The results are in!

Although we have been quiet on this website, we have been hard at work during the last three years! Our gene therapy trial wrapped up in September of last year and our results were just published online in the past month. As you may remember, this study investigated the safety and utility of gene therapy as a potential therapy for choroideremia. The goal of gene therapy is to reintroduce a functional copy of the CHM gene into the eye using a vector (or customized virus).

Our study included 6 participants aged 30-42 years who had a confirmed diagnosis of choroideremia. The viral vector which included the CHM gene was injected into the eye with worse vision of each participant. They were then followed over a 2-year period (Summer 2015-2017) with visual function testing every 6 months. The primary goal of the study was to determine whether the gene therapy was safe. The second goal was to measure any differences between the treated and untreated eye in each participant over time. We achieved both of these goals by measuring eye function and structure using different specialized testing equipment.

Five out of six participants had no serious adverse effects to the procedure but one participant did experience a reaction in his treated eye resulting in a notable decline in visual function.

In addition, the retinal tissue in all participants appeared to deteriorate in the treated eye at the same rate as the untreated eye during the course of the study.

Therefore, we concluded that the experiment itself did not improve visual function or halt the degradation of retinal tissue caused by choroideremia. We learned valuable information about safety and how to measure vision for future gene therapy trials.

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Summer 2015

There has been a lot of activity in our research lab these past few months with the start of our clinical trial and with our summer students being around. We are excited to share some of the work with you!

Here is the team on the first day of the gene therapy surgical procedure:

L-R: Dr. Riz Somani, Dr. Matt Tennant, Dr. Ioannis (John) Dimopoulos, Prof. Robert MacLaren, Dr. Ian MacDonald, Janice Hengsbach RN, Dr. Markus Groppe and Dr. Paul Freund.

  • Here is a list of recent articles, a radio interview and even a YouTube video about our ongoing study.
  • Calvin Tseng, one of our students, will be starting his second year of medical school this month. He had the opportunity to write about his experience working with our choroideremia clinical trial participants in an essay contest submission (not to worry – the individuals he interviewed were well aware of the contest!). Calvin has graciously allowed us to share his essay with you, our readers!
  • Our Grade 11 HYRS summer student worked on a 6-week project about a different genetic eye condition called X-linked retinoschisis and updated our lab member profiles in her spare time.
  • We also had the opportunity to attend and present at the 2015 International Society of Genetic Eye Diseases and Retinoblastoma Conference in Halifax, Nova Scotia earlier this month.

Overall, it has been a great, productive summer for all of us!

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First Canadian ocular gene therapy trial begins!

We are excited to announce that our experimental gene therapy trial to treat choroideremia has began. Here is the official news release. This could not have been possible for our team without the involvement of our funding  partners and our patients, past and present, far and wide. Thank you.

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2015 Update #1 (edited)

Hello everyone! Here is a long overdue update!

After plenty of hard work from both parties, the University of Alberta and NightstaRx have been able to finalize the research agreement necessary for this clinical trial to take place.

The brand new storage facility located on the University of Alberta campus and where the viral vector will be stored, has been validated and will be ready for the start of the trial.

Final pre-trial training requirements for the research team members have been scheduled.

Though delayed by a few months, patient selection is now well underway. We will be recruiting six patients in 2015. Patients who have already been in contact with us will be informed whether or not they are selected for this trial once this process is complete.

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A few steps closer…

We have now received our final No Objection Letter from Health Canada!

Our group has been actively working with NightStaRx Ltd. on finalizing the research agreement between this new biopharmaceutical company and the University of Alberta. This legal document will enable NightStaRx to supply the vector to us for the trial.

Two weeks ago, two members of our team along with a quality expert from TEC Edmonton travelled to perform an audit of the facility at Nationwide Children’s Hospital in Columbus, Ohio where the viral vector that will be used in our clinical trial was produced. The audit team provided a favorable review which is great news! This information will be necessary to import the vector to Canada.

We expect to select the patients for our trial in the next two months.

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Trial recruitment expected to begin Fall 2014

As a recap of the year so far, our team at the University of Alberta has been hard at work preparing for our upcoming choroideremia gene therapy clinical trial.  The study details, including the criteria for participation can be found here. We will begin recruiting participants in Fall 2014 when we receive final approval from Health Canada. Until we have that final approval we cannot recruit participants. At present, we have preliminary approval from Health Canada but need to provide some more information on the vector that we will be using in our clinical trial. We expect the information from the company providing the vector shortly.

Since our clinical trial will be a small Phase 1 study, we will only be enrolling 12 participants over the course of a year. We have put in place a formal process for selecting participants. Selection will be made by an external board of Canadian ophthalmologists. We need participants whose clinical symptoms are at the specific stage to give us the best possible results in the clinical trial. We also need to select participants who will stick to the follow-up schedule. The most important parts of the trial are the follow-up visits where we collect the results. Approval for any further, larger clinical trials will be based on the results of this trial.

We need to be prepared so that we can recruit as soon as we receive final Health Canada approval.  Therefore, we are looking at anonymous records in the Foundation Fighting Blindness Canada Patient Registry to later identify individuals who might meet our selection criteria and also going over the clinical records of Dr. MacDonald’s patients. As mentioned in the previous blog post, we have also been fine-tuning the methods and equipment we will eventually be using to assess vision in the clinical trial. Vision is the main outcome measure to assess the results of the clinical trial.

We want to make clear that the clinical data we will be using to assess eligibility does not need to come from our centre in Edmonton. We want to encourage interested people with choroideremia to register with the FFB Canada Patient Registry as we have full access to this data. This is also beneficial because other gene therapy research teams may also apply for access to this registry data.

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In Denver and in Edmonton.

Many members of our research team had the opportunity to attend the 2nd International Scientific Symposium for Choroideremia in Denver, Colorado in June 2014. This well-received 2-day symposium was organized by the Choroideremia Research Foundation for experts in many different fields to share their work and to potentially collaborate with each other. We heard from clinical and academic scientists located worldwide working in the areas of ophthalmology, genetics, advanced imaging technologies, public health, stem cell biology, gene therapy, drug therapy and cellular engineering. It was enlightening and invigorating to be among great researchers – we also had the opportunity to meet friends and colleagues, new and old!

Back at our lab, we have been fine-tuning the methods and equipment we will eventually be using to assess vision in the clinical trial. Vision is the main outcome measure to assess the results of the clinical trial. Local choroideremia patients have been helping us by allowing us to test our equipment on them. This testing also allows us to tell whether a patient meets the eligibility criteria for the clinical trial. This data is very similar to the clinical data that potential participants from across Canada have been sending us.

We’ve also added a new Publications & Presentations page that lists our recent scientific publications and presentations as well as the media coverage on our study. Check it out!

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Trial Approved by Health Canada

Happy New Year!

We are looking forward to 2014 and all that it will bring. We finished off 2013 on a high note: receiving our No Objection Letter (NOL) from Health Canada! This of course, means we have regulatory approval for the trial!

We will likely begin pre-screening potential participants soon. However, we are uncertain when actual recruitment will begin: valid questions raised by Health Canada in the NOL will need to be addressed prior to patient recruitment.

As an aside, the official funding announcement (article and video) from Alberta Innovates – Health Solutions was made in October.

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Summer Update

Hi Everyone!

We are all getting very excited about the upcoming trial this fall! In the last few months, we have had a pre-Clinical Trial Application (CTA) submission meeting with Health Canada and have since submitted our CTA. Now we are awaiting their response with anticipation!  In the meantime, we are preparing for the recruitment phase by putting together a patient selection committee made up of experts in the field of ophthalmology. The committee does not include our primary investigator, Dr. Ian MacDonald.

In July, our vitreoretinal surgeons, Drs. Matt Tennant and Riz Somani spent a few days with Professor MacLaren in Oxford to learn more about the testing protocols, surgical equipment and procedures used in the UK clinical trial. What they have learned will be invaluable for our Canadian trial.

As you may know this CHM clinical trial is part of the Alberta Ocular Gene Therapy program that was created in 2012. We are in the midst of hiring new staff to fill support positions within the Team and we’re thrilled to have new faces around the office.

Have a happy summer!

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Alberta Ocular Gene Therapy Team Funded by Alberta Innovates-Health Solutions

28 March 2013.

The Alberta Ocular Gene Therapy Team has been successful in securing $5 Million over 5 years from Alberta Innovates – Health Solutions. The Team, based at the University of Alberta, is led by Dr. Ian MacDonald, Chair of the Department of Ophthalmology and co-led by Dr. Tania Bubela, Associate Professor, School of Public Health.

The funding will create a Centre of Excellence for Ocular Gene Therapy at the University of Alberta. It will support a full translational platform to bring gene therapy research to the clinic. The platform will build on the “first” for Canada gene therapy clinical trial for choroideremia.  The funding will further support research into gene therapy for diseases of the cornea, led by Dr. Ordan Lehmann, and other genetic diseases of the retina.

Across the world, there are a number pending gene therapies for genetic eye diseases. Building a Centre of Excellence here in Alberta will create access for Albertans and Canadians to this promising therapeutic approach, whether developed in Alberta or internationally. Ocular gene therapy presents an achievable goal of personalized medicine and could either restore vision or prevent further vision loss.

The Team brings together basic scientists, gene therapy researchers, geneticists, genetic counsellors, clinicians, ophthalmic surgeons, ethicists, experts in regulatory affairs and public health policy to create a translational pathway for ocular gene therapy. The focused goal of this initiative is to develop the capacity and ability to deliver ocular gene therapy in the clinic in Alberta.

The Team will make use of the significant investment already made to the University of Alberta by the Canadian Foundation for Innovation and the Alberta Government in the creation a “GMP” (good manufacturing practice) facility that is capable of producing vectors for gene therapy. As well, both of these funders, have already invested in the purchase of clinical research test equipment that can be used in these clinical trials to determine the effectiveness of the proposed treatments. This testing will be led by Dr. Yves Sauvé.

The Team is further supported by a CIHR Rare Diseases Emerging Team Grant and funding from Foundation Fighting Blindness and the Choroideremia Research Foundation Canada.

Link to Funding Announcement:

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