Friday, December 30, 2011

Proof of principle for transfusion of in vitro–generated red blood cells

http://bloodjournal.hematologylibrary.org/content/118/19/5071.abstract?sid=8adc2d0f-2b78-41b4-a689-45896782328c

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UT Southwestern Medical Center
The future of medicine, today.

Tuesday, May 31, 2011

ViaCyte progresses toward trials in stem cell diabetes treatment

The Los Angeles Times provides us with an update on ViaCyte, a San Diego-based company that is looking to make the insulin shot a relic of the past for Type 1 diabetes patients. It hopes to replace the shot with implants filled with insulin-producing pancreatic cells. And the company has its own special recipe to make immature pancreatic cells out of embryonic stem cells.

FierceBiotechResearch first took a look at ViaCyte a little less than a year ago. ViaCyte tells the LA Times that it has successfully "cured" diabetes in hundreds of fortunate mice and is ready to give it all a go on humans by 2013 in trials pushed along in part by $26 million in grants and loans from California's Institute of Regenerative Medicine (CIRM). One problem--among many--with stem cell treatments is the body's immune system rejecting them. ViaCyte says it has attacked this problem by keeping the warring parties separated with a thin membrane envelope that allows sugar in and insulin out. The cells remain safely wrapped in the envelope and away from immune-system cells.

"The safety factor is pretty high," Alan Trounson, president of CIRM, says, as quoted by the LA Times. Essentially, says company director Eugene Brandon, ViaCyte is "creating a replacement pancreas."

The article points out that ViaCyte is in the middle of a patent dispute over part of the stem cell procedure with Geron Corp. of Menlo Park, CA. Still, the company says, the trials will go forward irrespective of the outcome of the litigation.

- read the full story in the Los Angeles Times

Related Articles:
New stem cell strategy cures diabetes in mice
ViaCyte wins kudos for ESC program to treat diabetes

Stem Cells type I diabetes ViaCyte


--Ankur

Friday, May 13, 2011

Daniel Kraft is the man: Future of medicine under the microscope - CNET News

iPierian fires top execs, shifts focus - FierceBiotech

Schepens grows retinas, recovers vision using adult stem cells - Mass High Tech Business News

Liver repaired with stem cells taken from skin and blood - Telegraph

Advances in tissue engineering hold great promise for pediatric urology - - ModernMedicine

Thursday, May 12, 2011

REGENERATIVE MEDICINE ACT OF 2011 INTRODUCED IN HOUSE OF REPRESENTATIVES w/ support fr/ A.R.M.

http://www.marketwire.com/press-release/regenerative-medicine-act-2011-introduced-house-representatives-with-support-from-alliance-1513980.htm

Shire in the hunt for promising new stem cell tech

The UK's Shire has been making a series of small-$3 million to $5 million-investments in stem cell developers as the CEO continues to hunt for viable new business opportunities. "The science is there," said Angus Russell, "the challenge is finding something you can turn into a business." Russell makes clear that he's taking a distinctly unsentimental approach to stem cell research, noting that he only wants to acquire technology that insurers will be willing to pay for. Report

Shire Pharmaceuticals Stem cells


--Ankur

Thursday, April 14, 2011

Plasticell spins out regenerative medicine company

 
 

Sent to you by Ankur Gupta via Google Reader:

 
 

via FierceBiotech by Maureen Martino on 4/14/11

With plans to focus on its core technology of high throughput stem cell technologies, London-based Plasticell has spun out its  regenerative drug discovery. The new entity, called Progenitor Labs, will use Plasticell's ProScreen technology to discover small molecules that can regenerate tissues. Progenitor will also create progenitor cells to use in ProScreen. Plasticell founder Dr. Yen Choo will lead development of Progenitor Labs, while CFO Dennis Saw will move on to become CEO of the new company.

"Regenerative drugs are an attractive alternative to the use of cells in regenerative medicine," explained Choo in a statement. "Drugs are relatively easier to develop, manufacture, package, distribute and administer, and have clearer regulatory and reimbursement processes. Crucially, this business model is aligned with that of the pharmaceutical companies that specialise in discovering and marketing small molecule therapeutics."

- here's the release from Plasticell

Related Articles:
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Westphal offers optimistic outlook for regenerative medicine


 
 

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Tuesday, April 5, 2011

Patient's own cells may hold therapeutic promise after reprogramming, gene c...

 
 

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ScienceDaily (Apr. 4, 2011) — Scientists from the Morgridge Institute for Research, the University of Wisconsin-Madison, the University of California and the WiCell Research Institute moved gene therapy one step closer to clinical reality by determining that the process of correcting a genetic defect does not substantially increase the number of potentially cancer-causing mutations in induced pluripotent stem cells.

Their work, scheduled for publication the week of April 4 in the online edition of the journal Proceedings of the National Academy of Sciences and funded by a Wynn-Gund Translational Award from the Foundation Fighting Blindness, suggests that human induced pluripotent stem cells altered to correct a genetic defect may be cultured into subsequent generations of cells that remain free of the initial disease. However, although the gene correction itself does not increase the instability or the number of observed mutations in the cells, the study reinforced other recent findings that induced pluripotent stem cells themselves carry a significant number of genetic mutations.

"This study showed that the process of gene correction is compatible with therapeutic use," says Sara Howden, primary author of the study, who serves as a postdoctoral research associate in James Thomson's lab at the Morgridge Institute for Research. "It also was the first to demonstrate that correction of a defective gene in patient-derived cells via homologous recombination is possible."

Like human embryonic stem cells, induced pluripotent stem cells can become any of the 220 mature cell types in the human body. Induced pluripotent stem cells are created when skin or other mature cells are reprogrammed to a pluripotent state through exposure to select combinations of genes or proteins.

Since they can be derived from a patient's own cells, induced pluripotent stem cells may offer some clinical advantages over human embryonic stem cells by avoiding problems with rejection. However, scientists are still working to understand subtle differences between human embryonic and induced pluripotent stem cells, including a higher rate of genetic mutations among the induced pluripotent cells and evidence that the cells may retain some "memory" of their previous lineage.

Gene therapy using induced pluripotent stem cells holds promise for treating many inherited and acquired diseases such as Huntington's disease, degenerative retinal disease or diabetes. The patient in this study suffers from a degenerative eye disease known as gyrate atrophy, which is characterized by progressive loss of visual acuity and night vision leading to eventual blindness.

While diseases such as genetic retinal disorders and diabetes offer attractive targets for induced pluripotent stem cell-based transplant therapies, concerns have been raised over the commonly occurring mutations in the cells and their potential to become cancerous.

Howden says that because gene targeting to correct specific genetic defects typically requires an extended culture period beyond initial induced pluripotent stem cell generation, researchers have been interested to learn whether the process would increase the number of mutations in the cells. The team set out to determine if it was possible to correct defects without introducing a level of mutations that would be incompatible with clinical applications.

In the study, the researchers used a technique called episomal reprogramming to generate the induced pluripotent stem cells. In contrast to techniques that use retroviruses, episomal reprogramming doesn't involve inserting DNA into the genome. This technique allowed them to produce cells that were free of potentially harmful transgene sequences.

The scientists then corrected the actual retinal disease-causing gene defect using a technique called homologous recombination. The stem cells were extensively "characterized" or studied before and after the process to assess whether they developed significant additional mutations or variations. The results showed that the culture conditions required to correct a genetic defect did not substantially increase the number of mutations.

"By showing that the process of correcting a genetic defect in patient-derived induced pluripotent cells is compatible with therapeutic use, we eliminated one barrier to gene therapy based on these cells," Howden says. "There is still much work to be done."

David Gamm, an author of the study and an assistant professor with the Department of Ophthalmology and the Waisman Center Stem Cell Research Program, says the ability to correct gene defects in a patient's own induced pluripotent stem cells should increase the appeal of stem cell technology to researchers striving to improve vision in patients with inherited blinding disorders.

"Although further development certainly is needed before such techniques may reach the clinical trial stage, our findings offer reason for continued hope," Gamm says. "Dr. Howden and our collaborative group have overcome an important hurdle which, when considered in the context of other recent developments, may lead to personalized stem cell therapies that benefit people with genetic visual disorders."

In addition to primary author Howden, who holds joint appointments with the Morgridge Institute for Research, the Department of Cell and Regenerative Biology and the Genome Center of Wisconsin, co-authors of the study included: Thomson, who in addition holds an appointment with the Department of Molecular, Cellular & Developmental Biology, University of California-Santa Barbara; Gamm, who holds joint appointments with the UW-Madison School of Medicine and Public Health's Department of Ophthalmology and Visual Sciences and the Waisman Center Stem Cell Research Program; Jeff Nie, Goukai Chen, Brian McIntosh, Daniel Gulbranson, Nicole Diol and David Vereide with the Morgridge Institute for Research; Athurva Gore, Zhe Li, Ho-Lim Fung and Kun Zhang, of the Department of Bioengineering at the University of California-San Diego; and Benjamin Nisler, Seth Taapken and Karen Dyer Montgomery of WiCell Research Institute.

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Story Source:

The above story is reprinted (with editorial adaptations by ScienceDaily staff) from materials provided by University of Wisconsin-Madison.


Journal Reference:

  1. Sara E. Howden, Athurva Gore, Zhe Li, Ho-Lim Fung, Benjamin S. Nisler, Jeff Nie, Goukai Chen, Brian E. McIntosh, Daniel R. Gulbranson, Nicole R. Diol, Seth M. Taapken, David T. Vereide, Karen Dyer Montgomery, Kun Zhang, David M. Gamm, and James A. Thomson. Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy. Proceedings of the National Academy of Sciences, 2011; DOI: 10.1073/pnas.1103388108

Note: If no author is given, the source is cited instead.

Disclaimer: This article is not intended to provide medical advice, diagnosis or treatment. Views expressed here do not necessarily reflect those of ScienceDaily or its staff.


 
 

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U-M creates new stem cell lines | detnews.com | The Detroit News

Accidental Find May Lead to a Cure For Baldness | Singularity Hub

Tuesday, March 29, 2011

Biotech: Healing One Cell at a Time

Researchers see future in stem cell therapy for macular degeneration

 
 

Sent to you by Ankur Gupta via Google Reader:

 
 

via News by Howard Lovy on 3/28/11

As members of the baby-boom generation enter their golden years, finding treatments for age-related illnesses continue to take on importance. Among the most prevalent of these diseases is age-related macular degeneration (AMD), a leading cause of blindness worldwide. A group of researchers at Georgetown University Medical Center have taken what they call a "significant step" in combining AMD research with another important study area--stem cell therapy. They say they've demonstrated, for the first time, that it is possible to create retinal cells out of stem cells and transplant them into AMD patients.

For those who are keeping score, the type of stem cell we're talking about are human-induced pluripotent stem cells, which means they're from adults and not embryos. "The discovery of human induced pluripotent stem (hiPS) cells has opened a new avenue for the treatment of degenerative diseases, like AMD, by using a patient's own stem cells to generate tissues and cells for transplantation," the researchers said in a release.

AMD is caused by the death of retinal pigment epithelium (RPE), which is a layer of cells that nourish the cells in the retina. So, in a study published in the journal Stem Cells, the researchers showed that they have taken a step in figuring out how to program hiPS cells to take on the characteristics of RPE.

"This is the first time that hiPS-RPE cells have been produced with the characteristics and functioning of the RPE cells in the eye," said the study's lead author, Nady Golestaneh, in a release. "That makes these cells promising candidates for retinal regeneration therapies in age-related macular degeneration."

However, this researchers come with the usual caveats when dealing with adult-derived stem cells. "This isn't ready for prime time, though," Golestaneh added in the release. "We also identified some issues that need to be worked out before these cells are ready for transplantation but overall, this is a tremendous step forward in regenerative medicine."

- read the release from Georgetown University Medical Center
- and the abstract in the journal Stem Cells

Related Articles:
Researchers discover key to macular degeneration
Scientists herald "breakthrough" on blindness
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Tuesday, March 22, 2011

CDI to Supply iPSC-Derived Human Heart Cells for Roche's Drug Development Efforts

starGenetic Engineering & Biotechnology News
March 22, 2011 9:09 AM


--Ankur

Bluebird bio's gene therapy hits a high note

starNews
March 22, 2011 7:04 AM
by John Carroll

Bluebird bio's gene therapy hits a high note

Over the last week, Cambridge, MA-based bluebird bio has taken several key steps forward. The gene therapy pioneer named Alexandre LeBeaut as its new chief medical officer, signed up an impressive roster of experts for its scientific advisory board and inked a $4.2 million pact with the French Muscular Dystrophy Association to develop a treatment for beta-thalassemia and sickle cell anemia.

For bluebird bio CEO Nick Leschly, the series of moves help represent a turning point for a biotech determined to prove that its novel approach can make a big difference in the treatment of rare genetic conditions--an increasingly popular field in biopharma in which rare diseases represent significant market opportunities.

"I think, broadly speaking, this is a really, really exciting time," Leschly tells FierceBiotech Research. The staff is likely to swell to up to the 30 to 35 range (from 25 to 30) in the near future and there's enough money in the bank--from the likes of Third Rock Ventures, TVM Capital, Forbion Capital Partners, Easton Capital and Genzyme Ventures--to get through 2012 and possibly into early 2013. During that time, bluebird plans to demonstrate the potential of its approach: Taking stem cells from a patient's bone marrow, adding a healthy version of a disease-causing gene and then growing it in culture before returning the treatment back to the patient.

Bluebird's LentiGlobin inserts a fully functional human beta-globin gene into the patient's hematopoietic stem cells. Researchers are conducting a Phase I/II trial examining the safety and efficacy of LentiGlobin in the treatment of beta-thalassemia and sickle cell anemia. And the platform technology can be used to design treatments for other genetic targets as well.

"We never use the word cure," says Leschly, who's quick to add that the developer is still some years away from any possible NDA. "This is such a delicate population; we're cautious about overpromising and under-developing." But if bluebird bio is right, they are on track to advance a potentially transformative approach that skirts some obvious potential complications associated with cell transplantation.

- check out the release on bluebird's new CMO
- read the release on the new scientific advisory board
- see the announcement on the deal with the French Muscular Dystrophy Association

Related Articles:
Bluebird in spotlight after gene therapy cures blood disorder
Cincinnati team taking sickle cell cure into the clinic

bluebird bio sickle cell anemia


--Ankur

Investigators see promise in stem cell therapy for MS

starNews
March 22, 2011 8:26 AM
by Tracy Staton

Investigators see promise in stem cell therapy for MS

A long-term study of 35 people suffering from the most aggressive version of multiple sclerosis has underscored the potential of a radical new stem cell treatment approach.

In the study, chemotherapy was used to eliminate all of a patient's blood cells, including the errant immune cells that trigger MS. Enriched bone marrow stem cells were transplanted back into the patients in the hope that it would "reboot" their immune system and moderate the disease.

Over 11 years tracking results, 16 of those patients experienced a significant improvement for two years on their scorecard for symptoms of the disease. Participants in the study also demonstrated a reduction in telltale brain lesions. Two of the patients died as a result of complications from the transplants.

Keeping in mind that much more research has to be done, lead investigator Vasilios Kimiskidis, MD, of Aristotle University of Thessaloniki Medical School in Greece added that "our feeling is that stem cell transplants may benefit people with rapidly progressive MS. This is not a therapy for the general population of people with MS but should be reserved for aggressive cases that are still in the inflammatory phase of the disease."

- read the story at Science Daily

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multiple sclerosis Stem Cells


--Ankur