Stem Cell Clinical Trials Expert to Lead Cardiology Division at Cedars-Sinai Heart Institute

Newswise LOS ANGELES (July 24, 2013) Timothy D. Henry, MD, an expert known for his innovative work in developing stem cell treatments for advanced heart disease patients, has joined the Cedars-Sinai Heart Institute as director of Cardiology.

Henry comes to Cedars-Sinai from Minneapolis, where he was director of research for the Minneapolis Heart Institute Foundation and professor of medicine at the University of Minnesota School of Medicine.

As Henry begins his tenure, Prediman K. Shah, MD, the renowned cardiologist who has led the Division of Cardiology for nearly 20 years, is stepping down to focus on patient care and on basic and clinical research, including his quest to develop a vaccine and mutant-gene-based therapies that would thwart heart attacks by preventing and possibly reversing arterial cholesterol buildup. Shah, the Shapell and Webb Family Chair in Clinical Cardiology, director of Oppenheimer Atherosclerosis Research Center and director of the Atherosclerosis Prevention and Treatment Center, also will continue treating patients and teaching medical residents and cardiology trainees.

Dr. Shah has been a wonderful colleague and a highly respected national leader, accelerating the pre-eminence of our Cardiology Division, and Dr. Henry will be starting his new position from a robust platform built over many decades of outstanding service, said Shlomo Melmed, MD, senior vice president of Academic Affairs, dean of the Cedars-Sinai medical faculty and the Helene A. and Philip E. Hixon Chair in Investigative Medicine. We are delighted that Dr. Shah will be remaining on as a senior cardiologist in our Heart Institute.

This is a milestone moment, said Eduardo Marbn, MD, PhD, director of the Cedars-Sinai Heart Institute. At the same time we celebrate Dr. Shahs achievements and his valuable contributions to the Heart Institute and to overall heart health, we are welcoming Dr. Henry and beginning a new era during which we foresee the next wave of leading-edge stem cell clinical trials for heart disease patients.

For several years, Henry has been collaborating with Marbn on stem cell clinical trials. The first such collaboration follows upon work at Cedars-Sinai in which heart attack patients received infusions of cardiac stem cells. This study, published in February 2012 in The Lancet, showed that patients who underwent the stem cell procedure experienced a significant reduction in the size of the scar left behind by a heart attack. Patients also experienced a sizable increase in healthy heart muscle following the experimental stem cell treatments.

The new study is being directed by Henry and by Raj Makkar, MD, director of Interventional Cardiology and associate director of the Cedars-Sinai Heart Institute. The national trial called ALLSTAR uses heart cells from unrelated donors in an effort to reverse lasting tissue damage after a heart attack.

My goal in joining the Cedars-Sinai Heart Institute is to help make stem cells a regular treatment option for heart disease, Henry said. Right now, many patients with advanced heart disease have limited treatment options. Stem cells are the next frontier.

A native of Mohall, N.D., Henry earned his bachelors degree at the University of North Dakota and his medical degree at the University of California, San Francisco. Following an internship, residency and cardiology research fellowship at University of Colorado Health Sciences Center in Denver, Henry moved to the University of Minnesota and the Minneapolis Heart Institute, where he has focused on interventional cardiology, treatment of patients with acute heart attacks and novel therapy for patients with advanced heart disease who are not candidates for bypass surgery or stenting.

He has authored hundreds of studies published in peer-reviewed medical journals. He currently serves on the Advisory Committee for the AHA Mission: Lifeline Program, the AHA Acute Cardiac Care Committee of the Council on Clinical Cardiology and the Board of the Society of Chest Pain Centers. He has served as national principal investigator of multiple large, multicenter trials in acute coronary syndromes, myocardial infarction and angiogenesis including several ongoing cardiovascular stem cell trials, including RENEW, ALLSTAR and ATHENA. He is also principal investigator for one of seven NIH Clinical Cardiovascular Stem Cell Centers. As a visiting professor, Henry has lectured throughout the world on systems of care for acute myocardial infarction, refractory angina and stem cell therapy for cardiovascular disease. The process to grow cardiac-derived stem cells was developed by Dr. Marbn when he was on the faculty of Johns Hopkins University. Johns Hopkins has filed for a patent on that intellectual property and has licensed it to Capricor, a company in which Cedars-Sinai and Marbn have a financial interest. Capricor is providing funds for the ALLSTAR clinical study at Cedars-Sinai.

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Stem Cell Clinical Trials Expert to Lead Cardiology Division at Cedars-Sinai Heart Institute

Stem cell transplant gives hope to leukemia patient

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On Thursday night, the 39-year-old woman from Newfoundland will undergo a stem cell transplant from an anonymous donor in Halifax. Julia Wong/Global News

HALIFAX Janice Davidson is getting a new lease on life.

On Thursday night, the 39-year-old leukemia patient will undergo a stem cell transplant from an anonymous donor in Halifax.

You cant believe there is someone out there in the world who you dont know who is willing to give you a gift that is like no other really, the mother of three said.

Davidson was diagnosed with leukemia five years ago. Since then, she has tried several different treatment options and relapsed twice. A stem cell transplant is now her best option to beat the disease.

Its not going to be an easy path but we know were on the right path, she said. We know what we need to do and we are very hopeful for the future.

The latest figures from One Match, the national program for blood stem cell donors and recipients, show that there are about 750 Canadians waiting to receive a stem cell transplant. Eight of those are in Nova Scotia.

Factors such as genetics, ethnic background and treatment affect wait time in receiving a transplant so Davidson is extremely thankful that she found a match. It is especially fortunate because her three siblings were not matches and there are only 317,810 donors registered in Canada. Of those, 10,337 live in Nova Scotia.

Husband Ian says he cannot express enough how thankful he is to the donor, adding that he or she is now like another member of their family.

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Stem cell transplant gives hope to leukemia patient

New stem cell gene therapy gives hope to prevent inherited neurological disease

Public release date: 24-Jul-2013 [ | E-mail | Share ]

Contact: Alison Barbuti alison.barbuti@manchester.ac.uk 44-016-127-58383 University of Manchester

Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time.

The method was used to treat Sanfilippo a fatal inherited condition which causes progressive dementia in children but could also benefit several neurological, genetic diseases.

Researchers behind the study, published in the journal Molecular Therapy this month, are now hoping to bring a treatment to trial in patients within two years.

Sanfilippo, a currently untreatable mucopolysaccharide (MPS) disease, affects one in 89,000 children in the United Kingdom, with sufferers usually dying in their mid-twenties. It is caused by the lack of SGSH enzyme in the body which helps to breakdown and recycle long chain sugars, such as heparan sulphate (HS). Children with the condition build up and store excess HS throughout their body from birth which affects their brain and results in progressive dementia and hyperactivity, followed by losing the ability to walk and swallow.

Dr Brian Bigger, from the University of Manchester's Institute of Human Development who led the research, said bone marrow transplants had been used to correct similar HS storage diseases, such as Hurler syndrome, by transplanting normal cells with the missing enzyme but the technique did not work with Sanfilippo disease. This is because monocytes, a type of white blood cell, from the bone marrow, did not produce enough enzyme to correct the levels in the brain.

Dr Bigger said: "To increase SGSH enzyme from bone marrow transplants, and to target it to the cells that traffic into the brain, we have developed a stem cell gene therapy which overproduces the SGSH enzyme specifically in bone marrow white blood cells.

"We have shown that mice treated by this method produce five times the normal SGSH enzyme levels in the bone marrow and 11 per cent of normal levels in the brain.

"The enzyme is taken up by affected brain cells and is enough to correct brain HS storage and neuro inflammation to near normal levels and completely corrects the hyperactive behaviour in mice with Sanfilippo.

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New stem cell gene therapy gives hope to prevent inherited neurological disease

Stem cell retina therapy treatment might provide miraculous 'cure' for blindness

The three blind mice might get the chance to see because of groundbreaking stem cell research being done in Britain.

Scientists in the UK took stem cells from mice embryos, put them in a petri dish and coaxed them into becoming photoreceptors, the cells in the retina that catch light.

After collecting 200,000 of the stem cells turned photoreceptors the scientists then injected the cells into the eyes of blind mice, and some of the cells integrated into the host retina and restored sight.

The rodents were then run through a maze and examined by optometry to confirm that they did indeed respond to light.

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The groundbreaking work was published by Britain's Medical Research Council and in the science journal "Nature Biotechnology."

While the work is still years away from helping humans, it is an extremely promising start to curing blindness caused by photoreceptor loss like retinitis pigmentosa and age-related macular degeneration.

The biggest breakthrough that the team in England was able to achieve was turning the finicky stem cells into stable photoreceptors, instead of deadly cancer cells.

Additionally, researchers in the past could turn stem cells into rod cells which are a component of the complex network of nerves in the retina but now they are able to create photoreceptors that comprise all the different nerves needed for sight.

"Over recent years, scientists have become pretty good at working with stem cells and coaxing them to develop into different types of adult cells and tissues," said lead researcher Robin Ali at the University College London Institute of Ophthalmology and Moorfields Eye Hospital.

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Stem cell retina therapy treatment might provide miraculous 'cure' for blindness

Stem Cell Therapeutics Provides Corporate Update

TORONTO, ONTARIO--(Marketwired - Jul 25, 2013) - Stem Cell Therapeutics Corp. (TSX VENTURE:SSS)(SCTPF), a biopharmaceutical company developing cancer stem cell-related therapeutics, today provided the following corporate update:

About Stem Cell Therapeutics:

Stem Cell Therapeutics Corp. (SCT) is a biopharmaceutical company dedicated to advancing cancer stem cell discoveries into novel and innovative cancer therapies. Building on over half a century of leading and groundbreaking Canadian stem cell research, the company is supported by established links to a group of Toronto academic research institutes and cancer treatment centers, representing one of the world's most acclaimed cancer research hubs. SCT's clinical stage programs include the recently in-licensed program focused on the structure of tigecycline, which is currently being evaluated in a multi-centre Phase I study in patients with acute myeloid leukemia (AML), as well as TTI-1612, a non-stem cell asset being tested in a 28-patient Phase I trial in interstitial cystitis ("IC") patients, which is near completion. The Company also has two premier preclinical programs, SIRPaFc and a CD200 monoclonal antibody (mAb), which target two key immunoregulatory pathways that tumor cells exploit to evade the host immune system. SIRPaFc is an antibody-like fusion protein that blocks the activity of CD47, a molecule that is upregulated on cancer stem cells in AML and several other tumors. The CD200 mAb is a fully human monoclonal antibody that blocks the activity of CD200, an immunosuppressive molecule that is overexpressed by many hematopoietic and solid tumors. For more information, visit http://www.stemcellthera.com.

Caution Regarding Forward-Looking Information:

This press release may contain forward-looking statements, which reflect SCT's current expectation regarding future events. These forward-looking statements involve risks and uncertainties that may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include changing market conditions; the successful and timely completion of pre-clinical and clinical studies; the establishment of corporate alliances; the impact of competitive products and pricing; new product development risks; uncertainties related to the regulatory approval process or the ability to obtain drug product in sufficient quantity or at standards acceptable to health regulatory authorities to complete clinical trials or to meet commercial demand; and other risks detailed from time to time in SCT's ongoing quarterly and annual reporting. Except as required by applicable securities laws, SCT undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

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Stem Cell Therapeutics Provides Corporate Update

Stem Cell Therapeutics Appoints ProActive Capital Group as Capital Markets & Digital Media Advisor

TORONTO, ONTARIO--(Marketwired - Jul 23, 2013) - Stem Cell Therapeutics Corp. (TSX VENTURE:SSS)(SCTPF), a biopharmaceutical company developing cancer stem cell-related therapeutics, today announced the appointment of ProActive Capital Group as its new Capital Markets Advisory and Digital Media Strategies firm.

"We are very pleased to have retained the services of ProActive Capital Group," stated Stem Cell Therapeutics' Chief Executive Officer, Dr. Niclas Stiernholm. "They are an extremely focused firm with an excellent reputation in the industry and we are excited to adopt an effective capital markets and digital media program. With upcoming key milestones quickly approaching, we feel that now is the opportune time to embark on a significant new and expanded program so that we can achieve a fair valuation that clearly reflects the intrinsic value of our scientific programs."

"We are delighted to have been selected by Stem Cell Therapeutics, a leading company in the cancer stem cell space," stated Jeff Ramson, Founder and CEO of ProActive Capital Group. "We look forward to developing and implementing a quality capital markets and digital media strategy to increase SCT's visibility among the investment community. We strongly believe that the company has significant prospects as it executes its growth strategy."

The Company also announced that the board of directors has approved the adoption of a Deferred Share Unit plan (the "DSU Plan") subject to the approval of the shareholders of the Company and acceptance by the TSX Venture Exchange. The DSU Plan provides an alternative non-cash form of compensation to satisfy annual and special bonuses payable to directors and executive officers, and to satisfy fees that may be payable to directors for acting as directors of the Company. This form of compensation promotes a greater alignment of interests amongst directors and executive officers and the Company's shareholders since DSU Units are ultimately exchanged for common shares of the Company. The maximum number of common shares that may be reserved for issuance under the DSU Plan is set at 2,000,000 common shares.

About Stem Cell Therapeutics:

Stem Cell Therapeutics Corp. (SCT), a Toronto-based biopharmaceutical company, is Canada's only public company dedicated to advancing cancer stem cell discoveries into novel and innovative cancer therapies. Building on over half a century of leading and groundbreaking Canadian stem cell research, the company is supported by established links to a group of Toronto academic research institutes and cancer treatment centers, representing one of the world's most acclaimed oncology research hubs. SCT's clinical stage programs include the recently in-licensed tigecycline program, which is currently being evaluated in a multi-centre Phase I study in patients with Acute Myeloid Leukemia (AML), as well as TTI-1612, a non-stem cell asset that recently completed a 28-patient Phase I trial in interstitial cystitis ("IC") patients. The Company also has two premier preclinical programs, SIRPaFc and a CD200 monoclonal antibody (mAb), which target two key immunoregulatory pathways that tumor cells exploit to evade the host immune system. SIRPaFc is an antibody-like fusion protein that blocks the activity of CD47, a molecule that is upregulated on cancer stem cells in AML and several other tumors. The CD200 mAb is a fully human monoclonal antibody that blocks the activity of CD200, an immunosuppressive molecule that is overexpressed by many hematopoietic and solid tumors. For more information, visit: http://www.stemcellthera.com

Caution Regarding Forward-Looking Information:

This press release may contain forward-looking statements, which reflect SCT's current expectation regarding future events. These forward-looking statements involve risks and uncertainties that may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include changing market conditions; the successful and timely completion of pre-clinical and clinical studies; the establishment of corporate alliances; the impact of competitive products and pricing; new product development risks; uncertainties related to the regulatory approval process or the ability to obtain drug product in sufficient quantity or at standards acceptable to health regulatory authorities to complete clinical trials or to meet commercial demand; and other risks detailed from time to time in SCT's ongoing quarterly and annual reporting. Except as required by applicable securities laws, SCT undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

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Stem Cell Therapeutics Appoints ProActive Capital Group as Capital Markets & Digital Media Advisor

Verastem Receives Orphan Drug Designation from the U.S. FDA for Defactinib in Mesothelioma

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

Verastem, Inc., (VSTM) focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, announced that lead cancer stem cell inhibitor, VS-6063 (defactinib), has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for use in the treatment of mesothelioma, a rare form of lung cancer. The designation is designed to encourage the development of drugs which may provide significant benefit to patients suffering from rare diseases.

Mesothelioma is among the most aggressive and lethal cancers but has limited treatment options, said Robert Forrester, Verastem President and Chief Executive Officer. We are pleased that the FDA recognizes the significant unmet medical need in mesothelioma. We previously received orphan medicinal product status for defactinib in Europe and these two designations are an important component of our development strategy.

Verastem recently outlined details of the registration-directed clinical study of defactinib in patients with malignant pleural mesothelioma. This study is designed as a double-blind, placebo-controlled trial with an expected enrollment of approximately 350-400 patients at clinical sites in 11 countries.

We are in discussions with the regulatory agencies and clinical investigators worldwide, said Dr. Joanna Horobin, Verastem Chief Medical Officer. We recently held our investigator meetings for the physicians conducting the trial in the US and Australia and we are on track to begin enrolling patients in the third quarter. We plan to open sites worldwide on a rolling basis as we clear regulatory and clinical review in each country.

Orphan drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design, and an exemption from FDA user fees.

Cancer stem cells play a central role in treatment resistance in many types of cancers, said Christoph Westphal, M.D., Ph.D., Verastem Executive Chairman. We believe new treatment options targeting cancer stem cells will be critical to achieve a durable clinical benefit for patients. This designation will provide us with many benefits as we pursue the development of defactinib for the treatment of mesothelioma.

In addition to mesothelioma, Verastem recently announced the completion of the Phase 1 stage and initial data from an ongoing Phase 1/1b study of defactinib in combination with weekly paclitaxel for patients with ovarian cancer. Verastem also expects to initiate a Phase 1 study in Japan, and a Phase 2 trial in KRAS-mutated Non-Small Cell Lung Cancer, for defactinib during the third quarter of 2013.

About Verastem, Inc.

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

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Verastem Receives Orphan Drug Designation from the U.S. FDA for Defactinib in Mesothelioma

Stem cell research to benefit Irish patients

The project involves researching, manufacturing and transplanting adult, human stem cells

Certain stem cell-based therapies could be available to Irish patients within a couple of years, as part of a new 2.5m research partnership between the Irish Blood Transfusion Service and research centres in Galway, Dublin and Cork.

The project will involve the researching, manufacturing and for the first time transplanting of adult human stem cells for the treatment of arthritis, burns and diabetic foot ulcers.

For decades, blood transfusions have been used as a basic, temporary form of cell therapy.

But time and technology has moved on and now the targeted delivery of cells to injured or diseased tissue is seen as a promising future method of treating a whole variety of illnesses and conditions.

As part of its objective to be involved in research, the Irish Blood Transfusion Service (IBTS) put out a call for ideas for research into cell-based therapies.

The winning proposal came from the Regenerative Medicine Institute (REMEDI) in NUI Galway, supported by the National Adult Stem Cell Transplant Centre in St James's Hospital and the Centre for Research in Vascular Biology in University College Cork.

The IBTS will provide 2.5m over the next five years to the project.

The objective of the collaboration is to build a network of scientists in Ireland to research, manufacture and transplant new cell-based therapies.

The researchers will use adult stem cells from umbilical chords and bone marrow.

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Stem cell research to benefit Irish patients