University Hospitals treats first cancer patient in Ohio with "game changing" CAR T therapy – News 5 Cleveland
By daniellenierenberg
CLEVELAND When 61-year-old Ken Anderson was diagnosed with Multiple Myeloma 3 years ago, he didnt know what to expect.
It kind of hits you. It hits you hard, he said. Its a blood cancer, and its in your bone marrow, and it degenerates your bones is what it does.
The cancer is incurable, but treatable.
You live with it and you have to have many rounds of chemotherapy to keep the myeloma at bay, said Dr. Ted Teknos, the president of University Hospitals Seidman Cancer Center.
With so many unknowns, the dad of 4 girls and grandfather of 2 knew one thing, he was going to fight.
You just have to look to the road ahead, he said.
For the past 3 years, that road has been filled with ups and downs and countless rounds of chemotherapy treatments and even a bone marrow transplant.
They give you your stem cells back and those regenerate and lasted for about 6 months, and then there was a relapse, said Anderson.
Through it all, he remained hopeful for a medical breakthrough. He read about the research and followed up on the results of clinical trials in something called CAR T therapy.
I didn't know how far out that would be. It didn't say how far out it was. It sounded, to me, something like 10 or 20 years.
But it wasnt 20 years, the FDA approved CAR T therapy for Multiple Myeloma patients, and University Hospitals is the first in Ohio to treat patients with it. Anderson, who is from Kirtland, is the first patient in Ohio to receive it.
These treatments, now, are available for those that have run out of options, said Dr. Teknos.
Dr. Teknos compared the treatment to something straight out of a science fiction movie.
In essence, its like a heat-seeking missile for the cells to go find the cancer and eradicate it, he said.
It works by taking a patients own white blood cells, genetically modifying them in a lab and then infusing them back into their body so the patients cells can fight off the cancer cells.
They will engineer them to attack my cancer cells, said Anderson.
Dr. Teknos calls it living therapy.
You're taking living cells out of a patient, you're modifying them, and then you're growing them up in the lab and then re-infusing them back into the patient, he said. It's their own cells that have been modified and fight the cancer.
Dr. Teknos said in clinical trials, about 75% of Multiple Myeloma patients had a response to therapy, and in 1/3 of patients, their cancer went away.
Its really a game changer, said Dr. Teknos. There are patients who literally had weeks to live and then a year and a half later, have no cancer at all.
Andersons cells are currently in the lab. He will receive his infusion next month. He is cautiously optimistic that the next stop on his journey will have him feeling better.
I won't have to be on the chemo anymore, so I'm just back to feeling like myself would be would be really exciting, he said. People who are out there and diagnosed with this, with this disease, know that we are on the cusp of some big things here in the treatment of it, and this is a huge advance.
While Anderson is currently fighting Multiple Myeloma, University Hospitals is also offering a new CAR T cell therapy treatment for patients diagnosed with Diffuse Large B-Cell Lymphoma.
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University Hospitals treats first cancer patient in Ohio with "game changing" CAR T therapy - News 5 Cleveland
How Covid-19 has disrupted efforts to care for blood cancer patients – The Independent
By daniellenierenberg
On the day of his Year 10 school prom, as other students excitedly prepared for the big occasion, then 15-year-old Rian Harvey was sat in a ward of Royal Marsden Hospital, awaiting the stem cell transplant that would save his life after a leukaemia relapse.
Despite the hot weather on that day back in July 2015, his hospital room windows had to remain sealed shut, as even the smallest bug bite could have killed him due to his compromised immune system.
Six years on, he finds himself grateful that he relapsed when he did, with five years to build his immunity before the Covid-19 pandemic hit.
Blood cancer patients are one of the most vulnerable groups of people at risk of Covid-19, according to research, being 57 per cent more likely to suffer severe disease compared to other cancer patients.
Recalling his own experience, Rian, now 22, says: Its scary, you look at everything that person has gone through, they had blood cancer and then had a stem cell transplant, they have gone through all the stress of only to be taken by a pandemic that came out of nowhere.
I know the vulnerability that you are in for stem cell transplants, Ive been there myself. Your immune system cant take anything.
Despite the high risk these patients face, charities such as Anthony Nolan, which assist blood cancer patients with finding a stem cell match, were left out of the allocated government budget that was announced in March.
The cancellation of face-to-face fundraising and events, despite the increase in demand for services, have led their gross income to be down by an estimated 5.5m for 2021.
Henny Braund, chief executive of the charity, said people with blood cancer and blood disorders were heavily impacted by the pandemic and everyone who needs treatment and support must be able to access it without delay.
This budget does not address the pressure currently facing cancer services across the UK, he adds.
Stem cell transplants are carried out to treat conditions such as blood cancer. The process involves removing the healthy stem cells of one person and transferring them to another, provided they have a similar or identical special genetic marker called the HLA.
While this match is sometimes present between family members, it is not always the case, leaving patients in the UK reliant on the British Bone Marrow Registry to find a suitable match. The odds of a match are one in 1000.
One of Anthony Nolans primary roles is to encourage more people to put themselves on the registry so patients have an increased chance to find a match. This can be done via a simple cheek swap, which provides sufficient HLA data for the initial matching process.
Will Briant, 24, from London, donated stem cells in 2015 after signing up to be on the registry at university. I think it ultimately is a huge part of who I am now, he says. Its something that I look to in my darker moments and find great inner strength from.
The identities of donors and recipients remain anonymous to one another, but they are allowed to exchange letters after the transplant.
I was incredibly emotional when I got the letter, he adds. He made it clear that not only was I giving him the chance of time for himself, but it was also for all of his family and friends, he told me he had a very big family. Looking back now, at a time where we cant all be with our families, it just highlights just how important and valuable that must have been for him.
Apart from encouraging people to sign up to the registry, the money Anthony Nolan raises go towards funding research, offering support and information to patients and families as well as providing post-transplant-care. They have helped 18,000 people find a match.
Unfortunately, they are part of the 35 per cent of charities who used the furlough scheme offered by the government to curb the loss of income. To ensure their survival, 24 per cent of surveyed charities said they were letting furloughed employees return as volunteers.
Terence Lovell, chief engagement and marketing officer at Anthony Nolan, says: We still desperately need funds to continue our life-saving work through providing stem cells transplants and co-ordinating efforts across the NHS to ensure patients receive the care and support they need.
Despite the circumstances, Rian has decided to make the most of his time in lockdown. He regularly shares his experience fighting cancer on his social media platforms and is currently in the process of writing a book and producing a podcast to further share his message.
The cancer mill is still very much open for business and I am trying to push people, that have not necessarily been through what Ive been through, to be more positive and see the world the way that I do, he says, I wake up in the morning, open my front door, take a deep breath of fresh air and I think this is amazing because five years ago I couldnt even open a window in the hospital.
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How Covid-19 has disrupted efforts to care for blood cancer patients - The Independent
Keeping the physical appointment was critical, the show of support appreciated by Renville County Commissioner – West Central Tribune
By daniellenierenberg
When he called the Olivia Hospital and Clinic to postpone his physical, he was urged to keep it. Physicals are important, he was reminded.
Keeping that date proved to be a lifesaving decision.
The physical went well, and shortly after he told his daughter that he was as fit as a horse.
But Dr. Jon Kemp, his primary physician who had urged him to keep the date for the physical, noticed a slight abnormality in a standard blood test. He recommended further testing.
On Dec. 20 Kramer was diagnosed with multiple myeloma.
Thanks to the early diagnosis, Kramer, age 62, has the means of keeping this disease at bay. Its a cancer of the plasma cells in bone marrow, and is the second most common blood cancer.
He is about to undergo a stem cell transplant this week as part of his treatment.
He learned that hes not alone on the journey ahead.
At Tuesdays meeting of the Renville County Board of Commissioners, fellow board members came wearing T-shirts proclaiming: In this county, nobody fights alone.
Organizers of the surprise sold 76 of the T-shirts to show support for Kramer and raise funds for the Renville County Walk in the Park campaign. More than 40 T-shirt wearing supporters joined the meeting via Zoom. Staff in the health department sang a song to express their support, and staff members told him they would keep him in their thoughts and prayers.
Thank you, said Kramer. He told the West Central Tribune that he was totally surprised by the display of support.
He has lots of support from family and friends, and its all-important. Kramer farms in eastern Renville County. He has lined up plenty of helping hands while he undergoes the stem cell transplant, which will sideline him for at least six weeks.
He said doctors are confident the stem cell transplant can knock the cancer into remission. They will be harvesting bone marrow cells and freezing a portion of them to make it possible to perform at least two more transplants in future years as well.
The decision to keep the date of that routine physical made all the difference. Absolutely, said Kramer.
Health providers told him that in too many cases, multiple myeloma is not diagnosed until a patient comes in with a broken leg or other bone, and wondering why. The cancer carves holes and weakens bones as it progresses unbeknownst to the person.
Thanks to the early diagnosis, Kramer said they found only pinholes in his bones, having caught the disease in the first of its three stages. He began chemotherapy in early January, and it has proven effective, he added.
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Keeping the physical appointment was critical, the show of support appreciated by Renville County Commissioner - West Central Tribune
Crawley doctor urges residents to save the life of a stranger – Crawley Observer
By daniellenierenberg
Dr Pria Suchak, 31, initially registered with blood cancer charity DKMS last July, when she was inspired by a message on social media.
Every 20 minutes someone in the UK is diagnosed with a blood cancer those that affect the body's bone marrow, blood or lymphatic system - such as leukaemia, myeloma or lymphoma.
Yet, only two per cent of the UK population are registered as potential blood stem cell donors.
Pria said: My friends nephew had leukaemia, so she was using her Facebook page to encourage strangers to sign up him.
"Her nephew is of mixed heritage - half Chinese and half Caucasian. So she was trying to encourage more people for minority ethnic communities to sign up.
"I wanted to help give someone a second chance of life, so I signed up with DKMS, and my husband registered at the same time.
Patients from black, Asian or other minority backgrounds have a 20 per cent chance of finding the best possible blood stem cell match from an unrelated donor, compared to 69 per cent for northern European backgrounds.
Pria ordered a home swab kit in July 2020 and was contacted by DKMS just five months later, informing her that she was a potential match for a stranger in need of a lifesaving blood stem cell transplant.
The mum-of-two said: I received a call from a lady at DKMS. She said I was extremely close to being a match, but there were also eight other people who were identified as possible matches too.
"A few weeks later, I received another call from DKMS saying that I was the best match out of the nine potential donors.
"I didnt expect that. As it was nine of us in total, you never expect you'll be chosen.
Following further tests and a medical examination, a date was set for Pria to donate her blood stem cells by peripheral blood stem cell collection (PBSC).
In the run-up to the procedure, donors are given a drug with the growth factor G-CSF to increase the number of stem cells in the blood.
Pria said: At the time I had so many things going on. We had just gotten past Christmas, both of my children had birthdays in January, and I was about to sit a final GP exam.
"DKMS were excellent and did their best to schedule my G-CSF injections the day after I sat the exam. Of course, they checked that this wouldnt impact the patient.
My actual donation was really nice, especially as there were other donors in the room at the same time donating for other patients.
"We all got on really well and chatted loads. The clinicians told us that we were the chattiest group they had ever had. Ive remained terrific friends with one of my fellow donors.
Because of the minimum two-year anonymity period in the UK, donors can only contact the patient anonymously, by letter or email.
Pria said: I dont know anything about my patient other than she is a woman. She really is a stranger, but I hope my blood stem cells help her to live a long life.
I strongly encourage people in Crawley to register with DKMS. By donating their blood stem cells, not only will you potentially help a stranger in desperate need, but you'll also help their family and friends by giving them more time together.
Crawley has a population of around 114,000 with 14 neighbourhoods, the largest inland town in West Sussex. Yet, just 865 residents have registered with DKMS.
On May 28, DKMS celebrates their day of awareness - World Blood Cancer Day. This May, the charity aims to register 2,000 new registrations (roughly one for every donor in the UK waiting) by the end of May 28.
If you are called upon to donate your blood stem cells it is because you are likely the patients best match.
There are two donation methods. Around 90 per cent of all donations are made through a method called peripheral blood stem cell (PBSC) collection.
This method is very similar to giving blood. It involves being connected to an apheresis machine. Apheresis means 'to separate'.
This machine separates blood being taken from one of the donor's arms, and separates the blood stem cells from it. The donor's blood is then returned to them through their other arm. This is an outpatient procedure that is usually completed in four-to-six hours.
In just ten per cent of cases, donations are made through bone marrow collection. Bone marrow is taken from the pelvic bone under general anaesthetic, and this lasts around an hour.
DKMS need blood stem cell donors from all backgrounds. If you are aged between 17-55 and in good general health, you can support Gareth and the other 2,000 people in need of a lifesaving blood stem cell transplant by registering online at http://www.dkms.org.uk/register-now for your home swab kit.
By registering, you'll join a group of over 840,000 other DKMS lifesavers-in-waiting, ready to make a difference by giving someone a much-needed second chance of life.
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Crawley doctor urges residents to save the life of a stranger - Crawley Observer
BMSCs differentiated into neurons, astrocytes and oligodendrocytes alleviated the inflammation and demyelination of EAE mice models – DocWire News
By daniellenierenberg
This article was originally published here
PLoS One. 2021 May 13;16(5):e0243014. doi: 10.1371/journal.pone.0243014. eCollection 2021.
ABSTRACT
Multiple sclerosis (MS) is a complex, progressive neuroinflammatory disease associated with autoimmunity. Currently, effective therapeutic strategy was poorly found in MS. Experimental autoimmune encephalomyelitis (EAE) is widely used to study the pathogenesis of MS. Cumulative research have shown that bone marrow mesenchymal stem Cells (BMSCs) transplantation could treat EAE animal models, but the mechanism was divergent. Here, we systematically evaluated whether BMSCs can differentiate into neurons, astrocytes and oligodendrocytes to alleviate the symptoms of EAE mice. We used Immunofluorescence staining to detect MAP-2, GFAP, and MBP to evaluate whether BMSCs can differentiate into neurons, astrocytes and oligodendrocytes. The effect of BMSCs transplantation on inflammatory infiltration and demyelination in EAE mice were detected by Hematoxylin-Eosin (H&E) and Luxol Fast Blue (LFB) staining, respectively. Inflammatory factors expression was detected by ELISA and RT-qPCR, respectively. Our results showed that BMSCs could be induced to differentiate into neuron cells, astrocytes and oligodendrocyte in vivo and in vitro, and BMSCs transplanted in EAE mice were easier to differentiate than normal mice. Moreover, transplanted BMSCs reduced neurological function scores and disease incidence of EAE mice. BMSCs transplantation alleviated the inflammation and demyelination of EAE mice. Finally, we found that BMSCs transplantation down-regulated the levels of pro-inflammatory factors TNF-, IL-1 and IFN-, and up-regulated the levels of anti-inflammatory factors IL-10 and TGF-. In conclusion, this study found that BMSCs could alleviate the inflammatory response and demyelination in EAE mice, which may be achieved by the differentiation of BMSCs into neurons, astrocytes and oligodendrocytes in EAE mice.
PMID:33983943 | DOI:10.1371/journal.pone.0243014
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BMSCs differentiated into neurons, astrocytes and oligodendrocytes alleviated the inflammation and demyelination of EAE mice models - DocWire News
Outlook for multiple myeloma: Figures and factors that affect it – Medical News Today
By daniellenierenberg
Multiple myeloma is a type of cancer that originates from white blood cells called plasma cells. Many factors affect the outlook for a person with this disease, including their age, overall health, and kidney function, as well as the stage of cancer at diagnosis.
Multiple myeloma is a cancer of the plasma cells, which are a type of white blood cell. Over time, myeloma cells multiply and accumulate in the bone marrow and solid parts of the bones.
Multiple myeloma can lead to organ damage that affects the kidneys, the bones, and the overall immune system.
In this article, we look at the outlook for people with different stages of multiple myeloma. We also look at the symptoms and treatment of multiple myeloma and what can affect a persons outlook.
The American Cancer Society (ACS) estimates that doctors will diagnose 34,920 new cases of multiple myeloma in 2021 and that there may be 12,410 deaths from the disease.
When a person receives a multiple myeloma diagnosis, the doctor will use the Revised International Staging System (RISS) to determine the stage of the cancer. This staging system is based on:
A person will receive a diagnosis of either stage 1, 2, or 3 multiple myeloma. There is also a stage 0, a slow-growing type of multiple myeloma that is called smoldering myeloma.
However, survival rates are based on summary staging, which the Surveillance, Epidemiology and End Results (SEER) program developed. This staging system groups cancers into:
As multiple myeloma does not spread to the lymph nodes, the regionalized stage is not relevant to this cancer.
The 5-year relative survival rate for multiple myeloma is as follows:
These statistics mean that a person with localized multiple myeloma is 75% as likely as someone without multiple myeloma to live for 5 years after receiving the diagnosis.
People who receive a smoldering myeloma diagnosis can live for years without any treatment. Additionally, beginning treatment early does not appear to affect the outlook.
The stage of multiple myeloma is among the factors that can affect a persons outlook.
Other factors include:
A small 2014 study involving 82 people with an average age of 61 years found that those with damaged kidneys had a median survival rate of 13 months, whereas those without kidney damage lived for an average of 41 months.
Additionally, changes in chromosomes and genetic abnormalities can affect a persons outlook. The specific chromosomal abnormalities that doctors consider high risk affect chromosomes 4, 14, 16, and 17.
The treatment for smoldering myeloma typically consists of watchful waiting, as this stage is slow growing.
Drug therapy for multiple myeloma consists of:
Other treatment options include:
Multiple myeloma can cause:
A doctor may recommend supportive therapies to help manage these side effects. These may include surgery to help support weakened bones and prevent fractures.
Learn more about the treatment options and how to manage the symptoms.
A person should contact a healthcare professional if they notice any symptoms of multiple myeloma.
After receiving a multiple myeloma diagnosis, a person may want to ask the following questions:
Multiple myeloma is a type of cancer that affects the blood. The outlook for people with multiple myeloma depends on the stage of the cancer at the time of diagnosis. It also depends on how well a persons kidneys are functioning and their age and overall health.
However, different treatment options are available. A person should talk with a healthcare professional about which treatment options would best suit them.
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Outlook for multiple myeloma: Figures and factors that affect it - Medical News Today
How One Round of Gene Therapy Fixed 48 Kids’ Immune Systems – Singularity Hub
By daniellenierenberg
Gene therapy has shown promise in recent years for treating a range of diseases, including sickle-cell anemia, hemophilia, various forms of inherited blindness, mesothelioma, and Duchenne muscular dystrophy. A new success story may soon be added to this list, with the publication yesterday of the outcomes of a clinical trial that used gene therapy to cure a rare immune system disorder in infants.
The study, described in the New England Journal of Medicine, was carried out by researchers from UCLA and Great Ormond Street Hospital in London over the course of five years, beginning in 2012.
Adenosine deaminase (ADA) is an enzyme found in a type of white blood cell called lymphocytes, which are primarily active in the brain, GI tract, and thymus gland. Lymphocytes make antibodies and attack infected cells, so theyre pretty crucial to the immune system.
ADAs job is to convert a molecule thats harmful to lymphocytes into a non-harmful version of itself. If ADA cant work its magic, that molecule starts to build up in lymphocytes, becoming toxic and ultimately killing the cellsand leaving the immune system virtually defenseless, highly vulnerable to invaders like viruses and bacteria.
Mutations in the ADA gene mean the body doesnt make enough of the enzyme to successfully do its job. This deficiency of ADA leads to a condition called severe combined immunodeficiency (SCID). Those suffering from SCID can not only get sick very easily, but conditions that would be neutralized by a normal immune system quickly become deadly for them.
SCID was more commonly known as bubble boy disease after David Vetter, a boy born in Texas in 1971, spent 12 of his 13 years of life enclosed in a plastic bubble to protect him from germs.
About 20 different genetic mutations can cause SCID; ADA-SCID refers to immunodeficiency caused by lack of the ADA enzyme: severe combined immunodeficiency due to adenosine deaminase deficiencya bit of a mouthful. The worst part of ADA-SCID is that it occurs in babies; most are diagnosed with the condition before theyre even six months old, and without treatment they typically dont live past age two.
ADA is rare, estimated to occur in about 1 in 200,000 to 1,000,000 newborns worldwide; both the mothers and the fathers ADA gene must have mutations for the child to end up with this condition.
The first step in the gene therapy treatment was to collect hematopoietic stem cells, which are those that manufacture blood cells, from the patients. The researchers then inserted an intact copy of the ADA gene into the stem cells using an RNA virus called a lentivirus (the most well-known lentivirus is HIV).
The altered cells were re-injected into the patients, where they started producing ADA normally, yielding healthy immune cells.
Out of 50 total patients30 in the US and 20 in the UKwith ADA-SCID, 48 appear to have been rid of their condition thanks to the gene therapy, with no complications reported. The two patients who didnt have success with the therapy went back to traditional treatment methods, and didnt experience any adverse effects as a result of having tried the therapy.
If, or hopefully when, gene therapy becomes the go-to treatment for ADA-SCID, it will be a welcome reprieve from traditional options, which are neither pleasant nor cheap: patients need weekly injections of ADA until a bone marrow transplant can be done, and absent a donor, they must consistently receive injections, take antibiotics, and undergo antibody infusions for life.
If approved in the future, this treatment could be standard for ADA-SCID, and potentially many other genetic conditions, removing the need to find a matched donor for a bone marrow transplant and the toxic side effects often associated with that treatment, said Dr. Claire Booth, co-author of the study and a consultant in pediatric immunology and gene therapy at Londons Great Ormond Street Hospital.
Theres no mention of the cost of the therapy, nor whether this could be a prohibitive factor to making it a viable option. Nonetheless, the study is encouraging not just for its potential to revolutionize treatment of ADA-SCID, but as a harbinger for the promise of gene therapy for a multitude of genetic conditions.
People ask us, is it a cure? Who knows long term, but at least up to three years, these children are doing well, said Dr. Stephen Gottschalk, who was not involved in this study but performed a similar gene therapy on kids with SCID at St. Jude Childrens Research Hospital in Memphis. The immune function seems stable over time so I think it looks very, very encouraging.
Image Credit: liyuanalison from Pixabay
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How One Round of Gene Therapy Fixed 48 Kids' Immune Systems - Singularity Hub
Genenta Phase I/II Glioblastoma Data at ASGCT Show Temferon Delivered Tumor-Focused Interferon ExpressionData presented at the 2021 American Society…
By daniellenierenberg
MILAN, Italy and NEW YORK, May 14, 2021 (GLOBE NEWSWIRE) -- Genenta Science, a clinical-stage biotechnology company pioneering the development of an investigational hematopoietic stem progenitor cell immuno-gene therapy for cancer (Temferon), will present new clinical data from a Phase I/IIa study of Temferon in patients affected by glioblastoma multiforme (GBM) in an oral presentation at the 2021 American Society for Gene and Cell Therapy (ASGCT) Annual Meeting, taking place virtually on May 11-14, 2021.
The data presented at ASGCT are from Genentas ongoing trial of Temferon in patients with GBM. The presentation focuses specifically on patients who have undergone a follow-up surgical procedure for their cancer. In addition to being a treatment option, follow-on surgery provides investigators with an opportunity to understand the impact of therapies at a cellular and molecular level.
The ASGCT presentation shows that genetic markers of Genentas Temferon were detectable in tumor specimens from all four patients with progressive disease who underwent follow-on surgery. Furthermore, the expression of interferon- (IFN) responsive gene signatures in those tumors was increased compared with pre-treatment levels, which suggests that interferon- (IFN-) had been released locally in the tumor by cells derived from Genentas investigational treatment.
Carlo Russo, Chief Medical Officer at Genenta Science, said: These preliminary results provide exciting indications that Temferon acts in the way we anticipated even in the relatively inaccessible setting of glioblastoma multiforme. The data are encouraging and in line with our pre-clinical results, with early evidence that Temferon delivers biological effects that may impact the progression of individual lesions.
One of the four patients had two lesions removed at the second surgery; one was a prior lesion that had not been removed during the first surgery and was stable; the other was a relapsing progressing lesion that had developed at the first surgery site. Compared with the progressing tumor, the stable lesion displayed a higher proportion of T cells and Tie2 Expressing Monocytes (TEMs) within the myeloid infiltrate and had a higher IFN-response signature.
The data presented at ASGCT also supported the initial safety and tolerability profile of Temferon. Concentrations of IFN- in the plasma and cerebrospinal fluid of patients remained low, while IFN- responses were identified in myeloid cells that infiltrate tumors. Temferon-derived differentiated cells also persisted in peripheral blood and bone marrow for up to 18 months at lower levels, indicating the potential durability of the intervention. No dose limiting toxicities have been identified.
Presentation Details:
Title: Changes in the Tumor Microenvironment in Patients with Glioblastoma Multiforme Treated with IFN-a Immune Cell & Gene Therapy (TEM-GBM_001 Study)
Time: Friday May 14, 2021 at 1.30 PM Eastern Time (7.30 PM CET)
Presenting: Carlo Russo, CMO
To access the abstract please visit https://annualmeeting.asgct.org/
About Genenta Science
Genenta (www.genenta.com) is a clinical-stage biotechnology company pioneering the development of a proprietary hematopoietic stem cell gene therapy for the treatment of a variety of cancers. Temferon is based on ex-vivo gene transfer into autologous hematopoietic stem/progenitor cells (HSPCs) to deliver immunomodulatory molecules directly via tumor-infiltrating monocytes/macrophages (Tie2 Expressing Monocytes - TEMs). Temferon, which is under investigation in a Phase I/IIa clinical trial in newly diagnosed glioblastoma multiforme patients, is not restricted to pre-selected tumor antigens nor type and has been designed to reach solid tumors, one of the main unresolved challenges in immuno-oncology. Genenta is based in Milan, Italy, and New York, USA.
About Glioblastoma MultiformeGlioblastoma multiforme (GBM) is a rapidly-growing cancer of the glial cells that support the nerve cells within the brain. The main treatment for GBM is surgery to reduce the bulk of the tumor, which can prolong the lives of patients and to improve quality of life. A second round of surgery is increasingly considered to have significant benefit in prolonging the lives of patients with GBM. Even with treatment, GBM virtually always recurs, typically resulting in death within the first 15 months from diagnosis.
Two local sisters share an unbreakable bond after bone marrow donation – Riverhead News Review – Riverhead News Review
By daniellenierenberg
Lorraine LaRosa faced a seemingly impossible decision.
She knew how fortunate she was to have not one, but three perfect matches for a bone marrow transplant, a procedure used to treat several cancerous and noncancerous diseases, such as leukemia and Hodgkins lymphoma. The statistics for finding a perfect match can be grim. The best odds rest in an immediate family member. Otherwise, a patient must rely on the bone marrow registry and the slim chance of matching with a stranger.
Ms. LaRosa had the benefit of a large family. Out of seven siblings, four were healthy enough to be tested. And three came back as matches: her sisters Jennifer Lappe and Melissa Senatore, who live in Calverton, and her brother Jason Klinge of Southampton.
I was in tears because I didnt know what to do and who was the better choice, Ms. LaRosa said.
A sense of urgency had arrived in early 2020. Ms. LaRosa, 62, who lives in Mattituck, was undergoing frequent blood transfusions due to a lack of platelets and low red blood cells.
Things were getting pretty bad at that point, she said.
She didnt want to put the burden on any of her siblings. She called her doctor to talk through her concerns. The time had come to move forward, so the doctor took the decision out of her hands.
After careful evaluation of all three siblings health and medical histories and considering Ms. LaRosas worsening situation the doctors choice became clear. The donor would be her sister Jennifer.
Ms. Lappe understood her sisters hesitancy to ask forsuch a serious commitment. But there was never a moment of doubt. She had seen her sister struggle for years with her illness and have to endure the uncertainty of misdiagnosis and multiple procedures.
I knew she was scared, Ms. Lappe said. Id be scared with what she had to go through. But shes a lot stronger than I think she thinks she is.
Ms. LaRosa texted her sister with the news that unfortunately she would be the donor.
To me there was never a question, said Ms. Lappe, 60. Ill do whatever you need. Im in a million percent. I said, Im selfish, I love you. I want you to be around forever.
The sisters, who were always so close from a young age and grew up in a tight-knit family, would soon form an unbreakable bond one saving the others life.
Before Ms. LaRosa received an ultimate diagnosis of myelodysplastic syndrome, or MDS, in February 2020, she endured years of joint pain and symptoms that seemed to mystify her doctors. One time when her shoulder hurt, she was told it was a torn rotator cuff, which turned out to be inaccurate. Before that, when she was struggling to walk with swollen feet, a podiatrist said she had a torn Achilles tendon, but she hadnt done anything that seemed to warrant an injury typically seen in athletes. Lupus was also incorrectly diagnosed.
She struggled on a continual journey from one doctor to another.
She ended up in an emergency room on Feb. 27, 2017, and a doctor there noticed that her platelets the smallest blood cells seemed low.
Two months later, a doctor at New York Cancer & Blood Specialists diagnosed large granular leukemia, a rare cancer of white blood cells. As time went on, however, her condition did not improve.
Ms. LaRosas daughter, Taylor, described her mom as a fighter who was always optimistic and never overly concerned about her health issues.
I was more so the worrywart, said Taylor, 29. I kind of forced her to go to all these appointments and all of these doctors chasing all of these kind of vague symptoms. No one could kind of come up with what was going on.
A 2009 Mattituck High School graduate, Taylor works as a physician assistant at Weill Cornell Medical Center in New York City. She connected her mom with Dr. Gail Roboz, who specializes in hematology/oncology, in November 2017. Dr. Roboz became well known as the doctor for Robin Roberts, an anchor of ABCs Good Morning America, who was diagnosed with MDS in 2012.
[Dr. Roboz] kind of took on my case and was monitoring me and was saying my blood didnt really make any sense, Ms. LaRosa said. There were mutations in my blood that werent making sense for the large granular leukemia diagnosis.
Extensive testing revealed that Ms. LaRosa had a predisposition to MDS, a bone barrow disorder and blood cancer that often goes unrecognized and under-diagnosed, according to the MDS Foundation.
Then it was kind of a weird watching and waiting game, Taylor said. I think we all hoped it couldnt turn into this [MDS], but we knew it could.
Low-risk patients who do not receive a bone marrow transplant face an average survival rate of up to six years, according to the MDS Foundation. High-risk patients face as little as five months.
Taylor said she braced herself for the possibility that her mother would need a bone marrow transplant at some point. Each December, her mother would undergo a checkup with her oncologist.
Taylor examined her mothers lab work after the December 2019 appointment and could see the results were abnormal. The family had booked a cruise around Christmastime, so Taylor reached out to her mothers oncologist to see if it would be safe to travel.
Dr. Roboz gave them the OK and said theyd deal with it when they returned.
We went on this cruise and I didnt know anything, Ms. LaRosa said. My husband didnt know anything, but my daughter had all this information. She had some emotional moments on the cruise. Now, looking back, I know why.
Taylor recalled the trip to the Bahamas like something out of a movie, where nothing went wrong.
It was like a perfect trip, she said.
When Ms. LaRosa returned to her doctor for a follow-up, the reality of the situation set in. Dr. Roboz referred her to Dr. Tsiporah Shore, who has expertise in bone marrow transplants, at Weill Cornell Medicine. They met on March 14, 2020.
She basically said we need to do this right away, Ms. LaRosa said. Things were really progressing.
Ms. Lappe could see her sisters health was declining.
Nothing they did was making her better and I know this was something she dreaded doing, she said.
Before determining who would be the match, Ms. Lappe said she underwent the most extensive testing of her life. To find a match, doctors analyze the patients tissue type, specifically the human leukocyte antigen, or HLA, tissue, the proteins found on most cells in the body, according to the nonprofit organization Be the Match.
Finding the match is just the initial step in assuring that the donor is suitable for the transplant and there are no other potential ailments that could be passed on.
Ms. Lappe had assumed her brother Jason would be the pick since she had an autoimmune disease and he did not. When she found out she had been selected, it coincided with the early stage of the pandemic. That added another layer of stress, since Ms. Lappe knew if she came down with the virus, it could upend the entire process.
Other questions loomed over her.
Youre worrying, is my body going to do what it needs to do? Is it going to work? Will her body reject it? she said.
To begin the donation last July, Ms. Lappe received injections to increase her white blood cell count. At the same time, her sister was undergoing radiation and chemotherapy to essentially wipe her immune system clear, eliminating a lifetime of protections that had been built up.
Ms. Lappe said she had been warned shed feel pain in her bones from the shots. When she didnt feel anything after the first shot, she worried it might not be working.
By the third and fourth shot, there was no mistaking the odd sensation.
You have these bone pains, she said. Ive never had that happen.
On the fifth day, the doctors did a blood test as the final determination to begin the donation process.
To read more about bone marrow donation, visit BeTheMatch.org.
The procedure, called peripheral blood stem cell donation, required Ms. Lappe to be connected to a machine for six hours as blood was removed via a port in her chest to separate out the blood-forming cells. The remaining blood circulated back into her body.
At the end of it, one bag of the pinkish liquid that would be used to save her sister had been accumulated.
I said to her afterwards, it was so emotional, Ms. Lappe said, adding that she knew she would feel an overwhelming sense of guilt if the procedure didnt work.
She took a picture of the bag and its label, which read, Donor: Jennifer Lappe and Recipient: Lorraine LaRosa. She texted the picture to her sister and said, Oh, my gosh.
Ms. Lappe finished her donation on a Wednesday and her sister began to receive her bone marrow the next morning, once the doctors had determined they had a sufficient number of stem cells to start the process.
Then the waiting game began.
The day of a transplant is Day Zero. Every day afterward continues an upward count toward engraftment, when the blood-forming cells received during the transplant begin to grow and create healthy blood cells.
I would say those days were the hardest, just waiting, Taylor said. They would draw her labs every morning at 4 a.m. and the results would be back at 6 a.m.
A nurse would write the number on a board, and for several days it remained at zero. To pass the time, Ms. LaRosa would play games like Yahtzee with her husband, Mark, who commuted each day into the city. Taylor would watch Netflix shows like Jane the Virgin with her mom. The days were largely a blur for Ms. LaRosa.
Taylor knew it could take one to two weeks for engraftment to begin. It was Day 11 when they saw the first sign of hope as a nurse wrote .1 on the board, signaling the first sign of growth.
I remember that day being like a huge relief and huge turn, Taylor said.
Ms. LaRosa spent over a month in the hospital for close monitoring as her counts continued to climb. Even after she was released, she had to stay at a nearby hotel for another week because of daily checkups. She set her sights on Day 100, another milestone moment in the recovery.
If you make past Day 100, its a good thing, she said.
Even after a successful procedure with a 100% match, theres never a moment of being entirely in the clear. Ms. LaRosa will continue to be monitored for the rest of her life and setbacks are always possible. Shes faced one setback already, with graft vs. host disease, which can be common after a bone marrow transplant. Shes also endured blood clots.
But the biggest thing is that shes now clear of MDS and feeling better than before the procedure. She still, however, faces residual effects from chemotherapy. Shes often tired.
When she returned home, she mostly stayed inside, unable to venture out with the threat of COVID-19 still hanging over everything. Her immune system was rebuilding from scratch. She remains on a special diet. She cant have plants in the house, which put her at risk of exposure to pathogens that can cause disease. She cant have alcohol.
I said, God, I really want a glass of wine, Ms. LaRosa said with a laugh.
Taylor said there are constant signs of progress. Her mother just recently had a port removed from her chest wall after close to nine months. She received her COVID-19 vaccine. Her hair is growing back.
Shes starting to like the style, Taylor said.
She looks forward to the next steps of returning to normal: going to a movie theater and eating dinner at their favorite restaurant, Grana in Jamesport. When they sit together and toast their wine glasses, Taylor said she knows shell cry. They have always shared a close bond, particularly since Taylor was adopted at around 3 months old after her mother endured years of infertility issues.
Shes been my best friend and my rock for my whole life, Taylor said.
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Two local sisters share an unbreakable bond after bone marrow donation - Riverhead News Review - Riverhead News Review
Stem Cell Therapy Market worth $401 million by 2026 – Exclusive Report by MarketsandMarkets – PRNewswire
By daniellenierenberg
CHICAGO, April 28, 2021 /PRNewswire/ -- According to the new market research report "Stem Cell Therapy Marketby Type (Allogeneic, Autologous), Therapeutic Application (Musculoskeletal, Wound & Injury, CVD, Autoimmune & Inflammatory), Cell Source (Adipose tissue, Bone Marrow, Placenta/Umbilical Cord) - Global Forecasts to 2026", published by MarketsandMarkets, the global market is projected to reach USD 401 million by 2026 from USD 187 million in 2021, at a CAGR of 16.5% during the forecast period.
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The Market growth is driven mainly by factors such as increasing investment in stem cell research and the rising number of GMP-certified stem cell manufacturing plants. However, factors such as ethical concerns and the high cost of stem cell research and manufacturing process likely to hinder the growth of this market.
The adipose tissue-derived MSCs segment accounted for the largest share of the cell source segment in the Stem Cell Therapy Market in 2020.
Based on the cell source from which stem cells are obtained, the global market is segmented into four sources. These include adipose tissue-derived MSCs (mesenchymal stem cells), bone marrow-derived MSCs, placenta/umbilical cord-derived MSCs, and other cell sources (which includes human corneal epithelium stem cells, peripheral arterial-derived stem cells, and induced pluripotent stem cell lines). In 2020, adipose tissue-derived MSCs accounted for the markets largest share due to their increasing utilization in treating inflammatory diseases and wounds & injuries. There are several associated advantages, such as ease of harvesting stem cells by minimally invasive methods, simplicity of the isolation procedure, and better quality & proliferation capacity of adipose tissue-derived stem cells.
The musculoskeletal disorders segment accounted for the largest share of the therapeutic application segment in the Stem Cell Therapy Market in 2020
Based on therapeutic application, the global market is segmented into musculoskeletal disorders, wounds & injuries, cardiovascular diseases, surgeries, inflammatory & autoimmune diseases, neurological disorders, and other therapeutic applications (which include ocular diseases, fat loss, and peripheral arterial diseases). In 2020, the musculoskeletal disorders segment accounted for the largest share of the therapeutic application segment. The large market share of this segment is attributed to the increasing prevalence of musculoskeletal disorders such as osteoarthritis, bone repair, and regeneration
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The Asia Pacific region is the fastest-growing region of the Stem Cell Therapy Market in 2020.
The Asia Pacific region is estimated to grow at the highest CAGR in the market during the forecast period. Some of the major factors fueling the growth of the APAC market include regulatory approvals and guidelines for product approvals and the presence of major stem cell players in countries such as South Korea, Japan, India, and Australia.
Key players in the Stem Cell Therapy Market include Smith & Nephew (UK), MEDIPOST Co., Ltd. (South Korea), Anterogen Co., Ltd. (South Korea), PHARMICELL Co., Ltd. (South Korea), JCR Pharmaceuticals Co., Ltd. (Japan), and NuVasive, Inc. (US).
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Stem Cell Manufacturing Market by Product (HSCs, MSCs, iPSCs, ESCs, Instruments, Media, Consumables), Application (Research, Target Identification, Therapy (Autologous, Allogeneic), Cell Banks), End User (Pharma, Hospitals) - Global Forecast to 2023https://www.marketsandmarkets.com/Market-Reports/stem-cell-manufacturing-market-70743403.html
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Bone Marrow Processing Systems Market Quantitative Market Analysis, Current and Future Trends – Good News Gum
By daniellenierenberg
Bone marrow aspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.
The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.
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Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.
In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.
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Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.
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Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.
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Bone Marrow Processing Systems Market Quantitative Market Analysis, Current and Future Trends - Good News Gum
Hematopoietic Stem Cell Transplantation (HSCT) Global Market Report (2020-2027) Segmented by Type, Application and region (NA, EU, and etc.) The…
By daniellenierenberg
The objective of the study is to define market sizes of different segments and countries in previous years and to forecast the values to the next Five years. The report is designed to incorporate both qualify qualitative and quantitative aspects of the industry with respect to each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as drivers and restraining factors which will define the future growth of the Hematopoietic Stem Cell Transplantation (HSCT) market.
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Major Participators LandscapeThese market players enjoyed broad industry coverage, outstanding operational ability, and strong financial resources. Manufacturers are focusing on product innovation, brand extension, and the introduction of new brands to cater to the preferences of consumers. Some of them will be endowed with vital future while others will show a weak growth during the prospective timeframe.Major market participators covered in our report are:Cryo-Save AG ViaCord Inc Lonza Group Ltd Pluristem Therapeutics Inc China Cord Blood Corp CBR Systems Inc Regen Biopharma Inc Escape Therapeutics Inc
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Hematopoietic Stem Cell Transplantation (HSCT) Market: Application OutlookPeripheral Blood Stem Cells Transplant (PBSCT) Bone Marrow Transplant (BMT) Cord Blood Transplant (CBT)
By Type:Allogeneic Autologous
Table of Content1 Report Overview1.1 Product Definition and Scope1.2 PEST (Political, Economic, Social and Technological) Analysis of Hematopoietic Stem Cell Transplantation (HSCT) Market2 Market Trends and Competitive Landscape3 Segmentation of Hematopoietic Stem Cell Transplantation (HSCT) Market by Types4 Segmentation of Hematopoietic Stem Cell Transplantation (HSCT) Market by End-Users5 Market Analysis by Major Regions6 Product Commodity of Hematopoietic Stem Cell Transplantation (HSCT) Market in Major Countries7 North America Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis8 Europe Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis9 Asia Pacific Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis10 Latin America, Middle East & Africa Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis 11 Major Players Profile
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Regional Segment AnalysisThe report focuses on detailed analysis of major regions like North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Columbia), and Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa).
Report Key AudienceHematopoietic Stem Cell Transplantation (HSCT) manufacturersDownstream vendors and end-usersTraders, distributors, and resellers of Hematopoietic Stem Cell Transplantation (HSCT)Hematopoietic Stem Cell Transplantation (HSCT) industry associations and research organizationsProduct managers, Hematopoietic Stem Cell Transplantation (HSCT) industry administrator, C-level executives of the industriesMarket Research and consulting firms
Hematopoietic Stem Cell Transplantation (HSCT) Report Provide:Potential opportunities and challenges analysis in Hematopoietic Stem Cell Transplantation (HSCT) market.Current and future market outlook in the developed and emerging regional markets.Detailed analysis of the segment that is expected to dominate the market.Regions that are expected to witness the fastest growth during the forecast period.Identify the latest developments, market shares, and strategies employed by the major market players.Comprehensive & in-depth research and after-sales warranty by Global Market Monitor.Analysis of Influences of COVID-19 to the present and future Hematopoietic Stem Cell Transplantation (HSCT) market and related industry.
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Hematopoietic Stem Cell Transplantation (HSCT) Global Market Report (2020-2027) Segmented by Type, Application and region (NA, EU, and etc.) The...
Desperate family of boy, 9, with leukaemia have 10 days to save his life… – The Sun
By daniellenierenberg
THE FAMILY of a nine-year-old leukaemia patient have been given 10 days to raise funds for life-saving treatment.
Nathaniel Nabenas family are appealing as he "clings on to life" after they were told they have until May 12 to find 201,000 for a stem cell transplant.
2
Without the operation, his cancer will be terminal.
Nathaniel is not entitled to free NHS treatment because he is not a British national.
He flew to the UK to have a 5,000 prosthetic eye fitted privately after losing it to a tumour in his home country Nigeria.
Doctors at Londons Great Ormond Street Hospital, moved by Nathaniels plight, have revealed they have waived their private consultant fees to help.
And Paul OGrady, who presents ITV series Little Heroes at the childrens hospital, has voiced his support.
It was only when he arrived here in November that doctors discovered he had acute myeloid leukaemia, a cancer so aggressive that he could have died within weeks without chemotherapy.
A stem cell match has been found but the family now have to find 201,103.
This money goes to the NHS for the cost of the transplant, treatment and after-care, based on a typical in-patient admission of eight weeks and a three-month follow-up as an outpatient.
2
Nathaniels cancer is in remission after six rounds of chemo but his consultant says it could return at any time.
If they raise enough, then a transplant will go ahead after tests due to take place on May 14.
Parents Ebisidor, a business analyst, and wife Modupe, 38, who are staying with family in Croydon, South London, were initially told the hospital bill could be as much as 825,000.
Ebisidor, 45, told the Mirror: Weve seen a dramatic turnaround from the hopeless situation we were in six months ago and we cant thank Sunday People readers enough.
Its incredible that the doctors are treating him in their private work without charging. They are wonderful. We are so grateful to everyone for giving us hope but at the same time asking people to help Nathaniel cling on to life. We know its a lot to ask.
Professor Ajay Vora, a consultant paediatric haematologist at GOSH, said the superhero fan had been incredibly brave.
But he warned: The cancer could come back at any time and the longer we wait the more likely it will return. Then Nathaniel will only have the option of palliative care.
"The tests we are doing in two weeks will reassure us it hasnt started to come back before we give him the transplant.
Prof Vora added: All the consultants involved in his care are working in a private capacity and have waived their fee because they want to help him.
Our time is not borrowed from the NHS because we are treating Nathaniel in our private service in our time.
Doctors had hoped Nathaniel would be able to have a bone marrow transplant from one of his two sisters Nadia, 11, and Nicole, 21 months. But they were not a match.
Instead, stem cells from a stored umbilical cord will be used to save him.
Doctors will give Nathaniel high doses of chemotherapy to kill off his stem cells and replace them with healthy ones.
Dad Ebisidor said: The faster we can do this transplant the more chance Nathaniel has of survival.
"We dont have this sort of treatment back home. We didnt bring him to the UK sick. He got poorly while he was here. If the operation doesnt work our only option will be to take him to a hospice.
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By law, non-UK residents get free emergency care but are charged for operations if they are admitted to hospital.
They pay for treatment at 150% of the NHS national tariff the cost normally incurred for eligible patients.
A Great Ormond Street spokesman said: Nathaniel has responded well to treatment, with our clinical teams working to provide the best care for him including looking at taking advantage of the short window of time for receiving a transplant.
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Desperate family of boy, 9, with leukaemia have 10 days to save his life... - The Sun
Orca-T Offers an Alternative to HSCT With Improved Patient Experience – OncLive
By daniellenierenberg
Advances in the treatment of patients with leukemias and lymphomas have led to a significant improvement in survival, which has increased the need for bone marrow transplant as a later-line therapy, said Mehrdad Abedi, MD, who added that Orca-T, a high precision cell therapy, confers significant antitumor activity, minimizes the incidence of acute and chronic graft-vs-host disease (GVHD), and causes less adverse effects (AEs) compared with standard bone marrow transplant among these patients.
There is a lot of research [ongoing]; the holy grail of research is trying to figure out whether we can separate the graft-vs-leukemia or graft-vs-lymphoma effect from GVHD, said Abedi. [The Orca-T trial] is basically looking at the past 10 years of research in this area to try to identify the cells that are good from those that are bad.
During the 2021 Transplantation and Cellular Therapy (TCT) Meetings, findings from an analysis of 2 studies demonstrated a significant reduction in cases of GVHD, a higher GVHD relapse-free survival rate, and a lack of treatment-related mortalities with Orca-T when historically compared with hematopoietic stem cell transplant (HSCT).
Additionally, the median time to neutrophil engraftment, median time to platelet engraftment, and median time from day 0 to hospital discharge was shortened with Orca-T compared with HSCT.
In an interview with OncLive during the 2021 TCT Meetings, Abedi, a professor of cancer, hematology/oncology, and internal medicine in the Department of Internal Medicine, Division of Hematology and Oncology at the UC Davis Comprehensive Cancer Center, discussed the challenges of standard transplant, how Orca-T could overcome some of those limitations, and the potential future of transplant in hematologic malignancies.
Abedi: HSCT has been around for more than 50 years in one form or another. It has been used mostly for patients with blood cancers, [such as] leukemia and lymphoma. Allogeneic bone marrow transplants, where we use cells from a donor, are very effective. [They are associated with] a very high response rate for patients who have no other options and whose disease is going to [recur] without transplant.
The problem [with allogeneic bone marrow transplant] is that it [is associated with] AEs. When we give donor cells to patients after high-dose chemotherapy, [which is given] so that the patients body doesnt reject [the cells], even though the cells are a match for the patient, they can still [develop] severe GVHD.
The acute form of GVHD can be life threatening, whereas the chronic form can become a nuisance for the rest of a patients life. A lot of patients suffer [from GVHD] to the point where they regret going through transplant. Fortunately, that is not everybody, but it is still a problem that needs to be solved and an unmet need for the field.
This research has been focused on [the question of]: Are there specific cells in the graft we give to the patients immune cells that can cause GVHD? Can we separate those cells from those that are responsible for causing graft-vs-leukemia effects?
Basically, Orca Bio approached UC Davis a few years ago to [start] collaborative research with our Good Manufacturing Practice [GMP] facility and to produce these products.
Each graft of [the Orca-T] product has stem cells and immune cells in it. The stem cells are what we need to maintain the graft and [allow the product to] stay in the patient for a long time. The immune cells are the ones that can cause graft-vs-leukemia [effects], which is what we want.
From the work that Robert Negrin, [MD] at Stanford University and many other investigators [did], it is very clear that there is a population of T cells called T-regulatory cells that can prevent GVHD. There are other populations, such as the nave T cells or conventional T cells that can cause GVHD. It looks like a smaller number of [those cells] may actually be helpful; they can cause graft-vs-leukemia, but not GVHD.
The graft is basically designed so that we can give stem cells, but they get rid of a lot of conventional T cells that can cause rapid GVHD. [The graft provides] a small amount of conventional T cells that can cause graft-vs-leukemia effects, as well as the regulatory T cells that can prevent GVHD. UC Davis got involved [with this work] because we have a very robust GMP facility. We helped Orca Bio design these protocols and manufacture the cells. Now, [Orca Bio] has moved on to their own facility. I have been involved [with this research] for the past few years.
[We] havent looked at the QOL data, but there have been several short-term and long-term benefits so far that we have seen.
We give high-dose chemotherapy that can get rid of all [the patients] stem cells and leukemia or lymphoma cells. Then, we give the graft; however, after the graft is given, it takes a couple of weeks usually to engraft [before] new cells [develop]. In between, the patients are sick because of the effects of the chemotherapy; patients also have low blood counts.
[With Orca-T] we have seen that the engrafting [occurs] a little bit earlier. For everyday [sooner] the engraftment [occurs], there is less risk of complication and suffering for the patient. That has been a major difference [with Orca-T].
Also, we have noticed that patients in general are doing better [with Orca-T compared with traditional transplant]. When we give high-dose chemotherapy, it is the same whether the patient is on a clinical trial or not. They may still get sick [with Orca-T] because of the effects of chemotherapy.
However, there is also inflammation [that can occur] as the donor cells are trying to establish themselves in the body because some of them attack the body. That inflammation also adds to the problems with chemotherapy. We havent seen that inflammation [with Orca-T]. We have seen some effects from the chemotherapy, but because we dont have the inflammation, other AEs that we see with the standard of care arent seen with Orca-T.
In general, patients do better [with Orca-T vs standard of care], and that has been a universal experience with all of our patients who have gone through the trial so far.
[Patients] just look and feel better. When they are discharged, they are not as sick compared with patients [receiving] the standard of care.
With the standard of care, after we give the graft, we have to give patients a new medication to prevent GVHD because it is such a lethal problem. If we dont put patients on immunosuppressive medications to prevent GVHD, there is a very high chance that they get and die from GVHD. Those immunosuppressive medications are critical.
The way the Orca-T graft is designed is that we dont need as many of those [immunosuppressive] medications. For example, there is a medication called methotrexate that we give on days 1, 3, 6, and 11 after [standard] transplant. That medication can cause a lot of other AEs, such as mouth sores, delayed engraftment, and kidney [problems], that can make the patient miserable. With Orca-T, we dont have to [give methotrexate], which by itself is a huge improvement.
We do give immunosuppressive medications, such as tacrolimus [Envarsus XR] or sirolimus [Rapamune] to these patients, but we dont give 2 or 3 medications as we usually do with the standard of care. Less immunosuppressive medications mean probably less infection, but more importantly, less AEs. Thats [a factor that has made] a big difference in patient experience.
That is a very loaded question. There are a lot of new drugs coming, some of which are very targeted to treat leukemia or lymphoma. Thus far, we havent seen any curative [benefit] with those drugs, so in most situations, we will still need an allogeneic stem cell transplant for patients.
The exceptions [to that] were rare diseases, such as chronic myeloid leukemia [CML], where [an oral medication was approved] and we dont need to transplant patients. That is an example of a targeted treatment that may exclude [the need to] transplant patients. That would be great because transplant is not a trivial procedure and it has a lot of AEs.
However, [CML] is a very specific disease with 1 gene that causes the disease. In most leukemias and lymphomas, multiple genes are involved, so we dont think single-target treatments will get rid of the disease. We still think that allogeneic transplant will be around for a long time.
In fact, if you look at any center in the country, the volume of transplant has substantially increased over time because patients are living longer. Patients end up being able to go to a transplant vs before when many were dying in the middle of their treatment and not getting to transplant.
That being said, there are 2 directions [we can go in]. One is to try to decrease the AEs associated with transplant. This [Orca-T cell therapy] is very effective [in doing this] by targeting the graft-vs-leukemia effect and preventing GVHD. The second direction is to use these allogeneic cells to specifically target the tumor cells. That is why gene modifications, CAR T-cell therapies, and other approaches are coming. They still use allogeneic cells from a donor, but now they are directing them to specifically go after tumor cells.
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Orca-T Offers an Alternative to HSCT With Improved Patient Experience - OncLive
Timely Bone Marrow Transplant by Fortis gives new lease of life to a patient with Multiple Myeloma – APN News
By daniellenierenberg
Published on April 2, 2021
Recently, a 43-year-old man was presented at Fortis Hospital, Noida, complaining of severe back pain. Upon investigation, it was found that he was suffering from a rare disease, multiple myeloma which is a type of cancer that forms in the white blood cells or plasma cells. Here cancerous plasma cells accumulate in the bone marrow and crowd around the healthy blood cell that help in fighting infections by building antibodies, this puts the patients life at high risk. He therefore urgently required a Bone Marrow Transplant (BMT). Dr Rahul Bhargava, Director and Head, Hematology and Bone Marrow Transplant, Fortis Hospital, Noida and his team took a timely decision to go ahead with BMT to save his life.
A bone marrow transplant is a procedure to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Bone marrow is the soft, fatty tissue inside your bones. The bone marrow produces blood cells. Stem cells are immature cells in the bone marrow that give rise to different blood cells. Bone marrow transplant has now revolutionised. It is like a peripheral blood stem cell transplant, meaning, it is just like a blood transfusion (like platelet apheresis) which does not require any anesthesia.
Upon further investigation it was revealed that chemotherapy was required before BMT. Following the chemotherapy, on the 10thday of admission the team of doctors engulfed stem cells in the patients body which provided the body with a new source of healthy cells. Safe hospital environment and the doctors expertise in the area ensured that the BMT was done smoothly, without any complications and within 15 days the patient had been discharged. Usually, a patient takes 25-30 days to recover but here, due to patients will to recover and the facilities provided to him in the hospital he recovered at a faster pace.
Dr Rahul Bhargava, Director and Head, Hematology and Bone Marrow Transplant, Fortis Hospital, Noida, said,The case was complicated as the patient was suffering from high-risk multiple myeloma, which is a rare form of cancer. We took the necessary precautions and performed chemotherapy first to which he responded well and post that a Bone Marrow Transplant (BMT) was performed successfully. The process was smooth, and no complications arose during the same. We request patients to not fear BMT and undergo the process when required.
Talking about the clinical excellence at Fortis Hospital, NOIDA,Mr Hardeep Singh, Zonal Director, Fortis Hospital, Noidasaid, The team at Fortis Hospital, Noida try their best to save lives and do not give up even if there is 1% chance of survival. The patient was suffering from high-risk multiple myeloma for which immediate bone marrow transplant was required. The case was managed extremely well and with a lot of patience by Dr Rahul Bhargava and his team. I applaud the team of doctors for their continued commitment towards clinical expertise and patient care.
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Timely Bone Marrow Transplant by Fortis gives new lease of life to a patient with Multiple Myeloma - APN News
Do Therapies for Alzheimer’s & Parkinson’s that Clear Abnormal Brain Proteins Make the Diseases Worse? – BioSpace
By daniellenierenberg
One of the common research approaches to treating brain diseases such as Parkinsons disease (PD) and Alzheimers disease (AD) is using antibodies designed to clear the accumulated misfolded proteins implicated in the diseases. To date, these drug trials have not been particularly successful at improving the disease, even when they are effective at removing the proteins. A new theory hypothesizes that the antibodies are actually increasing the neuroinflammation associated with PD and AD, basically making the disease worse while removing the proteins.
Stuart Lipton, a researcher at Scripps Research Institute, recently published research suggesting that this may be exactly what is happening. They ran a series of in vitro assays and experiments in mice that had brain grafts of human-induced pluripotent stem cell (hiPSC)-derived microglia (hiMG). They found that the antibodies that target the misfolded proteins found in PD and AD trigger the NLRP3 inflammasome, which can lead to cell death.
Our findings provide a possible explanation for why antibody treatments have not yet succeeded against neurodegenerative diseases, said co-senior author Lipton, Step Family Foundation endowed chair in the department of molecular medicine and founding co-director of the Neurodegeneration New Medicines Center at Scripps Research.
The results were published in Proceedings of the National Academy of Sciences (PNAS).
The research started when Dorit Trudler, a postdoctorate researcher in Liptons lab, attempted to make microglia in the lab. Microglia are the innate immune cells in the brain, and it is a notoriously difficult task because the cells dont originate from the same type of stem cells in the bone marrow that generates the rest of the immune system.
Instead of coming from the bone marrow like B and T cells and macrophages, microglia are created from the yolk sac that embryos swim in during early development, then migrate from the sac to the brain. Trudler was able to turn human-derived stem cells to turn into a yolk sac-like structure, and from there, developed cells that were identical to microglia removed from humans based on the mRNA they expressed.
Lipton indicated, They match as closely as possible.
They then exposed these microglia to either alpha-synuclein, the misfolded protein identified in Parkinsons patients, and the microglia shot off inflammatory signals. And when they exposed the microglia to amyloid-beta, the hallmark of Alzheimers, the inflammation grew worse.
They then worked with biopharma companies to obtain antibodies that bind to either alpha-synuclein or amyloid-beta. Although Lipton is not saying which companies, it did say one of them was not Biogens aducanumab, which is up for approval or rejection for Alzheimers by June 7 by the U.S. Food and Drug Administration (FDA).
The surprising and potentially extremely important result was that although the antibodies successfully attached to their protein targets, it didnt help with inflammation. Rather than make things better, it actually made things worse, Lipton said.
And they further found in humanized mice with both human and mice microglia, that the pro-inflammatory response was unique to the human cells. This means that biopharma companies, in working with laboratory animals on therapeutic antibodies for these diseases, would not have seen this reaction. Although unclear yet why its causing inflammation, they are convinced the NLRP3 pathway is involved, although they dont have the exact mechanism worked out. Potentially, however, it might be possible to treat patients with a combination of the protein-clearing drugs and anti-inflammatories that block the NLRP3 pathway.
In an unrelated study, researchers at Massachusetts General Hospital utilized whole-genome sequencing (WGS) to identify rare genomic variants associated with AD. In doing so, they found 13 mutations, which establish new genetic links between AD and synaptic function. They believe it could help guide the development of new drugs for AD.
The first group of genes identified that were associated with AD involve the accumulation of amyloid-beta. The next 30 AD gene mutations identified were linked to chronic inflammation in the brain. Loss of synapses is the neurological change most closely linked with the severity of dementia in AD, but until now clear genetic association had not been identified.
Rudolph Tanzi, vice chair of Neurology and director of the hospitals Genetics and Aging Research Unit, said, It was always kind of surprising that whole-genome screens had not identified Alzheimers genes that are directly involved with synapses and neuroplasticity.
Tanzi went on to note that identifying less-common mutations that increase the risk for AD may provide critical information about the disease. Rare gene variants are the dark matter of the human genome, he said.
There are many of them, as it turns out. Of the three billion pairs of nucleotide bases in the human genome, about 5o to 60 million are gene variants and 77% are rare.
LGL Leukemia: Overview, Symptoms, and Treatment – Healthline
By daniellenierenberg
Large granular lymphocytic (LGL) leukemia is a kind of cancer that affects blood cells. The disease is rare: Only about 1,000 people per year are diagnosed with it. It affects men and women in roughly equal numbers, and most of those diagnosed are over 60 years old.
Heres what we know about this form of leukemia.
Your blood is made up of four different parts:
Some of your white blood cells are larger than the rest. These cells contain tiny granules that can be seen under a microscope.
In people with LGL leukemia, these large, granular white blood cells copy themselves until there are too many. The fact that the white blood cells (also called lymphocytes) replicate themselves is what makes this disorder a type of cancer.
Your blood contains two different types of lymphocytes: T-cells (T-LGL) and B-cells, which are also known as natural killer cells (NK-LGL). B-cells fight off invading bacteria and viruses. T-cells attack other cells in your body that have become harmful, like cancer cells.
When your T-cells are copying themselves too much, you have T-LGL leukemia. If your natural killer cells are replicating too much, you have NK-LGL leukemia.
Most cases of LGL leukemia are chronic and slow-growing, whether theyre NK-LGL or T-LGL. Only around 10 percent of all LGL cases are aggressive, fast-growing cells.
Researchers dont yet know what causes LGL leukemia. The disorder is associated with a genetic change or mutation, usually to the STAT3 and STAT5b genes.
Between 10 and 40 percent of people with LGL leukemia also have a history of autoimmune disorders. The immune disorder most often associated with LGL leukemia is rheumatoid arthritis (RA).
About 20 percent of those with LGL leukemia also have RA. So far, researchers have been unable to determine which disorder began first.
Most people who are diagnosed with LGL leukemia will experience some of these symptoms:
A healthcare professional may look for other symptoms, too, including:
You should contact your doctor and seek treatment if youre having recurring infections, especially if you have a fever that doesnt go away or you have other infection symptoms, such as swelling or sores, that arent getting better.
To find out if you have LGL leukemia, a healthcare professional will analyze a sample of your blood. Your doctor may also take a sample of your bone marrow, often from your hip area, to look for abnormal cells.
To determine which type of LGL leukemia you have, your doctor could use a laser technology called flow cytometry to identify whether T-cells or NK-cells are replicating too much.
Most cases of LGL leukemia are slow growing. Doctors sometimes take a wait-and-watch approach to treatment.
You may not start treatment until tests or symptoms show that the condition has reached a certain level.
If tests show that your neutrophil levels have dropped too much, your doctor may start treatment at that time. Around 45 percent of people with this condition needed immediate treatment.
When treatment for LGL leukemia begins, it may or may not follow the same intensive course as other cancer treatments.
Most people will eventually need some combination of chemotherapy and immune-suppressing drug therapy. Your medications could include:
In some cases, treatment for LGL leukemia involves a bone marrow or stem cell transplant. Its also possible that your treatment could include removing your spleen, an organ in your abdomen that filters your blood and helps maintain your immune system.
Two to three times a year, you may need to visit a healthcare professional to have bloodwork done to monitor your health and the activity of your white blood cells.
While theres no cure for LGL leukemia, most cases progress very slowly, unlike other forms of leukemia. One study that followed 1,150 people with the disease found that they lived an average of 9 years after their diagnosis.
The more aggressive form of LGL leukemia doesnt respond well to treatment. Life expectancy is likely much shorter for those with this very rare subtype of LGL leukemia.
LGL leukemia is a rare type of cancer where large white blood cells copy themselves too much, making your body prone to frequent infections.
Most cases of LGL leukemia are slow-growing, so treatment might not be necessary at first.
Eventually, people with this condition might need a combination of chemotherapy and immunosuppressing medications to slow the growth of cancer cells. Theres no cure yet for LGL leukemia.
A small percentage of cases are a faster-growing type of leukemia that doesnt respond well to treatments. Life expectancy for this subtype is shorter than the slow-growing type.
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LGL Leukemia: Overview, Symptoms, and Treatment - Healthline
BioRestorative Therapies Announces Notice of Allowance for a New Patent Application Related to its Off-the-Shelf ThermoStem Program – GlobeNewswire
By daniellenierenberg
MELVILLE, N.Y., March 31, 2021 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (the Company) (OTC: BRTX), a life sciences company focused on stem cell-based therapies, today announced that the United States Patent Office has issued a notice of allowance for a patent application related to the Companys metabolic ThermoStem Program. The notice of allowance was issued on March 22, 2021 and is related to a new patent application not previously announced.
Claims granted under the new patent cover methodologies related to generating exosomes and brown adipocytes from human brown adipose-derived stem cells. Exosomes are small extracellular vesicles produced by cells that contain lipids, messenger-RNA, micro-RNA, cytokines and proteins. Therapeutic benefits of using exosomes have been demonstrated in various disease models and may provide a valuable therapeutic tool for treating disease.
We are pleased to see that we have been granted an additional patent by the USPTO for our ThermoStem Program, said Lance Alstodt, the Companys CEO. Our comprehensive portfolio of patents under our ThermoStem Program continues to expand as we develop and protect intellectual property related to large and growing markets where brown adipocyte therapeutics can be applied. Im very proud of our team, driving towards the achievement of our stated goals. The advancement of our technology is a core, fundamental value driver of our Company. Our family of intellectual property coupled with our financial reporting progress are critical factors contributing to our growth strategy.
It is expected that the exosome diagnostic and therapeutic market will reach $368 million by 2022 as the development of research, clinical tools and therapeutics continues to grow in this emerging technology.
About BioRestorative Therapies, Inc.
BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:
Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.
Metabolic Program (ThermoStem): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.
CONTACT:Email: ir@biorestorative.com
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BioRestorative Therapies Announces Notice of Allowance for a New Patent Application Related to its Off-the-Shelf ThermoStem Program - GlobeNewswire
All About Multiple Myeloma, Type of Blood Cancer Kirron Kher is Suffering From – India.com
By daniellenierenberg
New Delhi: People woke up to terrible news of actor-politician Kirron Kher suffering from multiple myeloma, a type of blood cancer. She is currently undergoing treatment in Mumbai. Chandigarh BJP President and Kirrons colleague, Arun Sood revealed this at a press conference on Wednesday after which Anupam Kher also confirmed the same in a long note on Thursday morning. Also Read - Confirmed: Kirron Kher Diagnosed With Blood Cancer, Anupam Kher Calls Her a 'Fighter'
She had suffered a broken left arm at her Chandigarh house on November 11 last year. After her medical tests at Post Graduate Institute of Medical Education and Research (PGIMER) in Chandigarh, she was diagnosed with multiple myeloma. The disease had spread to her left arm and right shoulder. For treatment she had to go to Mumbai on December 4, Sood said at the press conference. Also Read - Anupam Kher And Kirron Kher Wish Each Other on 35th Wedding Anniversary With This Romantic Picture
Fans and well-wishers of Kirron are devastated by the news with many fans not being able to comprehend the shocking news. Here are a few things to know about the disease Multiple Myeloma that Kirron has been diagnosed with: Also Read - Kirron Kher-Anupam Kher's Mushiness in THESE Throwback Pictures on 34th Anniversary is All Hearts
It is a type of cancer that forms in a type of white blood cells known as a plasma cell. Your healthy plasma cells will fight infections and make antibodies that will attack germs, as per Mayoclinic. The cancerous plasma cells accumulate in the bone marrow. In this case, the cancer cells start producing an abnormal protein which further leads to complications. In simpler words, Multiple Myeloma happens when plasma cells become cancerous and grow out of control.
The symptoms can vary depending on an individual. Few reports suggest that initial symptoms may not be noticeable. But after a while, when it starts getting aggressive, as per Healthline, 4 major symptoms are noticeable which are referred to by the acronym CRAB which means:
Body gets a high level of calcium which can cause:
The cause is still unknown. It initiates with abnormal plasma cells which multiply in the bone marrow. Myeloma cells do not multiple and die, they divide indefinitely, as per Healthline.
Till now there is no cure for multiple myeloma. There are multiple treatments available including chemotherapy, interventional radiology, radiation therapy, stem cell transplantation, targeted therapy. Treatments are used when the disease starts getting worse.
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All About Multiple Myeloma, Type of Blood Cancer Kirron Kher is Suffering From - India.com
Heres what happens when a beam of subatomic particles hits you in the face – The Next Web
By daniellenierenberg
What would happen if you stuck your body inside a particle accelerator? The scenario seems like the start of a bad Marvel comic, but it happens to shed light on our intuitions about radiation, the vulnerability of the human body, and the very nature of matter.
Particle accelerators allow physicists to study subatomic particles by speeding them up in powerful magnetic fields and then tracing the interactions that result from collisions. By delving into the mysteries of the Universe, colliders have entered the Zeitgeist and tapped the wonders and fears of our age.
As far back as 2008, the Large Hadron Collider (LHC), operated by the European Organization for Nuclear Research (CERN), was charged with creating microscopic black holes that would allow physicists to detect extra dimensions. To many, this sounds like the plot of a disastrous science-fiction movie.
It came as no surprise when two people filed a lawsuit to stop the LHC from operating, lest it produce a black hole powerful enough to destroy the world. But physicists argued that the idea was absurd and the lawsuit was rejected.
Then, in 2012, the LHC detected the long-sought Higgs boson, a particle needed to explain how particles acquire mass. With that major accomplishment, the LHC entered popular culture; it was featured on the album cover of Super Collider (2013) by the heavy metal band Megadeth, and was a plot point in the US television series The Flash (2014-).
Yet, despite its accomplishments and glamour, the world of particle physics is so abstract that few understand its implications, meaning, or use. Unlike a NASA probe sent to Mars, CERNs research doesnt produce stunning, tangible images.
Instead, the study of particle physics is best described by chalkboard equations and squiggly lines called Feynman diagrams. Aage Bohr, the Nobel laureate whose father Niels invented the Bohr model of the atom, and his colleague Ole Ulfbeck have even gone as far as to deny the physical existence of subatomic particles as anything more than mathematical models.
Which returns us to our original question: what happens when a beam of subatomic particles traveling at nearly the speed of light meets the flesh of the human body? Perhaps because the realms of particle physics and biology are conceptually so far removed, its not only laypeople who lack the intuition to answer this question, but also some professional physicists.
In a 2010 YouTube interview with members of the physics and astronomy faculty at the University of Nottingham, several academic experts admitted that they had little idea what would happen if one were to stick a hand inside the proton beam at the LHC. Professor Michael Merrifield put it succinctly: Thats a good question. I dont know is the answer. Probably be very bad for you.
Professor Laurence Eaves was also cautious about drawing conclusions. [B]y the scales of energy we notice, it wouldnt be that noticeable, he said, likely with a bit of British understatement. Would I put my hand in the beam? Im not sure about that.
Such thought experiments can be useful tools for exploring situations that cant be studied in the laboratory. Occasionally, however, unfortunate accidents yield case studies: opportunities for researchers to study scenarios that cant be experimentally induced for ethical reasons. Case studies have a sample size of one and no control group.
But, as the neuroscientist V S Ramachandran has pointed out in Phantoms in the Brain (1998), it takes only one talking pig to prove that pigs can talk. On 13 September 1848, for example, an iron rod pierced through the head of the US railway worker Phineas Gage and profoundly changed his personality, offering early evidence of a biological basis for personality.
And on 13 July 1978, a Soviet scientist named Anatoli Bugorski stuck his head in a particle accelerator. On that fateful day, Bugorski was checking malfunctioning equipment on the U-70 synchrotron the largest particle accelerator in the Soviet Union when a safety mechanism failed and a beam of protons traveling at nearly the speed of light passed straight through his head, Phineas Gage-style.
Its possible that, at that point in history, no other human being had ever experienced a focused beam of radiation at such high energy. Although proton therapy a cancer treatment that uses proton beams to destroy tumors was pioneered before Bugorskis accident, the energy of these beams is generally not above 250 million electron volts (a unit of energy used for small particles). Bugorski might have experienced the full wrath of a beam with more than 300 times this much energy, 76 billion electron volts.
Proton radiation is a rare beast indeed. Protons from the solar wind and cosmic rays are stopped by Earths atmosphere, and proton radiation is so rare in radioactive decay that it was not observed until 1970. More familiar threats, such as ultraviolet photons and alpha particles, do not penetrate the body past skin unless a radioactive source is ingested.
Russian dissident Alexander Litvinenko, for instance, was killed by alpha particles that do not so much as penetrate paper when he unknowingly ingested radioactive polonium-210 delivered by an assassin. But when Apollo astronauts protected by spacesuits were exposed to cosmic rays containing protons and even more exotic forms of radiation, they reported flashes of visual light, a harbinger of what would welcome Bugorski on the fateful day of his accident.
According to an interview in Wired magazine in 1997, Bugorski immediately saw an intense flash of light but felt no pain. The young scientist was taken to a clinic in Moscow with half his face swollen, and doctors expected the worst.
Ionizing radiation particles such as protons wreak havoc on the body by breaking chemical bonds in DNA. This assault on a cells genetic programming can kill the cell, stop it from dividing or induce a cancerous mutation. Cells that divide quickly, such as stem cells in bone marrow, suffer the most. Because blood cells are produced in bone marrow, for instance, many cases of radiation poisoning result in infection and anemia from losses of white blood cells and red blood cells, respectively.
But unique to Bugorskis case, radiation was concentrated along a narrow beam through the head, rather than being broadly distributed from nuclear fallout, as was the case for many victims of the Chernobyl disaster or the bombing of Hiroshima.
For Bugorski, particularly vulnerable tissues, such as bone marrow and the gastrointestinal track, might have been largely spared. But where the beam shot through Bugorskis head, it deposited an obscene amount of radiation energy, hundreds of times greater than a lethal dose by some estimates.
And yet, Bugorski is still alive today. Half his face is paralyzed, giving one hemisphere of his head a strangely young appearance. He is reported to be deaf in one ear. He suffered at least six generalized tonic-clonic seizures. Commonly known as grand mal seizures, these are the seizures most frequently depicted in film and television, involving convulsions and loss of consciousness.
Bugorskis epilepsy is likely a result of brain tissue-scarring left by the proton beam. It has also left him with petit mal or absence seizures, far less dramatic staring spells during which consciousness is briefly interrupted. There are no reports that Bugorski has ever been diagnosed with cancer, though that is often a long-term consequence of radiation exposure.
Despite having nothing less than a particle accelerator beam pass through his brain, Bugorskis intellect remained intact, and he successfully completed his doctorate after the accident. Bugorski survived his accident. And as frightening and awesome as the inside of a particle accelerator might be, humanity has thus far survived the nuclear age.
This article by Joel Frohlich was originally published at Aeon and has been republished under Creative Commons.
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Heres what happens when a beam of subatomic particles hits you in the face - The Next Web