Life Blood
Information from BBC Actionline
 
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In Life Blood, Horizon looks at the work of Dr Joanne Kurtzberg and staff at Duke University Hospital, North Carolina, USA, on a pioneering new treatment for children with blood diseases, in particular childhood leukaemia.
Please note that advice or information provided on this site should not be considered a substitute for advice from a qualified medical practitioner.
Cord blood transplant treatment
The treatment involves replacing the patient's diseased blood with blood from the placenta and umbilical cord of a new born baby. Previously, after birth, cord blood was disposed of until it was discovered that, although small in quantity, it was rich in blood stem cells which form the basis of new blood cells. The treatment is seen as a potential alternative to a bone marrow transplant in patients for whom no suitable bone marrow donor can be found and involves the destruction of diseased blood by chemotherapy and its replacement by cord blood from which new blood cells can grow.
Stem cells are the building blocks for new blood cells and if a cord blood transplant is successful it has the effect of rebuilding the patient's blood system from scratch.
Bone marrow was at one time thought to be the only source of blood stem cells. It is now known that close to the moment of birth, blood stem cells travel from the baby's liver to its bone marrow and are present in the small amount of blood left in the placenta and umbilical cord for possible use in transplants, where no bone marrow match can be found.
Cord blood transplants
Unlike a bone marrow transplant, cord blood transplants do not require a compatible donor. Compatibility in bone marrow transplants has nothing to do with blood groups but with genes.
Scientists have to find 12 key genes in the white blood cells that have to be the same in both donor and patient to achieve what is termed the perfect match. Cord blood transplants do not need this perfect match compatibility but at present cord blood cannot be mixed and as the amount of blood containing blood stem cells is very small this type of transplant has been more successful in children than adults who require larger numbers of blood stem cells to grow a new blood system.
Research is under way to attempt to artificially expand the blood stem cells in cord blood to overcome this difficulty. Meanwhile bone marrow transplants are still extremely important for both adults and children in the treatment of leukaemia and the present position is that cord blood transplants are seen as complementary to bone marrow transplants and not as a replacement.
Bone marrow
Bone marrow is a spongy material that fills the bones and produces the cells which develop into the three different types of blood cells:
red blood cells which carry oxygen to all the cells in the body
white blood cells which are essential for fighting infections
platelets which help the blood to clot and thereby control bleeding
All these cells normally stay inside the marrow until they are mature enough to circulate in the blood and perform their function properly.
White blood cells
The bone marrow produces two main types of blood cell:
granulocytes, produced from the myeloid cells in the bone marrow
lymphocytes
These cells work together to fight infection. The fully developed white cells circulate around the body in the blood.
Lymphocytes are also found in the lymph glands and in the lymphatic system, which is part of the body's natural defence against infection. The lymphatic system is made up of the tonsils, liver, spleen and the lymph glands. There are lymph glands in the neck, under the armpits, in each groin, and in the chest and abdomen. The spleen is under the ribs on the left hand side of your abdomen.
Bone marrow transplants
Bone marrow is the spongy tissue found in the cavities of the body's bones where all of the body's blood cells are produced. Every type of blood cell in the marrow begins its life as a stem cell. The stem cells then divide and form the different cells that make up your blood and your immune system. These include white cells that fight infection (leukocytes), red cells that carry oxygen (erythrocytes), and platelets, which are clotting agents.
Without bone marrow, and the disease-fighting blood cells it produces, your immune system is severely impaired and you have little defence against even the most common infection. You can see then why any change in bone marrow function can be life-threatening.
The goal of a bone marrow transplant is to cure many different types of cancer and diseases. The type of transplant you have depends on who donates the marrow.
Types of bone marrow transplant (BMT)
Allogeneic transplant - the person donating the bone marrow or stem cells is a genetically-matched family member (usually a brother or sister).
Unrelated allogeneic transplant - the person donating the marrow is unrelated to the patient. The chances of finding an unrelated compatible donor from the general population depend on the uniqueness of your tissue type. Genetic and ethnic background can also affect the likelihood of finding a donor.
Autologous transplant - the patient donates his/her own bone marrow or stem cells prior to treatment for re-infusion later.
Peripheral blood stem cell transplant - the patient or a donor donates stem cells collected from the circulating blood system instead of from bone marrow. The only difference with this kind of BMT is the source of the stem cells and how they are collected.
Your doctor will discuss what type of transplant is advisable for you; and the corresponding success rate. Many factors determine the odds, including the disease being treated, previously attempted treatments and the types of bone marrow donation.
Leukaemia
Leukaemia is a cancer of the white blood cells which are produced by the bone marrow. The four main types of leukaemia are chronic myeloid (CML), chronic lymphocytic (CLL), acute lymphoblastic (ALL) and acute myeloblastic (AML). Each disease has its own characteristics and treatment.
Symptoms of leukaemia
Because the disease progresses slowly, it is difficult to detect in its early stages. Some people have no symptoms and the disease may be discovered only when a blood test is taken for a different reason.
The symptoms of leukaemia can include:
frequent infections
tiredness
bleeding and bruising
swollen lymph glands
swollen abdomen
sweating or fever at night
weight loss
People with leukaemia are more prone to infections because they have a shortage of healthy white blood cells to fight off the bacteria and viruses.
A lack of red blood cells (anaemia) causes tiredness and sometimes breathlessness. There are not enough red blood cells because the abnormal white blood cells are taking up too much space in the bone marrow. Your platelet count may be low too, for the same reason. This can cause unexplained bruising or bleeding, such as nose bleeds.
Abnormal lymphocytes may collect in the lymph glands and cause painless swelling in your neck, armpits or groin. Your spleen may become enlarged and cause a tender lump in the upper left-hand side of your abdomen. Sweating or fever at night can also sometimes occur. Some people lose weight.
Treatment
Some people with leukaemia may never need treatment. (This mainly applies to
patients with chronic lymphocytic leukaemia whose illness is not causing
any symptoms and is progressing only very slowly.) Patients with chronic myeloid leukaemia normally receive treatment from soon after diagnosis and
all patients with acute leukaemia will require some form of treatment. For
younger patients (under about 60 years) with acute leukaemia, treatment is
normally aimed at attempting to eliminate the leukaemia cells and this may
achieve a cure. Older patients may not be fit enough to receive high doses
of treatment and their care will be designed to offer the best quality
of life.
The success rate for treatment is highest in childhood acute lymphoblastic
leukaemia (approaching 80% cure rate). In children with acute myeloid
leukaemia the cure rate is around 50%; for adults the cure rate is
highly variable depending on the patient's age, the exact type of leukaemia
and the response to therapy. The only treatment generally considered able
to cure chronic myeloid leukaemia is a successful donor stem cell
transplant.
The key element of treatment of leukaemia is chemotherapy, the use of drugs
to kill off or control numbers of leukaemia cells. Depending on the type of
leukaemia, this may be given in high doses (while patients are in hospital) or in low doses as an oral treatment. The side-effects are very variable
depending on the types of drugs used and the dosage. Your specialist will
decide along with you what is the best form of treatment.
Radiotherapy is sometimes used to treat bulky enlarged lymph nodes, or
an enlarged spleen. Alternatively, a large spleen may be removed surgically.
Only a minority of patients with leukaemia will receive radiation or
chemotherapy. Radiation to the central nervous system may be given to kill
off residual leukaemia cells in some children with leukaemia. There are
studies trying to determine whether this can be replaced with injections of drugs directly into the fluid of the central nervous system.
Some younger patients are offered treatment with high dose chemotherapy and
stem cell transplant. This treatment remains experimental, but may result
in long periods of remission (ie no active disease) for some people. In some
cases a successful donor (allogeneic) transplant may cure the condition. It
is important to stress that stem cell transplants are only appropriate for
a minority of patients.
A number of new treatment approaches are being studied using, for example,
special antibodies or other approaches to recruit the immune system to
fight the leukaemia or using treatments designed to make the leukaemia
cells mature and die like normal cells.
Contact information for support organisations
The Anthony Nolan Bone Marrow Trust
www.anthonynolan.org.uk/
The trust maintains a register of volunteers willing to donate bone marrow in circumstances where a match cannot be found within a person's family. For further information on the work of the Trust, or to find out how to become a donor, contact:
The Royal Free Hospital
Hampstead
London NW3 2QG
0901 88 22 234 (calls cost 25p per minute)
The phone line is a recorded message. Callers can leave details to be sent a bone marrow donor information pack or can make a credit card donation.
British Bone Marrow Registry
Contact:
National Blood Service
Southmead Road
Bristol BS10 5ND
0117 991 2068
peter.heard@nbs.nhs.uk
British Bone Marrow Donor Appeal
Aims to promote and support medical organisations which make a practical contribution towards of the Register of tissue types of potential bone marrow donors for persons suffering from leukaemia and other bone marrow related diseases.
18 Warwick Street
Rugby
Warwickshire
CV21 3DD
01527 518 588
Leukaemia Research Fund
http://dspace.dial.pipex.com/lrf-/
info@lrf.org.uk
Can provide free patient literature and background information. People should ask to speak with the clinical information officer.
43 Great Ormond Street
London WC1N 3JJ
020 7405 0101
Open 9.30am-5.30pm
Leukaemia Care Society
www.leukaemiacare.org/
enquiries@leukaemiacare.org.uk
The Society provides information for sufferers of the leukaemias, Hodgkin's and other lymphomas, myelodysplasia and aplastic anaemia.
2 Shrubbery Avenue
Worcester WR1 1QH
01905 330003 or 0845 7673203 (local rate)
CancerBACUP
www.cancerbacup.org.uk/
Can provide up to date information, practical advice and support for cancer patients and their families. Their free cancer information service is staffed by qualified and experienced cancer nurses in local centres in hospitals throughout the UK.
Head Office:
3 Bath Place
Rivington Street
London EC2A 3JR
freephone 0808 800 1234
Contacts who appear in Horizon
Prof Hal Broxmeyer, the doctor in the film who developed the science, which led to the first ever cord blood transplant
Walther Oncology Center
Rm 302, Indiana University School of Medicine
1044 West Walnut Street
Indianapolis, Indiana
IN 46202
USA
Prof Eliane Gluckman was a member of the team of physicians that performed the first cord blood stem cell transplant in 1988 on a child with Fanconi's anaemia, at Hôpital Saint-Louis, Paris in 1988.
Université Hôpital Saint-Louis (Paris)
1, Avenue Claude Vellefaux
75475 PARIS
Cedex 10
France
Prof Joanne Kurtzberg, one of the programme's main contributors and a pioneer of the technique discussed. Her work is based at Duke University Medical Center. Contacts there are as follows:
Bridget Archambault, Barbara Chance, Connie Stephens, Linda Issitt, Prof Joanne Kurtzberg.
Pediatric BMT Office
Box 3350, Duke University Medical Center
Durham, North Carolina
NC 27710
USA
www.mc.duke.edu/
Prof Frank Smith performs cord blood transplants and is an expert who comments on the science in the film.
Children's Hospital Medical Center
Division of Hematology/Oncology
R2303, 3333 Burnet Avenue
Cincinnati, Ohio
OH 45229-3039
USA
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