JaCeon Golden has only ever known the inside of hospitals. But the treatment hes receiving may have implications far beyond his as-yet isolated life.

Round-faced and big-eyed, with a perpetual pout that belies his sunny nature, he looks as healthy as any other 5-month-old. But JaCeon was born without a functioning immune system. Even the most banal of infections a cold, a diaper rash could be deadly.

Earlier this year, JaCeon became the first baby at UCSF Benioff Childrens Hospital at Mission Bay to undergo an experimental gene therapy treatment that, doctors hope, will nudge his body to build a new, robust immune system.

From right: Dannie Hawkins checks on her nephew Ja'Ceon Golden, who is being held by patient care assistant Grace Deng at UCSF Benioff Children's Hospital on Wednesday, March 8, 2017, in San Francisco, Calif. Golden, who is five months old, is diagnosed with severe combined immunodeficiency disease (SCID). He is a patient at UCSF, where he stays in a sterile room. The hospital is working on a new gene therapy treatment for SCID. Hawkins brought her nephew Golden from New Mexico for the experimental treatment.

From right: Dannie Hawkins checks on her nephew Ja'Ceon Golden, who...

So far, his results are promising. In a few weeks, JaCeons great aunt, whos also his guardian, hopes to introduce him to the world outside.

Am I going to see him smile when we walk out of here? Dannie Hawkins, 52, said with a glance at the baby, being fed from a bottle by a nurse wearing a gown and gloves. Hows he going to do in the free world?

It will be a while months, probably years before JaCeon is able to fully integrate with that wide world: go to school and birthday parties, ride a public bus, swim in a community pool. But that those activities may be in his future at all is extraordinary.

The treatment given to JaCeon is the result of decades of research into gene therapy that included a string of striking failures that led many doctors to abandon the pursuit altogether.

Gene therapy long had been considered a potential treatment for severe combined immunodeficiency disorder, or SCID, the condition JaCeon was born with, and some other genetic syndromes. The idea is to replace a single gene thats causing trouble.

Even as many doctors gave up on the promise of gene therapy, teams of stubborn scientists kept plugging away. And a few years ago, their experiments started to work, propelled by advances in the understanding of stem cells in this case, a type called hematopoietic stem cells that live in bone marrow and are responsible for generating blood and immune cells and improved methods of delivering genetic repairs.

JaCeon Golden is treated by patient care assistant Grace Deng (center) and pediatric oncology nurse Kat Wienskowski.

JaCeon Golden is treated by patient care assistant Grace Deng...

Now human gene therapy is being tested in trials at UCLA, where a team has treated 20 children with one type of SCID, and at UCSF in collaboration with St. Jude Childrens Research Hospital in Memphis. Both trials are funded by grants from the California Institute for Regenerative Medicine, the states stem cell agency, located in Oakland.

Researchers are studying similar therapies in hopes of curing genetic syndromes like sickle cell disease. And the stem cell agency is funding gene therapy research into potential treatments for HIV, brain cancer and Huntingtons disease, among others.

Gene therapy has been shown to work, the efficacy has been shown. And its safe, said Sohel Talib, a senior science officer at the state stem cell agency. The confidence has come. Now we have to follow it up.

JaCeon was born at a hospital in Las Cruces, N.M., and diagnosed with SCID just after birth as part of a standard newborn screening. He was flown to UCSF, one of a handful of facilities with expertise in SCID, when he was 3 weeks old. His great-aunt joined him about a month later, in November.

The immune disorder is commonly known as bubble baby disease, because until fairly recently kids born with it had to live in isolation, often in plastic bubbles in hospital rooms or their own homes to protect them from infections.

Babies born with SCID have a genetic mutation that leaves their immune system unable to develop disease-fighting cells. Without treatment, most will die within a year. Since the 1970s, some babies with SCID were cured with a bone-marrow transplant. But to be effective, a perfect match was required, almost always from a sibling, and only about a fifth of kids have such a match.

Ja'Ceon Golden is held by patient care assistant Grace Deng, as Deng bottle feeds Golden at UCSF Benioff Children's Hospital on Wednesday, March 8, 2017, in San Francisco, Calif. Golden, who is five months old, is diagnosed with severe combined immunodeficiency disease (SCID). He is a patient at UCSF, where he stays in a sterile room. The hospital is working on a new gene therapy treatment for SCID. Golden was brought from New Mexico for the experimental treatment.

Ja'Ceon Golden is held by patient care assistant Grace Deng, as...

The rest could undergo a bone marrow transplant from a partial match in JaCeons case, his great-aunt was one but even when that treatment was successful, kids were left with fragile immune systems that required constant maintenance with antibiotics and other boosts.

Gene therapy, though, may prove as effective as a bone marrow transplant from a perfect match.

The procedure starts with doctors harvesting stem cells from a babys own bone marrow, usually taken from the hip. In JaCeons case, his stem cells were sent in January to St. Jude in Memphis, where scientists are perfecting the gene-therapy delivery mechanism.

Sending away JaCeons stem cells was probably the most stressful time of my life, short of my own kids maybe being born, said Dr. Morton Cowan, the lead investigator of the UCSF trial, who has worked in SCID research for more than 30 years.

JaCeons stem cells were flown east over the first big weekend of major storms in California. Flights were being canceled around the clock, and doctors only had a window of about 36 hours to get the fresh cells to the labs in Memphis.

The trip was successful, but not without a hitch. After the cells were engineered and were being sent back to California, the material for a few heart-stopping hours got lost in the mail.

In a couple of months, Cowan said, he hopes to be able to do the gene-therapy delivery at UCSF labs, avoiding the travel headaches.

For now, that still happens at St. Jude. Doctors used a virus in fact, HIV, the virus that causes AIDS to deliver the gene therapy to JaCeons stem cells. The virus is neutered, with all of the disease-causing pieces inside removed.

Whats left is a missile-like shell designed to infiltrate a cell and deliver whatever payload doctors have inserted inside in this case, a healthy gene that will restore the stem cells ability to build normal immune cells.

Back in San Francisco, the cells were infused into JaCeon via a port in his chest. Because theyre his own cells, there was no fear his body would reject them.

He did have to undergo mild chemotherapy to kill off some of his own bone marrow and make room for the re-engineered stem cells to roost, but UCSF has been developing a technique for limiting the dosage of chemotherapy given in gene therapy procedures.

JaCeon suffered no obvious side effects from either the stem cell infusion or the chemotherapy drugs, doctors said.

Hes just thriving. Hes just hes great, Cowan said. He added, We cant open the Champagne just yet, but early tests show the new gene is active, and JaCeon has had an uptick of certain immune cells.

The infusion procedure took just 20 minutes, and JaCeon slept through it, but it felt momentous nonetheless.

It had been difficult to decide to enroll JaCeon in the trial, Hawkins said. Since she was a partial match for a bone marrow transplant, she had the option of giving him the traditional and well-tested therapy.

Shed said to his doctors, So youre telling me hes a guinea pig? They told her, she recalls, If it works, he can open the door for other kids.

That night, as Hawkins slept on the decision, I kept waking up, waking up, all night long, she said. If there was a possibility he could save someone else ... she added, and then broke off in tears.

She spends about six hours with JaCeon every day, beginning each morning with a bath in sterile water, brought by nurses in special tubs. Shes constantly wiping down his toys, clothes, bedding and stuffed animals.

Ive changed a lot of diapers in my time, but this is way more complicated than with other kids, Hawkins said, demonstrating the multistep process she uses to prevent diaper rash.

Im not going to say its been easy, she said. But hes doing fine. I wouldnt have it any other way.

Erin Allday is a San Francisco Chronicle staff writer. Email: eallday@sfchronicle.com

Twitter: @erinallday

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Amid advances in gene therapy, 'bubble baby' in SF gains hope - San Francisco Chronicle

Bone Marrow Stem Cells Comments Off on Amid advances in gene therapy, ‘bubble baby’ in SF gains hope – San Francisco Chronicle | March 10th, 2017

Cedars-Sinai investigators have identified a stem cell-regulating gene that affects tumor growth in patients with brain cancer and can strongly influence survival rates of patients. The findings, published in the online edition of Scientific Reports, could move physicians closer to their goal of better predicting the prognosis of patients with brain tumors and developing more personalized treatments for them.

To enhance understanding of how glioma cancer stem cells (GCSCs) reproduce and how they affect patient survival, investigators spent three years analyzing the genetic makeup of more than 4,000 brain tumors. During their investigation, they identified the gene, called ZEB1, that regulates tumor growth. The investigators' analysis suggests that brain cancer patients who don't have the gene tend to have lower survival rates.

"Patients without the gene in their tumors have more aggressive cancers that act like stem cells by developing into an uncontrollable number of cell types," said John Yu, MD, vice chair of neurosurgical oncology in the Department of Neurosurgery and senior author of the study. "This new information could help us to measure the mutation in these patients so that we are able to provide a more accurate prognosis and treatment plan."

Brain cancer occurs when cancer cells -- also called malignant cells -- arise in the brain tissue. This year, more than 23,000 people will develop primary cancerous tumors of the brain. Approximately 16,000 of those patients will die, according to the National Cancer Institute and the American Cancer Society.

Yu and fellow researchers noted that while some brain cancer patients are born without the gene, others have it but over time, the gene has become less powerful -- which could have had a role in causing the disease.

"We found an 8 -month shorter survival rate in lower grade glioma patients with the ZEB1 gene mutation compared to those individuals who have the gene," said Yu, who also serves as director of Surgical Neuro-Oncology at Cedars-Sinai. "We are learning that some chemotherapies are not effective in the population of individuals who have the gene deletion so we have to treat them with different medications."

The study was funded by FasterCures, a center of the Milken Institute, and the National Institutes of Health, grant #NS048959.

Cedars-Sinai investigators who collaborated on the study included Keith L. Black, MD; Lincoln A. Edwards, PhD; Dror Berel; Mecca Madany; Nam-Ho Kim, PhD; Minzhi Liu; Mitch Hymowitz; Benjamin Uy; Rachel Jung; Minlin Xu; Altan Rentsendorj, PhD; and Xuemo Fan, MD, PhD.

Also contributing were researchers from Neuro-Oncology Branch, National Cancer Institute.

The Yu Laboratory focuses on immune and stem cell therapy for brain tumors, cancer stem cells and their microenvironment in brain tumors. Led by Yu, investigators have developed vaccine-based immunotherapy for brain tumors that led to multi-institutional, randomized placebo-controlled clinical trials, as well as bone marrow-derived neural stem cells for the treatment of cancer and neurodegenerative diseases.

Yu said future studies will explore specific drug regimens and their efficacy in patients with gene mutations of the stem cell gene.

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Materials provided by Cedars-Sinai Medical Center. Note: Content may be edited for style and length.

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Atlanta Falcons linebacker Paul Worrilow kisses his 15-month-old daughter, Julie, after the first day of training camp in Flowery Branch, Ga., on July 28, 2016.(Photo: Curtis Compton, Associated Press)

Height, weight: 6 feet, 230 pounds.

Joined the Lions:Worrilow, who turns 27 in May, agreed to a one-year contract with the Lions on Wednesday.

NFL career: He made the Atlanta Falcons in 2013 as an undrafted free agent after being a walk-on at Delaware. Worrilow was the Falcons' starting middle linebacker job in 2013-15. He led the team in tackles each of his first two seasons. Last season, the Falcons wanted to get faster at linebacker, so they drafted two, and Worrilow lost his job to rookie Deion Jones. Worrilow was relegated mostly tospecial teams in 2016 and played just four defensive snaps in the playoffs -- none in the Super Bowl.I know I can go and play good ball, Worrilow told the Atlanta Journal-Constitution. Whether if thats here or somewhere else.

Off the field:In 2011, he signed up for Delawares bone-marrow program. He underwent a six-hour procedure to donate peripheral blood stem cells to an anonymous 21-year-old leukemia patient.

Lions to make Ricky Wagner highest-paid RT; he's 'living his dream'

Contact Carlos Monarrez: cmonarrez@freepress.com. Follow him on Twitter @cmonarrez.

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Lions LB Paul Worrilow gave stem cells to anonymous leukemia patient - Detroit Free Press

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March 8, 2017 by Thomas Bartosh, The Conversation Cancer cells, in red, cannibalize a type of stem cell, shown in green. The red cells with small specks of green are breast cancer cells that have eaten the stem cell.

Breast cancer death rates overall have steadily declined since 1989, leading to an increased number of survivors. But while breast cancer survivors are grateful their bodies show no trace of the disease, they still face anxiety. Breast cancer can and does return, sometimes with a vengeance, even after being in remission for several years.

By studying the "cannabilistic" tendency of cancer cells, my research team has made some progress in finding out why.

The chances of recurrence and disease outcome vary with cancer subtype. About one-third of patients diagnosed with triple negative breast cancer, the most aggressive subtype, may experience a recurrence in another part of the body. This is called distant recurrence.

It has been difficult, if not impossible, to predict if and when the same cancer will recur and to stop it. Recurrent disease may arise from just a single cancer cell that survived the initial treatment and became dormant. The dormancy allowed it to hide somewhere in the body, not growing or causing harm for an unpredictable amount of time.

Determining what puts these dormant cells to "sleep" and what provokes them to "wake up" and begin multiplying uncontrollably could lead to important new treatments to prevent a demoralizing secondary cancer diagnosis.

Recently, my research team and I uncovered several clues that might explain what triggers these breast cancer cells to go dormant and then "reawaken." We showed that cell cannibalism is linked to dormancy.

How do bone stem cells affect breast cancer?

Breast cancer can recur in the breast or in other organs, such as the lungs and bone. Where breast cancer decides to grow depends largely on the microenvironment. This refers to the cells that surround it, including immune cells, cells comprising blood vessels, fibroblasts and the select proteins they produce, among other factors.

Over a century ago, a surgeon named Stephen Paget famously compared the organ-specific prevalence of cancer metastasis to seeds and soil. Because breast cancer often relapses in bones, in this metaphor, which still holds forth today, the bone marrow provides a favorable microenvironment (the "soil") for dormant breast cancer cells (the "seeds") to thrive.

Thus, a substantial amount of recent work has involved trying to determine the role in cancer dormancy of a special type of cell, called mesenchymal stem cells (MSCs). These are found in bone marrow.

MSCs in bone marrow are highly versatile. They are able to form bone, cartilage and fibrous tissue, as well as cells that support the immune system and formation of blood. They are also known to travel to sites of tissue injury and inflammation, where they aid in healing.

Breast cancer cells readily interact with MSCs if they meet in the bone marrow. They also readily interact if the breast cancer cells recruit them to the site of the primary tumor.

My research team and I recently focused on potential outcomes of these cellular interactions. We found an odd thing happens, which may provide insight into how these breast cancer cells hide for a long time.

In the laboratory setting, we produced breast tumor models containing MSCs. We also re-created the hostile conditions that naturally challenge developing tumors in patients, such as localized nutrient deficits caused by rapid growth of cancer cells and overcrowding.

We discovered that cancer cells under this duress become dormant after eating, or "cannibalizing," the stem cells.

Our analysis provided compelling data demonstrating that the cannibalistic breast cancer cells did not form tumors as rapidly as other cancer cells, and sometimes not at all. At the same time, they became highly resistant to chemotherapy and stresses imposed by nutrient deprivation.

Dormant cells are widely linked to recurrence. We hypothesize that cannibalism thus is linked to recurrence.

What is cellular cannibalism, and why is it important in cancer?

Cellular cannibalism, in general, describes a distinct phenomenon in which one cell engulfs and eliminates neighboring, intact cells.

The percentage of cancer cells that show cannibalistic activity is relatively low, but it does appear to increase in more aggressive tumors.

There are several reasons breast cancer cells would want to eat other cells, including other cancer cells. It provides them with a way to feed when nutrients are in short supply. It also provides them a way to eliminate the very immune cells that naturally stop cancer growth. Cell cannibalism might also allow cancer cells to inherit new genetic information and, therefore, new and advantageous traits.

Notably, in our study, cannibalistic breast cancer cells that ate the stem cells and entered dormancy began to produce an array of specific proteins. Many of these proteins are also secreted by normal cells that have permanently stopped dividing, or senescent cells, and have been collectively termed the senescence-associated secretory phenotype (or SASP). Although cellular senescence is a part of aging, we are now realizing that it is also important for a variety of normal bodily processes, development of embryos and injury repair in adults.

This suggests that although dormant cancer cells do not multiply rapidly or form detectable tumors, they are not necessarily sleeping. Instead, at times they might be actively communicating with each other and their microenvironment through the numerous proteins they manufacture.

Overall, this might be a clever way for dormant cancer cells to "fly under the radar" and, at the same time, modify their microenvironment, making it more suitable for them to grow in the future.

Can cell cannibalism be exploited for diagnosis and treatment?

Although our results are promising, it's important to be cautious. While there appears to be a strong correlation between cell cannibalism and dormancy, for now we do not know if it is directly linked to cancer recurrence in patients. Studies are underway, however, to corroborate our findings.

Still, the fact that breast cancer cells cannibalize MSCs is intriguing. It provides an important foundation for developing new diagnostic tools and therapies. Indeed, we currently have several ways of applying our recent discoveries.

One exciting idea is to exploit the cannibalistic activity of cancer cells to feed them suicide genes or other toxic agents, using MSCs as a delivery vehicle, like a tumor-seeking missile.

Importantly, MSCs can be easily obtained from the body, expanded to large numbers in the laboratory, and put back into the patient. Indeed, they have already been used safely in clinical trials to treat a variety of diseases due to their ability to aid in tissue repair and regeneration.

A different avenue for drug development would involve keeping dormant cells in a harmless and nondividing state forever. It might also be possible to prevent cancer cells from eating the stem cells in the first place.

In our study, we were able to block cell cannibalism using a drug that targets a specific protein inside cancer cells. With this treatment approach, the cancer might essentially starve to death or be more easily killed by conventional therapies.

Explore further: A possible explanation for recurring breast cancer

This article was originally published on The Conversation. Read the original article.

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How 'cannibalism' by breast cancer cells promotes dormancy: A possible clue into cancer recurrence - Medical Xpress

Bone Marrow Stem Cells Comments Off on How ‘cannibalism’ by breast cancer cells promotes dormancy: A possible clue into cancer recurrence – Medical Xpress | March 9th, 2017

An adult stem cell center established by the Kansas Legislature in 2013 is almost ready for its first clinical trial.

Buddhadeb Dawn, executive director of the Midwest Stem Cell Therapy Center, told legislators Tuesday that the trial will focus on treating graft-versus-host disease and will begin after final approvals from the U.S. Food and Drug Administration.

Our goal was to do this (trial) in January, but we got delayed because of different things, Dawn said during a hearing of the House Health and Human Services Committee. So we are now hoping to start it perhaps in summer.

Based at the University of Kansas Medical Center in Kansas City, the stem cell center has analyzed trials done elsewhere and hosted a clinical trial sponsored by a biotech company that uses modified stem cells from bone marrow to treat stroke.

But the graft-versus-host disease trial would be the first homegrown one.

Download the Midwest Stem Cell Therapy Center annual update to legislators.

Graft-versus-host disease is a potential complication when a patient receives a transplant of tissue, like an organ or bone marrow, from another person.

The disease occurs when transplanted tissue fights the patients natural immune system, potentially damaging the liver, skin or other areas. Its a rare illness, with about 20,000 cases in the United States each year.

Rep. Randy Powell, a Republican from Olathe, said the trial was a welcome and exciting development. He said his wife is at risk for the illness following treatment for leukemia.

I know that graft-versus-host is a big thing, Powell said. I think my wife still has an annual checkup where they keep their eye out (to make sure) thats not sticking its head up and causing issues.

Dawn said the center would like to take the next step and move into clinical trials using adult stem cells to treat things like joint ailments, diabetes and amyotrophic lateral sclerosis, also known as Lou Gehrigs disease.

But the regulatory process takes time.

Wed like to be able to offer a portfolio of different disease conditions that adult stem cells can benefit, Dawn said. Im hoping that within the next five years we would at least have some FDA approval for treatment with adult stem cells for other conditions.

Dawn said successful trials could lead to more private investment dollars so we are self-sustaining at some point in the future.

The centers reliance on state funds has been a point of contention for fiscally conservative legislators in the past. Most of the facilitys budget still comes from the states payment, which was reduced by about $28,000 to $754,500 last year.

Were maximizing every opportunity we can with what we have right now.

Thats far less than what stem cell research facilities in other states receive.

Doug Girod, executive vice president of the KU medical center, said that given the budget, Dawn and his small team have done remarkable work.

We could be 10 times bigger than we are and doing 10 times as much if we had the resources, Girod said. But I think were maximizing every opportunity we can with what we have right now.

The center was spearheaded by socially conservative legislators, including Sen. Mary Pilcher-Cook, to showcase adult stem cell research as an alternative to using stem cells derived from human embryos.

About $56,000 of its annual budget goes to educating the public about the differences between embryonic stem cells and adult cells and hosting an annual conference about advances in adult stem cell treatment.

Rep. John Wilson, a Democrat from Lawrence, said he initially was skeptical about the facility because he thought the Legislature was inserting itself into a religious or philosophical fight. But he said his attitude has changed.

Im glad that despite my opposition to it the state has gone forward with funding some really terrific research, Wilson said. My concern now is how do we take it to the next level so all of this hasnt been for nothing.

Andy Marso is a reporter for the Kansas News Service, a collaboration of KCUR, Kansas Public Radio and KMUW covering health, education and politics in Kansas. You can reach him on Twitter@andymarso. Kansas News Service stories and photos may be republished at no cost with proper attribution and a link back to kcur.org.

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Kansas Stem Cell Center Close To First Clinical Trial - KCUR

Bone Marrow Stem Cells Comments Off on Kansas Stem Cell Center Close To First Clinical Trial – KCUR | March 9th, 2017

People with a certain gene have an adverse reaction to the antiretroviral efavirenz

Liz HighleymanCorrespondent An HIV-positive bone marrow transplant recipient at the Mayo Clinic experienced prolonged viral remission lasting nearly 10 months longer than the so-called Boston patients after interrupting antiretroviral therapy (ART), according to a report at the Conference on Retroviruses and Opportunistic Infections (CROI 2017) last month in Seattle.

Although his viral load eventually rebounded, his HIV reservoirs appeared to be reduced.

The only person known to be cured of HIV Timothy Ray Brown, known as the Berlin Patient stopped ART when he received a bone marrow transplant to treat leukaemia and has not had detectable virus for 10 years. Brown received a transplant from a donor with a double CCR5-delta-32 mutation, meaning they lack the CCR5 co-receptors most types of HIV use to enter T-cells.

It is unclear whether his sustained remission is attributable to the donors CCR5 mutation, the strong chemotherapy conditioning regimen used to kill off cancerous blood cells, a graft-versus-host reaction or multiple factors.

Bone marrow transplantation is apparently not sufficient to eradicate HIV.

A few years ago, Timothy Henrich reported on two HIV-positive bone marrow transplant patients in Boston who got stem cells from wild-type donors without the CCR5-delta-32 mutation, received a milder conditioning regimen and experienced acute graft-versus-host disease (GVHD). Both men maintained undetectable viral load longer than expected after interrupting ART, but eventually they experienced viral rebound at three and eight months after stopping HIV treatment.

The latest case, presented by Nathan Cummins of the Mayo Clinic in Rochester, Minnesota, and colleagues, involved a 55-year-old man who was diagnosed with HIV in 1990 and started combination ART in 1999 with a CD4 T-cell count of 300 cells/mm3. He stopped treatment between 2004 and 2009 for unexplained reasons, then restarted ART consisting of ritonavir-boosted atazanavir (Prezista) plus tenofovir disoproxil fumarate (DF) and emtricitabine (the drugs in Truvada).

In April 2013 the man was diagnosed with B-cell acute lymphoblastic leukaemia.

In anticipation of chemotherapy, his ART regimen was switched to raltegravir (Isentress), etravirine (Intelence), and tenofovir DF/emtricitabine. In October 2013 he underwent reduced intensity conditioning followed by an allogeneic stem cell transplant from a CCR5 wild-type donor.

At the time of transplantation the man had an HIV viral load of 25 copies/ml and a CD4 count of 288 cells/mm3, and he stayed on ART without interruption. After the transplant he developed opportunistic infections (E. coli septicaemia and pneumocystis pneumonia) and experienced GVHD at four months post-transplant.

The man continued on ART for more than two years after transplantation, mostly with detectable plasma viral load levels. HIV RNA was also undetectable in gut biopsy samples. HIV DNA in his peripheral blood cells became undetectable by day 56, and repeated leukapheresis procedures showed significant reductions in HIV RNA and DNA reservoir size.

In addition, that mans HIV antibody levels decreased, as indicated by weaker Western blot bands. However, single genome sequencing and phylogenetic analysis identified identical HIV clones at day 142, possibly due to homoeostatic proliferation, or replication of latently infected cells, while he had GVHD.

After having such low HIV levels for a prolonged period, the man underwent an analytic treatment interruption, or carefully monitored discontinuation of ART. His plasma HIV RNA levels were tested every two weeks for the first 12 weeks of ART interruption, then every four weeks.

At day 288 9,6 months after stopping ART he was found to have low-level viral rebound to 60 copies/ml. This rose to 1640 copies/ml by day 293, requiring that he restart HIV treatment.

The man had no evidence of drug resistance and his viral load was re-suppressed within a month.

Allogeneic peripheral blood stem cell transplantation in the setting of HIV is associated with significant reductions in HIV reservoir size by multiple measures, including prolonged combination ART-free remission, the researchers concluded.

They added that stem cell transplantation in the setting of suppressed viral replication may be associated with loss of HIV-specific immunity, and hypothesised that immune activation in the setting of GVHD without anti-HIV specific immunity may cause homoeostatic proliferation of latently infected cells, decreasing the chance of HIV eradication. http://www.aidsmap.com.

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Researchers report positive bone marrow transplant case - The Herald

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Dr. Kenneth Pettine, a board certified retired Orthopedic Surgeon and one of the Top 50 Spine Surgeons and Specialist to Know according to Becker's Spine Review, is offering to educate the public about mesenchymal stem cell therapy in Northern Colorado. Mesenchymal stem cells are being studied by Dr. Pettine for their ability to treat orthopedic conditions such as osteoarthritis and other joint and disc related chronic neck and back pain.

(PRWEB) March 07, 2017

Dr. Kenneth Pettine, a Northern Colorado board-certified retired orthopedic surgeon, is currently researching the use of the mesenchymal stem cell and their potential in treating orthopedic conditions such as osteoarthritis or other joint and disc related issues. He's now releasing more information about what these incredible stem cells are and the exciting therapeutic opportunities of mesenchymal stem cell therapy for the interested public in Northern Colorado.

Stem cells are rare, unique cells in our body that can duplicate themselves over and over again and can continue to regenerate throughout our lives. Each particular stem cell can only become either one type or a very limited number of cell types. As we get older, we have fewer stem cells in our body, which is why we have a decreased ability to heal and avoid diseases like cancer. Introducing new stem cells to the body could help counteract the effects of degenerated joints and chronic neck and back pain.

Mesenchymal stem cell are one type of stem cell, and the one that Dr. Pettine is using to focus his clinical research on stem cell therapy. This particular type of stem cell modulates your immune system, is extremely anti-inflammatory and has many positive effects in helping your body treat autoimmune conditions. The mesenchymal stem cell is the main cell helping your body heal from orthopedic and spine injuries and helping treat degenerative conditions such as arthritis.

Almost all of this research involves mesenchymal stem cells obtained from bone marrow. Most all these studies involved obtaining the mesenchymal stem cell from a donor's bone marrow and then expanding these cells in a laboratory. The number of mesenchymal stem cells in a petri dish can be increased to result in several hundred million cells. These cells are then put into recipient animals and humans to study their ability to treat numerous autoimmune diseases such as Parkinson's, multiple sclerosis, crohn's disease, asthma, lupus, to name just a few. These cells are also being studied to treat and regrow damaged heart muscle after a heart attack. Extensive research is being conducted to evaluate the safety and efficacy of utilizing mesenchymal stem cells to treat orthopedic conditions such as osteoarthritis of your joints and disc related chronic back and neck pain.

This has exciting implications for chronic neck and back pain sufferers. These Mesenchymal stem cells could help regenerate tissue in the spine, reversing issues like degenerative disc disease. The stem cells could also decrease pain-causing inflammation and help patients recover from accidents and injuries faster.

If you are a chronic neck or back pain sufferer and would like to learn more about Dr. Pettine's research on Mesenchymal stem cell therapy, visit his website at http://www.KennethPettine.com for more information.

About Dr. Kenneth Pettine Dr. Pettine is currently the principal investigator for 18 FDA studies evaluating non-fusion spine surgery implants and stem cells for their uses in treating spine pathology. He is considered a pioneer in the field of biologics to treat orthopedic and spine pathology. He founded The Rocky Mountain Associates in Orthopedic Medicine in 1991 to offer patients a non-fusion surgical option for their neck and back pain. He co-invented the FDA-approved Prestige cervical artificial disc and the Maverick Artificial Disc. He is currently focused on educating anyone interested in learning about the use of Mesenchymal stem cell therapy. You can learn more about stem cells and Dr. Pettine at his website, http://www.KennethPettine.com.

For the original version on PRWeb visit: http://www.prweb.com/releases/2017/03/prweb14124517.htm

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Northern Colorado Doctor Kenneth Pettine Releases Information on Mesenchymal Stem Cell Therapy for Chronic Neck ... - Benzinga

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Globalnews.ca

Could you save his life? Edmonton boy needs to find stem cell match Globalnews.ca Doctors say he needs a bone marrow transplant. Now, the family is desperately searching for a stem cell match. The world needs to know that this is what we need and this can save kids' lives, Mishio said. Not just Brady's life there's other ...

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Could you save his life? Edmonton boy needs to find stem cell match - Globalnews.ca

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Rich Garton, CTV Windsor Published Tuesday, March 7, 2017 9:15AM EST Last Updated Tuesday, March 7, 2017 3:50PM EST

A Windsor infant with a rare genetic disorder has found a bone marrow match.

CTV Windsor first brought you Madalayna Ducharme's story a few months ago -- when her parents, Tamara and Charles made a plea for people to become bone marrow and stem cell donors.

It was on the heels of them learning Madalayna suffers from a rare genetic disorder called malignant infantile osteopetrosis. The side effects cause progressive vision and hearing loss, bone density and could potentially be fatal.

Doctors contacted the family this week and Ducharme is already undergoing treatment and is being prepped for the bone marrow transplant.

CTV News spoke with the family -- and they are elated with the news, but the Ducharme's excitement is tempered by a fear of what lies ahead.

We're a little scared at the same time because of the stuff she has to go through, says Tamara Ducharme. She has a regiment she has to go through before she can get this transplant.

That includes heavy doses of chemotherapy, with potential side effects, before a bone marrow transplant can take place.

Chemo is not going to be pretty, says Tamara Ducharme. She's a happy little girl, she cries once in a while like a regular child, she's going to be very ill and I don't know if I'm ready for that."

For now, they will borrow strength from their warrior princess.

Tamara Ducharme took to Facebook this morning to provide the good news. "We are sending big THANKS to all who have supported us, got swabbed, volunteered and donated blood," she posted.

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Windsor infant "warrior princess" finds a bone marrow match - CTV News

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An HIV-positive bone marrow transplant recipient at the Mayo Clinic experienced prolonged viral remission lasting nearly 10 months longer than the so-called Boston patients after interrupting antiretroviral therapy (ART), according to a report at the Conference on Retroviruses and Opportunistic Infections (CROI 2017) last month in Seattle. Although his viral load eventually rebounded, his HIV reservoirs appeared to be reduced.

The only person known to be cured of HIV Timothy Ray Brown, known as the 'Berlin Patient' stopped ART when he received a bone marrow transplant to treat leukaemia and has not had detectable virus for ten years. Brown received a transplant from a donor with a double CCR5-delta-32 mutation, meaning they lack the CCR5 co-receptors most types of HIV use to enter T-cells. It is unclear whether his sustained remission is attributable to the donor's CCR5 mutation, the strong chemotherapy conditioning regimen used to kill off cancerous blood cells, a graft-versus-host reaction or multiple factors.

Bone marrow transplantation is apparently not sufficient to eradicate HIV. A few years ago, Timothy Henrich reported on two HIV-positive bone marrow transplant patients in Boston who got stem cells from 'wild-type' donors without the CCR5-delta-32 mutation, received a milder conditioning regimen and experienced acute graft-versus-host disease (GVHD). Both men maintained undetectable viral load longer than expected after interrupting ART, but eventually they experienced viral rebound at three and eight months after stopping HIV treatment.

The latest case, presented by Nathan Cummins of the Mayo Clinic in Rochester, Minnesota, and colleagues, involved a 55-year-old man who was diagnosed with HIV in 1990 and started combination ART in 1999 with a CD4 T-cell count of 300 cells/mm3. He stopped treatment between 2004 and 2009 for unexplained reasons, then restarted ART consisting of ritonavir-boosted atazanavir (Prezista) plus tenofovir disoproxil fumarate (DF) and emtricitabine (the drugs in Truvada).

In April 2013 the man was diagnosed with B-cell acute lymphoblastic leukaemia. In anticipation of chemotherapy, his ART regimen was switched to raltegravir (Isentress), etravirine (Intelence), and tenofovir DF/emtricitabine. In October 2013 he underwent reduced intensity conditioning followed by an allogeneic stem cell transplant from a CCR5 'wild-type' donor.

At the time of transplantation the man had an HIV viral load of 25 copies/ml and a CD4 count of 288 cells/mm3, and he stayed on ART without interruption. After the transplant he developed opportunistic infections (E. coli septicaemia and pneumocystis pneumonia) and experienced GVHD at four months post-transplant.

The man continued on ART for more than two years after transplantation, mostly with detectable plasma viral load levels. HIV RNA was also undetectable in gut biopsy samples. HIV DNA in his peripheral blood cells became undetectable by day 56, and repeated leukapheresis procedures showed significant reductions in HIV RNA and DNA reservoir size.

In addition, that man's HIV antibody levels decreased, as indicated by weaker Western blot bands. However, single genome sequencing and phylogenetic analysis identified identical HIV clones at day 142, possibly due to homeostatic proliferation, or replication of latently infected cells, while he had GVHD.

After having such low HIV levels for a prolonged period, the man underwent an analytic treatment interruption, or carefully monitored discontinuation of ART. His plasma HIV RNA levels were tested every two weeks for the first 12 weeks of ART interruption, then every four weeks.

At day 288 9.6 months after stopping ART he was found to have low-level viral rebound to 60 copies/ml. This rose to 1640 copies/ml by day 293, requiring that he restart HIV treatment. The man had no evidence of drug resistance and his viral load was re-suppressed within a month.

"Allogeneic peripheral blood stem cell transplantation in the setting of HIV is associated with significant reductions in HIV reservoir size by multiple measures, including prolonged combination ART-free remission," the researchers concluded.

They added that stem cell transplantation in the setting of suppressed viral replication may be associated with loss of HIV-specific immunity, and hypothesised that "immune activation in the setting of GVHD without anti-HIV specific immunity may cause homeostatic proliferation of latently infected cells, decreasing the chance of HIV eradication."

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Researchers report case of bone marrow transplant patient off ART for 288 days without HIV rebound - aidsmap

Bone Marrow Stem Cells Comments Off on Researchers report case of bone marrow transplant patient off ART for 288 days without HIV rebound – aidsmap | March 6th, 2017

FOX31 Denver

Sickle cell anemia patient 'cured' by gene therapy, doctors say FOX31 Denver Essentially, researchers extracted bone marrow from the patient, harvested the stem cells and altered the genetic instructions so that they would make normal hemoglobin. Next, they treated the patient with chemotherapy for four days to eliminate his ... Gene therapy shows early promise against sickle cellChicago Tribune Doctors Claim They've Cured a Boy of a Painful Blood Disorder ...Futurism Will Sickle Cell Be the Next Disease Genetic Engineering Cures?Gizmodo Ligue 1 Talk -IFLScience all 65 news articles »

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Sickle cell anemia patient 'cured' by gene therapy, doctors say - FOX31 Denver

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Almost 30 years ago, the federal government helped make it easier for patients with leukemia and lymphoma to receive lifesaving stem cell transplants. Now, we need the federal governments help again to ensure that Medicare patients with these cancers and other serious blood disorders can access the care they need.

In 1987, Congress approved funding for a national database of patients willing to donate bone marrow or peripheral blood stem cells. That database is now known as the Be The Match Registry, operated by the National Marrow Donor Program/Be The Match. According to the NMDP/Be The Match, patients searching the registry have access to 27 million potential volunteer bone marrow and peripheral blood stem cell donors worldwide, along with more than 680,000 units of cord blood donated by mothers after giving birth.

Having access to such a large registry has made it easier for patients to find a match if they dont have a fully matched sibling donor, which is the case for about 70 percent of patients who receive a stem cell transplant. The registry has helped 80,000 patients receive bone marrow transplants, peripheral blood stem cell transplants, or cord blood transplants from an unrelated donor.

While the federal governments foresight and financial support have helped make adult stem cell and cord blood transplants the only cure available for these diseases possible for thousands of patients, Medicare coverage policies have not kept pace with this breakthrough treatment.

Medicare is more restrictive than private insurance companies in deciding for what indications stem cell transplants and cord blood transplants will be covered. With private insurance companies, we have the opportunity to talk with a medical director about the indication and provide literature to support the decision for a transplant. This opportunity is not available for our Medicare patients.

In most cases, Medicare doesnt decide whether to cover a stem cell or cord blood transplant until after the procedure is completed. This leaves most Medicare patients an impossible choice: Turn down their only chance at a cure or potentially face paying the significant cost of a transplant themselves. Even when Medicare does decide to reimburse for these transplants, according to the NMDP/Be The Match, it covers less than half the cost of the transplant.

Addressing this issue is especially important because seniors make up a large portion of the patients with the cancers and blood diseases that can be cured by a stem cell or cord blood transplant. For example, 24 of the 65 patients who received stem cell or cord blood transplants at University of Virginia Health System in 2016 had Medicare coverage.

So I am asking the Centers for Medicare & Medicaid Services to expand Medicare coverage for stem cell and cord blood transplants, along with paying for the search and procurement costs as they already do for solid organ transplants.

The federal government has helped save the lives of tens of thousands of patients through better access to stem cell and cord blood transplants. I hope now they will act to make sure all Medicare patients who need one of these transplants can receive it.

Tamila L. Kindwall-Keller, DO, MS is the associate clinical director of the Stem Cell Transplant Program at the University of Virginia Health System.

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Opinion/Commentary: Seniors put at risk by outdated Medicare policies - The Daily Progress

Bone Marrow Stem Cells Comments Off on Opinion/Commentary: Seniors put at risk by outdated Medicare policies – The Daily Progress | March 5th, 2017

An Edmonton police officer is counting on the city he serves to help save his sons life.

Brady Mishio, 8, has an aggressive form of leukemia and needs a bone marrow transplant. His dad Terry desperately hopes someone who is a match will hear his plea.

Bradys family received the news of his illness in November after they took him for treatment of a persistent fever and flu.

You become that family that's searching for hope and have all these dreams and things for your children, and then one day, one day it's taken away from you so quickly, said Mishio.

The diagnosis was a form of cancer that starts in blood stem cells called acute myelogenous leukemia (AML). It is the most common leukemia found in adults but is less common in children. Brady began chemotherapy right away.

Four days into chemo he had a reaction to some of the drugs that they were giving him and he quit breathing and had a seizure, said Mishio, a 20-year veteran of the Edmonton Police Service who was once forced off work for a year with a brain injury after being kicked while making an arrest.

Bradys doctors found a drug combination that worked and the boy just finished his third round of treatment. He is in an isolation unit in hospital and the next step is a bone marrow transplant.

It gives Brady a second chance at life, at cleaning out his bone marrow and hopefully eliminating the cancer, said his father, while fighting tears.

Bradys family is not a match and Mishio is reaching out publicly, hoping he can help his child by finding a suitable donor. The test is a simple mouth swab. Donors must be between 17 and 35 and be in relatively good health, says Robyn Henwood, a stem cell territory manager for Canadian Blood Services. Younger people have fewer antibodies in their blood, lowering the risk of rejection.

Once registered, potential donors stay in the agencys registry until they are 60 years old.

Less than 25 per cent of patients find a bone marrow match in their own family, says Henwood.

So every single person we add to our database is giving hope to those who are looking for a match or who are likely going to die if we don't find them one.

In most cases, the process to donate bone marrow is much like giving blood and takes four to six hours to complete.

A clinic is being held Thursday, March 9 from 4 p.m. to 8 p.m. at Holy Cross Ukrainian Catholic Church. Interested donors who cant make that clinic can be tested at the Canadian Blood Services clinic next to University Hospital or register at blood.ca to be mailed a swab kit.

Mishio is amazed by the bravery of his son through this battle.

He's a fighter and there's many days where he'll be rubbing my back and kind of telling me it's going to be OK, and that's when I'm like, 'I got to be strong for him.'

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Edmonton police officer appeals for bone marrow donor to save 8 ... - CTV News

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THE family of a man urgently in need of a bonemarrow transplant has appealed to the Asian community to donate their stem cells in the hope of finding a suitable match to save his life.

Father of two, Yevi Ilangakoon, was diagnosed with myelofibrosis in 2009. It is a rare condition where scar tissue builds up inside the bone marrow, affecting its ability to create healthy blood cells, which affects one person in every 100,000.

The 63-year-old, who is originally from Sri Lanka and now lives in Whitstable, has seen his health deteriorate rapidly and his illness could now progress into leukemia if he is not treated.

His only option is to have a bone-marrow transplant using stem cells. However, specialists have been unable to find a 100 per cent match despite searching worldwide registers.

From the entire register, only four per cent are from a south Asian background.

Yevis son Yovaan told Eastern Eye: Its a lifethreatening disease and has been managed with medication for the past eight years, but the condition has got more and more aggressive, especially over the last few months. If he doesnt have a stem-cell transplant, it will be a few months to a year that he will have to live.

So it is quite crucial that we get as close to 100 per cent match as we can. He gets very, very tired because his hemoglobin levels are low. If he has an injury, it takes ages to heal. We are praying and being positive and trying to raise awareness.

Yovaan highlighted the issue on social media, which attracted the attention of Sri Lankan cricketer Mahela Jayawardena, but the family are still urging members of the public to get on the bone marrow register to find a match for Yevi.

The 29-year-old added: It could be your family member your mum or your dad, you dont know what position you are going to be in in a few years time.

If you are on the register, you have the chance of saving someones life. Its a really easy process.

Signing up online takes two minutes and participants simply need to swab the inside of their cheek with a cotton bud they are sent, and send it back in a pre-paid envelope.

Sarah Rogers of the Anthony Nolan charity said: We urgently need more people from Indian and South Asian backgrounds to register as stem cell donors to make sure that everyone, regardless of background, can receive a second chance at life.

At the moment we find a perfect match for about 60 per cent of northern European patients who need a transplant, but that drops to around 20 per cent for any patient of ethnic minority.

If you are above 30, go to: http://www.dkms.org.uk/en/ register-now. Under 30, register at http://www.anthonynolan.org/apply-join-bone-marrow-register.

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Family appeals for bone marrow donor to save father - Easterneye (press release) (subscription)

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Scientists have created an easy way to identify the state and fate of stem cells earlier than previously possible.

Understanding a stem cells fatethe type of cell it will eventually becomeand how far along it is in the process of development can help scientists better manipulate cells for stem cell therapy.

Having the ability to visualize a stem cells future will take some of the questions out of using stem cells to help regenerate tissue and treat diseases.

The beauty of the method is its simplicity and versatility, says Prabhas V. Moghe, a professor of biomedical engineering and chemical and biochemical engineering at Rutgers and senior author of a study published recently in the journal Scientific Reports. It will usher in the next wave of studies and findings.

Existing methods look at the overall population of cells but arent specific enough to identify individual cells fates. But when implanting stem cells (during a bone marrow transplant following cancer treatment, for example), knowing that each cell will become the desired cell type is essential.

Also, many protein markers used to distinguish cell types dont show up until after the cell has transitioned, which can be too late for some applications.

To identify earlier signals of a stem cells fate, scientists used super-resolution microscopy to analyze epigenetic modifications. Epigenetic modifications change how DNA is wrapped up within the nucleus, allowing different genes to be expressed.

Some modifications signal that a stem cell is transitioning into a particular type of cell, such as a blood, bone or fat cell. Using the new method, the team of scientists was able to determine a cells fate days before other techniques.

Having the ability to visualize a stem cells future will take some of the questions out of using stem cells to help regenerate tissue and treat diseases, says Rosemarie Hunziker, program director for tissue engineering and regenerative medicine at the National Institute of Biomedical Imaging and Bioengineering. Its a relatively simple way to get a jump on determining the right cells to use.

The approach, called EDICTS (Epi-mark Descriptor Imaging of Cell Transitional States), involves labeling epigenetic modifications and then imaging the cells with super resolution to see the precise location of the marks.

Were able to demarcate and catch changes in these cells that are actually not distinguished by established techniques such as mass spectrometry, Moghe says.

He described the method as fingerprinting the guts of the cell, and the results are quantifiable descriptors of each cells organization (for example, how particular modifications are distributed throughout the nuclei).

The team demonstrated the methods capabilities by measuring two types of epigenetic modifications in the nuclei of human stem cells cultured in a dish. They added chemicals that coaxed some of the cells to become fat cells and others to become bone, while another set served as control.

Within three days, the localization of the modifications varied in cells destined for different fates, two to four days before traditional methods could identify such differences between the cells. The technique had the specificity to look at regional changes within individual cells, while existing techniques can only measure total levels of modifications among the entire population of cells.

The levels are not significantly different, but how theyre organized is different and that seems to correlate with the fact that these cells are actually exhibiting different fates, Moghe says. It allows us to take out a single cell from a population of dissimilar cells, which can help researchers select particular cells for different stem cell applications.

The method is as easy as labeling, staining, and imaging cellstechniques already familiar to many researchers, he says. As the microscopes capable of super resolution imaging become more widely available, scientists can use it to sort and screen different types of cells, understand how a particular drug may disrupt epigenetic signaling, or ensure that stem cells to be implanted wont transform into the wrong cell type.

Collaborators are from Stanford University School of Medicine, Case Western Reserve University, Seoul National University, Princeton University, the University of Akron, the University of Pennsylvania, and MIT.

Source: Teal Burrell for the National Institute of Biomedical Imaging and Bioengineering via Rutgers University

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This simple method can predict a stem cell's fate - Futurity: Research News

Bone Marrow Stem Cells Comments Off on This simple method can predict a stem cell’s fate – Futurity: Research News | March 3rd, 2017

Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

RELATED: TRENDING LIFE & STYLE NEWS THIS HOUR

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the U.S., about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant. In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team led by Dr. Marina Cavazzana focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin to replace the abnormal "adult" hemoglobin seen in sickle cell.

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published in March in the New England Journal of Medicine.

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Gene therapy shows early promise against sickle cell - Chicago Tribune

Bone Marrow Stem Cells Comments Off on Gene therapy shows early promise against sickle cell – Chicago Tribune | March 3rd, 2017

WEDNESDAY, March 1, 2017 (HealthDay News) -- Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.

The findings come from just one patient, a teenage boy in France. But more than 15 months after receiving the treatment, he remained free of symptoms and his usual medications.

That's a big change from his situation before the gene therapy, according to his doctors at Necker Children's Hospital in Paris.

For years, the boy had been suffering bouts of severe pain, as well as other sickle cell complications that affected his lungs, bones and spleen.

Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.

It's not clear how long the benefits will last, they said. And the approach obviously has to be tested in more patients.

"This is not right around the corner," said Dr. George Buchanan, a professor emeritus of pediatrics at the University of Texas Southwestern Medical Center in Dallas.

That said, Buchanan called the results a "breakthrough" against a disease that can be debilitating and difficult to treat.

Buchanan, who wasn't involved in the research, helped craft the current treatment guidelines for sickle cell.

"This is what people have been wanting and waiting for," he said. "So it's exciting."

Sickle cell anemia is an inherited disease that mainly affects people of African, South American or Mediterranean descent. In the United States, about 1 in 365 black children is born with the condition, according to the U.S. National Heart, Lung, and Blood Institute.

It arises when a person inherits two copies of an abnormal hemoglobin gene -- one from each parent. Hemoglobin is an oxygen-carrying protein in the body's red blood cells.

When red blood cells contain "sickle" hemoglobin, they become crescent-shaped, rather than disc-shaped. Those abnormal cells tend to be sticky and can block blood flow -- causing symptoms such pain, fatigue and shortness of breath. Over time, the disease can damage organs throughout the body.

There are treatments for sickle cell, such as some cancer drugs, Buchanan pointed out, but they can be difficult to manage and have side effects.

There is one potential cure for sickle cell, Buchanan said: a bone marrow transplant.

In that procedure, doctors use chemotherapy drugs to wipe out the patient's existing bone marrow stem cells -- which are producing the faulty red blood cells. They are then replaced with bone marrow cells from a healthy donor.

A major problem, Buchanan said, is that the donor typically has to be a sibling who is genetically compatible -- and free of sickle cell disease.

"We've known for a long time that bone marrow transplants can work," Buchanan said. "But most patients don't have a donor."

That's where gene therapy could fit in. Essentially, the aim is to genetically alter patients' own blood stem cells so they don't produce abnormal hemoglobin.

In this case, the French team, led by Dr. Marina Cavazzana, of Necker Children's Hospital's biotherapy department, focused on a gene called beta globin. In sickle cell anemia, beta globin is mutated.

First, the researchers extracted a stem cell supply from their teen patient's bone marrow, before using chemotherapy to wipe out the remaining stem cells.

Then they used a modified virus to deliver an "anti-sickling" version of the beta globin gene into the stem cells they'd removed pre-chemo. The modified stem cells were infused back into the patient.

Over the next few months, the boy showed a growing number of new blood cells bearing the mark of the anti-sickling gene. The result was that roughly half of his hemoglobin was no longer abnormal.

In essence, Buchanan explained, the therapy "converted" the patient to sickle-cell trait -- that is, a person who carries only one copy of the abnormal hemoglobin gene. Those individuals don't develop sickle cell disease.

"This is encouraging," said Dr. David Williams, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center.

But, he cautioned, "the caveat is, this is one patient, and 15 months is a short follow-up."

Williams and his colleagues are studying a different approach to sickle cell gene therapy. It aims to restart the body's production of healthy fetal hemoglobin -- to replace the abnormal "adult" hemoglobin seen in sickle cell.

The hope, Williams said, is that gene therapy will ultimately offer a one-time treatment that cures sickle cell. But no one knows yet whether that will happen.

According to Williams, two key questions are: What's the long-term safety? And will the altered stem cells last for a patient's lifetime?

If gene therapy is proven to work, there will no doubt be practical obstacles to its widespread use, according to Buchanan. It's a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.

But, Buchanan said, the new findings have now "opened a door."

The study was partly funded by Bluebird Bio, the company developing the therapy.

The results were published March 1 in the New England Journal of Medicine.

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Gene Therapy: A Breakthrough for Sickle Cell Anemia? - Auburn Citizen

Bone Marrow Stem Cells Comments Off on Gene Therapy: A Breakthrough for Sickle Cell Anemia? – Auburn Citizen | March 2nd, 2017

News

by DAVID JENSEN posted 03.02.2017

The California stem cell agency this week approved nearly $33 million for clinical stage research projects testing treatments for type 1 diabetes, arthritis of the knee, ALS and an immunodeficiency affliction.

The awards were quickly approved with little discussion during a meeting at the Oakland headquarters of the California Institute for Regenerative Medicine or CIRM, as the agency is formally known.

The goal of the research is to regenerate knee cartilage through the use of a mesenchymal progenitor cell treatment, according to the agencys application review summary

The award likely to have an impact on the most people if it is successful is a relatively small, $2.3 million award to the Cellular Biomedicine Group, a Chinese firm with operations in Cupertino, Calif. The stem cell agency by law only finances work in Clifornia. The research would also be supported by $572,993 in co-funding.

The project is aimed at treating osteoarthritis of the knee. More than 51 million people in the United States suffer from arthritis, which is particularly common in the knee.

The goal of the research is to regenerate knee cartilage through the use of a mesenchymal progenitor cell treatment, according to the agencys application review summary. The funding would go to manufacture the product and complete work to secure Food and Drug Administration approval for a phase one safety trial. A treatment for the public would likely be years in the future.

Here are the other winners today of California stem cell cash with links to the summaries of the reviews.

Caladrius Biosciences of New Jersey won $12.2 million for a clinical trial for young people ages 12-17 for newly diagnosed type 1 diabetes. The firm plans to use regulatory T cells from the patients themselves to treat the disease. Caladrius has a California location in Mountain View. (Caladrius press release can be found here.)

St. Judes Research Hospital in Memphis, Tenn., was awarded $11.9 million for a phase one/two trial to treat infants with X-linked severe combined immunodeficiency. The trial would aim at enrolling at least six patients suffering from the catastrophic affliction. The treatment would use the patients own bone marrow stem cells after the cells were specially handled. The agency said in a press release that St. Judes is working with UC San Francisco. (St. Judes press release can be found here.)

The awards were previously approved behind closed doors by the agencys out-of-state reviewers, who do not disclose publicly their economic or professional interests.

Cedars-Sinai Medical Center in Los Angeles was awarded $6.2 million for a phase 1/2A trial to test a treatment for ALS, which has no treatment or cure. The CIRM review summary said a huge unmet need existed. About 20,000 persons in the United States suffer from the affliction.

CIRMs press release did not identify the researchers involved in any of the awards.

The agency is on a push to support more clinical trials, which are the last and most expensive research prior to the possibility of winning federal approval for widespread use of a therapy.

Currently the agency is participating in 27 trials and is planning on adding 37 more in the next 40 months. The agency is expected to run out of funds for new awards in June 2020 and has no source of future financing.

The awards were previously approved behind closed doors by the agencys out-of-state reviewers, who do not disclose publicly their economic or professional interests. The agencys directors rarely overturn a positive decision by the reviewers.

All of the winners have links to two or more members of the 29-member CIRM governing board. Those members are not allowed to vote on applications where they have conflicts of interest.

About 90 percent of the funds awarded by the board since 2005 have gone to institutions that have ties to members of the board, past or present, according to calculations by the California Stem Cell Report. Eds Note: David Jensen is a retired newsman who has followed the affairs of the $3 billion California stem cell agency since 2005 via his blog, the California Stem Cell Report, where this story first appeared. He has published more than 4,000 items on California stem cell matters in the past 11 years.

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Sickle cell disease. Image: Flickr

Sickle cell disease is a slow, vicious killer. Most people diagnosed with the red blood cell disorder in the US live to be between 40 and 60. But those years are a lifetime of pain, as abnormal, crescent-shaped hemoglobin stops up blood flow and deprives tissues of oxygen, causing frequent bouts of agony, along with more severe consequences like organ damage. Now, after decades of searching for a cure, researchers are announcing that, in at least one patient, they seem to have found a very promising treatment.

Two years ago, a French teen with sickle cell disease underwent a gene therapy treatment intended to help his red blood cells from sickling. In a paper published Thursday in the New England Journal of Medicine, the researchers revealed that today, half of his red blood cells have normal-shaped hemoglobin. He has not needed a blood transfusion, which many sickle cell patients receive to reduce complications from the disease, since three months after his treatment. He is also off all medicines.

To reiterate, the paper is a case study of just one patient. Bluebird Bio, the Massachusetts biotech company that sponsored the clinical trial, has treated at least six other trials underway in the US and France, but those results have not yet been fully reported. The gene therapy has not worked quite as well in some of those other patients; researchers say they are adjusting the therapy accordingly. It is also possible that the boy may eventually experience some blood flow blockages again in the future.

The results, though early, are encouraging. They represent the promise of new genetics technologies to address a disease that has long been neglected and tinged with racism. Sickle cell disease affects about 100,000 people in the US, most of whom are black. It is an inherited genetic disease caused by a mutation of a single letter in a persons genetic code.

This single-letter mutation makes it a promising candidate for cutting edge technologies, like the gene-editing technique CRISPR-Cas9, and other gene therapies. Recently, a rush of new research has sought to address it. Two other gene therapy studies for sickle cell are underway in the US one at UCLA and another at Cincinnati Childrens Hospital. Yet another is about to start in a collaboration between Harvard and Boston Childrens Hospital. Last fall, researchers all demonstrated the ability to correct the mutation in human cells using CRISPR, though that strategy will yet have to surpass significant scientific and political hurdles before reaching clinical trials.

In the new study, researchers took bone marrow stem cells from the boy and fed them corrected versions of a gene that codes for beta-globin, a protein that helps produce normal hemoglobin. The hope was that those altered stem cells would interfere with the boys faulty proteins and allow his red blood cells to function normally. They continued the transfusions until the transplanted cells began to produce normal-shaped hemoglobin. In the following months, the numbers of those cells continued to increase until in December 2016, they accounted for more than half the red blood cells in his body. In other words, so far so good.

Currently, the only long-term treatment for sickle cell disease is a bone marrow transplant, a high-risk, difficult procedure which many patients are not even eligible for. Pain and other side-effects are treated with blood transfusions for temporary relief. New technologies offer the hope of a solution that could provide long-term relief and allow patients to live some semblance of a normal life.

For decades, gene therapies have been touted as a cure for everything. But so far, successes have been infrequent, and often for very rare diseases. But early success in treating sickle cell disease means that soon, if were lucky, the benefits of this technology may reach hundreds of thousands of people.

[New England Journal of Medicine]

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NBC-5 news anchor Rob Stafford has been diagnosed with a rare blood disorder and will take a leave of absence from the station for several months as he undergoes a bone marrow transplant and chemotherapy.

Stafford credited his wife, Lisa, for pushing him to get an early diagnosis that doctors say greatly improved his prognosis. He is scheduled to begin treatment Friday at the Mayo Clinic in Rochester, Minn.

"I spent my whole life asking questions, but I asked very few questions about my own health because all I wanted to hear is I'm OK," Stafford said in an interview at his Hinsdale home Tuesday. "You have to ask questions."

Stafford told colleagues at WMAQ-Ch. 5 about his illness in an email Wednesday morning and planned to announce the news publicly Wednesday at the end of the 10 p.m. newscast.

He expects to be away from his desk anchoring the 5, 6 and 10 p.m. weekday news for four to six months while at the Mayo Clinic. Doctors there say Stafford is in the early stages, Stage 2, of the disease, which can lead to heart failure and death if undetected.

Stafford's disorder, called amyloidosis, occurs when an abnormal protein called amyloid is produced in bone marrow that can be deposited in tissues and organs. There are more than 40 types of the disorder that affects the heart, kidneys, liver, spleen, nervous system and digestive tract. Stafford's type known as light chain amyloidosis is rare, with fewer than 2,000 or so cases diagnosed in the U.S. each year, according to Dr. Ronald Go, Stafford's hematologist at the Mayo Clinic. Doctors are optimistic he will go into remission after treatment.

Before his diagnosis in January, Stafford had noticed lower energy levels while biking the Cal-Sag trail and hiking through the Rocky Mountains while on vacation. But the 58-year-old award-winning investigative reporter and father of three adult children dismissed the episodes as signs of getting older, he said.

After a physical detected slightly high cholesterol levels and more than usual amounts of protein in his urine, both indicators that his kidney had been damaged as a result of the blood disease, Stafford's wife insisted that he take the symptoms seriously.

"I believe there's a reason that this happened, and I think there's a calling that is to let people know the importance of early detection," said Lisa Stafford, his wife of 30 years who owns a medical marketing and communications company. She plans to stay with Stafford in temporary housing near the Mayo Clinic as he undergoes treatment, lives in a sterile environment and rebuilds his immune system while recovering from the effects of chemotherapy.

In his email to fellow staffers, Stafford joked that he did not wait for the end of sweeps to schedule treatment, but that Friday was the first opening on the Mayo schedule after Stafford completed all the required medical tests.

"I consider this early diagnosis a gift that left to my own devices I would not have received," Stafford wrote. "I'm going to take full advantage of my good fortune and hit this head on with the most aggressive and proven treatment available."

Stafford joined NBC-5 in 2009 after working as a Chicago-based correspondent for Dateline NBC for 11 years. Prior to that, he was a general assignment reporter at CBS2 Chicago. He has won two national and seven local Emmy awards, and an Edward R. Murrow award for a Dateline investigation into racial profiling.

Stafford said his experience meeting and interviewing people in the news, often "on their worst days," has allowed him to keep his health issues in perspective.

"This thing pales in comparison to 95 percent of the stories I do on a daily basis," said Stafford, who added that his work as a journalist also has helped him to seek out the best support, medical opinions and advice about the disorder.

During treatment, doctors will remove, or "harvest," stem cells from Stafford's own bone marrow and freeze millions of healthy ones. Chemotherapy then will be used to wipe out all of his bone marrow, including the unhealthy cells. As the final part of the treatment, doctors will transplant the healthy stem cells back into Stafford's bone marrow, and they will reproduce themselves, Go said.

"You're rescuing your bone marrow by the stem cells that you stored," Go said.

Although Stafford's parents were both treated for cancers in their 50s, doctors do not know the exact cause of Stafford's illness and do not believe it was is hereditary. Risk factors for amyloidosis include exposure to chemicals, radiation and aging, Go said.

Stafford confided in his NBC-5 co-anchor, Allison Rosati, and meteorologist Brant Miller about his illness shortly after his diagnosis in January. As medical tests forced him to travel, he alerted NBC-5 managers, who encouraged Stafford to do whatever it took to get the medical attention needed, he said.

"Rob is loved by his colleagues in the newsroom. We are encouraged by the news that his illness was detected early, and that he is in the excellent hands of the top doctors at the Mayo Clinic. We all wish Rob the best of luck in the weeks ahead, and we can't wait for his return to the newsroom," station manager and vice president of news Frank Whitaker said in an email.

NBC-5 weekend anchor Dick Johnson will be filling in for Stafford during his absence.

Stafford is expected to lose his hair as a result of the chemotherapy and has experienced weight loss. Doctors have encouraged him to pack on a few pounds in anticipation of further weight loss. He's obliged by indulging on Girl Scout cookies, deep dish pizza, cheeseburgers and milk, he said.

Stafford said he is trying to take emotion out of his upcoming battle, which he is approaching as a fight that he plans to win.

"I am not freaking out because I really am confident I am going to get through this," he said.

Lisa Stafford will be posting updates about her husband's condition on a blog: staffordrecovery.com. Rob Stafford also will offer updates on his Facebook Fan page.

Stafford's announcement came less than 24 hours after Hosea Sanders, veteran news anchor at WLS-Ch. 7, announced on his Facebook page he will undergo surgery for prostate cancer and that he was "very optimistic about the outcome."

Sanders told social media friends Tuesday night that he had been diagnosed several weeks ago and would be taking some time off from the ABC-owned station, for which he anchors the 7 p.m. newscast each weeknight.

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