Stem Cell Person of the Year 2016: Patient Advocate Ted Harada

Stem cell ethics meeting

Ted, Me, and Judy

Patient advocate Ted Harada is the recipient of this year’s Stem Cell Person of the Year Award.

Congrats also to the runner-up, HD patient advocate Judy Roberson. The three of us together are pictured at left.

You can read about the 20 nominees here and see the vote results that picked the 10 finalists here.

Very sadly, as many of you know, Ted passed away just a few months ago from a brain tumor so I am giving him this award posthumously. Accepting the award on his behalf is his wife Michelle.  Ted and I shared a deep commitment to our families. You can see a picture of Ted, Michelle, and their kids below. What a great family!

You can see a video of Ted talking about Right To Try below.

Each year that I’ve done the Stem Cell Person of the Year Award, I’ve been faced with the wonderful, but difficult challenge of picking one winner out of a group of outstanding finalists and this year was no different.

With this award that includes a $2,000 prize, I’m looking for an outside-the-box risk taker who has made a positive impact in the world of stem cells. Ted fit the bill perfectly. Ted Harada Family

Ted was a clinical trial participant for a new stem cell therapy for ALS in a trial run by the biotech Neuralstem. As such, Ted put himself at risk (transplanted cells have risks, immunosuppression has risks, etc.). He did this for the benefit of the field and for other patients. However, Ted went well beyond that. He was also a tireless patient advocate and educator who inspired countless people.

Ted respected other’s opinions and was a true class act. For instance, although Ted and I didn’t see entirely eye-to-eye on some things like Right to Try, that wasn’t a wedge. He served as a bridge between different parts of the community. Here at UC Davis we run an annual symposium on stem cell ethics and one year Ted was an invited speaker. He made a big, positive impact at our meeting.

Overall, Ted left the world including the stem cell and regenerative medicine arena a far better place. You can read my tribute to Ted after his death here. I only wish I could have given him this award in person.

Meeting review of unique stem cell ethics symposium @UCDAVIS

On Thursday here at UC Davis School of Medicine we held our second annual Stem Cell Ethics Symposium. The symposium was organized by Drs. Mark Yarborough and Nanette Joyce as well as me.

In this post I report on key take homes from the meeting and summarize the specific talks.

As far as I know, this is one of the few events in the world where there is such a wide diversity of stakeholders present both as speakers and in the audience to specifically discuss stem cell ethics for a full day. The participants included numerous patients and patient advocates, bioethicists, stem cell researchers, physicians, attorneys, institutional compliance officers, and more.

Stem cell ethics meeting

One of the things that I most appreciated about this meeting is that diverse views were welcomed and we dealt head on with some of the most challenging ethical issues related to stem cells.

I also got to meet in person some people I had only previously known on-line including keynote speaker Tim Caulfied, patient advocate Ted Harada, and advocate and frequent commenter on this blog, Cathy Danielson, which was fantastic. My colleague Leigh Turner was also present. Old friend and Huntington’s Disease advocate, Judy Roberson, was there as well. You can see the picture above of Ted, Judy, and me.

After a great introduction by our new Dean of the School of Medicine, Dr. Julie Freischlag, Nanette Joyce started things off with a very powerful talk about her experiences with her ALS patients. Her patients are facing incredibly intense challenges as they deal with all that ALS brings. Both in her medical specialty and amongst her patients, she noted, there is growing interest in stem cell treatments. Dr. Joyce expressed her concern over the lack of evidence of safety and efficacy, and how elements of hype have entered into the arena of ALS and stem cells. ALS is a very heterogeneous disease with ups and downs as well in individual patients, but life expectancy on average is very short after diagnosis.

One of Dr. Joyce’s patients, Michael, then gave a talk about his experiences with ALS. I found it really striking. Literally every day he goes on-line and checks out resources to search for opportunities for ALS patients such as clinical trials. Michael indicated that as far as he knows he’s not eligible for any trials so far. This was a theme that several people mentioned during the day: just because there are trials out there does not mean that patients with ALS or other conditions can be part of them. Patients are seeking other opportunities.

Next up was Ted Harada, who spoke passionately about his experiences with ALS, the ups and the downs, what is really like to get a stem cell treatment and be in a clinical trial. His talked about his willingness to take risks. He said he’d literally rather die trying something than not try to do something about his ALS. Ted has had two rounds of treatment for ALS as part of the Neuralstem clinical trial and he reported that it has really helped him. One of Ted’s concerns is that he may not be eligible to get a future third treatment from Neuralstem for various technical rules of the clinical trial, which is one reason he is such a strong advocate for Right To Try laws. However, Ted said he feels that most likely few or no patients will be directly treated as a result of Right to Try, but rather that it will open up a dialogue and create other positive outcomes that lead to more patient opportunities.

From both Michael’s and Ted’s talks, i also learned that it can take a very long time (potentially a few years) to definitively diagnosis ALS. Patients facing ALS are likely to have a much higher level of risk tolerance than others might imagine. Understandably they have a strong drive to take action.

I spoke next and focused on the growing challenge that the FDA and the stem cell field face of chains of stem cell clinics that appear to be selling unapproved stem cell drugs without FDA licensing. I talked a bit about the growth in providers offering unapproved stem cell “treatments” at Cell Surgical Network as an example. More broadly, the clinics argue that what they do is beyond the scope of the FDA’s regulatory authority. I discussed new FDA guidances that bear on this issue and which would seem to directly contradict the views of many of the clinics, squarely placing them and their products in the FDA sphere from my perspective. The clinics of course disagree. I also emphasized how the FDA currently is asking for comments on these guidances for the next couple weeks.Tim Caulfield

Tim Caulfield spoke next, giving a wonderful talk. In the stem cell world, hype is not just limited to dubious stem cell clinics. Tim focused on hype in the stem cell field and in particular hype involving scientific publications. He even talked about hype about hype. In the current environment there are strong pressures for scientists to hype their work, including in particular in abstracts. What is the relationship between hype in science articles and in the media? He discussed how widespread pressure to commercialize biomedical research including stem cell efforts sometimes has negative consequences and can contribute to hype.

Alison SorkinAlison Sorkin, Deputy General Counsel for University of Colorado Health, talked about Right To Try from her experiences in the trenches. With the passage of the Right To Try Law in Colorado, Sorkin was there for the intense repercussions. The next working day after the law was passed, patients starting contacting the University of Colorado seeking treatment specifically under the law. Sorkin talked about strict the Colorado law actually is, making it very limited in scope in reality. She also discussed problematic issues with the specifics of the law such as that patients would be responsible for paying for all of their own healthcare for 6 months after treatment under Right To Try as insurers would be exempt from having to provide ANY coverage. There seems to be a growing sense that Right To Try in Colorado may not actually lead to any patients getting non-FDA approved drugs. However, the FDA is paying close attention to Right To Try and one potential consequence of the various Right To Try laws passed in states is that the FDA could be influenced in terms of policies on expanded access (compassionate use).Leigh Turner

Leigh Turner gave a very timely, excellent talk on the FDA, stem cell clinics that do not have FDA licensing or approval, and the potential consequences of this current dynamic. Leigh went into helpful detail on the clinic claims, the types of stem cells they use, and how these relate to the new FDA guidances. He discussed Cell Surgical Network and Stemgenex. It is notable that Leigh gave specific examples and names clinics by name, which I agree is vital to the discussion of the regulatory sphere and the place of stem cell clinics.

Gerhard Bauer, my colleague here at UC Davis and one of my favorite stem cell scientists, gave a wonderful talk on his experiences and perspectives over the decades as a pioneering stem cell and gene therapy clinical researcher as well as earlier as a regulator at the FDA of Austria. Gerhard’s discussion ranged from his experiences in these roles to also his own person experiences as someone who has dealt with doctors and also lost many friends to disease including HIV. I also appreciate how he provided his views from his real world experiences wending his own clinical trials through the processes and how much work it takes just to get an IND. How great it feels to get to that point!

A few additional notes

Mary Ann Chirba, a voice for increased patient autonomy in terms of their cells, was going to be a speaker, but got snowed in at Boston. Richard Garr, Neuralstem CEO, was also scheduled originally to speak, but couldn’t make it. They were missed, but still we had a diverse, dynamic group of perhaps 70-80 attendees.

A big thanks to Julie Bechtel who helped to arrange the event.

Interview with Ted Harada Part 2: Right To Try, Neuralstem, and How He’s Doing

This is Part 2 of my interview with ALS and stem cell advocate, Ted Harada.

You can read Part 1 on where things stand today with ALS, the FDA, and the Ice Bucket Challenge.

What’s your opinion of the “Right To Try” law in Colorado and the concept more generally? It seems to have the potential to speed innovation, but also put patients at risk. How will this be balanced out? 

Ted: I am a strong advocate for Right To Try.  I even wrote an Op – Ed that ran in the Atlanta newspaper in support of it.  Even when we declared our independence as a nation it was written that people have the right to life, liberty and the pursuit of happiness.  So what I see the Right To Try doing in addition to speeding up innovation is giving terminally ill patients the right to try and keep living.  As I stated in my Op-Ed I appreciate that the FDA is the Gold standard of drug safety, however perhaps if you are out of options you are willing to settle for the silver standard.  It can take 10 – 15 years and sometimes nearly a billion dollars to complete a drug trial. Those dying do not have the luxury of waiting that time.
As far as patient risk with Right To Try the law stipulates that the patient has to have informed consent just as if you were involved in a trial.  Plus I believe that the monolithic and paternalistic approach taken by the FDA actually pushes patients into taking even greater risks such as the stem-cell tourism that I referred to earlier.  Another important safe guard of Right-To-Try is that the treatment has to have completed an FDA phase I portion of a trial and been approved for phase II which means in the small sample size of the first phase it showed it was safe.  This is a much higher standard of safety then many patients have afforded to them when they take risks on untested treatments overseas.

One other thing I would like to point out is that it is not called the right to a cure. It is called the right to try it. It is not a promise that it will help you but if you are interested and have a Doctors and Bio or Drug companies support you have the Right To Try.

Perhaps ultimately this law may push the FDA to update the regulatory practices they themselves referred to as outdated.

What’s your view of how NeuralStem is doing? Are there other companies out there that you support and/or are excited about related to ALS?

I obviously am very partial to what Neuralstem is doing.  I find them to be a very unique organization that while they understand their fiduciary obligations to their investors they want to balance that by also doing what is best for patients.  Richard Garr the CEO has taken the ice bucket challenge and attends and personally donates to ALS charities.  He attends the MDA night of Hope Gala, he flew to Atlanta last year and participated in my walk to defeat ALS team “Tread for Ted” in addition to donating.  Their Chief Scientific Officer Dr. Karl Johe attends every surgery and sits in the waiting room during the 5 hour surgery until he knows the procedure was completed safely.  It is a very different type of company.  I believe they truly want to make a difference. I know Brainstorm a company from Israel has shown some preliminary positive results using a patient’s own bone marrow stem cells in an ALS trial and will be starting a trial here in the U.S. soon.  That seems to hold some promise. I also know that Biogen, Knopf and Sanofi have all invested significant capital into ALS research.  I am sure there are others I am forgetting.

How are you doing? (if you don’t mind me asking) 

Honestly I am doing very well.  I have received the stem cells twice and both times it helped me.  I still have ALS but my situation is very stable as of now.  This is why I am so passionate about being the best advocate possible for the ALS community.  I am inspired by all of the people that are living with ALS and their caregivers I want everyone to have the same opportunity that I have had.  To whom much is given, much is expected.  As long as I can I will fight for the ALS community. Here is the link to my Op – Ed

Ted Harada Interview Part 1: Stem Cells for ALS, #IceBucketChallenge, & FDA

Ted Harada FamilyTed Harada is a wonderful patient advocate for the stem cell field and for the development of safe and effective stem cell products to treat ALS. Ted has ALS and received stem cell-based treatments for it with surprising, very encouraging results. The Harada family is pictured at left with Ted, his wife Michelle as well as their three children Ted, Jordan, and Ashleigh.

Ted was a finalist for this blog’s Stem Cell Person of the Year Award last year and he achieves a great deal by doing his homework and working very hard.

I just invited Ted to do an interview about whether things stand on ALS today.

Below you can find Part 1 of my interview with Ted including his perspectives on the Ice Bucket Challenge.

Part 2 will follow soon including Ted’s thoughts on Neuralstem, Right To Try laws, and how Ted’s doing these days.

What are the most important things the public needs to know about ALS?

Ted: ALS is 100% fatal. There are no survivors. There is no known cause and no known cure. Every 90 minutes in this Country someone is diagnosed with this insidious disease and every 90 minutes someone dies from ALS.

Where do you see things today overall for the prospect of using stem cell-based therapies to treat ALS? Hopeful? Discouraged? In the middle? Why?

Ted: Honestly all of the above. I am obviously hopeful, how can I not be hopeful with the results I had that were supported with empirical data. That being said a few things frustrate me. Cases like mine lead to what I believe are less than ethical stem cell clinics that pop up around the globe and even in our own Country. People who are dying are willing, understandably willing to try almost anything and pay a lot of money for it. So preliminary success definitely leads to I believe what the industry refers to as stem cell tourism.

The other thing that I am waiting with baited breath for is more documented stories like mine. I believe what really complicates all treatments for ALS stem cells or otherwise is that ALS is really a syndrome. So there are most likely many forms of ALS therefore as my Neurologist has said to me I may have found what can help your form of ALS but I have no idea what your form is or anyone else’s form.

I once heard a Neurologist give the example of saying a patient has ALS is like saying every time a radio breaks the radio has radio disease. We know there are many reasons why the radio stopped working. Therefore if you took 100 radios and put them in a trial where you were going to change all of their speakers logically we know that only a small percentage of those radios will be fixed by changing the speakers.

That being said I want to reiterate in the big picture I 100% believe that stem cells or perhaps a combination of stem cells and drugs hold the most promise for the future of finding a treatment for ALS.

The Ice Bucket Challenge has drawn some great attention to the ALS advocacy arena. Can you tell us more about it? Do you support it?

A young man that I have had the pleasure to meet Pete Frates a former Boston College baseball player who has ALS saw this novel concept on the internet but it was not tied to a specific charity. He took the idea named it the #ALS #icebucketchallenge and started challenging athletes he knew from Boston College from ice hockey and football and baseball etc and the phenomenon has obviously become a viral sensation that has done more to raise awareness and funding in just a few short weeks than has been done in the 75 years since Lou Gehrig gave his famous speech retiring from Baseball due to the fact that he was being killed by ALS. I am a huge supporter of it, I have participated in it twice and as of 8/20/14 the challenge has raised $31 million for the ALS association. Hopefully all this money will lead to a cure but in the meantime the disease while no less devastating today is certainly a lot less lonely. Please continue reading on Page 2!

Neuralstem flirting with stem cell noncompliance in Colorado via Right To Try Law?

Colorado is an independent-minded state. For example, it is one of only a few states including Washington State with legalized marijuana use.

More recently, Colorado passed a new law supported by the ultra-conservative Goldwater Institute, the so-called Right To Try Law. This law allows patients to try experimental, unapproved medical interventions that have completed a Phase 1 clinical trial. Most Phase 1 drugs ultimately fail to be proven safe and effective and Phase 1 only demonstrates a minimum baseline level of safety.Dallas_Buyers_Club_poster

Interestingly, Colorado is also a hot bed for experimental stem cell-related interventions. Putting these two realities together, when the Right-To-Try Law passed it seemed likely that we’d be seeing Colorado ending up as a magnet for patients wanting to try experimental, non-FDA approved stem cell therapies and that stem cell companies, including some that previously had records of compliance, would eventually begin federally non-compliant or at least unapproved operations in Colorado.

It didn’t take long for these predictions to be realized as stem cell biotech Neuralstem announced on June 5 that it would be offering its experimental, non-FDA approved human neural stem cell therapy for ALS in Colorado. And it might do it in a for-profit manner. Further, Neuralstem CEO Richard Garr has indicated that the company will not work with the FDA for the human experiments in Colorado. It is worth pointing out that CEO Garr is also Chair of the Goldwater Institute’s Right To Try National Advisory Council.

One of the big concerns about Right To Try-enabled operations of this kind is they could well end up being conducted in a manner that is noncompliant with federal law such as by exposing relatively large numbers of patients to risks.

Apparently the Colorado Right-To-Try Law was inspired by the movie, The Dallas Buyer’s Club, at least in a quote attributed to Colorado State Sen. Irene Aguilar. In his column on the law, David Kroll in turn quoted a column by bioethicist Art Caplan criticizing the law:

“Passing bills full of empty promises based on Hollywood rather than the real world of today is not compassionate but, actually, flat-out cruel.”

As much as I support expanded compassionate use for patients with a fatal illness such as ALS or terminal cancer, Right To Try is not likely to be the solution. It is not the same thing as carefully administered compassionate use, it could put patients at risk, and it could falsely raise hopes of patients. I can understand how some patients and their families would support Right To Try though.

There is a not so easily resolved tension between the completely understandable desire of terminally ill patients to be allowed to try experimental drugs and the concerns of those who advocate for appropriate regulatory oversight as well as evidence-based medicine. This is made all the more complicated by the current clinical trial system that is slow, expensive, and clunky, but absolutely something that should not be thrown out entirely by any means.

Until you are facing a potential terminal illness you don’t know what it is like to be in those shoes. When I was diagnosed with a very serious form of prostate cancer in late 2009, I thought for a while it might ultimately be a terminal cancer for me given what my doctors were telling me. Now four and a half years after surgery I’m doing great (knock on wood), but the cancer could come back. I know that and in part because of my own experience I respect patients greatly.

Still there are definitely some complexities and potential problems that are going to pop up in Colorado due to this new law. Patients are likely going to be put at risk and potentially end up spending tens of thousands of dollars for interventions that don’t work, but as a company Neuralstem is also potentially putting itself in jeopardy. Given that federal law trumps state law, it is unclear to me that Neuralstem can simply ignore the FDA.

It is also likely that the Right To Try Law will embolden other for-profit stem cell entities in Colorado to push the limits of what is permissible in terms of experiments on patients and may well lead companies from other states to do some stem cell interventions in Colorado thinking they are shielded by the state law. They might not be. To protect patients at least financially, I propose that Right To Try laws require that companies that engage in these activities be required to do so only in a not-for-profit manner. It would also be ideal if such companies consult with bioethicists in advance of beginning such operations.

Given that other states have already passed Right To Try laws including Louisiana and Missouri that are awaiting their respective governor’s signatures and that Arizona voters will see a similar bill on the ballot in November, will the FDA feel it needs to take swift action to avoid things spiraling out of control?

Importantly, PBS reports that a federal law has been proposed that is similar to Right To Try. The so-called the Compassionate Freedom Choice Act of 2014, if passed, would in my view introduce chaos into the federal system of careful medical oversight. A key part of this bill reads that it would:

“Amends the Federal Food, Drug, and Cosmetic Act to declare that nothing in it or in the Public Health Service Act shall prevent or restrict, and the Food and Drug Administration shall not implement or enforce any law to prevent or restrict, the manufacture, importation, distribution, or sale of investigational drugs or devices for terminally ill patient.”

In a sense, the Colorado Right-To-Try Law as it pertains to stem cells is itself a big human experiment and it will be probably end up being fascinating and perhaps disturbing to see how this all plays out even as there are concerns over risks it brings with it. I worry it might do more harm than good. What do you think?

For other takes on Right To Try laws, see this very thorough piece by Orac and a piece supporting these kinds of laws from the Goldwater Institute.