China’s First CRISPR Babies

Over the past decade, any regular consumer of medical news will have noticed the dramatic increase in reports surrounding CRISPR and gene editing technologies. From the host of potential applications showing promise in the lab to the first FDA-approved CRISPR treatment for sickle cell disease, the buzz surrounding the new technology seems to suggest it’s unstoppable, and quite rightly so, having the ability to target and alter the specific genes responsible for certain conditions is a huge leap forward in medical technology.

But it’s not all been smooth sailing, and unfortunately, the increasing hype took a bit of a hit to the image recently when a controversy from the no-so-distant past thrust it back into the limelight.

But the story starts several years before in 2018 when study leader, He Jiankui, a Chinese biophysicist, first attempted to publish his manuscript describing the birth of the HIV resistant twins; titled the “the Birth of Twins After Genome Editing for HIV Resistance.”

The research published by He’s so-called team had other experts calling out the possibility of manipulating data and/or faking results. In an op-ed by Kiran Musunuru, a gene-editing specialist at the University of Pennsylvania, Musunuru noted that the data showed gene-editing for reproduction was still premature and unsafe.

Of the ten authors, there are no officially licensed doctors. The only accredited American biophysicist, Michael Deem, sought to have his name removed from the paper, claiming he never agreed to submit the manuscript.

There were many other damning bits of evidence to suggest the information wasn’t entirely truthful. Together with unsafe procedures, there were signs that gene edits were not the same as the mutations noted, certain claims contradicted themselves, data had been misrepresented to show births took place, data claiming overall efficiency is thought to have been exaggerated, and parents of participating children may have been encouraged to take part for the wrong reasons, with uncertain promises.

The Chinese government wasn’t about to let Jiankui go for his actions; they sentenced him to three years’ imprisonment, and enacted a new law prohibiting the modification of human embryos for reproductive purposes,

Jiankui’s was released from prison in April, hence all the murmurs rising once again throughout the industry, especially since he plans to continue his journey into developing treatments utilizing gene therapy. He aims to make the technology more affordable, and ultimately treat more rare diseases and difficult to treat diseases, using these treatments.

Not Giving Up on CRISPR

Despite previous issues, He vows to continue to utilize gene-editing to find a cure for rare diseases…

It goes without saying that receiving bad press regarding ethics is a major no-no in the medical community, and although the controversy does highlight a number of potential ethical problems, it’s unlikely one parties actions will do much to tarnish a technology that is already being touted, tested and trialed by thousands, potentially even hundreds of thousands, of repeatable researchers worldwide.

chinese crispr babies
CRISPR Research

Gene editing has experienced a dramatic rise in interest largely thanks to the recent approval from the FDA for treatments for sickle cell disease and transfusion-dependent beta-thalassemia (TDT) in patients ages 12 and older, which essentially gives the green light for all those conducting similar research to continue their efforts knowing the road has already been paved, and that any effective treatment with sufficient supporting evidence can now follow the same journey on their way to market.

In developing these treatments and receiving FDA approval, the novel technique has quickly shifted from a concept showing promise as an effective treatment for all manner of rare diseases into a treatment that’s finally a reality for those living with the most uncommon of health conditions.

That said, it’s important to note the disparity in objectives held by key players at the forefront of developing the industry so as to see when these different ideas converge, for better or worse. For example, while scientists and researchers typically have ethical reasons driving their efforts, the companies that manufacture the treatments are often only motivated by finances.

These opposing desires raise a rather significant hurdle when treating rare diseases. Since the very nature of these rare conditions means fewer patients, any treatments developed will also be in low demand.

In addition to these business orientated hurdles, the actual challenge of developing treatments to combat rare diseases is by no means an easy one, largely thanks to the lack of documentation, as well as very the very few opportunities to conduct first hand research by studying patients with rare diseases.

Coincidentally, or perhaps quite ingeniously, He’s chosen plight to utilize gene-editing techniques to develop new treatments for rare diseases doesn’t just tackle the challenge of treating rare medical conditions; He’s unique situation simultaneously draws an air of attention unmatchable by others, even famous scientist-researchers, which in turn raises the awareness and support that’s essential to the development of effective treatments.

Should his new undertakings fail to change the opinions of experts, the sheer number of promising findings from other leading experts in the industry will likely quickly eclipse the one or two pitfalls encountered along the way.

Regardless of how the situation plays out, it’s almost certain that CRISPR technology is here to stay, and will no doubt see more potential treatments become fully fledges cures.

With the increased awareness regarding the technology’s true potential, it shouldn’t be long before we see widespread adoption of gene-editing treatments and, with them, a wider range of effective applications.

It’s no secret that cosmetic surgery accounts for a larger percentage of the medical industry, and since gene-editing has the ability to perform many cosmetic treatments, there’s no doubt the technology will be applied to such procedures, however, the most exciting breakthrough remains; the ability to effectively treat many elusive rare diseases.

And perhaps, if manufacturers attempt to even out any disparities from the beginning, it could be possible to create an environment where the popularity of cosmetic procedures is leveraged to subsidize the development of treatments for rare diseases, which in turn would raise a company profile and allow them to maximize the exposure and potentially recover any financial losses experienced from developing treatments for much those smaller patient populations.

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