I have shared with you in the past the efforts of Dr. Michael Kahn and myself to develop novel drugs which are completely different than the existing ones.
Even the smart drugs we have such as Avastin or Erbitux, which are monoclonal antibodies that attack important targets in cancer growth and progression, have disappointed, particularly if you are not able to select the patients who benefit the most.
Why are they not more effective?
The communications hot lines in cancer cells are extremely complicated and look like a subway/bus/tram network of large cites. Do you think when you knock out one even major intersection; it will be lethal to the city?
No. Annoying it may be, but we all know detours and ways to avoid these problem spots and so does the tumor. In fact tumor systems often have backup communication lines which can handle these attacks.
So what do we need to do? We cannot shut down one exit or one intersection we have to shut down major lines (red, purple whatever), which are essential to maintain traffic. In cancer biology we call those major lines pathways.
Dr. Kahn has developed a novel drug which shuts down one of the most important pathways in colon cancer called Wnt (pronounced wind). This drug has shown tremendous potential to treat many cancers including leukemia and even noncancerous disease such as pulmonary fibrosis.
On September 17, 2010 we filed an Investigational New Drug application to the FDA, which means we want approval from FDA to test this drug for the first time in patients. We expect that the trial will open in October or November.
We are very excited because this drug not only shuts down this important Wnt pathway, it also is effective treating colon cancer stem cells, which has not been possible in the past.
We all know when we treat colon cancer patients with metastases that the first chemo works usually the best, the second shorter, and with the third there is little chance. We all think this is due to the cancer adapting to the chemotherapy. However we think it is that these tumors are getting enriched with stem cell like cancer cells which are, by definition, resistant to chemotherapy. With this drug we can change that.
But this is not the best news. This drug has so far no shown any side effects, even when tested at 250 higher concentrations than needed. Obviously we need to await the clinical data but we are very excited.
USC is so excited that they gave us permission to create a new center called USC Center for Molecular Pathways and Drug Discovery because we have other hits and leads to develop and want to do it as fast as possible.