Stem Cell Clinic

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

Charmaine Chan and Rocio Ochoa – High School Stem Cell Research Interns Summer 2013, Part 2 – Video

Charmaine Chan and Rocio Ochoa - High School Stem Cell Research Interns Summer 2013, Part 2
Charmaine and Rocio are high school students doing stem cell research internships this summer. Their internships were funded by California #39;s Stem Cell Agency...

By: California Institute for Regenerative Medicine

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Charmaine Chan and Rocio Ochoa - High School Stem Cell Research Interns Summer 2013, Part 2 - Video

Stem cell research reveals clues to brain disease

The development of new drugs for improving treatment of Alzheimers and Parkinsons disease is a step closer after recent research into how stem cells migrate and form circuits in the brain.

The results from a study by researchers at The University of Aucklands Centre for Brain Research may hold important clues into why there is less plasticity in brains affected by Parkinsons and Alzheimers disease, and links to insulin resistance and diabetes.

The major five-year project to understand how stem cells start and stop migrating in the brain has also helped to unlock the secrets of how stem cells migrate during development and in adulthood.

The study revealed new information on how connectivity between brain cells is improved or worsened, says senior study author, Dr Maurice Curtis who conceived and directed the research. The experiments were carried out at the Centre for Brain Research laboratories by Dr Hector Monzo. Collaborators included a director of the CBR, Distinguished Professor Richard Faull, Dr Thomas Park, Dr Birger Dieriks, Deidre Jansson and Professor Mike Dragunow.

We have begun testing new novel drug compounds that target how polysialic acid is removed from the cell in the hope of improving neuron connectivity, says Dr Curtis.

He explains that stem cells in the brain are immature brain cells that must migrate from their birthplace to a position in the brain where they will connect with other brain cells, turn into adult brain cells (neurons) and become part of the brains circuitry.

Even once the neuron has found its location, the neurons tentacles (or dendrites) need to forage to find other neurons to connect with to form circuits. This would be easy except that in the adult brain the cells are surrounded by a fairly rigid matrix (extracellular matrix) and so migration or foraging becomes almost impossible in this high friction environment.

The way the cell overcomes this friction is by placing large amounts of a special slippery molecule called polysialic acid-neural cell adhesion molecule onto the cell surface, says Dr Curtis. This allows the cell to migrate or forage with only a fraction of the friction it once had and this also reduces the energy requirements of the cell.

Once the cell has migrated to its destination, the slippery coating is removed and the cell becomes locked in place ready to connect with other cells. In the case of the dendritic foraging, the polysialic acid must be removed in order for the dendrite to connect with another cell (synapse formation).

We have known for at least 20 years that this process occurs but despite extensive studies by a number of groups internationally we have been in the dark about what controls this process, he says. Studies in my laboratory have demonstrated what happens to the slippery molecules once the cell no longer needs them.

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Stem cell research reveals clues to brain disease

Stem cell advance in mice boosts prospects for retina treatment

PARIS: Blind mice have been able to see once more in a laboratory exploit that marks a further boost for the fast-moving field of retinal therapy, according to a study published on Sunday.

Scientists in Britain used stem cells -- early-stage, highly versatile cells -- taken from mice embryos, and cultured them in a lab dish so that they differentiated into immature photoreceptors, the light-catching cells in the retina.

Around 200,000 of these cells were then injected into the mice's retinas, some of which integrated smoothly with local cells to restore sight.

The rodents were put through their paces in a water maze and examined by optometry to confirm that they responded to light.

Embryonic stem cells "could in future provide a potentially unlimited supply of health photoreceptors for retinal transplantations to treat blindness in humans," Britain's Medical Research Council (MRC) said in a press release.

Photoreceptor loss lies behind degenerative eye diseases such as retinitis pigmentosa and age-related macular degeneration, also called AMD.

Stem cells have triggered a huge interest and investment on the back of hopes that they can become replacement tissue, grown in a lab dish, for cells damaged by disease or accident.

But the exciting field has to overcome big obstacles.

One is the ability to coax these immature cells into safely becoming the specialised cells that are needed, rather than turn cancerous.

This is where the new work marks a gain, according to lead researcher Robin Ali at the University College London Institute of Ophthalmology and Moorfields Eye Hospital.

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Stem cell advance in mice boosts prospects for retina treatment