Clinical trials by Caltech-led team could open the door to 'game-changing' genetic therapies for cancer, Parkinson's and chronic pain - Pasadena Star-News
PASADENA - Caltech researchers believe they are one-step closer to a cure for cancer, thanks to a "game-changing" genetic therapy that's actually working in some patients.
According to paper published Sunday in the journal Nature, the technique might even work in patients suffering from Parkinson's, Alzheimer's and chronic pain.
Lead researcher Mark Davis said he began to look for an alternative to chemotherapy after watching his wife Mary almost die from treatments.
"She basically said, 'There's got to be a better way than this, why don't you start working on it?" Davis said.
She was diagnosed with breast cancer in 1996 before anyone had proved this kind of genetic therapy could work on animals - much less humans.
"After quite a few years of work here at Caltech and proving that we could do these in animal models, we decided it was ready to go with humans," said Davis, a chemical engineer at Caltech.
This scientific breakthrough is particularly promising for "undruggable" diseases that can't respond to medicine, Davis said.
The therapy works by injecting tiny particles directly into the blood stream of cancer patients. These nanoparticles are stealthy inventions that are programmed to invade the cancer cells in tumors and snip the messenger RNA inside these cancerous cells in two.
This is the key - the therapy interrupts the process of cancer cells replicating by slicing what is called the messenger RNA. If the messenger RNA of these cells is destroyed,
these cancerous cells can't reproduce and tumors can't grow, Davis said.
For the first time, the "game-changing" idea of attacking a disease at the genetic level worked in clinical trials on actual patients.
"We're such complex organisms that you don't know how it's going to work (until you go into clinical trials)," Davis said.
Clinicians took a biopsy of the cancer patients. They found the "smoking gun signature" in the cancer - bits of RNA snipped in half exactly in the place researchers expected.
"It cut at exactly the right place," Davis said. "If you take that away, you take away the cancer."
Scientists have been interested in interfering with a disease at the genetic level since the idea was first floated in a paper by Andrew Fire and Craig Mello in 1998. The pair went on to win a Nobel Prize in Physiology in 2006 for their breakthrough, but they were working on the genes of worms - not humans.
The first phase of the clinical trials began in May 2008, and they are continuing in Los Angeles at the UCLA Jonsson Comprehensive Cancer Center and the South Texas Accelerated Research Therapeutics in San Antonio.
The trials are headed up by a Caltech start-up company, Calando Pharmaceuticals.