Category Archives: FDA Stem Cell Trials

Fraud & Misconduct Hidden from Journal Publishers & Public The documents were uncovered through freedom-of-information requests from Charles Seife, a professor at the Arthur L. Carter Institute of Journalism at New York University. Seife and his students cross-referenced FDAs actions against available published peer-reviewed studies. Their results are published in the most recent online issue of JAMA Internal Medicine, Medical News Today reports. The group found bad practices ranging from falsification of results to poor record keeping in the 57 trials that received action between 1998 and 2013. Official action indicated (OAI) is the FDAs most severe form of clinical trial violation. In the 2013 fiscal year, Seife reports, about 2 percent of the FDAs 644 inspections at trial sites were classified OAI. A study published in 2012, for example, makes positive claims about a stem cell treatment for 26 patients with severe loss of blood circulation to their legs. The study cited major clinical improvements for all, but did not mention that one patient had the leg amputated two weeks after the treatment. The FDA took action but Seife found no correction or retraction of the published results. In another case, the FDA deemed an entire study unreliable but … Continue reading →

Two emerging technologies look promising, but top oncology researchers are concerned about dangers seen during clinical trials. In some trials two new approaches have eliminated all traces of blood cancers in 40% to 90% of patients who had no remaining options. (Eric Gaillard, Reuters) A new wave of experimental cancer drugs that directly recruit the immune systems powerful Tcells are proving to be immensely effective weapons against tumours, potentially transforming the $100-billion global market for drugs that fight the disease. But top oncology researchers are concerned about the two emerging technologies, citing dangers seen repeatedly in clinical trials including the potentially fatal buildup of toxic debris from killed tumour cells and damage to healthy tissue. Such side effects could block regulatory approval if they arent controlled, say researchers and drug company executives. In some trials, the two new approaches, known as CAR T cells and bispecific antibodies, have eliminated all traces of blood cancers in 40% to 90% of patients who had no remaining options. The drugs could reap annual sales in the tens of billions of dollars for their manufacturers, especially if they can also eliminate solid tumours in such terminally ill patients. CAR T cells, or chimeric antigen … Continue reading →

Bristol-Myers Squibb Co. (BMY: Quote) has entered into a clinical collaboration agreement with Eli Lilly and Co. (LLY: Quote) to explore combination regimens from its immuno-oncology portfolio with other mechanisms of action that may accelerate the development of new treatment options for patients. As per the agreement terms, a phase 1/2 trial will evaluate Bristol-Myers Squibb's approved immunotherapy Opdivo in combination with Lilly's investigational Galunisertib as a potential treatment option for patients with advanced (metastatic and/or unresectable) glioblastoma, hepatocellular carcinoma and non-small cell lung cancer. Opdivo is approved by FDA for intravenous use for the treatment of patients with unresectable or metastatic melanoma while Galunisertib is currently under investigation as an oral treatment for advanced/metastatic malignancies, including phase 2 evaluation in hepatocellular carcinoma, myelodysplastic syndromes (MDS), glioblastoma, and pancreatic cancer. In other related news, Lilly has also entered into a collaboration agreement with Merck & Co. Inc. (MRK: Quote) to evaluate the safety, tolerability and efficacy of Merck's KEYTRUDA in combination with Lilly compounds in multiple clinical trials. Merck's KEYTRUDA was granted accelerated approval by FDA last September for unresectable or metastatic melanoma with disease progression following Ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor. BMY closed Tuesday's … Continue reading →

Contact Information Available for logged-in reporters only Newswise January 13, 2015 New York, NY Peter S. Kim has been named the Virginia and D.K. Ludwig Professor of Biochemistry at Stanford University School of Medicine. Established in 1994, Ludwig professorships have since been awarded to a total of 15 leading scientists at academic institutions affiliated with the six U.S.-based Ludwig Centers. With this appointment Kim also becomes a member of the Ludwig Center for Cancer Stem Cell Research and Medicine at Stanford. Kims lab focuses on the mechanisms by which viral membranes fuse with cell membranes, which has to happen for the virus to invade its target cell. His team also studies how that process might be disrupted by small molecules and antibodies. Kims lab is, for example, using such studies to engineer antigens for a vaccine that might elicit antibodies that block a key step in HIVs invasion of its target cell. The strategies that he is developing could be applied to design new preventive and therapeutic vaccines for cancers. His lab is also developing methods to identify small molecules that bind tightly and very specifically to proteins that have so far proved resistant to targeting by typical drug-like molecules. … Continue reading →

FDA and Adipose Stem Cells Several years ago we became fascinated with the potential of adipose stem cells for both cosmetic and medical purposes. However, we soon discovered that nothing in the written FDA guidelines specifically addressed the use of autologous adipose stem cells. Thus began our journey for an answer. In June 2009, we sent a letter to the FDA asking for a position statement on adipose stem cells. Our request focused specifically on autologous, freshly isolated, adipose stem cells for use in soft tissue reconstruction. These stem cells are from your own fat, for your own usage, and not culture expanded . After a very long wait, we recently received a written response from the FDA. First, a little bit of background for any stem cell newbies. Human cells and tissues intended for human transplant are regulated by the FDA. The FDA maintains two levels of classifications for cells and tissues: 1) HCT/P 361 and 2) HCT/P 351. uncultured stem cells from my own fat. a tissue or a drug? Category 361 is summarized as a tissue. A subset of category 361 includes procedures that take place in the same operative session . These same session operative procedures … Continue reading →

VAIL Regenerative medicine, including stem cells, seems miraculous, so of course the demand far outstrips the pace at which research can create a supply. The medical and scientific community are doing everything they can, but the demand for them always outpaces their availability. Research take a long time, said Dr. David Karli with the Steadman Clinic. The Vail Symposium is gathering three experts in the field to discuss whats available, what the limitations are, and whats in the pipeline. The program, The Patient Consumer: Navigating the Waters of Regenerative Medicine, takes place Thursday at Vail Mountain School. Along with Karli, Dr. Chris Centeno and Dr. Maynard Howe also are on the panel. OUTSTANDING PANEL The Vail Symposium had its first stem cell program last year, outlining the progress of stem cell science, said the Symposiums Dale Mosier. It was so fascinationg they decided to do one every year, Mosier said. This years presenters have been doing stem cell/regenerative medicine for many years and they have the successful history in different applications many patients to make for an outstanding panel, Mosier said. Thursdays panel will be moderated by Rohn Robbins. What is it? Regenerative medicine is anything that would facilitate regenerative … Continue reading →

When Pearland residents Todd and Linsey Hyatts son, Tucker Beau, received a diagnosis of Juvenile Rheumatoid Arthritis when he was just two years old, the future looked agonizing for the previously precocious little boy. His parents refused to lose hope, however, and took a chance on stem cell therapy to improve Tuckers quality of life. Now at age six, Tucker Beau looks and acts like any other normal boy his age, save for taking rests more often. He has had two separate stem cell infusions in August and November that have turned his slow, painful decline into a fading memory. Celltex Therapeutics Corp., a Houston-based biotechnology company located in the Galleria area, uses proprietary technology to isolate, multiply and store their clients own stem cells to be used for regenerative therapy. This therapy has been proven effective with many conditions, including vascular (e.g. Raynauds Disease, kidney artery disease), autoimmune (e.g. arthritis, multiple sclerosis, lupus) and degenerative (e.g. Parkinsons, Alzheimers) diseases. To get stem cells for a client, fat is extracted from the abdomen in a minimally invasive process that takes 15 30 minutes with no recovery time. This fat is then taken to Celltex, where the components of the fat … Continue reading →

According to Spectrum Pharmaceuticals, the Captisol-enabled Melphalan NDA was filed under a 505(b)(2) application and Spectrum expects FDA review to take approximately 10 months. Spectrum plans to launch this drug with its existing hematology/oncology sales force next year pending approval. We congratulate the team at Spectrum Pharmaceuticals on their development progress, said John Higgins, Ligands President and Chief Executive Officer. With a specialty sales force already in place, we look forward to Spectrums potential launch and commercial success with this valuable asset. Ligand licensedglobal development and commercialization rights toCE-Melphalan to Spectrum in 2013. Spectrum assumed the responsibility for a pivotal clinical trial and was responsible for filing the NDA. Under the license agreement, Ligand received a license fee and is eligible to receive revenue from Captisol material sales, milestone payments, as well as royalties on net sales following potential commercialization. According to previous announcements by Spectrum, the Phase 2 pivotal trial evaluating CE-Melphalan was a multicenter trial evaluating safety and efficacy. The primary objective of the study was to determine the overall safety and toxicity profile in multiple myeloma patients receiving 200 mg/m2 of CE-Melphalan as myeloablative therapy prior to autologous stem cell transplantation (ASCT). The secondary objectives evaluated the … Continue reading →

HENDERSON, Nev.--(BUSINESS WIRE)--Spectrum Pharmaceuticals (Nasdaq: 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 submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of Captisol-Enabled Melphalan (CE-Melphalan) HCl for injection (propylene glycol-free) for use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma. Spectrum is also seeking approval for the palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate. We believe the lack of propylene glycol in the preparation of CE-Melphalan eliminates the risk of the toxicities associated with that excipient, said Rajesh C. Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals. The improved solubility and stability of this novel melphalan formulation, CE-Melphalan, will make it an attractive treatment option for both transplant conditioning, and the palliative treatment of patients with multiple myeloma who cannot take oral melphalan. The enhanced stability of this new CE-Melphalan formulation simplifies clinical administration and is anticipated to allow for longer use and infusion times. This NDA submission represents another important step forward in bringing new … Continue reading →

PBR Staff Writer Published 15 December 2014 Ireland-based Jazz Pharmaceuticals has started rolling submission of a new drug application (NDA) to the US Food and Drug Administration (FDA) for defibrotide to treat severe hepatic veno-occlusive disease (VOD) in patients undergoing hematopoietic stem-cell transplantation (HSCT) therapy. The US FDA has also granted fast track designation to defibrotide for the treatment of severe VOD. In its severe form, VOD can be life-threatening and is related with multi-organ failure, while it is fatal in more than 80% of patients Jazz Pharmaceuticals executive vice-president and chief medical officer Jeffrey Tobias said: "Our start of the NDA submission for defibrotide marks an important step forward in our efforts to provide a treatment option for patients in the US who develop this rare, life-threatening complication of HSCT. "We expect to complete the submission of the NDA in the first half of 2015, at which time we will be requesting a Priority Review of the application, and we will continue to work closely with the FDA as we seek approval of the NDA. "As part of our commitment to ensure that eligible patients have access to defibrotide as we pursue US approval, we will continue to provide … Continue reading →