Patient Advocacy

Advancing Stem Cell Research at the CIRM Bridges Conference

/ Karen Ring / Leave a comment

Where will stem cell research be in 10 years?

What would you say to patients who wanted stem cell therapies now?

What are the most promising applications for stem cell research?

Why is it important for the government to fund regenerative medicine?

These challenging and thought-provoking questions were posed to a vibrant group of undergraduate and masters-level students at this year’s CIRM Bridges to Stem Cell Research and Therapy conference.

Educating the next generation of stem cell scientists

The Bridges program is one of CIRM’s educational programs that offers students the opportunity to take coursework at California state schools and community colleges and conduct stem cell research at top universities and industry labs. Its goal is to train the next generation of stem cell scientists by giving them access to the training and skills necessary to succeed in this career path.

The Bridges conference is the highlight of the program and the culmination of the students’ achievements. It’s a chance for students to showcase the research projects they’ve been working on for the past year, and also for them to network with other students and scientists.

Bridges students participated in a networking pitch event about stem cell research.

Bridges students participated in a networking pitch event about stem cell research.

CIRM kicked off the conference with a quick and dirty “Stem Cell Pitch” networking event. Students were divided into groups, given one of the four questions above and tasked with developing a thirty second pitch that answered their question. They were only given ten minutes to introduce themselves, discuss the question, and pick a spokesperson, yet when each team’s speaker took the stage, it seemed like they were practiced veterans. Every team had a unique, thoughtful answer that was inspiring to both the students and to the other scientists in the crowd.

Getting to the clinic and into patients

The bulk of the Bridges conference featured student poster presentations and scientific talks by leading academic and industry scientists. The theme of the talks was getting stem cell research into the clinic and into patients with unmet medical needs.

Here are a few highlights and photos from the talks:

On the clinical track for Huntington’s disease

Leslie Thompson, Professor at UC Irvine, spoke about her latest research in Huntington’s disease (HD). She described her work as a “race against time.” HD is a progressive neurodegenerative disorder that’s associated with multiple social and physical problems and currently has no cure. Leslie described how her lab is heading towards the clinic with human embryonic stem cell-derived neural (brain) stem cells that they are transplanting into mouse models of HD. So far, they’ve observed positive effects in HD mice that received human neural stem cell transplants including an improvement in the behavioral and motor defects and a reduction in the accumulation of toxic mutant Huntington protein in their nerve cells.

Leslie Thompson

Leslie Thompson

Leslie noted that because the transplanted stem cells are GMP-grade (meaning their quality is suitable for use in humans), they have a clear path forward to testing their potential disease modifying activity in human clinical trials. But before her team gets to humans, they must take the proper regulatory steps with the US Food and Drug Administration and conduct further experiments to test the safety and proper dosage of their stem cells in other mouse models as well as test other potential GMP-grade stem cell lines.

Gene therapy for SCID babies

Morton Cowan, a pediatric immunologist from UC San Francisco, followed Leslie with a talk about his efforts to get gene therapy for SCID (severe combined immunodeficiency disease) off the bench into the clinic. SCID is also known as bubble-baby disease and put simply, is caused by a lack of a functioning immune system. SCID babies don’t have normal T and B immune cell function and as a result, they generally die of infection or other conditions within their first year of life.

Morton Cowan

Morton Cowan, UCSF

Morton described how the gold standard treatment for SCID, which is hematopoietic or blood stem cell transplantation, is only safe and effective when the patient has an HLA matched sibling donor. Unfortunately, many patients don’t have this option and face life-threatening challenges of transplant rejection (graft-versus host disease). To combat this issue, Morton and his team are using gene therapy to genetically correct the blood stem cells of SCID patients and transplant those cells back into these patients so that they can generate healthy immune cells.

They are currently developing a gene therapy for a particularly hard-to-treat form of SCID that involves deficiency in a protein called Artemis, which is essential for the development of the immune system and for repairing DNA damage in cells. Currently his group is conducting the necessary preclinical work to start a gene therapy clinical trial for children with Artemis-SCID.

Treating spinal cord injury in the clinic

Casey Case, Asterias Biotherapeutics

Casey Case, Asterias Biotherapeutics

Casey Case, Senior VP of Research and Nonclinical Development at Asterias Biotherapeutics, gave an update on the CIRM-funded clinical trial for cervical (neck) spinal cord injury (SCI). They are currently testing the safety of transplanting different doses of their oligodendrocyte progenitor cells (AST-OPC1) in a group of SCI patients. The endpoint for this trial is an improvement in movement greater than two motor levels, which would offer a significant improvement in a patient’s ability to do some things on their own and reduce the cost of their healthcare. You can read more about these results and the ongoing study in our recent blogs (here, here).

Opinion: Scientists should be patient advocates

David Higgins gave the most moving speech of the day. He is a Parkinson’s patient and the Patient Advocate on the CIRM board and he spoke about what patient advocates are and how to become one. David explained how, these days, drug development and patient advocacy is more patient oriented and patients are involved at the center of every decision whether it be questions related to how a drug is developed, what side effects should be tolerated, or what risks are worth taking. He also encouraged the Bridges students to become patient advocates and understand what their needs are by asking them.

David Higgins, Parkinson's advocate and CIRM Board member

David Higgins

“As a scientist or clinician, you need to be an ambassador. You have a job of translating science, which is a foreign language to most people, and you can all effectively communicate to a lay audience without being condescending. It’s important to understand what patients’ needs are, and you’ll only know that if you ask them. Patients have amazing insights into what needs to be done to develop new treatments.”

Bridging the gap between research and patients

The Bridges conference is still ongoing with more poster presentations, a career panel, and scientific talks on discovery and translational stem cell research and commercializing stem cell therapies to all patients in need. It truly is a once in a lifetime opportunity for the Bridges students, many of whom are considering careers in science and regenerative medicine and are taking advantage of the opportunity to talk and network with prominent scientists.

If you’re interested in hearing more about the Bridges conference, follow us on twitter (@CIRMnews, @DrKarenRing, #CIRMBridges2016) and on Instagram (@CIRM_Stemcells).

Another way to dial back stem cell hype (but not hope): Put a dollar figure on it

/ Lisa Willemse / 2 Comments

In an effort to reign in the hype surrounding stem cell research that has led to a proliferation of unapproved and potentially dangerous stem cell therapies, the International Society for Stem Cell Research (ISSCR) recently released updated guidelines outlining conduct for stem cell researchers that,  for the first time, included communications activities.  At only 1.5 pages in the 37-page document, the statements around communications asked researchers, communications professionals, institutions and the media to be more proactive in combatting stem cell hype by ensuring accuracy and balance in communications activities.

Stock Image

Stock Image

It’s too early to know what the full impact of the guidelines will be, however, the communications recommendations did generate a good deal of interest and some media, at least, have taken steps to address the issue.

Whether directly influenced by the guidelines or not, in the final plenary session of the ISSCR annual meeting last week, Professor Roger Barker, a research-clinician at the University of Cambridge, provided a candid portrayal of some of the challenges of preclinical and early clinical research.

Though he may have poked a small hole in some of the optimism that characterized the four-day conference, in providing a rare glimpse of the real costs of research, Dr. Barker might also have given us a new way to frame research to downplay hype.

Dr. Roger Barker

Dr. Roger Barker

Dr. Barker is one of many researchers across the globe working on a potential cell-based treatment for Parkinson’s Disease. Parkinson’s is a rather straightforward disease to tackle in this way, because its cause is known: the death of cells that produce the chemical dopamine. Even so, the challenges in developing a treatment are many. Apart from the design of a clinical study (which includes, for example, careful selection of the Parkinson’s patients to include; as Barker pointed out, there are two main types of Parkinson progression and one type may respond to a treatment while the other may not. This is a real concern for Barker, who commented that “a lack of rigour in selecting patients has dogged the field for the past 25 years.”), there are several other factors that need to be addressed in the pre-clinical work, such as identifying the best type of cells to use, how to scale them up and make them both GMP-compliant and standardized for reproducibility.

Such work, Barker estimated, costs between £2 and £3 million (or roughly $3-5 million, valued at pre-Brexit currency rates, one would assume). And, having invested so much to this point, you don’t even have something that can be published yet.

Running the actual clinical phase 1 study, with roughly 20 patients, will cost millions more. If it doesn’t work, you’re back to lab and in search of more pre-clinical funding.

But, assuming the study nets the desired results, it’s still only looking at safety, not efficacy. Getting it to phases 2 and 3 costs several orders of magnitude more. Put in this light, the $3 billion USD given to the California Institute for Regenerative Medicine seems like not nearly enough. The Ontario Institute for Regenerative Medicine’s $25 million CAD is nothing at all. Not that we aren’t grateful — we do what we can to maximize impact and make even a small investment worthwhile. Every step counts.

Another point to consider is whether the final therapy will be more cost-effective than existing, approved medical interventions. If it’s not, there is little incentive in pursuing it. This is the notion of headroom that I’ve heard discussed more directly at commercialization-based conferences (and is very well explained here) but is one that will become increasingly relevant to research as more basic and translational work finds its way into the clinic.

Talking about money with regard to health can be seen as tedious and even crass. The three short talks given by patient advocates at the ISSCR meeting served to emphasize this – each outlined personal tragedy connected to illness or disease: congestive heart failure at 11 years of age, four generations of a family with sickle cell disease, retinitis pigmentosa that derailed a young woman’s budding career. You simply can’t put a price on a person’s life, happiness and well-being. Each of these patients, and millions more, have hope that research will find an answer. It’s a lofty goal, one that is sometimes hard to remember in the lab trenches when a grant doesn’t materialize or a negative result sends the work back to ground zero.

And therein lies some of the tension that can easily lead to hype. We do want to fly high. We do want to deliver cures and therapies. We need to be reminded, by interactions with the patient community, of what’s at stake and what we can gain for humanity. The field should and will continue to strive to achieve these goals.

But not without responsibility. And a dose of realism.

This post appears simultaneously on OIRM Expression and appears here with permission by the author Lisa Willemse.

Multi-Talented Stem Cells: The Many Ways to Use Them in the Clinic

/ Karen Ring / Leave a comment

CIRM kicked off the 2016 International Society for Stem Cell Research (ISSCR) Conference in San Francisco with a public stem cell event yesterday that brought scientists, patients, patient advocates and members of the general public together to discuss the many ways stem cells are being used in the clinic to develop treatments for patients with unmet medical needs.

Bruce Conklin, Gladstone Institutes & UCSF

Bruce Conklin, Gladstone Institutes & UCSF

Bruce Conklin, an Investigator at the Gladstone Institutes and UCSF Professor, moderated the panel of four scientists and three patient advocates. He immediately captured the audience’s attention by showing a stunning video of human heart cells, beating in synchrony in a petri dish. Conklin explained that scientists now have the skills and technology to generate human stem cell models of cardiomyopathy (heart disease) and many other diseases in a dish.

Conklin went on to highlight four main ways that stem cells are contributing to human therapy. First is using stem cells to model diseases whose causes are still largely unknown (like with Parkinson’s disease). Second, genome editing of stem cells is a new technology that has the potential to offer cures to patients with genetic disorders like sickle cell anemia. Third, stem cells are known to secrete healing factors, and transplanting them into humans could be beneficial. Lastly, stem cells can be engineered to attack cancer cells and overcome cancer’s normal way of evading the immune system.

Before introducing the other panelists, Conklin made the final point that stem cell models are powerful because scientists can use them to screen and develop new drugs for diseases that have no treatments or cures. His lab is already working on identifying new drugs for heart disease using human induced pluripotent stem cells derived from patients with cardiomyopathy.

Scientists and Patient Advocates Speak Out

Malin Parmar, Lund University

Malin Parmar, Lund University

The first scientist to speak was Malin Parmar, a Professor at Lund University. She discussed the history of stem cell development for clinical trials in Parkinson’s disease (PD). Her team is launching the first in-human trial for Parkinson’s using cells derived from human pluripotent stem cells in 2016. After Parmar’s talk, John Lipp, a PD patient advocate. He explained that while he might look normal standing in front of the crowd, his PD symptoms vary wildly throughout the day and make it hard for him to live a normal life. He believes in the work that scientists like Parmar are doing and confidently said, “In my lifetime, we will find a stem cell cure for Parkinson’s disease.”

Adrienne Shapiro, Patient Advocate

Adrienne Shapiro, Patient Advocate

The next scientist to speak was UCLA Professor Donald Kohn. He discussed his lab’s latest efforts to develop stem cell treatments for different blood disorder diseases. His team is using gene therapy to modify blood stem cells in bone marrow to treat and cure babies with SCID, also known as “bubble-boy disease”. Kohn also mentioned their work in sickle cell disease (SCD) and in chronic granulomatous disease, both of which are now in CIRM-funded clinical trials. He was followed by Adrienne Shapiro, a patient advocate and mother of a child with SCD. Adrienne gave a passionate and moving speech about her family history of SCD and her battle to help find a cure for her daughter. She said “nobody plans to be a patient advocate. It is a calling born of necessity and pain. I just wanted my daughter to outlive me.”

Henry Klassen (UC Irvine)

Henry Klassen, UC Irvine

Henry Klassen, a professor at UC Irvine, next spoke about blinding eye diseases, specifically retinitis pigmentosa (RP). This disease damages the photo receptors in the back of the eye and eventually causes blindness. There is no cure for RP, but Klassen and his team are testing the safety of transplanting human retinal progenitor cells in to the eyes of RP patients in a CIRM-funded Phase 1/2 clinical trial.

Kristen MacDonald, RP patient

Kristen MacDonald, RP patient

RP patient, Kristen MacDonald, was the trial’s first patient to be treated. She bravely spoke about her experience with losing her vision. She didn’t realize she was going blind until she had a series of accidents that left her with two broken arms. She had to reinvent herself both physically and emotionally, but now has hope that she might see again after participating in this clinical trial. She said that after the transplant she can now finally see light in her bad eye and her hope is that in her lifetime she can say, “One day, people used to go blind.”

Lastly, Catriona Jamieson, a professor and Alpha Stem Cell Clinic director at UCSD, discussed how she is trying to develop new treatments for blood cancers by eradicating cancer stem cells. Her team is conducting a Phase 1 CIRM-funded clinical trial that’s testing the safety of an antibody drug called Cirmtuzumab in patients with chronic lymphocytic leukemia (CLL).

Scientists and Patients need to work together

Don Kohn, Catriona Jamieson, Malin Parmar

Don Kohn, Catriona Jamieson, Malin Parmar

At the end of the night, the scientists and patient advocates took the stage to answer questions from the audience. A patient advocate in the audience asked, “How can we help scientists develop treatments for patients more quickly?”

The scientists responded that stem cell research needs more funding and that agencies like CIRM are making this possible. However, we need to keep the momentum going and to do that both the physicians, scientists and patient advocates need to work together to advocate for more support. The patient advocates in the panel couldn’t have agreed more and voiced their enthusiasm for working together with scientists and clinicians to make their hopes for cures a reality.

The CIRM public event was a huge success and brought in more than 150 people, many of whom stayed after the event to ask the panelists more questions. It was a great kick off for the ISSCR conference, which starts today. For coverage, you can follow the Stem Cellar Blog for updates on interesting stem cell stories that catch our eye.

CIRM Public Stem Cell Event

CIRM Public Stem Cell Event

Patient advocates a small but mighty force at BIO meeting

/ Don Gibbons / 2 Comments
Patient Advocacy Pavilion at BIO2016

Patient Advocacy Pavilion at BIO2016

A few hundred patient advocates operating from a small sub-section carved out of three cavernous exhibit halls could easily get lost amid the 16,000 scientists and business folks attending the BIO International meeting in San Francisco last week. But their voice was heard as they made great use of the meeting to remind companies developing therapies that they are the end user. They are the reason why the companies exist.

Talking to many advocates representing their constituents from the tiny two-foot by one-foot shelves and a stool they were each given within the advocate zone a couple of consensus points came through. The meeting provided incredibly valuable contacts for the patient advocates, and the attitudes of the companies are changing.

 “We want to make people aware that family caregivers are making care decisions,” said Mark Gibbons of the Caregiver Action Network. “It has been wonderful having companies reach out to us rather than us making cold calls on them.”

Bill Remak of the California Chronic Care Coalition had similar thoughts on the changing attitude, but on a different aspect of the patient-company interface:

 “This has been a very good meeting; we made really good contacts and had great discussions on business models, pricing and making products accessible to patients. The mentality is changing to more concern on patient access.”

We had a lengthy discussion with Sean Elkins, chief science officer, and Allison Moore, CEO, of the Hereditary Neuropathy Foundation. They fight the battle to get therapies to their constituents on two fronts: The battle to get funding for the research as well as the added barrier of working with orphan diseases. They represent folks with Charcot-Marie-Tooth (CMT) disease and a half dozen related neurologic conditions. And while a prevalence of one in 2,500 makes it one of the more common orphan diseases, they have no treatments, and still have a hard time getting some company’s attention.

Allison Moore

Allison Moore and Sean Elkins

As a result, they initiate many research projects themselves with their own donor-derived funds and federal grants. In one effort they developed an assay for whether existing drug compounds could impact the nerves of patients with CMT. They have been testing many existing compounds and finding a few candidate therapies. But Elkins lamented on Twitter that he wished the drug companies would train their exhibit staff better about rare diseases. “When you approach some of them and say you have tested some of their products in an orphan disease they act like a deer in the headlights.”

His colleague, Moore, noted their efforts to take the bull by the horns and bring in the next generation of scientist/business people to tackle their diseases. “The highlight of the meeting for us has been meeting with former academics starting companies who are excited about the prospect of working on something new.”

Moore’s own story highlighted the dedication evident among the advocates at the meeting. She is a patient herself and not just a foundation executive. She worked the meeting so hard that by the third day she had bandages on both legs to cover the blisters from the braces that allow her to walk despite the underlying illness.

Everyone working the patient advocate zone at the meeting seemed pleased to have the chance to make connections that might one day make things a bit better for their constituents. This was the first time attending for the team from the California Chronic Care Coalition and the group’s CEO, Liz Helms, was exuberant in stating their time was well spent:

 “This meeting was over the top valuable; everything we expected and more.”

Get your BIO on: Sneak Peak of the June 2016 BIO Convention in SF

/ Karen Ring / Leave a comment

Screen Shot 2016-06-01 at 8.43.36 AM

Summer is almost here and for scientists around the world, that means it’s time to flock to one of the world’s biggest biotech meetings, the BIO International Convention.

This year, BIO is hosted in the lovely city of San Francisco. From June 6-9th, over 15,000 biotechnology and pharma leaders, as well as other professionals, academics, and patients will congregate to learn, educate, and network.

There’s something for everyone at this convention. If you check out the BIO agenda, you’ll find a plethora of talks, events, education sessions, and fire side chats on almost any topic related to science and biotechnology that you can imagine. The hard part will be deciding what to attend in only four short days.

For those going to BIO this year, make sure to check out the myBIO event planning tool that’s free for attendees and allows you to browse events and create a personalized agenda. You can also set up a professional profile that will share your background and networking interests with others at BIO. With this nifty tool, you can search for scientists, companies, and speakers you might want to connect with during the convention. Think of all the potential networking opportunities right at your fingertips!

Will Smith (source)

Will Smith (source)

For those who can’t make it to BIO, don’t worry, we have you covered. CIRM will be at the convention blogging and live tweeting. Because our mission is to bring stem cell treatments to patients with unmet medical needs, the majority of our coverage will be on talks and sessions related to regenerative medicine and patient advocacy. However, there are definitely some sessions outside these areas that we won’t want to miss such as the Tuesday Keynote talk by Dr. Bennet Omalu – who helped reveal the extent of brain damage in the NFL – and actor Will Smith – who plays Dr. Omalu in the movie ‘Concussion’. Their join talk is called “Knowledge Precipitates Evolution.”

Here’s a sneak peak of some of the other talks and events that we think will be especially interesting:

Monday June 6th

Education Sessions on Brain Health and Mitochondrial Disease

Moving Out of Stealth Mode: Biotech Journalists Offer Real-World Advice on Working with Media to Tell Your Story

“In this interactive panel discussion, well-known biotech reporters from print and online outlets will share their insights on how to successfully work with the media. Session attendees will learn critical needs of the media from what makes a story newsworthy to how to “pitch” a reporter to strategies for translating complicated science into a story for a broad audience.”

The Bioethics of Drug Development: You Decide

“A discussion of the critical bioethical issues innovative manufacturers face in today’s healthcare ecosystem. Panelists will provide insights from a diverse set of perspectives, including investors, the patient advocacy community, bioethicists and federal regulators.”

Tuesday June 7th

Fireside Chat with Robert Califf, Commissioner of the US Food and Drug Administration (FDA)

Fireside Chat with Janet Napolitano, President of the University of California

Casting a Wider Net in Alzheimer’s Research: The Diversity of Today’s Approaches and Signs of Progress

“Hear clinical researchers, biotech CEOs, and patient advocates explain how the field is pivoting from the failures of past approaches to make use of the latest generation of beta-amyloid research results as well as pursue alternative therapeutic angles to improve brain health.”

From Ebola to Zika: How Can We Go Faster in a Global Emergency?

“This interactive panel of public health and industry leaders will discuss what has been learned through our global response to Ebola and what is and is not applicable to Zika or other pathogens of pandemic potential.”

Wednesday June 8th

Curative Therapies: Aligning Policy with Science to Ensure Patient Access

“The promise of curative treatments creates an urgent need to ensure access for patients, promote an environment conducive to developing new treatments, and manage the concentration of healthcare expenses in a sustainable manner.  A diverse set of panelists will tackle the tough questions around curative therapies and discern what changes are necessary for our health care delivery system to meet the challenges they pose.”

An Evolving Paradigm: Advancing the Science of Patient Input in the Drug Development and Regulatory Processes

“This panel will explore advances in the field of assessing patient views and perspectives, and highlight how the patient voice is being incorporated into development programs and informing FDA review and approval decisions.”

A Media Perspective

“Any press is good press or so they say. You want your story known at the right time and in the right light, but how do you get industry journalist to notice you? What peaks their interest and how do they go about story discovery? What will they be looking to write about in the next 3 to 12 months? Three top journalists will discuss their approaches to keeping current and what makes a story newsworthy.”
Patient Advocacy Meetup

Over 40 patient advocacy organizations will be discussing their latest partnerships and developments in the areas of advancing disease research and drug development.

Thursday June 9th

Novel Advances in Cancer R&D: Meeting the Needs of the Patient

“This panel will feature the views of patients and advocates, regulators, and companies who are working to change the way in which we diagnose and evaluate patients with cancer by better understanding the underlying biology of their disease.”

 To follow our coverage of BIO, visit our Stem Cellar Blog or follow us on Twitter at @CIRMNews.

On the Hunt for Huntington’s Disease Treatments in the New Millennium

/ Karen Ring / Leave a comment

“Over the next five to ten years, we want to make Huntington’s disease an increasingly treatable condition.”

This bold and inspiring statement was made by Dr. Ray Dorsey at the inaugural HD-CARE symposium for Huntington’s disease (HD) research held at UC Irvine last month. The event brought together scientists, doctors, patients, family members, and caregivers to discuss the latest discoveries in HD research and to talk openly about how we can address the unmet needs of patients suffering from this terrible, deadly neurodegenerative disease.

IMG_0957

Symposium speakers and HD-CARE Board Members

The symposium was hosted by HD-CARE, a non-profit organization established three years ago to support HD research and patient care. Frances Saldaña, HD-CARE president and a CIRM patient advocate, established this symposium with the goal of bringing new hope to HD patients and their family members.

Frances Saldana, HD-CARE President & Patient Advocate

Frances Saldana

“There is so much exciting research taking place all over the world that one can hardly contain oneself with excitement and hope,” explained Saldaña. “It is only right to share this scientific information and breakthroughs in HD research that has undoubtedly given our families so much hope.”

Recent breakthroughs

The symposium featured talks by scientists and doctors that spanned a broad range of topics including the recent progress of using human stem cells to model HD, the hope and hype of gene editing, and the benefits of in vitro fertilization (IVF) for HD families.

Dr. Ray Dorsey, Professor at the University of Rochester Medical Center, gave the keynote address. Inspiring from the start, he captured the audience’s attention by posing the question, “Why are we here?” To which he answered, “I think we are here to change this sign, which says Huntington’s disease is a fatal genetic neurodegenerative disease, to one that says HD is an increasingly treatable condition. Over the next five to ten years, we want to make HD an increasingly treatable condition.”

Referencing the HIV epidemic in the 1980s, Dorsey pointed out that there is precedent. What was thought to be a disease with a rapid death sentence is now, decades later, a treatable condition, and his belief is that the same can be done for HD patients in the near future.

Dorsey next highlighted major clinical advances in HD treatment including a record ten drugs currently in development in 2016. Treatments that he felt had particular promise included a gene silencing therapy by Ionis Pharmaceuticals, which is the first treatment being tested in clinical trials that targets the cause of HD. Dorsey also mentioned two drugs, Pridopidine and SD-809 (a modified version of the FDA-approved drug Tetrabenazine), that are used to treat symptoms of HD.

My favorite part of the talk was the end where he described his latest efforts to develop digital biomarkers that use smart phones and wearables to monitor a patient’s response to HD treatments in their own home.  This technology will not only make it easier to determine which treatments are effective for HD, but will also improve the quality of care patients receive during clinical trials.

Dr. Ray Dorsey

Dr. Ray Dorsey

“We think that these devices, which allow us to make assessments of how people are doing with a given condition, will soon be able to connect patients to clinicians so they can receive care regardless of who they are or where they live. We hope that for Huntington’s disease, these tools and technologies will enable us to connect patients to effective treatments for HD.”

Battle cry for change

While the science at the symposium was certainly encouraging, the voices of the patients and patient advocates made the strongest impression. Many of them spoke out to share their stories or ask questions. Others, like Saldaña, advocated for faster progress towards a cure.

“This disease is one in which family members and friends need to rally together and demand that research be properly funded to end this generational disease.  It is not one in which policy makers can sit around and wonder if they should fund it…it is a five-alarm fire that needs immediate action, and from the families, a fierce battle cry asking from policy makers and decision makers to fund aggressive research to end Huntington’s disease.”

Julie Rosling, Frances Saldana,

Julie Rosling receives the Patient Advocacy Award from HD-CARE’s Frances Saldana and Karen Thornburn

A particularly moving event was the presentation of the 2016 Patient Advocacy Award to Julie Rosling. Members of the HD-CARE board presented Rosling with a trophy to honor her brave efforts in advocating for HD patient rights. Saldaña described how Rosling was fearless at a HD patient-focused drug development meeting with the FDA in DC last fall. Along with other patients, she stood up and challenged the FDA to move HD into the fast track category for approving clinical trials.

A similar demand for regulatory change was brought up during the symposium regarding the approval of stem cell treatments for HD. As the representative for CIRM, I had a few moments to talk about our new Stem Cell Champions campaign, which is actively recruiting patient advocates that can work with us to help make the FDA approval process for stem cell treatments faster and more efficient. Our colleagues at Americans for Cures also spoke briefly about their efforts to promote the acceleration of stem cell treatments and improve the lives of HD patients.

By the end of the symposium, there was an overwhelming feeling of accomplishment and more importantly a renewed sense of hope for the future of HD treatments.

“It was extremely successful and I believe everyone left feeling very optimistic about the future for HD families,” said Saldaña. “There is a light at the end of the tunnel.”

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Patient Advocates Ron Shapiro, Adrienne Shapiro, David Saldana, Frances Saldana, Daniel Medina with Karen Ring from CIRM

Related Links:

Two National Parkinson’s Disease Organizations Join Forces

/ davidrhiggins / Leave a comment
David Higgins, Parkinson's advocate and CIRM Board member

David Higgins, Parkinson’s advocate and CIRM Board member

Guest blogger David R. Higgins, PhD, is a Parkinson’s Patient Advocate and a CIRM ICOC board member.

Two national Parkinson’s organizations have decided to join forces: The Parkinson’s Action Network (PAN) will be integrated into The Michael J. Fox Foundation (MJFF). Both have served the Parkinson’s community in separate and important ways, but as MJFF CEO Todd Sherer said in a joint press release, “Working as a single entity, MJFF and PAN will bring the passion and commitment of our joint community to bear on articulating and advancing key public policy priorities affecting millions of PD patients and families.”

Michael J. Fox Foundationmjff_vert_rgb_logo_300

Best known for its namesake founder and mission to fund Parkinson’s research, MJFF has awarded more than $450 million to fund Parkinson’s research. MJFF has distinguished itself as a fundraising giant and has kept their eye on their goal of funding high-quality PD research always with the goal of a cure in mind. In addition to funding research, the MJFF is known for its database of Parkinson’s related clinical trials, which it vets and makes available to anyone interested through a system that works a lot like a matchmaking service (i.e. the right trial for the right person based on personal preferences and interests). To learn more about the MJFF follow the link: https://www.michaeljfox.org.

Parkinson’s Action Networksite-header2

PAN has been a Washington, DC-based organization created to be a single voice for a united Parkinson’s community, with a proven track record for effective advocacy for federal legislation and policy that considers the needs of the Parkinson’s community. We used to call this “lobbying”, now we call it “advocacy”. To learn more about PAN follow the link: http://parkinsonsaction.org.

One + One = ThreePAN-MJFF-homepage-img

Integration of PAN into MJFF creates a Parkinson’s patient advocacy trifecta that brings together the strengths of each organization: patient care (access, knowledge, education), research support (fundraising to support research, new therapies and ultimately a cure) and political action (legislation and public policy supporting the Parkinson’s community).

The merged organization will have two groups of advisors, the Unified Parkinson’s Advocacy Council, which will maintain a single voice for advocacy, and a Public Policy Council, which will provide guidance and advice on policy strategies that are important to the Parkinson’s community. While MJFF will continue to be based in NYC, for the first time they will also have an office in Washington, DC. You can read more about the merge here.

PAN and CIRM Have Common Roots

As it turns out there is a strong connection between PAN and CIRM. California attorney and Parkinson’s patient Joan Samuelson founded PAN in 1991. Samuelson described her “surge of excitement” in 1990 when she read about a possible breakthrough in Parkinson’s therapies that used tissue transplants, and how that hope was “swept away” when she read about a federal policy banning any federal support for such research.

PAN was Samuelson’s brainchild and reflected her prescient vision that it would take a political effort at the national level to counter this anti-tissue transplant movement. PAN became famous for its national network of grassroots organizers who could muster thousands of calls to Congress with a day’s notice to voice support or opposition to legislation.

It should come as no surprise that Samuelson was a supporter of California’s Proposition 71 that voters approved in 2004, creating CIRM. Samuelson became CIRM’s first Parkinson Patient Advocate and served in that role until 2014. She was a famously outspoken member of the ICOC (CIRM’s governing Board) and supporter of the Parkinson’s community.

I am humbled to be the one to follow her as the Parkinson’s Patient Advocate on the ICOC, but have no illusions that Samuelson didn’t just leave “big” shoes behind to fill, she left behind shoes so big a family of four could live in them! Samuelson lives in retirement in Sonoma County, CA. I met her once, but never knew her other than by reputation. Words I have heard used to describe her include character, integrity, brilliance, tenacity and one who conveys a sense of urgency for making the world a better place for Parkinson’s patients.

Today, there is not yet a cure, but there is plenty of hope.

 

Notes on Parkinson’s disease

Parkinson’s disease (PD) is a progressive disease that affects the nervous system and for which there is no cure. Over a million people in the US are known to have this debilitating disease. PD is a type of movement disorder, characterized by so-called motor symptoms including too little, too much, or inappropriate movements of the body. Motor symptoms are attributed to the death of specialized cells in a part of the brain that controls movement. These cells produce dopamine, a chemical that allows these specialized nerve cells to “talk” to each other and thus direct movement. What causes these cells to die is an active area of research, including CIRM-funded research. Our understanding of PD has gotten more sophisticated and we now understand that in addition to motor symptoms there are so-called non-motor symptoms. Non-motor symptoms include severe fatigue, insomnia, depression, anxiety, mild cognitive impairment and dementia among others. PD patients often report that non-motor symptoms impact the quality of their lives more than motor symptoms.

Patient Advocates find their voice in a different language

/ Kevin McCormack / Leave a comment

Japan conference

Packed house for stem cell conference in Tokyo – Adrienne Shapiro front row, second from right

One of the many wonderful things about travel is that it opens up your eyes and mind to the fact that, while there are many ways in which people around the world differ from each other, there are also many ways we are all essentially the same.

I was in Japan last week attending the Symposium of Human Embryonic Stem Cell Therapy. The organizers wanted to do something that hadn’t really been done in Japan before, namely engaging Patient Advocates in supporting and advancing stem cell research. They wanted the researchers at the conference to better understand how to connect with patient communities, and the benefits those connections can produce.

Adrienne’s story

To help explain the role of the Patient Advocate they invited me, to talk about our experience at CIRM, and Adrienne Shapiro, from Los Angeles, to come and talk about her experience as a champion of stem cell research for sickle cell disease. Because sickle cell disease affects less than 100,000 people in the US it is classified as a rare disease here. But the numbers affected in Japan are much, much lower so it is considered a really rare disease there. Yet none of that mattered. When Adrienne told her story, the numbers and differences melted away, and what was left was our shared humanity.

Adrienne told the audience that no one chooses to be a Patient Advocate, that it is a role thrust on you by life, by a threat to your health or the health of someone you love. Adrienne explained that she is the fourth generation of women in her family to have a child with sickle cell disease and that she hadn’t been concerned she might pass the trait on to her daughter because a test had shown that her husband didn’t have the genetic mutation that causes sickle cell (to develop the disease an individual has to inherit the genetic mutation from both parents).

But the test was wrong. At nine months Adrienne’s daughter was diagnosed as having sickle cell disease. That’s when Adrienne started fighting. Her first act was to get hospitals to start using a more expensive, but more accurate test to detect if someone carries the genetic trait. She didn’t want anyone else to have their life shaken by a false test result. She won that fight, and hasn’t stopped fighting since.

Japan brochure

Conference brochure

Working together

Adrienne told the audience that patients and researchers need to be partners, because they have shared goals. They both want to see a new treatment, even a cure, for a wide range of deadly diseases. They both want adequate funding for the research. They both want to see the research advance as rapidly as possible.

She explained that patients are not just the recipients of treatments developed in the lab, that they are also people whose lives have been profoundly changed by disease, so they are willing to do everything they can to help the researchers trying to find treatments for their problem.

She talked about Axis Advocacy, the grass-roots organization she helped co-found, and how groups like this can help researchers by educating and raising awareness among the general public about the importance of stem cell research and the need to support it. She talked about the ability of Patient Advocates to do fund raising, or political lobbying, or helping the research team design a patient-friendly clinical trial – one more likely to succeed in recruiting and retaining the patients the trial needs to produce meaningful results, something that is often a real challenge with a rare disease where there are limited numbers of patients to start with.

 

Japan interview

Adrienne and I being interviewed by a reporter with Japan’s Nikkei News

Preaching the power of the Patient Advocates

I talked to the audience of 500 – a full house to the delight of the organizers – about the role of Patient Advocates at CIRM. I explained how Patient Advocates were instrumental in passing Proposition 71, creating the stem cell institute, and now help shape everything we do from the policies we adopt to the projects we fund and even the way we help researchers design patient-friendly clinical trials. I also talked about our work with Patient Advocates to help us speed up the way the FDA works, to make it easier and faster, but no less safe, to get the most promising stem cell therapies to those in need.

But it was Adrienne’s talk about her personal experience that really captivated the audience. The Japanese researchers seemed genuinely interested in learning more about the power of Patient Advocates to help them in their work. For some in the audience this may have been the first time they had heard from a Patient Advocate, the first time they had considered the advantages in partnering with them.

If Adrienne has anything to do with it, it won’t be the last.

Speaking of the power of the Patient Advocate’s voice, Axis Advocacy just launched its new podcast, appropriately enough it’s called The Power of Voices.

CIRM Alpha Stem Cell Clinics: Paving a Path to Cures

/ Karen Ring / 1 Comment

Our mission at CIRM is to accelerate stem cell treatments to patients with unmet medical needs. Over the past ten years, our agency has been tasked with carefully distributing $3 billion dollars of California state tax payer money to the best and brightest scientists in California (and outside too, providing they meet certain requirements). These pioneers are pushing the pace of stem cell research and paving the way for the development of new treatments for patients who desperately need them.

CIRM_Logo_AlphaClinic_300pxAnother way that CIRM is accelerating stem cell treatments is through the CIRM Alpha Stem Cell Clinics Network – a $24 million initiative approved in 2013 to develop the infrastructure to conduct high quality stem cell clinical trials at leading medical centers in California.

There are three clinical teams in the Alpha Clinics Network located at the City of Hope, UC San Diego, and a joint clinic between UC Los Angeles and UC Irvine. These “Regenerative Medicine” clinics were given the name “Alpha” because they are the first of their kind. They bring together the research, clinical and regulatory sides of clinical trials under one roof to streamline the process of getting stem cell therapies to patients.

Currently, over 20 trials are being conducted out of our three Alpha clinics in disease areas including cancer, severe combined immunodeficiency (SCID, also called bubble baby disease), spinal cord injury, diabetes, heart disease, HIV, and diseases of blindness. The clinics plan to expand their pipelines to include other disease areas.

CIRM Alpha Clinics Updates

With any CIRM initiative, it’s always important to update the public on our progress. Below are a few key updates regarding the clinics in the past year:

New UC San Diego Alpha Clinic Video

Recently, the UC San Diego Alpha clinic released a new video that gives an overview of their clinic and describes their commitment to putting patients first. It’s an eye opening video that is definitely worth checking out.

My favorite quote in the video was by Thomas Kipps, a Clinical Trial Expert in Cancer and Deputy Director of Research at the UC San Diego Moores Cancer Center.

“Clinical trials involve a very important skill set. You have to first and foremost put the patient first in any clinical trial. I think we cannot ignore the fact that these are human beings that are brave souls that have gone forward. These are the heroes who are going out and forging new territory.”

 

Alpha Clinics Network Symposium, March 17th 2016

The Alpha Clinics are hosting an annual network symposium this week featuring keynote speakers who’ve made important contributions in stem cell research and regenerative medicine. The day will include basic science presentations, talks on translational research and clinical implementations, and a panel discussion. The symposium will also feature discussions about patient advocacy and how patients and scientists can partner together to improve clinical trials for patients.

If you are interested in learning more about the Alpha Clinics symposium or would like to register for the event, please check out the following links:

Alpha clinics in the news

The Alpha Clinics have been busy this past year! For the latest news on each of our three clinics, check out our Alpha Clinics in the News page.

Related Links:

The Critical Role of Patient Advocates in Accelerating Stem Cell Cures

/ Karen Ring / 1 Comment

At CIRM, our goal is to bring stem cell therapies to patients with unmet medical needs, and we do that by funding the most promising and innovative research in regenerative medicine. A critical component of this goal is to support our patient advocates and make sure that their voices are heard.

At this year’s World Stem Cell Summit, patient advocates from around the world, representing a breadth of diseases and disorders, came together to share their stories, goals, and needs with the larger scientific community.

One session that particularly stood out, was “Accelerating Cures: The Critical Role of Patient Advocates” on Day 3 of the conference. This panel featured key leaders in patient advocacy:

  • Don Reed, the “Grandfather of Stem Cell Research Advocacy”, Vice President of Public Policy at the Americans for Cures Foundation
  • Frances Saldaña, an advocate for Huntington’s disease (HD) and founder of HD-Care at UC Irvine, which is a support group to advance HD research and clinical care
  • Tory Williams, the Executive Director of the Alabama Institute of Medicine (AIM) which raises funds and awareness for stem cell treatments and cures of disease and injury and the author of “Inevitable Collision“

The panel was moderated by our fearless leader and head of communications, Kevin McCormack. Each speaker shared their story about how they became a patient advocate and what they are currently doing to push the pace of stem cell research.

Don Reed, Kevin McCormack, Frances Saldana, Tory Williams.

Don Reed, Kevin McCormack, Frances Saldana, Tory Williams.

Don Reed described the heartbreaking story of his son Roman Reed, who suffered a severe spinal cord injury while playing football. Through Don and Roman’s relentless efforts, “Roman’s Law” was passed in 1999, which raised $17 million in California state funding for spinal cord injury research. Don was also a key instigator for the passage of Proposition 71, which gave $3 billion dollars to our agency to fund stem cell research. He continues to be a passionate advocate for stem cell research and spinal cord injury patients, and recently published a book called “Stem Cell Battles: Proposition 71 and Beyond” which you can read more about in our recent blog.

Next, Frances Saldana told a compelling story of raising a family of three beautiful children with a husband who had Huntington’s disease. Unaware of his condition when they were together, Frances’ world took a devastating turn when he died of HD, leaving her to question whether her children would face the same fate. Sadly, all three of Frances’s kids carried the HD mutation. Having to deal with the passing of her two daughters, and a son who is battling the end stages of this disease, Frances decided to share her experience with others and to create a support organization called HD-Care so that others wouldn’t have to face similar experiences alone. HD-Care is conducting an aggressive campaign to bring visibility to HD and supports cutting-edge research in the field including the work done by CIRM-grantee Dr. Leslie Thompson at UC Irvine.

Frances told the audience that her happiest moment since this all began was when her daughter Margie, already suffering from symptoms of HD, spoke at CIRM in 2007. She saw the Board and the scientists and thought, “somebody cares, and somebody will find a cure.” It was a new chapter for her, she explained, and she knew something good was going to happen.

Lastly, Tory Williams, introduced the Alabama Institute of Medicine, which is a non-profit organization that supports the stem cell community with education and public dialogue. She started the institute following both personal and family experiences with cancer and after TJ Atchinson, a close family friend, suffered a severe spinal cord injury. Along the way, she forged a close relationship with Roman Reed who helped her pass TJ’s law in 2013, which is an Alabama state law that promotes spinal cord injury research.

“The goal [of AIM],” said Williams, “is to make a difference in people’s lives affected by disease and injury by helping to advance medicine to eradicate these debilitating issues.”

Laurel Barchas, Student Society for Stem Cell Research

Laurel Barchas, Student Society for Stem Cell Research

When the session was opened up to questions, the atmosphere in the room turned electric. Patients and scientists stood up to tell their stories and asked hard questions. One question came from Laurel Barchas, one of the founders of the Student Society for Stem Cell Research, who asked how we as a society can advocate for mental illness and similar diseases where the symptoms are not visible and where patients are either embarrassed or hesitant to make their disease public. Another question was how emerging countries like Mexico who don’t have the same benefits and infrastructure as the US can promote and support patient advocacy.

The mood of the advocates was positive but measured. They know that new treatments and cures take time but they also pointed out that many people don’t have much time so we have to work as hard as we can to help them.

The panel ended with the consensus that the voices of patient advocates are invaluable, and that they will be the key to accelerating stem cell therapies into cures. Frances Saldaña urged other patient advocates that the key to progress is to be aggressive, and be unafraid to be out there. Don Reed concluded on a similar note with quote from Shakespeare’s Hamlet:

“Whether ’tis Nobler in the mind to suffer

The Slings and Arrows of outrageous Fortune,

Or to take Arms against a Sea of troubles,

And by opposing end them.”

Related links: