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Cellerant Awarded SBIR Contract Funding to Develop CLT-009 for Treatment of Thrombocytopenia

Posted: October 8, 2012 at 8:21 pm

SAN CARLOS, Calif.--(BUSINESS WIRE)--

Cellerant Therapeutics Inc., a biotechnology company developing novel hematopoietic stem cell-based cellular and antibody therapies for blood disorders and cancer, announced today that it has been awarded a Small Business Innovation Research (SBIR) Phase 1 contract and a Phase 2 option from the National Cancer Institute (NCI) valued up to $1,683,503. The SBIR Contract funds the development of CLT-009, a first-in-class, human allogeneic Megakaryocyte Progenitor Cell therapy for the treatment of thrombocytopenia in cancer patients and allows the Company to conduct studies to enable an Investigational New Drug (IND) Application to be filed with the FDA in the next two years.

Thrombocytopenia is characterized as a significant reduction in the concentration of circulating platelets. Platelets are crucial in the process of coagulation to stop bleeding, and thrombocytopenia can increase the risk of severe bleeding in patients. It is becoming an increasingly common problem among oncology patients and a significant dose-limiting toxicity, especially in the treatment of hematological malignancies. Chemotherapy and radiation therapy are the most common causes of thrombocytopenia because the platelet-producing cells, megakaryocytes, and their precursors are highly sensitive to myelosuppressive cytotoxics and ionizing radiation. Thrombocytopenia typically occurs during the initial cycles of high-dose chemotherapy and radiation therapy, usually 614 days after administration. According to Datamonitor, the estimated incidence of cancer patients who suffer from significant chemotherapy-induced thrombocytopenia worldwide was approximately 200,000 in 2008.

Occurrence of severe thrombocytopenia may require dose reductions for chemotherapy regimens which can impact subsequent disease control and survival, especially in the treatment of hematological malignancies such as acute leukemia and high-risk myelodysplastic syndrome. Current treatment options include platelet transfusions which are costly and labor intensive and are associated with risks such as contamination and transmission of viral and bacterial infections. Recombinant human interleukin-11 is the only approved agent for chemotherapy induced thrombocytopenia but its use is limited and has only modest efficacy and significant side effects. CLT-009, a human Megakaryocyte Progenitor Cell product, would be an alternative treatment option, providing the critical megakayocyte progenitor cellular support to rapidly produce platelets in vivo and shorten the duration of severe thrombocytopenia following chemotherapy treatment.

We are delighted to receive this contract from NCI to support the development of our novel, off-the-shelf, platelet product and address a high unmet need, said Ram Mandalam, Ph.D., President and Chief Executive Officer of Cellerant Therapeutics. This contract allows us to not only leverage our experience in developing cellular therapies but also provides us with the ability to bring CLT-009 closer to the clinic. Our unique product portfolio, which now includes CLT-009, along with our CLT-008 myeloid progenitor cell product and our therapeutic antibodies targeting cancer stem cells, demonstrates our continued commitment to developing novel products for the benefit of cancer patients.

In addition to this SBIR contract, Cellerant has previously received grants from the National Institute of Health (NIH) in 2008 2010 to conduct research studies in platelet recovery which it has successfully completed. In its previous studies, Cellerant demonstrated that megakaryocyte progenitor cells were able to produce human platelets in preclinical models with in vivo functionality similar to that of normal human platelets.

This program is funded with Federal funds from the National Institute of Health, Department of Health and Human Services, under Contract No.HHSN261201200076C.

About CLT-009

CLT-009 is a unique, off-the-shelf, cryopreserved, cell-based therapy that contains human Megakaryocyte Progenitor Cells derived from adult hematopoietic stem cells that have the ability to mature into functional platelets in vivo. Cellerant is developing CLT-009 as an effective treatment for chemotherapy and radiation-induced thrombocytopenia in cancer patients.

About Cellerant Therapeutics

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Biopharma with personalized cell therapy raises $45M in stock sale, forges collaboration deal

Posted: October 8, 2012 at 8:21 pm

A company with the first FDA-approved personalized cell therapy for reducing wrinkles has raised $45 million in a private stock sale. The financing was sought to improve manufacturing capacity for the therapy and advance other uses for it such as treating burn victims, according to a company statement.

Fibrocell Sciences aesthetic therapeutic, Laviv, secured got the green light from the U.S. Food and Drug Administration last year. Laviv uses individuals fibroblast cells to reduce nasolabial fold wrinkles, creases on the face that start from the outer corners of the nose and go down to the corners of the mouth. It also has an acne therapy in phase 3 clinical trials and a burn scar therapy in phase 2 trials.

The Exton, Pennsylvania biopharmaceutical companys personalized cell development platform technology isolates, purifies and multiplies a patients fibroblast cells, connective skin cells that make collagen.

Additionally, Fibrocell agreed to a strategic collaboration with biotechnology firm Intrexon which can provide genome engineering, cell processing, and cell system engineering, among other services, to help advance Fibrocells personalized cell therapy program.

As part of the financing deal, Third Security LLC will get two seats on Fibrocells board.

Personalized stem cell development fits into the broader category of personalized medicine, regarded as the future of medicine in which therapies will be better targeted to individuals and more effective.

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Nobel in medicine goes to stem cell researchers

Posted: October 8, 2012 at 6:20 pm

The 2012 Nobel Prize in Physiology or Medicine has been awarded to John B. Gurdon and Shinya Yamanaka for research into stem cells.

"The Nobel Prize recognizes two scientists who discovered that mature, specialized cells can be reprogrammed to become immature cells capable of developing into all tissues of the body. Their findings have revolutionized our understanding of how cells and organisms develop," the Nobel Committee at the Karolinska Institute wrote in a statement on Monday.

The Local asked the panel how the discovery affects debate about moral aspects of stem cell research.

"We have a very open debate in science, the Nobel Committee does not participate so much in that, but we as individual scientists and physicians do, and regulations evolve according to a general consensus of what is acceptable in society," said Anna Wedell, professor and researcher at the Karolinksa Institute.

"Every great discovery in biology that has applications in humans needs to be discussed and that is being done. We try to contribute to the best of our knowledge."

Gurdon discovered in 1962 that the specialization of cells is reversible. He replaced the immature cell nucleus in an egg cell of a frog with the nucleus from a mature intestinal cell. This modified egg cell developed into a normal tadpole. The DNA of the mature cell still had all the information needed to develop all cells in the frog.

Yamanaka discovered more than 40 years later, in 2006, how intact mature cells in mice could be reprogrammed to become immature stem cells. Surprisingly, by introducing only a few genes, he could reprogram mature cells to become pluripotent stem cells, i.e. immature cells that are able to develop into all types of cells in the body.

According to Jonas Frisn, a professor of stem cell research at Karolinska and a member of the Nobel Assembly, the discoveries of Gurdon and Yamanaka can be likened to finding the "master key" of cell biology.

"One can distinguish two major advances here. One is conceptual in how we understand how cells are locked into their specialist states and that it's actually possible to reverse this process," he told The Local.

"The other is in terms of applications and these discoveries have resulted in the technology to generate stems cells which can give rise to all cell types in the body and this is a very major practical advancement."

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Stem-Cell Pioneers Gurdon, Yamanaka Win Nobel Prize

Posted: October 8, 2012 at 6:20 pm

John B. Gurdon transferred DNA between a tadpole and a frog to clone the first animal. Shinya Yamanaka used Gurdons concept to turn ordinary skin into potent stem cells. Both won the Nobel Prize for medicine today.

Gurdon, 79, a researcher at the University of Cambridge in the U.K., and Yamanaka, 50, a professor at Kyoto University in Japan, will share the 8 million-kronor ($1.2 million) prize, the Nobel Assembly said today in Stockholm. The pairs findings have created new opportunities to study diseases and develop methods for diagnosis and therapy, the assembly said in a statement.

Gurdons feat, in 1962, paved the way in 1996 for the cloning of Dolly the sheep and, 10 years later, for Yamanaka, who turned mouse skin cells into stem cells with the potential to become any cell in the body. That achievement was lauded by some politicians and religious figures as a more ethical way to make stem cells because it doesnt destroy human life.

This field has had a long history, starting with John Gurdon, Yamanaka, who was born the same year Gurdon published his achievement, said in an interview on the Nobel Assemblys website. I was able to initiate my project because of his experiments 50 years ago.

Stem cells are found in human embryos and in some tissues and organs of adults, and have the potential to develop into different types of cells. Thats spurred scientists to look at ways of harnessing their power to treat diseases such as Alzheimers, stroke, diabetes and rheumatoid arthritis, according to the U.S. National Institutes of Health.

Gurdon showed that mature cells from specific parts of an animals body retain all the genetic information they had as immature stem cells. He took a cell from a tadpoles gut, extracted the nucleus, and inserted it into the egg cell of an adult frog whose own nucleus had been removed. That reprogrammed egg cell developed into a tadpole with the genetic characteristics of the original tadpole, and subsequent trials yielded adult frogs.

Gurdon overturned the prevailing view that as cells differentiate, they lose genes and their ability to generate other cells of any kind, said Alan Colman, the executive director of the Singapore Stem Cell Consortium, who gained his doctorate under Gurdon at Cambridge.

Hes amazingly passionate, Colman said in an interview before the award was announced. He was the sort of supervisor who you found it difficult to get appointments with, not because he was flying around the world, but because he was doing experiments all the time.

Gurdon was answering e-mails in his laboratory when he received the call from Sweden today about the prize, he said in an interview on the Nobel Assemblys website. His first reaction was, Its amazing if its really true, he said. Could it be that someones pulling your leg? That has happened before.

Gurdon will celebrate at a reception that his institute is hosting today, and then hell be back to work early tomorrow, he said at a London news conference today.

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NeoStem Announces Very Small Embryonic-Like Cells (VSEL(TM)) Publication in Stem Cells and Development

Posted: October 8, 2012 at 6:20 pm

NEW YORK, Oct. 8, 2012 (GLOBE NEWSWIRE) -- NeoStem, Inc. (NYSE MKT:NBS), an emerging leader in the fast growing cell therapy market, announced today that data from its collaborative studies with the University of Michigan School of Dentistry further expands the therapeutic potential of its proprietary regenerative cell therapy product, "VSELSTM" (very small embryonic-like stem cells), by demonstrating bone regeneration capabilities in a study published online ahead of print1 in the journal Stem Cells and Development (DOI: 10.1089/scd.2012.0327). The paper highlights that human VSEL stem cells form human bone when implanted in the bone tissue of SCID mice.

VSELs are a population of stem cells found in adult bone marrow with potential regenerative properties similar to those of embryonic stem cells. NeoStem has shown that these cells can be mobilized into the peripheral blood, enabling a minimally invasive means for collecting what NeoStem believes to be a population of stem cells that have the potential to achieve the positive benefits associated with embryonic stem cells without the ethical or moral dilemmas or the potential negative effects known to be associated with embryonic stem cells.

This published controlled study, funded by NIH and led by Dr. Russell Taichman, Major Ash Collegiate Professor and Co-Director of the Scholars Program in Dental Leadership Department of Periodontics & Oral Medicine, University of Michigan and Dr. Aaron Havens, Department of Orthodontics and Pediatric Dentistry at University of Michigan, involved isolating G-CSF mobilized VSEL stem cells from the blood of healthy donors and transplanting them into burr holes made in the cranial bones of SCID mice. After three months, it was observed that the implanted VSEL stem cells had differentiated into human bone tissue in the crania of the mice. Dr. Taichman stated, "I believe this work represents a true partnership between Industry and Academic Institutions. Our findings that VSEL cells can generate human bone in animals would not have been feasible without the help and vision that Dr. Denis Rodgerson and his team at NeoStem brought to the table. It was my privilege to have been a part of this collaborative effort, and I see the resulting data as a significant milestone in stem cell therapy development. It is truly inspiring."

Dr. Robin Smith, Chairman and CEO of NeoStem, added, "This is very exciting data that we believe will be the foundation for future VSEL stem cell studies of bone regeneration in humans. We look forward to moving the development work from the laboratory into the clinic to develop a therapeutic stem cell product to enhance bone formation in humans."

About NeoStem, Inc.

NeoStem, Inc. continues to develop and build on its core capabilities in cell therapy, capitalizing on the paradigm shift that we see occurring in medicine. In particular, we anticipate that cell therapy will have a significant role in the fight against chronic disease and in lessening the economic burden that these diseases pose to modern society. We are emerging as a technology and market leading company in this fast developing cell therapy market. Our multi-faceted business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC ("PCT"), with a medically important cell therapy product development program, enabling near and long-term revenue growth opportunities. We believe this expertise and existing research capabilities and collaborations will enable us to achieve our mission of becoming a premier cell therapy company.

Our contract development and manufacturing service business supports the development of proprietary cell therapy products. NeoStem's most clinically advanced therapeutic, AMR-001, is being developed at Amorcyte, LLC ("Amorcyte"), which we acquired in October 2011. Amorcyte is developing a cell therapy for the treatment of cardiovascular disease and is enrolling patients in a Phase 2 trial to investigate AMR-001's efficacy in preserving heart function after a heart attack. Athelos Corporation ("Athelos"), which is approximately 80%-owned by our subsidiary, PCT, is collaborating with Becton-Dickinson in the early clinical exploration of a T-cell therapy for autoimmune conditions. In addition, pre-clinical assets include our VSELTM Technology platform as well as our mesenchymal stem cell product candidate for regenerative medicine. Our service business and pipeline of proprietary cell therapy products work in concert, giving us a competitive advantage that we believe is unique to the biotechnology and pharmaceutical industries. Supported by an experienced scientific and business management team and a substantial intellectual property estate, we believe we are well positioned to succeed.

Forward-Looking Statements for NeoStem, Inc.

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management's current expectations, as of the date of this press release, and involve certain risks and uncertainties. Forward-looking statements include statements herein with respect to the successful execution of the Company's business strategy, including with respect to the Company's or its partners' successful development of AMR-001 and other cell therapeutics, the size of the market for such products, its competitive position in such markets, the Company's ability to successfully penetrate such markets and the market for its CDMO business, and the efficacy of protection from its patent portfolio, as well as the future of the cell therapeutics industry in general, including the rate at which such industry may grow. Forward looking statements also include statements with respect to satisfying all conditions to closing the disposition of Erye, including receipt of all necessary regulatory approvals in the PRC. The Company's actual results could differ materially from those anticipated in these forward- looking statements as a result of various factors, including but not limited to (i) the Company's ability to manage its business despite operating losses and cash outflows, (ii) its ability to obtain sufficient capital or strategic business arrangement to fund its operations, including the clinical trials for AMR-001, (iii) successful results of the Company's clinical trials of AMR-001 and other cellular therapeutic products that may be pursued, (iv) demand for and market acceptance of AMR-001 or other cell therapies if clinical trials are successful and the Company is permitted to market such products, (v) establishment of a large global market for cellular-based products, (vi) the impact of competitive products and pricing, (vii) the impact of future scientific and medical developments, (viii) the Company's ability to obtain appropriate governmental licenses and approvals and, in general, future actions of regulatory bodies, including the FDA and foreign counterparts, (ix) reimbursement and rebate policies of government agencies and private payers, (x) the Company's ability to protect its intellectual property, (xi) the company's ability to successfully divest its interest in Erye, and (xii) matters described under the "Risk Factors" in the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 20, 2012 and in the Company's other periodic filings with the Securities and Exchange Commission, all of which are available on its website. The Company does not undertake to update its forward-looking statements. The Company's further development is highly dependent on future medical and research developments and market acceptance, which is outside its control.

(1) Human Very Small Embryonic-Like Cells Generate Skeletal Structures, In Vivo. Havens A., et al., Stem Cells and Development.

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UK, Japan scientists win Nobel for adult stem cell discovery

Posted: October 8, 2012 at 6:20 pm

1 of 7. Kyoto University Professor Shinya Yamanaka (L) and John Gurdon of the Gurdon Institute in Cambridge are seen at a symposium on induced pluripotent stem cell in Tokyo, in this photo taken by Kyodo on April 2008.

Credit: Reuters/Kyodo

By Anna Ringstrom

STOCKHOLM | Mon Oct 8, 2012 11:27am EDT

STOCKHOLM (Reuters) - Scientists from Britain and Japan shared a Nobel Prize on Monday for the discovery that adult cells can be transformed back into embryo-like stem cells that may one day regrow tissue in damaged brains, hearts or other organs.

John Gurdon, 79, of the Gurdon Institute in Cambridge, Britain and Shinya Yamanaka, 50, of Kyoto University in Japan, discovered ways to create tissue that would act like embryonic cells, without the need to collect the cells from embryos.

They share the $1.2 million Nobel Prize for Medicine, for work Gurdon began 50 years ago and Yamanaka capped with a 2006 experiment that transformed the field of "regenerative medicine" - the search for ways to cure disease by growing healthy tissue.

"These groundbreaking discoveries have completely changed our view of the development and specialization of cells," the Nobel Assembly at Stockholm's Karolinska Institute said.

All of the body starts as stem cells, before developing into tissue like skin, blood, nerves, muscle and bone. The big hope is that stem cells can grow to replace damaged tissue in cases from spinal cord injuries to Parkinson's disease.

Scientists once thought it was impossible to turn adult tissue back into stem cells. That meant new stem cells could only be created by taking them from embryos, which raised ethical objections that led to research bans in some countries.

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Stem Cell Discoveries Snag Nobel Prize in Medicine

Posted: October 8, 2012 at 6:20 pm

Two scientists who discovered the developmental clock could be turned back in mature cells, transforming them into immature cells with the ability to become any tissue in the body pluripotent stem cells are being honored with the Nobel Prize in Physiology or Medicine.

The Nobel Prize honoring Sir John B. Gurdon and Shinya Yamanaka was announced today (Oct. 8) by the Royal Swedish Academy of Sciences.

Th duo's work revealed what scientists had thought impossible. Just after conception, an embryo contains immature cells that can give rise to any cell type such as nerve, muscle and liver cells in the adult organism; these are called pluripotent stem cells, and scientists believed once these stem cells become specialized to carry out a specific body task there was no turning back.

Gurdon, now at the Gurdon Institute in Cambridge, England, found this wasn't the case when in 1962 he replaced the nucleus of a frog's egg cell with the nucleus taken from a mature intestinal cell from a tadpole. And voila, the altered frog egg developed into a tadpole, suggesting the mature nucleus held the instructions needed to become all cells in the frog, as if it were a young unspecialized cell. In fact, later experiments using nuclear transfer have produced cloned mammals. [5 Amazing Stem Cell Discoveries]

Then in 2006, Yamanaka, who was born in 1962 when Gurdon reported his discovery and is now at Kyoto University, genetically reprogrammed mature skin cells in mice to become immature cells able to become any cell in the adult mice, which he named induced pluripotent stem cells (iPS). Scientists can now derive such induced pluripotent stem cells from adult nerve, heart and liver cells, allowing new ways to study diseases.

When Yamanaka received the call from Stockholm about his award, he was doing housework, according to an interview with the Nobel Prize website. "It is a tremendous honor to me," Yamanaka said during that interview.

As for his hopes for mankind with regard to stem cells, he said, "My goal, all my life, is to bring this technology, stem cell technology, to the bedside, to patients, to clinics." He added that the first clinical trials of iPS cells will begin next year.

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UK, Japan scientists win Nobel for stem cell breakthroughs

Posted: October 8, 2012 at 6:20 pm

STOCKHOLM (Reuters) - Scientists from Britain and Japan shared the Nobel Prize in Medicine on Monday for the discovery that adult cells can be reprogrammed back into stem cells which can turn into any kind of tissue and may one day repair damaged organs.

John Gurdon, 79, of the Gurdon Institute in Cambridge, Britain and Shinya Yamanaka, 50, of Kyoto University in Japan, discovered ways to create tissue that would act like embryonic cells, without the need to harvest embryos. They share the $1.2 million prize equally.

"These groundbreaking discoveries have completely changed our view of the development and specialisation of cells," the Nobel Assembly at Stockholm's Karolinska Institute said in a statement.

The big hope for stem cells is that they can be used to replace damaged tissues in everything from spinal cord injuries to Parkinson's disease.

All of the tissue in the body starts as stem cells, before developing into mature skin, blood, nerves, muscle and bone.

Scientists once thought it was impossible to turn adult tissue back into stem cells, which meant that new stem cells could only be created by harvesting embryos. But Yamanaka and Gurdon showed that development can be reversed, turning adult cells back into cells that behave like embryos.

With "induced pluripotency stem cells", or iPS cells, ordinary skin or blood cells from adults are transformed back into stem cells which doctors hope will be able to repair damaged organs without being rejected by the immune system.

There are concerns, however, that iPS cells could grow out of control and develop into tumours.

"The eventual aim is to provide replacement cells of all kinds," Gurdon's Institute explains on its website.

"We would like to be able to find a way of obtaining spare heart or brain cells from skin or blood cells. The important point is that the replacement cells need to be from the same individual, to avoid problems of rejection and hence of the need for immunosuppression."

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Analysis: Reprogrammed cells open new medical window

Posted: October 8, 2012 at 6:20 pm

LONDON (Reuters) - The Nobel Prize-winning discovery of how to reprogram ordinary cells to behave like embryonic stem cells offers a way to skirt around ethical problems with human embryos, but safety concerns make their future use in treating disease uncertain.

While researchers have already applied the scientific breakthroughs of Britain's John Gurdon and Japan's Shinya Yamanaka to study how diseases develop, making such cells into new treatments will involve a lot more checks.

Stem cells act as the body's master cells, providing the source material for all other cells. They could transform medicine by regenerating tissue for diseases ranging from blindness to Parkinson's disease.

Creating embryo-like stem cells without destroying embryos gets round a key controversy by avoiding the need to process embryos left over at fertility clinics - a system that has led to political objections in the United States and elsewhere.

Reprogrammed cells - known as induced pluripotent stem cells, or iPS cells - offer an ethically neutral alternative. They have been a source of intense research since Yamanaka discovered their potential in 2006, building on work that Gurdon did in frogs and tadpoles 40 years earlier.

SAFETY CONCERNS

Recently, however, different research groups have noticed problems with iPS cells, suggesting they may not be as good as embryonic ones. In one study, iPS cells died more quickly and another found multiple genetic mutations, raising concerns that they could cause tumors.

Despite this, Japanese researchers hope to test iPS cells in clinical trials for a form of blindness as early as next year - catching up with recent successful eye trials using embryonic stem cells.

Researchers in the West are generally more wary.

"There is a bit of a divergence between Japan and the rest of the world on this," Chris Mason, professor of regenerative medicine at University College London, told Reuters.

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Stem cell work wins Nobels for Japanese, Briton

Posted: October 8, 2012 at 6:19 pm

STOCKHOLM -- 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 and 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.

Gurdon, 79, has served as a professor of cell biology at Cambridge University's Magdalene College and is currently at the Gurdon Institute in Cambridge, which he founded. Yamanaka, 50, worked at the Gladstone Institute in San Francisco and Nara Institute of Science and Technology in Japan. He is currently at Kyoto University and also affiliated with the Gladstone Institute. Yamanaka is the first Japanese scientist to win the Nobel medicine award since 1987.

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