Stem Cell Agency’s Diane Winokur hailed as Visionary

Diane and JT

CIRM Board member Diane Winokur with CIRM Board Chair Jonathan Thomas at FFB Awards dinner

Generally speaking, I am not a huge fan of gala dinners. It’s not that I don’t like seeing people who do remarkable things getting a well-deserved honor. It’s just that the dinners often go on too long and the food is usually not very good (hey, this is San Francisco, those things matter). But last night’s Foundation Fighting Blindness Visionary Awards in San Francisco was definitely an exception to that rule.

Academy of Sciences Grand Opening

Academy of Sciences in San Francisco

Now it may be that the awards were held in the spectacular Academy of Sciences building in Golden Gate Park, or that the food was delicious. But I think the real reason is that CIRM Board member Diane Winokur was one of those being honored. The other honoree was Dr. Jacque Duncan, an amazing physician at UC San Francisco who has dedicated her life to battling diseases of the retina. The whole event was deeply emotional, and truly inspiring.

Now, Diane is a remarkable woman in many respects. She’s the Board’s Patient Advocate member for ALS (better known as Lou Gehrig’s disease) and multiple sclerosis. But Diane also considers herself a Patient Advocate for all Californians and works hard to help advance the research that could help them. She has a personal connection to vision loss as well; one of her dear friends has lost his sight because of retinitis pigmentosa, and his daughter is losing hers because of the same disease.

Diane at podiumDiane highlighted the work that CIRM is doing to help battle vision destroying diseases; how we have invested more than $125 million in 25 different projects. She talked about the encouraging news from clinical trials we are funding targeting retinitis pigmentosa and dry age-related macular degeneration. Diane said:

“These stem cell clinical trials show that progress is being made. Not as fast as we would like, but as everyone here knows, good science takes time. As a patient advocate on the CIRM Board it’s my role to represent the patient, to be their voice in making decisions about what projects to fund.

Patients are at the heart of everything we do at CIRM, from deciding on funding issues to supporting clinical trials. That’s why I feel so honored to get this award. It comes from an organization, that is equally committed to doing all it can to help people in need, to putting the patient at the center of everything they do.”

It’s clear that patients really are at the heart of the work the Foundation Fighting Blindness (FFB) does. As the organizations CEO Benjamin Yerxa said:

“We support 77 labs in the US, often funding projects no one else would. We do this because we know it is necessary to advance the field. And we are going to keep doing this as best we can, as fast as we can, for as long as we can, because we know so many people are depending on us to help them.”

The other honoree, Jacque Duncan, said after attending many previous Visionary Award dinners and seeing the people being honored it was humbling to be in that company. She talked about the exciting progress being made in the field and the people who are making it possible.

“None of this happens by chance. The path to developing new treatments takes the passion of scientists and doctors, and the commitment of patients to raising the funds needed to do this research. One gala dinner at a time, one Vision Walk at a time. All of this creates community and a common purpose. I truly believe that because of this, tomorrow will be brighter than today.”

Perhaps it’s only appropriate to leave the last word to Diane, who ended her speech saying:

“The Nobel prize winning physicist Heinrich Rohrer once said that science means constantly walking a tightrope between blind faith and curiosity; between expertise and creativity; between bias and openness; between experience and epiphany; in short, between an old today and a new tomorrow.

I believe that working together, CIRM and the Foundation Fighting Blindness, we can create that new tomorrow.”

jCyte Shares Encouraging Update on Clinical Trial for Retinitis Pigmentosa

Stepping out of the darkness into light. That’s how patients are describing their experience after participating in a CIRM-funded clinical trial targeting a rare form of vision loss called retinitis pigmentosa (RP). jCyte, the company conducting the trial, announced 12 month results for its candidate stem cell-based treatment for RP.

RP is a genetic disorder that affects approximately 1 in 40,000 individuals and 1.5 million people globally. It causes the destruction of the light-sensing cells at the back of the eye called photoreceptors. Patients experience symptoms of vision loss starting in their teenage years and eventually become legally blind by middle age. While there is no cure for RP, there is hope that stem cell-based therapies could slow its progression in patients.

Photoreceptors look healthy in a normal retina (left). Cells are damaged in the retina of an RP patient (right). (Source National Eye Institute)

jCyte is one of the leaders in developing cell-based therapies for RP. The company, which was founded by UC Irvine scientists led by Dr. Henry Klassen, is testing a product called jCell, which is composed of pluripotent stem cell-derived progenitor cells that develop into photoreceptors. When transplanted into the back of the eye, they are believed to release growth factors that prevent further damage to the surviving cells in the retina. They also can integrate into the patient’s retina and develop into new photoreceptor cells to improve a patient’s vision.

Positive Results

At the Annual Ophthalmology Innovation Summit in November, jCyte announced results from its Phase 1/2a trial, which was a 12-month study testing two different doses of transplanted cells in 28 patients. The company reported a “favorable safety profile and indications of potential benefit” to patient vision.

The patients received a single injection of cells in their worst eye and their visual acuity (how well they can see) was then compared between the treated and untreated eye. Patients who received the lower dose of 0.5 million cells were able to see one extra letter on an eye chart with their treated eye compared to their untreated eye while patients that received the larger dose of 3 million cells were able to read 9 more letters. Importantly, none of the patients experienced any significant side effects from the treatment.

According to the company’s news release, “patient feedback was particularly encouraging. Many reported improved vision, including increased sensitivity to light, improved color discrimination and reading ability and better mobility. In addition, 22 of the 28 patients have been treated in their other eye as part of a follow-on extension study.”

One of these patients is Rosie Barrero. She spoke to us earlier this year about how the jCyte trial has not only improved her vision but has also given her hope. You can watch her video below.

Next Steps

These results suggest that the jCell therapy is safe (at least at the one year mark) to use in patients and that larger doses of jCell are more effective at improving vision in patients. jCyte CEO, Paul Bresge commented on the trial’s positive results:

Paul Bresge

“We are very encouraged by these results. Currently, there are no effective therapies to offer patients with RP. We are moving forward as quickly as possible to remedy that. The feedback we’ve received from trial participants has been remarkable. We look forward to moving through the regulatory process and bringing this easily-administered potential therapy to patients worldwide.”

Bresge and his company will be able to navigate jCell through the regulatory process more smoothly with the product’s recent Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA). The FDA grants RMAT to regenerative medicine therapies for serious diseases that have shown promise in early-stage clinical trials. The designation allows therapies to receive expedited review as they navigate their way towards commercialization.

jCyte is now evaluating the safety and efficacy of jCell in a Phase2b trial in a larger group of up to 85 patients. CIRM is also funding this trial and you can read more about it on our website.


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A Patient Advocate’s Personal Manifesto

Janni and Obama

President Obama and Janni Lehrer-Stein

Janni Lehrer-Stein was just 26 when she was diagnosed with a degenerative eye disease and told she was going to be blind within six months. The doctor who gave her the news told her “But don’t worry, people like you are usually hit and killed by a bus long before they go completely blind.”

At the time she was recently married, had just graduated law school and landed her dream job with the government in Washington DC, litigating workplace discrimination. The news about her eyesight stopped her in her tracks.

But not for long. If you ever met Janni you would know that nothing stops her for long.

I was fortunate enough to hear Janni talk at a Foundation Fighting Blindness event in the San Francisco Bay Area last weekend. I was part of a panel discussion on new approaches to treating vision loss, including the research that CIRM is funding.

Janni didn’t talk about stem cells, instead she focused on the importance of the patient advocate voice, community, and their determination. She said one of the most important things anyone battling a life-threatening or life-changing disease or disorder needs to remember is that it’s not about disability, it’s about capability. It’s about what you can do rather than what you cannot.

Janni laid out her “manifesto” for things she says will help you keep that thought uppermost in your mind.

1) Show up. It’s that simple and that important. You have to show up. You have to get educated, you have to learn all you can about your condition so you know what you can do and what you can’t do. You have to share that information with others. You have to be there for others. Don’t just show up for yourself. Show up for others who can’t be there.

2) Share this information. Janni talked about a website called My Retina Tracker which is helping drive research into the causes of retinal diseases like retinitis pigmentosa and macular degeneration, and hopefully will lead to treatments and even cures. She says the more people work together, the more we combine our resources, the more effective we can be.

3) Support the researchers. Janni says while raising awareness is important, raising money is just as important. Without money there can be no research, and without research no treatments or cures. Janni says it doesn’t matter how you do it – a charity walk, a Go Fund me campaign, petitioning your state or federal elected representatives to urge them to fund research – everything counts, every dollar helps.

4) Remember you are part of a wider community. Janni says no one ever won a battle on their own; it takes a lot of people to fight and win the right to be treated equally. And it takes a lot of effort to stop those rights from being rolled back.

Janni hasn’t let losing her sight hold her back. In 2011, she was appointed by President Obama, and confirmed by the U.S. Senate, to the National Council on Disability where she served two terms advising the President and Congress on national disability policy.

Now she has returned home to the San Francisco Bay Area, but she is no less determined to make a difference and no less determined to fight for the rights of patients and patient advocates.

In an article on Medium she shares her feelings about being a patient advocate:

“The America that I so deeply respect is one that embraces, values and respects the contributions of us all. My America includes every one of us, regardless of our gender, race, age or disability. Our America is a place where, regardless of whether we are sighted or blind, we have the same opportunities, for which we are equally considered. Our America includes every one of us who wishes to make the world a more peaceful, responsible, and inclusive environment that is tolerant of all differences and abilities, physical or otherwise. To me, those differences make our lives richer, give our contributions more meaning, and lead to a brighter future for the next generation.”

 

CIRM-Funded Clinical Trials Targeting Brain and Eye Disorders

This blog is part of our Month of CIRM series, which features our Agency’s progress towards achieving our mission to accelerate stem cell treatments to patients with unmet medical needs.

 This week, we’re highlighting CIRM-funded clinical trials to address the growing interest in our rapidly expanding clinical portfolio. Our Agency has funded a total of 40 trials since its inception. 23 of these trials were funded after the launch of our Strategic Plan in 2016, bringing us close to the half way point of our goal to fund 50 new clinical trials by 2020.

Today we are featuring CIRM-funded trials in our neurological and eye disorders portfolio.  CIRM has funded a total of nine trials targeting these disease areas, and seven of these trials are currently active. Check out the infographic below for a list of our currently active trials.

For more details about all CIRM-funded clinical trials, visit our clinical trials page and read our clinical trials brochure which provides brief overviews of each trial.

Throwback Thursday: Progress to a Cure for Diseases of Blindness

Welcome back to our “Throwback Thursday” series on the Stem Cellar. Over the years, we’ve accumulated an arsenal of exciting stem cell stories about advances towards stem cell-based cures for serious diseases. This month we’re featuring stories about CIRM-funded clinical trials for blindness.

2017 has been an exciting year for two CIRM-funded clinical trials that are testing stem cell-based therapies for diseases of blindness. A company called Regenerative Patch Technologies (RPT) is transplanting a sheet of embryonic stem cell-derived retinal support cells into patients with the dry form of age-related macular degeneration, a disease that degrades the eye’s macula, the center of the retina that controls central vision. The other trial, sponsored by a company called jCyte, is using human retinal progenitor cells to treat retinitis pigmentosa, a rare genetic disease that destroys the light-sensing cells in the retina, causing tunnel vision and eventually blindness.

 

Both trials are in the early stages, testing the safety of their respective stem cell therapies. But the teams are hopeful that these treatments will stop the progression of or even restore some form of vision in patients. In the past few months, both RPT and jCyte have shared exciting news about the progress of these trials which are detailed below.

Macular Degeneration Trial Gets a New Investor

In April, RPT announced that they have a new funding partner to further develop their stem cell therapy for age-related macular degeneration (AMD). They are partnering with Japan’s Santen Pharmaceutical Company, which specializes in developing ophthalmology or eye therapies.

AMD is the leading cause of blindness in elderly people and is projected to affect almost 200 million people worldwide by 2020. There is no cure or treatment that can restore vision in AMD patients, but stem cell transplants offer a potential therapeutic option.

RPT believes that their newfound partnership with Santen will accelerate the development of their stem cell therapy and ultimately fulfill an unmet medical need. RPT’s co-founder, Dr. Dennis Clegg, commented in a CIRM news release, “the ability to partner with a global leader in ophthalmology like Santen is very exciting. Such a strong partnership will greatly accelerate RPT’s ability to develop our product safely and effectively.”

This promising relationship highlights CIRM’s efforts to partner our clinical programs with outside investors to boost their chance of success. It also shows confidence in the future success of RPT’s stem cell-based therapy for AMD.

Retinitis Pigmentosa Trial Advances to Phase 2 and Receives RMAT Status

In May, the US Food and Drug Administration (FDA) approved jCyte’s RP trial for Regenerative Medicine Advanced Therapy (RMAT) status, which could pave the way for accelerated approval of this stem cell therapy for patients with RP.

RMAT is a new status established under the 21st Century Cures Act – a law enacted by Congress in December of 2016 to address the need for a more efficient regulatory approval process for stem cell therapies that can treat serious or life-threatening diseases. Trial sponsors of RMAT designated therapies can meet with the FDA earlier in the trial process and are eligible for priority review and accelerated approval.

jCyte’s RMAT status is well deserved. Their Phase 1 trial was successful, proving the treatment was safe and well-tolerated in patients. More importantly, some of the patients revealed that their sight has improved following their stem cell transplant. We’ve shared the inspiring stories of two patients, Rosie Barrero and Kristin Macdonald, previously on the Stem Cellar.

Rosie Barrero

Kristin MacDonald

Both Rosie and Kristin were enrolled in the Phase 1 trial and received an injection of retinal progenitor cells in a single eye. Rosie said that she went from complete darkness to being able to see shapes, colors, and the faces of her family and friends. Kristin was the first patient treated in jCyte’s trial, and she said she is now more sensitive to light and can see shapes well enough to put on her own makeup.

Encouraged by these positive results, jCyte launched its Phase 2 trial in April with funding from CIRM. They will test the same stem cell therapy in a larger group of 70 patients and monitor their progress over the next year.

Progress to a Cure for Blindness

We know very well that scientific progress takes time, and unfortunately we don’t know when there will be a cure for blindness. However, with the advances that these two CIRM-funded trials have made in the past year, our confidence that these stem cell treatments will one day benefit patients with RP and AMD is growing.

I’ll leave you with an inspiring video of Rosie Barrero about her experience with RP and how participating in jCytes trial has changed her life. Her story is an important reminder of why CIRM exists and why supporting stem cell research in particular, and research in general, is vital for the future health of patients.


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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.

klassen

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.

 

 

jCyte starts second phase of stem cell clinical trial targeting vision loss

retinitis pigmentosas_1

How retinitis pigmentosa destroys vision

Studies show that Americans fear losing their vision more than any other sense, such as hearing or speech, and almost as much as they fear cancer, Alzheimer’s and HIV/AIDS. That’s not too surprising. Our eyes are our connection to the world around us. Sever that connection, and the world is a very different place.

For people with retinitis pigmentosa (RP), the leading cause of inherited blindness in the world, that connection is slowly destroyed over many years. The disease eats away at the cells in the eye that sense light, so the world of people with RP steadily becomes darker and darker, until the light goes out completely. It often strikes people in their teens, and many are blind by the time they are 40.

There are no treatments. No cures. At least not yet. But now there is a glimmer of hope as a new clinical trial using stem cells – and funded by CIRM – gets underway.

klassenWe have talked about this project before. It’s run by UC Irvine’s Dr. Henry Klassen and his team at jCyte. In the first phase of their clinical trial they tested their treatment on a small group of patients with RP, to try and ensure that their approach was safe. It was. But it was a lot more than that. For people like Rosie Barrero, the treatment seems to have helped restore some of their vision. You can hear Rosie talk about that in our recent video.

Now the same treatment that helped Rosie, is going to be tested in a much larger group of people, as jCyte starts recruiting 70 patients for this new study.

In a news release announcing the start of the Phase 2 trial, Henry Klassen said this was an exciting moment:

“We are encouraged by the therapy’s excellent safety track record in early trials and hope to build on those results. Right now, there are no effective treatments for retinitis pigmentosa. People must find ways to adapt to their vision loss. With CIRM’s support, we hope to change that.”

The treatment involves using retinal progenitor cells, the kind destroyed by the disease. These are injected into the back of the eye where they release factors which the researchers hope will help rescue some of the diseased cells and regenerate some replacement ones.

Paul Bresge, CEO of jCyte, says one of the lovely things about this approach, is its simplicity:

“Because no surgery is required, the therapy can be easily administered. The entire procedure takes minutes.”

Not everyone will get the retinal progenitor cells, at least not to begin with. One group of patients will get an injection of the cells into their worst-sighted eye. The other group will get a sham injection with no cells. This will allow researchers to compare the two groups and determine if any improvements in vision are due to the treatment or a placebo effect.

The good news is that after one year of follow-up, the group that got the sham injection will also be able to get an injection of the real cells, so that if the therapy is effective they too may be able to benefit from it.

Rosie BarreroWhen we talked to Rosie Barrero about the impact the treatment had on her, she said it was like watching the world slowly come into focus after years of not being able to see anything.

“My dream was to see my kids. I always saw them with my heart, but now I can see them with my eyes. Seeing their faces, it’s truly a miracle.”

We are hoping this Phase 2 clinical trial gives others a chance to experience similar miracles.


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A stem cell clinical trial for blindness: watch Rosie’s story

Everything we do at CIRM is laser-focused on our mission: to accelerate stem cell treatments for patients with unmet medical needs. So, you might imagine what a thrill it is to meet the people who could be helped by the stem cell research we fund. People like Rosie Barrero who suffers from Retinitis Pigmentosa (RP), an inherited, incurable form of blindness, which she describes as “an impressionist painting in a foggy room”.

The CIRM team first met Rosie Barrero back in 2012 at one of our governing Board meetings. She and her husband, German, attended the meeting to advocate for a research grant application submitted by UC Irvine’s Henry Klassen. The research project aimed to bring a stem cell-based therapy for RP to clinical trials. The Board approved the project giving a glimmer of hope to Rosie and many others stricken with RP.

Now, that hope has become a reality in the form of a Food and Drug Administration (FDA)-approved clinical trial which Rosie participated in last year. Sponsored by jCyte, a company Klassen founded, the CIRM-funded trial is testing the safety and effectiveness of a non-surgical treatment for RP that involves injecting stem cells into the eye to help save or even restore the light-sensing cells in the back of the eye. The small trial has shown no negative side effects and a larger, follow-up trial, also funded by CIRM, is now recruiting patients.

Almost five years after her first visit, Rosie returned to the governing Board in February and sprinkled in some of her witty humor to describe her preliminary yet encouraging results.

“It has made a difference. I’m still afraid of public speaking but early on [before the clinical trial] it was much easier because I couldn’t see any of you. But, hello everybody! I can see you guys. I can see this room. I can see a lot of things.”

After the meeting, she sat down for an interview with the Stem Cellar team to talk about her RP story and her experience as a clinical trial participant. The three-minute video above is based on that interview. Watch it and be inspired!

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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Cured by Stem Cells

cirm-2016-annual-report-web-12

To get anywhere you need a good map, and you need to check it constantly to make sure you are still on the right path and haven’t strayed off course. A year ago the CIRM Board gave us a map, a Strategic Plan, that laid out our course for the next five years. Our Annual Report for 2016, now online, is our way of checking that we are still on the right path.

I think, without wishing to boast, that it’s safe to say not only are we on target, but we might even be a little bit ahead of schedule.

The Annual Report is chock full of facts and figures but at the heart of it are the stories of the people who are the focus of all that we do, the patients. We profile six patients and one patient advocate, each of whom has an extraordinary story to tell, and each of whom exemplifies the importance of the work we support.

brenden_stories_of_hope

Brenden Whittaker: Cured

Two stand out for one simple reason, they were both cured of life-threatening conditions. Now, cured is not a word we use lightly. The stem cell field has been rife with hyperbole over the years so we are always very cautious in the way we talk about the impact of treatments. But in these two cases there is no need to hold back: Evangelina Padilla Vaccaro and Brenden Whittaker have been cured.

evangelina

Evangelina: Cured

 

In the coming weeks we’ll feature our conversations with all those profiled in the Annual Report, giving you a better idea of the impact the stem cell treatments have had on their lives and the lives of their family. But today we just wanted to give a broad overview of the Annual Report.

The Strategic Plan was very specific in the goals it laid out for us. As an agency we had six big goals, but each Team within the agency, and each individual within those teams had their own goals. They were our own mini-maps if you like, to help us keep track of where we were individually, knowing that every time an individual met a goal they helped the Team get closer to meeting its goals.

As you read through the report you’ll see we did a pretty good job of meeting our targets. In fact, we missed only one and we’re hoping to make up for that early in 2017.

But good as 2016 was, we know that to truly fulfill our mission of accelerating treatments to patients with unmet medical needs we are going to have do equally well, if not even better, in 2017.

That work starts today.