HIV Positive Men Show No Signs of HIV after Bone Marrow Transplant and Discontinuation of Anti-Retroviral Therapy

Source: Brigham and Women's Hospital

Date: July 3, 2013

Summary:

Boston, MA - Two Brigham and Women's Hospital patients with longstanding HIV infections who underwent bone marrow transplants have stopped anti-retroviral therapy and have no detectable HIV in their blood cells.  One patient stopped anti-retroviral therapy 15 weeks ago, the other stopped 7 weeks ago.  These new findings will be presented on July 3, 2013 at the International AIDS Society Conference (IAS 2013) in Kuala Lumpur, Malaysia by Timothy Henrich, MD and Daniel Kuritzkes, MD, physician-researchers in the Division of Infectious Diseases at Brigham and Women's Hospital.

StemCells, Inc. Announces First Patient Cohort Completes Spinal Cord Injury Trial - Gains in Sensory Function Persist 12 Months After Stem Cell Transplant

Source: StemCells, Inc.
Date: February 12, 2013

Summary:

NEWARK, Calif. -- StemCells, Inc. today announced that the twelve-month data from the first patient cohort in the Company's Phase I/II clinical trial of its proprietary HuCNS-SC® product candidate (purified human neural stem cells) for chronic spinal cord injury continued to demonstrate a favorable safety profile, and showed that the considerable gains in sensory function observed in two of the three patients at the six-month assessment have persisted. The third patient remains stable. A summary of the data was presented today by Martin McGlynn, President and CEO, at the 15th Annual BIO CEO & Investor Conference. By completing the twelve-month assessment, the first patient cohort has now completed the trial, and has entered into a separate follow-up study for long-term observation.

Neuralstem Receives FDA Approval To Commence Spinal Cord Injury Trial

Source: Neuralstem, Inc.
Date: January 14, 2013

Summary:

ROCKVILLE, Md. -- Neuralstem, Inc. announced that it received approval from the United States Food and Drug Administration (FDA) to commence a Phase I safety trial of its lead cell therapy candidate, NSI-566, in chronic spinal cord injury patients. This open-label, multi-site study, will enroll up to eight patients with thoracic spinal cord injuries (T2-T12), who have an American Spinal Injury Association (AIS) A level of impairment, between one and two years after injury. AIS A impairment refers to a patient with no motor or sensory function in the relevant segments at and below the injury, and is considered to be complete paralysis.

The primary objective of the study is to determine the safety and toxicity of human spinal stem cell transplantation for the treatment of paralysis and related symptoms due to chronic spinal cord injury (SCI). The secondary objectives of the study are to evaluate graft survival in the transplant site by MRI, as well as the effectiveness of transient immunosuppression.

StemCells, Inc. Announces First Transplant of Neural Stem Cells Into Patient in Clinical Trial for Dry Age-Related Macular Degeneration

Source: StemCells, Inc.
Date: October 4, 2012

 Summary:

 NEWARK, Calif. -- StemCells, Inc. today announced that the first patient in its Phase I/II clinical trial in dry age-related macular degeneration (AMD) has been enrolled and transplanted. The trial is designed to evaluate the safety and preliminary efficacy of the Company's proprietary HuCNS-SC® product candidate (purified human neural stem cells) as a treatment for dry AMD, and the patient was transplanted with the cells yesterday at the Retina Foundation of the Southwest (RFSW) in Dallas, Texas, one of the leading independent vision research centers in the United States. AMD afflicts approximately 30 million people worldwide and is the leading cause of vision loss and blindness in people over 55 years of age.

Neuralstem Cells Induce Significant Functional Improvement In Permanent Rat Spinal Cord Injury, Cell Study Reports

Source: Neuralstem, Inc.
Date: September 13, 2012

Summary;

ROCKVILLE, Md. -- Neuralstem, Inc. announced that its neural stem cells were part of a study, "Long-Distance Growth and Connectivity of Neural Stem Cells After Severe Spinal Cord Injury: Cell-Intrinsic Mechanisms Overcome Spinal Inhibition," published online today in a leading scientific journal CELL. In the study, rats with surgically transected spinal cords, which rendered them permanently and completely paraplegic, were transplanted with Neuralstem's spinal cord stem cells (NSI-566). The study reports that the animals recovered significant locomotor function, regaining movement in all lower extremity joints, and that the transplanted neural stem cells turned into neurons which grew a "remarkable" number of axons that extended for "very long distances" over 17 spinal segments, making connections both above and below the point of severance. These axons reached up to the cervical region (C4) and down to the lumbar region (L1). They also appeared to make reciprocal synaptic connectivity with the host rat spinal cord neurons in the gray matter for several segments below the injury.

Further study showed that re-transecting the spinal cord immediately above the graft abolished the functional gain, indicating that the regeneration of host axons into the human stem cell graft was responsible for the functional recovery. The cells that Neuralstem contributed to the study, NSI-566, are the same cells used in the recently completed Phase 1 clinical trial for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). Neuralstem has also submitted an application to the FDA for a trial to treat chronic spinal cord injury with these cells.

Stem Cell Researchers Use Gene Therapy to Restore Immune Systems in 'Bubble Boy' Disease

Source: University of California, Los Angeles (UCLA), Health Sciences
Date: September 11, 2012

Summary:

UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

StemCells, Inc. Reports Positive Interim Data From Spinal Cord Injury Trial Cells and Procedure Well Tolerated; Gains in Sensory Function Confirmed

Source: StemCells, Inc.
Date: September 3, 2012

Summary:

NEWARK, Calif. -- StemCells, Inc. today announced that interim six-month data from the first patient cohort in the Company's Phase I/II clinical trial of its proprietary HuCNS-SC® product candidate (purified human neural stem cells) for chronic spinal cord injury continues to demonstrate a favorable safety profile, and shows considerable gains in sensory function in two of the three patients compared to pre-transplant baselines. The third patient remains stable. The data was presented by Armin Curt, M.D., principal investigator for the clinical trial, at the 51st Annual Scientific Meeting of the International Spinal Cord Society in London, England. The trial represents the first time that neural stem cells have been transplanted as a potential therapeutic agent for spinal cord injury.

Patients in the study's first cohort all suffered a complete injury to the thoracic (chest-level) spinal cord. In a complete injury, there is no neurological function below the level of injury. All three patients were transplanted four to nine months after injury with a dose of 20 million cells at the site of injury. The surgery, immunosuppression and the cell transplants have been well tolerated by all the patients. There were no abnormal clinical, electrophysiological or radiological responses to the cells, and all the patients were neurologically stable through the first six months following transplantation. Changes in sensitivity to touch, heat and electrical stimuli were observed in well-defined and consistent areas below the level of injury in two of the patients, while no changes were observed in the third patient. Importantly, tests of perception of different sensory stimuli as well as measures of electrical impulse transmission across the site of injury correlate with the clinical examination, providing independent and objective confirmation of the changes in sensory function.

Neuralstem Updates ALS Stem Cell Trial Progress; Emory University Institutional Review Board Approves Amendment

Source: Neuralstem, Inc.
Date: June 5, 2012

Summary:

ROCKVILLE, Md., /PRNewswire/ -- Neuralstem, Inc. announced that the Emory University Institutional Review Board (IRB) approved the amendment to the ongoing Phase I trial evaluating Neuralstem's spinal cord stem cells in the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease). The amendment permits the return of three previously-treated patients to the trial to receive additional injections of cells. This modification to the protocol was approved earlier by the Food and Drug Administration (FDA). Implementation was contingent upon IRB approval, which has now been secured.

Genetically Modified T Cell Therapy Shown to be Safe, Lasting in Decade-Long Penn Medicine Study of HIV Patients

Source: University of Pennsylvania School of Medicine
Date: May 2, 2012

Summary:

HIV patients treated with genetically modified T cells remain healthy up to 11 years after initial therapy, researchers from the Perelman School of Medicine at the University of Pennsylvania report in the new issue of Science Translational Medicine. The results provide a framework for the use of this type of gene therapy as a powerful weapon in the treatment of HIV, cancer, and a wide variety of other diseases.

Stem Cells from Pelvic Bone May Preserve Heart Function

Source: Orlando Health
Date: April 11, 2012

Summary:

Stem cells from the pelvic bone may help hearts beat stronger. Doctors and other clinicians at the Orlando Health Heart Institute are researching the use of stem cells from pelvic bone marrow to restore tissue and improve heart function after muscle damage from heart attacks.

StemCells, Inc's Milestone Pelizaeus-Merzbacher Disease Clinical Trial Shows Evidence of Myelination Following Human Neural Stem Cell Transplantation

Source: StemCells, Inc.
Date: April 2, 2012

Summary:

NEWARK, Calif. -- StemCells, Inc. today announced preliminary evidence of progressive and durable donor-cell derived myelination in all four patients who underwent transplantation with the Company's proprietary HuCNS-SC® cells (purified human neural stem cells) in its clinical trial for Pelizaeus-Merzbacher disease (PMD), a rare hypo-myelination disorder in children. In addition, clinical assessment revealed small but measureable gains in motor and/or cognitive function in three of the four patients; the fourth patient remained clinically stable. The study was conducted by researchers at the University of California, San Francisco (UCSF).

Here's a link to a conference call held by Stem Cells, Inc. today discussing the results of the trial.

SANBIO ANNOUNCES ENROLLMENT OF FIRST COHORT OF PATIENTS IN ITS CLINICAL TRIAL OF STEM CELL THERAPY FOR CHRONIC STROKE

Source: SanBio Inc.
Date: March 20, 2012

Summary:

SanBio Inc. today announced the successful enrollment of the first dose cohort of patients in its Phase 1/2a clinical trial testing the safety and efficacy of a novel allogeneic stem cell therapy product, SB623, a cell therapy product consisting of cells derived from genetically engineered bone marrow stromal cells obtained from healthy adult donors, in patients suffering from chronic deficits resulting from previous stroke injuries. The first 6 patients, of a total of 18, have been successfully administered SB623. The trial is being conducted at Stanford University and the University of Pittsburgh. No safety concerns have been reported. Details of this clinical trial can be found here.

New transplant method may let kidney recipients live life free of anti-rejection medication

Source: University of Louisville / Northwestern Medicine
Date: March 7, 2012

Summary:

New ongoing research published March 7 in the journal Science Translational Medicine suggests organ transplant recipients may not require anti-rejection medication in the future thanks to the power of stem cells, which may prove to be able to be manipulated in mismatched kidney donor and recipient pairs to allow for successful transplantation without immunosuppressive drugs. Northwestern Medicine® and University of Louisville researchers are partnering on a clinical trial to study the use of donor stem cell infusions that have been specially engineered to “trick” the recipients’ immune system into thinking the donated organ is part of the patient’s natural self, thus gradually eliminating or reducing the need for anti-rejection medication.

Reuters published a news story on this finding today.

Fourteenth Patient Dosed in Neuralstem ALS Stem Cell Trial

Source: Neuralstem, Inc.
Date: March 7, 2012

Summary:

ROCKVILLE, Md., March 7, 2012 /PRNewswire/ -- Neuralstem, Inc. 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 could support 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.

Investigational human adult stem cell therapy studied in ischemic stroke patients

Source: Methodist Hospital, Houston
Date: March 6, 2012

Summary:

Houston, TX - Physicians at the Methodist Neurological Institute are studying the use of human stem cells as a possible treatment for acute ischemic stroke, a leading cause of death and disability. Each year, stroke affects more than 15 million people around the world. Patients whose ischemic strokes occur within one to two days of being admitted to The Methodist Hospital in Houston may be eligible to enroll in the double-blind, randomized, placebo-controlled phase 2 safety and efficacy trial of MultiStem®, a novel therapy being developed by Athersys, Inc.

The study will examine the effects of intravenous administration of adult stem cells that can be manufactured from a donor. In contrast to traditional bone marrow transplants, which require one donor for each patient that needs treatment, MultiStem is a patented formulation of early adult stem cells, and hundreds of thousands to millions of doses can be made from the bone marrow cells of one healthy donor. The product can be made in advance, and may be stored in the hospital and used “off the shelf”.

Researchers in the clinical trial will not only look at how well the investigational therapy works for stroke treatment, but they will also monitor for potential side effects and how potent the drug is compared to placebo.

Another goal of this study is to examine some of the stem cells’ effects on organs such as the spleen, which is thought to contribute to ongoing inflammation that could increase brain injury after the initial stroke. Published work from preclinical studies shows that MultiStem can provide benefits even when administered several days after a stroke has occurred, and some of the cell effects appear to occur through their action on the spleen. Animal models used in this research showed a statistically significant and durable improvement in motor skills relative to animals that received a placebo.

StemCells, Inc. Receives FDA Authorization for Age-Related Macular Degeneration Clinical Trial

Source: StemCells, Inc.
Date: February 2, 2012

Summary:

NEWARK, Calif. -- StemCells, Inc. today announced that the U.S. Food and Drug Administration (FDA) has authorized the initiation of a Phase I/II clinical trial of the Company's proprietary HuCNS-SC® product candidate (purified human neural stem cells) in dry age-related macular degeneration (AMD), the most common form of AMD. AMD is the leading cause of vision loss and blindness in people over 55 years of age, and approximately 30 million people worldwide are afflicted with the disease. There are no approved treatments for dry AMD.

The Phase I/II trial will evaluate the safety and preliminary efficacy of HuCNS-SC cells as a treatment for dry AMD. The trial will be an open-label, dose-escalation study, and is expected to enroll a total of 16 patients. The HuCNS-SC cells will be administered by a single injection into the space beneath the retina. Patients' vision will be evaluated using conventional methods of ophthalmological assessment at predetermined intervals over a one-year period. Patients will then be followed for an additional four years in a separate observational study.

Preclinical data submitted as part of the Company's Investigative New Drug application demonstrated that HuCNS-SC cells protect host photoreceptors and preserve vision in a well-established animal model of retinal disease that is relevant to dry AMD. HuCNS-SC transplants significantly protect against the degeneration of photoreceptors, the key cells of the eye involved in vision. Moreover, the number of cone photoreceptors, which are responsible for central vision, remain constant over an extended period, consistent with the sustained visual acuity and light sensitivity observed. In humans, degeneration of the cone photoreceptors account for the unique pattern of visual loss in dry AMD. A summary of the Company's preclinical data was published in the February issue of the international peer-reviewed European Journal of Neuroscience.

Scientists perform Ontario's first cardiac stem cell transplant

Source: University of Toronto
Date: January 27, 2012

Summary:

University of Toronto faculty members performed the first cardiac stem cell transplant in Ontario recently, using stem cells derived from the patient's own bone marrow, isolated completely within the operating room, and implanted into the heart at the time of coronary bypass surgery. The stem cells were injected following coronary artery bypass graft (CABG) surgery, by a multi-disciplinary team led by Dr. Terrence Yau, a U of T professor of surgery and director of the Cardiac Stem Cell Therapy Program at the hospital’s Peter Munk Cardiac Centre, part of the University Health Network (UHN).

The Toronto Star published a news story on the trial yesterday.

COVERAGE SUMMARY: ACT Publishes First Report of Embryonic Stem Cell (ESC)-Derived Cells Transplanted Into Patients

Below is a summary of media coverage of the announcement by Advanced Cell Technology Inc. that Phase 1/2 clinical data published in The Lancet as an early online publication demonstrate the safety of ACT’s human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) cells for the treatment of Stargardt’s macular dystrophy (SMD) and dry age-related macular degeneration (dry AMD):

Washington Post, January 23, 2012: "Embryonic stem cells appear to restore some vision to legally blind patient":

For the first time, an experimental treatment made from human embryonic stem cells has shown evidence of helping someone, partially restoring sight to two people suffering from slowly progressing forms of blindness. Although the purpose of the experiment was to test the safety of stem cells injected into the eye, both patients “had measurable improvement in their vision that persisted through the duration of the study,” said Robert Lanza, chief scientific officer at Advanced Cell Technology, the Massachusetts biotech company that sponsored the closely watched experiment.

NPR, All Things Considered, January 23, 2012, 11:46 am EST: "First Hints That Stem Cells Can Help Patients Get Better":

Two women losing their sight to progressive forms of blindness may have regained some vision while participating in an experiment testing a treatment made from human embryonic stem cells, researchers reported today. The report marks the first time that scientists have produced direct evidence that human embryonic stem cells may have helped a patient. The cells had only previously been tested in the laboratory or in animals.

Reuters, January 23, 2012 6:32 pm EST: "First patients shown to improve with embryonic stem cells":

Before treatment, the 51-year-old graphic artist was legally blind, unable to read a single letter on a standard eye chart. She has suffered from Stargardt's disease, the most common form of macular degeneration in young patients, since she was a teenager, and it was getting progressively worse.

A second patient, aged 78, suffered from dry macular degeneration - the leading cause of blindness in the elderly -and could not even see well enough to go shopping. But after being treated with stem cells from a donated human embryo, both women have improved dramatically, researchers said on Monday. Stem cells are master cells that can differentiate into any of the 200 kinds of cells in the human body. Their results are the first-ever report of the medical use of stem cells taken from human embryos, making them crucial barometers of whether the controversial technique will ever find widespread therapeutic uses.

Associated Press, January 23, 2012: "Study: Stem cells may aid vision in blind people":

Two legally blind women appeared to gain some vision after receiving an experimental treatment using embryonic stem cells, scientists reported Monday. While embryonic stem cells were first isolated more than a decade ago, most of the research has been done in lab animals. The new results come from the first tests in humans for a vision problem. Researchers caution the work is still very preliminary.

USA Today, January 23, 2012: "Stem cells offer first glimpse of blindness treatment":

In the first published results of a therapy using human embryonic stem cells, the controversial treatment slightly improved the vision of two legally blind patients, researchers reported Monday. Intended as a safety test, the experiment reported in the journal The Lancet follows more than a decade of debate over human embryonic stem cells. Seen as a potential treatment of diseases such as diabetes and blindness, the therapy has also drawn criticism from conservative groups that have decried the cells' origins — removed from early human embryos that are destroyed in the process, and then grown into colonies for research.

New York Times, January 23, 2012: "Stem Cell Treatment for Eye Diseases Shows Promise":

A treatment for eye diseases that is derived from human embryonic stem cells might have improved the vision of two patients, bolstering the beleaguered field, researchers reported Monday. The report, published online in the medical journal The Lancet, is the first to describe the effect on patients of a therapy involving human embryonic stem cells.

Daily Telegraph, 23 January 2012 4:18PM GMT: "Human stem cell therapy works in blind patients in first trial":

Scientists have improved the sight of two people who were almost blind by injecting their eyes with stem cells from embryos. Marcus Hilton is the first person in Europe to take part in stem cell trial for a rare eye disease. The two women, both registered as blind, saw their vision improve in a matter of weeks after being given the embryo-derived cells in the US safety trial. The breakthrough holds out the hope of a cure in the future for age-related macular degeneration, which currently affects some 500,000 people in Britain. The results, published this week in The Lancet, provide a major boost for the field of stem cell research.

Toronto Star, January 23, 2012: "Can stem cells cure blindness?":

Two women who are legally blind appear to have some vision restored after being treated with stem cells from a donated human embryo, scientists reported Monday. One patient, a 51-year-old graphic artist with Stargardt’s macular dystrophy, the most common form of macular degeneration in young patients, reported seeing a greater range of colours and felt comfortable riding a bike after the treatment. More crucially, the scientists reported no adverse affects or safety concerns in either woman four months after the experimental therapy. The study is the first-ever to report on the medical use of human embryonic stem cells in patients for the treatment of any disease.

HealthDay News, January 23, 2012: "Small Stem Cell Study Claims Early Success in Treating Eye Disease: Two patients appeared to benefit from therapy for macular degeneration":

Using human embryonic stem cells to treat the eye disease macular degeneration appears to be safe and leads to some vision improvement, a small, early-stage study found. The study included one elderly patient and one younger adult patient with different types of macular degeneration that had led to severe vision loss.

After four months, the embryonic stem cell transplants seemed safe and both patients had some improvement in vision, the U.S. researchers said. The study, published online Jan. 23 in The Lancet, is the first report of the use of human embryonic stem cells (hESC) in humans for any purpose, the researchers said in a news release from the journal.

ACT Publishes First Report of Embryonic Stem Cell (ESC)-Derived Cells Transplanted Into Patients

Source: Advanced Cell Technology, Inc.
Date: January 23, 2012

Summary:

MARLBOROUGH, Mass. —– Advanced Cell Technology, Inc., a leader in the field of regenerative medicine, announced today that Phase 1/2 clinical data published in The Lancet as an early online publication demonstrate the safety of ACT’s human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) cells for the treatment of Stargardt’s macular dystrophy (SMD) and dry age-related macular degeneration (dry AMD). Results were reported for two patients, the first in each of the Phase 1/2 clinical trials. In addition to showing no adverse safety issues, structural evidence confirmed that the hESC-derived cells survived and continued to persist during the study period reported. Both patients had measurable improvements in their vision that persisted for more than four months.

At four months following treatment, no hyperproliferation, tumorigenicity, ectopic tissue formation, or apparent rejection were observed in either patient at any time. Detailed clinical and diagnostic laboratory assessments were performed at multiple post-transplantation evaluations. Abnormal growth (or tumor formation) would be considered a significant safety concern for stem-cell based therapies, in particular those derived from hESCs due to their pluripotency; it is therefore critical to control the differentiation of hESCs. Results reported indicate that stem cell differentiation was well controlled in these patients. No adverse safety signals were detected.

BrainStorm Announces Clinical Data Supporting Safety and Efficacy of NurOwn™ Based on Initial Patient Results

Source: BrainStorm Cell Therapeutics Inc.
Date: January 17, 2012

Summary:

BrainStorm Cell Therapeutics Inc., an innovative developer of adult stem cell technologies and Central Nervous System (CNS) therapeutics, today announced that the data from the initial patients in its ALS Phase I/II human clinical trial treated with its NurOwn™ technology did not present any significant side effects and that the NurOwn™ treatment has so far proven to be safe. Prof. Dimitrios Karussis, who is leading the clinical trial at Hadassah Medical Center, stated, “There have been no significant side effects in the initial patients we have treated with BrainStorm’s NurOwn™ technology. In addition, even though we are conducting a safety trial, the early clinical follow up of the patients treated with the stem cells shows indications of beneficial clinical effects, such as an improvement in breathing and swallowing ability as well as in muscular power. I am very excited about the safety results, as well as these indications of efficacy, we are seeing. This may represent the biggest hope in this field of degenerative diseases, like ALS.”

After reviewing the safety data from the first four patients, the Hadassah Medical Center ethical committee granted approval for the trial to advance to transplanting the next patients.