Rare and Orphan Diseases

Host: Jennifer Caudle, DO

Guest: Naval Daver, MD

Not only is CD123 one of the most common antigens expressed on the surface of acute myeloid leukemia (AML) tumors, but it’s also associated with more proliferative disease that’s resistant to standard therapies. Given its prevalence and potential implications, a number of different CD123-targeting approaches are under investigation, including antibody-drug conjugates, bi-specific antibodies, fusion protein, and CAR T-cell therapy. In light of these new approaches, Dr. Jennifer Caudle and Dr. Naval Daver discuss the importance of targeting CD123 in AML. Dr. Daver is a Professor and Director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center in Houston.

Host: Charles Turck, PharmD, BCPS, BCCCP

Guest: Aaron Logan, MD, PhD

Although acute lymphoblastic leukemia (ALL) is a relatively rare disease with fewer than 7,000 people diagnosed each year in the US,1,2 there’s been a lot of progress in the management of B-cell ALL over the years. One key development comes from the E1910 Phase III trial, which explored the addition of blinatumomab to frontline consolidation chemotherapy for patients with Philadelphia chromosome-negative B-lineage ALL (B-ALL).3 Joining Dr. Charles Turck to discuss the impacts of this data on B-ALL clinical guidelines and practice is Dr. Aaron Logan. Dr. Logan is a Professor of Clinical Medicine in the Division of Hematology/Oncology and Director of the Hematologic Malignancies Tissue Bank at UCSF.

References:

1. National Cancer Institute. SEER Cancer Stat Facts: Acute Lymphocytic Leukemia (ALL). Accessed at https://seer.cancer.gov/statfacts/html/alyl.html on March 31, 2025
2. Dana-Farber Cancer Institute. Acute Lymphoblastic Leukemia (ALL). Accessed September 17, 2024. https://www.dana-farber.org/cancer-care/types/acute-lymphoblastic-leukemia
3. Litzow MR, et al. Blood. 2022;140(suppl 2):LBA-1

Guest: Lauren Schaff, MD

Glioblastomas are fast, aggressive, and resistant to many standard therapies. Dr. Lauren Schaff, a neuro-oncologist at Memorial Sloan Kettering Cancer Center, explains how new molecular understandings and treatment avenues are paving the way for a more personalized, hopeful approach to care.

Guest: Jennie Taylor, MD

The FDA approval of vorasidenib marks a new era for mutant isocitrate dehydrogenase (mIDH) gliomas. Approved in 2024 for grade 2 tumors after surgery, it doubled progression-free survival in the INDIGO trial. Dr. Jennie Taylor, Associate Professor of Neurology and Neurological Surgery at the University of California San Francisco, dives into the data and explains what questions remain about long-term use and broader applications.

Guest: Jennie Taylor, MD

Defined by the production of 2-hydroxyglutarate, mutant isocitrate dehydrogenase (mIDH) gliomas are diffuse, slow-growing tumors. Managing these tumors requires personalized strategies that consider resectability, histology, and long-term treatment impacts. Dr. Jennie Taylor, Assistant Professor of Neurology and Neurological Surgery at the University of California San Francisco, explains the complexities behind this type of tumor and implications for patient care. Dr. Taylor also spoke about this topic at the 2025 American Academy of Neurology Annual Meeting.

Host: Charles Turck, PharmD, BCPS, BCCCP

Guest: Kaitlin Batley, MD

Guest: Esra Caylan, MD

Thymidine kinase 2 deficiency (TK2d) presents with a variability of symptoms, including respiratory, neurological, and ocular ones, making it difficult to diagnose. Because of this, a multidisciplinary care team that’s tailored to each patient’s needs is key to effectively identifying and treating this disease. Hear about the role of a multidisciplinary team and strategies for personalizing treatment with Drs. Kaitlin Batley and Esra Caylan. Dr. Batley is the Director of Pediatric Neuromuscular Medicine at Children’s Health and an Assistant Professor at UT Southwestern Medical Center. Dr. Caylan is a pediatric pulmonologist at Children’s Health and an Assistant Professor in the Department of Pediatrics at UT Southwestern Medical Center.

Guest: Hans Katzberg, MD

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a complex immune-mediated condition affecting the peripheral nervous system. Join Dr. Hans Katzberg, Professor of Medicine at the University of Toronto, as he explains the pathophysiology behind CIDP, risk factors in disease development, and diagnostic strategies.

Guest: Sami Khella, MD

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) can present in multiple ways, making diagnosis complex. Dr. Sami Khella joins the program to outline how we can distinguish CIDP from similar neurological disorders and explain how response to therapy can guide diagnosis. Dr. Khella is the Director of Clinical Electrophysiology and a Professor of Clinical Neurology at the University of Pennsylvania.

Guest: Sami Khella, MD

With personalized approaches, alternatives to steroids, and novel therapies like efgartigimod, the treatment landscape for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is continuing to evolve. Join Dr. Sami Khella as he shares expert insights on our available options. Dr. Khella is the Director of Clinical Electrophysiology and a Professor of Clinical Neurology at the University of Pennsylvania.

Host: Nathan Falk, MD, MBA, FAAFP

Guest: Ashwin Basavaraj, MD

Early recognition and diagnosis of non-tuberculosis mycobacteria (NTM) and bronchiectasis are key for optimal patient management. However, these conditions are often misdiagnosed as COPD or asthma, leading to significant challenges and delays in treatment. Join Drs. Nate Falk and Ashwin Basavaraj as they share perspectives on accurately diagnosing NTM and bronchiectasis with early symptom recognition, a thorough patient history, and testing. Dr. Falk is a board-certified family medicine physician, a Professor and Founding Residency Director for Family Medicine at Florida State University in partnership with BayCare Health System, and the Assistant Dean for Graduate Medical Education at Florida State University. Dr. Basavaraj is an Associate Professor of Medicine at New York University Grossman School of Medicine, the Director of the Bronchiectasis and NTM Education Program at NYU Langone Health, and the Section Chief of Pulmonary, Critical Care, and Sleep Medicine at Bellevue Hospital Center.

This program is produced in partnership with the American College of CHEST Physicians and is sponsored by Insmed Incorporated.