Getting the inside scoop on the stem cell agency

There’s a wonderful moment at the end of the movie The Candidate (starring Robert Redford, 87% approval on Rotten Tomatoes!) about a modern political campaign for a US Senate seat. Redford (spoiler alert) plays a come-from-behind candidate and at the end when he wins he turns to his campaign manager and says “Now what?”.

I think that’s how a lot of people associated with Proposition 71 felt when it was approved by California voters in 2004, creating CIRM. Now what? During the campaign you are so focused on crossing the finish line that when the campaign is over you have to pause because you just realized it wasn’t the finishing line, it was actually the starting line.

For us “now what” involved hiring a staff, creating oversight groups of scientists and ethics experts, developing strategies and then mechanisms for funding, and then mechanisms for tracking that funding to make sure it was being used properly. It was creating something from scratch and trying to do something that no state agency had done before.

Fifteen years later we are coming to the end of the funding provided by Prop 71 and that question keeps popping up again, “Now what?” And that’s what we are going to be talking about in our next Facebook Live.

We have three great experts on our panel. They are scientists and researchers and leaders in biotech, but also members of our CIRM Board. We rely on their experience and expertise in making key decisions and you can rely on them to pull back the curtain and talk about the things that matter most to them in helping advance our mission, and in helping secure our legacy.

Anne-Marie Duliege MD, has more than 25 years of experience in the medical world, starting out as a pediatrician and then moving into research. She has experience developing new therapies for auto-immune disorders, lung problems and infectious diseases.

Like Anne-Marie, Joe Panetta, has years of experience working in the research field, and is currently President & CEO of Biocom, the California association that advocates for more than 1,200 companies, universities and research institutes working in biotechnology.

Finally, Dave Martin MD, came to CIRM after stints at the National Institutes of Health (NIH), UC San Francisco, Genentech, Chiron and several other highly-regarded organizations. He is also the co-founder, chairman and CEO of AvidBiotics, a privately held biotechnology company in South San Francisco.

Each brings a different perspective to the work that we do at CIRM, and each enriches it not just with their intelligence and experience, but also with their compassion for the patients and commitment to our mission.

So, join us on Thursday, July 25th from noon till 1pm (PDT) for a special Facebook Live “Ask the Stem Cell Team” to understand how we got where we are, how the rest of the field is doing, and what happens next. It promises to be a fascinating hour.

Breaking bad news to stem cell researchers

It’s never easy to tell someone that they are too late, that they missed the deadline. It’s particularly hard when you know that the person you are telling that to has spent years working on a project and now needs money to take it to the next level. But in science, as in life, it’s always better to tell people what they need to know rather than what they would like to hear.

And so, we have posted a notice on our website for researchers thinking about applying for funding that, except in a very few cases, they are too late, that there is no money available for new projects, whether it’s Discovery, Translational or Clinical.

Here’s that notice:

CIRM anticipates that the budget allocation of funds for new awards under the CIRM clinical program (CLIN1, CLIN2 and CLIN3) may be depleted within the next two to three months. CIRM will accept applications for the monthly deadline on June 28, 2019 but will suspend application submissions after that date until further notice. All applicants should note that the review of submitted applications may be halted at any point in the process if funds are depleted prior to completion of the 3-month review cycle. CIRM will notify applicants of such an occurrence. Therefore, submission and acceptance of an application to CIRM does not guarantee the availability of funds or completion of a review cycle.

The submission of applications for the CIRM/NHLBI Cure Sickle Cell Initiative (CLIN1 SCD, CLIN2 SCD) are unaffected and application submissions for this program will remain open.

We do, of course, have enough money set aside to continue funding all the projects our Board has already approved, but we don’t have money for new projects (except for some sickle cell disease projects).

In truth our funding has lasted a lot longer than anyone anticipated. When Proposition 71 was approved the plan was to give CIRM $300 million a year for ten years. That was back in 2004. So what happened?

Well, in the early years stem cell science was still very much in its infancy with most of the work being done at a basic or Discovery level. Those typically don’t require very large sums so we were able to fund many projects without hitting our $300m target. As the field progressed, however, more and more projects were at the clinical trial stage and those need multiple millions of dollars to be completed. So, the money went out faster.

To date we have funded 55 clinical trials and our early support has helped more than a dozen other projects get into clinical trials. This includes everything from cancer and stroke, to vision loss and diabetes. It’s a good start, but we feel there is so much more to do.

Followers of news about CIRM know there is talk about a possible ballot initiative next year that would provide another $5.5 billion in funding for us to help complete the mission we have started.

Over the years we have built a pipeline of promising projects and without continued support many of those projects face a difficult future. Funding at the federal level is under threat and without CIRM there will be a limited number of funding alternatives for them to turn to.

Telling researchers we don’t have any money to support their work is hard. Telling patients we don’t have any money to support work that could lead to new treatments for them, that’s hardest of all.

“A new awakening”: One patient advocate’s fight for her daughters life

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.

Nancy Stack is pictured in spring 2018 with her daughter Natalie Stack and husband Geoffrey Stack. (Lar Wanberg/Cystinosis Research Foundation)

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. 

Cystinosis is 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.

For our 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.

Medical 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 blindness.   

We started 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 disease. 

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 running out”. 

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.

Media matters in spreading the word

Cover of New Yorker article on “The Birth Tissue Profiteers”. Illustration by Ben Jones

When you have a great story to tell the best and most effective way to get it out to the widest audience is still the media, both traditional mainstream and new social media. Recently we have seen three great examples of how that can be done and, hopefully, the benefits that can come from it.

First, let’s go old school. Earlier this month Caroline Chen wrote a wonderful in-depth article about clinics that are cashing in on a gray area in stem cell research. The piece, a collaboration between the New Yorker magazine and ProPublica, focused on the use of amniotic stem cell treatments and the gap between what the clinics who offer it are claiming it can do, and the reality.

Here’s one paragraph profiling a Dr. David Greene, who runs a company providing amniotic fluid to clinics. It’s a fine piece of writing showing how the people behind these therapies blur the lines between fact and reality, not just about the cells but also about themselves:

“Greene said that amniotic stem cells derive their healing power from an ability to develop into any kind of tissue, but he failed to mention that mainstream science does not support his claims. He also did not disclose that he lost his license to practice medicine in 2009, after surgeries he botched resulted in several deaths. Instead, he offered glowing statistics: amniotic stem cells could help the heart beat better, “on average by twenty per cent,” he said. “Over eighty-five per cent of patients benefit exceptionally from the treatment.”

Greene later backpedals on that claim, saying:

“I don’t claim that this is a treatment. I don’t claim that it cures anything. I don’t claim that it’s a permanent fix. All I discuss is maybe, potentially, people can get some improvements from stem-cell care.”

CBS2 TV Chicago

This week CBS2 TV in Chicago did their own investigative story about how the number of local clinics offering unproven and unapproved therapies is on the rise. Reporter Pam Zekman showed how misleading newspaper ads brought in people desperate for something, anything, to ease their arthritis pain.

She interviewed two patients who went to one of those clinics, and ended up out of pocket, and out of luck.

“They said they would regenerate the cartilage,” Patricia Korona recalled. She paid $4500 for injections in her knee, but the pain continued. Later X-rays were ordered by her orthopedic surgeon.

He found bone on bone,” Korona said. “No cartilage grew, which tells me it failed; didn’t work.”

John Zapfel paid $14,000 for stem cell injections on each side of his neck and his shoulder. But an MRI taken by his current doctor showed no improvement.

“They ripped me off, and I was mad.” Zapfel said.      

TV and print reports like this are a great way to highlight the bogus claims made by many of these clinics, and to shine a light on how they use hype to sell hope to people who are in pain and looking for help.

At a time when journalism seems to be increasingly under attack with accusations of “fake news” it’s encouraging to see reporters like these taking the time and news outlets devoting the resources to uncover shady practices and protect vulnerable patients.

But the news isn’t all bad, and the use of social media can help highlight the good news.

That’s what happened yesterday in our latest CIRM Facebook Live “Ask the Stem Cell Team” event. The event focused on the future of stem cell research but also included a really thoughtful look at the progress that’s been made over the last 10-15 years.

We had two great guests, UC Davis stem cell researcher and one of the leading bloggers on the field, Paul Knoepfler PhD; and David Higgins, PhD, a scientist, member of the CIRM Board and a Patient Advocate for Huntington’s Disease. They were able to highlight the challenges of the early years of stem cell research, both globally and here at CIRM, and show how the field has evolved at a remarkable rate in recent years.

Paul Knoepfler

Naturally the subject of the “bogus clinics” came up – Paul has become a national expert on these clinics and is quoted in the New Yorker article – as did the subject of the frustration some people feel at what they consider to be the too-slow pace of progress. As David Higgins noted, we all think it’s too slow, but we are not going to race recklessly ahead in search of something that might heal if we might also end up doing something that might kill.

David Higgins

A portion of the discussion focused on funding and, in particular, what happens if CIRM is no longer around to fund the most promising research in California. We are due to run out of funding for new projects by the end of this year, and without a re-infusion of funds we will be pretty much closing our doors by the end of 2020. Both Paul and David felt that could be disastrous for the field here in California, depriving the most promising projects of support at a time when they needed it most.

It’s probably not too surprising that three people so closely connected to CIRM (Paul has received funding from us in the past) would conclude that CIRM is needed for stem cell research to not just survive but thrive in California.

A word of caution before you watch: fashion conscious people may be appalled at how my pocket handkerchief took on a life of its own.

The Past, the Present, and the Uncertain Future of Stem Cell Research

Ronnie, a boy who was born without a functioning immune system but who is thriving today because of CIRM funded research

When CIRM was created in 2004 the field of stem cell research was still very much in its infancy. Fast forward 15 years and it’s moving ahead at a rapid pace, probably faster than most scientists would have predicted. How fast? Find out for yourself at a free public event at UC San Diego on May 28th from 12.30 to 1.30p.

In the last 15 years CIRM has funded 53 clinical trials in everything from heart disease and stroke, to spinal cord injury, vision loss, sickle cell disease and HIV/AIDS.

UCSD was one of the first medical centers chosen to host a CIRM Alpha Stem Cell Clinic – a specialist center with the experience and expertise to deliver stem cell therapies to patients – and to date is running more than a dozen clinical trials for breast cancer, heart failure, leukemia and chronic lower back pain.

Clearly progress is being made. But the field is also facing some challenges. Funding at the federal level for stem cell research is under threat, and CIRM is entering what could be its final phase. We have enough money left to fund new projects through this year (and these are multi-year projects so they will run into 2021 or 2022) but unless there is a new round of funding we will slowly disappear. And with us, may also disappear the hopes of some of the most promising projects underway.

If CIRM goes, then projects that we have supported and nurtured through different phases of research may struggle to make it into a clinical trial because they can’t get the necessary funding.

Clearly this is a pivotal time in the field.

We will discuss all this, the past, the present and the uncertain future of stem cell research at the meeting at UC San Diego on May 28th. The doors will open at noon for registration (snacks and light refreshments will also be available) and the program runs from 12.30p to 1.30p.

The speakers are:

  • Dr. Catriona Jamieson, Director of the UC San Diego Health CIRM Alpha Stem Cell Clinic and Sanford Stem Cell Clinical Center.
  • Dr. Maria Millan, President and CEO of CIRM
  • Dr. David Higgins, CIRM Board member and Patient Advocate for Parkinson’s Disease.

And of course, we want to hear from you, so we’ll leave plenty of time for questions.

Free parking is available.

Go here for more information about the event and how you can register

Free free to share this with anyone you think might be interested in joining us and we look forward to seeing you there.

Organoids revolutionize approach to studying a variety of diseases

Organoids

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 News lead 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.

Stem Cell Agency Awards Almost $4 Million to Develop a Treatment for Spinal Degeneration

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.

Facebook Live – Ask the Stem Cell Team about Patient Advocacy

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.

Stem cell byproducts provide insight into cure for spina bifida

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.

Their findings were published in the Journal of the Federation of American Societies for Experimental Biology on February 12, 2019.

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.

Antibody effective in cure for rare blood disorders

3D illustration of an antibody binding to a designated target.
Illustration created by Audra Geras.

A variety of diseases can be traced to a simple root cause: problems in the bone marrow. The bone marrow contains specialized stem cells known as hematopoietic stem cells (HSCs) that give rise to different types of blood cells. As mentioned in a previous blog about Sickle Cell Disease (SCD), one problem that can occur is the production of “sickle like” red blood cells. In blood cancers like leukemia, there is an uncontrollable production of abnormal white blood cells. Another condition, known as myelodysplastic syndromes (MDS), are a group of cancers in which immature blood cells in the bone marrow do not mature and therefore do not become healthy blood cells.

For diseases that originate in the bone marrow, one treatment involves introducing healthy HSCs from a donor or gene therapy. However, before this type of treatment can take place, all of the problematic HSCs must be eliminated from the patient’s body. This process, known as pre-treatment, involves a combination of chemotherapy and radiation, which can be extremely toxic and life threatening. There are some patients whose condition has progressed to the point where their bodies are not strong enough to withstand pre-treatment. Additionally, there are long-term side effects that chemotherapy and radiation can have on infant children that are discussed in a previous blog about pediatric brain cancer.

Could there be a targeted, non-toxic approach to eliminating unwanted HSCs that can be used in combination with stem cell therapies? Researchers at Stanford say yes and have very promising results to back up their claim.

Dr. Judith Shizuru and her team at Stanford University have developed an antibody that can eliminate problematic blood forming stem cells safely and efficiently. The antibody is able to identify a protein on HSCs and bind to it. Once it is bound, the protein is unable to function, effectively removing the problematic blood forming stem cells.

Dr. Shizuru is the senior author of a study published online on February 11th, 2019 in Blood that was conducted in mice and focused on MDS. The results were very promising, demonstrating that the antibody successfully depleted human MDS cells and aided transplantation of normal human HSCs in the MDS mouse model.

This proof of concept holds promise for MDS as well as other disease conditions. In a public release from Stanford Medicine, Dr. Shizuru is quoted as saying, “A treatment that specifically targets only blood-forming stem cells would allow us to potentially cure people with diseases as varied as sickle cell disease, thalassemia, autoimmune disorders and other blood disorders…We are very hopeful that this body of research is going to have a positive impact on patients by allowing better depletion of diseased cells and engraftment of healthy cells.”

The research mentioned was partially funded by us at CIRM. Additionally, we recently awarded a $3.7 million dollar grant to use the same antibody in a human clinical trial for the so-called “bubble baby disease”, which is also known as severe combined immunodeficiency (SCID). You can read more about that award on a previous blog post linked here.