Overview

We are developing MultiStem®, our proprietary stem cell product, for the treatment of diseases and conditions in the neurological, inflammatory and immune, and cardiovascular disease areas, as well as other indications. Working with independent clinical and research institutions in the United States and abroad, we have established clinical trials in each of these areas. After isolation from a qualified donor, the MultiStem product may be expanded on a large scale, unlike other cell types, for future clinical use and stored frozen until needed. Cells obtained from a single donor undergo no genetic manipulation or modification and may be used to produce banks yielding hundreds of thousands to millions of doses of the MultiStem product – an amount that substantially exceeds other unmodified stem cells. Each MultiStem cell bank is extensively characterized to ensure product consistency and safety.

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FAQs

What is the source for MultiStem?

The MultiStem clinical product is developed from a special class of stem cells called Multipotent Adult Progenitor Cells, or MAPC®. These cells are distinct from mesenchymal stem cells (MSCs) and other cell types. They are obtained from healthy adult bone marrow and may be expanded outside the human body using proprietary manufacturing processes. These cells may be administered without tissue matching or immune suppression, and results show they are capable of promoting healing and tissue repair in multiple ways.

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What makes MultiStem different than other stem cells?

Drawing on our many years of research with the MAPC cells, we have developed a method to culture and expand the MultiStem product candidate very efficiently. Unlike some stem cell types that grow old or senesce over time and are hard to grow over multiple passages in the lab, a modest amount of MultiStem cells may be used to create a cell bank with the potential to yield millions of doses.

In addition, unlike fetal or embryonic stem cells, which require the harvesting of fetal or embryonic tissue to obtain the cells, there are none of these ethical concerns for MultiStem cell therapy. The cells are derived from the bone marrow of healthy, consenting adult volunteers.

Autologous stem cells (i.e., cells derived from the patient) have shown promise in some clinical areas but can have disadvantages, as well. The patient is required to undergo a procedure to isolate their stem cells, and the cells taken from the patient may need to be processed, modified and/or expanded after extraction in order to obtain enough cells to provide benefit. This expansion time can take days or even weeks, losing valuable time that might be particularly important in the case of patients who have suffered a stroke or other acute conditions. In addition, autologous cells obtained from older patients may not have the same therapeutic profile as cells obtained from a young, healthy donor source.

As we have demonstrated through our many clinical and preclinical studies, tissue matching is not required for administration of MultiStem cell therapy due to the nature of this special class of human stem cells, nor is administration of immune suppressive agents necessary (which is a typical requirement for transplantation or other donor-derived cell therapies).

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What does “off-the-shelf” mean?

MultiStem cell therapy represents an “off-the-shelf” product candidate, meaning the cell suspension may be conveniently stored frozen in a vial. The vial can be quickly thawed and administered to a patient, typically through a simple intravenous infusion. MultiStem can be administered like Type-O blood, as tissue matching is not required due to the nature of this special class of human stem cells, nor is administration of immune suppressive agents.

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How is MultiStem administered to a patient?

MultiStem cells are typically administered through a simple intravenous infusion. The cells are stored frozen in a vial until needed. The frozen suspension of cells in the vial is thawed when needed, the liquid cell suspension is removed from the vial using a sterile syringe and the cells are then added to an IV bag. The cells can be prepared for the patient in less than one hour and administered intravenously. Tissue matching is not required, nor are immune suppressive agents.

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How does MultiStem work?

Published research shows that MultiStem cell therapy works in several ways. The cells can express a range of therapeutically relevant proteins and other factors that have the potential to deliver therapeutic benefits in multiple ways, such as reducing inflammation, protecting damaged or injured tissue, and enhancing the formation of new blood vessels in regions of ischemic injury. These cells exhibit a “drug-like” profile, in that they act primarily through the production of factors that regulate the immune system, protect damaged or injured cells, and promote tissue repair and healing. The cells are subsequently cleared from the body over time – much like a traditional drug or biologic treatment.

Click here to learn more about the mechanisms of action.

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Does Athersys have patents on MultiStem?

Athersys has exclusive intellectual property rights to the MAPC technology that comprises the MultiStem product candidate. We have a broad intellectual property estate that covers our proprietary product candidates and technologies, as well as methods of production and methods of use. Our intellectual property is important to our business, and we take significant steps to protect its value. We have ongoing research and development efforts, both through internal activities and through collaborative research activities with others, which aim to develop new technologies, applications and intellectual property and also enable us to file patent applications that cover new applications of our existing technologies or product candidates. We currently have over 300 patents related to our technologies, providing protection in the United States, Europe, Japan and other areas.

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Is MultiStem FDA approved?

We are currently running clinical trials to evaluate the safety and efficacy of our MultiStem product candidate, under the guidance of the U.S. Food and Drug Administration, or FDA. Upon successful completion of registrational trials, we may be eligible to apply for approval to commercialize our products. Our most advanced clinical study is our MASTERS-2 clinical trial, which is a Phase 3 trial to evaluate the potential for MultiStem treatment of patients who have suffered an ischemic stroke. Among other regulatory designations, the FDA has agreed under a special protocol assessment that our MASTERS-2 trial is adequately designed to support a biologics license application if it is successful. We strongly support the FDA’s mission, and other leading regulatory agencies, to protect the safety and well-being of patients.

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