A funny thing happened on my way to a PhD: one scientists change of mind and change of direction

Laurel Barchas is an old and dear friend of the communications team here at CIRM. As a student at U.C. Berkeley she helped us draft our education portal – putting together a comprehensive curriculum to help high schools teach students about stem cells in a way that met all state and federal standards. But a funny thing happened on her way to her Ph.D., she realized she had changed her mind about research, and so she changed her career direction.  

Laurel recently wrote this blog about that experience for the new and improved website of the Student Society for Stem Cell Research (SSSCR) –

Laurel #1

Laurel Barchas at the World Stem Cell Summit 2013

Stem cell parental advice—you can grow up to be anything!

I was one of those students who, since high school, knew I was destined for the lab. Throughout some of high school, and all of college and graduate school, I had internships or positions in amazing labs that warmly took me in and trained me how to be a scientist. I loved designing and carrying out experiments on my stem cells, presenting at lab meetings, writing theses, and teaching others about my work through undergraduate lectures and high school presentations. My participation in the Student Society for Stem Cell Research hugely supported all of my efforts; it even enabled me to get one of my first jobs as a contract curriculum writer (a project manager role) with the California Institute for Regenerative Medicine, which launched my writing career.

Four years into my biology PhD program, things became clear that I didn’t want to do research anymore. I couldn’t handle the failure inherent in doing research. I wasn’t able to put in the time and focus necessary to do big experiments—then repeat them over and over. Although I loved science, I wasn’t meant to be a career scientist like many of my colleagues. I was a science communicator. Realizing this, and taking into account my personal struggles, my advisers and I decided the best thing was to get a terminal master’s degree.**

Differentiation—finding the right path

I struggled for a while finding a job that suited me. I worked as an education consultant, writing materials directed at teachers and students. I worked as a marketing, communications and operations assistant for a real estate group. I looked for jobs as a teacher, curriculum developer, and science education program coordinator, but none felt quite right for me. Although I had extensive experience in school developing materials for teachers and giving presentations to students, and I knew education could be a rewarding career path, I wasn’t sure I wanted to be in the academic world anymore.

Finally, I found some listings looking for technical writers. I didn’t even know what that was at the time. Various biotech companies had their feelers out for entry level writers with advanced degrees in biology or STEM fields—and a master’s degree was just fine. It turns out I was a perfect fit. Surprisingly, many people in the “tech com” (technical communications) and “mar com” (marketing communications) departments at my company had a similar experience; they didn’t want careers in research or the medical professions, so they chose communications.

Laurel #2

Life as a technical writer—feeling like a glial cell

As a technical writer at my company, I have many responsibilities beyond writing and editing user manuals, application notes, and diagrams. Tech writers are much like the oft-forgotten glial cells that “glue the brain together.” I manage each project from start to finish, and I get to work on all types of technical documentation and marketing collateral with a team of company scientists (R&D), graphic designers, marketing specialists, coders, product managers, and other writers. Often, I have major creative input on the content, design, and development of marketing campaigns. I enjoy starting with ideas—maybe a few bullet points or a rough draft—and building colorful, captivating content. It feels like solving a complex puzzle.

I’ve gotten the chance to write articles on human induced pluripotent stem cell-derived beta cells for a drug discovery publication and to create portals for our website. I’ve helped make booth panels and printed resources for conferences like the International Society for Stem Cell Research. Most importantly (to me), I’ve managed to stay within the field of stem cell research/regenerative medicine. I am the main writer for that product and service line, so I can use my expertise and experience (plus, knowledge of my audience) to present products that advance my audience’s basic, translational and clinical research.

I love my job. It pays well, has regular hours, and gives me a sense of belonging to a team. It’s fast paced, I’m working on a new thing every day, and I get to learn and write about the latest advancements from our R&D teams around the world. I could go on and on, but suffice it to say that the job fits like a glove, and I can see myself doing this long term. Also…I get to live in Silicon Valley! (Pros: great food, culture, people. Cons: cost of living, traffic.)

I hope you can get encouragement from the retelling of my experience that there is a space for you in this field. This is the first post in a series of articles about careers in regenerative medicine. I aim to take you through a tour of the vocational landscape—its ups, its downs—and am looking forward to hearing from you with any jobs/roles/scenarios you are curious about. Please comment on what you’d like to learn about next!

Remember: there are plenty of options and ways for you to apply your talent and experience to pushing our field forward. SSSCR is here to help!

*I want to thank everyone who serves in the research and medical areas. Without you our field would stop in its tracks. However, not everyone is cut out for such positions. Luckily, there are other options.

**Some reading this might say “awwwww, too bad, she was so close to that PhD” and some might say “that’s a major accomplishment and you can do a lot with that degree!” Both are right, but I choose to believe the latter, as I am so much happier now that I released myself from the allure of lab research and went into science communications. We tend to hold science and medicine up on pedestals; however, science communication facilitates almost all interactions between academic and industry scientists, clinicians, and the public. An understanding of and engagement with new science is critical to promoting a healthy democracy with citizens who can make informed decisions about their society’s future.

Laurel is a co-founder of SSSCR, the current Associate Director, and a member of the SSSCR International executive committee. She has been involved in SSSCR since 2004, when she helped start UC Berkeley’s chapter. Her main contributions are educating various communities about stem cell research and building career development opportunities for students. Along with a team of SSSCR members, Laurel created the California Institute for Regenerative Medicine’s stem cell education portal to provide teachers with the materials they need to engage students with the field. Currently, Laurel is a Senior Technical Writer focused on stem cell products and services.

FDA creates a forum for patients to guide its decision making

FDA

It’s not hard to find people who don’t like the US Food and Drug Administration (FDA), the government agency that, among other things, regulates medical therapies. In fact, if you type “do people like the FDA?” into an internet search engine you’ll quickly find out that for a lot of people the answer is “no”.

But the Agency is trying to change and deserves credit for taking seriously many of the criticisms that have been levelled at it over the years and trying to address them.

The latest example is the news that the FDA has set a date for the first-ever meeting of its first-ever Patient Engagement Advisory Committee (PEAC). On its website, the FDA says the PEAC will be focused on patient-related issues:

“The PEAC is a forum for the voice of patients. It will be asked to advise on complex issues related to medical devices and their impact on patients. The goal of PEAC is to better understand and integrate patient perspectives into our oversight, to improve communications with patients about benefits, risks, and clinical outcomes related to medical devices, and to identify new approaches, unforeseen risks or barriers, and unintended consequences from the use of medical devices.”

In the past, the FDA has created forums to allow patients to talk about the impact of a disease on their daily life and their views on treatment options. But those were considered by many to be little more than window dressing, providing a sounding boards for patients but not actually producing any tangible benefits or changes.

The FDA also has patient representatives who take part in FDA advisory committee meetings, but the PEAC is the first time it has ever had a committee that was solely focused on patients and their needs. The nine core members of the PEAC all have experience either as patients or patient advocates and care-givers for patients. A really encouraging sign.

We tip our CAP to the FDA

At CIRM we support anything that ensures that patients not only have a seat at the table, but also that their voices are heard and taken seriously. That’s why for every clinical trial we fund (and even some pre-clinical projects too) we create what we call a Clinical Advisory Panel or CAP (we do love our acronyms).

Each CAP consists of three to five members, with a minimum of one Patient Representative, one External Advisor and one CIRM Science Officer. The purpose of the CAP is to make recommendations and provide guidance and advice to the Project Team running the trial.

Having a Patient Representative on a CAP ensures the patient’s perspective is included in shaping the design of the clinical trial, making sure that the trial is being carried out in a way that has the patient at the center. Patients can ask questions or raise issues that researchers might not think about, and can help the researchers not only do a better job of recruiting the patients they need for the trial, but also keeping those patients involved. We believe a trial designed around the patient, and with the patient in mind, is much more likely to be successful.

In announcing the formation of the PEAC the FDA said:

“Patients are at the heart of what we do. It makes sense to establish an advisory committee built just for them.”

I completely agree.

My only regret is that they didn’t call it the Patient Engagement Advisory Committee for Health, because then the acronym would have been PEACH. And this is certainly a peach of an idea, one worthy of support.

Related Links:

 

 

 

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

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

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

By that standard Caleb Sizemore is a spellbinding speaker.

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

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

Video produced by Todd Dubnicoff/CIRM


Related Links:

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

ALS

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

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

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

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

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

BrainStorm

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

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

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

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

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

CIRM Board member and ALS Patient Advocate Diane Winokur

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

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

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

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

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

CIRM & NIH: a dynamic duo to advance stem cell therapies

NIH

National Institutes of Health

There’s nothing more flattering than to get an invitation, out of the blue, from someone you respect, and be told that they are interested in learning about the way you work, to see if it can help them improve the way they work.

That’s what happened to CIRM recently. I will let Randy Mills, who was our President & CEO at the time, pick up the story:

“Several weeks ago I got a call from the head of the National Heart. Lung and Blood Institute (NHLBI) asking would we be willing to come out to the National Institutes of Health (NIH) and talk about what we have been doing, the changes we have made and the impact they are having.”

Apparently people at the NIH had been reading our Strategic Plan and our Annual Report and had been hearing good things about us from many different individuals and organizations. We also heard that they had been motivated to engage more fully with the regenerative medicine community following the passage of the 21st Century Cures Act.

We were expecting a sit down chat with them but we got a lot more than that. They blocked out one and a half days for us so that we had the time to engage in some in-depth, thoughtful conversations about how to advance the field.

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Dr. Francis Collins, NIH Director

The meeting was kicked off by both Francis Collins, the NIH Director, and Gary Gibbons, the NHLBI Director. Then the CIRM team – Dr. Mills, Dr. Maria Millan, Gabe Thompson and James Harrison – gave a series of presentations providing an overview of how CIRM operates, including our vision and strategic priorities, our current portfolio, the lessons learned so far, our plans for the future and the challenges we face.

The audience included the various heads and representatives from the various NIH Institutes who posed a series of questions for us to answer, such as:

  • What criteria do we use to determine if a project is ready for a clinical trial?
  • How do we measure success?
  • How have our strategies and priorities changed under CIRM 2.0?
  • How well are those strategies working?

The conversation went so well that the one day of planned meetings were expanded to two. Maria Millan, now our interim President & CEO, gave an enthusiastic summary of the talks

“The meetings were extremely productive!  After meeting with Dr. Collins’ group and the broader institute, we had additional sit down meetings.   The NIH representatives reported that they received such enthusiastic responses from Institute heads that they extended the meeting into a second day. We met with with the National Institutes of Dental and Craniofacial Research, Heart, Lung and Blood, Eye Institute, Institute on Aging, Biomedical Imaging and Bioengineering, Diabetes, and Digestive and Kidney Diseases, and the National Center for Advancing Translational Sciences.  We covered strategic and operational considerations for funding the best science in the stem cell and regenerative medicine space.  We explored potential avenues to join forces and leverage the assets and programs of both organizations, to accelerate the development of regenerative medicine and stem cell treatments.”

This was just a first meeting but it laid the groundwork for what we hope will be a truly productive partnership. In fact, shortly after returning from Washington, D.C., CIRM was immediately invited to follow-up NIH workgroups and meetings.

As this budding partnership progresses we’ll let you know how it’s working out.

Baseball’s loss is CIRM’s gain as Stanford’s Linda Boxer is appointed to Stem Cell Agency Board

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Dr. Linda Boxer: Photo courtesy Stanford University

One of the things that fascinates me is finding out how people end up in the job they have, the job they love. It is rare that the direction they started out on is the one they end on. Usually, people take several different paths, some intended, some unintended, to get to where they want to be.

A case in point is Dr. Linda Boxer, a renowned and respected researcher and physician at the Stanford School of Medicine, and now the newest member of the CIRM Board (you can read all about that in our news release).

In Dr. Boxer’s case, her original career path was a million miles from working with California’s stem cell agency:

“The first career choice that I recall as a young child was professional baseball—growing up in Minnesota, I was a huge Twins fan—I did learn fairly quickly that this was not likely to be a career that was available for a girl, and it wasn’t clear what one did after that career ended at a relatively young age.”

Fortunately for us she became interested in science.

“I have always been curious about how things work—science classes in grade school were fascinating to me. I was given a chemistry kit as a birthday gift, and I was amazed at what happened when different chemicals were mixed together: color changes, precipitates forming, gas bubbles, explosions (small ones, of course).

Then when we studied biology in middle school, I was fascinated by what one could observe with a microscope and became very interested in trying to understand how living organisms work.

It was an easy decision to plan a career in science.  The tougher decision came in college when I had planned to apply to graduate school and earn a PhD, but I was also interested in human health and disease and thought that perhaps going to medical school made more sense.  Fortunately, one of my faculty advisors told me about combined MD/PhD programs, and that choice seemed perfect for me.”

Along the way she says she got a lot of help and support from her colleagues. Now she wants to do the same for others:

“Mentors are incredibly important at every career stage.  I have been fortunate to have been mentored by some dedicated scientists and physicians.  Interestingly, they have all been men.  There were really very few women available as mentors at the time—of course, that has changed for the better now.  It never occurred to me then that gender made a difference, and I just looked for mentors who had successful careers as scientists and physicians and who could provide advice to someone more junior.

One of the aspects of my role now that I enjoy the most is mentoring junior faculty and trainees.  I don’t think one can have too many mentors—different mentors can help with different aspects of one’s life and career.  I think it is very important for established scientists to give back and to help develop the next generation of physicians and scientists.”

Dr. Boxer is already well known to everyone at CIRM, having served as the “alternate” on the Board for Stanford’s Dr. Lloyd Minor. But her appointment by State Controller Betty Yee makes her the “official” Board member for Stanford. She brings a valuable perspective as both a scientist and a physician.

The Minnesota Twins lost out when she decided to pursue a career in science. We’re glad she did.

 

jCyte gets FDA go-ahead for Fast Track review process of Retinitis Pigmentosa stem cell therapy

21 century cures

When the US Congress approved, and President Obama signed into law, the 21st Century Cures Act last year there was guarded optimism that this would help create a more efficient and streamlined, but no less safe, approval process for the most promising stem cell therapies.

Even so many people took a wait and see approach, wanting a sign that the Food and Drug Administration (FDA) would follow the recommendations of the Act rather than just pay lip service to it.

This week we saw encouraging signs that the FDA is serious when it granted Regenerative Medicine Advanced Therapy (RMAT) status to the CIRM-funded jCyte clinical trial for a rare form of blindness. This is a big deal because RMAT seeks to accelerate approval for stem cell therapies that demonstrate they can help patients with unmet medical needs.

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jCyte co-founder Dr. Henry Klassen

jCyte’s work is targeting retinitis pigmentosa (RP), a genetic disease that slowly destroys the cells in the retina, the part of the eye that converts light into electrical signals which the brain then interprets as vision. At first people with RP lose their night and peripheral vision, then the cells that help us see faces and distinguish colors are damaged. RP usually strikes people in their teens and, by the time they are 40, many people are legally blind.

jCyte’s jCell therapy uses what are called retinal progenitor cells, injected into the eye, which then release protective factors to help repair and rescue diseased retinal cells. The hope is this will stop the disease’s progression and even restore some vision to people with RP.

Dr. Henry Klassen, jCyte’s co-founder and a professor at UC Irvine, was understandably delighted by the designation. In a news release, he said:

“This is uplifting news for patients with RP. At this point, there are no therapies that can help them avoid blindness. We look forward to working with the FDA to speed up the clinical development of jCell.”

FDA

On the FDA’s blog – yes they do have one – it says researchers:

“May obtain the RMAT designation for their drug product if the drug is intended to treat serious or life-threatening diseases or conditions and if there is preliminary clinical evidence indicating that the drug has the potential to address unmet medical needs for that disease or condition. Sponsors of RMAT-designated products are eligible for increased and earlier interactions with the FDA, similar to those interactions available to sponsors of breakthrough-designated therapies. In addition, they may be eligible for priority review and accelerated approval.”

Paul Bresge

jCyte CEO Paul Bresge

jCyte is one of the first to get this designation, a clear testimony to the quality of the work done by Dr. Klassen and his team. jCyte CEO Paul Bresge says it may help speed up their ability to get this treatment to patients.

 

“We are gratified by the FDA’s interest in the therapeutic potential of jCell and greatly appreciate their decision to provide extra support. We are seeing a lot of momentum with this therapy. Because it is well-tolerated and easy to administer, progress has been rapid. I feel a growing sense of excitement among patients and clinicians. We look forward to getting this critical therapy over the finish line as quickly as possible.”

Regular readers of this blog will already be familiar with the story of Rosie Barrero, one of the first group of people with RP who got the jCell therapy. Rosie says it has helped restore some vision to the point where she is now able to read notes she wrote ten years ago, distinguish colors and, best of all, see the faces of her children.

RMAT is no guarantee the therapy will be successful. But if the treatment continues to show promise, and is safe, it could mean faster access to a potentially life-changing therapy, one that could ultimately rescue many people from a lifetime of living in the dark.

 

 

CIRM’s Randy Mills leaving stem cell agency to take on new challenge

Mills, Randy Union Tribune K.C. Alfred

Some news releases are fun to write. Some less so. The one that CIRM posted today definitely falls into that latter group. It announced that CIRM’s President and CEO, Randy Mills, is leaving us to take up the role of President and CEO at the National Marrow Donor Program – NMPD/Be The Match.

It’s a great opportunity for him but a big loss for us.

Be The Match is a non-profit organization that delivers cures to patients in need of a life-saving marrow or cord blood transplant. The organization operates the national Be The Match Registry®—the world’s largest listing of potential marrow donors and donated umbilical cord blood units—matches patients with their marrow donor, educates healthcare professionals and conducts research so more lives can be saved. The organization also recently created a subsidiary—Be The Match BioTherapiesSM—that supports organizations pursuing new life-saving treatments in cellular therapy.

Randy has been at CIRM since April 2014. In that time he has dramatically re-shaped the agency, and, more importantly, dramatically improved the speed with which we are able to fund research. It’s no exaggeration to say that Randy’s drive to create CIRM 2.0 was a radical overhaul of the way we work. It made it easier for researchers to apply to us for funding, made our funding cycles more consistent and the application process simpler – though no less rigorous.

As our CIRM Board Chair Jonathan Thomas said in the news release:

“CIRM has experienced a remarkable transformation since Randy’s arrival. He has taken the agency to a new level by developing and implementing a bold strategic plan, the results of which include an 82% reduction in approval time for clinical trial projects, a 3-fold increase in the number of clinical trials, and a 65% reduction in the time it takes to enroll those trials. The opportunity for Randy to lead a tremendously important organization such as the NMDP/Be The Match is consistent with the values he demonstrated at CIRM, which put the well-being of patients above all else. We shall miss him but know he will do great things at NMDP/Be The Match.”

From a personal perspective, what most impressed me about Randy was his willingness to involve every person in the agency in changing the way we work. He could easily have come in and simply issued orders and told people what to do. Instead he invited every person at CIRM to sit in on the meetings that were shaping the new direction we took. You didn’t have to go, but if you did you were expected to offer thoughts and ideas. No sitting idly by.

Those meetings not only changed the direction of the agency, they also re-energized the agency. When people feel their voice is being heard, that their opinion has value, they respond by working harder and smarter.

The CIRM of today has the same mission as always – accelerating stem cell treatments to patients with unmet medical needs – but the people working here seem to have a renewed commitment to making that mission a reality.

Randy brought to CIRM energy and a renewed sense of purpose, along with some truly terrible jokes and a strange conviction that he could have been a great rock and roll drummer (suffice to say he made the right career choice when he went into research).

He changed us as an agency, for the better. We shall miss him, but know he will do great things in his new role at NMDP/Be The Match and we wish him success in his new job, and his family great joy in their new home.

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Maria Millan

Randy will be with us till the end of June and starting July 1st Dr. Maria Millan will take on the role of interim President and CEO.

 

 

 

You Are Invited: CIRM Patient Advocate Event, San Diego April 20th

Cured_AR_2016_cover

The word “cured” is one of the loveliest words in the English language. Last year we got to use it twice when we talked about stem cell therapies we are funding. Two of our clinical trials are not just helping people, they are curing them (you can read about that in our Annual Report).

But this was just part of the good news about stem cell research. We are making progress on many different fronts, against many different diseases, and we want to tell you all about that.

That’s why we are holding a special Patient Advocate event at UC San Diego on Thursday, April 20th from 12 – 1pm to talk about the progress being made in stem cell research, the problems we still face and need help in overcoming, and the prospects for the future.

We will have four terrific speakers:

  • Catriona Jamieson, Director of the CIRM UC San Diego Alpha Stem Cell Clinic and an expert on cancers of the blood
  • Jonathan Thomas, PhD, JD, Chair of CIRM’s Board
  • Jennifer Briggs Braswell, Executive Director of the Sanford Stem Cell Clinical Center
  • David Higgins, Patient Advocate for Parkinson’s on the CIRM Board

We will give updates on the exciting work taking place at UCSD and the work that CIRM is funding. We have also set aside some time to get your thoughts on how we can improve the way we work and, of course, answer your questions.

So we would love for you to join us, and tell your friends about the event as well. Here are the basic details.

What: Stem Cell Therapies and You: A Special Patient Advocate Event

When: Thursday, April 20th 12-1pm

Where: The Sanford Consortium for Regenerative Medicine, 2880 Torrey Pines Scenic Drive, La Jolla, CA 92037

Why: Because the people of California have a right to know how their money is helping change the face of regenerative medicine

Who: This event is FREE and open to the public

We have set up an EventBrite page for people to RSVP and let us know if they are coming.

We hope to see you there.

 

Raising awareness about Rare Disease Day

rare-disease-day-logo

One of the goals we set ourselves at CIRM in our 2016 Strategic Plan was to fund 50 new clinical trials over the next five years, including ten rare or orphan diseases. Since then we have funded 13 new clinical trials including four targeting rare diseases (retinitis pigmentosa, severe combined immunodeficiency, ALS or Lou Gehrig’s disease, and Duchenne’s Muscular Dystrophy). It’s a good start but clearly, with almost 7,000 rare diseases, this is just the tip of the iceberg. There is still so much work to do.

And all around the world people are doing that work. Today we have asked Emily Walsh, the Community Outreach Director at the Mesothelioma Cancer Alliance,  to write about the efforts underway to raise awareness about rare diseases, and to raise funds for research to develop new treatments for them.

“February 28th marks the annual worldwide event for Rare Disease Day. This is a day dedicated to raising awareness for rare diseases that affect people all over the world. The campaign works to target the general public as well as policy makers in hopes of bringing attention to diseases that receive little attention and funding. For the year 2017 it was decided that the focus would fall on “research,” with the slogan, “With research, possibilities are limitless.”

Getting involved for Rare Disease Day means taking this message and spreading it far and wide. Awareness for rare diseases is extremely important, especially among researchers, universities, students, companies, policy makers, and clinicians. It has long been known that the best advocates for rare diseases are the patients themselves. They use their specific perspectives to raise their voice, share their story, and shed light on the areas where additional funding and research are most necessary.

To see how you can help support the Rare Disease Day efforts this year, click here.

Groups like the Mesothelioma Cancer Alliance and the Mesothelioma Group are adding their voices to the cause to raise awareness about mesothelioma cancer, a rare form of cancer caused by exposure and inhalation of airborne asbestos fibers

Rare diseases affect 300 million people worldwide, but only 5% of them have an FDA approved treatment or cure. Malignant mesothelioma is among the 95 percent that doesn’t have a treatment or cure.

Asbestos has been used throughout history in building materials because of its fire retardant properties. Having a home with asbestos insulation, ceiling tiles, and roof shingles meant that the house was safer. However, it was found that once asbestos crumbled and became powder-like, the tiny fibers could become airborne and be inhaled and lodge themselves in lung tissue causing mesothelioma. The late stage discovery of mesothelioma is often what causes it to have such a high mortality rate. Symptoms can have a very sudden onset, even though the person may have been exposed decades prior.

Right now, treatment for mesothelioma includes the usual combination of chemotherapy, radiation, and surgery, but researchers are looking at other approaches to see if they can be more effective or can help in conjunction with the standard methods. For example one drug, Defactinib, has shown some promise in inhibiting the growth and spread of cancer stem cells – these are stem cells that can evade chemotherapy and cause patients to relapse.”

Some people might ask why spend limited resources on something that affects so few people. But the lessons we learn in developing treatments for a rare disease can often lead us to treatments for diseases that affect many millions of people.

But numbers aside, there is no hierarchy of need, no scale to say the suffering of people with Huntington’s disease is any greater or less than that of people with Alzheimer’s. We are not in the business of making value judgements about who has the greatest need. We are in the business of accelerating treatments to patients with unmet medical needs. And those suffering from rare disease are very clearly  people in need.

 


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