3 Dangerous Texas Bills Would Boost Stem Cell Clinics

Texit stem cells

The Calexit and Texit state secession campaigns for California and Texas to leave the union, which are linked to Russian President Putin, are never going to be successful. However, if some Texas lawmakers and stem cell clinics there have their way, Texas would take a big step away from the rest of us on the stem cell front, endangering patients. Such a development would strongly contrast to all the great, cutting edge stem cell research going on in labs across that state. Somehow this major development has not been covered yet by national or even Texas media.

What’s the scoop?

Three bills are pending at the Texas Capitol that if passed and signed into law would pave the way for unproven, risky stem cell therapies to be sold much more readily to patients by clinics. The Texas stem cell bills include HB 661 and HB 810 by Rep. Tan Parker, and HB 3236 by Kyle Kacal. You can learn more about the bills by following direct links to each bill here, here, and here.

HB 661 seems to be a very loose kind of right to try effort that concerningly would extend it from restricted just to patients with terminal illnesses to also those with chronic conditions that could be just about anything. In a sense, a stem cell clinic’s own doctor perhaps could decide whether their patient/customer has a chronic disease that is eligible. How often would the clinic doctor say “no” since that would mean the patient would not get the treatment and so would not pay them big bucks?

Stem cell cartoon

HB 810 is a stem cell-specific kind of right to try bill that would greatly lower oversight standards and put patients at greater risks. The third bill, HB 3236, is what I call “Right to Profit” for the clinics because if that bill passes then the clinics would have free rein to make millions in profits from vulnerable patients. How would that be a good thing for most Texans? It wouldn’t. In fact, I see it as a consumer ripoff bill.

Other than stem cell clinics, it’s hard imagine many fans of these bills. Most people I have talked to strongly oppose them including top stem cell scientists in Texas. The organization Texans for Cures, which has been very balanced, sensible and supportive of stem cell-based regenerative medicine for many years, strongly opposes these bills too. Here’s a statement from its Chairman David Bales:

“After careful examination of HB 661, HB 810 by Rep. Parker and HB 3236 by Texans for Cures Medical Advisory Committee, which includes leaders like Dr. Doris Taylor and Dr. William Decker, we decided to vigorously oppose all three bills because they jeopardize patient safety and responsible research in the State of Texas”.

There’s broader opposition too. For instance, the largest global stem cell research organization, ISSCR, is opposed to these stem cell bills. You can read more about ISSCR’s viewpoints in a letter from its President Sally Temple to Texas lawmaker Todd Hunter. Here’s a big picture quote from the ISSCR letter:

“…these bills will allow snake oil salesmen to sell unproven and scientifically dubious therapies to desperate patients.”

What businesses exactly would stand to benefit mostly at the expense of patients? Continue reading

What is hardest thing we do with our brains in a lifetime?

brainatwork

Wikimedia open image

What’s the hardest thing we ever do with our brains on average during a lifetime?

Our brains are wonderful things that some have called the most complicated structures in the known universe, but what exactly do we do with them? Do we tax them fully during our lives?

The old cliché that we only use 1% of our brain or some approximation thereof is malarkey in my view, but how hard is it for the average person’s brain to watch a boring YouTube video? Not very. On the flip side, what do we do with our brains that is incredibly hard? The hardest thing for the average person in their life? Continue reading

Big change for stem cell field with FDA quickly issuing RMATs

The 21st Century Cures Act has some important regenerative medicine language in it. One part of that was attempting to accelerate FDA review of promising investigation regenerative medicine therapies. The mechanism for this was a new designation called Regenerative Advanced Therapy, now renamed Regenerative Medicine Advanced Therapy (RMAT) Designation.

I don’t think anyone was sure how this would play out in terms of how quickly the FDA would give RMAT designations and to how many investigational products. Already it’s clear the FDA seems to be embracing the spirit of the Cures provisions as it has given out quite a few FDA granted RMAT designations and done so quickly.

The first that I could see was given to Humacyte, which has an artificial vessel research program (see image). The artificial vessels become colonized with endogenous stem cells after implantation.Humacyte

Keep in mind that RMAT designation does not equal FDA-approval overall, but it should mean the investigational therapy has solid data behind it and is promising, together equaling a faster FDA review and then potentially in the future approval should future data be strong. If some of the RMAT therapies are ultimately proven to be safe and effective, and via RMAT they were able to get to patients and help them more quickly, with few failures amongst the RMAT group, then the RMAT regulatory experiment will be proven a big success.

Here are the approved RMATs I could find so far:

  • Humacyte (Vascular Access for Hemodialysis)
  • Enzyvant (DiGeorge syndrome)
  • jCyte (Retinitis Pigmentosa)

There may be more already given, but not yet in the public domain. If you know of others please comment on the post to let us know and I’ll add them.

Burt Northwestern Stem Cell MS Trial Part 3: Funding, Patient Perspectives, & the Future

Today’s post is the last of a three-part series on the Burt stem cell trials at Northwestern. This piece is focused on funding and the future of the trial, and also includes perspectives from patients.

You can read Part 1 here and Part 2 here, which focused on issues related to potential encouragement of patient fundraising that unintentionally releases private information and on concerns over marketing of the ongoing trial as already known to be something like curative for MS. I’ve summarized my overall concerns in a flow chart below.

Stem cells for MS trial concerns

Trial funding

Should patients ever be required to scrape together one or two hundred thousand dollars to pay to get enrolled in any clinical trial? We have to keep in mind that in such trials patients have no guarantee of any benefits and could be harmed because the patients are research subjects. And what happens to the potentially large number of patients who cannot obtain that kind of money? They are excluded simply on that basis?

There may be certain instances where patients paying for a trial can be a responsible, positive thing. However, this is new territory overall for stem cell clinical research and there are sizable risks to this different kind of approach as compared to the past and to some other more traditional clinical experiments when sometimes patients were the ones receiving some kind of payment. As I discussed earlier in this series, extremely careful planning is needed to address bioethical and practical issues that may arise and anticipated risks to patients in such trials where patients have to contribute monetarily.

Some patients have said on the Internet that they are required to pay as much as $150,000 for participation in the Northwestern MS stem cell trial or in parallel off-study experimental administration of experimental stem cells.  If we estimate that about 100 patients have been required to pay (one way or another, such as themselves or via insurance or via online fundraising) on average $100,000 then that is in total $10 million for Northwestern University. Not a small sum, but clinical trials are expensive. If there have been 200 patients paying that much on average the total number could swell to $20 million. The big money involved here is just one more reason that NW and its IRB need to be extra careful in how it oversees this trial.

Do trial participants get any kind of discounted price on medical tests and procedures? Does the university cover all the research-specific (versus treatment) costs itself? How much does insurance pay for the average participant? How much does philanthropy by NW help the average patient? These same questions could be asked of any other trials that require patient payments as well, but there seems to be little if any information that I could find in the public domain on other stem cell IND-based trials that require large patient payments. Continue reading

President Mills Leaving, CIRM Needs New Leader to Navigate Future Challenges

Randy MillsCIRM announced today that its President and CEO, Randy Mills, is soon leaving for a new job as President of the National Marrow Donor Program/Be The Match in Minnesota. Update: Dr. Maria Millan, the CIRM Vice President of Therapeutics, will be its leader starting July 1 until a new leader is chosen.

For this kind of position three years is a relatively short tenure so CIRM will need to scramble a bit to keep continuity and momentum as it searches for and ultimately puts in place a new leader. It’s a critical time for CIRM as it and its allies consider big picture strategy for the future, approaches to future funding such as a possible new proposition for state funding (Prop 71 2.0), and how to continue all those exciting clinical trials and research beyond the current period of its funding.

In general, Mills had a big positive impact on CIRM and helped it go to the next level. About the only thing I wasn’t a fan of in terms of his leadership was my perception of his negativity toward the FDA and toward FDA oversight of stem cells, and how that manifested at CIRM during his time there. But good people can strongly disagree on policy. We’ll have to wait and see how the regulatory experiment of stem cell provisions in the 21st Century Cures Act, which Mills may have helped to make possible, will impact regenerative medicine in terms of changes in FDA oversight. It could also impact CIRM too.

Now CIRM’s Board has an exciting, difficult task ahead. Who do they want as their new leader to tackle CIRM’s challenge? What kind of background and future vision? The priorities, leadership skills, and vision of the new leader are likely to together be a major factor in CIRM’s future success. Who are the top possible candidates out there right now? I’m going to do a follow-up, future post on these questions and CIRM’s future.