Category Archives: FDA Stem Cell Trials

Published 18 February 2014 California Stem Cell has announced that the US Food and Drug Administration (FDA) has approved the companys application to begin a Phase II clinical trial exploring the potential of a patient-specific cancer immunotherapy in women with Stage III or IV ovarian, fallopian tube or primary peritoneal cancer. The randomized, double-blind study is the company's second cancer therapy approved for clinical trials by the FDA, bolstering its platform approach to treating late stage solid-tumor cancers. The company's patient-specific approach to treating metastatic melanoma, recently approved to begin a Phase III trial, provided sufficient safety data to proceed directly to a Phase II study in ovarian cancer. Led by chief medical officer Dr Robert Dillman, the study design randomizes an estimated 99 adult female patients to two treatment arms. The treatment group will receive Ovapuldencel-T, which is a combination of autologous dendritic cells loaded with irradiated autologous tumor cells in GM-CSF. Ovapuldencel-T is created by first isolating cancer stem cells from the patient's resected tumor sample, then enriching, inactivating and combining these cells with dendritic cells - or antigen-presenting immune cells - which are harvested from the patient's blood. The control group will receive the 'MC' treatment, harvested … Continue reading →

Hey, isn't it great that university scientists are working on making embryonic stem cell therapies? They've done some especially promising work in treating certain causes of blindness, including age-related macular degeneration. But now a strange but serious roadblock has come up. Because of certain federal rules, the majority of embryonic stem cell therapies being studied in universities actually aren't eligible to become sellable treatments, the journal Cell Stem Cell reports. Universities usually do research on U.S. National Institutes of Health-registered embryonic stem cells. However, the NIH's rules don't match up with rules from the U.S. Food and Drug Administration, which would have to approve any embryonic stem cell-based therapy for use the U.S. So these university-created treatments technically aren't allowed to move past the research stage. What a dilemma! Historically, the FDA has allowed clinical trials of therapies created from NIH-registered embryonic stem cells, which suggests the administration knows what's going on and is willing to move stem cell therapies along. So maybe the rules will change in the future. For now, however, they're at an impasse. This is a new problem, brought on by the speed of the advancement of embryonic stem cell research. This is a pretty new … Continue reading →

The drug, BMN 673, is what's known as a PARP inhibitor, which targets cancerous cells while minimizing damage to normal tissue. It isdesigned specifically to treat women like Sloan who have a particular type of geneticovarian and breast cancer that presents a genetic mutation in the BRCA1 gene.Sloans doctors said it was her best and perhaps only chance to survive. The drug has not yet been approved by the Food and Drug Administration, but agency officials said they would provide a waiver to her for "compassionate use" of the treatment if the drug maker gave her permission to use it. But BioMarin Pharmaceutical Inc., the company that manufactures the potentially life-saving treatment, refused to let her take it. On New Years Day, Sloan, a former corporate lawyer who quit her high-paying job several years ago to run an organization that provides free legal services for low-income victims of domestic violence, died of complications from pneumonia. She was 45. Andrea very much wanted to live, but she also wanted to make a difference for other patients out there who may not have the resources she had to put up such a public fight, said Michelle Wittenburg, Sloans closest friend, who's an … Continue reading →

By RTT News, February 06, 2014, 07:26:00 AM EDT (RTTNews.com) - Spectrum Pharmaceuticals ( SPPI ), a biotechnology company with fully integrated commercial and drug development operations and a primary focus in Hematology and Oncology, said its New Drug Application or NDA for Beleodaq, a novel, pan-histone deacetylase or HDAC inhibitor, has been accepted for filing by the U.S. Food and Drug Administration or FDA. FDA establishes review classification for this application as Priority Review. The FDA has assigned a Prescription Drug User Fee Act or PDUFA action date of August 9, 2014. Spectrum is seeking FDA approval of Beleodaq for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma or R/R PTCL. Chief Executive and President of Spectrum Pharma noted, "The Priority Review designation for the Beleodaq NDA acknowledges the potential significant improvement in its safety or effectiveness for the treatment for patients with the serious condition of R/R PTCLs when compared to standard applications. Since approximately 70% of R/R PTCL patients fail the currently approved treatments in the course of their therapy, Beleodaq could be an important additional treatment option for these patients. Importantly, several patients treated with Beleodaq were able to go on to potentially … Continue reading →

San Diego, CA (PRWEB) January 27, 2014 Stemedica Cell Technologies, Inc. a leader in adult allogeneic stem cell manufacturing, research and development, announced today that Craig W. Carlson has been appointed the Companys Chief Financial Officer effective March, 2014. Craig Carlson brings 34 years of financial, accounting, investor relations, and operational experience specifically in the biotech and biopharmaceutical industries to his role at Stemedica. Over the past 21 years of his career, Craig has played an integral role in raising over $420 million from private and public markets and has also had significant international experience having been responsible for various subsidiaries in China, Korea and the United Kingdom. Craigs solid background in financial and accounting management along with his experience in SEC reporting, investor relations and in supporting financing initiatives in emerging growth companies is well-suited for Stemedica, said Roger Howe, PhD, Stemedicas Executive Chairman. I have confidence that under Craigs leadership, Stemedica will continue to improve our operating and financial efficiency and continue to create shareholder value. Previously, Craig was the Senior Vice President, Chief Financial Officer and Secretary at Talon Therapeutics, a specialty biopharmaceutical company, where he was responsible for all finance, accounting, SEC filings, information technology, and … Continue reading →

PROFESSOR MIODRAG STOJKOVIC/SCIENCE PHOTO LIBRARY Advanced Cell Technology is running the only US trials of embryonic-stem-cell therapies. Advanced Cell Technology (ACT), a biotechnology company based in Marlborough, Massachusetts, has long flirted with fame and bankruptcy. The company is running the only US Food and Drug Administration (FDA)-approved clinical trials of embryonic stem (ES)-cell therapies. Later this month, ACT plans to report preliminary results from three trials to test the safety of its treatment for two different forms of vision loss. If all goes well, it could be the first clinical demonstration of the safety and perhaps also the therapeutic potential of ES cells. Yet a series of financial missteps could cost ACT the opportunity to see that potential become reality. On 22 January, the firm announced that its chief executive, Gary Rabin, was stepping down. The news came a month after ACT which had US$5.5 million in cash on-hand as of 30 September 2013 announced that it would pay $4 million to settle a Securities and Exchange Commission (SEC) charge alleging that the company had illegally sold billions of shares of stock. Thats a big hit for any biotechnology company, says Gregory Bonfiglio, a venture capitalist with Proteus Venture Partners … Continue reading →

by Dan Browning A decade-long Mayo Clinic research project on using stem cells to repair damaged heart tissue has won federal approval for human testing, a step that could have implications for millions of Americans with heart disease. The U.S. Food and Drug Administration has approved a multistate clinical trial of 240 patients with chronic advanced symptomatic heart failure to see if the new procedure produces a significant improvement in heart function, Mayo officials announced Friday. Safety testing in humans, completed earlier in Europe, showed a preliminary 25 percent improvement in cardiac outflow, according to Dr. Andre Terzic, director of the Mayo Clinic's Center for Regenerative Medicine. The procedure could be a "paradigm shift" in the treatment of heart disease, Terzic said. Going forward, he said, treatments won't just focus on easing the symptoms of heart disease, but rather on curing it. The process, developed in collaboration with Cardio3 BioSciences of Belgium, involves harvesting stem cells from a patient's bone marrow in the hip, directing the cells to become "cardiopoietic" repair cells, then injecting them back into the heart to do their work. Mayo researcher Dr. Atta Behfar and other members of Terzic's team isolated hundreds of proteins involved in … Continue reading →

Orphan Drug Designation is granted to therapeutics intended to treat rare diseases that affect not more than 200,000 people in the United States. Orphan Drug Designation entitles the sponsor to a seven-year marketing exclusivity period, clinical protocol assistance with the FDA, as well as federal grants and tax credits. The Orphan Drug Designation was granted for use of BL-8040, in combination with granulocyte colony-stimulating factor (G-CSF), to mobilize human stem cells from the bone marrow to the peripheral blood for collection for autologous or allogeneic (donor-based) transplantation. "We are very pleased to have our second Orphan Drug Designation for BL-8040, in this case for stem cell mobilization treatment, after already having received the designation last year for the treatment of acute myeloid leukemia," stated Dr. Kinneret Savitsky, chief executive officer of BioLineRx. "This is in line with our 2014 development plan for BL-8040, which includes clinical studies for both AML and stem cell mobilization. BL-8040 was already shown to be highly effective in mobilizing stem cells in combination with G-CSF in a Phase 1/2 study in multiple myeloma patients, and initial results of BL-8040's Phase 2 study for AML patients indicate that BL-8040 on a stand-alone basis triggers substantial mobilization … Continue reading →

Israel's Pluristem Therapeutics ($PSTI) is back in the spotlight with some positive results for its stem cell therapy in treating patients with a muscle injury, giving the biotech some good news in its up-and-down development path. In the Phase I/II trial, Pluristem injected its placenta-derived stem cell therapy into patients who suffered gluteus muscle injuries during hip replacement surgery. Using muscle contraction force as an efficacy endpoint, the study found that patients who got a 150-million-cell dose of Pluristem's drug posted a 500% improvement over placebo, while those on a 300-million-cell dose beat out the control arm by 300%. The study met its safety endpoint, as well, the company said. The treatment, dubbed PLX-PAD, also had a significant effect on muscle volume, Pluristem said, suggesting a bright future for the stem cell therapy in repairing other injuries, CEO Zami Aberman said. "This was a very important study not only for Pluristem but for the cell therapy industry in general," Aberman said in a statement. "... Based on these results, we intend to move forward with implementing our strategy toward using PLX cells in orthopedic indications and muscle trauma." The company's shares jumped about 5% to $4.60 on the news, their … Continue reading →

A research project undertaken by the Mayo Clinic for nearly a decade has finally won approval from the U.S. Food and Drug Administration (FDA) to go ahead with testing on humans. The research project involves using stem cells to fix damaged heart tissue, and this step forward is a hopeful sign for millions of people who live with heart disease. The clinical trial will be carried out across several states and will involve 240 patients with chronic advanced symptomatic heart failure. It will help researchers discern whether the stem cell technique will make a marked improvement in heart function, the Mayo Clinic announced last week. The trial will probably take until the end of the year. Previously, Mayo had completed some testing in humans in Europe, which showed promising results a 25 percent improvement in cardiac outflow, Dr. Andre Terzic, director of the Mayo Clinics Center for Regenerative Medicine, said. Terzic called the technique a potential paradigm shift. The technique involves the harvesting of stem cells from a persons bone marrow in the hip, altering the cells to become cardiopoietic repair cells, and then injecting them into the heart to do their fixing work. The procedure was developed with help … Continue reading →