“Brains” in a dish that can create electrical impulses

Brain organoids in a petri dish: photo courtesy UCSD

For several years, researchers have been able to take stem cells and use them to make three dimensional structures called organoids. These are a kind of mini organ that scientists can then use to study what happens in the real thing. For example, creating kidney organoids to see how kidney disease develops in patients.

Scientists can do the same with brain cells, creating clumps of cells that become a kind of miniature version of parts of the brain. These organoids can’t do any of the complex things our brains do – such as thinking – but they do serve as useful physical models for us to use in trying to develop a deeper understanding of the brain.

Now Alysson Muotri and his team at UC San Diego – in a study supported by two grants from CIRM – have taken the science one step further, developing brain organoids that allow us to measure the level of electrical activity they generate, and then compare it to the electrical activity seen in the developing brain of a fetus. That last sentence might cause some people to say “What?”, but this is actually really cool science that could help us gain a deeper understanding of how brains develop and come up with new ways to treat problems in the brain caused by faulty circuitry, such as autism or schizophrenia.

The team developed new, more effective methods of growing clusters of the different kinds of cells found in the brain. They then placed them on a multi-electrode array, a kind of muffin tray that could measure electrical impulses. As they fed the cells and increased the number of cells in the trays they were able to measure changes in the electrical impulses they gave off. The cells went from producing 3,000 spikes a minute to 300,000 spikes a minute. This is the first time this level of activity has been achieved in a cell-based laboratory model. But that’s not all.

When they further analyzed the activity of the organoids, they found there were some similarities to the activity seen in the brains of premature babies. For instance, both produced short bursts of activity, followed by a period of inactivity.

Alysson Muotri

In a news release Muotri says they were surprised by the finding:

“We couldn’t believe it at first — we thought our electrodes were malfunctioning. Because the data were so striking, I think many people were kind of skeptical about it, and understandably so.”

Muotri knows that this research – published in the journal Cell Stem Cell – raises ethical issues and he is quick to say that these organoids are nothing like a baby’s brain, that they differ in several critical ways. The organoids are tiny, not just in size but also in the numbers of cells involved. They also don’t have blood vessels to keep them alive or help them grow and they don’t have any ability to think.

“They are far from being functionally equivalent to a full cortex, even in a baby. In fact, we don’t yet have a way to even measure consciousness or sentience.”

What these organoids do have is the ability to help us look at the structure and activity of the brain in ways we never could before. In the past researchers depended on mice or other animals to test new ideas or therapies for human diseases or disorders. Because our brains are so different than animal brains those approaches have had limited results. Just think about how many treatments for Alzheimer’s looked promising in animal models but failed completely in people.

These new organoids allow us to explore how new therapies might work in the human brain, and hopefully increase our ability to develop more effective treatments for conditions as varied as epilepsy and autism.

Bridges to the Future: 10 Years and Counting!

Bridges conference 2019

When Californians voted for Proposition 71 in 2004, they were investing in hope… the hope that unraveling the mysteries of stem cells could lead to new types of treatments and perhaps one day, even cures for some of the most devastating illnesses and injuries known to mankind. Making this hope a reality, however, requires much more than scientific discovery, it requires a dedicated and skilled work force that can recognize and tackle the challenges that come with such an ambitious dream.

To jump start the nascent stem cell/regenerative medicine community in California, CIRM began offering Training Grants to major research and medical institutions to attract talented PhD students and postdoctoral fellows into the field. A few years later, a second type of training program was born to attract a different, yet equally important cadre of professionals – the undergraduate, Bachelors and Master’s level scientists who are the bread and butter of any successful research endeavor.

Bridges students

Over the past 10 years, CIRM has supported 16 of these programs, which have proven to be among the most popular and successful CIRM initiatives to date. As of 2019, the Bridges programs have trained well over 1400 scientists, about half of whom are working full time in research positions at biotechnology companies or academic laboratories, and another third of whom are currently enrolled in a graduate or professional school.

Today, there are 14 active Bridges Programs around the state, each with unique attributes, but all sharing the core elements of stem cell-based coursework, hands-on-training through internships at world-class laboratories or biotechnology companies, and formal activities involving patient engagement and community outreach. Every year, the programs produce up to 140 well-rounded, highly skilled individuals that are ready to hit the ground running.

Poster presentations at the Bridges conference

Each July, the most recent cohort of Bridges trainees gather for an Annual Conference to share their research outcomes, network with their peers, and learn more about the current opportunities and challenges facing the regenerative medicine community.

This year, the 10th Annual Bridges Conference was held in San Mateo, CA and included inspiring talks from scientists performing cutting edge research and running some of the first FDA-approved stem-cell based clinical trials in the state.

Anna Simos

Perhaps the biggest highlights were hearing the real-life stories of brave individuals like Anna Simos, whose experience with life-threatening complications from diabetes inspired her life’s work of providing hope and education to those facing similar challenges.

Byron Jenkins

Equally moving was the testimonial of Byron Jenkins, a multiple myeloma patient who received an experimental new CAR-T therapy in a CIRM-supported clinical trial sponsored by Poseida Therapeutics.

Ronnie Kashyup with parents Upasana and Pawash

Last but not least, little Ronnie Kashyup, recently cured of Bubble Baby Disease through another CIRM-funded clinical trial, charmed all attendees with his larger-than-life personality while his father, Pawash Priyank, shared the story of Ronnie’s diagnosis and treatment.

In the video segments to follow:

CIRM Bridges student Sneha Santosh at San Jose State University discusses the role CIRM plays in bridging together the patient advocates with the groundbreaking research conducted by scientists.

Samori Dobson and Esther Nair, CIRM Bridges students at California State University, San Marcos, briefly discuss the positive impact that the program has had on their lives.

Below are some pictures form the 10th Annual Bridges Conference in San Mateo, CA.

For more information about CIRM Bridges Programs, see the following link and video below:

CIRM-funded internship programs

CIRM board member Lauren Miller Rogen appointed to California Alzheimer’s Task Force

Lauren Miller Rogen, Hilarity for Charity co-founder and CIRM Board Member

California has the largest aging population in the United States. The U.S. Census Bureau has estimated that one in five Californians will be 65 or older by the year 2030. Unfortunately with age comes a wide of health related issues that can arise such as Alzheimer’s.

Alzheimer’s is caused by changes in the brain that affect memory and thinking skills. The disease can progress to the point where carrying out the simplest tasks become quite a challenge. In the United States alone, 5.8 million people are living with Alzheimer’s, 630,000 of whom live in California. By 2050, the number of people with Alzheimer’s in the United States is expected to increase to almost 14 million.

To address this growing problem, California Governor Gavin Newsom announced the creation of a California Alzheimer’s Task Force comprised of scientists, politicians, and other individuals dedicated to addressing the needs of the Alzheimer’s community and the impact the disease has on California. The new task force has been tasked with releasing a report on the disease and ways to address the challenges it poses by 2020.

One of these task force members is our very own Lauren Miller Rogen, who is a dedicated member of our governing Board and the co-founder of Hilarity for Charity, a charity organization that raises awareness about and funds for research into Alzheimer’s. In addition to her advocacy work, Lauren is also a screenwriter and actress, staring alongside her husband Seth Rogen in movies such as 50/50 and Superbad.

“I’m so honored to join the Task Force to fight for the 670,000 Californians currently living with Alzheimer’s and for those who care for them,” Miller Rogen said. “This is a tremendous and diverse group who intend to create and propose real ideas to change the course of this disease.”

For Lauren, her journey towards becoming an advocate for Alzheimer’s is a very personal one. Her grandfather died of Alzheimer’s when she was just 12 years old and her grandmother died of the disease six years after that. Now, her mother is struggling with Alzheimer’s, having been diagnosed at the age of 55.

You can read more about Lauren’s story on a previous blog post.

CIRM have given awards totaling over $56 million throughout the years dedicated towards Alzheimer’s related research.

A future scientist’s journey

All this week we have been highlighting blogs from our SPARK (Summer Program to Accelerate Regenerative medicine Knowledge) students. SPARK gives high school students a chance to spend their summer working in a world class stem cell research facility here in California. In return they write about their experiences and what they learned.

The standard for blogs this year was higher than ever, so choosing a winner was particularly tough. In the end we chose Abigail Mora, who interned at UC San Francisco. We felt the obstacles she overcame in getting to this point made her story all the more remarkable and engaging.

Abigail Mora

When I was 15, my mother got sick and went to several doctors. Eventually, she found out that she was pregnant with a 3-month-old baby. A month after, my mom fell from the stairs, which were not high but still dangerous. Luckily, everything seemed to be okay with the baby. In the last week of her six-month pregnancy, she went in the clinic for a regular check-up but she ended up giving birth to my brother, who was born prematurely. She stayed in the clinic for a month and my brother also had to stay so that his lungs could develop properly.

When he came home, I was so happy. I spent a lot of time with him and was like his second mom. After an initial period of hard time, he grew into a healthy kid. Then I moved to San Francisco with my aunt, leaving my parents and siblings in Mexico so that I could become a better English speaker and learn more about science. My experience with my brother motivated me to learn more about the condition of premature babies, since there are many premature babies who are not as fortunate. I want to study neurodevelopment in premature kids, and how it may go wrong.

I was so happy when I got into the SEP High School Program, which my chemistry teacher introduced me to, and I found the research of Eric Huang’s lab at UCSF about premature babies and stem cell development in the brain super interesting. I met Lakisha and Jean, and they introduced me to the lab and helped me walk through the training process.

My internship experience was outstanding: I enjoyed doing research and how my mentor Jiapei helped me learn new things about the brain. I learned that there are many different cell types in the brain, like microglia, progenitor cells, and intermediate progenitors.

As all things in life can be challenging, I was able to persevere with my mentor’s help. For example, when I first learned how to cut mouse brains using a cryostat, I found it hard to pick up the tissue onto slides. After practicing many times, I became more familiar with the technique and my slices got better. Another time, I was doing immunostaining and all the slices fell from the slide because we didn’t bake the slides long enough. I was sad, but we learned from our mistakes and there are a lot of trials and errors in science.

I’ve also learned that in science, since we are studying the unknown, there is not a right or wrong answer. We use our best judgement to draw conclusions from what we observe, and we repeat the experiment if it’s not working.

The most challenging part of this internship was learning and understanding all the new words in neuroscience. Sometimes, I got confused with the abbreviations of these words. I hope in the future I can explain as well as my mentor Jiapei explained to me.

My parents are away from me but they support me, and they think that this internship will open doors to better opportunities and help me grow as a person.

I want to become a researcher because I want to help lowering the risk of neurodevelopmental disorders in premature babies. Many of these disorders, such as autism or schizophrenia, don’t have cures. These are some of the hardest diseases to cure because people aren’t informed about them and not enough research has been done. Hopefully, one day I can work on developing a cure for these disorders.

CIRM’s Stephen Lin, PhD, who heads the SPARK program and Abigail after her blog won first prize

Mind altering, life changing experience in stem cell lab

This week we are featuring the best blogs from our SPARK (Summer Program to Accelerate Regenerative medicine Knowledge) students. SPARK gives high school students a chance to spend their summer working in a world class stem cell research facility here in California. In return they write about their experiences and what they learned.

The blog that won second place comes from Emily Bunnapradist who spent her summer at Cedars-Sinai Medical Center in Los Angeles.

Emily Bunnapradist by the poster presentation of her work

When I was in the third grade, my mom took me to the allergy wing in the UCLA Medical Center, hoping to find answers to a number of issues that accompanied my seemingly never-ending list of food allergies: dairy, eggs, nuts, legumes, and so on. Unexpectedly, without even an appointment, clinician Dr. Braskett spent an hour out of her already busy schedule just talking us through our worries in the lobby, checking out skin problems that arose as a result of my allergies and promising to see us again as soon as she could. Because of her overwhelming kindness and generosity, my mom and I went home with relieved smiles and assurance that my health concerns were manageable.

That was the day that I decided that I wanted to pursue medicine, to make an impact on people the way that she had on my family and me. However, my conception of the field of healthcare was quite limited. For the majority of my life, I was convinced that the only way to make a true connection in a patient’s well-being was as a clinician.

This unfounded claim quickly changed when I was accepted into the CIRM SPARK program at Cedars-Sinai. In the most action-packed summer I have ever had the opportunity to experience, I was exposed to the diverse field of healthcare. Transitioning between the clinical and research aspects of science, I saw firsthand the direct effect that researchers had on patients in fields I had not even considered.

While touring the blood transfusion facility at Cedars-Sinai, a technician proudly boasted about her connection to patient care in labeling and testing blood donations to ensure they were suitable for those in need. Upon viewing the imaging core, the manager of the center informed us about the revolutionary advances his team was making in developing software to identify cancerous indicators in patients. In visiting the microbiology lab, multiple lab scientists informed us about the hundreds of tests they perform on a daily basis to detect diseases such as influenza and adenovirus, without which clinicians wouldn’t be able to perform their job to the fullest degree.

In these past weeks, I have spent hundreds of hours in the lab. From drawing on sections with hydrophobic markers to loading gels with protein samples, I have gained tremendous experience in navigating a research environment. However, although I now know the mechanics of Western blots and immunostaining like the back of my hand, the most essential takeaways for me are not learning the procedures but understanding their applications. While I am now able to pipette fluids with a steady hand and make buffer solutions without second-guessing my calculations, I am also able to appreciate the science behind each protein band and cell plate. Being able to contribute to my project and hear about my peers’ experiments has shown me the scope of influence research can have on extending knowledge and generating cures to diseases.

While I had initially considered research to be cold and isolating, I have found more warmth and connection here than I believed possible. The passion that my mentors possess for their line of work, as well as their endless knowledge on essentially any topic imaginable, has shown me the importance and integrity of what they do.

The CIRM SPARK students at Cedars-Sinai (Emily is front right): Photo courtesy Cedars-Sinai

I could not be more grateful to have the guidance of Dr. Mehrnoosh Ghiam and Dr. Adam Poe, who I have formed strong relationships with and have helped me accomplish what I have this summer. Their mentorship, along with the resources of Cedars-Sinai, have granted me the most productive and exciting summer I’ve had yet!

Time and money and advancing stem cell research

The human genome

Way back in the 1990’s scientists were hard at work decoding the human genome, trying to map and understand all the genes that make up people. At the time there was a sense of hope, a feeling that once we had decoded the genome, we’d have cures for all sorts of things by next Thursday. It didn’t quite turn out that way.

The same was true for stem cell research. In the early days there was a strong feeling that this was going to quite quickly produce new treatments and cures for diseases ranging from Parkinson’s and Alzheimer’s to heart disease and stroke. Although we have made tremendous strides we are still not where we hoped we’d be.

It’s a tough lesson to learn, but an important one: good scientific research moves at its own pace and pays little heed to our hopes or desires. It takes time, often a long time, and money, usually a lot of money, to develop new treatments for deadly diseases and disorders.

Many people, particularly those battling deadly diseases who are running out of time, are frustrated at the slow pace of stem cell research, at the years and years of work that it takes to get even the most promising therapy into a clinical trial where it can be tested in people. That’s understandable. If your life is on the line, it’s difficult to be told that you have to be patient. Time is a luxury many patients don’t have.

But that caution is necessary. The last thing we want to do is rush to test something in people that isn’t ready. And stem cells are a whole new way of treating disease, using cells that may stay in the body for years, so we really need to be sure we have done everything we can to ensure they are safe before delivering them to people.

The field of gene therapy was set back years after one young patient, Jesse Gelsinger, died as a result of an early experimental treatment. We don’t want the same to happen to stem cell research.

And yet progress is being made, albeit not as quickly as any of us would like. At the end of the first ten years of CIRM’s existence we had ten projects that we supported that were either in, or applying to be in, a clinical trial sanctioned by the US Food and Drug Administration (FDA). Five years later that number is 56.

Most of those are in Phase 1 or 2 clinical trials which means they are still trying to show they are both safe and effective enough to be made available to a wider group of people. However, some of our projects are in Phase 3, the last step before, hopefully, being given FDA approval to be made more widely available and – just as important – to be covered by insurance.

Other CIRM-funded projects have been given Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA, a new program that allows projects that show they are safe and benefit patients in early stage clinical trials, to apply for priority review, meaning they could get approved faster than normal. Out of 40 RMAT designations awarded so far, six are for CIRM projects.

We are working hard to live up to our mission statement of accelerating stem cell treatments to patients with unmet medical needs. We have been fortunate in having $3 billion to spend on advancing this research in California; an amount no other US state, indeed few other countries, have been able to match. Yet even that amount is tiny compared to the impact that many of these diseases have. For example, the economic cost of treating diabetes in the US is a staggering $327 billion a year.

The simple truth is that unless we, as a nation, invest much more in scientific research, we are not going to be able to develop cures and new, more effective, treatments for a wide range of diseases.

Time and money are always going to be challenging when it comes to advancing stem cell research and bringing treatments to patients. With greater knowledge and understanding of stem cells and how best to use them we can speed up the timeline. But without money none of that can happen.

Our blog is just one of many covering the topic of “What are the hurdles impacting patient access to cell and gene therapies as part of Signal’s fourth annual blog carnival.

Next generation of stem cell scientists leave their mark

One of the favorite events of the year for the team here at CIRM is our annual SPARK (Summer Program to Accelerate Regenerative Medicine Knowledge) conference. This is where high school students, who spent the summer interning at world class stem cell research facilities around California, get to show what they learned. It’s always an engaging, enlightening, and even rather humbling experience.

The students, many of whom are first generation Californians, start out knowing next to nothing about stem cells and end up talking as if they were getting ready for a PhD. Most say they went to their labs nervous about what lay ahead and half expecting to do menial tasks such as rinsing out beakers. Instead they were given a lab coat, safety glasses, stem cells and a specific project to work on. They learned how to handle complicated machinery and do complex scientific experiments.

But most importantly they learned that science is fun, fascinating, frustrating sometimes, but also fulfilling. And they learned that this could be a future career for them.

We asked all the students to blog about their experiences and the results were extraordinary. All talked about their experiences in the lab, but some went beyond and tied their internship to their own lives, their past and their hopes for the future.

Judging the blogs was a tough assignment, deciding who is the best of a great bunch wasn’t easy. But in the end, we picked three students who we thought captured the essence of the SPARK program. This week we’ll run all those blogs.

We begin with our third place blog by Dayita Biswas from UC Davis.

Personal Renaissance: A Journey from Scientific Curiosity to Confirmed Passions

By Dayita Biswas

As I poured over the pages of my battered Campbell textbook, the veritable bible for any biology student, I saw unbelievable numbers like how the human body is comprised of over 30 trillion cells! Or how we have over 220 different types of cells— contrary to my mental picture of a cell as a circle. Science, and biology in particular, has no shortage of these seemingly impossible Fermi-esque statistics that make one do a double-take. 

My experience in science had always been studying from numerous textbooks in preparation for a test or competitions, but textbooks only teach so much. The countless hours I spent reading actually demotivated me and I constantly asked myself what was the point of learning about this cycle or that process — the overwhelming “so what?” question. Those intriguing numbers that piqued my interest were quickly buried under a load of other information that made science a static stream of words across a page. 

That all changed this summer when I had the incredible opportunity to work in the Nolta lab under my mentor, Whitney Cary. This internship made science so much more tangible and fun to be a part of.  It was such an amazing environment, being in the same space with people who all have the same goals and passion for science that many high school students are not able to truly experience. Everyone was so willing to explain what they were doing, and even went out of their way to help if I needed papers or had dumb questions.

This summer, my project was to create embryoid bodies and characterize induced pluripotent stem cells (iPSCs) from children who had Jordan’s Syndrome, an extremely rare neurodevelopmental disease whose research has applications in Alzheimer’s and autism.

 I had many highs and lows during this research experience. My highs were seeing that my iPSCs were happy and healthy. I enjoyed learning lab techniques like micro-pipetting, working in a biological safety hood, feeding, freezing, and passaging cells. My lows were having to bleach my beloved iPSCs days after they failed to survive, and having unsuccessful protocols. However, while my project consistently failed, these failures taught me more than my successes.

I learned that there is a large gap between being able to read about techniques and being “book smart” and actually being able to think critically about science and perform research. Science, true science, is more than words on a page or fun facts to spout at a party. Science is never a straight or easy answer, but the mystery and difficulty is part of the reason it is so interesting. Long story short: research is hard and it takes time and patience, it involves coming in on weekends to feed cells, and staying up late at night reading papers.         

The most lasting impact that this summer research experience had was that everything we learn in school and the lab are all moving us towards the goal of helping real people. This internship renewed my passion for biology and cemented my dream of working in this field. It showed me that I don’t have to wait to be a part of dynamic science and that I can be a small part of something that will change, benefit, and save lives.

This internship meant being a part of something bigger than myself, something meaningful. We must always think critically about what consequences our actions will have because what we do as scientists and researchers— and human beings will affect the lives of real people. And that is the most important lesson anyone can hope to learn.

                                                                                                   

And here’s a bonus, a video put together by the SPARK students at Cedars-Sinai Medical Center.

Boosting the blood system after life-saving therapy

Following radiation, the bone marrow shows nearly complete loss of blood cells in mice (left). Mice treated with the PTP-sigma inhibitor displayed rapid recovery of blood cells (purple, right): Photo Courtesy UCLA

Chemotherapy and radiation are two of the front-line weapons in treating cancer. They can be effective, even life-saving, but they can also be brutal, taking a toll on the body that lasts for months. Now a team at UCLA has developed a therapy that might enable the body to bounce back faster after chemo and radiation, and even make treatments like bone marrow transplants easier on patients.

First a little background. Some cancer treatments use chemotherapy and radiation to kill the cancer, but they can also damage other cells, including those in the bone marrow responsible for making blood stem cells. Those cells eventually recover but it can take weeks or months, and during that time the patient may feel fatigue and be more susceptible to infections and other problems.

In a CIRM-supported study, UCLA’s Dr. John Chute and his team developed a drug that speeds up the process of regenerating a new blood supply. The research is published in the journal Nature Communications.

They focused their attention on a protein called PTP-sigma that is found in blood stem cells and acts as a kind of brake on the regeneration of those cells. Previous studies by Dr. Chute showed that, after undergoing radiation, mice that have less PTP-sigma were able to regenerate their blood stem cells faster than mice that had normal levels of the protein.

John Chute: Photo courtesy UCLA

So they set out to identify something that could help reduce levels of PTP-sigma without affecting other cells. They first identified an organic compound with the charming name of 6545075 (Chembridge) that was reported to be effective against PTP-sigma. Then they searched a library of 80,000 different small molecules to find something similar to 6545075 (and this is why science takes so long).

From that group they developed more than 100 different drug candidates to see which, if any, were effective against PTP-sigma. Finally, they found a promising candidate, called DJ009. In laboratory tests DJ009 proved itself effective in blocking PTP-sigma in human blood stem cells.

They then tested DJ009 in mice that were given high doses of radiation. In a news release Dr. Chute said the results were very encouraging:

“The potency of this compound in animal models was very high. It accelerated the recovery of blood stem cells, white blood cells and other components of the blood system necessary for survival. If found to be safe in humans, it could lessen infections and allow people to be discharged from the hospital earlier.”

Of the radiated mice, most that were given DJ009 survived. In comparison, those that didn’t get DJ009 died within three weeks.

They saw similar benefits in mice given chemotherapy. Mice with DJ009 saw their white blood cells – key components of the immune system – return to normal within two weeks. The untreated mice had dangerously low levels of those cells at the same point.

It’s encouraging work and the team are already getting ready for more research so they can validate their findings and hopefully take the next step towards testing this in people in clinical trials.

Drug used to treat multiple sclerosis may improve glioblastoma outcomes

Dr. Jeremy Rich, UC San Diego

Glioblastoma is an aggressive form of cancer that invades brain tissue, making it extremely difficult to treat. Current therapies involving radiation and chemotherapy are effective in destroying the bulk of brain cancer cells, but they are not able to reach the brain cancer stem cells, which have the ability to grow and multiply indefinitely. These cancer stem cells enable the glioblastoma to continuously grow even after treatment, which leads to recurring tumor formation.

Dr. Jeremy Rich and his team at UC San Diego examined glioblastomas further by obtaining glioblastoma tumor samples donated by patients that underwent surgery and implanting these into mice. Dr. Rich and his team tested a combinational treatment that included a targeted cancer therapy alongside a drug named teriflunomide, which is used to treatment patients with multiple sclerosis. The research team found that this approach successfully halted the growth of glioblastoma stem cells, shrank the tumor size, and improved survival in the mice.

In order to continue replicating, glioblastoma stem cells make pyrimidine, one of the compounds that make up DNA. Dr. Rich and his team noticed that higher rates of pyrimidine were associated with poor survival rates in glioblastoma patients. Teriflunomide works by blocking an enzyme that is necessary to make pyrmidine, therefore inhibiting glioblastoma stem cell replication.

In a press release, Dr. Rich talks about the potential these findings hold by stating that,

“We’re excited about these results, especially because we’re talking about a drug that’s already known to be safe in humans.”

However, he comments on the need to evaluate this approach further by saying that,

“This laboratory model isn’t perfect — yes it uses human patient samples, yet it still lacks the context a glioblastoma would have in the human body, such as interaction with the immune system, which we know plays an important role in determining tumor growth and survival. Before this drug could become available to patients with glioblastoma, human clinical trials would be necessary to support its safety and efficacy.”

The full results to this study were published in Science Translational Medicine.

Stem cell progress and promise in fighting leukemia

Computer illustration of a cancerous white blood cell in leukemia.

There is nothing you can do to prevent or reduce your risk of leukemia. That’s not a very reassuring statement considering that this year alone almost 62,000 Americans will be diagnosed with leukemia; almost 23,000 will die from the disease. That’s why CIRM is funding four clinical trials targeting leukemia, hoping to develop new approaches to treat, and even cure it.

That’s also why our next special Facebook Live “Ask the Stem Cell Team” event is focused on this issue. Join us on Thursday, August 29th from 1pm to 2pm PDT to hear a discussion about the progress in, and promise of, stem cell research for leukemia.

We have two great panelists joining us:

Dr. Crystal Mackall, has many titles including serving as the Founding Director of the Stanford Center for Cancer Cell Therapy.  She is using an innovative approach called a Chimeric Antigen Receptor (CAR) T Cell Therapy. This works by isolating a patient’s own T cells (a type of immune cell) and then genetically engineering them to recognize a protein on the surface of cancer cells, triggering their destruction. This is now being tested in a clinical trial funded by CIRM.

Natasha Fooman. To describe Natasha as a patient advocate would not do justice to her experience and expertise in fighting blood cancer and advocating on behalf of those battling the disease. For her work she has twice been named “Woman of the Year” by the Leukemia and Lymphoma Society. In 2011 she was diagnosed with a form of lymphoma that was affecting her brain. Over the years, she would battle lymphoma three times and undergo chemotherapy, radiation and eventually a bone marrow transplant. Today she is cancer free and is a key part of a CIRM team fighting blood cancer.

We hope you’ll join us to learn about the progress being made using stem cells to combat blood cancers, the challenges ahead but also the promising signs that we are advancing the field.

We also hope you’ll take an active role by posting questions on Facebook during the event, or sending us questions ahead of time to info@cirm.ca.gov. We will do our best to address as many as we can.

Here’s the link to the event, feel free to share this with anyone you think might be interested in joining us for Facebook Live “Ask the Stem Cell Team about Leukemia”