Pink Sheet is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By



Executive Summary

SOMATIX GM-CSF/GENE THERAPY RENAL CANCER TRIAL CLEARS RAC during the National Institutes of Health's Recombinant DNA Advisory Committee meeting March 1. The protocol, submitted by lead investigator Jonathan Simons, MD, Johns Hopkins University, is a randomized Phase I dose-escalation study which seeks to treat metastatic renal cell cancer patients with autologous tumor cell injections of cells transduced with the coding sequence for granulocyte-macrophage colony stimulating factor. The protocol employs a novel viral vector (MFG) provided by Somatix Therapy Corp. Somatix plans to submit an IND for the trial, the company's first, within the next few months. "Although our initial target is renal cancer, we believe that this treatment may be effective in fighting many other cancers," Somatix said March 2. "It is believed that the genetically modified cells may be able to stimulate the immune system" to recognize tumor cell antigens "resulting in the elimination of tumor cells with these same antigens throughout the body." RAC reviewed a total of 10 protocols during the March 1-2 meeting. Two cystic fibrosis trials, one using vectors supplied by Genetic Therapy, Inc., were unanimously approved. Two trials proposing to alter bone marrow with the multiple drug resistance (MDR) gene in an attempt to reduce myelosuppression associated with Bristol-Myers Squibb's Taxol were deferred by the committee. Also deferred by the committee was a study proposing to transduce malignant melanoma patients' tumor cells with coding for gamma interferon using vectors supplied by Viagene. The next RAC meeting is scheduled for June 7-8. Three other gene transfer protocols using GTI vectors were approved by the committee. One, sponsored by Kenneth Culver, MD, University of Iowa, is similar to an ongoing glioblastoma trial sponsored by GTI. Two gene marking trials to be conducted by St. Jude Children's Hospital researcher and GTI collaborator Malcolm Brenner, MD, were also cleared by RAC. On March 2, the committee adopted a 10-point procedures guide for expedited review of gene therapy protocols. The proposal will be forwarded to NIH Director Bernadine Healy for publication in the Federal Register. The committee's final draft of the document retains the substance of the policy originally endorsed by RAC in January: the same standard used by RAC in its reviews should be applied to expedited reviews ("The Pink Sheet" Jan. 18, T&G-2). RAC developed the policy after a compassionate use approval was granted for gene therapy treatment of a San Diego glioblastoma patient by NIH and FDA ("The Pink Sheet" Jan. 4, T&G-4). The sponsors of the San Diego protocol, San Diego Regional Cancer Center researchers Robert Sobol, MD, and Ivor Royston, MD, were asked by RAC to submit the information used by FDA and NIH to grant the compassionate use approval for the March meeting. Although the committee did not vote on the issue, several RAC members declared that they would not have voted to support approval of the single-patient trial based on the information submitted.

You may also be interested in...

Part D Discount Liability Coming Into Focus: CMS Releases Drug Cost Data

Newly released Medicare Part D data sheds light on the sales hit that branded pharmaceutical manufacturers will face when the coverage gap discount program gets under way in 2011

FDA Skin Infections Guidance Spurs Debate On Endpoint Relevance

FDA appears headed for a showdown with clinicians and the pharmaceutical industry over the proposed new clinical trial endpoints for acute bacterial skin and skin structure infections, the guidance's approach for justifying a non-inferiority margin and proposed changes in the types of patients that should be enrolled in trials

Shire Hopes To Sow Future Deals With $50M Venture Fund

Specialty drug maker Shire has quietly begun scouting deals with a brand-new $50 million venture fund, the latest of several in-house investment arms to launch with their parent company's pipelines, not profits, as the measure of their worth




Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts