Treatments, cures and clinical trials: an in-person update on CIRM’s progress

Patients and Patient Advocates are at the heart of everything we do at CIRM. That’s why we are holding three free public events in the next few months focused on updating you on the stem cell research we are funding, and our plans for the future.

Right now we have 33 projects that we have funded in clinical trials. Those range from heart disease and stroke, to cancer, diabetes, ALS (Lou Gehrig’s disease), two different forms of vision loss, spinal cord injury and HIV/AIDS. We have also helped cure dozens of children battling deadly immune disorders. But as far as we are concerned we are only just getting started.

Over the course of the next few years, we have a goal of adding dozens more clinical trials to that list, and creating a pipeline of promising therapies for a wide range of diseases and disorders.

That’s why we are holding these free public events – something we try and do every year. We want to let you know what we are doing, what we are funding, how that research is progressing, and to get your thoughts on how we can improve, what else we can do to help meet the needs of the Patient Advocate community. Your voice is important in helping shape everything we do.

The first event is at the Gladstone Institutes in San Francisco on Wednesday, September 6th from noon till 1pm. The doors open at 11am for registration and a light lunch.

Gladstone Institutes

Here’s a link to an Eventbrite page that has all the information about the event, including how you can RSVP to let us know you are coming.

We are fortunate to be joined by two great scientists, and speakers – as well as being CIRM grantees-  from the Gladstone Institutes, Dr. Deepak Srivastava and Dr. Steve Finkbeiner.

Dr. Srivastava is working on regenerating heart muscle after it has been damaged. This research could not only help people recover from a heart attack, but the same principles might also enable us to regenerate other organs damaged by disease. Dr. Finkbeiner is a pioneer in diseases of the brain and has done ground breaking work in both Alzheimer’s and Huntington’s disease.

We have two other free public events coming up in October. The first is at UC Davis in Sacramento on October 10th (noon till 1pm) and the second at Cedars-Sinai in Los Angeles on October 30th (noon till 1pm). We will have more details on these events in the coming weeks.

We look forward to seeing you at one of these events and please feel free to share this information with anyone you think might be interested in attending.

High school students SPARK an interest in stem cell research

SPARK students at the 2017 Annual Meeting at the City of Hope.

High school is a transformative time for any student. It marks the transition from childhood to adulthood and requires discipline, dedication and determination to excel and get into their desired college or university.

The barrier to entry for college now seems much higher than when I was eighteen, but I am not worried for the current generation of high school students. That’s because I’ve met some of the brightest young minds this past week at the 2017 CIRM SPARK meeting.

SPARK is CIRM’s high school education program, which gives underprivileged students in California the opportunity to train as stem cell scientists for the summer. Students participate in a summer research internship at one of seven programs at leading research institutes in the state. They attend scientific lectures, receive training in basic lab techniques, and do an eight-week stem cell research project under the guidance of a mentor.

At the end of the summer, SPARK students congregate at the annual SPARK poster meeting where they present the fruits of their labor. Meeting these students in person is my favorite time of the year. Their enthusiasm for science and stem cell research is contagious. And when you engage them or listen to them talk about their project, it’s hard to remember that they are still teenagers and not graduate level scientists.

What impresses me most about these students is their communication skills. Each summer, I challenge SPARK students to share their summer research experience through social media and blogging, and each time they go above and beyond with their efforts. Training these students as effective science communicators is important to me. They are the next generation of talented scientists who can help humanize research for the public. They have the power to change the perception of science as a field to be embraced and one that should receive proper funding.

It’s also inspiring to me that this young generation can effectively educate their friends, family and the public about the importance of stem cell research and how it will help save the lives of patients who currently don’t have effective treatments. If you haven’t already, I highly recommend checking out the #CIRMSPARKlab hashtag on Instagram to get a taste of what this year’s group of students accomplished during their internships.

Asking students, many of whom are learning to do research for the first time, to post on Instagram once a week and write a blog about their internship is a tall task. And I believe with any good challenge, there should be a reward. Therefore, at this year’s SPARK meeting held at the City of Hope in Duarte, California, I handed out prizes.

It was very difficult to pick winners for our presentation, social media and blogging awards because honestly, all our students were excellent this year. Even Kevin McCormack, Director of CIRM’s Communications, who helped me read the students’ blogs said,

“This was really tough. The standard of the blogs this year was higher than ever; and previous years had already set the bar really high. It was really difficult deciding which were really good and which were really, really good.”

Ok, enough with the hype, I know you want to read these award-winning blogs so I’ve shared them below. I hope that they inspire you as much as they have inspired me.


Amira Hirara

Amira Hirara (Children’s Hospital Oakland Research Institute)

It was a day like any other. I walked into the room, just two minutes past 10:30am, ready for another adventurous day in the lab. Just as I settle down, I am greeted by my mentor with the most terrifying task I have ever been asked to perform, “Will you passage the cells for me…alone?” Sweat begins to pour down my cemented face as I consider what is at stake.

The procedure was possibly thirty steps long and I have only executed it twice, with the supervision of my mentor of course. To be asked to do the task without the accompaniment of an experienced individual was unthought-of. I feel my breath begin to shorten as I mutter the word “Ok”. Yet it wasn’t just the procedure that left me shaking like a featherless bird, it was the location of my expedition as well. The dreaded tissue culture room. If even a speck of dirt enters the circulating air of the biosafety cabinet, your cells are at risk of death…death! I’ll be a cell murderer. “Alright”, she said, “I’ll just take a look at the cells then you’ll be on your way.” As we walk down the hallway, my eyes began to twitch as I try to recall the first steps of the procedure. I remember freezing our plates with Poly-ornithine and laminin, which essentially simulates the extracellular environment and allows adhesion between the cell and the plate itself. I must first add antibiotics to rid the frozen plate of potential bacteria. Then I should remove my cells from the incubator, and replace the old solution with accutase and new media, to nourish the cells, as well as unbind them from the plate before. Passaging is necessary when the cell density gets too high, as the cells must be relocated to a roomier environment to better promote survival. As we approach the tissue culture room, my jaw unclenches, as I realize the whirlwind of ideas meant I know more than I thought. My mentor retrieves our cells, views them under the microscope, and deems them ‘ready for passaging’.

“Good luck Amira” she says to me with a reassuring smile. I enter the room ready for battle. Placing first my gloves and coat, I then spray my hands and all things placed in the cabinet with 70% ethanol, to insure a sterile work environment. Back to the procedure, I’ll place the cellular solution of accutase and media into a covalent tube. After, I’ll centrifuge it for two minutes until a cellular pellet forms at the bottom, then dissolve the cells in fresh media, check its density using a cell counter, and calculate the volume of cellular solution needed to add to my once frozen plates. Wait, once I do that, I’ll be all done. I eagerly execute all the steps, ensuring both accuracy and sterility in my work. Pride swells within me as I pipette my last milliliter of solution into my plate. The next day, my mentor and I stop by to check on how our sensitive neural stem cells are doing. “Wow Amira, I am impressed, your cells seem very confluent in their new home, great job!” I smile slyly and begin to nod my head. I now walk these hallways, with a puffed chest, brightened smile, and eagerness to learn. My stem cells did not die, and having the amazing opportunity to master their treatment and procedures, is something I can never forget.

 

Gaby Escobar

Gaby Escobar (Stanford University)

Walking into the lab that would become my home for the next 8 weeks, my mind was an empty canvas.  Up to that point, my perception of the realm of scientific research was one-sided. Limited to the monotonous textbook descriptions of experiments that were commonplace in a laboratory, I wanted more. I wanted to experience the alluring call of curiosity. I wanted to experience the flash of discovery and the unnerving drive that fueled our pursuit of the unknown. I was an empty canvas looking for its first artistic stroke.

Being part of the CIRM Research program, I was lucky enough to have been granted such opportunity. Through the patient guidance of my mentor, I was immersed into the limitless world of stem cell biology. From disease modeling to 3D bioprinting, I was in awe of the capabilities of the minds around me. The energy, the atmosphere, the drive all buzzed with an inimitable quest for understanding. It was all I had imagined and so, so much more.

However, what many people don’t realize is research is an arduous, painstaking process. Sample after sample day after day, frustration and doubt loomed above our heads as we tried to piece together a seemingly pieceless puzzle.  Inevitably, I faced the truth that science is not the picture-perfect realm I had imagined it to be. Rather, it is tiring, it is relentless, and it is unforgiving. But at the same time, it is incomparably gratifying. You see, the innumerable samples, the countless gels and PCRS, all those futile attempts to fruitlessly make sense of the insensible, have meaning. As we traversed through the rollercoaster ride of our project, my mentor shared a personal outlook that struck very deeply with me: her motivation to work against obstacle after obstacle comes not from the recognition or prestige of discovering the next big cure but rather from the notion that one day, her perseverance may transform someone’s life for the good.  And in that, I see the beauty of research and science: the coming together of minds and ideas and bewildering intuitions all for the greater good.

As I look back, words cannot express the gratitude I feel for the lessons I have learned. Undoubtedly, I have made countless mistakes (please don’t ask how many gels I’ve contaminated or pipettes I have dropped) but I’ve also created the most unforgettable of memories. Memories that I know I will cherish for the journey ahead of me. Having experienced the atmosphere of a vibrant scientific community, I have found a second home, a place that I can explore and question and thrive. And although not every day will hold the cure to end all diseases or hand an answer on a silver platter, every day is another opportunity.  And with that, I walk away perhaps not with the masterpiece of art that I had envisioned in my mind but rather with a burning spark of passion, ready to ignite.

 

Anh Vo

Ahn Vo (UC Davis)

With college selectivity increasing and acceptance rates plummeting, the competitive nature within every student is pushed to the limit. In high school, students are expected to pad up their resumes and most importantly, choose an academic path sooner rather than later. However, at 15, I felt too young to experience true passion for a field. As I tried to envision myself in the future, I wondered, would I be someone with the adrenaline and spirit of someone who wants to change the world or one with hollow ambitions, merely clinging onto a paycheck with each day passing? At the very least, I knew that I didn’t want to be the latter.

The unrelenting anxiety induced by the uncertainty of my own ambitions was intoxicating. As my high school career reached its halfway mark, I felt the caving pressure of having to choose an academic path.

“What do you want to be?” was one of the first questions that my mentor, Whitney Cary, asked me. When I didn’t have an answer, she assured me that I needed to keep my doors open, and the SPARK program was the necessary first step that I needed to take to discovering my passion.

As I reflected on my experience, the SPARK program was undoubtedly the “first step”. It was the first step into a lab and above all, into a community of scientists, who share a passion for research and a vehement resolve to contribute to scientific merit. It was the integration into a cohort of other high school students, whose brilliance and kindness allowed us to forge deeper bonds with each other that we will hold onto, even as we part ways. It was the first nervous step into the bay where I met the Stem Cell Core, a team, whose warm laughter and vibrancy felt contagious. Finally, it was the first uncertain stumble into the tissue culture room, where I conceived a curiosity for cell culture that made me never stop asking, “Why?”

With boundless patience, my mentor and the Stem Cell Core strove to teach me techniques, such as immunocytochemistry and continually took the time out of their busy day to reiterate concepts. Despite my initial blunders in the hood, I found myself in a place without judgement, and even after discouraging incidents, I felt a sense of consolation in the witty and good-humored banter among the Stem Cell Core. At the end of every day, the unerring encouragement from my mentor strengthened my resolve to continue improving and incited an earnest excitement in me for the new day ahead. From trembling hands, nearly tipping over culture plates and slippery gloves, overdoused in ethanol, I eventually became acquainted with daily cell culture, and most importantly, I gained confidence and pride in my work.

I am grateful to CIRM for granting me this experience that has ultimately cultivated my enthusiasm for science and for the opportunity to work alongside remarkable people, who have given me new perspectives and insights. I am especially thankful to my mentor, whose stories of her career journey have inspired me to face the future with newfound optimism in spite of adversity.

As my internship comes to a close, I know that I have taken my “first step”, and with a revived mental acquisitiveness, I eagerly begin to take my second.

Other 2017 SPARK Awards

Student Speakers: Candler Cusato (Cedars-Sinai), Joshua Ren (Stanford)

Instagram/Social Media: Jazmin Aizpuru (UCSF), Emily Beckman (CHORI), Emma Friedenberg (Cedars-Sinai)

Poster Presentations: Alexander Escudero (Stanford), Jamie Kim (CalTech), Hector Medrano (CalTech), Zina Patel (City of Hope)


Related Links:

Family, faith and funding from CIRM inspire one patient to plan for his future

Caleb Sizemore speaks to the CIRM Board at the June 2017 ICOC meeting.

Having been to many conferences and meetings over the years I have found there is a really simple way to gauge if someone is a good speaker, if they have the attention of people in the room. You just look around and see how many people are on their phones or laptops, checking their email or the latest sports scores.

By that standard Caleb Sizemore is a spellbinding speaker.

Last month Caleb spoke to the CIRM Board about his experiences in a CIRM-funded clinical trial for Duchenne Muscular Dystrophy. As he talked no one in the room was on their phone. Laptops were closed. All eyes and ears were on him.

To say his talk was both deeply moving and inspiring is an understatement. I could go into more detail but it’s so much more powerful to hear it from  Caleb himself. His words are a reminder to everyone at CIRM why we do this work, and why we have to continue to do all that we can to live up to our mission statement and accelerate stem cell treatments to patients with unmet medical needs.

Video produced by Todd Dubnicoff/CIRM


Related Links:

Stem cell agency funds Phase 3 clinical trial for Lou Gehrig’s disease

ALS

At CIRM we don’t have a disease hierarchy list that we use to guide where our funding goes. We don’t rank a disease by how many people suffer from it, if it affects children or adults, or how painful it is. But if we did have that kind of hierarchy you can be sure that Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, would be high on that list.

ALS is a truly nasty disease. It attacks the neurons, the cells in our brain and spinal cord that tell our muscles what to do. As those cells are destroyed we lose our ability to walk, to swallow, to talk, and ultimately to breathe.

As Dr. Maria Millan, CIRM’s interim President and CEO, said in a news release, it’s a fast-moving disease:

“ALS is a devastating disease with an average life expectancy of less than five years, and individuals afflicted with this condition suffer an extreme loss in quality of life. CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs and, in keeping with this mission, our objective is to find a treatment for patients ravaged by this neurological condition for which there is currently no cure.”

Having given several talks to ALS support groups around the state, I have had the privilege of meeting many people with ALS and their families. I have seen how quickly the disease works and the devastation it brings. I’m always left in awe by the courage and dignity with which people bear it.

BrainStorm

I thought of those people, those families, today, when our governing Board voted to invest $15.9 million in a Phase 3 clinical trial for ALS run by BrainStorm Cell Therapeutics. BrainStorm is using mesenchymal stem cells (MSCs) that are taken from the patient’s own bone marrow. This reduces the risk of the patient’s immune system fighting the therapy.

After being removed, the MSCs are then modified in the laboratory to  boost their production of neurotrophic factors, proteins which are known to help support and protect the cells destroyed by ALS. The therapy, called NurOwn, is then re-infused back into the patient.

In an earlier Phase 2 clinical trial, NurOwn showed that it was safe and well tolerated by patients. It also showed evidence that it can help stop, or even reverse  the progression of the disease over a six month period, compared to a placebo.

CIRM is already funding one clinical trial program focused on treating ALS – that’s the work of Dr. Clive Svendsen and his team at Cedars Sinai, you can read about that here. Being able to add a second project, one that is in a Phase 3 clinical trial – the last stage before, hopefully, getting approval from the Food and Drug Administration (FDA) for wider use – means we are one step closer to being able to offer people with ALS a treatment that can help them.

Diane Winokur, the CIRM Board Patient Advocate member for ALS, says this is something that has been a long time coming:

CIRM Board member and ALS Patient Advocate Diane Winokur

“I lost two sons to ALS.  When my youngest son was diagnosed, he was confident that I would find something to save him.  There was very little research being done for ALS and most of that was very limited in scope.  There was one drug that had been developed.  It was being released for compassionate use and was scheduled to be reviewed by the FDA in the near future.  I was able to get the drug for Douglas.  It didn’t really help him and it was ultimately not approved by the FDA.

When my older son was diagnosed five years later, he too was convinced I would find a therapy.  Again, I talked to everyone in the field, searched every related study, but could find nothing promising.

I am tenacious by nature, and after Hugh’s death, though tempted to give up, I renewed my search.  There were more people, labs, companies looking at neurodegenerative diseases.

These two trials that CIRM is now funding represent breakthrough moments for me and for everyone touched by ALS.  I feel that they are a promising beginning.  I wish it had happened sooner.  In a way, though, they have validated Douglas and Hugh’s faith in me.”

These therapies are not a cure for ALS. At least not yet. But what they will do is hopefully help buy people time, and give them a sense of hope. For a disease that leaves people desperately short of both time and hope, that would be a precious gift. And for people like Diane Winokur, who have fought so hard to find something to help their loved ones, it’s a vindication that those efforts have not been in vain.

Emotions and gratitude at changing of the guard at Stem Cell Agency

RandyFarewellFamily

Randy Mills and his family

Randy, as regular readers of this blog know, is, or rather was, the President and CEO of CIRM. James Harrison is less well known to the outside world but his imprint on CIRM, as our General Counsel and one of the key figures behind Proposition 71, is even bigger than that of Randy’s.

Randy came to the stem cell agency a little over three years ago and in pretty quick order completely refashioned us. Under his guidance CIRM 2.0 became a sleek, streamlined funding machine, turning what had been an almost two-year process from application to funding into one that took just 120 days. He revamped the frequency with which we offered specific programs, making it more predictable and so easier for researchers to know when the next round was coming up. He helped usher in a new Strategic Plan that is a blueprint for us until 2020.

But the changes he implemented were not just about the way we worked, it was also about how we worked and particularly how we worked together. He turned the agency into a true team, one where everyone felt they not only had a role to play but that what they did was important in determining the success of the agency.

Not surprisingly there was no shortage of people ready to praise him. CIRM Board Chair Jonathan Thomas (JT) thanked Randy for turning the agency around, transforming it into an organization that even the National Institutes of Health (NIH) now looks to as a model (more on that in a subsequent blog). Vice Chair Art Torres thanked Randy for his leadership and for his compassion toward patients, always putting them first in everything that he and the agency did. Board member Sherry Lansing called Randy “a genius and visionary”.

But perhaps the most moving tributes came from patients advocates.

Don Reed said; “When I first met Randy I didn’t like him. I thought CIRM was one of the best, if not the best, organization out there and who was this person to say they were going to come in and make it better. Well, you did Randy and we are all so very grateful to you for that.”

Adrienne Shapiro from Axis Advocacy, an organization dedicated to finding a cure for sickle cell disease, presented Randy with the “Heart of a Mother” award, thanking him for his tireless support of patients and their families.

Jake Javier, a participant in the Asterias spinal cord injury trial, wrote a note saying: “You positively affect so many through your amazing funding efforts for life changing research, and should be very proud of that. But something I will always remember is how personal and genuine you were while doing it. I hope you got the chance to meet as many of the people you helped as possible because I know they would remember the same.”

Randy – who is leaving to become President/CEO of the National Marrow Donor/Be The Match program – was clearly deeply moved by the tributes, but reminded everyone that he was leaving us in good hands. The Board named Dr. Maria Millan as the interim President and CEO, pending a meeting of a search committee to determine the steps for appointing a permanent replacement.

Randy praised Maria for her intelligence, compassion and vision:

“Maria Millan has been a great partner in all that we have achieved at CIRM. She was a key part of developing the Strategic Plan; she  understands it inside out and has been responsible for administering it. She is a wonderful leader and is going to be absolutely phenomenal.”

JamesFarewell_1920x1080

James Harrison (left) with CIRM Board members Jonathan Thomas and Bert Lubin

The tributes for James Harrison were ever bit as moving. James has been a part of CIRM since before there was a CIRM. He helped draft Proposition 71, the ballot initiative that created the stem cell agency, and has played a key role since as General Counsel.

JT: “James has been a part of literally every decision and move that CIRM has made in its entire history. He’s been integral in everything. When I first came to CIRM, I was told by Bob Klein (JT’s predecessor as Chair) ‘Don’t brush your teeth without checking with James first’ suggesting a level of knowledge and expertise that was admirable.”

Jeff Sheehy “We would not be here without James. He organized the defense when we were sued by our opponents in the early days, through the various leadership challenges we had, all of the legal difficulties we had James was there to guide us and it’s been nothing short of extraordinary. Your brilliance and steadiness is amazing. While we are screaming and pulling our hair out there was James. Just saying his name makes me feel more relaxed.”

Sherry Lansing: “One thing I never worried about was our ethics, because you protected us at all times. You have such strong ethical values, you are always calm and rational and no matter what was going on you were always the rock who could explain things to everyone and deal with it with integrity.”

James is leaving to take a more active role in the law firm Remcho, Johansen & Purcell, where he is partner. Succeeding him as General Counsel is Scott Tocher, who has been at CIRM almost as long as James.

Randy; “To have someone like Scott come in and replace someone who wrote Proposition 71 speaks for the bench strength of the agency and how we are in very good hands.”

Art Torres joked “Scott has been waiting as long as Prince Charles has to take over the reins and we’re delighted to be able to work with him.”

We wish Randy and James great good luck in their next adventures.

 

Bridging the divide: stem cell students helping families with rare diseases become partners in research

Bridges & Rare Science

CIRM’s Bridges students and Rare Science’s families with rare diseases

Sometimes it’s the simplest things that make the biggest impact. For example, introducing a scientist to a patient can help them drive stem cell research forward faster than either one could do on their own.

Want proof? This year, students in CIRM’s Bridges to Stem Cell Research and Therapy program at California State University (CSU) San Marcos teamed up with parents of children with rare diseases, and the partnerships had a profound impact on all of them, one we hope might produce some long-term benefits.

Christina Waters, who helped create the partnerships, calls it “science with love.”

“We wanted to change the conversation and have researchers and families communicate, making families equal stakeholders in the research. The students bonded with the families and I truly feel that we made a difference in the lives of future researchers, in knowing how much their work can make a life changing impact on the lives of patients’ families who now have hope.”

The CIRM Bridges program helps prepare California’s undergraduate and master’s graduate students for highly productive careers in stem cell research. Students get a paid internship where they get hands-on training and education in stem cell research. They also work with patients and take part in outreach activities so they get an understanding of research that extends beyond the lab.

That’s where Christina Waters comes in. Christina is the founder of Rare Science, a non-profit group focused on rare diseases in children – we blogged about her work here – and she teamed up with CSU San Marcos to partner their Bridges students with five patient families with different rare diseases.

Cutting edge science

One of those families was Aaron Harding’s. Aaron’s son Jaxon has SYNGAP, a genetic disorder that can cause seizures, mental retardation, speech problems and autistic-like behavior. Two of the Bridges students who were doing their internship at ThermoFisher Scientific, Uju Nwizu and Emily Asbury, were given the task of using the gene-editing tool CRISPR Cas9 to help develop a deeper understanding of SYNGAP.

The students say it was an amazing experience:

Uju: “It had a huge impact on me. Every time I thought about SYNGAP I saw Jaxon’s face. This motivated me a lot.”

Emily: “People who work in labs everyday are most often working out the minutiae of research. They don’t often get a chance to see how their research can change or save the lives of real people. Meeting patients is so motivating because afterwards you aren’t just studying a mechanism, you now have a friend with the disease, so you can’t help but be personally invested in the search for a treatment.”

Emily and Uju are working to create iPSCs (induced pluripotent stem cells) that have the SYNGAP mutation. They hope these can be used to study the disease in greater depth and, maybe one day, lead to treatments for some of the symptoms.

Aaron says for families like his, knowing there are scientists working on his child’s disorder is a source of comfort, and hope:

“Personalizing diseases by connecting scientists with those they seek to impact is so important. Emily and Uju took this opportunity and ran with it, and that says a lot about them, and the team at ThermoFisher, taking on an exploring the unknown. That attitude is the heart of a scientist.”

Hearing stories like this is very gratifying, not just for the students and families involved, but for everyone here at CIRM. When we created the Bridges program our goal was to help students get the skills and experience needed to pursue a career in science. Thanks to the people at CSU San Marcos and Rare Science these students got a whole lot more.

Christina Waters: “We learned, we shared hope, we celebrated the courage of our families and the commitment of the students. It takes a village, and it is all of us working together that will make great changes for kids with rare diseases.”

For Uju and Emily, their experience in the Bridges program has made them doubly certain they want to pursue a career in science.

Uju: “I love stem cells and the promise they hold. After this program I hope to be part of a team that is committed to accelerating new stem cell therapies for rare and chronic diseases.”

Emily: “I’ve learned that I love research. After I finish my bachelor’s degree at CSU San Marcos I plan to pursue a graduate degree in molecular or cellular biology.”

 

School is out which means SPARK is in for the summer!

It’s mid-June, which means that school’s out for the summer! While most students are cheering about their newfound freedom from the classroom, a special group of high school students are cheering about the start of the CIRM SPARK internship program.

SPARK is CIRM’s high school educational program that gives students from underrepresented communities the opportunity to conduct stem cell research at top-notch universities in California. Students will spend the summer working in stem cell labs under the guidance and mentorship of scientists, PhDs, master’s students and postdocs. They will learn basic lab techniques like how to do PCR and how to grow stem cells.

Each student will have their own research project that answers an important question in the stem cell field. Students will also attend scientific lectures at their host university, participate in patient-centered activities and write blogs and social media posts about their experiences in the lab. At the end of the summer, they will show off their hard work through posters and talks at the annual SPARK conference.

SPARK gives students early exposure to research and proves to them that science is not only fun but is also a promising career option within their reach. We’ve offered a high school internship summer program for the past few years, and many students who’ve previously participated have told us that they are excited to pursue an education in science or medicine in college.

Ranya taking care of her stem cells!

I’ve featured some of these exciting success stories previously on our blog. One of these stellar students is Ranya Odeh. She was a student in the UC Davis SPARK program and recently told us that she will attend Stanford University to pursue bioengineering after receiving the prestigious QuestBridge scholarship. Another student we featured recently is Shannon Larsuel who participated in the Stanford SPARK program. Shannon was inspired after she worked at the Stanford bone marrow registry as part of her SPARK experience and now plans to be a pediatric oncologist.

Now that the 2017 SPARK program is in session, we can look forward to another exciting summer of talented and motivated students. Our SPARK students are encouraged to document their summer experiences on social media, so you’ll be able to follow their journeys on Instagram. Make sure to check out @CIRM_stemcells Instagram account and the #CIRMSPARKlab hashtag on both Instagram and Twitter.

If you’re a student or teacher who wants to learn more about the CIRM SPARK program, visit our website for more details. And with that, I’ll leave you with a few of the most recent Instagram posts from our new cohort of SPARK students!

Looking at our infected tissue cells!! 🔬🔬#CIRMSPARKLab

A post shared by monse mendoza (@mawnsay) on

Happy #workwednesday! I'm so excited that the @cirm_stemcells #cirmsparklab high school stem cell program has begun! It's a summer internship program where students from underrepresented communities do research in stem cell labs at universities in California. . These smiling students are part of the @uc_davis_stem_cells SPARK program led by Dr. Gerhard Bauer (left). To get into SPARK, they had to win the UC Davis #teenbiotechchallenge by creating a website about a specific science topic. . These students will spend two months doing stem cell research in a lab at UC Davis with grad student and postdoc mentors. At the end of the summer they will present their work at the CIRM spark conference. . I'm so excited for this year's new batch of students. They are posting pictures of their lab work on Instagram (see #cirmsparklab) and their enthusiasm for communicating their science is contagious. I'll be sharing more pictures from this program this summer! 👍🔬 . PS thanks to Dr. Jan Nolta from UC Davis for this photo and for her dedication to the SPARK program as a mentor and teacher!

A post shared by Dr. Karen Ring (@drkarenring) on

Newest member of CIRM Board is a fan of horses, Star Trek and Harry Potter – oh, and she just happens to be a brilliant cancer researcher too.

malkas-linda

An addition to the family is always a cause for celebration, whether it be a new baby, a puppy, or, in our case, a new Board member. That’s why we are delighted to welcome City of Hope’s Linda Malkas, Ph.D., as the newest member of the CIRM Board.

Dr. Malkas has a number of titles including Professor of Molecular and Cellular Biology at Beckman Research Institute; Deputy Director of Basic Research, Comprehensive Cancer Center, City of Hope; and joint head of the Molecular Oncology Program at the Cancer Center.

Her research focus is cancer and she has a pretty impressive track record in the areas of human cell DNA replication/repair, cancer cell biomarker and therapeutic target discovery. As evidence of that, she discovered a molecule that can inhibit certain activities in cancerous cells and hopes to move that into clinical trials in the near future.

California Treasure John Chiang made the appointment saying Dr. Malkas is “extraordinarily well qualified” for the role. It’s hard to disagree. She has a pretty impressive resume:

  • She served for five years on a National Cancer Institute (NCI) subcommittee reviewing cancer center designations.
  • She has served as chair on several NCI study panels and recently took on an advisory role on drug approval policy with the Food and Drug Administration.
  • She has published more than 75 peer-reviewed articles
  • She sits on the editorial boards of several high profile medical journals.

In a news release Dr. Malkas says she’s honored to be chosen to be on the Board:

“The research and technologies developed through this agency has benefited the health of not only Californians but the nation and world itself. I am excited to see what the future holds for the work of this agency.”

With all this in her work life it’s hard to imagine she has time for a life outside of the lab, and yet she does. She has four horses that she loves to ride – not all at the same time we hope – a family, friends, dogs and cats she likes spending time with. And as if that wasn’t enough to make you want to get to know her, she’s a huge fan of Star Trek, vintage sci-fi movies and Harry Potter.

Now that’s what I call a well-rounded individual. We are delighted to have her join the CIRM Team and look forward to getting her views on who are the greater villains, Klingons or Death Eaters.

 

Don’t Be Afraid: High school stem cell researcher on inspiring girls to pursue STEM careers

As part of our CIRM scholar blog series, we’re featuring the research and career accomplishments of CIRM funded students.

Shannon Larsuel

Shannon Larsuel is a high school senior at Mayfield Senior School in Pasadena California. Last summer, she participated in Stanford’s CIRM SPARK high school internship program and did stem cell research in a lab that studies leukemia, a type of blood cancer. Shannon is passionate about helping people through research and medicine and wants to become a pediatric oncologist. She is also dedicated to inspiring young girls to pursue STEM (Science, Technology, Engineering, and Mathematics) careers through a group called the Stem Sisterhood.

I spoke with Shannon to learn more about her involvement in the Stem Sisterhood and her experience in the CIRM SPARK program. Her interview is below.


Q: What is the Stem Sisterhood and how did you get involved?

SL: The Stem Sisterhood is a blog. But for me, it’s more than a blog. It’s a collective of women and scientists that are working to inspire other young scientists who are girls to get involved in the STEM field. I think it’s a wonderful idea because girls are underrepresented in STEM fields, and I think that this needs to change.

I got involved in the Stem Sisterhood because my friend Bridget Garrity is the founder. This past summer when I was at Stanford, I saw that she was doing research at Caltech. I reconnected with her and we started talking about our summer experiences working in labs. Then she asked me if I wanted to be involved in the Stem Sisterhood and be one of the faces on her website. She took an archival photo of Albert Einstein with a group of other scientists that’s on display at Caltech and recreated it with a bunch of young women who were involved in the STEM field. So I said yes to being in the photo, and I’m also in the midst of writing a blog post about my experience at Stanford in the SPARK program.

Members of The Stem Sisterhood

Q: What does the Stem Sisterhood do?

SL: Members of the team go to elementary schools and girl scout troop events and speak about science and STEM to the young girls. The goal is to inspire them to become interested in science and to teach them about different aspects of science that maybe are not that well known.

The Stem Sisterhood is based in Los Angeles. The founder Bridget wants to expand the group, but so far, she has only done local events because she is a senior in high school. The Stem Sisterhood has an Instagram account in addition to their blog. The blog is really interesting and features interviews with women who are in science and STEM careers.

Q: How has the Stem Sisterhood impacted your life?

SL: It has inspired me to reach out to younger girls more about science. It’s something that I am passionate about, and I’d like to pursue a career in the medical field. This group has given me an outlet to share that passion with others and to hopefully change the face of the STEM world.

Q: How did you find out about the CIRM SPARK program?

SL: I knew I wanted to do a science program over the summer, but I wasn’t sure what type. I didn’t know if I wanted to do research or be in a hospital. I googled science programs for high school seniors, and I saw the one at Stanford University. It looked interesting and Stanford is obviously a great institution. Coming from LA, I was nervous that I wouldn’t be able to get in because the program had said it was mostly directed towards students living in the Bay Area. But I got in and I was thrilled. So that’s basically how I heard about it, because I googled and found it.

Q: What was your SPARK experience like?

SL: My program was incredible. I was a little bit nervous and scared going into it because I was the only high school student in my lab. As a high school junior going into senior year, I was worried about being the youngest, and I knew the least about the material that everyone in the lab was researching. But my fears were quickly put aside when I got to the lab. Everyone was kind and helpful, and they were always willing to answer my questions. Overall it was really amazing to have my first lab experience be at Stanford doing research that’s going to potentially change the world.

Shannon working in the lab at Stanford.

I was in a lab that was using stem cells to characterize a type of leukemia. The lab is hoping to study leukemia in vitro and in vivo and potentially create different treatments and cures from this research. It was so cool knowing that I was doing research that was potentially helping to save lives. I also learned how to work with stem cells which was really exciting. Stem cells are a new advancement in the science world, so being able to work with them was incredible to me. So many students will never have that opportunity, and being only 17 at the time, it was amazing that I was working with actual stem cells.

I also liked that the Stanford SPARK program allowed me to see other aspects of the medical world. We did outreach programs in the Stanford community and helped out at the blood drive where we recruited people for the bone marrow registry. I never really knew anything about the registry, but after learning about it, it really interested me. I actually signed up for it when I turned 18. We also met with patients and their families and heard their stories about how stem cell transplants changed their lives. That was so inspiring to me.

Going into the program, I was pretty sure I wanted to be a pediatric oncologist, but after the program, I knew for sure that’s what I wanted to do. I never thought about the research side of pediatric oncology, I only thought about the treatment of patients. So the SPARK program showed me what laboratory research is like, and now that’s something I want to incorporate into my career as a pediatric oncologist.

I learned so much in such a short time period. Through SPARK, I was also able to connect with so many incredible, inspired young people. The students in my program and I still have a group chat, and we text each other about college and what’s new with our lives. It’s nice knowing that there are so many great people out there who share my interests and who are going to change the world.

Stanford SPARK students.

Q: What was your favorite part of the SPARK program?

SL: Being in the lab every day was really incredible to me. It was my first research experience and I was in charge of a semi-independent project where I would do bacterial transformations on my own and run the gels. It was cool that I could do these experiments on my own. I also really loved the end of the summer poster session where all the students from the different SPARK programs came together to present their research. Being in the Stanford program, I only knew the Stanford students, but there were so many other awesome projects that the other SPARK students were doing. I really enjoyed being able to connect with those students as well and learn about their projects.

Q: Why do you want to pursue pediatric oncology?

SL: I’ve always been interested in the medical field but I’ve had a couple of experiences that really inspired me to become a doctor. My friend has a charity that raises money for Children’s Hospital Los Angeles. Every year, we deliver toys to the hospital. The first year I participated, we went to the hospital’s oncology unit and something about it stuck with me. There was one little boy who was getting his chemotherapy treatment. He was probably two years old and he really inspired to create more effective treatments for him and other children.

I also participated in the STEAM Inquiry program at my high school, where I spent two years reading tons of peer reviewed research on immunotherapy for pediatric cancer. Immunotherapy is something that really interests me. It makes sense that since cancer is usually caused by your body’s own mutations, we should be able to use the body’s immune system that normally regulates this to try and cure cancer. This program really inspired me to go into this field to learn more about how we can really tailor the immune system to fight cancer.

Q: What advice do you have for young girls interested in STEM.

SL: My advice is don’t be afraid. I think that sometimes girls are expected to be interested in less intellectual careers. This perception can strike fear into girls and make them think “I won’t be good enough. I’m not smart enough for this.” This kind of thinking is not good at all. So I would say don’t be afraid and be willing to put yourself out there. I know for me, sometimes it’s scary to try something and know you could fail. But that’s the best way to learn. Girls need to know that they are capable of doing anything and if they just try, they will be surprised with what they can do.

Rare diseases are not so rare

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Brenden Whittaker – cured in a CIRM-funded clinical trial focusing on his rare disease

It seems like a contradiction in terms to say that there are nearly 7,000 diseases, affecting 30 million people, that are considered rare in the US. But the definition of a rare disease is one that affects fewer than 200,000 people and the National Institutes of Health’s (NIH) Genetic and Rare Diseases Information Center (GARD) has a database that lists every one of them.

Those range from relatively well known conditions such as sickle cell disease and cerebral palsy, to lesser known ones such as attenuated familial adenomatous polyposis (AFAP) – an inherited condition that increases your risk of colon cancer.

Because disease like these are so rare, in the past many individuals with them felt isolated and alone. Thanks to the internet, people are now able to find online support groups where they can get advice on coping strategies, ideas on potential therapies and, just as important, can create a sense of community.

One of the biggest problems facing the rare disease community is a lack of funding for research to develop treatments or cures. Because these diseases affect fewer than 200,000 people most pharmaceutical companies don’t invest large sums of money developing treatments; they simply wouldn’t be able to get a big enough return on their investment. This is not a value judgement. It’s just a business reality.

And that’s where CIRM comes in. We were created, in part, to help those who can’t get help from other sources. This week alone, for example, our governing Board is meeting to vote on funding clinical trials for two rare and deadly diseases – ALS or Lou Gehrig’s disease, and Severe Combined Immunodeficiency or SCID. This kind of funding can mean the difference between life and death.

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For proof, you need look no further than Evie Vaccaro, the young girl we feature on the front of our 2016 Annual Report. Evie was born with SCID and faced a bleak future. But UCLA researcher Don Kohn, with some help from CIRM, developed a therapy that cured Evie. This latest clinical trial could help make a similar therapy available to other children with SCID.

But with almost 7,000 rare diseases it’s clear we can’t help everyone. In fact, there are only around 450 FDA-approved therapies for all these conditions. That’s why the National Organization for Rare Disorders (NORD) and groups like them are organizing events around the US on February 28th, which has been designated as Rare Disease Day. The goal is to raise awareness about rare diseases, and to advocate for action to help this community. Here’s a link to Advocacy Events in different states around the US.

Alone, each of these groups is small and easily overlooked. Combined they have a powerful voice, 30 million strong, that demands to be heard.