What’s the first rule of medicine again? Do no harm!
Rushing new medicines such as stem cell treatments to patients without appropriate preliminary studies to try to make money is like playing Russian Roulette with patients. Some of the time, everything will turn out OK, but other times….look what happened with thalidomide.
There are two main camps in the world of stem cells.
There are the folks, like me, who generally favor a relatively higher degree of FDA regulation of stem cell treatments, particularly for cells grown in a lab prior to transplant, due to safety concerns.
Then there are the rushers.
These are the folks who argue that FDA regulations are over the top, stifling innovation and slowing the transition of stem cell treatments to patients.
My argument for a relatively high degree of regulation is that from all my years of experience with growing cells and in particular stem cells, I don’t believe we know enough to safely treat patients with stem cells expanded in a lab without a rigorous clinical trial context. There are serious, potential dangers to stem cell therapies using “expanded” cells grown in a lab and we are still learning every day more about them.
Legitimate companies such as Advanced Cell Technology (ACT) and others expand their stem cell as well, but the key difference is that ACT is following FDA rules and rigorously has tested their stem cell products. Based on their pre-clinical studies and now their early clinical trial data, there is growing reason for realistic hope that ACT’s therapy for blindness is indeed safe. I’m a big fan of ACT these days!
The same cannot be said for the treatments of dubious, for-profit stem cell clinics.
Dubious clinics break the rules and generally try to hide from the FDA. They don’t test their products in pre-clinical studies before using them on patients, making their patients unwitting guinea pigs.
Should, as the WSJ recently speciously argued, these for-profit clinics that have not carefully pre-tested the safety of their grown stem cell treatments be allowed to operate without rigorous oversight?
Don’t believe or agree with me?
Read on….about the drug thalidomide. It’s a fascinating, if heart-wrenching story in what can go wrong with rushing innovative medicines and has eerie parallels to where we are today with dubious stem cell treatments (drugs).
There are heroes and villains.
About 50 years ago, the drug thalidomide was developed in Germany by an ex-Nazi scientist.
Despite its somewhat strange origins, it showed great promise as a drug for many ailments ranging from insomnia to morning sickness.
The potential of thalidomide seemed broad indeed, much like stem cells. Drug companies saw an opportunity to make a very large amount of money and pushed very hard for the widespread use of thalidomide even though relatively little was known about the chemical.
Akin to stem cells, thalidomide in the early days was hailed as a “wonder drug” reportedly leading to miraculous, positive outcomes in patients.
All of this was happening in Europe, but not in the U.S.
You see, thalidomide was barred from clinical use in the U.S. by the FDA even while it was being prescribed in Europe.
In particular one FDA pharmacologist, Dr. Frances Oldham Kelsey, M.D., expressed concerns that “further studies were needed” prior to its approval and use. In fact, she had to fight political pressure from a drug company that wanted to sell thalidomide in the U.S….reminds me of clinics pressuring the FDA to relax regulation of grown stem cells.
To say she was right is a tremendous understatement. JFK recognized Dr. Kelsey with the President’s Award for Distinguished Federal Civilian Service. See image above near top of the post from Wikipedia. I think she’s a true hero.
Thalidomide use in Europe was found to be disastrous over time.
You see, it caused birth defects in women who took it, a particularly horrific side effect given the use of the drug specifically in so many women with morning sickness and as a sedative during pregnancy, when sleep can be elusive.
Up to 20,000 children were born with birth defects conclusively due to thalidomide use by their mothers. As such thalidomide is classified as a teratogen. Intriguingly, scientists to this day still do not know conclusively how thalidomide causes birth defects, despite extensive study. This is a powerful lesson in humility about just how well we can think we understand something in the biomedical field, when in reality we have so much more to learn over time with further study.
Interestingly, we are now realizing that thalidomide may indeed have some very beneficial uses for other conditions such as for treatment of a variety of cancers in non-pregnant women and in men, and the drug is still being intensively studied 50 years after it first went into wide use without appropriate regulatory oversight in Europe.
Another lesson from the disaster of thalidomide in Europe was that the FDA sticking to its guns saved untold suffering and heartache in the U.S.
I can’t help but wonder if the same might be true of grown stem cells pitched by dubious clinics.
Maybe they are safe…but maybe not….or maybe they are safe only in certain patients but not others.
Grow stem cell treatments seem likely to be wonderful and to have many potential uses, but as Dr. Kelly said about thalidomide a half a century ago, I believe that when it comes to grown stem cells, “further studies are needed” beyond what dubious for-profit clinics assert.
I commend companies like ACT who do their homework, do the pre-clinical work, and perform the necessary thorough studies before treating patients….before trying to make money. There’s nothing wrong with trying to make money for a biotech company. In fact that is key to their continued existence and for helping patients, but the science should come before the money.
Finally, even if dubious for-profit stem cell clinics are now offering the unapproved cells in Europe (just as Europe forged ahead unwisely to treatment tens of thousands of women with thalidomide) to patients, it doesn’t mean the FDA should cave and let grown stem cells be used in a non-regulated manner in the U.S.
Even if politicians or businesspeople are pressuring the FDA to relax on stem cells, the FDA should do what is right.