Yesterday genome sequencing innovator J. Craig Venter announced his latest venture, Human Longevity, Inc. (HLI). The company has $70 million in startup funding to promote healthy aging through genomics and cell-therapy based therapeutics and diagnostics.
The first phase of this effort is to construct a large genome-sequencing center. When at full capacity, the center would be able to sequence 40,000 sequences per year, with a possible increase to 100,000 sequences per year. With the machines HLI has purchased, the company anticipates the cost per genome to be around $1,000.
The company will focus its efforts on several diseases: cancer, diabetes, obesity, dementia, heart and liver diseases. It’s established partnerships with several universities and research institutes to sequence the genomes of consenting clinical patients (though ScienceInsider’s report suggests patients will have to speak up if they don’t want to participate).
While the company is interested in making genomic sequencing cheaper, they are operating in a big industrial fashion that seems unlikely to pass along any improvements to the individual. That is, HLI is not likely to become a genomic equivalent to 23andMe. While that probably means the company will handle its interactions with the Food and Drug Administration much better than 23andMe, the proprietary aspect of HLI’s business models don’t leave much room for those who contribute their genomic data to benefit from their contributions. So the $1,000 genome sequence really won’t be the price for the person on the street.
A Food and Drug Administration advisory panel provided some filler for the 24-hour news channels this week. The reason: discussion of mitochondrial replacement as a means of in-vitro fertilization (IVF). It involves a ‘donor’ cell which has its nucleus removed to receive the nucleus of the mother (it bears some resemblance to somatic-cell nuclear transfer, except there is an additional DNA source involved). This method could be used for situations where the mother has mitochondrial defects that could be passed on to the offspring.
The method gets the superficial cable news attention because the resulting offspring would have DNA from all three donors. While the Presidential Commission for the study of Bioethical Issues has not weighed in (it doesn’t meet again until June), the FDA panel discussed the state of science and research on the technique, as well as the design requirements for early-phase clinical trials. Researchers have produced monkeys via this IVF technique, but panel members were reluctant to recommend human trials at this time.
Over on the other side of the Atlantic, the U.K. is further along in regulating the technique. The appropriate advisory bodies started assessing mitochondrial replacement in 2011, and the government announced last year that it was working on regulations. It issued a consultation on Thursday for the draft regulations (questions of interest are on pages 27-28). Responses will be accepted until May 21.
Here are some recent items of note.
Open Access: Both Science and The Royal Society have announced plans to establish open access journals. Science Advances will be financed through author charges, aim for rapid publication, and use an editorial model comparable to that of scientific societies. Royal Society Open Science will be the first broad-based journal from the Society, focusing on the full range of science and maths. It follows a similar financing model to Science Advances (gold open access), that some in the UK have criticized as not encouraging the development of institutional repositories.
Petitions: Two recent responses to petitions at the We The People website considered science-related topics. The Administration responded to a petition concerning net neutrality and another on access to clinical trials and compassionate use. While the net neutrality response was characterized as a reassertion of government policy, the White House demurred on making a statement on the second matter to avoid interfering in the determinations of an independent government agency.
Science Raps: Tom McFadden has been busy. His third rap video release this month focuses on metals, and comes from students at the Nueva School.
Physics Caucus: The Congressional Physics Caucus will likely have fewer members in the next Congress. Representative Rush Holt of New Jersey announced he will not seek re-election this fall. When he leaves in early January 2015, he will have served 16 years. He holds a Ph.D. in physics from New York University, and was assistant director of the Princeton Plasma Physics Laboratory at the time he was elected to Congress. Representative Bill Foster is the other current member of the Physics Caucus. Best wishes to Representative Holt in his next endeavor, whatever that might be (I’d wager that Franklin’s List is planning to get him involved).
The Food and Drug Administration (FDA) is going to study whether or not drug advertisements on television need to be changed. There are concerns that the current model, where all of the drug’s side effects are supposed to be listed, is giving a realistic picture of the benefits and harms of the advertised drug.
(FWIW, it certainly provides an easy, if sometimes lazy, vehicle for comedy.)
The Federal Register notice describes the possible study, and explains what the FDA hopes to accomplish. The idea is to examine the effects on consumer understanding of providing different levels of risk information in a direct-to-consumer advertisement (while the request is specific to television, I have to think any changes would influence other advertising).
“Our hypothesis is that, relative to inclusion of the full major statement, providing limited risk information along with the disclosure about additional risks will promote improved consumer perception and understanding of serious and actionable drug risks. We will also investigate other questions such as whether overall drug risk and benefit perceptions are affected by these changes.”
As the FDA is at the study phase of this particular regulatory process, it will be months before we have a different form of commercial for comedians to mock.
More music to share today.
Hot on the heels of the Brahe/Kepler fight, Tom McFadden has dropped the last in the series of Brahe’s B.A.T.T.L.E.S. This time two diseases fight it out for lethal supremacy. See how the black plague and the yellow fever try to one-up each other in this video. The choice of song to repurpose is quite apt.
While Tom’s current video project is now over, he continues to work in science education. You can follow his efforts on his website.
I stumbled upon Gia Mora’s one-woman show, Einstein’s Girl, thanks to a promotional email. The show uses advanced physics and mathematics as a way to explore relationships. Mora gets into much more detail about the why behind the piece on her blog, and she comes to science from the perspective of an interested non-scientist (she’s on the board of the SoCal Science Cafe). You can sample some of the songs from the show and the album before diving into it completely.
Finally, Francis Collins, National Institutes of Health Director, can’t help breaking into song. He did so at the end of his appearance on The Diane Rehm Show yesterday. Whether or not you think he should keep bringing the guitar with him, I think you have to respect his tenacity.
Two recent actions involving the Food and Drug Administration (FDA) suggest to me that the matter of whether stem cell patents are valid may not be so critical.
The D.C. Circuit Court of Appeals ruled last week that the FDA has jurisdiction over stem cells cultured for therapeutic use (H/T The Scientist). This decision upheld a lower court ruling that considered the act of culturing the cells more than ‘minimal manipulation,’ and therefore subject to FDA drug oversight regulations.
(IANAL, but I think this decision could be used to strengthen the case that stem cell patents – at least for cultures of said cells – would be valid. After all, if there was more than minimal manipulation, wouldn’t that be sufficiently transformative to make the cultures no longer products of nature? Again – I Am Not A Lawyer.)
Aside from the legal matters, there appears to be a big regulatory mismatch that will hinder commercialization of stem cell treatments. In the February 6 edition of Cell Stem Cell researchers note (H/T The Scientist) that differing regulations between the National Institutes of Health (NIH) and the FDA may reduce the number of stem cell lines that could be used in clinical practice.
FDA regulations require that stem cell donors be screened for various diseases (so that treatments derived from those cells cannot infect others). NIH regulations – not focused on commercial applications of stem cell research – do not have this requirement. Now it is possible, as the article from The Scientist notes, for the FDA to allow some treatments to be approved without such a screening, but some alternative measure will likely be needed to mitigate the risk of infection.
As part of its work on the ethics of neuroscience, the Presidential Commission for the Study of Bioethical Issues is seeking public input. Per this notice from the January 31 edition of the Federal Register, the commission “seeks public comment on the ethical considerations of neuroscience research and the application of neuroscience research findings.” Comments are due by April 1.
The request certainly is intended to support the Commission’s work on the ethics of neuroscience research and the applications of that research. Of particular interest to the Commission are the following topics:
- The diversity and scope of ethical considerations related to neuroscience as a field;
- core ethical standards that guide neuroscience research, including consistency (or lack thereof) across disciplines, and potential tension among the guiding standards;
- advances in neuroscience research that raise novel ethical issues or heighten existing ethical tensions;
- whether emphasis on particular aspects of the Common Rule (or other research ethics regulations) is needed given the particular implications of some neuroscience research or whether any part of the Common Rule needs clarification in order to adequately protect participants in neuroscience research specifically;
- potential implications of discoveries that might flow from studies of the brain and questions that might arise from neuroscience research findings and their applications, including questions about the potential implications for privacy, personal agency, and moral responsibility for one’s actions; stigmatization and discrimination; and the appropriate use of neuroscience in the justice system;
- strategies for integrating from a project’s inception ethical considerations into neuroscience research, technological development, and scientific research generally; and, ethical considerations concerning communication about neuroscience research and neuroscience research applications by scientists, journalists, and others.
The Commission is meeting in Washington on February 10 and 11, and this request will certainly be mentioned, if not discussed in detail. Of particular interest may be how this project will tie into the BRAIN initiative that prompted the Commission’s interest in the subject.
While recent Supreme Court activity (like the Myriad case) focused on the validity of gene patents, other biotechnology patents have been the object of scrutiny.
Consumer Watchdog asked the U.S. Patent and Trademark Office (USPTO) to review the patent it granted the University of Wisconsin-Madison in 2006 related to in vitro cultures of human embryonic stem cell lines. The USPTO agreed to review the patent, but ultimately upheld the patent after several reviews and appeals. In July 2013 Consumer Watchdog filed a brief with the U.S. Circuit Court of Appeals to have the patent invalidated. (February 9 – Edited To Add – I had posted about the matter back in 2010 as it worked its way through appeal(s))
As with many patent cases, there are a number of different arguments that can be made for deciding the case. But one of the arguments in the brief draws comparisons with the Supreme Courts arguments in the Myriad and related cases. Specifically Consumer Watchdog is arguing that like the isolated gene sequences, the stem cell cultures are isolated products of nature and should not be eligible for patent protection.
Now, the Court of Appeals may opt to let the USPTO’s decision stand. It will hear the case (it requested all parties submit briefs by January 16), and decide to validate or invalidate the patent based on any other claims put forth in the argument. There are any number of procedural questions (in the courts or at the USPTO) that could stop the case cold. And should the case make it through the Court of Appeals, a visit to the Supremes could certainly happen.
Edited to add - As noted in the comments, I managed to conflate the Food and Drug Administration with the Federal Trade Commission. This is perhaps the biggest screwup in the history of this blog, and I apologize. The following text has been edited to reflect that, with strikethroughs and new text in italics. (And I had to take two runs at revising the post title. I was inexcusably sloppy.)
While it may feel like it, the Federal Trade Commission (FTC) is not focusing all of its attention on the genetic testing company 23andMe is not the only genetic testing company getting scrutiny from federal regulators.
(The agency Food and Drug Administration (FDA) issued a warning letter to the company because it considered the marketing of its genetic testing kit to lack necessary approval from the agency. Given how the FTC FDA believes 23andMe was selling the kits to provide medical information, the agency concluded that the tests were subject to agency regulation. Months of negotiation to obtain evidence of the efficacy of 23andMe’s tests did not resolve the matter, which led to the warning letter. )
The Federal Trade Commission (FTC) has brought a lawsuit against GeneLink Biosciences related to a service it provides based on genetic tests. The company will provide nutritional supplements based on a customer’s DNA sample. They claim various health benefits based on the genetic tests, and the FTC is suing the company for what it considers false advertising.
In both cases the agency involved believes that genetic testing companies are making claims that they either have not or cannot provide evidence. If the claims are considered to be ahead of relevant science (or absent same), the FTC (or the FDA) will act.
GeneLink and the
agency FTC are negotiating a settlement agreement. The final agreement will include provisions to improve the security of collected information.
This week National Institutes of Health (NIH) Director met with institute directors. According to Nature News last month, he intended to pitch the institute directors with a proposal to fund more ‘high-risk, high-reward’ research proposals.
The idea could be implemented by expanding the NIH ‘Pioneer‘ research awards, given to individual researchers (as opposed to their research projects). Individual researchers receive roughly 5 percent of its annual budget, though private foundations like the Wellcome Trust and the Howard Hughes Medical Institute do award grants to individual researchers.
Part of the motivation for shifting a little more money (it’s unclear how much) to individual researchers is based on research indicating that individually funded researchers tend to produce more highly-cited papers. That’s consistent with the traditional academic metrics and rewards of excellence. But it’s not so obvious that more highly cited papers is the best measure of the return on public investment in scientific research (especially research geared toward biomedical fields). For instance, this language from the Pioneer website hints at other desired outcomes for this public investment:
“To be considered pioneering, the proposed research must reflect ideas substantially different from those already being pursued in the investigator’s laboratory or elsewhere”
Taking a risk seems to be a major factor in the Pioneer program, and it should not be confused with producing highly-cited papers. Certainly there are times when the risks lead to such outcomes, but correlation does not mean causation.