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National brain tumour research funding needs to increase to £30-35 million a year

Weekly pick of brain tumour research news from around the world

Our opening item has been written by the journalist with this week’s best name, Deanna Csomo McCool, and her piece looks at how common cholesterol drugs could slow spread of breast cancer to brain by interfering with the way breast cancer cells adapt to the microenvironment in the brain thereby preventing the cancer from taking hold. Evidently Statins were shown to interfere with a pathway that allows a cancer cell to recycle cell surface proteins and therefore make it easier for cancer cells to live within the brain.

This week we have space to look at the US designation of potential therapies as either “breakthrough therapy designation” or “orphan drug designation,” A breakthrough therapy designation is for a drug that treats a serious or life-threatening condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.

Hopefully, that will help understanding these two items better;

FDA Grants Breakthrough Therapy Designation to Vorasidenib CDx in IDH1/2-Mutant Low-Grade Gliomas

CNS Announces the FDA has Granted Orphan Drug Designation for Brain Cancer Drug Berubicin

The protein PHIP (pleckstrin homology domain-interacting protein) has now been shown to play an important role in the development and progression of glioblastoma. One reason that GBMs are difficult to treat is that they arise from a type of brain cell called astrocytes. Shaped like a star, these cells develop tentacle-like branches that are difficult to remove surgically. Astrocytes help control the amount of blood reaching neurones, cells that become cancerous gain access to a reservoir of blood vessels that allow the cells to grow and spread. PHIP resides at the leading edge of GBM cells, enabling other adhesion proteins to deepen the grip of cancerous cells that invade healthy brain tissue. PHIP also helps support the development of new blood vessels that nourish cancerous tissue in the brain. The findings of this research are the first to identify PHIP as a compelling target for GBM therapy.

Just one more US designation this week and it’s ‘fast track designation’ which has been given to TVI-Brain-1 an immunotherapy being developed by TVAX Biomedical  A potential therapy is added to the fast track programme if it might treat serious conditions with unmet clinical need, either because no treatments are currently available or because the potential treatment offers significant benefits over approved alternatives. You can read more about TVI-Brain-1 by clicking on the headline  Cancer Vaccine Plus Adoptive T-cell Therapy Placed on Fast TrackTVAX is planning to open a Phase 2b trial testing TVI-Brain-1 in people newly diagnosed with glioblastoma multiforme this year.

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