Category Archives: FDA Stem Cell Trials

NEW YORK, April 29, 2013 /PRNewswire/ --Doctors at the University of Minnesota performed an umbilical cord blood transplant for a young boy with both AIDS and leukemia that may cure him of both diseases. This announcement comes among an increasing number of stories about cutting-edge treatments that use stem cells from umbilical cord blood to treat previously incurable diseases and conditions. (Photo: http://photos.prnewswire.com/prnh/20130429/NY03049-INFO ) The increase in treatments using cord blood stem cells is correlated with the pace at which cord blood stem cell research is advancing. According to BioInformant Worldwide, a research company focused on the stem cell industry, the past five years yielded a 144% increase in ongoing clinical trials for cord blood research. Citing data from clinicaltrials.gov the report noted that there were 78 ongoing clinical trials for cord blood stem cells in 2008 as compared to 191 in 2012. BioInformant also reported that the number of diseases treated with cord blood stem cells has risen by more than 145%, from 33 to 81 in the same time period. The advancement in research and availability of treatments continues to stimulate interest in cord blood banking among parents-to-be. Martin Smithmyer, CEO of Americord Registry noted, "Double digit growth … Continue reading →

NEW YORK, NY--(Marketwired - Apr 29, 2013) - While many biotech and pharmaceutical companies are designing one drug after another to address specific health problems, Nuvilex, Inc. (OTCQB: NVLX) is using its own living cell encapsulation as a "one size fits all" type solution. The company is off to a great start at getting its "Cell-in-a-Box" technology on the map with its work in late stage, inoperable pancreatic cancer. Nuvilex is headquartered in Silver Spring, Maryland, and while it is a small international biotech, the company has been delivering big results, and now it's preparing for an even bigger stage -- Phase III clinical trials. When it comes to pancreatic cancer, you can bet the NYSE's Eli Lilly and NASDAQ's Celgene Corp. are all too familiar with Nuvilex and its technology. Currently, Eli Lilly's drug Gemzar is the only drug to date approved by the FDA as a single agent for the treatment of advanced, inoperable pancreatic cancer and the drug is considered the "gold standard" by the market. Eli Lilly introduced gemcitabine (Gemzar) back in 1996, and since that time, it has netted the drug's maker billions of dollars in sales. Nipping at Eli Lilly's heels are both Celgene … Continue reading →

Stems cells taken from just a few grams of body fat are a promising weapon against the crippling effects of osteoarthritis. For the past two decades, knee, hip or other joint replacements have been the standard treatment for the deterioration of joint cartilage and the underlying bone. But artificial joints only last about 15 years and are difficult to repair once they fail. Stem cell injections may offer a new type of therapy by either stopping the degenerative process or by regenerating the damaged cartilage, said pioneering researcher Dr. Farshid Guilak, a professor of orthopedic surgery and director of orthopedic research at Duke University. Guilak, one of the first researchers to grow cartilage from fat, explains why stem cells are a bright light in osteoarthritis research and why widespread clinical use is still years away. Below is an edited transcript of the interview. Q: How are stem cell injections purported to help? A: Several studies in animals show that stem cell injections may help by reducing the inflammation in the joint. Stem cells appear to have a natural capacity to produce anti-inflammatory molecules, and once injected in the joint, can slow down the degenerative process in osteoarthritis. (Since this interview, … Continue reading →

CMX001 demonstrated potential clinical utility in prevention of CMV infection in Phase 2 SUPPRESS trial anticipated to begin dosing mid-2013 with topline data expected in 2015 DURHAM, N.C., April 24, 2013 (GLOBE NEWSWIRE) -- Chimerix, Inc. (CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, announced today the final study design for the Phase 3 SUPPRESS trial of CMX001 in the prevention of cytomegalovirus (CMV) infection following an allogeneic hematopoietic stem cell transplant (HSCT). CMX001 is an investigational oral nucleotide analog lipid-conjugate that has shown broad-spectrum antiviral activity against double-stranded DNA (dsDNA) viruses including all of the herpesviruses, adenoviruses and polyomaviruses. Since the End of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) in May 2012, Chimerix has been working closely with the FDA on the study design of the Phase 3 SUPPRESS trial, and has now finalized the population, specifics and timing of the primary and multiple secondary endpoints, as well as the dose and duration of CMX001 therapy. Initial study designs considered that two active doses of CMX001, 100 mg twice weekly and 75 mg twice weekly, be carried forward into the Phase 3 trial in order to mitigate … Continue reading →

(WZZM) - Work in Michigan on a possible cure for a debilitiating disease has been approved to enter the next phase. The research involves stem cells injected directly into the spinal cords of patients with ALS or Lou Gehrig's disease. In 2011, WZZMvisited the stem cell lab at the University of Michigan where they were doing the research. At that time, neurologist Eva Feldman felt the research would lead to a cure in less than 10 years. Now after two years of phase one clinical trials, the FDA gave approval to move forward into phase two. "In this trial, we will be injecting stem cells into the upper part of the spinal cord of patients with ALS," said Dr. Feldman. "Our goal is to continue to show that this approach is safe and we will begin to look at whether this approach offers some benefit to our patients." The University of Michigan is now looking for approval to hold phase two trials here in Michigan. Fifteenpatients were part of the first phase of testing at Emory University in Georgia. Each received injections of up to 400,000 stem cells. You can find out more information on the trials by clicking here. … Continue reading →

BOGOTA, Colombia, April 12, 2013 /PRNewswire/ -- Startup biotechnology company Neuralgene (http://neuralgene.com) has announced that it will begin animal studies in May to evaluate the efficacy of PRCN-829, its new gene therapy agent for the treatment of amyotrophic lateral sclerosis (ALS). PRCN-829 is the first gene therapy for sporadic ALS. (Photo: http://photos.prnewswire.com/prnh/20130412/PH93428-a ) (Photo: http://photos.prnewswire.com/prnh/20130412/PH93428-b ) Neuralgene's neurotropic AAV-based gene therapy platform for the treatment of neurodegenerative diseases is based on the stem cell work performed by Jason Williams, M.D., founder and CEO of Neuralgene. "This technology addresses several key aspects of the underlying pathology of ALS," said Leonardo Gonzalez, M.D., clinical researcher for Neuralgene. "In his stem cell work, Dr. Williams had identified that production of Factor H by fat-derived mesenchymal stem cells may be a key mode of action." The gene therapy is based on Dr. Williams' discovery that certain proteins produced by stem cells inhibit the attack of ALS. During the development of the gene therapy, he added new targets: neural growth factors and a protein implicated in ALS named TDP-43. "When Dr. Williams demonstrated the concept behind stem cells and how to address the treatment of ALS using gene therapy, we immediately knew that this was … Continue reading →

WHITEHOUSE STATION N.J.--(BUSINESS WIRE)-- Merck (MRK), known as MSD outside the United States and Canada, today announced that its New Drug Application for an investigational, tablet formulation of the company's antifungal agent, NOXAFIL (posaconazole), has been accepted for review by the U.S. Food and Drug Administration (FDA). Merck currently markets NOXAFIL Oral Suspension for prophylaxis of invasive Aspergillus and Candida infections in patients 13 years of age and older who are at high risk of developing these infections due to being severely immunocompromised, such as patients who have received hematopoietic stem cell transplants and have graft-versus-host disease, or patients with cancers of the blood who are experiencing prolonged low white blood cell counts (neutropenia) as a result of chemotherapy. Invasive fungal infections are a significant cause of illness and death among severely immunocompromised patients, said Robin Isaacs, M.D., vice president, infectious disease clinical research, Merck Research Laboratories. This filing for a tablet formulation of NOXAFIL is an example of Mercks ongoing commitment to developing new therapy options for patients in the hospital setting. Merck is seeking FDA approval of NOXAFIL tablets for once-daily administration (following a twice-a-day loading dose on the first day of therapy). The company has filed a … Continue reading →

GREENWICH, Conn., April 5, 2013 /PRNewswire/ --Spencer Trask, a venture capital firm focused on developing big ideas into world-changing companies, congratulates InVivo Therapeutics (NVIV), for its recent approvals from the U.S. Food and Drug Administration (FDA). On the heels of FDA approval for Humanitarian Use Device (HUD) designation (an important benchmark and speed-to-market catalyst), InVivo has received yet another important FDA approval Investigational Device Exemption (IDE), allowing human trials to begin. With IDE approval, InVivo will now be able to run first-in-man clinical trials to confirm safety and performance of its biopolymer scaffold. There are currently no treatment options approved by the FDA, or in clinical trials, to intervene directly in the spinal cord following a spinal cord injury. "We believe InVivo's treatment for spinal cord injuries could have a profound impact. If this is approved, it will change lives," said Kevin Kimberlin, Chairman of Spencer Trask & Co. "This is what we are all about helping entrepreneurs brave enough to work on tough problems, like ending paralysis due to spinal cord injuries." Spencer Trask Ventures raised $13 million in early-stage funding for InVivo in December of 2010 through its network of individual investors at a share price of $1.00. … Continue reading →

SAN DIEGO, CA--(Marketwired - Apr 4, 2013) - Regen BioPharma, Inc. (Regen), a wholly-owned subsidiary of Bio-Matrix Scientific Group, Inc. (OTCQB: BMSN), provided an update on the Company's progress with HemaXellerate and other new developments. Currently, Regen is focused on two core areas: a)The HemaXellerate Program, which is based on treating disorders of blood production; and b) Our cancer immunotherapy Program which leverages specific cells called "dendritic cells" to specifically kill cancer but not healthy tissue. In June 2012 the Company acquired an option to license US patent # 6,821,513 covering use of endothelial cells to stimulate blood cell production.Subsequently Regen has filed patent applications # 61/648898 and # 61/670791 covering stimulation of blood cell production using placental and fat derived cells, respectively. Utilizing these technologies, as well as, results obtained from studies performed at the Torrey Pines Institute for Molecular Medicine by Sophia Khaldoyanidi, M.D., Ph.D., Regen has developed the HemaXellerate product, which is a patient-specific composition of cells that has previously been demonstrated to repair damaged bone marrow and stimulate production of blood cells. Together with opinion leaders from University of Utah, Indiana University, and Cook General Biotechnology, Regen has published a peer-reviewed paper describing preclinical data supporting … Continue reading →

SUNRISE, FL--(Marketwired - Apr 2, 2013) - Bioheart, Inc. (OTCQB: BHRT) announced today that it has completed the FDA tissue bank registration process.Bioheart is offering stem cell storage for patients enrolled in the studies at the centers of excellence outside the US.Patients can store additional stem cell doses in liquid nitrogen for future use in approved clinical trials.These trials focus on the use of LipiCell (adipose derived stem cells) in a variety of different degenerative diseases. Kristin Comella, Chief Science Officer, commented, "We are excited to offer these additional therapies to the patients who participate in our trials in Jamaica and Mexico.We now have the ability to store their stem cells for future use in repeat dosing studies." About Bioheart, Inc.Bioheart is committed to maintaining its leading position within the cardiovascular sector of the cell technology industry delivering cell therapies and biologics that help address congestive heart failure, lower limb ischemia, chronic heart ischemia, acute myocardial infarctions and other issues.Bioheart's goals are to cause damaged tissue to be regenerated, when possible, and to improve a patient's quality of life and reduce health care costs and hospitalizations. Specific to biotechnology, Bioheart is focused on the discovery, development and, subject to regulatory … Continue reading →