Science Market Update

The NIH has just announced $5.3M in two new awards through the Autism Centers of Excellence (ACE) program to support autism research studies led by two University of California investigative teams, at UCLA and the UC Davis Medical Center MIND Institute. ACE funding is earmarked for large, multi-disciplinary studies into the origins of autism spectrum neurological disorders and avenues for their treatment. In the case of the two latest awardees, one is a clinical behavioral study and one is a study of genetic variants. The $5.3M is initial one-year funding, with extensions of up to five years. The ACE program includes both centers and networks. Centers are made up of multiple investigators at one site working together on a specific research problem; networks include investigative teams from different sites engaged in a focused study. Both UCLA and UC Davis are ACE centers and will lead the current research projects, though in collaboration with colleagues at other research institutions, namely Harvard, UW, Vanderbilt, Emory, Johns Hopkins, and Yale. As with all ACE research, data and findings are collected centrally by the NIH to maximize their availability to the larger research community.

Synthetic biology is the application of engineering principles to altering components of biological systems, like genes and cells, towards creating new and revised living things (watch the video below for an introduction). It's arguably the most radical, cutting-edge laboratory science field today, and one that calls on its scientists to grapple with ethics as well as biotechnology. At the forefront of this life science revolution is the University of California Berkeley-led consortium SynBERC: the Synthetic Biology Engineering Research Center, with partner colleagues at UCSF, Stanford, MIT, and Harvard. Just this week, principal synbio investigators from these institutions came together with industry scientists and ethicists for a symposium on the UCB campus titled Programming Life: the revolutionary potential of synthetic biology, co-sponsored by SynBERC and Discover Magazine. Whether we are going to continue down the road of reengineering life was not the question so much as how we will go about that delicate task and what the implications and promises are of such a bold project.

The California Institute for Regenerative Medicine (CIRM) is a major funding agency for stem cell research in the Golden State. Since voters approved the establishment of the agency in 2004, the CIRM has spent billions on research and facilities with the aim of making California the stem cell capital of the US. Now, in a move to advance that research mission even further, the agency has announced awards of $32M to investigators and stem cell companies to create a biobank of diseased cell lines for the use of researchers around the world. Called the Human Induced Pluripotent Stem Cell (hiPSC) Initiative Awards, the project will generate and ensure the availability of high quality disease-specific hiPSC resources for disease modeling, target discovery and drug discovery and development for prevalent, genetically complex diseases. 

The tighter funding gets, the more likely it is that young investigators pursuing big ideas will get passed over and science grant money will stay with safer, more established projects. Fortunately there are exceptions to that general rule, including a new program established by the Paul G. Allen Family Foundation specifically to support select pioneering research projects that aim to unlock fundamental questions in biology. They recently awarded investigators from 5 prestigious US universities a total of $7.5M to pursue basic questions about the origins and mechanisms of cellular behavior. One of those 5 Distinguished Investigator awards, for $1.6M, is going to quantitative biologist and recent hire Suckjoon Jun, who works in physics and molecular biology at the University of California San Diego. His project title is "Cell-size control and its evolution at the single-cell level," and includes developing methods to perform long-term directed single-cell evolution experiments, as well as single-cell on-chip manipulation, sequencing, and mathematical modeling. 

Since its first lemonade stand was set up in 2000 by a little girl with cancer, the Alex's Lemonade Stand Foundation (ALSF) has raised over $60M to support pediatric cancer research at institutions across the United States. That's a lot of lemonade. While lemonade stands are still a staple of the organization's activities, celebrity support and large fund raising events like the recent "Lemon Ball" (which raised a record $825K) allow ALSF to leverage the kind of funds that really make a difference. In a recent round of funding awards, the University of California San Francisco's Benioff Children's Hospital was named a Center of Excellence by the foundation and given $1.75M to speed translational research programs and training over the next five years. Chief investigator on the grant is Dr. Katherine Matthay, chief of pediatric oncology at Benioff. She says of the ALSF award in a recent news release:

The Brain Activity Map project could be the next big federal life science research endeavor, with no less a goal than the mapping of the entire living brain and all its neuronal activity. Like the Human Genome Project of the 90's, the not insignificant financial outlay is being presented as an investment that will net even bigger returns, both in terms of new technology and a vastly increased understanding of the mind. President Obama is expected to include the multi-billion dollar, decade-long funding in his upcoming budget proposal, and neuroscience research was a topic he addressed specifically in his recent State of the Union address.

Plant pathogens like the one that led to the notorious Irish Potato Famine of the 1840's are still the subject of intense research at institutions like the University of California Riverside, as the battle continues between mega-crop farmers and diseases that have learned to infiltrate the plant’s immune system. Just what the genetic mechanism is that allows for that infiltration has remained elusive until recently. Studying the notorious oomycete pathogen Phytophthora in its multiple forms, UC Riverside researchers have identified a crucial step in the disease attack of the cell, namely the activity of virulence proteins in blocking RNA silencing pathways, which leads to immune system compromise. The role of RNA silencing as an important immune component is a new research direction and one that is being pioneered at UCR.

2012 was a rough year for two important biorepositories. In October, Hurricane Sandy wiped out power to New York University's main research building, leading to the loss of precious biological samples and hundreds of lab rodents. Earlier in the year, the Harvard University Brain Resource Center at McLean Hospital in Boston suffered a similar catastrophe when a freezer failure led to the compromise of 147 brain tissue samples. 54 of those were specifically for autism research, and the loss has been felt all over the country. As a recent episode of NPR's Morning Edition reported, post-mortem brain tissue samples from autism sufferers are in short supply already and have been for a long time. That autism research is able to make major advances in understanding the neurodisorder is especially impressive given the shortage of these samples. One such triumph has come out of the University of California San Diego's Autism Center of Excellence within the UCSD School of Medicine.

You might expect the barrage of brilliant lab-on-a-cell-phone inventions that come out of the Ozcan Nano/Bio Photonics Lab at the University of California Los Angeles to eventually dwindle, or perhaps only leave us moderately impressed after a while, but that's not the case. Less than three weeks into 2013, the Ozcan Research Group published on their development of a new optical microscopy platform which uses liquid nanolenses that self-assemble around tiny objects (in the sub–100-nanometer range), allowing it to detect viruses and nanoparticles. That paper was published online in the journal Nature Photonics and was the subject of a recent UCLA research news release. Also this month, the Royal Society of Chemistry published the paper Cost-effective and Rapid Blood Analysis on a Cell-phone. And the international society for optics and photonics, SPIE, announced a new annual award for 2013: the Biophotonics Technology Innovator Award. One guess who its first recipient is? Not a shabby way to start the new year at all.

2012 was a big year for the science of snipping DNA to introduce genetic changes into a cell, also known as genome editing. Though Science magazine hailed two new techniques for selectively cutting and pasting DNA in the field of genome engineering as together constituting one of the Top 10 scientific breakthroughs of the year, those methods may already have been surpassed by researchers at the University of California Berkeley using RNA and a single protein. Faster, simpler, and cheaper, the UCB team led by Dr. Jennifer Doudna published initial results of their work genetically modifying bacteria using the RNA-based DNA cleavage technique last summer. The response from the the life science community was extremely positive, with reviews calling it a "tour de force" and a "a real hit," according to the latest press release. Now three more papers are coming out based on the work of the Doudna Lab showing that the RNA programming technique using a bacterial enzyme known as Cas9 is equally effective in making alterations to human genes.