Archive for the ‘Bone Marrow Stem Cells’ Category

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

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

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 GP urges residents to save the life of a stranger - Crawley Observer

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

First-term GOP Reps. Marjorie Taylor GreeneMarjorie Taylor GreeneRep. Marjorie Taylor Greene says she's meeting with Trump 'soon' in Florida QAnon site shutters after reports identifying developer Republicans head to runoff in GA-14 MORE (Ga.) and Lauren BoebertLauren BoebertJuan Williams: The GOP is now the party of grifters and kooks The Memo: Boehner's blasts don't move today's GOP Overnight Energy: Progressives fear infrastructure's climate plans won't survive Senate | EPA to propose vehicle emissions standards by July's end | Poll shows growing partisan divide on climate change MORE (Colo.) were the only lawmakers to vote against a bill to reauthorize a bone marrow transplant program on Thursday evening.

The measure passed the House 415-2.It reauthorizes the C.W. Bill Young Cell Transplantation Program and National Cord Blood Inventory, which facilitate bone marrow and umbilical cord blood donations and transplants for people with leukemia or other blood diseases.

The bill also directs the Department of Health and Human Services to conduct a review of the state of the science on using adult stem cells and birthing tissues to develop new therapies that could potentially be included in the program.

The bipartisan measure was sponsored by Reps. Doris MatsuiDoris Okada MatsuiThe Hill's Morning Report - Presented by Tax March - CDC in limbo on J&J vax verdict; Rep. Brady retiring Hillicon Valley: Grid security funding not included in Biden's infrastructure plan | Russia fines Twitter | Lawmakers call for increased school cybersecurity Lawmakers urge Education Department to take action to defend schools from cyber threats MORE (D-Calif.), Gus Bilirakis (R-Fla.) and Chellie PingreeRochelle (Chellie) PingreeDemocrats condemn 'lawlessness' amid Capitol chaos Five House Democrats who could join Biden Cabinet Biden leads Trump by 11 points in Maine: survey MORE (D-Maine).

Greene and Boebert, bothvocal supporters of former President TrumpDonald TrumpBiden administration still seizing land near border despite plans to stop building wall: report Illinois House passes bill that would mandate Asian-American history lessons in schools Overnight Defense: Administration says 'low to moderate confidence' Russia behind Afghanistan troop bounties | 'Low to medium risk' of Russia invading Ukraine in next few weeks | Intelligence leaders face sharp questions during House worldwide threats he MORE, have quickly established reputations as controversial figures.

Greene wrote on Twitter that the bill did not have sufficient protections against the use of fetal tissue.

The Fake News Media is attacking me for being TOO PRO-LIFE (100%), she tweeted. Last night, Congress passed a bill which is not clear about preventing buying of body parts of babies murdered in the womb. I voted NO.

In a separate statement on Friday, Greene also said the bill was rushed.

The whole reason we are nearly $30 trillion in debt, have murdered over 62 million people in the womb, and have ZERO transparency on our spending is because Congress does not take the time to fully read and understand the bills it passes, she said.

"Im not voting for bills that dont go through committee and add hundreds of millions of dollars to the national debt," Boebert tweeted on Friday. The bill authorizes about $50 million per year for the transplant programs, though it will take an appropriations bill passing to actually spend that money.

Greene has been surrounded by controversy ever since coming to Congress. In February, she apologized to GOP colleagues for her past embrace of QAnon and other conspiracy theories, and the House voted to remove her from her committee assignments over previous endorsements ofracist dogma and violence against Democratic politicians.

Matsui released a statement Thursday praising the bill's passage and calling on the Senate to act.

Every three minutes, someone is diagnosed with a blood cancer, she said. For patients and families facing these fatal diseases, a bone marrow or cord blood transplant may be the best treatment or only potential for a cure.

Updated: 9 p.m.

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Greene, Boebert only lawmakers to vote against bone marrow transplant bill | TheHill - The Hill

Newswise A Phase II clinical trial to assess mesenchymal adult stem cells as a disease-modifying therapy for Parkinson's disease has been launched at The University of Texas Health Science Center at Houston (UTHealth).

"Studies have shown mesenchymal stem cells can migrate to the sites of injury and respond to the environment by secreting several anti-inflammatory and growth factor molecules that can restore tissue equilibrium and disrupt neuronal death," said Mya C. Schiess, MD, professor in the Department of Neurology and director and founder of the movement disorder subspeciality clinic and fellowship program at McGovern Medical School at UTHealth. "The stem cells interact directly with the immune cells, leading to an anti-inlammatory state that allows a restorative process to take place."

Safety and tolerability results, assessed on a previous trial, were recently published in the journal Movement Disorders. The Phase I study showed that there were no serious adverse reactions related to the stem cell influsion and no immunological reactions to the cells, which come from the bone marrow of a healthy adult donor. The study enrolled 20 patients with mild to moderate disease, who were infused with one of four different dosages and monitored for a year. Additionally, researchers reported a reduction in preripheral inflammatory markers and a reduction in motor symptoms.

Parkinson's diease is the second most common neurodegenerative disease, affecting more than a million Americans. It is also the fastest-growning of the neurodegenerative diseases, with more than 60,000 new cases identified every year. It is predicted that by 2040, Parkinson's disease will affect 17.5 million people worldwide.

Research has shown that one of the forces playing a critical role in the diease's development and progression is a chronic neuroinflammatory process that damages the brain's microenvironment and alters its healthy equilibrium. Inflammatiion perpetuates the neurodegenration in the brain areas that control movement, causing the tremors, imbalance, loss of speech, slowness, and other motor impairments.

The randomized, double-blind, placebo-controlled Phase II trial will investigate the safest and most effective number of repeat doses of stem cells to slow the progression of Parkinson's disease. The study will enroll 45 patients, ages 50 to 79, who will receive three infusions of either placebo or stem cell therapy at three-month intervals and will be followed for a year after the last infusion.

"Currently, there is no approved therapy that can delay the degenerative process in Parkinson's disease," Schiess said. "By investigating a treatment that can slow or stop the progression, we hope to improve the quality of life of those suffering from the disease. The ultimate goal is to use this treatment in individuals with a prodromal condition, meaning they are showing early signs of Parkinson's disease but are not yet clinically symptomatic. We hope to be able to potentially stop the diease's conversion or clinical manifestation in patients who are high-risk."

The Phase II trial, approved by the U.S. Food and Drug Administration, is supported with funding from the Michael J. Fox Foundation, John S. Dunn Foundation, and John and Kyle Kirksey.

Other McGovern Medical School faculty co-authors on the paper included Jessika Suescun, MD, Christopher Adams, MD, and Sean Savitz, MD, in the Department of Neurology. Marie-Francoise Doursout, PhD, Department of Anesthesiology; Charles Green, PhD, Department of Pediatrics; and Jerome G. Saltarrelli, PhD, Department of Surgery. Timothy M. Ellmore, PhD, Department of Psychology at the City College of New York, N.Y., was senior author.

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First in the nation, FDA-approved Phase II mesenchymal stem cell therapy for Parkinson's disease begins - Newswise

Newswise A clinical trial to assess the safety and efficacy of stem cell therapy for treatment-resistant bipolar depression launched recently at The University of Texas Health Science Center at Houston (UTHealth).

"Since mesenchymal stem cells are known to counteract inflammation and promote neurogenesis, we are hopeful that they provide an innovative therapy for patients with treatment-resistant biopolar depression," said Jair Soares, MD, PhD, chair of Louis A. Faillace, MD, Department of Psychiatry and Behavioral Sciences in McGovern Medical School at UTHealth. "Depending on the results, these stem cells could reduce morbidity and mortality associated with the disease."

Bipolar disorder is characterized by dramatic shifts in mood, energy, and activity levels that can affect a person's ability to carryout daily tasks, according to the National Institute of Mental Health. People with the disorder can swing from depression to mania. An estimated 2.8% of U.S. adults had bipolar disorder in 2016, and a large portion of them do not have a satisfactory response to available treatments.

This double-blind, randomized, placebo-controlled trial will use allogenic mesenchymal stem cells, which are multipotent stem cells taken from a bone marrow donor. The mesenchymal stem cells are manufactured in the Judith R. Hoffberger Cellular Theraputics Labratory at UTHealth, a state-of-the-art Food and DRug Administration-registered facility designed to comply with current Good Manufacturing Practice.

In a 2010 study published in Translational Research, scientists reported that stem cells showed efficacy in neurodegenerative illnesses that share several biological underpinnings of bipolar disorder, such as Parkinson's disease, with no adverse effects.

In previously published studies by researchers at UTHealth, stem cells have shown a dampening effect on inflammation, which has been linked to bipolar disease. Inflammatory markers have also been associated with a decreased likelihood of response to treatment in people with bipolar disease.

The trial will enroll 30 patients, who will recieve a single injection of either the stem cell product or placebo and continue to receive their usual care for bipolar depression for the eight weeks of the study.

UTHealth has been studying stem cells for traumatic brain injury and stroke for more than two decades.

Soares sees patients at UT Physicians, the clinical practice of McGovern Medical School.

McGovern Medical School co-investigators are Charles S. Cox Jr., MD; Fabio Triolo, PhD; Marsal Sanches, MD, PhD;Joo de Quevedo, MD, PhD; Sudhakar Selvaraj, MD, PhD; Antonio Teixeira Jr., MD, PhD; and Benson M. Irungu, PhD. Cox is a professor and George and Cynthia Mitchell Distinguished Chair in Neurosciences in the Department of Pediatric Surgery. Triolo is an associate professor and the Clare A. Glassell Distingued Chair in the Department of Pediatric Surgery. De Quevedo and Teixeira are professors; Sanches and Selvaraj are associate professors; and Irungu is an assistant professor in the Faillace Department of Psychiatry and Behavioral Sciences.

Soares, Cox, Triolo, and de Quevedo are also members of The University of Texas MD Anderson UTHealth Graduate School of Biomedical Sciences

More here:
Researchers investigate whether stem cell therapy is safe and effective for treatment-resistant bipolar disease - Newswise

Cell and gene therapies are overlapping fields of research and treatments. While both aim to treat and potentially cure diseases, they have slightly differing approaches and have different historical backgrounds. Due to growing interest surrounding this field, the general public still has much to learn and understand about each of these potentially life-saving therapies.

Below, we provide a general overview and brief historical context for each type of therapy.

Cell therapyis the process of replacing damaged or dysfunctional cells with new, healthy ones by transferring live cells into a patient. These can be autologous (also known as self-to-self, using cells from the patient receiving the treatment) or allogeneic (using cells from a donor for the treatment). While this field of treatment has recently begun to expand, some forms of cell therapy like the cancer-treating hematopoietic stem cell transplantation(HSCT) have been in practice for decades.

While many people have heard of bone marrow transplants, few realize that this procedure is a stem cell therapy. While stem cells can be derived from many sources, such as umbilical cord blood and mobilized peripheral blood, bone marrow derived stem cell therapy is the most commonly used today and has been for more than 50 years.

The first transfusion of human bone marrow was given to a patient with aplastic anemia in 1939. After World War II researchers diligently worked to restore bone marrow function in aplasia patients caused by exposure to radiation produced by the atomic bomb. After a decade of work they were able to show, in a mouse model, that aplasia could be overcome by bone marrow treatment.

The first allogeneic HSCT, which led the way to current protocols, was pioneered by E. Donnall Thomas and his team at the Fred Hutchinson Cancer Research Center and reported in the New England Journal of Medicine in 1957. In this study six patients were treated with radiation and chemotherapy and then received intravenous infusion of bone marrow rich stem cells from a normal donor to reestablish the damaged or defective cells. Since then the field has evolved and expanded worldwide. While almost half of HSCT are allogeneic, the majority of HSCT are autologous, the patient's own stem cells are used for treatment, which carries less risk to the patient.

In 1988, scientists discovered that they could derive stem cells from human embryos and grow the cells in a laboratory. These newly derived stem cells, referred to as embryonic stem cells (hESCs), were found to be pluripotent, meaning they can give rise to virtually any other type of cell in the body. This versatility allows hESCs cells to potentially regenerate or repair diseased tissue and organs. Two decades after they were discovered, treatments based on hESCs have been slow in coming because of controversy over their source and concerns that they could turn into tumours once implanted. Only recently, testing has begun as a treatment for two major diseases: heart failure and type 1 diabetes.

In 2006, researchers made a groundbreaking discovery by identifying conditions that would allow some cells to be 'reprogrammed' genetically. This new type of stem cell became known as induced pluripotent stem cells (iPSCs). Since this discovery, the field has expanded tremendously in the past two decades. Stem cell therapies have expanded in use and have been used to treat diseases such as type 1 diabetes, Parkinson's and even spinal cord injuries.

There has also been a growing focus on using other immune cells to treat cancer. Therapies such as CAR T-cellare dependent upon a patient's T-cells, which play a critical role in managing the immune response and killing cells affected by harmful pathogens. These cells are then reengineered to target and kill certain cancerous cells. Several CAR T-cell therapies have been FDA approved, with the first approval being given in 2017 for Yescarta and Kymriah, to be used for the treatment of B-cell leukemia in children and young adults.

Gene therapyis a process that modifies the expression of a gene or alters the biological process of living cells for therapeutic use. This process can take the form of replacing a disease-causing gene with a new, healthy one, inactivating the mutated gene, or introducing a new gene to help the patient's body fight a disease.

While the use of gene therapy to treat humans is fairly new, the science behind it has been used in science for decades. Farmers and geneticists have collaborated for years on crop improvement using cross pollination, genetic engineering and microinjection techniques to create stronger, more resilient crops.

The first human patient to be treated with gene therapy was a four-year old girlsuffering from severe combined immunodeficiencyin 1990. She received treatment for a congenital disease called adenosine deaminase (ADA). Since then, gene therapies have been used to treat diseases such as cancer, cystic fibrosis and hemophilia.In 2017, the FDA gave its first approval of a gene therapy called Luxturna, which is used to treat patients with established genetic vision loss that may result in blindness. Gene therapies are still being studied and developed, with over 1,000 clinical trialscurrently underway.

ThermoGenesis Holdings Inc., is a pioneer and market leader in the development and commercialization of automated cell processing technologies for the cell and gene therapy fields. We market a full suite of solutions for automated clinical biobanking, point-of-care applications and large-scale cell processing and manufacturing with a special emphasis on the emerging CAR-T immunotherapy market. We are committed to making the world a healthier place by creating innovative solutions for those in need.

For more information on the CAR-TXpress multi-system platform, please contact our Sales team.

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Thermogenesis Holdings Inc. published this content on 13 April 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 13 April 2021 07:10:03 UTC.

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Treatment options for chronic myeloid leukemia often include targeted therapies. Treatment plans and their effectiveness may depend on the phase of the condition.

Chronic myeloid leukemia (CML) is a slow-growing type of blood cancer that can affect white and red blood cells and platelets. It occurs in about 15% of adults who receive a leukemia diagnosis.

CML has three phases: chronic, accelerated, and blast.

The different phases can have an impact on a persons overall prognosis and how a doctor and the person approach the treatment plan.

This article discusses common treatments for CML and the differences between the phases of the condition.

The chronic phase is the earliest stage of CML.

In this phase, the cancer grows and spreads most slowly, and people typically experience few or no symptoms.

Moreover, during this stage of CML, people have less than 10% blast cells, which are cancerous immature white blood cells.

Most people receive a diagnosis of CML in the chronic phase.

During the chronic phase, the first line of treatment is tyrosine kinase inhibitors (TKIs). A doctor may try one or more TKIs, such as:

If a specific TKI is ineffective, a doctor may change a persons dose or use a different medication. On rare occasions, a doctor may recommend a bone marrow transplant.

During treatment, a doctor will need to check the progress regularly. To do this, they will need to draw blood and check for levels of BCR-ABL, a cancer-causing gene, every 36 months. A persons doctor should review the results of the tests with the person.

A 2017 long-term study found that the 10-year survival rate of people who received a diagnosis of chronic phase CML was about 83% when they took imatinib.

The American Cancer Society states that about 70% of people have a complete response to TKI treatments within the first year.

If the first treatment does not prove effective, a doctor may consider the following:

Treatment following a stem cell transplant can vary based on the response a persons body has to the transplant.

If the persons body does not reject the transplant, a doctor may try to have the immune system attack the cancer cells by either reducing the amount of immunosuppressors or introducing donor cells.

The second phase of CML is the accelerated phase, during which blast counts are higher, and symptoms are likely to develop.

In addition, during this stage, a person has increased cancer activity.

According to the American Cancer Society, a doctor will often diagnose the accelerated phase if one or more of the following occur:

A person with accelerated phase CML is also more likely to experience symptoms such as:

The American Cancer Society states treatment for the accelerated phase will be similar to that for the chronic phase. The main difference is that in the second phase of CML, long-term success with treatment is less likely.

Treatment options, which will depend on what doctors have already used, may include:

It is difficult to determine the life expectancy of a person who receives a diagnosis of CML in the accelerated phase.

The American Cancer Society indicates a person is less likely to have a long-term response to the treatment.

However, researchers are studying new therapies, which may help prolong the life expectancy of people with a diagnosis of accelerated CML.

The blast phase is the most advanced stage of CML.

People with a blast phase CML diagnosis have at least 20% blast cells in their blood. At this stage, the cancer has also spread beyond the blood into organs or other tissues.

Additionally, a person will likely experience fever, small appetite, and weight loss.

Treatment will vary between people depending on the cancer and the type of treatment a person has already undergone.

A cure for CML in the blast phase is unlikely. That is why doctors will possibly recommend medication and therapy to help a person feel better and relieve their symptoms.

According to the American Cancer Society, a doctor may recommend newer TKIs, such as bosutinib, dasatinib, or nilotinib. Chemotherapy drugs may be effective.

If treatment is successful, a doctor may recommend a stem cell transplant.

With newer therapies, the exact survival rate of people with a blast phase CML diagnosis is not clear.

People with blast phase CML are less likely to respond well to treatment and to recover from their condition than people with a chronic phase CML.

A 2018 study reports that people with CML whose cancer cells have the T315I mutation are less likely to respond to both older and newer TKIs.

As a result, doctors will likely recommend a different strategy, such as:

CML is a type of cancer. There are several potential therapies a doctor may recommend a person undergo to treat the cancer, slow its growth, or improve a persons quality of life.

Below, we describe some of the most common approaches.

Targeted therapies are medications that identify and attack cancer cells based on certain markers.

CML contains BCR-ABL, a gene that is not present in healthy cells. The gene causes the production of BCR-ABL protein, which is a type of tyrosine kinase. Targeted therapies for CML contain TKIs that stop the growth and reproduction of cancer cells with the protein.

According to the American Cancer Society, TKIs are a frequently used treatment option in the chronic phase of CML. However, doctors may also use them in later phases of the condition.

Interferon therapy is the most common treatment for CML.

It recreates interferons, a substance the immune system produces naturally. The therapy helps prevent the growth and division of cancer cells.

Chemotherapy, or chemo, which doctors use to treat many different types of cancer, slows or stops the growth and division of cancer cells.

It may cure the cancer, reduce the likelihood of it returning, or slow or stop its growth. It may also improve symptoms.

Chemotherapy used to be the primary treatment for CML. However, TKIs are now the first line of treatment.

Doctors will typically only recommend chemotherapy if a person does not respond well to TKIs or is undergoing a stem cell transplant.

Radiation therapy uses high doses of waves of energy to destroy cancer cells. The damaged cancer cells can no longer reproduce, and die as a result.

The National Cancer Institute states that it can take several weeks of treatment to damage cancer cells enough for them to start dying off. It could then take a few weeks or months for the cells to die off completely.

However, according to the American Cancer Society, radiation is not a common treatment for CML.

Doctors may use it to reduce the size of the spleen if the cancer has spread there, to treat bone pain resulting from bone damage. They may also use it during stem cell transplant throughout the body.

Surgery is not a typical treatment option for CML. That is because the cancer can spread throughout a persons bone marrow and other organs.

Doctors will typically only recommend surgery to remove the spleen if the cancer has affected it.

A stem cell transplant involves destroying cancer cells and some healthy cells in the bone marrow, where the leukemia starts.

Once the cancer is destroyed, a doctor replaces the cells with healthy bone marrow cells that a donor provided. Usually, doctors offer this treatment option to younger people who have a matched tissue donor.

While this is the only treatment that can cure CML, it has several associated risks, including infection and graft-versus-host disease.

A person with a diagnosed CML may wish to try alternative or complementary therapies to help alleviate symptoms. They should seek guidance from a doctor to find the most suitable therapies.

According to a 2016 study, traditional Chinese herbal medicine may be effective in managing CML when people use it in conjunction with Gleevec.

However, a person should speak with their doctor about this type of treatment before finding a licensed practitioner of traditional Chinese medicine.

Another study looked at several different herbs and fruits for the treatment of leukemia. Although the study indicates more research is necessary, it reports positive results when using herbs such as ginger, garlic, and carrots.

CML is a slow-growing type of leukemia that develops in the bone marrow.

Experts distinguish three phases of the condition: chronic, accelerated, and blast. Treatments across the three phases are often similar and involve using TKIs.

A person can work with their doctor to create the best treatment options for them. If the treatment is ineffective, a doctor may recommend other therapies to achieve remission or improve a persons quality of life.

View post:
Treating chronic myeloid leukemia (CML): By phase and more - Medical News Today

LCol Laura Laycock on deployment.

LCol Laura Laycock

It was Oct. 7, 2019, and life was not just good, it was amazing.

My career in the Royal Canadian Air Force was going great. I loved my job and was getting promoted. Throughout my Canadian Armed Forces career of over 20years, I had represented Canada around the world with NORAD, NATO and the UN. I had married the most incredible man. We relocated to Ottawa, started to travel the world together, and were ready to start a family.

Then, on Oct. 8, 2019, everything changed.

I was diagnosed with Chronic Myeloid Leukemia(CML) after blood work for vertigo showed extremely elevated white blood cell counts. CML is a blood cancer where the bone marrow overproduces white blood cells, which eventually impairs the development of white and red blood cells and platelets. Its usually caused by a spontaneous mutation in DNA, which contains our genetic code.

LCol Laycock

Twenty years ago, researchers developed a new line of drugs that combat this overproduction of white blood cells. These targeted oral chemotherapy pills have been revolutionary in the fight against CML. Most people who take them do so for the rest of their lives and have good survival rates; however, a stem cell transplant remains the only actual cure. But its risky and not needed for most people.

Its now been about 17months since my diagnosis and my body has not tolerated this targeted chemotherapy. I fall into that small fraction of people who get debilitating or life-threatening side effects from this medication. My doctors are discussing other treatment options, one of which is a stem cell transplant, but my mixed ethnicity (European/Middle Eastern) has made it difficult to find a donor match.

My journey since my diagnosis has been to slow down and educate myself so that I can heal and advocate for my care; to appreciate every little moment of joy; and to do my best to overcome each challenge that arises. I have found strength in the extraordinary support Ive received from my family, my friends and my community, both old and new.

With the help of family and friends, I recently began a social media campaign to increase stem cell donor education and registration in Canada and around the world. Many people are unaware of the potentially lifesaving role they can play by registering to become stem cell donors. Stem cell transplants are vital treatment options for people with a range of medical conditions including spinal cord injuries, heart disease, diabetes, and some cancers.

The process to donate is simple. First, you register online with Canadian Blood Services or Hma-Qubec and do a mail-in cheek swab., and then you wait. It could be months or years before you are identified as a match. During this waiting period, you should update your contact information with the registry if it changes.

When you are matched, you will be contacted to continue with the donation process. This process is similar to giving blood, but it has its differences. The cells are usually collected intravenously from peripheral blood in a non-surgical procedure but, in rare cases, they are collected directly from the bone marrow in a surgical procedure. In either case, the risks associated with donating are minor.

In Canada, individuals aged17 to 35 can register to become stem cell donors (ages18 to 35 in Quebec). Both CBS and Hma-Qubec are part of an international network of donor registries from over 50countries. This network has a pool of over 38million donors but, unfortunately, matches are rare.

Your stem cells could potentially help others around the world, and throughout this process donor privacy is assured at all times.

LCol Laycock on her wedding day.

Stem cell matching relies on Human Leukocyte Antigen typing, which is highly influenced by ethnicity. This means that a patients best chance of finding a matching donor is from those who share similar ethnic backgrounds. Research conducted by Gragert et al.(2014) has shown that the likelihood of finding a match for certain ethnic groups can be as low as 16 percent and as high as 75 percent for others. This disparity highlights the need for more ethnically diverse stem cell donors in our registries.

Today, I am calling on my DND and CAF families to register as stem cell donors to help people, like me, who are fighting for our lives. If you arent able to register, please share this call with those who can. You, or someone you know, could be the match that saves a life a simple swab is all it takes to be a hero.

Read this article:
Stem cell treatment needed to fight the good fight - Victoria Lookout

Its important to be able to control the timing and spacing of the gene knockouts so the researchers can look at the same mice over time, and test for different situations.

VHL helps control cells response to different oxygen levels, especially low levels. Our current data indicates that VHL gene deletion accelerates the production of red blood cells and helps create blood vessels in the bone and bone marrow, Spencer said. This, in turn, impacts how B-cells develop.

The researchers will be able to peer inside the bone marrow and see how the microenvironment has changed when VHL is deleted in the mesenchymal cells and pre-osteoblasts. Several diseases are linked to the marrow microenvironment, so the research has the potential to inform the work of many other research teams as could the development of the new mouse model. Manilay said many researchers have tried to build models with the same specifications she and Spencer need, but those models have not worked.

Were trying something different, so this could really help researchers all over the world if it works, she said. This is high-risk, high-reward research. The risk is this could completely fail, but even then, wed be able to provide information to other researchers.

This $400,000, two-year R21 grant builds on an NIH R15 award the team received last year to examine the effects of the VHL gene defect that makes the bones grow extremely dense, with little room for marrow. Because marrow is where both immune system and blood cells develop, less marrow than normal could have a wide range of negative consequences, and the team wanted to know whether immune cells were responding to altered oxygen conditions, which are also a hallmark of the defect. B-cells can sense when they are in low-oxygen conditions, Spencer said, and they can turn on genes to help them adapt.

Spencer, whose research focuses on biomedical imaging, became the first scientist to capture an image of native adult hematopoietic stem cells (HSC) within the bone marrow of a living organism. Manilay is interested in the relationship between bone and bone marrows HSCs on immune cells fates.

Spencer, with the Department of Bioengineering in the School of Engineering, and Manilay, with the Department of Molecular and Cell Biology in the School of Natural Sciences, are both members of the Health Sciences Research Institute.They worked on the foundation of this research for more than a year before the first grant started. Manilays lab had gathered preliminary data, including some work done by graduate student Betsabel Chicana, who recently won an NIH fellowship for her immunology work. The graduate students collaborated throughout, analyzing data and cross-training in each others disciplines.

Now that weve been doing this for more than a year, Im really seeing the benefits among my students, Spencer said. Exposing them to other research methods and other ways to ask questions and design experiments gives them a broader training, which will benefit their futures as researchers.

View post:
Collaboration Furthers Understanding of Immune Cell Development | Newsroom - UC Merced University News

Leukemia cutis can happen when leukemia cells enter your skin. This rare condition causes patches of discolored skin to appear on the body.

In some cases, the appearance of leukemia cutis lesions on the skin is the first sign of leukemia a cancer of the blood and bone marrow.

Along with standard leukemia therapies, this complication can usually be addressed with topical treatments to help heal the damaged skin. If you have leukemia cutis, your outlook will usually depend on your age and the type of leukemia you have.

Leukemia cutis is an uncommon complication, affecting only about 3 percent of people with leukemia. However, it is often a sign that the cancer is at an advanced stage.

With leukemia, malignant leukocytes (white blood cells) are usually only present in the bloodstream. In the case of leukemia cutis, the leukocytes have entered the skin tissue, causing lesions to appear on the outer layer of your skin. The word cutis refers to the skin, or dermis.

Generally, leukemia cutis results in one or more lesions or patches forming on the outer layer of skin. This condition can mean that the leukemia is more advanced and may have spread to your bone marrow and other organs.

Because there are fewer healthy white cells to combat infections caused by other diseases, rashes and sores may be more common among people with leukemia. Low blood platelets from leukemia can cause damage to blood vessels that appear as red spots or lesions on the skin.

These may include:

However, these skin changes are different than those brought on by leukemia cutis.

While the legs are the most common area for leukemia cutis lesions to appear, they can also form on the arms, face, trunk, and scalp. These skin changes can include:

The lesions usually dont hurt. However, with certain types of leukemia particularly acute myeloid leukemia (AML) the lesions may bleed.

A dermatologist may initially diagnose leukemia cutis based on a physical examination of the skin and a review of your medical history. A skin biopsy is needed to confirm the diagnosis.

Leukemia cutis is a sign of leukemia. It wont develop if the body isnt already dealing with this type of blood cancer.

But leukemia isnt just one disease. There are multiple types of leukemia, each one classified by the kind of cell affected by the disease.

You can also have an acute or a chronic form of leukemia. Acute means it comes on suddenly and usually with more severe symptoms. Chronic leukemia develops more slowly and often with milder symptoms.

The types of leukemia that most commonly trigger leukemia cutis are AML and chronic lymphocytic leukemia (CLL).

Scientists arent sure why cancerous leukocytes migrate to skin tissue in some people with leukemia. It may be that the skin is an optimal environment for healthy leukocytes to transform into cancerous cells.

One possible risk factor that has emerged is an abnormality in chromosome 8, which has been found more often in individuals with leukemia cutis than in those without it.

Treating leukemia cutis usually includes treatment for leukemia as the underlying condition.

The standard leukemia treatment is chemotherapy, but other options may be considered depending on your overall health, your age, and the type of leukemia you have.

Other leukemia treatment options include:

For blood cancers, external beam radiation is a typical form of treatment. With this therapy, a focused beam of radiation is delivered outside the body from various angles. The goal is to injure the DNA in cancer cells to stop them from reproducing.

Immunotherapy, a type of biological therapy, uses the bodys own immune system to fight cancer. It is typically given by an injection that either stimulates immune system cells activity or blocks the signals cancer cells send to suppress the immune response.

Immunotherapy may also be given orally, topically, or intravesically (into the bladder).

Stem cell transplantation is more commonly known as a bone marrow transplant. Bone marrow is where blood stem cells develop. Stem cells can become any type of cell.

Through stem cell transplantation, healthy blood stem cells replace stem cells damaged by the cancer or by chemotherapy or radiation therapy. However, not everyone is a good candidate for this treatment.

Only treating the leukemia cutis lesions will not address the underlying disease of leukemia. That means treatments designed to remove or reduce lesions should be done in combination with systemic treatment for leukemia itself.

Treatments for leukemia cutis symptoms can include:

Again, these treatments will only treat the leukemia cutis lesions, but systemic treatment of the leukemia itself will be needed as well.

The length of time leukemia cutis lesions may last depends on many factors, including how well the leukemia itself is responding to treatment. If the leukemia goes into remission, its unlikely more lesions will appear.

With effective treatment, existing lesions could fade. However, other factors, including your age and overall health, can affect how widespread the lesions are and how long they may last.

There are encouraging trends in the treatment of leukemia, but it remains a challenging disease to treat and live with.

For people with AML who dont have leukemia cutis, research suggests that the survival rate at 2 years is about 30 percent. However, the survival rate drops to 6 percent among people with the skin lesions.

A separate study of 1,683 people with AML found that leukemia cutis was associated with a poor prognosis, and that those with AML and leukemia cutis may benefit from more aggressive treatment.

The outlook for people with CLL is better, with about an 83 percent survival rate at 5 years. The presence of leukemia cutis doesnt seem to change that outlook very much, according to a 2019 study.

Leukemia cutis is a rare complication of leukemia. It happens when malignant leukocytes invade the skin and cause lesions on the skins outer surface.

AML and CLL are more often associated with leukemia cutis than other types of leukemia.

While leukemia cutis usually means the leukemia is in an advanced stage, there are treatments for both the cancer and this uncommon side effect that may help extend life and improve its quality.

Go here to see the original:
Leukemia Cutis: Symptoms and Treatment - Healthline

A lack of ethnic diversity among donors, coupled with cultural myths, have led to a paucity of bone marrow donors, resulting in hundreds of avoidable deaths caused by leukaemia and other blood-related diseases.

According to Jane Ward, the deputy director of the South African Bone Marrow Registry (SABMR), the lack of sufficient education to counter cultural beliefs, especially among black South Africans, contributes to donor resistance. A common myth is that regenerative stem cells are somehow body parts and cannot be parted with.

She said a major education and destigmatisation campaign is needed to help people donate and to realise that help is available.

Also, by the time many black patients go to a doctor or clinic to be referred upwards for treatment, its often too late. We can only transplant bone marrow when a patient is in remission, and that requires extensive treatment.

If you dont catch it quickly enough, you cant treat it. That and the lack of facilities, plus the costs which we cover for matching, donor collection and stem cell transport have proved the biggest hurdles, Ward said.

Youre more likely to find a match within your own ethnic group and its been shown that there are more white donors on registries worldwide. Theres simply not enough ethnic diversity among donors.

Two other societal phenomena have contributed to shrinking the local bone marrow registry base from 74000 to 73000 donors over the past five years: emigration and ageing.

Last year, of those donors removed from the local donor base, more than 6% were lost because of emigration, Ward revealed.

And because stem cells age with their hosts, donors are retired from the database at 60 years old.

Emigration is not a train smash because we contact them and transfer them to the registry of the destination country but that ups the cost in getting the donor cells transported back here, she explained.

A new partnership between the SABMR and Netcells, South Africas largest private cord blood bank, is set to improve access to more cost-effective, life-saving treatment for leukaemia and some 79 other blood related diseases.

While 550 patients have received matched unrelated transplants since the SABMR was established in 1991 an outflow of critical tissue matching capability required for Dr Chris Barnards historic 1967 heart transplant the chances of finding a bone marrow match remain slim.

This new partnership includes the establishment of a public community cord blood stem cell bank Africas first and marks another milestone in reducing the one-in-100000 odds of finding a bone marrow match for patients in South Africa.

While South Africa has been part of the world registry for bone marrow transplants for 20 years, giving it access to some 39-million registered donors, there are only 73000 South African donors in an ageing local registry.

This puts South Africans at a major cost and access disadvantage. One of the biggest stumbling blocks remains the few black (global and local) donors and the gap between South Africas funded private transplant funding and unfunded state sector transplants which the community cord blood stem cell bank aims to address.

There are other reasons why donor recruitment remains so low.

The SABMR only began recruiting donors in 2018, meaning it had to develop a recruitment division from scratch after the well-known Sunflower Fund relinquished this task as part of its cancer treatment funding efforts.

Ward said that recent advances in medical technology have brought umbilical cord blood stem cells to the table as an effective treatment. Besides infant cord blood stems cells, blood from young healthy males aged 18 to 25 is the preferred transplant choice. Although that age bracket is the preferred choice, any one who is healthy and younger than 60 can donate.

According to Ward, a consequence of this reality is that South Africa recently became only the second country globally to drop the donor recruitment age from 18 to 16 in its attempt to swell the registry.

Education is vital to counter myths and create a younger, informed donor base. We want everybody in the next generation to know about this life-saving treatment and how they can help.

One way to achieve this is to use new channels such as TikTok and Instagram for the younger generation being targeted.

Shelley Bredin, the managing director of Netcells, outlined how her company works and will partner with the SABMR.

Netcells receives umbilical cord blood stem cells from infants at birth, freezing them for future use. The parents opting to do this do so as an insurance against future blood-based diseases.

The umbilical stem cells are also a 25% match for a sibling, widening the insurance safeguard. Parents can, at the outset, choose whether to opt for community stem cell banking or private stem cell banking, the former costing half the price (R15000). This is in return for the stem cells being allocated on a priority needs basis to either their family or an outside matched recipient on a first-come-first-served basis via the SABMR registry.

If the community banked stem cells are used for an outside recipient, the parents are reimbursed fully. Once the initial selected storage period ends, when privately banking the stem cells, Netcells contacts the parents and asks them if they want to continue storage or to donate them to the SABMR as a philanthropic gesture.

If parents opt for private stem cell banking, the frozen cells are kept exclusively for their use, but the up-front price increases to R29000 for 10 years storage.

Its a very personal decision, she said. Our intention is to make the stem cells more attainable for more people and at the same time serving to expand the donor pool in South Africa. Some who have the money would still prefer to bank privately as they dont want to take the gamble.

Cost remains an ever-present reality, with Covid-19 dramatically reducing the global pool of available and usable donor blood cells.

Its no longer just an issue of an adult donor having to be medically fit. The question becomes, will the donor get Covid? You have to test [the donor], for that. Yet [umbilical] cord blood remains readily available, and you dont need a whole bunch of tests on the donor, Bredin said

This was yet another current contextual advantage of the new partnership, she emphasised.

Bredin said cord blood is a much simpler source of stem cells than an adults blood. By establishing a community cord blood bank, they hope to reduce the need for expensive global sourcing.

An adult blood cell donation can cost between R250000 and R270000 from a foreign country (just over half that locally) while foreign-sourced umbilical cord blood can cost up to R1-million. Only a very few local medical aids fund all sourced donations and transplants.

Leukaemia is the most common cancer among children and teens worldwide, accounting for 30% of all cancers diagnosed in children.

The miraculous, life-saving bone marrow transplant addressing end-stage haematological disorders happens just 12500 times a year globally, according to the SABMR.

Anyone wanting to find out more about stem cell harvesting and donation or who would like to contribute to the SABMR donor fund to help save lives should visitnextbio.co.za/netcells/ or sabmr.co.za/become-a-donor/. Alternatively they can email: [emailprotected]

Here is the original post:
More ethnically diverse bone marrow donors needed to save lives - Mail and Guardian

TEHRAN For the first time, MAHAK Charity Foundation has started using the Total Body Irradiation (TBI) cancer treatment method for children in the country.

Launched by the MAHAK Pediatric Cancer Treatment and Research Center (MPCTRC), TBI delivers radiation to the whole body from head to toe. It destroys remaining malignant cells, creates space in bone marrow for donors bone marrow stem cells, and prevents rejection by suppressing the patients immune system. TBI increases the survival rate in children with high-risk leukemia.

It is a part of the preparation procedure for hematopoietic (or bone marrow) stem cell transplantation. In this method, the immune system of the individual who needs transplantation gets suppressed allowing the recipient to accept foreign bone marrow stem cells.

Moreover, it can eradicate the remaining cancer cells and thereby increase survival rates of high-risk leukemia patients.

TBI is a method that has been used across the world for decades and is now mostly reserved for high-risk leukemia in need of transplantation. Using this type of irradiation in Iran would increase the survival rate of children with high-risk leukemia.

MAHAK is an NGO that supports services for cancer-stricken children and their families in accordance with international standards, as a result, over 35000 children with cancer have benefited from it during the past 29 years.

FB/MG

Original post:
New pediatric cancer treatment method being used in Iran - Tehran Times

Adipose-derived stem cells are mesenchymal stem cells obtained from adult fat tissues during various surgical interventions such as breast reduction, liposuction, or abdominoplasty. Adipose-derived stem cells (ADSCs) possess the ability to proliferate into numerous cell lineages such as chondrocytes, adipocytes, and others. Owing to this property, these multipotent stem cells can substitute bone marrow as a rich source of stem cells.

The report performs an insightful analysis of various fundamental market aspects, including SWOT analysis, CAGR during the projected period, recent developments, new product launches, technology upgradation, product offerings, application landscape, end-users, and geographical footprint of the leading companies. The market estimations carried out by our team of industry experts are based on various research methodologies and validated information on the present market scenario.

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Global Adipose-derived Stem Cell Market Research Scope:

The global market can be broadly segmented on the basis of product type, application spectrum, competitive landscape, geography, and end-use industries. Each of the market segments has been elaborately represented in the table of contents (ToC) included in the report, as well as in the format of graphs, tables, charts, etc. The report, additionally, expounds on the intensely competitive terrain of the global Adipose-derived Stem Cell market, taking into account some major factors like strategic business growth initiatives, product development, key market players, revenue share, and a wide range of research &development activities.

Some of the prominent players of the industry include Allocure, Inc., Celgene Corporation, Pluristem Therapeutics, Inc., Intrexon, Inc., Celleris SA, Tissue Genesis, Inc., Mesoblast Ltd., Cytori Therapeutics, Antria, Inc., ThermoFisher Scientific, American CrysoStem, Merck KGaA, Others

Market Segmentation:

The market is broadly categorized on the basis of product types offered in the market, region, broad application spectrum, and the leading manufacturers/companies.

Cell Type

Product Type

Disease Indication

End-user Industries

Application

Regional Segmentation:

The latest Adipose-derived Stem Cell market report highlights the ongoing market demands and trends, more importantly, in the market that is spread across the major regions of the country. The report entails an insightful analysis of the current growth opportunities for various regions of the Adipose-derived Stem Cell market. It further mentions the year-on-year growth rate of these regions over the forecast duration. The leading regional segments encompassed in the report include:

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Some Fundamental Market Parameters Elucidated in the Report:

Market dynamics: The Adipose-derived Stem Cell market report explains the scope of various commercial possibilities over the next few years and further estimates revenue build-up over the forecast years. It analyzes the key market segments and sub-segments and provides deep insights into the market to assist readers in developing vital strategies for profitable business expansion.

Competitive Outlook: The established market players operating in the Adipose-derived Stem Cell industry have been listed in this report, with a major focus on their geographical reach and production facilities. To gain a competitive advantage over the other players in the Adipose-derived Stem Cell industry, the leading players are focusing more on offering products at rational prices.

Objectives of the Report: The chief aim of the research report is to provide the manufacturers, distributors, suppliers, and buyers engaged in this sector with access to a deeper and improved understanding of the global Adipose-derived Stem Cell market.

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Read the original post:
Adipose-derived Stem Cell OverviewMarket Key Companies, Business Opportunities, Competitive Landscape and Industry Analysis Research Report by 2027 ...

12 April 2021

A clinical trial for a new gene therapy approach to treat sickle cell disease has been approved to proceed by the US Food and Drug Administration.

Patients with sickle cell disease have a mutation in the beta-haemoglobin gene, causing them to produce misshapen red blood cells that can block blood vessels leading to severe pain, anaemia and potentially life-threatening complications, such as organ damage and strokes.Currently, the only cureis a stem cell transplant from a healthy donor, but in the newly-approved trial, scientists from the University of California will use CRISPR/Cas9 genome editing to replace the faulty gene with a functional version.

'Gene therapy and genome editing allow each patient to serve as their own stem cell donor,' said Professor Donald Kohn, from the Broad Stem Cell Research Centre at the University of California Los Angeles, one of the clinical trial leaders. 'In theory, these approaches should be much safer than a transplant from another person and could become universally available because they eliminate the need to find the needle in a haystack that is a matched stem cell donor.'

In the trial, blood stem cells will be harvested from the patients and grown in the lab. CRISPR/Cas9 will be used to 'cut and replace' a sequence of DNA containing the mutation with a healthy copy. The edited cells will then be returned to the patient's body in the same way they would be if the patient was receiving donor stem cells.

'The goal of this form of genome editing therapy is to correct the mutation in enough stem cells so the resulting blood in circulation has corrected red blood cells,' said Dr Mark Walters, from the University of California San Francisco Benioff Children's Hospital, another of the clinical trial leaders.

The study will take place over four years, and include six adults and three adolescents with severe sickle cell disease, testing both safety and efficacy.

The treatment does have risks: the patientswill need to have high dose chemotherapy, to kill allremaining bloodstem cells before the modified stem cells are put back. This is also necessary before receiving donor stem cells and can cause severe side effects as the patient's immune system is temporarily disabled.

A similar trial, using CRISPR/Cas9 to activate bone marrow stem cells to produce an alternative version of haemoglobin, rather than correcting the faulty version, has recently shown promising results in a patient with sickle cell disease (see BioNews 1052).

Here is the original post:
CRISPR gene therapy for sickle cell disease approved by the FDA - BioNews

Apr 15th 2021

THE ANCIENT GREEKS were good at inventing fantastical animals. The chimera, for instance, was a thing of immortal make, not human, lion-fronted and snake behind, a goat in the middle. It was eventually slain by Bellerophon, with help from his flying horse.

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Not all chimeras are mythological. To biologists, the term describes organisms whose bodies consist of cells from two distinct lineages. In twin pregnancies, for example, one twin can occasionally absorb the other. The resulting individual is built from cells with separate genomes. A 2019 forensic-science conference discussed the case of a man who had received a bone-marrow transplant. Since bone marrow produces blood cells, subsequent DNA tests on the mans blood matched his donors genome, not his own. (More unexpectedly, the donors DNA also turned out to be present in swabs taken from the mans cheeks, and in his semen.)

For several decades scientists have been experimenting with cross-species chimeras, organisms which, as in the Greek myths, are composites of different animals. They have created mouse-rats, sheep-goats and chicken-quails. Now, in a paper published in Cell, Tao Tan, a biologist at Kunming University of Science and Technology, and a team of American, Chinese and Spanish researchers, report efforts to extend the principle to humans. They have managed to create embryos that are part-monkey and part-human.

The work builds on earlier endeavours by many of the same researchers. In 2017 Juan Carlos Izpisa Belmonte, a biologist at the Salk Institute in San Diego, announced the creation of chimeric human-pig embryos. But quite how successful those efforts were is uncertain. Only about one cell in 100,000 in the embryos were human, and it was unclear whether they contributed to the organisms growth. This time things are different. The human cells seem happy to co-operate, at least some of the time, with the monkey ones.

The researchers began with 132 embryos of the crab-eating macaque. Six days after fertilisation these were injected with human extended pluripotent stem cells, which can develop into any other cell type found in the body. Tagging the human cells with fluorescent markers allowed the researchers to track where in the developing embryo they, and their descendants, went.

In the early stages of development, mammal embryos develop into four distinct cell types. Epiblasts go on to form the organism itself; hypoblasts develop into the yolk sac; trophectoderms become the placenta and extra-embryonic mesenchyme cells make a membrane that surrounds the embryo. The chimeras human cells made their way into all four types of tissue, though they were outnumbered in every case. No more than 7% of the epiblast was made up of human cells, and just 5% of the hypoblast (in other areas the numbers were lower still).

The cells location seemed to influence which proteins they produced. Human cells in the chimeras epiblast behaved more like those found in human embryos than those found in monkey embryos. But that was not true of human hypoblast or extra-embryonic mesenchyme cells, both of which behaved more like monkey cells.

The monkey cells, in turn, were affected by the presence of the human ones. The researchers found 126 different sorts of cell-to-cell interactions among monkey cells in the chimeric embryos, compared with just 19 in non-chimeric ones, as well as differences in the activity levels of many genes.

The cells were grown in a lab, which imposed limitations. The number of surviving embryos began falling by day 15. By day 20 none was left. But that was enough time for a process called gastrulation to take place. Gastrulation is a vital development stage in which embryonic cells become primed to form different organs and tissues. The human cells took longer to reach this point than the monkey ones did. But they managed nevertheless, providing more evidence that the human cells were not merely passive passengers, but were mucking in to help with the process of embryonic development.

The researchers hope this biotechnological wizardry will help with two goals. One is to shed light on the complicated process of embryological development, which might eventually lead to treatments for some congenital diseases. Chimeras may offer a way around some of the ethical difficulties involved in experimenting on human embryos.

The other is the hope that chimeric animals might one day provide a source of organs to be transplanted into sick humans. In 2017 Japanese researchers demonstrated the principle by transplanting parts of a pancreas that had grown inside a mouse-rat chimera into a diabetic mouse, curing it. Whether that can work in people is, for now, unclear. And research into human chimeras is ethically fraught. America, for instance, forbids federal funding of such work. Most of the work reported in this latest paper happened in China.

But if chimeric human organs do become a reality, macaques are unlikely to be the animal of choice, says Dr Izpisa Belmonte. The most likely donor would probably be pigs (this is why his 2017 experiment focused on the animals). Their organs are roughly the size of their human equivalents, and, fairly or unfairly, they seem to provoke fewer moral qualms. (Pigs already provide thousands of people with replacement heart valves, for instance.)

The advantage of working with monkeys, at least for now, is that they are much closer, in evolutionary terms, to humans. That may have helped smooth out any compatibility issues between the two sets of cells. The hope is that lessons from experiments with humanitys close cousins might allow the researchers to revisit their work with its more distant, porcine relativesand get better results.

This article appeared in the Science & technology section of the print edition under the headline "Fantastic beasts and how to make them"

See the original post here:
Researchers have created embryos that are part-human and part-monkey - The Economist

The coronavirus pandemic has dominated the headlines and our daily lives for over a year. Medical News Today has covered this fast-moving, complex story with live updates about the latest news, interviews with experts, and an ongoing investigation into the deep racial disparities that COVID-19 has helped unmask.

However, this has not stopped us from publishing hundreds of fascinating stories on a myriad of other topics.

We begin this weeks Recovery Room with new findings of research into Parkinsons disease that may also have wider implications for the treatment of other neurodegenerative diseases and cancer.

Next comes a detailed exploration of the role that carbohydrates play in the development of diabetes. Its a complex picture, and a low carb diet may not be the panacea that some claim it to be.

We also have two reports about the influence of artificial intelligence (AI) on medical research, as well as articles covering breakthroughs in HIV prevention, sickle cell disease reversal, and heart tissue regeneration.

Finally, we look at research confirming that a steady income is good for self-confidence but may have a minimal effect on feelings about other people.

We highlight this research below, along with several other recent stories that you may have missed amid all the COVID-19 fervor.

We begin with the most-read article of the past week, with more than 124,000 page views: our report on new insights into a biochemical pathway that drives Parkinsons disease. The discovery may help scientists develop treatments for this disease, as well as type 2 diabetes and cancer.

Parkin is a protein that plays a key role in maintaining cellular energy by removing damaged mitochondria, a process known as mitophagy. The new research indicates that the biochemical pathway that activates Parkin is shorter than previously thought, which helps explain how Parkin triggers mitophagy within minutes of cellular distress occurring.

Type 2 diabetes, cancer, and some neurodegenerative diseases stem from metabolic dysregulation in damaged mitochondria, so the Parkin pathway is likely to have a role in their progression, too.

Click below to learn more about how drugs developed to treat diabetes may also be used to treat Parkinsons, or click here to visit our new Parkinsons disease resource page.

Learn more here.

Our new article exploring the role that carbohydrates play in diabetes was also very popular, with over 117,000 page views so far this week.

First, our editors looked at the relationship between carb consumption, insulin, and blood sugar. They also explored how many carbs a person with diabetes should consume, good carbs, foods to include in the diet and foods to avoid, and the effects of specific diets, including a low carb diet.

The takeaway is that not all carbs are bad for a person with diabetes, but the focus should be on healthy, whole foods rather than processed foods and those high in refined sugars.

Learn more here.

Good nutrition is important at every stage of life. This week, we published a new article on the critical role of nutrition in a childs brain development, especially in the first 3 years.

Our editors first list a variety of healthy brain foods. They then share ideas for breakfasts that may help a child stay focused at school and snacks for children to try while studying. Click the link below to see the variety of foods and nutrients that should be part of every childs diet.

Learn more here.

A reliable HIV vaccine has been difficult to develop because there are so many strains of the virus, around 50 million, according to one researcher involved in a new phase 1 clinical trial.

The new course of vaccines is designed to activatebroadly neutralizing antibodies (bnAbs) that target a wide variety of HIV strains. Only one very rare type of immune cell is able to produce bnAbs, and the vaccine activated these naive B cells in 97% of participants who received it, according to the researchers.

This novel technique could also be used to make vaccines for other diseases that have proven challenging for vaccine developers, including malaria, influenza, and hepatitis C.

Learn more here.

Two of this weeks articles highlighted how AI is being deployed to aid medical research.

First, we described ways that researchers are using powerful learning algorithms to model and predict how proteins behave in human cells. Existing AI technology used to process natural language was adapted to see if it could also predict protein language and expression.

The researchers made the technique available on a new web application, linked to in our article, that allows scientists to submit a protein sequence and view its predicted behavior.

Learn more here.

We also reported on how AI is being used to analyse the brain scans of thousands of people with multiple sclerosis (MS) in an effort to learn more about the disease.

The AI program, called SuStaIn, performed an unsupervised analysis of the brain scans and detected patterns that might otherwise have been missed. As a result, three new subtypes of the disease were identified, each presenting as different types of abnormalities in the brain.

These subtypes could be used to predict a persons response to different treatments. If this finding is supported by more clinical research, it could help ensure that the correct therapy is given at the correct time.

Learn more here.

This week, we reported on a new study that concludes that a drug that appears to reverse sickle cell disease in mice is safe for humans. The drug candidate, called FTX-6058, restored the ability to produce fetal hemoglobin.

Around 1 in 365 Black people in the U.S. are born with sickle cell disease, which is relatively rare in white people. Currently, the only cure is a stem cell or bone marrow transplant, but these are very risky. FTX-6058 will now be used in a phase 2 clinical trial that includes people with sickle cell disease, for the first time, by the end of this year.

Learn more here.

Unlike humans, adult zebra fish regenerate their hearts and other organs after injury. Until recently, the way that they do this has been unclear, but researchers are now looking more closely at the role that a protein called KLF1 plays in this remarkable ability.

Inhibiting the gene for KLF1 severely limited the ability of zebra fish to regenerate heart tissue. Investigating the role of KLF1 in human hearts will require much more work, but if the protein can help regenerate heart tissue after an injury in humans, it could be a game-changer in the treatment of heart disease.

Learn more here.

April is National Autism Awareness Month, and last weeks Recovery Room featured the experience of a parent who established a school following the diagnosis of her sons autism.

This week, MNT reported on a new approach to assessing autism spectrum disorder that focuses more on proficiency and growth than deficits in a childs development. Changing the narrative in this way could help develop a more constructive and holistic way to understand each childs unique requirements and help with the selection of therapies and interventions.

This approach is also better able to account for factors such as household income and other elements of social context, compared with measuring a childs progress against a rigid set of outcomes.

Learn more here.

Finally, this week, we reported on what claims to be the first study into the emotional benefits of living with a secure income. This large study tracked the emotional responses of 1.6 million people in 162 countries.

Its key findings are that people with a steady income are more likely to feel confident and proud of themselves and that these feelings can persist for decades. However, making more money did not appear to strengthen positive feelings about others, and nor did it affect feelings such as anger.

According to this research, earnings are only linked to some inward-facing emotions, so a societys affluence may not have as strong an influence on community harmony as policymakers might expect.

Learn more here.

We hope that this weeks Recovery Room has provided a taste of the stories that we cover atMNT. We will be back with a new selection next week.

We publish hundreds of new stories and features every month. Here are some upcoming articles that may pique our readers interests:

Visit link:
The Recovery Room: News beyond the pandemic April 16 - Medical News Today

Six decades ago, two researchers at the Ontario Cancer Institute at Princess Margaret Hospital made a startling discovery. James Till and Ernest McCulloch had found transplantable stem cells, special building block cells that have the ability to grow into any kind of human tissue.

Till and McCulloch were studying the effects of radiation at the time, but their work set off an explosion of research aimed at harnessing stem cells to treat all kinds of diseases and conditions. Subsequent breakthroughs in stem cell therapy have been used to treat more than 42,000 patients for hemophilia, restore sight to blind mice and even help a 78-year-old man regrow the end of a sliced-off fingertip. And researchers are still unlocking what might be possible.

The potential of regenerative medicine is astounding, says Michael May, president of the Centre for Commercialization of Regenerative Medicine (CCRM), a Toronto non-profit that helps bring new stem cell therapies and other regenerative medicine technologies to market. Researchers are harnessing stem cells to repair, replace or regenerate human cells, tissues and organs with the aim of improving treatments for conditions ranging from diabetes to blindness to heart failure and cancer.

More recent advances most notably Shinya Yamanakas Nobel Prize-winning 2012 discovery that regular adult tissue cells can be reprogrammed to become stem cells again, therefore endowing them with the ability to become any type of cell in the body have also ushered in a new wave of regenerative medicine research and what May calls a global race to bring newly possible cell therapies to market.

As president of CCRM, Mays job is to help move some of that research from the laboratory into the real world. Over the last decade, his organization has helped 11 companies come to market with regenerative medicine technologies, such as Montreals ExCellThera, which provides new therapeutic options for patients who suffer from myeloid leukemia and lack a traditional bone marrow donor.

While the last decade was defined by research and technological breakthroughs, May says the next decade will be all about lowering manufacturing costs and tackling patient access bottlenecks. Last November, CCRM announced that it would partner with McMaster Innovation Park in Hamilton to create Canadas first commercial-scale factory for making cells, which will be able to produce billions of cells enough to treat thousands of patients per week.

Weve just scratched the surface of whats possible in regenerative medicine, May says. He envisions a time when well eventually use these techniques not just to cure and fix human bodies, but also make them better. Now we can make cells, we can design them by genetically engineering them to do things that they naturally do, but that can be more than nature designed, says May. He says the editing of human traits in this way could eventually augment human abilities to such an extent that theyre unrecognizable.

Biomaterials are another technology that could transform regenerative medicine. Before joining CCRM, May himself helped found a Toronto biomaterials startup called Rimon Therapeutics, which developed a smart dressing for chronic wounds that used special polymers to support the bodys natural healing process. Similar advanced biomaterials could eventually be used in combination with cell therapies to not just fight aging and degeneration, but to also prevent it entirely, and even improve upon the human bodys natural baseline health.

Fifty years from now if theres some sort of blindness, well have a lens on the eye that will automatically focus and react or change as the eye ages, he says.

Nick Zarzycki is a freelancer who writes about technology for MaRS. Torstar, the parent company of the Toronto Star, has partnered with MaRS to highlight innovation in Canadian companies.

Disclaimer This content was produced as part of a partnership and therefore it may not meet the standards of impartial or independent journalism.

Originally posted here:
Being bionic: the future of regenerative medicine - Toronto Star

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Last weekends online meeting of the American Association for Cancer Research gave a glimpse at the newest ideas for fighting cancer. Among the most exciting were treatments that engineer natural cells into cancer-targeting torpedoes.

Engineered-cell treatments from Affimed (ticker: AFMD), Fate Therapeutics (FATE), and Rubius Therapeutics (RUBY) drew attention from Wall Street analysts.

The German company Affimed reported exciting results in a four-patient test of its AFM13 antibodies in patients with the blood cancer lymphoma. When administered with a kind of immune cell known as a natural killer cell, Affimeds antibody binds the NK cells to a target found on many cancer cells.

Before treatment started at the end of last year, all four lymphoma patients had been very sick. One had even been consigned to hospice.

The cancer receded in all four patients after treatment, with complete responses in two of them. The patient who had been sent to hospice is now eligible for a bone-marrow transplant. Side effects werent a problem.

They had all failed multiple lines of treatmentup to 14 lines of therapy, said Katy Rezvani, a professor at the University of Texas MD Anderson Cancer Center who is leading the study, on a Thursday morning conference call. The fact that we are seeing responses as it is, I think, is just incredible.

Dose levels of the Affimed-primed killer cells were kept deliberately low in the first administrations of the treatment. Higher doses may show deeper response and greater persistence of the natural killer cells, notes BMO Capital Markets analyst Do Kim in a Thursday note. Affimed is testing other antibodies in Phase 1 studies that target solid tumors.

Excitement over the lymphoma study results lifted Affimed stock from about $7.70 to $10.70 since last week. In recent trading, the shares were down 9.3%, at $9.38, after the development-stage company reported a slightly higher-than-expected loss of 0.50 euros a share (about 60 cents) for the 2020 year. BMOs Kim rates Affimed at Outperform, with a $15 price target.

One promising feature of natural-killer-cell treatments is that the cells can be obtained from donors and stored on the shelf. Current treatments with other engineered immune cells must harvest a patients own cells, then modify and grow them for re-administration. Another pioneer in developing NK cell therapies is Fate Therapeutics, and the company provided several updates at the AACR meeting on its treatments.

Fates stock enjoyed a remarkable run in the past year, soaring from $19 to a January 2021 peak of $119, before settling back to a recent $84.87. That puts an $8 billion market cap on a company thats yet to report revenue. But Fate has 10 clinical trials in Phase 1 for its NK cell technology, which takes undifferentiated stem cells (stored on the shelf) and transforms them into NK cells targeting a variety of solid and blood tumors.

At AACR, Fate focused on its laboratory studies that show the flexibility of its NK cell technology. In planned meetings later this year, it will review its human trials against various cancers. The stocks gain has some analysts rating it Neutral. Among them is H.C. Wainwrights Robert Burns, who wrote in January about the excitement that followed Fates report that its treatment had reversed the cancer in a patient whose lymphoma had resisted seven other kinds of therapy.

Burns may have a Neutral rating on Fates stock, but he thinks it could rise to $108 as the company reports more clinical trial results this year. Thats a better than 25% upside.

One more engineered cell therapy discussed at AACR was the unusual approach of Rubius Therapeutics, which turns off-the-shelf red blood cells into therapies that stimulate an immune system attack on cancer. Researchers detailed results first reported in March, from a Phase 1 trial against several different cancers. The treatment reversed cancers in one patient with metastatic melanoma and another with metastatic anal cancer, while stabilizing the disease in six other patients.

Rubius will escalate doses in the Phase 1 study, while proceeding with other trials that target different kinds of cancer or combine its treatment with other cancer therapies. Guggenheims Michael Schmidt projects that Rubius could start to see revenue from its unique approach in 2024, and rates the stock a Buy. He argues that its stock, now trading at $23.86, is worth at least $30.

Write to Bill Alpert at william.alpert@barrons.com

Read more:
'Natural Killer Cells' and Other Promising Cancer Treatments - Barron's

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While ElsaLys continues to work on the filing of marketing approval in Europe and the U.S. for inolimomab, the company confirms the renew of its cohort ATU in France and compassionate use programs submissions in several other countries

Lyon, FRANCE, April 15, 2020, ElsaLys Biotech confirmed that The French National Agency for the Medicines and Health Products Safety (ANSM) has renewed the Temporary Authorisation for Use (ATU) so-called cohort ATU (cATU) for inolimomab (LEUKOTAC) on December 24, 2020.

This renewed authorization includes the implementation of a reinforced monitoring (defined in the Protocol for Therapeutic Use) of the efficacy and safety data obtained in patients treated within the framework of this cATU.

Inolimomab is available to hematologists and physicians treating blood disorders and to hospital pharmacists for the treatment of acute cortico-resistant or corticosteroid-dependent graft-versus-host disease in adults and pediatric patients over 28 days of age. The indication should be discussed during a multidisciplinary consultation meeting. Inolimomab treatment can only be considered if the patient cannot be included in an ongoing clinical trial.

During its first year of administration under cATU and despite the pandemic, around 30 patients in France have been treated with inolimomab as it is considered a reliable treatment of this high-risk patient population.The number of patients included in the cohort ATU in France since December 2019 reflects a real medical need. This is the reason why ElsaLys is working on expanding compassionate use programs for inolimomab in a number of European countries. Several early access applications will be submitted.

" Inolimomab is already being administered in France before marketing autorisation in Europe and hopefully soon in other countries through compassionate use programs as it is considered a reliable treatment of this high-risk patient population with acute cortico-resistant or corticosteroid-dependent graft-versus-host disease. said Dr. Christine GUILLEN, CEO and co-founder of ElsaLys Biotech. " Data on clinical benefit and safety profile we expect to collect through these compassionate use programs will support our work on the filing of marketing authorization applications (MAA) in Europe and in the U.S.

About inolimomab

Inolimomab is an anti-IL-2 R monoclonal antibody active as an immunotherapy product for the treatment of steroid-refractory acute GvHD.

In acute GvHD, activated T cell lymphocytes from the allografts donor recognize and attack recipient tissues. T cell lymphocyte activation and proliferation is governed by the key IL-2/IL-2 receptor (IL-2 R) pathway.

By recognizing the subunit of the IL-2 Receptor complex (IL-2 R) which is upregulated on T cells upon activation, inolimomab blocks the binding of the cytokine IL-2 on IL-2 R thereby inhibiting IL-2 signalling and donor T cell proliferation.

The efficacy of inolimomab in aGvHD relies on its specific potent immunosuppression on T cell lymphocytes through the blocking of the IL-2/IL-2 R pathway triggering the disease.

Inolimomab received Orphan Drug Designation in Europe (March 2001) and in the U.S. (October 2002).

About steroid-resistant aGvHD

Formerly called bone marrow transplant, Hematopoietic Stem Cell Transplantation (HSCT) is the last therapeutic option for patients with certain blood cancers or severe immunodeficiency. In practice, the treatment is designed to replace the diseased blood cells of the patient with the hematopoietic stem cells of a matching donor (allograft).

Once grafted, these stem cells will produce new healthy and functional blood cells, including white blood cells that will allow patients to bridge their immune deficiency or to eliminate surviving cancer cells.

If this technique has made considerable progress in 60 years, half of transplant recipients are still victims of complications: side effects of conditioning treatments, immunosuppressive treatments before allograft (that aims to prevent transplant rejection), long-term susceptibility to infections and GvHD. In the latter case, the donors over-active T-cells turn against the patients tissues: mucous membranes, skin, gastro-intestinal tract, liver and lungs. The acute form appears just after the transplant, the chronic form occurring several months later (preceded or not by an acute GvHD episode).

Affecting between 30 to 50% of patients, GvHD is the main complication of hematopoietic stem cell transplantation. To halt this disease, physicians use corticosteroids. The fact remains that some 30 to 50% of aGvHD patients are refractory or dependent to the steroid treatment. To date limited therapeutic options are available for these patients with no standard treatment approved so far in Europe and only one in the US.

About ElsaLys Biotech

ElsaLys Biotech is a specialty pharmaceutical company, part of the Mediolanum Farmaceutici Spa group, focused on innovative medicines to address haemato-oncology related life-threatening and rare diseases.

Following strategic acquisitions and targeted developments, ElsaLys is establishing an immunotherapeutic portfolio focused on niche specialty pharmaceuticals to answer unmet medical needs.

Our commitment is to offer essential drugs meeting Public Health needs.

Founded in 2013, ElsaLys Biotech is located in the heart of the European cluster Lyon Biopole, in Lyon, France.

Stay in touch with ElsaLys Biotech and receive directly our press releases by filling our contact form on https://www.elsalysbiotech.com

And follow us on Twitter @ElsalysBiotech and on linkedin.com/company/elsalys-biotech/

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While ElsaLys continues to work on the filing of marketing approval in Europe and the US for inolimomab, the company confirms the renew of its cohort...

While youre receiving treatment for cancer, some of the drugs you take can cause painful sores to develop inside your mouth. You can also get them if youve had a bone marrow (stem cell) transplant as part of your cancer care.

Although they often heal on their own, these mouth sores can make it uncomfortable to eat and talk. Well discuss what you can do to relieve the pain and prevent them from getting worse.

Mouth sores can be a common side effect of cancer treatment. The condition, known as stomatitis or mucositis, is an inflammation of the tissues inside your mouth.

Whitish, ulcer-like sores can form on your cheeks, gums, lips, tongue, or on the roof or floor of your mouth. Even if you dont develop mouth ulcers, you may have patches that feel inflamed and painful, as if theyve been burned.

Anyone who is receiving chemotherapy, radiation therapy, or a bone marrow (stem cell) transplant can develop mouth sores as a side effect of these treatments.

If you have dry mouth or gum disease, or if your teeth and gums are not well taken care of, you may be at a higher risk of getting mouth sores during your treatment. Women and people who smoke or drink alcohol are also at a higher risk, according to the Oral Cancer Foundation.

If youre receiving chemotherapy, the sores could begin forming anywhere from 5 days to 2 weeks after your treatment. Depending on the specific cause, the sores could go away on their own in a few weeks, or they could last longer.

Its important to find ways to manage your pain and to watch for signs of an infection. Cancer-related mouth sores can lead to weight loss, dehydration, and other serious complications.

Cancer cells can grow very quickly. The aim of cancer treatment is to stop or slow down that growth. The cells in the mucous membranes lining your mouth are also fast-growing cells, so cancer treatments affect them, too.

Cancer treatments also keep the cells in your mouth from being able to repair themselves efficiently when theyre damaged.

Radiation therapy can also damage the glands in your mouth that make saliva. A dry mouth is more susceptible to infections that cause mouth sores.

Chemotherapy and radiation can both change the microbiome in your mouth, upsetting the balance between good and bad bacteria. The growth of harmful bacteria in your mouth can also lead to mouth sores.

Sometimes cancer treatments suppress your immune system, which may make it more likely that youll get a bacterial, viral, or fungal infection that causes mouth sores. An older infection (such as the herpes simplex virus) can also suddenly flare up again.

If youve had a bone marrow (stem cell) transplant, sores may be a sign that youve developed a condition known as graft-versus-host disease (GVHD).

When this happens, the cells in your body are attacking the transplanted cells as though they were an unhealthy invader. According to research published in Journal of Clinical and Experimental Dentistry, short-term (acute) GVHD occurs in 50 to 70 percent of stem cell transplant cases and longer-term (chronic) GVHD is seen in 30 to 50 percent of cases.

The form of GVHD that causes mouth sores is usually mild, and doctors often treat it with corticosteroid medications.

Its important to talk with your doctor if you develop mouth sores after a stem cell transplant, as some kinds of GVHD can turn serious if left untreated.

There is a good chance that youll experience mouth sores at some point during your cancer treatment. Researchers estimate that 20 to 40 percent of those who have chemotherapy and 80 percent of those who have high-dose chemotherapy will develop mucositis afterward.

Still, there are steps you and your cancer care team can take to lower your risk, reduce the severity of the sores, and promote faster healing.

About a month before your cancer treatment begins, schedule an appointment with your dentist to make sure your teeth and gums are healthy. If you have cavities, broken teeth, or gum disease, its important to come up with a dental treatment plan to take care of these conditions so they dont lead to infections later, when your immune system may be vulnerable.

If you wear braces or dentures, ask your dentist to check the fit and remove any part of the device you dont need during your treatment.

Its very important to maintain good oral hygiene practices throughout your treatment to lower your risk of infection. Brush and floss gently but regularly, avoiding any painful areas. You can also ask your dentist whether a mouth rinse with fluoride is advisable in your case.

For certain kinds of chemotherapy (bolus 5fluorouracil chemotherapy and some high-dose therapies), your healthcare team may give you ice chips to chew for 30 minutes before your treatment. This type of cold therapy can lower your risk of getting mouth sores later.

During treatment of some blood cancers, doctors may give you injections of palifermin, also known as human keratinocyte growth factor-1 (KGF-1), to prevent mouth sores.

If youre scheduled to receive high-dose chemotherapy or radiotherapy, your cancer care team may prepare your mouth using low-level laser therapy beforehand to keep you from getting mouth sores.

For people who have radiation therapy for head and neck cancers, doctors may prescribe this medicated mouthwash to minimize mouth sores.

The length of time your mouth sores may last depends on the specific cancer treatment youve had. Here are some estimates broken down by treatment:

You may notice symptoms anywhere between a few days and a few weeks after your cancer treatment. Heres what you may see and feel as mucositis develops:

You may notice that the sores become slightly crusty as they heal. Its important to keep track of your symptoms and let your oncologist know if the sores arent healing on their own.

Contact your doctor right away if you:

Untreated mouth sores can lead to malnutrition, dehydration, and life-threatening infections.

There are a few different ways that you can help mouth sores heal and avoid prolonger pain or an infection.

While the sores are healing, its very important to keep the inside of your mouth clean to prevent an infection from developing.

The National Cancer Institute recommends that you gently clean your teeth every 4 hours and just before you go to sleep at night. Here are a few tips to consider:

If the pain from mouth sores is interfering with your ability to eat and drink, your doctor may treat the condition with a opioid mouthwash or one containing doxepin or lidocaine.

To ease discomfort and keep your mouth from feeling dry, you may want to try rinsing with a mild saltwater or baking soda solution. Heres how to make each of them:

Your cancer care team may recommend that you use a lubricating liquid (artificial saliva) to moisten the inside of your mouth if dryness is a problem. These liquids are usually gel-like. They coat your mouth with a thin film to help ease discomfort and promote healing.

Some people have found it useful to rinse with a blend of medications called the magic mouthwash. Formulas for this mouthwash vary, but most of them include a combination of medications to treat different symptoms, including:

Magic or miracle mouthwash solutions usually have to be prescribed by a doctor and prepared by a pharmacist, although some people mix up an over-the-counter version at home.

There isnt enough research to say for sure whether magic mouthwash works. If you think youd like to try it, talk with your oncologist or a healthcare professional about whether its a good idea for you.

Here are a few more things you can try at home that may help ease pain from mouth sores:

Mouth sores are one of the most common side effects of cancer treatment. Shortly after chemotherapy, radiation, or transplant treatments, painful, ulcer-like sores can form on the inside of your mouth.

These sores may go away on their own. If they dont, its important to seek medical treatment for them because they can lead to very serious complications.

Before you start cancer treatments, visit a dentist to make sure your teeth and gums are healthy. Keeping up good dental hygiene practices during and after cancer treatment will help limit mouth sores.

If the sores are keeping you from eating and drinking, talk with your oncologist about medications could relieve the pain and speed up the healing process, so you can enjoy a better quality of life during treatment.

Its really important to keep track of any sores in your mouth so you can reach out to your healthcare team if they dont improve. Sores that deepen or worsen can lead to serious even life-threatening complications.

More:
Mouth Sores from Chemo: Symptoms, Causes, and Treatments - Healthline

SASKATOON -- An effort to increase stem cell donors within Black communities across Canada is being driven by a group of women whove had difficulty finding full genetic matches themselves.

Genetic matches are crucial for patients in need of stem cell transplants, such as those with leukemia and lymphoma, and matches are more commonly found within their own racial, ethnic and ancestral groups.

But the new Black Donors Save Lives campaign notes that fewer than two per cent of those in the Canadian Blood Services stem cell donor registry are Black.

And that decreases their chance of finding a match, campaign lead Sylvia Okonofua told CTVNews.ca in a phone interview. It becomes a numbers game for Black people on the stem cell waiting list, where its like finding a needle in a hay stack for them.

The recent University of Regina biochemistry graduate, with sights on becoming a hematologist, timed the virtual campaign to kick off during Black History Month.

It was overall frustrating to know that a patient from my community is so much less likely than other patients to be helped, she told CTVNews.ca. When you see that your people have a really, really low chance of being helped out, it takes you aback.

Okonofua noted part of the campaign uses TikToks, shareable infographics, and even an original song to get the message out and reach a wide audience.

And she said part of the outreach involves having Black stem cell recipients talk about their experiences with the health-care system and speak to the historical mistrust the Black community has towards the medical community.

She founded her campus chapter of Stem Cell Club, a non-profit organization with chapters across Canada which recruits Canadians as potential stem cell donors.

Registration for Black Donors Save Lives can be done online, where participants between the ages of 17 to 35 can fill out a questionnaire and have a swab kit mailed to their address. After they swab the inside of their cheeks and send the sample back, if there is a person in need, 90 per cent of donors will be asked to donate stem cells very similar to the way a person would be giving blood.

But a big difference is the donor is given a growth hormone a week before donation in order to increase the number of stem cells, as well as the process taking four to six hours.

Alternatively, one out of 10 donors will be asked if theyd like to donate stem cells via bone marrow surgery, which can take place over a day.

In 2017, Reve Agyepong experienced firsthand the lack of Black stem cell donors, to treat her sickle cell disease, which involve red blood cells becoming misshapen, which can block blood vessels and lead to damage to bones, brain, kidneys, and lungs, and can ultimately be fatal.

But Agyepong, who was born in Edmonton to Ghanaian parents, was fortunate to receive a stem cell transplant from her sister.

It is such a blessing to have a match within your own family because the percentages are just so low, she told CTVNews.ca by email. I am so fortunate to have found a match in my family or else transplant would have been off the table for me.

In fact, only one in four patients who need a stem cell transplant are able to find a matched donor within their family, with Black patients being less than half as likely as white patients to find a unrelated person they match with on a donor registry, according to the campaign.

For Jamaican-Canadian Dorothy Vernon-Brown, who helped inspire this months campaign, the current efforts are deeply personal. In 2013, she was diagnosed with acute myeloid leukaemia and was heartbroken to discover there were no stem cell matches in Canada's registry or internationally.

She ultimately received stem cells from her sister, who was a half-match, and has been spreading information to Black Canadians ever since, through her own advocacy group, Donor Drive for Dorothy.

Stem cell transplantation is a miracle for patients, and I wish people knew how easy it is to be a stem donor, she recounted on a Twitter thread for another stem cell awareness campaign. You could give someone an opportunity like my sister gave me, to be around and live the life I want. People want to live, so if that gift is in your hands, I appeal to you to see it as something significant to do in your life.

Okonofua and Vernon-Browns efforts are being aided by Dr. Warren Fingrut, a hematologist whos the director of the aforementioned Stem Cell Club.

He told CTVNews.ca in an email hes seen firsthand far too many patients from ethnic and racial minority groups in situations where they dont have fully-matched donors and are forced to seek other treatments.

I find this heart wrenching and I am very motivated to work to address this, Fingrut said.

That led to him founding his non-profit a decade ago, which has gone on to recruit more than 20,000 Canadians as stem cell donors, with more than 55 per cent being non-white. But in cases such as Vernon-Brown and others, those figures need to be much higher.

We started running national campaigns last year, focused on the recruitment of diverse peoples as donors, as well as males who are also preferred by transplant physicians (all else being equal) as they are associated with better outcomes for patients, Fingrut explained.

The campaign is also being done in partnership with several other groups, including the Katelyn Bedard Bone Marrow Association, Black Physicians of Canada, Black Medical Students Association of Canada and the National Black Law Students Association of Canada.

This campaign is one example of an initiative in the health-care sector, which seeks to address racial disparity impacting the care of Black patients, he wrote, noting Black people face many such disparities in access to care, and we want to see others in the health-care sector working with Black Canadians to tackle these issues and address them, in collaboration with Black communities.

Okonofua hopes next Black History Month, theyll be able to have in-person swabbing events in places of worship, community hubs, and cultural gatherings to show how easy it is.

Fingrut said this the first time his group has specifically engaged with one racial group and hopes to expand it to other ethnic and racial communities including South Asians, Indigenous peoples, and those of mixed ancestry in the near future.

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Meet the women hoping to recruit more stem cells donors from Black communities - CTV News

We can absolutely cut the number of cancer deaths down so that one day in our lifetimes it can be a rare thing for people to die of cancer, said Patrick Hwu, MD, president and CEO of Moffitt Cancer Center in Florida and among gene therapys pioneers. It still may happen here and there, but itll be kind of like people dying of pneumonia. Its like, He died of pneumonia? Thats kind of weird. I think cancer can be the same way.

The excitement returned in spades in 2017 when the FDA signed off on a gene-therapy drug for the first time, approving the chimeric antigen receptor (CAR) T-cell treatment tisagenlecleucel (Kymriah; Novartis) for the treatment of B-cell precursor acute lymphoblastic leukemia. At last, scientists had devised a way to reprogram a persons own T cells to attack tumor cells.

Were entering a new frontier, said Scott Gottlieb, MD, then the FDA Commissioner, in announcing the groundbreaking approval.

Gottlieb wasnt exaggerating. The growth in CAR T-cell treatments is exploding. Although only a handful of cell and gene therapies are on the market, FDA officials predicted in 2019 that the agency will receive more than 200 investigational new drug applications per year for cell and gene therapies, and that by 2025, it expects to have accelerated to 10 to 20 cell and gene therapy approvals per year.1

Essentially, you can kill any cancer cell that has an antigen that is recognized by the immune cell, Hwu said. The key to curing every single cancer, which is our goal, is to have receptors that can recognize the tumor but dont recognize the normal cells. Receptors recognizing and then attacking normal cells is what can cause toxicity.

Cell therapy involves cultivating or modifying immune cells outside the body before injecting them into the patient. Cells may be autologous (self-provided) or allogeneic (donor-provided); they include hematopoietic stem cells and adult and embryonic stem cells. Gene therapy modifies or manipulates cell expression. There is considerable overlap between the 2 disciplines.

Juliette Hordeaux, PhD, senior director of translational research for the University of Pennsylvanias gene therapy program, is cautious about the FDAs predictions, saying shed be thrilled with 5 cell and/or gene therapy approvals annually.

For monogenic diseases, there are only a certain number of mutations, and then well plateau until we reach a stage where we can go after more common diseases, Hordeaux said.

Safety has been the main brake around adeno-associated virus vector (AAV) gene therapy, added Hordeaux, whose hospitals program has the institutional memory of both Jesse Gelsingers tragic death during a 1999 gene therapy trial as well as breakthroughs by Carl June, MD, and others in CAR T-cell therapy.

Sometimes there are unexpected toxicity [events] in trials.I think figuring out ways to make gene therapy safer is going to be the next goal for the field before we can even envision many more drugs approved.

In total, 3 CAR T-cell therapies are now on the market, all targeting the CD19 antigen. Tisagenlecleucel was the first. Gilead Sciences received approval in October 2017 for axicabtagene ciloleucel (axi-cel; Yescarta), a CAR T-cell therapy for adults with large B-cell non-Hodgkin lymphoma. Kite Pharma, a subsidiary of Gilead, received an accelerated approval in July 2020 for brexucabtagene autoleucel (Tecartus) for adults with relapsed or refractory mantle cell lymphoma.

On February 5, 2021, the FDA approved another CD19-directed therapy for relapsed/refractory large B-cell lymphoma, lisocabtagene maraleucel (liso-cel; JCAR017; Bristol Myers Squibb). The original approval date was missed due to a delay in inspecting a manufacturing facility (see related article).

Idecabtagene vicleucel (ide-cel; bb2121; Bristol Myers Squibb) is under priority FDA review, with a decision expected by March 31, 2021. The biologics license application seeks approval for ide-cel, a B-cell maturation antigendirected CAR therapy, to treat adult patients with multiple myeloma who have received at least 3 prior therapies.2

The number of clinical trials evaluating CAR T-cell therapies has risen sharply since 2015, when investigators counted a total of 78 studies registered on the ClinicalTrials.gov website. In June 2020, the site listed 671 trials, including 357 registered in China, 256 in the United States, and 58 in other countries.3

Natural killer (NK) cells are the research focus of Dean Lee, MD, PhD, a physician in the Division of Hematology and Oncology at Nationwide Childrens Hospital. He developed a method for consistent, robust expansion of highly active clinical-grade NK cells that enables repeated delivery of large cell doses for improved efficacy. This finding led to several first-in-human clinical trials evaluating adoptive immunotherapy with expanded NK cells under an FDA Investigational New Drug application. He is developing both genetic and nongenetic methods to improve tumor targeting and tissue homing of NK cells. His eff orts are geared toward pediatric sarcomas.

The biggest emphasis over the past 20 to 25 years has been cell therapy for cancer, talking about trying to transfer a specific part of the immune system for cells, said Lee, who is also director of the Cellular Therapy and Cancer Immunology Program at Nationwide Childrens Hospital, at The Ohio State University Comprehensive Cancer Center Arthur G. James Cancer Hospital, and at the Richard J. Solove Research Institute.

The Pivot Toward Treating COVID-19 and Other Diseases

However, Lee said, NKs have wider potential. This is kind of a natural swing back. Now that we know we can grow them, we can reengineer them against infectious disease targets and use them in that [space], he said.

Lee is part of a coronavirus disease 2019 (COVID-19) clinical trial, partnering with Kiadis, for off-the-shelf K-NK cells using Kiadis proprietary platforms. Such treatment would be a postexposure preemptive therapy for treating COVID-19. Lee said the pivot toward treating COVID-19 with cell therapy was because some of the very early reports on immune responses to coronavirus, both original [SARS-CoV-2] and the new [mutation], seem to implicate that those who did poorly [overall] had poorly functioning NK cells.

The revolutionary gene editing tool CRISPR is making its initial impact in clinical trials outside the cancer area. Its developers, Jennifer Doudna, PhD, and Emmanuelle Charpentier, PhD, won the Nobel Prize in Chemistry 2020.

For patients with sickle cell disease (SCD), CRISPR was used to reengineer bone marrow cells to produce fetal hemoglobin, with the hope that the protein would turn deformed red blood cells into healthy ones. National Public Radio did a story on one patient who, so far, thanks to CRISPR, has been liberated from the attacks of SCD that typically have sent her to the hospital, as well from the need for blood transfusions.4

Its a miracle, you know? the patient, Victoria Gray of Forest, Mississippi, told NPR.

She was among 10 patients with SCD or transfusion-dependent beta-thalassemia treated with promising results, as reported by the New England Journal of Medicine.5 Two different groups, one based in Nashville, which treated Gray,5 and another based at Dana-Farber Cancer Institute in Boston,6 have reported on this technology.

Stephen Gottschalk, MD, chair of the department of bone marrow transplantation and cellular therapy at St Jude Childrens Research Hospital, said, Theres a lot of activity to really explore these therapies with diseases that are much more common than cancer.

Animal models use T cells to reverse cardiac fibrosis, for instance, Gottschalk said. Using T cells to reverse pathologies associated with senescence, such as conditions associated with inflammatory clots, are also being studied.

Hordeaux said she foresees AAV being used more widely to transmit neurons to attack neurodegenerative diseases.

The neurons are easily transduced by AAV naturally, she said. AAV naturally goes into neurons very efficiently, and neurons are long lived. Once we inject genetic matter, its good for life, because you dont renew neurons.

Logistical Issues

Speed is of the essence, as delays in producing therapies can be the difference between life and death, but the approval process takes time. The process of working out all kinks in manufacturing also remains a challenge. Rapid production is difficult, too, because of the necessary customization of doses and the need to ensure a safe and effective transfer of cells from the patient to the manufacturing center and back into the patient.7

Other factors that can slow down launches include insurance coverage, site certification, staff training, reimbursement, and patient identification. The question of how to reimburse has not been definitively answered; at this point, insurers are being asked to issue 6- or even 7-figure payments for treatments and therapies that may not work.8

CAR T, I think, will become part of the standard of care, Gottschalk said. The question is how to best get that accomplished. To address the tribulations of some autologous products, a lot of groups are working with off -the-shelf products to get around some of the manufacturing bottlenecks. I believe those issues will be solved in the long run.

References

1. Statement from FDA Commissioner Scott Gottlieb, MD, and Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies. News release. FDA website. January 15, 2019. https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics. Accessed January 13, 2021.

2. Bristol Myers Squibb provides regulatory update on lisocabtagene maraleucel (liso-cel). News release. Bristol Myers Squibb; November 16, 2020. Accessed January 11, 2021. https://news.bms.com/news/details/2020/Bristol-Myers-Squibb-Provides-Regulatory-Update-on-Lisocabtagene-Maraleucel-liso-cel/default.aspx

3. Wei J, Guo Y, Wang Y. et al. Clinical development of CAR T cell therapy in China: 2020 update. Cell Mol Immunol. Published online September 30, 2020. doi:10.1038/s41423-020-00555-x

4. Stein R. CRISPR for sickle cell diseases shows promise in early test. Public Radio East. November 19, 2019. Accessed January 11, 2021. https://www.publicradioeast.org/post/crisprsickle-cell-disease-shows-promise-early-test

5. Frangoul H, Altshuler D, Cappellini MD, et al. CRISPR-Cas9 gene editing for sickle cell disease and -Thalassemia. N Engl J Med. Published online December 5, 2020. DOI: 10.1056/NEJMoa2031054

6. Esrick EB, Lehmann LE, Biffi A, et al. Post-transcriptional genetic silencing of BCL11A to treat sickle cell disease. N Engl J Med. Published online December 5, 2020. doi:10.1056/NEJMoa2029392

7. Yednak C. The gene therapy race. PwC. February 5, 2020. Accessed January 11, 2021. https://www.pwc.com/us/en/industries/healthindustries/library/gene-therapy-race.html

8. Gene therapies require advanced capabilities to succeed after approval. PwC website. Accessed January 11, 2021. https://www.pwc.com/us/en/industries/health-industries/library/commercializing-gene-therapies.html

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The Untapped Potential of Cell and Gene Therapy - AJMC.com Managed Markets Network

In people with polycythemia vera (PV), the bone marrow produces too many blood cells. This overproduction can lead to complications, such as abnormal blood clotting, unusual bleeding, and an enlarged spleen.

In rare cases, scar tissue may replace the bone marrow. When this happens, the bone marrow can no longer produce enough healthy blood cells. Experts refer to this condition, which is a type of chronic leukemia, as myelofibrosis (MF). It can sometimes lead to acute myeloid leukemia, though this is rare.

People with PV have a shorter-than-average life expectancy. Some of the possible complications of the disease can be life threatening.

Getting treatment can help reduce the risk of certain complications from PV, including blood clots. As a result, a person will likely lead a longer and healthier life with this disease if they receive treatment.

According to an article in Blood Cancer Journal, the median survival time for people with PV is 14 years after diagnosis. The authors take this survival time from a study in which half of the participants were still alive 14 years after diagnosis.

Younger people tend to live for longer with the disease. Research suggests that the median survival time for those under 60 years of age is 24 years following diagnosis.

Multiple factors affect the outlook and life expectancy of people with PV, including:

Blood clots are the most common cause of death in people with PV. When blood clots form in blood vessels, they can block the flow of blood to vital organs. This can lead to life threatening complications, such as stroke, heart attack, and venous thrombosis.

Treatment for PV can help relieve symptoms and lower the risk of blood clots. In this way, it also reduces a persons risk of life threatening complications.

In most cases, healthcare providers prescribe regular blood draws to treat PV. Blood draws reduce the number of blood cells in the body, which may help improve blood flow.

Healthcare providers may also prescribe low dose aspirin to help prevent the formation of blood clots. Additionally, they may prescribe other medications, such as hydroxyurea (Hydrea) or busulfan (Myleran).

If a person develops MF as a complication of PV, their healthcare provider may prescribe one or more of the following treatments:

These treatments may help improve symptoms, increase life expectancy, or both.

For example, scientists have found that stem cell transplants may help improve long-term survival in people with MF. However, this treatment comes with a high risk of life threatening side effects. It is especially risky for older adults and people with other health conditions. As a result, healthcare providers often avoid prescribing this treatment.

Some studies have found that treatment with JAK inhibitors may also improve survival rates in people with MF. However, when scientists reviewed the available evidence on Jakafi and Inrebic, they found that the quality of evidence on survival rates is limited. More research is necessary to confirm how these treatments affect life expectancy.

Early research involving people with PV found that the median survival time for those who did not receive treatment was less than 2 years after diagnosis. This research took place before the medical community recognized blood draws as a treatment option, and it reflects the high risk of blood clots in people not receiving treatment.

People with PV who do not receive treatment are more likely to develop blood clots. According to the Leukemia & Lymphoma Society, 4060% of people with untreated PV may develop blood clots within 10 years of diagnosis.

Scientists have not yet developed a cure for PV. However, healthcare providers may prescribe blood draws, medications, or other treatments to help manage symptoms, reduce the risk of complications, and increase life expectancy in people with this disease.

Researchers are also continuing to develop and test potential new treatments for PV, such as the anticancer drug imatinib mesylate (Gleevec) and novel types of JAK inhibitors.

In some cases, a persons healthcare provider may encourage them to take part in a clinical trial. In this type of study, participants receive an experimental treatment. People interested in learning more about the potential benefits and risks of taking part in a clinical trial can talk with their healthcare provider or the researchers running the study.

When a person receives a diagnosis of PV, getting treatment is important. Treatment may help minimize symptoms, lower the risk of complications, and improve life expectancy.

A persons recommended treatment plan for PV will depend on many factors, including their age, overall health, and whether they have developed certain complications.

People with PV who wish to learn more about their treatment options and outlook should talk with their healthcare provider.

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Polycythemia vera life expectancy: With treatment and more - Medical News Today