Tuesday, March 29, 2011
Researchers see future in stem cell therapy for macular degeneration
Sent to you by Ankur Gupta via Google Reader:
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
New stem cell research slows loss of vision
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Friday, March 25, 2011
Thursday, March 24, 2011
Wednesday, March 23, 2011
Tuesday, March 22, 2011
CDI to Supply iPSC-Derived Human Heart Cells for Roche's Drug Development Efforts
March 22, 2011 9:09 AM
--Ankur
Bluebird bio's gene therapy hits a high note
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
--Ankur
Investigators see promise in stem cell therapy for MS
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
Related Articles:
Research offers hope for treatments to repair MS damage
Stem cells used to reverse MS symptoms
Immune systems created with stem cells
--Ankur