Treatments, cures and clinical trials: an in-person update on CIRM’s progress

Patients and Patient Advocates are at the heart of everything we do at CIRM. That’s why we are holding three free public events in the next few months focused on updating you on the stem cell research we are funding, and our plans for the future.

Right now we have 33 projects that we have funded in clinical trials. Those range from heart disease and stroke, to cancer, diabetes, ALS (Lou Gehrig’s disease), two different forms of vision loss, spinal cord injury and HIV/AIDS. We have also helped cure dozens of children battling deadly immune disorders. But as far as we are concerned we are only just getting started.

Over the course of the next few years, we have a goal of adding dozens more clinical trials to that list, and creating a pipeline of promising therapies for a wide range of diseases and disorders.

That’s why we are holding these free public events – something we try and do every year. We want to let you know what we are doing, what we are funding, how that research is progressing, and to get your thoughts on how we can improve, what else we can do to help meet the needs of the Patient Advocate community. Your voice is important in helping shape everything we do.

The first event is at the Gladstone Institutes in San Francisco on Wednesday, September 6th from noon till 1pm. The doors open at 11am for registration and a light lunch.

Gladstone Institutes

Here’s a link to an Eventbrite page that has all the information about the event, including how you can RSVP to let us know you are coming.

We are fortunate to be joined by two great scientists, and speakers – as well as being CIRM grantees-  from the Gladstone Institutes, Dr. Deepak Srivastava and Dr. Steve Finkbeiner.

Dr. Srivastava is working on regenerating heart muscle after it has been damaged. This research could not only help people recover from a heart attack, but the same principles might also enable us to regenerate other organs damaged by disease. Dr. Finkbeiner is a pioneer in diseases of the brain and has done ground breaking work in both Alzheimer’s and Huntington’s disease.

We have two other free public events coming up in October. The first is at UC Davis in Sacramento on October 10th (noon till 1pm) and the second at Cedars-Sinai in Los Angeles on October 30th (noon till 1pm). We will have more details on these events in the coming weeks.

We look forward to seeing you at one of these events and please feel free to share this information with anyone you think might be interested in attending.

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:

Why Stem Cell Advocates Texans for Cures say “Right to Try” Legislation Should be Fought

Texans for Cures 

This week in Washington DC a delegation from the stem cell advocacy group Texans for Cures is meeting with members of Congress from both parties. The focus of the meetings are three bills promoting “Right to Try” legislation. Supporters of the bills say they will empower patients battling terminal illness. Texans for Cures say, quite the contrary, that these laws will endanger patients. In this guest blog, Texans for Cures explain why they feel these laws are bad.

In 2014, the Goldwater Institute published a policy report titled, “Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of their Treatment.”[i] The report calls for states to pass “Right to Try” legislation as a means to reclaim patients’ medical autonomy and right to determine their own medical treatment.

This policy recommendation is built on the theory that the Food and Drug Administration (FDA) should not be able to restrict terminal patients’ access to potentially life-saving treatments so long as the treatment has been tested for basic safety. While this idea may seem immediately appealing, the policy undermines medical research in several ways that are harmful to the development of new treatments.

Texans for Cures opposes this legislation because it harms the sound development of treatments for future patients on the mere chance that it may provide relief to current patients that have received a terminal diagnosis. In short, Right to Try policies ignore the attendant risks and overemphasize the potential benefits.

draft_bill_legislation_law

“Right to Try” Model Legislation and State Enacted Variants

The Goldwater Institute’s policy report included model legislation for interested legislators, which it summed up as follows:

Simply stated, Right to Try allows a patient to access investigational medications that have passed basic safety tests without interference by the government when the following conditions are met:

  1. The patient has been diagnosed with a terminal disease;
  2. The patient has considered all available treatment options;
  3. The patient’s doctor has recommended that the investigational drug, device, or biological product represents the patient’s best chance at survival;
  4. The patient or the patient’s guardian has provided informed consent; and
  5. The sponsoring company chooses to make the investigational drug available to patients outside the clinical trial.

Since the Goldwater Institute published this policy report in 2014, 33 states have enacted Right to Try laws.[ii] These laws contain minor variations from the model legislation, but each operates similarly to limit the FDA’s oversight roll.

Right to Try is Loosely Grounded in the Constitution and May Require Federal Action

Due to the fact that these laws may infringe on the FDA’s authority over drug development and distribution, the policy report attempts to ground Right to Try in one’s constitutional right to liberty. This constitutional underpinning is loose and is not firmly supported by Supreme Court precedent.[iii] With the constitutional basis of Right to Try resting on a weak foundation, it is important for Right to Try proponents to pass a complimentary Right to Try statute on the federal level in Congress.

There are three bills actively working through the Congressional process that would prohibit the FDA or any other federal agency from interfering with a patient’s Right to Try: H.R. 878 by Representative Biggs,[iv] H.R. 2368 by Representative Fitzpatrick,[v] and S. 204 by Senator Johnson.[vi] Each of these bills shares three common provisions, while H.R. 2368 has two additional provisions:

Common Provisions:

  1. Prohibition on federal action
  2. No liability
  3. No use of outcomes

Provisions Unique to H.R. 2368:

  1. Manufacturers are not required to make treatments available
  2. Permits manufacturers to receive compensation or recover costs

All three of the federal bills would remove the FDA’s ability to intervene in state Right to Try programs. They also create a liability shield for any producer, manufacturer, distributor, prescriber, dispenser, possessor, or user participating in the program. And finally, each prohibits the use of outcomes from patients participating in Right to Try as a criteria for FDA review of the treatment. This means that harmed patients would have limited or no legal recourse, and the FDA may need another Act of Congress to grant them the authority to intervene in any programs that prove to be dangerous. However, it may be difficult to know if these programs are harming patients or not, because the bills do not provide any mechanism for tracking outcomes and using that information for oversight.

Each bill is drafted in a way that would remove FDA oversight authority and would allow states to proceed with Right to Try policies and grants states broad discretion to tailor these programs without federal oversight. However, H.R. 2368 contains two additional provisions that would compliment and potentially override state statute. First, the bill gives manufacturers the authority to deny patients access to investigational treatments, which is consistent with the Goldwater Institute’s model legislation. Second, the bill allows manufacturers to receive compensation or recover costs involved in making the drug available to patients. This second provision is particularly problematic in that it would allow manufacturers to charge patients for unproven treatments unless they were explicitly prohibited from doing so by state law.

Single pill

How Right to Try Laws Structurally Harm the Research Process

Right to Try laws create a number of problematic incentives and penalties that would likely harm the long term development of new therapies. First and foremost, under Right to Try, patients will be able to bypass the clinical trial process, request investigational treatments, and pay the cost of the drug, rather than enter into a clinical trial. Given that clinical trials may involve the use of placebos, Texans for Cures is concerned that patients may choose to exercise Right to Try rather than participate in a clinical trial, because under Right to Try the patient avoids the possibility of receiving a placebo.

Additionally, there is no mechanism in the proposed bills for tracking outcomes of patients participating in Right to Try, and there is no mechanism for government intervention if Right to Try proves to be unreasonably risky.

Right to Try seeks to shield all participants from liability, meaning that patients who are harmed will have limited or no legal recourse, even if manufacturers or physicians are negligent. Furthermore, Right to Try laws typically allow manufacturers to recover the cost of manufacturing the treatment for participating patients, but cost is not defined. Does cost include the cost of research and development or is it exclusively the cost of creating that specific treatment? The ambiguity surrounding this term is a cause for concern, because companies may be tempted to use this ambiguity to cover a broader sets of costs than the authors intended.

Conclusion

Texans for Cures opposes this legislative effort because the program could potentially harm patients and, if it does, the law does not provide adequate safeguards or remedies. Additionally, the law does not require any monitoring of outcomes and is therefore unscientific in its approach to treatments that are currently undergoing clinical research.

The FDA is already working to ease the burdens associated with Expanded Access programs, which achieve the end that Right to Try desires: providing access to research drugs for terminal patients. The difference is that Expanded Access has additional safeguards and a mechanism for FDA intervention if treatment is found to be dangerous or harmful to the clinical trial process.

Finding scientifically sound treatments for patients in need is the primary concern of Texans for Cures. Texans for Cures sympathizes with, and its members have similarly experienced, the pain of losing loved ones. The hope and emotion involved in Right to Try laws is not to be taken lightly, but it is precisely because strong emotions can cloud our judgment that we, as a society, must approach the clinical trial process with a clear mental state. Texans for Cures believes that Right to Try will harm the long term development of new treatments and therefore asks for your help in fighting this legislative effort.

Footnotes:

[1] Christina Corieri, “Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of their Treatment,” Goldwater Institute (2014), https://goldwater-media.s3.amazonaws.com/cms_page_media/2015/1/28/Right%20To%20Try.pdf

2 KHN Morning Briefing, “‘Right to Try’ Advocates Help Pass Laws In 33 States As Movement Gains National Foothold,” Kaiser Health News (2017), http://khn.org/morning-breakout/right-to-try-advocates-help-pass-laws-in-33-states-as-movement-gains-national-foothold/

3 The Goldwater Institute’s sole source for constitutional grounding for this law comes from a concurrence by Justice Douglas in Doe v. Bolton, 410 U.S. 179, 218 (1973), where he noted that individuals have a “right to care for one’s health and person.” The Goldwater Institute appears to recognize the precarious footing of their model legislation, stating in their policy report, “Although the right of terminal patients to access investigational medications has not yet been recognized by the Supreme Court, it is consistent with and can be supported by existing precedent.”

[1] H.R. 878 by Representative Biggs, https://www.congress.gov/bill/115th-congress/house-bill/878/text?q=%7B%22search%22%3A%5B%22right+to+try%22%5D%7D&r=2

[1] H.R. 2368 by Representative Fitzpatrick, https://www.congress.gov/bill/115th-congress/house-bill/2368/text?q=%7B%22search%22%3A%5B%22right+to+try%22%5D%7D&r=1

[1] S. 204 by Senator Johnson, https://www.congress.gov/bill/115th-congress/senate-bill/204/text?q=%7B%22search%22%3A%5B%22right+to+try%22%5D%7D&r=3

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.

One man’s journey with leukemia has turned into a quest to make bone marrow stem cell transplants safer

Dr. Lukas Wartman in his lab in March 2011 (left), before he developed chronic graft-versus-host disease, and last month at a physical therapy session (right). (Photo by Whitney Curtis for Science Magazine)

I read a story yesterday in Science Magazine that really stuck with me. It’s about a man who was diagnosed with leukemia and received a life-saving stem cell transplant that is now threatening his health.

The man is name Lukas Wartman and is a doctor at Washington University School of Medicine in St. Louis. He was first diagnosed with a type of blood cancer called acute lymphoblastic leukemia (ALL) in 2003. Since then he has taken over 70 drugs and undergone two rounds of bone marrow stem cell transplants to fight off his cancer.

The first stem cell transplant was from his brother, which replaced Wartman’s diseased bone marrow, containing blood forming stem cells and immune cells, with healthy cells. In combination with immunosuppressive drugs, the transplant worked without any complications. Unfortunately, a few years later the cancer returned. This time, Wartman opted for a second transplant from an unrelated donor.

While the second transplant and cancer-fighting drugs have succeeded in keeping his cancer at bay, Wartman is now suffering from something equally life threatening – a condition called graft vs host disease (GVHD). In a nut shell, the stem cell transplant that cured him of cancer and saved his life is now attacking his body.

GVHD, a common side effect of bone marrow transplants

GVHD is a disease where donor transplanted immune cells, called T cells, expand and attack the cells and tissues in your body because they see them as foreign invaders. GVHD occurs in approximately 50% of patients who receive bone marrow, peripheral blood or cord blood stem cell transplants, and typically affects the skin, eyes, mouth, liver and intestines.

The main reason why GVHD is common following blood stem cell transplants is because many patients receive transplants from unrelated donors or family members who aren’t close genetic matches. Half of patients who receive these types of transplants develop an acute form of GVHD within 100 days of treatment. These patients are put on immunosuppressive steroid drugs with the hope that the patient’s body will eventually kill off the aggressive donor T cells.

This was the case for Wartman after the first transplant from his brother, but the second transplant from an unrelated donor eventually caused him to develop the chronic form of GVHD. Wartman is now suffering from weakened muscles, dry eyes, mouth sores and skin issues as the transplanted immune cells slowly attack his body from within. Thankfully, his major organs are still untouched by GVHD, but Wartman knows it could be only a matter of time before his condition worsens.

Dr. Lukas Wartman has to use eye drops every 20 minutes to deal with dry eyes caused by GVHD. (Photo by Whitney Curtis for Science Magazine)

Hope for GVHD sufferers

Wartman along with other GVHD patients are basically guinea pigs in a field where effective drugs are still being developed and tested. Many of these patients, including Wartman, have tried many unproven treatments or drugs for other disease conditions in desperate hope that something will work. It’s a situation that is heartbreaking not only for the patient but also for their families and doctors.

There is hope for GVHD patients however. Science Magazine mentioned two promising drugs for GVHD, ibrutinib and ruxolitinib. Both received breakthrough therapy designation from the US Food and Drug Administration and could be the first approved treatments for GVHD.

Another promising therapy is called Prochymal. It’s a stem cell therapy developed by former CIRM President and CEO, Dr. Randy Mills, at Osiris Therapeutics. Prochymal is already approved to treat the acute form of GVHD in Canada, and is currently being tested in phase 3 trials in the US in young children and adults.

While CIRM isn’t currently funding clinical trials for GVHD, we are funding a trial out of Stanford University led by Dr. Judy Shizuru that aims to improve the outcome of bone marrow stem cell transplants in patients. Shizuru says that these transplants are “the most powerful form of cell therapy out there, for cancers or deficiencies in blood formation” but they come with their own set of potentially deadly side effects such as GVHD.

Shizuru is testing an antibody drug that blocks a signaling protein called CD117, which sits on the surface of blood stem cells and acts as an elimination signal. By turning off this protein, her team improved the engraftment of bone marrow stem cells in mice that had leukemia and removed their need for chemotherapy treatment. The therapy is in a Phase 1 trial for patients with an immune disease called severe combined immunodeficiency (SCID) who receive bone marrow transplants, but Shizuru said that her hope is the drug could also treat patients with certain cancers or blood diseases.

Advocating for better GVHD treatments

The reason the article in Science Magazine spoke to me is because of the power of Wartman’s story. Wartman’s battle with ALL and now GVHD has transformed him into one of the strongest patient voices advocating for the development of new GVHD treatments. Jon Cohen, the author of the Science Magazine article, explained:

“The urgency of his case has turned Wartman into one of the world’s few patients who advocate for GVHD research, prevention, and treatment. ‘Most people it affects suffer quietly,” says Wartman. ‘They’re grateful they’re alive, and they’re beaten down. It’s the paradox of being cured and dying of the cure. Even if you can get past that, you don’t have the energy to advocate, and that’s really tragic.’”

Patients like Wartman are an inspiration not only to other people with GVHD, but also to funding agencies and scientists working to advance GVHD research towards a cure. We don’t want these patients to suffer quietly. Wartman’s story is an important reminder that there’s a lot more work to do to make bone marrow transplants safer – so that they save lives without later putting those lives at risk.

4 things to know about stem cell clinical trials [Video]

Every day, we receive phone calls and emails from people who are desperately seeking our help. Sometimes they reach out on their own behalf, though often it’s for a family member or close friend. In every case, someone is suffering or dying from a disorder that has no available cure or effective treatment and they look to stem cell treatments as their only hope.

It’s heartbreaking to hear these personal stories that are unfolding in real time. Though they contact us from a wide range of places about a wide range of disorders, their initial set of questions are often similar and go something like this:

  • “Where can I find stem cell clinical trial for my condition?”
  • “What are my chances of being cured?”
  • “How much does it cost to be in a clinical trial?”
  • “How can I be sure it’s safe?”

We think anyone thinking about taking part in a clinical trial should consider these important questions. So, in addition to providing answers as we receive them through phone calls and emails, we wanted to find a way to reach out to as many people as possible. The result? The four-minute animation video you can watch below:

As mentioned in the video, the answers to these questions are only the tip of the iceberg for finding out if a particular clinical trial is right for you. The website, A Closer Look at Stem Cells, produced by the International Society for Stem Cell Research (ISSCR), is an excellent source for more advice on what things you should know before participating in a stem cell clinical trial or any experimental stem cell treatment.

And visit the Patient Resources section of our website for even more practical information including our growing list of CIRM-funded clinical trials as well as trials supported by our Alpha Stem Cell Clinic Network.

“A limitless future”: young crash victim regains hand, finger movement after CIRM-funded trial

Back in March, we reported on Asterias Biotherapeutics’ exciting press release stating that its CIRM-funded stem cell-based therapy for spinal cord injury had shown improvement in six out of the six clinical trial patients receiving a ten million cell dose. What’s even more exciting is hearing stories about the positive impact of that data on specific people’s lives. People like Lucas Lindner of Eden, Wisconsin.

Lucas Lindner was left paralyzed below the chin after a truck accident last May. Photo: Fox6Now, Milwaukee

Just over a year ago, Lucas headed out in his truck on a Sunday morning to pick up some doughnuts for his grandmother. Along the way, he suddenly saw a deer in the road and, in swerving to avoid hitting the animal, Lucas’ truck flipped over. He was thrown through the window and suffered a severe spinal cord injury leaving him without the use of his arms and legs.

Linder was the 2nd person to receive a 10 million dose of Asterias’ CIRM-funded stem cell-based therapy for spinal cord injury. Video still: Fox6Now, Milwaukee

Earlier this month, Lucas was featured in a local Milwaukee TV news report that highlights his incredible recovery since participating in the Asterias trial shortly after his accident. Surgeons at Medical College of Wisconsin – one of the clinical trial sites – injected 10 million AST-OPC1 cells into the site of the spinal cord injury a few inches below his skull. The AST-OPC1 product contains oligodendrocyte progenitor cells, which when fully matured are thought to help restore nerve signaling in the frayed spinal cord nerve cells.

Lucas was just the second person nationally to receive the 10 million cell dose, and since that time, he’s regained movement in his arms, hands and fingers. This improvement may seem moderate to an outside observer, but for Lucas, it’s life changing because it gives him the independence to pursue his dreams of working in the IT and electronics fields:

“Now that I have near 100% full range on all of my fingers, that pretty much brings everything I ever wanted to do back. It lets you contribute to society. Words can’t express how amazing it feels…The future really is limitless,” he said during the TV new segment.

While regaining movement spontaneously without a stem cell treatment is not unheard of, the fact that all six of the trial participants receiving 10 million cells had improvements suggests the stem cell-based therapy is having a positive impact. We’re hopeful for further good news later this year when Asterias expects to provide more safety and efficacy data on participants given the 10 million cell dose as well as others who received the maximum 20 million cell dose.

Advocating for Huntington’s Disease: Daniel Medina’s Journey

Daniel Medina

In honor of Huntington’s Disease (HD) Awareness Month, we’re featuring a guest blog by HD patient advocate Daniel Medina. Daniel became actively involved in the HD community when he learned that his younger brother was at risk for inheriting this devastating neurodegenerative disease. Since then he has been a champion for HD awareness by organizing HD patient support groups and walks in southern California and serving on the Board of HD Care, UC Irvine’s non-profit HD support group. 


Guest Blog by Daniel Medina

A visit to a care home back in April of 2012 changed my life forever. It all started when my mother took my 14-year-old half-brother to meet his grandfather for the very first time. My brother’s aunt led the way to what seemed to be an emotional, long overdue family encounter.  As they walked into his room they were impacted by what they saw.

They saw an elderly, bedridden gentleman that suffered from uncontrollable body movements. He was unable to communicate and was totally dependent on others. As the tears flowed, so did my mom’s sense of urgency to find out the name of his affliction. That’s when the words “Huntington’s disease” were uttered by my brother’s aunt. Her knowledge was limited to sharing that it was a genetic disease.

I immediately began my own research as the details of this encounter were relayed to me. My curiosity soon turned into despair and anguish as I learned that my brother was at risk of being a carrier of this horrible neurodegenerative disease.  I felt empowered as I began attending HD fundraising events. There I met so many courageous families that clung to the hope of a better tomorrow.  This hope came through the possibility of scientists working towards finding a treatment or a cure through stem cell research.

As of 2013 my role had evolved from an event attendee to a patient advocate. It became clear to me that there was an immediate need to fill voids that were unattended. In 2014, I started an HD support group in my area in order to tend to the needs of the HD community. The appreciation and gratitude I felt made every second I invested very much worthwhile.

In the last three years, we have seen the tremendous impact and growth HD organizations like Help4HD International, HD CARE and WeHaveAFace, have had on a local and global scale. It has been such an honor and a privilege to work alongside them. Our collaborative efforts have had a ripple effect of amazing results. The success of one is the success of all.

At the beginning of 2015, I was introduced to Americans for Cures. Working to promote and educate the public about the benefits of stem cell stem research was a perfect fit. Meeting advocates from other disease communities has educated me and taught me how our common goals towards finding cures unites us.

My HD Advocacy journey began with a simple visit to a care home. In a matter of a few years, it has transformed into a life mission to help those suffering the effects of this terrible disease.

2016 HD-CARE Conference. Patient Advocates Ron Shapiro, Adrienne Shapiro, David Saldana, Frances Saldana, Daniel Medina with Karen Ring from CIRM.

Stem Cell Patient Advocates, Scientists and Doctors Unite Around a Common Cause

Some phrases just bring a smile to your face: “It’s a girl/boy”, “Congratulations, you got the job”, and “Another beer sir?” (or maybe that last one is just me). One other phrase that makes me smile is “packed house”. That’s why I was smiling so much at our Patient Advocate Event at UC San Diego last week. The room was jammed with around 150 patients and patient advocates who had come to hear about the progress being made in stem cell research.

Jonathan Thomas, Chair of the CIRM governing Board, kicked off the event with a quick run-through of our research, focusing on our clinical trials. As we have now funded 29 clinical trials, it really was a quick run-through, but JT did focus on a couple of remarkable stories of cures for patients suffering from Severe Combined Immunodeficiency (SCID) and Chronic Granulomatous Disease.

His message was simple. We have come a long way, but we still have a long way to go to fulfill our mission of accelerating stem cell treatments to patients with unmet medical needs. We have a target of 40 new clinical trials by 2020 and JT stressed our determination to do everything we can to reach that goal.

David Higgins, Parkinson’s Disease Advocate and CIRM Board Member (Credit Cory Kozlovich, UCSD)

Next up was David Higgins, who has a unique perspective. David is a renowned scientist, he’s also the Patient Advocate for Parkinson’s disease on the CIRM Board, and he has Parkinson’s disease. David gave a heartfelt presentation on the changing role of the patient and their growing impact on health and science.

In the old days, David said, the patient was merely the recipient of whatever treatment a doctor determined was appropriate. Today, that relationship is much more like a partnership, with physician and patient working together to determine the best approach.

He said CIRM tries to live up to that model by engaging the voice of the patient and patient advocate at every stage of the approval process, from shaping concepts to assessing the scientific merits of a project and deciding whether to fund it, and then doing everything we can to help it succeed.

He said California can serve as the model, but that patients need to make their voices heard at the national level too, particularly in light of the proposed huge budget cuts for the National Institutes of Health.

Dr. Jennifer Braswell. (Credit Cory Kozlovich, UCSD)

U.C. San Diego’s Dr. Jennifer Braswell gave some great advice on clinical trials, focusing on learning how to tell a good trial from a questionable one, and the questions patients need to ask before agreeing to be part of one.

She said it has to:

  • Be at a highly regarded medical center
  • Be based on strong pre-clinical evidence
  • Involved well-informed and compassionate physicians and nurses
  • Acknowledge that it carries some risk.

“You all know that if it sounds too good to be true, it probably is. If someone says a clinical trial carries no risk that’s a red flag, you know that’s not true. There is risk. Good researchers work hard to reduce the risk as much as possible, but you cannot eliminate it completely.”

She said even sites such as www.clinicaltrials.gov – a list of all the clinical trials registered with the National Institutes of Health – have to be approached cautiously and that you should talk to your own physican before signing up for anything.

Finally, UC San Diego’s Dr. Catriona Jamieson talked about her research into blood cancers, and how her work would not have been possible without the support of CIRM. She also highlighted the growing number of trials being carried out at through the CIRM Alpha Stem Cell Clinic Network, which helps scientists and researchers share knowledge and resources, enabling them to improve the quality of the care they provide patients.

The audience asked the panelists some great questions about the need for;

  • A national patient database to make it easier to recruit people for clinical trials
  • For researchers to create a way of letting people know if they didn’t get into a clinical trial so the patients wouldn’t get their hopes up
  • For greater public education about physicians or clinics offering unproven therapies

Adrienne Shapiro, an advocate for sickle cell disease patients, asks a question at Thursday’s stem cell meeting in La Jolla. (Bradley J. Fikes)

The meeting showed the tremendous public interest in stem cell research, and the desire to move it ahead even faster.

This was the first of a series of free public events we are holding around California this year. Next up, Los Angeles. More details of that shortly.