Rare Disease Report

Oncology

Manuel Litchman, MD, President and CEO of Mustang Bio Inc, highlights the advantages of using CAR-T therapies as treatments for rare cancers. 
Researchers release updated data from the phase 3 ALEX trial comparing alectinib with crizotinib as first-line treatment against ALK-positive non-small cell lung cancer.
Manmeet Ahluwalia, MD, Head of Operations in the Brain Tumor Center at Cleveland Clinic, explains what a team approach should look like when treating brain tumors and other rare cancers. 
 
Researchers find that the use of PET scans at baseline and after induction therapy can help them tailor chemotherapy regimens in esophageal cancer patients, increasing survival.
Owen A. O’Connor, MD, PhD, Director of Center for Lymphoid Malignancies at Columbia University, emphasizes the importance of sharing experiences among physicians and researchers in the rare diseases like peripheral T-cell lymphomas.
Manmeet Ahluwalia, MD, Head of Operations in the Brain Tumor Center at Cleveland Clinic, discusses the importance of team approaches when treating brain tumors and other rare cancers.
 
Researchers report positive results for phase 1 trial of ivosidenib in patients with IDH1+ acute myeloid leukemia.
Results of a recent study demonstrate that treatment at a specialized cancer center is associated with better leukemia-specific survival in children, adolescents, and young adults. 
A new phase 3 clinical trial boasts highest survival rates of these T-cell malignancies.
First-in-man phase 1 trial meets its primary endpoints by demonstrating safety and immunogenicity of a mutation-specific IDH1R132H peptide vaccine.
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