Spurred by promising clinical results in an important trial, each of the three major CRISPR stocks had a great performance in the second half of 2019. Unfortunately, they didn't keep the momentum going in the first month of 2020.

Shares of Intellia Therapeutics (NASDAQ:NTLA) fell 18.8% in January, according to data provided by S&P Global Market Intelligence. That was followed by a 14.7% loss for shares of CRISPR Therapeutics (NASDAQ:CRSP) and a 10.7% tumble for shares of Editas Medicine (NASDAQ:EDIT).

While each has recovered some ground in the first week of February, this trio of pharma stocks is no stranger to volatility. Investors should probably expect that to continue as clinical programs advance in 2020.

Image source: Getty Images.

In November, CRISPR Therapeutics reported data for the first two individuals in the trial, one with sickle cell disease (SCD) and one with transfusion-dependent beta thalassemia (TDT), treated with its lead drug candidate CTX001. Both enjoyed significant benefits in their standard of living, which investors interpreted as a sign that CRISPR gene editing might actually live up to the hype.

That fueled annual gains of 113% for CRISPR Therapeutics last year. While Editas Medicine and Intellia Therapeutics gained only 30% and 7%, respectively, each had been sitting at a year-to-date loss in October.

What relevance does that have for the tumbles taken in January? First, it's not unusual for stocks to regress to the mean. Stocks that are red hot eventually cool off, while those that tumble without good reason eventually recover some ground.

Second, and the more important consideration for investors, is that the early stage results for CTX001 mean relatively little for the industry's pipeline of CRISPR-based gene editing drug candidates.

Consider that CTX001 is an ex vivo tool. Researchers harvest bone marrow from patients, extract specific types of stem cells, and engineer those with CTX001. The engineered stem cells are then grown in the lab before being reinjected into the patient.

Many other CRISPR-based drug candidates are designed as in vivo tools. That means the gene editing payloads are designed to engineer a patient's DNA while inside the body. An in vivo approach is inherently more complex and will be more difficult to control compared to an ex vivo approach.

Put another way, investors cannot take the promising, early stage results from CTX001 and extrapolate it broadly across all first-generation CRISPR tools. Wall Street certainly isn't, if the correlation between technical approach and stock performance is any guide.

Consider that the two most advanced drug candidates from CRISPR Therapeutics rely on ex vivo engineering. By contrast, the lead drug candidate from Editas Medicine relies on in vivo methods.

The lead pipeline asset from Intellia Therapeutics is also an in vivo tool, though unlike the lead assets from its peers, it has yet to advance to clinical trials.

Investors should expect 2020 to be a busy year for these CRISPR stocks. CRISPR Therapeutics will have more clinical data from CTX001 and the first set of data for its lead oncology asset CTX110.

Similarly, Editas Medicine should have results for EDIT101 and progress additional assets, while Intellia Therapeutics is preparing to finally enter the clinic with NTLA-2001 in the second half of the year.

Investors cannot know if the next batch of results will be as rosy as the initial data for CTX001, but they can probably expect another year of volatile stock movements.

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Here's Why CRISPR Stocks Fell in January - The Motley Fool

WARSAW (AFP) Submerged in liquid nitrogen vapour at a temperature of minus 175 degrees Celsius, hundreds of thousands of stem cells from all over Europe bide their time in large steel barrels on the outskirts of Warsaw.

Present in blood drawn from the umbilical cord of a newborn baby, stem cells can help cure serious blood-related illnesses like leukaemia and lymphoma, as well as genetic conditions and immune system deficits.

Polish umbilical cord blood bank PBKM/FamiCord became the industrys leader in Europe after Swiss firm Cryo-Save went bankrupt early last year.

It is also the fifth largest in the world, according to its management, after two companies in the United States (US), a Chinese firm and one based in Singapore.

Since the first cord blood transplant was performed in France in 1988, the sector has significantly progressed, fuelling hopes.

Mum-of-two Teresa Przeborowska has firsthand experience.

At five-years-old, her son Michal was diagnosed with lymphoblastic leukaemia and needed a bone marrow transplant, the entrepreneur from northern Poland said.

The most compatible donor was his younger sister, Magdalena.

When she was born, her parents had a bag of her cord blood stored at PBKM.

More than three years later, doctors injected his sisters stem cells into Michals bloodstream. It was not quite enough for Michals needs but nicely supplemented harvested bone marrow.

As a result, Michal, who is nine, is now flourishing, both intellectually and physically, his mum told AFP.

A cord blood transplant has become an alternative to a bone marrow transplant when there is no donor available, with a lower risk of complications.

Stem cells taken from umbilical cord blood are like those taken from bone marrow, capable of producing all blood cells: red cells, platelets and immune system cells.

When used, stem cells are first concentrated, then injected into the patient. Once transfused, they produce new cells of every kind.

At the PBKM laboratory, each container holds up to 10,000 blood bags. Safe and secure, they wait to be used in the future, its Head Krzysztof Machaj, said.

The bank holds around 440,000 samples, not including those from Cryo-Save, he said.

If the need arises, the blood will be ready to use without the whole process of looking for a compatible donor and running blood tests, the biologist told AFP.

For families who have paid an initial nearly EUR600 (USD675) and then an annual EUR120 euros to have the blood taken from their newborns umbilical cords preserved for around 20 years, it is a kind of health insurance promising faster and more effective treatment if illness strikes.

But researchers also warn against unrealistic expectations.

Bone marrow pioneer in Poland Haematologist Wieslaw Jedrzejczak describes promoters of the treatment as sellers of hope, who make promises that are either impossible to realise in the near future or downright impossible to realise at all for biological reasons.

He compares them to makers of beauty products who swear their cream will rejuvenate the client by 20 years.

Various researches is being done on the possibility of using the stem cells to treat other diseases, notably nervous disorders. But the EuroStemCell scientist network warns that the research is not yet conclusive.

There is a list of almost 80 diseases for which stem cells could prove beneficial, US Haematologist Roger Mrowiec, who heads the clinical laboratory of the cord blood programme Vitalant in New Jersey, told AFP.

But given the present state of medicine, they are effective only for around a dozen of them, like leukaemia or cerebral palsy, he said.

Its not true, as its written sometimes, that we can already use them to fight Parkinsons disease or Alzheimers disease or diabetes.

EuroStemCell also cautions against private blood banks that advertise services to parents suggesting they should pay to freeze their childs cord blood in case its needed later in life.

Studies show it is highly unlikely that the cord blood will ever be used for their child, the network said.

It also pointed out that there could be a risk of the childs cells not being useable anyway without reintroducing the same illness.

Some countries, such as Belgium and France, are cautious and ban the storage of cord blood for private purposes. Most European Union (EU) countries however permit it while imposing strict controls.

In the early 2000s, Swiss company Cryo-Save enjoyed rapid growth.

Greeks, Hungarians, Italians, Spaniards and Swiss stored blood from their newborns with the company for 20 years on payment of UER2,500 euros upfront.

When the firm was forced to close in early 2019, clients were left wondering where their stem cells would end up.

Under a kind of back-up agreement, the samples of some 250,000 European families were transferred for storage at PBKM.

The Polish firm, founded in 2002 with PLN2million (around EUR450,000, USD525,000), has also grown quickly.

Present under the FamiCord brand in several countries, PBKM has some 35 per cent of the European market, excluding Cryo-Save assets.

Over the last 15 months, outside investors have contributed EUR63 million to the firm, PBKMs Chief Executive Jakub Baran told AFP.

But the company has not escaped controversy: the Polityka weekly recently published a critical investigative report on several private clinics that offer what was described as expensive treatment involving stem cells held by PBKM.

Read more:
Europe's guardian of stem cells and hopes, real and unrealistic - Borneo Bulletin Online

"Grand View Research, Inc. - Market Research And Consulting."

According to report published by Grand View Research, the global cell harvesting systems market size was valued USD 3.17 billion in 2016 and is expected to grow at a CAGR of 13.9% over the forecast period.

The globalCell Harvesting System Marketis expected to reach USD 10.17 billion by 2025, according to a new report by Grand View Research, Inc. The increasing demand of stem cellbased therapies, owing to the growing base of aging population and increasing prevalence of chronic diseases, is one of the major factors contributing toward lucrative market growth.

Growing investment on stem cell research is one of the high impact rendering drivers contributing to the demand of stem cells, which thereby contributes to growth of cell harvesting system market. There has been a significant rise in stem cell transplantation rate globally, which is another major driver for increasing demand across the globe. Growth in autologous stem cell transplantation along with increasing stem cell banking is stimulating demand of cell harvesting system.

The potential use of stem cells in regenerative medicine, such as in case of cancer, trauma, congenital diseases, etc., is also one of the factors contributing to the demand for stem cells for research, thereby contributing toward growth of cell harvesting system market across the globe. The rising prevalence of certain diseases such as cancer is expected to drive the growth of this market over the forecast period.

Request a Sample Copy of the Global Cell Harvesting System Market Research Report@ https://www.grandviewresearch.com/industry-analysis/cell-harvesting-systems-market/request/rs1

Further Key Findings From the Report:

Have Any Query? Ask Our Experts@ https://www.grandviewresearch.com/inquiry/3739/ibb

Grand View Research has segmented the global cell harvesting system market on the basis of product, application, end use, and region:

Cell Harvesting System Application Outlook (Revenue, USD Million, 2014 - 2025)

Cell Harvesting System End-use Outlook (Revenue, USD Million, 2014 - 2025)

Cell Harvesting System Regional Outlook (Revenue, USD Million, 2014 - 2025)

Browse Related Reports @

Cell Sorting Market: https://www.grandviewresearch.com/industry-analysis/cell-sorting-market

Cell Therapy Market: https://www.grandviewresearch.com/industry-analysis/cell-therapy-market

About Grand View Research

Grand View Research provides syndicated as well as customized research reports and consulting services on 46 industries across 25 major countries worldwide. This U.S.-based market research and consulting company is registered in California and headquartered in San Francisco. Comprising over 425 analysts and consultants, the company adds 1200+ market research reports to its extensive database each year. Supported by an interactive market intelligence platform, the team at Grand View Research guides Fortune 500 companies and prominent academic institutes in comprehending the global and regional business environment and carefully identifying future opportunities.

Media ContactCompany Name: Grand View Research, Inc.Contact Person: Sherry James, Corporate Sales Specialist - U.S.A.Email: Send EmailPhone: 1-415-349-0058, Toll Free: 1-888-202-9519Address:201, Spear Street, 1100 City: San FranciscoState: CaliforniaCountry: United StatesWebsite: https://www.grandviewresearch.com/industry-analysis/cell-harvesting-systems-market

Excerpt from:
Cell Harvesting System Market Size is Estimated to Attain $10.17 Billion By 2025: Grand View Research, Inc - Press Release - Digital Journal

NEW YORK, Feb. 5, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc.(NYSE AMERICAN: ATNM)("Actinium") today announced that Sandesh Seth, Actinium's Chairman & CEO, will be presenting at the 22nd Annual BIO CEO & Investor Conference. Hosted by the Biotechnology Innovation Organization (BIO), the 22nd Annual BIO CEO & Investor Conference will take place February 10th and 11th at the New York Marriott Marquis in New York City.

Presentation Details

Date:Tuesday, February 11Time:10:15 am ETPresenter:Sandesh Seth, Chairman and CEOLocation:New York Marriott Marquis, Ziegfeld Room

Members of Actinium's Executive team will be available for one-on-one meetings with conference attendees. Those interested in scheduling a meeting with Actinium may do so by contacting Steve O'Loughlin, Principal Financial Officer via email at soloughlin@actiniumpharma.com.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, apamistamab-I-131 (Iomab-B) is being studied in the ongoing pivotal Phase 3Study ofIomab-B inElderlyRelapsed orRefractoryAcute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and promising single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. Apatmistamamb-I-131 will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc.

More information is available at http://www.actiniumpharma.com, http://www.sierratrial.com and our Twitter feed @ActiniumPharma, http://www.twitter.com/actiniumpharma.

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans VitzthumLifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

View original content to download multimedia:http://www.prnewswire.com/news-releases/actinium-pharmaceuticals-inc-to-present-at-the-22nd-annual-bio-ceo--investor-conference-300999431.html

SOURCE Actinium Pharmaceuticals, Inc.

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Actinium Pharmaceuticals, Inc. to Present at the 22nd Annual BIO CEO & Investor Conference - BioSpace

Cancer | One of the most common disease in the world

Cancer is the second leading cause of death globally which has caused over nine million deaths in 2018. As per the World Health Organization (WHO), one in 5 men and one in 6 women worldwide develop cancer during their lifetime, and one in 8 men and one in 11 women die from the disease.

But recently the first science minister of Colombia claimed that she has created a fungus extract drink which can treat cancer. After this unproven claim became public, it caused massive chaos which led to the resignation appeal to the minister.

Unproven claims by Mabel Gisela Torres

It was in 2019 December when Colombian President Ivn Duque Mrquez appointed molecular biologist Mabel Gisela Torres to be the first head of the newly created Ministry of Science, Technology and Innovation. Only a few nations knew who she was and Gustavo Quintero Hernndez, the dean of the School of Medicine and Health Sciences at Del Rosario University mentioned her as a "total stranger," after she took over the newly formed ministry.

But at this current moment, she is one of the well-known and most debated person as Torres, who is an expert in fungi from the Technological University of El Choc in Quibd who used an extract from a member of the Ganoderma family of mushrooms to create a tea-like drink to treat 40 people with cancer.

It should be mentioned that the Colombian minister had published 21 scientific articles, largely on a type of mushroom used in traditional Chinese medicine and claimed to have created cancer remedy. But claiming that she found a cancer curing fungus extract made her receive calls to resign. She claimed that already 40 patients took the medicine and some of them went into remission after consuming the potion.

Controversy over cancer treatment

After making such claims, the scientists asked her why she did not follow the widely considered steps to develop the medication. It was also revealed that her finding was not published in any peer-reviewed scientific journal. She neither offered a clinical trial for this cancer remedy nor consulted with an ethics committee, which caused fear that it was an unproven treatment method and that she might be putting the lives of her patients in danger.

But in an interview with Colombia's El Espectador newspaper, Torres defended treatment and said that some traditional treatments have a scientific basis, but the knowledge is not presented in the standard way. In addition, she mentioned that Ganoderma had previously passed toxicity tests in Asia more than 2,000 years ago.

As per Torres, she was looking for potential treatment and a male patient who took the potion was cancer-free after three months. But again, she could not provide any evidence to back her claims. She also compared the treatment with something as safe as having "mango juice at home."

Cancer treatment by Torres

During the interview, she was asked why did she give the patients medically unproven cancer treatment despite not carrying out pre-clinical or clinical studies, or publishing her findings. In response, she mentioned, "I wanted to do an act of rebellion and that is to say: I have the possibility to help people."

However, as per the new report by Nature, Torres would release her data, while the government confirmed that Torres would stay in office.

She said, "At no time have I simplistically proposed that this species [could] be the cure for cancer. I haven't offered a drug, let alone market it. I have rigorously observed the established ethical protocols for scientific experimentation in general and those that apply specifically in my disciplinary field."

Cancer treatment

The usual cancer treatment options include:

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Claims of Colombia science minister to cure cancer with her innovative fungus drink leave scientists shell-shocked - International Business Times,...

Electron micrograph showing ridges and grooves on MAP hydrogel microbeads caused by developing stem cells.

Courtsey of Daniel Alge

Baby diapers, contact lenses and gelatin dessert. While seemingly unrelated, these items have one thing in common theyre made of highly absorbent substances called hydrogels that have versatile applications. Recently, a type of biodegradable hydrogel, dubbed microporous annealed particle (MAP) hydrogel, has gained much attention for its potential to deliver stem cells for body tissue repair. But it is currently unclear how these jelly-like materials affect the growth of their precious cellular cargo, thereby limiting its use in regenerative medicine.

In a new study published in the November issue of Acta Biomaterialia, researchers at Texas A&M University have shown that MAP hydrogels, programmed to biodegrade at an optimum pace, create a fertile environment for bone stem cells to thrive and proliferate vigorously. They found the space created by the withering of MAP hydrogels creates room for the stem cells to grow, spread and form intricate cellular networks.

Our research now shows that stem cells flourish on degrading MAP hydrogels; they also remodel their local environment to better suit their needs, said Daniel Alge, assistant professor in the Department of Biomedical Engineering. These results have important implications for developing MAP hydrogel-based delivery systems, particularly for regenerative medicine where we want to deliver cells that will replace damaged tissues with new and healthy ones.

MAP hydrogels are a newer breed of injectable hydrogels. These soft materials are interconnected chains of extremely small beads made of polyethylene glycol, a synthetic polymer. Although the microbeads cannot themselves cling to cells, they can be engineered to present cell-binding proteins that can then attach to receptor molecules on the stem cells surface.

Once fastened onto the microbeads, the stem cells use the space between the spheres to grow and transform into specialized cells, like bone or skin cells. And so, when there is an injury, MAP hydrogels can be used to deliver these new cells to help tissues regenerate.

However, the health and behavior of stem cells within the MAP hydrogel environment has never been fully studied.

MAP hydrogels have superior mechanical and biocompatible properties, so in principle, they are a great platform to grow and maintain stem cells, Alge said. But people in the field really dont have a good understanding of how stem cells behave in these materials.

To address this question, the researchers studied the growth, spread and function of bone stem cells in MAP hydrogels. Alge and his team used three samples of MAP hydrogels that differed only in the speed at which they degraded, that is, either slow, fast or not at all.

First, for the stem cells to attach onto the MAP hydrogels, the researchers decorated the MAP hydrogels with a type of cell-binding protein. They then tracked the stem cells as they grew using a high-resolution, fluorescent microscope. The researchers also repeated the same experiment using another cell-binding protein to investigate if cell-binding proteins also affected stem cell development within the hydrogels.

To their surprise, Alges team found that for both types of cell-binding proteins, the MAP hydrogels that degraded the fastest had the largest population of stem cells. Furthermore, the cells were changing the shape of the MAP hydrogel as they spread and claimed more territory.

In the intact MAP hydrogel, we could still see the spherical microbeads and the material was quite undamaged, Alge said. By contrast, the cells were making ridges and grooves in the degrading MAP hydrogels, dynamically remodeling their environment.

The researchers also found that as the stem cells grew, the quantity of bone proteins produced by the growing stem cells depended on which cell-binding protein was initially used in the MAP hydrogel.

Alge noted that the insight gained through their study will greatly inform further research and development in MAP hydrogels for stem-cell therapies.

Although MAP hydrogel degradability profoundly affects the growth of the stem cells, we found that the interplay between the cell-binding proteins and the degradation is also important, he said. As we, as a field, make strides toward developing new MAP hydrogels for tissue engineering, we must look at the effects of both degradability and cell-binding proteins to best utilize these materials for regenerative medicine.

Other contributors to the research include Shangjing Xin from the Department of Biomedical Engineering at Texas A&M and Carl A. Gregory from the Institute for Regenerative Medicine at the Texas A&M Health Science Center.

This research was supported by funds from theNational Institute of Arthritis and Musculoskeletal and Skin Diseasesof the National Institutes of Health.

See original here:
Researchers Explore Hydrogels That Are Promising Materials For Delivering Therapeutic Cells - Texas A&M University

Background

Today's biologic quarry is the hair follicle, depicted on the left. While we all take hair for granted (perhaps the lyric "you don't know what you got till it's gone" is applicable), it is a complex structure lying within the skin. Stem cells are responsible for its growth and coloration, more about them shortly, and the milieu of hormones, as well as cell mediators, controls where hair grows along with its texture. In addition to housing a sebaceous gland, hair follicles have muscles, the arrector pili, responsible for raising your hair, as in the phrase "hair-raising scare," which is a real thing.

Hair follicles go through stages of growth (anagen), rest (telogen), loss (catagen and exogen), as well as renewal (kenogen). The bulbous area at the bottom of the follicle contains those previously mentioned stem cells. Hair follicle stem cells (HFSC) derived embryologically from the same layer as our skin produces a new hair follicle. In contrast, melanocyte stem cells (MeSC) derived from the layer that forms our nervous system, provides pigment to color the hair. During anagen, these stem cells are active; otherwise, they just hang out.

The Study

Looking at the pictures of our recent Presidents over two-terms suggests that stress is involved. But how? To find an answer, the researchers made use of black-coated mice, in a series of increasingly focused experiments.

So yes, my children's antics did turn my hair grey, a hair color I have earned, but not directly. It required the assistance of my sympathetic nervous system. And it may explain why some people under stress retain their hair color; they may well have more of the melanocyte stem cells to burn through. It also points towards the belief that our autonomic nervous system, of which the sympathetics are one component, has a role in cell differentiation and tissue maintenance. Perhaps being more chill can adds days to your life as well as less grey in your hair.

[1] I attribute my hair loss, on the other hand, to my wife.

Source: Hyperactivation of sympathetic nerves drives depletion of melanocyte stem cells Nature DOI: 10.1038/s41586-020-1935-3

Image of hair follicle courtesy ofHelix84derivative work in Wikimedia

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Why Does Hair Turn Grey? - American Council on Science and Health

There are two stories I would like to tell with this edition of Business Unusual, the first is about the Darpa funded research to build robots out of living cells, the second is the incredible history of the animal that was used to build the first biological robots - Platannas.

The Defense Advanced Research Projects Agency (DARPA) is an agency of the American Department of Defense. It has funded many projects for military projects that in time have come to be used for civilian applications. The best-known example is the predecessor of the internet.

Why a military agency would fund research into creating living robots might be concerning but the stated objectives include managing environmental clean-ups or improving drug delivery which certainly are worthy pursuits. Of greater concern, are the ethical questions that are raised by creating new forms of a living organism. At the moment the designs dont attempt to make them self-replicating but that is part of the future plans.

Robots typically are designed and programmed to perform a specific task. Until now they would have been constructed out of non-living materials. These robots are also designed for a specific task but created from living cells. The choice of cell and the specific construction determines what action or function the living robot can perform.

One function that was attempted was movement. Starting from scratch researchers used stem cells from a frog to create skin cells and heart cells. The heart cells are muscles and so can contract while heart cells are able to do so rhythmically. Using those properties a machine learning program was tasked with testing thousands of configurations to determine which design would use the least cells to achieve the motion required. Once the best designs were determined, the living robots were constructed by researchers manipulating individual cells under a microscope.

The tiny constructed robots demonstrated that living robots designed by computer could offer an alternative to traditionally constructed machines. Future versions would look to make the constructions more complex and eventually able to self replicate.

One intended function was using a swarm of living robots with the ability to decompose plastic to be used to remove microplastics in the ocean. That may be a long way off, but if it is to become a reality the best time to start working on it is now.

Another application might be to not find plastic in the sea, but cancers in your body. Your body is already very good at doing so, but as we age and at certain times of our lives it becomes more challenging to correctly identify and kill cancer cells when they are still only tiny tumours.

This would require building robots consisting of your own body cells arranged in a way to allow them to move through the body and specifically find the corrupted cells. Adding them in numbers as we age may reduce the chance of developing tumours or even help the body recover after exposure to damaging external factors like sun damage to your skin.

This too is a long way off, but if successful and added to the many other options for extending and improving our lives then the research is most welcome.

_Image credit: Wikipedia African clawed frog_

Setting the other issues relating to building living robots aside, you might wonder why a frog from South Africa was chosen to build the first living robots.

It was not a random choice but points to a fascinating history that makes this particular frog one that has helped humanity overcome medical issues on a number of occasions.

A pregnancy test these days simply requires peeing on a stick. The reaction to a specific hormone in the urine can be isolated in minutes and let you know if you are pregnant within days of it occurring. It was not always this easy, the first method we are aware of would see a potentially pregnant woman urinate on ungerminated wheat and barley and wait a week or so to see if it germinated. Incredibly it works and was first mentioned over 3 000 years ago by the Egyptians. It was scientifically tested in the 1960s and found to be 70% accurate.

There were a variety of other methods used most on the expectation that something in the urine of females could be used to confirm pregnancy. In the 1920s it was injecting urine into female rabbits that after a day would require the examination of the rabbit ovaries. If swollen the woman was pregnant. In order to do the examination the rabbit was always killed and so the search continued for a better option.

Enter Lancelot Hogben, an English researcher lecturing in Cape Town in the early 1930s. He advised a student to consider using the local platanna as a potential for use as a model organism for biological tests. His hunch proved correct with Hillel Shapiro and Harry Zwarenstein creating the test to use the frog to indicate pregnancy.

The frog would be injected and in hours if the woman was pregnant would produce eggs. Not only was it accurate, but it also would not harm the frog which was easy to keep in a lab and would live for over a decade. As a result, the remarkable frog was exported around the globe and provided the answer to the question, am I pregnant, to the largest population explosion in our history. Most baby boomers parents and indeed many baby boomers would have found out if they were pregnant thanks to this strange-footed frog.

Xenopus literally means strange foot, frogs typically dont have claws which is why the African clawed frog got the name and as for Platanna, that may be a reference to the frog being very flat - plat in Afrikaans.

Given its widespread use for pregnancy and acceptance as a good species for embryonic development when researchers attempted to clone an organism, this frog was once again a key in understanding the process. In 1958, Xenopus was cloned not from splitting an embryonic cell which was the original method, but by using the DNA from an adult specialised cell which replaced the original DNA in a frog egg. The method proved successful and paved the way to allow Dolly the sheep to be cloned from an adult sheep cell in 1996.

We owe a huge debt of gratitude to six species that for a variety of reasons have helped us understand biological processes and how best to deal with disease and the efficacy of drugs. There are nematode worms, fruit flies, zebrafish, chickens, mice and the African clawed toad.

These six animals are our real guinea pigs.

Image credit: Xenobot - Tuft University & University of Vermont

Read more here:
Biological robots, that is a thing now - 702

Thankfully, as a society we are now better aware of the importance of good mental health. Self-care has become a hotly talked about topic in recent years, with the power of yoga, meditation and journaling at the forefront of many discussions. However, whilst these activities do help to keep a lot of people grounded and happy, they aren't for everyone. It's super important to discover what type of self-care works best for you. For some, this might be an evening in, spent playing on some great bingo sites, while for others it might be going swimming, or taking time to cook up some delicious and healthy meals. We are all different and have different things that make us feel happy and relaxed. But, there are certainly a handful of general and simplistic things that make most of us feel our best.

Cut Back on Social Media

Without a doubt, there are lots of great benefits to social media, but too much screen time can leave us feeling disheartened and pretty blue. The constant barrage of perfectly filtered photos that appear on Instagram are bound to knock many people's self-esteem whether we consciously realise it or not. It's actually really difficult to go online and not compare yourself to others, so whilst it's nice to now and again see what our friends, colleagues and various different celebs are up too, too much time spent looking into the online lives of others is surely going to get you down in the long run. It is also thought that time spent on social media before bed can prevent us from getting a good night's sleep, which is another very important factor contributing to our health and wellness. It is sadly easy to miss out on living truly in the moment because of the distractions that our screens create. Staying away from social media more often in 2020 is without a doubt a kind thing to do for ourselves.

Get Exercising

Exercise is hugely important for both the health of our bodies and our minds, but that doesn't mean you have to hit the gym for hours on end in order to be kinder to yourself. There are many different types of exercise out there to choose from, from competitive sport, to jogging, to walking, to horse riding, to pilates, or even to running around a giant assault course if you so choose. There's a type of exercise out there suitable for everyone and getting into the habit of regular exercise will help to boost your overall mood and decrease your stress levels.

Eat Well

Like exercise, eating a healthy and well-balanced diet is not only important for the body but also the mind. Being deficient in certain nutrients, like magnesium for example, can contribute to feelings of anxiety and depression. Whether you are vegan, vegetarian, follow a keto diet or eat a bit of everything, it's important to understand what nutrients are in certain foods and make sure you eat sensibly and include a varied range of food types. Ordering in takeaways too often and snacking on too many sweets, crisps and chocolate can all too quickly end up taking its toll on your mental state as well as your physical state. However, it's of course important to allow yourself to indulge every now and then and not be too strict with yourself. Really, it's all about moderation.

Read, Watch Greats Films, Listen to Music You Love

Sometimes when we get into a bit of a rut, we forget to indulge in down time. Spending an evening reading a great book or watching our favourite film can really help us to unwind and feel re-energised. Listening to music on the way to and from work can also help to boost your mood and leave you feeling empowered.

Meet Up with Friends and Family

Spending time with the people we love and care about is so important to our mental well-being. It's an opportunity to get any worries off your chest and have a good laugh. Shutting yourself away from people is never a good thing in the long-term. If you don't have many close friends, which isn't at all uncommon in this day and age, then you can easily meet people who share the same interests as you at various different evening classes and clubs.

Being kinder to yourself should always be a priority. A lot of us beat ourselves up for a range of silly and ridiculous things, and we don't put enough time into making ourselves feel great. 2020 is the year to stop being mean to yourself and start helping yourself to feel empowered and truly content in life.

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Alternative Funding Options To Get Your Dream Business Off The Ground - SWAAY

It happens whenever there are news stories about a new treatment claiming to cure paralysis.

I get flooded with messages from distant relatives, or people I went to high school with but haven't seen in 10 years.

"OMG YOU TOTALLY NEED TO DO THIS."

I'm not saying other people shouldn't try whatever treatments they want to, but for me, there's too little certainty and too many unknown factors.

After 15 years in a wheelchair, I've gained the perspective that walking, in fact, does not equate happiness.

I'll never forget the metaphor used to explain to me how one would repair a spinal cord, even if I was a little high on morphine when I heard it.

I was 16, laying in a hospital bed with four large screws drilled into my skull to stabilize my spine.

"Imagine squeezing all of the toothpaste out of a tube. Now try and get that toothpaste back into the tube without changing it's shape or structure. That's how fragile your spinal cord is."

Sounds impossible, right? Maybe. Or maybe the technology just hasn't been invented yet.

The thing no one tells you about having a spinal cord injury is that not walking is the easiest adjustment. You don't need to check your eyes. You read that right.

The human body and muscle memory are pretty adaptable. Using a wheelchair is the easy part.

First, there are societal stigmas. They could fill their own novel.

Every person with a disability has a few horror stories. Personally, I applied to more than 600 jobs before getting a part-time, entry-level position. Shout out to the YMCA of Saskatoon for giving me a shot when no one else would!

Another thing that doesn't often get talked about is the secondary health issues that come along with a spinal cord injury. Low blood pressure, autonomic dysreflexia, inability to regulate your body temperature, bowel and bladder problems, and pressure sores to name a few.

These are the really hard parts.

These secondary health issues are why I have no interest in an epidural stimulation implant or any other elective surgery of that nature. I know it's exciting to see someone moving their leg after an injury or walking while assisted, but the truth is we don't know what other unknown factors such treatments might present.

I've seen plenty of stories about possible "cures." When I was first injured it was stem cells, then embryonic stem cells, then there were the paralyzed rats learning to walk again.

Every few years there's a new procedure that makes a splash. Everyone is positive that this time, this procedure, this one is the cure. None have come to fruition.

Hope and optimism are vital, but there's a fine line between hope and false hope. I've seen far too many people unable to overcome the false hope and remain bitter and angry that they can't be "normal."

The thing is, moving my leg or even walking (although it would be cool) wouldn't change the functional quality of my life. I'm already independent. I'm already quite happy with my life. Gaining the ability to move a leg still wouldn't address those secondary health issues.

I'm not saying any of these potential treatments aren't amazing advancements in medicine, but if you can't guarantee me the ability to sweat so I don't overheat in our prairie summers, or control of my bowels or bladder, it's not worth the unknowns or cost at this point in my life.

There's no guarantee. I won't compromise for a maybe.

This column is part of CBC'sOpinionsection. For more information about this section, please read thiseditor's blogand ourFAQ.

Interested in writing for us? We accept pitches for opinion and point-of-view pieces from Saskatchewan residents who want to share their thoughts on the news of the day, issues affecting their community or who have a compelling personal story to share. No need to be a professional writer!

Read more about what we're looking for here, then emailsask-opinion-grp@cbc.cawith your idea.

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Yes, I've seen the stories about that new spinal treatment. Here's why I'm not interested - CBC.ca

Contrary to what you've probably (definitely) read on the internet, there is at least one benefit to the month with an average national contiguous temperature of 32 degrees. It is that you are automatically granted the excuse to send that "raincheck? lol" text any chilly evening you so choose, and instead snuggle up with your ugliest jogger sweatpants, a glass of Rioja, and brand-new skincare products. (It's called self-care, look it up.)

With the plethora of face creams, cleansers, serums, treatments, and oils hitting the market this February, however, it can be hard to decide which formulas are truly deserving of your Friday night. That's why we've asked our beauty editors to share their favorite at-home spa-day indulgences ahead, so you can stock up on the skincare products worth canceling all your plans for this month. (Well, at least until your friends start responding with the eye roll emoji.)

Link:
17 Brand-New Skincare Products Our Editors Are Using to the Very Last Drop This Month - POPSUGAR

News Release

Monday, December 16, 2019

NEI-led study to test safety of treatment for a form of age-related macular degeneration that currently lacks treatment.

Researchers at the National Eye Institute (NEI) are launching a clinical trial to test the safety of a novel patient-specific stem cell-based therapy to treat geographic atrophy, the advanced dry form of age-related macular degeneration (AMD), a leading cause of vision loss among people age 65 and older. The geographic atrophy form of AMD currently has no treatment.

The protocol, which prevented blindness in animal models, is the first clinical trial in the U.S. to use replacement tissues from patient-derived induced pluripotent stem cells (iPSC), said Kapil Bharti, Ph.D., a senior investigator and head of the NEI Ocular and Stem Cell Translational Research Section. The NEI is part of the National Institutes of Health.

The therapy involves taking a patients blood cells and, in a lab, converting them into iPS cells, which have the potential to form any type of cell in the body. The iPS cells are programmed to become retinal pigment epithelial (RPE) cells, the type of cell that dies early in the geographic atrophy stage of macular degeneration. RPE cells nurture photoreceptors, the light-sensing cells in the retina. In geographic atrophy, once RPE cells die, photoreceptors eventually also die, resulting in blindness. The therapy is an attempt to shore up the health of remaining photoreceptors by replacing dying RPE with iPSC-derived RPE.

Before they are transplanted, the iPSC-derived RPE are grown in sheets one cell thick, replicating their natural structure within the eye. This monolayer of iPSC-derived RPE is grown on a biodegradable scaffold designed to promote the integration of the cells within the retina. Surgeons position the patch between the RPE and the photoreceptors using a surgical tool designed specifically for that purpose.

Under the phase I/IIa clinical trial protocol 12 patients with advanced-stage geographic atrophy will receive the iPSC-derived RPE implant in one of their eyes and be closely monitored for a period of at least one year to confirm safety.

A concern with any stem cell-based therapy is its oncogenic potential: the ability for cells to multiply uncontrollably and form tumors. In animal models, the researchers genetically analyzed the iPSC-derived RPE cells and found no mutations linked to potential tumor growth.

Furthermore, the use of an individuals autologous (own) blood cells is expected to minimize the risk of the body rejecting the implant.

Should early safety be confirmed, later study phases will include more patients to assess the efficacy of the implant to prevent blindness and restore vision in patients with geographic atrophy.

A Food and Drug Administration (FDA) requirement for moving forward with the clinical trial was the establishment of good manufacturing practice (GMP) protocols to ensure that the iPSC-derived RPE are a clinical-grade product. GMP protocols are key for making the therapy reproducible and for scaling up production should the therapy receive FDA approval.

The preclinical research for the trial was supported by the NEI Intramural Research Program and by an NIH Common Fund Therapeutic Challenge Award. The trial is being conducted at the NIH Clinical Center in Bethesda, MD.

NEI leads the federal governments research on the visual system and eye diseases. NEI supports basic and clinical science programs to develop sight-saving treatments and address special needs of people with vision loss. For more information, visit https://www.nei.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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NIH launches first U.S. clinical trial of patient-derived ...

I get asked many questions about stem cell therapies, but one of the most common over the years has been about the stem cell treatment cost. For instance, a reporter might ask, How much does a stem cell treatment for MS cost? and a patient might ask me, How much is a fair cost for a stem cell therapy for arthritis? Or, patients will voluntarily tell me what they paid or mention it in the comments. We hear various numbers thrown around about costs so I decided to do a poll on this. I even did an early update on the results of this poll, voicing my skepticism that the costs paid were worth it.

But the poll has gotten well over 500 responses now so I thought I would revisit it and what it might mean.

You can see a screenshot of the images. Its fair to say, as much as Internet polls arent considered particularly accurate, that this one largely fits with what is reported out in the field.

(On a side note, I wish there was such a thing as going out into the field for stem cell scientists as Ive always been a bit jealous of scientists who really do go out in the field. What do we do, go out in the wild and catch wild or feral stem cells in the bush?)

Patients self-reported most often paying between $2,500 and $7,500 for their stem cell therapy so if we take the average of those we get that $5,000 figure that is what I hear most often from others. Yes, not necessarily very rigorous, but the result makes good sense. Not far behind though were responses in the $7,500-20,000 range.

About 1 in 10 respondents reported paying $20,000 or more, including some beyond $100,000. Thats a whopping stem cell treatment cost, especially for something most often unproven and unapproved by the FDA.

If we consider these responses, the average cost may be more like $7,500-$10,000.

Notably, about 1/16 respondents indicated their stem cells were free. Im not sure what that means in terms of how that came to be.

Interestingly, most respondents who also went on to answer a 2nd poll in that post about where they got the treatment indicate it was at a stem cell clinic (scroll down in that Oct. 2017 post and youll see the 2nd poll). This 2nd poll has about 200 responses.

So today buying a simple stem cell treatment, most often unproven and non-FDA approved, is often not so different in cost than buying a 10-year old used car, while less often it is similar to buy various new cars including at the high end of stem cell therapy cost, some very expensive new cars. This cost and the risks involved are why I have suggested to patients in the past to be assertive when considering a stem cell treatment, ask questions, dont just accept too good to be true kinds of answers, etc. In short, be at least (or ideally much more) rigorous about unproven stem cell treatments as you are about buying a car.

Related

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Update on stem cell treatment cost for 2018 from ongoing ...

Hyperbaric oxygen therapy (HBOT) can improve heart functionality in healthy aging humans, according to a study by the Sagol Center for Hyperbaric Medicine and Research at Shamir Medical Center in Beer Yaacov.In this study, director of the Sagol Center for Hyperbaric Medicine and Research at Shamir Medical Center Prof. Shai Efrati and Dr. Marina Leitman, head of the Echocardiography Unit and Noninvasive Cardiology Service at Shamir Medical Center, turned their attention to HBOTs impact on cardiac function.According to the center, the study of HBOT for cardiac function has been limited, mostly evaluating patients during and after short-term exposures. However, for the first time, the study was conducted in humans and it demonstrated that repetitive HBOT protocols have a sustained effect on heart function.Healthy patients receiving HBOT to improve cognitive function underwent a 60-session treatment course using the Sagol Centers regenerative HBOT protocols. Using a high-resolution echocardiography, 31 patients were evaluated before HBOT was administered and three weeks after treatment concluded to identify the sustained effect of the treatment.HBOT includes the inhalation of 100% oxygen at pressures exceeding one atmosphere absolute [ATA], which is the average atmospheric pressure exerted at sea level, in order to increase the amount of oxygen dissolved in the body tissues, Efrati told The Jerusalem Post.Efrati, who has been pioneering new approaches for the application of HBOT treatments that specifically focus on HBOTs ability to trigger regeneration in the body, said that in the past HBOT was used mostly to treat chronic non-healing wounds.In recent years, there is growing evidence on the regenerative effects of HBOT, he said. We have now realized that the combined action of both hyperoxia (an excess of oxygen in the body) and hyperbaric pressure, leads to significant improvement in tissue oxygenation while targeting both oxygen and pressure sensitive genes, resulting in improved mitochondrial metabolism with anti-apoptotic (anti-cell death) and anti-inflammatory effects.According to Efrati, the newly developed protocols used in this study, which includes the intermittent increasing and decreasing of oxygen concentration, induces what is known as the Hyperoxic Hypoxic Paradox.This, he said induces stem cells proliferation and mobilization, leading to the generation of new blood vessels (angiogenesis) and tissue regeneration.Efrati said that during the first studies they conducted at the Sagol Center, they evaluated the beneficial effects of HBOT in treating traumatic brain injury and stroke. However, in this study we evaluated for the first time the effect of these new regenerative HBOT protocols on the normal aging heart. For the first time in humans we have demonstrated that HBOT can improve cardiac function.Efrati said for the last 12 years his team has developed an ongoing research program that investigates the regenerative effects of HBOT on different issues and degrees of damage. At the beginning we were focused on non-healing peripheral wounds. Then, we turned our focus to certain types of brain injuries.However, once the researchers found that HBOT induced many of the essential elements crucial to repairing almost any mechanism, we initiated a complementary research program that targets other organs such as the heart and other elements related to expected age-related functional decline.Along with normal aging, there is typically a decrease in cardiac function particularly in the mitochondrial cells of the heart, Efrati said.The mitochondria are the powerhouse of the cell [and] this is where we create energy, he said. HBOTs ability to improve mitochondrial function may explain the beneficial effects that we saw in the cardiac function of this normal aging population.By exposing the mitochondria to the fluctuations in oxygen by the use of HBOT, the team observed an improvement in contractility function of the heart meaning, the heart muscle contracted more efficiency over the course of the 60-session protocol.Efrati said the effect was particularly evident in the left ventricle, which is the chamber responsible for pumping oxygenated blood to the rest of the body.This is only the beginning of our understanding of the impact of HBOT on cardiac function in a normally aging population, and a larger and more diverse cohort will be required to further evaluate our initial findings, he said.Asked whether this treatment could also be used on people who are predisposed to heart conditions, Efrati said the short answer is yes, but he stressed that more research is needed.As far as we know, we are the first to identify HBOTs ability to improve cardiac function, Efrati said. Our study was on a group of 31 asymptomatic normal aging heart patients.We believe it is important to expand the scope of this study to a larger group, with both symptomatic and asymptomatic patients to understand the possibilities for HBOT as a treatment for patients with heart-related diseases, he said.The Sagol Center has also been studying the impact of HBOT on a variety of cognitive conditions.We have also conducted studies which showed positive results for the treatment of post-concussion syndrome as a result of traumatic brain injury, post-stroke recovery, fibromyalgia, Efrati said, adding that today, medical professionals understand that fibromyalgia is linked to issues in the brain center responsible for pain interpretation.Not every patient will benefit from HBOT, which is why patient selection should be done very carefully based on the damage seen in brain imaging assessments, he said.For example, if someone has a stroke, some of the tissue at the core of the stroke will die we will not be able to recover this tissue, Efrati said. But, other tissue that is damaged but not fully dead... is where HBOT can help.This damaged tissue, known as the metabolic dysfunction tissue (penumbra), is where we can have an impact and help recover lost function, he said.On the time line as to when using HBOT protocols may be put into effect on healthy aging patients in Israel, Efrati said these studies are already ongoing.I cant speak too much about this, as we are in the process of developing the results of the first study for publication, he said. However, we believe HBOT can positively impact both cognitive and physical performance in aging adults based on what we have seen at this point.Efrati said they will continue pursuing this line of research as it has the ability to transform how we look at aging.A number of research collaborations are ongoing, including research on cognitive decline, fibromyalgia and PTSD, he said.In addition, we have an ongoing research program on athletic performance both in professional and amateur level athletes, which looks at how HBOT may further improve performance, he said. Finally, we are studying the impact of HBOT on healthy aging adults to understand how HBOT may improve our health and cognitive performance as we age.When you look at aging as a disease that can be measured, then it can be treated, and this is a serious area of investigation for us, Efrati said.The study, led by Dr. Marina Leitman, Dr. Shmuel Fuchs, Dr. Amir Hadanny, Dr. Zvi Vered and Efrati, was published in the International Journal of Cardiovascular Imaging.

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Hyperbaric oxygen therapy can improve cardiac function in healthy, aging - The Jerusalem Post

RESEARCH TRIANGLE PARK, N.C. , Feb. 04, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical (AskBio), a clinical-stage adeno-associated virus (AAV) gene therapy company, and its NanoCor Therapeutics subsidiary today announced that the first patient has been dosed in a Phase 1 clinical trial of NAN-101. NAN-101 is a gene therapy that aims to activate protein phosphatase inhibitor 1 (I-1c) to inhibit the activity of protein phosphatase 1 (PP1), a substance that plays an important role in the development of heart failure.

Congestive heart failure (CHF) is a condition in which the heart is unable to supply sufficient blood and oxygen to the body and can result from conditions that weaken the heart muscle, cause stiffening of the heart muscles, or increase oxygen demand by the body tissues beyond the hearts capability.

"Dosing the first patient using gene therapy to target I-1c to improve heart function is a tremendous milestone not only for the AskBio and NanoCor teams but, more importantly, for patients whose quality of life is negatively affected by CHF, said Jude Samulski, PhD, Chief Scientific Officer and co-founder of AskBio. We initially developed this gene therapy as treatment for late-stage Duchenne muscular dystrophy patients who typically die from cardiomyopathy. Following preclinical studies, we observed that heart function improved, which led us to investigate treatment for all types of heart failure.

Were excited to be involved in this novel approach for patients with Class III heart failure, said Timothy Henry, MD, FACC, MSCAI, Lindner Family Distinguished Chair in Clinical Research and Medical Director of The Carl and Edyth Lindner Center for Research at The Christ Hospital in Cincinnati, Ohio, and principal investigator for the study. These patients currently have no other options besides transplant and left ventricular assist devices (LVAD). Today, we started to explore the potential of gene therapy to change their outcomes.

Heart disease is the leading cause of death worldwide, with CHF affecting an estimated 1% of the Western world, including over six million Americans. There is no cure, and medications and surgical treatments only seek to relieve symptoms and slow further damage.

Research by many investigators around the world has been trying to understand what exactly goes wrong in the heart and weakens its pumping activity until it finally fails, said Evangelia (Litsa) Kranias, PhD, FAHA, Hanna Professor, Distinguished University Research Professor and Director of Cardiovascular Biology at the University of Cincinnati College of Medicine. The aim has been to identify potential therapeutic targets to restore function or prevent further deterioration of the failing heart. Along these lines, research on the role of I-1c started over two decades ago, and it moved from the lab bench to small and large animal models of heart failure. The therapeutic benefits at all levels were impressive. It is thrilling to see I-1c moving into clinical trials with the hope that it also improves heart function in patients with CHF.

About the NAN-101 Clinical Trial NAN-CS101 is a Phase 1 open-label, dose-escalation trial of NAN-101 in subjects with NYHA Class III heart failure. NAN-101 is administered directly to the heart via an intracoronary infusion by cardiac catheterization in a process similar to coronary angioplasty, commonly used to deliver treatments such as stem cells to patients with heart disease. The primary objective of the study is to assess the safety of NAN-101 for the treatment of NYHA Class III heart failure, as well as assess the impact of this treatment on patient health as measured by changes in exercise capacity, heart function and other factors including quality of life.

AskBio is actively enrolling patients with NYHA Class III heart failure to assess three doses of NAN-101. Please refer to clinicaltrials.gov for additional clinical trial information.

Would you like to receive our AskFirst patient engagement program newsletter? Sign up at https://www.askbio.com/patient-advocacy.

About The Christ Hospital Health Network The Christ Hospital Health Network is an acute care hospital located in Mt. Auburn with six ambulatory centers and dozens of offices conveniently located throughout the region. More than 1,200 talented physicians and 6,100 dedicated employees support the Network. Its mission is to improve the health of the community and to create patient value by providing exceptional outcomes, the finest experiences, all in an affordable way. The Network has been recognized by Forbes Magazine as the 24th best large employer in the nation in the magazines Americas 500 Best Large Employers listing and by National Consumer Research as the regions Most Preferred Hospital for more than 22 consecutive years. The Network is dedicated to transforming care by delivering integrated, personalized healthcare through its comprehensive, multi-specialty physician network. The Christ Hospital is among only eight percent of hospitals in the nation to be awarded Magnet recognition for nursing excellence and among the top five percent of hospitals in the country for patient satisfaction. For more than 125 years, The Christ Hospital has provided compassionate care to those it serves.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process called Pro10 and an extensive AAV capsid and promoter library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy type 2i/R9 and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). For more information, visit https://www.askbio.com or follow us on LinkedIn.

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AskBio Announces First Patient Dosed in Phase 1 Trial Using AAV Gene Therapy for Congestive Heart Failure - BioSpace

When the tech VCs at Andreessen Horowitz entered biotech 4.5 years ago with the $200 million bio fund I, the idea was simple and hubristic: Were not going to do biotech, Vijay Pande said at the time, keeping a16zs longtime stance. Instead, the bio fund is really about funding software companies in the bio space.

In the near-half decade since, they havent softened their rhetoric. Pande and general partner Jorge Condes frequent blog posts often have the tone ofBurning Man technofuturists. Talking of a foundational shift in biology, bio-revolution, and the meaning of life, and dropping koans like what is medicine? has turned them into the well-financed New Age mystics of an AI-driven and bioengineered future.

Today, Andreessen Horowitz is launching bio fund III and putting $750 million behind it more than funds I and II combined. Theyve added new partners, as they did before fund I and II, picking up technologist and entrepreneur Julie Yoo and Vineeta Agarwala, a GV and Broad Institute alumn. Itll take much of the same tack as the earlier funds, investing early and occasionally up to Series B, and pouring funds not only into therapeutics, but also diagnostics, synthetic biology and startups bringing biological advances into other sectors, such as agriculture.

But Conde tells Endpoints News that the group has learned a thing or two since fund I. Pande had talked about extending Moores law to biology through digital therapeutics but they were wrong. It wasnt just about software and artificial intelligence. It was about the long list of ways how biology was done, how drugs were discovered and how the whole healthcare system functions. It was biotechs that worked both with machine learning and wet labs, and founders conversant in both.

Since then, theyve invested in companies like Insitro that integrate AI as a core but not sole part of a drug development chain and Asimov, which is trying to use AI and other tech systems to design a genome from scratch. They even invested in EQRx, Alexander Boriseys startup trying to use me-too drugs to change pricing.

In October, Conde, Pande and Yoo published their most soaring blog post yet: Biology is Eating the World: A Manifesto. They wrote: We are at the beginning of a new era, where biology has shifted from an empirical science to an engineering discipline.

Before the funds launch, though, Conde told Endpoints were at the end of the beginning for that era.

He talked about what theyve learned since bio I, where biology and biotech is headed and how well know when the convergence between engineering and biology hes been prophesizing has arrived.

You called this the end of the beginning for a new era. What does that mean?

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Striving for higher res imaging of cells, Harvard team debuts startup with backing from ARCH, Northpond - Endpoints News

Jack Chieh and Yinnie Wong with their baby boy, born last Friday (Chinese New Year). The couple donate her baby's cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre.Handout

Yinnie Wong and Jack Chiehs six-pound, 13-ounce baby boy as yet unnamed was born on an auspicious day, Jan. 24, Chinese New Year, and hes already doing good in the world.

Everyone was really happy, it is supposed to be a lucky day, said Wong.

Although the birth was a planned C-section, Wong had no control over the date hospital administrators chose for the birth. What she did have control over was the choice to donate her babys cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre, which has just celebrated its fifth anniversary.

Cord blood is blood that is taken from the umbilical cord and placenta immediately after the birth of a healthy infant. Cord blood is rich in stem cells, and can be used to treat over 80 diseases, including leukemia.

According to Canadian Blood Services, ethnically diverse donors are especially needed because although Stats Canada data shows 67.7 per cent of Canadians consider their ethnic origin to be diverse, only 31 per cent of Canadians with blood in Canadas stem-cell registry are from ethnically diverse backgrounds.

Crystal Nguyen, 20, is a former B.C. Childrens Hospital patient whose life was saved by a stem-cell transplant from donated cord blood. Nguyen was first diagnosed with acute myeloid leukemia at age 12. After chemo, she went into remission for almost three years. Then the cancer returned. She was told she needed a bone-marrow transplant.

Crystal Nguyen, now 20, was first diagnosed with acute myeloid leukemia at age 12. She found a stem-cell match for a needed bone-marrow transplant through the international cord blood bank.Handout

When I relapsed I was very confused, it was kind of surreal. The main thing about being told I needed the bone-marrow stem-cell transplant was confusion, fear and anxiety.

Nguyen is of Vietnamese descent and needed a match to survive. No one in her family was a match, nor was there a stem-cell match in the Canadian cord blood bank, but a match was found thanks to the Canadian Blood Services partnerships with 47 international blood banks.

I was told it came through the international cord blood bank from somewhere very far away, said Nguyen, who has been in remission since the transplant.

When she learned the stem-cell transplant had been successful, Nguyen, who is now studying to become a pediatric oncology nurse, said it felt too good to be true.

There was a lot of happiness, joy, excitement. Donating cord blood is such a simple way to save a life.

Although cord blood can be collected and stored for a fee by private companies and reserved for the donor familys use, cord blood donated through Canadian Blood Services is available free to the public whoever needs the match.

Wong didnt hesitate when her son was born. I felt like I wanted to do it if it helps someone in the public, and if it could save lives I would have been very happy to help another child, said Wong, who is a nurse at B.C. Womens hospital.

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Chinese New Year babys B.C. family gives gift of life in cord-blood donation - Vancouver Sun

Blocking the movement of cells from the bone marrow by inhibiting the CXCL12/CXCR4/CXCR7 signaling axis is a potential strategy for treating endometriosis, a recent study done in mice suggests.

The study, titled CXCR4 or CXCR7 antagonists treat endometriosis by reducing bone marrow cell trafficking, was published in theJournal of Cellular and Molecular Medicine.

Bone marrow-derived cells (BMDCs) play important roles in the normal functioning of the endometrium. For instance, stem cells from the bone marrow are involved in endometrial regeneration. But BMDCs also are involved in the formation of lesions in endometriosis.

The movement of BMDCs to uterine tissue whether for normal physiological reasons or as part of disease development is driven in large part by the signaling protein CXCL12. It acts through two protein receptors: CXCR4 and CXCR7. This CXCL12/CXCR4/CXCR7 signaling axis has been shown to be overactive in women with endometriosis.

Given the central role of the CXCL12/CXCR4/CXCR7 axis on BMDCs trafficking and in the pathogenesis [development] of endometriosis, we hypothesized that blocking CXCR4 or CXCR7 in endometriosis would inhibit the growth of endometriosis, the researchers said.

The scientists first used mouse models of endometriosis in which BMDCs were labeled with a fluorescent marker to confirm the presence of these cells in endometriotic lesions.

The BMDCs made up just over 10% of the total number of cells in lesions. Further, BMDCs that expressed CXCR4 represented about 4.4% of total lesion cells, while BMDCs expressing CXCR7 made up about 1.4%. CXCL12 also was highly expressed within the lesions.

The researchers then pharmacologically blocked each of the receptors, using Plerixafor (AMD3100) against CXCR4, and CCX771 against CXCR7. Plerixafor is used in stem cell transplants given to treat certain types of blood cancer. CCX771 is a small molecule without currently approved clinical uses.

Both treatments significantly reduced the percentage of BMDCs in lesions, suggesting that blocking this signaling axis did indeed stop the movement of these cells.

In addition, when either Plerixafor or CCX771 was given immediately after endometriosis establishment, the size of the endometriotic lesions was reduced by more than half compared with control mice. Blood vessel density also was significantly reduced, by about 40% for both receptors.

The treatments also reduced the expression of inflammatory signaling molecules known to be elevated in endometriosis, such as interleukin 6 (IL-6) and tumor necrosis factor alpha (TNFalpha).

In a separate experiment to further test the treatments potential, Plerixafor and CCX771 were administered a few weeks after the endometriosis lesions developed. This more closely models the preexisting lesions found in humans at the time of endometriosis diagnosis, the researchers said.

The results were similar to those seen in the earlier model: there were significant decreases in lesion size by about 60% as well as in levels of inflammatory signaling molecules.

Notably, neither drug had any detectable effect on hormone cycling in the mice, demonstrating that the effects of these agents worked [through] a hormone independent pathway, the researchers said.

Based on the data, the researchers concluded that blocking the CXCL12/CXCR4/CXCR7 signaling axis may treat endometriosis. However, these results alone do not demonstrate that this effect is directly because of reduced BMDC recruitment. It would be equally plausible to postulate that the effect is due to blocking CXCL12/CXCR4/CXCR7 signaling in the endometrial cells themselves, not BMDCs, the investigators said.

To test this idea, the team established endometriosis models in mice that were engineered so that the cells in their uteruses could not make CXCL12. There were no detectable differences between these endometriosis lesions and lesions in mice that could make CXCL12 in their uteruses. Further, Plerixafor had no detectable effect on human endometrial cells taken from people with endometriosis and treated in a dish.

This suggests that the beneficial effect induced by blocking CXCL12/CXCR4/CXCR7 signaling is due to an effect on cells outside of the uterus. Due to their prevalence in lesions, this most likely means BMDCs, the researchers said.

Clinical use [of these therapies] will likely depend on side effect profile; the effects of prolonged use are not well characterized, the team said. They added that future studies evaluating such drugs safety profiles and off-target effects, particularly with long-term use, will be needed before these results can be translated into clinical application.

CXCR4 and CXCR7 antagonists are promising novel, nonhormonal therapies for endometriosis, the researchers concluded.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

Total Posts: 146

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Blocking Bone Marrow Cell Movement May Be Non-Hormonal Treatment... - Endometriosis News Today

Spinal cord injuries can result in severe neurological dysfunction, including motor, sensory, and autonomic paralysis, and up until now there has been no cure or effective treatment for such injuries.

But the first human trial based on Nobel Prize winning induced pluripotent stem cells (IPSC) technology, is due to start in Japan, giving hope to hundreds of thousands of paralysed patients, that there might be light at the end of their tunnel.

The spinal cord is responsible for relaying signals up and down the body from the brain to the nervous system. The spinal cord is a bundle of nerves contained in the spinal canal, which is cocooned in the spinal column (not to be confused with the spinal cord they are two very separate entities).

The spinal cord itself has a protective sheath wrapped around it which acts as insulation whilst allowing nerve signals from the brain to travel even faster to where they need to go.

The spinal column is divided into five distinct sections:

The site of the spinal cord injury will determine the severity of the injury and injuries are classified as either:

The higher up the spinal cord the injury occurs, the more function and feeling will be lost. It is estimated that approximately every year there are between 8 to 246 cases per million incidences of spinal cord injuries worldwide.

Stem cell therapy is amongst the most exciting ongoing research for people with spinal cord injuries, in modern medicine. Because whilst the research is still in its infancy, legitimate trials are showing promising results.

According to the Journal of the American Academy of Orthopedic Surgeons, there are different stem cells which have varying abilities to restore certain functions.

Stem cells are self-renewing cells that can differentiate into one or more specific cell types. For people with spinal cord injuries, stem cells could prevent further cell death, stimulate cell growth from the existing cells and even replace the injured cells, restoring the communication channels between the body and the brain.

Until recently, stem cell research has involved looking at:

It is research into these induced pluripotent stem cells that the team in Japan are currently laying down the groundwork for. They are planning to conduct a first-in-human study of an induced pluripotent stem cell-based intervention, for subacute spinal cord injury.

Not only that, but it is the first such therapy to look into treating this kind of injury, that has ever received government approval for sale to patients.

However there are concerns by those who work in the field, but arent working on this particular project, that the evidence to support the suggestion that the treatment works, is insufficient. They state that the approval for the research was based on a small, poorly designed clinical trial.

Like the majority of scientific breakthroughs that have gone before, there will always be naysayers we used to think the world was flat and the sun orbited Earth, that the body was composed of four humours and an imbalance in those made us sick.

Those theories were disproved, and look how far weve come since then. Now imagine if we could make a paralysed person walk again. It will happen. But for now, lets celebrate and support this team for trying.

Because whilst there have been multiple attempts to develop stem cell transplantation approaches with the aim to regenerate damaged spinal cords before, this multicentre team is planning the first that might actually work, and be ethical to boot.

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Stem Cell Regeneration for Spinal Cord injuries

Jack Chieh and Yinnie Wong with their baby boy, born last Friday (Chinese New Year). The couple donate her baby's cord blood to the cord blood bank at B.C. Womens Hospital & Health Centre.Handout

By Denise Ryan

Yinnie Wong and Jack Chiehs six-pound, 13-ounce baby boy as yet unnamed was born on an auspicious day, Jan. 24, Chinese New Year, and hes already doing good in the world.

Everyone was really happy, it is supposed to be a lucky day, said Wong.

Although the birth was a planned C-section, Wong had no control over the date hospital administrators chose for the birth. What she did have control over was the choice to donate her babys cord blood to thecord blood bank at B.C. Womens Hospital & Health Centre, which has just celebrated its fifth anniversary.

Cord blood is blood that is taken from the umbilical cord and placenta immediately after the birth of a healthy infant. Cord blood is rich in stem cells, and can be used to treat over 80 diseases, including leukemia.

According to Canadian Blood Services, ethnically diverse donors are especially needed because although Stats Canada data shows 67.7 per cent of Canadians consider their ethnic origin to be diverse, only 31 per cent of Canadians with blood in Canadas stem-cell registry are from ethnically diverse backgrounds.

Crystal Nguyen, 20, is a former B.C. Childrens Hospital patient whose life was saved by a stem-cell transplant from donated cord blood. Nguyen was first diagnosed with acute myeloid leukemia at age 12. After chemo, she went into remission for almost three years. Then the cancer returned. She was told she needed a bone-marrow transplant.

When I relapsed I was very confused, it was kind of surreal. The main thing about being told I needed the bone-marrow stem-cell transplant was confusion, fear and anxiety.

Nguyen is of Vietnamese descent and needed a match to survive. No one in her family was a match, nor was there a stem-cell match in the Canadian cord blood bank, but a match was found thanks to the Canadian Blood Services partnerships with 47 international blood banks.

I was told it came through the international cord blood bank from somewhere very far away, said Nguyen, who has been in remission since the transplant.

When she learned the stem-cell transplant had been successful, Nguyen, who is now studying to become a pediatric oncology nurse, said it felt too good to be true.

There was a lot of happiness, joy, excitement. Donating cord blood is such a simple way to save a life.

Although cord blood can be collected and stored for a fee by private companies and reserved for the donor familys use, cord blood donated through Canadian Blood Services is available free to the public whoever needs the match.

Wong didnt hesitate when her son was born. I felt like I wanted to do it if it helps someone in the public, and if it could save lives I would have been very happy to help another child, said Wong, who is a nurse at B.C. Womens hospital.

dryan@postmedia.com

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Chinese New Year babys B.C. family gives gift of life in cord - The Province