Ark, Troubled Waters, and Rainbows for Gene Transfer

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This morning I awoke to a news report by National Public Radio’s Joe Palca on promising developments in gene transfer. In it, Palca provided a good account of the field’s travails, as well as some encouraging developments in the last few years. The story ended with the prediction that the coming “months and years” would bring landings for more common disorders like AIDS and cancer.


Coincidentally, the just released March issue of Nature Biotechnology ran a report on a front-runner for gene transfer commercialization: biotechnology company Ark Therapeutics gene transfer gliobastoma product Cerepro. The application for licensure of this product in Europe was unsuccessful (press release here). Recall that, last June, I described what seemed like unimpressive results from a phase 3 trial that were reported at an annual meeting of the American Society of Gene Therapy. Apparently, European drug regulators weren’t impressed either (they cited flaws in trial design, including a small sample size and unconcealed allocation; Ark has asked the agency to re-examine their application).

But for those awaiting the first commercialization of a gene transfer product in a country with a robust drug regulatory system, there is still some indication that the rains may be subsiding: according to the report in Nature Biotechnology, Amsterdam Molecular Therapeutics has filed with EMEA for marketing authorization of their AAV product for a rare hereditary disorder, LPL deficiency; the company will soon file in Canada as well (the disorder is more prevalent in Quebec) (photo credit: Occhiovivo 2007)

BibTeX

@Manual{stream2010-70,
    title = {Ark, Troubled Waters, and Rainbows for Gene Transfer},
    journal = {STREAM research},
    author = {Jonathan Kimmelman},
    address = {Montreal, Canada},
    date = 2010,
    month = mar,
    day = 8,
    url = {http://www.translationalethics.com/2010/03/08/ark-troubled-waters-and-rainbows-for-gene-transfer/}
}

MLA

Jonathan Kimmelman. "Ark, Troubled Waters, and Rainbows for Gene Transfer" Web blog post. STREAM research. 08 Mar 2010. Web. 27 Jun 2019. <http://www.translationalethics.com/2010/03/08/ark-troubled-waters-and-rainbows-for-gene-transfer/>

APA

Jonathan Kimmelman. (2010, Mar 08). Ark, Troubled Waters, and Rainbows for Gene Transfer [Web log post]. Retrieved from http://www.translationalethics.com/2010/03/08/ark-troubled-waters-and-rainbows-for-gene-transfer/


ASGT in San Diego

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This year’s annual meeting of the American Society of Gene Therapy is in San Diego.  I’ve been to several interesting talks thus far, and plan to post entries on a few. For now, here’s an overview of some major (or some not so major) clinical developments in gene transfer that are being reported at this meeting.

1- Last year, I predicted that the first gene transfer applications were nearing licensure.  Not so fast.  Because of concurrent sessions, I was unable to attend the entire talk given by Robert Shaw of the British biotech company Ark Therapeutics.  Ark has developed a gene transfer approach, Cerepro, that uses adenovirus to treat malignant glioma (which is one of the most aggressive types of cancer).  Ark recently applied to the European drug regulatory authority, EMEA, for registration of Cerepro.  Why not FDA? Dunno (though the speaker stated that the review standards are more or less the same).  The data behind the product are less than earth shaking.  According to information available over the web [proviso- these data are from August 2008], the pivotal phase 3 study of Cerepro showed only a 42-day increase in survival for patients in the active drug arm.  And the product caused “increases” in hemiparesis, aphasia, and fever.

2- Another somewhat discouraging indication of the challenges in reaching licensure for gene transfer products was a session titled “late stage industry clinical trials.”  To me, late stage means phase 3.  But three talks centered on phase 1/2 studies, and none presented phase 3 results.  The first talk was given by Ceregene on their Parkinson’s disease product Cerepro. The product did not show any significant advantage over sham for their primary endpoint.  

3- Last year, the “buzz” at ASGT was the preliminary results from three studies testing AAV vectors for a form of congenital blindness, LCA. I also discussed the somewhat ethically controversial decision to move this study into children.  I will look forward to attending Jean Bennett’s talk on Friday; her abstract reports that her LCA study has enrolled “9 children and young adults” ranging from age 8 to 26 years.  The abstract claims improvement in “subjective and objective” measures of vision.  To be continued… (photo credit: slack12, 2008)

BibTeX

@Manual{stream2009-99,
    title = {ASGT in San Diego},
    journal = {STREAM research},
    author = {Jonathan Kimmelman},
    address = {Montreal, Canada},
    date = 2009,
    month = may,
    day = 28,
    url = {http://www.translationalethics.com/2009/05/28/asgt-in-san-diego/}
}

MLA

Jonathan Kimmelman. "ASGT in San Diego" Web blog post. STREAM research. 28 May 2009. Web. 27 Jun 2019. <http://www.translationalethics.com/2009/05/28/asgt-in-san-diego/>

APA

Jonathan Kimmelman. (2009, May 28). ASGT in San Diego [Web log post]. Retrieved from http://www.translationalethics.com/2009/05/28/asgt-in-san-diego/


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