Recommended Weekend Science Reads

The pieces on my radar screen to read in my ample spare time as I work the weekend on literally 5 different grants include these pieces:

The research articles:

Monday, March 2: My Reddit AMA on 3-Person IVF – Mitochondrial Transfer

reddit-logoOn Monday, I’ll be doing another Reddit AMA, this time on 3-person IVF also known as 3-parent IVF, mitochondrial transfer, 3-parent babies, etc.

You can see the upcoming AMAs including mine listed in the right sidebar here.

Mine will start at 10AM PST/1PM EST.

I hope to discuss with a wide audience all the key issues surrounding this exciting, but highly controversial technology just legalized in the UK. I will answer as many questions as possible.

Here’s what the AMA intro will say:

Science AMA Series: Dr. Paul Knoepfler | 3-Parent Babies/Mitochondrial Transfer
Hi, I’m Paul Knoepfler, stem and cancer cell biologist and genomics researcher, author, & blogger. I have been closely following the recent development of 3-person IVF/mitochondrial transfer also sometimes referred to as 3-parent baby technology.
It’s a really intriguing, hot topic right now. In the UK, the first 3-parent baby (hopefully prevented from having mitochondrial disease) could be conceived as early as sometime this year. The technology is current prohibited in the US by the FDA, which looks to be at a minimum 2-5 years away from even possibly approving it with the delay meant to give more time to get data on safety and efficacy.
By way of disclosure, I’ve been advocating for waiting for more data before proceeding, but I am not in principle opposed to the technology when the time is right. Most UK scientists support moving forward on this technology asap.
Is this technology ready for prime time? Or is the US right to be more cautionary. Is it safe and would it be effective in preventing mitochondrial diseases? Since this technology would also create genetically modified humans, what bioethical issues should be discussed?
It brings into play many cutting edge, timely issues such as assisted reproduction, cloning, genomics technologies such as sequencing and gene editing, GMOs, and more.
You might want to check out my blog at http://www.ipscell.com and my new book on stem cells, Stem Cells: An Insider’s Guide
You can also want follow me on Twitter @pknoepfler if you like for all the latest, I will be answering questions starting at 1 PM EST (10 AM PST) Ask Me Anything!

Supreme Court Rejection of WARF hESC Patent Challenge Discussion

The challenge to the WARF/Jamie Thomson patents/IP on human embryonic stem cells (hESC) is at an end. The US Supreme Court declined to hear the case.

Earlier, the USPTO had turned down the challenge leading to a winding road in the courts.

What do you all think of this stem cell IP challenge and the outcome?

The hESC IP includes 3 patents: 5,843,7806,200,806, and 7,029,913.

For two great stories with helpful background on this development see here and here by David Jensen and Bradley Fikes, respectively. Scripps stem cell researcher Jeanne Loring was a driving force in the challenge.

UK OKs 3-Person IVF: Perspectives

With the House of Lords vote yesterday, the UK officially became the only country in the world to have legalized human genetic modification. The point of this “mitochondrial transfer” or “3-person IVF” technology is not directly to genetically modify humans specifically, but it still will lead to that result in any case.

The noble goal of this experiment is to prevent mitochondrial disease, but it is not quite that simple as the children produced (if any healthy children are produced) would have genomic elements from 3 human beings even if one of them contributes about 1000 times less than the other two. I would still call that a genetic modification. For example, plants and animals with even a single gene transferred to them from elsewhere are defined as GMOs.

Now we can hope that this technology is safe and effective, but we can and should do more than hope.

We can continue to ask for data, transparency, and accountability as this work proceeds. I’m not so sure that these elements are part of the law that passed as some had said that reporting of outcomes would not be required. Does anyone know for sure?

The proponents and practitioners-to-be of this work in the UK have, to use their own words yesterday, “won”. As they proceed with this experiment, I believe that they should account for every single mitochondrial transfer embryo implanted in women using this technology and publicly report the outcome data for each. Healthy birth? Miscarriage? Developmental Disorder? Problem in childhood later on? Genome integrity?

It is medically and scientifically crucial for the scientists and doctors involved to collect and publicly report all this data in a timely manner. This can be done while still protecting the privacy of the families involved.

I hope very single one is a healthy child, but as with all experiments there is no way to be sure in advance.

Red flags in Sally Davies letter on 3-person IVF mitochondrial transfer

There’s been some back and forth between me and some members of the UK Parliament and others in the UK on the issue of mitochondrial transfer 3-person IVF technology.

Some of this has involved the UK equivalent of the Surgeon General, Professor Dame Sally Davies, who at one point apparently called my views “bunk”. I wrote back to her via Lord Alton articulating my concerns and respectfully responding to the bunk allegation.

I am not opposed in principle to mitochondrial transfer technology, but rather from an impartial scientific and factual perspective it is clear to me that far more data from additional animal studies is needed. At some point with any experimental biotechnology intended for human use scientists and doctors have to take the plunge and try it in humans, but my view is that the animal data supporting mitochondrial transfer is incomplete at best.

Now I just recently indirectly received a letter from Davies, which seemed intended to rebut my concerns. I found some things about the letter to be surprising and in some cases to be red flags.

With all due respect, Davies seems to have a dismissive attitude toward those who disagree with her on the urgency for human mitochondrial transfer to proceed as soon as possible. This is evident in a newspaper article she wrote and also in this letter. You can see it without having to read between the lines in her word choices to describe the press and opponents of the immediate approval of the technology: ‘overly simplistic’, ‘sensationalist’, ‘unsophisticated’.

The most surprising element of her letter in reply to my concerns was her argument against a need for additional animal research and data.

It seems she and other proponents of this technology moving forward immediately have a tendency to pick and choose whether to value animal data depending on whether it supports their case or not. In this passage, they are fans of animal data, which they view as fairly definitive:

“The view of the Expert Panel was that experiments involving Pro-nuclear Transfer (PNT) and Maternal Spindle Transfer (MST) in animals (mice, macaques, and some other animals) have not given any cause for concern.”

So on the one hand she and others advocating immediate approval of this technology have asserted that mitochondrial transfer would be safe for humans based essentially entirely on animal studies including the non-human primate work of Mitalipov.

On the other hand, she goes on to argue that additional work of that kind would be bad:

“The use of non-human primate experiments was deemed by the Expert Panel, in its 2013 and 2014 reports, to no longer be necessary given the differences between non-human primate and human eggs / early embryos. Performing such unnecessary animal experiments would indeed be unethical given that non-human primates were not considered a good model for the human in this context.”

It sure seems like a self-contradictory argument, right?

I find the use of the word ‘unethical’ to be strikingly strong.

It is notable that in the past, if memory serves, the Expert Panel did feel that more data was needed (and that data is still not available), but it’s not clear what changed their minds.

Remarkably, then in a nutshell what she seems to be saying is that the only ethical thing to do since animal models are imperfect–except of course the specific past ones that we feel supported safety–is to experiment on humans and do it now.

That is more ethical?

This all sounds a lot more like politics than biomedical science. I say, let’s focus on the data and not the politics, but I’m not holding my breath.