Cell Therapy Pioneer David Epstein Expands Role with Rubius Therapeutics to Become Executive Chairman – Business Wire (press release)

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Rubius Therapeutics, a biotechnology company pioneering the creation of a new class of extraordinarily active, ready-to-use and potentially life-saving cellular therapies, today announced that David Epstein, previously chief executive officer of Novartis Pharmaceuticals, has expanded his role at Rubius to become executive chairman. Epstein was one of the first to identify the potential of chimeric antigen receptor (CAR) T-cell therapy, and subsequently led efforts at Novartis Pharmaceuticals to leverage the technology. Ultimately, his teams efforts resulted in a breakthrough product for pediatric/young adult acute lymphoblastic leukemia (ALL) and potentially other cancers.

Following my experience with CAR T-cell therapy, I assessed many technology platforms looking for an elegant way to take cellular therapy to the next level, said Epstein. I am excited by the unique potential of the Rubius Red Cell Therapeutics (RCT) platform to address many diseases for which no adequate treatments exist. When compared with most other cell therapy platforms, RCTs have broader application for a wider range of molecular targets. Moreover, RCTs can be produced at scale and stored in advance, allowing physicians to treat larger patient populations with an immediately accessible therapeutic option. I believe Rubius pioneering of the next generation of cellular therapy will meaningfully impact patients lives.

Epstein joined Rubius as chairman of the board early in 2017. In June, Rubius successfully completed an oversubscribed private financing of $120 million. The proceeds from the financing will be used to advance the Companys RCT product portfolio, further build out its team and prepare to enter human clinical trials in 2018.

I look forward to Davids continued partnership as we work to advance the next generation of cell therapy, said Torben Straight Nissen, Ph.D., president of Rubius Therapeutics. With over 25 years of experience in drug development, deal making and commercialization, his expertise is invaluable to the Company.

Epstein served as chief executive officer and division head of Novartis Pharmaceuticals from 2010 to 2016. In addition, he served as head of Novartis Oncology, building the oncology business from start-up to number two in the world. Epstein has overseen the development, filing and approval of more than 30 novel medicines, including Glivec, Tasigna, Gilenya, Entresto and Cosentyx. Epstein is also an executive partner at Flagship Pioneering.

About Red-Cell Therapeutics

Red-Cell Therapeutics are genetically engineered, enucleated red cells that are being developed to provide allogeneic, off-the-shelf therapies to patients across multiple therapeutic areas. RCT advantages over other therapies include immuno-privileged presentation of proteins within or on the red cell, high target avidity and affinity resulting in highly potent and selective therapies, and long circulation half-life. Rubius RCTs exhibit fundamentally unique biology and have been engineered to replace missing enzymes for patients living with a variety of rare diseases, to kill tumors, and upregulate or downregulate the immune system to treat both cancer and autoimmune disorders.

AboutRubiusTherapeutics

Rubius Therapeutics is developing Red-Cell Therapeutics (RCTs) as a new class of medicines to address a wide array of indications, with leading applications in cancer, rare and autoimmune disease, as well as additional potential in infectious and metabolic diseases. The company was founded and launched in 2014 by Flagship VentureLabs, the innovation foundry of Flagship Pioneering. Rubius has successfully engineered and manufactured red cells that express therapeutic proteins for use in the treatment of serious diseases. The Company is now demonstrating that these high performing, ready-to-use RCTs have preclinical activity across a spectrum of medical applications. Rubius has generated more than 200 prototypes to date. For more information, please visit http://www.rubiustx.com.

About Flagship Pioneering

Flagship Pioneering conceives, creates, resources and develops first-in-category life sciences companies. Its institutional innovation foundry, Flagship VentureLabs, is where Flagships team of scientific entrepreneurs systematically evolves enterprising ideas into new fields, or previously undiscovered areas of science into real-world inventions and ventures. Since its launch in 2000, the firm has applied its hypothesis-driven innovation process to originate and foster nearly 100 scientific ventures, resulting in over $20 billion in aggregate value, 500+ issued patents and more than 45 clinical trials for novel therapeutic agents. Since inception, Flagship has capitalized its growing portfolio with over $1 billion coming from $1.75 billion of aggregate investor capital committed across five funds. To learn more about Flagship Pioneering, please visit http://www.FlagshipPioneering.com.

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Cell Therapy Pioneer David Epstein Expands Role with Rubius Therapeutics to Become Executive Chairman - Business Wire (press release)

Gene Therapy Is Now Available, but Who Will Pay for It? – Scientific American

By Ben Hirschler

LONDON (Reuters) - The science of gene therapy is finally delivering on its potential, and drugmakers are now hoping to produce commercially viable medicines after tiny sales for the first two such treatments in Europe.

Thanks to advances in delivering genes to targeted cells, more treatments based on fixing faulty DNA in patients are coming soon, including the first ones in the United States.

Yet the lack of sales for the two drugs already launched to treat ultra-rare diseases in Europe highlights the hurdles ahead for drugmakers in marketing new, extremely expensive products for genetic diseases.

After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such as haemophilia. They argue that therapies offering one-off cures for intractable diseases will save health providers large sums in the long term over conventional treatments which each patient may need for years.

In the past five years, European regulators have approved two gene therapies - the first of their kind in the world, outside China - but only three patients have so far been treated commercially.

UniQure's Glybera, for a very rare blood disorder, is now being taken off the market given lack of demand.

The future of GlaxoSmithKline's Strimvelis for ADA-SCID - or "bubble boy" disease, where sufferers are highly vulnerable to infections - is uncertain after the company decided to review and possibly sell its rare diseases unit.

Glybera, costing around $1 million per patient, has been used just once since approval in 2012. Strimvelis, at about $700,000, has seen two sales since its approval in May 2016, with two more patients due to be treated later this year.

"It's disappointing that so few patients have received gene therapy in Europe," said KPMG chief medical adviser Hilary Thomas. "It shows the business challenges and the problems faced by publicly-funded healthcare systems in dealing with a very expensive one-off treatment."

These first two therapies are for exceptionally rare conditions - GSK estimates there are only 15 new cases of ADA-SCID in Europe each year - but both drugs are expected to pave the way for bigger products.

The idea of using engineered viruses to deliver healthy genes has fuelled experiments since the 1990s. Progress was derailed by a patient death and cancer cases, but now scientists have learnt how to make viral delivery safer and more efficient.

Spark Therapeutics hopes to win U.S. approval in January 2018 for a gene therapy to cure a rare inherited form of blindness, while Novartis could get a U.S. go-ahead as early as next month for its gene-modified cell therapy against leukaemia - a variation on standard gene therapy.

At the same time, academic research is advancing by leaps and bounds, with last week's successful use of CRISPR-Cas9 gene editing to correct a defect in a human embryo pointing to more innovative therapies down the line.

Spark Chief Executive Jeffrey Marrazzo thinks there are specific reasons why Europe's first gene therapies have sold poorly, reflecting complex reimbursement systems, Glybera's patchy clinical trials record and the fact Strimvelis is given at only one clinic in Italy.

He expects Spark will do better. It plans to have treatment centers in each country to address a type of blindness affecting about 6,000 people around the world.

Marrazzo admits, however, there are many questions about how his firm should be rewarded for the $400 million it has spent developing the drug, given that healthcare systems are geared to paying for drugs monthly rather than facing a huge upfront bill.

A one-time cure, even at $1 million, could still save money over the long term by reducing the need for expensive care, in much the same way that a kidney transplant can save hundreds of thousands of dollars in dialysis costs.

But gene therapy companies - which also include Bluebird Bio, BioMarin, Sangamo and GenSight - may need new business models.

One option would be a pay-for-performance system, where governments or insurers would make payments to companies that could be halted if the drug stopped working.

"In an area like haemophilia I think that approach is going to make a ton of sense, since the budget impact there starts to get more significant," Marrazzo said.

Haemophilia, a hereditary condition affecting more than 100,000 people in markets where specialty drugmakers typically operate, promises to be the first really big commercial opportunity. It offers to free patients from regular infusions of blood-clotting factors that can cost up to $400,000 a year.

Significantly, despite its move away from ultra-rare diseases, GSK is still looking to use its gene therapy platform to develop treatments for more common diseases, including cancer and beta-thalassaemia, another inherited blood disorder.

Rivals such as Pfizer and Sanofi are also investing, and overall financing for gene and gene-modified cell therapies reached $1 billion in the first quarter of 2017, according to the Alliance of Regenerative Medicine.

Shire CEO Flemming Ornskov - who has a large conventional haemophilia business and is also chasing Biomarin and Spark in hunting a cure for the bleeding disorder - sees both the opportunities and the difficulties of gene therapy.

"Is it something that I think will take market share mid- to long-term if the data continues to be encouraging? Yes. But I think everybody will have to figure out a business model."

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Gene Therapy Is Now Available, but Who Will Pay for It? - Scientific American

Daughter leads full life thanks to stem cell therapy – Independent Online

Erna West and daughter Gizelle, who was diagnosed with Fanconi anaemia aged 9. Her life was saved by a blood marrow transplant from her mother. Picture: SUPPLIED

Erna West and daughter Gizelle, who was diagnosed with Fanconi anaemia aged 9. Her life was saved by a blood marrow transplant from her mother. Picture: SUPPLIED

Erna West and daughter Gizelle, who was diagnosed with Fanconi anaemia aged 9. Her life was saved by a blood marrow transplant from her mother. Picture: SUPPLIED

The one thing I still remember is us driving in our car and my daughter asking me, Mommy, am I going to die? West recounted.

Now an ardent advocate for stem cell therapy and storage, West, a product specialist for CryoSave, credits stem cells with saving her daughters life.

Her daughter needed a bone marrow transplant, which involved the transplanting of stem cells.

She found she was an exact donor match for her daughters bone marrow transplant - a one-in-a-million occurrence.

When youre faced with a situation such as that as a parent, you want and are willing to do anything to save your childs life I just want parents to understand what stem cells can do.

Fast forward 21 years and stem cells are revolutionising health care and through modern technology, parents can store their newborn babys umbilical cord stem cells in case of any future illnesses or health care needs.

Stem cells are present in the human body throughout life, constantly repairing tissue damaged by normal activity, the environment and other extraneous factors. They can replicate or regenerate themselves and have the ability to differentiate into any kind of specialised cell in the body.

Africa is the only continent without a public stem cell bank - private stem cell storage banks are in increasing demand as research and medical innovation has shown that many blood cancers, blood disorders, autoimmune diseases and immunodeficiencies are treatable with cord blood.

Umbilical cord blood and stem cell banking is still a relatively novel concept in South Africa.

However, new parents are increasingly opting to have their newborn babies stem cells extracted from their umbilical cords.

According to CryoSave - which stores 7 800 client stem cell samples - the process is simpler and quicker than one might expect.

Once the baby is born, the umbilical cord is clamped and cut as per normal in any birth. It is only after this that the blood and tissue are collected from the cord - which is usually discarded as medical waste after the birth.

A babys umbilical cord stem cells are a 100% perfect match and biological parents stem cells will be at least a half-match.

There is a 25% probability of matching siblings and, unlike bone marrow transplants, one doesnt have to have a perfect match in transplants when making use of cord blood stem cells.

Today, umbilical cord blood stem cells are used in more than one-third of all blood stem cell transplants in the world.

Explaining the process behind the storage of umbilical cord cells at their labs, Christiene Botha, a lab quality manager said: The blood we receive goes through a rigorous sterilising, processing and freezing process.

The samples are then stored in liquid nitrogen tanks at a temperature of -196C.

But time is of the essence in this process.

The umbilical cord blood sample needs to reach the lab within 48 hours - and the cut off is at 64 hours - as blood cells start dying after 72 hours.

Depending on what product one uses to store the cells, storage rates can be from R250 to R300 a month.

The fact that we dont have a public national bank puts us at a disadvantage because it is the ideal. So there arent many choices for parents out there - but families can look after themselves through this type of storage.

"My daughter is 30-years-old, is married and lives a full life because of stem cells, West concluded.

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Daughter leads full life thanks to stem cell therapy - Independent Online

A Chip That Reprograms Cells Helps Healing, At Least In Mice – NPR

The chip has not been tested in humans, but it has been used to heal wounds in mice. Wexner Medical Center/The Ohio State University hide caption

The chip has not been tested in humans, but it has been used to heal wounds in mice.

Scientists have created an electronic wafer that reprogrammed damaged skin cells on a mouse's leg to grow new blood vessels and help a wound heal.

One day, creator Chandan Sen hopes, it could be used to be used to treat wounds on humans. But that day is a long way off as are many other regeneration technologies in the works. Like Sen, some scientists have begun trying to directly reprogram one cell type into another for healing, while others are attempting to build organs or tissues from stem cells and organ-shaped scaffolding.

But other scientists have greeted Sen's mouse experiment, published in Nature Nanotechnology on Monday, with extreme skepticism. "My impression is that there's a lot of hyperbole here," says Sean Morrison, a stem cell researcher at the University of Texas Southwestern Medical Center. "The idea you can [reprogram] a limited number of cells in the skin and improve blood flow to an entire limb I think it's a pretty fantastic claim. I find it hard to believe."

When the device is placed on live skin and activated, it sends a small electrical pulse onto the skin cells' membrane, which opens a tiny window on the cell surface. "It's about 2 percent of the cell membrane," says Sen, who is a researcher in regenerative medicine at Ohio State University. Then, using a microscopic chute, the chip shoots new genetic code through that window and into the cell where it can begin reprogramming the cell for a new fate.

Sen says the whole process takes less than 0.1 seconds and can reprogram the cells resting underneath the device, which is about the size of a big toenail. The best part is that it's able to successfully deliver its genetic payload almost 100 percent of the time, he says. "No other gene delivery technique can deliver over 98 percent efficiency. That is our triumph."

Chandan Sen, a researcher at Ohio State University, holds a chip his lab created that has reprogrammed cells in mice. Wexner Medical Center/The Ohio State University hide caption

Chandan Sen, a researcher at Ohio State University, holds a chip his lab created that has reprogrammed cells in mice.

To test the device's healing capabilities, Sen and his colleagues took a few mice with damaged leg arteries and placed the chip on the skin near the damaged artery. That reprogrammed a centimeter or two of skin to turn into blood vessel cells. Sen says the cells that received the reprogramming genes actually started replicating the reprogramming code that the researchers originally inserted in the chip, repackaging it and sending it out to other nearby cells. And that initiated the growth of a new network of blood vessels in the leg that replaced the function of the original, damaged artery, the researchers say. "Not only did we make new cells, but those cells reorganized to make functional blood vessels that plumb with the existing vasculature and carry blood," Sen says. That was enough for the leg to fully recover. Injured mice that didn't get the chip never healed.

When the researchers used the chip on healthy legs, no new blood vessels formed. Sen says because injured mouse legs were was able to incorporate the chip's reprogramming code into the ongoing attempt to heal.

That idea hasn't quite been accepted by other researchers, however. "It's just a hand waving argument," Morrison says. "It could be true, but there's no evidence that reprogramming works differently in an injured tissue versus a non-injured tissue."

What's more, the role of exosomes, the vesicles that supposedly transmit the reprogramming command to other cells, has been contentious in medical science. "There are all manners of claims of these vesicles. It's not clear what these things are, and if it's a real biological process or if it's debris," Morrison says. "In my lab, we would want to do a lot more characterization of these exosomes before we make any claims like this."

Sen says that the theory that introduced reprogramming code from the chip or any other gene delivery method does need more work, but he isn't deterred by the criticism. "This clearly is a new conceptual development, and skepticism is understandable," he says. But he is steadfast in his confidence about the role of reprogrammed exosomes. When the researchers extracted the vesicles and injected them into skin cells in the lab, Sen says those cells converted into blood vessel cells in the petri dish. "I believe this is definitive evidence supporting that [these exosomes] may induce cell conversion."

Even if the device works as well as Sen and his colleagues hope it does, they only tested it on mice. Repairing deeper injuries, like vital organ damage, would also require inserting the chip into the body to reach the wound site. It has a long way to go before it can ever be considered for use on humans. Right now, scientists can only directly reprogram adult cells into a limited selection of other cell types like muscle, neurons and blood vessel cells. It'll be many years before scientists understand how to reprogram one cell type to become part of any of our other, many tissues.

Still, Morrison says the chip is an interesting bit of technology. "It's a cool idea, being able to release [genetic code] through nano channels," he says. "There may be applications where that's advantageous in some way in the future."

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A Chip That Reprograms Cells Helps Healing, At Least In Mice - NPR

Texas Heart Institute Awarded Grant to Study Sex Differences in Cardiac Repair – Texas Medical Center (press release)

Earlier this year, Texas Heart Institute received Alpha Phi Foundations 2017 Heart to Heart Grant. The $100,000 grant will fund research led by Doris Taylor, Ph.D., director of the Regenerative Medicine Research and the Center for Cell and Organ Biotechnology at the Texas Heart Institute, to study cardiac repair in women at the cellular level.

Were just really passionate about these projects that have long-term clinical relevancy, as a women-driven organization and being committed to womens heart health, said Colleen Sirhal, vice chair of the Alpha Phi Foundation.

The study will explore sex differences in blood, bone marrow and stem cells of patients enrolled in cell therapy clinical trials.

While bone marrow cell therapy has been used to treat cardiovascular disease in clinical trials, very few studies have been conducted to assess the sex differences in efficacy and outcomes. By performing a proteomic analysis of the samples and evaluating the proteins that cells produce and secrete, the results could shed light on unanswered questions related to critical sex-specific differences in cardiovascular disease, potentially leading to improved cell therapies.

Its about time that were paying attention to sex differences, Taylor said. Were not just small men. The biology is different.

Heart disease remains the No. 1 cause of death in both men and women in the United States, yet theres a limited understanding in the scientific community as to why it affects men and women differently. For example, women 45 years old and younger have a higher likelihood than men of dying within a year of their initial heart attack.

In addition, women have a higher risk of developing small vessel disease, in which the walls of tiny vessels within the heart muscle become blocked rather than larger arteries, causing heart-related chest pain. Because the major coronary arteries may look normal, women with small vessel disease can have a heart attack go undiagnosed and untreated.

We know heart disease happens differently in men and women, Taylor said. More young women than men die of heart disease. Why is that? Is there something that happens early? If we only look at these women who are older, are we missing something major? By looking at healthy, normal younger women, were going to be able to do comparisons across time, comparisons by disease, and comparisons by sex. I think thats really exciting.

Historically, women and minorities have largely been underrepresented in research and clinical trials, especially pertaining to cardiovascular disease.

Dr. Taylors colleague at the Texas Heart Institute, Stephanie Coulter, M.D., a cardiologist and the director of the Center for Womens Heart and Vascular Health at Texas Heart Institute and a recipient of the 2013 Heart to Heart Grant, is actively recruiting younger women to participate in her research registry.

Since women are typically affected by heart disease a decade or more later than men, age may also have played a role in this underrepresentation, Coulter said. Our Womens Center research is focusing on women age 18 and older to address this very issue.

Coulter added that trials focusing on prevention in women, such as the Womens Health Initiative and Womens Health Study, have, in fact, had clinical impact. However, the percentage of women enrolling in clinical trials continues to be disproportionate to the prevalence of cardiovascular disease in women, but we are seeing improvements thanks to multiple initiatives in the U.S. that continue to address the issue of women in clinical trials.

Its easy for people to assume that if you study men, itll apply to women, but it seems anathema to people to assume that if you study women it might benefit men, Taylor said. At the end of the day, when it comes time to look at the data and ask, How does this treatment work in women? How does this treatment work in men?, oftentimes there arent enough women enrolled in the trials to split that out. Statistically, youd be doing yourself a disservice.

Taylor has spent nearly two decades studying key contributors to cardiac repair at the cellular level, specifically looking at proteins cells produce and secrete based on gender as a new frontier in cell therapy.

Early on in Taylors career, she studied how bone marrow cells behaved based on gender. She extracted cells from male mice and administered them to female mice and vice versa, allowing her to track the Y chromosome. The results showed that only the males treated with female cells improved. This phenomenon raised the question of whether or not the bone marrow cells were the same.

After measuring the bone marrow cells that were present in males and females, Taylor discovered that the cells were inherently different: In the male mice, there were more inflammatory cells, fewer progenitor and stem cells and a different number of immune cells than in the female mice. In addition, when the bone marrow cells were placed in a petri dish, the female cells produced more growth factors responsible for recruiting repair cells after an injury.

Taylor conducted follow-up experiments in which she gave female and male cells to both female and male mice. The results confirmed her hunch: The only cells that were reparative were the female cells.

It made me realize a critical detail for the first time:Every time we take bone marrow from a different person with the intention of delivering it back to them as a therapy, if we look at the cells present in the marrow, theyd be different, Taylor said. Which means, every time were doing an autologous cell therapytrial, in which you take bone marrow and deliver it back to an individual, you are giving each person a completely different or unique drug in that trial.

Through the Heart to Heart grant, the data from Taylors research will allow her to build upon her early research on sex differences and, hopefully, identify a way to optimize cell therapy.

Already cells are as good as some drugs. If we optimize them and choose the right cells for the right patient at the right time, maybe well hit the home run, Taylor said.

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Texas Heart Institute Awarded Grant to Study Sex Differences in Cardiac Repair - Texas Medical Center (press release)

Prostate Cancer Cells Become ‘Shapeshifters’ to Spread to Distant Organs – Laboratory Equipment

Johns Hopkins Kimmel Cancer Center scientists report they have discovered a biochemical process that gives prostate cancer cells the almost unnatural ability to change their shape, squeeze into other organs and take root in other parts of the body. The scientists say their cell culture and mouse studies of the process, which involves a cancer-related protein called AIM1, suggest potential ways to intercept or reverse the ability of cancers to metastasize, or spread.

Results of the research are described in the July 26 issue of Nature Communications.

For the study, the Johns Hopkins scientists mined publically available research data on the genetics and chemistry of hundreds of primary and metastatic cancers included in five studies of men with prostate cancer. They found that a gene called AIM1 (aka absent in melanoma 1), which makes proteins also called AIM1, is deleted in approximately 20 to 30 percent of prostate cancers confined to the gland and about 40 percent of metastatic prostate cancers. In addition, the scientists found, on average, two- to fourfold less amounts of AIM1 expression in metastatic prostate cancers compared with normal prostate cells or those from men with prostate cancers confined to the prostate, suggesting that reduction of AIM1 proteins is somehow linked to tumor spread.

Aside from its link to the development of melanoma, a deadly skin cancer, scientists knew little about the function of AIM1.

Our experiments show that loss of AIM1 proteins gives prostate cancer cells the ability to change shape, migrate and invade. These abilities could allow prostate cancer cells to spread to different tissues in an animal and presumably a person, said Michael Haffner, M.D., Ph.D., a pathology resident and former postdoctoral fellow at the Johns Hopkins Kimmel Cancer Center who is involved in the research. Its not the whole story of what is going on in the spread of prostate cancer, but it appears to be a significant part of it in some cases.

Looking more closely at the AIM1 gene and its protein levels in prostate cancer tissues, the Johns Hopkins scientists found that many times, even when the gene isnt completely deleted and its protein production is reduced, its location in the prostate cancer cell is highly abnormal compared with normal prostate cells. This occurs even in primary prostate cancer cells, which have invaded the local structures to form invasive cancer within the prostate gland, say the scientists.

The research team used dyes to track the location of AIM1 proteins in human cells grown in the lab and followed where they appear in normal and cancerous prostate tissues. In normal prostate cells, AIM1 was located along the outside border of each cell and paired up with a protein called beta-actin that helps form the cells cytoskeleton, or scaffolding. However, in prostate cancer cells, the protein spread away from the outer border of the cells and no longer paired up with beta-actin.

The scientists found this pattern among a set of human prostate tissue samples including 81 normal prostates, 87 localized prostate cancers and 52 prostate cancers that had spread to the lymph nodes.

It appears that when AIM1 protein levels drop, or when its abnormally spread throughout the cell instead of confined to the outer border, the prostate cancer cells scaffolding becomes more malleable and capable of invading other tissues, said Vasan Yegnasubramanian, M.D., Ph.D., associate professor at the Kimmel Cancer Center and a member of the research team. With AIM1, the scaffold, Yegnasubramanian says, keeps normal cells in a rigid, orderly structure. Without AIM1, cells become more malleable, shapeshifting nomads that can migrate to other parts of the body, he said.

To track how these shapeshifting cancer cells move, the Johns Hopkins scientists, with Steven An, Ph.D., an expert in cellular mechanics and an associate professor at the Johns Hopkins Bloomberg School of Public Health, took a close-up look at AIM1-lacking prostate cancer cells, using sophisticated and quantitative single-cell analyses designed to probe the material and physical properties of the living cell and its cytoskeleton.

They found that cells lacking AIM1 remodeled their scaffolding more than twice as much as cells that had normal levels of AIM1, and that they exert three- to fourfold more force on their surroundings than cells with normal levels of the protein. Such cellular properties are reminiscent of cells with high potential to invade and migrate, An noted.

In addition, the scientists found that AIM1-lacking prostate cells were capable of migrating to unoccupied spaces on a culture dish or invading through connective tissue-like materials at rates fourfold higher than cells with normal levels of AIM1.

Next, the scientific team implanted human prostate cancer cells engineered without AIM1 in five mice and found that the cells spread to other tissues at levels 10 to 100 times more than cells with normal levels of AIM1 that were implanted in five similar mice. However, the AIM1-lacking cells were not able to establish full colonies and tumors at those other tissues, suggesting that AIM1 depletion is not the whole story in the spread and growth of metastatic prostate cancer.

AIM1 may help prostate cancer cells disseminate throughout the body, but something else may be helping them form full-blown metastatic tumors when they get there, said Yegnasubramanian.

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Prostate Cancer Cells Become 'Shapeshifters' to Spread to Distant Organs - Laboratory Equipment

VistaGen Therapeutics (VTGN) Receives Notice of Allowance For Methods for Producing Blood Cells, Platelets and … – StreetInsider.com

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VistaGen Therapeutics Inc. (NASDAQ: VTGN), a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders, announced today that the Company has received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for U.S. Patent Application No. 14/359,517 regarding proprietary methods for producing hematopoietic precursor stem cells, which are stem cells that give rise to all of the blood cells and most of the bone marrow cells in the body, with potential to impact both direct and supportive therapy for autoimmune disorders and cancer.

The breakthrough technology covered by the allowed U.S. patent was discovered and developed by distinguished stem cell researcher, Dr. Gordon Keller, Director of the UHN's McEwen Centre for Regenerative Medicine in Toronto, one of the world's leading centers for stem cell and regenerative medicine research and part of the University Health Network (UHN), Canada's largest research hospital. Dr. Keller is a co-founder of VistaGen and a member of the Company's Scientific Advisory Board. VistaGen holds an exclusive worldwide license from UHN to the stem cell technology covered by the allowed U.S. patent.

"We are pleased to report that the USPTO has allowed another important U.S. patent relating to our stem cell technology platform, stated Shawn Singh, Chief Executive Officer of VistaGen. "Because the technology under this allowed patent involves the stem cells from which all blood cells are derived, it has the potential to reach the lives of millions battling a broad range of life-threatening medical conditions, including cancer, with CAR-T cell applications and foundational technology we believe ultimately will provide approaches for producing bone marrow stem cells for bone marrow transfusions. As we continue to expand the patent portfolio of VistaStem Therapeutics, our stem cell technology-focused subsidiary, we enhance our potential opportunities for additional regenerative medicine transactions similar to our December 2016 sublicense of cardiac stem cell technology to BlueRock Therapeutics, while focusing VistaStem's internal efforts on using stem cell technology for cost-efficient small molecule drug rescue to expand our drug development pipeline."

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VistaGen Therapeutics (VTGN) Receives Notice of Allowance For Methods for Producing Blood Cells, Platelets and ... - StreetInsider.com