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Nine Things to Know About Stem Cell Treatments

By Stem Cell Treatment

Stem cells have tremendous promise to help us understand and treat a range of diseases, injuries and other health-related conditions. Their potential is evident in the use of blood stem cells to treat diseases of the blood, a therapy that has saved the lives of thousands of children with leukemia; and can be seen in the use of stem cells for tissue grafts to treat diseases or injury to the bone, skin and surface of the eye. Important clinical trials involving stem cells are underway for many other conditions and researchers continue to explore new avenues using stem cells in medicine.

There is still a lot to learn about stem cells, however, and their current applications as treatments are sometimes exaggerated by the media and other parties who do not fully understand the science and current limitations, and also by clinics looking to capitalize on the hype by selling treatments to chronically ill or seriously injured patients. The information on this page is intended to help you understand both the potential and the limitations of stem cells at this point in time, and to help you spot some of the misinformation that is widely circulated by clinics offering unproven treatments.

It is important to discuss these Nine Things to Know and any research or information you gather with your primary care physician and other trusted members of your healthcare team in deciding what is right for you.

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Nine Things to Know About Stem Cell Treatments

Stem Cell Injection May Soon Reverse Vision Loss Caused By Age-Related Macular Degeneration

By Stem Cell Treatment

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Newswise LOS ANGELES (EMBARGOED UNTIL 7 A.M. EDT on APRIL 14, 2015) An injection of stem cells into the eye may soon slow or reverse the effects of early-stage age-related macular degeneration, according to new research from scientists at Cedars-Sinai. Currently, there is no treatment that slows the progression of the disease, which is the leading cause of vision loss in people over 65.

This is the first study to show preservation of vision after a single injection of adult-derived human cells into a rat model with age-related macular degeneration, said Shaomei Wang, MD, PhD, lead author of the study published in the journal STEM CELLS and a research scientist in the Eye Program at the Cedars-Sinai Board of Governors Regenerative Medicine Institute.

The stem cell injection resulted in 130 days of preserved vision in laboratory rats, which roughly equates to 16 years in humans.

Age-related macular degeneration affects upward of 15 million Americans. It occurs when the small central portion of the retina, known as the macula, deteriorates. The retina is the light-sensing nerve tissue at the back of the eye. Macular degeneration may also be caused by environmental factors, aging and a genetic predisposition.

When animal models with macular degeneration were injected with induced neural progenitor stem cells, which derive from the more commonly known induced pluripotent stem cells, healthy cells began to migrate around the retina and formed a protective layer. This protective layer prevented ongoing degeneration of the vital retinal cells responsible for vision.

Cedars-Sinai researchers in the Induced Pluripotent Stem Cell (iPSC) Core, directed by Dhruv Sareen, PhD, with support from the David and Janet Polak Foundation Stem Cell Core Laboratory, first converted adult human skin cells into powerful induced pluripotent stem cells (iPSC), which can be expanded indefinitely and then made into any cell of the human body. In this study, these induced pluripotent stem cells were then directed toward a neural progenitor cell fate, known as induced neural progenitor stem cells, or iNPCs.

These induced neural progenitor stem cells are a novel source of adult-derived cells which should have powerful effects on slowing down vision loss associated with macular degeneration, said Clive Svendsen, PhD, director of the Board of Governors Regenerative Medicine Institute and contributing author to the study. Though additional pre-clinical data is needed, our institute is close to a time when we can offer adult stem cells as a promising source for personalized therapies for this and other human diseases.

Next steps include testing the efficacy and safety of the stem cell injection in preclinical animal studies to provide information for applying for an investigational new drug. From there, clinical trials will be designed to test potential benefit in patients with later-stage age-related macular degeneration.

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Stem Cell Injection May Soon Reverse Vision Loss Caused By Age-Related Macular Degeneration

Stem cell injection may soon reverse vision loss due to age-related macular degeneration

By Stem Cell Treatment

LOS ANGELES (EMBARGOED UNTIL 7 A.M. EDT on APRIL 14, 2015) - An injection of stem cells into the eye may soon slow or reverse the effects of early-stage age-related macular degeneration, according to new research from scientists at Cedars-Sinai. Currently, there is no treatment that slows the progression of the disease, which is the leading cause of vision loss in people over 65.

"This is the first study to show preservation of vision after a single injection of adult-derived human cells into a rat model with age-related macular degeneration," said Shaomei Wang, MD, PhD, lead author of the study published in the journal STEM CELLS and a research scientist in the Eye Program at the Cedars-Sinai Board of Governors Regenerative Medicine Institute.

The stem cell injection resulted in 130 days of preserved vision in laboratory rats, which roughly equates to 16 years in humans.

Age-related macular degeneration affects upward of 15 million Americans. It occurs when the small central portion of the retina, known as the macula, deteriorates. The retina is the light-sensing nerve tissue at the back of the eye. Macular degeneration may also be caused by environmental factors, aging and a genetic predisposition.

When animal models with macular degeneration were injected with induced neural progenitor stem cells, which derive from the more commonly known induced pluripotent stem cells, healthy cells began to migrate around the retina and formed a protective layer. This protective layer prevented ongoing degeneration of the vital retinal cells responsible for vision.

Cedars-Sinai researchers in the Induced Pluripotent Stem Cell (iPSC) Core, directed by Dhruv Sareen, PhD, with support from the David and Janet Polak Foundation Stem Cell Core Laboratory, first converted adult human skin cells into powerful induced pluripotent stem cells (iPSC), which can be expanded indefinitely and then made into any cell of the human body. In this study, these induced pluripotent stem cells were then directed toward a neural progenitor cell fate, known as induced neural progenitor stem cells, or iNPCs.

"These induced neural progenitor stem cells are a novel source of adult-derived cells which should have powerful effects on slowing down vision loss associated with macular degeneration," said Clive Svendsen, PhD, director of the Board of Governors Regenerative Medicine Institute and contributing author to the study. "Though additional pre-clinical data is needed, our institute is close to a time when we can offer adult stem cells as a promising source for personalized therapies for this and other human diseases."

Next steps include testing the efficacy and safety of the stem cell injection in preclinical animal studies to provide information for applying for an investigational new drug. From there, clinical trials will be designed to test potential benefit in patients with later-stage age-related macular degeneration.

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Additional Cedars-Sinai authors include Dhruv Sareen, PhD; Yuchun Tsai, PhD; Bin Lu, MD, PhD; Benjamin Bakondi, PhD; Sergey Girman, PhD; and Anais Sahabian, PhD.

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Stem cell injection may soon reverse vision loss due to age-related macular degeneration

Hockey great Gordie Howe makes gritty fight back from stroke

By Stem Cell Treatment

LUBBOCK, Texas (AP) Ravaged by a stroke that left him unable to walk and barely able to speak, Gordie Howe decided it was time to quit.

His sons didn't want to hear it. Not from Mr. Hockey, whose 25-year career in the NHL was defined by his indomitable style and blend of grit and finesse.

"He was saying, 'Take me out back and shoot me,'" recalled Murray Howe, a diagnostic radiologist. "He was serious. It wasn't like a joke. I said, 'Dad, let's just see if we can help you first.'"

They found it in Mexico, where experimental stem cell treatments produced what his family called a "life-changing" turnaround that has put the 87-year-old Howe back on his feet. A second round of treatments is planned in June.

These days there's little doubt his spirits are high. At a recent session of occupational and physical therapy in Lubbock, Howe's eyes twinkled and he smiled now and then as he flirted with the two young women putting him through his paces. He even planted an innocent, light kiss on the cheek on his occupational therapist and twirled her once as if dancing.

"He is doing extremely well, very well for his age," said Nathalie Geddie, Howe's physical therapist, adding that he still has weakness on his right side. "To think about how far he's come since he's had his stroke, he's made significant functional gains."

Recent years have indeed been challenging for Howe, who set NHL marks with 801 goals and 1,850 points mostly with the Detroit Red Wings that held up until Wayne Gretzky surpassed him. He retired from hockey for good, but not until he was 52.

The body he counted on as an athlete has stayed relatively strong, but memory loss from the early stages of dementia became a problem even before his wife's death in 2009 after battling Pick's disease, a rare form of dementia similar to Alzheimer's. Colleen Howe's death seemed to hasten Howe's decline, Murray Howe said.

Howe's four children began taking turns having him live at their homes for weeks or months at a time.

Howe had a significant stroke Oct. 26, losing use of his right arm and leg, and his speech was slurred. He was still able to recognize people in family photos and those from his playing days, and he improved in the weeks that followed. But because he couldn't swallow following the stroke, he shed 35 pounds. And then came another blow the next month.

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Hockey great Gordie Howe makes gritty fight back from stroke

Brainstorm Cell Therapeutics (BCLI) Stock Gains Today on Potentially Positive Phase 2a Results

By Stem Cell Treatment

NEW YORK (TheStreet) -- Brainstorm Cell Therapeutics (BCLI) shares are up 9.13% to $5.02 as the biopharmaceutical company's stock gains momentum after the company announced that it will present positive results fromits phase 2a study of NurOwn when it releases the full set of data next weekat the American Academy of Neurology annual meeting.

The Israel-based developer of adult stem cell technologies for neurodegenerative diseases is developing NurOwn, a treatment being tested on patients with ALS or Lou Gehrig's disease, which is designed as a cell therapyin which bone marrow-derived mesenchymal stem cells are induced to secretfactors known to promote neuronal survival.

"We are thrilled to have the opportunity to share our results at this prestigious venue, and we look forward to discussing these findings with the medical community," said CEOTony Fiorino, MD, PhD. BCLI data by YCharts

Must Read: Warren Buffett's Top 25 Stocks for 2015

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Brainstorm Cell Therapeutics (BCLI) Stock Gains Today on Potentially Positive Phase 2a Results

Mesoblast takes partnership with Celgene, shares rise 27pc

By Stem Cell Treatment

Mesoblast chief executive Silviu Itescu says new strategic partner Celgene has a "strong franchise and sales and marketing. Photo: Josh Robenstone

Mesoblast chief executive Silviu Itescu says a $58.5 million investment from pharmaceutical giant Celgene gives the company a strong potential route to market as the stem cell therapy developernears its first sales.

The American giant Celgene, which had revenue of $US7.7 billion ($10 billion) from treatments for cancers and inflammatory diseases like psoriasis, picked up 15.3 million shares at $3.82 a share. Investors welcomed the deal, pushing the volatile stock up 24 per cent to $3.99 on Monday.

The surge boosted the value of Mr Itescu's 22 per cent stake in the company by $53 million to $272 million.

Mesoblast shares have lost about 16 per cent in the past year, compared with a 10 per cent rise in the benchmark S&P/ASX 200 index.

The stock last traded at the placement price a month ago, but has traded as high as $5.75 in the past year. Mr Itescu said Mesoblast and Celgene began talks about six months ago. He described the deal as "a fair transaction".

"The right time is when you come to an arrangement with a strategic partner," he said. "Where the share price is at any given time is much less relevant to the value of the technology and the partnership."

As part of the deal, which gives Celgene a stake of about 4.5 per cent, the company will have first right of refusal over licensing deals for the commercialisation of Mesoblast's adult stem cell products in the treatment of certain diseases.

That is, if Mesoblast is approached by another company to licence itsdrug candidates for the prevention and treatment of acute graft versus host disease (GVHD), certain oncologic diseases, inflammatory bowel diseases, and organ transplant rejection, Celgene will have six months in which it can mull whether it would like to trump the deal.

Mr Itescu said Celgene has a "strong franchise and sales and marketing capability" in cancer and inflammatory diseases. He said Mesoblast's candidate for the treatment of graft versus host disease, a common complication of bone marrow transplants used to treat cancer, "fits perfectly well into their cancer and oncology franchise."

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Mesoblast takes partnership with Celgene, shares rise 27pc

Study: Gene therapy superior to half-matched transplant for 'bubble boy disease'

By Stem Cell Treatment

Research first to compare alternative approaches to fully matched transplant for rare immune disorder

(WASHINGTON - April 13, 2015) - New research published online today in Blood, the Journal of the American Society of Hematology (ASH), reports that children with "bubble boy disease" who undergo gene therapy have fewer infections and hospitalizations than those receiving stem cells from a partially matched donor. The research is the first to compare outcomes among children with the rare immune disorder - also known as X-linked severe combined immunodeficiency (SCID-X1) - receiving the two therapeutic approaches.

Children with SCID-X1 are born with a genetic defect that prevents them from developing a normal immune system. Because they are prone to life-threatening infections, infants with SCID-X1 must be kept in a sterile, protective bubble and require extensive treatment for survival beyond infancy. Infants with SCID are most likely to survive if they receive a stem cell transplant from a fully matched donor - typically a sibling - a procedure that replaces an infant's diseased stem cells with healthy donor cells. Following a successful fully matched transplant, infants with SCID-X1 are able to produce their own immune cells for the first time.

In the absence of a fully matched stem cell donor, infants with SCID-X1 may receive a transplant from a partial, or "half-matched," donor - typically their mother or father. They may also undergo gene therapy, a much different approach. Gene therapy for SCID-X1 involves extracting an infant's own bone marrow, using a virus to replace faulty genetic material with a correct copy, and then giving "corrected" bone marrow back to the patient. Half-matched stem cell transplant and gene therapy represent secondary treatment approaches for infants with SCID-X1. Until recently, researchers had not yet compared outcomes among children treated with each respective approach.

"Over the last decade, gene therapy has emerged as a viable alternative to a partial matched stem cell transplant for infants with SCID-X1," said lead study author Fabien Touzot, MD, PhD, of Necker Children's Hospital in Paris. "To ensure that we are providing the best alternative therapy possible, we wanted to compare outcomes among infants treated with gene therapy and infants receiving partial matched transplants."

Dr. Touzot and colleagues studied the medical records of 27 children who received either partial-matched transplant (13) or gene therapy (14) for SCID-X1 at Necker Children's Hospital between 1999 and 2013. The children receiving half-matched transplants and the children receiving gene therapy had been followed for a median of six and 12 years, respectively.

The researchers compared immune, or T-cell, development among patients and also compared key clinical outcomes such as infections and hospitalization. Investigators observed that the 14 children in the gene therapy group developed healthy immune cells faster than the 13 children in the half-matched transplant group. In fact, in the first six months after therapy, T cell counts had reached normal values for age in more than three-fourths (78%) of the gene therapy patients, compared to roughly one-fourth (26%) of the transplant group. The more rapid growth of the immune system in gene therapy patients was also associated with faster resolution of some opportunistic infections (11 months in gene therapy group vs. 25.5 months in half-matched transplant group). These patients also had fewer infection-related hospitalizations (3 in gene therapy group vs. 15 in half-matched transplant group).

"Our analysis suggests that gene therapy can put these incredibly sick children on the road to defending themselves against infection faster than a half-matched transplant," Dr. Touzot said. "These results suggest that for patients without a fully matched stem cell donor, gene therapy is the next-best approach."

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Blood, the most cited peer-reviewed publication in the field of hematology, is available weekly in print and online. Blood is the official journal of the American Society of Hematology (ASH), the world's largest professional society concerned with the causes and treatment of blood disorders.

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Study: Gene therapy superior to half-matched transplant for 'bubble boy disease'

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