Category Archives: FDA Stem Cell Trials

By monitoring the path of stem cells in the body, scientists can better explore experimental therapies, and doctors can better tune treatments in patients. Giving patients stem cells packaged with silica nanoparticles could help doctors determine the effectiveness of the treatments by revealing where the cells go after theyve left the injection needle. Researchers from Stanford University School of Medicine report in a paper published on Wednesday in the journal Science Translational Medicine that silica nanoparticles taken up by stem cells make the cells visible on ultrasound imaging. While other imaging techniques such as MRI can show where stem cells are located in the body, that method is not as fast, affordable, or widely available as an ultrasound scanner, and more importantly, it does not offer a real-time view of injection, say experts. Stem cells have significant medical promise because they can be turned into other types of living cell. As well as helping doctors adjust therapeutic dosages in patients, the new technique could help scientists perfect stem cell treatments, says senior author Sanjiv Gambhir. For the most part, researchers shoot blindlythey dont quite know where the cells are going when they are injected, they dont know if they home … Continue reading →

SAN BRUNO,Calif., March 19, 2013 /PRNewswire/ --Cord Blood Registry (CBR), the world's largest newborn stem cell bank, is fueling innovation in newborn stem cell research. As CBR prepares to release its 250th cord blood unit for medical use this month, the newborn stem cell bank announces that 71% of all its units released for use have been for emerging applications in regenerative medicine, such as brain injury, autism and type 1 diabetes. The other 29% have been for traditional transplant use, such as leukemia and sickle cell disease. This rapid increase in the use of family banked units for regenerative medicine applications is a complete reversal from the figures just six years ago, where 25% of the units released were for regenerative medicine applications and 75% for traditional transplant use. More than 50% of all cord blood units released for use in emerging regenerative therapies by family banks have been processed and stored at CBR. CBR is the only family newborn stem cell bank to have established FDA-regulated trials and is connecting client families to more potential treatments. As the industry leader in this initiative, Cord Blood Registry continues to focus on advancing the clinical applications of newborn stem cells. … Continue reading →

BOTHELL, Wash.--(BUSINESS WIRE)-- Seattle Genetics, Inc. (SGEN) announced today that it has submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) supporting the use of ADCETRIS (brentuximab vedotin) for retreatment and extended duration beyond 16 cycles of therapy in relapsed Hodgkin lymphoma (HL) and systemic anaplastic large cell lymphoma (sALCL). ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of HL and sALCL, that was granted accelerated approval by the FDA in August 2011 for relapsed HL and relapsed sALCL. The sBLA submission includes data demonstrating ADCETRIS activity in managing HL and sALCL when used in the retreatment setting, as well as beyond the 16 cycles described in our current label, while retaining a manageable safety profile, said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. Our goal is to broaden the ADCETRIS U.S. labeling claims to provide both patients and physicians the opportunity to incorporate ADCETRIS into additional HL and sALCL treatment settings. The sBLA submission includes data that support these uses and we look forward to the regulatory outcome. The sBLA is based on results from a phase II clinical trial with two treatment arms. … Continue reading →

SAN DIEGO, CA--(Marketwire - Mar 18, 2013) - Regen BioPharma, Inc., a wholly-owned subsidiary of Bio-Matrix Scientific Group Inc. ( PINKSHEETS : BMSN ), announced today issuance of IND number 15376 for its proposed Phase I/II clinical trial evaluating use of its stem cell product, HemaXellerate, for treatment of aplastic anemia. As part of the clinical trial approval process, the FDA will issue comments on the proposed trial which must be addressed to the FDA's satisfaction before patient dosing is initiated.The clinical trial will treat ten patients with aplastic anemia that have not responded to conventional therapy and lack a bone marrow donor. "We are pleased that the company has been issued an IND number and has taken the first step towards regulatory approval in the USA," said David Koos, Chairman & CEO of Bio-Matrix. "We have compiled an internationally-renowned team, consisting of corporate and academic partners, which cover the manufacturing, regulatory, and medical aspects of the project.Members of the team have previously published with Regen the scientific rational for the trial in a peer-reviewed publication in January of last year which may be found at http://www.translational-medicine.com/content/pdf/1479-5876-10-231.pdf." HemaXellerate is a patient-specific composition of cells that have previously been demonstrated to … Continue reading →

SALT LAKE CITY, UT--(Marketwire - Mar 14, 2013) - Q Therapeutics, Inc. today announced the appointment of Diane Jorkasky, M.D., FACP, as an independent member of its Board of Directors. Dr. Jorkasky will assume the board seat formerly held by Joydeep Goswami, Ph.D., President of Life Technologies, Japan. With the addition of Dr. Jorkasky to Q Therapeutics' Board, the number of directors remains at six and includes Peter Barton Hutt, of Covington & Burling and former Chief Counsel to the FDA; Peter Grebow, Ph.D., former Executive Vice President of Cephalon Ventures, Executive Vice President Technical Operations and Senior Vice President of Drug Development at Cephalon; Linda Powers, Chief Executive Officer of Northwest Biotherapeutics and Managing Director of Toucan Capital; Dinesh Patel, Ph.D., Managing Director of Signal Peak Ventures and former Chief Executive Officer of TheraTech; and Deborah Eppstein Ph.D., President and CEO of Q Therapeutics. Commenting on her appointment, Dr. Jorkasky said, "I am most impressed by Q Therapeutics' cell therapy approach to treatment of neurodegenerative diseases. My decision to join Q's Board was spurred by the quality of their science, the knowledge of its team and the need for therapies in diseases such as ALS. Having had both a … Continue reading →

ROCKVILLE, Md., March 15, 2013 /PRNewswire/ --Neuralstem, Inc. (NYSE MKT: CUR) today provided an update on its clinical trial programs and reported its 2012 financial results for the year ended December 31, 2012. (Logo: http://photos.prnewswire.com/prnh/20061221/DCTH007LOGO) "2012 saw the company achieve success in all of its Phase I clinical trial objectives," said Karl Johe, Ph.D., Neuralstem's Chairman of the Board and Chief Scientific Officer. "We have been able to demonstrate the safety and tolerability of Neuralstem's novel core technologies, from intraspinal transplantation procedures, to the cells themselves in ALS patients, as well as our NSI-189 neurogenic small molecule drug in healthy volunteers. Additionally, we believe we have seen evidence of a treatment effect in some NSI-566 cell therapy patients over a sustained period of time, as measured by levels of functional recovery and a slowdown in the progression of ALS. In spinal cord injury, a leading peer-reviewed scientific journal, 'CELL,' published compelling evidence that NSI-566 cells can 'bridge the gap' in a severed spinal cord animal model and return functionality.We have recently been approved by the FDA to commence a trial treating chronic spinal cord injury patients. A highlight of 2012 was the completion of our ground-breaking ALS Phase I trial. … Continue reading →

HENDERSON, Nev.--(BUSINESS WIRE)--Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, today announced the Company has gained global development and commercialization rights to Ligand Pharmaceuticals (NASDAQ: LGND) Captisol-enabled, propylene glycol-free (PG-free) melphalan. Captisol-enabled melphalan is currently in a pivotal trial for use as a conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma. We are pleased to have forged this agreement Spectrum is assuming the responsibility for the ongoing pivotal clinical trial and will be responsible for filing an NDA, which is anticipated in the first half of 2014. Under the license agreement, Ligand will receive a license fee and is eligible to receive milestone payments, as well as royalties following potential commercialization. We are pleased to add this late-stage program to our portfolio, which includes belinostat, for which we anticipate an NDA filing mid-year, and apaziquone, for which we expect to file an NDA in 2014, stated Rajesh C. Shrotriya, M.D., Chairman, President and Chief Executive Officer of Spectrum Pharmaceuticals, Inc. Captisol-enabled melphalan is designed to meet the need for a formulation of melphalan that is free of propylene glycol, which … Continue reading →

As she prepared to give birth to twins, Natalie Grafals recalled her brother's fight with cancer as a child. Her brother, now 35, survived the disease. She and her husband, Freddy, decided to bank their babies' stem cells after their birth. "I hope we never have to use it," said Grafals, 38, of Pompano Beach. "It is not cheap to do, but it's like an insurance policy for your children and your family as well." The Grafalses used the services of Boca Raton-based AssureImmune's private bank. With a new infusion of capital, AssureImmune plans to expand by opening a public bank. That would enable hospitals and doctors to find a potential match for a patient who needs a procedure. It can be difficult to find a match in a diverse region like South Florida, the company's top executives say. Six years ago, company founder Javier Zylberberg looked at rapidly advancing medical technologies and the promise of stem-cell repair for organs and disease, and offered the firm's services to collect cord blood as well as adult stem cells for private, future use. AssureImmune is now able to expand with an investment from a new equity partner. Former MDVIP concierge health care … Continue reading →

LEXINGTON, Mass.--(BUSINESS WIRE)-- Synageva BioPharma Corp. (Synageva) (GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare disorders, today reported 2012 full year financial results and provided 2013 financial guidance. Synagevas management team will host a conference call today at 4:30 p.m. EDT to review the financial results and provide a general business update. To participate in todays call via telephone, please dial (877) 312-5514 for U.S. callers, or (970) 315-0452 for international callers. In addition, the conference call will be webcast live from the Webcasts & Presentations section of the Investor Relations tab on the home page of Synagevas website at http://www.synageva.com. 2012 marked our first full year as a public company. Synageva made significant progress in advancing our lead program, sebelipase alfa. We announced recently that enrollment has started in the ARISE global, Phase 3 trial. This was a key step towards providing an effective therapy to patients suffering from LAL Deficiency, said Sanj K. Patel, President and Chief Executive Officer of Synageva. We continue to advance our other preclinical programs, and we entered 2013 with a strong cash balance which will be thoughtfully and strategically allocated to facilitate the achievement of our goals for 2013 and … Continue reading →

A recent FDA-approved combination of therapies used to target HER2-positive breast cancer does not lead to increased cardiac problems for patients, but doctors should regularly perform cardiac monitoring on their cancer patients until additional long-term cardiac safety data become available, according to the first phase III study of the combined treatments. The study, published in The Oncologist, was led by Dr. Sandra M. Swain, MD, Medical Director of the Washington Cancer Institute at Medstar Washington Hospital Center, in collaboration with an international team of researchers. Durham, NC (PRWEB) March 08, 2013 HER2 a type of protein found in more aggressive types of breast cancer is commonly treated with trastuzumab, which has been associated with certain types of cardiac dysfunction. A newer therapy, pertuzumab had undergone limited testing for cardiac safety both alone and in combination with trastuzumab, but Dr. Swains study, called CLEOPATRA, was the first phase III trial to study the use of trastuzumab, pertuzumab and docetaxel in patients with HER2-positive metastatic breast cancer receiving first-line treatment. A randomized, double-blind trial in which some patients received a placebo instead of pertuzumab, CLEOPATRA produced efficacy results that led the FDA last year to approve using a combination of trastuzumab, pertuzumab … Continue reading →