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A phase 1b/2 study targeting apoptotic and signaling pathways in T-acute lymphoblastic leukemia

T-acute lymphoblastic leukemia (T-ALL) is an aggressive leukemia with limited treatment options after first-line chemotherapy. Our preclinical work in animal models of T-ALL demonstrated the activity of a novel-novel combination treatment strategy, which includes LP-118 (activator of suicide pathways within leukemic cells) and tyrosine kinase inhibitors (inhibiting growth-promoting LCK and ACK1 signaling pathways).

Meet an LLS Advocacy Volunteer: Amanda Steffy

Amanda Steffy of Pasadena, Calif. is one of The Leukemia & Lymphoma Society’s most passionate policy advocates. She frequently travels to Washington, D.C. to speak with lawmakers about the importance of high-quality, affordable healthcare for cancer patients. In California, where she works as an instrument systems engineer for NASA JPL, she recruits and teaches others to do the same.

This May, Steffy joins LLS for its first-ever virtual advocacy event. In this interview, Steffy explains how she first became involved in advocacy and why she’s so passionate about the work.

DEVELOPING RELATIONSHIPS ON CAPITOL HILL

As the voice for all blood cancer patients, The Leukemia & Lymphoma Society (LLS) establishes and grows meaningful relationships with policymakers

 

The Leukemia & Lymphoma Society is committed to removing barriers to care for blood cancer patients, and works with federal and state legislators to advocate for policies to ensure patients have access to quality, affordable care and treatments they need.

Here’s an inside look at how we cultivate these key connections:

211Astatine-CD123 Radioimmunotherapy for Cancer (Stem) Cell-Directed Treatment of Acute Leukemia

Because acute leukemias are very sensitive to radiation, radioisotopes are ideal payloads to arm antibodies against these difficult-to-cure, aggressive blood cancers. Here, we will develop fully human anti-CD123 antibodies carrying the highly potent alpha-emitter astatine-211 (211At) as a new therapy for acute leukemia.

Forbes Women’s Summit 2019: LLS’s Chief Medical Officer Joins Women In Leading the Way

More than 400 women from diverse backgrounds in business, entertainment, fashion, media, healthcare, science and finance came together this week to inspire and encourage one another at the 2019 Forbes Women’s Summit at Pier 60 Chelsea Piers in New York City.

The attendees engaged in animated discussions about how women are changing the world while still fighting to overcome obstacles they face in the workforce and on the world stage.

Targeting the interaction of leukemia stem cells with their niche to treat myelofibrosis

Bone marrow scar formation (fibrosis) is a hallmark of myelofibrosis and contributes significantly to the disease progression. We use mouse genetics to model myelofibrosis and understand the cellular and molecular makeup of the diseased microenvironment. We aim to understand the composition and alteration of the bone marrow microenvironment in myelofibrosis. This may provide novel therapeutic targets for myelofibrosis.

Therapeutic exploitation of novel mouse models and metabolic interventions in leukemia

Our research program aims to gain a deeper understanding of the pathobiology of T-ALL and HSTL. To this end, we will use novel mouse models, cutting-edge techniques and comprehensive genetic, pharmacological and metabolic interventions. In addition, we will perform unbiased experiments to identify novel therapeutic targets. Our goal is to uncover new tools and targets for the treatment of T-ALL and HSTL, which could be used for the benefit of patients in the short/mid-term.

Niclosamide for the Treatment of Relapsed/Refractory Pediatric Acute Myeloid Leukemia

Niclosamide is an FDA approved anti-parasitic drug that is well tolerated and acts synergistically with chemotherapy to kill AML cells. We will conduct a Phase I clinical trial with niclosamide in combination with cytarabine for children with relapsed/refractory pediatric AML. ShRNA/CRISPR screens demonstrated that Bcl-2 is upregulated in niclosamide resistant cells. We will study the effects of the Bcl-2 inhibitor venetoclax in combination with niclosamide in pediatric AML.

Cotargeting oncogenic protein translation and apoptosis in acute myeloid leukemia

The focus of my research is to evaluate the efficacy of and to unravel the molecular mechanisms underpinning a novel drug combination in AML targeting oncogenic protein translation and apoptosis. We will utilize genetic perturbation and other orthogonal approaches, including in vitro and ex vivo assays, and in vivo AML PDX models. The goal of my research is to transform the clinical management of AML patients, particularly for relapsed and difficult-to-treat subgroups.