Implanted neurons fuse with pre-existing brain wiring in the lab

Among the many hurdles to be cleared before human embryonic stem cells can achieve their therapeutic potential is determining whether or not transplanted cells can functionally integrate into target organs or tissues.

Writing in the Proceedings of the National Academy of Sciences (PNAS) , a team of Wisconsin scientists reports, in a study funded by the US National Institutes of Health, that neurons, forged in the lab from blank slate human embryonic stem cells and implanted into the brains of mice, can successfully fuse with the brain's wiring, and both send and receive signals.

Neurons are specialized, impulse conducting cells that are the most elementary functional unit of the central nervous system. The 100 billion or so neurons in the human brain are constantly sending and receiving the signals that govern everything from walking and talking to thinking. The work represents a crucial step toward deploying customized cells to repair damaged or diseased brains, the most complex human organ.

"The big question was can these cells integrate in a functional way," says Jason P. Weick, the lead author of the new study and a staff scientist at the University of Wisconsin-Madison's Waisman Center. "We show for the first time that these transplanted cells can both listen and talk to surrounding neurons of the adult brain."

The Wisconsin team tested the ability of their lab grown neurons to integrate into the brain's circuitry by transplanting the cells into the adult mouse hippocampus, a well-studied region of the brain that plays a key role in processing memory and spatial navigation. The capacity of the cells to integrate was observed in live tissue taken from the animals that received the cell transplants.

Weick and colleagues also reported that the human neurons adopted the rhythmic firing behavior of many brain cells talking to one another in unison. And, perhaps more importantly, that the human cells could modify the way the neural network behaved.

A critical tool that allowed the UW group to answer this question was a new technology known as optogenetics, where light, instead of electric current, is used to stimulate the activity of the neurons.

"Previously, we've been limited in how efficiently we could stimulate transplanted cells. Now we have a tool that allows us to specifically stimulate only the transplanted human cells, and lots of them at once in a non-invasive way," says Weick.

Weick explains that the capacity to modulate the implanted cells was a necessary step in determining the function of implanted cells because previous technologies were too imprecise and unreliable to accurately determine what transplanted neurons were doing.

Embryonic stem cells, and the closely related induced pluripotent stem cells can give rise to all of the 220 types of tissues in the human body, and have been directed in the lab to become many types of cells, including brain cells.

The appeal of human embryonic stem cells and induced pluripotent cells is the potential to manufacture limitless supplies of healthy, specialized cells to replace diseased or damaged cells. Brain disorders such as Parkinson's disease and amyotrophic lateral sclerosis, more widely known as Lou Gehrig's disease, are conditions that scientists think may be alleviated by using healthy lab grown cells to replace faulty ones. Multiple studies over the past decade have shown that both embryonic stem cells and induced cells can alleviate deficits of these disorders in animal models.

The new study opens the door to the potential for clinicians to deploy light-based stimulation technology to manipulate transplanted tissue and cells. "The marriage between stem cells and optogenetics has the potential to assist in the treatment of a number of debilitating neurodegenerative disorders," notes Su-Chun Zhang, a UW-Madison professor of neuroscience and an author of the new PNAS report. "You can imagine that if the transplanted cells don't behave as they should, you could use this system to modulate them using light."

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Editorial note: Outcome from this research will have a major role to play in retinal diseases, since retina is considered to be a part of the brain, and most stem cell treatments have not been successful due to issues with connectivity of the newly formed retinal cells derived from stem cells, with the functional ones that preexist in the retina. 

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!

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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.

FDA approves Eylea for patients with Age-related Macular Degeneration

Regeneron Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has approved Eylea (aflibercept) Injection, known in the scientific literature as VEGF Trap-Eye, for the treatment of patients with neovascular (wet) Age-related Macular Degeneration (AMD) at a recommended dose of 2 milligrams (mg) every four weeks (monthly) for the first 12 weeks, followed by 2 mg every eight weeks (2 months).

The approval of Eylea was granted under a Priority Review, a designation that is given to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.  This approval was based upon the results of two Phase 3 clinical studies.  In these studies, Eylea dosed every eight weeks, following three initial monthly injections, was clinically equivalent to the standard of care, Lucentis® (ranibizumab injection) dosed every four weeks, as measured by the primary endpoint of maintenance of visual acuity (less than 15 letters of vision loss on an eye chart) over 52 weeks.  The most common adverse reactions (frequency of 5% or more) reported in patients receiving Eylea were conjunctival hemorrhage, eye pain, cataract, vitreous detachment, vitreous floaters, and increased intraocular pressure.  The adverse event profile was similar to that seen with ranibizumab.

As per the experts, Eylea offers the potential of achieving the efficacy that the ophthalmic world had come to expect from the current anti-VEGF agents, but with less frequent injections and no monitoring requirements. This, as per these experts, may reduce the need for costly and time-consuming monthly office visits for patients and their caregivers.

About Eylea™ (aflibercept) Injection:

Vascular Endothelial Growth Factor (VEGF) is a naturally occurring protein in the body.  Its normal role in a healthy organism is to trigger formation of new blood vessels (angiogenesis) supporting the growth of the body's tissues and organs.  However, in certain diseases, such as wet age-related macular degeneration, it is also associated with the growth of abnormal new blood vessels in the eye, which exhibit abnormal increased permeability that leads to edema. Scarring and loss of fine-resolution central vision often results.  

Eylea, known in the scientific literature as VEGF Trap-Eye, is a recombinant fusion protein, consisting of portions of human VEGF receptors 1 and 2 extracellular domains fused to the Fc portion of human IgG1 and formulated as an iso-osmotic solution for intravitreal administration.  Eylea acts as a soluble decoy receptor that binds VEGF-A and placental growth factor (PlGF) and thereby can inhibit the binding and activation of these cognate VEGF receptors.

Eylea is indicated for the treatment of patients with neovascular age-related macular degeneration (wet AMD).  Eylea is contraindicated in patients with ocular or periocular infections, active intraocular inflammation, or known hypersensitivity to aflibercept or to any of the excipients in EYLEA.

The recommended dose for Eylea is 2 mg administered by intravitreal injection every four weeks (monthly) for the first 12 weeks (3 months), followed by 2 mg once every eight weeks (2 months).  Although Eylea may be dosed as frequently as 2 mg every four weeks (monthly), additional efficacy was not demonstrated when Eylea was dosed every four weeks compared to every eight weeks.

There is a potential risk of arterial thromboembolic events (ATEs) following use of intravitreal VEGF inhibitors, including Eylea, defined as nonfatal stroke, nonfatal myocardial infarction, or vascular death (including deaths of unknown cause).  The incidence of ATEs with Eylea in clinical trials was low (1.8%).

Serious adverse reactions related to the injection procedure have occurred in less than 0.1% of intravitreal injections with Eylea and include endophthalmitis, traumatic cataract, and increased intraocular pressure.

About the VIEW 1 and VIEW 2 Clinical Studies:

The safety and efficacy of Eylea were assessed in two randomized, multi-center, double-masked, active-controlled studies in patients with wet AMD.  A total of 2412 patients were treated and evaluable for efficacy (1817 with Eylea) in the two studies (VIEW 1 and VIEW 2). In each study, patients were randomly assigned in a 1:1:1:1 ratio to one of four dosing regimens: 1) Eylea administered 2 mg every eight weeks following three initial monthly doses Eylea 2Q8); 2) Eylea administered 2 mg every four weeks Eylea 2Q4); 3) Eylea 0.5 mg administered every four weeks Eylea 0.5Q4); and 4) ranibizumab administered 0.5 mg every four weeks (ranibizumab 0.5Q4).  Patient ages ranged from 49 to 99 years with a mean of 76 years.

In both studies, the primary efficacy endpoint was the proportion of patients who maintained vision, defined as losing fewer than 15 letters of visual acuity at week 52 compared to baseline.  Data are available through week 52.  Both the Eylea™ (aflibercept) Injection 2Q8 and 2Q4 dosing groups were shown to have efficacy that was clinically equivalent to the ranibizumab 0.5Q4 group for the primary endpoint.

Select results of the VIEW 1 and VIEW 2 studies as described in the full Prescribing Information for the Eylea 2 mg every four weeks and Eylea 2 mg every eight weeks dosing groups as compared to ranibizumab dosed monthly group are shown below.

To check the efficacy outcomes at week 52 in VIEW 1 and VIEW 2 Studies, please click on the source below. 

Safety of Eylea:

Eylea™ (aflibercept) Injection is contraindicated in patients with ocular or periocular infections, active intraocular inflammation, or known hypersensitivity to aflibercept or to any of the excipients in Eylea.

Intravitreal injections, including those with Eylea, have been associated with endophthalmitis and retinal detachments.  Proper aseptic injection technique must always be used when administering EYLEA.  Patients should be instructed to report any symptoms suggestive of endophthalmitis or retinal detachment without delay and should be managed appropriately. Acute increases in intraocular pressure have been seen within 60 minutes of intravitreal injection, including with Eylea.  Sustained increases in intraocular pressure have also been reported after repeated intravitreal dosing with VEGF inhibitors.  Intraocular pressure and the perfusion of the optic nerve head should be monitored and managed appropriately. There is a potential risk of arterial thromboembolic events (ATEs) following use of intravitreal VEGF inhibitors, including Eylea, defined as nonfatal stroke, nonfatal myocardial infarction, or vascular death (including deaths of unknown cause).  The incidence of ATEs with Eylea in clinical trials was low (1.8%). Serious adverse reactions related to the injection procedure have occurred in less than 0.1% of intravitreal injections with Eylea including endophthalmitis, traumatic cataract, and increased intraocular pressure. The most common adverse reactions (greater than or equal to 5%) reported in patients receiving Eylea were conjunctival hemorrhage, eye pain, cataract, vitreous detachment, vitreous floaters, and increased intraocular pressure.

To see the full prescribing Information for Eylea, please click here.

Regeneron is collaborating with Bayer HealthCare on the global development of Eylea.  Bayer submitted an application for marketing authorization in Europe for wet AMD in June 2011.

Bayer HealthCare will market Eylea outside the United States, where the companies will share equally the profits from any future sales of Eylea.  Regeneron maintains exclusive rights to Eylea in the United States.

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First human induced Pluripotent Sem cell therapy eyed in 2013

A clinical study into the use of lab-grown retina cells to treat age-related macular degeneration (AMD) has been slated for fiscal 2013, a senior staffer of the research body planning to undertake the project said Saturday.

The project might be the world's first to use induced pluripotent stem cells, or iPS cells, for the treatment of human diseases. 

The study will initially target several patients with the eye disease whose vision cannot be sufficiently restored through existing medication. It will then be expanded to include earlier-stage patients once the safety of the iPS cell treatment can be determined.

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Scientists find an answer to one of still unsolved mysteries of the eye

Scientists have a good overall understanding of human vision: when light enters our eyes, it is focused by the lens and strikes the retina in the back of the eye. The light causes some of the millions of photoreceptor cells that line the retina to undergo a chemical change, which send a message through the optic nerve to the brain, which ultimately creates an image. However, there are still a few unresolved questions in the details of the vision process, one of which is why the eye evolved to use a certain light-absorbing chromophore called 11-cis-retinal, instead of one of its isomers (i.e., molecules with the same atoms but in different arrangements), such as 7-cis, 9-cis, or 13-cis.

Chemists Sivakumar Sekharan from Emory University in Atlanta, Georgia, and Keiji Morokuma from Emory University and Kyoto University in Kyoto, Japan, describe the eye’s use of 11-cis-retinal as “one of the basic and unresolved puzzles in the chemistry of vision.” But by taking advantage of the rapid advances in hybrid quantum mechanics/molecular mechanics (QM/MM) computational modeling, the researchers have found that the answer to this puzzle lies in electrostatic interactions in the retina. Their study is published in a recent issue of the Journal of the American Chemical Society.

The retina contains light-sensitive photoreceptor cells known as rods and cones, which convert incoming light into electrical impulses that are sent to the brain. On the top of every rod and cone is a region that contains opsin proteins bound to 11-cis-retinal chromophores, which together are called rhodopsin. When light strikes the retina, the 11-cis-retinal chromophores absorb the light, which causes them to undergo an isomerization and change their molecular configuration from 11-cis-retinal to all-trans-retinal in a matter of picoseconds. The difference between these two isomers involves the positions of the hydrogen atoms, a shape change that causes the opsin protein to change shape in response. The opsin shape change, in turn, leads to a cascade of biochemical reactions in the photoreceptor cell that ultimately generate an electrical impulse.

Since the 11-cis-retinal is the retina’s first responder to incoming light, its unique geometric configuration clearly plays an important role in the vision process. However, theoretically there are a handful of other retinal isomers that seem capable of performing this task, yet for some reason photoreceptor cells only function with 11-cis-retinal (and the corresponding 11-cis-rhodopsin).

“Because the primary event in vision involves no breaking of chemical bonds but only a conformational change in the shape of the molecule from bent cis to the distorted all-trans form, scientists wondered why 7-cis-, 9-cis- or 13-cis- isomers could not achieve this goal,” Sekharan told PhysOrg.com.

To answer this question, the researchers built computational models of the rhodopsin found in the eyes of cows, monkeys, and squids. While all known animals’ eyes use 11-cis-retinal, the opsin in different animals contains different numbers and positions of amino acids. Using a cutting-edge QM/MM modeling method called ONIOM (Our own N-layered Integrated Molecular Orbital), the researchers prepared models that matched different animals’ opsins with 7-cis, 9-cis, 11-cis, and 13-cis molecules serving as chromophores. In these artificial rhodopsins, the researchers analyzed the structure, stability, energetics, and spectroscopy to try to find out what makes 11-cis-retinal nature’s preferred isomer.

The results of the modeling showed that differences in the electrostatic interactions between the opsin protein and the retinal chromophore played the biggest factor in the natural selection of 11-cis-retinal over the other cis isomers. Due to electric charges, the link between 11-cis-retinal and opsin has a higher stability than the links between other cis isomers and opsin, making it the most favorable choice.

“Our results show that the strong electrostatic interaction between retinal and opsin favors the natural selection of 11-cis- over other cis-isomers and arguably prepares the chromophore for the upcoming photochemical event,” Sekharan said. “This indeed is very surprising given the fact that, outside the protein environment, 11-cis-retinal is one of the least stable isomers. Apparently, our results on cow, monkey and squid demonstrate that organisms everywhere may tend to gravitate towards common selection.”

Sekharan added that the results not only provide a better understanding of the eyes on a molecular level, but could also have applications for artificial retinas.

“Because rhodopsin serves as a decisive crossing point between an organism and its environment, we have been always impressed with this interesting interface by seeing it, say, from the outside and not from the inside,” he said. “Using the ONIOM-QM/MM method we developed, we can ‘enter’ deep into the dark side of this fascinating molecule. One of interesting findings to emerge out of our investigation is that 9-cis-retinal is only slightly higher in energy compared to 11-cis-retinal. This provides strong evidence for the presence of 9-cis-rhodopsin in nature, which in turn may well aid in optimizing the parameters required for designing artificial retinas.”

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FDA Requests More Trials of Retinal Disease Treatment

Alimera Sciences, Inc., a biopharmaceutical company that specializes in the research, development and commercialization of prescription ophthalmic pharmaceuticals, today announced that it has received a complete response letter (CRL) from the U.S. Food and Drug Administration (FDA) in response to the New Drug Application (NDA) for ILUVIEN® for the treatment of diabetic macular edema (DME) associated with diabetic retinopathy.

A CRL is issued by the FDA's Center for Drug Evaluation and Research when their review of an application is completed and questions remain that precludes the approval of the NDA in its current form.

Alimera is seeking approval for Iluvien as a treatment for diabetic macular edema, a condition that can cause blurred vision and blindness.

The FDA stated that it was unable to approve ILUVIEN because there was no provide sufficient data to support that ILUVIEN is safe and effective in the treatment of patients with DME. The FDA stated that the risks of adverse reactions shown for ILUVIEN in the FAME® Study were significant and were not offset by the benefits demonstrated by ILUVIEN in these clinical trials. The FDA has indicated that Alimera will need to conduct two additional clinical trials to demonstrate that the product is safe and effective for the proposed indication.

The company officials will request a meeting with the FDA to clarify its next steps. 

ILUVIEN is Alimera's investigational, sustained drug delivery system that releases sub-microgram levels of fluocinolone acetonide (FAc) for the treatment of DME.

Alimera initially had asked the FDA to approve Iluvien in June 2010. In December, the FDA asked the company to report data from a third year of a clinical trial, and Alimera filed that data in May 2011. It also responded to the agency's concerns about manufacturing, packaging and sterilization of the drug. 

In December 2010, the FDA issued a CRL to Alimera related to its June 2010 NDA for ILUVIEN, which included data through month 24 of the FAME™ Study.

In that first CRL, the FDA asked for, among other things, analyses of the safety and efficacy data through month 36 of the FAME Study. Alimera submitted a response to the FDA on May 12, 2011, addressing the issues raised in the first CRL and including 36-month trial data. The FDA classified Alimera's response as a Class 2 resubmission, resulting in a six-month review period and a Prescription Drug User Fee Act, or PDUFA, date of November 12, 2011.

For Europe, Alimera expects to submit its formal response to the Preliminary Assessment Report to the Medicines and Healthcare products Regulatory Agency (MHRA) later this month. Based on this submission, the MHRA is expected to make a recommendation on the approvability of ILUVIEN to Alimera and the Concerned Member States (Austria, France, Germany, Italy, Portugal and Spain) by the end of this year, with a decision regarding the approval of ILUVIEN expected in the first half of 2012. The market opportunity in Europe is similar in size to the U.S. market opportunity.

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Success in clinical trial brings researchers closer to cure for blindness

Researchers at Hadassah Hospital in Israel, led by Dr. Eyal Banin, have completed a clinical trial that tested the use of gene therapy to restore sight to patients suffering from Leber's Congenital Amaurosis (LCA). Dana and Yossi, two participants in this study, suffer from Leber's Congenital Amaurosis (LCA), the most severe form of all inherited retinal dystrophies causing congenital blindness. Like others affected, they have experienced severe visual impairment since birth. LCA sufferers experience poor night vision, low visual acuity and a constricted visual field. This low vision continues to deteriorate, leading to total blindness. Other symptoms may include crossed eyes, roving eye movements, unusual sensitivity to light, and/or cataracts. LCA is usually inherited as an autosomal recessive genetic condition. Those with LCA suffer in darkness, without sight and without hope. Until now.

Dr. Eyal Banin, MD, Ph.D., at the Center for Retinal and Macular Degeneration at Hadassah University Medical Center, in collaboration with leading researchers in the United States and Great Britain, performed a clinical trial that has successfully demonstrated the efficacy of gene therapy in the treatment of LCA.  LCA is caused by a mutation in the RPE65 gene.  In this clinical trial, a normal RPE65 gene was injected into the retina to replace the damaged gene and renew protein production. Participants Dana and Yossi were treated with this gene therapy in just part of the retina of one eye, with dramatic results. Shortly after treatment, both participants noted a substantial improvement in their vision.

When asked about the results of this treatment, Yossi said, "I felt the real change, the real revolution, after 21 days. It was amazing because today I see things that I have never seen before. I'm very proud to be a part of this research." Dana said, "Learning of new treatment was a life-changing event. I'm experiencing a real change. I was surprised to see real improvement in my vision."

Yossi and Dana's self-reporting of visual improvement is corroborated by objective, quantitative measurements of the treated area that also show significant improvement. With the continuation of this research, these scientists will be able to develop gene therapy to treat additional retinal degeneration diseases and make it possible to treat many more patients.

To watch a just-released video with more information about this clinical trial, its researchers and study participants, please visit http://www.mvrf.org/news.php.

Dr. Banin says: "You cannot imagine what an effect this has had not only on the treated patients, their families and on us, but also on the wider population of patients with retinal and macular degenerations here in Israel, who suddenly feel some glimmer of hope.."

Keith A. Lampman, Executive Director of MVRF, says, "We are extremely excited about the results of this study and feel confident that, in close collaboration with our partners across the globe, we are closer than ever to a cure for retinal diseases."

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