Grim tally from gene therapy / More on French Anderson (11/5/2005)

1.Investing in gene therapy, despite the latest deaths
2.More on French Anderson the 'father of gene therapy' - 'Who's Afraid of Designer Babies?'

Prof Joe Cummins: "The article below [item 1] provides a very strange metaphor. It quotes a biotechnologist as asking 'there's been an awful auto accident, is there a future for the transportation industry?'.

Comparing gene therapy to the transportation industry is very nutty. In spite of the billions pored into the industry it has never produced anything that works! Biotech is rather like the invention of a square wheel, it really does not work!

There seems to be a sickening philosophy among the gene therapy researchers that the death of a few children and injury of others is a small price to pay for large grants and high salaries among the elite researchers."

For more on the abuse charges faced by French Anderson [the subject of item 2] see:

Quote from French Anderson: "...we realised that, that we just didn't know enough about what was going on, that the body had reactions, had defences that we didn't really understand, and it really required going back and really rethinking everything we were doing." (item 2)

1.Under the Microscope again
Some investors show new interest in gene therapy, despite the latest scare
By Clare Kittredge
The Scientist [shortened]
Volume 19 / Issue 9 / 15 / May 9, 2005

The tally is grim: three high-profile cancer cases, including one death, in French boys who underwent pioneering gene therapy for X-linked severe combined immunodeficiency (X-SCID). Yet Edward Lanphier is upbeat about the future of gene therapy.

"It's a very, very broad field," says Lanphier, CEO of Sangamo BioSciences, a Richmond, Calif., biotechnology company focused on gene regulation. "It's sort of like saying there's been an awful auto accident, is there a future for the transportation industry? Is there a future for gene therapy? Categorically yes. Will companies pursue this from a commercial perspective? Categorically yes. And the outcomes in France in some of the SCID trials, as tragic as they are, are extremely informative."

Lanphire was reacting to the latest setback in a field that has experienced dramatic lurches in the last two decades. Gene therapy, once hyped as offering boundless promise by fixing faulty genes, has seen some dark days. Yet some financial experts observe renewed investor interest in the field, and many scientists and CEOs say the technology is now too diverse to paint with the same broad brush.

"Gene therapy is not gene therapy is not gene therapy," says Rich Gregory, head of research for Genzyme in Cambridge, Mass. "Of course the French case has an impact because everybody understands these are issues associated with gene therapy, but from a pure technological point of view, many companies would say it does not relate to what they're doing."


Hopes for gene therapy soared in 2000 when Marina Cavavazzana-Calvo and Alain Fischer of the Necker Children's Hospital in Paris reported successfully using gene therapy on children afflicted with X-SCID. However, a boy treated by Fisher was diagnosed with a leukemia-like cancer in September 2002, and a second boy was diagnosed three months later. One of the boys died in October 2004, and a third was diagnosed in early 2005. Other children who are now free of X-SCID because of the treatment, including some in France and the UK, are being monitored. In late January, the Food and Drug Administration suspended three similar gene therapy trials in the United States.

Jean-Yves Bonnefoy, chief scientific officer of Transgene, a biotechnology company based in Strasbourg, France, says his company has largely moved away from gene therapy, although the move pre-dates the French cases and has more to do with seeking a larger market. "The problem with gene therapy is the targeting and delivery vehicle," say
Bonnefoy, worked with Fischer in the early 1990s. "How can you make sure it targets only the cell you want?"

Other high-profile gene therapy cases have not helped, such as the death of teenager Jesse Gelsinger in a 1999 trial at the University of
Pennsylvania. Some experts say his death was due to the high number of viral particles infused during the trial, rather than the gene therapy itself.

All this has investors cautious about betting on gene therapy.

Navdeep Jaikaria, director of equity research for the New York City investment firm of Rodman & Renshaw, says companies targeting a narrow spectrum of the field may be successful, although gene therapy has "definitely fallen out of favor with investors."

"You can measure that by the value gene therapy companies are enjoying compared to other similar-stage biotech companies. They are trading as a
group at a 30% to 40% discount," Jaikaria says. "Avigen recently announced that it is discontinuing its gene therapy program."

However, the situation could change rapidly. "Any success can bring investors back. We like companies addressing unmet medical needs like cancer and cardiovascular disease, because the risk-reward profile is such that the FDA is willing to look at novel therapies," says Jaikaria. "We view gene therapy to be no different from any other drug delivery platform, but there are questions which keep investors concerned."

Jaikaria says companies focusing on short-term gene therapies show commercial promise. "There are two kinds of gene therapy, if you can say that in a simplistic manner. One is you put a gene into a cell and it doesn't integrate with the genome of the individual, and that's what GenVec and Coratus [Genetics] and several other companies are trying to do. The other form of gene therapy is trying to correct a deficient gene in a cell, and we don't really see that kind of gene therapy succeeding
commercially in the next five to 10 years. But GenVec, Coratus, Targeted Genetics, Sangamo – we think they have promise and their programs can succeed."

Genzyme's Gregory says the French clinical trials have caused a public-relations problem for gene therapy in general, and they have affected small companies' ability to raise money. "It's tough for companies in the gene therapy space to raise additional funds because of the general impression in the venture capital community that gene therapy is not the place to be," Gregory says. However, the trials have had less impact on Genzyme, which has a long-term commitment to gene therapy and still sees it as a therapy of the future. "For us, it's been steady, hold-the-course, but I can see there are certainly fewer companies in the gene therapy space than there used to be, and my impression is it's tougher for those companies."

2.Who's Afraid of Designer Babies?
BBC TV: Horizon
*excerpt from programme transcript*

French Anderson has been hailed as one of the most remarkable scientists of our time. He pioneered what claimed to be the biggest revolution in modern medicine, treating genetic disease by inserting healthy genes into patients. It's called gene therapy.

Dr FRENCH ANDERSON: ...The key cells that we really want to get genes into are the so-called blood stem cells, because these are the cells which continue to repopulate the blood system for our whole lives. Some of our blood cells only last a few hours, some last a few days, some last a few weeks. But basically they're constantly turning over, so we need to get genes into that original stem cell that will, that will, that reproduces the bloo system.

NARRATOR: Anderson thought he might be able to get i

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