Some Important Scientific Breakthroughs From 2018

One week from today, we will be closing out 2018 and ringing in the New Year. By almost anyone’s estimation, it’s been a wild year, fraught with political drama and cataclysmic events: the California wildfires; the eruption of Kilauea on the Big Island of Hawaii, Hurricane Florence and much more. But at the same time, there were some astonishing and possibly game-changing scientific breakthroughs this year. So, in the spirit of ending 2018 on a positive note, I thought I’d share some of them with you this Christmas Eve.

A New Kind of Cancer Drug

New chemotherapy targets genetic markers vs. cancer type
Credit: canteen.org.au

A new drug that targets cancers based on their genetic signature rather than site of origin was approved by the Food and Drug Administration this year. Developed by Stamford, Connecticut, based Loxo Oncology in partnership with Bayer, the drug larotrectinib (marketed as Vitrakvi) won FDA approval for the treatment of all cancers with certain genetic characteristics (specifically, a genetic mutation called “TRK gene fusion”). Thus far it’s been used to treat breast, colon, lung and thyroid cancers and, according to Loxo’s data, the results have been quite promising. About 80 percent of patients treated saw some tumor response, and 17 percent had their tumors disappear.

The downside, unsurprisingly, is the cost: about $393,000 a year for an adult and $132,000 for a child. But Bayer has promised that any patient who needs the drug will have access, pledging that it will keep patient co-pays to about $20 per month.

A New Preventive for Migraine Sufferers

About 38 million people in the United States suffer from migraines — searingly painful headaches that are often accompanied by nausea, vomiting, visual changes, and sensitivity to sound and light. Yet, amazingly, there are almost no drugs currently on the market that are designed to prevent migraines (as opposed to treating them after they occur).

But that changed this year, when the drugmaker Eli Lilly received FDA approval for a new medication, galcanezumab, which will be marketed under the brand name Emgality. One of only three medicines in the class class of drugs known as CGRP-inhibitors, Engality acts on naturally occurring vasodilators known as calcitonin gene-related peptides, the levels of which have been shown to increase during migraines. The once-a-month injection costs $575, but Lilly has offered to provide the drug for free for a year to patients with health insurance as it negotiates coverage with insurers.

Miniature Cancer-Hunting Robots

It’s a scenario that would be right at home in a science fiction movie: tiny robots coursing through a person’s bloodstream, hunting for tumors and armed with medicines that kill cancer cells. Yet, as is often the case today, truth is stranger than fiction, and these robots actually exist.

Designed by a team of scientists from Arizona State University and the Chinese Academy of Sciences, the miniature robots are about 1,1000th the diameter of a human hair, folded origami-style, and enclosed in cancer-hunting strands of DNA. Inside they carry a substance called thrombin, an enzyme that causes the formation of blood clots. 

Miniature robots unfold to release thrombin at cancer site
Credit: ASU.edu

When the DNA recognizes a cancer, it unzips its contents, and the robot unfolds. Then out come the thrombin molecules, which immediately form a clot at the site, depriving tumor cells of blood and oxygen. According to a report on the research in the journal Nature, this leads to tissue death within 24 hours and eventually causes the tumor to shrink.

Thus far this futuristic nanotechnology has only been used on mice and a few miniature pigs. But the results have been promising. According to Business Insider’s report, the average survival times of the mice (who suffered from a variety of cancers) doubled, and several skin cancers disappeared entirely. 

Stem Cells for Macular Degeneration

Macular degeneration is the leading cause of vision loss in the United States. According to the American Macular Degeneration Foundation, the disease affects over 10 million Americans, more than cataracts and glaucoma combined. Caused by deterioration of the macula — the central portion of the retina — it causes central vision loss and, eventually, blindness.

The anatomy of a healthy eye
Credit: allaboutvision.com

Until recently there was no treatment for the most common form of macular degeneration, although lifestyle changes such as quitting smoking have been shown to slow the progression of the disease. But in an amazing scientific breakthrough, researchers at the University of Southern California Keck School of Medicine successfully used a stem cell patch to treat a small number of patients in a clinical trial this year. The patch, which consisted of a specially engineered sheet of stem cells, was applied to the back of the eye to replace the layer of cells destroyed by the disease.

According to the study’s lead researcher, Dr. Amir Kashan, the treatment seemed to halt vision loss in four of the first five people treated, and one patient’s vision significantly improved. “We didn’t really anticipate or expect dramatic improvement in vision,” Kashani said. “That was a very encouraging sign.”