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Optogenetic Therapy Takes First Step Forward in Clinical Trial

Retrosense logoRetroSense Therapeutics has reported that three participants have received injections of its potential optogenetic therapy, known as RST-01, in a Phase I/II clinical trial. The patients were given the lowest dose of RST-01, and no adverse ocular events were observed. Furthermore, the treatment showed some biological activity, though RetroSense did not provide details about what that activity was or what it meant.

More information on safety and efficacy will likely be reported about the RetroSense trial after more trial participants have been observed over a longer period of time, and after discussions with the U.S. Food and Drug Administration.

RetroSense’s optogenetic therapy is designed to restore vision to people who are completely blind from retinal degenerative diseases such as retinitis pigmentosa by bestowing light sensitivity to retinal ganglion cells, which survive after photoreceptors, the cells that make vision possible, are lost. Retinal ganglion cells normally don’t process light; rather, they perform image-processing functions downstream.

RetroSense’s treatment is a gene therapy, which uses a human-engineered adeno-associated virus (AAV) to deliver a gene to retinal ganglion cells that encodes channelrhodpsin-2, a light-sensing protein.

A benefit of the therapy is that it has the potential to work regardless of the gene mutation causing the patients’ vision loss.

The company plans to inject the treatment into what they call the “mid-dose” cohort within the next month. Dose escalation is a common practice in early clinical trials focused on safety. If no adverse events are observed at a lower dose level, the treatment developers will increase the dose, hoping they will observe efficacy (i.e., changes in vision).

RetroSense’s clinical trial is taking place at the Retina Foundation of the Southwest and is the first-ever for an optogenetic treatment.

The Foundation Fighting Blindness provided early funding for the development of RetroSense’s treatment and participated in discussions about the clinical trial with the U.S. Food and Drug Administration.


17 Responses to 'Optogenetic Therapy Takes First Step Forward in Clinical Trial'

  1. Lisa Davis says:

    Are these trials only being conducted on persons who have NLP? I have cone-rod dystrophy (genetic). My vision has progressively deteriorated over time and is now 20/400 with a field of vision about 10 degrees. I have researched possible studies or treatments with no success. Any information would be greatly appreciated.

    • EyeOnTheCure says:

      Dear Lisa, To obtain more information on the Retrosense, optogenetic trial, please see the following link to the Clinical Trials website: https://clinicaltrials.gov/ct2/show/NCT02556736?term=retrosense&rank=1 . As you will read, only individuals with RP who have vision at the hand motion / light perception level will be considered for this phase 1 safety trial. Once Phase 1 is completed, it is possible that the new criteria will exclude those with no light perception (NLP). For this treatment to succeed, one must have an intact optic nerve along with some light perception.

  2. Lawanda D Landsittel says:

    I have three siblings with RP, two sisters and one brother. They are all legally blind.

    • EyeOnTheCure says:

      Dear Lawanda, You and your siblings should know that there are three inherited forms of RP: recessive, dominant and X-linked. If you are not sure which type you have, you should ask your ophthalmologist. For more information on inheritance, please see the following web link to download a PDF document on inheritance:

      http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf

      Your siblings should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether the disease gene is identified or not, your siblings should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. Here is the link to clinical trials for retinitis pigmentosa: https://clinicaltrials.gov/ct2/results?term=retinitis+pigmentosa&Search=Search
      Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

  3. Raymond Wirebaugh says:

    How soon will this treatment be available to the public? I am a 60 year old male who has lost all vision to RP.

    • EyeOnTheCure says:

      Retrosense optogenetic clinical trial is currently in the “Phase 1” safety stage. Phase I studies are primarily concerned with assessing the drug’s safety and establishing a maximum tolerated dose for the drug. This initial phase of testing in humans is done in a very small number of patients.
      Phase II studies are initiated once a drug is found to be safe. In a Phase II study, the effectiveness of a given drug or treatment is evaluated. The duration of a phase II study depends greatly on the nature of the treatment being tested and the specific disease. Phase III studies allow investigators to better gauge the effectiveness and side effects of a drug in a large population of diverse patients. The large-scale testing provides the pharmaceutical company and the FDA with a more thorough understanding of the drug’s effectiveness, benefits, and range of possible adverse reactions. The time required to go from PHase 1 to Phase 3 can range anywhere from 3 to 10 years.

  4. Jay says:

    I need help. My name is Jay Tardugno.
    I am the president of ISP Surgical in bangkok Thailand !
    My best friends sisters, Robbie , is suffering with RP!
    She is in Maimi !
    Can we get her in the clinical ?
    We are desperate !
    Warm Regards,
    Jay
    President
    ISP Surgical
    Ispsurgical.com

    • EyeOnTheCure says:

      Hi Jay, There are a number of things that your friend’s sister can do. First of all, there is an excellent RP specialist at the Bascom Palmer Eye Clinic in Miami. His name is Dr. Byron Lam. Here is a weblink to his contact information: http://bascompalmer.org/doctors/profile/1244
      Dr. Lam should be able to help your friend’s sister obtain a specific diagnosis as there are many different types of RP. Dr. Lam will also be able to help her obtain genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, she may be able to get into one of the gene therapy trials that are taking place. For more information on the genetic testing process, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether your friend’s sister disease gene is identified or not, she should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials that are taking place. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. Finally, please check the FFB website (www.fightblindness.org) frequently as new information on retinal disease treatments is posted almost every week. I hope this information is of help to you and thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

  5. Mayur Patel says:

    my brother has problem of RP. He is 27 years. I am from Gujarat my cell no. is +91 7600526063 and +91 9998141398. And my email id is patelmayur21193@gmail.com. I shall be grateful to you in case of any suggestion of treatment. Also, in case any of you have any ideas or suggestion of stopping the spread of this disease.please replay near future can i get this treatment possible? Hope to get reply from your end soon.
    Thanks.

  6. David Barnes says:

    The research sounds promising. I have cone rod degeneration with loss of functional sight in both eyes. What are the participant eligibility requirements and are you accepting new participants? What are the risks, side effects, etc.? Have your participants noticed improvement? 48 yr old electrical eningeer, visual acuity 20/400

    • EyeOnTheCure says:

      Dear David, Regarding your interest in participating in the Retrosense optogenetic trial, please see the link below to the Retrosense trial on the Clinical Trials.Gov website. The website has information on the exclusion and inclusion requirements as well as a phone number for the clinical trial coordinator, which in this case is Dr. David Birch. Dr. Birch is happy to answer any questions you might have regarding possible side effects and benefits. As this trial has just begun the Phase 1 safety phase, there is no information available yet on efficacy. Here is the link: https://clinicaltrials.gov/ct2/show/NCT02556736?term=retrosense&rank=1

  7. John Poulli says:

    My 6 year old son has just been diagnosed with Rod Cone Dystrophy. As a parent I am desperate to find him help.
    Please let me know if there is anything you can do to help.

    Thanks

    • EyeOnTheCure says:

      Dear John, If you son was diagnosed with rod cone dystrophy, then he probably has a condition similar to retinitis pigmentosa. If on the other hand he was diagnosed with cone-rod dystrophy, he may have a condition similar to Stargardt disease. Regardless of the phenotypic diagnosis, you should consider genetic testing for him to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, he may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether his disease gene is identified or not, you should still consider enrolling him in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      You may also find it helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

  8. Andrea says:

    It looks like this clinical trial is currently recruiting participants according to http://www.clinicaltrials.gov. Copy and paste the following in your browser for more information. https://clinicaltrials.gov/ct2/show/study/NCT02556736?term=retrosense&rank=1#contacts

    Or go to http://www.clinicaltrials.gov and put in NCT02556736. They are recruiting adults 18 and over and the list of requirements are on the site. Hope this helps!

  9. sajad says:

    My son is suffering from RP by brith.He is 21 years old.He is loosing vision day by day.He has lost all hopes.I am trying to convance.soon there will be treatment.please help me. my phone no 9797111178.

    • EyeOnTheCure says:

      Dear Sajad, Your son should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf
      Your son should also consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/
      Finally, you and your son may find it helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

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