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)


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Translating great stem cell ideas into effective therapies

alzheimers

CIRM funds research trying to solve the Alzheimer’s puzzle

In science, there are a lot of terms that could easily mystify people without a research background; “translational” is not one of them. Translational research simply means to take findings from basic research and advance them into something that is ready to be tested in people in a clinical trial.

Yesterday our Governing Board approved $15 million in funding for four projects as part of our Translational Awards program, giving them the funding and support that we hope will ultimately result in them being tested in people.

Those projects use a variety of different approaches in tackling some very different diseases. For example, researchers at the Gladstone Institutes in San Francisco received $5.9 million to develop a new way to help the more than five million Americans battling Alzheimer’s disease. They want to generate brain cells to replace those damaged by Alzheimer’s, using induced pluripotent stem cells (iPSCs) – an adult cell that has been changed or reprogrammed so that it can then be changed into virtually any other cell in the body.

CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs and Alzheimer’s – which has no cure and no effective long-term treatments – clearly represents an unmet medical need.

Another project approved by the Board is run by a team at Children’s Hospital Oakland Research Institute (CHORI). They got almost $4.5 million for their research helping people with sickle cell anemia, an inherited blood disorder that causes intense pain, and can result in strokes and organ damage. Sickle cell affects around 100,000 people in the US, mostly African Americans.

The CHORI team wants to use a new gene-editing tool called CRISPR-Cas9 to develop a method of editing the defective gene that causes Sickle Cell, creating a healthy, sickle-free blood supply for patients.

Right now, the only effective long-term treatment for sickle cell disease is a bone marrow transplant, but that requires a patient to have a matched donor – something that is hard to find. Even with a perfect donor the procedure can be risky, carrying with it potentially life-threatening complications. Using the patient’s own blood stem cells to create a therapy would remove those complications and even make it possible to talk about curing the disease.

While damaged cartilage isn’t life-threatening it does have huge quality of life implications for millions of people. Untreated cartilage damage can, over time lead to the degeneration of the joint, arthritis and chronic pain. Researchers at the University of Southern California (USC) were awarded $2.5 million to develop an off-the-shelf stem cell product that could be used to repair the damage.

The fourth and final award ($2.09 million) went to Ankasa Regenerative Therapeutics, which hopes to create a stem cell therapy for osteonecrosis. This is a painful, progressive disease caused by insufficient blood flow to the bones. Eventually the bones start to rot and die.

As Jonathan Thomas, Chair of the CIRM Board, said in a news release, we are hoping this is just the next step for these programs on their way to helping patients:

“These Translational Awards highlight our goal of creating a pipeline of projects, moving through different stages of research with an ultimate goal of a successful treatment. We are hopeful these projects will be able to use our newly created Stem Cell Center to speed up their progress and pave the way for approval by the FDA for a clinical trial in the next few years.”

Young Minds Shine Bright at the CIRM SPARK Conference

SPARK students take a group photo with CIRM SPARK director Karen Ring.

SPARK students take a group photo with CIRM SPARK director Karen Ring.

Yesterday was one of the most exciting and inspiring days I’ve had at CIRM since I joined the agency one year ago. We hosted the CIRM SPARK conference which brought together fifty-five high school students from across California to present their stem cell research from their summer internships.

The day was a celebration of their accomplishments. But it was also a chance for the students to hear from scientists, patient advocates, and clinicians about the big picture of stem cell research: to develop stem cell treatments and cures for patients with unmet medical needs.

Since taking on the role of the CIRM SPARK director, I’ve been blown away by the passion, dedication, and intelligence that our SPARK interns have shown during their short time in the lab. They’ve mastered techniques and concepts that I only became familiar with during my PhD and postdoctoral research. And even more impressive, they eloquently communicated their research through poster presentations and talks at the level of professional scientists.

During their internships, SPARK students were tasked with documenting their research experiences through blogs and social media. They embraced this challenge with gusto, and we held an awards ceremony to recognize the students who went above and beyond with these challenges.

I’d like to share the winning blogs with our readers. I hope you find them as inspiring and motivating as I do. These students are our future, and I look forward to the day when one of them develops a stem cell treatment that changes the lives of patients. 

Andrew Choi

Andrew Choi

Andrew Choi, Cedars-Sinai SPARK student

Am I crying or is my face uncontrollably sweating right now? I think I am doing both as I write about my unforgettable experiences over the course of the past 6 weeks and finalize my poster.

As I think back, I am very grateful for the takeaways of the research field, acquiring them through scientific journals, lab experiments with my mentor, and both formal and informal discourses. It seems impossible to describe all the episodes and occurrences during the program in this one blog post, but all I can say is that they were all unique and phenomenal in their own respective ways.

Gaining new perspectives and insights and being acquainted with many of the techniques, such as stereology, immunocytochemistry and immunohistochemistry my peers have utilized throughout their careers, proved to me the great impact this program can make on many individuals of the younger generation.

CIRM SPARK not only taught me the goings on behind the bench-to-bedside translational research process, but also morals, work ethics, and effective collaboration with my peers and mentors. My mentor, Gen, reiterated the importance of general ethics. In the process of making my own poster for the program, her words resonate even greater in me. Research, education, and other career paths are driven by proper ethics and will never continue to progress if not made the basic standard.

I am thankful for such amazing institutions: California Institute of Regenerative Medicine (CIRM) and Cedars-Sinai Medical Center for enabling me to venture out into the research career field and network. Working alongside with my fellow seven very brilliant friends, motivated me and made this journey very enjoyable. I am especially thankful my mentor, Gen, for taking the time to provide me with the best possible resources, even with her busy ongoing projects. She encouraged me to be the best that I am.

I believe, actually, I should say, I KNOW Cedars-Sinai’s CIRM SPARK program does a SUPERB and astounding job of cultivating life-long learners and setting exceptional models for the younger generation. I am hoping that many others will partake in this remarkable educational program.

I am overall very blessed to be part of a successful summer program. The end of this program does not mark the end of my passions, but sparks them to even greater heights.

Jamey Guzman

Jamey Guzman

Jamey Guzman, UC Davis SPARK student

When I found out about this opportunity, all I knew was that I had a fiery passion for learning, for that simple rush that comes when the lightbulb sputters on after an unending moment of confusion. I did not know if this passion would translate into the work setting; I sometimes wondered if passion alone would be enough to allow me to understand the advanced concepts at play here. I started at the lab nervous, tentative – was this the place for someone so unsure exactly what she wanted to be ‘when she grew up,’ a date now all too close on the horizon? Was I going to fit in at this lab, with these people who were so smart, so busy, people fighting for their careers and who had no reason to let a 16-year-old anywhere near experiments worth thousands of dollars in cost and time spent?

I could talk for hours about the experiments that I worked to master; about the rush of success upon realizing that the tasks now completed with confidence were ones that I had once thought only to belong to the lofty position of Scientist. I could fill pages and pages with the knowledge I gained, a deep and personal connection to stem cells and cell biology that I will always remember, even if the roads of Fate pull me elsewhere on my journey to a career.

The interns called the experience #CIRMSparkLab in our social media posts, and I find this hashtag so fitting to describe these last few months. While there was, of course, the lab, where we donned our coats and sleeves and gloves and went to work with pipets and flasks…There was also the Lab. #CIRMSparkLab is so much more than an internship; #CIRMSparkLab is an invitation into the worldwide community of learned people, a community that I found to be caring and vibrant, creative and funny – one which for the first time I can fully imagine myself joining “when I grow up.”

#CIRMSparkLab is having mentors who taught me cell culture with unerring patience and kindness. It is our team’s lighthearted banter across the biosafety cabinet; it is the stories shared of career paths, of goals for the present and the future. It is having mentors in the best sense of the word, trusting me, striving to teach and not just explain, giving up hours and hours of time to draw up diagrams that ensured that the concepts made so much sense to me.

#CIRMSparkLab is the sweetest ‘good-morning’ from scientists not even on your team, but who care enough about you to say hi, to ask about your projects, to share a smile. It is the spontaneity and freedom with which knowledge is dispensed: learning random tidbits about the living patterns of beta fish from our lab manager, getting an impromptu lecture about Time and the Planck Constant from our beloved professor as he passes us at lunch. It is getting into a passionate, fully evidence-backed argument about the merits of pouring milk before cereal that pitted our Stem Cell team against our Exosome team: #CIRMSparkLab is finding a community of people with whom my “nerdy” passion for learning does not leave me an oddball, but instead causes me to connect instantly and deeply with people at all ages and walks of life. And it is a community that, following the lead of our magnificent lab director, welcomed ten interns into their lab with open arms at the beginning of this summer, fully cognizant of the fact that we will break beakers, overfill pipet guns, drop gels, bubble up protein concentration assays, and all the while never stop asking, “Why? Why? Why? Is this right? Like this? WHY?”

I cannot make some sweeping statement that I now know at age 16 exactly what I want to do when I grow up. Conversely, to say I learned so much – or I am so grateful – or you have changed my life is simply not enough; words cannot do justice to those sentiments which I hope that all of you know already. But I can say this: I will never forget how I felt when I was at the lab, in the community of scientists. I will take everything I learned here with me as I explore the world of knowledge yet to be obtained, and I will hold in my heart everyone who has helped me this summer. I am truly a better person for having known all of you.

Thank you, #CIRMSparkLab. 

Adriana Millan

Adriana Millan

Adriana Millan, CalTech SPARK student

As children, we all grew up with the companionship of our favorite television shows. We enjoyed sitcoms and other animations throughout our childhood and even as adults, there’s no shame. The goofy and spontaneous skits we enjoyed a laugh over, yet we did not pay much attention to the lessons they attempted to teach us. As a child, these shows play crucial roles in our educational endeavors. We are immediately hooked and tune in for every episode. They spark curiosity, as they allow our imaginations to run wild. For me, that is exactly where my curiosity stemmed and grew for science over the years. A delusional young girl, who had no idea what the reality of science was like.

You expect to enter a lab and run a full day of experimentations. Accidentally mix the wrong chemicals and discover the cure for cancer. Okay, maybe not mix the incorrect chemicals together, I learned that in my safety training class. The reality is that working in a lab was far from what I expected — eye opening. Working alongside my mentor Sarah Frail was one of the best ways I have spent a summer. It was not my ideal summer of sleeping in until noon, but it was worthwhile.

My experience is something that is a part of me now. I talk about it every chance I get, “Mom, can you believe I passaged cells today!” It changed the way I viewed the principles of science. Science is one of the most valuable concepts on this planet, it’s responsible for everything and that’s what I have taken and construed from my mentor. She shared her passion for science with me and that completed my experience. Before when I looked at cells, I did not know exactly what I was supposed to observe. What am I looking at? What is that pink stuff you are adding to the plate?

However, now I feel accomplished. It was a bit of a roller coaster ride, with complications along the way, but I can say that I’m leaving this experience with a new passion. I am not just saying this to please the audience, but to express my gratitude. I would have never even looked into Huntington’s Disease. When I first arrived I was discombobulated. Huntington’s Disease? Now I can proudly say I have a grasp on the complexity of the disease and not embarrass my mentor my calling human cells bacteria – quite embarrassing in fact.  I’m a professional pipette handler, I work well in the hood, I can operate a microscope – not so impressive, I have made possibly hundreds of gels, I have run PCRs, and my cells love me, what else can I ask for.

If you are questioning what career path you are to take and even if it is the slightest chance it may be a course in science, I suggest volunteering in a lab. You will leave with your questioned answered. Is science for me? This is what I am leaving my experience with. Science is for me.

Other SPARK 2016 Awards

Student Speakers: Jingyi (Shelly) Deng (CHORI), Thomas Thach (Stanford)

Poster Presentations: Jerusalem Nerayo (Stanford), Jared Pollard (City of Hope), Alina Shahin (City of Hope), Shuling Zhang (UCSF)

Instagram Photos: Roxanne Ohayon (Stanford), Anna Victoria Serbin (CHORI), Diana Ly (UC Davis)

If you want to see more photos from the CIRM SPARK conference, check out our Instagram page @CIRM_Stemcells or follow the hashtag #CIRMSPARKLab on Instagram and Twitter.

California high schoolers SPARK interest in stem cell research through social media

I have a job for you today and it’s a fun one. Open your Instagram app on your phone. If you’re not an Instagrammer, don’t worry, you can access the website on your computer.

Do you have it open? OK now type in the hashtag #CIRMSparkLab and click on it.

What you’ll find is around 200 posts of the most inspiring and motivating pictures of stem cell research that I’ve seen. These pictures are from high school students currently participating in the CIRM summer SPARK program, one of our educational programs, which has the goal to train the next generation of stem cell scientists.

The SPARK program offers California high school students an invaluable opportunity to gain hands-on training in regenerative medicine at some of the finest stem cell research institutes in the state. And while they gain valuable research skills, we are challenging them to share their experiences with the general public through blogging and social media.

Communicating science to the public is an important mission of CIRM, and the SPARK students are excelling at this task by posting descriptive photos on Instagram that document their internships. Some of them are fun lab photos, while others are impressive images of data with detailed explanations about their research projects.

Below are a few of my favorite posts so far this summer. I’ve been so inspired by the creativity of these posts that we are now featuring some of them on the @CIRM_Stemcells account. (Yes this is a shameless plug for you to follow us on Instagram!).

City of Hope SPARK program.

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I encourage you all to follow our talented SPARK students this summer as they continue to document their exciting journeys on Instagram. These students are our future and supporting their training and education in stem cell research is an honor for CIRM and a vital step towards achieving our mission of accelerating stem cell treatments to patients with unmet medical needs.

Stay tuned for more blog coverage about SPARK and our other educational program, the Bridges to Stem Cell Research program for undergraduate and master-level students. The annual Bridges conference that brings all the students together to present their research will be held next week, and the SPARK conference is on August 8th both in Berkeley.

Sickle Cell Disease Leaves No Organ Untouched

“There really isn’t an organ in the body that isn’t affected by sickle cell disease.”

This striking comment was made by the Dr. Bertram Lubin, the CEO and President of the Children’s Hospital Oakland Research Institute (CHORI) and a CIRM Board Member.

Yesterday Dr. Lubin visited CIRM headquarters to talk about sickle cell disease (SCD). SCD is a group of inherited disorders caused by unhealthy, sickle-shaped red blood cells. People with SCD have abnormal hemoglobin, an important protein in red blood cells used to transport oxygen from the lungs to organs and tissues throughout the body.

The What, Why and Who of SCD

A mutation in the globlin gene leads to sickled red blood cells that clog up blood vessels

A mutation in the globlin gene leads to sickled red blood cells that clog up blood vessels

Genetic mutations in the hemoglobin genes lead to changes in the hemoglobin protein that cause normal, healthy disc-shaped red blood cells to take on a crescent, sickle shape. These sickle cells are a big problem because they stick to each other and to the walls of blood vessels, causing blockage and impeding blood flow. This leads to a plethora of clinical complications that we will touch on later in this blog.

Dr. Lubin shared some shocking facts including that 2 million African Americans are carriers of SCD mutations and 100,000 Americans have the disease. In the US, 1000 babies are born with SCD each year, but this number pales in comparison to the 1000 African babies that are born with SCD each day.

“So anything we do here with CIRM has a direct impact on sickle cell disease,” Lubin explained. “It’s something we should consider because it could have a global impact on SCD.”

SCD Affects Every Organ in the Body

Dr. Bertram Lubin

Dr. Bertram Lubin

Dr. Lubin next discussed a laundry list of clinical manifestations associated with SCD, making it clear that SCD is not just a blood disorder, it affects every organ and tissue in the body. Examples he gave included infection, enlarged spleen, stroke, bone disease, retinopathy, and gastro-intestinal complications. And these were only a handful of the symptoms he discussed that SCD patients deal with.

However, Dr. Lubin emphasized that early detection of SCD in babies can drastically improve the quality and length of life of SCD patients. He proudly explained how California was the first state to screen every newborn baby for SCD (a procedure that is now done in every state) and that CHORI’s Center for Sickle Cell Disease and Thalassemia is one of the major SCD programs in the world. Their center “strives to improve public awareness of these diseases, expand the current knowledge base, and ultimately, to provide innovative treatment, care – and cures.”

Dr. Lubin also commented on the importance of knowing if patients who go to the ER or doctor have SCD:

Dr. Bertram Lubin

Dr. Bertram Lubin

“With new born screening before we identified who had sickle cell disease, an African American child could come to the emergency room with a 103 F temperature. And they would say, well this is a virus, go home, and half of those kids would die by the next day. Because those with pneumococcal sepsis [a bacterial infection that SCD patients have an increased risk for] don’t last very long. Now when someone comes into the emergency room with a 103 F temperature and we know they have sickle cell, they get antibiotics right away. That told us there is a different way to do it and that really showed how genetics and public health can have an impact on the overall health of the population.”

Treatments and Hope for SCD

Dr. Lubin ended his talk by discussing the current management and treatment strategies for SCD patients. Early identification through universal newborn screening and family education are essential as well as preventative measures like penicillin and immunization to avoid infection.

As for therapeutic interventions, he mentioned blood transfusions, hydroxyurea treatments (which boosts the levels of healthy hemoglobin in blood cells), and bone marrow stem cell transplants. He said while bone marrow transplants have successfully treated some SCD patients, there are still many barriers to this form of treatment. Only 14% of families of SCD patients have an HLA-identical sibling donor and only 19% have an unrelated HLA-matched donor. Additionally, some doctors avoid recommending bone marrow transplants to SCD patients because of the risks for transplant rejection (graft vs. host disease) and death.

However, Dr. Lubin is hopeful that recent advances in stem cell research and genome engineering will one day make stem cell transplants the go-to treatment for SCD patients.

He ended with:

“The future of curative therapies that will have broad availability for SCD might follow advances in genomic correction of sickle mutation in hematopoietic [bone marrow] stem cells.”


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