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.

Top 10 Insider Trends on Stem Cells to Look Out for at ISSCR 2015 Stockholm

isscr meetingThe annual ISSCR meeting has started in Stockholm.

This is always a great annual meeting both for the science and for connecting with people including new friends and colleagues as well as old friends.

Another element to the meeting is the insider conversations in the halls, restaurants, and bars that tell a behind the scenes story of the stem cell field.

Below are my top 10 things to look for that might be discussed over a beer or coffee this year. Also be sure to check out the wonderful guide to Stockholm from Heather Main and if you are there at the meeting enter our stem cell contests to win up to $100.

  • Clinics make an appearance? It’s a long shot, but I keep wondering if some of the stem cell clinic folks will show up at ISSCR some day to try to legitimize themselves even if they don’t speak, etc. Maybe they’ll sneak in with some posters or even just attend to make some connections. Unlikely, but if it happened could prove very interesting.
  • I’ll be curious if the Hanna-Silva feud of a sorts continues persist over ground state pluripotency, MBD3 and NuRD.
  • Does anyone still believe in VSELs?  A scandal is still smoldering there.
  • Anybody know what happened to Vacanti and the assumed to exist Brigham & Women’s/Harvard investigation over STAP cells? Last year in Vancouver at ISSCR STAP cells were one of the hottest topics.
  • Will Mitalipov continue to assert that NT-hESC are better than IPSC after the more recent paper (on which he was an author seemed to show otherwise)? More broadly will the SCNT/human therapeutic cloning folks continue to claim a clear path to the bedside?
  • Any news on Masayo Takahashi’s IPSC trial? More preliminary data?. I’m excited to see how that goes.
  • I keep hoping also that more biotechs will present at ISSCR and be given plenary talks.
  • Is ethics/policy given sufficient attention at the meeting?
  • Will CRISPR-Cas9 editing of stem cells be the talk of the meeting? The explosive trend of this amazing gene editing technology in science overall has really gripped everyone’s attention.
  • How many reports of clinical trial data will be given? Sometimes in the past ISSCR meetings have had a sizable tilt towards basic science. Could that be changing?

Do Patients Have a Fundamental Right to Choice?

What is the “proper” amount of freedom of choice for patients in medicine?

What if the treatments in question are experimental and come with their own baggage of associated risks, personal costs, and potential costs to society?

More broadly, do patients have a fundamental right to medical choice?

These questions seem particularly appropriate today on a number of fronts including Right To Try laws and vaccines as well as emerging stem cell and other biomedical technologies. The recent measles outbreaks including the one sparked at Disneyland are warning signs of what harm is awaiting us if the choice to opt out of vaccinations continues to be available to the general public. The California Senate today passed the mandatory vaccination bill. Assembly lawmakers are now weighing the bill.right to choice in medicine

As we discussed at our recent UCD Stem Cell Ethics Symposium, that phrase “a fundamental right” is very powerful. A potential benefit of medical freedom would be the power to make choices that may benefit oneself or one’s loved ones that otherwise would not be available if solely decided on a collective, governmental level.

For example, there may be a new experimental drug that a doctor and patient together decide is worth the risk to try, but the FDA has not approved it or even given the green light for a clinical trial on it. In principle, that drug might help the patient or it might hurt or even kill the patient. Advocates of medical freedom believe patients should broadly be allowed to evaluate and take such risks with their own personal physician if they so choose. They see the FDA as an obstacle to their freedom.

As someone who has faced a very serious form of prostate cancer, I get the sense that being in a serious medical situation can change one’s views. Everything feels different when you are faced with a potentially lethal medical diagnosis yourself or for a loved one. Sometimes it’s not that simple though.

As individuals our freedom and desire for choices can conflict with a greater good. For example, as a biomedical scientist and parent I do not believe that childhood vaccination should be optional. While there is no compelling evidence that vaccination is harmful beyond for that tiniest fraction of those who have severe reactions or pre-existing immune diseases, there is profound evidence–I would go so far as to say proof–of powerful benefits to both individuals and society as a whole of vaccination.

Some parents feel that the best choice is not to have their kids vaccinated. Should they be allowed that choice? I don’t think so in the sense of the unvaccinated kids still being allowed to go to school where they can interact with and potentially harm other children via infectious diseases. Not being vaccinated also poses risks as an adult for other workers in the workplace.

While some might say that mandatory vaccination is “anti-freedom” and that there are costs to such governmental mandates, from my view the benefits dramatically outweigh those risks. But some argue a libertarian view that there is a price for that in loss of free choice.

Another example of a hotly debated area today that shares some of the same issues is emerging stem cell technology. Some say that patients should be free to entirely make their own choices as to whether to get an experimental, potentially risky stem cell intervention. The argument is also in conjunction often made that patients are in the best position with their doctors to make such decisions. “Get the government out of my doctor’s office”, might be the clarion call for those folks.

These kinds of “freedom-based medical decisions”, as we might call them, have possible societal costs even if they do not involve highly contagious infectious diseases. If companies sell unapproved stem cell treatments, then patients buying those treatments are at the same time as they think they are trying to help themselves, also are undermining the authority of the FDA more generally and in that way indirectly putting future potential patients at risk. As much as I think the community that reads this blog can agree that the FDA is imperfect and could benefit from reforms, the FDA performs a very difficult, complex and crucial mission, without which we’d face medical chaos leading to great harm.

It seems appropriate at this point to also point out that many stem cell clinics also sell these interventions to children. As such, if you believe that people should be allowed to get any stem cell therapy they want, keep in mind that there are possible broader consequences to you making that choice beyond undermining the FDA mission. One might say, for instance, that you are supporting a sketchy company that is putting other people at risk. Some of those other potential patients may not be in the same position as you to make decisions about risks. They may be kids or they may be less educated than you. By giving this stem cell business your money you are enabling them to put others into risky situations.

As much as some people might advocate for specific stem cell clinics selling unapproved stem cell treatments, I’d say that within this community most of us also can point to some clinics and doctors (and fake doctors) that are truly frightening and dangerous. Even if reasonable people can disagree on broader issues regarding the appropriate level of governmental regulation and freedom of choice, relentlessly attacking the FDA for the cause of near universal freedom of medical choice for investigational stem cell treatments poses the risk of giving the wildly dangerous clinics a freer hand. Sometimes what we believe is best for ourselves cannot be viewed only in a bubble.

The bottom line is that freedom of medical choice for an individual is far more complicated than it might seem and it is easy to oversimplify it. The reality is that our medical freedom and decisions have impact on others. I don’t see that we in the America, for example, have a fundamental right to medical choice based on The Constitution. Our medical rights and ability to make health-care related choices change over time too.

Should there be a constitutional amendment for a right to freedom of medical choice?What’s the best goal for these rights (or lack thereof) even if not a constitutional right for the immediate future? How much freedom in this area is best in the longer term?

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.

UC Davis 2nd Annual Stem Cell Ethics Symposium: Save the Date Feb. 12

We here at UC Davis School of Medicine are gearing up for our 2nd annual Stem Cell Ethics Symposium (see flyer).stem cell ethics

As one of the organizers, I’m really excited about our lineup of speakers including Timothy Caulfield, Leigh Turner, Alison Sorkin, Mary Ann Chirba, and Richard Garr as well as others.

We will be focusing on “Right To Try” laws, FDA regulations, and stem cell tourism.

It should be a cutting edge, spirited meeting and discussion.

All are welcome.

Please RSVP to Julie Bechtel @ 916-734-6181 or julie.bechtel@ucdmc.ucdavis.edu.

Hope to see you there.