Update on Stem Cell Treatment

If you haven't already heard, results from the phase I trial using human embryonic stem cells for patients with Stargardt's Disease and Macular Degeneration have been published in the Lancet, and the results are very promising!

Nine patients were treated from each disease which involved surgical implantation of the stem cells into the retina of one affected eye. The main aim of phase I trials is to test the safety of the intervention, so whether there were an adverse events or complications from having the stem cells implanted, and in this case whether the stem cells 'stuck' to the eye. The investigators also looked at whether there was any improvement in vision.

Excitingly, the results were very promising - there was no major adverse events (although some patients with Stargardt's developed cataracts but this was treated surgically and didn't affect vision after treatment), no rejection of the implanted cells, no abnormal tumour growth and it looked as though the cells remained functioning at follow-up (which was up to two years after initial implantation). To top it off, there was also objectively reported improvement in vision in the treated eye!

To put it in perspective, this is still early days and we won't be running out to get stem cells injected tomorrow. Although it is definitely looking like a very possible treatment in the future and in our lifetime! The next step is to proceed to a phase II trial which will include a larger number of patients and find the best dose (number of cells to be injected) to gain the best results. Further evidence of the effectiveness will be developed to show how much vision can be restored, if it is permanent, whether the implantation needs to be done in the early stages of the disease and many other questions.

This is extremely exciting and shows how amazing modern medical science can be. It can give us all a little more hope that potentially in the future we may be able to see better, and if not us, the generations to come who will unluckily have to experience the hurdles we have faced. 

For more information here are the links to the results and news reports:

Wall Street Journal

The Lancet

Media giving vulnerable hope

The latest headline reads:

"Once they were blind, now they see. Patient's cured by stem cell 'miracle'"

This article was dated 24th January 2012 and posted on The Independent website (link below).

Are we not getting a little ahead of ourselves?

The article refers to the Advanced Cell Technology stem cell trials and their latest findings. As previously mentioned, they have treated two patients, one with Stargardt's, with RPE embryonic stem cells. This trial is purely for safety, and is not measuring the effectiveness of embryonic stem cells in the treatment of the diseases.

It is very exciting that of the two patients, so far they have not experienced any adverse effects - tumours or reactions. From what I have read it has been 4 months since the patients received the cells and they are doing well, that is to say are not doing badly.

Because the ACT trial is proving to be safe, another 2 Stargardt's patients will receive the stem cells, one in the US and one in the UK. This is great news for stem cell research.

The patients that have been treated have also stated improvement in their vision. ACT are not focusing on the visual improvement in this stage of trials and there is a link below to the published report.


So I am putting my skepticism hat on again and I am angry that the media can claim that this is a 'miracle' and 'the blind can see again.' These trials are for SAFETY and are not measuring whether stem cell treatment actually works! To say that the blind are cured is getting just a little too ahead of what is actually going on. ACT have stressed that these trials are safety trials and the media should pass the information to the public, instead of giving false hope to people like me with Stargardt's.

There is still a long way to go with stem cells. I will continue to wait in anticipation.

Below are links to recent articles about the ACT trial.

The Independent

Advanced Cell Technology

Australian Ageing Agenda

The Lancet

Aussie Stem Cells

There is a new Aussie initiative officially launched on the 10th November 2011 called "Stem Cells Australia". It is led by Professor Martin Pera in collaboration with various Australian Universities and Research Institutes and supported by the Australian Government. One aspect of their research is in human embryonic stem cells and their ability to become any bodily cells, which as we know is being trialled as a potential treatment for Stargardt's. They also aim to tackle ethical issues surrounding stem cells, which is sure to be an interesting debate!

As yet, there is no specific mention to Stargardt's or any ocular conditions, but it is a step in the right direction for Australia and for the possibility of treatments being made available here.

The website has a whole heap of information so check it out.

I am particularly looking forward to the ethical debates, since it is a potential treatment for Stargardt's and I know there are many groups opposing the use of embryonic stem cells.

Will keep all updated!

Stem Cells Australia

Updates on Advanced Cell Trials!

As I have mentioned previously, Advanced Cell Technology are undertaking in stem cell research to treat Stargardt's disease. They have now successfully treated the first Stargardt's patient with embryonic stem cells, and have recently been approved by the Data and Safety Monitoring Board to proceed to treat a further two patients with Stargardt's.

This is fantastic news and we are one step closer to hopefully having a treatment. It is also a positive sign that the trial has been cleared to continue as the patient must have responded well to the first treatment, or at least it was not harmful! I am looking forward to more information on the trial as I'm sure all of you are. Check out Advanced Cell Technology's press release below.

ACT Press Release

The London Project

I have been told by several people to check out 'The London Project' as they are making strong progress in finding treatment for blindness. The organisation aims to cure blindness through stem cells, and with what I have learnt to date about Stargardt's, this seems to be the future in our treatment. Their main focus is on macular degeneration, but this could definitely lead to treatment for Stargardt's, as they are focusing on finding a way to replace the damaged RPE cells of the eye. 


The London Project has finished the safety phases of its trials and will proceed to human clinical trials in 2012, which is very exciting news. Like Advanced Cell in the US, The London Project uses embryonic stem cells and hopefully success is in the near future.


The organization is currently asking for donations to make the next phase of trials happen. Anybody wishing to donate can do so on the link below.


The London Project

Stuff I've learnt from Twitter!

I've been following the Foundation Fighting Blindness (@gihtblindness) on Twitter over the past few weeks. This weekend they have held Vision 2011 conference in the US. They have been updating their posts regularly and I have found out some interesting facts. The following are the tweets that I found really interesting:


1. "Q: animal trials - any reversal of vision loss 4 Stargardt? A: going that direction, but nothing yet showing definite restoration."
2. "Dr. Jacque Duncan (involved in retinal disease clinical trial) said that retinal degeneration is usually slow so it's hard to detect changes in 2-3 year studies."

3. "From Stem Cell session panel - Must easier to save retinal cells than replace them."

4. "Gen etc testing and thorough exams important because many diseases look alike.. Cone rod, Stargardt, Best Disease can look similar."

5. "About 50% of cone rod dystrophy caused by ABCA4 - same gene that causes majority of Stargardt disease."

4. A quote from John Wellner - "It's a great time in history to be blind; way better than the 1400's". You'd hope so!!
The link to the Foundation Fighting Blindness is below.


Foundation Fighting Blindness

They have a great wealth of information and are a great foundation so check it out.

Update on Advance Cell Technology Progress

ACT, who were granted permission last year to conduct a human clinical trial treating Stargardt's patients with stem cells, has announced that they can procure embryonic stem cells without embryonic destruction - a positive ethical step. They state that they are also hoping to progress to the second phase of the Stargardt's specific trial in the first half of this year.

Check out the link below:

Advance Cell Technology Update

FYI

Advanced Cell Technology (ACT), who late last year were granted FDA approval to conduct clinical trials for embryonic stem cell treatments for Stargardt's patients, has now also been given approval to use embryonic stem cells to treat patients with Dry Age Related Macular Degeneration (AMD).. It seems to be a very similar trial, using the same cells to produce new RPE cells for the macular, and it is also in the first phase of safety trials. This trial could also be important for us Stargardt's patients as the conditions are similar. Check out the ACT website:

ACT - Approval for Trials of ESC's for AMD

Ethics and Embryonic Stem Cells - Opinions??

I've been doing a bit of research on the treatment that Advanced Cell Technology is beginning to trial to treat Stargardt's and came across an anti-cloning and embryonic stem cell research blog. This is put together by Australian doctors and scientists. There is a specific post relating to the ACT trial. The post states that ACT are really in it for the fame of discovering something "new" and that it will likely cause immuno-suppression to the treated patients. It also states that such embryonic stem cells can't ever be injected into a patent's eye, and the trial being conducted by ACT is actually to see what harm using such cells may have on people.

Naturally I was angry to read this as when I discovered that ACT was conducting an actual human trial I was ecstatic and was given hope that I would definitely one day regain some of my vision. So this lead me to research what this post has said and find out more about the ethics surrounding embryonic stem cells, as I am all for ethical research and treatment and reading these statements did make me feel somewhat uncomfortable with the whole idea of have such cells put into my eyes. This is what info I found:

1. Embryonic stem cells come from 5-6 day old human embryos. Embryos in humans are a fertilized egg of up to 8 weeks. To me, the though of using these cells made me uncomfortable, although when I researched further and found out that they were only 5-6 days old, merely a bundle of cells, my feelings somewhat eased. I'm not entirely sure where the embryos for the ACT trial are from, but I will try to find out. The following website is from the Australian Government about Biotechnology and has some useful information about stem cells:

Biotechnology Online - Stem Cells - Australian Government


2.Induced pluripotent stem cells (IPS) are another type of stem cells the post states that could be used instead of embryonic stem cells, as they do not have the ethical issues associated with embryonic stem cells as IPS cells are derived from the person's own body, reducing the risk of rejection of foreign cells and also immunosuppression to the person receiving the cells. This type of stem cell was first produced in 2006, making it quite new so obviously there still needs to be a lot of research on the impact of such cells. There is the thought that the use of such cells may impact caner causing genes, making them "turn on" and cause problems, although a study found a way to stop this from happening. I'd like to find out more on these IPS cells and see why such cells can't be used in place of embryonic stem cells.

I hope this information has been useful! I am a strong believer in ethics and I like to know everything about everything, which is why I felt the impulse to find out more about stem cells and the different types that could potentially help us  with Stargardt's. What are your thoughts?