Category Archives: Bone Marrow

Synthetic Bones: A Better Bone-Marrow Transplant? – The Scientist

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VARGHESE LAB, UC SAN DIEGOPeople with diseases of the blood often need bone marrow transplants to replace their blood-forming stem cells with those from healthy donors. But before those transplants, patients must eliminate their own bone marrow lest it compete with the introduced cells, and that process, which involves high doses of radiation and often drug treatments, too, has notoriously awful side effects, including nausea and fatigue.

Shyni Varghese of University of California, San Diego, and her colleagues have devised a way to skip this step: they have created a synthetic bone that can be filled with donor stem cells and transplanted without eliminating the recipient’s own cells first. As they reported in PNAS yesterday (May 8), the procedure works in mice.

“We’ve made an accessory bone that can separately accommodate donor cells. This way, we can keep the host cells and bypass irradiation,” Varghese said in a press release.

The synthetic transplants consisted of two layers of hydrogel matrices: an outer layer containing calcium phosphate minerals and an inner layer, which the researchers filled with stem cells from donor mice. The researchers inserted the artificial bones under the skin of recipient mice, and after a month, the transplants contained both host and donor blood cells. After six months, both cell types were circulating in the bloodstream, according to the release.

“[The transplants] functioned very much like our own tissue,” Varghese told KPBS.

Edward Gordon-Smith, an emeritus professor of hematology at St. George’s University of London, called the study a “splendid achievement” in an interview with New Scientist.

If it were to be translated into human use, the procedure would only work in patients with non-malignant bone marrow diseases, Varghese added in the release. Such diseases include aplastic anemia, low blood-cell counts, and cases where the immune system attacks the bone marrow. In cases of blood cancers, such as leukemia, where cells in the bone marrow may themselves cause problems, it will remain necessary to kill off a patient’s own bone marrow before a transplant.

Geneseo sophomore prepares for bone marrow transplant – Quad-Cities Online

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GENESEO — Trevor Schefsky’s battle of a rare genetic condition began at birth. Now a sophomore at Geneseo High School, that battle likely will make him miss the start of his high school junior year.

Mr. Schefsky, along with his parents, Tony and Lori Schefsky, and hsi brother, Zach, are at the Children’s Hospital in Cincinnati, Ohio, where he is scheduled to receive a bone marrow transplant later this month.

Before that happens, he will undergo chemotherapy to destroy the damaged cells in his bone marrow. Mrs. Schefsky said the process takes four to six months, keeping her son hospitalized until at least July.

His condition, DADA2, is a recessive genetic condition that causes mutations in the CECR1 gene preventing it from correctly encoding the enzyme Adenosine Deaminase 2 (ADA2) that stabilizes the lining of blood vessels.

“Without it, the body attacks its own healthy cells, which, in turn, leads to inflammation, immune deficiency, organ damage and the destruction of bone marrow,” Mrs. Schefsky said.

There is no enzyme replacement therapy available for ADA2 deficiency. Because little is known about the disease, there are relatively few treatments, Mrs. Schefsky said.

Trevor’s journey began shortly after his birth. Until he was 5 years old, his mother said, he suffered from rashes, hives, swollen eyes, high fevers, asthma, pneumonia, joint pain, mouth sores, allergic reactions to antibiotics and his legs “giving out.” She said it is believed he was in remission between the ages of 5 and 12.

“When Trevor was in seventh grade, he had a swollen lymph node in his neck, so we went to our family doctor,” she said. “They did blood work and Trevor’s white blood cells and absolute neutrophils were alarmingly low. We were put in the hands of St. Jude in Peoria.”

Because of his blood levels, he couldn’t attend school and be in public places. He went though many tests in search of a diagnosis; although doctors determined his illness was related to his immune system, no single cause of his illness was found.

He was referred to an immunologist at the Children’s Hospital in Cincinnati, Ohio, where they also did many tests — but reached no diagnosis.

“However, they were treating the low levels he had with infusions,” Mrs. Schefsky said. “As the infusions and medications have never helped with his numbers coming up, they have prevented him from illnesses.” 

Trevor could return to school as an eighth-grader, but was not allowed to play soccer, his favorite sport. He also couldn’t snow ski, play basketball or participate in any contact sports because of an enlarged spleen.

“That was a very hard year for Trevor,” Mrs. Schefsky said. “He lived his life with monthly infusions and daily medications and weekly blood work that showed to always be low. He was hospitalized in Peoria anytime he would run a fever of 99.6 or above.

“Our lives went on hold in the blink of an eye,” she said. “We lived our lives as normal as possible. Trevor was rarely sick and he didn’t like his life being on hold.

“We were able to get soccer approved with a spleen guard,” she said. “He was feeling like life was semi-normal.”

Trevor was selected for a research study at the Children’s Hospital in Cincinnati. About a year later, the family received a phone call with a diagnosis: DADA2.

“No one seems to know very much, but more is being learned every day,” Mrs. Schefsky said.

In November 2016, the family went to Washington, D.C., for the first-ever conference on the disease. They were able to meet families going through the same thing as they were, and the doctors working on a cure.

Mrs. Schefsky said Trevor has reached a point where medications and infusions are not raising his levels.

“His bone marrow is at 40 percent, so we cannot wait any longer,” she said. “It is time for a bone marrow transplant, which will hopefully be a cure for Trevor.

“In all of this — with other hardships and losses — our faith has kept us going and I have always felt God is with us,” she said. “I do not believe God gives us these hard times in our life, but I do completely believe that God is with us in these hard times. He is there in our doctors and, with as rare as a condition that Trevor has, it is a complete miracle that we have a diagnosis at all.

“God is good all the time,” she said. “All the time, God is good.”

Mrs. Schefsky hopes their story encourages others to learn more about DADA2 by visiting dada.org. “TrevStrong” T-shirts also are being sold to support Trevor’s journey.

The T-shirts include scripture from Joshua 1:19 on the back: “Be strong and courageous. Do not be afraid; do not be discouraged, for the Lord your God will be with you wherever you go.”     

Shirts may be ordered at trevstrongshirts@gmail.com. Contributions to TrevStrong may be mailed to Central Bank, 101 N. State St., Geneseo, IL 61254, made out to TrevStrong with “Schefsky” in the check memo line.

What’s It Like to go through Bone Marrow Transplant – Rabwah Times (press release) (blog)

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In 2014, the last year of my bachelors, I was diagnosed with Myelodysplastic Syndrome, a blood transfusion dependent rare disease causing the red blood cells production to cease. I didn’t know at that time what I would have to go through. I got blood transfusions, tried different medicines but there was no improvement. The only option I was left with was a bone marrow transplant (an expensive procedure with a lot of complications involving high doses of chemotherapy and transplantation of matched bone marrow)

I remained dependent on blood transfusions (2 bags/month) for one and a half years and was then admitted to Armed Forces Bone Marrow Transplant Centre (Combined Military Hospital) Rawalpindi, Pakistan for bone marrow transplant as my elder brother’s bone marrow matched mine. I received a high dose of chemo for 10 days and then bone marrow was transfused through the vein.

I lost my hair and my skin went dark. I came smiling but my smile had gone. I couldn’t eat and vomited whenever I tried to eat. I lost a lot of weight. Those 28 days in a small four walled room were the worst days of my life. I was finally discharged with a lot of medications and finally got to meet my parents. I had to wear a face mask and had to take care as my immunity was zero. I was prone to infections. I had to go for check-ups twice a week. I saw patients who couldn’t get a transplant as they couldn’t find a matched marrow. I saw patients lose their lives in the procedure. I saw patients whose transplant didn’t work and their condition got worse.

I received another chemotherapy as I was still transfusion dependent and they said my bone marrow was going into post-transplant pure red cell aplasia. After some days I got hemorrhagic cystitis as a side effect. There was blood in my urine and I had to go to the restroom every five minutes. Along with that, I got Cytomegalovirus Infection. I was readmitted for 20 more days. More than 30 cannulas pierced my veins. I saw salts and medications dripping through my veins all day long. I was discharged as I got better. I had lost 10 kg of my weight by that time. I used to think why I had accepted this treatment and why I was chosen for this. After some days my blood counts became better and I was no longer dependent on transfusions. With time my counts kept improving. I got my immunizations after 15 months. I also got another complication called Herpes Zoster infection. I got medications for that and I recovered from that too. Now it has been 21 months since the transplant and I have got a job in my field of Medical Laboratory Technology.

During my illness, I lost my father. His prayers kept me alive and strong and made me fight this monstrous disease.
I was lucky to have Major General Tariq Mehmood Satti as my doctor, who was very kind-hearted and caring. With time I saw many of my close friends distance themselves from me. I guess smoking was the only thing that kept us together and I gave it up before my transplant.

What I learned is that if God wants He can save you from so many complications and if He doesn’t, you can’t live for a single second.

The purpose for writing all this is solely to help those who are going through tough times in their lives and not to brag about my sufferings. Just believe in God and remember that He is the only one who can get you out of your tough times.

Man in need of bone marrow transplant finds perfect match: his brother – Fox17

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KENTWOOD, Mich. — A young man, unable to leave his home without wearing a mask, will soon be getting back to living a normal life. It’s all thanks to a bone marrow transplant. And as it turns out, the donor was closer than ever imagined.

It was his brother.

Malik Kirkwood,21, was born with sickle cell anemia, a disease where one’s body produces abnormally-shaped red blood cells that can cause a lot of pain. Malik has been through everything from blood transfusions, chemotherapy and now, this bone marrow transplant. Just three months post-surgery, Malik is already seeing change.

But right now, his immune system is compromised due to chemotherapy. Outside his home in Kentwood a sign reads: “Important Notice!! If you are sick, or have been in contact with someone who has shown symptoms of being sick, please come back another day when you are feeling healthy. This is the home of a young man recovering from a bone marrow transplant and chemotherapy. Keeping him healthy is our number one priority.”

For 21 years, Malik has endured a life filled with pain and hospital stays.

“It was pretty tough to go through, each time the pain would be different,” Malik said.

Malik in the hospital

Malik in the hospital

He hoped a bone marrow transplant would rid his body of the rare disease. But first, he’d have to find a match. To his surprise, his 17-year-old brother, Javion Kirkwood was a 100% match.

Javion said he didn’t even have to think twice.

“The doctors asked me several times do you want to do this I’m like ‘yeah,’” he said. “That’s my brother. It makes me feel good that I helped him get through the situation.”

They’re brothers by blood and friends by choice. Malik did jokingly admit, however, that he used to “bully” Javion. They both laughed.

“We hang out a lot now,” Javion said. “Years ago we were close, we just weren’t hanging out.”

Now, the two enjoy hanging out and playing video games with each other — especially now that Malik feels better than ever.

The boy’s mother, Estella Prater is overjoyed.

“They [doctors] said it would only be a 25 percent chance they would be a match,” Prater said.
“I can’t stop thanking God, I cry every day, tears of joy I still can’t believe it.”

Nor can Malik’s caregiver, Niki Smith.

“When we realized Javion was a 100% match it was a huge blessing,” Smith told FOX 17. “If Javion wasn’t a match, they would have had to search a donor bank, which would have been a much longer process.”

Thanks to a selfless act from a selfless brother, Malik’s life is forever changed.

“After the transplant, it’s been amazing,” Smith said. “He’s grown like physically he’s gotten bigger.”

Malik underwent chemo both before and after transplant. That’s the true reason his immune system is so compromised right now and will be for the next 40-something days.

We’re told that Malik now has 87 percent of his brother Javion’s cells, the rest are Maliks. The young man no longer has sickle cell disease, just sickle cell trait.

If you would like to donate to help Malik out, click here.

Doctors create ‘MAGIC algorithm’ to predict bone marrow transplant patients’ risk of dying – Science Daily

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Researchers at Mount Sinai Health System have discovered a way to predict whether blood cancer patients who received a bone marrow transplant will develop graft-versus-host disease, a common and often lethal complication, according to a study published in JCI (The Journal of Clinical Investigation) Insight.

This international study at 11 cancer centers examined blood samples from almost 1,300 bone marrow transplant patients and found that two proteins present in blood drawn a week after a transplant can predict whether a patient will develop a lethal version of graft-versus-host disease, weeks before the disease’s symptoms normally occur. Scientists at the Mount Sinai Acute GVHD International Consortium (MAGIC) created an algorithm, dubbed the “MAGIC algorithm,” that determines a patient’s risk of developing the disease by measuring concentrations of these proteins, ST2 and REG3a.

“The MAGIC algorithm gives doctors a roadmap to save many lives in the future. This simple blood test can determine which bone marrow transplant patients are at high risk for a lethal complication before it occurs,” says James L.M. Ferrara, MD, Professor of Pediatrics, Oncological Sciences and Medicine, Hematology and Medical Oncology at The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai, and Co-director of MAGIC. “It will allow early intervention and potentially save many lives.”

Doctors at Mount Sinai are now designing clinical trials to determine whether immunotherapy drugs, normally used during the onset of graft-versus-host disease, would benefit patients as soon as this new blood test determined they would be at high risk for severe onset of the disease. Researchers believe that if patients receive the drugs once the test is administered, which is well before symptoms develop, they would be spared the full force of the disease, and fewer of them would die.

“This test will make bone marrow transplant safer and more effective for patients because it will guide adjustment of medications to protect against graft-versus-host disease,” says John Levine, MD, MS, Professor of Pediatrics and Medicine, Hematology and Medical Oncology at The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai and Co-director of MAGIC. “If successful, the early use of the drugs would become a standard of care for bone marrow transplant patients.”

Graft-versus-host disease occurs when the bone marrow donor’s immune system sees the recipient’s body as foreign and launches an immune response, attacking the recipient’s tissue, primarily the skin, liver, and gastrointestinal tract. Between 40 and 60 percent of patients who receive bone marrow transplants later develop severe graft-versus-host disease, and about 40 percent of people who develop the disease die.

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Materials provided by The Mount Sinai Hospital / Mount Sinai School of Medicine. Note: Content may be edited for style and length.

New ‘MAGIC algorithm’ can predict transplant patient’s risk of developing graft-versus-host disease – News-Medical.net

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Researchers at Mount Sinai Health System have discovered a way to predict whether blood cancer patients who received a bone marrow transplant will develop graft-versus-host disease, a common and often lethal complication, according to a study published in JCI (The Journal of Clinical Investigation) Insight.

This international study at 11 cancer centers examined blood samples from almost 1,300 bone marrow transplant patients and found that two proteins present in blood drawn a week after a transplant can predict whether a patient will develop a lethal version of graft-versus-host disease, weeks before the disease’s symptoms normally occur. Scientists at the Mount Sinai Acute GVHD International Consortium (MAGIC) created an algorithm, dubbed the “MAGIC algorithm,” that determines a patient’s risk of developing the disease by measuring concentrations of these proteins, ST2 and REG3a.

“The MAGIC algorithm gives doctors a roadmap to save many lives in the future. This simple blood test can determine which bone marrow transplant patients are at high risk for a lethal complication before it occurs,” says James L.M. Ferrara, MD, Professor of Pediatrics, Oncological Sciences and Medicine, Hematology and Medical Oncology at The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai, and Co-director of MAGIC. “It will allow early intervention and potentially save many lives.”

Doctors at Mount Sinai are now designing clinical trials to determine whether immunotherapy drugs, normally used during the onset of graft-versus-host disease, would benefit patients as soon as this new blood test determined they would be at high risk for severe onset of the disease. Researchers believe that if patients receive the drugs once the test is administered, which is well before symptoms develop, they would be spared the full force of the disease, and fewer of them would die.

“This test will make bone marrow transplant safer and more effective for patients because it will guide adjustment of medications to protect against graft-versus-host disease,” says John Levine, MD, MS, Professor of Pediatrics and Medicine, Hematology and Medical Oncology at The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai and Co-director of MAGIC. “If successful, the early use of the drugs would become a standard of care for bone marrow transplant patients.”

Graft-versus-host disease occurs when the bone marrow donor’s immune system sees the recipient’s body as foreign and launches an immune response, attacking the recipient’s tissue, primarily the skin, liver, and gastrointestinal tract. Between 40 and 60 percent of patients who receive bone marrow transplants later develop severe graft-versus-host disease, and about 40 percent of people who develop the disease die.

Less invasive method of bone marrow transplant gives cancer patients more hope – KTVL

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KTVL

KTVL
“My father was diagnosed with Hodgkin’s disease at 25 years old and he was in need of a bone marrow transplant. It was at that time that they realized there weren’t many people on the registry and the odds of finding a match were very low,” Henderson said.

Aliquippa HS Football Player Needs Bone Marrow Transplant To Survive – CBS Local

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PITTSBURGH (KDKA) – Two years ago he was one of Pittsburgh’s most promising high school athletes, now an Aliquippa teen is in a fight for his life.

DiMantae Bronaugh rushed for 1200 yards and led his Aliquippa football team to the WPIAL Class AA title, but last year the teen was diagnosed with acute lymphoblastic leukemia.

KDKA first brought you DiMantae’s story last September when he was able to put on a hospital mask, and join his team along the sidelines.

A blood drive was held last October to help DiMantae.

“I didn’t think I was going to get this kind of support,” said DiMantae when he arrived at the event in his name at the Beaver United Methodist Church.
Classmates held a blood drive for DiMantae last December.

“DiMantae, the man, we love him to pieces,” said teammate Darnell Gardner. “He works hard when we lift together. You know, that’s just my friend. That’s one of my friends. That’s one of my brothers.”

After being in remission, his leukemia returned this past August, denying him the chance to return to the field with his teammates.

DiMantae’s doctors at UPMC Children’s Hospital say his only hope to survive is a bone marrow transplant from someone who shares his ancestry.

Only 7% of bone marrow registry members are black which is making it even more difficult to find a match.

Bronaugh was told by doctors they had found two tentative matches, but were devastated when the potential matches withdrew, no longer wanting to be marrow donors.

The Central Blood Bank is urging all Pittsburgh residents of black ancestry to visit a donor center where a simple swab could help save DiMantae’s life.

For more information on where you can donate, click here or call the Central Blood Bank at 412-209-7000.