Could freeze-dried gene therapy someday treat brain cancer?

Kathleen Blanchard's picture
Johns Hopkins team study freeze-dried gene therapy for brain cancer

Nanoparticle based gene therapy could be a safe, effective cancer treatment

Johns Hopkins researchers envision a day when gene therapy, using frozen water, could be delivered safely to treat brain cancer. In experiments, scientists found a way to store genes up to three months using nanoparticles that have an affinity for penetrating brain cancer cells.

Gene therapy for cancer treatment use viruses as a vector to deliver cancer therapy, but viruses can cause more health problems for patients.

Jordan Green, Ph.D., an assistant professor of biomedical engineering at Johns Hopkins explains:

"Most non-viral gene therapy methods have very low efficacy. Nanoparticle-based gene therapy has the potential to be both safer and more effective than conventional chemical therapies for the treatment of cancer."

For the research, which appears in the August issue of the journal “Biomaterials”, the scientists used molecules that they mixed in various combinations to produce different polymers.

Next, they mixed DNA into the polymer combinations to form nanoparticles and added them to human brain tumor cells and human brain tumor stem cells.


Forty eight hours later the researchers observed which cells took up the nanoparticles with the encoded DNA, shown by a “glowing” protein. They counted which cells died and which ones survived.

One combination in particular penetrated glioblastoma and brain tumor stem cells.

The Hopkins team then freeze- dried the nanoparticles and tested them at different intervals, finding the DNA could still enter cancer cells after three months, with no change in performance.

Alfredo Quinones-Hinojosa, M.D., Ph.D., an associate professor of neurosurgery and oncology at Johns Hopkins said:

"I envision that one day, as we understand the etiology and progression of brain cancer, we will be able to use these nanoparticles even before doing surgery—how nice would that be? Imagine avoiding brain surgery all together.'

He also says he could see the freeze-dried gene therapy being used in conjunction with brain surgery to treat cancer. The formula used by the researchers that showed most promise for treating brain cancer was so-called poly(beta-amino ester) nanoparticles that penetrated both glioblastoma and brain tumor stem cells.

Image: Glioblastoma
Credit: Wikimedia Commons