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Targeting cathepsin G in acute myeloid leukemia

We developed a chimeric antigen receptor (CAR) targeting an epitope of the myeloid associated antigen cathepsin G that is processed and presented in the contest of the MHC complex in myeloid leukemic cells. T cells expressing the cathepsin G specific CAR (CG1.CAR) recognize HLA-A2+ myeloid target cells expressing cathepsin G. We intend to study efficacy and safety of CG1.CAR-T cells in preclinical models in preparation of a phase I clinical study in patients with relapsed/refractory AML.

Role of HLX in leukemia induction and maintenance

We and others have shown how HLX overexpression keeps blood cells more immature by blocking their differentiation and promoting their proliferation, a characteristic which is inherent to AML. However, whether there is a causative role of HLX in the induction of AML is still unclear. Hence, the aim of my study is to better understand, using genetically engineered mice models, retroviral models, and human AML patient samples, how HLX drives AML at molecular level. This study will uncover potential therapeutic strategies for AML treatment in future.

T-Cell Leukemia Lymphoma Foundation

To provide education and support to patients and to encourage research into these rare cancers

  • Provides disease, treatment and clinical trial information for the T-cell leukemias and lymphomas
  • Organizes free educational forums
  • Offers physician referrals, e-newsletter and links to helpful resources
  • Supports scientific research on the T-cell leukemias and lymphomas.

Patients, caregivers, healthcare professionals, researchers

Website or 206-661-2253 http://www.tcllfoundation.org/

Acute Leukemia Advocates Network (ALAN)

To change the outcomes of acute leukemia patients by strengthening patient advocacy.

  • ALAN is an independent global network of patient organizations which aims to increase awareness about the acute leukemias, share best practices, advocate for better access and treatment, and improve education for healthcare professionals.

Healthcare professionals, patient advocates

Website http://acuteleuk.org/
International Resources
Canadian Resources International Resources U.S. Resources

Children’s Leukemia Research Association (CLRA)

To support research efforts towards finding the causes and cure for leukemia.

  • Patient Aid Program offers limited funds (up to $3,000 per year) to assist with certain treatments, services and prescriptions; funds are available on a first-come, first-serve basis
  • CLRA funds grants to doctors conducting promising research about leukemia.

Children and adults with blood cancer in the U.S., healthcare professionals and researchers

516-222-1944 http://www.childrensleukemia.org/
International Resources
Canadian Resources U.S. Resources

Acute Myeloid Leukemia 3D Model

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This model contains the following chapters. Click the "Interact in 3D" button to begin.

  • Healthy Bone Marrow
  • Normal Blood Cell Production
  • Proliferation of Cells and the Crowding Out of Normal Cells
  • Signs and Symptoms

Myles, Reese, Aaron, and Lydia's images placed together in a collage

Moving Forward: 4 Young Blood Cancer Survivors Look Ahead

Blood cancer survivors have a unique path ahead of them—and that can feel challenging.  

Questions come up: Does treatment mean I have to pause everything? Do I tell my friends or coworkers (and how)? With my medical bills, how can I afford the education I want?  

Eight Years and Counting: "The Pill"

I never would have imagined that I could stave off a deadly blood cancer by taking a pill. Yet that's exactly what I -- and thousands of others with chronic myeloid leukemia (CML) -- do every day. That little orange tablet known as Gleevec (imatinib) has led us back to normalcy. As a breast cancer survivor with many friends and family members waging their own cancer battles, I only wish there were more solutions this simple -- and this hopeful.

Achieving the Impossible: What’s ahead in 2018?

We’ve just concluded one of the most historic years on record in terms of new therapy approvals for blood cancers. In all, the U.S. Food and Drug Administration (FDA) approved 18 therapies to treat patients with blood cancers, including some entirely new agents and some new uses for already approved drugs.

Understanding Leukemia in Children with Down Syndrome to Develop Better Therapies

This Specialized Center of Research is focused on identifying the contributions of chromosome 21, which is present in three copies in individuals with Down syndrome (DS), to acute leukemia. Children with DS are at a 20-fold increased risk of leukemia compared to the overall pediatric population and frequently have other health issues that complicate leukemia treatment.

Mechanisms of oncogenic transcription in NPM1-mutant myeloid leukemia

NPM1-mutated leukemia is the most common AML in adult and characterized by upregulations of HOXA/B genes and MEIS1. Given the importance of oncogenic transcriptional program, I will determine regulatory molecules that cooperate with mutant NPM1 on chromatin by combining CRISPR/Cas9 screening approach in an innovative model system of endogenous transcription reporters with proteomics approach. This will facilitate identification of novel therapeutic targets specific for NPM1-mutated AML.

Discovering the function and targeting dysregulated nuclear condensates in myeloid leukemia

Although molecular targeted therapy has dramatically changed how we treat cancer, the treatment for acute myeloid leukemia (AML) remains focused on the use of cytotoxic drugs with many patients eventually relapsing with their disease. Our studies have a uncovered a new nuclear structure that is dysregulated in myeloid leukemia. This proposal studies the identity and function of this nuclear body in human AML and strives to identify novel therapeutic strategies and targets in leukemia.