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

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

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Smaller bone size less bone turnover in men with hypogonadism type 2 diabetes – Healio

Smaller bone size less bone turnover in men with hypogonadism type 2 diabetes
Healio
Men with hypogonadism and type 2 diabetes have a higher bone mineral density, but smaller bone area and lower bone turnover rate compared with men with hypogonadism but without diabetes, according to findings from a cross-sectional study.

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

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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

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

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

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

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Scientists May Have Found A Way To Make Old Stem Cells Act … – Digital Trends

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Scientists May Have Found A Way To Make Old Stem Cells Act … – Digital Trends

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

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Four Ways to Younger Skin Right Now – Forbes


Forbes
Four Ways to Younger Skin Right Now
Forbes
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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Four Ways to Younger Skin Right Now – Forbes

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

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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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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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Skin cells provide a new weapon against brain tumors – Blasting News

Scientists claim that #Stem Cells obtained from skin provide a new weapon against brain tumors. Jedd Wolchok, a cancer immunotherapy expert at the Memorial Sloan Kettering Cancer Center, says that nanoparticles are thinner than a human hair, and help to fight tumors. Previously, doctors used stem cells to target breast cancer tumors. Latest clinical trials show that the new therapy is useful for patients with brain tumors. According to a study published in the journal “Science Translational Medicine,” the treatment shrinks the tumors and extends the survival of victims.

Researcher says that it’s time to forget about drugs that spur the immune system to fight tumors. Stem cells will be used on a large scale to treat patients. Every year, pharmaceutical companies develop a number of antibodies and proteins that block the overexpressed molecules, enabling the immune system to target tumors. All these medicines are harmful to the nervous system. In contrast, the stem cells directly target a tumor without damaging the neurons. Jedd Wolchok believes that the current anti-cancer drugs work in only 10% to 40% of patients. There is no use of drugs that target only several cells of a tumor and fail to completely destroy it. Stem cells destroy a tumor within a few minutes. However, the process is very complicated and only experienced neurosurgeons should perform an operation. Once a patient receives radiation therapy to shrink a tumor, his immune system mounts an aggressive response that wipes out both the tumors and metastases throughout the body.

Jedd Wolchok will find out whether it is possible to use nontoxic nanoparticles to sensitize the immune system or not. He requires more time and further research before he publishes his findings. He says that it is not easy to pass the nanoparticles through the tumors as the particles are bigger than macrophages. However, specific blood proteins can be used to coat the nanoparticles, facilitating their uptake. Once these particles reach the brain tumor, they act as tumor killers. Jedd and his team will carry out an experiment on mice with breast cancer. Wolchok builds his study on an earlier discovery that brain stem cells have a weird affinity for cancers. #Beat The Clock

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Sun Exposure Is No Joke. You Need to Get Your Skin Checked ASAP – Reader’s Digest

Elena-Rudakova/Shutterstock

Twice a year, I strip down to my underwear, don a paper gown and subject myself to a full-body examination at the dermatologists office. These are done twice as often as most other patientsand for good reason. Not only am I freckly and fair-skinned, Ive had an unhealthy relationship with the sun, which makes me more susceptible to skin cancer.

During my teens and 20s, when I was a lifeguard and camp counselor, I spent the majority of my summers outdoors. Like my peers, Id wanted to achieve the perfect tan. Id worn sunscreen, but it was SPF 4barely any protection, compared with what doctors recommend today.

Now, Im paying the price. This past decade, Ive had a handful of suspicious-looking moles removed. Recently, my dermatologist sent me to a medical photographer for a full-body photo session to document my moles, in case they change.

wavebreakmedia/Shutterstock

My situation isnt unique. Countless people worldwide didnt protect themselves adequately from the suns ultraviolet rays during their youth. Decades ago, doctors didnt preach about sun protection, and researchers didnt realize that the suns ultraviolet rays could cause skin changes that can lead to melanoma, the deadliest form of skin cancer.

The most important reason for the increase in melanomas is thought to be due to increased exposure to ultraviolet radiation from sun and artificial tanning sources, says John J. DiGiovanna, staff clinician in the dermatology branch of the National Cancer Institutes Center for Cancer Research in Bethesda, Maryland.

Melanoma is only the ninth most commonly diagnosed cancer across Europe, but its rates have been rising sharply since the 1980s, six-fold among some groups.

Every year, 100,000 new cases of melanoma are diagnosed in Europe, says John Haanen, head of medical oncology at the Netherlands Cancer Institute in Amsterdam. Caucasians are at greatest risk, especially those with fair skin, red hair and freckles. Risk rises after age 40especially sun worshippers. Many experts refer to the increased prevalence as an epidemic.

I would not call it a melanoma epidemic but a skin cancer epidemic, says Reinhard Dummer, director of the Skin Cancer Centre at University Hospital Zrich. We expect in Switzerland that one out of five persons will develop skin cancers once in their lives.

Cultural changes over several decades are likely to blame. Bathing suits have gotten skimpier, and seaside vacations have become more common, exposing pale office workers to intense sunlight for short periods.

In Europe, low-cost air travel has increased the ability for people to travel to sunny, warmer climates for a week here and there, says Alex Menzies, medical oncologist at Melanoma Institute Australia, the country with the highest melanoma rates in the world. Intermittent exposure to the sun with burning is a major risk for melanoma.

Even if youve endured decades worth of sun exposure, there is hope.

The earlier you notice melanoma, the greater your chances are of being cured. Surgery is the primary treatment. If you picked up an early-changing mole, you could have a virtually normal life expectancy, says Girish Patel, lead investigator for the Skin Cancer Stem Cell Research Program at Cardiff University.

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Regular skin checks and meaningful lifestyle changes to limit further damage from the sun help improve the odds. Since Imogen Cheese, 37, of Gloucestershire, England, was diagnosed with stage II melanoma in 2013, shes screened by her medical team every three months. I cover up to avoid the midday sun, says Imogen. I wear high factor SPF, I am active and eat a healthy balanced diet. So far, her cancer has not progressed.

Researchers have made great strides in the treatment of advanced melanoma. One option: Targeted therapy, which can be given to stage IV patients with specific genetic mutations.

Melanoma researchers in Australia have been involved with targeted therapy research since the beginning, about seven years ago. We do testing on their tumors to look if there are any mutations in certain genes in the tumor, says Menzies. We have targeted therapy that can attack the BRAF mutation, which is found in about 50 percent of tumors from patients. If we give tablets for BRAF-mutant melanoma, almost every patient will have shrinkage of the tumor. On average, it will keep things under control for one year, and the one-year survival rate has improved to 70 percent, from 30 percent five years ago.

Five years after John Ambrose, 67, of New South Wales, Australia, had a grade IV skin melanoma removed he began coughing up blood. His disease had spread to both lungs and his prognosis was poor. He joined a targeted therapy clinical trial in 2013, and within three months, his tumors shrank by 70 percent. After 18 months, he had clear scans. Today, John travels, plays golf and spends time with his grandchildren.

My situation has not stopped me living a normal life, he says.

Texas native Jesse Thomas, 57, also benefited from targeted therapy after being diagnosed with stage IV melanoma in 2013, with tumors on his neck, liver and spine. Genomic testing revealed Jesse had an uncommon V600K BRAF mutation, and his oncologist was able to pinpoint a targeted therapy for him.

They expected the cancer to stop growing, but it actually shrank, Jesse says. Theres no way to cure it, but I am controllable.

Targeted therapy is only for stage IV patients, but researchers are studying its effects on stage III patients. We should know within a couple of years whether these treatments are beneficial, says John Haanen.

Researchers have been able to stimulate the T-cells in some melanoma patients immune systems to fight cancer, with astounding results.

T-cells kill off viruses and other things, Menzies says, but with cancer, theyre sitting there around the tumor, asleep. They know that the tumor is foreign, but the tumor has turned them off, stopping them from killing it. Immunology drugs turn on the T-cells and they kill the tumor.

Melanoma researchers consider immunology the biggest breakthrough in decades.

This is our penicillin moment in oncology, Menzies says. Melanoma can be turned into a chronic disease, and many people will not die from it in the near future if we continue to go the way were going.

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Immunotherapy doesnt work for everyone, but it can be quite effective. Cardiff Universitys Patel says, In the 45 or so percent of people who respond, they can respond for very long periods of time.

In 2013, Cardiff resident Vicky Brown, 62, was shocked to learn that a lump in her breast was actually melanoma, not breast cancer. Shed had early-stage melanoma in 2006, which returned in her breast and lungs.

Through a clinical trial, Brown received intravenous doses of two immunotherapy drugs. Within weeks, her tumors shrank. She discontinued the drugs due to side effects, but it kept the melanoma in check for a year. In 2015, after new lung tumors appeared, she received more immunology treatments. The drugs again shrank her tumors.

I am hoping this couple of doses will give me more time again, Vicky says. My grandson is now nine months old. I want to be able to make memories for him, as well as my four-year-old granddaughter.

Researchers are working to get more patients to have a positive response to the treatment. The notion is that clearly, if we can do it in a few, we should be able to do it in the majority, says Patel.

For years, researchers tried creating a melanoma vaccine, to no avail. Now, researchers are combining the success of immunotherapy with the concept of vaccines, leading to personalized melanoma treatments.

As we better understand how the immune system recognizes the melanoma cells, we are developing so-called personalized vaccines, Haanen says. We are starting now in metastatic patients and if this concept works well move to earlier stages.

Hein Jambroers, 50, of Roermond, Netherlands, has benefited from a personalized treatment called adoptive cell therapy (ACT). He was diagnosed with stage II melanoma in 2009, but a year later, he had stage IV disease, with tumors on his right leg and liver, and was told that he had less than six months to live.

After getting some short-term benefit from targeted therapy, Hein was referred to an ACT clinical trial in 2011. Doctors at the Netherlands Cancer Institute harvested some of his white blood cells, then monitored them in a laboratory to identify the healthiest T-cells to fight melanoma. They were replicated in large numbers. Hein received chemotherapy to kill his existing T-cells, then got an infusion of the laboratory-created T-cells, which basically gave him a new immune system that shrank his tumors within three months.

Hes what doctors call a complete responder. Hes had clean scans ever since; no trace of melanoma.

Complete responders have an excellent prognosis, says Haanen, who treated Hein. Cure is always difficult to say, but very long-term remissions which could be cureare seen in the majority of complete responders and in some partial responders.

Hein, who expected to die, is cautiously optimistic. Im very positive about my future, but Im always on a state of alert, he says. I sit in the shade. I cream up with sunscreen. I even do it for my child and my wife. I dont want to tempt the fates.

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Soon, doctors may defeat cancer by attacking stem cells.

Skin stem cells make thousands of healthy skin cells. Melanoma stem cells work similarly, except they make thousands of malignant melanoma cells. Researchers are targeting melanoma stem cells to stop tumors from spreading.

Its like killing off the queen bee, Patel says. The whole hive then dies away, because youve gotten to the cell thats giving rise to everything.

This is vastly different from chemotherapy, which aims to kill as much cancer as possible. Stem cells make up only one to three per cent of some skin cancers.

If you got rid of the cancer stem cell population, the whole tumor could not proliferate, Patel says. If you take the bulk of a tumor and regrow it in a mouse without stem cells, it cant form. But if you take a small part of the cancer stem cell population, it grows back fully.

Researchers have begun clinical trials, and treatments could be available in a decade.

Despite sun damage that I endured during my youth, Im optimistic that Im doing everything that I can to stay ahead of any problems that may crop up. Ive got photos of all of my moles and freckles now, which I use for monthly self-exams. Ill bring them to my dermatologist for my next full-body examination. Ive also been raising my children with 21st century values for sun exposureplenty of high-SPF sunscreen, hats and time in the shadeso hopefully the next generation wont have the melanoma worries that my generation does.

If youve been diagnosed with advanced melanoma, heres what patient advocates recommend:

See a specialist

Seek a facility where doctors specialize in melanoma. Our recommendation for patients is to get into a melanoma center of excellence, says Bettina Ryll, founder of Melanoma Patient Network Europe in Uppsala, Sweden. The new immunotherapies have very different side effects from anything weve ever had before, so you dont want to have a physician who has never seen this.

Consider a clinical trial

Availability of immunotherapy and targeted therapy varies in Europe. Cost is a factor in many countries. Many patients enter clinical trials to receive these drugs. A promising clinical trial may be farther from home than youd prefer, but the extra drive could be worth it. Rory Bernard, 47, of Clermont-Ferrand, France, travels four hours to Paris for targeted therapy treatments, which have shrunk his tumors and extended his life. The dermatologist said, If you stay here, youre dead in six months, says Rorys wife, Gilly Spurrier-Bernard, founder of Melanoma France. My aim is to inform patients that if they want to get the best treatment, they may need to move around. Translation translation transl translation translation transl translation translation.

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A two-step method to make microglia – Nature.com

A two-step method to make microglia
Nature.com
Microglia have been reported in some disease models to have beneficial effects; however, research into their potential as a cell therapy is limited by the lack of means to produce readily grafted, autologous microglial cells. Now, in Nature

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Ageing is a disease. Gene therapy could be the ‘cure’ – Wired.co.uk

Leon Csernohlavek

In September 2015, Elizabeth Parrish flew from Seattle to Colombia to receive an experimental treatment.

She had spent more than two years studying literature, talking to experts, and had decided to undergo gene therapy a treatment for genetic disorders that adds genes into cells to replace those that are faulty or absent. She ordered the therapeutic cells months in advance and arranged for a technician to administer the therapy in a clean room within a short distance of a hospital, in case she suffered a bad immune response. The gene therapy was shipped in a closed container and administered via an IV over approximately five hours. Parrish remained under observation for a few days and then flew home.

Was I anxious afterwards? Yes, Parrish says. I was definitely looking for indications that anything was wrong with my body. I was acutely aware of every ache and pain. She had become the first person to subject herself to gene therapy for the disease that affected her. Her condition? Ageing.

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In January 2013, Liz Parrish son was diagnosed with Type 1 diabetes. Every few days, he would have some devastatingly low blood sugar levels, Parrish says. I was continually reminded that we as humans spend a lot of time trying to pretend as if our death is not eminent. She remembers being told that her son was lucky because diabetes was treatable. I was really hit hard by the time I spent in children’s’ hospitals, Parrish says. She had read about the promises of modern medicine, in particular, gene therapy. I began trying to figure out why nothing was translating to hospitals where kids were dying.

Parrish began attending medical conferences on her own. I found this conference in Cambridge that looked to be about genetics, Parrish says. It turned out to be about longevity. There she learned how gene modifications had already extended the normal lifespan of worms up to 11 times and of mice by five times. It made me realise that if ageing was a disease and everyone was suffering from an illness, the fastest way to fund this research would be to essentially educate the world that was the case and get them to put money behind finding a cure, Parrish says.

At that point, Parrish, who up until then had been working part-time for software companies, started her own company, BioViva, to expedite therapeutics and give access to patients. Why did so many patients have to wait, suffer and die? Parrish asks. We became so risk adverse that patients die waiting for treatment. We have to change that drastically. We have millions of terminally ill patients on the planet right now. These patients should have access to the most promising therapeutics that don’t have a myriad of off-target effects. There is no artificial intelligence or meta analysis of these therapies that is going to replace what happens in the human body. And we let people die because we’re so concerned that a therapy might kill them. This is lawyering at its absolute worst.

Parrish then made another decision: she was going to try the first therapy on herself. I believed it was the most responsible and ethical thing to do. I believed the company should take its own medicine first before moving onto patients.

Parrish tried two therapies. One was a myostatin inhibitor, a drug designed to increase muscle mass, and the second was telomerase therapy, which lengthens the telomeres, a part of the chromosomes that protect genetic material from damage and allows the replication of DNA. Lengthening the telomeres can, at least in theory, extend cellular lifespan and make cells more resilient to damage.

The telomerase therapy had reversed ageing and extended lifespan in mice, Parrish says. I assumed this was the most promising therapy ever, and it was just sitting in research and wasn’t moving forward as a viable option due to what appeared to be patenting issues and a lot of academics sitting on the fence bickering. We will never know unless we get it in humans. It’s almost a moot point to try to continue to argue whether it works or not if we never use it. Its just like lemmings walking off the cliff, waiting for someone else to solve the problems.

A few weeks after the treatment, Parrish undertook follow-up exams, conducted by independent third parties. Her telomeres in her white blood cells had lengthened by more than 600 base pairs which, according to Parrish, implies they had extended by the equivalent of 20 years. A full-body MRI imaging revealed an increase in muscle mass and reduction in intramuscular fat. Other tests indicate Parrish now has improved insulin sensitivity and reduced inflammation levels.

The company was built essentially to prove these therapies work or not, Parrish says. Remember BioViva is not a research organisation. We are taking things like gene therapies and using them like technology. We would like to create an open market where people have access to acquiring these technologies, much like you would acquire a cellphone or a computer.

Further tests are being conducted at George Churchs lab in Harvard. Parrish and her team are currently working with other hospital clinics around the world to conduct more safety and feasibility studies in human subjects. I had already put things into perspective that without medicine, my son would be dead and he really was the meaning of my life, Parrish says. I was a person who quite honestly felt I had not really contributed that much to society and this was my opportunity to do so.

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Gene therapy: What personalized medicine means for you – CNET – CNET

Thuy Truong thought her aching back was just a pulled muscle from working out. But then came a high fever that wouldn’t go away during a visit to Vietnam. When a friend insisted Truong, 30, go to an emergency room, doctors told her the last thing she expected to hear: She had lung cancer. Back in Los Angeles, Truong learned the cancer was at stage 4 and she had about eight months to live.

“My whole world was flipped upside down,” says Truong, who had been splitting her time between the San Francisco Bay Area and Asia for a new project after selling her startup. “I’ve been a successful entrepreneur, but I’m not married. I don’t have kids yet. [The diagnosis] was devastating.”

Doctors at the University of Southern California took a blood sample for genetic testing. The “liquid biopsy” was able to detect tumor cells in her blood, sparing her the risky procedure of collecting cells in her lungs.

Genetic sequencing allowed the lab to isolate the mutation that caused her cancer to produce too much of the EGFR (epidermal growth factor receptor) protein, triggering cancer cells to grow and proliferate. Fortunately, her type of mutation responds to EGFR-targeting drugs, such as Tarceva or Iressa, slowing tumor growth.

Personalized medicine uses genetic information to design treatments targeted to individual patients.

Unlike chemotherapy, which blasts all fast-growing cells in its wake, targeted treatments go after specific molecules. That makes them more effective at fighting particular types of cancers, including breast, colorectal and lung cancers. Now the approach is being expanded to fight an even broader range of cancers. It’s all part of a new wave in health care called personalized, or precision, medicine.

“This is the future of medicine,” says Dr. Massimo Cristofanilli, associate director for translational research and precision medicine at Northwestern University. “There is no turning back. The technology is available and there are already so many targeted therapies.”

Most medical treatments have been designed for the average patient, leading to a one-size-fits-all approach. But with vast amounts of data at their disposal, researchers now can analyze information about our genes, our family histories and other health conditions to better understand which types of treatments work best for which segments of the population.

This is a big deal. But it requires the know-how of geneticists, biologists, experts in artificial intelligence and computer scientists who understand big-data analytics. Several startups have already begun this work.

Deep Genomics, founded by researchers at the University of Toronto, uses AI to predict how genetic mutations will change our cells and the impact those changes will have on the human body. Epinomics, co-founded by scientists and physicians from Stanford University, is building a map of what turns our genes on and off, giving physicians a guide they could use to craft personalized therapies. And Vitagene, a small San Francisco startup, provides personalized advice on nutrition and wellness based on your DNA.

Dr. Massimo Cristofanilli

Just like Facebook learns to automatically recognize Aunt Martha in your family photos, Deep Genomics finds and categorizes patterns in genetic data. Once it’s found those patterns, the company’s deep learning system can infer if and how changes to your DNA affect your body.

That’s a big step forward compared with current genetic tests. Most can only give a probability of, say, getting breast cancer based on data from an entire population. Other tests can’t even tell you if the genetic changes they’ve detected mean anything.

The work is personal for Brendan Frey, CEO and co-founder of Deep Genomics and a professor at the University of Toronto. Fourteen years ago, he and his wife discovered their unborn baby had a genetic condition.

“We knew there was a genetic problem, but our counselor couldn’t tell us if it was serious or if it was going to turn out to be nothing,” Frey says. “We were plunged into this very difficult, emotional situation.”

The experience made Frey want to bridge the divide between identifying genetic anomalies and understanding what they mean.

Deep learning or machine learning — when computers teach themselves as they see more data — can also help doctors know which drugs will most effectively treat a patient’s illness and whether that person is more likely to experience side effects.

It can also help predict how cancer cells will mutate. And that can help drug companies come up with new treatments as tumor cells change and patients no longer respond to the drugs that worked.

That could help turn a disease like cancer into a manageable chronic ailment, says Cristofanilli.

Where Deep Genomics analyzes patterns in genetic data to predict when mutations will make you sick, Epinomics looks at epigenomics, or the study of what turns our genes on and off.

The company describes it like this: If your genome, which shows what genes we have, is the hardware of our bodies, then the epigenome is its software programming. Epinomics aims to decode that programming.

Every cell in the body carries the same genetic code. But cells in the heart, brain, bone and skin function differently based on this programming. It happens because chemical markers attach to DNA to activate or silence genes. These markers, known as the epigenome, vary from one cell type to another and are affected by both nature (inheritance) and nurture, which can include the air we breathe and the food we eat.

Researchers think a disruption to the epigenome can cause illnesses such as Alzheimer’s disease, diabetes or cancer. Understanding it could give physicians a guide to the best options for each patient, like having a GPS for treatments at the molecular level.

“We are focusing on what is happening at the programming level of each cell,” says Epinomics co-founder Fergus Chan. “Once we understand how genes are being turned on and off, we’ll be able to better predict which treatments will work or whether changes to lifestyle will have an impact on health.”

When Vitagene co-founder and CEO Mehdi Maghsoodnia asked a doctor what vitamins he should be taking, he was handed a bottle of pills and told to hope for the best.

Fergus Chan

That was the beginning of Vitagene, which uses genetic data and other health information culled from a detailed questionnaire to deliver a personalized nutritional supplement plan that lists which vitamins you need and in what doses, as well as what to avoid.

Maghsoodnia offers an alternative to the one-size-fits-all $27 billion US dietary supplement industry. Customers pay $99 to have their DNA tested and blood analyzed. And for $69 a month, Vitagene will package and ship supplements in dosages tailored to your individual needs.

The Food and Drug Administration estimates there are more than 85,000 dietary supplements on the US market, most of which are unregulated. Nearly all are “promising everything from anti-aging to weight loss, and no science behind it to tell you what works for you,” says Maghsoodnia. “We help filter through the noise.”

Vitagene’s algorithm has been tested on patients who’ve had bariatric surgery for weight loss, which often leaves them deprived of key nutrients. Vitagene helped develop a supplement regimen to get these patients the nutrition they need after surgery.

Precision medicine is in its early days.

This is especially true for psychiatry and its exploration of how the brain responds to the environment, stress and genetic disorders. Now several companies are selling tests to help psychiatrists select drug treatments by looking at patients’ DNA mutations and their metabolizing rate.

See more from CNET Magazine.

But critics caution that these genetic tests may be overselling their capabilities.

“Precision medicine has been very promising in oncology,” says Jose de Leon, a professor of psychiatry at the University of Kentucky who specializes in psychopharmacology. “But we know a lot more about cancer and how it works. In psychiatry, it’s much harder because we don’t know enough about how the brain works.”

Yes, precision medicine holds enormous promise.

Even so, Northwestern’s Cristofanilli cautions clinicians to stay grounded in reality. “It can be difficult to understand where reality becomes imagination,” he says. “We want to make sure we are protecting patients from claims that we may not deliver.”

For her part, Truong is grateful to benefit from the work that’s already been done. “I’m an engineer,” she says.

“I don’t believe in miracles. I believe in science.”

This story appears in the spring 2017 edition of CNET Magazine. For other magazine stories, click here.

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