New drug approach shows promise against incurable blood cancer

Researchers have identified a promising new way to attack multiple myeloma, an incurable blood cancer that affects plasma cells in the bone marrow.

The international study, published in the prestigious Nature Portfolio journal Leukemia, one of the leading journals in haematology and oncology, investigated an experimental drug called CBPD-409. The drug showed powerful anti-cancer effects in laboratory studies and significantly reduced tumour growth in animal models.

Multiple myeloma remains incurable for many patients, and treatment becomes increasingly difficult when the cancer develops resistance to existing therapies. Finding new approaches is therefore urgently needed.

The research team, co-led by Dr Mark Williams from the Research Centre for Health (ReaCH) at Glasgow Caledonian University, focused on two proteins called CBP and p300. These proteins help myeloma cells survive, grow and multiply.

Unlike conventional drugs that simply block protein activity, CBPD-409 is designed to remove CBP and p300 from cancer cells altogether. It does this using PROTAC technology, a new class of targeted protein degrader that harnesses the body’s natural protein disposal system to destroy disease-driving proteins.

The researchers found that CBPD-409 killed myeloma cells at very low doses while having limited effects on healthy cells. In animal models, the treatment significantly slowed tumour growth.

Further analysis showed that degrading CBP and p300 disrupted key biological pathways that myeloma cells depend on, forcing the cancer cells to stop dividing and ultimately die.

The findings suggest that removing CBP and p300 could offer advantages over existing approaches that only partially inhibit these proteins.

Dr Williams said: “This study highlights the exciting potential of PROTAC-based therapies to selectively eliminate proteins that drive cancer growth. CBPD-409 showed strong anti-myeloma activity in preclinical models, and these findings provide an important foundation for further research.

“While more work is needed before this approach can be tested in patients, the results point towards a promising new strategy for developing more precise and effective treatments for multiple myeloma.

“PROTACs will likely become the next generation of highly effective treatments for cancers and non-cancerous conditions, and, rather excitingly in May of this year, the first PROTAC was clinically/FDA approved for the treatment of an aggressive type of breast cancer known as advanced or metastatic ESR1-mutated ER-positive/HER2-negative breast cancer.”

The full study is available in Leukemia: You can read the full study here.