Alexandra M. Levine, distinguished professor of medicine and chief of hematology at the Keck School of Medicine and medical director of the USC/Norris Cancer Hospital, presented results from the ongoing Veglin trial at the 40th annual meeting of the American Society of Clinical Oncology in June.
Veglin originated with USC/Norris Comprehensive Cancer Center researcher Parkash Gill, Keck School professor of medicine. Los Angeles-based VasGene Therapeutics Inc., which was co-founded by Gill, now leads its development.
In 2003, a team of USC/Norris scientists opened a phase I trial of Veglin for patients with any malignancy that failed to respond to previous treatment. The study evaluates the safety of the drug at increasing doses and examines response. So far, researchers have increased the dosage tenfold, with no significant toxic side effects seen.
Nearly three quarters of the 35 patients who have participated in the clinical trial so far have had solid tumors of the kidneys, colon or lung, or cancers such as melanoma or sarcoma. More than a quarter of the patients have had hematologic cancers, such as lymphoma, Kaposi’s sarcoma and myeloma.
Cancer cells develop rapidly and need an ever-increasing blood supply, researchers said. As a result, tumors must encourage new blood vessels to develop around them – a process called angiogenesis. VEGF is critical to the growth of these new blood vessels.
VEGF also directly helps certain cancers grow.
“VEGF serves as an autocrine growth factor for certain types of cancers,” Levine said. “The analogy would be the following: If a car were able to make its own gasoline, it would drive forever. The gasoline for the cancer cell is VEGF; it is made by the cancer cell and comes back to work on the cancer cell that made it, causing the cancer cell to divide and proliferate.”
Veglin is meant to counteract that. Called an antisense oligonucleotide, Veglin is a bit of DNA that binds directly to the gene that produces VEGF – essentially plugging it.
Researchers hope that if Veglin can keep tumor cells from producing VEGF, it will block their growth and metastasis, while also killing the cancer cells themselves.
Patients in the trial receive Veglin intravenously over two hours, for five straight days. They then get a week off. This cycle continues for four months.
Veglin lowered blood levels of a form of VEGF, called VEGF-A, in 47 percent of participants. It also lowered levels of VEGF-C in 21 percent of participants.
In addition to Kaposi’s sarcoma and cutaneous T-cell lymphoma patients, whose cancer growth was reversed for several months, other patients saw clinical benefits too. Veglin stabilized cancer levels in patients with renal cell cancer for two or more months, chondrosarcoma for five months and bronchoalveolar carcinoma for more than seven months.
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…in a couple medical studies as a patient. Being a guinea pig can have it’s good and bad points. If it’s a double blind study and you get the experimental drug, it might help. If you get the placebo, it’s confusing.
I’ve always wondered, why do these studies have such a small number of participants and what are the ramifications of the participants being from one locale? Running a statistical analysis on the usually small number of participants seems to be pushing credibility (but I have never trusted statistics). Plus, testing participants from a particular geographic locale could inflict a factor that tilts the results (for example, people from Three Mile Island or the left coast).
I would like to see these studies being simultaneously duplicated at various medical centers around the country. Let’s say thirty centers have thirty participants (the figure I gleaned from the USC/Norris site). Nine hundred results is more credible than thirty. But I doubt researchers would want to share — somebody else might publish first or come up with an improvement.
That said, rickyjames, the study was intriguing. It’s nice to know that cancer is being attacked from many fronts.
jon
p.s.. I got the placebo…
In response to the last message, I would like to comment. As a Chondrosarcoma Patient who has survived 40 years beyond expectations (possibly because of the Clinical Trial I participated in) I would like to say that when a person has a condition that might end in death, the opportunity to participate in a clinical trial, even with the possibility that it wont work, is still worth it.
“Nothing ventured…Nothing gained.”
That being said, I would also like to respond to the remarks about small trials (only 30 participants mentioned in the original article on Veglin) and keeping it in one location. I called the trial coordinator and spoke on the phone. The participants in the trial are not only from the Los Angeles area. They are accepting anyone who fits the criteria regardless of their home location.
As moderator of the online Chondrosarcoma Support Group, (yahoo groups) I can tell you that many Chondrosarcoma patients are willing to travel wherever they feel they can get treatment.
Why? Because Chondrosarcoma is so rare that there are only about 1200 people per year who are diagnosed with it. Chondrosarcoma is so unusual that standard cancer treatments are not effective.
Wouldn’t you hop on a plane and travel half way around the world to have the chance to save your life? I know several people who have done just this. I have only traveled 3,000 miles for some of my recurrences.
When a new treatment for a rare cancer comes along, having a large cohort of patients is not a possibility. So, if you have only 30 patients and the percentages of success are significant, it is an indication of effectiveness. That being said, please be aware that before any clinical trial can be approved, it has to pass many rigorous tests in the petri dish and with animal models who have the diseases the medication is intended for, before it becomes available as a clinical trial for human subjects.
Only time will tell, as the trial continues, and more patients are treated. A good example of this is how Gleevec came into use. Now, patients with the rare GIST (Gastro Intestinal Stromal Tumor) cancer can expect to have good chances of survival. If the researchers for the Clinical Trials for Gleevac were required to have a large cohort of patients it never would have been approved by the FDA.