For those of you who missed this story, Steve Fialkoff, a 68-year-old New Yorker with the Usher 2A RP mutation, stumbled on a combo of curcumin, black pepper and ginger last February that started giving him back bits of eyesight.

Determined to spread the word about his discovery, the video-editor-turned-playwright who should also consider a career as a comedian, reported his vision gains to a retinal specialist, who immediately dismissed his curcumin, black pepper & ginger claims. Undeterred by her lack of enthusiasm, he shared his news with Foundation Fighting Blindness, and was told that extensive research would be required to determined if his experience with curcumin and ginger was more than a fluke. So Steve took what he saw as the only logical next step: launching his own, informal Curcumin RP study – which he named I SPICE (Improving Sight Project Investigating Curcumin Experiment).

Steve’s ever-growing I SPICE group currently consists of 56 RP participants who are all experimenting with different combos of curcumin, black pepper and ginger. And the first survey results – tallied over Thanksgiving weekend – indicate that curcumin with ginger does indeed improve RP eyesight.

Of the thirty participants who took part in the survey, fifteen reported vision improvements. Some report expanded visual fields. Others have seen a jump in visual acuity and color. A couple have even received a boost in their night vision. Not everyone has experienced vision gains. My curcumin experiment has so far only yielded occasional, inconsistent increases in visual acuity. But I am excited about the results because they span several RP mutations — something that has now caught the attention of FFB. According to Steve, the foundation is now considering a curcumin RP study. In the meantime, Steve is tracking a curcumin macular degeneration study being conducted by the University of Illinois. Based on the positive results of a research study utilizing the amino acid N-Acetyl Cysteine (NAC) for RP treatment, some of us I SPICE participants are also starting to add it into the experiment mix.

It’s been so empowering to be on this journey with Steve and the other RP participants and if you are interested, we would love to have you join. The more data we can collect and information we can share – the closer we all are to seeing our world. To join the I SPICE experiment, email Steve at: tinymustacheprods@gmail.com.

And to get a sense for Steve and the amazing work he is doing on our behalf, check out his December 8 e-bulletin, which I’ve pasted below.

I SPICE NEWSLETTER #12

Dec. 8, 2022

Improving Sight Project 

Investigating Curcumin Experiment

Topics:

1) New reports

2) FFB on the case

3) New experimenters

1) New Reports:

A recent Curcumin experimenter with 99% vision loss with a recessive FAM gene type reports her latest doctor eye exam from Wills eye institute.

Her last check was two years ago.

She saw the first two lines of the Snellen chart. Big E, and the next line. She was told she had 20-400 vision. Also, she scored correctly on identifying black and white squares with her bad eye. Ok, so? It doesn’t sound so great. Well, she didn’t have any vision to measure two years ago. What was the eye doctor’s response to this test finding?:

“You must have had a bad day two years ago. I doubt Curcumin is responsible for these test results…”

To quote my own dialogue line from a play I wrote:

“Doctors Schmocktors!”

The same woman reports visiting her friend and seeing her new bangs on her forehead when she turned her head into he sunbeam coming in from the window.

What I love about this report is the modesty and uncertainty about her own improvement.

“Maybe it’s my imagination and I could see this stuff all along but just now hyper aware of any object in view” She told me over the phone.

Ok, let’s give it another month and see what happens.

She is also starting to take N-Acetyl Cysteine.

Which leads us to a second report from Me.

I haven’t noticed any big improvements since October, ’22.

But after having, coincidentally, blended ginger strips in my lentil soup, my left eye seems to be seeing more, enabling it to lock with my right eye for longer periods of time especially in daylight when there is more light out there. Less double vision means a wider tunnel to view the world through. I also take 1 gram of N-Acetyl Cysteine a day along with the Curcumin. (I don’t know if NAC increases anti-coagulation but I will check on that.) The NAC is about a month into my regimen.

Ok, it’s only been 3 days of this increased field but it is strange that it happened after eating ginger in lentil soup meal.

(yes, there was black pepper, garlic, olive oil, butter, salt, shallots in the flavorings well)

Was it good? 

Eh, it was very hearty.

Does it make a lot of gas?

NAC + lentils = gas

NAC + anything= gas

2) FFB on the case:

The word from the FFB is encouraging. Since twenty more people could have potentially reported but didn’t… our data sample was relatively tiny.

Very hard to spot a pattern or trend in such little data.

But, I believe the data shows that Curcumin doesn’t care which gene you have mutated. Recessive forms may respond better. USH2A seems to respond favorably. (I have that one, which is supposed to be the most common form of RP)

Too early to tell is the word but I believe that Autosomal dominant P23 H rhodopsin type isn’t the only RP to benefit from a possible Curcumin therapy.

All in all the fact that 15 out of 30 reports were positive improvement, is pretty compelling info.

Additionally,

I am awaiting safety and efficacy results from Univ. of Illinois at Chicago trial of curcumin on Macular degeneration.

I believe, if no adverse effects are found, then creating a human trial with curcumin and RP will be a no brainer.

We shall see.

3) New experimenters:

There are two new experimenters as of last week. The word is spreading albeit, slowly. Both of them have Ushers with hearing loss and RP.

Will Curcumin help hearing and seeing? Perhaps we can help two verbs with one pill?

Oh, that was terrible, steve!

Anyway, I hope to be hearing from people who just started at the end of October.

Keep in touch.

Avoid the flu.

Curcumin is derived mostly from turmeric root a member of the ginger family, which includes Black pepper. 

I use fresh ground black peppercorns, fresh sliced and peeled ginger root cooked in olive oil or butter or ghee or coconut oil.

My calculations show 1/5 of a teaspoon of ground black pepper supposedly contains enough Piperine to activate curcumin in the blood stream.

Or just take it in your favorite brand’s capsule. I don’t have an amount for ginger either. I just use a coin sized slice per day, after it’s been cooked.

I don’t know if sugared and brined ginger from the Sushi bar works. I also cannot say that it doesn’t work either.

Nobody said this was going to be easy.

Disclaimers:

Steve NYC

1- there is no proof in the literature that Curcumin will help humans with RP.

2- there are no human safety trials for RP people using Curcumin supplements

3- I am not a doctor

4- Curcumin at certain levels may interact with other medications you may be taking.  Large amounts may cause bleeding. Consult a doctor.

5- I do not know what these levels are.

6- Consult a medical professional before starting on any therapy

7- According to the Italian researchers in the May 2021 review article linked below, there are some forms of RP that will not respond to Curcumin

8- Short term risks or benefits have not been established.

9- This is information and not medical advice.

10- I suggest you read the research on the NIH.gov site.

11- I do not sell Curcumin or NAC or work for any company that makes any supplement product. Therefore I do not have any monetary compensation from the sale of this substance.

12- I’m just a guy with RP for sixty-eight years.

https://www.dovepress.com/recent-advances-and-disputes-about-curcumin-in-retinal-diseases-peer-reviewed-fulltext-article-OPTH

The end.

It hit me last night that I’ve done a really lousy job of this. I’ve been so focused on putting on a cheery face and a positive all-is-great attitude so that the Universe will deem me worthy of getting back my eyesight that I punish myself every time I make a misstep.

If I feel sad it means I’m focusing on my loss and drawing negative energy my way. If desperation sometimes overtakes me, it means that I’m not trusting and allowing that it’s out there for me. If I take a day off from staring into a color therapy lamp, applying micro current stimulation to my eyes and doing deep breathing to increase blood flow and circulation to my eyes along with every other thing I try, it means I’m not doing what it takes to earn back my vision. If I don’t accept my current lack of eyesight while I hold on to hope that an answer will soon materialize, it means I’m making my life harder for those I love. 

As I laid in bed last night pummeling myself for having all of these emotions, I realized I needed to take a step back and just love myself — and give myself some grace for being human.

I think it’s human nature to stuff down grief and trauma and charge forward with our lives, believing that if we give into the feelings it means we are somehow weak or not resilient. But I’ve realized that this approach can be really harmful. 

So I’m setting out on a new quest today. I’m really going to focus on self compassion and love. I’m going to be kind to myself when these emotions bubble up and trust that the Universe has my back anyway. My first step in self love is to admit that I have these emotions and that it’s okay. And tonight, instead of immersing myself in another self-help book in search of an answer, I’m going to settle into my hot tub with a full glass of wine and Billie Holiday singing away — and afterward, I might even indulge in some dark chocolate.

Okay — now it’s your turn. How are you showing yourself some compassion and love? Please let me know so I can share it here. And in the meantime, know that I am sending lots of love your way.

My memoir about my early journey with RP is finally available as an audiobook and I was given 25 free promo codes for anyone who is interested.

I wanted to share it here because I know my journey will resonate with a lot of you. My next book is going to end with me getting my eyesight back. 🙂

Below is more info on the book. If you are interested in listening to it and would like the promo code, click here.

From the Author of the New York Times bestseller Hippie Boy: A Girl’s Story

At the age of 37, Ingrid Ricks learned she suffered from Retinitis Pigmentosa, an incurable eye disease that was stealing her eyesight and would eventually leave her completely blind. Gripped with the terrifying fear that she would miss out on her daughters’ lives, become a burden to her husband, and lose the career and independence that defined her, Ingrid embarks on a quest to fix her eyesight that ends up fixing her life. 

Steve Fialkoff was only trying to boost his immune system when he started ingesting 250 milligrams of curcumin daily last December. The sixty-eight-year-old New Yorker had contracted Covid-19 and hoped the plant-based supplement — which is known for it’s anti-inflammatory and antioxidant properties — would help combat the virus. 

He didn’t give it much thought until February 8th, when he was out walking his guide dog. Usually Steve could see half of his dog’s head, one of his eyes or his tail wagging in a blurry narrow tunnel. But that morning, when he looked at his dog, Steve says he could see his entire head, whole body, individual hairs standing up and his tail all in one glance.

“Then I looked down on the sidewalk and realized that I could see the side of an apartment building and cars parked on the curb,” Steve adds. “In December, or even a week earlier, I wouldn’t even have been able to tell where I was because my vision was so narrow.”

Steve, a two-time Emmy-winning video editor who was forced to leave his career when his vision closed in on him, was convinced he was hallucinating. But when he returned to his Manhattan apartment and walked into his kitchen, he noticed that instead of seeing only one burner on his four-burner stove, he could see three and a half. And the only thing he could conclude is that his sudden jump in vision was connected to the curcumin he was taking.

His daughter did a Google search for the key words curcumin and Retinitis Pigmentosa and was immediately directed to a May 2021 research paper in which Italian researchers compiled all the studies done to date on curcumin and its positive impact on retinal diseases.

“The 30-page document talked about a study in which curcumin was fed to rats with RP and theorized that it would take a dose of three or four grams in people with RP to achieve the benefits,” notes Steve. “That’s a mistake because I was only taking 250 milligrams.”

In closer inspection of the label on his curcumin supplements, Steve discovered that it recommended 500 milligrams daily so he started taking it more religiously and upping the dose. On March 11th, Steve experienced another jump in vision. This time, he says he could see leaves on trees and people walking toward him.

Steve racked his brain, trying to figure out what else he was doing that could account for his vision gains. Once again, he pulled up the 30-page article on curcumin and retinal diseases. But this time, he decided to comb through it carefully in its entirety. Most of it, says Steve, was “garbley-gook”. But then he got to the end of it and discovered that the key to activating curcumin is piperine, the active ingredient in black pepper. According to the article, twenty milligrams of piperine increased curcumin absorption by two-thousand percent. Steve also discovered that it took about forty-eight hours for the curcumin to get into the retina after ingesting the two.

He started thinking about the ginger chicken he had ordered from his favorite Vietnamese restaurant three days earlier. It was a dish that contained an extensive amount of black pepper.

“I had my daughter check my credit card receipts and discovered that the last time I had ordered that dish was on February 4, a few days before my first jump in vision. I thought, aha, here it is. I accidentally took curcumin and accidentally took black pepper and it’s generating vision gains.”

Steve, who has the Usher 2a form of RP, was so excited by his discovery that in April he reported his vision gains to his retinal specialist. She conducted a visual acuity test and discovered that the vision in his right eye had increased from 20/400 to 20/250. Beyond that, Steve estimates that his central visual field has expanded to about six degrees, a big jump from the narrow two-degree hole he had been seeing out of. Because eye doctors had stopped measuring his visual field a decade ago, there was no way to measure that expansion during his visit. What’s more, Steve says his retinal specialist wasn’t interested when he told her his vision jump was due to curcumin.

Steve, who had heard about other treatments such as acupuncture and Chinese herbs that had helped RP sufferers for a month or two but then stopped working, sometimes had doubts and waited for the other shoe to drop. But he kept adding black pepper to his meals and kept taking the curcumin and his vision gains held steady. In July, he decided to look up the recipe for the ginger chicken dish that had precipitated both jumps in vision. That’s when he discovered that a heavy dose of black pepper was sautéed in oil with ginger for several hours. In further research, he learned that heat and fat also help with activation and absorption of curcumin, and that ginger is also beneficial for the retina. Steve began replicating the recipe at home and, two weeks later, he experienced another jump in vision. This time, he noticed that he could see when walking through a scaffolding tunnel where the light was lower, something he hadn’t been able to do for several years. He also noticed that he could see more detail in bright light.

He decided it was time to call the Foundation Fighting Blindness to share his vision gains with curcumin. He was routed to their science reporter, who noted that an Indian researcher they fund had identified curcumin as having a positive effect on a rare form of RP. But he also said they would need more double-blind studies to confirm this finding.

“I wasn’t going to argue with this guy,” notes Steve. “I didn’t want to sound like a petulant brat. But I know in my heart we don’t need a double-blind study. We need a study, with twenty or thirty people, and if they don’t go blind and some of them get better, then something is happening.”

This is where Steve comes in. He has launched his own informal curcumin experiment and currently has twenty people — most with RP and a few with other retinal diseases — on board. Not everyone is following his regimen. Some are ingesting piperine supplements with curcumin supplements. Others are experimenting with whole turmeric — the plant responsible for curcumin. Still others are mixing both turmeric and ginger. Steve will be sharing the results as they come in.

He doesn’t know if his curcumin concoction works for every form of RP or whether his eyesight will continue to improve. But for him, the vision gains he has experienced over the past eight months are nothing short of miraculous.

“In a way, it’s a good thing that I was almost blind. Because if my visual field was 15 or 20 degrees and I still had good central vision, I probably wouldn’t have noticed the vision gains and would have eventually stopped taking curcumin,” notes Steve. “Before, I would say that I was 98 percent blind. Now I estimate that I am 93 percent blind and back to the vision I had in 2018. I’m just going to keep going and see what happens.”

If you would like to join Steve’s curcumin experiment, please email him at: tinymustacheprods@gmail.com

To read the May 2021 article on curcumin and retinal diseases, click here.

To read the January 2022 article on curcumin and retinal diseases, click here.

Nanoscope Therapeutics was recently singled out for its ground-breaking optogenetics drug at the OIS Retina Innovation Summit in New York, the latest achievement in the company’s quest to restore meaningful vision to the millions of us suffering from debilitating retinal diseases.

Since announcing the remarkable results of its Phase 1/2a clinical trial for Retinitis Pigmentosa last June, the company has been on a tear — launching a Phase 2b clinical trial for RP as well as a Phase 2 clinical trial for Stargardt disease. Results from both of these trials will be announced first quarter of 2023. I have been watching Nanoscope closely over the past year because as far as I can tell, it offers the best hope for vision restoration in those of us living with late-stage RP — regardless of gene mutation. I had the opportunity to interview both CEO, Sulagna Bhattacharya, and President, Samarendra Mohanty, and was moved by their incredible passion and commitment, which at this point has been decades in the making.

To read more about Nanoscope’s remarkable results in their Phase 1/2a trial in India, click here.

For a deep dive into the decades-long journey behind this game-changing drug, click here.

Researchers at University of California, Berkeley and UC Santa Barbara have discovered that disulfiram, a drug that combats alcoholism, may also be the key to restoring vision in people with advanced RP and age-related macular degeneration.

The key is that disulfiram, which inhibits enzymes involved in the body’s ability to degrade alcohol, also impedes the enzymes that make retinoic acid. What makes this so critical is that researchers have found that retinoic acid interferes with light perception in those with RP. By inhibiting the enzyme that creates reitinoic acid, they were able to eliminate the interference in nearly blind mice, enabling them to see images on a computer screen.

I’ve reached out to the researchers involved in this trial for an interview but have not yet heard back from them. However, here is the gist:

• Disulfiram has already been approved by the FDA for treatment of alcoholism — which greatly reduces the barriers for making disulfiram available as a treatment for people suffering from RP.
• The drug has very little known side-affects, unless you drink alcohol — in which case, it will make you relive your worst hangover (think severe vomiting, headache, etc.).
• The next step, according to news reports, is a clinical trial in which researchers will team with a handful of ophthalmologists to administer the drug to patients with advanced RP (but not complete retinal degeneration).

Here’s a link to a detailed article that appeared in Berkeley News and here’s a link to the Science Advances Journal article.

Bottom line — great news.

I didn’t think doctors or researchers would ever crack the code to my RP-suspected vision loss. I don’t have any family history and the two genetic tests I underwent in 2018 came up empty. But with all the breakthrough medical advancements now being made on the RP front, that might as well have been light years ago. 

In the last three years alone, researchers have identified 140 new gene mutations associated with RP, bringing the total to around 320. So, at the urging of a new retinal specialist, I gave it another go. And lo and behold, eight gene mutations linked to RP were found. At first, I didn’t understand why knowing these mutations were of benefit to me because it’s not like I have a strong dominant gene that is being targeted for gene therapy. But as it turns out, having a known gene association with RP is a key criterion to participate in any of the promising RP clinical trials underway — including the gene agnostic optogenetics therapy developed by Nanoscope therapeutics now being tested in a phase 2B clinical trial throughout the US.

Beyond this, in my case, I’ve uncovered a critical clue that may be tied to both my vision loss and the triple-negative breast cancer I was diagnosed with six years ago: Arginine, an essential amino acid that is critical for blood flow and circulation, is being stripped from one of my genes.

This was a light bulb moment for me and has led me to adopt the Budwig protocol, which was developed by German Biochemist Johanna Budwig to treat cancer patients. What she discovered is that, like me, severely ill cancer patients were missing this essential amino acid in their cells. It’s a key ingredient in flaxseed oil. The problem, according to Budwig, was that in order for cells to absorb this essential amino acid, the oil needed to be delivered through a water-soluble mechanism. To solve this issue, she blended flaxseed oil with low-fat cottage cheese (or quark), which makes the oil water-soluble.

There is limited research on the Budwig Protocol, though many cancer survivors swear by it. And given that this essential amino acid is also critical to eye health, Budwig Protocol here I come. 

What I know for certain is that vision-restoring answers are finally on the way for us. In the meantime, we have got to do our part. We have to do everything we can to slow the degeneration so our retinas are in the best shape possible for the emerging therapies. Knowledge is power, so the more we learn about our specific mutations through the extensive genetic testing now available, the more we help researchers help us — and the more we may be able to help ourselves.

Because of my recent genetic test results, I have a new tool I can add to my personal eyesight retention and restoration plan. And that, along with the regular OTC scans I’m now undergoing to carefully track and monitor my retina and any changes occurring, ensures I can be ready to jump as soon as the right therapy for me materializes.

I’m shouting out my gratitude to every researcher, scientist, and eye doctor who are working on our behalf — as well as to the many foundations and individuals who have and continue to generously fund eye research. Thanks to all of you, I know I’m going to see my family’s faces and my world again.

On the surface, Nanoscope’s June 3rd news release announcing the remarkable vision-restoring results of its phase 1/2a clinical trial in India might sound like an out-of-nowhere miracle. But in reality, their breakthrough optogenetics drug that restored meaningful vision in 11 previously blind RP patients has been decades in the making. Without ongoing biomedical advancements combined with the rare skill set, unrelenting determination, and unwavering belief in the impossible by company president, Samarendra Mohanty, and his team, this therapy would not exist.

The Backstory

Mohanty, who grew up in a small town in India, was drawn to science because of its ability to create magic that could change the world. After graduating from the Indian Institute of Technology Delhi and the Indian Institute of Science, he began his career as a researcher and scientist at the Center for Advanced Technology in central India. It was there that he was first exposed to applications of light — especially lasers in the diagnosis and treatment of disease. His early work focused on developing devices and technology to detect oral and cervical cancers. But his focus turned to eye diseases when he read an article about optical tweezers by the inventor Dr. Arthur Ashkin, who was later awarded the Nobel prize for his discovery. 

Mohanty was soon in Germany working alongside another pioneer in the field, Professor Karl Otto Greulich, and eventually returned home to launch the first optical tweezers and laser micro manipulation lab in India.

By the time Mohanty landed at UC Irvine in 2006, he had a deep expertise in biophysics, lasers, and optics. It was a unique combo that made him the perfect fit for the newly emerging optogenetics field. Mohanty immediately began teaming with ophthalmologists and neuroscientists and set to work to crack the code that could restore vision in millions worldwide.

“Working in collaboration with neuroscientists, we developed different promoter-based opsin genes to target ganglion and bipolar cells of the retina,” notes Mohanty, who adds that this critical work was funded by the Greishaber Foundation. “I also demonstrated for the first time the non-viral delivery of opsin-genes to adult mice retinas by electroporation and spatially targeted laser delivery.” 

Like other researchers at UC Irvine, Mohanty started using monochromatic (blue) light to activate channelrhodopsin-expressing ganglion cells in the retina. But later in his career, while working as a professor at the University of Texas, he observed damage to opsin-expressing ganglion cells after chronic use of blue light. And beyond the toxicity issue, Mohanty wasn’t satisfied with just bringing back narrow bans of vision that required intense light and only resulted in restoring a patient’s ability to see shapes, shadows, and stripes. He was determined to restore meaningful vision that gave patients back their lives — which meant achieving vision restoration in a natural, broadband light environment. 

“I started fusing different monochromatic (blue, green, and red) opsins to generate broadband light sensitivity,” explains Mohanty. “However, the fused opsin gene was too large to be packed in a known-to-be-safe AAV viral vector. Therefore, we delivered such genes by a non-viral laser method, which needed further instrumentation and image guidance to be translated to humans. Moreover, I found that the broadband opsin generated by fusing three opsin-genes did not have enough sensitivity to be activated at natural/ambient light surroundings, which meant that, like narrowband opsins, we still needed an intensifier to instill vision in subjects with retinal degenerative diseases.” 

Mohanty continued to wrestle with the challenge while immersing himself in his academic work as a biophysics professor. Between faculty tasks, setting up biophysics labs, training students, teaching classes, and applying for grants, there wasn’t much time for focused research. But he continued to chip away at it — determined to give people who had been blinded by degenerative eye diseases the ability to see their world again. 

“I never considered giving up in spite of major personal and professional challenges,” says Mohanty. “I was distressed by my father’s health issues and death. I was disappointed when narrowband opsin and monochromatic light stimulation did not produce a safe and efficacious outcome. But as a self-motivated scientist and entrepreneur, I would never give up. I am passionate and driven by discovery, translation, and creating a real impact for patients with devastating neurodegenerative diseases.”

The Breakthrough

The breakthrough Mohanty and his team were searching for finally materialized in early 2016, when new MCO mutants were discovered. These opsins possessed three critical attributes: first, they were so sensitive to light that they eliminated the need for an intensifier. Second, they worked fast. And finally, they responded to broadband white or any color of visible light encountered in daily living. 

Mohanty began utilizing these MCO opsins — and that’s when the magic happened. He and his colleagues injected their newly refined optogenetics therapy into the bipolar cells of lab mice who had been blinded by RP and were able to activate them into light sensitive cells that worked in natural broadband light environments. While it was a monumental achievement, Mohanty and his colleagues immediately focused on the next step: testing the therapy in people blinded by RP. Then came serendipity. Dr. Mahapatra, a renowned ophthalmologist and retinal surgeon in India, was searching for something to help his patients with RP when he read the pre-clinical trial results published in the journal, Neurophotonics. In early 2017, he traveled to Texas to visit Nanoscope Therapeutics — which Mohanty had recently co-founded with CEO Sulagna Bhattacharya. 

“We found him to be equally passionate and truly dedicated to treat his RP patients with severe retinal degeneration,” explains Mohanty. “Since we did not have any experience in conducting a clinical study in India, Dr. Mahapatra introduced us to a CRO who could navigate and coordinate the clinical study.” 

Next Steps

Fast forward to now. Thanks to the remarkable results of the India trial, the FDA recently approved a Phase 2b clinical trial for late-stage RP that is currently underway at six locations throughout the United States. The results of that trial are expected to be delivered to the FDA in the third quarter of 2022. If the results from the U.S. trial mirror those of India, Mohanty hopes the FDA will consider an accelerated approval timeline so Nanoscope can start getting their therapy into the eyes of those of us who are clinging to the hope that we will once again be able to see our worlds.

“It means a lot to me and our team to give hope to the patients who have lost their vision due to degenerative eye diseases,” says Mohanty. “To me, it is the purpose of my life. I was very happy that some patients in our phase 1/2a study improved remarkably and that those patients/family members spoke of our therapy as extraordinary. I hope our phase 2b trial will have a similar outcome. The RP patients participating in our study will be contributing to our resolve to enable millions to see again.”

(Editor’s Note: I did everything in my power to get into this clinical trial but was excluded because of a stem cell therapy I received a few years back. I am so happy for those of you who were accepted into this trial and so hope that you will all be able to see your world again and that it will translate into a treatment that the rest of us will be able to access soon.) 

I’ve been trying to find the words to describe the flood of emotions that have been swirling inside of me since talking with the team at Nanoscope Therapeutics about their recently announced vision restoring optogenetics therapy for late stage RP. I’ve alternated between crying, doing a happy dance, and shouting out thank you prayers to the universe. After trying to claw my way out of a dark pit for the past 17 years, I’ve finally found light. And I’m so full of hope and gratitude that I’m ready to burst.

In case you somehow missed it, Nanoscope Therapeutics, a clinical stage biotech based in Bedford Texas, issued a June 3rd press release detailing the results of their Phase 1/2a optogenetics clinical trial that took place in India over the past 2 years. By all accounts, it appears that they have cracked the code when it comes to restoring vision in late stage RP patients — regardless of gene mutation.

I’m talking functional vision restoration — not shadows, or dark blobs, or white stripes. Real, functional vision that has previously-blind patients navigating complex public transportation on their own, watching TV, reading newspaper headlines and, in one case, even riding a bike and recognizing facial features.

What’s more, their therapy isn’t dependent on implanted microchips, or on wearing external devices such as cameras or goggles. It involves a single injection of molecules into the eye targeting bipolar cells that lay just beneath the photoreceptors cells that are destroyed by RP. The therapy activates those bipolar cells, turning them into light sensitive cells that react to ambient light — the kind of natural light found in everyday living. And as far as researchers can tell, the vision restoration appears to be permanent.

“The results varied among patients, with some better than others,” notes Nanoscope’s Co-Founder and President, Samarendra Mohanty, who has been working on the development of the therapy since 2006. “But every patient who received the therapy has experienced significant improvement — both objectively and subjectively.”

The eleven patients involved in the phase 1/2a clinical trial underwent extensive testing over the course of a year, utilizing every vision-measuring metric the team could apply.

From a straight visual acuity standpoint, Mohanty said the most dramatic improvement saw a patient’s visual acuity jump from 20/2000 to 20/200. But that was just the beginning.

Patients in the trial also underwent visual field tests to measure peripheral vision, tests to measure light perception in progressively dim light conditions, and even tests to measure shape and determination accuracy. A variety of mobility tests were also performed to see how accurately a patient could identify a light source and moving objects. Dramatic improvements were seen across the board.

Along with experiencing dramatic visual improvements within four weeks of the therapy injection, Mohanty said that Nanoscope’s optogenetics therapy seeks to address another critical factor: the halting of further degeneration.

“After the photoreceptors cells die, the retina continues to degenerate,” notes Mohanty. “But we have preliminary indication of halting further degeneration and that’s very very exciting for us ”

The next step in bringing this therapy to patients everywhere is the newly announced Phase 2b clinical trial in the United States that will kick off within the next few weeks and be administered at several locations throughout the country (for more information, go to clinicaltrials.gov or contact the clinical trial coordinator, Kristen Peterson, at info@nanostherapeutics.com.). Like in India, the U.S. clinical trial is focused on late stage RP patients with bare-to-no light perception. To qualify, a patient’s visual acuity must be 20/1600 or worse.

“We wanted to start with them because we wanted to give hope to people who have absolutely no hope and have been left behind,” explains Nanoscope’s Co-Founder and CEO, Sulagna Bhattacharya, who watched her beloved uncle go blind as a child and experienced the devastating impact it had on him and her entire family. “This is why we started with RP. Our therapy also has potential to help Stargardt, and we are confident that it will also potentially benefit those with dry age-related macular degeneration. But most of the Dry-AMD patients still have a little vision left, and we wanted to start with those who were suffering the most.”

The phase 2b trial results are expected in the third quarter of 2022. If all goes according to plan, the FDA may be able to expedite the approval process so that Nanoscope can get their life-transforming therapy to patients as soon as possible.

There is too much to this story to capture in a single post. But I’ll cover it in depth in several segments over the next month. These segments will include:

• The fifteen-year story behind the development of this breakthrough therapy and Mohanty’s determination to figure out how to restore vision in ambient light — which was key in activating the bipolar cells and restoring meaningful vision
• The remarkable journey of Bhattacharya, who has devoted her life to finding a cure for blindness after her uncle lost his eyesight — even while enduring unimaginable loss and grief
• The team’s commitment to making this life-changing therapy affordable and accessible to patients everywhere, and the uphill challenge to achieve this

Like all of you who have been following this blog since its inception in early 2013, I’ve refused to believe that I am destined to live in a world of darkness. Even after my eyesight took a nose-dive in 2018 and my retinal specialist told me it was time to transition to blindness, I’ve maintained my determination to see. Now, though there is still a ways to go, it feels close enough to touch. I am so grateful to all of the scientists and researchers out there who are devoting their lives and careers to bringing back our eyesight and our lives. And I’m making it my mission to do everything I can to raise awareness and help get these life-changing treatments out to the world.