Scientists from the University of California, Berkeley and Lawrence Berkeley National Laboratory, along with those of University of Pennsylvania, have demonstrated restoration of visual function in animal models that can help restore sight in blind patients afflicted by diseases such as Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA).
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Showing posts with label Pigmentosa. Show all posts
Showing posts with label Pigmentosa. Show all posts
Thursday, December 11, 2014
Wednesday, July 23, 2014
Treatment efficacy of topical unoprostone isopropyl in patients with retinitis pigmentosa
A study was conducted by Dr Akiyama & associates from the Department of Ophthalmology, Graduate School of Medical Sciences, Kyushu University, Fukuoka in Japan to evaluate the treatment effect of topical unoprostone isopropyl (unoprostone) (in the form of eye drops) on patients with retinitis pigmentosa (RP). This medication is currently used in glaucoma.
Forty patients with typical forms of RP were included in the study, out of which 17 patients were treated with 0.12% topical unoprostone twice daily in a randomly selected eye. The efficacy of the treatment was monitored by visual acuity and visual field testing using the Humphrey Field Analyzer (HFA). In addition, 12 RP patients who were included this study and 12 normal subjects were evaluated in terms of their macular blood flow of both eyes after instillation of unoprostone using the laser speckle method.
Forty patients with typical forms of RP were included in the study, out of which 17 patients were treated with 0.12% topical unoprostone twice daily in a randomly selected eye. The efficacy of the treatment was monitored by visual acuity and visual field testing using the Humphrey Field Analyzer (HFA). In addition, 12 RP patients who were included this study and 12 normal subjects were evaluated in terms of their macular blood flow of both eyes after instillation of unoprostone using the laser speckle method.
Is Valproic Acid effective in treating patients with Retinitis Pigmentosa?
There have been some reports in academic journals about the use of Valproic acid (VPA) in Retinitis Pigmentosa (RP). We thought we can review the current status of these trials and evaluate where we stand in the outcomes.
Saturday, May 10, 2014
New technique to determine gene carriers for autosomal recessive retinitis pigmentosa
Scientists from Bascom Palmer Eye Institute and Duke University Medical Center have developed a non-invasive technique to determine if individuals carry a gene for the autosomal recessive type of retinitis pigmentosa.
The work was being presented at the 2014 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Orlando, Florida.
The technique involves collecting a patient's urine and measuring the ratio between specific compounds. The non-invasive process makes subsequent testing clinic-friendly, especially for children being screened.
In search of quantitative biomarkers for the disease, the authors checked on the urinary and plasma dolichol profiles in autosomal recessive RP (arRP) patients and carriers with mutations in the DHDDS gene encoding dehydrodolichol diphosphate synthase, a key enzyme in dolichol biosynthesis. Dolichols are long chain polyisoprenoid alcohols composed of 17-21 isoprene units.
Mutations in the DHDDS gene lead to a characteristic shortening of plasma and urinary dolichols, which, as per the authors of this study, can be used as a functional readout of the enzyme. Urinary and plasma D18/D19 ratios reliably determine if a DHDDS genotype is disease-causing.
D18/D19 ratio is a viable objective functional biomarker and can be readily adapted as a clinical test for arRP diagnosis and carrier screening with DHDDS or other genetic mutations that impair dolichol biosynthesis.
Wednesday, May 7, 2014
DENAQ, a new chemical that may restore sight in retinitis pigmentosa and macular degeneration
Scientists from the University of California, Berkeley report on a chemical ‘photoswitch’ named DENAQ that may be a potential drug for treating patients suffering from blinding diseases such as retinitis pigmentosa (RP) and age-related macular degeneration (AMD).
RP and AMD are blinding diseases caused by the degeneration of rods and cones, leaving the remainder of the visual system unable to respond to light. In the paper published in Neuron, the authors led by Dr Richard H Kramer report about a chemical photoswitch named DENAQ that restores retinal responses to white light of intensity similar to ordinary daylight, compared to earlier attempts at photoswitch that required very bright ultraviolet light, making it unsuitable for use in patients. In experiments conducted on three-month to six-month-old healthy mice, and on mice carrying a mutation causing nearly all their rods and cones to degenerate by the time they were a month old, a single intraocular injection of DENAQ has been able to photosensitize the blind retina for about 3 days, restoring electrophysiological and behavioral responses with no toxicity. DENAQ also is able to rapidly turns itself off, allowing rapid repeated stimulation of the retina. The researchers compared the retinae of DENAQ-injected mice to untreated healthy mice and found no signs of toxicity for up to 30 days after the injection.
Retinas with damaged rods and cones are subject to several morphological and biochemical changes, making them different from a healthy retina in more than one way. Experiments on mouse strains with functional, nonfunctional, or degenerated rods and cones show that DENAQ is effective only in retinas with degenerated or dead photoreceptors. Apparently, the degenerated outer retinal cells help in DENAQ photosensitization due to changes in electrophysiological characteristics, while the presence of intact photoreceptors possibly prevent this action. This appears to give it significant advantage, since this selective action on diseased tissue alone may potentially reduce side effects on healthy retina.
DENAQ confers light sensitivity on a hyperpolarization-activated inward current that is enhanced in degenerated retina, enabling optical control of retinal ganglion cell firing. The acceptable light sensitivity, favorable spectral sensitivity, and selective targeting to diseased tissue make DENAQ a prime drug candidate for vision restoration in patients with end-stage RP and AMD.
The researchers plan on conducting more experiments, including in larger animals before its safety can be established and any clinical trials can be considered.
To watch a video of Dr Kramer explaining his work, click here.
Sources: Neuron, Univ of California, Berkeley, Kramer Lab, The Scientist
Editor's note: The drug appears to have an exciting potential to help patients with retinitis pigmentosa and age-related macular degeneration. There are two thoughts though.
1. Such a drug may only work in advanced stages of the disease, considering we will have to wait for the retinal cells to die before it can act.
2. We also need to know how long the effect of one injection lasts. Such treatment may require repeat injections over a long-term, something similar to Lucentis or Avastin.
RP and AMD are blinding diseases caused by the degeneration of rods and cones, leaving the remainder of the visual system unable to respond to light. In the paper published in Neuron, the authors led by Dr Richard H Kramer report about a chemical photoswitch named DENAQ that restores retinal responses to white light of intensity similar to ordinary daylight, compared to earlier attempts at photoswitch that required very bright ultraviolet light, making it unsuitable for use in patients. In experiments conducted on three-month to six-month-old healthy mice, and on mice carrying a mutation causing nearly all their rods and cones to degenerate by the time they were a month old, a single intraocular injection of DENAQ has been able to photosensitize the blind retina for about 3 days, restoring electrophysiological and behavioral responses with no toxicity. DENAQ also is able to rapidly turns itself off, allowing rapid repeated stimulation of the retina. The researchers compared the retinae of DENAQ-injected mice to untreated healthy mice and found no signs of toxicity for up to 30 days after the injection.
Retinas with damaged rods and cones are subject to several morphological and biochemical changes, making them different from a healthy retina in more than one way. Experiments on mouse strains with functional, nonfunctional, or degenerated rods and cones show that DENAQ is effective only in retinas with degenerated or dead photoreceptors. Apparently, the degenerated outer retinal cells help in DENAQ photosensitization due to changes in electrophysiological characteristics, while the presence of intact photoreceptors possibly prevent this action. This appears to give it significant advantage, since this selective action on diseased tissue alone may potentially reduce side effects on healthy retina.
DENAQ confers light sensitivity on a hyperpolarization-activated inward current that is enhanced in degenerated retina, enabling optical control of retinal ganglion cell firing. The acceptable light sensitivity, favorable spectral sensitivity, and selective targeting to diseased tissue make DENAQ a prime drug candidate for vision restoration in patients with end-stage RP and AMD.
The researchers plan on conducting more experiments, including in larger animals before its safety can be established and any clinical trials can be considered.
To watch a video of Dr Kramer explaining his work, click here.
Sources: Neuron, Univ of California, Berkeley, Kramer Lab, The Scientist
Editor's note: The drug appears to have an exciting potential to help patients with retinitis pigmentosa and age-related macular degeneration. There are two thoughts though.
1. Such a drug may only work in advanced stages of the disease, considering we will have to wait for the retinal cells to die before it can act.
2. We also need to know how long the effect of one injection lasts. Such treatment may require repeat injections over a long-term, something similar to Lucentis or Avastin.
Thursday, July 26, 2012
A new potential treatment for Retinitis Pigmentosa & Macular Degeneration
A team of researchers from the University of California, Berkeley, in collaboration with researchers at University of Munich and University of Washington in Seattle have found a chemical that can temporarily restore some amount of vision in blind mice.
This compound could eventually help those with Retinitis Pigmentosa, a common genetic disease that leads to blindness, as well as Age-related Macular Degeneration. The chemical, referred to as AAQ, acts on the remaining cells in the retina, which are normally "blind" cells, sensitive to light. AAQ is a photoswitch that binds to protein ion channels on the surface of retinal cells; when switched on by light, it alters the flow of ions through the channels and activates these neurons similar to the way rods and cones are activated by light.
This chemical has been shown to eventually wear off, and hence may offer a safer alternative to other approaches that restore sight, such as gene or stem cell therapies, which may permanently change the retina. It is also less invasive than implanting light-sensitive chips in the eye.
Considering that it is a simple chemical, it will be easy to change the dosage, use it in combination with other therapies, or discontinue the therapy if the need be. This would allow new and improved chemicals that may become available in time to be offered to the same patients.
The blind mice in the experiment had genetic mutations that made their rods and cones die within months of birth and inactivated other photopigments in the eye. After injecting very small amounts of AAQ into the eyes of the blind mice, the researchers confirmed that they had restored light sensitivity because the mice's pupils contracted in bright light. The mice also demonstrated light avoidance, a typical rodent behavior impossible without the animals being able to see some light. The researchers are hoping to conduct more sophisticated vision tests in rodents injected with the next generation of the compound.
It will be a while before this compound will find its way in humans. The researchers have to show that these compounds are safe and will work in patients the way they work in mice. BUt these preliminary results demonstrate that this class of compound restores light sensitivity to retinas blind from genetic disease.
The current technologies being evaluated for restoring sight to people whose rods and cones have died include injection of stem cells to regenerate the rods and cones; "optogenetics," a type of gene therapy where a photoreceptor gene is inserted into blind neurons to make them sensitive to light; and installation of electronic prosthetic devices, such as a small light-sensitive retinal chip with electrodes that stimulate blind neurons.
Eight years ago, Kramer, Trauner, a former UC Berkeley chemist now at the University of Munich, and their colleagues developed an optogenetic technique to chemically alter potassium ion channels in blind neurons so that a photoswitch could latch on. Potassium channels normally open to turn a cell off, but with the attached photoswitch, they were opened when hit by ultraviolet light and closed when hit by green light, thereby activating and deactivating the neurons.
Subsequently, Trauner synthesized AAQ (acrylamide-azobenzene-quaternary ammonium), a photoswitch that attaches to potassium channels without the need to genetically modify the channel.
Newer versions of AAQ now being tested have demonstrated better results, as per the researchers. They activate neurons for days rather than hours using blue-green light of moderate intensity, and these photoswitches naturally deactivate in darkness, so that a second color of light is not needed to switch them off.
Source
Tuesday, March 6, 2012
POSITIVE PRELIMINARY RESULTS FROM PHASE 1b TRIAL OF QLT091001 IN SUBJECTS WITH RETINITIS PIGMENTOSA DUE TO RPE65 AND LRAT MUTATIONS
QLT Inc. has
announced positive preliminary results from its international multi-center Phase 1b proof-of-concept
clinical trial of QLT091001 for the treatment of Retinitis Pigmentosa (RP) due to inherited genetic
mutations in retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT)
(also known as early-onset RP).
The Phase 1b study showed rapid, statistically significant and clinically meaningful changes in visual fields (VF) from baseline values, as well as improvements in visual acuity (VA), in the study of 17 RP subjects. In addition, small subsets of RP subjects were investigated for secondary effects on other key vision parameters impacted by RP, such as decreased retinal sensitivity, and the data available in these subsets showed notable and promising increases in average sensitivity levels. The single-course treatment data with QLT091001 represents the first stage of dose regimen testing as the basis for a longer term multiple course regimen in RP due to mutations in RPE65 and LRAT.
RP is a disabling group of genetic eye diseases associated with progressive loss of vision including night blindness, constricted peripheral vision resulting in difficulties with daily activities, and in later life, reduced central vision, inability to read, and in many cases progression to severe blindness. RP can be caused by many different gene defects and symptoms can start at varying ages; patients with mutations in the RPE65 and LRAT genes tend to show vision loss very early in life (this type of RP is also known as early-onset RP).
In the open-label, multi-center Phase 1b clinical study, 17 subjects (ranging in age from 6 to 55 years, mean 29 years) with either RPE65 (12 subjects) or LRAT (5 subjects) mutations received a 40 mg/m2/day dose of QLT091001 once daily for seven days with post-treatment follow-up at 7, 14, and 30 days. Visual fields and visual acuity are key measures of clinically relevant visual function. VF was assessed using Goldmann Visual Fields (GVF) and VA was assessed using best-corrected visual acuity (BCVA, ETDRS letters); GVF maps were converted to assess the remaining functional retinal area for analysis. After a single 7-day course of treatment with QLT091001, the average retinal areas from baseline showed statistically significant improvements of 34% at day 7 (p=0.005), 29% at day 14 (p=0.02) and trended towards a statistically significant improvement of 23% at day 30 (p=0.07) in the evaluable subjects meeting GVF test criteria (n=14 subset). In the intent-to-treat (ITT; all subjects enrolled) analysis (n=17), the average retinal area from baseline improved by 22% at day 7 (p=0.03, statistically significant), 16% at day 14 (p=0.13) and 18% at day 30 (p=0.096). The evaluable subset of 14 subjects excludes three patients in the VF analysis because they did not meet criteria as determined by a third-party reader. Nine of 17 subjects (53%) showed an improvement in VA over baseline in at least one eye by greater than or equal to five ETDRS letters.
Derived from the press release from QLT Inc.
The Phase 1b study showed rapid, statistically significant and clinically meaningful changes in visual fields (VF) from baseline values, as well as improvements in visual acuity (VA), in the study of 17 RP subjects. In addition, small subsets of RP subjects were investigated for secondary effects on other key vision parameters impacted by RP, such as decreased retinal sensitivity, and the data available in these subsets showed notable and promising increases in average sensitivity levels. The single-course treatment data with QLT091001 represents the first stage of dose regimen testing as the basis for a longer term multiple course regimen in RP due to mutations in RPE65 and LRAT.
RP is a disabling group of genetic eye diseases associated with progressive loss of vision including night blindness, constricted peripheral vision resulting in difficulties with daily activities, and in later life, reduced central vision, inability to read, and in many cases progression to severe blindness. RP can be caused by many different gene defects and symptoms can start at varying ages; patients with mutations in the RPE65 and LRAT genes tend to show vision loss very early in life (this type of RP is also known as early-onset RP).
In the open-label, multi-center Phase 1b clinical study, 17 subjects (ranging in age from 6 to 55 years, mean 29 years) with either RPE65 (12 subjects) or LRAT (5 subjects) mutations received a 40 mg/m2/day dose of QLT091001 once daily for seven days with post-treatment follow-up at 7, 14, and 30 days. Visual fields and visual acuity are key measures of clinically relevant visual function. VF was assessed using Goldmann Visual Fields (GVF) and VA was assessed using best-corrected visual acuity (BCVA, ETDRS letters); GVF maps were converted to assess the remaining functional retinal area for analysis. After a single 7-day course of treatment with QLT091001, the average retinal areas from baseline showed statistically significant improvements of 34% at day 7 (p=0.005), 29% at day 14 (p=0.02) and trended towards a statistically significant improvement of 23% at day 30 (p=0.07) in the evaluable subjects meeting GVF test criteria (n=14 subset). In the intent-to-treat (ITT; all subjects enrolled) analysis (n=17), the average retinal area from baseline improved by 22% at day 7 (p=0.03, statistically significant), 16% at day 14 (p=0.13) and 18% at day 30 (p=0.096). The evaluable subset of 14 subjects excludes three patients in the VF analysis because they did not meet criteria as determined by a third-party reader. Nine of 17 subjects (53%) showed an improvement in VA over baseline in at least one eye by greater than or equal to five ETDRS letters.
|
Measurement of Goldmann Visual Field Improvement from Baseline
|
|||
|
GVF analysis – retinal area
|
Day 7
|
Day 14
|
Day 30
|
|
Avg. increase from baseline – evaluable
subjects (n=14)
Avg. increase from baseline – ITT (n=17) |
34% (p=0.005)*
22% (p=0.03)*
|
29% (p=0.02)*
16% (p=0.13)
|
23% (p=0.07)
18% (p=0.096)
|
(*statistically significant)
Following single-course treatment with QLT091001, the Retinitis Pigmentosa patients in the clinical trial experienced a rapid and significant improvement in certain visual function parameters, as per Dr. Hendrik Scholl of the Wilmer Eye Institute at Johns Hopkins University. He also added that the discovery of the genetic cause of retinal degeneration has revolutionized insight into disease processes at a molecular level and has given the physicians encouragement for potential therapeutic approaches.
Baseline values in subjects showed a broad range of moderately to severely reduced visual acuities and visual fields. All subjects reported early onset of night blindness as one of the hallmarks of disease.
Additional tests included spectral-domain optical coherence tomography (OCT), full-field electroretinography (ERG), and quality of life assessments. Also, in small subsets of subjects, the effects of QLT091001 on several parameters of light sensitivity in dim light (night vision), pupillary reflexes, and responses of the visual cortex to potential changes in visual stimuli (functional magnetic resonance imaging, fMRI) were measured.
The fMRI substudy (n=2) showed activation of several previously quiet areas of the visual and parietal cerebral cortex after treatment. In addition, the substudy in dark adapted visual fields (n=2) showed a 16- fold average increase in sensitivity at 10%-50% of locations tested in each eye within days of the first dose. With longer dark adaptation, light sensitivity increases averaged 40-80-fold greater than baseline at 40%- 83% of the locations tested. Full-field sensitivity (FST) and pupillometry results supported the large increases in sensitivity in both eyes.
The additional secondary endpoints evaluated to date in this study support and are consistent with the changes in VF and VA observed following single course treatment with QLT091001.
Ongoing analysis of the safety profile of QLT091001 demonstrates a safety profile consistent with the Company’s Phase 1b study in patients with Leber Congenital Amaurosis (LCA). A retreatment study for the RP cohort has been initiated to assess the effects of repeat QLT091001 therapy in these patients. The database on this study remains open and further analyses of additional anatomic tests (OCT) and retinal sensitivity tests (ERG) are also ongoing to further explore the optimal baseline characteristics of responders for clinical development purposes.
Following single-course treatment with QLT091001, the Retinitis Pigmentosa patients in the clinical trial experienced a rapid and significant improvement in certain visual function parameters, as per Dr. Hendrik Scholl of the Wilmer Eye Institute at Johns Hopkins University. He also added that the discovery of the genetic cause of retinal degeneration has revolutionized insight into disease processes at a molecular level and has given the physicians encouragement for potential therapeutic approaches.
Baseline values in subjects showed a broad range of moderately to severely reduced visual acuities and visual fields. All subjects reported early onset of night blindness as one of the hallmarks of disease.
Additional tests included spectral-domain optical coherence tomography (OCT), full-field electroretinography (ERG), and quality of life assessments. Also, in small subsets of subjects, the effects of QLT091001 on several parameters of light sensitivity in dim light (night vision), pupillary reflexes, and responses of the visual cortex to potential changes in visual stimuli (functional magnetic resonance imaging, fMRI) were measured.
The fMRI substudy (n=2) showed activation of several previously quiet areas of the visual and parietal cerebral cortex after treatment. In addition, the substudy in dark adapted visual fields (n=2) showed a 16- fold average increase in sensitivity at 10%-50% of locations tested in each eye within days of the first dose. With longer dark adaptation, light sensitivity increases averaged 40-80-fold greater than baseline at 40%- 83% of the locations tested. Full-field sensitivity (FST) and pupillometry results supported the large increases in sensitivity in both eyes.
The additional secondary endpoints evaluated to date in this study support and are consistent with the changes in VF and VA observed following single course treatment with QLT091001.
Ongoing analysis of the safety profile of QLT091001 demonstrates a safety profile consistent with the Company’s Phase 1b study in patients with Leber Congenital Amaurosis (LCA). A retreatment study for the RP cohort has been initiated to assess the effects of repeat QLT091001 therapy in these patients. The database on this study remains open and further analyses of additional anatomic tests (OCT) and retinal sensitivity tests (ERG) are also ongoing to further explore the optimal baseline characteristics of responders for clinical development purposes.
The RP (early-onset RP) Phase 1b clinical study is being conducted at seven leading centers for the
treatment of inherited retinal diseases in the U.S., Europe and Canada.
About Synthetic Retinoid Drugs
Genetic diseases in the eye such as Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP) arise from gene mutations of enzymes or proteins required in the biochemistry of vision. QLT091001 is a replacement for 11-cis-retinal, which is an essential component of the retinoid-rhodopsin cycle and visual function, and is under investigation for the treatment of LCA and RP. QLT091001 has received orphan drug designations for the treatment of LCA and RP by the European Medicines Agency, and for the treatment of LCA and RP due to inherited mutations in the LRAT and RPE65 genes by the U.S. Food and Drug Administration (FDA). The drug has also been granted two Fast Track designations by the FDA for the treatment of the LRAT and RPE65 genetic mutations in both LCA and RP.
About Leber Congenital Amaurosis (LCA)
LCA is an inherited degenerative retinal disease characterized by abnormalities such as roving eye movements and sensitivity to light, and manifesting in severe vision loss from birth. Both rod and cone photoreceptors are affected in LCA. Eye examinations of infants with LCA reveal normal appearing retinas. However, a low level of retinal activity, measured by electroretinography, indicates very little visual function. According to current epidemiological estimates, LCA affects approximately one in 81,000 newborns worldwide, of which approximately 10% carry the inherited deficiencies of either RPE65 or LRAT.
About Retinitis Pigmentosa (RP) Due to RPE65 and LRAT Mutations
RP is a set of hereditary retinal diseases demonstrating clinical features similar to LCA. RP is also characterized by degeneration of rod and cone photoreceptors, but it presents with a more variable loss of vision in late childhood to adulthood. Deficits in dark adaptation and peripheral vision are particular hallmarks of RP. RP is currently estimated to affect at least 300,000 individuals worldwide, of which approximately 20%–30% are autosomal recessive (arRP). It is currently estimated that less than 3% of autosomal recessive RP patients carry the inherited deficiencies of either RPE65 or LRAT.
About QLT
QLT is a biotechnology company dedicated to the development and commercialization of innovative ocular products that address the unmet medical needs of patients and clinicians worldwide. We are focused on developing our synthetic retinoid program for the treatment of certain inherited retinal diseases, developing our proprietary punctal plug delivery system, as well as U.S. marketing of the commercial product Visudyne® for the treatment of wet age-related macular degeneration.
About Synthetic Retinoid Drugs
Genetic diseases in the eye such as Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP) arise from gene mutations of enzymes or proteins required in the biochemistry of vision. QLT091001 is a replacement for 11-cis-retinal, which is an essential component of the retinoid-rhodopsin cycle and visual function, and is under investigation for the treatment of LCA and RP. QLT091001 has received orphan drug designations for the treatment of LCA and RP by the European Medicines Agency, and for the treatment of LCA and RP due to inherited mutations in the LRAT and RPE65 genes by the U.S. Food and Drug Administration (FDA). The drug has also been granted two Fast Track designations by the FDA for the treatment of the LRAT and RPE65 genetic mutations in both LCA and RP.
About Leber Congenital Amaurosis (LCA)
LCA is an inherited degenerative retinal disease characterized by abnormalities such as roving eye movements and sensitivity to light, and manifesting in severe vision loss from birth. Both rod and cone photoreceptors are affected in LCA. Eye examinations of infants with LCA reveal normal appearing retinas. However, a low level of retinal activity, measured by electroretinography, indicates very little visual function. According to current epidemiological estimates, LCA affects approximately one in 81,000 newborns worldwide, of which approximately 10% carry the inherited deficiencies of either RPE65 or LRAT.
About Retinitis Pigmentosa (RP) Due to RPE65 and LRAT Mutations
RP is a set of hereditary retinal diseases demonstrating clinical features similar to LCA. RP is also characterized by degeneration of rod and cone photoreceptors, but it presents with a more variable loss of vision in late childhood to adulthood. Deficits in dark adaptation and peripheral vision are particular hallmarks of RP. RP is currently estimated to affect at least 300,000 individuals worldwide, of which approximately 20%–30% are autosomal recessive (arRP). It is currently estimated that less than 3% of autosomal recessive RP patients carry the inherited deficiencies of either RPE65 or LRAT.
About QLT
QLT is a biotechnology company dedicated to the development and commercialization of innovative ocular products that address the unmet medical needs of patients and clinicians worldwide. We are focused on developing our synthetic retinoid program for the treatment of certain inherited retinal diseases, developing our proprietary punctal plug delivery system, as well as U.S. marketing of the commercial product Visudyne® for the treatment of wet age-related macular degeneration.
Derived from the press release from QLT Inc.
Monday, November 21, 2011
Of disclosing ‘disability’ before marriage
There was a time when my mother and sister were searching a bride for me. I had insisted to have my visual ‘disability’ (as others call it) be totally disclosed. People initially showed interest and willingness in my proposal, but the moment my Retinitis Pigmentosa-caused blindness was disclosed, either they did not carry the issue further or politely tendered their refusals.
My parents and sister used to feel sorry for this, and they found it difficult to tell me that I again am being refused for the fear that I will get disheartened. But I had not committed a guilty or shameful act due to which I was facing rejections, so I saw no reason to get disheartened. Indeed, the fact that people were interested in me until my blindness was disclosed was a positive point for me. I have not caused my blindness; it is caused due to reasons beyond my control, then why to feel sorry over it? Yet, I must confess that sometimes I used to feel dejected, but there was light at the end of the tunnel.
We had a very old friendship with a family. They used to frequent us often, and once all of a sudden my mother kept my marriage proposal to them. They gladly agreed, the mother of my supposed wife merrily telling us “What is the use of asking? My daughter is yours, we know your son, he is our child, everything is settled.”
Both the families came in the mood to have a great celebration. Marriage celebrations or their preparations seem to be so divine that we feel as if everyone, even our bloodthirsty enemies, are loving and blessing us in their hearts. The noteworthy thing was that we had visited each other so much that it was clear that they know about my blindness. Moreover, my sister too has RP, and they had helped her often. Yet my sight problem was impressed upon them. But they just did not listen to us and even told my sister not to talk about that issue again as if it was hurting them.
Marriage is a big thing. Everything was clear but I felt some uneasiness and requested to talk with the girl because I did not want to take chances.
We went to meet them. It turned out that they were not taking my sight problem seriously because they were thinking that I have enough sight to do my work on my own. For instance, they knew that I work on computers and move around the city and out of the city independently (at the time these talks were taking place I was out of station). But the astonishing thing was that they could not realise that in the course of time I have become blind.
I told her mother point-blank: “I can only see light. I am looking towards your face because of your voice. I use screen reading technology to work on computers. And I use a cane when I walk alone. I cannot see.”
She gave a pause. That pause clearly expressed that she was broken. (Later, I came to know from my mother that her hands were shaking at that time.) Then I talked with the girl who had already known about the new condition I was in. She sounded perplexed and disinclined.
Their reaction, though, was normal. Anyone would have reacted in a similar manner after knowing about my blindness. My marriage date was to be fixed, but now they needed time and told us that they were unaware that I had lost my sight. I thought that the game was over.
But I was wrong.
Days passed. One good evening, the mother of the girl-who-could-be-my-wife came to our home and started showering praises on me. She talked to me in a tearfully sympathetic tone, though I had not needed it. Apologising from her expressions and tone, she told us that her daughter was not willing to marry me. I was thankful because if this condition had disclosed after marriage, I would have been in great trouble.
At my home, I gave a small party to my friends, and called it ‘In the name of my cancelled marriage’! It was meant to truly celebrate life; it was not one of those Bollywood parties in which bottles are uncorked, there is false enjoyment all around, and the main character ineptly tries to forget his grief in the make-believe. Thankfully, we really enjoyed our party, and since my room is quite separated, we made a lot of noise until the early hours of the morning.
Days passed. Wham! The mother of the girl-who-could-be-my-wife told us that her daughter wants to marry me! She was deeply moved by that honesty stuff. Earlier too, that delicate creature had cried and prayed for me a lot, on hearing that I have become blind. Her family members, too, had prayed and cried, and now the girl was willing to marry me. It was a U-turn!
Can you even guess what happened after that? Celebrations, excitement, religious and cultural rituals... no, nothing of the sort.
I was not very impressed with prayers and tears. (Though I always beg for God’s mercy and crave for prayers of His creations.) I had earlier told my sister that they have the right to reject me, but acceptance after rejection will not affect me.
I remained a bachelor.
I started to train myself to lead an unmarried life. I found many people (including two blind men) who were very sufficiently leading a lonesome life and asked myself: “If they can do it, why cannot I?”
Living alone is difficult, but not impossible. Loneliness humbles you, brings forth your good qualities and teaches you how to be happy in need. It is a lovely teacher which urges you to be independent of all except God.
I was not pessimistic to adopt such an approach; I only tried to be practical. Had I been pessimistic, I would have told my family members not to search a bride for me because “I want to live alone.” Besides, I have a small rule of life, which is to try to be happy in an unnatural or adverse situation, but never to willingly prolong or embrace it. True, bearing pain patiently brings forth our good qualities, but this does not mean that we don’t take steps to eliminate it.
I lived and enjoyed the present without caring about the future. I pursued my hobbies (reading, writing and travelling) and tried not to miss a chance to improve myself.
Days passed. The final shot readers! One fine evening I was introduced to a girl by my mother and sister to whom I told each and everything about my sight. It is close to midnight now, and guess what....that girl is with me because thankfully she is my wife!
My parents and sister used to feel sorry for this, and they found it difficult to tell me that I again am being refused for the fear that I will get disheartened. But I had not committed a guilty or shameful act due to which I was facing rejections, so I saw no reason to get disheartened. Indeed, the fact that people were interested in me until my blindness was disclosed was a positive point for me. I have not caused my blindness; it is caused due to reasons beyond my control, then why to feel sorry over it? Yet, I must confess that sometimes I used to feel dejected, but there was light at the end of the tunnel.
We had a very old friendship with a family. They used to frequent us often, and once all of a sudden my mother kept my marriage proposal to them. They gladly agreed, the mother of my supposed wife merrily telling us “What is the use of asking? My daughter is yours, we know your son, he is our child, everything is settled.”
Both the families came in the mood to have a great celebration. Marriage celebrations or their preparations seem to be so divine that we feel as if everyone, even our bloodthirsty enemies, are loving and blessing us in their hearts. The noteworthy thing was that we had visited each other so much that it was clear that they know about my blindness. Moreover, my sister too has RP, and they had helped her often. Yet my sight problem was impressed upon them. But they just did not listen to us and even told my sister not to talk about that issue again as if it was hurting them.
Marriage is a big thing. Everything was clear but I felt some uneasiness and requested to talk with the girl because I did not want to take chances.
We went to meet them. It turned out that they were not taking my sight problem seriously because they were thinking that I have enough sight to do my work on my own. For instance, they knew that I work on computers and move around the city and out of the city independently (at the time these talks were taking place I was out of station). But the astonishing thing was that they could not realise that in the course of time I have become blind.
I told her mother point-blank: “I can only see light. I am looking towards your face because of your voice. I use screen reading technology to work on computers. And I use a cane when I walk alone. I cannot see.”
She gave a pause. That pause clearly expressed that she was broken. (Later, I came to know from my mother that her hands were shaking at that time.) Then I talked with the girl who had already known about the new condition I was in. She sounded perplexed and disinclined.
Their reaction, though, was normal. Anyone would have reacted in a similar manner after knowing about my blindness. My marriage date was to be fixed, but now they needed time and told us that they were unaware that I had lost my sight. I thought that the game was over.
But I was wrong.
Days passed. One good evening, the mother of the girl-who-could-be-my-wife came to our home and started showering praises on me. She talked to me in a tearfully sympathetic tone, though I had not needed it. Apologising from her expressions and tone, she told us that her daughter was not willing to marry me. I was thankful because if this condition had disclosed after marriage, I would have been in great trouble.
At my home, I gave a small party to my friends, and called it ‘In the name of my cancelled marriage’! It was meant to truly celebrate life; it was not one of those Bollywood parties in which bottles are uncorked, there is false enjoyment all around, and the main character ineptly tries to forget his grief in the make-believe. Thankfully, we really enjoyed our party, and since my room is quite separated, we made a lot of noise until the early hours of the morning.
Days passed. Wham! The mother of the girl-who-could-be-my-wife told us that her daughter wants to marry me! She was deeply moved by that honesty stuff. Earlier too, that delicate creature had cried and prayed for me a lot, on hearing that I have become blind. Her family members, too, had prayed and cried, and now the girl was willing to marry me. It was a U-turn!
Can you even guess what happened after that? Celebrations, excitement, religious and cultural rituals... no, nothing of the sort.
I was not very impressed with prayers and tears. (Though I always beg for God’s mercy and crave for prayers of His creations.) I had earlier told my sister that they have the right to reject me, but acceptance after rejection will not affect me.
I remained a bachelor.
I started to train myself to lead an unmarried life. I found many people (including two blind men) who were very sufficiently leading a lonesome life and asked myself: “If they can do it, why cannot I?”
Living alone is difficult, but not impossible. Loneliness humbles you, brings forth your good qualities and teaches you how to be happy in need. It is a lovely teacher which urges you to be independent of all except God.
I was not pessimistic to adopt such an approach; I only tried to be practical. Had I been pessimistic, I would have told my family members not to search a bride for me because “I want to live alone.” Besides, I have a small rule of life, which is to try to be happy in an unnatural or adverse situation, but never to willingly prolong or embrace it. True, bearing pain patiently brings forth our good qualities, but this does not mean that we don’t take steps to eliminate it.
I lived and enjoyed the present without caring about the future. I pursued my hobbies (reading, writing and travelling) and tried not to miss a chance to improve myself.
Days passed. The final shot readers! One fine evening I was introduced to a girl by my mother and sister to whom I told each and everything about my sight. It is close to midnight now, and guess what....that girl is with me because thankfully she is my wife!
-----
Shadab Husain works as a receptionist at Chhatrapati Shahuji Maharaj Medical University, Lucknow. He has an MA in English literature, and has pursued a diploma in computer applications as well as a personality development course. He also writes a blog on personality development and improving English. To visit his blog, click PersonalityAndEnglish.blogspot.com.
Sunday, July 3, 2011
Development of new stem cell treatment for retinal diseases
The company ReNeuron has announced that it has signed a patent and know-how license agreement with Schepens Eye Research Institute, Boston, USA, regarding the Company’s ReN003 stem cell therapy programme focused on diseases of the retina.
An affiliate of Harvard Medical School, Schepens recently announced that it is to join forces with the MassachusettsEye and Ear Infirmary in Boston to create the world’s largest pre-clinical and clinical ophthalmology research centre. ReNeuron has been collaborating with Schepens in the early development of its human retinal precursor cells (hRPCs). Based on the successful results of this initial collaboration, the Company has, through this license agreement, secured the relevant intellectual property rights to develop and commercialise its hRPCs in the field of human retinal stem cell therapeutics. ReNeuron will continue to collaborate closely with lead Investigator Dr Michael Young and his team at Schepens to take the Company’s ReN003 programme through late pre-clinical development and into an initial clinical trial in the US in patients suffering from retinitis pigmentosa, a blindness-causing disease caused by degeneration of the photoreceptor cells in the retina.
Researchers at Schepens have already published data describing the ability of the hRPCs to integrate with host retinal tissue in rodent models of damaged retina and differentiate into the light-sensitive rod cells found in healthy retina. Subsequently, a novel and highly efficient proprietary cell expansion process has recently been optimised which does not involve genetic modification or other similar manipulation of the hRPCs. This expansion technology is currently being employed by ReNeuron to grow and bank clinical-grade hRPCs to the quantities required for future clinical studies.
Subject to regulatory advice and the results of IND-enabling late pre-clinical studies, the ReN003 programme is expected to enter its clinical phase in approximately 18 months. Importantly, although retinitis pigmentosa is the initial target disease, the hRPCs developed in the programme will almost certainly be applicable as cell therapy candidates for other blindness-causing diseases, such as age-related macular degeneration and diabetic retinopathy.
Schepens Eye Research Institute fights blindness by developing new technologies, therapies and knowledge to preserve and restore vision. Through a continuum of discovery, the Institute works toward a future in which blindness is prevented, alleviated, and, ultimately, cured.
Founded in 1950 by famed retinal surgeon Charles L. Schepens, M.D., Schepens Eye Research Institute is the largest independent eye research institute in the United States and an affiliate of Harvard Medical School. Since its inception, the Institute has trained more than 600 postdoctoral fellows in various disciplines of eye research; trained more than 500 eye surgeons who now practice around the world; and published more than 4,600 scientific papers and books about health and eye disease.
About ReNeuron:
ReNeuron is a leading, clinical-stage stem cell business. Its primary objective is the development of novel stem cell therapies targeting areas of significant unmet or poorly met medical need.
ReNeuron has used its unique stem cell technologies to develop cell-based therapies for significant disease conditions where the cells can be readily administered “off-the-shelf” to any eligible patient without the need for additional immunosuppressive drug treatments. ReNeuron’s lead candidate is its ReN001 stem cell therapy for the treatment of patients left disabled by the effects of a stroke. This therapy is currently in clinical development. ReNeuron’s ReN009 stem cell therapy is being developed as a treatment for peripheral arterial disease, a serious and common side-effect of diabetes. The Company is also developing stem cell therapies for other conditions such as blindness-causing diseases of the retina.
Thursday, March 10, 2011
New tools in search of treatments for Retinitis Pigmentosa
Retinitis pigmentosa (RP) is a cluster of genetically determined eye disorders that cause visual defects such as night blindness and narrowing of the field of vision, due to progressive loss of rod photoreceptors. As many as 45 different genes have been linked to the inheritance of this disease, which suggests diversity in etiology and makes development of a standardized animal model problematic. Thus, despite a range of clinical trials of nutritional and drug-based interventions for RP, the disease currently remains untreatable. Better platforms for modeling the disease and testing drug candidates in vitro are urgently needed.
Editor's note: Those who want to learn more about the genetic variations in RP and other diseases, please click here for more information.
New work by Zi-Bing Jin and colleagues in the Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology, Kobe, Japan, looks to add a set of powerful new tools for those searching for treatments for RP. In an article published in PLoS One, the team reports the generation of induced pluripotent stem cells (iPSCs) from patients carrying mutations in several RP-associated genes, and the subsequent differentiation and characterization of rod photoreceptors from these genetically distinct, patient-derived pluripotent cells.
After obtaining informed consent from five RP patients with distinct mutations in the RP1, RP9, PRPH2, or RHO gene, the team took samples of skin cells and used the fibroblasts as a starting point for generating iPSCs. Using the classic reprogramming cocktail of Oct4, Sox2, Klf4, and c-Myc delivered via a retroviral vector, Jin and colleagues generated cell lines from each patient and verified their conversion by tests for appropriate morphology, genetic and karyotypic integrity, and teratoma formation.
Using these iPSCs, the team next generated photoreceptors carrying the genetic signatures of each of the five patient donors using a previously established stepwise protocol that steered the cells over four months in culture from an undifferentiated ES cell-lie state through retinal progenitor, and photoreceptor precursor stages to the desired rod photoreceptor phenotype. The differentiated cells were shown to express the rod photoreceptor marker rhodopsin at high levels, and to have similar electrophysiological function.
Interestingly, rod photoreceptor cells generated from iPSC colonies carrying RP-linked mutations showed a tendency to degenerate, while cone photoreceptors and bipolar cells derived from the same iPSCs were stable. The mechanisms underlying this instability turned out to be dependent on the affected gene. Rod photoreceptors generated from iPSCs with a mutation in the RP9 gene showed evidence of DNA oxidation, while those from iPSCs with a mutation in the rhodopsin gene showed signs of stress on the endoplasmic reticulum, the site of protein synthesis.
As an initial proof-of-concept test of their suite of RP-specific rod photoreceptors in drug validation, Jin and colleagues examined the effects of antioxidant vitamins in preventing degeneration of these cells in vitro. Ascorbic acid, α-tocopherol, and β–carotene have all been tested in clinical trials as anti-oxidant therapies for RP, but all had not proved very effective. When the team tested these on individual cells lines by treating them with one of the three antioxidants for seven days at around the stage at which rod photoreceptor degeneration occurs, they found that α-tocopherol increased cell survival in the lines generated from two patients both carrying mutations in RP9. The same treatment was ineffective in cells from other patients, and ascorbic acid and β–carotene had no effect in any of the lines. These results, which show the efficacy of α-tocopherol in promoting survival in RP9 rod photoreceptors, highlights the potential of patient-derived induced pluripotent stem cells in the study of disease mechanisms and in vitro testing of treatment approaches.
Using iPSCs from cells donated by RP patients with different underlying genetic mutations, the authors were able to show that rod photoreceptors generated from these cells underwent apoptosis in vitro, and showed differing responses in a genetically determined manner to drug treatment. According to them, this is one of the first reports to demonstrate that patient-derived iPSCs may be useful in personalized medicine, as differential responses within a genetically diverse study group will tend to be lost in the crowd. Future improvements in differentiation protocols, screening techniques, cost and efficiency and the establishment of methods for isolating photoreceptors may open up new possibilities for the use of these cells in drug screening.
Reference: PLoS One. 2011 Feb 10;6(2):e17084. Modeling retinal degeneration using patient-specific induced pluripotent stem cells. By Jin ZB, Okamoto S, Osakada F, Homma K, Assawachananont J, Hirami Y, Iwata T, Takahashi M.
Wednesday, January 12, 2011
Breakthrough in possible treatment for dominant form of Retinitis Pigmentosa
Scientists have made a breakthrough in tackling a common form of retinitis pigmentosa (RP), which can eventually lead to blindness.
The breakthrough tackles the rhodopsin gene alteration that causes this inherited form of RP. Rhodopsin-linked RP is variable and at least 150 different alterations in the gene have been identified in RP families worldwide. This makes developing a gene-based therapy very complex, if not impossible, if the treatment targets the specific alteration.
Researchers in the Smurfit Institute of Genetics at the Trinity College of Dublin have been working for 20 years to identify the genes and find a potential treatment for RP. The paper has been published in the Molecular Therapy. The research is funded by Science Foundation Ireland, Fighting Blindness Ireland and the National Neurovision Research Institute, USA.
The rhodopsin-linked form of RP is caused by a mutant form of the rhodopsin gene. The therapy works by switching off both copies of the gene, the normal and the altered copies. Simultaneously, a replacement rhodopsin gene is introduced which has been subtly altered so it cannot be suppressed. It encodes normal protein, which allows the photoreceptors to work normally. The research restored visual function in mice with a dominant rhodopsin-linked form of RP exactly replicating the form of the disease, which affects humans.
The scientists hope that this basic research will move into more preclinical work, including a larger mammal, and eventually move on to human clinical trials.
The scientists believe that the research’s implications stretched far beyond RP, and may be applicable to a lot of dominant diseases. The key to finding a cure is to suppress the mutant gene causing the problems. Since this is the dominant gene, it is harder to treat, and hence the success of this research is important to treat those dominant diseases where the mutant protein drives the disease process.
Physician scientists and researchers can access the article here.
Saturday, November 20, 2010
Ocuseva, a drug in clinical trial in Japan for treatment of Retinitis Pigmentosa
R-Tech Ueno Ltd. (Tokyo, Japan) announced it has completed a phase 2 clinical trial of 0.15% UF-021 isopropyl unoprostone (Ocuseva), which is under development as a treatment for retinitis pigmentosa (RP). The trial investigated the possibility of improving visual function in the central part of the retina with UF-021 in patients with RP.
The randomized, multicenter, comparative study examined 112 patients with RP that had progressed to the mid-to late-stage, defined as a visual acuity of less than 6/18 with a narrow visual field. Patients received placebo or Ocuseva, instilled one drop per time or two drops per time (at a 5 minute interval), twice a day in the morning and evening for 24 weeks. The primary efficacy endpoint was the change seen on the visual field analysis as checked by an instrument called the MP-1 microperimeter (Nidek, Gamagori, Japan), Retinal sensitivity was also studies by a regular Humphrey visual field analyzer (10-2), visual acuity, contrast sensitivity, and health-related quality of life, using a questionnaire on visual function (VFQ-25).
After 24 weeks, positive change in the retinal sensitivity of the central 2 degree field of vision from baseline increased the most in the two-drops-per-time group, followed by the one drop- per-time group. The placebo group had the least significant increase in sensitivity.
The change in the retinal sensitivity from the pretreatment level by 4 dB or more was seen as improvement in 15.2% of patients in the placebo group, 7.9% in the one-drop-per-time group, and 18.4% in the two-drops-per-time group, whereas the change was seen as aggravation in 21.2% in the placebo group, 15.8% in the one-drop-per-time group, and 2.6% in the two-drops-per-time group. There were a significantly lower number of aggravated cases in the 2-drops-per-time group, compared with the placebo group. The retinal sensitivity measured with the Humphrey perimeter showed statistically significant improvement at weeks 4 and 8 in the two-drops-per-time group compared with the placebo group.
The main adverse effect of Ocuseva was ocular irritation, which, the company said, disappears several minutes after instillation.
The results show promise, specifically with the advantage that it only requires instillation of drops, rather than a completed surgical intervention. Ocular irritation, which anyway lasts a few minutes, is the only adverse effect. The follow-up in the trial is 6 months (24 weeks), which is considered good enough to consider in any retinal trial. The only thing difficult to understand is the group that was instilled with placebo drops (meaning no medication) still showed improvement in about 17 of the 112 patients in the trial (15.2%).
After 24 weeks, positive change in the retinal sensitivity of the central 2 degree field of vision from baseline increased the most in the two-drops-per-time group, followed by the one drop- per-time group. The placebo group had the least significant increase in sensitivity.
The change in the retinal sensitivity from the pretreatment level by 4 dB or more was seen as improvement in 15.2% of patients in the placebo group, 7.9% in the one-drop-per-time group, and 18.4% in the two-drops-per-time group, whereas the change was seen as aggravation in 21.2% in the placebo group, 15.8% in the one-drop-per-time group, and 2.6% in the two-drops-per-time group. There were a significantly lower number of aggravated cases in the 2-drops-per-time group, compared with the placebo group. The retinal sensitivity measured with the Humphrey perimeter showed statistically significant improvement at weeks 4 and 8 in the two-drops-per-time group compared with the placebo group.
The main adverse effect of Ocuseva was ocular irritation, which, the company said, disappears several minutes after instillation.
The results show promise, specifically with the advantage that it only requires instillation of drops, rather than a completed surgical intervention. Ocular irritation, which anyway lasts a few minutes, is the only adverse effect. The follow-up in the trial is 6 months (24 weeks), which is considered good enough to consider in any retinal trial. The only thing difficult to understand is the group that was instilled with placebo drops (meaning no medication) still showed improvement in about 17 of the 112 patients in the trial (15.2%).
Welcome to Retina India
Retina India is a not-for-profit organization, registered with the Charity Commissioner, Mumbai, India, established for empowering people with retinal disorders, and bringing them and their families on a common platform with physicians, researchers, counselors, low vision and mobility experts and other specialists.
Why do we need another not-for-profit organization?
India is home to approximately 24 million blind people, the largest in the world. Additionally, there are another 52 million visually impaired in the country. It is thought that if this trend is allowed to continue, the number of blind people would increase to 31.6 million by 2020.
The blindness-prevention programs that are sponsored by governmental agencies and by non governmental organizations (NGOs) usually focus on "avoidable" or "preventable" blindness that commonly includes cataract and corneal problems. Even though patients with preventable blindness in India are significant, the prevalence of retinal ailments, such as retinitis pigmentosa and allied disorders, macular degeneration, diabetic retinopathy, etc. is gradually increasing. Some of these diseases do not even have a cure at this time, and usually leave the affected people with permanent visual impairment for a lifetime. There has been no singular effort in India to unite the efforts in the fields of medical research, education, rehabilitation and welfare of people with retinal disorders.
It is this void that Retina India aims to fill.
Retina India is focused on spreading awareness amongst society, the NGOs and the Governmental agencies about people with retinal ailments and the specific issues they and their families face. We also wish to help them make a difference to their own lives, and to the lives of people around them.
Our Vision
To empower patients and families of patients with retinal ailments, and help them make a significant contribution to their own lives, and to the lives of people around them.
Our Mission
To increase awareness of retinal diseases and champion the cause of people who get affected by them, and to induce increased research efforts towards treatment for such diseases.
Our people
Simply said, Retina India is a movement, It is a movement that includes all of you. It is our strong belief that when people come together, and work towards a common cause, a lot can get done.
Retina India includes patients with retinal disorders and their families. It also includes retinal specialists and other ophthalmologists with an interest in retinal diseases, low vision experts, mobility experts, counselors, and others. We invite people with a social spirit, who have an inherent desire to do something good for others, and make a difference in someone's life, to volunteer and be a part of this movement. We also invite young adults, school and college students, to gain experience in working on a project for Retina India .
Our Key Objectives:
Patient Alliance: The alliance brings together patients with retinal disorders, along with their families and friends, to work for mutual benefit.
Medical Research and Treatments: Retina India highlights, coordinates and sponsors research in retinal treatment in India, while also informing the patients and their families about the current on-going research in India and around the world.
Clinical trials in India: We act as a channel to bring new treatments and technologies to India (Gene Therapy, Artificial Retina, Stem-Cell treatment, etc.) for Indian patients with retinal disorders.
Patient Registry: Retina India maintains databases (or registries) of patients with specific retinal diseases. Such registries will help inform patients likely to benefit from new treatments, such that they are not left to wonder whether a new treatment is beneficial to them or not, and in the process, spend a lot of time, effort and money in finding that out. Registries for Retinitis Pigmentosa, Macular Degeneration, Retinopathy of Prematurity, Leber's Congenital Amaurosis, etc. are already functional.
Education, Counseling & Advocacy: Activities range from encouraging beneficiaries to pursue their lives productively, counseling them about education, employment, marriage and family issues, to rehabilitation, independence training and mobility skills via associations with other organizations in the country. We are also commited to advocacy about the concerns of people with retinal ailments.
We welcome you to make a difference in your own life, and in the lives of people around you.
Why do we need another not-for-profit organization?
India is home to approximately 24 million blind people, the largest in the world. Additionally, there are another 52 million visually impaired in the country. It is thought that if this trend is allowed to continue, the number of blind people would increase to 31.6 million by 2020.
The blindness-prevention programs that are sponsored by governmental agencies and by non governmental organizations (NGOs) usually focus on "avoidable" or "preventable" blindness that commonly includes cataract and corneal problems. Even though patients with preventable blindness in India are significant, the prevalence of retinal ailments, such as retinitis pigmentosa and allied disorders, macular degeneration, diabetic retinopathy, etc. is gradually increasing. Some of these diseases do not even have a cure at this time, and usually leave the affected people with permanent visual impairment for a lifetime. There has been no singular effort in India to unite the efforts in the fields of medical research, education, rehabilitation and welfare of people with retinal disorders.
It is this void that Retina India aims to fill.
Retina India is focused on spreading awareness amongst society, the NGOs and the Governmental agencies about people with retinal ailments and the specific issues they and their families face. We also wish to help them make a difference to their own lives, and to the lives of people around them.
Our Vision
To empower patients and families of patients with retinal ailments, and help them make a significant contribution to their own lives, and to the lives of people around them.
Our Mission
To increase awareness of retinal diseases and champion the cause of people who get affected by them, and to induce increased research efforts towards treatment for such diseases.
Our people
Simply said, Retina India is a movement, It is a movement that includes all of you. It is our strong belief that when people come together, and work towards a common cause, a lot can get done.
Retina India includes patients with retinal disorders and their families. It also includes retinal specialists and other ophthalmologists with an interest in retinal diseases, low vision experts, mobility experts, counselors, and others. We invite people with a social spirit, who have an inherent desire to do something good for others, and make a difference in someone's life, to volunteer and be a part of this movement. We also invite young adults, school and college students, to gain experience in working on a project for Retina India .
Our Key Objectives:
Patient Alliance: The alliance brings together patients with retinal disorders, along with their families and friends, to work for mutual benefit.
Medical Research and Treatments: Retina India highlights, coordinates and sponsors research in retinal treatment in India, while also informing the patients and their families about the current on-going research in India and around the world.
Clinical trials in India: We act as a channel to bring new treatments and technologies to India (Gene Therapy, Artificial Retina, Stem-Cell treatment, etc.) for Indian patients with retinal disorders.
Patient Registry: Retina India maintains databases (or registries) of patients with specific retinal diseases. Such registries will help inform patients likely to benefit from new treatments, such that they are not left to wonder whether a new treatment is beneficial to them or not, and in the process, spend a lot of time, effort and money in finding that out. Registries for Retinitis Pigmentosa, Macular Degeneration, Retinopathy of Prematurity, Leber's Congenital Amaurosis, etc. are already functional.
Education, Counseling & Advocacy: Activities range from encouraging beneficiaries to pursue their lives productively, counseling them about education, employment, marriage and family issues, to rehabilitation, independence training and mobility skills via associations with other organizations in the country. We are also commited to advocacy about the concerns of people with retinal ailments.
We welcome you to make a difference in your own life, and in the lives of people around you.
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