Brave new world or dark threatening future: a clear-eyed look at genome editing and what it means for humanity

Frankenstein

   Is this the face of the future?

“Have you ever wished that there were something different about yourself? Maybe you imagined yourself taller, thinner or stronger? Smarter? More attractive? Healthier?”

That’s the question posed by UC Davis stem cell researcher (and CIRM grantee) Paul Knoepfler at the start of his intriguing new book ‘GMO Sapiens: The Life-Changing Science of Designer Babies’.

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You can find GMO Sapiens on Amazon.com

The book is a fascinating, and highly readable, and takes a unique look at the dramatic advances in technology that allow us to edit the human genome in ways that could allow us to do more than just create “designer babies”, it could ultimately help us change the definition of what it means to be human.

Paul begins by looking at the temptation to use technologies like CRISPR (we have blogged about this here), to genetically edit or alter human embryos so that the resulting child is enhanced in some ways. It could be that the editing is used to remove a genetic mutation that could cause a deadly disease (such as the BRCA1 gene that puts women at increased risk of breast and ovarian cancer) or it could be that the technique is used to give a baby blue eyes, to make it taller, more athletic, or to simply eliminate male pattern baldness later in life.

Paul says those latter examples are not as ridiculous as they sound:

Paul Knoepfler

Paul Knoepfler

“If you think these ideas sound far-fetched, consider that Americans alone spend tens of billions of dollars each year on plastic surgery procedures and creams to try to achieve these kinds of goals. Some of the time elective cosmetic surgery is done on children. In the future, we might have “cosmetic genetic surgeons” who do “surgery” on our family’s genes for cosmetic reasons. In other countries the sensibilities and cultural expectations could lead to other kinds of genetic modifications of humans for “enhancements”.

While the technology that enables us to do this is new, the ideas behind why we would want to do this are far from new. Paul delves into those ideas including a look at the growth of the eugenics movement in the late 19th and early 20th century advocating the improvement of human genetic traits through higher reproductive rates for people considered “superior”. And there was a darker side to the movement:

“Indiana had instituted the first law for sterilization of “inferior” people in the world in 1907. Astonishingly this state law and then similar laws (the original was revoked, but a new law was passed later) stayed on the books in that state until 1974.

This led to approximately 2,500 governmentally forced sterilizations. The poor, uneducated, people of color, Native Americans, and people with disabilities were disproportionately targeted.”

Paul explores the ethical and moral implications of changing our genetic code, changes that can then be passed on to future generations. While he understands the desire to use these technologies to create positive changes, he is also very clear in his concerns that we don’t yet have enough knowledge to be able to use them in a safe manner.

“CRISPR can literally re-write the genomic book inside of us. However, it remains unknown how often it might go to the wrong page or paragraph, so to speak, or stay on the right page, but make an undesired edit there.”

Tiny errors in editing the genome, particularly at such an early stage in an embryo’s development, could have profound and unintended consequences years down the road, resulting in physical or developmental problems we can’t anticipate or predict. For example, you might remove the susceptibility to one disease only to create an even larger problem, one that is now embedded in that person’s DNA and ready to be passed on to subsequent generations.

The book includes interviews with key figures in the field – scientists, bioethicists etc. – and covers a wide range of views of what we should do. For example, the Director of the US National Institutes of Health (NIH), Francis Collins, said that designer babies “make good Hollywood — and bad science,” while the Center for Genetics and Society has advocated for a moratorium on human genetic modification in the US.

In contrast, scientists such as Harvard professor George Church and CRISPR pioneer Jennifer Doudna of UC Berkeley, say we need to carefully explore how to harness the potential for these technologies.

For Church it is a matter of choice:

“The new technology enables parents to make choices about their children just as they might with Ritalin or cleft palate surgery to ‘improve’ behavior or appearance.”

For Doudna it’s acknowledging the fact that you can’t put the genie back in the bottle:

“There’s no way to unlearn what is learned. We can’t put this technology to bed. If a person has basic knowledge of molecular biology they can do it. It’s not realistic to think we can block it…We want to put out there the information that people would need to make an informed decision, to encourage appropriate research and discourage forging ahead with clinical applications that could be dangerous or raise ethical issues.”

The power of Paul’s book is that while it does not offer any easy answers, it does raise many important questions.

It’s a wonderfully well-written book that anyone can read, even someone like me who doesn’t have a science background. He does a good job of leading the reader through the development of these technologies (from the basic idea of genetically altering plants to make them disease resistant) to the portrayal of these concepts in literature (Frankenstein and Brave New World) to movies (Gattaca – 4 stars on Rotten Tomatoes  a great film if you haven’t already seen it).

It’s clear where Paul stands on the issue; he believes there should be a moratorium on human genetic modification until we have a much deeper understanding of the science behind it, and the ethics and morality underpinning it:

“This is a very exciting time to be alive and we should be open to embracing change, but not blindly or in a rush. Armed with information and passion, we can have a major, positive impact on how this biotech revolution unfolds and impacts humanity.”

By the way, Paul also has one of the most widely read blogs about stem cells, where you can read more about his thoughts on CRISPR and other topics.

 

Patients beware: warnings about shady clinics and suspect treatments

stem-cells therapy?

Every day we get a call from someone seeking help. Some are battling a life-threatening or life-changing disease. Others call on behalf of a friend or loved one. All are looking for the same thing; a treatment, better still a cure, to ease their suffering.

Almost every day we have to tell them the same thing; that the science is advancing but it’s not there yet. You can almost feel the disappointment, the sense of despair, on the other end of the line.

If it’s hard for us to share that news, imagine how much harder it is for them to hear it. Usually by the time they call us they have exhausted all the conventional therapies. In some cases they are not just running out of options, they are also running out of time.

Chasing hope

Sometimes people mention that they went to the website of a clinic that was offering treatments for their condition, claiming they had successfully treated people with that disease or disorder. This week I had three people mention the same clinic, here in the US, that was offering them “treatments” for multiple sclerosis, traumatic brain injury and chronic obstructive pulmonary disease (COPD). Three very different problems, but the same approach was used for each one.

It’s easy to see why people would be persuaded that clinics like this could help them. Their websites are slick and well produced. They promise to take excellent care of patients, often helping take care of travel plans and accommodation.

There’s just one problem. They never offer any scientific evidence on their website that the treatments they offer work. They have testimonials, quotes from happy, satisfied patients, but no clinical studies, no results from FDA-approved clinical trials. In fact, if you explore their sites you’ll usually find an FAQ section that says something to the effect of they are “not offering stem cell therapy as a cure for any condition, disease, or injury. No statements or implied treatments on this website have been evaluated or approved by the FDA. This website contains no medical advice.”

What a damning but revealing phrase that is.

Now, it may be that the therapies they are offering won’t physically endanger patients – though without a clinical trial it’s impossible to know that – but they can harm in other ways. Financially it can make a huge dent in someone’s wallet with many treatments costing $10,000 or more. And there is also the emotional impact of giving someone false hope, knowing that there was little, if any, chance the treatment would work.

Shining a light in shady areas

U.C. Davis stem cell researcher, CIRM grantee, and avid blogger Paul Knoepfler, highlighted this in a recent post for his blog “The Niche” when he wrote:

Paul Knoepfler

Paul Knoepfler

“Patients are increasingly being used as guinea pigs in the stem cell for-profit clinic world via what I call stem cell shot-in-the-dark procedures. The clinics have no logical basis for claiming that these treatments work and are safe.

As the number of stem cell clinics continues to grow in the US and more physicians add on unproven stem cell injections into their practices as a la carte options, far more patients are being subjected to risky, even reckless physician conduct.”

As if to prove how real the problem is, within hours of posting that blog Paul posted another one, this time highlighting how the FDA had sent a Warning Letter to the Irvine Stem Cell Treatment Center saying it had serious concerns about the way it operates and the treatments it offers.

Paul has written about these practices many times in the past, sometimes incurring the wrath of the clinic owners (and very pointed letters from their lawyers). It’s to his credit that he refuses to be intimidated and keeps highlighting the potential risks that unapproved therapies pose to patients.

Making progress

As stem cell science advances we are now able to tell some patients that yes, there are promising therapies, based on good scientific research, that are being tested in clinical trials.

There are not as many as we would like and none have yet been approved by the FDA for wider use. But those will come in time.

For now we have to continue to work hard to raise awareness about the need for solid scientific evidence before more people risk undergoing an unproven stem cell therapy.

And we have to continue taking calls from people desperate for help, and tell them they have to be patient, just a little longer.

***

If you are considering a stem cell treatment, the International Society for Stem Cell Research had a terrific online resource, A Closer Look at Stem Cells. In particular, check out the Nine Things to Know about Stem Cell Treatments page.

 

British Parliament votes to approve “three parent” baby law

After what is being described as “an historic debate”, the British Parliament today voted to approve the use of an IVF technique that critics say will lead to the creation of “three parent” babies.

UK Parliament

UK Parliament

Parliament voted 382 to 128 in favor of the technique known as mitochondrial donation, which will prevent certain genetic diseases being passed on from parents to children; diseases that can cause a wide range of conditions such as fatal heart problems, liver failure, brain disorders and blindness.

Mitochondrial donation involves replacing a small amount of faulty DNA from a mother’s egg with healthy DNA from a second woman. The technique involves taking two eggs, one from the mother and another from the donor. The nucleus of the donor egg is removed, leaving the rest of the egg contents, including the mitochondria. The nucleus from the mother’s egg is then placed in the donor egg. This means that the baby would have genes from the mother, the father and the female donor.

The vote makes the UK the first country in the world to endorse this process. It comes at the end of what supporters of the measure described in a letter to Parliament as “seven years of consultation and inquiry that have revealed broad scientific, ethical and public approval.”

Mitochondrial donation is a controversial process opposed by many religious and faith-based groups who say it creates “designer babies” because it involves implanting genetically modified embryos, and because it could result in genetic alterations that might be passed on to subsequent generations.

While many scientists support the technique some have raised concerns about it. Among those are Dr. Paul Knoepfler, a stem cell researcher at U.C. Davis, (CIRM is funding some of his work). In a recent blog on the process Paul wrote that while he is not opposed to the technique in theory, he thinks this move at this time is premature:

“There is no doubt that mitochondrial diseases are truly terrible and need to be addressed, but if the potential outcomes from the technology are still vague, there are safety concerns, and it raises profound ethical issues such as changing the human genome heritably as is the case here, then my view is that a careful approach is both practical and logical. We cannot at this time have a reasonable expectation that this technology would be safe and effective. That may change in coming years with new knowledge. I hope so.”

Supporters in the UK say the science is already good enough to proceed. Dame Sally Davies, Britain’s Chief Medical Officer, calls it the genetic equivalent of “changing a faulty battery in a car.”

Professor Lord Winston, a fertility expert at Imperial College, London, says:

“I think the case is self-evident and reasonable. This is about something that is unusual and will benefit a small number of patients. I know there are some people who think it is a slippery slope that the next thing will be choosing intelligence or blond hair, but I don’t think that. For 20 years, it’s been scientifically possible to have sex selection of embryos; we still don’t allow it in Britain apart from for heritable diseases.”

It’s important to point out that while the House of Commons passed the regulations they still have to be approved by the House of Lords before they become law. A vote is scheduled for the end of this month. Even then any future trial involving the technique will still require the approval of the Human Fertilisation and Embryology Authority (HFEA) before it can go ahead.

Even if the process is ultimately approved in the UK it will likely face an uphill battle to be approved here in the U.S. where the debate over the ethical, as well as the scientific and technical implications of the process, has already generated strong feelings on both sides of the divide.

A Christmas miracle or untested therapy? Why even feel-good stem cell stories need to be checked for accuracy

We’ve written several pieces over the last couple of years about the trend for professional athletes to turn to untested and/or unproven stem cell therapies to help them bounce back from injuries. This week, however, came news of something a little more worrying. Ice hockey legend Gordie Howe was given stem cells to help him recover from a series of debilitating strokes. As is often the case with these stories it’s not just the nature of the treatment that raises questions, it’s also the way the media has covered it.

Gordie Howe - photo courtesy Sean Hagen from Maple Ridge, Canada

Gordie Howe – photo courtesy Sean Hagen from Maple Ridge, Canada

The facts are pretty straightforward. Howe’s strokes left him “essentially bedridden with little ability to eat or communicate on his own”, according to a statement issued by his family. Two companies – Stemedica and Novastem – then “volunteered” their services, delivering a stem cell therapy to Howe. According to the family “The response was truly miraculous.”

And that was often the extent of the digging that dozens of media outlets that reported the news did. They reported the facts of the stroke, and then just reprinted the statement from the family without questioning what kinds of cells, how they might work, etc etc. They didn’t bother to interview other stem cell scientists about this kind of approach to see if it was something that might benefit other stroke patients. They didn’t even take a closer look at the two companies involved to see what their track record on this kind of research is.

In short, it’s clearly a feel-good story about a sports legend and no one wanted to be the one to say, “hey, wait a minute here, how do we know this is real.”

No one, except Dr. Paul Knoepfler. Paul, as regular readers of this blog know, is a CIRM-funded stem cell researcher at the University of California, Davis and an avid blogger. In a post on his blog he took a much closer look at the story, posed some thoughtful questions and raised some doubts about it. He also reached out to Stemedica who, to their credit, responded promptly to his questions. You can read what they had to say here.

Paul, like the rest of us, would love to be able to say that this kind of approach worked for Gordie Howe and could work for millions of others left disabled by strokes. But Paul, unlike many news outlets that reported the story, isn’t willing to just accept it on face value.

There’s an old adage in journalism: “If your mother tells you she loves you, check to see if it’s true.” It basically means don’t accept anything on face value; dig a little deeper to see if it’s really true. Paul is doing that, and doing it very well. Other journalists might do well to follow his lead.

Milton Berle and the Art of Writing an Award-Winning Stem Cell Essay

It’s not often that you hear famous comedian Milton Berle quoted in an essay about stem cells, but then U.C. Davis researcher and avid blogger Paul Knoepfler wasn’t looking for an ordinary essay as the winner for his recent contest.

Knoepfler launched the contest as a way to give some lucky individual a free registration to the International Society for Stem Cell Research (ISSCR) annual conference in Vancouver, Canada later this month.

Essay winner Mohamed Gatie

Essay winner Mohamed Gatie

The winning entry came from a student Mohamed Gatie. Here’s his winning essay.

The second place essay came from Sherry Hikita. We met Sherry at the World Stem Cell Summit in San Diego last year where she signed up as one of our Stem Cell Champions.

Second-place winner Sherry Hikita

Second-place winner Sherry Hikita

Congratulations to both Mohamed and Sherry, and of course kudos to Paul for coming up with the idea and helping a fine student get a chance to go to one of the biggest events of the year for a stem cell scientist.

kevin mccormack

Write your Ticket to the Stem Cell Science Event of the Year

For many stem cell scientists one of the highlights of the year is going to the annual International Society for Stem Cell Research (ISSCR) conference. It’s like Disneyland for researchers. This year the event is being held in Vancouver, Canada. But many younger scientists won’t be going because they can’t afford it. So UC Davis researcher and avid blogger Paul Knoepfler has teamed up with ISSCR to help one lucky scientist go. Here’s Paul’s guest blog explaining how they can apply:

What’s better than a four-day stem cell meeting held in beautiful Vancouver, B.C. that includes all of the newest and most cutting edge research?

If, like me, you are a lover of all things stem cell your first impulse may be to shout out “nothing!” and I am betting you will already know that I am talking about the ISSCR Annual Meeting being held this year in that fantastic locale.

Beautiful Vancouver, British Columbia home to this year’s ISSCR Annual Meeting

Beautiful Vancouver, British Columbia home to this year’s ISSCR Annual Meeting

However, the answer I had in mind is that the only thing better than going to ISSCR is getting to go to that meeting for free.

How do you get to that stem cell happy place?

I have teamed up with ISSCR to run a contest with the prize being free registration to the ISSCR meeting that runs from June 18-21.

All you have to do is write a short essay addressing this topic: why do I want to go to ISSCR 2014?

The winner gets free registration and their essay gets published on my blog to be read by the stem cell community.

For a few additional rules and details, check out the announcement of the contest here.

The deadline for entries is Friday May 30th. The winner will be announced on Monday June 2nd.

What am I looking for in a winning essay? As a stem cell researcher and writer myself, I am hoping to get entries that are clever, creative essays that convey genuine, interesting reasons for wanting to go to this great meeting. You will have to be concise too because the limit on length is a strict 300 words.

There may be additional entries published on my blog if more than one really wows me, and other perks like stem cell swag for entrants could come into play depending on the number of entrants and the quality of their writings.

So start brainstorming and writing and then send your entries to me at knoepfler@ucdavis.edu.

I hope to see you in Vancouver.

Paul Knoepfler