When the sequence of the human genome was published in 2001 it was hailed as a great achievement. But now we know our genomes are much more (and much more mysterious) than a simple linear sequence of nucleotide letters. It coils around and over itself in ways that seem mindbogglingly complex. But recently researchers have begun to unravel this mystery and realize that dynamic changes in the genomes three-dimensional structure affect how and when important genes are expressed.

Now dermatologist Paul Khavari, MD, PhD, and graduate student Adam Rubin, former graduate student Brook Barajas, PhD, and researcher Mayra Furlan-Magaril, PhD, have used new mapping techniques to peer into the deepest recesses of tissue-specific stem cells progenitor cells that hang out in specialized tissues like muscle waiting for the call to divide and specialize. They identified two types of DNA contacts that help these cells answer a call to action. They published their resultsin Nature Genetics.

As Khavari explained to me in an email:

How the human genome rearranges itself to express genes needed for specific processes, such as stem cell differentiation, has been a mystery. This work shows that this not only involves physically changing DNA contacts, but also functionally activating contacts between pieces of DNA that were already established.It revises our understanding of the genome to a more living, breathing, moving entity that literally reconfigures itself as it changes its expression rather than a static template that is merely copied.

Specifically, Khavari and his colleagues found that the transformation from a tissue-specific stem cell into a more specialized cell (a process called differentiation) involves a two-step process: First the genomes of stem cells are prepped through a looping process that brings functional parts of the genome into close contact. Then the cells bide their time until the moment of differentiation, when proteins called transcription factors are unleashed to bind to these new DNA neighbors and stimulate the expression of genes necessary to launch the coming transformation.

As Khavari said:

This research illuminates a fundamental mechanism of genome regulation that has not been appreciated before. Specifically, a stem cell is pre-wired with established contacts to express a specific set of differentiation genes but only activates them when the dynamic loops are engaged. By analogy with a race, the runners are all at the starting line and ready to run in that particular event but only the firing of the gun sets the specific event in motion.

This pre-wiring not only allows the stem cells to respond quickly to differentiation signals, but it also locks them into a specific fate, the researchers believe. In this way, a muscle stem cell avoids any missteps that could result in it mistakenly becoming a skin or a blood cell rather than a muscle cell. Interestingly, the researchers also found clues suggesting that perturbations in this looping process are sometimes associated with the development of certain diseases, including skin cancer and psoriasis.

Previously: Inducible loops enable 3D gene expression studies, The quest to unravel complex DNA structures gets a boost from new technology and NIH fundingand DNA origami: How our genomes foldPhoto by Braden Collum

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Genome architecture guides stem cell fate, Stanford researchers find - Scope (blog)

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BUFFALO, N.Y. People with multiple sclerosis (MS) knowall too well the frustration of hearing that success in treatingthe disease in mice had little or no effect in humans.

Unfortunately, with no large animal models for MS, results thatsuggest promising new treatments in mice often are ineffective inhumans.

Now, University at Buffalo researchers have developed andsuccessfully tested a method for determining how relevant to thehuman disease findings are from mouse models. The researchwas published Aug. 8 in Stem Cell Reports.

This is an important resource for the field as it allowsus to compare human and rodent cells, and provides a point ofreference to understand whether or not gene expression patterns areconserved between species, said Fraser Sim, PhD, seniorauthor and associate professor in the Department of Pharmacologyand Toxicology in the Jacobs School of Medicine and BiomedicalSciences at UB. Co-first authors are Suyog U. Pol PhD, now apostdoctoral fellow, and Jessie J. Polanco, a doctoral candidate,both in the medical school.

MS trial failures

There have been so many failures in clinical trials forMS when promising observations are translated from small animalmodels to the clinic, Sim said. Our primarymotivation was to try to understand, at a molecular level, how thehuman cells responsible for synthesizing myelin differ from theirmuch-better-studied mouse counterparts.

MS and some other neurological diseases occur when there isdamage to myelin the fatty sheath that allows nerve cellsto communicate. So the myelin-producing cells, called humanoligodendrocyte progenitor cells, or OPCs, found in the brain andspinal cord have been a major focus of efforts to better understandMS and develop potential new treatments for it.

Sim explained that undifferentiated OPCs are frequently found inthe brain lesions of MS patients, so boosting the differentiationof these cells could lead to myelination and a reduction ofsymptoms.

From OPCs to oligodendrocytes

One reason why so many clinical trials fail may be because offundamental differences in the types and levels of genes expressedbetween mice and humans. Sim and his colleagues addressed thisquestion by performing gene-expression analysis on differentiatinghuman OPCs.

In this paper, we describe the transcriptional eventsthat underlie how human OPCs develop into oligodendrocytes,said Sim.

To do it, they used a network analysis software tool calledweighted gene coexpression network analysis (WCGNA). The softwareclusters together genes with similar patterns of expression. Italso allows for analysis of both conserved and divergent geneexpression between humans and rodents.

WCGNA looks at the relationships between genes ratherthan absolute differences between conditions in any givenexperiment, Sim said.

He added that the information encoded in levels of geneexpression increasing or decreasing is very reliable andreproducible.

We performed WCGNA in exactly the same manner on cellsisolated from mice, rats and humans, and prepared these cells in asclose to matched conditions as possible, trying to keep things assimilar as possible to facilitate this comparison, saidSim.

It turned out several of the genes the team had identified asrelevant to human disease also are involved in mouse developmentand mouse models of myelin disease.

New myelin-repairing gene

Based on its findings from that analysis, the team had predictedthat GNB4, a protein involved in signal transduction, would beinvolved in the development of OPCs in humans. The researchersfound that over-production of GNB4, a protein involved in thetransduction of extracellular signals, could cause human OPCs torapidly undergo myelination when transplanted into a model forhuman cell therapy in MS.

So this proteins expression in oligodendrocyteprogenitor cells might ultimately become a therapeutic target,potentially promoting oligodendrocyte formation in MSpatients, said Sim.

The approach also identified several other important candidatesthat play key roles in regulating the development of humanoligodendrocytes.

Other co-authors on the paper are Melanie A. OBara,research scientist; Hani J. Shayya, a UB undergraduate and Karen C.Dietz, PhD, research assistant professor, all of the Department ofPharmacology and Toxicology and Richard A. Seidman, amasters candidate in neuroscience.

The research was funded by the National Multiple SclerosisSoociety, the Kalec Multiple Sclerosis Foundation, the SkarlowMemorial Trust and the Empire State Stem Cell Fund (NYSTEM) throughthe New York State Department of Health.

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Northern Ireland mum fighting MS: Russian medics are now my last hope

BelfastTelegraph.co.uk

A young Co Down mum is bravely undergoing a gruelling stem cell transplant in Russia in what she believes is her last hope of enjoying some quality of life.

http://www.belfasttelegraph.co.uk/news/northern-ireland/northern-ireland-mum-fighting-ms-russian-medics-are-now-my-last-hope-36023340.html

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A young Co Down mum is bravely undergoing a gruelling stem cell transplant in Russia in what she believes is her last hope of enjoying some quality of life.

Lindsay Rice (35) from Warrenpoint has exhausted every treatment on the health service - including chemotherapy normally given to cancer patients - in the hope of treating the chronic condition Rapidly Evolving Severe Relapsing Remitting Multiple Sclerosis.

Paralysis and temporary sight loss are just a few of the many debilitating symptoms which have left the mum-of-two unable to enjoy normal family life.

Desperate to get her help, her family launched an appeal on Facebook and Go Fund Me to raise 50,000 to send her to the National Pirogov Medical Surgical Centre in Moscow where she arrived two weeks ago to start her stem cell transplant.

The treatment alone is expected to cost up to 45,000 and, incredibly, in just 12 weeks the family has raised 32,000 towards a 50,000 target thanks to generous support from friends and the public.

Lindsay, who is married to Liam (36), a financial advisor, has two children, Jamie (17) and Olivia (8).

Liam says: "This is her last hope and she is doing it for her family and her kids and that's what she is focusing on. She just wants to be able to live a normal life and do normal things with the family."

Since starting her treatment on August 1 she has been keeping a daily dairy of her progress through a Facebook page - Lindsay's Last Hope.

While the groundbreaking treatment known as HSCT (Haematopoietic Stem Cell Transplant) is not a cure for MS, Lindsay's hope is that it will halt the progression of the disease and stop the frequent and severe relapses which are destroying her health.

Lindsay will spend a month in the clinic, most of it in isolation, and when she comes home she faces a long recovery period when she will have to remain isolated for up to a year due to the risk of infection.

HSCT aims to 'reset' the immune system to stop it attacking the central nervous system. It uses chemotherapy to remove the harmful immune cells and then rebuild the immune system using a type of stem cell found in the patient's bone marrow.

The haematopoietic stem cells used in the treatment can produce all the different cells in the blood, including immune cells. However, they can't regenerate permanently damaged nerves or other parts of the brain and spinal cord.

Lindsay has successfully had over two million stem cells extracted in a tough procedure which involved having a catheter inserted into her jugular vein. She has also had her head shaved this week in preparation for starting chemotherapy today.

The chemotherapy will wipe out her immune system and she will then have her stem cells transplanted back into her blood by a drip to help regrow a new, stronger immune system.

She will then have to spend 10 days in complete isolation while her new immune system builds.

Also, since arriving in Russia she has been told that her MS is now much worse than she realised and is now at the Secondary Progressive stage.

People with Secondary Progressive MS don't tend to recover completely from a relapse and can expect a general worsening of symptoms, making the treatment even more time-critical.

In a further blow, tests have picked up a potentially dangerous three-centimetre active lesion on her spine which wasn't spotted during MRI's here.

Lindsay faces a tough few weeks in her bid to halt the progression of the disease but as her husband Liam explains, the alternative is the prospect of life in a wheelchair: "Lindsay has come through a lot since her teens.

"She had Jamie quite young at 18 and her condition seemed to really deteriorate after that. She went to a lot of consultants and had many tests but it wasn't until after she had Olivia that she was finally diagnosed in 2011.

"She never knows from day to day how it will affect her. Fatigue is the number one problem and that is crippling. I would come home from work and after dinner she has to go to bed, and even sleep doesn't help it.

"It stops her from doing simple things like taking our daughter to the park or taking the dog for a walk.

"Her motability is not as good as an average person and the other big issue is the relapses.

"They have become very frequent and each relapse is worse in terms of how severe it is. During her last one in February she had to go into hospital and also had to use a walking frame.

"A common misconception is that after each relapse you go back to normal but that's not the case. It leaves its mark and any damage done is permanent. The nature of the relapses could leave her in a wheelchair."

It was after her last relapse and having exhausted all options for treatment on the Health Service that Lindsay decided she wanted to try HSCT.

Her neurologist in Belfast supported her decision and the family applied to the Russian clinic just 12 weeks ago expecting to wait up to two years before admission.

They were surprised to be offered a cancellation on August 1 leaving them facing a race against time to raise 50,000 to cover the cost of treatment and expenses.

Liam says: "We thought we would have at least 12 months and up to two years to get the money together and it has been amazing to see how people have rallied round and what they have done just from the kindness of their hearts, especially strangers.

"We've had quizzes and coffee mornings and online auctions and I recently did the Four Peaks challenge with a group of friends. Lindsay's mum and her best friend are organising a lot of events and we still have some way to go but we are amazed at how much has been raised and donated in such a short time."

Liam flew to Russia with Lindsay on July 31 and stayed with her for five days while she underwent tests to determine that she was suitable for the treatment.

It has already been a punishing two weeks for Lindsay who has come through a batch of invasive procedures including having a catheter inserted in her jugular to extract the stem cells.

Liam says: "It is an intense treatment and Lindsay is so positive and coping brilliantly. She got her hair cut short before she went and decided to have it shaved this week before the chemo starts and it falls out.

"She will have to spend 10 days in complete isolation to allow her immune system to build again and that will be tough.

"She will hopefully be home after 30 days and then when she comes home she will have a long recovery and will have to isolate herself from society for up to a year to keep her safe from infection.

"We will have to deep clean the house and we will all have to wear face masks as she can't risk even getting a cold."

Liam is back at work and trying to keep things as normal as possible at home for the couple's two children, who he said are coping well: "Jamie is 17 and approaching adulthood and understands why she is doing it and is okay, but obviously his mum is away and he has his sixth year exam results coming and he misses her.

"Olivia seems to be fine too. She understands her mum has MS, which stops her doing things with her and she knows this treatment is to help her to be a better mother.

"I've been trying as much as possible to keep her occupied with play dates and sleepovers."

The couple have been impressed by the level of care in the clinic and Lindsay has had the chance to meet and get to know other MS patients from all over the world.

Liam has nothing but admiration for her strength and the positive way she is enduring the extreme procedures she faces.

He adds: "Lindsay is the most determined person you could ever possibly meet. She has had bad days and it can be demoralising for her but she is determined to be as positive as she can be.

"It is not a cure. MS doesn't have a cure but we hope it will stop the progress of the disease. We just hope it will halt it by rebooting her immune system and hopefully stop the severe relapses."

Liam adds: "It is desperately hard and stressful for all of us and we have to put a positive spin, in the grand scheme of things it is just for a month of her life."

Follow Lindsay's journey at Facebook/Lindsay's Last Hope - HSCT in Russia

Fundraising continues as the family has only until the end of the month to reach their target. You can support this young mum in her bid to enjoy a normal quality of life by going to https://www.gofundme.com/lindsay-slasthope

Belfast Telegraph

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In the days after a heart attack, surviving patients and their loved ones can breathe a sigh of relief that the immediate danger is over but the scar tissue that forms during the long healing process can inflict lasting damage. Too often it restricts the hearts ability to fill properly between beats, disrupting rhythm and ultimately leading to heart failure. Yet a new possible treatment may help to revitalize an injured ticker.

A cadre of scientists and companies is now trying to prevent or reverse cardiac damage by infusing a cocktail of stem cells into weakened hearts. One company, Melbourne, Australiabased Mesoblast, is already in late-stage clinical trials, treating hundreds of chronic heart failure patients with stem cell precursors drawn from healthy donors hip bones. A randomized trial that includes a placebo group is scheduled to complete enrollment next year.

Mesoblasts earlier-stage trials, published in 2015 inCirculation Research, found that patients who received injections of its cell mixture had no further problems related to heart failure.

Promising results from the new trial would be a major step forward for a field that has long been criticized for studies that are poorly designed, incomplete or lack control-group comparisons, as well as for the peddling of unproved therapies in many clinics worldwide.

Another company, Belgium-based TiGenix, hopes to attack scar tissue before it forms by treating patients with a mixture of heart stem cells within seven days of a heart attack. This approach has just completed phase II trials, but no findings have yet been published.

There are still many unanswered questions about how stem cells typically derived from bones could help heal the heart. Leading theories suggest they may help fight inflammation, revitalize existing heart cells, or drive those cells to divide or promote new blood-vessel growth, says Richard Lee, leader of the cardiovascular program at the Harvard Stem Cell Institute. Other stem cell scientists, including Joshua Hare, who conducted earlier-stage Mesoblast research and directs the Interdisciplinary Stem Cell Institute at the University of Miami, say the cells may work in multiple ways to heal scar tissue. According to Hare, the stem cells could ultimately be a truly regenerative treatment.

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The LA Galaxy Foundation has teamed up with Gift of Life Marrow Registry, a club community partner curing blood cancer through marrow and stem cell donation, to co-host Kick Blood Cancer at The Grove in Los Angeles on Sunday, Aug. 13 from 1-4p.m. The event will feature family-friendly games, activities and LA Galaxy appearances in the effort to recruit potential donors to the worldwide marrow registry.

LA Galaxy goalkeeper Jon Kempin, LA Galaxy Star Squad and LA Galaxy mascot Cozmo will be in attendance. Kempin joined LA Galaxy in the off-season and is one of the brightest young talents in the organization, who earned his first MLS shutout earlier this season. He signed his first MLS contract with Sporting Kansas City at the age of 17.

Gift of Life believes every person battling blood cancer deserves a second chance at life and they are determined to make it happen. They are singularly passionate about engaging the public to help get everyone involved in curing blood cancer, whether as a donor, a volunteer or a financial supporter. It all begins with one remarkable person, one life-changing swab and one huge win finding a match and a cure.

For many patients who suffer from leukemia, lymphoma, or other types of blood cancer, transplantation of bone marrow or peripheral blood stem cells donated by unrelated volunteers offers the hope of a cure.

WHAT

Kick Blood Cancer

WHEN

Sunday, Aug. 13

1-4 p.m.

WHERE

The Grove

189 The Grove Drive

Los Angeles, CA 90036

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Join Jon Kempin, LA Galaxy Foundation and Gift of Life Marrow Registry for Kick Blood Cancer on August 13 - LA Galaxy

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The human body is an amazing thing. For each one of us, it's the most intimate object we know. And yet most of us don't know enough about it: its features, functions, quirks, and mysteries. Our series The Body explores human anatomy, part by part. Think of it as a mini digital encyclopedia with a dose of wow.

If you say someone's getting on your nerves, you could just cut to the chase and say they're getting on your sciatic nervethis nerve is plenty big enough for both minor and major irritations. It's the largest nerve in the body, running a lengthy route from each side of your lower spine, deep into your buttock, wrapping around to the back of the thigh and into the foot. Mental Floss spoke to Loren Fishman, medical director of Manhattan Physical Medicine and Rehabilitation in NYC andassociate clinical professor at Columbia Medical School. Here are 13 things we learned about this important part of the nervous system.

No wonder this nerve hurts when it gets irritatedat its biggest point, it's one heck of a large nerve, says Fishman.

The sciatic nerve is more accurately five nerves that come together on the right and left sides of the lower spine. Technically, the fourth and fifth lumbar nerves and the first three nerves in the sacral spine come together and merge into the unified sciatic.

"The sciatic nerve gives feeling and strength to the muscles and skin of the calf and foot, supplies sensation from the joints, bones, and just about everything else below the knee," says Fishman.

The nerve connects the spinal cord with the outside of the thigh, the hamstring muscles in the back of the thigh, and the muscles in your lower leg and feet. This is why sciatic nerve impingement often results in muscle weakness, numbness and/or tingling in the leg, ankle, foot, and toes.

After severe spinal cord injury, the nerve itself is often just fine, but the connection between it and the brain has been severed, Fishman says. Until now, there's been no way to fix such injuries, but "recent work with stem cells has begun to restore the connection in dogs and other animals."

A variety of lower back problems can lead to pain that radiates along the sciatic nerve. Most commonly, sciatica pain is caused when a herniated disc at the L5 (lower lumbar back) irritates the S1 (sacrum) nerve root in the lower spine. The exiting nerve roots are highly sensitive, and the bits of the disc that herniate contain inflammatory proteins such as interleukin and tumor necrosis factor that can also aggravate the nerve.

In a small number of people, a condition called cauda equina syndrome (so named because the nerve bundle at the base of the spinal cord resembles a horse's tail) can masquerade as sciaticabut it also usually causes weakness that extends to bowel or bladder incontinence and sometimes weakness or loss of sensation in the legs that gets progressively worse. In this case, immediate medical attention should be sought, and recovery may not be as quick as with common sciatica.

When the ancient Greek and Roman physicians were treating the pain we now commonly know as sciatica, they believed it stemmed from "diseases of the hip joint," according to a 2007 study in Spinal Cord. It wasn't until 1764, write the authors, "that leg pain of 'nervous' origin was distinguished from pain of 'arthritic' origin."

Among the many treatments Hippocrates and his ilk came up with for this painful condition were: "Fumigations, fasting, and subsequently, laxatives, and ingestion of boiled milk of the female ass." In his Treatise of the Predictions, Hippocrates noted that elderly patients with "cramps and colds at the loin and the legs" would experience their pain for up to a year, whereas young people could be free of pain in about 40 days.

The modern name for the disease, according to Fishman, comes from 15th-century Florence. "They called sciatica ischiatica, since they thought it came from tuberculosis that worked its way down to the ischial tuberosity (the sit-bones)," Fishman says. These medieval doctors had the cause wrong, but the name stuck.

Different researchers in different countries began to make sciatic breakthroughs when doing autopsies on corpses with fractured or herniated discs, where they noticed compression on the sciatic nerve.

A 1991 cross sectional study of 2946 women and 2727 men published in Spine found that neither gender nor body mass made any difference in the likelihood of developing sciatica. Body height did, however, in males between the ages of 50 and 64, with taller men being more likely to have the condition. Other studies have found a similar link [PDF]. Over 5'8"? Your risk is higher.

Sciatica has a surprisingly common negative impact on daily life. "Low back pain and sciatica are the second biggest reason for lost days of workjust behind the common cold," says Fishman.The condition is most commonly found in people over 50 andrarely seen in anyone under 20 years oldand then it most often has a genetic cause.

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GREENVILLE Onboard the next SpaceX cargo spacecraft launching to the International Space Station (ISS) from Pad 39A at the Kennedy Space Center will be a commercial research system owned and operated by Techshot Inc. The equipment will conduct regenerative medicine experiments onboard the station before returning to Earth in the same capsule for a splashdown off the coast of Southern California approximately 30 days later.

Techshots ADvanced Space Experiment Processor (ADSEP) is a device approximately the size of a microwave oven that contains three separate modules, each of which simultaneously can process experiments in three separate on-orbit replaceable automated mini-laboratory cassettes. Two of the three cassettes on the mission will conduct research for a team led by Robert Schwartz, Ph.D., from the University of Houston.

Funded by the Center for the Advancement of Science in Space (CASIS), the study will evaluate a new approach to growing human tissue for transplant. The microgravity environment onboard the ISS could improve cell growth and development and 3D tissue formation, enabling discoveries that will advance translational disease treatments. Previous studies on Earth by Schwartz and his collaborators at the Texas Heart Institute and the Baylor College of Medicine have found that low gravity environments help specially programmed stem cells move toward becoming new heart muscle cells, which may be used to repair damaged hearts on Earth.

The third cassette contains an experiment conducted by and for Techshot itself. The company is developing a 3D bioprinter for the ISS known as the Techshot BioFabrication Facility (BFF), which it expects to launch to the station near the end of 2018. Critical to the success of the printer will be the ability to provide nutrients and mechanical stress for organs and tissues it manufactures in space strengthening them and keeping them viable for transplantation back on Earth.

Approximately 36 hours prior to launch, Techshot scientists in a laboratory at the Kennedy Space Center will 3D print a one centimeter thick construct consisting of stem cells and heart muscle cells. Theyll then place it inside the prototype BFF cell culturing subsystem, which for this mission is temporarily housed inside an ADSEP cassette. The printer used in the lab will be the same modified nScrypt unit that was the first to 3D print cardiac constructs with adult human stem cells in microgravity aboard an aircraft in parabolic flight. Video captured inside the cassette during the month-long experiment, and the tissue itself which is expected to have developed its own micro blood vessels will be evaluated for effectiveness after return from space.

Techshots space bioprinting program leverages its terrestrially based technologies for cell isolation and vascular graft development, and its decades long experience culturing cells in space, said Techshot Chief Scientist Eugene Boland, Ph.D., in a news release. Being able to test our novel approach for culturing 3D printed cells more than a year before we fly the whole BFF is invaluable. The data from this mission will get us one step closer toward our goal of helping eliminate organ shortages.

Founded in 1988, Techshot Inc., develops technologies used in the aerospace, defense and medical industries. Through its Space Act Agreement with NASA, and its role as an official CASIS Implementation Partner, the company provides equipment and services that help federal, institutional and industrial customers live and work in space. http://www.Techshot.space

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The PASE, or post-implantation amniotic sac embryoid, is a structure grown from human pluripotent stem cells that mimics many of the properties of the amniotic sac that forms soon after an embryo implants in the uterus wall. The structures could be used to study infertility. Credit: University of Michigan

The first few weeks after sperm meets egg still hold many mysteries. Among them: what causes the process to fail, leading to many cases of infertility.

Despite the importance of this critical stage, scientists haven't had a good way to explore what can go wrong, or even what must go right, after the newly formed ball of cells implants in the wall of the human uterus.

But a new achievement using human stem cells may help change that. Tiny lab-grown structures could give researchers a chance to see what they couldn't before, while avoiding ethical issues associated with studying actual embryos.

A team from the University of Michigan reports in Nature Communications that they have coaxed pluripotent human stem cells to grow on a specially engineered surface into structures that resemble an early aspect of human development called the amniotic sac.

The cells spontaneously developed some of the same structural and molecular features seen in a natural amniotic sac, which is an asymmetric, hollow ball-like structure containing cells that will give rise to a part of the placenta as well as the embryo itself. But the structures grown at U-M lack other key components of the early embryo, so they can't develop into a fetus.

It's the first time a team has grown such a structure starting with stem cells, rather than coaxing a donated embryo to grow, as a few other teams have done.

"As many as half of all pregnancies end in the first two weeks after fertilization, often before the woman is even aware she is pregnant. For some couples, there is a chronic inability to get past these critical early developmental steps, but we have not previously had a model that would allow us to explore the reasons why," says co-senior author Deborah Gumucio, Ph.D. "We hope this work will make it possible for many scientists to dig deeper into the pathways involved in normal and abnormal development, so we can understand some of the most fascinating biology on earth." Gumucio is the Engel Collegiate Professor of Cell & Developmental Biology at Michigan Medicine, U-M's academic medical center.

A steady PASE

The researchers have dubbed the new structure a post-implantation amniotic sac embryoid, or PASE. They describe how a PASE develops as a hollow spherical structure with two distinct halves that remain stable even as cells divide.

One half is made of cells that will become amniotic ectoderm, the other half consists of pluripotent epiblast cells that in nature make up the embryonic disc. The hollow center resembles the amniotic cavity - which in normal development eventually gives rise to the fluid-filled sac that protects and cushions the fetus during development.

Gumucio likens a PASE to a mismatched plastic Easter egg or a blue-and-red Pokmon ball - with two clearly divided halves of two kinds of cells that maintain a stable form around a hollow center.

The team also reports details about the genes that became activated during the development of a PASE, and the signals that the cells in a PASE send to one another and to neighboring tissues. They show that a stable two-halved PASE structure relies on a signaling pathway called BMP-SMAD that's known to be critical to embryo development.

Gumucio notes that the PASE structures even exhibit the earliest signs of initiating a "primitive streak", although it did not fully develop. In a human embryo, the streak would start a process called gastrulation. That's the division of new cells into three cell layersendoderm, mesoderm and ectodermthat are essential to give rise to all organs and tissues in the body.

Collaboration provides the spark

The new study follows directly from previous collaborative work between Gumucio's lab and that of the other senior author, U-M mechanical engineering associate professor Jianping Fu, Ph.D.

In the previous work, reported in Nature Materials, the team succeeded in getting balls of stem cells to implant in a special surface engineered in Fu's lab to resemble a simplified uterine wall. They showed that once the cells attached themselves to this substrate, they began to differentiate into hollow cysts composed entirely of amnion - a tough extraembryonic tissue that holds the amniotic fluid.

But further analysis of these cysts by co-first authors of the new paper Yue Shao, Ph.D., a graduate student in Fu's lab, and Ken Taniguchi, a postdoctoral fellow in Gumucio's lab, revealed that a small subset of these cysts were stably asymmetric and looked exactly like early human or monkey amniotic sacs.

The team found that such structures could also grow from induced pluripotent stem cells (iPSCs)cells derived from human skin and grown in the lab under conditions that give them the ability to become any type of cell, similar to how embryonic stem cells behave. This opens the door for future work using skin cells donated by couples experiencing chronic infertility, which could be grown into iPSCs and tested for their ability to form proper amniotic sacs using the methods devised by the team.

Important notes and next steps

Besides working with genetic and infertility specialists to delve deeper into PASE biology as it relates to human infertility, the team is hoping to explore additional characteristics of amnion tissue.

For example, early rupture of the amnion tissue can endanger a fetus or be the cause of a miscarriage. The team also intends to study which aspects of human amnion formation also occur in development of mouse amnion. The mouse embryo model is very attractive as an in vivo model for investigating human genetic diseases.

The team's work is overseen by a panel that monitors all work done with pluripotent stem cells at U-M, and the studies are performed in accordance with laws regarding human stem cell research. The team ends experiments before the balls of cells effectively reach 14 developmental days, the cutoff used as an international limit on embryo researcheven though the work involves tissue that cannot form an embryo. Some of the stem cell lines were derived at U-M's privately funded MStem Cell Laboratory for human embryonic stem cells, and the U-M Pluripotent Stem Cell Core.

Explore further: Team uses stem cells to study earliest stages of amniotic sac formation

More information: Yue Shao et al, A pluripotent stem cell-based model for post-implantation human amniotic sac development, Nature Communications (2017). DOI: 10.1038/s41467-017-00236-w

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Amniotic sac in a dish: Stem cells form structures that may aid of ... - Phys.Org

Skin Stem Cells Comments Off on Amniotic sac in a dish: Stem cells form structures that may aid of … – Phys.Org | August 9th, 2017

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Cardiac Stem Cells Comments Off on Texas Heart Institute Awarded Grant to Study Sex Differences in Cardiac Repair – Texas Medical Center (press release) | August 9th, 2017

The new research has been developed by a team led by Dr. Samuel I. Stupp, who is the director of Northwestern Universitys Simpson Querrey Institute for BioNanotechnology. The researcher is also Professor of Materials Science and Engineering, Chemistry, Medicine and Biomedical Engineering.The new technology centers on the way that cells behave in the human body. Our cells are continually being signaled with various instructions, triggered by proteins and other molecules that are located in the matrices that surround them. As an example, such signals can be cues for cells to express specific genes in order for the cells to differentiate into other types of cells. Such a development is important for growth or regeneration of tissues. This sophisticated, biological signaling machinery has the pre-programmed capacity to make signals stop and re-start as needed; or to switch off one signal and activate an alternative signal in order to commence a complex processes. If this could be controlled by medics, then the process of addressing a range of diseases could be achieved. So far, the ability to produce such regenerative therapies has proved impossible.This could be set to change with the development of a synthetic material that can trigger reversibly certain types of signaling. This platform could lead to materials to control stem cells in order to produce regenerative therapies and to control cellular functions. The new technology should help with research into treatments for such diseases as Alzheimers disease, Parkinsons disease, problems with arthritic joints, spinal cord injuries, the effects of stroke, and other conditions requiring tissue regeneration.In trials, the researchers have taken spinal cord neural stem cells (neurospheres) and driven them to differentiate using a signal, helping the scientists to understand developmental and regenerative cues. This cell manipulation technology could help control which cells change and thereby address diseases like Parkinsons, such as converting a patients own skin cells into stem cells. Commenting on the implications of the technology, Dr. Stupp said, in a communication provided to Digital Journal: Its important in the context of cell therapies for people to cure these diseases or regenerate tissues that are no longer functional.The research is an example of the use of digital based bio-nanotechnology. The technology has been published in the journal Nature Communications. The paper Instructing cells with programmable peptide DNA hybrids.

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New technology manipulates cells for disease research - Digital Journal

Spinal Cord Stem Cells Comments Off on New technology manipulates cells for disease research – Digital Journal | August 8th, 2017

Genetically engineered skin cells grafted onto mice can treat the animals diabetes and obesity, according to new research published August 2, 2017 in Cell Stem Cell.

Researchers edited skin stem cells from newborn mice using CRISPR-based technology so that the cells secreted a peptide that regulates blood sugar. Transplanting the cells onto mice showed the grafts increased insulin secretion and reversed weight gain from a high-fat diet, as well as overturned insulin resistance. The result is a small step toward developing a safe and durable gene therapy to treat diabetes in humans.

Weve had this idea for a long time, so its exciting to see that, indeed, it can work to deliver therapeutics, coauthor Xiaoyang Wu, a stem cell biologist at the University of Chicago, tells GEN.

In the study, Wu and colleagues worked with skin because it is a large organ and easily accessible. The cells multiply quickly and are easily transplanted. And, transplanted cells can be removed, if needed. Skin is such a beautiful system, Wu says, noting that its features make it a perfect medium for testing gene therapies.

The team worked with the gene that produces glucagon-like peptide 1 (GLP-1), a hormone that stimulates the pancreas to secrete insulin. The additional insulin takes excessive glucose out of the bloodstream, which regulates complications from diabetes. The hormone can also decrease appetite. Using the genetic engineering tool CRISPR, the team inserted a mutation, adding an antibody fragment to the gene that would make the GLP-1 last longer in the blood and an additional modification to the targeting vector that would also attach an inducible promoter. This switch turns the gene on, as needed, to make more GLP-1. The switch would be triggered by the administration of the antibiotic doxycycline.

Wu and colleagues then inserted the altered gene into skin cells and grew the cells in a culture. Once the skin cells had grown into multiple layers, the team transplanted the patches onto mice with intact immune systems. Surprisingly, the mice didnt reject the graftsa feat in itselfsince human skin transplants are far more advanced than mice grafts, partly due to the animals furry skin.

Next, the team fed the mice small amounts of doxycycline. As a result, the animals released GLP-1 into the blood and had higher levels of insulin and lower levels of glucose. When fed a high-fat diet, the mice gained weight and became obese. But when the mice also were fed doxycycline so they secreted GLP-1, they gained less weight, showing the gene therapy was successful.

This kind of therapy could be potentially effective for many metabolic disorders, Wu says. The grafts could be used in patients who cant process protein or in individuals with hemophilia. The team is now testing the gene-therapy technique in combination with other medications.

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CRISPR Gene Therapy via Skin Grafts Treats Obesity and Diabetes in Mice - Genetic Engineering & Biotechnology News

Skin Stem Cells Comments Off on CRISPR Gene Therapy via Skin Grafts Treats Obesity and Diabetes in Mice – Genetic Engineering & Biotechnology News | August 6th, 2017

Most nights, Marcia Dunlap attaches seven or eight leeches around her husband John's eyes as part of an effort to restore some of his vision. Tom Bailey/The Commercial Appeal

With the help of his wife Marcia, John Dunlap receives his nightly leech treatment at his home in East Memphis. Marcia places several leeches on his face in an effort to increase pressure in his left eye. In conjunction with stem cell treatment, the Dunlaps hope that one day John may be a viable candidate for a procedure that could return some of his vision.(Photo: Jim Weber/The Commercial Appeal)

At home most evenings, Memphis, Tennessee, attorney John Dunlap, 80, unbuttons and removes his white dress shirt and counting his steps and remembering which way to turn carefullywalks with a tall white canefrom the living room to the dining table, where his wife Marcia has a plastic container of leeches.

Twenty-six months ago,the couple's schizophrenic sonAndrewattacked them in theirhome. The injuries blinded Dunlap. He's in total darkness.

After drapinga large, peach-colored towel around John's neck, Marcia reaches into the water for the skinniest leeches. Those are the hungriest and most likely to latchonto John's face.

One at a time, she gently presses four leeches to the skin around John's left eye and three around the right. She waits patiently wait for eachto bite and stay connected to John's skin.

"You can feel a bite,'' he says. "A little, stinging bite... And then after awhile you don't feel anything.''

The Dunlaps have carried out this unusualroutine60 or so times since December. It's a type of therapy prescribed by a Los Angeles doctor who offers experimental stem cell therapy designed to regenerate tissue.

"In the beginning he made it very clear he's not anophthalmologist and not an eye surgeon but he had had some success with stem cells in treating blindness. It's experimental,'' Dunlap said.

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The doctor prescribed the leech therapy as a preliminary step because, Dunlap said, the leech enzymesenhance the blood supply to the eye and nourishthe eye tissue.

The left eye had atrophied, or withered. The idea wasto restore health to the eyebefore the stem cell treatment. There is no right eye, but the hope is that the leech enzymes will help revive that optic nerve in case a transplant is ever possible.

Since the leech therapy,the pressure in the right eye has improved significantly, Dunlapsaid, referring to follow-upexams. The retina, which had folded into an ice-cream cone shape after the trauma, has begun returning to its normal shape, he said.

Even though he still cannot see out of the left eye and the optic nerve remains severed from the retina, Dunlap said, "I now have a live eye.''

The Dunlaps decline to identify the California doctor, describing him as a"humble'' person whodoes not seek the publicity.

With the help of his wife Marcia, John Dunlap receives his nightly leech treatment at his home in East Memphis. Marcia places several leeches on his face in an effort to increase pressure in his left eye. In conjunction with stem cell treatment, the Dunlaps hope that one day John may be a viable candidate for a procedure that could return some of his vision.(Photo: Jim Weber/The Commercial Appeal)

Andrew, the Dunlaps' mentally ill son, is charged with attempted murder and domestic assault, and remains in jail awaiting trial. Thecouplehave told authorities that they mainly want Andrew to receive mental health treatment.

The Dunlapshave experienced tragedy long before the 2015 assault.

Their son Jeff, one of four children, was a St. Jude Children's Research Hospital patient who died of cancer at age 10, in September 1974.

Dunlap recalls a return car tripfrom Knoxville, where he and Marcia had been visiting grandchildren shortly after he was released from rehab.

"As we were driving back I started thinking of all the things I won't get to do again. In my mind, I was going down the list,'' he said.

It would be a long list, including some leisure activities he loves. An avid Cubs fan, heenjoyed attending spring training games in Arizona. A passionate golfer, he enjoyedwatching how the ball flew when he struck it well.

But Dunlap stopped himself from completing the list of losses, telling himself, " 'You don't want to dwell on that'. . . It's as if the Lord sent me a message that hit me across my forehead, saying, 'John, get over it. It could be a whole lot worse.'

"Anytime I want to start thinking about the things I'm missing or not doing what I used to do, I think 'Get over it. Move on'.''

Sudden blindness is such a change in lifestyle. "I guess some people may feel the world has ended for them, but it hasn't,'' he said.

Marcia Dunlap gets special leeches for her husband John's nightly treatment from the laundry room where she keeps it out of sight. Marcia places several leeches on his face in an effort to increase pressure in his left eye. In conjunction with stem cell treatment, the Dunlaps hope that one day John may be a viable candidate for a procedure that could return some of his vision.(Photo: Jim Weber/The Commercial Appeal)

The stem cell and leech therapy is expensive and not covered by health insurance. Some have expressed their skepticism about the legitimacy of the experimental treatments.

"You have some people who are concerned for you, that your approach is not going to be effective,'' Dunlap said.

"Yet, several folks up herehave said, 'John, I'd take a shot at it. It is expensive but you're the one with the white cane and the one who is blind and has to live with it. You have everything to gain and nothing to lose.'''

While some might be concerned about the unusual treatments, many others are inspired by the Dunlaps,saidBlanche Tosh, a fellow church member and friend since high school.

"I have told them so many times, 'You just can't begin to know the lives you have affected,'' Tosh said.

"I know so many people who look at the way they are dealing with multiple things. How could anybody endure that and just go on and be pleasant and make it from day to day with the consistent attitude that the world sees.

"You are not going to find many people whoever see one of them without a smile,'' Tosh said.

She was inspired to start a gofundme account (gofundme.com/johndunlapvision) to help coverthe Dunlaps' expenses. As of midweek, $8,795 of the $100,000 goal had been raised.

Memphis lawyer John Dunlap and his wife Marcia continue to search for some medical procedure to restore at least partial vision after John was blinded a few years ago when their mentally ill son attacked him. (Photo: Jim Weber/The Commercial Appeal)

Since December, Dunlap has undergone two-and-a-half rounds of leech therapy and two series ofstem cell treatments. The couple traveled to California in June for the most recent stem cell procedures, and returned home with stem-cell eye drops and injections.

Nowthey are in the middle of the leech therapy they resumed this summer.

John has a follow-up exam next week, when he will learn if there's been continued progress from the stem cell and leech therapies.

The California doctor "indicated it would take two to three months to see if we were getting any results from stem cell therapy out there,'' Dunlap said. That time could come sometime this month or in September.

If the stem cell therapy has not worked by then, he said,"We'll just have to see what any third plan looks like, and the cost involved.''

Late in life, Dunlap has been forced to learn to type, work a computer, navigate with a cane, count the steps and memorize the turns from one spot to another, communicate with Siri, and smile as blood-sucking leeches dangle from his cheeks.

Asked about his sources of inner-strength, he responded, "I don't know I'd call it inner-strength.

"I can tell you I certainly believe in the Lord. We pray daily. I appreciate the prayers of others. I think it certainly is a faithissue.''

He also credits his late mother, Cora, a single parentwho managed a grocery. "She was a very optimistic, loving person,'' he recalled.

"And I've had Marcia's support. Marcia wasn't going to let me give up, just sit down and do nothing.''

The Dunlaps are starting to consider resuming their annual trips to Cubs spring training in Arizona. Maybe next spring.

"You may have your vision by then,'' Marcia told John.

"I might,'' he responded."We'll see.''

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Will putting leeches on his face help this blind man see? - USA TODAY

Skin Stem Cells Comments Off on Will putting leeches on his face help this blind man see? – USA TODAY | August 5th, 2017

Affimed Therapeutics (NASDAQ:AFMD)

Q2 2017 Earnings Conference Call

August 1, 2017 8:30 AM ET

Executives

Anca Alexandru Head of Communications

Adi Hoess Chief Executive Officer

Florian Fischer Chief Financial Officer

Analysts

Maury Raycroft Jefferies

Do Kim BMO Capital Markets

Michael Schmidt Leerink

Peter Lawson SunTrust

Operator

Good day and welcome to the Affimed Second Quarter 2017 Financial Results and Corporate Update Conference Call. Todays conference is being recorded. At this time, I would like to turn the conference over to Anca Alexandru. Please go ahead.

Anca Alexandru

Thanks. I would like to welcome you to our investor and analyst call on the results for the second quarter of 2017. On the call with me today are Adi Hoess, CEO of Affimed, who will present the corporate update; and Florian Fischer, Affimeds CFO, who will walk you through the financials.

Slide 2, before we start, please note that this call and the Q&A session contains forward-looking statements, including statements regarding our future financial condition, business strategy, and our plans and objectives for our future operations.

These statements represent our beliefs and assumptions only as of the date of this discussion. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

These forward-looking statements are subject to risks and uncertainties and actual results may differ materially from those expressed or implied in the forward-looking statements due to various factors including, but not limited to those identified under the section entitled risk Factors in our filings with the SEC and those identified under the section entitled cautionary statements regarding forward-looking statements in our Form 6-K filed with the SEC earlier today.

Thank you for your understanding. I will now hand the call over to our CEO, Adi Hoess, who will provide the corporate update.

Adi Hoess

Thanks a lot, Anca. Affimed has developed an immune cell engager and our clinical and preclinical pipeline based on tetravalent bi and trispecificantibody formats. Were an industry leader in NK cell engagement and our lead product candidate AFM13 is to our knowledge, the most advanced NK-cell engager in clinical development.

We also have a well-differentiated T-cell based approach, which includes our clinical candidate AFM11 and well provide an update on these clinical programs as well as our pre-clinical programs today. We employ about 75 full time equivalents with our headquarter located in Heidelberg, Germany, and affiliate offices in the U.S., that is Affimed Inc., as well as our subsidiary AbCheck in Plze, in the Czech Republic.

Slide 4. We have an unencumbered clinical and pre-clinical pipeline of NK and T-cell engagers, with our NK-cell engagers being developed in hematological diseases and solid tumors. Based on our NK-cell platform, we have one clinical and two pre-clinical programs in developments. And based on our T-cell platform, we have one program in our own clinical development. And second T-cell engager program based on our platform called AMV564 is being developed by Amphivena, a company of which we own about 18.5% fully diluted. AMV564 has recently entered clinical development.

Slide 5 summarizes our second quarter updates for our NK cell engager program. For AFM13, we have completed the dose escalation part of our Phase 1b combination study with Mercks Keytruda in Hodgkin Lymphoma and initiated the expansion phase. The AFM13 Phase 2a monotherapy trial in Hodgkin Lymphoma sponsored by the German Hodgkin Study Group is open to recruit under new study design, which includes patients pre-treated with both brentuximab vedotin and anti-PD1.

Columbia University has recently initiated a translational study of AFM13 in CD30-positive lymphoma with cutaneous manifestation and I will provide more detail later. We made further progress in our collaboration with MD Anderson Cancer Center to evaluate AFM13 in combination with MD Andersons NK cell product. In June, we presented new data for our NK cell engagers AFM24 and AFM26 at two conferences and I will go into detail later on this.

Slide 6 summarizes the progress, we have made with our T-cell engager. Two Phase 1 dose-escalation studies are ongoing with AFM11, which offer a significant opportunity to address the high unmet medical need in diffuse large B-Cell lymphoma and mantle cell lymphoma. We believe that both the properties of AFM11 and the design of our studies can attract, specifically, mutations of other drugs in development.

Both dose escalation studies, which are conducted in ALL and in NHL respectively are designed with accelerated titration followed by a classical 3+3 design. In both studies, AFM11 was overall well tolerated with no dose limits and toxicity observed to date. In the AFM11 study in relapsed refractory ALL, which was initiated in September 2016, patients are currently being recruited into the fourth dose cohort. 12 sites are open and recruiting in the Czech Republic, Poland, Russia, Austria and Israel.

As mentioned, no DLTs were observed in particular no AFM11 related grade 3 or grade 4 neurotoxicity or side effects are frequently observed with T-cell engaging antibody agents observed. In our study of AFM11 in relapsed refractory NHL, patients are currently being recruited into the third dose cohort. Recall that this study has been amended in the past to enroll patients under a new revised study design.

We believe that we have addressed this lower than effective recruitment by opening further trial sites. A total of 10 sites are now opened in the Czech Republic, Poland, Germany as well as the U.S. Like in our ALL trial, no AFM11 related grade 3 or grade 4 neurotoxicity was observed to date under their revised study design. We intend to provide regular update on both the AFM11 studies in the future.

A second T-cell engager program based on our platform is AMV564, a bispecific tetravalent CD33/CD3 antibody developed by Amphivena in AML. A Phase 1 study is recruiting. However, no further updates have been provided by Amphivena.

Slide 7 shows our platform, which is very distinguished from others as, in contrast, the most comparatives were developing tetravalent bispecific molecules. The bivalent binding of two receptors on two different cells enables high-affinity binding through the avidity effect, which is advantageous to maintain high specificity at very high affinity.

We believe that this is very important in order to obtain a favorable safety profile. Furthermore, our platform allows multi-specificity in the tailored PK. Further differentiating Affimed, while most immune cell engaging approaches to date focus on T cells, our technology platform reliably generates both T and NK-cell engagers.

While increasingly NK-cells are becoming a cornerstone of cancer immunotherapy, and we're excited to be pioneering this development. There are a number of reasons why NK-cell-based approaches are very attractive and one of the reasons is that there seems to be a positive correlation between NK-cell infiltration and clinical outcome in patients.

In this context, it has been described that a low cytotoxicity is associated with higher incidence of cancer. In addition, recent clinical data show improved anti-tumor responses of ex vivo expanded and activated NK-cell populations. NK-cell-based immunotherapy has recently advanced with different treatment approaches, including engagers, check points, cytokines and adoptive cellular transfer.

To-date, it seems that NK-cell-based approaches have this strong advantage of controlling a well manageable favorable safety profile. This creates an opportunity for NK-cell redirection to address the lack of recognition of cancer cells and also allows for potential combination of NK-cells with other approaches to enhance efficacy.

A common theme in all different cancer types is the ability of the tumor cell to evade recognition by the immune system and, specifically, by NK-cells as shown on Slide 9. Normally, NK-cells are capable of killing foreign or aberrant cells, like tumor cells, have acquired mechanisms to escape the so-called immune surveillance.

As a result, such NK-cells cannot recognize tumor cells as foreign or aberrant and, therefore, cannot fight them. We believe that our platform has the potential to overcome these limitations by disabling the tumor evasion mechanisms, and I will explain on the next slide what this belief is based on.

Our expertise and leadership in natural-killer cell-based approaches is one of our key assets. As we can see here, there are a multitude of activating and inhibitory NK-cell receptors being discovered that CD16A, a dominant activating receptor on innate immune cells, is the only activating receptor that triggers the cytotoxic activity of nave human NK cells, even in the absence of costimulatory signals.

Based on these properties and on our preclinical and clinical data generated to date, we believe that targeting CD16A is key for efficient recruitment of and killing by NK cells and macrophages. We have secured a solid IP position around CD16A targets.

Slide 10. We believe that through targeting CD16A with high affinity and specificity, the significant limitations of IgGs can differ. With our tetravalent bispecific immune cell engagers, we can restore NK-cell killing in tumor immune control, and this is depicted here.

Let me explain in more detail why we believe that our approach is superior compared to IgG-based approaches. The human body is not using NK-cell engagement by IgG to eliminate cancer cells. However, this mechanism is used for cells infected by viruses or bacteria.

In this situation, the human immune system generates a collagen antibody response that highly decorates such infected cells or organisms.

Highly decorated means that many different proteins are expressed on the cell surface, which can then be found bound by antibodies. This polyclonal and high-density binding leads to NK-cells killing upon high avidity XP binding, plus antibodies for CD16A on the NK-cell and other XP gamma receptors, for example, CD32 and CD64.

In the setting of targetable cancer cells, however, with IgG, the situation is very different. Firstly, the therapeutic molecule targets a single epitope. Hence, it confers ammonia killing response. And secondly, there are cancer cells which express only very low numbers of the desired target.

The consequence of this very low target density is an insufficient amount of IgG, decorating the cancer cells and thereby not being able to efficiently recruit immune cells. This is shown in the middle picture. Interesting, most therapeutic monoclonal antibodies are target-modulating antibodies, such as cetuximab, polatuzumab, gevokizumab, just to mention a few of them.

We are addressing this limitation by targeting CD16A with high affinity and specificity, as shown. Indeed, our immune cell engagers has the potential to elicit a robust NK-cell killing and immune control due to multivalent and apparent high-affinity binding to CD16A even at limiting antigen densities on the target.

Slide 11, furthermore, CD16A in confers additional superior engager features. The binding of immune cells through CD16A with high affinity and specificity induces NK-cell activation, which triggers an integrated immune response that can be mediated by both innate and adaptive immune cells. In particular, our NK-cell engagers do not bind to CD16B and neutrophils, which avoids the sync effect. Their affinity has been demonstrated to be over 1,000 fold higher than that of monoclonal antibodies and our engagers bind independently of the 158 valine phenylalanine polymorphism.

Most importantly, theres virtually no competition with plasma IgG, which is shown here. In the ground stage, CD16A on innate immune cells is occupied by polyclonal plasma IgG. But there is a huge excess of plasma IgG versus therapeutic antibodies, this creates a significant threshold for FC-based therapeutic antibodies, however, not for CD16A target enhancement.

Our tetravalent and bispecific molecules, which recognize a different epitope from CD16A, are virtually unaffected by plasma IgG. All these unique features result in overall increased potency and efficacy of NK-cell engagers.

Slide 12. Our lead candidate, CD30/CD16A-specific NK-cell engager, AFM13 is a first-in-class antibody suitable for mono and combination therapy. This has demonstrated safety and clinical activity in heavily pretreated Hodgkin lymphoma patients in a Phase 1 study. In this Phase 1 study, tumor shrinkage and potential responses were observed in patients treated with four weekly doses of at least 1.5 mg/kg of AFM13. In 62% of patients, which was eight out of thirteen patients, we observed tumor shrinkage in 23% of patients, which was a total of three out of thirteen experienced partial response. None of the patients experiencing a PR had been previously treated with brentuximab vedotin.

Recall, that in our investigation the Phase 2a trial for AFM13 in relapsed and refractory Hodgkin lymphoma, which is led by the German Hodgkin Study Group, we have previously guided to change the study protocol to ensure a recruitment of a homogeneous patient population pre-treated with both BV and anti-PD1 antibodies. The study is now open to recruit under the new study design.

We had also provided some preliminary data from patients enrolled under the original study protocol, where partial responses were observed in two of seven evaluated patients who had been pre-treated with brentuximab vedotin, but were anti-PD1 naive. This suggests, now, for the first time that AFM13 is active as a single agent in this heavily pre-treated group of patients and, in particular, that AFM13 is active post brentuximab vedotin. We have learned from the study sponsor that both after-responders had failed BV as the most recent treatment prior to AFM13 therapy, with one patient experiencing stable disease and the other one partial in the progressive disease under the BV treatment.

As previously guided, full data from the ongoing study will be presented upon its anticipated completion in 2019. And prior to that, a decision of data publication time points will be made together with the German Hodgkin Study Group.

We are further developing AFM13 as a combination therapy. Preclinical affinity has been demonstrated in combination with anti-PD1 in vivo in a PDX model. This has been the basis of our Phase 1b trial in relapsed refractory Hodgkin lymphoma in combination with Mercks Keytruda. And here, we have completed the dose escalation part of the trial. In detail, three patients were enrolled into dose levels one and two, respectively, and six patients were enrolled into dose level three. While no grade three or four adverse events related to the study treatment were observed, one DLT was observed in cohort 3, which was a repeated grade two infusion-related reaction, leading to discontinuation of AFM13 treatment. This event is classified as a DLT according to the protocol definition. No further DLTs occurred.

The dose expansion cohort has been initiated with the highest dose explored during dose escalation. Data readout is ongoing in the treated cohort and we intend to present data from the dose escalation at a scientific medical conference in the second half of 2017.

Another update this quarter is that Columbia University has initiated a translational Phase 1b/2a study to evaluate the validity of activity of AFM13 in patients with relapsed and refractory CD30-positive lymphoma with cutaneous manifestation. Affirmed is supporting this trial which is designed to allow for serial biopsies, thereby enabling assessment of NK-cell biology and tumor cell killing within the tumor environment. The first patient was enrolled into the study in July 2017. In general, we view CD30-positive lymphoma as an attractive indication that may broaden the potential of AFM13. In terms of further guidance, we will work together with Columbia University to provide update on this study.

Slide 13. Additional opportunities for our NK-cell engagers include combinations with adoptive NK-cell transfer. Patients on NK cells can be stimulated by monotherapy using NK-cell engagers to overcome tumor immune evasion and immunosuppression. Ex vivo expansion and stimulation of autologous NK-cells followed by reinfusion alone or in combination with NK cell engager, is a viable therapeutic approach providing increased numbers of activated NK cells. Alternatively, NK cells can be derived from peripheral blood, cord blood or IPS cells from healthy donors, which is an allogeneic setting, or from immortalized cells. After ex vivo stimulation and expansion, the NK cells are infused into the patients in combination with NK cell engagers.

We are investigating this approach with our partner MD Anderson. Initially, we plan to investigate AFM13 with MDACCs NK-cell product in the transplant setting. Preclinical research activities are on track and these are intended to be followed by Phase 1 clinical trial. Proof-of-concept for this combination would also pave the way for combinations of other pipeline product such as for AFM23.

Affimed holds an option to exclusive worldwide rights to develop and commercialize any product developed under the collaboration. In addition to our clinical product candidates, we have created a strong preclinical pipeline. Over the last quarter we have further characterized our most advanced preclinical candidates, AFM24 and AFM26, which we are developing for three solid tumors and multiple myeloma respectively.

Despite several marketed agents such as cetuximab and tyrosine kinase inhibitor or TKIs, there is a significant medical need for a novel approach to treat EGF receptor-positive tumor. Both efficacy and toxicity can be addressed. EGFR-blocking drugs have been described to have side-effects including serious skin toxicity which might impact physicians willingness to prescribe a drug. In terms of efficacy, there is a need to overcome intrinsic or acquired resistance. For example, there is no clear indication of efficacy of EGFR-blocking antibodies in patients with RAS mutation.

We are developing a first-in-class NK cell engager designed to overcome the limitations of conventional therapy. AFM24 is designed to effectively treat EGFR-expressing solid tumors, such as lung and neck, or colon cancers. It is an EGFR/CD16A targeting tetravalent bispecific antibody that is well differentiated from cetuximab, it is more potent cytotoxicity in vitro and in vivo including a potential to kill RAS-mutant cell lines. There is novel mechanism of action in safety profile and it has the potential to overcome intrinsic or acquired resistance, which is described by many patients with EGFR positive tumors.

AFM24s potent NK cell recruitment may enable the shift of the validated target EGF receptor, primary receptor block toward immuno-oncology. We have identified several development candidates for which we have initiated IND-enabling studies.

Slide 15, there are several factors which differentiate AFM24 from other therapy. Firstly, AFM24 is differentiated through its efficacy. Here you can see that in vitro, our NK cell engager which is highly potent tumor cell killing independent of RAS mutational status. In vivo, we have demonstrated efficacy in tumors resistant to EGFR targeting agents. Importantly, as shown in the graphs on the right hand side, AFM24 was similarly efficacious in a cetuximab-sensitive model.

Secondly, AFM24 is differentiated through safety, Slide 16. We have completed pilot toxicity studies in cynomolgus monkeys with no major safety findings. At the EACR-AACR-SIC Special Conference, we presented data on a dose-range binder study in which AFM24 was dosed up to 93.75 mg/kg and a repeated dose study in which AFM24 was dosed up to 30 mg/kg in 4 weeks.

No AFM24-related macro or microscopic changes were seen in tissues including vital organs, skin and injection site. Importantly, there was no evidence of skin toxicity in those studies. Also no signs of delayed toxicity was observed in the repeated dose study recovery animals. On a molecular level, we learned from in vitro toxicology studies but there was no cytokine release or NK cell proliferation in the absence of target cells. This further substantiates AFM24s potential beneficial safety profile.

Slide 17, like for EGFR targeted tumors, there is a significant need for a novel approach to treat multiple myeloma. Even though, new therapies have significant improved outcomes, cure still remains elusive and the medical need to achieve minimal residual disease negativity is not yet addressed.

MRD positivity is associated with a poorer prognosis, and it has been recorded that persistent MRD by predictive marker of unsustained complete response. A particular hurdle for therapeutics aimed at immune cell engagement are very high M-protein serum levels up to 170mg/mL. Indeed the competition by serum IgG is known to strongly impair antibody-dependent cell-mediated cytotoxicity, the activity of monoclonal antibodies.

We are developing AFM26 to overcome the limitations of conventional therapies in multiple myeloma. AFM26 is a first-in-class tetravalent bispecific antibody targeting BCMA/CD16A. Targeting BCMA and employing NK cell engagement offers the potential to achieve MRD-negativity. For AFM26, NK cell binding is largely unaffected by circulating IgG, which creates the potential of NK cell activation in the presence of M-protein.

Indeed, the high affinity binding to both target and NK cells leads to a prolonged cell retention. This is shown on the right on the slide on the right bottom. AFM26 shows high cytotoxicity cytotoxic activity towards both low and high BCMA-expressing myeloma cells. AFM26 may be potentially safer than T cell-based approaches, which would allow for faster development timelines. Based on these characteristics, AFM26 might be positioned in first line of combination with adoptive NK-cell transfer during ASCT or in a salvage setting.

AFM26 binds the B-cell maturation antigen, which is an antigen ubiquitously expressed on malignant plasma cells. Its expression on healthy tissues is limited to plasma cells and peripheral dendritic cells. We believe the BCMA is an ideal target for immunotherapy of multiple myeloma.

At ASCO and at the EACR-AACR-SIC, both in June, well present the data on AFM26 NK-cell binding properties and activity. As shown here, these data underscore that compared to native and FC-enhanced IgG. AFM26 shows improved binding and cell surface retention.

Slide 20, we also show that AFM26 is well differentiated through target cell binding in potent NK-cell mediated tumor cell lysis. And this is shown here in comparison with two marketed agents, daratumumab and anti-CD38 antibody and elotuzumab, which targets PS1. Importantly, other than described for daratumumab and elotuzumab, AFM26 did not induce NK-cell mutation.

Slide 21, like our other NK-cell engagers, AFM26 is also well differentiated for other agents [indiscernible] safety. Here you can see that compared to a T-cell engager, AFM26 is similarly potent that shows a reduced cytokine release pattern. This point is going to improve safety profile, making AFM26 uniquely suited to engage NK-cells with multiple myeloma.

I will now hand over the call to our CFO, Florian Fischer, who will provide further details on the financial figures.

Florian Fischer

Thank you, Adi. Affimeds consolidated financial statements have been prepared in accordance with IFRS as issued by the International Accounting Standards Board or IASB. The consolidated financial statements are presented in euro, which is the companys functional and presentation currency. Therefore, all financial numbers that I will present here in this call unless otherwise noted will be in euros. Any numbers referring to Q2 2017 and Q2 2016 are unaudited.

Cash and cash equivalents and financial assets totaled 48.9 million as of June 2017 compared to 44.9 million as of December 31, 2016. The increase was primarily attributable to the net proceeds of 16.4 million from a public offering of common shares in the first quarter, and of 2.5 million from the drawdown of the second tranche of the loan from Silicon Valley Bank, largely offset by operational expenses.

Net cash used in operating activities was 13.1 million for the six months ended June 30, 2017compared to 17 million for the six months ended June 30, 2016. The decrease was primarily related to lower cash expenditure for research and development in connection with Affimeds development and collaboration programs and to the expiration of the Amphivena collaboration.

Affimed expects to have cash to fund our operations at least until the end of 2018. This provides runway for the planned development of our clinical programs, as well as for product discovery and early development activity.

Revenue for the second quarter of 2017 was 0.5 million compared to 2.1 million for the second quarter 2016. Revenue in the 2017 period was primarily derived from AbCheck services, while revenue in 2016 period predominantly to Affimeds collaboration with Amphivena.

R&D expenses for the second quarter of 2017 were 5.4 million compared to 8.6 million for the second quarter of 2016. The decrease was primarily related to lower expenses for AFM13 and our discovery and early stage development activities and the expiration of the Amphivena collaboration.

G&A expenses for the second quarter of 2017 were unchanged at 2.0 million compared to the second quarter of 2016. Net loss for the second quarter of 2017 was 7.9 million, or 0.18 per common share, compared to a net loss of 8 million or 0.24 per common share for the second quarter of 2016.

The decrease of operating expenses was offset by lower revenue. In addition, the result was affected by finance costs of 1.2 million in the second quarter of 2017, whereas finance income of 0.5 million was shown in the second quarter of 2016.

I will now turn the call back over to Adi for a summary of our two clinical programs and our pipeline. Adi?

Adi Hoess

Thanks a lot Florian. Our strategy is to maximize the value of our unencumbered clinical and preclinical pipeline of NK-cell and T-cell engagers, as well as from our platform. Were leveraging our lead product, AFM13, for CD30-positive lymphoma initially focusing on the Hodgkin Lymphoma salvage setting enabling a fast development path and allowing the establishment of a cost efficient marketing and sales structure.

In addition, we believe investigating AFM13, both as monotherapy and in combination with Keytruda, reduces its development. Overall, our preclinical and clinical strategy is designed from the scientific leadership of our NK-cell platform with CD16A as proprietary target. We are expanding the preclinical and clinical activities of our tetravalent and bispecific NK-cell engager platform in solid tumors with our preclinical candidate AFM24 and in hematologic diseases, where we intend to leverage additional opportunities for AFM13 and AFM26, for example, in combination with adoptive NK-cells. We also develop T-cell engagers and our lead T-cell engager, AFM11, is being investigated in two ongoing ALL and NHL trials. BMV564, a T-cell engager derived from our technology platform, is in clinical development through Amphivena to treat AML.

In addition, as mentioned earlier, moving beyond our standard format, we are developing different tetravalent bispecific antibody formats tailored to specific indications and patient populations. And as outlined in previous earnings calls, we have more projects ongoing at the discovery stage and preclinically, including molecules developed from our MHD type complex targeting platform.

Thank you very much for your interest. The call is now open for questions.

Question-and-Answer Session

Operator

Thank you. [Operator Instructions] Our first question now comes from Maury Raycroft from Jefferies. Please go ahead.

Maury Raycroft

Good morning. Thanks for taking my questions. So I was wondering if you can mention what the AFM13 dose was that the DLT patient received in the combo trial? And then what youre going to use in the expansion cohort? And then is this dose higher, lower or in line with your predictions?

Adi Hoess

Hi, Maury, this is Adi. What we have done is we have used or given a PD-1 as its active dose and have dosed up AFM13 under the following strategy, under the following regime. We always are initially giving AFM13 three times per week for two weeks. Then, we give AFM13 once weekly for six weeks and, subsequently, we dose AFM13 every three weeks. The starting dose was 0.15 mg/kg and then switching to 0.5 mg/kg, the next one was 0.5 mg/kg going to 1.5 mg/kg, and the highest dose was 3 mg/kg going then to 7 mg/kg. So the three is always three times per week, and the seven is the weekly or every three weeks. We have seen 1 DLT in the highest dose. So at three times 3 mg/kg and once 7 mg/kg then have included an additional three patients and have not observed another DLT. So thats why we decided to go with the highest dose of 3 mg/kg three times per week subsequently given and then subsequently followed by 7 mg/kg.

Maury Raycroft

Got it, okay. And you also mentioned earlier about the two PRs generated with the monotherapy treatment? And I think you said there is a stable disease, but I missed some of the additional context, and I was just wondering if you can recap that for me?

Adi Hoess

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Affimed Therapeutics' (AFMD) CEO Adi Hoess on Q2 2017 Results - Earnings Call Transcript - Seeking Alpha

IPS Cell Therapy Comments Off on Affimed Therapeutics’ (AFMD) CEO Adi Hoess on Q2 2017 Results – Earnings Call Transcript – Seeking Alpha | August 5th, 2017

THURSDAY, Aug. 3, 2017 (HealthDay News) -- Scientists have created genetically altered skin cells that may control type 2 diabetes in lab mice. And they believe the general concept could someday be used to treat various diseases.

Using a combination of stem cells and "gene editing," the researchers created patches of skin cells that were able to release a hormone called GLP1 in a controlled manner.

The hormone, which is normally produced in the digestive tract, spurs the production of insulin -- the body's key regulator of blood sugar levels.

The scientists found that transplanting the engineered skin patches onto diabetic lab mice helped regulate their blood sugar levels over four months.

Xiaoyang Wu, a stem cell biologist at the University of Chicago, led the "proof of concept" study. He said it raises the possibility that "therapeutic skin grafts" could be used to treat a range of diseases -- from hemophilia to drug dependence.

Wu's team focused on type 2 diabetes in these initial experiments because it's a common condition.

However, a researcher not involved in the study doubted the usefulness of the approach for diabetes specifically.

People with type 2 diabetes already manage the disease with diet, exercise and medications -- including ones that target GLP1, said Juan Dominguez-Bendala.

Using high-tech gene therapy to get the same result seems unlikely, said Dominguez-Bendala, an associate professor at the University of Miami's Diabetes Research Institute.

"I don't see something like this coming to the clinic for diabetes," he said.

But Dominguez-Bendala also pointed to what's "cool" about the experiments.

Wu's team used a recently developed technology called CRISPR (pronounced "crisper") to create the skin patches. The technique, heralded as a major breakthrough in genetic engineering, allows scientists to make precision "edits" in DNA -- such as clipping a particular defect or inserting a gene at a specific location.

Before CRISPR, scientists could not control where an inserted gene would be integrated into the genome. It might end up in a "bad" location, Dominguez-Bendala explained, where it could, for example, "awaken" a tumor-promoting gene.

Wu and colleauges used CRISPR to make specific edits in GLP1, including one that allowed the gene to be turned "on" or "off" as needed, by using the antibiotic doxycycline.

The modified gene was inserted into mouse stem cells, which were then cultured into skin grafts in the lab. Finally, those grafts were transplanted onto lab mice.

The researchers found that when the mice were fed food with tiny amounts of doxycycline, the transplanted skin released GLP1 into the bloodstream. In turn, the animals' insulin levels rose and their blood sugar dipped.

The engineered skin also seemed to protect the mice from the ravages of a high-fat diet. When the mice were fed a fat-laden diet, along with doxycycline, they gained less weight versus normal mice given the same diet. They also showed less resistance to the effects of insulin, and lower blood sugar levels.

According to Wu, the study lays the groundwork for more research into using skin cells as a way to deliver "therapeutic proteins."

For instance, he said, skin cells could be engineered to provide an essential protein that is missing because of a genetic defect. As an example, he cited hemophilia -- a genetic disorder in which people lack a protein that allows the blood to clot properly.

Skin cells could be an ideal way to deliver such therapies, Wu said.

For one, the safety of skin grafts in humans is well-established, he pointed out. Since the 1970s, doctors have known how to harvest skin stem cells from burn victims, then use those cells to create lab-grown skin tissue.

Because the skin is generated from a patient's own stem cells, that minimizes the issue of an immune system attack on the tissue.

Dominguez-Bendala agreed that using skin cells has advantages. For one, he noted, the skin graft can be easily removed if something goes awry.

But a lot of work remains before therapeutic skin grafts could become a reality for any human disease. And research in animals doesn't always pan out in humans.

A next step, Wu said, is to see whether the skin grafts maintain their effects in lab mice over a longer period. The researchers will also monitor the animals for any immune system reactions against the GLP1 protein itself.

The findings were published online Aug. 3 in Cell Stem Cell.

The U.S. National Institutes of Health has a primer on gene therapy.

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Engineered Skin Cells Control Type 2 Diabetes in Mice: Study - Arizona Daily Star

Skin Stem Cells Comments Off on Engineered Skin Cells Control Type 2 Diabetes in Mice: Study – Arizona Daily Star | August 4th, 2017

We lost an American icon Thursday with the death of actor and playwright Sam Shepard. He had ALS (amyotrophic lateral sclerosis), more commonly known in the U.S. as Lou Gehrigs disease. Its an invariably fatal neurological disease that robs individuals of their ability to move muscles, their ability to swallow, and eventually, their ability to breathe.

ALS often starts in a fairly nonspecific way, with weakness in a persons hand or foot. Although I never examined the late Mr. Shepard, even in public photos from 2016, the atrophy of his hand muscle was evidenta hallmark of the loss of muscle that occurs in ALS.

In about 90% of cases diagnosed by neurologists, ALS happens out of the blueits sporadic, and the cause isnt known. About 10% of the time, ALS is inherited through a defective gene; that is, a patient has a family member who also had the disease. We can readily diagnose inherited ALS with a relatively simple blood test.

Five years ago, we learned that even in some patients who have no family history of ALS, a defect in a gene known as C9orf72 underlies the disease. In some patients, the disease may be initially diagnosed incorrectly as a nerve problem in the hands or wrist (carpel tunnel syndrome), or a pinched nerve in the neck or back. But those conditions are commonly associated with painALS is not generally a painful disease.

The weakness typically progressesslowly over many years in some patients, or rapidly over a few months in othersprogressing from one hand to the other, from hand to foot, or foot to hand. Eventually it affects ones ability to chew, swallow, and breathe. The weakness of the breathing muscles is what makes ALS fatal. Unlike cancer, with its rare but real remissions, ALS is always fatal. Patients might choose to have a ventilator artificially breathe for them; that intervention delays death, but not the progressive weakening and paralysis of all muscles.

As treating physicians, we have a paucity of options to slow down the disease and have no real effective drug to halt its relentless progression or to recover functionno cure. ALS is not really one disease, but a combination of different genetic, even environmental, insults, that culminate in this horribly disabling and life-ending malady. Not unlike what we have learned about cancers, there may be many different causesgenetic, molecular, biochemicalthat underlie the disease. In cancers, sampling the actual diseased tissue, commonly through tissue biopsies, has provided a trove of clues about what underlies the basis of the different cancers and how to approach the different forms, sometimes quite successfully. But with ALS, we cannot readily take a chunk of someones brain or spinal cord, so we are often left guessing as to what may underlie the cause of the disease and how to best treat it. That antiquated approach may soon end.

Advances in the generation of stems cells from individual patients provide the most powerful way to generate their own brain cells. We are now able to take a small tube of blood or skin and turn those cells into stem cells (by a procedure that won the Noble prize several years ago), and then, by adding a few more chemicals and special genes, turn those cells into motor neuronsbrain and spinal cord cells that die in ALS.

This procedure, which in essence creates a biopsy of the brain/spinal cord of ALS patients, will allow us to achieve what has been so successful in cancerto truly understand the different kinds of ALS, to use our patients brain cells to discover their individual disease causes, and to develop a more individualized pathway for drug therapy. We aim to personalize ALS therapywhat we call Answer ALS. That is the hope on the horizon for ALS, along with drugs now already under development or in clinical trials that are specifically targeted to patients with known genetic mutations. How far that horizon is in the distance, we dont know, but we can see it. We only wish Mr. Shepard and all our past patients could have reached that hopeful horizon.

Jeffrey D. Rothstein MD, PhD, a neurologist and professor at Johns Hopkins University, is the director of the universitys Brain Science Institute, ALS clinic and Robert Packard Center for ALS Research.

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Sam Shepard Died of ALS. Here's Why It's so Difficult to Treat. - Fortune

Skin Stem Cells Comments Off on Sam Shepard Died of ALS. Here’s Why It’s so Difficult to Treat. – Fortune | August 2nd, 2017

Though the jury is out on whether we should try to modify the genes of human embryos, that hasnt stopped researchers from finessing the widely lauded CRISPR gene-editing technique. So far three attempts by Chinese researchers have made the pitfalls clear: the technique introduces more errors than it fixes. It also produces mosaic embryos where some cells get fixed, others dont.

Now, as reported in Nature, an international team led by Shoukhrat Mitalipov at Oregon Health and Science University has found a way to move past these pitfalls. Its a staggering result, says geneticist Leanne Dibbens, at the University of South Australia. This is what weve all been looking for.

Shoukrat Mitalipov.

Dieter Egli, NYSCF

Mitalipov and his colleagues have convincingly repaired embryos carrying the faulty gene, cardiac myosin-binding protein C (MYBPC3). The defective gene causes hypertrophic cardiomyopathy, the most common cause of sudden cardiac arrest in young athletes. The condition affects one in 500 people. By using this technique, its possible to reduce the burden of this heritable disease on the family and eventually the human population, Mitalipov says. Every generation on would carry this repair because weve removed the disease-causing gene variant from that familys lineage.

Three previous attempts to edit the genes of human embryos by labs in China all showed problems with mosaicism and mistakes, so-called off-target effects. The first two of those studies used defective IVF embryos that could never develop into a baby (they had been inadvertently fertilised with two sperm) as a way to sidestep the ethical minefield.

The first study, published in 2015, attempted to repair a defective gene causing the blood disease beta thalassemia. The second study, published in 2016, edited a gene to confer HIV resistance to the embryo. The third, published in March this year, edited genes associated with the diseases beta thalassemia and favism. This time the researchers used normal embryos, which they found increased the proportion of embryos that were edited from 14% to 50%. Nevertheless the embryos still showed evidence of off-target effects and mosaicism.

The Mitalipov-led team is the first to demonstrate error-free editing of human embryos. They seem to have a knack when it comes to manipulating embryos. Mitalipov also carries the distinction of being the first to crack the long-standing problem of cloning human embryos and deriving embryonic stem cells.

The key to the current success appears to come down to when the CRISPR editor is introduced to the embryo. Past attempts introduced CRISPR once the embryo had already been fertilised; in the current report, CRISPR was added to eggs at an earlier stage, at the same time as the sperm.

The sperm came from a donor with hypertrophic cardiomyopathy. Like all those affected, he carried both a normal and a defective copy of the MYBPC3 gene so his sperm population was a 50:50 mix of normal and defective. That meant half the fertilised embryos would be normal; half defective.

The researchers co-injected the affected donors sperm together with the CRISPR editor. They then analysed the embryos after they had undergone two or three divisions. Out of 58 embryos, 42 showed the normal gene in every cell. This means the technique successfully increased the number of healthy embryos from 50% to 70%.

Researchers at collaborating labs in South Korea and China also carried out thorough checks of the embryos DNA to see if there had been mistakes elsewhere. Remarkably, no off-target effects were detected.

Another remarkable finding was the way the repairs to the embryos faulty DNA took place. Normally the CRISPR editor is added together with a snippet of DNA carrying the correct DNA code. It uses this as a template to make the corrections rather like checking a dictionary when you correct the spelling of word. The surprise was that instead of checking the foreign DNA to make the corrections, the embryo checked the mothers copy of the MYBPC3 gene. The preferential use of the mothers own template may have something to do with using very early stage embryos. It may also explain why the editing was so accurate. Says co-author Jun Wu of the Salk Institute in San Diego: Our technology successfully repairs the disease-causing gene mutation by taking advantage of a DNA repair response unique to early embryos.

The authors believe their success at avoiding mosaicism also lies in editing early embryos. By co-delivering the CRISPR editor with sperm, there was time for the embryo to carry out its repairs well before it began dividing, avoiding the possibility of cells splitting before receiving a corrected copy of the DNA.

Not all the embryos were perfectly fixed, though: 16 showed erroneous fixes to their MYBPC3 gene.

However, the authors say that, by increasing the number of healthy embryos from 50% to 70%, their work could provide couples with a larger number of healthy embryos, improving the chance of successful IVF. According to another co-author, Paula Amato, professor of obstetrics and gynaecology in OHSUs School of Medicine: If proven safe, this technique could potentially decrease the number of cycles needed for people trying to have children free of genetic disease.

Clearly there is still work to do and debates still to be resolved. As Dibbens puts it: The study advances our understanding of gene editing technologies and again highlights the need for discussions on what situations gene editing will be used in in the future.

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Error-free editing of human embryos achieved by US researchers - Cosmos

Cardiac Stem Cells Comments Off on Error-free editing of human embryos achieved by US researchers – Cosmos | August 2nd, 2017

Kristin Comella, Chief Science Officer

Chronic obstructive pulmonary disease (COPD) is the fourth leading cause of death in the United States, and is projected to be the third by 2020. COPD is associated with an exaggerated chronic inflammatory response causing airway abnormalities. Patients typically undergo a progression of declining lung function, characterized by an increase of cough, shortness of breath, and mucus production. Extra-pulmonary manifestations of COPD include osteoporosis, cardiovascular disease, skeletal muscle abnormalities, and depression. There is currently no cure and the manifestations can only be treated symptomatically. It afflicts more than 5% of the population in many countries and accounts for more than 600 billion in health care costs, morbidity, and mortality.

Adult stem cells are found in every part of the body and their primary role is to heal and maintain the tissue in which they reside. Stem cells are unspecialized cells capable of renewing themselves by cell division. In addition, they have the ability to differentiate into specialized cell types. Adult stem cells can be harvested from a patients own tissue, such as adipose (fat) tissue, muscle, teeth, skin or bone marrow. One of the most plentiful sources of stem cells in the body is the fat tissue. In fact, approximately 500 times more stem cells can be obtained from fat than bone marrow. Stem cells derived from a patients own fat are referred to as adipose-derived stem cells (ADSCs). Adipose derived stem cells have been explored with respect to their activity in diseases involving significant inflammatory or degenerative components. More recently, adult stem cells have been identified as having the potential to reverse the effects of diseases like COPD.

The mixed population of cells that can be obtained from fat is called a stromal vascular fraction (SVF). The SVF can easily be isolated from fat tissue in approximately 30-90 minutes in a clinic setting (under local anesthesia) using a mini-lipoaspirate technique. The SVF contains all cellular elements of fat, excluding adipocytes. Tens to hundreds of millions of ADSCs can be obtained in the context of the SVF acquired from 20-200 ml of adipose tissue during this out-patient procedure. This sets the stage for their practical use at the point-of-care, in which a preparation of ASC can be provided for infusion or injection after the mini-liposuction. COPD patients who have undergone stem cell therapies often express the willingness to receive additional cell infusions if possible, due to a feeling of well-being associated with the injection. There is early evidence of feasibility and safety of infusions into the patients with COPD. In relevant studies, intravenous infusion of cultured adipose stem cells has been demonstrated to remarkably improve the onset and progression of smoke exposure-induced emphysema in rodents.

Stem cells possess enormous regenerative potential. The potential applications are virtually limitless. Patients can receive cutting edge treatments that are safe, compliant, and effective. Our team has successfully treated over 7000 patients with very few safety concerns reported. One day, stem cell treatments will be the gold standard of care for the treatment of most degenerative diseases. We are extremely encouraged by the positive patient results we are seeing from our physician-based treatments. Our hope is that stem cell therapy will provide relief and an improved quality of life for many patients. The future of medicine is here!

For additional information on Stem Cell Centers of Excellences South Miami clinic, visit http://www.stemcellcoe.com.

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Stem Cells Offer New Solutions for Lung Disease - Miami's Community Newspapers

Skin Stem Cells Comments Off on Stem Cells Offer New Solutions for Lung Disease – Miami’s Community Newspapers | August 1st, 2017

Marie Grim of Langley is looking for 100 people between the ages of 17 and 35 who are willing to take a few moments to have their cheeks swabbed.

Theres no pain, no fuss, she said.

And you could save someones life, anywhere in the world.

You could match with someone in Japan.

The campaign to sign up more potential stem cell donors, people who are willing to allow DNA samples to taken using cotton swabs, was inspired by the experience of her sister-in-law.

Cloverdale resident Tania Grim, a mother of four was diagnosed with leukemia in January.

She had to wait several months before a compatible donor was found whose stem cells will be used to replace bone marrow and abnormal white blood cells eradicated by a combination of chemotherapy and radiation.

We have been on quite the journey, Marie said.

I have sat with her at appointments and heard others get news of their donor while she had not.

Now that Tania has her donor, Marie would like to improve the odds for other families.

She already has a location and tentative date to collect the swabs September 8 at Immanuel Christian Reformed Church in Langley if she can round up enough donors.

Tania, who is preparing for her stem cell procedure in September, urged prospective stem cell contributors to sign up.

I am so grateful that the word is being spread about the huge need for donors, Tania said.

It is a very simple thing to do that can save a life.

If you are the right age to be a donor, you can contact Marie at 604-530-1326 or by email at mariegrim@hotmail.com.

Interested donors can also contact Canadian Blood Services directly at https://blood.ca/en.

More than 390,000 Canadians have joined the OneMatch Stem Cell and Marrow Network registry maintained by Canadian Blood Services, volunteering to be stem cell donors for any patient in need of a transplant, anywhere in the world.

Right now, the agency says about 70 per cent of eligible donors on the registry are Caucasian, which means the odds of finding match for other ethnicities, such as Canadians with indigenous, Asian or African heritage, are not good.

The Canadian registry connects to an international network established by the World Marrow Donor Association (WMDA) that has access to over 28 million donors in over 53 countries.

Not everyone who registers will be matched to a patient and asked to donate, but each registrant provides hope for those waiting, a message posted to the agency website states.

A person could be a match within a few months of registering, a year later or even seven years later.

If a volunteer donor is found to be a match, they face a relatively minor surgical procedure and can expect to make a quick recovery.

The agency says over 80 diseases and disorders can be treated with a stem cell transplant.

There are hundreds of patients in Canada waiting for a match, but only half of them find a match.

Patients are more likely to find a matching donor from within their own ethnic group.

The odds of family members matching is extremely slight, the agency said, which is why it does not support donor drives targeting relatives.

RELATED STORY: Surrey teen rallies stem cell donors to help with desperate need for South Asians

dan.ferguson@langleytimes.com

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Drive for stem cell donors in Langley - Surrey Now-Leader

Bone Marrow Stem Cells Comments Off on Drive for stem cell donors in Langley – Surrey Now-Leader | August 1st, 2017

Cataracts are the single leading cause of blindness worldwide, afflicting roughly 42 percent of the global population, including more than 22 million Americans. The disease, which causes cloudy patches to form on the eye's normally clear lens, can require surgery if left untreated. That's why Google's DeepMind AI division has teamed with the UK's National Health Service (NHS) and Moorfields Eye Hospital to train a neural network that will help doctors diagnose early stage cataracts.

The neural network is being trained on a million anonymized optical coherence tomography (OCT) scans (think of a sonogram, but using light instead of sound waves) in the hopes it will eventually be able to supplement human doctors' analyses, increasing both the efficiency and accuracy of individual diagnoses.

"OCT has totally revolutionized the field of ophthalmology. It's an imaging system for translucent structures that utilizes coherent light," Dr. Julie Schallhorn, an assistant professor of ophthalmology at UC San Francisco, said. "It was first described in 1998 and it gives near-cell resolution of the cornea, retina and optic nerve.

"The optic nerve is only about 200 microns thick, but you can see every cell in it. It's given us a much-improved understanding of the pathogenesis of diseases and also their response to treatments." The new iteration of OCT also measures the phase-shift of refracted light, allowing doctors to resolve images down to the capillary level and observe the internal structures in unprecedented detail.

"We're great at correcting refractive errors in the eyes so we can give you good vision far away pretty reliably, or up close pretty reliably," Schallhorn continued. "But the act of shifting focus from distance to near requires different optical powers inside the eye. The way the eye handles this when you're young is through a process called 'accommodation.'" There's a muscle that contracts and changes the shape of the lens to help you focus on close objects. When you get older, even before you typically develop cataracts, the lens will stiffen and reduce the eye's ability to change its shape.

"The lenses that we have been putting in during cataract surgery are not able to mimic that [shapeshifting] ability, so people have to wind up wearing reading glasses," Schallhorn said. There's a lot of work in the field to find solutions for this issue and help restore the eye's accommodation.

There are two front-runners for that: Accommodating lenses, which use the same ciliary muscle to shift focus, and multifocal lenses, which work just like your parents' multifocal reading glasses except that they sit directly on the eye itself. The multifocals have been on the market for about a decade, though their design and construction has been refined over that time.

To ensure the lenses that doctors are implanting are just as accurate as the diseased ones they're removing, surgeons are beginning to use optiwave refractive analysis. Traditionally, doctors relied on measurements taken before the surgery to know how to shape the replacement lenses and combined those with nomograms to estimate how powerful the new lens should be.

The key word there is "estimate." "They especially have problems in patients who have already had refractive surgery like LASIK," Schallhorn explained. The ORA system, however, performs a wavefront measurement of the cornea after the cataract has been removed to help surgeons more accurately pick the right replacement lens for the job.

Corneal inlays are also being used. These devices resemble miniature contact lenses but sit in a pocket on the cornea that's been etched out with a LASIK laser to mimic the process of accommodation and provide a greater depth of focus. They essentially serve the same function as camera apertures. The Kamra lens from AcuFocus and the Raindrop Near Vision Inlay from Revision Optics are the only inlays approved by the FDA for use in the US.

Glaucoma afflicts more than 70 million people annually. This disease causes fluid pressure within the eye to gradually increase, eventually damaging the optic nerve that carries electrical signals from the eye to the brain. Normally, detecting the early stages of glaucoma requires a comprehensive eye exam by a trained medical professional -- folks who are often in short supply in rural and underserved communities. However, the Cambridge Consultants' Viewi headset allows anyone to diagnose the disease -- so long as they have a smartphone and 10 minutes to spare.

The Viewi works much like the Daydream View, wherein the phone provides the processing power for a VR headset shell -- except, of course, that instead of watching 360 degree YouTube videos, the screen displays the flashing light patterns used to test for glaucoma. The results are reportedly good enough to share with you eye doctor and take only about five minutes per eye. Best of all, the procedure costs only about $25, which makes it ideal for use in developing nations.

And while there is no known cure for glaucoma, a team of researchers from Stanford University may soon have one. Last July, the team managed to partially restore the vision of mice suffering from a glaucoma-like condition.

Normally, when light hits your eye, specialized cells in the retina convert that light into electrical signals. These signals are then transmitted via retinal ganglion cells, whose long appendages run along the optic nerve and spread out to various parts of the brain's visual-processing bits. But if the optic nerve or the ganglion cells have been damaged through injury or illness, they stay damaged. They won't just grow back like your olfactory sensory nerve.

However, the Stanford team found that subjecting mice to a few weeks of high-contrast visual stimulation after giving them drugs to reactivate the mTOR pathway, which has been shown to instigate new growth in ganglion cells, resulted in "substantial numbers" of new axons. The results are promising, though the team will need to further boost the rate and scope of axon growth before the technique can be applied to humans.

Researchers from Japan have recently taken this idea of cajoling the retina into healing itself and applied it to age-related macular degeneration cases. AMD primarily affects people aged 60 and over (hence the name). It slowly kills cells in the macula, the part of the eye that processes sharp detail, and causes the central focal point of their field of vision to deteriorate, leaving only the peripheral.

The research team from Kyoto University and the RIKEN Center for Developmental Biology first took a skin sample from a human donor, then converted it into induced pluripotent stem (IPS) cells. These IPS cells are effectively blank slates and can be coerced into redeveloping into any kind of cell you need. By injecting these cells into the back of the patient's eye, they should regrow into retinal cells.

In March of this year, the team implanted a batch of these cells into a Japanese sexagenarian who suffers from AMD in the hope that the stem cells would take hold and halt, if not begin to reverse, the damage to his macula. The team has not yet been able to measure the efficacy of this treatment but, should it work out, the researchers will look into creating a stem-cell bank where patients could immediately obtain IPS cells for their treatment rather than wait months for donor samples to be converted.

And while there isn't a reliable treatment for dry-AMD, wherein fatty protein deposits damage the Bruchs membrane, a potent solution for wet-AMD, which involves blood leaking into the eyeball, has been discovered in a most unlikely place: cancer medication. "Genentech started developing a new drug when an ophthalmologist in Florida just decided to inject the commercially available drug into patients eyes," Schallhorn explained.

"Generally this is not a great idea because sometimes things will go terribly wrong," she continued, "but this worked super-well. It basically stops and reverses the growth of these blood vessels." The only problem is that the drugs don't last, requiring patients to receive injections into their eyeballs every four to eight weeks. Genentech and other pharma companies are working to reformulate the drug -- or at least develop a mechanical "reservoir" -- so it has to be injected only once or twice a year.

Stem-cell treatments like those used in the Kyoto University trial have already proved potentially effective against a wide range of genomic diseases, so why shouldn't it work on the rare genetic condition known as choroideremia? This disease is caused by a single faulty gene and primarily affects young men. Similar to AMD, choroideremia causes light-sensitive cells at the back of the eye to slowly wither and die, resulting in partial to complete blindness.

In April of 2016, a team of researchers from Oxford University performed an experimental surgery on a 24-year-old man suffering from the disease. They first injected a small amount of liquid into the back of the eye to lift a section of the retina away from the interior cellular wall. The team then injected functional copies of the gene into that same cavity, replacing the faulty copies and not only halting the process of cellular death but actually restoring a bit of the patient's vision.

Gene therapy may be "surely the most efficient way of treating a disease," lead author of the study, Oxford professor Robert MacLaren, told BBC News, but its widespread use is still a number of years away. Until then, good old-fashioned gadgetry will have to suffice. Take the Argus II, for example.

The Argus II bionic eye from Second Sight has been in circulation since 2013, when the FDA approved its use in treating retinitis pigmentosa. It has since gotten the go-ahead for use with AMD in 2015. The system leverages a wireless implant which sits on the retina and receives image data from an external camera that's mounted on a pair of glasses. The implant converts that data into an electrical signal which stimulates the remaining retinal cells to generate a visual image.

The Argus isn't the only implantable eyepiece. French startup Pixium Vision developed a similar system, the IRIS II, back in 2015 and implanted it in a person last November after receiving clearance from the European Union. The company is already in talks with the FDA to bring its IRIS II successor, a miniaturized wireless subretinal photovoltaic implant called PRIMA, to US clinical trials by the end of this year.

Ultimately, the goal is to be able to replace a damaged or diseased eye entirely, if necessary, using a robotic prosthetic. However, there are still a number of technological hurdles that must be overcome before that happens, as Schallhorn explained.

"The big thing that's holding us back from a fully functional artificial eye is that we need to find a way to interface with the optic nerve and the brain in a way that we transmit signals," she said. "That's the same problem we're facing with prosthetic limbs right now. But there are a lot of smart people in the field working on that, and I'm sure they'll come up with something soon."

Read more from the original source:
High-tech solutions top the list in the fight against eye disease - Engadget

IPS Cell Therapy Comments Off on High-tech solutions top the list in the fight against eye disease – Engadget | July 13th, 2017

JOEL INESON

Last updated14:01, July 9 2017

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Andrea Cameron-Hill has lived with multiple sclerosis for about 10 years. She wants to receive treatment in Russia that could stop the disease.

Andrea Cameron-Hill thought having to lift her leg to get in the carwhile pregnant was part of carrying twins.

About 10 weeks after they were born she learned she had multiple sclerosis (MS).

"To start with, you wouldn't know I had MS at all. But now it's 10 years on and I'm having to walk with a crutch," she said.

IAIN MCGREGOR/STUFF

MS sufferer Cameron-Hill wants to receive treatment that could stop the disease in its tracks but, despite it being offered here to treat some cancer, must travel to Russia to get it.

"If I have to go down to the floor to load the fire with wood, the difficulty for me now is getting off the floor."

READ MORE: *Multiple sclerosis sufferer Royce Brewer cleared after experimental treatment in Russia *Multiple sclerosis patient to receive 'experimental' treatment in Russia *Friends rallying to help Upper Hutt woman reach Mexico for stem cell treatment *Marlborough woman's search for cure *Plea to help fund stem-cell treatment for Andrea Campbell *Hunt after cure for MS disease

Cameron-Hill has injured her shoulder fromfalls and reliedon her husband, Paul, and sons Lachlanand Oliverto help with household chores.

IAIN MCGREGOR/STUFF

Normal household chores like washing are a challenge because of the debilitating condition.

She wanted to do things like go onto the rugby field while her childrenplayed, but MS meant she had to watch from the car.

"We've got a basketball hoop [at home], which they quite like playing, but they normally play with Grandma because I can't."

"I feel like a spectator in their lives. I hate it. I hate it with a passion."

IAIN MCGREGOR/STUFF

Cameron-Hill's sons, Lachlan and Oliver, pictured, help her complete tasks many do effortlessly.

Cameron-Hill's condition drove herto look at a treatmentused in New Zealand for some forms of cancer, but not available for MS.

She plans to head to Russia for undergohematopoieticstem cell transplantation (HSCT).

HSCT would remove, purifyand concentrate her stem cells.Chemotherapy would wipeher immune system before the stem cells were returned. An extended period of recovery would follow.

IAIN MCGREGOR/STUFF

Cameron-Hill wants to take part in her children's lives, rather than watch from the sidelines.

Cameron-Hill mustraise about $80,000 to get the treatment.So far about $10,000 has been raised through fundraising events and aGivealittlepage.

Leading New Zealand neurologist Dr Deborah Mason said HSCT wasunlikely to be trialledin New Zealand because it wouldnot make drug companies money.

Treatment for MSin New Zealandreliesonimmunosuppressantdrugs.HSCTwaslikened more to a surgical procedure than drug treatment.

"It's incredibly expensive ... We're very keen to participate in [trials and research] and I'd certainly enroll patients, but it's just finding somebody who would fund that, and nobody will because there's no drug involved," Mason said.

Mason saidHSCT might help young patients in the early stages of MS.

She said the treatment was "unproven" and came with risks.

"It's really hard to imagine why I get all these calls about bone marrow transplants ... all bone marrow transplant allows us to [do is] give industrial doses of chemotherapy."

"People talk aboutrebootingthe immune system and all of that. There isn't a lot of proof of that."

Mason knew of about six or seven people who had travelled forHSCT, but did not have a lot of follow-updata.

Studies and clinical trials New Zealanders took part in primarily focussed on medication.

Her "big beef" was with the government not allowing the use of some drugs, "which we absolutely know will benefit the patients".

The Multiple Sclerosis Society of New Zealand recently changed its standpoint on HSCTafter a report indicated the treatment workedfor some with MS in Australia and the UK.

Vice president Neil Woodhams said the group asked the Ministry of Health to start a process to lead to eligible people havingHSCTin New Zealand.

Cameron-Hill's MS had not progressed since 2013and she had not been on medication for it since.

She recently met Christchurch manRoyce Brewer who lived with MS for about 20 years. After undergoing HSCT in Russia in early 2016, hereturned to work as a landscaper at the end of the year.

"I'd just really love to be able to do ... normal stuff that parents do with their kids," Cameron-Hill said.

The Health Research Council of New Zealandhad not funded any research regarding MS andHSCT, a spokeswoman said.

-Stuff

Read the original:
Christchurch MS patient Andrea Cameron-Hill plans Russia trip for stem cell treatment - The Press

Bone Marrow Stem Cells Comments Off on Christchurch MS patient Andrea Cameron-Hill plans Russia trip for stem cell treatment – The Press | July 9th, 2017