Stem Cell Clinic

CAR-T Cell Therapy Means A Lot More Than One Or Two New Drug Approvals – Seeking Alpha

By Uncategorized

In the world of cancer medicine, immunotherapy has taken over with a vice-like grip, offering far-reaching potential for nearly every tumor type known to man. Most prominent in the marketplace have been the immune checkpoint inhibitors, with almost every one of the big five PD-1/PD-L1 antibodies (nivolumab, pembrolizumab, atezolizumab, avelumab, durvalumab) gaining some high-profile drug approval in the last 5 years, to say nothing of the landmark approval of Yervoy in 2011 to kick all of this fervor off.

But before immune checkpoint inhibitors were approved, we had cell-based immunotherapy, notably with the introduction (and subsequent challenges) of Provenge. Cell-based immunotherapy actually goes all the way back to the late 1800s, with bacterial infection being used as a vector to stimulate an immune response in cancer patients.

Now we've gotten more sophisticated. Three companies, Kite Pharma (NASDAQ:KITE), Juno Therapeutics (NASDAQ:JUNO), and Novartis (NYSE:NVS) have been frontrunners in the race to bring so-called CAR-T cell therapy to market for hematologic malignancies.

A primer

Source for image: cancer.gov

The intricate details of CAR-T cell manufacture are too complex to manage in a short publication like this one. But it can be simplified in broad terms down to a few steps:

Some of the more curious among us might be asking...why go to all this trouble? We can train the body's immune system to recognize specific targets. Heck, we've been doing it for decades now with Herceptin and Rituxan. What's wrong with the body's natural defense?

The answer is that CAR-T cells present a few extra advantages to ramp up the immune response: the CAR itself - The name of the technique gives this away. "Chimeric" isn't just a cool word (which it certainly is); it signifies that we've done something special to the receptor in question. In the current line of techniques, we've fused the antigen recognition portion of an antibody to the part of the T cell receptor that tells the cell to grow and divide.

This differs from the normal method the body has to detect a foreign antigen and develop T cells against it:

Source: Srivastava, et al.

You may not recognize the names of the molecules in this figure, but you should be able to see that on the left side, there is careful coordination of a large number of molecules that is required to activate a T cell.

CARs short circuit the whole process, allowing for direct activation of the T cells by tumor cells. This MHC-independent T cell activation is the linchpin of the whole process, bypassing a number of tumor cell defenses and allowing us to develop a special subset of T cells that specifically look for and eliminate any cells in the body that express the antigen we're looking for. In the current case, this is CD19, which is a marker of B cells, hence why all of these latest studies are looking at diseases like B-cell leukemia and diffuse large B-cell lymphoma.

Bioengineered T cells have an end in sight, with several techs being reviewed at the FDA

Since the seminal publication by Maude, et al in 2014 showing incredible response rates in a small cohort of children with relapsed/refractory acute lymphoblastic leukemia (ALL), the world has been watching and waiting for the emergence of CAR-T cell therapy and its revolutionary potential.

No rides are ever smooth in biotech, it seems. For a while, the three big players- KITE, JUNO, NVS- were chasing three different patient populations.

NVS had CTL019, which was being studied in pediatric patients with ALL.

JUNO had JCAR015 for adult patients with ALL.

KITE decided to chase a different beast first, focusing on patients with diffuse large B-cell lymphoma (DLBCL), an aggressive form of non-Hodgkin lymphoma.

In my mind, this presented three distinct patient classes that could allow all three technologies to be marketed simultaneously. In the United States, ALL in kids and adults is not generally managed by the same hematologists; pediatric doctors handle children, specifically.

Unfortunately, fate was not kind to JUNO, who had to suspend their ROCKET trial in adults due to life-threatening toxicity risk. I wrote about this episode last year, and even though the clinical hold was lifted, JUNO eventually terminated development of its JCAR015 platform in March 2017, choosing instead to focus on JCAR017 for DLBCL.

KITE and NVS, in contrast, have achieved significant progress in moving CAR-T cells to the clinic. Both axicabtagene ciloleucel and CTL019 are now being reviewed by federal regulators, and it is likely we'll see responses by the end of 2017.

Given results like those we've seen with the ZUMA and ELIANA (the former I covered in my digest series, 3 Things You Should Learn Today in Biotech), it seems like CAR-T cell therapy presents an enormously promising treatment strategy for these intractable B cell malignancies. Aside from the risk of cytokine storm (an active area of research), these CAR-T platforms are not associated with an outsized risk of severe toxicity, either. I am going to be very surprised if these two techs do not get the nod from the FDA.

Approval of just one of these methods has the promise to usher in a new era for immunotherapy

It is difficult to overstate how reticent the FDA can be to accept a new therapeutic strategy into the fold. They are definitely conservative, and I say this is a very GOOD thing. The history of cancer medicine is peppered with charlatans who have generated excitement and clamor for new, promising cancer therapies.

The FDA needs to be the voice of reason and consider everything, from manufacturing to efficacy to every bit of safety they can uncover. As such, many are frustrated with the speed at which they move.

But the data on CAR-T cells are too compelling to ignore. I think this is going to prompt the FDA to get more familiar with cell-based immunotherapy in general and develop a different tolerance for risk of these approaches.

This represents a major, major inroad for other forms of cell therapy, including JUNO's JCAR015 and the other KITE/NVS platforms for CAR-T cell therapy. We could potentially see approvals for CAR-T cells emerge quickly in other hematologic malignancy settings.

But it also could signal an increasing tolerance for other approaches. And this is the biggest implication for those looking for diamonds in the rough with the stock market. Lots of small up-and-comers are exploring cell-based immunotherapy in various forms. To name just a few:

It's time to get ready for a wild ride in immunotherapy

To be clear, pointing out these companies does not mean I'm suggesting you buy, buy, buy. There are still risks associated with all these nascent technologies, and many will not pan out. Hematologic malignancies have had a long history of achieving groundbreaking therapeutics results that do not translate to solid tumors, so CAR-T cell therapy for, say, pancreatic cancer sounds tantalizing, as this is a huge unmet need. But pancreatic cancer chews through "promising" technologies like nothing else. The graveyard is long and grim there.

Still, my thesis here is that the likely approval of CAR-T cells in heme malignancies is going to give the FDA more experience with "live" immunotherapies, which will help them produce better guidance for other players in the field. This will almost certainly generate substantial excitement, and intrepid investors had better get on the ball sooner rather than later, or else they'll find themselves chasing the gold. Use the experience of JUNO, KITE, and NVS to your favor, and learn what you can about these promising therapies. It will come to play a major role in your due diligence.

Disclosure: I am/we are long ADXS.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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CAR-T Cell Therapy Means A Lot More Than One Or Two New Drug Approvals - Seeking Alpha

Stem Cells for Knees: Promising Treatment or Hoax? – WebMD

By Stem Cell Treatment

April 14, 2017 -- At 55, George Chung of Los Angeles could keep up with skiers decades younger, taking on difficult slopes for hours and hours. "Skiing was my passion," he says.

Then the pain started, and the bad news. He had severe osteoarthritis, the ''wear-and-tear'' type, in both knees. Doctors suggested surgery, but he chose instead an investigational treatment -- injections of stem cells. Two months after the first treatment, he was out of pain. "I had been in pain of various degrees for 6 years," he says.

Now, nine treatments and 3 years later, he is back to intense skiing. Last year, he also took up long-distance cycling, completed five double-century cycling rides, and earned the prestigious California Triple Crown cycling award.

Treatments with stem cells -- which can grow into different types of cells -- are booming in the U.S., with an estimated 500 or more clinics in operation. Some clinics offer treatment for conditions ranging from autism to multiple sclerosis to erectile dysfunction, often without scientific evidence to support how well they work.

Treatment for knee arthritis is especially popular. Its one type of osteoarthritis, which afflicts 30 million Americans. Fees vary, but $2,000 per treatment for knee arthritis is about average. Insurance companies usually deny coverage, although in rare cases they may cover it when done alongwith another, established procedure.

Many doctors and scientists view the growth of stem cell treatments as very promising. But that growth comes as the FDA debates whether to tighten regulations on stem cell clinics after recent reports of patients suffering severe damage from treatment. The only stem cell-based product approved by the FDA is for umbilical cord blood-derived stem cells for blood cancers and other disorders.

In an editorial published March 16 in TheNew England Journal of Medicine, FDA officials warned the lack of evidence for unapproved stem cell treatments is ''worrisome." The officials cited reports of serious side effects, including two people who became legally blind after receiving the treatment in their eyes for macular degeneration.

In another case, a patient who received stem cell injections after a stroke developed paralysis and needed radiation treatment.

The FDA also notes that stem cell treatments potentially have other safety concerns, such as causing tumors to grow. And because patients mayreceive the treatmentsoutside of formal research studies, it can bedifficult to track their side effects.

Doctors say that treating the kneehasless of a chance forcomplications. It is also the body part with perhaps the most research.

Still, even doctors who offer the treatment for arthritic knees say more study is needed.

"We don't have a lot of controlled trials yet," says Keith Bjork, MD, an orthopedist in Amarillo, TX, who has given stem cell treatments to about 500 patients with knee arthritis in the past 5 years. "Their results are the strongest evidence," he says.

The most common side effects are joint stiffness and pain at the injection site as well as swelling, according to the results of one study.

For knee injections, doctors often take stem cells from the patient's bone marrow, fat tissue, or blood. Doctors who do the treatments cite anecdotal evidence as validation that the treatments work.

Marc Darrow, MD, the Los Angeles physical medicine specialist who cares for Chung, says he has done thousands of stem cell treatments. He uses stem cells from the patient's own bone marrow, a process he says is simple and fast.

His patients pain often subsides after knee injections, he says. He also has had cases in which the ''before'' and ''after'' X-rays suggest an increase in cartilage, he says.

Harvey E. Smith, MD, an assistant professor of orthopedic surgery at the Hospital of the University of Pennsylvania, says its clear the treatment has an effect. What is not as clear is how it lessens pain. Researchers are studying whether the stem cells themselves cut inflammation or if they release substances that affect other cells. They also are looking at whether the treatments can regenerate worn-out cartilage.

Published studies have produced mixed results. One from 2014 showed that stem cell injections given aftersurgery to remove torn knee cartilage showed evidence of cartilage regeneration and lessened pain. In March, researchers who reviewed the findings of six studies on stem cells for knee arthritis found that patients reported good results with no serious side effects. More data is needed, however, before researchers can recommend it.

''There is still not enough evidence to suggest this should be routine treatment for knee early osteoarthritis," says Wellington Hsu, MD, the Clifford C. Raisbeck professor of orthopedic surgery at Northwestern University Feinberg School of Medicine. Even so, he says, ''there is very little damage you are going to do with an injection to the knee. I think stem cells appear to be safe in orthopedic applications."

There is, of course, the risk that an investment of a couple thousand dollars will do nothing. But Hsu says that ''you are not going to find the catastrophic cases that will shut down a clinic [as may occur for other body parts].''

For people who have knee arthritis, the most invasive treatment is total knee replacement, Hsu says. Doctors are also testing other injectable therapies, including platelet-rich plasma, hyaluronic acid, and steroids, he says.

Consumers who decide to try stem cell treatments for achy knees should research their doctor and the specifics on the stem cell treatment. It's crucial to ask the clinic where the stem cells come from, Smith says. Ask if they will retrieve them from your own bone marrow or fat tissue, or if they will come from donors. The FDA requires donor cells and tissues to be tested for communicable diseases. There is no consensus on which source is best, but most doctors use stem cells from fat, Hsu says.

The FDA suggests patients who decide to get stem cells for any purpose should speak to their doctor about the potential risks and benefits, and ask whether they are part of an FDA-approved clinical trial. Most often, doctors who offer stem cell treatments are orthopedists, plastic surgeons, or physical medicine and rehabilitation doctors,

The reduction in pain, however, isnt permanent, Smith says. "The effect may last 6 months," he says, citing results from knee studies. When people are paying out of pocket, he adds, they may over-report good effects to feel like they got their money's worth.

Chung, the skier-cyclist, says the investment has been worth it. He plans to continue his injections once or twice a year, as needed, so he can stay active on the bike and the slopes.

SOURCES:

Wellington Hsu, MD, Clifford C. Raisbeck professor of orthopedic surgery, Feinberg Northwestern University School of Medicine, Chicago.

Harvey E. Smith, MD, assistant professor of orthopedic surgery, University of Pennsylvania, Philadelphia.

Keith Bjork, MD, orthopedic surgeon, Amarillo, TX; clinical advisory staff member, Amnio Technology.

Julian Cameron, MD, orthopedic surgeon, Tamarac, FL.

Marc Darrow, MD, Los Angeles physical medicine specialist.

George Chung, stem cell recipient, Los Angeles.

CDC: "Osteoarthritis Fact Sheet."

The Journal of Bone and Joint Surgery: "Adult Human Mesenchymal Stem Cells Delivered via Intra-Articular Injection to the Knee Following Partial Medial Meniscectomy."

The New England Journal of Medicine: "Clarifying Stem-Cell Therapy's Benefits and Risks."

American Academy of Orthopaedic Surgeons annual meeting, presentation: ''Platelet-Rich Plasma, Bone Morphogenetic Protein, and Stem Cells: What Surgeons Need to Know." March 14, 2017, San Diego.

International Society for Stem Cell Research. "Stem Cell Facts."

Andrea Fischer, FDA spokeswoman.

FDA: "Consumer Information on Stem Cells."

Link:
Stem Cells for Knees: Promising Treatment or Hoax? - WebMD

Parkinson’s breakthrough: Scientists one step closer to making a BRAIN out of stem cells – Express.co.uk

By Stell Cell Research

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'Cradle of life' stem cells taken from skin samples were developed into three-dimensional brain-like organisms capable of exchanging signals between each other in a network.

The petri dish cells behave in a similar way to the brain cells which produce messenger dopamine from neurons - and scientists hope they will be able to use them to come up with a cure for Parkinson's.

Dopamine maintains smooth body movements, but when the neurons die off, tremors, rigid muscles and other Parkinson's disease symptoms begin to take over.

The new developments mean scientists can now use the cells to test what environmental factors like pollutants have on the onset of the disease and potentially find a cure.

Lead author Professor Jens Schwamborn said: "Our cell cultures open new doors to brain research.

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"We can now use them to study the causes of Parkinson's disease and how it could possibly be effectively treated."

Our cell cultures open new doors to brain research

Professor Jens Schwamborn

The stem cells can be transformed into any cell type of the human body but cannot produce a complete organism.

PHD student Anna Monzel developed a procedure to convert the stem cells into brain cells as part of her doctoral thesis.

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Getty Images

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Tremor - One of the most noticeable signs of Parkinson's is a tremor that often starts in the hands or fingers when they are relaxed

She said: "I had to develop a special, precisely defined cocktail of growth factors and a certain treatment method for the stem cells, so that they would differentiate in the desired direction."

Prof Schwamborn from the Luxembourg Centre for Systems Biomedicine at Luxembourg University said: "Our subsequent examination of these artificial tissue samples revealed that various cell types characteristic of the midbrain had developed."

"The cells can transmit and process signals.

"We were even able to detect dopaminergic cells - just like in the midbrain."

The scientists say their petri dish study can also reduce the amount of animal testing in brain research.

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Because cell cultures in the petri dishes are of human origin in some aspects they resemble human brains more than the brains of lab animals such as rats or mice.

Professor Schwamborn added: "There are also attractive economic opportunities in our approach.

"The production of tissue cultures is highly elaborate.

"In the scope of our spin-off Braingineering Technologies Sarl, we will be developing technologies by which we can provide the cultures for a fee to other labs or the pharmaceutical industry for their research."

The study was published in the Stem Cell Reports journal.

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Parkinson's breakthrough: Scientists one step closer to making a BRAIN out of stem cells - Express.co.uk

General American Capital Partners Invests up to $5 Million in U.S. Stem Cell, Inc. – Yahoo Finance

By Stem Cell Clinic

SUNRISE, FL / ACCESSWIRE / April 13, 2017 / U.S. Stem Cell, Inc. (USRM), a Florida corporation and leader in novel regenerative medicine solutions and physician-based stem cell therapies for human and animal patients, has received a commitment to invest up to $5,000,000 from private equity firm General American Capital Partners LLC (GACP) in exchange for up to 63,873,275 shares of common stock.

"We see exponential growth in the stem cell industry, estimated to grow to $170 billion by 2020," said Joseph DaGrosa, Jr., a Principal with General American Capital Partners. "We are very pleased to join forces with U.S. Stem Cell, Inc., a leader in regenerative medicine solutions, to help expand our role in this important market."

The 21st Century Cures Act, signed into effect in December of 2016, builds on the FDA's ongoing efforts to advance medical product innovation and ensure that patients get access to treatments as quickly as possible, with continued assurance from high quality evidence that they are safe and effective.

"Patient demand for regenerative medicine procedures as a viable alternative to surgery, as well as the transformative capacity of stem cell therapies, are leading the way to increased acceptance by both the medical and regulatory communities," said Mike Tomas, President and CEO of U.S. Stem Cell, Inc.

About U.S. Stem Cell, Inc.

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

Our business, which includes three operating divisions (US Stem Cell Training, Vetbiologics, and US Stem Cell Clinic) includes the development of proprietary cell therapy products, as well as revenue generating physician and patient based regenerative medicine / cell therapy training services, cell collection and cell storage services, the sale of cell collection and treatment kits for humans and animals, and the operation of a cell therapy clinic. Management maintains that revenues and their associated cash in-flows generated from our businesses will, over time, provide funds to support our clinical development activities, as they do today for our general business operations. We believe the combination of our own therapeutics pipeline combined with our revenue generating capabilities provides the Company with a unique opportunity for growth and a pathway to profitability.

Forward-Looking Statements:

Except for historical matters contained herein, statements made in this press release are forward-looking statements. Without limiting the generality of the foregoing, words such as "may," "will," "to," "plan," "expect," "believe," "anticipate," "intend," "could," "would," "estimate," or "continue," or the negative other variations thereof or comparable terminology are intended to identify forward-looking statements. Forward-looking statements involve known and unknown risks, uncertainties, and other factors which may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Also, forward-looking statements represent our management's beliefs and assumptions only as of the date hereof. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

The Company is subject to the risks and uncertainties described in its filings with the Securities and Exchange Commission, including the section entitled "Risk Factors" in its Annual Report on Form 10-K for the year ended December 31, 2016, and its Quarterly Reports on Form 10-Q.

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Media Contact:

U.S. Stem Cell, Inc. 13794 NW 4th Street, Suite 212 Sunrise, Fl 33325 Phone: 954.835.1500 Email: usstemcell@us-stemcell.com

SOURCE: U.S. Stem Cell, Inc.

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General American Capital Partners Invests up to $5 Million in U.S. Stem Cell, Inc. - Yahoo Finance

Human Embryonic Stem Cells (hESC) Market to Reach $1 Billion by 2025 – Application of hESCs as a Promising Donor … – Business Wire (press release)

By Embryonic Stem Cells

DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of the "Human Embryonic Stem Cells (hESC) Market, 2014 - 2025" report to their offering.

The global human embryonic stem cells (hESCs) market is anticipated to reach USD 1.06 billion by 2025. Application of hESCs as a promising donor source for cellular transplantation therapies is anticipated to bolster progress through to 2025. hESCs technology tends to be useful for tissue engineering in humans due to high histocompatibility between host and graft.

Maintenance of developmental potential for contribution of derivatives of all three germ layers is an important feature of these cells. This ability remains consistent even after clonal derivation or prolonged undifferentiated proliferation, thus pronouncing its accelerated uptake.

In addition, these are capable in expressing high level of alkaline phosphatase, key transcription factors, and telomerase. These factors are found to be of great importance in the maintenance of the inner cellular mass pluripotency.

Furthermore, hESCs can be easily differentiated into defined neurons, neural lineages, oligodendrocytes, and astrocytes. Aforementioned characteristic makes it useful in studying the sequence of events that take place during early neurodevelopment.

However, use of stem cells derived from viable embryos is fraught with ethical issues, prompting scientists to explore other methods to generate ESCs. The other methods include derivation of embryonic germ cells, stem cells from dead embryos, and other techniques.

Companies Mentioned

Key Topics Covered:

1 Research Methodology

2 Executive Summary

3 Human Embryonic Stem Cells Market Variables, Trends & Scope

4 Human Embryonic Stem Cells Market: Application Estimates & Trend Analysis

5 Human Embryonic Stem Cells Market: Regional Estimates & Trend Analysis, by Application

6 Competitive Landscape

For more information about this report visit http://www.researchandmarkets.com/research/w7n75n/human_embryonic

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Human Embryonic Stem Cells (hESC) Market to Reach $1 Billion by 2025 - Application of hESCs as a Promising Donor ... - Business Wire (press release)

Global Human Embryonic Stem Cells Market Size, Growth, Share, Trends and Forecast by 2025- Market Research … – MilTech

By Embryonic Stem Cells

The global human embryonic stem cells (hESCs) market is anticipated to reach USD 1.06 billion by 2025, according to a new report by Grand View Research, Inc. Application of hESCs as a promising donor source for cellular transplantation therapies is anticipated to bolster progress through to 2025. hESCs technology tends to be useful for tissue engineering in humans due to high histocompatibility between host and graft.

Browse the report: http://www.orbisresearch.com/reports/index/human-embryonic-stem-cells-hesc-market-analysis-by-application-regenerative-medicines-stem-cell-biology-research-tissue-engineering-toxicology-testing-by-country-u-s-uk-germany-japan-china-and-segment-forecasts-2014-2025

Maintenance of developmental potential for contribution of derivatives of all three germ layers is an important feature of these cells. This ability remains consistent even after clonal derivation or prolonged undifferentiated proliferation, thus pronouncing its accelerated uptake.

In addition, these are capable in expressing high level of alkaline phosphatase, key transcription factors, and telomerase. These factors are found to be of great importance in the maintenance of the inner cellular mass pluripotency.

Furthermore, hESCs can be easily differentiated into defined neurons, neural lineages, oligodendrocytes, and astrocytes. Aforementioned characteristic makes it useful in studying the sequence of events that take place during early neurodevelopment.

Request a sample of the report: http://www.orbisresearch.com/contacts/request-sample/240830

However, use of stem cells derived from viable embryos is fraught with ethical issues, prompting scientists to explore other methods to generate ESCs. The other methods include derivation of embryonic germ cells, stem cells from dead embryos, and other techniques.

Further Key Findings from the Study Suggest:

hESC derivation provides a unique opportunity for early human development studies.

It is believed to hold a substantial potential for regenerative medicine and biopharma.

Differentiated derivatives of these cells are applicable for screening assays in development of novel pharmaceutical moieties.

Screening for mutagenic as well as toxic compounds can also be carried out using such derivatives.

Buy the report@http://www.orbisresearch.com/contact/purchase/240830

Presence of lack of suitable donor organs and tissues for regenerative medicine is expected to increase the demand thus influencing growth.

Stem cell research is anticipated to exhibit fastest growth amongst the other applications.

However, presence of controversies pertaining to their use as a consequence of ethical considerations is responsible for steady growth.

Europe accounts for considerable share of the market, following North America.

As per a recent survey carried out by Swiss government, citizens there are more willing to accept embryonic stem cell research than politicians.

Asia Pacific is anticipated to drive market with fastest YoY growth.

Rising awareness amongst the population and physicians with respect to associated therapies is anticipated to propel progress.

Key players contributing in this market are CellGenix GmbH, International Stem Cell Corporation, Thermo Fisher Scientific, Inc.; Kite Pharma, PromoCell GmbH, and Lonza.

Presence of clinical trial pipeline for embryonic stem cell derived therapy for targeting different diseases is expected to fuel growth.

The diseases targeted include macular degeneration, Parkinsons disease, type I diabetes mellitus, and spinal cord injury.

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Global Human Embryonic Stem Cells Market Size, Growth, Share, Trends and Forecast by 2025- Market Research ... - MilTech

An important step towards new malaria medicine – Phys.Org

By Uncategorized

April 13, 2017 On Fig 1. you see two virtual slices. On the left side it is through the parasite. The digestive vacuole is light gray and the hemozoin crystals are black. In addition you see two nuclei. To the right you see a slice behind the vacuole with a third nucleus. The number of nuclei can be used to calculate when the cell was infected. Credit: Niels Bohr Institute

An international research team, led by Sergey Kapishnikov from the X-ray and Neutron Science section at the Niels Bohr Institute, has developed new techniques in analyzing malaria infected red blood cells, an important step towards finding more effective medicine. This amoeba is the biggest killer in the world earth's most dangerous animal.

The parasite has a complicated life cycle, and during most of the time, the parasite is inside a cell, making it very difficult for the immune system to respond. No patient has been found with permanent immunity, making the probability of developing a vaccine very low.

Dr Kapishnikov's group used advanced technologies to produce virtual cell slices, and examined them with soft and hard x-rays. In particular, they focused on the concentrations of iron, sulfur and potassium, in order to find the concentrations of iron in hemoglobin and in the hemozoin crystals found inside the parasites. They also found that the potassium concentration in infected cells was seven times lower than in pristine cells, but that the overall concentration was the same, suggesting that the potassium was absorbed by the parasite.

The parasite digests hemoglobin, using the protein as a nutrient. Heme molecules are formed during the degradation of hemoglobin. Heme is poisonous to the parasite, so it is immediately stored in pairs inside the digestive vacuole as hemozoin crystals. Now they are harmless, so an obvious goal to develop new cures against malaria could be to prevent this hemozoin formation, so that the heme's negative effects would remain.

Explore further: Malarial parasites dodge the kill

Scientists have uncovered a potential mode of parasite drug resistance in malaria infection, according to a report published in The Journal of Experimental Medicine.

The iron-containing molecule heme is necessary for life. Cells require heme to perform the chemical reactions that produce energy, among other critical tasks.

Two new studies from the Francis Crick Institute shed light on how the malaria parasite grows inside a host's red blood cells and breaks out when it's ready to spread to new host cells.

The compound that detectives spray at crime scenes to find trace amounts of blood may be used one day to kill the malaria parasite.

Despite decades of malaria research, the disease still afflicts hundreds of millions and kills around half a million people each year - most of them children in tropical regions. Part of the problem is that the malaria parasite ...

Malaria parasites cause red blood cells to become bendier, helping the parasites to enter and cause infection, says a new study.

A team of scientists from the Broad Institute of MIT and Harvard, the McGovern Institute for Brain Research at MIT, the Institute for Medical Engineering & Science at MIT, and the Wyss Institute for Biologically Inspired ...

People's ability to make random choices or mimic a random process, such as coming up with hypothetical results for a series of coin flips, peaks around age 25, according to a study published in PLOS Computational Biology.

The bacterial flagellum is one of nature's smallest motors, rotating at up to 60,000 revolutions per minute. To function properly and propel the bacterium, the flagellum requires all of its components to fit together to exacting ...

There are many processes that take place in cells that are essential for life. Two of these, transcription and translation, allow the genetic information stored in DNA to be deciphered into the proteins that form all living ...

A research group led by Hitoshi Kurumizaka, a professor of structural biology at Waseda University, unveiled the crystal structure of an overlapping dinucleosome, a newly discovered chromatin structural unit. This may explain ...

Hunting is a major threat to wildlife particularly in tropical regions, but a systematic, large-scale estimate of hunting-induced declines of animal numbers has been lacking. A study published in Science on April 14 fills ...

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An important step towards new malaria medicine - Phys.Org

Parkinson’s stem cell therapy 2.0: New treatment coaxes the brain to repair itself – Genetic Literacy Project

By Stem Cell Treatment

For the past five decades pharmaceutical drugs like levodopa have been the gold standard for treating Parkinsons disease. These medications alleviate motor symptoms of the disease, but none of them can cure it.

Now a study from the Karolinska Institute in Stockholm shows it is possible to coax the brains own astrocytescells that typically support and nurture neuronsinto producing a new generation of dopamine neurons.

The reprogrammed cellscould alter the course of Parkinsons, according to the researchers. You can directly reprogram a cell that is already inside the brain and change the function in such a way that you can improve neurological symptoms, says senior author Ernest Arenas, a professor of medical biochemistry at Karolinska.

Directly converting astrocytes already present in patients brains could eliminate the need to search for donor cells[and the treatment may] be less likely to cause side effects compared with current drugs. This is like stem cell 2.0. Its the next-generation approach to stem cell treatments and regenerative medicine, says James Beck, vice president and chief scientific officer, for the nonprofit Parkinsons Disease Foundation.

[Read the original study]

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Cell Therapy 2.0: Reprogramming the Brains Own Cells for Parkinsons Treatment

For more background on the Genetic Literacy Project, read GLP on Wikipedia

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Parkinson's stem cell therapy 2.0: New treatment coaxes the brain to repair itself - Genetic Literacy Project

How Medical Research Is Boosting Connecticut’s Economy – Yale News

By Stell Cell Research

by Kathleen Raven April 13, 2017

Jason Thomson, a core lab manager at the Yale Stem Cell Center, rebounded after large pharmaceutical companies retrenched in Connecticut. Photo credit: Robert Lisak

Six years ago, Jason Thomson learned that his 13-year position in research at Pfizer would come to an end. He was among 1,100 employees laid off at the companys drug development laboratory in Groton. He feared that his career was in jeopardy. He didnt want to move his family and worried he wouldnt be able to land a comparable job in Connecticut.

But things worked out much better than he expected. I was fortunate, says Thomson, a resident of Colchester. I was out of work for just over six months. Today, hes a lab manager at the Yale Stem Cell Center in New Haven. He plays a key role at the center, overseeing the preparation of stem cells that other researchers use to pursue their studies.

Thomsons personal journey illustrates an economic shift in Connecticut. Over the past decade, several large pharmaceutical companies have either closed their doors here or cut hundreds of jobs from their local payrolls. These moves pose a threat to the state economy. For Connecticut to thrive in the future, say state political, academic and business leaders, more jobs are needed in groundbreaking biomedical research and a home-grown biotech industry.

The 10-year-old Yale Stem Cell Center, which is within Yale School of Medicine, is an example of how this can be done. It has already created more than 200 jobs; involves more than 450 Yale faculty, post-docs and students; has produced more than 350 patent applications; and has three therapies currently being tested in clinical trials. And, because this type of research typically takes many years to have maximum impact, its likely that the best is yet to come.

So far, three clinical trials are testing drugs based on scientific advances produced by Stem Cell Center researchers. They include using cell-based tissue engineering to cure congenital heart defects, and using skeletal stem cells to treat stroke and spinal cord injuries.

Here's an infographic explaining how the Yale Stem Cell Center contributes to society.

This is about faculty members and researchers making breakthrough discoveries and passing them along to business experts to take to the market.

Yale School of Medicine plays a critical role in fostering a fast-growing bioscience industry in the New Haven area. Already, upwards of 40 biotech and medical device companies employ more than 5,000 people in greater New Haven. This is about faculty members and researchers making breakthrough discoveries and passing them along to business experts to take to the market, says Susan Froshauer, president of Connecticut United for Research Excellence (CURE), the bioscience industrys advocacy group.

At Pfizer, Thomsons job was to determine the safety profile of drugs using embryonic stem cells from mice. The New York native, who studied animal science at Cornell University, loved the company and his job, but he wasnt surprised when the bad news came. He had seen evidence that a retrenchment in the pharmaceutical industry was underway. For instance, just a few years earlier, Bayer Healthcare began shutting down its West Haven facility, which displaced about 1,000 workers. (The sprawling facility is now Yale Universitys West Campus.)

When Thomson received the layoff notice, leaving Connecticut and moving to another state wasnt an attractive option. He didnt want to disrupt his wifes career as a tenured high school teacher, nor the lives of his two young daughters.

He recalled hearing about efforts in the state to foster its strengths in biosciencein part by funding university research. Thomson began monitoring university websites. After a few nervous months, he got his big break. The Yale Stem Cell Center posted what he considered a dream job. Thomson appliedand got it.

Hes now a respected leader and colleague at the center. Caihong Qiu, Ph.D., who is the technical director of the Centers two core science labs, says researchers there admire Thomson for his deep scientific knowledge and helpful manner. Jason is the face of the core. He is very thorough and dedicated, Qiu says.

At the center, Thomson grows stem cells so scientists can conduct experiments to better understand the underlying cause of diseases, or to learn how to build new human organs. He provides feedback on study designs, orders lab supplies, and oversees the nitrogen tanks and other machinery that keep 10 years worth of cells frozen. He calls the core labs the special forces unit within the center. No matter how difficult the task is, they get it done.

Thomson loves working with stem cells because they contain clues to many unanswered questions surrounding how humans grow and develop. The long lab hours and a two-hour round-trip daily commute from his home in Colchester dont dampen his enthusiasm. Says Thompson: You have to love what you do for a living, and I do.

This article was submitted by Stephen Hamm on April 12, 2017.

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How Medical Research Is Boosting Connecticut's Economy - Yale News

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