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Regenexx Network Using Regenerative Medicine Technologies in Interventional Orthopedics to Treat Pain – Yahoo Finance

By Dr. Matthew Watson

BROOMFIELD, Colo., April 17, 2017 /PRNewswire/ -- "Interventional orthopedics in pain medicine practice" was recently published by Elsevier as a chapter in Techniques in Regional Anesthesia and Pain Management. The chapter, authored by Regenexx founder Christopher J. Centeno, MD examines less invasive ways to treat orthopedic pain and injuries through autologous biologics, such as stem cells and platelet rich plasma (PRP), and the shift from surgical orthopedics to interventional orthopedics.

Interventional orthopedics utilizing advanced technologies, such as ultrasound and X-ray guidance, precise percutaneous injections of autologous biologics, and bone marrow concentrate, (BMC) expand nonsurgical options in the field of orthopedics. Citing the dramatic reduction in cardiac surgery rates since the adoption of the specialty interventional cardiology, the authors reveal, "We are poised on the brink of the same change in orthopedic care." The authors also state, "The field of autologous biologics has the potential to alter the playing field of orthopedic care by allowing percutaneous injections to replace the need for more invasive orthopedic surgeries."

The chapter covers three important tenets in the developing field that will allow Interventional Orthopedics to alter traditional orthopedic care in the future. First is the rapid expansion of injectates (material being injected), such as stem cells and PRP, that can help heal damaged tissue and that can effectively treat musculoskeletal tissues. Second is the precise image-guided placement of those injectates into those damaged tissues. And third is the development of new tools that will advance this regenerative-medicine technology. The chapter also highlights research that supports the use of bone marrow stem cells and the importance of education standards and organization, training, and retraining of physicians to meet these standards.

The full chapter "Interventional orthopedics in pain medicine practice" can be found online at http://www.sciencedirect.com/science/article/pii/S1084208X16300052.

Christopher J. Centeno, MD, is the CEO of Regenexx and an international expert and specialist in regenerative medicine and the clinical use of mesenchymal stem cells in orthopedics. Dr. Centeno maintains an active research-based practice and has multiple publications listed in the US National Library of Medicine.He has also served as editor-in-chief of a medical research journal dedicated to traumatic injury and is one of the few physicians in the world with extensive experience in the culture expansion of and clinical use of adult stem cells to treat orthopedic injuries.

MEDIA CONTACT Mark Testa 155014@email4pr.com (303) 885-9630

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Billings Clinic accredited for using stem cell method to ‘successfully treat’ a rare cancer – Billings Gazette

By JoanneRUSSELL25

The prognosis was dire when Cheryl Grantham learned she had multiple myeloma, a rare form of cancer, in March 1999.

"I thought I'd be dead by Christmas," she said.

The best treatment to extend her life was a round of specialty high-dose chemotherapy, a course more potent than the normal chemotherapy prescribed to combat more common cancers.

Multiple myeloma is cancer of the plasma cells and the high-dose chemotherapy treatments fight it by destroying the cancerous cells in the bone marrow, where plasma originates. The treatments are intense enough that it can kill a patient. But it's one of the most effective ways to treat the cancer.

So doctors a few decades ago created a workaround using stem cells, extracting them from the patient's blood before administering the high-dose chemotherapy and then transplanting them back in to repair the damaged bone marrow after the chemo has been given.

Stem cells are given back to the patient like a blood transfusion, saidBrock Whittenberger, Grantham's doctor at Billings Clinic.

Billings Clinic has been using this stem cell approach with its myeloma cancer treatments for years, and Whittenberger has been the one performing procedure.

"What it's allowed us to do is successfully treat the cancer," he said. "There's a fairly rapid recovery."

Billings Clinic was recently accredited by the Foundation for the Accreditation of Cellular Therapy for its stem cell treatment.With the FACT accreditation, those treatments will be more accessible.

The accreditation also will make it easier for insurance companies to approve the procedure and will allow Billings Clinic to conduct trials on the stem cell treatment.

Billings Clinic is currently the only FACT-accredited center in Montana.

Grantham, who was an infusion nurse at the time of her diganosis, elected to have the treatment and has outlived her initial prognosis by almost two decades.

"I've been fine," she said. "I've been alive for 18 years."

Unexpectedly, the treatments helped her become a better nurse.

"It made me more empathetic," she said.

The stem cell treatment eradicates certain forms of lymphoma but it won't cure Grantham's cancer. At some point themultiple myeloma will return.

Until then, she visits with her doctor every three months for blood work and works to keep her focus on the now.

"With a diagnosis like that you have short-term goals," she said.

Her youngest son was in high school in 1999, and she was still working full time as a nurse. As much as she wanted to crawl under her covers and not face the reality of her cancer diagnosis, she had no choice but to move forward.

"It made me be normal," she said.

And it helped her focus on what was important in the moment. The Christmas before she began her treatments, she took her three sons to the Cayman Islands for the holidays.

"Because everything was going to change," she said."You just do it."

And it's an attitude she still carries. Her youngest son, long graduated from high school, is now married. These days, she's hopeful he'll give her a grandchild.

"That's my goal now," she said, smiling.

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Bone Marrow Drive & Fundraiser To Support Sarina Vito, 18, Battling Leukemia – Long Island Press

By Sykes24Tracey

On March 13th, Sarina Vito, an 18-year-old from Elwood, was preparing to spend a weekend away with her mother ather very first orientation as an incoming fall freshman at High Point University in North Carolina.

Instead, the John Glenn High School seniors entire world was flipped upside down bya devastating phone call: Sarina was diagnosed with acute myeloid (AML) leukemiaa cancer characterized by the production of a large number of abnormal white blood cells inthe bone marrow.

Sarina was immediately admitted into the Cohen Childrens Medical Center, where shewill require a lifesaving bone marrow/stem cell transplant after her third round of chemotherapy. She will also have to undergo fertility treatments in order to be able to have children of her own.

To help Sarina find her donor match as well as raise awareness and much-needed funds for not just her and her familys battle with this disease, but also for childhood cancer foundations, her family, friends and the Elwood community are holding a bone marrow drive and fundraiser at John Glenn High School on Tuesday, April 25, from 4 p.m. to 8:30 p.m. (Scroll down for official flier.)

This Bone Marrow Registry Drive & Sarinas Strands of Strength Ponytail Drive will include bone-marrow test swabbing provided by Be The Match Foundation, a bake sale, a raffle with prizes, vendor tables, music by Tony Bruno, and hair extensions by Cactus Salons. Among its proud supporters: Be The Match Foundation, Mondays at Racine, Hair We Share and Cactus Salon.

The Sarina Strong Fund also has a GoFundMe Page collecting donations.

Although Sarina will be missing many things that she worked very hard for, this event will help her and her family in many other ways. As the organizers of this benefit understand, no family should have to suffer the devastating emotional, mental and financial strains a disease such as AML leukemia causes them, especially not alone.

Consider this post an open invitation for local businesses and members of the Long Island community to contribute and lend support by donating baskets, gift certificates, services, raffle prizes, food and/or refreshments, become a sponsor with monetary donations, or simply help spread the wordthats why a special hashtag #SarinaStrong has been created to help raise awareness across social media; spread it far and wide!

Sarina and her family thank you in advance: Together, we can do this!

Featured Photo: The Elwood Community is hosting the #SarinaStrong Bone Marrow Registry Drive & Fundraiser for Sarina Vito, 18, who is battling AML leukemia, at John Glenn High School in Elwood, on April 25, 2017. (Photo: #SarinaStrong GoFundMe page)

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Ensure Medicare Access to Blood and Marrow Transplants for Seniors with Cancer – Morning Consult

By Dr. Matthew Watson

Last year, the Centers for Medicare and Medicaid Services enacted a significant policy change improving access to blood and marrow transplants for Medicare patients diagnosed with life-threatening blood cancers. The change came in the form of a Medicare rule on how outpatient blood and marrow transplants are reimbursed by the federal health care program beginning on Jan. 1, 2017.

While this move a step in the right direction, this rule does not address the vast majority of transplants (97 percent) that are performed in the inpatient setting. Sadly, Medicare continues to provide inadequate reimbursement to hospitals performing inpatient transplants and this limitation threatens to limit access to seniors needing this lifesaving therapy.

It is estimated that a new patient is diagnosed with a blood cancer every three minutes. More than 170,000 Americans will receive a blood cancer diagnosis like leukemia, lymphoma or myeloma this year alone; approximately 1.2 million Americans currently live with these diseases.

Blood and marrow transplants using a donor (allogeneic transplants) remain the only curative treatment for many blood cancers. The process of transplantation typically involves treating the patient with chemotherapy and then restoring healthy cells in the recipient by an infusion of blood or bone marrow stem cells, obtained from a matched related or unrelated donor or from umbilical cord blood. These donor cells also help to eliminate any cancer cells that survive chemotherapy.

The fatal blood diseases that require transplants occur most commonly in older individuals, who are also most likely to be covered by Medicare. Historically, the risks of transplant were too great to allow us to safely transplant many seniors. However, rapid clinical advances have resulted in dramatically improved outcomes in older adults. In fact, patients over the age of 65 are now the most rapidly growing population in U.S. transplant centers.

Despite the overwhelming clinical evidence demonstrating the curative potential of transplants in older patients, transplant access for seniors is threatened by Medicares chronic underfunding for both the transplant itself and the costs required to obtain matched bone marrow or cord blood. Medicare, for the most part, adequately reimburses transplants of solid organs such as hearts and lungs, appropriately covering the costs of acquiring those organs.

Surprisingly, Medicare treats the cost of acquiring bone marrow differently. Currently, Medicare pays for the cost of acquiring bone marrow and the transplant procedure and hospitalization in a single payment. Unfortunately, the amount currently reimbursed falls well short of the costs of providing the complex care required for blood and marrow transplant recipients, who are vulnerable to complications including infections in the post-transplant period. Unlike solid organ transplants, the cost of obtaining unrelated donor blood, bone marrow or cord blood is not directly and completely reimbursed.

This inadequate reimbursement threatens the ability of transplant centers to continue to take on the complex care of seniors with blood cancers. Unless reimbursement policies change, some seniors may face limited access to their only curative treatment option.

Thanks to national investment in research and continued innovation, seniors diagnosed with cancer today have more treatment options than they had in the past. Poor federal reimbursement policies must be updated to provide patients with access to the treatments that offer them the best possible outcomes, including transplantation.

While last years policy change was a marked improvement in reimbursement for those three percent of transplants occurring in the outpatient setting, it is important that similar payment reforms now address themajority of blood and marrowtransplants that are performed as inpatient procedures.

I urge Medicare to revise its payment policies for blood and marrow transplants to strengthen reimbursement in the inpatient hospital setting to ensure American seniors the full range of life-saving treatment options for cancer that they deserve.

Krishna Komanduri is president of the American Society for Blood and Marrow Transplantation and the Kalish Family Chair in Stem Cell Transplantation, Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine.

Morning Consult welcomes op-ed submissions on policy, politics and business strategy in our coverage areas. Updated submission guidelines can be foundhere.

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UW Treats First Participant in Trial of Stem-Cell Therapy for Heart Failure – University of Wisconsin-Madison

By Sykes24Tracey

A research team at University of Wisconsin School of Medicine and Public Health has treated its first patient in an innovative clinical trial using stem cells for the treatment of heart failure that develops after a heart attack.

The trial is taking place at University Hospital, one of three sites nationwide currently enrolling participants. The investigational CardiAMP therapy is designed to deliver a high dose of a patients own bone-marrow cells directly to the point of cardiac injury to potentially stimulate the bodys natural healing response.

The patient experience with the trial begins with a cell-potency screening test. Patients who qualify for therapy are scheduled for a bone-marrow aspiration. The bone marrow is then processed on-site and subsequently delivered directly to the damaged regions in a patients heart in a minimally invasive procedure.

Patients living with heart failure experience a variety of negative symptoms that can greatly impact their day-to-day life, said UW Health cardiologist Dr. Amish Raval, associate professor of medicine and one of the principal investigators for the trial. By being at the forefront of research for this debilitating condition, we look forward to studying the potential of this cell therapy to impact a patients exercise capacity and quality of life.

The primary outcome to be measured is the change in distance during a six-minute walk 12 months after the initial baseline measurement.

Heart failure commonly occurs after a heart attack, when the heart muscle is weakened and cannot pump enough blood to meet the body's needs for blood and oxygen. About 790,000 people in the U.S. have heart attacks each year. The number of adults living with heart failure increased from about 5.7 million (2009-2012) to about 6.5 million (2011-2014), and the number of adults diagnosed with heart failure is expected to dramatically rise by 46 percent by the year 2030, according to the American Heart Association (AHA).

The CardiAMP Heart Failure Trial is a phase III study of up to 260 patients at up to 40 centers nationwide. Phase III trials are conducted to measure effectiveness of the intervention, monitor side effects and gather information for future use of the procedure. Study subjects must be diagnosed with New York Heart Association (NYHA) Class II or III heart failure as a result of a previous heart attack.

Information about eligibility or enrollment in the trial is available at http://www.clinicaltrials.gov, or through a cardiologist.

The trial is funded by Biocardia, Inc., which developed the potential therapy.

Date Published: 04/17/2017

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Cheyenne’s Day of Giving provides an opportunity to save a life – Wyoming Business Report

By JoanneRUSSELL25

CHEYENNE Had it not been for a single bone marrow transplant, the Day of Giving would never have started.

Its a bone marrow transplant that saved founder Greta Morrows life, and what inspired her to launch a community charity event that encourages, among other things, the importance of donating blood, tissue, bone marrow and organs.

Greta is a prime example of somebody who is a survivor because of someone being on the bone marrow donation registry, said Caroline Veit, a longtime Day of Giving volunteer and a past president for the event. Its life saving. When somebody is at the end of their treatment options for blood cancer, a bone marrow transplant can be the key for their survival.

One of the most compelling reasons to sign up as a bone marrow donor at the Day of Giving now in its 12th year is not only is it capable of saving a life, but the process of actually donating is fairly straightforward, no different than giving blood something the Day of Giving also offers onsite.

Jamie Spradlin, a 22-year-old teacher at Hobbs Elementary School in Cheyenne, learned that fact firsthand late last year, when she was called on to donate bone marrow.

It was at a Relay for Life event about four years ago, they had a booth and they were explaining, Would you like to save a life; do you want to know how? And I was with a group and all of us signed up, Spradlin said. It had to have been October or November of last year when they called me and said Hey, youre actually a match for someone.

Many people who register as potential bone marrow donors never get such a call. Thats because unlike blood, which falls into one of four types plus a positive or negative Rh factor, a bone marrow can be much harder to find. Only about 30 percent of patients looking for a bone marrow match can even find one in their own family the rest have to hope a stranger in the national donor registry happens to match them, according to the nonprofit Institute for Justice.

Only about 2 percent of Americans belong to the national bone marrow registry, and at least 3,000 people die each year because they cannot find a matching donor. The odds are worse for minorities, since bone marrow type is based partly on ethnic background while Caucasians can find an unrelated donor 75 percent of the time, the percentage drops to the 40s for Hispanic and Asian patients, and 25 percent for African Americans.

What happens if you do turn out to be a match for someone?

First they asked if I was still interested in donating, and I said yes, so then they had to wait on the person I was donating to to make sure it would all work out, Spradlin said.

In December 2016, she had to take a physical to ensure she was healthy enough to donate. Be The Match, the national bone marrow registry, paid all the expenses of her testing as well as travel.

They let me choose where I went for the physical, and my sister lives in Florida, so I went to do it there, Spradlin said. A few days after that they called and said everything was great, so then I went back down to Florida for the actual donation.

The donation process takes nearly a week of preparation. Twice a day, for five days, Spradlin said she went to a clinic to receive shots that caused her bones to produce more marrow stem cells.

The first day wasnt bad, but as I continued to get them every day thats when I started noticing my back and knees getting sore, she said. You know when you go to the gym and the next day your muscles are sore? Its just like that, but with your bones.

But that was the only real discomfort, she said, and given the stakes, it wasnt a tough call to keep going. For the donation itself, Spradlin underwent a process known as apheresis, where blood is removed from the body, the marrow stem cells are separated out, and blood is then returned.

Its kind of like donating blood. They had a needle in each of my arms, she said. One needle takes out the blood, a machine separates the stem cells from the blood and then the other needle puts the blood back in your arm.

Two months after the donation, Spradlin got an email from Be The Match informing her the recipient of her bone marrow was doing well Spradlins bone marrow had taken root, and the recipients body was regaining its ability to produce healthy blood cells.

Due to confidentiality concerns, Spradlin still doesnt know whose life she saved. It wont be until a year has passed that Be The Match offers to introduce donors to recipients.

All they told me was that she was a female, 41 years old and had some type of blood cancer, Spradlin said. But even knowing just that much, she added, I would absolutely do it again. It was an easy process to save someones life, and I think its crazy not many people sign up to become donors because its not a hard process.

I mean, I got to see my sister twice in Florida and they paid for everything, she added. Frankly, I felt lucky I got to be this persons donor.

How to help

This years community-wide Day of Giving will be from 8 a.m. to 5 p.m. Friday, May 12, at the Kiwanis Community House in Lions Park. A youth event will take place there the day before, May 11, from 3:30-6:30 p.m.

There are seven ways to help on the Day of Giving:

Day of Giving sorts and delivers all donations to local agencies.

For more information, visitCheyenneDayofGiving.org.

James Chilton is the Wyoming Tribune Eagles local government reporter. He can be reached atjchilton@wyomingnews.comor 307-633-3182. Follow him on Twitter at @JournoJChilton.

To go directly to the Wyoming Tribune Eagle's website, click here.

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N.Y. bowler rolls on following bone marrow transplant – USA TODAY High School Sports

By daniellenierenberg

When you think of tough athletes, football and hockey players quickly come to mind.

But a bowler?

Someone who learned that with determination and the love of family, friends, teammates and one anonymous bone marrow donor living 1,500 miles away striking down a rare and deadly blood disease is indeed possible?

Cameron Hurwitz stands 4-foot-11 and weighs 84 pounds with Skittles in his pockets.

But the Brighton (Rochester, N.Y.) High School freshman is a big man on the lanes, leading the Barons this season with a 216.5 average, making the coveted six-man state tournament composite team, where he led Section V to a third-place finish, and being named All-Greater Rochester for the second time in three seasons.

He has rolled three 300-games (two sanctioned) and just recently recorded a personal-best 799 series in competition.

There was a time when opponents sized up Hurwitz and took him for an easy mark. No more.

Hes pretty well-known now, Brighton coach Jason Wasserman said. What they cant believe is thathes only in ninth grade and doing as well as he is. He reads lane conditions as good as anyone out there. Hes able to make adjustments on the fly, he knows what equipment to use at what time and then hes just so consistent with his shots.

Thats what happens when you bowl nearly every day from the time youre eye level to a ball rack. When you have parents, Caryn and Scott Hurwitz, who nurture your gifts with unconditional love. When a big brother, Reese, a senior on the Brighton team with a fine 210 average of his own and is headed to Purdue to bowl, is always there to cheer the strikes and help you handle the splits and open frames of life.

Cameron, 14, a hard-throwing right-hander, throws a ball that takes a sharp, last-second right-to-left hook into the pocket that makes pins explode like fireworks on the Fourth of July.

He has had many mentors but in large part he is a self-taught prodigy.

As a big PBA fan who would like to compete on tour someday, he has long watched bowling on television and the internet. He reads bowling magazines, studies the history of the gameand can recite the career statistics of PBA stars. His favorite player is a kindred spirit, 5-foot-5 Norm Duke, a family friend whose autograph he wears proudly on his green Storm bowling shirt.

For good measure, Cameron drills his own balls, customizes his own bowling shoes (blue and fluorescent green on this day), and has ideas for other bowling products that his dad, who owns a motorcycle parts manufacturing business, helps bring to life. Some have already caught the attention of people in the industry.

I think it came from watching the pros on television all the time and picking it up, Cameron said when asked where his style and passion for all things bowling comes from. I love all the physics behind bowling and just the fact you have to use your mind to be able to perform. Anybody of any size can be great at bowling as long as you know the right way to do it and as long as you know what each piece of equipment does for a particular oil pattern.

Bowling alone during off-hours, wearing a mask to prevent against infection, Cameron Hurwitz never gave up on dream of normal life and returning to Brighton High School team.(Photo: CARYN HURWITZ)

Understanding bowling science helped Cameron enjoy his best season so far, but it was medical science that got him back on the lanes.

A little more than two years ago while in the seventh grade, Cameron was getting ready to leave for the Section V tournament when his mother spotted black-and-blue marks on his arms and legs. A phone call to their family doctor led to blood work, which led to instructions to take her son to the emergency room immediately.

He had extremely low platelets, which clot your blood, and they told us to pack a bag, youll be there for many days, Caryn Hurwitz said.

It was six days to be exact, during which Cameron was diagnosed with Aplastic Anemia, a rare and serious blood disorder in which the body stops making enoughnew white and red cells and platelets.

His bone marrow had just shut down and with so few platelets he was at great risk, and with no immunity he couldnt be around people, Caryn Hurwitz said.

While undergoing treatments at Golisano Childrens Hospital, Cameron was unable to attend school and was quarantined at home for over five months. When given the OK by doctors, his lone escape was making trips to area bowling centers where generous owners allowed him to practice during off-hours to the public.

Encouraged by upticks in his white cell counts, Camerons caregivers couldnt say no when he begged to compete in the prestigious United States Bowling Congress Junior Gold national championships in the Chicago area in July 2015. While wearing an antiviral mask and in between receiving seven-hour blood transfusions at a Chicago hospital, Cameron made the televised final, placing second in the U12 division.

The boy behind the mask became a media celebrity and inspiration in the bowling community. He made the cover of Bowlers Journal and PBA stars became his fans. Hall of Famer Pete Weber posted a good luck video message on Facebook to Cameron.

Hed bowl without hardly any oxygen (in his bloodstream), Caryn Hurwitz said. I dont think people really understood how hard it was for him, but as long as he could go, even with the low blood counts, he kept bowling. When I think about, Im amazed.

Unfortunately for Cameron, the treatments he received didnt produce the desired results and as his eighth-grade school year began, he was placed on the national Be the Matchbone marrow registry.

Waiting times for a match can vary, but in Camerons case one was found in just a few months. And on Dec. 29, 2015 he underwent a transplant at Boston Childrens Hospital, a painstaking procedure where a patients body is re-started with new stem cells that need time to grow and take hold.

Six weeks in the hospital were followed by six more months of isolation, school tutoring, the entire Hurwitz family living in the germ-free lane, and the family bonding like an alleys glued wooden strips.

Throughout his recovery, Cameron kept bowling after hours, determined to be ready for his freshman season. Bowling had become his medicine.

For the full story, visit the Rochester (N.Y.) Democrat and Chronicle

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DKMS Creates Celebrity Driven #Castingforahero Campaign – Yahoo Finance

By raymumme

NEW YORK--(BUSINESS WIRE)--

DKMS, the international non-profit leading the fight against blood cancer, teamed with the film industry and launched #CastingforaHero, a campaign designed to raise awareness about diversity in bone marrow donation and to urge more people to swab their cheeks and register as potential lifesaving bone marrow/blood stem cell donors. The campaign was first launched by actor/producer Vin Diesel, has been supported on social media by Fast franchise players Tyrese Gibson, Cris Bridges (Ludacris), Don Omar and the brother of late actor Paul Walker, Cody Walker, and will be joined by other cast members to support the campaign this month. It has also garnered support by a number of celebrities including Guardians of the Galaxy actress, Zoe Saldana and Larenz Tate. Tate appeared on the television show POWER, alongside Charlie Murphy, who passed away earlier this month from complications related to blood cancer.

This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20170417005737/en/

Diesel launched the campaign on his Instagram (@vindiesel) posting, Today, premiere day (April 8), I am proud to launch#CastingforaHero- a campaign to save lives by increasing the multicultural community's presence in the worldwide bone marrow registries.

The campaign was conceived by DKMS through a partnership with Samantha Vincent, (Executive Producer, the Fate of the Furious) and Frank E. Flowers (Director, Haven) after they lost a family member to leukemia and became aware of the overwhelming odds faced by minorities and those of mixed race backgrounds of finding an unrelated match due to being underrepresented on the registry. Of all donors registered only 6% are African American, 9% are Hispanic/Latino, 6% are Asians, and 4% are Mixed Race.

In partnership with the community, one of the nations premier cross cultural creative advertising agencies, the #castingforahero concept was developed and executed through social and experiential channels leveraging key influencers with the goal of raising awareness and activating younger donors. The campaign was launched with the support of Universal Pictures, Saturday, April 8 during the Fate of the Furious #F8 premiere at Radio City Music Hall in NYC, with #castingforahero photo booths present at the F8premiere after party which gave VIP guests an opportunity to register.

In the companion video, written and directed by Flowers and produced by Andrew Molina, Anne McCarthy (Casting Director, Furious 7) and her associates audition real-life unknown actors for a hero role where they are asked to cold-read scripts about the lack of diversity on the bone marrow registry. The actors become emotional upon learning the scripts are in fact about themselves and their own chances to beat a disease like leukemia if there are not enough potential donors registered who share the same ancestry. The video calls for more trueheroes potential bone marrow/blood stem cell donors to join the effort to fight blood cancer and help find more matches for people of all ethnic backgrounds.

The newly launched website, castingforahero.com, allows people to create their own casting photo with custom skins representing unique identities and share on their social media platforms, while directing them to register with DKMS as a potential lifesaving donor.

Each year thousands of people lose the fight against blood cancer because they are unable to find their hero: a lifesaving bone marrow match, said Katharina Harf, co-founder of DKMS US. #CastingforaHero will help bring attention to the need for more diversity among potential bone marrow donors. By registering to become a DKMS donor, you can change the odds and become a life-saving hero yourself.

For more information about #CastingforaHero, visit http://www.castingforahero.com. To learn more about DKMS or register as a potential lifesaving donor, please visit http://www.dkms.org/register or @dkms.us.

About DKMS

DKMS is an international nonprofit organization dedicated to eradicating blood cancers like Leukemia and other blood-related illnesses inspiring both men and women around the world to register as bone marrow and blood stem cell donors. DKMS is providing patients with a second chance at life, working closely with families from diagnosis to transplant and beyond. The donor journey begins with a swab of the cheek that takes less than 60 seconds and can be the action that leads to a lifesaving transplant. DKMS, originally founded in Germany in 1991 by Dr. Peter Harf, has organizations in Poland, Spain, the United Kingdom and the United States. The U.S. office was started in 2004. Globally, DKMS has registered more than 7.2 million people. To join the fight against blood cancer or for more information, please go to dkms.org or @DKMS.us.

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Stem cells can now be gathered from urine samples – The Plaid Zebra (blog)

By raymumme

BY: DUSTIN BATTY

Stem cell research is a controversial topic that is often vilified in the minds of the general public. This is in part because of the vast mainstream media coverage of the debates surrounding the use of embryonic stem cells, and their tendency to refer to the issue simply as the stem cell controversy rather than specifying that the problematic stem cells are those harvested from embryos.

Embryonic stem cells aside, though, there is still some discussion in bioethical circles about the harvesting of stem cells from bone marrow and even from skin. According to a study exploring an alternative method of obtaining stem cells, the debates surrounding the extraction of stem cells from even a mildly invasive procedure such as a skin biopsy are particularly relevant when one is procuring cells from vulnerable populations, such as children and individuals with intellectual disability. The study was undertaken to prove the viability of a non-invasive method of procuring stem cells from individuals with Down syndrome.

The method used by the researchers was surprisingly successful. They managed to extract cells from urine samples that were able to become induced pluripotent stem cells (iPSC), which means that the cells were altered so they could act like stem cells. Notably, the iPSCs obtained from the urine samples were superior to those harvested from skin biopsies and other methods because theyd had no exposure to ultraviolet light, and thus their DNA was generally undamaged.

Perhaps the most significant advantage that iPSCs from urine samples have over other methods is their completely non-invasive nature. This is particularly true when collecting stem cells from individuals with Down syndrome; in the past, a significant percentage of such individuals or their parents or guardians have refused to go forward with skin biopsies, limiting the availability of material for developing treatment methods. Research ethics boards have also been known to prevent the wide-scale use of skin biopsies in individuals with DS [Down syndrome]. This new method is expected to relieve the anxieties of the individuals involved, and should be easily accepted by ethics boards as well.

The researchers expect that the use of this method will improve both the quality of cells used and the quantity available to be studied. This increased availability is important to the efficient continuation of research into treatments for Down syndrome. Although such research begins with the use of lab mice to test the viability of new treatment methods, mouse physiology is so much simpler than that of humans that such tests arent sufficient. Eventually, the treatment needs to be tested on human cells. Stem cells are particularly useful for these kinds of tests because they are able to grow into a variety of different cells, which can be tested with the treatment individually.

The researchers conclude with the assurance that the techniques they implemented could be useful not only for research into Down syndrome, but also in the study of other neurodevelopmental and neurodegenerative disorders.

Providing better quality cells with increased participation and no ethical concerns, this new method of harvesting stem cells could be the answer that medical researchers were looking for.

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Scientists have made a 3D-printed patch that can help heal the damaged heart tissue – Tech2 (blog)

By LizaAVILA

Scientists have created a revolutionary 3D-bioprinted patch that can help heal scarred heart tissue after a heart attack. The discovery is a major step forward in treating patients with tissue damage after a heart attack, researchers at University of Minnesota in the US said. During a heart attack, a person loses blood flow to the heart muscle and that causes cells to die.

Our bodies can not replace those heart muscle cells so the body forms scar tissue in that area of the heart, which puts the person at risk for compromised heart function and future heart failure. Researchers used laser-based 3D-bioprinting techniques to incorporate stem cells derived from adult human heart cells on a matrix that began to grow and beat synchronously in a dish in the lab.

When the cell patch was placed on a mouse following a simulated heart attack, the researchers saw significant increase in functional capacity after just four weeks. Since the patch was made from cells and structural proteins native to the heart, it became part of the heart and absorbed into the body, requiring no further surgeries. This is a significant step forward in treating the No 1 cause of death in the US, said Brenda Ogle, an associate professor at the University of Minnesota.

We feel that we could scale this up to repair hearts of larger animals and possibly even humans within the next several years, said Ogle. Ogle said that the research is different from previous ones as the patch is modelled after a digital, three- dimensional scan of the structural proteins of native heart tissue. The digital model is made into a physical structure by 3D printing with proteins native to the heart and further integrating cardiac cell types derived from stem cells.

Only with 3D printing of this type can we achieve one micron resolution needed to mimic structures of native heart tissue, researchers said. We were quite surprised by how well it worked given the complexity of the heart. We were encouraged to see that the cells had aligned in the scaffold and showed a continuous wave of electrical signal that moved across the patch, Ogle said.

Ogle said they are already beginning the next step to develop a larger patch that they would test on a pig heart, which is similar in size to a human heart. The study was published in the journal Circulation Research.

Publish date: April 16, 2017 12:57 pm| Modified date: April 16, 2017 12:57 pm

Tags: 3D-Bioprint, Brenda Ogle, cells, Heart, heart attack, heart failure, Journal Circulation Research, scientists, structural proteins, University of Minnesota

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CESCA Therapeutics to Present at the 2017 International Symposium of Translational Medicine in Stem Cell … – GlobeNewswire (press release)

By Dr. Matthew Watson

April 11, 2017 07:15 ET | Source: Cesca Therapeutics Inc.

RANCHO CORDOVA, Calif., April 11, 2017 (GLOBE NEWSWIRE) -- Cesca Therapeutics Inc. (Nasdaq:KOOL), a market leader in automated cell processing and point-of-care, autologous cell-based therapies, today announced that Dr. Xiaochun (Chris) Xu, Chairman and Interim Chief Executive Officer and Chairman of Boyalife Group, will present an overview of the Companys cardiovascular clinical research program at the 2017 International Symposium of Translational Medicine in Stem Cell Myocardial Repair, being held April 10-12, 2017 at the Hope Hotel in Shanghai, China.

Details of the presentation are as follows:

Despite recent therapeutic and surgical advances, the effects of peripheral arterial disease, including heart attack and critical limb ischemia (CLI), remain among the worlds leading causes of morbidity and mortality and represent a rapidly escalating public health crisis, noted Dr. Xu. I look forward to presenting a review of our latest findings, including key feasibility study results and an overview of our Phase 3 Critical Limb Ischemia Rapid Stemcell Treatment (CLIRST) trial, which we believe highlight the potential of Cesca Therapeutics proprietary AutoXpress point-of-care platform to deliver autologous cell-based therapies that may represent a new paradigm in patient treatment going forward.

About the Symposium of Translational Medicine in Stem Cell Myocardial Repair

The 2017 International Symposium of Translational Medicine in Stem Cell Myocardial Repair brings together more than 650 of the worlds cardiac disease thought leaders to discuss the potential of translational and regenerative medicine in treating myocardial infarction (MI) and cardiac failure. The symposium is co-sponsored by the Shanghai Society for Cell Biology, the Institute of Health Sciences, the Shanghai Cardiovascular Disease Institute, the Guangzhou Institutes of Biomedicine and Health, and the Key Laboratory of Stem Cell Biology, Shanghai.

About Cesca Therapeutics Inc.

Cesca is engaged in the research, development, and commercialization of cellular therapies and delivery systems for use in regenerative medicine. The Company is a leader in the development and manufacture of automated blood and bone marrow processing systems that enable the separation, processing and preservation of cell and tissue therapeutics. Cesca is an affiliate of the Boyalife Group (http://www.boyalifegroup.com), a China-based industrial-research alliance among top research institutes for stem cell and regenerative medicine.

Forward-Looking Statement

The statements contained herein may include statements of future expectations and other forward-looking statements that are based on managements current views and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. A more complete description of risks that could cause actual events to differ from the outcomes predicted by Cesca Therapeutics' forward-looking statements is set forth under the caption "Risk Factors" in Cesca Therapeutics annual report on Form 10-K and other reports it files with the Securities and Exchange Commission from time to time, and you should consider each of those factors when evaluating the forward-looking statements.

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Andrews Institute to study stem cells’ impact on knee – Pensacola News Journal

By NEVAGiles23

Joseph Baucum , jbaucum@pnj.com Published 4:06 p.m. CT April 16, 2017 | Updated 26 minutes ago

Andrews Institute is conducting new stem cell research that could impact the FDA approval of certain treatments. Joseph Baucumjbaucum@pnj.com

Dr. Andrew Anz, an orthopedic surgeon and sports medicine specialist at the Andrews Institute in Gulf Breeze is working on cutting-edge stem cell research for cartilage therapy.(Photo: Tony Giberson/tgiberson@pnj.com)Buy Photo

By the time most reach age 55, Adam Anz estimatesas much as 30 percent of the population will incur some form of knee degeneration, a problem that equals pain and in many cases, surgery.

Its a problem that were all going to face at some point in our lives, said Anz, orthopaedic surgeon at Andrews Institute for Orthopaedics & Sports Medicine.

But in May, a new study will begin at Andrews Institute in Gulf Breeze that could play a game-changing role in evolving the range of medicine available for treating knee injuries. In the process, the research may also help drive down patients costs.

Anz will help spearhead a study next month into increasing the amount of stem cells doctors are able to harvest from bone marrow transplants with the goal of utilizing those cells to regrow cartilage in knees. Cartilage, a tough and flexible material, is essential to the knee, because it acts as a cushion between the bones in the joint. Damaged cartilage can often necessitate knee replacement.

ADDITIONAL CONTENT:Andrews Institute expands prep athletics care in region

In the study, Anz said researchers will attempt to increase the amount of stem cells in participants bone marrow, which would then empty from the marrow into their bloodstream. Researchers would collect the blood, separate the stem cells from it and inject the cells into patients knees. Doctors would then monitor if the marrow cells transform into cartilage cells and spark regeneration.

Its about determining how can we obtain those cells in efficient quantities and put those cells in the right place at the right time to help with healing patients injuries, Anz said.

Because the Food & Drug Administration has not approved the vast majority of stem cell-based remedies, not all treatments involving the cells are available for patients, including the cartilage procedure. For the treatments that are offered, health insurance providers do not cover them without the FDAs consent. Patients who choose to undergo them must pay out-of-pocket prices.

The study at Andrews Institute could push a stem cell cartilage treatment closer to FDA approval and by extension, availability and affordability. The research is an official FDA study. It is led by Khay Yong Saw, a Malaysian physicianwho has already demonstrated conceptual proof of the treatment in an animal study in 2006. He completed a randomized control trial in 2012. This study is the next step in proving the safety and efficacy of the procedure to gain federal endorsement.

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Anz, optimistic about the studys potential, emphasized more research must be conducted into the effectiveness of stem cell treatments those already available and those still in the testing phase.

Its important to be excited about technologies, but its also important to be honest with the patients that more must be done to show these treatments are effective, said Anz, who estimated the cartilage study to require two years for participant enrollment and another two years before researchers can observe outcomes.

But some who have undergone stem cell treatments advocate for the procedures federal approval. Jody Falvey, a retired Pensacola resident, had a stem cell procedure conducted at Andrews Institute on her knee in the fall of 2012.

Falvey, 67, tore the medial and lateral meniscus in her knee during a family visit to South Florida while brewing coffee in the morning. The sensation, she said, felt like a knife slicing through her joint.

Following a consultation with Anz, who described an available stem cell treatment known as bone marrow aspirate concentrate, Falvey chose to have the procedure done. The treatment utilized cells from her own body to repair the knee. The process, from procedure to recovery, spanned about two years.

Falvey said her knee does not feel like it ever underwent surgery. The fact that it helped prevent her from having to undergo a knee replacement made the operation even better.

I did not want metal in my body, she said. This was just one of the greatest alternatives I had heard of. I would do it again in a heartbeat.

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Stem Cell Research Advancing Rapidly – Healthline

By JoanneRUSSELL25

Stem cells have been touted as treatments for everything from hair loss to heart disease.

But are those claims scientifically sound?

Research on the technology continues to look promising, but many of its human applications are still preliminary and their effectiveness anecdotal.

Samumed, a $12 billion biotech start-up based in San Diego, profiled this month in Business Insider, exemplifies both sides of the coin.

The company has promised a bevy of age-reversing cures, including regrowing hair, treating wrinkles, and regenerating cartilage in people with osteoarthritis

However, their research isnt conclusive.

None of their treatments have received government approval yet.

Read more: Rheumatoid arthritis and stem cell treatments

Its easy to get excited about all this research.

Samumed Is Trying to Create the Fountain of Youth, says one headline.

Samumed Aims to Reverse Aging with Eternal Youth Treatments, says another.

Combined with $300 million in investment funding, the company has more than just buzz going for it in the biotech industry.

Their treatment for androgenetic alopecia (hair loss) is currently in phase II trials.

Its program to help people with osteoarthritis regrow cartilage in their knees is in phase III.

In total, the company has seven drugs in phase II trials, with plans to expand into more areas of disease research this year.

However, Samumed has raised some eyebrows in the industry with its secrecy. Some skeptics have likened the company to Theranos, a biotech start-up that was valued at $9 billion before an investigation by the Wall Street Journal led to a shutdown of the companys labs.

Samumed has been more open about presenting their data to the public but not about the actual treatments.

We're basically telling everyone, here's proof that it works, Samumed Chief Executive Officer, Osman Kibar, told Business Insider. How it works you just need to wait a little longer because we want to build as much of a head start as we can.

Read more: Stem cell treatments for multiple sclerosis

Beyond the applications of stem cells at Samumed, the technology is also being used to treat some of the United States most widespread health issues.

New research from the American Heart Association this month demonstrated the effectiveness of implanted stem cells into the hearts of people with cardiomyopathy.

Although the sample size was small (only 27 people), scientists noted function and symptomatic improvements of heart functioning as well as less frequency of hospitalization and lower medical costs. They conclude that the stem cell procedure is a feasible treatment for cardiomyopathy, but they note that a larger clinical follow-up is needed for more conclusive results.

In the past week, Newsweek reported on miracle stem cell treatments for burn victims that will promote healing without scars.

Stat News wrote about research on stem cells in mice that could potentially help cure Parkinsons disease.

Read more: Unproved stem cell treatments offer hope and risk

Some researchers in the industry are somewhat measured in their optimism of the technologys human applications.

I want to make sure that we provide a real cautionary note, especially to those individuals and those institutions that tout stem cells as the panacea for any ill, Dr. Cato Laurencin, director of the Institute for Regenerative Engineering at the University of Connecticut, told Healthline.

Laurencin, a medical practitioner at the forefront of stem cell technology, is a firm believer in the benefits of the treatment, but also remains skeptical of some of the claims associated with it.

Much of the evidence is still preliminary or anecdotal, and when people operate on information that is preliminary or anecdotal, there is the possibility for harm, he said.

His work in regenerative engineering a term he coined several years ago looks at the healing properties of implanted stem cells in the human body.

In research published this month, Laurencin and his team concluded that stem cells effectively improved healing to torn rotator cuff tendons in rats.

Rotator cuff tendon tears are a relatively common injury in humans and can be difficult to treat.

Unlike other tendons in the body, the rotator cuff tendon is unable to heal itself, said Laurencin.

Once it is torn, it is liable to be reinjured again and again.

However, the research released this month is about more than just applying stem cells to a certain kind of injury, its about how the stem cells are applied.

Read more: Scientists use 3-D environment to speed up growth of stem cells

Laurencin describes his field as an evolution of earlier work from 30 years ago in tissue engineering: a convergence of bringing together new technologies to create new science and new possibilities.

In this case, nanotechnology is at the heart of this stem cell operation.

Currently there are a variety of ways that stem cells can be implanted into a subject, including injections and bone marrow transplants.

For his research, Laurencin and his team used biomaterial based fiber matrices a nanomaterial conducive to growing and attaching stem cells to implant into the wounded area.

The results are promising, but Laurencin and his team will have to continue working with animals for some time before the process can be applied to humans.

The key is in understanding that stem cells have the potential for more than just regrowing damaged parts of the body.

The way we commonly think about a stem cell is it becoming a new tissue. But were also understanding that the stem cell itself can secrete biological factors that help regeneration occur. Thats what we think is happening here, said Laurencin.

His research into stem cells as a medicinal element in the body could have far reaching implications for all kinds of wound therapy.

Despite his measured approach, Laurencin is still willing to hypothesize about the excitement that the future of the field undoubtedly holds with proper time, funding, and research.

There are newts and salamanders that can regenerate a limb, he told Healthline.

How do we harness the cues that are taking place in these types of animals, and can we utilize what weve learned from these types of animals in humans?

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What are stem cells? – Craig A. Kohn | TED-Ed

By LizaAVILA

Stem cells are a rapidly advancing field of biological research. Since Dr. James Thomson first cultivated human embryonic stem cells at the University of Wisconsin - Madison in the late 1990s, this field of researched has exploded with potential. The links below provide access to a curriculum developed under the supervision of Dr. Thomson as well as the co-directors and staff of the UW Stem Cell & Regenerative Medicine Center. The material has been reviewed for accuracy by the scientists actually conducting the research and was compiled and formatted by Craig Kohn, a high school teacher with research experience, for a high school audience. The PowerPoint presentation works in conjunction with the notesheet, allowing for students to work independently if preferred. More information about specific instructional practices can be found below in Teacher Notes. PowerPoint: http://bit.ly/ted-stemcells Notesheet: http://bit.ly/ted-stemcellsnotesheet Quiz: http://bit.ly/ted-stemcellsquiz Additional resources about stem cells can be found at: http://www.stemcells.wisc.edu/node/386 http://stemcells.nih.gov/Pages/Default.aspxhttp://www.stemcellschool.org/http://www.nursingdegree.net/blog/750/25-best-blogs-for-following-stem-cell-research/

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Stem cell invented that can grow into any tissue in the body – The … – The San Diego Union-Tribune

By LizaAVILA

Salk Institute and Chinese researchers report creating a new kind of stem cell, one that is more versatile than any other normally grown in the lab.

Called an extended pluripotent stem cell, it can give rise to every cell type in the body, the researchers say in a recent study. This includes the extra-embryonic tissues such as the placenta that support the developing baby. Just one cell can generate a complete organism.

Embryonic stem cells and artificial embryonic stem cells called induced pluripotent stem cells cant make these extra-embryonic tissues. So neither embryonic nor IPS cells can give rise to a complete embryo, because the supportive tissues necessary for an embryo to survive arent there.

But the extended pluripotent stem cells can reliably give rise to both types of cells, and thus whole embryos and offspring, the scientists report.

The EPS cells were made from human and mouse embryonic stem cells. In addition, they were produced from skin cells, or fibroblasts by treating them with a chemical cocktail. IPS cells, invented in 2006, are generated from fibroblasts by a similar reprogramming process.

Use of IPS cells is regarded as morally acceptable by those who oppose use of human embryonic stem cells, because they cant form an entire embryo. This is the reasoning of the Catholic Church. But since the EPS cells can make whole embryos, at least in mice, how the church will react is unclear.

To demonstrate this ability to make all cell types, the researchers grew an entire mouse from just one EPS cell. They also grew chimeric mice, with human EPS cells integrating into the mice better than embryonic stem cells did.

The study on these new stem cells was published April 6 in the journal Cell. It can be found at j.mp/extendedstem.

Better tool

That characteristic of creating every cell in the body, called totipotency, is normally found only at the very beginning of embryonic development. Embryonic stem cells are usually extracted too late, when the cells have already divided into the embryonic and extra-embryonic lineages.

Totipotent stem cells have been observed in the lab, but they lasted briefly, and didnt yield stable totipotent cell lines.

Salk Institute stem cell researcher Juan Carlos Izpisa Bemonte was a cosenior author of the paper along with Hongkui Deng of Peking University in Beijing. The first authors were Yang Yang, Bei Liu, Jun Xu, and Jinlin Wang; all of Peking University, and Jun Wu, of the Salk Institute.

EPS cell lines provide a useful cellular tool for gaining a better molecular understanding of initial cell fate commitments and generating new animal models to investigate basic questions concerning development of the placenta, yolk sac, and embryo proper, the study stated.

Furthermore, they also provide an unlimited cell resource and hold great potential for in vivo disease modeling, in vivo drug discovery, and in vivo tissue generation in the future. Finally, our study opens a path toward capturing stem cells with intra- and/or inter-species bi-potent chimeric competency from a variety of other mammalian species.

Organs for transplant

The creation of chimeric mice is part of Izpisa Bemontes longstanding goal of growing human organs in animals for transplant.

While mice are too small to grow organs for transplant, they serve as a model to understand how cells from a different species, can be grown in a host body. In this new study, the mice served as a model of how well the EPS cells can integrate.

Izpisa Bemonte is now working to translate his research on chimeric mice to pigs, which are large enough to provide human organs. In January, a team he led reported on work with human-pig chimeras, which were not allowed to grow past the embryonic stage. They also created rat-mice chimeras.

The superior chimeric competency of both human and mouse EPS cells is advantageous in applications such as the generation of transgenic animal models and the production of replacement organs, Wu said in a Salk statement. We are now testing to see whether human EPS cells are more efficient in chimeric contribution to pigs, whose organ size and physiology are closer to humans.

We believe that the derivation of a stable stem cell line with totipotent-like features will have a broad and resounding impact on the stem cell field, Izpisua Belmonte said in the statement.

The work was funded by a number of sources. They include: the National Key Research and Development Program of China; the National Natural Science Foundation of China; the Guangdong Innovative and Entrepreneurial Research Team Program; the Science and Technology Planning Project of Guangdong Province, China; the Science and Technology Program of Guangzhou, China; the Ministry of Education of China (111 Project); the BeiHao Stem Cell and Q9 Regenerative Medicine Translational Research Institute; the Joint Institute of Peking University Health Science Center; University of Michigan Health System; Peking-Tsinghua Center for Life Sciences; the National Science and Technology Support Project; the CAS Key Technology Talent Program; the G. Harold and Leila Y. Mathers Charitable Foundation; and The Moxie Foundation.

bradley.fikes@sduniontribune.com

(619) 293-1020

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Telomerase reverse transcriptase – Wikipedia

By LizaAVILA

TERT Identifiers Aliases TERT, CMM9, DKCA2, DKCB4, EST2, PFBMFT1, TCS1, TP2, TRT, hEST2, hTRT, telomerase reverse transcriptase External IDs OMIM: 187270 MGI: 1202709 HomoloGene: 31141 GeneCards: TERT Genetically Related Diseases breast cancer, interstitial lung disease, adenocarcinoma of the lung, prostate cancer, se atraganto con un caramelo, testicular germ cell cancer, idiopathic pulmonary fibrosis, malignant glioma[1] RNA expression pattern More reference expression data Orthologs Species Human Mouse Entrez Ensembl UniProt RefSeq (mRNA) RefSeq (protein) Location (UCSC) Chr 5: 1.25 1.3 Mb Chr 13: 73.63 73.65 Mb PubMed search [2] [3] Wikidata View/Edit Human View/Edit Mouse

Telomerase reverse transcriptase (abbreviated to TERT, or hTERT in humans) is a catalytic subunit of the enzyme telomerase, which, together with the telomerase RNA component (TERC), comprises the most important unit of the telomerase complex.[4][5]

Telomerases are part of a distinct subgroup of RNA-dependent polymerases. Telomerase lengthens telomeres in DNA strands, thereby allowing senescent cells that would otherwise become postmitotic and undergo apoptosis to exceed the Hayflick limit and become potentially immortal, as is often the case with cancerous cells. To be specific, TERT is responsible for catalyzing the addition of nucleotides in a TTAGGG sequence to the ends of a chromosomes telomeres.[6] This addition of repetitive DNA sequences prevents degradation of the chromosomal ends following multiple rounds of replication.[7]

hTERT absence (usually as a result of a chromosomal mutation) is associated with the disorder Cri du chat.[8][9]

Telomerase is a ribonucleoprotein polymerase that maintains telomere ends by addition of the telomere repeat TTAGGG. The enzyme consists of a protein component with reverse transcriptase activity, encoded by this gene, and an RNA component that serves as a template for the telomere repeat. Telomerase expression plays a role in cellular senescence, as it is normally repressed in postnatal somatic cells, resulting in progressive shortening of telomeres. Studies in mice suggest that telomerase also participates in chromosomal repair, since de novo synthesis of telomere repeats may occur at double-stranded breaks. Alternatively spliced variants encoding different isoforms of telomerase reverse transcriptase have been identified; the full-length sequence of some variants has not been determined. Alternative splicing at this locus is thought to be one mechanism of regulation of telomerase activity.[10]

The hTERT gene, located on chromosome 5, consists of 16 exons and 15 introns spanning 35 kb. The core promoter of hTERT includes 330 base pairs upstream of the translation start site (AUG since it's RNA by using the words "exons" and "introns"), as well as 37 base pairs of exon 2 of the hTERT gene.[11][12][13] The hTERT promoter is GC-rich and lacks TATA and CAAT boxes but contains many sites for several transcription factors giving indication of a high level of regulation by multiple factors in many cellular contexts.[11] Transcription factors that can activate hTERT include many oncogenes (cancer-causing genes) such as c-Myc, Sp1, HIF-1, AP2, and many more, while many cancer suppressing genes such as p53, WT1, and Menin produce factors that suppress hTERT activity .[13][14] Another form of up-regulation is through demethylation of histones proximal to the promoter region, imitating the low density of trimethylated histones seen in embryonic stem cells.[15] This allows for the recruitment of histone acetyltransferase (HAT) to unwind the sequence allowing for transcription of the gene.[14]

Telomere deficiency is often linked to aging, cancers and the conditions dyskeratosis congenita (DKC) and Cri du chat. Meanwhile, over-expression of hTERT is often associated with cancers and tumor formation.[8][16][17][18] The regulation of hTERT is extremely important to the maintenance of stem and cancer cells and can be used in multiple ways in the field of regenerative medicine.

hTERT is often up-regulated in cells that divide rapidly, including both embryonic stem cells and adult stem cells.[17] It elongates the telomeres of stem cells, which, as a consequence, increases the lifespan of the stem cells by allowing for indefinite division without shortening of telomeres. Therefore, it is responsible for the self-renewal properties of stem cells. Telomerase are found specifically to target shorter telomere over longer telomere, due to various regulatory mechanisms inside the cells that reduce the affinity of telomerase to longer telomeres. This preferential affinity maintains a balance within the cell such that the telomeres are of sufficient length for their function and yet, at the same time, not contribute to aberrant telomere elongation [19]

High expression of hTERT is also often used as a landmark for pluripotency and multipotency state of embryonic and adult stem cells. Over-expression of hTERT was found to immortalize certain cell types as well as impart different interesting properties to different stem cells.[13][20]

hTERT immortalizes various normal cells in culture, thereby endowing the self-renewal properties of stem cells to non-stem cell cultures.[13][21] There are multiple ways in which immortalization of non-stem cells can be achieved, one of which being via the introduction of hTERT into the cells. Differentiated cells often express hTERC and TP1, a telomerase-associated protein that helps form the telomerase assembly, but does not express hTERT. Hence, hTERT acts as the limiting factor for telomerase activity in differentiated cells [13][22] However, with hTERT over-expression, active telomerase can be formed in differentiated cells. This method has been used to immortalize prostate epithelial and stromal-derived cells, which are typically difficult to culture in vitro. hTERT introduction allows in vitro culture of these cells and available for possible future research. hTERT introduction have an advantage over the use of viral protein for immortalization in that it does not involve the inactivation of tumor suppressor gene, which might lead to cancer formation.[21]

Over-expression of hTERT in stem cells changes the properties of the cells.[20][23][24] hTERT over-expression increases the stem cell properties of human mesenchymal stem cells. The expression profile of mesenchymal stem cells converges towards embryonic stem cells, suggesting that these cells may have embryonic stem cell-like properties. However, it has been observed that mesenchymal stem cells undergo decreased levels of spontaneous differentiation.[20] This suggests that the differentiation capacity of adult stem cells may be dependent on telomerase activities. Therefore, over-expression of hTERT, which is akin to increasing telomerase activities, may create adult stem cells with a larger capacity for differentiation and hence, a larger capacity for treatment.

Increasing the telomerase activities in stem cells gives different effects depending on the intrinsic nature of the different types of stem cells.[17] Hence, not all stem cells will have increased stem-cell properties. For example, research has shown that telomerase can be upregulated in CD34+ Umbilical Cord Blood Cells through hTERT over-expression. The survival of these stem cells was enhanced, although there was no increase in the amount of population doubling.[24]

Deregulation of telomerase expression in somatic cells may be involved in oncogenesis.[10]

Genome-wide association studies suggest TERT is a susceptibility gene for development of many cancers,[25] including lung cancer.[26]

Telomerase activity is associated with the number of times a cell can divide playing an important role in the immortality of cell lines, such as cancer cells. The enzyme complex acts through the addition of telomeric repeats to the ends of chromosomal DNA. This generates immortal cancer cells.[27] In fact, there is a strong correlation between telomerase activity and malignant tumors or cancerous cell lines.[28] Not all types of human cancer have increased telomerase activity. 90% of cancers are characterized by increased telomerase activity.[28]Lung cancer is the most well characterized type of cancer associated with telomerase.[29] There is a lack of substantial telomerase activity in some cell types such as primary human fibroblasts, which become senescent after about 3050 population doublings.[28] There is also evidence that telomerase activity is increased in tissues, such as germ cell lines, that are self-renewing. Normal somatic cells, on the other hand, do not have detectable telomerase activity.[30] Since the catalytic component of telomerase is its reverse transcriptase, hTERT, and the RNA component hTERC, hTERT is an important gene to investigate in terms of cancer and tumorigenesis.

The hTERT gene has been examined for mutations and their association with the risk of contracting cancer. Over two hundred combinations of hTERT polymorphisms and cancer development have been found.[29] There were several different types of cancer involved, and the strength of the correlation between the polymorphism and developing cancer varied from weak to strong.[29] The regulation of hTERT has also been researched to determine possible mechanisms of telomerase activation in cancer cells. Glycogen synthase kinase 3 (GSK3) seems to be over-expressed in most cancer cells.[27] GSK3 is involved in promoter activation through controlling a network of transcription factors.[27]Leptin is also involved in increasing mRNA expression of hTERT via signal transducer and activation of transcription 3 (STAT3), proposing a mechanism for increased cancer incidence in obese individuals.[27] There are several other regulatory mechanisms that are altered or aberrant in cancer cells, including the Ras signaling pathway and other transcriptional regulators.[27]Phosphorylation is also a key process of post-transcriptional modification that regulates mRNA expression and cellular localization.[27] Clearly, there are many regulatory mechanisms of activation and repression of hTERT and telomerase activity in the cell, providing methods of immortalization in cancer cells.

If increased telomerase activity is associated with malignancy, then possible cancer treatments could involve inhibiting its catalytic component, hTERT, to reduce the enzymes activity and cause cell death. Since normal somatic cells do not express TERT, telomerase inhibition in cancer cells can cause senescence and apoptosis without affecting normal human cells.[27] It has been found that dominant-negative mutants of hTERT could reduce telomerase activity within the cell.[28] This led to apoptosis and cell death in cells with short telomere lengths, a promising result for cancer treatment.[28] Although cells with long telomeres did not experience apoptosis, they developed mortal characteristics and underwent telomere shortening.[28] Telomerase activity has also been found to be inhibited by phytochemicals such as isoprenoids, genistein, curcumin, etc.[27] These chemicals play a role in inhibiting the mTOR pathway via down-regulation of phosphorylation.[27] The mTOR pathway is very important in regulating protein synthesis and it interacts with telomerase to increase its expression.[27] Several other chemicals have been found to inhibit telomerase activity and are currently being tested as potential clinical treatment options such as nucleoside analogues, retinoic acid derivatives, quinolone antibiotics, and catechin derivatives.[30] There are also other molecular genetic-based methods of inhibiting telomerase, such as antisense therapy and RNA interference.[30]

hTERT peptide fragments have been shown to induce a cytotoxic T-cell reaction against telomerase-positive tumor cells in vitro.[31] The response is mediated by dendritic cells, which can display hTERT-associated antigens on MHC class I and II receptors following adenoviral transduction of an hTERT plasmid into dendritic cells, which mediate T-cell responses.[32] Dendritic cells are then able to present telomerase-associated antigens even with undetectable amounts of telomerase activity, as long as the hTERT plasmid is present.[33]Immunotherapy against telomerase-positive tumor cells is a promising field in cancer research that has been shown to be effective in in vitro and mouse model studies.[34]

Induced pluripotent stem cells (iPS cells) are somatic cells that have been reprogrammed into a stem cell-like state by the introduction of four factors (Oct3/4, Sox2, Klf4, and c-Myc).[35] iPS cells have the ability to self-renew indefinitely and contribute to all three germ layers when implanted into a blastocyst or use in teratoma formation.[35]

Early development of iPS cell lines were not efficient, as they yielded up to 5% of somatic cells successfully reprogrammed into a stem cell-like state.[36] By using immortalized somatic cells (differentiated cells with hTERT upregulated), iPS cell reprogramming was increased by twentyfold compared to reprogramming using mortal cells.[36]

The reactivation of hTERT, and subsequently telomerase, in human iPS cells has been used as an indication of pluripotency and reprogramming to an ES (embryonic stem) cell-like state when using mortal cells.[35] Reprogrammed cells that do not express sufficient hTERT levels enter a quiescent state following a number of replications depending on the length of the telomeres while maintaining stem cell-like abilities to differentiate.[36] Reactivation of TERT activity can be achieved using only three of the four reprogramming factors described by Takahashi and Yamanaka: To be specific, Oct3/4, Sox2 and Klf4 are essential, whereas c-Myc is not.[15] However, this study was done with cells containing endogenous levels of c-Myc that may have been sufficient for reprogramming.

Telomere length in healthy adult cells elongates and acquires epigenetic characteristics similar to those of ES cells when reprogrammed as iPS cells. Some epigenetic characteristics of ES cells include a low density of tri-methylated histones H3K9 and H4K20 at telomeres, as well as an increased detectable amount of TERT transcripts and protein activity.[15] Without the restoration of TERT and associated telomerase proteins, the efficiency of iPS cells would be drastically reduced. iPS cells would also lose the ability to self-renew and would eventually senesce.[15]

DKC (dyskeratosis congenita) patients are all characterized by the defective maintenance of telomeres leading to problems with stem cell regeneration.[16] iPS cells derived from DKC patients with a heterozygous mutation on the TERT gene display a 50% reduction in telomerase activity compared to wild type iPS cells.[37] Conversely, mutations on the TERC gene (RNA portion of telomerase complex) can be overcome by up-regulation due to reprogramming as long as the hTERT gene is intact and functional.[38] Lastly, iPS cells generated with DKC cells with a mutated dyskerin (DKC1) gene cannot assemble the hTERT/RNA complex and thus do not have functional telomerase.[37]

The functionality and efficiency of a reprogrammed iPS cell is determined by the ability of the cell to re-activate the telomerase complex and elongate its telomeres allowing for self-renewal. hTERT is a major limiting component of the telomerase complex and a deficiency of intact hTERT impedes the activity of telomerase, making iPS cells an unsuitable pathway towards therapy for telomere-deficient disorders.[37]

Although the mechanism is not fully understood, exposure of TERT-deficient hematopoietic cells to androgens resulted in an increased level of TERT activity.[39] Cells with a heterozygous TERT mutation, like those in DKC (dyskeratosis congenita) patients, which normally exhibit low baseline levels of TERT, could be restored to normal levels comparable to control cells. TERT mRNA levels are also increased with exposure to androgens.[39] Androgen therapy may become a suitable method for treating circulatory ailments such as bone marrow degeneration and low blood count linked with DKC and other telomerase-deficient conditions.[39]

As organisms age and cells proliferate, telomeres shorten with each round of replication. Cells restricted to a specific lineage are capable of division only a set number of times, set by the length of telomeres, before they senesce.[40] Depletion and uncapping of telomeres has been linked to organ degeneration, failure, and fibrosis due to progenitors' becoming quiescent and unable to differentiate.[19][40] Using an in vivo TERT deficient mouse model, reactivation of the TERT gene in quiescent populations in multiple organs reactivated telomerase and restored the cells abilities to differentiate.[41] Reactivation of TERT down-regulates DNA damage signals associated with cellular mitotic checkpoints allowing for proliferation and elimination of a degenerative phenotype.[41] In another study, introducing the TERT gene into healthy one-year-old mice using an engineered adeno-associated virus led to a 24% increase in lifespan, without any increase in cancer.[42]

The hTERT gene has become a main focus for gene therapy involving cancer due to its expression in tumor cells but not somatic adult cells.[43] One method is to prevent the translation of hTERT mRNA through the introduction of siRNA, which are complimentary sequences that bind to the mRNA preventing processing of the gene post transcription.[44] This method does not completely eliminate telomerase activity, but it does lower telomerase activity and levels of hTERT mRNA seen in the cytoplasm.[44] Higher success rates were seen in vitro when combining the use of antisense hTERT sequences with the introduction of a tumor-suppressing plasmid by adenovirus infection such as PTEN.[45]

Another method that has been studied is manipulating the hTERT promoter to induce apoptosis in tumor cells. Plasmid DNA sequences can be manufactured using the hTERT promoter followed by genes encoding for specific proteins. The protein can be a toxin, an apoptotic factor, or a viral protein. Toxins such as diphtheria toxin interfere with cellular processes and eventually induce apoptosis.[43] Apoptotic death factors like FADD (Fas-Associated protein with Death Domain) can be used to force cells expressing hTERT to undergo apoptosis.[46] Viral proteins like viral thymidine kinase can be used for specific targeting of a drug.[47] By introducing a prodrug only activated by the viral enzyme, specific targeting of cells expressing hTERT can be achieved.[47] By using the hTERT promoter, only cells expressing hTERT will be affected and allows for specific targeting of tumor cells.[43][46][47]

Aside from cancer therapies, the hTERT gene has been used to promote the growth of hair follicles.[48]

A schematic animation for gene therapy is shown as follows.

Telomerase reverse transcriptase has been shown to interact with:

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Breakthrough in cardiac health: 3D-printed patch can help mend a ‘broken’ heart – Economic Times

By NEVAGiles23

WASHINGTON D.C: Scientists have developed a revolutionary 3D-bioprinted patch that could one day be used to repair damage to the human heart.

The patch can help heal scarred heart tissue after a heart attack. The discovery is a major step forward in treating patients with tissue damage after a heart attack.

The researchers from the University of Minnesota-Twin Cities, University of Wisconsin-Madison, and University of Alabama-Birmingham used laser-based 3D-bioprinting techniques to incorporate stem cells derived from adult human heart cells on a matrix that began to grow and beat synchronously in a dish in the lab.

When the cell patch was placed on a mouse following a simulated heart attack, the researchers saw significant increase in functional capacity after just four weeks. Since the patch was made from cells and structural proteins native to the heart, it became part of the heart and absorbed into the body, requiring no further surgeries.

"This is a significant step forward in treating the No. 1 cause of death in the U.S.," said researcher Brenda Ogle. "We feel that we could scale this up to repair hearts of larger animals and possibly even humans within the next several years."

Ogle said that this research is different from previous research in that the patch is modelled after a digital, three-dimensional scan of the structural proteins of native heart tissue. The digital model is made into a physical structure by 3D printing with proteins native to the heart and further integrating cardiac cell types derived from stem cells. Only with 3D printing of this type can we achieve one micron resolution needed to mimic structures of native heart tissue.

"We were quite surprised by how well it worked given the complexity of the heart," Ogle noted. "We were encouraged to see that the cells had aligned in the scaffold and showed a continuous wave of electrical signal that moved across the patch."

Ogle said they are already beginning the next step to develop a larger patch that they would test on a pig heart, which is similar in size to a human heart.

The research study is published in Circulation Research, a journal published by the American Heart Association.

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New hope with haplo-identical bone marrow transplant – Star2.com

By daniellenierenberg

Having worked at University Malaya Medical Centres (UMMC) Paediatrics Department for 20 years, senior consultant paediatric oncologist Prof Dr Hany Mohd Ariffin has had to tell her fair share of parents that there is nothing more that can be done for their terminally ill child.

As head of the Paediatric Haematology-Oncology and Bone Marrow Transplantation Unit, this is usually because there is no suitable donor available for a life-saving bone marrow transplant for the child.

Bone marrow transplants, also called stem cell transplants, are used in conditions where the patients bone marrow is damaged or destroyed by disease or intensive cancer treatment, and is unable to carry out its job of producing healthy red blood cells, white blood cells and platelets.

Because white blood cells or leukocytes are part of the immune system that protects our body against foreign invaders, it is critical in such a procedure to match the so-called immunological fingerprints of the patient and the donor.

As Prof Hany explains, these fingerprints are known as human leukocyte antigens (HLAs).

HLAs help the immune system distinguish between the bodys own cells and foreign cells, usually bacteria and viruses that infect us, so that our white blood cells can find and destroy them.

It is crucial that a bone marrow donor and the patient have the same HLAs in order to minimise the chances of the donated bone marrows white blood cells considering its new host body as foreign and attacking it.

Perfect match needed

Standard bone marrow transplantations require that all 10 HLAs in both patient and donor are a match.

As HLAs are inherited half from each parent, this means that only a patients siblings are a possible perfect match.

Explains Prof Hany: If you look at statistics, out of four, one sibling will be completely matched, one sibling will be completely not matched, and two siblings would be half-matched.

So, the chances of finding a match is 25%, but that is statistical randomisation.

In the real world, you can have 10 siblings and all of them might not be matched with you.

If a patient does not have a sibling that matches perfectly with them, or does not have a sibling at all, their only other option is to check for an unrelated match in international stem cell registries or blood banks.

However, Prof Hany notes that this usually requires a sum of RM100,000 for a unit of bone marrow and at least three to four months of waiting two luxuries not all patients have.

She adds: But it is not easy to get a good match for Asians as these registries are usually Caucasian.

And its even worse if you are an Indian patient, as you cant even go to a Taiwanese blood bank.

In the case of Muhammad Yusuff Iskandar Mohd Hambali, time was a critical factor.

The firstborn of two teachers had been referred to UMMC at 10 months of age for recurrent pneumonia.

His mother, secondary school physical education teacher, Aduratun Nasyihin Mokhtar shares: He started falling sick at the age of seven months he had a persistent cough.

Initially, the doctor thought it was pertussis, but it didnt get better after three months as pertussis should, so he was admitted to the hospital.

However, none of the antibiotics they tried worked, so he was referred to UMMC to check his lungs.

This filepic shows a thalassaemia patient with his infusion pump machine for iron-chelating therapy. Thalassaemia is one of the conditions curable by a bone marrow transplant.

It was in UMMC that Yusuff, as he is called, was discovered to have X-linked severe combined immunodeficiency (SCID).

This rare genetic condition, also known as bubble boy disease, results in the malfunction or lack of two specialised white blood cells called T and B cell lymphocytes.

This means that Yusuff effectively had a non-existent immune system.

This was the reason he could not fight off the pneumonia. In fact, his lungs had deteriorated so badly that he was on oxygen therapy from the age of eight months.

In addition, the Mycobacterium bovis in his BCG vaccination had spread to his back, he had chronic diarrhoea and he was very much underweight.

Yusuff needed a bone marrow transplant, and he needed it fast.

Having reached out to her international colleagues at that time, Prof Hany says: One thing constant in all their advice was that if we delayed the procedure, he would never get better from his disseminated BCG, his pneumonia would just worsen, and once you reach a critical point, there would be no turning back.

He would have been dead by six months.

The problem was that Yusuff was then an only child.

Although his mother was pregnant with his younger sister at that time, she would not have been born in time to help him, assuming that she was a match for him in the first place.

With no time to waste, Prof Hany and her team decided to try a procedure called haplo-identical bone marrow transplantation.

On whether she and her team were ready to carry out the new procedure, Prof Hany says that you will never be ready until a life is dangling precariously in front of you. Photo: The Star/Samuel Ong

In this procedure, only five out of 10 HLAs need to be matched in order for the donor to be able to give bone marrow to the patient.

The beauty of this procedure is that you always have two parents (to donate), says Prof Hany.

So, Yusuffs father, sports science and physical education teacher Mohd Hambali Din @ Ismail, could now donate his bone marrow cells to his son.

First though, Yusuff needed to be fattened up via nutritional fluids infused into his veins, his pneumonia brought under control and his M. bovis infection treated with anti-tuberculosis therapy.

This was so that he would be in a decent enough condition to withstand the procedure.

Following the protocol established by Johns Hopkins University in the United States, but modified to suit Yusuffs condition, Prof Hany and her team first killed off Yusuffs remaining bone marrow cells through chemothera-py, before infusing 30ml of his fathers donated bone marrow into him.

Prof Hany explains that it takes two to three weeks for the new bone marrow cells to grow, during which time the patient is completely vulnerable to any infection.

This is why they remain in a completely sealed room where the air is hepa-filtered, they receive no visitors, and their food and linen are completely sterile, she says.

He was also treated with high-dose cyclophosphamide, a chemotherapy drug that targets T cell lymphocytes.

This was in order to destroy the half-matched mature T cells that came with his fathers donated bone marrow.

T cells are your soldier cells. His fathers T cells would recognise Yusuff as foreign and destroy everything in their wake.

And that is what has precluded mismatched transplants all this while, explains Prof Hany.

After the mature T cells are destroyed, she says: What you then get are T cells from stem cell origin, which learn to tolerate the environment of being in Yusuffs body, and therefore, they will be less aggressive and more friendly to these cells that they consider foreign.

Despite that, Yusuff still experienced graft-versus-host disease (GvHD) where his new white blood cells attacked the cells of his skin, gut and lungs.

In between, he also had two episodes of sepsis and he had to go to the ICU once.

He also had to go on the ventilator at one point, says Prof Hany.

She explains that GvHD, which is due to aggressive donor white blood cells, and infections, which are due to the still incomplete immune system, can co-exist, creating a dilemma for the medical team.

On the one hand, to ameliorate GvHD, you have to give steroids (in addition to standard immunosuppresants) to dampen down the immune system.

You dampen down the immune system, then you allow bacteria and fungi to grow.

And that is why it is very challenging, she says.

She admits: For the first 20 days, it was all very smooth and you think, Wah, Im a hero, but then the challenges came.

There were certain moments when I thought, Thats it, were going to lose him.

It took 149 days after the transplant before Yusuff was deemed well enough to be sent home.

And it was one year before Prof Hany and her team felt confident enough to declare him cured.

We estimate anything between six months to a year for the new bone marrow cells to grow and propagate.

So usually, after a year, if the GvHD doesnt appear anymore, it is very unlikely to suddenly appear, she explains.

This first anniversary of Yusuffs transplant, celebrated at UMMC on April 6, was not just sweet because of Yusuffs survival, it was also the opening of a new path for Prof Hany and her team.

On a personal note, there were many times when you have this period of self-doubt.

So, you think that we are just a bunch of stupid, gung-ho people, who are unrealistic; this is not America, this cannot be done that sort of feeling.

There were some moments when you think, have I done a disservice to this child? Would if it have been better to just let go, for the parents to just let go? Is God just testing me? shares Prof Hany.

However, a few months after Yusuffs transplant, she received the case of a baby boy with myelodysplastic syndrome.

Myelodysplastic children will progress to develop acute myeloid leukaemia within a year, and it is only curable with transplant, or not it is certain death by two years, she explains.

And this patient had two siblings, both of whom were only half-matched.

But we were able to offer a transplant to this child, because we knew that from the experience of Yusuff, if he has very bad GvHD of the gut, skin, lung, we would be able to handle it been there, done that.

We were already scarred for life, she says with a laugh. And in fact, due to their prior experience, Prof Hany and her team were able to more precisely determine the amount of donated bone marrow cells needed for transplant.

As a result, she says: The second patient sailed through and was discharged after only five weeks, as opposed to five months for Yusuff.

Explaining the potential impact of having this treatment option available, Prof Hany shares that bone marrow transplantation is a cure for conditions like leukaemia, blood disorders like thalassaemia, congenital defective immune systems and certain rare congenital metabolic conditions.

The major reason why transplants are not being done is because of the lack of an available donor, she says.

But haplo-identical bone marrow transplantation now opens the way for many more potential donors to help the patient.

The learning curve is steep, Prof Hany admits, but adds that after Yusuff, they were able to apply what they learnt to their second patient with great effect.

Im not saying it is easy, but I think it is worth developing further, because it can solve one of the greatest health problems in our country, which is inherited blood disorders.

Giving the example of thalassaemia, she estimates that it costs some RM3.5mil to treat a patient with regular blood transfusions and iron-chelating therapy for 30 years.

A haplo-identical bone marrow transplant costs approximately RM45,000 and will cure the patient.

The risk of dying from this procedure usually because of infections and GvHD during the period when the patient has no working immune system is estimated to be about 10%.

This is at the upper limit for standard bone marrow transplants, where the risk ranges from 5% to 10%.

She adds that studies have shown that the risk of severe GvHD is similar for haplo-identical transplants and sibling-matched transplants, which are both lower than transplants from an unrelated donor.

Although Yusuff is the first successful haplo-identical bone marrow transplant patient in the country, to the best of Prof Hanys knowledge, she believes that the procedure can be easily done in other major hospitals around the country.

The facilities are already there and specialists trained in bone marrow transplants need only learn the procedure once before they should be able to conduct it, she says.

So its not just having a big celebration to tell the world that we saved one boy with SCID, its having the ability to tell parents that there is always hope, as we can now do haplo-identical transplants in our centre, says Prof Hany.

It is about no longer having to tell parents that nothing more can be done for their terminally ill child.

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Sumitomo Dainippon buys cell therapy processing tech from Hitachi – In-PharmaTechnologist.com

By Dr. Matthew Watson

Sumitomo Dainippon Pharma Co Ltd has ordered cell culture technologies from Hitachi as part of its effort to develop a treatment for Parkinsons disease.

The order financial terms of which were not provided will see Hitachi supply automated cell culturing technologies designed for the manufacture of induced pluripotent stem cells (iPS).

Dainippon is developing a cell therapy for Parkinsons-related dopamine neuron loss and neurodegeneration in collaboration with both Hitachi and Center for iPS Cell Research and Application, Kyoto University (CiRA).

Part of the project which is funded by the Japanese Agency of Medical Research and Development (AMED) - involves the development of processing methods and technologies for the production of stem cells for regenerative therapies.

The Japanese drug firm has announced several regenerative medicine-based research projects in recent years, beginning in 2015 when it partnered with Sanbio to develop SB623, an allogenic cell therapy for ischemic stroke to improve motor abilities.

Regenerative meds

Regenerative medicine which engineers or replaces damaged cells within human patients has become a popular area of research in Japan sinceShinya Yamanaka won the 2012 Noel Prize for medicine for the discovery that mature cells can be reprogrammed to become pluripotent.

Regenerative medicine is also a big focus for the Japanese Government.

Laws introduced in November 2014 therevised pharmaceutical affairs law and newregenerative medicines legislation mean such products could be reviewed and approved in just two years, if deemed to be effective.

Japans Government further underlined its commitment to regenerative medicine in its budget in January 2015, allocating Y2.5bn ($20.8bn) to the industrialisation of regenerative medicine evaluation fundamental technology development business.

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Desperate mum’s race against time to fund treatment before she forgets her two little girls – Mirror.co.uk

By LizaAVILA

A desperate mum-of-two says she is losing her memory so fast she may soon be unable to recognise her young daughters.

Chantelle Fox was diagnosed with multiple sclerosis (MS) last May after suffering fatigue and "a little numbness" in her arm.

Since the devastating diagnosis, the 41-year-old's condition has quickly deteriorated, leaving her fearful for the future.

She has 79 lesions on her brain and her short-term memory is fading, meaning she often forgets where she has put things.

She also forgets promising her "beautiful" daughters, Lilly, five, and Edie, three, that she will take them somewhere special.

Her worst fear is that in just four years, she may not even remember the little girls at all.

Now, she faces a race against time to fund radical stem cell treatment in Russia - which she believes could halt the progress of her disease.

MS is a chronic condition, for which there is currently no cure.

The disease is caused by the immune system malfunctioning and mistakenly attacking nerve cells in the brain and spinal cord.

It can lead to patients suffering from a range of mild or severe symptoms.

In a bid to stop the progress of the "monster" condition, Chantelle, from Australia, plans to undergo surgery in Moscow.

MS Australia has deemed the treatment risky, while one neurologist told the mum there is no evidence it works and it could be dangerous.

However, another neurologist reportedly told her she would be a great candidate for a haematopoietic stem cell transplant (HSCT) trial.

But Chantelle, who lives in Melbourne, said waiting a long time for the chance to take part in a clinical trial in her home country wasn't an option.

Instead, she plans to travel abroad in June to undergo surgery.

I have two beautiful kids and I might not remember them in four years time if I dont go to Russia," she told the Herald Sun .

She added: I have to fight for my kids. I want to help them study, to see them married, to be a grandparent."

Chantelle has been accepted into an autologous haematopoietic stem cell transplant programme in Moscow.

She claimed the treatment has an "86 per cent success rate" in halting the progress of the neurological condition.

However, her family said the costs involved are "crippling".

They are trying to raise $150,000 (120,000) to cover the price of the treatment and transport to and from Russia.

Chantelle's sister, Maxine Parker, has set up dedicated GoFundMe and Facebook pages to help raise money for the surgery.

On the GoFundMe page, she describes how her younger sibling was "devastated" when she was diagnosed with MS.

She writes: "Chantelle was first diagnosed in May 2016. What started out as fatigue and a little numbness in her left arm, she put it down to just being tired from being a full time working mum of two young girls...

"A trip to her doctor one Sunday afternoon changed her and her family life forever. She was told to head straight to hospital, the dr believes she may have had a minor stroke.

"24 hours later, sitting in the hospital bed the neurologist suggests its either a brain tumour, motor neurone disease or MS and the only way to confirm is with a lumbar puncture and full MRI of her brain & spine.

"I will never forget sitting there holding my baby sister's hand as she lays on the bed with the nurse injecting a large needle into her spine to obtain spinal fluid. Almost an hour and half goes by and they confirm its been unsuccessful and they will need to try again.

"Next is the MRI and after two hours my sister returns to her hospital bed waiting for the news that will change her life forever."

She adds: "That neurologist returns to deliver the news, Chantelle you have multiple sclerosis... Chantelle is devastated, all she can think about is her two young girls and if she will be around to watch them grow up."

In a post on the page, Chantelle herself pays tribute to her sister, her husband Dara O'Donoghue and her two little girls.

Addressing Lilly and Edie, she writes: "You are the reason, I will never give up fighting this terrible disease, MS.

"You are my world and I will love you for eternity."

She also expresses her gratitude to her other relatives and friends.

It is estimated that around 100,000 people in the UK have MS.

HSCT involves the intravenous infusion of stem cells derived from peripheral blood, bone marrow or umbilical cord blood.

In autologous cases, the patient's own stem cells are used.

Their immune system is usually wiped out with chemotherapy treatment before it is regrown using their stem cells.

To visit Chantelle's GoFundMe page, click here .

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Desperate mum's race against time to fund treatment before she forgets her two little girls - Mirror.co.uk

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categoriaSpinal Cord Stem Cells commentoComments Off on Desperate mum’s race against time to fund treatment before she forgets her two little girls – Mirror.co.uk | dataApril 14th, 2017
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