On a flight not too long ago, I came across a Time magazine story about gene editing and a potential future in which humans could cut out the bad parts of our DNA in order to avoid health conditions like diabetes and its complications.

This may seem like a futuristic take on the current state of research, but it’s not a stretch to think it could be possible some day soon. In fact, work is already in motion on using gene editing in research toward a "biological" diabetes cure. No kidding!

On Sept. 18, international biopharm company CRISPR Therapeutics and San Diego-based regenerative medtech company ViaCyte announced their collaboration using gene editing to supplement islet cell encapsulation, which has the potential to protect the transplanted beta cells from the inevitable immune system attack that normally kills them off.

Remember, ViaCyte is the startup that's been working for years on an implantable device that encapsulates newly-developed insulin-producing cells that can take hold within a person's body to start regulating glucose and insulin again. Their Encaptra device has made headlines and created a lot of excitement in the D-Community, especially in the past year when ViaCyte finally received FDA OK for its first human clinical studies.

According to Time, the five-year-old concept CRISPR-Cas9 “is transforming research into how to treat disease, what we eat and how we'll generate electricity, fuel our cars and even save endangered species. Experts believe that CRISPR can be used to reprogram the cells not just in humans but also in plants, insects -- practically any piece of DNA on the planet.”

Wow! Now, working together, the two companies state, "We believe the combination of regenerative medicine and gene editing has the potential to offer durable, curative therapies to patients in many different diseases, including common chronic disorders like insulin-requiring diabetes."

But just how real is this? And how much hope should we PWDs (people with diabetes) hang on the concept of gene editing for ending our disease?

 

Reprogramming DNA

The idea of gene editing of course is aiming to "reprogram our DNA" -- the basic building blocks of life.

Researchers at MIT and Harvard’s Broad Institute are developing a tool that can reprogram how parts of DNA regulate and express themselves – potentially paving the way for gene manipulation to prevent chronic conditions.

There are ethical considerations with all of this, to be sure. An international committee of the U.S. National Academy of Sciences (NAS) and the National Academy of Medicine in Washington, DC, issued a report in early 2017 that basically gave a yellow light to proceed with embryonic gene editing research, but on a cautionary and limited basis. The report noted that this kind of future human gene editing might be allowed someday, but only after much more risk-benefit research and “only for compelling reasons and under strict oversight.”

It’s anyone’s guess what that might mean, but one thought is it could be limited to couples who both have a serious genetic disease and whose only last resort to have a healthy child might be this kind of gene editing.

As for gene editing in the cells of patients living with diseases, clinical trials are already underway on HIV, hemophilia and leukemia. Existing regulatory systems for gene therapy are good enough to oversee that work, the committee found, and while gene manipulation “should not proceed at this time," the committee panel did say research and discussions should continue.

This type of gene editing research is well underway on a number of fronts, including some diabetes-specific projects:

  • This story from December 2016 reports that at the Lund University Diabetes Centre in Sweden, researchers have used CRISPR to “turn off” one of the genes thought to play a part in causing diabetes, effectively decreasing beta cell death and increasing insulin production in the pancreas.
  • At New York City's Memorial Sloan Kettering Cancer Center, biologist Scott Lowe is developing therapies that turn genes in tumor cells on and off to make them easier for the immune system to destroy.
  • Malaria researchers are exploring a number of ways that CRISPR can be used to manipulate mosquitoes to make them less likely to transmit the malady; the same is happening with mice that transmit bacteria causing Lyme disease.
  • This 2015 research concludes that this type of gene editing tool will become more precise and help us better understand diabetes in years to come, and a recent study published in March 2017 shows the promise of gene therapy using this technique, for potentially curing T1D someday (!), though it's only been studied in animal models, so far.

Also on the diabetes research front, programs like TrialNet are aggressively seeking certain autoimmune biomarkers to trace the genetics of T1D through families, in order to target early treatment and even future prevention.

Meanwhile, these new gene editing methods are reported to have potential to improve the health and welfare of food producing animals -- for example, hornless cattle, pigs resistant to African swine fever or porcine reproductive and respiratory virus -- and alter specific traits of food plants or fungi, like non-browning mushrooms, for example.

 

A Biological vs. Functional Cure for Diabetes

Prior to the collaboration with CRISPR, ViaCyte's approach was referred to as a "functional cure," because it could only replace the missing insulin cells in a PWDs body, but not address the autoimmune roots of the disease. But working together, they can do both, to pursue a true "biological cure."

"The combined power of this collaboration lies in the expertise from both companies," ViaCyte President and CEO Dr. Paul Laikind tells us.

He says the collaboration is still in early stages, but is an exciting first-step on the path to creating a stem-cell derived product that can resist immune system attack -- basically by reworking the cells' DNA to evade the immune system attack.

OK, we can't help noting how reminiscent this all is of the novel Brave New World and the designer babies controversy, making one ponder the ethics: Is it right to meddle with aspects of humanity we don’t like, to strive for health “perfection”?  

Not to get too deep into politics or religion here, but clearly we all want a cure for diabetes, and other diseases. Yet are we willing (or required) to "play God" to get there? Food for thought, to be sure.