Stem Cell Clinic

US Stem Cell Inc (OTCMKTS:USRM) Explodes Higher Ahead of RMAT Decision – The Oracle Dispatch

US Stem Cell Inc (OTCMKTS:USRM) is a penny player in the stem cell space that caught a massive jolt of bullish energy to start off this week. We didnt see any clear new catalyst emerge, but the move likely hooks into the companys near-term narrative, which is highly entangled with a decision of whether or not to approve the companys application to the FDA for Regenerative Medicine Advanced Therapy (RMAT) Designation for the MyoCell product as part of its MARVEL trial.

According to a press release last month, the trial had previously been placed on Inactive Status as patients were not actively being enrolled. A request was placed to the FDA to reactivate the protocol and consider the therapy for RMAT designation. We have recently heard from the FDA who has notified us that the protocol has been placed on Reactivation Status after reviewing details on the protocol and data collected on patients to date. The FDA has also notified us that they are still reviewing our submission for RMAT.

US Stem Cell Inc (OTCMKTS:USRM) bills itself as a company committed to the development of effective cell technologies to treat a variety of diseases and injuries. By harnessing the bodys own healing potential, we may be able to reverse damaged tissue to normal function.

U.S. Stem Cells discoveries include multiple cell therapies in various stages of development that repair damaged tissues throughout the body due to injury or disease so that patients may return to a normal lifestyle.

USRM is focused on regenerative medicine. While most stem cell companies use one particular cell type to treat a variety of diseases, U.S Stem Cell utilizes various cell types to treat different diseases. It is our belief that the unique qualities within the various cell types make them more advantageous to treat a particular disease.

According to company materials, US Stem Cell, Inc. (formerly Bioheart, Inc.) is an emerging enterprise in the regenerative medicine / cellular therapy industry. We are focused on the discovery, development and commercialization of cell based therapeutics that prevent, treat or cure disease by repairing and replacing damaged or aged tissue, cells and organs and restoring their normal function. We believe that regenerative medicine / cellular therapeutics will play a large role in positively changing the natural history of diseases ultimately, we contend, lessening patient burdens as well as reducing the associated economic impact disease imposes upon modern society.

Subscribe below and well keep you on top of whats happening before $USRM stock makes its next move.

As noted above, the stock shifted into bull mode to start this week. However, traders are scrambling to uncover the catalyst for the move.

We would say the most likely catalyst has to do with expectations surrounding its application for RMAT status with the FDA. That was apparently expected to hit as a decision sometime in May. Today marks the outset of the new month, and we may have either rumors or speculation fueling the idea that one must not wait any longer if one wants to catch the action into that catalyst the idea being, approval would seriously juice this stock higher.

Recent action has seen 33% piled on for shareholders of the company during the trailing week. This is emblematic of the stock. USRM has evidenced sudden upward volatility on many prior occasions. Whats more, the listing has benefitted from a jump in recent trading volume to the tune of 16% over what the stock has registered over the longer term.

According to the company, Thanks to the REGROW component of the Cures Act, the FDA will grant RAT designation for a regenerative medicine therapy that is intended to treat, modify, reverse, or cure a serious or life-threatening disease and demonstrates preliminary clinical evidence that the product has the potential to address unmet medical needs for a disease. We believe that our MyoCell product meets these requirements, as we have demonstrated clinical efficacy in both preclinical and clinical studies, including our most recent MARVEL trial publication.

Earning a current market cap value of $44.2M, USRM has a reported chunk ($271K) of cash on the books, which compares with about $3M in total current liabilities. USRM is pulling in trailing 12-month revenues of $3.1M. In addition, the company is seeing major top line growth, with y/y quarterly revenues growing at 66.6%. We will update the story again soon as developments transpire. For continuing coverage on shares of $USRM stock, as well as our other hot stock picks, sign up for our free newsletter today and get our next hot stock pick!

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US Stem Cell Inc (OTCMKTS:USRM) Explodes Higher Ahead of RMAT Decision - The Oracle Dispatch

Presidential Symposium at the American Society of Gene and Cell … – Yahoo Finance

FREMONT, Calif., May 3, 2017 /PRNewswire/ --Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company pioneering the field of regenerative medicine, today announced that data from its AST-OPC1 clinical program for severe cervical spinal cord injury will be presented during the Presidential Symposium at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting, being held in Washington, D.C. during May 10-13, 2017.

"The ASGCT decision to include a presentation on AST-OPC1 in its Presidential Symposium signifies the ground-breaking nature of our program, and reflects the encouraging efficacy and safety data we have seen to date in patients with severe spinal cord injuries that have been treated with AST-OPC1," said Steve Cartt, President and Chief Executive Officer of Asterias. "Data will be presented from our SCiStar study demonstrating the potential of AST-OPC1 to help patients with complete paralysis regain increased arm, hand and finger function, and thus greater ability to live independently."

Jane S. Lebkowski, Ph.D., Asterias' President of R&D and Chief Scientific Officer, will be one of the presenters during the Presidential Symposium session scheduled on Friday, May 12, 2017 at 1:00pm Eastern Time. Dr. Lebkowski's presentation, titled "498 - Safety and Efficacy of Human Embryonic Stem Cell Derived Oligodendrocyte Progenitor Cells (AST-OPC1) in Patients with Subacute Cervical Spinal Cord Injury," is expected to begin at 2:15pm Eastern Time. The abstract for Dr. Lebkowski's presentation at the ASGCT meeting is available online at: http://www.abstractsonline.com/pp8/#!/4399/presentation/1996.

ASGCT is the primary professional membership organization for gene and cell therapy. The Society's members are scientists, physicians, patient advocates, and other professionals. Its members work in a wide range of settings including universities, hospitals, government agencies, foundations, biotechnology and pharmaceutical companies. Its mission is to advance knowledge, awareness, and education leading to the discovery and clinical application of gene and cell therapies to alleviate human disease.

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may retain some minimal sensory function below their injury site. AST-OPC1 is being administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

The study is being conducted at six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.

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In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries, which represents the first targeted population for registration trials.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/presidential-symposium-at-the-american-society-of-gene-and-cell-therapy-asgct-20th-annual-meeting-will-feature-presentation-on-asterias-ast-opc1-for-spinal-cord-injury-300450272.html

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Presidential Symposium at the American Society of Gene and Cell ... - Yahoo Finance

Cell Therapy Company Joins Forces with Biotech in New Transplantation Research Alliance – Pharmaceutical Processing

Be The Match BioTherapies announces collaboration agreement with Magenta Therapeutics.

MINNEAPOLIS, May 2, 2017 Be The Match BioTherapiesSM, an organization offering solutions for delivering autologous and allogeneic cellular therapies, today announced that it has entered into a strategic partnership with Magenta Therapeutics, a biotechnology company developing therapies to improve and expand the use of curative stem cell transplantation. The collaboration is intended to support efforts to improve transplant outcomes and expand the application of stem cell transplantation into disease indications that include autoimmunity, serious inherited immune and metabolic disorders, blood defects and blood cancers. Be The Match BioTherapies also announced today that it is participating in Magentas Series B financing round, its first equity investment as an organization.

Under the terms of the collaboration agreement, Be The Match BioTherapies and Magenta will explore opportunities to work together across Magentas discovery, clinical development and product delivery efforts. The collaboration leverages a wide range of Be The Match BioTherapies research assets and services, including the National Marrow Donor Program(NMDP)/Be The Match marrow registry, the largest in the world with nearly 16 million volunteer marrow donors. Magenta may also collaborate with Be The Match BioTherapies in the design of clinical trials and of its cell therapy delivery platform and services.

Partnering with Magenta in its efforts to revolutionize the current state of stem cell transplantation aligns with our core mission to help organizations deliver cellular therapies that save more lives and improve the quality of life for patients, said Amy Ronneberg, President of Be The Match BioTherapies. Our collaboration with Magenta exemplifies how cell and gene therapy companies can benefit from our robust network of products and services regardless of where they are in the development life cycle. We look forward to lending our expertise in cellular therapy and leveraging our deep-rooted relationships, partnerships and global infrastructure to support the development of powerful treatment options with great potential to improve patient outcomes in a range of disease areas.

Jason Gardner, D. Phil., Chief Executive Officer, President, and Cofounder of Magenta, added: We believe that Be The Match BioTherapies extensive experience and network in stem cell transplant medicine, coupled with Magentas work in patient conditioning and stem cell harvesting and growth, could accelerate our development path and ability to positively impact patients lives.

Be The Match BioTherapies launched in 2016 as a subsidiary of NMDP/Be The Match, the national organization with a 30-year history of connecting patients with their donor match for a life-saving bone marrow or umbilical cord blood transplant. As experts in providing services and expertise to organizations pursuing life-saving treatments in the cellular therapy space, Be The Match BioTherapies aims to help critically ill patients who can benefit from these treatments.

(Source:Business Wire)

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Cell Therapy Company Joins Forces with Biotech in New Transplantation Research Alliance - Pharmaceutical Processing

Stem Cells in Culture Have Tendency to Develop Cancer-Linked Mutations – Genetic Engineering & Biotechnology News

Stem cells that are grown in the lab are known to acquire mutations, but whether these mutations are particularly numerous or risky remains unclear. Mutations acquired in stem cell culture, it is feared, would complicate efforts to deploy stem cells in regenerative medicine. At the least, lab-grown stem cells may need to be screened for deleterious mutations, with special attention devoted to vulnerable portions of the genome or flaws that could lead to dire consequences, such as cancer.

To characterize the mutations that may arise among stem cells in vitro, scientists have been introducing gene-sequencing tests. For example, in a recent study, scientists based at Harvard have determined that human pluripotent stem cells are prone to develop mutations in the TP53 gene, which ordinarily helps suppress cancer. The mutated versions of the TP53 found by the Harvard team, however, tend to drive cancer development.

Details of this work appeared April 26 in the journal Nature, in an article entitled, Human Pluripotent Stem Cells Recurrently Acquire and Expand Dominant Negative P53 Mutations. This article describes how the Harvard team sequenced the protein-coding genes of 140 human embryonic stem cell (hES) cell lines26 of which were developed for therapeutic purposes using Good Manufacturing Practices, a quality control standard set by regulatory agencies in multiple countries. The remaining 114 human pluripotent stem cell lines were listed on the NIH registry of human pluripotent stem cells. This gene-sequencing exercise was followed by computational work that allowed the scientists to identify mutations present in a subset of cells in each cell line.

[We] identified five unrelated hES cell lines that carried six mutations in the TP53 gene that encodes the tumour suppressor P53, wrote the articles authors. The TP53 mutations we observed are dominant negative and are the mutations most commonly seen in human cancers. We found that the TP53 mutant allelic fraction increased with passage number under standard culture conditions, suggesting that the P53 mutations confer selective advantage.

The scientists also mined published RNA sequencing data from 117 human pluripotent stem cell lines, and observed another nine TP53 mutations, all resulting in coding changes in the DNA-binding domain of P53. In three lines, the authors of the Nature paper detailed, the allelic fraction exceeded 50%, suggesting additional selective advantage resulting from the loss of heterozygosity at the TP53 locus.

These findings suggest that cell lines should be screened for mutations at various stages of development as well as immediately before transplantation.

"Our results underscore the need for the field of regenerative medicine to proceed with care," said the study's co-corresponding author Kevin Eggan, Ph.D. "[They] indicate that an additional series of quality control checks should be implemented during the production of stem cells and their downstream use in developing therapies. Fortunately, these genetic checks can be readily performed with precise, sensitive, and increasingly inexpensive sequencing methods."

"Cells in the lab, like cells in the body, acquire mutations all the time," added Steve McCarroll, Ph.D., co-corresponding author. "Mutations in most genes have little impact on the larger tissue or cell line. But cells with a pro-growth mutation can outcompete other cells, become very numerous, and 'take over' a tissue. We found that this process of clonal selectionthe basis of cancer formation in the bodyis also routinely happening in laboratories."

Although the Harvard scientists expected to find some mutations in stem cell lines, they were surprised to find that about 5% of the stem cell lines they analyzed had acquired mutations the TP53 gene, which encodes the tumor suppressor protein P53.

Nicknamed the "guardian of the genome," P53 controls cell growth and cell death. People who inherit p53 mutations develop a rare disorder called Li-Fraumeni Syndrome, which confers a near 100% risk of developing cancer in a wide range of tissue types.

The specific mutations that the researchers observed are "dominant negative" mutations, meaning, when present on even one copy of P53, they are able to compromise the function of the normal protein, whose components are made from both gene copies. The exact same dominant negative mutations are among the most commonly observed mutations in human cancers.

The researchers performed a sophisticated set of DNA analyses to rule out the possibility that these mutations had been inherited rather than acquired as the cells grew in the lab. In subsequent experiments, the Harvard scientists found that P53 mutant cells outperformed and outcompeted nonmutant cells in the lab dish. In other words, a culture with a million healthy cells and one P53 mutant cell, said Dr. Eggan, could quickly become a culture of only mutant cells.

"The spectrum of tissues at risk for transformation when harboring a P53 mutation include many of those that we would like to target for repair with regenerative medicine using human pluripotent stem cells," noted Dr. Eggan. Those organs include the pancreas, brain, blood, bone, skin, liver, and lungs.

However, Drs. Eggan and McCarroll emphasized that now that this phenomenon has been found, inexpensive gene-sequencing tests will allow researchers to identify and remove from the production line cell cultures with concerning mutations that might prove dangerous after transplantation.

The researchers point out in their paper that screening approaches to identify these P53 mutations and others that confer cancer risk already exist and are used in cancer diagnostics. In fact, in an ongoing clinical trial that is transplanting cells derived from induced pluripotent stem cells, or iPSCs, gene sequencing is used to ensure the transplanted cell products are free of dangerous mutations.

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Stem Cells in Culture Have Tendency to Develop Cancer-Linked Mutations - Genetic Engineering & Biotechnology News

Stem cell therapy relieves pain, restores joints – The Herald-News

The 360+ joints in the human body link bones and keep our bodies flexible. Until they become painful. Physical discomfort, where bones meet to form a joint, can be mild to intensely agonizing when the joints cartilage, ligaments, tendons, or muscles become inflamed and sore. Pain in one joint may be the result of an injury, or a condition such as tendonitis. Pain in multiple joints may indicate arthritis or gout.

Many joint pain problems in the knees, hips, and shoulders can be relieved with stem cell injection therapy, according to Dr. Frank Ostir, Director, Ostir Physical Medicine in Joliet. He explains that stem cells have anti-inflammatory properties plus growth factors. They relieve pain and rebuild damaged joints by regenerating into new tissue and cartilage. Stem cells can also heal torn ligaments.

They speed up the bodys own healing abilities. Theres no risk of tissue rejection, and no toxic substances or side effects. This regenerative treatment gives the best results in the shortest amount of time, Ostir says.

This phenomenal advancement in regenerative medicine makes it possible for patients to be pain-free and possibly avoid surgery through a minimally invasive procedure performed in the office. The actual procedure takes about 15 minutes. We used guided imaging from fluoroscopy and ultrasound technology to inject the stem cells to the exact site of affliction. The stem cells are mixed with a local anesthetic and injected through a small needle, to minimize any discomfort.

Ostir continues, The patient usually rests on the day of the injection, and resumes regular activity afterwards. About 80 percent of healing occurs in the first two months of treatment, due to the rate of stem cells replicating. Its our hope that this truly amazing therapy will eliminate the need for drugs and surgery.

For more information, contact Ostir Physical Medicine, (815) 729-2022, or visit http://www.ostirphysicalmed.com.

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Stem cell therapy relieves pain, restores joints - The Herald-News