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Category Archives: Pennsylvania Stem Cells

Head to Head Survey: BioRestorative Therapies (OTCMKTS:BRTX) & Livongo Health (OTCMKTS:LVGO) – Mitchell Messenger

Posted: December 1, 2019 at 8:48 pm

Livongo Health (NASDAQ:LVGO) and BioRestorative Therapies (OTCMKTS:BRTX) are both medical companies, but which is the superior business? We will contrast the two companies based on the strength of their valuation, earnings, profitability, analyst recommendations, risk, institutional ownership and dividends.

Insider and Institutional Ownership

18.4% of Livongo Health shares are owned by institutional investors. 17.9% of BioRestorative Therapies shares are owned by company insiders. Strong institutional ownership is an indication that large money managers, hedge funds and endowments believe a stock will outperform the market over the long term.

This table compares Livongo Health and BioRestorative Therapies net margins, return on equity and return on assets.

Valuation & Earnings

This table compares Livongo Health and BioRestorative Therapies top-line revenue, earnings per share (EPS) and valuation.

BioRestorative Therapies has lower revenue, but higher earnings than Livongo Health.

Analyst Recommendations

This is a breakdown of recent ratings and price targets for Livongo Health and BioRestorative Therapies, as provided by MarketBeat.com.

Livongo Health currently has a consensus price target of $44.30, suggesting a potential upside of 55.11%. Given Livongo Healths higher probable upside, equities analysts plainly believe Livongo Health is more favorable than BioRestorative Therapies.

Summary

Livongo Health beats BioRestorative Therapies on 7 of the 10 factors compared between the two stocks.

Livongo Health Company Profile

Livongo Health, Inc. provides an integrated suite of solutions for the healthcare industry in North America. It solutions promote health behavior change based on real-time data capture supported by intuitive devices and insights driven by data science. The company offers a platform that provides cellular-connected devices, supplies, informed coaching, data science-enabled insights, and facilitates access to medications. Its products include Livongo for Diabetes, Livongo for Hypertension, Livongo for Prediabetes and Weight Management, and Livongo for Behavioral Health by myStrength. The company was formerly known as EosHealth, Inc. and changed its name to Livongo Health, Inc. in 2014. Livongo Health, Inc. was incorporated in 2008 and is headquartered in Mountain View, California.

BioRestorative Therapies Company Profile

BioRestorative Therapies, Inc. develops therapeutic products and medical therapies using cell and tissue protocols, primarily involving adult stem cells for the treatment of disc/spine disease and metabolic disorders. The company's lead cell therapy candidate is the BRTX-100, which focuses on providing non-surgical treatment for protruding and bulging lumbar discs in patients suffering from chronic lumbar disc disease. It also develops the ThermoStem program, a pre-clinical program for the treatment of metabolic diseases, such as type 2 diabetes, obesity, hypertension, and other metabolic disorders, as well as cardiac deficiencies. In addition, the company provides curved needle device, a needle system with a curved inner cannula that allows access to difficult-to-locate regions for the delivery or removal of fluids and other substances. Further, it offers skin care products under the Stem Pearls brand name. BioRestorative Therapies, Inc. has a research and development agreement with Rohto Pharmaceutical Co., Ltd.; and a research agreement with Pfizer, Inc. and the University of Pennsylvania. The company was formerly known as Stem Cell Assurance, Inc. and changed its name to BioRestorative Therapies, Inc. in August 2011. BioRestorative Therapies, Inc. was incorporated in 1997 and is headquartered in Melville, New York.

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Head to Head Survey: BioRestorative Therapies (OTCMKTS:BRTX) & Livongo Health (OTCMKTS:LVGO) - Mitchell Messenger

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Critical Review: BioRestorative Therapies (OTCMKTS:BRTX) and Livongo Health (OTCMKTS:LVGO) – NCTY News

Posted: November 16, 2019 at 4:46 am

BioRestorative Therapies (OTCMKTS:BRTX) and Livongo Health (NASDAQ:LVGO) are both medical companies, but which is the better investment? We will compare the two companies based on the strength of their profitability, institutional ownership, analyst recommendations, dividends, risk, earnings and valuation.

Valuation & Earnings

This table compares BioRestorative Therapies and Livongo Healths top-line revenue, earnings per share (EPS) and valuation.

BioRestorative Therapies has higher earnings, but lower revenue than Livongo Health.

Insider & Institutional Ownership

0.3% of Livongo Health shares are held by institutional investors. 17.9% of BioRestorative Therapies shares are held by insiders. Strong institutional ownership is an indication that large money managers, hedge funds and endowments believe a stock will outperform the market over the long term.

Profitability

This table compares BioRestorative Therapies and Livongo Healths net margins, return on equity and return on assets.

Analyst Ratings

This is a summary of recent recommendations and price targets for BioRestorative Therapies and Livongo Health, as reported by MarketBeat.com.

Livongo Health has a consensus price target of $44.30, suggesting a potential upside of 73.39%. Given Livongo Healths higher possible upside, analysts plainly believe Livongo Health is more favorable than BioRestorative Therapies.

Summary

Livongo Health beats BioRestorative Therapies on 7 of the 10 factors compared between the two stocks.

BioRestorative Therapies Company Profile

BioRestorative Therapies, Inc. develops therapeutic products and medical therapies using cell and tissue protocols, primarily involving adult stem cells for the treatment of disc/spine disease and metabolic disorders. The company's lead cell therapy candidate is the BRTX-100, which focuses on providing non-surgical treatment for protruding and bulging lumbar discs in patients suffering from chronic lumbar disc disease. It also develops the ThermoStem program, a pre-clinical program for the treatment of metabolic diseases, such as type 2 diabetes, obesity, hypertension, and other metabolic disorders, as well as cardiac deficiencies. In addition, the company provides curved needle device, a needle system with a curved inner cannula that allows access to difficult-to-locate regions for the delivery or removal of fluids and other substances. Further, it offers skin care products under the Stem Pearls brand name. BioRestorative Therapies, Inc. has a research and development agreement with Rohto Pharmaceutical Co., Ltd.; and a research agreement with Pfizer, Inc. and the University of Pennsylvania. The company was formerly known as Stem Cell Assurance, Inc. and changed its name to BioRestorative Therapies, Inc. in August 2011. BioRestorative Therapies, Inc. was incorporated in 1997 and is headquartered in Melville, New York.

Livongo Health Company Profile

Livongo Health, Inc. provides an integrated suite of solutions for the healthcare industry in North America. It solutions promote health behavior change based on real-time data capture supported by intuitive devices and insights driven by data science. The company offers a platform that provides cellular-connected devices, supplies, informed coaching, data science-enabled insights, and facilitates access to medications. Its products include Livongo for Diabetes, Livongo for Hypertension, Livongo for Prediabetes and Weight Management, and Livongo for Behavioral Health by myStrength. The company was formerly known as EosHealth, Inc. and changed its name to Livongo Health, Inc. in 2014. Livongo Health, Inc. was incorporated in 2008 and is headquartered in Mountain View, California.

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Critical Review: BioRestorative Therapies (OTCMKTS:BRTX) and Livongo Health (OTCMKTS:LVGO) - NCTY News

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Reviewing BioRestorative Therapies (OTCMKTS:BRTX) and Livongo Health (OTCMKTS:LVGO) – Mitchell Messenger

Posted: November 16, 2019 at 4:46 am

BioRestorative Therapies (OTCMKTS:BRTX) and Livongo Health (NASDAQ:LVGO) are both medical companies, but which is the superior business? We will compare the two businesses based on the strength of their profitability, institutional ownership, earnings, valuation, dividends, analyst recommendations and risk.

Profitability

This table compares BioRestorative Therapies and Livongo Healths net margins, return on equity and return on assets.

This is a breakdown of recent recommendations and price targets for BioRestorative Therapies and Livongo Health, as provided by MarketBeat.

Livongo Health has a consensus price target of $44.30, suggesting a potential upside of 74.68%. Given Livongo Healths higher possible upside, analysts plainly believe Livongo Health is more favorable than BioRestorative Therapies.

Institutional and Insider Ownership

0.3% of Livongo Health shares are owned by institutional investors. 17.9% of BioRestorative Therapies shares are owned by company insiders. Strong institutional ownership is an indication that large money managers, endowments and hedge funds believe a stock will outperform the market over the long term.

Earnings and Valuation

This table compares BioRestorative Therapies and Livongo Healths revenue, earnings per share and valuation.

BioRestorative Therapies has higher earnings, but lower revenue than Livongo Health.

Summary

Livongo Health beats BioRestorative Therapies on 7 of the 10 factors compared between the two stocks.

About BioRestorative Therapies

BioRestorative Therapies, Inc. develops therapeutic products and medical therapies using cell and tissue protocols, primarily involving adult stem cells for the treatment of disc/spine disease and metabolic disorders. The company's lead cell therapy candidate is the BRTX-100, which focuses on providing non-surgical treatment for protruding and bulging lumbar discs in patients suffering from chronic lumbar disc disease. It also develops the ThermoStem program, a pre-clinical program for the treatment of metabolic diseases, such as type 2 diabetes, obesity, hypertension, and other metabolic disorders, as well as cardiac deficiencies. In addition, the company provides curved needle device, a needle system with a curved inner cannula that allows access to difficult-to-locate regions for the delivery or removal of fluids and other substances. Further, it offers skin care products under the Stem Pearls brand name. BioRestorative Therapies, Inc. has a research and development agreement with Rohto Pharmaceutical Co., Ltd.; and a research agreement with Pfizer, Inc. and the University of Pennsylvania. The company was formerly known as Stem Cell Assurance, Inc. and changed its name to BioRestorative Therapies, Inc. in August 2011. BioRestorative Therapies, Inc. was incorporated in 1997 and is headquartered in Melville, New York.

About Livongo Health

Livongo Health, Inc. provides an integrated suite of solutions for the healthcare industry in North America. It solutions promote health behavior change based on real-time data capture supported by intuitive devices and insights driven by data science. The company offers a platform that provides cellular-connected devices, supplies, informed coaching, data science-enabled insights, and facilitates access to medications. Its products include Livongo for Diabetes, Livongo for Hypertension, Livongo for Prediabetes and Weight Management, and Livongo for Behavioral Health by myStrength. The company was formerly known as EosHealth, Inc. and changed its name to Livongo Health, Inc. in 2014. Livongo Health, Inc. was incorporated in 2008 and is headquartered in Mountain View, California.

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Reviewing BioRestorative Therapies (OTCMKTS:BRTX) and Livongo Health (OTCMKTS:LVGO) - Mitchell Messenger

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These Israeli Companies Are Finding Innovative Ways To Improve Diabetes Care, Treatment | Health News – NoCamels – Israeli Innovation News

Posted: November 16, 2019 at 4:46 am

Diabetes is a major public health crisis that is approaching epidemic proportions around the globe and growing at an alarming rate. According to the International Diabetes Federation (IDF), over 425 million adults are currently living with diabetes. That number is expected to rise to 629 million by 2045.

The disorder, which occurs when the pancreas does not produce enough insulin (the hormone that regulates blood sugar) or when the body cannot use its produced insulin, has become so deadly, that the International Diabetes Federation said it was responsible for four million deaths in 2017 alone. The World Health Organization estimated diabetes to be the seventh leading cause of death globally in 2016.

Over 350 million people are at risk of developing Type 2 diabetes, when the body becomes to resistant to insulin due to lifestyle facts such as excess weight gain, while more than 1.1 million children are living with Type 1 diabetes, the disorder that occurs when the bodys immune system attacks cells of the pancreas that produce insulin, according to the International Diabetes Federation.

World Diabetes Day, marked annually on November 14 to honor the co-discoverer of insulin Canadian Dr. Frederick Banting, born on that day in 1891, aims to raise awareness of the impact of this disease while promoting its management, care, and prevention. For the past two years (including in 2019,) the theme of World Diabetes Day has been The Family and Diabetes promoting the familys role in awareness and education of the disorder.

To mark the day, NoCamels is taking a closer look at Israels role in advancing diabetes research and treatment as well as some of the companies with cutting-edge technology that stand out in the field.

Dr. Irit Yaniv, a general manager at Accelmed Ventures II, a new $100 million venture early-stage health tech fund for medical device and digital health startups and co-founder and chairperson of Type 2 diabetes medical device startup Digma Medical, calls diabetes the most dangerous global epidemic. Israel is no exception, she says, citing half a million people living with the disease in the country.

Additionally, about 300,000 are prediabetic, a condition manifesting as borderline high blood sugar levels and an increased risk to progress to diabetes Type 2 within a few years, she explains.

Dr. Yaniv says life science entrepreneurs address the challenges presented by the epidemic in a number of ways, including creating companies and projects aimed at reducing the risk of developing the diseases, development of novel drug delivery methods for diabetes drugs, and unique medical devices for managing the disorder.

SEE ALSO: 6 Israeli Companies At The Forefront Of Diabetes Care, Prevention, and Treatment

There are a few interesting examples such as oral insulin and nasal insulin delivery systems that were developed in Israel, Yaniv says. A recent success story is Nutrino, [an Israeli] software company for the management of diabetes that was acquired by Medtronic.

Treatment for Type 2 diabetes has been focused on lifestyle changes and pharmacologic solutions. Both have challenges including patient compliance and adverse effects such as weight gain, hypoglycemia, and other reactions, Yaniv says. More than 50 percent of the patients arent able to control the disease, even with combination therapeutics.

The medical device industry has made many efforts in recent years to address this need, Yaniv says, Companies such as GI Dynamics have paved the way for new therapeutic alternatives. Digma Medical has demonstrated initial positive clinical data with its unique duodenal ablation system.

Yaniv founded Digma Medical in 2013 with Ilan Ben Oren. Backed by leading venture capital firms such as Arkin Holdings and Peregrine Ventures, the company is dedicated to the development of its DiaGone device to treat insulin resistance. DiaGone is an endoscopic, disposable device, that uses innovative laser technology to treat the duodenum, a segment of the small intestine, without an implant. The Duodenal Glycemic Control procedure is a one-time 30 minute GI procedure, in which the gastroenterologist uses DiaGone to treat the duodenum for restoring the natural ability of the body to control glucose levels, said to provide long term remission from Type 2 diabetes and other metabolic syndrome-related diseases.

For Rami Epstein, who assumed the role of CEO at stem cell company Kadimastem in May 2019, a need exists to find a better solution for the management of diabetes in order to decrease the morbidity, mortality, and costs linked to it and its medical-related care, he tells NoCamels. This method is more than just controlling insulin levels in the body. It is controlling the dose of insulin administered to the body.

All Type 1 diabetes and 30 percent of Type 2 diabetes patients depend on the daily administration of insulin in order to control their glucose levels in the blood. This is not ideal since patients have to calculate insulin dosages and take into account meal times and portions, physical activity, and other parameters, he explains. Unfortunately, many patients do not manage to stabilize their blood glucose levels properly, thereby risking complications that arise from episodes of hypoglycemia or hyperglycemia,

Through Kadimastems groundbreaking stem-cell therapy technology, developed by the companys chief scientist Professor Michel Revel at the Weizmann Institute of Science and used as the basis for the companys founding in 2009, the Ness Ziona-based firm has developed and manufactured an off-the-shelf cell product for the treatment of insulin-dependent diabetes based on its proprietary tech platform. The tech platform has been used to treat multiple diseases, including ALS, through the expansion and differentiation of Human Embryonic Stem Cells (hESCs) into clinical-grade functional cells.

The product, called IsletRx, is currently in pre-clinical trials. Its goal is to free patients from continuous monitoring of blood sugar levels and repeated insulin injections. The drug contains an endless source of pancreatic functional islet cells, which produce and secrete insulin and glucagon in response to external glucose levels.

Meanwhile, Tel Aviv-based clinical-stage pharmaceutical company Oramed, which is focused on the development of oral drug delivery systems, announced this week that Phase IIb trial evaluating the efficacy and safety of its lead oral insulin candidate, ORMD-0801, has had positive results.

The study was a 90-day, double-blind, randomized, multi-center trial designed to evaluate the safety and efficacy of ORMD-0801 as a treatment for patients with type 2 diabetes, Oramed said in a statement. The primary efficacy endpoint was a reduction in Hemoglobin A1c (A1C, also known as HbA1c, is a key clinical measure of blood glucose control) at Week 12, with no weight gain.

Israeli scientists are taking diabetes management solutions one step further and adding machine learning and AI to the mix. DreaMed Diabetes, a medical tech startup founded in 2014 to develop these types of personalized solutions, announced in September that it had received clearance from the US Food and Drug Administration (FDA) as well as a CE Mark for its DreaMed Advisor Pro, an AI-based insulin dosing decision support software. The software is for patients with Type 1 diabetes using insulin pump therapy with continuous glucose sensors and blood glucose meters (BGMs)

The decision-support platform uses proprietary algorithms to process data from a range of connected devices, including insulin pumps and self-management glucometers. The data is then analyzed to provide an optimized insulin dosing treatment plan to maintain a balanced glucose level.

SEE ALSO: Israeli AI Startup Can Predict Which Diabetes Patients Will develop Kidney Disease

This clinical and technological advance leverages the power of artificial intelligence to optimize insulin administration in a streamlined and cost-effective manner, the company said in a statement.

This year I am more optimistic that a change will happen in how we manage diabetes. We see more technology adopted in all markets, more sharing of data between patients providers and industry all for the benefit of offering better care for patients, the companys CEO Eran Atlas tells NoCamels in an email. DreaMed is happy to be part of the leaders in this effort, by offering a unique artificial intelligence technology that can analyze data and recommend in only a few seconds how to optimize the technology. With such technology, the proliferation of expert care can be achieved even in emerging markets.

Another key player operating in artificial intelligence in the diabetes management market is Sweetch. Founded in 2013, Sweetch offers an AI-based platform that aims to identify those at high risk of developing Type 2 diabetes. The company calls itself the first AI-powered therapeutics solution to help people with the disorder and comes with a mobile app and a wireless Bluetooth-connected scale. The early prevention platform announced last year that it will partner with US-based integrated healthcare system WellSpan Health and provide its app to 15,000 employees, including 200 primary care and specialty physicians, as well as advanced practice clinicians in central Pennsylvania and northern Maryland.

In 2016, the startup raised $3.5 million in a Series A round led by equity crowdfunding platform OurCrowd and Philips.

Diabetes can cause circulation problems and related conditions such as peripheral arterial disease (PAD) which occurs when plaque builds up in the arteries and reduces blood flow to the limbs. For some patients, high levels of blood glucose can damage blood vessels and cause plaque build-up affecting healthy blood flow.

The northern Israel-based startupElastiMedhas developed a wearable medical device that doesnt treat diabetes patients directly but can help the patient improve his or her circulation.

It cant treat the disease directly, but it can treat some of its symptoms, says Elastimed CEO and founder Omer Zelka.

Compression socks improve circulation by squeezing the foot and calf muscles, which straightens out the vein walls to a better working state, says Advanced Tissue, the leading wound care supply provider. Compression therapy is particularly beneficial for diabetes patients because they improve circulation in a non-invasive manner, helping to maintain the right amount of pressure in feet and legs.

ElastiMeds sock uses battery-operated technology to activate a smart material that compresses and massage the legs to stimulate circulation. The pulses mimic contractions in the calf muscles that in turn increase blood flow.

The sock provides patients with a comfortable, easy-to-wear, highly effective, and cost-effective treatment option to prevent symptoms such as swelling, blood clots, leg ulcers and reduce athletes recovery time.

ElastiMed is currently finishing an ongoing clinical study to demonstrate the safety and the feasibility of this device and its ability to increase venous blood flow. The study is led by Dr. Vered Shuster Ben-Yosef, R&D Lab Manager of the company and is currently taking place at Hillel Yaffe Medical Center in Hadera. The company currently has a working prototype and aims to get the product on the market in early 2021.

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Reviewing Madrigal Pharmaceuticals Inc. (MDGL)’s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)’s results – MS Wkly

Posted: October 30, 2019 at 1:46 pm

We will be contrasting the differences between Madrigal Pharmaceuticals Inc. (NASDAQ:MDGL) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) as far as profitability, analyst recommendations, risk, institutional ownership, dividends, earnings and valuation are concerned. The two businesses are rivals in the Biotechnology industry.

Valuation & Earnings

Table 1 shows gross revenue, earnings per share (EPS) and valuation of the two companies.

Profitability

Table 2 shows us the return on assets, return on equity and net margins of both companies.

Volatility & Risk

Madrigal Pharmaceuticals Inc.s current beta is 1.16 and it happens to be 16.00% more volatile than Standard & Poors 500. Brainstorm Cell Therapeutics Inc.s 1.19 beta is the reason why it is 19.00% more volatile than Standard & Poors 500.

Liquidity

Madrigal Pharmaceuticals Inc. has a Current Ratio of 45.8 and a Quick Ratio of 45.8. Competitively, Brainstorm Cell Therapeutics Inc.s Current Ratio is 1 and has 1 Quick Ratio. Madrigal Pharmaceuticals Inc.s better ability to pay short and long-term obligations than Brainstorm Cell Therapeutics Inc.

Analyst Recommendations

The table shown features the ratings and recommendations for Madrigal Pharmaceuticals Inc. and Brainstorm Cell Therapeutics Inc.

$150.5 is Madrigal Pharmaceuticals Inc.s average target price while its potential upside is 57.59%. Meanwhile, Brainstorm Cell Therapeutics Inc.s average target price is $9, while its potential upside is 147.25%. Based on the results shown earlier, Brainstorm Cell Therapeutics Inc. is looking more favorable than Madrigal Pharmaceuticals Inc., analysts view.

Insider & Institutional Ownership

Institutional investors held 98.9% of Madrigal Pharmaceuticals Inc. shares and 11.4% of Brainstorm Cell Therapeutics Inc. shares. Insiders held roughly 11.33% of Madrigal Pharmaceuticals Inc.s shares. Comparatively, Brainstorm Cell Therapeutics Inc. has 0.6% of its share held by insiders.

Performance

Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year Madrigal Pharmaceuticals Inc. has -22.56% weaker performance while Brainstorm Cell Therapeutics Inc. has 12.96% stronger performance.

Summary

Brainstorm Cell Therapeutics Inc. beats Madrigal Pharmaceuticals Inc. on 6 of the 10 factors.

Madrigal Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on the development and commercialization of therapeutic candidates for the treatment of cardiovascular, metabolic, and liver diseases. The companys lead candidate is MGL-3196, which is in Phase II clinical trials, a liver-directed thyroid hormone receptor- (THR-) agonist used for the treatment of nonalcoholic fatty liver disease, nonalcoholic steatohepatitis, and familial hypercholesterolemia. It is also developing MGL-3745, which is in pre-clinical stage, a THR- agonist that is in preclinical trials. The company was founded in 2011 and is headquartered in West Conshohocken, Pennsylvania.

Brainstorm Cell Therapeutics Inc., a biotechnology company, develops adult stem cell therapies for neurodegenerative disorders that include amyotrophic lateral sclerosis, multiple sclerosis, Parkinsons disease, and others. The company holds rights to develop and commercialize its NurOwn technology through a licensing agreement with Ramot of Tel Aviv University Ltd. Its NurOwn technology is based on a novel differentiation protocol, which induces differentiation of the bone marrow-derived mesenchymal stem cells into neuron-supporting cells and secreting cells that release various neurotrophic factors, including glial-derived neurotrophic factor, brain-derived neurotrophic factor, vascular endothelial growth factor, and hepatocyte growth factor for the growth, survival, and differentiation of developing neurons. The company was formerly known as Golden Hand Resources Inc. and changed its name to Brainstorm Cell Therapeutics Inc. in November 2004 to reflect its new line of business in the development of novel cell therapies for neurodegenerative diseases. Brainstorm Cell Therapeutics Inc. was founded in 2000 and is headquartered in Hackensack, New Jersey.

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Reviewing Madrigal Pharmaceuticals Inc. (MDGL)'s and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI)'s results - MS Wkly

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Gene Therapy/Editing Series 1: A Brief Introduction To Gene Therapy – Seeking Alpha

Posted: October 30, 2019 at 1:46 pm

The recent approval of various gene therapies, for example, Luxturna and Zolgesma and high premium acquisitions of gene therapy companies have shifted the investor focus to this rapidly growing biotechnology field. In this series of review articles, I will review the gene therapy and gene editing field, starting first with the basics, including a brief overview of the history of the field and then moving on to some technical aspects, for example, the manufacturing, different methods of delivery, and then moving on to discussing the competitive landscape covering one genetic disease in each article.

Let's first define what is a gene? A gene is a sequence of nucleotides in DNA that encodes the synthesis of a gene product, which is usually a protein.

(F8 gene, mutations in the gene cause Hemophilia A)

Usually, the code (in the form of a specific arrangement of nucleotides or base pairs contained in the gene is used to form mRNA (called transcription) which acts as a messenger to take the code to the target organ of the body. The information stored in the mRNA is then used to encode and synthesize the target protein (called translation) which then performs its intended function in the body.

(Steps involved in synthesizing a protein from the code in a gene)

An estimated number of protein-coding genes in the human body is approximately 20,000 to 25,000, which has been revised down from the initial prediction of 100,000 genes. Each gene contains a number of base pairs, the number of which is estimated to range from about 50 million to 300 million in the human body. In general, a gene therapy can be broadly defined as delivering in a new gene into the cells to compensate for a defective gene. In gene therapy, a newly delivered gene can perform different functions; for example, it can either replace the defective gene or it can silence an abnormal gene.

(An example of a gene therapy using an adenoviral vector to deliver the normal gene)

While I will discuss the various steps and delivery systems in gene therapy in detail later, viruses like lentiviruses and adenovirus are most commonly used as vectors in gene therapy. It was as early as 1950s that scientists first discovered that a virus can be used to inject the DNA in the cells of the host. In 1970s, various experiments started to use viruses as delivery systems for genes in the human body. In 1971, Merril, et al conducted a scientific experiment showing that DNA could be injected into the human cells to fix a biological problem in the cells. This group of scientists extracted the cells from patients suffering from a disease called Galactosemia. It is important to note that this first gene therapy experiment involves the manipulation of genes ex vivo, that is in cells growing in a petri dish outside the body in a lab, which is easier to perform than manipulating the genes inside the human body, called in vivo approach. In 1972, a famous article in the prestigious journal Science by authors Friedman and Roblin first proposed that the gene therapy may ameliorate some human genetic diseases in the future. During the 1980s, various scientists like Martin Cline and French Anderson conducted experiments on using viruses as delivery vehicles for DNA in human or mouse cells. The first human trials of gene therapy started in the late 1980s and the results were reported in early 1990s. One of the first reported clinical studies in humans involved ex vivo modification of white blood cells taken from patients with advanced melanoma, using a retroviral vector to insert a gene coding for interleurkin-2 and injecting the genetically altered cells back into the patients. During the 1990s, French Anderson reported a successful clinical trial where a retroviral vector was used to transfer a gene encoding for adenosine deaminase, ADA in children with severe combined immunodeficiency, SCID. During the 1990s, most of the work in gene therapy continued in the therapeutic area of ADA-SCID.

Despite reasonably successful clinical results, the field of gene therapy suffered a serious setback in 1999. Jesse Gelsinger, an 18-year-old patient with a disease called ornithine transcarbamylase, OTC deficiency, which results due to a missing gene coding OTC died 4 days after receiving the gene therapy in a clinical trial conducted by the University of Pennsylvania due to massive immune response resulting in multi-organ failure. As a result, FDA put a suspension on various gene therapy clinical trials.

The field of gene therapy was then suspended for almost a decade. Glybera, a gene therapy was approved in Europe for reading a genetic disease, lipoprotein lipase deficiency in 2012. However, Glybera was a commercial failure after insurers in Europe were reluctant to pay for its expensive $1 million per patient tag. Finally, uniQure (QURE) the company that developed Glybera discontinued it.

Another commercial gene therapy failure was Strimvelis, a stem cell gene therapy to treat ADA-SCID. Despite its price being lower than Glybera ($665,000 per year), the therapy was not commercially successful in Europe and was sold by GlaxoSmithKline (GSK) to Orchard Therapeutics (ORTX) in 2018. In the US, the first approved gene therapy was Kymriah, an autologous CAR-T therapy to treat autologous lymphoblastic leukemia (ALL), which was developed by Novartis (NYSE:NVS).

After Kymriah, another autologous CAR-T therapy, Yeskarta (by Kite Pharmaceuticals) was approved by FDA to treat adult diffuse large B-cell lymphoma. Kite was later acquired by Gilead (NASDAQ:GILD). The first in vivo gene therapy approval in the US was Luxturna, an AAV gene therapy for patients with RPE 65 mutation-associated retinal dystrophy, which was developed by Spark Therapeutics which also was later acquired. Luxturna was another major milestone in the history of gene therapy as it resulted in a miraculous effect of restoring vision to children who were blind since birth. Recently, bluebird bio's (BLUE) gene therapy for transfusion-dependent beta-thalassemia was approved in Europe.

The developmental landscape of gene therapies can be summarized in some excellent figures from the journal Molecular Therapy published by the American Society of Gene and Cell Therapy (ASGCT). A group of researchers reviewed the medical literature and identified 336 gene therapies being developed for 138 different clinical indications covering 165 genetic targets excluding oncology. The researchers found that 74% of these 336 gene therapies were concentrated in five medical specialties, that is, hematology, endocrinology, neurosciences, cardiology, and ophthalmology. When classifying by different disease families, inborn errors of metabolism was the disease category with a majority of ongoing gene therapy trials.

(Landscape of gene therapy programs by organ system and disease area, source: Mol. Therapy)

When looking at specific clinical indications, Duchenne muscular dystrophy (DMD) was the clinical indication with the highest number of gene therapies being developed (15). HIV gene therapies (12 gene therapy programs) and hemophilia (11 gene therapy programs) took the second and third place respectively.

In terms of the number of gene therapy/editing programs being developed by a particular company or organization, Sangamo Therapeutics (SGMO) took the top spot (see the figure below).

(Landscape of gene therapy programs by company/organization, source: Mol. Therapy)

In conclusion, gene therapy has recovered from its earlier setbacks to emerge as one of the most innovative areas in biotechnology. In this first article of the series, I have provided a brief background about gene therapy, its history, and a broad top-down landscape. In the next article in the premium service, I will discuss various delivery systems for gene therapy.

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Disclosure: I am/we are long BLUE, ORTX, QURE, SGMO, AXGT, CRSP. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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AI uncovers genes linked to heart failure – FierceBiotech

Posted: September 27, 2019 at 9:44 am

Artificial intelligence has been embraced for its ability to offer insight from big data. By applying the technology to genetics, a research team led by Queen Mary University of London has found clues that they say could aid the development of new drugs for heart failure and identify people at risk of the disease.

Based on an AI analysis of heart MRI images from 17,000 volunteers in UK Biobank, the researchers linked genetic factors to 22% to 39% of abnormalities in the size and function of the hearts left ventricle, which pumps blood into the aorta. They published the findings in the journal Circulation.

The team identified or confirmed 14 regions in the human genome that play a part in determining the size and function of the left ventricle, becausethey contain genes that regulate the early development of heart chambers and the contraction of heart muscle. Enlargement of left ventricle is a condition that can hamper the heart muscles ability to contract and pump blood, putting the patient at high risk of heart attack.

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This study has shown that several genes known to be important in heart failure also appear to regulate the heart size and function in healthy people, said study co-author Steffen Petersen of Queen Mary in a statement. That understanding of the genetic basis of heart structure and function in the general population improves our knowledge of how heart failure evolves.

RELATED:Bayer teams up with AI firm Sensyne Health to mine NHS data for its heart disease pipeline

There is a growing interest in using AI to gain insights into cardiovascular disease. Bayer recently partnered up with Sensyne Health, which uses AI to mine patient data from the U.K. National Health Service, including genomic sequencing data and real-world evidence, to help design clinical studies and accelerate drug discovery.

Many research teams having been looking at different ways to treat heart disease, including using immune therapies and regenerative approaches. Scientists at the University of Pennsylvania, for example,developed genetically modified T cells to attack and remove cardiac fibroblasts, which can lead to cardiac fibrosis. Vanderbilt University researchers identified Roches SYN0012, originally designed to treat rheumatoid arthritis, as a promising candidate that could dampen inflammation of heart tissue after a heart attack. Such inflammation can progress to acute episodes andchronic heart failure.

To help repair damaged cardiac tissue after a heart attack, scientists at the University of Cambridge in the United Kingdom and the University of Washington combined two types of cells derived from human stem cellsheart muscle cells and supportive epicardial cells that help the muscle cells live longer. A team at the the Morgridge Institute for Research previously added a drug called RepSox to stem cells to build better smooth muscle cells that can grow into functional arterial cells.

The Queen Mary researchers believe the 14 regions of the genome they fingered in their new study could be just the beginning of a larger story about genes and heart disease. Our academic and commercial partners are further developing these AI algorithms to analyze other aspects of cardiac structure and function,lead researcher Nay Aung said in the statement.

Aung and colleagues argue the genetic markers theyve already uncovered could help identify those at high risk of developing heart disease or open up new avenues for targeted treatments. The genetic risk scores established from this study could be tested in future studies to create an integrated and personalized risk assessment tool for heart failure, Aung said.

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University of Pennsylvania || Cell and Molecular Biology …

Posted: September 7, 2019 at 4:35 pm

Overview|Leadership|Research |Communication & Outreach| Academic Requirements

Program OverviewStudents within DSRB address key questions in Developmental biology, Stem cell/niche interactions, and Regenerative Biology. The thread that unites these areas is the compelling drive to understand how tissues are first formed, how they are maintained, and how they can be repaired. The primary mission of DSRB faculty is to train students in hypothesis-driven research at the forefront of these areas.

Individualized Training:The curriculum in DSRB is set up to provide students with a strong foundation in fundamental developmental biology, as well as in stem cell and regenerative biology (see Academics). The curriculum is complemented by an Individualized Training Plan tailored to your background, to your interests, and to your career plans. You will develop this plan in consultation with faculty advisors, and take advantage of unique resources provided by the program, such as the extensive network of alumnae distributed among various post-PhD career paths.

Stunning Research Accomplishments:Evidence of the quality of research and the strength of the training plan is shown by the success rate among DSRB students who have applied for individual research training grants. Fully two-thirds of our students have been awarded NIH grants for their work (20/30; an unheard of 67% success rate). Those successes are spread across diverse areas of research within DSRB labs .

A Cohesive Program:We celebrate the diverse research within our community in our renowned Research-in-Progress Lunchtime series, where students present current work, with opportunity for discussion over good food. The series serves as one mechanism to polish communication skills and receive timely feedback, both on the science and on its presentation. Connections among our students are also reinforced by participation in the Developmental Biology Journal Club and the Student-invited Distinguished Lecturer Series. Our students support each other through a peer-mentoring program, and by running mock critique sessions to prepare for the oral Preliminary Exam and for fellowship proposals. Finally, students appointed to the Developmental Biology Training Grant (https://www.med.upenn.edu/dbtraininggrant/) are fully integrated into our program.

Continued Success as Your Career Progresses:Once students have received their PhD, their success continues as they leave our program and step onto their chosen path. Of the almost 50 DSRB students who received their PhD over the last 10 years, 90% went directly into academic research, medicine, the pharmaceutical or biotech industries.

PhiladelphiaMany other major Academic and Medical Institutions are right here, across Philadelphia, including the Childrens Hospital of Philadelphia, Fox Chase Cancer Center, The Wistar Institute, Thomas Jefferson University and several others. The Greater Philadelphia area has always been home to various Biotech and Pharma institutions. But, over the last five years it is becoming a hub for Next Generation therapeutics:http://www.phillymag.com/business/2017/06/17/cellacon-valley-philadelphia-medical-technology/ All of this converges to provide wonderful opportunities as you navigate your career choices.

This all can happen for you while experiencing an historical and vibrant city, alive with the Performing Arts, Culture, Sports and rich in world-class Museums and unique Collections. Both the University and the City of Philadelphia are incredibly diverse and welcoming communities, with opportunities for all: http://www.upenn.edu/life-at-penn.

DSRB Faculty Leadership & Committee Structure

DSRB Executive Committee:Jonathan Raper, Professor of NeuroscienceMichael Granato, Professor of Cell & Developmental BiologySarah Millar, Professor of DermatologyMontserrat Anguera, Assistant Professor of Animal Biology, the Vet SchoolCamille Syrett, DSRB Student Representative

DSRB Admissions Committee:Greg Bashaw, Professor of NeurosciencePaul Gadue, Associate Professor, Pathology & Lab Medicine

DSRB First year Advisors:Mary Mullins, Professor of Cell & Developmental BiologyMeera Sundaram, Professor of Genetics

DSRB Second (Prelim) year advisors:Paul Gadue, Associate Professor, Pathology & Lab Medicine Judith Grinspan, Professor of NeurologyWenqin Luo, Assistant Professor of NeurosciencePatrick Seale, Associate Professor of Cell & Developmental Biology

DSRB Curriculum Committee:Dan Kessler, Professor of Cell & Developmental BiologyChris Lengner, Associate Professor of Animal BiologyMary Mullins, Professor of Cell & Developmental Biology

ResearchBreadth of Research in DSRBDSRB Students are exposed to interdisciplinary training in gametogenesis; embryonic and fetal development; nervous system development and its wiring; the genesis of tissues and organ systems as well as their homeostasis, metabolism and repair. Exciting work is unraveling the basic biology of these processes, as well as understanding the consequences of and working toward the amelioration of diseases affecting their function. Complementing these areas is work on Embryonic Stem Cells (ESCs) and Induced Pluripotent Stem Cells (iPSCs; patient-derived iPSCs), using genome-scale approaches to understand disease processes in vitro in order to develop cell replacement strategies for therapy. The proximity of the University of Pennsylvania Health System, a world-renowned academic medical center, provides unparalleled opportunity for translational research.

State-of-the-Art Tools at your disposalDSRB Students have at their disposal all the tools of the modern genomic era. For example, some students have been developing high-throughput and genomic-scale screens, using deep sequencing, ChIP-SEQ, RNAi tools or small molecule libraries, while others are capitalizing on classical genetic analyses or CRISPR/Cas9-based screens. Some have been utilizing high-resolution microscopy techniques, including live-imaging of cells, organs and tissues in vivo, while others have adapted novel biochemical, epigenetic and cell biological approaches to their questions. Some students are actively isolating and manipulating ESCs and iPSCs, as well as characterizing the nuclear reprogramming events required to coax these cells into distinct neuroepithelial, mesodermal or endodermal lineages, an essential step in taking these powerful, biomedically promising entities into the clinic. Facilitating this work is access to world-class cores, including those for Next Generation Sequencing, Proteomics, the Zebrafish facility, Stem cell & Xenografting, and Microscopy (http://www.med.upenn.edu/cores/).

Expansive Choice of Systems in which to workYou have the opportunity to choose among a wide variety of experimental model systems. This facilitates comparative and collaborative work, and can significantly speed your work. For example, you might begin attacking a question in a system that is highly adapted to a particular approach, and then can shift your work to another system more amenable to a different approach, or for addressing the follow-up part of the question. The systems used in DSRB labs include plants, such as Arabidopsis; the yeasts; several invertebrate organisms, such as the nematode worm, fruitfly and ant; various vertebrate models, such as zebra fish, the frog, chick, rat and mouse; and, of course, various pluripotent types of stem cells, both normal and patient-derived.

Links to the Faculty areas of research in DSRB:

GametogenesisEmbryo & Fetal developmentCell Migration, Polarity and Morphogenesis Neurogenesis and Axonogenesis Tissue & Organ FormationStem Cell/Niche Interactions Regenerative Biology Epigenetics, gene regulatory networks & signal integration

Communication & Outreach

Communicating your science:An important facet of your training is developing the ability to communicate effectively and interact productively with scientific peers. For this reason, the DSRB program uses several mechanisms to promote communication of your research. This includes the popular student research-in-progress lunches -- a very successful forum, fostering cohesion among the students in DSRB. Communication skills are also sharpened by opportunities to host student-invited seminar speakers; to give presentations at various symposia, such as the CAMB Graduate Symposium, various Departmental or Institute symposia, and, of course, at National and International meetings.

Outreach to the Next GenerationStudents trained in DSRB have an important responsibility to enhance scientific and technological understanding within our communities. We want our students to convey to diverse audiences the general excitement about DSRB research as well as of their own specific accomplishments. For these reasons, we support and encourage the participation in any of several acclaimed outreach activities. Opportunities range from a semester as a teaching assistant to participation in a nationally renowned outreach program, or mentoring an intern in a research project for a summer. An appropriate time for our graduate students to participate in outreach would be in the middle-to-out years of their work, when coursework is complete and a thesis project is solidly underway.

-- BioEYES: with over 12 years in area public schools, the program is run by Dr. Jamie Shuda, an experienced educator who develops university-community partnerships in science. She will provide training, help establish relationships with students and teachers, and serve as a mentor to help assess impact and effectiveness. Students are encouraged to adapt the existing programs to best communicate their research focus to the most appropriate audience. http://bioeyes.org/index.html

-- The Summer Undergraduate Internship Program (SUIP):http://www.med.upenn.edu/bgs/applicants_suip.shtmlRun successfully by Biomedical Graduate Studies for over 15 years, the program serves some 30 college interns each summer, and particularly so those from underrepresented minority groups or disadvantaged backgrounds, those with disabilities, or who attend small colleges. In this program you will directly oversee the interns research in collaboration with your PI, and coach them in their summers end presentation to the Leadership Alliance National Symposium.

-- Mentoring local high school students in STEM science fair projects (Science, Technology, Engineering & Math); Running our DSRB booth at the Philadelphia Science on the parkway fair.

Academics

Program course requirementsTypical course & research schedule for DSRB students:

Fall of first year:- Cell Biology (BIOM 600)- First year seminar (literature readings + discussions; CAMB 605)- Lab Rotation #1 (11 weeks)

Spring of first year: - Regulation of the Genome (BIOM 555)- Biological Data Analysis (BIOM 611) - Principles of Development (required for DSRB; CAMB 511) - Lab Rotations #2 & #3 (each lasting 11 weeks)

Summer between first & second year:- Research in prospective thesis lab

Fall of second year:- Required for DSRB: one of two concept-based, seminar offerings:

- Neurodevelopment, Regeneration & Repair Seminar (Bashaw & Luo; CAMB 597).The course integrates neural specification & development, with essential functions of neurons in guidance, synapse formation and function, along with neural regeneration & repair.

- Stem Cells (Gadue & Rompolas; CAMB 697) This course covers the challenges in stem cell & regeneration biology including reprogramming, embryonic and tissue-specific stem cells, tissue regeneration, and tissue engineering.

- One Elective: any graduate-level course selected based on research interest (can also be whichever of the above was not chosen to satisfy the DSRB requirement)

- Thesis lab Research

Spring of second year:

- Two Electives: selected based on research interest- Prelim prep writing course (CAMB 695)- Thesis lab Research

Elective choices:Students have tremendous flexibility, and in consultation with faculty advisors will select electives that fit their research interests and prospective directions. Suggested electives:

CAMB 534:Seminar on Current Genetic ResearchCAMB 542:Topics in Molecular MedicineCAMB 550:Genetic PrinciplesCAMB 597:Neural Development, Regeneration and RepairCAMB 608:Regulation of Eukaryotic Gene ExpressionCAMB 632:Cell Control by Signal Transduction PathwaysCAMB 691/CAMB 692:Advanced Topics in Cell Biology and PhysiologyCAMB 697:The Biology of Stem CellsCAMB 709: Quantitative Imaging and Analysis for Biologists (QIAB)GCB 535: Introduction to BioInformaticsGCB 536: Computational BiologyBIOL 446: Statistics for BiologistsEPID 575: Introduction to Genetic Epidemiology

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Robert Lanza – Wikipedia

Posted: May 12, 2019 at 4:49 am

Robert P. Lanza

Lanza at a laboratory in October 2009.

Robert Lanza

Robert Lanza (born 11 February 1956) is an American medical doctor, scientist and philosopher. He is currently Head of Astellas Global Regenerative Medicine,[1] and is Chief Scientific Officer of the Astellas Institute for Regenerative Medicine and Adjunct Professor at Wake Forest University School of Medicine.

Lanza was born in Boston, Massachusetts, and grew up south of there, in Stoughton, Massachusetts.Lanza "altered the genetics of chickens in his basement," and came to the attention of Harvard Medical School researchers when he appeared at the university with his results.Jonas Salk, B. F. Skinner, and Christiaan Barnard[citation needed] mentored Lanza over the next ten years.[2]Lanza attended the University of Pennsylvania, receiving BA and MD degrees.There, he was a Benjamin Franklin Scholar and a University Scholar. Lanza was also a Fulbright Scholar. He currently resides in Clinton, Massachusetts.

Lanza was part of the team that cloned the world's first early stage human embryos,[3][4] as well as the first to successfully generate stem cells from adults using somatic-cell nuclear transfer (therapeutic cloning).[5][6]

Lanza demonstrated that techniques used in preimplantation genetic diagnosis could be used to generate embryonic stem cells without embryonic destruction.[7]

In 2001, he was also the first to clone an endangered species (a Gaur),[8] and in 2003, he cloned an endangered wild ox (a Banteng)[9] from the frozen skin cells of an animal that had died at the San Diego Zoo nearly a quarter-of-a-century earlier.

Lanza and his colleagues were the first to demonstrate that nuclear transplantation could be used to reverse the aging process[10] and to generate immune-compatible tissues, including the first organ grown in the laboratory from cloned cells.[11]

Lanza showed that it is feasible to generate functional oxygen-carrying red blood cells from human embryonic stem cells under conditions suitable for clinical scale-up. The blood cells could potentially serve as a source of "universal" blood.[12][13]

His team discovered how to generate functional hemangioblasts (a population of "ambulance" cells[14]) from human embryonic stem cells.In animals, these cells quickly repaired vascular damage, cutting the death rate after a heart attack in half and restoring the blood flow to ischemic limbs that might otherwise have required amputation.[15]

In 2012 Lanza and a team led by Kwang-Soo Kim at Harvard University reported a method for generating induced pluripotent stem (iPS) cells by incubating them with proteins, instead of genetically manipulating the cells to make more of those proteins.[16][17][18]

Lanza's team at Advanced Cell Technology were able to generate retinal pigmented epithelium cells from stem cells, and subsequent studies found that these cells could restore vision in animal models of macular degeneration.[19][20]

In 2009 Geron became the first company that received approval for a clinical trial of an embryonic stem cell-based treatment for use in people with spinal cord injury; in 2010 ACT received the second FDA approval to use its ESC-based cell therapy for Stargardt disease.[21][22] In 2011 ACT received approval in the UK to expand the Stargart trial there; this was the first approval to study an ESC-based treatments in Europe.[23][24] The first person received the embryonic stem cell treatment in the UK in 2012.[25]

The results were reported in the Lancet in 2012,[26] with a follow up paper in 2014.[27]

In 2007, Lanza's article titled "A New Theory of the Universe" appeared in The American Scholar.[28] The essay addressed Lanza's idea of a biocentric universe, which places biology above the other sciences.[29][30][31] Lanza's book Biocentrism: How Life and Consciousness are the Keys to Understanding the Universe followed in 2009, co-written with Bob Berman.[32] Reception for Lanza's hypothesis has been mixed.[33]

Lanza has received numerous awards and other recognition, including TIME Magazines 2014 Time 100 list of the "100 Most Influential People in the World",[34] Prospect magazine 2015 list of Top 50 World Thinkers,[35] Marquis Whos Who 2018 Lifetime Achievement Award,[36] the 2013 Il Leone di San Marco Award in Medicine (Italian Heritage and Culture Committee, along with Regis Philbin, who received the award in Entertainment),[37] a 2010 National Institutes of Health (NIH) Directors Award for Translating Basic Science Discoveries into New and Better Treatments;[38] a 2010 Movers and Shakers Who Will Shape Biotech Over the Next 20 Years (BioWorld, along with Craig Venter and President Barack Obama);[39] a 2005 Wired magazine "Rave Award" for medicine For eye-opening work on embryonic stem cells,[40] and a 2006 Mass High Tech journal All Star award for biotechnology for pushing stem cells future.[41][42]

Lanza has authored and co-edited books on topics involving tissue engineering, cloning, stem cells, regenerative medicine, and world health.

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Cary Ortho Utilizes Bone Marrow Stem Cells for General …

Posted: May 12, 2019 at 4:49 am

Stem cells are the next frontier in the treatment of orthopaedic and spinal disorders, and the Cary Orthopaedics team is leading the way.

Using stem cells harvested from an adult patients own bone marrow,Dr. Sameer Mathurand Dr. Nael Shanti both board-certified orthopaedic spinal surgeons have developed a minimally invasive remedy for those suffering from degenerative disc disease, back pain and spinal arthritis. Applying a similar approach, Cary OrthosDr. Douglas Martini a fellowship-trained, board-certified orthopaedic surgeon specializing in sports medicine has crafted a pain-relief solution for patients living with osteoarthritis and soft tissue injuries.

Multiple research studies have shown a significant reduction in low back and joint pain and improved function after stem cell injections. While these treatments are new, 80% to 90% of patients are already reporting improvement in their symptoms after orthopaedic stem cell treatments.

Many patients suffering from degenerative disc diseases or low back pain are often not ideal candidates for surgery, and some who have chosen to undergo surgery have had unsatisfactory results. Therefore, the typical remedy for chronic orthopaedic conditions is extensive physical therapy combined with oral anti-inflammatory medications. The result: The majority of patients still had to live with pain.

Physicians at Cary Orthopaedics are utilizing orthopaedic stem cell treatment using the patients own bone marrow, the soft, spongy tissue found in the center of bones. Bone marrow in adults contains a rich reservoir of multipotent stem cells also known as Mesenchymal Precursor Cells (MPCs) that can be extracted from the patients pelvis or hip bone. Due to their unique, regenerative composition, these cells can become various types of tissues including soft tissue, bone or cartilage, which make them an excellent resource for repairing and rebuilding damaged tissue, accelerating the healing process and improving overall function.

Thanks to advancements in technology, the removal and harvesting process has become easier and less expensive. Since this is a minimally invasive procedure, it has fewer side effects compared to traditional surgery, and it causes minimal discomfort to the patient.

Bone marrow injections are a breakthrough for patients in pain. Dr. Martini, a sports medicine physician at Cary Orthopaedics, has been active in the sports medicine community, previously serving as team physician for the Carolina Hurricanes, numerous colleges, and local high schools. After 25 years of experience in sports medicine, he realizes the need for improved treatment options for the greying athlete. He has begun incorporating bone marrow aspirate concentrate (BAC) into the treatment of both acute and chronic soft tissue and joint-related injuries. I believe this will be equally helpful to the patient who needs to exercise for overall health benefits as it would be for those who need to stay at their peak athletic performance, says Dr. Martini.

We have found based on our research and experience that stem cell therapy can be very safe and effective when used with the appropriate patient population, said Kevin G. Morrison, PA-C, a member of Dr. Martinis team. All the feedback to this point has been quite positive, both on the process of having the procedure done as well as the early response. But ultimately long-term data will need to be compiled and critically examined.

Much of the previous research into stem cells has centered around placental stem cells, which can also adapt into other types of tissues. However, these have not performed well when put to the test for orthopaedic treatment. Bone marrow aspirate concentrate provides MPCs that can transform into osteocytes, chondrocytes and adipocytes, all of which are important in treating orthopedic conditions.

The latest research around mesenchymal stem cells, specifically bone marrow aspiration, is certainly promising. Dr. Martini will continue to collect more data and review patients responses.

Dr. Mathur has been an instrumental force in elevating the level of patient care at Cary Orthopaedic Spine Center since joining the practice in 2008. Dr. Mathur completed his medical school at the University of Pennsylvania and spinal reconstructive fellowship at the Rush University Medical Center in Chicago. He also taught at Dana Farber Cancer Institute in Boston. Over the last 10 years, in conjunction with the National Institutes of Health, he has conducted significant study of disc degeneration and analysis of the expression of genes that may damage the disc.

In the past decade, there have been several advancements in spinal surgery, but regenerative medicine is the next frontier, said Dr. Mathur. I see so many patients that have low back pain and leg pain from degenerative disc disease. For many, there is no good surgical treatment, and stem cell injections may be a viable option.

As an orthopaedic spine specialist, Dr. Mathur is not only an expert in spinal surgery but also in the diagnosis and treatment of a wide range of spinal problems. His depth of experience allows him to best determine whether a patient would benefit from physical therapy, stem cell injections or surgical intervention. When providing stem cell treatment, Dr. Mathur performs a single injection for all patients, whereas other clinics typically require multiple injections over several weeks.

There is currently extensive, ongoing research on the application of stem cell therapy and tissue regeneration, including an application for spinal cord injury and disc pathology, which is very exciting, said Dr. Shanti, who has dedicated a great deal of time researching the potential impact stem cell therapy can provide for his patients. Dr. Shanti believes stem cell therapy is the next great advancement in healthcare with an application for a wide spectrum of medical conditions.

Recently recognized as Top Orthopaedic Doctor by The Leading Physicians of the World for the outstanding patient care, Dr. Shantis in-depth experience and understanding of the spine allows him to guide his patients especially those with chronic back pain to the most appropriate path of treatment with the shared collaborative goal of pain relief. Dr. Shanti completed his spine surgery fellowship training at the prestigious New England Baptist Hospital, Tufts University program with an emphasis on minimally invasive spine surgery, and he has authored and presented multiple papers and textbooks on the advancement of minimally invasive spine surgery.

Orthopaedic stem cell treatment is an excellent solution for patients with degenerative disc disease and also those suffering from arthritis of the spine, bulging disc, low back pain, facet joint pain or disc with annular tears.

The stem cell injection is a same-day procedure that generally takes one hour to perform. The actual extraction of bone marrow takes up to 10 minutes. The bone marrow extraction site typically the back of the patients hip or pelvis bone is numbed using a mixture of local anesthetics. A suctioned syringe is attached to a long needle that reaches the posterior aspect of the hip. The patient may experience a minimal amount of discomfort during the extraction.

The sample is collected, transferred through a filter, and then placed into a centrifuge for spinning. The speed separates the stem cells and platelets from the bone marrow. This concentration of stem cells is then reintroduced into the degenerative or painful area under image guidance with fluoroscopy to confirm accurate placement.

The harvesting site will be numb for 1 to 2 hours after the procedure, so the patient will need to have transportation home. It is permissible to fly after the treatment, but this may cause increased pain or discomfort.

Stem cell therapy relies on the bodys own regenerative process to heal, which takes time. Patients have seen the benefits in two to three months after treatment; however, many have noticed improvements in symptoms sooner.

The recommended age range for the treatment is 20 to 70 years old. As the body ages, the quality and quantity of stem cells slowly decline. After age 70, patients may experience a sharper decline in stem cells, resulting in less beneficial outcomes.

If you think you might be a candidate for orthopaedic stem cell therapy treatment, contact Cary Orthopaedics to schedule a consultation.

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