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Despite what social pressures may lead us to believe, theres no magical age when it comes to getting pregnant and starting a family. When I was pregnant with my first child, I was 27. For me, it felt like the perfect age I wasnt too young and I wasnt too old, either. Plus, by starting in my twenties, I figured I had plenty of time to give my baby siblings down the road. My husband and I were married for two years first, withstable jobs and a decent amount of savings in the bank. The timing felt more than just right.

That said, once my daughter arrived, things got complicated. As a freelance writer, I dont work full-time in an office. And if I did, I couldnt commit to being a full-time mom at home in the way I do now. Many years (and one kid)later, Im honestly still trying to figure out this whole work-lifebalance thing, and how that plays into my role as a mother. And Im certainly not alone.

For many couples, the question over when to start a family and how it might impact their career paths in the process is fraught with many what ifs.According to New York Magazine, thats also the reason why so many millennial couples are now choosing to freeze their eggs, so they can put parenting on hold while they chase their corporate dreams.

Its called elective freezing, and has become so popular that there are actuallyboutique egg freezing clinics in big cities all across the country that cater to couples whod like to delay parenthood a bit longer. One such clinic, Extend Fertility, is located in midtown Manhattan inNew York City, where its solely focused on removing and storing a womans eggs, as opposed to just treating infertility and offering in-vitro fertilization.

During theegg freezing process, a patient undergoes the same hormone-injection process as they would during in-vitro fertilization. The only difference is that following egg retrieval, they are frozen for a period of time before theyre thawed, fertilized, and transferred to the uterus as embryos.

Trust Me, There's Never a "Perfect" Time to Have Kids

According to New York Magazine, the boutique clinics also solve another issue: Many women who choose this process have expressed mixed emotions about going to a clinic where other women are actively tryingto get pregnant. Some even say it makes them feel selfish for wanting to put motherhood on hold while seeing others struggle to conceive at the same time.

As Martin Varsavsky, an entrepreneur who raised over $200 million for his fertility start-up, told the mag:

Its sort of like we are customizing the experience for young working women who want to get in and out. These are not women who are traumatized because they cant have a child.

Makes sense, right?

As hard as the work-life equation is, I cant say that I wish I had waited longer before having children myself. Ive always told my friends and family that when my children are old enough to take care of their physical needs on their own, Ill go back to work full-time. Theres no such thing as too late.

But my own experience aside, I do see how freezing your eggs can make the timing a little easier, in a whole lot of ways. And for single women entering their thirties, I can certainly see how the idea of egg freezing is especially appealing, because it gives them more time to find the right partner without having to worry about their ticking biological clocks. After all, peak fertility years do coincide with our peak career-buildingones for better or worse. But hey, if theres a way to extend those years (and take off a little stress in the process), then I say, why not?

Article Posted 9 hours Ago

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Boutique Egg Freezing Clinics Are the Latest Trend for Those Looking to Delay Parenthood - Babble (blog)

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Science Daily

New autoimmune disease triggered by thymomas Science Daily A Japanese research group has discovered that a newly-identified autoimmune endocrine disease that leads to hypopituitarism is caused by thymomas (a type of tumor originating from the thymic gland). These underlying mechanisms could help to ...

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New autoimmune disease triggered by thymomas - Science Daily

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Osteoporosis is a complication of androgen deprivation therapy (ADT) in men with prostate carcinoma. The best defense against osteoporosis in prostate cancer is to identify patients with a high risk for fracture during the first clinical visit, select an effective anti-osteoporosis agent, and advise the patient to change his lifestyle and diet to prevent further bone loss. New agents include denosumab, a human monoclonal antibody that inhibits the RANK ligand (RANKL). RANKL promotes the formation, activity, and survival of osteoclasts and, thus, supports the breakdown of bone.

This is a multicenter, randomized, double-blind prospective study on use of denosumab versus alendronate in the therapy of secondary osteoporosis related to ADT in prostate cancer patients in three European countries (Italy, France, Switzerland).

In this 24-month observation study we enrolled 234 patients with diagnosis of osteoporosis underwent ADT for prostate cancer. All patients aged 55 years and had a dual-energy X-ray absorptiometry (DEXA) T-score <-1.0 (hip or spine, measured within last 2 years) and 1 fragility fracture. Patients were randomly assigned 1:1 to receive denosumab 60 mg subcutaneously every 6 months or alendronate (70 mg weekly) for 2 years. All patient received supplemental vitamin D (600 IU per day) and supplemental calcium to maintain a calcium intake of 1200 mg per day. Effectiveness of therapy in both groups (denosumab group and alendronate group) was assessed by changes in bone turnover markers (BTMs), Bone Mineral Density (BMD), fracture incidence, Visual Analogue Scale (VAS) score for back pain, and Short Form-8 (SF-8TM) health survey score for health-related quality of life (HRQoL). Percent changes from baseline in BTMs and BMD were assessed using the paired t test; a P-value 0.05). Mean changes in BMD at final follow-up differed significantly between two groups. BMD changes at the lumbar spine at 24 months were 5.6% with denosumab vs -1.1% with alendronate (P<0.001). New vertebral fractures developed in fewer patients in the denosumab group than in the alendronate group during the 24-month period, although this difference was not significant (P=0.10). Back pain significantly (P<0.001) improved from baseline at all time points during the study in both study groups. SF-8 health survey scores significantly improved following treatment with both drugs. Incidence of adverse drug reactions were similar in both groups.

In our study denosumab and alendronate showed similar clinical efficacy in the therapy of ADT-related osteoporosis in men with prostate carcinoma; both drugs provided significant improvements in back pain and general health conditions. Denosumab showed significant increase of BTMs and BMD than alendronate with lower rate of new vertebral fractures.

Clinical cases in mineral and bone metabolism : the official journal of the Italian Society of Osteoporosis, Mineral Metabolism, and Skeletal Diseases. 2017 Feb 10 [Epub]

Carlo Doria, Paolo Tranquilli Leali, Federico Solla, Gianluca Maestretti, Massimo Balsano, Robero Mario Scarpa

Orthopaedic Department, University of Sassari, Italy., Orthopaedic Department, Paediatric Hospital Nice CHU-Lenval, France., Spinal Unit - Cantonal Hospital Fribourg, Switzerland., Orthopaedic Department, Santorso Hospital AUSSL 4 Schio, Italy., University of Turin, Italy.

PubMed http://www.ncbi.nlm.nih.gov/pubmed/28228781

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Denosumab is really effective in the treatment of osteoporosis secondary to hypogonadism in prostate carcinoma ... - UroToday

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WEDNESDAY, March 1, 2017 (HealthDay News) -- Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the United States, about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene -- one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow -- causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant.

In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells -- which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible -- and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team, led by Dr. Marina Cavazzana, of Necker Children's Hospital's biotherapy department, focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait -- that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin -- to replace the abnormal "adult" hemoglobin seen in sickle cell.

The hope, Williams said, is that gene therapy will ultimately offer a one-time treatment that cures sickle cell. But no one knows yet whether that will happen.

According to Williams, two key questions are: What's the long-term safety? And will the altered stem cells last for a patient's lifetime?

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published March 1 in the New England Journal of Medicine.

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Gene Therapy: A Breakthrough for Sickle Cell Anemia? - Auburn Citizen

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News

by DAVID JENSEN posted 03.02.2017

The California stem cell agency this week approved nearly $33 million for clinical stage research projects testing treatments for type 1 diabetes, arthritis of the knee, ALS and an immunodeficiency affliction.

The awards were quickly approved with little discussion during a meeting at the Oakland headquarters of the California Institute for Regenerative Medicine or CIRM, as the agency is formally known.

The goal of the research is to regenerate knee cartilage through the use of a mesenchymal progenitor cell treatment, according to the agencys application review summary

The award likely to have an impact on the most people if it is successful is a relatively small, $2.3 million award to the Cellular Biomedicine Group, a Chinese firm with operations in Cupertino, Calif. The stem cell agency by law only finances work in Clifornia. The research would also be supported by $572,993 in co-funding.

The project is aimed at treating osteoarthritis of the knee. More than 51 million people in the United States suffer from arthritis, which is particularly common in the knee.

The goal of the research is to regenerate knee cartilage through the use of a mesenchymal progenitor cell treatment, according to the agencys application review summary. The funding would go to manufacture the product and complete work to secure Food and Drug Administration approval for a phase one safety trial. A treatment for the public would likely be years in the future.

Here are the other winners today of California stem cell cash with links to the summaries of the reviews.

Caladrius Biosciences of New Jersey won $12.2 million for a clinical trial for young people ages 12-17 for newly diagnosed type 1 diabetes. The firm plans to use regulatory T cells from the patients themselves to treat the disease. Caladrius has a California location in Mountain View. (Caladrius press release can be found here.)

St. Judes Research Hospital in Memphis, Tenn., was awarded $11.9 million for a phase one/two trial to treat infants with X-linked severe combined immunodeficiency. The trial would aim at enrolling at least six patients suffering from the catastrophic affliction. The treatment would use the patients own bone marrow stem cells after the cells were specially handled. The agency said in a press release that St. Judes is working with UC San Francisco. (St. Judes press release can be found here.)

The awards were previously approved behind closed doors by the agencys out-of-state reviewers, who do not disclose publicly their economic or professional interests.

Cedars-Sinai Medical Center in Los Angeles was awarded $6.2 million for a phase 1/2A trial to test a treatment for ALS, which has no treatment or cure. The CIRM review summary said a huge unmet need existed. About 20,000 persons in the United States suffer from the affliction.

CIRMs press release did not identify the researchers involved in any of the awards.

The agency is on a push to support more clinical trials, which are the last and most expensive research prior to the possibility of winning federal approval for widespread use of a therapy.

Currently the agency is participating in 27 trials and is planning on adding 37 more in the next 40 months. The agency is expected to run out of funds for new awards in June 2020 and has no source of future financing.

The awards were previously approved behind closed doors by the agencys out-of-state reviewers, who do not disclose publicly their economic or professional interests. The agencys directors rarely overturn a positive decision by the reviewers.

All of the winners have links to two or more members of the 29-member CIRM governing board. Those members are not allowed to vote on applications where they have conflicts of interest.

About 90 percent of the funds awarded by the board since 2005 have gone to institutions that have ties to members of the board, past or present, according to calculations by the California Stem Cell Report. Eds Note: David Jensen is a retired newsman who has followed the affairs of the $3 billion California stem cell agency since 2005 via his blog, the California Stem Cell Report, where this story first appeared. He has published more than 4,000 items on California stem cell matters in the past 11 years.

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Stem cell: Knee arthritis in new $33 million research plan - Capitol Weekly

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By Andy Coghlan

Sarah Harrison and Gaelle Recher, Zernicka-Goetz Lab, University of Cambridge

Artificial mouse embryos grown from stem cells in a dish could help unlock secrets of early development and infertility that have until now evaded us.

Magdalena Zernicka-Goetz at the University of Cambridge and her team made the embryos using embryonic stem cells, the type of cells found in embryos that can mature into any type of tissue in the body.

The trick was to grow these alongside trophoblast stem cells, which normally produce the placenta. By growing these two types of cell separately and then combining them in a special gel matrix, the two mixed and started to develop together.

After around four-and-a-half days, the embryos resembled normal mouse embryos that were about to start differentiating into different body tissues and organs.

They are very similar to natural mouse embryos, says Zernicka-Goetz. We put the two types of stem cells together which has never been done before to allow them to speak to each other. We saw that the cells could self-organise themselves without our help.

This is the first time something resembling an embryo has been made from stem cells, without using an egg in some way. Techniques such as cloning, as done for Dolly the sheep and other animals, bypass the need for sperm, but still require an egg cell.

The artificial embryos are providing new insights into how embryos organise themselves and grow, says Zernicka-Goetz. The team engineered the artificial embryos so the cell types fluoresced in different colours, to reveal their movements and behaviour as the embryos go through crucial changes.

Mammal embryos were already known to start as a symmetrical ball, then elongate, form a central cavity and start developing a type of cell layer called mesoderm, which ultimately goes on to form bone and muscle.

We didnt know before how embryos form this cavity, but weve now found the mechanism for it and the sequential steps by which it forms, says Zernicka-Goetz. Its building up the foundations for the whole body plan.

The work is a great addition to the stem cell field and could be extended to human stem cell populations, says Leonard Zon at Boston Childrens Hospital, Massachusetts. Using the system, the factors that participate in embryo development could be better studied and this could help us understand early events of embryogenesis.

But Robin Lovell-Badge at the Francis Crick Institute in London says that the embryos lack two other types of cell layer required to develop the bodies organs: ectoderm, which forms skin and the central nervous system, and endoderm, which makes our internal organs.

Zernicka-Goetz hopes to see these types of cell layers develop in future experiments by adding stem cells that normally form the yolk sac, a third structure involved in embryonic development, to the mix.

If a similar feat can be achieved using human stem cells, this could tell us much about the earliest stages of our development. Current research is limited by the number of excess embryos that are donated from IVF procedures. But the new technique could produce a limitless supply, making it easier to conduct in-depth research. These artificial embryos may also be easier to tinker with, to see what effect different factors have in early embryogenesis.

Disrupting development in this way may provide new insights into the causes of abnormal embryo development and miscarriage. You would be able to understand the principles that govern each stage of development. These are not normally accessible, because they happen inside the mother, says Zernicka-Goetz.

But it is doubtful that this work could ever lead to fully grown babies in the lab. Lovell-Badge says the artificial embryos are unlikely to develop in vitro much further than shown in the study, as they would soon need the supply of nutrients and oxygen that a placenta normally channels from the mother.

Were not planning to make a mouse in the lab using stem cells, says Zernicka-Goetz. But she is hopeful that adding yolk sac stem cells will allow these artificial embryos to survive long enough to study the beginnings of organs like the heart.

Journal reference: Science, DOI: 10.1126/science.aal1810

Read more: Its time to relax the rules on growing human embryos in the lab

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Artificial embryo grown in a dish from two types of stem cells - New Scientist

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event

Date: 14 June 2017 - 17 June 2017

Location: Boston Convention and Exhibition Center 415 Summer Street Boston 02210 United States

Website: ISSCR2017.org

Email: [emailprotected]

Telephone: +1 224-592-5700

The International Society for Stem Cell Research (ISSCR) 2017 annual meeting will be held 14-17 June in Boston, Mass., U.S., at the Boston Convention and Exhibition Center. The meeting brings together 4000 stem cell researchers and clinicians from around the world to share the latest developments in stem cell research and regenerative medicine. In a series of lectures, workshops, poster presentations, and a dynamic exhibition floor, researchers focus on recent findings, technological advances, trends, and innovations that are realizing progress in using stem cells in the discovery and validation of novel treatments.

In 2017, the ISSCR is expanding its translational and clinical programming with two half-day, pre-meeting educational sessions geared toward bringing new therapies to the clinic. The Workshop on Clinical Translation (WCT) and the Clinical Advances in Stem Cell Research (CASC) programs are designed for scientists and physicians interested in learning more about the process of developing stem cell-based therapies and advances in stem cell applications in the clinic.

The Presidential Symposium recognizes a decade of progress in iPS cell research and application with a distinguished lineup of speakers including Shinya Yamanaka, discoverer of iPSCs. Additional plenary presentations include distinguished speakers from around the world focusing on organoids and organogenesis, the making of tissues and organs; stem cells and cancer; chromatin and RNA biology; stress, senescence and aging; tissue regeneration and homeostasis; and the frontiers of cell therapy.

Concurrent sessions feature new and innovative developments across the breadth of the field, and incorporate more than 100 abstract-selected speakers. Disease modelling, tissue engineering, stem cell niches, epigenetics, hematopoietic stem cells, and gene modification and gene editing are just a few of the 28 topic areas presented.

Other meeting offerings include career development sessions and networking opportunities. A full listing of the ISSCR 2017 meeting programming can be found at ISSCR2017.org.

Map

42.3461949 -71.04563680000001

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ISSCR 2017 - Drug Target Review

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Cryopreservation

Cryopreservation is the process of freezing organs and tissues at very low temperatures in order to preserve them. While it sounds simple in theory, only a handful of cells and tissues have survivedthis method. This is because while science has successfully developed ways to cool organs to the very low temperatures required for preservation, thawing them out has proven far more difficult. As the specimen thaws, itforms ice crystals, which can damage the tissue and render organs unusable.

Right now, the process is only a viable option for small samples, such as sperm or embryos. Previous efforts using slow warming techniques have proven to be effective on samples of that size, but havent worked forlarger tissue samples, like whole human organs. The inability to safely thaw the tissue has also precluded the theoretical concept of cryogenically preservingentire human bodies, with the intention of reanimating them later. The concept has roots in cryogenic technology, but is actually referred to as cryonics, and the scientific community generally considers it to be more science fiction than science fact at least for the time being.

A recentstudy has made a significant breakthrough which may well begin closing that gap even more. Using a new technique, scientists were able to cryopreserve human and pig samples, then successfully rewarm it without causing any damage to the tissue.

As lead researcher John Bischof from the University of Minnesota notes:

This is the first time that anyone has been able to scale up to a larger biological system and demonstrate successful, fast, and uniform warming of hundreds of degrees Celsius per minute of preserved tissue without damaging the tissue.

By using nanoparticles to heat the tissues at an equal rate, scientists were able to prevent the formation ofthose destructive ice crystals. The researchers mixed silica-coated iron oxide nanoparticles in a solution and applied an external magnetic field to generate heat. The process was tested on several human and pig tissue samples, and it showed that nanowarming achieves the same speed of thawing as the use of traditional convection techniques.

One theoretical application for this discovery would be, of course, bringing cryogenic life-extension techniques out of the realm of science fiction and into reality. But were not quite there yet.

A more practical application for the technique wouldbeto safely preserve and store organs for extended periods, thus improving the logistical challenges behind organ transplantation.

According to statisticsfrom the United Network for Organ Sharing, 22 people die every dayin the US while waiting for organ transplants. Contrary to popular belief, this isnt because there is a shortage of organs being donated its because organs cannot be preserved for more than a few hours. So, while there are available organs ready to be transplanted, the time it takes to find a matching recipient and transport the organ safely to their location often exceeds the window of time in which the organ remains viable for transplant.

Over half of donated hearts and lungs are thrown out each year because they dont make it to patients in time. They can only be kept on ice for four hours, and while some organs can last longer than others without a blood supply during transport, its still not a longenough in many cases.

If only half of these discarded organs were transplanted, then it has been estimated that wait lists for these organs could be extinguished within two to three years, Bischof adds. With the help of cryopreservation technology, we may be well on our way to keeping donated organs viable for longer meaning they could be transported to patients who need them even if distance and time stands between them.

Read more:
Scientists Make Huge Breakthrough In Cryogenics - Futurism

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Potential Treatment

Gene therapy has been available for quite some time now. Advances in modern medical science, particularly in stem cell research, have made it possible to use DNA to compensate for malfunctioning genes in humans. The therapies haveeven proven effective fortreating rare forms of diseases. Now, a research team in France has shown that gene therapy may be used to cure one of the most common genetic diseases in the world.

The team, led by Marina Cavazzana at the Necker Childrens Hospital in Paris, conducted stem cell treatment on a teenage boy with sickle cell disease. The disease alters theblood through beta-globin mutations, which cause abnormalities in the blood proteinhemoglobin. These abnormalities cause the blood cells (which have an irregular shape, like a sickle, hence their name) to clump together. Patients with sickle cell disease usually need transfusions to clear the blockages their cells cause, and some are able to have bone marrow transplants. About 5 percent of the global population has sickle cell disease,according to the WHO. In the United States alone, the CDC reports that approximately 100,000 people have sickle cell disease.

The patient is now 15 years old and free of all previous medication, Cavazzana saidwhen discussing the outcome of their study. He has been free of pain from blood vessel blockages, and has given up taking opioid painkillers. Their research is published in the the New England Journal of Medicine.

The particular treatment given to the teenage boy at Necker Childrens Hospitalbegan when he was 13 years old. The team took bone marrow stem cells from the boy and added mutated versions of the gene that codes for beta-globin before putting these stem cells back into the boys body. The mutated genes were designed to stop hemoglobin from clumping together and blocking blood vessels the hallmark of sickle cell disease.

Two years later, the boys outcomelooks promising.All the tests we performed on his blood show that hes been cured, but more certainty can only come from long-term follow-up, Cavazzan said. Her team also treated seven other patients who also showed promising progress.

If the method shows success in larger scale clinical trials, it could be a game changer, saidDeborah Gill at the University of Oxford, The fact the team has a patient with real clinical benefit, and biological markers to prove it, is a very big deal.

Other research involving gene therapy is also showing similar promise. One which has already been approved by the FDA is a potential treatment for blindness. Others look at treating Parkinsons disease or evenprolonging human life. What these studies show is that gene therapyand stem cells may be able togive hope to patients with diseases that have long been considered incurable.

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Doctors Claim They've Cured a Boy of a Painful Blood Disorder Using Gene Therapy - Futurism

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March 1, 2017 by Marilynn Marchione This 2009 colorized microscope image made available by the Sickle Cell Foundation of Georgia via the Centers for Disease Control and Prevention shows a sickle cell, left, and normal red blood cells of a patient with sickle cell anemia. Researchers say a French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder. The case is detailed in the March 2, 2017 issue of the New England Journal of Medicine. (Janice Haney Carr/CDC/Sickle Cell Foundation of Georgia via AP)

A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.

The first-in-the-world case is detailed in Thursday's New England Journal of Medicine.

About 90,000 people in the U.S., mostly blacks, have sickle cell, the first disease for which a molecular cause was found. Worldwide, about 275,000 babies are born with it each year.

"Vexing questions of race and stigma have shadowed the history of its medical treatment," including a time when blacks who carry the bad gene were urged not to have children, spurring accusations of genocide, Keith Wailoo of Princeton University wrote in a separate article in the journal.

The disease is caused by a single typo in the DNA alphabet of the gene for hemoglobin, the stuff in red blood cells that carries oxygen. When it's defective, the cells sickle into a crescent shape, clogging tiny blood vessels and causing bouts of extreme pain and sometimes more serious problems such as strokes and organ damage. It keeps many people from playing sports and enjoying other activities of normal life.

A stem cell transplant from a blood-matched sibling is a potential cure, but in the U.S., fewer than one in five people have a donor like that. Pain crises are treated with blood transfusions and drugs, but they're a temporary fix. Gene therapy offers hope of a lasting one.

The boy, now 15, was treated at Necker Children's Hospital in Paris in October 2014. Researchers gave him a gene, taken up by his blood stem cells, to help prevent the sickling. Now, about half of his red blood cells have normal hemoglobin; he has not needed a transfusion since three months after his treatment and is off all medicines.

"It's not a cure but it doesn't matter," because the disease is effectively dodged, said Philippe Leboulch, who helped invent the therapy and helped found Bluebird Bio in Cambridge, Massachusetts, the company that treated the boy. The work was supported by a grant from the French government's research agency.

Bluebird has treated at least six others in the U.S. and France. Full results have not been reported, but the gene therapy has not taken hold as well in some of them as it did in the French teen. Researchers think they know why and are adjusting methods to try to do better.

Two other gene therapy studies for sickle cell are underway in the U.S.at the University of California, Los Angeles and Cincinnati Children's Hospitaland another is about to start at Harvard and Boston Children's Hospital using a little different approach.

"This work gives considerable promise" for a solution to a very common problem, said Dr. Stuart Orkin, a Boston Children's Hospital doctor who is an inventor on a patent related to gene editing.

"The results are quite good in this patient," he said of the French teen. "It shows gene therapy is on the right track."

Explore further: BCL11A-based gene therapy for sickle cell disease passes key preclinical test

More information: Gene therapy: ghr.nlm.nih.gov/primer/therapy/availability

2017 The Associated Press. All rights reserved.

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As people get older so do the hematopoietic stem cells (HSCs) that form their blood, creating an increased risk for compromised immunity and certain blood cancers. Now researchers are reporting in the scientific journal EMBO ...

A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.

A research team, led by the University of Minnesota, has discovered a groundbreaking process to successfully rewarm large-scale animal heart valves and blood vessels preserved at very low temperatures. The discovery is a ...

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Let's see. AIDS kills young white Americans, Sickle cell kills young black Americans. Let's just give all the research funding to AIDS!

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Gene therapy lets a French teen dodge sickle cell disease - Medical Xpress

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"The numbers are fantastic," said Dr Fred Locke, a blood cancer expert at Moffitt Cancer Center in Tampa who co-led the study.

"These are heavily treated patients who have no other options."

The treatment, which has been dubbed 'a living drug' by doctors, works by filtering a patient's blood to remove key immune system cells called T-cells, which are then genetically engineered in the lab to recognise cancer cells.

Cancer cells are very good a evading the immune system, but the new therapy essentially cuts the brakes, allowing immune cells to do their job properly.

Martin Ledwick, Cancer Research UKs head cancer information nurse, said: These results are promising and suggest that one day CAR-T cells could become a treatment option for some patients with certain types of lymphoma.

"But, we need to know more about the side effects of the treatment and long term benefits.

Read this article:
Terminal cancer patients in complete remission after one gene therapy treatment - Telegraph.co.uk

Recommendation and review posted by simmons

Sickle cell disease. Image: Flickr

Sickle cell disease is a slow, vicious killer. Most people diagnosed with the red blood cell disorder in the US live to be between 40 and 60. But those years are a lifetime of pain, as abnormal, crescent-shaped hemoglobin stops up blood flow and deprives tissues of oxygen, causing frequent bouts of agony, along with more severe consequences like organ damage. Now, after decades of searching for a cure, researchers are announcing that, in at least one patient, they seem to have found a very promising treatment.

Two years ago, a French teen with sickle cell disease underwent a gene therapy treatment intended to help his red blood cells from sickling. In a paper published Thursday in the New England Journal of Medicine, the researchers revealed that today, half of his red blood cells have normal-shaped hemoglobin. He has not needed a blood transfusion, which many sickle cell patients receive to reduce complications from the disease, since three months after his treatment. He is also off all medicines.

To reiterate, the paper is a case study of just one patient. Bluebird Bio, the Massachusetts biotech company that sponsored the clinical trial, has treated at least six other trials underway in the US and France, but those results have not yet been fully reported. The gene therapy has not worked quite as well in some of those other patients; researchers say they are adjusting the therapy accordingly. It is also possible that the boy may eventually experience some blood flow blockages again in the future.

The results, though early, are encouraging. They represent the promise of new genetics technologies to address a disease that has long been neglected and tinged with racism. Sickle cell disease affects about 100,000 people in the US, most of whom are black. It is an inherited genetic disease caused by a mutation of a single letter in a persons genetic code.

This single-letter mutation makes it a promising candidate for cutting edge technologies, like the gene-editing technique CRISPR-Cas9, and other gene therapies. Recently, a rush of new research has sought to address it. Two other gene therapy studies for sickle cell are underway in the US one at UCLA and another at Cincinnati Childrens Hospital. Yet another is about to start in a collaboration between Harvard and Boston Childrens Hospital. Last fall, researchers all demonstrated the ability to correct the mutation in human cells using CRISPR, though that strategy will yet have to surpass significant scientific and political hurdles before reaching clinical trials.

In the new study, researchers took bone marrow stem cells from the boy and fed them corrected versions of a gene that codes for beta-globin, a protein that helps produce normal hemoglobin. The hope was that those altered stem cells would interfere with the boys faulty proteins and allow his red blood cells to function normally. They continued the transfusions until the transplanted cells began to produce normal-shaped hemoglobin. In the following months, the numbers of those cells continued to increase until in December 2016, they accounted for more than half the red blood cells in his body. In other words, so far so good.

Currently, the only long-term treatment for sickle cell disease is a bone marrow transplant, a high-risk, difficult procedure which many patients are not even eligible for. Pain and other side-effects are treated with blood transfusions for temporary relief. New technologies offer the hope of a solution that could provide long-term relief and allow patients to live some semblance of a normal life.

For decades, gene therapies have been touted as a cure for everything. But so far, successes have been infrequent, and often for very rare diseases. But early success in treating sickle cell disease means that soon, if were lucky, the benefits of this technology may reach hundreds of thousands of people.

[New England Journal of Medicine]

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Will Sickle Cell Be the Next Disease Genetic Engineering Cures? - Gizmodo

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In world-first breakthrough, French doctors use gene therapy to treat rare blood disease - RFI

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For mostof us,stressis just apart of life. It can last for a few hours like the time leading up to a final exam or for years like when youre taking care of an ailing loved one.

Cleveland Clinic is a non-profit academic medical center. Advertising on our site helps support our mission. We do not endorse non-Cleveland Clinic products or services. Policy

Stress is sometimesa motivator that helpsyou rise to the occasion. At other times, its simply overwhelming. Whatever the case, if its chronic, it can takea toll on your immune system.

Clinical immunologistLeonard Calabrese, DO, offers insights on how stress impacts your immunity and what you can do to minimize the effect.

Eliminating or modifying these factors in ones life is vital to protect and augment the immune response, he says. Its necessary to buffer the inevitability of the aging process.

Stress occurs when life events surpass your abilities to cope. It causes your body to produce greater levels of the stress hormone cortisol.

In short spurts, cortisolcan boost your immunity by limitinginflammation. But over time, your body can get used to having too much cortisol in your blood. And this opensthe door for more inflammation, Dr. Calabrese says.

In addition, stress decreases the bodys lymphocytes the white blood cells that help fight off infection. The lower your lymphocyte level, the more at risk you are for viruses, including the common cold and cold sores.

High stress levels also can causedepressionand anxiety, again leading to higher levels of inflammation.In the long-term, sustained, high levels of inflammation point to an overworked, over-tired immune system that cant properly protect you.

If you dont control high stress levels, chronic inflammation can accompany it and can contribute to the development and progression of many diseases of the immune system such as:

Under sustained, long-term stress, you also can develop cardiovascular problems, including a fast heart rate andheart disease, as well as gastric ulcers. Youll also be at greater risk fortype 2 diabetes, various cancers and mental decline.

Stress reduction strategies not only give your mind a break, but they can also relieve the pressure on your immune system. You can take steps to reduce short-term and long-term stress, Dr. Calabrese says. Two tactics are most effective:

Stress in acute situations, however, can be healthful and protective, so its not all bad for us. Remember: its chronic stress that we seek to control.

Continued here:
What Happens When Your Immune System Gets Stressed Out? - Health Essentials from Cleveland Clinic (blog)

Recommendation and review posted by sam

Should an increased risk for colon cancer among young and middle-aged adults change screening recommendations?

A new study showing a dramatic rise in colorectal cancers in this non-traditional population meaning those under the age of 50 has many asking that question.

So far, though, screening guidelines remain the same, which has researchers pushing for people to know the warning signs, the risks of their lifestyle factors, and their genetic risks. Experts are searching for less invasive screening procedures as well.

The new study, led by the American Cancer Society, finds that those born after 1990 have double yes, double the risk for colon cancer and quadruple the risk for rectal cancers compared to those born around 1950. Researchers believe the underlying cause may be due to the ever-increasing weight of the nation, unhealthy diets, and sedentary lifestyles.

Related: Cancer Kills, Even at Age 20

Our finding that colorectal cancer risk for millennials has escalated back to the level of those born in the late 1800s is very sobering, Rebecca Siegel, MPH, strategic director of Surveillance Information Services at the American Cancer Society, said in a press release.

Educational campaigns are needed to alert clinicians and the general public about this increase to help reduce delays in diagnosis, which are so prevalent in young people, but also to encourage healthier eating and more active lifestyles to try to reverse this trend, she added.

Another large international study this week in the BMJ links obesity with cancersmainly of the digestive organs as well as hormone-related malignancies.Strong associations were found betweena high body mass index (BMI) with risk of esophageal, bone marrow, and colon (in men), rectal (in men), biliary tract system, pancreatic, endometrial (in premenopausal women), and kidney cancers.

Weight gain was also associated with a higher risk ofgall bladder, gastric cardia, and ovarian cancers, and mortality from multiple myeloma.

Only about 38percentof those prescribed a colonoscopy actually comply.

Current recommendations call for most people to be screened for colorectal cancer starting at age 50, unless they have a family history of the disease. The study authors, however, are proposing that screening be considered before age 50, especially as those who are younger and who develop the disease are diagnosed late-stage largely because theyve not been thinking cancer is a risk and have brushed off the symptoms for years.

"This has been something that has been of concern in the screening and cancer prevention communities," saidDr. Kiesel, a gastroenterologist at Mayo Clinic and assistant professor of medicine at Mayo Clinic College of Medicine."Would patients under the age of 50 but of average risk meaning, lacking a hereditary reason to be screened for colon cancer, benefit from screening at an earlier age?"

Kiesel told LifeZette that a "line in the sand" has been drawn around the age of 40: If screening begins then, would it make a meaningful dent in the course of the illness? "Weve hypothesized that we would, but there really isnt any data to support that decision-making. This study emphasizes that we need a higher level of research to determine if the benefit that we hope for will be realized."

The concern with screening too early is the cost versus the benefit. Procedures are often done that may not be necessary or do more harm than good to a patient. And screenings aren't cheap that is another consideration.

But we tend to avoid screenings anyway. We don't like the process. Only about 38percentof those prescribed a colonoscopy comply. Andrelying on patients to recognize the symptoms to catch a cancer early may be inadequate, said Kiesel.

Related: Five Wise Ways to Avoid Cancer

It's why he and the Mayo Clinic have worked withExact Sciencesto develop a less-invasive screening test, Cologuard.In June of 2016, the U.S. Preventive Services Task Force (USPSTF) issued an A rating to the newer screening option anoninvasive at-home stool DNA test, as well as virtual colonoscopy (CT colonography). The hope is that more Americans will be open to getting these less invasive screenings.

Until then, Americans can take some very simple steps to reduce the risk of colorectal cancers:eat more vegetables, fruits, and whole grains; get regular exercise; watch your weight; avoid tobacco; limit your alcohol; and know the signs. Where colorectal cancers are concerned specifically, any unintended weight loss, weakness and fatigue, or a change in bowel habits or blood in your stool that lasts more than a few days are signs that need to be checked by a physician.

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Even More Reason to Kick That Sedentary Lifestyle - LifeZette

Recommendation and review posted by Bethany Smith

- Couples who are obese may take longer to achieve pregnancy than partners who aren't as overweight, a recent U.S. study suggests.

Previous studies in women have linked obesity to difficulties getting pregnant. In the current study, neither male nor female obesity alone was linked to taking a longer time to conceive, but when both partners were obese, the couple took up to 59 percent longer to conceive than non-obese counterparts.

"If our results are confirmed, fertility specialists may want to take couples' weight status into account when counseling them about achieving pregnancy," said lead study author Rajeshwari Sundaram of the Eunice Kennedy Shriver National Institute of Child Health and Human Development in Bethesda, Maryland.

"The benefits of a healthy weight are well known: obesity increases the risk for diseases such as type 2 diabetes, heart disease and cancer," Sundaram added by email.

Sundaram and colleagues focused on the relationship between pregnancy and body mass index (BMI), a ratio of weight to height. A BMI between 18.5 and 24.9 is considered a healthy weight, while 25 to 29.9 is overweight, 30 or above is obese and 40 or higher is what's known as morbidly obese. (The National Heart, Lung and Blood Institute has an online BMI calculator here: http://bit.ly/1D0ZqDv.)

An adult who is 5 feet 9 inches tall and weighs 160 pounds, for example, would have a BMI of 23.6, which is in the healthy range. An obese adult at that height would weigh at least 203 pounds and have a BMI of 30 or more.

Researchers categorized individuals into two subgroups: obese class I, with a BMI from 30 to 34.9, and obese class II, with a BMI of 35 or greater.

Overall, 27 percent of the women and 41 percent of the men were obese class I or heavier.

Then, the researchers compared the average time to conceive for couples where neither partner was obese to couples where both fell into the obese class II group.

Couples in the obese class II group took 55 percent longer to achieve pregnancy than their normal weight counterparts, the study team calculated.

After accounting for other factors that influence fertility such as age, smoking status, exercise and cholesterol levels, obese class II couples took 59 percent longer to get pregnant.

About 40 percent of the men and 47 percent of the women also had enough excess fat around the midsection to potentially influence fertility.

In addition, 60 percent of the women and 58 percent of the men said they exercised no more than once a week, the researchers report in Human Reproduction.

Beyond its small size, another limitation of the study is that it wasn't a controlled experiment designed to determine whether obesity directly causes infertility, the authors note. It also focused on couples in the general population, not people undergoing treatment for infertility, so the results might not reflect what would happen for all couples trying to conceive, the researchers point out.

However, unlike many other studies of obesity and fertility, the current analysis used height and weight measured by clinicians instead of relying on participants to report this information themselves, which may make the findings more accurate.

Obesity can influence fertility by altering hormone levels in both men and women, converting testosterone to estrogen, said Dr. Jeffrey Goldberg, section head of reproductive endocrinology at the Cleveland Clinic in Ohio.

"If you have more fat there is more conversion from testosterone to estrogen," Goldberg, who wasn't involved in the study, said.

It makes sense that obese couples would take longer to conceive because excess weight doesn't just impact fertility in women.

"For women extra weight impairs ovulatory function," Goldberg said. "For guys, having lower testosterone and higher estrogen impairs sperm production and having a lot of fat around the scrotum, fat thighs and fat around the abdomen raises the scrotal temperature and that can also have an adverse effect."

Read more:
Obese couples may take longer to conceive - Fox News

Recommendation and review posted by sam

News | March 01, 2017 | Cardiac Imaging, CT

Coronary CT angiography (CCTA) shows older men who use testosterone gel have a significantly greater increase in coronary artery noncalcified plaque volume, according to a study published in the Journal of the American Medical Association.

Researchers from nine states in the U.S. undertook a double-blinded, placebo-controlled trial to determine if testosterone use among older men slowed progression of noncalcified coronary artery plaque volume or increased cardiovascular risk.

A total of 138 men completed the study out of 170 who enrolled. The men were aged 65 or older with an average of two serum testosterone levels lower than 275 ng/dL and had symptoms suggestive of hypogonadism. The subjects received testosterone gel dose adjusted to maintain the testosterone level in the normal range for young men (73 subjects), or placebo gel for 12 months (65 subjects).

The primary outcome was noncalcified coronary artery plaque volume, as determined by CCTA, and secondary outcomes included total coronary artery plaque volume and coronary artery calcium score (range of 0 to more than 400 Agatston units, with higher values indicating more severe atherosclerosis). At baseline, 70 men (50.7%) were found to have a coronary artery calcification score higher than 300 Agatston units, reflecting severe atherosclerosis.

Coronary CT angiogram results showed testosterone treatment compared with placebo was associated with a significantly greater increase in noncalcified plaque volume from baseline to 12 months, from median values of 204 mm3 to 232 mm3 versus 317 mm3 to 325 mm3, respectively, with an estimated difference of 41 mm3. For the secondary outcomes, the median total plaque volume increased from baseline to 12 months from 272 mm3 to 318 mm3 in the testosterone group versus from 499 mm3 to 541 mm3 in the placebo group, with an estimated difference of 47 mm3. The median coronary artery calcification score changed from 255 to 244 Agatston units in the testosterone group versus 494 to 503 Agatston units in the placebo group, with an estimated difference of 27 Agatston unit. No major adverse cardiovascular events occurred in either group.

The researchers concluded that older men with symptomatic hypogonadism who undergo treatment with testosterone gel for one year have a significantly greater increase in coronary artery noncalcified plaque volume. Larger studies are needed to understand the clinical implications of this finding, they wrote.

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Increased Plaque Volume on CCTA in Older Men Who Use Testosterone Gel - Diagnostic Imaging

Recommendation and review posted by Bethany Smith

KARAIKAL: Recent advancements in stem cells research have given hope for successfully treating diabetic heart disease (DHD), renowned New Zealand-based researcher in cardiovascular diseases Dr Rajesh Katare said today.

DHD affected the muscular tissues of the heart leading to complications and it had been demonstrated that resident stem cells of myocardium can be stimulated to repair and replace e degenerated cardiac myocytes resulting in a novel therapeutic effect and ultimately cardiac regeneration, he said.

Katare, Director of Cardiovascular Research Division in the University of Otago, New Zealand, was delivering the keynote address at the continuing medical education programme on "Role of Micro-RNAs and stem cells in cardiac regeneration in diabetic heart disease" at the Karaikal campus of premier health institute JIPMER.

Presenting clinical evidences, Katare said stem cell therapy certainly presented a new hope for successfully treating DHD.

Jawaharlal Institute of Post Graduate Medical Education (JIPMER) Director Dr Subash Chandra Parija pointed out that it was the first such programme on the role of stem cells in cardiac regeneration in the whole of the country.

He said as diabetes was highly prevalent in the country, providing treatment for DHD had become a big challenge. Patients suffering from the condition have to undergo lifelong treatment and medications. "In this backdrop, advancements in stem cell therapy assume significance," he said.

See the original post here:
Stem cell therapy can help treat diabetic heart disease - Economic Times

Recommendation and review posted by sam

Xconomy Texas

San Antonio StemBioSys, the life sciences company with a system for growing stem cells, has licensed an experimental technology from University of Texas Health San Antonio that may help identify healthy young adult stem cells among large pools of other cells.

Theres plenty of research examining how to possibly use adult stem cells as treatments for medical conditions, ranging from cardiac disease to metabolic disorders, but current uses are rather limited to therapies like bone-marrow transplants for blood disorders, especially in children. Treatments that use patients own stem cells may be safer than using stem cells from someone else because they might reduce the potential for an immune response, according to StemBioSys CEO Bob Hutchens. Thats still theoretical, he says.

Finding large quantities of usable adult stem cells is difficult, though. StemBioSys believes its new technology can potentially identify a few thousand high-quality, young stem cells from a sample of tens of thousands of cells taken from a patient, Hutchens sayspotentially being a key word.

The research is quite earlythe technology has only been studied in animal models and in vitro, and StemBioSys is in the process of applying for federal grants to take the research into animal trials. If StemBioSys new intellectual property can successfully isolate the stem cells, Hutchens says they could grow more of them with StemBioSys core product.

StemBioSys sells a so-called extracellular matrix product made of proteins that provide a hospitable environment for stem cells, helping them divide and produce more stem cells.

Whats intriguing to us is that its a really interesting application of our technology, Hutchens says. You take this combination of identifying this very small population of young healthy cells in elderly people, and use our technology to expand it.

If the company can indeed find the young stem cells of a single patient and replicate them, it would give researchers and physician an accessible pool of the cells that theyd want for potential stem cell transplants and other treatments, Hutchens says.

Terms of the deal werent disclosed. StemBioSys, which was founded based on other University of Texas System research, acquired a portfolio of issued and pending patents. Famed MIT researcher and Xconomist Robert Langer is on the companys board of directors.

Again, theres plenty to prove out with this early stage research, so it will take time before any potential commercialization comes to fruition. Travis Block, the researcher who helpeddevelopthe technology while earning his PhD. last year at the University of Texas Health Science Center at San Antonio, will help shepherd the project along and other regenerative medicine work as StemBioSyss senior scientist.

David Holley is Xconomy's national correspondent based in Austin, TX. You can reach him at dholley@xconomy.com

Continue reading here:
StemBioSys Lands Experimental UT Tech That Finds Young Stem Cells - Xconomy

Recommendation and review posted by simmons

SAN CARLOS, Calif. & BALTIMORE--(BUSINESS WIRE)--Johns Hopkins Medicine, the Maryland Stem Cell Research Fund (MSCRF) and BioCardia, Inc. (OTC:BCDA) today announced that the first patient has been treated in the pivotal Phase III CardiAMP clinical trial of a cell-based therapy for the treatment of ischemic heart failure that develops after a heart attack. The first patient was treated at Johns Hopkins Hospital by a team led by Peter Johnston, MD, a faculty member in the Department of Medicine and Division of Cardiology, and principal investigator of the trial at Johns Hopkins.

The investigational CardiAMP therapy is designed to deliver a high dose of a patients own bone marrow cells directly to the point of cardiac dysfunction, potentially stimulating the bodys natural healing mechanism after a heart attack.

The patient experience with CardiAMP therapy begins with a pre-procedural cell potency screening test. If a patient qualifies for therapy, they are scheduled for a bone marrow aspiration. A point of care cell processing platform is then utilized to concentrate the autologous bone marrow cells, which are subsequently delivered in a minimally-invasive procedure directly to the damaged regions in a patients heart.

This cell-based therapy offers great potential for heart failure patients, said Carl Pepine, MD, professor and former chief of cardiovascular medicine at the University of Florida, Gainesville and national co-principal investigator of the CardiAMP trial. We look forward to validating the impact of the therapy on patients quality of life and functional capacity in this important study.

In addition to Dr. Johnston, the CardiAMP research team at Johns Hopkins includes Gary Gerstenblith, MD, Jeffrey Brinker, MD, Ivan Borrello, MD, Judi Willhide, Katherine Laws, Audrey Dudek, Michele Fisher and John Texter, as well as the nurses and technicians of the Johns Hopkins Cardiovascular Interventional Laboratory.

Funding the clinical trial of this cell therapy, which could be the first cardiac cell therapy approved in the United States, is an important step towards treatments, said Dan Gincel, PhD., executive director of the MSCRF at TEDCO. Through our clinical program, we are advancing cures and improving healthcare in the State of Maryland.

The CardiAMP Heart Failure Trial is a phase III, multi-center, randomized, double-blinded, sham-controlled study of up to 260 patients at up to 40 centers nationwide, which includes an optional 10-patient roll-in cohort. The primary endpoint for the trial is a significant improvement in Six Minute Walk distance at 12 months post-treatment. Study subjects must be diagnosed with New York Heart Association (NYHA) Class II or III heart failure as a result of a previous heart attack. The national co-principal investigators are Dr. Pepine and Amish Raval, MD, of the University of Wisconsin.

For information about eligibility or enrollment in the trial, please visit http://www.clinicaltrials.gov or ask your cardiologist.

About BioCardia BioCardia, Inc., headquartered in San Carlos, CA, is developing regenerative biologic therapies to treat cardiovascular disease. CardiAMP and CardiALLO cell therapies are the companys biotherapeutic product candidates in clinical development. For more information, visit http://www.BioCardia.com.

About Johns Hopkins Medicine Johns Hopkins Medicine (JHM), headquartered in Baltimore, Maryland, is one of the leading health care systems in the United States. Johns Hopkins Medicine unites physicians and scientists of the Johns Hopkins University School of Medicine with the organizations, health professionals and facilities of The Johns Hopkins Hospital and Health System. For more information, visit http://www.hopkinsmedicine.org.

About Maryland Stem Cell Research Fund The Maryland Stem Cell Research Act of 2006was established by the Governor and the Maryland General Assembly during the 2006 legislative session and created the Maryland Stem Cell Research Fund. This fund is continued through an appropriation in the Governor's annual budget. The purpose of the Fund is to promote state-funded stem cell research and cures through grants and loans to public and private entities in the State. For more information, visit http://www.MSCRF.org.

Forward Looking Statements This press release contains forward-looking statements as that term is defined under the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, among other things, references to the enrollment of our Phase 3 trial, commercialization and efficacy of our products and therapies, the product development timelines of our competitors. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the inherent uncertainties associated with developing new products or technologies, unexpected expenditures, the ability to raise the additional funding needed to continue to pursue BioCardias business and product development plans, competition in the industry in which BioCardia operates and overall market conditions, and whether the combined funds will support BioCardias operations and enable BioCardia to advance its pivotal Phase 3 CardiAMP cell therapy program. These forward-looking statements are made as of the date of this press release, and BioCardia assumes no obligation to update the forward-looking statements.

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Johns Hopkins Medicine, Maryland Stem Cell Research Fund and ... - Business Wire (press release)

Recommendation and review posted by sam

Wednesday, March 1, 2017 6:01 AM UTC

PRESS RELEASE

TiGenix to present at Cowen's 37thAnnual Health Care Conference in Boston

Leuven (BELGIUM) - 1st March, 2017, 07:00h CET - TiGenix NV (Euronext Brussels and Nasdaq: TIG), an advanced biopharmaceutical company focused on developing and commercializing novel therapeutics from its proprietary platforms of allogeneic expanded stem cells, today announced that Eduardo Bravo, CEO of TiGenix, will be presenting at the 37th Annual Cowen and Company's Health Care Conference in Boston (USA) at The Boston Marriott Copley Place on Monday, March 6 at 3:20-3:50PM (EST) in Regis, 3rd Floor (breakout at 4:00 PM-04:30PM (EST) at Boston University, 3rd Floor).

The presentation will be webcast live and can be accessed on the day of the event at this link. A replay of the webcast will be available on the Company's website for 30 days following the presentation. To ensure timely connection, it is recommended that users register at least 10 minutes prior to the scheduled webcast.

The TiGenix management team will be available for one-to-one meetings from Monday, March 6th to Wednesday, March 8th. Please contact Investor Relations at Investor@tigenix.com for a meeting request.

For more information:

Claudia D'Augusta Chief Financial Officer T: +34 91 804 92 64 claudia.daugusta@tigenix.com

About TiGenix

TiGenix NV (Euronext Brussels: TIG) is an advanced biopharmaceutical company focused on developing and commercializing novel therapeutics from its proprietary platforms of allogeneic, or donor-derived, expanded stem cells. Our lead product candidate from the adipose-derived stem cell technology platform is Cx601, which is in registration with the European Medicines Agency for the treatment of complex perianal fistulas in Crohn's disease patients. Our adipose-derived stem cell product candidate Cx611 has completed a Phase I sepsis challenge trial and a Phase I/II trial in rheumatoid arthritis. Effective July 31, 2015, TiGenix acquired Coretherapix, whose lead cellular product candidate, AlloCSC-01, is currently in a Phase II clinical trial in Acute Myocardial Infarction (AMI). In addition, the second product candidate from the cardiac stem cell-based platform acquired from Coretherapix, AlloCSC-02, is being developed in a chronic indication. On July 4, 2016, TiGenix entered into a licensing agreement with Takeda, a large pharmaceutical company active in gastroenterology, under which Takeda acquired the exclusive right to commercialize Cx601 for complex perianal fistulas outside the United States. TiGenix is headquartered in Leuven (Belgium) and has operations in Madrid (Spain).

About Cx601

Cx601 is a suspension of allogeneic expanded adipose-derived stem cells (eASC) locally injected. Cx601 is an investigational agent being developed for the treatment of complex perianal fistulas in Crohn's disease patients with inadequate response to at least one conventional or biologic therapy including antibiotics, immunosuppressants, or anti-TNF agents. Crohn's disease is a chronic inflammatory disease of the intestine and patients can suffer from complex perianal fistulas for which there is currently no effective treatment. In 2009, the European Commission granted Cx601 orphan designation for the treatment of anal fistulas, recognizing the debilitating nature of the disease and the lack of treatment options. Cx601 has met the primary end-point in the Phase III ADMIRE-CD study in Crohn's disease patients with complex perianal fistula, a randomized, double-blind, placebo-controlled trial run in Europe and Israel and designed to comply with the requirements laid down by the EMA. 'Madrid Network' issued a soft loan to help finance this Phase III study, which was funded by the Secretary of State for Research, Development and Innovation (Ministry of Economy and Competitiveness) within the framework of the INNTEGRA plan. The study's primary endpoint was combined remission, defined as clinical assessment at week 24 of closure of all treated external openings draining at baseline despite gentle finger compression, and absence of collections >2cm confirmed by MRI. In the ITT population (n=212), Cx601 achieved statistically significant superiority (p=0.024) on the primary endpoint with 50% combined remission at week 24 compared to 34% in the placebo arm. Efficacy results were robust and consistent across all statistical populations. Treatment emergent adverse events (non-serious and serious) and discontinuations due to adverse events were comparable between Cx601 and placebo arms. The 24-weeks results have been published by The Lancet, one of the most highly regarded and well known medical journals in the world. The Phase III study has completed a follow-up analysis at 52 weeks confirming its sustained efficacy and safety profile. Top line follow-up data showed that in the ITT population Cx601 achieved statistical superiority (p=0.012) with 54% combined remission at week 52 compared to 37% in the placebo arm. The 52-week data also showed a higher rate of sustained closure in those patients treated with Cx601 and in combined remission at week 24 (75.0%) compared to patients in the placebo group (55.9%). Based on the positive 24-weeks Phase III study results, TiGenix has submitted a Marketing Authorization Application to the EMA in early 2016. TiGenix is preparing to develop Cx601 in the U.S. after having reached an agreement with the FDA through a special protocol assessment procedure (SPA) in 2015. On July 4, 2016 TiGenix entered into a licensing agreement with Takeda, a pharmaceutical company leader in gastroenterology, whereby Takeda acquired an exclusive right to commercialize Cx601 for complex perianal fistulas in Crohn's patients outside of the U.S.

Forward-looking information

This press release may contain forward-looking statements and estimates with respect to the anticipated future performance of TiGenix and the market in which it operates. Certain of these statements, forecasts and estimates can be recognised by the use of words such as, without limitation, "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will" and "continue" and similar expressions. They include all matters that are not historical facts. Such statements, forecasts and estimates are based on various assumptions and assessments of known and unknown risks, uncertainties and other factors, which were deemed reasonable when made but may or may not prove to be correct. Actual events are difficult to predict and may depend upon factors that are beyond the Company's control. Therefore, actual results, the financial condition, performance or achievements of TiGenix, or industry results, may turn out to be materially different from any future results, performance or achievements expressed or implied by such statements, forecasts and estimates. Given these uncertainties, no representations are made as to the accuracy or fairness of such forward-looking statements, forecasts and estimates. Furthermore, forward-looking statements, forecasts and estimates only speak as of the date of the publication of this press release. TiGenix disclaims any obligation to update any such forward-looking statement, forecast or estimates to reflect any change in the Company's expectations with regard thereto, or any change in events, conditions or circumstances on which any such statement, forecast or estimate is based, except to the extent required by Belgian law.

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TiGenix to present at Cowen's 37th Annual Health Care Conference in Boston - EconoTimes

Recommendation and review posted by sam

NBC-5 news anchor Rob Stafford has been diagnosed with a rare blood disorder and will take a leave of absence from the station for several months as he undergoes a bone marrow transplant and chemotherapy.

Stafford credited his wife, Lisa, for pushing him to get an early diagnosis that doctors say greatly improved his prognosis. He is scheduled to begin treatment Friday at the Mayo Clinic in Rochester, Minn.

"I spent my whole life asking questions, but I asked very few questions about my own health because all I wanted to hear is I'm OK," Stafford said in an interview at his Hinsdale home Tuesday. "You have to ask questions."

Stafford told colleagues at WMAQ-Ch. 5 about his illness in an email Wednesday morning and planned to announce the news publicly Wednesday at the end of the 10 p.m. newscast.

He expects to be away from his desk anchoring the 5, 6 and 10 p.m. weekday news for four to six months while at the Mayo Clinic. Doctors there say Stafford is in the early stages, Stage 2, of the disease, which can lead to heart failure and death if undetected.

Stafford's disorder, called amyloidosis, occurs when an abnormal protein called amyloid is produced in bone marrow that can be deposited in tissues and organs. There are more than 40 types of the disorder that affects the heart, kidneys, liver, spleen, nervous system and digestive tract. Stafford's type known as light chain amyloidosis is rare, with fewer than 2,000 or so cases diagnosed in the U.S. each year, according to Dr. Ronald Go, Stafford's hematologist at the Mayo Clinic. Doctors are optimistic he will go into remission after treatment.

Before his diagnosis in January, Stafford had noticed lower energy levels while biking the Cal-Sag trail and hiking through the Rocky Mountains while on vacation. But the 58-year-old award-winning investigative reporter and father of three adult children dismissed the episodes as signs of getting older, he said.

After a physical detected slightly high cholesterol levels and more than usual amounts of protein in his urine, both indicators that his kidney had been damaged as a result of the blood disease, Stafford's wife insisted that he take the symptoms seriously.

"I believe there's a reason that this happened, and I think there's a calling that is to let people know the importance of early detection," said Lisa Stafford, his wife of 30 years who owns a medical marketing and communications company. She plans to stay with Stafford in temporary housing near the Mayo Clinic as he undergoes treatment, lives in a sterile environment and rebuilds his immune system while recovering from the effects of chemotherapy.

In his email to fellow staffers, Stafford joked that he did not wait for the end of sweeps to schedule treatment, but that Friday was the first opening on the Mayo schedule after Stafford completed all the required medical tests.

"I consider this early diagnosis a gift that left to my own devices I would not have received," Stafford wrote. "I'm going to take full advantage of my good fortune and hit this head on with the most aggressive and proven treatment available."

Stafford joined NBC-5 in 2009 after working as a Chicago-based correspondent for Dateline NBC for 11 years. Prior to that, he was a general assignment reporter at CBS2 Chicago. He has won two national and seven local Emmy awards, and an Edward R. Murrow award for a Dateline investigation into racial profiling.

Stafford said his experience meeting and interviewing people in the news, often "on their worst days," has allowed him to keep his health issues in perspective.

"This thing pales in comparison to 95 percent of the stories I do on a daily basis," said Stafford, who added that his work as a journalist also has helped him to seek out the best support, medical opinions and advice about the disorder.

During treatment, doctors will remove, or "harvest," stem cells from Stafford's own bone marrow and freeze millions of healthy ones. Chemotherapy then will be used to wipe out all of his bone marrow, including the unhealthy cells. As the final part of the treatment, doctors will transplant the healthy stem cells back into Stafford's bone marrow, and they will reproduce themselves, Go said.

"You're rescuing your bone marrow by the stem cells that you stored," Go said.

Although Stafford's parents were both treated for cancers in their 50s, doctors do not know the exact cause of Stafford's illness and do not believe it was is hereditary. Risk factors for amyloidosis include exposure to chemicals, radiation and aging, Go said.

Stafford confided in his NBC-5 co-anchor, Allison Rosati, and meteorologist Brant Miller about his illness shortly after his diagnosis in January. As medical tests forced him to travel, he alerted NBC-5 managers, who encouraged Stafford to do whatever it took to get the medical attention needed, he said.

"Rob is loved by his colleagues in the newsroom. We are encouraged by the news that his illness was detected early, and that he is in the excellent hands of the top doctors at the Mayo Clinic. We all wish Rob the best of luck in the weeks ahead, and we can't wait for his return to the newsroom," station manager and vice president of news Frank Whitaker said in an email.

NBC-5 weekend anchor Dick Johnson will be filling in for Stafford during his absence.

Stafford is expected to lose his hair as a result of the chemotherapy and has experienced weight loss. Doctors have encouraged him to pack on a few pounds in anticipation of further weight loss. He's obliged by indulging on Girl Scout cookies, deep dish pizza, cheeseburgers and milk, he said.

Stafford said he is trying to take emotion out of his upcoming battle, which he is approaching as a fight that he plans to win.

"I am not freaking out because I really am confident I am going to get through this," he said.

Lisa Stafford will be posting updates about her husband's condition on a blog: staffordrecovery.com. Rob Stafford also will offer updates on his Facebook Fan page.

Stafford's announcement came less than 24 hours after Hosea Sanders, veteran news anchor at WLS-Ch. 7, announced on his Facebook page he will undergo surgery for prostate cancer and that he was "very optimistic about the outcome."

Sanders told social media friends Tuesday night that he had been diagnosed several weeks ago and would be taking some time off from the ABC-owned station, for which he anchors the 7 p.m. newscast each weeknight.

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NBC-5 news anchor Rob Stafford to undergo bone marrow ... - Chicago Tribune

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Abby West first decided to become a bone-marrow donor when she was a young journalist covering a bone-marrow drive. Little did she know that nearly two decades later, shed be called upon to try to help save someones life.

The newspaper I had joined had a reporter who had passed away of sickle cell anemia, and I had become aware of the need for African-Americans to join the registry, West, a senior editor at Yahoo Celebrity, tells Yahoo Beauty about her decision to sign up with Be The Match. I was covering a bone-marrow drive for African-Americans, and it seemed like the right thing to do. West says she simply got her cheek swabbed which yields a sample of cells that doctors use to compare specific protein markers with those of patients who need a bone-marrow transplants and then went about her life.

But in June 2014 17 years later she received an email from Be the Match, a registry of the National Marrow Donation Program, saying that her bone marrow was a potential match for someone in need. She called the organization to learn more. You get a sense of urgency, need, and what is expected of you, she says. To make sure she was a healthy donor, West needed to fill out forms, and once it was determined she met all of the donor criteria, she moved on to a physical examination.

(Photo courtesy Abby West)

West says she underwent seven weeks of blood testing and prep work before she donated bone marrow. Part of the process was making a decision about whether she would do a surgical bone-marrow donation, in which a person is put under general or local anesthesia and liquid marrow is taken from the back of the pelvic bone, or a nonsurgical peripheral blood stem cell (PBSC) harvest, in which blood is drawn from one arm into a machine, where it is spun and the stem cells are extracted. The remaining blood is then returned to the persons body via the other arm. West chose the latter option, and started receiving Neupogen shots, which increased her bodys production of stem cells. It puts your stem cells on overdrive, she explains.

West says the message from Be the Match was always clear: It was her body and she could change her mind at any point. However, she says, they request that if youre going to change your mind, you do so before you begin the shots. Heres why: At this point, the patient she would be donating to was starting chemotherapy in anticipation of receiving her stem cells. You get the sense that someones life is in the balance and theres no turning back only moving forward, she says. You need to understand the commitment to potentially saving someones life.

West says the shots werent painful, but she did experience some flu-like symptoms. You feel a little achy, she says. Your body is becoming laden with stem cells. Its uncomfortable, but not painful. A nurse came to Wests office to give her the shots, but donors can also go to clinics to get them, or do them on their own. They try to make it as seamless and painless as possible, she says, noting that the shots made her feel moretired than usual. I wasnt on my gym grind that week, she says.

The day of the donation, West reported to a blood donation center at 7 a.m., along with a friend to keep her company, and sat in a chair for about eight hours while her blood was drawn, spun, and returned to her body. When you have to go to the bathroom, you just stop, unhook, go the bathroom, and hook back up again, she says. It wasnt a hardship I cant complain about it. It was eight hours of sitting and talking. West says the process was mildly uncomfortable, but she knew that in about 48 hours, someone was going to have a life-saving operation. Afterwards, she felt tired and went to sleep for a little while. I did it on Friday and was back at work on Monday, but I would have been fine to go to work the next day, she says.

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Stem cells are regenerative, and there is no long-term harm to the donor, Muzzafar Qazilbash, MD professor of stem cell transplantation at the University of Texas MD Anderson Cancer Center explains to Yahoo Beauty.

Photo: Courtesy of Abby West

While there is a need for people of all races and ethnicities to donate bone marrow, there a special need for African-Americans to do so, Qazilbash says. African-Americans make up about 10 percent of the U.S. population, and only about 25 percent to 30 percent of people who could potentially benefit from a bone-marrow transplant have a perfectly matched sibling donor available, he says. The rest of the patients have to find matched, unrelated donors from the National Marrow Donor Program. However, the overwhelming majority of approximately 25 million volunteer donors registered with NMDP are people of Western and Northern European origins, and as a result it is very hard to find matching, unrelated matchingdonors for African-American patients, he says. Encouraging more people of African ancestry will increase the possibility of finding unrelated donors for African-American patients, which can be life saving.

Jack Jacoub, MD, medical oncologist and director of thoracic oncology at MemorialCare Cancer Institute at Orange Coast Memorial Medical Center in Fountain Valley, Calif., tells Yahoo Beauty while some ethnicities have very little genetic variability, there is a lot of genetic variability in the African-American population making the probability of having two genetically similar people less likely. Then you bring up the issue of how few African-American donors there are, and there is difficulty finding an adequate donor, he says.

Donor stem cellscan be used to treat life-threatening conditions such as leukemia and lymphoma, as well as sickle-cell anemia, Jacoub says. They can also help people with bone-marrow failure syndromes such as aplastic anemia a condition in which a persons body stops producing blood cells as well as help treat children born with severe immune system deficiencies, Qazilbash says.

West never received information about the person she donated bone marrow to, other than the fact that the patient was a man. She doesnt know how he fared after the donation, but learned that he struggled with graft-versus-host disease, a complication that can occur after a stem-cell or bone-marrow transplant. Its fairly common for people to have it and move forward, but Ive never found out how he ultimately ended up, she says.

West was so moved by her experience that she eventually joined the board of Be the Match, and now urges others tobecome bone-marrow donors. You always think about what you would want someone to do for you or your family member, she says, noting that shes still on the registry and would donate again if there was a need.

Of course, blood and needles are involved, which can scare some people off, but West says its worth it in the end. In order to do something heroic, you have to overcome some discomfort and some trepidations, she said.

To find out more about becoming a bone-marrow donor, please visit BeTheMatch.org.

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Exactly What It's Like to Donate Bone Marrow and Why More ... - Yahoo Health

Recommendation and review posted by simmons

Johns Hopkins Medicine doctors have treated the first person in a key phase of a clinical trial where a high dose of the patient's own bone marrow cells was used to treat heart failure after a heart attack.

The dose was directed precisely at the point of dysfunction in the heart in the hope that it will stimulate the body's natural healing process.

The Hopkins patient was the first to receive what is called the CardiAMP therapy as part of the third phase of a trial taking place at 40 medical centers across the country. Once phase three trials show a treatment works well, doctors can apply for approval with the Food and Drug Administration.

BioCardia, Inc., headquartered in San Carlos, CA., developed the therapy and The Maryland Stem Cell Research Fund provided research money. The fund was created by the Maryland General Assembly in 2006 to promote state-funded stem cell research and cures through grants and loans to public and private entities.

"Funding the clinical trial of this cell therapy, which could be the first cardiac cell therapy approved in the United States, is an important step towards treatments," Dan Gincel, executive director of the Maryland Stem Cell Research Fund, said in a statement. "Through our clinical program, we are advancing cures and improving health care in the State of Maryland."

Heart disease is the number one killer in the United States. About 610,000 people die from heart disease every year and it accounts for one in every four deaths, according to the Centers for Disease Control and Prevention.

The first patient was treated at Johns Hopkins Hospital by a team led by Peter Johnston, faculty member in the Department of Medicine and Division of Cardiology, and principal investigator of the trial at Johns Hopkins.

amcdaniels@baltsun.com

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Patient's own bone marrow used to treat heart failure - Baltimore Sun (blog)

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Wound healing: The stem cell dynamic Science Daily It was particularly exciting to observe that the repair of the skin epidermis involves the activation of very different stem cells that react the same way to the emergency situation of the wound and have the power to completely restore the damaged ...

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Wound healing: The stem cell dynamic - Science Daily

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