ALS Research Forum | To Evaluate Stem Cell Therapies, Think … – ALS Research Forum

Testing stem cell therapies unilaterally?A side-by-side comparison of strength of key muscles may enable scientists to evaluate stem cell therapies for the disease. The approach aims to control for the variability of the disease internally, without historical cohorts and/or the use of a placebo (see Donofrio and Bedlack, 2011; Glass et al., 2016).The biceps and triceps appear to be most reliable muscles to monitor progression in people with ALS according to this analysis (Rushton et al., 2017). [Neural progenitor cells. Courtesy of Nature Cell Biology. Reproduced with permission.]

Motor neurons degenerate in ALS. Why these cells are destroyed remains unclear. Therefore, how to slow or stop this destruction of motor neurons in ALS remains an open question.

In the meantime, a growing number of scientists are turning to stem cells in hopes to promote motor neuron survival in people with ALS and/or reduce their toxicity (see December 2015 conference news). But how to evaluate these strategies in the clinic remains hotly debated.

Now, a research team at Cedar Sinai Medical Center in Los Angeles, California reports that an emerging outcome measure, which involves monitoring muscle strength, may facilitate the evaluation of stem cell therapies for the disease (Rushton et al., 2017). The study, led by Clive Svendsen, found that functional decline of key muscles on the left and right side of people with ALS progressed at a similar rate. The results suggest that at least some stem cell therapies could be evaluated unilaterally by comparing the strength of muscles on the treated and untreated side for each of these muscle groups.

This side-by-side comparison, according to a subsequent power analysis, may enable clinicians to evaluate stem cell therapies for ALS in a smaller sample size without the need for sham surgeries and/or placebo injections.

This unilateral approach is emerging as an alternative to evaluate a growing number of potential neuroprotective strategies for neurodegenerative diseases including ALS (see NCT02943850, NCT02478450; Glass et al., 2016).

The study is published on June 9 in Neurology.

The retrospective analysis, performed in collaboration with Cedar Sinais Robert Baloh, studied the rates of decline of 6 upper and lower muscle groups in nearly 750 people with ALS determined by fixed dynamometry. These longitudinal datasets, previously collected by physical therapist Pat Andres and colleagues, now at Massachusetts General Hospital, capture the decline in strength of key muscles in people with ALS during at least a 16-month period measured by either the TUFTS Quantitative Neuromuscular Exam (TQNE) or more recently, the Accurate Test of Limb Isometric Strength (ATLIS) system (Andres et al., 1986; Shields et al., 1998; Andres et al., 2012.

Analyzing therapies by hand. Meanwhile, Biogen scientists in Cambridge, Massachusetts are turning to hand-held dynamometry to evaluate potential therapies for ALS. The emerging strength-based measure highly correlates with the progressive loss of motor function (ALS-FRS-R) and breathing capacity (FVC) according to a retrospective analysis of 924 people with ALS presented at the 2017 meeting of the American Academy of Neurology (see May 2017 news). And, according to a subsequent side-by-side comparison, these musclesdecline at similar rates. [Image: Douma et al., 2014 under CC BY 2.0 license.]

The study builds on previous work, led by Barrow Institutes Jeremy Shefner in Phoenix, Arizona and Biogens Toby Ferguson in Cambridge, Massachusetts, which found that monitoring the strength of key muscles using hand-held dynamometry is a reliable and reproducible approach to measure progression of ALS in a clinical setting and thereby, may facilitate the evaluation of potential therapies (see May 2017 conference news; Shefner et al., 2014).

Now, Svendsens team is gearing up to evaluate their potential stem cell therapy for ALS. The strategy uses genetically engineered neural progenitor cells (NPCs) to deliver GDNF into the CNS in hopes to protect motor neurons in people with the disease (see April 2017 news; Gowing et al., 2014). The approach is at the phase 1 stage. Stay tuned.

Featured Paper

RushtonDJ, Andres PL, Allred P, Baloh RH,SvendsenCN. Patients with ALS show highly correlated progression rates in left and right limb muscles. Neurology. 2017 Jun 9. [PubMed].

References

ShefnerJM, Liu D, Leitner ML, Schoenfeld D, Johns DR, Ferguson T, Cudkowicz M.Quantitativestrengthtesting in ALS clinical trials. Neurology. 2016 Aug 9;87(6):617-24. [PubMed].

Andres PL, Skerry LM, Munsat TL, Thornell BJ, Szymonifka J, Schoenfeld DA, Cudkowicz ME. Validation of a new strength measurement device for amyotrophic lateral sclerosis clinical trials. Muscle Nerve. 2012 Jan;45(1):81-5. [PubMed].

Andres PL, Hedlund W, Finison L, Conlon T, Felmus M, Munsat TL.Quantitative motor assessment in amyotrophic lateral sclerosis. Neurology. 1986 Jul;36(7):937-41.[PubMed].

Glass JD, Hertzberg VS, Boulis NM, Riley J, Federici T, Polak M, Bordeau J, Fournier C, Johe K, Hazel T, Cudkowicz M, Atassi N, Borges LF, Rutkove SB, Duell J, Patil PG, Goutman SA, Feldman EL. Transplantation of spinal cord-derived neural stem cells forALS: Analysis of phase 1 and 2 trials. Neurology. 2016 Jul 26;87(4):392-400.[PubMed].

Gowing G, Shelley B, Staggenborg K, Hurley A, Avalos P, Victoroff J, Latter J, Garcia L, Svendsen CN. Glial cell line-derived neurotrophic factor-secreting human neural progenitors show long-term survival, maturation into astrocytes, and no tumor formation following transplantation into the spinal cord of immunocompromised rats. Neuroreport.2014 Apr 16;25(6):367-72. [PubMed].

Further Reading

Atassi N, Beghi E, Blanquer M, Boulis NM, Cantello R, Caponnetto C, Chi A, Dunnett SB, Feldman EL, Vescovi A1, Mazzini L; attendees of the International Workshop on Progress in Stem Cells Research for ALS/MND. Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials? Cytotherapy.2016 Dec;18(12):1471-1475. [PubMed].

Donofrio PD, Bedlack R. Historical controls in ALS trials: a high seas rescue? Neurology. 2011 Sep 6;77(10):936-7. [PubMed].

clinical trial clinical trial design disease-als gdnf neuralstem neuroprotection stem cell topic-clinical topic-randd

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Ebensburg native celebrates 5-year cancer survival at center’s event – TribDem.com

EBENSBURG About five years ago, a very physically active Dale Lauer became tired while playing a match of tennis with a friend.

Something just wasnt right.

His wife, Cheryl, immediately made him a doctors appointment. Blood tests revealed Lauer had leukemia.

Today, Lauer, an Ebensburg native, is cancer-free.

Last week, he joined more than 135 fellow five-year cancer survivors at Midwestern Regional Medical Center in Zion, Illinois, to participate in Cancer Treatment Centers of Americas 29th annual Celebrate Life event.

Celebrate Life is an event that brings together cancer survivors and caregivers for a day of support, celebration and encouragement for those who arestill fighting their battles with cancer.

Lauer said a friend of the family connected him with the Cancer Center in Zion, where he completed two unsuccessful rounds of induction chemotherapy.

On Oct. 3 of that year, Lauer had a stem-cell transplant a procedure with a success rate of less than 10 percent that allowed him to be cancer-free ever since.

It was extremely aggressive treatment, he recalls.

On June 9, Lauer, his wife and three sons Joel, Ryan and Brock arrived with the other survivors at the Midwestern Regional Medical Center, walking down a red carpet lined with family, friends, caregivers and hospital care team members.

After the red carpet walk, Lauer and his family were part of a commemorative tree-planting ceremony.At each of the 29 annual Celebrate Life events, a tree is planted in honor of each five-year cancer survivor in attendance.

After five years of survivorship, Im back to doing the things I love, Lauer said. By staying positive, paying attention to my health and maintaining a will to live, Ive returned to riding bikes, playing tennis and surrounding myself with my loving family and community.

Five years ago, this amazing group of cancer survivors came to Cancer Treatment Centers of America with a great sense of hope, searching for answers and cancer care that fit their specific needs, said Scott Jones, president and CEO of Cancer Treatment Centers of America at Midwestern.While everyones journey and experience is unique, we honor and respect the strength and perseverance it takes for them to be here.

Lauer said his longest say at the Cancer Center in Zion was about four months, during which every staff member treated him like family.

Its not a hospital, its heaven on earth, he said.

When you walk in the door, theres people in there who want you to have the best day of your life. Its really like a family.

Lauer is also involved with the Cancer Fighters program at Cancer Treatment Centers of America, where he said hes thrilled to share my story.

Cancer Fighters allows survivors and caretakers such as Lauer and his wife to answer questions and share their journey with recently diagnosed patients seeking treatment at Cancer Treatment Centers across the country.

Lauer especially creditsthe support of family, friends, neighbors, doctors and nurses provided during his battle against leukemia.

The miracles that came my way were just astronomical, he said.

Jocelyn Brumbaugh is a reporter for the Tribune-Democrat. Follow her on Twitter @JBrumbaughTD.

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Hyderabad team grows miniature eyes using stem cells – The Hindu


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Hyderabad team grows miniature eyes using stem cells
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The iPS cells are produced by genetically manipulating human skin cells to produce embryonic-like stem cells that are capable of forming any cell types of the body. Small portions of the corneal tissue were separated from the miniature eyes and used ...

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Hyderabad team grows miniature eyes using stem cells - The Hindu

Low-cost biochip isolates cells for clinical diagnosis [PreClinical] – 2 Minute Medicine

1. Researchers manufactured a microfluidic biochip, using an inkjet printing system to apply conductive nanoparticles to a polymer substrate.

2. The biochip used an electric field to separate and capture cell populations, allowing for downstream characterization and further analysis.

Evidence Rating Level: 2 (Good)

Study Rundown: Some infectious diseases and cancers can be detected early through the identification of rare cell populations. The sorting and identification of these cells currently require bulky and expensive equipment, preventing their effective use in areas unable to obtain these technologies. The goal of this study was to develop a low-cost, reusable, and effective cell separation platform that could be used for diagnostics in resource-limited areas.

The biochip was designed to have three components: a reusable polyethylene terephthalate substrate with inkjet-printed conductive nanoparticles, a disposable microfluidic platform made of polydimethylsiloxane, and an insulating barrier. The biochip induced dipole moments, resulting in a dielectrophoresis (DEP) force that moved varying cell types based on differences in size and dielectric properties. This allowed for the effective separation and storage of different cell populations. A mixture of breast adenocarcinoma cells, yeast cells, and polystyrene microspheres was used to test the biochip. The three particle types were collected with high separation efficiency. The cells were viable after being on the biochip, indicating they could be used for downstream analyses.

Future work must evaluate the biochip using more clinically relevant cell mixtures. However, the biochip demonstrates potential for diagnostics and research studies on rare cell populations. Without the need for clean rooms or time consuming processing, manufacturing the biochip only requires vector-drawing software and inkjet printing technology. With a production time of around 20 minutes and a materials cost of $0.01 per chip, this biochip could be feasibly produced in developing and low-income areas. Not only can clinical samples be reused after processing on the biochip, but the chip itself is also a reusable platform. This technology could enable faster diagnostic capabilities and early detection of rapidly progressing conditions.

Click here to read the study in PNAS

Relevant Reading: A microfluidic biochip for complete blood cell counts at the point-of-care

In-Depth [in vitro study]: To collect single cells, the biochip used contactless dielectrophorectic-traps and an array of facing electrodes. When particles were introduced to the chip, each cell experienced negative DEP forces that trapped it in a chamber within the electrical field. Once a cell was trapped in a chamber, no other cells could enter.

To optimize the parameters of the biochip, polystyrene microspheres were put in the chip and subjected to various voltages and signal frequencies to determine their effects on the resulting velocity and DEP force. Flow rates were adjusted to optimize the capture efficiency, with higher flow rates resulting in a 510% drop in the efficiency of capturing the microspheres. These parameters were then validated using a breast adenocarcinoma cell line (MDA-MD-231) and yeast cells.

Cells were isolated from the chip and assessed for viability. A 1.5-fold increase in transformation efficiency was noted in the yeast cells, confirming the safety of this technology. A mixture of MDA-MD-231 cells, yeast cells, and streptavidin-coated polystyrene microspheres was put into the biochip. Because each of these particles have different polarization properties, they could be separated by the biochip. Separation efficiency was found to be 79, 88, and 86% for the breast cancer cells, yeast cells, and microspheres, respectively.

Image: PD

2017 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.

2 Minute Medicines The Classics in Medicine: Summaries of the Landmark Trials is available now in paperback and e-book editions.

This text summarizes the key trials in:General Medicine and Chronic Disease, Cardiology, Critical and Emergent Care, Endocrinology, Gastroenterology, Hematology and Oncology, Imaging, Infectious Disease, Nephrology, Neurology, Pediatrics, Psychiatry, Pulmonology, and Surgery.

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Low-cost biochip isolates cells for clinical diagnosis [PreClinical] - 2 Minute Medicine

Israeli Scientists: Stem Cell Therapy Not Good for All Heart Patients – The Jewish Press – JewishPress.com

Photo Credit: Nati Shohat / Flash 90

Patients with severe and end-stage heart failure have few treatment options available to them apart from transplants and miraculous stem cell therapy. But a new Tel Aviv University study has found that stem cell therapy may in fact harm patients with heart disease.

The research, led by Prof. Jonathan Leor of TAUs Sackler Faculty of Medicine and Sheba Medical Center and conducted by TAUs Dr. Nili Naftali-Shani, explores the current practice of using cells from the host patient to repair tissue and contends that this can prove deleterious or toxic for patients. The study was recently published in the journal Circulation.

We found that, contrary to popular belief, tissue stem cells derived from sick hearts do not contribute to heart healing after injury, said Prof. Leor. Furthermore, we found that these cells are affected by the inflammatory environment and develop inflammatory properties. The affected stem cells may even exacerbate damage to the already diseased heart muscle.

Tissue or adult stem cells blank cells that can act as a repair kit for the body by replacing damaged tissue encourage the regeneration of blood vessel cells and new heart muscle tissue. Faced with a worse survival rate than many cancers, a number of patients with heart failure have turned to stem cell therapy as a last resort.

But our findings suggest that stem cells, like any drug, can have adverse effects, said Prof. Leor. We concluded that stem cells used in cardiac therapy should be drawn from healthy donors or be better genetically engineered for the patient.

Hope for improved cardiac stem cell therapy

In addition, the researchers also discovered the molecular pathway involved in the negative interaction between stem cells and the immune system as they isolated stem cells in mouse models of heart disease. After exploring the molecular pathway in mice, the researchers focused on cardiac stem cells in patients with heart disease.

The results could help improve the use of autologous stem cells those drawn from the patients themselves in cardiac therapy, Prof. Leor said.

We showed that the deletion of the gene responsible for this pathway can restore the original therapeutic function of the cells, said Prof. Leor. Our findings determine the potential negative effects of inflammation on stem cell function as theyre currently used. The use of autologous stem cells from patients with heart disease should be modified. Only stem cells from healthy donors or genetically engineered cells should be used in treating cardiac conditions.

The researchers are currently testing a gene editing technique (CRISPER) to inhibit the gene responsible for the negative inflammatory properties of the cardiac stem cells of heart disease patients. We hope our engineered stem cells will be resistant to the negative effects of the immune system, said Prof. Leor.

Meanwhile, for those unable to profit from stem cell therapy, researchers at Ben Gurion University of the Negev (BGU) have developed a revolutionary new drug that may reverse the damage and repair the diseased heart.

The newly developed drug is a polymer which reduces the inflammation in cardiovascular tissue and stops plaque build-up in arteries. Then it goes one step further and removes existing plaque in the heart, leaving healthy tissue behind.

Professor Ayelet David, a researcher at BGU revealed the drug might also help people suffering from diabetes, hypertension and other conditions associated with old age.

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Israeli Scientists: Stem Cell Therapy Not Good for All Heart Patients - The Jewish Press - JewishPress.com

Therapists say Ryan Custer moved two fingers on his right hand – WRGT TV Fox 45

WSU Basketball players sending well wishes to injured Ryan Custer (Photo courtesy Assistant Coach of WSU basketball Nick Goff)

CHICAGO (WKEF/WRGT) - A Wright State basketball player who fractured his C-5 vertebrae at an off-campus party in late April was able to move two of his fingers on his right hand, according to his therapist.

Ryan Custer's mother posted the update on his recovery Facebook page, saying therapists saw him move his ring and little fingers, but his parents have yet to see the movement themselves.

Custer will also be moved to Craig Hospital in Denver, the family's choice rehab facility, after his time at Shirley Ryan Ability Lab in Chicago. The family said Craig Hospital did not take patients on anti-rejection medication for stem cell, so it had to be temporarily ruled out while he underwent his experimental surgery and follow-up treatment.

They said therapy will be more individualized and Custer's workouts will come in a gym-like facility after the move, which works for him since he grew up playing on sports teams.

Custer also reportedly had another bout of C. diff, which was worse for him this time around, and has been having a lot of discomfort.

He's set to go to Rush on June 20 for follow-up before flying out to Denver the day after.

Custer was originally injured when he jumped into a makeshift pool and hit his head off someone's knee. His team has been out to Chicago to see him.

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Therapists say Ryan Custer moved two fingers on his right hand - WRGT TV Fox 45

Back from Mexico, Indian Land teen hopes she gave herself the gift of life – The Herald


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Back from Mexico, Indian Land teen hopes she gave herself the gift of life
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Later this month, Grace will go Houston to see Dr. Ian Butler, a pediatric neurologist who's been treating her since late last year, and Dr. Stanley Jones. They'll evaluate her condition since the first stem cell treatment, and hopefully prescribe ...

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Back from Mexico, Indian Land teen hopes she gave herself the gift of life - The Herald

In a first, Mumbai doctors use dad’s cells to fight blood disorder – Times of India

MUMBAI: Three-year-old Kinaya Shah was diagnosed with thalassemia at the tender age of three months and has been undergoing regular blood transfusions ever since. The only cure for thalassemia is a bone marrow transplant (BMT), a form of stem cell therapy. Typically, the donor of the stem cells would be a sibling of the patient such that the stem cells of the donor are a near perfect match to those of the patient. The only complication was that Kinaya was a lone child.

So, city doctors in a first used stem cells donated by Kinaya's father - who was only a half or haploidentical match - to cure the child of the blood disorder. "We went to Vellore, Bangalore and Pune but no one was willing to do the transplant without a full match donor," said Kinaya's parents, Aneri and Shripal Shah. They approached Dr Santanu Sen at the Kokilaben Dhirubhai Ambani Hospital, Andheri, in October of 2016, after reading about a similar surgery that he had performed.

While haploidentical bone marrow transplants are carried out to cure leukaemia, it has only been done about half a dozen times for thalassemia in a couple of Indian cities. ``Haploidentical transplants are gradually increasing because of better techniques,'' said Dr Sen.

Dr Sen has completed 36 BMTs in the last two years, of which 12 were haploidentical donors. ``But this is the first time that a haploidentical transplant has been done in western India to cure thalassemia,'' he said.

Chennai-based haematologist Dr Revathy Raja said that there is a 85% chance of cure in thalassemia with a fully matched donor. ``The success rate falls to 70% with a half-match or haploidentical donor. We have hence not started it at our Chennai centre. Hopefully, techniques will further improve in the coming years,'' she said.

In order to perform the surgery, Dr Sen conditioned Kinaya's immune system over three months, with slight chemotherapy, to increase the chances of her body accepting the graft. "We found that her father's stem cells were a 70% match through genetic tests and decided to use them for the transplant. In the case that the graft was rejected we froze a couple of Kinaya's stem cells as insurance. The positive is that children have lower rejection rates for foreign cells as they have barely developed any active immunity," said Dr Sen. "BMT is the most viable treatment to cure thalassemia, the only barrier thus far was the necessity of a full match donor," he added.

However, Vinay Shetty of NGO Think Foundation, which works for thalassemia patients, said that it would be prudent to wait for a statistically significant number of successful halploidentical transplants before recommending it to all patients.

Post the three months of conditioning, stem cells were collected from her father's bone marrow and the transplant was performed on May 10, 2017. After several tests to confirm that the graft was accepted, Kinaya was finally discharged from the hospital on June 13.

"The future of thalassemia treatment probably lies in gene therapy, but at the moment, haploidentical transplants have made BMT much more accessible," said Dr Sen, adding that he has two more cases such as Kinaya lined up. Kinaya is expected to be completely independent of medication and any trace of thalassemia in the coming six months.

What is Thalassemia?

Thalassemia is a genetic blood disorder when the body produces abnormal hemoglobin. Patients require regular blood transplant and well as dietary control to ensure that blood irons level stay suppressed.

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In a first, Mumbai doctors use dad's cells to fight blood disorder - Times of India

Stem Cell Therapy for Glaucoma – Are We There Yet? – Newswise (press release)

Newswise There is great interest among glaucoma patients, scientists and doctors alike, in discovering regenerative therapies for the optic nerve and translating them from the laboratory to the clinic and stem cell therapy is one of several promising approaches being studied.

Recently, we heard from a glaucoma patient who enrolled in a patient-funded trial in which the person paid $20,000 and received stem cell injections around one eye. Patient-funded trials are studies in which patients pay to participate. This approach was developed because clinical trials are expensive and funding from traditional sources (such as the NIH, pharmaceutical companies, or private foundations) is decreasing.

Although patient-funded research would appear superficially to provide an avenue for patients to obtain therapies under investigation, it is controversial for a number of reasons, both scientific and ethical. Scientific considerations are deeply concerning: the gold standard for evaluating an experimental treatment is a randomized clinical trial in which participants are randomly allocated to the experimental treatment group or a control group that may not receive any treatment (placebo-controlled study), or may receive standard, approved therapies. Usually, study patients and doctors are both unaware of who receives which treatment and this study design minimizes bias towards a particular treatment.

In contrast, patient-funded trials do not have a control group, since it is extremely unlikely that patients would pay when there is a possibility of not receiving the experimental treatment. A control group is very important to determine if an experimental treatment really has an effect and also to compare the efficacy and risks of the experimental treatment versus other treatments. Although it is common in an early, phase 1 trial to treat perhaps the first 3-12 patients in an open-label, non-randomized study, by phase 2 trials, a randomized, masked design is ideal.

Even more concerning are the ethical considerations. These include disparity in access to the treatment, and risk of exploitation of vulnerable patients who have exhausted all treatment options and may be willing to undergo an unproven treatment at any cost. In addition, a reported lack of proper oversight and monitoring for patient-funded trials implies the possibility that these may be thinly-veiled attempts to make money by the treating clinic or physician before proper FDA approval of new treatment methods.

Since a gold standard research study is not always feasible, decisions regarding the benefits and risks of a particular treatment may have to be made with the evidence at hand. Given these considerations, we consulted Dr. Jeffrey Goldberg regarding stem cell therapy for glaucoma. Dr. Goldberg is a leading expert in therapies to regenerate the optic nerve and is part of the Catalyst for Cure team. His laboratory is developing novel stem cell approaches for glaucoma, and working steadily towards a translational program to bring discoveries out of the laboratory and into human testing once safety and efficacy in pre-clinical models is established.

Q: Dr. Goldberg, how might stem cells be helpful for patients with glaucoma?

A: Stem cells may be helpful for patients with glaucoma in different ways. Stem cells can be turned into trabecular meshwork cells in the front of the eye and transplanted in such a way as to lower eye pressure. This is an interesting approach but is not fundamentally about vision restoration.

For protecting or restoring vision, we really need to talk about stem cells in the back of the eye, at the retina. There, stem cells may have two positive effects. First, early in the disease, they may protect retinal ganglion cells from degenerating providing a neuroprotective effect. Later in the disease when patients have lost considerable numbers of retinal ganglion cells and optic nerve axons, and have thereby lost considerable vision, stem cells may be useful to replace lost ganglion cells and restore the connections from the eye to the brain. This last approachregrowing optic nerve fibers back to the brainhas been the most challenging but its also the most exciting.

What is the current status of research on stem cell therapy for glaucoma?

Our laboratory and a number of other laboratories have made considerable progress on the two main fronts of bringing stem cell therapy to optic nerve restoration for glaucoma. First, we and others have discovered molecular pathways that can be used to coax stem cells to turn into neurons that look and act like real retinal ganglion cells. This will allow us to turn large numbers of stem cells into retinal ganglion cells for cell replacement therapy. Second, we are just beginning to make progress in transplanting retinal ganglion cells into the retina in pre-clinical models, to study their integration into the adult retina, how they respond to light and grow back down the optic nerve to the brain. Together these advances have brought us to an exciting moment in stem cell research for optic nerve restoration in glaucoma.

Are you aware of any studies in which stem cell therapy stabilized or reversed vision loss from glaucoma?

Stem cells have not yet been properly tested in patients with glaucoma to look for their ability to stabilize or reverse vision loss. The careful move from the laboratory to clinical testing is still ahead of us, although with the intellectual energy and resources ready to deploy, such proper testing may not be far off.

If your family member had vision loss from glaucoma, would you recommend stem cell therapy at this time?

I am often asked by my patients if they should sign up for a patient-funded trial for stem cells for glaucoma, and I am in the habit of counseling against this. I am not aware of any properly designed stem cell trials for glaucoma with well-tested cell therapies being moved to human testing at this time, but I do think these will come.

Are there any risks or complications reported with stem cell therapy for glaucoma?

Indeed the risks for undergoing stem cell injections in any trial could be significant. Risks of infection, inflammation, and more severe vision loss will always be present; we are publishing a paper about 3 patients in the U.S. who participated in a patient-funded trial and lost significant vision due to severe inflammation in their eyes called endophthalmitis. These 3 unfortunate patients point to the importance of a cell therapy first undergoing proper testing in pre-clinical models before moving to human testing. Then, with properly designed and sequenced trials, I believe cell therapies can be safely tested in the eye as with the rest of the body. Indeed there are a number of cell therapies for macular degeneration already in human testing with a reassuring safety record thus far.

What type of study would you design for evaluating stem cell therapy with glaucoma?

After demonstrating safety and efficacy of a cell therapy product in pre-clinical models, a small pilot study designed to assess for safety after injection in humans and analysis of the results should be the first step. After this, a move to a randomized trial with a control group and masked observers will be best to assess efficacy in phase 2 and eventually phase 3 trials.

Article by Sunita Radhakrishnan, MD, Jeffrey L. Goldberg, MD, PhD, and Andrew Iwach, MD.

For additional information on this topic, following are links to related articles from the American Academy of Ophthalmology:

Unregulated Stem Cell Treatments Can Be Dangerous [June 6, 2017]

American Academy of Ophthalmology Statement on Stem-Cell Therapy for Treating Eye Disease [March 20, 2017]

Unapproved stem cell treatment blinds 3 patients [March 17, 2017]

Stem Cell Therapy for Eye Disease: What You Need to Know [June 24, 2016]

Intraocular Stem Cell Therapy [June 2016]

Excerpt from:
Stem Cell Therapy for Glaucoma - Are We There Yet? - Newswise (press release)

On the cusp of payoffs for patients, stem cell therapy faces threat from unregulated clinics – STAT

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On the cusp of payoffs for patients, stem cell therapy faces threat from unregulated clinics - STAT