Thursday, May 18, 2006

Lanza: Sometimes I spend more time on the phone with lawyers than I do on the science

From an article in MIT Technology Review:

[ACT's] Lanza suspects that, because of the shortage of eggs and the unknown efficiency of the cloning process, the therapeutic use of cloned stem cells will end up looking more like a kidney transplant than like the ingestion of a widely prescribed drug. "We do recognize it's not the broad cure we had hoped, but I'm convinced it will save some individuals," he says. "Perhaps a mother would donate a round of eggs to create stem cells for her sick child." IPBiz note: that is, the use of therapeutic cloning is not going to be like a "blockbuster drug" for big pharma/biotech. The whole idea is to make genetically-customized cells for a single end user, not one drug that a zillion people all need to take. This basic reality suggests that patent strategy for [therapeutic] embryonic cloning is quite different from that of the typical big pharma blockbuster. But note that there is a different use for embryonic stem cells that is nearer to realization on the horizon, and will involve a still different patent strategy: disease models. The TechRev article does get into that [see below].

The TechReview article does mention New Jersey. A key point to contemplate is what the word "earmarked" means:

In addition, California, Connecticut, and New Jersey have all earmarked state funds to support stem cell research not funded by the federal government. The California initiative, by far the biggest at $3 billion, has encountered pitfalls at every turn, demonstrating the difficulties that arise when states get into the research-funding business. The California Institute for Regenerative Medicine, the oversight entity created by the state's Proposition 71, has grappled with accusations of conflicts of interest among those who determine the distribution of funds and with controversies over how the state will reap the financial benefits of stem cell research -- a promise that was part of the proposition.

The TechReview article gets into the egg donation issue at several places. For example:

Almost all embryonic-stem-cell research in the United States faces funding obstacles and ethical objections, but because nuclear transfer is the most contentious topic in the field -- it involves not only the destruction but also the creation of embryos specifically for research -- scientists and universities planning nuclear-transfer programs are extracautious. "The spotlight is on anyone doing this kind of research," says Lanza. For example, Massachusetts law mandates criminal penalties for people who violate laws governing egg and embryo procurement. "If we slip up anywhere, we'll be crucified," Lanza says. [IPBiz: ask Hwang Woo-Suk, but one could also ask ACT about the spotlight it got in 2001.]

There is a discussion of the UCSF program:
UCSF hopes the new facility will help it become a frontrunner in therapeutic cloning. The university was the first in the United States to attempt nuclear transfer, albeit unsuccessfully, in the 1990s. "Now we hope to start again where those studies left off," says Arnold Kriegstein, director of the university's Institute for Stem Cell and Tissue Biology. IPBiz: the TechRev article gives the misimpression that the UCSF work was "way back" in the 1990's. Actually, it was 1999-2001 (see earlier post on IPBiz), ending the year of the Bush funding restriction.

There is mention of the IVF connection:
Reijo Pera and colleagues started cloning experiments at another off-site facility in April, possibly the first U.S. group to try human nuclear transfer since Lanza's team halted its work in 2004. In the UCSF lab, they will use "fail to fertilize" eggs from an in vitro fertilization clinic, which are much easier to get than donor human eggs. When they have optimized the experimental conditions, they will start using human eggs donated specifically for research. IPBiz: As Bill Clinton would say, it depends on what you mean by "donor." For the above paragraph, "donor" means eggs donated specifically for research, while "fail to fertilize" eggs means eggs that did not work in an IVF procedure, and then are donated to research [for example, see the first "60 Minutes" stem cell program of Feb 06 (Feb 12?) for this nuance.]

There is mention of reprogramming:
During reprogramming, some still-unknown factors in the egg turn off the genes that make a cell, say, a neuron and turn on the genes that are expressed in embryos. To uncover the genes controlling this conversion, Brambrink [in Rudolf Jaenisch's lab at Whitehead] has engineered adult cells to express the genes that are selectively activated in eggs. If a particular gene expressed by one of these cells is crucial to the reprogramming process, it will activate genes that are known to be involved in the process's later stages; those genes have been tagged with markers that make the cell glow green. In the best-case scenario, the activator gene might trigger reprogramming itself, creating a clump of stem cells where once sat differentiated fibroblasts. ... Once scientists understand the [reprogramming] process, they can create new technologies to turn adult cells directly into stem cells.

Of a different impact of Hwang:
However, Lanza and his colleagues, who had been so close to cloning stem cells, watched dejectedly. "It was embarrassing," says Lanza. "This obscure group [Hwang's] announced they had done it." ACT was already on shaky financial ground, but Hwang's achievement made its situation even more precarious. The company also abruptly lost its supply of human eggs -- a crucial ingredient in cloning research -- because clinics that ran donor programs felt no further need to participate in research whose central goal had been achieved.

The Hwang fraud as a car wreck:
"It was like watching a car wreck taking place in slow motion. The magnitude of the problem was just really horrifying," says Snyder. "The world had been reset to pre-2004, like when Superman turned the world backwards. If you were pursuing cloning in 2004, you began pursuing it again. If you were sitting on the sidelines, you were back sitting on the sidelines." IPBiz: The TechRev article never mentions the Newcastle work, which purportedly achieved SCNT, but NOT a stem cell line.

Deja vu, all over again?
Ronald Green, an ethicist at Dartmouth College: "But Hwang's claims gave people a glimpse of what would be possible with cloned stem cells, and the consequence was a renewed interest in therapeutic cloning." IPBiz: Jules Verne? This reminds one of the attempted resurrection of Jan-Hendrik Schon's claims. One does note that there was criticism of Schon's claims BEFORE the Beasley report, but there was not much (nothing?) said about Hwang's claims by peers until people started paying attention to the insider's fraud claims. A "glimpse of what would be possible" has more than one meaning here, one as to possible good things and one as to ease of fraud.

Importantly, the article touches on the true, near-term value of embyronic stem cells--as disease models:

But Friedmann and Snyder are focusing on an application that could have much broader implications -- and is closer at hand. Instead of using the cells as a form of -therapy themselves, the researchers plan to use them to study Lesch-Nyhan disease and test new treatments. Experts say this type of application could dramatically improve our understanding of how any disease with a genetic component unfolds at the cellular level. "You could make a stem cell line that has ALS or Parkinson's, using DNA from a patient that really has the symptoms," says Snyder.

Scientists could prod the cells to develop into the type of cells damaged by a disease, such as dopamine neurons in Parkinson's, and study the intricate progression of the disease from its earliest stirrings to its final cellular death knell. Because the cells would be genetically identical to the patient's DNA, they would undergo many of the same molecular changes that underlie the patient's disease.

The article by Emily Singer appears in the May/June 2006 issue of the
MIT TechnologyReview.

The blog californiastemcellreport contains many posts of relevance to stem cell work.


Blogger Lawrence B. Ebert said...

Note that in August 2007, we now have a half-million dollar prize for the "inventor" of therapeutic cloning:

9:10 AM  

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