Stem Cell Clinic

Skin graft gene therapy could treat obesity and diabetes – ResearchGate (blog)

By adminAugust 3, 2017Somatic Stem Cells

In a new study, researchers at the University of Chicago have provided proof of concept for a new form of gene therapy that is administered via a skin transplant. In the study, they treated type-2 diabetes and obesity in mice by inserting the gene for a glucagon-like peptide 1 (GLP1) that stimulates the pancreas to secrete insulin. The extra insulin can prevent diabetes complications by removing excessive glucose from the bloodstream. It can also delay gastric emptying and reduce appetite.

We spoke to one of the studys authors, Xiaoyang Wu, about the work.

ResearchGate: What motivated this study?

Xiaoyang Wu: We have been working on skin somatic stem cells for a long time. As one of the most studied adult stem cell systems, skin stem cells have several unique advantages as the novel vehicle for somatic gene therapy. For one, the system is well established. Human skin transplantation using a CEA device developed from skin stem cells has been used clinically for decades for burn wound treatment, and is proven to be safe and effective.

RG: Can you tell us what you achieved?

Xiaoyang Wu: We established a novel mouse to mouse skin transplantation system to test skin gene therapy. In the proof-of-concept study, we showed that we can achieve the systematic release of GLP1 at therapeutic concentration by engineered skin grafts.

RG: How does this work to treat obesity and diabetes?

Xiaoyang Wu: When engineered to express therapeutic hormones, such as GLP1, the skin grafts can be used to suppress body weight gain, and development of type 2 diabetes.

RG: What were some of the challenges in development? How did you overcome them?

Xiaoyang Wu: The mouse skin transplantation system has not been well established before. We circumvented the technical issues by building a novel skin organoid culture system in vitro.

RG: Are there alternate methods to delivery this type of therapy, and if so why is skin better?

Xiaoyang Wu: The GLP1 receptor agonist can be applied with an injection, but the half-life will be short. Skin based gene delivery provides a long term and safe way for drug delivery in vivo.

RG: Do think this would have a similar effect in humans?

Xiaoyang Wu: Our proof-of-concept work demonstrated its possible to use engineered skin grafts to treat many non-skin diseases. Clinical translation of our findings will be relatively easy, as skin transplantation in human patients has been well established and clinically used for treatment of burn wounds for many years.

RG: Whats next for your research?

Xiaoyang Wu: Before clinical translation, we will further characterize our mouse model of skin therapy, looking at potential immune reaction, stability of skin grafts, and duration of the therapeutic effects. We are also interested in using our mouse model to test other potential applications of skin gene therapy, such as human genetic diseases, including hemophilia and urea cycle disorders.

Featured image courtesy ofMehmet Pinarci.

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Skin graft gene therapy could treat obesity and diabetes - ResearchGate (blog)

Kite Seeks EU Approval for CAR T-Cell Therapy in 3 Lymphoma Subtypes – Lymphoma News Today

By adminAugust 3, 2017Uncategorized

Kite Pharma submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) requesting the approval of its CAR T-cell therapy, axicabtagene ciloleucel, as treatment for patients with certain lymphomas.

The include relapsed or refractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL), who cannot receive autologous stem cell transplants.

This is the first application for a CAR T-cell therapy ever submitted to the EMA.

The MAA submission of axicabtagene ciloleucel marks an important global milestone in the development of engineered T-cell therapy, Arie Belldegrun, MD, president and CEO of Kite, said in a press release.

He said the company is excited to be working with the EMAs Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to help bring this potentially transformative therapy to patients in the EU.

Axicabtagene ciloleucel, formerlyknown as KTE-C19, is a CAR (chimeric antigen receptor) T-cell therapy. The treatment consist of collecting the patients own T-cells and modifying them to express aCAR protein that recognizes the surface protein CD19, a molecule that is widelyexpressed by B-cell lymphomas and leukemias.

Kites application includes primary data from the ZUMA-1 trial (NCT02348216), a Phase 1/2 trial testing axicabtagene ciloleucel in treatment-resistant or relapsed aggressive non-Hodgkins lymphomapatients. The trial included 101 patients withDLBCL, PMBCL, or TFL, most with advanced-stage disease.

Data presented late June at the 22nd Congress of theEuropean Hematology Association(EHA),showed that 82 percent of patients responded to treatment after a single infusion of axicabtagene ciloleucel. This positive response was sustained in 44 percent of patients after amedian follow-up time of 8.7 months. At that time, 39 percent of patients exhibiteda complete response.

The most common severe treatment-related adverse eventsreported inZUMA-1 included cytokine release syndrome and neurologic events, which were generally reversible aftersuitable management.

Axicabtagene ciloleucelhas been designated a breakthrough therapy by the U.S. Food and Drug Administration for the treatment of DLBCL, TFL, and PMBCL, and received priority medicines (PRIME) regulatory support in the European Union. These designationsare meant to support a drugsdevelopment and accelerate its regulatory review.

The FDA is reviewingKites biologics license application (BLA) submitted for axicabtagene ciloleucel for the treatment of refractory aggressive non-Hodgkins lymphoma. A final decision is expected by Nov. 29.

Scottish firm gets EU funding for ‘off the shelf’ cancer cell therapy – pharmaphorum

By adminAugust 3, 2017Uncategorized

Scottish biotech TC BioPharm (TCB) has won European Union funding to develop a new off-the-shelf cell therapy for several types of solid tumour cancers.

The 4 million euro (3.6 million) grant represents the largest EU award to any UK company for development of a healthcare therapy.

TCBs technology is based around it gamma-delta T-cell (GDT) therapy, an autologous cell therapy formulated to treat patients with various tumours including malignant melanoma, kidney, and lung cancer.

Autologous cell therapies use the patients own cells to treat their tumour which is a costly and logistically complex approach.

But the grant from the EU Horizon 2020 initiative will allow TCB to develop a next-generation allogeneic approach, meaning treatments can be manufactured using existing cells from donors, stored in a bio-bank.

The technique is more scientifically complex because therapeutic cells will have been derived from a single donor to treat many people.

Allogeneic off-the-shelf approaches have significant advantages over existing autologous treatments, as a larger target population of cancer sufferers can be treated with a more reproducible product which has been campaign-manufactured in bulk to keep costs much lower.

With the EU funding, TCB will manufacture allogeneic cell banks during 2017/18 with a view to treating first cancer patients with the novel approach early in 2019.

The biotech with use its chimeric antigen receptor (CAR) to instruct the cells to attack specific tumour types.

This week, Kite Pharma filed its CAR T-cell cancer therapy with EU regulators in a rare form of blood cancer but this relies on harvesting a patients own cells.

Existing CAR-T therapies can only be used for blood cancers, so TCBs therapy could be a game-changer if its scientists can get it to work in solid tumours.

TCB is working with clinical centres of excellence to treat cancer patients in Glasgow, Edinburgh, Oxford, Southampton, London, Leeds, Cardiff, Manchester, Sheffield and Belfast.

It has already raised over 25 million euros (22.4 million) in funding since commencing operations in February 2014. It has premises in Glasgow, Edinburgh and London, employing over 50 members of staff.

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Scottish firm gets EU funding for 'off the shelf' cancer cell therapy - pharmaphorum

Sancilio Completes Enrollment in Clinical Trial of Altemia as Treatment for Children with Sickle Cell Conditions – Sickle Cell Anemia News

By adminAugust 3, 2017Uncategorized

Sancilio Pharmaceuticalshas completed enrolling patients in a Phase 2 clinical trial evaluating Altemia (docosahexaenoic acid)as a treatment forchildren with sickle cell anemia (SCA) and sickle cell disease (SCD).

The SCOT trial (NCT02973360) is aimed at finding an optimal dose of the therapy as well as evaluating its safety and effectiveness. Children aged 5 to 17 will berandomized to receive either Altemia or a placebo.

One of the four groups will receive 20 mg of Altemia per kg of body weight. A second group will receive 36 mg, a third 60 mg, and a fourth soybean oil as a placebo. The oral therapy is designed to be taken once a day.

The primary objective of the trial is to see whether Altemia lowers certain red blood cell fatty acids over four weeks,Sancilio said.Itexpects the study to be finished in August.

Research in the early 1990s suggested that some fatty acids decrease red blood cell destruction in mammals. Studies also showed that sickle cell patients have abnormally high levels of fatty acids in their red blood cells, white blood cells, platelets and plasma. The findings led to scientists surmising that certain fatty acids could play a role in the treatment of SCA and SCD.

In 2001, a small clinical trial showed that specific fatty acids could also reduce pain episodes in sickle cell patients. In addition, fatty acids can increase hemoglobin the molecules in blood that carry oxygen and reduce sickle cell patients anemia, organ damage and other complications.

Based on these findings, Sancilio designed Altemia to replenish the fatty acids destroyed by mutations of the beta thalassemia gene, which controls hemoglobin levels.The abnormalities are responsible for SCD, scientists say.

Altemias ultimate goal is restoring the fluidity or normal movement of red blood cell membranes. Sickle cell patients blood cells lose fluidity, triggeringa cascade of events that leads to organ damage. By minimizing this damage, the premise is that Altemia can reduce sickle cell crises and death.

We are very encouraged by the high retention rate and adherence to treatment seen thus far in the study,Fred Sancilio, president and chief executive officer of Sancilio Pharmaceuticals, said in a press release. This study will further our understanding of the impact of Altemia on SCD.

The faster than expected enrollment in this study highlights the intense interest and enthusiasm of the investigators, their staffs, as well as the patients and their families, in achieving this important step for our development program,said Dr. Adrian L. Rabinowicz, Sancilios chief medical officer. We look forward to reporting top-line results from the study early Q4 this year.

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Sancilio Completes Enrollment in Clinical Trial of Altemia as Treatment for Children with Sickle Cell Conditions - Sickle Cell Anemia News

High school student gets an early start in stem cell research at USC – USC News

By adminAugust 2, 2017Stell Cell Research

Even though Richard Lopez is still in high school, he can already tell you a thing or two about the ureteric bud, the metanephric mesenchyme and the developing kidney.

More impressively, he was familiar with these terms before starting his summer internship in the lab of Andy McMahon, kidney researcher and director of the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC.

I knew I was going to come here, Lopez said. So from December on, I was just reading papers that were written by Dr. McMahons lab. And so I read about the development of the kidney, kidney organoids, experimental methods like in situ hybridization, immunohistochemistry, all that stuff. Im really glad I did all of that because now that Im here, I understand whats going on.

Lopez undertook this intense preparation as part of the Science Research Program at his Connecticut boarding school, Choate Rosemary Hall. In addition to familiarizing him with the McMahon labs research, the program provided experience with useful molecular biology techniques, ranging from gel electrophoresis to polymerase chain reaction.

Lopez didnt start his high school career at Choate. Growing up in Lennox near the Los Angeles International Airport, he attended local public schools until his sophomore year in high school. At that point, his exceptional scores on the California Standardized Test attracted the attention of the Young Eisner Scholar program, which empowers underserved students to fulfill their potential.

As an Eisner Scholar, he earned both admission and a full scholarship to attend Choate. But the decision to leave home wasnt easy.

I was terrified at first, leaving everything behind, he said. I talked to my mom about it, and at first she was hesitant because I was born and raised here, and Im the only child. But then she realized that this is an amazing opportunity, and I cant let it go by.

Lopez recalls that Choate was initially in a huge culture shock from the occasional Maserati to the international student body to the exceptional academic opportunities such as the Science Research Program that brought him to USC.

In the McMahon lab, Lopez has learned about the molecular signals that drive the branching development of the kidney, and he has practiced a wide range of lab techniques.

Im really excited about science because I know its potential.

Richard Lopez

Im really excited and passionate about science because I know its potential, he said. If you pair that with math, you have no boundaries. If you look at the lab where Im working right now creating kidney organoids, learning about kidney development, these kinds of things can solve really burdensome illnesses that are fatal to some people, like end-stage renal disease and polycystic kidney disease.

To get to the lab every day, Lopez bike commutes a total of 32 miles from his home in Lennox to USCs Health Sciences Campus. Hes run the Los Angeles Marathon once and the San Francisco Marathon twice. In November, hes planning to travel to Florida to celebrate his 18th birthday with his first Ironman Triathlon a 2.4-mile swim, 112-mile bike ride and 26.2-mile run.

Hes participating in these events not only for fun and fitness, but also as a way to give back. Hes currently raising sponsorship money for the Partnership Scholars Program, which provides underserved junior high and high school students with educational and cultural experiences, ranging from theatergoing to restaurant outings to college tours. His goal is to raise $54,000 to fund three new scholars.

I was very lucky, he said. So I want to raise money for the scholarships that have helped me out along the way.

More stories about: Research, Stem Cells

Middle and high school students visit labs and tour USCs stem cell research center,cancer center and Keck Hospital of USC.

Its never too early to get teens interested in science, as evidenced by these enthusiastic researchers.

The teens boost their scientific IQ by conducting research in USC labs.

Andy McMahon and his colleagues investigate ways to help the millions who suffer from the chronic ailment.

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High school student gets an early start in stem cell research at USC - USC News

Surrey teen rallies stem cell donors to help with ‘desperate’ need for South Asians – Surrey Now-Leader

By adminAugust 2, 2017Stem Cell Clinic

Joban Bal in front of one of his tents during his swab drive calling for people to join the stem cell donor registry, which is in desperate need. (Photo: Alex Wilks)

Thirty-seven South Asian Canadian patients in desperate need of a stem cell transplant, says CBS

Alex Wilks, Surrey Now-Leader contributor

SURREY Canadian Blood Services (CBS) is in desperate need of donors to add to the registry and a Surrey teen wants to help.

Eighteen-year-old Joban Bal organized a stem cell swab event to help CBS build up the donor list.

He held his event on Saturday (July 29) during the annual Miri Piri Nagar Kirtan celebration at the Guru Nanak Sikh Gurdwara.

I saw the great need in hospitals for blood donors and especially stem cell donors, explained Bal. Essentially its rare that diseases do occur that need a stem cell donor but then that donation can make a real impact on a persons life.

Bal has been driven for the cause since high school where he launched Tamanawis Secondary Schools first blood donor clinic and stem cell swabbing campaigns.

I think of it as an investment in our future. Getting the younger generation really into blood donation because were trying to build up that community engagement with it, he said.

Bal is the founder of the One Blood for Life Foundation and a 2017 Winner of Surreys Top 25 under 25 Awards. Alongside a group of enthusiastic volunteers he hopes to recruit donors and educate people about the importance of blood and stem cell donation.

Stem cell transplantation is a procedure that replaces unhealthy blood-forming cells with healthy cells. It can be used to treat illnesses such as leukemia, lymphoma and various forms of cancer.

I dont want to say its personal. I want to say its more community oriented, explained Bal. I havent per se ever been affected by it, but I dont think it should take the point to where I am personally affected to see the impact it has.

It only takes about ten minutes to register. Donors are then briefed on what to expect if they are ever called in to donate. Following that is a quick cheek swab.

There are over 400,000 people in our registry and most people will never ever be called to be a match because it is that hard to find a match, said Territory Manager for CBS Sarah Jasmins.

Currently 37 South Asian Canadian patients are in desperate need of a stem cell transplants she emphasized.

They dont have a match, she said. Our registry right now is only six per cent South Asian and the important thing about a stem cell transplant is that the odds are youre only going to find somebody within your own ancestry.

So what were doing today is actually appealing to the South Asian population and getting them to join our registry, she adds.

Jasmins aims to build up the South Asian representation on the registry and with the help of Bal encourage people to donate blood as well.

It goes hand-in-hand. Usually somebody that requires a stem cell transplant also requires blood transfusions, she explained.

Bal chose to set up his red and white donor tents at the Miri Piri festival last weekend because of its unique South Asian diversity.

Its really important to bring awareness across cultural barriers and language barriers, he said. Because when we realize it theres something that connects us all, its blood. Regardless of what race, gender, ethnicity or if your old or young.

CBS requires donors to be between the ages of 17 to 35, in general good health and willing to help all Canadian patients.

I feel like every multicultural event should include something of this nature, explained Bal. It really is very closely integrated to community involvement. If we break down barriers between education and bring it to community events where we can facilitate this, we have the people; we have the resources, lets connect the two.

Joban Bal (far left) and some of his volunteers. (Photo: Alex Wilks)

One of the two swab tents set up on the parade route. (Photo: Alex Wilks)

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Surrey teen rallies stem cell donors to help with 'desperate' need for South Asians - Surrey Now-Leader

NantKwest (NK) Says Phase I Trial of aNK Cell Therapy in Patients … – StreetInsider.com

By adminAugust 2, 2017Uncategorized

Get daily under-the-radar research with StreetInsider.com's Stealth Growth Insider Get your 2-Wk Free Trial here.

NantKwest Inc. (Nasdaq: NK), a pioneering, next-generation, clinical-stage immunotherapy company focused on harnessing the unique power of the immune system using natural killer (NK) cells to treat cancer, infectious diseases, and inflammatory diseases, today announced the final results of a phase I clinical trial of the companys aNK cell therapy platform in relapsed hematological malignancies.

The data published in the journal Oncotarget by investigators at Princess Margaret Cancer Centre in Toronto, Canada, demonstrated continuing evidence of safety and efficacy, with an overall response rate of 42% and no evidence of grade 3 or 4 adverse events from the infusions. Of note 2 out of the 12 patients in the safety study with relapsed Hodgkin's Lymphoma and Multiple Myeloma, demonstrated durable complete response with single agent aNK therapy, and remain free of disease to date, 10 years and 2 years respectively.

Our study was designed to assess safety and preliminary evidence of efficacy in patients with relapsed, refractory hematological malignancies whose disease recurred after autologous hematopoietic cell transplantation (AHCT). In this heavily pretreated patient population that has a particularly poor prognosis, we demonstrated safety with minimal toxicity and showed preliminary evidence of efficacy, taking advantage of the unique properties of natural killer (NK) cells as an immunotherapeutic agent, said Armand Keating, MD, FRCP(C), Director, Cell Therapy Program, Princess Margaret Cancer Centre and University Health Network.

Dr. Keating continued, In this clinical study completed in 2015 of 12 patients with lymphoma and multiple myeloma who had relapsed after AHCT for refractory/relapsed disease, we report encouraging results with four patients (33%) currently alive and two complete responses, one for over two years and the second for over 10 years and conclude that aNK cell therapy warrants further clinical investigation.

Patrick Soon-Shiong, Chairman and CEO of NantKwest commented, Consistent with previous studies, Dr. Keatings clinical trial results, reporting a 42% overall response rate, provide additional clinical validation of the unique potential to deliver long-term remissions with limited toxicity using the companys novel NK cell therapy. Our aNK cell therapy is currently in an ongoing Phase II clinical study in Merkel cell carcinoma and represent a critical, foundational component in the companys recently launched NANT Cancer Vaccine clinical trial program.

Dr. Soon-Shiong continued, We believe our product portfolio of novel off-the-shelf NK cell therapies is unique in offering a more uniform, consistent and optimized product potency with minimal toxicity and we remain focused on translating these unique advantages to patient care as rapidly as possible.

aNK Cell Therapy Platform

NantKwests aNK cell therapy platform is being developed as an allogeneic, off-the-shelf therapy, offering a potent, standardized, uniform and consistent product, further optimized to enhance the key capability of natural killer cells to directly target and kill cancer cells. This novel cell therapy is currently in an ongoing Phase II clinical trial in Merkel cell carcinoma and is a critical, foundational component in the companys recently launch NANT Cancer Vaccine clinical trial program.

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TC BioPharm Wins $4.7M in EU Horizon 2020 Funding – Genetic Engineering & Biotechnology News

By adminAugust 2, 2017Uncategorized

Looking Beyond the U.K.

At present, TCB said, it is working with Clinical Centres of Excellence to treat cancer patients across the U.K. in Glasgow, Edinburgh, Oxford, Southampton, London, Leeds, Cardiff, Manchester, Sheffield, and Belfast. Plans call for the eventual clinical testing and treatment of patients with outside the U.K. as well.

I look forward to developing our novel allogeneic GDT cell therapies with clinical partners at trial sites in Prague, Madrid, Paris, Amsterdam, and Brussels, TCB chief executive Michael Leek, Ph.D., MBA, said in a statement.

Added TCB COO Angela Scott: We are thrilled that H2020 funding has been awarded, allowing us to treat large numbers of cancer patients across the EU and in North America.

Headquartered in Scotland at the Pentlands Science Park outside Edinburgh, TCB has raised more than 25 million ($29.6 million) in funding since it began operations in February 2014.

Back in January, TCB said it raised more than 6.25 million ($6.7 million) from investors toward advancing ImmuniCell. Funds came primarily from NIPRO Corp. of Osaka, Japan, and the Scottish Investment Bank, as well as several unnamed family-based investment sources.

The company employs more than 50 people at facilities in Glasgow, Edinburgh, and London.

The grant awarded to TCB is the largest such EU-award to any U.K. company for development of a therapeutic product. TCB said it was one of only 57 projects selected for funding out of 1514 applications to H2020s small- and medium-enterprise (SME) funding instrument, where fewer than 4% of companies applying to Phase 2 areselected.

Companies are evaluated for SME funding on criteria that include scientific excellence, business impact, and implementation quality.

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TC BioPharm Wins $4.7M in EU Horizon 2020 Funding - Genetic Engineering & Biotechnology News

Orbsen Therapeutics Receives Approval from European Regulators … – Business Wire (press release)

By adminAugust 2, 2017Uncategorized

NEW YORK--(BUSINESS WIRE)--Orbsen Therapeutics (www.orbsentherapeutics.com) breakthrough stromal cell immunotherapy, OBRCEL-M, developed as part of NEPHSTROM, a European Union Horizon 2020-funded research project coordinated by NUI Galway, has been approved to begin testing in a randomised, double blind, and placebo-controlled European clinical trial to treat diabetic kidney disease.

CEO of Orbsen Therapeutics, Dr Larry Couture, commented: This approval is an important step toward the clinical demonstration of the tremendous potential of Orbsens off-the-shelf allogeneic ORBCEL-M second generation stromal cell immunotherapy for the treatment of chronic diseases such as diabetic kidney disease.

Orbsens ORBCEL-M, a novel highly purified positively-selected stromal cell therapy for diabetic kidney disease, has demonstrated significant improvements in kidney function in pre-clinical models of diabetic kidney disease, which represents a significant step towards preparing this therapy for clinical application.

Chief Scientific Officer at Orbsen Therapeutics, Dr Steve Elliman, said: Approval of this first-in-man trial of ORBCEL-M is the result of a collaborative effort comprising Orbsen and nine clinical and cell manufacturing teams across Europe and was made possible by the EU Horizon 2020 program.

Diabetic kidney disease is the single leading cause of end stage renal disease in the industrialised world, accounting for 40% of new cases of end stage renal disease in the US and EU. The five-year mortality rate is 39% a rate comparable to many cancers.

Dr Elliman added: ORBCEL-M offers new hope for patients with diabetic kidney disease, a leading cause of death in the US and Europe, and a disease for which no new treatments have been approved in nearly 2 decades.

The pan-European clinical trial is being led by the renowned nephrologist, Professor Giuseppe Remuzzi at the Mario Negri Institute in Bergamo, Italy with clinical trial recruitment sites in Italy, Ireland (HRB Clinical Research Facility, Galway), and the UK (UHBFT, Birmingham and BHSCT, Belfast).

The primary aim of the clinical trial is to establish the safety and efficacy of ORBCEL-M, and to show that important markers of diabetic kidney disease are improved, thereby indicating the safety and efficiency of ORBCEL-M.

Commenting on the approval, Professor Timothy OBrien, Dean of the College of Medicine, Nursing and Health Sciences at NUI Galway, and NEPHSTROM coordinator, said: This approval is a vital step towards the initiation of clinical development of this promising new approach to the treatment of DKD, and patient enrolment will commence this summer.

The trial successfully secured voluntary harmonisation procedure (VHP) approval in April 2017. The VHP is designed to simplify clinical trials across multiple European member states by providing a centralised application procedure for clinical trial approval.

Dr Jack Kavanaugh, Orbsens chairman stated: I am thrilled to be associated with Orbsen, a leader in regenerative therapy, the incredible Orbsen team that has been assembled and its association with NEPHSTROM and NUI Galway.

About Orbsen Therapeutics

Orbsen Therapeutics, Ltd. is a leading company in the development of regenerative medicine therapies across a range of diseases and medical conditions. Founded in 2006, Orbsen Therapeutics Ltd., is a privately held company led by global leaders in the field of regenerative medicine. The company, which spun-out from Irelands Regenerative Medicine Institute at the National University or Ireland, Galway, has developed proprietary technologies that enables the isolation of pure and allogeneic stromal cells from human tissues which can be purified from a single donor, expanded and frozen to generate multiple doses of a high-margin, off the shelf therapeutic product.

For more information please contact info@orbsentherapeutics.com.

About NUI Galway

The University was established in the heart of Galway City, on the west coast of Ireland, in 1845. Since then it has advanced knowledge teaching and learning, through research and innovation, and community engagement.

With an extensive network of industry, community and academic collaborators around the world, NUI Galway researchers are tackling some of the most pressing issues of our times. Internationally renowned research centres based here include CRAM Centre for Research in Medical Devices, Insight Centre for Data Analytics, Moore Institute, Institute for Life course and Society and The Ryan Institute for Environmental, Marine and Energy.

For more information contact Gwen OSullivan, Press and Information Officer, NUI Galway at gwen.osullivan@nuigalway.ie

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Orbsen Therapeutics Receives Approval from European Regulators ... - Business Wire (press release)

Stem Cell Glossary – Closer Look at Stem Cells

By adminAugust 2, 2017Induced Pluripotent Stem Cells

Stem cell science involves many technical terms. This glossary covers many of the common terms you will encounter in reading about stem cells.

Adult stem cells A commonly used term for tissue-specific stem cells, cells that can give rise to the specialized cells in specific tissues. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent stem cells.

Autologous Cells or tissues from the same individual; an autologous bone marrow transplant involves one individual as both donor and recipient.

Basic research Research designed to increase knowledge and understanding (as opposed to research designed with the primary goal to solve a problem).

Blastocyst A transient, hollow ball of 150 to 200 cells formed in early embryonic development that contains the inner cell mass, from which the embryo develops, and an outer layer of cell called the trophoblast, which forms the placenta.

Bone marrow stromal cells A general term for non-blood cells in the bone marrow, such as fibroblasts, adipocytes (fat cells) and bone- and cartilage-forming cells that provide support for blood cells. Contained within this population of cells are multipotent bone marrow stromal stem cells that can self-renew and give rise to bone, cartilage, adipocytes and fibroblasts.

Cardiomyocytes The functional muscle cells of the heart that allow it to beat continuously and rhythmically.

Clinical translation The process of using scientific knowledge to design, develop and apply new ways to diagnose, stop or fix what goes wrong in a particular disease or injury; the process by which basic scientific research becomes medicine.

Clinical trial Tests on human subjects designed to evaluate the safety and/or effectiveness of new medical treatments.

Cord blood The blood in the umbilical cord and placenta after child birth. Cord blood contains hematopoietic stem cells, also known as cord blood stem cells, which can regenerate the blood and immune system and can be used to treat some blood disorders such as leukemia or anemia. Cord blood can be stored long-term in blood banks for either public or private use. Also called umbilical cord blood.

Cytoplasm Fluid inside a cell, but outside the nucleus.

Differentiation The process by which cells become increasingly specialized to carry out specific functions in tissues and organs.

Drug discovery The systematic process of discovering new drugs.

Drug screening The process of testing large numbers of potential drug candidates for activity, function and/or toxicity in defined assays.

Embryo Generally used to describe the stage of development between fertilization and the fetal stage; the embryonic stage ends 7-8 weeks after fertilization in humans.

Embryonic stem cells (ESCs) Undifferentiated cells derived from the inner cell mass of the blastocyst; these cells have the potential to give rise to all cell types in the fully formed organism and undergo self-renewal.

Fibroblast A common connective or support cell found within most tissues of the body.

Glucose A simple sugar that cells use for energy.

Hematopoietic Blood-forming; hematopoietic stem cells give rise to all the cell types in the blood.

Immunomodulatory The ability to modify the immune system or an immune response.

Induced pluripotent stem cells (iPSCs) Embryonic-like stem cells that are derived from reprogrammed, adult cells, such as skin cells. Like ESCs, iPS cells are pluripotent and can self-renew.

In vitro Latin for in glass. In biomedical research this refers to experiments that are done outside the body in an artificial environment, such as the study of isolated cells in controlled laboratory conditions (also known as cell culture).

In vivo Latin for within the living. In biomedical research this refers to experiments that are done in a living organism. Experiments in model systems such as mice or fruit flies are an example of in vivo research.

Islets of Langerhans Clusters in the pancreas where insulin-producing beta cells live.

Macula A small spot at the back of the retina, densely packed with the rods and cones that receive light, which is responsible for high-resolution central vision.

Mesenchymal stem cells (MSCs) A term used to describe cells isolated from the connective tissue that surrounds other tissues and organs. MSCs were first isolated from the bone marrow and shown to be capable of making bone, cartilage and fat cells. MSCs are now grown from other tissues, such as fat and cord blood. Not all MSCs are the same and their characteristics depend on where in the body they come from and how they are isolated and grown. May also be called mesenchymal stromal cells.

Multipotent stem cells Stem cells that can give rise to several different types of specialized cells in specific tissues; for example, blood stem cells can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain.

Neuron An electrically excitable cell that processes and transmits information through electrical and chemical signals in the central and peripheral nervous systems.

Pancreatic beta cells Cells responsible for making and releasing insulin, the hormone responsible for regulating blood sugar levels. Type I diabetes occurs when these cells are attacked and destroyed by the body's immune system.

Photoreceptors Rod or cone cells in the retina that receive light and send signals to the optic nerve, which passes along these signals to the brain.

Placebo A pill, injection or other treatment that has no therapeutic benefit; often used as a control in clinical trials to see whether new treatments work better than no treatment.

Placebo effect Perceived or actual improvement in symptoms that cannot be attributed to the placebo itself and therefore must be the result of the patient's (or other interested person's) belief in the treatment's effectiveness.

Pluripotent stem cells Stem cells that can become all the cell types that are found in an embryo, fetus or adult, such as embryonic stem cells or induced pluripotent (iPS) cells.

Preclinical research Laboratory research on cells, tissues and/or animals for the purpose of discovering new drugs or therapies.

Precursor cells An intermediate cell type between stem cells and differentiated cells. Precursor cells have the potential to give rise to a limited number or type of specialized cells. Also called progenitor cells.

Progenitor cells An intermediate cell type between stem cells and differentiated cells. Progenitor cells have the potential to give rise to a limited number or type of specialized cells and have a reduced capacity for self-renewal. Also called precursor cells.

Regenerative Medicine An interdisciplinary branch of medicine with the goal of replacing, regenerating or repairing damaged tissue to restore normal function. Regenerative treatments can include cellular therapy, gene therapy and tissue engineering approaches.

Reprogramming In the context of stem cell biology, this refers to the conversion of differentiated cells, such as fibroblasts, into embryonic-like iPS cells by artificially altering the expression of key genes.

Retinal pigment epithelium A single-cell layer behind the rods and cones in the retina that provide support functions for the rods and cones.

RNA Ribonucleic acid; it "reads" DNA and acts as a messenger for carrying out genetic instructions.

Scientific method A systematic process designed to understand a specific observation through the collection of measurable, empirical evidence; emphasis on measurable and repeatable experiments and results that test a specific hypothesis.

Self-renewal A special type of cell division in stem cells by which they make copies of themselves.

Somatic stem cells Scientific term for tissue-specific or adult stem cells.

Stem cells Cells that have both the capacity to self-renew (make more stem cells by cell division) and to differentiate into mature, specialized cells.

Stem cell tourism The travel to another state, region or country specifically for the purpose of undergoing a stem cell treatment available at that location. This phrase is also used to refer to the pursuit of untested and unregulated stem cell treatments.

Teratoma A benign tumor that usually consists of several types of tissue cells that are foreign to the tissue in which the tumor is located.

Tissue A group of cells with a similar function or embryological origin. Tissues organize further to become organs.

Tissue-specific stem cells Stem cells that can give rise to the specialized cells in specific tissues; blood stem cells, for example, can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent cells. Also called adult or somatic stem cells.

Totipotent The ability to give rise to all the cells of the body and cells that arent part of the body but support embryonic development, such as the placenta and umbilical cord.

Translational research Research that focuses on how to use knowledge gleaned from basic research to develop new drugs, treatments or therapies.

Zygote The single cell formed when a sperm cell fuses with an egg cell.

Link:
Stem Cell Glossary - Closer Look at Stem Cells