The excitement at Jerusalem-based Gamida Cell, a maker of cell and immune therapy technologies, is palpable.

The biotechnology company has started enrolling patients for a last-stage clinical trial for a drug it believes will help increase the success of bone marrow transplants in blood cancer patients, and help them better withstand the ordeal of the lifesaving procedure.

The patients are being enrolled in the US, Spain, The Netherlands and Singapore. Should the results of the trial, as hoped, be positive, that would lead to the launch of a commercially available product in 2020, Gamida Cells CEO Yael Margolin said in an interview with The Times of Israel.

We are at an exciting transition point, and moving from being a research and development firm, based in Israel, to an international commercial firm, said Margolin who has headed the company for the past 12 years in her sun-drenched office at the biotech firms headquarters in Jerusalem. We need to prepare to commercialize the product. We are now looking at various sites in Israel for a new manufacturing facility and looking to employ some 100 people. These workers will be added to the 40 already employed in Jerusalem.

Gamida Cells CEO, Dr. Yael Margolin (Courtesy)

Preliminary clinical data has already revealed that the risk of their leading product for blood cancers, NiCord, not meeting its targets in the Phase 3 trial, is low, added Margolin.

The drug has already received a breakthrough therapy designation by the US Food and Drug Administration (FDA). The designation is given to a drug that is meant to treat a serious or life-threatening condition, and where preliminary clinical evidence indicates that it may demonstrate a substantial improvement on at least one clinically significant target (endpoint) over other available therapies. The designation also entitles the company to get more and closer FDA guidance to help bring the treatment faster to patients.

The combination of the low clinical risk based on the previous trial results and the lower regulatory risk, because the drug is being developed in close collaboration with the FDA, has spurred the company into a flurry of activity that is aimed at scaling up its production facilities to get ready for the day NiCord hits the markets.

The company said last month it raised $40 million from investors including Novartis, which is already a major shareholder in the firm. The funds will support the ongoing Phase 3 stage for NiCord. The company also announced, on July 20, that it received a $3.5-million grant from the Israeli government that will support the further development of NiCord and other drugs that the company is developing, including therapies for sickle cell disease and for blood and solid cancers. Gamida has also appointed a new chief medical officer, Ronit Simantov, who will be based in the US.

The first market for our drug will be the US, Margolin said.

The Gamida Cell lab in Jerusalem where umbilical cord blood is stored in tanks, July 16, 2017. (Shoshanna Solomon/Times of Israel)

NiCord, which would be the first drug developed by Gamida to hit the market if the trial goes well is believed to increase the chances of a successful bone marrow transplantation process for patients who do not have a rapidly available, fully matched, bone marrow donor.

Today some high-risk blood cancers cannot be cured unless patients undergo a bone marrow graft. For that purpose, a perfect 100-percent match needs to be found, a process that in the US takes an average of three to four months, if the patient is lucky. Sometimes, no match is found.

There are 70,000 patients a year globally with blood cancers who need a bone marrow transplant, Margolin said. It is a rare condition. But for that transplant, you need a donor with full tissue matching. As many as 50% dont get to the transplant phase, because they havent found a matching donor in time.

Umbilical cord blood collected from newborn babies contains stem cells, which can be used to treat diseases. Today cord-blood banks around the world store the cord blood. It great advantage is that because it is so young, there is no need for a full tissue matching.

The big advantage with umbilical blood is that you dont need full tissue matching; a partial match is enough, Margolin continued. Most patients generally find at least one unit of cord blood that partially matches them.

Stem cells in a bag in Gamida Cells Jerusalem lab, July 16, 2017 (Shoshanna Solomon/Times of Israel)

The problem is that the quantity of cells in each unit is not huge, and it is the number of stem cells in the cord blood that is critical to the success of transplantation.

Our idea is to leverage the advantages of the cord blood and overcome the limitations of the cell number by applying our own platform technology, called NAM Technology, added Margolin. This technology allows us to take one unit of umbilical cord blood and expand the number of stem cells within it and enhance their performance.

Gamida Cell selects the stem cells from the unit and puts them in a culture together with a molecule called Nicotinamide (NAM) a form of Vitamin B3 and adds other ingredients. This culture, to which the firm holds intellectual property rights, increases the number of stem cells, and enhances their functionality, Margolin said.

The cells are then harvested from the culture, frozen in a small blood-bag in a final formulation that is ready for infusion, and then shipped to hospitals via couriers. Doctors thaw the product by the bedside of the patients and infuse the fluid into them.

From start to finish, our process takes three weeks, Margolin said. The average search for a bone marrow match takes three to four months.

The clinical trial underway is enrolling patients aged 16 years and older.

An earlier trial of the drug showed that patients transplanted with NiCord showed a more rapid engraftment the amount of time needed for the development of a minimal amount of white blood cells, or neutrophils, in the blood. That minimum amount indicates the patient is now less vulnerable to infections and bleeding following the transplant, and is an indication of success.

In the pilot phase clinical trials, the median time to neutrophil engraftment with NiCord was 11 days, compared to three to four weeks in patients who received standard umbilical cord blood. The results in a study conducted at Duke University also showed a lower rate of infection 22% vs 54%; and a lower duration of hospitalization compared to standard umbilical cord engraftment, Margolin said.

Now the company is enrolling patients for its larger, Phase 3 multi-national, randomized controlled registration study. And in February it said it had already transplanted its first patient, as part of the trial.

We hope to publish positive topline data from the Phase3 study in the first half of 2019 and launch the product on the market in 2020, she said.

Metal barrel with a frozen bag of umbilical cord stem cells ready for delivery from Gamida Cells Jerusalem lab, July 16, 2017. (Shoshanna Solomon/Times of Israel)

A metal barrel within which was a frozen bag of umbilical cord stem cells was waiting to be picked by a courier in the lobby of the Gamida Cell offices, ready to be thawed and injected into a patient somewhere around the world.

We have a sophisticated infrastructure that coordinates everything between the cord bank blood and our manufacturing site and the hospital where the patient is to be treated, Margolin continued. This infrastructure is 100% robust, but we plan to scale this up toward commercialization.

The $40 million in funds the company raised last month is expected to last until late 2019. After that, she added, all options are on the table: an IPO, or teaming up with a strategic partner, are both possibilities for the future.

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Cell therapy firm in flurry of activity as hope nears for bone marrow patients - The Times of Israel

Credit: University of California, San Francisco

A virus hiding quietly in the gut may trigger the onset of a severe complication known as graft-versus-host disease (GvHD) in patients who receive bone marrow transplants, according to a new study led by scientists at UC San Francisco and Saint-Louis Hospital in Paris, France.

GvHD affects up to 60 percent of patients who undergo bone marrow stem-cell transplants, and kills about half of those affected. After transplants, to prevent a recipient's immune cells from laying siege to unfamiliar donor cells and rejecting them, clinicians often use drugs to suppress the immune response. GvHD is a mirror image of organ rejection, in which immune cells in the transplant attack its new host, the patient.

Despite the pervasiveness of this disease, there isn't yet a clear way of foretelling patients' risk of developing it before they go into surgery. The new study, published online July 31, 2017, in Nature Medicine, unveils a viral biomarker that could allow clinicians to assess patients' risk of an acute form of the disease known as enteric GvHD, which affects the gastrointenstinal system.

The team used a technique known as metagenomic next-generation sequencing (mNGS) which can rapidly and concurrently sequence genetic material of all organisms present in any biological sample to catalog microbes in patients' digestive tracts, monitoring the evolving bacterial and viral population throughout the transplantation process.

Although mNGS analyses of bacterial populations, called microbiomes, have been much in the news, fewer studies have focused on "viromes," the term for viral populations.

"Viromes can play an important part in health and disease," said Charles Chiu, MD, PhD, an associate professor of laboratory medicine at UCSF and principal investigator of the study. "Our goal was to understand what impact transplantation has on the gut virome."

In the new work, the researchers scanned stool samples taken from 44 patients before they received a transplant and up to six weeks after, and sequenced all the DNA and RNA in the samples in order to assemble a roster of their microbial passengers.

Using this technique, the researchers identified a number of viruses that flared up in the guts of patients who developed the deadly condition. Of particular note were members of the picobirnavirus (PBV) family: the presence of these viruses before transplantation, even in very small populations, was a reliable sign that a patient would likely develop the disease after a transplant.

"I would've expected herpesviruses or adenoviruses to be the more likely cause of infection," said Chiu. "We wouldn't have picked up picobirnaviruses were it not for the metagenomics approach."

PBVs are a very diverse family of viruses more diverse than HIV, said Jrme Le Goff, PhD, associate professor at the University of Paris Diderot and lead author of the new study. "It's very difficult to design a single test to detect all viruses simultaneously," said Le Goff. "So for many years, labs did not have the means to look for PBV." Indeed, each of the 18 patients who tested positive for PBV was carrying a different strain, a diversity that makes it challenging to detect PBVs using a simple lab test.

The team also observed a previously unreported "bloom" of other resident viruses in patients that occurred three to five weeks after they had received transplants. Intriguingly, the onset of GvHD appeared to trigger the late awakening of these covert viruses, laying to rest a longstanding chicken-and-egg debate: which comes first, viral infection or GvHD? The researchers conclude that much of the viral flare they saw is due to reactivation of latent gut infections following transplantation.

Given the potential utility of PBV as a predictive biomarker, Chiu and his team now hope to develop a metagenomics-based test to screen patients before transplantation. "We also saw shifts in the microbiome but those in the virome were more pronounced," said Chiu. "Loss of bacteria colonizing the gut has been thought to predispose patients to GvHD; here we show that shifts in the virome may also play a role in the occurrence of this disease."

Although the new study strongly implicates PBVs in the onset of GvHD, it is too early to tell whether or how these viruses trigger the disease. The team is now enrolling more adult and pediatric patients both in Paris and at UCSF to expand their analyses and uncover the mechanism by which the virus modulates the risk of disease. A systematic understanding of the virus's role could ultimately inform whether using antiviral drugs or tweaking the body's immune response would be the best strategy to temper the disease.

"It would be great to have a tool that can be used to assess GvHD risk in these patients before they undergo a transplant," Chiu said, a step that Le Goff said could lead to new therapies. "We hope that in the next few years we will find a way to prevent virus-associated GvHD," said Le Goff.

Explore further: Researchers develop new strategy to limit side effects of stem cell transplants

More information: Jrme Legoff et al. The eukaryotic gut virome in hematopoietic stem cell transplantation: new clues in enteric graft-versus-host disease, Nature Medicine (2017). DOI: 10.1038/nm.4380

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Gut viruses tied to potentially deadly complication of bone marrow transplant - Medical Xpress

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

JoAnne Viviano The Columbus Dispatch @JoAnneViviano

Dennis Matko was headed for a knee replacement when he discovered a new therapy that would instead inject his own stem cells and plasma into the joint to help prevent degradation.

The 69-year-old Clintonville resident said he had been pretty active in his 50s, leading to problems with the right knee. He eventually had his meniscus removed. He had been through physical therapy, cortisone shots and gel injections, but the pain persisted.

The therapy, he said, was a no-brainer. He was sold because the procedure involved putting his own fluids into his body with no foreign objects and no drugs.

Dr. Joe Ruane, the orthopedic doctor who treated Matko, introduced the therapy at OhioHealth, but there are a number of places using the therapy around the state and country.

It's used to treat people with osteoarthritis, the type of arthritis caused by wear and tear.

Ruane said that the need for total knee replacements in the U.S. is expected to climb by 600 percent in the next 20 years, and there is concern that there might not be enough surgeons to perform the procedures.

We need an alternative, and patients are looking for alternatives, and given the choice between a knee replacement and an injection, many patients would choose an injection, he said.

The treatment involved removing Matkos bone marrow from the back of his pelvic bone, a process done in the office under general anesthesia. The marrow was then processed to form a concentrate of stem cells and other growth factors.

Matko also had blood drawn to create platelet-rich plasma, which acts as a signaling system to get the stem cells to respond.

Ruane injected both components into the knee, delivering more than 100 stimulating and growth factors to the joint.

Ruane said the process inhibits irritating chemicals that contribute to inflammation, decreases the activity of enzymes that break down cartilage, and helps the knee to make some of its own joint fluid again.

And, to a small degree, it does help regrow some of the tissue in the knee that has been destroyed by the arthritis, Ruane said.

The procedures are most effective in young patients with early arthritis, said Dr. Adolph Lombardi of Joint Implant Surgeons in New Albany, where stem-cell and platelet-rich plasma injections are offered as separate therapies. It won't help with bone-on-bone disease, he said.

While other injections might offer short-term pain relief, platelet-rich plasma has been shown to offer a full year of relief, said Lombardi, who works with the Mount Carmel Health System. The idea is that bone-marrow stem cells, when injected into a hip or knee, can differentiate into cartilage cells and help with regeneration.

"All of this is very new but it seems to be extremely promising," Lombardi said. "This is using their own bodies' healing potential to maintain cartilage and relieve pain."

Dr. Michael Baria performs the procedure at Wexner Medical Center at Ohio State University, where the bone-marrow and platelet-rich plasma injections also are offered as separate treatments. He agreed that the hope with the bone-marrow injections is that the stem cells turn into cartilage cells, improving or halting the osteoarthritis disease.

But in his experience, the treatment is helpful for patients with advanced disease.

"The most common patient we see for this is going to be in late-stage arthritis, so kind of at the end of their rope," Baria said. "Platelet-rich plasma is usually not as good for bone-on-bone arthritis. Bone marrow doesnt seem to be limited by bone on bone."

The body has trouble healing arthritis because cartilage doesnt get enough blood supply, Ruane said. Injecting the stem cells boosts the bodys own process.

While platelet-rich plasma has been shown to decrease inflammation, stem-cell use is newer and has yet to be proven effective, Baria noted.

OhioHealth andJoint Implant Surgeons are currently in the midst of controlled randomized trials, hoping to prove the effectiveness of the procedures and obtain approval from the U.S. Food and Drug Administration.

Unless that happens, the procedure will be considered experimental, and insurance doesnt cover costs. Matko paid $2,800 for the injections at OhioHealth.

Before the treatment, Matko was having trouble with mundane things like going up and down stairs and with other activities, such as taking hikes or walks with his wife or working out. A retired police officer, he now works as a business consultant and spends a lot of time on his feet, so he was looking for better mobility there as well.

Matko said the injections have helped his knee, which is getting progressively better over time. He said hes been able to increase his activity, getting back to the gym and taking hikes and walks. He has minimal pain climbing stairs and hes more comfortable in his work.

Im not saying its all better but its much better, Matko said. Its headed in the right direction.

He realizes the treatment is not a cure.

Im not looking for a miracle, he said. I just want to forestall problems as long as possible.

.

.

jviviano@dispatch.com

@JoAnneViviano

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Patients' plasma, stem cells help knee problems - The Columbus Dispatch

BrainStorm Cell Therapeutics of Petah Tikva is recruiting American patients for a Phase 3 clinical study of its NurOwn stem-cell treatment intended to halt progression of amyotrophic lateral sclerosis (ALS).

The announcement was made in a patient webinar last week.

The NurOwn platform grew out of a technique developed at Tel Aviv University for growing and enhancing stem cells harvested from patients own bone marrow. The enhanced cells, injected via lumbar puncture, secrete elevated levels of nerve-growth factors believed to protect existing motor neurons, promote motor neuron growth and reestablish nerve-muscle interaction.

A 24-week Phase 2 safety study was concluded in 2016 on 48 participants (36 treated, 12 placebo) with possible, probable and definite ALS. This study was done at the University of Massachusetts Medical School, Massachusetts General Hospital and the Mayo Clinic.

The Phase 3 double-blind, placebo-controlled study, to begin enrollment in August, will look at efficacy and safety of repeated doses. The California Institute for Regenerative Medicine has awarded Brainstorm a $16 million grant to support the pivotal trial.

This study will accept 200 randomized study participants between the ages of 18 and 60 (half getting the treatment and half a placebo) at the three previous centers as well as California Pacific Medical Center in San Francisco, UC-Irvine near Los Angeles and another site not announced.

Potential participants must live within about 100 miles of one of the centers for ease of follow-up. They will receive three doses over a 16-week treatment phase and then undergo 28 weeks of follow-up.

BrainStorm President and CEO Chaim Lebovits said he hopes to get approval by the end of the year for a hospital exemption program in Israel an accelerated regulatory pathway that would clear the way for a first batch of 50 patients to receive NurOwn at Tel Aviv Sourasky Medical Center. However, there will be no compassionate treatment using NurOwn in Israel or elsewhere.

The NurOwn platform technology also has potential applications in any neurodegenerative disease, such as multiple sclerosis and Parkinsons.

For more information, click here.

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ALS treatment to begin Phase 3 clinical trials in US - ISRAEL21c

The hypothalamus, a command center deep in the brain, helps control everything from hunger to sleep.

Roger Harris/Science Source

By Mitch LeslieJul. 26, 2017 , 1:00 PM

If these sweltering summer days prompt you to reach for a cold drink, you can thank your hypothalamus, a region of the brain that helps us regulate body temperature and other internal conditions. Butthe region may failus when we get older. A new study in mice suggests that the hypothalamus promotes aging, hastening physical and mental decline as its stem cells die off.

Its a pretty stunning paper, says Charles Mobbs, a neuroendocrinologist at the Icahn School of Medicine at Mount Sinaiin New York City. The new aging mechanism is totally novel and quite unexpected, adds neuroendocrinologist Marianna Sadagurski of Wayne State University in Detroit, Michigan.

Tucked away deep in the brain, the hypothalamus monitors and maintains our blood concentration, our body temperature, and other physiological variables. Researchers have also suspected that it plays a role in aging. The hypothalamus becomes inflamed as we get older, and 4 years ago a team led by neurodendocrinologist Dongsheng Cai of Albert Einstein College of Medicine in New York City showed that quelling this inflammation delays physical deterioration and boosts life span in mice.

In the new study, the team turned its attention to the hypothalamuss stem cells, which in young animals divide to produce replacements for dead and damaged cells. As mice get older, the scientists found, the number of stem cells in the hypothalamus plunges. By later ages they are basically all gone,Cai says.

To determine whether this loss promotes aging, researchers tried to speed up the process, genetically altering mice so that stem cells in the hypothalamus died when the animals were dosed with an antiviral drug. Knocking off some 70% of the cells shortened the mices lives by about 8%, the team reports today in Nature. The mices memory, coordination, and endurance also suffered. Behaviorally, they were like grumpy grandparents, less social and curious than youthful rodents. For example, when researchers put a new object into their cages, controlmice spent about twice as long exploring it than did their modified counterparts.

Next, the team tried to reverse this deterioration by injecting stem cells into the hypothalami of middle-aged animals. Mice that received the stem cells outlived mice injected with a different type of brain cell by more than 10%, and they retained more of their physical and mental capabilities. In humans, the extra boost could mean a few more years of healthy life, Mobbs notes.

Researchers assume the loss of stem cells causes organs and tissues to wear out gradually because they cant replenish their lost cells. But because injecting stem cells into the mice produced benefits quickly, Cai and his colleagues concluded a faster-acting mechanism was at work.

Their suspicions fell on RNA molecules known as microRNAs, which stem cells manufacture and release. These microRNAs ferry messages to other cells, altering which proteins they produce. The researchers found that stem cells from the hypothalamus pump out huge amounts of microRNAs, packaged in tiny containers called exosomes. They also found that injecting mice with microRNA-rich exosomes isolated from cultures of young hypothalamus stem cells slowed the animals physical and cognitive breakdown almost as much as injections of stem cells.

The big question is how those microRNAs influence function, Mobbs says. The molecules could spur other cells to curb inflammation or stress, Cai says, though he isnt certain how they work. Where the microRNAs exert their effects is also a mystery. Their targets may be other cells in the brain or the spinal cord, but they might also slip into the bloodstream and prod cells elsewhere in the body.

The work suggests that protecting or replacing the hypothalamuss stem cellsor replicating the effects of the microRNAscould slow aging in humans. It might also be possible to suppress the inflammation that provokes the stem cell die-off, Sadagurski says. She says some current drugs, including the diabetes treatment acarbose, curb inflammation in the hypothalamus and may be worth testing.

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The breakdown of this brain region may accelerate aging - Science Magazine

Some big scientific breakthroughs are set to take place this year.

And fortunes could be made with a few off-the-radar biotech pioneers.

What I am talking about are the handful of small biotech companies applying regenerative medicine platforms to degenerative diseases and ailments like paralysis.

These degenerative diseases have always been tough to treat, and few therapies are available. Thats not surprising, since weve lacked until recently the ability to create healthy and functioning human cells to replace ones lost in the body to injury and disease.

Now, though, a handful of breakthrough companies are aiming to correct that lack with new pluripotent stem cell technology.

Stem cells have the ability to reproduce and change into functional cells and tissues. And pluripotent stem cells are the most potent. They can turn into any cell type in the human body. Furthermore, they can divide and reproduce without end.

This makes them an ideal starting point for manufacturing cell-based therapies.

One company using this platform to help heal spinal cord injuries and paralysis is already heaving great success in FDA trials with a new cell line.

Their recent FDA Phase 1 trial data shows that patients treated with a new cell line are seeing a significant return of nerve function thanks to these grafts.

And last month, this same company announced nine-month follow-up data for patients that were given a 10 million cell dose to their injury. Even though this is only half of what researchers believe will be a full dose, at least 50% of patients have already shown signs of recovery.

This includes two levels of improvement in motor function, as well as improvements in arm, hand and finger function.

The nine-month data confirms what the company reported at three and six months, too.

Showing us that the new cell therapy has great durability. The introduced cells help heal the injury, and the result is more than temporary.

Under the accumulating evidence, the FDA has also decided to approve an expansion to this trial to include patients with spinal cord injury at the C4 location which is in the middle of the neck.

This is a very significant development.

The C4 spinal location is one of the most common locations for a spinal cord injury, and it often results in paralysis from the neck down.

And now the FDA is allowing the company to expand its treatment window from 1430 days after injury to 2142 days.

All these data bode very nicely for the future.

And not only for the developers of this specific cell therapy, but for other small biotech companies also trying to carve out market share in the pluripotent stem cell therapy space.

According to the National Spinal Cord Injury Statistical Center, there are some 17,000 new spinal cord injuries per year. This represents a large potential market for any company that masters this new technology and provides viable treatments for degenerative injuries.

Even more promising is that this new cell technology may also be used to treat cancer. And another group of tiny biotech companies are leading the way in research for this application, too.

We hope to see the data from these other FDA trials focusing on developing cancer vaccines during the second half of 2017.

If any of these new trial results comes back positive as early indications are showing, then were looking a tsunami sized wave of new opportunities in biotech. And for the handful of small companies and their investors, this should be a game changer.

As it stands currently, most of the companies making the push into regenerative medicine using new stem cell technology remain largely undiscovered.

As the FDA data become more actionable and these treatments move closer to market introduction, there will be some great plays to make.

And when that happens Ill be right here to make sure you have the best chance for the big score with this new technology.

To a bright future,

Ray Blancofor The Daily Reckoning

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Off-the-Radar Biotechs About to Break Out - Daily Reckoning

Malfunctioning glial cells that keep nerve cells from forming working communication networks may be the basis of the wiring problems in the brains of people with schizophrenia, new research suggests.

The inability of these cells to do their jobappears to be a primary contributor to the disease.

When researchers transplanted human brain cells generated from individuals diagnosed with childhood-onset schizophrenia into mice, the animals nerve cell networks did not mature properly and the mice exhibited the same antisocial and anxious behaviors seen in people with the disease.

The findings of this study argue that glial cell dysfunction may be the basis of childhood-onset schizophrenia, says neurologist Steve Goldman, co-director of the Center for Translational Neuromedicine at the University of Rochester Medical Center (URMC) and lead author of the study.

The inability of these cells to do their job, which is to help nerve cells build and maintain healthy and effective communication networks, appears to be a primary contributor to the disease.

Glia are an important family of support cells found in the brain and play a critical role in the development and maintenance of the brains complex interconnected network of neurons. Glia includes two major types: astrocytes and oligodendrocytes.

Astrocytes are the brains principal support cells, while oligodendrocytes are responsible for producing myelin, the fatty tissue that, like the insulation on electrical wires, wraps the axons that connect different nerve cells. The source of both these cells is another cell type called the glial progenitor cell (GPC).

Astrocytes perform several functions in the brain. During development, astrocytes colonize areas of the brain and establish domains in which these cells help direct and organize the network of connections between nerve cells.

Individual astrocytes also send out hundreds of long fibers that interact with synapsesthe junction where one neurons axon meets anothers dendrite. The astrocytes help facilitate the communication between neurons at the synapses by regulating the flow of glutamate and potassium, which enable neurons to fire when they are communicating with each other.

In the new study, the researchers obtained skin cells from individuals with childhood-onset schizophrenia and reprogrammed the cells to create induced pluripotent stem cells (iPSC) which, like embryonic stem cells, are capable of giving rise to any cell type found in the body. Next, the team manipulated the iPSCs to create human GPCs.

The human GPCs were then transplanted into the brains of neonatal mice. These cells out-competed the animals own native glia, resulting in mice with brains comprised of animal neurons and human GPCs, oligodendrocytes, and astrocytes.

The researchers observed that human glial cells derived from schizophrenic patients were highly dysfunctional. The development of oligodendrocytes was delayed and the cells did not create enough myelin-producing cells, meaning signal transmission between the neurons was impaired.

The development of astrocytes was similarly tardy so that the cells were not present when needed and were thus ineffective in guiding the formation of connections between neurons. The astrocytes also did not mature properly, resulting in misshapen cells that could not fully support the signaling functions of the neurons around them.

The astrocytes didnt fully mature and their fibers did not fill out their normal domains, meaning that while they provided control to some synapses, others had no coverage, says Martha Windrem, also with the Center for Translational Neuromedicine and first author of the study. As a result, the neural networks in the animals became desynchronized and uncoordinated.

The researchers also subjected the mice to a series of behavioral tests. They observed that the mice with human glial cells from individuals diagnosed with schizophrenia were more fearful, anxious, anti-social, and had a variety of cognitive deficits compared to mice transplanted with human glial cells obtained from healthy people.

The studys authors point out that the new research provides scientists with a foundation to explore new treatments for the disease. Because schizophrenia is a unique to humans, until now scientists have been limited in their ability to study the disease. The new animal model developed the by the researchers can be used to accelerate the process of testing drugs and other therapies in schizophrenia.

The study also identifies a number of glial gene expression flaws that appear to create chemical imbalances that disrupt communication between neurons. These abnormalities could represent targets for new therapies.

Additional coauthors of the study are from the University of Rochester, the University of Copenhagen, George Washington University, Johns Hopkins University, and Case Western University.

The study appears in the journal Cell. Funding from National Institute of Mental Health, the National Institute of Neurological Disorders and Stroke, the G. Harold and Leila Y. Mathers Charitable Foundation, the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation, and the Novo Nordisk and Lundbeck Foundations supported the research.

Source: University of Rochester

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Glial cells botch wiring in childhood schizophrenia - Futurity: Research News

Over the past twenty years, Jamie Sherrill has become one of the most in-demand skincare gurus in Hollywood. But you may not know her name--she goes by the moniker Nurse Jamie, which is also the name of her line of cult-favorite beauty tools and potions, as well as her Los Angeles spa, Nurse Jamie Beauty Park. Before you ask--yes, Sherrill is, in fact, a nurse, but she's also a certified aesthetician, which means she can offer her devoted clients, who range from Jessica Alba to Ruby Rose, a wide-range of services that promise flawless skincare through some very unique methods that can be done both at home and in the office. "At Nurse Jamie Beauty Park, our vision is simple to offer not only the best non-surgical beauty solutions available on the market, but also a customized combination of the most cutting-edge technical advances in anti-aging, skincare and beauty today," Sherrill explains. "High-tech tools, devices and home-based care are a big part of my regiment and I make everyone participate."

Here, Sherrill offers insight into the most in-demand celebrity beauty desires, and offers tips on improving your complexion at home.

You have a wide range of high profile clients, all unique with their own concerns and skincare regimens. What are the most common concerns you hear?Celebrities come in all shapes, sizes and ages, so everyone is going to have a different treatment plan. This year body sculpting is big from banning the bra strap fat to firming the tush, while laser hair removal, Botox, fillers and glowing skin are year round trends. Those requests never go out of style.

What types of treatments are most requested before a red carpet appearance?Some are genetically blessed and don't really do more than an oxygen facial and an electric facial"to be fully red carpet ready. But that said--we start to lose collagen production and skin elasticity starting at 25, so we will typically use a range of key technologies in lasers for skin texture and complexion.TheACELLeratorat home beauty tool is idealto help serums and product be absorbed for a lifting and tightening effect, and has a great anti-inflammatory property. You can use every day but specifically just before an event for a more open eye look or more defined cheek even if you just flew in!This works well for the face and body, so it helps with stretch marks and skin smoothing for waistline, hips and thighs. Trust me this is acelebsecret. If you don't believe me, do one side of your face for just one minute then look into a mirror.

But don't forget red carpet prep needs to happen every day, too. Eat well, sleep well on the right pillow, take off make-up at night and use good quality products with the best raw ingredients. Home care matters as much as in office does.

When your clients are on location for months at a time, what tips do you give them?Think maintain, not reclaim and always try to be preventative.Think of the rules of eating that are good for your body; most apply to your skin as well. It is the largest organ of the body so treat it like one.Be consistent with taking off makeup nightly and never with a washcloth. Use a hypoallergenic and antibacterial surface to cleanse your skin. Exfoliate regularly, manually or with a tool, but gently and consistently.

Invest in a beautytool to help increase absorption of products like my Instant Uplift or ACELLerator Ultra. Just like the machines we have in office, they increase absorption and efficacy of your products while helping to improve and maintain tone. Also, wear sunscreen.It seems basic, but all helps. At-home devices are the future of beauty -- you can have the best raw ingredients in the world, but as skin is the largest organ of the body its main function is to protect. The number one cause of aging is UV damage, the number two is smoking, and the third is sleeping on a traditional pillow.Use satin only and a shape that will help you train to sleep on your back, so that the most delicate areas around the eyes, cheeks and neck do not form permanent wrinkles.

It's the middle of summer. Other than sunscreen and hats, what other advice do you have for fending off skin discoloration?Use good quality products with the best raw ingredients. Old school skincare was to use aggressive products that caused chemical cell turnover reaction, which can make you more susceptible to sun damage. (Retin-A is so 1980s!) My opinion is to use retinol ingredients sparingly. Epidermal Growth Factor (EGF) - causes cell turnover and has significant effects on delaying the aging process - including preserving skins cells and skins overall vitality and radiance, without leaving you red, flaky, and shiny. I hate the shiny face -it kills meovertime I see I can spot theglare from across the room.The Nurse Jamie tools that you incorporate into your treatments seem to have a loyal following of their own. Like the Beauty Stamp, for example. How does that work?The Beauty Stamp may very well be the best investment anyone can make. A small pad features a cross section of micro needles in a grid that helps with micro exfoliation, opens channels for product delivery and efficacy and aids in the body process of collagen andelastinproduction. It is my triple threat. For day of events you need to focus on complexion and texture in a non-invasive way or only protocols with no downtime and no risk. Don't try something new with a high risk to low reward for the day of an event. Nothing worse than redness or inflammation when you are dressed to impress and need your face to match! How about the Accelerator Ultra?TheACELLeratorat home beauty tool is ideal for a daily regimentto help serums and product be absorb lifting and tightening effect and has a great anti-inflammatory property.You can use every day but specifically just before an event for a more open eye look or more defined cheek--even if you just flew in! This works well for face and body so it helps with stretch marks and skin smoothing for waistline, hips and thighs, too. Trust me, this is acelebsecret dont believe me? Do one side of your face for just one minute then look into a mirror.What is your top selling tool?UpLift Massaging Beauty Roller. It has a huge celebrity following.Are there any foods or vitamins that you recommend for vibrant skin?A B12 Energy Shot. Close to a decade ago I injected Paris Hilton and Nicole Richie with it right in their bums on national television forThe Simple Life,and in turn injectable vitamins became one of our most popular treatments...

What are the biggest skincare mistakes people make?Side sleeping and over exfoliating. We need to treat our skin like a silk fabric not a piece of leather. When youoverexfoliate(physically and chemically) and withtoo much frequency it destroys the protective barrier that your skin has - once it is removed or compromised you are you exposing your skin to environmental toxins, sun damage pre-mature aging, acne, etc. It's very common.

What is your personal daily skin routine?Taking off my make-up--I can't go to bed with my make-up on. Period. The UpLift Facial Massaging Beauty Roller, EGF Stem Cell Complex--I dont go anywhere without this cream. I would bathe in it if I could--and I use my ACELLerator for 10 minutes each night on both sides of my face while I sit in bed.I practice what I preach.That way I can give them my best face - and tell them it is what I do and mean it! Ive dedicated my life to skin and created my line for products that I felt that were missing in the marketplace. As a busy working mom of three toddlers Im proud to say that Im my own client.

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10 Celebrities Who've Quit Botox and Fillers Throughout the Years

Watch: History of the Best of Celebrity Fashion in the Hamptons

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Celebrity Skincare Guru Nurse Jamie on Why At-Home Beauty Tools Are the Future - W Magazine

Advances in medical imaging enable bespoke tissues and organs to be developed for transplant or engraftment with remarkable resolution and definition using 3D bioprinting. The incorporation of stem cell therapies into these 3D tissue constructs is incredibly promising for the delivery of pioneering stem cell regenerative therapies. Typically, 3D bioprinting requires use of a biomaterial to aid with deposition, which can cause negative host responses. To avoid such problems, US researchers have developed a biomaterial-free cardiac patch (Scientific Reports 7 4566).

Heart disease affects thousands of people every year and effective repair of cardiac tissue would reduce a large medical health care burden. Researchers from the Narutoshi Hibino lab at Johns Hopkins Hospital and Johns Hopkins University have devised a 3D-bioprinting procedure that allows for the biofabrication of cardiac tissue patches to deliver regenerative stem cells, without using biomaterials. The process utilises aggregated balls of cardiac cells (cardiospheroids), which are directly printed into a cardiac patch construct. The cardiospheroids are identified, picked up by a vacuum and bioprinted directly onto a needle microarray (a video of the 3D-bioprinting process used is available from JOVE). This novel method allows the patch to be constructed with cells alone and will avoid detrimental effects induced by biomaterial grafts.

Stem cell techniques for tissue regeneration typically rely on biomaterial scaffolds to provide structure and support for cells during grafting. The grafting or introduction of biomaterials to a patient induces an immune response, or can create scar tissue from the graft, potentially damaging the region of tissue intended to be repaired. Through developing a biomaterial-free graft, it is possible to avoid these detrimental factors. And by using a patient's own stem cells it is possible to create native tissue that is fully biocompatible.

3D bioprinting was crucial to the development of effective cardiac patches, with specific spatial distribution being crucial to mechanical integrity. Cardiospheres without specific placement to overlap with other cardiospheres disintegrated after removal from the needle array; although partially disintegrated regions were able to fuse back together eventually. This effect removed the structural definition of the patch, negating the advantages of using bioprinting for developing a cardiac patch of specified dimensions.

The researchers grafted patches onto rat hearts and after a week saw signs of blood vessel formation, with viable cells and red blood cells present in the cardiac patch. Tissue protein stains showed that collagen was present in the patch, indicating the deposition of a native extracellular matrix from the cells, crucial to cell integration. Further staining showed the presence of human nucleic acid in rat tissue, implying that the human cell derived patch had successfully grafted with the rat tissue.

This biomaterial-free cardiac patch was developed using pluripotent cardiomyocyte stem cells, cardiac fibroblasts and human umbilical vein endothelial cells (HUVECs), which were aggregated into cardiospheroids for bioprinting. Cardiospheroids were able to develop a functional phenotype after 48 hours, with spontaneous beating and electrical conductivity a week after bioprinting. Cardiomyocytes alone were not able to reproduce this functional phenotype.

This process demonstrates a novel approach to eliminating biomaterial-induced damage. Further development of this 3D bioprinting technique in conjunction with stem cell therapies could progress biomaterial-free cardiac patches into the popular domain.

3D printers help build a better cranial nerve4D bioprinting: adding dynamic actuationThe first laser-printed 3D cellular tubes3D-printed polymer stents evolve

Geoffrey Potjewyd is a PhD Student contributor to medicalphysicsweb, working in the Division of Neuroscience and Experimental Psychology, as part of the CDT in Regenerative Medicine at The University of Manchester. He is studying the neurovascular unit in relation to vascular dementia and Alzheimer's disease, using biofabrication, biomaterials and stem cell based techniques.

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3D bioprinted cardiac patches are biomaterial free - Medical Physics Web (subscription)

Cellular Dynamics International, the stem cell company founded by UW-Madison stem cell pioneer James Thomson, is backing off on moving its headquarters to a big, new building in Verona and will stay in Madison, at least for now, as it prepares to push forward with its first potential stem cell-based treatment in early 2018.

CDI president Kaz Hirao said Thursday the company is shelving plans to shift operations to a $40 million, 133,700-square-foot building that was to be built for CDI on Kettle Moraine Trail in Verona. The building was expected to house 280 employees, with so-called clean rooms, quality-control labs, processing rooms and offices.

Instead, CDIs main offices and labs will remain at 525 Science Drive in University Research Park and the company will remodel an existing building whose site has not yet been determined to house several clean rooms that will meet government standards for manufacturing stem cells for use in clinical drug trials.

Fujifilm (CDIs parent company) has a very strong commitment and wants to see (the) Madison (site) grow in the future. Strategy-wise, that has not changed, Hirao said. Madison has a great ecosystem for our businesses.

He said the National Eye Institute plans to submit an application to the U.S. Food and Drug Administration in January 2018 for a retinal cell therapy it has been developing with CDI for age-related macular degeneration, an eye disease that can lead to blindness. The National Eye Institute has conducted animal studies on the drug, Hirao said.

It is the first of a series of stem cell-based drugs the company is working on. CDI expects to file investigational new drug applications for treating Parkinsons disease and for cardiac disease in 2019, he said.

In order to make stem cells that meet government standards for use in human clinical trials, Hirao said the company will establish clean rooms that meet regulations for current good manufacturing practices. He said he expects to designate a location in the next month or two, within about a 15-minute drive of CDI headquarters, to handle the companys stem cell manufacturing needs for the immediate future.

Next year, CDI will review its plans again, Hirao said, and will again consider a move to a larger, consolidated building. If it decides to go ahead with that, Verona would be one of the preferred options, he said.

CDI had obtained up to $6 million in financial incentives from the city of Verona for the building that was to be built and owned by developer John K. Livesey.

Verona planning and development director Adam Sayre called CDIs decision to pull back on the plans unfortunate, but said city officials will keep in contact with Cellular Dynamics over the coming months.

The city would continue to welcome them with open arms, Sayre said. Well see what the next year brings.

At University Research Park, CDI occupies about 55,000 square feet, director Aaron Olver said. Weve recently provided CDI with some additional space to help them grow, he said.

CDI is one of the true gems among companies powered by UW-Madison research, and we would certainly do anything we could to help them find clean room space to continue their work, Olver said.

Founded in 2004, CDI was acquired by Fujifilm Holdings Corp. for $307 million in April 2015.

The company has 165 employees, including about 125 in Madison. Hirao said he expects to add employees, but said its too soon to estimate how many, or how quickly the company will grow.

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CDI ditches move to Verona - Madison.com

NEW YORK, July 12, 2017 /PRNewswire/ -- The global stem cell market is expected to reach USD 15.63billion by 2025, growing at a CAGR of 9.2%, according to a new report by Grand View Research, Inc.

Augmentation in research studies that aim at broadening the utility scope of associated products is anticipated to drive the market growth. These research projects have opened the possibility of implementation of several clinical applications of these cells, thereby impacting disease-modifying treatments.

Read the full report: http://www.reportlinker.com/p04998556/Stem-Cells-Market-Analysis-By-Product-Adult-Stem-Cells-hESC-Induced-Pluripotent-Stem-Cells-By-Application-Regenerative-Medicine-Drug-Discovery-By-Technology-By-Therapy-And-Segment-Forecasts.html

Scientists are engaged in discovering novel methods to create human stem cells. This is to address the increasing demand for stem cell production for potential investigation in disease management. This factor is certainly expected to accelerate the development of regenerative medicine, thus driving industrial growth.

Moreover, cellular therapies are recognized as the next major advancements in transforming healthcare. Firms are expanding their cellular therapy portfolio, understanding the future potential of this arena in the treatment of Parkinson's disease, type 1 diabetes, spinal cord injury, Alzheimer's disease, and others.

In March 2016, Scientists at Michigan State University unveiled new kind of cells "induced XEN cells" from a cellular trash pile. This discovery is expected to drive advancements in regenerative medicine. Such discoveries are anticipated to bolster research and sales in this market over the forecast period.

Further key findings from the report suggest: Adult stem cells dominated the market and is expected to maintain its dominance. This can be attributed to the several factors such as lower rejection rates, long term renewal property, and no ethical concerns associated with their usage Application wise regenerative medicine is estimated to hold the substantial share of the revenue Presence of significant number pipeline projects for regenerative medicine is expected to fuel growth in the market In addition, exploding research projects have driven the need of harvesting techniques, thereby propelling progress of acquisition technology Increased R&D activities and huge funds granted by funding bodies to advance cellular research in the U.S. have resulted into the large share of North America Extensive research carried out in Singapore and Japan is anticipated to drive progress with lucrative avenues Advanced Cell Technology Inc, Osiris Therapeutics Inc, Celgene Corporation, BIOTIME, INC., Cynata, and STEMCELL Technologies Inc., are some of the major companies operating in this market A number of companies are engaged in seeking investment from overseas organizations and also developing partnerships with the pharmaceutical organizations

Read the full report: http://www.reportlinker.com/p04998556/Stem-Cells-Market-Analysis-By-Product-Adult-Stem-Cells-hESC-Induced-Pluripotent-Stem-Cells-By-Application-Regenerative-Medicine-Drug-Discovery-By-Technology-By-Therapy-And-Segment-Forecasts.html

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According to recent study, advancements in materials from this study could potentially help patients requiring stem cell therapies for spinal cord injuries, stroke, Parkinsons disease, Alzheimers disease, arthritic joints or any other condition requiring tissue regeneration. Earlier research revolved around the role of autoimmunity in terms of a treatment.

Its important in the context of cell therapies for people to cure these diseases or regenerate tissues that are no longer functional, shared Samuel I. Stupp, director of Northwesterns Simpson Querrey Institute for BioNanotechnology and Board of Trustees Professor of Materials Science and Engineering, Chemistry, Medicine and Biomedical Engineering.

Cells in our bodies are constantly being signalled with many types of instructions coming from proteins and other molecules present in the matrices that surround them. For example, these can be cues for cells to express specific genes so they can proliferate or differentiate into several types of cells leading to growth or regeneration of tissues. One of the marvels of this signalling machinery is the built-in capacity in living organisms to make signals stop and restart as needed, or to switch off one signal and activate a different one to orchestrate very complex processes.

Building artificial materials with this type of dynamic capacity for regenerative therapies has been virtually impossible so far. The new work published today reports the development of the first synthetic material that has the capability to trigger reversibly this type of dynamic signalling. The platform could not only lead to materials that manage stem cells for more effective regenerative therapies, but will also allow scientists to explore and discover in the laboratory new ways to control the fate of cells and their functions.

One of the findings is the possibility of using the synthetic material to signal neural stem cells to proliferate, then at a specific time selected by the operator, trigger their differentiation into neurons and then return the stem cells back to a proliferative state on demand. The paper also reports that spinal cord neural stem cells, initially grouped into structures known as neurospheres, can be driven to spread out and differentiate using a signal.

But when this signal is switched off, the cells spontaneously regroup themselves into colonies. This uncovers strong interactions among these cells that could be important in understanding developmental and regenerative cues. The potential use of the new technology to manipulate cells could help cure a patient with Parkinsons disease. The patients own skin cells could be converted to stem cells using existing techniques.

The new technology could help expand the newly converted stem cells in vitro in the lab and then drive their differentiation into dopamine-producing neurons before transplantation back to the patient. In the new technology, materials are chemically decorated with different strands of DNA, each designed to display a different signal to cells.

People would love to have cell therapies that utilize stem cells derived from their own bodies to regenerate tissue. In principle, this will eventually be possible, but one needs procedures that are effective at expanding and differentiating cells in order to do so. Our technology does that, noted Stupp. While this process is currently only done in vitro with the vision of then transplanting cells, Stupp said in the future it might be possible to perform this process in vivo.

The stem cells would be implanted in the clinic, encapsulated in the type of material described in the new work, via an injection and targeted to a particular spot. Then the soluble molecules would be given to the patient to manipulate proliferation and differentiation of transplanted cells. The study was published in journal Nature Communications.

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Cell therapy may be key to treating Alzheimer's & Parkinson's - Economic Times

Regenerative medicine is a revolutionary approach to treating many degenerative conditions and includes a variety of different techniques including stem cell therapy. This field joins nearly all disciplines of science and holds the realistic promise of repairing damaged tissue by harnessing the bodys ability to heal itself.

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. 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 a mixture of cells including adipose-derived stem cells or ADSCs and growth factors and has been depleted of the adipocyte (fat cell) population.

ADSCs are multi-potential and can differentiate into a variety of different types of tissue including but not limited to bone, cartilage, muscle, ligament, tendon and fat. These cells have also been shown to express a variety of different growth factors and signaling molecules (cytokines), which recruit other stem cells to facilitate repair and healing of the affected tissue. ADSCs are very angiogenic in nature and can promote the growth of new blood vessels.

Based on research performed in our FDA registered facilities, stem cell quality and functionality can vary greatly depending on the methods utilized to obtain the cells. It is important to utilize a product that has undergone full characterization to include safety, identity, purity and potency. We have developed a method for harvesting and isolating stem cells from fat for therapeutic use. The use of a cell population that retains the ability to function in vivo will lead to more consistent patient results with long term success.

Adipose stem cells can be obtained from the patient easily, abundantly, and with minimal patient discomfort. Clinical applications for patients can be performed in an office setting safely, legally, and ethically using autologous ADSCs. Current applications include orthopedic conditions (tendon/ligament injuries, osteoarthritis, etc.), degenerative conditions (COPD, diabetes), neurological (MS, Parkinsons, spinal cord injuries, TBI, etc.) and auto-immune (RA, Crohns, colitis, lupus).

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 our South Miami clinic, visit http://www.stemcellcoe.com.

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Regenerative Medicine: The Future of Medicine is Here Miami's ... - Miami's Community Newspapers

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."

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High-tech solutions top the list in the fight against eye disease - Engadget

When the Japanese researcher Shinya Yamanaka managed to reprogram adult cells into an embryonic-like state to yield induced pluripotent stem cells (iPSCs), this was supposed to herald a revolution in regenerative medicine. But 10 years after their discovery, a therapeutic breakthrough is still outstanding.

The overall stem cell therapy field has failed today to show a very clear cut clinical benefit, told me Georges Rawadi, VP for Business Development at Celyad. The field now needs some significant success to attract attention.

Even though investors prefer placing their bets on the hot T cell therapies these days, some stem cell technologies such as iPSCs are starting to get traction as big industry players are exploring the territory. Last year, Bayer and Versant threw $225M into the pot to launch BlueRock Therapeutics, a regenerative medicine company that plans to develop iPSC-based therapies. A year before, Fujifilm spent $307M to acquire the iPSC company Cellular Dynamics.

Although a big success story is still lagging behind, recent advances in the field argue that stem cells indeed have the potential to translate into effective therapies for currently intractable diseases. Heres an overview of what biotechs stem cells are up to!

Stem cell treatment is not a new concept hematopoietic stem cells (HSCs) were described as early as the 1960s and bone marrow transplants have been used to treat blood cancer for decades.

The reason that we get excited about stem cell therapies comes from our experience with the hematopoietic stem cells. If you want to see what a mature stem cell therapy is like, you only need to look at bone marrow transplantation explained James Peyer, Managing Partner at Apollo Ventures, who has a Ph.D. in stem cell biology.

According to Peyer, the hematopoietic stem cell field is one of the most active areas in the stem cell world right now, mainly fueled by our advances in the gene editing space. Tools like CRISPR and TALEN allow for the genetic modification of a patients own bone marrow stem cells, which can then be expanded and returned to the patient for the correction of a genetic defect.

Last year, regulators gave green light to one of the first therapies of this kind. Strimvelis, developed by GSK, consists of an ex vivo stem cell gene therapy to treat patients with the very rare type of Severe Combined Immunodeficiency (SCID). Using the patients own cells avoids the risk of graft versus host disease (GvHD), which still affects around 30% of people receiving a bone marrow transplant.

Small wonder that the CRISPR companies, CRISPR Therapeutics, Editas, and Intellia are all active in this field, with preclinical programs in a number hematological diseases.

To date, the most prominent stem cells in the clinic are mesenchymal stem cells (MSCs), which are moving through more than 300 registered clinical trials for a wide array of diseases. These cells are able to form a variety of tissues including bone, cartilage, muscle or fat, and can be readily harvested from patients or donors for use in autologous or allogeneic therapies.

While MSCs have deluded the biotech scene with good safety profiles in clinical trials, their actual regenerative potential remains controversial, and there have been a great number of clinical failures, which many blame on a lack of demonstrated mechanisms of action.

As Peyer explained, The problem here is that, as opposed to other adult stem cells, the MSC has been unclearly defined. We know roughly what it does but we dont fully understand the molecular mechanisms driving these cells. On top of being unclearly defined, the regenerative powers of MSCs have been massively over-claimed in the past.

Another reason for the lack of clinical benefit has also been attributed to the use of undifferentiated MSCs, as Rawadi explained to me. The Belgian biotech Celyad, which has been pioneering cell therapy in the cardiovascular space, is using bone-marrow derived autologous MSCs and differentiates them into cardiomyocyte precursors to produce new heart muscle in patients with heart failure.

Although the company missed its primary endpoint in a phase III trial last year, Celyad has staked out a patient subpopulation that showed significant improvement. Its technology still has the confidence of the FDA, which just handed out a Fast Track designation and Celyad is now planning a refined Phase III trial.

One of Celyads major competitors, Australian Mesoblast, is forging ahead using allogeneic MSCs with Phase III programs in heart failure, chronic low back pain (CLBP) due to disc degeneration, as well as a range of inflammatory conditions including GvHD and rheumatoid arthritis.

Although the ability of MSCs to regenerate tissues remains questionable, the Mesoblasts approach hinges on a body of evidence showing that MSCs can suppress inflammation and mobilize endogenous repair mechanisms through indirect effects on immune cells.

Indeed, the first-ever approved stem cell therapy, Prochymal, also depends on this mechanism. Prochymal was developed by US-based Osiris Therapeutics and in 2012 received Canadian approval to treat acute GvHD. But after Sanofi opted to shelve its partnership with Osiris prior to FDA approval, the biotech sold out its off-the-shelf stem cell platform to Mesoblast in a $100M deal.

In Belgium, companies like TiGenix and Promethera are also banking on the immunomodulatory properties of MSCs. The companies are developing treatments for patients with Crohns disease and liver diseases, respectively.

The ultimate hope for stem cell therapies has been to regenerate damaged or diseased tissues as found in diabetes, heart failure or blindness. Holostem Terapie Avanzate, a spin-off from the University of Modena and Reggio Emilia was the first company to move towards this goal.

Building on 20 long years of research, the biotech has developed Holoclar, the first and only autologous stem cell therapy (apart from bone marrow transplants) to enter the European market. Holoclar is based on limbal stem cells, located in a part of the eye called the limbus, which can be used to restore eyesight in patients that have lost sight due to burn injuries.

Meanwhile, UK-based Reneuron is developing off-the-shelf therapies that aim to restore the cognitive function of patients following a stroke. Backed by no other than Neil Woodford, the company recently raised an impressive 100M to advance its lead therapy to the market.

The biotechs fetal-derived neural stem cell line CTX was able to significantly reduce the disability of post-stroke patients in a Phase II trial and ReNeuron is now planning to push its candidate into pivotal trials.

A major question in the space a decade ago was safety. Today, theres been a lot of trials done that show that safety is not an issue. I think safety is kind of off the table but efficacy is still a question mark. And thats what were trying to deliver now, Olav Helleb, CEO of ReNeuron, told me.

While neural stem cells and other tissue-specific stem cells are able to regenerate the cells of a particular tissue, Embryonic Stem Cells (ESCs) and their engineered counterparts, iPSCs, are capable of making every cell type in the body, a property known as pluripotency. Pluripotent stem cells can also expand indefinitely in culture and their identification unlocked massive expectations for these cells to transform the regenerative medicine field.

Yet, these cells come with significant challenges associated with the safety of the final preparation. Apart from ethical issues surrounding ESCs, today, a lot of companies have been cautious about using these cells for therapy, because undifferentiated pluripotent cells can drive tumor formation, explained Rawadi. Since ESCs can, in principle, form every cell type, they can lead to the formation of teratomas.

A major reason for the fairly slow progress in the field is based on the difficulties of directing a pluripotent cell to exactly the cell type that is needed for cell therapy. We can readily drive the cells from the undifferentiated state to the differentiated state. However, getting those cells to pause anywhere in the middle of this continuum to yield progenitor cells is incredibly challenging, Peyer explained. Another challenge, he says, is to engraft the cells in the right place to enable them to become fully integrated.

Besides initial hurdles, companies like US-based Asterias or ViaCyte are now running the first Phase I/II trials with ESC-derived cells to treat patients with spinal cord injuries and to restore the beta cells in type I diabetes. So far, the eye has been the the dominant organ for many of the first human clinical trials with pluripotent stem cells, where the cells are assessed in diseases such as age-related macular degeneration (AMD) to restore the loss of the retinal epithelium.

Deriving retinal epithelium from pluripotent cells is relatively easy and in fact, researchers in Japan are now running the very first clinical trial using donor-derived iPSCs to treat patients with AMD. For reasons of safety and standardization, the trial is based on an allogeneic approach. However, since this doesnt offer an exact genetic match, allogeneic therapies raise the prospect of immune rejection, an issue that has been plaguing the use of ESCs.

But the scientists in Japan have contended that iPSC banks could potentially solve this problem. The team in Japan is currently establishing an iPSC bank, consisting of HLA-characterized cell lines from 5-10 different donors, which should match 3050% of Japans population.

Such haplobanks have the benefits of allogeneic cell therapy, namely cost-effectiveness and standardization, but you still have matching immune systems, Peyer agrees.

For now, this remains a vision for the future, but the potential seems enormous. As Julian Howell, CMO of ReNeuron, told me, iPSCs have still got an awful long way to go. For the iPSC program running in Japan, they recently acknowledged that it took about $1.5M and 6 months to treat each patient. Its a great idea but its still got some way to go before it reaches the scale that could get into the clinic.

Images via nobeastsofierce,Natali_ Mis,vchal/ Shutterstock

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Regenerating the Body With Stem Cells Hype or Hope? - Labiotech.eu (blog)

SINGAPORE The use of stem cell treatment to repair liver cirrhosis, or hardening of the liver, will be tested in a clinical trial here involving 46 patients and costing S$2.6 million.

The four-year study, which was launched yesterday, came amid a growing waiting list in Singapore for a liver transplant, which is currently the only cure for patients with end-stage liver cirrhosis.

Conducted by a multi-centre team from several restructured hospitals here, the study is led by the National University Hospital (NUH).

Liver failure is one of the top 20 causes of death in Singapore, but many patients are not suitable for a transplant due to factors such as age and surgical fitness.

Out of every five patients doctors see with end-stage liver disease, only one qualifies for a liver transplant, said Dr Dan Yock Young, principal investigator of the clinical trial and senior consultant at NUHs division of gastroenterology and hepatology.

(A liver transplant) is curative, but it is a complex procedure, and many patients are not suitable for it. For these patients, treatment is limited, but morbidity and mortality rates are high as high as 50 per cent in one year and this is probably worse than many (of the) other terminal illnesses we talk about today, he said.

Animal studies conducted over the last five years have shown that stem cells can reconstruct the micro-environment of a normal liver.

Like how branches are of critical importance in supporting the leaves and fruits of a tree, the endothelial (stem) cells contribute to supporting a nutritious environment for the hepatocyte (liver) cells, Dr Dan explained.

While similar stem-cell studies have been conducted in other centres in Asia, there has been no definitive evidence of the benefits of the treatment for liver patients.

The study will recruit 46 patients aged between 40 and 70 years old, and who are at the terminal stages of chronic liver disease, over three years. It is funded by the National Medical Research Council.

During the clinical trial, patients will be divided into a therapeutic group and a control group.

All patients will receive an injection to stimulate their bone marrow cells as part of the supportive treatment for their liver cirrhosis. However, only patients in the study group will have the stem cells from the bone marrow extracted and deposited directly into their liver for more targeted repair.

Using ones own stem cells will avoid the problem of cell rejection.

The liver tissue will be examined three months later, and an investigation to compare pre- and post-transplant results will be conducted after a year.

Since invasive surgery is not required for stem-cell therapy, the fatality risk is significantly lowered for the patient. However, other risks such as severe bleeding and infections still remain, given the patients weakened condition.

NUH also noted that the stem-cell therapy does not replace liver transplants, and the latter remains the best available treatment for liver cirrhosis.

It is very painful to turn patients away when we cannot offer them a liver transplant, said Dr Dan, adding that this stem cell therapy will serve as an alternative option.

We hope that this is a stepping stone to trials for stem cell candidates, he added.

MORE WAITING FOR A LIVER

The number of people on the waiting list for a liver transplant has been growing in recent years. In June last year, it was reported that there were 54 people on the list, more than double the 24 patients in 2011.

Chronic Hepatitis B remains the primary cause of non-alcoholic fatty liver disease, which refers to a range of liver conditions affecting people who drink little to no alcohol. However, obesity has become a contributing factor to the illness as well.

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New NUH study to test stem cells as treatment for liver disease - TODAYonline

Yaser and Vicki Martini, who launched a worldwide campaign to find a bone marrow donor for their daughter Margot, today joined the huge public outpouring after the Nigerian international's blood cancer diagnosis was revealed.

Margot died aged just two after an unsuccessful bone marrow transplant but her story prompted thousands of people to join the stem cell register.

But it also exposed the difficulty in finding donor matches for people from ethnic minorities or mixed heritage.

Bone marrow stem cell transplants are one of the major treatment for leukaemia, as well as chemotherapy, radiation, biological and targeted therapy.

Mr Martini, whose wife Vicki hails from Essington, said: "We are shocked to hear about Carl's diagnosis and our hearts go out to him and his family. Its ironic, because everyone at Wolves have been so supportive of Team Margot and we want to send our best wishes and our support, as they fight his cancer together."

They set up the Team Margot Foundation with a string of friends, family and supporters which is aimed at getting more people to join the stem cell register particularly those from ethnic minorities and mixed backgrounds.

Mr Martini added: As yet, its unclear whether Carl will need to have a bone marrow transplant, but its a certainty that he will need blood and platelet donations to stay clinically well. Anyone reading this can help in two ways by signing up to become a blood donor and also registering as a potential bone marrow donor.

Regarding bone marrow donation, we know first hand from Margots experience that people with a mixed heritage and those from the black, Asian and minority ethnic communities have only a 21 per cent chance of finding a donor with a matching tissue type. For this reason, we urgently need more people from these communities to join the bone marrow registers.

"In that sense, we each have a unique contribution that we can make and Im not exaggerating when I say that you could be the only one who can save a life.

Wolves players, including club legend Steve Bull, have backed Team Margot's campaign.

Margot's uncle Durand Bailey, who lives in Tettenhall and runs Diffusion designer clothing store in Lichfield Street, knows Ikeme. He said: Carl is such a lovely man, a real gentleman. He has done so much to help others. We all wish him and his family well."

Wolves announced the 31-year-old had returned 'abnormal blood tests' as he returned for pre-season and further checks confirmed the diagnosis.

He is to start a lengthy treatment programme.

To sign up as a blood donor visit: blood.co.uk. To find out how you can become a potential stem cell donor, go to teammargot.com

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Carl Ikeme: Family of toddler Margot Martini support Wolves star after leukaemia diagnosis - expressandstar.com

Fear becomes a constant companion when ones child is desperately ill.

Kim Lahti-Scranton knows this all too well as her daughter Jane is fighting for her life at BC Childrens Hospital.

As much as Janes journey is all-consuming, Kim is helping a desperate woman to get young people to donate bone marrow in order to find a better match for her son, Noah, who is in critical need of a bone marrow transplant soon.

The Scranton family is living in Vancouvers Ronald McDonald House and shares a kitchen with 17-year-old Noah Stoltes family.

Noah survived a first round of cancer when he was 10. In April, the family was given the terrifying news that he had relapsed.

His mom thought they were going to find a match within family; theyre 100 per cent Dutch, says Kim, noting family members are usually the best choice, but his three sisters are only a 50 per cent match.

Out of the worldwide pool of donors, there are two people who are a 75 per cent genetic match and are being considered, one of whom lives in Europe. The lower the match, the more chance there is for complications and a less than optimal outcome.

There is a time crunch as Noah needs to have a transplant sometime in September and it can take a couple of months from registration to donation.

One of Kims nephews is an NHL player, who used his connections to help Noahs aunt interest 85 people in becoming bone marrow donors in Edmonton.

Unable to get help from Canadian Blood Services to organize a similar drive in Vancouver because of staffing issues, Kim and Noahs mother, Stacey VanderLee Stolte, went to a Vancouver Canadians game to raise awareness and hopefully encourage people to sign up to be on the registry for stem cell or bone marrow donation.

We were met with a whole range of responses from people who were incredibly receptive, to people who completely ignored us and everything in between, says Kim. We got some interesting comments like no kid would want my bone marrow, Ive done too many drugs, to other people saying no because they thought if they were a match that donating bone marrow would be too painful.

Dr. Lucy Turnham, a clinical associate who oversees the outpatient oncology clinic at BC Childrens Hospital, has performed Janes procedures and says males between the ages of 17 and 35 are the best resource.

Its one of the most wonderful charitable donations you can do, Turnham says, noting many people never receive a call. It has a huge impact on a patient who has no other options.

Turnham explains that donors are educated before they do a swab. If they are told they are a match, they can still say no at any time. They then undergo further testing and maybe counselling at that point, and can still back out an any time.

If they had said yes and the patient begins treatment to get rid of the diseased cells prior to transplant, and then the donor backs out, the patients life is at serious risk, so it is important to know what you are getting into before saying yes, Kim stresses. If the donor says yes to the procedure, they are told beforehand when the treatment would start and told when the last chance to back out would be.

Stem cell donations can be made in two ways: the donor is hooked to a machine with IVs in each arm. Blood is taken from one arm, stem cells are removed, the blood is replaced through the IV in the other arm where the body grows more stem cells.

Or, the donor is anesthetized, a needle is placed into the hip bone from the back and part of the bone marrow is sucked out.

You might be a little bit sore afterwards, but for less than 24 hours, Turnham says, noting women can be donors but not if they have been pregnant. Young bone marrow is more robust and we regenerate marrow and blood all the time.

Becoming a donor is not a speedy process for people living in smaller communities, but would-be donors can receive a swab kit through the mail and return it postage-free.

Marc Plante, a representative with the national office of Canadian Blood Services, says people can go to http://www.blood.ca and proceed to the Stem Cells tab at the top of the page.

If you have an opportunity to save a life and just put up with a couple of days of discomfort, I would do it in a heartbeat, says Kim, who must also face the reality that Jane could one day be in the same dilemma. If most people had the opportunity to save a life, they would do it.

The Scrantons have been living at Ronald McDonald House for several months, while Jane receives treatment.

Shes as good as can be expected; shes very compromised and we need to make sure shes not exposed to anything, says Kim, noting the first, and this, the fourth phase of treatment, are considered to be the toughest. She has nothing to fight off infection.

There is another terrifying aspect to this disease.

As well as living with the fear of potential relapse, some of the chemo drugs being used to treat Jane increase the risk of heart disease and cause secondary cancer, neuro-cognitive issues and behavioural issues.

We dont know if its coming or what it will be, but we dont have a choice, we have to save her life now and deal with the consequences later, Kim says. Youre just kind of waiting for the other shoe to drop, for things to take a turn, its not fun. And while Jane will require a lot of follow-up assessment in the years to come, the tough little hero turns six on Aug. 12.

She has everything she needs; I just want people to sign up for the bone marrow registry or donate blood, Kim says, noting she is grateful for the communitys support throughout the ordeal.

If you cant donate but would like to help Jane celebrate, you can take birthday cards to the Salmar Grand Theatre where manager Daila Duford will make sure they get to their destination.

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Bone marrow transplant last chance - Revelstoke Review - Revelstoke Review

Investigators from Brigham and Womens Hospital (BWH) and the Harvard Stem Cell Institute (HSCI) may have discovered a way to kill tumor cells that have metastasized to the brain.

The team has developed cancer-killing viruses that can deliver stem cells via the carotid artery, and applied them to metastatic tumors in the brains of clinically relevant mouse models. The elimination of metastatic skin cancer cells from the brains of these preclinical models resulted in prolonged survival, the investigators report. The study, published online this week in the journal PNAS, also describes a strategy of combining this therapy with immune checkpoint inhibitors.

Metastatic brain tumors often from lung, breast, or skin cancers are the most commonly observed tumors within the brain and account for about 40 percent of advanced melanoma metastases. Current therapeutic options for such patients are limited, particularly when there are many metastases, said Khalid Shah, director of the Center for Stem Cell Therapeutics and Imaging (CSTI) in the BWH Department of Neurosurgery, who led the study. Our results are the first to provide insight into ways of targeting multiple brain metastatic deposits with stem-cell-loaded oncolytic viruses that specifically kill dividing tumor cells.

In their search for novel, tumor-specific therapies that could target multiple metastases in the brain without damaging adjacent tissues, the research team first developed different BRAF wild-type and mutant mouse models that more closely mimicked what is seen in patients.

They found that injecting patient-derived, brain-seeking melanoma cells into the carotid arteries of the preclinical models resulted in metastatic tumors forming throughout the brain, mimicking what is seen in advanced melanoma cancer patients. The injected cells express markers that allow them to enter the brain and are labeled with bioluminescent and fluorescent markers to enable tracking by imaging technologies.

To devise a potential new therapy, the investigators engineered a population of bone marrow-derived mesenchymal stem cells loaded with oncolytic herpes simplex virus (oHSV), which specifically kills dividing cancer cells while sparing normal cells.

Previous research by Shah and his colleagues had shown that different stem cell types were naturally attracted to tumors in the brain. After first verifying that stem cells injected to the brain would travel to multiple metastatic sites and not to tumor-free areas in their model, the team injected the oHSV-laden stem cells into the carotid arteries of metastasis-bearing mice. This led to significantly slower tumor growth and increased survival, compared with the models that received unaltered stem cells or control injections.

Shah and his colleagues also developed an immunocompetent melanoma mouse model and explored treatments with both stem cell-loaded oHSV and immune checkpoint blockers such as those that target the PD-1/PD-L1 pathway. They found that PD-L1 immune checkpoint blockade significantly improved the therapeutic efficacy of stem cell-based oncolytic virotherapy in melanoma brain metastasis.

We are currently developing similar animal models of brain metastasis from other cancer types, as well as new oncolytic viruses that have the ability to specifically kill a wide variety of resistant tumor cells, said Shah, who is also a professor at Harvard Medical School and a principal faculty member at the Harvard Stem Cell Institute. We are hopeful that our findings will overcome problems associated with current clinical procedures. This work will have direct implications for designing clinical trials using oncolytic viruses for metastatic tumors in the brain.

The study was supported by a Department of Defense Idea Award and a grant from the National Institutes of Health.

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New approach may kill tumor cells in the brain - Harvard Gazette