Category Archives: Stem Cell Treatments

Shinya Yamanaka MD, PhD

What are induced pluripotent stem cells?

Induced pluripotent stem cells, or iPS cells, are a type of cell that has been reprogrammed from an adult cell, such as a skin or blood cell. iPS cells are pluripotent cells because, like embryonic stem cells, they can develop into virtually any type of cell. iPS cells are distinct from embryonic stem cells, however, because they are derived from adult tissue, rather than from embryos. iPS cells are also distinct from adult stem cells, which naturally occur in small numbers in thehuman body.

In 2006, Shinya Yamanaka developed the method for inducing skin cells from mice into becoming like pluripotent stem cells and called them iPS cells. In 2007, Yamanaka did the same with adult human skin cells.

Yamanakas experiments revealed that adult skin cells, when treated with four pieces of DNA (now called the Yamanaka factors), can induce skin cells to revert back to their pluripotent state. His discovery has since led to a variety of methods for reprogramming adult cells into stem cells that can become virtually any cell type such as a beating heart cell or a neuron that can transmit chemical signals in the brain. This allows researchers to create patient-specific celllines that can be studied and used in everything from drug therapies to regenerative medicine.

How are iPS cells different from embryonic stem cells?

iPS cells are a promising alternative to embryonic stem cells. Embryonic stem cells hold tremendous potential for regenerative medicine, in which damaged organs and tissues could be replaced or repaired. But the use of embryonic stem cells has long been controversial. iPS cells hold the same sort of promise but avoid controversy because they do not require the destruction of human embryos. Nor do they require the harvesting of adult stem cells. Rather, they simply require a small tissue sample from a living human.

Why is iPS cell technology so important?

In addition to avoiding the controversial use of embryonic stem cells, iPS cell technology also represents an entirely new platform for fundamental studies of human disease. Rather than using models made in yeast, flies or mice for disease research, iPS cell technology allows human stem cells to be created from patients with a specific disease. As a result, the iPS cells contain a complete set of the genes that resulted in that disease and thus represent the potential of a farsuperior human model for studying disease and testing new drugs and treatments. In the future, iPS cells could be used in a Petri dish to test both drug safety andefficacy for an individual patient.

What has happened since Shinya Yamanaka developed iPS technology?

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Stem Cell Science Q & A

A British researcher and a Japanese scientist won the Nobel Prize in physiology or medicine on Monday for discovering that ordinary cells of the body can be reprogrammed into stem cells, which then can turn into any kind of tissue -- a discovery that may led to new treatments.

Scientists want to build on the work by John Gurdon and Shinya Yamanaka to create replacement tissues for treating diseases like Parkinson's and diabetes, and for studying the roots of diseases in the laboratory -- without the ethical dilemma posed by embryonic stem cells.

In announcing the 8 million kronor ($1.2 million) award, the Nobel committee at Stockholm's Karolinska Institute said the discovery has "revolutionized our understanding of how cells and organisms develop."

Gurdon showed in 1962 -- the year Yamanaka was born -- that the DNA from specialized cells of frogs, like skin or intestinal cells, could be used to generate new tadpoles. That showed the DNA still had its ability to drive the formation of all cells of the body.

At the time, the discovery had "no obvious therapeutic benefit at all," Gurdon told reporters in London.

"It was almost 50 years before the value -- the potential value -- of that basic scientific research comes to light," he said.

In 1997, the cloning of Dolly the sheep by other scientists showed that the same process Gurdon discovered in frogs would work in mammals.

More than 40 years after Gurdon's discovery, in 2006, Yamanaka showed that a surprisingly simple recipe could turn mature cells back into primitive cells, which in turn could be prodded into different kinds of mature cells.

Basically, the primitive cells were the equivalent of embryonic stem cells, which had been embroiled in controversy because to get human embryonic cells, human embryos had to be destroyed. Yamanaka's method provided a way to get such primitive cells without destroying embryos.

"The discoveries of Gurdon and Yamanaka have shown that specialized cells can turn back the developmental clock under certain circumstances," the committee said. "These discoveries have also provided new tools for scientists around the world and led to remarkable progress in many areas of medicine."

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Stem cell researchers awarded Nobel Prize for medicine

The Associated Press Published Monday, Oct. 8, 2012 7:18AM EDT Last Updated Monday, Oct. 8, 2012 7:49AM EDT

STOCKHOLM, Sweden -- British researcher John Gurdon and Shinya Yamanaka of Japan won this year's Nobel Prize in physiology or medicine on Monday for discovering that mature, specialized cells of the body can be reprogrammed into stem cells -- a discovery that scientists hope to turn into new treatments.

Scientists want to harness that reprogramming to create replacement tissues for treating diseases like Parkinson's, diabetes and for studying the roots of diseases in the laboratory.

The prize committee at Stockholm's Karolinska Institute said the discovery has "revolutionized our understanding of how cells and organisms develop."

Gurdon showed in 1962 -- the year Yamanaka was born -- that the DNA from specialized cells of frogs, like skin or intestinal cells, could be used to generate new tadpoles. That showed the DNA still had its ability to drive the formation of all cells of the body.

In 1997, the cloning of Dolly the sheep by other scientists showed that the same process Gurdon discovered in frogs would work in mammals.

More than 40 years after Gurdon's discovery, in 2006, Yamanaka showed that a surprisingly simple recipe could turn mature cells back into primitive cells, which in turn could be prodded into different kinds of mature cells.

Basically, the primitive cells were the equivalent of embryonic stem cells, which had been embroiled in controversy because to get human embryonic cells, human embryos had to be destroyed. Yamanaka's method provided a way to get such primitive cells without destroying embryos.

"The discoveries of Gurdon and Yamanaka have shown that specialized cells can turn back the developmental clock under certain circumstances," the committee said. "These discoveries have also provided new tools for scientists around the world and led to remarkable progress in many areas of medicine."

Just last week, Japanese scientists reported using Yamanaka's approach to turn skin cells from mice into eggs that produced baby mice.

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Stem cell researchers share Nobel medicine prize

Japanese researchers have demonstrated in mice that eggs and sperm can be grown from stem cells and combined to produce healthy offspring, a finding that could lead to new treatments for infertility.

If the achievement can be repeated in humans, the technique could make it easier for women in their 30s or 40s to bear children. It could also help men and women whose reproductive organs have been damaged by cancer treatments or other causes.

About one in 10 American women of childbearing age have trouble becoming or staying pregnant, and more than one-third of infertile couples must contend with a medical problem related to the prospective father, according to the national Centers for Disease Control and Prevention in Atlanta.

Using current technology, only about one-third of attempts at assisted reproduction result in live births, CDC data show. Scientists, doctors and patients would like to boost that percentage.

"These studies provide that next level of evidence that in the future fertility could be managed with stem cell intervention," said Teresa Woodruff, chief of fertility preservation at Northwestern University Feinberg School of Medicine.

The prospect of using stem cells to grow new eggs is particularly tantalizing, since women are born with a set number and don't make more once they are gone.

In a sense, the therapy would allow them to turn back their biological clocks, said Stanford researcher Renee Reijo Pera, who studies reproduction.

"This is

Dr. Mitinori Saitou and colleagues at Kyoto University detailed how they generated the functional mouse eggs in a report published online Thursday, Oct. 4, by the journal Science. Last year, the researchers reported in the journal Cell that they had done the same thing with mouse sperm.

In both cases, the team started with embryonic stem cells, which have the potential to develop into all of the different types of cells in the body.

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Mouse eggs grown from stem cells

In a long-sought achievement, Japanese researchers have demonstrated in mice that both eggs and sperm can be grown from stem cells and combined to produce healthy offspring, pointing the way to a new avenue for fertility treatments.

If the milestone accomplishment can be repeated in humans -- and experts said they are optimistic that such efforts will ultimately succeed -- the technique could make it easier for women in their 30s or 40s to become mothers. It could also help men and women whose reproductive organs have been damaged by cancer treatments or other causes.

About 10% of American women of childbearing age have trouble becoming or staying pregnant, and more than one-third of infertile couples must contend with a medical problem related to the prospective father, according to the U.S. Centers for Disease Control and Prevention in Atlanta.

Using current technology, only about one-third of attempts at assisted reproduction result in live births, CDC data show. Scientists, doctors and patients would like to boost that percentage.

"These studies provide that next level of evidence that in the future fertility could be managed with stem cell intervention," said Teresa Woodruff, chief of fertility preservation at Northwestern University's Feinberg School of Medicine.

The prospect of using stem cells to grow new eggs is particularly tantalizing, because women are born with a set amount and don't make more once they are lost. In a sense, the therapy would allow them to turn back their biological clocks, said Stanford stem cell researcher Renee A. Reijo Pera, who studies reproduction.

"This is a get-them-back strategy," she said.

Using stem cells to create sperm and eggs in mice is a feat researchers have attempted, without much success, for more than a decade, said Dr. George Q. Daley, a leading stem cell researcher at Children's Hospital in Boston.

Dr. Mitinori Saitou and colleagues at Kyoto University detailed how they generated the functional mouse eggs in a report published online Thursday by the journal Science. Last year, the researchers reported in the journal Cell that they had done the same thing with mouse sperm.

In both cases, the team started with embryonic stem cells, which have the potential to develop into all of the different types of cells in the body. The scientists exposed the embryonic stem cells to stimuli that coaxed them to become egg and sperm precursors.

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Stem cells could lead to future fertility treatments, study says

Reaching a long-sought milestone, Japanese researchers have demonstrated in mice that eggs and sperm can be grown from stem cells and combined to produce healthy offspring, pointing to new treatments for infertility.

If the achievement can be repeated in humans and experts said they are optimistic that such efforts will ultimately succeed the technique could make it easier for women in their 30s or 40s to become mothers. It could also help men and women whose reproductive organs have been damaged by cancer treatments or other causes.

About one in 10 American women of childbearing age have trouble becoming or staying pregnant, and more than one-third of infertile couples must contend with a medical problem related to the prospective father, according to the national Centers for Disease Control and Prevention in Atlanta.

Using current technology, only about one-third of attempts at assisted reproduction result in live births, CDC data show. Scientists, doctors and patients would like to boost that percentage.

"These studies provide that next level of evidence that in the future fertility could be managed with stem cell intervention," said Teresa Woodruff, chief of fertility preservation at Northwestern University Feinberg School of Medicine.

The prospect of using stem cells to grow new eggs is particularly tantalizing, since women are born with a set number and don't make more once they are gone. In a sense, the therapy would allow them to turn back their biological clocks, said Stanford stem cell researcher Renee A. Reijo Pera, who studies reproduction.

"This is a get-them-back strategy," she said.

Dr. Mitinori Saitou and colleagues at Kyoto University detailed how they generated the functional mouse eggs in a report published online Thursday by the journal Science. Last year, the researchers reported in the journal Cell that they had done the same thing with mouse sperm.

In both cases, the team started with embryonic stem cells, which have the potential to develop into all of the different types of cells in the body.

The scientists exposed the embryonic stem cells to stimuli that coaxed them to become egg and sperm precursors.

Read more here:
Mouse stem cells used to produce eggs, Japanese scientists say

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

Verastem, Inc., (VSTM) a clinical-stage biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, announced that members of the management team will present at the BIO Investor Forum on October 9, 2012, at the Palace Hotel in San Francisco, CA.

The company presentation is at 8:30 am PT in the Presidio room and Jonathan Pachter, Ph.D., Verastem Vice President and Head of Research, will participate on a cancer stem cell panel from 10:30-11:25 am PT in the Twin Peaks room.

The panel will bring together scientific experts who are at the forefront of developing novel medicines targeting cancer stem cells. Dr. Pachter will discuss the potential of this approach to create durable clinical responses for patients with cancers that are typically refractory to conventional treatments. Verastem is translating basic science into clinical products based on the seminal publications in Cell and PNAS by Robert Weinberg, Ph.D., Verastem co-founder and chair of the Scientific Advisory Board.

A webcast of the company presentation can be accessed by visiting the investors section of the Companys website at http://www.verastem.com. A replay of the webcast will be archived on the Verastem website for two weeks following the presentation date.

About Verastem, Inc.

Verastem, Inc. (VSTM) is a clinical-stage biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells. Cancer stem cells are an underlying cause of tumor recurrence and metastasis. Verastem is developing small molecule inhibitors of signaling pathways that are critical to cancer stem cell survival and proliferation: focal adhesion kinase (FAK), PI3K/mTOR and Wnt. For more information, please visit http://www.verastem.com.

Forward-looking statements:

Any statements in this press release about future expectations, plans and prospects for the Company constitute forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

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Verastem to Present at BIO Investor Forum and Participate in Cancer Stem Cell Panel

Public release date: 3-Oct-2012 [ | E-mail | Share ]

Contact: Garth Sundem garth.sundem@ucdenver.edu University of Colorado Denver

The University of Colorado Cancer Center, together with other participating academic medical centers, recently opened a phase I human clinical trial of the drug OMP-54F28 in patients with advanced solid tumor cancers. OMP-54F28, a candidate investigational drug discovered by OncoMed Pharmaceuticals, targets cancer stem cells (CSCs), also known as tumor-initiating cells, which many researchers believe are at the root of tumor occurrence and growth. These CSCs are notoriously resistant to existing chemotherapies and so may survive current treatments to repopulate a tumor, leading to relapse and metastasis.

"It's a terrific opportunity to put a drug targeting cancer stem cells in the clinic, especially a drug with as much promise in preclinical studies as this one," says Antonio Jimeno, MD, PhD, investigator at the CU Cancer Center, director of the university's Cancer Stem Cell-Directed Clinical Trials Program, and principal investigator of the clinical trial at the CU Cancer Center site. "It is a privilege to work with such a science-focused partner, whose vision totally aligns with ours: bringing to the clinic cutting-edge drugs and ideas that are supported by robust scientific data. In the context of the collaboration between the Gates Center for Stem Cell Biology and the CU Cancer Center this will be the second clinical trial we will be offering to our patients with the specific intent to target the CSCs in their tumors."

Specifically, OMP-54F28 is an antagonist of the Wnt pathway, a key CSC signaling pathway that regulates the fate of these cells. The Wnt pathway has been intensively studied and is now known to be inappropriately activated in many major tumor types, including colon, breast, liver, lung and pancreatic cancers, and is thought to be critical for the function of CSCs. Because of this extensive preclinical validation, the Wnt pathway has been a major focus of anti-cancer drug discovery efforts. OMP-54F28 and a sister compound also developed by OncoMed, OMP-18R5, are believed to be two of the first therapeutic agents targeting this key pathway to enter clinical testing. Both OMP-54F28 and OMP-18R5 are part of OncoMed's Wnt pathway strategic alliance with Bayer Pharma AG.

In multiple preclinical models, OMP-54F28 has shown its effectiveness in reducing CSC populations, leading to associated anti-tumor activity, either as a single agent or when combined with chemotherapy.

The Phase I clinical trial of OMP-54F28 is an open-label dose escalation study in patients with advanced solid tumors for which there is no remaining standard curative therapy. These patients are assessed for safety, immunogenicity, pharmacokinetics, biomarkers, and initial signals of efficacy. The trial is being conducted at Pinnacle Oncology Hematology in Scottsdale, Arizona, the University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan, and the CU Cancer Center under the direction of Principal Investigators Dr. Michael S. Gordon, Dr. David Smith and Dr. Antonio Jimeno, respectively.

"We all hope and expect this drug to live up to its preclinical potential," Jimeno says. "And if it does, we will have a powerful new therapy, exploiting a novel pathway to target this most dangerous subpopulation of cancer cells."

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About OncoMed Pharmaceuticals

Continued here:
CU Cancer Center opens phase i clinical trial of anti-cancer stem cell agent OMP-54F28

OCEANSIDE, Calif., Oct. 1, 2012 /PRNewswire/ --Health Link Medical Center, a national leader in regenerative interventional orthopedics and advanced biological cell therapies, today announced the planned opening of their new location in Mill Valley, California in October 2012. Health Link Medical Center's Oceanside, California clinic is currently California's only provider of Regenexx orthopedic platelet and autologous stem cell procedures.

Regenexx Procedures offer non-surgical treatment options for common joint injuries and degenerative conditions, such as osteoarthritis. The procedures utilize a patient's own stem cells to help heal damaged tissues, tendons, ligaments, bone, or cartilage. Regenexx patients experience less downtime and avoid the lengthy and painful rehabilitation periods that follow surgery.

"We're excited for the opportunity to bring Regenexx Procedures to the San Francisco area," said Dr. Norman Deitch, CEO of Health Link Medical Center. "Patients regularly travel across the country for the opportunity to receive these leading non-surgical treatments. This expansion makes them conveniently accessible to the millions of individuals in northern California."

The Mill Valley Center will include a biological cell laboratory, capable of the advanced laboratory processing of platelets and stem cells required for the same-day procedures. Paul Handleman, D.O., has joined Health Link with an extensive interventional orthopedic background and has been in practice in Marin County, California for more than 15 years. Dr. Handleman has undergone advanced training at the Regenexx / Centeno-Schultz home clinic in Broomfield, Colorado.

Regenexx Procedures are currently performed at Health Link's Oceanside, CA. location. The Mill Valley Center opening is slated for October 2012 and Health Link is already scheduling patients for the new location. For more information, visit http://www.healthlinkcenter.com or call 800-281-3757.

About Health Link Medical Center

Based in Oceanside, California, Health Link Medical Center is a leader in regenerative interventional orthopedics and advanced biological cell therapies. Health Link is California's first provider of Regenexx Stem Cell and Blood Platelet Procedures. Learn more at http://www.healthlinkcenter.com.

About Regenexx and the Regenexx Physician Network

Regenexx Procedures offer non-surgical treatments for joint injuries and degenerative conditions. For more information on Regenexx Procedures and the Regenexx Physician Network, visit: http://www.regenexx.com

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Health Link Medical Center Introduces the Regenexx™ Orthopedic Stem Cell Treatments to the San Francisco Area

By Jeffrey M. O'Brien, contributor

Stem cells stored in liquid nitrogen at Advanced Cell Technology in Marlborough, Mass.

FORTUNE -- Imagine yourself the proud but rueful owner of an ancient Jaguar. Every day you dread the uncertainty that comes with trying to get from here to there -- there, more often than not, being the shop. No sooner does one ailment find repair than another appears. At best, it's a slow, uncomfortable ride. Lonely too. There's really no one around who fully understands your plight.

That is how Patricia Riley describes life in a 95-year-old body. Riley, who reached that milestone birthday last St. Patrick's Day, lives alone in the same 1,100-square-foot house in Plainfield, Conn., that she's called home for 64 years, having survived her husband (heart disease), a daughter (breast cancer), and every friend she ever had. "All the people I knew have all gone, Jeffrey," she says in a quivering voice laced with melancholy. "They've all died. I go to church and I never see people my age." Her remaining family includes two daughters, five grandchildren, and eight great-grandchildren, including my two young sons. In a nod to her French-Canadian heritage, we call her Mme.

Mme attributes her longevity to good genes, but she clearly owes a debt to modern medicine. Over the years she's had a cholecystectomy, a hysterectomy, esophageal surgery, a stroke, and ulcerative colitis. Lately she relies on a cane and a walker, and her daily regimen includes pain pills for arthritis, two inhalers for asthma, high-blood-pressure meds, a statin, vitamins, digestion aids, and an anti-anxiety drug that she calls "my nerve pill." Her vision also comes courtesy of medical science. Three years ago Mme was diagnosed with a form of age-related macular degeneration, or AMD, a disease of the back of the retina that is the leading cause of vision loss in the developed world. The ophthalmologist gave her a choice: a needle into her eyeballs every six weeks, or blindness. Mme opted for the injections and now receives shots of an off-label cancer drug called Avastin, which has demonstrated efficacy in halting the progress of her type of AMD. Holding the ailment at bay is all she can hope for. "I'll have to go for as long as I live," she says. "It's just a treatment -- it's not a cure."

Treatments, not cures. This, in a nutshell, is the MO of our health care system, and it's precisely the reason that regenerative medicine -- and stem cell therapy in particular -- has been the subject of so much hope and hype over the past decade or so. Stem cell therapies promise to empower a body to fight ailments by enabling it to build new parts. Think about growing new neurons or heart tissue. Think about the difference between perpetually slathering that old Jag with Bondo and having it heal itself overnight in the garage.

MORE:Stem cell dollars: California leads the way

While stem cells have ignited plenty of religious outrage and political grandstanding, behind the headlines the underlying science has been advancing the way science often does -- by turns slowly and dramatically. To be clear, the earliest stem cell therapies are almost certainly years from distribution. But so much progress has been made at venerable research institutions that it now seems possible to honestly discuss the possibility of a new medical paradigm emerging within a generation. Working primarily with rodents in preclinical trials, MDs and Ph.D.s are making the paralyzed walk and the impotent virile. A stem cell therapy for two types of macular degeneration recently restored the vision of two women. Once they were blind. Now they see! Some experts assert that AMD could be eradicated within a decade. Other scientists are heralding a drug-free fix for HIV/AIDS. Various forms of cancer, Parkinson's, diabetes, heart disease, stroke, and ALS have already been eradicated in mice. If such work translates to humans, it will represent the type of platform advancement that comes along in medicine only once in a lifetime or two. The effect on the economy would be substantial. Champions of stem cell research say it would be on the order of the Internet or even the transistor.

The obstacles along the road from lab rat to human patients are many, of course, but the biggest by far is money. With the dramatic events in the lab, you might think that a gold rush would be under way. That's far from true. Long time horizons, regulatory hurdles, huge R&D costs, public sentiment, and political headwinds have all scared financiers. Wall Street isn't interested in financing this particular dream. Most stem cell companies that have dared go public are trading down 90% or more from their IPOs. Sand Hill Road is AWOL. The National Venture Capital Association doesn't even have a category to track stem cell investments.

Big Pharma would seem to be the most obvious benefactor. The drug companies understand the complexities (and billion-dollar outlays) involved in bringing therapies to market. A few drug companies have kicked the tires on stem cells over the years, but waiting for them to undo the current model is akin to banking on Big Oil to rethink energy. They may do it, but it's unlikely to be by choice. Which leaves stem cell researchers begging for state and federal grants at a time scientific funding is under siege.

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The great stem cell dilemma