For over 20 years autologous hematopoietic stem cell treatment (AHSCT) has been a therapy for autoimmune diseases such as multiple sclerosis, rheumatoid arthritis and lupus; however, the exact mechanism of action remains unclear. Recent clinical research has also been exploring the use of stem cell therapy for type 1 diabetes, another autoimmune disease which affects over 422 million individuals globally.

Type 1 diabetes, formerly known as juvenile or insulin-dependent diabetes, is a chronic condition where little or no insulin is produced by the pancreas. Immune cells attack pancreatic beta cells which produce insulin, leading to inflammation. Insulin is an essential hormone for energy production as it enables the breakdown of sugars to enter the cells and produce energy. The onset of type 1 diabetes occurs when significant inflammation damages beta cells and results in insufficient maintenance of glucose haemostasis (balance of insulin and glucagon to maintain blood glucose levels).

Therapies currently used in type 1 diabetes treatment include insulin administration, blood glucose monitoring and screening for common comorbidities and diabetes-related complications. However, these treatments fail to reduce the damage on a patients immune system. The use of autologous hematopoietic stem cells as a potential type 1 diabetes therapy is based upon the ability of the stem cells to reset the immune system. Autologous hematopoietic stems cells are retrieved from a patients own bone marrow or peripheral blood (blood which circulates the body and contains red blood cells, white blood cells and platelets) and after conditioning are injected intravenously.

A recent study by Ye and colleagues published in Stem Cell Research & Therapy (2017) investigated the effects AHSCT had on the immune response in recently diagnosed diabetes type 1 patients. The study included 18 patients (12-35 years old) with type 1 diabetes who had been diagnosed within less than 6 months. Of these 18 participants, 10 received a traditional insulin injection as treatment and eight received AHSCT. An additional 15 patients who matched in age, gender and BMI of these two groups were enrolled as a control group.

To test the effects of the treatment on immune response, patients peripheral blood cells were assessed. Samples were taken before they started treatment and then again 12 months after either the AHSCT or insulin-only therapies were administered.

Before treatment, peripheral blood cell distribution was almost equivalent in the two groups; however, after 12 months a significant difference was observed. The results of this clinical trial showed that patients receiving AHSCT exhibited significantly reduced development and function of Th1 and Th17 cells (types of T cells which cause inflammation in autoimmune diseases), compared to those only receiving the insulin treatment.

The inhibition of T-cell proliferation and function, along with decreased production of cytokines (pivotal chemical messengers which aid an immune response) observed in patients receiving AHSCT treatment suggests there is a strong link between the therapy and effects on the patients immune response. This may explain why AHSCT results in better therapeutic effects when compared with insulin-only traditional therapy.

The authors note that the small number of participants and length of the study are the two main limitations. Future clinical studies should include a larger number of patients and long-term follow up, especially since AHSCT can cause damage to the bone marrow and lead to potentially serious infections.

Progression of type 1 diabetes, as mentioned above, results in unavoidable immune damage from inflammation. This study suggests the combination of therapies including AHSCT treatment and high-dose immunosuppressive drugs may be a potential new therapeutic approach to type 1 diabetes. It is hypothesized that this combination has the ability reset the immune system and increase the recovery capacity of beta cells. Further clinical studies are essential though, to shed more light on the mechanism and use of stem cell therapy for type 1 diabetes.

Written By:Lacey Hizartzidis, PhD

Original post:
Stem Cell Therapy for Type 1 Diabetes - Medical News Bulletin

The calls had been coming for a few years, and Bill Holowaty couldn't say yes. His baseball spirit was willing. His body wasn't.

Holowaty won four national championships and 1,404 games before he stepped down in 2013 after 45 years as coach at Eastern Connecticut. Becoming president of the Greater Hartford Twilight Baseball League seemed perfect for a septuagenarian with baseball in his DNA, baseball in his blood.

The problem was this: Holowaty's DNA isn't the same. His blood type isn't the same.

That's what happens with Myelodysplastic Syndrome. That's what happens when your body that had carried you through the third most victories in Division III history no longer could make enough healthy blood cells. In short, Holowaty had bone marrow failure and needed a stem cell transplant last June 23 that changed his DNA and blood type from O to A. Otherwise, he wasn't going to be around for long.

"I'm celebrating my first birthday," Holowaty said recently. "June 23, my new birthday."

Fortunately, Type A loves baseball, too.

So Holowaty said yes this past winter to becoming president of the GHTBL, the amateur wood-bat league now in its 88th year. Over the decades, it is a league that has produced a large number of major leaguers, including 2017 Hall of Fame inductee Jeff Bagwell. It also is a league that has had to fight softball, other baseball leagues and the evolution of modern sports interest to keep its place on the map.

The first thing Holowaty did was bring together the managers for a couple of meetings at his house.

"I was extremely impressed with their enthusiasm and their desire to make the league better," Holowaty said. "I needed that. They motivated me. Look, I'm not going to change the world and make it the best league in the United States, etc. I told them I'll try to help. I just love to watch baseball and see it played the right way."

Holowaty, who played basketball at UConn, played for Wally Widholm on the playoff champion Hamilton Standard team in the summer of 1966. His sons played in the GHTBL, too.

"Wally taught me how to win, how to play the game of baseball," Holowaty said. "Later on, my son came to me and he said, 'Dad, I played in wood-bat leagues and played all over the place. I had my best experience playing for Gene Johnson this past summer.' Winning was important, not showing off. I loved that."

There was no way Holowaty could do this by himself. He surrounded himself with a strong executive committee that includes vice presidents Bill DePascale, Ed Slegeski and former UConn coach Andy Baylock.

"I've known Billy forever, since the '60s," said Baylock, who played two summers in the GHTBL. "He has had a lot health problems, but this is something he can put his heart into. He called and asked me to be a vice president. I said, 'Billy, will this make you happy if I join?' He said yes. I told him, 'I'll be with you.' Gene Johnson, who was such a mainstay in the league, died [in November 2014] and I felt this would be a good way to give back to the league and Gene."

The two state baseball legends obviously add recognition to the league. Yet it had to be more than that.

There is nothing worse, Holowaty said, than playing on a lousy field. Trinity College has a beautiful new facility. The league secured it for the playoffs. The teams are going to play throughout July 9 at Dunkin' Donuts Park. Holowaty, convinced the job of running a team is too big for one guy, wants each team to have a general manager. There were a couple of new teams added this year. There were sponsorships found. Holowaty also wants each team to have a mentor or two. On opening day, Holowaty and Baylock talked to the players about playing the game smart, aggressively, hustling, showing up on time. Little things that can become big things, like coaches wearing protective helmets at first and third base.

They've gone to games at various sites.

"Not to be a cop," Baylock said, "but to try to make sure things look good."

"We're not out there second-guessing managers," Holowaty said. "But a lot of great players have played in the league over nearly 90 years. I don't want a beer league. Baseball is one of the hardest games to teach and play. We've got a good league and want to make it better, a nice, competitive league where the guys enjoy themselves and learn the right way to play."

Those words came over the phone from Omaha a couple of weekends ago. He was out there for the College World Series. Holowaty is on the board of the American Baseball Coaches Association, its past president. This was a big trip for Holowaty.

"I couldn't go on an airplane for a year, or go out to eat," he said. "I had to wear a mask and gloves on the plane. The doctor told me I could go but have to be careful. My daughter [Jennifer] came with me to give my wife [Jan] four days' vacation.

"My wife has been taking care of me. Thank God for her."

In 2015, he was inducted into the National College Baseball Hall of Fame. It was in August of that year that Holowaty, after undergoing knee surgery, was told his blood cell counts had been dropping. He consulted a hematologist. He would have a bone marrow test late in 2015. Holowaty would need a stem cell transplant or else to use his words "I wasn't going to be around long, maybe a year." With plans to spend the winter in Florida, he would go to the Mayo Clinic in Jacksonville. There he began his treatment before returning to Connecticut.

A match in Germany, a young man, was found for Holowaty. On June 17, 2016, he went to the Dana-Farber/Brigham and Women's Cancer Center in Boston. For nearly a week he underwent chemotherapy for six hours a day to kill his old blood cells. The stem cells were flown overnight from Germany and the next day, June 23, Holowaty was receiving a transplant.

There would be more chemo. The fight has been hard. His immune system had to start from scratch. He must be ultra-careful to avoid germs, mold, etc., thus the gloves and the mask.

Holowaty went through his problems like he was reading a lineup card. He had pneumonia. A blood vessel broke when he had a lung biopsy. He had some blood clots in his legs and lung that took months to be rid of. His heart went out of rhythm. He had an aneurysm in his stomach. The man always was a tough coach and now, physically, mentally, spiritually, he has been called on to be even tougher.

Jan drives Bill up to Boston once or twice a week.

"They take my blood and see where I am with red and white blood cells," Holowaty said. "You get new blood. The remaining old blood tries to fight off the new blood.

"You feel good. You want to feel good. You just can't feel good. You go to bed, get a night's sleep and wake up tired. I'll feel great and then last week I had a hard time walking across the room. It's exhausting. It's not painful. I'm fighting it. I could never do this alone."

He has found a source of inspiration in his former ECSU assistant coach Ron Jones.

"Ron has had the same thing," Holowaty said. "He started calling me up and telling me how to prepare myself, helping me get through this. Here's the thing he has called me every day since last June. We just talked today. He has had a tough time. Last October, he had pacemaker put in, and he's doing well now.

"Think about that. He calls me every single day."

That's what great baseball guys do. They take care of each other.

Holwaty paused for a second on the phone.

"The Twilight League," he said softly, "this is my way of giving back to the game I love."

See the original post:
Jeff Jacobs: Hall Of Fame Coach Holowaty Fights Illness And Gives Back - Hartford Courant

LUCY Clarke was facing a downhill spiral when she flew to Russia to undergo a cutting edge stem cell transplant.

Two years on she says the procedure not only halted her illness in its tracks, but reversed much of the damage inflicted by multiple sclerosis.

The 41-year-old from Inverness is now backing crowdfunding efforts so that her friend and neighbour, Rona Tynan, can receive the same life-changing operation in Mexico before she becomes too ill to qualify.

Mrs Tynan, 50, has until the end of August to raise the 60,000 needed.

However, both are angry at a cross-border divide which means that a small number of MS patients in England can undergo the treatment for free on the NHS, while in Scotland despite having some of the highest rates of MS in the world the health service has refused patients' funding and no clinical trials are planned.

Mrs Clarke, a chemistry graduate and acupuncturist, began investigating AHSCT (autologous haematopoietic stem cell transplantation) in 2014 after her condition progressed from relapsing-remitting to secondary progressive MS. At the time her son was three and she feared ending up in a wheelchair.

Although the treatment has been available overseas for decades, it has never been routinely available on the NHS and is considered unproven by many neurologists.

It is also a highly aggressive therapy, using intensive chemotherapy to strip out sufferers faulty immune systems before replenishing it with stem cells harvested from their own bone marrow or donor tissue. Despite the risks, many patients including Mrs Clarke credit it with transforming their lives.

She underwent the procedure in Moscow over a period of four weeks in April and May 2015. She said: From when my son was three to when I had the transplant, my walking had deteriorated, I needed to use a walking stick all the time, I had very poor balance, debilitating fatigue, I had brain fog, I used to slur my words.

"Im left-handed and my left hand was really weak so my writing was bad. Other things would come and go numbness in my legs, tingling, cramps in my calves, sore and painful legs. The majority of them have gone since the transplant.

I noticed quite quick improvements in things like balance. The biggest thing is not really having fatigue, and the brain fog completely went. I stopped slurring my words quite quickly after treatment. I was more alert. I had more concentration, more focus. Within six months the shaking in my left arm had gone. Ive still got drop foot in my right leg and I still use a walking stick, but once youve got to the stage of secondary progressive it all gets a bit scary. Things are going downhill and youre told theres nothing that can be done, so really my goal from treatment was just to halt the progression to know I wasnt getting any worse. Thankfully, and luckily, I have seen lots of benefits.

Eighteen months on, MRI brain scans show no signs of disease progression and while Mrs Clarke stresses that the treatment is neither a magic bullet nor a walk in the park, she is supporting Rona Tynans bid to undergo the same surgery in October.

Mrs Tynan, a retired Metropolitan police sergeant and mother-of-two from Inverness, also has secondary progressive MS. She is already in a wheelchair and fears that unless she undergoes the treatment soon she will become too ill. She said: Im a 7.5 out of 10 on the disease progression scale, where 10 is death. Most clinics stop taking you at seven, but Mexico just raised it to 8.5. Thats brilliant for people like myself, but I cant afford to get any more ill.

So far, Mrs Tynans fundraising page on JustGiving has raised nearly 4000, but she is frustrated that more is not being done to help Scottish patients. In England, clinical trials are ongoing in London and Sheffield but a small number of patients with relapsing-remitting MS can be referred for the treatment off-trial, for free, on the NHS. In Scotland, however, eligible patients have been turned down for NHS funding.

Mrs Tynan said: It seems crazy to me that Brits are going to Chicago and Mexico and Russia for a treatment that in the long-run could save the NHS loads of money. Scotland is one of the worst places in the world for MS yet in England you can get this treatment for free. Why arent we fighting in Scotland to get this?

Mrs Clarke added: Its very unfair. It just seems a no brainer to me why they wouldnt make it available not for all patients but for some. The Scottish Government said referral decisions were "for clinicians".

A spokesman said: "Whilst the vast majority of healthcare provided by NHS Scotland is delivered in Scotland, NHS boards can commission treatment in other countries on an ad hoc basis, particularly where highly specialised treatment is involved. Decisions to refer patients are for clinicians, based on agreed guidelines, which ensure best practice, equity of access and consistency of treatment for all patients.

"HSCT is not currently widely available anywhere on the NHS, but people from Scotland can participate in trials held in other centres across the UK, where clinically determined appropriate and beneficial."

More:
Anger as Scots patients miss out on 'breakthrough' stem cell therapy offered by NHS England - Herald Scotland

When 14-year-old Aarav Gulati (name changed on request) met with an accident a couple of years ago while playing football, he injured his knee. A portion of the cartilage was damaged, and doctors used turned to a radical new procedure for a solution. They took Gulatis own cells, grew them in a lab and used them to replace the cartilage and repair the damage in a natural way.

He was an ideal case for the use of regenerative cell therapy that was a fairly new phenomenon in orthopaedic treatment in India, says Dr Yash Gulati, senior joint replacement and spine surgeon, New Delhis Indraprastha Apollo Hospital.

The regenerative cell therapy got US FDA approval this year, and the Apollo group partnered with RMS REGROW, a company that specialises in cell therapy technology, to exclusively offer the treatment to patients in India.

Instead of using artificial implants, the technique helps in healing the bone or cartilage damage in a natural way using a persons own cells to regain normal function. Cultured cells (grown in a lab) are injected into a patient to replace diseased or dysfunctional cells.

Instead of using artificial implants, the technique helps in healing the bone or cartilage damage in a natural way using a persons own cells to regain normal function. Cultured cells (grown in a lab) are injected into a patient to replace diseased or dysfunctional cells. (Illustration: Siddhant Jumde)

A small part of the joint cartilage is taken through a keyhole procedure, and is grown in a special manner to convert it into stem cells in the laboratory, says Dr Gulati. This is then applied on the area showing loss of joint cartilage.

Dr Gulati has so far treated 10 people using this therapy in Apollo, Delhi.

Stem cells lead to growth of joint cartilage in defective areas, and normal new cartilage re-grows. In bone damage, bone marrow cells are harvested, cultured and implanted in the area to be able to re-grow in a healthy way, Dr Gulati says.

In Mumbai, those in need of joint replacement because of injury, wear and tear or other lifestyle and ageing, are also realising that new cartilage can be grown in a lab from ones own cells and used instead of artificial materials.

Chondron or cartilage cell therapy is a patient-specific regenerative medical treatment which naturally regrows original cartilage. The therapy is used for repairing articular cartilage of the knee, ankle and shoulder joints and to help replace missing areas of cartilage.

This is a process where a biopsy of cartilage cells (chondrocytes) is taken from the patients knee, ankle or shoulder, says Satyen Sanghavi, chief scientific officer of Regenerative Medical Services Regrow, a biotechnology company in Mumbai.

Causes of bone or cartilage damage

They are then cultured to grow and multiply in a lab for 3-4 weeks into a surplus population of several million. The cultured cells are then re-implanted in the damaged area in a minimally invasive surgical procedure.

The process comes from eight years of work in cell and tissue therapy research. Chondron ACI is the countrys first cell therapy product.

These cells grow and repair tissue with properties similar to that of normal cartilage present in other joints, says Sanghavi. But replacement alone doesnt solve problems. Patients are expected to follow a rehabilitation program, to help the body adjust to new cells and them get back to day-to-day physical.

Its easy to see the advantages of a process like this. Experts say it may avoid the need for future prosthetic joints replacement (especially partial joint replacement) and allows patients the freedom to continue physical activities as before.

It also poses less risk of disease transmission or infection since it comes from the patients own tissue (no foreign material or metal goes inside the body). It may also halt further progression to osteoarthritis, a common problem with those in need of joint replacement.

The procedure costs Rs.3 to 3.5 lakhs.

In India, more than 500 patients have been treated with both bone and cartilage cell therapy procedures, says Sanghavi.

There is a success rate of more than 95%. During our clinical trials and research, we have treated working professionals, housewives, athletes, army men and mountaineers. Almost all of them have successfully recovered and got back to their active life.

However, this new technique has a flip side, too.

The price could be a bit steep for some because stem cell treatment is expensive; and the treatment gets prolonged as a patient has to wait for some time as cell culture takes time and one cannot bear weight on the affected area while the healing is on. Also, not all patients are suitable for it because it can correct only if damage isnt extensive, says Dr Ankit Goyal, associate professor, Safdarjung Sports Injury Centre (SIC) in Delhi.

Safdarjung Hospital had also treated about 35 patients, who had damaged their cartilage, with the technique a few years ago.

We would send cartilage for culture but only in cases where damage was limited. This is definitely not a substitute for knee or hip replacement procedure where the entire joint is extensively damaged. However, it may prevent the need for replacement later on in life, especially in young patients, he says.

Read more here:
Damaged bone or cartilage? Now, grow your own cells in a lab - Hindustan Times

Updated: Jul 1, 2017 - 6:08 AM

BOSTON - Its been nearly four years since a bone marrow donation saved Mandy Manocchios life, but the anticipation of the last few hours before she met the donor seemed like they took forever.

"When you hear that you have less than a year to live and your life's basically laying on the line and if I didn't find a donor it would've been catastrophic, but she's my angel, she said.

At Brigham and Women's Hospital in November 2013, Mandy had a bone marrow transplant to treat acute myeloid leukemia - on Friday night at The Harp in Boston she finally got to meet Magdelena Kruger, the woman who saved her life.

"She allowed me to watch my children grow up and have another, Mandy said.

Kruger had just landed after an 11-hour flight from Germany. When the two women saw each other there was no need for words.

Stem cells from Kruger were carried 4,000 miles from Germany. Through a translator, she said it was the first time shed ever donated.

I just wanted to help somebody who's sick and needs help, she said.

Now both women are advocates for bone marrow donations. They say the process is relatively simple and life-changing on both ends.

"It's so rewarding to see that immediate result of how you can help somebody, Kruger said.

To learn more about bone marrow donation or to register as a bone marrow donor, please visit dkms.org.

2017 Cox Media Group.

Read the original post:
Embrace expresses more than words for marrow donor who saved woman's life - My Fox Boston

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Gosselies, Belgium,26 June 2017; 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the bone cell therapy company addressing high unmet medical needs in orthopaedics and bone diseases, today announces that the European Patent Office (EPO) has notified the Company of its intention to grant a key patent covering its first-in-class allogeneic cell therapy technology.

Once granted, the patent titled, Osteogenic differentiation of bone marrow stem cells and mesenchymal stem cells using a combination of growth factors, will provide legal protection to Bone Therapeutics both for the manufacturing methods and for the distinct cell type used in its allogeneic cell therapy technology. Specifically, the patent covers methods to manufacture differentiated and biologically active osteoblastic (bone-forming) cells from bone marrow stem cells, using a specific combination of growth factors, and also covers a new class of osteoblastic cells suitable for allogeneic administration to the patient.

Bone Therapeutics will now validate the patent in several countries in the European Union, potentially allowing IP protection for its allogeneic bone cell therapy platform until 2029. Patents from the same patent family have already been granted in Japan, Australia and Singapore and applications are pending in the USA, Canada, India and South Korea. ALLOB, Bone Therapeutics most advanced allogeneic bone cell therapy product, is currently being evaluated in Phase I/IIA clinical trials for delayed-union fractures and spinal fusion, for which interim results are expected in the third quarter this year.

Thomas Lienard, Chief Executive Officer of Bone Therapeutics, commented: This notice from the European Patent Office confirms our allogeneic bone cell therapy technology is both innovative and distinctive. When granted, this European patent will significantly strengthen our IP position in the field of bone cell therapy, giving us further validation for the scientific and commercial development of our cell therapy products whilst also enhancing our position with respect to new partnerships.

Dr. Miguel Forte, Chief Medical Officer of Bone Therapeutics, further noted: Obtaining this patent is an important step in the development of our allogeneic bone cell therapy technology. It will provide a solid IP protection for our current work and for future technological advances, allowing us to continue our efforts to create patient-centric and commercially interesting bone cell therapy solutions.

About Bone Therapeutics

Bone Therapeutics is a leading cell therapy company addressing high unmet needs in orthopaedics and bone diseases. Based in Gosselies, Belgium, the Company has a broad, diversified portfolio of bone cell therapy products in clinical development across a number of disease areas targeting markets with large unmet medical needs and limited innovation. Our technology is based on a unique, proprietary approach to bone regeneration which turns undifferentiated stem cells into osteoblastic, or bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery. Our primary clinical focus is ALLOB, an allogeneic off-the-shelf cell therapy product derived from stem cells of healthy donors, which is in Phase II studies for the treatment of delayed-union fractures and spinal fusion. The Company also has an autologous bone cell therapy product, PREOB, obtained from patients own bone marrow and currently in Phase III development for osteonecrosis and non-union fractures.

Bone Therapeutics cell therapy products are manufactured to the highest GMP standards and are protected by a rich IP estate coveringnine patent families. Further information is available at: http://www.bonetherapeutics.com.

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

Josh Sandberg has been an executive search consultant focused exclusively on orthopedic and spine start-ups since 2004. He has had a tremendous impact in helping his clients avoid costly hiring mistakes by his deep industry knowledge and network. In 2010, Josh co-founded Ortho Spine Companies, which is the parent company of Ortho Spine Distributors (OSD), Surg.io and Ortho Sales Partners (OSP). OSD a searchable database that helps ease the frustration of finding orthopedic distributors throughout the country. Surg.io is the ultimate distributor toolkit that offers distributors the tools necessary to build the foundation of a scalable and highly functioning sales organization. OSP is an end-to-end solution that helps companies approach the Global Market in a cost efficient way. Our team has hundreds of years of experience and can help you navigate the many challenges present in bringing new technologies to the market.

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Bone Therapeutics receives Intent to Grant Notice from European Patent Office for allogeneic bone cell therapy platform - OrthoSpineNews

Oneyoung Weymouth man is climbing Mount Snowdon on his 21st birthday to help in the fight against cancer.

Paul Cashman-Roberts, 20, is hiking up the mountain on Friday, September 8, to raise money for the Anthony Nolan charity, in support of his aunt and his cousin.

His cousin, Avril Bailey, was diagnosed with breast cancer and had to have one of her breasts removed, while his aunt, Terina Cashman-Stimpson, still suffers from several forms of cancer one of which is blood cancer.

Anthony Nolan works to deliver lifesaving stem cell and bone marrow transplants to those with blood cancer or other blood disorders and conducts vital research into making these transplants more effective.

Paul expressed admiration for both his cousin and his aunt and said he wanted to do something in support of their struggle.

He said: I thought I would do something to raise money for experts who are going to do some fantastic research in the field. I did the Duke of Edinburgh while at Wey Valley School and I love walking my dog all the time.

Paul said that he specifically chose Mount Snowdon to walk up partly because it was more accessible than Ben Nevis.

When he announced he was going to be doing the climb up Snowdon last Thursday Paul received an amazing response from people.

He said: Within the first 24 hours we managed to raise 120.

Initially it was hoped the climb would raise up to 1,000.

Paul added: However, I have been in contact with the guys at Ayya nightclub in Weymouth and we are going to be holding a mini-music festival there to raise even more money.

The young hiker is hoping this will help him to go beyond the 1,000 mark. Both Pauls family and the people at Anthony Nolan have been extremely supportive of him and he is looking forward to his climb up the tallest mountain in England and Wales.

He said: I could not be more thrilled. Really, the end result is about how much we can raise and about saving peoples lives that is really what its all about.

To donate search for Pauls name at http://www.justgiving.com

Read more:
Paul from Weymouth to climb up Snowdon to help fight cancer - Dorset Echo

Free Press Lions beat writer Dave Birkett answers your Twitter questions in a video mailbag June 26, 2017, before summer vacation.

Detroit Lions linebacker Paul Worrilow with daughters Juliet, left, and Rowan, right.(Photo: Paul Worrilow)

Paul Worrilow has a great back story.

The veteran linebacker, who signed with the Lions in March, was undrafted out of Delaware.

He made the Atlanta Falcons, against all odds.

Cracked the starting lineup, against all odds.

Became the teams leading tackler, against all odds.

Stuck around for four seasons, against all odds.

But theres more.

Its a story about being selfless and thinking about others. Its a story that should be repeated, if only to inspire others to follow his lead.

More Lions news:

Detroit Lions CBs coach: Interceptions 'will come' for Darius Slay

And it started with a simple cheek swab.

Its so simple, Worrilow said.

When Worrilow was a sophomore at Delaware, he joined the Be The Match Foundation Registry, hoping to become a bone marrow donor.

Four months later, he was matched with a 23-year-old woman with leukemia.

Worrilow donated peripheral blood stem cells to the woman, although he doesnt know what happened to her. He doesnt know her name. He never has met her.

Its so simple, Worrilow said. They do a cheek swab. You get put in the database. If you match somebody, there are two ways to do it. You can donate actual bone marrow or do it like I did, peripheral blood stem cells.

Lions linebacker Paul Worrilow takes part in OTAs on Wednesday, May 24, 2017 at the Allen Park practice facility.(Photo: Kirthmon F. Dozier, Detroit Free Press)

About one in 40 registry members will be called for additional testing.

About one in 300 will be selected as the best possible donor for a patient.

And about one in 430 on the registry go on to donate bone marrow or peripheral blood stem cells.

Odds are, you never will be asked to donate.

But you just might give somebody hope.

Its so simple, Worrilow said. Its not painful. Its a small part of your time, to have a great impact, a tremendous impact on another person and their family. Its a no-brainer. You can have a great impact at such a small cost to yourself.

Worrilow is on a mission to let people know about it: The cool part about it is being able to share it (with people) who are ignorant to the process and their ability to help other people. Encouraging people. People who just dont know what Be The Match Foundation is. Or how you can help people with blood cancers, or how you can join.

Worrilow signed with the Lions during the off-season.

This team is tremendous, Worrilow said. The family-oriented vibe here. From the head coach, from the ownership on down, you can feel it. Its a family vibe. They look after you, care for you.

In June, during minicamp, Worrilow played weak-side linebacker. But he can also play in the middle.

Its going good, he said. There is a lot of competition. The team is great that way, pushing each other. Any action I can get on the field is exciting. When youre winning games, and everyones in here practicing hard, its just awesome.

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Worrilow has a track record for good tackling, but he has been criticized for his inability to cover.

If (the criticism) is there, its probably there for a reason, he said. Criticism, if it doesnt come from a bad place, it is probably warranted. And thats something I have to improve on. Thats with all parts of my game. I dont feel like I have played my best football yet.

Worrilow said he is adjusting to the Lions defense, making defensive calls.

Compared to the other places Ive played, there is a bigger volume of calls, he said. Thats something I like. Both linebackers do it. One guy has the indicator, but if you are not out there talking, you arent going to be out there.

And now, as he takes time off before training camp, he feels confident about himself and this team.

I dont feel like I could be in a better place, life-wise, work-wise, everything, he said. Its an encouraging place to come in and work every day. The linebacker group is awesome. Its young. Its competitive. Thats what you want.

So, this guy keeps breaking the odds.

Sticking in the NFL. And trying to use that platform to help others.

Trying to raise awareness.

Trying to encourage people to make a difference.

Trying to break the odds.

Help us, Detroit Lions: You are Detroit sports fans' only hope right now

For new Detroit Lions LB Paul Worrilow, dad duties come first

Contact Jeff Seidel: jseidel@freepress.com. Follow him on Twitter @seideljeff. To read his recent columns, go to freep.com/sports/jeff-seidel/.

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Detroit Lions' Paul Worrilow has beaten odds, inspires others to help - Detroit Free Press

Fiona Geekie, a nurse from Croydon who is apractitioner for TheCentral London Community Healthcare NHS Trust, donated bone marrow to a person she had never met earlier this year.

According to Fiona (49), about 2,000 people in the UK require a bone marrow or stem cell transplant every year.

For most of them its their last chance of surviving blood cancer, she added.

The nurse, who is a member of the Merton enhanced rapid intervention team,described why she went on the register to donate through the Anthony Nolan Trust 15 years ago.

Three quarters cant find a matching donor in their family so rely upon the generosity of a complete stranger already on the bone marrow register. So I joined up, it was simple.

Last Christmas Fiona was called for blood tests as she was a possible match for someone.

I was surprised to be called in the first place and I must confess I was a little apprehensive, Fiona admitted.

However, she continued: But then I thought what if someone in my family needed a bone marrow transplant. Its the most fantastic gift you can give to total stranger.

After donating, Fiona said she felt quite emotional and wanted the recipient to get well again.

She concluded: Overall, it was a wonderful experience and I have no hesitation in recommending all young people aged 16-30 to consider joining the bone marrow donor register.

See the article here:
Nurse goes the extra mile and donates bone marrow to a complete stranger through the Anthony Nolan Trust - Sutton Guardian

Honghao Zhang,1,2 Satoshi Komasa,1 Chiho Mashimo,3 Tohru Sekino,4 Joji Okazaki1

1Department of Removable Prosthodontics and Occlusion, Osaka Dental University, Hirakata, Osaka, Japan; 2Department of Stomatology, Nanfang Hospital and College of Stomatology, Southern Medical University, Guangzhou, Guangdong, China; 3Department of Bacteriology, Osaka Dental University, Hirakata, 4The Institute of Scientific and Industrial Research, Osaka University, Suita, Osaka, Japan

Purpose: Alkali-treated titanium with nanonetwork structures (TNS) possesses good osteogenic activity; however, the resistance of this material to bacterial contamination remains inadequate. As such, TNS implants are prone to postoperative infection. In this work, we attempted to alter the biological properties of TNS by treatment with short-duration high-intensity ultraviolet (UV) irradiation. Methods: TNS discs were treated with UV light (wavelength =254 nm, strength =100 mW/cm2) for 15 minutes using a UV-irradiation machine. We carried out a surface characterization and evaluated the discs for bacterial film formation, protein adsorption, and osteogenic features. Results: The superhydrophilicity and surface hydrocarbon elimination exhibited by the treated material (UV-treated titanium with a nanonetwork structure [UV-TNS]) revealed that this treatment effectively changed the surface characteristics of TNS. Notably, UV-TNS also showed reduced colonization by Actinomyces oris during an initial attachment period and inhibition of biofilm formation for up to 6 hours. Moreover, compared to conventional TNS, UV-TNS showed superior osteogenic activity as indicated by increased levels of adhesion, proliferation, alkaline phosphatase activity, osteogenic factor production, and osteogenesis-related gene expression by rat bone marrow mesenchymal stem cells (rBMMSCs). This inverse relationship between bacterial attachment and cell adhesion could be due to the presence of electronhole pairs induced by high-intensity UV treatment. Conclusion: We suggest that simple UV treatment has great clinical potential for TNS implants, as it promotes the osseointegration of the TNS while reducing bacterial contamination, and can be conducted chair-side immediately prior to implantation.

Keywords: implant, nanonetwork, postoperative infection, UV treatment, superhydrophilicity, osteointegration

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License. By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Effect of ultraviolet treatment on bacterial attachment and osteogenic activity to alkali-treated titanium with ... - Dove Medical Press

AVERY Beal has seen more suffering in her three short years than many people see in a lifetime.

The plucky toddler was diagnosed with Acute Lymphoblastic Leukaemia in August 2014 and has been fighting for her life ever since.

In the last three years, she has lived through chemotherapy, stem cell transplants, and bone marrow transplants.

It's been a rough few years on the rest of the family too.

Avery's mum Jen has spent the last two and a half years living in between the Beal family home on the Sunshine Coast and Lady Cilento's Children Hospital in Brisbane to care for Avery's medical needs.

Dad David has cared for the couple's other five children on the Coast, working to support them while home schooling their autistic twins.

To top it all off, Mr Beal said the family was recently given no choice but to move house after their lease ended.

Despite the tumultuous last few years, the ordeal might finally be over for Avery - although she's still very high risk, doctors have deemed her well enough to come home.

"Avery's been doing really good," Mr Beal said.

"She managed to get transplant stem cells from a baby's (umbilical) cord from another country.

"She managed to get to day 100 after the transplant, which at that stage doctors were happy for Jen and Avery to come home."

Avery had a second bone marrow transplant in March after her first one failed to stimulate Avery's blood cells to create healthy cells instead of cancerous ones.

"We're feeling good but the challenge is that she's still so high risk," Mr Beal said.

"With children, they'll generally speaking only do two bone marrow transplants.

"If it does come back there is literally nothing they can do. They would just make her comfortable.

"At the moment, we have tests done on her bone marrow every month so see that she's still cancer free."

Yesterday Avery had her central line - a long, thin, flexible tube used to give medicines, fluids, nutrients, or blood transfusions -removed for the first time since her diagnosis in 2014.

The bubbly three-year-old will finally be able to go swimming - an experience that Avery has missed out on living on the Sunshine Coast.

Although the spritely tot has an 85% chance of relapsing, the family are confident that this is a good sign.

Despite all life has thrown at her, Avery is a happy child that lights up the lives of those around her.

"She's just incredible," Mr Beal said.

"It just amazes me over and over again how amazing she is on top of the treatments and the drugs; I think she's on eight different meds every morning and evening."

Mr Beal said the biggest hurdle the family currently face is the cost of Avery's multiple medications.

"She had a number on different things to get her body to a place for the transplant she's had in march," he said.

"Since then she's been on lots of different meds to make sure her body doesn't reject the transplant.

"For us finances are the biggest thing.

"Now that Avery is out of hospital we have to pay for medication and Jen's still having to do trips to Brisbane every week or fortnight"

To help the Beals visit http://www.facebook.com/averysupport.

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Glimmer of hope in Avery's heartbreaking cancer battle - The Sunshine Coast Daily

Today the Charlotte Lozier Institute announced the release of its latest testimonial video at StemCellResearchFacts.org, a project of the Washington, D.C.-based research and policy group. The video revisits Jackie Stollfus, a lupus survivor whose story was first told in a video released in 2014.

Diagnosed at the age of 21 with systemic lupus, an autoimmune disease with no known cure, Stollfus endured years of debilitating symptoms that did not respond to medication before undergoing a transplant of her own bone marrow stem cells. Seven years later, she is healthy, active, and has been able to start a family. Adult stem cells saved my life, gave me a chance to have a life, gave me that chance to be a mom, she says.

Dr. David Prentice, Vice President and Research Director of the Charlotte Lozier Institute and an international expert on stem cells, hailed the new video, saying:

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Autoimmune diseases are notoriously challenging to treat, which makes Jackie Stollfuss recovery that much more striking. As this video shows, adult stem cells are the gold standard for stem cells when it comes to patient-centered science. Jackies story is only the latest example of innovation using adult stem cells. These non-controversial cells have led to validated healing in FDA-approved studies and peer-reviewed publications for patients with various diseases and conditions. Derived from bone marrow, umbilical cord blood, and other ethical sources, they have already been used to help over one million suffering patients around the globe.

Charlotte Lozier Institute President Chuck Donovan praised Congressional efforts to prioritize NIH funding for the most promising research:

The initial successes for these innovative therapies must be followed up with expanded resources to bring more treatments to the clinic and the bedside. The bipartisan, aptly-named Patients First Act (H.R. 2918) introduced by Rep. Jim Banks and Rep. Dan Lipinski is a good example of how policymakers can advance cutting-edge medicine. It directs resources for stem cells where they will do the most good for patients.

StemCellResearchFacts.org, a project of the Charlotte Lozier Institute, was established in 2009 to facilitate and form a worldwide community dedicated to helping individuals, patients and families discover, learn and share the latest advances in adult stem cell research. To that end, the website has published 16 video testimonials backed by peer-reviewed published science. These testimonials feature patients who have undergone successful therapies for a variety of conditions including autoimmune diseases, cancer, spinal cord injury, heart disease, and more using adult stem cells. They also convey the testimony of doctors and researchers on the merits of these treatments.

Continued here:
Adult Stem Cells Save Woman Ravaged by Lupus, Now She Can be a Mom - LifeNews.com

A Winnipeg toddlerwith acute myeloid leukemia has died after hundreds came forward to register as donors in an effort to help him.

After being diagnosed with the disease on Oct. 25, 20-month-old Tegveer Minhaswent through two rounds of chemotherapy, losing his hair and a lot of weight.

During that time, his family put out calls to the public to come forward and register bone marrow and stem cell information, in hopes that someone would be a match.

Hundreds of people in Manitoba, Ontario and Alberta were swabbed, and Tegveer got a stem cell donation, but his dad said it didn't work.

"After eight months of struggle, he passed away on June 18, early in the morning at 6 a.m.," said his dad,Sukhbir Minhas.

Minhas saidhis family is trying to stay strong, but they are having a hard time.

"He was a happy soul. He loved to go out. We took him to Clear Lake on June 4, and I wish I knew that he would love it so much. We were planning to go back again," Minhas said.

The hundreds of strangers who registered as donors, prayed for and even phoned the family to offer supportmeant the world to Minhas and his wife, he said.

"There was a time in the hospital, it was in January I think, we were so sure that my son's going to be all right, because it's just not me and my wife, it's thousands of other people who are praying for him," he said.

He urged people, especially young people aged 18 to 35, to register as donors to help other families.

"I respect every person from the bottom of my heart who went and got themselves swabbed, and even those who just had a thought of going and get themselves swabbed. That means they care for my son as I and my wife," he said.

"It feels a little better than if there was nobody for us."

Original post:
'He was a fighter,' says father after toddler dies of leukemia - CBC.ca

Regulatory News:

Cellectis (Alternext: ALCLS; Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited CAR T-cells (UCART), announced today the first administration in the Phase I clinical study in Acute Myeloid Leukemia (AML) for its investigational product UCART123, one of the Companys wholly-controlled TALEN gene-edited product candidates. This marks the first allogeneic, "off-the-shelf gene-edited CAR T-cell product candidate targeting CD123 to be investigated in clinical trials.

This clinical research in AML is led by Principal Investigator Dr. Gail J. Roboz, Professor of Medicine at Weill Cornell Medicine and Director of the Clinical and Translational Leukemia Programs at Weill Cornell Medicine and NewYork-Presbyterian Hospital.

The clinical trial will investigate the safety and efficacy of UCART123 in patients with AML. AML is a devastating clonal hematopoietic stem cell neoplasm which is characterized by uncontrolled proliferation and accumulation of leukemic blasts in bone marrow, peripheral blood and, occasionally, in other tissues. These cells disrupt normal hematopoiesis and rapidly cause bone marrow failure. In the U.S., there are an estimated 19,950 new AML cases per year, with 10,430 estimated deaths per year. While complete response rates can be as high as 80 percent in younger patients who undergo initial induction cytotoxic chemotherapy, the majority of AML patients relapse and die from the disease. AML patients with high-risk genetic features have an especially urgent unmet medical need, as their outcomes are dismal with all existing treatment modalities, including allogeneic stem cell transplantation.

"After being granted rapid approval from Regulatory Authorities and Institutional Review Boards to initiate UCART123 studies, the enrollment and treatment of the first patient represents a major milestone for Cellectis, and we are eager to hit the ground running with the recruitment of our first patient for our second UCART123 Phase I study in BPDCN soon, said Dr. Loan Hoang-Sayag, Cellectis Chief Medical Officer. "This first program targeting CD123 will be a paradigm shift for our Company, as it will provide a wealth of valuable additional knowledge and data to drive our gene-edited allogeneic CAR T-cell platform.

"We are excited to be enrolling our first patient with UCART123 and are hopeful that this novel immunotherapy modality will prove to be a significant and effective weapon against AML, said Dr. Roboz.

The clinical trial is part of a strategic translational research alliance that was formed between Cellectis and Weill Cornell Medicine in 2015. Dr. Monica Guzman, an associate professor of pharmacology in medicine at Weill Cornell Medicine, is co-principal investigator whose work focuses on preclinical and early-stage testing to optimize the development of stem cell-targeted cancer drugs.

About Cellectis

Cellectis is a clinical-stage biopharmaceutical company focused on developing a new generation of cancer immunotherapies based on gene-edited T-cells (UCART). By capitalizing on its 17 years of expertise in gene editing built on its flagship TALEN technology and pioneering electroporation system PulseAgile Cellectis uses the power of the immune system to target and eradicate cancer cells.

Using its life-science-focused, pioneering genome engineering technologies, Cellectis goal is to create innovative products in multiple fields and with various target markets.

Cellectis is listed on the Nasdaq market (ticker: CLLS) and on the NYSE Alternext market (ticker: ALCLS). To find out more about us, visit our website: http://www.cellectis.com

Talking about gene editing? We do it. TALEN is a registered trademark owned by the Cellectis Group.

Disclaimer

This press release contains "forward-looking statements that are based on our managements current expectations and assumptions and on information currently available to management. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. The risks and uncertainties include, but are not limited to, the risk that the preliminary results from our product candidates will not continue or be repeated, the risk of not maintaining regulatory approval to pursue UCART123 clinical trials, the risk of not obtaining regulatory approvals to commence clinical studies on UCART123 in other countries or on other UCART product candidates, the risk that any one or more of our product candidates will not be successfully developed and commercialized. Further information on the risks factors that may affect company business and financial performance, is included in filings Cellectis makes with the Security Exchange Commission from time to time and its financial reports. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

View source version on businesswire.com: http://www.businesswire.com/news/home/20170627006309/en/

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First in Human Administration of UCART123 in Cellectis' AML Phase I Clinical Trial at Weill Cornell Medicine ... - Markets Insider

Ottawa Sun

Jonathan Pitre still ailing as doctors search for answers Ottawa Sun Pitre checked back into hospital earlier this month just three days after being released following a stem cell transplant that had successfully taken root in his bone marrow. Bone marrow stem cells produce most of the body's blood cells, and are ... and more »

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Jonathan Pitre still ailing as doctors search for answers - Ottawa Sun

Photo of Jonathan Pitre and his mother, Tina Boileau, taken in Minnesota. Tina Boileau / -

Doctors in a Minnesota hospital continue to search for answers to a mysterious infection that has left Jonathan Pitre feverish, nauseated and short of breath.

Pitre, 17, of Russell, has been in the University of Minnesota Masonic Childrens Hospital for the past two weeks, suffering from an array of complications more two months after his stem cell transplant. Doctors are also trying to adjust his medications to better deal with his increased pain levels.

Hes having a tough run, said his mother, Tina Boileau, and I really dont know when it will get better.

The teenager suffers from a severe form of epidermolysis bullosa (EB), a painful and progressive skin disease that has left deep, open wounds on his body.

Last week, Pitres face and neck became swollen in response to what doctors believed was some kind of viral infection. That swelling has been brought under control, but a battery of tests has yet to reveal the source of the infection, which continues to cause problems.

Pitres breathing is laboured and hes running a high-grade fever of about 104 F (40 C); he has also developed bleeding and painful sores in his mouth.

We still have no idea what were dealing with, said Boileau. Its frustrating because Im at the point where it would be nice to see that all that Jonathan has gone through has been worth it.

Doctors are monitoring Pitre for graft-versus-host-disease (GVHD), but all of his tests have so far been inconclusive.Anyone who receives stem cells from another person is at risk of developing GVHD, a condition in which the donors white blood cells turn on the patients own tissues and attack them as foreign. It can range from mild to life-threatening.

About one-third of the almost 50 EB patients who have had a stem cell transplant at the Masonic Childrens Hospital have experienced the condition.

Pitre checked back into hospital earlier this month just three days after being released following a stem cell transplant that had successfully taken root in his bone marrow. Bone marrow stem cells produce most of the bodys blood cells, and are responsible for arming its immune system.

Pitre has been in Minnesota since mid-February to undergo the transplant, his second. The first ended in disappointment on Thanksgiving Day last year.

Tests show Pitres latest transplant remains fully engrafted, and there are signs that it has started to improve the condition of his skin.

Originally posted here:
Jonathan Pitre still ailing as doctors search for answers - Ottawa Citizen

Richard Sennott, Star Tribune file During a family portrait in 2000, Molly Nash gives her 4-week-old brother, Adam, a kiss. Molly Nash received some umbilical blood from her brother, saving her from a fatal genetic disease.

Adam Nash was dubbed Little Frankenstein by the New York Post in 2000 because he was conceived via in vitro fertilization specifically so doctors at the University of Minnesota could collect stem cells from his umbilical cord blood to save his sister, Molly.

Today, back home in Colorado, Adam has a drivers license and helps disabled children ski. His sister once weeks from death due to a condition called Fanconi anemia is debating whether to focus on oceanography or graphic design in college. And IVF to produce an ideal child for a siblings stem cell transplant is common, albeit with lingering ethics concerns.

A squirrelly trio of teens is vindication for Adams mother, Lisa Nash, who felt the weight of the ethical questions when the Us Dr. John Wagner suggested IVF in 1995.

View post:
'Little Frankenstein,' conceived so Minnesota doctors could save sister, is now a happy teen - Minneapolis Star Tribune

Bellicum Pharmaceuticals Inc (NASDAQ: BLCM) was at its volatile best all this week. After a 14-percent gain Monday, the stock pulled back slightly Tuesday and retreated by less than 2 percent Wednesday.

It rallied over 10 percent Thursday, only to slip by about 7 percent Friday amid the Bellicum's presentation at the European Hematology Association conference, in Spain.

Bellicum Pharma is into the business of developing cellular immunotherapies for various forms of cancer, including both hematological and solid tumors, as well as orphan inherited blood disorder.

Following Bellicum's update at the EHA meeting, Cantor Fitzgerald said the company announced additional data from its ongoing phase 1/2 study with BPX-501 in patients receiving blood stem cell transplant due to malignant and non-malignant blood diseases. The data provided in the update was from 98 patients at 180-days of follow up or greater, as opposed to the 81 number reported previously in the abstract.

Giving the key takeaways, analysts Elemer Piros and Justin Kim said:

GvHD occurs after the transplant of a bone marrow or stem cell belonging to another individual, as the transplanted cells treat the recipient's body as foreign and attack it.

Detailing the data, Cantor Fitzgerald said BPX-501 treatment led to a 5-percent rate of transplant-related mortality, with a 3-percent non-relapse mortality and 15-percent disease relapse rate among malignant disease patients. The performance of the patients, according to the firm, was well above historical matched unrelated donor, or MUD, publications. The results of the study showed 6878 percent 1-year overall survival.

Source: Bellicum Pharma

The firm reminded that the E.U. primary endpoint of the study would assess event-free survival composite of death, GvHD and infection at six months compared with approximately 40 matched MUD patients.

"We expect the observation MUD study, which is in the process of being initiated, to provide relevant context for BPX-501," the firm said.

The firm estimates that an additional $100 million in capital is required to reach commercialization, which it thinks could be sourced from potential licensing fees or from issuing new equity.

A such, Cantor Fitzgerald reiterated its Overweight rating on the shares of Bellicum and the $35 price target it has for its shares.

At time of writing, Bellicum shares were down a steep 7.33 percent at $12.95.

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2017 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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Despite A Volatile Trading Week, Bellicum's EHA Presentation Merits A Second Look - Benzinga

Fiona Geekie, a nurse practitioner for TheCentral London Community Healthcare NHS Trust, donated bone marrow to a person she had never met earlier this year.

According to Fiona, about 2,000 people in the UK require a bone marrow or stem cell transplant every year.

For most of them its their last chance of surviving blood cancer, she added.

The nurse, who is a member of the Merton enhanced rapid intervention team,described why she went on the register to donate through the Anthony Nolan Trust 15 years ago.

Three quarters cant find a matching donor in their family so rely upon the generosity of a complete stranger already on the bone marrow register. So I joined up, it was simple.

Last Christmas Fiona was called for blood tests as she was a possible match for someone.

I was surprised to be called in the first place and I must confess I was a little apprehensive, Fiona admitted.

However, she continued: But then I thought what if someone in my family needed a bone marrow transplant. Its the most fantastic gift you can give to total stranger.

After donating, Fiona said she felt quite emotional and wanted the recipient to get well again.

She concluded: Overall, it was a wonderful experience and I have no hesitation in recommending all young people aged 16-30 to consider joining the bone marrow donor register.

Read the original here:
Nurse goes the extra mile and donates bone marrow to a complete stranger through the Anthony Nolan Trust - Your Local Guardian

A BRAVE Barrow girl is delighted to be back at school after eight months away fighting leukemia and recovering from complications following a stem cell transplant.

Bubbly Aimee Robinson returned to St James' CE Junior School this week to a warm welcome from her friends and teachers, who have all missed having her at the Barrow primary.

The eleven-year-old last attended the Blake Street school for three weeks in September as she is a patient at the Royal Manchester Children's Hospital, where she has battled leukemia.

Following aggressive chemotherapy, Aimee had a stem cell transplant using umbilical cord blood. She did well following the transplant and spent time in isolation. But she later developed graft versus host disease. This is when particular types of white blood cell in the donated bone marrow or stem cells attack a bodies own cells.

Aimee had to spend further time in isolation as she recovered from GVHD.

Aimee, who was first diagnosed with leukemia in January 2016, is now in remission and the treatment for GVHD is also working. She was eventually allowed home to Barrow last month, but she has treatment at the Manchester hospital every fortnight.

Medics then gave her the okay to return to school this week to complete her final year of primary school, Year Six, before she prepares to attend Furness Academy in September.

Aimee, who is a house captain and school council member at St James' school, said: "It feels great to be at school with my friends. St James' is the best school ever."

Her great friend Abbie Gelling, 11, said it is really great to have Aimee back, as they had to keep in touch through FaceTime, texts and letters.

Angela Rawlinson, the headteacher at St James' CE Junior School, said: "We are so thrilled to have Aimee back at school. It's such great news. Aimee loves school and learning.

"It was very important for Aimee to get back to school before they all move on to secondary school."

The St James' school community raised 3,000 to help Aimee and her family who have spent so much time away from home. The community also fundraised to support the pupil.

Aimee has been doing her schooling at hospital with input from St James' school.

Aimee's mum Joanne Robinson, said: "Aimee has been raring to get back to school, she missed all her friends and teachers. She wanted to go back as soon as possible.

"Nothing bothers Aimee, she just gets on with it. She is a superstar.

"There is no sign of the leukemia now, her bone marrow is working brilliantly."

Mrs Robinson thanked all the medics, the St James' community and the wider community.

She said: "Thank you to everyone for the love and support they have given our family over the past 18 months and for the support we continue to receive."

Read more here:
Brave Aimee delighted to be back at Barrow school after months in hospital - NW Evening Mail