Q&A video with Paul Episode 2: stem cell & CRISPR questions answered

This is the second in a series of videos I’m doing where I answer questions about the stem cell field. You can see the Episode 1 video here.

Today I tackle more questions on the stem cell field. If you have more stem cell questions, let me know and I’ll try to answer them next time.

One reader asked me about stem cells for stroke and about one company, Athersys, in particular, but I haven’t had time to tackle that one in such a way as to do it justice so stay tuned on that good question.

I realized after the fact that regarding stem cell epigenetics I should have said broadly that this is a process of regulation of gene expression.

Top 10 questions for stem cell field bouncing around on a Friday morning

top ten listWhat are the most interesting questions for the stem cell field in 2014 right now?

What’s on your mind?

I’m not going to give this deep thought, but rather just list those questions bouncing around at the moment in no particular order. You can see a lot of translational/clinical things are on my mind. I’ve put possible answers, but these are highly speculative and/or based on hope in some cases.

  • What’s next for ACT’s hESC-based clinical trials and Takahashi’s iPS cell-based clinical study for macular degeneration? I’m optimistic on safety for ACT and hopeful on efficacy, while it’s harder to predict for the iPS cell study.
  • How will ViaCyte’s new early hESC-based trial for Diabetes go? I’m cautiously optimistic.
  • In a year, where will the iPS cell IP/patent arena stand and who is BioGatekeeper? I expect no more clarity in a year overall, but BioGatekeeper will be outed by then or much sooner.
  • Will the trend of for-profit networks of stem cell clinics exploding across America continue? Unfortunately, yes.
  • Is the FDA planning to get back into the ballgame of regulating such stem cell clinics or has it punted due to (fill in the blank: budget problems at the agency, other priorities, a change of philosophy, etc.) Who knows? It’s been a disappointing year for the agency in this area.
  • Will the FDA apply Fast Track, Accelerated or Priority Review or Breakthrough Therapy Designation to any emerging stem cell products? It’s about time. They should.
  • What will the impact of the likely growing number of state Right To Try laws be? I expect it will increase both risk and innovation, but unfortunately much more of the former than the latter.
  • Was the flood of very high profile stem cell paper retractions so far in 2014 an aberration or will it painfully continue? I hope it does continue, but it’s a worry as the field needs public trust and respect.
  • Will a big pharma acquire a small biotech like Athersys, ACT, StemCells, Inc., Neostem, in the coming year? More money is sure flowing from big to small (witness ViaCyte’s new influx of money) and we saw this with happen California Stem Cell. I think we’ll see this trend of money flow or even acquisitions accelerate although there are structural obstacles to being acquired for certain companies.

What questions are on your mind right at the moment related to stem cells and regenerative medicine?

Note this post is simply informational and is not financial advice. Only make financial decisions after consulting with a certified financial planner, which I am definite not. I hold no stake in any of the companies mentioned.

Ask Me Anything on Stem Cells: Join me on Monday for Reddit AMA

Reddit Journal ScienceI did an “Ask Me Anything” or AMA on Reddit about 5-6 months ago on stem cells and the response was great.

There were so many wonderful questions and comments. I only wished I could have gotten to all of them. In total there were more than 1,100 comments. Yeah, people are really excited and interested in stem cells.

In that spirit of answer more questions, I talked with Reddit and we have a Stem Cell AMA 2.0 coming up this Monday, August 11. Of course a lot has happened in the stem cell field in the last 6 months as well.

I hope you’ll join us there for a great discussion and, per the name, ask me anything on stem cells.

Update: You can go to the link I included on Monday morning and you can start posting questions in the morning EDT. I’m planning to start answering questions and discussing things with people no later than 1PM EDT/10AM PDT.


Updated Patient Resource: The Top 10 Questions List to Bring With You to the Stem Cell Clinic

Stem Cell Patients Questions ListMany patients ask me what questions they should be asking doctors and clinics about possible stem cell therapies.

Here is my top 10 list of key items (in both graphic summarized form and detailed text form) that you need to know about and questions about a potential stem cell therapy.

Some of these questions are indeed probing, but that’s appropriate when you consider these folks may inject stem cells in your body, a therapy that may never go away completely. It’s not like a pill you can just stop taking. On the other hand, pills certainly have their own risks too.

Update: A hat tip to reader/commenter Barbara, who made the point, which I think is an excellent one, that no one doctor or clinic is likely to be able to answer all these tough questions. The list is intended as a helpful tool to get more info for patients as they are weighing risks and benefits, etc., not as a strict test that must be perfectly passed or anything like that.

I would also note that this same list could prove very helpful when a patient is considering enrolling in a clinical trial at a university hospital or similar center.

Bring this list with you to the clinic or email it to them and see how they do answering the questions about them and their products. Then talk to your own doctor with whom you have a long-term relationship. Below each item I have briefly explained the reason you should ask about this in italicized text (A big hat tip to Lee Buckler for the idea and I used some of his verbiage too for the explanations of why to ask! This post is still a work in progress….)

  • 1. Pre-clinical studies. How did the product do in preclinical studies? Did animals such as rats or mice who received the stem cells develop side effects? Did they show evidence of efficacy? How long did the transplanted cells stay in the body of the animals? Were the studies published? If there are no pre-clinical studies, why not?

Why ask about this? The goal here is to determine whether any pre-clinical studies were performed. If not, this raises a red flag. They may say there have been lots of studies performed on this cell type by other people but there is a high degree of likelihood that their product is slightly different than the one used in such studies (perhaps sourced differently, processed differently, etc) even though it is a similar type of product. Often we don’t know which of these changes may trigger a safety concern.

  • 2. Cell numbers & types. Just how many cells are in the product? Why was this cell number chosen? How were the cell numbers measured and with what accuracy? How many cells are alive versus dead? Might there be too few living cells to be helpful? What types of cells are in this product? How are the different cell types assessed? If it is a stem cell-focused product, how many non-stem cells are present? What does this mean for the therapy?

The goal with this line of questioning is to assess how well they know their product. It is not necessarily true that more cells equals better product but certainly if a provider cannot answer simple questions about what their product is made of every time this raises red flags.

  • 3. Methods & costs. How do you administer the treatment? IV? Local injection? How was this chosen? I understand that many clinics recommend more than one treatment, so on average, how many treatments do your patients receive and what is the total cost for each patient on average? How do you decide how much to charge? If the treatment does not work, will you refund my money?

This line of questioning is just being a smart consumer and being aware of how the clinic/doctor decided on the best method to give you the therapy.

  • 4. Pathogens, non-cellular components, and risk of xeno or cross contamination. Was the product tested for the presence of pathogens such as bacteria (and endotoxin, a potent poison made by bacteria), viruses, and fungi? Are the test results timely, signed and documented? Besides cells, are there are other things present like growth factors? What are these exactly? Were they added intentionally or are byproducts of the production process? Do they have functional properties such as growth factors? Were the cells exposed to animal antigens called “xeno contamination”? This can happen with growth of cells in fetal bovine serum or addition of synthetic growth factors from non-human species. If the product was exposed to animal materials, how was this matter addressed and documented? What steps does the clinic or company take to avoid cross contamination of one patient’s samples with another patients?

This line of questioning is really related to how much testing the provider has done to ensure the product they want to give you is safe. If they can’t address simple questions of safety test performed and data gathered, this raises red flags.

  • 5. Manipulations. How was the cellular product made? Why was this particular protocol used? Do the manipulations have the potential to change the cells? Is the product a drug? If so, is it FDA approved? If not, why not? Does the company have a relationship with the FDA (i.e. have had a dialogue).

This line of questioning is useful to try and help you determine (a) how safe the product it and (b) whether or not it is one that is regulated by the FDA or equivalent regulatory authority. The more manipulated a cell product is, the higher the risk is for contamination (so the bar is raised on those safety questions you asked earlier) and the more likely it is that it is regulated by the FDA and should be part of a formal clinical trial registered with the FDA or equivalent regulatory authority.

  • 6. Follow up. What is your clinic’s plan for following up on patients? How long do you do follow up and why was this period of time chosen? What is the plan if I develop a side effect or problem after the treatment?

This line of questioning is about (a) being a prudent consumer and (b) knowing whether or not the provider really cares about learning/knowing how patients react to the treatment or they just want to treat you and move on.

  • 7. Regulatory compliance status of business. Is the business compliant with applicable state and federal laws? Ask to see documentation of compliance.

For the companies that sell stem cells grown in culture, compliance is particularly relevant and important.

  • 8. Training and licensure. Is the physician or physicians who work there appropriately trained in stem cells and licensed? What was the training exactly and do you have documentation of the training? Does the doctor have training and expertise specifically in your disease?

You want a doctor who is truly a stem cell expert with years of experience and ideally experience as well in your specific disease state. For example, it might be riskier for you if you have a neurological condition like MS if you are being given a stem cell transplant by a physician who has no particular knowledge or training in MS or even the nervous system (e.g. a dermatologist or general surgeon).

  • 9. Data supporting the cellular product’s efficacy for this disease, injury or condition. What evidence is there that the stem cell or cellular product will work for the disease in question? Do you have publications? What are the alternatives to the proposed treatment? What are the strengths and weaknesses of the proposed treatment and the alternatives?

This line of questioning is also just about being a good consumer. Know what data exists out there to support why this might work. Make your own assessment with your personal physician of risk weighing the speculative nature of this treatment vs the potential risks including that you may be precluding other treatment alternatives.

  • 10. Data supporting its safety. What are the potential risks from the intervention in question? How have patients who have received this treatment fared in the past? Have any patients developed pulmonary emboli (clots in the lungs) or infections? Ask to see documentation of patient outcomes and if possible publications.

These questions are important because all medical treatments have risks and safety concerns. As a patient you want as much information as available to make decisions with your doctor about whether a particular treatment is the wise way to go and information about safety is just about as important as it gets.

There are more questions that can be asked, but with information on these top 10 items, we have at least gone a long way to assessing whether a specific stem cell-based cellular medicine product is appropriate for use.

Disclosure. This post is not medical advice.