Ionis and the University of California, San Diego (UCSD) have formed a team to investigate antisense oligonucleotide technology as a treatment for multiple myeloma. Their drug, ION251, has shown positive results when administered to mouse models of the rare cancer. This data has led to a Phase 1 trial that is currently enrolling patients.

About Multiple Myeloma

Multiple myeloma is a form of cancer that impacts plasma cells. It causes them to accumulate in the bone marrow and push out healthy cells, resulting in M proteins. It is these proteins that cause the characteristic symptoms, which include bone pain, weight loss, nausea, loss of appetite, fatigue, kidney problems, constipation, frequent infections, mental fogginess, low blood count, excessive thirst, weakness or numbness in the legs, and hypercalcemia. While medical professionals do not know what causes this cancer, they do know that many myeloma cells are missing part or all of chromosome 13. Treatment options for multiple myeloma include chemotherapy, steroids, immunomodulatory drugs, HDAC inhibitors, and proteasome inhibitors.

ION251 for Multiple Myeloma

ION251 is an antisense oligonucleotide, which is a form of treatment that binds to mRNA in order to stop certain genes from expression and producing proteins that cause disease. In this case, ION251 binds to and silences the IRF4 gene, as it aids in the growth and reproduction of malignant cells.

Using mouse models of multiple myeloma, researchers from Ionis and UCSD were able to study the drug’s impact. They evaluated the mice for six weeks, administering a dose of ION251 every day for the first week and three doses a week afterwards. In terms of results, 70% of the mice survived, which can be compared to the control group in all of the mice died. Researchers stated that the drug enhanced survival and significantly reduced the number of cancer cells.

Looking Forward

Because of the success of preclinical trials, researchers are now recruiting for a Phase 1 study of ION251 in patients with treatment-resistant multiple myeloma.

Ionis is excited by the positive results, as ION251 is now one of several oncology drugs in their pipeline. It will join other antisense oligonucleotides in the pipeline, including treatments for metabolic, renal, cardiovascular, neurological, and lung diseases.

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