The following is a summary of the May 21st 2013 Webcast “Stem Cells & Cerebral Palsy: The Promise and the Progress”, presented by Cerebral Palsy Alliance in Australia:
Main topics to be covered:
What does the future hold to prevent, treat, or even cure CP?
What is fact, fiction, or somewhere in between?
What has been achieved and what may be achieved soon?
Summary of Dr. Iona Novak’s presentation-Head of Research at CP Alliance in Australia
Why are researchers interested in exploring stem cells for CP?
We need better/more effective treatment for CP and it’s related conditions. Current interventions and treatments offer typically only 4-10% gain or improvement. According to world wide CP registers we have the following statistics related to CP and some of its secondary associated conditions:
1 in 3 cannot walk, 1 in 4 cannot talk, 1 in 4 have epilepsy, 3 in 4 are in pain and some cannot communicate it, 1 in 3 have hip displacement, 1 in 10 are blind, 1 in 5 have a sleep disorder.
One of the most under-recognized problems with CP is pain and this is an aspect of CP that they would like to do something about. Some people with CP can’t find relief and/or talk about the pain they feel.
Summary of Dr. Megan Munsie’s presentation-Head of Education, Ethics, Law & Community Awareness Unit at the ARC funded Stem Cells Australia Initiative
Our body is made up of over 200 types of cells. The nucleus of the cell tells it what to do and codes for genes. Different cells of the body look and behave differently. Different cells express different genes.
What is a stem cell?
A primitive cell that has the capacity to grow into one or more specialized cells. It has the capacity to grow into a copy of itself. It can repopulate tissue. Stem cells are being used to understand more about disease. Researchers are using them in the lab and with different drugs to see where they may be able to make a difference.
What is a blood stem cell?
Blood stem cells are the most well known and come from the bone marrow in the long bones. Many people have heard of bone marrow transplants where cells that are not functioning correctly are replaced with new ones. Can we use blood stem cells and their applications as an example from which to apply similar principals to other types of stem cells? It may be a bit more complicated than what we can currently do with bone marrow, but it’s a concept researchers can potentially begin to explore.
What are MSC’s?
Also found in the bone marrow they are a companion cell to blood stem cells and they can dampen potentially harmful inflammatory immune responses.
What is cord blood?
Used since the 1980s, it’s isolated from the placenta and can be processed and stored for later use. Many different types of cells can come from cord blood. At the moment the only proven stem cell treatments are for diseases of the blood and immune system, some skin graft treatments and some causes of blindness.
All of the organs of the body have stem cells even if the organs are not inherently regenerative. The is a new concept. How do we recruit cells from different organs and get them into activity? This is a big area of research!
What are embryonic stem cells?
Scientists can grow these since discovery of them in the late 90s. They can grow them into lots of different types of stem cells. Embryonic cells have a large capacity for change which makes them unique. Researchers are just starting to harness their potential.
What are IPS cells?
Discovered in 2007 by a Japanese researcher who received the Nobel Prize last year for his discovery. He was able to change four genes in a skin cell and turn it into a stem cell with pluripotent (can differentiate into many different types of cells) characteristics. The Japanese government is investing lots of money in stem cell research now.
There are different types of stem cells that are beginning to be tested in clinical trials throughout the world.
Dr. Munsie cautions people about using Google when looking for information about stem cell treatments. She encourages people to discuss what they find with their medical professionals:
It’s tough to work out which sites are discussing clinical trials and which are treating people. Stem cells have enormous potential but testing for safety and efficacy needs to be properly established first. Dr. Munsie is very wary of clinics offering to treat people with a wide range of problems and complexity using one approach. She cautions consumers to think twice, and go to your doctors who don’t have a vested interest in selling an expensive product.
Dr. Euan M. Wallace-Director of the Ritchie Centre and the Carl Wood Chair of Obstetrics and Gynecology at Monash University.
Dr. Wallace limited his discussion to cord blood cells and CP:
1. According to Dr. Wallace, experimental data suggests strongly that giving cord blood cells to children at very high risk for developing CP may prevent them from developing it. Clinical trials are underway around the world that are looking at this possibility now.
2. What happen when you give cord blood to children who already have CP (in order to treat them)? No animal studies have investigated the use of cord blood where there is an established early brain injury (and the animal studies typically precede the human studies).
Historical use of cord blood:
Twenty-five years ago Matt Farrow was a patient of Dr. Joanne Kurtzberg, current Chief Scientific Officer and Medical Director at Robertson Cell and Translational Therapy Program at Duke University. Matt was five and had Fanconi’s Anemia which at the time people typically died from before the age of 10. Dr. Kurtzberg collaborated with a researcher and physician in Paris so that Matt could receive the first cord blood transplant. It cured him and saved his life. He is now 30 years old and married with his own children.
At the time Dr. Kurtzberg and the doctors she collaborated with thought that Matt Farrow had a 10% chance of being cured of that disease and they still did it. Today over 30,000 people have received cord blood transplants for blood disorders and blood cancer. Dr. Wallace would like to see researchers put effort and resources into exploring stem cells for CP. He wants to see what the results would be. **More info from CP Daily: Dr. Kurtzberg is currently conducting a clinical trial in the US on the use of cord blood in CP. The trial is expected to be concluded in 2014. Dr. Wallace definitely is pushing for Australia to start their own clinical trials as well.
Return to summary of Dr. Wallace’s presentation:
There was a recent South Korean trial using donated cord blood that was published in a journal called Stem Cells. There were 90 children who participated. There were three groups (included in this trial were also the use of EPO and cyclosporine) within the trial and all groups improved . There were some questions as to whether all three groups progressed because of the intensive rehab and/or because the children were getting older as the trial progressed. The biggest improvement was in the 31 children who received cord blood. Children less than 3 had the best reported improvement. The closer the blood match of the participant and donor, the better the outcomes.
There were no dramatic changes in the Korean clinical trial but Dr. Wallace believes it showed sufficient improvement for launching a national trial in Australia.
Summary of Stephen Archer’s presentation-Parent contributor who pursued stem cell treatment at Duke University
Mr. Archer has a son Zach and at the time they pursued getting him a cord blood transplant he was 5.
Zach had a stroke (of unknown cause) which caused his CP. He has right-sided hemiplegia. He has muscle spasticity in his lower leg and upper right arm. They began using serial casting and Botox at an early age. Zach started having seizures at 2 years old. In five years anti-convulsants had not had much effect and it was not uncommon for him to have up to 100 seizures per day. Epilepsy was thought to be unrelated to his stroke when doctors evaluated him for surgery (to try and address his seizures) so the surgeons would not proceed. The family kept looking for other possibilities/treatments.
They investigated the use of cord blood but couldn’t get Zach infused with his own in Australia. They were able to arrange to take his cord blood to Duke University where they asked Dr. Kurtzberg to provide one treatment not associated with a clinical trial (Mr. Archer mentions the challenge of having to take multiple trips overseas if they did participate in the US clinical trial). *Note from CP Daily Living: The clinical trial taking place at Duke includes three visits over a three year period but only one of those visits includes the cord blood transfusion. The family isn’t told which visit includes the administration of the cord blood. If the Archers participated in the clinical trial it would have been free (except for travel expenses) versus the $40,000 (which included travel expenditures) they spent to have the transfusion done outside of the trial.
Outcome: Treatment was November 2010 and they have not seen significant progress or changes. Mr. Archer doesn’t think the treatment was a “failure”. He felt they had to give this a try especially since it was considered by their consulting doctors to be a “no harm” scenario for their son. He said he would do it all over again despite the expense and outcome.
*Despite Mr. Archer saying that their consulting doctors felt there was “no harm” in their son receiving a transfusion of his own cord blood, I personally would like more information about what has or has not been studied or recorded in the literature with respect to autologous cord blood transfusion and potential risks.
If you would like to watch a recording of the live Webcast you may view it by clicking here.