FIRST HUMAN CLINICAL TRIAL TO TREAT RP BEGINS
ReNeuron, in partnership with Boston Eye and Ear, has announced that the first patient has been treated in a phase 1 stem cell trial for Retinitis Pigmentosa [RP]. This new trial uses human retinal progenitor cells [hRPC] and pre -clinical trial tests have shown that the cells not only protect the retina from further degeneration but also integrate into the retina and produce new photoreceptor cells. 65 different genetic mistakes have already been linked to RP and this stem cell treatment could potentially treat any of these genetic variants while gene replacement therapy only treats one specific gene variant. The Phase I/II clinical trial is only the first step in a long process and will evaluate the safety, tolerability and preliminary efficacy of ReNeuron’s hRPC cell therapy. 15 Patients with advanced RP will receive the treatment.
Internationally acclaimed researcher Eric Pierce, MD, PhD, Director of the Ocular Genomics Institute and Berman Gund Laboratory for Study of Retinal Degenerations at Massachusetts Eye and Ear and Principal Investigator for the clinical trial, commented: “We are delighted to have treated the first patient in this important Phase I/II clinical trial. The human Retinal Progenitor Cells being tested in the study are promising since they can make photoreceptors. The implanted cells may not only prevent degeneration of patients’ vision but may possibly restore some vision by replacing degenerated photoreceptor cells. We look forward to reporting future progress with this study in the months ahead.”
DIS-CHEM RIDE FOR SIGHT
The 28th Ride for Sight was held on Sunday 14th February 2016 at the Boksburg City Stadium. A very successful event resulted in a sizeable amount being donated to the National Research Fund. Dis-Chem Pharmacies were once again the naming sponsors and we are most grateful for their tremendous support of the event. Thanks also go to our important partners – the City of Ekurhuleni, Springs Wheelers, Hamnet Emergency Communications, ASM Safety Consultants, Road Rangers and Benoni Red Cross. Thank you to all our other sponsors- Dark Horse Brands -suppliers of Trek Bikes and Bontrager, Michamvi Sunset Bay Resort, Eqstra Flexi Rent , Tour De Frans Cycles, The Bicycle Company, Venter Trailers , Safilo- suppliers of Polaroid, Smith and Carrera sunglasses, Coca Cola Canners, Cycle Events, Ezee Quip, Vision Works, Carry On Projects, Go Advertising, Vita-thion, Bausch and Lomb, Transact, Biogen, Ferrimed, Piz Buin, One Stop Tool Hire, Bene Water, B One, Liquor City Towers, Pick n Pay Towers, Club Mykonos, Sealy Posturepedic, 082 Carwash, Linden Cycles, Cyclelab, Karcher, Cathedral Peak Hotel, Mangwanani Spa, Braun, Protea Hotels, Protea Parktonian All Suite Hotel, Executive Search International, the Sanctuary Guest House, Integrated Pump Technology, The Wilds Lions Club, Exclusive Hire; water table sponsors – Eqstra Flexi Rent, Helivac, Farrarmere Primary School, City of Ekhurhuleni, Voith South Africa, Dupra Construction, RMS, SNF Auto; Wits Physiotherapy Department, Marshalls- Road Rangers, Rotary and Lions clubs; the Utsaha Yoga school for dozens of muffins; the hundreds of volunteers who helped on the day- we are grateful to every single person, company and organization. Grateful thanks to DSTV, Prime Media and all the radio stations and press who gave us support to advertise the ride. The cyclists who donated R62000 to the competition draw to win a Trek MTB or a holiday at Michamvi Sunset Bay resort in Zanzibar. The Dis-Chem Foundation who generously matched this amount. THANK YOU to everyone for their generous assistance.
[Pictured from left, Nolan Hoffman 2nd place, Ekurhuleni Director for Sport Atwell Sijadu and the winner of the 2016 Dis-Chem Ride for Sight Raynard Butler]
OPTOGENETIC NEWS – The first RP patient has been injected with a virus containing DNA from a light sensitive alga at the Retina Foundation in Texas. Pre -clinical trials showed that in this type of Photoswitch therapy, the alga DNA integrates into the ganglion nerve cells enabling them to send visual information to the brain. This will bypass the damaged photoreceptors and restore some vision. 14 More patients will receive the injection in this phase 1 clinical trial organized by Retrosense Therapeutics.
STEM CELL THERAPY FOR CATARACTS
Cataracts – the clouding of the natural lens that focuses light onto the retina is the leading cause of vison loss worldwide. The only current treatment is to extract the lens and replace it with an artificial intra- ocular lens. An article published in NATURE on 9 March reported on the successful use of stem cells to regenerate the lens. Researchers have developed a method to isolate lens epithelial stem/ progenitor cells whilst removing the damaged cells. The therapy has been successful in mammals and human infants born with congenital cataracts. Patients with retinal degeneration [RD] often develop cataracts and it is hoped that in time this therapy will be commonly available. For a full report see :http://www.nature.com/nature/journal/vaop/ncurrent/full/nature17181.html
NEW HOPE FOR DOMINANT RP
Spark Therapeutics USA has acquired Genable Technologies, an Irish based leading gene therapy innovator. Genable’s RhoNova is a promising treatment for the Rhodopsin form of Dominant RP. The treatment uses an adenoassociated virus to both suppress the faulty gene and to deliver a normal copy of the gene. Rhodopsin mutations are one of the most common causes of Dominant RP worldwide. The treatment has been granted Orphan Drug Designation in both the U.S. and Europe in addition to the Advanced Therapy Medicinal Product designation from the European Medicines Agency. Spark Therapeutics are also bringing the RPE65 gene replacement therapy to the market soon. A. High, MD, co-founder, president and chief scientific officer of Spark said . “We look forward to using our validated gene therapy development platform and expertise to accelerate the clinical development of RHoNova, while examining other potential applications for the Genable suppression/augmentation approach”.
Both the Foundation Fighting Blindness [USA] and Fighting Blindness Ireland were early investors in Genable. They now have the satisfaction of seeing the research move towards therapy but have also gained a modest return on their investment. ” This acquisition is a great example of how patient led research can progress towards therapies.” said Christina Fasser, President of Retina International.
Many exciting treatments for Age Related Macular Degeneration [AMD] are under investigation. However, various forms and combinations of Anti –VEGF treatments remain the best options for treatment of the wet form of this devastating retinal degeneration at this time. The monthly injections that are needed to maintain any visual gain is very onerous on the patient. Use of Encapsulated Cell Technology [ECT] for the slow release of Lucentis has moved to a Phase 2 clinical trial – NT -503. ECT is a capsule that is implanted into the eye and releases the drug slowly over a long period and will negate the monthly injections of anti- VEGF. ECT was originally developed to administer growth factors and is still being investigated for both AMD and RP.
- A new oral drug X82 in being tested by the biopharmaceutical company Tyrogenex, to inhibit VEGF and stop the formation of leaking blood vessels.
- A surgical device that delivers a focused dose of radiation [epimacular brachytherapy] is still under investigation. The radiation is now being administered in conjunction with Lucentis anti- VEGF.
- Another study is evaluating the benefits of low voltage external beam radiotherapy.
There are over 1000 ongoing clinical trials for both the wet and dry form of AMD and they offer real hope for more affordable, less intrusive and more effective future therapy.
IMT NOW IN SOUTH AFRICA
CentraSight’s IMT device has been extensively tested in the USA. It is a tiny telescope that is implanted inside the eye to improve vision in AMD. Smaller than a pea, the telescope implant uses micro-optical technology to magnify objects that would normally be seen in one’s central visual field. This image is projected onto a healthy part of the retina not affected by the disease. The telescope implant is embedded in an Intraocular lens which is then inserted into the natural lens capsule. Although there is much to be gained with the telescope patients will lose the right to drive. The CentraSight treatment program focuses on comprehensive patient care, requiring potential patients to undergo medical and vision evaluation to determine whether they may be a good candidate. A unique aspect of the evaluation is the ability to simulate, prior to surgery, what a person may expect to see once the telescope is implanted to determine if the improvement possible will meet the patient’s expectations. After an eye surgeon implants the telescope in an outpatient procedure, the patient then works with vision specialists to learn how to use their new vision in their everyday activities. The telescope implant is not a cure. Patients must meet age and cornea health requirements to be considered a good candidate. Possible side effects include decreased vision or vision impairing corneal swelling. The CentraSight treatment program is now available at select treatment centers in South Africa. For more information contact Retina South Africa or visit www.CentraSight.com.
CLINICAL TRIALS AND YOU
Retina South Africa is actively working to bring reputable clinical trials to South Africa, and we are hoping this will happen in the near future. Will you be eligible? You need to: Check that your contact details are up to date. Ensure that you have a recent clinical diagnosis and if you have a genetic result ensure that Retina South Africa has a copy.
Where possible assist in our fund raising projects: Order a raffle book- R100 for 10 tickets to win a car; take a ticket in our Circle of Light competition- R250 for a chance to win R500 per month for a year; SMS “Dream” to 38267 – R10 per SMS and encourage all your friends and family to do the same. Contact you nearest branch to offer your help. With your help we can make the dream of bringing clinical trials to SA a reality. Donations exceeding R2000 are eligible for a Section 18a tax deduction certificate.
A membership fee of R100 per family is now due. Please deposit membership fees plus any donations and fundraising contribution to Retina South Africa, Standard Bank, Bedfordview branch, Code 018305, Account number 020312164. For overseas payment the swift code is SBZAZAJ. Credit card payments are accepted via the national office on 0860595959. Please send us a copy of the EFT and your latest contact details so that your membership fee can go to your local branch.
THANK YOU BAUSCH AND LOMB FOR YOUR CONTINUED SUPPORT OF OUR WORK.