(REUTERS/Suzanne Plunkett)
A tray containing cancer cells sits on an optical microscope in the Nanomedicine Lab at UCL's School of Pharmacy in London.
As researchers look for new ways to approach tough-to-treat forms of cancer, many are focusing on genetics — more specifically, they're looking at the type of genetic tweaks found in cancerous tumors.
One early-stage cancer company, Loxo Oncology, hopes to eventually sequence the tumors of its patients and use that data to find out what drugs their disease would respond best to. With this kind of tool, the type of cancer someone has (breast cancer vs. lung cancer, for example) wouldn't matter so much as the genetic information gleaned from the tumor.
In data presented Sunday night at the American Association of Cancer Research, Loxo gave an update on their ongoing Phase 1 trial for their drug, LOXO-101, which works in cancer patients with a mutation called a "TRK gene fusion." Phase 1 clinical trials for cancer drugs are used to figure out what dose is safe, how it should be given, (in this case orally), and if it has any effect on the people it's treating.
All the patients involved in the trial had exhausted all other treatment options for their particular tumor. Out of the 43 patients that took part in the trial, seven had the specific genetic tweak Loxo was looking for. Of these seven, five met the threshold for what's considered a partial response, meaning their tumor displayed signs that it was shrinking. The sixth showed some tumor regression, but didn't hit the threshold, while the seventh participant joined the trial too recently for there to be any conclusive results with this update. And those patients have managed to maintain results, with the longest participant still on the drug after 14 months.
The most interesting part? People with the TRK mutation did not all have the same type of cancer. Some had sarcoma, while others had gastrointestinal stromal tumor cancer, salivary cancer, thyroid cancer, or non-small cell lung cancer. That means that, unlike most traditional cancer drugs, the medication didn't just work based on cancer type, but rather it worked on a genetic level. This idea isn't new: Scientists have seen genetic patterns across cancer types for years, an idea that gained notoriety in 2013 with the discovery that endometrial cancer was genetically similar to forms of ovarian and breast cancer.
Loxo CEO Dr. Josh Bilenker told Business Insider that going into the trial, the company had a good feeling that patients with the fusion would respond while those without likely wouldn't see any benefit.