A Local Couple Created a New Oral Cancer Therapy

Shacham and Kauffman. Photo provided to bostonmagazine.com

Sharon Shacham and Michael Kauffman, a Boston-based couple who run Karyopharm Therapeutics, met a 74-year-old lymphoma patient last year with a tumor in his arm so large that the limb was rendered unusable and amputation looked likely. But, after two weeks on a Karyopharm Therapeutics treatment, the patient improved noticeably. Six months later, his tumor was gone.

Karyopharm Therapeutics has achieved results like this one with a drug called Selinexor, which Shacham developed in 2009. Unlike chemotherapy, which destroys cancerous cells but kills healthy cells in the process, and targeted therapies, which only work on certain types of cancer, the couple says Selinexor is an easy, diverse, and effective treatment that could change cancer therapy.

“[Sharon’s] idea was to target something that would actually reactivate our own edit and audit checks that we have in all of our cells to identify when a cell becomes cancerous, and these edit checks would then only lead to the death of cancer cells,” Kauffman explains. “Because this mechanism restores some of the normal body’s mechanisms for fighting cancer internal to the cell, it can work across all the different cancer types. It’s very broadly active, but it’s also targeted to cells that are damaged in their DNA.”

Because the drug helps the body restore some of the cancer prevention processes that are lost as the disease spreads, it can also be used alongside other treatments. “It restores the normal cells’ function, so it combines well with other chemotherapies and with other targeted stuff,” she says. “If you combine it with anything that creates DNA damage, it will restore the ability of the tumor suppressor protein to identify DNA damage so we get a very potent response, so we can really combine it with many types of chemotherapies and other therapies.”

The drug is currently being tested in hospitals across the country (including Dana-Farber) as well as in Canada and Europe, and Shacham says it could be approved for mass market by 2017. So far, Kauffman says, the testing has been very successful: 80 percent of patients with blood cancers have seen tumor growth halt and 30 to 40 percent of those patients have had their tumors shrink, while half of patients with solid tumors see growth stop and 10 percent have the size decrease.

In addition to efficacy, Shacham says the drug, which is taken orally, offers a much easier treatment process than what’s currently used. “It’s an oral drug, so it makes the cancer care better. After the first month, they come up to the clinic twice a month,” she says. “This is something that we’re very focused on, the day-to-day life of the patient, maximizing the quality of life.”

The duo is hopeful that Selinexor could be the key to a new, easier era of cancer treatment. “The vision is to really change cancer from a fatal disease into one that we can chronically control and still maintain a quality of life,” Kauffman says. “We hope to see that across as many cancers as possible.”