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New Teva Representative Joins Mesoblast Board of Directors

May 11th, 2012 8:14 am

MELBOURNE, Australia, May 10, 2012 /PRNewswire/ -- Global regenerative medicine company Mesoblast Limited (MSB.AX) today announced the appointment of Dr Ben-Zion Weiner, Special Adviser to the CEO of Teva Pharmaceutical Industries Ltd, as a non-executive Director of Mesoblast, effective immediately.

Dr Weiner has been Teva's head of global research and development for over three decades, most recently as Chief R&D Officer and a member of the Teva Executive Committee. In this role, he has directly overseen all pharmaceutical R&D and innovative branded product pipeline development.

Dr Weiner has been responsible for the development of hundreds of generic products for the US, EU and other markets. In parallel, he has been responsible for the development and regulatory approval of Teva's innovative product portfolio. Dr Weiner has twice been the recipient of the Rothschild prize for innovation, including for the commercialization of Copaxone in the treatment of multiple sclerosis.

Dr Weiner said: "I am happy and proud to be a Board member of Mesoblast. I trust that stem cell technology is the future of medicine, and that Mesoblast is at the cutting edge of this exciting new field.

"I believe that Mesoblast's unique technology and professional execution capabilities have the potential to deliver multiple innovative biologic products across a broad range of clinical indications," added Dr Weiner.

Mesoblast Chairman, Mr Brian Jamieson, said: "Dr Weiner's extensive pharmaceutical industry experience and his current role as Special Adviser to the Teva CEO make him a very valuable and strategic addition to the Board. We also acknowledge the important contributions and insights provided by Teva's retiring board representative, Kevin Buchi, and wish him well.

"As Mesoblast expands its clinical product portfolio and approaches product commercialization, we will continue to broaden the mix of skills and international expertise of our Directors to ensure that the Board is in the best position to deliver maximal shareholder value."

About Mesoblast Mesoblast Limited (MSB.AX) is a world leader in commercialising biologic products for the broad field of regenerative medicine. Mesoblast has the worldwide exclusive rights for a series of patents and technologies developed over more than 10 years relating to the identification, extraction, culture and uses of adult Mesenchymal Precursor Cells (MPCs). http://www.mesoblast.com

For further information, please contact: Julie Meldrum Corporate Communications Mesoblast Limited E: julie.meldrum@mesoblast.com

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Regenerative medicine: Could the ways animals regenerate hair and feathers help restore human fingers and toes?

May 11th, 2012 8:14 am

ScienceDaily (May 10, 2012) This summer's action film, "The Amazing Spider-Man," is another match-up between the superhero and his nemesis the Lizard. Moviegoers and comic book fans alike will recall that the villain, AKA Dr. Curt Connors, was a surgeon who, after losing an arm, experimented with cell generation and reptilian DNA and was eventually able to grow back his missing limb.

The latest issue of the journal Physiology contains a review article that looks at possible routes that unlock cellular regeneration in general, and the principles by which hair and feathers regenerate themselves in particular.

The authors apply what is currently known about regenerative biology to the emerging field of regenerative medicine, which is being transformed from fantasy to reality.

Review Article

While the concept of regenerative medicine is relatively new, animals are well known to remake their hair and feathers regularly by normal regenerative physiological processes. In their review, the authors focus on (1) how extrafollicular environments can regulate hair and feather stem cell activities and (2) how different configurations of stem cells can shape organ forms in different body regions to fulfill changing physiological needs.

The review outlines previous research on the role of normal regeneration of hair and feathers throughout the lifespan of various birds and mammals. The researchers include what is currently known about the mechanism behind this re-growth, as well as what gaps still exist in the knowledge base and remain ripe for future research.

The review examines dozens of papers on normal "physiological regeneration" -- the re-growth that happens over the course of an animal's life and not in response to an injury. This regeneration takes place to accommodate different stages in an animal's life (e.g., replacing downy chick feathers with an adult chicken's, or replacing the fine facial hair of a young boy with the budding beard of an adolescent), or in response to various environmental conditions (e.g., cats shedding a thick winter coat in the summer heat but re-growing it when the seasons change again, or snowshoe hares switching from brown in the summer to white in the winter for camouflage).

These changes seem to respond both to internal cues such as physiology of the hair follicle itself, or external cues such as the environment, but the mechanisms behind these normal alterations are largely unknown. Stem cells inside the follicle prompt hair and feather regeneration, but researchers are still unsure how to guide those cells to form the shape, size, and orientation of these "skin appendages" so that controlled re-growth is possible. Additionally, scientists are still unsure how to re-grow hair on skin in people after severe injuries that lead to scar tissue.

Importance of the Findings

The reviewed studies suggest that while researchers are making headway in understanding how and why hair and feathers regenerate after normal loss or in response to different life stages, much still remains unknown. This missing knowledge could hold valuable clues to learning how to regenerate much more complicated and valuable structures after loss to injury, such as fingers and toes.

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Study Identifies Cell Subtypes For Potential Personalized Cellular Therapies

May 11th, 2012 8:13 am

Connie K. Ho for RedOrbit.com

A new study by researchers at the University of California, Los Angeles (UCLA) has discovered two adult stem cell-like subpopulations in adult human skin.

The findings allow for further research to be done in the area of personalized medicine and patient-specific cellular therapies.

The study, using technology from Fibrocell Science, allowed the researchers to identify and confirm two types of cells in human skin cell cultures; the possible source of stem cell-like subpopulations from skin biopsies would be faster to perform, painless, and less invasive than current extractions from adipose tissues and bone marrow.

The research, featured in the inaugural issue of BioResearch Open Access, discusses two subtypes of cells. BioResearch Open Access is a bimonthly, peer-reviewed journal. It features scientific topics like biochemistry, bioengineering, gene therapy, genetics, microbiology, neuroscience, regenerative medicine, stem cells, systems biology, tissue engineering and biomaterials, and virology.

Being able to identify two sub-populations of rare, viable and functional cells that behave like stem cells from within the skin is an important finding because both cell types have the potential to be investigated for diverse clinical applications, commented Dr. James A. Bryne, lead author of the report.

Brynes research, first at Stanford University then at UCLA, focused on reprogramming beginnings of cells from animals and then humans. A graduate of Cambridge University, Bryne studied the intra- and inter-species of epigenetic reprogramming. His work also highlighted how primate embryonic stem cells could be derived from somatic cell nuclear transfers.

The study published in BioResearch Open Access confirmed previous research that identified a rare population of cells in adult human skin that had a marker called stage-specific embryonic antigen 3 (SSEA3). Bryne and his colleagues found that there was an increase in the amount of SSEA3 expressing cells after injury to the human skin. It showed that the SSEA3 biomarker could be used to help identify and isolate cells with tissue-regenerative traits.

Finding these rare adult stem cell-like subpopulations in human skin is an exciting discovery and provides the first step towards purifying and expanding these cells to clinically relevant numbers for application to a variety of potential personalized cellular therapies for osteoarthritis, bone loss, injury and/or damage to human skin as well as many other diseases, remarked Bryne, an Assistant Professor of Molecular and Medical Pharmacology at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

Bryne and his team used Fibrocell technology to collect cells from skin samples, cultured the cells in the lab, and purified them by fluorescence-activated cell sorting (FACS). The FACS tagged suspended cells with fluorescent markers for undifferentiated stem cells. The researchers were able to separate the rare cell subpopulations from other kinds of cells.

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Seattle Genetics Announces Data from Investigator Trial of ADCETRIS™ in Relapsed Cutaneous T-Cell Lymphoma

May 11th, 2012 8:12 am

BOTHELL, Wash.--(BUSINESS WIRE)--

Seattle Genetics, Inc. (Nasdaq: SGEN - News) today announced that interim results from an investigator-sponsored phase II clinical trial of ADCETRIS (brentuximab vedotin) in patients with relapsed cutaneous T-cell lymphoma (CTCL) were presented at the Society for Investigative Dermatology annual meeting being held May 9-12, 2012 in Raleigh, NC. ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30. ADCETRIS has not been approved for use in CTCL.

The trial enrolled CTCL patients with mycosis fungoides (MF) or Sezary syndrome. At the time of data analysis, 17 patients had been enrolled, including 16 with MF and one with Sezary syndrome. Patients had received a median of six prior therapies, including a median of four prior systemic therapies. The primary endpoint of the trial is clinical response rate. Secondary endpoints include correlation of clinical response with CD30 expression levels, duration of response, progression-free survival and safety. The study is led by principal investigator Dr. Youn H. Kim, Professor, Department of Dermatology, and Director, Multidisciplinary Cutaneous Lymphoma Program at Stanford University School of Medicine in Stanford, CA. Key findings include:

This is the second data set reported with ADCETRIS in CTCL patients. At the T-Cell Lymphoma Forum in January 2012, interim data were presented from a phase II investigator-sponsored trial in CD30-positive CTCL patients, including lymphomatoid papulosis, primary cutaneous anaplastic large cell lymphoma (pcALCL) or MF. In the trial, which is being conducted by Dr. Madeleine Duvic at The University of Texas MD Anderson Cancer Center, 11 of 17 evaluable patients (65 percent) achieved an objective response, including seven complete remissions (CRs) and four partial remissions (PRs). The most common adverse events were Grade 1, including diarrhea, chest tightness, alopecia, nausea, elevated liver enzymes and peripheral neuropathy.

Seattle Genetics and Millennium: The Takeda Oncology Company recently initiated a randomized phase III clinical trial of ADCETRIS for relapsed CD30-positive CTCL patients. The trial will assess ADCETRIS versus investigators choice of methotrexate or bexarotene in patients with CD30-positive CTCL, including those with pcALCL or MF. The primary endpoint of the study is overall response rate lasting at least 4 months. Approximately 124 patients will be enrolled in the pivotal trial. The phase III trial is being conducted under a Special Protocol Assessment agreement from the U.S. Food and Drug Administration (FDA). The study also received European Medicines Agency scientific advice.

About CTCL

Lymphoma is a general term for a group of cancers that originate in the lymphatic system. There are two major categories of lymphoma: Hodgkin lymphoma and non-Hodgkin lymphoma. Cutaneous lymphomas are a category of non-Hodgkin lymphomas that primarily involve the skin. According to the Cutaneous Lymphoma Foundation, CTCL is the most common type of cutaneous lymphoma and typically presents with red, scaly patches or thickened plaques of skin that often mimic eczema or chronic dermatitis. Progression from limited skin involvement is variable and may be accompanied by tumor formation, ulceration and exfoliation, complicated by itching and infections. Advanced stages are defined by involvement of lymph nodes, peripheral blood and internal organs. According to published literature, up to 50 percent of CTCL patients lesions express CD30.

About ADCETRIS

ADCETRIS (brentuximab vedotin) is an ADC comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-expressing tumor cells.

ADCETRIS (brentuximab vedotin) received accelerated approval from the U.S. Food and Drug Administration for two indications: (1) the treatment of patients with Hodgkin lymphoma after failure of autologous stem cell transplant (ASCT) or after failure of at least two prior multi-agent chemotherapy regimens in patients who are not ASCT candidates, and (2) the treatment of patients with systemic anaplastic large cell lymphoma (sALCL) after failure of at least one prior multi-agent chemotherapy regimen. The indications for ADCETRIS are based on response rate. There are no data available demonstrating improvement in patient-reported outcomes or survival with ADCETRIS.

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Angie the chimp undergoes revolutionary stem cell treatment | Video

May 11th, 2012 8:11 am

Chimp receives stem cells 25-year-old chimp received treatments for a torn ...

Photo by Eric Hasert

ERIC HASERT/TREASURE COAST NEWSPAPERS Dr. Darrell Nazareth, of the Florida Veterinary League in Vero Beach (background) injects chimpanzee stem cells with the help of veterinarian Dr. Jocelyn Bezner, of Save the Chimps, into the knee of Angie, a 25-year-old female chimp suffering from a torn anterior cruciate ligament, at the Save The Chimps Sanctuary in Fort Pierce. The surgery was performed inside the sanctuary's mobile surgical unit, which involved extraction of fat and blood cells that were transported to the Florida Veterinary League in Vero Beach to extract approximately 2.3 billion stem cells, then returning to the chimp sanctuary to finish the one-day procedure. "It feels wonderful, I can't wait to see the results two to three weeks out," Nazareth said about performing the procedure.

Photo by Eric Hasert

ERIC HASERT/TREASURE COAST NEWSPAPERS Nicole Devlin, a laboratory technician at the Florida Veterinary League in Vero Beach, works on a procedure to remove stem cells from blood and fat removed from Angie, a female chimp at Save The Chimps Sanctuary in Fort Pierce. After the stem cells were isolated, they were transported back to the chimp sanctuary in Fort Pierce to be injected back into Angie.

FORT PIERCE A 25-year-old female chimpanzee at the Save-the-Chimps sanctuary in Fort Pierce may be able to run again, thanks to a revolutionary stem cell treatment performed on Wednesday.

Angie, one of the 271 chimpanzees that live at the 150-acre sanctuary, received the cutting-edge treatment for a torn anterior cruciate ligament in her right knee, thanks to its Florida developer, Stemlogix LLC in Weston, and the Florida Veterinary League in Vero Beach.

The procedure, which normally would cost about $2,000, uses an animal's own fat to obtain adult stem cells, which are then injected into the problem area to stimulate growth of healthy cells.

Save-the-Chimps Veterinarian Dr. Linda Gregard handled the stem cell recovery procedures. Under anesthesia, fat was removed from chimp's abdomen Wednesday morning and transported to Dr. Darrell Nazareth at the Florida Veterinary League. Nazareth then isolated stem and regenerative cells from the fat, suspended them in platelet-rich plasma and transported the stem cells back to the sanctuary for the chimp's treatment.

"Hopefully, it stops the inflammation and encourages the injury to heal," said Nazareth, who has performed a similar treatment on 15 dogs and cats from his practice. He estimates that within two to three weeks, improvement will be seen in both the chimp's mobility and pain level.

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Angie the chimp undergoes revolutionary stem cell treatment

May 11th, 2012 8:11 am

Chimp receives stem cells 25-year-old chimp received treatments for a torn ...

Photo by Eric Hasert

ERIC HASERT/TREASURE COAST NEWSPAPERS Dr. Darrell Nazareth, of the Florida Veterinary League in Vero Beach (background) injects chimpanzee stem cells with the help of veterinarian Dr. Jocelyn Bezner, of Save the Chimps, into the knee of Angie, a 25-year-old female chimp suffering from a torn anterior cruciate ligament, at the Save The Chimps Sanctuary in Fort Pierce. The surgery was performed inside the sanctuary's mobile surgical unit, which involved extraction of fat and blood cells that were transported to the Florida Veterinary League in Vero Beach to extract approximately 2.3 billion stem cells, then returning to the chimp sanctuary to finish the one-day procedure. "It feels wonderful, I can't wait to see the results two to three weeks out," Nazareth said about performing the procedure.

Photo by Eric Hasert

ERIC HASERT/TREASURE COAST NEWSPAPERS Nicole Devlin, a laboratory technician at the Florida Veterinary League in Vero Beach, works on a procedure to remove stem cells from blood and fat removed from Angie, a female chimp at Save The Chimps Sanctuary in Fort Pierce. After the stem cells were isolated, they were transported back to the chimp sanctuary in Fort Pierce to be injected back into Angie.

FORT PIERCE A 25-year-old female chimpanzee at the Save-the-Chimps sanctuary in Fort Pierce may be able to run again, thanks to a revolutionary stem cell treatment performed on Wednesday.

Angie, one of the 271 chimpanzees that live at the 150-acre sanctuary, received the cutting-edge treatment for a torn anterior cruciate ligament in her right knee, thanks to its Florida developer, Stemlogix LLC in Weston, and the Florida Veterinary League in Vero Beach.

The procedure, which normally would cost about $2,000, uses an animal's own fat to obtain adult stem cells, which are then injected into the problem area to stimulate growth of healthy cells.

Save-the-Chimps Veterinarian Dr. Linda Gregard handled the stem cell recovery procedures. Under anesthesia, fat was removed from chimp's abdomen Wednesday morning and transported to Dr. Darrell Nazareth at the Florida Veterinary League. Nazareth then isolated stem and regenerative cells from the fat, suspended them in platelet-rich plasma and transported the stem cells back to the sanctuary for the chimp's treatment.

"Hopefully, it stops the inflammation and encourages the injury to heal," said Nazareth, who has performed a similar treatment on 15 dogs and cats from his practice. He estimates that within two to three weeks, improvement will be seen in both the chimp's mobility and pain level.

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Stem cell collaboration could set stage for company’s growth

May 11th, 2012 8:11 am

A stem cell breakthrough at UCLA could mark a big step for a biopharmaceutical company to use its proprietary technology to forge partnerships with pharmaceutical companies and other research institutions.

Fibrocell Sciences technology isolates, purifies and multiplies a patients fibroblast cells, connective skin cells that make collagen. In a research collaboration with the company, UCLA used the technology to isolate, identify and increase the number of different skin cell types, which lead to two rare adult stem cell-like subpopulations being identified in adult human skin SSEA3-expressing regeneration-associated cells associated with skin regeneration after injuries and mesenchymal adult stem cells.

The findings could have broad applications for personalized medicine. Currently, adult stem cells are derived from adipose tissue and bone marrow. Using mesenchymal stem cells would be less invasive and could be more efficient. Mesenchymal stem cells are being used in research to develop osteoblasts, or bone cells; chondrocytes, or cartilage cells; and adipocytes, or fat cells.

David Pernock, the chairman and CEO of Fibrocell, said the move could mark a significant step in the companys growth.

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Stem cells boost brain tumor treatments for some patients, study finds

May 11th, 2012 8:11 am

(CBS News) Patients with brain cancer may face devastating side effects from chemotherapy, but a new study offers a possible solution: stem cells.

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The stem cells form a shield of sorts against the toxic side effects from chemo, according to the researchers behind the study. It was a small trial that involved only three patients with glioblastoma, the most aggressive and common form of a malignant brain tumor that's usually fatal.

Two of the patients survived longer than predicted with help from the stem cell treatment - an average of 22 months - and a third man from Alaska remains alive today with no disease progression almost three years following treatment.

How does it work?

Many patients with the deadly form of brain cancer possess a gene called MGMT. The MGMT gene is typically turned on and counters the effects from some chemotherapy agents, such as temozolomide, rendering them less effective. As such, people with such a gene often have a particularly poor prognosis.

A drug called benzylguanine can block the MGMT gene, thus making tumors more receptive to chemotherapy, but the combination of the drug and chemo are often too toxic for healthy bone marrow cells.

That's where the new stem cell treatment comes in. By combining bone marrow stem cells with a modified version of MGMT in the form of the new treatment, patients' cells were protected from the toxic effects of the cancer drugs and chemotherapy while keeping the tumor cells targeted.

"This therapy is analogous to firing at both tumor cells and bone marrow cells, but giving the bone marrow cells protective shields while the tumor cells are unshielded," study author Dr. Jennifer Adair, a researcher at the Fred Hutchinson Cancer Research Center in Seattle, said in a news release.

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Pluristem stem cell therapy saves a patient, shares jump

May 11th, 2012 8:10 am

Wed May 9, 2012 3:35pm BST

(Reuters) - Pluristem Therapeutics Inc said a 7-year old girl suffering from a bone marrow disease experienced a reversal of her condition after receiving its experimental stem cell therapy, sending the Israeli company's shares up 32 percent.

The girl, suffering from aplastic bone marrow in which the patient has no blood-forming stem cells, had a significant rise in her red cells, white cells and platelets following an injection of Pluristem's therapy -- PLacental eXpanded cells.

"The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow," Reuven Or, the child's physician at Hadassah Medical Center, was quoted in a statement by Pluristem.

Last September, the company said animal studies showed that the therapy had the potential to treat blood tissue complications related with acute radiation syndrome, commonly called radiation sickness.

Last month, the U.S. health regulators gave a go ahead to the company to start a mid-stage trial of the therapy for treating Intermittent Claudication -- a subset of peripheral artery disease.

Pluristem shares, which have gained 5 percent since receiving the FDA nod for the mid-stage trial, were up 15 percent at $2.70 in morning trade on the Nasdaq. They touched a high of $3.10 earlier.

(Reporting by Esha Dey in Bangalore; Editing by Gopakumar Warrier)

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‘Cell shield’ for cancer patients

May 10th, 2012 11:10 am

9 May 2012 Last updated at 14:14 ET By James Gallagher Health and science reporter, BBC News

It may be possible to use "stem cell shielding" to protect the body from the damaging effects of chemotherapy, early results from a US trial suggest.

Chemotherapy drugs try to kill rapidly dividing cancer cells, but they can also affect other healthy tissues such as bone marrow.

A study, in Science Translational Medicine, used genetically modified stem cells to protect the bone marrow.

Cancer Research UK said it was a "completely new approach".

The body constantly churns out new blood cells in the hollow spaces inside bone. However, bone marrow is incredibly susceptible to chemotherapy.

The treatment results in fewer white blood cells being produced, which increases the risk of infection, and fewer red blood cells, which leads to shortness of breath and tiredness.

Researchers at the Fred Hutchinson Cancer Research Center, in Seattle, said these effects were "a major barrier" to using chemotherapy and often meant the treatment had to be stopped, delayed or reduced.

They have tried to protect the bone marrow in three patients with a type of brain cancer, glioblastoma.

One of the researchers, Dr Jennifer Adair, said: "This therapy is analogous to firing at both tumour cells and bone marrow cells, but giving the bone marrow cells protective shields while the tumour cells are unshielded."

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Julio C. Voltarelli, Pioneer in Cell Transplantation, Dies at 63

May 10th, 2012 11:10 am

Dr. Julio C. Voltarelli, who made a significant impact in cell transplantation, dies at 63

Distinguished Brazilian professor pioneered bone marrow transplantation

Newswise Tampa, Fla. (May. 9th , 2012) Julio C. Voltarelli, MD, PhD, professor at the Ribeiro Preto School of Medicine at the University of So Paulo, Brazil, died March 21, 2012 at the age of 63. Dr. Voltarelli, who was on the editorial board of the Cell Transplantation journal, published by Cognizant Communication Corporation, and an important factor in the journals success, was a distinguished stem cell researcher and head of the bone marrow transplantation unit at the Ribeiro Preto School of Medicine.

Dr. Voltarelli had a significant impact on Brazilian stem cell transplantation science, said Dr. Maria C. O. Rodrigues, Dr. Voltarellis longtime colleague. He was driven to bring the benefits of the newest cellular therapies to those with ALS, MS and type 1 diabetes. His efforts and dedication will be greatly missed.

Dr. Voltarelli, a graduate of the Ribeiro Preto School of Medicine, served post-doctoral fellowships at the University of California San Francisco, the Fred Hutchinson Cancer Research Center in Seattle, and the Scripps Research Institute in San Diego. He returned to Brazil in 1992 and started a highly ranked bone marrow transplantation program at the Ribeiro Preto School of Medicine. In 2002, Dr. Voltarelli initiated the schools research efforts in stem cell transplantation for autoimmune diseases, later focusing on diabetes, graft-versus-host disease and sickle cell anemia.

At the time of his death, Dr. Voltarelli, in addition to serving as head of the bone marrow transplantation unit, also served as research coordinator for the Center for Cellular Therapy at the So Paulo Research Foundation and the National Institute of Science and Technology in Stem Cells and Cell Therapy. He was recently elected president of the Brazilian Society of Bone Marrow Transplantation.

His publications included the first books on stem cell transplantation and clinical immunology written in Portuguese. He also founded the Brazilian Society of Stem Cell Transplantation.

His colleagues in Brazil called his lifelong contributions priceless and remembered him for his leadership skills, vision, and sense of humor.

# The Coeditor-in-chiefs for CELL TRANSPLANTATION are at the Center for Neuropsychiatry, China Medical University Hospital, TaiChung, Taiwan, and the Diabetes Research Institute, University of Miami Miller School of Medicine. Contact, Shinn-Zong Lin, MD, PhD at shinnzong@yahoo.com.tw or Camillo Ricordi, MD at ricordi@miami.edu or David Eve, PhD at celltransplantation@gmail.com #

News release by Florida Science Communications http://www.sciencescribe.net

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Pluristem stem cell therapy saves a patient, shares jump

May 10th, 2012 11:10 am

Wed May 9, 2012 3:35pm BST

(Reuters) - Pluristem Therapeutics Inc said a 7-year old girl suffering from a bone marrow disease experienced a reversal of her condition after receiving its experimental stem cell therapy, sending the Israeli company's shares up 32 percent.

The girl, suffering from aplastic bone marrow in which the patient has no blood-forming stem cells, had a significant rise in her red cells, white cells and platelets following an injection of Pluristem's therapy -- PLacental eXpanded cells.

"The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow," Reuven Or, the child's physician at Hadassah Medical Center, was quoted in a statement by Pluristem.

Last September, the company said animal studies showed that the therapy had the potential to treat blood tissue complications related with acute radiation syndrome, commonly called radiation sickness.

Last month, the U.S. health regulators gave a go ahead to the company to start a mid-stage trial of the therapy for treating Intermittent Claudication -- a subset of peripheral artery disease.

Pluristem shares, which have gained 5 percent since receiving the FDA nod for the mid-stage trial, were up 15 percent at $2.70 in morning trade on the Nasdaq. They touched a high of $3.10 earlier.

(Reporting by Esha Dey in Bangalore; Editing by Gopakumar Warrier)

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N.Y. medical schools chart progress with stem cells

May 7th, 2012 12:13 pm

ALBANY -- Almost halfway through a $600 million state program supporting stem cell research, eight medical schools around New York are reporting progress on projects such as replicating liver cells and eradicating leukemia cells.

A new report from Associated Medical Schools of New York updates work at the institutions where hundreds of researchers are starting to unravel causes and potential treatments for conditions ranging from autism to heart disease and cancer. Stem cells are self-renewing and have the ability to develop into other types of cells.

The Mount Sinai School of Medicine reported finding a method to transform human skin cells into stem cells and turned differentiated human stem cells into heart cells. Those findings are expected to result in better understanding of how heart disease develops and allow initial testing of new treatments on stem cells before they are used on human subjects.

The Empire State Stem Cell Program was intended to fund projects in early stages, including those that initially have been unable to get federal or private funding. Grants have also been used for capital projects like renovating labs and establishing new stem cell centers.

The Albert Einstein College of Medicine reported replicating liver cells that could help reduce the need for liver transplants using live donors and cadavers.

Dr. Allan Spiegel said 12 new researchers have been hired with state funding at the Bronx school, which also lists anemia, brain disorders, heart disease and obesity among its stem cell research subjects.

The 11-year program has awarded nearly $223 million in research grants since 2008, with medical schools awarded $137.5 million. This year's state budget includes $44.8 million, the same as last year and down from the $50 million originally planned.

According to the report, the funding has supported about 400 research and related positions from New York City to Buffalo and Rochester.

"This research has the potential to have significant impact on the treatment of patients with specific types of leukemia and will be useful in treating lymphoma and multiple myeloma," the report said.

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Iranian researcher helps treating muscular dystrophy using stem cells

May 7th, 2012 12:13 pm

Source: ISNA, Tehran

Iranian researcher and lecturer Radbod Darabi jointly with his collogues from the University of Minnesota's Lillehei Heart Institute have effectively treated muscular dystrophy in mice using human stem cells derived from a new process which for the first time makes the production of human muscle cells from stem cells efficient and effective.

Radbod Darabi, MD, PhD with Rita Perlingeiro, PhD. (Credit: Image courtesy of University of Minnesota Academic Health Center)

The research outlines the strategy for the development of a rapidly dividing population of muscle-forming cells derived from induced pluripotent (iPS) cells.

IPS cells have all of the potential of embryonic stem (ES) cells, but are derived by reprogramming skin cells. They can be patient-specific, which renders them unlikely to be rejected, and do not involve the destruction of embryos.

This is the first time that human stem cells have been shown to be effective in the treatment of muscular dystrophy.

According to the researchers, there has been a significant lag in translating studies using mouse stem cells into therapeutically relevant studies involving human stem cells.

This lag has dramatically limited the development of cell therapies or clinical trials for human patients.

The latest research from the University of Minnesota provides the proof-of-principle for treating muscular dystrophy with human iPS cells, setting the stage for future human clinical trials.

As the researchers noted one of the biggest barriers to the development of cell-based therapies for neuromuscular disorders like muscular dystrophy has been obtaining sufficient muscle progenitor cells to produce a therapeutically effective response.

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NY medical schools chart progress with stem cells

May 7th, 2012 12:13 pm

ALBANY Almost halfway through a $600 million state program supporting stem cell research, eight medical schools around New York are reporting progress on projects such as replicating liver cells and eradicating leukemia cells.

A new report from Associated Medical Schools of New York updates work at the institutions where hundreds of researchers are starting to unravel causes and potential treatments for conditions ranging from autism to heart disease and cancer. Stem cells are self-renewing and have the ability to develop into other types of cells.

The Mount Sinai School of Medicine reported finding a method to transform human skin cells into stem cells and turned differentiated human stem cells into heart cells. Those findings are expected to result in better understanding of how heart disease develops and allow initial testing of new treatments on stem cells before they are used on human subjects.

Dr. Ihor Lemischka, director of the Black Family Stem Cell Institute at Mount Sinai, said recreating heart cells in a dish from a patient with LEOPARD Syndrome, a disease caused by a genetic mutation, has opened ongoing avenues for researching the disease and screening potential drugs.

The shared research facility at Mount Sinai supports the work at 80 different labs, Lemischka said.

The Empire State Stem Cell Program was intended to fund projects in early stages, including those that initially have been unable to get federal or private funding. Grants have also been used for capital projects like renovating labs and establishing new stem cell centers.

The Albert Einstein College of Medicine reported replicating liver cells that could help reduce the need for liver transplants using live donors and cadavers.

Dr. Allan Spiegel said 12 new researchers have been hired with state funding at the Bronx school, which also lists anemia, brain disorders, heart disease and obesity among its stem cell research subjects.

It offers tremendous potential for understanding the causes of and developing better treatments for diseases like cancer, type 1 diabetes and Parkinsons, he said.

Einstein scientists also report advanced work on blood stem cell function. The school has opened a new $25 million research institute funded through private philanthropy. The report said state money has been used by the schools to leverage both federal and private grants.

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NY medical schools chart progress with stem cells

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Treatment spares Lebanon man from amputation

May 7th, 2012 12:10 pm

LEBANON Retired Dartmouth College professor Roger Smith said he had nothing to lose by joining a stem cell therapy clinical trial. In fact, if he didn't join, he did have something to lose possibly his leg.

In the end it appears the experiment saved his leg. And Smith, who'd already lost two toes to amputation, said he's proud to have played a part in a study that could dramatically improve the outcome for many other patients facing lower-limb amputations resulting from diabetes, high cholesterol, smoking, genetic predisposition and other causes.

The three-year study that ended last year was led by vascular surgeons at Dartmouth-Hitchcock Medical Center in Lebanon, who believe the treatment may offer new hope to sufferers of peripheral artery disease, a condition that causes nearly 60,000 lower-limb amputations every year.

Dr. Richard J. Powell, chief of vascular surgery at Dartmouth-Hitchcock, was the lead investigator of the second-phase national study, which included 72 patients from 20 different sites across the United States.

It's a winner, Powell said. For me, it was dramatic, because there has been nothing that has been shown to work. The results of the third-stage trial are to be presented to the Food and Drug Administration to be approved as a treatment for patients, Powell said.

Peripheral artery disease afflicts more than 9 million patients in the United States, according to Dartmouth-Hitchcock. The condition results from blockages in blood vessels caused by atherosclerosis hardening of the arteries. Options for these patients are limited to the insertion of stents or bypass surgery.

And for about 150,000 patients in the United States who have the most severe form of the disease, amputation is the only option. The results of this recent study suggest amputation could be prevented in the majority of these severe cases.

And if the third phase of the clinical trials confirms the earlier results, the lives of those patients with severe cases could be tremendously affected, Powell said.

This is the first potential treatment that is non-surgical for really severe cardiovascular disease in the legs, he said. Roger Smith's story

About six years ago, Smith, 79, said he was having pain in his leg and trouble sleeping. He was initially misdiagnosed as having the nerve-related condition referred to as spinal stenosis, more commonly known as sciatica. When his condition worsened, he was correctly diagnosed with advanced peripheral artery disease. He lost two of his toes to amputation and was in danger of losing his leg.

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Treatment spares Lebanon man from amputation

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Stem cell therapy to battle HIV?

May 7th, 2012 12:10 pm

(SACRAMENTO, Calif.) -- UC Davis Health System researchers are a step closer to launching human clinical trials involving the use of an innovative stem cell therapy to fight the virus that causes AIDS.

In a paper published in the May issue of the Journal of Virology, the UC Davis HIV team demonstrated both the safety and efficacy of transplanting anti-HIV stem cells into mice that represent models of infected patients. The technique, which involves replacing the immune system with stem cells engineered with a triple combination of HIV-resistant genes, proved capable of replicating a normally functioning human immune system by protecting and expanding HIV-resistant immune cells. The cells thrived and self-renewed even when challenged with an HIV viral load.

"We envision this as a potential functional cure for patients infected with HIV, giving them the ability to maintain a normal immune system through genetic resistance," said lead author Joseph Anderson, an assistant adjunct professor of internal medicine and a stem cell researcher at the UC Davis Institute for Regenerative Cures. "Ideally, it would be a one-time treatment through which stem cells express HIV-resistant genes, which in turn generate an entire HIV-resistant immune system."

To establish immunity in mice whose immune systems paralleled those of patients with HIV, Anderson and his team genetically modified human blood stem cells, which are responsible for producing the various types of immune cells in the body.

Building on work that members of the team have pursued over the last decade, they developed several anti-HIV genes that were inserted into blood stem cells using standard gene-therapy techniques and viral vectors (viruses that efficiently insert the genes they carry into host cells). The resulting combination vector contained:

These engineered blood stem cells, which could be differentiated into normal and functional human immune cells, were introduced into the mice. The goal was to validate whether this experimental treatment would result in an immune system that remained functional, even in the face of an HIV infection, and would halt or slow the progression toward AIDS.

The results were successful on all counts.

"After we challenged transplanted mice with live HIV, we demonstrated that the cells with HIV-resistant genes were protected from infection and survived in the face of a viral challenge, maintaining normal human CD4 levels," said Anderson. CD4+ T-cells are a type of specialized immune cell that HIV attacks and uses to make more copies of HIV.

"We actually saw an expansion of resistant cells after the viral challenge, because other cells which were not resistant were being killed off, and only the resistant cells remained, which took over the immune system and maintained normal CD4 levels," added Anderson.

The data provided from the study confirm the safety and efficacy of this combination anti-HIV lentiviral vector in a hematopoietic stem cell gene therapy setting for HIV and validated its potential application in future human clinical trials. The team has submitted a grant application for human clinical trials and is currently seeking regulatory approval, which is necessary to move on to clinical trials.

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Stem cell therapy to battle HIV?

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Complex cancer industry trial literature is too confusing for patients to understand

May 6th, 2012 3:58 pm
By J. D. Heyes

Have you ever read something so complex and confusing that it frustrated you to
the point of distraction? Well, a new study has found that cancer trial
literature causes that kind of frustration - and may be misleading to patients
as well.

According to Prof. Mary Dixon-Woods, professor of Medical Sociology at the
University of Leicester Department of Health Sciences in Great Britain, a
number of cancer patients found information leaflets describing cancer trials
too long, too incomprehensible and too intimidating.

"These information sheets are poorly aligned with patients' information
needs and how they really make decisions about whether to join a cancer
trial," said Dixon-Woods, lead author of the research http://www.eurekalert.org/pub_releases/2012-03/uol-cti032612.php,
which was published in the international journal Sociology of Health and
Illness.

"Some patients did find them very useful, but many others paid them little
attention. They preferred to rely on discussions they had with their doctor to
make up their minds," she said. Read more…

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Another > $100M month for companies in the cell therapy space

May 6th, 2012 3:58 pm

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Last month we reported here on this blog that March was more than a $100M month for companies in the stem cell and cell-based regenerative medicine space in terms of monies raised.  

What we missed was a $15M grant from Cancer Prevention and Research Institute of Texas (CPRIT) for UK-based CellMedica.  This pumps last month's total to just under $140M.

This month, according to our sources, betters even March's better numbers by coming in at just over $170M though that is largely on the back of one large deal in Asia.  Here's how the numbers break down.

Allocure kicked off the month with a decent $25M Series B round from new syndicate member Lundbeckfond Ventures, as well as previous investors SV Life Sciences and Novo A/S.  Allocure is headed into phase 2 for acute kidney injury with an allogeneic mesenchymal stem cell therapeutic they currently call AC607.  


Little-known Canadian-based, Sernova then announced a $3.6M PIPE to fund continued development of its proprietary Cell Pouch System(TM), and, in particular, to fund the upcoming first-in-man clinical trial for patients with diabetes receiving an islet transplant.  The application to proceed with this trial is currently under review by Health Canada.


Next up was NeoStem closing a $6.8M public offering for "expanding" their contract manufacturing business, Progenitor Cell Therapy, and "enrolling the PreSERVE AMR-001 Phase 2 clinical trial for preserving heart function after a heart attack".  


The biggest deal of the month was a $65M convertible debt financing of China Cord Blood by none other than global powerhouse Kohlberg Kravis Roberts (KKR) through it KKR China Growth Fund L.P., a China-focused investment fund managed by KKR.  We believe this is deal is certainly an investment in the future of China's healthcare market potential but that it is bigger than that.  We believe a significant driver for this deal may likely have been the opportunity to consolidate this sector globally - to use a significant operation and 'war chest' to fund mergers and acquisitions on both the public and private cord blood banking sector worldwide.


The only classic first-round venture raise this month was a milestone-based $5M Series A by Bay City Capital into Phil Coelho's new company, SynGen, to fund his latest iteration of stem cell processing devices.


Forbion Capital then announced that it was leading a series D round, joined by fellow existing investors TVM Capital, Lumira Capital, Intersouth Partners, Caisse de depot et placement du Quebec, Morningside Group, and Aurora Funds, of $25M into Argos Therapeutics in order to kick them into their phase 3.  The hope here is that with some early phase 3 data they may be able to attract the elusive partner they couldn't land with a mere bucket of phase 2 data.


Innovacell landed the only European deal by announcing an 8.3M Euro (~$11M) investment by Buschier, Fides, HYBAG, and Uni Venture.  This will be used for the continued clinical development of its cell-therapy (ICES13) for the treatment of stress-urinary incontinence currently in a ph 3 study in several European countries.


ReNeuron announced a private placement also open to existing shareholders that brought in just under $10M (£6.1M) to support their phase 1 trial in stroke and other pre-clinical, clinical, and regulatory milestones. 


Finally, the Bio-Matrix Scientific Group, in an apparent ongoing quest to continuously reinvent itself, announced at month's end that they had formed a new subsidiary named Regen BioPharma and that they had raised $20M in a financing commitment from Southridge Partners II to purchase its common stock as required over the term of the agreement at a price set by an agreed formula.  This money is said to be dedicated to the acquisition of discovery-stage intellectual property and driving it through to phase 2 trials in an exercise of maximum value creation over a period they claim to be as short as 18-24 months.


..


So in the end, the month saw companies in the space raise just over $170M and even if you back out the stem cell banking deal its still over $100M for cell therapy companies.  


Over the 2 months, then, we've seen just over $311M raised through a variety of means by companies at every stage of maturity and for intended purposes ranging from acquisition, consolidation, early stage clinical development, and phase 3 testing.


--Lee


p.s. If you are aware of other deals in the sector this month, let us know and we'll update this accordingly.


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Gene Hunt Is On for Mental Disabilities in Children

May 6th, 2012 3:58 pm

By Ewen Callaway of Nature magazine

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