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Three women blinded after clinical trial went wrong – Normangee Star

But its always been clear that they could be risky too, especially if theyre not used carefully. The LCSB team has published its results in the scientific journal PLOS Biology.

This study shows that for the first time, targeting the proliferating tumor mass and dormant cancer stem cells with combination therapy effectively inhibited tumor growth and prevented metastasis compared to monotherapy in mice, said Wang, who is a member of the UCLA Jonsson Comprehensive Cancer Center and of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. However, as of April 2016, new rules on human cells and tissue require FDA oversight and approval for such procedures.

Although the women had moderate vision loss prior to the stem cell treatments, a year later their vision ranged from total blindness to 20/200, which is considered legally blind.

NPR contacted the FDA, and was told by a spokeswoman that the agency is now finalizing a series of new guidelines regulating how clinics could use stem cells for treatment purposes. So far, however, scientists only partially understand how the body controls the fate of these all-rounders, and what factors decide whether a stem cell will differentiate, for example, into a blood, liver or nerve cell. He wrote an editorial accompanying the two papers.

As reported Wednesday in the New England Journal of Medicine, one of the women, a 72-year-old, went completely blind after doctors injected stem cells into her eye in an attempt to cure the disease.

But within a week of starting the off-the-charts dangerous therapy at an American clinic, the patients suffered complications.

Two of the patients sought treatment at the universitys hospital for the complications they suffered. The agency also noted that it had previously issued a warning to patients. She said that they were treating patients with their own stem cells.

In addition to charging a fee for treatment, there were several other red flags in the Florida cases that consumers should watch for when considering participation in a clinical trial, Goldberg said. They sought treatment at a Florida clinic that had announced a study to treat the condition on clinicaltrials.gov, a federal database of research studies.

Within days of the stem cell injections she was almost blind and ultimately progressed to complete blindness. Their attorney, Andrew Yaffa of Coral Gables, said that the case was resolved to the mutual satisfaction of the parties but that neither he nor his clients could comment beyond that.

She acknowledged, however, that the clinic had been performing the stem cell procedures.

Shoddy preparation of the stem cells may have led to some of the complications, said the study authors. We feel very confident about the procedures that we do, and weve had great success in many different indications. We believe that regenerative medicine / cellular therapeutics will play a large role in positively changing the natural history of diseases ultimately, we contend, lessening patient burdens, as well as reducing the associated economic impact disease imposes upon modern society.

The body produces a variety of stem cells. It is also costly, at almost $900,000 to develop and test the iPS cells for the first trial, Takahashi adds.

Whatever happened, experts said there was no evidence to suggest the procedure would have helped restore vision, since so little study has been done on whether adipose-derived stem cells can mature into the kinds of retinal cells that are involved in macular degeneration.

This represents a landmark, says Daley. But it proved too slow and expensive, says Shinya Yamanaka of Kyoto University in Japan, who first discovered how to create iPS cells and is a co-author of the NEJM paper. The registry may be useful as a starting point, but patients should then discuss potential trials with qualified physicians, an academic medical center.

A second patient was supposed to be treated, but transplantation was called off after the cells were found to have potential genetic problems. The cells were extracted their from fat, mixed with blood plasma and injected into their eyes.

Even though the safety and effectiveness of this procedure is unknown, all three patients received injections in both eyes. Dr. Thomas Albini of the University of Miami examined the women after they were treated at a clinic in Florida.

Before the procedure, all three women still had at least some vision. Medical experts said the episode raises questions about whether the government and doctors are doing enough to protect patients from the dangers of unapproved therapies.

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Novel gene therapy experiment offers hope for people with certain hearing loss and dizziness disorder – Science Daily


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Novel gene therapy experiment offers hope for people with certain hearing loss and dizziness disorder
Science Daily
In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of Medicine showed that gene ...

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Human heart muscle made from stem cells – Free Press Journal

By FPJ Bureau|Mar 20, 2017 06:26 pm

Melbourne: Scientists have created a functional beating human heart muscle from stem cells, a significant step forward in cardiac disease research. Researchers at The University of Queensland (UQ) in Australia developed models of human heart tissue in the laboratory so they can study cardiac biology and diseases in a dish.

The patented technology enables us to now perform experiments on human heart tissue in the lab, said James Hudson from the UQ School of Biomedical Sciences. This provides scientists with viable, functioning human heart muscle to work on, to model disease, screen new drugs and investigate heart repair, said Hudson.

In the laboratory we used dry ice to kill part of the tissue while leaving the surrounding muscle healthy and viable, Hudson said. We found those tissues fully recovered because they were immature and the cells could regenerate in contrast to what happens normally in the adult heart where you get a dead patch. Our goal is to use this model to potentially find new therapeutic targets to enhance or induce cardiac regeneration in people with heart failure, he said.

Studying regeneration of these damaged, immature cells will enable us to figure out the biochemical events behind this process. Hopefully we can determine how to replicate this process in adult hearts for cardiovascular patients, said Hudson.

Each year, about 54,000 Australians suffer a heart attack, with an average of about 23 deaths every day, researchers said. Heart Foundation Queensland CEO Stephen Vines said the charity was excited to fund such an important research project.

Heart attack survivors who have had permanent damage to their heart tissue are essentially trying to live on half an engine, Vines said. The research will help unlock the key to regenerating damaged heart tissue, which will have a huge impact on the quality of life for heart attack survivors, he added.

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Stem cell treatments can go wrong – Jamaica Observer

Stem cells are the foundation of all our body cells before they differentiate to become specialised cells that grow into our tissues and organs, such as kidney cells, muscle cells, nerve cells, and so on.

They commonly come from two sources: The embryo (embryonic stem cells formed in early development after the human egg is fertilised by a sperm); and adult tissue (adult stem cells, such as those existing in bone marrow to later differentiate to form red blood cells, white blood cells and other components of the blood).

The use of human embryonic stems cells for treatment or research is often frowned upon by some people, as they regard the human embryo as a person that should not be discarded after such endeavours. Consequently, much scientific work has recently been focused on the use of adult stem cells.

THE USE OF STEM CELLS

Stem cells may be beneficial in treating diseases that are amenable to cell replacement. However, this is still a young science, and belief that a particular treatment helps two or three people does not convince the scientific community or the whole society that the treatment will work for everyone so afflicted.

Scientific proof comes from conducting clinical trials, the international gold standard often involving hundreds of people so afflicted and comparing them with an equivalent number of people not afflicted to determine whether a treatment really works for those who receive it.

Whilst many stem cell research projects are currently being conducted in various centres around the world to determine whether they produce benefits, and what may be the possible risks involved, there are also medical clinics that are using stem cells not in a registered research project, but rather in the actual treatment of affected people.

TREATMENT CAN CAUSE HARM

A recent report in the highly respected New England Journal of Medicine informed that three elderly women in Florida had been blinded by an unproven treatment.

They had signed up for a purported clinical trial in 2015 for which they had to pay US$5,000 each. Before surgery, the vision in their eyes varied from 20/30 to 20/200, but within one week after surgery, they experienced a variety of complications, including vision loss, detached retinas and bleeding into their eyes, resulting in total blindness.

The authors of the article from the Standard University School of Medicine sought to make patients, doctors and the various regulatory agencies aware of the risks of such a minimally regulated, patient-funded research. It stated that some clinics appeal to patients that are desperate for care and who hope that stem cells will be their answer, but as in the case of these women, some of these current enterprises are very dangerous.

At this particular clinic, fat cells were taken from the patients abdomens and processed to obtain stem cells which were then injected into their eyes. The patients reported that the entire process took less than one hour. The patients had both eyes treated at once, even though most doctors would opt for a conservative approach to observe how the first eye responds.

THE NEED FOR THE REGULATION OF RESEARCH

The article stated that while there is a lot of well-founded evidence for the positive potential of stem cell treatment for many human diseases, such treatments should be conducted in a well-designed clinical trial based on pre-clinical research.

The treatment done for the women lacked nearly all the components of a properly designed clinical trial, including a hypothesis based on laboratory experiments, the involvement of a control group of people and a treatment group, the safe collection of data, the masking of clinical and patient groups, and plans for follow-up.

Clinics offering stem cell treatments exist in Jamaica, The Bahamas and Cuba. However, while both The Bahamas and Cuba have developed regulations that stipulate in law the conditions to be met for stem cell treatments and research within their jurisdictions, Jamaica has developed no such regulation.

THE MEDICAL ACT DOES NOT PROVIDE PROTECTION

The Medical Act of Jamaica was passed in 1976, but does not mention or provide any guidance or protection regarding research with human participants.

Its focus was to: Register medical practitioners; appoint examiners to conduct exams for people applying for registration, and ensure the maintenance of proper professional conduct by practitioners.An amendment in 2004 added the requirement of continuing medical education for practitioners.

Guyana and St Lucia are the only countries in the Caribbean that have joined the progressive countries who all have legislation governing research with human participants within their borders. Regulations should stipulate the requisite conditions, including that treatment and research be monitored by an appropriate ethics committee to meet all international standards.

Without this, vulnerable people seeking health benefits will unknowingly continue to subject themselves to risks of harm without the protection that proper regulations can provide.

Derrick Aarons MD, PhD is a consultant bioethicist/family physician, a specialist in ethical issues in medicine, the life sciences and research, and is the Ethicist at the Caribbean Public Health Agency (CARPHA). (The views expressed here are not written on behalf of CARPHA)

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Man with 45% burns healed with stem cell treatment – ETHealthworld.com

Mumbai: A 45-year-old man -- suffering from 45 per cent burns due to a chemical spill at work -- has been healed with stem cell treatment, said the authorities at a hospital here on Friday.

Ram Naik (name changed) was brought to city-based StemRx Bioscience hospital after receiving first aid in another hospital. Nearly 45 per cent of his upper body was burned due to a chemical spill during work.

The impact of the burns led to a charred look on his face and body. Also, joint mobility due to the burns was reduced. The outer layer of the skin was affected, facial burns were of grade II level and in some instances grade III burns were also present, leading to deeper structures like the subcutaneous tissue also being affected.

According to the doctors, burn wound healing involves a series of complex processes, with healing time and scar tissue being the most important parameters that affect treatment outcomes. Burn injuries, especially severe ones, are proving to have devastating effects on the affected patients.

They said that stem cells have been recently applied in burn wounds to promote superior healing of the wounds. Not only have stem cells been shown to promote better and faster healing of the burn wounds, they are also capable of decreasing inflammation and prevent scar progression and fibrosis.

Therefore, the doctors decided to provide Naik stem cell treatment.

Regenerative Medicine researcher at Stemrx Bioscience hospital Pradeep Mahajan said that within two days, a notable improvement in his condition was observed and the swelling and charred appearance started reducing.

"Mild eyelid movements were noticed and on the third day the burns started drying on the face and he could open his mouth and eyes. Growth factors derived from platelets, cells, fibroblasts, collagen-based gel etc. was used during treatment. In addition, in areas with deep burns, sheets of PGLA coated with cells and growth factors were used," said Mahajan, adding that different medication and treatments were imparted and closed dressing was avoided.

"Blood transfusion and supplementary fluids were given intravenously to maintain systemic homeostasis," said Mahajan.

Stating that on 5th and 6th day following treatment, dry scales from the face and body started peeling off, the doctor's team also observed impressive changes such as new skin forming within a week of treatment with cells and growth factors.

By conventional modalities, it takes more than eight weeks for the patient to heal and many additional months for the patient to be able to regain joint and facial movements.

"By the 10th day of the treatment, dry scales completely peeled off and by the 14th day the patient had no tenderness or burning pain. Joint movements became free as well, Steady rate of progression of healthy skin formation is being noticed. Areas with deep burns are also healing at a rapid rate and I am confident that within a month we will accomplish thorough healing and the patient will be back to normal," Mahajan said.

Medical sciences say that such cases are challenging to manage considering the degree of impairment they result in due to prolonged healing period. Also, through conventional therapeutic modalities healing occurs with scar formation and results in contractures. Chances of systemic complications and infection are also high.

However according to the medical team, by using stem cells, the natural healing potential of the body is used, leading to reduction of healing time and promoting regeneration of affected tissues. This also reduces the mental trauma and financial burden that a patient goes through when under conventional management.

"Stem cell-based therapy has offered a novel and powerful strategy in almost every medical specialty including burns and wound management. Stem cells have proven to have tremendous potential in enhancing wound healing and facilitating skin regeneration," Mahajan said.

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Stem cells seem speedier in space – Concord Register

Cultured stem cells. Credit: BioServe Inc., University of Colorado

Growing significant numbers of human stem cells in a short time could lead to new treatments for stroke and other diseases. Scientists are sending stem cells to the International Space Station to test whether these cells proliferate faster in microgravity without suffering any side effects.

Therapeutic uses require hundreds of millions of stem cells and currently no efficient way exists to produce such quantities. Previous research suggests that could help, and the space station is home to the nations only national lab in microgravity.

Some types of stem cells grow faster in simulated microgravity, according to Abba Zubair, a researcher at the Mayo Clinic in Jacksonville, Florida. Zubair is principal investigator for the Microgravity Expanded Stem Cells investigation, which is cultivating human stem cells aboard the for use in clinical trials back on Earth. He holds a doctor of medicine degree in transfusion medicine and cell therapy and a doctorate of philosophy in tumor immunology.

Human stem cells are cells that have not yet specialized in function and can divide into a spectrum of cell types, rejuvenating and repairing tissue throughout a persons lifetime. Stem cells in every organ of the body, including skin and bones, maintain those organs and repair tissue by dividing and differentiating into specialized cells.

The Plate Habitat (PHAB) containing BioCell cassettes for the Expanded Stem Cell investigation aboard the space station. Credit: BioServe Inc., University of Colorado

Harvesting a persons stem cells and growing enough of them for use in therapies has proven difficult, though. Researchers have successfully grown , found in bone marrow, but growing sufficient quantities takes weeks. That could be too late for treatment of some conditions.

Stem cells are inherently designed to remain at a constant number, Zubair explains. We need to grow them faster, but without changing their characteristics.

The first phase of the investigation, he adds, is answering the question: Do stem cells grow faster in space and can we grow them in such a manner that they are safe to use in patients?

Investigators will examine the space-grown cells in an effort to understand the mechanism behind microgravitys effects on them. The long-term goal is to learn how to mimic those effects and develop a safe and reliable way to produce stem cells in the quantities needed.

Abba Zubair in his lab at Mayo Clinic in Jacksonville, Fla. Credit: BioServe Inc., University of Colorado

The second phase will involve testing clinical application of the cells in patients. Zubair has been studying treatment of stroke patients with lab-grown stem cells and plans to compare those results with use of the space-grown stem cells.

What is unique about this investigation is that we are not only looking at the biology of the and how they grow, but focusing on application, how we can use them to treat patients, he says.

The investigation expands existing knowledge of how microgravity affects stem cell growth and differentiation as well as advances future studies on how to produce large numbers of for treating stroke and other conditions.

The faster that happens, the better for those who could benefit from .

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Scientists know how to grow human heart tissue – Institute …

Scientists used stem cells to grow human heart tissue that contracted spontaneously in a petri dish marking progress in the quest to manufacture transplant organs.

A team from the University of Pittsburgh, Pennsylvania, used induced pluripotent stem (iPS) cells generated from human skin cells to create precursor heart cells called MCPs. iPS cells are mature human cells reprogrammed into a versatile, primitive state from which they can be prompted to develop into any kind of cell of the body. The primitive heart cells created in this way were attached to a mouse heart scaffold from which the researchers had removed all mouse heart cells, they wrote in the journal Nature Communications.

The scaffold is a network of non-living tissue composed of proteins and carbohydrates to which cells adhere and grow on. Placed on the 3D scaffold, the precursor cells grew and developed into heart muscle, and after 20 days of blood supply the reconstructed mouse organ began contracting again at the rate of 40 to 50 beats per minute, said a University of Pittsburgh statement.

It is still far from making a whole human heart, added senior researcher Lei Yang. Ways have to be found to make the heart contract strongly enough to pump blood effectively and to rebuild the hearts electrical conduction system. However, we provide a novel resource of cells iPS cell-derived MCPs for future heart tissue engineering, Yang told AFP by email. We hope our study would be used in the future to replace a piece of tissue damaged by a heart attack, or perhaps an entire organ, in patients with heart disease.

According to the World Health Organisation, an estimated 17 million people die of cardiovascular ailments every year, most of them from heart disease. Due to a shortage of donor organs, end-stage heart failure is irreversible, said the study. More than half of patients with heart disease do not benefit from drugs. Heart tissue engineering holds a great promise based on the reconstruction of patient-specific cardiac muscle, the researchers wrote.

Last month, scientists in Japan said they had grown functional human liver tissue from stem cells in a similar process. Creating lab-grown tissue to replenish organs damaged by accident or disease is a Holy Grail for the pioneering field of stem cell research. Until a few years ago, when iPS cells were created, the only way to obtain stem cells was to harvest them from human embryos. This was controversial because it required the destruction of the embryo, a process to which religious conservatives and others object.

Source: http://news.sudanvisiondaily.com

As the Chief Doctor of the Institute of Cell Therapy, Y.V.Gladkikh, MD, PhD, Dr. med. sc. commented: In addition to laboratory success in obtaining the functional cardiac tissue, currently there is evidence of successful implantations of heart valves and blood vessels fragments, grown from stem cells, to patients. And in 2012, the Ministry of Health of Ukraine officially approved method of treatment of critical limbs ischemia with the use of cell preparation Angiostem, developed by the biotechnological laboratory of the Institute of Cell Therapy.

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Money for research, hope for a cure: Race honors Eliza O’Neill and Sanfilippo kids – The State


The State
Money for research, hope for a cure: Race honors Eliza O'Neill and Sanfilippo kids
The State
On Saturday, Hickey and some of her genetic counseling peers joined dozens of others at a 5K race fundraiser to increase awareness of the rare, degenerative disease and raise money for research into an effective treatment or cure. There's a lot of ...
They isolated themselves for 726 days to give their daughter a chance at lifeDurham Herald Sun

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Fighting Cancer With Fundraising: Meet Milwaukee’s Dr. John Hanson – Milwaukee Magazine

Dr. John Hanson has a colorful, stylish office in the Cudahy Tower, converted from an old apartment, reducing his morning commute to a short stroll from a condominium in the building. A retired oncologist, Hanson now spends his time spearheading his 5-year-old John P. Hanson Foundation for Cancer and Cellular Research, which supports research into cell therapy and other forms of cancer immunotherapy, advanced treatments that direct the power of the bodys immune system (including T-cells) against unwanted cancer cells. Hanson is currently gathering funding to support up to three young researchers at the cutting-edge Robert H. Lurie Comprehensive Cancer Center at Northwestern University, with hopes of one day endowing a professorship.

Should cancer patients challenge their doctors?I think thats fine. Theres no cure. There might be two or three different options. And the next thing is, is there a clinical trial or an attempt to improve on standard practices available? Its all about the patient getting better.

Must the immune system miss something for a tumor to form? No. Its not missing something. What happens is the cancer outfoxes everyone. Instead of supporting the body, it begins to support itself and becomes self-aggrandizing.

Cell therapy sounds great. Does it have limitations?It works extremely well with melanoma because there are large genetic differences. Breast cancer has been treated successfully. A few colon cases have been treated successfully. A pancreatic cancer has been treated successfully, one or two [times]. The imperative is you must develop this [approach] for common cancers because theyre what kill people. It works extremely well with melanoma because there are large genetic differences. Breast cancer has been treated successfully. A few colon cases have been treated successfully. A pancreatic cancer has been treated successfully, one or two [times]. The imperative is you must develop this [approach] for common cancers because theyre what kill people.

During immunotherapy, can the immune system target things other than cancer? That does happen. Diabetes can occur. Hypothyroidism can occur. Hypopituitarism can occur, and bowel diseases can happen. Its a reaction to the immune system being turned on too much.

What was it like to work with cancer patients? Did you find it rewarding? Oh sure. You can help the sick. The art is to listen to what the patient wants. If you listened enough early on, you could say, This is what you want, and this is what I can do. If they wanted to do something high risk, we would talk about it for a week or two to make sure they got it and understood what the risks were. The goal was, I am going to get you through this. You can do this, and you will do this. We have to get to the end of a course [of treatment] to see if it works. Theres a sorrow at death, but we tried as best we could. Can we learn something to help the other families, the other patients? Almost always we do. Was it worth it? Absolutely. Every human being who has cancer wants to live.

Why hasnt immunotherapy taken over the field?Doctors are committed to the life and well-being of patients, and theyre not convinced it works. But youre dealing with people who are dying and sick. I think theres an imperative. You cant keep putting up with the same old shit. Its not in human nature to accept that what is [is beyond improvement]. You have to try.

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Measuring Heart Toxicity of Cancer Drugs | Technology Networks – Technology Networks

A stem cell-derived heart muscle cell. Proteins that are important for muscle cell contraction are highlighted in red and green, and cell nuclei are blue. Credit: Joseph C. Wu, M.D., Ph.D., Stanford Cardiovascular Institute

Using human heart cells generated from adult stem cells, researchers have developed an index that may be used to determine how toxic a group of cancer drugs, called tyrosine kinase inhibitors (TKIs), are to human cells. While 26 TKIs are currently used to treat a variety of cancers, some can severely damage patients hearts, causing problems such as an irregular heartbeat or heart failure.

For the study, reported February 15 in Science Translational Medicine, the researchers used stem cell-derived heart cells from 13 volunteers to develop a cardiac safety index that measures the extent to which TKIs kill or alter the function of heart cells. They found that the TKIs' toxicity score on the index was generally consistent with what is known about each drug's heart-related side effects.

This work follows on the heels of an earlier study from the same research team, published in Nature Medicine, in which they assessed the heart cell toxicity of doxorubicin, a chemotherapy drug that also causes heart-related side effects, including heart failure. In that study, the researchers used stem cell-derived heart cells from women with breast cancer to correctly predict how sensitive each womans heart cells were to doxorubicin.

Such tests could ultimately help the pharmaceutical industry identify drugs that cause heart-related side effects earlier in the drug development process and help the Food and Drug Administration (FDA) during the drug review and approval process, said the study's senior author Joseph C. Wu, M.D., Ph.D., director of the Stanford Cardiovascular Institute.

I hope this research will be helpful for individual patients, once we further implement precision medicine approaches, he added.

Ranking Heart Toxicity

To assess the potential risk of heart toxicity for drugs in development, pharmaceutical companies use laboratory tests involving animals (usually rats or mice) or cells from animals or humans that are engineered to artificially express heart-related genes. Drug candidates that appear to have an acceptable balance of benefits and risks typically proceed to testing in human clinical trials.

But there can be biological differences between these existing models and humans, so non-clinical lab tests can have significant limitations, explained Dr. Wu.

Currently, the first time humans are exposed to a new drug is during clinical trials, he said. We think it would be great if you could actually expose patients heart, brain, liver, or kidney cells to a drug in the lab, prior to clinical treatment, allowing researchers to determine whether the drug has any toxic effects.

Dr. Wu, a cardiologist by training, studies toxicities cancer drugs cause in heart cells. Human heart muscle cells (called cardiomyocytes), however, are hard to obtainrequiring risky heart surgery that may be of no direct benefit to the patientand are notoriously difficult to grow in the lab.

As an alternative, researchers have developed a method to produce heart cells from human induced pluripotent stem cells (hiPSCs). hiPSCs are created by genetically engineering normal human skin or blood cells to express four specific genes that induce them to act like stem cells. Chemical treatments can prompt hiPSCs to develop into mature cell types, such as heart muscle cells.

A large body of research has established that human adult stem cell-derived heart cells, which function and grow in cell culture, can be used as an initial model to screen drug compounds for toxic effects on the heart, said Myrtle Davis, Ph.D., chief of the Toxicology and Pharmacology Branch of NCIs Division of Cancer Treatment and Diagnosis, who was not involved in the studies.

For the Science Translational Medicine study, Dr. Wu and his colleagues set out to determine if a panel of human stem cell-derived heart cells could be used to evaluate the heart toxicity of 21 different FDA-approved TKIs.

They generated hiPSC-derived heart endothelial, fibroblast, and muscle cells from 13 volunteers: 11 healthy individuals and 2 people with kidney cancer who were being treated with a TKI. Using drug concentrations equivalent to what patients receive, the investigators next determined how lethal each TKI was to the heart cells.

They found that several TKIs were very lethal to endothelial, fibroblast, and heart muscle cells from all 13 individuals, while others were more benign.

Stem cell-derived heart muscle cells grown in a dish spontaneously contract as a beating heart does, so the researchers also analyzed the effects of TKIs on the cells beat rate, or contractility. They found that several TKIs altered the cells beat rate before they were killed by the drug treatment. If severe enough, an irregular heartbeat (called an arrhythmia), can disrupt normal heart function.

From these lethality and contractility experiments, the team developed a cardiac safety index, a 0-to-1 scale that identifies how toxic a TKI is to heart cells (with 0 being the most toxic). They then used the index to rank the 21 TKIs. The control treatment scored a 1, while a few TKIs that are labeled by the FDA with boxed warnings for severe heart toxicity scored close to 0.

Safety indices like this one can be very useful during drug discovery, said Dr. Davis, and the applicability of the index developed by Dr. Wu and his colleagues will become clear when they evaluate its performance with more compounds.

And for the safety index to be applicable to more patients, the panel of cells used to develop it would need to be gathered from a sufficiently representative population of people reflecting different ages, races/ethnicities, health statuses, and other characteristics, said Lori Minasian, M.D., deputy director of NCIs Division of Cancer Prevention, who was not involved in either study.

For example, the study did not include cells derived from patients with [pre-existing] cardiac disease, said Dr. Davis.

A Personalized Approach

In addition to their potential application during drug development, Dr. Wu believes that stem cell-derived heart cells could potentially be used to predict toxicity risk for individual patients. He and his colleagues explored this possibility in their Nature Medicine study.

Doxorubicin, used on its own or in combination with other drugs, is an effective treatment for breast cancer and several other types of cancer. Like TKIs, however, it is known to cause heart toxicities, such as arrhythmias and heart failure, in a small proportion of patients. But there has been no way to predict which patients will experience these side effects.

The researchers developed stem cell-derived heart cells from eight women with breast cancer who had been treated with doxorubicinhalf of whom experienced cardiotoxicity from the treatment and half who did not.

In several different lab tests, the heart cells from women who had experienced cardiotoxicity were more sensitive to doxorubicin than those from women who had not. More specifically, in heart cells from women who had experienced cardiotoxicity, doxorubicin treatment caused more severe irregularities in cell contractility, and even low concentrations of the drug killed the cells.

An Improved Model

While the stem cell-derived heart cell model may be an improvement over the current [drug testing] system, its not perfect, said Dr. Minasian. For example, the model does not capture contributions of other organs and cells to the toxic effects of a drug, she explained. The drug may be broken down in the liver, for instance, and side products (called metabolites) may also cause toxic effects.

In addition, the lab-grown stem cell-derived version of someones heart cells are not going to be exactly the same as the cells found in that persons heart, Dr. Wu noted. Nevertheless, they reflect the same genetics and they are pretty good at predicting drug response, he said.

Looking forward, Dr. Minasian said, figuring out how to best use this approach is going to take more work, but being able to better predict human response [to cancer drugs] is important.

The research teams next steps include conducting prospective studies to determine whether they can use a patients stem cell-derived heart cells to potentially predict if that person will develop heart toxicity before they actually receive cancer treatment.

This article has been republished frommaterialsprovided byNCI. Note: material may have been edited for length and content. For further information, please contact the cited source.

Reference

Sharma, A., Burridge, P. W., McKeithan, W. L., Serrano, R., Shukla, P., Sayed, N., ... & Matsa, E. (2017). High-throughput screening of tyrosine kinase inhibitor cardiotoxicity with human induced pluripotent stem cells. Science translational medicine, 9(377), eaaf2584.

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Lungs make platelets, store blood stem cells: Study – The San Diego … – The San Diego Union-Tribune

Challenging a long-held model about how blood is formed, a study led by UC San Francisco researchers has found that the lungs play a crucial role in the process, producing half of blood platelets and also storing blood-forming stem cells.

The study, performed in mice, also found that blood stem cells and progenitor cells travel freely between the lungs and bone marrow, long considered the primary source of blood production.

If found to occur in humans, this discovery about the lungs role in blood production could provide new approaches for treating blood diseases, pulmonologist Mark R. Looney, M.D., senior author of the study, said in a statement.

Moreover, the success of lung transplantation might be increased by better understanding this process. Immune reaction between donor blood cells in the lungs and the host could contribute to transplant rejection, the study stated.

The study was published Wednesday in the journal Nature. When placed online, the study can be found at j.mp/lungblood.

"This finding definitely suggests a more sophisticated view of the lungs -- that they're not just for respiration but also a key partner in formation of crucial aspects of the blood," Looney said. "What we've observed here in mice strongly suggests the lung may play a key role in blood formation in humans as well."

"Dr. Looney and his team have disrupted some traditional ideas about the pulmonary role in platelet-related hematopoiesis, paving the way for further scientific exploration of this integrated biology," said Traci Mondoro, of the National Heart, Lung and Blood Institute, in the statement.

While it has been known for decades that platelets can be made in the lungs, the study indicates that lung production is a more important factor than previously thought, said Mondoro, project officer at the Translational Blood Science and Resources Branch of the NHLBI, a division of the National Institutes of Health.

Researchers studied the lungs of mice genetically engineered to make a green fluorescent protein in platelets and platelet-making cells called megakaryocytes. They found a larger than expected number of these cells.

Megakaryocytes that release platelets in the lungs originate from extrapulmonary sites such as the bone marrow; we observed large megakaryocytes migrating out of the bone marrow space, the study said. The contribution of the lungs to platelet biogenesis is substantial,accounting for approximately 50% of total platelet production or 10 million platelets per hour.

After discovering this process, the researchers looked for more signs of blood cells residing in the lungs. They found progenitor cells that turn into megakaryocytes, along with blood-forming, or hematopoietic, stem cells. a total of 1 million per mouse lung.

These cells constitute a reservoir that can replenish the bone marrow, the study said.

Under conditions of thrombocytopenia (platelet deficiency) and relative stem cell deficiency in the bone marrow, these progenitors can migrate out of the lungs, repopulate the bone marrow, completely reconstitute blood platelet counts, and contribute to multiple hematopoietic lineages, the study stated. These results identify the lungs as a primary site of terminal platelet production and an organ with considerable hematopoietic potential.

The studys co-first authors are Emma Lefranais and Guadalupe Ortiz-Muoz, both of UCSF. It was supported by the UCSF Nina Ireland Program in Lung Health; the UCSF Program for Breakthrough Biomedical Research, and the National Heart, Lung, and Blood Institute.

bradley.fikes@sduniontribune.com

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‘If that was my little girl I’d want someone to step up’: Stem cell donor on lifesaving transplant – ChronicleLive

Selfless Ray Noble may never meet the stranger whose life he saved.

The 29-year-old registered as a stem cell and bone marrow donor four years ago after a young girl his wife knew was diagnosed with cancer.

If that was my little girl Id want someone to be there for her, he said.

Ive been a blood donor for a while, so I thought why not sign up to the stem cell register as well.

And last year dad-of-one Ray, from Wallsend, made a life-saving donation after being told he was a match for an unknown patient in urgent need of a transplant.

Now blood cancer charity Anthony Nolan have urged more people to follow Rays example after a survey revealed that 50% of young men from the North East could not be encouraged to sign up to a blood stem cell or bone marrow register for any reason.

Every year there are 2,000 people in the UK in need of a bone marrow or stem cell transplant. This is usually their last chance of survival.

For Ray, the path to becoming a blood cancer patients last hope started when a relative of his wifes friend was diagnosed with the disease.

The process was pretty simple, he said.

I followed the instructions on the Anthony Nolan website about how to sign up.

Within a week or two they sent me a spit test, where I basically had to spit into a tube and send it off so it could be analysed.

I then got a card a few weeks later saying I was on the register.

Since signing up, Ray has been identified as a potential match for two patients.

About two years ago Anthony Nolan got in touch to say that I was a potential match for someone and I had to go and give some samples.

On that one they managed to find a closer match - I was eight out of 10 and they found a 10 out of 10, which was obviously better for the patient.

Then around Christmas last year they confirmed that I was a match for someone.

After undergoing several health checks and injections to stimulate the stem cells in his blood, Ray travelled down to Sheffield in April last year to make the donation.

All in all it took about four or five hours, he said. Id been aching a bit before the procedure because of the injections but afterwards I felt totally fine.

Ray, who is dad to two-year-old Ariana, has since convinced several friends and relatives to sign up.

For me its a question of, why not?, he said.

Its not that likely that youre ever going to be asked to donate - its just a case of being on there for someone if they need it.

I always ask people: How would you feel if it was your child or parent or cousin, if they needed a donor and you werent a match - would you want someone to step up and help them?

Every 20 minutes someone in the UK finds out they have a blood cancer.

Around 2,000 people in the UK in need of a bone marrow or stem cell transplant every year. This is usually their last chance of survival.

75% of UK patients wont find a matching donor in their families. So they turn to Anthony Nolan to find them an unrelated donor.

Healthy adults aged between 16 and 30 can sign up for a simple, pain-free test through the Anthony Nolan Trust.

The charity particularly need more young men to sign up. They produce more stem cells than women and are six times more likely to donate, but make up just 15% of the register. They also need more donors from black and minority ethnic backgrounds as they often struggle to find matches for people in these groups.

Check the list of criteria to make sure youre eligible to join and fill in an application form, either online or at an Anthony Nolan recruitment event.

If you come to a recruitment event and your application is OK, you can give your saliva sample there. If you apply online, youll be sent spit kit in the post. All you need to do is spit into a small tube and post it back.

The sample will be tested and the results put in the charitys database. Every time someone needs a transplant, theyll automatically compare their tissue to yours and the 620,000 other individuals on the register.

You can donate your stem cells in two ways.

Nearly 90% of people donate their stem cells quickly and easily in a process similar to giving blood, called peripheral blood stem cell collection.

The other 10% donate through bone marrow, where they give cells from the bone marrow in their pelvis.

If youre on the register, you must be happy to donate stem cells in either way.

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'If that was my little girl I'd want someone to step up': Stem cell donor on lifesaving transplant - ChronicleLive

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Injection with own stem cells alleviates chest pains, angina, study finds – Genetic Literacy Project

A non-surgical treatment that uses a patients own bone marrow stem cells to treat chest pain or angina improved both symptoms and the length of time treated patients could be physically active, according to recent research.

We injected a catalyst molecule that caused bone marrow stem cells to enter the patients blood, then harvested them to re-inject into the patient,said Hadyanto Lim, Ph.D., study senior author.

Thirty minutes after the cell separation procedure finished, the collected stem cells were injected back into the patient through an IV.

Four weeks after receiving the treatment, patients experienced significantly fewer angina-related symptoms, and they were able to exercise at a higher intensity and for a longer period of time.

The studys limitations are the small number of patients and absence of a control group. Because no control group was used, the placebo effect cannot be ruled out, Lim noted.

Although this treatment is currently used to treat some cancers multiple myeloma and lymphoma it will need more investigation before it can be made available to the general public to treat angina, according to Lim.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Hard to treat chest pain may be improved with a patients own stem cells

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Space wombs for stem cells: Satellites could help accelerate the discovery of disease cures – Salon

This week a very special delivery was made from space that will help further research that could eventually lead to a mind-blowing, futuristic way to cure diseases: shooting unmanned satellite wombs into orbit and then retrieving from them batches of stem cells that can be used to treat patients. Regardless of the outcome, the scientific experiment will still advance our knowledge of these unique cells.

On Thursday Dr. Abba Zubairat the Mayo Clinic in Jacksonville, Florida, received frozen stem cells grown at the International Space Station. The package was part of the 5,400 pounds of scientific samples and equipment that splashed down on Sunday off the coast of California inside a SpaceX Dragon-10 capsule completing a historic round-trip mission.

Up there, one of the astronauts helped us to image the cells, harvest the cells and freeze them in a way that we can use them here on Earth and compare them to cells we grew here in the lab, Zubair, the principal investigator of the stem cell experiment, told Salon.

Zubairs team will look to see if the culture grown in the near-zero gravity of low-space orbit, about 250 miles above the Earths surface, results in healthier cells than onesgrownin aterrestrial lab. If so then it would helpconfirm the theory that microgravity, which resembles the weightless-likebuoyancyof female womb, is best environment for growing stemscells.

Stem cells, from which all other types of cells originate, are the bodys raw materials, and as such offer immense potential to cure many diseases. Doctors already use stem cells forbone-marrow transplants and treating blood-related diseases like leukemia, as well asfor some eye-related disorders. Researchers believe were only in the very early stages of developing revolutionary stem cell therapiesto combat cancer, Alzheimers disease, Parkinsons disease, Type 1 diabetes, heart disease and strokes. In the future, stems cellscience could even lead to growing organs in a lab that can be transplanted into humans.

But stem cells are finicky. As they replicate in a lab, many of them develop imperfections and have to be discarded. It can take a month to grow the roughly 200,000 cells needed to treat one patient, Zubair said. Gravity might be the culprit.

In nature, these cells start their life after an egg is fertilized. Humans, right from conception, develop almost in a microgravity environment, Zubair said. Fetuses develop in amniotic fluid. Theyre buoyant, which cancels the effect of gravity because theyre suspended in a liquid. Thats how three-dimensional growth in a fluid environment is possible. We think gravity does play a role in the shape and development of the cells and how organs develop.

In other words, if the cells are suspended in fluid, they can grow and move in any direction, producing more of them, compared withhow they grow on a flat surface, like in a petri dish.

This is why stem cells are typically grown in a bioreactor, a common bioengineering tool that gently stirswater containing the seed cells and certain nutrients that promote growth. But because of the way gravity affectsfluids, many of the cells become damaged and cant be used for treatment. (In the language of physics, the problem has to do with something called shearing force.) By placing a bioreactor in the microgravity of orbit, the effects of gravity on liquid mechanics is virtually eliminated.

If growing stem cells in spaceproves to be efficient, thats when things get interesting. Growing stem cells at the International Space Station is anexperimental endeavor, so its not really a viable place to begin manufacturing themin great quantities. But theoretically, Zubair says, bioreactor satellites could be put into orbit and left there to grow cells until theyre remotely called back to Earth or sent wherever future interplanetary pilgrims wind up. As the cost of sending small satellites into low orbit falls, this system could be commercially viable.

There are companies that are interested in developing a floating lab in space to grow not only stem cells but also tissues and organs down the road for human use or for use elsewhere as we hopefully colonize other planets, like Mars, Zubair said.

This might seem out of this world, but the technology for growing stem cells remotely already exists. If space is the place to grow human parts and this research will help to determine that then designing systems and deploying these bioreactor space wombs might not be that far off in the future.

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An unexpected new lung function has been found – they make blood … – ScienceAlert

Researchers have discovered that the lungs play a far more complex role in mammalian bodies than we thought, with new evidence revealing that they don't just facilitate respiration - they also play a key role in blood production.

In experiments involving mice, the team found that they produce more than 10 million platelets (tiny blood cells) per hour, equating to the majority of platelets in the animals' circulation. This goes against the decades-long assumption that bone marrow produces all of our blood components.

Researchers from the University of California, San Francisco also discovered a previously unknown pool of blood stem cells that makes this happen inside the lung tissue - cells that were incorrectly assumed to mainly reside in bone marrow.

"This finding definitely suggests a more sophisticated view of the lungs - that they're not just for respiration, but also a key partner in formation of crucial aspects of the blood," says one of the researchers, Mark R. Looney.

"What we've observed here in mice strongly suggests the lung may play a key role in blood formation in humans as well."

While the lungs have been known to produce a limited amount of platelets - platelet-forming cells called megakaryocytes have been identified in the lungs before - scientists have long assumed that most of the cells responsible for blood production are kept inside the bone marrow.

Here, aprocess called haematopoiesiswas thought tochurn out oxygen-laden red blood cells, infection-fighting white blood cells, and platelets - blood components required for the clotting that halts bleeding.

But scientists have now watched megakaryocytesfunctioning from within the lung tissue to produce not a few, but most of the body's platelets.

So how did we miss such a crucial biological process this whole time?

The discovery was made possible by a new type of technology based on two-photon intravital imaging - a similar technique to one used by a separate team this week to discover a previously unidentified function of the brain's cerebellum.

The process involves inserting a substance called green fluorescent protein (GFP) into the mouse genome - a protein that's naturally produced by bioluminescent animals such as jellyfish, and is harmless to living cells.

The mouse platelets started to emit bright green fluorescence as they circulated around the body in real time, allowing the team to trace their paths like never before.

They noticed a surprisingly large population of platelet-producing megakaryocytes inside the lung tissue, which initially didn't make much sense, seeing as they're usually associated with bone marrow.

"When we discovered this massive population of megakaryocytes that appeared to be living in the lung, we realised we had to follow this up," says one of the team, Emma Lefranais.

They found that this huge supply of megakaryocytes is actually producing more than 10 million platelets per hour in the lungs of mice, which means at least half of the body's total platelet production is occurring in the lungs.

Here's what it looks like:

Further experiments also revealed vast amounts of previously hidden blood stem cells and megakaryocyte progenitor cells (cells that give rise to megakaryocyte and red blood cells) sitting just outside the lung tissue - about 1 million per mouse lung.

When the researchers traced the entire 'life cycle' of the megakaryocytes, they found that they likely originate in the bone marrow, then make their way to the lungs, where they start platelet production.

"It's fascinating that megakaryocytes travel all the way from the bone marrow to the lungs to produce platelets," says one of the team, Guadalupe Ortiz-Muoz.

"It's possible that the lung is an ideal bioreactor for platelet production because of the mechanical force of the blood, or perhaps because of some molecular signalling we don't yet know about."

The researchers wanted to investigate if their discovery could have an effect on how we treat disorders such aslung inflammation, bleeding, and transplantation in the future, by transplanting lungs with fluorescent megakaryocyte progenitor cells into mice with low platelet counts.

The transplants produced a massive burst of platelets that quickly restored the depleted platelet counts to normal levels, and the effect lasted for several months.

Another experiment tested what would happen if the bone marrow wasn't playing a role in blood production.

The team implantedlungs with fluorescent megakaryocyte progenitor cellsinto mice that had been engineered to have no blood stem cells in their bone marrow.

As Michael Irving reports for New Atlas, they watched as the fluorescent cells from the transplanted lungs made their way to the bone marrow, where they not only helped to produce platelets, but also other key blood components, such as neutrophils, B cells and T cells.

The findings will need to be replicated in humans before we can know for sure that the same process is occurring within our own bodies, but the study makes a strong case for this hidden function in what could be one of our most underrated organs.

It will likely also prompt scientists to investigate further how the bone marrow and lungs work together to produce our blood supply.

"It has been known for decades that the lung can be a site of platelet production, but this study amplifies this idea by demonstrating that the [mouse] lung is a major participant in the process," Traci Mondoro from the US National Heart, Lung, and Blood Institute, who was not involved in the study, said in a press statement.

"Looney and his team have disrupted some traditional ideas about the pulmonary role in platelet-related hematopoiesis, paving the way for further scientific exploration of this integrated biology."

The research has been published in Nature.

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Man with 45% burns healed with stem cell treatment | Zee News – Zee News

Mumbai:A 45-year-old man, who was suffering from 45 per cent burns due to a chemical spill at work, has been healed with stem cell treatment, said the authorities at a hospital here on Friday.

Ram Naik (name changed) was brought to city-based StemRx Bioscience hospital after receiving first aid in another hospital. Nearly 45 per cent of his upper body was burned due to a chemical spill during work.

The impact of the burns led to a charred look on his face and body. Also, joint mobility due to the burns was reduced. The outer layer of the skin was affected, facial burns were of grade II level and in some instances grade III burns were also present, leading to deeper structures like the subcutaneous tissue also being affected.

According to the doctors, burn wound healing involves a series of complex processes, with healing time and scar tissue being the most important parameters that affect treatment outcomes. Burn injuries, especially severe ones, are proving to have devastating effects on the affected patients.

They said that stem cells have been recently applied in burn wounds to promote superior healing of the wounds. Not only have stem cells been shown to promote better and faster healing of the burn wounds, they are also capable of decreasing inflammation and prevent scar progression and fibrosis.

Therefore, the doctors decided to provide Naik stem cell treatment.

Regenerative Medicine researcher at Stemrx Bioscience hospital Pradeep Mahajan said that within two days, a notable improvement in his condition was observed and the swelling and charred appearance started reducing.

"Mild eyelid movements were noticed and on the third day the burns started drying on the face and he could open his mouth and eyes. Growth factors derived from platelets, cells, fibroblasts, collagen-based gel etc. was used during treatment. In addition, in areas with deep burns, sheets of PGLA coated with cells and growth factors were used," said Mahajan, adding that different medication and treatments were imparted and closed dressing was avoided.

"Blood transfusion and supplementary fluids were given intravenously to maintain systemic homeostasis," said Mahajan.

Stating that on 5th and 6th day following treatment, dry scales from the face and body started peeling off, the doctor's team also observed impressive changes such as new skin forming within a week of treatment with cells and growth factors.

By conventional modalities, it takes more than eight weeks for the patient to heal and many additional months for the patient to be able to regain joint and facial movements.

"By the 10th day of the treatment, dry scales completely peeled off and by the 14th day the patient had no tenderness or burning pain. Joint movements became free as well, Steady rate of progression of healthy skin formation is being noticed. Areas with deep burns are also healing at a rapid rate and I am confident that within a month we will accomplish thorough healing and the patient will be back to normal," Mahajan said.

Medical sciences say that such cases are challenging to manage considering the degree of impairment they result in due to prolonged healing period. Also, through conventional therapeutic modalities healing occurs with scar formation and results in contractures. Chances of systemic complications and infection are also high.

However according to the medical team, by using stem cells, the natural healing potential of the body is used, leading to reduction of healing time and promoting regeneration of affected tissues. This also reduces the mental trauma and financial burden that a patient goes through when under conventional management.

"Stem cell-based therapy has offered a novel and powerful strategy in almost every medical specialty including burns and wound management. Stem cells have proven to have tremendous potential in enhancing wound healing and facilitating skin regeneration," Mahajan said.

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Four Ways to Younger Skin Right Now – Forbes


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Four Ways to Younger Skin Right Now
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Her Hydrating and Plumping Serum No1 combats the environmental stressors that skin faces every day to detoxify and rejuvenate the face and subsequently enacting anti-aging properties. By using plant-stem cells, hyaluronic acid, marine snail peptides ...

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We’re About to Enter a New Era in Parkinson’s Disease Treatments – Futurism

Before we get to the therapeutic stuff, here is a reminder of the main problem people with Parkinsons disease face.

Researchers are reasonably sure that the accumulation of a protein called alpha-synuclein is responsible for neurons dying in people with PD. However, there are two competing theories as to how it builds up andspreads,the threshold theoryandthe ascending theory(also called the prion hypothesis). The ascending theory states that alpha-synuclein spreads from cell to cell, infecting cells as the protein moves up through the brain.The threshold theory recently put forward by Dr. Ole Isacson and Dr. Simone Engelender, proposes that alpha-synuclein builds up independently in each affected cell.

Regardless, an improved understanding of exactly how such proteins misfold and clump together is at the heart of the riddle that is Parkinsons as well asa long list of other diseases. Thankfully a number of labs around the world have been working on this sticky problem. Additionally, if anyone wants to help you can do so very easily from any computer, watch this video to learn how.

The ongoing revolution in genetics is playing an increasingly important role in our understanding of the disease while also revealing whyit varies so much from patient to patient. There havebeen dozens of mutations and variants associated so far with the disease. We are just beginning to understand the role our genes play in the development of neurological diseases but an immense amount of progress has been made in the last 15 years since the human genome was sequenced. Now that sequencing costs have plummeted to around a thousand dollars we are on the verge of a new era in medicine that promises to give patients treatments tailored to their specific condition.

Personalized medicine is healthcare based on your unique genetic and molecular blueprint. Each individual has distinct genetic makeup, biomolecule and metabolic profiles, set of gut microbes, and so on. Similarly, there is no one-size-fit-all in healthcare. How you stay healthy or how you are treated for disease should be catered to match your unique profile. Knowledge of your genomics, proteomics, metabolomics, microbiotics, and other bioinformatics allow for the improvement in the quality of life, from disease prevention to therapy best suited to you. (from the Personalized Medicine Initiative in British Columbia.)

A better understanding ofgeneticswill help unlock a cascade of other problems that surround this disease includingmitochondrial dysfunction, lysosomal degradation, neuroinflammation,gut bacteria, andepigenetics, among others. And thankfully there is now a large interconnected global community of researchers working to solve these problems with more resources and better tools than in all of human history combined. This growth in a variety of public and private sector health initiatives across disciplines has lead a growing number of experts to believe that we will make more progress in the next decade than we did in the past century, which is good reason to be hopeful consideringwhat medicine was like a hundred years ago.

This medical revolution will be further bolstered by new and improved imaging techniques.A big part of the problem we still have with this disease is that we cant actually see what is wrong. Every person who has PDhas slightly different symptoms but we dont really know why primarily because we cant accurately see inside patients heads. Soon a new line of imaging techniques will be available that will give surgeons and researchers a much better understanding of what is going on inside the heads of each patient.

In addition, there are some immense ongoing collaborations such as theEuropean human brain projectand theU.S. brain initiativethat are trying to do for the brain what the human genome project did for our understanding of the genome. If successful it will give researchers unprecedented insight into how our minds are pieced together.

Then there are the new therapies themselves.

Levadopa For 50 years now this wonder drug has brought relief to millions. Of course, problems still persist, namely in getting it past that stubborn blood brain barrier and making sure a more steady supply is delivered to reduce on/off fluctuations. To get around some of those problems we now havepatches, slow release and extended release capsules, as well asintestinal pumps that deliver a steady flow of the drug directly into the intestines. Of course this drug is not an ideal solution as there are nasty side effects that come from long term use, predominantly dyskenisia which gives people the motor control of a blob of jelly, but for now, it is still the best stop-gap solution we have.

Deep Brain Stimulation This science-fiction wonder has become the undisputed Queen of modern treatments. It has already proven itself to be a miracle worker, re-animating hundreds of thousands with its electric wizardry. It too is steadily improving, from John Palfermans book,Brain Storms,Instead of implanting devices that simply deliver a continuous electrical stimulation, they are developing technologies that deliver stimulating jolts only when required. ..The idea is to design DBS so that the system can monitor the electrical activity in the basal ganglia, and when it detects an abnormal signal, it can respond automatically with an appropriate stimulation. A smart device

New Drugs There is along list of promising drugs that are already in clinical trial.Some of these drugs have the potential to not only offer symptomatic relief but hit the holy grail that is actual disease modifying therapies.

Neuromodulation techniques A number of novelneuromodulation techniques are being tested for clinical use. The most prevalent is called transcranialmagnetic stimulation in which magnets are attached to the outside of patients headsthat send a focused electric current deep into the target areas of the brain. Already an approved therapy for depression, TMS is now being tried in PD.

Immunotherapies The relatively recent identification of alpha-synuclein as playing a key role in disease formation has lead researchers to believe that we may be able to harness the bodies immune system to stop the protein from clumping while also mitigating the bodies natural inflammatory responses that damages neurons.

Pharmacogenetics The genetic revolutionhas spurred the development of a relatively new field of pharmacology called pharmacogenetics. Eventually, instead of making one drug for everybody, we will be able to tailor drugs to better fit each persons unique condition.

Stem Cell Therapies Though there were a series of trials in the 90s that had mixed results, recently a number of labs around the world have begun reexamining the therapeutic potential of stem cells. This is thanks in part to the 2007 discovery of anew type of stem cell called IPS cells which allow researchers to grow fully functioning stem cells from patients own skin cells. This has opened the door to a new set of therapies while also giving us better disease models. Since those first trials we have also made a series of other advances in our understanding of how to use stem cells which has lead to somestunning results in trials on other apes. Some labsare hoping to push forward with human trials starting at the end of this year.

Gene Modification Therapies As discussed earlier, the field of genetics is blowing up and one of the biggest benefits to society that will come from it is a new set of therapies called gene modification therapies.The most popular one today is called CRISPR, a technique that already allows researchers to cut and paste genetic code, changing the genome of living organisms. A number of articles have come out touting these kind of gene-editing techniques as the future of medicine. This first use ofCRISPRwas in a lung cancer patient in Chinalast fall, but it is also being used to help us understand neurodegenerative disordersincludingParkinsons disease.

Direct Programming In conjunction with gene therapy, direct programming is believed to bethe final solution to the problem of neurodegeneration. It is a subset of the new field of synthetic biologythatwill eventually allow us to change cell types in living organisms. For example, inpeople with Parkinsons disease we will be able toreprogram other healthy cells in the affected area, such as glial cells or astrocytes, and directly turn them into dopamine-producing cells.

When it comes right down to it, the reason why we have not been able to cure a lot of the diseases that are still with us today, such as neurodegeneration or cancer, is that there are an incredible number of factors to consider when trying to treat them, possibly too many for any human, or even any group of humans, to make sense of. But there might be a solution to this problem as we are now figuring out ways to export more and more of our intellectual abilities into computers. Already computers have become as good ashumans at diagnosing certain conditions, and astaggering number of healthcare companieshave now invested heavily in applyingartificial intelligence to the medical industry.This, along with further advances in nanotechnology,has a lot of potentialin helping us understand diseases such as Parkinsons and may reveal novel insights into how to treat them.

As you can see, there is plenty in the pipeline. While there may not be any magic bullet, there is no doubt that we will continue to see improvements in the treatment of Parkinsons disease that will benefit millions. While it is important to remain skeptical of all the promises being made, there is very good reason to believe that afflictions such as Parkinsons disease may one day be a thing of the past.

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Novel gene therapy experiment offers hope for people with certain hearing loss and dizziness disorder – Medical Xpress

March 23, 2017 This image shows stereocilia bundles on inner hair cells from whirler mice after whirlin gene therapy. These hair-like protrusions allow sensory hair cells to detect sound and motion. The whirler mutant mouse has very short stereocilia bundles. After whirlin gene therapy, the stereocilia bundles are increased to normal length (red) and whirlin expression is restored (green). Credit: Johns Hopkins Medicine

In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of Medicine showed that gene therapy was able to restore balance and hearing in genetically modified mice that mimic Usher Syndrome, a genetic condition in humans characterized by partial or total hearing loss, dizziness, and vision loss that worsens over time. The hearing loss and dizziness is caused by abnormalities of the inner ear.

Dizziness and hearing loss are among the most common disabilities affecting humans and can be severe and debilitating. According to the National Health and Nutrition Examination Survey, more than 35% of U.S. adults aged 40 years and older have some degree of balance dysfunction, a major cause of falls in the elderly. According to the Centers for Disease Control, approximately one in three people in the United States between the ages of 65 and 74 has hearing loss, and nearly half of those older than 75 have difficulty hearing. Men are more likely to experience hearing loss than women.

Primary investigator Wade Chien, M.D., a neurootologist and associate professor with the Johns Hopkins Otolaryngology-Head and Neck Surgery team who also practices at the Johns Hopkins Healthcare and Surgery Center in Suburban Hospital in Bethesda, MD., and his team administered gene therapy to the inner ears of genetically modified mice carrying a mutation in a gene which is associated Usher syndrome. These mutant mice are deaf and have significant balance problems from birth. After gene therapy administration, the balance function of the mutant mice was completely restored. In addition, these mutant also had improvement in hearing. This study was one of the first to show that gene therapy can be used to improve hearing and balance functions in a mouse model of hereditary hearing loss. This study was funded by the NIDCD intramural research program.

"Inner ear gene therapy offers tremendous potential as a new way to help patients with hearing loss and dizziness," Chien said.

While the positive results are striking the researchers caution that the results are preliminary and will require additional research in humans to demonstrate fully their utility in treating humans. However, they are optimistic that their data indicate that inner ear gene therapy hold promise for treating a variety of human inherited vestibular and hearing disorders, including Usher syndrome.

Explore further: Number of people in US with hearing loss expected to nearly double in coming decades

More information: Kevin Isgrig et al. Gene Therapy Restores Balance and Auditory Functions in a Mouse Model of Usher Syndrome, Molecular Therapy (2017). DOI: 10.1016/j.ymthe.2017.01.007

In a study published online by JAMA Otolaryngology-Head & Neck Surgery, Adele M. Goman, Ph.D., of Johns Hopkins University, Baltimore, Md., and colleagues used U.S. population projection estimates with current prevalence ...

In the summer of 2015, a team at Boston Children's Hospital and Harvard Medical School reported restoring rudimentary hearing in genetically deaf mice using gene therapy. Now the Boston Children's research team reports restoring ...

In a study published online by JAMA Otolaryngology-Head & Neck Surgery, Kathleen M. Schieffer, B.S., of the Pennsylvania State University College of Medicine, Hershey, Pa., and colleagues examined the association between ...

Severe hearing loss is the third most prevalent chronic condition in older Americans and more than 15% of people in their 30s are also affected. The condition leads to communication problems, social isolation, depression, ...

A new gene therapy approach can reverse hearing loss caused by a genetic defect in a mouse model of congenital deafness, according to a preclinical study published by Cell Press in the July 26 issue of the journal Neuron. ...

Using a novel form of gene therapy, scientists from Harvard Medical School and the Massachusetts General Hospital have managed to restore partial hearing and balance in mice born with a genetic condition that affects both.

Monash University researchers have discovered the mechanism underlying the fainting disorder, Postural Orthostatic Tachycardia Syndrome (POTS), the condition famously affecting the former lead singer of The Wiggles.

A person carrying variants of two particular genes could be almost three times more likely to develop multiple sclerosis, according to the latest findings from scientists at The University of Texas Medical Branch at Galveston ...

Researchers with the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health, and their collaborators, have successfully used facial recognition software to diagnose a rare, genetic disease ...

In a first-of-its-kind study published in the March 1, 2017 edition of Molecular Therapy, researchers from the National Institute on Deafness and Other Communication Disorders (NIDCD) and Johns Hopkins University School of ...

An international team of researchers from institutions around the world, including Baylor College of Medicine, has discovered that mutations of the OTUD6B gene result in a spectrum of physical and intellectual deficits. This ...

Researchers at Baylor College of Medicine, Texas Children's Hospital and Rice University have uncovered a gene mutation that may provide answers to unexplained female infertility. The study appears in Scientific Reports, ...

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Novel gene therapy experiment offers hope for people with certain hearing loss and dizziness disorder - Medical Xpress

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Cancer Gene Therapy Market Deep Research Study with Forecast by 2025 – MilTech

Albany, NY (SBWIRE) 03/24/2017 Global Cancer Gene Therapy Market: Overview

Cancer results from the multiple mutations in a single cell that makes it to proliferate out of control. Cancer cells invade new cellular territories, have a high metabolic rate, and an altered shape. The various methods to treat cancers are surgery, radiation, and chemotherapy. When the aforementioned therapies fail to achieve desired results, gene therapy is leveraged. Gene therapy involves the insertion of a functional gene, also known as therapeutic DNA, into the cells of a cancer patient to rectify the metabolism, to change or repair an acquired genetic abnormality, and to provide a new function to a cell. The two main types of gene therapy are germinal and somatic.

Global Cancer Gene Therapy Market: Key Trends

Majorly promoting the global cancer gene therapy market is the swift pace of technological breakthroughs and the growing popularity of emerging genomic technologies like next-generation sequencing and high-density DNA microarrays. Additionally, the government support for these technologies is also slated to stoke growth in the near future. The Center for Disease Control and Prevention (CDC), for example, supports screening programs for breast cancer control and cervical and colorectal cancers among low-income group women sans health insurance in the U.S.

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Besides, the rising occurrence of cancer worldwide is will substantially drive up demand for gene therapy in the years ahead. According to WHO, cases of cancer will likely touch US$15 million mark by the end of the decade.

Global Cancer Gene Therapy Market: Market Potential

At present, most of the cancer gene therapy products are in being tested. The market is predicted to grow once the trials bear results. An US pharmaceutical company named Kite Pharma, for example, recently revealed the results from the initial six months of the trial of a new gene therapy treatment called CAR-T cell therapy. It helped up patients own immune cells and has eliminated the disease from one third of terminal patients. Around 36 per cent of the 101 patients on the trial were still in complete remission at six months, and eight in 10 saw their cancer reduced by at least half during the study.

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Groundbreaking therapies such as this is slated to revolutionize the global cancer gene therapy market.

At present, adenoviral vector is a popular oncology application because of its effective nuclear mechanism and low pathogenicity. Adenoviral vectors are leveraged in gene replacement approaches, suicide gene, gene-based immunotherapy, and syndicate gene with chemotherapy. Retroviral vector-mediated gene transfer also plays a key role in the gene therapy industry for it brings about the crucial benefit of changing the single stranded RNA genome into a double stranded DNA molecule, which eventually integrates into the target cell genome.

Global Cancer Gene Therapy Market: Regional Outlook

North America and Europe are key regions in the global cancer gene therapy market on account of a massive elderly population and significant technological progress in the region. In the years ahead, however, the market is Asia Pacific is forecasted to surge on account of supportive government initiatives, improving economy, bettering healthcare infrastructure, and growing thrust on research and development.

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Global Cancer Gene Therapy Market: Competitive Analysis

Some of the prominent players in the global cancer gene therapy market are Altor Bioscience Corporation, SiBiono., Shanghai Sunway Biotech company Limited, BioCancell, GlobeImmune, Inc.,Aduro Biotech, OncoGeneX, New Link Genetics., ZioPharm Oncology, and GENELUX. At present the market is led by small pioneering biotech firms who may eventually collaborate with prominent players for clinical development or commercialization of products.

About TMR Research TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

Our savvy custom-built reports span a gamut of industries such as pharmaceuticals, chemicals and metals, food and beverages, and technology and media, among others. With actionable insights uncovered through in-depth research of the market, we try to bring about game-changing success for our clients.

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Cancer Gene Therapy Market Deep Research Study with Forecast by 2025 - MilTech

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JAMA confirms DTC drove ‘Low-T’ craze – BioPharma Dive

Dive Brief:

"Low-T" was not a common term before 2009, but direct-to-consumer advertising by the makers of testosterone medications made it a household condition, despite there being little evidence of an actual problem.

Testosterone gels and creams are typically approved for men with pathological hypogonadism, a low hormone condition that can arise after chemotherapy for prostate cancer. Yet, the pharmaceutical industry spent years advertising the products to treat an unsubstantiated "lifestyle" condition for men who werent feeling quite "manly" or had a low sex drive, despite little to no safety or efficacy data supporting these claims.

"Advertising intensity varied by geographic region and time, with the highest intensity seen in the southeastern United States and with months ranging from no ad exposures to a mean of 13.6 exposures per household," noted the JAMA report. "Non-branded advertisements were common prior to 2012, with branded advertisements becoming more common during and after 2012. Each household advertisement exposure was associated with a monthly increase in rates of new testosterone testing."

The "Low-T" craze became an exercise in how pharma companies can abuse the direct-to-consumer advertising paradigm. DTC ads are common across therapeutics areas in the U.S., but generally not allowed in other countries. The practice of advertising drugs directly to patients has long been criticized by those within and outside the industry.

AbbVie precursor Abbott Laboratories acquired AndroGel, a major player in the low-T segment, in 2010 from Solvay Pharmaceuticals. The commercialization engine at AbbVie then began promoting the drug. By 2013, AndroGel became AbbVies best-selling drug behind its blockbuster rheumatoid arthritis treatment Humira, with more than $1 billion in sales. The drug had grown by more than 55% during the course of 2012 alone, largely driven by the off-label use in "Low-T" patients.

AndroGel was the dominant player in this space until the Food and Drug Administration began cracking down on the treatments. In 2016, the FDA issued its third warning in three years about the testosterone category, reporting that abuse of the treatments could cause heart attack, heart failure, stroke, depression, hostility, aggression, liver toxicity, and male infertility.

The first of the product liability suits for AndroGel are expected to begin in June of this year, alleging that the company did not adequately warn of the risks of the drug and promoted it off-label for the use of "Low-T."

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JAMA confirms DTC drove 'Low-T' craze - BioPharma Dive

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Pathologists and Clinical Laboratories May Soon Have a Test for Identifying Cardiac Patients at Risk from Specific … – DARKDaily.com – Laboratory…

Published: March 22 2017

Stanford University School of Medicine researchers grew heart muscle cells and used them, along with CRISPR, to predict whether a patient would benefit or experience bad side effects to specific therapeutic drugs

What would it mean to pathology groups if they could grow heart cells that mimicked a cardiac patients own cells? What if clinical laboratories could determine in vitro, using grown cells, if specific patients would have positive or negative reactions to specific heart drugs before they were prescribed the drug? How would that impact the pathology and medical laboratory industries?

We may soon know. Researchers at Stanford University School of Medicine (Stanford) have begun to answer these questions.

May Be Feasible for Clinical Laboratories to Use Pluripotent Stem Cells for Assays

In a Stanford press release, researchers stated that induced pluripotent stem cells (iPS cells), coupled with CRISPRtechnology, could be used to determine:

1) Whether a patient would benefit from a specific therapeutic drug; and

2) The likelihood that the patient might have a negative reaction or bad side effect from that drug.

Thirty percent of drugs in clinical trials are eventually withdrawn due to safety concerns, which often involve adverse cardiac effects. This study shows that these cells serve as a functional readout to predict how a patients heart might respond to particular drug treatments and identify those who should avoid certain treatments, said Joseph Wu, MD, PhD, in the Stanford press release. Wu is Director of Stanfords Cardiovascular Institute and a Professor of Cardiovascular Medicine and Radiology.

The researchers believe their discovery could become a form of diagnostic and prognostic testing performed by pathologists and clinical laboratories if it passes further clinical trials.

Heart Muscle Made from Stem Cells, Study Advances Precision Medicine

The iPS cells are stem cells created in a lab, usually from a persons skin sample, and then induced into becoming cells from other parts of the body. Heart muscle cells made from iPS cells mirror the expression patterns of key genes in the donors native heart tissue. This means the cells can be leveraged to predict a patients likelihood of experiencing drug-related heart damage, according to the Stanford release.

The Stanford study also advanced precision medicine. It combined genetics, large-scale data research, and individualized testing to determine the best treatments for patients, noted an article in United Press International (UPI).

Researchers were motivated by a need to understand individual susceptibility to drug-induced cardiotoxicity, to improve patient safety, and to prevent drug attrition, according to the Stanford study, which was published in the research journal Cell Stem Cell.

Human iPS cells enable the study of pharmacological and toxicological responses in patient-specific cardiomyocytes and may serve as preclinical platforms for precision medicine, the authors noted in the study summary.

Furthermore, the researchers idea could have implications for medical conditions beyond cardiomyopathy, noted an article in LabRoots.

Cardiomyopathy is a disease of the heart muscle that affects millions of people worldwide each year.

Joseph Wu, MD, PhD (above left), and Elena Matsa, PhD (above right), both with Stanford University School of Medicine, led a team of researchers who published a study involving CRISPR that suggests heart muscle cells made from induced pluripotent stem cells (iPS cells) could be used to identify cardiac patients who could benefit from or who could be damaged by certain cardiac medications. (Photo credits: Stanford University.)

Testing Tissues in the Stanford University Research Lab

Heres how the research progressed, according to the Stanford press release:

Matsa, Wu, and their colleagues created heart muscle cells, or cardiomyocytes, from iPS cells taken from seven people not known to be genetically predisposed to cardiac problems;

They sequenced the RNA molecules made by the heart muscle cells to learn which proteins the cells were making, and by how much;

They then compared the results within individualslooking at the gene expression patterns of cardiomyocytes derived from several batches of iPS cells from each personas well as among all seven study subjects.

They also investigated how the cardiomyocytes from each person responded to increasing amounts of two drugs: Rosiglitazone (marketed as Avandia by GlaxoSmithKline), which is sometimes used to treat Type 2 diabetes; and Tacrolimus (marketed as Prograf by Astellas Pharma), which serves as an immunosuppressant to inhibit the rejection of transplanted organs. Each of the two drugs has been associated with adverse cardiac effects in some people, but it has not been possible to predict which patients will experience heart damage.

Gene expression patterns of the iPS cell-derived cardiomyocytes from each individual patient correlated very well, said Elena Matsa, PhD, Stanford Instructor, Cardiovascular Institute, and the studys lead author. But there was marked variability among the seven people, particularly in genes involved in metabolism and stress responses. In fact, one of our subjects exhibited a very abnormal expression of genes in a key metabolic pathway.

Gene Editing Reveals Drug Response Information

Enter the Clustered Regularly Interspaced Short Palindromic Repeats, or CRISPR (pronounced crisper), gene editing technology. CRISPR technology has advanced the study and practice of genetic medicine.

Researchers could not pinpoint a specific gene mutation responsible for abnormal cardiomyocyte response. But they did identify a metabolic pathway that influenced Rosiglitazones response.

They corrected the abnormality using CRISPR-Cas9 (a simplified version of the CRISPR/Cas system). This genome editing technique enables researchers to edit parts of the genome by removing or changing in some manner the DNAsequence, according to yourgenome, an information website dedicated solely to DNA, genes, and genomes.

The results? The Stanford researchers reported boosting a gene expression in the pathway, restoring normal function, and prompting a response to Rosiglitazone that was consistent to that of the other subjects cardiomyocytes.

Clinical Laboratories Become Even More Integral to Cardiac Diagnosis and Treatment

Can iPS-derived cardiomyocytes reliably replicate human heart tissue? Researchers were not sure. So, they created iPS cells from another three people who had heart biopsies or transplants. They then compared the cells made in the clinical laboratory with the gene native cells and found that they were similar in many significant ways.

In the end, cardiomyocytes derived from human iPS cells correlated with patient participants in the Stanford study. And, most importantly, the study revealed a potential ability to test drugs for adverse reactions and improve treatment for millions of people with cardiomyopathy. Should additional research confirm these findings, it could provide medical laboratories with a new approach to improving diagnosis and therapeutic selection for patients with heart disease.

Donna Marie Pocius

Related Information:

Heart Muscle Grown from Stem Cells May Help Doctors Test Treatments

Heart Muscle Made from Stem Cells Aids Precision Cardiovascular Medicine

Transcriptome Profiling of Patient-Specific Human iPSC-Cardiomyocytes Predicts Individual Drug Safety and Efficacy Responses in Vitro

Heart Stem Cells for Individualized Medicine in Cardiology

Stem Cells Create Faithful Replicas of Native Tissues, According to Stanford Study

CRISPR/Cas9 and Targeted Genome Editing: A New Era in Molecular Biology

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Pathologists and Clinical Laboratories May Soon Have a Test for Identifying Cardiac Patients at Risk from Specific ... - DARKDaily.com - Laboratory...

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Old blood can be made young again and it might fight ageing | New … – New Scientist

Fresh young cells

Dennis Kunkle Microscopy/Science Photo Library

By Jessica Hamzelou

BLOOD from the young seems to have healing powers, but how can we harness them without relying on donors? The discovery of a protein that keeps blood stem cells youthful might help.

The rejuvenating properties of young blood came to light in macabre experiments that stitched young and old mice together to share a circulatory system. The health of the older mice improved, while that of the younger ones deteriorated. Other animal studies have since shown that injections of young or old blood have similar effects.

This may work in people too. Young blood is being trialled as a treatment for conditions like Alzheimers, and aged mice that received injections of blood from human teenagers showed improved cognition, memory and physical activity levels.

We think the drug will improve signs of ageing and boost the immune systems of older people

But these studies rely on young people donating their blood: if this became the go-to therapy for age-related disease it would be difficult to get enough donations to fulfil demand.

The stem cells in our blood could provide an alternative approach. Our red and white blood cells are made by stem cells that themselves come from mother stem cells in bone marrow. But as we age, the number of these mother stem cells declines. One of the worlds longest-lived women seemed to only have two left in her blood when she died at age 115.

The decline in mother stem cells causes people to have fewer red blood cells, and white blood cells called B and T lymphocytes. These declines can cause anaemia and weaken the immune system. Usually the immune system in the elderly is not prepared to fight infections very hard, says Hartmut Geiger at the University of Ulm in Germany.

When Geigers team examined the bone marrow in mice, they found that older animals have much lower levels of a protein called osteopontin. To see if this protein has an effect on blood stem cells, the team injected stem cells into mice that lacked osteopontin and found that the cells rapidly aged.

But when older stem cells were mixed in a dish with osteopontin and a protein that activates it, they began to produce white blood cells just as young stem cells do. This suggests osteopontin makes stem cells behave more youthfully (EMBO Journal, doi.org/b4jp). If we can translate this into a treatment, we can make old blood young again, Geiger says.

Its exciting, says Hanadie Yousef at Stanford University in California. But longer term studies are needed to see whether this approach can rejuvenate the whole blood system, she says.

Until now, most efforts to use blood as a rejuvenation agent have focused on plasma, the liquid component, as some believe it carries dissolved factors that help maintain youth. But Geiger thinks the cells in blood might play a key role, because they are better able to move into the bodys tissues.

Both soluble factors and blood cells are likely to be important, says Yousef. While injections of young plasma rejuvenate older animals, the treatment doesnt have as strong an effect as when young and old animals share a circulatory system, she says.

Geigers team is developing a drug containing osteopontin and the activating protein to encourage blood stem cells to behave more youthfully. It should boost the immune system of elderly people, he says.

Such a drug might have benefits beyond fighting infection and alleviating anaemia. The team also think the protein will boost levels of mother stem cells. Having only a small number of such cells has been linked to heart disease, so Geiger says there is a chance that boosting them may help prevent this.

Osteopontin might also be useful for treating age-linked blood disorders, such as myelodysplasias that involve dysfunctional cells, says Martin Pera of the Jackson Laboratory in Bar Harbor, Maine. It is possible that rejuvenating bone marrow stem cells could help with these conditions, he says.

This study provides more evidence that cells can be rejuvenated, says Ioakim Spyridopoulos at Newcastle University, UK. They have made old blood look young again, although whether it acts young or not will have to be shown in clinical trials.

This article appeared in print under the headline Old blood made young again

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Old blood can be made young again and it might fight ageing | New ... - New Scientist

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‘Butterfly boy’ Jonathan Pitre cleared for second stem cell transplant – Ottawa Citizen

Jonathan Pitre readies for his second stem cell transplant, which will take place April 13th at the University of Minnesota Masonic Children's Hospital. Tina Boileau / -

Fully recovered from a series of infections, Jonathan Pitre has received medical clearance to undergo a second stem cell transplant.

Pitre, 16, will check into hospital on the last day of March to begin eight days of high-dose chemotherapy and one day of radiation. His stem cell transplant what doctors call Day Zero is scheduled forApril 13 at the University of Minnesota Masonic Childrens Hospital.

The night before he goes into hospital, Pitre will attend the Ottawa Senators game against the Minnesota Wild at the Xcel Energy Centre in Saint Paul. It will be a good night of fun before it all starts again, said Pitres mother, Tina Boileau.

She shared the latest news on her Facebook page on Wednesday.

After many weeks of tests, procedures and appointments at the hospital, Jonathan got the green light to proceed with the second transplant, she said. He has completely recovered from his infections and his body is as strong as can be This time it will work!

Last September, Pitre suffered nausea, hair loss, fevers and exhaustion in the aftermath of his first transplant, which ultimately failed when his own stem cells recolonized his bone marrow.His second transplant has been delayed because of lung and blood infections.

In an interview earlier this month, Pitre told the Citizen hes staying positive even though he understands the physical test that he faces in hospital.

Its mostly thinking about sticking together with the people you care about, your family, he said . You have to stick to them very, very tightly and tell each other that, Its going to be OK, and that Were stronger than this. Were going through this together, not just alone.

Pitre suffers from a rare, painful and deadly form of epidermolysis bullosa (EB), a blistering skin disease.

Hes the first Canadian to take part in a clinical trial operated by the University of Minnesotas Dr. Jakub Tolar, a pediatric transplant specialist who has adapted stem-cell therapy as a treatment for the most severe forms of EB.Although the procedure comes with the potential for life-threatening complications, it has produced dramatic improvements in two-thirds of those EB patients who have survived the transplant: tougher skin, reduced blistering and better wound healing.

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'Butterfly boy' Jonathan Pitre cleared for second stem cell transplant - Ottawa Citizen

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Plasma and stem cells: The future of regenerative medicine | WEAR – WEAR

Plasma and stem cells: The future of regenerative medicine

Blood platelet injections and stem cell treatments may sound like the future, but physicians at the Andrews Institute are already practicing these forms of regenerative medicine.

Weight lifting mixed with normal wear and tear left Howie Webber in constant pain.

"I probably felt it about four months ago," said Howie. "I did some stretching, thinking I could make it go away, but it just continued to get worse."

That's when Howie went to the doctor and found out he had two options: surgery or regenerative medicine; he picked the latter.

"I just added up the amount of time I'd be out of work and the cost of surgery, plus the copay and this whole thing just seemed like it would be a little faster and a little easier, and it ended up being just that," said Howie.

Physicians at the Andrew's Institute currently offer two different types of regenerative medicine, platelet rich plasma, or PRP and bone marrow aspirate concentrate, or BMAC.

With PRP, physicians take the patient's blood, separate the platelets and inject those platelets back into the patient at the site of injury. The idea is that platelets carry growth factors and molecules to stimulate the healing process.

BMAC utilizes platelets too, but also the patient's bone marrow harvested from the pelvis.

Both regenerative medicine methods have benefits, perhaps the biggest according to Dr. Brett Kindle, is avoiding invasive surgeries.

"If we need surgery, we need surgery, and that's what it is, but if we can avoid it, that often times is very beneficial from a financial standpoint, missing less work, etc.," said Dr. Kindle. "Also from a quality of life, to be able to get back to doing activities in a more timely manner."

The main difference between the two is price and neither are covered by insurance. BMAC costs upwards of $3,000, while PRP costs anywhere from $600 to $800. Howie opted for PRP.

"It hurt for about three days, then within a week I was pain free," said Howie. "Maybe a little discomfort that you would expect, but it wasn't near as bad as it was before."

Howie's issue was with his hamstrings, but Dr. Kindle said both PRP and BMAC can be used to treat a variety of aches and pains.

"Anything in the limbs," said Dr. Kindle. "Shoulders, elbows, hands, wrists, hips, knees, foot, ankle, all of those areas."

Recovery for both PRP and BMAC procedures is typically one to two weeks. Full effects of the injections don't usually kick in until six to eight weeks later. For more information about regenerative medicine or to schedule a consultation with an Andrews Institute physician, call (850) 916-8700.

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Plasma and stem cells: The future of regenerative medicine | WEAR - WEAR

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