Certain diseases and treatments can deplete a child's healthy stem cells. Sometimes the body needs help to replenish those cells. When this happens, your child may require a very complex process called a stem cell or bone marrow transplant.

Since 1986, Cook Children's Bone Marrow and Stem Cell Transplant program has performed more than 1,000 transplants in children with cancer, blood disorders or inherited conditions. That's what makes this program one of the more diverse and experienced pediatric transplant programs in the Southwest.

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Over the last three years, 30 to 40 transplants were performed every year for a variety of diseases, with leukemia being the most common primary diagnosis.

The goal of the program is to provide a stem cell or marrow transplant to any child who needs one and to improve the outcomes for these patients who do not have better therapy options. We work to achieve this goal through excellent clinical care from several services within Cook Children's, quality initiatives and ongoing comparison of our processes and performance against large academic transplant centers and international data.

Common referral diagnoses:

Stem cells are cells in the body that have the potential to turn into anything, such as a skin cell, a liver cell, a brain cell, or a blood cell. Stem cells that turn into blood cells are called hematopoietic stem cells. These cells are capable of developing into the three types of blood cells:

Stem cells may come from the patient or from a donor. Stem cells that come from a patient may come from their own cord blood cells if they were harvested from the mother's placenta immediately after the child was born and frozen for later use. Stem cells may also be harvested and frozen before the child or teen undergoes treatment. These stem cells are thawed and put back into the patient's body after treatment is complete.

Donor stem cells come from a compatible family member or through a match from a national registry of donors. Depending on the particular needs of your child, one or all three types of a donor's stem cells will be harvested:

While all three types can replenish a patient's blood and bone marrow cells, there are advantages and disadvantages to each. The doctor will discuss these issues and suggest the best type of stem cell for your child's illness.

If your child has been diagnosed, you probably have lots of questions. We can help. If you would like to schedule an appointment, refer a patient or speak to our staff, please call our offices at 682-885-4007.

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Overview

A bone marrow transplant is a procedure that infuses healthy blood stem cells into your body to replace your damaged or diseased bone marrow. A bone marrow transplant is also called a stem cell transplant.

A bone marrow transplant may be necessary if your bone marrow stops working and doesn't produce enough healthy blood cells.

Bone marrow transplants may use cells from your own body (autologous transplant) or from a donor (allogeneic transplant).

Mayo Clinic's approach

A bone marrow transplant may be used to:

Bone marrow transplants can benefit people with a variety of both cancerous (malignant) and noncancerous (benign) diseases, including:

Bone marrow is the spongy tissue inside some bones. Its job is to produce blood cells. If your bone marrow isn't functioning properly because of cancer or another disease, you may receive a stem cell transplant.

To prepare for a stem cell transplant, you receive chemotherapy to kill the diseased cells and malfunctioning bone marrow. Then, transplanted blood stem cells are put into your bloodstream. The transplanted stem cells find their way to your marrow, where ideally they begin producing new, healthy blood cells.

A bone marrow transplant poses many risks of complications, some potentially fatal.

The risk can depend on many factors, including the type of disease or condition, the type of transplant, and the age and health of the person receiving the transplant.

Although some people experience minimal problems with a bone marrow transplant, others may develop complications that may require treatment or hospitalization. Some complications could even be life-threatening.

Complications that can arise with a bone marrow transplant include:

Your doctor can explain your risk of complications from a bone marrow transplant. Together you can weigh the risks and benefits to decide whether a bone marrow transplant is right for you.

If you receive a transplant that uses stem cells from a donor (allogeneic transplant), you may be at risk of developing graft-versus-host disease (GVHD). This condition occurs when the donor stem cells that make up your new immune system see your body's tissues and organs as something foreign and attack them.

Many people who have an allogeneic transplant get GVHD at some point. The risk of GVHD is a bit greater if the stem cells come from an unrelated donor, but it can happen to anyone who gets a bone marrow transplant from a donor.

GVHD may happen at any time after your transplant. However, it's more common after your bone marrow has started to make healthy cells.

There are two kinds of GVHD: acute and chronic. Acute GVHD usually happens earlier, during the first months after your transplant. It typically affects your skin, digestive tract or liver. Chronic GVHD typically develops later and can affect many organs.

Chronic GVHD signs and symptoms include:

You'll undergo a series of tests and procedures to assess your general health and the status of your condition, and to ensure that you're physically prepared for the transplant. The evaluation may take several days or more.

In addition, a surgeon or radiologist will implant a long thin tube (intravenous catheter) into a large vein in your chest or neck. The catheter, often called a central line, usually remains in place for the duration of your treatment. Your transplant team will use the central line to infuse the transplanted stem cells and other medications and blood products into your body.

If a transplant using your own stem cells (autologous transplant) is planned, you'll undergo a procedure called apheresis (af-uh-REE-sis) to collect blood stem cells.

Before apheresis, you'll receive daily injections of growth factor to increase stem cell production and move stem cells into your circulating blood so that they can be collected.

During apheresis, blood is drawn from a vein and circulated through a machine. The machine separates your blood into different parts, including stem cells. These stem cells are collected and frozen for future use in the transplant. The remaining blood is returned to your body.

If a transplant using stem cells from a donor (allogeneic transplant) is planned, you will need a donor. When you have a donor, stem cells are gathered from that person for the transplant. This process is often called a stem cell harvest or bone marrow harvest. Stem cells can come from your donor's blood or bone marrow. Your transplant team decides which is better for you based on your situation.

Another type of allogeneic transplant uses stem cells from the blood of umbilical cords (cord blood transplant). Mothers can choose to donate umbilical cords after their babies' births. The blood from these cords is frozen and stored in a cord blood bank until needed for a bone marrow transplant.

After you complete your pretransplant tests and procedures, you begin a process known as conditioning. During conditioning, you'll undergo chemotherapy and possibly radiation to:

The type of conditioning process you receive depends on a number of factors, including your disease, overall health and the type of transplant planned. You may have both chemotherapy and radiation or just one of these treatments as part of your conditioning treatment.

Side effects of the conditioning process can include:

You may be able to take medications or other measures to reduce such side effects.

Based on your age and health history, your doctor may recommend lower doses or different types of chemotherapy or radiation for your conditioning treatment. This is called reduced-intensity conditioning.

Reduced-intensity conditioning kills some cancer cells and somewhat suppresses your immune system. Then, the donor's cells are infused into your body. Donor cells replace cells in your bone marrow over time. Immune factors in the donor cells may then fight your cancer cells.

Your bone marrow transplant occurs after you complete the conditioning process. On the day of your transplant, called day zero, stem cells are infused into your body through your central line.

The transplant infusion is painless. You are awake during the procedure.

The transplanted stem cells make their way to your bone marrow, where they begin creating new blood cells. It can take a few weeks for new blood cells to be produced and for your blood counts to begin recovering.

Bone marrow or blood stem cells that have been frozen and thawed contain a preservative that protects the cells. Just before the transplant, you may receive medications to reduce the side effects the preservative may cause. You'll also likely be given IV fluids (hydration) before and after your transplant to help rid your body of the preservative.

Side effects of the preservative may include:

Not everyone experiences side effects from the preservative, and for some people those side effects are minimal.

When the new stem cells enter your body, they begin to travel through your body and to your bone marrow. In time, they multiply and begin to make new, healthy blood cells. This is called engraftment. It usually takes several weeks before the number of blood cells in your body starts to return to normal. In some people, it may take longer.

In the days and weeks after your bone marrow transplant, you'll have blood tests and other tests to monitor your condition. You may need medicine to manage complications, such as nausea and diarrhea.

After your bone marrow transplant, you'll remain under close medical care. If you're experiencing infections or other complications, you may need to stay in the hospital for several days or sometimes longer. Depending on the type of transplant and the risk of complications, you'll need to remain near the hospital for several weeks to months to allow close monitoring.

You may also need periodic transfusions of red blood cells and platelets until your bone marrow begins producing enough of those cells on its own.

You may be at greater risk of infections or other complications for months to years after your transplant.

A bone marrow transplant can cure some diseases and put others into remission. Goals of a bone marrow transplant depend on your individual situation, but usually include controlling or curing your disease, extending your life, and improving your quality of life.

Some people complete bone marrow transplantation with few side effects and complications. Others experience numerous challenging problems, both short and long term. The severity of side effects and the success of the transplant vary from person to person and sometimes can be difficult to predict before the transplant.

It can be discouraging if significant challenges arise during the transplant process. However, it is sometimes helpful to remember that there are many survivors who also experienced some very difficult days during the transplant process but ultimately had successful transplants and have returned to normal activities with a good quality of life.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Living with a bone marrow transplant or waiting for a bone marrow transplant can be difficult, and it's normal to have fears and concerns.

Having support from your friends and family can be helpful. Also, you and your family may benefit from joining a support group of people who understand what you're going through and who can provide support. Support groups offer a place for you and your family to share fears, concerns, difficulties and successes with people who have had similar experiences. You may meet people who have already had a transplant or who are waiting for a transplant.

To learn about transplant support groups in your community, ask your transplant team or social worker for information. Also, several support groups are offered at Mayo Clinic in Arizona, Florida and Minnesota.

Mayo Clinic researchers study medications and treatments for people who have had bone marrow transplants, including new medications to help you stay healthy after your bone marrow transplant.

If your bone marrow transplant is using stem cells from a donor (allogeneic transplant), you may be at risk of graft-versus-host disease. This condition occurs when a donor's transplanted stem cells attack the recipient's body. Doctors may prescribe medications to help prevent graft-versus-host disease and reduce your immune system's reaction (immunosuppressive medications).

After your transplant, it will take time for your immune system to recover. You may be given antibiotics to prevent infections. You may also be prescribed antifungal, antibacterial or antiviral medications. Doctors continue to study and develop several new medications, including new antifungal medications, antibacterial medications, antiviral medications and immunosuppressive medications.

After your bone marrow transplant, you may need to adjust your diet to stay healthy and to prevent excessive weight gain. Maintaining a healthy weight can help prevent high blood pressure, high cholesterol and other negative health effects.

Your nutrition specialist (dietitian) and other members of your transplant team will work with you to create a healthy-eating plan that meets your needs and complements your lifestyle. Your dietitian may also give you food suggestions to control side effects of chemotherapy and radiation, such as nausea.

Your dietitian will also provide you with healthy food options and ideas to use in your eating plan. Your dietitian's recommendations may include:

After your bone marrow transplant, you may make exercise and physical activity a regular part of your life to continue to improve your health and fitness. Exercising regularly helps you control your weight, strengthen your bones, increase your endurance, strengthen your muscles and keep your heart healthy.

Your treatment team may work with you to set up a routine exercise program to meet your needs. You may perform exercises daily, such as walking and other activities. As you recover, you can slowly increase your physical activity.

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When the healthy stem cells come from you, the procedure is called an autologous transplant. When the stem cells come from another person, called a donor, it is an allogeneic transplant. Blood or bone marrow transplants most commonly are used to treat blood cancers or other kinds of blood diseases that decrease the number of healthy blood cells in the body. These transplants also may be used to treat other disorders.

For allogeneic transplants, your doctor will try to find a donor whose blood cells are the best match for you. Your doctor will consider using cells from your close family members, from people who are not related to you and who have registered with the National Marrow Donor Program, or from publicly stored umbilical cord blood. Although it is best to find a donor who is an exact match to you, new transplant procedures are making it possible to use donors who are not an exact match.

Blood or bone marrow transplants are usually performed in a hospital. Often, you must stay in the hospital for one to two weeks before the transplant to prepare. During this time, you will have a narrow tube placed in one of your large veins. You may be given medicine to make you sleepy for this procedure. You also will receive special medicines and possibly radiation to destroy your abnormal stem cells and to weaken your immune system so that it wont reject the donor cells after the transplant.

On the day of the transplant, you will be awake and may get medicine to relax you during the procedure. The stem cells will be given to you through the narrow tube in your vein. The stem cells will travel through your blood to your bone marrow, where they will begin making new healthy blood cells.

After the transplant, your doctor will check your blood counts every day to see if new blood cells have started to grow in your bone marrow. Depending on the type of transplant, you may be able to leave, but stay near the hospital, or you may need to remain in the hospital for weeks or months. The length of time will depend on how your immune system is recovering and whether or not the transplanted cells stay in your body. Before you leave the hospital, the doctors will give you detailed instructions that you must follow to prevent infection and other complications. Your doctor will keep monitoring your recovery, possibly for up to oneyear.

Although blood or bone marrow transplant is an effective treatment for some conditions, the procedure can cause early or late complications. The required medicines and radiation can cause nausea, vomiting, diarrhea, tiredness, mouth sores, skin rashes, hair loss, or liver damage. These treatments also can weaken your immune system and increase your risk for infection. Some people may experience a serious complication called graft-versus-host disease if the donated stem cells attack the body. Other people may reject the donor stem cells after the transplant, which can be an extremely serious complication.

VisitBlood-Forming Stem Cell Transplantsfor more information about this topic.

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HEMATOPOIETIC CELL TRANSPLANTATION OVERVIEW

Hematopoietic cell transplantation (also called bone marrow transplantation or stem cell transplant), is a type of treatment for cancer (and a few other conditions as well). A review of the normal function of the bone marrow will help in the understanding of stem cell transplantation.

Hematopoietic stem cell functionBone marrow is the soft, spongy area in the center of some of the larger bones of the body. The marrow produces all of the different cells that make up the blood, such as red blood cells, white blood cells (of many different types), and platelets. All of the cells of the immune system are also made in the bone marrow. All of these cells develop from a type of precursor cell found in the bone marrow, called a "hematopoietic stem cell."

The body is able to direct hematopoietic stem cells to develop into the blood components needed at any given moment. This is a very active process, with the bone marrow producing millions of different cells every hour. Most of the stem cells stay in the marrow until they are transformed into the various blood components, which are then released into the blood stream. Small numbers of stem cells, however, can be found in the circulating blood, which allows them to be collected under certain circumstances. Various strategies can be employed to increase the number of hematopoietic stem cells in the blood prior to collection. (See 'Peripheral blood' below.)

Hematopoietic cell transplantationSome of the most effective treatments for cancer, such as chemotherapy and radiation, are toxic to the bone marrow. In general, the higher the dose, the more toxic the effects on the bone marrow.

In hematopoietic cell transplantation, you are given very high doses of chemotherapy or radiation therapy, which is intended to more effectively kill cancer cells and unfortunately also destroy all the normal cells developing in the bone marrow, including the critical stem cells. After the treatment, you must have a healthy supply of stem cells reintroduced, or transplanted. The transplanted cells then reestablish the blood cell production process in the bone marrow. Reduced doses of radiation or chemotherapy that do not completely destroy the bone marrow may be used in some settings. (See 'Non-myeloablative transplant' below.)

The cells that will be transplanted can be taken from the bone marrow (called a bone marrow transplant), from the bloodstream (called a peripheral blood stem cell transplant, which requires that you take medication to boost the number of hematopoietic stem cells in the blood), or occasionally from blood obtained from the umbilical cord at the time of birth of a normal newborn (called an umbilical cord blood transplant).

TYPES OF HEMATOPOIETIC CELL TRANSPLANTATION

There are two main types of hematopoietic cell transplantation: autologous and allogeneic.

Autologous transplantIn autologous transplantation, your own hematopoietic stem cells are removed before the high dose chemotherapy or radiation is given, and they are then frozen for storage and later use. After your chemotherapy or radiation is complete, the harvested cells are thawed and returned to you.

Allogeneic transplantIn allogeneic transplantation, the hematopoietic stem cells come from a donor, ideally a brother or sister with a similar genetic makeup. If you do not have a suitably matched sibling, an unrelated person with a similar genetic makeup may be used. Under some circumstances, a parent or child who is only half-matched can also be used; this is termed a haploidentical transplant.

Myeloablative transplantA myeloablative transplant uses very high doses of chemotherapy or radiation prior to transplantation with autologous or allogeneic hematopoietic stem cells.

Non-myeloablative transplantA non-myeloablative transplant, sometimes referred to as a "mini" or reduced intensity transplant, allows you to have less intensive chemotherapy before transplantation with allogeneic hematopoietic stem cells. This approach may be recommended for a variety of reasons including your age, type of disease, other medical issues, or prior therapies.

Which type of transplant is best?Your physician will determine whether allogeneic or autologous transplantation is best, based on many factors including the type of cancer, your age and overall health, and the availability of a suitable donor. As a general rule, autologous transplantation is associated with fewer serious side effects, since you are given cells from your own body. However, an autologous transplant may be less effective than an allogeneic transplant in treating certain kinds of cancer.

In an allogeneic transplant, the donor's immune system, which is generated from the transplanted hematopoietic stem cells, recognize your cells, including the tumor cells, as foreign and rejects them. This beneficial reaction is called the graft-versus-tumor effect. In many cancers, the immune response caused by the transplanted cells improves the overall effectiveness of the treatment. This immune response helps kill off any residual cancer cells remaining in your body.

A major concern is that you will have an immune response against normal tissues as well, called graft-versus-host disease. (See 'Graft-versus-host disease' below.)

In a non-myeloablative transplant, it is hoped that the graft-versus-tumor effect, rather than the high-dose chemotherapy, will help eradicate the cancer, although graft-versus-host disease is a concern (see 'Graft-versus-host disease' below).

CHOOSING A DONOR

There are many possible choices for an allogeneic hematopoietic stem cell donor. These are described below. (See "Donor selection for hematopoietic cell transplantation".)

Matched donorTo help minimize the problems that can be caused by the expected immune response, a donor who has similar genetic makeup to you is preferred. Your cells will seem "less foreign" to the transplanted donor cells. Siblings (ie, brothers and sisters who share the same parents as you) are typically the only members of your family that are tested for being a donor because they have a one in four chance of sharing genetic characteristics with you; these characteristics are critical for your body to accept the graft. In general, parents, children, and relatives are not suitable donors since they do not share the same parents, and therefore do not have the same genetic material.

An exception is called a haploidentical transplant, which may be considered under certain circumstances.

Matched unrelated donorIf no siblings are available, or if testing the blood of the siblings does not reveal a match, a matched unrelated donor may be used. The search for an appropriate donor can be accomplished using transplant registries throughout the world.

Mismatched related donor or umbilical cord blood donorSome patients are offered treatment with cells from a partially matched family member (called mismatched related donor). The hematopoietic stem cell product may be specially prepared to minimize the immune response in the patient. Another alternative is to use umbilical cord blood, collected from a healthy newborn infant at the time of delivery; this blood is a rich source of hematopoietic stem cells.

PRE-HEMATOPOIETIC CELL TRANSPLANTATION PROCEDURES

Stem cell transplantation regimens vary from one patient to another, and depend upon the type of cancer, the treatment program used by the medical center, the clinical trial protocol (if the patient is enrolled in a clinical trial), as well as other factors. The most common components of the hematopoietic cell transplantation procedure are outlined here. You should talk with your transplant team about specific details of their program. (See "Preparative regimens for hematopoietic cell transplantation".)

Health evaluationBefore undergoing hematopoietic cell transplantation, you will have a complete evaluation of your health. Your complete health history is reviewed by the transplant team. Most patients also have a number of tests.

Your mental health is reviewed because of the stress and demands of stem cell transplantation; some patients meet with a mental health counselor to discuss concerns and to plan coping strategies.

You will also meet with a transplant coordinator or nurse to discuss the transplant process. Because patients who receive donor bone marrow are hospitalized for several weeks to months, it is important that you have a clear understanding of what will happen and what services are available. Some patients prefer to have a friend or family member accompany them, tape record the conversation with the transplant physician, or have this information in writing so that they can review it later.

In many cases, patients undergo hematopoietic cell transplantation while they are in remission from their underlying disease. You may feel well going into treatment, but you should be prepared to feel poorly for a period of time. You must understand that you will require intensive treatment and monitoring, but that there are long-term benefits from the treatment.

Life planningPatients who will be in the hospital for several weeks or months need to make plans regarding their family, home, finances, pets, and employment. The National Marrow Donor Program has excellent information about these and other stem cell transplantation related topics.

During the pre-transplant planning process, you should consider completing an advanced directive. This is a legal document that describes the type of care you want in case you are unable to communicate. Advance directives include a living will, durable power of attorney, and healthcare proxy; a social worker or attorney can provide guidance about what documents are needed. The laws surrounding these documents vary from one state to another, so it is important to be sure the correct guidelines are used.

Central line placementA number of medications will be required before, during, and after hematopoietic cell transplantation. To avoid the need for multiple intravenous lines and needle sticks, most patients will have a central line placed before treatment begins. This requires a short surgical procedure to insert a thin, flexible plastic tube into a large vein in the chest, above the heart. The line usually has two or three ports, which can be used to infuse medications or blood products (including the hematopoietic stem cell product), as well as to withdraw blood samples.

After the central line is placed, you must keep the area clean and watch for signs and symptoms of infection (pain, redness, swelling, or fluid drainage from the site, fever or chills).

Harvesting hematopoietic stem cellsIf you are having an autologous transplant, hematopoietic stem cells will be removed from your body before intensive chemotherapy or radiation begins. The most common sources for hematopoietic stem cells are bone marrow and blood.

Bone marrowIf your bone marrow has been invaded with cancer cells, hematopoietic stem cell removal may be preceded by one or more courses of chemotherapy. Removal (called harvest) of bone marrow stem cells is done while you are under general or epidural anesthesia. The harvest is done by using a long needle to repeatedly remove a sample of bone marrow fluid from multiple areas in your pelvic and hip bones.

Peripheral bloodThe harvest of peripheral blood stem cells is similar to the process of platelet donation and is more frequently used than a bone marrow harvest. It uses an apparatus, called an apheresis device, which removes hematopoietic stem cells and other cells from blood by a filtration process. Blood is removed from a vein in one location, filtered, and then returned to a vein in another location. The process does not require anesthesia.

In order for there to be sufficient numbers of hematopoietic stem cells in the blood, you (or the donor) must first be treated with either chemotherapy or a growth factor that stimulates the production and release of hematopoietic stem cells into the blood. Healthy donors only receive growth factor; patients with cancer may receive growth factor alone or chemotherapy plus growth factor. The most commonly used growth factor is granulocyte colony-stimulating factor (G-CSF or Neupogen).

Allogeneic bone marrow harvestPeople who donate their bone marrow will undergo harvest the day of transplant or one day prior. The donor is usually given general anesthesia to prevent pain.

Following the procedure, pain in the donor is usually relatively minor and can be treated with pain medications such as acetaminophen. The donor may be hospitalized overnight following the procedure, and generally returns to his or her prior state of health within the following one to two weeks.

Myeloablative therapyAs noted above, many patients receiving hematopoietic cell transplantation will undergo myeloablative therapy, which destroys bone marrow function as part of the intensive treatment for the patient's underlying cancer. The purpose of this treatment is to reduce the amount of cancer in the body and also to suppress the immune system adequately so that the graft will not be rejected. Depending upon the underlying disease and other factors, this phase of treatment may involve intensive chemotherapy, total body irradiation (radiation therapy), or both.

Preventing infectionWhen bone marrow function is destroyed, you are at risk for developing life-threatening infections because you have temporarily lost your ability to produce white blood cells (the infection-fighting cells in the blood). You are also at risk for excessive bleeding due to the reduced number of platelets in the blood. (See "Prevention of infections in hematopoietic cell transplant recipients".)

It is important to minimize your exposure to bacteria, viruses, and fungi after myeloablative therapy because even a small number of organisms (that are usually encountered every day) can cause serious infection.

Patients who undergo allogeneic transplant are often placed in protective isolation in a private room. The room's air is filtered and air from the room is forced out when the door is opened (called a positive-pressure room). This isolation, combined with feeling poorly, can be challenging to some people who may feel depressed and/or anxious. Discussing these issues with your health care team is very important.

Special precautions are required for all persons who enter the room to reduce the chance of infection. Hand washing is one of the most important precautions, and has been shown to significantly reduce the chance of transmitting infection. Visitors should NOT bring fresh fruit, plants, or flowers into your room because these can harbor microorganisms that are dangerous.

Other measures may be taken to reduce the chance of infection. For example, antibiotics, antifungal, and/or antiparasitic medications may be given to prevent infections, and your diet may be restricted to exclude items that contain potentially infectious organisms. For example, all foods should be cooked until hot, raw fruits and vegetables should be avoided, and drinking water should be sterilized.

Most patients can shower. There has been a concern that showers can aerosolize fungal spores, and some centers prefer that patients take a tub or sponge bath. You can wear a hospital gown or your own clean clothing.

Different transplant centers use different precautions and your health care team will discuss the precautions and procedures that they expect.

Blood product transfusionsDuring the time that the marrow is not functioning, you will likely require transfusion of blood products, such as red cells, which carry oxygen to the tissues, or platelets, which help prevent bleeding. These blood products have no white blood cells and are irradiated to reduce the risk of an immune response.

HEMATOPOIETIC CELL TRANSPLANTATION PROCEDURE

When the intensive chemotherapy and/or radiation are complete, you will be given an infusion of the harvested bone marrow or peripheral blood stem cells. The infusion is given through an intravenous (IV) line, usually the central line. The infusion usually takes about an hour, and usually causes no pain.

The cells find their way to the bone marrow, where they will reestablish normal production of blood cells; this process is called engraftment. Determining when engraftment has occurred is important because it is used to determine when it is safe for you to go home and/or reduce isolation procedures. Medications that stimulate the bone marrow to produce white and red cells may be used when engraftment is slower than expected. (See "Hematopoietic support after hematopoietic cell transplantation".)

Engraftment is measured by performing daily blood cell counts. Neutrophils are a type of white blood cell that are a marker of engraftment; the absolute neutrophil count (ANC) must be at least 500 for three days in a row to say that engraftment has occurred. This can occur as soon as 10 days after transplant, although 15 to 20 days is common for patients who are given bone marrow or peripheral blood cells. Umbilical cord blood recipients usually require between 21 and 35 days for neutrophil engraftment.

Platelet counts are also used to determine when engraftment has occurred. The platelet count must be between 20,000 and 50,000 (without a recent platelet transfusion). This usually occurs at the same time or soon after neutrophil engraftment, but can take as long as eight weeks and even longer in some instances for people who are given umbilical cord blood.

HEMATOPOIETIC CELL TRANSPLANTATION SIDE EFFECTS

The high-dose chemotherapy and total body irradiation required for hematopoietic cell transplantation can have serious side effects. You should discuss the expected side effects, toxicities, and risks associated with stem cell transplant before deciding to undergo the procedure. You will be asked to sign a consent form indicating that you have received verbal and written information to understand the risks and benefits of the proposed treatment, possible treatment alternatives, and that all your questions have been answered.

Common side effectsSome of the most common side effects include:

Mucositis(mouth sores) and diarrhea Mucositis and diarrhea are caused by the damage done to rapidly dividing cells (such as skin cells in the mouth and digestive tract) by chemotherapy and radiation. If mucositis is severe and affects your ability to eat, intravenous nutrition (called TPN, total parenteral nutrition) may be given. Pain medications are usually given as well.

Nausea and vomiting Nausea and vomiting can be prevented and treated with a combination of medications, usually including a 5-HT3 receptor antagonist (dolasetron, granisetron, ondansetron, tropisetron, or palonosetron), an NK1 receptor antagonist (aprepitant [Emend]), and a steroid (dexamethasone).

Loss of hair Loss of hair is temporary, and generally includes hair on the head, face, and body. After high-dose chemotherapy and radiation are completed, hair begins to regrow. No treatment is available to prevent hair loss or speed its regrowth.

Infertility The risk of permanent infertility after stem cell transplant depends upon the treatments used (high-dose chemotherapy versus total body irradiation, ablative versus non-ablative regimen) and dosage given. If you are of reproductive age, you should speak with your healthcare provider about options for lowering the risks of infertility and the option of donating eggs or sperm before treatment begins. (See "Fertility preservation in patients undergoing gonadotoxic treatment or gonadal resection".)

Organ toxicity The lungs, liver, and bones are at greatest risk of damage as a result of treatments used with stem cell transplantation. People who have total body irradiation can develop cataracts in the eyes, although this complication is less common with current methods of delivering radiation treatment.

Secondary cancers There is a small risk of a second cancer developing in patients who undergo stem cell transplantation, probably as a result of the treatments used for the first cancer as well as the treatments required for transplant. The second cancer usually develops several years (typically three to five) after stem cell transplantation. (See "Malignancy after hematopoietic cell transplantation".)

Graft-versus-host diseaseBetween 10 and 50 percent of patients who receive an allogeneic transplant experience a side effect known as graft-versus-host disease (GVHD). Graft-versus-host disease is separated into acute and chronic phases due to timing and clinical presentation. This problem does not occur following autologous transplantation (when the patient is the donor). (See "Prevention of acute graft-versus-host disease".)

The "graft" refers to the transplanted hematopoietic stem cells; the "host" refers to the patient. Thus, graft-versus-host disease refers to a condition in which the donor's immune cells attack some of your organs. GVHD is the biggest single threat, other than the underlying disease, to the success of a stem cell transplant.

Treatments are given to help prevent GVHD, and generally include immunosuppressive medications, antibiotics, and sometimes steroids. If GVHD develops, additional treatment with high-dose steroids may lessen its severity. Symptoms can include skin rash, diarrhea, liver damage, or other problems, depending upon the organ that is affected. (See "Treatment of chronic graft-versus-host disease".)

Graft failureFailure of engraftment is a rare complication that occurs in approximately one percent of cases following hematopoietic cell transplantation. The risk of graft failure can be higher depending upon the type of transplant and the source of hematopoietic stem cells. Discuss these risks with the transplant team prior to treatment. (See "Immunotherapy for the prevention and treatment of relapse following hematopoietic cell transplantation".)

Risk of deathHematopoietic cell transplantation carries a risk of treatment-related death. The risk of death depends upon your age, the nature of the underlying disease, the type of transplant (autologous or allogeneic), and other factors, including the skill and expertise of the institution where treatment is offered. Your risk, as well as the potential benefits of hematopoietic cell transplantation, should be discussed with the treatment team before any decision is made about undergoing a transplant procedure.

POST-HEMATOPOIETIC CELL TRANSPLANTATION CARE

After engraftment occurs, blood cell counts continue to rise and the immune system becomes stronger. You will usually be cared for by the transplant team and monitored closely for complications.

Non-myeloablative transplants may be done on an outpatient basis, allowing you to sleep at home. Other types of transplantation require you to stay in the hospital for three to four weeks following transplantation. In all cases, frequent visits to the healthcare provider's office are needed following discharge. If you live a distance from your provider, you should arrange to live in a place within reasonable driving distance to the treatment center until at least 100 days have passed since the transplant.

Patients who undergo hematopoietic cell transplantation are at an increased risk of infection for many months following transplantation. You should be aware of these risks and monitor yourself for symptoms of infection, including fever (temperature greater than 100.4F or 38C), pain, or chills. You may be given antibiotics to prevent infections.

Studies have shown that most patients who undergo transplant and remain free of cancer have a good quality of life. Most patients are able to be active, employed, and in reasonably good health. Quality of life usually continues to improve in the months following transplant.

CLINICAL TRIALS

A clinical trial is a carefully controlled way to study the effectiveness of new treatments or new combinations of known therapies, and patients who will undergo hematopoietic cell transplantation may be asked to participate. Ask a healthcare provider for more information about clinical trials, or read further at the following web sites.

http://www.cancer.gov/clinicaltrials/

http://clinicaltrials.gov/

Videos addressing common questions about clinical trials are available from the American Society of Clinical Oncology (http://www.cancer.net/pre-act).

SUMMARY

Hematopoietic cell transplantation (also called bone marrow transplantation or stem cell transplant) is a treatment used in some types of cancer particularly malignancies of the blood.

Bone marrow is the soft, spongy area in the center of some of the larger bones of the body. The marrow produces all of the different cells that make up the blood, such as red blood cells, white blood cells, and platelets. All of these cells develop from a type of basic cell found in the bone marrow, called a stem cell.

In hematopoietic cell transplantation, the patient is given very high doses of chemotherapy or radiation therapy, which kills cancer cells and destroys all the normal cells developing in the bone marrow, including the critical stem cells. After the treatment, the patient must have a healthy supply of hematopoietic stem cells reintroduced, or transplanted.

There are two types of hematopoietic cell transplantation, autologous and allogeneic. An autologous hematopoietic cell transplant uses a patient's own bone marrow or blood. An allogeneic hematopoietic cell transplant uses a donor's bone marrow or blood. The donor is usually a relative of the patient (eg, sister), although unrelated donors are sometimes used.

Most patients who have hematopoietic cell transplantation must remain in the hospital for several days or weeks during their treatment and recovery. It is important to understand and follow the hospital's stem cell transplantation treatment plan to minimize the risk of complications (eg, infection) and to know what to expect in advance.

The treatments required before and during hematopoietic cell transplantation can have serious side effects. Patients should be aware of the most common side effects (eg, diarrhea, nausea, vomiting, mouth sores) as well as the types of treatments that are available to improve comfort.

Following hematopoietic cell transplantation, most people stay in the hospital for several weeks. However, even after going home, frequent visits with a doctor or nurse are needed for three to six months.

Clinical trials are carefully controlled studies of new treatments or new combinations of current treatment. Clinical trials help researchers to learn the best way to treat specific conditions. Some patients who have stem cell transplantation will be asked to participate in a clinical trial.

WHERE TO GET MORE INFORMATION

Your healthcare provider is the best source of information for questions and concerns related to your medical problem.

This article will be updated as needed on our website (www.uptodate.com/patients). Related topics for patients, as well as selected articles written for healthcare professionals, are also available. Some of the most relevant are listed below.

Patient level informationUpToDate offers two types of patient education materials.

The BasicsThe Basics patient education pieces answer the four or five key questions a patient might have about a given condition. These articles are best for patients who want a general overview and who prefer short, easy-to-read materials.

Patient education: Bone marrow transplant (The Basics)Patient education: Donating bone marrow or blood stem cells (The Basics)Patient education: Leukemia in adults (The Basics)Patient education: Leukemia in children (The Basics)Patient education: Lymphoma (The Basics)Patient education: Acute lymphoblastic leukemia (ALL) (The Basics)Patient education: Acute myeloid leukemia (AML) (The Basics)Patient education: Chronic lymphocytic leukemia (CLL) (The Basics)Patient education: Chronic myeloid leukemia (CML) (The Basics)Patient education: Diffuse large B cell lymphoma (The Basics)Patient education: Follicular lymphoma (The Basics)Patient education: Hodgkin lymphoma in adults (The Basics)Patient education: Hodgkin lymphoma in children (The Basics)Patient education: Myelodysplastic syndromes (MDS) (The Basics)Patient education: Sickle cell disease (The Basics)Patient education: Immune thrombocytopenia (ITP) (The Basics)Patient education: Beta thalassemia major (The Basics)Patient education: Chronic granulomatous disease (The Basics)Patient education: Invasive aspergillosis (The Basics)Patient education: When your child has sickle cell disease (The Basics)Patient education: Neutropenia and fever in people being treated for cancer (The Basics)

Beyond the BasicsBeyond the Basics patient education pieces are longer, more sophisticated, and more detailed. These articles are best for patients who want in-depth information and are comfortable with some medical jargon.

This topic currently has no corresponding Beyond the Basics content.

Professional level informationProfessional level articles are designed to keep doctors and other health professionals up-to-date on the latest medical findings. These articles are thorough, long, and complex, and they contain multiple references to the research on which they are based. Professional level articles are best for people who are comfortable with a lot of medical terminology and who want to read the same materials their doctors are reading.

Donor selection for hematopoietic cell transplantationHematopoietic support after hematopoietic cell transplantationImmunotherapy for the prevention and treatment of relapse following hematopoietic cell transplantationPreparative regimens for hematopoietic cell transplantationPrevention of acute graft-versus-host diseaseSources of hematopoietic stem cellsTreatment of chronic graft-versus-host diseasePrevention of infections in hematopoietic cell transplant recipientsFertility preservation in patients undergoing gonadotoxic treatment or gonadal resectionMalignancy after hematopoietic cell transplantation

The following organizations also provide reliable health information.

National Library of Medicine

(www.nlm.nih.gov/medlineplus/healthtopics.html)

National Marrow Donor Program

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Hematopoietic cell transplantation (bone marrow ...

Bone Marrow Stem Cells Comments Off on Hematopoietic cell transplantation (bone marrow … | November 11th, 2017

If you or a loved one will be having a bone marrow transplant or donating stem cells, what does it entail? What are the different types of bone marrow transplants and what is the experience like for both the donor and recipient?

A bone marrow transplant is a procedure in which when special cells (called stem cells) are removed from the bone marrow or peripheral blood, filtered and given back either to the same person or to another person.

Since we now derive most stem cells needed from the blood rather than the bone marrow, a bone marrow transplant is now more commonly referred to as stem cell transplant.

Bone marrow is found in larger bones in the body such as the pelvic bones. This bone marrow is the manufacturing site for stem cells. Stem cells are "pluripotential" meaning that the cells are the precursor cells which can evolve into the different types of blood cells, such as white blood cells, red blood cells, and platelets.

If something is wrong with the bone marrow or the production of blood cells is decreased, a person can become very ill or die. In conditions such as aplastic anemia, the bone marrow stops producing blood cells needed for the body. In diseases such as leukemia, the bone marrow produces abnormal blood cells.

The purpose of a bone marrow transplant is thus to replace cells not being produced or replace unhealthy stem cells with healthy ones.

This can be used to treat or even cure the disease.

In addition for leukemias, lymphomas, and aplastic anemia, stem cell transplants are being evaluated for many disorders, ranging from solid tumors to other non-malignant disorders of the bone marrow, to multiple sclerosis.

There are two primary types of bone marrow transplants, autologous and allogeneic transplants.

The Greek prefix "auto" means "self." In an autologous transplant, the donor is the person who will also receive the transplant. This procedure, also known as a "rescue transplant" involves removing your stem cells and freezing them. You then receive high dose chemotherapy followed by infusion of the thawed out frozen stem cells. It may be used to treat leukemias, lymphomas, or multiple myeloma.

The Greek prefix "allo" means "different" or "other." In an allogeneic bone marrow transplant, the donor is another person who has a genetic tissue type similar to the person needing the transplant. Because tissue types are inherited, similar to hair color or eye color, it is more likely that you will find a suitable donor in a family member, especially a sibling. Unfortunately, this occurs only 25 to 30 percent of the time.

If a family member does not match the recipient, the National Marrow Donor Program Registry database can be searched for an unrelated individual whose tissue type is a close match. It is more likely that a donor who comes from the same racial or ethnic group as the recipient will have the same tissue traits.

Learn more about finding a donor for a stem cell transplant.

Bone marrow cells can be obtained in three primary ways. These include:

The majority of stem cell transplants are done using PBSC collected by apheresis (peripheral blood stem cell transplants.) This method appears to provide better results for both the donor and recipient. There still may be situations in which a traditional bone marrow harvest is done.

Donating stem cells or bone marrow is fairly easy. In most cases, a donation is made using circulating stem cells (PBSC) collected by apheresis. First, the donor receives injections of a medication for several days that causes stem cells to move out of the bone marrow and into the blood. For the stem cell collection, the donor is connected to a machine by a needle inserted in the vein (like for donating blood.) Blood is taken from the vein, filtered by the machine to collect the stem cells, then returned back to the donor through a needle in the other arm. There is almost no need for a recovery time with this procedure.

If stem cells are collected by bone marrow harvest (much less likely,) the donor will go to the operating room and while asleep under anesthesia, a needle will be inserted into either the hip or the breastbone to take out some bone marrow. After awakening, there may be some pain where the needle was inserted.

A bone marrow transplant can be a very challenging procedure for the recipient.

The first step is usually receiving high doses of chemotherapy and/or radiation to eliminate whatever bone marrow is present. For example, with leukemia, it is first important to remove all of the abnormal bone marrow cells.

Once a person's original bone marrow is destroyed, the new stem cells are injected intravenously, similar to a blood transfusion. The stem cells then find their way to the bone and start to grow and produce more cells (called engraftment.)

There are many potential complications. The most critical time is usually when the bone marrow is destroyed so that few blood cells remain. Destruction of the bone marrow results in greatly reduced numbers of all of the types of blood cells (pancytopenia.) Without white blood cells there is a serious risk of infection, and infection precautions are used in the hospital (isolation.) Low levels of red blood cells (anemia) often require blood transfusions while waiting for the new stem cells to begin growing. Low levels of platelets (thrombocytopenia) in the blood can lead to internal bleeding.

A common complication affecting 40 to 80 percent of recipients is graft versus host disease. This occurs when white blood cells (T cells) in the donated cells (graft) attack tissues in the recipient (the host,) and can be life-threatening.

An alternative approach referred to as a non-myeloablative bone marrow transplant or "mini-bone marrow transplant" is somewhat different. In this procedure, lower doses of chemotherapy are given that do not completely wipe out or "ablate" the bone marrow as in a typical bone marrow transplant. This approach may be used for someone who is older or otherwise might not tolerate the traditional procedure. In this case, the transplant works differently to treat the disease as well. Instead of replacing the bone marrow, the donated marrow can attack cancerous cells left in the body in a process referred to as "graft versus malignancy."

If you'd like to become a volunteer donor, the process is straightforward and simple. Anyone between the ages of 18 and 60 and in good health can become a donor. There is a form to fill out and a blood sample to give; you can find all the information you need at the National Marrow Donor Program Web site. You can join a donor drive in your area or go to a local Donor Center to have the blood test done.

When a person volunteers to be a donor, his or her particular blood tissue traits, as determined by a special blood test (histocompatibility antigen test,) are recorded in the Registry. This "tissue typing" is different from a person's A, B, or O blood type. The Registry record also contains contact information for the donor, should a tissue type match be made.

Bone marrow transplants can be either autologous (from yourself) or allogeneic (from another person.) Stem cells are obtained either from peripheral blood, a bone marrow harvest or from cord blood that is saved at birth.

For a donor, the process is relatively easy. For the recipient, it can be a long and difficult process, especially when high doses of chemotherapy are needed to eliminate bone marrow. Complications are common and can include infections, bleeding, and graft versus host disease among others.

That said, bone marrow transplants can treat and even cure some diseases which had previously been almost uniformly fatal. While finding a donor was more challenging in the past, the National Marrow Donor Program has expanded such that many people without a compatible family member are now able to have a bone marrow/stem cell transplant.

Sources:

American Society of Clinical Oncology. Cancer.Net. What is a Stem Cell Transplant (Bone Marrow Transplant)? Updated 01/16. http://www.cancer.net/navigating-cancer-care/how-cancer-treated/bone-marrowstem-cell-transplantation/what-stem-cell-transplant-bone-marrow-transplant

U.S. National Library of Medicine. MedlinePlus. Bone Marrow Transplant. Updated 10/03/17. https://medlineplus.gov/ency/article/003009.htm

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bone marrow/stem cell transplant - verywell.com

Bone Marrow Stem Cells Comments Off on bone marrow/stem cell transplant – verywell.com | November 8th, 2017

A stem cell or bone marrow transplant replaces damaged blood cells with healthy ones. It can be used to treat conditions affecting the blood cells, such as leukaemia and lymphoma.

Stem cells arespecial cells produced bybone marrow (aspongytissue found in the centre of some bones) that can turn into different types of blood cells.

The three maintypes of blood cellthey can become are:

A stem cell transplant involves destroying any unhealthy blood cells and replacing them with stem cells removed from the blood or bone marrow.

Stem cell transplants are used to treat conditions in which the bone marrow is damaged and is no longer able to produce healthy blood cells.

Transplants can also be carried out to replace blood cells that are damaged or destroyed as a result of intensive cancer treatment.

Conditions that stem cell transplants can be used to treat include:

A stem cell transplant will usually only be carried out if other treatments haven't helped, the potential benefits of a transplant outweigh the risks and you're in relatively good health, despite your underlying condition.

A stem cell transplant can involve taking healthy stem cells from the blood or bone marrow of one person ideally a close family member with the same or similar tissue type (see below) and transferring them to another person. This is called an allogeneic transplant.

It's also possible to remove stem cells from your own body and transplant them later, after any damaged or diseased cells have been removed. This is called an autologous transplant.

Astem celltransplant has five main stages. These are:

Having a stem cell transplant can be an intensive and challenging experience. You'll usually need to stay in hospital fora month or more until the transplant starts to take effect and itcan takea year or two to fully recover.

Read more about what happens during a stem cell transplant.

Stem celltransplants arecomplicated procedures with significant risks. It's important that you're aware of both the risks and possible benefits before treatment begins.

Possible problems that can occur during or after the transplant process include:

Read more about the risks of having a stem cell transplant.

Ifit isn't possible to use your own stem cells for the transplant (see above), stem cells will need to come from a donor.

To improve the chances ofthetransplant being successful, donated stem cells need tocarry a special genetic marker known as a human leukocyte antigen (HLA) that'sidentical or very similar to that of the person receiving the transplant.

The best chance of getting a match is from a brother or sister, or sometimes another close family member. If there are no matches in your close family,a search of theBritish Bone Marrow Registry will be carried out.

Most peoplewill eventually find a donor in the registry,although a small number of people may find it very hard or impossibleto find a suitable match.

The NHS Blood and Transplant website has more information about stem cell and bone marrow donation.

Page last reviewed: 08/10/2015

Next review due: 01/10/2018

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Stem cell and bone marrow transplants - NHS Choices

Bone Marrow Stem Cells Comments Off on Stem cell and bone marrow transplants – NHS Choices | October 24th, 2017

Stem cell therapy is a two-step process. First, the patients blood cells are destroyed by chemotherapy, radiation therapy or immunosuppression. This conditioning process also eradicates any cancer cells that survived first-line treatment. Second, the patient receives stem cells harvested from a donors bone marrow or peripheral blood (circulating blood). While this can be an effective cure, it can cause graft-versus-host disease (GVHD) in up to 50% of patients. GVHD is more likely to develop in patients who have received a peripheral blood transplant and can kill 15%-20% of patients.

Two types of GVHD can develop, acute and chronic, and patients may develop either one, both or neither type. GVHD is less likely to occur and symptoms are milder if the donor cells closely match those of the patient. Acute GVHD can develop within 100 days of a transplant. The first step of stem cell therapy can cause tissue damage, and bacteria from the gut can escape into the bloodstream. This primes the patients antigen-presenting cells (cells that activate the immune response), which subsequently encourage donor T cells to proliferate and attack the patients tissues. Symptoms include vomiting, diarrhea, skin rashes, nausea, vomiting and liver problems. This can be resolved relatively quickly in one third of patients using immunosuppressive treatments, but some patients can progress to chronic GVHD.

The biological mechanisms responsible for chronic GVHD are not completely understood, but scientists believe that other immune system cells from the donor (B cells and macrophages) are stimulated and damage the patients tissues. Symptoms include dry eyes, mouth sores, muscle weakness, fatigue and joint problems.

Unfortunately, development of effective treatments for GVHD is not keeping up with the increasing number of GVHD patients or with advances in understanding this disease. At present, standard treatments include corticosteroids and drugs that reduce IL-2, an immune system chemical that helps T-cells multiply and diversify. These treatments have various side effects including suppressing the patients immune system, thereby increasing risk of infection.

One challenge stalling drug research is that a small degree of graft-versus-host response must occur for successful stem cell therapy: donor cells will destroy any cancer cells that remain after the first stage of therapy. This challenge is discussed in a recent article in Science Health.Although several treatments have been trialed, success is variable and often targets only acute GVHD or chronic GVHD. Biomarkers have also been detected that may help identify individuals at risk of developing severe GVHD, information that may aid the development of personalized treatment strategies. Drugs that have been approved for other diseases, but not for GVHD, show promise and include ibrutinib for chronic GVHD (approved for specific blood cancers) and ruxolitinib for acute GVHD (approved for bone marrow disorders).

The impact of stem cell therapy must not be underestimated: up to 50% of recipients will develop GVHD. Unfortunately, some individuals will develop chronic GVHD, a condition that is just as difficult to survive as cancer. This highlights the importance of developing continued care strategies for individuals receiving stem cell therapy as a final defence against cancer.

Written byNatasha Tetlow, PhD

Reference: Cohen J. A stem cell transplant helped beat back a young doctors cancer. Now, its assaulting his body. Science Health. 2017. Available at: DOI: 10.1126/science.aan7079

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Stem Cell Therapy: A Lethal Cure - Medical News Bulletin

Bone Marrow Stem Cells Comments Off on Stem Cell Therapy: A Lethal Cure – Medical News Bulletin | September 8th, 2017

On Saturday morning, a convoy of vintage Ford Broncos carrying some very precious cargo made a stop at La Caadas Descanso Gardens.

En route from Childrens Hospital Los Angeles, the motorcade was led by a golden 1971 Bronco with Thousand Oaks resident Tyler Kelly at the helm. Tucked safely into a car seat in the back was 2-year-old Pierce Kelly, known by family and friends as Fierce Pierce, still recovering from a July 21 bone marrow transplant.

At the La Caada home of relatives Donna and Dave McLaughlin, Pierce will recuperate under the watchful eye of mom Aubrey. For 100 days following the procedure, he must reside within a 30-minute drive of Childrens Hospital for monitoring.

Saturdays 13-mile drive was just one portion of a tumultuous journey the Kellys have been on since April 7, when Pierce was diagnosed with acute myeloid leukemia. His chance of surviving the devastating illness with treatment alone was only 50%, according to mom Aubrey. But there was one hope if the Kellys could find a bone marrow donor, Pierces odds would improve by at least another 15%.

We were at the mercy of whoever had registered, Aubrey Kelly recalled.

Among the nearly 13.5 million Americans already listed as donors on the Be the Match Marrow Registry, there were no donors close enough to be a match with Pierce.

Raquel Edpao, a community outreach specialist for Be the Match, said on any given day there are 14,000 people like the Kellys, searching registries for a bone marrow match. Its her job to help educate people how simple it is to join the registry and to donate if called.

Potential donors register online at bethematch.org, then receive and turn in a cheek swab. After that, theyre contacted if they are a potential match for someone. Edpao estimates about one out of every 430 registrants will be asked to donate.

There are so many misconceptions about donating, she said, invoking myths about spinal drilling, painful extractions and missed days at work. Its usually as simple as donating blood.

In about 20% of cases donors are asked to undergo a marrow extraction, a 45-minute outpatient procedure involving a general anesthetic.

Luckily for the Kellys, a search of donors worldwide returned a single donor in France whose human leukocyte antigen (HLA) protein was a 10-out-of-10 match with Pierces. While the marrow was shipped, the 2-year-old underwent chemotherapy to destroy most of his damaged stem cells in preparation for the donation.

Its a fine balance of leaving him with enough cells to receive the new ones, but not so many that the new cells dont have enough room to grow, Aubrey Kelly said, explaining how her sons blood type switched from A positive, his own type, to the donors O negative.

Pierces recovery from the transplant requires a sterile environment that means he cannot stay with siblings Sierra, 4, and 6-month-old Harper. Donna McLaughlin, a cousin of Aubrey Kellys dad, said she and husband Dave were happy to offer their home in La Caadas Paradise Valley neighborhood for his recovery.

Ive worked for the past week cleaning my house its never been so clean, she said of her preparation for Pierce and Aubreys 57-day visit. Im being paranoid, I know, but he is going to be OK on my watch.

Knowing he would have to return to Thousand Oaks to take care of Pierces sisters, Tyler Kelly wanted to ensure his sons trip from the hospital would be a special one. The Bronco the same vehicle his mother drove to the hospital in 1981 so he could be delivered, and the same one he and Aubrey have used to get to the delivery room in time for the birth of their own three children seemed a fitting conveyance.

We wanted to continue the tradition, he said.

Hoping to assemble a retinue for the drive, Tyler Kelly reached out to enthusiast club SoCal Broncos and classicbroncos.com. Several people responded, including Agoura Hills Bronco owner Dan Bennett, for whom the cause was personal. About 10 years ago he saved a life by donating his own bone marrow.

To be able to go in and help play an intrinsic role in saving someones life is a really special thing, Bennett said. I think everybody should do it.

sara.cardine@latimes.com

Twitter: @SaraCardine

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Convoy from Children's Hospital to La Caada carries precious cargo a 2-year-old bone marrow recipient - Los Angeles Times

Bone Marrow Stem Cells Comments Off on Convoy from Children’s Hospital to La Caada carries precious cargo a 2-year-old bone marrow recipient – Los Angeles Times | September 8th, 2017

The Oncology Institute of Hope Innovation welcomes Dr. Homsi to its team of specialists.

Downey, CA (PRWEB) September 08, 2017

The Oncology Institute of Hope Innovation welcomes Dr. Homsi to its team of specialists.

Dr. Yaser Homsi is a passionate and knowledgeable Hematologist that received his Medical education from the University of Aleppo in Aleppo, Syria. After graduation, Homsi moved to Indianapolis, Indiana where he completed his internship, residency and fellowship at the Indiana School of Medicine. Homsi's fellowship training included: Blood and Bone Marrow Stem Cell Transplant, Hematology and Oncology.

Dr. Homsi has completed multiple medical researches including Cellular Therapy and Hematopoietic Stem Cell Transplantation for Cancer and has published papers in various disciplines of Oncology including the Role of Angiogesis in Cancer and The Outcome of the combination of Tacrolimus, Sirolimus and ATF.

Dr. Homsi is Fluent in Arabic.

Professional Memberships:

American Society of Clinical OncologyAmerican Society of HematologyPatient Philosophy:

Dr. Homsi believes in treating each of his patients as individuals as he aids his patients and their families through their treatment plan. He believes strongly in communication and strives to clearly educate all of his patients. He and his staff make every effort to give the best treatment and care possible.

For the original version on PRWeb visit: http://www.prweb.com/releases/2017/09/prweb14669766.htm

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Dr. Yaser Homsi Joins The Oncology Institute of Hope and Innovation - Benzinga

Bone Marrow Stem Cells Comments Off on Dr. Yaser Homsi Joins The Oncology Institute of Hope and Innovation – Benzinga | September 8th, 2017

Grief is a personal matter.Each of us has our own mechanisms to copethere is no format set in stone, there are no boundaries. For me, the week leading up to my son Arjan Vir's birthday has always been the most difficult to deal with.

I am overwhelmed by a well of emotions: On the one hand, there are all those happy memories, so much excitement building up to planning those wonderful birthday parties themes to be decided, lists to be made, cards to be distributed, menus, games and oh, the return gifts one mustn't forget and then this sudden feeling of hollowness, the sinking depths of which words cannot describe.

Beyond words

I lost my 26-year-old son Arjan Vir to Leukaemia in 2012. Arjan was one of those hugely social people with an enviable optimism about him he loved to have people around him and had the enormous ability to attract people, make friends and share his life with them. His friendships were deeply honest and truly meaningful, there was nothing hollow about them. Those around Arjan loved his happy-go-lucky nature and his laidback attitude towards life.

Losing Arjan did not just leave us his family and friends with an irrevocable sense of vacuum, it was felt by the many lives he had touched in some way or the other. Photo:Simi Singh

My son never lost his ability to make friends despite the battle he was fighting with cancer. Arjan had a battalion of friends in the hospital: ward boys, nurses, lab technicians and resident doctors could be seen about his room whenever they had spare time; some asking for advice on which phone to buy, to have the odd computer issue sorted, if nothing else, just to watch him play computer games.

Losing Arjan did not just leave us his family and friends with an irrevocable sense of vacuum, it was felt by the many lives he had touched in some way or the other.

An intensely sensitive child, Arjan worried more about others than himself he was an avid reader, wrote beautiful poetry and had an imagination that went beyond words.

His passion for computer games had pre-determined his career options, he had decided to study computer graphics and 3D computer animation. Even at the hospital, as he underwent treacherous rounds of chemotherapy, cycle after cycle, his imagination worked overtime planning some game or the other based on his treatment.

Knowing BMT

A Leukaemia patient, Arjan needed a bone marrow transplant (BMT). In a layperson's terms, BMT means that the unhealthy bone marrow is killed under highly sanitised conditions by giving the patient very high doses of chemotherapy and radiation and replaced by a healthy bone marrow. That sounds perfectly simple, but bone marrow transplant remains a complicated and dangerous procedure.

What consequences does that come with?

For the uninitiated, bone marrow is the soft tissue where all our vital blood components RBCs, WBCs, platelets, plasma and stem cells are formed. Killing one's bone marrow essentially means there is no immunity left to take care of our body.

Where does the healthy bone marrow come from if we are to attempt to rid the body of cancer?

There are two broad types of BMT: Autologous where the unhealthy bone marrow from our body is removed, worked upon or mutated and replaced, and the allogenic transplant in which another person's healthy bone marrow replaces our own.

With the second type of transplant come incredible complications and the daunting task of finding the donor bone marrow that must replace ours: one needs to find another person whose DNA is identical to ours. The first and most obvious choice, of course, would be a sibling.

However, the chances of finding the identical DNA HLA typing that matches your siblings' is only 1:4, and if such a match isn't possible, where do we go?

In Arjan's case, our younger son's HLA typing did not match, and the chances of finding an unrelated donor match were one in a million.

This was the worst possible news we could get, worse than the news of Arjan being diagnosed with Leukaemia.

How does one find an identical HLA typing match in this whole world where do you start, whom do you turn to?

[Photo: Weill Cornell Medecine]

Discovering stem cell registry

In 2012, there were no substantial HLA typing registries in India unlike in developed countries, which maintain nationwide registries that are linked to the worldwide bone marrow registry.

The doctors guided us to approach All India Institute of Medical Sciences (AIIMS) while AIIMS did not have a significant registry of its own, it had a membership with the World Marrow Donors Association (WMDA), and hence could do a worldwide search to find an HLA match for Arjan.

However, institutes likeAIIMS have become desensitised to the urgency that such cases demand and we got no response from them.

At the time, Datri in Chennai was the sole functioning stem cell registry it had about 12,000 donors in its data bank, but we did not get a quick response from them either.

Our son's doctors here told us that we were sitting on a "time bomb" we needed to act swiftly, we could lose no time and that's when we decided to take Arjan to the US for his further treatment and then, hopefully, a BMT.

Arjan was distressed to discover the situation in India; when he heard about the lack of registries, his first thought was that once he had recovered, he would set up a meaningful registry at home. His biggest concern was: What do the poor do, where do they go?

And so, five years on, the Arjan Vir Foundation was set up in the memory of our very dear son. Our aim is to run a widespread registry that addresses all blood disorders.

We hope to provide assistance at all stages of treatment, recovery, after care, and the rehabilitation and resettlement of patients.

Registering as a donor is easy: any individual over age 18 can become a donor and be a part of the registry till the age of 60, provided they are healthy.

All that one needs is a simple mouth swab test and the consent to donate stem cells when the need arises. The swabs are sent to a highly-specialised laboratory in the US for HLA typing and the results shared with the worldwide registry maintained by WMDA.

Upon finding a match for a patient, the registry contacts the concerned donor.

The process is not complicated, it is exactly like platelet donation, only a few hours longer: avolunteer must undergo a complete medical check-up prior to donating stem cells and is put on stem cell boosting therapy for about four days before the procedure. No incision is involved and the donor does not require hospitalisation.

It just takes one day of your life and busy schedule to save a life.

***

Today, as I sat down to write this article, I also planned another kind of a celebration for Arjan's birthday on September 6: this year, we are holding a camp to bring about awareness about stem cells and register donors at a university in Noida.

Once again there is excitement, albeit of a different kind one held together with a sense of pathos.

Also read: Memories of my mother that Alzheimer's can't wipe clean

Link:
My son died of cancer: Why I'm celebrating his birthday with stem cell awareness - DailyO

Bone Marrow Stem Cells Comments Off on My son died of cancer: Why I’m celebrating his birthday with stem cell awareness – DailyO | September 7th, 2017

MIAMI, Sept. 6, 2017 /PRNewswire/ --Longeveron LLC, a regenerative medicine company developing cellular therapies, announced today that it treated its first patient in the Company's Phase 2b clinical trial evaluating the safety and efficacy of Longeveron human Allogeneic Mesenchymal Stem Cells (LMSCs) in patients with Aging Frailty Syndrome. This trial is being conducted pursuant to an Investigational New Drug Application (IND) in conformance with U.S. Food & Drug Administration (FDA) regulations. Aging Frailty is a common geriatric medical condition that is serious and life-threatening, and for which there are currently no U.S. Food and Drug Administration-approved therapeutics available.

The clinical trial is designed to enroll 120 subjects from approximately 10 medical centers around the U.S. The primary objective of the study is to evaluate the effect that LMSCs have on functional mobility and exercise tolerance in elderly Aging Frailty subjects. Three different LMSC dose groups will be compared to placebo over 12 months in a randomized, double-blinded, parallel arm design.Specifically, the trial will evaluate changes to the following:

"Frailty Syndrome is a very common and difficult situation to manage from a clinician's and caregiver's standpoint," stated Marco Pahor, M.D., Director of the Institute on Aging at the University of Florida. "The goal of intervention is to stop or slow the progression towards dependence and adverse health outcomes common to the syndrome, and to restore the patient to a state of healthy aging and functional independence. Longeveron's regenerative medicine trial is an important step towards the development of an effective therapeutic."

Allogeneic mesenchymal stem cells (MSCs) were previously tested in a Phase I/2 proof-of-concept study conducted by investigators at the University of Miami'sMiller School of Medicine. In that study, MSCs were shown to be safe and well-tolerated in frail, elderly subjects in a Phase 1 open label single ascending dose trial (publication link here) with a similar safety profile observed in the randomized, placebo-controlled Phase 2 study (publication link here) Subjects treated with a dose of 100 million MSCs showed significant improvements in six minute walking distance, and significant decreases in systemic inflammation, both relative to baseline.

"As individuals age, stem cell production and proliferation decreases, systemic inflammation increases, and a person's ability to repair and regenerate worn out or damaged tissue diminishes," remarked Suzanne Liv Page, Longeveron Chief Operating Officer. "In frail individuals this is particularly problematic. Our hypothesis is that exogenously infused allogeneic mesenchymal stem cells that are derived from the bone marrow of a healthy young donor, and culture expanded in our lab, will have potent regenerative and restorative effects."

Participants in this study must be between the ages of 70 and 85, be diagnosed as mildly to moderately frail due primarily to aging, and be able to walk between 200 and 400 meters over six minutes. Detailed information about the trial, subject eligibility and participating centers can be found by clicking here or by visiting the website http://www.clinicaltrials.gov and entering trial ID: NCT03169231.

About LMSCs

LMSCs is an allogeneic product, which means it is produced from stem cells derived from human donor bone marrow, and not from the patient's own stem cells, (referred to as autologous). LMSCs are manufactured at Longeveron's Cell Processing Facility in Miami, Fl. using a proprietary ex vivo culture expansion process.

About Longeveron

Longeveron is a regenerative medicine therapy company founded in 2014. Longeveron's goal is to provide the first of its kind biological solution for aging-related diseases, and is dedicated to developing safe cell-based therapeutics to revolutionize the aging process and improve quality of life. The company's research focus areas include Alzheimer's disease, Aging Frailty and the Metabolic Syndrome. Longeveron produces LMSCs in its own state-of-the-art cGMP cell processing facility. http://www.longeveron.com

Contact:Suzanne Liv Pagerel="nofollow">spage@longeveron.com305.909.0850

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A new therapy changes the balance of osteoblasts (pictured here) and fat cells in the bone marrow, leading to stronger bones.

Science Picture Co/Science Source

By Emma YasinskiSep. 5, 2017 , 2:59 PM

A drug that can reverse diabetes and obesity in mice may have an unexpected benefit: strengthening bones. Experiments with a compound called TNP (2,4,6-trinitrophenol, which is also known as picric acid), which researchers often use to study obesity and diabetes, show that in mice the therapy can promote the formation of new bone. Thats in contrast to many diabetes drugs currently in wide use that leave patients bones weaker. If TNP has similar effects in humans, it may even be able to stimulate bone growth after fractures or prevent bone loss due to aging or disuse.

As more and more patients successfully manage diabetes with drugs that increase their insulin sensitivity, doctors and researchers have observed a serious problem: Thedrugs seem to decrease the activity of cells that produce bone, leaving patients prone to fractures and osteoporosis.

There are millions and millions of people that have osteoporosis [with or without diabetes], and it's not something we can cure, says Sean Morrison, a stem cell researcher at University of Texas Southwestern in Dallas. We need new agents that promote bone formation.

Morrison and his colleagues have shown that a high-fat diet causes mice to develop bones that contain more fat and less bone. The diet increased the levels of leptina hormone produced by fat cells that usually signals satiety in the brainin the bone marrow, which promoted the development of fat cells instead of bone cells. That suggests that nutrition has a direct effect on the balance of bone and fat in the bone marrow.

After reading Morrisons work, Siddaraju Boregowda, a stem cell researcher at the Scripps Research Institute in Jupiter, Florida, was reminded of genetically altered mice that dont gain body fat or develop diabetes, even when fed high-fat diets. He and his boss, stem cell researcher Donald Phinney, wondered whetherthose mice were also protected from the fattening of the bone marrow that accompanies a high-fat diet.

They contacted Anutosh Chakraborty, a molecular biologist who was studying such mice down the hall at Scripps at the time. The animals lack the gene for an enzyme called inositol hexakisphosphate kinase 1 (IP6K1), which is known to play a role in fat accumulation and insulin sensitivity. The scientists suspected that the lost enzyme might affect the animals' mesenchymal stem cells (MSCs)stem cells found in the bone marrow that are capable of developing into both thebone cells and fat cells that make up our skeletons. If too many fat cells develop, they take the place of bone cells, weakening the bone.

The researchers fed genetically altered and normal mice a high-fat diet for 8weeks. Not only did the genetically altered mice develop fewer fat cells than their normal counterparts, but their production of bone cells was higher than that of the normal mice, the team reported last month in Stem Cells.

The scientists then set out to see whetherthey could use a drug to achieve the same effect in normal mice. For 8weeks, they fed normal mice a high-fat diet and gave them daily injections of either TNP, a well-known IP6K1 inhibitor, or a placebo. When they analyzed the animals bones and marrow, they found that mice that had received TNP had significantly more bone cells, fewer fat cells, and greater overall bone area. The IP6K1 inhibitor apparently protected the mice from the detrimental effects of the high-fat diet.

The study provided thesurprising result that one new therapy currently being explored to lower insulin resistance promotes, rather than decreases, the formation of bone in mice, says DarwinProckop,a stem cell researcher at Texas A&M College of Medicine in Temple, who was not involved in the work.

The researchers still need to figure out how to deliver TNPs effects only to MSCs, instead of the entire body, given that it sometimes blocks other enzymes along with IP6K1. Inhibition of IP6K1 is a promising target for patients with both diabetes and obesity, Boregowda says. He says he and his colleagues are now enthusiastic about testing their findings in a wide range of bone-related diseases and disorders. It might even help heal broken bones, he speculates.

Phinney, on the other hand, is aiming even higher. He wonders whetherthe therapy could also be useful for space travel, because bones are especially vulnerable to deterioration in zero gravity. Its a whole new field of science and drug discovery.

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Every mom anticipates her childs first day at kindergarten.

For Jessica Pequeno, that kind of milestone is something this mom is only now ready to imagine.

The last time the Napa Valley Register wrote about the Pequeno family, it was October 2015. Their then 22month-old toddler Xavier was about to begin the fight of his life against an immune deficiency disorder so rare it had no name.

Today, there is good news about Xavier and his family.

The now 4-year-old completed a grueling, yet successful stem cell transplant, just started his second year of preschool and is making progress with his health, said his mother. Come this time next year, shell be enrolling him in kindergarten.

Every day is different. We still deal with so many unknowns, she said. But, Hes doing so much better than anybody would have ever expected.

Just getting to this point was a long road.

Two years ago, the Pequenos told their story in hopes of finding a bone marrow transplant match for their son. Doctors originally told them there was no match within the family. Fortunately, after a second set of tests, the Pequenos middle son, Higinio Pequeno IV, was identified as a partial match.

That news was awesome, said Jessica Pequeno.

The family prepared for the transplant to take place in June 2015, but a stubborn infection put those plans on hold. By December, his health care team at the University of California at San Francisco wouldnt even give us odds as his percentage of survival because they didnt know, she said.

They finally had a name for his disease IKBa gain of function mutation with ectodermal dysplasia but there were too many unknowns.

Pequeno said she realized the stem cell transplant was a kind of a now-or-never situation.

We just kept saying, We just have to keep doing this. Giving up wasnt an option.

On Dec. 1, 2015, Xavier was admitted to the hospital for the transplant. The process began with eight days of chemotherapy followed by the stem cell transplant.

Putting a line in his femoral artery, blood was collected from Higinio, then 10. Then a machine separated the stem cells from the blood. Higinios stem cells were then given to Xavier. The stem cells were put into a vein, much like a blood transfusion. The stem cells are then meant to travel to the bone marrow, engraft, and hopefully begin making new, normal blood cells.

On the day of the transplant, the whole Pequeno family, including her husband Higinio, son Higinio and daughter Maya and Jessica Pequenos mom were there. Seeing those potentially life-saving cells go into her son was very emotional, said Jessica Pequeno.

We all cried, she said. It was really scary, but you cant stop. You have to keep going.

During the procedure, Xavier was awake, she said. But the side-effects of the chemotherapy were starting to set in. His hair was falling out, and he had stopped eating and drinking because his mouth sores were so bad and painful, she said. He was on morphine for the pain.

The waiting began. Would the stem cell transplant be a success?

The family was told that Xavier would likely spend many months in the hospital. We planned to be separated as a family for at least six months, said Pequeno. We just expected it to be really hard.

She spent her nights in the room with her son, sleeping on a blow-up twin mattress. The rest of her family went back to Napa. Because Jessica was unable to work and her husband couldnt work because he needed to have knee surgery, the family had moved in with Jessicas mother.

Meanwhile, doctors continued to check Xaviers blood to see if his body was responding to the stem cell transplant.

Every day Id ask, Where are we at? his mother said.

And then, one day in early January, the doctors came to see Xavier, and they said, We have good news.

The transplant was starting to work and the new cells were starting to grow, she said.

I cried, said Pequeno. It happened so much faster than what they had expected.

By the end of January, Xavier was well enough to go home to Napa.

It was scary to come home and super exciting, she said.

Back at home, a new routine was created. Xavier was still taking 25 different medications, some multiple times per day. He had a gastrostomy or G tube for feeding the nutritional liquid he eats and a central line a thin, flexible tube used to give medicines, fluids, nutrients, or blood products over a long period of time.

Honestly I dont remember a lot of it. It becomes a big blur, said Pequeno.

The family continued to visit UCSF at least once a week for blood counts and other checks. There were more ups and downs. Infections and illnesses caused him to be hospitalized for days at a time in February, May and June. His central line got infected. He got shingles.

His immune system was still really weak, said Pequeno.

But he kept bouncing back.

Just two weeks ago, doctors finally removed his central line.

It was a huge step, she said.

Challenges remain. Before the stem cell transplant, Xavier had about 5 to 10 percent of a normal immune system. Now he has about 60 to 70 percent, doctors said.

Were starting to learn hes really prone to sinus and respiratory infections, and viruses, said Pequeno. His body just doesnt fight like everyone elses.

Other habits are harder to change.

Before Xavier went to preschool, Pequeno and her family were able to carefully control his exposure to germs.

When he was able to go to preschool, I wasnt in control of those environments anymore. Its really hard. It gets easier, but it takes a while to learn how to kind of let go, she said.

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Today, Xavier takes only six medications a day and can receive infusions of antibodies at home. Visits to UCSF have been cut back to once every four to five weeks.

Developmentally, Xavier is doing well, she said.

He has some hearing loss, which we continue to monitor. Its hard to say if its a side effect of chemo or other drugs. Right now it doesnt affect his speech. He also has skin, hair and teeth health issues to manage.

Xavier doesnt complain at lot, said Pequeno.

Hes always handled everything so well. When he suffered, He would get quiet. Even now when hes not feeling good, instead of crying like many small children would, Xavier is quiet.

Financially, its hard because Im still not able to work, she said.

Xaviers medical care is provided by Partnership HealthPlan/Medi-Cal and California Childrens Services. Her husband went back to work. Pequeno is taking classes at Napa Valley College while her son is in preschool.

I want to be a nurse but I want to go into pediatrics I want to teach parents how to advocate for their kids.

One of the most significant changes for Pequeno was becoming more confident in working with health care providers regarding her sons care.

Nobody could hand me a book when this started (that said) these are the things you need to know and questions to ask. No one told me I was the captain of his team. Her confidence grew. You have to get comfortable in that role.

The past several years have left a lasting imprint on the whole family, she said. Signs of post-traumatic stress have been seen in all family members. Learning coping skills and how to manage stress is important.

Especially for their son Higinio, said his mother. Its not easy for young boy to come to terms with what his brother went through and his own unique contribution.

I dont think any 10-year-old is capable of understanding the weight that carries, she said.

The struggles havent ended, said Pequeno.

Weve just learned to manage them and adjust and deal with the financial part. We juggle. You learn how to change your priorities.

Its easy to say her son looks healthy, said Pequeno, but thats also frustrating because it takes so much work to get him to continue to look like that.

It definitely takes a toll and lot of work and sacrifice to keep him where hes at, she said.

And Xaviers condition isnt going away, she noted. This is something we will manage for the rest of his life one way or the other.

People say, Oh youre so strong. But I think that as a mom, you just do it, said Pequeno. You pull the strength from somewhere. Because you dont give up on your kids.

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Kathmandu, September 4

Civil Hospital is launching haploidentical stem cell transplant within a few months.

Its a treatment process for patients with blood-related cancers and certain blood disorders.

Patients who need a stem cell transplant and cant find a donor who matches their tissue type will benefit from the transplant. Haploidentical transplant is a modified form of stem cell transplant in which a healthy first degree relative a parent, or sibling can often serve as a donor.

When no matched donor is available, half-matched related (haploidentical) donors are safely used in stem cell transplantation, informed Dr Bishesh Poudyal, associate professor and chief of Clinical Hematology and Bone Marrow Transplant Unit at the hospital.

The cost of the transplant will be around 12 to 15 lakh rupees. People suffering from blood cancer, aplastic anaemia, sickle cell anaemia and thalassemia will benefit from the transplant.

The hospital has been performing allogeneic and autotransplant stem cell transplant where only siblings can be donors.

Nine patients had undergone autotransplant and one had undergone allogeneic stem cell transplant in the hospital after it started bone marrow transplant in the hospital in 2016.

A version of this article appears in print on September 05, 2017 of The Himalayan Times.

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TEL AVIV, Israel, Sept. 5, 2017 /PRNewswire/ -- Cellect Biotechnology Ltd. (NASDAQ: APOP), a developer of stem cells selection technology, announced today that theU.S. Food and Drug Administration(FDA) has granted orphan drug designation for Cellect's ApoGraft for the prevention of acute and chronic graft versus host disease(GvHD) in transplant patients.

GvHD is a transplant associated disease representing an outcome of two immune systems crashing into each other. In many transplantations from donors, and especially in Bone Marrow Transplantations (BMT), the transplanted immune mature cells (as opposed to stem cells) attack the host (patient receiving the transplant) and create severe morbidity and in many cases even death.

This disease happens as a result of current practices being unable to separate the GvHD causing cells from the much needed stem cells.Cellect's ApoGraft was designed to eliminate immune responses in any transplantation of foreign cells and tissues.

Cellect's AppoGraft technology can be utilized already today to help thousands of development and research centers globally engaged in adult stem cells based therapeutics by providing them with a simplified and cost efficient enriched stem cells for use as a raw material for a wide range of stem cells based therapeutics R&D. Before Cellect's ApoGraft, such procedures were extremely complex, inefficient and required substantial resources in both cost, time and infrastructure requirements. ApoGraft can now be used to significantly advance the use of stem cells across multiple therapeutics indications as well as research and biobanking purposes.

The FDA Orphan Drug Act provides incentives for companies to develop products for rare diseases affecting fewer than 200,000 people inthe United States. Incentives may include tax credits related to clinical trial expenses, an exemption from theFDAuser fee, FDAassistance in clinical trial design and potential market exclusivity for seven years following approval.

About Cellect Biotechnology Ltd.

Cellect Biotechnology (NASDAQ: "APOP", "APOPW") has developed a breakthrough technology for the selection of stem cells from any given tissue that aims to improve a variety of stem cell applications.

The Company's technology is expected to provide pharma companies, medical research centers and hospitals with the tools to rapidly isolate stem cells in quantity and quality that will allow stem cell related treatments and procedures. Cellect's technology is applicable to a wide variety of stem cell related treatments in regenerative medicine and that current clinical trials are aimed at the cancer treatment of bone marrow transplantations.

Forward Looking Statements

This press release contains forward-looking statements about the Company's expectations, beliefs and intentions. Forward-looking statements can be identified by the use of forward-looking words such as "believe", "expect", "intend", "plan", "may", "should", "could", "might", "seek", "target", "will", "project", "forecast", "continue" or "anticipate" or their negatives or variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical matters. For example, forward-looking statements are used in this press release when we discuss the Company's pathway for commercialization of its technology. These forward-looking statements and their implications are based on the current expectations of the management of the Company only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. In addition, historical results or conclusions from scientific research and clinical studies do not guarantee that future results would suggest similar conclusions or that historical results referred to herein would be interpreted similarly in light of additional research or otherwise. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications, which could cause the actual results or performance of the Company to differ materially from those contemplated in such forward-looking statements. Any forward-looking statement in this press release speaks only as of the date of this press release. The Company undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. More detailed information about the risks and uncertainties affecting the Company is contained under the heading "Risk Factors" in Cellect Biotechnology Ltd.'s Annual Report on Form 20-F for the fiscal year ended December 31, 2016 filed with the U.S. Securities and Exchange Commission, or SEC, which is available on the SEC's website, http://www.sec.gov. and in the Company's period filings with the SEC and the Tel-Aviv Stock Exchange.

ContactCellect Biotechnology Ltd. Eyal Leibovitz, Chief Financial Officerwww.cellect.co+972-9-974-1444

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At just four-years-old, Erica Honoways son has gone through more than most people will experience in a lifetime.

In February 2016, the family received devastating news, her son Lincoln had been diagnosed with bone marrow failure. He was just three years old at the time.

Lincoln needed a bone marrow transplant, and doctors were only able to find two matches in the entire world. The first donor fell through, so Lincoln was left with only one option.

It was terrifying. We didnt know what we were dealing with, Honoway said. We didnt know what the chances were they would find a match for him. Even if they did, we didnt know if he would make it through the transplant, so it was the scariest experience of our lives.

After the blood transfusions, chemotherapy, radiation and bone marrow transplant, Lincoln is now a happy and active four year old, all thanks to an unknown hero.

This person has just been our angel, Honoway said. We love her and we dont even know her. We say her We have a feeling its a woman but we dont know anything about this person. We dont know where in the world they live, we dont know if its a man or a woman, we dont know anything. But all we know is that they are our hero.

Honoway added that they must wait a minimum of two years before they can meet the donor.

Lincolns successful transplant was the reason Honoway and her family were supporting the University of Reginas Get Swabbed event on Monday, to encourage students between the ages of 17 and 35 to get their cheeks swabbed and enter a national stem call database.

I heard about Erica and Lincoln and I just thought it was amazing how someone just saved his life, and she doesnt even know who he is or who she is, I just think its amazing, U of R Stem Cell Club president Sylvia Okonofua said. I felt like if I take up this initiative and actually run drives where people [can get] on the stem cell registry, [it can] help save a life someday.

Getting students involved and realizing their impact of their involvement through something like this was one of the main goals, U of R student engagement co-ordinator Doug OBrien said. Another goal of having todays Get Swabbed initiative was obviously to support the stem cell database for Canada and through the One Match program.

Approximately 80 students took part in Mondays Get Swabbed event, and organizers are hoping to increase that number for the next event on Sept. 14.

Its a simple way to help save a life.

I hope people realize that they have the opportunity to save someones life, imagine what that would feel like, Honoway said. Youd get to know forever that you saved another humans life. Its pretty special.

2017Global News, a division of Corus Entertainment Inc.

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Scanning electron micrograph of blood cells. From left to right: human erythrocyte, thrombocyte (platelet), leukocyte. Credit: public domain

For more than a century, physicians have anecdotally noted that patients with an underactive thyroidoften caused by iodine deficiencytended to also have anemia. But the link between thyroid hormone and red blood cell production has remained elusive. That is, until two postdoctoral researchers in the lab of Whitehead Institute Founding Member Harvey Lodish, Xiaofei Gao and Hsiang-Ying "Sherry" Lee, decided to investigate.

During the development of red blood cells, specialized bone marrow stem cells mature through several stages until they finally turn on the genes for hemoglobin and other red blood cell proteins and become mature red blood cells. In order to simulate this process in the lab, researchers have previously found that culturing blood cell progenitors in serum helps them turn on all of the proper proteins to take the final step and become a red blood cell.

Gao and Lee, now Principal Investigators at Westlake Institute for Advanced Study and Peking University, respectively, wondered if something in the serum was key to flipping the switch to becoming a mature red blood cell. To narrow down which of the molecules in the serum is the trigger, Gao and Lee ran the serum through a standard laboratory filter that many of us use everyday for our tap water: charcoal.

Long known for sucking odors out of the air and flavors from water, charcoal attracts and retains hydrophobic (water repellent) molecules. Gao and Lee noticed that once filtered, the serum no longer supported red blood cell production; they deduced that one of the hydrophobic molecules trapped by charcoal is the key to the final step of red blood cell maturation. Gao and Lee determined that when just the thyroid hormone thyroxin is added back to the serum, the red blood cell progenitors once again start down the path to maturation. Thyroid hormone's role is so important in stimulating red blood cell maturation, they discovered, that if it is added at an earlier stage of development, red blood cells short-circuit their usual developmental processes and begin turning into mature red blood cells.

Gao and Lee then teased apart the mechanism behind thyroid hormone's effect on red blood cell maturation. They pinpointed the specific type of receptor inside maturing red blood cells to which thyroid hormone binds. From there, they identified a protein that is necessary for thyroid hormone stimulation and that acts as a regulator of the final step of red blood cell production.

With this better understanding of the connection between thyroid hormone and red blood cell maturation, scientists may be able to identify new therapies that trigger red blood cells maturation in patients with specific types of anemia, including those with an underactive thyroid.

The study is published in PNAS.

Explore further: Low thyroid hormone before birth alters growth and development of fetal pancreas

More information: Xiaofei Gao et al. Thyroid hormone receptor beta and NCOA4 regulate terminal erythrocyte differentiation, Proceedings of the National Academy of Sciences (2017). DOI: 10.1073/pnas.1711058114

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The US Food and Drug Administration (FDA) intendsto investigate the use of unproven stem cell therapies being offered in the country'sclinics.

Tighter enforcement from the FDA comes as an inspection at StemImmune Inc based in San Diego, California, revealed the use of potentially dangerous treatments administered to vulnerable cancer patients.

Only a small number of stem cell treatments are currentlyFDA approved, including use of bone marrow transplants in cancer patients and cord blood for specific blood-related disorders.However stem cell treatments using only the patient's own cells are not subject to the same level of regulation as drugs if the cells are only 'minimally manipulated'.

FDA commissioner Dr Scott Gottlieb said in a statement:'The FDA will not allow deceitful actors to take advantage of vulnerable patients by purporting to have treatments or cures for serious diseases without any proof that they actually work. I especially wont allow cases such as this one to go unchallenged, where we have good medical reasons to believe these purported treatments can actually harm patients and make their conditions worse.'

Five vials,each containing 100 doses of the live Vaccinia Virus Vaccine, were seized from StemImmune Incby US marshals on25August 2017.

The vaccine, which is usedagainst smallpox, and is not commercially available was combined with stem cells derived from body fat to create an unapprovedtherapy. The concoction was injected directly into tumours of cancer patients at California Stem Cell Treatment Centres in Rancho Mirage and Beverly Hills.

The effects of the vaccine in immunocompromised cancer patients have the possibility to cause severe complications such as inflammation and swelling of the heart and surrounding tissues.

In a separate case, awarning letter was also sent to chief scientific officerKristin Comellaat US Stem Cell Clinic in Sunrise, Florida, after three patients with macular degeneration were blinded following the use of unapproved stem cell injections into their eyes, in a sponsored study (see BioNews 893). The letter lists a number of non-compliance to procedures and 'significant deviations' to current good manufacturing practice and good tissue practice.

'Our actions today should also be a warning to others who may be doing similar harm, we will take action to ensure Americans are not put at unnecessary risk,' Dr Gottlieb commented. 'I also urge health care providers, patients and consumers to report these kinds of activities or any adverse events associated with these unproven treatments to the agency through MedWatch a safety reporting programme.'

Professionals in the field blame the past lack of FDA attention for the widespread problem and are calling for stringent regulation. ProfessorLeigh Turner, fromthe Centre for Bioethics at the University of Minnesota, told CNN: 'This is a space where the FDA could have taken action four or five years ago as far as making this a policy priority.'

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Irish researcher Prof Daniel Kelly has secured 150,000 in funding to develop a novel implant for treating cartilage damage.

As a recipient of one of the European Research Councils Proof of Concept grants, Prof Daniel Kelly will now spend the next 18 months developing his 3D-printed project entitled Anchor.

Using the 150,000, Kelly will look to develop and commercialise his new medicinal product for cartilage regeneration, employing a postdoctoral researcher to help.

Those active in many sports would be familiar with cartilage damage as a result of injury, of which many cases occur in the knee joint. If left untreated, it can lead to difficulties such as osteoarthritis (OA).

OA can be a debilitating condition, with 80pc of those over the age of 60 experiencing limitations in movement and 25pc saying they cannot perform their major daily activities, according to the World Health Organisation.

Kellys product uses 3D-printed, biodegradable polymer components to make a scaffold, which acts as a template to guide the growth of new tissue by recruiting endogenous bone marrow derived from stem cells.

This, Kelly believes, gives it a competitive edge over similar implants, as standard ones are designed with a finite lifespan, making them unsuitable for younger patients with OA.

Kelly, a principal investigator at AMBER the Trinity College Dublin materials science research centre explained why it could be a major breakthrough for other conditions, such as arthritis.

Our 3D-printed polymer posts will anchor the implant into the bone and will be porous to stimulate the migration of stem cells from the bone marrow into the body of the scaffold, he said.

While various scaffolds like this have been available for some time, they have had limited success, partly because scaffolds need to be anchored securely due to the high forces experienced within the joint. Our 3D-printed posts overcome this problem.

Prior to Anchor, Kelly had worked on this technology in previous projects, such as the ERC-funded StemRepair project to develop a range of porous cartilage-derived scaffolds, and JointPrint.

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Irish researcher bags 150000 to make 3D-printed knee implant - Siliconrepublic.com

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Drugs developed to treat heart and blood vessel problems could be used to treat leukaemia

Drugs developed to treat heart and blood vessel problems could be used in combination with chemotherapy to treat an aggressive form of adult leukaemia.

Researchers at the Francis Crick Institute, Kings College London and Barts Cancer Institute discovered that acute myeloid leukaemia (AML) causes bone marrow to leak blood, preventing chemotherapy from being delivered properly.

Drugs that reversed bone marrow leakiness boosted the effect of chemotherapy in mice and human tissue, providing a possible new combination therapy for AML patients.

To study how AML affects bone marrow, the researchers injected mice with bone marrow from AML patients. Later, they compared their bone marrow with healthy mice using a technique called intravital microscopy that allows you to see biological processes in live animals. They found that pre-loaded fluorescent dyes leaked out of the bone marrow blood vessels in AML mice, but not healthy mice.

Next, the team tried to understand what caused the bone marrow in AML mice to become leaky by studying molecular changes in the cells lining the blood vessels. They found that they were oxygen-starved compared to healthy mice, likely because AML cells use up a lot of oxygen in the surrounding tissue. In response to a reduction in oxygen, there was an increase in nitric oxide (NO) production a molecule that usually alerts the body to areas of low oxygen.

As NO is a muscle relaxant, the team suspected that it might be causing bone marrow leakiness by loosening the tight seams between cells, allowing blood to escape through the gaps. By blocking the production of NO using drugs, the team were able to restore bone marrow blood vessels in AML mice, preventing blood from leaking out. Mice given NO blockers in combination with chemotherapy had much slower leukaemia progression and stayed in remission much longer than mice given chemotherapy alone.

When the vessels are leaky, bone marrow blood flow becomes irregular and leukaemia cells can easily find places to hide and escape chemotherapy drugs, said researcher Dr Diana Passaro. Leaky vessels also prevent oxygen reaching parts of the bone marrow, which contributes to more NO production and leakiness.

By restoring normal blood flow with NO blockers, we ensure that chemotherapy actually reaches the leukaemia cells, so that therapy works properly, she added.

In addition to ensuring that chemotherapy drugs reach their targets, the team also found that NO blockers boosted the number of stem cells in the bone marrow. This may also improve treatment outcomes by helping healthy cells to out-compete cancerous cells.

The team also found that bone marrow biopsies from AML patients had higher NO levels than those from healthy donors, and failure to reduce NO levels was associated with chemotherapy failure.

Our findings suggest that it might be possible to predict how well people with AML will respond to chemotherapy, said Dr Dominique Bonnet, senior author of the paper and Group Leader at the Francis Crick Institute.

Weve uncovered a biological marker for this type of leukaemia as well as a possible drug target. The next step will be clinical trials to see if NO blockers can help AML patients as much as our pre-clinical experiments suggest.

We found that the cancer was damaging the walls of blood vessels responsible for delivering oxygen, nutrients, and chemotherapy. When we used drugs to stop the leaks in mice, we were able to kill the cancer using conventional chemotherapy, said Dr Passaro. As the drugs are already in clinical trials for other conditions, it is hoped that they could be given the green light for AML patients in the future.

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Chemo-boosting drug discovered for leukaemia - Drug Target Review - Drug Target Review

Bone Marrow Stem Cells Comments Off on Chemo-boosting drug discovered for leukaemia – Drug Target Review – Drug Target Review | September 4th, 2017