Potential stem cells treatment for retinitis pigmentosa and other retinal degenerations

A team of researchers from The Scripps Research Institute was able to preserve visual function in mice that were genetically predisposed to developing a profound degenerative disease that destroys their retinas.

The team injected adult bone marrow-derived stem cells from mice or humans into the back of mouse eyes at an appropriate stage of development and these injections dramatically curtailed retinal degeneration. In the latest issue of the Journal of Clinical Investigation, the team shows that the treated eyes of the mice, when compared to the fellow untreated eyes, had a completely normal retinal vasculature, had significantly improved retinal tissue, and responded to light.

“The surprising findings by Dr. Friedlander and colleagues establish a dramatically new paradigm for understanding and potentially treating retinal degenerative diseases using a cell-based approach,” says Paul A. Sieving, M.D., Ph.D., director of the National Eye Institute, National Institutes of Health. “Determining the precise mechanism of this cell-mediated rescue presents an exciting research challenge and is a high priority for the Institute.”

This approach could potentially be used to treat disorders of the retina that have vascular and neuronal degeneration. Such inherited degenerative retinal disorders are known collectively as retinitis pigmentosa. According to the National Eye Institute, more than 100,000 Americans suffer from retinitis pigmentosa, which is caused by more than 100 different genetic mutations.

"For patients with retinitis pigmentosa, this may be tremendously important," says Martin Friedlander, M.D., Ph.D., who led the study. "In the mouse, we have used both mouse and human cells to preserve nearly normal vascular and neuronal architecture of the retina in a disease model that ordinarily exhibits profound degeneration. Our hope is that if these results translate into humans with the disease, we would be able to maintain vision for these patients longer."

Currently, there is no way to treat patients with retinitis pigmentosa and no way even to slow the disease, says Friedlander, who is an associate professor in the Department of Cell Biology and the chief of the Retina Service in the Division of Ophthalmology, Department of Surgery, at Scripps Clinic. Friedlander has had a longstanding research program looking for new and better ways of treating eye diseases such as age-related macular degeneration, diabetic retinopathy, and retinitis pigmentosa. Four years ago several members of his group, including Atsushi Otani, M.D., Ph.D., and Michael Dorrell, Ph.D., began to explore the potential utility of adult bone marrow-derived stem cells for the treatment of these disorders, and a series of studies published over the past three years has confirmed that such an approach may be useful both scientifically and clinically.

The Key to Treating Retinitis Pigmentosa

The retina is akin to an extension of the brain into the back of the eye. It is a layer of blood vessels and nervous tissue that covers about two-thirds of the back of the eyeball and is connected to the brain through the optic nerve. Its primary purpose is to capture light and transduce those physical cues into electrical signals, which it then sends to the back of the brain where the sensory signals are interpreted into the visual experience.

Retinas contain a number of specialized cells, including the rods and cones, which capture light and send electrical signals to the brain, and glial support cells. The retina also has an extensive vasculature—a fine mesh of blood vessels formed in the third trimester of human gestation and in the first month postnatally in mice by endothelial cells, the major cell type lining blood vessels.

In normal mice, these retinal blood vessels form during the first three to four weeks after birth and provide blood to the inner two thirds of the retina. In mice that are predisposed to developing retinal eye disease, the outer layer of the retina containing the rods and cones as well as other neuronal layers degenerate within a few weeks after birth. Most of the cell death occurs by apoptosis, or programmed cell death. The retinal blood vessels are present in three layers, and in the model of retinal degeneration the two deeper retinal vessel layers completely degenerate by about one month after birth.

In humans with retinitis pigmentosa, a very similar process occurs, and this leads to profound vision loss and eventual blindness. The disease starts as progressive night blindness and the gradual loss of peripheral vision, and it leads eventually to complete tunnel vision or in some cases total blindness.

More than 100 different types of gene mutations have been documented that lead to this degeneration of the retina, and about one in every 3,500 people suffer from loss of vision caused by retinitis pigmentosa.

But at least in mice, Friendlander and his colleagues found that blindness can be prevented by injecting adult bone marrow-derived stem cells into the back of the eye.

A New Kind of Protection

The group's basic approach starts with selecting what are called lineage negative stem cells from the bone marrow. Adult bone marrow stem cells are "pluripotent" and have the potential to develop into a number of different cell types, such as red blood cells, platelets, or white blood cells. Lineage negative stem cells have the capability, among other things, of becoming endothelial cells—the major type of cell that lines the body’s blood vessels.

Friedlander and his colleagues found that the lineage negative stem cells, once injected into the mouse eye, would localize to a type of star-shaped glial support cells called astrocytes. During prenatal human development, astrocytes guide endothelial cells into place where they can proliferate and form blood vessels.

Later in life, under certain circumstances, astrocytes will proliferate and can do the same thing, acting as beacons to bring the stem cells to the retinal vasculature. These stem cells were guided by the retinal astrocytes to the vasculature in the back of the eye. There some were incorporated into the vasculature while others took up positions very close to the blood vessels; both were able to survive—in fact, they seemed to be protected from death.

Once at sites of the retinal vasculature, the stem cells would provide a protective effect, rescuing and stabilizing the retinal vessels when they would otherwise degenerate.

Significantly, the injected stem cells protected the retinal neurons from death at the same time. The neuronal protection seemed to be specific for the cones, the types of photoreceptors found predominantly in the human macula, the center of the retina responsible for fine, or reading, vision.

Friedlander and his colleagues investigated the molecular basis of this process. It turns out that these stem cells are loaded with a type of protein known as “heat shock proteins.”

These cells are sort of like firefighters, says Friedlander. They are protected from apoptosis the way that a firefighter is protected from heat and flames by specialized gear. And the stem cells extend their protection to the surrounding cells of the retina, much as a firefighter, by protecting one position, might protect an entire area of a burning building.

Once inside the retina, these stem cells produce their heat shock proteins and probably induce other cells to produce them as well, thus preventing the retinal and vascular degeneration ordinarily observed in this mouse models of retinitis pigmentosa.

The next step, says Friedlander, would be to perform additional preclinical studies aimed at determining dosage and possible toxicities of a treatment based on this research and then taking the approach into clinical trials.

“The clinical paradigm is novel and, frankly, we were very surprised at the results,” says Friedlander. “Our data in two mouse models of retinal degeneration suggest that it may be possible to use autologous bone marrow-derived stem cell grafts to provide a dramatic vasculo- and neurotrophic protective effect in a variety of retinal degenerative diseases including RP and macular degeneration.”

However, warns Friendlander, as encouraging as it is that they were able to achieve the same rescue effect in mice with human bone marrow cells, the technique is still some distance from the clinics. At the moment, Friedlander and his colleagues are continuing their efforts with human bone marrow-derived stem cells.

“These cells are truly remarkable and provide a rationale basis for using vascular reconstructive approaches in the treatment of diseases in which the endogenous vasculature is subject to degeneration or malfunction,” he says. “Since most diseases that cause profound visual loss have abnormalities in the vasculature, the potential clinical application of this approach is quite broad.”

The research article “Rescue of retinal degeneration by intravitreally injected adult bone marrow-derived lineage negative hematopoietic stem cells” was authored by Atsushi Otani, Michael Ian Dorrell, Karen Kinder, Stacey K. Moreno, Steven Nusinowitz, Eyal Banin, John Heckenlively, and Martin Friedlander and appears in the September 15, 2004 issue of the Journal of Clinical Investigation. The JCI article can be accessed at http://www.jci.org.


  1. usha kanoongo usha kanoongo India says:

    What are the latest developments in this area after this report? My 39 year old sister has retinitis pigmentosa and is suffering a lot due to this problem. Is there any hope? Kindly tell along with necessary details.
    Usha Kanoongo

  2. R.Babu R.Babu India says:

    My brother aged 32 is suffering from RP. Kindly inform me how long it will take to cure human beings after the sucess in mice test.

  3. jamie dawson jamie dawson United Kingdom says:

    I have hearing and sight loss due to retinitis pigmentosa, please can you kindly inform me how long for the treatment to get my eyes sight back. I am 49 years old who has suffered since I was 14 years old - why wait more years - getting older - need action now.

    Best Regards Jamie Dawson

    • trenton trenton United States says:

      What strand of retinitis pigmentosa do you have, it is a disease that affects the eyes not your hearing, maybe you have something else wrong, I am 13 and have RP 10 with 60 degrees of vision left and either you have a strand I haven't heard of or maybe you just have more than rp wrong with you, thanks.

  4. B.Muralidharan B.Muralidharan India says:

    I am a high myopic patient from childhood and after IOL surgery in the year 1999, I was feeling slightly comfortable. However my doctor had already told me that I am affected by the macular retinal degeneration for which there is no immediate solution and I was advised to have periodic check up to see that it does not get further deteriorated. With lots of advancements that are taking place in the medical field, I am too curious to hear any development that has come of late to rectify this decease. Please post me of any development in this area.
    With regards,
    B.Muralidharan, Chennai

    • RP
      ammalraaj ammalraaj India says:


      I  am also from Chennai having RP. Have you been able to get some improvement from RP



  5. ashok ashok India says:

    My brother aged 30 is suffering from RP. Kindly inform me how long it will take to cure human beings after the sucess in mice test.

  6. Ivan Ivan United States says:

    I see my dad and I feel extremely devastated knowing that I don't have a cure for him. I pray for him and everyone dealing with this. We fought this and have overcome some of the emotional stress. My dad is not giving up, we are not giving up, and hoping for the best. Regards to all.

    Thank You
    San Diego, California  

  7. bhumi patel bhumi patel India says:

    my husband aged 39 years old is suffering from R.P.  is there any hope for treatment of this?

  8. mohamed mohamed Egypt says:

    Dear doctors, I am 46 years old and was born with retinitis pigmentosa. Now I suffer from night blindness and narrow vision. I have tried many treatments like ozone, acupuncture, herbs, cupping therapy, current stimulation and last year I had an operation in Moscow but I got worse. Is there any hope to improve my case?
    Best regards
    Mohamed Mostafa
    Tel 0020101750813

  9. Dibyendu Mondal Dibyendu Mondal India says:

    My two brother in laws are suffering from RP. One of them is 40 Years who completely lost his vision and another one (38 years old) who almost lost his vision. But my wife is completely fine till now. Is there any hope for my two brother in law? And is there any chances to effect my baby as my wife is pregnant now.

  10. Preetham Preetham   says:

    Hi All, I want to know if there is any hope for people suffering from Retina Pigmentosa, my mother and two of her brothers are suffering from this disease. Please let me know if anyone has come to know about the treatment.

  11. Peter Almeida Peter Almeida Australia says:

    Come on the world please get the cure for retinitis pigmentosa as soon as possible, my wife and a lot of people are going blind and it's sad...very sad indeed, I pray that a cure will be available soon. If there is anyone out there who can at least improve one's vision please help me to cure my wife of this terrible disease.

    • Mohammed Mohammed U.A.E. says:

      My Friend, I am also desperately praying to God for medicine. Looking at the current technology, very soon we can hear good news.
      Don't loose hope. Be patient and support your wife in all aspects.
      If you come across any progess plese update me. Good Luck.

    • Silky Silky India says:

      Hi Peter Almeida, this is Silky. I just wanna tell you there is a treatment for retinitis pigmentosa in kerala. Hospital name is Sreedhareeyam "http://www.sreedhareeyam.com/" this is link you can see. My sister is also suffering from same disease though she is having little bit of profit. But some people is totally cured there. You must go there with your wife. This may help you.
      I"ll pray for your wife. If you come across any progess please update me. Good Luck.

  12. S.M. Agarwal S.M. Agarwal India says:

    Hi All, I want to know if there is any hope for people suffering from Retina Pigmentosa, my wife is suffering from this disease. Please let me know if anyone has come to know about the treatment.

  13. Kathryn Gustafson Kathryn Gustafson United States says:

    Since the 1970's, I have been hearing of this cure, and that cure, just on the horizon. The money that is being spent to fund all of these different testings is astronomical. All end up with the same end: Nothing. Usually, you don't even hear an update on what the outcome was (why it was vacated, etc). How disheartening it is to keep seeing this year after year, decade after decade, as your sight slowly fades away. I used to go to clinical testings in Chicago to help these scientists, but I quit doing that simply because I never received an outcome report, nor did I ever see anything come out of the studies. Call me cynical, but this had me on the edge of my seat reading this article. I kept thinking "how soon can I be there to volunteer". Then to realize the date was from 2004 and so many people have been on here asking for an update.

    Same old news, different decade. It is very frustrating! Why don't they just take all of that money, split it amongst the ones that are suffering and we'll just let you know plain and simple: there is no cure and probably never will be.

  14. Vicky Vicky India says:

    Hey all!!! as you all i am also quite hoping for the cure very soon and wish all th best to all till then...@ Kathryn:: i just would like to recall a saying at your comment " Mostly people fail because they dont realize how close they were to success when they quit" Also i can understand how depressing it is to hear the arrival of treatment and suddenly realizing its still on animals and would take another 5 years to come and so on again and again...but THATS LIFE...and try living it as we people are living it on the edge..so CHEERS!!! very soon we all would be writing comments here again telling stories of our successful treatment and seeing the stars again :0)

  15. Joan Joan Philippines says:

    I have a fiance' who has a same problem... I felt pity everytime that people where getting mad at him when he hit them accidentally. He's the type of person who has so many dreams and goal in life.. He has so may plan, but he think that his time where almost running out due to his illness. I hope and I pray that they could find a cure for this kind of disease. I love my fiance', I dont want him to suffer so much.

  16. M S Sodhi M S Sodhi India says:

    I have 19 yrs old daughter who is suffering from retinis pigmentosa for the last 9 yrs.  She is studying in first year of graduation .  She can see in daylight only but in the night she cant.  I hope a succesful treatment will comeout for all the people suffering from this disease and they could enjoy their life as other people.

  17. shailesh joshi shailesh joshi India says:

    I am 36 years old retinitis pigmentosa patient. I hav lost my vison. I am a school leacturer. I reach aiims delhi for steam cell treatment but I get no success. What cure is possible. Please inform me?

  18. Meska Memedov Meska Memedov Denmark says:

    I am 40 years old an is suffering with the diseased stem cells and retinitis pigmentosa for the last 15 years. I want to know if there's a cure, and I want to participate in research. My eye doctor told me to join here, so I hope there will come some answer to me quickly!

  19. Gerard de Vres Gerard de Vres Netherlands says:

    I've found articles from the 1980's with the same sort of promise of cures that do not materialize besides apparently in mice.
    Let us be clear RP is bad, and it's terrible to slowly lose your eyesight.

    However never forget that the day you heard you had RP was also the day you could have heard you had terminal cancer. Even though we all hope and pray for a cure we shouldn't forget how much worse off we all could be.

    Listen to your eye doctor they will usually be more informed on possible treatment and research into possible cures and how many hurdles are left to be taken before it can be performed on humans in practice.

    They will tell you that cures are not on the horizon, they will tell you to seek support from RP support groups and low vision specialists that can help you make life more tolerable with your disability.

    Enjoy whatever sight you have left and enjoy the life and the time you are given.

    Don't lose hope but don't expect a cure anytime soon.

  20. praveen praveen India says:

    Hi This is Praveen. My sister is suffering from Retina Pigmantosa since her childhood. Now she is in a dangerous position. She is feeling that she is loosing her eye sight day by day. We have treated her in Chennai (Kasyap eye hospital) but they told that until now there is no treatment for RP. Tell me what is next for my Sister? Is any hope in future will arise? I am waiting for better response ....thanks

  21. Silky Silky India says:

    Hi Praveen, Can you give me your mail id so that i'll give you more information on that.

    • Preetham Preetham India says:

      Hey can you please give more information on RP, even my Mom and 2 of my uncles are suffering from RP. Please help me in regarding this.

  22. Phil Phil United States says:

    I have a daughter who has retinitis pigmentosa. She is 22 years old and has suffered for too long all these years. Kindly feedback me with hopes.

  23. dr.ahmad tabl dr.ahmad tabl Egypt says:

    Sadly no definite treatment for RP till now ... studies for Stem cell transplantation and implanting micro chips called "Encapsulated cell technology " to enhance vision and retinal photoreceptors
    all we have to provide today is Magnification glasses " enlarge field of view " and vit.A supplementation 15000 u\day to just slow the progress of disease till new treatment exists

  24. David Terry David Terry United States says:

    I am legally blind in my left eye but still have central vision in right eye. Please let me know when treatment is available.

  25. manjot singh manjot singh India says:

    My maternal uncle is suffering from the disease of the retina pigmentosa. We have tried a lot and had taken consultation of the every renown doctor but we have nt got any result. My maternal uncle vision is getting dim slowly and slowly and we are helpless. we have consulted with everywhere but we are not getting any cure for it. please if somebpdy have any suggestion, latest cure or treatment for it. Then please inform me. I shall be very obliged to You.

  26. yogesh rai yogesh rai India says:

    Hi, my name is yogesh and i am also suffering 4m d same diseease - stay in touch my email id is [email protected]

  27. Ashutosh Ashutosh India says:

    Dear Dr.,My father has RP from age 15, he is currently 66. He has been recently getting treatment from Aurvedic methodology from last 1 year from India. There is 10% improvement, however the improvement is slow.

    My wife is expecting a baby & I have been through a presentation which talks about preserving the stem cells through the umbilocal cord and also the umbilocal blood as these stem cells can treat lot of diseases.

    Do let me know if the babys stem cells can be used for my dads benefit in treating RP.


    M: +91-9216524321

  28. PSKI PSKI United States says:

    Can someone explain how we can overcome all of the complexities of space travel, nano technology, etc and yet we have no cure (let alone a viable treatment) for RP? As an engineer (mechanical) I feel that if enough effort is given, we as humans have and can overcome major obsticles in order to improve the quality of life of people living with RP such as my deaf and almost blind mother is today. So what gives? Please explain if you have answers.  

  29. Dan Dan India says:

    Dear all, Honestally there is no cure for  Retinitis Pigementosa. My Brother is a patient for the last three years there in Sreedhariyam. He hasn't got any cure. Gradually he is getting blind now..They all are exlpoting human and making money. thats all. Please do onething pray to develop stem cell therapy for this. That is the only hope.  

  30. D.P.PANDEY D.P.PANDEY India says:

    What are the latest developments in this area after this report? My 35 year old sister has retinitis pigmentosa and is suffering a lot due to this problem. Is there any hope? Kindly tell along with necessary details.

The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
Post a new comment

While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. We do not provide medical advice, if you search for medical information you must always consult a medical professional before acting on any information provided.

Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles.

Please do not ask questions that use sensitive or confidential information.

Read the full Terms & Conditions.

You might also like...
Psilocybin and undisclosed ingredients found in popular mushroom gummies