WASHINGTON -- Patients who are lucky enough to get a transplant for a failed organ usually face a lifetime on anti-rejection drugs, which are expensive, dangerous and not always effective.

But in the future, those drugs may not be needed. A new study suggests that patients receiving an organ that's less than a perfect match can be protected against rejection by a second transplant -- this time of the organ donor's imperfectly matched stem cells.

Though preliminary, the new study is being hailed as a potential game-changer in the field of transplantation, a mystifying development that could offer hope to hundreds of thousands of patients who await or have received donor kidneys and depend on a harsh regimen of daily anti-rejection pills.

The small pilot study, reported Wednesday in the journal Science Translational Medicine, describes a novel regimen that combined old-fashioned cancer treatments with 21st century cell therapy to induce five patients' immune systems to accept donor kidneys as their own despite significant incompatibility.

If the technique proves successful in a larger group of people, future transplant patients may need to take anti-rejection drugs only briefly, and some who rely on them now could discontinue them safely. The recipients of kidneys as well as other organs, including heart, lung, liver and pancreas, might also benefit from access to a wider pool of organs.

The strategy could offer hope, too,

"Few transplant developments in the past half century have been more enticing," wrote pioneer transplant surgeons James Markmann and Tatsuo Kawai of Massachusetts General Hospital, in a commentary accompanying the study. If borne out, they wrote, the findings "may potentially have an enormous, paradigm-shifting impact on solid-organ transplantation."

In an interview, Markmann said that the greatest benefit of techniques described in the new research would be to greatly improve the lives of transplant patients by freeing them of a lifetime reliance on anti-rejection drugs.

But it might also ease the shortage of transplantable organs somewhat by reducing the number lost to rejection, he said. According to the National Kidney Foundation, 4,573 U.S. patients died in 2008 awaiting a kidney transplant due to a donor shortage.

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Stem cells may aid organ-transplant patients, study finds

3/7/2012 5:12 AM ET (RTTNews) - Stem cells have set the scientific world agog because it has been proposed as candidates to treat a myriad of diseases ranging from alzheimer's to arthritis, blindness, burns, cancer, diabetes, heart disease, liver disorders, multiple sclerosis, parkinson's, spinal cord injury and stroke.

Engaged in the development of novel stem cell therapeutics targeting diseases of the central nervous system and liver is clinical-stage company StemCells Inc. (STEM: News ).

For readers who are new to this Palo Alto, California-based company, here's what to expect in the coming months...

StemCells' lead product candidate is HuCNS-SC cells, a highly purified composition of human neural stem cells, currently in clinical development for spinal cord injury and for Pelizaeus-Merzbacher Disease, or PMD, a fatal myelination disorder in children.

A phase I/II clinical trial of HuCNS-SC cells in chronic spinal cord injury was initiated by the company last March. The trial, which is the world's first neural stem cell trial in spinal cord injury, is designed to enroll patients with thoracic (chest-level) neurological injuries with progressively decreasing severity of injury in three sequential cohorts.

The first patient in the trial was successfully transplanted with the company's proprietary HuCNS-SC adult neural stem cells last September, and enrollment in the first cohort of the spinal cord injury trial was completed last December. Following transplantation, the patients are being evaluated regularly over a 12-month period in order to monitor and evaluate the safety and tolerability of the HuCNS-SC cells.

The trial, which is currently open for enrollment for the remaining cohorts, is being conducted in Switzerland at the Balgrist University Hospital, University of Zurich.

In November 2011, Geron Corp. (GERN), the first company to get FDA approval for a clinical trial of an embryonic stem cell-based therapy, abandoned its phase I stem cell trial in patients paralyzed by spinal cord injuries - largely because of financial reasons.

The difference between the spinal cord injury trials of StemCells and Geron lies in the type of stem cells being evaluated. While Geron used human embryonic stem cells to treat spinal cord injuries in its trial, StemCells is using tissue-derived "adult" (non-embryonic) stem cells in its trials.

Yet another trial of StemCells that is underway is a phase I trial evaluating the safety and preliminary efficacy of HuCNS-SC cells as a treatment for Pelizaeus-Merzbacher Disease that primarily affects infants and young children.

Read more:
Will StemCells Walk The Talk?

Lindsay Porter knew she would eventually need a kidney transplant. She was 19 years old when her mother died from polycystic kidney disease -- a genetic condition that Porter had 50/50 odds of inheriting, and did.

"It didn't really affect me much until my early 30s," said Porter, an actress and mother living in Chicago. "And as I got into my 40s, my kidneys started getting very big with multiple cysts. They were huge."

Porter's kidneys weighed 16 pounds, causing an obvious bulge in her tiny frame.

"It was like two full-term babies inside me," she said, adding that people often mistook her for pregnant. "They had to be removed."

In May 2010, doctors removed Porter's overgrown and failing kidneys. Two months later, a friend gave her one of his. But it was no ordinary transplant. Along with the fist-size organ, doctors at Northwestern Memorial Hospital in Chicago transplanted bone marrow stem cells -- an experimental procedure they hoped would eliminate the need for anti-rejection drugs.

"These drugs are currently an absolute necessity, but they have a downside," said Dr. Joseph Leventhal, Porter's transplant surgeon at Northwestern Memorial Hospital and director of kidney and pancreas transplantation at Northwestern University Feinberg School of Medicine.

Anti-rejection drugs suppress the immune system, preventing it from attacking the donated organ like an infection. But suppressing the immune system makes the body vulnerable to infections and even cancer. And the drugs, which carry toxic side effects, can't ward off rejection forever. "Many individuals will still lose their transplants over time due to chronic rejection," said Leventhal.

To coax Porter's body into recognizing the new kidney as her own, Leventhal and colleagues wiped out part of her immune system and replaced it with the donor's. It took four days of chemotherapy, whole-body irradiation and a bone marrow transplant -- no walk in the park, according to Porter. But over time, the donor bone marrow stem cells gave rise to immune cells that accepted the kidney as if it was Porter's own -- a process called induced immune tolerance.

"At first I was taking 24 pills a day," said Porter, describing the "cocktail" of anti-rejection drugs needed to fend off an attack on her new kidney while the bone marrow stem cells were setting up shop. "And you really can't miss a dose. I had to set my cell phone alarm for every 12 hours every single day to remind me."

After six months, Porter started weaning herself off the drugs. And after a year, she no longer needed them at all.

Continued here:
New Transplant Approach Changes Lives

In just two years after going commercial, MediVet Americas headquarters in Nicholasville has seen exponential growth with no sign of slowing down.

The privately owned company is one of a handful of developers and researchers worldwide working on stem-cell regenerative therapy for animals suffering from osteoarthritis, hip dysplasia and other degenerative diseases. MediVet also provides other services such as stem-cell storage and selling stem cell extraction kits to veterinarians around the globe.

Founded as a research company six years ago in Sydney, Australia, MediVet as a whole is now represented in 26 counties and has hubs in 44 American states.

In 2010, there were only two full-time working employees at the office and lab building located in Nicholasville. The headquarters now employs 12 people with more sales, research and manufacturing jobs expected to open up in the next few months.

The Nicholasville lab has seen an increase of 3,000 percent in monetary growth since February 2011, said director of lab services Katherine Wilkie.

A University of Kentucky graduate, Wilkie said the lab has seen tremendous increase in clientele, as well. Currently, the facility banks approximately 600 different animal stem cells that account for more than 2,000 samples from all across the country and Canada.

In September, we received a sample from Alaska, she said. That now gives us an animal from each of the 50 states.

Stem-cell regenerative therapy has been used to treat everything from a mouse to an elephant, and Wilkie said they may soon do the procedure on a dolphin.

The companys blooming success boils down to its devolvement of advanced technologies in extracting, activating and storing stem cells that have cut the cost by one-third of their competitors, CEO Jeremy Delk said.

MediVets research and developments has made the procedures and other services they offer economically viable to the average pet owner, he said.

Read the rest here:
Pet stem cell biz booming for MediVet

By Michelle Fay Cortez - Wed Mar 07 19:16:55 GMT 2012

Kidney transplant patients given a mixture of stem cells from their organ donor were able to quit taking anti-rejection medicine in a small study, suggesting that life-long reliance on the toxic drugs may be avoidable.

Five of eight patients treated were able to stop taking about a dozen pills a day to suppress their immune systems. The drugs, which prevent rejection and stop tissue from a donated kidney from attacking the patient, can damage the transplant and cause diabetes, infections, heart disease and cancer.

The breakthrough, reported in the journal Science Translational Medicine, mixed stem cells from the donors infection-fighting immune system with the patients natural immune system. The result enabled tissue from both to co-exist in the transplant patient without either being seen as foreign by the immune system, researchers said.

The results may potentially have an enormous, paradigm- shifting impact on solid-organ transplantation, wrote James Markmann and Tatsuo Kawai from Massachusetts General Hospital in Boston, in an editorial accompanying the study. Although only a taste of things to come, few transplant developments in the past half-century have been more enticing than these that put transplantation tolerance within our grasp.

The findings are particularly striking since the patients werent perfect tissue matches with the living donors. The mismatch traditionally makes it more difficult for the donated organ to survive since the patients immune system perceives the unfamiliar tissue as a threat.

Its been a longstanding goal in transplantation to achieve tolerance, to get the recipient to see the donor organ as part of itself, said Joseph Leventhal, a surgeon at Northwestern Memorial Hospital in Chicago and the lead author. A road to tolerance now exists, he said.

Having two immune systems blend into one is called a chimerism. The long-lasting effect seen in the study may stem from the manipulation of stem cells taken from the donor in advance of the surgery, according to the report.

The cells were sent to Suzanne Ildstad, director of the Institute of Cellular Therapeutics at the University of Louisville in Kentucky. There facilitating cells that help transplants take hold were identified and used to enrich the mixture, which was given to the patient the day after surgery.

The researchers didnt provide details on how they crafted the stem cell mix, which may make it difficult for other investigators to confirm the findings, Markmann and Kawai wrote.

Originally posted here:
Stem Cells Help Kidney-Transplant Patients Skip Rejection Drugs in Study

ROCKVILLE, Md., March 7, 2012 /PRNewswire/ -- Neuralstem, Inc. (NYSE Amex: CUR) announced that the second patient to receive stem cells in the cervical (upper back) region of the spine was dosed on February 29th in the ongoing Phase I trial of its spinal cord neural stem cells in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). Patient 14 is also the first woman to be treated in the trial. Stem cell transplantation into the cervical region of the spinal cord couldsupport breathing, a key function that is lost as ALS progresses. The first twelve patients in the trial received stem cell transplants in the lumbar (lower back) region of the spinal cord only.

(Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO )

"This cohort of patients represents another first for our trial, as we transplant cells directly into the gray matter of the spinal cord in the cervical region," said Karl Johe, PhD, Neuralstem's Chairman and Chief Scientific Officer. "We are delighted that the surgeries are progressing in a region that could have a significant impact on the quality of life for ALS patients. With the safe transplantation of our 14th patient, we are well are on our way to demonstrating the safety of our novel procedure."

About the Trial The Phase I trial to assess the safety of Neuralstem's spinal cord neural stem cells and intraspinal transplantation method in ALS patients has been underway since January 2010. The trial is designed to enroll up to 18 patients. The first 12 patients were each transplanted in the lumbar (lower back) region of the spine, beginning with non-ambulatory and advancing to ambulatory cohorts. The trial has now progressed to the final six patients. Each is in the cervical (upper back) region of the spine. The entire 18-patient trial concludes six months after the final surgery.

About Neuralstem Neuralstem's patented technology enables the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells constitutively into mature, physiologically relevant human neurons and glia. Neuralstem is in an FDA-approved Phase I safety clinical trial for amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig's disease, and has been awarded orphan status designation by the FDA.

In addition to ALS, the company is also targeting major central nervous system conditions with its cell therapy platform, including spinal cord injury, ischemic spastic paraplegia and chronic stroke. The company has submitted an IND (Investigational New Drug) application to the FDA for a Phase I safety trial in chronic spinal cord injury.

Neuralstem also has the ability to generate stable human neural stem cell lines suitable for the systematic screening of large chemical libraries. Through this proprietary screening technology, Neuralstem has discovered and patented compounds that may stimulate the brain's capacity to generate new neurons, possibly reversing the pathologies of some central nervous system conditions. The company has received approval from the FDA to conduct a Phase Ib safety trial evaluating NSI-189, its first small molecule compound, for the treatment of major depressive disorder (MDD). Additional indications could include schizophrenia, Alzheimer's disease and bipolar disorder.

For more information, please visit http://www.neuralstem.com and connect with us on Twitter and Facebook.

Cautionary Statement Regarding Forward Looking Information This news release may contain forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this press release regarding potential applications of Neuralstem's technologies constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem's periodic reports, including the annual report on Form 10-K for the year ended December 31, 2010 and the quarterly report on Form 10-Q for the period ended September 30, 2011.

Read more:
Fourteenth Patient Dosed in Neuralstem ALS Stem Cell Trial

7 March 2012 Last updated at 11:47 ET By James Gallagher Health and science reporter, BBC News

Stem cells taken from the back of a human eye have restored some vision to blind rats, according to researchers.

They say the findings could help treat blindness, caused by glaucoma, if similar results can be repeated in humans.

The study, published in the journal Stem Cells Translational Medicine, used the cells to form new nerves in the eye.

These hooked up with the existing nerves, restoring sight.

Glaucoma can lead to blindness and is caused by a build-up of pressure within the eye. This kills retinal ganglion cells, the nerves which take information from the retina and pass it onto the brain.

Researchers at University College London and Moorfields Eye Hospital believe they have regenerated the retinal ganglion cells using human stem cells.

With permission from families, cell samples were taken from eyes which had been donated for cornea transplants.

It is a significant step towards our ultimate goal of finding a cure for glaucoma and other related conditions

Very rare cells in the eye, Muller glia stem cells, were collected. These were grown in the laboratory and converted into retinal ganglion cells.

Read the original here:
Human stem cells 'help blind rat'

Approach Could Free Organ Patients From Anti-Rejection Drugs

March 7, 2012 -- New research holds the promise of freeing many organ transplant patients from a lifetime of anti-rejection drugs.

In the first study of its kind, eight kidney transplant patients received stem cells from their kidney donors manipulated to trick their bodies into accepting the foreign organ as its own.

Transplant recipients who are not perfectly matched with their donors typically take several drugs a day for the rest of their lives to keep their bodies from rejecting the new organ and to treat the side effects of those drugs.

Lindsay Porter, who was the last of the eight patients enrolled in the new study, had her kidney transplant in the summer of 2010 and was weaned off all anti-rejection drugs within a year.

The Chicago actress and mother says she feels better than she has in 15 years and sometimes has to remind herself that she had a kidney transplant.

I was 45 when I had the surgery, and I knew I would probably need another kidney at some point, she tells WebMD. The opportunity to have a transplant that would last for the rest of my life and to avoid all of those drugs was very appealing.

The ongoing research is the culmination of many years of work by researcher Suzanne Ildstad, MD, of the University of Louisville, and other researchers, including transplant surgeon Joseph Leventhal, MD, PhD, of Chicagos Northwestern University.

The new wrinkle is that organ donors who are not a perfect genetic match with the patient donate blood as well as a kidney for the procedure.

Bone marrow stem cells collected from the blood were processed in an 18-hour procedure to remove cells associated with organ rejection, leaving behind facilitating cells that do not promote rejection, Ildstad says.

Excerpt from:
Altered Stem Cells Limit Transplant Rejection