The news can be a damper sometimes, and in 2017, it seemed to be a heavy weight a lot. But there were sparks of hope in some places, especially in medicine, where the next frontier of health technology made what we formerly thought impossible pretty damn possible. Between gene editing, mind control, and viruses carrying secret fixes to medical problems like a spy behind enemy lines, it’s been a year of advancements that sometimes seem like a great science fiction thriller. Except it’s not; it’s real and awesome. Here are the five medical advances that made the world a healthier, better place in 2017.
1. Blind people can see again
One week before Christmas, the FDA approved a gene therapy for the first time ever, paving the way for the technique’s broader use in solving the most dire medical issues of today that stem from genetic mutations. Luxturna got an enthusiastic go-ahead from the FDA for people suffering from an inherited condition called Leber congenital amaurosis (LCA), which slowly degrades vision to the point of blindness for some patients. Luxturna targets the RPE65 protein that LCA patients can’t produce correctly by inserting DNA for the protein directly into the eye via a harmless virus. The virus funnels itself into the retina, enters cells, and re-programs them by “infecting” them so begin the proper production of RPE65 and correcting the retina’s faulty mechanism. While Luxturna isn’t a complete cure for blindness—it allows LCA patients to discern shapes and light—it provides a sense of independence and freedom to explore the world that is often taken for granted by sighted individuals. The therapy offers hope for people like 17-year-old Christian Guardino, who competed on America’s Got Talent and was treated for the disorder at just 13. Guardino told The Daily Beast that before Luxturna, he couldn’t see the moon traipsing across the night sky. Now, “I can see the stars.”
Doctors wrap a child’s burnt skin with sterilized tilapia fish skin at Dr. Jose Frota Institute in the northeastern costal city of Fortaleza, Brazil, May 3, 2017.
2. Burns can be soothed with fish scales
Burn victims face a critical time and medical need when skin is scorched: If they’re not covered to prevent infection, patients can die. In developing countries like Brazil, options that are used in places like the United States—human skin, pig skin, and artificially grown lab alternatives—aren’t widely available. In the small coastal town of Fortazela, however, doctors have come up with an ingenious, effective, and cheap fix: sterilized tilapia scales. Tilapia scales have the unique ability to protect burned skin and stay on thanks to a collagen proteins that are abundant in tilapia skin that promote healing and reduce scarring in humans. It also helps that tilapia scales retain moisture and are resistant, allowing them to be a strong but tender barrier for delicate, burned people. Refrigerated and sterilized, the tilapia skin stays fresh and usable for a couple years. Add to this the fact that tilapia scales speed healing time, require far fewer changes, and make the healing process less painful, and tilapia scales come across as practically a miracle. Tilapia scales are not going to arrive in the United States any time soon for burn victims—other alternatives are plentiful, and preserving tilapia scales actually can be more costly here—but for developing countries that don’t have access to these options, can sterilize the scales, and where tilapia are plentiful, the tilapia scale burn hack is nothing short of awesome.
3. Brain implants can help quadriplegics move with thought
Paralysis used to be an end-all for people with spinal cord injuries: Damage to the fragile cord that runs messages from the brain to the body’s nervous system about movement—jerk your hand off the hot iron, flick your head this way, shimmy around the table corner, step back from the oncoming car—meant a lifetime of being unable to move independently. The tragedy of spinal cord injuries and paralysis ranged from those who couldn’t move a limb to others who were quadriplegic, unable to move any part of their body from the neck down. But in March at Case Western Reserve University, quadriplegic patient Bill Kochevar was able think about actions and actually perform them for the first time in eight years. This was thanks to BrainGate2, a system of a pair of electrodes implanted in Kochevar’s brain that communicated with another set of electrodes embedded in Kochevar’s arm muscles. The results are “amazing,” he said in a video: Kochevar can extend his arm and grab objects, an action that seems simple but requires a complex set of instructions from his brain to his arm that was impossible without BrainGate2. “I thought about moving my arm and it did,” Kochevar said. “I can move it in and out, up and down.” For a man who couldn’t use his arms at all last year, that’s truly amazing.
4. CAR T-cell therapy can cure incurable cancers
In mid-December, in a televised moment that warmed hearts, former Vice President Joe Biden clutched hands with Meghan McCain, whose father, Arizona Sen. John McCain, is battling brain cancer—the same disease that killed Biden’s son, Beau. Biden cited the groundbreaking research being done at the University of Pennsylvania that holds hope for people battling the terminal cancer. The chimeric antigen receptor (CAR) T-cell could augment the first line of defense of a cell’s immune response to an invader, whether that be cancer or the flu. Researchers are working on genetically re-engineering these T-cells with the CAR molecule on their surfaces so they target tumor cells with an army of similar CAR T-cell copies and destroy cancerous cells. For brain cancer patients, this could at the very least prolong life, and at the very optimistic end, get rid of brain cancer tumors, which are unusually aggressive and latch onto organs, making them difficult to saw off. The FDA has already given the go-ahead to two CAR T-cell therapies: Kymriah, for patients below 25 battling acute lymphoblastic leukemia, and Yescarta, for non-Hodgkin’s lymphoma patients. It might take a while for these treatments to trickle down to brain cancer patients, but the astounding results “borders on miraculous,” one doctor told The Daily Beast.
Brian Madeux sits with his girlfriend Marcie Humphrey while waiting to receive the first human gene editing therapy at the UCSF Benioff Children’s Hospital in Oakland, Calif. Madeux, who has Hunter syndrome, received the treatment on Monday, Nov. 13.
5. Genes can be directly edited in a living, breathing person
Gene editing technology like CRISPR has captured the imagination of scientists and futurists, launching ethical debates about designer babies and questions about the accuracy of chopping off blocks of DNA or squeezing in bits to “perfect” the human genome. But until November of this year, gene editing was limited to the petri dish sort: Isolated chains carefully edited using tools outside the human body. In November, however, that changed for the first time ever. Brian Madeaux, who suffers from Hunter syndrome (a condition that prevents Madeaux from properly breaking carbohydrates down due to an enzymatic malfunction), had a non-infectious virus carrying two zinc finger proteins injected into him. In other words, the gene editing was occurring right in his body, not outside of it, which pushed the boundaries of this research forward into new frontiers. Madeux’s proteins carried instructions for cells to fix the enzyme issue, which was then copied to the liver. That’s huge: The very mechanism that Madeux had suffered from for his entire life was getting corrected right at the base with precision. “We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,” a doctor told the AP. Madeux is still under constant supervision but he’s “nervous and excited” to see what this gene therapy could do, and what it might mean for the millions of people dealing with errors in their genetic code.
