10 Life-Saving Medical Breakthroughs

HEART FAILURE. RECTAL cancer. Brain bleeds. Each of the people in this package of stories might not be alive today without a key medical innovation that took many years, millions of dollars, and countless setbacks and breakthroughs to get quite right. Who are the next people to be saved?

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8. Gene Editing

Earlier this year, KJ Muldoon, a baby boy at the Children’s Hospital of Philadelphia, became the first person in the world to receive gene editing treatment that corrected a rare genetic disorder.

KJ was born with a deficiency in an enzyme that breaks down ammonia, a by-product of protein digestion. He had to follow a special diet and take medication to keep ammonia from accumulating in his blood and damaging his organs. About half of children with this disorder die in infancy.

When he was 7 months old, and again at 8 months, KJ was treated using the CRISPR gene editing system to correct the problem at its source. Scientists carefully designed RNA molecules and put them into tiny lipid envelopes. These took messenger RNA to the right target in his cells to cut and fix the gene causing the issue.

Within two months, KJ started eating more protein and taking less medication. Researchers say the technology could be adapted to treat other rare genetic diseases.

9. Pig Organ Transplants

In February, 66-year-old Tim Andrews, a New Hampshire man with end-stage kidney disease and a blood type that complicated his search for a donor, became the fourth living person to receive a pig kidney transplant. Pig organs come in sizes and shapes like ours, but they don’t go straight from the farm to the operating room. The animals are bred using genetic engineering that makes them produce proteins that human bodies won’t reject. This could help increase the supply of organs that people desperately need.

Since receiving the kidney at Mass General Hospital, Andrews has held the record for living the longest with a pig organ in his body. In June, 54-year-old Bill Stewart received a new pig kidney, also at Mass General. And in August, researchers in China reported another milestone: the first pig-human lung transplant.

10. AI Drug Innovations

Researchers are using AI to figure out whether your next life-changing treatment is already sitting on a pharmacy shelf. “We typically think that the drugs we have are being used for all the diseases they can treat,” says David Fajgenbaum, MD, of the University of Pennsylvania. But a lot of drugs could help a lot more people.

Dr. Fajgenbaum’s team uses AI knowledge graphs to scan global research results. He likens it to the tech that TV streaming services use to recommend shows based on your viewing habits, assigning a score to a program to indicate how well it matches what you’ve already watched. In science, AI rates the strength of the match between drugs and diseases.

Success so far: AI predicted that a drug used for autoimmune disorders, adalimumab, would work in idiopathic multicentric Castleman disease (iMCD), a disorder that inflames the lymph nodes. Dr. Fajgenbaum’s team checked tissue samples from people with severe iMCD and found that a protein inhibited by adalimumab—tumor necrosis factor—was elevated. They recommended adalimumab to a man with iMCD who was about to enter hospice, with no hope of recovery. He recovered and has been healthy for the past two years.