Headline, July11, 2013

'''SMASHIN​G -CANCERS- UGLY HEADS'''

Carolyn had a rare form of cancer, a sarcoma, that started in the uterus and spread to her liver and lungs even after she had a hysterectomy. She looked like a goner; her doctor warned her  -chemotherapy would make her sick and wouldn't do much good.

Then in May 2003 Carolyn became the first patient to try a new cancer drug. Two years later the cancer on her liver has disappeared and the cancer on her lung was gone to. Even Scientists at Ariad were shocked. ''Goodness me, we got a response,'' Ariad's scientific officer recalls thinking.

The bigger shock was that Ariad, a publicly held shop founded on work by Harvard and MIT scientists, pursued this new drug at all. Code named AP23573 and a little later in middle-phase trials of 176 patients, it was aimed at an awfully small market: Only 12,000 to 13,000 new cases of sarcoma are diagnosed in the U.S. each year, compared with 213,000 new cases of breast cancer.

A new treatment for sarcoma, a grab bag of connective tissues tumors that show up mostly in muscle, cartilage and bone, hadn't been approved in two decades. Ariad and other biotechs once pursued only the broadest markets, but they developed a newfound interest in curing the rarest of cancers. The revenue upside turned out to be surprisingly lucrative.

This is because the cause of a rare form of cancer often can be narrowed down to a single gene, providing a clear target, and because few other remedies existed, the promise of a new drug became instantly visible.There model though was Gleevec, the Novartis drug for a rare form of leukemia that occurs in only 4,600 new patients a year in the U.S.  -but brought in over $2.2 billion.

Gleevec was so good at attacking chronic myelogenous leukemia that it won approval in less than three months, after only three phases of trials that the feds usually require. Ariad had planned to follow suit. Its AP23573 pill had shown amazing promise in sarcoma, which kills 5,000 people in the U.S. each year. It is treated with surgery, radiation and chemo, but when it returns there is little hope.

''We've seen this drug do things that no other drug has done yet,'' said George D. Demetri of the Dana Farber Cancer Institute, who codesigned trials of AP23573 after big successes with other fast-tracked pills, such as Gleevec and Pfizer's Sutent.  

The drug works by tripping a cellular switch that was discovered serendipitously by scientists studying an unrelated drug  -Rapamune, a Wyeth chemical used to suppress the immune systems of organ transplant patients. The weird part : In cancer no drug should suppress the immune system :

Unwanted side effects on the immune system were still anticipated but early data did provide hope.

Sarcoma is just the head of the train,'' said Ariad founder and Chief Executive Harvey Berger.
This is the drug that is going to help people!''

The prospect of quick riches has biotechs targeting the rarest of cancers. But some great knowledgeable scientists did sound : Mission Improbable.

With respectful dedication to all research scientists of the world.

With respectful dedication to Shaukat Khanum Cancer Hospital, Pakistan.

Good Night & God Bless!

SAM Daily Times - the Voice of the Voiceless