Updates on Clinical Trials

There are numerous clinical trials and research projects happening all over the globe for Stargardt's in different scientific and technological fields giving us great hope for the future. Here are the current clinical trials taking place:

Drug Trial of ALK-001
Alkeus Pharamceuticals are conducting a phase 2 trial of an oral drug in the United States. A phase 2 trial looks at the safety of the drug, what side effects it may have, how long it stays in a person's body and the dosing required. This study will also look at whether it affects the progression of Stargardt's. The trial will follow participants over two years assessing these key factors. For more information click here. If you are located in the United States and are interested in finding out more about participating in the trial visit their website here.

Phase 1 Trial using Gene Therapy in the US and France
This is a gene therapy trial currently recruiting in the US and France. It is a phase 1 trial thus the main focus is on looking at the dose of the sub-retinal injection and the effects it may have. If you are interested in participating, the two centres involved are the Casey Eye Institute in the US and the Centre Hospitalier Nationale d'Ophthalmologie des Quinze-Vingts in France. For more information on the specifics of the trial click here.

Stem Cell Trials
The stem cell trial using human derived embryonic stem cells (hESC) I have previously mentioned is still in progress. This trial is run by Ocata over three sites in the US (California, Florida and Pennsylvania). The trial involves an injection of the stem cells into the retina. The researchers will be observing the participants over a 15 year period to monitor the progression of the stem cells, whether they become retinal cells, how this affects vision and if there are any long term complications. More information can be found here and here.

Investigative Research
There are a couple of research projects taking place looking at different aspects of Stargardt's Disease such as its progression. It is important that researchers continue to look at the how, what, where and why of SD to better understand the disease as we don't have a lot of information about it. This information can then be used for targeted treatments and even prevention of disease progression. These projects are always good to participate in because they involve little risk (unlike treatment trials) as they simply gather information, test results and images. The more participants these types of studies get the more information can be gathered and learnt about a condition. 
The National Institute for Health Clinical Centre in Maryland, USA, is currently recruiting for a natural history study of SD. If you are interested in participating check out their website.
The other project is the Inherited Retinal Degenerative Disease Register which is not only for SD but other inherited retinal diseases. This is run by the Foundation Fighting Blindness and more information can be found here.

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

Update on Current Clinical Trials

It is an exciting time in medicine and especially for Stargardt's sufferers and others with inherited retinal diseases. Here is a summary of current trials:

Phase I/IIa study of StarGen (Gene Therapy)

This study is being conducted by Oxford Biomedica at two sites, Oregon Health and Science University (US) and Institut de la Vision (France). Recruitment was suspended earlier this year to investigate impurities in the substance. The study will resume in about three months. 

StarGen - Foundation Fighting Blindness

Sub retinal injection of hESC into RPE (Stem Cells) 

The phase I trial being conducted by Advanced Cell Technology is continuing. The second cohort was recruited in July. There have been no further updates since the last press release. 

Stem Cell Therapy - Advanced Cell Technology

Stem Cell Ophthalmology Treatment Study SCOTS

This stem cell study is using cells derived from autologous (your own) bone marrow which is then injected into the retina. The study is looking at whether there is improvement in visual acuity and visual fields. It is not only looking at Stargardt's patients but also patients with optic nerve diseases and other retinal diseases. The study is being conducted by the Retinal Associates of South Florida (US). This will be an interesting study to monitor.

Bone Marrow Stem Cells - Retinal Associates of South Florida

Safety and Tolerability of MA09-hRPE cells (Stem Cells)

This is another embryonic stem cell trial currently recruiting in Korea, conducted by CHA Bio and Diostech. The trial is similar to the ACT trial.

Stem Cells - CHA Bio and Diostech

Saffron Supplementation and SD

Another study currently recruiting is looking at the effects of saffon supplementation on Stargardt's specifically. The study is being conducted at the Catholic University of the Sacred Heart in Italy. The study is a crossover design meaning that each participant will receive the placebo and treatment for a period of time. I have personally tried saffron and feel as though my sight improved so I am looking forward to the results of this study.

Saffron Supplement Trial

ProgSTAR Natural History Study

This study is funded by the Foundation Fighting Blindness and aims to investigate the disease further and also follow the progression of the disease. This information can then be used in future intervention trials. This is a multi centre trial across the US, UK and Germany. This would be a great study to participate in if you are local to one of the centres. 

ProgSTAR - Foundation Fighting Blindness

Natural History of Eye Diseases Related to ABCA4 Mutations

Here is another study looking at disease progression in Stargardt's . It is being conducted by the National Eye Institute in Maryland (US). To participate you must be at least 12 years old and follow up is over five years. 

National Eye Institute - Natural History Study

Novel Quantification Methods to Detect Progression in SD

This is another trial currently recruiting which is using a specific technique is learning more about the progression of Stargardt's. The trial is being conducted by the Kellogg Eye Centre in Michigan (US). This study is focusing on kids and teens (ages 5-18).

Kellogg Eye Centre Trial

High Resolution Retinal Imaging Study

This study is looking at the use of an adaptive optics scanning laser opthalmoscope AOSLO to take pictures of the retina. The aim is then to analyse these pictures to better understand Stargardt's. This study is also being conducted by the National Eye Institute. 

AOSLO study - National Eye Institute

Looking forward to seeing the results of all the trials.I will keep you all posted. 

More hope!

Another clinical trial is being conducted in Rome, Italy, directly relating to Stargardt's!! This is very exciting news! The study centres around Saffron (a spice) as it is thought to be a "retinal neuro protectant against oxidative damage" (see link below).

The study plans to give saffron supplements to participants to study its effects. This is very exciting news as it seems to be a much simpler way of treating or at least halting the disease. I am even considering going to my local health food shop and getting some supplements or maybe even eating some spicy saffron food!

Fingers crossed this trial is successful!

Check out the link below for more info.

Stargardt's Saffron Trial