Archive for the ‘Bone Marrow Stem Cells’ Category

Two-year-old Nathan DeAndrea who underwent two stem cell transplants to treat neuroblastoma is free of cancer, according to his mother.

Testing last week that included a CT scan and a full-body scan showed no evidence of cancer, Shannon DeAndrea said during an interview at her home Monday morning.

No more cancer! said Nathans sister, 4-year-old Kailynn.

However, the DeAndreas are awaiting the results of a bone marrow biopsy performed on Nathan last week, Shannon said. Everyone is optimistic.

The doctor said he has never seen a bone marrow biopsy come back positive when everything else is clear, she said.

Results are expected this week, Shannon said.

Nathan was diagnosed with stage 4 neuroblastoma on Aug. 23, 2016. He had a tumor in his abdomen that spread to his bone marrow. He had spots on his skull, ribs and spine. He has had several rounds of chemotherapy, radiation and two stem cell transplants.

Neuroblastomas are cancers that begin in early nerve cells of the sympathetic nervous system, according to the American Cancer Society.

The scans results brought relief to Shannon and her family.

Its like I could breathe, she said.

As Kailynn put it, We said, hooray!

The next phase of treatment will include strengthening Nathans immune system. He will be in the hospital one week a month for six months, Shannon said.

Its to keep it [the cancer] from coming back, she said.

His immune system is still compromised. The genetic makeup of Nathans tumor put him at a higher risk of relapse, Shannon said.

Nathans first transplant included four or five days of chemo. The new stem cells following the chemo that killed off his old stem cells from the transplant were like a rescue, she said.

Its wiping you out and then giving you your cells back to restart your immune system, DeAndrea said.

A second round of heavy chemo was to try to kill what was left of the cancer and replenish cells, she said.

Nathans stem cell transplants were from his own cells, Shannon said.

Two types of stem cell transplants include autologous, which uses stem cells from the patients own body, and allogeneic using stem cells from another person.

The procedure is used for conditions including multiple myeloma, lymphoma, sickle cell anemia and leukemia, and other blood and immune disorders.

Stem cell transplants began in the late 50s/early 60s with the first successful procedure done in an identical twin. However, stem cell transplants were limited until medicines that prevent rejections became available.

The number of procedures increased in the 1980s.

Betsie Letterle, community engagement representative with BeTheMatch in Burlington, North Carolina, said there are more than 14 million bone marrow/stem cell donors in the BeTheMatch registry.

Bone marrow transplants traditionally involved taking the marrow from the back of the donors hip. But since then, weve progressed tremendously, Letterle said.

The newest way is to take stem cells from a vein in the donors arm, Letterle said. The donor receives an injection of medication to help their body manufacture a large amount of stem cells, she said.

Those are taken from the vein, similar to a plasma donation. Letterle said.

Anyone aged 18-44 can join the registry, but commitment is paramount among donors, she said.

Commitment is important because patients depend on us, Letterle said. We dont want anyone whos not really sure they could donate if called.

Only about one in 540 registered donors end up donating, she said. Everyone is an active donor until they turn 61, Letterle said.

Younger donors are healthier and make the most stem cells, she said.

We want to give the patient the optimum opportunity to get the best stem cells they can, she said.

If a donor comes up as a match, they will be asked for about 20-30 hours of their time over several weeks, Letterle said.

We work around the donors schedule, she said.

They get blood work done, and a physical to make sure theyre healthy enough to donate, Letterle said.

The donor never pays for anything, she said.

The doctor determines whether the procedure would be a stem cell or a traditional bone marrow transplant. That depends on the patients or recipients age and condition, Letterle said.

About 80 percent of registered donors are Caucasian, and BeTheMatch is looking for more minority donors, Letterle said. Many minority patients have trouble finding a match, she said.

The recipients blood type becomes whatever blood type the donor has, Letterle said.

Dr. William Clark with the Massey Cancer Center at Virginia Commonwealth University will speak about bone marrow and stem cell transplants from 11:30 a.m. to 1 p.m. July 11 at Ballou Recreation Center. A bone marrow/stem cell donor drive will also be held that day.

For more information on stem cell/bone marrow transplants, call Betsie Letterle at BeTheMatch at (877) 601-1926, ext. 7721.

JohnCrane reports for the Danville Register & Bee. Contact him atjcrane@registerbee.comor(434) 791-7987.

Link:
Tests show no signs of cancer for Danville 2-year-old - GoDanRiver.com

BAREILLY: Dog owners from across the country, including Delhi and Gujarat, are turning up with their paralytic pets at the Indian Veterinary Research Institute (IVRI) here for stem cell therapy. Scientists treat a paralyzed dog by transplanting stem cells from healthy dogs. IVRI is the second institute in the country to offer this treatment, after Madras Veterinary College, Chennai.

According to scientists, no research has been conducted to determine the number of dogs who suffer from paralysis every year in India. However, the institute receives at least four cases every week of spinal trauma which causes paralysis in dogs. IVRI recorded 143 cases of posterior paralysis in 2016. These were treated with stem cell therapy and medicines.

"If dogs are treated only with medicines, recovery is witnessed only in a few cases," said Amarpal (who goes by his first name), head and principal scientist, division of surgery, IVRI. On an average, 17% recovery rate was noted among dogs administered only medicines.

However, the best response was recorded among severely affected dogs when they were treated using stem cells, where almost all the patients responded to treatment to variable extent, said the scientist. "Though we have cases where recovery was 100%, the average recovery rate is about 50%. The experiment proved the efficacy of stem cell therapy in cases of paralysis due to spinal trauma," said Amarpal. After seven years of research, stem cell therapy was started at IVRI five years ago for clinical purposes on a nominal registration fee of Rs 30.

Due to its success, pet owners from various parts of the country have started visiting the institute.

See the article here:
Dog owners flock IVRI for pets' cure - Times of India

Visakhapatnam: Lack of awareness on the importance of becoming a blood stem cell donor is hindering the treatment of people suffering from blood cancer and other fatal blood disorders like thalassemia and aplastic anaemia.

Since the only cure is a blood stem cell transplant, the need of the hour is to sensitise people of the city on the necessity of becoming a potential blood stem cell donor to save lives, experts say.

According to oncologists based in Vizag, the cases of blood cancer are increasing and the treatment options are very few. Moreover, patients with blood cancer are sent to Hyderabad and other centres for treatment as neither there are donors nor equipment for stem cell transplant in the port city.

For a successful transplant, patients suffering from fatal blood disorders need blood stem cells from a healthy and genetically matched donor. Unfortunately, the probability of finding a genetically matched donor is one in 10,000 to one in over a million.

Only about 25 per cent of the patients find a donor from within their family. Rest need to wait for a life-saving donor.

"The chances of finding a match for patients suffering from these fatal blood disorders could only widen if there are more number of donors registered," says Ravindranath Chava, co-ordinator of a Chennai-based blood stem cell donors registry- DATRI.

Read the original:
Lack of stem cell donations plagues patients - Times of India

Bareilly: Dog owners from across the country, including Delhi and Gujarat, are turning up with their paralytic pets at the Indian Veterinary Research Institute (IVRI) here for stem cell therapy. Scientists treat a paralyzed dog by transplanting stem cells from healthy dogs. IVRI is the second institute in the country to offer this treatment, after Madras Veterinary College, Chennai.

According to scientists, no research has been conducted to determine the number of dogs who suffer from paralysis every year in India. However, the institute receives at least four cases every week of spinal trauma which causes paralysis in dogs. IVRI recorded 143 cases of posterior paralysis in 2016. These were treated with stem cell therapy and medicines.

If dogs are treated only with medicines, recovery is witnessed only in a few cases, said Amarpal (who goes by his first name), head and principal scientist, division of surgery, IVRI. On an average, 17% recovery rate was noted among dogs administered only medicines.

However, the best response was recorded among severely affected dogs when they were treated using stem cells, where almost all the patients responded to treatment to variable extent, said the scientist. Though we have cases where recovery was 100%, the average recovery rate is about 50%. The experiment proved the efficacy of stem cell therapy in cases of paralysis due to spinal trauma, said Amarpal.

The paralytic dog is first administered anesthesia before the stem cells are injected into its spinal cord. It takes only one session for a dog to undergo the therapy and it is discharged the same day.. After this, the owner has to bring his pet for check-ups for two or more times so that vets can monitor how the animal is responding to the treatment and if it is suffering from any reaction, said Amarpal.

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Stem cell therapy to treat paralytic dogs draws pet owners from across country to IVRI - Times of India

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."

Read the rest here:
Jeff Jacobs: Hall Of Fame Coach Holowaty Fights Illness And Gives Back - Hartford Courant

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

See original here:
Stem Cell Therapy for Type 1 Diabetes - Medical News Bulletin

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."

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

The Hindu

A ray of hope for autistic children The Hindu Dr. Gokulchandran said a number of autistic children from AP visit the hospital for the therapy that involves collecting stem cells from the patient's bone marrow and injecting them into the spinal fluid to regenerate the inactive part of the brain ... and more »

Read the original:
A ray of hope for autistic children - The Hindu

Updated: Jun 30, 2017 - 11:30 PM

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 couriered 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.

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

By Manisha Samy, ARK analyst

Discovered nearly thirty years ago in 1988[1], stem cell technology has failed to transform the field of regenerative medicine until now. After delivering only one treatment during three decades of development, stem cell technology could finally become the all-purpose tool for repairing the body, thanks to rapid and precise genome editing techniques such as CRISPR and TALENs. Several obstacles in stem cell technology- high costs, safety concerns, and bioethical considerations are beginning to fall away. In particular, the introduction of induced pluripotent stem cells (iPSCs) eliminated many of the initial bioethical concerns stirred by the the source of stem cells. Now, advances in genome-editing is accelerating the pace of progress.

Stem cells exist in a state of possibility. Two key markers of stem cells are 1) the ability to self-renew and 2). The ability to become any specialized cell. Broadly speaking, a stem cell can evolve into any of the ~200 specialized human cell types, as illustrated in Figure 1. Directing stem cells to become any one of the 200 cell types has proven challenging.

Moreover, before the introduction of iPSCs, which are derived from adult cells, most human stem cells were sourced from human embryos or cord blood. The political and ethical controversies surrounding embryonic research curtailed stem cell funding. As a result, after a quarter century of research, bone marrow transplantation is the only FDA-approved stem cell application in the United States.

Figure 1

Breakthroughs in cheap, rapid genome-editing have re-invogored momentum in stem cell research. As shown in Figure 2, the number of publications on PubMed mentioning both stem cells and gene-editing hit a tipping point upon the discovery of CRISPR in 2012, three years after the first instance of precise TALENs-based genome-editing in human cells. Based on the current trajectory, ARK estimates that the number of scientific publications including both genome-editing and stem cells will approach 300 this year, accounting for nearly 20% of the total number of publications.

Figure 2

As illustrated in Figure 3, while the discovery of iPSC cells in 2006 boosted stem cell research noticeably beginning in 2008, the introduction of CRISPR in 2012 has further catapulted the stem cell field, with combined gene-editing and stem cell publications taking share.

Figure 3

Why Is The Addition of Gene-Editing So Important to Stem Cell Research and Therapy?

Figure 4

Ultimately, CRISPR edited iPSCs should unlock the code to human disease at a cellular level. Three public CRISPR companies are in a good position for the impending stem cell revolution. Editas Medicine (EDIT) signed a stem cell pact with GlaxoSmithKline (GSK) and Biogen (BIIB) for next-gen stem-cell therapies; CRISPR Therapeutics (CRSP) CRSP),+Casebia+Therapeutics+Sign+Commercial+License+Agreement+to+MaxCyte/12664463.html" rel="nofollow">licensed a CRISPR delivery mechanism for blood stem cells and has formed a CRISPR joint venture with Bayer (BAYZF); and Intellia Therapeutics (NTLA) has partnered with biotech giant Novartis NVS to focus on stem-cell based therapies. These companies, among others, finally might unleash the limitless possibilities that stem cells once promised in regenerative medicine, extending human life spans considerably.

Notes

[1] The Weissman Lab at Stanford University first isolated hematopoietic (blood) stem cells from mice bone marrow in 1988; it would be another decade before human stem cells were isolated.

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Disclaimer: 2017, ARK Investment Management LLC ("ARK"). All content is original and has been researched and produced by ARK unless otherwise stated. No part of ARK's original content may be reproduced in any form, or referred to in any other publication, without the express written permission of ARK. The content is available for informational purposes only and is subject to change without notice. All statements made regarding companies or securities or other financial information on this site or any sites relating to ARK are strictly beliefs and points of view held by ARK or the third party making such statement and are not endorsements by ARK of any company or security or recommendations by ARK to buy, sell or hold any security. For a list of all purchases and sales made by ARK for client accounts during the past year that could be considered by the SEC as recommendations, click here. It should not be assumed that recommendations made in the future will be profitable or will equal the performance of the securities in this list. For full disclosures, please see the Terms of Use for this site.

Editor's Note: This article discusses one or more securities that do not trade on a major U.S. exchange. Please be aware of the risks associated with these stocks.

Read more from the original source:
The Stem Cell Revolution - Seeking Alpha

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

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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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

Jonathan Pitre is a teenager who loves to write science fiction as an escape from the painful disease that causes his body to be coated with wounds.

But the breakthrough bone-marrow transplant he just received at the University of Minnesota is anything but fantasy.

A decade after performing the worlds first bone marrow transplants to treat epidermolysis bullosa a rare and potentially fatal skin disease university researchers believe they have discovered a powerhouse new formula that advances their research, helps the body grow new skin and will allow patients such as Pitre, 17, to live longer, less painful lives.

Its really not miraculous. It certainly isnt science fiction, said Dr. Jakub Tolar, director of the Us stem cell institute and the world leader in transplant therapies for EB. Its based on the hard work of our predecessors. You accomplish something and then you use that knowledge to enhance the next step and the next step.

When they conducted the first transplants using donor bone marrow and umbilical cord blood in 2007, Tolar and colleagues were trying to produce a collagen that binds skin together and is lacking in EB patients. But they had little certainty about the types of cells that would work best.

Since then, research discoveries have allowed them to home in on mesenchymal stem cells, which they believe are uniquely good at bullying their way into the body and producing the missing collagen.

This is the first time ever, that I know of, when you are infusing them with the goal that these cells will stay, Tolar said. They will graft into the skin, set up shop there. Its as if these mesenchymal stem cells are coming home.

The doctors have also focused on transplants involving bone marrow from relatives, which is more familiar to the body and less likely to be rejected by the recipients.

A transplant like Jonathans occurs in a one-two punch. After receiving radiation and chemotherapy treatments to suppress the immune system, the patient receives an infusion of hematopoietic blood stem cells from a donor. Their job in this procedure is to give the patient a new immune system that wont reject the donors mesenchymal cells when they are transplanted later.

Since the U received federal approval last fall to offer the treatment experimentally, seven patients have undergone the procedure.

Tolar said all seven are progressing though Jonathan needed a second transplant this spring because the first one failed to knock out his old immune system.

Jonathan suffered an infection after his most recent transplant, which forced him to return to the hospital this month with high fevers and blisters on his face and mouth. Even so, Jonathans mother, Tina Boileau, said she has been taking pictures since the latest transplant to document the progress for her son, whose back is covered with wounds but for a healthy spot on his right shoulder blade.

Theyre actually in scabs, a sign of healing, said Boileau, who was the bone marrow donor for her sons transplant. Which Ive never seen before.

10 patients died

EB afflicts about one in every 30,000 to 50,000 people, though some forms are more severe than others. While it is known largely for the grotesque skin wounds it causes, the disease is often fatal because it leads to severe infections or skin cancers. It can also create internal wounds to the patients digestive tract, which impairs eating.

The desperation of children with the disorder and their families compelled the first transplants at the university in 2007. Even using the old approach, about two-thirds of patients saw improvements, but 10 of the first 30 recipients died from their diseases or complications of treatment.

The Us latest success with mesenchymal stem cells might end up being an incremental step. Earlier this year, Tolar and his colleagues published research showing success in an even more advanced therapy: laboratory testing using gene editing that can reprogram the patients cells to produce healthy skin cells and tissue.

Further successes could lead to clinical trials in which a patients own dysfunctional cells would be reprogrammed, preventing the need for chemotherapy and the replacement of their immune systems.

Before they came to the U, Boileau said, her son had run out of options. Managing his pain, once possible with over-the-counter Advil, had come to require opioid painkillers such as methadone. That made him groggy and complicated his already awkward life at school back home in Ottawa. Jonathan wasnt even able to eat lunch in the school cafeteria for fear of being accidentally bumped and suffering fresh wounds.

Then the Canadian government approved funding to make him his countrys first recipient of an experimental bone marrow transplant for EB. And his home community rallied to support the family. Among other things, he has visited with pro hockey players from the Ottawa Senators, which also issued a contract adding him to their scout staff.

After seeing the pain her son has endured, Boileau said shell never complain about a blister from new shoes. She marvels at his optimism and his use of science fiction reading and writing to escape.

Inspired by the success of Christopher Paolini, who wrote the acclaimed Eragon science fiction novel as a teen, Jonathan has resolved to write his own science fiction book about a teen who develops the ability to overcome EB. The project resulted in long visits and e-mail exchanges between Tolar and his patient about medicine and physics, because Jonathan wants his story grounded in reality.

Theyre almost soul mates, Boileau said.

Tolar said he enjoys the intellectual relationship and that his patient is providing an example of hope and teaching others about the disease: He may be the only person [who] can bring this kind of view to others, Tolar said.

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U team discovers 'powerhouse' new treatment in fight against deadly skin disease - Southernminn.com

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:

Follow LifeNews.com on Instagram for pro-life pictures.

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.

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Adult Stem Cells Save Woman Ravaged by Lupus, Now She Can be ... - LifeNews.com

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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

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

After being diagnosed with the disease on Oct. 25, 2016, 20-month-old Tegveer Minhaswasforced to go 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 was able to receive a stem cell donation, but Minhas said it didn't work.

"After 8 months of struggle, he passed away on June 18th, early in the morning at 6 a.m.," said his dad,Sukhbir Minhas.

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

"He was a happy soul, he loved to go out, we took him to Clear Lake on June 4th, 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 like his.

"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."

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

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

A BRAVE Barrow girl is delighted to be back at school after eight months away fighting leukaemia 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 leukaemia.

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 body's own cells.

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

Aimee, who was first diagnosed with leukaemia 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 OK 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 in 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 leukaemia 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."

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

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

Matthew Medinas doctors diagnosed him with a rare blood disease a few months ago and told him he would probably die without a bone marrow transplant.

With that prognosis came another: The 40-year-old Los Angeles police officer had a less than 50% chance of finding a donor because he is not white.

Most successful matches for bone marrow transplants involve a donor and patient of the same ethnicity. But the majority of the 25 million registered donors nationwide are white, and Medina is Filipino. So far, no match has been found.

Youre basically looking for a genetic twin, said Athena Mari Asklipiadis, who runs Mixed Marrow, an L.A.-based organization that is trying to increase diversity in the bone marrow donor registry. Its not like we have more of a chance we would get a disease, or that were harder to match, its just that theres not representation in the national registry.

Its a familiar problem for any nonwhite person who has needed a bone marrow transplant.

A white American of European descent has a 75% chance of finding a perfect match in the national donor registry, compared with a 40% chance for Filipinos. Few Filipinos in the U.S. have signed up as potential donors, and there is no registry in the Philippines.

Researchers are experimenting with ways to perform bone marrow transplants on people who cant find matches. But while those treatments are being perfected, thousands of people are diagnosed every year with leukemia, lymphomas and other blood diseases whose only hope for a cure is a marrow transplant. And for them, it can come down to ethnicity.

Medinas wife, Angelee, has watched dozens of people at sign-up events across Southern California, particularly in the Filipino community, volunteer to donate bone marrow with the hope of curing her husband. Were very thankful for that, she said. Were hoping something comes up.

For now, Medina is being kept alive with transfusions.

All you want is for that loved one to have a chance, said Officer Dante Pagulayan, Medinas partner at the LAPD and a childhood friend. Thats what were praying for.

Medina went to the doctor in March because he had a rash. His blood work revealed something far more dangerous.

Medina was diagnosed with aplastic anemia, a disease in which the bone marrow stops working. Bone marrow is spongy material inside bones that produces the essential components of blood white blood cells, red blood cells and platelets.

Between 600 and 900 Americans are diagnosed with aplastic anemia each year, according to the Aplastic Anemia and MDS International Foundation. The disease can be caused by exposure to toxic chemicals or a virus, but most cases, including Medinas, are unexplained.

One day he wakes up and the doctor tells him he has this. It could happen to anyone, said Pagulayan, who went to high school and Cal State Long Beach with Medina and now works alongside him in the gang unit in the LAPDs Harbor Division.

Blood transfusions can sustain Medina for now, but the only possible cure for aplastic anemia is a transplant, said Dr. Len Farol, a bone marrow transplant specialist at City of Hope National Medical Center and one of Medinas physicians.

Medina is quarantined at his home in Bellflower, where he lives with his wife and two young daughters. But he needs a transplant soon because his immune system is so weakened from his disease that exposure to a common virus could kill him, Farol said.

Doctors checked to see if Medinas sister could be a match, but she wasnt siblings provide a match only about 25% of the time. They started combing through the registry, but trying to find a donor there can be like finding a needle in a haystack, Farol said.

Doctors look to see if the patient and potential donor share eight cell markers called human leukocyte antigens, or HLA. All eight have to match, but thats rare because there are thousands of possibilities for each marker, experts say.

There could be billions of combinations, said Stephen Spellman, director of immunobiology and observational research for the Center for International Blood and Marrow Transplant Research. Within any group, finding a match for HLA is difficult.

Spellman said that people whose ancestors are from the same place tend to have the same markers because they evolved over time in response to different pathogens and diseases that were present in their environment.

According to a 2014 report in the New England Journal of Medicine, a person of white European descent has the highest chance of finding a perfect match eight out of eight HLA markers in the national registry of any ethnic group.

What's your chance of finding a perfect match? If you're...

Source: HLA Match Likelihoods for Hematopoietic Stem-Cell Grafts in the U.S. Registry, New England Journal of Medicine, 2014

Pagulayan, who is also Filipino, said neither he nor Medina knew ethnicity would affect his chances of being cured. Finding out that less than 1% of people in the registry were Filipino was very disheartening, he said.

There are international registries, but the vast majority of people worldwide whove signed up to donate bone marrow are from the U.S and Europe.

Plus, nonwhite populations in general tend to have more genetic diversity. African Americans, for example, have highly diverse genetics because of mixing with other groups since arriving in the United States, experts say. Filipinos are also very diverse because of the countrys long history of colonization.

Still, experts say that everyone who wants to help Medina should sign up for the registry, regardless of ethnicity.

Matches often break down along ethnic lines, but not always. Sometimes markers in one population also appear in another, or people dont know their lineage.

Maya Chamberlin, who is half Indian and half white, had two bone marrow transplants after she was diagnosed with a rare blood disease called HLH in 2009 when she was 4.

Mayas first donor was half Japanese and half Latino, and her second was half Japanese and half Filipino.

So you never know how this works until you get on the registry, said her mother, Mina Chamberlin, who lived in Torrance when Maya was diagnosed but has since relocated to Cincinnati to be closer to physicians who specialize in her daughters disease. You just never know.

Angelee Medina canceled her familys vacation to Mexico scheduled for this summer. Shed been commuting to a job as a graphic designer 20 miles from home when Matt was diagnosed, but found a closer place to work so she could take care of the kids and be near her husband.

It was very, very overwhelming in the beginning, she said. With all the support were getting from everyone around us, it feels hopeful.

More than 1,000 people have signed up to donate bone marrow over the past few months through dozens of drives for Medina, said Chris Chen, a recruitment coordinator for Little Tokyo-based nonprofit A3M, which focuses on getting more Asians to sign up for the Be the Match registry to donate bone marrow.

Potential donors submit cell samples by having the inside of their cheek swabbed. The cells are then analyzed to determine their HLA markers.

About 70% of transplants employ a process called peripheral blood stem cell donation, which is similar to a blood donation but can take several hours. In the other 30% of cases, donors are admitted to the hospital and anesthetized so doctors can remove marrow from their pelvic bone with a needle.

Ayumi Nagata, recruitment manager for A3M, knows that asking people to volunteer for a medical procedure they dont need themselves can be a hard sell. But she tries to impress upon them how they could be the cure for someones cancer or other disease and save their life.

How often do we have that kind of opportunity? Nagata said.

The Medinas 8-year-old daughter, Cassiah, made a sticker thats distributed at donor drives that says, Keep calm and help our daddy fight! When Angelee picked up Cassiah from day care recently, she found out that her daughter had been asking the other kids parents: Did you get swabbed? Have you gotten swabbed yet?

Doctors are testing ways to perform transplants on patients who cant find a bone marrow match. Some are using umbilical cord blood, donated by mothers whove just given birth, which scientists say has a lower chance of rejection even if its not a complete match.

Haploidentical transplants, in which the donor and patient share only half of the eight markers, have also been successful in clinical trials, Spellman said.

Medinas doctors think his best shot is still a perfect match for a bone marrow transplant, his wife said.

Thats just what were waiting for, she said. I remind him one day soon, hopefully everything will be better.

What to know about joining the bone marrow registry

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An LAPD officer needs a bone marrow transplant. His ethnicity limits his chances of getting one - Los Angeles Times

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.

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'Little Frankenstein' is now a happy teenager - Minneapolis Star Tribune

Elizabeth Nega knows that time is not on her side.

The Vancouver mom was diagnosed with acute lymphoblastic leukemia in February and she needs a bone marrow transplant.

Finding a suitable donor has been difficult.

Im very lucky if I get that person who [is a] match to me, she said. My brother and my sister didnt match. If somebody matches, thats a miracle.

She has been undergoing regular chemotherapy treatments since her diagnosis.

The first time they told me if I get 90 per cent remission, they will treat it for two years with chemotherapy, then I will be fine, Nega said. Unfortunately, it didnt show 90 per cent remission.

Nega, who goes by the name Elsa, and her family set up a website and social media channels to help in the search for a donor.

READ MORE: I need a man: Ethnic donors desperately needed for bone marrow registry

Nega, who was born in Ethiopia, needs a match from someone of African descent, which could prove to be a challenge.

Ethnic diversity in the stem cell donor bank is woefully limited, according to Trudi Goels of Canadian Blood Services.

Our registry is about 69 per cent Caucasian, which is huge and it doesnt really reflect the diversity in Canada accurately, she said.

So what we really want to encourage is people who are of African descent to come forward and register to support Elsa.

The ideal candidate is between 17 and 35 years of age. Men are preferred, but women are just as welcome.

Nega hopes a candidate can be found quickly. Her doctor said she can only do about two more chemotherapy treatments before theyll have to stop.

If I get bone marrow transplant, that means another chance to survive, she said. A second chance in my life. If I get that I will survive. Thats my thinking, thats my hope.

2017Global News, a division of Corus Entertainment Inc.

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BC woman in need of bone marrow transplant pleas for more donors of African descent - Globalnews.ca

Founded in 2004, LifeCcell has technological collaboration with the US-based Cryo-Cell Internationalthe worlds first private stem cell bank with over 25 years of experience. (PTI)

Chennai-based LifeCell, the provider of preventive healthcare services for family wellness, is the worlds second-largest provider of umbilical cord stem cells. Founded in 2004, the company has technological collaboration with the US-based Cryo-Cell Internationalthe worlds first private stem cell bank with over 25 years of experience. As many as 2 lakh Indian parents have chosen to trust their newborns umbilical cords to LifeCell through its umbilical cord banking service BabyCord. The company has a 60% share in the Indian market.Stem cells are mother cells that have the potential to become any type of cell in the body. One of the main characteristics of stem cells is their ability to self-renew or multiply, while maintaining the potential to develop into other types of cells. These cells can repair and rebuild damaged tissue. The uses of stem cells are still being researched. In fact, stem cell tissues have proved effective in cancer treatment too. The applications have been steadily increasing in the last few years. They have been used for treating wound healing, including diabetic foot ulcers. In a country where concepts like bone marrow donations and stem cell banking are still not widely known, Mayur Abhaya, the CEO and managing director of the company, is betting on these treatments of the future.

The company is the most accredited stem cell bank in the country, with certifications from national and international organisations for standards. It is also the only player in the industry providing comprehensive stem cell solutions, including menstrual stem cell banking, R&D and point-of-care stem cell therapy for orthopaedic and vascular specialities.Mayur has been heading LifeCell since 2008. He comes from the family that set up Shasun Group of companiesthe provider of contract pharmaceutical manufacturing services for global companies. Mayur studied biotechnology in India and the US, and then worked in the US for a year. Before moving to LifeCell, he worked for many years at Shasun Pharmaceuticals, where he led their new product development, intellectual property and licensing initiatives. In 2013, LifeCell International got an investment of Rs35 crore from Helion Venture Partners, an India-focused venture fund, to support its plans of increasing market penetration of stem cell banking in India and enabling the development of novel cell-based therapies.

Also Watch: Mayur says LifeCell currently operates in 150 cities, employing more than 1,500 people. We have given an opportunity to our sales people to become their own bosses. They remain on company rolls and get to enjoy all the company benefit plans, such as insurance and welfare schemes. They grow with the company and also have the opportunity to explore and add non-conflicting products or services to their distribution network and enhance their earnings. These internal franchisees bring 50% of our revenue and it is growing. More than 50 such entrepreneurs have been created.LifeCell recently bought over the stake held by Helion Ventures with borrowings from family-owned firms. Three months ago, it changed its business model. We are introducing an on-demand model for sharing cord blood cells, Mayur says. Parents can let the company know if their babies cord blood cells can be used for other needy patients. Cancer patients cannot be treated with their own stem cells. Patients usually do not have much time. Cord cells can be used even if all the six parameters that are required to transplant tissues do not match. By letting their stem cells be used by others, parents and their children get access to cord blood cellsof the entire cord blood cell bankwhen they are in need. So far, stem cells were banked only for the baby from whom these were removed.

Our inventory will come to the aid of people who do not have babies. We will refund the amount paid for having their babys stem cell stored. The processing fee is Rs17,000 and the storage fee each year is Rs4,000. Mayur says that the worlds largest birthing country has a long way to go to create a viable stem cell bank. We are going to follow the blood bank model and hope to bank 2,50,000 cords, which is the critical amount, he adds. We hope to contribute significantly to the ever-developing scope of transplant medicine. Currently, India is importing cord cells, which are prohibitively expensive. With scale, prices will come down in the country. Parents in India will have higher future access to stem cells than even those enjoyed by patients in advanced countries such as the US. We will have a linkage with global inventory. Earlier this month, LifeCell was invited by AABB (formerly American Association of Blood Banks) to present the concept of Community Stem Cell Banking at the 15th International Cord Blood Symposium held in San Diego, US. In 15 years, it is the only second stem cell bank to present its innovation at such a prestigious global platform.

With a turnover of Rs126 crore, LifeCell is operationally profitable. It has enough cash to run its business, but is yet to make net profits. However, Mayur believes very soon LifeCell will turn profitable, and that this year the number of stem cells brought into the labs will be higher by at least 30%. Mayur has extended LifeCells services to introduce and popularise the concept of essential preventive diagnostics for mothers and babies. BabyShield has been introduced to bring down infant mortality ratio. Addressing gaps in marketplace and with innovative business models, it has established market leadership in newborn screening. It has also acquired a prenatal screening service provider. In India, only 2% babies go through prenatal and newborn screening. Nobody has focused on this. We will also be providing diagnostic medication. Doing this can prevent so many false positives. We are building all this together as a package and are offering it at an affordable price, he says.

Read more:
How LifeCell became the most accredited stem cell bank in India - Financial Express

The Allums family is looking for a bone marrow match for their oldest son. The same match could later help their youngest daughter, who shares the same rare form of anemia as her brother. Courtesy Cole Prine

Caroline, 11, Elizabeth, 3, Jon Thomas, 13 and James Christopher Allums, 20, do everything as a family. James Christopher and Elizabeth both have a rare medical condition. Their mother, Ellen Allums, said they all go through the process together and support each other with faith and love.(Photo: Courtesy)

Chris Allums was raised in Shreveport. He attended Captain Shreve High, and his family still visits his mother there. He said the community is special to his family.

He and his wife Ellen have four childrenJames Christopher, 20;Jon Thomas, 13;Caroline,11;andElizabeth, 3.

James Christopher and Elizabeth have Fanconi anemia, a disease that affects the bone marrow's ability to produce blood. Bone marrow or blood stem cell transplants are considered the best treatments, andthey have not found a match for either child.

The disease is genetic. According to theNational Organization for Rare Disorders, the incidence rate is 1 in 136,000 births. Ellen said her children are two ofsix in Louisiana affected by FA.It has a variety of symptoms such as fatigueand can lead to bone marrow or organ failure. Ellen and Chris said FA patients are 500 times more likely to develop some cancers, such as leukemia.

James Christopher was diagnosed 12 years ago and told he had about 18 monthsto live. The family was told he must received a bone marrow transplant. He is subject to constant screenings to monitor his blood and check for cancers.

Ellen said they immediately started praying for a miracle. They planned to grow their faith and help as many families as they can along the way until a match is found.

More than 16,000 people have been added to the worldwide bone marrow registry as a result of drives held on behalf of the Allums. Ellen said they know of at least 41 lives that have been saved because of those efforts, and they're asking more people to commit to donate.

From 9 a.m. to 3 p.m. June 24, Tiger Rock Martial Arts of Shreveport, 1409 E. 70th St., Shreveport, will host a bone marrow drive to help the Allums find a match.

Want to go?

Ellen said the process to donate blood stem cells, which is the most common donation method, involves a needle in each arm for four to six hours.

"It's not even a surgery. It's not like giving a kidney or a lung or a heart, even, but the benefits are that strong. It can truly save a life, but yet all you have to do is like giving blood," Ellen said.

To test for a match, she said, it's less of a commitment. It takes about five minutes to fill out paperwork and provide a swab from inside the cheek. Anyone 18-55 in good health can register. A month after testing, people will get a phone call to confirm their position on the registry.

A bone marrow transplant won't cure someone with FA, but it can help prolong life.

One donor, once found, could help both children.Elizabeth's blood counts have been OK, but doctors have said James Christopher has an immediate need for a transplant. DNA needs to be close to an exact match, and many families find a relative who can donate.Elizabeth is a 100 percent match, but she's ineligible because of her FA.

James Christopher Allums, 20, holds his sister Elizabeth Allums, 3. The siblings both have a rare medical condition called Fanconi anemia.(Photo: Courtesy)

If a match is found, the entire family would move to New York for six to eight months. The couple did their homework on hospitals that specialize in the disease and settled on Memorial Sloan Kettering Hospital's cancer center. It had the best survival rates, and they've been going for 12 years.

Ellen said the a bone marrow recipient with FA will have to go through chemotherapy for two weeks to kill off the patient's natural bone marrow.

"When the cells are dead, then they receive someone else's bone marrow. It's a liquid, it looks just like an IV, and they lie there and you just pray to God that it's going to take," she said.

After the transplant, the patient is in isolation for 30-40 days. They stay at the transplant hospital for six to eight months and keep a medical mask on for one year. Chris said you hope graph vs. host disease isn't an issue.

Chris said it's important for people to get registers and commit to helping if a match for anyone is found. He said the life you end up saving might be your own. They know of at least one woman who found a quick match when she needed a donor because she had already registered.

FA patients can require blood and platelet transfusions, after which they may become dependent and need additional rounds, which would require a bone marrow transplant quickly.

James Christopher received his first blood transfusion earlier this year. Chris was the donor.

A few weeks ago, James Christopher's counts came back dangerously low. The family had to skip travel to a relative's wedding in Colorado, but Chris said they dressed up, bought a wedding cake and arranged a viewing via Skype. They made lemonade out of lemons, he said.

Chris can donate again in three week, and James Christopher is opting not have his counts check for now.

Want to register?

Order a testing kit online atdkms.orgorbethematch.org.

Online

CheckThe Friends of James Christopher and Elizabeth Allumson Facebook or visitcaringbridge.organd searchJames Christopher Allums.

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You could be a hero for this family - Shreveport Times