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Dissecting the role of a key epigenetic modulator in Mixed Lineage Leukemia
We study how a protein called Dpy30 controls blood cancers by regulating chromatin, the physical structure where our genes reside. We study how this protein controls addition of a specific chemical group onto chromatin, thereby regulating expression of genes for leukemia in cells and animals. We are also developing chemicals to inhibit Dpy30’s activity in leukemia. We hope to better understand the role of Dpy30 in leukemia and identify Dyp30-inhibiting chemicals for leukemia treatment.
FDA Approves New Maintenance Treatment for AML
Some promising news to kick off Blood Cancer Awareness Month – on September 1, 2020, the U.S. Food and Drug Administration approved a new oral maintenance treatment for certain adult patients with acute myeloid leukemia (AML).
CC-486 (Onureg®) is now approved for the continued treatment of adult patients with AML who achieved first complete remission (CR) or CR with incomplete blood count recovery (Cri) following intensive induction chemotherapy and who are not able to undergo intensive curative therapy, such as hematopoietic stem cell transplant.
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.Imatinib mesylate
Imatinib mesylate is FDA approved to treat
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.Remembering Cancer with a Tattoo, New Career and Anniversary Party
In Jon Christoffersen’s house, important occasions - such as the end of chemotherapy - are worthy of great celebration. First it was a tattoo to mark the beginning and end of his treatment, and now, on his 10-year anniversary of being cancer free, it’s time to party.
New Targeted Therapies for Pediatric Acute Myeloid Leukemia
Our research focuses on the preclinical evaluation of new targeted therapies for high-risk subtypes of childhood AML. We are deploying screening approaches to delete each gene, one-by-one, to identify genes whose deletion leads to death of the leukemia cells. We will evaluate drugs developed against these targets in state-of-the-art models of pediatric AML. Our goal is to translate the most promising findings to clinical trials for children with these very poor outcome subsets of AML.Families Fighting for Cancer Cures: Generation LLS
As an organization founded by a family for families, The Leukemia & Lymphoma Society (LLS) is celebrating 70 years of impact on generations of families, researchers and volunteers while looking to the future by redoubling its commitment to improve treatments and care for children with cancer.
Looking Ahead: LLS Panel Highlights Trends and Advances
The Leukemia & Lymphoma Society recently brought together a world-renowned panel of thought leaders to discuss the latest advances and insights into blood cancer research and treatment, drug costs and patient access, and patient advocacy and policy.
“There’s never a good time to get cancer but this is a phenomenal time to be fighting it,” said Louis J. DeGennaro, president and CEO of The Leukemia & Lymphoma Society (LLS).
Cytarabine
Cytarabine is FDA approved to prevent and treat leukemia that has spread to the meninges (the three membranes that cover the brain and spinal cord). It is also FDA approved for use with other drugs to treat acute myeloid leukemia, acute lymphoblastic leukemia and chronic myeloid leukemia. This medicine may cause a temporary loss of hair in some people. After treatment with cytarabine has ended, normal hair growth should return.
Liposomal cytarabine (DepoCyt®) is given by intrathecal injection. It is FDA approved to treat lymphoma that has spread to the meninges.
B-Cell Prolymphocytic Leukemia (B-PLL)
B-cell prolymphocytic leukemia (B-PLL) is a very rare and typically aggressive malignancy (cancer) characterized by the out of control growth of B-cells (B-lymphocytes). B-cells are a type of white blood cell that is part of the immune system. B-PLL usually affects older adults with a median age at diagnosis of 69 years, and it is slightly more common in men than women. Most of the time, B-PLL occurs as a transformation or evolution of a more slow-growing B-cell cancer, such as chronic lymphocytic leukemia. Rarely, this is a primary disorder.
Targeting Leukemia Stem Cells with the Novel Antibody Drug Conjugate
To improve the cure rate of patients suffering from acute myeloid leukemia (AML), our study aims to target resistant leukemia stem cells by developing an 'antibody-drug conjugate' (ADC) against CD99, a protein expressed on these cells. Initial tests of two ADC versions have shown promise in combating AML. Our next steps involve refining the anti-CD99 antibody, identifying the optimal drug for conjugation, and testing the ADC on patient-derived leukemia models.T-Cell Prolymphocytic Leukemia (T-PLL)
T-cell prolymphocytic leukemia (T-PLL) is an extremely rare and typically aggressive malignancy (cancer) that is characterized by the out of control growth of mature T-cells (T-lymphocytes). T-cells are a type of white blood cell that protects the body from infections. T-PLL affects older adults with a median age at diagnosis of 61 years, and it is more common in men than in women.
Targeting AML
Not for lack of trying, we’ve seen very little progress over the past 40 years in treating acute myeloid leukemia (AML), an aggressive and deadly blood cancer. But we’re finally starting to see the needle move and The Leukemia & Lymphoma Society (LLS) is playing a leading role in going on the offensive against the disease.
Molecular basis and new therapeutic strategies in lineage ambiguous leukemia
Lineage-ambiguous leukemias are high-risk blood cancers with unclear biologic basis and suboptimal treatment options. Here, I will identify the cell of origin of lineage ambiguous leukemia and investigate new therapeutic strategies through in vitro and in vivo experimental modeling approaches and preclinical drug studies in patient-derived xenografts. These studies will clarify the cellular and molecular alterations driving lineage ambiguity and advance a new, rational therapeutic approach.Blinatumomab
Blinatumomab is FDA approved for the treatment of adult and pediatric patients one month and older with:
Accelerating Care and Cures for Children
Kaitlyn's Story
Eight-year-old Kaitlyn Johnson has had to deal with cancer for the majority of her young life. In fact, she was only 18 months old when she was diagnosed with acute lymphoblastic leukemia (ALL), the most common cancer among children and young adults. Her young life was quickly consumed with cancer treatments, doctors’ visits, and hospital stays. Throughout this time, Kaitlyn was brave, positive and continued to pursue her passion for dancing – no matter the location.
Understanding the role of Metabolic Regulator SIRT5 in Acute Lymphoblastic Leukemia
SIRT5 is a master regulator of central energy metabolism. The survival and growth of Acute Myeloid Leukemia (AML) cells depend on SIRT5. I will employ genetic SIRT5 disruption and small molecule inhibitors to target SIRT5 in Acute Lymphoblastic Leukemia (ALL) cells and primary samples. This study aims to 1) determine the effects of SIRT5 inhibition on ALL in vitro and in vivo, and 2) identify SIRT5-regulated pathways and mechanisms underlying SIRT5 dependency in T-ALL.Diagnosed with a Blood Cancer? Important Questions You Might Not Think to Ask
What is my actual diagnosis? Find out your exact diagnosis. Ask your doctor to write down the exact name of your sub-type and take the paper with you. For example, knowing you have “a B-cell lymphoma” isn’t good enough. Follicular and diffuse large B-cell are both B-cell lymphomas but with very different prognoses and treatment plans. Leukemia also has different sub-types. Knowing your specific sub-type helps you understand what disease you are dealing with, how aggressive it is, and what to expect from treatment.
The Bright Light (Actually Two!) at the End of the Tunnel
I was 28, married for two years, and my husband and I had just celebrated our one-year anniversary at my business, Gigi’s Cupcakes. We were happy and healthy and ready to start thinking about having a family. Life was perfect!
Panelists Discuss the AML Patient Experience
What is AML? What research is underway? And what resources are available to patients?
The Leukemia & Lymphoma Society recently joined up with Patient Power and the MDS Alliance to host an AML Awareness Day and answer those questions.
The April 21 webcast was moderated by Carol Preston, host of Patient Power, an online portal offering cancer information for both patients and professionals. Preston is also a chronic lymphocytic leukemia (CLL) survivor.
Investigational CLL Therapy to Get Priority Review by FDA
Marking another promising advance for the treatment of patients with chronic lymphocytic leukemia, including those with a rare subset of this blood cancer, the U.S. Food and Drug Administration (FDA) has granted priority review for an investigational compound that has shown positive results in a Phase II clinical trial.