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A team of researchers has found a potential treatment for diffuse intrinsic pontine glioma (DIPG) and very aggressive pediatric brain cancer and fast growing that frequently causes the death of the patient in just one year after diagnosis. This type of brain tumor forms in the brain stem, above the back of the neck, and its surgical removal is almost impossible due to the risks involved, and its treatment options are very limited.

The new therapy that could improve the prognosis of this serious disease has been developed using a technology of antisense oligonucleotides (ASO)molecules that can control protein levels in cells and slowed tumor growth and reversed some changes in cancer cells that managed to increase survival in mice with this brain cancer, study results published in Science Translational Medicine.

the teacher of Laboratorio de Cold Spring Harbor Adrian Krainer is known for his groundbreaking research on ASOs and his studies led to Spinraza®, the first FDA-approved treatment for spinal muscular atrophy (SMA) – a fatal neurodegenerative disease – that it is also authorized in the European Union.

“After the treatment we saw that the tumor cells were differentiating into healthy nerve cells, which means that the malignant changes in the DIPG are reversible to some extent”

The success obtained with SMA encouraged Krainer to continue investigating other diseases in which ASO molecules could be useful and, according to what he has stated, they contacted him to ask if it would be possible to apply this therapy in cases of DIPG. “Of course, each disease has its own barriers and obstacles, but it seemed doable. We think it might be possible to develop a therapy.”

Reversing the malignant changes of pediatric brain tumor

Krainer, graduate student Qian Zhang, and colleagues have developed a potential therapeutic tool to combat diffuse intrinsic pontine glioma using ASO technology similar to Spinraza’s. “While working at Spinraza we learned how deliver ASO to the spinal cord and brain“, Explain. “They have long-lasting effects there, so we knew there was potential to treat other diseases.”

The new drug ASO deactivates a mutated protein called H3.3K27M. The dominant mutation in DIPG prevents closely related proteins from turning many genes on and off, leading to uncontrolled cell growth, ie cancer. When the researchers administered the drug ASO to mice with DIPG, the affected genes returned to normal, the tumors stopped growing as fast and the animals lived longer.

“After treatment, the cancer looked very different,” Krainer says. “We were able to see far fewer proliferating cells, and the tumor cells were differentiating into healthy nerve cells, which means that the malignant changes in DIPG are reversible to some extent.” Despite the promising results, the scientist has pointed out that there is still a long way to go before this new therapy can be tested in clinical trials, and that it is likely that the potential drug will have to be combined with another treatment, such as radiotherapy or immunotherapy.


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