The world of stem cell research lost a good friend this weekend. Eli Broad, a generous supporter of science, education and the arts, passed away at the age of 87.
Eli came from humble origins, born in the Bronx to an immigrant father who worked as a house painter and a mother who was a seamstress. He went to Michigan State University, working a number of jobs to pay his way, including selling women’s shoes, working as a door-to-door salesman for garbage disposal units, and delivering rolls of film to be developed. He graduated in three years and then became the youngest person ever to pass the CPA exam in Michigan.
He started out as an accountant but quickly switched to housing and development and was a millionaire by the time he was 30. As his wealth grew so did his interest in using that money to support causes dear to him and his wife Edythe.
With the passage of Proposition 71 in 2004 Broad put up money to help create the Broad Stem Cell Centers at UCLA, UC San Francisco and the University of Southern California. Those three institutions became powerhouses in stem cell research and the work they do is a lasting legacy to the generosity of the Broads.
“Science has lost one of its greatest philanthropic supporters,” says Jonathan Thomas, PhD, JD, Chair of the CIRM Board. ” Eli and Edye Broad set the table for decades of transformative work in stem cell and gene therapy through their enthusiastic support for Proposition 71 and funding at a critical time in the early days of regenerative medicine. Their recent additional generous contributions to USC, UCLA and UCSF helped to further advance that work. Eli and Edye understood the critical role of science in making the world a better place. Through these gifts and their enabling support of the Broad Institute with Harvard and MIT, they have left a lasting legacy in the advancement of medicine that cannot be overstated.”
“As a businessman Eli saw around corners, as a philanthropist he saw the problems in the world and tried to fix them, as a citizen he saw the possibility in our shared community, and as a husband, father, mentor and friend he saw the potential in each of us.”
Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved new clinical trials for COVID-19 and sickle cell disease (SCD) and two earlier stage projects to develop therapies for COVID-19.
Dr. Michael Mathay, of the University of California at San Francisco, was awarded $750,000 for a clinical trial testing the use of Mesenchymal Stromal Cells for respiratory failure from Acute Respiratory Distress Syndrome (ARDS). In ARDS, patients’ lungs fill up with fluid and are unable to supply their body with adequate amounts of oxygen. It is a life-threatening condition and a major cause of acute respiratory failure. This will be a double-blind, randomized, placebo-controlled trial with an emphasis on treating patients from under-served communities.
This award will allow Dr. Matthay to expand his current Phase 2 trial to additional underserved communities through the UC Davis site.
“Dr. Matthay indicated in his public comments that 12 patients with COVID-related ARDS have already been enrolled in San Francisco and this funding will allow him to enroll more patients suffering from COVID- associated severe lung injury,” says Dr. Maria T. Millan, CIRM’s President & CEO. “CIRM, in addition to the NIH and the Department of Defense, has supported Dr. Matthay’s work in ARDS and this additional funding will allow him to enroll more COVID-19 patients into this Phase 2 blinded randomized controlled trial and expand the trial to 120 patients.”
The Board also approved two early stage research projects targeting COVID-19.
Dr. Stuart Lipton at Scripps Research Institute was awarded $150,000 to develop a drug that is both anti-viral and protects the brain against coronavirus-related damage.
Justin Ichida at the University of Southern California was also awarded $150,00 to determine if a drug called a kinase inhibitor can protect stem cells in the lungs, which are selectively infected and killed by the novel coronavirus.
“COVID-19 attacks so many parts of the body, including the lungs and the brain, that it is important for us to develop approaches that help protect and repair these vital organs,” says Dr. Millan. “These teams are extremely experienced and highly renowned, and we are hopeful the work they do will provide answers that will help patients battling the virus.”
The Board also awarded Dr. Pierre Caudrelier from ExcellThera $2 million to conduct a clinical trial to treat sickle cell disease patients
SCD is an inherited blood disorder caused by a single gene mutation that results in the production of “sickle” shaped red blood cells. It affects an estimated 100,000 people, mostly African American, in the US and can lead to multiple organ damage as well as reduced quality of life and life expectancy. Although blood stem cell transplantation can cure SCD fewer than 20% of patients have access to this option due to issues with donor matching and availability.
Dr. Caudrelier is using umbilical cord stem cells from healthy donors, which could help solve the issue of matching and availability. In order to generate enough blood stem cells for transplantation, Dr. Caudrelier will be using a small molecule to expand these blood stem cells. These cells would then be transplanted into twelve children and young adults with SCD and the treatment would be monitored for safety and to see if it is helping the patients.
“CIRM is committed to finding a cure for sickle cell disease, the most common inherited blood disorder in the U.S. that results in unpredictable pain crisis, end organ damage, shortened life expectancy and financial hardship for our often-underserved black community” says Dr. Millan. “That’s why we have committed tens of millions of dollars to fund scientifically sound, innovative approaches to treat sickle cell disease. We are pleased to be able to support this cell therapy program in addition to the gene therapy approaches we are supporting in partnership with the National Heart, Lung and Blood Institute of the NIH.”
Andy McMahon is one of the most understated, humble and low-key people you are ever likely to meet. He’s also one of the smartest. And he has a collection of titles to prove it. He is the W.M. Keck Provost and University Professor in USC’s departments of Stem Cell Biology and Regenerative Medicine at the Keck School of Medicine, and Biological Sciences at the Dornsife College of Letters, Arts and Sciences, a fellow of the American Association for the Advancement of Science, the American Academy of Arts and Sciences, the European Molecular Biology Organization, and the Royal Society.
Now you can add to that list that Andy is a member of the National Academy of Sciences (NAS). Election to the NAS is no ordinary honor. It’s one of the highest in the scientific world.
In a USC news release Dean Laura Mosqueda from the Keck School praised Andy saying: “We’re delighted that Dr. McMahon is being recognized as a newly elected member of the National Academy of Sciences. Because new members are elected by current members, this represents recognition of Dr. McMahon’s achievements by his most esteemed peers in all scientific fields.”
Not surprisingly CIRM has funded some of Andy’s work – well, we do pride ourselves on working with the best and brightest scientists – and that research is taking on added importance with the spread of COVID-19. Andy’s area of specialty is kidneys, trying to develop new ways to repair damaged or injured kidneys. Recent studies show that between 3 and 9 percent of patients with COVID-19 develop an acute kidney injury; in effect their kidneys suddenly stop working and many of these patients have to undergo dialysis to stay alive.
Even those who recover are at increased risk for developing more chronic, even end-stage kidney disease. That’s where Andy’s work could prove most useful. His team are using human stem cells to create mini artificial kidneys that have many of the same properties as the real thing. These so-called “organoids” enable us to study chronic kidney disease, come up with ideas to repair damage or slow down the progression of the disease, even help improve the chances of a successful transplant if that becomes necessary.
We often talk about the important role that patient advocates play in helping advance research. That was demonstrated in a powerful way last week when the CIRM Board approved almost $12 million to fund a clinical trial targeting a rare childhood disorder called cystinosis.
The award, to Stephanie Cherqui and her team at UC San Diego (in collaboration with UCLA) was based on the scientific merits of the program. But without the help of the cystinosis patient advocate community that would never have happened. Years ago the community held a series of fundraisers, bake sales etc., and used the money to help Dr. Cherqui get her research started.
That money enabled Dr. Cherqui to get the data she needed to apply to CIRM for funding to do more detailed research, which led to her award last week. There to celebrate the moment was Nancy Stack. Her testimony to the Board was a moving celebration of how long they have worked to get to this moment, and how much hope this research is giving them.
Hello my name is Nancy Stack and I am the founder and president of the Cystinosis Research Foundation. Our daughter Natalie was diagnosed with cystinosis when she was an infant.
a rare disease that is characterized by the abnormal accumulation of cystine in
every cell in the body. The build-up of
cystine eventually destroys every organ in the body including the kidneys,
eyes, liver, muscles, thyroid and brain.
The average age of death from cystinosis and its complications is 28
years of age.
children and adults with cystinosis, there are no healthy days. They take
between 8-12 medications around the clock every day just to stay alive –
Natalie takes 45 pills a day. It is a
relentless and devastating disease.
complications abound and our children’s lives are filled with a myriad of
symptoms and treatments – there are g-tube feedings, kidney transplants, bone
pain, daily vomiting, swallowing
difficulties, muscle wasting, severe gastrointestinal side effects and for some
the Foundation in 2003. We have worked
with and funded Dr. Stephanie Cherqui since 2006. As a foundation, our resources are limited
but we were able to fund the initial grants for Stephanie’s Stem Cell studies. When CIRM awarded a grant
to Stephanie in 2016, it allowed her to complete the studies, file the IND and
as a result, we now have FDA approval
for the clinical trial. Your support has changed the course of this
When the FDA
approved the clinical trial for cystinosis last year, our community was filled
with a renewed sense of hope and optimism.
I heard from 32 adults with cystinosis – all of them interested in the
clinical trial. Our adults know that
this is their only chance to live a full life. Without this treatment, they
will die from cystinosis. In every
email I received, there was a message of hope and gratitude.
I received an
email from a young woman who said this, “It’s a new awakening to learn this
morning that human clinical trials have been approved by the FDA. I reiterate
my immense interest to participate in this trial as soon as possible because my
quality of life is at a low ebb and the trial is really my only hope. Time is
And a mom of a 19 year old young man who wants to be the first
patient in the trial wrote and said this, “On the day the trial was announced I started to cry tears of pure
happiness and I thought, a mother somewhere gets to wake up and have a child who
will no longer have cystinosis. I felt so happy for whom ever that mom would
be….I never imagined that the mom I was thinking about could be me. I am so
humbled to have this opportunity for my son to try to live disease free.
My own daughter ran into my arms that day and we cried tears of
joy – finally, the hope we had clung to was now a reality. We had come full
circle. I asked Natalie how it felt to
know that she could be cured and she said, “I have spent my entire life thinking
that I would die from cystinosis in my 30s but now, I might live a full life
and I am thinking about how much that changes how I think about my future. I never
planned too far ahead but now I can”.
As a mother, words can’t possible convey what it feels like to know that my child has a chance to live a long, healthy life free of cystinosis – I can breathe again. On behalf of all the children and adults with cystinosis, thank you for funding Dr. Cherqui, for caring about our community, for valuing our children and for making this treatment a reality. Our community is ready to start this trial – thank you for making this happen.
CIRM will be celebrating the role of patient advocates at a free event in Los Angeles tomorrow. It’s at the LA Convention Center and here are the details. And did I mention it’s FREE!
Tue, June 25, 2019 – 6:00 PM – 7:00 PM PDT
Petree Hall C., Los Angeles Convention Center, 1201 South Figueroa Street Los Angeles, CA 90015
And on Wednesday, USC is holding an event highlighting the progress being made in fighting diseases that destroy vision. Here’s a link to information about the event.
Don Reed has been a champion of CIRM even before there was a CIRM. He’s a pioneer in pushing for funding for stem cell research and now he’s working hard to raise awareness about the difference that funding is making.
In a recent article on Daily Kos, Don highlighted one of the less celebrated partners in this research, the humble rat.
A BETTER RAT? Benefit #62 of the California Stem Cell Agency
By Don C. Reed
When I told my wife Gloria I was writing an article about rats, she had several comments, including: “Oo, ugh!” and also “That’s disgusting!”
Obviously, there are problems with rats, such as
when they chew through electrical wires, which may cause a short circuit
and burn down the house. Also, they are blamed for carrying diseased
fleas in their ears and spreading the Black Plague, which in 1340 killed
half of China and one-third of Europe—but this is not certain. The
plague may in fact have been transmitted by human-carried parasites.
But there are positive aspects to rats as well. For
instance: “…a rat paired with another that has a disability…will be
very kind to the other rat. Usually, help is offered with food,
cleaning, and general care.”—GUIDE TO THE RAT, by Ginger Cardinal.
Above all, anyone who has ever been sick owes a
debt to rats, specifically the Norway rat with that spectacular name,
rattus norvegicus domesticus, found in labs around the world.
I first realized its importance on March 1, 2002,
when I held in my hand a rat which had been paralyzed, but then
recovered the use of its limbs.
The rat’s name was Fighter, and she had been given a derivative of embryonic stem cells, which restored function to her limbs. (This was the famous stem cell therapy begun by Hans Keirstead with a Roman Reed grant, developed by Geron, and later by CIRM and Asterias, which later benefited humans.)
As I felt the tiny muscles struggling to be free,
it was like touching tomorrow— while my paralyzed son, Roman Reed, sat
in his wheelchair just a few feet away.
Was it different working with rats instead of mice? I had heard that the far smaller lab mice were more “bitey” than rats.
Wanting to know more about the possibilities of a “better rat”, I went to the CIRM website, (www.cirm.ca.gov) hunted up the “Tools and Technology III” section, and the following complicated sentence::
“Embryonic stem cell- based generation of rat models for assessing human cellular therapies.”
Hmm. With science writing, it always takes me a
couple of readings to know what they were talking about. But I
recognized some of the words, so that was a start.
“Stemcells… rat models… human therapies….”
I called up Dr. Qilong Ying, Principle Investigator (PI) of the study.
As he began to talk, I felt a “click” of recognition, as if, like pieces of a puzzle, facts were fitting together.
It reminded me of Jacques Cousteau, the great
underwater explorer, when he tried to invent a way to breathe
underwater. He had the compressed air tank, and a mouthpiece that would
release air—but it came in a rush, not normal breathing.
So he visited his friend, race car mechanic Emil
Gagnan, and told him, “I need something that will give me air, but only
when I inhale,”– and Gagnan said: “Like that?” and pointed to a metal
contraption on a nearby table.
It was something invented for cars. But by adding
it to what Cousteau already had, the Cousteau-Gagnan SCUBA (Self
Contained Underwater Breathing Apparatus) gear was born—and the ocean
could now be explored.
Qi-Long Ying’s contribution to science may also be a piece of the puzzle of cure…
A long-term collaboration with Dr. Austin Smith centered on an attempt to do with rats what had done with mice.
In 2007, the Nobel Prize in Medicine had been won by Dr. Martin Evans, Mario Capecchi, and Oliver Smithies. Working independently, they developed “knock-out” and “knock-in” mice, meaning to take out a gene, or put one in.
But could they do the same with rats?
“We and others worked very, very hard, and got nowhere,” said Dr. Evans.
Why was this important?
Many human diseases cannot be mimicked in the
mouse—but might be in the rat. This is for several reasons: the rat is
about ten times larger; its internal workings are closer to those of a
human; and the rat is considered several million years closer (in
evolutionary terms) to humans than the mouse.
In 2008 (“in China, that is the year of the rat,” noted Dr. Ying in our conversation) he received the first of three grants from CIRM.
“We proposed to use the classical embryonic stem
cell-based gene-targeting technology to generate rat models mimicking
human heart failure, diabetes and neurodegenerative diseases…”
How did he do?
In 2010, Science Magazine honored him with
inclusion in their “Top 10 Breakthroughs for using embryonic stem
cell-based gene targeting to produce the world’s first knockout rats,
modified to lack one or more genes…”
And in 2016, he and Dr. Smith received the McEwen Award for Innovation, the highest honor bestowed by the International Society for Stem Cell Research (ISSCR).
Using knowledge learned from the new (and more
relevant to humans) lab rat, it may be possible to develop methods for
the expansion of stem cells directly inside the patient’s own bone
marrow. Stem cells derived in this fashion would be far less likely to
be rejected by the patient. To paraphrase Abraham Lincoln, they would
be “of the patient, by the patient and for the patient—and shall not
perish from the patient”—sorry!
Several of the rats generated in Ying’s lab (to mimic human diseases) were so successful that they have been donated to the Rat Research Resource center so that other scientists can use them for their study.
“Maybe in the future we will develop a cure for some diseases because of knowledge from using rat models,” said Ying. “I think it’s very possible. So we want more researchers from USC and beyond to come and use this technology.”
Triple negative breast cancer is more aggressive and difficult to treat than other forms of the disease and, as a result, is more likely to spread throughout the body and to recur after treatment. Now a team at the University of Southern California have identified a protein that could help change that.
The research, published in the journal Nature Communications, showed that a protein called TAK1 allows cancer cells from the tumor to migrate to the lungs and then form new tumors which can spread throughout the body. There is already an FDA-approved drug called OXO that has been shown to block TAK1, but this does not survive in the blood so it’s hard to deliver to the lungs.
The USC team found a way of using nanoparticles, essentially a tiny delivery system, to take OXO and carry it to the lungs to attack the cancer cells and stop them spreading.
In a news release Min Yu, the principal investigator on the team, said that although this has only been tested in mice the results are encouraging:
“For patients with triple-negative breast cancer, systemic chemotherapies are largely ineffective and highly toxic. So, nanoparticles are a promising approach for delivering more targeted treatments, such as OXO, to stop the deadly process of metastasis.”
Mosquito spit and your immune system
Mosquito bite: Photo courtesy National Academy of Sciences
Anyone who has ever been bitten by a mosquito knows that it can be itchy and irritable for hours afterwards. But now scientists say the impact of that bite can last for much longer, days in fact, and even help prime your body for disease.
The scientists say that every time a mosquito bites you they inject saliva into the bite to keep the blood flowing freely. But that saliva also has an impact on your immune system, leaving it more vulnerable to diseases like malaria.
OK, so that’s fascinating, and really quite disgusting, but what does it have to do with stem cells? Well, researchers at the National Institute of Health’s (NIH) Malaria and Vector Research Laboratory in Phnom Penh, Cambodia engrafted human stem cells into mice to study the problem.
They found that mice with the human stem cells developed more severe symptoms of dengue fever if they were bitten by a mosquito than if they were just injected with dengue fever.
In an article in Popular Science Jessica Manning, an infectious disease expert at the NIH, said previously we had no idea that mosquito spit had such a big impact on us:
“The virus present in that mosquito’s saliva, it’s like a Trojan horse. Your body is distracted by the saliva [and] having an allergic reaction when really it should be having an antiviral reaction and fighting against the virus. Your body is unwittingly helping the virus establish infection because your immune system is sending in new waves of cells that this virus is able to infect.”
The good news is that if we can develop a vaccine against the saliva we may be able to protect people against malaria, dengue fever, Zika and other mosquito-borne diseases.
“No matter how much one prepares, the first patient is always something very special.” That’s how Dr. Mark Humayun describes his feelings as he prepared to deliver a CIRM-funded stem cell therapy to help someone going blind from dry age-related macular degeneration (AMD).
Humayun, an ophthalmologist and stem cell researcher at USC, spent years developing this therapy and so it’s understandable that he might be a little nervous finally getting a chance to see if it works in people.
It’s quite a complicated procedure, involving turning embryonic stem cells into the kind of cells that are destroyed by AMD, placing those cells onto a specially developed synthetic scaffold and then surgically implanting the cells and scaffold onto the back of the eye.
There’s a real need for a treatment for AMD, the leading cause of vision loss in the US. Right now, there is no effective therapy for AMD and some three million Americans are facing the prospect of losing their eyesight.
The first, preliminary, results of this trial were released last week and they were encouraging. You can read about them on our blog.
Thanks to USC you can also see the team that developed and executed this promising approach. They created a video capturing the moment the team were finally taking all that hard work and delivering it where it matters, to the patient.
Watching the video it’s hard not to think you are watching a piece of history, something that has the potential to do more than just offer hope to people losing their vision, it has the potential to stop and even reverse that process.
The video is a salute to the researchers who developed the therapy, and the doctors, nurses and Operating Room team who delivered it. It’s also a salute to the person lying down, the patient who volunteered to be the first to try this. Everyone in that room is a pioneer.
An eye affected by dry age-related macular degeneration
Dry age-related macular degeneration (AMD) is the leading cause of vision loss in the U.S. By 2020 it’s estimated that as many as three million Americans will be affected by the disease. Right now, there is no effective therapy. But that could change. A new CIRM-funded clinical trial is showing promise in helping people battling the disease not just in stabilizing their vision loss, but even reversing it.
In AMD, cells in the retina, the light-sensitive tissue at the back of the eye, are slowly destroyed affecting a person’s central vision. It can make it difficult to do everyday activities such as reading or watching TV and make it impossible for a person to drive.
Researchers at the University of Southern California (USC) Roski Eye Institute at the Keck School of Medicine, and Regenerative Patch Technologies, have developed a therapy using embryonic stem cells that they turned into retinal pigment epithelium (RPE) cells – the kind of cell destroyed by AMD. These cells were then placed on a synthetic scaffold which was surgically implanted in the back of the eye.
Imaging studies showed that the RPE cells appeared to integrate well into the eye and remained in place during follow-up tests 120 to 365 days after implantation.
Of the five patients enrolled in the Phase 1/2a trial, four maintained their vision in the treated eye, two showed improvement in the stability of their vision, and one patient had a 17-letter improvement in their vision on a reading chart. In addition, there were no serious side effects or unanticipated problems.
There were other indications the implants were proving beneficial. People with normal vision have the ability to focus their gaze on a single location. People with advanced AMD lose that ability. In this trial, two of the patients recovered stable fixation. These improvements were maintained in follow-up tests.
Abla Creasey, Ph.D., CIRM’S Vice President of Therapeutics and Strategic Infrastructure says even these small benefits are important:
“Having a therapy with a favorable safety profile, that could slow down the progression, or even reverse the vision loss would benefit millions of Americans. That’s why these results, while still in an early stage are encouraging, because the people treated in the trial are ones most severely affected by the disease who have the least potential for visual recovery.”
This study reflects CIRM’s long-term commitment to supporting the most promising stem cell research. The Stem Cell Agency began supporting USC’s Dr. Mark Humayun, the lead inventor of the implant, in 2010 and has been a partner with him and his team since then.
In a news release Dr. Humayun said they plan to recruit another 15 patients to see if these results hold up:
“Our study shows that this unique stem cell–based retinal implant thus far is well-tolerated, and preliminary results suggest it may help people with advanced dry age-related macular degeneration.”
While the results, published in the journal Science Translational Medicine, are encouraging the researchers caution that this was a very early stage clinical trial, with a small number of patients. They say the next step is to continue to follow the four patients treated in this trial to see if there are any further changes to their vision, and to conduct a larger trial.
The Bee Gees, pioneers in trying to find ways to mend a broken heart. Photograph: Michael Ochs Archives
This may be the first time that the Australian pop group the Bee Gees have ever been featured in a blog about stem cell research, but in this case I think it’s appropriate. One of the Bee Gees biggest hits was “How can you mend a broken heart” and while it was a fine song, Barry and Robin Gibb (who wrote the song) never really came up with a viable answer.
Happily some researchers at the University of Southern California may succeed where Barry and Robin failed. In a study, published in the journal Nature Genetics, the USC team identify a gene that may help regenerate damaged heart tissue after a heart attack.
When babies are born they have a lot of a heart muscle cell called a mononuclear diploid cardiomyocyte or MNDCM for short. This cell type has powerful regenerative properties and so is able to rebuild heart muscle. However, as we get older we have less and less MNDCMs. By the time most of us are at an age where we are most likely to have a heart attack we are also most likely to have very few of these cells, and so have a limited ability to repair the damage.
Michaela Patterson, and her colleagues at USC, set out to find ways to change that. They found that in some adult mice less than 2 percent of their heart cells were MNDCMs, while other mice had a much higher percentage, around 10 percent. Not surprisingly the mice with the higher percentage of MNDCMs were better able to regenerate heart muscle after a heart attack or other injury.
So the USC team – with a little help from CIRM funding – dug a little deeper and did a genome-wide association study of these mice, that’s where they look at all the genetic variants in different individuals to see if they can spot common traits. They found one gene, Tnni3k, that seems to play a key role in generating MNDCMs.
Turning Tnni3K off in mice resulted in higher numbers of MNDCMs, increasing their ability to regenerate heart muscle. But when they activated Tnni3k in zebrafish it reduced the number of MNDCMs and impaired the fish’s ability to repair heart damage.
While it’s a long way from identifying something interesting in mice and zebrafish to seeing if it can be used to help people, Henry Sucov, the senior author on the study, says these findings represent an important first step in that direction:
“The activity of this gene, Tnni3k, can be modulated by small molecules, which could be developed into prescription drugs in the future. These small molecules could change the composition of the heart over time to contain more of these regenerative cells. This could improve the potential for regeneration in adult hearts, as a preventative strategy for those who may be at risk for heart failure.”
How retinitis pigmentosa destroys vision – new stem cell research may help reverse that
“A pale hue”. For most of us that is a simple description, an observation about color. For Kristin Macdonald it’s a glimpse of the future. In some ways it’s a miracle. Kristin lost her sight to retinitis pigmentosa (RP). For many years she was virtually blind. But now, thanks to a clinical trial funded by CIRM she is starting to see again.
Kristin’s story is one of several examples of restoring sight in an article entitled “Why There’s New Hope About Ending Blindness” in the latest issue of National Geographic. The article explores different approaches to treating people who were either born without vision or lost their vision due to disease or injury.
Two of those stories feature research that CIRM has funded. One is the work that is helping Kristin. Retinitis pigmentosa is a relatively rare condition that destroys the photoreceptors at the back of the eye, the cells that actually allow us to sense light. The National Geographic piece highlights how a research team at the University of California, Irvine, led by Dr. Henry Klassen, has been working on a way to use stem cells to replace and repair the cells damaged by RP.
“Klassen has spent 30 years studying how to coax progenitor cells—former stem cells that have begun to move toward being specific cell types—into replacing or rehabilitating failed retinal cells. Having successfully used retinal progenitor cells to improve vision in mice, rats, cats, dogs, and pigs, he’s testing a similar treatment in people with advanced retinitis pigmentosa.”
We recently blogged about this work and the fact that this team just passed it’s first major milestone – – showing that in the first nine patients treated none experienced any serious side effects. A Phase 1 clinical trial like this is designed to test for safety, so it usually involves the use of relatively small numbers of cells. The fact that some of those treated, like Kristin, are showing signs of improvement in their vision is quite encouraging. We will be following this work very closely and reporting new results as soon as they are available.
The other CIRM-supported research featured in the article is led by what the writer calls “an eyeball dream team” featuring University of Southern California’s Dr. Mark Humayun, described as “a courteous, efficient, impeccably besuited man.” And it’s true, he is.
The team is developing a stem cell device to help treat age-related macular degeneration, the leading cause of vision loss in the US.
“He and his fellow principal investigator, University of California, Santa Barbara stem cell biologist Dennis Clegg, call it simply a patch. That patch’s chassis, made of the same stuff used to coat wiring for pacemakers and neural implants, is wafer thin, bottle shaped, and the size of a fat grain of rice. Onto this speck Clegg distributes 120,000 cells derived from embryonic stem cells.”
Humayun and Clegg have just started their clinical trial with this work so it is likely going to be some time before we have any results.
These are just two of the many different approaches, using several different methods, to address vision loss. The article is a fascinating read, giving you a sense of how science is transforming people’s lives. It’s also wonderfully written by David Dobbs, including observations like this:
“Neuroscientists love the eye because “it’s the only place you see the brain without drilling a hole,” as one put it to me.”
For a vision of the future, a future that could mean restoring vision to those who have lost it, it’s a terrific read.