At 18 years old, there are several life-changing moments that young people look forward to. For some, it involves graduating from high school, starting college, and being able to cast a vote in an election. For others, this momentous occasion symbolizes the official start of adulthood.
For James O’ Brien, this milestone was marked by a rather unfortunate event where ammonia was thrown at his face in a random attack. As a result of this incident, the surface of his right eye was burned and he was left completely blind in his right eye.
Fast forward 25 years and thanks to an experimental stem cell treatment, James is able to see out of his right eye for the first time since the attack.
“Being able to see with both eyes – it’s a small thing that means the world. Basically I went from near-blindness in that eye to being able to see everything.” said O’Brien in a news release from Daily Heralds.
Dr. Sajjad Ahmad and a team of surgeons at the Moorfields Eye Hospital in London removed healthy stem cells from O’Brien’s left eye and grew these cells in a lab for months. After an adequate number of healthy stem cells from O’Briens left eye were grown, the surgeons then cut the scar tissue in his right eye and replaced it with the healthy stem cells.
They then waited a year after the procedure for the cells to settle down before inserting a cornea – which plays a key role in vision and focuses light – from a deceased donor.
“This is going to have a huge impact. A lot of these patients are young men so it affects their work, their lives, those around them. It’s not just the vision that drops, it’s the pain.” said Dr. Ahmad in the news release previously mentioned.
The procedure used took over 20 years to develop and Dr. Ahmad hopes to continue to develop the procedure for patients that have been blinded in both eyes by chemicals or have lost their vision through degenerative conditions.
CIRM has funded three clinical trials in vision loss to date. Two of these trials are being conducted by Dr. Henry Klassen for an eye condition known as retinitis pigmentosa and have shown promising results. The third trial is being conducted by Dr. Mark Humayun for another eye condition known as age-related macular degeneration (AMD) which has also shown promising results.
See video below for a news segment of James O’Brien on BBC News:
For years CIRM and others in the stem cell community (hello Paul Knoepfler) have been warning people about the dangers of going to clinics offering unproven and unapproved stem cell therapies. Recently the drum beat of people and organizations coming out in support of that stand has grown louder and louder. Mainstream media – TV and print – have run articles about these predatory clinics. And now, Google has joined those ranks, announcing it will restrict ads promoting these clinics.
“We regularly review and revise our
advertising policies. Today, we’re announcing a new Healthcare and
medicines policy to prohibit advertising for unproven or experimental
medical techniques such as most stem cell therapy, cellular (non-stem) therapy,
and gene therapy.”
“Google’s new policy banning
advertising for speculative medicines is a much-needed and welcome step to curb
the marketing of unscrupulous medical products such as unproven stem cell
therapies. While stem cells have great potential to help us understand and
treat a wide range of diseases, most stem cell interventions remain
experimental and should only be offered to patients through well-regulated
clinical trials. The premature marketing and commercialization of unproven stem
cell products threatens public health, their confidence in biomedical research,
and undermines the development of legitimate new therapies.”
Speaking of Deepak – we can use first
names here because we are not only great admirers of him as a physician but also
as a researcher, which is why we have funded
some of his research – he has just published a wonderfully well written
article criticizing these predatory clinics.
The article – in Scientific
American – is titled “Don’t Believe Everything You Hear About Stem Cells”
and rather than paraphrase his prose, I think it best if you read it yourself.
So, here it is.
Don’t Believe Everything You Hear about Stem Cells
The science is progressing rapidly,but bad
actors have co-opted stem cells’ hope and promise by preying on unsuspecting
patients and their families
Stem cell science is moving forward
rapidly, with potential therapies to treat intractable human diseases on the
horizon.Clinical trials are now underway to test the safety
and effectiveness of stem cell–based treatments for blindness,spinal
cord injury,heart disease,Parkinson’s
disease, and more,some with early positive results.A
sense of urgency drives the scientific community, and there is tremendous hope
to finally cure diseases that, to date, have had no treatment.
But don’t believe everything you hear about stem cells. Advertisements and pseudo news articles promote stem cell treatments for everything from Alzheimer’s disease,autism and ALS, to cerebral palsy and other diseases.The claims simply aren’t true–they’re propagated by people wanting to make money off of a desperate and unsuspecting or unknowing public.Patients and their families can be misled by deceptive marketing from unqualified physicians who often don’t have appropriate medical credentials and offer no scientific evidence of their claims.In many cases, the cells being utilized are not even true stem cells.
Advertisements for stem cell treatments are showing up everywhere, with too-good-to-be-true
claims and often a testimonial or two meant to suggest legitimacy or efficacy.Beware of the following:
• Claims that stem
cell treatments can treat a wide range of diseases using a singular stem cell
type. This is unlikely to be true.
• Claims that stem
cells taken from one area of the body can be used to treat another, unrelated
area of the body. This is also unlikely to be true.
• Patient testimonials used to validate a
particular treatment, with no scientific evidence. This is a red flag.
• Claims that
evidence doesn’t yet exist because the clinic is running a patient-funded
trial. This is a red flag; clinical trials rarely require payment for
• Claims that the
trial is listed on ClinicalTrials.gov and is therefore NIH-approved. This may
not be true. The Web site is simply a listing; not all are legitimate trials.
• The bottom line:
Does the treatment sound too good to be true? If so, it probably is. Look for
concrete evidence that the treatment works and is safe.
Hundreds of clinics offer costly, unapproved and unproven stem cell
interventions, and patients may suffer physical and financial harm as a result.A Multi-Pronged Approach to Deal with
The International Society for Stem Cell Research (ISSCR)has
long been concerned that bad actors have co-opted the hope and promise of stem
cell science to prey on unsuspecting patients and their families.
We read with sadness and disappointment the many stories of people trying unproven therapies and being harmed, including going blind from injections into the eyes or suffering from a spinal tumor after an injection of stem cells.Patients left financially strapped, with no physical improvement in their condition and no way to reclaim their losses, are an underreported and underappreciated aspect of these treatments.
Since late 2017, the Food and Drug Administration has stepped up its
regulatory enforcement of stem cell therapies and provided a framework
for regenerative medicine products that provides guidelines for work in
this space.The agency has alerted many clinics and centers
that they are not in compliance and has pledged to bring additional enforcement
action if needed.
A Multi-Pronged Approach to Deal with Bad Actors The International Society for Stem Cell Research (ISSCR) has long been concerned that bad actors have co-opted the hope and promise of stem cell science to prey on unsuspecting patients and their families.
We read with sadness and disappointment the many stories of people trying
unproven therapies and being harmed, including going
blind from injections into the eyesor suffering from a spinal
tumor after an injection of stem cells.Patients left
financially strapped, with no physical improvement in their condition and no
way to reclaim their losses, are an underreported and underappreciated aspect
of these treatments.
Since late 2017, the Food and Drug Administration has stepped up its
regulatory enforcement of stem cell therapies and provided a framework
for regenerative medicine products that provides guidelines for work in
this space.The agency has alerted many clinics and centers
that they are not in compliance and has pledged to bring additional enforcement
action if needed.
In recent weeks, a federal judge granted the FDA a permanent injunction
against U.S. Stem Cell, Inc. and U.S. Stem Cell Clinic, LLC for adulterating
and misbranding its cellular products and operating outside of regulatory
authority.We hope this will send a strong message to other
clinics misleading patients with unapproved and potentially harmful cell-based
The Federal Trade Commission has also helped by identifying and curtailing
unsubstantiated medical claims in advertising by several clinics. Late in 2018
the FTC won a $3.3-million judgment against two California-based clinics for
deceptive health claims.
The Federal Trade Commission has also
helped by identifying and curtailing unsubstantiated medical claims in
advertising by several clinics. Late in 2018 the FTC won a $3.3-million
judgment against two California-based clinics for deceptive health claims.
These and other actions are needed to stem the tide of clinics offering
unproved therapies and the people who manage and operate them.
Improving Public Awareness
We’re hopeful that the FDA will help improve public awareness of these
issues and curb the abuses on ClinicalTrials.gov,a government-run Web site being misused by rogue clinics looking to
legitimize their treatments. They list pay-to-participate clinical trials on
the site, often without developing, registering or administering a real
The ISSCR Web site A Closer
Look at Stem Cellsincludes patient-focused information
about stem cells,with information written and vetted by stem
cell scientists.The site includes how and where to report
adverse events and false marketing claims by stem cell clinics.I
encourage you to visit and learn about what is known and unknown about stem
cells and their potential for biomedicine.The views expressed are those of the
author(s) and are not necessarily those of Scientific American.
It’s always gratifying to see research you have helped support go from being an intriguing idea to something with promise to a product that is now the focus of a company. It’s all the more gratifying if the product in question might one day help millions of people battling diabetes.
That’s the case with
a small pouch being developed by a company called Encellin. The pouch is the
brainchild of Tejal Desai, Ph.D., a
professor of bioengineering at UCSF and a CIRM grantee.
“It’s a cell encapsulation device, so this material can essentially protect beta cells from the immune system while allowing them to function by secreting insulin. We are placing stem cell-derived beta cells into the pouch which is then implanted under the skin. The cells are then able to respond to changes in sugar or glucose levels in the blood by pumping out insulin. By placing the device in a place that is accessible we can easily remove it if we have to, but also we can recharge it and put in new cells as well.”
While the pouch was developed in Dr. Desai’s lab, the idea
to take it from a promising item and try to turn it into a real-world therapy
came from one of Dr. Desai’s former students, Crystal Nyitray, Ph.D.
After getting her PhD, Nyitray went to work for the pharmaceutical giant Sanofi. In an article in FierceBiotech she says that’s where she realized that the pouch she had been working on at UCSF had real potential.
“During that time, I started to realize
we really had something, that everything that pharma or biotech was looking at
was something we had been developing from the ground up with those specific
questions in mind,”
So Dr. Nyitray went to work for QB3, the institute created
by UC San Francisco to help startups develop their ideas and get funding. The
experience she gained there gave her the confidence to be the co-founder and
CEO of Encellin.
Dr. Desai is a scientific advisor to Encellin. She says
trying to create a device that contains insulin-secreting cells is not new.
Many previous attempts failed because once the device was placed in the body,
the immune system responded by creating fibrosis or scarring around it which
blocked the ability of the cells to get out.
But she thinks their approach has an advantage over previous
“This is not a new idea, the idea has been around for 40 or
more years but getting it to work is hard. We have a convergence of getting the
right cell types and combining that with our knowledge of immunology and then
the material science where we can design materials at this scale to get the kind
of function that we need.
Dr. Nyitray ““If we can reduce fibrosis, it really
helps the cells get nutrients better, survive better and signal more
effectively. It’s really critical to their success.”
Dr. Desai says the device is still in the early stages of
being tested, but already it’s showing promise.
“We have done testing in animals. Where the company is
taking this is now to see if we can take this to larger animals and then
She says without CIRM’s support none of this would have
“CIRM has been really instrumental in helping us refine the
cell technology piece of it, to get really robust cells and also to support the
development to push the materials, to understand the biology, to really
understand what was happening with the cell material interface. We know we have
a lot of challenges ahead, but we are really excited to see if this could
We are excited too. We are looking forward to seeing what
Encellin does in the coming years. It could change the lives of millions of
people around the world.
A powerful opening statement by Angela Ramirez Holmes, Founder & President of the California Action Link for Rare Diseases (CAL RARE).
Tuesday of last week, patient advocates, patient advocacy organizations, and members of the public filled a room at the California Capitol for an informational hearing on research related to rare diseases. One of the organizations present was CAL RARE, a non-profit organization that is dedicated to improving the lives of California patients with rare diseases. Angela’s opening statement reflects CAL RARE’s core mission of bringing awareness of rare diseases to the general public and decision makers in order to improve access to physicians, treatments, and social services.
One of the first presenters was Dr. Martin Cadeiras from the Department of Cardiovascular Medicine at UC Davis. His presentation focused on a rare disease named amyloidosis, which occurs when a protein called amyloid builds up in the body’s organs and tissues. This can lead to problems in the heart, skin, kidneys, liver, and digestive tract. There are several different types of amyloidosis, one of which is hereditary and another form that can occur after chronic infection. Dr. Cadeiras spoke in detail about the scientific complexities behind amyloidosis and shared images of patients affected with the disease as well as the complications associated with their condition.
To elaborate more on the patient perspective of this disease, patient advocate Len Strickland shared his journey living with amyloidosis. In addition to living with the disease, Len also has the sickle cell trait, meaning he has one copy of the sickle cell disease gene but one normal copy.
In his early life, Len was a typical young adult with no health problems. Unfortunately for him that changed in 2006, when he started having problems with shortness of breath and heart palpitations almost overnight. He visited many doctors, all of which were perplexed by his condition and were unable to diagnose him.
“My normal life was gone, and I was very concerned.” said Strickland.
One year later, after multiple tests and specialists, he was finally diagnosed with the hereditary version of amyloidosis. As a result of his condition, he was in dire need of a heart transplant. On March 4, 2008 he was placed on the transplant list. Because he was relatively lower on the priority list, he was told to keep hope to a minimum. Fortunately, on June 10, 2008 a matching donor heart was found and by the next day, Len had successfully received the heart transplant.
Len wrote a thank you letter to the mother of the deceased donor and regularly keeps in touch with her. She hopes to one day meet Len in person so that she can hug Len and hear her son’s heartbeat.
Although the amyloid deposits have spread to Len’s hand and feet, he is still able to live his life.
Len ended his speech by telling the crowd,
“Make the best of the time you have, if I can do it, so can you.”
The challenges Len faced with getting a proper diagnosis brought up the need for technology that can better screen rare diseases. The next presenter, Dr. Lauge Farnaes of Rady Children’s Institute for Genomic Medicine, discussed a project that focused on just that. Under a two million dollar Medi-Cal program titled Project Baby Bear, Dr. Farnaes and his team have used genome sequencing as a diagnostic test for critically ill newborns. The ultimate goal is to get this screening as a Medi-Cal covered benefit.
Comprehensive early testing enables physicians to make early decisions about and minimize the damage accumulated before diagnosis. “We have a chance to go in early on and make a difference in the life of patients.” said Farnaes.
Dr. Farnaes told stories of some of the children enrolled in the screening program. One was a young girl that had problems related to the heart. She was enrolled February 6th and diagnosed two days later with Timothy Syndrome, making her one of the youngest patients ever diagnosed. She was implanted with a defibrillator to help with her heart problems. Dr. Farnaes had stated that without the screening, she would have likely just been prescribed beta blockers, which would only have worsened her condition.
Another child enrolled in the program had difficulty breathing as a result of bone fractures. Because of the bone fractures, it was thought that the child had undergone abuse at the hands of the parents. However, thanks to the screening technology, it was found to be the result of a genetic condition. Dr. Farnaes talked about how this technology vindicated the parents, who were already going through the difficult process of having a sick child without throwing other problems into the mix.
To date, 116 children have been diagnosed with genetic conditions early on using this technology and the number is expected to eventually approach 150.
Last, but not least, Assemblymember Mike Gipson shared an update on the work that the rare disease caucus has made with relation to sickle cell disease. He mentioned how the legislative black caucus had successfully advocated for allocating $15 million for sickle cell disease. This money will be used to open seven new sickle cell centers across California.
The meeting in the California Capitol highlighted the impact that patient stories have on policy, as well as the ongoing need of funding and new technologies to address the disparities in rare disease.
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.
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.
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.
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.
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
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.
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.
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.
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:
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.
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.
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.
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!
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
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
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.
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
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
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
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
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.