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ACTIP Bulletin 50 September 2007

Next meeting ACTIP: The next plenary meeting of ACTIP will be held in Ivrea, Italy, November 29-30, 2007 and is hosted by RBM-MerckSerono. Central themes will be PAT implementation and removing bottlenecks in downstream processing. Information on the programme will be sent separately to ACTIP member companies through the ACTIP Secretariat   In this issue: Information sources Bioprocessing and production facilities Business News Drug discovery Research News Stem cell news Gene therapy news Regulatory news News from the Commission Agenda   Information sources   YouTube for scientists' to link researchers worldwide SciVee, (www.scivee.tv) a video-sharing site for scientists is set to be officially launched on 1 September. Already online, it allows scientists to upload their technical papers and a podcast videos in which authors explain their papers' results. Jointly operated by three US partners - the Public Library of Science (PLoS), the National Science Foundation (NSF) and the San Diego Supercomputer Center (SDSC) - "SciVee is about the free and widespread dissemination and comprehension of science," explains the portal. 'Created by scientist for scientists', it also aims to facilitate the creation of science communities around specific issues, articles and keywords and promises more exposure for scientists' publications. The portal is not targeted at the general non-scientist audience and attracting nonspecialist viewers will depend on scientists' ability to present their work in an understandable way. Source: EurActiv News, August 30, 2007   WorldWideScience portal goes online In parallel with the European Commission's efforts to boost access to science, WorldWideScience.org, an internet portal providing a single entry point to several national and international scientific databases, has been launched by the US and UK.The site was launched in June 2007 to offer a gateway to science information, with 15 national portals in nine countries contributing to date. The site developers - the United States Department of Energy (DOE) and the British Library - are now inviting more national and international science databases to join the venture and make their collections accessible. Source: www.Euractiv.com, July 17, 2007     Bioprocessing and production facilities   Organic solvents for improved protein purification US researchers from Dow Chemical Company and Pfizer have shown certain organic solvents can be used to selectively extract therapeutic proteins from host bacteria, avoiding the need for cell lysis and increasing product purity by a factor of five. The researchers used glycol ether solvents to selectively extract a proprietary therapeutic protein from genetically engineered Pseudomonas fluorescens (P. fluorescens) bacteria, dramatically increasing the purity of the product and reducing the amount of downstream purification needed. Dow has developed the Pfenex expression technology based on P. fluorescens as the bacteria can be readily modified to produce proteins within the periplasmic space. This system can produce yields ten times greater than Escherichia coli (E. coli) and also correctly folds the proteins which are often misfolded by E. coli. The Pfenex system consistently gives good yields of high quality protein and this new recovery technology enhances that, and really adds to the lower cost of goods which often is a benefit of this system. The use of glycol ether solvents increases the permeability of the cell wall and allows isolation of the proteins within the periplasmic space without rupturing the inner cell wall and releasing the impurities held within. This technique leaves behind about 80 per cent of the cell leading to a five-fold purification of the protein. In addition, the researchers also found that heating the glycol ether extract to 55°C caused the solution to split into an aqueous layer and an organic layer with the aqueous layer containing essentially all of the recovered protein in a volume of less than 15 per cent of the original extract volume. More reading: Biotechnology Progress Source: www.DrugResearcher.com, September 4, 2007   Synthetic MAb purification tech edges closer to market ProMetic BioSciences this week announced that its MAbsorbent ligands, used for the purification of monoclonal antibodies (MAbs) and recombinant antibody fragments, had successfully met pre-set performance targets that make them real contenders in the MAb purification market. ProMetic's purification products are synthetic chemical entities, unlike biological ligands commonly used in traditional protein purification techniques. The performance of the synthetic ligands was confirmed by seven leading antibody producers, all of whom existing ProMetic clients. The MAbsorbent ligands offer improvements in stability and cost-effectiveness, according to the company - a significant plus-point when a litre of a biological ligand such as protein A can cost as much as $10,000 (¤ 7,300). An important point made by the company is that a growing number of monoclonal antibody products are in fact antibody fragments (as opposed to full chain antibodies). These can be tricky to purify as they lack the region to which protein A binds (the Fc region), making this standard purification technique unusable. Prometic has tackled this problem by developing a new synthetic ligand that binds to a different region of the antibody molecule, providing an effective solution for the capture and purification of antibody fragments. Following the successful development of its ligand products, the next stage for the company is to move on with manufacturing scale-up and preparations for market launch. Source: www.DrugResearcher.com, August 28, 2007   GE boosts biotech production GE Healthcare has installed a new automation system at its global headquarters in Uppsala, Sweden, which is due to boost production of protein separation media at the site by 50 per cent. The system, provided by Swiss firm ABB, is the third 800xA extended automation solution that has been installed at the Uppsala site, making it the world's most advanced facility for the production of protein separation media. The system itself promises to deliver improvements in productivity and profitability, and has clearly proven a hit with GE who has seen great success with the ABB solutions and has been dealing with the company for 15 years. The regulatory-compliant system will control and monitor 4,000 input/output signals from tanks, reactors, valves, stirrers and other process equipment, and also includes other functions such as integrated batch control and management, and record protection and retrieval. According to ABB, plants can waste up to 40 per cent of their process production capacity through equipment downtime, process bottlenecks, and quality issues. The firm refers to these undetected losses as a company's 'hidden plant' opportunities Source: www.DrugResearcher.com, June 11, 2007   Vivalis duck cell lines show MAb production power French firm Vivalis has announced the first data evaluating its proprietary EBx cell lines (duck derived embryonic stem cells) for the production of monoclonal antibodies (MAbs). The antibodies produced using the EBx cell lines showed remarkably low fucose content. Vivalis has been working in collaboration with another French firm, Mat Biopharma, to evaluate its EBx cell lines for the production of Mat Biopharma's monoclonal antibodies. The companies demonstrated that as well as the reduced fucose content, the glycosylation profile of monoclonal antibody of IgG1 subtype produced in the avian EBx cells was similar to human antibody glycosylation profiles. Already having generated a great deal of industry interest for its potential applications in vaccine manufacture, this is the first data showing that Vivalis' EBx cell lines could prove an attractive alternative to traditional Chinese Hamster Ovary (CHO) cell lines in MAb production. The duck EBx cells continue to display some striking ES features, like telomerase expression and expression in ES cell markers, but in addition display industrial features like growth in suspension in animal serum-free medium up to high cell densities. The EBx suspension cells can be efficiently produced in stirred-tank bioreactors, with production times and costs similar to traditional MAb production methods in CHO cell lines. Source: www.DrugResearcher.com, July 25, 2007     Business news   Big pharma's wish list Some of the world's largest pharma firms have set out what therapeutic areas and specific mechanisms are becoming more important to them, and which will be left by the wayside. Senior executives from Pfizer, Wyeth, Merck & Co. and Novartis sat down at the recently held Drug Discovery and Development of Innovative Therapeutics (DDT) conference in Boston, US, to discuss what areas they are looking to expand in, both through internal research and new partnerships. Merck & Co. Dr Mervyn Turner, senior vice president of worldwide licensing and external research at Merck Research Laboratories revealed that the rapidly growing oncology market has become increasingly important to the company. The move also "sits well with Merck's thinking on targeted therapies, a direction we think the industry will go in general," added Turner. Further, the company is very interesting in increasing its advanced data mining capabilities, which was also evidenced by Merck's acquisition of Rosetta. In terms of mechanisms, Merck & Co. has invested heavily in RNA interference (RNAi) - they recently bought Sirna Therapeutics for $1.1bn (¤850m). The pharma giant sees this as an attractive area due to the technology's ability to "expand the druggable universe" and its potential for a persistent effect. This, and other advantages of RNAi, can increase the probability of success of potential drugs. However, despite around a decade's research in amma-aminobutyric acid (GABA) biology, the firm is ready to give up on the protein as a drug target. Wyeth Dr Thomas Hofstaetter, senior vice president of global business development for Wyeth, said that Alzheimer's disease is "the one largest increasing R&D area for Wyeth." The company now has over 15 programmes in Alzheimer's from discovery through to one drug that is about to enter Phase III clinical trials. The company is leveraging its expertise in small molecules, vaccines and biologics to attack the disease. In contrast, Wyeth sees its presence in the oral contraceptives market as complete following the recent approval of Lybel (levonorgestrel). The pharma giant has been involved in bio-molecules for a long time, including antibodies, but is now looking into antibody-like therapies. Further, Wyeth has stopped all research efforts in antisense and cell therapy, largely because delivery problems limit their efficacy. Pfizer Dr BJ Bormann, head of worldwide strategic alliances for Pfizer explained that the world's largest pharma company is increasingly interested in ophthalmology and pain therapeutics, while gastrointestinal and dermatology indications have waned in the eyes of the company. Further, companion diagnostics are seen as an integral part of Pfizer's future. Mechanisms that Pfizer is losing interest in are anything with systemic delivery issues such as antisense - although the company do have a "productive" ophthalmology antisense collaboration it is still actively pursuing. The company is also disappointed with the concept of black box screening, where Pfizer has a number of expensive collaborations that were "productive but the science didn't actually fulfil what our hopes was," said Bormann. Novartis Dr Jeremy Levy is the global head of business development and strategic alliances for Novartis Institutes for Biomedical research. He explained that Novartis has recently increased their research in the area of diseases that relate to an excess of interleukin-1 and are looking to expand in this area further. Levy explained that there are "very few therapeutic areas not of importance," and the company is willing to look into any and all diseases, especially once proof of concept has been established. In terms of mechanistic approaches to therapy, Novartis is increasing its R&D into biologics since it intends to become a leader in this field, while at the same time stopping cell therapy research. This latter decision is due to difficulties in scaling cell therapies. Source: www.DrugResearcher.com, August 13, 2007   No lack of innovation in pharma, says Wyeth The often cited decline in R&D productivity and dwindling innovation within the pharma industry is actually a thing of the past, according to Dr Thomas Hofstaetter, Wyeth's head of business development. He said the misunderstanding is caused by the long timelines in the business. "It takes 15 years to get an idea to market and so the dearth of new products making it onto the market that we see now is the result of changes in drug discovery approaches made 15 years ago. However, the pipelines are more diverse and better quality than ever before." The stats do seem to at least partly back up this last statement. Dr Mervyn Turner, senior vice president of worldwide licensing and external research at Merck Research Laboratories, aggregated the pipelines of seven large healthcare firms. This showed that, in the world of big pharma at least, the late-stage pipeline may have essentially flattened out over the previous few years, but the number of molecules in early stage development is increasing rapidly. Hofstaetter believes these huge pipelines will translate into interesting and innovative new products. However, he doesn't believe the pharma industry is out of the woods just yet: pharma simply has to become much more efficient in everything it does from drug discovery to delivery. By becoming more efficient, Hofstaetter clearly hopes to eventually reduce the time it takes to bring a drug to market and to reduce the number of costly drug failures, especially at a late stage. Source: www.DrugResearcher.com, August 14, 2007   Pfizer expands biologics R&D Pfizer is breaking ground on a $50m expansion of its biologics pilot plant in Chesterfield, at a time when the drug giant is cutting back in other business areas. It is roughly doubling the size of its facility by adding 50,000 sq ft. as it actively pushes into the potentially very profitable biologics market. The investment comes at a time when Pfizer is instigating several cost-cutting strategies as it battles patent expiries, scales back production on lacklustre drugs and faces fierce market competition. At the same time, the drug giant is moving head strong into biologics, with the aim to have 20 per cent of its pipeline product portfolio in the sector by 2009. Over the last 10 years, Pfizer has gone from having one biologic to 25 in the pipeline and these include monoclonal antibodies (mAbs), small interfering RNA (siRNA) drugs, vaccines and aptamers. At present, the company only has one biologic on the market but its first mAb, which targets CTLA-4 as an anti-cancer treatment, is due to be launched "in the next year." The Chesterfield facility is the only plant Pfizer owns for the production of large quantities of biologics for clinical trials; although it does also have a manufacturing agreement with Boehringer Ingelheim. Source: www.DrugResearcher.com, August 23, 2007     Drug discovery   Novartis approach to drug toxicity Safety has always been a key issue in drug research but since Merck & Co. was forced to pull its painkiller Vioxx (rofecoxib) in 2004, when it was discovered the drug increases the risk of heart problems, it has never been more in the public eye. Dr Dimitri Mikhailov, US team lead in cheminformatics at the Novartis Institutes for Biomedical Research, recently outlined how the pharma giant has set about predicting the safety of drug candidates. Mikhailov, told delegates at the recent drug Discovery and Development of innovative Therapeutics (DDT) conference in Boston, US, that although there are several commercially available tools for predicting toxicity and these form a "good basis" for research, they are often difficult to interpret and they don't include internal scientific knowledge. In 2003, Novartis began developing its own computational programme to help predict some of these issues, called ToxCheck. The company set about developing a programme that could be accessed from any desktop computer, was easy to use and interpret and was fast and interactive. In that way, it could form a fundamental part of the decision making process at all levels. Using public and internal data on thousands of compounds, Novartis developed a system that correlates a compound's structure with potentially toxic properties, such as cardiotoxicity and genotoxicity. This information is then used to develop toxicity alerts based on around 160 molecule substructures that Novartis believes can cause drug safety problems. Recent additions to the system are an index of adverse reactions and in vitro profiling which are used to predict off-target binding and side-effects. The final programme is now used by thousands of chemists and biologists in Novartis but Mikhailov was keen to stress that the researchers can use this information as a warning sign for experimental follow up - rather to kill a compound's development straight off. Source: www.DrugResearcher.com, August 28, 2007   In-silico model for drug-induced liver injury Life science computer modelling expert Entelos has entered a collaboration with the FDA to develop a computer model of drug-induced liver injury. The company has previously developed an in silico model for diabetes that 'maps' data from preclinical animal studies onto a 'virtual human' to allow better prediction of human responses. The two-year collaboration is part of the FDA's 'Critical Path Initiative' that is aiming to reduce the time it takes to develop therapeutically important medical products and bring them onto the market. With the pharma industry being criticised for its lack of productivity and the rising cost of expensive failures, such as Pfizer's Torcetrapib, the need for better methods of assessing the toxicity of drugs earlier in the drug discovery process has never been more evident. The computer model will focus on defining healthy human liver function and create a cohort of healthy patients to represent 'tolerator, adaptor and susceptible patient phenotypes' and predict what combinations make patients susceptible to liver damage following exposure to specific drugs or drug classes. Source: www.DrugResearcher.com, August 6, 2007   Lonza acquires in-silico protein analysis platform Switzerland-based Lonza is establishing its biopharma portfolio with the acquisition of Zyentia's Aggresolve technology this week. The purchase would see the protein technology incorporated into Lonza's Biopharmaceuticals Mammalian R&D business unit, forming a new function, entitled Advanced Protein Technologies. Aggresolve technology is a comprehensive in silico protein analysis platform which can be applied to solve the problems of protein aggregation. Protein aggregation is a commonly occurring phenomenon where proteins fail to fold correctly. In the body, misfolded proteins can cause a range of diseases known as amyloidoses. The Aggresolve technology, developed in collaboration with scientists at the University of Cambridge, aims to predict protein design and aggregation which can lead to selecting or engineering proteins that would not have the aggregation problem. The technology would be used for both Lonza's development pipelines and for contract manufacturing clients. Source: www.DrugResearcher.com, August 2, 2007   Human antibodies block human and animal SARS Viruses An international team of investigators identified the first human antibodies that can neutralize different strains of the virus responsible for outbreaks of SARS. Two human antibodies were identified that bind to a region on the SARS virus' spike glycoprotein that is called the receptor binding domain (RBD). One of the antibodies, called S230.15, was found in the blood of a patient who had been infected with SARS and later recovered. The second antibody, m396, was taken from a library of human antibodies the researchers developed from the blood of 10 healthy volunteers. The m396 antibody binds to the region on the RBD that allows the virus to attach to host cells. When tested in cells, both antibodies potently neutralized samples of the virus from both outbreaks. The antibodies also neutralized samples of the virus taken from wild civets, though with somewhat lower potency. In addition, mice that received m396 or S230.15 were fully protected from infection by SARS from humans as well as infection by SARS from civets, though the latter not completely. The m396 antibody neutralizes all strains of SARS tested and is likely to neutralize all strains of the virus with known sequences. There are no other reports for such antibodies available. Further reading: July 2 early online edition of the Proceedings of the National Academy of Sciences. Source: Gen News Highlights, July 3, 2007     Research news   Normal prions play preventive role in Alzheimer's Researchers at the University of Leeds found that the normal prions produced by the body help to prevent the plaques that build up in the brain to cause Alzheimer's disease. "In vCJD, the normal version of prion protein, PrPc, found naturally in the brain, is corrupted by infectious prions to cause disease. Our experiments have shown that the normal prion proteins found in brain cells reduce the formation of beta amyloid, a protein that binds with others to build plaques in the brain that are found in Alzheimer's disease," explains Nigel Hooper, Ph.D., a professor at Leeds and leader of the research. Using cells grown in the lab, the team looked at the effect of high and low levels of normal prion protein in the successful formation of beta amyloid. They found that beta amyloid did not form in cells with higher than usual levels of PrPc. In comparison, when the level of PrPc was low or absent, beta amyloid formation was found to go back up again. Mice genetically engineered to lack PrPc were also studied. Again, this revealed that in its absence, the harmful beta amyloid proteins were able to form. It appears that PrPc, the normal prion protein, exerts its beneficial effect by stopping beta secretase from cutting up amyloid protein into the smaller beta amyloid fragments needed to build plaques. Source: Gen News Highlights, July 5, 2007   Silicon nanowires conduct drugs into cells New research has shown that silicon nanowires can be used to deliver toxic agents into both human and bovine epithelial cell lines, opening up new options for drug targeting and delivery. The research details the use of silicon nanowires (NW) that are 50,000 times smaller in diameter than a human hair as delivery vehicles for the Escherichia coli (E. coli) Shiga toxin 1 A subunit into both human and bovine cells. The researchers coated the nanowires with fibronectin (Fn) to encourage the internalisation of the nanowires by cells and used them to deliver a recombinant Shiga toxin type 1 (Stx1) A subunit (StxA1) into cultured human laryngeal epithelial cells (HEp-2) and bovine mammary epithelial MAC-T cells. Using a neutral red cytoxicity assay the researchers determined that while most cells treated with uncoated nanowires and those only functionalised with Fn were viable, those treated with NW-Fn-StxA1 suffered significant cytotoxicity and cell death. The assay also showed that the HEp-2 cells were more sensitive to the NW-Fn-StxA1 than the MAC-T cells. The researchers are continuing their studies to try to improve the internalisation of the nanowires as well as incorporating ligands that will target specific target cells. Source: www.DrugResearcher.com, August 30, 2007   Businesses urge more funding for applied nanotech R&D A report entitled 'Nanotechnology in Europe - ensuring that the EU competes effectively on the world stage' identifies a series of measures to be put in place to remove barriers to the commercialisation of nanotechnology in Europe as. The report is a result of the outcomes of a Nanoforum survey on nanotech commercialisation in Europe and a series of workshops held in June 2007. It was drafted by the European Nanotechnology Trade Alliance (ENTA ), which represents the interests of nanotechnology businesses across Europe. To overcome barriers to the commercialisation of nanotech in Europe, the report urges more application-driven funding for nanotechnology and more training and communication activities. The report also argues that "lack of venture capital and lack of patents, in comparison with the rest of the world" is a problem in Europe. Lack of capital is due to risks of nanotechnology, which are described as "imminent to investors who know little about the area". The risk areas referred to are customer acceptance, health and safety. The Commission is currently preparing a mid-term review of the EU nanotech strategy. Further reading: NanoForum press release: "Ensuring the EU Competes Effectively on the World Stage - New NanoForum Report Published" (6 July 2007) Source: EurActiv News, July 10, 2007   A cheaper way to monitor HIV In the poorest parts of Africa many patients never know their T-cell count--an indicator of the health of the immune system that helps doctors decide when to start medication and assess how well the medicine is working. A group of scientists at Harvard Medical School aim to change that. They are developing a T-cell counter the size of a business card that is inexpensive and easy to use. The device will soon be tested in Rwanda. Researchers Rodriguez and Mehmet Toner developed a small chip lined with a tiny channel 4 millimeters wide and 50 micrometers tall. The channel is spotted with molecules that bind to the CD4 receptor found on T cells. As blood flows through the channel, these molecules effectively grab the target T cells. The number of captured cells can then be counted using a simple light microscope. The microfluidics device delivered T-cell counts that were closely correlated to those collected with standard flow cytometry. Several companies have already expressed interest in licensing and commercializing the technology. Toner and his collaborators are working on further improvements, including a new version of the chip that can be read without a microscope. They have just developed a way to count cells electrically by bursting them open while still in the chamber to release the charged particles within. Tiny electrodes in the chip then measure the resulting electrical change, which is proportional to the number of cells. "Ultimately, we need a self-contained system, like the glucose-monitoring devices you can buy at the drugstore," says Toner. Source: Journal of AcquiredImmune DeficiencySyndromes (July issue) and MIT Technology Review, July 17, 2007   Vinegar 'simple and cheap' cervical cancer test A simple, inexpensive method could be used to detect cervical cancer in countries where women do not have access to Pap smears or other screening programmes, say researchers. The study &emdash; of 49,311 women between 30 and 59 years in Tamil Nadu in India &emdash; found that visual inspection of the cervix using acetic acid (VIA) is effective as a method of cervical cancer screening. The group of women who underwent VIA had a 25 per cent reduction in cervical cancer incidence and a 35 per cent reduction in deaths compared with the control group, who received existing care. The technique involves applying four per cent acetic acid (vinegar) to the uterine cervix and examining it with the naked eye under bright light. If a well-defined white area on the cervix is observed after one minute, the test is positive. The authors recommend that, to overcome problems of correctly applying the method and interpreting results, VIA should be routinely taught to health workers. Reference: The Lancet 307, 398 (2007) Source: SciDevNet, August 6, 2007     Stem cell news   EU ethics group sets guidelines for embryonic stem-cell research The European Group on Ethics of Science and New Technologies (EGE ) published its opinion on guidelines for the ethics review of EU funded human embryonic stem cells (hESC) research projects on 12 July 2007. The opinion was delivered at Commission President Barroso's request, and aims to assure that ethical rules and requirements with regard hESC in the EU's Seventh Framework Programme for research and development (FP7) are fully met. EU member states have different positions regarding the regulation of human embryonic stem-cell research and laws in different countries reflect the different ethical, philosophical and religious beliefs. As to the US, President Bush has repeatedly vetoed the use of federal funding for stem-cell research. EU's FP7 allows hESC research under certain conditions. No funding will, for example, be granted to research activities that destroy human embryos or are aimed at the procurement of stem cells, and no activity will be funded that is forbidden in all member states. In addition, research projects will only be considered for funding from member states where the research is legal. According to the EGE opinion, EU-funded human embryonic stem-cell research needs, in addition to the FP7 ethics rules, to respect the following: hESC lines have to result from non-implanted IVF embryos; if alternatives to hESC with the same scientific potential as embryo-derived stem cells will be found in the future, their use should be maximised, and; donors' rights in terms of health, informed consent, data protection and free donation have to be protected and safeguarded. Furthermore, EGE stresses the need to maximise the use of hESC lines banked in the European Registry and to take concrete actions to stimulate public debate on hESC research. EU official documents European Group on Ethics of science and new technologies (EGE): Recommendations on the ethical review of hESC FP7 research projects (20 June 2007) European Group on Ethics of science and new technologies (EGE) press release: European Group on Ethics gives its opinion on the ethics review of projects using human embryonic stem cells funded from the 7th Research Framework Programme (12 July 2007) [FR] [DE] Source: www.Euractiv.com, July 16, 2007   Scientists press EU to free up stem cell research Leading scientists have called on EU countries to remove political and legislative barriers to stem cell research, asking Germany and Italy in particular to drop threats of prison sentences against researchers that take part in European projects as ethical dilemmas remain concerning the status of the human embryo. The scientists, from the two major European-funded stem cell research consortia EuroStemCell and ESTOOLS , deplore that research projects that are perfectly legal in Sweden and the UK can result in a three-year prison sentence in Germany and that researchers from countries with very restrictive legislation might also become liable by taking on a position of responsibility in European projects. "This incongruency creates a plethora of problems for international collaboration. Despite common funding by the 6th and 7th framework of the European Commission, scientists within Europe cannot freely exchange personnel and cell lines", said the coordinator of the ESTOOLS consortium, Professor Peter Andrews. The scientists are calling for the harmonisation of current laws in the hope that their European counterparts are able to collaborate on international projects without fear of legal reprisal. Further reading: Joint statement by the Directors of ESTOOLS and EuroStemCell: The impact of legislation in Europe on our ability to perform research using stem cells Source: EurActiv News, July 27, 2007   UK gets go-ahead for hybrid embryo research Eight months after the debate started over whether hybrid embryo research should be allowed in the UK, the Human Fertilisation and Embryology Authority (HFEA) has given researchers the thumbs up - but only on a case-by-case basis. The HFEA has said it could find no fundamental reason for it to prevent cytoplasmic hybrid research. However, due to the contentious nature of the research, tight regulation would be needed. The consultation was called after Dr Stephen Minger, Kings College London and Dr Lyle Armstrong, Newcastle University, applied to the HFEA for licenses to carry out research that involved cloning human genetic material inside the shell of rabbit or cow egg. This type of research would make use of a technique known as somatic cell nuclear transfer (SCNT), which is currently legal in the UK and the USA if human eggs are used. The resulting clones, which would be 99.5 per cent human and could be used as a source of embryonic stem cells. The real need for such research is the shortage of human embryos left over from fertility treatments, from which stem cells can be generated. By creating more relevant cell lines that better represent specific disease conditions, more effective drug treatments will be able to be developed. In addition, cell lines that better represent human populations will allow more effective preclinical research into drug toxicity issues and help to reduce the need for testing in animals as well as reducing the risk when drugs are first tested in man. Source: www.DrugResearcher.com, September 6, 2007   Invitrogen takes the stress out of stem cell 'passaging' The launch of Invitrogen's Stempro EZPassage should substantially reduce the time and stress involved in dividing up cultures of human embryonic stem cell (hESC) colonies. The company claims their new disposable tool to do this is both faster than standard manual techniques and also produces more uniform results. Invitrogen's STEMPRO EZPassage is an innovative product that works fast, is easy to use, produces reliable, uniform results and makes the process of growing stem cells less intimidating for those new to the field. The new tool is ergonomically designed to reduce stress as well as being made from a cell culture-safe and inert material that facilitates the cutting of culture colonies simply by rolling the tool over the culture. This means that instead of taking 20 minutes, the passaging process can be done and dusted in just one. In addition to increased efficiency, this time reduction also means that the cells are outside of their optimal environment in the incubator for shorter periods, with Invitrogen claiming this leads to faster cell proliferation and higher cell viability. Source: www.DrugResearcher.com, August 13, 2007     Gene therapy news   Death of patient: new setback for gene therapy Seattle-based Targeted Genetics announced last week it had halted the PhaseI/II study of its investigational gene therapy for the treatment of inflammatory arthritis after one of the trial subjects died. "Even though the cause of the illness wasn't known, and is still uncertain, the agency immediately placed the trial on clinical hold - meaning no further product can be administered and no new patients can be enrolled," the US Food and Drug Administration (FDA) said in a statement last week. The FDA said that the drug tested uses an adeno-associated virus (AAV) vector to deliver the gene encoding for tumor-necrosis factor receptor (TNF-R) - a key mediator of inflammation in arthritis - directly into the joints. More than 100 subjects have been enrolled in the trial, according to the Targeted Genetics, and since it started in October 2005 no similar serious events have appeared. At the moment, there are currently around 30 gene therapy trials conducted in the US using AAV as a delivery method. They will all be reviewed again by the FDA. Source: www.DrugResearcher.com, July 31, 2007   Speculation on gene therapy trial death 'premature' Targeted Genetics said it was 'premature as well as irresponsible' to draw any conclusions on what caused the death of a patient who participated in a clinical trial testing the safety of the firm's new gene therapy. The biotech company was reacting to an article published in the Washington Post which alleged that the way the recruitment of the patient, who died on 24 July, was conducted was in serious breach of clinical trial regulation standards. According to the Washington Post report, "two fundamental rules of clinical research were violated that day [the day the patient was enrolled], experts said. First, consent forms are to be taken home and considered, not signed on first sight. Second, when a patient's own doctor is a principal investigator in a study, someone else is supposed to make the proposal." However, Targeted Genetics maintains that it stuck to the rules and that its drug is safe. More than 100 subjects have been enrolled in the trial, according to Targeted Genetics, and since it started in October 2005 no similar serious events have appeared. It is not the first casualty this area of research has recorded. In 1999, Jesse Gelsinger, an 18-year-old with a rare metabolic disease who was participating in an experimental gene therapy at the University of Pennsylvania died of a strong immune reaction to the treatment. Source: www.DrugResearcher.com, August 8, 2007     Regulatory news   Humanised antibodies lead the way in cancer approvals The latest report published by the Tufts Center for the Study of Drug Development (CSDD), found that while the number of new cancer drugs entering clinical development had more than doubled since the early 1990s, US marketing approval rates had dropped from 20 per cent for the period 1993-1997 to only 8 per cent during the mid-2000s. While overall success rates had dropped to 8 per cent for all candidates, small molecule drugs achieved a 10 per cent approval rate, while monoclonal antibodies (mAbs) of all types achieved a 9 per cent approval rate. However, humanised mAbs achieved a 14 per cent approval rate, the highest of all the classes looked at. In addition the study found that if mAbs were to fail, they failed earlier in their development cycle with mAbs suffering the lowest Phase 1-to-2 and Phase 2-to-3 transitions. However, mAbs had the highest Phase 3-to-FDA review and review-toapproval rates of all drug classes. Source: www.DrugResearcher.com, September 6, 2007   Biotech industry backs new EU biosimilars guidance EuropaBio said the European Medicines Agency's (EMEA) new document on biosimilars, entitled "Questions and Answers on biosimilar medicines", reinforces the agency's communication on biosimilars and recognises that these products cannot be classified as generics in the way standards drugs may be. In addition, it said, the EMEA document clarifies that "since biosimilar and biological reference medicines are similar but not identical, the decision to treat a patient with a reference or a biosimilar medicine should be taken following the opinion of a qualified healthcare professional". This advice against automatic substitution of one biological medicine over another reinforces the central role of the physician-patient relationship when using biotechderived therapies, said EuropaBio. Meanwhile in the US, a long-awaited regulatory pathway for the Food and Drug Administration (FDA) approval of follow-on biologics could be closer to fruition. A draft bill has recently been released that finally reaches a compromise between warring factions within the US senate over three main bones of contention regarding the new legislation, first proposed in February, which would be an amendment to the Public Health Service Act. Source: www.DrugResearcher.com, July 24, 2007   Replacing animal tests in quality control toxoid vaccines The new ECVAM Workshop Report no. 61 with the title "The Potential of Physicochemical and Immunochemical Assays to Replace Animal Tests in the Quality Control of Toxoid Vaccines" is now available. The Workshop Report can be downloaded from here or from the ECVAM Website. Please select inside the section "Publications / Workshop Reports". Source: www.ECVAMnews.com, July 25, 2007   A slow year so far for innovative drug approvals So far, the Food and Drug Administration (FDA) has only ticked off seven drugs that contain an active ingredient never marketed in the US, so-called New Molecular Entities (NMEs), or Biologic License Application (NBA) applications. This is compared to eleven in the first six months of 2006 (three of which were NBAs). Over the past decade or more, there has been a general, well-known, industry trend of increasing research and development (R&D) costs not being reflected in the number of new drug approvals. While pharma companies work out how to best deal with the problem, the pattern seems set to continue. A recent US Government Accountability Office report (GAO) revealed that between 1993 and 2004, R&D spending went up 147 per cent to $ 40bn (¤ 29bn) but the number of New Drug Applications (NDAs) only increased by 38 per cent. Most of those were for modifications to existing drugs; the number of NME applications only increased seven per cent. Source: www.DrugResearcher.com, July 17, 2007   Pharming (transgenic rabbits) gets GMP approval Dutch biotech company Pharming announced it complied with the standards of Good Manufacturing Practices (GMP) following inspections by the European Medicines Evaluation Agency (EMEA) of five facilities used in the manufacturing of Rhucin, a therapeutic protein produced in transgenic rabbits. The GMP approval, the first in the world for a transgenic rabbit facility, marks a positive step towards the anticipated European launch of the drug later this year, which would make it the second transgenic animal-derived drug on the market since last year's approval of GTC Biotherapeutic's goat-derived ATryn. Rhucin, a recombinant human C1 esterase inhibitor, is a human protein to treat acute attacks of Hereditary Angioedema (HAE), a rare disease characterised by painful swelling of soft tissue. The transgenic rabbits were able to produce between 10 and 12 grams of protein per litre of milk. This is compared to highly optimised cell cultures that can typically generate 0.2 to 1 grams of protein per litre of culture medium. Rabbits can produce up to 10 litres of milk a year. Scaling up is an easy process, basically consisting of breeding more rabbits. While Pharming awaits EMEA marketing authorisation of Rhucin in Europe, expected later this year, the company is still to apply for US Food and Drug Administration approval. An additional trial is required for the US application, which is currently ongoing but expected to be finished within the next few months, with the application to be submitted later this year. The company is also developing human lactoferrin as a food supplement, recombinant human fibrinogen for blood clotting using the protein production technology, which also includes transgenic cattle. Other firms active in the transgenic arena include US firm Hematech, owned by Japan's Kirin Brewery, which is focusing on human antibody-producing cows that will be used for the development of large quantities of polyclonal antibodies, while US-based Avigenics is pursuing avian transgenesis for treatments in oncology, infectious disease and autoimmune disease. Source: www.DrugResearcher.com, July 16, 2007     News from the Commission   EU outstrips US in scientific publishing output A new report by the National Science Foundation, a US government agency, shows a global shift in scientific-publication output. An overview of scientific articles conducted between 1988 and 2003 reveals that US share of published articles is declining as the EU and emerging countries gain expertise. The EU has outstripped the US in total number of articles published, with an average annual growth rate of 2.8% between 1992 and 2003, four times more than the US. Moreover, the report observes a large increase of scientific publications in Asian countries during the same period, with a growth rate of 15.9% for China, Singapore, South Korea and Taiwan. However, the report states that US science and research output remains influential. The number of published articles and their citation in scientific journals is an indicator of research capability, and publishing policy is considered as one of the basic pillars of research activity. Further reading: CORDIS Number of published US science and engineering articles stagnating, finds report National Science Foundation (US): Number of Published Science and Engineering Articles Flattens, But U.S. Influence Remains Strong Source: EurActiv News, July 23, 2007   EU fails potential of highly qualified women scientists According to the latest EU statistics, "She Figures 2006", women make up more than 50% of EU students and earn 43% of EU doctoral degrees but on average only get to 15% of senior academic - and thereby research - decision-making positions. In some countries and in some disciplines, these percentages are even lower. Women also only make up 18% of the scientists in the private sector and, in the majority of the EU member states, only represent less than 20% on scientific boards and panels. Thus, European research and European research policy is losing the potential of highlyqualified women scientists. In 2003, the percentage of female PhD graduates in some countries was below the European average of 43%, for example, in the Czech Republic (35%), in Belgium (36%), in Germany, Malta and Turkey (38%) and in France and the UK (42%). Other countries recorded much higher rates such as Latvia (67%), Lithuania (62%), Estonia and Romania (58%) and Portugal (56%). Other relevant statistics show that, while women represent an average of 29% of the researchers in Europe, countries like Latvia (53%), Lithuania (48%), Bulgaria (47%) and Portugal (44%) are above this average. The Netherlands (17%), Germany (19%), Belgium, the Czech Republic and France (28%) register less significant scores. Differences among countries also exist in terms of gender awareness, the gender pay gap and networking among women scientists. The factors that lead to the under-representation of women in science and research decision making are manifold. They include lack of gender awareness and persistent gender stereotypes, predominantly male decision-making bodies and insufficient network support with respect to women's career advancement. Also, the lack of transparency in recruitment procedures, gate-keeping and the operation of "old boys networks", to which women often do not have access, have an important influence on reaching the top level. Using the full potential and scientific excellence of women scientists is of central importance to the realisation of the European Research Area (ERA) and the Lisbon goal of Europe becoming the world's most competitive knowledge-based economy. The European Union must significantly increase the number of female researchers among the estimated 700,000 additional researchers needed to reach the Lisbon goal. Source: EurActivNews, September 3, 2007   MEPs hold back EIT until viable budget proposed The Parliament's industry committee is waiting for the Commission and Council to come up with a realistic solution for the budget of the European Institute of Technology (EIT), before considering adoption of the proposal in plenary this Autumn. The Parliament's plan is to vote on the proposal in the plenary at the end of September 2007. However, "before we can adopt the proposal in plenary, we have to make sure that there is a stable financing concept" added Paasilinna, urging the Commission and the Council to come up with "a realistic solution for the budget soon". Reino Paasilinna is the EP Industry Committee's rapporteur. The Parliament urges the EIT to be funded with fresh money and opposes any plans suggesting EIT financing at the expense of other important innoavtion and competitiveness programmes. The MEPs also voted, during the Industry Committee, to rename the EIT European institute of Innovation and Technology. The Council has approved the Commission's proposal to allocate some ¤ 308.7 million community money for the EIT's initial phase, from 2008-2013. It is likely that part of this amount will be assigned for the EIT from the 2008 budget. EU official documents Parliament news: Industry Committee backs creation of a European Institute of Innovation and Technology (10 July 2007) Source: EurActiv News, July 10, 2007     Agenda   13th European Congress on Biotechnology 16 - 19 September, Barcelona, Spain Information: www.ecb13.eu Advances in stem cell research 12-14 October 2007, Stockholm Sweden Further information: www.eurostemcell.org/news ACTIP meeting: focus on PAT implementation and downstream processing November 29-30, 2007, Ivrea, Italy Information: ACTIP Secretariat

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