Clinical Research

GlaxoSmithKline collaborates with the Harvard Stem Cell Institute

Attila Csordas — Fri, 26 Sep 2008 05:28:00 -0700

Description

GlaxoSmithKline, the world’s second-biggest pharmaceutical company and the Harvard Stem Cell Institute (HSCI) recently (in July, 2008) announced a five-year, $25 million-plus collaborative agreement.

GSK’s investment, one of the largest by a pharmaceutical company in stem cell science, will support innovative research at Harvard University and in at least four Harvard-affiliated hospitals in the areas of neuroscience, heart disease, cancer, diabetes, musculoskeletal diseases and obesity. In addition, GSK will fund an annual grant, which supports early stage research in stem cell biology, as part of HSCI’s seed grant program “GSK believes stem cell science has great potential to aid the discovery of new medicines by improving the screening, identification and development of new compounds. We have carefully chosen the Boston biomedical community to collaborate with on this important venture. It has the highest concentration of leading stem cell scientists, and the Harvard Stem Cell Institute is the epicentre of that community,” said Patrick Vallance, Head of Drug Discovery at GSK.

Biomedical Sciences and Biotechnology

Source

http://www.gsk.com/media/pressreleases/2008/2008_pressrelease_10089.htm
http://www.xconomy.com/boston/2008/07/25/harvard-stem-cell-institute-wins-25-million-investment-from-glaxosmithkline/
http://www.hsci.harvard.edu/
http://www.gsk.com/

Pfizer's growing and various interests in stem cells

Attila Csordas — Fri, 26 Sep 2008 03:46:44 -0700

Description

Pfizer Inc. is one of the biggest research-based pharmaceutical company and ranks number one in the world in sales. The company opened a “regenerative medicine unit” in Cambridge, Mass. last year and now moves to the other Cambridge, U.K. to open another similar shop around November. On the other hand Pfizer has already invested $3 million in shares of EyeCyte a La Jolla based early stage stem/progenitor cell-based ophthalmology research and development company. The growing interest can be partly explained by the role that induced pluripotent stem cells can play in drug testing and the first uses will probably be in early-stage safety testing. The risk-taking in the new and unknown field is especially interesting considering the tough times in the pharma industry.

These cells will be tremendous in drug discovery,” an R&D exec told Reuters. “They will help us understand personalized medicine, genetic variation, ethnic populations, what biomarkers to follow.” John McNeish will run Pfizer's U.S. unit, which will focus on heart disease and diabetes. In November, the company plans to open a standalone regenerative medicine unit in Cambridge, United Kingdom, to focus on research in ophthalmology and diseases of the central nervous system. McNeish said the overall operation will eventually have 50 to 60 scientists working on stem cell therapies, and they are working with academic researchers and smaller biotech companies.

Biomedical Sciences and Biotechnology

Source

http://www.reuters.com/article/reutersEdge/idUSTRE48MBY020080923
http://blogs.wsj.com/health/2008/09/24/pfizer-moves-into-stem-cell-research/
http://en.wikipedia.org/wiki/Pfizer
http://www.medicalnewstoday.com/articles/112558.php

Regaining vision with gene therapy using adeno-associated viruses

Attila Csordas — Tue, 23 Sep 2008 04:22:28 -0700

Description

Adeno-associated viruses (AAV) are more and more promising candidates as viral vectors for gene therapy. They are small and not pathogenic according to our current knowledge, causing very mild immune response and are able to stably integrate into the host genome's chromosome 19 at a a particular site even in non-dividing cells.

Using viral vectors made out of adeno-associated viruses researchers were able to fully restore the sight of 2 people suffering from Leber's Congenital Amaurosis, an incurable congenital blindness syndrome.

"The RPE65 gene encodes the isomerase of the retinoid cycle, the enzymatic pathway that underlies mammalian vision. Mutations in RPE65 disrupt the retinoid cycle and cause a congenital human blindness known as Leber congenital amaurosis (LCA). We used adeno-associated virus-2-based RPE65 gene replacement therapy to treat three young adults with RPE65-LCA and measured their vision before and up to 90 days after the intervention. All three patients showed a statistically significant increase in visual sensitivity at 30 days after treatment localized to retinal areas that had received the vector. There were no changes in the effect between 30 and 90 days. Both cone- and rod-photoreceptor-based vision could be demonstrated in treated areas. For cones, there were increases of up to 1.7 log units (i.e., 50 fold); and for rods, there were gains of up to 4.8 log units (i.e., 63,000 fold). To assess what fraction of full vision potential was restored by gene therapy, we related the degree of light sensitivity to the level of remaining photoreceptors within the treatment area. We found that the intervention could overcome nearly all of the loss of light sensitivity resulting from the biochemical blockade. However, this reconstituted retinoid cycle was not completely normal. Resensitization kinetics of the newly treated rods were remarkably slow and required 8 h or more for the attainment of full sensitivity, compared with <1 h in normal eyes. Cone-sensitivity recovery time was rapid. These results demonstrate dramatic, albeit imperfect, recovery of rod- and cone-photoreceptor-based vision after RPE65 gene therapy."

Biomedical Sciences and Biotechnology

Source

http://www.pnas.org/content/early/2008/09/19/0807027105.abstract
http://en.wikipedia.org/wiki/Adeno-associated_virus
http://blog.wired.com/wiredscience/2008/09/gene-therapy-bl.html

Lightweight, open, mobile, cheaper MRI brain scanner prototype working in lab

Attila Csordas — Wed, 17 Sep 2008 15:13:20 -0700

Description

Those giant, claustrophobic, tunnel forming magnets used for magnetic resonance imaging (MRI) in labs and hospitals look so last century! They are pricey and heavy, making MRI systems immobile and demanding to install. Any alternative? Yes by implementing the pre-polarized MRI concept introduced some 15 years ago:

"Writing in the Journal of Magnetic Resonance, Vadim Zotev and colleagues report success in imaging a human brain using a different type of MRI system: lightweight, open, mobile and significantly cheaper.

By dividing the functions of these large-field magnets between two sets of magnets with different characteristics, Zotev et al. have produced the prototype of a machine that would be smaller and more open, as well as being capable of performing magnetic resonance imaging and magnetoencephalography at the same time.

Conventional MRI machines reconcile these different requirements by using magnets that are both powerful and homogeneous. But could the same effect be achieved by using two simpler magnets and switching between them? The first magnet, strong but relatively inhomogeneous, would polarize the sample, whereas the second, weak but highly homogeneous, would be optimized for collecting resonance signals. This concept, termed pre-polarized MRI, was originally introduced by Macovski and Conolly2 some 15 years ago, and has been pursued by several research teams since.

Zotev et al. now report obtaining images of a living human brain using pre-polarization at 30 millitesla (mT) and image data collection at just 46 microT, a similar strength to that of Earth's magnetic field and about 30,000 times weaker than that of typical clinical MRI machines. Using such small magnetic fields means that the frequencies of the signals produced by the oscillating nuclear spins are similarly reduced from the usual radiofrequency range to around 2 kilohertz — a frequency readily audible to the human ear (approximately three octaves above middle C)."

Biomedical Sciences and Biotechnology

Source

Klaas P. Pruessmann: Medical imaging: Less is more
Nature 455, 43-44 (4 September 2008) | doi:10.1038/455043a
http://www.nature.com/nature/journal/v455/n7209/full/455043a.html

Zotev et al.: Microtesla MRI of the human brain combined with MEG.
J Magn Reson. 2008 Sep;194(1):115-20.
http://www.ncbi.nlm.nih.gov/pubmed/18619876?dopt=Abstract&holding=npg