The ARCH program builds upon the ASH Bridge Grant, developed in 2012 in response to federal research fund cuts. This year\u2019s award cohort is the largest yet, sustaining critical hematology research amid an uncertain federal funding landscape.<\/p>\n
\u201cThe ARCH award will give investigators the resources to stay funded and continue their essential research in a climate where investigators are now having to fill broader and more frequent gaps in funding,\u201d\u00a0ARCH Study Section at ASH, Kellie Machlus, PhD,\u00a0of Boston Children\u2019s Hospital, previously told Heme Today.<\/a><\/p>\n The ASH ARCH award expands the ASH Bridge Grant\u2019s eligibility criteria by removing the NIH R01 application requirement. This allows investigators of all career levels to apply for the award, according to Dr. Machlus.<\/p>\n \u201cThe ASH ARCH funding mechanism was designed to make research in hematology more accessible to investigators like myself hoping to make impactful translational discoveries that have the potential to improve outcomes for patients with blood cancers,\u201d Sheng Cai, MD, PhD, ARCH program awardee and Leukemia Specialist & Early Drug Development Specialist at the Memorial Sloan Kettering Cancer Center, told Heme Today.<\/p>\n Dr. Cai is researching concurrent menin inhibition and Ikaros degradation to overcome resistance mutations in the MEN1 gene for patients with relapsed acute leukemia. A phase 1\/2 trial will investigate dual targeting of acute leukemia with revumenib and mezigdomide, which targets the Ikaros protein.<\/p>\n \u201cThe strategy we are testing is to attack the same leukemia through a second, independent point of vulnerability at the same time,\u201d Dr. Cai explained. \u201cThe ARCH award supports the analyses of the bio specimens collected during this trial. We will assess blood and bone marrow throughout treatment and use assays sensitive enough to detect a single resistant cell among roughly 100,000.\u201d<\/p>\n \u201cReceiving the ASH ARCH grant is a tremendous honor and a meaningful validation of our lab\u2019s ongoing work,\u201d added Carol Miao, PhD, a professor of pediatrics at Seattle Children\u2019s Research Institute. \u201cBeing recognized by the American Society of Hematology\u2014a community of leading experts and peers in hematology\u2014underscores the importance and potential impact of our research direction.\u201d<\/p>\n The ASH ARCH grant is funding Dr. Miao\u2019s research on redosable nonviral gene therapy for patients with hemophilia A. Her research aims to overcome limitations of current gene therapies, such as vector immunity and declining therapeutic factor levels.<\/p>\n \u201cWe are evaluating nonviral platforms, specifically utilizing ultrasound-mediated gene delivery, lipid nanoparticles, and a novel combination of both technologies. Our goal is to establish an efficient, targeted, and repeatable platform for factor VIII gene delivery,\u201d Dr. Miao told Heme Today. \u201cBy developing a redosable platform, we hope to make gene therapy more accessible, customizable across a patient\u2019s lifespan, and safer, ultimately moving us closer to a long-term, functional cure for individuals living with hemophilia A.\u201d<\/p>\n