
Multistem is an investigational human stem cell product designed to treat strokes. The treatment of acute cortical ischemic stroke is currently being evaluated in a Phase 3, randomized, double-blind, placebo-controlled study. The study will enroll approximately 300 subjects across 60 sites worldwide, with the total study duration for safety follow-up being 12 months. The results from the Phase 3 trial, along with other clinical data, will form the basis for a regulatory submission seeking approval for Multistem. While the exact timeline for FDA approval is unknown, the completion of the Phase 3 trial and subsequent submission of the regulatory package are expected to be the next key steps in the process.
Characteristics | Values |
---|---|
Study Name | MultiStem Administration for Stroke Treatment and Enhanced Recovery Study-2 (MASTERS-2) |
Study Type | Phase 3, Randomized, Double-Blind, Placebo-Controlled |
Number of Patients | 300 |
Patient Location | North America and Europe |
Treatment | Single intravenous dose of MultiStem cell therapy or placebo |
Treatment Timing | Within 18-36 hours of the occurrence of the stroke |
Study Duration | 12 months |
What You'll Learn
MultiStem's Phase 3 clinical trial
MultiStem is an allogeneic cell therapy treatment that has been shown to be safe in clinical trials of myocardial infarction and graft vs. host disease. It has also demonstrated promising preclinical evidence of activity in stroke and other neurological damage models. The treatment consists of a population of multipotent adherent bone marrow cells.
The Phase 2 clinical trial of MultiStem was a randomized, double-blind, placebo-controlled, multicenter dose-escalation trial. It consisted of three treatment cohorts, including a placebo group and two treatment groups involving dose tiers of either 400 million or 1200 million cells per patient. The patients were treated 24-36 hours after their stroke. The two primary objectives were to determine the highest well-tolerated and safe single dose of MultiStem up to a maximum of 1200 million total cells in patients with ischemic stroke, and to determine the efficacy of MultiStem on functional outcomes in patients with stroke, as measured by the modified Rankin Scale at 90 days.
The Phase 2 trial also evaluated patients using the National Institutes of Health Stroke Scale and Barthel Index. Additionally, the study explored other aspects, including the unique measurement of spleen size after a stroke by magnetic resonance imaging or computed tomography imaging.
If the Phase 2 trial demonstrated that MultiStem was safe and effective, the plan was to proceed with a late-stage Phase IIb-III trial.
Platelet Counts and Strokes: Is There a Link?
You may want to see also
MultiStem's potential impact on stroke recovery
MultiStem is an investigational human stem cell product that has been developed to treat strokes. It is a patented regenerative medicine product that has shown the ability to promote tissue repair and healing in a variety of ways. The product is unique as it can be manufactured in a scalable manner, may be stored for years in frozen form, and is administered without tissue matching or the need for immune suppression.
MultiStem has been developed by Athersys, an international biotechnology company. The treatment has been designed to be administered intravenously to patients who have suffered an ischemic stroke, which is caused by a blockage of blood flow to the brain. This is the most common type of stroke and can lead to brain damage, temporary or permanent disabilities, and even death.
The Phase 2 clinical study of MultiStem demonstrated a significantly higher rate of complete or nearly full recovery for patients. The one-year data showed that MultiStem-treated subjects on average continued to improve through one year and had a significantly higher rate of "Excellent Outcome" compared to placebo subjects. The relative improvement was more pronounced in patients who received MultiStem treatment within 36 hours of the stroke.
The Phase 3 study of MultiStem is currently evaluating the benefit and potential side effects of the treatment. The study will enroll approximately 300 subjects who have experienced an acute cortical ischemic stroke across 60 sites worldwide. The total study duration for safety follow-up will be 12 months.
Stroke Symptoms: Gradual or Sudden?
You may want to see also
MultiStem's benefits and side effects
MultiStem is an intravenously-administered product developed using Multipotent Adult Progenitor Cells (MAPCs). MAPCs are derived from bone-marrow stroma and are non-hematopoietically adherent. They possess immunomodulatory and pro-angiogenic properties and are mostly non-immunogenic.
The main focus of MultiStem is immunological, with MAPCs producing factors that regulate the immune system, protect damaged and injured cells, and promote tissue repair and healing. The therapy acts by reducing inflammation and lessening the activation and migration of the peripheral immune system to the site of injury. It can also decrease activation of the peripheral immune response to the initial injury, resulting in reduced inflammation, scarring, and secondary tissue damage, while also allowing for accelerated repair processes.
Outside of immunological applications, MAPCs are also being investigated for ischemic injury treatment and cardiovascular repair. They have a stronger angiogenic protein release profile compared to MSCs and can induce more extensively developed vasculature. Additionally, MAPCs hold potential as a functional cell source for orthopedic applications and to promote tissue regeneration.
Evidence suggests that MAPCs can differentiate into cells of mesenchymal lineages, including bones, bone marrow, cartilage, fat, muscles, and tendons. This presents a potential treatment option for bone and cartilage diseases. Furthermore, MAPCs show a greater inclination for endothelial differentiation compared to MSCs.
Regarding side effects, as MultiStem is still being investigated, there may be adverse effects that have not yet been identified. However, the therapy's low immunogenicity means it can be administered without tissue matching or immunosuppressive agents, reducing potential complications.
Stroke Victims: Workplace Rights and Filing for Compensation
You may want to see also
MultiStem's development and regulatory pathway
MultiStem is a patented regenerative medicine product developed by Athersys, an international biotechnology company. It is a unique "off-the-shelf" stem cell product derived from bone marrow that can be manufactured in a scalable manner and has a long shelf life. The product has been shown to promote tissue repair and healing through the production of therapeutic factors in response to inflammation and tissue damage.
In 2012, MultiStem was in Phase II clinical trials for the treatment of inflammatory bowel diseases, acute myocardial infarction, and ischemic stroke. Safety studies (Phase I clinical trials) had also been completed, demonstrating that MultiStem administration was well-tolerated and reduced the incidence and severity of Graft-versus-Host Disease (GvHD).
In September 2016, Athersys received agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the design and planned analysis of a Phase 3 clinical trial of MultiStem for the treatment of ischemic stroke. The SPA clarified and de-risked the development pathway for MultiStem, providing agreement from the FDA on the acceptability of the protocol design, clinical endpoints, planned conduct, and statistical analyses to support a regulatory submission for approval.
The Phase 3 clinical trial, "MultiStem Administration for Stroke Treatment and Enhanced Recovery Study-2" (MASTERS-2), was designed as a randomized, double-blind, placebo-controlled trial enrolling 300 patients in North America and Europe who had suffered moderate to severe ischemic stroke. The enrolled subjects would receive either a single intravenous dose of MultiStem cell therapy or a placebo, in addition to the standard of care. The primary endpoint would evaluate disability using the modified Rankin Scale (mRS) scores at three months, comparing the distribution or "shift" between the MultiStem treatment and placebo groups.
The results from the MASTERS-2 trial, along with other available clinical data, would form the foundation of the regulatory package to be submitted for marketing approval. Athersys stated that the SPA clarified and de-risked an accelerated development pathway, meaning that the successful completion of the MASTERS-2 trial could enable them to apply for marketing approval in the United States.
In addition to the FDA agreement, Athersys also received acceptance from Japan's Pharmaceutical and Medical Devices Agency (PMDA) in September 2016 to commence a confirmatory clinical trial evaluating the safety and efficacy of MultiStem for the treatment of ischemic stroke in Japan. This trial was part of a partnership and license agreement between Athersys and Healios K.K. (Healios), focused on the development and commercialization of novel cellular therapies for ischemic stroke and potentially other indications.
While the above information provides insights into the development and regulatory pathway for MultiStem, it is important to note that the timeline for FDA approval can vary depending on various factors, including the results of clinical trials and the specific requirements of the regulatory agencies involved. Therefore, it is challenging to provide an exact timeline for when MultiStem might be approved by the FDA for stroke treatment.
Strokes in the Hispanic Community: Annual Incidents and Insights
You may want to see also
MultiStem's marketing approval
The MultiStem cell therapy product is being developed by Athersys, an international biotechnology company. MultiStem has received FDA agreement under a Special Protocol Assessment (SPA) for the design and planned analysis of a Phase 3 clinical trial for the treatment of ischemic stroke. The SPA provides agreement from the FDA that the protocol design, clinical endpoints, planned conduct, and statistical analyses encompassed in Athersys' planned Phase 3 study are acceptable to support a regulatory submission for approval.
The Phase 3 clinical trial, known as MASTERS-2, will be a randomized, double-blind, placebo-controlled study enrolling 300 patients in North America and Europe who have suffered moderate to moderate-severe ischemic stroke. The enrolled subjects will receive either a single intravenous dose of MultiStem cell therapy or a placebo, in addition to the standard of care. The primary endpoint will evaluate disability using modified Rankin Scale (mRS) scores at three months, comparing the distribution or "shift" between the MultiStem treatment and placebo groups.
The results from the MASTERS-2 trial, along with other available clinical data, will form the foundation of the regulatory package to be submitted for marketing approval. According to Dr. Gil Van Bokkelen, Chairman and Chief Executive Officer of Athersys, the SPA is important as it clarifies and de-risks an accelerated development pathway, meaning that the successful completion of the MASTERS-2 trial could enable them to apply for marketing approval in the United States.
The MultiStem cell therapy product is a patented regenerative medicine product that has shown the ability to promote tissue repair and healing through the production of therapeutic factors in response to signals of inflammation and tissue damage. It is a unique "off-the-shelf" stem cell product that can be manufactured in a scalable manner, stored for years in frozen form, and administered without tissue matching or the need for immune suppression.
Heat Stroke: Quick Onset, Deadly Results
You may want to see also
Frequently asked questions
MultiStem is an investigational human stem cell product designed to treat strokes.
MultiStem is a regenerative medicine product that promotes tissue repair and healing through the production of therapeutic factors in response to inflammation and tissue damage signals.
MultiStem is currently in Phase 3 of its clinical trial, which is a randomised, double-blind, placebo-controlled study with 300 patients enrolled.
The FDA has agreed to the design and planned analysis of the Phase 3 clinical trial. If the trial is successful, MultiStem could be approved for marketing in the United States.
MultiStem has the potential to help stroke patients who do not have access to or did not benefit from other therapies. It is a unique "off-the-shelf" product that can be manufactured at scale, stored for years in frozen form, and administered without tissue matching or immune suppression.