CIRM-funded research at Sanford Burnham Prebys Medical Discovery Institute in San Diego is identifying compounds that could be used to help children battling a deadly brain cancer.
The cancer is choroid plexus carcinoma (CPC), a rare
brain tumor that occurs mainly in children. As it grows the tumor can affect
nearby parts of the brain resulting in nausea, vomiting and headaches.
Treatment involves surgery to remove the tumor followed
by chemotherapy and radiation. However, many of the children are too young to
undergo radiation and only around 40 percent are still alive five years after
being diagnosed. Even those who do survive often experience life-long
consequences such as developmental disabilities.
One obstacle to developing better therapies has been the
lack of a good animal model to enhance our understanding of the disease. That’s
where this later research, published in the journal Cancer Research, comes in.
The team at Sanford Burnham developed a new mouse model,
by knocking out p53, a gene known to suppress tumor formation, and activating a
gene called Myc, which is known to cause cancer.
Robert Wechsler-Reya
In a news release, Robert Wechsler-Reya, the senior author of the paper, says this new model mirrors the way CPC grows and develops in humans.
“This model is a
valuable tool that will increase our understanding of the biology of the cancer
and allow us to identify and test novel approaches to therapy. This advance
brings us one step closer to a future where every child survives—and thrives—after
diagnosis with CPC.”
As proof of that
the team tested nearly 8,000 compounds against the mouse tumor cells, to see if
they could help stop or slow the progression of the disease. They identified
three that showed potential of not just stopping the cancer, but of also not
harming healthy surrounding cells.
“These compounds are promising, much-needed leads in the
quest for an effective CPC treatment,” says Wechsler-Reya. “Our laboratory
plans to evaluate these and additional compounds that can effectively treat
this cancer.”
Getting older is a normal, at times existential, part of life. The outward changes are abundant and noticeable: thinning of the hair, greying of the hair, and added lines to the face. There are also changes that happen that are not so abundantly clear in terms of outward appearance: slowing of healing time for bone fractures and a gradual loss of bodily function. The process of aging poses one very fundamental question — Could understanding how stem cells age lead to a greater understanding of how diseases develop? More importantly, could it guide the approach towards developing potential treatments? Two different studies highlight the importance of evaluating and understanding the process of aging in stem cells.
The first study, led by Dr. Michael Fehlings, looked at the impact of donor age in relation to stem cell therapies for spinal cord injuries (SCI). Dr. Fehlings, with a team of investigators from the University of Toronto and Krembil Research Institute, University Health Network, used an adult rat model to look at how cells derived from young vs. old stem cells affected tissue regeneration and recovery after a spinal cord injury.
Some rats with a SCI received cells derived from stem cells in the umbilical cord blood, which are considered “young” stem cells. The other rats with a SCI received cells derived from stem cells in the bone marrow, which are considered “old” stem cells. The results showed, ten weeks after treatment, that rats given the “young” stem cells exhibited a better recovery in comparison to those given the “old” stem cells.
“Together, this minimally invasive and effective approach to cell therapy has significant implications on the treatment of traumatic cervical SCI and other central nervous system injuries. These results can help to optimize cell treatment strategies for eventual use in humans.”
The full results to this study were published in Stem Cells Translational Medicine.
The second, separate study, conducted by Dr. Stephen Crocker at UConn Health, looks at brain stem cells in people with multiple sclerosis (MS), a neurodegenerative disease caused by the inflammation and destruction of the insulation around the nerves, also known as myelin. Problems with insulation around the nerves can prevent or complicate the electrical signals sent from the brain to the body, which can lead to problems with walking or other bodily movements.
Drawing of a healthy nerve cell with insulation (left) and one damaged by multiple sclerosis (right). Image courtesy of Shutterstock
Dr. Crocker and his team found that brain stem cells in patients with MS look much older when compared to the brain stem cells of a healthy person around the same age. Not only did these brain stem cells look older, but they also acted much older in comparison to their healthy counterparts. It was also discovered that the brain stem cells of MS patients were producing a protein that prevented the development of insulation around the nerves. What is more remarkable is that Dr. Crocker and his team demonstrated that when this protein is blocked, the insulation around the nerves develops normally again.
In a press release, Dr. Valentina Fossati, a neurologist at the New York Stem Cell Foundation who evaluated these brain stem cells, stated that,
“We are excited that the study of human stem cells in a dish led to the discovery of a new disease mechanism that could be targeted in much-needed therapeutics for progressive MS patients.”
The complete study was published in the Proceedings of the National Academy of Sciences (PNAS).
For some years now CIRM has been raising the alarm about the growing numbers of clinics offering unproven and unapproved stem cell therapies. But we are not alone. Now a leader of the California state Assembly is taking action, trying to ensure the clinics follow the law and don’t endanger patients.
Kevin Mullin is the Speaker pro Tem in the Assembly. He is championing a bill, AB 617, that will create a Stem Cell Clinic Regulation Advisory Group. In a news release Mullin said the motivation behind the bill is simple:
“As the Chair of the Select Committee on Biotechnology, I have heard from patients who have experienced both sides of the treatment continuum. It is clear that more must be done to ensure the proper regulation of for-profit stem cell clinics.”
Concerns about these clinics are well-founded. The clinics claim the treatments they offer – usually involving the use of the patient’s own fat or blood cells – can help address everything from arthritis to Alzheimer’s but offer little or no proof. Because the “therapies” are not approved by the FDA they are not covered by insurance, so people spend thousands, sometimes tens of thousands of dollars for something that is almost guaranteed to do little to help. In some cases, the “treatments” have had disastrous results, harming patients.
The news release
from Speaker pro Tem Mullin’s office says CIRM has helped position California
as a leader in stem cell research.
“Unfortunately,
not all stem cell clinics are adhering to the expected high standards of review
within the industry and, as a result, patients have been subjected to
unscrupulous, sometimes harmful practices. AB 617 will address those entities
by creating a Stem Cell Clinic Regulation Advisory Group.”
The Advisory Group will review existing licensing and certification laws for clinics offering stem cell therapies. The Group would then make recommendations to the Legislature about ways to improve the existing rules and ensure greater protection for patients. CIRM has been working with Speaker pro Tem Mullin on AB 617 and, as our President & CEO, Maria Millan, said we will continue to do so.
“We fully support AB 617
and Speaker pro Tem Mullin’s efforts to protect California consumers from
unregulated and unproven stem cell treatments. AB 617 will help
patients, their families and the medical community identify legitimate clinics
that offer scientifically tested clinical trials and treatments that meet
federal regulatory requirements. The field of regenerative medicine
and cell and gene therapy are coming of age and entering the realm of medical
practice, so AB 617 would set up an important foundation for ensuring that the
highest quality care is provided to patients seeking these treatments.”
There are limitations to studying cells under a microscope. To understand some of the more complex processes, it is critical to see how these cells behave in an environment that is similar to conditions in the body. The production of organoids has revolutionized this approach.
Organoids are three-dimensional structures derived from stem cells that have similar characteristics of an actual organ. There have been several studies recently published that have used this approach to understand a wide scope of different areas.
In one such instance, researchers at The University of Cambridge were able to grow a “mini-brain” from human stem cells. To demonstrate that this organoid had functional capabilities similar to that of an actual brain, the researchers hooked it up to a mouse spinal cord and surrounding muscle. What they found was remarkable– the “mini-brain” sent electrial signals to the spinal cord that made the surrounding muscles twitch. This model could pave the way for studying neurodegenerative diseases such as spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS).
Spinal muscular atrophy
Speaking of SMA, researchers in Singapore have used organoids to come up with some findings that might be able to help people battling the condition.
SMA is a neurodegenerative disease caused by a protein
deficiency that results in nerve degeneration, paralysis and even premature
death. The fact that it mainly affects children makes it even worse. Not much
is known how SMA develops and even less how to treat or prevent it.
That’s where the research from the A*STAR’s Institute of
Molecular and Cell Biology (IMCB) comes in. Using the iPSC method
they turned tissue samples from healthy people and people with SMA into spinal
organoids.
They then compared the way the cells
developed in the organoids and found that the motor nerve cells from healthy
people were fully formed by day 35. However, the cells from people with SMA
started to degenerate before they got to that point.
They also found that the protein
problem that causes SMA to develop did so by causing the motor nerve cells to
divide, something they don’t normally do. So, by blocking the mechanism that
caused the cells to divide they were able to prevent the cells from dying.
In an article in Science
and Technology Research Newslead researcher Shi-Yan Ng said this
approach could help unlock clues to other diseases such as ALS.
“We are one of the first labs to report the formation of spinal organoids. Our study presents a new method for culturing human spinal-cord-like tissues that could be crucial for future research.”
Just yesterday the CIRM Board awarded almost $4 million to Ankasa Regenerative Therapeutics to try and develop a treatment for another debilitating back problem called degenerative spondylolisthesis.
And finally, organoid modeling was used to better understand and study an infectious disease. Scientists from the Max Planck Institute for Infection Biology in Berlin created fallopian tube organoids from normal human cells. Fallopian tubes are the pair of tubes found inside women along which the eggs travel from the ovaries to the uterus. The scientists observed the effects of chronic infections of Chlamydia, a sexually transmittable infection. It was discovered that chronic infections lead to permanent changes at the DNA level as the cells age. These changes to DNA are permanent even after the infection is cleared, and could be indicative of the increased risk of cervical cancer observed in women with Chlamydia or those that have contracted it in the past.
Today the
governing Board of the California Institute for Regenerative Medicine (CIRM)
awarded $3.9 million to Ankasa Regenerative Therapeutics for a promising approach to treat a
degenerative condition that can cause chronic, progressive back pain.
As we get
older, the bones, joints and ligaments in our back become weak and less able to
hold the spinal column in alignment. As
a result, an individual vertebral bone in our spine may slip forward over the
one below it, compressing the nerves in the spine, and causing lower back pain
or radiating pain. This condition,
called degenerative spondylolisthesis, primarily affects individuals over the
age of 50 and, if left untreated, can cause intense pain and further
degeneration of adjacent regions of the spine.
Current
treatment usually involves taking bone from one of the patient’s other bones,
and moving it to the site of the injury.
The transplanted bone contains stem cells necessary to generate new
bone. However, there is a caveat to this
approach— as we get older the grafts become less effective because the stem
cells in our bones are less efficient at making new bone. The end result is little or no bone
healing.
Ankasa has developed ART352-L, a protein-based drug product
meant to enhance the bone healing properties of these bone grafts. ART352-L works by stimulating bone stem cells
to increase the amount of bone produced
by the graft.
The award is in the form of a CLIN1 grant, with the goal of
completing the testing needed to apply to the Food and Drug Administration
(FDA) for permission to start a clinical trial in people.
This is a project that CIRM has supported through earlier
phases of research.
“We are excited to see the development that this approach has made since its early stages and reflects our commitment to supporting the most promising science and helping it advance to the clinic,” says Maria T. Millan, MD, President & CEO of CIRM. “There is an unmet medical need in older patients with bone disorders such as degenerative spondylolisthesis. As our population ages, it is important for us to invest in potential treatments such as these that can help alleviate a debilitating condition that predisposes to additional and fatal medical complications.”
See the animated video below for a descriptive and visual synopsis of degenerative spondylolisthesis.
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.”
Dr. Michael Milyavsky (left) and his research student Muhammad Yassin (right). Image courtesy of Tel Aviv University.
Every three minutes, one person in the United States is diagnosed with a blood cancer, which amounts to over 175,000 people every year. Every nine minutes, one person in the United States dies from a blood cancer, which is over 58,000 people every year. These eye opening statistics from the Leukemia & Lymphoma Society demonstrate why almost one in ten cancer deaths in 2018 were blood cancer related.
For those unfamiliar with the term, a blood cancer is any type of cancer that begins in blood forming tissue, such as those found in the bone marrow. One example of a blood cancer is leukemia, which results in the production of abnormal blood cells. Chemotherapy and radiation are used to wipe out these cells, but the blood cancer can sometimes return, something known as a relapse.
What enables the return of a blood cancer such as leukemia ? The answer lies in the properties of cancer stem cells, which have the ability to multiply and proliferate and can resist the effects of certain types of chemotherapy and radiation. Researchers at Tel Aviv University are looking to decrease the rate of relapse in blood cancer by targeting a specific type of cancer stem cell known as a leukemic stem cell, which are often found to be the most malignant.
Dr. Michael Milyavsky and his team at Tel Aviv University have developed a biosensor that is able to isolate, label, and target specific genes found in luekemic stem cells. Their findings were published on January 31, 2019 in Leukemia.
“The major reason for the dismal survival rate in blood cancers is the inherent resistance of leukemic stem cells to therapy, but only a minor fraction of leukemic cells have high regenerative potential, and it is this regeneration that results in disease relapse. A lack of tools to specifically isolate leukemic stem cells has precluded the comprehensive study and specific targeting of these stem cells until now.”
In addition to isolating and labeling leukemic stem cells, Dr. Milyavsky and his team were able to demonstrate that the leukemic stem cells labeled by their biosensor were sensitive to an inexpensive cancer drug, highlighting the potential this technology has in creating more patient-specific treatment options.
In the article, Dr. Milyavsky said:
” Using this sensor, we can perform personalized medicine oriented to drug screens by barcoding a patient’s own leukemia cells to find the best combination of drugs that will be able to target both leukemia in bulk as well as leukemia stem cells inside it.”
The researchers are now investigating genes that are active in leukemic stem cells in the hope finding other druggable targets.
CIRM has funded two clinical trials that also use a more targeted approach for cancer treatment. One of these trials uses an antibody to treat chronic lymphocytic leukemia (CLL) and the other trial uses a different antibody to treat acute myeloid leukemia (AML).
How often do you get to ask an expert a question about something that matters deeply to you and get an answer right away? Not very often I’m guessing. That’s why CIRM’s Facebook Live “Ask the Stem Cell Team About Patient Advocacy” gives you a chance to do just that this Thursday, March 14th from noon till 1pm PST.
We have three amazing individuals who will share their experiences, their expertise and advice as Patient Advocates, and answer your questions about how to be an effective advocate for your cause.
The three are:
Gigi McMillan became a Patient Advocate when her 5-year-old son was diagnosed with a brain tumor. That led her to helping develop support systems for families going through the same ordeal, to help researchers develop appropriate consent processes and to campaign for the rights of children and their families in research.
Adrienne Shapiro comes from a family with a long history of Sickle Cell Disease (SCD) and has fought to help people with SCD have access to compassionate care. She is the co-founder of Axis Advocacy, an organization dedicated to raising awareness about SCD and support for those with it. In addition she is now on the FDA’s Patient Engagement Collaborative, a new group helping the FDA ensure the voice of the patient is heard at the highest levels.
David Higgins is a CIRM Board member and a Patient Advocate for Parkinson’s Disease. David has a family history of the disease and in 2011 was diagnosed with Parkinson’s. As a scientist and advocate he has championed research into the disease and worked to raise greater awareness about the needs of people with Parkinson’s.
Also, make sure to “like” our FaceBook page before the event to receive a notification when we’ve gone live for this and future events. If you miss the broadcast, not to worry. We’ll be posting it on our Facebook video page, our website, and YouTube channel shortly afterwards.
We want to answer your most pressing questions, so please email them directly to us beforehand at info@cirm.ca.gov.
And, of course, feel free to share this information with anyone you think might be interested.
Last week’s news headlines were dominated by one big story, the use of a stem cell transplant to effectively cure a person of HIV. But there were other stories that, while not quite as striking, did also highlight how the field is advancing.
A new way to boost brain cells
(in mice!)
It’s hard to fix
something if you don’t really know what’s wrong in the first place. It would be
like trying to determine why a car is not working just by looking at the hood
and not looking inside at the engine. The human brain is far more complex than
a car so trying to determine what’s going wrong is infinitely more challenging.
But a new study could help give us a new option.
Researchers in
Luxembourg and Germany have developed a new computer model for what’s happening
inside the brain, identifying what cells are not operating properly, and fixing
them.
Antonio del Sol, one
of the lead authors of the study – published in the journal Cell
– says their new model allows them to identify which stem cells are active and
ready to divide, or dormant.
“Our results constitute an important
step towards the implementation of stem cell-based therapies, for instance for
neurodegenerative diseases. We were able to show that, with computational
models, it is possible to identify the essential features that are
characteristic of a specific state of stem cells.”
The work, done in
mice, identified a protein that helped keep brain stem cells inactive in older
animals. By blocking this protein they were able to help “wake up” those stem
cells so they could divide and proliferate and help regenerate the aging brain.
And if it works in
mice it must work in people right? Well, that’s what they hope to see next.
Deeper understanding of fetal development
According to the Mayo
Clinic between 10 and 20 percent of known pregnancies end in
miscarriage (though they admit the real number may be even higher) and our lack
of understanding of fetal development makes it hard to understand why. A new
study reveals a previously unknown step in this development that could help
provide some answers and, hopefully, lead to ways to prevent miscarriages.
Researchers at the
Karolinska Institute in Sweden used genetic sequencing to follow the
development stages of mice embryos. By sorting those different sequences into a
kind of blueprint for what’s happening at every stage of development they were
able to identify a previously unknown phase. It’s the time between when the
embryo attaches to the uterus and when it begins to turn these embryonic stem
cells into identifiable parts of the body.
Qiaolin Deng, Karolinska Institute
Lead researcher Qiaolin Deng says this finding provides vital new evidence.
“Being able to follow the
differentiation process of every cell is the Holy Grail of developmental
biology. Knowledge of the events and factors that govern the development of the
early embryo is indispensable for understanding miscarriages and congenital
disease. Around three in every 100 babies are born with fetal malformation
caused by faulty cellular differentiation.”
The study is
published in the journal Cell
Reports.
Could a new drug discovery
reduce damage from a heart attack?
Every 40 seconds someone in the US has a heart attack. For many it is fatal but even for those who survive it can lead to long-term damage to the heart that ultimately leads to heart failure. Now British researchers think they may have found a way to reduce that likelihood.
Using stem cells to
create human heart muscle tissue in the lab, they identified a protein that is
activated after a heart attack or when exposed to stress chemicals. They then
identified a drug that can block that protein and, when tested in mice that had
experienced a heart attack, they found it could reduce damage to the heart
muscle by around 60 percent.
Prof Michael Schneider,
the lead researcher on the study, published in Cell
Stem Cell, said this could be a game changer.
“There are no
existing therapies that directly address the problem of muscle cell death and
this would be a revolution in the treatment of heart attacks. One reason why
many heart drugs have failed in clinical trials may be that they have not been
tested in human cells before the clinic. Using both human cells and animals
allows us to be more confident about the molecules we take forward.”
A diagram of an infant born with spina bifida, a birth defect where there is an incomplete closing of the backbone portion of the spinal cord. Photo courtesy of the Texas Children’s Hospital website.
Some of you might remember a movie in the early 2000s by the name of “Miracle in Lane 2”. The film is based on an inspirational true story and revolves around a boy named Justin Yoder entering a soapbox derby competition. In the movie, Justin achieves success as a soapbox derby driver while adapting to the challenges of being in a wheelchair.
Scene from “Miracle in Lane 2”
The reason that Justin is unable to walk is due to a birth defect known as spina bifida, which causes an incomplete closing of the backbone portion of the spinal cord, exposing tissue and nerves. In addition to difficulties with walking, other problems associated with this condition are problems with bladder or bowel control and accumulation of fluid in the brain.
According to the Center for Disease Control (CDC) , each year about 1,645 babies in the US are born with spina bifida, with Hispanic women having the highest rate of children born with the condition. There is currently no cure for this condition, but researchers at UC Davis are one step closer to changing that.
Dr. Aijun Wang examining cells under a microscope. He has identified stem cell byproducts that protect neurons. Photo courtesy of UC Regents/UC Davis Health
Dr. Aijun Wang, Dr. Diana Farmer, and their research team have identified crucial byproducts produced by stem cells that play an important role in protecting neurons. These byproducts could assist with improving lower-limb motion in patients with spina bifida.
Prior to this discovery, Dr. Farmer and Dr. Wang demonstrated that prenatal surgery combined with connective tissue (e.g. stromal cells) derived from stem cells improved hind limb control in dogs with spina bifida. Below you can see a clip of two English bulldogs with spina bifida who are now able to walk.
The team will use their findings to perfect the neuroprotective qualities of a stem cell treatment developed to improve locomotive problems associated with spina bifida.
In a public release posted by EurekaAlert!, Dr. Wang is quoted as saying, “We are excited about what we see so far and are anxious to further explore the clinical applications of this research.”
The discovery and development of a treatment for spina bifida was funded by a $5.66 million grant from CIRM. You can read more about that award and spina bifida on a previous blog post linked here.
The Stem Cellar 