Girl, four, saves baby brother’s life by donating her stem cells on his 1st birthday – The Mirror

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Brave Aubrey Austin, four, donated her own stem cells and saved her baby brother Carey's life on the day he turned one, after he was diagnosed with a rare type of blood cancer aged just eight months

Image: Supplied via Lucy Laing)

A brave little girl saved the life of her baby brother on his first birthday.

Carey Austin was diagnosed with a rare type of blood cancer when he was just eight months old.

His only hope of survival was a stem-cell transplant.

Against all odds, his sister Aubrey, four, was a perfect match.

Surgeons operated on Careys first birthday and six months later he is cancer-free thanks to his big sister.

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Their mum Naomi said: She absolutely adores Carey and when we explained to her about the transplant she wanted to do everything she could to save him.

Shes only four years old, yet she was only thinking of how she could help him. We felt so guilty putting her through an operation too, but it was Careys only chance of survival.

"She was so brave about it. She knew that her blood was going to save him.

During a two-hour procedure at Great Ormond Street Hospital, London, surgeons took out Aubreys stem cells and they were put into Careys body via a drip.

Naomi said: The fact that the transplant took place on Careys birthday was so significant that she was giving him a second chance at life on that special day.

The doctors and nurses said they had never seen anyone have a stem cell transplant on their birthday before.

Aubrey was very groggy and woozy when she came around from the operation, and she had puncture wounds on her back from where the stem cells had been taken out.

But she was still smiling through it all. She was so brave. She never complained about being in pain and she was just pleased to see how her little brother was afterwards.

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When the brother and sister saw each other for the first time after the operation, there was not a dry eye in the room.

Naomi said: It was so sweet when they were reunited.

We took Aubrey to see Carey and she gave him a cuddle. They were thrilled to see each other again.

After a two-day hospital stay for Aubrey and seven weeks for Carey, the family were able to settle back into life back home in Brighton, East Sussex.

Carey is now in remission, with no signs of the cancer cells in his body.

But his parents have been warned that the disease is so aggressive that until March next year there is a 40% chance of it returning. After that, the likelihood falls to just 5%.

Naomi added: Two other children lost their lives on the cancer ward while we were there, so we know how lucky Carey has been.

He and Aubrey have always been close but now their bond is stronger than ever.

"Shes a superstar and he couldnt have wanted anything more from a big sister. Hes doing so well now. He loves playing with his cars and hes just learning to walk too.

Aubrey is with him all the time she just adores him. She knows that she has saved his life and she loves being a big sister to him. They play cars together and hes learning to walk, so she stands with him encouraging him to take his steps.

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Carey fell ill last November but Naomi, a paediatric audiologist, and her husband Simon, a CPS lawyer, both 43, thought it was bronchitis because his sister had recently had the same thing.

A GP agreed but two days later he was rushed to hospital by ambulance with breathing difficulties.

Doctors at Great Ormond Street diagnosed juvenile myelomonocytic leukaemia, or JMML, which cannot be treated with chemotherapy. There are only 1.2 cases per million children in the UK each year.

Naomi said: I was hysterical. I kept trying to tell them that it wasnt cancer, it was bronchilitis. I couldnt accept what was happening.

Because parents are not suitable donors, Aubreys bone marrow was tested, a process that involves drawing a sample out using a needle.

Naomi said: There is only a 25% chance of any sibling being a match, so even with Aubrey we knew that the odds werent in our favour.

"If she hadnt been a match then we would have had to wait until doctors found an anonymous donor, but that may not have happened in time for Carey.

When the results came back to say that she was a perfect match for him, we couldnt believe it. We had been praying that she would save him, so to get the news that she was a match for him was just incredible.

When we heard I couldnt stop crying, it was so emotional. To think that Carey was going to have a chance of survival thanks to his big sister was the answer to our prayers.

The mum added: We did feel guilty about putting her through the procedure, but when we spoke to her about it, all she wanted to do was help. We were so proud of her.

The transplant was made even more special as it took place on March 15, which was Careys first birthday, giving the family a double celebration.

They are keen to raise awareness of the cancer symptoms and the charity Childhood Cancer and Leukaemia Group, which has helped them throughout their ordeal.

Naomi said: Having a child with cancer is one of the worst things that can happen to you. We didnt realise that it was leukaemia so we are thankful that it was spotted in time.

We received amazing support throughout from the hospital and from the CCLG.

We feel so lucky that Carey has come through it and it feels like a miracle to have him with us now.

Geoff Shenton, a childrens cancer specialist at Newcastle Upon Tyne Hospitals NHS Foundation, said: In a very small proportion of cases JMML can disappear on its own, but this is rare.

Most children will need a bone-marrow or stem-cell transplant. There is still a significant chance that the disease can relapse. There may be a possibility of a second transplant if this happens, but despite our best efforts, children still die from JMML.

For more information and support visit cclg.org.uk

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Girl, four, saves baby brother's life by donating her stem cells on his 1st birthday - The Mirror

September is Blood Cancer Awareness Month: All You Need to Know – News18

September is observed as Blood Cancer Awareness month all over the world. During this month, activists and stakeholders work to raise awareness about the disease and the efforts being taken to fight blood cancers including leukemia, lymphoma, myeloma and Hodgkins disease.

The term blood cancer is a general description of various hematopoietic cancers. Our blood flows through blood vessels to supply all tissues in the body with nutrients. In the approximately 5 litres of blood circulating in our body there are billions of blood cells that carry out various vital functions. All blood cells originate from hematopoietic stem cells.

Haematopoietic stem cells are known as mother cells and do not yet have a specific function. They are able to renew and differentiate into cells with a specific function, thus replacing cells that die. In bone marrow, blood stem cells divide and develop into progenitor cells. Through further division, the progenitor cells mature and transform into different types of blood cells and then enter the bloodstream, says Dr Nitin Agarwal, HOD, Donor Request Management, DKMS BMST Foundation India.

Blood cancer is an abnormal proliferation (abnormal growth) of cells in the bone marrow especially white blood cells (WBCs). Cancer cells flood the bloodstream and drive out healthy cells. As a result, the blood can no longer perform its basic tasks, such as transporting oxygen and protecting the body from infection.

Leukemia This cancer is found in the bone marrow and the bloodstream. It is caused by abnormal rapid production of WBCs and high number of abnormal WBCs which cannot fight against infection, and they impair the bone marrows ability to produce red blood cells and platelets, says Dr Jimmy Mirani, Consultant Onco Surgeon, Wockhardt Hospital, Mumbai Central.

Lymphoma A type of blood cancer which affects the lymphatic system, which removes the risk excess fluids from body and generates immune cells. Lymphocytes are blood cells which are used to fight against infections. These abnormal lymphocytes become lymphoma cells which multiply and get collected in the tissues, adds Dr Mirani.

There are two types of lymphoma, namely, Hodgkins lymphoma and non-Hodgkins lymphoma.

Non-Hodgkins lymphoma: It mainly impacts the B-cell or T-cell. This type of lymphoma occurs more commonly than Hodgkins lymphoma. Can vary clinically and diagnostically into slow-growing ones to very aggressive types, notes Dr. Amrita Chakrabarti, Consultant, Haemato-Oncology & Bone Marrow Transplant, Max Hospital, Shalimar Bagh.

Hodgkins lymphoma This type of lymphoma affects the B cells. Broadly divided into classical Hodgkins and nodular lymphocyte predominant types. Occurs in the adolescence or elderly age group.

MyelomaIt is the cancer of plasma cells; WBCs which produce disease and infection fighting anti-bodies. Myeloma cells prevent the functions and productions of these antibodies leaving a week immune system.

Multiple myeloma This starts in the bone marrow when plasma cells begin to grow uncontrollably. As the cells grow, they compromise the immune system and impair the production and function of white and red blood cells causing bone disease, organ damage and anemia among other conditions, adds Dr Agarwal.

In most cases of blood cancer, the patient feels tired and weak. This happens because the number of red blood cells in the blood starts decreasing due to which there is a lack of blood in the person. Someof the commonsymptoms of blood cancers are fever, severe fatigue, bleeding from gums or skin, back ache, or bone pains, says Dr Pravas Mishra, Head Haematology/ Medical Oncology and BMT, Amrita Hospital, Faridabad.

Patients with myeloma might first present to an orthopaedical with a fracture originating from trivial trauma or to a nephrologist with a kidney dysfunction.Pain in bones and joints can be a symptom of not only arthritis but also blood cancer. Blood cancer is a disease in the bone marrow that is found in large amounts around the bones and joints.

Patientsmight present with nodes in the neck or axilla or groin or swelling in any part of the body. However most often a patientwith blood cancermight present with just a low haemoglobin. It is strongly advised not to ignore any anaemia, warns Dr Mishra.

A person suffering from blood cancer is prone to repeated infections. When leukemia cells develop in the body, then complaints of infection can be seen in the patients mouth, throat, skin, lungs, etc.

People who have cancer tend to have an abnormally low weight. If the body weight is reduced without any obvious cause, then it can be seen as the primary symptom of cancer.

The abnormal formation of leukemia cells in the body prevents the bone marrow from forming healthy blood cells such as platelets. Due to its deficiency, more bleeding problems can be seen from the nose of the patient, during menstruation and gums.

Blood cancer is diagnosed with the help of a wide range of diagnostic methods along clinical evaluation, such as blood tests, bone marrow tests, cytogenetic/karyotyping and molecular analysis, flow cytometry.

Myth: Blood cancer cannot be treated?

Fact: Once a patient is diagnosed with blood cancer, the first concern that comes to ones mind Is blood cancer curable?

Blood cancer is one type of cancer that has a high curability rate especially due to the advancement in the medical field, availability of newer, improved chemotherapy regimens, targeted therapy, and improved infection control measures. Timely diagnosis, especially early diagnosis, increases the chances of cure from blood cancer. Some of the other factors that impact the cure of blood cancer include the age of the patient, physical condition, presence of other comorbidities, stage of the disease, subtype of cancer, molecular factors, whether low grade/high grade, acute or chronic, the body parts that are affected and whether the disease is new onset or has come back after a previous cure.

You must understand that the cure or recovery from cancer is unpredictable, adds Dr. Chakrabarti.

There are cases when the patient has recovered even in the later stages of blood cancer. On the other hand, there are recorded cases where the patient couldnt recover even in the initial stages of blood cancer. So, its important to have realistic expectations and focus on following a healthy lifestyle with the advised treatment and measures. Early diagnosis and treatment play an important role in attaining cure.

Myth: All blood cancer patients need a bone marrow transplant

Fact: No, majority of patients suffering from blood cancers are treated without bone marrow transplant. A combination of chemotherapy, targeted therapy and immunotherapy is the best line of treatment.

Myth: Blood cancer occurs only in children?

Fact: No, blood cancers can occur in all age groups. All have a higher incidence in young children whereas Myeloid Leukaemia (MLL) is more frequently seen in senior citizens.

India is reeling under pressure of many misconceptions that exist amongst people about blood stem cell donation, its process and even its after-effects.

Myth: Once you donate blood stem cells, you will lose them forever.

Fact: Only a fraction of total stem cells is extracted during the process. Also, all the cells are naturally replenished within a few weeks

Myth: Donating stem cells is a really invasive and painful process

Fact: Blood stem cells are collected through peripheral blood stem cell collection (PBSC) which is completely safe and a non-surgical procedure. The process is similar to blood platelet donation that takes approximately three to four hours to complete and the donor can leave the collection center the same day.

Myth: Blood donation and a blood stem cell donation are same

Fact: Unlike blood collection for transfusion, blood stem cells are collected only when there is a match between the donor and patients human leukocyte antigen (HLA) combination (tissue type). So, you could be potentially the only match and life saver for a person with blood cancer in need of a transplant, adds Dr Nitin Agarwal. Blood stem cell donors donate only blood stem cells and the process is similar to a platelet donation.

Myth: Pregnant women cant register

Fact: This is untrue, a woman can register even during her pregnancy.

Myth: Stem cell donation leaves prolonged side-effects

Fact: No, there are no major side effects post blood stem cell donation. A person may only experience minor flu like symptoms because of the GCSF injections given to him/her before the donation, to mobilize blood stem cells in your blood stream.

Myth: Piercing and/or tattoo is a restricting factor

Fact: Piercing or a tattoo doesnt stop you from registering yourself to be a potential donor.

Myth: My blood stem cells can be stored

Fact: Your blood stem cells will not be stored. They last for around 72 hours and are delivered for the recipient straight to the hospital by a special courier. If the recipients body accepts them, the stem cells will start making healthy blood cells.

Myth: Joining a blood stem cell registry is no use. Most patients can find a stem cell donor within their own families

Fact: Per statistics, only 30% of blood disorder patients in need of a stem cell transplant are able to find a sibling match. About 70% of patients need an unrelated donor.

A registry like DKMS BMST Foundation India is a data bank of potential blood stem cell donors that houses details on thousands of committed blood stem cell donors. Any patient can benefit from this registry provided an HLA match.

Some of the blood cancer treatments include the following

Chemotherapy

This is the most important aspect of blood cancer treatment and involves using certain chemicals to kill the cancer-causing cells in the patients body. The prescribed drugs are given in a particular timeframe for the best possible improvement in the patients health. In some patients, a stem cell transplant is provided along with high dose chemotherapy.

Radiation therapy Radiation therapy helps to destroy cancer cells with the help of specific high-energy beams to kill cancer cells in precise areas of the body. This treatment is much beneficial for patients with lymphoma

Bone marrow transplant In this procedure, healthy stem cells are utilized to replace the cells affected by cancer. This helps the patients recover in the best possible manner. Can be autologous (where stem cells are taken from the patients own body) or allogenic (when a healthy donor gives stem cells to the patient.)

Targeted Therapy

Usually in the form of oral medications or pills. They are given alongside chemotherapy/ or radiotherapy and affect specific cancer cells and help in destroying them.

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September is Blood Cancer Awareness Month: All You Need to Know - News18

The New York Stem Cell Foundation Mourns the Loss of CEO Susan L. Solomon – PR Newswire

Dr. Derrick Rossi Named Interim CEO

NEW YORK, Sept. 9, 2022 /PRNewswire/ -- The New York Stem Cell Foundation (NYSCF) today announced the death of its Chief Executive Officer and Co-Founder, Susan L. Solomon, on September 8th, shortly after she had stepped down as CEO, after a long battle with ovarian cancer. Dr. Derrick Rossi, a member of the NYSCF Board of Directors and co-founder of Moderna Therapeutics, has been named Interim CEO of NYSCF.

The New York Stem Cell Foundation today announced the death of its CEO and Co-Founder, Susan L. Solomon

NYSCF is a New York-based non-profit organization that supports stem cell scientists around the world and operates the NYSCF Research Institute, the largest independent stem cell laboratory in the United States. As CEO, Ms. Solomon raised over $400M for stem cell research, helping to catalyze the field and transform the future of medical research.

"This is the end of an incredible era for NYSCF," said Dr. Roy Geronemus, Chairman of the NYSCF Board of Directors. "Susan founded this organization in 2005, and guided it for over 17 years. She imagined the impossible and made it happen. I speak on behalf of the entire Board when I say that we will forever be grateful for all she did for NYSCF and for the field of stem cell research to advance better treatments and cures for patients everywhere. We are confident that Dr. Rossi as Interim CEO, and the rest of the NYSCF team, will continue the trajectory that Susan led us on to move NYSCF's mission forward.The Board has begun a search for a permanent CEO."

A lawyer by training and a longtime entrepreneur and business executive, Ms. Solomon began her role as a health-care advocate in 1992 when one of her sons was diagnosed with type 1 diabetes. After conversations with clinicians and scientists, she identified stem cells as the most promising way to address unmet patient needs and felt an independent organization was needed to help translate cutting-edge stem cell research into clinical breakthroughs and cures for patients. She co-founded NYSCF in 2005. Since then, advances from NYSCF research have twice been named Time magazine's #1 scientific breakthrough of the year, and NYSCF-supported research has led to over 20 major clinical breakthroughs that are already or very soon bringing clinical treatments for devastating diseases. During her time as CEO, Ms. Solomon served on many Boards, including the College Diabetes Network and the Regional Plan Association, and received numerous awards, including the New York State Women of Excellence Award from the Governor of New York, the Triumph Award from the Brooke Ellison Foundation, and recognition as a Living Landmark from the New York Landmarks Conservancy.

During a meeting earlier in the week with NYSCF staff to announce the CEO transition, Ms. Solomon relayed the following message:

"Building NYSCF has been the privilege of a lifetime and I am incredibly proud of the contributions we have made to the field of stem cell research and developing new and more effective treatments and cures to improving the lives of patients. I am confident that our outstanding and dedicated leadership and staff will continue to move our programs forward under Derrick's leadership and that of our longtime COO/CFO Jeff Wallerstein while the Board conducts a search for my successor."

"It has been a great privilege to serve on the NYSCF Board of Directors and I am honored to now serve as Interim CEO," said Dr. Rossi. "Since I first met Susan in 2010 and became a member of the NYSCF community, I have been in awe. Susan was a force of nature, a fierce and effective advocate for science and patients, and a true visionary. She was also a dear friend. Without question, Susan's and NYSCF's impact on science has been enormous and, quite frankly, unmatched.Though I wish that Susan could have continued her incredible and effective leadership of NYSCF for the next hundred years, I am nonetheless honored and ready to lead NYSCF over the coming months as we search for a permanent leader."

Dr. Rossi, a biotechnology entrepreneur and stem cell scientist, is the co-founder of Moderna Therapeutics, and co-founder of Intellia Therapeutics, Magenta Therapeutics, and Stelexis Therapeutics. Until his retirement from academia, he was an Associate Professor at Harvard Medical School and Harvard University, and an investigator at Boston Children's Hospital where he led an academic team working on stem cell biology and regenerative medicine. In 2010, Derrick was named a NYSCF Robertson Stem Cell Investigator and he joined the Board of Directors in 2020. His efforts in the development of cutting-edge technologies and new therapeutic strategies are at the forefront of regenerative medicine and biotechnology. Time magazine named Dr. Rossi as one of the 100 Most Influential People in the world (Time 100) in 2011. Dr. Rossi earned his B.Sc. and M.Sc. from University of Toronto, and his PhD from the University of Helsinki.

Prior to founding NYSCF, Ms. Solomon had a diverse career spanning many decades. After graduating from New York University, she received her JD from Rutgers University School of Law while raising her eldest son as a single mother and serving as an editor of the Law Review. She began her career as an attorney at Debevoise & Plimpton. The work she was most passionate about was her pro bono work, including the representation of a woman suing the NYC Fire Department for sexual discrimination based on the firefighting qualification testing that was biased toward male applicants.

She later continued her law career as counsel for Warner Amex Satellite Entertainment Corporation, a joint venture in the then-new industry of cable television to develop television networks, including MTV, Nickelodeon, and Showtime.

After jobs at United Satellite Entertainment and CBS Productions, a film arm of CBS, Ms. Solomon joined MacAndrews & Forbes to help in the area of media acquisitions, and later APAX, formerly MMG Patricof and Company, another financial firm.

Ms. Solomon subsequently joined Sony Corporation to establish and serve as President of a new radio network, Sony Worldwide Networks, which was the first to do internet radio broadcasting. She then moved on to her last media job as the founding CEO of Sothebys.com, where she helped to develop the first online auction platform.

Prior to founding NYSCF in 2005, she started her own strategic management consulting firm, Solomon Partners LLC, through which she worked with a range of non-profit and media companies.

Ms. Solomon is survived by her husband Paul Goldberger and three sons and daughters-in-law, Adam and Delphine Hirsh, Ben Goldberger and Melissa Rothberg, and Alex Goldberger and Carolyna De Laurentiis, and six grandchildren Thibeaux and Josephine Hirsh, Julian and Gabriel Goldberger, and Arlo and Celeste Goldberger.

About The New York Stem Cell Foundation Research Institute

The New York Stem Cell Foundation (NYSCF) Research Institute is an independent non-profit organization accelerating cures and better treatments for patients through stem cell research. The NYSCF global community includes over 200 researchers at leading institutions worldwide, including the NYSCF Druckenmiller Fellows, the NYSCF Robertson Investigators, the NYSCF Robertson Stem Cell Prize Recipients, and NYSCF Research Institute scientists and engineers. The NYSCF Research Institute is an acknowledged world leader in stem cell research and in the development of pioneering stem cell technologies, including the NYSCF Global Stem Cell Array, which is used to create cell lines for laboratories around the globe. NYSCF focuses on translational research in an accelerator model designed to overcome barriers that slow discovery and replace silos with collaboration.

David McKeon 212-365-7440 [emailprotected]

SOURCE The New York Stem Cell Foundation

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New Hope Against ALS: They Manage To Transplant Stem Cells That Could Prevent Paralysis – Nation World News

An experimental therapy developed by researchers at Cedars-Sinai Center in Los Angeles, California (USA) has shown that stem cell transplant Possible and safe for patients suffering from amyotrophic lateral sclerosis (ALS). The results are published in the peer-reviewed scientific journal nature medicine,

By combining gene and cell therapy, scientists have shown that these cells can cross the blood-brain barrier and release a protective protein from the motor neurons of the spinal cord. This could potentially prevent its worsening and paralysis of muscle functions that characterize this neurodegenerative disease.

Stem cells are a powerful method for Transport of important proteins in the brain or spinal cord which otherwise could not cross the blood-brain barrier, explains Clive Svendsen, MD, director of medicine and biomedical sciences at the Cedars-Sinai Institute for Regenerative Medicine and senior author of the current study.

We have been able to show that these laboratory-modified stem cells can be safely transplanted into the spinal cord. And, after a one-time treatment, these cells survive and Protein production for more than three years What we know protects neurons that die as ALS progresses, Svendson continues.

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These cells were intended maintain leg function In patients, one of the first manifestations of paralysis is associated with amyotrophic lateral sclerosis. Subsequently, the disease automatically leads to the inability to walk, speak, and finally breathe. The trial involved 18 volunteers, and none of them experienced serious side effects.

The modified cells in Svendsons lab were designed to produce GDNF. a protein called, stands for glial cell line-derived neurotrophic factor. These glial cells are affected by ALS and stop protecting the neurons responsible for passing the nerve signals that enable muscle function and movement, causing them to deteriorate.

GDNF alone cannot cross the blood-brain barrier But stem cell transplantation is a method that manages to move the protein to where it should be, explains Dr. Pablo Avalos, co-author of this work. In this first therapeutic approach, the technique was to demonstrate that that it did not cause adverse in patients.

ALS often results in a loss of strength in both legs simultaneously, so researchers applied therapy. only one of them Through the half of the cord that controls it, so that the results can be compared with the other untreated leg. After transplantation, patients were followed for one year. During this period, it was found that the implant did not negatively affect the muscle strength of the operated leg.

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One of the minor side effects, however, manifested itself when the transplanted cells went to higher levels of the marrow, affecting sensory area and causing instances of pain. there were also cases of benign growth, As Svendson points out, these phenomena should be studied in future trials with a deeper focus and a different surgical approach.

The next trial, he explains, will enroll more patients with earlier ALS cases, to verify the long-term effects. This time they will focus on the lower part of the spine. In addition, these cells will be tested Direct implants in the cerebral motor cortex In hopes of better preserving the neurons responsible for the use of the hand.

We are very grateful to the study participants, Svendsson says. ALS is a very difficult disease to treat, and this research a new Hope To get closer to a way to delay the progression of this disease through therapy.

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New Hope Against ALS: They Manage To Transplant Stem Cells That Could Prevent Paralysis - Nation World News

Positive stress can boost tooth regeneration – Tech Explorist

Nowadays, most people face teeth-related issues, expecting to retain their natural teeth or want functional teeth replacement with modern techniques. Modern techniques for missing tooth replacement include complete dentures, cast partial dentures, Implants, and prosthetic crowns. But these are not as equivalent to functional teeth and natural teeth. Recent research is ongoing on boosting tooth regeneration by inducing positive stress.

The study published online in the Journal of Dental Research by Dr. Waruna Dissanayaka, an Assistant Professor in Oral Biosciences, has overcome adaptive mechanisms in tooth stem cells preconditioning stress that boost tooth pulp tissue generation. This positive stress can have good changes in tooth stem cells by making them more resistant to disease and injury. So, in the future, this can help to improve implant cell survival and pulp tissue regeneration.

How positive stress can boost tooth regeneration:

When the tooth is injured or decayed, the vital tissue inside the tooth is exposed to harmful bacteria, which is susceptible to infection. If the tooth pulp is infected, the recent approach is to remove it and fill it with artificial material; when the pulp-less tooth is filled with inert material, the tooth dries, which makes the tooth brittle and more prone to crack and reinfection.

The only way is to replace the tooth with a prosthesis or extraction, but stem cell therapy can help in dental pulp regeneration, as per a recent study. Stem cells of human exfoliated deciduous teeth (SHED) preconditioned to a hypoxic condition by hypoxia-inducible factor 1 (HIF-1) stabilization via knockdown of prolyl hydroxylase domain-containing protein 2 (PHD2) using lentiviral short hairpin RNA. HIF-1 was inserted in Pura Matrix hydrogel, which was injected in the root canal of a human tooth fragment and implanted in immunodeficient mice. Then within 28 days, dental pulp-like tissue formation was seen with a higher level of vascularization, which helps enlist host blood vessels.

Dr. Waruna Disasanayaka and his team focus on developing the lost tooth pulp, which revitalizes the tooth and functions like a normal tooth. The tooth root canal is surrounded by hard dental tissue with less blood supply creating a harsh environment for cells of low oxygen and nutrients. So, the research team develops a preconditioning protocol that helps cells in low oxygen conditions to activate the protein.

Dr. Yuanyuan Han, a co-investigator of the team, pointed out: As this protein was reported to activate several key adaptive mechanisms, we wondered whether this phenomenon can be applied to improve cell survival following transplantation until a sufficient blood supply is achieved.

He also explained, In our study, we found that these cells activate a metabolic mechanism to produce energy under low oxygen conditions and scavenge harmful metabolites produced in stress conditions.

Dr. Dissanayaka says, Interestingly, we also found that preconditioned cells significantly enhanced the dental hard tissue formation within the regenerated pulp tissue. Former research has revealed that our cells possess number of adaptive mechanisms for stress, which are regulated by several key genes encoded in our DNA that are normally inactive.

If we can activate these genes, downstream expression of specific proteins can prime less vulnerable to injury, Dr. Dissanyaka said. He also said, Our aim is to find ways to take advantage of this capacity and use positive stress to help regenerate the dental tissues.

He aims to find new strategies to enhance the therapeutic potential of tooth stem cells.

This stem cell therapy helps to regenerate tooth tissue pulp which can help to retain natural tooth functioning. In the future, it will help to overcome teeth-related problems by avoiding the replacement of teeth by various means.

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Positive stress can boost tooth regeneration - Tech Explorist

Great oaks grow from small acorns: Oncology is committed to doing its part for sustainability – EurekAlert

Late-breaking results presented at the ESMO Congress 2022 elucidate the link between air pollution and lung cancer

ESMO partners with EONS to launch the Cancer Prevention across Europe (PrEvCan) campaign

Study confirms accuracy of multi-cancer early detection blood testing, paving the way for a new era in cancer screening

Paris, France, 9 September 2022 Sustainability will be at the heart of many discussions at the ESMO Congress 2022, as illustrated by the new results and initiatives spanning cancer prevention, early detection and treatment that were announced today during the opening press conference to the annual meeting of the international oncology community taking place 9-13 September in Paris, France.

By definition, sustainability is about being able to maintain important, high-quality processes over time. In oncology, seeing the rise in cancer cases, we need to ask ourselves how we can make sure the essential process of caring for patients can be maintained, said ESMO President Prof. Solange Peters. Sustainability encompasses the notion of avoiding degradation, meaning that we also have to look at maintaining the quality which, in cancer, includes the availability of and access to anticancer drugs. It also includes quality of life, which is still dependent on the environment, and as ESMO we need to start looking at the environmental sustainability of everything we do.

Underscoring the multifaceted nature of sustainability as a societal goal, late-breaking results to be presented at the ESMO Congress 2022 offer a deeper understanding of the long-established link between air pollution and non-small cell lung cancer (NSCLC) arising in people who have never smoked, and make clear the link between climate change and human health. Pollution has a known association with lung cancer, but we didnt know if and how it directly causes the disease, said study author and ESMO 2022 Scientific Co-Chair Prof. Charles Swanton, the Francis Crick Institute and Cancer Research UK Chief Clinician, London, UK, explaining the background to this work. (1)

The research, based on human and laboratory studies, showed for a population of nearly half a million people living in England, South Korea and Taiwan that exposure to increasing concentrations of airborne particulate matter (PM) 2.5 micrometres (m) in diameter was linked to increased risk of NSCLC with mutations in the EGFR gene, which are known to be present in about half of people with lung cancer who have never smoked. In laboratory mouse models, the same pollutant particles (PM2.5) were seen to directly cause lung cancer by acting through lung tissue inflammation, driving the release of a molecule known as interleukin-1 that causes epithelial cells to transdifferentiate into cancer stem-like cells. In the presence of mutations in EGFR and in another gene linked to lung cancer called KRAS, these cells can then bloom into a tumour.

These mutations can be found in over half of normal lung tissue biopsies and are a natural process of ageing. They are necessary, but not sufficient to drive cancer: it is in combination with pollution that the cancer stem cells can expand and initiate a tumour. This begins to explain how environmental carcinogens that dont induce DNA mutations can drive cancer, said Swanton, deriving from this discovery a public health mandate to lower the levels of these pollutants, which are produced by the combustion of fossil fuels. We have to achieve a 50% reduction in greenhouse gas emissions by 2030, and by doing so we will naturally reduce levels of PM2.5. We can all play a part here: we need to cycle more, walk more. Its worth bearing in mind that PM2.5 cause 8 million deaths a year, not just due to cancer but also to other diseases like cardiovascular disease, strokes, dementia that is more than the deaths caused by tobacco globally.

In light of the fact that his research confirmed the blockade of interleukin-1 could inhibit lung cancer initiation by blocking the pollution-induced transformation of airway cells into cancer stem cells, Swanton also suggested that targeting interleukin-1 should be further explored in the future as a potential new approach to cancer prevention.

The findings come in a context where the global incidence of respiratory cancers is on the rise, with annual new cases expected to jump by about 70% over the next two decades. In Europe alone, similar trends observed for other malignancies could result in an increase in overall cancer mortality, from 2 million annual deaths in 2020, to as many as 3 million by 2040. (2) As up to half of all cancers are thought to be preventable, prevention is considered by the World Health Organisation to be the most cost-effective, and thus the most sustainable, long-term strategy for cancer control.

The ESMO Vision 2025, made very clear that if we want to succeed in tackling cancer, we need to develop a clear plan for primary and secondary prevention, continue to offer the optimal care for cancers that cannot be prevented and adequately support cancer survivorship. Focusing on only one of these areas and neglecting the others would lead to failure, said ESMO Director of Public Policy Dr. Rosa Giuliani.

In line with the Societys commitment to promoting research-based cancer prevention, the ESMO representatives joined European Oncology Nursing Society (EONS) Executive Board member Dr. Lena Sharp, Regional Cancer Centre, Stockholm, Sweden, in announcing the launch today of the Cancer Prevention across Europe Campaign (PrEvCan).

Led by EONS with ESMO as key partner alongside other international organisations, the campaign will over a one-year period dedicate each month to promoting and explaining the scientific evidence for each one of the 12 recommendations of the European Code Against Cancer (ECAC) to prevent the disease, starting in October with smoking as one of the most important cancer risk factors.

What is new here is that it is the cancer care workforce leading the way, said Sharp, the PrEvCan project leader. We are the ones who meet patients and their families, so we could intervene on a daily basis supporting and advising people to adopt healthier lifestyles to reduce the risk of new cancers but also to reduce negative effects on the current disease.

The campaign will target the general public, including the most vulnerable groups who can be difficult to reach with health promotion and lifestyle advice, but equally healthcare professionals, who according to Sharp can also take a more prominent role in supporting vaccination and screening programmes.

The ESMO President added: We thought for a while that prevention should be in the hands of family doctors, but then we started to learn that preventing the disease must be at least partly in the hands of the specialists of a specific disease, in order to convince people about its importance. Particularly after COVID, a certain degree of suspicion can happen in medicine. You need to make sure that everything you propose has a basis and one of these bases for cancer prevention is the burden of cancer, what it represents not only in terms of lost years of life but also in terms of the sustainability of our societies and healthcare systems. Peters further highlighted that oncologists should view prevention as an integral part of oncology care, also because the science of prevention still requires more data.

For cancers that are not currently avoidable, screening and early detection has the potential to both maximise individuals chances of survival, and alleviate the burden on health systems by reducing the proportion of patients with advanced disease who require costly, chronic therapies and care.

A study to be presented at this years Congress could lead to a major paradigm shift in this field, having confirmed the feasibility of multi-cancer early detection (MCED) blood testing as a method of screening for up to 50 different cancer types simultaneously. (3)

This is one of the very first studies where the detection of cancer DNA in the blood has allowed us to detect cancer at an early stage, said ESMO 2022 Scientific Co-Chair Prof. Fabrice Andr, Institute Gustave Roussy, Villejuif, France. If this test works, in the future, it will be good news for patients, but most cancer centres are not equipped to scale up surgeries for hundreds more patients with, say, early-stage pancreatic cancer. With this landmark study comes a need for a wake-up call for hospitals to see what will happen in 10 years and start now to train fellows and change their infrastructures accordingly.

Highlighting the time and patience required to turn promising research results into meaningful innovation for patients, Swanton observed: ESMO 2022 is a celebration of the collaboration between basic scientists and healthcare professionals to advance care for our patients. Some of the breakthroughs that will be discussed over the next four days have come from biological studies of worms, yeast, bacteria and plants but they took 30 or 40 years of painstaking science from the bench to the bedside. We need our funders to recognise that and to sustain investment in discovery research to generate the medicines of the future.

Among other highly anticipated results to be presented at the ESMO Congress 2022 , Andr drew attention to several examples of novel approaches which could soon become a reality in the clinic: from the phase III trial of gamma secretase inhibitor (GSI) nirogacestat, a first-in-class drug targeting a new molecular alteration in a rare category of cancers known as desmoid tumours, through a landmark trial of cell therapy using tumour-infiltrating lymphocytes (TILs) to improve the outcomes of patients with advanced melanoma, all the way to several late-phase trials of immunotherapy, including for non-small cell lunger cancer patients not eligible to standard platinum chemotherapy. Andr welcomed the presence of studies for underrepresented patient populations in the Congresss scientific programme, concluding: We cannot exclude patients from clinical trials.

In closing, ESMO 2022 Press Officer Dr. Antonio Passaro called for a wide and wise dissemination of the data to be presented: We have here a community of about 25,000 people, with more than 1,900 abstracts and 76 LBAs that will be presented in the coming days. We need to pass these messages to all of our colleagues and the public in order to dramatically improve the future of our patients, which risks being worse than it is today considering current cancer incidence trends.

-END-

Notes to Editors

Please make sure to use the official name of the meeting in your reports: ESMO Congress 2022

Official Congress Hashtag: #ESMO22

Follow the conversation on Twitter, LinkedIn, Instagram, Facebook and watch video material on YouTube

References

1 LBA1 Mechanism of action and an actionable inflammatory axis for air pollution induced non-small cell lung cancer in never smokers will be

presented by Charles Swanton during Presidential Symposium 1 on Saturday, 10 September, 16:30 to 18:00 CEST in Paris Auditorium. Annals of

Oncology, Volume 33 Supplement 7, September 2022

2 Source: Globocan

3 Abstract 903O A prospective study of a multi-cancer early detection blood test will be presented by Deb Schrag during the proffered paper

session Basic science and translational research on Sunday, 11 September, 16:30 to 18:00 CEST in Orlans Auditorium. Annals of Oncology,

Volume 33 Supplement 7, September 2022

4 The ESMO ANMS 2.0 survey about access to cancer medicines will be discussed during the educational session The Universal Health Coverage (UHC) dilemma: Can we afford to pay for what we want? on Saturday, 10 September, 8:30 to 10:00 CEST in Marseille Auditorium.

About the European Society for Medical Oncology (ESMO)

ESMO is the leading professional organisation for medical oncology. With 25,000 members representing oncology professionals from over 160 countries worldwide, ESMO is the society of reference for oncology education and information.Driven by a shared determination to secure the best possible outcomes for patients, ESMO is committed tostanding by those who care about cancerthroughaddressing the diverse needs of#ONEoncologycommunity,offering#educationforLIFE, andadvocating for#accessiblecancerCARE.www.esmo.org

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Great oaks grow from small acorns: Oncology is committed to doing its part for sustainability - EurekAlert

What happens to the brain on prescription steroids? – Medical News Today

Glucocorticoids also known as corticosteroids or just steroids are a class of medications prescribed for a variety of different diseases and conditions. These are different from anabolic steroids that may be used to increase muscle mass.

Concerningly, prescription steroids can sometimes come with harsh side effects, including neurological issues, such as mood disorders and cognitive issues.

Now a team of scientists from Leiden University Medical Center in The Netherlands has found evidence suggesting the use of prescribed steroids causes structural and volume changes in the white and gray matter of the brain.

This study recently appeared in the journal BMJ Open.

Doctors mainly prescribe corticosteroids to help lower inflammation in the body, suppress the bodys immune system, or balance hormone levels

They normally prescribe them in tablet or inhaler form, although sometimes people require prescribed steroid injections. There are also topical corticosteroids in the form of lotions or creams.

A doctor might prescribe steroids for the following conditions:

Using glucocorticoids for an extended time increases a persons risk of developing certain side effects, such as:

According to doctoral researcher Merel van der Meulen, from the Department of Medicine in the Division of Endocrinology at Leiden University Medical Center and lead author of this study, previous research of people with Cushings disease, who have very high levels of the bodys own glucocorticoid cortisol, shows that long-term exposure to glucocorticoids can affect both the function and the structure of the brain.

Correcting cortisol levels can at least partially reverse these changes. But what about people whose steroid levels increase due to other medical needs?

A few small studies in selected populations also showed that long-term systemic glucocorticoid medication use is associated with some differences in the brain, van der Meulen told Medical News Today.

We wondered whether these effects of glucocorticoids on brain structure could also be observed in the large population-based cohort of the UK Biobank, including inhaled glucocorticoid users, she added.

The research team examined data, including questionnaires and MRI scans, from 222 systemic glucocorticoid users meaning they took the prescribed medication orally or through an injection and 557 inhaled glucocorticoid users from the UK Biobank population recruited between 2006 and 2010.

None of the participants had a history of neurological, psychiatric, or hormonal issues. Researchers compared the data from glucocorticoid users to that of 24,106 people who did not use steroids.

The researchers found that participants using either systemic or inhaled prescribed steroids had less intact white matter structure in the brain compared to non-steroid users. However, this observation increased in systemic steroid users compared to inhaled steroid users.

White matter occurs deep in the brain and is made up of bundles of nerve cells. It plays a role in neuronal connections and signaling in the brain.

The scientists moreover found that participants taking systemic steroids had a larger caudate a part of the gray matter of the brain involved in high-level activities like planning the execution of movements, learning, and memory compared to non-users.

And participants using inhaled glucocorticoids had a smaller amygdala compared to those not taking prescribed steroids. The amygdala is also part of the brains gray matter and is linked to the processing and regulation of emotions.

MNT spoke with Dr. Santosh Kesari, a neurologist at Providence Saint Johns Health Center in Santa Monica, CA, and Regional Medical Director for the Research Clinical Institute of Providence Southern California about this study.

I was excited to know someone did this study that really validates what weve known for a long time that steroids cause brain atrophy and a lot of neuropsychiatric symptoms or side effects, he stated.

This study showed that steroids do have an effect on the structure of the brain, Dr. Kesari continued. You do lose white matter, which [makes up] the connections from one neuron to another. Theres also some loss of the gray matter, the actual neurons, that needs to be studied [further].

Dr. Kesari explained that white matter is the conduit for information from one neuron to another:

When you lose white matter, everything slows down, meaning slower response, some memory issues potentially, or cognitive issues. And then there [are] also psychiatric issues, so they [people who take prescription steroids] can get agitated, depressed, mood disorders, things like that.

Adding to the white matter discussion, van der Meulen said that previous research shows that glucocorticoids can have psychiatric side effects, such as depression and anxiety.

In our observational study, we report associations between glucocorticoids and a lower white matter microstructure in the brain, she continued. It is possible that these associations may be related to the psychiatric side effects of glucocorticoids, but more research is needed to confirm this.

MNT also spoke with Dr. Ilan Danan, a sports neurologist and pain management specialist at the Center for Sports Neurology and Pain Medicine at Cedars-Sinai Kerlan-Jobe Institute in Los Angeles, CA.

He cautioned that it is important to note there is a difference between the prescribed steroids discussed in this study compared to those taken by athletes.

As opposed to the steroids that may be prescribed by physicians, the ones that athletes will look into are going to be more for performance enhancement, he explained. Those are anabolic, androgenic-type steroids that dont necessarily apply in this context.

As for the next steps in this research, van der Meulen said that many questions remain unanswered that she hopes to address in the future.

For example, are these effects reversible? she wondered. How do they depend on the dose and duration of glucocorticoid use and the type of glucocorticoid medication used? And could selective glucocorticoid receptor modulators a type of glucocorticoid-like medication that has a more selective effect and therefore potentially [fewer] side effects prevent these effects from happening?

Dr. Danan stated that he would like to see more details regarding how long participants used prescribed steroids and whether the systemic glucocorticoid users took the medication orally or through an injection.

Those are things that as a physician [I] would want to know so that I can tie in whether or not this has a potential impact on my patient base, he added.

And Dr. Kesari said that although this study documents atrophy of the brain, more research is required to understand how that happens.

We need to do more basic science research to understand the mechanisms of how steroids are causing this brain damage, and then how we can mitigate it with other medications or reparative mechanisms in the future, whether its stem cells or growth factors that may stimulate stem cells, he said.

Read more here:
What happens to the brain on prescription steroids? - Medical News Today

Dr. Lin on Patient Eligibility for CAR T-cell Therapy in Hematologic Caners – OncLive

Yi Lin, MD, PhD, discusses patient eligibility for CAR T-cell therapy in hematologic cancers.

Yi Lin, MD, PhD, hematologist, oncologist, Department of Hematology, CAR T-Cell Therapy Program, Mayo Clinic, discusses patient eligibility for CAR T-cell therapy in hematologic cancers.

Many oncologists have experience referring patients for stem cell transplant, and referring patients for CAR T-cell therapy can work in a similar fashion, though it may be more beneficial since CAR T-cell therapy can be effective in patients with active disease, Lin explains. When referring patients to CAR T-cell therapy, it is important to consider a patients number of prior lines of therapy, their comorbidities, and organ function, Lin adds.

Though clinical trials evaluating CAR T-cell therapies in hematologic malignancies have strict parameters, it can be difficult for many patients to fall inside all of those parameters in real-world practice, Lin continues. In reports on real-world data for patients with leukemia or lymphoma who received CAR T-cell therapy, approximately half of patients treated fall outside of at least 1 parameter that was used for CAR T-cell therapies in clinical trials, Lin notes.

Through coordination between clinicians and the CAR T-cell therapy team, patients who fall outside the parameters used in clinical trials can still have comparable outcomes from CAR T-cell therapy, Lin concludes.

The rest is here:
Dr. Lin on Patient Eligibility for CAR T-cell Therapy in Hematologic Caners - OncLive

Higher Doses of CAR T-Cell Therapy May Improve Survival for Young Patients With B-Cell ALL – The ASCO Post

By The ASCO Post Staff Posted: 8/16/2022 10:52:00 AM Last Updated: 8/16/2022 3:13:09 PM

Young people with B-cell acute lymphoblastic leukemia (ALL) who received doses of tisagenlecleucel, a chimeric antigen receptor (CAR) T-cell therapy, at the higher end of the approved dosing range had significantly better survival rates at 1 year compared with those who received lower doses within this range, according to research published by Stefanski et al inBlood Advances.

Since its approval as the first gene therapy available in the United States in 2017, tisagenlecleucel has offered a welcome treatment option for pediatric patients with B-cell ALL whose cancer does not respond to chemotherapy or recurs after prior response. However, the wide dosing range approved for the therapy can pose a conundrum for doctors who sometimes must choose whether to use a higher or lower dose with little evidence to guide these decisions. This new study offers the first insights into optimal dosing based on real-world data.

In the past, we did not have data to guide clinical decisions around commercial CAR T-cell dosing and didnt know if higher doses would affect toxicity and compromise outcomes, or support enhanced antileukemia effect, said Liora Schultz, MD, a pediatric oncologist at the Stanford Children's HealthLucile Packard Children's Hospital and the studys lead author. [These data have] direct clinical applicability, as it supports use of higher dosing, as available, within the approved tisagenlecleucel dose range.

More About B-Cell ALL and Tisagenlecleucel

ALL is the most common type of cancer in children, and B-cell ALL is its most common subtype. It is often treatable with chemotherapy, but about 20% of patients either do not respond to chemotherapy or subsequently relapse. CAR T-cell therapy is becoming an integral part of standard care as an alternative or supplement to stem cell transplantation for pediatric patients with relapsed or refractory B-cell ALL.

Tisagenlecleucel is approved at a dosing range of 0.2 to 5 million CAR T cells/kg for patients weighing 50 kg or less, or 10 to 250 million CAR T cells for patients weighing over 50 kg. In practice, the number of CAR T cells that are manufactured for each patient varies substantially based on the number of T cells that are obtained initially and the rate at which the modified cells grow in the laboratory.

Any amount of CAR T cells within the approved range is considered an acceptable dose, but if more than the minimum number of cells are available within the approved range, doctors can decide whether to use a higher amount or a lower amount. Clinical trials for tisagenlecleucel provided guidance on dosing leading to the therapys approval, but real-world data are useful to fine-tune dosing and inform decision-making when there are multiple options available.

Study Details

For the study, researchers analyzed rates of overall survival, event-free survival, and relapse-free survival at 1 year among 185 patients aged 26 years or younger who received tisagenlecleucel for relapsed or refractory B-cell ALL. They found that patients who received a dose at the higher end of the approved range (between 2.4 and 5.1 million cells/kg) had significantly higher survival rates according to all three measures compared with patients who received a dose at the lower end (between 0 and 1.3 million cells/kg). In the highest-dose group, 86% of patients were alive at 1 year, compared to 59% in the lowest-dose group. Researchers did not observe any signs of increased toxicity or safety concerns with higher doses.

The findings suggest that administering doses of tisagenlecleucel at the higher end of the approved range could help to achieve a more effective and long-term responsewithout raising the toxicity risk.

A lot of effort is focused on complex engineering and development of next-generation CAR T-cell therapies, said Dr. Schultz. This study aims to explore if clinical manipulations using our current approved construct, tisagenlecleucel, can achieve even incremental advances in the field.

The researchers plan to further examine the data to determine how additional clinical variables might influence outcomes following CAR T-cell therapy.

Disclosure: For full disclosures of the study authors, visit ashpublications.org/bloodadvances.

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Higher Doses of CAR T-Cell Therapy May Improve Survival for Young Patients With B-Cell ALL - The ASCO Post

Haematopoietic Stem Cell Gene Therapy in IEM – Physician’s Weekly

Due to donor-derived cells capacity to deliver enzymes to enzyme-deficient tissues and organs for the rest of ones life, allogeneic hematopoietic stem cell transplantation (allo-HSCT) has become widely used as a therapeutic approach for many inborn errors of metabolism (IEM) over the past 30 years. However, patients who have IEM were the only ones who could clinically benefit from allo-HSCT, patients still had a large burden from their residual illness, and allo-HSCT was still linked to severe short- and long-term toxicities as well as transplant-related death.

In the 1990s, hematopoietic stem/progenitor cell gene therapy (HSPC-GT) was developed for the treatment of a few monogenic primary immunodeficiencies, and in recent years, its use has expanded to include a number of IEM. Gene-corrected hematopoietic progenitors could provide supra-physiological enzyme levels to hard-to-reach regions, such as the brain and skeleton, with a possible improved therapeutic effect. This made HSPC-GT particularly appealing in neurodegenerative IEM.

Furthermore, HSPC-GT had lower rates of morbidity and death than allo-HSCT, albeit it must be weighed against the chance of insertional mutagenesis. Clinical trials in the IEM sector were multiplying quickly, and certain HSPC-GT items had just lately been given the go-ahead. In contrast to tried-and-true treatment approaches like allo-HSCT, the review discussed the development of ex vivo HSPC-GT in a variety of IEM with an emphasis on current findings from GT clinical trials and risks vs. benefits considerations.

Reference: onlinelibrary.wiley.com/doi/10.1111/bjh.18179

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Haematopoietic Stem Cell Gene Therapy in IEM - Physician's Weekly