Gene therapy (and stem cell) triumph – at last

After many years of slow progress and some highly publicized setbacks, gene therapy finally seems to have its first major success, reported this week in the New England Journal of Medicine.

Gene therapy uses genetic techniques to insert genes into a person that correct a genetic disease. For example, a “fixed” version of a flawed gene can be inserted, with the hope that the fixed gene can somehow take hold and provide a cure. The problem has long been that, even if you can create cells with corrected versions of the gene, inserting those cells into a person leads to, at best, just a temporary fix. The cells eventually die and the person is back to where they started. Creating a long-lasting cure has been elusive, until now.

In this new study, Alessandro Aiuti and colleagues (from the University of Milan and other institutions) are using gene therapy gene to treat severe combined immunodeficiency (SCID), a usually fatal disorder in which the immune system is severely crippled by the lack of an enzymme called adenosine deaminase (ADA). Children with SCID are sometimes called “bubble children” because they have to live in a sealed environment to prevent infections.

Aiuti et al. created bone marrow stem cells (the cells that produce blood cells) for the SCID children in which the ADA gene was fixed. They then injected these cells back into the children, and hoped that the cells would “take”; i.e., that they would become stable parts of the children’s bone marrow. So far (the trial isn’t yet over), 8 out of 10 children have had successful results: their blood cells are producing ADA on their own, and they don’t have life-threatening side effects. More important, these children can now lead a normal life – they don’t have to live in a “bubble.”

These results are still preliminary, but they look very, very encouraging. Let’s hope this is just the first of many successful gene therapy and stem cell therapy cures.

Help me get NCCAM defunded

Today I received email from the co-chair of Obama's presidential transition team asking me to contribute suggestions on what the new administration should do to improve the country. I know it's a mass email, but I decided to give it a shot, and I'm asking readers of this blog if they can help in a small way.

I've been calling for NIH to shut down NCCAM for a while now. Many others have tried to shut it down as well, including the previous NIH Director, Harold Varmus, who is now on Pres-elect Obama's scientific advisory council. It's a great way to save $225 million (or more), which could be put back into the NIH budget to fund real science instead of pseudoscience.

I submitted this suggestion on the Obama team's website, and you can help me by voting in favor of it. Here's the link. To vote, you'll need to create an account and login, which takes only about 30 seconds.

Within 10 minutes of my posting this suggestion (under Health Care), someone had already voted against it, so I need your support! (10 minutes later someone else voted for it, giving it a net vote of 0 points.) I noted that many of the suggestions under this topic were proposing just the opposite - that Obama should increase funding for alternative medicine, should allow insurance to cover it, etc. We need to at least show the incoming administration that some of us believe that the government should only support evidence-based medicine, not pseudoscience.

UPDATE: Saturday, Jan. 17. As of this morning, voting on my proposal passed 11,000 points! But please go and vote if you haven't already - there are a growing number of people voting "no" on the proposal, leaving comments describing all kinds of quackery that these people insist really work. The vote total is a score - each vote "for" adds 10 points, and votes against deduct 10 points.
Thanks to 2 bloggers at scienceblogs.com, PZ Myers (Pharygula) and Orac (Respectful Insolence) for pulling in such terrific support of my proposal. And thanks also to the bloggers at Science-Based Medicine for their post, which just went up today.

I'm on YouTube: my talk on autism and vaccines

I gave a talk recently on the pseudoscience surrounding autism and vaccines, and on the poor job that the press does presenting the science around this topic.  The good people at NCAS posted the whole talk (almost) on YouTube.  My talk title was "Autism and Vaccines: How Bad Science Confuses the Press and Harms the Public," and it's in five parts of about 8-9 minutes each:

Part 1 is here

and all 5 segments are linked here, on the NCAS (National Capital Area Skeptics) YouTube page.

The first segment already has some anti-vaccination comments on it, which I guess is not too surprising.  There are a couple of supportive comments as well.

I wrote "almost" above because my talk included two video segments that NCAS didn't post due to copyright concerns.  One was a 60 Minutes interview with Andrew Wakefield, about 5-6 minutes, and the other was a segment where Larry King interviewed a group of anti-vaccine zealots.  The Larry King bit can be found elsewhere on YouTube.  

meditation, ADHD, and bad reporting

There's an excellent discussion over at Space City Skeptics that I wish I'd written myself. In it, Skepticpedi illustrates both the credulous reporting of the media on science, and a particularly poor study that was just reported. I say "reported" because it wasn't really published - the report was based on an article in an online education journal edited by graduate students.

What did the study (and the article) claim? Just this: that Transcendental Meditation (TM) was beneficial in the treatment of childhood attention-deficit hyperactivity disorder, also called ADHD.  The study did almost everything wrong - in fact, if you wanted to illustrate what *not* to do in a clinical study, this one would provide a wealth of examples:

1. There were only 10 subjects, too few to make any statistically valid conclusions.
   
2. There was no control group - all 10 subjects (children) were told to try TM techniques. By the way, TM basically consists of sitting and chanting a nonsense word to yourself, over and over.
   
3. The results were based purely on self-reporting by the teachers.
   
4. There was no "blinding" - all the teachers knew what the study was trying to show.
   
5. The students were probably coached in what they were supposed to say.
   
6. The headmaster of the school (all the students were at the same school) is a strong proponent of TM, and is on the board of the foundation that funded the study!
   
7. The "scientist" (sorry, have to put that in quotes) who did the study is also on the board of the foundation that funded it. Yikes!
   

So basically, we have a small, terribly-designed, self-funded study by a proponent of TM who claims that she's produced evidence that TM helps treat children with ADHD. What's surprising here is that Reuters Health news service wrote a report on this (as did other news organizations, apparently). And by the way, the person who ran the study, Sarina Grosswald, has a strongly self-promoting website where she claims that she "recent directed a landmark research study" - this one! - and trumpeted the fact that it was widely reported in the media.  Calling your own study a "landmark", especially when it was self-labeled a "pilot" and published in an obscure journal, really takes some chutzpah.

So here's a big raspberry to Reuters for terrible medical news reporting. They can do better. And a big thumbs-up to Skepticpedi for calling them on it.