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Category Archives: Cell Medicine
Gene therapy lets a French teen dodge sickle cell disease – Medical Xpress
Posted: March 1, 2017 at 11:41 pm
March 1, 2017 by Marilynn Marchione This 2009 colorized microscope image made available by the Sickle Cell Foundation of Georgia via the Centers for Disease Control and Prevention shows a sickle cell, left, and normal red blood cells of a patient with sickle cell anemia. Researchers say a French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder. The case is detailed in the March 2, 2017 issue of the New England Journal of Medicine. (Janice Haney Carr/CDC/Sickle Cell Foundation of Georgia via AP)
A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.
The first-in-the-world case is detailed in Thursday's New England Journal of Medicine.
About 90,000 people in the U.S., mostly blacks, have sickle cell, the first disease for which a molecular cause was found. Worldwide, about 275,000 babies are born with it each year.
"Vexing questions of race and stigma have shadowed the history of its medical treatment," including a time when blacks who carry the bad gene were urged not to have children, spurring accusations of genocide, Keith Wailoo of Princeton University wrote in a separate article in the journal.
The disease is caused by a single typo in the DNA alphabet of the gene for hemoglobin, the stuff in red blood cells that carries oxygen. When it's defective, the cells sickle into a crescent shape, clogging tiny blood vessels and causing bouts of extreme pain and sometimes more serious problems such as strokes and organ damage. It keeps many people from playing sports and enjoying other activities of normal life.
A stem cell transplant from a blood-matched sibling is a potential cure, but in the U.S., fewer than one in five people have a donor like that. Pain crises are treated with blood transfusions and drugs, but they're a temporary fix. Gene therapy offers hope of a lasting one.
The boy, now 15, was treated at Necker Children's Hospital in Paris in October 2014. Researchers gave him a gene, taken up by his blood stem cells, to help prevent the sickling. Now, about half of his red blood cells have normal hemoglobin; he has not needed a transfusion since three months after his treatment and is off all medicines.
"It's not a cure but it doesn't matter," because the disease is effectively dodged, said Philippe Leboulch, who helped invent the therapy and helped found Bluebird Bio in Cambridge, Massachusetts, the company that treated the boy. The work was supported by a grant from the French government's research agency.
Bluebird has treated at least six others in the U.S. and France. Full results have not been reported, but the gene therapy has not taken hold as well in some of them as it did in the French teen. Researchers think they know why and are adjusting methods to try to do better.
Two other gene therapy studies for sickle cell are underway in the U.S.at the University of California, Los Angeles and Cincinnati Children's Hospitaland another is about to start at Harvard and Boston Children's Hospital using a little different approach.
"This work gives considerable promise" for a solution to a very common problem, said Dr. Stuart Orkin, a Boston Children's Hospital doctor who is an inventor on a patent related to gene editing.
"The results are quite good in this patient," he said of the French teen. "It shows gene therapy is on the right track."
Explore further: BCL11A-based gene therapy for sickle cell disease passes key preclinical test
More information: Gene therapy: ghr.nlm.nih.gov/primer/therapy/availability
2017 The Associated Press. All rights reserved.
A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children's ...
Sickle cell disease and the blood disorder beta thalassemia affect more than 180,000 Americans and millions more worldwide. Both diseases can be made milder or even cured by increasing fetal hemoglobin (HbF) levels, but current ...
Scientists at the Center for Regenerative Medicine (CReM) at Boston Medical Center (BMC) and Boston University School of Medicine (BUSM) are creating an induced pluripotent stem cell (iPSC)-based research library that opens ...
UCLA stem cell researchers have shown that a novel stem cell gene therapy method could lead to a one-time, lasting treatment for sickle cell diseasethe nation's most common inherited blood disorder.
A team of researchers at the Stanford University School of Medicine has used a gene-editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward ...
Scientists have developed a new approach to repair a defective gene in blood-forming stem cells from patients with a rare genetic immunodeficiency disorder called X-linked chronic granulomatous disease (X-CGD). After transplant ...
A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.
A research team, led by the University of Minnesota, has discovered a groundbreaking process to successfully rewarm large-scale animal heart valves and blood vessels preserved at very low temperatures. The discovery is a ...
Working with yeast and human cells, researchers at Johns Hopkins say they have discovered an unexpected route for cells to eliminate protein clumps that may sometimes be the molecular equivalent of throwing too much or the ...
By changing one small portion of a stimulus that influences part of one molecule's function, engineers and researchers at Washington University in St. Louis have opened the door for more insight into how the molecule is associated ...
A minimally invasive, fiber-optic technique that accurately measures the passive stretch and twitch contraction of living muscle tissue could someday be an alternative to the painful muscle biopsies used to diagnose and treat ...
An in-depth computational analysis of genetic variants implicated in both schizophrenia and rheumatoid arthritis by researchers at the University of Pittsburgh points to eight genes that may explain why susceptibility to ...
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Gene therapy lets a French teen dodge sickle cell disease - Medical Xpress
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In cleaning up misfolded proteins, cell powerhouses can break down – Medical Xpress
Posted: March 1, 2017 at 11:41 pm
March 1, 2017 Mitochondria (red) that have imported misfolded proteins (green). Credit: Erli Jin and Linhao Ruan/Johns Hopkins Medicine
Working with yeast and human cells, researchers at Johns Hopkins say they have discovered an unexpected route for cells to eliminate protein clumps that may sometimes be the molecular equivalent of throwing too much or the wrong trash into the garbage disposal. Their finding, they say, could help explain part of what goes awry in the progression of such neurodegenerative diseases as Parkinson's and Alzheimer's.
Proteins in the cell that are damaged or folded incorrectly tend to form clumps or aggregates, which have been thought to dissolve gradually in a cell's cytoplasm or nucleus thanks to an enzyme complex called the proteasome, or in a digestive organelle called the lysosome.
But in experiments on yeast, which has many structures similar to those in human cells, the Johns Hopkins scientists unexpectedly found that many of those protein clumps break down in the cell's energy-producing powerhouses, called mitochondria. They also found that too many misfolded proteins can clog up and damage this vital structure.
The team's findings, described March 1 in Nature, could help explain why protein clumping and mitochondrial deterioration are both hallmarks of neurodegenerative diseases.
Rong Li, Ph.D., professor of cell biology, biomedical engineering and oncology at the Johns Hopkins University School of Medicine and a member of the Johns Hopkins Kimmel Cancer Center, who led the study, likens the disposal system to the interplay between a household's trash and a garbage disposal in the kitchen sink. The disposal is handy and helps keep the house free of food scraps, but the danger is that with too much trash, especially tough-to-grind garbage, the system could get clogged up or break down.
In a previous study, Li and her team found protein aggregates, which form abundantly under stressful conditions, such as intense heat, stuck to the outer surface of mitochondria. In this study, they found the aggregates bind to proteins that form the pores mitochondria normally use to import proteins needed to build this organelle. If these pores are damaged by mutations, then aggregates cannot be dissolved, the researchers report. These observations led the team to hypothesize that misfolded proteins in the aggregates are pulled into mitochondria for disposal, much like food scraps dropped into the garbage disposal. Testing this hypothesis was tricky, Li says, because most of the misfolded proteins started out in the cytoplasm, and most of those that enter mitochondria quickly get ground up.
As a consequence, Li and her team used a technique in which a fluorescent protein was split into two parts. Then, they put one part inside the mitochondria and linked the other part with a misfolded and clumping protein in the cytoplasm. If the misfolded protein entered the mitochondria, the two parts of the fluorescent protein could come together and light up the mitochondria. This was indeed what happened.
"With any experiment," Li says, "you have a hypothesis, but in your head, you may be skeptical, so seeing the bright mitochondria was an enlightening moment."
To see what might happen in a diseased system, the team then put into yeast cells a protein implicated in the neurodegenerative disease known as amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease. After a heat treatment that caused the ALS protein to misfold, it also wound up in the mitochondria. The researchers then did an experiment in which a lot of proteins in the cytoplasm were made to misfold and found that when too much of these proteins entered mitochondria, they started to break down.
The team wanted to make sure that the phenomenon it had observed in the yeast cells could also happen in human cells, so the scientists used the same split-fluorescent protein method to observe misfolded proteins to enter the mitochondria of lab-grown human retinal pigmented epithelial cells. As observed in yeast, misfolded proteins, but not those that were properly folded, entered and lit up mitochondria.
Biological systems are in general quite robust, but there are also some Achilles' heels that may be disease prone, Li says, and relying on the mitochondrial system to help with cleanup may be one such example. While young and healthy mitochondria may be fully up to the task, aged mitochondria or those overwhelmed by too much cleanup in troubled cells may suffer damage, which could then impair many of their other vital functions.
Explore further: Cell disposal faults could contribute to Parkinson's, study finds
More information: Linhao Ruan et al, Cytosolic proteostasis through importing of misfolded proteins into mitochondria, Nature (2017). DOI: 10.1038/nature21695
A fault with the natural waste disposal system that helps to keep our brain cell 'batteries' healthy may contribute to neurodegenerative disease, a new study has found.
To stay healthy, neurons must prevent protein aggregates and defective organelles such as mitochondria from accumulating inside them. We now know that an animal species has found a solution to its neuronal trash problemone ...
Scientists at the Stowers Institute for Medical Research have made a surprising finding about the aggregates of misfolded cellular proteins that have been linked to aging-related disorders such as Parkinson's disease. The ...
A new University of Colorado Boulder study shows for the first time the final stages of how mitochondria, the sausage-shaped, power-generating organelles found in nearly all living cells, regularly divide and propagate.
A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.
A research team, led by the University of Minnesota, has discovered a groundbreaking process to successfully rewarm large-scale animal heart valves and blood vessels preserved at very low temperatures. The discovery is a ...
Working with yeast and human cells, researchers at Johns Hopkins say they have discovered an unexpected route for cells to eliminate protein clumps that may sometimes be the molecular equivalent of throwing too much or the ...
By changing one small portion of a stimulus that influences part of one molecule's function, engineers and researchers at Washington University in St. Louis have opened the door for more insight into how the molecule is associated ...
A minimally invasive, fiber-optic technique that accurately measures the passive stretch and twitch contraction of living muscle tissue could someday be an alternative to the painful muscle biopsies used to diagnose and treat ...
An in-depth computational analysis of genetic variants implicated in both schizophrenia and rheumatoid arthritis by researchers at the University of Pittsburgh points to eight genes that may explain why susceptibility to ...
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In cleaning up misfolded proteins, cell powerhouses can break down - Medical Xpress
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Regenestem, LLC to Host 2nd Annual Inter American Regenerative and Cellular Medicine Conference in Cuba – Benzinga
Posted: March 1, 2017 at 11:41 pm
Regenestem, LLC (http://www.regenestem.com), a U.S.-based medical practice company that focuses on adult stem cell treatments for patients around the world as well as physician training on the latest technologies, is pleased to announce that for the second straight year it will be hosting a conference on regenerative medicine on the island of Cuba. The 2017 Inter American Regenerative and Cellular Medicine Conference (http://www.regenestemconference.com) will be held October 25-27, in Havana.
Miami, Florida (PRWEB) March 01, 2017
Coming off the success of its inaugural conference on regenerative medicine held in Cuba last October, the American company Regenestem (http://www.regenestem.com) has announced that it will host the second such event later this year. The company looks to continue the scientific collaboration that took place at last year's conference between medical professionals from the U.S., Cuba and around the world.
Regenestem, an international medical practice firm based in South Florida and focused on adult stem cell therapies and physician training, will present the Second Inter American Regenerative and Cellular Medicine Conference, October 25-27, at the Palacio de Convenciones, in Havana. The event is held in association with the Cuban Institute of Hematology.
"Our first regenerative medicine conference in Cuba was such a tremendous success that as it was winding down, we knew we had to continue the momentum and hold the second conference in 2017," said Ricardo De Cubas, conference organizer and CEO of Regenestem. "International research initiatives must start with face-to-face interactions between physicians from various countries. This event successfully brings together clinician-researchers worldwide who all have a focus in regenerative medicine, while at the same time enhancing the scientific collaboration specifically between the U.S. and Cuba."
In October 2016, more than 180 physicians and other medical professionals from 14 countries attended the first annual conference on regenerative medicine.
The three-day event explores adult stem cell therapies as a standard form of medical treatment. The program also addresses specific issues involving the process of replacing, reengineering or regenerating human cells, tissues and organs to restore or establish a body's normal function.
Along with the presentations and discussions, the conference also provides a series of hands-on training workshops that focus on the procedures for conducting many of the more popular adult stem cell therapies.
Those physicians already confirmed as conference speakers for the second year are:
For more information or to register for the conference, go to http://www.regenestemconference.com or call (305) 224-1858.
About Regenestem, LLC
Regenestem (http://www.regenestem.com) is an international medical practice company focused on providing comprehensive solutions involving adult stem cell treatments and research. The company has assembled a talented staff of medical specialists - professionals trained in the latest cutting-edge procedures and protocols in cellular medicine. Regenestem is certified for the medical tourism market, and staff physicians are board-certified or board-eligible, providing services in more than 10 specialties. Regenestem investigates, shares, utilizes and integrates the latest protocols in the adult stem cell arena to deliver the best medical solutions to its patients. The brand includes a membership association of regenerative medicine clinics, a training and education division, and an online store.
Recently, Regenestem received approval for an information page at Wikipedia, the free online encyclopedia, at https://en.wikipedia.org/wiki/Regenestem.
For the original version on PRWeb visit: http://www.prweb.com/releases/2017/03/prweb14113450.htm
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Regenestem, LLC to Host 2nd Annual Inter American Regenerative and Cellular Medicine Conference in Cuba - Benzinga
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Johns Hopkins Medicine, Maryland Stem Cell Research Fund and … – Business Wire (press release)
Posted: March 1, 2017 at 11:41 pm
SAN CARLOS, Calif. & BALTIMORE--(BUSINESS WIRE)--Johns Hopkins Medicine, the Maryland Stem Cell Research Fund (MSCRF) and BioCardia, Inc. (OTC:BCDA) today announced that the first patient has been treated in the pivotal Phase III CardiAMP clinical trial of a cell-based therapy for the treatment of ischemic heart failure that develops after a heart attack. The first patient was treated at Johns Hopkins Hospital by a team led by Peter Johnston, MD, a faculty member in the Department of Medicine and Division of Cardiology, and principal investigator of the trial at Johns Hopkins.
The investigational CardiAMP therapy is designed to deliver a high dose of a patients own bone marrow cells directly to the point of cardiac dysfunction, potentially stimulating the bodys natural healing mechanism after a heart attack.
The patient experience with CardiAMP therapy begins with a pre-procedural cell potency screening test. If a patient qualifies for therapy, they are scheduled for a bone marrow aspiration. A point of care cell processing platform is then utilized to concentrate the autologous bone marrow cells, which are subsequently delivered in a minimally-invasive procedure directly to the damaged regions in a patients heart.
This cell-based therapy offers great potential for heart failure patients, said Carl Pepine, MD, professor and former chief of cardiovascular medicine at the University of Florida, Gainesville and national co-principal investigator of the CardiAMP trial. We look forward to validating the impact of the therapy on patients quality of life and functional capacity in this important study.
In addition to Dr. Johnston, the CardiAMP research team at Johns Hopkins includes Gary Gerstenblith, MD, Jeffrey Brinker, MD, Ivan Borrello, MD, Judi Willhide, Katherine Laws, Audrey Dudek, Michele Fisher and John Texter, as well as the nurses and technicians of the Johns Hopkins Cardiovascular Interventional Laboratory.
Funding the clinical trial of this cell therapy, which could be the first cardiac cell therapy approved in the United States, is an important step towards treatments, said Dan Gincel, PhD., executive director of the MSCRF at TEDCO. Through our clinical program, we are advancing cures and improving healthcare in the State of Maryland.
The CardiAMP Heart Failure Trial is a phase III, multi-center, randomized, double-blinded, sham-controlled study of up to 260 patients at up to 40 centers nationwide, which includes an optional 10-patient roll-in cohort. The primary endpoint for the trial is a significant improvement in Six Minute Walk distance at 12 months post-treatment. Study subjects must be diagnosed with New York Heart Association (NYHA) Class II or III heart failure as a result of a previous heart attack. The national co-principal investigators are Dr. Pepine and Amish Raval, MD, of the University of Wisconsin.
For information about eligibility or enrollment in the trial, please visit http://www.clinicaltrials.gov or ask your cardiologist.
About BioCardia BioCardia, Inc., headquartered in San Carlos, CA, is developing regenerative biologic therapies to treat cardiovascular disease. CardiAMP and CardiALLO cell therapies are the companys biotherapeutic product candidates in clinical development. For more information, visit http://www.BioCardia.com.
About Johns Hopkins Medicine Johns Hopkins Medicine (JHM), headquartered in Baltimore, Maryland, is one of the leading health care systems in the United States. Johns Hopkins Medicine unites physicians and scientists of the Johns Hopkins University School of Medicine with the organizations, health professionals and facilities of The Johns Hopkins Hospital and Health System. For more information, visit http://www.hopkinsmedicine.org.
About Maryland Stem Cell Research Fund The Maryland Stem Cell Research Act of 2006was established by the Governor and the Maryland General Assembly during the 2006 legislative session and created the Maryland Stem Cell Research Fund. This fund is continued through an appropriation in the Governor's annual budget. The purpose of the Fund is to promote state-funded stem cell research and cures through grants and loans to public and private entities in the State. For more information, visit http://www.MSCRF.org.
Forward Looking Statements This press release contains forward-looking statements as that term is defined under the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, among other things, references to the enrollment of our Phase 3 trial, commercialization and efficacy of our products and therapies, the product development timelines of our competitors. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the inherent uncertainties associated with developing new products or technologies, unexpected expenditures, the ability to raise the additional funding needed to continue to pursue BioCardias business and product development plans, competition in the industry in which BioCardia operates and overall market conditions, and whether the combined funds will support BioCardias operations and enable BioCardia to advance its pivotal Phase 3 CardiAMP cell therapy program. These forward-looking statements are made as of the date of this press release, and BioCardia assumes no obligation to update the forward-looking statements.
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Johns Hopkins Medicine, Maryland Stem Cell Research Fund and ... - Business Wire (press release)
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US Stem Cell, Inc. (OTCMKTS: USRM) Shares Spike Higher By 62% – Street Register
Posted: February 28, 2017 at 12:44 pm
Shares of U.S. Stem Cell, Inc. (OTCMKTS: USRM) are surging early in the session today. The stock price is up over 62% on heavy volume. According to OTC Markets, the stock over the last 30 days trades about 15.6M shares per day, however; more than 43.5M shares have traded thus far. The stock price is currently trading around $0.19.
U.S. Stem Cell, Inc. (OTCMKTS: USRM) is a biotech company dedicated to expanding regenerative medicine. Weve seen shares rise significantly in 2017. However, that doesnt come without its fair share of volatility. Over the last 52-weeks the stock has traded in a range between $0.0016 to $0.40.
Nonetheless, we have not seen any news in U.S. Stem Cell, Inc. (OTCMKTS: USRM) to justify the move today. Based on Fridays close, the company had a market cap of around $697K. In addition, has a one star quantitative valuation from Morningstar.
About U.S. Stem Cell, Inc.
Founded in 1999, U.S Stem Cell, Inc. is committed to the development of effective cell technologies to treat a variety of diseases and injuries. By harnessing the bodys own healing potential, we may be able to reverse damaged tissue to normal function. U.S. Stem Cells discoveries include multiple cell therapies in various stages of development that repair damaged tissues throughout the body due to injury or disease so that patients may return to a normal lifestyle. U.S Stem Cell is focused on regenerative medicine. While most stem cell companies use one particular cell type to treat a variety of diseases, U.S Stem Cell utilizes various cell types to treat different diseases. It is our belief that the unique qualities within the various cell types make them more advantageous to treat a particular disease.
Most Recent Headline:
CSO Kristin Comella Publishes Paper on the Implantation of Stromal Vascular Fraction in Patients with Degenerative Disc Disease
SUNRISE, FL / ACCESSWIRE / January 23, 2017 / US STEM CELL, Inc. (USRM), a Florida corporation and leader in novel regenerative medicine solutions and physician-based stem cell therapies to human and animal patients, is pleased to announce our newest publication by our Chief Scientific Officer, Kristin Comella.
Effects of the intradiscal implantation of stromal vascular fraction plus platelet rich plasma in patients with degenerative disc disease was published in the January volume of the Journal of Translational Medicine. The study focused on the implantation of stromal vascular fraction (SVF) in patients suffering from degenerative disc disease. Patients underwent a local tumescent liposuction procedure to remove approximately 60 ml of fat tissue from the abdomen. The fat was separated to isolate the SVF and the cells were delivered directly into the damaged discs. Patients were monitored for a period of 6 months post-treatment, noting considerable decreases in pain and increases in flexion. A copy of the paper can be accessed on pubmed: https://www.ncbi.nlm.nih.gov/pubmed/28086781.
Ms. Comellas previous paper, Effects of the intramyocardial implantation of stromal vascular fraction in patients with chronic ischemic cardiomyopathy, was released in the Journal of Translational Medicines June 2016 edition. Using the same procedure, chronic ischemic cardiomyopathy patients were evaluated after SVF injection and able to walk more than 80 additional meters 3 to 6 months after treatment. A copy of the paper can be accessed on pubmed: https://www.ncbi.nlm.nih.gov/pubmed/27255774.
At U.S. Stem Cell, Inc., we are committed to new technological advancements and therapies that give a renewed sense of hope to patients with degenerative diseases. SVF is the latest therapy in a long line of successful treatments we have pioneered. Ms. Comella plans to continue her work with SVF, which has consistently repeated its strong safety profile and success in treating patients.
Weve seen a tremendous amount of volatility in the biotech and medical space thus far this year. It will be interesting to see how politics play a role in stocks like USRM. If youd like to keep up with biotech stocks like USRM, make sure to sign up to our newsletter below. We make it quick and easy for our subscribers to stay on top of the markets. And yes, its free Which makes our membership affordable for everyone.
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US Stem Cell, Inc. (OTCMKTS: USRM) Shares Spike Higher By 62% - Street Register
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Cellular Biomedicine Group Awarded $2.29 Million Grant from the California Institute for Regenerative Medicine (CIRM … – EconoTimes
Posted: February 28, 2017 at 12:44 pm
Cellular Biomedicine Group Awarded $2.29 Million Grant from the California Institute for Regenerative Medicine (CIRM) to Fund AlloJoin Allogeneic Stem Cell Therapy for Knee Osteoarthritis (KOA) in the U.S.
SHANGHAI, China and CUPERTINO, Calif., Feb. 27, 2017 -- Cellular Biomedicine Group Inc. (NASDAQ:CBMG)(CBMG or the Company), a clinical-stage biopharmaceutical firm engaged in the development of effective immunotherapies for cancer and stem cell therapies for degenerative diseases, announced today that the governing Board of the California Institute for Regenerative Medicine (CIRM), California's stem cell agency, has awarded the Company $2.29 million to support pre-clinical studies of AlloJoinTM, CBMGs Off-the-Shelf Allogeneic Human Adipose-derived Mesenchymal Stem Cells for the treatment of Knee Osteoarthritis in the United States.
While CBMG recently commenced two Phase I human clinical trials in China using CAR-T to treat relapsed/refractory CD19+ B-cell Acute Lymphoblastic Leukemia (ALL) and Refractory Diffuse Large B-cell Lymphoma (DLBCL) as well as an ongoing Phase I trial in China for AlloJoinTM in Knee Osteoarthritis (KOA), this latest announcement represents CBMGs initial entrance into the United States for its off-the-shelf allogeneic stem cell candidate AlloJoinTM.
The $2.29 million was granted under the CIRM 2.0 program, a comprehensive collaborative initiative designed to accelerate the development of stem cell-based treatments for people with unmet medical needs. After the award, CIRM will be a more active partner with its recipients to further increase the likelihood of clinical success and help advance a pre-clinical applicants research along a funding pipeline towards clinical trials. CBMGs KOA pre-clinical program is considered late-stage, and therefore it meets CIRM 2.0s intent to accelerate support for clinical stage development for identified candidates of stem cell treatments that demonstrate scientific excellence.
"We are deeply appreciative to CIRM for their support and validation of the therapeutic potential of our KOA therapy, said Tony (Bizuo) Liu, Chief Executive Officer of CBMG. We thank Dr. C. Thomas Vangsness, Jr., in the Department of Orthopaedic Surgery at the Keck School of Medicine of the University of Southern California and Dr. Qing Liu-Michael at the Broad Center for Regenerative Medicine and Stem Cell Research at USC, who helped significantly with the grant application process. The CIRM grant is the first step in bringing our allogeneic human adipose-derived mesenchymal stem cell treatment for knee osteoarthritis (AlloJoinTM) to the U.S. market.
Our AlloJoinTM program has previously undergone extensive manufacturing development and pre-clinical studies and is undergoing a Phase I clinical trial in China. In order to demonstrate comparability with cell banks previously produced in China for our U.S. IND filing, we are addressing the pre-clinical answers required for the FDA. With the funds provided by CIRM, we will replicate and validate the manufacturing process and control system at the cGMP facility located at Childrens Hospital Los Angeles to support the filing of an IND with the FDA. The outcome of this grant will enable us to have qualified final cell products ready to use in a Phase I clinical trial with Dr. Vangsness as the Principal Investigator and the Keck School of Medicine of USC as a trial site. Dr. Vangsness is familiar with both stem cell biology and KOA, and has led the only randomized double-blind human clinical study to investigate expanded allogeneic mesenchymal stem cells to date. Our endeavor in the U.S. market will further strengthen our commercialization pipeline.
CBMG recently announced promising interim 3-month safety data from its Phase I clinical trial in China for AlloJoinTM, its off-the-shelf allogeneic stem cell therapy for KOA. The trial is on schedule to be completed by the third quarter of 2017.
About CIRM
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and to act with a sense of urgency commensurate with that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies.
With $3 billion in funding and over 280 active stem cell programs in our portfolio, CIRM is the world's largest institution dedicated to helping people by bringing the future of medicine closer to reality.
For more information, please visit http://www.cirm.ca.gov.
About Knee Osteoarthritis
According to the Foundation for the National Institutes of Health, there are 27 million Americans with Osteoarthritis (OA), and symptomatic Knee Osteoarthritis (KOA) occurs in 13% of persons aged 60 and older. The International Journal of Rheumatic Diseases, 2011 reports that approximately 57 million people in China suffer from KOA. Currently no treatment exists that can effectively preserve knee joint cartilage or slow the progression of KOA. Current common drug-based methods of management, including anti-inflammatory medications (NSAIDs), only relieve symptoms and carry the risk of side effects. Patients with KOA suffer from compromised mobility, leading to sedentary lifestyles; doubling the risk of cardiovascular diseases, diabetes, and obesity; and increasing the risk of all causes of mortality, colon cancer, high blood pressure, osteoporosis, lipid disorders, depression and anxiety. According to the Epidemiology of Rheumatic Disease (Silman AJ, Hochberg MC. Oxford Univ. Press, 1993:257), 53% of patients with KOA will eventually become disabled.
About Cellular Biomedicine Group (CBMG)
Cellular Biomedicine Group, Inc. develops proprietary cell therapies for the treatment of cancer and degenerative diseases. Our immuno-oncology and stem cell projects are the result of research and development by CBMGs scientists and clinicians from both China and the United States. Our GMP facilities in China, consisting of twelve independent cell production lines, are designed and managed according to both China and U.S. GMP standards. To learn more about CBMG, please visit http://www.cellbiomedgroup.com.
Forward-looking Statements
This press release contains forward-looking statementsincluding descriptions of plans, strategies, trends, specific activities, investments and other non-historical factsas defined by the Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking information is inherently uncertain, and actual results could differ materially from those anticipated due to a number of factors, which include risks inherent in doing business, trends affecting the global economy (including the devaluation of the RMB by China in August 2015), and other risks detailed in CBMGs reports filed with the Securities and Exchange Commission, quarterly reports on form 10-Q, current reports on form 8-K and annual reports on form 10-K. Forward-looking statements may be identified by terms such as "may," "will," "expects," "plans," "intends," "estimates," "potential," "continue" or similar terms or their negations. Although CBMG believes the expectations reflected in the forward-looking statements are reasonable, they cannot guarantee that future results, levels of activity, performance or achievements will be obtained. CBMG does not have any obligation to update these forward-looking statements other than as required by law.
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Cellular Biomedicine Group Awarded $2.29 Million Grant from the California Institute for Regenerative Medicine (CIRM ... - EconoTimes
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‘Small Town Throwdown’ for Zach Standen on March 11 – The Redding Pilot
Posted: February 27, 2017 at 4:42 pm
Bobby Paultauf and his band will play the throwdown.
Those in the Redding, Easton area are probably aware of what happened to Joel Barlow High School student Zach Standen in the summer of 2016. Zach was in a devastating auto accident that left him partially paralyzed.
He needs hope and support from as many people as possible. He needs countless medical procedures in order to gain movement to his legs again through stem cell treatment due to a tragic car accident that made him paralyzed.
To help support Standens recovery, local musicians Bobby Paltauf, of the Bobby Paltauf Band, and Grayson Hugh, of Grayson Hugh & the Moon Hawks, will play a benefit show on Saturday, March 11 at the Fairfield Theater Company.
The family has started a GoFundMe account where people are able to donate to this expensive treatment. Lets all get together and help him walk again, Paultaufs mother, Tiffany, wrote in a press release.
Bobby Paltauf is a senior at Joel Barlow High School, where Standen goes.
Lets all get together and support local live music, especially where it benefits the hope of Zach being able to walk again, his mother wrote.
For the concert benefitting Standen, more information can be found on The Bobby Paltauf Band page on Facebook, and tickets are available at http://www.fairfieldtheatre.org.
Zach Standen coached soccer Oct. 8 at Seaside Park in Bridgeport with, back row, left to right, Mark Roman, Alastair Bruce and Peter Syku.
Standen may be helped by stem cell medical treatments that can be administered in Panama.
The Standen family is in discussions and communication with the Cell Medicine Institute in that country and are pursuing this line of treatment for the young man, who is paralyzed.
We have done much research into stem cell therapy for spinal cord injuries here in the U.S. and Canada, and there just are no clinical trials or clinics that have the experience and track record like this one, the family said in a recent letter. Cell Medicine has been doing this specific treatment since 2006 and has a 60% to 70% success rate of some kind of improvements in most patients within a year.
Each procedure costs $37,200, which includes all medical procedures and ancillary needs. This is not covered by medical insurance.
The family is hoping everyone reading this could go to his GoFundMe page and donate $25, to help Zachs recovery.
To donate, visit: https://www.gofundme.com/stem-cell-therapy-for-zach-standen.
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'Small Town Throwdown' for Zach Standen on March 11 - The Redding Pilot
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Penn State and the University of Michigan collaborate on cancer cell and infection research – The Daily Collegian Online
Posted: February 23, 2017 at 1:43 pm
As Penn State students worked to raise money for childhood cancer research, two researchers at Penn State worked on a study determining the best treatment methods for cancer and infections.
Dr. Andrew Read and Elsa Hansen, from Penn State, and Dr. Robert Woods, from the University of Michigan, all co-authored a paper on the research. The main goal of their research was to determine whether containment or aggressive medication was the best method of treatment for cancer or a serious infection. The research hinges on delaying drug resistance in cells, which would make the problem much harder to treat.
When containing cancer or an infection, just enough drug is used to keep the cancer or infection from spreading too much beyond its original area.
Elsa Hansen, a research associate at Penn State and co-author of the study, explained why just containment may sometimes be a surprisingly good treatment.
Health care providers and researchers are starting to question whether the traditional approach of aggressively treating a cancer or infection is really the best option for a patient, Hansen said via email.
Using a containment strategy may seem counterintuitive, but the idea is to leverage the fact that drug sensitive cells can competitively suppress the expansion of drug resistant cells, Hansen said, mentioning that drugs without immunity to medicine can block the spread of resistant cancer cells or infection. A milder form of treatment is used to control medicine-sensitive cells, which then control the resistant cells.
In these situations, aggressively treating the patient with a drug may cause the patients cancer or infection to develop an immunity to the treatment. In the case of infections, the containment method leads to the bodys immune system fighting off the resistant cells. In the case of cancer, it slows the spread as long as possible.
This research proves that containment of cells is a viable option those in the medical field should consider more often. However, the research also highlighted that there are situations where aggressive treatment is the preferred choice. The traditional approach to treating cancer and infections is still viable when containment will not improve the situation.
As resistance is involved in most cancer and infection associated deaths, Hansen believes researching new drugs and novel forms of treatment is crucial. However, she stated that its also important to figure out how to extend the life of the drugs we have, part of the teams research goal.
Collaborating with a clinical lecturer from the University of Michigan proved paramount to the success of the research. Dr. Robert Woods, co-author of the study and a specialist in internal medicine, infectious diseases at the University of Michigan, had unique skills to contribute to the research. As an expert in putting research into use in the medical field, particularly in the hospital on the universitys campus, Woods had the unique opportunity to see the research happening in his workplace.
Its sometimes hard to translate evolutionary concepts into clinical use, Woods said.
The combination of his practical experience with treating cancer and infection cells combined with the mathematical and theoretical work of Hansen and Read led to the success of this research, Woods added.
He focused on the physical aspects of the research instead of the theoretical aspects as someone who works in the hospital. Here, the research might have an effect by changing the way doctors treat cancer or infections.
Both Hansen and Woods said they found the collaborative research to be a positive experience, as the unique combination of skills and resources at the two universities made the research such a success.
With today's technology, I think long distance collaborations can work really well, Hansen said. Increasing ease of communication across universities can open up the opportunity for more collaborations such as this study.
This research has been highly regarded by Reads and Hansens colleagues at Penn State.
This is really important research because it points to a course of treatment that is not just the hell and fury approach, hit the cancer with all we have, Dr. David Hughes, an assistant professor of entomology and biology at Penn State said. These models will considerably aid in better decision making.
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Penn State and the University of Michigan collaborate on cancer cell and infection research - The Daily Collegian Online
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‘Small Town Throwdown’ will benefit Zach Standen – Easton Courier
Posted: February 22, 2017 at 10:43 pm
Bobby Paultauf and his band will play the throwdown.
Those in the Redding, Easton area are probably aware of what happened to Joel Barlow High School student Zach Standen in the summer of 2016. Zach was in a devastating auto accident that left him partially paralyzed.
He needs hope and support from as many people as possible. He needs countless medical procedures in order to gain movement to his legs again through stem cell treatment due to a tragic car accident that made him paralyzed.
To help support Standens recovery, local musicians Bobby Paltauf, of the Bobby Paltauf Band, and Grayson Hugh, of Grayson Hugh & the Moon Hawks, will play a benefit show on Saturday, March 11 at the Fairfield Theater Company.
The family has started a GoFundMe account where people are able to donate to this expensive treatment. Lets all get together and help him walk again, Paultaufs mother, Tiffany, wrote in a press release.
Bobby Paltauf is a senior at Joel Barlow High School, where Standen goes.
Lets all get together and support local live music, especially where it benefits the hope of Zach being able to walk again, his mother wrote.
For the concert benefitting Standen, more information can be found on The Bobby Paltauf Band page on Facebook, and tickets are available at http://www.fairfieldtheatre.org.
Standen may be helped by stem cell medical treatments that can be administered in Panama.
The Standen family is in discussions and communication with the Cell Medicine Institute in that country and are pursuing this line of treatment for the young man, who is paralyzed.
We have done much research into stem cell therapy for spinal cord injuries here in the U.S. and Canada, and there just are no clinical trials or clinics that have the experience and track record like this one, the family said in a recent letter. Cell Medicine has been doing this specific treatment since 2006 and has a 60% to 70% success rate of some kind of improvements in most patients within a year.
Each procedure costs $37,200, which includes all medical procedures and ancillary needs. This is not covered by medical insurance.
The family is hoping everyone reading this could go to his GoFundMe page and donate $25, to help Zachs recovery.
To donate, visit: https://www.gofundme.com/stem-cell-therapy-for-zach-standen.
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'Small Town Throwdown' will benefit Zach Standen - Easton Courier
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Researchers take broad look at stem cells – Science Daily
Posted: February 22, 2017 at 10:43 pm
Sanford Research scientists recently published a review article in an issue of Stem Cells Translational Medicine focused on the study of and utility of adult-derived stem cells.
Earlier this month, Sanford began enrolling participants in the Safety and Efficacy of Adult Adipose-Derived Stem Cell Injections into Partial Thickness Rotator Cuff Tears clinical trial. The trial uses stromal vascular fraction, a mixture of cells and nutrients isolated from a patient's own body that contain adipose-derived stem cells, as a potential therapy for partial-thickness rotator cuff tears. Sanford scientists and clinicians are exploring the application of this type of stem cells for other conditions.
The team put together the review after recognizing that the medical and general communities have limited knowledge about the various types of stem cells and how they could be used in medicine.
The article, "Fat and Furious: Harnessing the Full Potential of Adipose-Derived Stromal Vascular Fraction," is a review of the various types of stem cells found in humans and how they can be used in medical applications. The researchers emphasized the difference between the SVF isolated from adipose tissue and the pure adipose-derived stem cells that have been purified and maintained in a culture dish. Understanding those differences can help dictate appropriate therapies and regulations, particularly in countries where the SVF could be less regulated than other stem cells. It's also important to understand how SVF composition varies in healthy versus disease states.
"Continued research into the application of SVF and adipose derived stem cells has the potential to transform treatments and therapy options," said Daniel Kota, assistant research scientist for Sanford Research. "But it all starts with putting scientists on the same page -- tracking results following transfusions, using appropriate nomenclature and examining regulations."
Stem Cells Translational Medicine publishes papers in the evolving field of translational medicine, with a focus on helping speed emerging discoveries into clinical trials.
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Researchers take broad look at stem cells - Science Daily
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