Myeloma cells are gone! The results of animal experiments on antisant oligonucleotide therapy were positive

Researchers at the University of California, San Diego (UCSD) School of Medicine and Ionis Pharmaceuticals are using a new targeted therapy to treat multiple myeloma.
they used anisotym oligonucleotides (ASOs) to silence the IRF4 gene associated with the proliferation of myeloma progenito cells and tumor cells, and achieved positive results in animal experiments.
study was published today in Cell Stem Cell, part of Cell.
UCSD School of Medicine and Ionis have a long history of collaboration in the development of experimental ASOs.
ASO developed by Ionis has been approved, including FDA-approved Spinraza, a treatment for spinal muscular dystrophy.
, the researchers tested the therapeutic effect of ION251 developed by Ionis in in-body and mouse models.
ION251 is an ASO therapy that targets the MRNA of the IRF4 gene.
IRF4 is a gene that allows myeloma progeny and tumor cells to proliferate and survive.
previous studies have shown that high levels of IRF4 are associated with lower overall survival rates in patients with the disease.
in-body trials, ION251 was able to remove myeloma ancestral cells while preserving healthy blood cells.
in a model of multiple myeloma formed by transplanting human myeloma cells into mice, mice treated with ION251 had significantly fewer myeloma cells after 2-6 weeks.
, animal survival rates in different treatment groups reached 70 to 100 percent, while no mice survived in the control group without treatment.
"the results of these preclinical studies are surprising.
in the treated mice, we couldn't find any residual myeloma cells for us to continue our research.
, "It makes our research harder, but it gives patients hope," said Professor Leslie A. Crews of the UCSD School of Medicine and one of the co-authors of the study.
show that using Ionis's antonym techniques to target "drug-free" targets in cancer may bring promising new drugs to patients in dire need.
," said A. Ionis, vice president and head of oncology. Dr. Robert MacLeod said.
, phase 1 clinical trials to test the safety of ION251 have been launched and patients are being recruited at UCSD's Moores Cancer Center and other clinical trials.
: This article is intended to introduce medical and health research, not treatment options recommended.
if you need guidance on treatment options, visit a regular hospital.
resources: s1. Designer DNA therapeutic wipes out cancer stem cells, treats multiple myeloma in mice. Retrieved January 20, 2021, from [2] Mondala et al., (2021). Selective antisense oligonucleotide inhibition of human IRF4 prevents malignant myeloma regeneration via cell cycle disruption. Cell Stem Cell,