Stem Cell Clinic

Nerve cells actively repress alternative cell fates | News Center … – Stanford Medical Center Report

Until now, researchers have focused only on identifying these types of single-lineage repressors, said Wernig. The concept of an everything but repressor is entirely new.

In 2010, Wernig showed that it is possible to convert skin cells into functional neurons over the course of three weeks by exposing them to a combination of just three proteins that are typically expressed in neurons. This direct reprogramming bypassed a step called induced pluripotency that many scientists had thought was necessary to transform one cell type into another.

One of the proteins necessary to accomplish the transformation of skin to neurons was Myt1l. But until this study the researchers were unaware precisely how it functioned.

Usually we think in terms about what regulatory programs need to be activated to direct a cell to a specific developmental state, said Wernig. So we were surprised when we took a closer look and saw that Myt1l was actually suppressing the expression of many genes.

These genes, the researchers found, encoded proteins important for the development of lung, heart, liver, cartilage and other types of non-neuronal tissue. Furthermore, two of the proteins, Notch and Wnt, are known to actively block neurogenesis in the developing brain.

Blocking Myt1l expression in the brains of embryonic mice reduced the number of mature neurons that developed in the animals. Furthermore, knocking down Myt1l expression in mature neurons caused them to express lower-than-normal levels of neural-specific genes and to fire less readily in response to an electrical pulse.

Wernig and his colleagues contrasted the effect of Myt1l with that of another protein called Ascl1, which is required to directly reprogram skin fibroblasts into neurons. Ascl1 is known to specifically induce the expression of neuronal genes in the fibroblasts.

Together, these proteins work as a perfect team to funnel a developing cell, or a cell that is being reprogrammed, into the desired cell fate, said Wernig. Its a beautiful scenario that both blocks the fibroblast program and promotes the neuronal program. My gut feeling would be that there are many more master repressors like Myt1l to be found for specific cell types, each of which would block all but one cell fate.

Wernig is a member of Stanfords Cardiovascular Institute, Child Health Research Institute, Cancer Institute, Neurosciences Institute and Bio-X.

Other Stanford co-authors of the paper are postdoctoral scholars Soham Chanda, PhD, Bo Zhou, PhD, Xuecai Ge, PhD, and Philip Brennecke, PhD; graduate students Cheen Ang, Thomas Vierbuchen and Daniel Fuentes; research assistant Sarah Grieder; undergraduate student Brandon Walker; professor of genetics Lars Steinmetz, PhD; and professor of molecular and cellular biology Thomas Sudhof, MD.

The research was supported by the German Research Foundation, the National Institutes of Health (grant R01MH092931), the California Institute for Regenerative Medicine, the New York Stem Cell Foundation, the Howard Hughes Medical Institute, the Swedish Research Council, the Swedish Government Initiative for Strategic Research Institute, the Department of Health and Human Services and Spectrum Child Health.

Stanfords Department of Pathology also supported the work.

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Nerve cells actively repress alternative cell fates | News Center ... - Stanford Medical Center Report

The Alliance for Regenerative Medicine Releases Agenda for Fifth … – Yahoo Finance

WASHINGTON, DC--(Marketwired - Apr 6, 2017) - The Alliance for Regenerative Medicine (ARM) today released the complete agenda for its upcoming fifth annual Cell & Gene Therapy Investor Day, taking place April 27, 2017 in Boston, MA. This event, co-hosted by Piper Jaffray and held in partnership with Cowen and Company is the only investor conference specifically focused on cell and gene therapies, offering exclusive access to the field's most promising companies.

This year's Cell & Gene Therapy Investor Day is expected to attract 350+ attendees, including 175+ active investors and analysts and will feature presentations by 30+ companies, along with panels and fireside chats by the field's foremost thought leaders.

2017 Panel Sessions and Speakers:

Fireside Chat Olivier Danos, Ph.D., Chief Scientific Officer, REGENXBIO (moderator) James M. Wilson, M.D., Ph.D., Rose H. Weiss Professor and Director, Orphan Disease Center; Professor of Medicine and Pediatrics; Director, Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania

Panel I: Cell Therapy Beyond Oncology: Where Does the Greatest Potential Lie? Edward Tenthoff, Managing Director & Senior Research Analyst, Piper Jaffray (moderator) Eduardo Bravo, CEO, TiGenix Adam Gridley, President and CEO, Histogenics Paul Laikind, President and CEO, ViaCyte Chaim Lebovits, President and CEO, BrainStorm Cell Therapeutics Emile Nuwaysir, Ph.D., CEO, BlueRock Therapeutics

Panel II: Gene Therapy: Commercialization Readiness & Market Access Challenges Joshua Schimmer, M.D., Managing Director & Senior Research Analyst, Piper Jaffray (moderator) Faraz Ali, Chief Business Officer, REGENXBIO Sven Kili, M.D., VP and Head of Gene Therapy Development, GlaxoSmithKline Arthur Tzianabos, Ph.D., President and CEO, Homology Medicines Elizabeth White, Ph.D., Assistant VP, Early Commercial Planning, Rare Disease and Gene Therapy, Pfizer Innovative Health

Panel III: Immuno-Oncology: What Are the Key Issues as First Products Approach Commercialization? Timothy Schroeder, CEO, CTI Clinical Trial and Consulting (moderator) Usman Azam, M.D., President and CEO, Tmunity Therapeutics David Epstein, Executive Partner, Flagship Pioneering Rick Fair, President and CEO, Bellicum Pharmaceuticals Jeffrey Walsh, Chief Financial and Strategy Officer, bluebird bio

2017 Presenting Companies:

4D Molecular Therapeutics, Abeona Therapeutics, Adverum Biotechnologies, AGTC, Argos Therapeutics, Audentes Therapeutics, AVROBIO, BioCardia, bluebird bio, Bone Therapeutics, Caladrius Biosciences, Capricor Therapeutics, Celyad, Fate Therapeutics, Fibrocell, GenSight Biologics, Histogenics, Homology Medicines, Juventas Therapeutics, Kiadis Pharma, Lysogene, Mesoblast, MiMedx, Orchard Therapeutics, Oxford BioMedica, Pluristem Therapeutics, Precision Biosciences, ReNeuron, Sangamo Therapeutics, Semma Therapeutics, Synpromics, TiGenix, TxCell, uniQure, Vericel, and Voyager Therapeutics

In addition to the event's co-host Piper Jaffray, sponsors include Cowen and Company; Cognate BioServices; Cryoport; CTI Clinical Trial and Consulting Services; Kawasaki; Lonza; Medpace; PCT, a Caladrius Company; and Edison. For more information please visit the event's website at http://www.arminvestorday.com.

Credentialed investors and life science strategic partners can indicate their interest in attending here. Members of the media interested in attending are asked to please contact Lyndsey Scull at lscull@alliancerm.org.

The event will be held April 27, 2017, beginning at 7:30am at The State Room, 60 State Street, Boston, MA 02109.

About The Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 250 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

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The Alliance for Regenerative Medicine Releases Agenda for Fifth ... - Yahoo Finance

Can Stem Cell ‘Patch’ Help Heart Failure? – Montana Standard

WEDNESDAY, April 5, 2017 (HealthDay News) -- Scientists report another step in the use of stem cells to help treat people with debilitating heart failure.

In an early study of 27 patients, Japanese researchers used patients' own muscle stem cells to create a "patch" that was placed on the heart.

Over the next year, the patients generally showed small improvements in their symptoms -- including the ability to walk without becoming breathless and fatigued.

However, experts cautioned that while the results are encouraging, there's a lot of work left ahead before stem cells can be used to treat heart failure.

"They've shown that this approach is feasible," said Dr. Eiran Gorodeski, a heart failure specialist at the Cleveland Clinic in Ohio.

But it's not clear whether the stem-cell tactic was actually effective, said Gorodeski, who was not involved in the study.

That's because the study didn't include a comparison group that did not receive stem cells.

So it's possible, Gorodeski explained, that the "modest" symptom improvements would have happened anyway. All of the patients were on standard medications, and some had heart devices implanted.

Stem cells are primitive cells that mature into the various cells that make up the body's tissues. In the past 15 years or so, scientists have tried to use the cells to help repair some of the damage seen in heart failure.

Heart failure is a progressive disease where the heart muscle is too damaged to efficiently pump blood throughout the body. It often arises after a heart attack.

Symptoms of heart failure include fatigue, breathlessness and swelling in the limbs. The condition cannot be cured, although medications and implantable devices can treat the symptoms.

In the new study, the researchers used stem cells from the patients' own thigh muscle to create a patch they placed on the heart.

That's in contrast to many past studies, where researchers have injected stem cells -- often from patients' bone marrow -- into the heart.

The patch tactic could have some advantages, said senior researcher Dr. Yoshiki Sawa, of Osaka University.

He said animal research suggests that cells in sheet form survive for a longer period, compared to injections.

To test the safety of the approach, Sawa's team recruited 27 patients who had debilitating symptoms despite standard heart failure therapies. The scientists extracted stem cells from each patient's thigh muscle, then cultured the cells so that they formed a sheet.

The sheet was placed on each patient's heart.

The tactic appeared safe, the researchers said, and there were signs of symptom improvements over the next six months to a year.

Why would stem cells from the thigh muscle affect the heart? It's not clear, Sawa acknowledged.

The stem cells don't grow into new heart muscle cells. Instead, Sawa explained, they seem to produce chemicals called cytokines that can promote new blood vessel growth in damaged areas of the heart. The theory, he said, is that "hibernating" cells in the heart muscle can then function better.

Still, it's too soon to know what the new findings mean, said Gorodeski.

This type of trial, called phase 1, is designed to look at the safety and feasibility of a therapy, Gorodeski said. It takes later-phase trials -- where some patients receive the treatment, and others do not -- to prove that a therapy actually works.

Those trials are underway, Sawa said.

Other studies are further along. Last year, researchers reported on a trial testing infusions of stem cells taken from the bone marrow of patients with severe heart failure.

Patients who received the therapy were less likely to die or be hospitalized over the next year, versus those given standard treatment only. But the study was small, and the stem cells had only a minor impact on patients' heart function.

So it's not clear why the stem-cell patients fared better, Gorodeski said.

For now, he stressed, all stem-cell therapies for heart failure remain experimental.

"There's no cell therapy that we can offer patients right now," Gorodeski said.

The message for patients, he added, is that heart failure can be treated, and researchers are looking for "innovative" ways to improve that treatment.

The study was published April 5 in the Journal of the American Heart Association.

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Can Stem Cell 'Patch' Help Heart Failure? - Montana Standard

Global 2017 Cell Therapy Technologies, Markets and Companies … – Yahoo Finance

Dublin, April 06, 2017 (GLOBE NEWSWIRE) -- Research and Markets has announced the addition of Jain PharmaBiotech's new report "Cell Therapy - Technologies, Markets and Companies" to their offering.

This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

The cell-based markets was analyzed for 2016, and projected to 2026.The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 305 of these are profiled in part II of the report along with tabulation of 291 alliances. Of these companies, 170 are involved in stem cells.

Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 64 Tables and 22 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

Key Topics Covered:

Part I: Technologies, Ethics & Regulations

Executive Summary

1. Introduction to Cell Therapy

2. Cell Therapy Technologies

3. Stem Cells

4. Clinical Applications of Cell Therapy

5. Cell Therapy for Cardiovascular Disorders

6. Cell Therapy for Cancer

7. Cell Therapy for Neurological Disorders

8. Ethical, Legal and Political Aspects of Cell therapy

9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions

10. Markets and Future Prospects for Cell Therapy

11. Companies Involved in Cell Therapy

12. Academic Institutions

13. References

For more information about this report visit http://www.researchandmarkets.com/research/s5g673/cell_therapy

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Global 2017 Cell Therapy Technologies, Markets and Companies ... - Yahoo Finance

Making CAR T-Cell Therapy Safer – The Scientist

Making CAR T-Cell Therapy Safer The Scientist JCAR015 is not the first CAR T-cell therapy to have been associated with patient deaths. In fact, even those trials considered a success sometimes have troubling safety profiles. For example, Novartis's lead candidate, the CD19-targeting CLT019, ...

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Making CAR T-Cell Therapy Safer - The Scientist

CAR T-Cell Therapy Yields High Response Rate in Aggressive … – Cancer Network

Patients with advanced non-Hodgkin lymphoma have limited options after treatment failure with chemotherapy or transplant.

In this video, Frederick Locke, MD, of the Moffitt Cancer Center in Tampa, Florida, discusses results of the ZUMA-1 trial, which tested chimeric antigen receptor (CAR) T-cell therapy with axicabtagene ciloleucel (axi-cel; KTE-C19) in this patient population. The treatment is given as a single infusion after conditioning chemotherapy with fludarabine and cyclophosphamide.

The multicenter clinical trial enrolled 111 patients with diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma, and transformed follicular lymphoma. Nearly all (99%) were able to have axi-cel manufactured from their cells. A total of 101 patients (91%) received the CAR T-cell therapy (10 patients were unable to receive therapy after cell collection, most due to disease progression).

Locke highlights the overall and complete response rates in the trial, compares the results to previous data involving other treatments in a similar patient population, and reviews toxicities associated with CAR T-cell therapy.

The results of ZUMA-1, which showed a 6-month overall survival of 80% (compared to 55% for historical controls), were presented at the 2017 American Association for Cancer Research Annual Meeting, held April 15 in Washington, DC.

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CAR T-Cell Therapy Yields High Response Rate in Aggressive ... - Cancer Network