04 August 2023

RNAi therapy against the most 'hard-to-treat' cancer worked three times in the lab

A new RNAi therapy could help treat bone marrow cancer.

RNA therapy is becoming a promising cancer treatment, but some types of pathology are not amenable to it.

Now scientists at Tel Aviv University have shown how to use RNAi drugs to treat multiple myeloma. This is a form of chronic lymphoblastic leukemia, which is characterized by bone marrow damage, destruction of bone tissue and production of identical immunoglobulins. Such blood oncopathology affects the flat bones, spine, and kidneys. In multiple myeloma, plasma immune cells are affected. They become cancerous and accumulate in the bone marrow.

Although the disease can be managed, it is almost impossible to cure. Multiple myeloma is truly a "hard-to-treat cancer."

In a new study, researchers at Tel Aviv University have demonstrated a promising new treatment technique using an RNA-based drug. The active ingredient is small interfering RNA (siRNA) molecules. They silence the action of the target gene and stop it producing protein.

In this case, the RNA targets the CKAP5 gene. Without the corresponding protein, cells can no longer divide. To ensure that this effect only applies to cancer, the scientists developed lipid nanoparticles. They deliver the drug, and a special coating of antibodies helps them "cling" only to structures on a few myeloma cancer cells.

Авторы исследования протестировали метод на множественных раковых клетках миеломы в нескольких различных конфигурациях. Наночастицы уничтожили 90% патологических клеток, выращенных в лаборатории. Затем ученые протестировали терапию на раковых клетках пациентов с множественной миеломой — уничтожено 60%. Также ученые вводили препарат мышам с болезнью. Препарат ликвидировал около 66% раковых клеток. Также ученые отметили значительное улучшение симптомов животных.

Хотя предстоит еще много работы, прежде чем начнутся испытания на людях, авторы исследования уверены — их первые результаты очень многообещающие.

Исследование опубликовано в журнале Advanced Science.

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