UC Anschutz startup gets $6M boost to fight disease with stem cells – BusinessDen
By Sykes24Tracey
Screenshot of Taigas website.
A startup at University of Colorado Anschutz is in the middle of a substantial capital raise.
Taiga Biotechnologies, which is developing new therapies for cancer, HIV and other diseases, has raised $6 million and is looking for an additional $14 million, according to a recent SEC filing.
The date of the first sale was March 16, and so far, the startup has 14 individual investors.
Founded in 2006, Taiga creates therapies for cancer, immune diseases and other serious medical conditions using stem cells, proteins and other molecular compounds.
In 2012, the firmreceived a patent to produce significant amounts of adult blood stem cells using blood from umbilical cords or bone marrow. Blood samples could be stored and expanded to be used after chemotherapy or radiation treatment, instead of having multiple bone marrow transplants.
Last summer, Taiga developed a product to help children with severe immune deficiencies, forcing them to live in protected and sterile environments. The product, which garnered an Orphan Drug Designation from the Food and Drug Administration, was approved for clinical trial in Israel.
Taiga is led by co-founders Brian Turner and Yosef Refaeli.
The company received $12 million in a raise ending in 2015, as well as $246,000 in 2010, according to SEC filings.
Taiga is basedat 12635 E. Montview Blvd. at the University of Colorado Anschutz Medical Campus.
Kate Tracy is a BusinessDen reporter who covers nonprofits, startups and the outdoors industry. She is a graduate of Corban University. Email her at kate@BusinessDen.com.
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UC Anschutz startup gets $6M boost to fight disease with stem cells - BusinessDen
Improving Blood and Marrow Transplantation – Seton Hall University News & Events
By Dr. Matthew Watson
Friday, March 31, 2017
By Nicholas Snow
The Rose Mercadante Chemistry Seminar Series is pleased to present a seminar entitled "Improving Blood and Marrow Transplantation" by Dr. Robert Korngold of the John Theurer Cancer Center, Hackensack University Medical Center.
The seminar will be held on Tuesday April 4, 2017 at 5:45 p.m. in the Helen Lerner Amphitheater, McNulty Hall, Science and Technology Center, Seton Hall University.
Dr. Korngold specializes in basic science and translational research in the field of blood and marrow stem cell transplantation. In 1978, he demonstrated in mouse models that mature T cells in donor bone marrow were responsible for causing graft-versus-host disease (GVHD) directed to minor histocompatibility antigens in transplanted recipients. This landmark study had significant impact on the future course of clinical treatment for patients undergoing transplantation from matched sibling or unrelated matched donors. Since then he has devoted his career to studying the immunological mechanisms of GVHD and refining the hematopoietic stem cell transplantation process to avoid disease and allow for enhanced anti-leukemia immune reactivity. He is widely recognized as a leading researcher in his field and as such he has served since 2001 as Editor-in-Chief of the journal Biology of Blood and Marrow Transplantation. Dr. Korngold is an author of 140 research articles, reviews and book chapters.
The Department of Chemistry and Biochemistry offers BS, MS and PhD degrees with specializations in all areas of chemistry. Our unique research environment, including traditional full-time students and part-time students is designed to foster collaborations with industry and colleagues in other disciplines. The Rose Mercadante Seminar Series is named for Rose, our departmental secretary for over 40 years, in honor of our alumni, her "boys and girls".
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Improving Blood and Marrow Transplantation - Seton Hall University News & Events
Donating the umbilical cord could save someone’s life – WNDU-TV
By raymumme
After a baby is born and the umbilical cord is cut, ever wonder where that umbilical cord ends up?
Most of the time, it becomes waste but that cord still has some valuable resources that can save a life.
The blood that is found in it is called umbilical cord blood or cord blood for short.
It contains all the normal elements of blood, such as red and white blood cells. It is also jam packed with stem cells, similar to the ones found in bone marrow.
Birth is pretty exciting, its pretty dramatic. A lot of things are happening, says James E. Baumgartner, M.D., Pediatric Surgeon.
One of those things that people rarely hear about is the option to donate cord blood. Bone marrow and cord blood contain the same type of stem cells, but those from cord blood have more advantages. Since stem cells from cord blood are less mature than stem cells from an adult's bone marrow, a recipient's body is less likely to reject them.
Another benefit is that taking cord blood is less invasive than a bone marrow transplant. Once an umbilical cord is clamped, it is wiped with antiseptic and a needle is inserted into one of the veins to withdraw a few ounces of blood. The procedure takes just a few minutes and is painless.
We all collect prospective data to look for risk for, you know, lung damage, kidney damage, liver damage, heart damage. Were looking at the nervous system pretty carefully and we found nothing. So that we really believe that its safe, explains Baumgartner.
About 70% of patients who need a stem cell transplant dont have a matching donor in their own family, which leads to the main advantage of cord blood. Stem cells from cord blood dont need to be exactly matched to the patient like bone marrow transplants from adult donors. One drawback to cord blood though is that the number of stem cells available is relatively small. This means young children will benefit because they need less.
Families can either save cord blood for themselves or donate it to a bank.
You need to talk to your doctor at least three months before your due date to find out if you are eligible to donate cord blood.
CORD BLOOD TREATMENT SAVES LIVES REPORT #2401
BACKGROUND: A stem cell transplant is a treatment that is used to treat cancers that affect blood and immune system like leukemia, multiple myeloma, and some types of lymphoma. Stem cell transplants are used to treat these types of cancer since the stem cells that the body naturally produces most often die due to treatments like radiation and chemotherapy. Human beings need stem cells to survive, therefore, a stem cell transplant gives patients blood cells that they cant produce anymore. Furthermore, donated cells can often find and kill the cancerous cells better than the patients own cells. Stem cells include:
* Red blood cells (RBCs) * White Blood cells (WBCs) * Platelets (Source: http://www.cancer.net/navigating-cancer-care/how-cancer-treated/bone-marrowstem-cell-transplantation/what-stem-cell-transplant-bone-marrow-transplant & https://www.cancer.org/treatment/treatments-and-side-effects/treatment-types/stem-cell-transplant/why-stem-cell-transplants-are-used.html)
CORD BLOOD: In the past, the only location where stem cells could be taken for a transplant was in the bone marrow. In recent years cord blood, the blood that is found in the umbilical cord, has been used for stem cell transplants. They possess the same quantity of stem cells as the bone marrow, and they come with more advantages. To start off, no surgery is needed like with bone marrow. Since the umbilical cord is natural in every birth, the mother can choose to donate her cord around three months before she is due. Once the cord is clamped, it is cleaned with antiseptic. Later, a needle is inserted into one of the veins in order to gather the necessary blood. Furthermore, since the cord blood stem cells are less mature than those stem cells from an adults bone marrow, the recipients body is less likely to reject the transplant. This is very important for people with ethnic backgrounds. With bone marrows stem cells, the match between the donor and the recipient has to be 8/8; with cord blood cells, on the other hand, the match can be partial. For recipients that come from an ethnic background, a perfect match can be harder to find. (Source: http://www.nationalcordbloodprogram.org/qa/what_are_advantages.html)
PROS & CONS: Other advantages that come with core blood cells are the association of lower incidence of GvHD (Graft vs. Host Disease), and the lower risk of viral infections. Nevertheless, the cord blood cells have a drawback: the amount of stem cells found in them is very small. Because of the low number, children benefit from this transplant procedure more than adults. Since childrens bodies are smaller, they need fewer cells for their body to start reproducing them naturally. On the other hand, adults naturally need more cells than the ones the cord blood produces because of their size. (Source: http://www.nationalcordbloodprogram.org/qa/how_is_it_collected.html)
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Donating the umbilical cord could save someone's life - WNDU-TV
WSC’s first-ever Bone Marrow Drive – The Wayne Stater
By LizaAVILA
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The National Bone Marrow registry took place in the Bluestem Room last Friday. This donor drive was hosted by Love Your Melon and Cardinal Key, with Gail Chism and Mary Kelly acting as representatives from Be The Match as well. On average, one person in 430 is called to donate, but the likelihood of being called also depends on the race of the donor. In total, 57 donors were added to the registry by the end of the event.
Thadd Simpson
Thadd Simpson
The National Bone Marrow registry took place in the Bluestem Room last Friday. This donor drive was hosted by Love Your Melon and Cardinal Key, with Gail Chism and Mary Kelly acting as representatives from Be The Match as well. On average, one person in 430 is called to donate, but the likelihood of being called also depends on the race of the donor. In total, 57 donors were added to the registry by the end of the event.
March 29, 2017 Filed under News
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Fifty-seven students registered to give DNA at the first-ever Bone Marrow Drive at Wayne State College. The drive was in the Bluestem Room of the Kanter Student Center on Friday. That puts WSC at 279 students on the bone marrow registry when combined with MAZE. The drive was put on by Be The Match, a nonprofit organization that helps people diagnosed with diseases such as leukemia and lymphoma to get them the blood that could save their life. Be The Match is operated by the National Marrow Donor Program. We want to get Wayne State on the bone marrow registry, said student Kelsi Anderson said, who runs the Love Your Melon group on campus. A donor can give someone battling blood cancer a second chance. Its crucial for them to have a donor. Those who registered simply gave a cheek swab of their DNA, which will be analyzed to determine if it matches with someone who needs a bone marrow transplant. Its all about the DNA makeup, said Gail Chism of Be The Match. The DNA needs to be as close as possible. A donor could have closer DNA to the patient than a family member. If a match is made, the donor will be sent somewhere local for the bone marrow transplant. A courier will then take the bone marrow to the patient, who could be anywhere in the country. Eighty percent of the time it is like giving plasma, Chism said. Anderson said that in other cases a needle is injected into the pelvic bone todraw the marrow out. Blood cancers such as leukemia and lymphoma produce abnormal blood cells, other than the normal red blood cells, white blood cells and platelets. Blood cells develop from stem cells in bone marrow. A bone marrow transplant helps the patient produce more normal blood cells that help the body with functions such as fightingoff infections or preventing serious bleeding. Anderson said the drive was a shared idea between herself and Jaelyn Lewis, the leader of Cardinal Key. They hope it will become an annual event in the future. I really appreciate what Kelsi has done, Chism said. Shes really been on it. It takes great leadership to put this together. What we get out of here today is priceless.
Thadd Simpson WSC student Lily Roberts swabs her mouth in order to join the National Bone Marrow registry in the Bluestem Room last Friday.
Tags: 2015, Be The Match, Cardinal Key, Gail Chism, Kelsi Anderson, Love Your Melon, NE, Neb., Nebraska, Nebraska State College System, Spring 2015, Thadd Simpson, The Wayne Stater, Wayne, Wayne State College, Wayne State Wildcats, Wildcats, WSC, WSC Cats, WSC Wildcats, WSCs first-ever Bone Marrow Drive
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WSC's first-ever Bone Marrow Drive - The Wayne Stater
Maxwell Airman donates to stranger in need – Maxwell-Gunter Air Force Base
By raymumme
MAXWELL AIR FORCE BASE, Ala. --
With an upcoming permanent change of station, Maj. Mathew Carter, a Jeanne M. Holm Center for Officer Accessions and Citizen Development instructor at Air University, had hundreds of reasons to say no when he was asked to travel to Washington, D.C., to donate bone marrow to a complete stranger. Instead, he decided this opportunity was too important to loose.
Over the weekend of March 25, he underwent a bone marrow extraction procedure in the hope of helping a 7-year-old child he never met.
This story begins in 2003 when he registered with the Department of Defenses Salute to Life program.
Salute to Life, also known as the C.W. Bill Young Department of Defense Marrow Donor Recruitment and Research Program, was initiated in 1991 and is tailored to work exclusively with military members. Over time, the program has recruited more than 1 million donors.
Carter had registered for the program while his father was in the Army. His father was stationed at Ft. Sam Houston, Texas. Carter and his family attended a bone marrow drive held for their neighbor who was diagnosed with cancer.
After 14 years, he received not only a letter, but an email and a voicemail from the organization informing him of a match.
I was kind of caught off-guard by it. It was one of those things that had a lot of opportunities for me to say no, but I have a 5-year-old, and when they told me there was a child that has a very serious life-threatening disease, there was really no question. It was the right thing to do, so I said yes, said Carter.
Carter began the process in early February by being tested again to confirm the match and getting a physical. By late March he was ready for the procedure.
During the bone marrow extraction, the patient is under local anesthesia while the doctor uses a needle to remove the marrow from the back of the pelvic bone.
Initially before [the surgery] started I was a little anxious. Before you go into any surgery you get a little anxiety, but it was one of those things that I was ready to just do, he said. Afterward, it was just relief knowing that I had done all that I could possibly do to help this person out.
He compared bone marrow donation to other bodily donations in the sense that when you donate other organs, they are permanently removed. However, with bone marrow or stem cells, the body regenerates what is lost.
For the two to four weeks of being sore and tired after the procedure, you look at what the recipient is going through, and it pales in comparison, so having the opportunity to do something like that is just amazing, he said.
For the donor and the recipients safety, they are left completely anonymous.. Once a year has passed after the surgery, they are then given the choice to reach out to each other.
When asked what he would say to the child, he thought for a moment and said, Live your life to the fullest.
Carter hopes that through this experience he can help raise awareness about bone marrow and stem cell donation, and encourages other to sign-up as donors.
Youll be a little sore and tired, but have the opportunity to do something amazing, he said.
For more information about bone marrow or stem cell donation through Salute to Life, visit http://www.salutetolife.org.
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Maxwell Airman donates to stranger in need - Maxwell-Gunter Air Force Base
Lifesaving donor needed for TV debt collector Delroy – The Voice Online
By daniellenierenberg
Image Text:
APPEAL: Delroy Anglin, star of TV's 'Cant' Pay? We'll Take it Away!' (photo credit: DCBL)
DELROY ANGLIN, a star bailiff of the TV series, 'Cant Pay? Well Take it Away!' has launched an appeal to encourage more African and Caribbean people living in the UK to join the Stem Cell Register.
The #Match4Delroy appeal which is to be led by blood cancer charity, the African Caribbean Leukaemia Trust (ACLT) is hoping to find an unmatched donor for Delroy who requires a lifesaving stem cell, specifically, a bone marrow transplant if he is to beat his battle with leukaemia.
Delroy, aged 56, was diagnosed with Acute Myeloid Leukaemia (AML) last November. Since his diagnosis, Delroy has managed with drugs and receiving two rounds of chemotherapy, but tests show the leukaemia remains. Doctors have confirmed Delroy will need an urgent stem cell transplant to beat the illness.
With siblings having a one in four chance of being a match, it was no surprise out of Delroys five siblings, none were found to be a match to help their brother.
The pain and anguish of dealing with a loved one being diagnosed with blood cancer is something Delroy and his family are all too familiar with, as it was 40 years ago, Delroys brother lost his battle against leukaemia which makes Delroys illness that much more painful for his loved ones to deal with.
Delroys sister Janet Hills, who is Chair at the Met Black Police Association (MBPS) and President at National Black Police Association, or NBPA, said:
When I tell people that Delroy from Cant Pay? Well Take it Away! is my brother, there is an immediate outpouring of warmth and love. I'm praying this appeal turns that love into action. FAMILY: Janet Hills of the Met Black Police Association and Delroy Anglin's sister (photo credit: David Sillitoe/The Guardian) ACLT is the preferred charity for the MBPS and the NBPA. Our members continually engage with the charity and organise community events to raise awareness and funds. If you love Del on the show as much as I love him as my brother, then please, please, please make that commitment today to join the stem cells (bone marrow) register.
Delroy is being supported by his loving family which includes his children and mother. His daughter Domenique Anglin said:
Dad is an active, charismatic person, he loves socialising with his family and friends. He is a fantastic father to my siblings and a wonderful grandfather too. I am appealing on his behalf to all Caribbean and African people in the UK and abroad to join the register, in the hope they might be the match that saves his life.
Anglin said:
Its going to be difficult to find me a perfect matched donor unless we have a lot more Caribbean and African people on the register. It takes 15 minutes to register and is almost painless to donate. I want to beat this illness, but I will only be able to do so, with the help of the Caribbean and African community.
I am requesting for more people of Caribbean and African heritage to join the register to help me and others like me.
Beverley De-Gale, ACLT co-founder said:
Delroys life has done a complete 360 in the last four months. From being on a popular documentary series to being diagnosed with a life-threatening illness. We hope fans of the show come together and help save the life of the individual they have come to love on-screen, in addition to Joe-public who dont watch the show.
If youre 16 55 and in good health, you could potentially be the person to save Delroys life.
You could be a #Match4Delroy. Join the Stem Cell Register now, by clicking here.
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Lifesaving donor needed for TV debt collector Delroy - The Voice Online
Cellect Announces Successful First Cancer Patient Stem Cell Transplant – GlobeNewswire (press release)
By daniellenierenberg
March 27, 2017 07:02 ET | Source: Cellect Biotechnology Ltd.
Cellects technology, ApoGraft, aims to become a game changerin stem cells transplantations for cancer treatments
Company gets green light from DSMB Board for enrolling additional 2 cancer patients for ApoGraft transplantation treatments
TEL AVIV, Israel, March 27, 2017 (GLOBE NEWSWIRE) -- Cellect Biotechnology Ltd. (Nasdaq:APOP) (TASE:APOP), a developer of stem cell selection technology, announced today that the first stem cell transplant procedure has been successfully performed using its ApoGraft technology in the Companys Phase I/II clinical trial in a blood cancer patient.
Up to 50 percent of stem cell transplant procedures, such as bone marrow transplants, result in life-threatening rejection disease, known as Graft-versus-Host-Disease (GvHD). Cellects ApoGraft technology is aiming to turn stem cell transplants into a simple, safe and cost effective process, reducing the associated severe side effects, such as rejection and many other risks.
Dr. Shai Yarkoni, Cellects CEO said, After 15 years of research, this is the first time we have used our technology on a cancer patient suffering from life-threatening conditions. It is a first good step on a road that we hope will lead to stem cell based regenerative medicine becoming a safe commodity treatment at every hospital in the world.
Based on the successful transplantation results, the independent Data and Safety Monitoring Board (DSMB) approved the enrollment of 2 additional patients for ApoGraft treatment to complete the first study cohort as planned.
About GvHD
Despite improved prophylactic regimens, acute GvHD disease still occurs in 25% to 50% of recipients of allogeneic stem cell transplantation. The incidence of GvHD in recipients of allogeneic stem cells transplantation is increasing due to the increased number of allogeneic transplantations survivors, older recipient age, use of alternative donor grafts and use of peripheral blood stem cells. GvHD accounts for 15% of deaths after allogeneic stem cell transplantation and is considered the leading cause of non-relapse mortality after allogeneic stem cell transplantation.
About ApoGraft01 study
The ApoGraft01 study (Clinicaltrails.gov identifier: NCT02828878), is an open label, staggered four-cohort, Phase I/II, safety and proof-of-concept study of ApoGraft process in the prevention of acute GvHD. The study, which will enroll 12 patients, aims to evaluate the safety, tolerability and efficacy of the ApoGraft process in patients suffering from hematological malignancies undergoing allogeneic stem cell transplantation from a matched related donor.
About Cellect Biotechnology Ltd.
Cellect Biotechnology is traded on both the NASDAQ and Tel Aviv Stock Exchange (NASDAQ:APOP)(NASDAQ:APOPW)(TASE:APOP). The Company has developed a breakthrough technology for the isolation of stem cells from any given tissue that aims to improve a variety of stem cell applications.
The Companys technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cell related treatments and procedures. Cellects technology is applicable to a wide variety of stem cell related treatments in regenerative medicine and that current clinical trials are aimed at the cancer treatment of bone marrow transplantations.
Forward Looking Statements This press release contains forward-looking statements about the Companys expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as believe, expect, intend, plan, may, should, could, might, seek, target, will, project, forecast, continue or anticipate or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss Cellects aim to make its ApoGraft technology a game changer in stem cell transplantations for cancer treatments and procedures, Cellects Apograft technology aiming to turn stem cell transplants into a simple, safe and cost effective process, reducing the associated severe side effects, such as rejection and many other risks, Cellects hope that stem cell based regenerative medicine will become a safe commodity treatment at every hospital in the world and that Cellects technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cell related treatments and procedures. These forward-looking statements and their implications are based on the current expectations of the management of the Company only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from procedures, scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications, which could cause the actual results or performance of the Company to differ materially from those contemplated in such forward-looking statements. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading Risk Factors in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2016 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov and in the Companys period filings with the SEC and the Tel-Aviv Stock Exchange.
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Cellect Announces Successful First Cancer Patient Stem Cell Transplant - GlobeNewswire (press release)
Pioneering stem cell gene therapy cures infants with bubble baby disease – UCLA Newsroom
By daniellenierenberg
FINDINGS
UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.
In a phase 2 clinical trial led by Dr. Donald Kohn of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Researchat UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohns treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the childs own cells, has cured 30 out of 30 babies during the course of several clinical trials.
Adenosine deaminase-deficient severe combined immunodeficiency, also known as ADA-SCID or bubble baby disease, is caused by a genetic mutation that results in the lack of the adenosine deaminase enzyme, which is an important component of the immune system. Without the enzyme, immune cells are not able to fight infections. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.
Currently, there are two commonly used treatment options for children with ADA-SCID. They can be injected twice a week with the adenosine deaminase enzyme a lifelong process that is very expensive and often does not return the immune system to optimal levels. Some children can receive a bone marrow transplant from a matched donor, such as a sibling, but bone marrow matches are rare and can result in the recipients body rejecting the transplanted cells.
The researchers used a strategy that corrects the ADA-SCID mutation by genetically modifying each patients own blood-forming stem cells, which can create all blood cell types. In the trial, blood stem cells removed from each childs bone marrow were corrected in the lab through insertion of the gene responsible for making the adenosine deaminase enzyme. Each child then received a transplant of their own corrected blood stem cells.
The clinical trial ran from 2009 to 2012 and treated 10 children with ADA-SCID and no available matched bone marrow donor. Three children were treated at the National Institutes of Health and seven were treated at UCLA. No children in the trial experienced complications from the treatment. Nine out of ten were babies and they all now have good immune system function and no longer need to be isolated. They are able to live normal lives, play outside, go to school, receive immunizations and, most importantly, heal from common sicknesses such as the cold or an ear infection. The teenager, who was not cured, continues to receive enzyme therapy.
The fact that the nine babies were cured and the teenager was not indicates that the gene therapy for ADA-SCID works best in the youngest patients, before their bodies lose the ability to restore the immune system.
The next step is to seek approval from the Food and Drug Administration for the gene therapy in the hopes that all children with ADA-SCID will be able to benefit from the treatment. Kohn and colleagues have also adapted the stem cell gene therapy approach to treat sickle cell disease and X-linked chronic granulomatous disease, an immunodeficiency disorder commonly referred to as X-linked CGD. Clinical trials providing stem cell gene therapy treatments for both diseases are currently ongoing.
Kohn is a professor of pediatrics and microbiology, immunology and molecular genetics at the David Geffen School of Medicine at UCLA and member of the UCLAChildrens Discovery and Innovation Institute at Mattel Childrens Hospital. The first author of the study is Kit Shaw, director of gene therapy clinical trials at UCLA.
The research was published in the Journal of Clinical Investigation.
The research was funded by grants from the U.S. Food and Drug Administrations Orphan Products Clinical Trials Grants Program (RO1 FD003005), the National Heart, Lung and Blood Institute(PO1 HL73104 and Z01 HG000122), the California Institute for Regenerative Medicine (CL1-00505-1.2 and FA1-00613-1), the UCLA Clinical and Translational Science Institute (UL1RR033176 and UL1TR000124) and the UCLA Broad Stem Cell Research Center.
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Pioneering stem cell gene therapy cures infants with bubble baby disease - UCLA Newsroom
What are mesenchymal stem cells? – Palm Beach Post
By raymumme
In the United States alone, more than 400,000 lumbar discectomies and 500,000 spinal fusions are performed each year for symptoms related to lumbar disc degeneration. The ability to get these to heal without surgery has been a long-term goal of many patients and physicians alike. The Spine Center continues to be on the forefront of treatment options and is proud to offer stem cell therapy treatments for patients as part of our comprehensive non-operative treatment options.
Adult stem cells are divided into different categories. For example, the types of adult stem cells Dr. Theofilos uses to treat musculoskeletal issues are known as mesenchymal stem cells (MSCs). These are multi-potent cells that can differentiate into bone cells, cartilage cells, or fat cells.
The human body has multiple storage sites for stem cells to repair degenerated and injured structures. Dr. Theofilos has found that obtaining stem cells from the hip bone (iliac bone) is easily performed within minutes and, in most cases, is a fairly painless procedure for the patient. The stem cells are obtained from bone marrow; just minutes later, they are used for treatment.
This procedure is done in our office and after the procedure, the syringe of stem cells is taken to the lab and placed in a specialized machine called a centrifuge. The centrifuge spins the bone marrow solution and stem cells are separated from the non-useful cells. Now, the stem cells are ready for the treatment.
For those whom are ideal candidates, this provides great hope with reduction in pain and improved quality of life without the need for major surgery.
Voted as one of Americas Top Surgeons, Charles S. Theofilos, MD, Neurosurgeon and Founder of The Spine Center is a leading provider of the state-of-the-art, most comfortable and effective surgical, minimally invasive and non-surgical treatment options for a full range of cervical and spinal ailments, including stem cell therapy and artificial disc replacement. He was among a field of 20 top neuro and orthopedic surgeons in the U.S. chosen to participate in the groundbreaking Artificial Disc Study, which compared the clinical outcome of disc replacement versus traditional spinal fusion. A widely sought after educator and lecturer, Dr. Theofilos has offices in Palm Beach Gardens and Port St. Lucie. In an effort to maintain and honor the commitment to our patients, we will continue to accept Medicare and Medicare Advantage insurance plans for all new and follow up appointments.
11621 Kew Gardens Ave., Suite 101;Palm Beach Gardens
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What are mesenchymal stem cells? - Palm Beach Post
Not knowing what it means is OK, follow your path – Southernminn.com
By NEVAGiles23
Id like to believe that our lives have meaning, that the day-to-day decisions we make steer our steps down a variety of roads in life. As in the poem by Robert Frost, moving in one direction can leave us wondering about what would have happened had we taken the other road.
I dont believe Frost meant that we regret our choices, simply that new realities unfold as we move forward, and they are different than if we had moved left or right instead. Our decisions affect others as well, unfolding and connecting in ways we can only accept and not really understand.
One of my favorite thoughts is that from each difficulty we encounter, no matter how painful, some good will come of it; our tempering or pruning, if you will. It is our place to trust.
This I know. Eleven years ago, Heidi, the daughter of my dear friend Sue Sands, was diagnosed with Leukemia. Her family held a sign-up for Be The Match, the National Bone Marrow Donor Registry, in hopes of finding a stem cell donor to cure her.
I couldnt attend that evening, but shortly after I had a vivid dream in which I was a bone marrow match for Heidi. I knew I had to get tested, so I found another sign-up, did a cheek swab and entered the registry. I wasnt called to be a match for Heidi. In fact, she underwent treatment and was able to recover without a bone marrow transplant.
There was more to come. In 2009 I turned out to be a perfect match for a little girl who happened to be the same age as my daughter. Its more than just matching a blood type; a person must closely match on human leukocyte antigen. I agreed to donate bone marrow and it was a completely anonymous donation.
Let me give you the simplified version of donating bone marrow and stem cells. There are two ways; blood stem cells and bone marrow. In the former, the donor is given a medicine to boost stem cell formation and then the cells are drawn from their blood. In the latter, the donor undergoes surgery where the stem cells are harvested from the back of their hip bones. The stem cells are given to the recipient in about 48 hours, and if all goes well, they are completely cured of their disease.
People tell me they are amazed I would do this for someone I dont know. How could I not? When I was about 23, my mother died of multiple myeloma bone cancer. A little over a year ago, my stepmother succumbed to pancreatic cancer. I know intimately what it is like to feel helpless when a loved one is sick. I know the sense of loss that remains.
I recovered from the bone marrow donation. Yes I was sore for several weeks, and tired, but it felt good to have done something. Yet, there was more to the story. Mayo Clinic doctors had discovered in my pre-donation blood work that I had elevated calcium levels. It was due to a parathyroid tumor, and it was taking calcium from my bones. It didnt affect my ability to donate, but they advised removal of the tumor soon after.
Had I not been a donor, I might never have known about the parathyroid issue until it had severely affected my health. It still gives me shivers to think about it.
Fast forward to this past December. I was working on page layout one afternoon when I received a call from Be the Match. I was a potential match for another patient, this time an adult woman with Leukemia. I am at the top of the preferred age group, but I was the perfect match to potentially save this persons life.
Now, back to the idea of paths intermingling. My friends daughter Heidi, who had beat her leukemia, was dealt a tough blow. A few weeks ago she was diagnosed once again with leukemia, not the same type, but different. It was like lightning striking twice.
My mind instantly went back to my initial dream. What can this mean, that I am called to donate twice, defying so many odds, at the same time she is diagnosed again, against so many odds?
Last week I completed the second bone marrow donation for an unknown recipient. The doctors and nurses on the ninth floor of the Charleton Building at Mayo Clinic were amazing. They took such good care of me and thanked me repeatedly. The procedure went well and my APG employers have been gracious to give me time off for the whole thing.
I am oh so very tired, but the soreness in my hips is better each day. The need to build up the hemoglobin in my blood has made me extra conscientious of my diet. I am thankful for my good health and also for the friends, who brought food, checked in on me and have provided support through this donation process.
What does it all mean? We need each other. What we can do for another person in desperate need, we should do.
I hope and pray for a full recovery for Heidi. They will look for a bone marrow match for her this time around. Maybe I am a match for her after all? The doctor last week said that I could theoretically donate again once I was 100 percent recovered and if my hemoglobin levels were perfect.
Maybe I am not a match, but through my story, others will join the registry and Heidis match will be found. I guess its OK to not know what it all means. Knowing that something good will come is enough.
Reach Publisher and Editor Terri Lenz at 333-3148, or follow her on Twitter.com @KenyonLeader
Reach Publisher and Editor Terri Lenz at 333-3148, or follow her on Twitter.com @KenyonLeader
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Not knowing what it means is OK, follow your path - Southernminn.com
Protein Found in Young Blood Could Be The Key To Fight Aging – Wall Street Pit
By NEVAGiles23
As people age, so does their blood. The question is: what exactly is in the blood of older people that makes it age? And in the same light, what is in the blood of younger people that can help rejuvenate old blood?
The idea of using young blood to rejuvenate old blood was not an automatic conclusion, of course. While it does seem logical, it remained a theory until tests that involved conjoining (i.e. stitching together) of old and young mice for the purpose of swapping blood revealed that the concept did have merit. With shared blood, the health of younger mice deteriorated while the health of older mice improved.
Another kind of experiment done was non-invasive blood swapping using tubes. The results were similar, though different explanations emerged for the change in health conditions of both old and young mice. When conjoined, the mice shared more than just blood; their organs got affected too. In non-invasive blood swapping, the old blood got diluted.
While these experiments were done on mice, theres a chance they might work in people as well. However, this involves blood donation from young people, which might mean the supply will be limited when it comes to fulfill demand.
As an alternative, a research team at Germanys University of Ulm led by Hartmut Geiger turned to stem cells, specifically, what are being referred to as mother stem cells those stem cells in the bone marrow that produce red and white blood cells, and whose number become fewer and fewer as a person ages. With fewer of these cell-generating cells, older people become more susceptible to conditions like anemia and heart disease. They become less capable of fighting infection as well.
By examining mice bone marrow, Geigers team discovered that older mice have considerably lower levels of a protein known as osteopontin. To check the effect of this protein on blood stem cells, they injected stem cells into mice that had low levels of osteopontin. What happened was, the cells aged much quicker.
However, when they mixed older stem cells with osteopontin and a protein that activates osteopontin, the old stem cells started producing white blood cells as if they were young stem cells. This suggests that osteopontin might indeed have a hand in rejuvenating old stem cells and making them behave as if they were young again.
While majority of blood rejuvenation efforts focus on the liquid part of blood (or plasma), Geiger believes blood cells might also play a vital role since cells can move better in the bodys tissues.
Following the initial results of their experiments, the team is now working on developing a drug that contains osteopontin and its corresponding protein activator. The hope is that this drug can promote youthful behavior in blood stem cells and boost the number of mother stem cells. Ultimately, this can help in the treatment of age-related blood disorders, and possibly boost the immune system of the elderly too so they dont get sick as easily.
Details of the study have been reported in The EMBO Journal.
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Protein Found in Young Blood Could Be The Key To Fight Aging - Wall Street Pit
Cellect Succeeds In First Stem Cell Transplant (APOP) – Investopedia
By daniellenierenberg
Investopedia | Cellect Succeeds In First Stem Cell Transplant (APOP) Investopedia It includes more than half the stem cell transplant procedures, including bone marrow transplant, resulting in a serious rejection disease called Graft-versus-Host-Disease (GvHD). GvHD is a medical disorder which results from receipt of transplanted ... Cellect Announces Successful First Cancer Patient Stem Cell Transplant Why Cellect Biotechnology Ltd. (APOP) Stock Is Soaring Today |
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Cellect Succeeds In First Stem Cell Transplant (APOP) - Investopedia
Living Organ Donors: Proposed Tax Deduction Could Help Defray Donation Expenses – Hartford Courant
By JoanneRUSSELL25
The decision to become a living organ donor is a significant one and, among the many factors to weigh, donors should consider potential financial consequences of their altruism.
Medical costs associated with organ, tissue and bone marrow donations don't fall on the donor; they typically are paid by the recipient and often covered by insurance, Medicare or Medicaid.
But other related expenses including travel, lodging and loss of wages due to time out of work are the donor's responsibility.
Most of the roughly 6,000 living donations that occur annually are between relatives and close friends, people who have a vested interest in the recipient's outcome, according to the U.S. Department of Health and Human Services (HHS).
But even people who wouldn't hesitate to help a loved one should know about potential costs.
When Sally McCartin of North Branford donated a kidney to a fellow "hockey mom" in 2013, she knew she'd have to take time off from her job at the state's Department of Revenue Services.
"I was worried about the first two weeks that I would be out of work because I had used up all my sick time getting the necessary testing done to donate," she says. "Initially, I would have gone with no pay for the first two weeks."
The recipient of her kidney offered to cover McCartin's loss of wages, but the expense ultimately was covered by financial donations from McCartin's coworkers and contributions from her union, so she bore none of the cost.
For others, out-of-pocket expenses can be problematic and may deter some from donating altogether, says Sen. Martin Looney, D-New Haven. He proposed legislation in January that would help ease some of the burden.
Under his bill, people who donate organs or bone marrow after Jan. 1, 2017, could deduct up to $10,000 from their income, under the state personal income tax, to cover unreimbursed costs of travel, lodging and lost wages they incur as a result of donating. The bill also aims to allow state employees, beginning in 2018, to take up to 30 days of paid leave from work for organ donation and up to seven days of paid leave for bone marrow donation.
The legislation, co-sponsored by Sen. Catherine Osten, D-Columbia, is before the General Assembly's Public Health Committee and slated to receive a public hearing. It's intended to "limit potential barriers to people agreeing to be a live donor," Looney says.
The matter hits close to home for the senator, who suffered from kidney failure and received a kidney donation from a live donor in December. Nineteen states already have similar laws on the books, Looney says.
Some donors may have some expenses covered by their own insurance, depending on their plan, he says, but not all do.
"In some cases, the out-of-pocket expenses are minimal to the donor, and in other cases they can be substantial," he says, especially when it comes to missed work. "The donor is going to be out of work probably for a minimum of two weeks after the procedure. That would probably be the biggest hardship of all."
Many things can be donated by living donors, according to the HHS, including six vital organs: heart, kidneys, pancreas, lungs, liver and intestines. (In some cases a portion of the organ, such as the liver, is donated. Living heart donations are rare but happen. They occur when a donor has a pulmonary condition that necessitates removal of the heart and lungs for new ones lungs-only operations are considered riskier but the donor's heart is in good enough condition to be transplanted in another patient.)
Donors also may give certain tissues including skin, cornea and blood vessels as well as bone marrow, stem cells and umbilical cord blood.
April has been dubbed "National Donate Life Month," an effort by nonprofit advocacy group Donate Life America to encourage people to register as organ, eye and tissue donors.
About 20 to 25 living donations occur annually at the Hartford Hospital Transplant Program, according to registered nurse and Living Donor Transplant Coordinator Kari Rancourt. Most of them are kidney transplants.
"We do talk a little [with prospective donors] about out-of-pocket expenses," she says. Some donors get help covering costs through social fundraising platforms like GoFundMe, she says.
There also are foundations that offer grants to help would-be donors afford travel and lodging, she adds, which have "helped limit barriers to donations."
McCartin, who donated at Yale-New Haven Hospital, says that she did have to consider the potential expense but that wouldn't have stopped her from donating. She is so passionate about it, she recently began Kid-U-Not, a Connecticut chapter of the American Living Organ Donor Fund that raises funds to assist donors.
"I felt that, if I was healthy enough to donate, then it was a no-brainer," she says. "How could I not help a single mom of three children? I would hope that if I was ever in the same situation someone would step up for me."
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Living Organ Donors: Proposed Tax Deduction Could Help Defray Donation Expenses - Hartford Courant
Lungs Play Previously Unknown Role in Blood Production – Sci-News.com
By Dr. Matthew Watson
Using video microscopy in a living mouse lung, a team of researchers at the Universities of California, San Francisco (UCSF) & Los Angeles (UCLA), has revealed that the lungs play a previously unrecognized role in blood production.
Visualization of resident megakaryocytes in the lungs. Image credit: Emma Lefranais et al, doi: 10.1038/nature21706.
The team, headed by UCSF Professor Mark R. Looney, found that the lungs produced more than half of the platelets blood components required for the clotting that stanches bleeding in the mouse circulation.
In another finding, the team also identified a previously unknown pool of blood stem cells capable of restoring blood production when the stem cells of the bone marrow, previously thought to be the principal site of blood production, are depleted.
This finding definitely suggests a more sophisticated view of the lungs that theyre not just for respiration but also a key partner in formation of crucial aspects of the blood, Prof. Looney said.
What weve observed here in mice strongly suggests the lung may play a key role in blood formation in humans as well.
The study was made possible by a refinement of a technique known as two-photon intravital imaging.
The authors were using this technique to examine interactions between the immune system and circulating platelets in the lungs, using a mouse strain engineered so that platelets emit bright green fluorescence, when they noticed a surprisingly large population of platelet-producing cells called megakaryocytes in the lung vasculature.
When we discovered this massive population of megakaryocytes that appeared to be living in the lung, we realized we had to follow this up, said team member Dr. Emma Lefranais, from the UCSF Department of Medicine.
More detailed imaging sessions soon revealed megakaryocytes in the act of producing more than 10 million platelets per hour within the lung vasculature, suggesting that more than half of a mouses total platelet production occurs in the lung, not the bone marrow, as researchers had long presumed.
Video microscopy experiments also revealed a wide variety of previously overlooked megakaryocyte progenitor cells and blood stem cells sitting quietly outside the lung vasculature estimated at 1 million per mouse lung.
Proposed schema of lung involvement in platelet biogenesis. The role of the lungs in platelet biogenesis is twofold and occurs in two different compartments: (a) platelet production in the lung vasculature; after being released from the bone marrow or the spleen, proplatelets (a1) and megakaryocytes (a2) are retained in the lung vasculature, the first capillary bed encountered by any cell leaving the bone marrow, where proplatelet formation and extension and final platelet release are observed; (b) mature and immature megakaryocytes along with hematopoietic progenitors are found in the lung interstitium; in thrombocytopenic environments, hematopoietic progenitors from the lung migrate and restore bone marrow hematopoietic deficiencies. Image credit: Emma Lefranais et al, doi: 10.1038/nature21706.
The discovery of megakaryocytes and blood stem cells in the lung raised questions about how these cells move back and forth between the lung and bone marrow.
To address these questions, Prof. Looney, Dr. Lefranais and their colleagues conducted a clever set of lung transplant studies.
First, they transplanted lungs from normal donor mice into recipient mice with fluorescent megakaryocytes, and found that fluorescent megakaryocytes from the recipient mice soon began turning up in the lung vasculature.
This suggested that the platelet-producing megakaryocytes in the lung originate in the bone marrow.
In another experiment, the team transplanted lungs with fluorescent megakaryocyte progenitor cells into mutant mice with low platelet counts.
The transplants produced a large burst of fluorescent platelets that quickly restored normal levels, an effect that persisted over several months of observation much longer than the lifespan of individual megakaryocytes or platelets.
This indicated that resident megakaryocyte progenitor cells in the transplanted lungs had become activated by the recipient mouses low platelet counts and had produced healthy new megakaryocyte cells to restore proper platelet production.
Finally, the researchers transplanted healthy lungs in which all cells were fluorescently tagged into mutant mice whose bone marrow lacked normal blood stem cells.
Analysis of the bone marrow of recipient mice showed that fluorescent cells originating from the transplanted lungs soon traveled to the damaged bone marrow and contributed to the production not just of platelets, but of a wide variety of blood cells, including immune cells such as neutrophils, B cells and T cells.
These experiments suggest that the lungs play host to a wide variety of blood progenitor cells and stem cells capable of restocking damaged bone marrow and restoring production of many components of the blood.
To our knowledge this is the first description of blood progenitors resident in the lung, and it raises a lot of questions with clinical relevance for the millions of people who suffer from thrombocytopenia, Prof. Looney said.
The findings were published online March 22, 2017 in the journal Nature.
_____
Emma Lefranais et al. The lung is a site of platelet biogenesis and a reservoir for haematopoietic progenitors. Nature, published online March 22, 2017; doi: 10.1038/nature21706
This article is based on text provided by the University of California, San Francisco.
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Lungs Play Previously Unknown Role in Blood Production - Sci-News.com
How Big Data is Being Mobilized in the Fight Against Leukemia – Drug Discovery & Development
By JoanneRUSSELL25
Healthy cell function relies on well orchestrated gene activity. Via a fantastically complex network of interactions, around 30,000 genes cooperate to maintain this delicate balance in each of the37.2 trillion cellsin the human body.
Broadly speaking, cancer is a disruption of this balance by genetic changes, or mutations. Mutations can trigger over-activation of genes that normally instruct cells to divide, or inactivation of genes that suppress the development of cancer. When a mutated cell divides, it passes the mutation down to its daughter cells. This leads to the accumulation of non-functioning, abnormal cells that we recognise as cancer.
Our laboratoryis focused on understanding how one particular cancer chronic myeloid leukaemiaor CML works. Each year more than 700 patients in the UK andover 100,000worldwide are diagnosed with CML. After recent advances,almost 90%of patients under the age of 65 now survive for more than five years.
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But in the vast majority of patients CML is currently incurable and lifelong treatment means that patients must live with side effects and the chance of drug resistance arising. With increasing numbers of CML patients surviving (and treatment costing between 40,000 and 70,000 per patient a year), increasing strain is being placed on health services.
A single mutation
CML is perhaps unique in cancers in that a single mutation, namedBCR-ABL, underlies the disease biology. This mutation originates in a singleleukaemic stem cell, but is then propagated throughout the blood and bone marrow as leukaemia cells take over and block the healthy process of blood production. The presence of BCR-ABL affects the activity of thousands of genes, in turn preventing these cells from fulfilling their normal function as blood cells.
Drugsthat specifically neutralise the aberrant effects of this mutation were introduced to the clinic from the early 2000s. These drugs have revolutionised CML patient care. Many are now able to live relatively normal lives with their leukaemia under good control.
But while these drugs kill the more mature daughter cells of the originally mutated leukaemia stem cell, they have not fully lived up to their initial billing as magic bullets in the fight against cancer. This is because the original seed population of leukaemic stem cellsevade therapy,lying dormant in the bone marrowto stimulate new cancer growth when treatment is withdrawn.
To truly cure CML we must expose, understand the inner workings of, and uproot the leukaemia stem cells. And to do this, we need to learn more about them. How do they survive the treatment that so readily kills their more mature counterparts? Which overactive or inactivated genes protect them?
We believe that the answers to these questions lie in the analysis of biological big data. Genome-scale technologies now allow scientists to measure the activity (or expression) of every gene in the genome simultaneously, in any given population of cells, or even at the level of a single cell. Comparison of expression data generated from leukaemia stem cells with the same data generated from healthy blood stem cells will reveal single genes or networks of genes potentially targetable in the fight against leukaemia.
Big data to the rescue
In a project funded by Bloodwise and the Scottish Cancer Foundation, we have createdLEUKomics. This online data portal brings together a wealth of CML gene expression data from specialised laboratories across the globe, including our own at the University of Glasgow.
Our intention is to eliminate the bottleneck surrounding big data analysis in CML. Each dataset is subjected to manual quality checks, and all the necessarycomputational processingto extract information on gene expression. This enables immediate access to and interpretation of data that previously would not have been easily accessible to academics or clinicians without training in specialised computational approaches.
Consolidating these data into a single resource also allows large-scale, computationally-intensive research efforts by bioinformaticians (specialists in the analysis of big data in biology). From a computational perspective, the fact that CML is caused by a single mutation makes it an attractive disease model for cancer stem cells. However, existing datasets tend to have small sample numbers, which can limit their potential.
The more samples available, the higher the power to detect subtle changes that may be crucial to the biology of the cancer stem cells. By bringing all the globally available CML datasets together, we have significantly increased the sample size, from two to six per dataset to more than 100 altogether. This offers an unprecedented opportunity to analyse gene expression data to expose underlying mechanisms of this disease.
As of March 2017, theportalis up and running in the public domain. We are planning to tour Scotland and present at international conferences, aiming to train researchers in how best to exploit this new resource. Ultimately, we hope that this tool will lead to new ideas and approaches, and attract more funding, in the fight against CML. And while we continue to expand our representation of CML data in real time from research centres all over the world, we also plan to begin incorporating data from other types of leukaemia.
In recent years, targeted therapies have becomehugely importantin cancer research. By providing these data to the CML research community withinLEUKomics, we hope to mobilise new research into cancer-causing leukaemic stem cells, and ultimately design treatments to target them without affecting healthy cells. Our database provides a critical stepping stone in this process.
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How Big Data is Being Mobilized in the Fight Against Leukemia - Drug Discovery & Development
Stem cell treatments can go wrong – Jamaica Observer
By NEVAGiles23
Stem cells are the foundation of all our body cells before they differentiate to become specialised cells that grow into our tissues and organs, such as kidney cells, muscle cells, nerve cells, and so on.
They commonly come from two sources: The embryo (embryonic stem cells formed in early development after the human egg is fertilised by a sperm); and adult tissue (adult stem cells, such as those existing in bone marrow to later differentiate to form red blood cells, white blood cells and other components of the blood).
The use of human embryonic stems cells for treatment or research is often frowned upon by some people, as they regard the human embryo as a person that should not be discarded after such endeavours. Consequently, much scientific work has recently been focused on the use of adult stem cells.
THE USE OF STEM CELLS
Stem cells may be beneficial in treating diseases that are amenable to cell replacement. However, this is still a young science, and belief that a particular treatment helps two or three people does not convince the scientific community or the whole society that the treatment will work for everyone so afflicted.
Scientific proof comes from conducting clinical trials, the international gold standard often involving hundreds of people so afflicted and comparing them with an equivalent number of people not afflicted to determine whether a treatment really works for those who receive it.
Whilst many stem cell research projects are currently being conducted in various centres around the world to determine whether they produce benefits, and what may be the possible risks involved, there are also medical clinics that are using stem cells not in a registered research project, but rather in the actual treatment of affected people.
TREATMENT CAN CAUSE HARM
A recent report in the highly respected New England Journal of Medicine informed that three elderly women in Florida had been blinded by an unproven treatment.
They had signed up for a purported clinical trial in 2015 for which they had to pay US$5,000 each. Before surgery, the vision in their eyes varied from 20/30 to 20/200, but within one week after surgery, they experienced a variety of complications, including vision loss, detached retinas and bleeding into their eyes, resulting in total blindness.
The authors of the article from the Standard University School of Medicine sought to make patients, doctors and the various regulatory agencies aware of the risks of such a minimally regulated, patient-funded research. It stated that some clinics appeal to patients that are desperate for care and who hope that stem cells will be their answer, but as in the case of these women, some of these current enterprises are very dangerous.
At this particular clinic, fat cells were taken from the patients abdomens and processed to obtain stem cells which were then injected into their eyes. The patients reported that the entire process took less than one hour. The patients had both eyes treated at once, even though most doctors would opt for a conservative approach to observe how the first eye responds.
THE NEED FOR THE REGULATION OF RESEARCH
The article stated that while there is a lot of well-founded evidence for the positive potential of stem cell treatment for many human diseases, such treatments should be conducted in a well-designed clinical trial based on pre-clinical research.
The treatment done for the women lacked nearly all the components of a properly designed clinical trial, including a hypothesis based on laboratory experiments, the involvement of a control group of people and a treatment group, the safe collection of data, the masking of clinical and patient groups, and plans for follow-up.
Clinics offering stem cell treatments exist in Jamaica, The Bahamas and Cuba. However, while both The Bahamas and Cuba have developed regulations that stipulate in law the conditions to be met for stem cell treatments and research within their jurisdictions, Jamaica has developed no such regulation.
THE MEDICAL ACT DOES NOT PROVIDE PROTECTION
The Medical Act of Jamaica was passed in 1976, but does not mention or provide any guidance or protection regarding research with human participants.
Its focus was to: Register medical practitioners; appoint examiners to conduct exams for people applying for registration, and ensure the maintenance of proper professional conduct by practitioners.An amendment in 2004 added the requirement of continuing medical education for practitioners.
Guyana and St Lucia are the only countries in the Caribbean that have joined the progressive countries who all have legislation governing research with human participants within their borders. Regulations should stipulate the requisite conditions, including that treatment and research be monitored by an appropriate ethics committee to meet all international standards.
Without this, vulnerable people seeking health benefits will unknowingly continue to subject themselves to risks of harm without the protection that proper regulations can provide.
Derrick Aarons MD, PhD is a consultant bioethicist/family physician, a specialist in ethical issues in medicine, the life sciences and research, and is the Ethicist at the Caribbean Public Health Agency (CARPHA). (The views expressed here are not written on behalf of CARPHA)
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Stem cell treatments can go wrong - Jamaica Observer
Young blood: Scientists say this protein could be key to reversing aging process – Yahoo News
By NEVAGiles23
The suggestion that young blood may be the key toreversingsome of the negative aspects of aging sounds like the setup to a horror movie. In reality, however, itrefers to some groundbreaking work being carried out byscientists at the University of Ulm in Germany.
Theyve been examining the ways that old blood can be made young again, and they hypothesize that it might help fight some of the effects of aging.To achieve this, theyve discovereda protein capable of boosting blood stem cells, which prompts them to act like the stem cells of younger people.
More: Scientists may have discovered how to reverse the natural aging process
Older stem cells have several disadvantages, Dr. Hartmut Geiger, co-author of a paper describing the work, told Digital Trends. Theyresult in fewer red blood cells, and fewer of an important immune cell that fights infection. They also tend to produce more leukemia, which is a cancer of the blood. All of this is linked, at least in part, to the aging of stem cells. If you have younger stem cells, we think that we can reverse some of these effects.
Older stem cells tend to possess these negative characteristics thanks to the declining number of so-called mother stem cells in our bone marrow as we age. This decline triggers the effects Geiger describes.
In trials with mice, Geiger and his team discovered that older mice have lower levels of a protein called osteopontin. They decided to testthe effect of this protein on blood stem cells.
We took the stem cells from aged mice, incubated them with the osteopontin protein, and then gave them back, he continued.These blood stem cells, which had come from older animals, were found to have a lot of the features of stem cells coming from much younger animals in terms of function.
Its still early days for the research, but the suggestion that osteopontin could make stem cells behave in a more youthful way is certainly promising. The team is now developing a drug made up of osteopontin and a protein for activating it.
Hopefully human trials can follow in the not-too-distant future while were still around to benefit from them.
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Young blood: Scientists say this protein could be key to reversing aging process - Yahoo News
Scientists May Have Found A Way To Make Old Stem Cells Act … – Digital Trends
By NEVAGiles23
Get today's popular DigitalTrends articles in your inbox:
Why it matters to you
Nobody likes getting older. Reversing some of the bad aspects of the process helps us all age gracefully.
The suggestion that young blood may be the key toreversingsome of the negative aspects of aging sounds like the setup to a horror movie. In reality, however, itrefers to some groundbreaking work being carried out byscientists at the University of Ulm in Germany.
Theyve been examining the ways that old blood can be made young again, and they hypothesize that it might help fight some of the effects of aging.To achieve this, theyve discovereda protein capable of boosting blood stem cells, which prompts them to act like the stem cells of younger people.
More: Scientists may have discovered how to reverse the natural aging process
Older stem cells have several disadvantages, Dr. Hartmut Geiger, co-author of a paper describing the work, told Digital Trends. Theyresult in fewer red blood cells, and fewer of an important immune cell that fights infection. They also tend to produce more leukemia, which is a cancer of the blood. All of this is linked, at least in part, to the aging of stem cells. If you have younger stem cells, we think that we can reverse some of these effects.
Older stem cells tend to possess these negative characteristics thanks to the declining number of so-called mother stem cells in our bone marrow as we age. This decline triggers the effects Geiger describes.
In trials with mice, Geiger and his team discovered that older mice have lower levels of a protein called osteopontin. They decided to testthe effect of this protein on blood stem cells.
We took the stem cells from aged mice, incubated them with the osteopontin protein, and then gave them back, he continued.These blood stem cells, which had come from older animals, were found to have a lot of the features of stem cells coming from much younger animals in terms of function.
Its still early days for the research, but the suggestion that osteopontin could make stem cells behave in a more youthful way is certainly promising. The team is now developing a drug made up of osteopontin and a protein for activating it.
Hopefully human trials can follow in the not-too-distant future while were still around to benefit from them.
Follow this link:
Scientists May Have Found A Way To Make Old Stem Cells Act ... - Digital Trends
Swedish scientists successfully implant 3D-printed human cartilage cells in baby mice – TechCrunch
By Sykes24Tracey
In what could potentially serve as an importantmoment in the quest to 3D-print body parts, a team of scientists from Swedens Sahlgrenska Academy and Chalmers University of Technology have managed to successfully implant human cartilage cells in six-week-old baby mice.
The researchers created a gel composed of human cartilage cells, printed it through a CELLINK3D bioprinter and implanted the material inside the lab mice. Once implanted, the tissue began to grow and proliferate inside the animal, eventually vascularizing, with blood vessels growing inside the implanted material. After two months, the material began to more closely resemble human cartilage, which was further stimulated with the addition of stem cells.
The team worked with local plastic surgeons to implant the material, which could one day be used to create more natural implants for patients who have lost ears, noses or knees due to accidents or diseases like cancer.
There is no solution for missing ears, research lead professor Paul Gatenholm tells TechCrunch. You have plastic and silicone implants, which you need to put in with a titanium screw. Thats it. The surgical procedure is that you put in cartilage from a rib from the patient and carve. Its very painful and the outcome is very bad. If we can use cells from the nose and beef it up with stem cells from the patients bone marrow or fat, we will be able to print that full 3D structure.
Gatenholm adds, hopefully, It will have a great impact on healthcare for tissue regeneration and implementation. I think the first breakthrough will be of the skin and then the cartilage and then the bone. Beyond that, the technology could some day also be used for even more complex human parts like organs.
Gatenholm cites a paper from February of last yearin his discussion. In it, a team of researchers, including regenerative medicine professor Anthony Atala, detail how theyve used 3D bioprinting to construct bone and muscle using computer imaging to translat[e] the model into a program that controls the motions of the printer nozzles, which dispense cells to discrete locations.
Similarly, 3D modeling in the form of CAD (computer-aided design) files can be used by the printer to create better anatomical pieces than plastic surgery modeling.
The process is still a ways from becoming a viable reality for future reconstructive surgery, including all of the regulatory approvals that come with implementing of this sort of invasive procedure. But the teams work marks a promising step in the process, which could some day extend beyond cartilage to other key human tissue.
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Swedish scientists successfully implant 3D-printed human cartilage cells in baby mice - TechCrunch
Injection with own stem cells alleviates chest pains, angina, study finds – Genetic Literacy Project
By Dr. Matthew Watson
A non-surgical treatment that uses a patients own bone marrow stem cells to treat chest pain or angina improved both symptoms and the length of time treated patients could be physically active, according to recent research.
We injected a catalyst molecule that caused bone marrow stem cells to enter the patients blood, then harvested them to re-inject into the patient,said Hadyanto Lim, Ph.D., study senior author.
Thirty minutes after the cell separation procedure finished, the collected stem cells were injected back into the patient through an IV.
Four weeks after receiving the treatment, patients experienced significantly fewer angina-related symptoms, and they were able to exercise at a higher intensity and for a longer period of time.
The studys limitations are the small number of patients and absence of a control group. Because no control group was used, the placebo effect cannot be ruled out, Lim noted.
Although this treatment is currently used to treat some cancers multiple myeloma and lymphoma it will need more investigation before it can be made available to the general public to treat angina, according to Lim.
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Injection with own stem cells alleviates chest pains, angina, study finds - Genetic Literacy Project