StemCell Therapy

Doctors in Sweden have replaced a vital blocked blood vessel in a 10-year-old girl using the first vein grown in a lab from a patient's own stem cells.

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A CPAP device, the Darth Vader-like mask used to ease breathing in sleep apnea sufferers, might be the least attractive thing a man can wear at night, but it could wind up improving his sex life, according to a new study released today at an annual meeting of sleep experts.

The successful transplant operation, reported online in The Lancet medical journal on Thursday, marks a further advance in the search for ways to make new body parts.

It could open the door to stem cell-based grafts for heart bypass and dialysis patients who lack suitable blood vessels for replacement surgery, and the Swedish team said it is now working with an undisclosed company to commercialize the process.

"I'm very optimistic that in the near future we will be able to get both arteries and veins transplanted on a large scale," said Suchitra Sumitran-Holgersson, professor of transplantation biology at the University of Gothenburg, and a member of the team that performed the operation in March 2011.

The advantage of using tissue grown from a patient's own cells is that there is no risk of organ rejection and hence no need for lifelong immunosuppressive drugs.

Four years ago, a 30-year-old woman received the world's first transplant of a tailor-made windpipe, grown in a similar way by seeding a stripped-down donor organ with her own stem cells. Other such trachea operations have followed since.

The latest case involved a young girl with an obstructed hepatic portal vein, which drains blood from the intestines and spleen to the liver. Its blockage can be fatal.

The team from the University of Gothenburg took a 9 cm (3.5 inch) section of groin vein from a deceased donor and removed all the living cells, leaving just a protein scaffold tube. Stem cells extracted from the girl's bone marrow were then injected onto the tube and two weeks later the graft was implanted.

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Vein grown from stem cells saves girl

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

Millennium: The Takeda Oncology Company, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited (TSE:4502), today announced updated survival data from a pivotal Phase II clinical trial of single-agent brentuximab vedotin in patients with relapsed or refractory Hodgkin lymphoma (HL) after autologous stem cell transplant (ASCT) showing that the median overall survival has not been reached after a 26.5 month median follow-up. The data will be reported during an oral presentation at the 17th European Hematology Association (EHA) Annual Meeting being held June 14-17, 2012 in Amsterdam, Netherlands. Brentuximab vedotin is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of the majority of types of HL.

Heavily pretreated Hodgkin lymphoma patients who relapse following autologous stem cell transplant often have a poor prognosis and there is a high unmet medical need for effective treatment options, said Scott Smith M.D., Ph.D., Loyola University Medical Center. These updated overall survival results from the pivotal trial are encouraging and suggest that brentuximab vedotin may play an important role in the treatment of patients with relapsed or refractory disease.

Long-term Follow-up Results of an Ongoing Pivotal Study of Brentuximab Vedotin in Patients with Relapsed or Refractory Hodgkin Lymphoma

A pivotal trial was conducted in 102 patients with relapsed or refractory HL after ASCT. The primary endpoint was objective response rate (ORR) per independent review. The secondary endpoints were complete remission (CR) rate, duration of response, progression-free survival (PFS), overall survival (OS), and safety and tolerability. At the time of the long-term follow-up analysis, the median observation time from first dose was 26.5months. Data, to be presented by Dr. Smith, include:

Patients received 1.8milligrams per kilogram of brentuximab vedotin every 3 weeks as a 30-minute outpatient intravenous infusion for up to 16cycles. Patients received a median of nine cycles of brentuximab vedotin while on trial. The median age of patients in the pivotal trial was 31 years. Enrolled patients had received a median of 3.5 (range 113) prior cancer-related systemic therapies, excluding ASCT. Seventy-one percent of patients had primary refractory disease, defined in the study protocol as patients who relapsed within three months of attaining CR or failed to achieve a CR, and 42 percent had not responded to their most recent prior therapy.

Details of the oral presentation are as follows:

About Brentuximab Vedotin

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.

Brentuximab vedotin is not approved for use outside the United States. The marketing authorization application for brentuximab vedotin in relapsed or refractory Hodgkin lymphoma and sALCL, filed by Takeda Global Research & Development Centre (Europe), was accepted for review by the European Medicines Agency for review in June 2011.

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Millennium Highlights Updated Survival Data from ADCETRIS® (Brentuximab Vedotin) Pivotal Trial in Patients with ...

June 14, 2012, SEATTLE /PRNewswire/ -- Cell Therapeutics, Inc. ("CTI") (NASDAQ and MTA: CTIC), a company focused on translating science into novel cancer therapies, today announced that former OncoMed Pharmaceuticals executive, Steven E. Benner, M.D., M.H.S., has joined CTI as Executive Vice President and Chief Medical Officer ("CMO"), reporting to James A. Bianco, M.D., Chief Executive Officer. Dr. Benner will take over all drug development activities at the company.Dr. Benner was previously senior vice president and chief medical officer at OncoMed, a venture-backed biotechnology company focused on the development of cancer stem cell targeting agents. Prior to OncoMed, he was CMO at Protein Design Labs ("PDL"), where he was accountable for all development activities including clinical development, clinical operations, biometry, regulatory affairs, and safety. He also served as Chair of the Portfolio and Clinical Development Management Committees of PDL. Before PDL he held several senior executive roles at Bristol-Myers Squibb in global development, life cycle management, and licensing and alliances.

"Dr. Benner brings to CTI his proven track record of success in advancing the development of innovative therapies for cancer patients," said Dr. Bianco. "His appointment is the first step in re-aligning our portfolio efforts, as we focus on advancing pacritinib into Phase III pivotal studies later this year."

With the new company initiative of the planned Pixuvri launch in Europe later this year, Jack W. Singer, M.D., will assume the newly-created role of Executive Vice President ("EVP") of Global Medical Affairs and Translational Medicine, responsible for cancer drug development strategy, global medical affairs, and life cycle management.

"Given Jack's impressive academic credentials, the respect he receives from an international network of key opinion leaders in the field, and his track record in oncology drug development, this was a natural promotion as we introduce Pixuvri in Europe," said Dr. Bianco.

"CTI has assembled an impressive late-stage portfolio of novel targeted therapies that address a spectrum of blood related cancers," said Dr. Benner. "With two drugs in Phase III and two more expected to enter Phase III trials within a year, this is an exciting and transformational time to join the team at CTI."

About Pixuvri (pixantrone)Pixuvri is a novel aza-anthracenedione with unique structural and physio-chemical properties. Unlike related compounds,Pixuvri forms stable DNA adducts and in preclinical models has superior anti-lymphoma activity compared to related compounds. Pixuvri was structurally designed so that it cannot bind iron and perpetuate oxygen radical production or form a long-lived hydroxyl metabolite -- both of which are the putative mechanisms for anthracycline induced acute and chronic cardiotoxicity. These novel pharmacologic properties allow Pixuvri to be administered to patients with near maximal lifetime exposure to anthracyclines without unacceptable rates of cardiotoxicity, and, because Pixuvri is not a vesicant, allow it to be safely delivered via a peripheral intravenous catheter.

In May 2012 Pixuvri received conditional marketing authorization in the EU as monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive NHL. The benefit of pixantrone treatment has not been established in patients when used as fifth line or greater chemotherapy in patients who are refractory to last therapy.The Summary of Product Characteristics ("SmPC") has the full prescribing information, including the safety and efficacy profile of Pixuvri in the approved indication. The SmPC is available at http://ec.europa.eu/health/documents/communityregister/html/h764.htm#ProcList.

Pixuvri is currently available in the EU through Named Patient Programs.

Pixuvri does not have marketing approval in the United States.

About Conditional Marketing AuthorizationSimilar to accelerated approval regulations inthe United States, conditional marketing authorizations are granted in the EU to medicinal products with a positive benefit/risk assessmentthat address unmet medical needs and whose availability would result in a significant public health benefit. A conditional marketing authorization is renewable annually. Under the provisions of the conditional marketing authorization for Pixuvri, CTI will be required to complete a post-marketing study aimed at confirming the clinical benefit previously observed.

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Cell Therapeutics Appoints New Chief Medical Officer

Cryo-Save Group N.V. (Euronext: CRYO, `Cryo-Save`, or `the Group`), the leading international stem cell storage company and the largest family stem cell bank in Europe, announced today that it did not get the permission to process and store stem cells in France along with an update on the cost savings program.

In addition to the cessation of the Cryo-Lip activities in the US and the cutback of the Cryo-Lip activities in Europe as previously disclosed in the first quarter trading update, the Group decided to dismantle its French operations. After more than 3 years of disputes with the healthcare authorities in France, including several court cases, Cryo-Save recently got confirmed that it will not get the permission from the authorities to collect, process and store stem cells from umbilical cord blood. The decision includes laying-off the French employees, discontinuing the litigations and indemnity procedures and selling off the building in Lyon. Together with some other smaller implemented cost savings, the Group has currently realised sustainable cost savings of at least 2 million per annum. Further cost savings are being considered.

Arnoud van Tulder, Chief Executive Officer, commented:

"We are certainly very disappointed that the French authorities take this negative position, which deviates from all other European countries. Expectant parents in France are now refrained from storing their stem cells from umbilical cord blood and tissue for their family, which is, beside France, allowed across the whole world.

We are truly convinced that the impact of the cost saving measures together with the restoration of the Cryo-Save business, which is well underway, will result in sustainably improved top- and bottom-line results as of the second half of the year."

Enquiries:

Free footage is available on http://www.videobankonline.com.

About Cryo-Save (www.cryo-save.com/group)

Cryo-Save, the leading international family stem cell bank, stores more than 200,000 samples from umbilical cord blood, cord tissue and adipose tissue. There are already many diseases treatable by the use of stem cells, and the number of treatments will only increase. Driven by its international business strategy, Cryo-Save is now represented in over 40 countries on 3 continents, with ultra-modern processing and storage facilities in Belgium, Germany, Dubai, India and South Africa.

The owner of this announcement warrants that: (i) the releases contained herein are protected by copyright and other applicable laws; and (ii) they are solely responsible for the content, accuracy and originality of the information contained therein.

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Cryo-Save Group N.V: Further cost savings with closure of France

For the first time doctors have successfully transplanted a vein grown with a patient's own stem cells, another example of scientists producing human body parts in the lab.

In this case, the patient was a 10-year-old girl in Sweden who was suffering from a severe vein blockage to her liver. Last March, the girl's doctors decided to make her a new blood vessel to bypass the blocked vein instead of using one of her own or considering a liver transplant.

They took a 9-centimetre section of vein from a deceased donor, which was stripped of all its cells, leaving just a hollow tube. Using stem cells from the girl's bone marrow, scientists grew millions of cells to cover the vein, a process that took about two weeks. The new blood vessel was then transplanted into the patient.

Because the procedure used her own cells, the girl did not have to take any drugs to stop her immune system from attacking the new vein, as is usually the case in transplants involving donor tissue.

"This is the future for tissue engineering, where we can make tailor-made organs for patients," said Suchitra Sumitran-Holgersson of the University of Gothenburg, one of the study's authors.

She and colleagues published the results of their work online Thursday in the British medical journal Lancet. The work was paid for by the Swedish government.

The science is still preliminary and one year after the vein was transplanted, it needed to be replaced with another lab-grown vein when doctors noticed the blood flow had dropped. Experts from University College London raised questions in an accompanying commentary about how cost-effective the procedure might be, citing "acute pressures" on health systems that might make these treatments impractical for many patients.

Sumitran-Holgersson estimated the cost at between $6,000 and $10,000.

Similar methods have already been used to make new windpipes and urethras for patients. Doctors in Poland have also made blood vessels grown from donated skin cells for dialysis patients.

Patients with the girl's condition are usually treated with a vein transplant from their own leg, a donated vein, or a liver transplant. Those options can be complicated in children and using a donated vein or liver also requires taking anti-rejection medicines.

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Vein grown from girl's own stem cells transplanted

June 14, 2012

Brett Smith for redOrbit.com

A successful transplant operation in Sweden points to a medical future where your doctor can grow a transplant organ from your own cells, making organ donation a thing of the past.

Doctors have now successfully transplanted a vein grown with a patients own stem cells without complications or the need for immunosuppressants, according to a report published this week in The Lancet. The patient was a 10-year-old girl in Sweden who was suffering from a potentially fatal blockage in the vein which drains blood from the intestines and spleen to the liver.

Last March, a team of doctors at the University of Gothenburg decided to grow the new blood vessel used to bypass the blocked vein instead of using an invasive neck or leg surgery to extract one of her own.

The young girl in this report was spared the trauma of having veins harvested from the deep neck or leg with the associated risk of lower limb disorders, and avoided the need for a liver or multivisceral transplantation, Martin Birchall and George Hamilton of University College London wrote in The Lancet.

To start the procedure, doctors took a three-inch section of a cadaver groin vein and stripped it of all living cells, leaving only an inert protein structure. The team then injected it with blood-forming stem cells taken from the girls bone marrow. After growing the vein for two weeks in an incubator, the stem cells had multiplied and converted into vein wall cells, to create a biologically-engineered replacement. The new vein was then implanted into the patient a year ago.

The new stem-cells derived graft resulted not only in good blood flow rates and normal laboratory test values but also, in strikingly improved quality of life for the patient, the report said.

In noting the success of the transplant, the doctors reported that the patient grew 2 inches and gained 11 pounds over the following year. In addition, her parents said that she was more physically active, had improved articulated speech, and had concentrated better on her studies.

The only major complication was the slight constriction of the vein nine months after the operation, which was corrected in a follow-up procedure. During the course of following up on the operation, scientists found no antibodies for the donor vein in the girls blood. This meant her body was not rejecting the transplant because it was recognized as being made of her own cells.

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Doctors Use Stem Cells To Grow Vein For Young Patient

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

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that the company is presenting at two upcoming conferences: the 2012 Bio International Convention and Clinical Outlooks for Regenerative Medicine meeting, both in Boston, on Tuesday, June 19. The presentations will cover the companys three ongoing clinical trials using human embryonic stem cell-derived retinal pigment epithelial cells to treat macular degeneration, and other programs.

Gary Rabin, chairman and CEO, will present at the 2012 Bio International Convention on Tuesday, June 19 at 8:15 a.m. EDT, at the Boston Convention & Exhibition Center.

Matthew Vincent, Ph.D., director of business development, will present at the Clinical Outlooks for Regenerative Medicine meeting at 9:15 a.m. EDT on the same date, at the Starr Center, Schepens Eye Research Institute, at 185 Cambridge Street in Boston.

Both presentation slide decks will be available on the conference presentations section of the ACT website.

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

Forward-Looking Statements

Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words will, believes, plans, anticipates, expects, estimates, and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the companys periodic reports, including the report on Form 10-K for the year ended December 31, 2011. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Companys clinical trials will be successful.

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Advanced Cell Technology to Present at the 2012 Bio International Convention and the Clinical Outlooks for ...

SARANAC LAKE, N.Y.--(BUSINESS WIRE)--

The Trudeau Institute today announced that faculty member Laura Haynes, Ph.D., was awarded a grant from the National Institutes of Health (NIH) to study how aging impacts the immune systems response to influenza infection and vaccination. Entitled Aging and Immunity to Infections, the award is one of the largest program grants funded this year by the NIHs National Institute on Aging, amounting to more than $9 million over a five-year period. The grant further funds Dr. Haynes breakthrough study regarding age-related decline in immune functionality that was just released online in the scientific journal Aging Cell.

Infectious diseases, such as influenza, lead to increased disease and mortality in the elderly. Annually in the United States, influenza causes 200,000 hospitalizations and 36,000 deaths, 90 percent of which occur in older adults. Additionally, the elderly are more vulnerable to infection because vaccine efficacy is significantly reduced in older populations. While it is well known that the adaptive immune response to influenza infection and immunization declines with aging, the Haynes laboratorys recent studies found that there are additional changes in the immune system that had not been previously appreciated. These newly discovered changes contribute significantly to the age-related decline in immune function.

Earlier research from Dr. Haynes lab has shown that there are age-related declines in the function of immune cells that respond to vaccination. Specifically, immune cells from aged individuals show slower and reduced overall functional responses when compared with cells from younger individuals. They also successfully demonstrated that these changes are intrinsic to the immune cells themselves. That is, even if aged cells are transferred into young hosts, they still exhibit reduced function.

Breakthrough Research in the Immune Response in the Elderly Leads to NIH Grant

Dr. Haynes recent investigation of the factors responsible for the decline in immune response to vaccination in elderly patients has led to a major breakthrough. Their study, led by postdoctoral fellow Julie Lefebvre, was published online on June 11, 2012 in the scientific journal Aging Cell. The study demonstrates that, in addition to intrinsic defects in aged immune cells, there are changes in the aged environment, including secondary lymphoid tissues such as the spleen and lymph nodes. These changes are the result of disorganized expression of molecules that direct immune cell movement. The result is that immune cells in aged individuals are not directed properly, and their response is slower as a result.

This revelation helps explain how aging impacts the immune system and the mechanisms involved. Only when the specific defects are determined can strategies be developed to overcome these defects and develop more effective vaccines for the elderly.

Importantly, this study also demonstrates that one of the main factors responsible for this delay is an age-related change in the molecules that direct the movement of immune cells. Small molecules in the immune system, known as chemokines, are responsible for the proper movement of immune cells during a response to vaccination or infection. Proper movement of the cells is essential for a robust immune response since many immune system cells function via direct cell-to-cell contact with each other. Without this contact, the immune response is much less effective. This is the first time this kind of mechanism has been appreciated in aged individuals.

Future Research Focuses on How Aging Impacts Response to Flu and Infection

With the support of the $9 million multi-year grant from the NIH, the Trudeau research will focus on how aging impacts the immune response to influenza infection and vaccination and then importantly move this research to human trials.

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Trudeau Institute announces $9 Million Translational Research Award

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

Seattle Genetics, Inc. (SGEN) today announced updated survival data from a pivotal clinical trial of single-agent ADCETRIS (brentuximab vedotin) in patients with relapsed or refractory Hodgkin lymphoma (HL) after autologous stem cell transplant (ASCT) showing that the median overall survival has not been reached after a 26.5 month median follow-up. The data will be reported during an oral presentation at the 17th European Hematology Association (EHA) Annual Meeting being held June 14-17, 2012 in Amsterdam, Netherlands. ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30.

Heavily pretreated Hodgkin lymphoma patients who relapse following autologous stem cell transplant often have a poor prognosis and there is a high unmet medical need for effective treatment options, said Scott Smith M.D., Ph.D., Loyola University Medical Center. These updated overall survival results from the pivotal trial are encouraging and demonstrate that ADCETRIS may play an important role in the treatment of patients with relapsed or refractory disease.

Long-term Follow-up Results of an Ongoing Pivotal Study of Brentuximab Vedotin in Patients with Relapsed or Refractory Hodgkin Lymphoma

A pivotal trial was conducted in 102 patients with relapsed or refractory HL after ASCT. The primary endpoint was objective response rate (ORR) per independent review. The secondary endpoints were complete remission (CR) rate, duration of response, progression-free survival (PFS), overall survival (OS), and safety and tolerability. At the time of the long-term follow-up analysis, the median observation time from first dose was 26.5months. Data, to be presented by Dr. Smith, include:

Patients received 1.8milligrams per kilogram of ADCETRIS every 3 weeks as a 30-minute outpatient intravenous infusion for up to 16cycles. Patients received a median of nine cycles of ADCETRIS while on trial. The median age of patients in the pivotal trial was 31 years. Enrolled patients had received a median of 3.5 (range 113) prior cancer-related systemic therapies, excluding ASCT. Seventy-one percent of patients had primary refractory disease, defined in the study protocol as patients who relapsed within three months of attaining CR or failed to achieve a CR, and 42 percent had not responded to their most recent prior therapy.

Details of the oral presentation are as follows:

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 received accelerated approval from the U.S. Food and Drug Administration (FDA) 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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Seattle Genetics Highlights Updated Survival Data from ADCETRIS® Pivotal Trial in Patients with Relapsed or Refractory ...

SARATOGA, Calif.--(BUSINESS WIRE)--

The Myelin Repair Foundation (MRF) today announced the achievement of a myelin repair Phase 1 clinical trial for multiple sclerosis earlier than the foundations goal set for 2014. By establishing its Accelerated Research Collaboration (ARC) Model to advance myelin repair treatments forward into clinical trial Phase 1 within a decade, the Myelin Repair Foundation achieved this critical milestone ahead of its goal, validating the efficiency of the ARC model to speed drug development.

This Phase 1 clinical trial conducted at Cleveland Clinic will examine the efficacy of a new myelin repair therapeutic pathway with mesenchymal stem cells (MSCs), based on MRF supported research conducted by MRF Principal Investigator Dr. Robert Miller, Professor of Neurosciences and Vice President for Research & Technology Management at Case Western Reserve University. To date, half of the 24 patients planned for this initial trial have been enrolled.

Scientists hope that one day their research will reach clinical trials, and Im thrilled to achieve this milestone in my career, said Dr. Robert Miller. Without the support of Myelin Repair Foundation funding a critical component of our research that is the basis of this trial, this achievement would not have been possible. Our partnership with the Myelin Repair Foundation has helped identify new pathways to treat disease that reverses damage, ultimately accomplishing so much more than the suppression of MS symptoms.

Funded by the Myelin Repair Foundation, Dr. Millers team of scientists identified an innovative clinical pathway through mesenchymal stem cell signals that not only protect myelin, which is damaged by the autoimmune reaction in MS, but also facilitates myelin repair. Current MS drugs on the market only focus on the suppression of the immune system to protect myelin from future damage; patients have no treatment options available to repair myelin once damage occurs in MS.

Our goal to support research that would enter Phase 1 trials within a decade was deemed nearly impossible, said Scott Johnson, president and CEO of the Myelin Repair Foundation. To think we achieved this ambitious goal even earlier than we planned illustrates the effectiveness of our innovative research model that accelerates promising scientific discoveries into clinical trials. Even with this success, we refuse to rest on our laurels and will continue to progress myelin research into multiple clinical trials. We remain focused on our singular goal: To speed the development of an effective myelin repair treatment to reach patients with multiple sclerosis.

For more information about the clinical trial and enrollment, please visit http://www.clinicaltrials.gov.

About the Myelin Repair Foundation

The Myelin Repair Foundation (MRF) (http://www.myelinrepair.org) is a Silicon Valley-based, non-profit research organization focused on accelerating the discovery and development of myelin repair therapeutics for multiple sclerosis. Its Accelerated Research Collaboration (ARC) model is designed to optimize the entire process of medical research, drug development and the delivery of patient treatments.

About Case Western Reserve University

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The Myelin Repair Foundation Achieves Phase 1 Myelin Repair Clinical Trial

Featured Article Academic Journal Main Category: Bones / Orthopedics Also Included In: Stem Cell Research Article Date: 14 Jun 2012 - 4:00 PDT

Current ratings for: 'Fat Stem Cells Grow Bone Faster And Better'

They write about their work in the 11 June online first issue of a paper published in the new peer-reviewed journal Stem Cells Translational Medicine, which aims to span stem cell research and clinical trials.

The two co-senior authors of the study are Chia Soo, vice chair for research at University of California - Lost Angeles (UCLA) Plastic and Reconstructive Surgery, and Bruno Pault, professor of Orthopedic Surgery at UCLA. Both are members of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

Soo told the press that fat tissue is considered a good source of mesenchymal stem cells, the sort that can be coaxed to form various tissue types such as bone, cartilage and muscle, because there is plenty of it and it is easy to get hold of with procedures like liposuction.

One conventional method of growing these stem cells from fat tissue relies on culturing the fat cells for weeks to isolate the stem cells that form bone. These processes can increase the risk of infection and lead to genetic instability.

Another traditional method, called stromal vascular fraction (SVF), uses fresh, non-cultured cells, but it is not easy to extract SVF cells from fat tissue because there are many kinds of them, not all capabale of forming bone.

For this study, the researchers isolated and purified human perivascular stem cells (hPSC) from fat tissue, and using lab animals, showed these cells are a better option for making bone than SVF cells.

They also showed that a growth factor called NELL-1, speeded up bone formation.

Soo told the press:

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Fat Stem Cells Grow Bone Faster And Better

Stem cells are touted as a future source to replenish damaged tissue Researchers find skeletal muscle stem cells can survive for 17 days in human and 16 days in mice This is some two weeks more than the one or two days currently thought 'Highly promising' research requires more testing and validation before it can be tested in humans

By Graham Smith

PUBLISHED: 07:51 EST, 13 June 2012 | UPDATED: 07:52 EST, 13 June 2012

Some stem cells can lay dormant for more than two weeks in a dead person and then be revived to divide into new, functioning cells, scientists revealed yesterday.

The research unlocks further knowledge about the versatility of these cells, touted as a future source to replenish damaged tissue.

The scientists, led by Fabrice Chretien of the Pasteur Institute in Paris, said in a statement: 'Remarkably, skeletal muscle stem cells can survive for 17 days in humans and 16 days in mice post-mortem, well beyond the one to two days currently thought.'

Breakthrough: A fusion of several stem cells - or a myotube - obtained in vitro from a human muscle collected 17 days after an individual's death

The stem cells retained their ability to differentiate into perfectly functioning muscle cells, they found.

The researchers added: 'This discovery could form the basis of a new source, and more importantly new methods of conservation, for stem cells used to treat a number of pathologies.'

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Could dead bodies soon be harvested for their stem cells? Scientists find 'miracle' cells stay alive for 17 days in ...

(CBS News) A 10-year-old girl made medical history when a vein created from her own stem cells was transplanted into her body to treat a life-threatening blockage.

PICTURES: First lab-grown windpipe saves cancer patient

The girl had a condition called hepatic portal vein obstruction in which there is a blockage in the vein that drains blood from the intestines and spleen to the liver. A blockage here can lead to major complications like bleeding, developmental delays, an enlarged spleen and even death. Typical treatments include removing veins from other parts of the body - such as the leg - and transplanting them elsewhere to restore blood flow, but the procedures can be risky and have had mixed success.

For the new procedure, the girl was admitted to the Sahlgrenska University Hospital in Gothenburg, Sweden, where a team had already taken a 9 centimeter segment of vein from the groin of a deceased donor. The doctors stripped all cells from the vein, leaving just a tube of scaffolding, which was then injected with stem cells obtained from the girl's own bone marrow. After two weeks in a bioreactor, the graft was re-implanted in the 10-year-old girl, and her condition has been improving ever since.

The medical milestone is described in the June 14 issue of the The Lancet.

"The young girl in this report was spared the trauma of having veins harvested from the deep neck or leg with the associated risk of lower limb disorders," and avoided the need for a liver transplant, explained Dr. Martin Birchall, chair of laryngology, and Dr. George Hamilton, professor of vascular surgery, both at the University College London, U.K., in a commentary published in the same issue.

The girl had no complications from the operation and her blood flow was restored immediately.

In the year since the procedure, the girl has grown from about 4 feet 4.5 inches to almost 4 feet 7 inches and her weight increased from 66 pounds to 77 pounds. However over that year her blood flow decreased and the graft narrowed, requiring a second stem cell-based procedure. She has remained well since the second procedure, taking long walks of up to two miles and participating in light gymnastics. Especially noteworthy is her immune system has not attempted to fight off the donor tissue, despite her not taking any immunosuppressive drugs which often carry side effects.

"The new stem-cells derived graft resulted not only in good blood flow rates and normal laboratory test values but also, in strikingly improved quality of life for the patient," wrote the surgeons, led by Dr. Michael Olausson, a profsesory of surgery at the University of Gothenburg in Sweden. They added that their work opens up the possibility of trying to reproduce arteries for surgical use, such as for coronary bypass surgery.

This isn't the first procedure to use a patient's stem cells to create new tissue to save a person's life. HealthPop reported in 2011 of an Eritrean man with late-stage throat cancer who received the world's first synthetic windpipe. The organ was grown from the man's stem cells and then applied to a plastic scaffold, eliminating the need for a donor organ.

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10-year-old girl gets new vein made from her own stem cells in medical first

LONDON: Scientists have successfully transplanted a vein made from a 10-year-old girl's own stem cells into her body. It is the first time such an operation has been reported and marks an important step in the practical ability of doctors to use stem cells to grow replacement cells for damaged or diseased tissue.

Writing in the medical journal The Lancet, a team led by Professor Suchitra Sumitran-Holdgersson, of the University of Gothenburg in Sweden, described how the girl had a blocked hepatic portal vein, which takes blood away from the gut and spleen to the liver.

The blockage can lead to complications including internal bleeding, developmental problems and even death. The usual treatment for the condition is to remove the blocked vein and replace it with sections of healthy vein from other parts of the body.

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The team instead grew a vein for the young girl using her own bone marrow stem cells.

They started with a nine-centimetre section of vein taken from the groin of a donor and stripped it of its cells, leaving behind a tubular protein scaffold. This was seeded with the girl's stem cells and the resulting vein was transplanted into the girl.

The procedure restored blood flow out of her liver immediately.

''The patient increased in height from 137 to 143 centimetres and increased in weight from 30 to 35 kilograms in the one year since the first operation,'' the authors wrote. ''Although we undertook no neurocognitive tests, the parents reported that the patient had enhanced physical activity (increased long distance walks of two to three kilometres and light gymnastics) and improved articulated speech and concentration power in school activities.''

Nine months after the operation, the vein had constricted slightly in size and this was corrected in a follow-up procedure. Most significantly, scientists found no antibodies for the donor vein in the girl's blood. Her body was not rejecting the transplant because it was recognised as being made of her own cells.

''The young girl in this report was spared the trauma of having veins harvested from the deep neck or leg with the associated risk of lower limb disorders, and avoided the need for a liver or multivisceral transplantation,'' Professors Martin Birchall and George Hamilton of University College London wrote in an accompanying commentary article in The Lancet.

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Girl gets vein grown from her own stem cells for transplant

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Posted June 14, 2012

COLUMBIA, Md. --Osiris Therapeutics, Inc.(NASDAQ: OSIR) has received consent from New Zealand to market its first-in-class stem cell therapy Prochymal (remestemcel-L), for the treatment of acute graft-vs-host disease (GvHD) in children. New Zealand joins Canada, which last month became the world's first internationally recognized regulatory authority to grant approval to a stem cell drug. Prochymal is also the first therapy approved for GvHD - a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected, many within just weeks of diagnosis.

"With each of our approvals it becomes clearer that the time for life-saving stem cell therapies in the practice of medicine has arrived, and we are humbled to have a leading role," saidC. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris. "I would like to thank the professionals at Medsafe for their thoughtful and expeditious review of this complex application. I would also like to thank the team at Osiris that continues to do an outstanding job of making Prochymal available to children around the world suffering from the devastating effects of GvHD."

Osiris submitted a New Medicine Application to Medsafe (New Zealand's medical regulatory agency) in May of 2011, and was granted Priority Review in June of 2011. Priority review provides expedited review for new drugs which offer a significant clinical advantage over current treatment options. Prochymal was granted provisional consent under Section 23 of the Medicines Act 1981.

"The incidence of GvHD is likely to rise as the demographic profile of our transplant population evolves," saidHans Klingemann,M.D., Ph.D., a Professor of Medicine and the Director of the Bone Marrow & Hematopoietic Stem Cell Transplant Program at Tufts University School of Medicine. "Effective strategies to manage the often lethal consequences of GvHD reduce the overall risk to transplantation and provide the transplant physician with better options when approaching their most difficult cases."

Clinical trials have shown that Prochymal is able to induce an objective, clinically meaningful response in 61-64 percent of children with GvHD that is otherwise refractory to treatment. Furthermore, treatment response with Prochymal resulted in a statistically significant improvement in survival.

"As a mother who watched my son Christian suffer and die from the horrifying effects of GvHD, while waiting for the regulatory approvals necessary to allow him access to Prochymal, words cannot express how happy I am that significant progress is finally being made," saidSandy Barker,President and Co-founder of the Gold Rush Cure Foundation. "We are proud to stand side-by-side with Osiris in this historic battle for our children around the world. Our motto is 'not one more child, not one more family' and when it comes to GvHD mortality, zero is the only acceptable number."

Prochymal is now approved in Canada and New Zealand, and is currently available in seven other countries including the United States under an Expanded Access Program (EAP). It is expected that Prochymal will be commercially available in New Zealand later this year.

Osiris Therapeutics, Inc. is a stem cell company, having developed the world's first approved stem cell drug, Prochymal. The company is focused on developing and marketing products to treat medical conditions in inflammatory, cardiovascular, orthopedic and wound healing markets. Osiris has developed an extensive intellectual property portfolio to protect the company's technology, including 48 U.S. and 144 foreign issued patents.

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Osiris Receives Second Approval for Stem Cell Drug

ReNeuron Group (Berlin: RQE.BE - news) plc

(the "Company")

Interim data from clinical trial of ReNeuron's stem cell therapy for stroke to be presented at leading scientific conference

Data show no safety concerns and evidence of sustained reductions in neurological impairment and spasticity

Guildford, UK, 14 June 2012: ReNeuron Group plc (the "Company") (LSE: RENE.L) is pleased to announce the presentation of interim data from the PISCES (Pilot Investigation of Stem Cells in Stroke) clinical trial of its ReN001 stem cell therapy for disabled stroke patients. In this open label, dose-ranging Phase I safety study, ReNeuron's ReN001 stem cell therapy is being administered in ascending doses to a total of 12 stroke patients who have been left disabled by an ischaemic stroke, the most common form of the condition.

The primary aim of the study is to test the safety and tolerability of the treatment in ascending doses of the ReN001 cells, in patients with moderate to severe functional neurological impairments resulting from their stroke. The secondary aim of the study is to evaluate efficacy measures for the design of future clinical trials with ReN001, including imaging measures as well as a number of tests of sensory, motor and cognitive functions.

To date, six patients have been treated in the PISCES stroke study, representing the first two of four dose cohorts. The interim data being presented are from the first five patients treated, at 2 x 12 month, 1 x six month and 2 x three month follow-up points.

No cell-related adverse events or adverse immune-related responses were reported in any of the patients treated to date. A number of the patients experienced minor procedure-related adverse events such as asymptomatic bleeds or superficial scalp infections at the implantation wound site.

Reductions in neurological impairment and spasticity were observed in all five patients compared with their stable pre-treatment baseline performance and these improvements were sustained in longer term follow-up.

Neurological deficits were measured using the National Institutes of Health Stroke Scale (NIHSS), a higher score representing a worse deficit. Patients are required to have a NIHSS score of at least 6 to participate in the study. The pre-treatment median score for the first five patients was 8 (range 6 to 10) and the three month post-treatment median score was 4 (range 3 to 9).

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ReNeuron Group plc - PISCES Trial Interim Data

14 June 2012 Last updated at 07:25 ET By Eleanor Bradford BBC Scotland Health Correspondent

The first patients to take part in a clinical trial of a stem cell treatment for stroke have seen reductions in their disability, according to doctors.

Six patients in the west of Scotland had human stem cells inserted close to the damaged part of their brain.

After receiving the treatment, they saw improvements in the limb weakness they suffered as a result of their stroke.

Howeve, doctors have cautioned against reading too much into the early results of the clinical trial.

It is the world's first trial of a neural stem cell therapy for stroke.

Stroke is the third largest cause of death and the single largest cause of adult disability in the developed world.

The trial is being conducted at the Institute of Neurological Sciences at the Southern General Hospital in Glasgow, and is being led by Glasgow University neurologist Professor Keith Muir.

He said: "So far we've seen no evidence of any harmful effects. We're dealing with a group of people a long time after a stroke with significant disability and we don't really expect these patients to show any change over time.

"So it's interesting to see that in all the patients so far they have improved slightly over the course of their involvement in the study."

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Stem cells 'help' stroke patients

COLUMBIA, Md.--(BUSINESS WIRE)--

Osiris Therapeutics, Inc. (OSIR), announced today it has received consent from New Zealand to market its first-in-class stem cell therapy Prochymal (remestemcel-L), for the treatment of acute graft-vs-host disease (GvHD) in children. With this decision New Zealand joins Canada, which last month became the worlds first internationally recognized regulatory authority to grant approval to a stem cell drug. Prochymal is also the first therapy approved for GvHD - a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected, many within just weeks of diagnosis.

"With each of our approvals it becomes clearer that the time for life-saving stem cell therapies in the practice of medicine has arrived, and we are humbled to have a leading role, said C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris. I would like to thank the professionals at Medsafe for their thoughtful and expeditious review of this complex application. I would also like to thank the team at Osiris that continues to do an outstanding job of making Prochymal available to children around the world suffering from the devastating effects of GvHD."

Osiris submitted a New Medicine Application (NMA) to Medsafe(New Zealand's medical regulatory agency) in May of 2011, and was granted Priority Review in June of 2011. Priority review provides expedited review for new drugs which offer a significant clinical advantage over current treatment options. Prochymal was granted provisional consent under Section 23 of the Medicines Act 1981.

"The incidence of GvHD is likely to rise as the demographic profile of our transplant population evolves," said Hans Klingemann, M.D., Ph.D., a Professor of Medicine and the Director of the Bone Marrow & Hematopoietic Stem Cell Transplant Program at Tufts University School of Medicine. "Effective strategies to manage the often lethal consequences of GvHD reduce the overall risk to transplantation and provide the transplant physician with better options when approaching their most difficult cases.

Clinical trials have shown that Prochymal is able to induce an objective, clinically meaningful response in 61-64 percent of children with GvHD that is otherwise refractory to treatment. Furthermore, treatment response with Prochymal resulted in a statistically significant improvement in survival.

As a mother who watched my son Christian suffer and die from the horrifying effects of GvHD, while waiting for the regulatory approvals necessary to allow him access to Prochymal, words cannot express how happy I am that significant progress is finally being made, said Sandy Barker, President and Co-founder of the Gold Rush Cure Foundation. We are proud to stand side-by-side with Osiris in this historic battle for our children around the world. Our motto is 'not one more child, not one more family' and when it comes to GvHD mortality, zero is the only acceptable number.

Prochymal is now approved in Canada and New Zealand, and is currently available in seven other countries including the United States under an Expanded Access Program (EAP). It is expected that Prochymal will be commercially available in New Zealand later this year.

About GvHD

GvHD represents a major unmet medical need with no approved treatment until Prochymal. GvHD is the leading cause of transplant related mortality, in which immune cells contained within the transplanted marrow recognize the recipient as foreign and mount an immunologic attack. Severe GvHD can cause blistering of the skin, intestinal hemorrhage and liver failure. Severe GvHD is extremely painful and fatal in up to 80 percent of cases. Currently, steroids are used as first-line therapy with a success rate of only 30-50 percent. When steroids fail, treatment options are limited to immunosuppressive agents used off-label with little benefit and significant toxicities.

The rest is here:
Osiris Receives Second Approval for Life-Saving Stem Cell Drug; Prochymal Granted Marketing Consent by New Zealand

June 14, 2012, SEATTLE /PRNewswire/ -- Cell Therapeutics, Inc. ("CTI") (NASDAQ and MTA: CTIC), a company focused on translating science into novel cancer therapies, today announced that former OncoMed Pharmaceuticals executive, Steven E. Benner, M.D., M.H.S., has joined CTI as Executive Vice President and Chief Medical Officer ("CMO"), reporting to James A. Bianco, M.D., Chief Executive Officer. Dr. Benner will take over all drug development activities at the company.Dr. Benner was previously senior vice president and chief medical officer at OncoMed, a venture-backed biotechnology company focused on the development of cancer stem cell targeting agents. Prior to OncoMed, he was CMO at Protein Design Labs ("PDL"), where he was accountable for all development activities including clinical development, clinical operations, biometry, regulatory affairs, and safety. He also served as Chair of the Portfolio and Clinical Development Management Committees of PDL. Before PDL he held several senior executive roles at Bristol-Myers Squibb in global development, life cycle management, and licensing and alliances.

"Dr. Benner brings to CTI his proven track record of success in advancing the development of innovative therapies for cancer patients," said Dr. Bianco. "His appointment is the first step in re-aligning our portfolio efforts, as we focus on advancing pacritinib into Phase III pivotal studies later this year."

With the new company initiative of the planned Pixuvri launch in Europe later this year, Jack W. Singer, M.D., will assume the newly-created role of Executive Vice President ("EVP") of Global Medical Affairs and Translational Medicine, responsible for cancer drug development strategy, global medical affairs, and life cycle management.

"Given Jack's impressive academic credentials, the respect he receives from an international network of key opinion leaders in the field, and his track record in oncology drug development, this was a natural promotion as we introduce Pixuvri in Europe," said Dr. Bianco.

"CTI has assembled an impressive late-stage portfolio of novel targeted therapies that address a spectrum of blood related cancers," said Dr. Benner. "With two drugs in Phase III and two more expected to enter Phase III trials within a year, this is an exciting and transformational time to join the team at CTI."

About Pixuvri (pixantrone)Pixuvri is a novel aza-anthracenedione with unique structural and physio-chemical properties. Unlike related compounds,Pixuvri forms stable DNA adducts and in preclinical models has superior anti-lymphoma activity compared to related compounds. Pixuvri was structurally designed so that it cannot bind iron and perpetuate oxygen radical production or form a long-lived hydroxyl metabolite -- both of which are the putative mechanisms for anthracycline induced acute and chronic cardiotoxicity. These novel pharmacologic properties allow Pixuvri to be administered to patients with near maximal lifetime exposure to anthracyclines without unacceptable rates of cardiotoxicity, and, because Pixuvri is not a vesicant, allow it to be safely delivered via a peripheral intravenous catheter.

In May 2012 Pixuvri received conditional marketing authorization in the EU as monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive NHL. The benefit of pixantrone treatment has not been established in patients when used as fifth line or greater chemotherapy in patients who are refractory to last therapy.The Summary of Product Characteristics ("SmPC") has the full prescribing information, including the safety and efficacy profile of Pixuvri in the approved indication. The SmPC is available at http://ec.europa.eu/health/documents/communityregister/html/h764.htm#ProcList.

Pixuvri is currently available in the EU through Named Patient Programs.

Pixuvri does not have marketing approval in the United States.

About Conditional Marketing AuthorizationSimilar to accelerated approval regulations inthe United States, conditional marketing authorizations are granted in the EU to medicinal products with a positive benefit/risk assessmentthat address unmet medical needs and whose availability would result in a significant public health benefit. A conditional marketing authorization is renewable annually. Under the provisions of the conditional marketing authorization for Pixuvri, CTI will be required to complete a post-marketing study aimed at confirming the clinical benefit previously observed.

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Cell Therapeutics Appoints New Chief Medical Officer

By Makiko Kitamura - 2012-06-13T22:30:00Z

The first vein grown from a patients own stem cells was successfully transplanted into a 10-year-old girl, potentially offering a way for those lacking healthy veins to undergo dialysis or heart bypass surgery.

A team led by Michael Olausson of the University of Gothenburg took a 9-centimeter (3.5-inch) segment of vein from a human donor and removed all living cells, the Swedish researchers wrote in a study in The Lancet medical journal today. The resulting protein scaffolding was injected with stem cells from the girls bone marrow, and two weeks later was implanted in the patient, who had a blockage in the vein that carries blood from the spleen and intestines to the liver.

The result points to what may be a safer source of stem cells, the building blocks of life which can grow into any type of tissue in the body. Using cells from the patient may limit the risk that the immune system would attack the transplant, which can occur with tissue taken from healthy people and given to the sick. The girl hasnt developed signs of rejection, even without taking drugs to suppress her immune system, the researchers said.

The successful procedure establishes the feasibility and safety of a novel paradigm for treatment, the researchers wrote in the study. Our work opens interesting new areas of research, including trying to reproduce arteries for surgical use in patients.

The recipient had no complications from the operation, and a year later, has grown 6 centimeters and gained 5 kilograms (11 pounds) in weight.

Olausson and colleagues report suggests that tissue- engineered vascular grafts are promising, but one-off experiences such as the procedure they describe need to be converted into full clinical trials in key target populations, Martin Birchall and George Hamilton, professors at the University College London, wrote in a commentary accompanying the Lancet publication.

The study was funded by the Swedish government.

To contact the reporter on this story: Makiko Kitamura in London at mkitamura1@bloomberg.net

To contact the editor responsible for this story: Phil Serafino at pserafino@bloomberg.net

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First Vein Grown From Human Stem Cells Transplanted