January 18, 2019 News by Patricia Inacio, PhD Blood Stem Cell Transplant Better than DMTs at Reducing Risk of Disease Progression in RRMS Autologous hematopoietic stem cell transplant is better than disease-modifying therapies (DMT) at reducing the risk of disease progression in patients with relapsing-remitting multiple sclerosis (RRMS), results from the MIST clinical trial show. The study āEffect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression…
April 27, 2018 News by Alice MelĆ£o, MSc #AAN2018 ā Stem Cell Transplant is Effective Treatment for ‘Aggressive’ MS, Study Shows Autologous hematopoietic stem cell transplantation, also known as aHSCT, has been shown to be safe and highly effective to treat patients with "aggressive" multiple sclerosis. Tested in 19 patients, transplantation of stem cells was found to induce clinically meaningful improvements in disability. These findings were shared at the 2018 Annual Meeting of the American Academy of Neurology (AAN) in Los Angeles, California. aHSCT uses a patientās own healthy bone marrow stem cells, in combination with a much less aggressive chemotherapy and/or radiation regimen, to prepare the patient for the transplant. Previous studies have suggested that aHSCT is an effective strategy to treat patients with highly active relapsing-remitting MS (RRMS) who do not respond to available disease-modifying therapies (DMTs), and international guidelines advocate for its use in patients with "aggressive" MS. To further demonstrate the potential of aHSCT as a treatment for "aggressive" MS, a research team evaluated its safety and effectiveness in MS patients who had not been treated previously with DMTs. A total of 19 patients were treated across several clinical centers: seven patients were from Sheffield, U.K., seven from Uppsala, Sweden, four from Ottawa, Canada, and one patient was from Florence, Italy. All patients received aHSCT between May 2004 and May 2017. In addition to aHSCT, patients were treated with BEAM (carmustine, etoposide, cytarabine, melphalan) chemotherapy plus antithymocyte globulin (ATG) to reduce transplant rejection, or with Cytoxan (cyclophosphamide) with ATG, or the triple combination of Cytoxan, ATG, plus busulfan as conditioning regimens. Patients had a median age of 33 years at diagnosis and received the aHSCT by a median time of nine years after symptom onset. They had a median disability score of 6.5 before the treatment, as determined by the Expanded Disability Status Scale (EDSS). After a median follow-up period of 30 months, patients had a median EDSS score of 2.0, which represented a median improvement of 2 points (the higher the score, the worse the patient's disability level). None of the patients had clinical relapse following the transplant of stem cells. Only three patients developed new brain lesions detectable by magnetic resonance imaging (MRI) at the first six-month follow-up evaluation, but no additional new lesions were detected in the following scans. The adverse effects reported during the study were comparable to those previously observed in similar treatments. No deaths related to the treatment were reported. Based on these preliminary results, the researchers concluded that aHSCT is āsafe and highly effective in inducing rapid and sustain remissionā in highly active MS, and "was associated with a significant improvement of [patientās] level of disability.ā āaHSCT should be considered as first line therapy in patients with āaggressiveā MS,ā the team concluded. Another study presented at the AAN 2018 meeting further supports these findings, demonstrating the superior effectiveness of aHSCT over conventional DMTs for RRMS.
April 23, 2018 News by Patricia Inacio, PhD #AAN2018 – Blood Stem Cell Transplant Superior to DMDs in Highly Active RRMS, MIST Trial Shows Autologous non-myeloablative hematopoietic stem cell transplant was found to be significantly better at reducing risks for disability in relapsing-remitting multiple sclerosis (RRMS) patients compared to disease-modifying drug (DMD) therapies, interim results of the MIST clinical trial show. The results will be shared at the 2018 Annual MeetingĀ of theĀ American…
February 19, 2018 Columns by Ed Tobias MS News that Caught My Eye Last Week: Stem Cell Trial, Medical Marijuana, Myelin Repair, Tysabri Study Stem Cell Treatment Benefits Three-fourths of MS Patients in Phase 1 Trial This is encouraging news for MS patients hoping to see some action in the stem cell area. A Phase 1Ā mesenchymal stem cell trial is reporting positive results, and a Phase 2 trial is underway in…
February 14, 2018 News by Alice MelĆ£o, MSc Stem Cell Treatment Benefits Three-fourths of MS Patients in Phase 1 Trial A stem cell treatment improved the neurological symptoms of three-fourths of the multiple sclerosis patients in a Phase 1 clinical trial, New York researchers reported. The results prompted the team at theĀ Tisch MS Research Center of New YorkĀ to start a Phase 2 trial to further assess the therapy’s…
January 9, 2018 News by Alice MelĆ£o, MSc American CyroStem Warned by FDA to Stop Using Stem Cell Therapy Without Approval and to Correct Safety Issues American CryoStem has received a warning letter from the U.S. Food and Drug Administration (FDA) for marketing its adipose-derived stem cell product Atcell without required regulatory approval, and for "significant deviations" from manufacturing processes that potentially raise safety concerns. The company has 15 working days to respond to the concerns raised by the agency and detail how they will be corrected, or risk "enforcement actions," the FDA said in a Jan. 4 press release. StudiesĀ suggest that mesenchymal stem cells can be used to alleviate symptoms and possibly treat several degenerative disorders, including multiple sclerosis. Atcell is aĀ therapy based on the ability of mesenchymal stem cells isolated from adipose tissue (fatty tissue) to transform into a subset of mature cells, which include adipose cells, bone cells, and cartilage cells. Although not approved for use, Atcell is being distributed directly to physicians to treat patients affected by several life-threatening diseases, including Parkinsonās disease, amyotrophic lateral sclerosis, stroke, and multiple sclerosis, the FDA said in its release. It is administeredĀ intravenously, intrathecally (injection or infusion into the central nervous system) or by aerosol inhalation. The productĀ is designed to be used in the same individual (autologous use) the cells are collected from, an approach intended to reduce risk. The cells are extractedĀ using the companyās proprietary Cellect collection system. They are then expanded in the laboratory using the company's ASCelerate SF-M serum free (animal-product free) media,Ā providing compounds needed for the cells to survive and proliferate.Ā Stem cells put through this process are Ā ready to be used as therapy or to be stored for future use. A recent FDA inspection found that Atcellās manufacturing steps are not in line withĀ current good manufacturing practice requirements. Specifically, the manipulation of the adipose tissue was more than "minimal," the FDA reported, changing "relevant characteristics" of the original tissue that could introduce contamination by microorganisms or product defects and represent a "risk of harm" to patients. Because of this manipulation, FDA review is required by law to ensure Atcell's safety and efficacy, the agency said. Evidence of an inadequately controlled manipulation environment, lack of control of components used in production, andĀ insufficient and inadequately validated product testing were also reported. āThe use of Atcell raises potential significant safety concerns, due in part to the fact that there is little basis on which to predict how the product will perform in a patient,ā Peter Marks, director of the FDAās Center for Biologics Evaluation and Research, said in the release. āIn addition, this product may also cause harm to patients who may put their trust in an unproven therapy and make the decision to delay or discontinue medical treatments proven to be safe and effective,ā Marks added. American CyroStem, based in New Jersey, did respond to observations raised by the FDA at the time of its inspection. But they were found inadequate to support Atcellās marketing, and failed to acknowledge that FDA approval was required, either by filing a biological license or investigational new drug application. āAs part of our comprehensive policy framework for the efficient development and regulation of cell-based regenerative medicine, weāre going to be stepping up enforcement activities against those who manufacture and market products in ways that put patients at risk,ā said Scott Gottlieb, FDA commissioner. āWe see great promise from the field of cell based regenerative medicine, but there are also novel risks,ā Gottlieb added. Healthcare professionals and patients who have used Atcell are asked to report any adverse events related to the treatment using the FDAāsĀ MedWatch Online Voluntary Reporting Form. Completed forms can be submitted online, or via fax to 1-800-FDA-0178. Ā
September 25, 2017 Columns by Ed Tobias MS News That Caught My Eye: Diagnostic Blood Test, Fatigue, New Trials and Stem Cell Therapy IQuity Taking Orders for RNA-based Blood Test That Can Detect MS Early with 90% Accuracy Can it be that there’s now a blood test that can help diagnose MS? This company says it has one and doctors can order it. For a disease that’s always been…
July 10, 2017 News by Charles Moore Experts Call for Tighter Regulation of Stem Cell Therapies in Use at Clinics Worldwide Advertising for stem cell therapies not supported by clinical research ā often made directly to patients and sometimes promoted as a "cure" for diseases like multiple sclerosis or Parkinson's ā is a growing problem that needs to be addressed and regulated, a team of leading experts say, calling such "stem cell tourism" potentially unsafe. Stem cell tourism is the unflattering name given to the practice of encouraging patients to travel outside their home country to undergo such treatment, typicaly at a private clinic. The article, titled "Marketing of unproven stem cellābased interventions: A call to action" and recently published in the journal Science Translational Medicine, was co-authored by scientists with universities and hospitals in the U.S., Canada, U.K., Belgium, Italy, Japan, and Australia. It focuses on the global problem of the commercial promotion of stem cell therapies and ongoing resistance to regulatory efforts. Its authors suggest that a coordinated approach, at national and international levels, be focused on "engagement, harmonization, and enforcement in order to reduce risks associated with direct-to-consumer marketing of unproven stem cell treatments." Treatments involving stem cell transplants are now being offered by hundreds of medical institutions worldwide, claiming efficacy in repairing tissue damaged by degenerative disorders like MS, even though those claim often lack or are supported by little evidence . They also noted that the continued availability of these treatments undermines the development of rigorously tested therapies, and potentially can endanger a patient's life. The researchers emphasize that tighter regulations on stem cell therapy advertising are needed, especially regarding potential clinical benefits. They support the establishment of international regulatory standards for the manufacture and testing of human cell and tissue-based therapies. "Many patients feel that potential cures are being held back by red tape and lengthy approval processes. Although this can be frustrating, these procedures are there to protect patients from undergoing needless treatments that could put their lives at risk," Sarah Chan, a University of Edinburgh Chancellorās Fellow and report co-author, said in a news release. Chan and her colleagues are also calling for the World Health Organization to offer guidance on responsible clinical use of cells and tissues, as it does for medicines and medical devices. "Stem cell therapies hold a lot of promise," Chan said, "but we need rigorous clinical trials and regulatory processes to determine whether a proposed treatment is safe, effective and better than existing treatments." According to the release, the report and its recommendations followed the death of two children at a German clinic in 2010. The clinic has since been shut down. Certain stem cell therapies ā mostly involving blood and skin stem cells ā have undergone rigorous testing in clinical trials, the researchers noted. A number of these resulted in aproved treatments for certain blood cancers, and to grow skin grafts for patients with severe burns. Information about the current status of stem cell research and potential uses of stem cell therapies is available on the website EuroStemCell.
February 14, 2017 Columns by Ed Tobias Stem Cell Treatment for MS: Can’t We Move Any Faster? There is some good news about stem cell therapy. A just-published study concludes that one form of human stem cell therapy is more effective at treating multiple sclerosis than the best of the MS medications being used currently. The not-so-good news is that approval of this…
November 21, 2016 News by Patricia Silva, PhD New Company Specializing in Stem Cell Platform for MS and Other Ills Raises $48.5M Magenta TherapeuticsĀ has completed itsĀ first round of financing, raisingĀ $48.5 million to develop ways of bringing bone marrow stem cell transplants to more patients with autoimmune diseases, such as multiple sclerosis (MS), among other illnesses. The new companyĀ aimsĀ to develop the first complete platform that can overcomeĀ the challenges in stem cell transplants,…
June 13, 2016 Columns by admin Stem Cell MS Patients Lead Normal Life Thanks To aHSCT Procedure People with MS who were involved in a long-term clinical trial are out and about enjoying a full and normal life with no signs of the disease. This follows their recovery from the stem cell transplants involving aggressive chemotherapy, or aHSCT as the procedure is known. Results of the clinical…
March 24, 2016 News by admin MS Animal Study Suggests That Placental Cells Might Serve as Future Stem Cell Therapy Research on a specific type ofĀ stem cell found in the placenta, known asĀ decidua-derived mesenchymal stem cells (DMSC), suggests these cells might be the source of futureĀ treatments for multiple sclerosis. The report,Ā “Restrained Th17 response and myeloid cell infiltration into the central nervous system by human decidua-derived mesenchymal stem…
October 15, 2015 News by Maureen Newman The Future of Stem Cell Therapy for Multiple Sclerosis Patients Relies on Clinical Trial Participation Among the different therapeuticĀ approaches being explored for treating MS,Ā adult stem cell therapy continues to beĀ one of the most discussed and anticipatedĀ in the MS community. āStem cellsā ā the common term for undifferentiated, self-renewing proliferating cells āĀ are currently being investigated for their ability to treatĀ patients in a wide range of disease…
September 7, 2015 News by admin New Study Unravels How Myelin is Repaired, May Suggest New MS Treatments Japanese scientists have discovered new information about howĀ the myelin sheath is repaired following damage. Myelin is a fatty substance that wraps around nerve cells and helps them to conduct impulses. The researchĀ could have major implications for how multiple sclerosis is understood and even treated.Ā The study, titled “Inactivation…
June 4, 2015 News by Patricia Silva, PhD Another Study Focused on Stem Cells and Autoimmune Diseases Shows Promise Researchers at Monash University and the MIMR-PHI Institute of Medical Research in Australia recently proposed that specific human stem cells with immunomodulatory properties represent a new promising therapeutic strategy for diseases like multiple sclerosis (MS). The study was published in the Journal of Neuroinflammation and…
May 15, 2015 News by Patricia Silva, PhD Singer Releases Song About MS Recovery After Receiving Stem Cell Therapy Singer Kristen King began to experience fatigue, muscle weakness and later paralysis in some parts of the body in 2013, and it came asĀ a shock when she was diagnosed withĀ multiple sclerosis (MS). After struggling to deal with the unfortunate newsĀ and her new reality, the singer has now…
April 22, 2015 News by admin Two Already-Approved Medications Could Treat MS Two already available medicationsĀ could be used to treatĀ multiple sclerosis (MS). In a newĀ study titled, “Drug Based Modulation of Endogenous Stem Cells,” published in the journal NatureĀ on April 20, 2015, scientists report that twoĀ drugs couldĀ activate stem cells in the brain, possibly repairing MS-induced damage to…
April 9, 2015 News by Patricia Silva, PhD MS Patient Travels Abroad to Receive Stem Cell Treatment AĀ stem cell therapyĀ being administeredĀ to treat multiple sclerosis (MS) outside the United States was recently touted by an MSĀ patient who traveled to Mexico to receive the therapy, revealing significantĀ improvements in herĀ quality of life.Ā Debbie Bertrand, an MS patient who was diagnosed in 2001, is sharing her…
February 16, 2015 News by Maureen Newman Stem Cells Used to Treat Secondary Progressive Patients in Clinical Trial What may work better than existing drugs to treat severe multiple sclerosis? Stem cells. A phase 2 clinical study from an international group of research centers compared head-to-head autologous hematopoietic stem cell transplantation (AHSCT) and mitoxantrone in treating patients with secondary progressive or relapsing-remitting multiple sclerosis. The findings showed that…
February 10, 2015 News by Patricia Silva, PhD Creative Medical Seeks Patent for Stem Cell Therapy for MS Phoenix, Arizona-basedĀ Creative Medical Health (CMH) has just announced it has submitted a patent application for its proprietary mesenchymal stem cell pipeline product, indicated for autoimmune diseases. The companyĀ is already preparing to launch the product into its first line of clinical tests, which will…
September 3, 2014 News by Patricia Silva, PhD Researchers Advance New Stem Cell Methods for Remyelination in MS Patients In-vitro laboratory experiments using stem cells have long-since been the subject of debate between scientists and naturalists, but when it comes to developing effective methods to grow human cells and tissue for use in a potentially life-saving procedure or restoration of bodily function, researchers are increasingly more confident that…
August 4, 2014 News by Patricia Silva, PhD UPDATE: Australian Authorities Warn About Unapproved MS Stem Cell Treatments After Death in Russia Australian authorities are alerting Multiple Sclerosis patients to the risk of unproven stem cell treatments that are being performed both in the country and overseas, after the recent death of a woman in Russia undergoing a controversial treatment for the disease. The continued marketing campaigns to…
July 18, 2014 News by Patricia Silva, PhD Startup ImStem Raising Funds For Novel Stem-Cell MS Treatment Startup biotech company ImStem BiotechnologyĀ is looking to raise additional funding for their upcoming clinical trials after publishing promising research on a unique stem-cellĀ treatment used on mice withĀ multiple sclerosis. Ā The company has already received previous funding…
May 16, 2014 by Charles Moore Researchers Report Encouraging Results in Mouse Model MS Stem Cell Therapy Research Scientists at the Scripps Research Institute (TSRI), the University of California (UC), Irvine and The University of Utah report that mice crippled by an autoimmune disease similar to multiple sclerosis (MS) regained the ability to walk and run after a team of researchers implanted human stem cells…