Every so often you hear a story and your first reaction is “oh, I have to share this with someone, anyone, everyone.” That’s what happened to me the other day.
I was talking with Kristin MacDonald, an amazing woman, a fierce patient advocate and someone who took part in a CIRM-funded clinical trial to treat retinitis pigmentosa (RP). The disease had destroyed Kristin’s vision and she was hoping the therapy, pioneered by jCyte, would help her. Kristin, being a bit of a pioneer herself, was the first person to test the therapy in the U.S.
Anyway, Kristin was doing a Zoom presentation and wanted to look her best so she asked a friend to come over and do her hair and makeup. The woman she asked, was Rosie Barrero, another patient in that RP clinical trial. Not so very long ago Rosie was legally blind. Now, here she was helping do her friend’s hair and makeup. And doing it beautifully too.
That’s when you know the treatment works. At least for Rosie.
There are many other stories to be heard – from patients and patient advocates, from researchers who develop therapies to the doctors who deliver them. – at our CIRM 2020 Grantee Meeting on next Monday September 14th Tuesday & September 15th.
It’s two full days of presentations and discussions on everything from heart disease and cancer, to COVID-19, Alzheimer’s, Parkinson’s and spina bifida. Here’s a link to the Eventbrite page where you can find out more about the event and also register to be part of it.
Like pretty much everything these days it’s a virtual event so you’ll be able to join in from the comfort of your kitchen, living room, even the backyard.
And it’s free!
You can join us for all two days or just one session on one day. The choice is yours. And feel free to tell your friends or anyone else you think might be interested.
You know you are working with some of the finest scientific minds in the world when they get elected to the prestigious National Academy of Sciences (NAS). It’s the science equivalent of the baseball, football or even Rock and Roll Hall of Fame. People only get in if their peers vote them in. It’s considered one of the highest honors in science, one earned over many decades of hard work. And when it comes to hard work there are few people who work harder than U.C. San Diego’s Dr. Lawrence Goldstein, one of the newly elected members of the NAS.
For more than 25 years Larry’s work has targeted the brain and, in particular, Alzheimer’s disease and amyotrophic lateral sclerosis (ALS) better known as Lou Gehrig’s disease.
In 2012 his team was the first to create stem cell models for two different forms of Alzheimer’s, the hereditary and the sporadic forms. This gave researchers a new way of studying the disease, helping them better understand what causes it and looking at new ways of treating it.
His work has also helped develop a deeper understanding of the genetics of Alzheimer’s and to identify possible new targets for stem cell and other therapies.
Larry was typically modest when he heard the news, saying: “I have been very fortunate to have wonderful graduate students and fellows who have accomplished a great deal of excellent research. It is a great honor for me and for all of my past students and fellows – I am obviously delighted and hope to contribute to the important work of the National Academy of Sciences.”
But Larry doesn’t intend to rest on his laurels. He says he still has a lot of work to do, including “raising funding to test a new drug approach for Alzheimer’s disease that we’ve developed with CIRM support.”
Jennifer Briggs Braswell, PhD, worked with Larry at UCSD from 2005 to 2018. She says Larry’s election to the NAS is well deserved:
“His high quality publications, the pertinence of his studies in neurodegeneration to our current problems, and his constant, unwavering devotion to the next generation of scientists is matched only by his dedication to improving public understanding of science to motivate social, political, and financial support.
“He has been for me a supportive mentor, expressing enthusiastic belief in the likely success of my good ideas and delivering critique with kindness and sympathy. He continues to inspire me, our colleagues at UCSD and other communities, advocate publicly for the importance of science, and work tirelessly on solutions for neurodegenerative disorders.”
As the coronavirus pandemic continues to spread, one of the few bright spots is how many researchers are stepping up and trying to find new ways to tackle it, to treat it and hopefully even cure it. Unfortunately, there are also those who are simply trying to cash in on it.
In the last few years the number of predatory clinics offering so-called “stem cell therapies” for everything from Alzheimer’s and multiple sclerosis to autism and arthritis has exploded in the US. The products they offer have not undergone a clinical trial to show that they work; they haven’t been approved by the US Food and Drug Administration (FDA); they don’t have any evidence they are even safe. But that doesn’t stop them marketing these claims and it isn’t stopping some of them from now trying to cash in on the fears created by the coronavirus.
One company is hawking what it calls a rapid COVID-19 test, one that can determine if you have the virus in under ten minutes (many current tests take days to produce a result). All it takes is a few drops of blood and, from the comfort of your own home, you get to find out if you are positive for COVID-19. And best of all, it claims it is 99 percent accurate.
What could be the problem with that? A lot as it turns out.
If you go to the bottom of the page on the website marketing the test it basically says “this does not work and we’re not making any claims or are in any way responsible for any results it produces.” So much for 99 percent accurate.
It’s not the only example of this kind of shameless attempt to cash in on COVID-19. So it’s appropriate that this week the Alliance for Regenerative Medicine (ARM), issued a statement strongly condemning these attempts and the clinics behind them.
ARM warns about the growing number of “stem cell clinics” (that) are taking advantage of the “hype” around stem cells – and, in certain cases, the current concern about COVID-19 – and avoiding regulation by falsely marketing illegal and potentially harmful products to patients seeking cures.”
These so called “therapies” or tests do more than just take money – in some cases tens of thousands of dollars – from individuals: “Public health is at risk when unscrupulous providers offer stem cell products that are unapproved, unproven and fail to adhere to established rules for good manufacturing practices. Many of these providers put patients at risk by falsely marketing the benefits of treatments, and often promoting the stem cells for conditions that are outside of their area of medical expertise.”
It’s sad that even in times when so many people are working hard to find treatments for the virus, and many are risking their lives caring for those who have the virus, that there are unscrupulous people trying to make money out of it. All we can do is be mindful, be careful and be suspicious of anything that sounds too good to be true.
There are no miracle cures. No miracle treatments. No rapid blood tests you can order in the mail. Be aware. And most importantly of all, be safe.
The CIRM Board recently held a meeting to approve $5 million in emergency funding for rapid research into potential treatments for COVID-19.
Way back in 2013, the CIRM Board invested $32 million in a project to create an iPSC Bank. The goal was simple; to collect tissue samples from people who have different diseases, turn those samples into high quality stem cell lines – the kind known as induced pluripotent stem cells (iPSC) – and create a facility where those lines can be stored and distributed to researchers who need them.
Fast forward almost seven years and that idea has now become the largest public iPSC bank in the world. The story of how that happened is the subject of a great article (by CIRM’s Dr. Stephen Lin) in the journal Science Direct.
In 2013 there was a real need for the bank. Scientists around the world were doing important research but many were creating the cells they used for that research in different ways. That made it hard to compare one study to another and come up with any kind of consistent finding. The iPSC Bank was designed to change that by creating one source for high quality cells, collected, processed and stored under a single, consistent method.
Tissue samples – either blood or skin – were collected from thousands of individuals around California. Each donor underwent a thorough consent process – including being shown a detailed brochure – to explain what iPS cells are and how the research would be done.
The diseases to be studied through this bank include:
Age-Related Macular Degeneration (AMD)
Autism Spectrum Disorder (ASD)
Cardiomyopathies (heart conditions)
Fatty Liver diseases
Hepatitis C (HCV)
Primary Open Angle Glaucoma
The samples were screened to make sure they were safe – for example the blood was tested for HBV and HIV – and then underwent rigorous quality control testing to make sure they met the highest standards.
Once approved the samples were then turned into iPSCs at a special facility at the Buck Institute in Novato and those lines were then made available to researchers around the world, both for-profit and non-profit entities.
Scientists are now able to use these cells for a wide variety of uses including disease modeling, drug discovery, drug development, and transplant studies in animal research models. It gives them a greater ability to study how a disease develops and progresses and to help discover and test new drugs or other therapies
The Bank, which is now run by FUJIFILM Cellular Dynamics, has become a powerful resource for studying genetic variation between individuals, helping scientists understand how disease and treatment vary in a diverse population. Both CIRM and Fuji Film are committed to making even more improvements and additions to the collection in the future to ensure this is a vital resource for researchers for years to come.
There’s a lot of
fiction, a lot of misinformation surrounding stem cells and stem cell research.
There are claims that are not based on solid science and clinics that are
offering so-called “treatments” that are unproven, even dangerous for patients.
Now you have a chance to talk to the experts in the field and get solid answers
from them about what’s working, what’s not, and how you can find a therapy that
might be appropriate for you.
Do you have
questions about the latest in research using stem cells to help people
recovering from a stroke? We’ll have someone who can answer them.
Want to know if stem
cells can help people battling cancer? Or what’s happening in finding a stem
cell treatment for diabetes or sickle cell disease, even autism, Alzheimer’s or
Parkinson’s disease? We’ll have experts to answers those.
This is all
happening in a special Facebook Live “Ask the Stem Cell Team” event on Thursday,
December 12th from 10.30am to 11.30amPDT. To take part
all you have to do is tune in on the day and post a question or you can send us
one ahead of time at email@example.com
We will do our best
to answer as many of them as we can during the Facebook Live event, and those
we don’t have time to get to we’ll answer in a blog at a later date.
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.
In ancient Greek mythology, a Chimera was a creature that was usually depicted as a lion with an additional goat head and a serpent for a tail. Due to the Chimera’s animal hybrid nature, the term “chimeric” came to fruition in the scientific community as a way to describe an organism containing two or more different sets of DNA.
A CIRM-funded study conducted by Dr. Mathew Blurton-Jones and his team at UC Irvine describes a way for human brain immune cells, known as microglia, to grow and function inside mice. Since the mice contain a both human cells and their own mice cells, they are described as being chimeric.
In order to develop this chimeric “mighty mouse” model, Dr. Blurton-Jones and his team generated induced pluripotent stem cells (iPSCs), which have the ability to turn into any kind of cell, from cell samples donated by adult patients. For this study, the researchers converted iPSCs into microglia, a type of immune cell found in the brain, and implanted them into genetically modified mice. After a few months, they found that the implanted cells successfully integrated inside the brains of the mice.
By finding a way to look at human microglia grow and function in real time in an animal model, scientists can further analyze crucial mechanisms contributing to neurological conditions such as Alzheimer’s, Parkinson’s, traumatic brain injury, and stroke.
For this particular study, Dr. Blurton-Jones and his team looked at human microglia in the mouse brain in relation to Alzheimer’s, which could hold clues to better understand and treat the disease. The team did this by introducing amyloid plaques, protein fragments in the brain that accumulate in people with Alzheimer’s, and evaluating how the human microglia responded. They found that the human microglia migrated toward the amyloid plaques and surrounding them, which is what is observed in Alzheimer’s patients.
In a press release, Dr. Blurton-Jones expressed the importance of studying microglia by stating that,
“Microglia are now seen as having a crucial role in the development and progression of Alzheimer’s. The functions of our cells are influenced by which genes are turned on or off. Recent research has identified over 40 different genes with links to Alzheimer’s and the majority of these are switched on in microglia. However, so far we’ve only been able to study human microglia at the end stage of Alzheimer’s in post-mortem tissues or in petri dishes.”
Furthermore, Dr. Blurton-Jones highlighted the importance of looking at human microglia in particular by saying that,
“The human microglia also showed significant genetic differences from the rodent version in their response to the plaques, demonstrating how important it is to study the human form of these cell.”
The full results of this study were published in Cell.
From Day One CIRM’s goal has been to advance stem cell research in California. We don’t do that just by funding the most promising research -though the 51 clinical trials we have funded to date clearly shows we do that rather well – but also by trying to bring the best minds in the field together to overcome problems.
Over the years we
have held conferences, workshops and symposiums on everything from Parkinson’s
palsy and tissue
engineering. Each one attracted the key players and stakeholders in the
field, brainstorming ideas to get past obstacles and to explore new ways of
developing therapies. It’s an attempt to get scientists, who would normally be
rivals or competitors, to collaborate and partner together in finding the best
It’s not easy to do,
and the results are not always obvious right away, but it is essential if we
hope to live up to our mission of accelerating stem cell therapies to patients
with unmet medical needs.
For example. This
past week we helped organize two big events and were participants in another.
The first event we
pulled together, in partnership with Cedars-Sinai Medical Center, was a
workshop called “Brainstorm Neurodegeneration”. It brought together leaders in stem
cell research, genomics, big data, patient advocacy and the Food and Drug
Administration (FDA) to tackle some of the issues that have hampered progress
in finding treatments for things like Parkinson’s, Alzheimer’s, ALS and
ambitiously subtitled the workshop “a cutting-edge meeting to disrupt the field”
and while the two days of discussions didn’t resolve all the problems facing us
it did produce some fascinating ideas and some tantalizing glimpses at ways to
advance the field.
Two days later we partnered with UC San Francisco to host the Fourth Annual CIRM Alpha Stem Cell Clinics Network Symposium. This brought together the scientists who develop therapies, the doctors and nurses who deliver them, and the patients who are in need of them. The theme was “The Past, Present & Future of Regenerative Medicine” and included both a look at the initial discoveries in gene therapy that led us to where we are now as well as a look to the future when cellular therapies, we believe, will become a routine option for patients.
different groups together is important for us. We feel each has a key role to
play in moving these projects and out of the lab and into clinical trials and
that it is only by working together that they can succeed in producing the
treatments and cures patients so desperately need.
As always it was the patients who surprised us. One, Cierra Danielle Jackson, talked about what it was like to be cured of her sickle cell disease. I think it’s fair to say that most in the audience expected Cierra to talk about her delight at no longer having the crippling and life-threatening condition. And she did. But she also talked about how hard it was adjusting to this new reality.
Cierra said sickle
cell disease had been a part of her life for all her life, it shaped her daily
life and her relationships with her family and many others. So, to suddenly
have that no longer be a part of her caused a kind of identity crisis. Who was
she now that she was no longer someone with sickle cell disease?
She talked about how
people with most diseases were normal before they got sick, and will be normal
after they are cured. But for people with sickle cell, being sick is all they
have known. That was their normal. And now they have to adjust to a new normal.
It was a powerful
reminder to everyone that in developing new treatments we have to consider the
whole person, their psychological and emotional sides as well as the physical.
And so on to the third event we were part of, the Stanford Drug Discovery Symposium. This was a high level, invitation-only scientific meeting that included some heavy hitters – such as Nobel Prize winners Paul Berg and Randy Schekman, former FDA Commissioner Robert Califf. Over the course of two days they examined the role that philanthropy plays in advancing research, the increasingly important role of immunotherapy in battling diseases like cancer and how tools such as artificial intelligence and big data are shaping the future.
CIRM’s President and CEO, Dr. Maria Millan, was one of those invited to speak and she talked about how California’s investment in stem cell research is delivering Something Better than Hope – which by a happy coincidence is the title of our 2018 Annual Report. She highlighted some of the 51 clinical trials we have funded, and the lives that have been changed and saved by this research.
The presentations at
these conferences and workshops are important, but so too are the conversations
that happen outside the auditorium, over lunch or at coffee. Many great
collaborations have happened when scientists get a chance to share ideas, or
when researchers talk to patients about their ideas for a successful clinical
It’s amazing what happens when you bring people together who might otherwise never have met. The ideas they come up with can change the world.
In 1983 President Ronald Reagan named November as Alzheimer’s Awareness month, to raise awareness about the growing impact the disease was having on Americans. At the time there were less than two million people with the disease. Today that number has grown to more than five million and is expected to reach 16 million by the year 2050. There is no cure and no effective treatments.
To mark Alzheimer’s Awareness month we are reprinting an article that CIRM Board member and Patient Advocate for Alzheimer’s, Lauren Miller, wrote for Lenny magazine, charting her own personal journey with the disease.
Two thousand eleven was a year of nonstop high points in my life. My creative dreams came true when I filmed a little indie movie I co-wrote about friendship and phone sex called For a Good Time, Call … My romantic dreams came true when I married the love of my life at a beautiful wedding surrounded by our friends and family. And then my professional dreams exploded with magical confetti when we sold our tiny, candy-colored, female-driven comedy to Focus Features after it premiered at the Sundance Film Festival. So many moments I had dreamed about for years were actually happening.
It was the happiest time of my life!
Except that it also wasn’t. It was actually kind of the worst.
This was around five years into my mom’s diagnosis of Alzheimer’s. Up until that point, her disease had been in the early stages. Her symptoms manifested in the repeated stories she told, occasionally slurred or confused speech, and a serious attachment to my dad. But Alzheimer’s is like a train that can’t be stopped once it leaves the station. And although these early days were really scary, they were nothing compared to what happened as the disease barreled through my mom’s once vibrant and beautiful mind.
She lived in Florida, and I was all the way across the country in Los Angeles. When she was first diagnosed, she told me I wasn’t allowed to move back to Florida to be with her. She wanted me to live my life — one that she had worked so hard to support. She was selfless like that. It was more important to her that I pursue my dreams, my relationship, and my own life as an independent woman than to have me close while she was disappearing piece by piece.
I visited Florida as often as I could, and, in the first few years of her disease, she and my dad visited me in LA. In 2007, they came out for the premiere of Knocked Up — a huge celebration for my then-boyfriend, as it was his first starring role in a movie. We had a barbecue for friends and family at our house the day before the premiere, and while it was a delicious taco, rib, mac-’n’-cheese extravaganza, what I remember is having to show my mom where the silverware drawer was a dozen times. She also kept adding turkey to the vegetarian chili and I had to keep taking it out. I remember my sister-in-law asking if my mom was OK, and I remember telling her “she’s not.”
As the years went by, she got worse. When we were making For A Good Time, Call…, I wanted my parents to come to Los Angeles to visit the set of the movie I had worked so hard to put together. But my mom had started wandering off and having bathroom issues — this reality was enough to keep them home. It devastated my movie-buff dad (who had a closet full of 500-plus VHS tapes of all the greatest hits) that he couldn’t visit the set of his daughter’s first film, which his son was also executive producing. And it devastated me that I couldn’t show my mom that I was actually doing what she wanted — I was living my life. But there was no time to be sad about it; there was a pink phone ringing and I had to answer it with a smile because the cameras were rolling and I was literally living my dream! Or so I told myself.
The night before my wedding, at the rehearsal dinner, my mom told me, “I just want to go home. I don’t want to be here.” I knew it was because she didn’t know where she was. The pain of knowing that she didn’t know she was at my wedding was like that Alzheimer’s train plowing right through my heart.
I brought my new husband and his parents to Florida for Thanksgiving that year — we went to Disney World. I showed them where I was a cheerleader for my awesome high-school football team (go Dreadnaughts!) and made them eat a Publix sub sandwich, my favorite hometown food. We also witnessed my mom pacing the house endlessly, not making much sense with her words, and mistaking the floor for a toilet. It was not quite the way I envisioned introducing my in-laws to the home I grew up in.
Although things were finally happening in my career in a way I had dreamed about for years — I was going on auditions, having meetings with producers about writing exciting scripts, and traveling around as my little-movie-that-could was released in actual theaters — my outlook on life was growing darker and darker with each passing day. Every person I saw run a stop sign was responsible for ruining the world. If I heard someone say something I didn’t agree with, it meant everyone was stupid and there was no hope for humanity. And most of all, I was convinced that the whole concept of life was just utter shit if this is what happened to the greatest woman I’ve ever known, a woman who had lived her life giving so much to so many.
Once my stress resulted in silver-dollar-size bacne, three (minor) car accidents in four months, and general life-rage that was bordering on getting out of control, it seemed like the right time to talk to a professional.
My new therapist listened, she gave me tissues, she told me it was OK to feel all the pain I was feeling. She let me go on and on to her about how life was just so unfair. And she didn’t even make me feel ungrateful for discounting all the legitimately amazing stuff in my life, because the agony I was in needed attention.
But eventually, she started to suggest that perhaps not everything was totally awful. Maybe there were a few good things in my life. Like, maybe my wonderful husband? Or my generous and awesome friends? Or even my career that felt like it was finally happening?
“Nope!” I insisted. “None of those things matter because everyone is dumb and there’s no point to any of this, and it’s all just a cycle of sadness and devastation, yadda, yadda, negative, negative, fuck, ahhhh! …”
As it always does, time passed. My parents moved to Los Angeles, and I got a front-row seat to my mom’s decline. She was now fully incontinent, had stopped walking, and had essentially stopped communicating. Having her close felt good, but seeing her become a shell of who she used to be was draining. When I wasn’t at her house, life continued as it normally did for me — spending my days alone, writing about and ruminating on my anger, spending my evenings and weekends laughing with friends over good food, and occasionally attending premieres for my (very fortunate and hard-working) husband’s movies. My therapist kept insisting that “in life, there can be good and there can be bad, and, in fact, they can coexist.” Even though I was living it, I refused to believe her.
Until eventually, I did.
In the fall of 2012, I started writing a screenplay about a woman who is left at the altar and ends up going on what would have been her honeymoon cruise with her estranged father. Obviously, this story has nothing to do with my real life (you just read about my wedding and my awesome dad), but I was drawn to the idea of a female character whose life doesn’t go the way she wants it to but who finds a way through the pain. It took me almost two years to finish a full draft, in part because I was still finding my own way through the pain. But when I did finish, I realized had written a story about a woman whose life has been seriously rocked, and, through her journey to get to the other side of her sadness, she experiences laughter, friendship, and adventure, as well as heartache, anger, and struggle for acceptance. Just like me.
I recently started thinking of my life as a dramedy. It’s full of jokes about penises, people comically falling, and moments of pure love and joy. It’s also full of pain, loss, and real anger. Ups and downs, highs and lows. But somewhere along the way, through my work in therapy, through talking about what I had been going through with my mom, through creating Hilarity for Charity, through writing, through living through my pain, I began to let in some of the good with the bad. To understand that it’s OK to feel both sad when I leave my mom’s house and excited about the dinner with friends I’m going to that night. That perhaps the point of life is to appreciate both the happy moments and the sad moments together. That they give each other meaning. That we have to have both, that we all have both, that experiencing both is actually OK. And if we need to spend a little time in the sad lane, the happy lane really can still run alongside it, if we allow it to.
Lauren Miller Rogen is a filmmaker and Alzheimer’s advocate living in Los Angeles. Her directorial debut,Like Father, which she also wrote and produced, launched globally on Netflix on Friday, August 3.
New developments in Alzheimer’s research are bringing us closer to more precise therapies for this debilitating disease.
Alzheimer’s disease, is characterized by the formation of amyloid plaques in the brain, which interfere with the normal communication flow between brain cells, leading to debilitating symptoms like memory loss and impaired decision-making. These plaques are made out of beta-amyloid proteins that stick together.
Over the past few years, researchers from several institutions have been working to develop antibodies that bind to and neutralize the toxic effects of the beta-amyloid. The search for effective antibodies, although promising, has been riddled with setbacks. Knowing this, a team of researchers from Brigham and Women’s Hospital in Boston, MA, decided to approach this issue from a different angle – by conducting experiments to identify a better way of targeting beta-amyloid. Their goal was to develop a more efficient antibody to be used in Alzheimer’s therapy.
Principal investigator Dominic Walsh and team came up with a novel technique to collect beta-amyloid and to prepare it in the laboratory.
Dominic Walsh, PH.D.
“Many different efforts are currently underway to find treatments for Alzheimer’s disease, and anti-[beta-amyloid] antibodies are currently the furthest advanced,” says Walsh. “But the question remains: what are the most important forms of [beta-amyloid] to target? Our study points to some interesting answers,” the lead researcher adds, and these answers are now reported in an open access paper published in the journal Nature Communications.”
Beta-amyloid can be found in many forms. At one end of the spectrum, it exists as a single protein, or monomer, which isn’t necessarily toxic.
At the other end, there is the beta-amyloid plaque, in which many beta-amyloid proteins become tangled together. Beta-amyloid plaques are large enough to be observed using a traditional microscope, and they are involved in the development of Alzheimer’s.
In the current study, as well as in a previous one, Walsh and team looked at beta-amyloid structures to identify the ones that are most harmful in the brain.
Typically specialists use synthetic beta-amyloid samples to create a laboratory model of Alzheimer’s disease in the brain. Very few scientists actually collect beta-amyloid from the brains of individuals diagnosed with the disease.
In the current study, Walsh and team focused on finding better a more specific antibody to target the toxic forms of beta-amyloid but not the less harmful forms. To do so, they developed a novel screening test that requires extracting beta-amyloid from brain samples from people with Alzheimer’s. They added these extracts to induced pluripotent stem cell-derived human neurons and observed the ability of the different antibodies to block the toxic effects of the beta-amyloid.
This screening test allowed the team to discover a particular antibody — called “1C22” — that is able to block toxic forms of beta-amyloid more effectively than other antibodies currently being tested in clinical trials.
Walsh explained the implications of their novel screening method:
“We anticipate that this primary screening technique will be useful in the search to identify more potent anti-[beta-amyloid] therapeutics in the future.”