Coverage Summary: Geron Corporation Embryonic Stem Cell Clinical Trial

Below is a summary of media coverage from various sources of the recent announcement by Geron Corporation that it received federal regulatory approval from the Food and Drug Administration to begin resuming human clinical trials using human embryonic stem cells to attempt to treat spinal cord injuries:

New York Times, July 30, 2010, 11:21 AM EDT: "F.D.A. Clears Way for Embryonic Stem Cell Trial Using Patients":

The world’s first authorized test in people of a treatment derived from human embryonic stem cells has been cleared to begin by the Food and Drug Administration. The trial will test cells developed by Geron Corporation and the University of California, Irvine in patients with new spinal cord injuries.

United Press International, July 30, 2010 at 9:28 PM: "FDA: Stem cell trial can proceed":

The Food and Drug Administration has given approval to proceed with the world's first human clinical trial of a human embryonic stem cell-based therapy. Geron Corp., headquartered in Menlo Park, Calif., says it will proceed with its trial of GRNOPC1, a stem-cell therapy intended to treat patients with acute spinal cord injury, a company release said Friday.

Bloomberg News, July 30, 2010: "FDA: Stem cell trial can proceed":

Geron Corp. said it was cleared by U.S. regulators to proceed with the first human test of an embryonic stem-cell therapy, aimed at patients with spinal-cord injuries. The shares rose 17 percent. The Food and Drug Administration lifted a clinical hold on the study imposed last August when the company revealed that mice used in experimental work had developed cysts, Geron said in a statement. The company may start recruiting patients with new spinal cord injuries in about one month, said Thomas Okarma, Geron’s president and chief executive officer, in a telephone interview today.

The FDA’s action will allow the company to proceed with a long-awaited milestone -- the first authorized clinical trial in the world using stem cells derived from human embryos. The approval comes after almost a year of Geron’s testing and genetic analysis to resolve FDA questions, and paves the way for future trials.

San Jose Mercury News, July 31, 2010 : "FDA approves Geron's groundbreaking study of embryonic cells":

A Menlo Park biotech firm said Friday that federal regulators will let it proceed with the world's first human test of a treatment made from embryonic stem cells, a much-anticipated but controversial study of patients with spinal cord injuries that had been placed on hold for nearly a year because of safety concerns.

If the treatment from Geron works, it 'would be revolutionary,' said Dr. Richard Fessler, a neurological surgeon at Northwestern University, who will lead the study of a stem-cell treatment designed to be injected into patients with spinal injuries to restore their motor function. "The therapy would provide a viable treatment option for thousands of patients who suffer severe spinal cord injuries each year."

Associated Press, July 30, 2010: "Geron says FDA lifts hold on stem cell trial":

NEW YORK — Regulators on Friday gave the all-clear to a clinical trial that will test embryonic stem cells as a treatment for spinal cord injury, potentially the first time embryonic stem cells are tested on humans. The developer of the treatment, Geron Corp., said the Food and Drug Administration removed a clinical hold on its GRNOPC1 therapy. The FDA accepted Geron's study application in January 2009, which gave the company clearance to test GRNOPC1 on humans. But the FDA placed any potential study on hold in August because some mice treated with GRNOPC1 developed microscopic spinal cysts.

Geron hopes to start testing GRNOPC1 on humans by year-end. The company plans to enroll eight to 10 patients in the study at sites nationwide. The trial will take about two years, with each patient being studied for one year. Early-stage clinical trials are primarily designed to test a therapy's safety, although Geron said it will also measure the effectiveness of GRNOPC1.
A successful test would lead to larger and longer studies that would focus on the effectiveness of GRNOPC1. The company plans to continue monitoring patients for a total of 15 years for safety.

KGO-TV, San Francisco, CA, July 30, 2010: "Menlo Park-based Geron resumes stem cell trials":

MENLO PARK, CA (KGO) -- The Food and Drug Administration has given Menlo Park-based Geron the green light to resume trials of a stem cell treatment that could help repair injured spinal cords. The new drug by Geron will be injected into patients within seven days of a spinal cord injury.

Researchers Build New Joint with Stem Cells

Source: Columbia University
Date: July 30, 2010

Summary:

A pioneering study published "Online First" in The Lancet this week shows that failing joints can be replaced with a joint grown "naturally," using the host's own stem cells. The work paves the way for future joints that would last longer than today's artificial joints. The work was carried out in the Tissue Engineering and Regenerative Medicine Laboratory of Dr. Jeremy Mao, the Edward V. Zegarelli Professor at Columbia University, along with his team at Columbia University Medical Center, and colleagues from the University of Missouri and Clemson University.

Geron to Proceed with First Human Clinical Trial of Embryonic Stem Cell-Based Therapy

Source: Geron Corporation
Date: July 30, 2010

Summary:

Geron Corporation announced today that the U.S. Food and Drug Administration (FDA) has notified the company that the clinical hold placed on Geron's Investigational New Drug (IND) application has been lifted and the company's Phase I clinical trial of GRNOPC1 in patients with acute spinal cord injury may proceed.

The FDA notification enables Geron to move forward with the world's first clinical trial of a human embryonic stem cell (hESC)-based therapy in man. The Phase I multi-center trial is designed to establish the safety of GRNOPC1 in patients with "complete" American Spinal Injury Association (ASIA) Impairment Scale grade A subacute thoracic spinal cord injuries.

Scientists find cell of origin for human prostate cancer

Source: University of California - Los Angeles
Date: July 29, 2010

Summary:

University of California, Los Angeles scientists have identified for the first time a cell of origin for human prostate cancer, a discovery that could result in better predictive and diagnostic tools and the development of new and more effective targeted treatments for the disease. The researchers, from UCLA's Jonsson Comprehensive Cancer Center, proved that basal cells found in benign prostate tissue could become human prostate cancer in mice with suppressed immune systems, a finding that bucks conventional wisdom.

The study appears July 30 in the peer-reviewed journal Science.

Researchers Make Progress Toward Regenerating Tissue to Replace Joints

Source: National Institute of Biomedical Imaging and Bioengineering
Date: July 29, 2010

Summary:

A team of NIH-funded researchers has successfully regenerated rabbit joints using a cutting edge process to form the joint inside the body, or in vivo. Regenerative in vivo procedures are performed by stimulating previously irreparable organs or tissues to heal themselves. In this study, bioscaffolds, or three-dimensional structures made of biocompatible and biodegradable materials in the shape of the tissue, were infused with a protein to promote growth of the rabbit joint. The experiment demonstrated the feasibility of an approach to growing dissimilar tissues, such as cartilage and bone, derived entirely from the host’s own cells. Results of the study are in the July 29 issue of The Lancet.

Rabbits grow their own joint replacements in study

Source: Reuters
Posted: July 28, 2010 6:32pm EDT

Summary:

Reuters reports rabbits implanted with artificial bones re-grew their own joints:

Rabbits implanted with artificial bones re-grew their own joints, complete with cartilage, researchers reported on Thursday. Only a single compound called a growth factor was needed to induce the rabbits' bodies to remodel the joint tissue, said the team at Columbia University in New York, Clemson University in South Carolina and the University of Missouri.

Gene essential to stem cell health discovered

Source: University of Hawaiʻi at Mānoa
Date: July 28, 2010

Summary:

Researchers at the University of Hawai‘i at Mānoa’s John A. Burns School of Medicine (JABSOM) have discovered a gene that is essential to keeping stem cells healthy. The gene, hypoxia inducible factor 1, helps keep levels of telomerase constant. Telomerase is an enzyme that is critical to a stem cell’s lifespan, helping to prevent or slow deterioration in the cells. When telomerase is reduced in a stem cell, the stem cell ages faster. The research results are published in July’s online edition of the Proceedings of the National Academy of Sciences (PNAS).

Irradiating brain's stem cell niche doubles survival time for patients with brain cancers

Source: University of California - Los Angeles
Date: July 26, 2010

Summary:

Patients with deadly glioblastomas who received high doses of radiation that hit a portion of the brain that harbors neural stem cells had double the progression-free survival time as patients who had lower doses or no radiation targeting the area, a study from the Radiation Oncology Department at UCLA's Jonsson Comprehensive Cancer Center has found.

Patients who underwent high doses of radiation that hit the specific neural stem cell site, known as the stem cell niche, experienced 15 months of progression-free survival, while patients receiving lower or no doses to this region experienced 7.2 months of progression-free survival, said Dr. Frank Pajonk, an associate professor of radiation oncology, a cancer center researcher and senior author of the study.

Pajonk said the study, published in the early online edition of the journal BMC Cancer, could result in changes in the way radiation therapy is given to patients with these deadly brain cancers.

Scientists isolate the first stages of tissue production in human embryonic stem cells

Source: University of California - Los Angeles
Date: July 20, 2010

Summary:

Scientists at the UCLA Broad Stem Cell Research Center have described a population of cells that mark the very first stage of differentiation of human embryonic stem cells as they enter a developmental pathway that leads to production of blood, heart muscle, blood vessels and bone.

Researchers hope that these cells could one day be used for clinical treatments of a wide range of medical conditions as the discovery may help scientists create better and safer tissues for use in regenerative medicine. It also will allow scientists to better understand the differences between pluripotent stem cells, which can become every cell in the body, and cells that have lost their pluripotency and are on their way to becoming specific types of tissue cells.

The study appears today in the early online edition of the peer-reviewed journal Proceedings of the National Academy of Sciences.

Natural Substance NT-020 Aids Aging Brains in Rats, Study Finds

Source: University of South Florida
Date: July 19, 2010

Summary:

A combination of nutrients called NT-020 promoted adult neural stem cell proliferation in aged rats and boosted their memory performance, reported University of South Florida researchers studying natural therapeutic approaches to promoting the health of neurons in the aging brain.

Researchers from the USF Department of Neurosurgery and Brain Repair tested two groups of aged laboratory rats; one group received NT-020 and another, the control group, did not. In the NT-020 group, the process by which neurons are generated -- called neurogenesis -- increased. The study was published in the current issue of Rejuvenation Research (Vol. 13 No. 5, June, 2010). The NT-020 formula was patented by USF and licensed to Natura Therapeutics, Inc.

Reprogrammed Cells 'Remember,' Retain Characteristics of Their Cells of Origin

Source: Massachusetts General Hospital
Date: July 19, 2010

Summary:

Investigators at the Massachusetts General Hospital (MGH) Center for Regenerative Medicine have confirmed that induced pluripotent stem cells (iPSCs) retain some characteristics of the cells from which they were derived, something that could both assist and impede potential clinical and research uses. In their report that will be published in Nature Biotechnology and has received early online release, the researchers also describe finding that these cellular "memories" fade and disappear as cell lines are cultured through successive generations.

Unearthing King Tet: Key Protein Influences Stem Cell Fate

Source: University of North Carolina at Chapel Hill School of Medicine
Date: July 16, 2010

Summary:

Take a skin cell from a patient with Type 1 diabetes. Strip out everything that made it a skin cell, then reprogram it to grow into a colony of pancreatic beta cells. Implant these into your patient and voilà! She’s producing her own insulin like a pro.
This type of personalized therapy is the ultimate goal of most stem cell research. But to reliably achieve that goal for treating diabetes and other diseases, there’s a whole network of genes, proteins and miniscule chemical reactions to decipher first.
Findings published today in the journal Nature put us a step closer to untangling that web. University of North Carolina biochemist Yi Zhang, PhD and his team have discovered that a protein called Tet 1 helps stem cells renew themselves and stay pluripotent—able to become any type of cell in the body.

Blind Mice Can 'See' Thanks to Special Retinal Cells

Source: Johns Hopkins University
Date: July 15, 2010

Summary:

A study published July 15 in the journal Neuron, provides new hope to people who have severe vision impairments or who are blind. The study shows mice without rods and cones function can still see -- and not just light, but also patterns and images -- thanks to a third kind of photosensitive cell in the retina. Johns Hopkins University researchers found that mice that didn't have any rods and cones function could still see -- and not just light, but also patterns and images -- courtesy of special photosensitive cells in the rodents' retinas. Until now, it was presumed that those cells, called intrinsically photosensitive Retinal Ganglion Cells, (or ipRGCs), didn't play a role in image formation, but instead served other functions, such as dictating when the animals went to sleep or woke up. (All mammals, including humans, have ipRGCs, as well as rods and cones.)

Stanford Develops New Method To Grow Adult Stem Cells

Source: KGO AM 810 - San Francisco, CA
Date: July 15, 2010

Stanford researchers have come up with a better petri dish. KGO's Jenna Lane explains it's a special surface for growing stem cells.

New discovery brings hope to treatment of incurable blood cancer

Source: Uppsala University
Date: July 15, 2010

Summary:

Multiple myeloma is one of the most common blood cancers, and at present considered to be incurable. In a new study from Uppsala University, researchers now present a conceptually new model for the development and progression of multiple myeloma. The study was done in collaboration with Vrije Universitet Brussels and is published in the July edition of the on-line journal PLoS ONE.

Using large cohorts of myeloma patients the researchers have identified a profile of genes that are silenced by epigenetic mechanisms in the malignant plasma cell. The silenced gene profile was compared and contrasted to normal plasma cells, which are highly specialised and for which growth and lifetime is tightly controlled.

The silenced genes have a common denominator in being targets and controlled by the Polycomb repressor complex (PcG). This complex has previously been implicated in self-renewal and division of normal embryonic stem cells. In the study the researchers found that inhibitors of PcG also could decrease the growth of tumour cells in an animal model of myeloma.

Scientists develop new way to grow adult stem cells in culture

Source: Stanford University Medical Center
Date: July 15, 2010

Summary:

STANFORD, Calif. — Researchers at the Stanford University School of Medicine have developed a technique they believe will help scientists overcome a major hurdle to the use of adult stem cells for treating muscular dystrophy and other muscle-wasting disorders that accompany aging or disease: They've found that growing muscle stem cells on a specially developed synthetic matrix that mimics the elasticity of real muscle allows them to maintain their self-renewing properties.

Adult stem cells already exist in the body, and are important in regenerating tissues like blood, muscles and neurons in the brain. But scientists have struggled to produce them in quantities needed for therapies because the cells differentiate and lose their "stemness" as soon as they're placed in a tissue culture dish. This new method of growing the cells creates a way to study the behavior of many types of adult stem cells in culture and may revolutionize the ability to produce these cells for future therapies, say the researchers. The research will be published online July 15 in Science Express.

Researchers Reverse Cognitive Decline in Fruit Flies With Alzheimer’s Gene Mutation

Source: University of Pennsylvania School of Medicine
Date: July 15, 2010

Summary:

PHILADELPHIA – Investigators have found that fruit fly (Drosophila melanogaster) males -- in which the activity of an Alzheimer’s disease protein is reduced by 50 percent -- show impairments in learning and memory as they age. What’s more, the researchers were able to prevent the age-related deficits by treating the flies with drugs such as lithium, or by genetic manipulations that reduced nerve-cell signaling.

The research team -- Thomas A. Jongens, Ph.D., associate professor of Genetics at the University of Pennsylvania School of Medicine; Sean M. J. McBride M.D, Ph.D. and Thomas McDonald M.D., at the Albert Einstein College of Medicine; and Catherine Choi M.D., Ph.D. at Drexel University College of Medicine – worked with the familial form of Alzheimer’s disease (FAD), an aggressive form of the disease that is caused by mutations in one of the two copies of the presenilin (PS) or amyloid precursor protein (APP) genes. Studies in animal models have previously shown that the FAD-linked PS mutations lead to less presenilin (psn) protein activity.

Their findings are published in this week’s issue of the Journal of Neuroscience.

Stem cells to aid study of Parkinson's

Source: University of Oxford
Date: 15 July 2010

Summary:

A new stem cell technology is to be used by Oxford University researchers to better understand the causes of Parkinson’s disease. The technique will use skin samples to grow the brain cells thought to be responsible for the onset of Parkinson’s disease, allowing these important neurons to be studied in detail.

Researchers will gather data from over 1,000 patients with early stage Parkinson’s disease and take small samples of skin tissue to grow special stem cells – induced pluripotent stem cells (iPS cells). iPS cells can be generated from accessible tissue such as the skin and then used to generate specific types of cell. The researchers will use the iPS cells to grow dopamine neurons, the brain cells responsible for the production of dopamine. It is these cells which die in patients with Parkinson’s, leading to the onset of the disease.

Genetic mechanism once thought rare may allow rapid cell production

Source: Children's Hospital Boston
Date: July 8, 2010

Summary:

We take our blood for granted, but its creation requires a complicated series of steps, starting with the formation of blood stem cells during early embryonic development, followed by progressive differentiation into the progenitors of red cells, white cells and platelets, and ultimately the full set of blood cells. Now, in the July 9 issue of Cell, researchers at Children's Hospital Boston report a surprising twist in how mature red blood cells form - which may explain the body's ability to rapidly replenish them in response to injury.

Researchers identify factors behind blood-making stem cells

Source: University of Montreal
Date: July 6, 2010

Summary:

A team of researchers from the Institute for Research in Immunology and Cancer (IRIC) of the Université de Montréal have made significant progress in the understanding of blood-producing (hematopoietic) stem cells. The study led by IRIC Chief Executive Officer and Scientific Director, Dr. Guy Sauvageau, identifies factors that control the production of hematopoietic stem cells. Published in the journal Cell Stem Cell, the research offers interesting insight critical to the development of novel regenerative therapies and treatments for leukemia.

Neural stem cells attack glioblastoma cells

Source: Helmholtz Association of German Research Centres
Date: July 6, 2010

Summary:

In their latest research, scientists of the Max Delbruck Center for Molecular Medicine (MDC) Berlin-Buch, Germany, have demonstrated how the brain's own stem cells and precursor cells control the growth of glioblastomas. Of all brain tumors, glioblastomas are among the most common and most aggressive. Dr. Sridhar Reddy Chirasani, Professor Helmut Kettenmann and Dr. Rainer Glass have now shown in cell culture and mouse model experiments just how the body's own protective mechanism they identified in an earlier study, actually works (Brain, July 6, 2010).

Glioblastomas are brain tumors that are most common in adults in their mid-fifties or early sixties. The causes for developing the disease are not yet known. Researchers assume that misdirected neural stem cells / precursor cells mutate into cancer cells and can form glioblastomas.

Researchers Create HIV-Resistant Cells

Source: University of Southern California
Date: July 6, 2010

Summary:

Researchers at the Keck School of Medicine of USC successfully have transplanted blood stem cells modified to be resistant to HIV into mice, allowing the animals to control HIV infections. If the approach can be translated to human patients, it would enable the long-term generation of HIV-resistant T cells in a patient’s body, and the potential for the patient’s own cells to suppress HIV. The strategy is explained in a new study published online in the journal Nature Biotechnology.

The approach targets a gene called CCR5, one of the two gateway molecules that HIV uses to enter human cells. Cannon’s strategy arose from the observation that people with a mutation in a gene called CCR5 are naturally resistant to infection with the most common strains of HIV and do not develop AIDS.

The team used enzymes called zinc finger nucleases — which physically cut DNA — to knock out the the CCR5 gene in human blood stem cells. The researchers transplanted these modified stem cells into mice, where they developed into mature cells of the human immune system, including the T cells that HIV infects. When the researchers then infected the animals with HIV, they found that the mice were able to maintain normal levels of the human T cells and suppress HIV to very low levels, unlike control mice that received unmodified stem cells.

Biologists Find Way to Lower Tumor Risk in Stem Cell Therapies

Source: University of California - San Diego
Date: July 2, 2010

Summary:

One of the characteristics of embryonic stem cells is their ability to form unusual tumors called teratomas. These tumors, which contain a mixture of cells from a variety of tissues and organs of the body, are typically benign. But they present a major obstacle to the development of human embryonic stem cell therapies that seek to treat a variety of human ailments such as Parkinson’s, diabetes, genetic blood disorders and spinal cord injuries.

Now a team of biologists at UC San Diego funded by a grant from the California Institute for Regenerative Medicine, the state’s stem-cell funding agency, has discovered a way to limit the formation of teratomas. In this week’s issue of the Proceedings of the National Academy of Sciences, the researchers report that they have identified a new signaling pathway critical for unlimited self propagation of embryonic stem cells. Using small molecule compounds that inhibit this pathway, the scientists were able to dramatically reduce the potential of embryonic stem cells to form teratomas.

Scientists uncover important clues in the biology of stem cells

Source: Samuel Lunenfield Research Institute of Mount Sinai Hospital
Date: July 2, 2010

Summary:

Mount Sinai Hospital researchers including Drs. Andras Nagy and Jeff Wrana have discovered new insights into the genesis of stem cells, which will improve the efficiency of stem cell creation for use in tissue regeneration and in the development of new drugs. The study was published today in the leading biomedical journal Cell Stem Cell.

The goal of the study was to explore the process of changing fully mature cells of the body (known as somatic cells) into a pluripotent state (i.e., cells that can develop into most other cell types), and understand the molecular and genetic changes that occur during the cells’ reprogramming. Understanding this process will help researchers identify limitations in making induced pluripotent stem (iPS) cells, which are a source of great hope for use in regenerative medicine, as well as in the development of new drugs to prevent and treat various diseases.

Biologists discover how T cells make a commitment

Source: California Institute of Technology
Date: July 1, 2010

Summary:

PASADENA, Calif.—When does a cell decide its particular identity? According to biologists at the California Institute of Technology (Caltech), in the case of T cells—immune system cells that help destroy invading pathogens—the answer is when the cells begin expressing a particular gene called Bcl11b. The activation of Bcl11b is a "clean, nearly perfect indicator of when cells have decided to go on the T-cell pathway," says Ellen Rothenberg, the Albert Billings Ruddock Professor of Biology at Caltech and senior author of a paper about the discovery that appears in the July 2 issue of the journal Science.

Work-life balance: Brain stem cells need their rest, too

Source: Salk Institute for Biological Studies
Date: July 1, 2010

Summary:

LA JOLLA, CA—Stem cells in the brain remain dormant until called upon to divide and make more neurons. However, little has been known about the molecular guards that keep them quiet. Now scientists from the Salk Institute for Biological Studies have identified the signal that prevents stem cells from proliferating, protecting the brain against too much cell division and ensuring a pool of neural stem cells that lasts a lifetime.

The research, which will be published in the July 1 issue of Cell Stem Cell, highlights the importance of bone morphogenetic factor protein (BMP) signaling for the maintenance of a neural stem cell reservoir throughout adult life and may provide the key to understanding the interplay between exercise, aging and neurogenesis.

Gene regulating human brain development identified

Source: University of Wisconsin-Madison
Date: July 1, 2010

Summary:

With more than 100 billion neurons and billions of other specialized cells, the human brain is a marvel of nature. It is the organ that makes people unique. Now, writing in the journal Cell Stem Cell (July 1, 2010), a team of scientists from the University of Wisconsin-Madison has identified a single gene that seems to be a master regulator of human brain development, guiding undifferentiated stem cells down tightly defined pathways to becoming all of the many types of cells that make up the brain.

The new finding is important because it reveals the main genetic factor responsible for instructing cells at the earliest stages of embryonic development to become the cells of the brain and spinal cord. Identifying the gene — known as Pax6 — is a first critical step toward routinely forging customized brain cells in the lab. What's more, the work contrasts with findings from animal models such as the mouse and zebrafish, pillars of developmental biology, and thus helps cement the importance of the models being developed from human embryonic stem cells.

Patients With Treatment-Resistant Chronic Leukemia Respond Positively to Stem Cell Transplants

Source: American Society of Hematology.
Date: July 1, 2010

Summary:

(WASHINGTON) – Allogeneic (donor-derived) stem cell transplant (alloSCT) may be a promising option for patients with treatment-resistant chronic lymphocytic leukemia (CLL), regardless of the patient’s underlying genetic abnormalities, according to the results of a study published online today in Blood, the journal of the American Society of Hematology.

In alloSCT, blood stem cells are collected from a donor and then infused into the patient where they travel to the bone marrow and begin to produce new blood cells, replacing those that have been affected as a result of the disease. This type of treatment can pose serious complications, some of which are potentially fatal. In this prospective phase II study, a total of 90 patients with treatment-resistant CLL received alloSCT, and stem cell donors were either healthy siblings or unrelated, but matched, volunteers.

Prior to the transplant, patients in this study received conditioning, a standard therapy administered immediately before a stem cell transplant to help prepare the body to receive and accept the transplanted cells. The research team used a reduced-intensity conditioning approach with two common chemotherapies (fludarabine and cyclophosphamide) to reduce complications and allow the donor stem cells to fight the disease themselves.

After treatment with alloSCT, more than 40 percent of participants with this otherwise fatal disease enjoyed long-term freedom from relapse. These findings suggest that alloSCT is a feasible and potentially curative treatment for patients with high-risk CLL and should be considered for this patient population.