Diabetic foot wounds kill millions, but high-tech solutions and teamwork are making a difference – Thehour.com
By daniellenierenberg
(The Conversation is an independent and nonprofit source of news, analysis and commentary from academic experts.)
David G. Armstrong, University of Southern California
(THE CONVERSATION) What if someone told you that theres a disease you could catch where you couldnt feel any symptoms coming on? And that this occurs every 1.2 seconds somewhere in the world?
What if you were stricken with this disease then there would be a 5% chance youd lose a limb within a year and a 50-70% chance youd be dead in five years? What if you were told that this problem cost more than the five most expensive cancers in the U.S. but far less than one one-thousandth of comparative federal and private funding is spent on attacking it?
Ladies and gentlemen, please allow me to introduce you to the humble diabetic foot ulcer. While the problem may strike at the end of the body, far away from the heart or the brain, its effects are far-reaching.
I have spent my career treating and researching the lower extremity complications of diabetes. Based on my research and experience, I believe our society could eliminate immeasurable suffering if we collectively paid more attention to this problem.
How do diabetic foot ulcers occur?
OK, I know this isnt a sexy topic. Foot wounds are ugly. Many people who have them are poor. But bear with me. They are a reality for far too many Americans and people across the globe. The ages of these patients are bimodal, in that there is one population of people who are old and getting older. Conversely, with more and more people being diagnosed with Type 2 diabetes earlier, there is a population that is younger than ever being afflicted with wounds, infections and amputation. Ignoring the problem is an example of ignoring the needs of a silent and vulnerable population.
About 31 million people in the U.S. have diabetes, and about half a billion worldwide.
Diabetic foot ulcers develop because people with diabetes slowly lose the gift of pain. Over many years, people with diabetes lose feeling in their extremities. This occurs first and generally most profoundly in their feet.
Once this occurs, people with diabetes might wear a hole in their foot, just as you or I might wear a hole in a sock or shoe. This hole is called a diabetic foot ulcer.
About half the time, the ulcer will become infected. This increases the risk of further tissue damage and, in the face of frequent vascular disease, high-level amputation. Often all of this occurs with few, if any, symptoms until it is too late.
Solutions and hope
There is also good news. Studies have suggested that high-level amputations seem to decrease when interdisciplinary care is in place, regardless of the country.
Interdisciplinary teams consist of podiatric and vascular surgeons, the so-called Toe and Flow model. The concept is simple; these two specialists, can manage a great deal of the medical, surgical and biomechanical aspects of healing and aftercare.
When we add core physical therapy to this, then the threesome (what we in the field call Toe, Flow and Go) is really quite formidable. For example, our clinics at the University of Southern California and Rancho Los Amigos in Los Angeles have active participation from more than eight specialists ranging from plastic surgery to prosthetics/orthotics, to occupational therapy to nutrition to general practice to infectious disease to diabetology to nurse case management.
Truly, it takes a village to preserve a limb.
Smart boots, high-tech vacuums and sheets of stems cells
It has long been said in wound care that its not what one puts on a wound that heals it, but what one takes off. That maxim is absolutely true in the diabetic foot. Protection of the wound is key.
The gold standard for protecting the wound has been, believe it or not, to put the patient into a special cast. This device works so well because it protects the foot in a process known as offloading, or taking the burden off the foot. By its design, this cast is not easy to remove.
While this has been my personal favorite device to heal these foot wounds, patients dont like it and most doctors dont, either. In fact, fewer than 2% of centers in the country use this as their primary means of offloading. Reasons for this include fear of putting an open wound into a cast (even though the data largely refute this), the time required to apply and remove it and patients being miserable in a hot and heavy device.
Very recently, tech company offshoots have begun to partner with prosthetic/orthotic companies to create next-gen devices that can coax patients into wearing their protective device rather than forcing it upon them. They are using phone calls and a smartwatch.
After focusing on offloading pressure, the next question is what can be done to heal the wound.
Technologies ranging from fancy vacuums, to donated placental tissue, to repurposing blood cells into a dressing to topical oxygen systems have shown recent promise. Active research is being conducted with stem cell sheets consisting of specialized cells seeded on a clear sheet, spread-on skin, and gene therapy.
Remission
As challenging as healing the wound heals, the real challenge is whats next. Following healing, 40% of foot wounds will recur in one year, about two-thirds at three years, and nearly three out of four at five years.
At USC, along with colleagues in the National Health Service in the U.K., we have developed remission clinics designed to extend and promote an active life for this high-risk patient population.
This has also been combined with things like smart insoles, socks and home-based bathmats that can identify wounds before they occur. These technologies will likely initially be subscription-based but may expand beyond that.
Diabetic foot ulcers are common, complex and costly. Theyre sinister in that they come on quietly. Perhaps, though, it is now up to us to alert our own families, communities and leaders to this condition. It is, I believe, only by teaming up that we can stem the tide and preserve not only limbs, but extend lifespan, healthspan and hope.
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This article is republished from The Conversation under a Creative Commons license. Read the original article here: http://theconversation.com/diabetic-foot-wounds-kill-millions-but-high-tech-solutions-and-teamwork-are-making-a-difference-127218.
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Diabetic foot wounds kill millions, but high-tech solutions and teamwork are making a difference - Thehour.com
Discover The Latest Frontier in Anti-aging Medicine and 3 Regenerative Therapies You Should Know… – YourObserver.com
By daniellenierenberg
Regenerative Medicine and Stem Cell technologies... All Hype? Or The Future of Anti-Aging?
We have entered into the rapidly evolving age of Regenerative Medicine, using breakthroughs in cell to cell communication to reset your body's natural ability to heal and repair itself. Advances in Stem Cell technology race forward at an ever increasing rate as people just like you are demanding non-surgical alternatives to the multiple degenerative conditions of aging and inflammation.
If you are asking What is a Stem Cell?
Think of them as simply the master cells of rebuilding the body. They are the building blocks of our genetic code and cellular programming that coordinate healing through bio-signaling to restore our innate capacity of repair & regeneration.
Regenerative Medicine is the vanguard of 21st century health According to Journal: American College of Cardiology
Regenerative Aesthetics is a new field of regenerative medicine that aims to restore and renew the body at the cellular level, dramatically reversing the sands of time and maintaining an aesthetically desirable youthful appearance.
What we really now know is that men and women all over the world want their hair back, they want their sexual organs to work and they want to look and feel their best into their later years.
This brings us to the 3 Regenerative Therapies you should know about.
The first and most widely known of these regenerative therapies is Platelet Rich Plasma or PRP which utilizes your own Blood Plasma and Platelets in a concentrated form to activate the healing cascade. Recruiting your own innate Stem Cells for accelerated wound healing and tissue regeneration.
Known as Liquid Gold, your platelets and plasma have been shown to rejuvenate the
Unfortunately, we are finding clinically that not all PRP is created equally. In fact, some people have a very low concentration of these regenerative growth factors or a high amount of inflammatory cytokines resulting in inconsistencies from person to person, session to session. As PRP continues to get more popular in the mainstream, we find it important to share some of the newer, more optimal Regenerative Technologies that have been emerging.
Which leads us to... The second regenerative therapy you should know about.
Stem Cell Growth factors & Cytokines. Sourced from Bone Marrow Mesenchymal Stem Cells (MSC's) however, they do not contain any actual stem cells or DNA. Growth factors are naturally occurring proteins in your body that regulate cellular growth, healing, proliferation and differentiation under controlled conditions and play a role in cellular communication. . They are master bio-signals acting as command and control over your body's natural healing processes and modulation of inflammation.
It has been shown that cells in aging skin generate less growth factors than cells in youthful skin. For example, by the time you are 50, on average 4% of these regenerative bone marrow MSCs are in circulation compared to what you had when you were in your teens. Hence, we age because we damage faster than we repair in our later years.
By simply adding concentrated MSC Growth factors & Cytokines to our Regenerative Therapies we can consistently improve hair loss, skin rejuvenation, collagen growth, sexual organ function, and more. We know for a fact that daily use of skin care products containing stable growth factors and cytokines help reduce the appearance of fine lines & wrinkles and improve skin tone & texture.
Lastly, and most importantly is the latest frontier and the 3rd regenerative therapy you should know about.
The Future of Regenerative Medicine... Known as Stem Cell Exosomes. Science is showing us that the optimal way to provide true stem cell therapy is to directly provide the cell bio-signals in high concentrations. After all, the signaling is what we really require to regenerate a normal healthy physiology.
Exosomes are regarded as the purest form of cellular therapy available today, providing a safe and anti-inflammatory environment for healing and repairing.
The AABB recently reported that up to one in three people in the U.S. could benefit from regenerative medicine.
At Rejuvenate 528 Regenerative Aesthetics Medical Spa, we can include exosomes for enhanced wellness to the majority of our Regenerative Aesthetic Services. This can benefit your overall health and vitality as this is reversing challenges of Inflammation!
Beauty radiates and vibrates at different frequencies in everyone I see! We love to uplift and Rejuvenate both the inner vitality and the outer Radiance of all of our patients and clients. They come for the personal attention and integrative approach using regenerative medicine modalities with ancient technologies. PA Sheri Suiter
Tap into your own healing potential with these types of bio-hacking technologies to enhance your regenerative potential and get the results you truly desire. Live longer, stronger and younger.
Sheri Suiter CLT, MS, PA-C, Founder of Rejuvenate 528 Regenerative Aesthetics Medical Spa in Sarasota, FLRejuvenate528.com
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Discover The Latest Frontier in Anti-aging Medicine and 3 Regenerative Therapies You Should Know... - YourObserver.com
Scottish baby spent first Christmas in hospital after she was diagnosed with two types of cancer – Scotland on Sunday
By daniellenierenberg
The parents of a baby girl were left heartbroken after she was diagnosed with two types of blood cancer.
Amelia Topa, who turns two today, is in remission from leukaemia after having a stem cell transplant using a newborn babys umbilical cord blood which was specially flown in from America.
Now doing well, Amelia has received a Cancer Research UK for Children & Young People Star Award, supported by TK Maxx, in recognition of the remarkable courage she demonstrated since being diagnosed with cancer.
Amelias parents, Kerri Paton, 23, and Igor Topa, 24, of Turriff, Aberdeenshire are hugely proud of their little girl.
Kerri said: Anyone who meets Amelia would agree that shes a star.
READ MORE - Scottish actor Gray O'Brien reveals he has been treated for stage four cancer
Being told your child has cancer is the worst sentence any parent could ever hear. I felt mad at first that someone so tiny should have to go through this horrible disease. But Amelia has been a little fighter from the day she was born. I have felt amazed by her strength and lucky to have good support from friends, family and hospital staff.
We will forever be grateful to a family in America well probably never get a chance to meet. The stem cells from America looked just like a small bag of blood but they had the power to make Amelia well again.
Around 140 children are diagnosed with cancer in Scotland every year.
Mum Kerri recalls vividly the moment their lives were turned upside down when only hours after Amelia was born on November 27 2017 medics at Dr Grays Hospital in Elgin explained that raised purple spots across Amelias body could be a sign of something seriously wrong.
Following tests, on December 14 2017 Amelia was diagnosed with leukaemia. Unusually, doctors diagnosed Amelia a mix of two types, acute lymphoblastic leukaemia and acute myeloid leukaemia. The family were transferred to the Queen Elizabeth University Hospital in Glasgow the following day.
READ MORE - Gary Rhodes: Celebrity chef dies suddenly aged 59
Kerri said: It didnt hit me properly until I walked out of the room and then I started crying, a lot.
Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of. We were looked after by the hugely experienced Professor Brenda Gibson. It helped to know we had the best oncology doctor on our side.
Amelia spent her first Christmas in hospital as the first of four rounds of chemotherapy treatment started. By spring, the family were advised that Amelias best chance of survival was a bone marrow transplant using stem cells. A match was found and the transplant went ahead on June 28 2018. The family were told was that the stem cells had been donated from a man aged between 16 and 30.
Amelia recovered well and tests showed that the transplant had worked. By autumn last year Amelia was well enough to go home and the family slowly settled back in to life in Aberdeenshire. And after a difficult year, it was a boost when Kerri discovered she was pregnant again.
Oscar was the first baby to be born in the new maternity unit in Aberdeen when he arrived on October 30 2018. Now Amelia was big sister to Oscar, Kerri dared to hope they could settle in to an ordinary family life.
But tragedy struck again. Kerris mum, Angela McNabb who had stood by the family every step of the way suddenly died from heart failure aged 48- just the day before Amelias birthday.
Kerri said: My mum was my best friend, she was everything to me.
Mum absolutely loved Amelia and was so close to her. My major source of support was gone and I hadnt even had the chance to say goodbye. I couldnt believe it. It was so unfair. Last Christmas was heartbreaking.
And it was a hammerblow on February 11 this year when tests showed that Amelias cancer had come back. Doctors were uncertain at first whether anything else could be done but they suggested a second stem cell transplant, this time using stem cells from umbilical cord blood.
Amelia had intense chemotherapy in an isolation room before she was ready for the transplant at the Queen Elizabeth University Hospital in Glasgow. The transplant went ahead on June 28 this year- exactly a year after the first transplant.
Kerri said: They had to fly the umbilical cord blood over from America.
Doctors explained to us that this was the best option to keep the leukaemia away. Amelia soared through the transplant and shes doing really well now. Were finally looking forward to a happy Christmas as a family and I couldnt be prouder. I hope Amelias story will help other families going through cancer. There is a light at the end of the tunnel.
The Cancer Research UK for Children & Young People Star Awards, supported by TK Maxx, are open to all under-18s who currently have cancer or have been treated for the disease in the last five years. There is no judging panel because every child diagnosed with cancer deserves special recognition. Everyone nominated receives a trophy, 50 TK Maxx gift card, t-shirt and a certificate signed by a host of famous faces, including Nanny McPhee and Last Christmas star Dame Emma Thompson, This Mornings Dr Ranj and childrens favourite entertainer Mister Maker. Their siblings also receive a certificate.
Now they are encouraging families across Scotland to nominate their stars for the honour in the run up to Christmas.
Lisa Adams, spokeswoman for Cancer Research UK for Children & Young People in Scotland, said: Our Star Awards, supported by TK Maxx, shine an important light on children and young people with cancer.
We know that a cancer diagnosis is devastating at any age, but that it can be particularly difficult for a child or young person and their families. Thats why were calling on families across Scotland to nominate inspirational youngsters for an award so that we can recognise their incredible courage.
The Cancer Research UK for Children & Young People Star Awards are supported by TK Maxx, the biggest corporate supporter of the charitys research in to childrens and young peoples cancers. Since the partnership began, the retailer has raised over 34 million for research in to these cancers to help more children and young people survive cancer.
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Scottish baby spent first Christmas in hospital after she was diagnosed with two types of cancer - Scotland on Sunday
The Best Thanksgiving – Yahoo Finance
By daniellenierenberg
Family Celebrates Daughters Cure From Deadly Disease and the Love of Two Moms in Two Countries Who Made Cure Possible
Bethesda, Maryland, Nov. 26, 2019 (GLOBE NEWSWIRE) -- One Texas family has lots to be thankful for this Thanksgiving. Their daughter, now 13, is doing well after undergoing a bone marrow transplantthe only chance for a cure for her rare and deadly disease. But Emis story is not only a story about the triumph of medical research that is making her cure possibleits also a story about extraordinary parental love and sacrifices by her birth mom and her adoptive family that are giving this very ill girl the best chance at life. Emi's birth mom donated her stem cells to make the lifesaving transplant possible.
We are most thankful for an answer to years of prayers, Emis adoptive mom says. Emi got a new start at life, a rebirth day. Every holiday this year will be like the first. Were so grateful to the doctors, nurses and The Childrens Inn.
Emi and her family will be celebrating Thanksgiving at The Childrens Inn at NIH, a nonprofit hospitality house that provides free lodging and a wide variety of support services to families of children with rare and serious diseases whose best chance for a treatment is a clinical research study at the National Institutes of Health. Emi and her mom have spent several months at The Childrens Inn so far and bonded with other families. On Thanksgiving Day, families staying at The Childrens Inn who cannot go home for the holiday will be served a traditional Thanksgiving meal prepared by a group of dedicated volunteers.
It took two moms who love this little nugget to fight for her right to life, Emis adoptive mom says. We finally are getting to see that beautiful part of the story that we always knew was there.
Read Emis full story.
See photos of Emi and her family.
About The Childrens Inn at NIH:
The Childrens Inn at NIH provides free lodging and a wide range of supportive services to more than 1,500 children and their families every year whose best chance for a treatment is a clinical trial at the National Institutes of Health. Opened in 1990 and located across from the NIH Clinical Center, the worlds largest hospital dedicated entirely to medical research, The Childrens Inn has welcomed children from all 50 states and 94 countries. Children staying at The Childrens Inn are making important contributions to rare disease and cancer research, including the successful treatment of childhood leukemia, as well as treatments for HIV/AIDS, childhood asthma, bone and growth diseases, childhood onset schizophrenia and other mental health issues, neurofibromatosis type 1 and a wide variety of genetic and rare diseases. For more information, visit http://www.childrensinn.org. To support The Childrens Inn, make a donation at http://www.childrensinn.org/donate.
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Attachments
Sonja LueckeThe Children's Inn at NIH9013401975sonja.luecke@nih.gov
Mysba RegisThe Children's Inn at NIH240-274-2101mysba.regis@nih.gov
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The Best Thanksgiving - Yahoo Finance
Upstate SC toddler survives rare cancer and the risky procedure used to treat it – Greenville News
By daniellenierenberg
Outcome means a special Thanksgiving
Hailie and Treylin Hyman saw the bruising on their baby girls leg as a sign that the active 1-year-old was learning to walk.
But as a blood test would later reveal, little Maci was actually suffering from an extremely rare blood cancer that threatened her life without a risky treatment - atreatmentalmost as dangerous as the disease.
In the beginning, it was very scary, Hailie Hyman told The Greenville News.
I couldnt think of anything but the bad things, she confessed. It was all about the statistics. And the statistics arent good.
Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)
Terrifying months followed the diagnosis, punctuated by one critical complication after another, leaving the Boiling Springs couple to wonder if Maci would survive.
Somehow, though, the blue-eyed toddler pulled through.And now her family is looking forward to a special Thanksgiving with much to be grateful for.
The Hymans journey began last February atMacis 1-year-old well-child checkup.
We had no idea anything was wrong, her mom said.But they did a routine (blood test) and a couple of hours later, we got a call saying her platelets were very low.
The Hymans were referred to a hematologist who found other abnormalities in Macis blood and scheduled a bone marrow biopsy to investigate further.
Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)
During the procedure, the child suffered an aneurysm in an artery and went into cardiac arrest. The team performed CPR on her for 20 minutes before she was stabilized, her mom said.
Later, in the pediatric intensive care unit, she suffered internal bleeding, too.
It was really hard, she said. There were many nights that I would just pray and pray and pray.
Initially believing Maci had leukemia, doctors subsequently determined she had myelodysplastic syndrome, or MDS.
The condition occurs when abnormal cells in the bone marrow leave the patient unable to make enough blood, according to the American Cancer Society.
Its rare, afflicting as few 10,000 Americans a year, though the actual number is unknown.
Maci Hyman, 1, interacts with hospital staff before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)
In children, its rarer still. Most people arediagnosed in their 70s.
We were told that just four out of 1 million children get it every year, Hailie Hyman said.
That made the diagnosis elusive at first, said Dr. Nichole Bryant, a pediatric hematologist-oncologist with Prisma Health-Upstate, formerly Greenville Health System.
Shes the only one Ive seen in my career, she said.
Maci had to have regular blood transfusions, antibiotics and other medications to fight the MDS, Bryant said. But the only hope for a cure was a stem cell transplant at the Medical University of South Carolina in Charleston.
When they said that was the only treatment plan for MDS, I of course went to Google, Hailie Hyman said. I read about transplant patients and ...all the complications. It was terrifying. But no matter how many bad things I saw, we had to do it. There is no other option.
The transplantis extremely risky.
Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)
First, high doses of chemotherapy are given to destroy the diseased bone marrow, leaving the patient without an immune system, so fighting infections becomes a challenge. Then healthy donor marrow is infused.
Its also fraught with potentially life-threatening complications, including graft vs. host disease, which occurs when immune cells from the donor attack the patients body, Bryant said. Other complications include permanent kidney damage and gastrointestinal problems.
They have to go to hell and back, she said. But its the only option for long-term survival.
Maci had a really rough start, suffering lots and lots and lots of complications, Bryant said.
Her kidneys failed, so she wound up on dialysis. When she couldnt breathe on her own, she was put on a ventilator. And because she couldnt eat, she had to be tube fed.
Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)
She had blistering sores in her mouth and throughout her GI tract, her mom said. Because her liver wasnt functioning properly, her abdomen filled up with fluid that had to be drained. She was bleeding so profusely in her lungs that one of them collapsed.
Maci, who was sedated through much of it, was put on full life support, she said.
That night we almost lost her, her mom said. We were in the hallway crying our eyes out. We didnt know what do to or think. It was pretty scary for a while.
Somehow, Maci made it.
There were so many times during her first months that it seemed like she would not survive, Bryant said. So the fact that she is here ... is really a miracle.
Macis family found an unrelated donor through the National Marrow Donor Program, enlisting hundreds of other people to join the registry in the process, Bryant said.
Nichole Bryant, M.D.(Photo: Provided)
It was an important part of their journey that maybe didnt directly benefit Maci, she said. But if everybody did that, we wouldnt have difficulty finding a donor for anybody.
Doctors have no explanation for why Maci got MDS. She didnt carry the genetic mutation for it and there is no family history.
She is a rare child - and not in a good way, her mom said, adding,Youve got to laugh sometimes or youre going to cry.
Maci was admitted to MUSC on June 2 and released on Oct. 14.
The Hymans, both 22, spent the entire time in Charlestonwhile Hailies mom cared for their older daughter, Athena, now 2.
Treylins employer held his welding job open for him. And other friends and family members did what they could to help.
We had many, many people very generously donate to us to cover expenses at home and living expenses where we were, Hailie Hyman said.
We are thankful for everyone who helped us through it the cards, the gifts, the donations. Every single cent is greatly appreciated.
They still need to travel to Charleston once a week to see the transplant doctor. In between, Maci is seen in Greenville.
She's doing well, but recovery from a transplant can take months to years, Bryant said.
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Her kidneys are functioning again so she was able to come off dialysis. But she still must take many medications, including anti-rejection drugs that suppress her immune system and leaveher at risk for infection. And she still must be tube fed.
She is miles ahead of where she was two months ago, Bryant said. But she still has a long way to go. Its a long, long road.
Macis mom says she can be up and playing one day and flopped over on the couch another. She still experiences a lot of nausea and vomiting, but is doing well compared to where she was.
Hailie Hyman pulls her daughter Maci, 1, in a wagon in the hallway before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)
So as the nation pauses to give thanks this Thanksgiving, she says the family will be countingtheir many blessings family andfriends, Gods mercy, andthe doctors and nurses who saved Macis life.
She has battled a lot and overcome a lot, she said. I have no doubt she will be able to get through.
Want to know more about becoming a marrow donor? Go to bethematch.org.
Read or Share this story: https://www.greenvilleonline.com/story/news/health/2019/11/27/upstate-sc-toddler-survives-rare-cancer-and-risky-procedure-treat/4158606002/
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Upstate SC toddler survives rare cancer and the risky procedure used to treat it - Greenville News
AVITA Medical Teams With Gates Center to Advance Therapeutic Skin Restoration – CU Anschutz Today
By daniellenierenberg
AVITA Medical (ASX: AVH, NASDAQ: RCEL), a regenerative medicine company with a technology platform positioned to address unmet medical needs in therapeutic skin restoration, and scientists at the Gates Center for Regenerative Medicine at the University of Colorado School of Medicine have announced a preclinical research collaboration to establish proof-of-concept and explore further development of a spray-on treatment of genetically modified cells for patients with epidermolysis bullosa (EB), with potential applicability to other genetic skin disorders.
The partnership will pair AVITA Medicals patented and proprietary Spray-On Skin Cells technology and expertise with the Gates Centers innovative, patent pending combined reprogramming and gene editing technology to allow cells to function properly. Under the terms of the Sponsored Research Agreement (SRA), AVITA Medical retains the option to exclusively license technologies emerging from the partnership for further development and commercialization. The Gates Center team is further supported by the EB Research Partnership in New York, the Los Angeles-based EB Medical Research Foundation, the London-based Cure EB Charity and government grants, in a collaborative effort to rapidly develop and translate this technology to the clinic for meaningful impact on patient lives.
The Gates Center is a leader in developing therapeutic approaches for genetic skin diseases. Researchers at the Gates Center have developed a powerful new approach for treating genetic skin disorders and improving the lives of patients with epidermolysis bullosa, said Mike Perry, PhD, chief executive officer of AVITA Medical and adjunct professor at the Gates Center for Regenerative Medicine. We look forward to collaborating with the team at the Gates Center on the expanded use of our technology. This agreement marks an important milestone in AVITAs mission to harness the potential of regenerative medicine to address unmet medical needs across a broad range of dermatological indications, including genetic disorders of the skin.
Epidermolysis bullosa is a group of rare and incurable skin disorders caused by mutations in genes encoding structural proteins resulting in skin fragility and blistering, leading to chronic wounds and, in some sub-types, an increased risk of squamous cell carcinoma or death. There are no approved curative therapies, and current treatment is palliative - focused primarily on pain and nutritional management, itching relief, wound care, and bandaging.
Its very exciting to partner with AVITA Medical to help advance our epidermolysis bullosa program, said Director of the Gates Center for Regenerative Medicine Dennis Roop, PhD. Were looking forward to exploring a novel approach to delivering gene-edited skin cells to patients that addresses current treatment challenges.
We believe that Spray-On Skin Cells technology combined with our genetically corrected cells has the potential to be game changing in the treatment of this disease. This combination could reduce time to treatment, lower manufacturing complexity, reduce costs and improve patient outcomes, said Ganna Bilousova, PhD, assistant professor of dermatology, who is a co-principal investigator on this research program.
ABOUT THE CHARLES C. GATES CENTER FOR REGENERATIVE MEDICINE
The Charles C. Gates Center for Regenerative Medicine was established in 2006 with a gift in memory of Denver industrialist and philanthropist, Charles C. Gates, who was captivated by the hope and benefit stem cell research promised for so many people in the world. The Gates Center aspires to honor what he envisionedby doing everything possible to support the collaboration between basic scientific researchers and clinical faculty to transition scientific breakthroughs into clinical practice as quickly as possible.
Led by Founding Director Dennis Roop, PhD, the Gates Center is located at the University of Colorados Anschutz Medical Campus, the largest new biomedical and clinical campus in the United States. Operating as the only comprehensive Stem Cell Center within a 500-mile radius, the Gates Center shares its services and resources with an ever-enlarging membership of researchers and clinicians at the Anschutz Medical Campus, which includes University of Colorado Hospital, Childrens Hospital Colorado and the Veterans Administration Medical Center, as well as the Boulder campus, Colorado State University, the Colorado School of Mines, and business startups. This collaboration is designed to draw on the widest possible array of scientific exploration relevant to stem cell technology focused on the delivery of innovative therapies in Colorado and beyond.
ABOUT THE UNIVERSITY OF COLORADO SCHOOL OF MEDICINE
Faculty at the University of Colorado School of Medicine work to advance science and improve care. These faculty members include physicians, educators and scientists at University of Colorado Hospital, Childrens Hospital Colorado, Denver Health, National Jewish Health, and the Denver Veterans Affairs Medical Center. The school is located on the CU Anschutz Medical Campus, one of four campuses in the University of Colorado system. To learn more about the medical schools care, education, research and community engagement, visit its web site.
ABOUT AVITA MEDICAL LIMITED
AVITA Medical is a regenerative medicine company with a technology platform positioned to address unmet medical needs in burns, chronic wounds, and aesthetics indications. AVITA Medicals patented and proprietary collection and application technology provides innovative treatment solutions derived from the regenerative properties of a patients own skin. The medical devices work by preparing a REGENERATIVE EPIDERMAL SUSPENSION (RES), an autologous suspension comprised of the patients skin cells necessary to regenerate natural healthy epidermis. This autologous suspension is then sprayed onto the areas of the patient requiring treatment.
AVITA Medicals first U.S. product, the RECELL System, was approved by the U.S. Food and Drug Administration (FDA) in September 2018. The RECELL System is indicated for use in the treatment of acute thermal burns in patients 18 years and older. The RECELL System is used to prepare Spray-On Skin Cells using a small amount of a patients own skin, providing a new way to treat severe burns, while significantly reducing the amount of donor skin required. The RECELL System is designed to be used at the point of care alone or in combination with autografts depending on the depth of the burn injury. Compelling data from randomized, controlled clinical trials conducted at major U.S. Burn Centers and real-world use in more than 8,000 patients globally, reinforce that the RECELL System is a significant advancement over the current standard of care for burn patients and offers benefits in clinical outcomes and cost savings. Healthcare professionals should read the INSTRUCTIONS FOR USE - RECELL Autologous Cell Harvesting Device (https://recellsystem.com/) for a full description of indications for use and important safety information including contraindications, warnings and precautions.
In international markets, our products are marketed under the RECELL System brand to promote skin healing in a wide range of applications including burns, chronic wounds and aesthetics. The RECELL System is TGA-registered in Australia and received CE-mark approval in Europe.
To learn more, visit http://www.avitamedical.com.
Photo at top: From left, Igor Kogut, PhD, Ganna Bilousova, PhD, and Dennis Roop, PhD.
Guest contributor: Gates Center for Regenerative Medicine/ASX
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AVITA Medical Teams With Gates Center to Advance Therapeutic Skin Restoration - CU Anschutz Today
Seven Products Our Beauty Editor Used to the Last Drop – Sarasota
By daniellenierenberg
When it comes to our beauty and skincare products, it's easy for years to pass without an updateor even an upgrade. But because advances in beauty technology are happening at the speed of light, its always important to pause and reassesswhat we're usingfrom dog shampoo to stem-cell skincare. SoI've rounded up the best of the latest trends that have earned a place inmymakeup bag.
Its no secret that Ima major fan of Kjaer Weisthere is something utterly fresh and clean about everything for the face and body, and its as organic as it can get. Enter the latest additions to the line: the cleanser ($95) and toner ($85, both pictured above). Not only does the soft, gel-like cleanser effectively remove all makeup, but its also calming. Follow it with a quick spritz of the toner and you have hydrated and re-balanced skin. Plus, the scents aredreamy.If I could accurately describe them, I would. But I was in Bluemercury downtown recently they have sample bottles to give it a go yourself.Bluemercury, 1500 Main Street, Sarasota. (941) 365-0020
Two things on my must-do-better list: Sunscreen and preventing this neck from aging. Addressing the first, Alastin Skincares HydraTint Pro Mineral Sunscreen SPF 36 ($55) is a revelation. Not only is it lightweight, with broad-spectrum UVA/UVB sun protection, it also protects against environmental pollution and it has a universal tint that enhances most skin tones. Its the first thing Iput on in the morning before taking the dogs for a walk; I love the just-right tinted coverage.
Second, that neck thing. As much as I prefer organic and natural skincarewhen possible, I tend to lean on science for combatting aging. Enter: Nectifirm Advanced ($133). Its next-gen technology based on the ecosystem of the skins microbiome, plus eight peptides that helps skin appear firmer and lifted while lessening the appearance of lines and wrinkles. Not to mention that those in the know at Sarasota Facial Aesthetics rave about the results. Get both products atSarasota Facial Aesthetics, 1445 South Osprey Ave., Suite 2, (941) 955-8384.
I was of the mind that a razor is a razoris a razor. Well, thats changed since theFlamingo razor($9.99) came on my radar. The team raised the bar on shaving after spending years talking to women (what a concept!) who shared the nuances of their personal care rituals and how typical razors fell short. Use this once and it will be clear that they did not overlook those edges of our bodies that need extra attention.Target, 101 N. Cattlemen Road, (941) 360-7520
Speaking of: here's another kind of sunscreen, this time for the eye area. Who knew? I recently discovered Colorescience Total Eye3-in-1 Renewal Therapy SPF 35 ($74)they say it visibly improves the appearance of dark circles, puffiness, fine lines and wrinkles, while protecting the delicate eye area against photoaging with 100 percent SPF 35 mineral sunscreen. I say its great coverage, and if it comes with all of those benefits then...yay!L. Spa, 556 Pineapple Ave., (941) 906-1358
Brace yourself (and maybe your credit card) because Augustinus Baders The Cream ($265) is right there at the cutting edge for stem cell skincare. Get this: the stem cells found in skin lie dormant, awaiting an activation signal to repair the damage inflicted by life and environmental factors.The patented technology TFC8Bader's proprietary "Trigger Factor Complex"is comprised of natural amino acids, high-grade vitamins and synthesized molecules that are found naturally in the skin. Its a repairing force in an ultra-lightweight cream that guides key nutrients and powerful natural ingredients to the skin cells, creating an optimal environment for the body's innate processes of repair and renewal.Thats a lot, but all I know is that I can see the results after a lotta life has happened to my skin. Its crazy good, and I guess for the price it should be. Saks Fifth Avenue, 120 University Town Center Drive, Sarasota. (941) 364-5300
Lastly, this one is for the love of our fur kids, especially those with sensitive skin. The Malin + Goetz Dog Shampoo ($28) is infused with natural botanical amino acids to gently cleanse fur and skin without drying, stripping or irritating. And I can attest that fur dries soft and oh-so-shiny. Malin + Goetz, malinandgoetz.com
Excerpt from:
Seven Products Our Beauty Editor Used to the Last Drop - Sarasota
AVITA Medical and the Gates Center for Regenerative Medicine at the University of Colorado Anschutz Medical Campus Enter into Collaboration to Explore…
By daniellenierenberg
Nov. 24, 2019 23:45 UTC
VALENCIA, Calif., MELBOURNE, Australia & AURORA, Colo.--(BUSINESS WIRE)-- AVITA Medical (ASX: AVH, NASDAQ: RCEL), a regenerative medicine company with a technology platform positioned to address unmet medical needs in therapeutic skin restoration, and scientists at the Gates Center for Regenerative Medicine at the University of Colorado School of Medicine announced today a preclinical research collaboration to establish proof-of-concept and explore further development of a spray-on treatment of genetically modified cells for patients with epidermolysis bullosa (EB), with potential applicability to other genetic skin disorders.
The partnership will pair AVITA Medicals patented and proprietary Spray-On Skin Cells technology and expertise with the Gates Centers innovative, patent-pending combined reprogramming and gene-editing technology to allow cells to function properly. Under the terms of the Sponsored Research Agreement (SRA), AVITA Medical retains the option to exclusively license technologies emerging from the partnership for further development and commercialization. The Gates Center team is further supported by the EB Research Partnership in New York, the Los Angeles-based EB Medical Research Foundation, the London-based Cure EB Charity, and government grants in a collaborative effort to rapidly develop and translate this technology to the clinic for meaningful impact on patient lives.
The Gates Center is a leader in developing therapeutic approaches for genetic skin diseases. Researchers at the Gates Center have developed a powerful new approach for treating genetic skin disorders and improving the lives of patients with epidermolysis bullosa, said Dr. Mike Perry, Chief Executive Officer of AVITA Medical and adjunct professor at the Gates Center for Regenerative Medicine. We look forward to collaborating with the team at the Gates Center on the expanded use of our technology. This agreement marks an important milestone in AVITAs mission to harness the potential of regenerative medicine to address unmet medical needs across a broad range of dermatological indications, including genetic disorders of the skin.
Epidermolysis bullosa is a group of rare and incurable skin disorders caused by mutations in genes encoding structural proteins resulting in skin fragility and blistering, leading to chronic wounds and, in some sub-types, an increased risk of squamous cell carcinoma or death. There are no approved curative therapies, and current treatment is palliativefocused primarily on pain and nutritional management, itching relief, wound care, and bandaging.
Its very exciting to partner with AVITA Medical to help advance our epidermolysis bullosa program, said Director of the Gates Center for Regenerative Medicine Dr. Dennis Roop. Were looking forward to exploring a novel approach to delivering gene-edited skin cells to patients that addresses current treatment challenges.
We believe that Spray-On Skin Cells technology combined with our genetically corrected cells has the potential to be game changing in the treatment of this disease. This combination could reduce time to treatment, lower manufacturing complexity, reduce costs, and improve patient outcomes, said Dr. Ganna Bilousova, assistant professor of dermatology, who is a co-principal investigator on this research program.
ABOUT THE CHARLES C. GATES CENTER FOR REGENERATIVE MEDICINE
The Charles C. Gates Center for Regenerative Medicine was established in 2006 with a gift in memory of Denver industrialist and philanthropist Charles C. Gates, who was captivated by the hope and benefit stem cell research promised for so many people in the world. The Gates Center aspires to honor what he envisionedby doing everything possible to support the collaboration between basic scientific researchers and clinical faculty to transition scientific breakthroughs into clinical practice as quickly as possible.
Led by Founding Director Dennis Roop, Ph.D., the Gates Center is located at the University of Colorados Anschutz Medical Campus, the largest new biomedical and clinical campus in the United States. Operating as the only comprehensive Stem Cell Center within a 500-mile radius, the Gates Center shares its services and resources with an ever-enlarging membership of researchers and clinicians at the Anschutz Medical Campus, which includes University of Colorado Hospital, Childrens Hospital Colorado, and the Veterans Administration Medical Center, as well as the Boulder campus, Colorado State University, the Colorado School of Mines, and business startups. This collaboration is designed to draw on the widest possible array of scientific exploration relevant to stem cell technology focused on the delivery of innovative therapies in Colorado and beyond.
ABOUT THE UNIVERSITY OF COLORADO SCHOOL OF MEDICINE
Faculty at the University of Colorado School of Medicine work to advance science and improve care. These faculty members include physicians, educators, and scientists at University of Colorado Hospital, Childrens Hospital Colorado, Denver Health, National Jewish Health, and the Denver Veterans Affairs Medical Center. The school is located on the Anschutz Medical Campus, one of four campuses in the University of Colorado system. To learn more about the medical schools care, education, research, and community engagement, visit its web site.
ABOUT AVITA MEDICAL LIMITED
AVITA Medical is a regenerative medicine company with a technology platform positioned to address unmet medical needs in burns, chronic wounds, and aesthetics indications. AVITA Medicals patented and proprietary collection and application technology provides innovative treatment solutions derived from the regenerative properties of a patients own skin. The medical devices work by preparing a REGENERATIVE EPIDERMAL SUSPENSION (RES), an autologous suspension comprised of the patients skin cells necessary to regenerate natural healthy epidermis. This autologous suspension is then sprayed onto the areas of the patient requiring treatment.
AVITA Medicals first U.S. product, the RECELL System, was approved by the U.S. Food and Drug Administration (FDA) in September 2018. The RECELL System is indicated for use in the treatment of acute thermal burns in patients 18 years and older. The RECELL System is used to prepare Spray-On Skin Cells using a small amount of a patients own skin, providing a new way to treat severe burns, while significantly reducing the amount of donor skin required. The RECELL System is designed to be used at the point of care alone or in combination with autografts depending on the depth of the burn injury. Compelling data from randomized, controlled clinical trials conducted at major U.S. Burn Centers and real-world use in more than 8,000 patients globally, reinforce that the RECELL System is a significant advancement over the current standard of care for burn patients and offers benefits in clinical outcomes and cost savings. Healthcare professionals should read the INSTRUCTIONS FOR USE - RECELL Autologous Cell Harvesting Device (https://recellsystem.com/) for a full description of indications for use and important safety information, including contraindications, warnings, and precautions.
In international markets, our products are marketed under the RECELL System brand to promote skin healing in a wide range of applications, including burns, chronic wounds, and aesthetics. The RECELL System is TGA-registered in Australia and received CE-mark approval in Europe.
To learn more, visit http://www.avitamedical.com.
CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
This letter includes forward-looking statements. These forward-looking statements generally can be identified by the use of words such as anticipate, expect, intend, could, may, will, believe, estimate, look forward, forecast, goal, target, project, continue, outlook, guidance, future, other words of similar meaning and the use of future dates. Forward-looking statements in this letter include, but are not limited to, statements concerning, among other things, our ongoing clinical trials and product development activities, regulatory approval of our products, the potential for future growth in our business, and our ability to achieve our key strategic, operational and financial goal. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Each forward- looking statement contained in this letter is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others, the timing of regulatory approvals of our products; physician acceptance, endorsement, and use of our products; failure to achieve the anticipated benefits from approval of our products; the effect of regulatory actions; product liability claims; risks associated with international operations and expansion; and other business effects, including the effects of industry, economic or political conditions outside of the companys control. Investors should not place considerable reliance on the forward-looking statements contained in this letter. Investors are encouraged to read our publicly available filings for a discussion of these and other risks and uncertainties. The forward-looking statements in this letter speak only as of the date of this release, and we undertake no obligation to update or revise any of these statements.
View source version on businesswire.com: https://www.businesswire.com/news/home/20191124005098/en/
Vampire facials and other medical witchcraft you can buy to stay youthful today – Citizen
By daniellenierenberg
You can maintain your youthful appearance and be less likely to have more invasive procedures later on in life if you put it effort to maintain the appearance. The same with skincare, if you put more effort now, with daily sunscreen, look after the skin and rejuvenate the skin, the likelihood of you having wrinkly sun-damaged skin later on is obviously much less.
This is the view of Dr Alexandra Grubnik, a plastic and reconstructive surgeon from Nip Tuck at the Netcare Milpark Hospital and Netcare Rosebank Hospital.
While surgical facial procedures are still being requested by South Africans, Dr Grubnik says there has been a rise in non-surgical procedures, with botox being one of the commonest procedures.
All the non-surgical procedures are done in rooms and theres absolutely no downtime. This is why theyre gaining popularity worldwide. There are people who say that in 20 years we will be doing no surgical operations.
A botox procedure involves injecting a serum that weakens the muscles to avoid getting wrinkles, while some use it to get rid of frown lines.
There is a very good twin study identical twins who participated in the study. One of them had regular botox injections every three to four months and the other just had it once upfront and did not have any for 10 years. The difference is absolutely remarkable. The other one without botox looks 20 years older than their sister, says Dr Grubnik.
This is sometimes confused with a filler, which is done to restore volume to the face and make it look more youthful.
As you age there is some resorption on the bone in the face, because theres bone loss and the soft tissues hang. When these people maintain themselves with filler, when they get to their 60s they may not need a facelift because they didnt have that droopiness that the previous generation would have had.
A filler injection (per millimetre) can cost you up to R3,000 each, while botox (per unit) is around R60.
A facelift, also common among South African women, is a procedure that involves removing excess facial skin from the lower half of the face including the chin and neck and tightening loose skin in different areas in the face.
According to Dr Grubnik, this is a big operation and requires recovery time.
There will be bruising, swelling in the face.
You can expect to pay around R95,000 for a facelift.
Also read: They do it for sexual satisfaction, says surgeon on rise in vaginal rejuvenation
Another common facial procedure in South Africa is blepharoplasty (eyelid surgery). This involves taking out excess skin in the upper and lower eyelid to remove bags in the eyelids.
Model and businesswoman Kim Kardashian shocked her social media followers a few years ago after telling them she regularly got vampire facials to keep her face looking younger.
The procedure has gained popularity among women, and Dr Grubnik says its because the procedure actually works.
A vampire facial involves taking blood from parts of your body (apart from the face), and spinning it to separate the red blood cells and the plasma.
In the plasma the platelets are in the blood. Its called platelet red plasma and this platelet red plasma is a stem cell. Stem cells have growth factors, so they rejuvenate the skin. We inject it in the face we can micro-needle it in the face.
Youre allowed to have it once every six weeks. It definitely works, theres a reason why Kim Kardashian is having it, explains Dr Grubnik.
A vampire treatment could cost you at least R3,300 per procedure and R4,200 with PRP (platelet-rich plasma) injections.
Other skin care procedures include acne and oily skin treatments (R880), skin brightening treatment (R880), hydrating treatment (R880) and the red carpet peel for R1,300.
While Dr Grubnik encourages the use of these procedures for those who are willing, she also advocates for good skin care with the use of a sunscreen.
Sunscreen is paramount because the sun damages skin skin quite badly, so before you know it you will have very bad wrinkles and sun damage with pigmentation, regardless of the skin tone or colour everybody suffers equally.
People say there is a genetic predisposition to how you age and, to a certain extent it is true. If your mother looks fantastic at 70, youre blessed with those genes as well, but its not only the genetics. Looking after yourself always makes a difference.
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Vampire facials and other medical witchcraft you can buy to stay youthful today - Citizen
Hatching disease in a dish: The new frontier in drug testing – Maclean’s
By daniellenierenberg
Over lunch at the Canadian Centre for Alternatives to Animal Methods (CCAAM), Charu Chandrasekera nonchalantly mentions one of the projects her team is working on. We are just printing some human liver tissue right now, she says.
Chandrasekera launched the CCAAM at the University of Windsor in 2017, with help from the schools vice-president of research and innovation, Michael Siu, and dean of science, Chris Houser. The centre promotes non-animal methods in biomedical research, education, and regulatory (chemical safety) testing. In October 2019, the centre received a million-dollar gift from the Eric S. Margolis Family Foundation, which Chandrasekera says was instrumental in establishing the state-of-the art research laboratory, and in launching a number of important initiatives.
Chandrasekera says the move away from animal testing to human-based research models isnt radical but inevitable. After many years working in biomedical research with mouse models of heart disease and diabetes, It became very obvious that the work I was doing was not translatable [to humans], she says. Nothing was really reproducible; there were so many discrepancies and contradictions, even among the top-notch researchers.
Ninety-five per cent of drugs tested to be safe and effective in animal models fail in human clinical trials, says Chandrasekera. Alzheimers disease99.6 per cent drug failure rate, she says. It has been cured in mice. But we dont even understand the molecular mechanisms of this disease in humans, much less a cure.
RELATED:I am mine: This is what Alzheimers is like at 41
Empirical evidence from across a whole host of biomedical science disciplines shows us that animal models are failing both science and human health, echoes Elisabeth Ormandy, co-founder and executive director of Animals in Science Policy Institute, a registered Canadian charity working to promote better science without animals. Animal models can falsely show that a drug is effective, she says. They can also falsely show no effect, in which case a drug that would have been shown to be effective in humans never gets advanced to human clinical trials.
The result, she says, is billions of public tax dollars being wasted on research using ineffective animal models, and diversion of precious research funding away from other lines of scientific inquiry that might hold greater promise in terms of predicting drug safety, risk, and effectiveness.
Those other promising lines of scientific inquiry, say Ormandy and Chandrasekera, are human biology-based models. We can use human cells and tissues from cadavers, biopsies, and explanted organs [from surgeries], says Chandrasekera. And we can also engineer them. With adult stem cell technology, you can take a small biopsylike two-to-three millimetres from a persons skinto create any cell type in your body, she says. And if that person has a disease, such as Alzheimers, it will still be present in these cells. These cells can then be assembled to form tissue-like structures called organoids, or engineered through 3D-bioprinting to create more complex tissues, all of which can be combined to create what has become known as disease-in-a-dish. At present,Chandrasekera iscreating diabetes-in-a-dish.
Further, those cells and tissues can also be placed onto computer chips the size of thumb drives, where a large number of drugs can be tested to select whats most appropriate for youpersonalized medicine based on your cells, your tissues, your biologynot mouse biology, Chandrasekera explained in her April 2019 TedX Talk. The goal of the scientific community at large is to create a human-on-a-chip to emulate human biology better than animals, she says, which I think will happen over the next decade.
Currently there is no data on the success rates of human biology-based methods, because there are no drugs that have been approved without animal testing, since animal testing was mandated by regulatory guidelines several decades ago, says Chandrasekera.
However, a growing body of scientific data and internationally approved guidelines in chemical safety testing, indicate that alternative methods are equal or superior to animal models in predicting human biology, Chandrasekera says. Even computer simulations are out-predicting animal-derived data.
RELATED:Health care cannot modernize unless health policy changes first
Ifdisease-in-a-dish and toxicity-on-a-chip effortscontinue to advance at a fast pace with a sense of urgencybacked by global scientific, financial, legislative, and ethical mandates, she says, we will come to a point where we can test drugs without relying on animals.
And while Chandrasekera is busy both in the lab and on the global stage promoting her work, she is also focused on enlightening future scientists. Shes working the development of courses and degrees to train the next generation, she says, to think outside the cage.
Continued here:
Hatching disease in a dish: The new frontier in drug testing - Maclean's
Star Trek’s William Shatner Receives Stem Cell Treatment to Restore his Youth – Magazine of Santa Clarita
By daniellenierenberg
The 88-year-old actor known for his role as Captian James T. Kirk on the popular cinema and television series Star Trek, William Shatner recently Tweeted, Today I received restorative stem cells and told his followers Is it possible to turn back the clock? I will let you know.Mr. Shatner also tweeted the Stem Cells are manufactured by Invitrx.Center for Regenerative Medicine & Stem Cell Therapy at Valencia Medical Center is a pioneer in stem cell regenerative medicine in Santa Clarita Valley has been producing PRP and stem cell treatments for cosmetic treatments for cosmetic rejuvenation, hair restoration and chronic knee pain due to arthritis knee meniscus injury, cartilage, ligaments (ACL, MCL), osteoarthritis treatment. Invitrx, a California native is a global research-based company in regenerative medicine and is a major source of stem cell products for Valencia Medical Center.Non-surgical regenerative cell-based treatment uses the bodys natural healing ability to repair damaged bones, muscles, cartilage, tendons and ligaments. Knee injuries are painful and often patients are unable to walk. Our treatment protocol always uses products following FDA guidelines. Injections done with ultrasound guided needle recognition capability to ensure safety as well target the area needing treatment. Plasma; Alpha-2-Macroglobulim (A2M) is the new biologic treatment for your arthritic knee (osteoarthritis)When your hips hurt, or your knee is stiff, or your back is throbbing, that means your joint is bone on bone and there is no lubrication to ease movement.Regenerative medicine giving new hope to patients suffering from painful joint injuries such as knee, shoulder and hip with a chance to live a pain free life.Regenerative cell-based ultrasound guided injection now available to treat pain associated with joint injury. There are indications that it reduces the pain and swelling of the joints and helps lubricating and improve movements.Commonly Treated Conditions: Osteoarthritis of the Hips, Knee, and Shoulders Rotator Cuff tears of the Shoulder Meniscus, ACL and PCL tears of the kneeOur stem cell treatment using your own stem cells and with using imaging guidance ensures precise injection of stem cell, it is a highly-specialized practice.Besides treating above injuries we have advance stem cell micro-needling treatment for the following: Cell-based PRP Hair Restoration combining micro-needling with growth factors and hair follicles voluma vitamins plus BLotinyl T1, Biotin, Anti-aging and Kopexil. Non-toxin facial renewal Anti-Aging APGF Advanced Peptide Micro-needling PRP, Dual Anti-Aging Ampoules for deep hydration, more collagen to reduce wrinkles and firm skin.Dr. Ibrahim is the staff physician at Valencia Medical Center specializing in regenerative medicine, pain management, and rejuvenation. Call for a consultation at 661-222-9117.
The SEVEN myths and truths about healthy skin – Stock Daily Dish
By daniellenierenberg
Skin is our largest organ and something we may take for granted when its healthy.
As an academic dermatologist I frequently hear misleading facts that seem to be stubbornly enduring.
Here are some of the most commonly shared myths that can be cleared up immediately, and some truths you can rely on.
TRUE Skin constantly renews itself
The skin provides a dynamic barrier between your bodys internal environment and the outside world.
Cells called keratinocytes in the epidermis (the outer layer of skin) are constantly dividing to produce a supply of cells that move up through this layer and are shed from its surface.
Skin is a rich source of stem cells with the capacity to divide and renew themselves.
FALSEDrink two litres of water a day for healthy skin
The amount of water you drink does not directly affect your skin.
Water is supplied to the skin by blood flowing through the dermis, the inner layer of skin; water is lost from the epidermis, especially in a dry environment.
Water is needed to maintain skin hydration and when you become seriously dehydrated your skin appears dull and is less elastic.
In a healthy person the internal organs kidneys, heart and blood vessels control the amount of water reaching the skin.
There is no fixed volume of water that you need to drink, it simply depends on the amounts you are using and losing.
TRUE Stress can make skin unhealthy
There are many health issues in modern life that we blame on stress, but several skin conditions have been shown in scientific studies, to be worsened by life events, possibly via stress hormones including cortisol (a steroid hormone made in the adrenal glands).
Notable examples are alopecia areata, an auto-immune condition where the bodys immunity begins to attack the hair follicles, causing hair to fall out; psoriasis, another auto-immune condition that causes skin thickening, scaling and inflammation; and eczema, itchy red skin inflammation often occurring alongside asthma, hay fever and other allergies.
Unfortunately a flare up of these skin conditions is exactly what you dont need when you are feeling stressed or under pressure.
FALSE Eating chocolate causes acne
Acne vulgaris, the common teenage acne which can actually persist into your 30s and 40s, occurs as a result of the interaction between hormonal effects on grease glands in the skin, plus the skins immune response to blocked pores and microbes living on the skin.
Eating a high fat diet is unhealthy for many reasons, but it doesnt cause acne.
In fact some tablets prescribed for severe acne such as oral isotretinoin are better absorbed when pills are swallowed with a fatty meal and that could include chocolate.
FALSE Washing powder causes eczema
Eczema is a condition where the skin is dry, itchy and red. It is caused by a combination of genetic factors (how your skin is made) and environmental effects, leading to inflammation.
Soap, detergents and washing powders can irritate the skin and contribute to dryness because they remove oil from the skin (just as washing-up liquid removes grease from your dishes).
Biological washing powders contain enzymes proteins that break down fats and other proteins to remove stains and these can irritate sensitive skin, so they may worsen eczema.
It is important that any washing power is thoroughly rinsed out of clothing before it is worn, to avoid skin irritation.
FALSE White marks on nails = calcium deficiency
Itchiness usually occurs due to dry skin, an infection or an allergic reaction.
However, it can also be a sign of cancer.
Although it is unclear how some forms of the disease cause itchiness, medics believe it may be due to substances released by the tumour or how the body reacts to the growth.
This itching tends to be all over the body but worse on the legs and chest.
It usually goes away once cancer treatment starts.
But cancer drugs themselves can also cause itchiness, which can be a sign they are working.
Certain anti-depressants, steroid creams and complementary therapies like foot massages can help ease the irritation.
To cope people should limit the numbers of baths they have, apply unscented moisturisers, wear natural fabrics and keep rooms cool.
Keeping nails short also reduces the damage scratching the skin can cause.
Source:
Nails are manufactured in the nail matrix, an area under the skin at the top edge of your nail.
If the matrix is traumatised, bumped or bitten, an irregularity in the developing nail occurs and air can become trapped.
This appears as a white mark as the nail grows out. Calcium is important for healthy nails (as well as bones and teeth) but these white marks are not a sign of deficiency.
TRUE AND FALSE Sunshine is good for you
Many people have experienced the feel-good factor of a sunny day, but there are good and bad effects of sunlight.
Light from the sun includes a mixture of different wavelengths of light: some are visible to the human eye, some are shorter than the colours we can see these are called ultraviolet (UV) and some are longer, the infrared. Different wavelengths have different effects on skin.
UVB is used by skin to manufacture vitamin D which is essential for bone health. Without sun exposure this vitamin must be obtained from the diet.
Dermatologists use specific wavelengths of UVA and UVB in carefully controlled doses to reduce skin inflammation, a valuable treatment for some skin conditions.
But when the skin is exposed to too much UV it can damage the skin cells DNA, leading to uncontrolled growth the basis of cancer.
As a simple rule, unless you have a disease or treatment that suppresses your immune system, sunshine is good for you in moderation, but always avoid getting sunburned.
KEEP IT SIMPLE
The basic principles of keeping skin healthy are mainly common sense. You should wash your skin regularly to remove dirt, but not so much that you remove the essential moisture and water-proofing substances.
Use a moisturiser if your skin feels tight or dry a greasy ointment works best unless you have acne-prone skin, in which case you should use a non-greasy water-based cream.
Avoid stress if possible, eat a healthy diet and drink water when you feel thirsty. And finally, protect your skin from too much sun with a hat and clothing or sunscreen.
See more here:
The SEVEN myths and truths about healthy skin - Stock Daily Dish
A primer: stem cell and regenerative medicine as ‘the’ emerging therapy – Chiropractic Economics
By daniellenierenberg
No longer just for professional athletes, these are the stem cell and regenerative medicine options DCs need to know about
The health care landscape continues to evolve at a dizzying pace. Insurance deductibles are increasing, and this has placed a financial burden on patients who are required to self-pay for necessary and yet uncovered services.
The opioid crisis has left physicians with limited clinical options to treat chronic pain and dysfunction. At the same time, pressure has been placed on health care providers to provide affordable alternatives to invasive procedures that provide limited clinical options with high failure rates. This confluence of supply and demand has resulted in the growth of emerging therapies in the field of stem cell and regenerative medicine. These therapies are bringing hope to patients and new opportunities to health care providers who deliver them.
Regenerative medicine is the process of replacing or regenerating cells and tissues to restore normal function. Initially popularized by professional athletes, these therapies have become mainstream. More than 27 million Americans suffer from osteoarthritis today, and in 2030 25% of U.S. adults will be diagnosed with osteoarthritis. The global regenerative medicine market is predicted to reach more than $100 billion by 2022.
These moderately-invasive regenerative procedures are eclipsing traditional highly-invasive procedures, such as hip and knee implantation, which will have a global market of $35 million over the same period.
There are four primary regenerative medicine options:
Irritant therapies include prolotherapy, ozone and prolozone. Theyincludeadding multipleirritatingsubstances along with numbing agents into degenerated or injured joints, and areas of pain.
These therapies cause inflammation to kick-start regeneration by stimulating the body to send in macrophages, which are cells that ingest and destroy theirritantsolution and trigger the healing response. Irritant therapies are an excellenttreatmentfor all forms of musculoskeletal and joint pain includingchronic neck and back pain, and rotator cuff injuries.
The effect of irritant therapies is analogous to jump-starting the battery in a tractor to get the engine to turn over.
Protease inhibition therapy eliminates the factors causing cartilage degradation, tissue breakdown, inflammation and pain. It cleans and protects joints. It is most commonly used for patients with osteoarthritis (OA) and degenerative disc disease (DDD).
It includes therapies such as alpha-2-macroglobulin (A2M) and interleukin-1 receptor antagonist protein (IRAP). A2M and IRAP are proteins found naturally in our blood. They act as protease inhibitors by binding to and inactivating damaging proteases in the body. Proteases are catabolic enzymes that break larger molecules into smaller units. Proteases trapped in the joints catabolize cartilage and break it down, causing arthritis. A2M is a large protein made in the liver. It blocks activity for all known molecules that cause cartilage breakdown. It works like a Venus flytrap by having a bait-and-trap mechanism on two sides.
Once the proteases are bound on both sides, the molecule initiates a suicide cascade and dies, allowing it to be flushed out of the area by the body.
The binding effect of protease inhibition therapy is analogous to de-weeding a garden and tilling the soil before planting.
A fibronectin-aggrecan complex test (FACT) may be used to determine the presence of FAC, which is a biomarker or indicator of cartilage breakdown caused by proteases. FAC is a unique molecular complex that is specific for painful inflammation of the spine and cartilage.
A small sample of fluid is taken from the joint or disc and sent to a lab for testing. The test looks for the presence of FAC in the fluid sample and determines where you are: FAC+ or FAC-. FAC+ patients are identified as ideal candidates for A2M injections and have a 90% rate of responding to the A2M therapy.
Stem cell therapy is focused on concentrating the workhorses of regeneration and restoration of tissues: stem cells. This results in greater cell signaling and cell recruitment than other regenerative therapies. Stem cells are known as mesenchymal signaling cells. They are considered pluripotent, which means they are undifferentiated and can replicate into various cell and tissue types.
Stem cells are found in bone marrow, the soft spongy tissue found at the center of large bones. Introducing stem cells into an injured area initiates the healing response, repairing damaged tissue by growing new, healthy tissue. The most common stem cell therapies include bone marrow aspirate concentrate (BMA), nanofat and stromal vascular fraction.
Injecting stem cells into an injured area is analogous to planting seeds in a garden.
Growth factor therapies are focused on cell signaling and cell recruitment. Blood is made up of white blood cells, red blood cells, and platelets that are suspended in plasma. Platelets are most widely known for their ability to clot blood. Platelets are also highly rich in growth factors that are proteins that stimulate healing. When an injury occurs, platelets become activated, migrate to the site of injury and release growth factors.
Growth factor therapies are the most popular provider choice for the low-cost regeneration of tissues and include platelet-rich plasma (PRP) and platelet-rich fibrin matrix (PRFM). The therapy includes drawing the patients blood followed by centrifugation to concentrate the platelets and exclude other unwanted blood products.
Another type of growth factor therapy is amniotic fluid growth factor (GF) injection therapy. Amniotic fluid surrounds the fetus during pregnancy and provides protection and nourishment. Human amniotic fluid is sourced from consenting mothers during full-term C-sections. It contains over 200 growth factors, cytokines and proteins. The therapeutic use of amniotic fluid is regulated by the FDA. It must be tested for disease and may not include any viable cells. Amniotic fluid GF therapy has both anti-inflammatory and anti-microbial properties and includes naturally-occurring hyaluronic acid for lubrication. It is most commonly used to promote the repair and reconstruction of soft tissues including cartilage and tendons.
Exosomes are being heralded as the next frontier of growth factor therapies. While they are not cells, exosomes play a vital role in the communication and rejuvenation of all the cells in the body. Exosomes are extracellular vesicles, or small bubbles, released from cells, especially from stem cells. These culture-expanded cell secretions are derived from human placental tissue. They allow for cell-to-cell communication, transporting molecules that are important regulators of intracellular information. Exosomes act as a food source for stem cells and prolong their activity. Exosomes are anti-inflammatory and include more than 300 growth factors, cytokines and proteins.
Patients with Lyme disease, burns, chronic inflammation, autoimmune disease and other chronic degenerative diseases may benefit from including exosomes in their treatment regimen. The application of growth factor therapies is analogous to applying fertilizer to a garden to help the crop grow and flourish.
Moving stem cell and regenerative medicine forward in the treatment algorithm may eliminate the need for other ineffective or potentially harmful therapies. These therapies provide new hope for patients whose only alternatives have been long-term medication, steroid injections, and costly and time-consuming surgery and rehab.
Stem cell and regenerative medicine therapies may only be provided by licensed medical professionals following all appropriate rules and regulations. An understanding of these emerging therapies and the benefits they may provide is essential as the collaboration between doctors of medicine and chiropractic increases and we join forces to combat chronic pain, dysfunction and disease.
MARK SANNA, DC, ACRB LEVEL II, FICC, is a member of the Chiropractic Summit and a board member of the Foundation for Chiropractic Progress. He is the president and CEO of BreakthroughCoaching, and can be reached at mybreakthrough.com or800-723-8423.
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A primer: stem cell and regenerative medicine as 'the' emerging therapy - Chiropractic Economics
2 Small-Cap Biotechs That Soared Last Week – Motley Fool
By daniellenierenberg
The stocks of Magenta Therapeutics (NASDAQ:MGTA) and Molecular Templates (NASDAQ:MTEM) bolted skywards last week, to the tune of 39% and 28% respectively.
Cutting-edge gene-editing therapies, chimeric antigen receptor T-cell (CAR-T) treatments, and stem cell transplants all require priming or conditioning regimens. Doctors today utilize older chemotherapy drugs or radiation, which often lead to infection or hospitalization. Magenta Therapeutics and Molecular Templates are among the companies seeking to develop less toxic, non-chemotherapy options for patients.
Image source: Getty Images.
On Nov. 18, Molecular Templates and Vertex Pharmaceuticals (NASDAQ:VRTX) forged a discovery and development collaboration to create novel targeted conditioning regimens applicable to gene-editing, CAR-T, and stem cell transplants. Vertex shelled out $38 million of up-front cash and an equity investment in Molecular Templates. The stock barely flinched, losing $0.03 from the prior day's closing price.
The next day, Nov. 19, Vertex and its collaborator CRISPR Therapeutics announced positive safety and efficacy data for the gene-editing therapy CTX001 in its first two patients. One patient had severe sickle cell disease; the other had beta thalassemia. These interventions edit a patient's genome, potentially allowing for a one-time curative treatment. Both patients received the chemotherapy busulfan prior to CTX001.
Revisiting the prior day's collaboration announcement, biotech investors focused on comments made by Vertex about how Molecular Templates could benefit the CTX001 program.
Vertex's Chief Scientific Officer David Altshuler said,
"We believe that gene editing holds significant promise in the treatment of severe hemoglobinopathies such as sickle cell disease and beta thalassemia, and Molecular Templates' unique technology platform could play an important role in creating a targeted conditioning regimen that could replace chemotherapy currently required in conditioning regimens and thus enhance the overall future treatment experience for patients."
Investors jumped on the message from Vertex, one of the biotech industry's stalwarts: Non-chemotherapy conditioning approaches are the future for gene and cell therapies.
In response, the stocks of other companies focused on achieving that goal (like Magenta) shot up. In fact, Magenta's nearly 40% gain in share price came during a week when it didn't release any news.
Magenta plans to present data on Dec. 6 at the American Society of Hematology's Annual Meeting for its lead program CD117-ADC. Targeting a protein on hematopoietic stem cells called CD117, the treatment eliminated mutated cells without the need for chemotherapy or radiation. Magenta believes CD117-ADC can potentially be used for genetic diseases like sickle cell disease, prior to either gene therapy or hematopoietic stem cell transplantation (HSCT).
Magenta and Molecular Templates are not the only players in the field. Forty Seven and bluebird bio paired up earlier this month to develop antibody-based conditioning regimens for HSCT. According to the World Health Organization, 50,000 HSCT procedures are performed annually worldwide.
Furthermore, recently approved CAR-T for cancer, such as Kymriah from Novartis or Yescarta from Gilead Sciences, require three days of cyclophosphamide and fludarabine. Developers of these and next-generation CAR-T treatments also seek to eliminate chemotherapy or radiation.
Patients greatly need less toxic methods to prepare them for gene- and cell-based therapies, or stem cell and bone marrow transplants. Many patients, particularly the elderly, are deemed ineligible for these interventions because the toxicity could be too severe. Any success could have broad implications for the treatment of cancers and genetic diseases.
While a variety of successful approaches may ultimately emerge, Magenta has taken an early lead with CD117-ADC. Molecular Templates, with Vertex as a seasoned partner by its side, may soon leap onto the scene with a targeted approach derived from its "engineered toxin bodies" platform.
The investor takeaway is clear: New treatment modalities will be dependent on non-chemotherapy conditioning. Investors in biotech companies that can figure out that piece of the puzzle should be richly rewarded.
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2 Small-Cap Biotechs That Soared Last Week - Motley Fool
Thankful People — ‘He’s gonna owe me big’: Matthew McKee receives bone-marrow donation from twin sister Allee – Southeast Missourian
By daniellenierenberg
Allee McKee exchanges a glance with her 11-year-old twin brother Matthew as he receives a blood transfusion Oct. 29 at St. Louis Children's Hospital.
RACHAEL LONG
In late October, laughter permeated The Olson Family Garden at St. Louis Children's Hospital as Matthew McKee got the chance to do something abnormal: run and play outside.
The 11-year-old Trinity Lutheran student was diagnosed in August with aplastic anemia, a rare condition in which damage to stem cells hinders the bone marrow's production of red blood cells, white blood cells and platelets.
According to the Aplastic Anemia and MDS International Foundation, between 600 and 900 people in the United States learn they have aplastic anemia each year. Anyone can be diagnosed with the disease, but according to the foundation, aplastic anemia is most commonly diagnosed in children, young adults and older adults.
Before his diagnosis, Matthew was experiencing life the way you'd expect a young person his age would -- by spending time with his friends, attending school, tagging along on float trips and annoying his twin sister, Allee.
Just before the first week of school, strange things started happening to Matthew.
Roughly two weeks before he was hospitalized, Allee and Matthew had been wrestling when -- as part of what could only have been an epic battle between siblings -- Allee bit her brother. Their father, Jason McKee, recalled seeing a "horrific" bite mark near his son's shoulder.
"I was so angry with Allee," Jason remembered. "I said, 'Why would you bite him that hard?' And she said, 'Dad, I didn't bite him that hard.'"
On Aug. 3, Matthew returned from a float trip covered in "significant" bruising, and as his mother, Wendy McKee, recalls, "more bruising than what it should be for a normal 11-year-old boy."
Three days later, Matthew had a nosebleed that lasted for three hours. Not normal; we'll take him to see the doctor tomorrow, his mother thought.
But when tomorrow came, Matthew awoke with something his parents described to look like a "nasty rash" called petechiae, a condition that causes pinpoint, round spots to appear on the skin as a result of bleeding.
That day, the McKees took Matthew to Saint Francis Medical Center in Cape Girardeau. A few blood tests confirmed some bad news: Matthew would have to be taken to St. Louis, immediately.
Transported north by way of ambulance, Wendy and Matthew left to find answers -- they have not returned home since.
On Dec. 25, 2007, Allee was born 2 minutes before Matthew -- an important time difference, depending on who you ask.
The siblings have what their mother calls a "love-hate" relationship. It's a phase -- she hopes.
But when Matthew got sick, Allee didn't hesitate for a moment. Her parents recall one of the first things Allee said: "What can I do?"
Allee McKee maintains her balance while running atop a ledge Oct. 29 in The Olson Family Garden at St. Louis Children's Hospital.
RACHAEL LONG
"We were blessed with twins 11 years ago for a reason," Wendy said with a smile.
While half of her family has been living temporarily in St. Louis, Allee has had to go on with life in Cape Girardeau as though things are normal. But when a sibling is suddenly diagnosed with a life-threatening illness, "normal" doesn't exist.
"Oh, it's really made an impact [on Allee]," Jason said. "You know, an 11-year-old girl, it's hard for her to express her emotions. But inside, you know there's just an ocean of emotion ... about this. ... We think of Matthew, but it's so much her story, too."
Though no one can take the place of her twin, Jason said it helps Allee to have extended family and friends around.
If everything else about Allee's life has changed, her relationship with Matthew is ever the annoying, hilarious, infuriating, loving sibling relationship it always has been.
Allee McKee erupts in laughter after grossing out her 11-year-old twin brother Matthew during a break in a day of medical appointments Oct. 29 in The Olson Family Garden at St. Louis Children's Hospital.
RACHAEL LONG
Just before she was anesthetized for the transplant, Jason said Allee was beginning to feel anxious about the imminent procedure. Not for a moment forgetting the many ways to leverage something over her younger twin, Allee said, "He's gonna owe me big."
More than a month later, sitting beside Matthew while he received a blood transfusion, Allee's message remained the same. Asked how she feels about the chance to donate blood marrow to her brother, Allee, with a mischievous grin, said, "It's good because I can bring it up and he owes me."
Before they knew what was making Matthew sick, his parents said all signs pointed to leukemia.
"He had zero platelets," Wendy said of the initial blood tests run at Saint Francis.
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In fact, doctors had to rule everything else out before they could officially diagnose Matthew with aplastic anemia. Once diagnosed, the discussion surrounding odds for locating a donor tissue match was no walk in the park.
When Matthew's doctors laid out his treatment options, Jason remembered them saying, "First and foremost, we see he has a sibling; we would like to test her to see if a bone marrow transplant is even a possibility."
A successful bone-marrow transplant can cure a case of aplastic anemia in a young person, where other treatment options may be more complicated and less effective.
Allee had a 1-in-4 chance of being the right genetic match to donate and save her brother's life. Other treatment options presented to the family, as Jason remembers them, included a "drug-induced protocol that had a lesser success rate but [one that] still would have given him a chance," and placement on a national donor list, an option with a higher risk of rejection.
"As a realist, when you hear a 25% chance, I'm already thinking of Step 2, thinking about the [other options], just [crossing] my fingers and praying that Allee is a match," Jason said.
Matthew's donor needed to be a human leukocyte antigen protein match, not a blood-type match. One of Matthew's doctors in the hematology and oncology clinic at St. Louis Children's Hospital explained the science behind a human leukocyte antigen protein match.
"You get half of those proteins from your mom and half of those proteins from your dad," said Dr. Shalini Shenoy, a pediatric oncologist and the director of the pediatric stem-cell transplant program at St. Louis Children's Hospital. "Fifty percent of the time, you're going to be half-matched, so you'll get the right set from mom and maybe the wrong set from dad. ... Twenty-five percent of the time, you share no antigens, no proteins at all because you got the wrong set."
But the other 25% of the time, as was the case for Allee and Matthew, the donor and recipient will be a full match.
Shenoy explained Allee could not have been a better match for her brother, even if she had been born his identical twin.
The fact Allee and Matthew are non-identical twins, Shenoy said, means there was no guarantee they would be a match. But, hypothetically, if Matthew had an identical twin, Shenoy said there would have been "some concern" about that kind of match.
Cape Girardeau twins Matthew and Allee McKee wrestle in The Olson Family Garden during a day of medical appointments Oct. 29 at St. Louis Children's Hospital.
RACHAEL LONG
"Something happened to [Matthew's] bone marrow. His immune system just worked against his bone marrow and knocked it off. Would that have [been the case for an] identical twin? It would have been hard to say. Even if the twin was normal at the time of the transplant, would that bone marrow have held? Or would it have done the same thing again?
"Luckily they were matched, and so that made her the best donor for him," Shenoy said.
Before Matthew could receive his sister's donation, his medical team had to eliminate what was left of his immune system by way of chemotherapy. It was a 21-day process involving an isolation room and constant fear of infection.
"[There were] so many things that could be just devastating, that could make him gravely ill," Jason said. "Those 21 days, they lasted forever."
The treatment Matthew went through didn't just cost him his immune system, it also cost him his hair.
"He's written in school papers that his best attribute is his hair," Jason said. "You tell a kid he is going to lose his hair, and he fought that until the bitter end."
"He spends more time in the bathroom than myself and his sister, doing his hair," Wendy said.
Cape Girardeau twins Allee and Matthew McKee sit near their mother, Wendy McKee, as they laugh at a joke made by their father, Jason McKee (not pictured) on Oct. 29 in the Olson Family Garden at St. Louis Children's Hospital.
RACHAEL LONG
After being told he would lose most or all of his hair, Matthew stubbornly -- and with no small amount of pride -- held on to about 25% to 30% of his hair, Jason said.
"He's pretty proud of that," Jason said, laughing.
Despite prolonged isolation, chemotherapy, a bone-marrow transplant, being away from home and missing school, Matthew never lost his good spirits.
"He's had a smile on his face every day," Wendy said. "He is a very good-spirited boy; he kind of goes with the flow, and he may not like what he's doing, but by God, he's got a very positive attitude when he does it."
Matthew must remain in St. Louis for 100 days after his transplant, which took place Sept. 19. After that time is up, barring any complications, Matthew will finally return home, though he will be restricted to settings with a small number of people and limited visits from friends.
"He gets to go home but stay at home, more or less," Jason said. "We're going to have to be super, duper diligent in screening anybody that comes in to make sure they don't have any symptoms of any kind of illness."
Because his immune system had to be completely erased in order to receive a transplant, Matthew will also need to be revaccinated before he can return to life as he knew it.
"He has the immune system of a newborn," Jason said.
Some of those vaccinations he will be able to receive a year after his transplant; but for others, the waiting period is longer.
"We're looking at two years out before he can actually live life like a normal teenage boy," Wendy said.
Matthew's parents are optimistic he could return to Trinity Lutheran for the next school year.
Matthew McKee sits on top of the world during a day of medical appointments Oct. 29 at St. Louis Children's Hospital.
RACHAEL LONG
Though Wendy and Matthew have not returned to Cape Girardeau since August, life back home hasn't fallen apart -- not by any means.
"We have a wonderful family at home that is supporting us," Wendy said, noting family members have brought her winter clothes during visits, as the temperature was upwards of 90 degrees when she left town.
The family is living temporarily in a furnished Ronald McDonald apartment, keeping them close by the hospital and allowing Matthew distance from outside germs. Allee is mostly in Cape Girardeau, but she often makes trips to see her family.
Everywhere the McKees go, a community waits to support them.
"You don't realize how supportive people can be until you're put in a situation where you're in need of help," Wendy said.
A family member set up a GoFundMe fundraiser -- which may be found at gofundme.com/f/team-mckee-matthewallee-bone-marrow-transplant -- for the McKee family to help with medical bills, everyday expenses and other costs they have incurred over the last three months.
"It's so hard to take a gift from somebody," Jason said. "But so many people have come to me and said, 'This is all we can do for you, and we've got to do something.'"
But that's not the only way the community has stepped forward to help the McKees. Trinity Lutheran School in Cape Girardeau has hosted fundraisers and a blood drive in Matthew's honor.
The school even took the time to recognize Allee during one of her volleyball games.
"They had her stand up and said some words, and they gave her a standing ovation," Jason said. "It was just very special for her."
The school even sold T-shirts with the words "Team McKee" as a fundraiser for the family.
"The community has just been wonderful ... Cape Girardeau, his school, family and friends -- they've all just been amazing," Jason said.
There is no easy way to navigate life after sickness touches a family, especially for parents of a sick child. But the McKees continue to give thanks in spite of their situation.
"I am most thankful the Lord is giving us a road that can be traveled," Jason said. "Because some patients here don't ... as bad as the road is gonna be, at least there is a road."
Stem Cell Banking Market to Expand Steadily in the Coming Years till 2018-2026 – Crypto Journal
By daniellenierenberg
Stem Cell Banking Market Report 2018-2026includes a comprehensive analysis of the present Market. The report starts with the basic Stem Cell Banking industry overview and then goes into each and every detail.
Stem Cell Banking Market Report contains in depth information major manufacturers, opportunities, challenges, and industry trends and their impact on the market forecast. Stem Cell Banking also provides data about the company and its operations. This report also provides information on the Pricing Strategy, Brand Strategy, Target Client, Distributors/Traders List offered by the company.
Description:
High potential of cord blood and tissues for the treatment of patients with autoimmune diseases is expected to propel the market growth. Moreover, currently available immunosuppressive agents such as steroids, induce long term side effects despite temporary improvements. According to the Health Research Funding, 2015, around 28% of cord blood transplants have been used to treat genetic diseases, with the most common genetic disease treated being severe combined immune deficiency, followed by aplastic anemia. According to the National Cord Blood Program, 2015, cord blood from unrelated donors has been used as an alternative to bone marrow or mobilized stem cells, as a source of hematopoietic stem cells, with over 35,000 stem cell transplants successfully performed worldwide.
Stem Cell Banking Market competition by top manufacturers/players, with Stem Cell Banking sales volume, Price (USD/Unit), Revenue (Million USD) and Market Share for each manufacturer/player; the top players including: Allergan, Plc., Galderma S.A., Integra LifeSciences Corporation, Merz Pharma GmbH & Co. KGaA., Sanofi S.A., SciVision Biotech Inc., Sinclair Pharma Plc., Suneva Medical, Valeant Pharmaceuticals International, Inc., and Anika Therapeutics, Inc.
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Important Features that are under offer & key highlights of the report:
What all regional segmentation covered? Can the specific country of interest be added?Currently, the research report gives special attention and focus on the following regions:North America (U.S., Canada, Mexico), Europe (Germany, U.K., France, Italy, Russia, Spain etc), South America (Brazil, Argentina etc) & Middle East & Africa (Saudi Arabia, South Africa etc)** One country of specific interest can be included at no added cost. For inclusion of more regional segment quote may vary.
What all companies are currently profiled in the report?The report Contain the Major Key Players currently profiled in this market.** List of companies mentioned may vary in the final report subject to Name Change / Merger etc.
Can we add or profiled new company as per our need?Yes, we can add or profile new company as per client need in the report. Final confirmation to be provided by the research team depending upon the difficulty of the survey.** Data availability will be confirmed by research in case of a privately held company. Up to 3 players can be added at no added cost.
Can the inclusion of additional Segmentation / Market breakdown is possible?Yes, the inclusion of additional segmentation / Market breakdown is possible to subject to data availability and difficulty of the survey. However, a detailed requirement needs to be shared with our research before giving final confirmation to the client.** Depending upon the requirement the deliverable time and quote will vary.
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Stem Cell Banking Market Dynamics in the world mainly, the worldwide 2018-2026 Stem Cell Banking Market is analyzed across major global regions. CMI also provides customized specific regional and country-level reports for the following areas:
Region Segmentation:
North America (USA, Canada and Mexico)Europe (Germany, France, UK, Russia and Italy)Asia-Pacific (China, Japan, Korea, India and Southeast Asia)South America (Brazil, Argentina, Columbia etc.)Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)
Further in the report, the Stem Cell Banking market is examined for Sales, Revenue, Price and Gross Margin. These points are analyzed for companies, types, and regions. In continuation with this data, the sale price is for various types, applications and region is also included. The Stem Cell Banking industry consumption for major regions is given. Additionally, type wise and application wise figures are also provided in this report.
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In this study, the years considered to estimate the market size of 2018-2026 Stem Cell Banking Market are as follows:History Year: 2015-2017Base Year: 2017Estimated Year: 2018Forecast Year 2018 to 2026
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Stem Cell Banking Market to Expand Steadily in the Coming Years till 2018-2026 - Crypto Journal
BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND – GlobeNewswire
By daniellenierenberg
NEW YORK, Nov. 26, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today that the Company is proud to be a gold sponsor of the 30th International Symposium on ALS/MND.
The symposium will take place December 4 6, 2019, at the Perth Convention and Exhibition Centre in Perth, Australia. The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields.
Ralph Kern MD MHSc, BrainStorms Chief Operating and Chief Medical Officer, will deliver a podium presentation: Modulation of innate immunity by MSC-NTF (NurOwn) cells correlates with ALS clinical outcomes, on December 4, from 11:50 12:10 pm AWST during the opening day Clinical Trials Session. In addition to the podium presentation, the Company will also present Poster 153: MSC-NTF Differentiation Increases the Neurotrophic Effects of MSC Cells: Live Imaging Analysis, that directly demonstrates the neuroprotective effects of NurOwn in a neuronal cell culture model.
Our fully-enrolled phase 3 clinical trial is one of the most advanced clinical programs in ALS, stated Chaim Lebovits, President and CEO of BrainStorm. He added, The International Symposium on ALS/MND is an important venue to update the community on our clinical and scientific efforts towards the advancement of therapies that may address the unmet needs of those living with ALS. BrainStorm Cell Therapeutics is proud to serve as a sponsor of this important annual symposium which underscores our commitment to the international community of ALS and MND patients, their families and their caregivers.
Ralph Kern, MD, stated, It is a privilege to present our innovative biomarker and preclinical research at the International Symposium on ALS/MND. He added, Every year, symposium participants gather together and discuss the opportunities and the challenges that we will face during the upcoming year. Research and medical breakthroughs for the ALS and MND community continue to make significant progress and we look forward to sharing our insights and engaging with colleagues from around the globe. The International Symposium on ALS/MND reminds us how far we have come in investigational therapies and how much more progress is still needed to bring patients a better and more promising future.
About NurOwn
NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.
About BrainStorm Cell Therapeutics Inc.
BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. For more information, visit BrainStorm's website at http://www.brainstorm-cell.com.
The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields. The Symposium is planned as two parallel meetings, one on biomedical research and the other on advances in the care and management of people affected by ALS/MND. Joint sessions consider issues of mutual concern, challenging current views and practices.
Safe-Harbor Statements
Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect," "likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.
BRAINSTORM CONTACTS:Investors:Uri Yablonka, Chief Business OfficerBrainStorm Cell Therapeutics IncPhone: : +1-201-488-0460Email: uri@brainstorm-cell.com
Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839Email:sean.leous@icrinc.com
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BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND - GlobeNewswire
Homology Medicines Announces Peer-Reviewed Publication Demonstrating its AAVHSC Vectors Crossed the Blood-Brain-Barrier and Blood-Nerve-Barrier in…
By daniellenierenberg
BEDFORD, Mass., Nov. 26, 2019 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today a peer-reviewed publication demonstrating its proprietary adeno-associated viral vectors (AAVHSCs) crossed the blood-brain-barrier and blood-nerve-barrier in non-human primates (NHPs), highlighting their potential to deliver gene therapy for central and peripheral nervous system disorders.
The publication includes the initial characterization of biodistribution with three of Homologys 15 AAVHSCs, including their ability to transduce, or target, key cells following a single intravenous (I.V.) administration in NHPs. AAVHSCs are naturally occurring vectors originally isolated from human hematopoietic stem cells.
Many neurological diseases, including lysosomal storage and neuromuscular disorders, have cognitive and systemic components requiring a genetic medicine to reach multiple tissues to target the disease-relevant cell types, said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. Here we evaluated the ability of three of our novel AAVHSCs to cross the blood-brain-barrier and the blood-nerve barrier after I.V. administration in NHPs in addition to other key tissues, which allows us to choose the vectors best suited for particular diseases. We have observed that small sequence changes among our family of AAVHSCs are associated with differences in their ability to target disease-relevant tissues. We continue to characterize these properties and the potential of AAVHSCs as vehicles for therapeutic delivery.
Following I.V. administration of AAVHSC -7, -15 and -17 in NHPs, analyses showed transduction and transgene expression:
The publication, Clade F AAVHSCs Cross the Blood Brain Barrier and Transduce the Central Nervous System in Addition to Peripheral Tissues Following Intravenous Administration in Nonhuman Primates, was peer-reviewed and published in the journal PLOS ONE. For more information, please visithttps://journals.plos.org/plosone/article?id=10.1371/journal.pone.0225582or http://www.homologymedicines.com/publications.
About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.
Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; beliefs about preclinical data and the properties and potential of our AAVHSCs; and our position as a leader in the development of genetic medicines. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarter endedSeptember 30, 2019and our other filings with theSECcould cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.
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Homology Medicines Announces Peer-Reviewed Publication Demonstrating its AAVHSC Vectors Crossed the Blood-Brain-Barrier and Blood-Nerve-Barrier in...
‘I was bawling’: Mother of former Humboldt Broncos player says she’s stunned by his progress after surgery – The Globe and Mail
By daniellenierenberg
Former Humbolt Broncos player Ryan Straschnitzki. Doctors implanted an epidural stimulator in Straschnitzkis spine earlier this month and a week later injected stem cells above and below the injury in the hope that will help reverse some of the damage.
Todd Korol/The Canadian Press
The mother of a hockey player paralyzed in the Humboldt Broncos bus crash says shes stunned by the progress he has made since receiving spinal surgery in Thailand.
Doctors implanted an epidural stimulator in Ryan Straschnitzkis spine earlier this month and a week later injected stem cells above and below the injury in the hope that will help reverse some of the damage.
The 20-year-old from Airdrie, Alta., is to remain in Thailand until early December.
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Hands down Im 200-per-cent behind this. I didnt expect this kind of result this quickly, Michelle Straschnitzki said in an interview. Its definitely not a quick fix. Its not a cure, but its certainly progress and its more than weve had in 19 months.
Tom Straschnitzki, who is also in Thailand, has posted a number of videos of his sons rehab, including one where the young man was able to move a leg. Another video shows him strapped into a harness as physiotherapists slowly help him walk with the use of a machine on wheels.
Bout time he got off his ass. 1st time since he boarded the bus that horrendous day, Straschnitzki tweeted.
Therapist helping with knees and ankles so they dont buckle. Ryan did so good, I sent him to the beer store for me.
Ryan Straschnitzki was one of 13 players who were injured when an inexperienced truck driver blew through a stop sign and into the path of the Saskatchewan junior hockey teams bus in April, 2018. Sixteen others on the bus died.
Straschnitzki, who was paralyzed from the chest down, has said he isnt expecting a cure, but hopes the implant will restore some muscle movement and things such as bladder control.
A small device like a remote control is to send electrical currents to his spinal cord to try to stimulate nerves and move limbs. The implant is being programmed to stimulate certain nerves mapped out by surgeons and therapists.
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The surgery can cost up to $100,000 and isnt covered by public health care or insurance, because the epidural procedure has not been approved by Health Canada. The family is paying for it themselves. It is also performed in countries such as the United States and Switzerland, but it is much cheaper in Thailand.
The players mother, who didnt go to Thailand, said hes been low key when shes talked to him.
In typical Ryan fashion hes very quiet. All he says is hes very tired and you can tell. His body, his mind, everything is tired because hes pushing as far as he can.
Her son takes part in nerve mapping in the morning, does physio in the afternoon and then does more work with the implant, she said. He still plans to hit the ice in Bangkok with his hockey sledge before returning home.
Straschnitzki said seeing her boys progress on the videos stunned her.
I was just absolutely floored. It obviously brought the tears. I was bawling. It was unreal, she said.
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Tom said the last time Ryan walked was when he walked on the bus and then, to watch him moving his legs, walking essentially, that just rocked me.
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A sledge hockey team made up of talented Tier 1 players will be wearing Calgary Flames jerseys when they hit the ice at the upcoming 2019 USA Hockey Sled Classic which will be presented by the NHL in St. Louis next month. The Canadian Press
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'I was bawling': Mother of former Humboldt Broncos player says she's stunned by his progress after surgery - The Globe and Mail
Adult Leukemia: What You Need to Know – Dana-Farber Cancer Institute
By daniellenierenberg
Medically reviewed by Richard M. Stone, MD
More than 60,000 new cases ofadult leukemiaare diagnosed in the U.S. each year. Although it is one of the more common childhood cancers,leukemia occurs more often in older adults.
Leukemia is a cancer of the bodys blood-forming tissues that results in large numbers of abnormal or immature white blood cells. The main types of leukemia are:
AML causes the bone marrow to produce immature white blood cells (called myeloblasts). As a result, patients may have a very high or lowwhite blood cellcount, and lowred blood cellsandplatelets.
CLL is the second most common type of leukemia in adults. It is a type of cancer in which the bone marrow makes too many maturelymphocytes(a type of white blood cell).
ALL is a type of leukemia in which the bone marrow makes too many immaturelymphocytes. Similar to AML, the white blood cells can be high or low and oftentimes the platelets and red blood cells are low. This form of leukemia is more common in children than adults.
CML is usually a slowly progressing disease in which too many mature white blood cells are made in the bone marrow.
People with leukemia may experience:
Because these symptoms can be caused by a variety of other conditions, its important to check with your doctor if they arise.
While studies have shown men to be more atrisk than women, some other risk factors include:
While test procedures vary based on the type of leukemia, the two most common procedures are thecomplete blood count(CBC) test and the bone marrow aspiration biopsy.
CBC is a procedure used to check the redblood cell and platelet counts as well as the number and type of white bloodcells (the red cells carry oxygen, the white cells fight and prevent infection,and platelets control bleeding). A bone marrow aspiration biopsy involvesremoving a sample of bone marrow, including a small piece of bone by insertinga needle into the hipbone. The sample is then examined for abnormal cells.
Treatment for leukemia varies depending on the type and specific diagnosis.
The treatment for acute leukemias may be lengthy up to two years in ALL and is usually done in phases. The first phase, known as remission induction therapy, involves administering several chemotherapy drugs over a several-week period. The goal is to destroy as many cancer cells as possible to achieve a remission (in which cancer cells are undetectable, but small amounts are still present).
The second phase, known aspost-remission or consolidation therapy, seeks to kill leukemia cells thatremain after remission induction therapy. This phase may involve chemotherapyand/or a stem cell transplant.
Additional treatments may also be necessary. ALL patients, for example, may receive special treatment to prevent the disease from recurring in the spinal cord or brain.
The treatment for CML has been revolutionized by the advent of the oral medication imatinib and the second- and third-generation drugs known as tyrosine kinase inhibitors (TKIs). These are oral medications that work to inhibit the function of theBCR-ABLprotein. Many patients take these medications for the rest of their lives. In rare instances, a patient may require a stem cell transplant.
Some patients with CLL are recommended formonitoring and observation. Others,usually those with symptoms or low red cell or platelet counts, requiretreatment. Such treatment may involve intravenous chemotherapy, but often withoral therapy with pills that inhibit the function of a key protein, Brutonstyrosine kinase.
Treatments for leukemia can include:
Drugs that harness the immune system in fighting leukemia have shown considerable promise. Some monoclonal antibodies synthetic versions of immune system proteins are already in use to treat certain forms of leukemia and others are being studies in clinical trials.
Another form of immunotherapy, immune checkpoint inhibitors, which release a pent-up immune system attack on tumor cells, is being tested in several forms of leukemia. Cancer vaccines, which boost the immune systems ability to fight cancer, are being studied for use in leukemia.
CAR T-cell therapy, which uses modified immune system T cells to better target and kill tumor cells, has achieved impressive results in trials involving children and adults up to age 25 with relapsed ALL.
Research into new treatments for adult leukemia is moving along several tracks in addition to immunotherapy.
By tracking the specific abnormal genes within leukemia cells, physicians are increasingly able to tailor treatment to the unique characteristics of the disease in each patient. Targeted drugs such as imatinib and dasatinib, for example, are now used in treating patients with ALL whose leukemia cells have an abnormality known as the Philadelphia chromosome. Targeted agents including IDH or FLT3 inhibitors, which zero in on proteins made from mutated genes, have been approved to treat some patients with AML, while other such inhibitors are being tested in clinical trials.
New tests make it possible to detect ever smaller amounts of leukemia that remain after treatment. Investigators are exploring how these minute levels may influence a patients prognosis and how they might impact treatment.
Researchers are testing whether treatment periods for certain drugs can be safely reduced in some patients. For instance, studies are under way to determine if drugs such as imatinib, which are currently taken for life, can be safely stopped in some patients with CML. Researchers hope to test whether treating patients with CLL with the drug ibrutinib plus other medicine for a fixed amount of time is safe and effective.
Patients may consider treatment through a clinical trial.Dana-Farber currently has more than 30 clinical trials for adult leukemia. A national list of clinical trials is available atclinicaltrials.gov.
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Adult Leukemia: What You Need to Know - Dana-Farber Cancer Institute