Gamida Cell Completes Enrollment for Phase I/II Study of Second Pipeline Product NiCord® for Hematological Malignancies

JERUSALEM--(BUSINESS WIRE)--

Gamida Cell, a leader in adult stem cell expansion technologies and products, announced today that it has completed enrollment for a Phase I/II clinical trial of NiCord, the companys second pipeline product.

NiCord is in development as an experimental treatment for a series of indications that potentially could be cured with a bone marrow transplantation including hematological malignancies (blood cancer), sickle cell disease, thalassemia, severe autoimmune diseases and metabolic diseases. The clinical trial announced today (clinicaltrials.gov identifier NCT01221857) is studying NiCord as an alternative investigational treatment for hematological malignancies (HM). A combined total of 11 patients were transplanted at Duke University Medical Center and at Loyola University Medical Center. Dr. Mitchell E. Horwitz of Duke University Medical Center is the principal investigator. Final results of the Phase I/II study are expected within 6 months. The company is also actively enrolling for a Phase I/II study of NiCord as an experimental treatment for sickle cell, a genetic blood disease (clinicaltrials.gov identifier NCT01590628).

NiCord is an expanded cell graft derived from an entire unit of umbilical cord blood enriched with stem cells. NiCord was developed based on Gamida Cells proprietary NAM technology. As the Phase I/II trial for HM is a first in man safety and efficacy study, for this stage, NiCord was transplanted with a second un-manipulated cord blood unit in a double cord blood configuration.

Dr. Tony Peled, chief scientific officer and vice president of research & development at Gamida Cell, said, Pre-clinical data demonstrated the uniqueness of NAM technology in not only decreasing the aging process but also preserving the characteristics and functions of ex vivo expanded stem cells (Experimental Hematology 2012;40:342355). Of significance, the Phase I/II clinical trial data have already shown that many of the patients in the study engrafted with the expanded cells of NiCord rather than with the second un-manipulated unit. This is the first time, in a situation where two units are transplanted, that the cultured stem cells demonstrated prompt and durable long-term engraftment (over one year) in the clinic. We look forward to sharing the complete results of this study in the coming months.

Dr. Yael Margolin, CEO of Gamida Cell, said, The clinical progress of the companys second pipeline product reaffirms Gamida Cells leadership role in the stem cell industry and the companys expertise in bone marrow transplantation. In the near future we plan to not only release the NiCord Phase I/II data but also the long awaited complete results of the Phase III study of StemEx, also for hematological malignancies, but clearly further ahead in development. The company remains on course with the development of StemEx and is considering strategic partners to develop its expanding pipeline of products and to bring StemEx to market.

About Gamida Cell

Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. The companys pipeline of stem cell therapy products are in development to treat a wide range of conditions including blood cancers, solid tumors, non-malignant hematological diseases such as hemoglobinopathies, neutropenia and acute radiation syndrome, autoimmune diseases and metabolic diseases as well as conditions that can be helped by regenerative medicine. Gamida Cells therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood, bone marrow and peripheral blood, which are expanded in culture. Gamida Cells current shareholders include: Elbit Imaging (NASDAQ: EMITF), Clal Biotechnology Industries (TASE: CBI), Israel Healthcare Venture, Teva Pharmaceutical Industries (NADAQ:TEVA), Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: http://www.gamida-cell.com.

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Gamida Cell Completes Enrollment for Phase I/II Study of Second Pipeline Product NiCord® for Hematological Malignancies

MEP fights European proposal to restrict stem cell research

Andrew Duff, MEP

Proposals to restrict stem cell research have been opposed by a Cambridge-based MEP.

Liberal Democrat Andrew Duff voted against a report in the European Parliament which he said would result in stricter requirements on voluntary and unpaid donations of tissues and cells for medical research.

Afterwards, he said: The report swings the debate on stem cell research in the wrong direction, allowing individual EU member states to stop important medical science in its tracks.

It is unhelpful to call for further restrictions on the donation of tissues and cells in the EU at a time when the number of patients in need of treatment based on stem cells is growing exponentially.

The report was drawn up by a Conservative MEP.

Mr Duff added: Cambridge is a world leader on life sciences. The European Parliaments restrictive conservative philosophy usually driven by ill-judged ethical motives puts our research centres at risk and could drive away high quality science abroad, for example to India.

Regenerative medicine relies on a steady supply of stem cells.

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MEP fights European proposal to restrict stem cell research

Leading stem cell scientists to focus on diabetes, eye diseases at Cedars-Sinai symposium

Public release date: 17-Sep-2012 [ | E-mail | Share ]

Contact: Nicole White nicole.white@cshs.org 310-423-5215 Cedars-Sinai Medical Center

LOS ANGELES Sept. 17, 2012 Leading scientists and clinicians from across the nation will discuss the latest findings on potential stem cell treatments for diabetes and eye diseases at the second Cedars-Sinai Regenerative Medicine Scientific Symposium.

WHO: Stem cell scientists, clinicians and industry leaders.

The symposium is being hosted by the Cedars-Sinai Regenerative Medicine Institute, led by Clive Svendsen, PhD. The institute brings together basic scientists with specialist clinicians, physician scientists and translational scientists across multiple medical specialties to convert fundamental stem cell studies to therapeutic regenerative medicine.

FEATURED RESEARCH: The symposium's morning session will feature an overview of the current state of stem cells and diabetes, including efforts to start the first clinical trials with stem cells for the treatment of diabetes. Other research to be presented includes an update on regenerative medicine approaches to treating macular degeneration, a progressive deterioration of the eye that causes gradual loss of vision. This will include an update from Gad Heilweil , MD, on a key, stem-cell clinical trial on macular degeneration at the University of California Los Angeles.

WHEN: Sept. 21, 2012 8:30 a.m. to 6 p.m. Thomson's lecture begins at 8:40 a.m.

WHERE: Harvey Morse Auditorium Cedars-Sinai Medical Center 8700 Beverly Boulevard Los Angeles, CA 90048

How to register: http://www.cedars-sinai.edu/RMI

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Leading stem cell scientists to focus on diabetes, eye diseases at Cedars-Sinai symposium

America Stem Cell, Inc. Awarded a Phase I STTR to Explore the Therapeutic Potential of Its Platform Technology (ASC …

SAN ANTONIO--(BUSINESS WIRE)--

America Stem Cell, Inc. (ASC) today announced that it has been awarded an Advanced Technology Small Business Technology Transfer Research (STTR) grant from the National Heart Lung and Blood Institute at the National Institutes of Health. This grant will be conducted in collaboration with scientists at the Wake Forest Institute of Regenerative Medicine (WFIRM) in Winston-Salem, NC, and will explore the combination of two technologies: ASC-101 developed by America Stem Cell and amniotic fluid-derived stem cells discovered and pioneered by Dr. Shay Soker and colleagues at WFIRM. We will examine the effect of ASC-101-treated amniotic fluid-derived stem cells in an experimental model of compartment syndrome. Compartment syndrome results from a variety of injuries such as fractures, contusions, burns, trauma, post-ischemic swelling and blast injuries such as gunshot wounds. If not addressed quickly, it can lead to considerable loss of muscle tissue. Musculoskeletal disorders are the primary cause of disability in the United States with associated costs of more than $800 billion annually. In addition to civilian injuries, more than 42,000 soldiers have been injured since the beginning of the Iraq and Afghanistan wars: the majority of these injuries were musculoskeletal in nature.

America Stem Cell has demonstrated that ASC-101 enhances the ability of stem cells to migrate to their target tissue. While most companies are concerned with the type of cells used for cell therapy (i.e. the hardware), America Stem Cell addresses how to get the cells to go where they are needed most (i.e. the software). With this award, America Stem Cell will expand the potential for therapeutic application of ASC-101 with amniotic fluid-derived stem cells. According to Dr. Leonard Miller, the Co-Principal Investigator on the grant, The successful combination of ASC-101 with amniotic fluid-derived stem cells would be directly relevant to improving the treatment of muscle damage that occurs following compartment syndrome as well as multiple other types of injuries.

America Stem Cell, Inc. is a clinical stage company that is in clinical trials at the University of Texas M.D. Anderson Cancer Center for improving clinical outcomes for cancer patients undergoing hematopoietic stem cell transplantation. This award enables America Stem Cell to expand the development of ASC-101 to yet another cell type. Lynnet Koh, CEO of America Stem Cell, noted, The combination of ASC-101 with amniotic fluid-derived stem cells could synergistically enhance the therapeutic and regenerative capacity of these cells and most importantly provide an off-the-shelf, effective solution for tissue damage due to multiple types of injuries or diseases. ASC-101 is a transformative technology with the potential to improve clinical outcomes for patients undergoing a wide variety of cell therapies for the treatment of diseases such as graft versus host disease, diabetic complications, and ischemic diseases such as myocardial infarctions, retinopathy and critical limb ischemia. America Stem Cell has established a number of collaborations examining the potential of ASC-101 to improve cell therapies for multiple clinical conditions using a wide variety of cell types.

About America Stem Cell, Inc.

America Stem Cell is a privately held biotechnology company based in San Antonio, TX, with offices in San Diego, CA, and is dedicated to the development and commercialization of enabling technologies to enhance and expand the therapeutic potential of cell therapies. The key technology platforms (ASC-101 and ASC-102) are designed to improve the homing and engraftment of cells to target organs. ASC-101 is currently in clinical trials to improve the therapeutic potential of hematopoietic stem cells for patients in need of hematopoietic stem cell transplantation. Additionally, these technologies have the potential to enhance the efficacy of cell therapies for the treatment of inflammation from chemotherapy/radiation, autoimmune diseases, and ischemic diseases including myocardial infarction and stroke. America Stem Cell has partnerships and collaborations with Kyowa Hakko Kirin, Spectrum Medical Innvoations, Florida Biologix, and various medical research institutions including the University of Texas M.D. Anderson Cancer Center, Oklahoma Medical Research Foundation, Fred Hutchinson Cancer Center,,University of California San Diego, Sanford-Burnham Institute, Indiana University, Juvenile Diabetes Research Foundation, as well as corporate partnerships. For additional information, please contact Lynnet Koh at 210-410-6427, or view http://www.americastemcell.com.

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America Stem Cell, Inc. Awarded a Phase I STTR to Explore the Therapeutic Potential of Its Platform Technology (ASC ...

Paralyzed Rats Walk Again After Stem Cell Transplant

Rats once paralyzed from complete surgical cuts through their spinal cords can walk again after stem cells were transplanted into the site of the injury, report researchers today in the journal Cell. The results suggest that stem cells might work as a treatment for patients even if they have completely severed cords, a potential therapy that has been viewed skeptically by many in the field.

Neural stem cells, derived from aborted fetal spinal cord tissue, were implanted onto each side of the spinal cord injury in the rats along with a supportive matrix and molecular growth factors. The human stem cells grew into the site of injury and extended delicate cellular projections called axons into the rats spinal cord, despite the known growth-inhibiting environment of the injured spinal cord. The rats' own neurons sent axons into the transplanted material and the rats were able to move all joints of their hind legs.

The cells are produced by a Rockville, Maryland company called Neuralstem. The same cells are also being tested in ALS patients (see "New Cells for ALS Patients") where they have shown some promise of stabilizing the progressive disease. Last month, the company announced that it has asked to FDA to approve a trial to test the cells in spinal cord-injured patients.

Researchers are currently testing neural stem cells from a Newark, California-based company called StemCells Inc, in spinal cord injured patients; two of the three patients have reported the recover of some sensation (see "Human Stem Cells Found to Restore Memory" for an overview of the company).

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Paralyzed Rats Walk Again After Stem Cell Transplant

Marlee Matlin fears for deaf stem-cell treatment

British researchers have been able to rebuild nerves in the ears of gerbils, and it is believed the same technique could one day be applicable to deaf humans.

However, Oscar-winning actress Matlin, who has been deaf since she was 18 months old, is worried about the implications of the development.

In a series of posts on Twitter.com, she writes, "'Deafness cure' is trending (on Twitter). My concern is that it's bigger than a 'trend.' It involves people & not as simple as the 4 letters in 'cure.' For those who think being deaf is a handicap, there are millions of Americans who sign, who are deaf, and are not a 'disease' to cure.

"Think about this. What if someone told you that you could've been made different than the content person you are with genetics. Would you? Now think how millions of deaf people who lead productive lives, would feel when told that babies born deaf can be 'cured'."

Matlin also told followers, "Be proud of who you are, regardless of what people think is a 'handicap' or 'normal.' Normal is what you want to be. Don't let anyone tell you who or what you should be."

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Marlee Matlin fears for deaf stem-cell treatment

Human stem cell treatment restores hearing in gerbils

A UK study in the journal Nature reports that deaf gerbils have had their hearing restored following a human stem cell treatment.

The researchers at the University of Sheffield, including Dr. Marcelo Rivolta, aimed to replace damaged nerve cells, called spiral ganglion neurons, that are unable to convert sound waves in the air into electrical signals to your brain. Roughly one in 10 people with profound hearing loss have this auditory damage, according to the LA Times.

The researchers used stem cells from a human embryo, added that to a "chemical soup," as the BBC referred to it, that converted them into cells similar to the spiral ganglion neurons. The cells were then injected into the inner ears of 18 gerbils.

Over the course of the 10 week study the gerbils' hearing improved by an average of 45 percent.

Rivolta told the BBC, "It would mean going from being so deaf that you wouldn't be able to hear a lorry or truck in the street to the point where you would be able to hear a conversation. It is not a complete cure, they will not be able to hear a whisper, but they would certainly be able to maintain a conversation in a room."

The LA Times noted that the researchers hope this study will spark a new interest in using stem cells to treat hearing loss in people.

But stem cell research is still highly controversial. The AP explained that human embryonic stem cells are initially obtained by destroying embryos, but they can be manipulated to produce any type of cell.

http://www.globalpost.com/dispatch/news/health/120913/human-stem-cell-treatment-restores-hearing-gerbils

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Human stem cell treatment restores hearing in gerbils

Deaf Gerbils Can Hear Again After Stem Cell Treatment

Researchers at the University of Sheffield in the UK have been able to restore the hearing of deaf gerbils using stem cells, they reported yesterday in the journal Nature. If it works in humans, this new therapeutic strategy could improve the lives of people that are hard-of-hearing.

There are different types of deafness, but this research focuses on auditory neuropathy. This disorder occurs when sound enters the inner ear normally but the signals created by the ear are lost along the way to the brain. In the ear, sound waves are translated into electrical signals when they vibrate tiny hair cells in your inner ear. Loss or damage of these hair cells and the brain cells they communicate with make hearing difficult.

The animals were deafened in one ear using a drug to destroy their auditory nerves before receiving an injection of around 50,000 human embryonic stem cells, which had previously been treated with chemicals to coax them into becoming ear cells.

Gerbils were used because of they hear a similar sound range as humans. After the treatment the researchers looked for brain signals created in response to sounds to detect improvement: Some gerbils restored up to 90 percent of the hearing within 10 weeks of the treatment.

BBC News spoke to Dr. Marcelo Rivolta of the Department of Biomedical Sciences at the University of Sheffield and he acknowledges that this is not a complete cure and while someone may still not be able to hear a whisper, they will certainly be able to maintain a conversation in a room.

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Deaf Gerbils Can Hear Again After Stem Cell Treatment

Stem cell treatment restores hearing in gerbils

A novel treatment using human embryonic stem cells has successfully restored some hearing to previously deaf gerbils, according to a study published this week in the journal Nature.

Hearing loss is generally caused by the interruption of two different types of cells: The loss of hair cells in the ear, which transform vibrations into electrical signals, and loss of the auditory nerve, which transmits the signals detected by the hair cells to the brainstem. While cochlear implants have proven effective in restoring hearing in cases of hair cell damage, no such treatment has existed for the roughly 10% cases in which the auditory nerve itself is damaged.

The new strategy, designed by Marcelo Rivolta and his team at the University of Sheffield, uses techniques the group has recently developed to coax human embryonic stem cells to differentiate into what are called "otic progenitor cells" -- cells that have the possibility to develop further into either hair cells or auditory nerve cells. The progenitor cells are then transplanted into the ears of gerbils with damaged auditory nerves, and allowed to differentiate further. Gerbils were used in the experiment because they hear a similar range of sounds as humans do.

At that point, the researchers held their breath, hoping that the cells would integrate themselves with the existing infrastructure and take their place in the chain of sensory signaling between the hair cells and the brainstem. In nearly all cases, the scientists could clearly see under the microscope that the new cells had targeted the right spots, reconnecting the hair cells to the brainstem.

But the ultimate test is hearing itself. To test this, the researchers used a standard approach called auditory-evoked responses, which are detected in the brainstem and provide a clear verdict of whether or not sound is being successfully transmitted to the brain.

Control animals with their auditory nerves knocked out did not recover during the experiment -- in order for a sound to register an auditory-evoked response in the brainstem, the control animals basically had to be at a rock concert, requiring a 76-decibel sound. But in the treated animals, that number dropped to 50 decibels on average, and in some animals approached the levels of animals whose hearing was never damaged at all. The strength of the effect was akin to suddenly being able to hear someone talking while previously not being able to hear them yell.

The researchers hope that their method will spark a new interest in using stem cells to treat hearing loss in people, though much work needs to be done before that is a real possibility. Hurdles include developing a surgical technique to access the appropriate part of the ear in people, and ensuring that the treatment sticks over long periods of time.

Nevertheless, the scientists are optimistic that the approach can be directly translated to humans with hearing loss, finally allowing people who cannot benefit from a cochlear implant to hear again.

You can read a summary of the paper here.

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Stem cell treatment restores hearing in gerbils

Neuralstem Gains on Stem Cell Therapy for Paralyzed Rats

By Ryan Flinn - 2012-09-13T20:06:09Z

Neuralstem Inc. (CUR), a biotechnology company with no approved products, gained the most ever after saying its stem cell treatment restored paralyzed rats ability to move in an early study.

Neuralstem rose 38 percent to $1.38 at the close of trading in New York, its largest single-day gain since the shares first started trading in December 2006. The Rockville, Maryland-based companys stock has gained 43 percent this year.

Researchers severed the spinal vertebrae of 12 rats, then gave half of them Neuralstems stem cells a week after the injury, according to the study published today in the journal Cell. The rats that received the injections gained significant locomotor recovery, according to a company statement.

The U.S. Food and Drug Administration placed a hold on Neuralstems proposed human trial to treat spinal cord injury in October 2010, according to a company filing.

We think that this paper is the last piece of the puzzle to get the FDA to take our spinal cord injury trial off hold, Richard Garr, chief executive officer, said in an interview.

Neuralstem also is testing its therapy in early human clinical trials for amyotrophic lateral sclerosis, known as Lou Gehrigs disease, and for depression.

To contact the reporter on this story: Ryan Flinn in San Francisco at rflinn@bloomberg.net

To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net

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Neuralstem Gains on Stem Cell Therapy for Paralyzed Rats