Category Archives: Stem Cell Treatment


Cayden's second chance: 5-year-old has stem cell injection that may help his vision

The quest to save her grandson's eyesight began more than four years ago when Carmie Carr discovered a business on the Internet offering experimental stem cell injections in China.

At 4 months old, Cayden Baggett was diagnosed with optic nerve hypoplasia, a condition where the nerves in the eyes never fully develop. His family was told he would eventually go blind. Right now, Cayden can see light and dark, but little else.

Soon after learning about the stem cell treatments, the Carr family launched a campaign to raise $50,000 to cover the costs. The 2010 Gulf oil spill, coupled with the 2011 tornadoes in Tuscaloosa that destroyed their retail business, made collecting the funds next to impossible, they said.

Then, a friend told the family about a construction project he was working on just down the street. It was for a local radiologist who planned to offer stem cell injections to athletes with sports-related injuries.

He introduced the Carrs to Dr. Jason Williams. After talking with the family and researching the procedures, Williams agreed to help.

"There is no doubt in my mind this is the road the Lord has been leading us down," said Carmie Carr. "It connected like a perfect puzzle. We thought we were going to have to travel around the world. Instead, we drove four blocks."

A goal of Cayden's stem cell treatment is to stimulate new nerve growth that might improve his vision. Some children with similar health problems have shown marked improvement in recent years after being treated with stem cells in China, Carmie Carr said.

When the day of his procedure finally arrived, a groggy Cayden, now 5, was carried by his mother, Leanna Carr, into the Gulf Shores medical practice. During the procedure, Williams used the little boy's own fat through liposuction to harvest stem cells that were processed, then injected into his back.

Stem cells, sometimes called the body's master cells, are precursor cells that can develop into blood, bones and organs, according to the U.S. Food and Drug Administration, which regulates their use. Their promise in medicine, researchers say, is that the cells have shown potential to help regenerate other cells.

For about four months, Williams, a radiologist, has been performing stem cell injections, which are considered investigational in the U.S., at his Baldwin County practice. He has said the procedures meet FDA guidelines because the stem cells are collected from a patient's fat tissue and administered back to the same person.

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Cayden's second chance: 5-year-old has stem cell injection that may help his vision

ACT Treats 10th Patient in Embryonic Stem Cell Trials for Macular Degeneration

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, today announced treatment of the final patient in the first patient cohort in its Phase 1/2 clinical trial for Stargardts macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The surgery was performed on Friday, July 27 at Moorfields Eye Hospital in London, the same site as the first two treatments, by a team of surgeons led by Professor James Bainbridge, consultant surgeon at Moorfields and Chair of Retinal Studies at University College London. The outpatient transplant surgery was performed successfully without any complications, and the patient is recovering uneventfully. This is the tenth patient overall to now be treated with the RPE cell therapy developed by the company.

Our European trial is making very steady progress, having now completed enrollment of the first patient cohort, commented Gary Rabin, chairman and CEO. We are very encouraged and look forward to receiving clearance to initiate the treatment of the second patient cohort in the coming weeks.

The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the studys primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format. It is similar in design to the U.S. trial for SMD that was initiated in July 2011.

This is a significant month for the company, continued Mr. Rabin. One year ago we treated the first of our patients in our two U.S. clinical trials. The one-year follow-up for those initial patients indicates that the improvements in visual acuity we initially reported have in fact persisted now for a year. Indeed, we are consistently observing improvements in subjective and objective visual acuity for patients being treated at the various clinical centers involved in our trials. Again, these trials are still at very early stages, but these preliminary results indicate that we are on the right track.

The European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has officially designated ACT's human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells as an orphan medicinal product for the treatment of SMD.

About Stargardts Disease

Stargardts disease or Stargardts Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration.

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc. is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

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ACT Treats 10th Patient in Embryonic Stem Cell Trials for Macular Degeneration

Stem Cell Treatment = Drug

A US federal court rules that procedures in which a patients own stem cells are extracted, manipulated, and reinjected should be regulated by the FDA.

By Bob Grant | July 30, 2012

Leonardini | stock.xchng

After years of legal wrangling, the US District Court in Washington, DC, last week upheld the Food and Drug Administrations power to regulate adult stem cell treatments in which the cells are more than minimally manipulated before being injected back into the patient. The court ruled that the FDA was operating within its legal mandate when it filed suit against Colorado-based stem cell treatment clinic Regenerative Sciences in 2010 to stop them from extracting, processing, and then reinjecting patients own bone marrow stem cells to treat bone and joint disorders.

The FDA argued that the treatment fell under its purview and was subject to approval like any new drug because the extracted cells were significantly modified using reagents that cross state lines. Regenerative Sciences disagreed, characterizing the treatment as a simple medical procedure, which dont require FDA approval. The court sided with the FDA, making similar stem cell clinics popping up in the United States take notice. University of Minnesota bioethicist Leigh Turner told Nature that the ruling was spot on. It is much too simplistic to think that stem cells are removed from the body and then returned to the body without a manufacturing process that includes risk of transmission of communicable diseases, he said. Maintaining the FDAs role as watchdog and regulatory authority is imperative.

But Chris Centeno, Regenerative Sciences medical director told Nature that the clinic plans to continue offering patients 3 of its 4 stem cell treatments, in which cells are only processed for 2 days before reinjection. He added that the company will continue to treat patients using the process now prohibited by the FDA in a clinic located in the Cayman Islands and that Regenerative Sciences plans to appeal the courts ruling.

By Edyta Zielinska

The National Institutes of Health will fund 17 projects developing lab-on-a-chip applications to improve drug screening.

By Cristina Luiggi

After treating terminally ill patients with an unauthorized experimental probiotic procedure, two California doctors can no longer participate in human research.

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Stem Cell Treatment = Drug

Jamie’s dad to be stem cells donor

Father of Jamie Inglis to be stem cells donor

9:37am Saturday 28th July 2012 in News By Kate Liptrot, kate.liptrot@thepress.co.uk

THE father of a six-year-old boy bravely fighting cancer is likely to be a stem cell donor for medical treatment which could potentially save his life.

Jamie Inglis has a 75 per cent chance of survival if he successfully goes through a clinical trial in Germany, according to his oncologist at Leeds General Infirmary, an expert on neuroblastoma.

This form of cancer is so aggressive that treatment in the UK is inadequate and will prolong Jamies life only for months, so his family are in the process of raising 250,000 to fund the treatment. They have currently raised about 80,000.

Jamies parents, John and Vicky, of Kelfield, near Selby , have been tested to see who would be the most appropriate donor.

John has been chosen by the experts because he has the best properties to assist Jamies immune system, which does not recognise cancer cells.

He is due to travel to Germany in early August, when he will have tests to detect any defects and to clarify that he can definitely be a donor.

Mr Inglis said: We are really pleased, we feel quite positive we have got to the main point, which is killing the disease he had. We are getting closer to receiving the treatment. He said Jamie is also due for an appointment with his oncologist next week and for an MRI scan soon.

Jamie, a pupils at Lord Deramores Primary School in York, is due to travel to undergo treatment in Tubingen in Germany from August 20.

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Jamie’s dad to be stem cells donor

Stem cells for neck injury: $20 million

Stem cells for neck injury: $20 million July 27th, 2012, 3:45 pm posted by Pat Brennan, science, environment editor

Human neural stem cell. Image courtesy StemCells Inc.

The states stem-cell institute has awarded $20 million to UC Irvine researchers, along with a private company, to prepare the way for human testing of a treatment for spinal-cord injuries in the neck region one that could restore movement and independence for some of the 1.3 million spinal-cord injury sufferers in the United States.

The treatment, developed by the husband and wife research team, Aileen Anderson and Brian Cummings, along with StemCells Inc. of Newark, Ca., would involve injecting versatile human neural stem cells into the neck area.

The cells, capable of transforming themselves based on cues from the body, could then migrate to the injured area and perhaps repair the protective sheaths, known as myelin, around nerve cells. If the treatment works as expected, it would restore movement and body control for patients with debilitating injuries.

While the treatment has the potential to allow the paralyzed to walk again, more modest gains are more likely and well worth the effort, Anderson said Friday.

UC Irvine husband-wife research team, Brian Cummings and Aileen Anderson. Courtesy UC Irvine.

Obviously that would be, of course, what we in our wildest dreams would see in a clinical trial, she said. But likely what youre going to see for any spinal cord injury is much more incremental improvement in function. For people with spinal cord injuries, that could be a huge thing. It could help with health care costs, the ability to function independently. If you can type on a computer, versus not, or write with a pen it changes an awful lot.

The $20 million was among $150 million authorized on Thursday by the board of the California Institute for Regenerative Medicine, a stem-cell funding body created by a California voter initiative in 2004.

Anderson and Cummings are among a cadre of stem-cell scientists at UCIs Sue & Bill Gross Stem Cell Research Center, and have already pushed the field forward.

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Stem cells for neck injury: $20 million

UC Davis gets $53 million in stem cell funds to study Huntington's, other diseases

The University of California, Davis, scored a major coup in stem cell funding with a $53 million award Thursday for research into Huntington's disease, limb ischemia and osteoporosis.

The grants were approved Thursday afternoon by CIRM the California Institute for Regenerative Medicine. They are a major milestone for the university, which had received $73 million in past funding from the state agency.

"We're here to bring this new era of medicine to patients," UC Davis stem cell program director Jan Nolta said.

For Melissa Biliardi of Santa Maria, the vote symbolizes hope. Her son, James Birdsall, 32, was diagnosed four years ago with Huntington's disease. The degenerative brain disorder could prove fatal over the next 10 to 15 years. There is currently no cure or treatment, but with the grant, UC Davis researchers hope to deliver an effective therapy in four years.

"This is the most hope we've ever had for a cure or treatment," Biliardi said.

Her son suffers from involuntary movement and fatigue, all symptoms of the disease, and relies on a wheelchair to get around. Birdsall is one of 30,000 Americans living with the genetic disorder, according to Nolta. Another 150,000 are at risk, but many aren't diagnosed until their early 30s.

Created by voters in 2004, CIRM is financed by state bonds. The agency started with a $3 billion fund in 2007. Since then, it has doled out a quarter of its money about $900 million to various universities and private companies doing stem cell work in the state.

"We're driving opportunity here," CIRM President Alan Trounson said.

Huntington's is caused by toxic proteins that kill nerves in the brain. Limb ischemia causes blood clots that eventually lead to amputation. Osteoporosis is characterized by a loss in bone mass.

Together, the diseases afflict millions of Americans each year. UC Davis researchers said they are on the cusp of a major breakthrough to treating all three.

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UC Davis gets $53 million in stem cell funds to study Huntington's, other diseases

State stem cell research funding agency awards $20 million to UCI, StemCells Inc.

Public release date: 27-Jul-2012 [ | E-mail | Share ]

Contact: Cathy Lawhon clawhon@uci.edu 949-824-1151 University of California - Irvine

Irvine, Calif., July 26, 2012 Efforts to begin human clinical trials using stem cells to treat cervical spinal cord injury in the U.S. received a $20 million boost Thursday, July 26, from the state's stem cell research funding agency, the California Institute for Regenerative Medicine.

The award will be shared by Aileen Anderson and Brian Cummings, associate professors of physical medicine & rehabilitation at UC Irvine's Sue & Bill Gross Stem Cell Research Center, and Nobuko Uchida of StemCells Inc. in Newark, Calif. Anderson and Cummings proved that transplanting human neural stem cells discovered and developed by Stem Cells, Inc. into rodents with thoracic spinal cord injury could restore mobility. The CIRM award announced Thursday will fund the collection of data necessary to establish human clinical trials in the U.S.

"Our therapeutic approach is based on the hypothesis that transplanted human neural stem cells integrate into the injured spinal cord to repair the protective myelin sheath and spinal circuitry," Anderson said. "Any therapy that can partially reverse some of the effects of spinal cord injury could substantially change the quality of life for patients by altering their dependence on assisted living and medical care."

CIRM's governing board on Thursday gave authorized $150 million for eight teams at five institutions statewide. The projects backed are considered critical to the institute's mission of translating basic stem cell discoveries into clinical cures.

"CIRM's support for UC Irvine's efforts to advance novel stem cell-based therapies for a variety of diseases is extremely gratifying," said Peter Donovan, director of the university's Sue & Bill Gross Stem Cell Research Center. "This latest award for spinal cord treatment holds great promise. We are delighted."

About 1.3 million Americans suffer chronically from spinal cord injuries. In California, nearly 147,000 individuals are living with such damage, which can severely impair the movement, sensation and autonomic function of otherwise healthy people. Recovery from spinal cord injury is often limited, even after aggressive emergency intervention with steroids and surgery, followed by rehabilitation.

"That's crushing for anyone," Anderson noted. "It's very tough for patients and their families. We believe stem cell therapies could provide significant functional recovery, improve quality of life and reduce the cost of care for those with spinal cord injury. That's our goal."

Anderson's and Cummings' laboratory has a long history of collaboration with StemCells Inc. in addressing spinal cord injury, including studies that led to the world's first clinical trial of a neural stem cell therapy for chronic spinal cord injury. This Phase I/II clinical trial, currently under way in Zurich, recently reported positive safety data from the first cohort of treated patients and continues to enroll subjects.

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State stem cell research funding agency awards $20 million to UCI, StemCells Inc.

ACT Issued Broad Patent for Human RPE Cells Derived From All Types of Pluripotent Stem Cells

MARLBOROUGH, Mass.--(BUSINESS WIRE)--

Advanced Cell Technology, Inc. (ACT; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that it has been issued a patent in Australia, patent number 2005325753, Improved modalities for the treatment of degenerative diseases of the retina. The patent broadly covers the use of human retinal pigment epithelial (RPE) cells generated from pluripotent stem cells in the manufacture of pharmaceutical preparations of RPE cells, and the use of those preparations to treat patients with degenerative diseases of the retina such as Age-related Macular Degeneration. The patent covers the pharmaceutical formulation of human RPE cells made from a range of pluripotent stem cells, including both human embryonic stem cells (hESCs) and human induced pluripotent stem (iPS) cells.

We continue to make great progress with our patent estate covering RPE therapies, said Gary Rabin, chairman and CEO of ACT. Our ongoing success in securing broad patent protection around the world, including this newly-issued Australian patent, is a testament to our innovative chief scientific officer, Dr. Robert Lanza, and the rest of our scientific team.

The efficient production of highly pure RPE cell preparations represents a critical step in the creation of renewable sources of transplantable cells that can be used to target degenerative diseases of the eye such as Stargardts Macular Dystrophy (SMD) and dry Age-related Macular Degeneration (dry AMD).

Our current embryonic stem cell trials pave the way for other pluripotent stem cell therapies, commented Dr. Lanza. ACTs cellular reprogramming technologies using iPS cells are in an advanced stage of development, and we hope to be in a position to move toward clinical translation in the not-too-distant future. Since iPS cells can be made from the patients own cells such as skin or blood cells they may allow us to expand our cell therapies beyond immune-privileged sites such as the eye without the risk of immune rejection.

Mr. Rabin concluded, We are aggressively pursuing patent protection for a variety of aspects of our programs. Our intellectual property strategy includes both vigilance in pursuing comprehensive coverage from our initial patent filings, such as this new Australian patent, and filing for protection around our scientific teams various innovations. At the same time we are paying close attention to including within our patent coverage those ways others may wish to adapt our technology for commercial use, such as through the choice of stem cell source, or the use of solid supports or cell suspensions for delivery. Following this strategy, we are establishing both formidable barriers-to-entry for potential competitors, as well as strong potential licensing opportunities for others, translating into solid revenue generation possibilities for the company.

About Advanced Cell Technology, Inc.

Advanced Cell Technology, Inc., is a biotechnology company applying cellular technology in the field of regenerative medicine. For more information, visit http://www.advancedcell.com.

Forward-Looking Statements

Statements in this news release regarding future financial and operating results, future growth in research and development programs, potential applications of our technology, opportunities for the company and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words will, believes, plans, anticipates, expects, estimates, and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for future capital, risks inherent in the development and commercialization of potential products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the companys periodic reports, including the report on Form 10-K for the year ended December 31, 2011. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the companys management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Companys clinical trials will be successful.

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ACT Issued Broad Patent for Human RPE Cells Derived From All Types of Pluripotent Stem Cells

Arroyo might undergo stem cell therapy

MANILA, Philippines - Former President Gloria Macapagal-Arroyo might undergo stem cell therapy to improve her health, according to the alternative medicine facility in Tagaytay City that the Pampanga lawmaker visited Thursday.

Arroyo came to the facility complaining of difficulty in swallowing because of a bulge in her throat, according to a statement from the Green 8 Young Health & Wellness Center.

Her voice has also changed and she is losing weight because she can't swallow solid food. She also has angina, the center said.

Arroyo also complained of continuing neck and back pain.

"Our center is accepting her for possible stem cell therapy," the alternative medicine facility said. "The stem cell therapy is... strongly considered."

It said the Arroyo can undergo such therapy in the Tagaytay center while her physical therapy will continue at the Veterans Memorial Medical Center 4 times a week.

The Pampanga lawmaker is seeking treatment at the center through her sister, Cielo Macapagal-Salgado.

The center said Salgado was previously diagnosed with a cancerous lump in her breast.

"She consulted several doctors and was subsequently subjected to a myrad of treatment procedures. These, however, did not produce the desired results. When she came to our center she was cured of her cancer," it claimed.

Arroyo heads to Pampanga

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Arroyo might undergo stem cell therapy

Seventeenth Patient Dosed in Neuralstem ALS Stem Cell Trial

ROCKVILLE, Md., July 25, 2012 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that the seventeenth patient was treated in the ongoing Phase I trial of its spinal cord neural stem cells for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). This patient is also the second to return to the trial for additional injections. In this treatment, the patient received five injections in the cervical (upper back) region of the spinal cord, in addition to the ten he had previously received in the lumbar (lower back) region, for a total of 15 injections. The final previously treated patient of this cervical cohort is expected to return to the trial in August, provided the inclusion requirements continue to be met. This ground-breaking stem cell trial is taking place at Emory University Hospital in Atlanta, Georgia.

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

"We are pleased that this phase of the trial, in which we have been permitted by the FDA to take the unprecedented step of dosing patients for the second time, is progressing as planned," said Karl Johe, PhD, Neuralstem's Chairman and Chief Scientific Officer. "These are the first patients in the world to receive our cells in both the lumbar and cervical regions of their spinal cords, where the stem cell therapy could support both walking and breathing."

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 then advanced to transplantation in the cervical (upper back) region of the spine. The first cohort of three was treated in the cervical region only. The current cohort of three is receiving injections in both the cervical and lumbar regions of the spinal cord. In an amendment to the trial design, The Food and Drug Administration (FDA) approved the return of previously treated patients to this cohort. The second of these returning patients was just treated. 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 is in a Phase Ib safety trial evaluating NSI-189, its first neurogenic small molecule compound, for the treatment of major depressive disorder (MDD). Additional indications could include CTE (chronic traumatic encephalopathy), Alzheimer's disease, anxiety, and memory disorders.

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Seventeenth Patient Dosed in Neuralstem ALS Stem Cell Trial