Why my patient advocacy organization is investing in CRISPR

In last year’s action film “Rampage,” which featured Dwayne “The Rock” Johnson, CRISPR gene-editing technology turns a gorilla, a wolf, and a crocodile into monsters the size of skyscrapers that destroy Chicago.

If you missed the movie, you may have caught wind of He Jiankui. Near the end of 2018, the Chinese scientist reported having used CRISPR to produce the first-ever gene-edited babies. (He currently faces criminal charges in his home country for violating national guidelines, not to mention global scientific ethical standards.)

As the mother of a son with Duchenne muscular dystrophy, a cruel genetic disease that breaks down muscle until the body can no longer function, I don’t see CRISPR as scary or dangerous. To me, it represents the best hope we have for curing my son’s disease and possibly many others. I believe CRISPR is a weapon — not a giant gorilla type of weapon — but a quiet, surgically precise weapon against diseases that have long confounded the scientific community.

I believe so strongly in CRISPR’s potential to change the world that I put my money where my mouth is. Understanding that academic research moves slowly, I founded the advocacy organization CureDuchenne to support new and exciting technologies that will challenge the status quo through venture philanthropy. We invest capital in research and development of cutting-edge therapies, including CRISPR.

We’re not in the business of developing drugs, but rather identifying the best science and funding the team. That’s exactly what happened when we met Dr. Eric Olson at the University of Texas Southwestern and provided seed funding for his company, Exonics Therapeutics, the first company dedicated to finding a cure for Duchenne through CRISPR.

In a preclinical study last year, Olson and his team restored 92 percent of dystrophin — a protein that helps keep muscle cells intact and that Duchenne depletes — in animal hearts, inspiring hope for human trials in the near future. This CRISPR-based treatment is delivered to the tissues of individuals affected by Duchenne, affecting that person only and not his children.

In contrast, He edited embryos, meaning the twin girls that were born can pass their genetic alterations to their offspring. His goal was to make their cells less susceptible to the virus that causes AIDS, but there’s no way to know if it worked, or if it might make them more susceptible to other viruses, or other diseases. Removing dangerous mutations from the human germline may someday be part of medicine, but that method simply hasn’t been proven to be safe.

CRISPR is still a new technology that demands a high level of respect and caution. This is why no reputable scientists have tested CRISPR in humans yet, though other gene-editing technologies have been used in much the same way Olson and his colleagues could eventually use CRISPR.

If anything goes wrong with the twin girls in China, it could have ramifications that set back the entire field. Investors might balk at companies researching CRISPR, fearing negative publicity, and Congress or the FDA might limit research into CRISPR in response to He’s reckless actions.

We need to ask ourselves what CRISPR should be used for. I think of it in much the same way that I think of an eraser on a pencil: correcting genes like correcting misspelled words on a page. We should use this technology to fix the most dangerous mutations that have no cure. That includes 15,000 people in the U.S. — 300,000 around the world — who suffer from Duchenne, a common form of muscular dystrophy that mainly affects boys. It could someday be applied to the 10,000 or so genetic diseases that afflict 75 million people around the globe, or even to chronic diseases like cancer and diabetes that affect billions of people.

I oppose “designer” gene editing — making changes to perfectly normal genes for the sake of changing appearance or improving intelligence or creating immunity to a virus. There is no medical need, it wastes valuable resources, and I question the motivation behind it. Fixing broken genes should take precedence over altering healthy ones.

Used wisely, CRISPR has the potential to wipe out terrible diseases. That, in turn, would lower health care costs across the board and eliminate the need for patients to buy medications that can cost hundreds of thousands of dollars a year. (A new drug for Duchenne muscular dystrophy, for example, costs at least $300,000 a year.) If CRISPR proves to be safe, it could have a dramatic impact on many diseases.

That is, of course, if we hear more about CRISPR’s positive impact than about genetically edited twins or former wrestlers and a city threatened by giant mutant animals.

Debra Miller is the CEO and founder of CureDuchenne, the global leader in research, patient care, and innovation for improving and extending the lives of children with Duchenne muscular dystrophy.