Grant Initiates New Stem Cell Research for Patients Suffering with Corneal Blindness

Newswise LOS ANGELES (June 21, 2013) Corneal blindness affects roughly 8 million people worldwide, and traditional treatments and surgical interventions have seen little long-term success. With a new vision for stem cell corneal transplantation, and a $1.25 million grant from the National Eye Institute, Cedars-Sinai co-investigators Alexander V. Ljubimov, PhD, FARVO, and Clive Svendsen, PhD, hope to treat previously untreatable patients suffering with corneal blindness.

Our proposed and first-of-a-kind research deals with collecting human eye cells, reprogramming them back in time to a stem cell state and then using them to create a corneal cell source for transplantation. If successful, this could result in future human clinical trials that may lead to improved vision and overall quality of life, said Ljubimov, principal investigator of the grant and director of the Eye Program at the Cedars-Sinai Regenerative Medicine Institute. We are first experimenting with allogeneic cells from eye donors, but hope to use persons own cells in the near future.

A common cause of corneal blindness is limbal epithelial stem cell deficiency, which means that a patients history of genetics or eye injuries results in corneal scarring, blood vessel growth, and outgrowth of conjunctiva, all of which lead to vision loss.

This stem cell therapy approach uses induced pluripotent stem cells (iPSC), which are adult cells that are genetically reprogrammed to function as embryonic stem cells. Typically, iPSC technology has used stem cells from other places in the patients body, such as the skin, or from donor cells. However, advances in stem cell technology could make it possible for doctors to harvest stem cells from a patients own cornea or sclera (the white of the eye) and then transplant the reprogrammed cells back into the cornea of the patient.

If successful, this new approach may pave the way for human trials in patients affected by limbal epithelial stem cell deficiency, said Svendsen, director of the Cedars-Sinai Regenerative Medicine Institute. The engineering of a patients own adult stem cells to embryonic stem-like cells for the use in corneal transplantation may provide a noteworthy treatment alternative to patients affected worldwide.

Svendsen and Ljubimov lead a highly respected research team that includes Mehrnoosh Saghizadeh, PhD, co-investigator of the grant and assistant professor of surgery; Yaron Rabinowitz, MD, director of Ophthalmology Research and co-investigator of the grant; and Dhruv Sareen, PhD, director of the Regenerative Medicine Institute's iPSC core facility.

The research project grant is the first NIH R01 grant awarded to the newly established Cedars-Sinai Regenerative Medicine Institute and was given under the application Stem Cell Approaches to Developing New Therapies for Ocular Diseases."

This grant aims to restore function in diseased eye tissues through the revitalization of existing cells or the transplantation of new cells. The National Eye Institute, part of the National Institutes of Health, leads the federal government's research on the visual system and eye diseases and supports basic and clinical science programs that result in the development of sight-saving treatments.

NIH/NEI Funding: 1R01EY023429-01

# # #

Go here to see the original:
Grant Initiates New Stem Cell Research for Patients Suffering with Corneal Blindness

Stem cell treatment rapidly gains reputation as means to cure illnesses – Video

Stem cell treatment rapidly gains reputation as means to cure illnesses
News@1 Junior Edition - Stem cell treatment rapidly gains reputation as means to cure illnesses (Reported by EO Galvez) [June 16, 2013] For more news, visit:...

By: PTV PH

Visit link:
Stem cell treatment rapidly gains reputation as means to cure illnesses - Video

A new standard in pluripotent stem cell characterization — TaqMan® hPSC Scorecard™ Panel – Video

A new standard in pluripotent stem cell characterization -- TaqMan hPSC Scorecard trade; Panel
See it now at http://www.lifetechnologies.com/scorecard For Research Use Only. Not for use in diagnostic procedures. A new standard in pluripotent stem cell ...

By: LifeTechnologiesCorp

Continue reading here:
A new standard in pluripotent stem cell characterization -- TaqMan® hPSC Scorecard™ Panel - Video

Stem cell breakthrough can lead to one diabetes jab a year

London, Jun 17:

Scientists have made a stem cell breakthrough that could make it possible to treat diabetics with an annual insulin jab, eliminating the need for painful daily injections.

The new technique involves engineering blood stem cells into insulin-secreting cells.

Experts at Londons Imperial College, led by Professor Nagy Habib, and scientists at Hammersmith Hospital are now planning human trials of the new treatment after success in laboratory studies, the Daily Express reported.

This is a fantastic breakthrough that we hope will end the burden of daily jabs for diabetics, said Dr Paul Mintz, a leading stem cell researcher at Imperial College, who is part of the team pioneering the research.

The beauty of this treatment is that we manipulate the patients own stem cells, avoiding the complication of giving them something foreign which their body will reject, he said.

In diabetes the pancreas fails to make any insulin which crucially controls blood sugar levels or it doesnt make enough.

In laboratory studies, the researchers were able to get 35 per cent of engineered cells to make insulin.

They are now planning to nurture and grow these cells so they have a colony of 100 per cent insulin-making cells that can be injected into a patients body.

The team is working to develop stem cells that could release insulin for up to a year by coating the cells in a special biodegradable matrix.

View original post here:
Stem cell breakthrough can lead to one diabetes jab a year

NYSCF and Columbia researchers demonstrate use of stem cells to analyze causes, treatment of diabetes

Public release date: 17-Jun-2013 [ | E-mail | Share ]

Contact: David McKeon dmckeon@nyscf.org 212-365-7440 New York Stem Cell Foundation

NEW YORK, NY (June 17, 2013) A team from the New York Stem Cell Foundation (NYSCF) Research Institute and the Naomi Berrie Diabetes Center of Columbia University has generated patient-specific beta cells, or insulin-producing cells, that accurately reflect the features of maturity-onset diabetes of the young (MODY).

The researchers used skin cells of MODY patients to produce induced pluripotent stem (iPS) cells, from which they then made beta cells. Transplanted into a mouse, the stem cell-derived beta cells secreted insulin in a manner similar to that of the beta cells of MODY patients. Repair of the gene mutation restored insulin secretion to levels seen in cells obtained from healthy subjects. The findings were reported today in the Journal of Clinical Investigation.

Previous studies have demonstrated the ability of human embryonic stem cells and iPS cells to become beta cells that secrete insulin in response to glucose or other molecules. But the question remained as to whether stem cell-derived beta cells could accurately model genetic forms of diabetes and be used to develop and test potential therapies.

"We focused on MODY, a form of diabetes that affects approximately one in 10,000 people. While patients and other models have yielded important clinical insights into this disease, we were particularly interested in its molecular aspectshow specific genes can affect responses to glucose by the beta cell," said co-senior author Dieter Egli, PhD, Senior Research Fellow at NYSCF, who was named a NYSCFRobertson Stem Cell Investigator in 2012.

MODY is a genetically inherited form of diabetes. The most common form of MODY, type 2, results in a loss-of-function mutation in one copy of the gene that codes for the sugar-processing enzyme glucokinase (GCK). With type 2 MODY, higher glucose levels are required for GCK to metabolize glucose, leading to chronic, mildly elevated blood sugar levels and increased risk of vascular complications.

MODY patients are frequently misdiagnosed with type 1 or 2 diabetes. Proper diagnosis can not only change the patient's course of treatment but affect family members, who were previously unaware that they, too, might have this genetic disorder.

NYSCF scientists took skin cells from two Berrie Center type 2 MODY patients and "reprogrammed"or revertedthem to an embryonic-like state to become iPS cells. To examine the effect of the GCK genetic mutation, they also created two genetically manipulated iPS cell lines for comparison: one fully functional (two correct copies of the GCK gene) and one with complete loss of function (two faulty copies of the GCK gene). They then generated beta cell precursors from the fully functional and loss-of-function iPS cell lines and transplanted the cells for further maturation into immune-compromised mice.

"Our ability to create insulin-producing cells from skin cells, and then to manipulate the GCK gene in these cells using recently developed molecular methods, made it possible to definitively test several critical aspects of the utility of stem cells for the study of human disease," said Haiqing Hua, PhD, lead author on the paper, a postdoctoral fellow in the Division of Molecular Genetics, Department of Pediatrics and Naomi Berrie Diabetes Center at Columbia University and the New York Stem Cell Foundation Research Institute.

Continued here:
NYSCF and Columbia researchers demonstrate use of stem cells to analyze causes, treatment of diabetes

Takeda and Seattle Genetics Highlight Post-Hoc Analysis Examining Progression-free Survival with ADCETRIS® …

LUGANO, Switzerland--(BUSINESS WIRE)--

Takeda Pharmaceutical Company Limited (TSE:4502) and Seattle Genetics, Inc. (SGEN) today announced data from a post-hoc analysis examining progression-free survival (PFS) following treatment with ADCETRIS (brentuximab vedotin) versus last prior therapy in patients diagnosed with relapsed or refractory Hodgkin lymphoma (HL) post-autologous stem cell transplant (ASCT) or relapsed or refractory systemic anaplastic large cell lymphoma (sALCL). The data were highlighted during a presentation at the 12th International Conference on Malignant Lymphoma (ICML) being held June 1922, 2013 in Lugano, Switzerland.

ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of classical HL and sALCL.

The post-hoc analysis compared investigator assessed PFS following ADCETRIS single-agent treatment to the last prior systemic therapy in patients taking part in two pivotal Phase 2 studies. The post-hoc analysis was conducted in patients with relapsed or refractory HL post-ASCT or relapsed or refractory sALCL in the intent-to-treat (ITT) population. It also included prior systemic treatment histories and post-ADCETRIS stem cell transplant experience for each patient in the ITT populations.

These encouraging data suggest that ADCETRIS may delay disease progression compared to prior therapies used in this heavily pretreated patient population, said John Radford, M.D., Professor of Medical Oncology, University of Manchester, Manchester, UK. ADCETRIS is a CD30-targeted treatment option for patients with relapsed or refractory HL or relapsed or refractory sALCL that has shown a high overall response rate, including durable complete responses in both of its approved indications.

Progression-free survival analyses of two pivotal phase 2 studies of brentuximab vedotin in patients with relapsed or refractory Hodgkin lymphoma or systemic anaplastic large-cell lymphoma (Poster #303)

The analysis, presented by Dr. Radford, included:

Relapsed or Refractory HL post-ASCT

Relapsed or Refractory sALCL

Details of the poster presentation are as follows:

Original post:
Takeda and Seattle Genetics Highlight Post-Hoc Analysis Examining Progression-free Survival with ADCETRIS® ...

Scotland Continues to Forge Ahead in Stem Cell Research

BOSTON, June 12, 2013 /PRNewswire/ --Scotland's standing as Europe's largest and most highly regarded stem cell research community has been reaffirmed recently by announcements of funding from the UK Medical Research Council (MRC) and the British Heart Foundation to the MRC Centre for Regenerative Medicine (CRM) based at the Edinburgh BioQuarter. As part of a major $25m boost for UK regenerative medicine, the Edinburgh center will play a key role in the development of new therapies.

This and other recent news exemplify Scotland's strength and depth of expertise in this field. Developments at an academic level describing increased understanding of the mechanism of cellular reprogramming were recently reported by Dr. Keisuke Kaje from the MRC-CRM in Nature, whilst Scottish company Roslin Cells and the MRC-CRM have been actively collaborating on iPS cell line production ensuring a more quality-oriented perspective and generating more commercial opportunities.

Elsewhere, exciting research at Heriot-Watt University has seen the use of 3D printing techniques to produce clusters of viable stem cells that could speed up progress towards creating artificial organs. In the immediate future, Professor Will Shu and his team hope the technique can be used to generate biopsy-like tissue samples for drug testing.

At a clinical level also, Scotland has seen some major developments. ReNeuron has announced the successful conclusion of its Phase 1 stem cell clinical trial (PISCES) using its ReN001 therapy for treatment of stroke. This trial has been conducted at the Southern General Hospital, Greater Glasgow & Clyde, by Professor Keith Muir. The company is currently seeking final regulatory and ethical approvals for a Phase II studyscheduled to begin this summer. In a separate announcement ReNeuron has selected Dundee as the location of choice for another Phase 1 stem cell-based clinical trial, this time focused on critical limb ischemia.

Also underway is a 20-patient Phase 1 clinical trial using limbal cells to repair corneal blindness. This is being led by Professor Bal Dhillon in Edinburgh and is funded jointly by the UK Stem Cell Foundation and Scottish Enterprise in partnership with Scotland's Chief Scientist's Office.

Finally, a significant boost to Scotland's capabilities for supporting clinical trials was received with news that the new GMP Manufacturing Facility within the MRC-CRM, and managed by Roslin Cells along with the Scottish National Blood Transfusion Service (SNBTS), has been awarded a license by the Medicines and Healthcare products Regulatory Authority (MHRA), which permits the manufacture and release of Advanced Therapy Medicinal Products (ATMPs) for use in clinical trials.

"Scotland is once again demonstrating its expertise in academic, translational and clinical applications in stem cells and regenerative medicine" says Dr. Ed Hutchinson, spokesperson for life sciences at Scottish Enterprise. "These advances have been made possible through a collaborative approach whereby Scotland's universities, companies and health boards all actively participate in developments in this exciting area. With a supportive supply chain in place, Scotland can be seen as the place to go to undertake stem cell clinical trials and as a base for the European market."

This week, a Scottish delegation including the MRC Center for Regenerative Medicine, Roslin Cells and Universities of Edinburgh and Glasgow, in addition to stem-cell focused companies such as Biogelx and Sistemic, will attend the International Society for Stem Cell Research (ISSCR) conference in Boston. Further information on Scotland's stem cell industry can be found at the Scottish Development International booth, number 331, at the ISSCR conference or visit http://www.sdi.co.uk.

Media Contact: Rebecca Gehman Development Counsellors International rgehman@aboutdci.com; 212-725-0707

View original post here:
Scotland Continues to Forge Ahead in Stem Cell Research

Researchers demonstrate use of stem cells to analyze causes, treatment of diabetes

June 17, 2013 A team from the New York Stem Cell Foundation (NYSCF) Research Institute and the Naomi Berrie Diabetes Center of Columbia University has generated patient-specific beta cells, or insulin-producing cells, that accurately reflect the features of maturity-onset diabetes of the young (MODY).

The researchers used skin cells of MODY patients to produce induced pluripotent stem (iPS) cells, from which they then made beta cells. Transplanted into a mouse, the stem cell-derived beta cells secreted insulin in a manner similar to that of the beta cells of MODY patients. Repair of the gene mutation restored insulin secretion to levels seen in cells obtained from healthy subjects. The findings were reported today in the Journal of Clinical Investigation.

Previous studies have demonstrated the ability of human embryonic stem cells and iPS cells to become beta cells that secrete insulin in response to glucose or other molecules. But the question remained as to whether stem cell-derived beta cells could accurately model genetic forms of diabetes and be used to develop and test potential therapies.

"We focused on MODY, a form of diabetes that affects approximately one in 10,000 people. While patients and other models have yielded important clinical insights into this disease, we were particularly interested in its molecular aspects -- how specific genes can affect responses to glucose by the beta cell," said co-senior author Dieter Egli, PhD, Senior Research Fellow at NYSCF, who was named a NYSCF-Robertson Stem Cell Investigator in 2012.

MODY is a genetically inherited form of diabetes. The most common form of MODY, type 2, results in a loss-of-function mutation in one copy of the gene that codes for the sugar-processing enzyme glucokinase (GCK). With type 2 MODY, higher glucose levels are required for GCK to metabolize glucose, leading to chronic, mildly elevated blood sugar levels and increased risk of vascular complications.

MODY patients are frequently misdiagnosed with type 1 or 2 diabetes. Proper diagnosis can not only change the patient's course of treatment but affect family members, who were previously unaware that they, too, might have this genetic disorder.

NYSCF scientists took skin cells from two Berrie Center type 2 MODY patients and "reprogrammed" -- or reverted -- them to an embryonic-like state to become iPS cells. To examine the effect of the GCK genetic mutation, they also created two genetically manipulated iPS cell lines for comparison: one fully functional (two correct copies of the GCK gene) and one with complete loss of function (two faulty copies of the GCK gene). They then generated beta cell precursors from the fully functional and loss-of-function iPS cell lines and transplanted the cells for further maturation into immune-compromised mice.

"Our ability to create insulin-producing cells from skin cells, and then to manipulate the GCK gene in these cells using recently developed molecular methods, made it possible to definitively test several critical aspects of the utility of stem cells for the study of human disease," said Haiqing Hua, PhD, lead author on the paper, a postdoctoral fellow in the Division of Molecular Genetics, Department of Pediatrics and Naomi Berrie Diabetes Center at Columbia University and the New York Stem Cell Foundation Research Institute.

When given a glucose tolerance test three months later, mice with MODY beta cells had decreased sensitivity to glucose but a normal response to other molecules that stimulate insulin secretion. This is the hallmark of MODY. Mice with two faulty copies of the GCK gene secreted no additional insulin in response to glucose. When the researchers repaired the GCK mutation using molecular techniques, cells with two restored copies of GCK responded normally to the glucose stress test. Unlike other reported techniques, the researchers' approach efficiently repaired the GCK mutation without introducing any potentially harmful additional DNA.

"Generation of patient-derived beta cells with gene correction could ultimately prove to be a useful cell-replacement therapy by restoring patients' ability to regulate their own glucose. This result is truly exciting," said Susan L. Solomon, Chief Executive Officer of The New York Stem Cell Foundation.

See original here:
Researchers demonstrate use of stem cells to analyze causes, treatment of diabetes