Stem cell research under threat: show your support

News just out shows the importance of embryonic stem cells. Another type of stem cell – iPS cells – have had a setback.

http://www.muscular-dystrophy.org/research/news/3824_unexpected_setback_for_stem_cell_therapy

UPDATE: Despite everybody’s efforts signing the petition, unfortunately the European Court went ahead and banned the patents on embryonic stem cells. Its not all bad news though – read about it here:

http://www.muscular-dystrophy.org/research/news/4656_european_court_bans_patents_on_embryonic_stem_cells

Yes all the research we fund is on adult stem cells so luckily that’s all ok. It is nice to have as many irons in the fire as possible though, so hopefully the embryonic research can find away around this ban. Not just for neuromuscular conditions but for lots of other conditions too.

Hi Laura,

Stem cell treatments are not yet available in the UK, or in most Western countries for that matter. This is because they have not been proven to work and their safety is questionable. Although there have been some promising results in animals using stem cells for neuromuscular conditions, there have not yet been any clinical trials in humans. It will probably be a long time yet until the work on stem cells currently ongoing in the lab can be translated into reality in the clinic.

I’m not sure how Stem Genex and Medra are able to perform stem cell treatments in the USA, they are getting through a loop hole in the law somehow. A clinic in Germany was doing a similar thing until recently when they were shut down because a patient died and several others had severe side effects. Brynn (http://stemcellsforbrynn.com/home) also writes in her blog about going to Mexico for treatment?? Is that where the clinic is?

Brynn writes in her blog about the improvements she’s seeing after stem cell treatment. It is convincing when you read it straight from the horses mouth (so to speak) like that. However, it will be interesting to follow her blog over the coming years to see if there is any long term benefit. You have to be wary of a placebo effect that can sneak up on anybody that is really keen to get better, no matter how objective they try to be. That is why we need clinical trials of stem cells, but this won’t happen until the technology looks promising enough and passes certain safety standards.

We always warn people not to take part in unproven therapies in other countries. We fully understand that people with muscular dystrophy are willing to try anything that might offer them a treatment. A lot of people feel that they don’t have anything to lose by trying such options. However, we are deeply concerned about unregulated clinics abroad selling stem cell transplants for large sums of money without any evidence of their effectiveness or safety. As such, they may be at best ineffective, or at worst positively dangerous and potentially fatal. I know of a family who have taken their son to stem cell clinics in India and Germany and paid large sums of money for many treatments and there was absolutely no improvement in his condition.

The difficulty with all types of muscular dystrophy is they are genetic conditions. This means that every cell in the body has the same mistake in the DNA. So if adipose stem cells were taken from your body and injected into your muscles the new muscle cells that might be made would have the same mistake in the DNA and would also waste away. The conditions listed on the stem genex website are not genetic conditions – Alzheimers disease, autoimmune conditions, MS, stroke, diabetes, Parkinson disease. Sometimes people might have a change in their DNA which makes them slightly more susceptible to getting these conditions but there isn’t a direct link as there is with muscular dystrophy. That is, if you have the change in your DNA that causes muscular dystrophy, you will definitely develop muscular dystrophy. So stem cell treatments for muscular dystrophy are going to need to be much more clever – the genetic mistake would need to be corrected in the stem cells before injecting them, and scientists are working on this.

If stem cell treatment had been proven to work, we can assure you that we would make sure that it was available in the UK and paid for by the NHS. Regarding the main heading of this thread “stem cell research under threat”, this is not stopping research in the UK on stem cells. It is just a change in the laws to prohibit patenting of embryonic stem cells (stem cells from embryos). Research into muscle stem cells and adipose stem cells is full steam ahead. Even embyronic stem cell research seems to be unaffected so far, scientists are just saying that if a treatment is developed out of embryonic stem cells they won’t be able to put a patent on it. Maybe they will just come up with other ways to protect their invention?

If you want to continue digging for more information on stem cells for muscular dystrophy there is an excellent website that has a list of questions you could ask Stem Genex and Medra http://www.closerlookatstemcells.org/What_to_Ask.htm
Also this website has some info about stem cell treatment for muscular dystrophy http://www.treat-nmd.eu/resources/ethics/stem- … ersus-hype.

The research for FSHD is moving forward quite quickly at the moment, so you shouldn’t lose hope. You may have heard about a type of gene therapy called exon skipping for Duchenne muscular dystrophy? This is now in phase 3 clinical trial and is looking promising. Similar technology could be applied to FSHD in the future. http://www.muscular-dystrophy.org/research/news/4356_two_research_teams_make_steps_towards_a_treatment_for_fsh

If you have any further questions, or if I’ve missed something don’t hesitate to ask – here or email me k.elvidge@muscular-dystrophy.org.

Kristina.