Category Archives: FDA Stem Cell Trials

THOUSAND OAKS, Calif., Dec. 8, 2014 /PRNewswire/ --Amgen (NASDAQ: AMGN) today announcedthat new data from a pivotal Phase 2 study evaluating BLINCYTO (blinatumomab) for the treatment of adult patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (ALL) was presented at the 56thAmerican Society of Hematology (ASH) Annual Meeting and Exposition. In one analysis from the '211 study, 40 percent of patients treated with BLINCYTO who achieved a complete remission (CR) or complete remission with partial hematologic recovery (CRh) were enabled to proceed to allogeneic hematopoietic stem cell transplant (HSCT). Additionally, a secondary analysis from the study found that 82 percent of patients who had a CR or CRh also had a minimal residual disease (MRD) response, a measure used to predict disease recurrence in patients with ALL. "The data from the '211 study expand the evidence of Amgen's BiTE immunotherapy as an advance in the management of this difficult-to-treat cancer, and importantly, served as the basis for the recent U.S. Food and Drug Administration (FDA) approval of BLINCYTO," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "In this study, BLINCYTO helped patients bridge to a stem cell transplant after achieving a remission, a key … Continue reading →

International Stem Cell Corp., a Carlsbad-based biotech company developing stem cell therapies and biomedical products, announced that the U. S. Food and Drug Administration has cleared the companys human parthenogenetic stem cell line for investigational clinical use. Human embryonic stem cells typically come from fertilized eggs. In 2007, however, scientists at International Stem Cell Corp. (ISCO) reported the first successful creation of human stem cell lines from unfertilized eggs, according to Scientific American. They used a process called parthenogenesis, in which researchers use chemicals to induce the egg to begin developing as if it had been fertilized. The egg called a parthenote behaves just like an embryo in the early stages of division. Because it contains no genetic material from a father, however, it cannot develop into a viable fetus. Just like embryonic stem cells, parthenogenetic stem cells can be coaxed to grow into different kinds of human cells or tissue, ready to be transplanted into diseased areas of the body. Many stem cell lines can never be used to develop commercial therapeutic products because they dont meet the FDAs ethical and quality standards, said Ruslan Semechkin, ISCOs chief scientific officer. With this clearance from the FDA, based on the … Continue reading →

Once All of the FDA Comments Are Addressed, the Company Aims to Initiate Gene Silencing Immunotherapy for Metastatic Breast Cancer Patients SAN DIEGO, CA--(Marketwired - Nov 4, 2014) - Regen BioPharma, Inc. (OTCBB: RGBP) announced today issuance of IND (Investigational New Drug Application) number 16200 from the FDA for a proposed Phase I/II clinical trial assessing safety with signals of efficacy of the dCellVax gene silenced dendritic cell immunotherapy for treating breast cancer. The proposed trial will recruit 10 patients with metastatic breast cancer and will involve 4 monthly injections of the dCellVax gene-silenced dendritic cell therapy. The trial will last one year, with tumor assessment before therapy and at 6 and 12 months. Regen's dCellVax therapy is based on US Patent # 8,389,708, acquired from Professor Wei-Ping Min at the University of Western Ontario, Canada, as well as a collaboration between Dr. Min and the Company's Chief Science Officer Dr. Thomas Ichim. Preclinical studies have demonstrated efficacy in mouse models of breast cancer and melanoma. "We see the granting of an IND number as an important first step in the clinical development of this new personalized therapy that will hopefully add a new treatment option to patients without the … Continue reading →

PBR Staff Writer Published 03 November 2014 Dutch biopharmaceutical firm Kiadis Pharma has received orphan drug designation (ODD) from the European Medicines Agency (EMA) for its T-cell immunotherapy based medicinal product ATIR to treat acute myeloid leukemia (AML). Previously, ATIR was also granted ODD by both the EMA and the US Food and Drug Administration (FDA) for the prevention of acute graft versus host disease (GvHD) following an allogeneic bone marrow transplantation. ATIR allows stem cell transplantations using partially mismatched family members as donors for patients suffering from blood cancer who do not have a standard of care matching stem cell donor available. For many patients hematopoietic stem cell transplantation (HSCT) is the only potentially curative option but a matching donor is available for only half of the patients in need. Kiadis Pharma chief executive officer Manfred Ruediger said: "This second Orphan Drug Designation for our lead product ATIR in the European Union represents another very important milestone in the development of our product. "This, coupled with the previously granted ODDS for ATIR by the FDA and EMA, further highlights our product's importance as a novel approach which may help provide a potentially life-saving transplant from a family member to … Continue reading →

According to a Geron Corp press release, the FDA is allowing Geron Corp to begin human clinical tests of embryonic stem cells (ESC) for the treatment of acute spinal injuries. Geron will use ESC to try to regrow nerve tissue in patients. Although the FDA had received the request to begin clinical trials since May 2008, they remained silent until now - possibly still reading the 21,000 page document. The FDA action coming just a few days after Obama became President is heartening to patients who have chronic diseases such as Parkinson's, juvenile diabetes, MS. ALS and many other illnesses. It holds great promise for new treatments and transplants. Geron Corp (GERN) is a San Francisco, California based biotech company developing biopharmaceuticals for treatments of cancer and degenerative diseases. Shares are trading up as of the announcement. addenda 1/24/09 for additional information refer to: http://www.medicalnewstoday.com/articles/136413.php Commentary about action and use of adult vs embryonic stem cells: http://www.medicalnewstoday.com/articles/136553.php See the original post here: Parkinson's Focus Today: FDA Okays Embryonic Stem Cell ... … Continue reading →

(JNS.org) Stem cell treatment developed by Israeli company BrainStorm Cell Therapeutics has been designated by the U.S. Food and Drug Administration (FDA) as a fast-track treatment of amyotrophic lateral sclerosis (ALS). The treatment, called NurOwn, is currently undergoing mid-stage clinical trials in Jerusalem and in the U.S. on patients with ALS, which is also known as Lou Gehrigs Disease. The FDAs designation will speed up patients access to the treatment. The ALS Association reports that 5,600 people in the U.S. are diagnosed each year with the disease. The receipt of fast-track designation from the FDA is an acknowledgement of the unmet medical need in ALS, BrainStorm Chief Executive Tony Fiorino said Tuesday, Oct. 7, according to Reuters.What is so valuable about fast track designation to a small company like BrainStorm is the opportunity to have increased meetings with and more frequent written communication from the FDA, he said, adding that few other cellular therapies have received FDA approval. Here is the original post: FDA fast-tracks Israeli ALS treatment … Continue reading →

PUBLIC RELEASE DATE: 6-Oct-2014 Contact: Jenny Song jenny@solvingkidscancer.org 212-588-6625 Solving Kids' Cancer @SolveKidsCancer The U.S. Food and Drug Administration (FDA) granted orphan drug designation for a neuroblastoma vaccine from MabVax Therapeutics, providing development incentive with market exclusivity of the novel treatment for children with this deadly childhood cancer. The vaccine harnesses the power of a child's own immune system to recognize and destroy cancer cells that express the two antigens most commonly found on the surface of neuroblastoma cellsGD2 and GD3, potentially promoting long-term remissions. A phase I trial with the vaccine was recently completed at Memorial Sloan-Kettering Cancer Center with 15 patients and demonstrated very promising results; the results were published in the March 2014 issue of Clinical Cancer Research. "The coveted FDA designation for this agent to treat neuroblastoma represents a win for the childhood cancer community. This will continue to encourage drug development for pediatric cancer, an arena which is often ignored by companies due to the small patient populations," said Scott Kennedy, Executive Director of Solving Kids' Cancer. This past May, INBRACED--an international neuroblastoma research collaboration founded by the Neuroblastoma Children's Cancer Alliance (NCCA UK), Joining Against Cancer in Kids (J-A-C-K), and Solving Kids' Cancer--announced its … Continue reading →

(Short Hills, NJ) January 23, 2009 -- Peter T. Wilderotter, president and CEO of the Christopher & Dana Reeve Foundation, issued the following statement today in response to FDA clearance of Gerons human clinical trial of embryonic stem cell-based therapy: This is an exciting first step for Geron and for spinal cord injured patients whose lives may improve due to advances in medical research. There has been so much speculative and unsubstantiated information about the use of stem cells in spinal cord injury; it is important to study these cells in a rigorously designed clinical trial that is monitored by the FDA. Wilderotter continued, Christopher and Dana Reeve brought national attention to this issue and they gave voice to all those who are suffering the devastating physical, emotional and financial impact of challenging neurological conditions. Their belief in the promise of stem cell research became a part of their lasting legacy. Shortly before his death, Christopher Reeve wrote, It is our responsibility to do everything possible to protect the quality of life of the present and future generationsNo obstacle should stand in the way of responsible investigation of their [stem cells] possibilities. Wilderotter concluded, This is the dawn of basic … Continue reading →

Company plans for the future of stem cell use by Samantha Schmieder Staff Writer Next Healthcare Inc. of Germantown recently launched a partnership with Arizona Cardinals wide reciever Larry Fitzgerald to promote its newest venture, CelBank Pro to other professional athletes. Next Healthcares CelBank is the collection of cell samples and storage of their blood, skin or stem cells to be used in the future. Stem cells are unspecialized cells that are able to renew themselves through cell division and can be scientifically manipulated to become another type of cell with a more specialized function. They offer hope to provide new ways to fight disease or injuries, according to the National Institutes of Health. Essentially we are in the business of banking cells for people, Vin Singh, the founder and CEO of Next Healthcare, said. While CelBank is geared toward anyone interested in using their own cells later in their life, CelBank Pro is geared toward sports players who are very likely to get injured or just worn down during their career. Skin cells and stem cells are stored at a healthy time at someones life for later use in regenerative medicine, Singh said. In 2006 and 2007, Singh, who … Continue reading →

ROCKVILLE, Md.--(BUSINESS WIRE)--MEDIPOST America Inc. (CEO: Dr. Antonio Lee) today announced the US FDA approval of Phase 1/2 clinical trial for PNEUMOSTEM. The Phase 1/2 trial will assess the safety and efficacy of PNEUMOSTEM on prematurely born infants who will be at high-risk of developing Bronchopulmonary Dysplasia (BPD). PNEUMOSTEM is made from allogeneic human Umbilical Cord Blood-derived Mesenchymal Stem Cells (hUCB-MSCs), and is expected to have effect on regenerating the lung tissue and improving inflammatory responses in premature infants. BPD is the leading cause of mortality and severe complications in premature infants and currently no therapy or approved drug exists. Hence, the PNEUMOSTEM trial is expected to draw global attention in the field of neonatal medicine. PNEUMOSTEM received Orphan Drug designation in Korea by the Ministry of Food and Drug Safety (MFDS) and the Phase 2 study in Korea is 80% complete. The US FDA also granted Orphan Drug designation for PNEUMOSTEM, demonstrating its medical value and commercial potential. MEDIPOST America will move quickly to commence the PNEUMOSTEM trial in the U.S. while continuing the licensing and technology transfer negotiations with multinational pharmaceutical companies. The clinical trial approval of PNEUMOSTEM by the US FDA which implements strict regulations on medicinal … Continue reading →