ASH: Gene therapy for rare genetic disorder, molecular insights into leukemia growth and more

Researchers from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA will present their latest research and clinical trial results at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition. 

The meeting, which will take place from December 4-10, 2024 in San Diego and online, brings together more than 18,000 experts from nearly 100 countries, representing the world's largest gathering of hematology professionals.

UCLA research highlights include:

Successful long-term results for patients who received blood stem cell gene therapy for ADA-SCID (Abstract 1051). Adenosine deaminase–deficient severe combined immunodeficiency, or ADA-SCID, is a life-threatening condition in which babies are born without an immune system. Currently, few therapeutic options — and no FDA-approved curative options — exist. Led by senior author Dr. Donald Kohn, distinguished professor of microbiology, immunology and molecular genetics, this abstract highlights long-term follow-up results on 62 patients treated with a blood stem cell gene therapy for the immune system deficiency in clinical trials at UCLA and Great Ormond Street Hospital in London. After a median of 7.5 years, 100% of patients had survived, and 96% showed sustained immune system restoration. The findings represent 473 cumulative years of patient follow-up and include patient data 11 years post-treatment. Dr. Katelyn Masiuk, a clinical project lead in the laboratory of Dr. Kohn, will present these findings on Monday, December 9 at 4:30pm PT. 

Uncovering the molecular mechanisms driving leukemia progression (Abstract 2743). A major challenge in treating leukemia is understanding how these cancer cells alter their genetic and metabolic processes to survive and grow. Dr. Dinesh Rao, a professor of pathology and laboratory medicine, explores how the RNA binding protein IGF2BP3, found at high levels in many leukemias, regulates cancer cell metabolism and gene expression. Using advanced techniques such as metabolomics and RNA sequencing, Rao’s research team revealed how IGF2BP3 impacts metabolic pathways that are essential for cancer survival. Experimental, pre-clinical inhibition of IGF2BP3 was found to disrupt leukemia cells’ ability to metabolize energy and carry out cellular modifications required for their growth. The findings could serve as a foundation for innovative therapeutic strategies targeting metabolic vulnerabilities in blood cancers. Dr. Gunjan Sharma, a postdoctoral scholar in the laboratory of Dr. Rao, will present these findings on Sunday, December 8 at 6pm PT. 

For more information and to set up interviews with UCLA experts, contact Ani Vahradyan, communications and media relations manager, at AVahradyan@mednet.ucla.edu or 310-968-6144.