Elon Musk has said he thinks nanotechnology is BS (and launched a Twitter tirade to prove it) but a paper published in the peer-reviewed journal Scientific Reports shows just how game-changing this micro-scale science can be, particularly from a medical perspective. And just how wrong Musk is.
MIT scientists have successfully treated the tumors of an especially nasty type of brain cancer called glioblastoma using tiny, tiny drug-containing capsules only a little more than 100 nanometers big. These nanoparticles deliver treatment straight to the tumor, killing the cancerous cells and preventing regrowth.
To do so, the nanoparticles, known as liposomes, carry two different drugs – one damages the DNA of the tumor cells (temozolomide) and the other obstructs the process that usually repairs the damage (bromodomain inhibitors). This gives a "one-two punch" that inhibits the cell's ability to repair its DNA before attacking the DNA when its defenses are compromised.
The researchers based their design on previous work by Scott Floyd and Michael Yaffe, who are both authors on this new paper, loading the nanoparticle's core with temozolomide and inserting the bromodomain inhibitor into the fatty outer shell.
The particles are then coated in a protein called transferrin, which hones in on and latches onto proteins in the surface layer of tumor cells. This is what stops it from attacking healthy cells and ensures the drugs only hurts the glioblastoma. Because treatment is so targeted, higher doses of chemotherapy drugs can be administered without provoking side effects like bruising and nausea.
Transferrin is what also allows the nanoparticles to pass the blood-brain barrier – the brain's defense system, shielding it from any potentially hazardous molecules lurking in the bloodstream. Without it, the nanoparticles wouldn't even be able to reach the tumor.
The published paper details the success of the medication – but only in mice so far. Those treated with the transferrin-coated nanoparticles not only lived twice as long as those treated with different medications (for example, having or temozolomide and JQ-1 injected into the bloodstream independently), they also showed less non-tumor tissue damage
“This was really a proof-of-concept study [showing] that we can deliver novel combination therapies using a targeted nanoparticle system across the blood-brain barrier,” Fred Lam, a Koch Institute research scientist and lead author, explained in a statement.
But it (hopefully) won't be too long before they begin clinical trials in humans, paving the way for a treatment that can help cure a cancer as aggressive as glioblastoma, which has an average survival rate of just one year.
“Because there’s such a short list of drugs that we can use in brain tumors, a vehicle that would allow us to use some of the more common chemotherapy regimens in brain tumors would be a real game-changer,” Floyd says.
“Maybe we could find efficacy for more standard chemotherapies if we can just get them to the right place by working around the blood-brain barrier with a tool like this.”