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Category Archives: Stem Cells

Stem Cell Transplant Program Offered at UVA Medical Center

Posted: March 23, 2012 at 2:46 am

What used to be medical trash is now treating cancer. The University of Virginia's Medical Center is the first place in Virginia to take advantage of stem cells from umbilical cords and they are pleased with the results.

Dr. Mary Laughlin, the director of stem cell transplantation at UVA,said, "These are cells that are routinely thrown away, these cells save lives."

A lab within the UVA Medical Center contains numerous tubes where non-embryonic stem cells reside. They come from umbilical cord blood and give hope topatients suffering leukemia, multiple myeloma and lymphoma.

Dr. Laughlin added, "They can completely replace a patient's bone marrow in the immune system. Oneof 10 cancer patients are able to find those cells through existing adult registries."

Thefive million babies that are born each year will soon solve that problem. The cells that are normally tossed out attack cancer cells.

Denise Mariconda, a nurse within the stem cell transplant program, stated, "It looks like a blood transfusion." Dr. Laughlin added, "It is in many ways like a cancer vaccine."

The first transplants were made in January and the transplant program at the UVA Medical Center admits it takes getting used to.

Mariconda said, "It is a process that's not like having your heart fixed in a one-day setting and you know that it's better."

These cells are not cause for controversy. Dr. Laughlin said, "Use of cord-blood is approved by all religious groups including the Vatican."

Babies' immune systems are not fully educated at the time of birth, making these cells effective. Dr. Laughlin, added, "That allows us to cross transplant barriers."

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Somatic stem cells obtained from skin cells; pluripotency ‘detour’ skipped

Posted: March 23, 2012 at 2:46 am

ScienceDaily (Mar. 22, 2012) Breaking new ground, scientists at the Max Planck Institute for Molecular Biomedicine in Mnster, Germany, have succeeded in obtaining somatic stem cells from fully differentiated somatic cells. Stem cell researcher Hans Schler and his team took skin cells from mice and, using a unique combination of growth factors while ensuring appropriate culturing conditions, have managed to induce the cells' differentiation into neuronal somatic stem cells.

"Our research shows that reprogramming somatic cells does not require passing through a pluripotent stage," explains Schler. "Thanks to this new approach, tissue regeneration is becoming a more streamlined -- and safer -- process."

Up until now, pluripotent stem cells were considered the 'be-all and end-all' of stem cell science. Historically, researchers have obtained these 'jack-of-all-trades' cells from fully differentiated somatic cells. Given the proper environmental cues, pluripotent stem cells are capable of differentiating into every type of cell in the body, but their pluripotency also holds certain disadvantages, which preclude their widespread application in medicine. According to Schler, "pluripotent stem cells exhibit such a high degree of plasticity that under the wrong circumstances they may form tumours instead of regenerating a tissue or an organ." Schler's somatic stem cells offer a way out of this dilemma: they are 'only' multipotent, which means that they cannot give rise to all cell types but merely to a select subset of them -- in this case, a type of cell found in neural tissue -- a property, which affords them an edge in terms of their therapeutic potential.

To allow them to interconvert somatic cells into somatic stem cells, the Max Planck researchers cleverly combined a number of different growth factors, proteins that guide cellular growth. "One factor in particular, called Brn4, which had never been used before in this type of research, turned out to be a genuine 'captain' who very quickly and efficiently took command of his ship -- the skin cell -- guiding it in the right direction so that it could be converted into a neuronal somatic stem cell," explains Schler. This interconversion turns out to be even more effective if the cells, stimulated by growth factors and exposed to just the right environmental conditions, divide more frequently. "Gradually, the cells lose their molecular memory that they were once skin cells," explains Schler. It seems that even after only a few cycles of cell division the newly produced neuronal somatic stem cells are practically indistinguishable from stem cells normally found in the tissue.

Schler's findings suggest that these cells hold great long-term medical potential: "The fact that these cells are multipotent dramatically reduces the risk of neoplasm formation, which means that in the not-too-distant future they could be used to regenerate tissues damaged or destroyed by disease or old age; until we get to that point, substantial research efforts will have to be made." So far, insights are based on experiments using murine skin cells; the next steps now are to perform the same experiments using actual human cells. In addition, it is imperative that the stem cells' long-term behaviour is thoroughly characterized to determine whether they retain their stability over long periods of time.

"Our discoveries are a testament to the unparalleled degree of rigor of research conducted here at the Mnster Institute," says Schler. "We should realize that this is our chance to be instrumental in helping shape the future of medicine." At this point, the project is still in its initial, basic science stage although "through systematic, continued development in close collaboration with the pharmaceutical industry, the transition from the basic to the applied sciences could be hugely successful, for this as well as for other, related, future projects," emphasizes Schler. This, then, is the reason why a suitable infrastructure framework must be created now rather than later. "The blueprints for this framework are all prepped and ready to go -- all we need now are for the right political measures to be ratified to pave the way towards medical applicability."

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The above story is reprinted from materials provided by Max-Planck-Gesellschaft.

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Somatic stem cells obtained from skin cells; pluripotency 'detour' skipped

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First Stem Cell Transplants in Virginia Performed at UVA

Posted: March 22, 2012 at 3:07 pm

The Stem Cell Transplant Program at the University of Virginia Health System recently performed the first two stem cell transplants in Virginia, using non-embryonic stem cells from umbilical cord blood.

The program offers both bone marrow and stem cell transplants, with a focus on cord blood, to treat leukemia, lymphoma, Hodgkins disease and other blood diseases.

While it will take several months to know how effective the cord blood transplants were, the initial results are promising, says Mary Laughlin, MD, an internationally known stem cell expert recruited to UVA to head the program. In both patients, the stem cells began engrafting producing new cells 14 days after the transplant instead of the 24 to 28 days it normally takes.

Why cord blood stem cells? As an obstetrician once told Laughlin: Something thrown away in my OB suite saves a life in your cancer suite.

The cord blood used for these stem cell transplants comes from placentas that otherwise would be discarded following childbirth, Laughlin says. The cord blood is used with the permission of the new parents, she says. By using cord blood stem cells instead of embryonic stem cells, UVAs program sidesteps the ethical, religious and political concerns commonly associated with stem cells, she says.

Other benefits: Cord blood stem cells are also faster and easier to collect than stem cells from other sources; they are also immune tolerant.

Speed is important because there is a narrow window of opportunity to perform a transplant when a patients disease is in remission. And because the cord blood stem cells are immune tolerant meaning they will not attack other cells in the body the chances of a successful transplant are higher and the donor match doesnt have to be as exact, giving more patients the opportunity to receive a transplant.

Stem cell transplants: Part of a fast-growing program Laughlin heads up a team of 29 staff members, including four additional transplant physicians, who began seeing patients in September. The demand for transplants has already been greater than Laughlin and her team expected. The program had initially planned to do 15 transplants in its first year. Instead, it expects to do 100.

Its reflective of this unmet need, Laughlin says. Patients who otherwise would have to travel many states away to have these same procedures, now they can do a fairly short drive from Roanoke, or down from Winchester. Because of our central location, its ideal for them.

What are stem cells? Learn more about how they work.

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First Stem Cell Transplants in Virginia Performed at UVA

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Procognia Announced That the Feasibility Stage of the Company’s Research in Stem Cells in Cooperation with the …

Posted: March 22, 2012 at 3:07 pm

TEL-AVIV, Israel--(BUSINESS WIRE)--

Procognia (TASE:PRCG) is pleased to announce that the feasibility stage of the research, which focused on the glycosylation structures of stem cells, has been successfully completed. The research, directed by Prof. Dov Zipori from the Weizmann Institute of Science, was designed to develop a platform that will significantly improve the ability to identify and develop unique stem cells for transplant and treatment. Today, stem cell treatment faces a number of challenges, which both parties aim to handle successfully and therefore make a significant contribution to this field.

The feasibility stage focused on mesenchymal stem cells, and the results displayed the ability to:

Each of the above factors has the potential to significantly improve the abilities of the medical and scientific communities to successfully use stem cells for effective, successful transplants and medical treatment. In cooperation with Yeda Research & Development, the commercial arm of the Weizmann Institute, Procognia has submitted a US patent that covers the research and its unique findings.

According to the results of the feasibility stage, Procognia will continue its research with Prof. Zipori to develop a platform that will:

Mesenchymal stem cells migrate towards tumors and affect them, and therefore can also be used as carriers for drugs that will affect the tumors.

Within the framework of the agreement, Procognia has the option to commercialize products for diagnostic and therapeutic uses on the basis of the joint development process, in exchange for Yeda Research & Development receiving appropriate royalties.

Procognia estimates that the development stage of this platform will take approximately two years.

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SA cracks stem cell conundrum

Posted: March 22, 2012 at 3:07 pm

Scientists in SA have generated non-embryonic stem cells for the first time, the Council for Scientific and Industrial Research (CSIR) announced on Tuesday.

These "induced adult pluripotent stem cells" were developed from adult skin cells and can be prompted to grow into any type of adult cell, such as those in the heart or brain.

The technology is important for research into regenerative medicine, but is not yet widely used.

While the technology is not novel, the development of the capacity to grow these stem cells in SA is important for researchers investigating diseases affecting Africans, said CSIR post-doctoral fellow Janine Scholefield. The CSIR had replicated techniques devised by Japanese researchers in 2007.

"Cutting-edge medical research is not useful to Africans if knowledge is being created and applied only in the developed world," said CSIR head of gene expression and biophysics Musa Mhlanga. "Given the high disease burden in Africa, our aim is to become creators of knowledge, as well as innovators and expert practitioners of the newest and best technologies," The CSIR said that adult-generated stem cells were more acceptable to people who objected to using stem cells from embryos.

"The other critical thing is the cells (that will be grown) are an exact genetic match to the person who donated the skin cells, so we can circumvent the problem of tissue rejection," Dr Scholefield said.

"We can also develop models of disease in a petri dish in the laboratory," she said, explaining that this would enable researchers to investigate rare diseases without the need for human subjects.

"We are getting closer to using stem cells as part of routine medical practice, but are still a long way off from using these cells for degenerative diseases of the central nervous system," said Michael Pepper, professor of i mmunology at the University of Pretoria.

Prof Pepper said there were several hundred clinical trials using stem cells under way around the world, but most were still at an early stage.

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Researcher Alert: California Stem Cell Agency To Alter How It Administers Grants

Posted: March 18, 2012 at 3:57 pm


Stem cell researchers and institutions throughout the state are likely to be affected by proposed changes – to be discussed online publicly Tuesday – dealing with how the California stem cell agency will handle its $3 billion in grants.

An important online session – open to all interested parties – comes up then, but advance registration is required.

The proposals are wide-ranging and detailed. The nearly 500 recipients of CIRM grants should examine them closely in addition to any persons seriously interested in California stem cell affairs. The changes deal with such subjects as milestones for research grants, indirect costs, travel costs, withholding payments for failure to file a progress report and much, much more.

Here is a link to the main page for all this, which has instructions on how to register for the online session along with links to the changes and their rationale.

(Editor's note: This item was filed from the Rio Sabana in the Darien in Panama when we found a weak Internet cellular link. We are still underway so postings are unlikely between now and later this month.)

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Researcher Alert: California Stem Cell Agency To Alter How It Administers Grants

Posted: March 18, 2012 at 3:56 pm


Stem cell researchers and institutions throughout the state are likely to be affected by proposed changes – to be discussed online publicly Tuesday – dealing with how the California stem cell agency will handle its $3 billion in grants.

An important online session – open to all interested parties – comes up then, but advance registration is required.

The proposals are wide-ranging and detailed. The nearly 500 recipients of CIRM grants should examine them closely in addition to any persons seriously interested in California stem cell affairs. The changes deal with such subjects as milestones for research grants, indirect costs, travel costs, withholding payments for failure to file a progress report and much, much more.

Here is a link to the main page for all this, which has instructions on how to register for the online session along with links to the changes and their rationale.

(Editor's note: This item was filed from the Rio Sabana in the Darien in Panama when we found a weak Internet cellular link. We are still underway so postings are unlikely between now and later this month.)

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http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss

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‘Forged’ brain cells offers hope for Huntington’s disease treatment

Posted: March 16, 2012 at 8:59 pm

Washington, Mar 16 (ANI): A special type of brain cell forged from stem cells could help restore the muscle coordination deficits that cause the uncontrollable spasms characteristic of Huntington's disease, a new study has suggested.

Huntington's disease, the debilitating congenital neurological disorder that progressively robs patients of muscle coordination and cognitive ability, is a condition without effective treatment, a slow death sentence.

"This is really something unexpected," said Su-Chun Zhang, a University of Wisconsin-Madison neuroscientist and the senior author of the new study, which showed that locomotion could be restored in mice with a Huntington's-like condition.

Zhang is an expert at making different types of brain cells from human embryonic or induced pluripotent stem cells.

In the new study, his group focused on what are known as GABA neurons, cells whose degradation is responsible for disruption of a key neural circuit and loss of motor function in Huntington's patients.

GABA neurons, Zhang explained, produce a key neurotransmitter, a chemical that helps underpin the communication network in the brain that coordinates movement.

In the laboratory, Zhang and his colleagues at the UW-Madison Waisman Center have learned how to make large amounts of GABA neurons from human embryonic stem cells, which they sought to test in a mouse model of Huntington's disease.

The goal of the study, Zhang noted, was simply to see if the cells would safely integrate into the mouse brain.

To their astonishment, the cells not only integrated but also project to the right target and effectively re-established the broken communication network, restoring motor function.

The results of the study were surprising, Zhang explained, because GABA neurons reside in one part of the brain, the basal ganglia, which plays a key role in voluntary motor coordination.

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Stem cells hint at potential treatment for Huntington’s Disease

Posted: March 15, 2012 at 10:08 pm

Public release date: 15-Mar-2012 [ | E-mail | Share ]

Contact: Su-Chun Zhang zhang@waisman.wisc.edu 608-265-2543 University of Wisconsin-Madison

MADISON -- Huntington's disease, the debilitating congenital neurological disorder that progressively robs patients of muscle coordination and cognitive ability, is a condition without effective treatment, a slow death sentence.

But if researchers can build on new research reported this week (March 15, 2012) in the journal Cell Stem Cell, a special type of brain cell forged from stem cells could help restore the muscle coordination deficits that cause the uncontrollable spasms characteristic of the disease.

"This is really something unexpected," says Su-Chun Zhang, a University of Wisconsin-Madison neuroscientist and the senior author of the new study, which showed that locomotion could be restored in mice with a Huntington's-like condition.

Zhang is an expert at making different types of brain cells from human embryonic or induced pluripotent stem cells. In the new study, his group focused on what are known as GABA neurons, cells whose degradation is responsible for disruption of a key neural circuit and loss of motor function in Huntington's patients. GABA neurons, Zhang explains, produce a key neurotransmitter, a chemical that helps underpin the communication network in the brain that coordinates movement.

In the laboratory, Zhang and his colleagues at the UW-Madison Waisman Center have learned how to make large amounts of GABA neurons from human embryonic stem cells, which they sought to test in a mouse model of Huntington's disease. The goal of the study, Zhang notes, was simply to see if the cells would safely integrate into the mouse brain. To their astonishment, the cells not only integrated but also project to the right target and effectively reestablished the broken communication network, restoring motor function.

The results of the study were surprising, Zhang explains, because GABA neurons reside in one part of the brain, the basal ganglia, which plays a key role in voluntary motor coordination. But the GABA neurons exert their influence at a distance on cells in the midbrain through the circuit fueled by the GABA neuron chemical neurotransmitter.

"This circuitry is essential for motor coordination," Zhang says, "and it is what is broken in Huntington patients. The GABA neurons exert their influence at a distance through this circuit. Their cell targets are far away."

That the transplanted cells could effectively reestablish the circuit was completely unexpected: "Many in the field feel that successful cell transplants would be impossible because it would require rebuilding the circuitry. But what we've shown is that the GABA neurons can remake the circuitry and produce the right neurotransmitter."

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Verastem, Inc. Reports Year-End 2011 Financial Results

Posted: March 14, 2012 at 10:04 pm

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

Verastem, Inc., (NASDAQ: VSTM - News) a biopharmaceutical company focused on discovering and developing drugs to treat breast and other cancers by targeting cancer stem cells, today reported financial results for the year ended December 31, 2011 and also commented on certain corporate accomplishments and plans.

"We achieved important milestones in the discovery and development of drugs targeting cancer stem cells last year and anticipate further progress this year," said Christoph Westphal, M.D., Ph.D., Chairman, President and Chief Executive Officer of Verastem, Inc. "In particular, we acquired our focal adhesion kinase (FAK) program and are progressing two lead candidates, VS-4718 and VS-5095 in parallel through preclinical studies. In our Wnt/Beta-catenin program, VS-507 is currently in IND-enabling toxicology studies. We expect to initiate clinical trials with VS-507 and one of either VS-4718 or VS-5095 over the next 12-15 months.

We anticipate that the capital raised through our recently completed initial public offering will provide sufficient funds for operations into 2016, said Robert Forrester, Chief Operating Officer of Verastem, Inc. We believe that the IPO gives us the ability to maintain rights to our compounds through Phase 2 and provides the capital we need to do the right science and the right clinical trials without a financing overhang.

Recent Accomplishments

Our significant recent accomplishments include the following:

Full Year 2011 Financial Results

As of December 31, 2011, Verastem had cash, cash equivalents, short-term investments and long-term investments of $56.8 million compared to $3.6 million on December 31, 2010. In February 2012, we received net proceeds from our IPO of approximately $56.7 million.

Net loss for the year ended December 31, 2011 was $13.7 million, or $10.59 per share applicable to common shareholders, as compared to $784,000, or $0.91 per share, for the period from August 4, 2010 (inception) to December 31, 2010 (2010 Period). Net loss includes stock-based compensation expense of $1.6 million and $52,000 for the year ended December 31, 2011 and the 2010 Period, respectively.

For the year ended December 31, 2011, research and development expense was $9.9 million compared to $400,000 for the 2010 Period. The major components of research and development expense in 2011 were: contract research organizations ($3.7 million), payroll expense ($1.5 million), consulting fees ($1.3 million), laboratory supplies ($1.0 million), non-employee stock-based compensation ($1.0 million) and license fees ($842,000).

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Verastem, Inc. Reports Year-End 2011 Financial Results

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