Madalayna Ducharme is celebrating her first birthday, on Aug, 22, 2017. She's shown recently in a family pool.Courtesy of the Ducharme family / Windsor Star

Bowling for Bone Marrow fundraiser Saturday

To support the families of those who need astem cell/bone marrow transplant, head to this weekends 12th annual Bowling for Bone Marrow Throw a Strike for the Gift of Life.

The Katelyn Bedard Bone Marrow Association fundraiser is Saturday at Rose Bowl Lanes with a noon check-in time and bowling between 1 and 3 p.m. Walk-ins are welcome and the cost is $20 without a pledge form.Children under age 12 get in free and there will be a clown and childrens activities.

To register, call (519) 564-4119 or go online atwww.givemarrow.net/index.html.

Windsors warrior princess Madalayna Ducharme celebrates her first birthday Tuesday.

Were so happy and grateful that weve had her for a year, her mom, Tamara Ducharme, said Monday. I know back at the six-month mark we had a little celebration for her just in case we didnt have a year birthday.

On March 17, little Madalayna received a bone marrow transplant to save her from a rare genetic disorder.

Madalayna will get to try cake for the first time, even if its just for her tiny fingers to play in, and her mom plans to go live on Facebook in the late afternoon for the approximately 3,000 supportive followers on the Miracle for Madalaynasite.

I cant believe how many people love her and support us. It makes us so happy, Ducharme said. We could be going through this alone. I feel like there are 3,000 fighters in our corner.

Madalayna, dubbed the warrior princess, was just two months old when doctors noticed issues that a few months later would be diagnosed as malignant infantile osteopetrosis which leads toabnormal thickening of the bone. Without treatment, the one-in-200,000 genetic disorder would dramatically reduce the infants life expectancy.

The Windsor community rallied around the family, and there were efforts made to get more people to join the bone marrow registry. Ducharme said shes thankful for the support from the Katelyn Bedard Bone Marrow Association. She said getting swabbed for the registry wasnt just for Madalayna but to help all those waiting for a match.

Because of the genetic disorder, at first doctors werent looking to family members for a match but Madalaynas two-year-old brother Henrik proved a perfect match and doctors consulted in the United States and Europe agreed his bone marrow was the familys best option.

The family didnt get Madalayna home from Toronto and London hospitals until July, and the little warrior fought off a virus that is worrisome with transplant patients, her mom said. So far, blood tests are looking good but the family wont know until after more extensive tests later this week in Toronto whether the transplant is working.

Ducharme is asking for prayers for good news in Toronto. The transplant is as close to a cure as possible, she said. Madalayna may have hearing and sight issues from the disease, but if the bones look better and the transplant is working, it gives her a chance at a longer life. Ducharme has heard of a man who had the disease and a transplant as a baby and is now 25 years old.

Madalyna, who loves music and looks like a princess in her tutu and frilly dresses, is a bit delayed with all that shes been through, but a week ago she sat up for the first time and she likes to dance by bouncing and swaying to techno music. She still needs the tube in her nose and doesnt like drinking liquids and isnt eating properly. Shes improving but her mom doesnt know what her baby will think of birthday cake.

Were excited.

shill@postmedia.com

twitter.com/winstarhill

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First birthday for Windsor's 'warrior princess' after lifesaving transplant - Windsor Star

What if one day, we could teach our bodies to self-heal like a lizards tail, and make severe injury or disease no more threatening than a paper cut?

Or heal tissues by coaxing cells to multiply, repair or replace damaged regions in loved ones whose lives have been ravaged by stroke, Alzheimers or Parkinsons disease?

Such is the vision, promise and excitement in the burgeoning field of regenerative medicine, now a major ASU initiative to boost 21st-century medical research discoveries.

ASU Biodesign Institute researcher Nick Stephanopoulos is one of several rising stars in regenerative medicine. In 2015, Stephanopoulos, along with Alex Green and Jeremy Mills, were recruited to the Biodesign Institutes Center for Molecular Design and Biomimetics (CMDB), directed by Hao Yan, a world-recognized leader in nanotechnology.

One of the things that that attracted me most to the ASU and the Biodesign CMDB was Haos vision to build a group of researchers that use biological molecules and design principles to make new materials that can mimic, and one day surpass, the most complex functions of biology, Stephanopoulos said.

I have always been fascinated by using biological building blocks like proteins, peptides and DNA to construct self-assembled structures, devices and materials, and the interdisciplinary and highly collaborative team in the CMDB is the ideal place to put this vision into practice.

Yans research center uses DNA and other basic building blocks to build their nanotechnology structures only at a scale 1,000 times smaller than the width of a human hair.

Theyve already used nanotechnology to build containers to specially deliver drugs to tissues, build robots to navigate a maze or nanowires for electronics.

To build a manufacturing industry at that tiny scale, their bricks and mortar use a colorful assortment of molecular Legos. Just combine the ingredients, and these building blocks can self-assemble in a seemingly infinite number of ways only limited by the laws of chemistry and physics and the creative imaginations of these budding nano-architects.

Learning from nature

The goal of the Center for Molecular Design and Biomimetics is to usenatures design rulesas an inspiration in advancing biomedical, energy and electronics innovation throughself-assembling moleculesto create intelligent materials for better component control and for synthesis intohigher-order systems, said Yan, who also holds the Milton Glick Chair in Chemistry and Biochemistry.

Prior to joining ASU, Stephanopoulos trained with experts in biological nanomaterials, obtaining his doctorate with the University of California Berkeleys Matthew Francis, and completed postdoctoral studies with Samuel Stupp at Northwestern University. At Northwestern, he was part of a team that developed a new category of quilt-like, self-assembling peptide and peptide-DNA biomaterials for regenerative medicine, with an emphasis in neural tissue engineering.

Weve learned from nature many of the rules behind materials that can self-assemble. Some of the most elegant complex and adaptable examples of self-assembly are found in biological systems, Stephanopoulos said.

Because they are built from the ground-up using molecules found in nature, these materials are also biocompatible and biodegradable, opening up brand-new vistas for regenerative medicine.

Stephanopoulos tool kit includes using proteins, peptides, lipids and nucleic acids like DNA that have a rich biological lexicon of self-assembly.

DNA possesses great potential for the construction of self-assembled biomaterials due to its highly programmable nature; any two strands of DNA can be coaxed to assemble to make nanoscale constructs and devices with exquisite precision and complexity, Stephanopoulos said.

Proof all in the design

During his time at Northwestern, Stephanopoulos worked on a number of projects and developed proof-of-concept technologies for spinal cord injury, bone regeneration and nanomaterials to guide stem cell differentiation.

Now, more recently, in a new studyin Nature Communications, Stephanopoulos and his colleague Ronit Freeman in the Stupp laboratory successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways.

In the new technology, materials are first chemically decorated with different strands of DNA, each with a unique code for a different signal to cells.

To activate signals within the cells, soluble molecules containing complementary DNA strands are coupled to short protein fragments, called peptides, and added to the material to create DNA double helices displaying the signal.

By adding a few drops of the DNA-peptide mixture, the material effectively gives a green light to stem cells to reproduce and generate more cells. In order to dynamically tune the signal presentation, the surface is exposed to a soluble single-stranded DNA molecule designed to grab the signal-containing strand of the duplex and form a new DNA double helix, displacing the old signal from the surface.

This new duplex can then be washed away, turning the signal off. To turn the signal back on, all that is needed is to now introduce a new copy of single-stranded DNA bearing a signal that will reattach to the materials surface.

One of the findings of this work is the possibility of using the synthetic material to signal neural stem cells to proliferate, then at a specific time selected by the scientist, trigger their differentiation into neurons for a while, before returning the stem cells to a proliferative state on demand.

One potential use of the new technology to manipulate cells could help cure a patient with neurodegenerative conditions like Parkinsons disease.

The patients own skin cells could be converted to stem cells using existing techniques. The new technology could help expand the newly converted stem cells back in the lab and then direct their growth into specific dopamine-producing neurons before transplantation back to the patient.

People would love to have cell therapies that utilize stem cells derived from their own bodies to regenerate tissue, Stupp said. In principle, this will eventually be possible, but one needs procedures that are effective at expanding and differentiating cells in order to do so. Our technology does that.

In the future, it might be possible to perform this process entirely within the body. The stem cells would be implanted in the clinic, encapsulated in the type of material described in the new work, and injected into a particular spot. Then the soluble peptide-DNA molecules would be given to the patient to bind to the material and manipulate the proliferation and differentiation of transplanted cells.

Scaling the barriers

One of the future challenges in this area will be to develop materials that can respond better to external stimuli and reconfigure their physical or chemical properties accordingly.

Biological systems are complex, and treating injury or disease will in many cases necessitate a material that can mimic the complex spatiotemporal dynamics of the tissues they are used to treat, Stephanopoulos said.

It is likely that hybrid systems that combine multiple chemical elements will be necessary; some components may provide structure, others biological signaling and yet others a switchable element to imbue dynamic ability to the material.

A second challenge, and opportunity, for regenerative medicine lies in creating nanostructures that can organize material across multiple length scales. Biological systems themselves are hierarchically organized: from molecules to cells to tissues, and up to entire organisms.

Consider that for all of us, life starts simple, with just a single cell. By the time we reach adulthood, every adult human body is its own universe of cells, with recent estimates of 37 trillion or so. The human brain alone has 100 billion cells or about the same number of cells as stars in the Milky Way galaxy.

But over the course of a life, or by disease, whole constellations of cells are lost due to the ravages of time or the genetic blueprints going awry.

Collaborative DNA

To overcome these obstacles, much more research funding and recruitment of additional talent to ASU will be needed to build the necessary regenerative medicine workforce.

Last year, Stephanopoulos research received a boost with funding from the U.S. Air Forces Young Investigator Research Program (YIP).

The Air Force Office of Scientific ResearchYIP award will facilitate Nicks research agenda in this direction, and is a significant recognition of his creativity and track record at the early stage of his careers, Yan said.

Theyll need this and more to meet the ultimate challenge in the development of self-assembled biomaterials and translation to clinical applications.

Buoyed by the funding, during the next research steps, Stephanopoulos wants to further expand horizons with collaborations from other ASU colleagues to take his research teams efforts one step closer to the clinic.

ASU and the Biodesign Institute also offer world-class researchers in engineering, physics and biology for collaborations, not to mention close ties with the Mayo Clinic or a number of Phoenix-area institutes so we can translate our materials to medically relevant applications, Stephanopoulos said.

There is growing recognition that regenerative medicine in the Valley could be a win-win for the area, in delivering new cures to patients and building, person by person, a brand-new medicinal manufacturing industry.

Stephanopoulos recent research was carried out at Stupps Northwesterns Simpson Querrey Institute for BioNanotechnology. The National Institute of Dental and Craniofacial Research of the National Institutes of Health (grant 5R01DE015920) provided funding for biological experiments, and the U.S. Department of Energy, Office of Science, Basic Energy Sciences provided funding for the development of the new materials (grants DE-FG01-00ER45810 and DE-SC0000989 supporting an Energy Frontiers Research Center on Bio-Inspired Energy Science (CBES)).

The paper is titled Instructing cells with programmable peptide DNA hybrids. Samuel I. Stupp is the senior author of the paper, and post-doctoral fellows Ronit Freeman and Nicholas Stephanopoulos are primary authors.

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Bio-inspired Materials Give Boost to Regenerative Medicine - Bioscience Technology

First came the prospect of pigs incubating human organs. Now a medical ethicist is raising new moral questions by suggesting scientists create human-animal chimeras to produce human eggs.

While the goal, for now, would be to create a ready supply of eggs purely for biomedical research purposes, should the hybrid human eggs turn out to be as good as ones produced by humans, I do not see any reason for not using them for treating human infertility, said Csar Palacios-Gonzlez, of the Centre of Medical Law and Ethics at Kings College London.

In a commentary in Reproductive BioMedicine Online, Palacios-Gonzlez tests arguments against creating chimeras for human gamete production, and finds all of them wanting.

Despite ongoing research and scientific and ethical discussions about the development of chimeras capable of producing solid organs such as kidneys and hearts for transplantation purposes, he writes, no wide discussion of the possibility of creating chimeras-IHGP (intended for human gamete production) has taken place. If anything, scientists have fallen over themselves to reassure the public steps will be taken to avoid creating such creatures.

A leading Canadian reproductive biologist called the paper deeply thought provoking and says the idea isnt outside the realm of possibility.

Humans are mammals and there is really nothing intrinsically different about the process of reproduction between humans and every other mammal, said Roger Pierson, a world expert on ovarian physiology at the University of Saskatchewan.

There is really nothing intrinsically different about the process of reproduction between humans and every other mammal

Were talking here not about what the combination of mammalian gametes might become, but were talking about the actual biological processes of passing our DNA from one generation to the next, he said.

The biology that comes out of this analysis is questioning some of the tenets of our assumptions about reproduction.

In theory, the process could involve interspecies blastocyst complementation the same technique researchers are exploring to create pigs capable of generating human organs for transplant.

A blastocyst an early embryo is taken from an animal and genes crucial for the development of a particular cell line or organ edited out. In this case you would aim at the reproductive system, Palacios-Gonzlez said in an interview.

Next, human pluripotent stem cells (cells that have the potential to develop into any type of tissue in the body) taken from a donors skin are injected into the blastocyst to compensate for the existing niche, he said. In this case human stem cells would complete the reproductive system, which would then create gametes.

What conceivably could result is the ovary of a sow (or cow or other animal) that produces human eggs.

In January, Salk Institute scientists reported in the journal Cell they had succeeded in creating the first human-pig chimera embryos. None were allowed to grow beyond four weeks and half were abnormally small. But in others, the human stem cells survived and turned into progenitors for different tissues and organs.

The achievement was hailed a scientific tour de force. It also rattled ethicists, who warned of the remote but not impossible risk human stem cells intended to morph into a new liver, pancreas or heart could wend their up to the animals brain, raising the prospect of a chimera with human consciousness.

Others worried about transplanted human stem cells generating reproductive tissues. Few people want to see what might result from the union between a pig with human sperm and a sow with human eggs, the New York Times warned.

Palacios-Gonzlez said that as far as he is aware, no one is actively pursuing creating chimeras capable of producing human sperm or eggs. But maybe I am wrong, the world is just too big. (The research that comes closest, he said, was published in 2014, when stem cells were taken from a skin sample from a man who produced no sperm and transplanted into the testicles of a mouse, where they became immature sperm.)

However, Palacios-Gonzlez argues that claims that the creation of chimeras violates human dignity are just false.

Most dont consider lab mice grafted with human cells such a violation, he writes in Reproductive BioMedicine.Neither do we consider that human dignity is violated when someone receives a pig heart valve, which effectivelyturnsthem into a chimera.

If human dignity is tied tothe possession of certain higher mental capacities, he added, gene-editing tools like CRISPR could be used to avoid generating brain tissue, thereby reducingthe possibility of accidentally creating a chimera with human brain cells.

Fears a human egg-producing chimera could become pregnant is a practical issue that could easily be avoided by, for example, creating only female chimeras, he writes.This would be the most sensible thing to do given that there is no shortage of human sperm for research purposes.

Even if it should one day become desirable to create chimeras capable of producing both eggs and sperm,we could just take the appropriate measures for (the chimeras) to be segregated by sex.

He also argues that whether generated by humans or chimeras human gametes do not possess intrinsic worth capable of being debased and that the eggs incubated by chimeras could go toward research capable of saving peoples lives.

Pierson said that, with focused work and funding, this kind of work could be done in probably a year or less. This is not far fetched.

This is not about having a male mouse thats ejaculating human sperm, coupled with a female mouse thats ovulating human eggs and creating a human embryo in the mouse, Pierson said.

Rather, among research questions, Its about understanding what our reproductive processes are and what they could become, he said. We need to lay down the ethical principles for exploring these new types of ideas.

Pierson said it could be the next step toward the completely lab-based generation of sperm and eggs. In vitro gametogenesis, or IVG, a technique still in its infancy, is aimed at creating functional sperm and eggs from induced stem cells. Last year, researchers in Japan reported in the journal Nature they had created mouse pups born from eggs created in a petri dish.

Pierson said any eggs generated from a nonperson chimera would likely come from a cow, and not a mouse, noting cows and humans share similar ovarian function.

NYU School of Medicine bioethicist Arthur Caplan said the technology is a decade or more away and would need safety testing in animals for another few years, if it even worked.

Safety issues are huge for chimeras, just huge, he added, including unknown mutations, subtle chemical differences in the derived eggs and the risk of communicating animal viruses.

Email: skirkey@nationalpost.com | Twitter:

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No moral reason not to create chimeras capable of making human eggs, ethicist argues - National Post

How long do you expect to live?

Thats a question that can make a lot of people feel suddenly lost for an answer.

In fact, its not a question that anybody would like to answer.

However, for scientific, socio-economic, and other legitimate reasons, average life expectancy per region are being documented. According to the World Factbook by the Central Intelligence Agency, the average life expectancy at birth of the following countries as of 2016 are as follows:

The rest of the world has an average life expectancy of 80 years downwards, with Chad ranking the lowest at 50.20 years.

Life is short, too short.

Its the reason why the pursuit of anything and everything under the sun that can stop aging is mankinds obsession.

We want to live longer; if possible, forever.

Forever is definitely too, too far away. But, longer, yes. Its more probable.

Heres the latest news on anti-aging, and this time its about stem cells. Stem cells from a young heart may help in regaining vitality which we lose as we grow old.

Researchers from the Cedars-Sinai Heart Institute have recently discovered that upon application of Cardiosphere-derived cells (CDC), which they took from newborn mice and injected into the hearts of 22-month-old mice, had resulted to better heart functionality, hair regrowth at a faster rate, 20 percent longer exercise endurance, and longer cardiac telomeres.

The findings on the effect of CDC cells on telomeres is very significant since these compound structures located at the tip of chromosomes function as the cells time-keepers. In fact, another study is focusing on methods to lengthen telomeres to fight the effects of progeria and help prolong life.

Our previous lab studies and human clinical trials have shown promise in treating heart failure usingcardiac stem cell infusions, saidCedars-Sinai Heart Institute and lead researcher Eduardo Marbn, MD, PhD, Now we find that these specialized stem cells could turn out to reverse problems associated with aging of the heart.

According to Dr. Marban, the CDC cells work on reversing the aging process by secreting very small vesicles that are full of signaling molecules like proteins and ribonucleic acid (RNA). The vesicles appear to have all the necessary information in producing cardiac and systemic rejuvenation.

In 2009, the LA-based team achieved the worlds first stem cell infusion which they hope to use in treating patients with Duchenne muscular dystrophy and cases of heart failure with preserved ejection fraction. However, this was the first time that they have observed this kind of rejuvenating effects of CDC cells.

Nevertheless, Dr. Marban and his team acknowledge that they still have a lot to do and figure out. They havent determined yet if the CDC cells could lengthen life, or just produce a younger heart in an aged physique. They also have to find out if the cells must come from younger hearts for the stem cell treatment to be effective.They will obviously need more time and tests to find the right answers to these very important questions.

But, if Dr. Marban and his team succeed, CDC cells may be a key to restoring youth and vigor. It will also help globally the large number of people who suffer from cardiovascular diseases-heart disease is the worlds number 1 killer and accounts for 17.3 million deaths per year.

The study was published on theEuropean Heart Journal.

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In World First, Scientists Reverse Aging in Old Hearts by Injecting Younger Cells - Wall Street Pit

Researchers at the Mayo Clinic and the University of Minnesota say theyre on the brink of a new era in cancer care one in which doctors extract a patients white blood cells, have them genetically engineered in a lab, and put them back to become personalized cancer-fighting machines.

The so-called CAR T cellular therapies are expected to receive federal approval this fall for certain rare blood cancers B-cell forms of lymphoma and leukemia. But scientists at the Minnesota institutions hope thats just the first step that will lead to better treatment of solid tumor cancers as well.

This is really the first approval of a genetically modified product for cancer therapy, said Dr. Jeffrey Miller, deputy director of the Masonic Cancer Center at the University of Minnesota. If the proof of concept works, he said, we might be on the right track to get away from all of that toxic chemotherapy that people hate.

Participating in industry-funded clinical trials, the Minnesota researchers hoped to determine if patients with leukemia or lymphoma would be more likely to survive if their own stem cells were extracted to grow cancer-fighting T-cells that were then infused back into their bodies.

One analysis, involving trials by Kite Pharmaceuticals at Mayo and other institutions, found a sevenfold increase in lymphoma patients whose cancers disappeared when they received CAR T instead of traditional chemo-based treatment.

I often tell patients that T-cells are like super robocops, said Dr. Yi Lin, a Mayo hematologist in Rochester. Were now directing those cells to really target cancer.

The U.S. Food and Drug Administration is widely expected this fall to approve CAR T products made by Kite and Novartis, which genetically engineer T-cells to target so-called CD19 proteins found on the surface of leukemia and lymphoma cells.

The side effects can be harsh, because the T-cell infusions trigger an immune system response that can produce fever, weakness, racing heart and kidney problems. Short-term memory and cognitive problems also have occurred. Brain swelling led to five deaths of cancer patients who took part in a CAR T trial by Juno Pharmaceuticals. The trial was shut down as a result.

Lin said brain swelling appeared mostly in adults with leukemia. For now, she expects Kites CAR T therapy to be approved for diffuse large B-cell lymphoma and the Novartis therapy to be approved for acute lymphoblastic leukemia in children. Federal regulations also might restrict CAR T for patients whose cancers survived traditional treatments.

Current practice to treat these cancers generally involves chemotherapy and radiation. Physicians then transplant stem cells, often from donor bone marrow, to regrow the patients immune systems, which are weakened in the process of treatment.

CAR T differs in that patients will receive infusions of their own T-cells, genetically modified, which their bodies will be less likely to reject.

Its individualized medicine, Lin said.

Im on my way

Before he tried CAR T at Mayo as part of a clinical trial, John Renze of Carroll, Iowa, had received two rounds of chemo, two rounds of radiation, and an experimental drug that did nothing to stop the spread of lymphoma.

After you fail about four times, you start to wonder if anything is going to work, the 58-year-old said.

At first, there was no room for him in the Mayo trial which has been a problem nationwide as desperate cancer patients have searched for treatment alternatives. But then he got the call one morning last summer while ordering coffee at his local cafe.

Can you get up here by one? the Mayo official asked.

Im on my way, Renze replied.

Even before federal approval comes through, researchers such as Miller are looking beyond the first-line CAR T therapies, and wondering if the approach can be used on solid tumors. Roughly 80,000 blood cancers occur each year in the U.S. that could be treated with CAR T, but the total number of cancers diagnosed each year is nearly 1.7 million.

The challenge is that solid tumors dont have the same protein targets as blood cancers. And T-cells would have to be more discriminating if infused to eliminate tumors in solid organs, Miller said. If you destroy normal lung tissue (along with lung cancer), thats not going to work, he said.

Mayo researchers are studying whether CAR T can work against multiple myeloma, a cancer of the bone marrow, while U researchers are exploring ways to better control the CAR T-cells after they are infused in cancer patients.

Researchers also are trying to understand whether CAR T produces memory in the immune system, so it knows to react if cancers resurface.

In addition, Miller is studying whether NK cells, which also play a role in the human immune system, can be genetically modified and infused instead of T-cells to target cancer. The body doesnt reject NK cells from donors as much, he said. So NK cells from donor bone marrow or umbilical cord blood could be collected and mass produced to potentially provide faster and cheaper treatments.

Like many breakthrough therapies, CAR T will be expensive, with a price likely to exceed $200,000 per patient. How insurers plan to cover it remains unclear. Blue Cross and Blue Shield of Minnesota is evaluating evidence regarding CAR Ts effectiveness, and will set a coverage policy after it receives FDA approval, said Dr. Glenn Pomerantz, Blue Cross chief medical officer.

A surge for Mayo?

Mayo expects a surge of hundreds of cancer patients per year if CAR T is approved, because it will initially be provided by large medical centers that have experience with the therapy and its side effects. The Rochester hospital is planning to add staff and space dedicated to CAR T.

Miller said the U is developing advice for referring doctors and hospitals statewide, so they know what to do if CAR T patients show up with complex symptoms.

They can be a bit delayed and you cant just keep people in the hospital to see if they develop these things, he said.

Renzes stem cells were taken last July, and his modified T-cells were put back a month later. He lost weight and felt sick for weeks, and had to drive three hours to Mayo for frequent checkups.

But as of last Aug. 31, the cancer had vanished.

Every three months, he returns to Mayo to make sure the cancer hasnt re-emerged. Then he returns to Carroll, where he owns farmland and car dealerships and dotes on his grandchildren.

For people like me that have already failed a bunch of times, youre happy to try anything, he said. I mean, what else would I have done?

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Mayo, U develop 'robocop' stem cells to fight cancer - StarTribune.com - Minneapolis Star Tribune

Representational image

Washington D.C. : A study has recently revealed that vitamin C may tell faulty stem cells in the bone marrow to mature and die normally, instead of multiplying to cause blood cancers.

According to researchers, certain genetic changes are known to reduce the ability of an enzyme called TET2 to encourage stem cells to become mature blood cells, which eventually die, in many patients with certain kinds of leukemia.

The new study found that vitamin C activated TET2 function in mice engineered to be deficient in the enzyme.

Corresponding study author Benjamin G. Neel said, "We're excited by the prospect that high-dose vitamin C might become a safe treatment for blood diseases caused by TET2-deficient leukemia stem cells, most likely in combination with other targeted therapies."

The results suggested that changes in the genetic code (mutations) that reduce TET2 function are found in 10 percent of patients with acute myeloid leukemia (AML), 30 percent of those with a form of pre-leukemia called myelodysplastic syndrome, and in nearly 50 percent of patients with chronic myelomonocytic leukemia.

The study results revolve around the relationship between TET2 and cytosine, one of the four nucleic acid "letters" that comprise the DNA code in genes.

To determine the effect of mutations that reduce TET2 function in abnormal stem cells, the team genetically engineered mice such that the scientists could switch the TET2 gene on or off.

The findings indicated that vitamin C did the same thing as restoring TET2 function genetically. By promoting DNA demethylation, high-dose vitamin C treatment induced stem cells to mature, and also suppressed the growth of leukemia cancer stem cells from human patients implanted in mice.

"Interestingly, we also found that vitamin C treatment had an effect on leukemic stem cells that resembled damage to their DNA," said first study author Luisa Cimmino.

"For this reason, we decided to combine vitamin C with a PARP inhibitor, a drug type known to cause cancer cell death by blocking the repair of DNA damage, and already approved for treating certain patients with ovarian cancer," Cimmino added.

The findings appear in journal Cell.

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Vitamin C may help genes to kill blood cancer stem cells - ETHealthworld.com

Yaron Ramati, Director of Regulatory Affairs at Pluristem Therapeutics

Over the past few years, the regulatory landscape for cell therapy development has grown increasingly complex. There are now accelerated pathways for advanced therapy medicinal products (ATMPs) in several countries worldwide, including the U.S., Japan, and South Korea. While the possibility for accelerated commercialization has resulted from these changes, substantial complexity has also been introduced, making it a more elaborate process to move cell therapy products from bench to bedside.

In the interview with Yaron Ramati, Director of Regulatory Affairs at Pluristem Therapeutics, we get an experts perspective on how the regulatory environment has changed and new opportunities that exist for bringing cell therapy products through the clinical trial process and into the global marketplace.

Yaron Ramati: I have 10 years of experience in regulatory affairs in biotechnology companies in Israel.

I have a PhD in Philosophy of Biology from the London School of Economics and an M.Sc. from the Technion in Neurobiology

Yaron Ramati:The United States, Japan, and South Korea are countries that have accelerated pathways that are unique for cell and gene therapies. Legislation took effect in Japan in late 2014, in South Korea in 2016, and in the United States in 2017.

Additionally, the EU has a program for product acceleration the Adaptive Pathways. Although it is not explicitly for cell and gene therapies, these have been given a lot of attention by the group.

Yaron Ramati:

In the United States: Regenerative medicine advanced therapy (RMAT) designation.Cell therapies that aim to treat serious medical conditions with high unmet need, and have preliminary favorable clinical data, can get the designation. It allows for accelerated approval (i.e., the use of biomarkers and intermediate endpoints for BLA, priority review).

In Japan: Conditional time-limited marketing authorization.This program allows for regenerative therapies (cell, gene and tissue therapies) to receive conditional marketing authorization for up to 7 years, following confirmation of safety and an initial proof of efficacy in Japan in diseases that are serious and have a high unmet need.

In South Korea: Conditional marketing authorization for cell therapy.As in Japan, this program allows for cell therapies to receive conditional marketing authorization for a limited time, following an initial proof of efficacy in serious diseases.

In EU: Adaptive Pathways pilot program. This program is a pilot program established by the EMA to explore ways in which the EMA can assist the streamlining the development of new promising therapies for serious conditions with high unmet need. Although this program is not explicitly for cell or gene therapy, it is the main focus of the group.

Yaron Ramati: All EU countries have a joint definition for ATMPs as set by EU regulation. Other countries have separate definitions that only partially overlap.

Yaron Ramati: Only few countries in the world are willing to be the first to provide marketing authorization for novel therapies. For ATMPs, European regulation does not allow individual countries in the union to provide marketing authorization, and so the EMA is the only gateway for ATMPs in Europe.

The U.S. FDA, Japan PMDA, and South Korea KFDA are the only others that are willing to be first to approve ATMPs.

Yaron Ramati: Currently, the EMA and PMDA are leading with four marketing approvals of cell and gene therapies each. RMAT designation procedure in the U.S. is expecting to give a boost to the products that are being developed for the U.S. market.

Yaron Ramati: Pluristem is very active in the field of accelerated development of its products. PLX-PAD of Pluristem has been accepted to the Japan conditional time-limited marketing authorization scheme by PMD, as well as to the adaptive pathways program of the EMA. It is active in both programs.

In addition, Pluristem intends to make use of the accelerated pathways offered for regenerative therapies in both the U.S. and in South Korea.

Yaron Ramati: The focus of Pluristem in these programs is the advancement of PLX-PAD. Pluristem had achieved understandings with EMA and PMDA regarding the accelerated approval of PLX-PAD for the treatment of critical limb ischemia (CLI).

It is the intention of Pluristem to achieve similar understandings with FDA, EMA, PMDA and KFDA regarding the development of PLX-PAD for the treatment of patients following hip fractures.

Yaron Ramati: PLX-PAD was accepted into the EMA adaptive pathways pilot program in 2015. Since then, Pluristem has taken advantage of this program in coming to an understanding with the EMA on the desired regulatory path of PLX-PAD in CLI. In addition, Pluristem undertook parallel scientific advice with the EMA and leading health technology assessment (HTA) bodies in Europe.

In this meeting, Pluristem received valuable feedback on the expectations that these bodies have for purposes of reimbursement in Europe. Pluristem has designed the Phase 3 PACE study in CLI patients in view of the feedback received from both the EMA and the HTA bodies, with the purpose of addressing their respective expectations.

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Those suffering from hair loss problems could soon be worry free thanks to a bunch of researchers at UCLA. The team found that by activating the stem cells in the hair follicles they could make it grow. This type of research couldnt come soon enough for some. We may have finally found a cure for patients suffering from alopecia or baldness.

Hair loss is often caused by the hair follicle stem cells inability to activate and induce a new hair growth cycle. In doing the study, researchers Heather Christofk and William Lowry, of Eli Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCLA discovered that the metabolism of hair follicle stem cells is far different to any other cell found within the skin. They found that as hair follicle stem cells absorb the glucose from the bloodstream they use it to produce a metabolite called pyruvate. The pyruvate is then either sent to the cells mitochondria to be converted back into energy or is converted into another metabolite called lactate.

Christofk is an associate professor of biological chemistry and molecular and medical pharmacology and he says, Our observations about hair follicle stem cell metabolism prompted us to examine whether genetically diminishing the entry of pyruvate into the mitochondria would force hair follicle stem cells to make more lactate and if that would activate the cells and grow hair more quickly. First, the team demonstrated how blocking the lactate production in mice prevented the hair follicle stem cells from activating. Then, with the help of colleagues at the Rutter lab at the University of Utah, they increased the lactate production in the mice and as a result saw an accelerated hair follicle stem cell activation and therefore an increase in the hair cycle.

Once we saw how altering lactate production in the mice influenced hair growth, it led us to look for potential drugs that could be applied to the skin and have the same effect, confirms Lowry, a professor of molecular, cell and developmental biology. During the study, the team found two drugs in particular that influenced hair follicle stem cells to promote lactate production when applied to the skin of mice. The first is called RCGD423. This drug is responsible for allowing the transmission of information from outside the cell right to the heart of it in the nucleus by activating the cellular signaling pathway called JAK-Stat. The results from the study did, in fact, prove that JAK-Stat activation will lead to an increased production of lactate which will enhance hair growth. UK5099 was the second drug in question, and its role was to block the pyruvate from entering the mitochondria, forcing the production of lactate and accelerating hair growth as a result.

The study brings with it some very promising results. To be able to solve a problem that affects millions of people worldwide by using drugs to stimulate hair growth is brilliant. At the moment there is a provisional patent application thats been filed in respect of using RCGD423 in the promotion of hair growth and a separate provisional patent in place for the use of UK5099 for the same purpose. The drugs have not yet been tested in humans or approved by the Food and Drug Administration as fit for human consumption.

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Scientists Discover New Hair Growth Technique Using Stem Cells ... - TrendinTech

Cardiac stem cells derived from young hearts helped reverse the signs of aging when directly injected into the old hearts of elderly rats, astudypublished Monday in the European Heart Journal demonstrated.

The old rats appeared newly invigorated after receiving their injections. As hoped, the cardiac stem cells improved heart function yet also provided additional benefits. The rats fur, shaved for surgery, grew back more quickly than expected, and their chromosomal telomeres, which commonly shrink with age, lengthened.

Its extremely exciting, said Dr. Eduardo Marbn, primary investigator on the research and director of the Cedars-Sinai Heart Institute. Witnessing the systemic rejuvenating effects, he said, its kind of like an unexpected fountain of youth.

The working hypothesis is that the cells secrete exosomes, tiny vesicles that contain a lot of nucleic acids, things like RNA, that can change patterns of the way the tissue responds to injury and the way genes are expressed in the tissue, Marbn said.

It is the exosomes that act on the heart and make it better as well as mediating long-distance effects on exercise capacity and hair regrowth, he explained.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Unexpected fountain of youth found in cardiac stem cells, says researcher

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Are cardiac stem cells a 'fountain of youth'? | Genetic Literacy Project - Genetic Literacy Project

On Monday, a group of scientists at Cedars-Sinai Heart Institute in Los Angeles, CA, discovered througha world-first experimenta form to rejuvenate elder rats old hearts by injecting cardiac stem cells from much younger rats with healthier hearts. They hope this process might eventually become useful to humans.

The first time an experiment like this was carried out was in 2009 by the same Los Angeles-based team. Now, they also proved the possibility of reversing aging in old hearts.

Heart failure is a typical cause of death in humans. Around 48 percent of women and 46 percent of men die a year from heart attacks and other heart-related diseases. They are the first reason of death worldwide, and a leading cause of death in the United States, killing over 375,000 Americans a year. Nearly half of all African-American population suffers from heart diseases.

Researchers took stem cells from the hearts of 4-month-old rats, shaped them into cardiosphere-derived cells and injected them into the hearts of other rats of 22 monthsold, an age that makes them be considered as old. They carried out a similar process to another group of rats but injected saline instead. Scientists later compared both groups.

After receiving the stem cells injection, researchers noted a significant change in the way old rats continued to live. They turned much more active and improved their functionalities. Not just their heart rates got better and faster, but also the way they ran and breathed. Their hair started to grow faster, their chromosomal telomeres which commonly shrink with age lengthened, plus other benefits. The rodents began to progressively improve their capacity of exercise along with their stamina overall.

The animals could exercise further than they could before by about 20%, and one of the most striking things, especially for me (because Im kind of losing my hair) the animals regrew their fur a lot better after theyd gotten cells compared with the placebo rats, said Dr Eduardo Marbn, director of the Cedars-Sinai Heart Institute and lead author, who is also extremely excited for having witnessed the unexpected fountain of youth.

In 2009, his team successfully repaired the damaged heart of a man who had suffered a heart attack, using his own heart tissue.

Stem cells are a really basic type of cells that can be molded and converted into other much-specialized cells through a process called differentiation, which is basicallyshaping them into any kind of body cell.They form in embryos like embryonic stem cells -, which help in the growth process of babies, along with the millions of other different cell types they need before their birth.

One of many cells scientists generated from stem cells is called progenitor cell, which shares some of the same properties. But unlike the original cells, progenitor cells are not able to divide and reproduce indefinitely. Dr. Marbn also said they discovered cardiosphere-derived cells, which tend to promote the healing of a condition that affects more than 50 percent of patients suffering from heart failure.

Our previous lab studies and human clinical trials have shown promise in treating heart failure using cardiac stem cell infusions, said Dr Marbn. Now we find that these specialized stem cells could turn out to reverse problems associated with aging of the heart.

According to Dr. Marbn, stem cells secrete exosomes, tiny vesicles which contain a lot of nucleic acids, things like RNA, that can change patterns of the way the tissue responds to injuries, and the way genes are expressed in the tissue. They are placed into the heart, and act to transform it into a better organ, helping it at the same time to improve exercise capacity and hair regrowth, he explained.

Now, Dr. Marbn is exploring a much easier way to deliver the stem cells intravenously, instead of injecting them directly into the heart. Thus avoiding surgeries, which tend to be more complicated and expensive for the patient.

Striking benefits are demonstrated not only from a cardiac perspective but across multiple organ systems, said Dr. Gary Gerstenblith, a professor of medicine in the cardiology division of Johns Hopkins Medicine, who did not contribute to the new research. The results suggest that stem cell therapies should be studied as an additional therapeutic option in the treatment of cardiac and other diseases common in the elderly.

Now, scientistsneed to make more extensive studies before using the technique in humans.

Source: CNN

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Scientists discovered how to rejuvenate rats by injecting stem cells ... - Pulse Headlines

18 Aug 2017

Astrocytes are more than bystanders in neurotransmissionthey take an active role in synaptic activity. However, their functions are hard to study because the cells are difficult to grow in vitro and its hard to coax them to mature from progenitors. Now, researchers from the labs of Sergiu Paca and Ben Barres, both at Stanford University School of Medicine, California, report that astrocytes come of age in spherical balls of human brain cells cultured in a dish for almost two years. As reported in the August 16 Neuron, these astrocytes develop much like those from real brains, undergoing similar transcriptomic, morphologic, and functional changes. Studying the processes involved in this astrocyte maturation will help researchers understand neurodevelopmental disorders such as autism and schizophrenia, researchers say, and might even shed light on problems in adultbrains.

That these 3D cultures can be maintained for such a long time allows us to capture an interesting transition in astrocytes, said Paca. We are starting to appreciate aspects of human brain development to which we would not otherwise haveaccess.

The breakthrough is that they can develop human astrocytes very close to maturity in their 3D culture models, said Doo Yeon Kim, Massachusetts General Hospital, Charlestown, who uses 3D culture models to study pathological process that occur in Alzheimers disease. Some researchers are using 3D cultures to model other neurodegenerative disorders, such as ALS, and still others are planning to use cultured astrocytes for cell therapy. If astrocytes are not mature enough in culture, patterns [we see] may not be the same as in the diseased brain, saidKim.

This developing human astrocyte (red), which comes from a 350-day-old cortical spheroid, is taking shape as a mature cell. [Image courtesy of Sloan et al.Neuron]

A few years back, Pacas group developed a method for differentiating human induced pluripotent stem cells (hiPSCs) into a 3D culture of brain cells. They used special dishes that the cells could not easily attach to, coaxing them to stick to each other instead. Under these conditions the iPSCs balled up into neural spheroids that grew to about 4 mm in diameter. A cocktail of growth factors early on encouraged them to form excitatory pyramidal cells like those in the cortex, and the cells spontaneously organized into layers. These cortical spheroids survived a year or more and spontaneously grew astrocytes in addition to neurons (Paca et al., 2015). Not long after, the Barres lab reported that astrocytes in the adult human brain look different from those isolated from fetuses. They called the latter astrocyte progenitor cells (APCs). Each had their own transcriptional patterns and functions (Jan 2016 news). Together, Barres and Paca wondered if it was possible to see the APCs morph into mature astrocytes in these long-lived corticalspheroids.

To find out, first author Steven Sloan and colleagues examined spheroids generated from iPSCs derived from healthy human fibroblasts. Sloan grew the spheroids for about 20 months. Along the way, he took samples, isolated the astrocytes, and compared them to those isolated from fetal and postnatal humanbrain.

At about 100 days in culture, astrocytes began to sprout spontaneously from within the mostly neuronal milieu of the cortical spheroids. At first, these cells were simple, adorned by few branches and expressing genes akin to those active in APCs. But as the spheroids reached about 250 days, the astrocytes therein looked more mature, having numerous processes. After this point, APC gene expression tapered off and the astrocytes started producing proteins typical of matureastrocytes.

Astrocytes also underwent functional changes as they matured. Early versions divided in fast and furious fashion, much like their counterparts from the fetal tissue. That division slowed as the spheroids aged. Dividing APCs dropped from 35 percent of all astrocytes at day 167 to 3 percent at day 590. Taken from the spheroids at day 150 and cultured in a 2D layer, immature astrocytes also harbored a voracious appetite for added synaptosomes, much like immature astrocytes recently characterized in mice (see image below; Dec 2013 conference news on Chung et al., 2013). However, that hunger waned as astrocytes approached the 590-daymark.

At the older end of the spectrum, mature astrocytes seemed to take on a supportive role, strengthening calcium signaling in nearbyneurons.

Studying the neurons and astrocytes in these cortical spheroids could be useful for addressing certain unanswered questions about human biology, said other researchers. This could be a very strong opportunity to understand what goes wrong in human genetic disorders that affect astrocyte function, said M. Kerry OBanion, University of Rochester Medical Center, New York. Its also possible that such cultures could reveal as yet unknown facets of familial mutations that cause Alzheimers disease, he suggested. However, given that these cultures take a long time to grow and develop, they are unlikely to completely supplant other types of cultures or faster-maturing animal models, hesaid.

Kim agreed, saying, The results are very exciting, but not practical yet for disease modeling." However, Kim hopes that researchers will make progress on accelerating the maturationprocess.

The Barres and Paca labs are trying just that with the spheroid. They will also analyze what they secrete to support neuronal signaling. In addition, they are exploring how to make the astrocytes reactive, as they often are in neurodegenerative diseases, such as Alzheimers. Doing so might reveal how such astrocytes interact withneurons.

An immature astrocyte taken from a 150-day-old spheroid gobbles up added synaptosomes (red). [Neuron, Sloan et al.2017]

To Pacas knowledge, these cortical spheroids are some of the longest human cell cultures ever reported. His group has continued to cultivate these clumps, with the oldest still going strong at day 850. Granted, these systems are missing many cell types: endothelial cells, oligodendrocytes, and microglia to name a few, he said. However, his lab has introduced new ways to add in other cells. Earlier this year, he reported 3D cultures of cortical glutamatergic neurons and GABAergic interneurons that fused together when they were placed side-by-side (Birey et al., 2017).

Clive Svendsen, Cedars-Sinai Medical Center in Los Angeles, California, saw clinical implications for this paper. It shows iPSC derived astrocytes can mature to an adult phenotype, he said. This further supports their use in clinical transplantation, as we are planning to do. His group has begun a Phase 1 clinical trial that implants human fetal astrocytes into the spinal cords of ALS patients.Gwyneth DickeyZakaib

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Brain Spheroids Hatch Mature Astrocytes - Alzforum

A pregnant British mom hopes she and her unborn baby will be the answer to help prolong her ailing brothers life.

Georgina Russell, of Preston, England, said she was desperate to help her brother, Ashley, when doctors diagnosed him with a slow-growing but deadly brain tumor earlier this year, according to the Daily Mail.

Georgina said she began researching his condition, glioblastoma, online and looking for answers that could save his life. She found one: her pregnancy.

Stem cells produced in the umbilical cord between her and her unborn baby potentially could be used in a treatment to shrink Ashleys tumor, according to the report. Once Georgina gives birth, she said doctors will be able to harvest and store the stem cells until Ashley needs them.

There is no harm to the baby or the mother when doctors harvest stem cells from the umbilical cord unlike embryonic stem cells, which only can be taken by killing a human life in the embryonic stage.

Georgina told the Mail: The blood from the cord is being used in trials across the world. It can do amazing things to help the body repair itself. If we store the stem cells, they can be kept to be used throughout Ashleys treatment when he needs them.

They might be able to inject them into the spinal fluid, to shrink the tumour on the brain, or they may be able to use the tissue grown from them to repair any damage to other parts of his body, if he has to have chemotherapy or radiotherapy.

Ashley Russell, a British military veteran, husband and father, said doctors found the tumor after he began suffering from headaches, dizzy spells and mini-seizures about six months ago. Later, he said he also began having blurred vision. Doctors ran a series of tests before discovering the tumor on his brain.

He said doctors suggested surgery, but the procedure has high risks. They gave him about five years to live, according to the report.

Georgina said she was devastated for her brother and his family, and she began researching ways to help him. In her research online, she said she discovered how stem cells collected from the umbilical cord are helping to treat people with tumors and other diseases.

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Her brother said the idea seemed odd at first, but he is willing to try anything.

I am quite a positive person so although the diagnosis was difficult, I am determined to do whatever I can to keep going, Ashley said. I did think about not being around to see my little girl get married and knew that if there was anything that might help, I would give it a go.

Georgina currently is 33 weeks pregnant with her unborn child, the report states.

Stem cells are so powerful and his new niece or nephew could save his life, she said.

The family set up a JustGiving page to help pay for the storage of the stem cells and Ashleys treatment.

Adult stem cells and those from umbilical cords are proving to be live-saving, while life-destroying embryonic stem cells have not been effective.

David Prentice, vice president and research director for the Charlotte Lozier Institute, explained more about the effectiveness of these life-saving stem cells in 2014:

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Umbilical cord blood stem cells have become an extremely valuable alternative to bone marrow adult stem cell transplants, ever since cord blood stem cells were first used for patients over 25 years ago. The first umbilical cord blood stem cell transplant was performed in October 1988, for a 5-year-old child with Fanconi anemia, a serious condition where the bone marrow fails to make blood cells. That patient is currently alive and healthy, 25 years after the cord blood stem cell transplant.

Prentice said more than 30,000 cord blood stem cell transplants have been done across the world. These stem cells have helped treat people with blood and bone marrow diseases, leukemia and genetic enzyme diseases, he said.

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Twelve years ago, Robb Martin was an active police officer with Prescott Police Department when a recreational accident left him paralyzed from the chest down.

I was on a four-wheeler in the sand dunes, Martin, 42, said. I was on my way back to camp just putting along when I hit a bump. It threw me off the front, my helmet got stuck in the sand, my legs just kept going and I broke my back right at the chest level.

After getting out of the hospital and going through some rehabilitation to get his arms, shoulders and neck moving normally, he continued to work for the police department in the dispatch center and has been there ever since.

Despite his condition, Martin has remained incredibly active.

The guy is always busy, said Tom Newell, a longtime friend of Martins.

With some help from his friends, he managed to build a workshop on his property and is consistently in there modifying objects to fit his needs or assisting friends and family with various projects.

If hes not helping his wife with her business, hes in his shop welding something, making something or building something to help somebody else out, Newell said.

Since the accident, Martin has looked for ways to improve his mobility. Physical therapy has been helping, allowing him to regain back and stomach muscles in recent years.

I can do pushups and actually support my waist, which is amazing, he said.

His goal, however, is to once again be on his feet.

Just to even stand up and grab something out of a cabinet would be phenomenal, Martin said.

That dream might come true if he can raise the funds to have a recently developed procedure done in Thailand by a company called Unique Access.

The procedure, referred to as epidural stimulation, involves surgically implanting a device along a damaged portion of the nervous system, according to the companys website. The device then applies a continuous electrical current.

It acts kind of like a jumper cable, for lack of a better term, Martin said. It just connects above the affected area and allows the brain to reconnect with the spinal cord under the affected area.

In combination with the implant, several million stem cells are injected into the area to help the regenerative process. These, as well as

an assisted rehabilitation process, take about 40 days to complete.

The procedure has yet to be seriously implemented in the U.S., Martin said, because of how new it is to the medical industry. So far, however, he hasnt heard of any unusual risks associated with the procedure and has spoken with two individuals who successfully went through it.

One guy is walking up to 30 meters unassisted, Martin said. Another guy, the day after surgery, he was standing up by himself in a pool.

Altogether, Martin said its going to cost him $100,000 out of pocket.

Not able to afford that between him and his wife, hes turned to the community for help. Friends and family have already been busy contributing and organizing events.

Just last Saturday, Aug. 12, about $5,000 was raised on his behalf from two fundraising events hosted by his friends Tony and Liko Harwood.

Tony wanted to be involved and couldnt just sit still and not make any money for Rob so here we are, Liko said Saturday during one of the events.

Another $2,000 was raised from a donation bucket placed inside Scouts Gourmet Grub in Prescott.

Quite a bit more was also raised by fundraisers hosted by the Northern Arizona Regional Training Academy (NARTA), the local police academy.

Sitting at about $15,000, Martin is hoping to continue raising money in whatever way he can to reach the full $100,000.

My surgery is approved, theyre just waiting for me to set up a date, Martin said. The funding is really all Im waiting on.

For more information about Martins story and to donate, go to RobbMartin.com.

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Disabled former police officer raising money for operation in Thailand - The Daily Courier

In a new studyin Nature Communications, Stephanopoulos and his colleague Ronit Freeman successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways. Credit: Northwestern University

What if one day, we could teach our bodies to self-heal like a lizard's tail, and make severe injury or disease no more threatening than a paper cut?

Or heal tissues by coaxing cells to multiply, repair or replace damaged regions in loved ones whose lives have been ravaged by stroke, Alzheimer's or Parkinson's disease?

Such is the vision, promise and excitement in the burgeoning field of regenerative medicine, now a major ASU initiative to boost 21st-century medical research discoveries.

ASU Biodesign Institute researcher Nick Stephanopoulos is one of several rising stars in regenerative medicine. In 2015, Stephanopoulos, along with Alex Green and Jeremy Mills, were recruited to the Biodesign Institute's Center for Molecular Design and Biomimetics (CMDB), directed by Hao Yan, a world-recognized leader in nanotechnology.

"One of the things that that attracted me most to the ASU and the Biodesign CMDB was Hao's vision to build a group of researchers that use biological molecules and design principles to make new materials that can mimic, and one day surpass, the most complex functions of biology," Stephanopoulos said.

"I have always been fascinated by using biological building blocks like proteins, peptides and DNA to construct self-assembled structures, devices and materials, and the interdisciplinary and highly collaborative team in the CMDB is the ideal place to put this vision into practice."

Yan's research center uses DNA and other basic building blocks to build their nanotechnology structuresonly at a scale 1,000 times smaller than the width of a human hair.

They've already used nanotechnology to build containers to specially deliver drugs to tissues, build robots to navigate a maze or nanowires for electronics.

To build a manufacturing industry at that tiny scale, their bricks and mortar use a colorful assortment of molecular Legos. Just combine the ingredients, and these building blocks can self-assemble in a seemingly infinite number of ways only limited by the laws of chemistry and physicsand the creative imaginations of these budding nano-architects.

Learning from nature

"The goal of the Center for Molecular Design and Biomimetics is to use nature's design rules as an inspiration in advancing biomedical, energy and electronics innovation through self-assembling molecules to create intelligent materials for better component control and for synthesis into higher-order systems," said Yan, who also holds the Milton Glick Chair in Chemistry and Biochemistry.

Prior to joining ASU, Stephanopoulos trained with experts in biological nanomaterials, obtaining his doctorate with the University of California Berkeley's Matthew Francis, and completed postdoctoral studies with Samuel Stupp at Northwestern University. At Northwestern, he was part of a team that developed a new category of quilt-like, self-assembling peptide and peptide-DNA biomaterials for regenerative medicine, with an emphasis in neural tissue engineering.

"We've learned from nature many of the rules behind materials that can self-assemble. Some of the most elegant complex and adaptable examples of self-assembly are found in biological systems," Stephanopoulos said.

Because they are built from the ground-up using molecules found in nature, these materials are also biocompatible and biodegradable, opening up brand-new vistas for regenerative medicine.

Stephanopoulos' tool kit includes using proteins, peptides, lipids and nucleic acids like DNA that have a rich biological lexicon of self-assembly.

"DNA possesses great potential for the construction of self-assembled biomaterials due to its highly programmable nature; any two strands of DNA can be coaxed to assemble to make nanoscale constructs and devices with exquisite precision and complexity," Stephanopoulos said.

Proof all in the design

During his time at Northwestern, Stephanopoulos worked on a number of projects and developed proof-of-concept technologies for spinal cord injury, bone regeneration and nanomaterials to guide stem cell differentiation.

Now, more recently, in a new study in Nature Communications, Stephanopoulos and his colleague Ronit Freeman in the Stupp laboratory successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways.

In the new technology, materials are first chemically decorated with different strands of DNA, each with a unique code for a different signal to cells.

To activate signals within the cells, soluble molecules containing complementary DNA strands are coupled to short protein fragments, called peptides, and added to the material to create DNA double helices displaying the signal.

By adding a few drops of the DNA-peptide mixture, the material effectively gives a green light to stem cells to reproduce and generate more cells. In order to dynamically tune the signal presentation, the surface is exposed to a soluble single-stranded DNA molecule designed to "grab" the signal-containing strand of the duplex and form a new DNA double helix, displacing the old signal from the surface.

This new duplex can then be washed away, turning the signal "off." To turn the signal back on, all that is needed is to now introduce a new copy of single-stranded DNA bearing a signal that will reattach to the material's surface.

One of the findings of this work is the possibility of using the synthetic material to signal neural stem cells to proliferate, then at a specific time selected by the scientist, trigger their differentiation into neurons for a while, before returning the stem cells to a proliferative state on demand.

One potential use of the new technology to manipulate cells could help cure a patient with neurodegenerative conditions like Parkinson's disease.

The patient's own skin cells could be converted to stem cells using existing techniques. The new technology could help expand the newly converted stem cells back in the laband then direct their growth into specific dopamine-producing neurons before transplantation back to the patient.

"People would love to have cell therapies that utilize stem cells derived from their own bodies to regenerate tissue," Stupp said. "In principle, this will eventually be possible, but one needs procedures that are effective at expanding and differentiating cells in order to do so. Our technology does that."

In the future, it might be possible to perform this process entirely within the body. The stem cells would be implanted in the clinic, encapsulated in the type of material described in the new work, and injected into a particular spot. Then the soluble peptide-DNA molecules would be given to the patient to bind to the material and manipulate the proliferation and differentiation of transplanted cells.

Scaling the barriers

One of the future challenges in this area will be to develop materials that can respond better to external stimuli and reconfigure their physical or chemical properties accordingly.

"Biological systems are complex, and treating injury or disease will in many cases necessitate a material that can mimic the complex spatiotemporal dynamics of the tissues they are used to treat," Stephanopoulos said.

It is likely that hybrid systems that combine multiple chemical elements will be necessary; some components may provide structure, others biological signaling and yet others a switchable element to imbue dynamic ability to the material.

A second challenge, and opportunity, for regenerative medicine lies in creating nanostructures that can organize material across multiple length scales. Biological systems themselves are hierarchically organized: from molecules to cells to tissues, and up to entire organisms.

Consider that for all of us, life starts simple, with just a single cell. By the time we reach adulthood, every adult human body is its own universe of cells, with recent estimates of 37 trillion or so. The human brain alone has 100 billion cells or about the same number of cells as stars in the Milky Way galaxy.

But over the course of a life, or by disease, whole constellations of cells are lost due to the ravages of time or the genetic blueprints going awry.

Collaborative DNA

To overcome these obstacles, much more research funding and recruitment of additional talent to ASU will be needed to build the necessary regenerative medicine workforce.

Last year, Stephanopoulos' research received a boost with funding from the U.S. Air Force's Young Investigator Research Program (YIP).

"The Air Force Office of Scientific Research YIP award will facilitate Nick's research agenda in this direction, and is a significant recognition of his creativity and track record at the early stage of his careers," Yan said.

They'll need this and more to meet the ultimate challenge in the development of self-assembled biomaterials and translation to clinical applications.

Buoyed by the funding, during the next research steps, Stephanopoulos wants to further expand horizons with collaborations from other ASU colleagues to take his research team's efforts one step closer to the clinic.

"ASU and the Biodesign Institute also offer world-class researchers in engineering, physics and biology for collaborations, not to mention close ties with the Mayo Clinic or a number of Phoenix-area institutes so we can translate our materials to medically relevant applications," Stephanopoulos said.

There is growing recognition that regenerative medicine in the Valley could be a win-win for the area, in delivering new cures to patients and building, person by person, a brand-new medicinal manufacturing industry.

Explore further: New technology to manipulate cells could help treat Parkinson's, arthritis, other diseases

More information: Ronit Freeman et al. Instructing cells with programmable peptide DNA hybrids, Nature Communications (2017). DOI: 10.1038/ncomms15982

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Bio-inspired materials give boost to regenerative medicine - Medical Xpress

Stem cell science involves many technical terms. This glossary covers many of the common terms you will encounter in reading about stem cells.

Adult stem cellsA commonly used term for tissue-specific stem cells, cells that can give rise to the specialized cells in specific tissues. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent stem cells.

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AutologousCells or tissues from the same individual; an autologous bone marrow transplant involves one individual as both donor and recipient.

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Basic researchResearch designed to increase knowledge and understanding (as opposed to research designed with the primary goal to solve a problem).

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BlastocystA transient, hollow ball of 150 to 200 cells formed in early embryonic development that contains the inner cell mass, from which the embryo develops, and an outer layer of cell called the trophoblast, which forms the placenta.

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Bone marrow stromal cellsA general term for non-blood cells in the bone marrow, such as fibroblasts, adipocytes (fat cells) and bone- and cartilage-forming cells that provide support for blood cells. Contained within this population of cells are multipotent bone marrow stromal stem cells that can self-renew and give rise to bone, cartilage, adipocytes and fibroblasts.

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CardiomyocytesThe functional muscle cells of the heart that allow it to beat continuously and rhythmically.

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Clinical translationThe process of using scientific knowledge to design, develop and apply new ways to diagnose, stop or fix what goes wrong in a particular disease or injury; the process by which basic scientific research becomes medicine.

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Clinical trialTests on human subjects designed to evaluate the safety and/or effectiveness of new medical treatments.

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Cord bloodThe blood in the umbilical cord and placenta after child birth. Cord blood contains hematopoietic stem cells, also known as cord blood stem cells, which can regenerate the blood and immune system and can be used to treat some blood disorders such as leukemia or anemia. Cord blood can be stored long-term in blood banks for either public or private use. Also called umbilical cord blood.

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CytoplasmFluid inside a cell, but outside the nucleus.

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DifferentiationThe process by which cells become increasingly specialized to carry out specific functions in tissues and organs.

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Drug discoveryThe systematic process of discovering new drugs.

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Drug screeningThe process of testing large numbers of potential drug candidates for activity, function and/or toxicity in defined assays.

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EmbryoGenerally used to describe the stage of development between fertilization and the fetal stage; the embryonic stage ends 7-8 weeks after fertilization in humans.

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Embryonic stem cells (ESCs)Undifferentiated cells derived from the inner cell mass of the blastocyst; these cells have the potential to give rise to all cell types in the fully formed organism and undergo self-renewal.

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FibroblastA common connective or support cell found within most tissues of the body.

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GlucoseA simple sugar that cells use for energy.

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HematopoieticBlood-forming; hematopoietic stem cells give rise to all the cell types in the blood.

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ImmunomodulatoryThe ability to modify the immune system or an immune response.

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Induced pluripotent stem cells (iPSCs)Embryonic-like stem cells that are derived from reprogrammed, adult cells, such as skin cells. Like ESCs, iPS cells are pluripotent and can self-renew.

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In vitroLatin for in glass. In biomedical research this refers to experiments that are done outside the body in an artificial environment, such as the study of isolated cells in controlled laboratory conditions (also known as cell culture).

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In vivoLatin for within the living. In biomedical research this refers to experiments that are done in a living organism. Experiments in model systems such as mice or fruit flies are an example of in vivo research.

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Islets of LangerhansClusters in the pancreas where insulin-producing beta cells live.

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MaculaA small spot at the back of the retina, densely packed with the rods and cones that receive light, which is responsible for high-resolution central vision.

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Mesenchymal stem cells (MSCs)A term used to describe cells isolated from the connective tissue that surrounds other tissues and organs. MSCs were first isolated from the bone marrow and shown to be capable of making bone, cartilage and fat cells. MSCs are now grown from other tissues, such as fat and cord blood. Not all MSCs are the same and their characteristics depend on where in the body they come from and how they are isolated and grown. May also be called mesenchymal stromal cells.

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Multipotent stem cellsStem cells that can give rise to several different types of specialized cells in specific tissues; for example, blood stem cells can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain.

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NeuronAn electrically excitable cell that processes and transmits information through electrical and chemical signals in the central and peripheral nervous systems.

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Pancreatic beta cellsCells responsible for making and releasing insulin, the hormone responsible for regulating blood sugar levels. Type I diabetes occurs when these cells are attacked and destroyed by the body's immune system.

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PhotoreceptorsRod or cone cells in the retina that receive light and send signals to the optic nerve, which passes along these signals to the brain.

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PlaceboA pill, injection or other treatment that has no therapeutic benefit; often used as a control in clinical trials to see whether new treatments work better than no treatment.

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Placebo effectPerceived or actual improvement in symptoms that cannot be attributed to the placebo itself and therefore must be the result of the patient's (or other interested person's) belief in the treatment's effectiveness.

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Pluripotent stem cellsStem cells that can become all the cell types that are found in an embryo, fetus or adult, such as embryonic stem cells or induced pluripotent (iPS) cells.

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Preclinical researchLaboratory research on cells, tissues and/or animals for the purpose of discovering new drugs or therapies.

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Precursor cellsAn intermediate cell type between stem cells and differentiated cells. Precursor cells have the potential to give rise to a limited number or type of specialized cells. Also called progenitor cells.

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Progenitor cellsAn intermediate cell type between stem cells and differentiated cells. Progenitor cells have the potential to give rise to a limited number or type of specialized cells and have a reduced capacity for self-renewal. Also called precursor cells.

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Regenerative MedicineAn interdisciplinary branch of medicine with the goal of replacing, regenerating or repairing damaged tissue to restore normal function. Regenerative treatments can include cellular therapy, gene therapy and tissue engineering approaches.

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ReprogrammingIn the context of stem cell biology, this refers to the conversion of differentiated cells, such as fibroblasts, into embryonic-like iPS cells by artificially altering the expression of key genes.

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Retinal pigment epitheliumA single-cell layer behind the rods and cones in the retina that provide support functions for the rods and cones.

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RNARibonucleic acid; it "reads" DNA and acts as a messenger for carrying out genetic instructions.

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Scientific methodA systematic process designed to understand a specific observation through the collection of measurable, empirical evidence; emphasis on measurable and repeatable experiments and results that test a specific hypothesis.

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Self-renewalA special type of cell division in stem cells by which they make copies of themselves.

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Somatic stem cellsScientific term for tissue-specific or adult stem cells.

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Stem cellsCells that have both the capacity to self-renew (make more stem cells by cell division) and to differentiate into mature, specialized cells.

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Stem cell tourismThe travel to another state, region or country specifically for the purpose of undergoing a stem cell treatment available at that location. This phrase is also used to refer to the pursuit of untested and unregulated stem cell treatments.

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TeratomaA benign tumor that usually consists of several types of tissue cells that are foreign to the tissue in which the tumor is located.

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TissueA group of cells with a similar function or embryological origin. Tissues organize further to become organs.

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Tissue-specific stem cellsStem cells that can give rise to the specialized cells in specific tissues; blood stem cells, for example, can produce the different types of cells that make up the blood, but not the cells of other organs such as the liver or the brain. Includes all stem cells other than pluripotent stem cells such as embryonic and induced pluripotent cells. Also called adult or somatic stem cells.

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TotipotentThe ability to give rise to all the cells of the body and cells that arent part of the body but support embryonic development, such as the placenta and umbilical cord.

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Translational researchResearch that focuses on how to use knowledge gleaned from basic research to develop new drugs, treatments or therapies.

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ZygoteThe single cell formed when a sperm cell fuses with an egg cell.

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Stem Cell Glossary

Digital Trends

Want to live longer? Forever Labs wants to help, using your stem cells Digital Trends Using a patented device, Forever Labs collects stem cells from your blood marrow, which the team calls a wellspring for stem cells that replenish your blood, bone, immune system, and other vital tissues. The whole process is said to take around 15 ...

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Want to live longer? Forever Labs wants to help, using your stem cells - Digital Trends

The study suggests it may encourage blood cancer stem cells to die.

Researchers say Vitamin C may "tell" faulty stem cells in the bone marrow to mature and die normally, instead of multiplying to cause blood cancers.

They explained that certain genetic changes are known to reduce the ability of an enzyme called TET2 to encourage stem cells to become mature blood cells, which eventually die, in many patients with certain kinds of leukaemia.

The new study, published online by the journal Cell. found that vitamin C activated TET2 function in mice engineered to be deficient in the enzyme.

Study corresponding author Professor Benjamin Neel, of the Perlmutter Cancer Centre in the United States, said: "We're excited by the prospect that high-dose vitamin C might become a safe treatment for blood diseases caused by TET2-deficient leukemia stem cells, most likely in combination with other targeted therapies."

He said changes in the genetic code that reduce TET2 function are found in 10 per cent of patients with acute myeloid leukaemia (AML), 30 per cent of those with a form of pre-leukaemia called myelodysplastic syndrome, and in nearly 50 per cent of patients with chronic myelomonocytic leukaemia.

Such cancers cause anaemia, infection risk, and bleeding as abnormal stem cells multiply in the bone marrow until they interfere with blood cell production, with the number of cases increasing as the population ages.

Prof Neel said the study results revolve around the relationship between TET2 and cytosine, one of the four nucleic acid "letters" that comprise the DNA code in genes.

To determine the effect of mutations that reduce TET2 function in abnormal stem cells, the researchers genetically engineered mice such that the scientists could switch the TET2 gene on or off.

Similar to the naturally occurring effects of TET2 mutations in mice or humans, using molecular biology techniques to turn off TET2 in mice caused abnormal stem cell behaviour.

Prof Neel said, remarkably, the changes were reversed when TET2 expression was restored by a genetic trick.

Previous work had shown that vitamin C could stimulate the activity of TET2 and its relatives TET1 and TET3.

Because only one of the two copies of the TET2 gene in each stem cell is usually affected in TET2-mutant blood diseases, the researchers hypothesised that high doses of vitamin C, which can only be given intravenously, might reverse the effects of TET2 deficiency by turning up the action of the remaining functional gene.

They found that vitamin C did the same thing as restoring TET2 function genetically.

By promoting DNA demethylation, high-dose vitamin C treatment induced stem cells to mature, and also suppressed the growth of leukaemia cancer stem cells from human patients implanted in mice.

Study first author Doctor Luisa Cimmino, of New York University Langone Health, said: "Interestingly, we also found that vitamin C treatment had an effect on leukaemic stem cells that resembled damage to their DNA.

"For this reason, we decided to combine vitamin C with a PARP inhibitor, a drug type known to cause cancer cell death by blocking the repair of DNA damage, and already approved for treating certain patients with ovarian cancer."

The researchers found that the combination had an enhanced effect on leukaemia stem cells, further shifting them from self-renewal back toward maturity and cell death.

Dr Cimmino said the results also suggest that vitamin C might drive leukaemic stem cells without TET2 mutations toward death, given that it turns up any TET2 activity normally in place.

Corresponding author Professor Iannis Aifantis, also of NYU Langone Health, added: "Our team is working to systematically identify genetic changes that contribute to risk for leukaemia in significant groups of patients.

"This study adds the targeting of abnormal TET2-driven DNA demethylation to our list of potential new treatment approaches."

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Blood cancer: High doses of vitamin C could encourage stem cells ... - Express.co.uk

WASHINGTON D.C: Good news! A study has recently revealed that vitamin C may tell faulty stem cells in the bone marrow to mature and die normally, instead of multiplying to cause blood cancers.

According to researchers, certain genetic changes are known to reduce the ability of an enzyme called TET2 to encourage stem cells to become mature blood cells, which eventually die, in many patients with certain kinds of leukemia.

The new study found that vitamin C activated TET2 function in mice engineered to be deficient in the enzyme.

Corresponding study author Benjamin G. Neel said, "We're excited by the prospect that high-dose vitamin C might become a safe treatment for blood diseases caused by TET2-deficient leukemia stem cells, most likely in combination with other targeted therapies."

The results suggested that changes in the genetic code (mutations) that reduce TET2 function are found in 10 percent of patients with acute myeloid leukemia (AML), 30 percent of those with a form of pre-leukemia called myelodysplastic syndrome, and in nearly 50 percent of patients with chronic myelomonocytic leukemia.

The study results revolve around the relationship between TET2 and cytosine, one of the four nucleic acid "letters" that comprise the DNA code in genes.

To determine the effect of mutations that reduce TET2 function in abnormal stem cells, the team genetically engineered mice such that the scientists could switch the TET2 gene on or off.

The findings indicated that vitamin C did the same thing as restoring TET2 function genetically. By promoting DNA demethylation, high-dose vitamin C treatment induced stem cells to mature, and also suppressed the growth of leukemia cancer stem cells from human patients implanted in mice.

"Interestingly, we also found that vitamin C treatment had an effect on leukemic stem cells that resembled damage to their DNA," said first study author Luisa Cimmino.

"For this reason, we decided to combine vitamin C with a PARP inhibitor, a drug type known to cause cancer cell death by blocking the repair of DNA damage, and already approved for treating certain patients with ovarian cancer," Cimmino added.

The findings appear in journal Cell.

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Vitamin C could help genes kill blood cancer stem cells - Economic Times

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Bone Marrow Transplant gives local cancer patient more time with his family - KTBS

Scientists recently used a gene-editing tool to fix a mutation in a human embryo. Around the world, researchers are chasing cures for other genetic diseases.

Now that the gene-editing genie is out of the bottle, what would you wish for first?

Babies with perfect eyes, over-the-top intelligence, and a touch of movie star charisma?

Or a world free of disease not just for your family, but for every family in the world?

Based on recent events, many scientists are working toward the latter.

Earlier this month, scientists from the Oregon Health & Science University used a gene editing tool to correct a disease-causing mutation in an embryo.

The technique, known as CRISPR-Cas9, fixed the mutation in the embryos nuclear DNA that causes hypertrophic cardiomyopathy, a common heart condition that can lead to heart failure or cardiac death.

This is the first time that this gene-editing tool has been tested on clinical-quality human eggs.

Had one of these embryos been implanted into a womans uterus and allowed to fully develop, the baby would have been free of the disease-causing variation of the gene.

This type of beneficial change would also have been passed down to future generations.

None of the embryos in this study were implanted or allowed to develop. But the success of the experiment offers a glimpse at the potential of CRISPR-Cas9.

Still, will we ever be able to gene-edit our world free of disease?

According to the Genetic Disease Foundation, there are more than 6,000 human genetic disorders.

Scientists could theoretically use CRISPR-Cas9 to correct any of these diseases in an embryo.

To do this, they would need an appropriate piece of RNA to target corresponding stretches of genetic material.

The Cas9 enzyme cuts DNA at that spot, which allows scientists to delete, repair, or replace a specific gene.

Some genetic diseases, though, may be easier to treat with this method than others.

Most people are focusing, at least initially, on diseases where there really is only one gene involved or a limited number of genes and theyre really well understood, Megan Hochstrasser, PhD, science communications manager at the Innovative Genomics Institute in California, told Healthline.

Diseases caused by a mutation in a single gene include sickle cell disease, cystic fibrosis, and Tay-Sachs disease. These affect millions of people worldwide.

These types of diseases, though, are far outnumbered by diseases like cardiovascular disease, diabetes, and cancer, which kill millions of people across the globe each year.

Genetics along with environmental factors also contribute to obesity, mental illness, and Alzheimers disease, although scientists are still working on understanding exactly how.

Right now, most CRISPR-Cas9 research focuses on simpler diseases.

There are a lot of things that have to be worked out with the technology for it to get to the place where we could ever apply it to one of those polygenic diseases, where multiple genes contribute or one gene has multiple effects, said Hochstrasser.

Although designer babies gain a lot of media attention, much CRISPR-Cas9 research is focused elsewhere.

Most people who are working on this are not working in human embryos, said Hochstrasser. Theyre trying to figure out how we can develop treatments for people that already have diseases.

These types of treatments would benefit children and adults who are already living with a genetic disease, as well as people who develop cancer.

This approach may also help the 25 million to 30 million Americans who have one of the more than 6,800 rare diseases.

Gene editing is a really powerful option for people with rare disease, said Hochstrasser. You could theoretically do a phase I clinical trial with all the people in the world that have a certain [rare] condition and cure them all if it worked.

Rare diseases affect fewer than 200,000 people in the United States at any given time, which means there is less incentive for pharmaceutical companies to develop treatments.

These less-common diseases include cystic fibrosis, Huntingtons disease, muscular dystrophies, and certain types of cancer.

Last year researchers at the University of California Berkeley made progress in developing an ex vivo therapy where you take cells out of a person, modify them, and put them back into the body.

This treatment was for sickle cell disease. In this condition, a genetic mutation causes hemoglobin molecules to stick together, which deforms red blood cells. This can lead to blockages in the blood vessels, anemia, pain, and organ failure.

Researchers used CRISPR-Cas9 to genetically engineer stem cells to fix the sickle cell disease mutation. They then injected these cells into mice.

The stem cells migrated to the bone marrow and developed into healthy red blood cells. Four months later, these cells could still be found in the mices blood.

This is not a cure for the disease, because the body would continue to make red blood cells that have the sickle cell disease mutation.

But researchers think that if enough healthy stem cells take root in the bone marrow, it could reduce the severity of disease symptoms.

More work is needed before researchers can test this treatment in people.

A group of Chinese researchers used a similar technique last year to treat people with an aggressive form of lung cancer the first clinical trial of its kind.

In this trial, researchers modified patients immune cells to disable a gene that is involved in stopping the cells immune response.

Researchers hope that, once injected into the body, the genetically edited immune cells will mount a stronger attack against the cancer cells.

These types of therapies might also work for other blood diseases, cancers, or immune problems.

But certain diseases will be more challenging to treat this way.

If you have a disorder of the brain, for example, you cant remove someones brain, do gene editing and then put it back in, said Hochstrasser. So we have to figure out how to get these reagents to the places they need to be in the body.

Not every human disease is caused by mutations in our genome.

Vector-borne diseases like malaria, yellow fever, dengue fever, and sleeping sickness kill more than 1 million people worldwide each year.

Many of these diseases are transmitted by mosquitoes, but also by ticks, flies, fleas, and freshwater snails.

Scientists are working on ways to use gene editing to reduce the toll of these diseases on the health of people around the world.

We could potentially get rid of malaria by engineering mosquitoes that cant transmit the parasite that causes malaria, said Hochstrasser. We could do this using the CRISPR-Cas9 technique to push this trait through the entire mosquito population very quickly.

Researchers are also using CRISPR-Cas9 to create designer foods.

DuPont recently used gene editing to produce a new variety of waxy corn that contains higher amounts of starch, which has uses in food and industry.

Modified crops may also help reduce deaths due to malnutrition, which is responsible for nearly half of all deaths worldwide in children under 5.

Scientists could potentially use CRISPR-Cas9 to create new varieties of food that are pest-resistant, drought-resistant, or contain more micronutrients.

One benefit of CRISPR-Cas9, compared to traditional plant breeding methods, is that it allows scientists to insert a single gene from a related wild plant into a domesticated variety, without other unwanted traits.

Gene editing in agriculture may also move more quickly than research in people because there is no need for years of lab, animal, and human clinical trials.

Even though plants grow pretty slowly, said Hochstrasser, it really is quicker to get [genetically engineered plants] out into the world than doing a clinical trial in people.

Safety and ethical concerns

CRISPR-Cas9 is a powerful tool, but it also raises several concerns.

Theres a lot of discussion right now about how best to detect so-called off-target effects, said Hochstrasser. This is what happens when the [Cas9] protein cuts somewhere similar to where you want it to cut.

Off-target cuts could lead to unexpected genetic problems that cause an embryo to die. An edit in the wrong gene could also create an entirely new genetic disease that would be passed onto future generations.

Even using CRISPR-Cas9 to modify mosquitoes and other insects raises safety concerns like what happens when you make large-scale changes to an ecosystem or a trait in a population that gets out of control.

There are also many ethical issues that come with modifying human embryos.

So will CRISPR-Cas9 help rid the world of disease?

Theres no doubt that it will make a sizeable dent in many diseases, but its unlikely to cure all of them any time soon.

We already have tools for avoiding genetic diseases like early genetic screening of fetuses and embryos but these are not universally used.

We still dont avoid tons of genetic diseases, because a lot of people dont know that they harbor mutations that can be inherited, said Hochstrasser.

Some genetic mutations also happen spontaneously. This is the case with many cancers that result from environmental factors such as UV rays, tobacco smoke, and certain chemicals.

People also make choices that increase their risk of heart disease, stroke, obesity, and diabetes.

So unless scientists can use CRISPR-Cas9 to find treatments for these lifestyle diseases or genetically engineer people to stop smoking and start biking to work these diseases will linger in human society.

Things like that are always going to need to be treated, said Hochstrasser. I dont think its realistic to think we would ever prevent every disease from happening in a human.

Continue reading here:
Will Gene Editing Allow Us to Rid the World of Diseases? - Healthline