B-cell acute lymphoblastic leukemia: What to know – Medical News Today

B-cell acute lymphoblastic leukemia is a type of acute lymphoblastic leukemia (ALL). It is cancer that affects white blood cells known as B-cell lymphoblasts.

Lymphocytes form part of the immune system and protect the body against infections. People who have B-cell acute lymphoblastic leukemia have too many immature B-cell lymphoblasts in their bloodstream and bone marrow that do not work correctly.

As these leukemia cells increase, there are fewer healthy, mature white blood cells that fight infections and disease, red blood cells, and platelets. People with B-cell acute lymphoblastic leukemia may experience infections and anemia. They may also bleed easily.

B-cell lymphoblastic leukemia is the most common type of acute lymphoblastic leukemias or lymphomas, making up around 75% of adult leukemia cases. It can affect both adults and children.

This article looks at B cell acute lymphoblastic leukemia, as well as its causes, symptoms, and prognosis. It also explores how doctors diagnose and treat the condition.

ALL accounts for less than 0.5% of all cancers in the United States and 80% of these cases occur in children.

Doctors do not understand the exact cause of B-cell acute lymphoblastic leukemia. They do know that it involves the DNA in the bone marrow that controls how white blood cells divide, grow, and die.

In B-cell acute lymphoblastic leukemia, mutations or changes in the DNA mean that the cells do not follow the normal process of growth and maturation. Instead, the cells replicate and divide more rapidly and release immature white blood cells. These are called leukemia cells.

Mature B-cells mark infected cells with a protein, which other immune system cells then destroy. Leukemia cells are not capable of protecting the body from infection.

Scientists remain unsure why this happens but think it may be a combination of genetic and environmental factors.

Genetic changes can make someone more likely to develop leukemia by increasing the likelihood that mutations occur within their bone marrow.

For example, in adults, a change can happen if the DNA from a chromosome breaks off and attaches to a different chromosome. This is called translocation. In adults with ALL, the most common translocation is between chromosomes 9 and 22, and results in what is called the Philadelphia chromosome.

People with chromosomal conditions like Down syndrome also have an increased risk of developing B-cell acute lymphoblastic leukemia.

Usually, people develop the DNA mutations that can lead to ALL, rather than inheriting them. They may happen because the person had exposure to radiation or cancer-causing chemicals, but often the cause is unclear. Mutations can also occur because people have undergone radiotherapy and chemotherapy for other cancer.

Certain other factors can increase a persons risk of developing B-cell acute lymphoblastic leukemia, including high birth weight and smoking.

Many ALL symptoms are due to shortages of normal mature blood cells and can include:

People may also have a swollen abdomen due to a build-up of leukemia cells in the liver and spleen. If the leukemia cells build up near the surface of the bone or inside joints, this can cause pain.

Doctors diagnose ALL by using various tests to inspect the bone marrow or blood for signs of abnormal cells and to identify specific cells. Understanding someones diagnosis helps the doctor estimate how ALL may progress and determine the best treatment for them.

Tests may include:

Doctors confirm a diagnosis of ALL if 20% of the bone marrow cells are lymphoblasts.

There are no proven methods to prevent someone from developing leukemia. In general, people should avoid unnecessary exposure to cancer-causing chemicals like benzene, pesticides, and radiation as much as possible.

People should also avoid smoking and smoke inhalation, a risk factor for many cancers, including leukemias like ALL.

For adults with ALL, doctors usually use long-term chemotherapy. More intensive regimens may result in better responses but can cause more side effects, like low white blood cell counts.

Treatment usually has induction, consolidation, and maintenance phases that span around 2 years, although the ALL subtype and other factors can affect the length and intensity of the regime.

The first phase of treatment is induction therapy, which uses medication to help stabilize and reduce the number of lymphoblasts and regulate the individuals blood cell production. After this phase, for most people, the leukemia is in remission, meaning that there are no leukemia cells in bone marrow samples, and the person has normal blood counts.

The other stages of treatment aim to destroy any remaining leukemia cells in the body. The stages vary in length and intensity depending on how the leukemia responds.

Stem cell transplants are an option for some individuals to replace bone marrow affected by lymphoblasts with healthier, new bone marrow. Doctors need to match the donor bone marrow carefully to the individual.

Children with ALL receive a similar three-phase chemotherapy regime but may also need prolonged hospital stays for treatment, as infections and other complications can occur.

Children also receive chemotherapy in the cerebrospinal fluid (CSF) to kill any cancer cells in the brain and spinal cord.

The prognosis for individuals with B-cell acute lymphoblastic leukemia depends on various factors such as the persons age at diagnosis.

Children and young people are significantly more likely to undergo successful treatment and enter remission. For children with ALL, the 5-year survival rate is 85%. For adults with ALL, the 5-year survival rate is 69.9%.

A persons white blood cell count at the point of diagnosis also plays a role. People with a lower white blood cell count are more likely to make a full recovery. Additionally, how well a person responds to chemotherapy affects their recovery.

People who have concerns about ALL should contact their doctor or healthcare practitioner for advice. Cancer facilities often have support staff who can direct people to resources and support.

In the United States, the Leukemia and Lymphoma Society and American Cancer Society offer support and information for people living with ALL and other types of leukemia.

B-cell acute lymphoblastic leukemia is one of the most common types of leukemia in children but is rare in adults.

Various treatments aim to put the individual in remission and typically involve an extended chemotherapy regime.

The outlook for individuals with B-cell acute lymphoblastic leukemia is improving, especially among children, who often make a full recovery.

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Chronic lymphocytic leukemia and stomach pain: What is the link? – Medical News Today

Chronic lymphocytic leukemia (CLL) is a type of blood cancer. It affects lymphocytes, a type of immature white blood cell that the body produces in the bone marrow.

People with CLL may experience discomfort, swelling, and pain in the abdomen if their spleen enlarges. In rare cases, CLL can also affect mucosal tissues, such as those lining the gastrointestinal (GI) tract.

This article discusses the link between CLL and stomach pain in more detail. It also looks at ways to prevent and treat CLL, the other possible symptoms, and the outlook for people with this condition.

People with CLL may experience various symptoms involving the abdomen or stomach, such as:

In most cases, people with CLL do not experience abdominal symptoms due to the disease until it progresses and becomes more severe.

Research suggests that CLL infiltrates and affects the GI tract in about 5.713% of cases. When CLL affects the GI tract, doctors may call it Richters syndrome.

People with CLL may experience abdominal swelling, discomfort, and tenderness as a result of their spleen becoming larger. Less commonly, they may also feel full after eating small amounts of food, as the spleen can press on the stomach, making it smaller and able to hold less.

In rare cases, CLL infiltrates the lining of the GI tract, causing inflammation and ulcers or open wounds. People may experience symptoms similar to those of inflammatory bowel disease (IBD) and malabsorption disorders. These symptoms may include diarrhea, nausea, vomiting, abdominal pain and cramping, and unintentional weight loss.

CLL is cancer that develops in lymphocytes, which are white blood cells that form in the bone marrow and help fight infection.

Lymphocytes make up most of the lymph tissues in the lymph nodes, thymus gland, adenoids, tonsils, and spleen. They are also present in the GI tract, bone marrow, and respiratory system.

CLL is a type of leukemia that develops gradually over time.

About 5075% of people with CLL do not experience noticeable symptoms. Due to this, doctors diagnose most people with CLL during routine blood work.

The symptoms of CLL often begin when the cancerous cells crowd out healthy cells in the bone marrow or migrate to other organs or tissues. When symptoms first appear, they are typically mild, but they then become increasingly severe. CLL can cause many nonspecific symptoms, so a person may feel as though they have a cold or the flu.

Possible symptoms of CLL that do not relate to the abdominal area include:

Doctors do not yet have a way to prevent leukemia. However, some types of leukemia, including CLL, may have links to toxins, such as herbicides, pesticides, radon, and tobacco exposure. People can help reduce the risk of CLL by avoiding or practicing extreme caution around these toxins.

Many people do not experience symptoms of CLL for years and do not require treatment. However, as the disease progresses, these individuals may need treatment to extend their lives.

When and how a doctor treats someones CLL depends on a few factors, including:

Chemotherapy is typically the first-line therapy for CLL.

Doctors may use chemotherapy in conjunction with other treatment options, such as monoclonal antibody therapy. This therapy binds antibodies to cancer cells and destroys them. Treatment can also include medications to treat or prevent infections or improve low blood cell levels.

For instance, some people may take a combination of the monoclonal antibody rituximab and the chemotherapy drugs fludarabine and cyclophosphamide. Alongside the oral chemotherapy medication chlorambucil, doctors use obinutuzumab or ofatumumab, which have the same drug target as rituximab.

Small molecule inhibitors, such as bendamustine hydrochloride, idelalisib, and ibrutinib, can also sometimes form part of a CLL treatment regimen.

In 2017, the Food and Drug Administration (FDA) approved the combination medication Rituxan Hyecela (rituximab and hyaluronidase human) for CLL treatment.

In recurring or aggressive cases of CLL, an individual may have a blood or bone marrow stem cell transplant. This procedure replaces diseased cells with healthy blood cells that are able to mature into bone marrow cells.

Doctors may treat CLL until the symptoms lessen and then stop treatment until the symptoms worsen again.

Many people with CLL live for many years with a high quality of life.

There is no cure for CLL, so treatment aims to extend and improve someones life by reducing their symptoms. Doctors treat many people intermittently as their symptoms reoccur.

A persons outlook depends on their age, overall health, underlying conditions, and stage of CLL. Typically, people who are over the age of 65 years or have a more advanced stage of CLL have a less positive outlook.

Genetic changes in CLL cells and increased beta-2 microglobulin protein levels in the blood can make CLL more challenging to treat, potentially affecting a persons outlook.

Doctors classify people with CLL into different risk groups depending on certain factors. Based on these risk groups, the estimated percentages of people surviving 5 years or more after their diagnosis is:

People with more advanced or severe CLL may experience abdominal swelling, discomfort, tenderness, and pain. They may also feel full after eating small amounts. More rarely, someone with CLL may develop GI tract inflammation or ulcers, which can cause symptoms such as diarrhea, nausea, vomiting, cramping, and unexplained weight loss.

Anyone who thinks that they may have CLL should speak with a doctor. People with a confirmed diagnosis who experience symptoms of more advanced or severe CLL, such as abdominal pain, should also seek medical care.

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Chronic lymphocytic leukemia and stomach pain: What is the link? - Medical News Today

BlueRock and Senti Collaborate to Build Smarter, Disease-Fighting Cell Therapies – BioSpace

Determined to take its next-generation engineered cell therapies to the next level, BlueRock Therapeuticsis teaming up withSenti Biosciences with futuristic medicines in mind.

BlueRockhas proved its merit with a therapy for Parkinsons disease with preclinical data demonstrating the ability to restore motor function and increase dopamine release over time. APhase I trial.for the drug started in Canada last month.

Using BlueRocks cell+gene platform to create universal pluripotent stem cells, Senti will step in to design, build and test Smart Sensors and Regulator Dials in BlueRocks regenerative medicine candidates. The addition of Sentis gene circuits will precisely control cell differentiation and therapeutic payload expression.

There is a tremendous opportunity at the intersection of cell, gene and systems biology. With control of all three axes, we believe we can further engineer the cells inherent potential to integrate multiple physiologic inputs to produce powerful therapeutic benefit in vivo, said Emile Nuwaysir, CEO of BlueRock.

Sentifounder Tim Lu compared the companys gene circuit tech to that of a Roomba vacuum in aTedMed talk. While the Roomba can constantly calculate where its been, where it needs to go next, and where the mess is to clean your home effectively, Senti is building cells that can compute information to do the same. Therapies of the future would enter the body inactive, go to the area of the body where needed and switch on to affect only the cells required to treat the disease.

Lus company has demonstrated how this can work in cancer cells. Through an "and" logic gate, the cancer therapy is designed to only attack when it senses two cancer signatures, over just one, ensuring it kills cancer cells and not healthy cells. The program then kills the cancer cell directly and recruits the rest of the immune system into the battle to reduce the ability of tumor cells to escape.

This latest team up with BlueRock would not only program cells that would only activate in the presence of a particular set of indicators but also come with fine tuning. After the therapy has already entered the body, doctors could introduce an alreadyFDA-approved small molecule drug to increase or decrease the delivered therapeutic payload. Most current cell and gene therapies cannot be adjusted once a patient has received them.

While the companies chose not to disclose the target of their programs at what is a very early stage of the collaboration, the main areas of focus, according to BlueRock, are neurology, immunology and cardiology.

Lu commented, We are engineering gene circuits to reprogram cells with biological logic to sense inputs, compute decisions and respond to their cellular environments. By combining BlueRocks iPSC platform with our sophisticated gene circuits, we believe that we have the potential to create the next generation of programmable regenerative medicines together.

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BlueRock and Senti Collaborate to Build Smarter, Disease-Fighting Cell Therapies - BioSpace

Top 12 Disruptive Gene and Cell Therapy Technologies Announced – GlobeNewswire

Boston, MA, May 21, 2021 (GLOBE NEWSWIRE) -- Mass General Brigham today announced its selections for the sixth annual Disruptive Dozen, 12 emerging gene and cell therapy (GCT) technologies with the greatest potential to impact health care in the next few years. The technologies were featured as part of the World Medical Innovation Forum held virtually from Boston to examine GCTs potential to impact patient care including a range of diseases and health system opportunities.

The 2021 Mass General Brigham Disruptive Dozen are:

Researchers have pinpointed key genes involved in cholesterol and lipid metabolism that represent promising targets for new cholesterol-lowering treatments. Instead of disabling a disease-related protein, gene-silencing therapies prevent the protein from being made at all. That durability means patients could receive an injection of a gene-silencing drug every six months to control their blood cholesterol. Another transformative genetic medicine can alter the instructions written in a particular gene. Known as CRISPR base editing, this technology offers precision and potential permanence: patients may be able to undergo a one-time treatment and maintain healthy cholesterol levels for a lifetime.

Currently, devastating diseases such as sickle-cell disease and beta-thalessemia can only be cured by a bone marrow transplant, which can be risky and not feasible due to the lack of suitable donors. Now, new genome editors tools that make precise changes to a persons DNA are paving the way toward a different kind of cure. One approach uses a type of genome editing called CRISPR, and involves reactivating fetal hemoglobin, which can substitute for the missing or faulty adult version in these diseases. This CRISPR-based gene therapy is now being tested in clinical trials and early results are encouraging. Other gene therapies are also in the works, including those based on older technologies that augment rather than repair defective genes.

Genome editing technologies are having a significant impact across biomedicine, especially on the field of gene therapy. Despite their precision and ease of use, these tools cannot fix every genetic mutation, including those that change a single genetic base, similar to a one-letter misspelling on a page. More than 30,000 point mutations in the human genome are known to cause disease. Thanks to a new class of genome-editing tools, known as base editors, it is feasible to correct some of these so-called point mutations. The first base-editing therapies are now under development for a range of human diseases including sickle cell disease, inherited blindness, and genetic forms of high cholesterol. As base editing technologies continue to mature, researchers are also working to apply it to more common diseases, such as Alzheimers disease.

The first gene therapies to reach the clinic use viruses which have been molecularly honed and tailored to allow for the safe, effective delivery of human genes. While these viruses can transfer genes into cells a requirement for gene therapy they are not a perfect solution. Now, as scientists seek to build next-generation gene therapies, they are pursuing alternatives for gene delivery. These include highly sophisticated bubbles fashioned from nanoparticles, which help protect and direct gene therapies to their intended destination within the body. If gene therapies can be targeted more precisely to specific organs or tissues, they could be used to treat a broader range of disorders. These efforts are boosted by the recent development of a pair of highly effective coronavirus vaccines that use lipid-based nanoparticles to deliver their therapeutic cargo.

Some life-saving therapies, including certain forms of gene therapy, depend on bone marrow stem cells. But these cells are not easily accessible, and the protocol is long and can cause pain, nausea, and other complications. Scientists are developing a new approach that promises to streamline this process and help reduce the barriers that can hinder the delivery of some gene therapies. This new method holds promise not only for bone marrow transplantation, but also for gene therapies that depend on manipulating bone marrow stem cells. These treatments known broadly as ex vivo gene therapy require isolating bone marrow stem cells, treating the cells outside of the body with gene therapy, and then infusing the modified cells back into patients bloodstream.

One of the first gene therapies approved in the U.S. treats a rare genetic form of blindness with a one-time injection into the eye. Its success is paving the way for many other eye gene therapies that are now under development. Some 200 genes in humans are directly linked to vision problems, underscoring the incredible potential of the technology. Scientists are also pursuing novel gene therapies for another critical sensory organ, the ear. With more than 150 genes tied to hearing loss and deafness, there is a great need for treatments that can help protect and restore hearing. Millions of people in the U.S. suffer from hearing loss, yet there are currently no FDA-approved medicines to treat it. Unlike the eye, the inner ear is difficult to reach with therapeutics. To help overcome this hurdle, scientists have fine-tuned the molecular make-up of the viruses used in gene therapy to create versions that can penetrate the ears internal structures.

Approximately 10 million people worldwide suffer from Parkinsons disease, a chronic condition that stems from the progressive loss of dopamine-producing neurons in the brain, which help control movement. Unfortunately, there is no available drug that protects or stops the neurons from dying. But scientists and clinicians are developing a revolutionary approach to replace these lost neurons, harnessing stem cell-based methods to convert patients own blood cells into dopamine-producing neurons. Although this cell therapy does not fix the root causes of Parkinsons disease, it could provide a functional cure by replacing the dopamine-producing neurons in patients brains and restoring normal movement to their bodies.

Type 1 diabetes affects over a million people in the U.S. Patients must keep track of their blood sugar levels and inject themselves periodically with insulin, all because the cells in their own bodies that supply the hormone have been destroyed by the immune system. Scientists are working on a novel cell-based treatment for type 1 diabetes that involves replacing these lost insulin-producing cells with a special laboratory-grown variety. Over the last several years, scientists have developed several formulas for generating these cells using different stem cells as the key ingredient, along with cloaking strategies and efforts to enable replacement cells to release their own immune-blocking signals. As these technologies continue to advance toward the clinic, researchers hope to bring them to bear on another disease: type 2 diabetes. Worldwide there are over 400 million people with type 2 diabetes, many require insulin injections, underscoring the need for a more durable solution.

CAR-T therapy is a groundbreaking form of gene and cell therapy in which a patients own immune cells are isolated, genetically rewired in the laboratory with certain therapeutic properties, and then infused back into the bloodstream. For difficult to treat blood cancers, CAR-T therapies have proven remarkably effective, with some patients living for years cancer-free. Researchers are now working to expand the reach of this transformative technology by simplifying cell production and manufacturing and applying the approach to other disease areas. Scientists are also creating off-the-shelf versions of CAR-T therapies, selecting from an assortment of pre-made options for an immunological match for a patient. This could help expand the number of patients who could receive CAR-T therapies and minimize the time between doctors prescribing the treatment and patients receiving it. There are also efforts underway to broaden the diseases that CAR-T therapies can treat, including development of CAR-T therapies that can kill solid tumors or target entirely new areas, like autoimmune disease.

A virus found in nature has become a workhorse of gene therapy. Known asadeno-associated virus, or AAV, it is a popular choice among gene therapy developers because of its long track record and overall safety. But its not a perfect solution. Thats why scientists are working to create designer AAVs in the laboratory that address some of the virus shortcomings. The work promises to expand the clinical impact of gene therapy by broadening the number of patients and diseases that can benefit. Using data-driven methods, scientists are modifying the molecular make-up of the viruses to generate enhanced versions that home to specific organs, like the lung and kidney, which are not targeted by the current slate of therapeutic AAVs. Researchers are also fine-tuning AAVs to infect some cells in a tissue but not others for example, a specific subtype of neurons in the brain. Finally, efforts are underway to create AAVs that can evade detection by the immune system, which would help expand the clinical impact of gene therapy by making more patients eligible to receive it.

Some gene therapies seek to repair or replace whats been lost, like genes that are abnormally silent because of a genetic misspelling that terminates their usual function. But other genes can be broken in a different way that gives them new, often unexpected behaviors. To address these wayward genes, scientists have devised a class of innovative gene therapies called antisense oligonucleotides, or ASOs. They are designed with biochemical precision to shut down the activity of a target gene at its molecular roots and hold promise for neurodegenerative diseases. ASOs are relatively straightforward to engineer, so they can often be designed more quickly than other therapies. Over the last four years, six new ASO drugs were approved by the FDA, and many more are under development for a range of conditions, including neurodegenerative diseases such as ALS, Huntingtons disease, and Alzheimers disease.

Glioblastoma is the most common type of brain cancer in adults, and, tragically, most patients die within a year to 18 months of diagnosis. Now, using a variety of approaches from cancer-killing viruses to rewired immune cells to even cancer cells themselves researchers are working to develop a slate of innovative treatments with the power to eradicate glioblastoma tumors and give patients longer, cancer-free lives. One approach involves cancer-killing viruses, engineered in the laboratory to seek and destroy tumors. Researchers are also applying CAR-T cell technology, in which patients own immune cells are isolated, molecularly rewired with therapeutic powers, and then put back in the body. Another novel cell therapy builds on a remarkable, decade-old discovery: cancer cells that spread within the body can find their way back to their original tumor. This re-homing is spurring efforts to genetically engineer patients own tumor cells to endow them with cancer-killing properties. Once the cells are placed back into the body, they can return home and destroy their counterparts.

For detailed information on each of the Disruptive Dozen technologies, including video updates, please visit https://worldmedicalinnovation.org/2021-disruptive-dozen/

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About Mass General Brigham Mass General Brigham is an integrated academic healthcare system, uniting great minds in medicine to make life-changing impact for patients in our communities and people around the world. Mass General Brigham connects a full continuum of care across a system of academic medical centers, community and specialty hospitals, a health insurance plan, physician networks, community health centers, home care, and long-term care services. Mass General Brigham is a non-profit organization that is committed to patient care, research, teaching, and service to the community. In addition, Mass General Brigham is one of the nations leading biomedical research organizations and a principal teaching affiliate of Harvard Medical School. For more information, please visit massgeneralbrigham.org.

About Mass General Brigham Innovation Innovation is the 150-person business development unit of Mass General Brigham responsible for the worldwide commercial application of the unique capabilities and discoveries of Mass General Brigham's 74,000 employees. Innovation supports the research requirements of its 6,200 Harvard Medical School faculty and research hospitals. It has responsibility for industry collaborations, venture investing, international consulting, licensing, innovation management, company creation, technology marketing, open innovation alliances, and workforce development. Its annual World Medial Innovation Forum is underway virtually May 19-21.

Media Contact: Rich Copp Mass General Brigham: rcopp@partners.org

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Living with blood cancer – Lara Said – Times of Malta

World Blood Cancer Day is marked today. The celebration of this day was instituted in 2014 because, in 1991, Methchild Ehringer could not find a match by a German non-profit organisation DKMS founded by Methchilds family. She died because she was unable to find a match. The aim was and still remains to find a potential donor for every person needy of a bone marrow/stem cell transplant.

Today, there are 10 million potential donors registered when compared to the initial 3,000. Some might remember me as the person who needs Daratumumab included in the government formulary for free medicinals. I am interested in other matters too!

World Blood Cancer Day! This day highlights a more personal issue that concerns the realisation that I do not stand alone in my blood cancer journey. An issue that cuts across the three most common blood cancers: leukaemia, lymphoma and myeloma. Of these three, the lymphomas and the myelomas are the more common. For many people, such as myself, the diagnosis of a blood cancer is a shock. Like water, blood is meant to sustain a person.

Blood cancers bring along with them a lot of uncertainty and anxiety. For some blood cancers, such as multiple myeloma, a cure does not exist. To suddenly acknowledge that what is being generated in your bone marrow and what is circulating around your body and through your own blood is threatening you and your body, is psychologically invasive in a way like no other. I felt robbed. Robbed by my own body, my own immune system and, perhaps, by my own past lifestyle choices.

Stem cell transplantation offers hope of increased longevity, when appropriate, to a good proportion of blood cancer patients. A successful stem cell transplant means time out of hospital, visiting usually only every few months for monitoring. There are two types of stem cell transplantation: autologous and allogenic.

Autologous transplantation is when a person donates to oneself. As was the case with myself and my two attempts for autologous transplantation.

For many people, such as myself, the diagnosis of a blood cancer is a shock

Allogenic requires matching a donor to a patient. This is only suitable in specific cases. The government of Malta does pay for such transplantation. Charities such as Puttinu are incredibly supportive in supporting those undergoing stem cell transplantation by providing accommodation. On World Blood Cancer Day 2021, my wish is that the Maltese public understand what I consider to be three critical issues.

First, for many, stem cell/bone marrow transplants and, increasingly, cellular innovative therapies, potentially require a donor. Second, for a portion of those requiring such intervention/therapy they cannot donate to themselves and/or find a donor from their family. Third, millions of people are required to donate their stem cells.

As of today, I am under the impression that Malta and its generally very good healthcare system does not yet, have a register for stem cell donation. I hope I am wrong. Should I be right, I urge the powers that be to strongly consider this as part of their long-term strategic vision. What I do know, however, is that Malta has the local expertise and the equipment to collect stem cell transplants.

Perhaps because it is a small-island state, Malta does not have the facility for storage. The healthcare system is probably likely, especially at this point in time, not to possess as much capacity to assure consistent and sustained storage of stem cells according to European and international gold-standard criterion. This is likely to be primarily due to space issues given that the local expertise is available and excellent.

In more recent times especially, monoclonal agents, such as Daratumumab, are increasingly offered as more frontline treatment to those with an early diagnosis of multiple myeloma, at least in other EU countries and to those able to afford payment.

Chimeric Antigen therapy (CAR-T cell), which forms part of cellular therapeutic options, is also in the pipeline.

Monoclonal therapy and CAR-T cell are consequently likely to decrease the need for stem cell transplants. This is positive but, if anything, highlights even more the need for a stem cell database, register and repository. Newer therapies generally tend to be increasingly stem cell therapy dependent in some form or other.

What I would like the reader to appreciate is that I am not a medical professional. I have been at times accused of being a dreamer but life has taught me two things.

To turn lemons into lemonade and to give without the expectation of taking or receiving.

Today, I would like to go a bit beyond my myeloma, so to speak. I want to celebrate, as a blood cancer survivor, what works for me. I urge all of you to read about blood cancers and try to empathise with all blood cancer survivors.

Above all, let us not forget their carers: spouses, children, friends, doctors, nurses and everybody else whom I have inadvertently omitted.

I, for one, would not be here, especially, without the excellent care and patience of doctors, nurses, physiotherapists, etc. alongside the emotional care, motivation and interest offered by my sons, friends, work colleagues and those generally understanding and supportive of my condition, and, yes, my Lara still needs Dara quest! Thank you.

Lara Saids dream is to set up a non-profit organisation to advocate especially for the rights of patients with myeloma, leukaemia and lymphoma for Malta and Gozo. She is here appealing to survivors, their relatives and/or carers to help her set up a patient group.

Lara Said, Multiple myeloma survivor, member, Myeloma Patients Europe

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