Partner Therapeutics Announces Publication of a Retrospective Cohort Review of 15 LEUKINE Treated Patients with Pediatric Malignancies and Invasive Fungal Disease Refractory to Antifungal Therapy
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Pediatric
LEXINGTON, Mass., Dec. 15, 2022 /PRNewswire/ -- Partner Therapeutics, Inc. (PTx) announced publication of a single institution retrospective cohort review of 15 patients with pediatric malignancies and invasive fungal diseases that were refractory to antifungal therapy and treated adjunctively with Leukine (sargramostim; yeast-derived, glycosylated recombinant human granulocyte-macrophage colony-stimulating factor [rhu GM-CSF]).1 Additionally, the publication included a systematic review of 50 published reports of rhu GM-CSF use for invasive fungal disease. Open Forum Infectious Diseases published the manuscript entitled "Recombinant Human Granulocyte-Macrophage Colony-Stimulating Factor (rhu GM-CSF) as Adjuvant Therapy for Invasive Fungal Diseases." Leukine is not approved to treat invasive fungal disease.
The authors conclude: "Sargramostim may be a potential adjunctive immunomodulator for selected patients with hematological malignancies and refractory [invasive fungal diseases] IFDs." The overall response rate for sargramostim use in the retrospective cohort was 92%. 80% of patients were refractory to antifungal therapy prior to sargramostim use. The infections were caused by Candida spp., Trichosporon sp., and molds (Aspergillus spp., Scedosporium sp), as well as the mucormycosis-causing pathogens Rhizopus sp., Lichtheimia sp.
Endogenous GM-CSF functions as a hematopoietic growth factor while also regulating the broader immune system via activation of macrophages, monocytes, and neutrophils.2 GM-CSF enhances macrophage activities such as pathogen killing and clearance as well as neutrophil fungicidal activity.3 Based on the mechanism of endogenous GM-CSF, clinicians have utilized Leukine adjunctively in treating infections, as summarized in the publication.1
The research was led by Thomas J. Walsh, MD, PhD (Hon), FIDSA, FAAM, FECMM, of the Transplantation-Oncology Infectious Disease Program at Weill Cornell Medicine and Center for Innovative Therapeutics and Diagnostics, and Tempe K. Chen, MD, of MemorialCare Miller Children's and Women's Hospital Long Beach and Division of Infectious Diseases at University of California Irvine School of Medicine. Dr. Walsh shared the significance of this work: "Building upon laboratory investigations of GM-CSF and pathogenic fungi that we have conducted; this paper is especially important as the translational evidence of sargramostim's efficacy in managing invasive fungal disease in immunocompromised pediatric and adult patients. The paper imparts clinical significance to more recent preclinical data supporting the role of GM-CSF in mucosal host defenses and macrophage bioenergetics."
Bioenergetics refers to how cells transform energy. GM-CSF signaling regulates mitochondrial bioenergetics critical to macrophage function including energy utilization, cellular metabolism, and expression of mitochondrial proteins.
Dr. Mahmoud Ghannoum, Director of Integrated Microbiome Core and Center for Medical Mycology at Case Western Reserve University School of Medicine and University Hospitals Cleveland Medical Center, added: "A host directed therapy such as sargramostim (Rh GM-CSF) is a promising approach to treatment of intracellular pathogens, especially in the immune compromised patient. By attempting to restore the patient's immune processes physicians may have a new tool to combat the development of antifungal resistance." This October, the World Health Organization (WHO) Antimicrobial Resistance Division issued a report to focus and drive policy interventions to strengthen the response to fungal infections with antifungal resistance. The pathogens were ranked into three priority groups: critical, high, and medium. All of the pathogens treated with Leukine in this review were due to pathogens classified as critical or high threats due to their distribution and antifungal resistance patterns.
PTx has a broad development program in place for Leukine including clinical investigations for the treatment of infectious diseases.
References
ABOUT LEUKINE
LEUKINE (sargramostim) is a glycosylated recombinant human granulocyte-macrophage colony-stimulating factor (rhu GM-CSF) produced by recombinant DNA technology in yeast.
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Adverse events occurring in >10% of patients receiving LEUKINE in controlled clinical trials and reported at a higher frequency than in placebo patients are:
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Partner Therapeutics, Inc. (PTx), an integrated biotechnology company, focuses on development and commercialization of late-stage therapeutics to improve health outcomes in treatment of cancer and other serious diseases. The company believes in delivering products and supporting medical teams with the purpose of achieving superior outcomes for patients and their families. Visit www.partnertx.com
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