Biotechnology

Pfizer to Study Liver Cancer in Korean Patients with Samsung Medical Center

Pfizer formed a research partnership with Samsung Medical Center to generate gene-expression profiles of tumors from Koreans with liver cancer. The hope is that the findings will lead to targeted therapeutics that can be used not just in Korea but also in the rest of Asia.

A research team led by Samsung Medical Center scientists including Prof. Park Cheol-Guen, Prof. Im Ho-Young, and Prof. Paik Soon-Myung, director of the cancer research center, will conduct research in Seoul. Neil Gibson, Ph.D., vp of oncology research, will be responsible for the joint research program at Pfizer.

Samsung Medical Center has built a base of specimens in the liver cancer area. “This partnership will serve as a great opportunity to combine Pfizer's know-how in drug development and Samsung Medical Center's extensive genome information and technology in the liver cancer area,” says Dr. Gibson. “We further plan to share the ownership of collected and analyzed data with Samsung Medical Center.”

Pfizer signed a memorandum of understanding with the Ministry of Health and Welfare in 2007, agreeing to invest $300 million in R&D in Korea. As part of its commitment, the company also formed a strategic partnership with the Korea Research Institute of Bioscience and Biotechnology and has been leading joint research since then.

In February Pfizer linked up with Eli Lilly and Merck & Co. to set up the Asian Cancer Research Group (ACRG) to concentrate on drug R&D for the most common cancers in Asia. The nonprofit company will initially focus on lung and gastric cancers, which are two of the most common cancers in Asia.

The aim for ACRG is to generate a pharmacogenomic cancer database comprising data from about 2,000 lung and gastric cancer tissue samples. The resulting data will be made publicly available to researchers and expanded through the addition of clinical data from a longitudinal analysis of patients.

The ACRG will initially establish collaborative relationships throughout the Asian region to collect tissue samples and data. “The ACRG is about sharing information for the common good,” stresses Kerry Blanchard, M.D., Ph.D., vp and leader of drug development in China for Lilly.

Aestus and Eisai Ally to Develop Treatments for Psychiatric Disorders

Aestus Therapeutics and Eisai have agreed to work together on the R&D of therapeutics for psychiatric disorders. The multiyear collaboration will entail preclinical validation of Aestus’ compounds leading to clinical proof-of-concept studies.

Aestus’ candidates were identified through its proprietary analysis of gene-expression datasets, coupled with biological pathway mining on data from relevant disease models. Along with Eisai, the firm will work toward picking lead candidates for further development.

Aestus reports that it has several products for neuropathic pain that are ready to enter Phase II studies. On June 28, Aestus licensed Astellas Pharma’s FK614, a PPAR-gamma agonist, to develop the product for postherpetic neuralgia. The first clinical trial is expected to start later this year. On June 9, the company was awarded a $2 million SBIR grant from the NIH to advance its pain candidates.

Beyond neuropathic pain, the Aestus technology is currently being applied to ALS and schizophrenia. On March 17, the ALS Therapy Development Institute and Aestus reported plans to test potential small molecule compounds to slow or stop the progression of ALS.

Pfizer and SMC Collaborate on Liver Cancer

Source:PRNewswire

NEW YORKJuly 14 /PRNewswire-FirstCall/ — Samsung Medical Center and the world's leading bio-pharmaceutical company Pfizer Inc. (NYSE: PFE) announced that they have formed a research partnership to jointly analyze tumors from Korean patients to generate gene expression profiles and that may ultimately direct therapies and enhance clinical outcomes in the patients with liver cancer.

(Logo: http://photos.prnewswire.com/prnh/20100416/PFIZERLOGO )

(Logo: http://www.newscom.com/cgi-bin/prnh/20100416/PFIZERLOGO )

The two organizations held a signing ceremony at the main conference hall located on the fifth floor of Samsung Medical Center in Seoul on June 14 to commemorate the initiation of the collaboration.  A research team led by top scientists at Samsung Medical Center, including Prof.Park Cheol-Guen, Prof. Im Ho-Young and Prof. Paik Soon-Myung, Director of the Cancer Research Center, will conduct research in Seoul, while Dr. Neil Gibson, Vice President of Oncology Research, will be responsible for the joint research program at Pfizer.

Pfizer expanded into the market for targeted anticancer agents with the launch of Sutent, an anticancer agent used to treat an advanced form of kidney cancer.  Since then, the company has been consistently investing in research and development of innovative drug candidates and potential treatments for patients with liver cancer, a type of cancer especially prevalent in Asia, to address the growing need for an anticancer drug treating liver cancer in the Asian market in the future.  Seeing the world-class clinical environment and outstanding research capabilities in Korea, the global pharmaceutical company formed a research partnership with Samsung Medical Center as part of its commitment.

Samsung Medical Center has been laying the solid foundation for development of new anticancer drugs, providing top-quality medical service through organic collaboration with other organizations; building an extensive base of specimens in the liver cancer area; and accumulating the know-how for diagnosis and treatment of liver cancer.  Samsung Medical Center was named to join the "Group of Leading Research-based Hospitals," a large-scale national project led by the Ministry of Health and Welfare in 2009, to work with leading local and global pharmaceutical companies for an open research project designed to develop novel bio drugs that will help the country to cure intractable diseases.

"We are pleased to have an opportunity to work with the world's No. 1 pharmaceutical company Pfizer to better understand cancer in Korean patients, with the goal of being able to send a new message of hope for patients with liver cancer across the world (especially in Asia)," said Choi Han-Yong, president of Samsung Medical Center.

"This partnership will serve as a great opportunity to combine Pfizer's know-how in drug development and Samsung Medical Center's extensive genome information and technology in the liver cancer area," said Neil Gibson, vice president of Oncology Research Unit in Pfizer Inc.  "We further plan to share the ownership of collected and analyzed data with Samsung Medical Center, contributing to advance of a variety of oncology research in Korea."  Pfizer signed a memorandum of understanding with the Ministry of Health and Welfare in 2007, agreeing to invest 300 million dollars in R&D in Korea.  As part of its commitment, the company also formed a strategic partnership with the Korea Research Institute of Bioscience and Biotechnology and has been leading joint research since then.

Cavity Fighting Bacteria

We've all been taught that the way to prevent cavities is to brush, floss, and visit the dentist regularly. Researchers at the University of Florida have taken a different approach to fighting tooth decay. They have altered the bacterium Streptococcus mutans, which is known to cause tooth decay, so that it is no longer harmful to teeth.

The leading researcher in the study, dentist J. D. Hillman, accomplished this task by stripping the bacterium of its ability to produce lactic acid. It is this byproduct of the breakdown of sugar by Streptococcus mutans that causes tooth decay. If the bacteria are not able to produce lactic acid tooth decay is stopped.

As many as 500 different species of bacteria inhabit your mouth and can colonize on your teeth and gums. When you eat a meal bacteria help to digest the food and sugar left on your teeth and gums. In the process lactic acid is produced which breaks down tooth enamel and leads to cavitiesStreptococcus mutans has been found to be the most cariogenic (promotes tooth decay) of these bacteria.


Streptococcus mutans. Image courtesy of Fusa
o Ota, University of Tokushima, Japan.
 

The genetically altered strain of Streptococcus mutans appeared to thrive on sugar. It was tested on rats with positive results. Researchers introduced a solution containing the bacterial strain into the oral cavity of rats. The rats were fed a high sugar diet and showed no evidence of tooth decay. Researchers found that the strain was able to stay on the surface of the teeth indefinitely and prevented the natural strain from colonizing on the teeth. The altered strain is genetically stable and no ill effects have been noted.

Hillman is hopeful that human trials will begin this year. These trials will attempt to determine the number of applications needed to prevent tooth decay permanently. Researchers warn that this does not mean that you can get rid of your tooth brush. Brushing and other forms of dental hygiene would still be recommended to prevent plaque build-up.

 

 

Gene Therapy Reverses Muscular Dystrophy

Using a novel approach, scientists at the Children's National Medical Center and the University of Pittsburgh have successfully repaired muscles in hamsters that were ravaged by a form of muscular dystrophy called limb girdle muscular dystrophy.

In the study, the researchers used a non-replicating adeno-associated virus (AAV) as a gene vector. The virus carried a gene for a component of skeletal muscle called sarcoglycan protein. This protein is not made properly by people suffering from limb girdle muscular dystrophy. AAV is unique in that it does not provoke a response from the immune system and can reside in cells without initiating a response. Once integrated within a cell, it will stay there permanently. This allows the sarcoglycan protein to be produced over the long term.

The AAV vector along with the sarcoglycan gene was injected into the leg muscles of hamsters with limb girdle muscular dystrophy. After approximately a month, the treated muscles were tested and the results were startling. The muscles had increased almost 100 percent in strength and were nearly normal in size. This was the first time that researchers had observed entire muscles being functionally repaired and restored after being ravaged by the effects of muscular dystrophy.

Limb girdle muscular dystrophy causes degeneration of the large muscles attached to the hips and shoulders. It is fatal and has no known effectiby limb girdle muscular dystrophy and perhaps other forms of muscular dystrophy as well.

Can Microbes Help Stem the BP Oil-Spill Disaster?

Current technology used by BP is proving hopelessly inadequate in curbing the tide of oil gushing from beneath the Gulf of Mexico. Now, novel technologies that remain untested at such an incomprehensible scale will be needed in anticipation of years of remediation to resuscitate a dying ecosystem.

“It is of grave concern,” David Kennedy of the National Oceanic and Atmospheric Administration(NOAA), said back in April. “And the efforts that are going to be required to do anything about it, especially if it continues, are just mind-boggling.”

On March 24, 1989, the tanker Exxon Valdez en route from Valdez, Alaska, to Los Angeles ran aground on Bligh Reef in Prince William Sound, Alaska. It spilled approximately 10.9 million gallons of its 53 million gallon cargo of crude oil. Driven by a storm, the oil quickly covered about 1,100 miles of noncontinuous coastline in Alaska. By comparison, as of June 11, 90 million gallons of oil had spurted into the Gulf, according to the new worst-case estimate from the government, which anticipates 20,000–40,000 barrels (about 42 gallons per barrel) leaking daily. This number keeps changing, though and has even gone up to 60,000 barrels a day.

Coast Guard Admiral Thad Allen, who leads the government's relief effort, said in June, “We're no longer dealing with a large, monolithic spill. We're dealing with an aggregation of hundreds of thousands of patches of oil that are going in a lot of different directions.” He noted that while cleaning up the oil spill on the surface will go on for a couple of months after the well is plugged, long-term issues of restoring the environment and the habitats will take years.

Bioremediation may have some role to play in that restoration provided the cure isn’t worse than the disease. Bioremediation involves using microorganisms or their enzymes to return environments altered by contaminants to their original conditions. In the case of oil spills multiple techniques may be used, including the addition of nutrients to the environment to enhance and facilitate crude oil decomposition by specific bacteria or the introduction of oil-eating bacteria. The former approach was used as part of the cleanup effort after the Exxon Valdez spill. The addition of bacteria has been less successful.

Lessons from Exxon Valdez

Bioremediation protocols employed during cleanup of the Exxon Valdez oil spill effectively demonstrated that application of nutrients in the form of fertilizer (nitrogen and phosphorus) could increase oil biodegradation rates. At the time, however, fertilizers used to boost bacterial populations caused some concern, mostly focused on the 2-butoxy-ethanol component in Inipol™ and its potential toxicity to wildlife and cleanup workers. Besides safety guidelines being followed to protect workers during application of fertilizers Inipol and Customblen, wildlife deterrents were used during the first 24 hours when toxicity was of most concern.

Ten days after treatment, the surfaces of the oil-blackened rocks on the shoreline turned white and appeared to be free of surface oil. The striking visual results strongly supported fertilizer application, which sustained higher numbers of oil-degrading microorganisms in oiled shorelines, according to EPA’s 1990 interim report. Additionally, the EPA noted, biodegradation rates were enhanced as evidenced by the chemical changes detected in recovered oil from treated and untreated reference sites.

Oil-Eating Microbes

But while encouraging microbe growth with biostimulation on contaminated shorelines appears useful, unleashing oil-eating microbes themselves has produced less than stellar results. For example, biotreatment using a bacterial culture from Alpha Environmental was deployed following a spill that occurred on June 8, 1990, following an explosion on the Norwegian tanker Mega Borg. An estimated 100,000 barrels of crude oil were burned or released into the Gulf of Mexico, 57 miles southeast of Galveston, during the next week.

Bioremediation tests were conducted on June 15 and 18, 1990. “These were the first tests of a bioremediation agent on an oil spill in open waters in the United States,” the NOAA incident report noted. “The bioremediation agent used was AE BioSea Process, developed by Alpha Environmental. AE BioSea Process contains oil-metabolizing bacteria and nutrients. The results of the tests were inconclusive.”

While the Texas General Land Office said that the bioremediation was effective, independent observations indicated that treated oil changed in physical appearance and may have emulsified as a result of addition of the Alpha product. Chemical analyses on samples from impacted and reference sites failed to demonstrate that treatment with the Alpha product enhanced rates of petroleum biodegradation.

 

Genetic Engineering vs. Experimental Evolution

Evolugate, a Florida company developing microbes for bioremediation, says that adding microbes to the site of a spill hasn’t worked well before because the microbes haven’t been adapted to the specific environment in which they need to survive. The firm is evolving microbes specifically to survive on oil from the Gulf spill in the Gulf environment by maintaining populations of microbial cells under controlled conditions of growth and environment for an indefinite duration. It says that this is a prerequisite for experimentally evolving natural isolates of wild-type species or recombinant strains.

The company grows the microbes in proprietary continuous cell culture vessels to select microbes that have higher proliferation rates under specific conditions. The innovation behind Evolugate’s continuous culture vessels is that they are engineered to prevent microbes from sticking to the walls, a common strategy by which microbes evade selective pressure in other continuous culture technologies.

The Evolugate technology works via partial dilution: As a culture grows and becomes saturated, a small proportion of the grown culture is replaced with fresh medium, allowing the culture to continually grow at close to its maximum population size. Thomas Lyons, Ph.D., principal research scientist and board member of the firm, told GEN that in adapting the microbes for the Gulf oil spill, “we add more microbes every day to bolster genetic diversity.

“When we first started the culture we saw a die-off, and we expected that the dispersants and oil in the Gulf water-containing medium would kill some microbes. But after one week we saw a huge increase in cell density suggesting that adaptive variants arose. Within two weeks we already have robust growth on oil samples taken from the Gulf.

 “The beauty of what we do is that we have built in evolutionary trade-offs: The longer the microbes spend evolving to the oil the less robust they become under other conditions. Once the oil is gone they will lose their competitive advantage and will no longer survive in that environment.”

Dr. Lyons noted that producing such designer microbes through genetic engineering would be hard to pull off. Oil is so full of complicated substances that jamming all the genes needed to digest and metabolize it into a single microbe and then expecting it to reproduce and flourish might be asking too much, he said. Experimental evolution, on the other hand, simultaneously changes metabolic capabilities as well as optimizes growth rates.

He also pointed out that right now the company’s proposal to select and introduce designer oil-eating microbes into the Gulf is in BP’s hands. “It’s in their pipeline, but we are not waiting for a response. We know our approach stands the best chance to make bioremediation work, and we are proceeding accordingly. ”

Genetic engineers did indeed have a shot at enhancing oil-eating microbes. One species of oil eaters in the genus Pseudomonas was used during the Valdez cleanup, but it didn’t work efficiently or very quickly. The oil-eating superbug was developed at General Electric in 1975 by Ananda Mohan Chakrabarty, Ph.D., now distinguished professor of microbiology and immunology at the University of Illinois college of medicine laboratories.

To underscore Dr. Lyons’ point, while there are four oil eaters in this bacterial genus, each uses a different component of the oil as its food source and they all compete with one another when added to the same oil sample. In 1981, Dr. Chakrabarty received a patent on a genetically modified Pseudomonas bacterium that would eat up oil spills, the first patent of its kind; he was the first person to win a patent on a living organism. 

Dr. Chakrabarty and his team inserted plasmids from all four species of the oil eaters and put them into a single microbe. While these plasmids would usually not operate together in the same cell, exposing the cell to ultraviolet light caused the plasmids to join into one that could express components of all four pathways of the original plasmids so that several oil components could be broken down.

 

Time for Creative Thinking

One problem with this creative approach to digestion, however, is that the microbes don’t eat enough nor quickly enough; it takes a lot of them a few days to go through an eyedropper full of oil. There is also concern over what the environmental impact of releasing engineered bacterial strains might be. Additionally, some have pointed out that in the case of oil-eating bacteria, oil companies might not want organisms literally eating their lunch.

Naturally seeping oil in the Gulf already feeds some microbes; low level inputs of hydrocarbons to the oceans, including natural slow oil leaks into the Gulf of Mexico in the form of geological seeps, pine droppings, etc., exist as natural marine ecosystem components. Thus, according to marine biologists, some bacteria within the marine microbial community have evolved the ability to break down hydrocarbons and exploit the considerable energy stored in the chemical bonds of these compounds.

Such organisms aren’t very prevalent, though, since other sources of food are more appealing, pointed out Terry Wade, Ph.D., deputy director of environmental science at Texas A&M University. “It’s like going into the supermarket and instead of buying a steak or a potato, eating the floor tiles.”

Right now ingenuity and insight into using microbes safely to help clean up the oil mess is desperately needed. Sitting around gob-smacked won’t help anything, and environmentalists firmly opposed to the introduction of any organism into the environment will need to suspend disbelief long enough to try desperate measures.

 

The Empty and Misleading Promises of Do-It-Yourself Genetic Tests

"If you could know when and how you would die, would you want to know?" How often have we encountered this question in one form or another, be it between children at the schoolyard or depicted in film and literature? It is a query that resounds within the realm of philosophy: How do we cope with mortality?  Does the certainty of knowing make it better? Or worse? And what if we cannot be given certainty at all? Is it valuable to know how you might die – to engage in an unwinnable contest of crippling probabilities that may or may not offer clues to your fate?

Science can provide no certainties about an individual's medical fate, although some people would like to think otherwise. When the human genome was sequenced, there were definitely some who looked to the nucleotide script as ancient people looked to stars … for answers. We would like to believe that it is that simple – that our DNA sequence can give us the certainty that all of us, at some level of our human consciousness, craves. Am I destined to die of Alzheimer's? Must I ready myself for an arduous battle against cancer? Am I one of the blessed few who will simply drift from this world in my sleep? And while genetic variants have definitely been useful – as with classically Mendelian heritable diseases and biomedical research – by and large, genetic sequence offers little more than "what if's" and "could be's". They may provide an "increased chance" of this or a "decreased chance" of that, but they really don't tell us anything concrete (or useful, for that matter) at all.

That is why I find it particularly worrisome that we are entering the age of affordable, personalized genome sequencing and do-it-yourself genetic tests. While these companies are peddling the promise of knowledge, that is not a commodity that they are actually able to deliver to consumers. Knowing that you have a gene variant that increases your chance for breast cancer, for example, does not mean that you will get breast cancer. Yet, one can easily see how such news could cause undue turmoil among members of the unscientifically-trained public. For what these kits and genome sequencings provide is but a minute glimpse behind the curtain veiling the mechanisms of gene expression. Sure, nucleotides are the "building blocks of life" and all that, but genetic sequence alone will never be capable of providing us with accurate, predictive insight regarding which medical maladies we will acquire.  

For one thing, genetics aren't everything. The "nature versus nurture" argument went by the wayside a long time ago, as it is now recognized that genetic and environmental influences coalesce to determine what occurs within our cells. Secondly, you will recall from your introductory biology classes that DNA sequence is only the first stage of gene expression. One must consider such events as RNA splicing, RNA silencing, and protein modifications (to name a few) if one hopes to attain an accurate assessment of how a gene is expressed. 

Let's also throw in the aspects of cellular context and epigenetic modifications, and you'll soon see that any company that promises to deliver certainty (or even an accurate assessment of probabilities) regarding different medical conditions based solely on DNA sequence is simply preying upon consumers' ignorance. In essence, they are handing consumers a single piece of a 5,000-piece puzzle, telling them that what is displayed on that one fragment is indicative of the pattern of the puzzle as a whole.  

I am not the only one concerned about the less-than-honest promises of at-home genetic tests, as drugstores Walgreens and CVS both canceled their debuts of Pathway Genomics' at-home genetic tests that were scheduled for earlier this month. This was after the Food and Drug Administration started asking for more information from Pathway Genomics about the tests. Just this week Congress got involved, as its House Energy and Commerce Committee launched its own investigation of direct-to-consumer genetic tests. So what does this mean for the future of do-it-yourself genetic testing? Will I ever be able to air-mail a swab of my saliva for my genetic read-out?  Hopefully not. 

The sale of a test that, by its very nature of being direct-to-consumer, can never offer "full disclosure" to the people who buy it should not be permitted. Without extensive disclaimers and interpretation from scientists or medical personnel, consumers don't stand a chance in understanding what the test does and does not tell them about their health. I wouldn't argue that more comprehensive genetic analysis may not be beneficial to people's health in the future, but I think that the only appropriate place for genetic analysis is in a healthcare setting, wherein an informed professional can explain the results to the patient. It is not on a drugstore shelf. And that's one thing about which I am certain.

Award Winning Mayo Clinic Scientist Joins Cardio3 BioSciences

Source:PRNewswire

MONT-SAINT-GUIBERT, BelgiumJuly 14, 2010 /PRNewswire/ –

- Dr. Atta Behfar Recognised With Prestigious Award for Science Behind Cardio3 BioSciences' C-Cure Technology

Cardio3 BioSciences, a leading Belgian biotechnology company specialising in regenerative therapies for the treatment of cardiovascular diseases, today announces that Dr. Atta Behfar is joining its research and development laboratories as Director for Advanced Research. Dr. Atta Behfar, one of the key scientists involved in the development of C-Cure(R), will spend a year at Cardio3 BioSciences on assignment from Mayo Clinic to develop and strengthen the research pipeline.

Dr. Behfar is a member of the clinician investigator program in cardiology at the Mayo Clinic inRochester, Minnesota, USA and has played a key role in the research underlying Cardio3 BioSciences' lead product, C-Cure, a revolutionary stem cell treatment for heart failure. His work was recognised by the Herman K. Gold Young Investigator Award at the Annual American College of Cardiology meeting in March 2010, in GeorgiaAtlanta highlighting the quality of the science behind Cardio3 BioSciences' approach.

C-Cure is designed to reprogram the patient's own stem cells into new heart cells to rebuild the heart. Dr Behfar has played an active part in developing the technology that directs the patient's cells to become cardiopoietic cells – cells 'programmed' to become new heart muscle cells when injected back into the heart of a patient, replacing those cells lost during heart failure and restoring heart function.

Cardio3 BioSciences has recently announced positive three-month safety data and preliminary efficacy results from its Phase II stage clinical trial of C-Cure in heart failure. C-Cure demonstrated a very good safety profile and positive trends in physiological and clinical measures that suggest that C-Cure, as anticipated from animal model data, is acting on heart muscle in a way that could yield important clinical benefits

Dr. Behfar will work with Cardio3 BioSciences' scientific in-house team as of July 1st, 2010, where he will have the opportunity to work directly with the Company as it continues to further develop its unique technology.

Dr. Atta Behfar said: "It is a very exciting time for Cardio3 BioSciences with C-Cure having shown encouraging early results in a clinical setting. I look forward to working with the in-house team as they further advance the product from being a scientific concept towards becoming a therapy that could potentially treat one of the world's greatest unmet medical needs. Many additional treatments may also be leveraged out of the science that lead to C-Cure discovery, and my role will be to fully develop the potential of Cardio3 BioSciences' unique technology."

Dr. Christian Homsy, CEO of Cardio3 BioSciences, added: "We are delighted that Dr Behfar has chosen to come to work with Cardio3 BioSciences and believe this demonstrates not only the quality of the work carried out at Cardio3 BioSciences but also the standard of research inBelgium more generally."

About Cardio3 BioSciences

Cardio3 BioSciences is a leading Belgian biotechnology company specialising in regenerative therapies for the treatment of cardiovascular disease. The Company's lead product, C-Cure(R), is a highly innovative approach to the treatment of heart failure, one of the world's most pressing unmet medical needs. Based on a strategy developed by Cardio3 BioSciences' founders and leveraging technology licensed from Mayo Clinic, C-Cure is designed to reprogram the patient's own stem cells into new heart cells to rebuild the heart.

The Cardio3 BioSciences team has extensive experience in developing and commercialising new pharmaceutical products and medical technologies and the Company's strategy is to drive the clinical development of C-Cure and to market the product itself in major territories.

Cardio3 BioSciences was founded in July 2007 and is based in Mont-Saint-Guibert in the Walloon region of Belgium.

About C-Cure and Heart Failure

Heart failure is a serious and common condition in which the heart cannot pump enough blood through the body, leaving the patient debilitated and unable to conduct a normal life. It can result from heart attacks or a number of other causes. Patients suffering from the condition can experience shortness of breath and extreme exhaustion. It affects 28 million patients worldwide and this number is predicted to double by 2020. Therapies available for chronic heart failure aim at slowing down the disease progression, but with the exception of heart transplant, existing drugs or devices do not cure chronic heart failure.

C-Cure is produced by taking a patient's own stem cells and, through a proprietary process, differentiating them into cardiopoietic cells that can regenerate damaged heart muscle. The cardiopoietic cells are injected into the heart of a patient with heart failure where they are designed to behave identically to those cells lost in heart failure without carrying the risk of rejection, something that has not been achieved with previous cell therapies for this indication. C-Cure is the outcome of multiple years of research conducted at Mayo Clinic (Rochester, Minnesota, USA) and at the Cardiovascular Center in Aalst (Aalst, Belgium).

Disclosures

Mayo Clinic holds equity in Cardio3 BioSciences as a result of intellectual property licensed to the company.

Heart to Heart Tool

We all know the statistics: millions suffer from heart disease, and many suffer heart attacks. Even more alarming is the fact that by some estimates more than 350,000 people die from so-called "sudden death" after a heart attack. Researchers have been baffled by the exact nature and cause of these deaths. Last week, scientists at the University of North Carolina Chapel Hill unveiled a promising new modeling procedure which may shed some light on the mechanisms associated with heart attacks and sudden death.

This new system involved cultured heart cells. In a heart attack, some cells become deprived of oxygen while others continue to receive sufficient oxygen. The region between these two types of cells, called the border zone, is simulated in the cultured model. Since the culture focuses on the interactions in the border zone, it could be very helpful in studying arrhythmias. Arrhythmias are irregularities in the heartbeat usually associated with decreased blood flow in the coronary arteries.

The complex interactions within a particular organism and the size of the actual zone have made animal models particularly difficult in studying the border zone. Likewise, single cells don't display the border irregularities. By using a culture of cells, the research team was able to overcome both limitations. The border zone can be produced and the interactions are essentially isolated for practical purposes.

The cultures appear to be stable for a couple of hours, thus allowing the team to do basic time-based studies.

Since the causes and mechanisms associated with sudden death are so poorly understood, researchers are optimistic that this new modeling system will shed some light on sudden death. By focusing on the internal changes associated with cells and the resulting interactions, the team believes that the culture model will be successful.

What do you think? Might this new technique be helpful in reducing the instances of sudden death? What other therapies and/or procedures might be used to produce a synergistic effect? Come over to the Biology Forum and share your thoughts, opinions, and feelings.

Mylotarg Withdrawal Raises Questions

Pfizer agreed today to withdraw its therapy for acute myeloid leukemia, Mylotarg (gemtuzumab ozogamicin), from the U.S. market, effective October 15. The reason? It didn’t work, and people died. 

So far, the story makes sense. Developed by Wyeth, the drug was fast-tracked to treat patients ages 60 and older with recurrent AML who were not candidates for other chemotherapy. It was approved by the FDA in May 2000 based upon a surrogate endpoint, because it treated a serious disease that had no other viable therapy. 

Four years later, a confirmatory trial was begun to confirm the results of the 142 patients who participated in the three previous clinical trials. Given the circumstances, the failure and subsequent withdrawal makes sense. 

What doesn’t make sense is what comes next. Wyeth stopped the 2004 trial early because patients experienced no clinical benefit and died in greater numbers than those receiving chemotherapy alone. 

So, why did it take so long to withdraw the drug?  Why is Mylotarg being withdrawn only for the U.S. market? Why does the withdrawal become effective October 15, three and one half months from now? 

Why are patients who are on Mylotarg allowed to continue a therapy that, according to the FDA, “demonstrates no clinical benefit?”