Leukaemia and lymphoma have a good survival rate – The Star Online
By daniellenierenberg
Of all blood cancers, leukaemia and lymphoma are among the most curable.
However, many people, including doctors, still believe the disease leads to immediate death.
This is no longer true today as they are not fatal.
With optimal treatment, the majority of patients go into remission and are considered cured.
These two cancers have been more extensively studied than other forms of cancer, due to the ease in obtaining samples from blood, bone marrow or lymph nodes, spurring the advent of novel targeted therapies for a cure, says consultant haematologist Dr Ng Soo Chin.
Most blood cancers start in the bone marrow, where blood is produced.
Bone marrow contains stem cells, which mature and develop into red blood cells, white blood cells or platelets.
In most blood cancers, normal cell development is interrupted by the uncontrolled growth of an abnormal type of a particular blood cell.
These abnormal blood cells, which are cancerous, prevent your blood from performing many of its functions, like fighting off infections or preventing serious bleeding.
Leukaemia or white blood is classified into acute and chronic disease, which is then divided further into subtypes: acute lymphocytic leukaemia, acute myeloid leukaemia, chronic lymphocytic leukaemia (CLL) and chronic myeloid leukaemia (CML).
The presentation between acute and chronic leukaemia differs.
The acute person will tell you he was well a week ago and is now down with symptoms such as lethargy, anaemia and recurrent infection.
Suddenly, he may look pale, so we check his blood count for any abnormalities. A bone marrow exam will further confirm whether it is acute.
With chronic leukaemia, the patient can be unwell for a couple of months.
We are increasingly picking up cases early because of blood test availability.
The survival rate has improved tremendously for acute leukaemia, with more than 50% fully cured because bone marrow transplants are easily available in the country.
For CLL and CML, 95% of patients are alive at the 10-year mark, says Dr Ng.
Generally, chronic leukaemia patients belong to the older age group (50 years and above), but acute leukaemia can occur in all ages.
Leukaemia symptoms are often vague and not specific, so its easy to overlook them as they may resemble symptoms of the flu and other common illnesses.
In fact, chronic leukaemia may initially produce no symptoms and can go unnoticed or undiagnosed for years.
Lymphomas, a type of blood cancer that begins in a subset of white blood cells called lymphocytes, can be classified into Hodgkins and non-Hodgkins.
The main difference between Hodgkins and non-Hodgkins lymphoma is the specific lymphocyte each involves.
Lymphocytes are an integral part of your immune system, which protects you from germs.
Five-year survival rates are high with Hodgkins lymphoma at 86% and non-Hodgkins lymphoma at 70%.
You can beat the disease even if it is detected at a late stage.
Multiple myeloma, which is the third kind of blood cancer, forms in a type of white blood cell called a plasma cell.
Patients often complain of bone pain, and unfortunately, this type of cancer has no cure.
Blood cancers typically involve abnormal white blood cells and can affect paople of all ages, depending on the type of cancer. 123rf.com
Fear of treatment
Chemotherapy is a much dreaded word among cancer patients.
But with advances in medicine, newer chemotherapy-free treatments are now available.
Dr Ng says, Traditionally, cancer is treated via surgery or radiation the layman says we fry and poison them, which is not far from the truth!
Radiation means burning the cancerous area, but a lot of times, the cancer can also be present elsewhere, so there is limitation to this treatment.
With chemotherapy, we use cytotoxic (cell-killing) drugs they go in and knock off both cancer and normal cells.
The short-term effects include vomiting, hair loss, appetite loss and weight loss.
But as doctors, we are looking at a different perspective. We are more worried about white cells dropping (neutropenia) because the patient can pick up an infection that can potentially kill him.
Neutropenia is a condition that results when the body does not have enough neutrophils, a type of white blood cell that is an essential first line of defence against infections.
Thats one risk of chemotherapy, although we can now improve neutropenia by giving a growth factor injection.
But for certain cancers, we need to step up the drugs.
He adds: We are scared of neutropenia, but patients are more concerned about bodily changes.
The older ones get upset over losing hair because they cannot take it when others ask them what has happened to their hair.
Young people are not as concerned with hair loss because it can be trendy.
We understand that chemotherapy is less than pleasant and strong doses can impair fertility in young patients, especially women.
Despite current technology, only one-third of patients are successful in freezing their eggs.
What he is concerned about is that chemotherapy can actually increase the patients risk of getting another cancer, especially blood cancer.
It can happen the day after! says Dr Ng.
Most experts believe chemotherapy damages stem cells, so if youre unlucky, you might get acute myeloid leukaemia after undergoing chemotherapy for breast cancer.
Its just like crossing the road there is always a risk of being knocked down.
All our cells have a biological clock and there is an orderly exchange of old and new cells.
But with blood cancers such as leukaemia, there is a clone of abnormal cells.
Cancer cells have an advantage over normal cells because they can survive longer.
Chemotherapy is still needed to treat most acute blood cancers, although if the mutation is known, targeted therapies can be applied.
For chronic blood cancers, there is no need for chemotherapy. Oral drugs are enough to combat the disease.
Eventually, many patients are able to wean off the drugs.
As we may be aware, immunotherapy is the buzzword in cancer treatment today.
Also called biologic therapy, it is a type of cancer treatment that boosts the bodys natural defences to fight cancer.
It uses substances made by the body or in a laboratory to improve or restore immune system function.
One of the latest treatment modalities is the CAR T-cell therapy, a form of immunotherapy that uses specially altered T cells a part of the immune system to fight cancer.
A sample of a patients T cells are collected from the blood, then modified to produce special structures called chimeric antigen receptors (CARs) on their surface.
When these CAR T-cells are reinfused into the patient, the new receptors enable them to latch onto a specific antigen on the patients tumour cells and kill the cells.
At the moment, this intravenous therapy is available in the United States and hasnt reached our shores yet. It has to be properly regulated first, says Dr Ng.
A volunteer is having his head shaved to donate hair to make wigs for cancer patients in this filepic. Hair loss is one of the side effects of chemotherapy that affect patients the most.
Following natural remedies
The consultant haematologist errs on the side of caution when patients ask about natural cancer remedies, or the dos and donts during treatment.
We always believe there should be a scientific approach to the problem.
If patients are doing okay while undergoing treatment and there is no weight loss, I tell them to go ahead and do what they always do.
However, just be particular about food hygiene, as there is a chance you may get food poisoning.
If youre undergoing chemotherapy, then youll land yourself in hospital, and if your luck is bad, you may even land up in the ICU (intensive care unit).
So make sure the food is cooked and not left overnight to reduce your chances of infection.
Eat a balanced diet, he advises.
When it comes to exercise, he says to work out within your limit.
Instead of pushing the body and running marathons or climbing mountains, go for walks.
Dr Ng says, Life should go on, but be sensible.
Dont go to crowded places because you may pick up an infection, but dont be withdrawn either. All humans need social interaction.
With the billion-dollar dietary supplements industry, companies are constantly trying to lure customers into buying their products.
A lot of supplements are just glorified vitamins in different packaging.
The more expensive they are, the more people will buy them, thinking they are good.
There are people with good intentions, but unfortunately, there are also a lot of scammers out there that is life.
For the amount you spend on supplements, why not keep the money aside and go for a trip once your treatment is over? he suggests.
Often, the late diagnosis is due to preference for alternative treatment.
These alternative treatments are like fashion shows, after some time, they go out of trend.
For me, youre wasting valuable time because cancer is not your friend.
Yes, chemotherapy is tough, but with the latest chemo-free regimen, patients are more willing to come forward.
The earlier it is treated, the higher your chances of recovering, he says.
To share his 30-odd years of knowledge and experience in the field, Dr Ng has written his third book titled Understanding Blood Disorders.
Intended for patients, caregivers and healthcare professionals, proceeds from the sales of the 270-page book will go to the newly set-up Faith Hope Love Hospice Care Malaysia in Petaling Jaya, Selangor.
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Abington working toward big win – Sports – The Intelligencer
By daniellenierenberg
The Galloping Ghosts are looking beyond the scoreboard to help classmate Juwan Adams battle pediatric cancer.
Khalis Whiting and her Abington girls basketball teammates have a different perspective this holiday season. A perspective that changed when through Kisses for Kyle they were put in touch with two area families who have a child battling pediatric cancer.
One George Hamlin is a student in Upper Dublin High School. The other, Juwan Adams, is a senior at Abington. Both families were on the receiving end of gifts and gift cards at the Abington-Upper Moreland game last week. It is the 10th annual giving back game since coach Dan Marsh initiated the project.
It was an amazing experience, said Whiting, the Ghosts sophomore point guard. I was not familiar with the families, but I have seen (Adams) a few times in school, but I never really knew the story until this came up and I researched him.
He has a powerful story, and hes such a strong young man to be going through this. Every time I see him in the hallways, hes always smiling and so positive. So you know give it to his family.
Adams, who has been battling Hodgkins Lymphoma since April of 2016, is in need of a match for an allogeneic stem cell transplant.
Right now, he is out of options, his mother, Andrea Adams, said. We know for sure that an allo stem cell transplant will cure him, but unfortunately, in order to do that, we need a donor.
We have tested everyone in our family, and no one is a full match, so were relying on the Be the Match registry to locate a donor. Sadly, for minorities theres a very slim chance to actually find a full 10-out-of-10 match because we dont have enough minorities on the registry.
Hes had almost every treatment available for Hodgkins, including an auto stem cell transplant, which is where he gave himself his own cells in February of 2017. He was in remission for about a year, and unfortunately, in May of this year, we found out the cancer is back, and its been spreading consistently since May.
Despite missing more than half of the last three-and-a-half years of school, Juwan maintains a 4.25 GPA and is a member of the National Honor Society.
Abington School District has been really great with accommodating him, giving him tutors, and whenever hes an in-patient, he does hospital school, Andrea said.
Juwan is the drum captain and lieutenant of Abingtons marching band, and since September is Pediatric Cancer Awareness month, for the past four years, the marching band has worn gold ribbons on their uniforms.
Since his diagnosis in eighth grade, Juwan has been active in pediatric cancer awareness events, and for the past four years, he has held toy and book drives that he presents to the children at CHOP on his birthday in July.
Last week, Juwan and his family were on the receiving end.
It was great because Juwan never wants gifts, Andrea said. Having a child with cancer, financially its tough, especially now that we have only have one person working fulltime, and that lifted a tremendous burden, and it was so unexpected.
To have him honored by his school and his peers, he was super excited about it, and he really appreciated it because a lot of kids (battling cancer) have to give up school, or their friends tend to abandon them.
I want to thank the team and the Abington students because it is very easy to turn your back on kids that are going through these things. These kids have really rallied around him. This is one of the reasons he fights so hard to be in school. He sometimes sneaks to school because he feels the love from his school and his peers. That was one thing he definitely wanted to do to finish his senior year with his class. They have really rallied around him, and this kind of thing gives him the extra push he needs to keep fighting.
Statistics say Juwan has a 23 percent chance of finding a perfect match, but the Abington senior has been proactive.
When they told him the odds of finding his match, he said he had to do something about it, Andrea said. He set a goal to register a thousand people on the national registry, half of those being minorities.
We have gone around holding bone marrow drives. Hes now an ambassador for Be the Match, and hes been an ambassador for CHOP for about two years.
Juwan is inviting everyone to join his fight.
"People are starting to wake up and realize kids with cancer more and more are dying each day, and if they have a chance to do something, they should take every step they can," Juwan said earlier this fall. It's been hard sometimes, but I have my friends and family to support me.
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Abington working toward big win - Sports - The Intelligencer
Little Mila on amazing path to recovery – Croatia Week
By daniellenierenberg
Mila Roncevic (Photo: Private album)
24 December 2019 There has been some great news just before Christmas from America where little Mila Roncevic has been receiving life-saving treatment.
Mila, from Rijeka in Croatia, has been fighting leukaemia (acute myelogenous megakaryocytic leukaemia AML M7) and earlier this year was given just a 1% chance of beating it. A massive fundraising campaign in Croatia and abroad back in April helped raise $5.6 million to get her to the Childrens Hospital of Philadelphia for urgent treatment.
Mila has been at the hospital since 3 April and on Monday her father has shared some incredible news that after a successful bone marrow transplant, the leukemia has receded.
Mila is fine right now. She underwent a bone marrow transplant from a donor on 1/11/2019 and so far its better than okay. It was not a surgery per se, but rather a bone marrow transplant after achieving and maintaining remission for six months. Transplantation is not an operation but a complex procedure which includes preparation of the so-called conditioning, that is, achieving the conditions for receiving donor stem cells and then recovering and waiting for defence cells to be created on their own, in which case she had to remain in complete isolation because there is no immunity. In order to receive the transplant, the disease must recede, which they achieved within a month of arrival and maintained for almost half a year, her father Marin told 24sata.
Marin says that just three weeks after the transplant, Mila was released from hospital and to the amazement and disbelief of all the doctors, she is now running, lifting, eating and drinking alone and does everything that was considered impossible in the early post-transplant period.
She looks and acts like a perfectly healthy baby, even though we know that there are still many potential dangers ahead, Marin said.
Marin says going to Philadelphia was certainly the right decision, which has been confirmed by the successful outcome of the treatment.
In Croatia, the chances for successful treatment were below 1%. It was also the right decision because at that moment we had no alternative, nor enough time to look for it. It is difficult to actually talk about percentages, because as far as we are told, cases with similar diagnoses that have been successfully cured in Croatia and Europe do not exist, he said, before thanking everyone again who helped get Mila to the United States for treatment.
(Photo: Private album)
Thank you people. If it wasnt for your empathy, kindness and intrinsic desire to help, none of this would be possible. Every kuna, every prayer and every good thought saved Mila and will help countless other children for whom funds will be provided for. This action also showed how important it is for all of us to stick together and how much we can do that way. We are incredibly grateful to have genuinely and openly begged for help and received it. Its not just about the money. It is about the love, support and prayers of all people who have felt our affliction and suffering.
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Little Mila on amazing path to recovery - Croatia Week
A blood cancer diagnosis helped me find true happiness – The Guardian
By daniellenierenberg
December 2017, and the Christmas party season is in full flow. Everywhere I look are scenes from some hilariously awful Dickens pastiche: revellers squeezed into warm pubs; joyful chatter spilling out on to the street; the sound of carols and the scent of mulled wine in the air. Its as if I am peering in at it all through frosted glass, wishing my own Christmas could be as carefree.
Instead, I have spent a morning turned on my side on a hospital bed while a nervous-looking young doctor works up a sweat attempting to force a long needle into my hipbone. He needs it to go deep enough that he can suck out some of the marrow inside, but my tough bones are making life difficult for him.
I dont feel so tough. My wife and I spend the next fortnight anxiously waiting for the results. Results that should confirm why my body is behaving in unexpected ways: the unusual infections; the crushing fatigue; the old jeans that suddenly slip off my waist.
Christmas is never a nice time to feel alone. Yet, despite the fact I am surrounded by loved ones, that is how I feel: terrifyingly alone. The emotions of the season get warped and amplified. I attempt to go to one party, see a friend who is going through her own hellish time, and we both sob on each others shoulders for five minutes straight. Everything feels raw and heavy. My little girl is not even 18 months old, and I love her more than anything but I find it hard to even be in the same room as her. Its all too much.
If Christmas has lost its religious meaning, then it hasnt for me. I try praying for the first time in about three decades: Er, yes, it has been a while sorry about that but could you just help me out with this one thing? I promise God and Santa Ill be all sorts of good if things turn out OK.
***
My results arrive on 22 December. There is a wait in a hospital corridor that is still too triggering to think about properly. And then a doctor calls me in, sits me down and tells me that I have a rare blood cancer called essential thrombocythemia, which sounds like some cult artist signed to Warp Records in the 90s (the doctor doesnt say that bit). There is no known cure. But dont worry, he says, its manageable. I just need to take some aspirin and keep an eye on it. You will lead a normal life, he says. My wife tells me my face instantly changed colour, the pallid grey lifting for the first time in weeks.
My little girl throws up all over the seat when we pull out of the drive, and it doesnt even feel slightly annoying
Its a strange gift, receiving blood cancer for Christmas. In some ways I preferred the Mr Frosty slushy-making kit I got when I was eight, and maybe even the Scalextric that never quite played out the way you hoped it would from the adverts. And yet what the doctor is telling me you will lead a normal life feels like the biggest and best present I have ever received. Queueing up to be discharged, I let wave after wave of euphoria run through me and think to myself: This has to be the weirdest cancer diagnosis ever.
A day later, we pack up the car and head off to my parents. My little girl throws up all over the back seat as soon as we pull out of the drive, and it doesnt even feel slightly annoying. We laugh. Life is good. That Christmas, for the first time since I can remember, I am truly happy; just living in the moment. The light seems brighter and more beautiful. I notice dew drops on plants and the smell of fresh air. I hug my wife and daughter even more tightly than usual.
***
All this relief is not to last long. In the first week of 2018, I attend a follow-up appointment and am told that, sorry, they hadnt seen all of the bone marrow samples before. My condition is, in fact, developing into a much more serious disease called myelofibrosis, which needs treatment.
A week on from that, I turn up at the hospital, steeled to start chemotherapy. But there is worse news: a team of specialists have discussed my case and they believe I am at high risk of developing acute myeloid leukaemia, a swift and deadly cancer. They recommend you have a stem cell transplant, says the doctor. I ask when. As soon as possible. If I can find a match on the stem cell donor register, then I will be dosed up with drugs so intense that my entire immune system will be wiped out; then a strangers cells will be fed into me and we will all cross our fingers and hope that my body doesnt reject them. The chance of survival and the disease not returning does not seem to me to be all that much better than 50/50. Even if it all succeeds, the recovery process will be long and gruelling.
I spend the next few weeks in a state of catatonic depression. Or do I? Because I am somehow getting things done: I organise a will, I arrange a sperm bank visit (the transplant, even if successful, will leave me infertile), I cry myself senseless writing a letter to my daughter in case the worst should happen. I also drink all the good bottles of wine I had been saving for special occasions. A bottle of Domaine Dujac Morey Saint-Denis 2012 on a Tuesday night with defrosted Quorn chilli not the pairing Id had in mind, but saving it for the future seems silly.
Through all the gloom I see something with startling clarity. I realise that what Im mourning is not so much my old life before all this started a life of pointless anxieties, petty rivalries and overthinking but rather the carefree, optimistic version of life I had briefly glimpsed over Christmas. And yet no sooner have I understood all this than the chance to enact it has been snatched away. I feel like an old professor who has finally unravelled the mysteries of the universe with his dying breath.
***
Over the next few months, something happens that I still find hard to believe. I am transferred to a new hospital with a more specialist team on the case. There are more blood tests and scans, and another long needle is forced into my hip. And then I get another gift, this one in time for Christmas 2018: my condition is not so serious as I was led to believe. It appears to be a peculiar version of a peculiar cancer caught somewhere between the relatively benign essential thrombocythemia and the more concerning myelofibrosis. But it is stable, at least for now, with no signs to suggest it will progress any time soon.
***
I like to think that this year I have made good on my promise to live like I did during the Christmas of 2017. My outlook has certainly changed. When people ask how, I always say the same thing: that its great to get older. The idea of panicking about a milestone such as my imminent 40th seems so ridiculous now. Instead, just think what a privilege it is to be able to get there.
I am more present for my family these days, and less consumed with things I cant control. I have returned to the volunteering role I thought I didnt have time for; I have got fit; I dont let work define my happiness; I am kinder to myself. I have bought lots more nice wine to replace the nice wine I drank with defrosted Quorn chilli.
Do I still get annoyed by delayed trains, lost keys or the fact my daughter is taking half an hour to put on a pink tutu, the only item of clothing in the house that shell wear? It would be a lie to say no. But the second I think: But youre not quite likely to die any more, the problem disappears. I am, undeniably, a happier person.
I still have a malfunction inside me and I still have to think about it every day. Its hard not to my spleen, inflated with excess blood cells, gently nudges against my ribs like an annoying acquaintance who would hate me to forget that all is not quite right. At some point in the future and not even the best doctors can predict exactly when the disease might whirr into life and start scarring my bone marrow, turning it into a barren wasteland that can no longer produce enough blood to keep me alive. Im hopeful that science will find a fix before that time comes. There are encouraging signs on the horizon. And if not? Well, these days I try not to dwell on the future. I am here, instead, for the present. I am alive. I am alive with the spirit of Christmas.
MPN Voice provides information and emotional support to people diagnosed with a myeloproliferative neoplasm
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A blood cancer diagnosis helped me find true happiness - The Guardian
Stem Cells Market Poised to Expand at a Robust Pace Over 2017 to 2025 – Market Research Sheets
By daniellenierenberg
In theglobalstem cells marketa sizeable proportion of companies are trying to garner investments from organizations based overseas. This is one of the strategies leveraged by them to grow their market share. Further, they are also forging partnerships with pharmaceutical organizations to up revenues.
In addition, companies in the global stem cells market are pouring money into expansion through multidisciplinary and multi-sector collaboration for large scale production of high quality pluripotent and differentiated cells. The market, at present, is characterized by a diverse product portfolio, which is expected to up competition, and eventually growth in the market.
Some of the key players operating in the global stem cells market are STEMCELL Technologies Inc., Astellas Pharma Inc., Cellular Engineering Technologies Inc., BioTime Inc., Takara Bio Inc., U.S. Stem Cell, Inc., BrainStorm Cell Therapeutics Inc., Cytori Therapeutics, Inc., Osiris Therapeutics, Inc., and Caladrius Biosciences, Inc.
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As per a report by Transparency Market Research, the global market for stem cells is expected to register a healthy CAGR of 13.8% during the period from 2017 to 2025 to become worth US$270.5 bn by 2025.
Depending upon the type of products, the global stem cell market can be divided into adult stem cells, human embryonic stem cells, induced pluripotent stem cells, etc. Of them, the segment of adult stem cells accounts for a leading share in the market. This is because of their ability to generate trillions of specialized cells which may lower the risks of rejection and repair tissue damage.
Depending upon geography, the key segments of the global stem cells market are North America, Latin America, Europe, Asia Pacific, and the Middle East and Africa. At present, North America dominates the market because of the substantial investments in the field, impressive economic growth, rising instances of target chronic diseases, and technological progress. As per the TMR report, the market in North America will likely retain its dominant share in the near future to become worth US$167.33 bn by 2025.
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Investments in Research Drives Market
Constant thrust on research to broaden the utility scope of associated products is at the forefront of driving growth in the global stem cells market. Such research projects have generated various possibilities of different clinical applications of these cells, to usher in new treatments for diseases.Since cellular therapies are considered the next major step in transforming healthcare, companies are expanding their cellular therapy portfolio to include a range of ailments such as Parkinsons disease, type 1 diabetes, spinal cord injury, Alzheimers disease, etc.
The growing prevalence of chronic diseases and increasing investments of pharmaceutical and biopharmaceutical companies in stem cell research are the key driving factors for the stem cells therapeutics market. The growing number of stem cell donors, improved stem cell banking facilities, and increasing research and development are other crucial factors serving to propel the market, explains the lead analyst of the report.
This post was originally published on Market Research Sheets
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Stem Cells Market Poised to Expand at a Robust Pace Over 2017 to 2025 - Market Research Sheets
5 Things to Know About Hair loss and Scalp Care Centre Papilla Haircare – Singapore Tatler
By daniellenierenberg
By Gerald Tan December 23, 2019 Tatler Focus
The centre offers the latest scalp innovations that address all your hair thinning woes by getting to the root cause
While you are pampering your skin with the most luxurious creams and lotions, dont forget to show your crowning glory some tender loving care, too. Beautiful tresses require plenty of effort and dedication to upkeep, but when you are faced with unfortunate scalp ailments or hair-loss issues, however, maintaining its volume and healthy shine can seem like anuphill task.
Enter hair loss and scalp care centre Papilla Haircare, which might have the solution for all your hair woes.
From state-of-the-art equipment to medicallybacked technologies, here are five things to know about the brand:
Thankfully, advances in science and technology can help alleviate many hairrelated problems. Papilla Haircare has the latest innovative solutions. Located at Ngee Ann City, it is a one-stop hub that utilises the latest medicallybacked technologies. The centre collaborates with doctors and scientists to concoct serums rich in stem cells in its own Korean laboratory to ensure the highest safety standards.
(Related: 7 Natural Beauty Products Your Skin Will Love You For)
Boasting sleek black and gold accents, Papilla Haircares contemporary interiors are a reflection of its cutting-edge services. Its clinically proven programmes are the result of extensive scientific research, meticulously developed by a group of Korean dermatologists and hair transplant surgeons. Thanks to their efficacies, these remedies have also been adopted for post-procedure use at top hair transplant centres in South Korea.
(Related: 5 Foods To Eat For Healthy Hair And Nails)
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5 Things to Know About Hair loss and Scalp Care Centre Papilla Haircare - Singapore Tatler
Five hot topics in autism research in 2019 – Spectrum
By daniellenierenberg
This year, researchers unearthed clues to the causes of autism and how to treat it from a variety of sources.
Advances in tiny models of the human brain bared new details about the biology of autism and provided possible platforms for testing therapies. Studies of heart rate put a spotlight on the autonomic nervous system as a potential wellspring of autism traits. And others forged a controversial connection between the gut microbiome and autism.
A few studies revealed important information about the time points at which different forms of autism are amenable to therapy. This year also saw scrutiny of tests used for screening and diagnosis, revealing gaps and limitations in the system for identifying autistic children.
Here are the years top five topics in autism research.
Brain organoids start as mere clusters of stem cells, which then are coaxed to mature into brain cells. This year, the life span of these brains-in-a-dish grew to one year and then nearly two, enabling them to mature and mimic some aspects of the human brain. In the longest-lived organoids, researchers tracked changes in the expression of autism genes. Organoids derived from the skin cells of autistic people have a shortage of cells that suppress brain activity, they found. The finding supports the signaling imbalance theory of autism, which holds that the brains of autistic people are hyper-excitable.
This year, scientists also built tiny replicas of two brain areas bridged by a long fiber tract that might reveal how long-range connections are altered in the brains of people with autism.
Brain organoids spun from people with fragile X syndrome may help explain why some experimental fragile X drugs work in mice but not in people and generate leads for effective therapies. Organoids could provide a platform for testing treatments, too, as researchers can now churn out hundreds of these brain-like blobs in parallel and make them uniform in shape and composition.
More distant applications include studies of consciousness and the effects of microgravity on the brain. In a fledgling sign of the former, brain organoids showed synchronized neuronal firing patterns, some aspects of which look like those in preterm infants.
New evidence emerged tying autism to the workings of the autonomic nervous system, which controls breathing, heart rate and digestion. Differences in the system could explain a range of autism traits, including social difficulties and sensory sensitivity, as well as heart problems and digestive issues.
Many of these differences show up in the heart rate. Heart rate remains steady in autistic people as they breathe instead of the typical pattern of slowing slightly on exhale and quickening on inhale. This discrepancy arises after 18 months of age, around the same time that the conditions core traits emerge. Children with Rett syndrome also have unusual heart-rate patterns.
These differences may persist beyond childhood. One study showed that autistic adults resting heart rates rarely vary; an even heart rate suggests a lack of flexibility in responding to environmental changes.
Autistic children are unusually prone to gastrointestinal problems. This association may not be a coincidence: Certain genetic mutations or alterations in the microbiome the mix of microbes in the intestines may contribute to both autism and gut problems.
Four mouse studies in 2019 offered up fresh evidence some of it controversial to support this idea. In one study, researchers replaced the gut microbes in mice with those from autistic boys. The mice have repetitive behaviors, make fewer vocalizations and spend less time socializing than controls do, providing the first evidence that gut microbes contribute to autism traits.
But within hours of the studys publication, several experts criticized its small sample size and highly variable results. Others found a possible statistical error.
In an unrelated study, researchers revealed that oral doses of Lactobacillus reuteri, a type of gut bacteria found in yogurt and breast milk, boost social behavior in three mouse models of autism. And two other sets of findings suggested that mutations in NLGN3, a high-confidence autism gene, alter gut function. One of them showed that a mutation in this gene disrupts the mices microbiome.
Drugs for autism may be most effective when given during a critical period of brain development. Researchers delineated the windows for treating autism traits in mouse and rat models of the condition.
One study revealed that by the time mice reach adulthood, they have lost their ability to learn from social experiences. Giving adult mice an injection of 3,4-methylenedioxymethamphetamine (MDMA), the active ingredient in ecstasy, reopens the critical window for learning.
In another study, researchers fed the cholesterol drug lovastatin to rat models of fragile X syndrome. The treatment, if given at 4 weeks of age (the rat equivalent of childhood), prevents cognitive problems, the researchers found.
The timing of treatments may be more important for some forms of autism than for others. A study of mice missing UBE3A, the gene mutated in Angelman syndrome, showed that the earlier in life the gene is restored, the more the mice improve.
By contrast, a mutation in the autism gene SCN2A has many of the same effects on neurons when introduced into adolescent mice as it does when it is present from conception. And unpublished results show that correcting an SCN2A mutation in adulthood reverses these problems.
A series of studies this year called into question the accuracy of early screening and revealed racial disparities in autism diagnoses.
Some studies cast doubt on the utility of a widely used screening tool, the Modified Checklist for Autism in Toddlers: The test identifies less than 40 percent of autistic children, and 85 percent of those it does flag do not have autism.
Of the toddlers the test flags, most do not receive follow-up evaluations. And for those who are seen again, a definitive diagnosis may not be possible right away. Some children who screen negative at age 3 meet the diagnostic criteria for autism only after age 5.
Not all children have equal access to autism evaluations, with black and Hispanic children at a disadvantage in several U.S. states. In New Jersey, black children are half as likely as white children to receive an autism assessment by age 3.
About 9 percent of autistic children may outgrow an autism diagnosis but still have other conditions that require support, highlighting the need for continued observation to adapt to their evolving needs.
Read more here:
Five hot topics in autism research in 2019 - Spectrum
BeiGene Announces Acceptance of a Supplemental New Drug Application in China for REVLIMID in Relapsed or Refractory Indolent Lymphoma – BioSpace
By daniellenierenberg
BEIJING, China and CAMBRIDGE, Mass., Dec. 22, 2019 (GLOBE NEWSWIRE) -- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, today announced that the China National Medical Products Administration (NMPA) has accepted a supplemental new drug application (sNDA) for REVLIMID (lenalidomide), in combination with rituximab, for the treatment of patients with relapsed or refractory indolent lymphoma (follicular lymphoma or marginal zone lymphoma). REVLIMID was first approved in China in 2013 for the treatment of multiple myeloma in combination with dexamethasone, in adult patients who have received at least one prior therapy, and the label for the combination was expanded in 2018 to include adult patients with newly-diagnosed multiple myeloma (NDMM) who are not eligible for transplant. It is currently marketed in China by BeiGene under an exclusive license from Celgene Logistics Sarl, a Bristol-Myers Squibb company.
This milestone for REVLIMID marks another step in the expansion of our hematology franchise into non-Hodgkins lymphoma (NHL) in China, where significant unmet medical needs remain. Together with the pending approvals of tislelizumab for Hodgkins lymphoma and zanubrutinib for mantle cell lymphoma and chronic lymphocytic leukemia as well as Revlimid for multiple myeloma, Vidaza for myelodysplastic syndromes and acute myeloid leukemia and additional products from the collaboration we have announced with Amgen, we are working to build a market-leading presence in the treatment of hematological cancers in China, said Dr. Xiaobin Wu, General Manager of China and President of BeiGene. We are excited about this opportunity and look forward to working closely with Bristol-Myers Squibb and the NMPA to bring this chemotherapy-free treatment option to patients with relapsed or refractory follicular lymphoma or marginal zone lymphoma in China as soon as possible.
The sNDA is supported by a clinical, non-clinical, and chemistry, manufacturing and control (CMC) data package, including the results from the pivotal Phase 3 AUGMENT study (NCT01938001) sponsored and conducted by Bristol-Myers Squibb. AUGMENT is a randomized, double-blind, multicenter trial in which a total of 358 patients with relapsed or refractory follicular or marginal zone lymphoma were randomized 1:1 to receive REVLIMID and rituximab (R2) or rituximab and placebo. With a median follow-up of 28.3 months (range: 0.1 to 51.3 months), R2 demonstrated clinically meaningful and statistically significant improvement in progression-free survival (PFS), evaluated by an independent review committee (IRC), relative to the control arm with a 54% reduction in the risk of progression or death (hazard ratio [HR] = 0.46; 95% confidence interval [CI]: 0.34, 0.62; p < 0.0001). The median PFS was 39.4 months for the R2 arm and 14.1 months for the control arm with an improvement by more than 2 years. Overall response rate (ORR), a secondary endpoint, was 78% in the R2 arm vs. 53% in the control arm, as assessed by the IRC. Duration of response (DoR) was significantly improved for R2 vs. control with median DoR of 37 vs. 22 months, respectively (P =0.0015; HR: 0.53; 95% CI, 0.36-0.79). The most frequent adverse event (AE) in the R2 arm was neutropenia (58%), vs. 22% in the control arm. Additional commonly observed AEs in more than 20% of patients included diarrhea (31% in the R2 arm vs. 23% in the control arm), constipation (26% vs. 14%), cough (23% vs. 17%), and fatigue (22% vs. 18%). Adverse events that were reported at a higher rate (>10%) in the R2 arm were neutropenia, constipation, leukopenia, anemia, thrombocytopenia and tumor flare.
About follicular lymphoma (FL) and marginal zone lymphoma (MZL)
FL and MZL are two major types of indolent lymphomas;1 FL is the most common subtype, constituting approximately 20% to 25% of all NHL,2 followed by MZL (approximately 5% to 17% of all NHLs).3 NHL incidence in China is 88,090 according to the World Health Organizations Globocan 2018 database.4 Given the incurable nature of relapsed or refractory FL/MZL, the efficacy and safety limitations of current treatment options, and the fact that patients are typically older and with comorbidities, a high unmet medical need exists for the development of novel treatment options with new differentiated mechanisms of action and a more tolerable safety profile that can improve the quality of response and PFS in the setting of previously treated FL/MZL.
About REVLIMID
In China, REVLIMID was approved in combination with dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma (MM) who are not eligible for transplant in 2018. It received approval in China in 2013 for the treatment of multiple myeloma in combination with dexamethasone in adult patients who have received at least one prior therapy.
REVLIMID is approved in Europe and the United States as monotherapy, indicated for the maintenance treatment of adult patients with newly diagnosed MM who have undergone autologous stem cell transplantation. REVLIMID as combination therapy is approved in Europe, in the United States, in Japan and in around 25 other countries for the treatment of adult patients with previously untreated MM who are not eligible for transplant. REVLIMID is also approved in combination with dexamethasone for the treatment of patients with MM who have received at least one prior therapy in nearly 70 countries, encompassing Europe, the Americas, the Middle-East and Asia, and in combination with dexamethasone for the treatment of patients whose disease has progressed after one therapy in Australia and New Zealand.
REVLIMID is also approved in the United States, Canada, Switzerland, Australia, New Zealand and several Latin American countries, as well as Malaysia and Israel, for transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities and in Europe for the treatment of patients with transfusion-dependent anemia due to low- or intermediate-1-risk MDS associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.
In addition, REVLIMID is approved in Europe for the treatment of patients with mantle cell lymphoma (MCL) and in the United States for the treatment of patients with MCL whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib. In Switzerland, REVLIMID is indicated for the treatment of patients with relapsed or refractory MCL after prior therapy that included bortezomib and chemotherapy/rituximab.
REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials.
U.S. Indications for REVLIMID
REVLIMID (lenalidomide) in combination with dexamethasone (dex) is indicated for the treatment of adult patients with multiple myeloma (MM).
REVLIMID is indicated as maintenance therapy in adult patients with MM following autologous hematopoietic stem cell transplantation (auto-HSCT).
REVLIMID is indicated for the treatment of adult patients with transfusion-dependent anemia due to low-or intermediate-1risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities.
REVLIMID is indicated for the treatment of adult patients with mantle cell lymphoma (MCL) whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib.
REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated follicular lymphoma (FL).
REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated marginal zone lymphoma (MZL).
REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials.
REVLIMID is only available through a restricted distribution program, REVLIMID REMS.
Important Safety Information
WARNING: EMBRYO-FETAL TOXICITY, HEMATOLOGIC TOXICITY, and VENOUS and ARTERIAL THROMBOEMBOLISM
Embryo-Fetal Toxicity
Do not use REVLIMID during pregnancy. Lenalidomide, a thalidomide analogue, caused limb abnormalities in a developmental monkey study. Thalidomide is a known human teratogen that causes severe life-threatening human birth defects. If lenalidomide is used during pregnancy, it may cause birth defects or embryo-fetal death. In females of reproductive potential, obtain 2 negative pregnancy tests before starting REVLIMID treatment. Females of reproductive potential must use 2 forms of contraception or continuously abstain from heterosexual sex during and for 4 weeks after REVLIMID treatment. To avoid embryo-fetal exposure to lenalidomide, REVLIMID is only available through a restricted distribution program, the REVLIMID REMS program.
Information about the REVLIMID REMS program is available at http://www.celgeneriskmanagement.com or by calling the manufacturers toll-free number 1-888-423-5436.
Hematologic Toxicity (Neutropenia and Thrombocytopenia)
REVLIMID can cause significant neutropenia and thrombocytopenia. Eighty percent of patients with del 5q MDS had to have a dose delay/reduction during the major study. Thirty-four percent of patients had to have a second dose delay/reduction. Grade 3 or 4 hematologic toxicity was seen in 80% of patients enrolled in the study. Patients on therapy for del 5q MDS should have their complete blood counts monitored weekly for the first 8 weeks of therapy and at least monthly thereafter. Patients may require dose interruption and/or reduction. Patients may require use of blood product support and/or growth factors.
Venous and Arterial Thromboembolism
REVLIMID has demonstrated a significantly increased risk of deep vein thrombosis (DVT) and pulmonary embolism (PE), as well as risk of myocardial infarction and stroke in patients with MM who were treated with REVLIMID and dexamethasone therapy. Monitor for and advise patients about signs and symptoms of thromboembolism. Advise patients to seek immediate medical care if they develop symptoms such as shortness of breath, chest pain, or arm or leg swelling. Thromboprophylaxis is recommended and the choice of regimen should be based on an assessment of the patients underlying risks.
CONTRAINDICATIONS
Pregnancy: REVLIMID can cause fetal harm when administered to a pregnant female and is contraindicated in females who are pregnant. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential risk to the fetus.
Severe Hypersensitivity Reactions: REVLIMID is contraindicated in patients who have demonstrated severe hypersensitivity (e.g., angioedema, Stevens-Johnson syndrome, toxic epidermal necrolysis) to lenalidomide.
WARNINGS AND PRECAUTIONS
Embryo-Fetal Toxicity: See Boxed WARNINGS.
REVLIMID REMS Program: See Boxed WARNINGS. Prescribers and pharmacies must be certified with the REVLIMID REMS program by enrolling and complying with the REMS requirements; pharmacies must only dispense to patients who are authorized to receive REVLIMID. Patients must sign a Patient-Physician Agreement Form and comply with REMS requirements; female patients of reproductive potential who are not pregnant must comply with the pregnancy testing and contraception requirements and males must comply with contraception requirements.
Hematologic Toxicity: REVLIMID can cause significant neutropenia and thrombocytopenia. Monitor patients with neutropenia for signs of infection. Advise patients to observe for bleeding or bruising, especially with use of concomitant medications that may increase risk of bleeding. Patients may require a dose interruption and/or dose reduction. MM: Monitor complete blood counts (CBC) in patients taking REVLIMID + dexamethasone or REVLIMID as maintenance therapy, every 7 days for the first 2 cycles, on days 1 and 15 of cycle 3, and every 28 days thereafter. MDS: Monitor CBC in patients on therapy for del 5q MDS, weekly for the first 8 weeks of therapy and at least monthly thereafter. See Boxed WARNINGS for further information. MCL: Monitor CBC in patients taking REVLIMID for MCL weekly for the first cycle (28 days), every 2 weeks during cycles 2-4, and then monthly thereafter. FL/MZL: Monitor CBC in patients taking REVLIMID for FL or MZL weekly for the first 3 weeks of Cycle 1 (28 days), every 2 weeks during Cycles 2-4, and then monthly thereafter.
Venous and Arterial Thromboembolism: See Boxed WARNINGS. Venous thromboembolic events (DVT and PE) and arterial thromboses (MI and CVA) are increased in patients treated with REVLIMID. Patients with known risk factors, including prior thrombosis, may be at greater risk and actions should be taken to try to minimize all modifiable factors (e.g., hyperlipidemia, hypertension, smoking). Thromboprophylaxis is recommended and the regimen should be based on the patients underlying risks. ESAs and estrogens may further increase the risk of thrombosis and their use should be based on a benefit-risk decision.
Increased Mortality in Patients With CLL: In a clinical trial in the first-line treatment of patients with CLL, single-agent REVLIMID therapy increased the risk of death as compared to single-agent chlorambucil. Serious adverse cardiovascular reactions, including atrial fibrillation, myocardial infarction, and cardiac failure, occurred more frequently in the REVLIMID arm. REVLIMID is not indicated and not recommended for use in CLL outside of controlled clinical trials.
Second Primary Malignancies (SPM): In clinical trials in patients with MM receiving REVLIMID and in patients with FL or MZL receiving REVLIMID + rituximab therapy, an increase of hematologic plus solid tumor SPM, notably AML, have been observed. In patients with MM, MDS was also observed. Monitor patients for the development of SPM. Take into account both the potential benefit of REVLIMID and risk of SPM when considering treatment.
Increased Mortality With Pembrolizumab: In clinical trials in patients with MM, the addition of pembrolizumab to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with MM with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials.
Hepatotoxicity: Hepatic failure, including fatal cases, has occurred in patients treated with REVLIMID + dexamethasone. Pre-existing viral liver disease, elevated baseline liver enzymes, and concomitant medications may be risk factors. Monitor liver enzymes periodically. Stop REVLIMID upon elevation of liver enzymes. After return to baseline values, treatment at a lower dose may be considered.
Severe Cutaneous Reactions: Severe cutaneous reactions including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug reaction with eosinophilia and systemic symptoms (DRESS) have been reported. These events can be fatal. Patients with a prior history of Grade 4 rash associated with thalidomide treatment should not receive REVLIMID. Consider REVLIMID interruption or discontinuation for Grade 2-3 skin rash. Permanently discontinue REVLIMID for Grade 4 rash, exfoliative or bullous rash, or for other severe cutaneous reactions such as SJS, TEN, or DRESS.
Tumor Lysis Syndrome (TLS): Fatal instances of TLS have been reported during treatment with REVLIMID. The patients at risk of TLS are those with high tumor burden prior to treatment. Closely monitor patients at risk and take appropriate preventive approaches.
Tumor Flare Reaction (TFR): TFR has occurred during investigational use of REVLIMID for CLL and lymphoma. Monitoring and evaluation for TFR is recommended in patients with MCL, FL, or MZL. Tumor flare may mimic the progression of disease (PD). In patients with Grade 3 or 4 TFR, it is recommended to withhold treatment with REVLIMID until TFR resolves to Grade 1. REVLIMID may be continued in patients with Grade 1 and 2 TFR without interruption or modification, at the physicians discretion.
Impaired Stem Cell Mobilization: A decrease in the number of CD34+ cells collected after treatment (>4 cycles) with REVLIMID has been reported. Consider early referral to transplant center to optimize timing of the stem cell collection.
Thyroid Disorders: Both hypothyroidism and hyperthyroidism have been reported. Measure thyroid function before starting REVLIMID treatment and during therapy.
Early Mortality in Patients With MCL: In another MCL study, there was an increase in early deaths (within 20 weeks); 12.9% in the REVLIMID arm versus 7.1% in the control arm. Risk factors for early deaths include high tumor burden, MIPI score at diagnosis, and high WBC at baseline (10 x 109/L).
Hypersensitivity: Hypersensitivity, including angioedema, anaphylaxis, and anaphylactic reactions to REVLIMID has been reported. Permanently discontinue REVLIMID for angioedema and anaphylaxis.
ADVERSE REACTIONS
Multiple Myeloma
Myelodysplastic Syndromes
Mantle Cell Lymphoma
Follicular Lymphoma/Marginal Zone Lymphoma
DRUG INTERACTIONS
Periodically monitor digoxin plasma levels due to increased Cmax and AUC with concomitant REVLIMID therapy. Patients taking concomitant therapies such as erythropoietin-stimulating agents or estrogen-containing therapies may have an increased risk of thrombosis. It is not known whether there is an interaction between dexamethasone and warfarin. Close monitoring of PT and INR is recommended in patients with MM taking concomitant warfarin.
USE IN SPECIFIC POPULATIONS
Please see full Prescribing Information, including Boxed WARNINGS, for REVLIMID.
Please see the rituximab full Prescribing Information for Important Safety Information at http://www.rituxan.com.
About BeiGene
BeiGene is a global, commercial-stage, research-based biotechnology company focused on molecularly-targeted and immuno-oncology cancer therapeutics. With a team of over 3,000 employees in the United States, China, Australia, and Europe; BeiGene is advancing a pipeline consisting of novel oral small molecules and monoclonal antibodies for cancer. BeiGene is also working to create combination solutions aimed to have both a meaningful and lasting impact on cancer patients. In the United States, BeiGene markets and distributes BRUKINSA (zanubrutinib) and in China, the Company markets ABRAXANE (paclitaxel for injection [albumin bound]), REVLIMID (lenalidomide), and VIDAZA (azacitidine) under a license from Celgene Logistics Sarl, a Bristol-Myers Squibb company.5
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding BeiGenes plans and expectations for further development and commercialization of REVLIMID in China and the potential implications for patients. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGenes limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates, as well as those risks more fully discussed in the section entitled Risk Factors in BeiGenes most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.
______________________1 Bello C, Zhang L, Naghashpour M. Follicular lymphoma: current management and future directions. Cancer Control. 2012;19:187-95.
2 Sousou T, Friedberg J. Rituximab in indolent lymphomas. Semin Hematol. 2010; 47(2):133-42.
3 Zinzani, P. L. (2012). The many faces of marginal zone lymphoma. Hematology, 2012(1), 426432.
5 ABRAXANE is registered trademark of Abraxis Bioscience LLC, a Bristol-Myers Squibb company; REVLIMID and VIDAZA are registered trademarks of Celgene Corporation, a Bristol-Myers Squibb company.
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BeiGene Announces Acceptance of a Supplemental New Drug Application in China for REVLIMID in Relapsed or Refractory Indolent Lymphoma - BioSpace
Stem Cell Therapy Market Consumer Outlook 2025 | MEDIPOST Co., Ltd., Osiris Therapeutics, Inc. – Market Research Sheets
By daniellenierenberg
Stem Cell Therapy Market: Snapshot
Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.
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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.
It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.
Global Stem Cell Therapy Market: Overview
Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.
Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.
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Global Stem Cell Therapy Market: Key Trends
The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.
On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.
Global Stem Cell Therapy Market: Market Potential
A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.
In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.
Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.
Global Stem Cell Therapy Market: Regional Outlook
The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.
Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.
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Global Stem Cell Therapy Market: Competitive Analysis
Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.
Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.
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Stem Cell Therapy Market Consumer Outlook 2025 | MEDIPOST Co., Ltd., Osiris Therapeutics, Inc. - Market Research Sheets
BeiGene Announces Acceptance of a Supplemental New Drug Application in China for REVLIMID in Relapsed or Refractory Indolent Lymphoma – GlobeNewswire
By daniellenierenberg
BEIJING, China and CAMBRIDGE, Mass., Dec. 22, 2019 (GLOBE NEWSWIRE) -- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, today announced that the China National Medical Products Administration (NMPA) has accepted a supplemental new drug application (sNDA) for REVLIMID (lenalidomide), in combination with rituximab, for the treatment of patients with relapsed or refractory indolent lymphoma (follicular lymphoma or marginal zone lymphoma). REVLIMID was first approved in China in 2013 for the treatment of multiple myeloma in combination with dexamethasone, in adult patients who have received at least one prior therapy, and the label for the combination was expanded in 2018 to include adult patients with newly-diagnosed multiple myeloma (NDMM) who are not eligible for transplant. It is currently marketed in China by BeiGene under an exclusive license from Celgene Logistics Sarl, a Bristol-Myers Squibb company.
This milestone for REVLIMID marks another step in the expansion of our hematology franchise into non-Hodgkins lymphoma (NHL) in China, where significant unmet medical needs remain. Together with the pending approvals of tislelizumab for Hodgkins lymphoma and zanubrutinib for mantle cell lymphoma and chronic lymphocytic leukemia as well as Revlimid for multiple myeloma, Vidaza for myelodysplastic syndromes and acute myeloid leukemia and additional products from the collaboration we have announced with Amgen, we are working to build a market-leading presence in the treatment of hematological cancers in China, said Dr. Xiaobin Wu, General Manager of China and President of BeiGene. We are excited about this opportunity and look forward to working closely with Bristol-Myers Squibb and the NMPA to bring this chemotherapy-free treatment option to patients with relapsed or refractory follicular lymphoma or marginal zone lymphoma in China as soon as possible.
The sNDA is supported by a clinical, non-clinical, and chemistry, manufacturing and control (CMC) data package, including the results from the pivotal Phase 3 AUGMENT study (NCT01938001) sponsored and conducted by Bristol-Myers Squibb. AUGMENT is a randomized, double-blind, multicenter trial in which a total of 358 patients with relapsed or refractory follicular or marginal zone lymphoma were randomized 1:1 to receive REVLIMID and rituximab (R2) or rituximab and placebo. With a median follow-up of 28.3 months (range: 0.1 to 51.3 months), R2 demonstrated clinically meaningful and statistically significant improvement in progression-free survival (PFS), evaluated by an independent review committee (IRC), relative to the control arm with a 54% reduction in the risk of progression or death (hazard ratio [HR] = 0.46; 95% confidence interval [CI]: 0.34, 0.62; p < 0.0001). The median PFS was 39.4 months for the R2 arm and 14.1 months for the control arm with an improvement by more than 2 years. Overall response rate (ORR), a secondary endpoint, was 78% in the R2 arm vs. 53% in the control arm, as assessed by the IRC. Duration of response (DoR) was significantly improved for R2 vs. control with median DoR of 37 vs. 22 months, respectively (P =0.0015; HR: 0.53; 95% CI, 0.36-0.79). The most frequent adverse event (AE) in the R2 arm was neutropenia (58%), vs. 22% in the control arm. Additional commonly observed AEs in more than 20% of patients included diarrhea (31% in the R2 arm vs. 23% in the control arm), constipation (26% vs. 14%), cough (23% vs. 17%), and fatigue (22% vs. 18%). Adverse events that were reported at a higher rate (>10%) in the R2 arm were neutropenia, constipation, leukopenia, anemia, thrombocytopenia and tumor flare.
About follicular lymphoma (FL) and marginal zone lymphoma (MZL)
FL and MZL are two major types of indolent lymphomas;1 FL is the most common subtype, constituting approximately 20% to 25% of all NHL,2 followed by MZL (approximately 5% to 17% of all NHLs).3 NHL incidence in China is 88,090 according to the World Health Organizations Globocan 2018 database.4 Given the incurable nature of relapsed or refractory FL/MZL, the efficacy and safety limitations of current treatment options, and the fact that patients are typically older and with comorbidities, a high unmet medical need exists for the development of novel treatment options with new differentiated mechanisms of action and a more tolerable safety profile that can improve the quality of response and PFS in the setting of previously treated FL/MZL.
About REVLIMID
In China, REVLIMID was approved in combination with dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma (MM) who are not eligible for transplant in 2018. It received approval in China in 2013 for the treatment of multiple myeloma in combination with dexamethasone in adult patients who have received at least one prior therapy.
REVLIMID is approved in Europe and the United States as monotherapy, indicated for the maintenance treatment of adult patients with newly diagnosed MM who have undergone autologous stem cell transplantation. REVLIMIDas combination therapy is approved inEurope, inthe United States, inJapanand in around 25 other countries for the treatment of adult patients with previously untreated MM who are not eligible for transplant. REVLIMID is also approved in combination with dexamethasone for the treatment of patients with MM who have received at least one prior therapy in nearly 70 countries, encompassingEurope, theAmericas, theMiddle-EastandAsia, and in combination with dexamethasone for the treatment of patients whose disease has progressed after one therapy inAustralia and New Zealand.
REVLIMIDis also approved inthe United States,Canada,Switzerland,Australia,New Zealandand several Latin American countries, as well asMalaysiaandIsrael, for transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities and inEuropefor the treatment of patients with transfusion-dependent anemia due to low- or intermediate-1-risk MDS associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.
In addition, REVLIMIDis approved inEuropefor the treatment of patients with mantle cell lymphoma (MCL) and inthe United Statesfor the treatment of patients with MCL whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib. InSwitzerland, REVLIMID is indicated for the treatment of patients with relapsed or refractory MCL after prior therapy that included bortezomib and chemotherapy/rituximab.
REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials.
U.S. Indications for REVLIMID
REVLIMID (lenalidomide) in combination with dexamethasone (dex) is indicated for the treatment of adult patients with multiple myeloma (MM).
REVLIMID is indicated as maintenance therapy in adult patients with MM following autologous hematopoietic stem cell transplantation (auto-HSCT).
REVLIMID is indicated for the treatment of adult patients with transfusion-dependent anemia due to low-or intermediate-1risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities.
REVLIMID is indicated for the treatment of adult patients with mantle cell lymphoma (MCL) whose disease has relapsed or progressed after two prior therapies, one of which included bortezomib.
REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated follicular lymphoma (FL).
REVLIMID in combination with a rituximab product is indicated for the treatment of adult patients with previously treated marginal zone lymphoma (MZL).
REVLIMID is not indicated and is not recommended for the treatment of patients with chronic lymphocytic leukemia (CLL) outside of controlled clinical trials.
REVLIMID is only available through a restricted distribution program, REVLIMID REMS.
Important Safety Information
WARNING: EMBRYO-FETAL TOXICITY, HEMATOLOGIC TOXICITY, and VENOUS and ARTERIAL THROMBOEMBOLISM
Embryo-Fetal Toxicity
Do not use REVLIMID during pregnancy. Lenalidomide, a thalidomide analogue, caused limb abnormalities in a developmental monkey study. Thalidomide is a known human teratogen that causes severe life-threatening human birth defects. If lenalidomide is used during pregnancy, it may cause birth defects or embryo-fetal death. In females of reproductive potential, obtain 2 negative pregnancy tests before starting REVLIMID treatment. Females of reproductive potential must use 2 forms of contraception or continuously abstain from heterosexual sex during and for 4 weeks after REVLIMID treatment. To avoid embryo-fetal exposure to lenalidomide, REVLIMID is only available through a restricted distribution program, the REVLIMID REMS program.
Information about the REVLIMID REMS program is available at http://www.celgeneriskmanagement.com or by calling the manufacturers toll-free number 1-888-423-5436.
Hematologic Toxicity (Neutropenia and Thrombocytopenia)
REVLIMID can cause significant neutropenia and thrombocytopenia. Eighty percent of patients with del 5q MDS had to have a dose delay/reduction during the major study. Thirty-four percent of patients had to have a second dose delay/reduction. Grade 3 or 4 hematologic toxicity was seen in 80% of patients enrolled in the study. Patients on therapy for del 5q MDS should have their complete blood counts monitored weekly for the first 8 weeks of therapy and at least monthly thereafter. Patients may require dose interruption and/or reduction. Patients may require use of blood product support and/or growth factors.
Venous and Arterial Thromboembolism
REVLIMID has demonstrated a significantly increased risk of deep vein thrombosis (DVT) and pulmonary embolism (PE), as well as risk of myocardial infarction and stroke in patients with MM who were treated with REVLIMID and dexamethasone therapy. Monitor for and advise patients about signs and symptoms of thromboembolism. Advise patients to seek immediate medical care if they develop symptoms such as shortness of breath, chest pain, or arm or leg swelling. Thromboprophylaxis is recommended and the choice of regimen should be based on an assessment of the patients underlying risks.
CONTRAINDICATIONS
Pregnancy: REVLIMID can cause fetal harm when administered to a pregnant female and is contraindicated in females who are pregnant. If this drug is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential risk to the fetus.
Severe Hypersensitivity Reactions: REVLIMID is contraindicated in patients who have demonstrated severe hypersensitivity (e.g., angioedema, Stevens-Johnson syndrome, toxic epidermal necrolysis) to lenalidomide.
WARNINGS AND PRECAUTIONS
Embryo-Fetal Toxicity: See Boxed WARNINGS.
REVLIMID REMS Program: See Boxed WARNINGS. Prescribers and pharmacies must be certified with the REVLIMID REMS program by enrolling and complying with the REMS requirements; pharmacies must only dispense to patients who are authorized to receive REVLIMID. Patients must sign a Patient-Physician Agreement Form and comply with REMS requirements; female patients of reproductive potential who are not pregnant must comply with the pregnancy testing and contraception requirements and males must comply with contraception requirements.
Hematologic Toxicity: REVLIMID can cause significant neutropenia and thrombocytopenia. Monitor patients with neutropenia for signs of infection. Advise patients to observe for bleeding or bruising, especially with use of concomitant medications that may increase risk of bleeding. Patients may require a dose interruption and/or dose reduction. MM: Monitor complete blood counts (CBC) in patients taking REVLIMID + dexamethasone or REVLIMID as maintenance therapy, every 7 days for the first 2 cycles, on days 1 and 15 of cycle 3, and every 28 days thereafter. MDS: Monitor CBC in patients on therapy for del 5q MDS, weekly for the first 8 weeks of therapy and at least monthly thereafter. See Boxed WARNINGS for further information. MCL: Monitor CBC in patients taking REVLIMID for MCL weekly for the first cycle (28 days), every 2 weeks during cycles 2-4, and then monthly thereafter. FL/MZL: Monitor CBC in patients taking REVLIMID for FL or MZL weekly for the first 3 weeks of Cycle 1 (28 days), every 2 weeks during Cycles 2-4, and then monthly thereafter.
Venous and Arterial Thromboembolism: See Boxed WARNINGS. Venous thromboembolic events (DVT and PE) and arterial thromboses (MI and CVA) are increased in patients treated with REVLIMID. Patients with known risk factors, including prior thrombosis, may be at greater risk and actions should be taken to try to minimize all modifiable factors (e.g., hyperlipidemia, hypertension, smoking). Thromboprophylaxis is recommended and the regimen should be based on the patients underlying risks. ESAs and estrogens may further increase the risk of thrombosis and their use should be based on a benefit-risk decision.
Increased Mortality in Patients With CLL: In a clinical trial in the first-line treatment of patients with CLL, single-agent REVLIMID therapy increased the risk of death as compared to single-agent chlorambucil. Serious adverse cardiovascular reactions, including atrial fibrillation, myocardial infarction, and cardiac failure, occurred more frequently in the REVLIMID arm. REVLIMID is not indicated and not recommended for use in CLL outside of controlled clinical trials.
Second Primary Malignancies (SPM): In clinical trials in patients with MM receiving REVLIMID and in patients with FL or MZL receiving REVLIMID + rituximab therapy, an increase of hematologic plus solid tumor SPM, notably AML, have been observed. In patients with MM, MDS was also observed. Monitor patients for the development of SPM. Take into account both the potential benefit of REVLIMID and risk of SPM when considering treatment.
Increased Mortality With Pembrolizumab: In clinical trials in patients with MM, the addition of pembrolizumab to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with MM with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials.
Hepatotoxicity: Hepatic failure, including fatal cases, has occurred in patients treated with REVLIMID + dexamethasone. Pre-existing viral liver disease, elevated baseline liver enzymes, and concomitant medications may be risk factors. Monitor liver enzymes periodically. Stop REVLIMID upon elevation of liver enzymes. After return to baseline values, treatment at a lower dose may be considered.
Severe Cutaneous Reactions: Severe cutaneous reactions including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN), and drug reaction with eosinophilia and systemic symptoms (DRESS) have been reported. These events can be fatal. Patients with a prior history of Grade 4 rash associated with thalidomide treatment should not receive REVLIMID. Consider REVLIMID interruption or discontinuation for Grade 2-3 skin rash. Permanently discontinue REVLIMID for Grade 4 rash, exfoliative or bullous rash, or for other severe cutaneous reactions such as SJS, TEN, or DRESS.
Tumor Lysis Syndrome (TLS): Fatal instances of TLS have been reported during treatment with REVLIMID. The patients at risk of TLS are those with high tumor burden prior to treatment. Closely monitor patients at risk and take appropriate preventive approaches.
Tumor Flare Reaction (TFR): TFR has occurred during investigational use of REVLIMID for CLL and lymphoma. Monitoring and evaluation for TFR is recommended in patients with MCL, FL, or MZL. Tumor flare may mimic the progression of disease (PD). In patients with Grade 3 or 4 TFR, it is recommended to withhold treatment with REVLIMID until TFR resolves to Grade 1. REVLIMID may be continued in patients with Grade 1 and 2 TFR without interruption or modification, at the physicians discretion.
Impaired Stem Cell Mobilization: A decrease in the number of CD34+ cells collected after treatment (>4 cycles) with REVLIMID has been reported. Consider early referral to transplant center to optimize timing of the stem cell collection.
Thyroid Disorders: Both hypothyroidism and hyperthyroidism have been reported. Measure thyroid function before starting REVLIMID treatment and during therapy.
Early Mortality in Patients With MCL: In another MCL study, there was an increase in early deaths (within 20 weeks); 12.9% in the REVLIMID arm versus 7.1% in the control arm. Risk factors for early deaths include high tumor burden, MIPI score at diagnosis, and high WBC at baseline (10 x 109/L).
Hypersensitivity: Hypersensitivity, including angioedema, anaphylaxis, and anaphylactic reactions to REVLIMID has been reported. Permanently discontinue REVLIMID for angioedema and anaphylaxis.
ADVERSE REACTIONS
Multiple Myeloma
Myelodysplastic Syndromes
Mantle Cell Lymphoma
Follicular Lymphoma/Marginal Zone Lymphoma
DRUG INTERACTIONS
Periodically monitor digoxin plasma levels due to increased Cmax and AUC with concomitant REVLIMID therapy. Patients taking concomitant therapies such as erythropoietin-stimulating agents or estrogen-containing therapies may have an increased risk of thrombosis. It is not known whether there is an interaction between dexamethasone and warfarin. Close monitoring of PT and INR is recommended in patients with MM taking concomitant warfarin.
USE IN SPECIFIC POPULATIONS
Please see full Prescribing Information, including Boxed WARNINGS, for REVLIMID.
Please see the rituximab full Prescribing Information for Important Safety Information at http://www.rituxan.com.
About BeiGene
BeiGene is a global, commercial-stage, research-based biotechnology company focused on molecularly-targeted and immuno-oncology cancer therapeutics. With a team of over 3,000 employees in the United States, China, Australia, and Europe; BeiGene is advancing a pipeline consisting of novel oral small molecules and monoclonal antibodies for cancer. BeiGene is also working to create combination solutions aimed to have both a meaningful and lasting impact on cancer patients. In the United States, BeiGene markets and distributes BRUKINSA (zanubrutinib) and in China, the Company markets ABRAXANE (paclitaxel for injection [albumin bound]), REVLIMID (lenalidomide), and VIDAZA (azacitidine) under a license from Celgene Logistics Sarl, a Bristol-Myers Squibb company.5
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding BeiGenes plans and expectations for further development and commercialization of REVLIMID in China and the potential implications for patients. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGenes limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates, as well as those risks more fully discussed in the section entitled Risk Factors in BeiGenes most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.
______________________1 Bello C, Zhang L, Naghashpour M. Follicular lymphoma: current management and future directions. Cancer Control. 2012;19:187-95.
2 Sousou T, Friedberg J. Rituximab in indolent lymphomas. Semin Hematol. 2010; 47(2):133-42.
3 Zinzani, P. L. (2012). The many faces of marginal zone lymphoma. Hematology, 2012(1), 426432.
5 ABRAXANEis registered trademark ofAbraxis Bioscience LLC, aBristol-Myers Squibb company; REVLIMIDand VIDAZAare registered trademarks ofCelgene Corporation, aBristol-Myers Squibbcompany.
Wisconsin teen diagnosed with cancer while battling rare ‘butterfly skin’ disease: ‘He is resilient’ – Fox News
By daniellenierenberg
At just 13 years old,Charlie Knuth, of Darboy, Wisc., has known more pain than most others do in a lifetime. The teen, who suffers from epidermolysis bullosa, a rare disease that causes his skin to blister incredibly easily, is near-constantly wrapped in bandages to protect his fragile skin. He takes special baths to soothe his sores, which can form from the slightest touch and are lanced before he is covered in fresh dressings.But the so-called butterfly child a name often given to EB sufferers as their skins fragility is similar to that of a butterfly wing has another battle ahead: cancer.
Its unimaginable, Trisha Knuth, Charlies mother, told Fox News. Even as his mom, when I see him taking it in stride, I cant even believe that he can.
BOY, 2, HAS RARE 'SCALE'-LIKE SKIN CONDITION THAT AFFECTS 1 IN 500,000: 'HES OVERCOME SO MUCH'
Charlies biological parents abandoned him at the hospital shortly after his birth. Knuth and her husband, Kevin, had long fostered children with complex medical needs. But just weeks before they received a call about Charlie, they were readying to let their license expire; the tragic cases were simply becoming too much. Even so, Knuth said shecouldnt say no to Charlie she knew to do so was likely a death sentence. They began the lengthy adoption process shortly after bringing him home.
Trisha Knuth and Charlie, 13. (Trisha Knuth/Facebook)
When I went to the children's hospital in Milwaukee, he was slathered from head-to-toe in Vaseline," she recalled."Nobody ever came for him. I worked with the nurses and learned his care but EB is so rare that many hospitals don't know how to care for those with [the condition]. They sent me home with morphine and a few things and it was a learning process from there.
Thirteen years later, Charlie didnt end up dying, he ended up thriving, she said. When he was 5 years old, he underwent an experimental skin grafting procedure at the University of Minnesota in an attempt to make his skin stronger and less prone to blistering. Knuth called it a transformation for her young son, who had two really good years before his body rejected the graft and he began to suffer from aplastic anemia, a potentially deadly condition that occurs when the body doesn't produce enough red blood cells.
In 2012, he underwent a stem cell transplant in an attempt to treat his severe EB. He was hospitalized for six months but eventually pulled through.
Charlie, who suffers from EB, was recently diagnosed with cancer. (Trisha Knuth/Facebook)
Hes done pretty well after that second time. But he is constantly wounded, very fragile, said Knuth.
But in recent months, Charlie began to complain of a sore throat not uncommon for those with EB, as blisters can form on the inside of the body as well on the outside. The mouth and throat are commonly affected.But there were no visible blisters, raising his doctor's suspicions. ACT scan later revealed enlarged lymph nodes in his neck and armpits. A biopsy later confirmed lymphoma, a type of cancer that affects the bodys lymphatic system. Knuth called the diagnosis another hurdle in his very hard life.
The pain was masked by EB. Its hard to tell whats what because EB causes so much pain, she said.
Cancer treatment often consisting of chemotherapy, radiation, and surgery is hard enough on an average persons body. But those with EB face an entirely different battle; Knuth said nurses inserting an IV cant use medical tape to help attach the drip, as the adhesive ripsher sons skin when removed. Oxygen and anesthesia masks are often a struggle as well, as are blood pressure cuffs.
Charlie (R) when he was younger. (Trisha Knuth/Facebook)
How do you treat someone who cant be touched? Knuth questioned, noting she has gone into the operating room with Charlie in times past to ensure he is not injured. You cant even imagine. [Its like] being burned every day, and then bandaged, and nowundergoing cancer treatment it boggles the mind.
When speaking to Fox News, Knuth and Charlie were in Minnesota, where doctors are working to build atreatment plan. The day after Christmas which the pair are celebrating in an Airbnb Charlie is slated to undergo a procedure to remove fluid from his spine and bone marrow from his hips. One of his affected lymph nodes will be taken for further testing.
In the meantime, Kevin is home with the couples 2-year-old adopted daughter, who also suffers from EB.
"He puts on a great outward attitude, but I know there is trauma."
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He is very brave and resilient and funny, said Knuth of Charlie. But in addition to the physical pain, He does have emotional pain; he puts on a great outward attitude, but I know there is trauma.
When asked how she and Kevin manage it all, Knuth acknowledged theirs is a crazy life. But, she quickly noted, I am very happy with this life. Its hard. But when I die, I'll know my life was fulfilled and great.
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Wisconsin teen diagnosed with cancer while battling rare 'butterfly skin' disease: 'He is resilient' - Fox News
Essential oil compound may speed the healing of wounds – New Atlas
By daniellenierenberg
Although essential oils are typically associated with aromatherapy, new research indicates that medicines based on them could also help to heal skin wounds when applied topically. It all comes down to a certain substance in some of the oils, that reduces inflammation.
The chemical compound in question is known as beta-carophyllene it's found in the oils of lavender, rosemary and ylang ylang, among other sources. In a study conducted at Indiana University, beta-carophyllene extracted from these plants was applied to superficial wounds on mice.
It was observed that doing so increased cell growth and cell migration to the wound site, causing the injuries to heal faster than similar untreated wounds. Additionally, the scientists noted increased gene expression of hair follicle stem cells in the treated injuries. This suggests that there would ultimately be less scarring.
Based on previous research, it was already known that beta-carophyllene activates a receptor in the body, which in turn produces an anti-inflammatory response. It is this response that is likely the key.
"In the wound healing process, there are several stages, starting from the inflammatory phase, followed by the cell proliferation stage and the remodelling stage," says the lead scientist, Assoc. Prof. Sachiko Koyama. "I thought maybe wound healing would be accelerated if inflammation was suppressed, stimulating an earlier switch from the inflammatory stage to the next stage."
That said, Koyama believes that there may be additional factors at work, which further research should hopefully reveal. She also advises against simply applying essential oils to wounds, as the beta-carophyllene used in the study was of a known purity, and was diluted in a specific concentration.
"There are many things to test before we can start using it clinically, but our results are very promising and exciting," she says. "Someday in the near future we may be able to develop a drug, and drug delivery methods, using the chemical compounds found in essential oils."
A paper on the research was published this week in the journal PLOS ONE.
Source: Indiana University
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Essential oil compound may speed the healing of wounds - New Atlas
Scientists hope MND cure is a step closer after stem cell breakthrough discovery – The National
By daniellenierenberg
SCIENTISTS hope a cure for motor neurone disease (MND) is a step closer after a research breakthrough identified cells key to the degenerative condition.
There is currently no known cure for MND, which causes signals from motor neurone nerve cells in the brain and spinal cord needed to control movement to gradually stop reaching the muscles.
Notable people who have lived with MND include Scottish rugby star Doddie Weir and Stephen Hawking.
Researchers used stem cell technology to identify a type of cell that can cause motor neurones to fail.
Using stem cells from patient skin samples, they found glial cells, which normally support neurones in the brain and spinal cord, become damaging to motor neurones in the patients with the condition.
By testing different combinations of glial cells and motor neurones grown together in the lab, researchers found glial cells from MND patients can cause motor neurones in healthy people to stop producing the electrical signals needed to control muscles.
READ MORE:BBCSports Personality of the Year award to honour Doddie Weir
Gareth Miles, a professor of neuroscience at the University of St Andrews, helped lead the joint project with the University of Edinburgh.
Miles said: We are very excited by these new findings, which clearly point the finger at glial cells as key players in this devastating disease.
Interestingly, the negative influence of glial cells seems to prevent motor neurones from fulfilling their normal roles, even before the motor neurones show signs of dying.
We hope that this new information highlights targets for the development of much-needed treatments and ultimately a cure for MND.
The joint research was published in the scientific journal Glia.
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Scientists hope MND cure is a step closer after stem cell breakthrough discovery - The National
Boston Stem Cell Center – Bone Marrow Stem Cells in …
By daniellenierenberg
The problem with the embryonic stem cells are the many complications associated with them. Besides the ethical considerations, from a practical point of view, we are still a long way from being able to utilize these cells in a safe and consistent manner.
When using embryonic stem cells, you are inheriting any potential diseases that the baby may have. For instance, the baby may have a gene that increases susceptibility to cancer. In fact, the embryonic cells themselves may act as a tumor since there is no natural check on these cells. Furthermore, these cells are foreign materials to the body, and the body will react and attack these cells in an immune response. This can sometimes cause a serious medical condition called graft versus host disease. In that case, the patient may have to be placed on immunosuppressant drugslike an organ transplant patient. With our present technology, embryonic stem cells are not the answer. For those reasons, the FDA has put significant restrictions on the use of this type of cell in humans.
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Boston Stem Cell Center - Bone Marrow Stem Cells in ...
National Marrow Donor Program/Be The Match Applauds Congress for Its Support of Patients with Blood Cancers and Other Diseases – Benzinga
By daniellenierenberg
Bipartisan legislation provides record funding for lifesaving cellular transplants and eliminates a Medicare payment barrier for seniors.
Washington, DC, December 20, 2019 --(PR.com)-- National Marrow Donor Program (NMDP)/Be The Match applauds Congress for passing bipartisan legislation that provides record levels of funding to increase access for patients to cellular transplants, which can be the only curative treatments for blood cancers such as leukemia or lymphoma and other blood diseases. The bill also increases access to these same therapies for senior citizens by fixing a Medicare reimbursement issue that can be a barrier for them to these life-saving procedures.
Increasing funding levels for these programs and bringing Medicare payment policies for these procedures up to date represents major victories for the 1.3 million Americans fighting blood cancers, said Brian Lindberg, Chief Legal Officer and General Counsel of NMDP/Be The Match. By increasing funding for life-saving cellular transplants and removing Medicare barriers that inhibit access to care, Congress has given hope to patients in need of these curative treatments.
We are honored to have broad support from members in the House and Senate who stand with us and our mission to find matched donors for every patient in need of these cellular therapies, Lindberg added. Increasing patient access to life-saving bone marrow and cord blood transplant is NMDP/Be The Matchs top priority.
The program works closely with organizations throughout the nation to recruit volunteer donors for the registry and with public and private insurers to ensure that all patients have equal access to treatment.
The $30 million included in the final legislation for the C.W. Bill Young Cell Transplantation Program, an increase of $5.4 million over last year, and the $17.3 million for the National Cord Blood Inventory, an increase of $1.0 million, will help reduce barriers to transplant by:
Advancing new and innovative methods of providing the best possible transplant to every patient in need, regardless of socioeconomic status, age, ethnic ancestry, or any other individually defining characteristic; Continuing to simplify processes and systems to reduce time to transplant, providing the patient and their physician the therapy the patient needs exactly when he/she needs it; and Protecting access to transplant by allowing NMDP to pursue our vision of achieving equal outcomes for all.
In the case of older Americans, inadequate Medicare transplant reimbursement, primarily for donor-related costs, poses a significant barrier to patient access.
Unlike Medicare payment policies for the acquisition of solid organs for transplant, Medicare does not provide separate payments for the cost of acquiring the cells for transplant (which can include the cost of identifying genetically matched donors, collecting the cells, and transporting them to the transplant hospital). As a result, hospitals take substantial financial losses on these life-saving procedures, which often require a 20-to-30-day hospital stay on average, because the reimbursement rate does not come close to covering the true costs of treatment.
NMDP/Be The Match looks forward to working closely with the Centers for Medicare & Medicaid Services (CMS), which operates the Medicare program, to ensure that this critical payment reform is implemented as quickly as possible, so that Medicare beneficiaries are not at risk of being denied the bone marrow, peripheral blood stem cell, or cord blood transplant they need to survive.
About National Marrow Donor Program/Be The MatchFor people with life-threatening blood cancers such as leukemia and lymphoma, a cure exists. National Marrow Donor Program(NMDP)/Be The Match connects patients with their donor match for a life-saving marrow or umbilical cord blood transplant and works to identify and eliminate financial and other barriers faced by these patients. NMDP also provides patients and their families one-on-one support, education, and guidance before, during and after transplant.
Contact Information:National Marrow Donor ProgramEllen Almond(703) 548-0019Contact via Emailhttps://bethematch.org/
Read the full story here: https://www.pr.com/press-release/802090
Press Release Distributed by PR.com
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National Marrow Donor Program/Be The Match Applauds Congress for Its Support of Patients with Blood Cancers and Other Diseases - Benzinga
Why Proper Hydration Is Necessary for the Body to Function – Guardian Liberty Voice
By daniellenierenberg
Some people believe that it is impossible to ionize water, others believe the product is too good to be true.
It is important to note that the ionization of water can be tested using scientific measuring devices such as pH and ORP meters. According to The Miraculous Properties of Ionized Water, the changes that ionization produceareradical, immediate, and measurable.
Ionized water is the charging of water by creating negative ions (-OH) and positive ions (+OH). They are created through electrolysis and then the ions are separated through a membrane, so the water is negatively charged.
There has been a considerable amount of research conducted on the effects of ionized water. Most of the research has been conducted by the Japanese. All of the research has arrived at the same conclusion: ionized water benefits everything it comes in contact with as long as its used correctly. There is hard scientific evidence to back up this claim.
There are strong detoxing effects of ionized water, which proves it is not like any other water consumed. These detoxing effects are due to the small water-molecule cluster size. Water-molecule cluster size is measured using a Nuclear Magnetic Resonance device. The change in the surface tension of ionized water can also be determined. It is measured in dynes. The lower the surface tension, the smaller the water-molecule clusters.
Water is the most essential element needed by the body; therefore, health begins with water. It is impossible to be truly healthy without being properly hydrated.
When babies are born, their bones are mostly water. They are more pliable at birth than at any other time in their lives. As people grow older, the bones dry out and become brittle. The body takes the calcium from the bones to distribute it to other parts of the body that need it more.
By the age of 65, the average person is 50-70 percent dehydrated.This is whythe elderly are riddled with disease and constipation.
Bone marrow is critical to the immune system because it contains T-cells, B-cells and other cells that together form the immune system. White blood cells produce antibodies that have a multitude of other immune functions. Red blood cells carry oxygen, produce stem cells and blood platelets that allow the blood to clot.
Stem cells are primitive cells that continue to divide into infinity and form any other cell the body needs. These cells are birthed out of bone marrow, which makes hydration critical to the body. It is imperative to keep bone marrow hydrated, cleansed, and rejuvenated for great health and longevity.
Every single organ in the body requires water to properly function to its full capacity. The body is 69 percent water. The brain is 85 percent water, bones 35 percent water, blood 83 percent water, and the liveris90 percent water.
When the body is dehydrated, it puts ones health in immediate danger. Each day people should drink half their body weight in ounces. However, The Miraculous Properties of Ionized Water recommends people drink much more than half their body weight in water because that amount of water is lost through basic functions of the human body, such as urinating, sweating, breathing, and defecating.
The National Research Council guidelines suggest that the body requires one milliliter of water for every calorie of food consumed, which tends to be half of ones body weight. For example, if one weighs 200 pounds, they should drink 100 ounces of water. According to the official report from the National Research Council states that most of this quantity is contained in prepared foods. This implies that people will get the water they need from the foods they eat and drinking water is not necessary.
These types of misconceptions lead people to believe that water is a choice, not a necessity. Digestion is dehydrating and requires a large amount of water from the bodys reserves. Additionally, the typical American diet consists of dry, cooked and processed foods. Raw foods contain significant amounts of water and are better for ones diet.
There are some who believe that water consumed in raw foods is enough to hydrate the body. This is not true. Even if one could eat enough raw foods to hydrate the body, water would still be required to flush the digestive tract between meals. This is one of the healthiest things one can do. None of the bodys processes will function to capacity if the body is not well hydrated.
By Jeanette Vietti
Source:
The Miraculous Properties of Ionized Water
Image Courtesy of Rubbermaid Products Flickr Page Creative Commons License
body, spot
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Why Proper Hydration Is Necessary for the Body to Function - Guardian Liberty Voice
These Cult Beauty Brands Offer The Most Luxurious Spa Experience at Their Boutiques: Sulwhasoo, La Mer, Dior and More – Singapore Tatler
By daniellenierenberg
By Chloe Pek December 20, 2019
How to make the most of your holy grail skincare products at these beauty spas in Singapore
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We all have our favourite beauty brandsit could be a holy grail skincare product or a go-to beauty routine you rely on for a clear, radiant complexion. But are you truly making the most out of your skincare products? Only the experts will knowwhich is why there's no better way to experience your cult favourites than at their boutiques, where assistants are at hand to offer tips and tricks.
These beauty brands below do it better: with a complete spa experience that will leave your skin glowing and your mind, relaxed.
(Related: Biohack Your Way To Beauty And Health Using Your DNA And Stem Cells At These Wellness Retreats Around The World)
Want to harness the full benefits of SK-IIs miracle water? Then the SK-II Boutique Spa by Senze Salus is the place to go. Operating under an exclusive license by SK-II, the spa offers a selection of facials that last between 60 minutes to 105 minutes, catering to a myriad of skincare concerns like hydration, uneven skin tone, and clogged pores.
Besides their trademark miracle water, pure water is also the secret to their holistic spa experience. A four-stage water filtration system ensures that all water used in treatments are free of impurities, and only 100 per cent pure distilled water is used for facial steaming. Theres even a lot of thought put into the water that they serveonly alkaline water for hydration and antioxidant properties.
(Related: Why Water Is The Essence Of SK-II's Spa Treatments)
Currently exclusive to CldePeauBeauts Diamond tier members, the beauty brands SoindeBeaut offers the ultimate experience of their signature skincare ranges. Treatments available include the 60-minute Intensive Brightening Facial Treatment using CldePeauBeauts Key Radiance Care and targeted Brightening range for a radiant, glowing complexion, as well as the 60-minute Firming Supreme Facial Treatment that helps to firm and lift contours while enhancing your glow with the brands Supreme skincare range.
These services are available at CldePeauBeauts Mandarin Gallery flagship and department store counters.
(Related: Cl de Peau Beaut Revamps Its Signature La Crme Face Cream For Spring 2020)
Touted as the temple of Dior beauty, Dior Institut strives to offer a unique sensory experience for every guest. Each treatment begins with a consultation and examination of the skin with a Dior Skincare Expert to address skincare concerns. Then, a massage using Dior Instituts exclusive tissue massage techniques help to soothe customers and relieve muscle tensions.
The wide suite of services include the Brightening and Radiance-Activating Treatment, Age-Delay and Beautifying Treatment, the Dior Homme Treatment for men, and also sculpting treatments for facial contours and around the eyes. Youll also return home with tips and techniques to maximise your beauty routine. Dior Institut can be found at Dior Beauty's The Shoppes at Marina Bay Sands flagship, Robinsons The Heeren, and Tangs at Tang Plaza.
(Related: Dior Makeup's Peter Philips Reveals Why The Brand's New Lipsticks Are Infused With Flower Oil)
Available in Sulwhasoo boutiques at Capitol Building, Ion Orchard, Westgate and the Sulwhasoo Facial Treatment Studio at Tangs, Sulwhasoo offers a suite of facial treatments to address various skin concerns, from the moisturising Essential Treatment to the rejuvenating Timetreasure Renovating Treatment.
A specialised anti-ageing facial for men is also available. Creating a holistic experience for consumers, each treatment begins with a meridian point massage using a fragrance of the customers choice, followed by a foot bath using ginseng peels and red ginseng water. Traditional applicators like jade, amber and white porcelain are also used to enhance the efficacy of the treatments.
(Related: A Holistic Approach To Beauty At The Sulwhasoo Beauty Lounge)
Previously only available at the Ritz-Carlton Spa, fans of La Mer can now indulge in the full pampering experience with the recent opening of La Mers flagship at The Shoppes at Marina Bay Sands, an experiential space complete with a VIP consultation area and a facial suite. On top of the complimentary services available, the flagship also offers a menu of facial services, including the 75-minute Miracle Broth Facial, which harnesses the healing energies of La Mers signature ingredient, the Miracle Broth.
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These Cult Beauty Brands Offer The Most Luxurious Spa Experience at Their Boutiques: Sulwhasoo, La Mer, Dior and More - Singapore Tatler
GSK announces positive headline results in phase 3 study of Benlysta in patients with lupus nephritis | Antibodies | News Channels -…
By daniellenierenberg
DetailsCategory: AntibodiesPublished on Friday, 20 December 2019 13:14Hits: 83
- BLISS-LN achieves primary endpoint and all major secondary endpoints
- On-track for regulatory submission during the first half of 2020
LONDON, UK I December 18, 2019 I GSK today announced positive headline results for intravenous (IV) Benlysta (belimumab) in the largest controlled phase 3 study in active lupus nephritis (LN), an inflammation of the kidneys caused by systemic lupus erythematosus (SLE) which can lead to end-stage kidney disease.
The Efficacy and Safety of Belimumab in Patients with Active Lupus Nephritis (BLISS-LN) study, involving 448 patients, met its primary endpoint demonstrating that a statistically significant greater number of patients achieved Primary Efficacy Renal Response (PERR) over two years when treated with belimumab plus standard therapy compared to placebo plus standard therapy in adults with active LN (43% vs 32%, odds ratio (95% CI) 1.55 (1.04, 2.32), p=0.0311).
Dr Hal Barron, Chief Scientific Officer and President R&D, GSK said: "Lupus nephritis is one of the most common and serious complications of SLE, occurring in up to 60% of adult patients. The results of the BLISS-LN study show that Benlysta could make a clinically meaningful improvement to the lives of these patients who currently have limited treatment options."
Dr Richard Furie,Chief of the Division of Rheumatology and Professor at the Feinstein Institutes atNorthwell Health and Lead Investigator of BLISS-LN said: "My journey with Benlysta began nearly twenty years ago when we performed the very first clinical research trial in lupus patients. To see it culminate in a successful phase 3 lupus nephritis study is a key achievement as the inadequate response of our patients with kidney disease to conventional treatment has long been an area in need of major improvement."
Belimumab also demonstrated statistical significance compared to placebo across all four major secondary endpoints: Complete Renal Response (CRR) after two years (the most stringent measure of renal response), Ordinal Renal Response (ORR) after two years, PERR after one year, and the time to death or renal-related event. In BLISS-LN, safety results for patients treated with belimumab were generally comparable to patients treated with placebo plus standard therapy. The safety results are consistent with the known profile of belimumab.
Benlysta is currently not recommended for use in severe active lupus nephritis anywhere in the world because it has not been previously evaluated in these patients. Based on these positive phase 3 data, GSK plans to progress regulatory submissions in the first half of 2020 to seek an update to the prescribing information.
The full results will be submitted for future presentation at upcoming scientific meetings and in peer-reviewed publications.
About lupus nephritisSystemic lupus erythematosus (SLE), the most common form of lupus, is a chronic, incurable, autoimmune disease associated with a range of symptoms that can fluctuate over time including painful or swollen joints, extreme fatigue, unexplained fever, skin rashes and organ damage. In lupus nephritis (LN), SLE causes kidney inflammation, which can lead to end-stage kidney disease. Despite improvements in both diagnosis and treatment over the last few decades, LN remains an indicator of poor prognosis.1,2 Manifestations of LN include proteinuria, elevations in serum creatinine, and the presence of urinary sediment.
About BLISS-LNBLISS-LN,which enrolled 448 adult patients, was a phase 3, 104-week, randomised, double-blind, placebo-controlled post-approval commitment study to evaluate the efficacy and safety of IV belimumab 10 mg/kg plus standard therapy (mycophenolate mofentil for induction and maintenance, or cyclophosphamide for induction followed by azathioprine for maintenance, plus steroids) compared to placebo plus standard therapy in adult patients with active lupus nephritis. Active lupus nephritis was confirmed by kidney biopsy during screening visit using the 2003 International Society of Nephrology/Renal Pathology Society (ISN/RPS) criteria, and clinically active kidney disease.
The primary endpoint PERR was defined as estimated Glomerular Filtration Rate (eGFR) 60 mL/min/1.73m2 or no decrease in eGFR from pre-flare of > 20%; and urinary protein:creatinine ratio (uPCR) 0.7; and not a treatment failure. The most stringent secondary endpoint CRR was defined as eGFR is no more than 10% below the pre-flare value or within normal range; and uPCR < 0.5; and not a treatment failure. ORR was defined as complete, partial or no response.
About Benlysta (belimumab)Benlysta, a BLyS-specific inhibitor, is a human monoclonal antibody that binds to soluble BLyS. Benlysta does not bind B cells directly. By binding BLyS, Benlysta inhibits the survival of B cells, including autoreactive B cells, and reduces the differentiation of B cells into immunoglobulin-producing plasma cells.
The current US and EU indication for Benlysta are summarised below:
In the US, "Benlysta is indicated for the treatment of patients aged 5 years and older with active, autoantibody-positive, systemic lupus erythematosus (SLE) who are receiving standard therapy. Limitations of Use: The efficacy of Benlysta has not been evaluated in patients with severe active lupus nephritis or severe active central nervous system lupus. Benlysta has not been studied in combination with other biologics or intravenous cyclophosphamide. Use of Benlysta is not recommended in these situations."
Full US prescribing information including Medication Guide is available at: https://www.gsksource.com/pharma/content/dam/GlaxoSmithKline/US/en/Prescribing_Information/Benlysta/pdf/BENLYSTA-PI-MG.PDF
In the EU, "Benlysta is indicated as "add-on therapy in patients aged 5 years and older with active, autoantibody-positive systemic lupus erythematosus (SLE) with a high degree of disease activity (e.g., positive anti-dsDNA and low complement) despite standard therapy."
The Precaution and Warnings for Benlysta includes information that "Benlysta has not been studied in the following adult and paediatric patient groups, and is not recommended: severe active central nervous system lupus; severe active lupus nephritis; HIV; a history of, or current, hepatitis B or C; hypogammaglobulinaenia (IgG < 400mg/dl) or IgA deficiency (IgA < 10 mg/dl); a history of major organ transplant or hematopoietic stem cell/marrow transplant or renal transplant."
The EU Summary of Product Characteristics for Benlysta is available on: http://www.ema.europa.eu
Benlysta is available as an intravenous and a subcutaneous formulation. The Benlysta subcutaneous formulation is not approved for use in children.
GSK's commitment to immunologyGSK is focused on the research and development of medicines for immune-mediated diseases, such as lupus and rheumatoid arthritis, that are responsible for a significant health burden to patients and society. Our world-leading scientists are focusing research on the biology of the immune system with the aim to develop immunological-based medicines that have the potential to alter the course of inflammatory disease. As the only company with a biological treatment approved for adult and paediatric lupus, GSK is leading the way to help patients and their families manage this chronic, inflammatory autoimmune disease. Our aim is to develop transformational medicines that can alter the course of inflammatory disease to help people live their best day, every day.
SOURCE: GlaxoSmithKline
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GSK announces positive headline results in phase 3 study of Benlysta in patients with lupus nephritis | Antibodies | News Channels -...
Is Momo (MOMO) Stock Outpacing Its Computer and Technology Peers This Year? – Nasdaq
By daniellenierenberg
Investors interested in Computer and Technology stocks should always be looking to find the best-performing companies in the group. Momo (MOMO) is a stock that can certainly grab the attention of many investors, but do its recent returns compare favorably to the sector as a whole? One simple way to answer this question is to take a look at the year-to-date performance of MOMO and the rest of the Computer and Technology group's stocks.
Momo is one of 630 companies in the Computer and Technology group. The Computer and Technology group currently sits at #7 within the Zacks Sector Rank. The Zacks Sector Rank considers 16 different sector groups. The average Zacks Rank of the individual stocks within the groups is measured, and the sectors are listed from best to worst.
The Zacks Rank is a proven system that emphasizes earnings estimates and estimate revisions, highlighting a variety of stocks that are displaying the right characteristics to beat the market over the next one to three months. MOMO is currently sporting a Zacks Rank of #1 (Strong Buy).
Over the past 90 days, the Zacks Consensus Estimate for MOMO's full-year earnings has moved 11.33% higher. This signals that analyst sentiment is improving and the stock's earnings outlook is more positive.
According to our latest data, MOMO has moved about 53.94% on a year-to-date basis. In comparison, Computer and Technology companies have returned an average of 32.75%. This shows that Momo is outperforming its peers so far this year.
Breaking things down more, MOMO is a member of the Internet - Software and Services industry, which includes 19 individual companies and currently sits at #74 in the Zacks Industry Rank. On average, this group has gained an average of 34.46% so far this year, meaning that MOMO is performing better in terms of year-to-date returns.
Going forward, investors interested in Computer and Technology stocks should continue to pay close attention to MOMO as it looks to continue its solid performance.
Momo Inc. (MOMO): Free Stock Analysis Report
To read this article on Zacks.com click here.
The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.
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Is Momo (MOMO) Stock Outpacing Its Computer and Technology Peers This Year? - Nasdaq
Human fat cells tweaked to work like heart’s pacemaker: Study – ETHealthworld.com
By daniellenierenberg
Houston: In a first, researchers, including one of Indian-origin, have reprogrammed the human body's fat cells into those similar to the heart's pacemaker cells which control heartbeat by creating rhythmic electrical impulses , an advance that may lead to new therapies for cardiac failure.
The study, published in the Journal of Molecular and Cellular Cardiology, noted that the new pacemaker-like cell may become a useful alternative treatment for heart conduction system disorders, and to bridge the limitations of current treatments such as artificial electronic pacemaker implants.
According to an earlier study, published in the Journal of Geriatric Cardiology, each year more than one million artificial pacemakers are implanted in patients globally.
The device is placed in the chest or abdomen, and uses electrical pulses to prompt the heart to beat normally.
In the current study, researchers, including Suchi Raghunathan from the University of Houston in the US, tweaked unspecialised stem cells to turn them into conducting cells of the heart that could carry electrical current.
The researchers, in an earlier study, had turned the stem cells residing in the human body's fat cells into cardiac progenitor cells.
With the current study, they showed that these cardiac progenitor cells can be programmed to conduct current and keep hearts beating.
They said, its functioning is similar to the heart's natural node of cells called the sinoatrial node (SAN) -- part of the electrical cardiac conduction system (CCS).
The scientists noted that the SAN is the primary pacemaker of the heart, responsible for generating the electric impulse or its 'beat'.
According to the study, the heart's native cardiac pacemaker cells are confined within the SAN -- a small structure comprised of just a few thousand specialized pacemaker cells.
It noted that a failure of the SAN, or a block at any point in the CCS resulted in irregular heartbeats, also called arrhythmias.
"Batteries will die. Just look at your smartphone. This biologic pacemaker is better able to adapt to the body and would not have to be maintained by a physician. It is not a foreign object," said study co-author Bradley McConnell from the University of Houston.
He said the cells would be able to grow with the body, and become much more responsive to what the body is doing.
As part of the study, the scientists infused lab-grown fat cells from the heart with a unique cocktail of three molecules called transcription factors that could induce gene activity.
They also supplied these cells with molecules called plasma membrane channel proteins, which are gates opening up in cells to allow outside chemicals.
Using these molecules, the team could reprogram the heart cells in vitro.
"In our study, we observed that the SHOX2, HCN2, and TBX5 (SHT5) cocktail of transcription factors and channel protein reprogrammed the cells into pacemaker-like cells," McConnell said.
The researchers said the combination of these biomolecules may facilitate the development of cell-based therapies for various cardiac conduction diseases.
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Human fat cells tweaked to work like heart's pacemaker: Study - ETHealthworld.com