Followup: Doctors should start human trials in earnest soon

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As reported last month, Drs. Maria Castro and Pedro Lowenstein, medical researchers at the University of Michigan, have embarked on an amazing journey to cure brain tumors through genetic engineering. They’ve graciously allowed The Ann to follow their work. If you have questions for the doctors, send an email to info@theannmagazine.com.

By Jud Branam

Dr. Pedro Lowenstein and his partner and spouse Dr. Maria Castro have won FDA approval to conduct the first phase of clinical human trials to fight deadly glioblastoma multiforme brain tumors in human patients.

First, however, come administrative details.

“It’s just getting everything to work in the particularly right way,” Lowenstein said in his German-accented English. “(The trials) will definitely start in late June or July unless anything happens to delay it.”

Castro and Lowenstein’s work has advanced further than any other gene therapy approach to brain cancer, but it has years to go before reaching the marketplace. It involves injecting cancerous areas with a highly tailored virus which can pass along payloads of genetic material but which has been stripped of its ability to make people sick. The new DNA then enters the system and inhibits the growth or recurrence of the cancer by either stimulating the body’s immune system to attack the cancerous cells or by killing off cells on which the tumor feeds.

The first step, which has been cleared by the FDA, is known as a “safety trial,” meaning that the medicine must be proven safe for use on humans before anything else occurs. That will take roughly 30 months before trials on the treatment’s efficacy can begin. However, the researchers expect to gain valuable insight into how well their treatment works during the safety phase.

U-M neurosurgeons will recruit volunteers for the trial from their existing cases. Finding willing patients is not expected to be a problem as the extremely aggressive GBM tumors yield a bleak prognosis and short survival rate, leaving many patients willing to try new approaches for themselves and for future patients.

Current activity focuses on planning and coordinating between the Castro-Lowenstein lab, U-M’s Department of Neurosurgery, the Human Applications Laboratory that is storing the vector viruses and the U-M pharmacy that will administer the doses. Once that’s complete, the couple, who have pursued an answer to this vexing cancer for 15 years with stops in England, California and now Ann Arbor, will hopefully see their quest move closer to reality.

Meanwhile, Lowenstein passed his math final exam – he’s both an undergrad math major and a professor of medicine – and he’s started summer classes.

Reader question

Reader Casey Quinlan  asked if Drs. Castro and Lowenstein are working on the same gene as in an experiment reported in The Telegraph, about “curing” mice of lung cancer with gene therapy.

As reported in that story, British researchers targeted a protein known as myc, which can lead to uncontrolled cell growth and the onset of cancer if it is overproduced by the body. Mice were given an antibiotic in their drinking water which activated a mutant gene known as omomyc, which blocked the production of myc.

Dr. Lowenstein called The Telegraph story “interesting,” but said the study in question is “completely different” from his work. “We are not working on the same gene. Our initial gene therapy will target adult brain tumors in which the particular gene cited in the question – omomyc – does not play such an important role.”


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1 Comment

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    July 17, 2014 at 12:04 am

    I would like to get tested on.