Sutent appears headed for approval
By Norman Scherzer
Each month we try to find out the latest news about clinical trials for GIST, a process that sometimes feels like describing the latest layout of chairs on the Titanic. As the relentless spread of GIST resistance claims an increasing number of lives, the need for a drug that repeats the initial miracle of Gleevec intensifies. So, too, do the conflicts inherent in clinical trials — where drug companies seek a proof of concept that wins government approval and finds a subsequent market niche that’s competitive, and what GIST patients need to survive.
Sutent: To date there is one post- Gleevec drug that seems to be heading for FDA approval — SU11248 or Sutent (formerly called Sugen). This Pfizer drug has completed its initial phase III trial and is now available in a growing number of clinical trial locations as a treatment protocol no longer requiring a placebo. Preliminary data has shown that Sutent helps a substantial number of GIST patients whose cancer is resistant to Gleevec, generally by slowing down the progression of disease.
Interestingly, Sutent seems to work best among GIST patients who don’t have an exon 11 mutation — the opposite of Gleevec, which works best on patients with an exon 11 mutation.
To improve access to Sutent, Life Raft representatives met with their counterparts at Pfizer and its contractor, Emerging Med, which manages access to its clinical trials, at the annual meeting of the American Society of Clinical Oncology. A special Emerging Med liaison and phone number has been created to help GIST patients and caregivers via the Life Raft Web site (see www.liferaftgroup.org/ treat_trials_su11248.html) or call the Life Raft office at 1-973-837-9092).
A pleasant surprise at this meeting was the willingness of Pfizer to open Sutent access to pediatric GIST patients under age 18 on a special need basis. The same Emerging Med liaison person will provide pediatric GIST families with help in getting Sutent.
As we go to press, we learn how important this assistance will have to be. A pediatric GIST family trying to get Sutent was unable to do so at a clinical trial site at major medical center because the trial physician was not comfortable treating a young GIST patient. The family is now searching for an alternative location.
AMG706: Coming out of the clinical trial starting date a little later than Sutent is AMGEN’s AMG706. This phase II trial is close to its enrollment target of about 100 patients.
Although there is no published data yet, the Life Raft has anecdotal reports that this drug is active. Some GIST specialists opine that this drug will prove to be as effective as Sutent, perhaps with fewer side effects. The challenge AMGEN faces is whether it can compete with Pfizer and win government approval. The challenge that GIST patients may face is whether they will be able to retain some access to AMG706 once clinical trial enrollment closes. (Note: after surveying a dozen GIST specialists in the United States and abroad, the Life Raft found an interest and need for continued access to AMG706 after the clinical trial enrollment closes. This was reported back to AMGEN.)
NEW PHASE II TRIALS
Two new phase II trials have begun enrolling patients with Gleevec- resistant GIST.
Gleevec and Perifosine: Perifosine (KRX-0401), made by KERYX Biopharmaceuticals, is an oral drug that inhibits AKT, an anti-apoptosis protein. Apoptosis is a form of controlled cell death, a type of cellular suicide. A phase II trial is now seeking to enroll GIST patients at M.D. Anderson Cancer Center in Houston, Texas.
CCI-779: Made by Wyeth Pharmaceuticals, CCI-779 is an intravenous mTOR inhibitor. A phase II trial is now seeking to enroll patients at the Mayo Clinic in Rochester, Minn., and in the District of Columbia, Maryland, Michigan, Missouri and Wisconsin. For a complete listing, see the Life Raft Group’s Web site at www.liferaftgroup.org.
— Norman Scherzer is executive director of the Life Raft
