Of Mice and Men

Since this is Sunday, I'll take a break from speechifying and let this YouTube video do the talking. I bet the audience would not be laughing if they were watching three people on the spinning rod instead of three mice. Here's another one about this type of experiment.

On another note, I found out as I was looking for additional YouTube videos about SCA that there is an experimental rock group called Ataxia. If a punk rock group can call themselves the Dead Kennedys, I guess anything is up of grabs.

International Ataxia Awareness Day

Next Tuesday, September 25, 2007, is International Ataxia Awareness Day. The purpose is to create awareness of this relatively rare disease. I am getting a head start in doing my part by posting to this blog in the hopes that you will click here to learn more about Ataxia and how you can help in this effort.

Please consider making a donation to the National Ataxia Foundation by clicking here. Your gift will help Richard and others suffering from this terrible disease through funding the research needed to find the cure. Thank you for your support.

In terms of Richard's progress, there's not much change. He has recently started to work at home with a personal trainer once a week on additional exercises to strengthen his muscles. Despite Richard's diligence in working out at home, at the Y and at his weekly Pilates sessions, he has found that his muscles are getting weaker. Moreover, his sleep has been poor lately due to night terrors. He'll probably ask Dr. Perlman if there's medication for the night disturbances.

An evening with Dr. Andrew Fire

We're back from Vancouver. It is good to be home. Having traveled quite a bit in the last three weeks, we've become more aware of how airports and airlines differ in accommodating people with special needs. Hong Kong, Burbank and San Jose airports are amongst those that are the friendliest with the best wheelchair attendants. Southwest Airlines deserve special mention for going the extra mile for disabled passengers. We've flown with Southwest more times than we can count and each time, they have been unfailingly helpful, courteous and unobtrusive, on top of that they also offer $49 fares to LA.

In contrast, at both JFK and Vancouver, our wheelchair attendants were no-shows while at SFO, we had to push Richard up a very steep ramp and then waited from another 20 minutes for the wheelchair attendant to appear. While generally it is possible to push Richard around most airports through security checks, departure gates and baggage claim, there are airports, JFK being the most notorious, where in order to get to the elevators to go down to baggage claim and transportation, one needs to go behind and into restricted areas. This is where the wheelchair attendant becomes vital since he/she has the proper i.d. or key to operate the elevator. Enough of my ranting ....

Richard and I recently heard Dr. Andrew Fire speak on his research on RNAi. Dr. Fire, together with Dr. Craig Mello, received the 2006 Nobel Prize for Physiology or Medicine for the discovery of RNA interference (RNAi). RNAi is a means to "silence" the messenger RNA (mRNA) from giving instructions to make a certain protein. In the case of Polyglutamine Diseases, (including Huntington's, SCAs 1, 2, 3, 6, 7 & 16) RNAi therapy can potentially be used to stop the mutant gene from being expressed. As promising as this breakthrough discovery is, Dr. Fire cautioned that it would take many years before RNAi therapy can become a practical reality. He said that the human physiology is much more complex than that of the C. elegens, a nematode worm, which was his research subject in the RNAi experiment.

You can find the link to his Nobel lecture of RNAi here. Although we probably understood less than half of the highly technical material that Dr. Fire presented, his passion and enthusiasm for the subject matter is easy to see.

Persona non grata

It's official --- the pointer to our blog has been removed from the stemcellschina.com website. Thanks to our friend Nick who brought this to our attention.

An apology

I want to apologize to the ex-Nanshan patients to whom I've e-mailed requesting their opinion on the last post concerning ethics. In hindsight, I was most insensitive and inconsiderate in putting them in an awkward and no-win situation by asking them to either support my view or Mr. Hakim's. Mea maxima culpa.

A question of ethics

I had an interesting discussion with Jon Hakim, director of the foreign patient services department at Beike Biotech today about ethics. On several occasions, Jon e-mailed us with requests that we did not comply with because we deemed them unethical and improper. Jon, on the other hand, thinks that these requests were reasonable. So to settle this debate, we agree to poll this audience or, more specifically, the folks who have received treatment in Nanshan. I will forward the contents of this post via e-mail to the Nanshan patients whose email addresses I have. I welcome the opinions of the readers of this post as well.

Point 1: Is it ethical for Jon to ask Richard to post the actual medical report of his visit with Dr. Perlman this past May when Richard was given the SARA (Scale for the Assessment and Rating of Ataxia) which quantified his progress after the stem cell treatment? Jon made this request after he read our May 9 post about the results of the test. We had never given Jon any indication that we would be open to sharing Richard's medical records and reports publicly nor did we ask for his input on our visit with Dr. Perlman. We received his e-mail on May 16, which reads:

I was wondering if you could post the medical reports that you have gotten from Dr. Perlman on your web site? It would add some credibility to it, I believe. Thank you.

We responded by saying that we would not because the information is private. We have already stated in our blog that there was measurable improvement. Why do we, the patients, need to lend "credibility" to the clinical assessment? Who is going to challenge our word? The purpose of our blog is to keep our friends and relatives abreast of Richard's progress. We are not in the business of proving or disproving that the treatment works nor are we hawking Beike's stem cell treatment.

Jon countered that various patients, including Kim Poor, have expressed their desire to post their medical records publicly to substantiate the effectiveness of the treatment. Since we have posted Richard's numerical SARA score in our post, Jon assumed that it was OK for him to ask us to post the actual medical report.

My problem with Jon's request not only stems from his casual treatment of such personal information but that the request came from Beike and not the Nanshan doctors. Isn't there a conflict of interest here? What would possibly justify the need for the representative of the company that sells the treatment to ask for medical data directly from the patient? Isn't this the domain of the doctors at Nanshan? In the US, medical institutions must follow specific guidelines to ensure the confidentiality of patients' medical records (HIPAA). Not only that, but there is strict protocol about who should and should not contact the patient in clinical trials so as to safeguard the impartiality of the data and experiment.

Point 2: When we returned from China, we were scheduled to see Dr. Perlman for a post-treatment consultation on May 7. As we mentioned in our May 1 post, Richard had sprained his foot in late April. We received an e-mail from Jon on May 4, asking us to consider postponing our visit with Dr. Perlman for another week because, in Jon's exact words, he wanted the doctor to see Richard on "an up day instead of a down day."

Since we live in the San Francisco Bay Area, and Dr. Perlman is at UCLA in Southern California, a last minute change in plans (2 days notice and on a weekend) translates into time and money lost in rescheduling flights and lodging. Moreover, the lead time to get an appointment to see the doctor is easily 2 to 3 months. Those who have traveled with a disabled companion understand the complicated logistics involved in not only packing for special needs but also arranging for other specific accommodations and reservations necessary to make such travel happen. A request that would disrupt such carefully laid plans should not come lightly and without good cause.

Would delaying Richard's visit a week have a significant effect on the accuracy of the SARA score? I don't know. I do know that Dr. Perlman emphasized before our China trip that it was very important for her to examine Richard as soon as we returned in order to measure his progress. Our May 7th visit was almost a month after we had left Nanshan. Kim Poor wrote in his blog that he saw Dr. Perlman as soon as he deplaned in Los Angeles from China.

Would Jon have cause to ask Kim to postpone his visit if he had known that Kim was going to see Dr. Perlman after a long and tiring plane ride? How would jet lag impact Kim's performance during Dr. Perlman's examination? If Jon had made this request to him, would Kim have complied? Are these questions even relevant since a physician is trained to take into consideration how factors like travel, a sustained ankle sprain or fatigue might affect the performance of the patient?

One thing on which Jon and I agreed is that his e-mail telling us that he would rather us not mention about the recent Wall Street Journal article here on our blog is ethically questionable. His e-mail further said that "the WSJ is evil, especially that reporter." In another e-mail, Mr. Hakim commented that "this journalist in general is well known to spin his stories anti China as much as possible and I had firsthand experience." Not only did I mention the article in the blog but I also wrote this. I believe in presenting both sides of the story and letting the reader decide.

I cannot shake the feeling that Big Brother is watching. Is Jon using our blog to monitor us? I'd like to believe that he is interested in Richard's health as a friend and not as a salesperson of stem cell treatment. However, some of his requests can be construed as too self-serving.

Simply put, we believe that our medical records are private and should only be shared on a "need to know" basis with people who are empowered and qualified to tend to our welfare. Beike should not make unsolicited suggestions and requests to the patients in matters pertaining to the patient's medical decision-making process such as the disclosure of medical reports and physician visits.

What do you think? We'd like to hear your perspective.

Everything you've always wanted to know about stem cells

Richard and I spoke to our stem cell class at Stanford this past Monday. Each week, Professor Scott asks for student volunteers to make short (10 minute) presentations on any topic pertaining to the subject of stem cells. We spoke about our experience in China, and the response from the class was tremendous - our 20 minute presentation ran over an hour. Afterwards, Professor Scott wrote about it in his blog here.

For those who want to learn more about stem cells and the latest technological advances in this area, there are free podcasts of each week's class on iTunes. Go to iTunes, click on iTunesU, then choose Stanford University, click on "Straight Talk About Stem Cells." If you have trouble accessing iTunes, Professor Scott's book, Stem Cell Now, is a great source in understanding the basics of stem cells. We read this book in the class.

Afterward our representation, many people thanked us, but we really should be thanking them for letting us share our experience and for their insightful, perceptive questions. Some of our classmates work in the medical field while others are teachers, engineers and researchers. Most have a good understanding of stem cells. Here is a sample of some of the Q&As:

Q. Did the Chinese doctors follow up with Richard after he was released from Nanshan Hospital?

A. No. We were given a discharge report with no specific instructions on how or when to follow up. We were, however, told that we could contact the hospital staff should we have any questions or concerns.

It is standard medical protocol in the U.S. for doctors to follow-up on the patient's progress after the patient is discharged from the hospital. This typically involves medical prescriptions, instructions on follow-up care and a check-up appointment with the doctor. So far we have not been contacted by the Nanshan doctors, but we have received a group email from a U.S.-based Beike representative asking Richard about his progress.

Q. How did we hear about stem cell therapy at Nanshan? What studies or reports did we read that convinced us in going to China?

A. We heard about Nanshan from fellow MJDers' experiences. There were no formal medical studies or reports published or provided by Beike about the Nanshan stem cell patients that we were aware of. We researched Beike's website, sent an inquiry to Beike and corresponded with their staff. We accept the fact that we had no guarantee that the treatment was going to work.

I distinctively remember cringing as I listened to my answer to this question. Did we sound desperate and naïve? There is so little that is published about Chinese clinical results that prospective patients mostly rely on anecdotal evidence from stem cell blogs like this one and others at stemcellschina.com. In the package of materials provided by Beike to prospective patients, there was a list of publications by Dr. Yang Bo of Nanshan Hospital, but none of these papers are easily accessible (outside of China). My suspicion is that even if they were available, the text would be in Chinese.

Q. (Directed to Prof. Scott) Were there any representatives from China at the Annual Meeting of the International Society of Stem Cell Research in Cairns, Australia last month?

A. No. The organizers have hoped that by holding the meeting in Australia that it would encourage more participants from Asian countries. Japan was well-represented.

There was some speculation from the class as to why China did not participate. One classmate who is ethnic Chinese suggested the language barrier might be a factor. Perhaps Chinese researchers and doctors are not fluent in English and thus not confident in presenting their work in English. Perhaps, she also hypothesized, their non-attendance can be attributed to the traditional Chinese belief that one should not toot one's horn for fear of being called immodest. I subscribe to the former argument.

I can understand why there is so much skepticism about stem therapy in China. The burden of proof as to whether or not the treatment works has shifted to the patients and not the Chinese doctors or researchers. Patients write about their experience and progress through blogs that are used as anecdotal testimonials. The patients then cite reports of medical visits with doctors in their home countries to further support that there is measurable improvement. In contrast, Chinese doctors seem disinterested in documenting and following up on their patients. This curious lack of interest and initiative on China's part may lead the West to believe that China has something to hide. If the treatment works, then why not show the world solid empirical data instead of relying on soft anecdotal support? Patients can talk themselves blue in the face about how they think the treatment worked for them; unless the Chinese are willing to subject the trials to peer review, however, the treatment will never gain any legitimacy in the West.

Someone in class asked us if we had any regrets about going to China. We told her that we had no regrets whatsoever because we entered into this venture without any expectations of a miracle cure. I still believe that stem cells are the future of medicine. There has been much finger-pointing about how the West is determined to short-change China's efforts. As an ethnic Chinese, I want China to succeed in becoming a premier provider of stem cell therapy. But it is not going to get there without self-advocacy and opening itself to Western scrutiny. There needs to be more effort in the documentation, disclosure and discussion of its clinical experiments. If China wants its accomplishments to be recognized in the West, she must abide by the same protocol and regulations of the Western scientific community, particularly peer review. I can only hope that discussion on blogs like these and in other forums will help facilitate greater cross-cultural understanding and dialogue on stem cell treatments.