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What Clinical Trials Can We Expect for RD in the Future?

What Clinical Trials Can We Expect for RD in the Future?. Groups of investigators are now working on several of the Rare Diseases to move to Trials.

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What Clinical Trials Can We Expect for RD in the Future?

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  1. What Clinical Trials Can We Expect for RD in the Future? Groups of investigators are now working on several of the Rare Diseases to move to Trials. • Leber Disease: Three groups have started Clinical Trials on Gene Replacement Therapy for a form of LCA. At least one other group also is planning an LCA Trial.

  2. Future Clinical Trials 2) Stargardt Disease: Dr. Rando Allikmets and coworkers are working on Gene Replacement Therapy of the mutated gene in a rodent model of Stargardt Disease. So far, the results are preliminary but good and, if finally positive for safety and efficacy, a Clinical Trial is planned. Other research groups are working on similar Gene Therapy efforts for Stargardt Disease.

  3. Future Clinical Trials 3) Usher Disease: Research groups are working on both Usher 1 and Usher 3 to replace the mutated genes in the two conditions through Gene Therapy. • Producing good animal models in both conditions has been a problem that is currently being overcome. • Progress is being made as reported by Dr. Flannery

  4. Future Clinical Trials 4) Choroideremia: Scientists are close to producing a good rodent model for Choroideremia. They will then use Gene Therapy to replace the mutated gene in the model. If successful, a human Clinical Trial can be planned. 5) Similar Gene Replacement is planned for some of the other Rare Diseases such as Retinoschisis.

  5. What about AMD?CURRENT AMD TREATMENTS Dry AMD – nutrition therapy. The antioxidants studied in the AREDS clinical trial of the NEI are available. Wet AMD – several drugs now have been approved by government agencies in many countries to slow the growth of new, abnormal blood vessels. The best known is Lucentis.

  6. Nutrition and AMD AREDS Antioxidant Treatment A Clinical Trial for antioxidants in treating AMD has been completed by the National Eye Institute. It was called the Age-Related Eye Disease Study (AREDS). It found that some nutritional supplements helped in AMD. The antioxidant nutrients studied were B-carotene and vitamins C and E along with the mineral zinc. The antioxidants only slow the course of the disease at a specific (mid) stage of AMD. The antioxidants are available for sale although a physician should be consulted before using them.

  7. Clinical Treatments – Wet AMD • Lucentis – Genentech has an agent, “Lucentis” for wet AMD. Lucentis is an antibody that works against VEGF, a small protein that calls in the new, abnormal blood vessels. Lucentis actually improves vision. • One of the problems with Lucentis though is that it must be injected into the eye. • Also, there usually have to be repeat injections. • Another problem is the high cost but a similar agent, Avastin, is available at much lower cost.

  8. AMD Clinical Trials –Gene Therapy Trial • GenVecis conducting a Gene Therapy Clinical Trial for wet AMD. It delivers the PEDF gene into the eye. Phase 1 of the Trial is completed and the safety results are very good. • PEDF is a natural protein that has both antineovascular and neuron-survival properties. Thus, it could be used for both AMD and RP. • If the results on wet AMD are positive, GenVec could then move on to the use of PEDF in RP and in dry AMD along with wet AMD.

  9. Clinical Trials – Wet AMD Several other agents are being tested as anti-neovascular agents. For example: • The company Oxigene has an agent, Combretastatin, that they have used as an antineovascular agent in cancer. • It is now in a Clinical Trial for wet AMD. • This drug can be delivered to the eye through the blood so is fairly safe. • Other similar agents are being developed.

  10. AMD Clinical Trials - Nutrition • A Nutrition Trial called AREDS2 with lutein is underway in AMD patients at the NEI. • Lutein/Zeaxanthin are carotenoids, the colored pigments in fruits and vegetables, that are concentrated in the human retina – especially the macula. It is thought that they act as antioxidants and thus protect photoreceptor cells from oxidative damage. • This Trial will take several years though to complete. • Until then, take your mother’s advice and “Eat your fruits and vegetables!”

  11. In Conclusion…. Several Clinical Trials are planned or are already in progress for different types of RD therapies. For example, Gene Therapy work in RP animal models shows not only sight restoration but a long term, positive effect. Is this a “cure”? • Other basic work in the fields of gene therapy, stem cell research, pharmaceutical therapy, nutrition and electronic implants shows promise with several Clinical Trials in progress. • The retinal prosthetic device could be the best hope for sight restoration for patients with advanced retinal degeneration.

  12. A Final Thought….. • We can treat and, in some cases, maybe even cure diseases in many animal models of retinal degeneration. • Many human Clinical Trials are starting such that treatments are coming soon. • These are expensive and time consuming….. but who can put a price on restoring sight?

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