Cutting-edge science and the development of groundbreaking and lifesaving drugs get a lot of attention, but the everyday practice of medicine is often more mundane: It doesnt involve being CRISPRd or having gene therapy delivered into your cells. The truth is that most people who are very sick and visit their doctor get treated with very ordinary and sometimes very old drugs.

But in recent years supplies of some of these critical standby medicines have become disrupted. The shortages have forced doctors to make hard treatment decisions. Drugs have to be rationed or, in the worst cases, patients who need care can be turned away.

Dr. Ben Davies is a professor of urology at the University of Pittsburgh and a BioTwitter influencer (I dont think anyone has ever called me a BioTwitter influencer before, he says.). Davies recently chatted with STAT about the recurring problem of drug shortages.

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Youve had experience with shortages of a medicine called BCG. Whats the history of BCG and why is it so important for bladder cancer patients?

BCG is a very special and powerful antineoplastic drug. Its been around since the 1980s and Im sure you remember it being used for tuberculosis vaccination.

Its basically an attenuated microbacterium and it does three very powerful things. If you get bladder cancer thats non-muscle-invasive, it can prevent recurrences and it can stop progression of disease. That would be extremely helpful because bladder cancer can progress into your muscle, which requires a different form of treatment, like radical surgery. And it also can actually treat residual tumors that your doctor may not have seen on your initial look in your bladder. So its a very important drug. It has a great response rate of about 68%. Its very well-tolerated most of the time. I mean, its our first line of defense in treating non-muscle-invasive bladder cancer.

Why is BCG so vulnerable to shortages?

Because they make it in massive batches and if the batch goes bad, youre just basically done. And depending on how big the batches are, you could go for months making a big supply of it and then youre done if it gets contaminated. So its unlike making a pill where if you just notice a few bad pills you can throw them away and restart. This thing really makes you start again, like a few months later, and youre behind the eight ball.

Merck is the only company that makes BCG today. At one time Sanofi (SNY) was a supplier but it abandoned the drug in 2016. Are you surprised that a drug thats so widely used has only a single manufacturer?

Well, I would be surprised if I didnt know the economics behind it. The problem is that the barrier to entry is quite high and extremely expensive. You know, the Mylans of the world youd think would just jump into this market. But theres a huge barrier to get in there $100 million or more. That and the drug is relatively cheap. So youre not going to make much money if you jump in. Im not surprised there arent so many suppliers now. We need to change those things to make it a more sought-after and more manufactured drug.

Were saying: We dont have the drug thats going to help you and that its going to cost you more.

Dr. Ben Davies, University of Pittsburgh

I mean, I am surprised that Merck has done such a Im gonna use a urologic term piss-poor job of making it since 2016. So weve had a good almost three years of years of problems getting the drug just their problem basically of getting it to patients and I dont know why they havent done a more robust response to the need. I mean, its not like the actual amount of bladder cancer patients changes over time. Its pretty stable. They know how much they need. They just havent been able to do it.

As a physician, when you have a patient before you with bladder cancer, what do you do when BCG is in short supply or not in supply at all?

When its not in supply at all, we go to alternative drugs like mitomycin, which is much more expensive. I wrote a small paper in the New England Journal about how as soon as BCG is unavailable, the price of mitomycin spiked by four times. But we go to other drugs. If we dont have enough, we stop something called maintenance therapy in a lot of patients. Thats a time when you get the drug continually only to help stop recurrences and wed lower the doses. So therere a few things we can do, which seems to be OK. But its certainly not as efficacious as getting the whole drug.

If you had power or influence over the supply of BCG, what would you do to try to make the drug more plentiful?

Oh, God, I think the number one thing, and Ive said this publicly and Ive written about it, you have to think, unfortunately, even though Im a card-carrying liberal, you have to raise the price of the drug. If manufacturers arent going to make any money on the drug, theyre simply not going to be that interested in making it. And thats just the system we live in. So if we dont do that, then we can think about federal mandates, but that has not worked well in the history of drug problems and shortages. So in my view, you have to increase the price and find a willing manufacturer, unlike Merck, that is willing to make the drug correctly and in appropriate amounts.

BCG is not the only drug thats often in short supply. At any given time there can be dozens, sometimes hundreds, of older medicines that appear on the FDAs drug shortage list. Do you think this problem is getting enough attention?

I dont think its really in peoples wheelhouse because they dont see it. I mean, I see it every day.

Its really a horrific situation. When I sit down with the patient, I tell him I cant give you the drug. It has such a great response rate. Instead Im going to give you an inferior drug that has a poor response rate. And by the way, its four times as much money, which is an important consideration in many of the medical plans that have people pay considerable amounts of money out of pocket for their drugs. And thats basically what were saying: We dont have the drug thats going to help you and that its going to cost you more. So its a major problem.

Much of this issue is tied up in the economics of how drugs are bought and paid for in this country. Recently Jeremy Corbyn in the U.K. proposed starting a government-run generics manufacturing establishment over there and there was a similar plan that Elizabeth Warren had described in terms of getting the government involved. What do you think of that as an idea for an issue like BCG?

I think it would work for BCG. I mean, I kind of mentioned my political stripes so I dont mind government intervention when its necessary. We already have this program, by the way. The governments heavily involved right now in getting appropriate vaccinations to people and supports it with its own money. It doesnt have the manufacturing capabilities. I think we need some sort of federal mandate where that means they have the private sector contracted or something like that. And I think that I would be completely in support of that for issues like this.

Theres another company proposing to do this a little bit. Nothings actually happened, but a conglomerate of universities were all going to come together and make, like, saline and epinephrine and all these bizarre drugs that we dont have available. Civica Rx. It sounds like a great scheme, but nothings ever actually happened. Its been mostly talking.

I was talking to my good friend Amitabh Chandra from Harvard who is a health economist about this. He says that a lot of this, he thinks, could be solved by better distribution. In other words, right now if I have enough BCG in my hospital but the hospital across the street does not have any, theres no real easy mechanism for me to give them that BCG, even though I could. There is a massive sort of inter-hospital or between-physician lack of communication and resources to exchange drugs or therapies with ease. You can imagine theres a good reason for that, for regulatory reasons.

But there is an opportunity, I think, to increase abilities to hand off drugs to other people easier and that would obviously alleviate some of the problems.

A good example of that is we can commonly have doctors in the community who dont have any BCG at all for whatever reason and so then those patients will come to see me, often many hundreds of miles to get BCG because their doctor cant get any. Well, thats silly. Why cant I ship that BCG to that doctor? So I mean theres things like that which can be done which would ameliorate the situation. But the bottom line is that we have a failure of manufacture. We have a failure, really, of imagination on how to fix this problem. This has been going on since at least 2012. Just nothing has ever happened to fix it.

Let us know when you start the BCG socialist collective.

Want me to start one?

This is a lightly edited transcript from a recent episode of STATs biotech podcast, The Readout LOUD. Like it? Consider subscribing to hear every episode.

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What one doctor thinks about drug shortages and how to solve them - STAT

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Mathew Knowles, music executive and father of Beyonc, has been diagnosed with breast cancer.

Knowles, 67, told Good Morning America Wednesday that he had a mastectomy to remove the cancer and is planning a preventive one in the other breast because testing found he has a genetic mutation for the disease. The mutation, BRCA2, means his children including Beyonce and Solange have a 50 percent chance of carrying the gene too.

The BRCA2 gene sharply increases the risk of breast and ovarian cancer in women. In men, it increases the risk of prostate, skin and pancreatic cancer in addition to breast cancer.

Knowles said he wanted to share his story to draw attention to the fact men can get breast cancer too. About 2,500 men are diagnosed with the disease annually, about 1 percent of all breast cancers.

Treatment for male breast cancer is similar to treatment for female breast cancer surgery, chemotherapy, radiation therapy and targeted therapy. Most male patients undergo a full mastectomy instead of a lumpectomy, the removal of just the tumor and small amount of surrounding tissue. Survival for male breast cancer is slightly lower than female breast cancer, mostly because it tends not to be diagnosed until men are older and the disease is more advanced.

Knowles medical journey began when he began noticing red dots on his undershirt and bed sheets. He squeezed his right nipple and blood came out.

The next day, Knowles saw his doctor, who sent a sample for further testing and then ordered up a mammogram. The mammogram found a Stage 1 tumor, among the earliest and easiest to treat. Follow-up testing revealed Knowles has BRCA2, prompting his plans to have a second mastectomy in January.

Besides bleeding, other signs of possible male breast cancer are a lump under the nipple or arm. Medical experts advise men who notice such symptoms to see a doctor for further evaluation.

Knowles is best known for managing Destinys Child. He also managed the careers of Beyonc and Solange.

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Beyoncs father diagnosed with breast cancer - Houston Chronicle

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Researchers at the National Institutes of Health (NIH) have developed a new and improved viral vector--a virus-based vehicle that delivers therapeutic genes--for use in gene therapy for sickle cell disease. In advanced lab tests using animal models, the new vector was up to 10 times more efficient at incorporating corrective genes into bone marrow stem cells than the conventional vectors currently used, and it had a carrying capacity of up to six times higher, the researchers report.

The development of the vector could make gene therapy for sickle cell disease much more effective and pave the way for wider use of it as a curative approach for the painful, life-threatening blood disorder. Sickle cell disease affects about 100,000 people in the United States and millions worldwide.

"Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease," said study senior author John Tisdale, M.D., chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute (NHLBI). "It's the new kid on the block and represents a substantial improvement in our ability to produce high capacity, high efficiency vectors for treating this devastating disorder."

Researchers have used virus-based vehicles for years in gene therapy experiments, where they have been very effective at delivering therapeutic genes to bone marrow stem cells in the lab before returning them to the body. But there's always room for improvement in their design in order to optimize effectiveness, Tisdale noted. He compared the new virus-based vehicle to a new and improved car that is also far easier and cheaper for the factory to produce.

The study was supported by the NHLBI and the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), both part of the NIH. It was published online today in Nature Communications.

Sickle cell disease is an inherited blood disorder caused by a mutation, or misspelling, in the beta-globin gene (or -globin gene). This mutation causes hemoglobin, the main ingredient of blood cells, to produce sickle-shaped cells that can stick to the walls of blood vessels, causing blockage, pain, anemia, organ damage, and early death. With gene therapy, doctors modify the patient's bone marrow hematopoietic (blood-producing) stem cells in the lab by adding a normal copy of the beta-globin gene through the use of a viral vector. They then reinfuse the modified stem cells into the patient to produce normal, disc-shaped red blood cells.

For the past 30 years, researchers have been designing these beta-globin vectors in a reverse structural orientation, meaning the therapeutic genes incorporated into the virus are translated, or "read," from right to left by the viral vector-making machinery--much like reading an English sentence backwards. The reason for the reverse orientation is the sensitive expression of a key molecular component of the vector called intron 2. This segment is required for high-level beta-globin gene expression but gets clipped out during the normal vector preparation process if it is left in the natural, forward direction. Gene therapy trials using reverse-oriented vectors for sickle cell disease and beta-thalassemia have largely been encouraging, the researchers said, but this complicated gene translation process has made vector preparation and gene-transfer efficiency more difficult.

About 10 years ago, Tisdale and Naoya Uchida, M.D., Ph.D., a staff scientist in his lab, searched for an improved delivery vehicle--like designing a better car--and decided to undertake a radical redesign of the beta-globin vector. They came up with a unique work-around design that left intron 2 intact and created the new forward-oriented beta-globin vector. In contrast to the old vector, the gene sequence, or "message," of the new beta-globin vector is read from left to right--like reading a normal sentence--making the gene translation approach less complicated, Tisdale explained.

The researchers tested the new vectors in mice and monkeys and compared the results to reverse-oriented vectors. They found that the new vectors could transfer a much higher viral load--up to six times more therapeutic beta-globin genes than the conventional vectors--and had four to 10 times higher transduction efficiency, a measure of the ability to incorporate corrective genes into repopulating bone marrow cells. The new vectors also showed a capacity for longevity, remaining in place four years after transplantation. Researchers also found that they could be produced in much higher amounts than the conventional vectors, potentially saving time and lowering costs associated with large-scale vector production.

"Our lab has been working on improving beta-globin vectors for almost a decade...and finally decided to try something radically different--and it worked," Tisdale said. "These findings bring us closer to a curative gene therapy approach for hemoglobin disorders."

Reference: Uchida et al. 2019.Development of a forward-oriented therapeutic lentiviral vector for hemoglobin disorders. Nature Communications. DOI: https://doi.org/10.1038/s41467-019-12456-3.

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

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Improved Gene Therapy in Sickle Cell Disease - Technology Networks

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Since the gene-editing potential of CRISPR systems was realized in 2013, they have been utilized in laboratories across the world for a wide variety of applications. When this gene-editing power is harnessed with the proliferative potential of stem cells, scientists level up their understanding of cell biology, human genetics and the future potential of medicine.

Thus far, the feasibility to edit stem cells using CRISPR technology has been demonstrated in two key areas: modeling and investigating human cell states and human diseases, and regenerative medicine.1 However, this has not been without challenges.

In this article, we explore some of the latest research in these spaces and the approaches that scientists are adopting to overcome these challenges. Deciphering cell-specific gene expression using CRISPRi in iPSC-derived neurons

Deploying CRISPR technology in iPSCs has been notoriously challenging, as Martin Kampmann, of the Kampmann lab at the University of California San Francisco, says: "CRISPR introduces DNA breaks, which can be toxic for iPSCs, since these cells have a highly active DNA damage response." To overcome the issue of toxicity, as a postdoc in the lab of Professor Jonathan Weissman, Kampmann co-invented a tool known as CRISPR interference (CRISPRi), where the DNA cutting ability of CRISPR/Cas9 is disabled.2 The "dead" Cas9 (or, dCas9) is still recruited to DNA as directed by a single guide RNA. It can therefore operate as a recruitment platform to target protein domains of interest to specific places in the genome.

CRISPRi permits gene repression at the transcription level, as opposed to RNAi which controls genes at the mRNA level. This allows researchers to repress certain genes within stem cells and decipher their function. Kampmann explains: "For CRISPRi, we target a transcriptional repressor domain (the KRAB domain) to the transcription start site of genes to repress their expression. This knockdown approach is highly effective and lacks the notorious off-target effects of RNAi-based gene knockdown."In a study published just last month, Kampmann's laboratory adopted a CRISPRi-based platform to conduct genetic screens in human neurons derived from iPSCs: "CRISPR-based genetic screens can reveal mechanisms by which these mutations cause cellular defects, and uncover cellular pathways we can target to correct those defects. Such pathways are potential therapeutic targets."3"We expressed the CRISPRi machinery (dCas9-KRAB) from a safe-harbor locus in the genome, where it is not silenced during neuronal differentiation. We also developed a CRISPRi construct with degrons, stability of which is controlled by small molecules. This way, we can induce CRISPRi knockdown of genes of interest at different times during neuronal differentiation."

Image: iPSC-derived neurons. Credit: Kampmann Lab, UCSF.Previous CRISPR-based screens have focused on cancer cell lines or stem cells rather than healthy human cells, thus limiting potential insights into the cell-type-specific roles of human genes. The researchers opted to screen in iPSC-derived neurons as genomic screens have revealed mechanisms of selective vulnerability in neurodegenerative diseases, and convergent mechanisms in neuropsychiatric disorders.

The large-scale CRISPRi screen uncovered genes that were essential for both neurons and iPSCs yet caused different transcriptomic phenotypes when knocked down. "For me, one of the most exciting findings was the broadly expressed genes that we think of as housekeeping genes had different functions in iPSCs versus neurons. This may explain why mutations in housekeeping genes can affect different cell types and tissues in the body in very different ways," says Kampmann. For example, knockdown of the E1 ubiquitin activating enzyme, UBA1, resulted in neuron-specific induction of a large number of genes, including endoplasmic reticulum chaperone HSPA5 and HSP90B1.

These results suggest that comprised UBA1 triggers a proteotoxic stress response in neurons but not iPSCs aligning with the suggested role of UBA1 in several neurodegenerative diseases. The authors note: "Parallel genetic screens across the full gamut of human cell types may systematically uncover context-specific roles of human genes, leading to a deeper mechanistic understanding of how they control human biology and disease."

Video credit: UCSF.

Developing and testing cell-based therapies for human disease using CRISPR

Several laboratories across the globe are in an apparent race to develop the first clinically relevant, efficacious and safe cell-based therapy utilizing CRISPR gene-editing technology.

Whilst a plethora of literature demonstrates the efficacy of CRISPR in editing the genome of mammalian cells in vitro, for in vivo application, particularly in humans, rigorous long-term testing of safety outcomes is required. This month, researchers from the laboratory of Hongkui Deng, a Professor at Peking University in Beijing, published a paper in The New England Journal of Medicine.4 The paper outlined their world-first study in which they transplanted allogenic CRISPR-edited stem cells into a human patient with HIV.

The rationale for the study stems back to the "Berlin patient", referring to Timothy Ray Brown who is one of very few individuals in the world that has been cured of HIV. Brown received a bone marrow transplant from an individual that carries a mutant form of the CCR5 gene. Under normal conditions, the CCR5 gene encodes a receptor on the surface of white blood cells. This receptor effectively provides a passageway for the HIV to enter cells. In individuals with two copies of the CCR5 mutation, the receptor is distorted and restricts strains of HIV from entering cells.

Deng and colleagues used CRISPR to genetically edit donor hematopoietic stem and progenitor cells (HSPCs) to carry either a CCR5 insertion or deletion. They were able to achieve this with an efficiency of 17.8%, indicated by genetic sequencing. The CRISPR-edited HSPCs were then transplanted into an HIV patient who also had leukaemia and required a bone-marrow transplant, with the goal being to eradicate HIV.

"The study was designed to assess the safety and feasibility of the transplantation of CRISPRCas9modified HSPCs into HIV-1positive patients with hematologic cancer," Deng says. He continues: "The success of genome editing in human hematopoietic stem and progenitor cells was evaluated in three aspects including editing persistence, specificity and efficiency in long-term engrafting HSPCs." Long-term monitoring of the HIV patient found that, 19 months after transplantation, the CRISPR-edited stem cells were alive however, they only comprised five to eight percent of total stem cells. Thus, the patient is still infected with HIV.

Despite the seemingly low efficiency in long-term survival, the researchers were encouraged by the results from the safety assessment aspect of the study: "Previously reported hematopoietic stem and progenitor cells-based gene therapies were less effective because of random integration of exogenous DNA into the genome, which sometimes induced acute immune responses or neoplasia," Deng says. "The apparent absence of clinical adverse events from gene editing and off-target effects in this study provides preliminary support for the safety of this gene-editing approach."

"To further clarify the anti-HIV effect of CCR5-ablated HSPCs, it will be essential to increase the gene-editing efficiency of our CRISPRCas9 system and improve the transplantation protocol," says Deng.

The marrying of CRISPR gene-editing and stem cell research isn't just bolstering therapeutic developments in HIV. An ongoing clinical trial is evaluating the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ HSPCs for the treatment of transfusion-dependent -thalassemia, a genetic blood disorder that causes hemoglobin deficiency.

The therapeutic approach known as CTX001 involves extracting a patients HSPCs and using CRISPR-Cas9 to modify the cells at the erythroid lineage-specific enhancer of the BCL11A gene. The genetically modified cells are then infused back into the patient's body, where they produce large numbers of red blood cells that contain fetal hemoglobin. Currently no results are available, but reports confirm that participants have been recruited on to the trial.

A bright future

Our understanding of cell biology and diseased states has been majorly enhanced by the combined use of CRISPR technology and stem cells. Whilst this article has focused on current study examples, Zhang et al.'s recent review provides a comprehensive view of the field and insights provided by earlier studies.5

In such review, the authors comment "Undoubtedly, the CRISPR/Cas9 genome-editing system has revolutionarily changed the fundamental and translational stem cell research." Solutions are still required to resolve the notorious off-target effects of CRISPR technology, to improve the editing efficiency as outlined by Deng and to exploit novel delivery strategies that are safe for clinical stem cell studies. Nonetheless, the future looks bright for CRISPR and stemcell-based research. In their review published this month, Bukhari and Mller say, "We expect CRISPR technology to be increasingly used in iPSC-derived organoids: protein function(subcellular localization, cell type specific expression, cleavage, and degradation) can be studied in developing as well as adult organoids under their native conditions."

References:

1. Jehuda, Shemer and Binah. 2018. Genome Editing in Induced Pluripotent Stem Cells using CRISPR/Cas9. Stem Cell Reviews and Reports. DOI: 10.1007/s12015-018-9811-3.

2. Qi et al. 2013. Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression. Cell. DOI: 10.1016/j.cell.2013.02.022

3. Tian et al. 2019. CRISPR Interference-Based Platform for Multimodal Genetic Screens in Human iPSC-Derived Neurons. Neuron. https://doi.org/10.1016/j.neuron.2019.07.014.

4. Xu et al. 2019. CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia. The New England Journal of Medicine. DOI: 10.1056/NEJMoa1817426.

5. Zhang et al. 2019. CRISPR/Cas9 Genome-Editing System in Human Stem Cells: Current Status and Future Prospects. Molecular Therapy Nucleic Acids. DOI: 10.1016/j.omtn.

Read more from the original source:
Exploring the Latest in CRISPR and Stem Cell Research - Technology Networks

Recommendation and review posted by Bethany Smith

Updated October 04, 2019 14:43:48

The hardest thing Daniel Roberts has ever had to do was watch his young niece Remy battle aplastic anaemia.

Remy was just a toddler when diagnosed with the condition, in which the body stops producing enough new blood cells.

The once-energetic and vivacious girl became lethargic, covered in deep bruises.

Her only hope was a bone marrow transfusion, but finding a match was easier said than done.

"I wanted to be able to save my brother's little girl, but no-one in the family was a match, which was quite unbelievable," Mr Roberts said.

Remy's condition deteriorated she required blood transfusions every seven to 10 days but thankfully an unrelated donor was found.

Now six, she is back at school and getting stronger every day.

"Remy, she was right on the cusp of not being here with us, so to see her with us today is absolutely amazing," he said

Despite not being able to help his niece, Mr Roberts stayed on the bone marrow donor list only one in 1,500 donors are asked to donate in any given year.

Just two years after a random donor saved his niece's life, Mr Roberts got the call.

"I'm six-foot-three and pretty bulletproof. I'm tough as nails and never get a tear in my eye or anything like that, but I couldn't stop actually crying," he said.

"I just couldn't believe I got to pay it back so quickly."

His donation helped save a five-year-old boy who was also fighting aplastic anaemia.

"The amount of tests and all that you go through is nothing compared to what that little fella was probably going through," Mr Roberts said.

"But that said, I'd do it again in a heartbeat.

"My niece was dying. She got a random match from somebody, and now she's fantastic. I just can't believe you can pay someone back so quickly."

Every 40 minutes, someone in Australia is diagnosed with a blood cancer.

And for most, a blood stem cell or bone marrow transplant from a stranger is their only hope.

But if you are Indigenous or ethnically diverse, the chances of finding a match are much harder, because donors need to have the same genetic background as the recipient.

Less than 1 per cent of people on the bone marrow donor registry are from Aboriginal or Middle Eastern background, less than 3 per cent are Asian, and less than 5 per cent are Pacific islander.

The head of the registry, Lisa Smith, said despite Australia's multicultural make-up, eight of every 10 people on the registry were of north Caucasian background.

"The registry itself is largely unknown and really lacking diversity that reflects Australia's multiculturalism," she said.

"It genuinely is one-in-a-million odds to get a match, so when we ring a donor, they could very well be the only person on the planet that's able to help that patient."

The Australian Bone Marrow Donor Registry is trying to find 5,000 new donors this year.

"We need donors that are young and ethnically diverse, because our current donor pool, if characterised as a person, is a middle-aged white female," Ms Smith said.

"From a transplant clinician's perspective, they're looking for donors that are young between 18 and 30, are male and are reflecting the ethnicity of their patient."

Pamela Bou Sejean knows the importance of a strong donor list better than most.

She was diagnosed with Hodgkin lymphoma in 2010, and after chemotherapy and radiation failed, she was told her "last chance" was a stem cell transplant.

Stem cells from the umbilical cord blood of a Spanish donor gave Ms Bou Sejean a second chance at life.

"To know someone's out there that's actually saved my life, 'thank you' doesn't seem enough," she said.

She went into remission in 2012 and founded the charity Ur The Cure, which aims to demystify stem-cell donations and promote greater diversity on the donor list.

"Often it's people's last chance for a cure, and you need a stem cell match with someone who shares an ethnical or cultural heritage."

There is a 30 per cent chance that a match can generally be found within a person's immediate family. After that, it gets harder.

"It's kind of like winning TattsLotto, but it's not winning money it's winning the chance to live."

For Warrnambool mother Julia Thompson, storing her umbilical cord for stem cells was a no-brainer.

Following her own cancer battle, Ms Thompson gave birth to her son Hugh in 2017.

She is now in remission, having used her stem cells as part of her treatment.

It was that treatment that inspired her to store Hugh's umbilical cord and associated stem cells for future use.

"It's difficult that it's only available in selected hospitals in Australia, but it's really an amazing insurance policy for him," she said.

"We knew that because of my history with cancer, the likelihood of Hugh having siblings was very slim.

"Given my history and the fact that stem cell blood saved my life, I knew how important that was. It's really something to consider for the future of your child and family."

Topics:leukaemia,bones-and-muscles,people,medical-research,health,science-and-technology,human-interest,warrnambool-3280,geelong-3220,melbourne-3000

First posted October 02, 2019 06:54:09

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Bone marrow donor registry pleas for more diversity to help save people with cancer - ABC News

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PHOENIX, Oct. 1, 2019 /PRNewswire/ --R3 Stem Cell, a national leader in stem cell therapy marketing and education, today announced that they are launching a new Master Class centered around the topic of stem cells and regenerative procedures. To commemorate their launch, they will be giving away one out of the eight episodes of their Master Class series for free and the rest will be available for only $49. Purchase the R3 Stem Cell Master Class series and receive Stem Cell Therapy with an R3 Center for $500 off. Register for the Master Class at https://stemcellmasterclass.org/.

The Master Class offers those considering regenerative procedures with stem cells vital information such as discovering how the process works, understanding the options that are given to them, learning the research behind it and gaining realistic expectations.

"Experiencing chronic pain is tiring and can lead to anxiety, depression, obesity, and addiction of medication," said David Greene, MD, MBA, Founder and CEO of R3 Stem Cell. "For those who are considering a stem cell procedure, our Master Class will provide them with the knowledge needed in order to make the decision. Questions regarding common misconceptions in the ever-growing field will be uncovered, giving the patient a realistic understanding of what a stem cell is, where they come from and what they can do."

The Master Class series explains what to expect with a regenerative procedure investment, what the different ways are to optimize stem cell therapy outcomes, what to look for in a stem cell clinic, and a patient's real-life experience with a stem cell procedure. The series is divided into eight different episodes, offering the patient a four-hour long learning opportunity with information on discovering a procedure that is right for them.

"It is important for patients to fully understand what type of stem cell therapies are being offered to them and know the full effect it can have on their everyday lives," continues Dr. Greene. "Regenerative therapies may give patients the opportunity to return to pain-free personal everyday activities. Our purpose is to make people educated consumers in a time when so much misinformation is being disseminated."

About R3 Stem Cell

R3 Stem Cell offers regenerative stem cell therapies to those who suffer from chronic pain. Their sole purpose is to repair, regenerate, and restore damaged tissue from the body. Giving hope and options for patients of relieving chronic pain and avoiding surgery. Stem cell procedures are done using bone marrow, adipose, amniotic, PRP or umbilical cord tissue containing platelets, cytokines, growth factors and exosomes that work to start a healing process within the body and repair damaged tissue. R3 Stem Cell has over 35 nationwide Centers and there is most likely a clinic near you. To learn more about R3 Stem Cell, visit their website at http://www.r3stemcell.comor call (844) GET-STEM.

Media Contact

Jennifer RodriguezFirecracker PR(888)317-4687 ext. 703223962@email4pr.com

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R3 Stem Cell Launches Master Class Series on Stem Cell Therapy and Regenerative Medicine - PRNewswire

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Anthony DeAngelis reads the birthday card his daughter Alyssa gave him after she donated two-thirds of her liver so he could undergo a transplant. Jerry Carino, @njhoopshaven

Such encounters are rare, especially across an ocean. 'She's like my daughter,' Lael McGrath said.

After four months of battling acute myeloid leukemia, Lael McGraths lifeline arrived in a cooler, the kind youd take to a football tailgate or picnic in the park.

Only it didnt contain ham-and-cheese subs from the local deli. These were stem cells from Germany, a lifesaving gift from an anonymous donor.

Last week McGrath, a grandmother of six who lives in Toms River, got to thank that donor face to face. Wiebke Rudolph visited New Jersey and the two celebrated their connection at Robert Wood Johnson University Hospital in New Brunswick.

For me its very emotional, McGrath said. Shes like my daughter. Thats how I feel. Its a life relationship that will always be there.

Paying it forward:Neptune family holds blood drive in honor of 2-year-old with cancer

Neptune: Daughter donates two-thirds of her liver, giving dad 2nd chance after alcoholism

WATCH: In the video atop this story, Neptune's Anthony DeAngelis reads a birthday card from his daughter, who donated two-thirds of her liver to him.

Bone marrow donor Wiebke Rudolph from Germany (left) hugs transplant recipient Lael McGrath of Toms River (right) at Robert Wood Johnson University Hospital New Brunswick.(Photo: Daniel DellaPiazza)

Fitness always has been a priority for McGrath, who turned 68 on Monday. In August of 2016 she didnt think much of it when her breathing became a bit labored. The flu, she figured.

Because I was such a fit person, my body didnt have any sickly warnings, she said.

The diagnosis was acute myeloid leukemia (AML), which occurs mostly in older adults the average age at diagnosis is 68. According to the American Cancer Society, there are about 21,000 new cases and 11,000 deaths from AML each year in the U.S. For treatment, McGrath was referred to the Blood and Marrow Transplant Program at Rutgers Cancer Institute of New Jersey and Robert Wood Johnson University Hospital New Brunswick. She would need a stem cell transplant.

Every four minutes a new person in the U.S. is being diagnosed with blood cancer and will need to find a donor, said Dr. Vimal Patel, McGraths hematologist/oncologist at Rutgers Cancer Institute of New Jersey and assistant professor of medicine at Rutgers Robert Wood Johnson Medical School.It can be hard (to find a match). We first look at siblings; each sibling has a 1-in-4 chance of being a match.

Do you enjoy tales of remarkable people in Monmouth and Ocean counties? Maybe you'd like to read about the Miss NJ USA contestant who learned to dealwith the suicide of her mother and grandmother and help others too. Or how a woman with Lyme disease wouldn't give up her dream of becoming a doctor. You can read more of them by becoming a digital subscriber to APP.com and downloading our app today.

(from left) Bone marrow transplant coordinator Mary Kate McGrath, transplant recipient Leal McGrath, hematologist/oncologist Dr. Vimal Patel, and bone marrow donor Wiebke Rudolph at a special celebration with members of the Blood and Marrow Transplant team at Rutgers Cancer Institute of New Jersey and Robert Wood Johnson University Hospital, New Brunswick.(Photo: Daniel DellaPiazza)

None of McGraths siblings came up as a match. So McGraths care team turned to Be The Match, the worlds largest bone-marrow donor registry. They hit the jackpot with Rudolph, although her identity remainedanonymous in keeping with widely accepted transplant protocol.

I just did it, and I didnt think much about it, Rudolph said of her decision to enroll in the registry. I didnt think I would ever know the name (of the recipient) or meet them. I just thought, You get some real-life karma if you do that.

The transplant went well. McGrath returned home two weeks later, on Jan. 1,2017. Then came three months of recovery at home. Now, two-and-a-half years later, McGrath says she is in remission, running a couple of days per week and doing power yoga.

Shes amazing, said daughter Torrey DiMeo, who also lives in Toms River. There are 20-year-olds in her (yoga) class and she flies past them. She is still standing on her head at 67.

Bone marrow donor Wiebke Rudolph from Germany (right) stands with transplant recipient Lael McGrath of Toms River (left) at Robert Wood Johnson University Hospital in New Brunswick.(Photo: Daniel DellaPiazza)

Upon learning each others identity, McGrath and Rudolph began a long-distance correspondence that blossomed into a friendship. Last week they met for the first time and visited Robert Wood Johnson together. It was a big moment for all involved.

Its pretty rare in terms of having an unrelated donor meet up with the actual patient themselves, Patel said. To see it happen is remarkable truly heartwarming.

DiMeo was similarly moved.

The emotions were just a whirlwind, she said. Words cant thank someone enough. I really didnt know what to say besides thank you.

McGrath already had begun paying it forward. In 2017 she threw a block party where representatives from Be The Matchregistered dozens of people. One of them could end up rescuing someone halfway around the world, just like Rudolph did.

This is the reason why I do what I do, Patel said. Its amazing to see the pieces of the puzzle that needed to come together, amazing to see the donor and her generosity the gift she gave, the gift of life.

For more information on Be The Match, visit http://www.bethematch.org.

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Jerry Carino is news columnist for the Asbury Park Press, focusing on the Jersey Shores interesting people, inspiring stories and pressing issues. Contact him atjcarino@gannettnj.com.

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Toms River grandmother with leukemia meets the stem-cell donor who saved her life - Asbury Park Press

Recommendation and review posted by Bethany Smith

A gene therapy approach using a so-called antibody-drug conjugate (ADC) conditioning regimen led to safe and sustained production of factor VIII (FVIII) in platelets, and prevented joint bleeding in a mouse model of hemophilia A, according to new research.

The study, Nongenotoxic antibody-drug conjugate conditioning enables safe and effective platelet gene therapy of hemophilia A mice, appeared in the journal Blood Advances.

Prior work in mice showed that stem cell-based gene therapy specifically targeting platelets leads to the production of FVIII the clotting protein missing or defective in people with hemophilia A and induces immune tolerance (no development of exacerbated immune response).

However, the procedure required a conditioning regimen with chemotherapy or total body irradiation (TBI) that may be toxic to genetic material (or genotoxic), so patients may not be agreeable to using the protocol.

Safer approaches may come from ADCs, which use antibodies against cell surface proteins for more specific targeting of cell populations.

A team of researchers from the Blood Research Institute, in Milwaukee, tested ADC-based conditioning with stem cell-based F8 gene therapy that targets platelets in a mouse model of hemophilia A. This ADC consists of saporin a plant-derived toxin that halts protein production bound to antibodies specific for the CD45.2 and CD117 cell surface proteins.

The scientists found that, three weeks after hematopoietic stem cell transplant (HSCT), peripheral blood counts were higher with ADC conditioning than with TBI. This suggested a lower risk of cytopenia (reduced number of mature blood cells), the investigators said.

Then they observed that the new conditioning regimen led to effective engraftment, the process through which transplanted stem cells establish themselves in the bone marrow and start producing new blood cells. At 20 weeks after transplant, all mice receiving the ADC regimen had more than 15% of donor-derived white blood cells.

Sixteen weeks after HSCT, the number of copies of therapeutic genes was similar in the mice receiving ADC and the controls on TBI. Two-thirds of the mice receiving the viral-delivered gene therapy under ADC conditioning showed sustained increases in FVIII levels in platelets.

The ADC regimen also led to increasing reconstitution of platelets and white blood cells, meaning a higher percentage of cells derived from donors.

Subsequent experiments showed that ADC also targeting the CD8 protein led to significantly higher white blood cell reconstitution, frequency of regulatory T-cells (which dampen excessive immune responses), and FVIII levels in platelets than the regimen specific for CD117 and CD45.2 only.

These effects were sustained long-term, as found when transplanting bone marrow cells from animals that had undergone HSCT to new mice under the same conditioning regimen.

Blood clotting was normalized with this gene therapy approach, and hemoglobin levels were higher than in mice with no FVIII. Joint bleeding and limping were effectively prevented in a knee joint injury model. Also, no animal with sustained FVIII expression in platelets developed anti-FVIII inhibitors.

The central finding of this report is that platelet-directed HSC-based FVIII gene therapy is safe and effective for eliminating [hemophilia A] using nongenotoxic hematopoietic-targeted ADC conditioning, the researchers wrote.

This safe and effective treatment strategy could be especially meaningful for [hemophilia A] patients who are especially wary of standard preconditioning, they added.

Jos is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimers disease.

Total Posts: 121

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Hemophilia A Study Finds Benefits With New Gene Therapy Approach - Hemophilia News Today

Recommendation and review posted by Bethany Smith

SAN DIEGO, Sept. 30, 2019 /PRNewswire/ -- Results from a study sponsored by Stemedica Cell Technologies, Inc., a global biotechnology company that uses allogeneic stem cells for ischemic conditions, form the basis for a peer-reviewed paper published inStrokeentitled "Phase I/II Study of Safety and Preliminary Efficacy of Intravenous Allogeneic Mesenchymal Stem Cells in Chronic Stroke." Co-authors include Michael L. Levy, MD, PhD, John R. Crawford, MD, Nabil Dib, MD, Lev Verkh, PhD, Nikolai Tankovich, MD, PhD and Steven C. Cramer, MD.

As indicated in theStrokepublication, "Stroke is perennially among the leading causes of human disability and the leading neurological cause of lost disability-adjusted life years. The mean survival after stroke is 6-7 years, with more than 85% of patients living past the first-year post-stroke, many with years of enduring disability. Many restorative therapies are under study to improve outcomes after stroke." However, restorative therapies often have a short time window for improvement usually measured in days-months.

"Based on Stemedica's preclinical data that supported the safety and efficacy of its MSCs as a restorative therapy to improve outcomes after stroke, the company was granted approval by the FDA to conduct a Phase I/IIa dose escalation trial that examined the effects of a single IV infusion of Stemedica's cGMP manufactured allogeneic ischemia-tolerant MSCs," said Dr. Lev Verkh, Chief Regulatory and Clinical Development Officer of Stemedica.

Study:The target population included patients with chronic ischemic stroke and substantial functional deficits; a group for whom treatment options remained limited. The primary outcome of the study was safety, based on serial measures of behavior, CT scans, and laboratory testing. Four secondary endpoints were scored serially to derive estimates of behavioral changes relatively to the baseline over a period of 12 months: NIH Stroke Scale (NIHSS) for neurological assessment, Barthel Index (BI) for ability to perform daily tasks, Mini-Mental Status Exam (MMSE) for mental status, and Geriatric Depression Scale (GDS) for degree of depression. The study was conducted at three centers: University of California, San Diego (UCSD); Mercy Gilbert Medical Center, Gilbert, Arizona; and University of California, Irvine (UCI).

Entry criteria included ischemic stroke >6 months prior to administration, substantial functional deficits (subject confined to a wheelchair, had home-nursingcare, orneeded assistance withactivities ofdailyliving), no substantial improvement in neurologic orfunctional deficits for the2 months prior to enrollment in thestudypermedical history, and NIHSS score=6-20.

Enrollees received a single intravenous dose of allogeneic mesenchymal bone marrow cells. Phase I used a dose escalation design (3 tiers, n=5 each). Phase IIa (n=21) was an expanded safety cohort. The primary endpoint was safety over 1-year. Secondary endpoints examined behavior, with a pre-specified focus at 6-months.

Subject status at enrollment prior to treatment:At baseline, subjects (n=36) averaged 4.24.6 years post-stroke, age 61.110.8 years, NIHSS score 8 [6.5-10], and Barthel Index 6529.

Safety:Study testing disclosed no safety concerns. No subject showed a positive reaction to intradermal testing. In Phase I, each dose (0.5, 1.0, and 1.5 million cells/kg body weight) was found safe, as a result Phase IIa subjects received 1.5 million cells/kg. Two subjects were lost to follow-up, one was withdrawn, and two died (unrelated to study treatment). There were 15 serious adverse events, none possibly or probably related to study treatment. Two mild adverse events were possibly related to study treatment, a urinary tract infection and IV site irritation. Treatment was determined to be safe based on serial exams, EKGs, laboratory tests, and pan-CT scans.

Behavioral Effects:Improvements across all subjects post-transfusion and for all four secondary endpoints were achieved. Improvements in each index were: Barthel Index (6.811.4 points, p=0.002); in NIHSS (-1.251.7 points, p<0.001); Mini Mental Status Exam (1.82.8 points, p<0.001); and Geriatric Depression Scale (-1.63.8 points, p=0.015). At baseline 11.4% (4/35 subjects) had Barthel Index=95-100 (favorable outcome); at 6-months, 27.3% (9/33); by 12-months, 35.5% (11/31).

Conclusions:The current study is the largest trial of intravenous MSCs in patients with chronic stroke and the first to evaluate allogeneic MSC therapy in this population. It is also the first study to evaluate MSCs grown under hypoxic conditions favorable to cell proliferation, gene expression, cytokine production and migration. While patients with stroke in the chronic stage generally show significant functional decline, enrollees in the current study showed 12 months of continued functional improvements across all secondary endpoints.

Intravenous transfusion of allogeneic ischemia tolerant MSCs in patients with chronic stroke and substantial functional deficits was safe and suggested behavioral gains. These data support proceeding to a randomized, placebo-controlled study of this therapy in this population.

Dr. Nikolai Tankovich, President and Chief Medical Officer added, "Stemedica is encouraged by the results of the study which demonstrated safety and preliminary efficacy of its cell therapy product for the treatment of chronic ischemic stroke patients. It is a significant milestone for Stemedica to bring this new cellular medication to patients with debilitating conditions caused by a stroke. Stemedica plans to move forward to a Phase-IIb discussion with the FDA."

Michael Levy, MD, PhD, FACS, FAANS, Professor of Neurosurgery at UCSD and the Principal Investigator of this study commented: "Based on my clinical trial work in Stemedica's Ischemic Stroke trial, my experience to date with Stemedica's allogeneic ischemic tolerant mesenchymal stem cell product suggests that the product is first and foremost safe and secondarilyhas the potential to produce unparalleled medical benefits."

About Stemedica Cell Technologies, Inc.Stemedica Cell Technologies, Inc. is a global biopharmaceutical company that manufactures best-in-class allogeneic adult stem cells. The company is a government licensed manufacturer of cGMP, clinical-grade stem cells currently used in US-based clinical trials for ischemic stroke, and Alzheimer's Disease. Stemedica's cell are also used on a worldwide basis by research institutions and hospitals for pre-clinical and clinical (human) trials. Stemedica is currently developing additional clinical trials for other medical indications using adult, allogeneic stems cell under the auspices of the FDA and other international regulatory institutions. The company is headquartered in San Diego, California and can be found online atwww.stemedica.com.

Forward Looking StatementsThis press release may contain forward-looking statements. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance and you are cautioned not to place undue reliance on these forward-looking statements. These statements reflect the views of Stemedica as of the date of this press release with respect to future events and, except as required by law, it undertakes no obligation to update or revise publicly any forward looking statements, whether as a result of new information, future events or otherwise after the date of this press release.

Media ContactStemedica Cell Technologies, Inc.Dave McGuiganEVP, Marketing & Business Developmentdmcguigan@stemedica.com+1 858-658-0910 x7203

SOURCE Stemedica Cell Technologies, Inc.

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Positive Study Results of Phase IIa Clinical Trial Using Intravenous Administration of Mesenchymal Stem Cells for Ischemic Stroke Published in...

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass.--(BUSINESS WIRE)--ElevateBio, a Cambridge-based biotechnology holding company, today announced it has established and launched HighPassBio, a company dedicated to advancing novel targeted T cell immunotherapies. The companys lead product is an engineered T cell receptor (TCR) T cell therapy for HA-1 expressing tumors, which is designed to treat and potentially prevent relapse of leukemia following hematopoietic stem cell transplant (HSCT). The product and approach were developed by researchers at Fred Hutchinson Cancer Research Center.

At ElevateBio we are committed to building therapeutics companies with the worlds leading innovators in cell and gene therapy to advance novel treatments that have strong potential to dramatically improve patient lives, said David Hallal, Chairman and CEO of ElevateBio. We look forward to leveraging our centralized industry-leading cell and gene therapy process development and manufacturing capabilities while working closely with Dr. Marie Bleakley and her team, to accelerate their impressive work through clinical development with the goal of serving patients who have no other treatment options. Additionally, we will explore this approach as a potential treatment for other diseases that are treated by stem cell transplants.

HighPassBios scientific founder is Marie Bleakley, M.D., Ph.D., M.ClinEpi, pediatric oncologist and stem cell transplant physician at Fred Hutchinson Cancer Research Center and chair of the scientific advisory board (SAB) of HighPassBio. As the chair of HighPassBios SAB, Dr. Bleakley will continue working with ElevateBio to advance and accelerate this innovative program through clinical development toward patients.

A Phase 1 clinical trial has treated initial patients and is recruiting adult and pediatric patients who have relapsed with leukemia or related conditions following blood and marrow transplantation (also known as stem cell transplantation). More details on http://www.clinicaltrials.gov under the study ID number NCT03326921.

About TCR-Engineered T Cell Therapy

A key role of the immune system is to detect tumor antigens, engage T cells and eradicate the tumor. However, the immune response to tumor antigens varies and is often insufficient to prevent tumor growth and relapse. An approach known as adoptive T cell therapy, using T cell receptors, or TCRs, can overcome some of the obstacles to establishing an effective immune response to fight off the target tumor. TCRs are molecules found on surface of T cells that can recognize tumor antigens that are degraded to small protein fragments inside tumor cells. Unlike CAR T cells that recognize only surface antigens, TCRs can recognize small protein fragments derived from intracellular and surface antigens offering a more diverse way to attack tumors. These small protein fragments show up on the tumor cell surface with another protein called major histocompatibility complex (MHC), get recognized by the TCRs and consequently signal the bodys immune system to respond to fight off and kill the tumor cells.

Tumor-specific TCRs can be identified and then engineered into T cells that recognize and attack various types of cancers, representing a novel approach to treating and potentially preventing disease.

Adoptive T cell therapy can be applied to tackling relapse of leukemia post hematopoietic stem cell transplant (HSCT) by targeting the antigens expressed only by the patient and not by the stem cell transplant donor, known as minor histocompatibility antigens, or HA1. HA1 is expressed predominantly or exclusively on hematopoietic cells and leukemic cells. There is evidence that T cells specific for HA1 can induce a potent and selective antileukemic effect. HA1 TCR T cell therapy is a new investigational immunotherapy for the management of post transplantation leukemia relapse.

About Leukemia post HSCT Treatment and the Risk of Relapse

Leukemia, a cancer of the blood or bone marrow characterized by an abnormal proliferation of blood cells, is the tenth most common type of cancer in the U.S. with an estimated 60,140 new cases and 24,400 deaths in 2016. Leukemia arises from uncontrolled proliferation of a specific type of hematopoietic (blood) cell that is critical for a functional immune system. As a result, when patients are given very high doses of chemotherapy to eradicate leukemic cells most normal cells are killed as well, necessitating a transplant of hematopoietic stem cells from a donor to reconstitute the patients bone marrow and circulating hematopoietic cells. In some cases, the transplanted T cells from the donor can also recognize and eliminate the hematopoietic cells, including leukemia, from the recipient, thus preventing relapse.

While the majority of HSCT recipients are cured, 25-50 percent of HSCT recipients relapse, leukemia relapse remains the major cause of allogeneic HSCT failure, and the prognosis for patients with post-HCT relapse is poor. Relapse occurs following allogeneic HSCT in approximately one-third of patients with acute leukemia who undergo the procedure, and most patients subsequently die of their disease.

About HighPassBio

HighPassBio, an ElevateBio portfolio company, is working to advance a novel approach to treating hematological malignancies by leveraging T cell receptor (TCR)-engineered T cells, known as TCR T cells. The companys lead program is designed to treat or potentially prevent relapse of leukemia in patients who have undergone hematopoietic stem cell transplant (HSCT). The technology was born out of Fred Hutchinson Cancer Research Center by world renowned expert, Dr. Marie Bleakley.

About ElevateBio

ElevateBio, LLC, is a Cambridge-based cell and gene therapy holding company, established to create and operate a broad portfolio of cell and gene therapy companies with leading academic researchers, medical centers and entrepreneurs. ElevateBio builds single- and multi-product companies by providing scientific founders with fully integrated bench-to-bedside capabilities including world-class scientists, manufacturing facilities, drug developers and commercial expertise. ElevateBio BaseCamp, a company-owned Cell and Gene Therapy Center of Innovation, will serve as the R&D, process development and manufacturing hub across the entire ElevateBio portfolio while also supporting selected strategic partners.

ElevateBios lead investors are the UBS Oncology Impact Fund (OIF) managed by MPM Capital, as well as F2 Ventures. Investors also include EcoR1 Capital, Redmile Group, and Samsara BioCapital.

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ElevateBio Launches HighPassBio to Advance Novel Targeted T Cell Immunotherapies with Technology from Fred Hutchinson Cancer Research Center -...

Recommendation and review posted by Bethany Smith

Press release

Gosselies, Belgium, 2 October 2019, 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the leading biotech company focused on the development of innovative cell and biological therapies to address high unmet medical needs in orthopaedics and bone diseases, announces that the Company will today present at the 27th Annual Meeting of the European Orthopaedic Research Society (EORS), in Maastricht, The Netherlands.

The Annual EORS Meeting is Europe's Summit for orthopaedic research and is attended by scientists, clinicians and entrepreneurs in the field. In the oral presentation, Bone Therapeutics will highlight additional preclinical in vitro and in vivo results demonstrating the potent osteogenic properties of its allogeneic bone-forming cell therapy platform, ALLOB, to promote bone-formation and improve fracture healing in relevant models.

ALLOB is the Companys allogeneic product that consists of human bone-forming cells derived from cultured bone marrow mesenchymal stem cells of healthy adult donors, and is manufactured through a proprietary, scalable production process. ALLOB successfully completed two Phase II studies in two indications and the Company is on track to submit a Clinical Trial Application with the regulatory authorities before the end of the year to allow the start of the next clinical development phase in patients with delayed-union fractures.

Presentation Details:

Title: Injectable cryopreserved allogenic bone-forming cells derived from bone marrow MSC (ALLOB) display potent osteogenic and bone repair propertiesAuthors: Sylvain Normand, Delphine De Troy, Coline Muller, Pierre-Yves Laruelle, Anna Tury, Sandra PietriSession: Cellular Regenerative Medicine (FP16)Date: Wednesday, 2 October 2019Time: 5:30pm CESTLocation: Room 08, Maastricht Exhibition & Congress Centre (MECC), Maastricht, The Netherlands

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopaedics and bone diseases. Based in Gosselies, Belgium, the Company has a broad, diversified portfolio of bone cell therapy and an innovative biological product in later-stage clinical development across a number of disease areas, which target markets with large unmet medical needs and limited innovation.

Bone Therapeutics core technology is based on its allogeneic cell therapy platform (ALLOB) which uses a unique, proprietary approach to bone regeneration, which turns undifferentiated stem cells from healthy donors into bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery, and are produced via a proprietary, cutting-edge manufacturing process.

The Companys ALLOB product pipeline includes a cell therapy product candidate that is expected to enter PhaseII/III clinical development for the treatment of delayed-union fractures and a PhaseII asset in patients undergoing a spinal fusion procedure. In addition, the Company is also developing an off-the-shelf protein solution, JTA-004, which is expected to enter PhaseIII development for the treatment of pain in knee osteoarthritis.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. Further information is available at http://www.bonetherapeutics.com.

Contacts

Bone Therapeutics SAThomas Lienard, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0) 71 12 10 00investorrelations@bonetherapeutics.com

International Media Enquiries:Consilium Strategic CommunicationsMarieke VermeerschTel: +44 (0) 20 3709 5701bonetherapeutics@consilium-comms.com

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: + 33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors` current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such person`s officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics to present preclinical data on the osteogenic properties of ALLOB in bone repair at 27th Annual Meeting of the European Orthopaedic...

Recommendation and review posted by Bethany Smith

Global Cell Therapy Technologies Market

was valued US$ 12 billion in 2018 and is expected to reach US$ 35 billion by 2026, at CAGR of 12.14 %during forecast period.

The objective of the report is to present comprehensive assessment projections with a suitable set of assumptions and methodology. The report helps in understanding Global Cell Therapy Technologies Market dynamics, structure by identifying and analyzing the market segments and projecting the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, growth strategies, and regional presence. To understand the market dynamics and by region, the report has covered the PEST analysis by region and key economies across the globe, which are supposed to have an impact on market in forecast period. PORTERs analysis, and SVOR analysis of the market as well as detailed SWOT analysis of key players has been done to analyze their strategies. The report will to address all questions of shareholders to prioritize the efforts and investment in the near future to the emerging segment in the Global Cell Therapy Technologies Market.

Request For Free Sample Report : https://www.maximizemarketresearch.com/market-report/global-cell-therapy-technologies-market/31531/#requestforsample

Global Cell Therapy Technologies Market: OverviewCell therapy is a transplantation of live human cells to replace or repair damaged tissue and/or cells. With the help of new technologies, limitless imagination, and innovative products, many different types of cells may be used as part of a therapy or treatment for different types of diseases and conditions. Celltherapy technologies plays key role in the practice of medicine such as old fashioned bone marrow transplants is replaced by Hematopoietic stem cell transplantation, capacity of cells in drug discovery. Cell therapy overlap with different therapies like, gene therapy, tissue engineering, cancer vaccines, regenerative medicine, and drug delivery. Establishment of cell banking facilities and production, storage, and characterization of cells are increasing volumetric capabilities of the cell therapy market globally. Initiation of constructive guidelines for cell therapy manufacturing and proven effectiveness of products, these are primary growth stimulants of the market.

Global Cell Therapy Technologies Market: Drivers and RestraintsThe growth of cell therapy technologies market is highly driven by, increasing demand for clinical trials on oncology-oriented cell-based therapy, demand for advanced cell therapy instruments is increasing, owing to its affordability and sustainability, government and private organization , investing more funds in cell-based research therapy for life-style diseases such as diabetes, decrease in prices of stem cell therapies are leading to increased tendency of buyers towards cell therapy, existing companies are collaborating with research institute in order to best fit into regulatory model for cell therapies.Moreover, Healthcare practitioners uses stem cells obtained from bone marrow or blood for treatment of patients with cancer, blood disorders, and immune-related disorders and Development in cell banking facilities and resultant expansion of production, storage, and characterization of cells, these factors will drive the market of cell therapy technologies during forecast period.

On the other hand, the high cost of cell-based research and some ethical issue & legally controversial, are expected to hamper market growth of Cell Therapy Technologies during the forecast period

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AJune 2016, there were around 351 companies across the U.S. that were engaged in advertising unauthorized stem cell treatments at their clinics. Such clinics boosted the revenue in this market.in August 2017, the U.S. FDA announced increased enforcement of regulations and oversight of clinics involved in practicing unapproved stem cell therapies. This might hamper the revenue generation during the forecast period; nevertheless, it will allow safe and effective use of stem cell therapies.

Global Cell Therapy Technologies Market: Segmentation AnalysisOn the basis of product, the consumables segment had largest market share in 2018 and is expected to drive the cell therapy instruments market during forecast period at XX % CAGR owing to the huge demand for consumables in cell-based experiments and cancer research and increasing number of new product launches and consumables are essential for every step of cell processing. This is further expected to drive their adoption in the market. These factors will boost the market of Cell Therapy Technologies Market in upcoming years.

On the basis of process, the cell processing had largest market share in 2018 and is expected to grow at the highest CAGR during the forecast period owing to in cell processing stage,a use of cell therapy instruments and media at highest rate, mainly in culture media processing. This is a major factor will drive the market share during forecast period.

Global Cell Therapy Technologies Market: Regional AnalysisNorth America to held largest market share of the cell therapy technologies in 2018 and expected to grow at highest CAGR during forecast period owing to increasing R&D programs in the pharmaceutical and biotechnology industries. North America followed by Europe, Asia Pacific and Rest of the world (Row).

Browse Full Report with Facts and Figures of Cell Therapy Technologies Market Report at: https://www.maximizemarketresearch.com/market-report/global-cell-therapy-technologies-market/31531/

Scope of Global Cell Therapy Technologies Market

Global Cell Therapy Technologies Market, by Product

Consumables Equipment Systems & SoftwareGlobal Cell Therapy Technologies Market, by Cell Type

Human Cells Animal CellsGlobal Cell Therapy Technologies Market, by Process Stages

Cell Processing Cell Preservation, Distribution, and Handling Process Monitoring and Quality ControlGlobal Cell Therapy Technologies Market, by End Users

Life Science Research Companies Research InstitutesGlobal Cell Therapy Technologies Market, by Region

North America Europe Asia Pacific Middle East & Africa South AmericaKey players operating in the Global Cell Therapy Technologies Market

Beckman Coulter, Inc. Becton Dickinson and Company

MAJOR TOC OF THE REPORT

Chapter One: Cell Therapy Technologies Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Cell Therapy Technologies Market Competition, by Players

Chapter Four: Global Cell Therapy Technologies Market Size by Regions

Chapter Five: North America Cell Therapy Technologies Revenue by Countries

Chapter Six: Europe Cell Therapy Technologies Revenue by Countries

Chapter Seven: Asia-Pacific Cell Therapy Technologies Revenue by Countries

Chapter Eight: South America Cell Therapy Technologies Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Cell Therapy Technologies by Countries

Chapter Ten: Global Cell Therapy Technologies Market Segment by Type

Chapter Eleven: Global Cell Therapy Technologies Market Segment by Application

Chapter Twelve: Global Cell Therapy Technologies Market Size Forecast (2019-2026)

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Global Cell Therapy Technologies Market Industry Analysis and Forecast (2018-2026) - Markets Gazette

Recommendation and review posted by Bethany Smith

By: Portia Wofford

September is National Sickle Cell Awareness Month. Sickle Cell hits close to home for me. My nephew has the trait. My goddaughter has the disease and is frequently in crisis. Several other family members and friends are also fighting this illness.

One of the most common issues they all deal with is treatment by healthcare professionals particularly nurses when they seek treatment for this disease. Its shameful, but often their symptoms often cause them to be mistaken for:

These are all terms thrown at people with Sickle Cell Disease (SCD). The treatment or mistreatment of Sickle Cell patients contradicts our title as the most trusted profession.

So, nurses, what can we do? How do we advocate, treat, and care for this population of patients?

SCD is a group of red blood cell disorders. Unlike healthy red blood cells (RBC) which are round, patients with SCD have RBC that take on a C or sickle shape. Sickle cells die early which causes a constant shortage of red blood cells.

SDC commonly affects those whose ancestors came from:

The only cure for SCD is bone marrow or stem cell transplant.

When sickle cells travel through small blood vessels, they get stuck and clog the blood flow causing excruciating pain and other serious problems including:

SCD warriors and their caregivers report being stigmatized when they seek care. With patients showing signs as early as birth, nurses attitudes can contribute to negative stigmatization and may affect patients' response to sickle cell cues, potentially causing patients not to seek care and negatively impacting patient outcomes.

Cleverly Changing founder Elle Cole's daughter has SCD. She gives a brief description of an ER visit after a physicians assistant at their primary care office suggested her daughter go to the nearest hospital via ambulance.

Elle recalls, In the ER, the nurse was upset and asked why we were there and which clinic sent us. She stated my daughter didnt need any oxygen, the hematologist was busy (but would come in about an hour), and she needed to get a mucus sample. My daughter was scared and started to cry. Then, the nurse told four nurses to join her, and they proceeded to hold my daughter down and extract the mucus from her nostrils. I was completely terrified! My husband was at work. I felt alone and scared with my daughter.

One mom, Shaynise Robinson, drives three to four hours to seek care for her daughter, because of the lack of understanding from nurses and other healthcare professionals at their local hospital.

In an article posted on Pubmed, researchers found that sickle cell patients in one hospital waited for 60% longer to get pain medication although other patients reported less severe pain. They were also triaged into a less serious category. 63% of nurses surveyed said many patients with sickle cell are addicted to opioids, according to another study. But according to Dr. Alexis Thompson, president of the American Society of Hematology, rates of addiction among SCD patients are no higher than the general populations.

Elle Cole, who has a blog that brings awareness to SCD, wants nurses to be patient. Asking questions that indicate that you are genuinely concerned about your patients wellbeing can help open communication with your patient.

Shauna Chin, RN, says, In my experience, in addition to the pain, many patients with SCD exhibit symptoms of depression. The nurse needs to distinguish between solemness due to pain and solemness due to despondence. Many symptoms of depression go undiagnosed and can be remedied by encouraging health providers to engage in dialogue with the patient. She expresses that nurses can advocate best for patients with SCD by identifying early non-verbal signs and symptoms of pain and anticipate their patient's needs.

Ayana Spearman, RN, BSN, believes more education is needed during nursing school. Noting in the last five years, few co-workers were adequately informed about the disease. SCD was just 'glanced over' and not taught about in-depth, in nursing school, she says. Spearman believes that SCD patients need to have a holistic care approach.

Shaynise Robinson encourages nurses to go through diversity training. Give patients the benefit of the doubt. Understand that they are looking for pain relief and equal treatment, she says. Proper bedside manner is a concern for her as well.

Sickle Cell Disease doesn't just affect the body, but the holistic health of those with the disease. Education and awareness are critical for nurses to provide proper care. Shaynise Robinson leaves us with this: The same compassion thats shown to cancer patients should be shown to sickle cell patients.

To learn more about sickle cell visit:

Sickle Cell Disease Association of America

American Sickle Cell Anemia Association

Sickle Cell Society

Portia Wofford is a staff development and quality improvement nurse, content strategist, healthcare writer, entrepreneur, and nano-influencer. Chosen as a brand ambassador or collaborative partner for various organizations, Wofford strives to empower nurses by offering nurses resources for developmentwhile helping healthcare organizations and entrepreneurs create engaging content. Follow her on Instagram and Twitter for her latest.

See more here:
The Shocking Culture of Nurses and the Treatment of Patients with Sickle Cell - Nurse.org

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, today announced senior appointments to its leadership team. Sadik Kassim, PhD, a cell and gene therapy bioprocessing and translational research expert, joins Vor from Kite Pharma as Chief Technology Officer. Tirtha Chakraborty, PhD, a hematological and gene engineering research specialist with experience at Sana Biotechnology and CRISPR Therapeutics, joins as Vice President of Research. These new positions follow Vors recent move into an integrated headquarters in Cambridge, Mass., the appointment of Robert Ang, MBBS, MBA, as President and Chief Executive Officer and a $42 million Series A financing directed at developing Vors platform technology and advancing its pipeline of eHSC-based candidates.

Vor is bringing a fundamentally novel approach to hematopoietic stem cells to empower targeted cancer therapies, and we are rapidly building an industry-leading team to realize the value in this scientific foundation, said Dr. Ang. Dr. Kassim brings his substantial experience with the complex methods and processes that are required for manufacturing genetically-manipulated cell therapies, and Dr. Chakraborty provides deep expertise in hematology and genetic engineering. Their complementary knowledge will aid Vors expansion, platform development and the move towards our first Investigational New Drug filing for VOR33.

I am impressed that compelling in vivo data already supports the potential of Vors cellular engineering platform to protect healthy cells from antigen-directed therapies via antigen removal, said Dr. Kassim. This is especially noteworthy when therapeutic effectiveness is so often highly limited by co-location of target antigens on healthy immune cells, creating a huge opportunity for Vor to significantly broaden the applicability of these and future therapies.

Its exciting to join the Vor team during this period of accelerated expansion, said Dr. Chakraborty. As a geneticist and cell biologist, I look forward to developing this new approach to treat a range of devastating cancers, beginning with VOR33 in acute myeloid leukemia.

Dr. Kassim is a former Executive Director at Kite Pharma where he led the development of manufacturing processes for autologous CAR- and TCR-based gene-modified cell therapies. Prior to Kite, he served as Chief Scientific Officer at Mustang Bio, where he was the first employee and oversaw the foundational build-out of the companys preclinical and manufacturing activities. Prior to Mustang, Dr. Kassim was Head of Early Analytical Development for Novartis Cell and Gene Therapies Unit, where he contributed to the BLA and MAA filings for Kymriah. Earlier in his career, Dr. Kassim was a research biologist at the National Cancer Institute, where he was involved in early research and CMC work that led to the development of several first-in-human TCR and CAR-T products, including Kites Yescarta. Dr. Kassim has also conducted preclinical immunology research at Janssen and was a research fellow in the University of Pennsylvania Gene Therapy Program, where he led the initial discovery and preclinical studies for an AAV8 gene therapy for familial hypercholesterolemia, a program that is now in the clinic. Dr. Kassim earned his BS in Cell and Molecular Biology from Tulane University and received his PhD in Microbiology and Immunology from Louisiana State University.

Dr. Chakraborty joins Vor from Sana Biotechnology, where he served as the Vice President of Cell Therapy Research. Prior to Sana, Dr. Chakraborty was the Head of Hematology at CRISPR Therapeutics, where his teams work on hemoglobin disorders paved the way for the first clinical trial for the CRISPR industry. Before that, at Moderna Therapeutics, Dr. Chakraborty led synthetic mRNA platform technology research. He was trained as an RNA biologist and an immunologist during his postdoctoral research at Harvard Medical School. Dr. Chakraborty received his PhD from the Tata Institute of Fundamental Research in Mumbai, India.

About VOR33Vors lead engineered hematopoietic stem cell (eHSC) product candidate, VOR33, is in development for acute myeloid leukemia (AML). VOR33 is designed to produce healthy cells that lack the receptor CD33, thus enabling the targeting of AML cells through the CD33 antigen, while avoiding toxicity to the bone marrow. Currently, targeted therapies for AML and other liquid tumors can be limited by on-target toxicity. By rendering healthy cells invisible to CD33-targeted therapies, VOR33 aims to significantly improve the therapeutic window, utility and effectiveness of these AML therapies, with the potential to broaden clinical benefit to different patient populations.

About Vor BiopharmaVor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. Vors eHSCs are designed to generate healthy, fully functional cells with specific advantageous modifications, protecting healthy cells from the toxic effects of antigen-targeted therapies, while leaving tumor cells vulnerable.

Vors platform could potentially be used to change the treatment paradigm of both hematopoietic stem cell transplants and antigen-targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. A proof-of-concept study for Vors lead program has been published in Proceedings of the National Academy of Sciences.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil. Vor was founded by Dr. Mukherjee and PureTech Health and is supported by leading investors including 5AM Ventures and RA Capital Management, Johnson & Johnson Innovation JJDC, Inc. (JJDC), Novartis Institutes for BioMedical Research and Osage University Partners.

Read the rest here:
Vor Biopharma Hires Senior Cell and Gene Therapy Leaders as Chief Technology Officer and Vice President of Research - Business Wire

Recommendation and review posted by Bethany Smith

Shauna Stewart Douglas was struggling with infertility. It caught her and husband, John, by surprise.

I assumed that if you can get pregnant once, then you can get pregnant again, Douglas told Fox News.

She had become pregnant almost two years earlier with her daughter, but this time around even in vitro fertilization (IVF) wasnt working.

At age 35, Douglas found herself struggling with secondary infertility.

People always say to imagine what you want your kitchen table to look like in the future when youre thinking about how many kids to have, Douglas said.

And in my mind it has been my husband and all of our kids and that was all fading away. It was all going away.

Reports estimate that over 3 million couples in the United States face secondary infertility, which is the inability to become pregnant or to carry a baby to term after previously giving birth.

Dr. Kecia Gaither, an OB-GYN and director of perinatal services at NYC Health + Hospitals/Lincoln, says several conditions can cause secondary infertility like obesity, polycystic ovary syndrome (PCOS), the use of some medications, prior surgery, endometriosis, issues with cervical mucus and the age of both partners.

Many health conditions can be present without symptoms, until such a time as the couple wishes to become pregnant, Gaither told Fox News.

If there is an issue within a year of trying in couples less than 35 years of age or after six months in couples older than 35, its time to see your physician.

Douglas, founder of Permission to Profit, said they tried two rounds of IVF with the second time ending in miscarriage before they decided that they couldnt do it anymore.

Maybe it would have happened if we had kept on going and trying again and again, but I couldnt do it, I just I couldnt do the rollercoaster anymore.

Douglas said a medical condition, which she preferred not to disclose, and her age of almost 36 when they started trying for their second child, most likely led to her secondary infertility.

The biggest culprit typically in secondary infertility is the ovarian reserve, Dr. Brooke Hodes Wertz, a reproductive endocrinologist at NYU Langone Fertility Center told Fox News.

The ovary loses eggs in number and quality over time. So it gets harder to get pregnant over time.

Treatment for secondary infertility is the same as it is for primary infertility. Doctors should first start with an evaluation of both partners, Wertz said.

Youre going to do a semen analysis for the male partners, Wertz explained.

The females typically undergo blood testing that can look at how their ovaries are doing as well as testing to look at the inside of their cavity, whether its a hysterosalpingogram or an ultrasound, to look at the cavity and make sure the tubes are open.

The most common blood tests for women are called FSH (follicle-stimulating hormone), which give a reflection of the egg quality and AMH (anti-mullerian hormone) which show the number of eggs the patient has.

If there appears to be an issue, a doctor may recommend certain treatments at a fertility clinic.

We have simple treatments which involve oral medicine and often taking the sperm and releasing it very close to where the egg gets released, Wertz said.

And then we have more aggressive treatments like in vitro fertilization (IVF).

IVF typically uses fertility drugs to induce ovulation and then extracts the eggs and fertilizes them with sperm in a lab. Once the embryo forms, doctors then transfer the embryo into the uterus.

Wertz also recommended egg freezing as a way to possibly avoid secondary infertility.

We have the ability to freeze eggs and embryos when the ovaries are younger and put them back in at an older age when it would have been harder to get pregnant, Wertz said. A lot of women dont realize a couple of years makes a difference.

While there have been success stories among women who have frozen their eggs when they are over 40 years old, Wertz said it is preferable to freeze your eggs earlier in life, ideally before the age of 35.

Even though Douglas, now 41, didnt think that more rounds of IVF would give her family a second child and her daughter a sibling, there was another option for her adoption.

Families are made up in all kinds of different ways and, for us, we have a biological child and we have an adoptive child, Douglas said.

Going down that path was an incredibly beautiful thing because now we have my son, which is amazing and Im really grateful for that.

Excerpt from:
Secondary infertility: Why it happens to couples who have already had successful pregnancies - New York Post

Recommendation and review posted by Bethany Smith

Isabelle is a Newcomb College Institute Reproductive Rights & Reproductive Health intern and a Planned Parenthood Gulf Coast intern.

As abortion restrictions sweep across Louisiana, many are growing fearful that those seeking an abortion may now take matters into their own hands. These restrictions greatly decrease accessibility to medically safe abortions, putting individuals at risk of disability and death when making a choice about their own bodily autonomy.

With the passage of a series of abortion bans in Louisiana this summer, care options are scarce for reproductive health organizations like Planned Parenthood Gulf Coast, which provided for 13,185 patients statewide from 2017-18 in family planning. In particular, the passage of the heartbeat bill this summer prohibits abortions once a fetal heartbeat is detected, which often is as early as six weeks into pregnancy.

The Louisiana House of Representatives has also passed an amendment stating that no provision in the constitution can be used to protect the right to abortion or require the funding of abortion. Several more passages add further limitations to the mix, making it more difficult for physicians to transparently provide care for current and potential patients.

These restrictions will make self-managed abortion more common. Self-managed abortion is defined as an individual managing their own abortion without the guidance of a licensed medical professional. One is more likely to choose self-managed abortion should clinical care be unavailable, inaccessible or undesirable all aspects that are shaped by multiple socioeconomic, cultural and political factors.

When abortions were illegal in the 1950s, self-managed abortions were estimated at 200,000 to 1.2 million per year. These abortions were often carried out in back alleys where the individual often endured danger and abuse.

Other at-home treatments included ingesting malaria medicines, smearing potassium permanganate in the vagina and inserting foreign objects such as coat hangers. These caused chemical burns, shock and infection, and remain some of the lasting scars of a time before Roe v. Wade.

Today, the use of medication abortions is becoming increasingly common. While it has a higher success rate, many side effects some of which are fatal occur. In clinics, mifepristone and misoprostol are used together to manage abortions and have earned the moniker the abortion pill. Mifepristone blocks progesterone, the hormone required to preserve the uterine lining, while misoprostol aids in shedding to end the pregnancy. It is effective 96% of the time.

Yet, for self-managed abortions mifepristone is much more heavily regulated and often times only misoprostol is accessible, with a lower success rate of 85%. Misoprostol itself is typically prescribed for stomach ulcers and miscarriages.

Despite its status as an essential medicine by the World Health Organization, misoprostol is illegal to use without a prescription in the United States. It is still considered an over-the-counter drug in Mexico, however, under the name of Cyrux. Its $30 price tag makes it a much more affordable option than the abortion pill, which can fall around $500.

Information about this drug is circulated throughout the internet. The WHO provides a general protocol to follow on its website, for pregnancies up to 12 weeks.

Those with later-term pregnancies use forums as medical information to estimate proper dosages. These dosages depend on the stage of pregnancy, which often can be difficult information to obtain without medical guidance.

It is in these cases when exact details are scarce that misoprostol can be extremely harmful. The side effects include abdominal aches, intense pain, excessive bleeding and tearing of the uterus, with symptoms lasting longer the further along the pregnancy is. In cases of complications, it may be necessary to see a physician.

Since these symptoms mirror miscarriage symptoms and there are no tests that can detect misoprostol in the system, it is in the best legal interest of the patient to withhold this information from the doctor.

At least 20 arrests of women who have ended their pregnancies without clinical supervision have occurred. Now, many of the recent bills are taking a step further by criminalizing medical practitioners who assist women in clinical abortions, creating fear for all involved.

Self-managed abortions are often a last-ditch effort or the only affordable option for many individuals. Their symptoms are painful and long-lasting, without a guarantee of success.

Yet, with aggressive and harmful legislation closing in on reproductive health options and threatening medical practitioners in Louisiana, this will ultimately begin to limit the availability of clinical abortions and force individuals to make more risky choices.

A version of this article originally appeared in Newcomb College Institutes newsletter, ReproNews.

Read more from the original source:
Louisiana laws will lead to life-threatening self-managed abortions - Tulane Hullabaloo

Recommendation and review posted by Bethany Smith

Life for many transgender individuals involvesa constant struggle of being misgendered or referred to by the wrong pronoun or name. In many aspects of their lives, this ranges from annoying to personally invalidating. But when it comes to healthcare, it can be dangerous or even deadly.

Whats happening is that with a lot of transgender patients, the provider isnt being notified that the patients due for a procedure because these systems are not accurately pulling in the correct information, Chris Grasso, associate vice president for informatics and data services at the Fenway Institute, one of the worlds largest LGBTQ-focused health centers, told Healthcare IT News. Whats happening is when you see higher rates of cancer, lower rates of screening, a lot of it is because these systems arent actually incorporating things like anatomical inventory. So [for] someone whose sex is listed as female but they actually dont have a cervix, were sending them a letter that they really shouldnt be receiving that says Youre due for your pap smear.

Missed cancer screenings are just one example of a ways that mishandling data for transgender people puts them at risk. For instance, things like reference ranges for lab tests can also cause harm if the system pulls them for the wrong sex.

Having accurate information is also increasingly important as the EHR moves from a billing tool to a resourcefor population health.

The EHR was created for a documentation tool, but now we want it to be a lot smarter. We want it to provide us with more statistics, and more data, and how do we best care for these patients, said JoAnne Dombrowskas, MSHI, RN, manager of MGH's eCare clinical informatics team.

And, perhaps more important than any of that, when every staff person at a hospital greets a transgender person with the right name and uses the right pronouns, theyre more likely to stick around, come backand get the care that they need.

One special thing about this community ispeople talk to each other, Mitch Kellaway, a training specialist in MGH's Patient Access Services department, said. Whos safe? Who knew the word genderqueer when I said it and didnt bat an eye? And people, trans folks, nonbinary folks, genderqueer folks really notice those things. And it cant just be the clinicians. We [front desk and registration staff] get to them first, and they could walk out the door before they see a physician. So I tell the staff you really have a part in helping these folks get better care.

Grasso says that Fenway has been including SOGI information (sexual orientation and gender identity) in its own EHR since 1997, and started documenting it systematically about 10 years ago.

But a couple moves on the federal level have hastened a national adoption of SOGI fields in the EHR: In 2016, the US Bureau of Primary Healthcare at HRSA began requiring that all federally-qualified health centers collect and report that data. And in January 2018, thanks in part to the work of Grasso and her colleagues, having fields for SOGI data became a requirement for Meaningful Use.

Several years ago, the Healthy People 2020 initiative and the Institute of Medicine convened national experts, reviewed the existing literature and concluded that there really are unique health disparities experienced by gender and sexual minority people that are best addressed by making gender and sexual minority patients visible within healthcare, so we know what everyones SOGI is and can provide tailored patient-centered care based on that information, so that gender and sexual minority people can enjoy the same standard of health as the general population, saidAlex Keuroghlian, MD, who serves as director of the National LGBT Health Education Center at Fenway as well as the MGH Psychiatry Gender Identity Program.

Keuroghlian and Grasso agree that movement on the federal level has represented praiseworthy progress and have even conducted research thats starting to show that when the question is asked, LGBTQ+ individuals self-report at expected rates. But theres a lot of work still to do.

The first step is to have distinct fields in both the clinical and registration record for sex assigned at birth, legal sex, gender identity, and sexual orientation, as well as fields fornames used and pronouns. Legal names and sexes still need to live in the system because those are generally the names and sexes found on a patients insurance.

But Keuroghlian and Grasso believe the next step is anatomical inventoriesthat will put an end to assumptions about what organs someone has based on their sex.

A key part of this is to build in anatomical inventories that track body modifications people have had, so you could do preventative cancer screening based on the retained organs in someones body and not just on their chart sex, Keuroghlian said. Thats the future. Thats really where things need to go.

In addition to inventories, Grasso cited a need to make sure that interoperability initiatives like FHIR keep up with these new fields. The sensitivity of SOGI information, for patients who might not be out, adds another layer of complexity.

So if someones going to see a specialist outside your organization, they may feel like its helpful to share that information ahead of time or there may be times when they dont want to, she said. So we should be able to add those controls within the system.

Massachusetts General has been incorporating SOGI information into the clinical side of its EHR for a few years, but the push to get that information entered at registration is only about a year old. As of now, patients can even update some fields gender identity, name used and sex assigned at birth through the patient gateway.

The data infrastructure and training initiative runs parallel to the hospitals transgender health program, a special clinic offering primary care, hormone therapy treatmentsand mental healthcare to trans patients. This month, the clinic opens up pediatric as well as adult care options.

Primary care is really important, Robbie Goldstein, MD, the medical director of the Transgender Health Program, said. I dont think this works with just prescribing hormones and just having a space for people to come in and get testosterone, estrogen, whatever it may be. The reality is that there are a lot of things that come up related to gender. And to have a primary care doctor and a primary care practice who can manage those issues is incredibly helpful.

I also think theres a component of not making people tell their stories a million times, added Ariel Frey Vogel, MD, an internist and pediatrician who recently joined the program. Feeling known and heard. So when Robbie meets with a new patient, its with the social workers. Dallas [Ducar, who offers mental health care] is there if needed. Its all very integrated and embedded. In any medical care model theres a lot of things feeling very disjointed, so what were trying to do is make a model where it doesnt feel disjointed, where youre not repeating yourself over and over again, and where things feel really integrated.

Ducar, a psychiatric nurse practitioner who also joined the clinic recently, noted that the clinic has done a good job of creating a welcoming environment.

Something that Ive seen in my recent time here is, patients come in and visit even if they dont have an appointment with a provider, which is pretty spectacular, she said. They just want to spend their time here. With LGBTQ health and mental health theres not always been the best history. So being able to reduce that stigma and integrate with primary care allows for much easier conversations about mental health and for the conversation not just to be about whats going wrong with you, but whats going right with you.

Extending that welcome throughout the whole hospital is an ongoing project atMGH. That includes training both physicians and staff to not only use the systems, but to ask the right questions and project a safe and welcoming environment.

If were not asking these questions in a fully inclusive and affirming healthcare environment, it all falls flat, Keuroghlian said. You cant just do one thing and not the other. People need to feel safe [during phone intake] before they walk in the door, after they walk in the door, [with] what kind of materials, postersor pamphlets are in the waiting room. [They want to be asked] what is your sexual orientation, your gender identity, your sex assigned at birth. We also have to ask about name and pronouns and then transmit that information so that other staff can communicate correctly.

Preliminary research suggests that discomfort with SOGI questions tends to be more imagined than actual, Keuroghlian said.

There was a large study that found that 78 percent of staff were convinced patients would refuse to provide their sexual orientation, but the same study asked patients and only 10 percent of patients refused to provide their sexual orientation, he said. Another study had two demographic forms, one with SOGI information, one without. It found that there was no difference in people being offended by the form with SOGI questions than the one without, and the percentage offended at all was only 3 percent.

In Kellaways firsthand experience, when it comes to patients, the ones who need the new fields tend to be appreciative and the ones who dont will just brush the question off. And staff tends to be most concerned about learning the right terminology to ask the questions without offending anyone.

And when it comes to staff, their concerns tend to be more about "getting it right,"and evaporate once theyve been trained.

I think a lot of folks sit on this ability to add SOGI to the registration record even if they could because theyre make assumptions about discomfort, Kellaway said. Discomfort on the side of the patients being asked, and discomfort on the part of the staff that have to do the asking. From my experience, you would be surprised and you have to have faith in your staff. As people who care about patients, they feel the need to be educated in order to get it right, the terminology, the scripting. But they chose to get into healthcare for a reason.

Another assumption that isnt borne out by the data is about which sorts of hospitals are willing to adopt SOGI data nationwide.

When we looked at the data we actually looked at it by rural versus urban areas, Grasso said. And the rural areas actually did a better job of collecting and reporting data. And this is where the data becomes so critical, because I think it really can dispel some of those fallacies that gay people dont live everywhere or transgender people dont live everywhere, or people arent getting care at small places or dont want to provide that information in a small health center, because in fact they do, and theyre actually doing a better job of collecting it.

Massachusetts General is far from the only hospital in the country working on improving care for transgender and nonbinary patients. But the staff there is making a dedicated and visible effort and they hope other hospitals will follow suit.

If anyone is ever reading this article and feel like theyre waiting for that culture change, and dont know when its going to start, one thing that we really know is with the large rate of suicide in the trans community, one thing that brings that down to average levels across the nation is support, Ducar said. Its a team effort. Everyone has a part to play, and surely, no matter where you are in your journey everyone can offer support.

Goldstein added that the demographic trends will favor hospitals that go out of their way to serve this community

This is the right thing to do but its also the smart thing to do, because this is a growing population, he said. Right now about 0.5 percent of the adult population identifies as trans or nonbinary. Recent research has shown that about 2 percent of people under the age of 18 in the US identify as trans or nonbinary. This is a growing population that is going to come into the healthcare system and wed better get it right, because otherwise theyre going to go someplace else.

Ultimately, though, it isnt the business savvy that drives Goldstein, Frey-Vogel, Keuroghlian, Grasso, Ducar, Dombrowskasand Kellaway.

We have a mission to take care of everyone whos around us, and that includes the trans and nonbinary communities, it includes anyone who walks in that door, said Goldstein. So every time I meet with hospital leadership about trans health and why its so important, I always say This is part of our fundamental mission. We are doing this because trans folks are walking through the door every single day. They need to have a bathroom they feel comfortable going to, they need to have an EHR that understands who they are, and they need to have a doctor or nurse taking care of them whos capable of understanding who they are and what is their gender identity.

Continue reading here:
More inclusive EHRs can help extend welcome, save transgender lives - Healthcare IT News

Recommendation and review posted by Bethany Smith

The prime objective of Endocrine Testing Market report is to help the user understand the market in terms of its definition, segmentation, market potential, influential trends, and the challenges that the market is facing. Deep researches and analysis were done during the preparation of the Endocrine Testing Market report. The readers will find this report very helpful in understanding the market in depth. The facts and data are represented in the report using diagrams, graphs, pie charts, and other pictorial representations.

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By TestEstradiol (E2) Test, Follicle Stimulating Hormone (FSH) Test, Human Chorionic Gonadotropin (hCG) Test, Luteinizing Hormone (LH) Test, Dehydroepiandrosterone sulfate (DHEAS) Test, Progesterone Test, Testosterone Test, Thyroid Stimulating Hormone (TSH) Test, Others (Gastrin, Thymosin, Secretin, etc.)

By TechnologyTandem Mass spectrometry, Immunoassay (Enzyme immunoassays, Radioimmunoassays (RIA)) Technologies, Monoclonal and Polyclonal Antibody Technologies, Sensor (Electrochemical, Biosensors, etc.) Technologies, Clinical Chemistry Technologies, Others (Liquid Chromatography + Mass Spectrometry (LC-MS),

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Major Chapters covered in the report:

Here is the original post:
Endocrine Testing Market Cost Analysis, Revenue And Gross Margin Analysis With Its Important Types And Application 2019 - The Washington Observer

Recommendation and review posted by Bethany Smith

The future of genetically modified babies may lie in the hands of Russian president Vladimir Putin,Bloomberg reported over the weekend.

Secret summit:According to Bloomberg, top Russian geneticists held a secret meeting this summer with government health officials in Moscow to debate a bid by a scientist there, Denis Rebrikov, to create babies genetically modified with the gene-editing technology CRISPR.

The first such children were born in China last year as part of a project to make HIV-resistant humans. That undertaking was halted amid pointed criticism of its ethical failings and a criminal investigation.

Putins choice:The question now is whether Russia will grab the CRISPR baton where China dropped it. Dmitry Peskov, the spokesman for Russias leader, declined to give Bloomberg a position, saying gene editing is not a presidential issue.

Putin has already made some comments about gene editing, likening the technology to a nuclear bomb and citing the possibility ofcreating soldiers who feel no pain. According to Bloomberg, Putin last year directed $2 billion to be spent on genetic research that he said will determine the future of the whole world.

Read full, original post: Putin could decide for the world on CRISPR babies

Read the rest here:
Will the future of CRISPR babies be decided by Russian president Vladimir Putin? - Genetic Literacy Project

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Illinois State University researchers are part of a team thats creating a new crop that could help both the environment and farmers' bottom lines.

TheU.S. Department of Agricultureis givingthem $10 million for researchthat will lead to the planting and harvesting of pennycress, a penny-shaped weed that grows in the spring, as a winter cover crop, which processors can then convert into fuel.

Pennycress has a number of natural attributes that makes it perfect for being a crop, Illinois State University genetics professor John Sedbrook said on WGLT's Sound Ideas. It has extreme cold tolerance, it soaks up the nitrogen so it keeps nitrogen from running into the streams to keep streams clean, and its related to canola.

Cover crops tend to have more environmental than monetary value and provides soil health and natural benefits. Sedbrook and the research team are looking to change that.

To achieve the goal of the project, researchers are using gene editing, called CRISPR, to change the very nature of the original plant.

With CRISPRthis is game changing and going to improve our lives in a lot of different ways, not just crop improvement but treating human diseases. Weve been able to apply CRISPR to rapidly improve pennycress genetically, Sedbrook said.

With just two genetic changes, the team has been able to make pennycress oil and meal edible.

Not only can we use it for food, Sedbrook said, we can also use it for making biodiesel or jet fuel. Its really quite versatile. Another change we made was reducing the fiber content in the seed so the meal has the same nutritional value as canola adding value to pennycress along with the breeding program to get the yields higher where now its economical.

That means farmers can benefit off the cover crop, putting more money in their pockets during what Sedbrook calls a challenging time in farming.

Sedbrook also said converting the oil to biodiesel is not that difficult. With current technology and modern techniques a simple plant like pennycress can be converted to even jet fuel.

But what does it mean to domesticate a plant?

In the past it took thousands of years for cavemen to wait for the right genetic change to come along, Sedbrook said. For wheat there is a handful of changes and for corn there are six changes they have identified that changed the weed teosinte into what we know as corn.

It takes hundreds of thousands of years for evolutionary changes in plants. But with genetic science, changes can be made rapidly.

Sedbrook said the demand for the new strain of pennycresscalledCoverCressis already there.

Theres the old saying build it and theyll come," he said. "There are people just waiting for us to produce this oil and theyre ready to process it."

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A recent study published in JAMA Oncology analyzed outcomes for patients with early breast cancer with a high 21-gene recurrence score who underwent adjuvant chemotherapy plus endocrine therapy. The outcomes were superior compared to those associated with endocrine therapy alone.

The 21-gene recurrence score (RS) assay provides prognostic information for distant recurrence in hormonereceptor-positive, ERBB2-negative early breast cancer that is independent of clinicopathologic features, and is also predictive of chemotherapy benefit when the RS is high, the authors reported, adding, In this report we provide a descriptive analysis of clinical outcomes, characteristics, and adjuvant chemotherapy regimens for patients with an RS of 26 to 100 assigned to receive chemotherapy, including regimens largely including taxanes and/or anthracyclines, plus endocrine therapy.

The study was a secondary analysis of outcomes associated with the TAILORx trial. The multicenter, randomized clinical trial included women with hormone receptorpositive, ERBB2-negative, axillary node-negative breast cancer who had a high 21-gene recurrence score of 26 to 100. Patients received adjuvant chemotherapy, selected by the treating doctor, in conjunction with endocrine therapy. The primary outcome measure was freedom from breast cancer recurrence at a distant site and freedom from recurrence, second primary cancer, and deathor invasive disease-free survival (IDFS).

Of the 9,719 eligible total patients (mean age [range] 56 [23-75] years), 1,389 (14%) had a recurrence score between 26 and 100; of the high recurrence score group, 598 (42%) had a score between 26 and 30 and 791 (58%) had a score > 30. Regarding chemotherapy regimens, most patients (n = 589, 42%) received docetaxel/cyclophosphamide; 334 (24%) received an anthracycline without a taxane, 244 (18%) received an anthracycline and taxane, 52 (4%) received cyclophosphamide/methotrexate/5-fluorouracil, 81 (6%) received other regimens, and 89 (6%) did not receive chemotherapy.

After five years, the freedom from breast cancer recurrence at a distant site rate was about 93.0% (standard error [SE], 0.8%). Breast cancer recurrence at a distant and/or local regional site freedom was about 91.0% (SE, 0.8%); IDFS was about 87.6% (SE, 1.0%), and overall survival was about 95.9% (SE, 0.6%).

The authors wrote in sum, The estimated rate of freedom from recurrence of breast cancer at a distant site in women with an RS of 26 to 100 treated largely with taxane and/or anthracycline-containing adjuvant chemotherapy regimens plus endocrine therapy in the prospective TAILORx trial was 93% at 5 years, an outcome better than expected with endocrine therapy alone in this population.

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Mathew Knowles, father of Beyonc,recently announcedthat hes being treated for breast cancer coming as a surprise to many who still dont understand that, while its relatively rare, men, just like women, can be diagnosed with the disease.

I need men to speak out if theyve had breast cancer, hetold Good Morning America on Wednesday. I need them to let people know they have the disease, so we can get correct numbers and better research Men want to keep it hidden, because we feel embarrassed and theres no reason for that.

Knowles reports that he had a unilateral mastectomy the surgical removal of all breast tissue in July, and plans to have his second breast removed in January. That, he says, should reduce the risk of recurrence meaning a metastasis, which is the spreading of breast cancer cells to another part of the body, for which there is no cure from 5 percent to 2 percent.

Breast Cancer Awareness Monthis October, making it the ideal time to learn more about the disease, which, while rare, will affect about 1 in 833 men and 1 in 8 women over a lifetime. In 2019, there will be about 2,670 new cases diagnosed in U.S. males, according to theAmerican Cancer Society(compared with the estimated 268,600 cases in women).

Heres what else you need to know about male breast cancer.

Yes, men have breasts.

We are mammals also, Harold Burstein, MD, a breast oncologist with theDana-Farber Cancer Institutein Boston, tells Yahoo Lifestyle. But while men do have breasts, we obviously dont undergo the secondary developmental changes that come for women with puberty and then pregnancy, which could contribute to the confusion around men having breast tissue, he says. But mostly why you dont hear about men having breast cancer so much is because the incidence is very rare, with roughly one male case for ever 100 womens cases.

Its the same disease that affects women.

Fundamentally, the breast cancers are more or less the same, Burstein says. Up to 90 percent of cases in men have thehormone-receptor statusknown as estrogen-receptor positive and HER2 negative, he says. The male symptoms a painless lump or thickening of breast tissue; changes to skin in the breast area (such as dimpling or scaling), changes in nipple appearance; or nipple discharge (as with Knowles) are the same as in women. The treatment approach, such as surgery, radiation, chemotherapy or hormone therapy, are also the same. Ounce for ounce, Burstein says, men have outcomes very similar to that of women although since we dont do mammograms on men, sometimes its found later.

Thats why its important for men to have a general awareness of their bodies, and, if they notice changes or something that doesnt seem right, Burstein says, they should head to their doctor and have it checked out.

Our slogan is, Dont be afraid to touch yourself for a reason, explains Bret Miller, founder of theMale Breast Cancer Coalition.

You dont hear about male breast cancer too much because it comes with a powerful, silencing stigma.

Its a womens disease, its emasculating, men cant get it because we dont have boobs, are all reasons that some men associate male breast cancer with shame, according to Miller, a breast cancer survivor. He founded the coalition as a supportive resource six years ago, and says, There still are people that do not know that men can get breast cancer. Its crazy. Thats why its important for people like Knowles to speak out about being diagnosed, adds Miller to help build awareness for early detection, to help create strength in numbers and, ultimately, to fuel the push for more research thats specific to breast cancer affecting men.

Theres historically been a lack of research into male breast cancer, but that may be about to change.

Such research is now being officially pushed by the Food and Drug Administration (FDA), according to anAugust draft guidance encouraging the inclusion of male patients in breast cancer clinical trials. Less than one percent of all breast cancer cases occur in men, but men are more likely to be diagnosed at an older age and have a more advanced stage of disease. As breast cancer in men is rare, they have typically not been included in clinical trials for breast cancer treatment. This has led to a lack of data, so their treatment is generally based upon studies and data collected in women, notedRichard Pazdur, MD, director of the FDAs Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDAs Center for Drug Evaluation and Research. While some FDA-approved treatments are gender-neutral in their indication, many therapies are only approved for women and further data may be necessary to support labeling indications for men.

It's just as important for men as it is for women to get tested for BRCA gene mutations.

One of the rare known causes of breast cancer in men is a hereditary predisposition, accounting for a somewhat larger percentage of male breast cancer than female breast cancer, Burstein says. (Other risk factors include aging, family history, having the congenitalKlinefelter syndrome, and radiation exposure.) And Knowles has shared that hisBRCA test a blood test using DNA to identify mutations in breast cancer susceptibility genes (BRCA1 and BRCA1) showed he has a mutation on his BRCA2. As a result, he said he has four things to be concerned about: prostate cancer, pancreatic cancer, melanoma and breast cancer. The rest of my life I have to be very aware and conscious and do all of the early detection: constant mammograms, constant prostate exams, MRIs.

Burstein stresses that all men diagnosed with breast cancer should be tested for gene mutations, which mostly relates to opportunities for prevention such as indicating a double mastectomy, instead of single, with the second being preventative. Also, since having the mutation raises the risk for the other cancers Knowles mentioned, knowing you have it would theoretically push men to get regular screenings.

Those with a BRCA2 mutation, in particular, will have about a 6 in 100 lifetime risk of developing breast cancer,Leigha Senter-Jamieson, a licensed genetic counselor at the Ohio State University Comprehensive Cancer Center, tells Yahoo Lifestyle. So most wont get it, she says, but that risk is still much, much higher than the usual risk for men, which is about 1 in 100.

Sometimes men arent asked about their family history of breast cancer or ovarian cancer at their checkup, Senter-Jamieson notes even though the answers could indicate whether or not they should be tested for BRCA gene mutations. Something else men are not often aware of when it comes to these mutations, she says, is that it impacts their kids the same way it would with a woman yet another reason to get tested.

When high-profile people share their stories, it does a good thing, she adds, both for the people getting tested and for people, like myself, asking the questions of patients.

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Targeted treatments approved this year for three aggressive breast cancers illustrate the ongoing advances in the field of medical oncology, local cancer doctors say.

There is more hope in the world of cancer, Medical Director Ibrahim Sbeitan said at Conemaugh Cancer Center in Johnstown. We dont lose as many of our patients to cancer. Its much better for cancer than 10 years ago.

One of the new treatments was proven through clinical trials that included patients with UPMC Hillman Cancer Centers, which has locations in Johnstown, Altoona, Indiana and Latrobe.

We were part of that trial through Hillman and UPMC, Clinical Services Director Rashid Awan said at UPMC Hillman Cancer Center, John P. Murtha Pavilion, 337 Somerset. St., Johnstown.

We are trying to be innovative for our patients right here in Johnstown, he said.

The three new medical oncology drugs target specific characteristics in previously hard-to-treat breast cancer and other cancers.

There are three groups of receptors that are targets for cancer drugs.

The Centers for Disease Control and Prevention website compares them to three locks on a door. One is a receptor for the hormone estrogen; another is a receptor for the hormone progesterone; the third is human epidermal growth factor protein, abbreviated HER2.

If the cancer has any of those receptors, doctors have developed keys to open them for targeted treatments.

Two of the new treatments alpelisib and ribociclib are improving the odds for patients with metastatic cancer that has receptors for the hormones, but not for HER2.

The third atezolizumab has been shown to help those with triple-negative breast cancer, which has been difficult to unlock because it is negative for all three receptors.

Hormone therapy has been the standard of care for patients with metastatic hormone-receptor positive, HER2 negative cancer. However, after initial improvement with the hormone medicine, the cancer has usually advanced.

Bodys own defense system

Prior to the Food and Drug Administrations approval of atezolizumab in March, chemotherapy was still the main option for triple-negative breast cancer, Sbeitan said.

The drug, sold under the brand name Tecentriq, is the first immunotherapy drug approved for triple-negative cancer.

Immunotherapy is starting to break into triple negative, Sbeitan said.

Although he acknowledges that the treatment hasnt been around long enough to show long-term survival, Sbeitan says it has slowed progression with less side effects.

Block that protein

The UPMC Hillman patients were part of the MONALEESA clinical trials funded by Novartis Pharmaceuticals Corp. of Switzerland for its Kisqali brand of ribociclib.

Awan explains ribociclib and other CDK4/6 inhibitors block the CDK proteins to slow cell growth.

Cancer cells use the protein to grow, he said. Its like fuel for them. This blocks the fuel and there is very good response.

The drug is also significant because the study focused on younger, premenopausal breast cancer patients, Physicians Weekly said, citing Dr. Sara Hurvitz of the University of Southern California Los Angeles Janssen Comprehensive Cancer Center. For women under 45, breast cancer is a leading cause of cancer deaths.

This is the first study to demonstrate improved survival for a targeted therapy, Hurvitz said at an American Society of Clinical Oncology press conference covered by Physicians Weekly.

Find the mutation

The third new treatment uses a different approach to target a sub-group of advanced-stage hormone-positive, HER2 negative patients.

Alpelisib, sold as Piqray, targets cancer with a mutation known as PIK3CA that has grown despite hormonal treatment.

The drug is used for postmenopausal women and men with breast cancer.

Normal cells became cancer because a mutation took place, Sbeitan said. We try to find the mutation and reset the mutation.

Block by block

Sbeitans colleague, medical oncologist Sheetal Higbee, says alpelisib is one of the second-line cancer drugs that is used for patients who dont respond to the standard treatments. The second- and third-line drugs help patients live longer while more treatments are developed.

Their life with cancer is a lot more bearable, she said at the Conemaugh Cancer Center. We have been giving them hope because we have so many options.

Progress may seem slow, Sbeitan admits, but the results have begun to show.

Cancer treatment is a block by block treatment, Sbeitan said.

You have to have the attitude that each treatment is going to add to the last.

One of Conemaughs patients has been receiving treatments for metabolic cancer since 2006.

She is fully functioning, Sbeitan said.

Although treatments for some cancer is curative up front, Sbeitan says most require ongoing treatments. And there may be setbacks, he adds.

For those patients, the goal is to allow them to enjoy a longer quality of life while keeping the cancer at bay.

They are living cancer-free, he said. We dont use the word cure.

Awan compares it to chronic conditions such as diabetes and heart disease. All can be controlled with medication and lifestyle changes.

They can live with the disease and have a normal life, he said.

Slowing progress

It is getting more challenging to develop new treatments because of existing cancer care, successes, medical oncologist Dr. Michael Voloshin said at UPMC Hillman in Johnstown.

Researchers dont want to be depriving cancer patients of the proven standard of care in order to test experimental treatments.

We have a lot well mapped out on how to treat cancer, Voloshin said.

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A recent article published by Catholic news outlet LifeSiteNews alleged that the drugs used to treat gender dysphoria in some transgender children are linked to thousands of deaths.

The story went viral on right-wing news websites such as the Christian Post and the Daily Wire. According to CrowdTangle, a social media metric platform, these posts including shares by Daily Wire founder Ben Shapiro and commentator Matt Walsh are currently some of the top performing LGBTQ-related content on Facebook and Twitter.

The problem is: the thousands of people who die while taking these drugs are likely the terminally ill cancer patients who receive hormone blockers to fight hormone-sensitive cancers, like prostate cancer, according to experts.

Joshua Safer, a professor of medicine and the executive director of the Mt. Sinai Center for Transgender Medicine and Surgery, said Lupron, or leoprolide acetate, is used for treating precocious puberty, infertility and certain types of cancer, particularly prostate cancer.

Prostate cancer is worsened by the presence of certain hormones, so people fighting this disease are sometimes given hormone blockers puberty blockers to slow the cancers progression.

I think all they did is went into the FDA database and looked at reports, Safer said. Theres no study here, thats just a big smorgasbord of reports and so the problem with that is you don't even know that those deaths are connected to the agent they are reported to be connected to.

Much more likely, Safer said, is that the 6,370 deaths over four decades the FDA lists as connected to this drug are in terminally ill cancer patients who are prescribed Lupron as a palliative, not curative, treatment.

They wouldnt even be using it if they werent at risk of death, Safer said of the drugs use in prostate cancer patients.

The American Cancer Association estimates that there are roughly 30,000 deaths from prostate cancer annually in the United States.

The original LifeSiteNews story, which was modified after initial publication, said that the UKs National Health Service is investigating these drugs. A spokesperson for the NHS told NBC News that no special review is being launched into the use of this drug for the treatment of gender dysphoria and noted that all transgender health care services are regularly reviewed.

The NHS own guidelines for the treatment of children with gender dysphoria notes that psychological support and puberty suppression have both been shown to be associated with an improved global psychosocial functioning in youth. Both interventions may be considered effective in the clinical care of psychosocial functioning difficulties in adolescents with [gender dysphoria].

Every decision in medicine involves weighing risks and benefits, said Jack Turban, a resident physician in psychiatry who researches transgender youth at the Massachusetts General Hospital. Turban said that for trans youth, the potential mental health benefits of pubertal suppression far outweigh any potential risks.

Allowing puberty to progress is not a neutral decision for many transgender youth, Turban said. Many of these youth see their mental health drastically deteriorate as puberty starts to progress. While pubertal suppression is reversible, puberty itself is not.

Heron Greenesmith, a senior research analyst at Political Research Associates, tracks anti-transgender rhetoric in mainstream media and said the article exemplifies LifeSiteNews' membership in the Christian-right anti-transgender disinformation ecosystem.

LifeSite platforms the small number of anti-trans researchers, academics, and right-wing professional associations, giving their work a veneer of scientific validity, Greenesmith said. Advocacy organizations can then cite LifeSite, in turn giving their advocacy a veneer of journalistic independence."

Gillian Branstetter, a spokesperson for the National Center for Transgender Equality, said the publication of this article was dangerous.

Transgender youth face a public health crisis in this country, and families must already fight through significant barriers to accessing adequate health care, Branstetter wrote. Much like vaccines, I would encourage news outlets and social media to be extremely sensitive to the risks posed by lies about transition-related health care promoted by bad actors.

NBC News has reached out to LifeSiteNews for comment.

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Sgt. Anna Lange filed a lawsuit against the county where she works in Georgia for refusing to allow her health insurance plan to cover gender-affirmation surgery. Audra Melton for NPR hide caption

Sgt. Anna Lange filed a lawsuit against the county where she works in Georgia for refusing to allow her health insurance plan to cover gender-affirmation surgery.

A sheriff's deputy in Perry, Ga., filed a lawsuit in federal court Wednesday against the county where she works for refusing to allow her health insurance plan to cover her gender-affirmation surgery.

Sgt. Anna Lange came out as transgender in 2017, after working in the Houston County Sheriff's Office since 2006. She has taken hormone therapy and outwardly changed her appearance over the past three years to treat gender dysphoria, the distress resulting from the mismatch between her sex assigned at birth and her gender identity.

Her next step was going to be gender-affirmation surgery, but that plan came to a halt when, as NPR previously reported, her insurance provider denied coverage for the procedure, based on an exclusion specified by her employer.

Now, Lange is suing the Houston County Board of Commissioners to remove that exclusion. Early on Wednesday, she and her lawyer, Noah Lewis of the Transgender Legal Defense and Education Fund, filed suit in U.S. District Court in Macon, Ga., alleging unlawful discrimination under federal and state equal protection clauses, Title VII of the Civil Rights Act and the Americans with Disabilities Act.

County officials did not return calls for comment.

Lange's case is the latest in the U.S. to challenge the exclusion of transgender care from state and municipal employee insurance plans and could create legal precedents for cases across the South.

Other transgender people have won similar fights elsewhere. The managers of Wisconsin's state employee insurance program excluded transgender employees from coverage but later reversed that decision. Separately, two University of Wisconsin employees sued the state and won. Another lawsuit successfully challenged transgender exclusions in Wisconsin's Medicaid plan.

Earlier this year, Jesse Vroegh, a transgender employee of the Iowa Department of Corrections, won a lawsuit he'd filed after being denied coverage by his employer's health insurance plan.

And in Georgia, the state's university system recently settled an insurance exclusion claim for gender-affirmation surgery filed by Skyler Jay, known for his appearance on the Netflix series Queer Eye. In addition to changing its employee health plan to be inclusive of transgender care, the university system paid Jay $100,000 in damages.

"The university clearly agreed that it was discrimination," says Lange's attorney, Lewis, who also represented Jay. "That's why they wanted to do the right thing and remove the exclusion."

In 2011, another Georgia case, Glenn v. Brumby, set the legal precedent protecting transgender people from employment discrimination. However, that case did not address discrimination in employee benefits and, like Jay's case, many that deal with benefits have been settled out of court, according to Lewis.

The Affordable Care Act, which took effect in 2014, specifically prohibits discrimination by health insurance issuers on the basis of gender identity, and Title VII of the Civil Rights Act also has been interpreted to prohibit such discrimination.

Despite broad legal consensus that transgender insurance exclusions are unlawful, state and local governments continue to pursue expensive legal fights to preserve them. The issue remains contentious for many social conservatives.

"Ultimately, what's happening is that, politically, I presume they think it's unpopular or they think they have to defend" the law or regulation, says John Knight, an attorney with the American Civil Liberties Union.

Resisting paying for such care can be more expensive than providing it. Not including the costs of state attorneys' salaries or appeals, Wisconsin's litigation against the employees of its university system cost the state more than $845,000, while Iowa's cost about $125,000.

Furthermore, the cost of managing untreated gender dysphoria can outweigh the costs of providing transgender-inclusive health care, according to a 2015 study.

"Given the small number of people who actually need this kind of care and the large pool of people, it will have absolutely no impact on the total cost of insurance for any state," Knight says.

While settlements such as Jay's may be good for individuals, they do not require institutions to admit wrongdoing and do not result in a legal precedent that other, lower courts must follow.

"The court doesn't have to look at that settlement and say, 'Oh, this was discrimination,' " Lewis says. "Transgender workers in the South are being left behind, which is why we're seeking a court ruling to clearly establish that this conduct is unlawful throughout the South."

Lange's suit argues that the county's exclusion of transgender health care from coverage was deliberate: In documents Lewis obtained under the Freedom of Information Act, Kenneth Carter, the county's personnel director, opted out of compliance with Section 1557 of the Affordable Care Act, which prohibits discrimination by health programs on the basis of gender identity.

"Houston County will be responsible for any penalties that result if the plan is determined to be noncompliant," he wrote in a letter to a representative of Anthem Blue Cross and Blue Shield, which administers the plan.

Carter did not return calls for comment.

Lange's case could end up before the 11th U.S. Circuit Court of Appeals, yielding a decision that could influence other courts in Alabama, Florida and Georgia. And, if the ruling is in Lange's favor, Lewis says, that would signal that transgender exclusions should be removed nationwide.

Lange's suit argues that the county's exclusion of transgender health care from coverage was deliberate. Audra Melton for NPR hide caption

Lange's suit argues that the county's exclusion of transgender health care from coverage was deliberate.

In its next term, the U.S. Supreme Court will hear three cases that will determine workplace protections of LGBTQ individuals, including one case involving a transgender woman.

Lange says she merely wants the same protections everyone else has. The co-workers with whom she shares a health plan might have used "something on the policy that I may never use or need, but it's covered," she says. "When it's finally something that I need that one of my co-workers will probably never use or need, mine's excluded. And that's just not fair."

Keren Landman, a practicing physician and writer based in Atlanta, covers topics in medicine and public health. Kaiser Health News is a nonprofit, editorially independent program of the Kaiser Family Foundation. KHN is not affiliated with Kaiser Permanente.

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