Mount Sinai Scientists Break Brain Barrier for Drug Delivery
Scientists at Mount Sinai Hospital have made a groundbreaking discovery that could revolutionize the treatment of complex brain diseases. They've found a way to deliver drugs directly to the brain, bypassing a major obstacle known as the blood-brain barrier.
The blood-brain barrier, a natural defense mechanism, prevents harmful substances from entering the brain but also blocks beneficial drugs. Researchers have long sought a way to overcome this challenge. The Mount Sinai team, led by Dr. Eric Holland, has achieved this by exploiting a biological process called γ-secretase-mediated transcytosis.
In tests on model mice with ALS, the team successfully injected drugs that significantly reduced harmful genes causing the disease, with no observed side effects. This method also reduced the gene responsible for encoding the TAU protein, linked to Alzheimer's and other dementias. The potential implications are vast, opening new avenues for the treatment of complex brain diseases such as ALS, Alzheimer's, and potentially even brain tumours.
Meanwhile, a research group at Georg-August-University Göttingen is working on a similar approach. They aim to temporarily open the blood-brain barrier to allow cancer drugs to enter the brain directly, aiming to improve treatment for brain tumours in children.
This innovative method allows drugs to be administered through an intravenous injection, penetrating directly into the brain tissue. It effectively and safely delivers biomacromolecules and oligonucleotides into the nervous system. While more research is needed, these findings offer fresh hope for patients and families affected by these devastating diseases.
