New Gene Therapy — Hype for Funding?

Nice articles, here and here, on a new appoach to chronic leukemia. In a phase 1 study (study to evaluate toxicity) the approach seems to have activity.

Two participants in the Phase I trial have been in remission for up to a year. A third had a strong anti-tumor response, and his cancer remains in check. The research group plans to treat four more patients with CLL before moving into a larger Phase II trial.

Just 3 patients have been treated. That there is evidence of efficacy is hopeful but certainly not conclusive.

It should also be noted that:

All of the funding for the University of Pennsylvania’s gene therapy work has come from the academic community, but the work is expensive.

“We are looking for corporate partners as we head into Phase II trials,” Kalos said.

Medicynical Note: The study does give hope for a new approach to refractory leukemias, and it may well be applicable to other tumors.

The funding for this study, the basic research and initial phase 1 trial in all likelihood came from public money, federal grants and such–“from the academic community.” As implied, the researchers are courting corporate sponsors to fund further studies.

In our “system” you and I fund the training and research of basic scientists and their advances and then allow, and, yes, even encourage them to sell their advance to industry for commercialization. If the drug is useful, even marginally so, we get to pay the inflated price demanded by Pharma for a generation (the patent protected time period), with no credit for the funding of the initial advance.

It heads you lose, tails they win.

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