News

Scrambling cell communications could thwart Type 1 diabetes

John Carroll — Tue, 30 Jun 2009 09:05:31 -0400

A group of researchers at Children's Hospital of Pittsburgh have been developing a novel approach to preventing the onset of Type 1 diabetes. While companies like MacroGenics, Eli Lilly and Tolerx have been working on antibodies that thwart an attack of rogue T cells responsible for the disease, the researchers in Pittsburgh are at work on a vaccine that scrambles the signals white blood cells send to call T cells to the pancreas. If you can stop the signal, they theorize, you can stop the attack. And they've begun safety tests in humans after seeing the approach work well in animal studies.

"Treating at onset in children is the best chance we have," Pittsburgh immunologist Dr. Massimo Trucco tells the AP. And he's been advancing a vaccine that is developed with a patient's blood. The safety tests are being conducted in adult diabetics whose Type 1 cases are too advanced to be affected by the treatment. But the next step will be to test the vaccine in children and determine if this new approach can stop diabetes before it begins.

- read the report from the Associated Press

Geron shares jump on GE stem cell pact

John Carroll — Tue, 30 Jun 2009 08:40:49 -0400

This morning the share price of Geron shot up 23 percent after the biotech company announced that it is teaming up with GE Healthcare to develop embryonic stem cell products that researchers can use in drug discovery work and tox screening. No terms were announced, but the new partners said that they would be using cell lines available through the National Institutes of Health's Human Pluripotent Stem Cell Registry.

A number of companies have been springing up recently to get into this promising new field. Researchers believe that stem cells have the potential to play a major role in testing new drugs. In their release, the companies note that toxicity often goes undetected until the preclinical or clinical phase of development, a blind spot that can cost developers immense amounts of time and money. These new stem cell products can shine a light on tox issues much earlier in the development process.

"Geron is intensely focused on developing hESC-based cell therapies, and the expertise that we have developed in scalable manufacturing and differentiation of hESCs to specific cell types is directly applicable to the production of these cells for drug discovery," said David J. Earp, J.D., Ph.D. Geron's SVP of business development. "In GE Healthcare we have found the ideal partner with whom to develop this near-term commercial opportunity. There is much anticipation of the availability of hESC-derived cells for drug discovery applications within the pharmaceutical industry and we look forward to working closely with GE Healthcare to deliver these promising products."

- check out the press release
- read the report from Reuters

Animal study offers new clues to Ambien 'awakenings'

John Carroll — Tue, 30 Jun 2009 08:19:15 -0400

An animal study is shedding new light on the complicated chemistry of the brain, and how a drug intended to do one thing can have entirely unintended consequences.

Researchers at Georgetown University Medical Center set out to determine why some patients who take the sleep drug Ambien wind up on their feet, talking on the phone, even driving a car while not awake. The next day, they would have no recollection of what they had done.

To mimic the effect of Ambien (zolpidem), the scientists trimmed the whiskers of mice-depriving them of neural activity-and found that some inhibitory neurons their brains used to monitor neural activity were shut down. They concluded that Ambien was similarly shutting down neurons that play a key role in inhibiting other neural activity. Without the inhibitory neurons to monitor and control activity, people taking the drug experienced "awakenings."

"When brain activity is silenced, many neurons automatically react to this change. We see this in our study which suggests that inhibitory neurons responsible for stopping neural activity are themselves shut down by zolpidem," explains Molly M. Huntsman, an assistant professor in the department of pharmacology. "The excitatory neurons, responsible for transmitting activity, are then allowed to re-awaken and become active again, without monitoring because the inhibitory neurons are 'asleep'."

- check out the press release

Scientists zero in on next-gen alpha-7 drugs

John Carroll — Mon, 29 Jun 2009 13:13:55 -0400

A key protein that plays a critical role in nicotine addiction--the alpha-7 receptor--could also be a key player in the fight against obesity, Alzheimer's, schizophrenia and a long list of other such ailments.

Researchers at Brown University have focused their attention on the alpha-7 receptor and discovered interactions with 55 other proteins. A new generation of alpha-7 drugs, they add, could make up for a loss of acetylcholine found in Alzheimer's and similar types of diseases.

"These [receptors] as a group are very important because they control a lot of critical functions, like heart rate,'' lead author Edward Hawrot tells the Boston Globe. "What was surprising was that we saw a connection between [the] proteins, because these two receptor families are very, very different.''

"When you don't have enough acetylcholine released to activate the receptors, people can't remember and they have cognitive impairment,'' Merouane Bencherif, vice president at Targacept, tells the newspaper. "What we would do is provide a drug that is not metabolized that acts on nicotinic receptors. So essentially, you're compensating for the deficit that exists in Alzheimer's disease.''

- read the article in the Boston Globe

'Trojan horse' approach eradicates cancer cells

John Carroll — Mon, 29 Jun 2009 12:58:11 -0400

Researchers for Australia's EnGeneIC have outlined a strikingly successful new approach to conquering cancer. In several preclinical studies the scientists injected minicells into animals which are designed to deliver a payload of toxins and gene inhibitors and are coated with an antibody that latches on to tumor cells. In one recent study all the mice implanted with a tumor were free of tumor cells after a 70-day treatment period. All the mice left untreated died.

A separate study using dogs with advanced brain cancer produced similar results and safety trials using human subjects begin next month in three Melbourne hospitals. Jennifer A. MacDiarmid and Himanshu Brahmbhatt add that the company is also engaged in talks to partner the cancer program.

"I consider the approach is remarkable and more than intriguing," said Dr. Stephen Friend, the former head cancer scientist at Merck who is now at Sage Bionetworks. But he adds the warning that cancer cells are so versatile that they often defeat the drugs used to attack them in humans.

The minicells are dubbed EDVs, for EnGenelC Delivery Vehicle. "The beauty is that our EDVs operate like 'Trojan Horses.' They arrive at the gates of the affected cells and are always allowed in," MacDiarmid told Reuters.

- read the article in the New York Times
- check out the story in Reuters

Optogenetics starts to shine a light on brain diseases

John Carroll — Tue, 23 Jun 2009 10:41:34 -0400

Starting from the premise that all drugs used to treat brain ailments clumsily flood the entire system with therapeutics, researchers say that fiber optics can help pinpoint the exact regions of the brain that need to be targeted when treating Alzheimer's, Parkinson's, epilepsy and other such ailments. And that approach can lead to startling insights into new therapies.

The goal is to stimulate neurons that control key functions in the brain. In animal studies researchers have used the technology to make mice run in circles and flies flap their wings. The scientists have also been able to use animal studies to suggest a new approach to treating Parkinson's.

Karl Deisseroth, a psychiatrist and professor of bioengineering at Stanford University, says optogenetics led him to the conclusion that magnets could be used to stimulate cells on the surface of the brain that play a major role in Parkinson's.

"We only could have come to this insight using optogenetics," Dr. Deisseroth says. "It has the potential to radically change the way Parkinson's is treated."

- read the report from the Wall Street Journal

Research highlights genetic causes of neuroblastoma

John Carroll — Tue, 23 Jun 2009 10:13:30 -0400

A missing stretch of DNA may provide a key clue to determining why some children are threatened with neuroblastoma. And the new research also highlights how genetic deletions or malfunctioning repetitions can trigger disease--as opposed to single-letter variations that have attracted much of the attention to date.

"This is the first paper to really show that copy number variation--which is just another mechanism of evolution for why you and I are different--can be involved in predisposition of cancer," Dr. John Maris, a researcher at Children's Hospital of Philadelphia, told Reuters. Maris has identified a new gene that plays a role in neuroblastoma. He identified three others last year.

There's already been a tremendous amount of research showing how single-nucleotide polymorphisms--SNPs--play a role in cancer. Maris and his colleagues have been exploring neuroblastoma, which is responsible for 15 percent of all cancer deaths in children.

"Only two years ago, we had very little idea of what causes neuroblastoma," Maris said in a statement. "Now we have unlocked a lot of the mystery of why neuroblastoma arises in some children and not others."

- read the report from Reuters

Scientists offer new research path for eradicating HIV

John Carroll — Tue, 23 Jun 2009 09:52:04 -0400

Drugs can stop HIV from progressing, but until now researchers have been stumped in their efforts to eradicate the deadly virus.

Now a team of researchers from VGTI Florida and the University of Montreal say that they have identified subsets of memory T-cells where the virus can linger, hidden within the body's immune system. And HIV replicates itself in the T-cells through cell division, a completely different replication process that takes place in other cells. New drugs can be developed to stop both mechanisms, they say, offering a new research avenue that can ultimately defeat HIV.

"Based on this research, we believe one possible method for eliminating HIV in the body is to use a combined approach," Rafick-Pierre Sékaly, Ph.D., current scientific director for VGTI Florida. "We propose the use of medications that target viral replication of HIV throughout the body, in combination with drugs that prevent infected memory T-cells from dividing. We believe that by attacking the disease in these distinct two ways at once for an extended period of time, we can eliminate the reservoirs of HIV that currently persist within the human body, leaving an individual disease-free."

- read the press release

Synthetic peptide effective against MRSA in animal study

John Carroll — Tue, 23 Jun 2009 08:59:49 -0400

With every antibiotic fated to become obsolescent, researchers are always working to find a new generation of antibacterials. And with the spread of drug-resistant infections on the rise, the work has never been more urgent.

Now a group of Japanese scientists say that they believe they are on to something that could prove to be a very effective guard against MRSA and other lethal bacterial infections. They say a synthetic antimicrobial peptide called L5--developed out of antibacterial proteins from Sarcophaga peregrina--effectively treated mice infected with MRSA. And when administered in advance of being infected, death rates were greatly reduced.

"...[W]e found that L5 induced the activation of the host immune responses and protected the mice from death due to infection," say the researchers. "We propose a novel therapeutic intervention that activates the host immunity in infectious diseases and has an advantage in treatment of antibiotic-resistant bacterial infection."

- check out the press release

Start-up aims at customizing monoclonal antibodies

John Carroll — Tue, 23 Jun 2009 08:46:16 -0400

With their superior binding skills, monoclonal antibodies have become one of the hottest areas of biomedical research, with around 200 programs in development. All of them hope to mimic the success of the first antibodies in use against cancer, but there is one critical hurdle that they have to overcome. A process called antibody-dependent cell-mediated cytotoxicity prevents antibodies from binding to all patients with the same specificity. And a start-up developer called PIKAMAB believes it has a new approach that can overcome that hurdle.

"A one-size-fits-all antibody drug in this case doesn't work," CEO Vijay Ramakrishnan tells MIT Technology Review. Factoring in the genetic background of the patient can make all the difference. And PIKAMAB is developing a theragnostic test that can identify just how likely it is that patients will respond to an antibody therapy. Then he wants to engineer antibodies so that they can bind to cells, making each therapy a match for one of nine different groups identified by the test.

Cancer immunologist Louis Weiner at Georgetown University tells Technology Review that he sees value in the theragnostic test, but doubts that a custom design is the best way to go in overcoming the problem. New monoclonal antibody therapies that bind better to all immune cells would be a better approach.

- read the article from MIT Technology Review

Related Article:
MAbs are hottest segment of biotech industry (2007)

FierceBioResearcher is now FierceBiotech Research

Tue, 16 Jun 2009 13:14:39 -0400

As a publisher, there is one metric I track above all else: reader engagement. By any measure of that, FierceBioResearcher continues to be one of our most successful publications. While I'm mindful not to bore you with online publishing industry terminology, we at Fierce are understandably excited about the unparalleled level of interaction we have with our readers. We measure that through our high unique open rates, click-through rates, web pages viewed per visitor, and a few proprietary in-house Fierce metrics.

In short, we judge ourselves on how relevant we are to our readers and their everyday work. We're always looking for ways to improve the publication so we can continue serving you.

I want to extend a big thanks to the hundreds of readers I've spoken to in person and by e-mail over the last few months. While I plan to put several of their suggestions to work by the end of this year, there is one immediate change we're making effective today: FierceBioResearcher is now known as FierceBiotech Research.

Outside of the name, everything else you and 34,000 of your colleagues in early stage drug development have come to rely on remains the same. John Carroll, our Editor-in-Chief, will continue bringing pre-clinical research and development news and analysis with his authoritative editorial voice. We're making the name change to better align our weekly publications with their parent publications FierceBiotech and FiercePharma--it's a minor touch that we're confident readers will appreciate. I'll have more on this in the coming weeks and months.

As always, I'm eager to hear questions and comments from our readers, so please get in touch. Thanks for your continued participation in the FierceBiotech Research community.

Arsalan Arif, Publisher
(E-mail | Twitter)

PS: Please remember to add editors@fiercebiotechresearch.com to your safe senders list. Next week, FierceBiotech Research will be sent from this new address.

Lilly opens a portal to new drug collaborations

John Carroll — Tue, 16 Jun 2009 09:51:36 -0400

Eli Lilly is opening up a web portal that might just open up new collaborative initiatives with lab researchers around the world.

Called the Lilly Phenotypic Drug Discovery Initiative, or PD2 (PD-squared), the portal will employ Lilly's disease-state assays to evaluate compounds synthesized at university and biotech labs around the globe. After biological testing is completed, Lilly provides the outside researchers a data report including a complete biological profile of the compound across four assay modules: Alzheimer's disease, cancer, diabetes and osteoporosis. And Lilly says the in vitro model systems will provide researchers with broader assessments of a compound's biological profile than what is generally available today in academic or government laboratories.

In return for the data, Lilly gains first rights to exclusively negotiate a collaboration or licensing agreement with the researchers who submit their compounds.

"Each year, researchers throughout the world design and synthesize compounds in university and biotechnology laboratories that are never fully evaluated as potential drug candidates," said Alan D. Palkowitz, Ph.D., vice president of discovery chemistry research and technologies at Lilly. "There's an untapped source of ideas and compounds in the greater scientific community that could ultimately impact patients' lives following further evaluation and development."

- read the Lilly release

Facing attacks, Iowa sends research underground

John Carroll — Tue, 16 Jun 2009 09:27:04 -0400

Faced with the threat of attacks from animal rights activists, the University of Iowa is taking its animal research lab underground.

The university's board of regents has approved an $11.1 million budget to create a 35,000-square-foot vivarium that will be dug out under a grassy courtyard. The new lab will connect the university's Medical Education Research Facility and the Carver Biomedical Research Building, providing greater security with no need to transport research animals above ground. The board noted that other research centers, including the University of Southern California, have also built underground labs to protect researchers and their work from attack.

It's not a vague threat. Five years ago the Animal Liberation Front claimed responsibility for organizing a break-in by masked intruders at the University of Iowa in which computers were trashed and hundreds of research animals were stolen. And in southern California researchers' homes and vehicles have been subject to attacks as well. Even more aggressive strategies are used by animal rights groups in the UK, which have organized campaigns to intimidate researchers who undertake experiments with animals.

- read the report from the Des Moines Register

Ex-Pfizer scientists, university partner on drug program

John Carroll — Tue, 16 Jun 2009 08:38:14 -0400

Wayne State University has teamed up with an innovative company created by a group of former Pfizer scientists to advance a new therapy discovered in one of its academic labs into clinical development.

The Michigan Technology and Research Institute, founded in Ann Arbor by seven Pfizer scientists after the pharma giant left town two years ago, will undertake early-stage research with the help of a $118,000 grant from the state. At the same time the institute will develop a business plan and reach out to venture capital groups who may be interested in backing a new biotech company.

The institute's scientists have been consulting with Michigan State and the University of Michigan. The Wayne State project involves an experimental therapy discovered by Aloke Dutta, who teaches pharmaceutical sciences. The drug targets dopamine, a new approach to treating depression. And the same therapy could be effective in Parkinson's disease, pain and substance abuse, according to the Detroit News.

"It holds a great deal of promise," said David G. Pegg. "Depression is an area of medicine where there is a need for new drugs that are safer and more effective."

- read the story from the Detroit News

India forecasts fast-paced growth in stem cell research

John Carroll — Mon, 15 Jun 2009 17:14:12 -0400

Following the Obama administration's decision to loosen the purse strings on federal support of stem cell research, scientists at the Stem Cell Research Forum of India expect discovery work in the subcontinent to barrel ahead to an estimated $540 million next year with an annual growth rate of 15 percent.

"India is a cheaper alternative for research and clinical trials. The country is not too far behind the US in terms of practical aspects of stem cells. Many companies are eyeing India because of its huge population base and genetic versatility," said Karan Goel, founder and chairman of Stem Cell Global Foundation.

Vishwa Mohan Katoch, the director general of the Indian Council of Medical Research, said the council plans to develop new collaborations with U.S. institutions. "It's important to develop the public-private partnership in the field of stem cell research and therapy, as it has a direct impact on the translation of technology. You may see many such partnerships in future."

"There are several laboratories that are researching stem cells. A lot of this is tailored to address diseases that are important in the local context and this stimulates innovation," said Maneesha Inamdar, associate professor for vascular biology laboratory and stem cell laboratory at the Jawaharlal Nehru Centre for Advanced Scientific Research.

- read the article from Economic Times

Blocking key molecule could blunt acute cancer pain

John Carroll — Mon, 15 Jun 2009 09:55:51 -0400

Cancer tumors produce a hormone-line molecule that spurs the growth of nerve endings in neighboring tissue--which is partly responsible for the acute pain triggered by the disease. And German scientists say that a new drug that blocks that molecule would be a valuable addition to the pharmacopeia of cancer therapies needed to treat patients.

Researchers at Heidelberg University spotlighted the role of the molecules in triggering pain. The same molecules also are important in the development of blood cells in bone marrow. Now the backers of the study want developers to target the pain right at the tumor site, which would significantly improve the lives of cancer patients while limiting the side effects of the disease.

"Identifying one of the ways in which cancer causes pain--in fact, perhaps the main mechanism--is a crucial step towards drugs that could bring relief to cancer sufferers across the world," Dr. Mark Matfield, scientific adviser to the Association for International Cancer Research, tells the BBC. The AICR helped fund the study.

- read the report from the BBC

Farm animals make good models for research

John Carroll — Tue, 09 Jun 2009 09:53:28 -0400

Mice may be the animal of choice for many in medical research, but scientists are finding that barnyard animals may be a better choice. "Farm animals are more closely related to humans genetically and physiologically," said Jim Ireland, a professor of animal science and physiology at Michigan State University. Report

Genetic engineering controls obesity in animal study

John Carroll — Tue, 09 Jun 2009 09:36:01 -0400

Transplanting the fat-burning pathway of bacteria into mice allowed the rodents to eat a high-fat diet while remaining lean. And the results of the study point to a whole new approach to treating ailments in humans who could benefit from some other species' biologic functions.

Senior author James Liao said that the scientists concentrated on the glyoxylate shunt, a pathway made of two enzymes that a cell in bacteria uses to convert fat to sugar. Liao believes that humans don't have this mechanism because our bodies have been conditioned to store fat rather than burn it.

By introducing the genes for the enzymes into human cells, the scientists found that the new mechanism didn't convert fat, it burned it away entirely. The genes triggered a cellular response that caused cells to metabolize fat. The team then transplanted the genes into mice livers and found that normal mice became fat while the genetically engineered mice "remained skinny despite the fact that they ate about the same and produced the same waste."

- read the story in MIT Technology Review

NY gov offers $100M to leverage federal funds

John Carroll — Tue, 09 Jun 2009 08:03:01 -0400

Faced with a perfect political storm brought on by low poll numbers and declining tax revenue, New York Gov. David Paterson has come up with a novel plan aimed at ginning some big bucks for biomedical research. The governor is setting aside $100 million for research institutions in the state, and he wants to leverage each dime in that budget for $1 in federal stimulus money.

Medical research, energy and technology will all be boosted with new state money, says the governor, whose plan drew a mixed response from a wide slate of political friends and foes. Everyone, though, seemed to agree that boosting the state's research institutes, which includes some of the top biomedical research labs in the country, made sense at a time Wall Street is producing less and less tax revenue.

"We love the tax revenues [the financial markets] generate and the jobs that they create, but the concentration and dependence on any one industry is great in good times, bad in down times," said New York Mayor Bloomberg said.

F. Michael Tucker, CEO of the Center for Economic Growth, said that grant applications with state backing will stand a better chance in gaining federal support. And the research groups will be linked with private companies to help drive the fruits of their work to market.

- here's the Buffalo Business Journal story
- read the report from the New York Daily News

Lab worm points to new ways to extend life span

John Carroll — Tue, 09 Jun 2009 07:38:14 -0400

Scientists exploring ways to significantly increase our longevity have been focused on the insulin-signaling pathway. Deactivating a gene in the pathway can extend the lives of animals, probably for the same reasons that extreme low-calorie diets produce a similar effect, reports the New York Times.

The insulin-signaling pathway also activates a gene regulator-dubbed FOXO in humans-which controls a variety of gene pathways, including metabolism. Sean P. Curran and Gary Ruvkun led a team at Massachusetts General Hospital which concluded that it flips the switch on two genes in ordinary body cells (somatic cells) that are typically only active in germline cells, which produce eggs and sperm --the essential ingredients for new life. The two genes protect the cells' DNA, and in turn add to longevity.

"This ability for somatic cells to gain a stemlike character could be really important in extending life span," Dr. Ruvkun told the Times. The lesson here is that if you can find new ways to protect somatic cells, you can extend life. The Mass General team studied the lab worm C. elegans. The work now will extend to studying the gene regulator in mice and people.

- read the story in the New York Times

Nobel Prize-winner will head to Max Planck in Florida

John Carroll — Mon, 08 Jun 2009 13:45:30 -0400

The Max Planck Society has recruited a Nobel Prize-winning scientist to become the science director of its new research center in Palm Beach County, Florida. Dr. Bert Sakmann won the Nobel Price for physiology and medicine in 1991 and has plans to develop a three-dimensional atlas of the brain that can used to advance new approaches to curing Alzheimer's and other degerative diseases.

Sakmann and his researchers--who he's recruiting now--will create a three-dimensional atlas of nerve cell bodies, dendrites and axons by first labeling the different cell types with specific fluorescent markers and then imaging and quantifying the neuron distributions to create a three-dimensional map of the normal brain.

"The research will not only help further our basic understanding of the degenerative disease process, but could also serve as a platform to measure the beneficial changes caused by novel therapeutic approaches such as new drugs and stem cell therapy," the institute said. Sakmann currently works at the Max Planck Institute of Neurobiology in Munich.

Florida and Palm Beach County have divvied up the costs for the $188 million research facility, which will occupy 100,000 square feet of space when the center opens in 2011.

- check out the press release
- read the report from the Palm Beach Post

Agency lures biotechs with $20M for stem cell teams

John Carroll — Fri, 05 Jun 2009 09:50:34 -0400

California's stem cell agency is mapping out ambitious plans to provide $20 million to each of 10 to 12 research teams that can be in the clinic with a new therapy inside of four years. And the agency's chief expects a biotech company to be included in the teams.

Agency president Alan Trounson says that it's time to start moving stem cell work out of the lab and into human trials. And he says that this is the right time to partner up with biopharma companies that are being lured into the stem cell field.

"Pharma is moving into this space in a big way," Trounson told Bloomberg in an interview. "They're now generally interested in cell therapy. That's a big change."

California's Institute of Regenerative Medicine has spurred big joint investments in new research facilities in the state, leveraging its $3 billion in grant money in partnerships with state universities. But after the $3 billion runs out, the agency may be able to continue to operate using industry funds. And these initial stem cell teams are a step in that direction.

- read the report from Bloomberg

Blood-pressure med reverses liver damage

John Carroll — Tue, 02 Jun 2009 10:14:27 -0400

A blood-pressure medicine has been shown to reverse the effects of early-stage liver failure in some patients. Newcastle University researchers analyzed a small clinical trial of losartan, a drug normally prescribed for hypertension, on 14 patients in Spain, who had Hepatitis C. The illness was at an advanced stage causing fibrosis--scarring in the liver--which would usually have progressed to liver failure. Half of the patients in the trial saw the scars in their liver shrink, allowing the organ to repair itself. Release

Advanced melanoma therapy effective in early trial

John Carroll — Tue, 02 Jun 2009 10:00:44 -0400

A small, early-stage clinical trial of an experimental skin cancer drug delivered some impressive results. Researchers for Plexxikon and Roche say that more than half of the patients taking PLX4032 (R7204) saw the extent of their malignant melanoma--the most lethal form of skin cancer--reduced by at least 30 percent.

The drug targets tumor cells with a BRAF mutation, a condition common to 60 percent of all skin cancer cases. People taking the drug in the 16-patient study lived an average of six months without disease progression. Now Roche and Plexxikon are working on a diagnostic test to identify BRAF-positive cases as they pursue larger clinical trials.

"While these results are interesting, they need to be followed up in much larger studies before we know if this is a suitable new treatment for people with advanced melanoma," said Cancer Research UK's Dr Jodie Moffat. "Melanoma is difficult to treat when it is at an advanced stage so it's crucial to find new treatments to help beat the disease."

- read the report from the BBC

Related Article:
Bullish Roche to reveal new cancer data at ASCO

Cancer researchers ID new drug target

John Carroll — Tue, 02 Jun 2009 09:23:01 -0400

Researchers at Children's Hospital in Boston have identified a protein that appears to play a key role in about 15 percent of all cancers, offering a new drug target that could be particularly effective for hard-to-treat patients.

George Daley, Srinivas Viswanathan and Richard Gregory say that the protein LIN28 pushes a cell to a cancerous state and appears abundant in advanced cancers like liver cancer, ovarian cancer and acute myeloid leukemia, according to a report in The Medical News. A drug that inhibits LIN28 could have wide application, they believe.

"Linking this protein to advanced cancer is a very exciting new result," says Daley, Director of Stem Cell Transplantation at Children's. "It gives us a new target to attack, especially in the most resistant and hard-to-treat cases. LIN28 is a fascinating protein that acts both in stem cells and cancers, and is teaching us that cancer is often a disease of stem cells."

- read the report from The Medical News

Transgenic monkeys made for research programs

John Carroll — Tue, 02 Jun 2009 09:09:18 -0400

Viral DNA was used to introduce a gene for a green fluorescent protein into monkeys, creating a new line of "glow-in-the-dark" transgenic primates for drug research work. Researchers involved in the instantly controversial project say this is the first time that a monkey has been genetically engineered so that a new gene entered into its DNA and was passed down to its offspring.

Scientists were able to create a transgenic monkey last year that could be used to study Huntington's disease, but the genetic rearrangement wasn't passed down to subsequent generations. Now scientists say this new approach will be able to develop a line of primates that can be genetically orchestrated to more closely match humans. That would give drug makers a better animal model for preclinical studies as they try to determine a drug's chances of working in people. In particular, the transgenic animals--which glow green under a fluorescent light--are expected to lead the way to new animal models that can be used for neurological diseases like Parkinson's and Huntington's disease.

The work by Japanese scientists was published in Nature and heralded by drug researchers. But animal rights groups and some bioethicists were quick to condemn the genetic engineering work.

"We may find ourselves gradually drifting towards the genetic engineering of human beings," said Dr David King, from the group Human Genetics Alert.

- read the report from the Washington Post
- read the story from Sky News with some of the reaction

'Voila!' Safer patient-specific stem cells

John Carroll — Mon, 01 Jun 2009 16:41:51 -0400

A team of scientists from Harvard and Advanced Cell Technology have devised a new and evidently safer way to reprogram skin cells into induced pluripotent stem cells. They soaked skin cells in genetically engineered proteins fused with a peptide, which transported the proteins into the cells and made them act like embryonic stem cells capable of developing into a wide variety of tissues.

Over the past few years scientists have been working feverishly to develop a safe and efficient way to spur stem cells to act like ESCs. Genetic reengineering was successful in producing ESC-like cells, but raised some potentially catastrophic health risks. This new process could make it much easier for stem cell companies to do their work without using actual ESCs, which has caused a significant amount of controversy. And ACT believes it's close to making this a mainstream practice.

"After a few more flight tests--in order to assure everything is working properly--it should be ready for commercial use," ACT's Robert Lanza told Reuters. "This method eliminates the risks associated with genetic and chemical manipulation, and provides for the first time a potentially safe source of iPS cells for translation into the clinic. This is the ultimate stem cell solution--you just add some proteins to a few skin cells and voila! Patient-specific stem cells!"

- read the report from Reuters
- check out the AFP story

Salk researchers unveil breakthrough on genetic diseases

John Carroll — Mon, 01 Jun 2009 16:39:20 -0400

Researchers at the Salk Institute for Biological Studies in La Jolla, CA, have combined stem cell and gene therapy techniques into a new approach that could point the way to a cure for some of the world's most devastating ailments.

Their research project involved taking a defective cell from patients suffering from Fanconi anemia--a genetic disease that can trigger leukemia and other fatal diseases--and correcting the defective gene that causes the condition. The repaired cells were then made into induced pluripotent stem cells, or iPS cells, which readily differentiated into hematopoietic progenitor cells primed to differentiate into healthy blood cells.

"We haven't cured a human being, but we have cured a cell," says the study's leader, Juan-Carlos Izpisúa Belmonte. "In theory we could transplant it into a human and cure the disease."

"If we can demonstrate that a combined iPS-gene therapy approach works in humans, then there is no limit to what we can do," says Inder Verma, who worked on the project.

- check out the press release

Oxford team engineers virus to attack cancer cells

John Carroll — Tue, 26 May 2009 10:20:03 -0400

A team of researchers at Oxford University say that they have refined their ability to engineer a virus that targets specific cells, offering a new approach to treating cancer and creating a new generation of vaccines for viral diseases.

This new approach maintains "wild type" virus potency for tumor cells but is turned off for other tissues. This engineered virus worked in mice, but the scientists note that it will have to be re-engineered again before it can be tested in humans.

"This approach is surprisingly effective and quite versatile. It could find a range of applications in controlling the activity of therapeutic viruses, both for cancer research and also to engineer a new generation of conditionally-replicating vaccines, where the vaccine pathogen is disabled in its primary sites of toxicity," Professor Len Seymour says.

- read the press release

Report: Collins is leading candidate for NIH job

John Carroll — Tue, 26 May 2009 10:00:56 -0400

Francis Collins, a pioneering geneticist, is the leading candidate for the top job at the NIH, according to a report in Bloomberg.

Quoting sources, Bloomberg says that Collins has the inside track on the job, which looks a lot more attractive now that the Obama administration is funneling billions of additional dollars to support scientific research work at thousands of labs around the U.S.

The NIH is immensely influential in drug research, operating a network of 27 scientific institutes and centers that employ 18,000 people. Collins ran the National Human Genome Research Institute as it cracked the sequence of the human genome.

"NIH is a huge enterprise, and I think Francis has very good experience with getting the best out of a huge enterprise from what he did in the genome project," biologist David Baltimore told Bloomberg in February. "He's also very well liked in Congress."

- read the report from Bloomberg