May 7, 2010 by

Biomedical Ethics in a Brave, New World

Dennis Trammell Pastor of the First Baptist Church at
Possum Kingdom Lake, near Graford was diagnosed with multiple sclerosis in 1999
when his vision decreased in one eye, a symptom of multiple sclerosis called
optic neuritis. Over the years he has been receiving numerous conventional
medications that dealt with the symptoms of multiple sclerosis. However in July
2008, his illness advanced to the secondary progressive phase of the disease.
Having no treatment options available, two months later, he went to the
Cellmedicine clinic to receive adult stem cell therapy.

To date over 200 patients with multiple sclerosis have been
treated with adult stem cells by Cellmedicine. Adult stem cells such as those
derived from the patient’s own fat have the ability to help the nervous system
heal itself from damage, as well as "reprogram" the immune system to stop
attacking the body. This is explained in a scientific publication that
Cellmedicine and collaborators from the University of California San Diego have
written

www.translational-medicine.com/content/pdf/1479-5876-7-29.pdf and is
explained in this video

www.youtube.com/watch?v=wC0VkR3gRoA.

Alluding to the controversy surrounding stem cells, which
was particularly relevant to Pastor Trammell, he stated, "I really questioned
before agreeing to take part in the treatments what type of stem cells were
used". Several stem cell clinics use fetal-derived stem cells. Pastor Trammell
highly objected to this possibility based on moral principal. From a medical
perspective fetal stem cells are dangerous given the possibility of cancer
formation. Accordingly, he was pleased to learn that that Cellmedicine clinic
uses only stem cells from adult sources.

The Pastor reports a significant increase in his energy
level, "I had gotten to the point where a nap was needed on a regular basis. But
since the treatment, a daily nap is no longer needed," he said. Other multiple
sclerosis patients treated at Cellmedicine have reported similar beneficial
effects. Holly Huber from San Diego suffered from loss of balance,
incontinence, fatigue and like Pastor Trammell, optic neuritis. Here is a video
of Holly describing her story
www.youtube.com/watch?v=cqtBfArn1I0.

The issue of using stem cells is considered by many
stakeholders in the religious community. "We’re still dealing with the age-old
question: "Given what can be done, ought we?’ But the list of ‘can-do’ options
in health care get longer each day; hence, also the ‘ought’ questions and the
complexities of knowing right from wrong, good from bad," said Tarris Rosell,
professor at Central Baptist Theological Seminary, and the Rosemary Flanigan
Chair in the Center for Practical Bioethics in Kansas City, Mo.

However, it seems like adult stem cells are not only
acceptable, but in some cases endorsed as an alternative to embryonic stem
cells. This is highlighted by the recent funding of adult stem cell research by
the Vatican

www.cbsnews.com/stories/2010/04/23/world/main6424439.shtml

Filed Under: Adult Stem Cells, News, Stem Cell Research Tagged With: , ,
May 4, 2010 by

Muscular Dystrophy Sufferers Hope New Treatment Can Answer Prayers

Adult stem cell therapy has been used for many diseases
including heart failure, liver failure, stroke, multiple sclerosis, and even
drug resistant tuberculosis. The biological basis for how stem cell therapy
works seems to be two-fold. On the one hand, the stem cells appear to have the
ability to become new tissues, on the other hand, the stem cells produce various
proteins that stimulate the body to heal itself. One condition for which stem
cell therapy may offer great hope is Duchenne Muscular Dystrophy, a disease in
which muscle cells deteriorate due to the presence of a mutated gene (dystrophin)
whose protein produce is involved in muscle contraction.

Researchers from Cellmedicine in collaboration with
Medistem has previously published a case report in the peer-reviewed literature
demonstrating improvement in a Duchenne’s patient treated with mesenchymal stem
cells (Ichim et al. Mesenchymal stem cells as anti-inflammatories:
implications for treatment of Duchenne muscular dystrophy. Cell Immunol.
2010;260(2):75-82). The patient described in the paper, Ryan Benton, was
the subject of a previous news report which is available at

http://www.youtube.com/watch?v=Jyt2LHayjcs.

Today a news report was published describing follow-up on
Ryan Benton as well as another Duchenne’s patient Ian Conner that was treated
with stem cells by Cellmedicine.

Ryan and Ian have known each other all of their lives,
having watched their condition progressively deteriorate. Last year Ian’s
condition substantially worsened.

"At the time, I didn’t think I was going to live much
longer," Conner said. His mother Laurie Conner stated "Last year, I thought it
would be very soon that he would be dying. We needed to get ready, because he
was so sick, in bed a lot and he felt terrible." However there was a glimmer of
hope. Ian’s mother told him about the response Ryan had after receiving stem
cell therapy.

"We got a muscle biopsy back and it has produced dystrophin
and it’s producing normal amounts of dystrophin," Ryan said. He continued "The
main difference I’ve noticed I’ve gained a lot of weight I was down to 77
pounds."

The treatment appears to show greater effects the more
times the stem cells are injected. Both Ian and Ryan are hoping that stem cell
therapy for Duchenne’s will one day be approved in the United States so that
they do not have to travel outside of the country.

Dr.
Riordan has been in discussions with various organization and welcomes any input
on collaborations that can be used to accelerate implementation of this approach
through the Food and Drug Administration.

Filed Under: Muscular Dystrophy, News, Stem Cell Research Tagged With: , , ,
May 3, 2010 by

Stem cells approved to treat ‘orphan’ disease

Stargardt’s Macular Dystrophy is the most common form of
genetic juvenile macular degeneration. Manifestation of the condition begins in
late childhood, leading to legal blindness. It is symptomatically similar to
age-related macular degeneration, and it affects approximately one in 10,000
children.  Olympic and Paralympic skier Brian McKeever is the best-known victim
of the disease, which has no treatment.  Today the company Advanced Cell
Technology obtained "Orphan Drug Status" of use of its embryonic stem cell
derived product MA09-hRPE as a treatment for Stargardt’s disease. 

Orphan Drug Status is a mechanism the government uses to
promote interest of pharmaceutical in disease for which a small market exists. 
Typically to obtain this the target market must be less than 200,000 people in
the United States.  Orphan Drug Status allows a company to retain market
exclusivity for seven years, as well as allows for various clinical trial tax
incentives.

Human embryonic stem cells have been demonstrated to be
capable of giving rise to the cells that make up practically every tissue in the
body.  The ability of these cells to make anything from liver, to lung, to nerve
cells makes them attractive as sources of replacement tissues to biomedical
researchers. Last year the Obama Administration opened federal funding to
more-recent generations of such stem cells, and in January allowed research
funding to cells such as ACT’s, grown from a single cell clipped from an
early-stage embryo.

Designation of Orphan Drug Status is not approval of the
embryonic stem cell based product for sale, but only classifies the cells as a
product in development.  Approval of a drug, whether it is a chemical, a
biologic, or a cell, requires clinical trials in which safety and efficacy is
demonstrated.  The initial hurdle companies must pass is to obtain
Investigational New Drug status.  This was only granted to one embryonic stem
cell company, Geron, for use of their embryonic stem cell derived
oligodendrocytes for treatment of spinal cord injury.  The approval, however,
was rapidly retracted after additional preclinical data demonstrated development
of abnormal growths in treated animals.  Subsequent to IND approval companies
have to demonstrate safety in Phase I studies, efficacy in Phase II studies, and
double blinded efficacy in Phase III studies which usually involve numerous
clinical trial sites. 

The major problem with embryonic stem cell derived products
is the risk of tumor formation.  In general embryonic stem cells are defined by
the ability to form a type of cancer called teratoma.  These tumors are highly
aggressive and comprise numerous cells of the body.  When Advanced Cell
Technologies or Geron are differentiating retinal epithelia cells, or
oligodendrocytes, respective, they must demonstrate to the FDA that no
contaminating stem cells are present in the injection mixture that could
possibly lead to tumor formation.  Another drawback of embryonic stem cell
technology is that it is extremely difficult to selectively add new cells to the
area of injury.  Specifically, the de novo created functional body cells must be
capable of integrating into the existing cells and taking over their function. 
Optimization of these approaches requires understand the molecular cues involved
in natural stem cell differentiation into cells of the body.    Yet another
drawback is that embryonic stem cell lines are not patient-specific.  This
requires the use of immune suppression, which often comes with numerous side
effects. 

Filed Under: Embryonic Stem Cells, News, Stem Cell Research Tagged With: , , , ,
April 30, 2010 by

Stem Cells Don’t have to be Alive to Be Beneficial

The use of stem cells in patients who have poor circulation
is well-known.  In fact, the first use of stem cells for conditions other than
blood disorders was in patients who were undergoing bypass surgery.  Usually
patients undergo bypass because of advanced atherosclerosis that is inhibiting
the flow of blood to the heart muscle.  Despite success of bypass surgery, the
underlying problem of thickened blood vessels remains.  Japanese scientists (Hamano
et al. Local implantation of autologous bone marrow cells for therapeutic
angiogenesis in patients with ischemic heart disease: clinical trial and
preliminary results. Jpn Circ J. 2001 Sep;65(9):845-7) in 1999 treated 5
patients with ischemic heart disease with their own bone marrow cells injected
into the heart muscle during bypass.  Of these 5 patients, 3 demonstrated
increased blood flow at the area where the stem cells were injected.  Subsequent
to this numerous clinical trials have been conducted using bone marrow stem
cells for increasing circulation both to the heart and also to legs that lack
proper blood flow (particularly in patients with critical limb ischemia see
video

http://www.youtube.com/watch?v=dcCwZ4CsiKc ). 

One of the major questions has always been how the injected
stem cells improve circulation.  Originally the idea was that the stem cells
become new blood vessels, and that these new blood vessels take over the
function of the older blood vessels.  However, recent data suggests that the
stem cells injected actually collaborate with the stem cells that are already in
the patient.  For example, it was demonstrated that in patients lacking oxygen
in their legs who receive bone marrow stem cell therapy, the responders actually
have increased levels of their own circulating stem cells.  Here is a video
describing this

http://www.youtube.com/watch?v=OwIOL13vXQ4.

It is believed that bone marrow stem cells, particularly
mesenchymal stem cells, are capable of producing proteins that stimulate the
body’s own stem cells into making new blood vessels.  These proteins include
IGF-1, VEGF, and HGF. 

A recent study from Stanford University (Hoffmann et al.
Angiogenic Effects Despite Limited Cell Survival of Bone Marrow-Derived
Mesenchymal Stem Cells under Ischemia. Thorac Cardiovasc Surg. 2010
Apr;58(3):136-142) should to investigate the cellular and molecular
interactions which are associated with formation of new blood vessels after
administration of bone marrow mesenchymal stem cells.

The investigators first began by assessing production of
the protein VEGF from bone marrow mesenchymal stem cells under conditions of
normal oxygen, and under reduced oxygen conditions.  The idea being that if
mesenchymal stem cells are responsible for producing growth factors, then it
would make sense that production of these factors would increase in response to
needs of the body (eg reduced oxygen).  As a control, fibroblast cells were
assessed side by side with the mesenchymal stem cells.  It was found using in
vitro experiments that mesenchymal stem cells produced much higher levels of
VEGF under hypoxia as compared to fibroblasts, however, mesenchymal stem cells
died faster than fibroblasts in response to hypoxia.

To determine whether mesenchymal stem cells or fibroblasts
cause formation of new blood vessels in animals, a model of critical limb
ischemia was developed in which the artery feeding the leg of mice was ligated. 

One week after induction of ischemia in the leg, 1 million
mesenchymal stem cells, or fibroblasts were injected into the muscles of the
animals.  The cells were labeled genetically so that the injected cells could be
distinguished from the endogenous cells. 

Substantially elevated levels of new blood vessels, and
improved circulation, was observed in the mice that received mesenchymal stem
cells as compared to fibroblasts.  Interestingly, at 3 weeks after
administration, despite improved circulation, the mice receiving mesenchymal
stem cells had much lower numbers of injected cells as compared to mice that
received fibroblasts.

This study suggests that mesenchymal stem cells seem to use
the natural mechanisms of the body in order to generate new blood vessels. 
Something else of interest from this study is that fibroblasts live longer in
hypoxia as compared to mesenchymal stem cells.  Hypothetically it may be
possible to transfect fibroblasts with genes that stimulate production of new
blood vessels.  Unfortunately, the proper combination of growth factors and
concentration are still not known for creation of new blood vessels.

Filed Under: Heart Disease, News, Stem Cell Research Tagged With: , , ,
April 18, 2010 by

Saginaw stem-cell therapy study breaking new ground

Use of stem cells for treatment of degenerative diseases offers great hope. Unfortunately, this hope is tempered by practical
considerations. For example, in patients with heart attacks it is known that readministration of their own stem cells into the infarct related area results in profound improvement
http://www.youtube.com/watch?v=flv0RmzPyLU. The problem with this is that stem cells need to be extracted from the bone marrow of the heart attack patient, which is a difficult and invasive procedure, and additionally the patient’s stem cells need to be processed extensively before they can be re-infused. To compound the problem, the stem cells from patients with heart attacks usually have suboptimal function. Therefore novel ways of performing stem cell therapy are needed that would make this approach practical.

Osiris Therapeutics has developed a clinical product called Prochymal, that consists of expanded bone marrow mesenchymal stem cells. This product has been demonstrated safe by intravenous infusion and perhaps more importantly, does not require matching with the recipient. Currently Osiris has completed Phase III trials and is in discussions with the FDA regarding its commercialization path for this product.

Recently Osiris completed a Phase I trial using Prochymal for treatment of post infarct pathological cardiac remodeling. These results were positive and prompted the company to initiate a Phase II study. Part of this study is being conducted at Michigan Stem & Regenerative Medicine Program of the Michigan CardioVascular Institute and Covenant Health Care under the leadership of Dr. Safwan Kassas, an interventional cardiologist .

"Stem cells represent a promising cardiac research avenue, given their potential to preserve heart function in at-risk patients," Kassas said. "Cardiologists today are unable to reverse cardiac deterioration following a severe heart attack."

To date 2 patients, William C. Smith, 60, of Bay City, in December, and Brenda Sigmund, 62, of Caro, in January where infused with the Prochymal product.

Kathleen Mostek, director of research and regenerative medicine for MCVI, 1015 S. Washington in Saginaw, said the stem cells were taken from five volunteers, grown in a lab and stored in a bank in a freezer. He stated "This protocol is adult stem cells, they are the same for everyone in the world, with no rejection,".

The study is a double-blind, placedo-controlled stem cell investigation being conducted in the U.S. and Canada and will enroll about 220 patients. To be eligible for the trial, patients must have experienced their first heart attack within seven days.

Filed Under: Adult Stem Cells, News, Stem Cell Research Tagged With: , , ,
April 12, 2010 by

Magnetic Attraction of Stem Cells to Injured Heart Creates Potent Treatment

The intracoronary administration of bone marrow stem cells in patients who have suffered a heart attack has been demonstrated to cause beneficial effects in double blind studies, as discussed in this video http://www.youtube.com/watch?v=flv0RmzPyLU. Intracoronary administration has potential side effects since a balloon needs to be expanded in the area where the heart attack occurred, which may cause exacerbation of the existing damage. A more attractive method of stem cell delivery would be via the intravenous route. Unfortunately, intravenous administration has the drawback that some of the cells become lodged in organs such as the lung and liver.
Despite this, intravenous administration has demonstrated positive results, for example in a clinical trial conducted by Osiris (Hare et al. A randomized, double-blind, placebo-controlled, dose-escalation study of intravenous adult human mesenchymal stem cells (prochymal) after acute myocardial infarction. J Am Coll Cardiol. 2009 Dec 8;54(24):2277-86) an improvement in heart pumping
ability was observed.

One way of improving stem cell homing to the area of need is through direct administration of proteins, or genes encoding the proteins, that specifically attract stem cells. This approach has been performed with SDF-1 in animal models, and now the company BioHeart is doing Phase I clinical trials. Other ways include the use of laser therapy to induce expression of stem cell homing molecules as being developed by the San Diego company Entest Biomedical.

Today a new approach was reported in the journal Circulation Research, which is published by the American Heart Association.
Scientists at Cedars-Sinai Heart Institute have loaded stem cells with iron-nanoparticles and administered them intravenously in animals that were induced to undergo a heart attack by ligation of the coronary artery. The scientists found that by
applying magnetic fields to the heart, they could increase the number of injected stem cells that lodged into the heart by 3-times. This was accompanied by functional improvement.

"Stem cell therapies show great promise as a treatment for heart injuries, but 24 hours after infusion, we found that less than 10 percent of the stem cells remain in the injured area. Once injected into a patient’s artery, many stem cells are lost due to the combination of tissue blood flow, which can wash out stem cells, and cardiac contraction, which can squeeze out
stem cells. We needed to find a way to guide more of the cells directly to the area of the heart that we want to heal." Said Eduardo Marban, M.D., director of the Cedars-Sinai Heart Institute.

Commenting on the success of the present study, he stated "This remarkably simple method could easily be coupled with current stem cell treatments to enhance their effectiveness."

Filed Under: Adult Stem Cells, Heart Disease, News, Stem Cell Research Tagged With: , ,
April 12, 2010 by

Using Stem Cells to Study Alcohol Dependence

One of the major advancements in the area of stem cell research has been the establishment of techniques for "retro-differentiating" of old cells into younger cells. Perhaps one of the best examples of this is the discovery by the Japanese group of Yamanaka the skin cells can be coaxed to take the resemblance of embryonic stem cells by transfection with 4 genes. These cells, called inducible pluripotent stem (iPS) cells have numerous applications in many fields.

From a therapeutic perspective, iPS cells allow for the first time the possibility of "reprogramming" adult cells into younger cells, thus opening the door to autologous stem cell therapy for tissue regeneration. In other words, the therapeutic dream of iPS is for one day to be able to take patient skin cells, transform them into stem cells, and then have a large supply of young cells that can be used for repairing any organ of the body.

The other major area in which iPS cells have made a major contribution is in the field of basic research/drug development. Currently when scientists develop new drugs the drugs are tested in human cancer cells that resemble the tissue that the scientist is interested in. So if someone was developing a drug to stimulate pancreatic cells to produce more insulin, the drug initially would be tested on insulinoma cells. If the drug has some positive effects it is then tested in animals, and if successful, in humans.
There are several problems with this model. The first is that many times the cancer cell lines that resemble healthy tissue do not resemble it well. This causes a lot of drugs that appear to work in cells not to work in animals. To some extent this problem is addressed by using cells derived from humans that are not cancerous. The drawback with this is that human cells are expensive and
possess great variability.

Since iPS cells are capable of generating human cells that are "younger", and since they can be created from skin of people with various diseases, the use of iPS cells to generate cells for drug testing has become very popular. For example, if someone wants to study the effects of drugs on ALS, neurons from ALS patients can be easily created from iPS cells in larger quantities than can be extracted from cadaver sources. Dr. Stormy Chamberlain from the UConn Health Center is performing work using iPS cells to develop an in vitro model of alcoholism. Specifically, skin cells will be extracted from alcoholic and non-alcoholic patients. iPS cells will be generated from these skin cells, and then converted into neurons in tissue culture. The neurons will be assessed for abnormalities that are specific to the alcoholic patients.

A collaborator, Dr. Jonathan Covault stated "As proof of principle, we have used skin cells from six subjects to generate pluripotent stem cells, and we have successfully created neural cultures from three of these to develop mature neurons," says Covault. "Going forward, we will compare neurons derived from healthy subjects with those from alcohol-dependent patients. We’ll be evaluating their ability to support electrical signaling and form neuron-to-neuron connections, as well as their pattern of chemical and gene expression responses to single and repeated exposures to alcohol."

Filed Under: News, Stem Cell Research Tagged With: , , , ,
April 12, 2010 by

Can A Nutritional Supplement Grow Stem Cells?

The Center for Improvement of Human Functioning International (CIHFI) was lead author on a peer reviewed study published in the
Journal of Translational Medicine http://www.translational-medicine.com/content/pdf/1479-5876-8-34.pdf describing how Stem-Kine, a commercially available nutritional supplement, significantly elevated levels of two types of stem cells in circulation.

This appears to be the first publication in the peer-reviewed literature describing a non-drug having the ability to increase
levels of the body’s own "repair cells". There is another nutrient that supposedly increases stem cells in blood, however the elevation is only 25% and this occurs only over the period of 1 hour. In contrast, the current study demonstrated increased stem cell activity in the blood over a 2 week period.

"The focus of our Institution for the past 35 years has been assisting the body to heal itself using natural, non-toxic approaches, which have provided benefit for patients with degenerative diseases. Today’s findings support, at a cellular level, how some of our interventions may be functioning," said Dr. Nina Mikirova, lead author of the study.

The study described in the publication examined 18 healthy volunteers who received oral Stem- Kine administration in the morning and at night. Circulating endothelial progenitor cells, which are involved in healing blood vessels, and hematopoietic stem cells, which produce various healing growth factors, were significantly increased as compared to pre-treatment values. Given that both endothelial progenitor cells and hematopoietic stem cells are involved in acceleration of tissue regeneration and healing, it may be
possible that Stem-Kine could improve outcomes in conditions ranging from diabetes to heart failure, to neurodegenerative conditions.

"We congratulate Dr. Mikirova and her team of Industry and University collaborators for applying the objective scientific method to the area of nutritional modulation of health." Said Brian Riordan, CEO of CIHFI. "It is the Center’s vision to integrate patient observations with detailed cellular and molecular biology in order to provide the best care for our patients."

Stem-Kine is produced by Aidan Products and can be purchased from the website www.stem-kine.com.

Filed Under: News, Stem Cell Research Tagged With: ,
April 8, 2010 by

Cord Blood Stem Cell Therapy for Cerebral Palsy in Clinical Trial

Cerebral palsy is characterized by hypoxia/reperfusion
induced damaged to the brain in the perinatal period. It is manifested in four main types: a)
Spastic, which occurs in 70-80% of cases and is associated with damage to the
corticospinal tract or the motor cortex; b) Ataxic, occurs in 10%, is
associated with damage to the cerebrum, and causes deficiencies in walks, hearing
and speech; c) Athetoid/dyskinetic is caused by injury to the to the
extrapyramidal motor system and/or pyramidal tract and to the basal ganglia, it
occurs in approximately 20% of cases. Cerebral
palsy is a non-progressive disorder in which recovery does not occur and
treatments revolve around addressing symptomology. The possibility of stem cell therapy for
cerebral palsy was proposed by Cellmedicine several years ago and is discussed
in this video http://www.youtube.com/watch?v=egRxgUXDN4Y
.

One type of stem cell that has been used for cerebral palsy
comes from the cord blood. Usually cord
blood stem cells are used for treatment of hematological (blood) disorders such
as leukemias or genetic metabolic conditions. Cellmedicine proposed the use of cord blood for conditions such as
cerebral palsy
http://www.translational-medicine.com/content/pdf/1479-5876-5-8.pdf
because of: a) its superior growth factor producing ability to other types of
adult stem cells; b) the possibility of using cord blood with minimal matching;
and c) the ability of cord blood stem cells to directly differentiate into
other types of cells relevant to cerebral palsy such as neurons and glial
cells.

In order to test validity of the possibility that cord blood
may be useful for such a condition, the developmental cycle that occurs with
drugs has to be applied. That is,
firstly animal data needs to support the possibility of efficacy, as well as
the safety of the intervention. Secondly, pilot human studies are needed to determine if it is feasible
to administer the cells in patients with the particular disease without
possibility of adverse effects. Thirdly,
formal clinical trials need to be initiated. These usually begin with Phase I trials that assess safety and maximally
tolerated dose, Phase II trials that assess efficacy in a non-blinded manner,
and Phase III trials that seek efficacy in a
double-blind placebo-controlled manner.

Groups like Cellmedicine have been involved in treatment of
patients with cord blood. Additionally,
Dr. Joanne Kurtzburg from Duke has been using the patient’s own cord blood in
treatment of patients with cerebral palsy http://www.youtube.com/watch?v=xLmY7Ps65wQ. Both
of these treatments were considered part of the "practice of medicine" and may
be comparable to "pilot investigations" in that safety data was generated and
the medical procedure for physically administering the cells was
developed.

Today a group at the Medical College of Georgia announced
initiation of Phase I/II Placebo-Controlled, Observer-Blinded, Crossover Study
to Evaluate the Safety and Effectiveness of a Single, Autologous, Cord Blood
Stem Cell Infusion for the Treatment of Cerebral Palsy in Children.

The trial involves 40 patients between ages 2-12 who are
seizure-free and have clinical evidence
of a non-progressive motor disability due to brain dysfunction. The subjects recruited
will not have the ability to sit independently by one year of age or the
ability to walk by 18 months of age.

Patients will be
divided into 2 groups, with the first group receiving red-cell depleted, mononuclear
cell enriched cord blood unit prepared for infusion (treatment) and the second
being administered saline combined with the inert stem cell administration
solution lacking stem cells. The
observer and patient will not know who is receiving cells from which
group.

The main observation endpoints of the trial will be safety of autologous (patient’s own)
cord blood infusion in children with cerebral palsy by repeated follow-up over
one year with clinical and laboratory evaluations. The secondary endpoint will
be determination of whether a beneficial effect has occurred in the
recipients. This will be measured using
a patient questionnaire and standardized Gross Motor Function Measure
evaluation with effects anticipated to be seen within 3-4 months.

Conceptually this study is a very safe one because it is the
patient’s own cord blood stem cells that are being used. This however could also be a negative
issue. There is some evidence that when
stem cells from another individual (allogeneic) are used, it is the reaction
between the recipient and donor that gives rise to production of numerous
growth factors. Since this current
treatment is only using the patient’s own cells, it may be similar to simply
adding your own blood back into you. The
animal studies previously performed involved using human cord blood cells in
mice lacking part of the immune system. Additionally they used much higher concentration of cord blood cells per
kilogram of body weight. Regardless, it
is very important to state that this study lays the groundwork for translation
of numerous stem cell approaches that have previously been used for patient
treatment outside of the US, for US approval.

Parents of patients interested in trial participation should
contact James E Carroll, M.D. the Principle Investigator of the study at 706-721-3371 jcarroll@mcg.edu

Filed Under: Adult Stem Cells, Cerebral Palsy, News, Stem Cell Research Tagged With: , , , ,
March 8, 2010 by

Stem cell therapy shows early promise: Celgene

Crohn’s disease is a favorite amongst mesenchymal stem cell
development companies. This may be because on the one hand, this type of stem
cell possesses anti-inflammatory properties, and on the other hand it has the
potential to regenerate injured tissue. Additionally since the quality of life
of patients with advanced Crohn’s Disease is so poor, and current treatments are
generally ineffective at addressing the root cause, that new treatments usually
receive much support from regulatory agencies. Crohn’s disease is characterized
as a chronic inflammatory condition of the gastrointestinal tract. It is
believed to affects almost one million people in the United States.

Today Celgene announced Phase I safety data on its
placental mesenchymal stem cell product PDA-001 in a trial of 12 patients. The
patients suffered from active moderate-to-severe Crohn’s and were unresponsive
to at least one prior conventional therapy. The treatment with stem cells
comprised two infusions of PDA-001 one week apart. The patients were divided
into 2 groups with 6 patients being administered a lower number of cells and six
a higher number.

According to Celgene, "The study met its primary safety
goal and demonstrated encouraging signs of clinical benefit, including clinical
remission among four patients in the low dose group". Interestingly the company
declined to speculate on why the lower number of cells elicited superior
benefit. As an interesting aside, the company Osiris Therapeutic conducted a
similar clinical trial in Crohn’s Disease using stem cells derived not from
placenta but from bone marrow sources.

The CEO of Celgene’s Cellular Therapeutics unit, Dr. Robert
Hariri stated "We are encouraged that in these patients with Crohn’s disease our
unique, placenta-derived therapies show signs of clinical benefit," he continued
"We will continue to aggressively pursue the clinical development of this and
other cellular therapies derived from what we see as one of the richest sources
of uniquely functional and versatile cells."

The company anticipates moving into Phase II clinical
trials not only in the area of Crohn’s but also in other degenerative
indications.

It is an interesting point that the cells were administered
intravenously. There are some groups that believe stem cells only work if
administered locally. This study suggests that the need for local injection may
not be as important as some others believe. Additionally, since companies like
Cellmedicine use various mesenchymal stem cell sources, the current results
provide US-based scientific evidence supporting at least the rationale for this
approach.

Filed Under: Adult Stem Cells, Clinical Trials, Crohn's Disease, News, Stem Cell Research Tagged With: , ,
« Previous Page
Next Page »