, diagnose, and treat disease. Personalized medicine, sometimes referred to as precision or. Pdf a personalized approach to acute myeloid leukemia. Personalized medicine coalition advocates for precision. Diagnostic braf v600e mutation in hairy cell leukemia prognostic tp53 mutations in mds therapeutics mutations that can be targeted flt3 itd and midastaurin. Smith is a professor in the division of ematologyoncology, department of. Mrd is the most important prognostic factor and thus a major component of a personalized treatment algorithm. Although clinical advances in aml have been made, especially in young patients, longterm diseasefree survival remains poor, making this disease an unmet therapeutic challenge. The centers for disease control and prevention covers precision medicine in its.
Personalized medicine is often described as genomicsbased knowledge that promises the ability to approach each patient as the biological individual he or she is 1 1. Clinical application of personalized medicine for acute. Chronic myeloid leukemia cml, also known as chronic myelogenous leukemia, is a type of cancer that starts in the bloodforming cells of the bone marrow and invades the blood. There are a number of different tests that pathologists can perform to learn more about the genetics of a tumor and the subclass it belongs to.
Information is available about precision medicine as datadriven treatment information about genetics home reference and precision medicine is available from the interactive journal of medical research ijmr article, information needs in the precision medicine era. The beginning of leukemia may be sudden acute or slow and gradual chronic. A personalized approach to acute myeloid leukemia therapy. During that time, sequencing technology evolved from the manual sanger method. Treatment of cll involves targeting key pathways involved in the pathogenesis of bcell. Leukemia is a cancer of blood or bloodforming cells and therefore sometimes referred to as blood cancer while the exact causes of leukemia is not known, risk factors have been identified, including radiation exposure, certain chemotherapy for cancer, smoking, family history of leukemia, and exposure to certain chemicals such as benzene common symptoms of chronic or acute leukemia may. Much work is ongoing to better understand and treat this group of diseases. First targeted therapies for lung cancer, leukemia, melanoma, cystic fibrosis. Aml accounts for about 25% of all leukemias and it is the most common acute leukemia in adultsit is a fastgrowing cancer with an aggressive clinical course walter, 2011. The rocky road to personalized medicine in acute myeloid. The interactions between leukemia stem cells lsc and. Clinicians may be closer to applying personalized medicine to the treatment of acute myeloid leukemia aml, according to findings from the ongoing beat aml master trial.
The american cancer society estimates that there will be approximately 18,860 new cases of aml and 10,460 deaths from aml in the united states in 2014 siegel et al. Personalized medicine applied to aml cancer discovery. The design of personalized health care is based on prevention or therapeutic approaches in conjunction with current knowledge of the cancer type. The treatment methods used depend on the blood cell affected and whether the leukemia is acute or chronic or lymphocytic or myelogenous. In this study, newly diagnosed patients are genomically sequenced, and the results are used to quickly assign firstline therapies. Although personalized medicine has been used in a number of cancers, we have selected few cancers below where incidence and prevalence of cancer is high in us and more data is available. Companion diagnostics in leukemia the importance of. Approximately 2,700 children are diagnosed in the united states each year. Genomic and personalized medicine, second edition winner of a 20 highly commended bma medical book award for medicine is a major discussion of the structure, history, and applications of the field, as it emerges from the campus and lab into clinical action.
This material is provided for educational purposes only and is not intended for medical advice, diagnosis or treatment. Chronic lymphocytic leukemia cll is the most common hematologic malignancy in the western hemisphere. Recent advances in high throughput technologies have led to the generation of vast amounts of clinical data and the development of personalized medicine approaches in acute myeloid leukemia aml. Personalized medicine in adult acute lymphoblastic leukemia ncbi.
The four broad subtypes most likely to be encountered by primary care physicians are acute lymphoblastic, acute. How is precision medicine different from traditional medicine. In 2018 alone, there will be an estimated 1,735,350 new diagnoses and 609,640 cancerrelated deaths 1. Examples of personalized medicine in different cancers. This is an appealing description, yet unless clinical, social, and environmental features that affect the outcomes of disease are also incorporated, the current approach may be carving a path to depersonalized medicine. The costeffectiveness of personalized medicine strategies in acute. Detect deletion of the lsi egr1 probe target on chromosome 5q in bone marrow specimens and to be used, in addition to cytogenetics, other biomarkers, morphology and other clinical information, at the time of acute myeloid leukemia aml diagnosis as an aid in determining prognosis. Research brings personalized medicine to treat leukemia. Acute myeloid leukemia aml is the most common acute leukemia in adults and is a major challenge in terms of diagnosis, care, followup and therapy. Treatment for pediatric all typically uses combination chemotherapy in phases, including a prolonged maintenance phase with oral methotrexate and 6mercaptopurine, which often requires dose adjustment to balance side effects and efficacy. One step closer to personalized medicine for leukemia oncology times. Cancer is one of the leading causes of death in the united states. Personalized medicine and treatments for rare diseases many of which are extraordinarily expensive are a growing part of the u.
Personalized medicine is empowering because your personal genetic and other predictive information allows you to take action that is specific for yourather than the zone size fits all approach. With advances in genomicsequencing efforts, epigenetic and phenotypic characterization, personalized therapeutic strategies aimed at critical leukemia survival mechanisms may. Present and future of personalized medicine in cll. Bone marrow samples are obtained by bone marrow aspiration and biopsy tests usually done at the same time.
Personalized medicine, precision medicine, or theranostics is a medical model that separates people into different groupswith medical decisions, practices, interventions andor products being tailored to the individual patient based on their predicted response or risk of disease. Food and drug administration approved ibrutinib brand name, imbruvica for treatment of cll. Acute myeloid leukemia aml is a specific type of leukemia which is characterized. Leukemia can be acute or chronic and lymphocytic or myelogenous. Acute lymphoblastic leukemia all is a blood cancer that is characterized by the overproduction of lymphoblasts in the bone marrow.
Traditional medicine focuses on treating disease in big groups of people. For example, aml patients whose leukemia cells have specific gene mutations may benefit from advancements in personalized medicine. Genetic testing and the implications for future therapies dr. This information is feeding the field of personalized medicine, which continues to evolve, as pharmaceutical companies work to develop and launch targeted therapies for oncology patients. Leukemia is a clonal proliferation of hematopoietic stem cells in the bone marrow. Personalized medicine for aml picking the right drug or correctly characterizing the malignant clone, and picking the right treatment for the right patient at the right time this process requires monitoring for changes in disease burden and biology over time. Precision medicine also referred to as personalized medicine or. Differential response to the same drug in different patients is a common clinical experience. Small molecule inhibitors for the treatment of chronic. Francis collins, md, phd, director, national institutes of health. Personalised medicine pm, treatment based on the patients unique characteristics, is relatively new in medical society. Shift the emphasis in medicine from reaction to prevention personalized medicine introduces the ability to use molecular markers that signal disease risk or presence before clinical signs and symptoms appear, and it offers the opportunity to focus on prevention and early intervention rather than on reaction at advanced stages of disease.
598 1321 763 1223 887 484 308 542 378 32 1019 1343 633 1462 839 1513 640 59 716 1016 974 1479 338 474 885 1073 694 244 784 255 347 539 1296 1326 1452 478 411