From Wikipedia:
“Stargardt disease, or fundus flavimaculatus, is an inherited form of juvenile macular degeneration that causes progressive vision loss usually to the point of legal blindness. The onset of symptoms usually appears between the ages of six and thirty years old (average of about 16–18 years). Several genes are associated with the disorder. Symptoms typically develop by twenty years of age, and include wavy vision, blind spots, blurriness, impaired color vision, and difficulty adapting to dim lighting.
The long-term prognosis for patients with Stargardt disease is widely variable although the majority of people will progress to legal blindness. Stargardt disease has no impact on general health and life expectancy is normal. Some patients are able to drive.”
From my Retina Specialist:
“If you enjoy reading books or driving, you should probably do a lot of it before you turn 50.”
Random takeaways from yesterday’s appointment:
– Stargardt disease requires inheriting one specific gene from each parent. Because of this it often/usually skips generations.
– The Dean McGee Eye Institute sees approximately 4 cases of Stargardt’s disease each year.
– Typically, Stargardt disease affects both eyes at the same rate. In my case, it hasn’t. The doctor called my case unique and referred to it as a rare form of a rare disease.
– Dean McGee has asked me to come back for more research so they can gather more information about this rare form of Stargardt’s.
– Stargardt disease is unrelated to Horner’s Syndrome (the reason I have different colored eyes). The fact that I have both is a coincidence, although it may explain why the Stargardt has affected my eyes at different rates.
– Stargardt only affects your central/focused vision, not your peripheral vision. That’s why it affects your ability to read or recognize people’s faces, but not your ability to maneuver a room, for example.
– The previous diagnosis of macular degeneration/atrophy is really just a product of Stargardt disease. None of the treatments (vitamins, shots, etc) will have any affect on my vision.
– While some research with stem cells is being done, there is no current treatment, prevention, or slowing down the progression of the disease.
– Legal blindness is defined as 20/200. The vision in my left eye is 20/1500. My right eye is currently 20/25.
– The doctor expects me to be legally blind within the next 10 years, give or take.
I feel the same as when I found out about Gage. I wish I could fix this. I love you and would do anything for you. Very few people enjoy life the way you, Susan and the kids do. This is not fair. I am so sorry and will pray that you are the exception.
The chairman of my board of directors is a world-renown specialist on macular degeneration. I am going to ask for his opinion, if that is ok with you.
Wrote a big long note but decided it was sappy and not suitable for publication. Just imagine if it were one of your kids telling you this, and how you would feel, and know I feel that way. I love you, and I’ll be here for you til my dying day. Love, Mom
Much love to you, my friend. But, I can totally see you kicking this in the ass and putting those statistics to shame. You’re a hard ass brotha!!! ;)
Love and hugs to you!!!!!
Please google “Robert Lanza” and Ocata.com
Hope is on the way
We believe in the power of vision. Ocata Therapeutics, Inc. (“Ocataâ€; NASDAQ Global Market: OCAT), formerly named Advanced Cell Technology, is a clinical stage biotechnology company focused on the development and commercialization of regenerative medicine and cell therapy technologies. The company’s most advanced products are in clinical trials for the treatment of Stargardt’s macular degeneration, dry age-related macular degeneration and myopic macular degeneration. Ocata’s preclinical programs involve cell therapies for the treatment of other ocular disorders and for diseases outside the field of ophthalmology, including autoimmune, inflammatory and wound healing-related disorders.
Blastomere pluripotent stem cell derived RPE therapy is about to enter a pivotal trial in England and SPA approval from the FDA for Stargardt’s Disease to secure data break through status and phase 2/3 clinical trials for dry Age Related Macular Degeneration in the next few months.
Times since the initial review process at the NIH in 2010 and the technology has changed and progress has been made with these cells and human clinical trials have shown to be safe and stability for nearly 4 plus years with great effectiveness without any side effects. Now, below a scientific justification is FDA approval clinical trials form an IND for AMD and SMD RPE cell line derived from Blastomere technology and other patent and recent papers for justification and validation to move the define definition of human embryonic stem cell lines for use in Federal and NIH research.
Another likely validation of the technology is the patents with USPTO. Why? A Patent Abstract are posted below, but most recently Ocata blastomere pluripotent technology the pre embryonic cells in questions for the NIH definition change was awarded May 25, 2015 “United States Patent and Trademark Office (USPTO) issued three new U.S. Patents, Nos. 9,040,770, 9,040,039 and 9,040,038, which expand the scope of protection of the four previously issued U.S. patents covering its retinal pigment epithelium (RPE) transplant technology. These three new patents extend protection to include all formats and formulations of RPE cells, including suspensions and sheets of cells, for use as therapeutic agents, and the use of these formulations for treating ophthalmic diseases such as dry age related macular degeneration (Dry AMD) and Stargardt’s macular degeneration (SMD)â€. The MA-09 technology of blastomere pluripotent stem cells in fact has “Ocata has a total of seven U.S. patents that specifically protect retinal pigmented epithelial (RPE) products derived from human embryonic stem cells (hESC) as well as any other pluripotent stem cell source including induced pluripotent stem cell (iPSC) sources.â€
And Dr. Eddy Anglade, Chief Medical Officer of Ocata. “For the first time a pluripotent stem cell derived therapy is about to enter a pivotal trial for Stargardt’s Disease and phase 2 clinical trials for dry Age Related Macular Degeneration, bringing the possibility of these long awaited therapies that much closer to reality.â€
http://ir.ocata.com/press-releases/detail/2780
Continuing on to the patent claims from other patents “The present invention generally relates to novel preparations of mesenchymal stromal cells (MSCs) derived from hemangioblasts, methods for obtaining such MSCs, and methods of treating a pathology using such MSCs. The methods of the present invention produce substantial numbers of MSCs having a potency-retaining youthful phenotype, which are useful in the treatment of pathologies.â€
The key words of this patent are having a potency-retaining youthful phenotype, CD10+, CD24+Stro-1 negative.(see patent references (WO2013082543))
This claim from the patent eliminates most hESc cell lines and all adult MSC. “Claim #11 . The preparation or cell bank of any of claims 1 -5, wherein at least 30% of the
mesenchymal stromal cells are positive for CD 10.†Not seen in federal funded NIH adult research cell lines.
“Tissue source: Blastomere cell derived from fertilized oocytes. Pre-embryo consent use provided as supernumerary tissue for cell extraction for science rather than discarding during standard IVF treatment. Patented non-embryo destructive technology (NED) used to establish hESC cell lineâ€
“Processing steps: Chemical ZP breaching, single blastomere cell extraction, isolated cell co-culture with pluripotent/hES cells in a Quinn’s Cleavage Medium, further culture steps using Quinn Blastocyst Medium on mitomycin C-treated MEF feeder layer with DMEM & bFGF. hESC master cell line established & banked. RPE spontaneous derivation following continued hES cell culturing methods, on MEF in medium without LIF, FGF and Plasmanate or from suspended embryoid body overgrowths in long term culture. The resulting pigmented islands of cells are enzymatically digested and RPE cells isolated and plated for differentiated primary cell cultures for passaging, expansion, working cell bank, harvest & cryopreservation.â€
“Stability/Safety: Genetically stable, normal karyotype, normal senescence & terminally differentiated to its RPE fateâ€
“Proposed mechanisms of action and potency: RPE cell replacement therapy aimed to restore lost retina cells at the base of the macula to establish a new and healthy support layer to the photoreceptors (Vitamin A, phagocytosis & waste processes, nutrients & factors). It is envisioned that by replacing the lost RPE layer the remaining photoreceptors will once again function properly thereby either arresting the decline of the age related condition or actually improve lost visual acuity.â€
From Patent
Note: Text based on automatic Optical Character Recognition processes. Please use the PDF version for legal matters
“Pivotal Clinical Trial Program Design for Stargardt’s Macular Degeneration
Related Links
MARLBOROUGH, Mass.– Ocata Therapeutics, Inc. (“Ocataâ€; NASDAQ: OCAT), a leader in the field of regenerative ophthalmology, today announced that the company has received written formal guidance from the European Medicines Agency (EMA) related to the advancement of its Retinal Pigment Epithelium Program (RPE) into a pivotal clinical trial for Stargardt’s Macular Degeneration (SMD) from the London trials.†All twelve(12) patients injected with human RPE derived from MA-09 derived cell line, London data has been not reported out as of 5-27-2015 by Ocata Therapeutics.
Hi Rob,
I wish you good luck in your treatments and pray that your Stargardts will spare you. I became aware of your posting here through another discussion blog, and though I’m an investor in this company, Ocata, I thought you might want to know about their ongoing trials, which are quite promising. Of course, it is all still experimental, but the results are encouraging and I’m just sharing them to let you know, there may be some hope. They will start a PIVOTAL Phase II trial shortly, and have trials in the EU and US, with a partner that is doing separate trials in Korea for Stargardt’s.
I’ve included links below to peer reviewed journal articles, including a link to the company web site at the bottom, and you might contact them about possibilities of being in their trials, though, unfortunately, to satisfy critics permanently, there will be a placebo arm to the trial. In Phase I, they used the patient’s other eye as the placebo, but there were calls to make it a double blind trial, so some patients will not get the actual treatment, just FYI.
But I highly recommend that you both read the links, and then go to google and research. Read everything, there are some videos and presentations on YouTube as well. I think this is the best trial, with the best results, and best potential right now, but you should read for yourself, and recognize that the trials are at the top eye centers in the US, and UK and that the doctors at those centers are signatories to the journal articles to which I will link. That means, generally speaking, that they are independent of the company. Additionally, many companies just PR their results, but these results are peer reviewed. I think that means that the results carry great weight, at least for me.
In the article with 18 published patient results, you’ll see that 13 of 18, had some recovery of visual acuity, but a total of 17 of 18 patients, had the disease improve or stop, and 1 patient did not suffer adverse affects from the cells, though they did not improve and had their disease progress to wet AMD (advanced macular degeneration). The patients are being treated for either Stargardts or Advanced Macular Degeneration, different diseases.
The company is also hoping to bring a trial for other types of eye cells. Retinal cells are meant to preserve the environment to save rods and cones in the eye that have gone dormant because of macular degeneration. But they hope eventually to replace lost rods and cones with another set of cells. Retinal cells simply preserve the retina tissue, but retina tissue does not generate the signals for one’s eyes, rather a healthy retina creates the proper environment for healthy rods and cones.
Good luck again!
2014 results: http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(14)61376-3/abstract
First two Patients: http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(12)60028-2/abstract
A total of 42 patients have been treated worldwide, and pivotal Phase II should start soon. I don’t know where you live, but there are trial sites at a number of university hospitals around the country.
http://www.popsci.com/yes-you-can-reverse-disease
Data from the company’s first two clinical trials, published last year, confirm the treatment works. Ten of 18 people experienced some improvement in their vision, and the therapy seemed to halt the loss of vision in another seven. Some even had a dramatic recovery: A 75-year-old rancher who had gone blind in one eye was able to start riding his horses again. It’s likely still a few years from Food and Drug Administration (FDA) approval, but Anglade hopes the treatment will one day become as common as cataract surgery.
https://www.ocata.com
http://en.wikipedia.org/wiki/Ocata_Therapeutics
NPR story, that you can listen to, from a few YEARS ago, not current:
http://www.npr.org/sections/health-shots/2012/01/23/145636849/stem-cells-show-promise-as-blindness-treatment-in-early-study
WSJ article on most recent trial results, with a video and sound:
http://www.wsj.com/articles/stem-cells-show-potential-benefits-for-eye-diseases-1413327157
Just for your information, the company’s name USED to be Advanced Cell Technology, Inc. It’s name is now Ocata.
The Chief Scientist of the company, is Dr. Robert Lanza:
http://en.wikipedia.org/wiki/Robert_Lanza
http://www.robertlanza.com
The embryonic stem cells for this trial were derived by a special means that, by itself, does not destroy an early 4 to 8 celled embryo, that came from an Invitro Fertilization Clinic and was slated to be destroyed by the clinic, after the couple who wanted a child, had accomplished that task:
One embryo derived all treatments and will do so indefinitely. One stem cell, creates a LINE of stem cells, and those cells grow forever and create unlimited treatments. So once you create a line, it can go on forever as vats of pluripotent cells that can become any part of the human body. The retina is immune privileged, which means the tissue does not have to match, like a heart transplant. So when they transplant these cells for each patient, they are cells from the same original single embryo.
The cells for the RPE treatments, for stargardts and AMD, were all generated from one embryo, and generated by a biopsy of that single embryo. They do this just like when they biopsy the embryos before they implant them in IVF clinics for couples, to ensure they don’t have an genetic disease. So they do not have to destroy an embryo, to start a stem cell LINE, and that LINE of cells will last indefinitely for all treated patients.
The company has de-emphasized this technology below (blastomere technology), just because it doesn’t seem to satisfy certain critics anyway, but you might find this interesting: Human embryonic stem cell lines derived from single blastomeres (without embryo destruction). http://www.ncbi.nlm.nih.gov/pubmed/16929302
http://www.cell.com/cell-stem-cell/abstract/S1934-5909(07)00330-X?_returnURL=http%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS193459090700330X%3Fshowall%3Dtrue
Summary
To date, the derivation of all human embryonic stem cell (hESC) lines has involved destruction of embryos. We previously demonstrated that hESCs can be generated from single blastomeres ( Klimanskaya et al., 2006 ). In that “proof-of-principle†study, multiple cells were removed from each embryo and none of the embryos were allowed to continue development. Here we report the derivation of five hESC lines without embryo destruction, including one without hESC coculture. Single blastomeres were removed from the embryos by using a technique similar to preimplantation genetic diagnosis (PGD). The biopsied embryos were grown to the blastocyst stage and frozen. The blastomeres were cultured by using a modified approach aimed at recreating the ICM niche, which substantially improved the efficiency of the hESC derivation to rates comparable to whole embryo derivations. All five lines maintained normal karyotype and markers of pluripotency for up to more than 50 passages and differentiated into all three germ layers.
They hope to have the treatments on the market for Stargardts and maybe AMD by the latest 2019, we all hope. But dates can slip, it’s a highly regulated process, and nothing is ever certain in this sort of endeavor. Again, good luck!
For the Wall Street Journal / WSJ article, with video, it’s a pay site, but if you use this google link, and click the first link to the WSJ article, you can get it for free:
http://www.wsj.com/articles/stem-cells-show-potential-benefits-for-eye-diseases-1413327157
Apologies, I meant to add this link:
https://www.google.com/search?client=safari&rls=en&q=Stem+Cells+Show+Potential+Benefits+for+Eye+Diseases&ie=UTF-8&oe=UTF-8#q=Stem+Cells+Show+Potential+Benefits+for+Eye+Diseases+wsj
Rob,
I’m a retired, former consultant to the ophthalmic industry (over 35 years), who now (also) writes a blog, focused on new technologies to treat retinal diseases, including Stargardt’s. I also publish continuously updated tables of who is doing what to treat ophthalmic diseases, again including Stargardt’s. If you are interested in learning more about either my blog or my tables, please email me at iarons(at)erols.com. There is a lot of research and clinical trials underway in seeking an answer to this disease.
In the meantime, you should think about joining the Stargardt’s and Macular Degeneration support group on Facebook (link: https://www.facebook.com/groups/Stargardts/). I’d be happy to invite you if you are interested. (It is a closed group, joined by invitation only.)
Please let me know if I can be of assistance.
Irv Arons
Here is the latest talk by Dr. Lanza. He goes very fast but there are a number of stunning statements in there that say we are on the cusp of a total revolution in Regenerative Medicine. I’m invested in Ocata for over 8 years, when it was Advanced Cell Technology. I also have AMD. Still seeing ok. Just need the freakin’ government to get out of the way.
Sorry, forgot to paste: https://www.youtube.com/watch?v=4B8U7AfnbXk