New Mesenchymal Stem Cell Population from Human Pluripotent Stem Cells Displays Potent Immunomodulatory and Therapeutic Properties
4:55 PM ET 3/24/14
Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC), a leader in the field of regenerative medicine, and its collaborators reported today that it has discovered a new method to generate a potent and replenishable population of mesenchymal stem cells (MSCs) from pluripotent stem cells. The research appears online ahead of print in Stem Cell and Development, one of the top stem cells journals, published by Mary Ann Liebert, Inc. This new and proprietary population of pluripotent stem cell-derived MSCs displays potent immunomodulatory and therapeutic properties and has a greater than 30,000 fold proliferative capacity, relative to ordinary bone marrow-derived MSCs, the most commonly used source for MSCs in clinical trials. The paper is available free online at online.liebertpub.com.
There are currently over 300 clinical trials evaluating MSC therapeutic utility in a variety of diseases. Unlike other types of cellular therapies, MSCs can be used in allogeneic settings without immunosuppressive therapy due to the cells' ability to evade immune detection. MSCs home to injured tissue and provide therapeutic support through a multifaceted mechanism. They secrete a dynamic assortment of bioactive cytokines, trophic factors, and anti-inflammatory molecules in response to environmental cues. The traditional sources of MSCs are from adult tissues and have limited expansion capacity and so must be constantly replenished from more donors, and screened for pathogens. Moreover, there is an appreciable loss of potency upon propagation of adult MSCs in culture, which along with inconsistent quality of current MSC sources, limits the scalability of their use in therapy. We believe our new method can overcome these limitations as we have taken advantage of a versatile precursor cell called the "hemangioblast." hESC-derived hemangioblasts replace the need for donors, and our pre-clinical studies have demonstrated these hESC-MSCs have the capacity to respond to environmental cues, influence immune cell function, and exert therapeutic effects to reduce clinical symptoms in two different autoimmune disease models.
"This population of MSCs may have a therapeutic effect that could overcome many of the obstacles that currently plague the use of stem cells in regenerative medicine and may serve as a scalable alternative to current MSC sources," said Robert Lanza, MD, Chief Scientific Officer at ACT, and senior author of the study. "In addition to being easy to derive in large numbers, they appear to be more potent than adult MSCs and have a longer duration of action. If these cells prove to have a better therapeutic index than adult MSCs, they may provide for treatments of inflammatory and autoimmune conditions for which adult MSC treatment is currently not tenable. As reported in the study, the cells preserved kidney function and increased the lifespan of animals with lupus. There is currently no cure for this devastating disease. In our study, most untreated animals died in the first few months, whereas all of the animals treated with two injections of our MSCs survived during the same time period. These new cells also had a therapeutic effect in animals with both mild and severe uveitis. In humans this disease accounts for approximately 10% of blindness in the United States."
"We are encouraged by the publication of these study results and we view our MSC program to be a promising component of our pre-clinical pipeline," stated Ted Myles, Interim President, CFO and EVP of Corporate Development, of ACT. "Drs. Lanza and Kimbrel, and their colleagues, have demonstrated that MSCs derived from pluripotent stem cells may provide a viable alternative to adult-derived MSCs. ACT is currently running a number of additional studies in other animal disease models that may help to define the range of indications in which hESC-MSCs could provide therapeutic benefit."
The paper's other authors are Erin A. Kimbrel (first author of the paper), Nicholas A. Kouris, Gregory Yavanian & Jianlin Chu of ACT; and Yu Qin, Ann Chan, Ram P. Singh, Deborah McCurdy, Lynn Gordon, and Ralph D. Levinson of the Division of Rheumatology and Department of Ophthalmology, Jules Stein Eye Institute, David Geffen School of Medicine at UCLA.
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc. is a Marlborough, Mass.-based biotechnology company focused on the development and commercialization of human embryonic stem cell (hESC) and adult stem cell technology. The company's most advanced products are in clinical trials for the treatment of dry age-related macular degeneration and Stargardt's macular degeneration. ACT's preclinical programs involve cell therapies for the treatment of other ocular disorders and for diseases outside the field of ophthalmology, including autoimmune, inflammatory and wound healing-related disorders. The company's intellectual property portfolio includes pluripotent stem cell platforms - hESC and induced pluripotent stem cell (iPSC) - and other cell therapy research programs. For more information, visit advancedcell.com.
Forward-Looking Statements
Statements in this news release regarding future financial and operating results, the relevance and applicability of clinical trials in animals to studying the effect of products in humans, future growth in animal and human research and development programs, potential new applications of and expanded indications covering our technology, the potential therapeutic application of donorless sources of stem cells , and any other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "will," "believes," "plans," "anticipates," "expects," "estimates," and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements, including: limited operating history, need for and limited sources of future capital, failures or delays in obtaining regulatory approval of products, risks inherent in the development and commercialization of potential products, reliance on new and unproven technology in the development of products, protection of our intellectual property, and economic conditions generally. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in the company's periodic reports, including the Quarterly Report on Form 10-Q for the three months ended September, 2013. Forward-looking statements are based on the beliefs, opinions, and expectations of the company's management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. Forward-looking statements are based on the beliefs, opinions, and expectations of the company's management at the time they are made, and the company does not assume any obligation to update its forward-looking statements if those beliefs, opinions, expectations, or other circumstances should change. There can be no assurance that the Company's clinical trials will be successful.
SOURCE: Advanced Cell Technology, Inc.
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