drug discovery

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Rethinking Cancer Research Through ‘Exceptional Responder’ Patients

Grace Silva and her oncologist, Jochen Lorch (Photo: Sam Ogden, Dana-Farber Cancer Institute.)

Grace Silva and her oncologist, Jochen Lorch (Photo: Sam Ogden, Dana-Farber Cancer Institute.)

By Richard Knox

By all odds, Grace Silva should have died more than three years ago. Instead, this 58-year-old grandmother is helping scientists rethink cancer treatment and research.

Silva’s case, detailed in this week’s New England Journal of Medicine, is one of only three recently published accounts of what cancer doctors call “exceptional responses” to a drug called everolimus (brand name Afinitor).

It was approved two years ago to treat certain breast cancers and is also used against some kidney and pancreas tumors. A couple of months after Silva started taking the drug, her thyroid tumors, which had spread to her lungs, melted away to nearly nothing. That basically never happens with this aggressive tumor, known as anaplastic thyroid cancer. “It was a near-complete response,” says her oncologist at Dana-Farber Cancer Institute, Dr. Jochen Lorch. “That in itself is exceptional. When we saw it, it was one of the better days around here.”

Studying The Exceptions

More remarkable still, Silva’s tumor stopped growing for 18 months. We’ll come back to what happened after that. But first, you should understand this story isn’t about everolimus or any particular cancer drug. It’s about how cancer specialists are learning how cancer works at the most basic level — by studying exceptional responders like Grace Silva.

And to appreciate why her case is important, you need to know how researchers figured out why she was an exceptional responder. It’s partly due to a five-year-old technology called next generation sequencing. It’s a cheap and rapid way of spelling out the genetic code of, in this case, individual patients’ tumors. Researchers can then look for gene mutations that are driving the uncontrolled growth that is cancer.

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Children’s Picks For Top 10 Innovation Trends Of 2012


It’s naturally Children’s-centric, but it’s my favorite Top 10 of the new year: Vector, the Children’s Hospital Boston “science and clinical innovation blog,” offers its take on where the cutting edge will be this year, and much of it rings true far beyond the hospital’s walls. (It’s also written by Nancy Fliesler, who may draw a marketing salary but could often be busted for committing journalism.)

The list is a heartening litany of the many ways that research could make medicine better. The headlines are below, and I thought the work described in this paragraph sounded particularly promising:

Getting a completely untested drug through FDA approval is a long, hard road. So researchers and pharmaceutical companies are increasingly repurposing medicines that have already been approved. Rapamycin is a great example of drug that is seemingly useful for just about everything, from immunosuppression to neurocognitive disorders to congenital heart defects. Using high-throughput assays, researchers are taking whole libraries of FDA-approved compounds, throwing them at new medical problems and finding new therapeutic “hits.” The FDA and private companies are lending a hand, and researchers at Stanford created a program that matches the gene activity caused by a disease with drugs inducing the opposite gene activity.

The headlines:
1. Whole-genome sequencing enters the clinic
2. Innovation meets healthcare reform
3. Global health: Medical missions give way to telemedicine
4. Timely diagnosis for behavioral disorders
5. Digital health apps 2.0
6. Repurposing medicines – finding new uses through mass screens
7. Rethinking clinical practice pays off
8. Making the flu less devastating
9. Taking tissue engineering to the next level
10. New pharma R&D models empower academic medical centers

The full post is here.