Another potential cure by Alkeus!

I have come across another company looking into a treatment for Stargardt's Disease. The company is Alkeus Pharmaceuticals in the US. They have created new compounds to treat Stargardt's as well as Age Related Macular Degeneration. So far they have tested these compounds on mice with success.

They have a page where you can register to be apart of future trials and also a Stargardt's registry. The trials will take place in New York so it is a good excuse for a holiday! I've posted the link below so check it out and register!

Alkeus Stargardt's Registry

Update on the ACT embryonic stem cell trials

So we are all aware of the upcoming trials by Advanced Cell Technology to see whether Stargardt's and also Dry Age Related Macular Degeneration can be treated with embryonic stem cells. ACT released more information a few days ago about the upcoming trials involving 12 patients for each condition.

The phase of the study isn't actually testing whether using the stem cells will have any benefit, rather on the safety of using such technology.

The am of the treatment is to replace the malfunctioning RPE cells in the macular with functioning cells to halt the damage being done.

Hopefully the first phrase of the trials are successful and safe, with minimal harm being placed on the brave Stargardt's patients being involved.

Check out more information on the links below.

Red Orbit

UK Mail online

Google News

A Cure in Accutane?

One ray of hope that I was given from my opthamologist was the possibility that the drug Accutane could help treat Stargardt's. I was told that if a doctor could have a good enough reason to prescribe the drug to me, my opthamologist would study its effects on my eyes.

Accutane is a drug used to treat severe skin treatments and also cancers. It is known to have severe side effects such as birth defects, kidney problems and depression.

Overall, this drug does not sound like something that I would be willing to take, which I decided after I researched into the side effects, and would probably not be a treatment I would consider for the same reasons even if it would halt the progression of Stargardt's. Although it must be remembered that the progression of Stargardt's can not be predicted so would I risk taking the drug for a condition that may not even get seriously worse? Any thoughts?

Check out the following links for more info on Accutane:

MD Support - Accutane and Stargardt's

Accutane

Wikipedia - Accutane

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?

Treatment on the horizon!!

In November 2010, Advanced Stem Cell Technology in Massachusetts was approved to conduct a human study in patent's with Stargardt's!! What fabulous news!!

From my research, they will be using embryonic stem cells, which are stem cells from embryos from a fertilized egg in vitro - so these eggs are fertilized in a lab and not in a woman's body.

The following website is an article outlining the study:

US starts human blindness trial using stem cells

Let's keep our fingers crossed and hope all goes well in this trial. Hopefully in the near future we will all be able to be treated!

For all the Aussies - Inherited Retinal Diseases Study!

I've recently become involved with a department as part of Sir Charles Gairdner Hospital in Western Australia and their Inherited Retinal Diseases Register and DNA bank. The aim of this organisation is to be able to study the mutations to the gene that is causing Stargardt's. So I decided I would consider donating my DNA so hopefully somebody can find a treatment!

I'm still yet to consider the ethics and legality of this, and my main concern was who would have access to my DNA - I definately don't want insurance companies or the police accessing my very, VERY personal information I don't even know about myself! Once I recieve the kit and read through the organization's information I can make my judgement but it is definately looking promising!

If anybody else want sto participate here are the contact details for the department responsible:

Department of Medical Technology & Physics
Inherited Retinal Disease Register & DNA Bank
Sir Charles Gairdner Hospital
Nedlands, WA 6009
Ph: (08) 93462408

I have been informaed that so far there are only 115 people in Australia registered on the database who have Stargardt's! This makes me feel kind of special and unique!