Category Archives: FDA Stem Cell Trials

NEW YORK & PETACH TIKVAH, Israel--(BUSINESS WIRE)-- BrainStorm Cell Therapeutics (BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, announced that it has successfully completed a 12-week repeat dose toxicity study with its NurOwn cells in mice. The company believes that the positive data from the study will support its efforts to obtain approval for a future repeat dose clinical study in ALS patients. The study was conducted at Harlan Israels laboratories, according to GLP (Good Laboratory Practice) standards of the FDA. The study protocol was approved by the Israeli Ministry of Health. We believe that repeat dosing is the key to the long-term clinical efficacy of NurOwn and we are anxious to begin testing in ALS patients. Our proprietary cryopreservation protocol will enable us to do so without having to perform repeat bone marrow aspirations for each dose, commented Dr. Adrian Harel, BrainStorms CEO. In the study, the animals received up to three intramuscular injections of NurOwn, produced from cryopreserved, or frozen, cells at concentrations 50100 times the currently administered clinical trial dose. The treatment was well tolerated, and no adverse clinical effects were observed. Analyses included cytokine profile assays, clinical pathology, necropsy and macroscopic examination, … Continue reading →

Peregrine Pharmaceuticals Inc. (PPHM: Quote) soared 80% after the company announced that an internal review of a phase II study of Bavituximab in second-line non-small cell lung cancer supports advancing the compound into phase III development. The review was prompted by the discovery of vial coding discrepancies while preparing for an end of Phase II meeting with the FDA. The company said it is now preparing for discussions with the FDA and worldwide regulatory agencies. Thoratec Corp. (THOR: Quote) has received FDA approval to commence a study, dubbed REVIVE-IT, to compare the use of its HeartMate II LVAD with optimal medical management in patients with New York Heart Association Class III heart failure. Another complementary study known as ROADMAP is currently enrolling patients, and the company expects the full cohort of 200 patients to be enrolled in the ROADMAP study by the end of 2013. Orexigen Therapeutics Inc. (OREX: Quote) says it expects to resubmit the Contrave NDA in the second half of 2013 with potential approval in early 2014. The company's lead obesity drug candidate - Contrave, was issued a complete response letter in January of 2011 on cardiovascular safety concerns, and was asked to conduct a cardiovascular outcomes … Continue reading →

ROCKVILLE, Md., Jan. 4, 2013 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) announced that President and CEO Richard Garr will present at the fifth annual Biotech Showcase in San Francisco on Tuesday, January 8, 3:45 p.m. PT, in Room Powell. Garr will present a business overview, including a first look at the Phase I/II Stroke/NSI-566 cell therapy trial expected to commence in Beijing within several weeks. Garr will also provide an update on the company's ALS cell therapy trial and plans for the Phase II. The trial's investigators recently announced a substantial grant for the Neuralstem ALS Phase II trial from the National Institutes of Health (NIH). Garr will also update the trial progress of its first-in-class neurogenic NSI-189 small molecule drug which stimulates new neuron growth in the hippocampus, currently in a Phase Ib major depressive disorder (MDD) trial. (Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO) The audio webcast will be available in real-time at http://www.media-server.com/m/p/y46fee25and archived for one year. The webcast link will also be posted on the Investor Center home page on Neuralstem's website: http://www.neuralstem.com. The 2013 Biotech Showcase will be held at the Parc 55 Wyndham Hotel in San Francisco, January 7-9. For more information, see: http://www.ebdgroup.com/bts/index.php. About Neuralstem Neuralstem's patented technology … Continue reading →

REDWOOD CITY, Calif.--(BUSINESS WIRE)-- OncoMed Pharmaceuticals, a clinical-stage company developing novel therapeutics that target cancer stem cells (CSCs), or tumor-initiating cells, today announced that the U.S. Food and Drug Administration (FDA) has removed a partial clinical hold on its demcizumab (OMP-21M18) clinical program. Demcizumab is a humanized monoclonal antibody that targets Delta Like Ligand 4, or DLL4, in the Notch signaling pathway. Jakob Dupont, MD, OncoMeds Chief Medical Officer, stated, We are pleased that the FDA has responded favorably to the demcizumab clinical data and development strategy and removed the partial clinical hold to allow further development of demcizumab in the United States. OncoMed had submitted an extensive data package to the FDA with safety and efficacy results from four demcizumab clinical trials: a Phase 1a solid tumors study and three Phase 1b studies in non-small cell lung cancer (NSCLC), pancreatic cancer and colorectal cancer. The company plans to initiate new demcizumab clinical trials in 2013 in the United States. Specifically, the company will initiate a Phase 1b/2 study of demcizumab and paclitaxel in patients with epithelial ovarian cancer in collaboration with Drs. Robert Coleman and Anil Sood at the MD Anderson Cancer Center (MDACC) as part of the MDACC … Continue reading →

Susanne Posel Occupy Corporatism December 27, 2012 Dr. Leon R. Kass, the Addie Clark Harding Professor Emeritus in the Committee on Social Thought and the College at the University of Chicago and former chairman of the Presidents Council on Bioethics under former President George W. Bush, has written a report entitled The Meaning of Life In the Laboratory. Kass discusses the morality surrounding the US governments funding of research on human embryonic stem cells, cells derived from early embryos produced by in vitro fertilization in assisted-reproduction clinics. According to the report, the use of human embryos is justified by the advancements of science which demands that new policies be written to account for the development of regenerative medicine. Kass maintains that stem cells save lives, and therefore their use in research are necessary. Although he states that we must also strive to protect and preserve human dignity, Kass believes that with regard to abortions and stem cell research the continuation of this medical research is worth preserving. Earlier this year, the Obama Administration has approved the use of aborted fetuses in the use of research, even though this practice was previously banned. Obama utilized a loophole in the legislation that … Continue reading →

When cosmetic surgeon Allan Wu first heard the womans complaint, he wondered if she was imagining things or making it up. A resident of Los Angeles in her late sixties, she explained that she could not open her right eye without considerable pain and that every time she forced it open, she heard a strange clicka sharp sound, like a tiny castanet snapping shut. After examining her in person at The Morrow Institute in Rancho Mirage, Calif., Wu could see that something was wrong: Her eyelid drooped stubbornly, and the area around her eye was somewhat swollen. Six and a half hours of surgery later, he and his colleagues had dug out small chunks of bone from the womans eyelid and tissue surrounding her eye, which was scratched but largely intact. The clicks she heard were the bone fragments grinding against one another. About three months earlier the woman had opted for a relatively new kind of cosmetic procedure at a different clinic in Beverly Hillsa face-lift that made use of her own adult stem cells. First, cosmetic surgeons had removed some the womans abdominal fat with liposuction and isolated the adult stem cells withina family of cells that can … Continue reading →

A woman presented herself in Dr. Allan Wu's office with a symptom the cosmetic surgeon had never before encountered: she heard a distinct clicking sound, "like a tiny castanet snapping shut," every time she opened her right eye. The eye was painful and swollen. When Wu operated, he found bone shards growing inside her eyelid. Why did this happen? Because the patient had previously undergone a facelift that used her own adult stem cells. Stem cells as is their wont will grow into a variety of tissues. Under certain conditions, that includes bone. As Scientific American reports: First, cosmetic surgeons had removed some the woman's abdominal fat with liposuction and isolated the adult stem cells within a family of cells that can make many copies of themselves in an immature state and can develop into several different kinds of mature tissue. In this case the doctors extracted mesenchymal stem cells which can turn into bone, cartilage or fat, among other tissues and injected those cells back into her face, especially around her eyes. The procedure cost her more than $20,000, Wu recollects. Such face-lifts supposedly rejuvenate the skin because stem cells turn into brand-new tissue and release chemicals that help … Continue reading →

As usual, at the end of the year, I overview the most interesting and important events in cell therapy and Regen industry. The list of top 10 events reflects solely my opinion. 1. Debate on regulation of adult stem cells as drugs I think, it was the most important and interesting debate in 2012. In July, the lawsuit of Regenerative Sciences versus FDA got a new turn US Federal court ruled in favor of FDA regulation cultured adult stem cells as drugs. This ruling and ongoing debate has far-reaching implications for cell therapy industry as a whole. Based on court decision, FDA has a power to regulate stem cell clinics, which attempt to offer autologous adult stem cells (AASC) as a medical procedure on the US market. Yet another notable provocative event was an inspection of CellTex facility with following warning letter. FDA consistently and persistently says that almost all of AASC should be regulated as drugs or any other biologics, means via clinical trials (IND) and BLA (Biologics License Application). Federal court ruling and CellTex inspection triggers a lot of good and productive debates among cell therapy industry professionals, patient advocates, academics and physicians. But, it also polarized community … Continue reading →

REDWOOD CITY, Calif.--(BUSINESS WIRE)-- OncoMed Pharmaceuticals, Inc., a clinical-stage company developing novel therapeutics that target cancer stem cells (CSCs), or tumor-initiating cells, today announced that patient dosing has begun in a Phase 1 clinical trial of OMP-52M51 in patients with hematologic cancers. OMP-52M51 is OncoMeds fifth product candidate to enter clinical development. OMP-52M51 is a proprietary monoclonal antibody that targets the Notch1 receptor. Enrollment of the first patient in the Anti-Notch1 Phase 1 trial has triggered a $4 million milestone payment from the companys strategic collaborator GlaxoSmithKline (GSK). The first Phase 1 clinical trial of OMP-52M51 is an open-label dose escalation and expansion study in patients with hematologic cancers. These patients are assessed for safety, pharmacokinetics, pharmacodynamics, and initial evidence of efficacy, and the clinical trial will also assess a predictive biomarker-based patient selection approach. OncoMed also has filed an additional IND application with the FDA to evaluate this monoclonal antibody in patients with solid tumors. The trial is being conducted at several sites in the United States including Sarah Cannon Research Institute (SCRI) in Nashville, Tennessee. According to Dr. Ian Flinn of SCRI, who treated the first patient with OMP-52M51, It is exciting to bring a novel antibody such … Continue reading →

SILVER SPRING, Md., Dec. 18, 2012 (GLOBE NEWSWIRE) -- Nuvilex, Inc. (NVLX), an international biotechnology provider of cell and gene therapy solutions, announced today that major aspects of its core, "platform," cell encapsulation technology were discussed recently by Nuvilex's COO, Dr. Gerald W. Crabtree, during an interview with SmallSector.com. Advantages of Nuvilex's cell encapsulation technology, as well as its possible applications in the development of treatments for serious, debilitating, and even fatal diseases were emphasized. In discussing Nuvilex's unique, proprietary cell encapsulation technology, Dr. Crabtree noted our capsules (composed largely of cellulose) are "inert" in the body, are "tiny" and can contain large numbers of cells. In order for the cells to remain healthy, the capsules have "pores" through which nutrients for the encapsulated cells enter and waste products and "beneficial" factors produced by the cells are able to exit. Advantages of our cell encapsulation technology mentioned include: (a) it is a "platform" upon which treatments for many diseases may be built - virtually any type of cells (e.g. drug-activating cells, drug-producing cells, stem cells, probiotic bacteria) can be encapsulated with only very minor modifications of our process; (b) it is "mature" and has been developed and validated using Good … Continue reading →