SUMMARY OF THE CORD BLOOD STEM CELL GROUP

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SUMMARY OF THE CORD BLOOD STEM CELL GROUP Powered By Docstoc
					     Cord Blood Stem Cell Therapy Programme – Professor Colin P.McGuckin
                  Professor of Regenerative Medicine, University of Newcastle upon Tyne
                       c.mcguckin@newcastle.ac.uk or c_mcguckin@hotmail.com
                            Mobile +44 07971 266764; Office +44 191 222 7785


                                  Professor Colin McGuckin

              Advanced cell therapy – European legislation and discussion.


Index:
A. WHY STEM CELLS?:
B. WHAT ARE STEM CELLS?:
C. WHY ARE EMBRYONIC STEM CELLS NOT READY FOR TREATMENT?:
D. WHY ARE CORD BLOOD STEM CELLS GOOD TO USE?:
E. HYPE AND REALITY - WHY ARE STEM CELLS NOT USED MORE?:
F. THE UNIVERSITY OF NEWCASTLE CORD BLOOD RESEARCH WORK:
G. WHY SHOULD STEM CELL THERAPY BE REGULATED ACROSS EUROPE?:
H. THE POLITICS OF STEM CELLS:
I. NATIONAL AND EUROPEAN – THE ISSUE OF SUBSIDIARITY:
J. THE ISSUE OF CORD BLOOD AND TISSUE BANKING?:
K. THE ISSUE OF FRAMEWORK 7 FUNDING AND ADVANCED CELL
THERAPIES:
L. FOUR KEY QUESTIONS THAT SHOULD BE ASKED OF ANY CELL
THERAPY:

Conclusion.



A. WHY STEM CELLS?:
        Drug therapy can be used to help in some human disease, but not all. Complex
human disease is often the result of a number of different cell systems breaking down. To
treat those diseases it may be necessary to use cell intervention to directly replace or
“retrain” the damaged system. Stem Cells, which may be able to form different systems
and to respond to damaged tissue, may be able to help where drugs cannot.

B. WHAT ARE STEM CELLS?:
       Stem cells have the ability to divide and form different tissues in the human body.
Some stem cells are known to form all the tissues in the human body and some can only
form some, or even just one tissue type. When inside the maternal womb, Embryonic
stem cells can form the whole embryo and developing human. Outside the body some
primary Embryonic stem cell lines have been formed and are being developed for
research. Cord Blood stem cells are taken from the discarded blood in the umbilical cord
and placenta after birth. Bone Marrow stem cells can be taken from any human adult.

C. WHY ARE EMBRYONIC STEM CELLS NOT READY FOR TREATMENT?:
       (i) Right now, not enough Embryonic Stem Cell lines (ES) are available across
the world to cover all tissue types of the human population. Some researchers propose


                                UK Centre for Cord Blood                                  1
                            Researching Adult Stem Cells for Human Therapy
     Cord Blood Stem Cell Therapy Programme – Professor Colin P.McGuckin
                  Professor of Regenerative Medicine, University of Newcastle upon Tyne
                       c.mcguckin@newcastle.ac.uk or c_mcguckin@hotmail.com
                            Mobile +44 07971 266764; Office +44 191 222 7785


that ES lines could be used without proper tissue typing, but this would not be possible as
the human immune system would reject them when they develop into a tissue type. (ii)
When grown for over a certain period of time, ES lines “mutate” or change and could
potentially cause cancer or a harmful effect. (iii) Many ES lines have been made with
animal products and could potentially cause problems. Not enough research has been
realistically carried out to be able to move towards human clinical trials with these cells.

D. WHY ARE CORD BLOOD STEM CELLS GOOD TO USE?:
        (i) Over 100 million children are born each year, which is a huge source of stem
cells and a wide variety of tissue types to cover the population. (ii) Cord Blood is
discarded after birth and is an ethical stem cell source to most of the world’s population.
(iii) Taking Cord Blood is of NO HARM to the mother and child. (iv) Cord Blood has
been SUCCESSFULLY TRANPLANTED for @ 20 years for blood transplantation. (v)
Cord Blood has been used to treat patients with Sickle Cell Disease, Krabbes Disease,
Anoxic Brain injury, Immune Deficiency and others. (vi) Cord Blood stem cells are only
9 months old and can be expanded safely to treat adult patients. (vii) Cord Blood can be
banked. (viii) There is a lower risk of infection and contamination compared to other
stem cell sources.
        However, we must be clear – while cord blood has already been successful for
some treatments, it is still in development for others and we need to continue to develop
and research these diseases to find the best possible treatment.

E. HYPE AND REALITY - WHY ARE STEM CELLS NOT USED MORE?:
        Stem Cell research is very slow and very expensive – a lot more work needs to be
done, to make sure that when we put these cells into people they are in the right form and
will not cause more harm than good. Governments should work closely with Stem Cell
researchers to make sure that proper procedures are in place to protect vulnerable and
sick people. Governments should ensure that from the very start, patients are protected.

F. THE UNIVERSITY OF NEWCASTLE CORD BLOOD RESEARCH WORK:
         Professor McGuckin leads a team which is the first in the world to characterise
Human Embryonic Stem Cells from Umbilical Cord Blood. These cells, which
express human embryonic stem cell markers are the worlds first ethical alternative to the
use of human embryonic sources in research and therapy. Although we do not know yet if
these have all the capacity of human ES cells, we already do know that they CAN form
many different tissues, including (i) Blood, (ii) Endothelium, (iii) various types of neural
cells, (iv) liver, (v) pancreatic tissue – and we are further now investigating cardiac
tissues from these cells.
         In collaboration with the BioE Corporation in the USA we have also worked on a
cell group from Human Cord Blood (called MLPC) which has the capability to form (i)
pancreatic, (ii) liver, (iii) nerve, (iv) skeletal muscle, (v) fat, (vi) bone and (vii) vascular
tissue. This group of cells have also been investigated by the University of Minnesota,
USA, and have been shown to be able to also form lung tissues.



                                UK Centre for Cord Blood                                      2
                            Researching Adult Stem Cells for Human Therapy
     Cord Blood Stem Cell Therapy Programme – Professor Colin P.McGuckin
                  Professor of Regenerative Medicine, University of Newcastle upon Tyne
                       c.mcguckin@newcastle.ac.uk or c_mcguckin@hotmail.com
                            Mobile +44 07971 266764; Office +44 191 222 7785


        So, for the first time reproducible data is now available to show that Human
Umbilical Cord Blood Stem Cells is a serious alternative to embryonic stem cells in the
development of new therapies to treat human disease.
        Our work is, however, held back by a lack of funding. Much of the national
funding for stem cells in the UK is heading towards other types of stem cells including
embryonic stem cells from embryo sources, which is amazing given that embryonic stem
cells have still not been shown to be a widely viable treatment in the way cord blood is.

G. WHY SHOULD STEM CELL THERAPY BE REGULATED ACROSS
EUROPE?:
          Stem cell research is very expensive and getting cells to grow reproducibly and
homogeneously is an extreme problem. Further researchers using embryonic stem cells
from embryo sources have found that they cannot be expanded or grown for more than a
limited period before the cells start to change their genome – ie mutate – and whilst this
is not always necessarily going to lead to a cancer – it might. It is also not easy to expand
stem cells up to enough tissue to be useful to an adult human and this is still a work in
progress across the world. Although some breakthroughs have been reported, they are
still fairly rare. Whilst we expect that stem cell therapies in general have a great potential
to treat human disease and human tissue damage, there is still a great deal of work to be
done. HOWEVER – THIS WORK MUST BE DONE PROPERLY.
          Some companies are already setting up with a stated intention to treat patients
with stem cell therapy. But we must be aware – and be vigilant – as to which companies
are good and which companies are proposing to carry out untested, unregulated and
sometimes unvalidated therapies. Some patients with very serious and life threatening
conditions have travelled to countries where so-called “stem cell therapies” are being
carried out. They have been encouraged to raise large sums of money which they can not
afford and have received treatments which have not helped them and wasted their money.
Worse, these so called “stem cell therapies” can sometimes cause side effects and make
the patient worse in the long term.
          Without legislation right across the European Community, and links to the rest of
the world, cell therapies will continue to be used without proper rules and conditions to
keep our patients safe.
          If there was a Pan-European Advanced Cell Therapy Agency to whom new
protocols could be submitted for approval (in the same way that the USA has a cross
states Food and Drug Administration) this would be helpful.

H. THE POLITICS OF STEM CELLS:
        When people hear about stem cells they often think about embryonic stem cells.
This is natural and obvious, as embryonic stem cells are the very first cells produced at
conception and the cells which can – inside the mother’s womb – make a baby. However,
we cannot allow the ethical and religious debate about whether embryonic stem cells are
to be used or not to be used to stop legislation. It should be based on basic science and
what stem cells can actually do. It is very clearly known what stem cells cannot do at this
stage and we are only finding out the full potential – and sometimes limitations – of these
new cells as “Advanced Cell Therapies”. Scientifically right now, embryonic stem cells

                                UK Centre for Cord Blood                                     3
                            Researching Adult Stem Cells for Human Therapy
     Cord Blood Stem Cell Therapy Programme – Professor Colin P.McGuckin
                  Professor of Regenerative Medicine, University of Newcastle upon Tyne
                       c.mcguckin@newcastle.ac.uk or c_mcguckin@hotmail.com
                            Mobile +44 07971 266764; Office +44 191 222 7785


have scientific problems allowing them to get to clinical trials. Maybe these limitations
will be overcome and maybe they won’t but we cannot wait to find out before we
legislate the use of cellular therapies, because loopholes in the law allow unscrupulous
companies and organisations to offer experimental therapies which could damage
European citizens.
         Further, when adult stem cells like Cord Blood Stem Cells have the potential to be
matched to a particular patient and are increasingly being shown to be useful to patients,
we have to prepare to bring new Cord Blood treatments to patients – this will also require
careful planning and legislation.
         Legislation has to be responsive and not set in stone. Legislations for advanced
cell therapies have to understand that new treatments will appear all the time. So, the
legislation should require any hospital, treatment centre, or, company, to comply with
submitting clinical protocols to clinical peer review committees which can approve on a
treatment by treatment basis what can be done. Certain types of therapies should be
carefully reviewed and if the evidence from preclinical studies – at cGMP grade- and in
animals is not convincingly pointing towards a benefit then a licence cannot be
realistically given.
         Clinical trials in hospitals have to go through internal procedures and generally
through national procedures to gain approval to test any new treatment. However,
companies often are able to avoid this and national and European legislation must come
in line together to ensure that companies developing stem cells are regulated properly.

I. NATIONAL AND EUROPEAN – THE ISSUE OF SUBSIDIARITY:
        Whilst we should carefully regulate what hospitals and companies do, we should
also NOT make it difficult to get new treatments to patients. For example, ONE company
based in ONE European country could potentially be prevented or held up in bringing a
new treatment to patients because they have to go to EACH of the European countries in
turn to get approval for their work.
        Conversely, bad companies could use the loop holes created by either no
legislation, or subsidiarity, to avoid having to apply for a licence for a particular therapy.
This is happening right now across Europe.
        So, on the issue of health European countries have to reach a consensus with
regard to the approval of new treatments and treatment-based products. We do not want
to slow up patients getting new help, but we also do not want bad treatments to reach the
patients. Currently the approval procedures – and sometimes lack of approval
requirements – are different across the European Union, and this is detrimental to both
protecting patients and also to helping patients.

J. THE ISSUE OF CORD BLOOD AND TISSUE BANKING?:
        Critics of stem cell therapy have often said that cord blood and tissue banks are a
waste of time and money. They have also said that companies operating in this area are
playing on the insecurities of parents who fear their children will become sick.
        This argument is no longer valid for cord blood. Cord blood has extensively and
successfully been used to treat patients – via autologous (to the same patient), sibling-
related (to the sister/brother) and also allogeneic (to another unrelated patient). Cord

                                UK Centre for Cord Blood                                      4
                            Researching Adult Stem Cells for Human Therapy
     Cord Blood Stem Cell Therapy Programme – Professor Colin P.McGuckin
                  Professor of Regenerative Medicine, University of Newcastle upon Tyne
                       c.mcguckin@newcastle.ac.uk or c_mcguckin@hotmail.com
                            Mobile +44 07971 266764; Office +44 191 222 7785


blood stem cells have further been used outside of blood therapy and increasingly so.
Trials for the use of cord blood in brain injury in children are underway and cord blood is
being developed for many other therapies including Diabetes and Liver Therapy to name
just two. Embryonic scientists have sometimes spoken out against this type of therapy
saying that embryonic stem cells would be better – but this is not currently the case and
evidence in clinical trials on humans is simply not in evidence to show this.
         The issue of who pays for cord blood and tissue banks is a more relevant debate.
It is not reasonable to think that we can store the blood of every baby born – it would be
too expensive and probably not needed. We recommend that cord blood be stored of
children who have a clear disease history in their family. We also recommend that public
banks be set up in the major metropolitan cities across the world, which are networked;
the public banks would be similar to current blood transfusion banks – for everyone to
use. If a particular patient needed a unit of blood, then a computer system would be able
to find a match in the international banks. It would take some time to create large enough
banks to cover most of the tissue types of even one country, but in the long run it would
be worth it and once set up, would only require “topping up”.
         Until public banks are available public-private partnerships are required. If a
particular family wish to store their own blood, we should not prevent them from doing
so, but the cost should be taken by them, unless they have a clear disease-related family
history. Private banks should be allowed – BUT – should be regulated to ensure that the
blood samples are being stored in the best possible way and that this changes as
necessary when new and better storage procedures become available with new
technologies.


K. THE ISSUE OF FRAMEWORK 7 FUNDING AND ADVANCED CELL
THERAPIES:
         Although the debate about Framework 7 continues, the importance of it to the
development of Advanced Cell Therapies should not be underestimated. Cellular
therapies are complicated and in-depth protocols which require careful and extensive
development. Much of this work would be very difficult on a national level and the
cooperative nature of the Framework programme allows much to be achieved which
cannot be done on a country by country basis.
         The Framework programme also gives us a unique opportunity to bring industrial
companies on board during the development phase of new therapies and, therefore, to
assist them in developing new products for the citizens of Europe.
         Again, the debate on the use of embryos and embryonic stem cells should not be
allowed to hold up all cellular therapy and stem cell related therapy funding schemes in
Framework 7. The use of embryonic stem cells is so far a very small part of what can be
done with other stem cell groups including cord blood, bone marrow and adipose related
tissues. Further, cell therapies could go further than just being stem cell related and there
is potential for as yet unidentified protocols to be developed – but this requires more
research and hence more Framework 7 funding.
         The issue of subsidiarity should be carefully considered here, since it could cause
problems with regard to Framework 7 and the debate, or any subsidiary requirement –

                                UK Centre for Cord Blood                                    5
                            Researching Adult Stem Cells for Human Therapy
     Cord Blood Stem Cell Therapy Programme – Professor Colin P.McGuckin
                  Professor of Regenerative Medicine, University of Newcastle upon Tyne
                       c.mcguckin@newcastle.ac.uk or c_mcguckin@hotmail.com
                            Mobile +44 07971 266764; Office +44 191 222 7785


such as in the UK, to allow embryonic work no matter what the rest of Europe wants -
should be considered separately to what is clearly already agreed across Europe.


L. FOUR KEY QUESTIONS THAT SHOULD BE ASKED OF ANY CELL
THERAPY:

       -can the cells being used be tissue matched to the patient?
              -if yes, then there is a chance the cells will stay in the patient.
              -If no, then there is a potential the immune system of the patient will reject
              them and/or cause problems in that patient.

       -if the cells being used cannot be perfectly tissue matched, is the mismatching
       sufficiently small to be reasonably overcome with immunosuppression and at
       a level which the patient will have reasonable quality of life?

       -is the use of the cell therapy adding benefit that drug or surgical
       intervention cannot provide?
               -no one type of therapy – whether stem cell related or drug related is likely
               to be the whole answer for every human disease. Contrary to the media
               hype, stem cells will not cure everyone.

       -what are the longer term implications of the use of cells rather than
       standard drug or surgical therapy?
              -fixing one problem today that causes another tomorrow needs to be
              considered and the long term benefits need to be assessed, because unlike
              drugs, cells and their progeny can stay around for a long time in the
              human system. Animal experiments may not be able to answer that
              question!
              Long term quality of life has to be the goal of any therapy.


CONCLUSION:

The time to regulate and legislate human advanced cell therapies is now. The rapid
changes in the way patients have been treated in recent years requires us to develop a
responsive legislation that will not only provide a fast route for successful treatments, but
also checkpoints, to stop bad treatments from reaching European Citizens.

Cellular therapies are already being used. New cell therapies are on their way.
Technology is developing rapidly and we have to be ready for the changes that are
coming.
                                                              Professor Colin McGuckin
                                                           c.mcguckin@newcastle.ac.uk


                                UK Centre for Cord Blood                                    6
                            Researching Adult Stem Cells for Human Therapy