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Side Effects of a Bone Marrow Transplant (Stem Cell Transplant)

By daniellenierenberg

A bone marrow transplant is a medical treatment that replaces your bone marrow with healthy bone marrow stem cells. It is also called a stem cell transplant or, more specifically, a hematopoietic stem cell transplant. This type of transplantation can treat certain types of cancer and other diseases that affect the bone marrow. Like any cancer treatment, it can cause side effects. These side effects can be different for everyone and depend on the type of transplant you receive, your general health, and other factors.

It is a good idea to talk with your health care team about the possible side effects before starting your transplant process. This includes short-term side effects that are expected to go away over time, as well as side effects that may occur later, last longer, or be permanent. This will help you feel more prepared and supported if a side effect does occur.

And, talk with your health care team regularly about any symptoms or side effects you experience throughout your transplantation process and recovery. This includes when a side effect worsens or a new problem appears. Managing side effects is an important part of cancer care and treatment and it is especially important during transplantation. This type of care is called palliative or supportive care. It can help people with any stage of cancer feel better.

There are different kinds of bone marrow transplants and the side effects can be different. The side effects for an autologous transplant and an allogenic transplant are detailed below.

An autologous bone marrow transplant is also called an AUTO transplant or stem cell rescue. During an AUTO transplant, your own stem cells are removed from your body before an intensive chemotherapy treatment. This intensive treatment, which can also include radiation therapy, damages your stem cells. The healthy stem cells are then put back in your body to "replace" the ones damaged by the treatment.

Many side effects of an AUTO transplant are similar to common side effects of chemotherapy and radiation therapy. The most serious side effect is a higher risk of infection from your body's low levels of white blood cells.

Infection. Chemotherapy and some other treatments weaken your body's infection-fighting system, called the immune system. This is especially true of treatment given for a bone marrow/stem cell transplant, because the bone marrow is part of the immune system. When your immune system is weakened, your body cannot protect itself as well against germs. Most of these germs already live in your body. When your immune system is strong, these germs do not make you sick. But after a transplant, they can cause an infection. Fortunately, most of these infections can be easily treated with antibiotics.

About 2 weeks after your transplant day, your immune system will begin to recover. You have the highest risk of infections in the first few weeks after transplant, but you will still be at a higher risk of infections for a year or more after. Your health care team will talk to you about ways to reduce your risk of infections during your recovery. Learn more about infections as a side effect of cancer treatment.

Other immediate side effects of AUTO transplants. The following side effects can develop right after the high doses of chemotherapy used for AUTO transplants:

Long-term side effects of AUTO transplants. Some transplant side effects happen months or years later. These can include:

An allogenic transplant is also called an ALLO transplant. In an ALLO transplant, the replacement cells come from another person, called a donor. After a round of chemotherapy and sometimes radiation therapy, you will receive the donor's healthy cells.

The side effects of an ALLO transplant are similar to common side effects of chemotherapy and radiation therapy. This includes a high risk for infections. You are also at risk of side effects caused by having another person's stem cells, including a risk of graft-versus-host disease (GVHD; see below). Many people also have a "graft-versus-cancer-cell effect" along with GVHD. This is because the new stem cells recognize and destroy cancer cells that are still in the body. It is the main way ALLO transplants work to cure cancers like leukemia.

Infection. After an ALLO transplant, your doctor will give you chemotherapy, with or without radiation therapy or other drugs, to keep your body's immune system from destroying the new donated cells. These treatments affect your immune system and make infection risk higher. A weak immune system makes you more likely to get infections.

You are at the highest risk of infection in the first few weeks after receiving the donor's cells. The risks lessen over time, but infection risk reduction is an important part of your long-term recovery.

Graft-versus-host disease (GVHD). Sometimes donor cells can attack your body, causing inflammation. This is a specific side effect of ALLO transplantation called GVHD. Even if your donor was a 100% match, you can still get GVHD. Your health care team can give you medication to prevent GVHD. If you still experience GVHD, your doctor will give you more medications to manage the condition. GVHD can be life-threatening in some cases.

There are 2 types of GVHD: acute and chronic. Both can range from mild to severe.

This form of GVHD happens in the first 3 months after an ALLO transplant. It often affects the skin, intestines, and liver. It can cause rashes, diarrhea, and jaundice. Jaundice is a liver problem that makes skin and the whites of the eyes look yellow.

The treatment for acute GVHD is to block T cells. T cells are white blood cells that help the immune system fight infections. Blocking them keeps your transplanted immune system from attacking your body's own cells.

Chronic GVHD usually develops more than 3 months after an ALLO transplant. It can last a few months or the rest of your life.

Chronic GVHD may or may not cause symptoms or need treatment. You may need treatment for specific problems. Some common problems of chronic GVHD include:

There are 2 medications approved by the U.S. Food and Drug Administration (FDA) to treat chronic graft-versus-host disease.

Ruxolitinib (Jakafi) in adults and children 12 years and older after 1 or more treatments with systemic therapy

Ibrutinib (Imbruvica) in children 1 year and older after 1 or more treatments with systemic therapy

Chronic GVHD can be treated with medications called corticosteroids. If this does not work well, you might take other medications to make your immune system less active.

Other immediate side effects. Side effects that can develop right after the high doses of chemotherapy used for ALLO transplantation include the following.

Late or long-term side effects. Some transplantation side effects can happen months or years later. These can include:

People who have less powerful chemotherapy treatments before their transplant tend to have fewer long-term physical effects.

Talk with your health care team about possible physical side effects of your bone marrow transplant, as well as what signs to watch for. They can help answer your questions and make a plan to manage short-term and long-term side effects.

Bone marrow transplantation is an extended medical process, and many people experience a variety of emotional and social challenges during this treatment and recovery. This can include anxiety and depression. It can also include the uncertainty and stress that cancer brings, self-image changes, changes in relationships with loved ones, feelings of isolation, and grieving losses from cancer and its treatment.

Be sure to share your feelings, including with your health care team. They want to know how you are feeling during and after transplantation. There are many ways to help support your mental health during this stressful time, including counseling, joining a support group, journaling, art therapy, mindfulness, and meditation.

It is important to talk often with your health care team about different types of side effects, before, during, and after your transplant. This helps you gather information and make decisions on treatment and care. Here are some possible questions to ask.

What tests will be done before the transplant process starts to check my general health?

When could I start to experience side effects during this process?

What specific side effects are common with this type of transplant? How can each one be managed or relieved?

Who should I call if I experience any side effects from my transplant?

What signs of an infection should I look out for?

What precautions to prevent infection should I follow? For how long?

What side effects should I tell my health care team right away?

If I will have an ALLO transplant, will I take any medications to prevent GVHD?

If I will have an ALLO transplant, what are the signs of GVHD that I should watch for?

What tests will I need later? How often?

What are the possible late effects of a transplant? How can they be managed or relieved?

How will having a transplant affect my daily life? Can I work? Can I exercise and do regular activities? Or, when can I restart these activities during my recovery?

Will having a transplant affect my sex life? If so, how and for how long?

Will having this transplant affect my ability to have a child in the future? If so, can you refer me to a fertility specialist before treatment begins?

Why is good nutrition important during and after a transplant? Should I meet with an oncology registered dietitian?

Who can I talk with about the emotional effects of cancer and this treatment?

What can I do at home to keep myself as healthy as possible?

What is a Bone Marrow Transplant (Stem Cell Transplant)?

Resources on Bone Marrow/Stem Cell Transplant

Coping With the Fear of Treatment-Related Side Effects

Survivorship

Bone Marrow Transplant and Older Adults

Be the Match: Life After Transplant

Be the Match: GVHD Signs and Symptoms

BMT InfoNet: Transplant Basics

National Bone Marrow Transplant Link: Publications on Side Effects and Survivorship

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Side Effects of a Bone Marrow Transplant (Stem Cell Transplant)

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December 25th, 2022

28-year-old cancer patient at Nebraska Medicine advocates for diversity in bone marrow registry – KMTV 3 News Now Omaha

By daniellenierenberg

28-year-old cancer patient at Nebraska Medicine advocates for diversity in bone marrow registry KMTV 3 News Now Omaha

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December 17th, 2022

Stem Cell Technologies and Applications Market Report 2022-2032 – Yahoo Finance

By daniellenierenberg

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December 9th, 2022

Fred Hutch at ASH: Global insights on AML outcomes, COVID-19 and cancer, CD19 CAR T-cell therapy updates, latest on precision oncology and more -…

By daniellenierenberg

Fred Hutch at ASH: Global insights on AML outcomes, COVID-19 and cancer, CD19 CAR T-cell therapy updates, latest on precision oncology and more Newswise

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Fred Hutch at ASH: Global insights on AML outcomes, COVID-19 and cancer, CD19 CAR T-cell therapy updates, latest on precision oncology and more -...

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December 9th, 2022

Types of Stem Cell and Bone Marrow Transplants – American Cancer Society

By daniellenierenberg

Stem cell transplants are used to give back stem cells when the bone marrow has been destroyed by disease, chemotherapy (chemo), or radiation. Depending on where the stem cells come from, the transplant procedure may be called:

They can all be called hematopoietic stem cell transplants.

In a typical stem cell transplant for cancer, very high doses of chemo are used, sometimes along with radiation therapy, to try to kill all the cancer cells. This treatment also kills the stem cells in the bone marrow. This is called myeloablation or myeloablative therapy. Soon after treatment, stem cells are given (transplanted) to replace those that were destroyed. The replacement stem cells are given into a vein, much like ablood transfusion. The goal is that over time, the transplanted cells settle in the bone marrow, begin to grow and make healthy blood cells. This process is called engraftment.

There are 2 main types of transplants. They are named based on who donates the stem cells.

In this type of transplant, the first step is to remove or harvest your own stem cells. Your stem cells are removed from either your bone marrow or your blood, and then frozen. (You can learn more about this process at Whats It Like to Donate Stem Cells?) After you get high doses of chemo and/or radiation as your myeloablative therapy, the stem cells are thawed and given back to you.

Benefits of autologous stem cell transplant: One advantage of autologous stem cell transplant is that youre getting your own cells back. When you get your own stem cells back, you dont have to worry about them (called the engrafted cells or the graft) being rejected by your body.

Risks of autologous stem cell transplant: The grafts can still fail, which means the transplanted stem cells dont go into the bone marrow and make blood cells like they should. Also, autologous transplants cant produce the graft-versus-cancer effect. A possible disadvantage of an autologous transplant is that cancer cells might be collected along with the stem cells and then later put back into your body. Another disadvantage is that your immune system is the same as it was before your transplant. This means the cancer cells were able to escape attack from your immune system before, and may be able to do so again.

This kind of transplant is mainly used to treat certain leukemias, lymphomas, and multiple myeloma. Its sometimes used for other cancers, like testicular cancer and neuroblastoma, and certain cancers in children. Doctors can use autologous transplants for other diseases, too, like systemic sclerosis, multiple sclerosis (MS), and systemic lupus erythematosis (lupus).

To help prevent any remaining cancer cells from being transplanted along with stem cells, some centers treat the stem cells before theyre given back to the patient. This may be called purging. While this might work for some patients, there haven't been enough studies yet to know if this is really a benefit. A possible downside of purging is that some normal stem cells can be lost during this process. This may cause your body to take longer to start making normal blood cells, and you might have very low and unsafe levels of white blood cells or platelets for a longer time. This could increase the risk of infections or bleeding problems.

Another treatment to help kill cancer cells that might be in the returned stem cells involves giving anti-cancer drugs after the transplant. The stem cells are not treated. After transplant, the patient gets anti-cancer drugs to get rid of any cancer cells that may be in the body. This is called in vivo purging. For instance, lenalidomide (Revlimid) may be used in this way for multiple myeloma. The need to remove cancer cells from transplanted stem cells or transplant patients and the best way to do it continues to be researched.

Doing 2 autologous transplants in a row is known as a tandem transplant or a double autologous transplant. In this type of transplant, the patient gets 2 courses of high-dose chemo as myeloablative therapy, each followed by a transplant of their own stem cells. All of the stem cells needed are collected before the first high-dose chemo treatment, and half of them are used for each transplant. Usually, the 2 courses of chemo are given within 6 months. The second one is given after the patient recovers from the first one.

Tandem transplants have become the standard of care for certain cancers. High-risk types of the childhood cancer neuroblastoma and adult multiple myeloma are cancers where tandem transplants seem to show good results. But doctors dont always agree that these are really better than a single transplant for certain cancers. Because this treatment involves 2 transplants, the risk of serious outcomes is higher than for a single transplant.

Sometimes an autologous transplant followed by an allogeneic transplant might also be called a tandem transplant. (See Mini-transplants below.)

Allogeneic stem cell transplants use donor stem cells. In the most common type of allogeneic transplant, the stem cells come from a donor whose tissue type closely matches yours. (This is discussed in Matching patients and donors.) The best donor is a close family member, usually a brother or sister. If you dont have a good match in your family, a donor might be found in the general public through a national registry. This is sometimes called a MUD (matched unrelated donor) transplant. Transplants with a MUD are usually riskier than those with a relative who is a good match.

An allogeneic transplant works about the same way as an autologous transplant. Stem cells are collected from the donor and stored or frozen. After you get high doses of chemo and/or radiation as your myeloablative therapy, the donor's stem cells are thawed and given to you.

Blood taken from the placenta and umbilical cord of newborns is a type of allogeneic transplant. This small volume of cord blood has a high number of stem cells that tend to multiply quickly. Cord blood transplants are done for both adults and children. By 2017, an estimated 700,000 units (batches) of cord blood had been donated for public use. And, even more have been collected for private use. In some studies, the risk of a cancer not going away or coming back after a cord blood transplant was less than after an unrelated donor transplant.

Benefits of allogeneic stem cell transplant: The donor stem cells make their own immune cells, which could help kill any cancer cells that remain after high-dose treatment. This is called the graft-versus-cancer or graft-versus-tumor effect. Other advantages are that the donor can often be asked to donate more stem cells or even white blood cells if needed, and stem cells from healthy donors are free of cancer cells.

Risks of allogeneic stem cell transplants: The transplant, or graft, might not take that is, the transplanted donor stem cells could die or be destroyed by the patients body before settling in the bone marrow. Another risk is that the immune cells from the donor may not just attack the cancer cells they could attack healthy cells in the patients body. This is called graft-versus-host disease. There is also a very small risk of certain infections from the donor cells, even though donors are tested before they donate. A higher risk comes from infections you had previously, and which your immune system has had under control. These infections may surface after allogeneic transplant because your immune system is held in check (suppressed) by medicines called immunosuppressive drugs. Such infections can cause serious problems and even death.

Allogeneic transplant is most often used to treat certain types of leukemia, lymphomas, multiple myeloma, myelodysplastic syndrome, and other bone marrow disorders such as aplastic anemia.

For some people, age or certain health conditions make it more risky to do myeloablative therapy that wipes out all of their bone marrow before a transplant. For those people, doctors can use a type of allogeneic transplant thats sometimes called a mini-transplant. Your doctor might refer to it as a non-myeloablative transplant or mention reduced-intensity conditioning (RIC). Patients getting a mini transplant typically get lower doses of chemo and/or radiation than if they were getting a standard myeloablative transplant. The goal in the mini-transplant is to kill some of the cancer cells (which will also kill some of the bone marrow), and suppress the immune system just enough to allow donor stem cells to settle in the bone marrow.

Unlike the standard allogeneic transplant, cells from both the donor and the patient exist together in the patients body for some time after a mini-transplant. But slowly, over the course of months, the donor cells take over the bone marrow and replace the patients own bone marrow cells. These new cells can then develop an immune response to the cancer and help kill off the patients cancer cells the graft-versus-cancer effect.

One advantage of a mini-transplant is that it uses lower doses of chemo and/or radiation. And because the stem cells arent all killed, blood cell counts dont drop as low while waiting for the new stem cells to start making normal blood cells. This makes it especially useful for older patients and those with other health problems. Rarely, it may be used in patients who have already had a transplant.

Mini-transplants treat some diseases better than others. They may not work well for patients with a lot of cancer in their body or people with fast-growing cancers. Also, although there might be fewer side effects from chemo and radiation than those from a standard allogeneic transplant, the risk of graft-versus-host disease is the same. Some studies have shown that for some cancers and some other blood conditions, both adults and children can have the same kinds of results with a mini-transplant as compared to a standard transplant.

This is a special kind of allogeneic transplant that can only be used when the patient has an identical sibling (twin or triplet) someone who has the exact same tissue type. An advantage of syngeneic stem cell transplant is that graft-versus-host disease will not be a problem. Also, there are no cancer cells in the transplanted stem cells, as there might be in an autologous transplant.

A disadvantage is that because the new immune system is so much like the recipients immune system, theres no graft-versus-cancer effect. Every effort must be made to destroy all the cancer cells before the transplant is done to help keep the cancer from coming back.

Improvements have been made in the use of family members as donors. This kind of transplant is called ahalf-match (haploidentical) transplant for people who dont have fully matching or identical family member. This can be another option to consider, along with cord blood transplant and matched unrelated donor (MUD) transplant.

If possible, it is very important that the donor and recipient are a close tissue match to avoid graft rejection. Graft rejection happens when the recipients immune system recognizes the donor cells as foreign and tries to destroy them as it would a bacteria or virus. Graft rejection can lead to graft failure, but its rare when the donor and recipient are well matched.

A more common problem is that when the donor stem cells make their own immune cells, the new cells may see the patients cells as foreign and attack their new home. This is called graft-versus-host disease. (See Stem Cell Transplant Side Effects for more on this). The new, grafted stem cells attack the body of the person who got the transplant. This is another reason its so important to find the closest match possible.

Many factors play a role in how the immune system knows the difference between self and non-self, but the most important for transplants is the human leukocyte antigen (HLA) system. Human leukocyte antigens are proteins found on the surface of most cells. They make up a persons tissue type, which is different from a persons blood type.

Each person has a number of pairs of HLA antigens. We inherit them from both of our parents and, in turn, pass them on to our children. Doctors try to match these antigens when finding a donor for a person getting a stem cell transplant.

How well the donors and recipients HLA tissue types match plays a large part in whether the transplant will work. A match is best when all 6 of the known major HLA antigens are the same a 6 out of 6 match. People with these matches have a lower chance of graft-versus-host disease, graft rejection, having a weak immune system, and getting serious infections. For bone marrow and peripheral blood stem cell transplants, sometimes a donor with a single mismatched antigen is used a 5 out of 6 match. For cord blood transplants a perfect HLA match doesnt seem to be as important, and even a sample with a couple of mismatched antigens may be OK.

Doctors keep learning more about better ways to match donors. Today, fewer tests may be needed for siblings, since their cells vary less than an unrelated donor. But to reduce the risks of mismatched types between unrelated donors, more than the basic 6 HLA antigens may be tested. For example, sometimes doctors to try and get a 10 out of 10 match. Certain transplant centers now require high-resolution matching, which looks more deeply into tissue types and allow more specific HLA matching.

There are thousands of different combinations of possible HLA tissue types. This can make it hard to find an exact match. HLA antigens are inherited from both parents. If possible, the search for a donor usually starts with the patients brothers and sisters (siblings), who have the same parents as the patient. The chance that any one sibling would be a perfect match (that is, that you both received the same set of HLA antigens from each of your parents) is 1 out of 4.

If a sibling is not a good match, the search could then move on to relatives who are less likely to be a good match parents, half siblings, and extended family, such as aunts, uncles, or cousins. (Spouses are no more likely to be good matches than other people who are not related.) If no relatives are found to be a close match, the transplant team will widen the search to the general public.

As unlikely as it seems, its possible to find a good match with a stranger. To help with this process, the team will use transplant registries, like those listed here. Registries serve as matchmakers between patients and volunteer donors. They can search for and access millions of possible donors and hundreds of thousands of cord blood units.

Be the Match (formerly the National Marrow Donor Program)Toll-free number: 1-800-MARROW-2 (1-800-627-7692)Website: http://www.bethematch.org

Blood & Marrow Transplant Information NetworkToll-free number: 1-888-597-7674Website: http://www.bmtinfonet.org

Depending on a persons tissue typing, several other international registries also are available. Sometimes the best matches are found in people with a similar racial or ethnic background. When compared to other ethnic groups, white people have a better chance of finding a perfect match for stem cell transplant among unrelated donors. This is because ethnic groups have differing HLA types, and in the past there was less diversity in donor registries, or fewer non-White donors. However, the chances of finding an unrelated donor match improve each year, as more volunteers become aware of registries and sign up for them.

Finding an unrelated donor can take months, though cord blood may be a little faster. A single match can require going through millions of records. Also, now that transplant centers are more often using high-resolution tests, matching is becoming more complex. Perfect 10 out of 10 matches at that level are much harder to find. But transplant teams are also getting better at figuring out what kinds of mismatches can be tolerated in which particular situations that is, which mismatched antigens are less likely to affect transplant success and survival.

Keep in mind that there are stages to this process there may be several matches that look promising but dont work out as hoped. The team and registry will keep looking for the best possible match for you. If your team finds an adult donor through a transplant registry, the registry will contact the donor to set up the final testing and donation. If your team finds matching cord blood, the registry will have the cord blood sent to your transplant center.

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December 1st, 2022

Getting a Stem Cell or Bone Marrow Transplant – American Cancer Society

By daniellenierenberg

When the decision is made to have a stem cell or bone marrow transplant, there are several steps in theprocess. The steps are much the same, no matter what type of transplant youre going to have.

You will first be evaluated to find out if you are eligible for a transplant. A transplant is very hard on your body. For many people, transplants can mean a cure, but for some people, problems can lead to severe complications or even death. Youll want to weigh the benefits and risks before you start.

Transplants can also be hard emotionally. They often require being in the hospital, being isolated, and theres a high risk of side effects. Many of the effects are short-term, but some problems can go on for years. This can mean changes in the way you live your life. For some people its just for a while, but for others, the changes may be lifelong. Some of the side effects are really unpleasant and can be serious. Your cancer care team will do everything they can to make you comfortable, but some of the side effects may not be completely controlled or relieved.

Before you have a transplant, you need to discuss the transplant process and all its effects with your doctors. It also helps to talk with others who have already had transplants.

Its also very hard going through weeks and months of not knowing how your transplant will turn out. This takes a lot of time and emotional energy from the patient, caregivers, and loved ones. Its very important to have the support of those close to you. For example, youll need a responsible adult who will be with you to give you medicines, help watch for problems, and stay in touch with your transplant team after you go home. Your transplant team will help you and your caregiver learn what you need to know. The team can also help you and your loved ones work through the ups and downs as you prepare for and go through the transplant.

Many different medical tests will be done, and questions will be asked to try to find out how well you can handle the transplant process. These might include:

You will also talk about your health insurance coverage and related costs that you might have to pay.

You may have a central venous catheter (CVC) put into a large vein in your chest. This is most often done as outpatient surgery, and usually only local anesthesia is needed (the place where the catheter goes in is made numb). Nurses will use the catheter to draw blood and give you medicines.

If youre getting an autologous transplant, a special catheter can be placed that can also be used when your stem cells are being removed or harvested.

The CVC will stay in during your treatment and for some time afterward, usually until your transplanted stem cells have engrafted and your blood counts are on a steady climb to normal.

Younger people, people who are in the early stages of disease, or those who have not already had a lot of treatment, often do better with transplants. Some transplant centers set age limits. Some people also may not be eligible for transplant if they have other major health problems, such as serious heart, lung, liver, or kidney disease. A mini-transplant, described under Allogeneic stem cell transplant in Types of Stem Cell Transplants for Cancer Treatment may be an option for some of these people.

The hospitals transplant team will decide if you need to be in the hospital to have your transplant, if it will be done in an outpatient center, or if you will be in the hospital just for parts of it. If you have to be in the hospital, you will probably go in the day before pre-transplant chemo or radiation treatment begins (see the next section), the transplant team makes sure you and your family understand the process and want to go forward with it.

If you will be having all or part of your transplant as an outpatient, youll need to be very near the transplant center during the early stages. Youll need a family member or loved one to be a caregiver who can stay with you all the time. You and the caregiver will also need reliable transportation to and from the clinic. The transplant team will be watching you closely for complications, so expect to be at the clinic every day for a few weeks. You may still need to be in the hospital if your situation changes or if you start having complications.

To reduce the chance of infection during treatment, patients who are in the hospital are put in private rooms that have special air filters. The room may also have a protective barrier to separate it from other rooms and hallways. Some have an air pressure system that makes sure no unclean outside air gets into the room. If youre going to be treated as an outpatient, you will get instructions on avoiding infection. Usually, people who have transplants are in a separate, special part of the hospital to keep as many germs away as possible.

The transplant experience can be overwhelming. Your transplant team will be there to help you prepare for the process physically and emotionally and to discuss your needs. Every effort will be made to answer questions so you and your family fully understand what will be happening to you as you go through transplant.

Its important for you and your family to know what to expect, because once conditioning treatment begins (see the next section), theres no going back there can be serious problems if treatment is stopped at any time during transplant.

Having a transplant takes a serious commitment from you and your caregiver and family, so it is important to know exactly what to expect.

Conditioning, also known as pre-transplant treatment,bone marrow preparation, or myeloablation, is usually treatment with high-dose chemo and/or radiation therapy. Its the first step in the transplant process and typically takes a week or two. Its done for one or more of these reasons:

The conditioning treatment is different for every transplant. Your treatment will be planned based on the type of cancer you have, the type of transplant, and any chemo or radiation therapy youve had in the past.

If chemo is part of your treatment plan, it will be given in your central venous catheter and/or as pills. If radiation therapy is planned, its given to the entire body (called total body irradiation or TBI). TBI may be given in a single treatment session or in divided doses over a few days.

This phase of the transplant can be very uncomfortable because very high treatment doses are used. Chemo and radiation side effects can make you sick, and it may take you months to fully recover. A very common problem is mouth sores that will need to be treated with strong pain medicines. You may also have nausea, vomiting, be unable to eat, lose your hair, and have lung or breathing problems.

Conditioning can also cause premature menopause in women and often makes people sterile (unable to have children). (See Stem Cell Transplant Side Effects.)

After the conditioning treatment, youll be given a couple of days to rest before getting the stem cells. They will be given through your central venous catheter, much like a blood transfusion. If the stem cells were frozen, you might get some drugs before the stem cells are given. These drugs are used to help reduce your risk of reacting to the preservatives that are used when freezing the cells.

If the stem cells were frozen, they are thawed in warm water then given right away. There may be more than 1 bag of stem cells. For allogeneic or syngeneic transplants, the donor cells may be harvested (removed) in an operating room, and then processed in the lab right away. Once they are ready, the cells are brought in and given to you theyre not frozen. The length of time it takes to get all the stem cells depends on how much fluid the stem cells are in.

You will be awake for this process, and it doesnt hurt. This is a big step and often has great meaning for patientsand their families. Many people consider this their rebirth or chance at a second life. They may celebrate this day as they would their actual birthday.

Side effects from the infusion are rare and usually mild. The preserving agent used when freezing the stem cells causes many of the side effects. For instance, you might have a strong taste of garlic or creamed corn in your mouth. Sucking on candy or sipping flavored drinks during and after the infusion can help with the taste. Your body will also smell like this. The smell may bother those around you, but you might not even notice it. The smell, along with the taste, may last for a few days, but slowly fades away. Often having cut up oranges in the room will offset the odor. Patients who have transplants from cells that were not frozen do not have this problem because the cells are not mixed with the preserving agent.

Other side effects you might have during and right after the stem cell infusion include:

Again, side effects are rare and usually mild. If they do happen, they are treated as needed. The stem cell infusion must always be completed.

The recovery stage begins after the stem cell infusion. During this time, you and your family wait for the cells to engraft, or take, after which they start to multiply and make new blood cells. The time it takes to start seeing a steady return to normal blood counts varies depending on the patient and the transplant type, but its usually about 2 to 6 weeks. Youll be in the hospital or visit the transplant center daily for a number of weeks.

During the first couple of weeks youll have low numbers of red and white blood cells and platelets. Right after transplant, when your counts are the lowest, you may be given antibiotics to help keep you from getting infections. You may get a combination of anti-bacterial, anti-fungal, and anti-viral drugs. These are usually given until your white blood cell count reaches a certain level. Still, you can have problems, such as infection from too few white blood cells (neutropenia), or bleeding from too few platelets (thrombocytopenia). Many patients have high fevers and need IV antibiotics to treat serious infections. Transfusions of red blood cells and platelets are often needed until the bone marrow starts working and new blood cells are being made by the infused stem cells.

Except for graft-versus-host disease, which only happens with allogeneic transplants, the side effects from autologous, allogeneic, and syngeneic stem cell transplants are much the same. Problems may include stomach, heart, lung, liver, or kidney problems. (Stem Cell Transplant Side Effects goes into the details.) You might also go through feelings of distress, anxiety, depression, joy, or anger. Adjusting emotionally after the stem cells can be hard because of the length of time you feel ill and isolated from others.

You might feel as if you are on an emotional roller coaster during this time. Support and encouragement from family, friends, and the transplant team are very important to get you through the challenges after transplant.

The discharge process actually begins weeks before your transplant. It starts with the transplant team teaching you and your primary (main) caregiver about:

For the most part, transplant centers dont send patients home until they meet the following criteria:

(Why Are Stem Cell Transplants Used as Cancer Treatment? has more information about neutrophils, platelets, and hematocrit).

If you do not meet all of these requirements, but still dont need the intensive care of the transplant unit, you might be moved to another oncology unit. When you do go home, you might need to stay near the transplant center for some time, depending on your condition.

The process of stem cell transplant doesnt end when you go home. Youll feel tired, and some people have physical or mental health problems in the rehabilitation period. You might still be taking a lot of medicines. These ongoing needs must now be managed at home, so caregiver and friend/family support is very important.

Transplant patients are followed closely during rehab. You might need daily or weekly exams along with things like blood tests, and maybe other tests, too. During early rehab, you also might need blood and platelet transfusions, antibiotics, or other treatments. At first youll need to see your transplant team often, maybe even every day, but youll progress to less frequent visits if things are going well. It can take 6 to 12 months, or even longer, for blood counts to get close to normal and your immune system to work well. During this time, your team will still be closely watching you.

Some problems might show up as much as a year or more after the stem cells were infused. They can include:

Other problems can also come up, such as:

Your transplant team is still there to help you, even though the transplant happened months ago. Its important that you tell them about any problems you are having they can help you get the support you need to manage the changes that you are going through. They can also help you know if problems are serious, or a normal part of recovery. The National Bone Marrow Transplant Link helps patients, caregivers, and families by providing information and support services before, during, and after transplant. They can be reached at 1-800-LINK-BMT (1-800-546-5268) or online at http://www.nbmtlink.org.

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December 1st, 2022

Woman, 41, With Bubbles In Her Urine Dismissed By Doctors. Turns Out To Have The Blood Cancer Multiple Myeloma. – SurvivorNet

By daniellenierenberg

Woman, 41, With Bubbles In Her Urine Dismissed By Doctors. Turns Out To Have The Blood Cancer Multiple Myeloma. SurvivorNet

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Woman, 41, With Bubbles In Her Urine Dismissed By Doctors. Turns Out To Have The Blood Cancer Multiple Myeloma. - SurvivorNet

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December 1st, 2022

Stem cell and bone marrow transplants – Cancer Research UK

By daniellenierenberg

Stem cell or bone marrow transplant is a way of giving very high dose chemotherapy. This treatment aimsto cure some types of cancer, including Hodgkin lymphoma.

Bone marrow is a spongy material that fills the bones.

It contains early blood cells, called stem cells. These develop into the 3 different types of blood cell.

You have a stem cell or bone marrow transplant after very high doses of chemotherapy. The chemotherapy has a good chance of killing the cancer cells but also kills the stem cells in your bone marrow.

Before your high dose chemotherapy, your team collects your stem cells or bone marrow. Or they collect adonor's stem cells or bone marrow. After the treatment you have the cells into a vein through a drip. The cells find their way back to your bone marrow. Then you can make the blood cells you need again.

You might have this intensive treatment if your Hodgkin lymphoma comes back after the first course of treatment. It can get rid of the lymphoma again for many people. Your doctor might also suggest this treatment if your Hodgkin lymphoma has not responded to the standard treatment.

You might have a course of high dose BEAM or LEAM chemotherapy. Then most people with Hodgkin lymphoma have their own stem cells or bone marrowback after the high dose treatment. This is called an autologous transplant.

You're now more likely to have a stem cell transplant (also called peripheral blood stem cell transplant) than a bone marrow transplant.

This is because:

You have injections of growth factors before, and sometimes after, the stem cell transplant. Growth factors are natural proteins that make the bone marrow produce blood cells.

You have daily injections of growth factor for between 5 and 10 days. Sometimes you might have low doses of chemotherapy with the growth factor injections.

After your growth factor injections, you have blood tests every day to see if there are enough stem cells in your bloodstream. When there are enough cells, you have them collected. This is called harvesting. Collecting the stem cells takes 3 or 4 hours. You are awake during this process. You lie down on a couch. Your nurse puts a drip into each of your arms and attaches it to a machine.

Your blood passes out of one drip. It goes through the machine and back into your body through the other drip. The machine filters the stem cells out of your blood. They are collected and frozen until after your high dose treatment.

You mayneed to go back the following day for a second harvest if they don'thaveenough cells from the first collection.

You might feel very tired after having your stem cell collection.

You might have:

This happens if your calcium level gets low during your collection. Your nurses will give you extra calcium through a drip if this happens.

You have your bone marrow taken (bone marrow harvest) under a general anaesthetic. This means you are asleep and can't feel anything during the procedure.

You lie on your side on a couch. Your doctor puts a needle through your skin into the hip bone (pelvis). The doctor gently draws out the bone marrow through the needle into a syringe. To get enough bone marrow the doctor needs to put the needle into several parts of the pelvis. You have about 2 pints (1 litre) of bone marrow taken out and then it's frozen until it's needed.

You might have a stem cell or bone marrow transplant using cells from a donor. This is called an allogeneic transplant. The cells need to be as similar as possible to yours.

So these can be from:

Youmight have bone marrow from a donor if:

Doctors are still learning how best to use allogeneic transplants for Hodgkin lymphoma.

The side effects of having a stem cell or bone marrow transplantare caused by high dose chemotherapy.

The main side effectsinclude:

You can call the Cancer Research UK nurses to talk about any worries you might have about having a transplant. The number is freephone 0808 800 4040, and the lines are open Monday to Friday, 9am to 5pm.

Visit link:
Stem cell and bone marrow transplants - Cancer Research UK

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November 22nd, 2022

Donating Bone Marrow Experience | Be The Match

By daniellenierenberg

Bone marrow donation is one of two methods of collecting blood forming cells for bone marrow transplants. Bone marrow donation is a surgical procedure that takes place in a hospital operating room. Doctors use needles to withdraw liquid marrow (where the bodys blood-forming cells are made) from both sides of the back of your pelvic bone. You will be given anesthesia and feel no pain during the donation. After donation, your liquid marrow is transported to the patients location for transplant.

Typically, the hospital stay for marrow donation is from early morning to late afternoon, or occasionally overnight for observation. The donation will take place in a hospital that is experienced and participates in marrow collections for Be The Match.

Common side effects of marrow donation reported 2 days after donation: Back or hip pain 84%, Fatigue 61%, Throat pain 32%, Muscle pain 24%, Insomnia 15%, Headache 14%, Dizziness 10%, Loss of appetite 10%, Nausea 9%.

The median time to full recovery for a marrow donation is 20 days. Recovery after marrow donation: 5% - 2 days, 18%-7 days, 71%-30 days, 97%-180 days, 99%-1 year

Learn more about what happensafter you donate.

Continue reading here:
Donating Bone Marrow Experience | Be The Match

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November 22nd, 2022

Learn How to Donate Bone Marrow | Be The Match

By daniellenierenberg

Join Be The Match Registry

The first step to being someone's cure is to join Be The Match Registry. If you are between the ages of 18-40, committed to donating to any patient in need, and meet the health guidelines, there are two ways to join.

Join in-person at a donor registry drive in your community.Be The One to Save a Life

Find a donor registry drive

Or join online today:

Join online

If you are between the ages of 18 and 35 patients especially need you. Research shows that cells from younger donors lead to more successful transplants. Doctors request donors in the 18-35 age group nearly 75% of the time.

Under 18 years old? Click here to sign up for the Under 18 Pre-Registry. You will receive information about ways to stay involved with our life-saving mission and a reminder to join when you're eligible.

There are many other ways you can be the cure for patients with blood cancers.

Check outFAQs about donationor call us at 1 (800) MARROW2 for more information about bone marrow donation.

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October 29th, 2022

Stem Cell Transplantation Program – DanaFarber Cancer Institute

By daniellenierenberg

Stem cell/bone marrow transplant offers some patients with blood cancers and blood disorders the possibility of a cure, and others a longer period of disease-free survival. Founded in 1972, our Adult Stem Cell Transplant Program is one of the largest and most experienced in the world.

Our stem cell/bone marrow transplant program performs approximately 500 transplants each year and has performed more than 11,180 transplants in the programs history. This includes more than 5,500 allogeneic transplants and more than 5,100 autologous transplants. This experience makes a difference for our patients.

Our patients' outcomes regularly exceed expected outcomes as established by the Center for International Blood and Marrow Transplant Research, which reports and analyzes outcomes for recipients of allogeneic hematopoietic stem cell transplant. In the most recent report (2020), only 10% of centers achieved this outcome level. Dana-Farber Brigham Cancer Center was the largest center to achieve this outcome.

Stem cell/bone marrow transplant can be an effective treatment for a variety of hematologic malignancies, bone marrow failure syndromes, and rare and congenital blood disorders. We are experienced in stem cell transplant for a variety of hematologic malignancies, bone marrow failure syndromes, and rare and congenital blood disorders. This includes:

We perform both autologous and allogeneic stem cell/bone marrow transplants.

For allogeneic patients (i.e., those requiring donor stem cells), we offer:

Reduced-intensity transplants use lower doses of chemotherapy and have been a major factor in extending stem cell/bone marrow transplants for older adults up into their 70s. Our program has transplanted more than 5,000 patients over 55 years old. Our Older Adult Hematologic Malignancies Program provides dedicated support for older patients.

From exceptional medical care to support with housing and other logistics, we offer many services to international patients:

Learn more about international referrals and services.

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Stem Cell Transplantation Program - DanaFarber Cancer Institute

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October 29th, 2022

A CRISPR Alternative for Correcting Mutations That Sensitize Cells to DNA Damage – The Scientist

By daniellenierenberg

Fanconi anemia is a rare genetic disease in which essential DNA repair pathway genes are mutated, disrupting the DNA damage response. Patients with Fanconi anemia experience hematological complications, including bone marrow failure, and are predisposed to cancer. The only curative therapy for the hematological symptoms of Fanconi anemia is an allogeneic hematopoietic stem cell transplant, in which a patient receives healthy stem cells from a donor. While this may cure or prevent some of the diseases complications, stem cell transplantation can cause additional difficulties, including graft-versus-host disease (GvHD) and exacerbated cancer risk.1

There is growing interest in applying genome editing technologies like CRISPR-Cas9 to correct Fanconi anemia mutations in patient-derived cells for autologous transplants, in which corrected stem cells are given back to the patient. However, this disease poses a unique challenge: How do you apply a genome editing technique in cells that are particularly sensitive to DNA damage? Fanconi anemia cells cannot resolve the double-strand breaks that conventional CRISPR-Cas9 gene editing creates in the target DNA, which prevents researchers from effectively correcting disease-causing mutations with this method.

In a study published in International Journal of Molecular Science, a research team at the University of Minnesota led by Branden Moriarity and Beau Webber used Cas9-based tools called base editors (BEs) to edit genes in Fanconi anemia patient-derived cells without inducing double-strand DNA damage.2 BEs are fusion proteins made of a Cas9 enzyme that cleaves target DNA (nCas9) and a deaminase that converts cytidine to uridine (cytosine base editor, CBE) or adenosine to inosine (adenosine base editor, ABE). During DNA replication or repair, sites targeted by a BE are rewritten as thymine in the case of CBEs, or guanine with ABEs.

Although base editors do not induce double-strand breaks, they still nick the DNA and trigger a DNA repair response. Because of this, the researchers first examined if CBEs and ABEs would work on non-Fanconi anemia genes in patient-derived cells. There was that mystery, you know, because [Fanconi anemia patient cells are] DNA repair deficient. So we weren't surewe thought maybe it would work, but not as well as a normal cell. But indeed, it works on the same level, basically. So that was pretty exciting, Moriarity explained.

The research team then demonstrated that CBEs and ABEs can correct Fanconi anemia-causing mutations in the FANCA gene in primary patient fibroblast and lymphoblastoid cell lines. Base editing restored FANCA protein expression and improved the ability of the patient-derived cells to grow in the presence of a DNA damaging chemical. Additionally, in culture, fibroblasts with corrected FANCA mutations outgrew cells in which the base editing failed. Finally, the researchers assessed if BEs could correct mutations in different Fanconi anemia genes. Using an algorithm, they predicted that most Fanconi anemia mutations were correctable either by BEs or by another nCas9-fusion technology called prime editing (PE), which is capable of large genetic insertions and deletions.

This work comes on the heels of a preprint from another research group at The Centre for Energy, Environmental and Technological Research and ETH Zurich, who investigated ABEs in patient blood cell lines. This group also effectively targeted Fanconi anemia genes with BE technology, and their investigation went one step further: they corrected mutations in patient-derived hematopoietic stem cells.3This was something that Moriarity and Webber were unable to dobecause the disease is a bone marrow failure syndrome, these cells are scarce. Basically, these patients do not have stem cells, explains Annarita Miccio, a senior researcher and lab director at Institute Imagine of Paris Cit University, who was not involved in either study. These are very challenging experiments, and more than the experiments, the challenge of [treating] Fanconi anemia is exactly thatthe number of cells.

Despite this challenge, the researchers have laid the groundwork for genome editing as a treatment approach in Fanconi anemia, without the need for double-strand DNA breaks. I think the study we did is a good, solid proof of concept, and sets the stage for the next steps, but certainly, it's not the end of the story, said Webber.

References

Follow this link:
A CRISPR Alternative for Correcting Mutations That Sensitize Cells to DNA Damage - The Scientist

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October 13th, 2022

Stem Cell Manufacturing Global Market Report 2022: Widespread Product Utilization in Effective Disease Management, Personalized Medicine, and Genome…

By daniellenierenberg

Company Logo

Global Stem Cell Manufacturing Market

Dublin, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The "Stem Cell Manufacturing Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.

The global stem cell manufacturing market size reached US$ 11.2 Billion in 2021. Looking forward, the publisher expects the market to reach US$ 18.59 Billion by 2027, exhibiting a CAGR of 8.81% during 2021-2027.

Stem cells are undifferentiated or partially differentiated cells that make up the tissues and organs of animals and plants. They are commonly sourced from blood, bone marrow, umbilical cord, embryo, and placenta. Under the right body and laboratory conditions, stem cells can divide to form more cells, such as red blood cells (RBCs), platelets, and white blood cells, which generate specialized functions.

They are widely used for human disease modeling, drug discovery, development of cell therapies for untreatable diseases, gene therapy, and tissue engineering. Stem cells are cryopreserved to maintain their viability and minimize genetic change and are consequently used later to replace damaged organs and tissues and treat various diseases.

Stem Cell Manufacturing Market Trends:

The global market is primarily driven by the increasing venture capital (VC) investments in stem cell research due to the rising awareness about the therapeutic potency of stem cells. Apart from this, the widespread product utilization in effective disease management, personalized medicine, and genome testing applications are favoring the market growth. Additionally, the incorporation of three-dimensional (3D) printing and microfluidic technologies to reduce production time and lower cost by integrating multiple production steps into one device is providing an impetus to the market growth.

Furthermore, the increasing product utilization in the pharmaceutical industry for manufacturing hematopoietic stem cells (HSC)- and mesenchymal stem cells (MSC)-based drugs for treating tumors, leukemia, and lymphoma is acting as another growth-inducing factor.

Story continues

Moreover, the increasing product application in research applications to produce new drugs that assist in improving functions and altering the progress of diseases is providing a considerable boost to the market. Other factors, including the increasing usage of the technique in tissue and organ replacement therapies, significant improvements in medical infrastructure, and the implementation of various government initiatives promoting public health, are anticipated to drive the market.

Key Players

Anterogen Co. Ltd.

Becton Dickinson and Company

Bio-Rad Laboratories Inc.

Bio-Techne Corporation

Corning Incorporated

FUJIFILM Holdings Corporation

Lonza Group AG

Merck KGaA

Sartorius AG

Takara Bio Inc.

Thermo Fisher Scientific Inc.

Key Questions Answered in This Report:

How has the global stem cell manufacturing market performed so far and how will it perform in the coming years?

What has been the impact of COVID-19 on the global stem cell manufacturing market?

What are the key regional markets?

What is the breakup of the market based on the product?

What is the breakup of the market based on the application?

What is the breakup of the market based on the end user?

What are the various stages in the value chain of the industry?

What are the key driving factors and challenges in the industry?

What is the structure of the global stem cell manufacturing market and who are the key players?

What is the degree of competition in the industry?

Key Market Segmentation

Breakup by Product:

Consumables

Culture Media

Others

Instruments

Bioreactors and Incubators

Cell Sorters

Others

Stem Cell Lines

Hematopoietic Stem Cells (HSC)

Mesenchymal Stem Cells (MSC)

Induced Pluripotent Stem Cells (iPSC)

Embryonic Stem Cells (ESC)

Neural Stem Cells (NSC)

Multipotent Adult Progenitor Stem Cells

Breakup by Application:

Research Applications

Life Science Research

Drug Discovery and Development

Clinical Application

Allogenic Stem Cell Therapy

Autologous Stem Cell Therapy

Cell and Tissue Banking Applications

Breakup by End User:

Pharmaceutical & Biotechnology Companies

Academic Institutes, Research Laboratories and Contract Research Organizations

Hospitals and Surgical Centers

Cell and Tissue banks

Others

Breakup by Region:

North America

United States

Canada

Asia-Pacific

China

Japan

India

South Korea

Australia

Indonesia

Others

Europe

Germany

France

United Kingdom

Italy

Spain

Russia

Others

Latin America

Brazil

Mexico

Others

Middle East and Africa

Key Topics Covered:

1 Preface

2 Scope and Methodology

3 Executive Summary

4 Introduction

5 Global Stem Cell Manufacturing Market

6 Market Breakup by Product

7 Market Breakup by Application

8 Market Breakup by End User

9 Market Breakup by Region

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Stem Cell Manufacturing Global Market Report 2022: Widespread Product Utilization in Effective Disease Management, Personalized Medicine, and Genome...

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October 13th, 2022

Stem Cell Manufacturing Global Market Report 2022: Widespread Product Utilization in Effective Disease Ma – Benzinga

By daniellenierenberg

Stem Cell Manufacturing Market Trends:

Key Players

Key Questions Answered in This Report:

Key Market Segmentation

Breakup by Product:

Breakup by Application:

Breakup by End User:

Breakup by Region:

Key Topics Covered:

1 Preface

2 Scope and Methodology

3 Executive Summary

4 Introduction

5 Global Stem Cell Manufacturing Market

6 Market Breakup by Product

7 Market Breakup by Application

8 Market Breakup by End User

9 Market Breakup by Region

10 SWOT Analysis

11 Value Chain Analysis

12 Porters Five Forces Analysis

13 Price Analysis

14 Competitive Landscape

For more information about this report visit https://www.researchandmarkets.com/r/5iujo7

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Stem Cell Manufacturing Global Market Report 2022: Widespread Product Utilization in Effective Disease Ma - Benzinga

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October 13th, 2022

Gene therapy approvals now at four with treatments for inherited anemia and degenerative brain condition but costs are stratospheric. Why? – Genetic…

By daniellenierenberg

The FDA recently approved two gene therapies with hefty price tags, the first for an inherited anemia and the second for a degenerative brain condition. The two new treatments, from bluebirdbio, double the number of gene therapies on the market.

Most biotechnologies evolve over three decades or so, but the idea of gene therapy has been around since the late 1950s, blooming soon after Watson and Crick solved the structure of DNA. When my book The Forever Fix: Gene Therapy and the Boy Who Saved Itwas published a decade ago, it would still be 5 years before the first approval. That treatment, the subject of my book, enabled the blind to see, sometimes in just days.

Why has the pace of gene therapy been so slow? Cost is one barrier. Other concerns are the degree to which a gene therapy actually helps, how long the effect lasts, and what proportion of patients respond.

FDAs gene therapy roster ishere, but a caveat is necessary.

The list lumps gene therapy in with cell therapy, inviting unintentional hype from media folks unfamiliar with the science. Most entries actually refer to using stem cells to treat blood cancers and related conditions. An example: cartilage cells are sampled from a person with abum knee, mass-produced in a dish, and then injected into the knee, where they fuel production of more cartilage.

My favorite example of not-really-gene-therapy on the FDAs list targetsfacial wrinkles, also using patients lab-expanded cells: 18 million fibroblasts injected three times churn out collagen, filling in the offending skin craters.

Buried in the FDAs list are the first twoactualgene therapy approvals.Luxturna(Spark Therapeutics) treats RPE65 mutation-associated retinal dystrophy and has restored vision in many patients since its approval at the end of 2017. The second approved gene therapy, in 2019, isZolgensma, to treat spinal muscular atrophy, from Novartis Gene Therapies.

FDA approvedZynteglo on August 17, aka betibeglogene autotemcel or eli-cel. It treats the blood disorder beta thalassemia, which causes weakness, dizziness, fatigue, and bone problems. People with severe cases need transfusions of red blood cells every two to five weeks, which can lead to dangerous buildup of iron.

Zynteglo is a one-time infusion of stem cells descended from a patients bone marrow in which functional beta globin genes have been introduced aboard lentiviruses disabled HIV. The $2.8 million treatment is approved for adults and children.

Two clinical trials enrolled 91 patients, 36 of whom improved enough to no longer need transfusions. Bluebird estimates that 1,300 to 1,500 people in the U.S. may be candidates for Zynteglo.

The second go-ahead is forSkysona, approved September 16 for early active cerebral adrenoleukodystropy (CALD). The condition destroys the protective myelin sheath around brain neurons.

A stem cell transplant can cure CALD. Skysona is for the 700 or so boys aged 4 to 17 who cant find matched donors. Nearly fifty percent of them die within five years of symptom onset.

But like many gene therapies, Skysona isnt a magic bullet. In the two ongoing clinical trials, the metric for assessing improvement is slowing neurologic decline, tracking major functional disabilities. These include loss of communication skills, vision, and of voluntary movement, which impairs mobility, eating, and urinary retention.

The 2-year study that led to the FDA approval followed boys with mild or no symptoms, diagnosis possible early due to newborn screening in many states. Those who received Skysona had a 72% likelihood of survival over the two years without developing new major functional disabilities, compared to 43% among untreated boys. The trial will follow participants for 15 years. Since many states are nowscreening newborns for ALD, perhaps boys destined to develop symptoms can receive Skysona before that if someone will pick up the $3 million tab per patient.

Gene therapy companies have long justified high costs with the expense of the bench-to-bedside trajectory. So I was surprised to see a new study published inJAMA Network Open, Association of Research and Development Investments With Treatment Costs for New Drugs Approved From 2009 to 2018, finding none. The authors admonish companies to make further data available to support their claims that high drug prices are needed to recover research and development investments, if they are to continue to use this argument to justify high prices.

Becausethe paperuses terms like first-in-class, accelerated approval, breakthrough therapy, orphan, and priority review language Ive often seen attached to descriptions of gene therapy I assumed it would include Luxturna, which costs $850,000 for both eyes. But the new report omits drug names, instead citing a2020 paperfrom the team that did.No Luxturna. Thats probably because the researchers evaluated R&D costs only for products with publicly available data thats 63 drugs, a mere fifth of new approvals. The new report, of course sent out in news release form to the media, provides more a glimpse than a revelation.

So perhaps gene therapy is an exception for which high prices are indeed required to recoup investment. A viral vector to deliver DNA can cost $500,000 or more to produce, let alone engineer and develop.

Companies also use the one-and-done strategy to justify high prices. The homepage of bluebird bios website, for example, proclaims were pursuing curative gene therapies, although the data on Skysona for CALD indicate incremental change.Axios reports on how Medicaid, private insurers, and companies will help address cost concerns.

While bluebird bio bats around the c word cure it also introduces a long-needed granularity to the terminology. The company has replaced gene therapy with the more accurate gene addition therapy. Thats what the four approved gene therapies actually do add working copies of genes, not fixing them in place. Gene therapy is a little like patching a flat tire, not replacing it.

But the next stage of the evolving technology will in fact befixing genes, courtesy of gene and genome editing. This more precise strategy circumvents the problem of a piece of DNA inserting willy-nilly into a chromosome, perhaps disrupting a cancer-causing gene.

Gene editing with CRISPR has now been around for a decade. The components of the toolkit have been refined to minimize so-called off-target effects that can harpoon unintended genes.

A team atSt. Jude Childrens Research Hospitalhas developed what hematologist Yong Cheng terms the Google Maps of editing the genome. We provide a new approach to identify places to safely integrate a gene cassette. We created step-by-step directions to find safe harbor sites in specific tissues. The recipe is published inGenome Biologyand the tool availablehere.

The approach is seemingly simple. Using data from the 1000 Genomes Project, the tool identifies parts of the genome that often bear inserted or deleted DNA sequences among healthy people (and therefore are harmless) and are highly variable. These are the places where unwound DNA loops about itself when replicating just before a cell divides, and could tolerate a healing gene harpoon going astray.

Safe gene therapy requires two things. Number one, maintaining high expression of the new gene. And number two, the integration needs to have minimal effects on the normal human genome, Cheng said.

Gene addition therapy and gene/genome editing are slowly taking their places among other weapons against genetic disease. These include antisense treatments that glom onto mutant genes, small molecule-based drugs, repurposing existing drugs, supplements, and perhaps most important, the therapies that impact life on a daily basis. And so the toolbox expands to tackle the errors in our genes.

Ricki Lewis has a PhD in genetics and is a science writer and author of several human genetics books.She is an adjunct professor for the Alden March Bioethics Institute at Albany Medical College.Follow her at herwebsiteor Twitter@rickilewis

A version of this article originally appeared at PLOS and is reposted here with permission. Find PLOS on Twitter @PLOS

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Gene therapy approvals now at four with treatments for inherited anemia and degenerative brain condition but costs are stratospheric. Why? - Genetic...

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October 13th, 2022

Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene…

By daniellenierenberg

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces data presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Edinburgh, United Kingdom, taking place October 11-14, 2022. Presentations will include clinical data from Rockets lentiviral vector (LV)-based gene therapy programs for Leukocyte Adhesion Deficiency-I (LAD-I), Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD). Donald B. Kohn, MD, Distinguished Professor of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Pharmacology at University of California, Los Angeles (UCLA) and Director of the UCLA Human Gene and Cell Therapy Program, will also give an Invited Talk incorporating previously disclosed data from the RP-L201 trial for LAD-I.

Positive Updated Safety and Efficacy Data from Phase 2 Pivotal Trial for Fanconi Anemia (FA)

The poster and presentation include updated safety and efficacy data from the Phase 2 pivotal trial of RP-L102, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of FA.

Positive Top-line Clinical Data from Phase 2 Pivotal Trial for Severe Leukocyte Adhesion Deficiency-I (LAD-I)

The oral presentation includes previously disclosed efficacy and safety data at three to 24 months of follow-up after RP-L201 infusion for all patients and overall survival data for seven patients at 12 months or longer after infusion. RP-L201 is Rockets ex-vivo lentiviral gene therapy candidate for the treatment of severe LAD-I.

Interim Data from Ongoing Phase 1 Trial for Pyruvate Kinase Deficiency (PKD)

The poster and presentation include previously disclosed safety and efficacy data from the Phase 1 trial of RP-L301, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of PKD.

Details for Rockets Invited Talk and poster presentations are as follows:

Title: Interim Results from an ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)Session: Clinical Trials (Plenary 2)Presenter: Donald B. Kohn, MD - University of California, Los Angeles, Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics, and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy ProgramSession date and time: Wednesday, 12 October at 11:10-13:15 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV20

Title: Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical TrialsSession: Poster Session 1Presenter: Julin Sevilla MD, PhD - Fundacin para la Investigacin Biomdica, Hospital Infantil Universitario Nio JessSession date and time: Wednesday, 12 October at 19:30-21:00 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P139

Title: Preliminary Conclusions of the Phase I/II Gene therapy Trial in Patients with Fanconi Anemia-ASession: Blood Diseases: Haematopoietic Cell DisordersPresenter: Juan Bueren, PhD - Unidad de Innovacin Biomdica, Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT)Session date and time: Thursday, 13 October at 15:30-17:30 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV41

Title: Interim Results from an Ongoing Global Phase 1 Study of Lentiviral-Mediated Gene Therapy for Pyruvate Kinase DeficiencySession: Poster Session 2Presenter: Jos Luis Lpez Lorenzo, MD, Hospital Universitario Fundacin Jimnez DazSession date and time: Thursday, 13 October at 17:30-19:15 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P128

Abstracts for the presentations can be found online at: https://www.esgct.eu/.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 4,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacin Sanitaria de la Fundacin Jimnez Daz (IIS-FJD).

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rockets future expectations, plans and prospects, including without limitation, Rockets expectations regarding its guidance for 2022 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Danon Disease, the expected timing and data readouts of Rockets ongoing and planned clinical trials, the expected timing and outcome of Rockets regulatory interactions and planned submissions, Rockets plans for the advancement of its Danon Disease program and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rockets ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rockets dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rockets Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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October 13th, 2022

Cellectis Presents Data on Two TALEN-based Gene Therapy Preclinical Programs for Patients with Sickle Cell Disease and Mucopolysaccharidosis type I at…

By daniellenierenberg

Cellectis Inc.

NEW YORK, Oct. 11, 2022 (GLOBE NEWSWIRE) -- Cellectis (the Company) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that the Company will present both an oral and poster at the European Society of Gene and Cell Therapys (ESGCT) 29th Congress, to be held in Edinburgh from October 11-14, 2022.

Arianna Moiani, Ph.D., Senior Scientist & Team Leader Innovation Gene Therapy, will give an oral presentation on encouraging pre-clinical data that leverages TALEN gene editing technology to develop a hematopoietic stem and progenitor cell (HSPCs)-based gene therapy to treat sickle cell disease.

Eduardo Seclen, Ph.D., Senior Scientist & Team Leader, Gene Editing, will present a poster illustrating a TALEN-based gene editing approach that reprograms HSPCs to secrete alpha-L-iduronidase (IDUA), a therapeutic enzyme missing in Mucopolysaccharidosis type I (MPS-I).

The pre-clinical data presented at ESGCT further demonstrate our ability to leverage TALEN gene editing technology to potentially address genetic diseases, namely, sickle cell disease and lysosomal storage diseases. By correcting a faulty mutation or inserting a corrected gene at the HSPC level, we aim to provide a lifelong supply of healthy cells in a single intervention, said Philippe Duchateau, Ph.D., Chief Scientific Officer at Cellectis. These new milestones bring us one step closer to our goal: providing a cure to patients that have failed to respond to standard therapy.

Presentation details

Pre-clinical data presentation on a non-viral DNA delivery associated with TALEN gene editing that leads to highly efficient correction of sickle cell mutation in long-term repopulating hematopoietic stem cells

Sickle cell disease stems from a single point mutation in the HBB gene which results in sickle hemoglobin.

Cellectis leveraged its TALEN technology to develop a gene editing process that leads to highly efficient HBB gene correction via homology directed repair, while mitigating potential risks associated to HBB gene knock-out. Overall, these results show that non-viral DNA delivery associated with TALEN gene editing reduces the toxicity usually observed with viral DNA delivery and allows high levels of HBB gene correction in long-term repopulating hematopoietic stem cells.

Story continues

The oral presentation titled Non-viral DNA delivery associated to TALEN gene editing leads to highly efficient correction of sickle cell mutation in long-term repopulating hematopoietic stem cells, will be made on Thursday, October 13th, 8:30AM-10:45AM BST by Arianna Moiani, Ph.D., Senior Scientist & Team Leader Innovation Gene Therapy. The presentation can be found on the Cellectis website on the day of the presentation.

Presentation details

Pre-clinical data presentation on TALEN-mediated engineering of HSPC that enables systemic delivery of IDUA

Mucopolysaccharidosis type I (MPS-I) is caused by deficiencies in the alpha-L-iduronidase (IDUA) gene and it is associated with severe morbidity representing a significant unmet medical need.

Cellectis established a TALEN-basedex vivogene editing protocol to insert an IDUA-expression cassette into a specific locus of HSPC.

Editing rates in vivo were 6-9% sixteen weeks after injection, depending on the tissue analyzed (blood, spleen, bone marrow). Lastly, 8.3% of human cells were edited in the brain compartment.

Cellectis established a safe TALEN-based gene editing protocol procuring IDUA-edited HSPCs able to engraft, differentiate into multiple lineages and reach multiple tissues, including the brain.

The poster presentation titled TALEN-mediated engineering of HSPC enables systemic delivery of IDUA, will be made on Thursday, October 13th, 5:30PM - 7:15PM BST by Eduardo Seclen, Ph.D., Senior Scientist & Team Leader, Gene Editing, and can be found on Cellectis website.

About Cellectis

Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. Cellectis utilizes an allogeneic approach for CAR-T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to make therapeutic gene editing in hemopoietic stem cells for various diseases. As a clinical-stage biopharmaceutical company with over 22 years of experience and expertise in gene editing, Cellectis is developing life-changing product candidates utilizing TALEN, its gene editing technology, and PulseAgile, its pioneering electroporation system to harness the power of the immune system in order to treat diseases with unmet medical needs. Cellectis headquarters are in Paris, France, with locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS).

For more information, visit http://www.cellectis.com. Follow Cellectis on social media: @cellectis, LinkedIn and YouTube.

For further information, please contact:

Media contacts:Pascalyne Wilson,Director,Communications,+33 (0)7 76 99 14 33, media@cellectis.comMargaret Gandolfo, Senior Manager, Communications, +1 (646) 628 0300

Investor Relation contact:Arthur Stril, Chief Business Officer, +1 (347) 809 5980, investors@cellectis.comAshley R. Robinson, LifeSci Advisors, +1 617430 7577

Forward-looking StatementsThis press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as anticipate, believe, intend, expect, plan, scheduled, could, may and will, or the negative of these and similar expressions. These forward-looking statements, which are based on our managements current expectations and assumptions and on information currently available to management. Forward-looking statements include statements about the potential of our preclinical programs and product candidates. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including with respect to the numerous risks associated with biopharmaceutical product candidate development. With respect to our cash runway, our operating plans, including product development plans, may change as a result of various factors, including factors currently unknown to us. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F and the financial report (including the management report) for the year ended December 31, 2021 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

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October 13th, 2022

Bone Marrow: What it is & Why it is Important – Cleveland Clinic

By daniellenierenberg

OverviewWhat is bone marrow?

Bone marrow is the soft, fatty tissue inside of bone cavities. Components of your blood including red and white blood cells and platelets form inside of your bone marrow.

Bone marrow makes nearly all the components of your blood. It's responsible for creating billions of red blood cells daily, along with white blood cells and platelets. Bone marrow also stores fat that turns into energy as needed.

Bone marrow makes the components of your blood that you need to survive. Bone marrow produces red blood cells that carry oxygen, white blood cells that prevent infection and platelets that control bleeding. The absence of bone marrow can be fatal since it's an essential part of your body.

Yes, bone marrow and the healthy cells it produces are necessary for humans to live. Often, cell mutations harm healthy bone marrow cells, and a bone marrow transplant would be a treatment option for people diagnosed with blood cancers like leukemia.

A bone marrow transplant takes healthy cells from a donor and puts them into your bloodstream. The donors cells help your body grow healthy red and white blood cells and platelets.

There are three parts to the anatomy of your bones: compact bone, spongy bone and bone marrow. Compact bone is the strong, outer layer of your bones. Spongy bone makes up the ends of your bones. Bone marrow is in the center of most bones and in the end of spongy bones in your body. Bone marrow and blood vessels fill cavities in your bones, where they store fat and stem cells and produce blood cells that make your whole blood.

Bone marrow is a spongy, soft tissue that resembles a jelly or jam that you would spread on toast. It comes in two colors, red and yellow. Bone marrow fills the cavities of your bones and holds cells that create red and white blood cells and platelets, which make whole blood. The color of red bone marrow is the result of red blood cell production.

There are two types of bone marrow in your body, which are characterized by their color. Your body holds just under 6 lbs. (about 2.5 kg.) of red and yellow bone marrow.

Red bone marrow makes up all of your bone marrow until about age seven. Yellow bone marrow gradually replaces red bone marrow as you age.

Bone marrow is made of stem cells. These stem cells make red bone marrow, which creates blood cells and platelets for your blood. Yellow bone marrow consists mostly of fat and stem cells that produce bone and cartilage in your body.

Directly targeting bone marrow is leukemia, which is a blood and bone marrow cancer. Leukemia forms when a cell mutation occurs in your bone marrow and mutated cells multiply out of control, reducing the production of healthy, normal cells.

Since bone marrow is the foundation for the creation of blood cells, blood-related conditions often are the result of abnormally functioning bone marrow. These conditions include:

Common symptoms of bone marrow conditions include:

There are two tests to check the health of your bone marrow and/or blood cells:

For a bone marrow test or donation, youll receive an anesthetic, so you won't feel any pain during the procedure. After the procedure, you may feel side effects, which include aches and pain at the site of the incision. Each individual experiences pain differently, so the severity could vary from person to person. The pain may last for a few days or up to several weeks.

Treatments for bone marrow conditions vary based on the severity and progress of the diagnosis. Treatment options include:

Bone marrow is the foundation of your bones, blood and muscles. Keeping your bone marrow healthy focuses on supporting components of your body that grow from bone marrow cells. You can keep your bone marrow healthy by:

A note from Cleveland Clinic

Bone marrow is the soft center of the bones in your body. Bone marrow is necessary to create components of your blood and store fat. The best way to keep your bone marrow healthy is to support the parts of your body that your bone marrow produces, like your blood, muscles and bones.

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October 5th, 2022

A protocol for isolation and culture of mesenchymal stem cells from …

By daniellenierenberg

We explain a protocol for straightforward isolation and culture of mesenchymal stem cells (MSCs) from mouse bone marrow (BM) to supply researchers with a method that can be applied in cell biology and tissue engineering with minimal requirements. Our protocol is mainly on the basis of the frequent medium change in primary culture and diminishing the trypsinization time. Mouse mesenchymal stem cells are generally isolated from an aspirate of BM harvested from the tibia and femoral marrow compartments, then cultured in a medium with Dulbecco's modified Eagle's medium (DMEM) and fetal bovine serum (FBS) for 3 h in a 37 degrees C-5% CO(2) incubator. Nonadherent cells are removed carefully after 3 h and fresh medium is replaced. When primary cultures become almost confluent, the culture is treated with 0.5 ml of 0.25% trypsin containing 0.02% ethylenediaminetetraacetic acid for 2 min at room temperature (25 degrees C). A purified population of MSCs can be obtained 3 weeks after the initiation of culture.

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October 5th, 2022

Are immunotherapy and chemotherapy the same thing? How cancer treatments work – Nebraska Medicine

By daniellenierenberg

As cancer treatments continue to advance and new therapies are introduced, it's easy to get lost in your search for information. To help you better understand the differences between specific cancer treatments and how they work, we spoke with medical oncologist Bhavina Sharma, MD, MPH.

"Chemotherapy are drugs designed to directly attack all rapidly dividing cells in the body, including cancer cells," explains Dr. Sharma. "It relies on the idea that cancer cells reproduce much faster than most healthy cells in our body."

Chemotherapy drugs can be given by infusion or in pill form. Unfortunately, these drugs can't tell the difference between cancerous cells and fast-growing healthy cells like the gastrointestinal tract and hair follicles, leading to side effects such as diarrhea and hair loss. Thankfully, recent advancements in chemotherapy have helped lessen side effects such as nausea, pain and lethargy.

Targeted therapy are special drugs designed to target differences within cancer cells that help them thrive. Unlike chemotherapy, targeted therapy drugs actually change the inner workings of the cancer cell. Because targeted therapy focuses on the part of the cancer cell that makes it different from the normal, healthy cell, it often has fewer side effects than standard chemotherapy treatments.

Immunotherapy is very different than chemotherapy in that it helps our immune system to find and kill cancer cells.

"Cancer cells are abnormal cells that have formed in our body because of cell damage or mutations," explains Dr. Sharma. "Cancer cells hide from your immune system by shutting down certain pathways of the immune response. Immunotherapy unlocks those pathways so your immune system can recognize and remove the cancer cells."

Cellular therapies are treatments that improve the body's ability to fight cancer. "Stem cell therapy falls under the umbrella of cellular therapy," explains Dr. Sharma. "It uses stem cells to mount an immune response to attack your cancer cells."

Stem cells from blood and bone marrow can be used in transplants. These stem cells can either come from a matched donor (allogeneic) or from the patient themselves (autologous).

Chimeric antigen receptor therapy or CAR T-cell, is a type of cellular therapy.

"T cells are white blood cells that help our bodies fight infection and cancer," explains Dr. Sharma. "With CAR T-cell therapy, your own T cells are collected from your blood. These T cells are modified to recognize cancer as a foreign cell and attack it."

CAR T-cell therapy has been approved by the Food and Drug Administration to treat lymphoma, leukemia and multiple myeloma.

Hormone therapy slows or stops the growth of cancer that uses hormones to grow. It is also called hormonal therapy, hormone treatment or endocrine therapy. Hormone therapy is recommended for cancers that are hormone-receptor positive, such as certain breast and prostate cancers. It can't be used in cancers that don't carry hormone receptors.

"Hormone therapy can be used for both early stage and metastatic hormone-receptor positive breast cancers," explains Dr. Sharma. "In patients with early-stage breast cancer, it is used after surgery to help reduce the risk of the cancer coming back."

Chemotherapy, immunotherapy, targeted therapy, and hormone therapy are just a few of the treatments we use to treat cancer. Many of these cancer treatments can be combined with others like cancer surgery and radiation therapy. Every person's journey through cancer is different. Your oncology team will help you sort through the best therapies available to create your treatment plan.

The information in this article is for information purposes only. For specific questions regarding your medical condition or treatment plan, please consult with your doctor directly. To schedule an appointment with a Nebraska Medicine cancer specialist, call 402.559.5600.

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October 5th, 2022

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Side Effects of a Bone Marrow Transplant (Stem Cell Transplant)

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A protocol for isolation and culture of mesenchymal stem cells from …

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Are immunotherapy and chemotherapy the same thing? How cancer treatments work – Nebraska Medicine

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