Another sign that the long-awaited promise of gene therapy (a form of personalized medicine) can be fulfilled is evident in two patients who are doing well after 18 months of it. Yet according to Deloitte, the US healthcare system appears to be reluctant to ride the trend toward personalized medicine.

Perhaps Deloitte missed the significant contribution the US government’s US$1 billion Cancer Genome Atlas project (mentioned in our January issue) will make to personalized medicine, so here’s a Wall Street Journal article as a refresher. The US healthcare system was also very much involved in the recent surprising discovery of a genetic factor in Parkinson’s Disease, and in the discovery of a genetic cause for epilepsy.

Not least, the market for genomic tests is taking off in the US, and with it the era of personalized medicine.

All in all, while the current Administration’s policies toward science and research are less than enlightened and in many ways making the US less competitive, the NIH is doing a valiant job and maybe it’s a bit premature to write off the US in this new arena of medicine.

Gene Therapy Success

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For the past 18 months, Swiss, British, and American researchers have been treating two men afflicted by chronic granulomatous disease (CGD) with gene therapy for 18 months. They used a virus to repair a defective gene in myeloid cells that was inhibiting production of an infection-fighting enzyme.

Both patients are in good shape and no longer have to take round-the-clock antibiotics, reports Gautam Naik in the Wall Street Journal . “Abscesses that had formed in the lungs of one patient and in the liver of the other have disappeared. So far, neither patient is suffering negative side effects.”

But Can We Handle It?

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A study by Northwestern University’s Kellogg School of Management said that globalization, an aging population, and economic forces are pushing the trend toward personalized medicine, which, says a Deloitte Consulting executive citing his company’s ongoing study, the US healthcare system is slow to adopt.

Pharmacogenomic Approach to Cancer Accelerating

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With a nod to the Cancer Genome Atlas project (see article in our January issue), Wall Street Journal reporter Amy Dockser Marcus notes “a significant shift” in the understanding of cancer, and therefore of research and clinical responses to it. Instead of focusing mainly on the organ in which a tumor appears, researchers and clinicians are focusing more on the patient’s genetic makeup, finding the specific genetic changes driving an individual’s cancer and targeting those changes with drugs.

Examples include the lung cancer drugs Tarceva and Iressa, leukemia and sarcoma drug Gleevec (Gleevec’s maker claims it is also effective for four other diseases, including certain blood cancers and a kind of skin tumor), and breast cancer drug Herceptin, which work in certain patients with certain genetic makeup but not in others. New cancer subtypes, she says, have been discovered in liver, brain, and prostate cancer patients.

The goal of the Cancer Genome Atlas and other projects taking the new approach is to characterize genetic mutations in cancer, identify genetic variations in patients, and find a way to target treatments accordingly. Tests are available to identify patients who have the genetic mutation that makes them likely to respond to Tarceva or Iressa.

“The idea isn’t to treat everyone the same and end up with drugs that make many people ‘only 20% better’,” writes Dockser, quoting a cancer researcher; “’Instead, we’d like to start making 20% of the people 100% better’.”

Parkinson’s Link

Epilespsy/Autism Cause

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A genetic cause for epilepsy has been found by researchers at TGen and the Clinic for Special Children in Strasburg, Pennsylvania. It could lead to better medicines for both epilepsy, and the first-ever drug for autism, as the researchers turn their attention to developing drugs to prevent the diseases by changing the way the brain reacts to the culprit gene.

The discovery of the genetic mutation has already enable doctors to identify at-risk newborns before they show symptoms.

Genomic Test Market Grows

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Genomic Health’s “Oncotype DX” test is “part of a new wave of sophisticated genetic or protein tests that are starting to remake the diagnostics business,” writes Andrew Pollack in the New York Times . The new generation of tests, he says, “represents some of the first fruit of the long-anticipated genome revolution and could help pave the way to personalized medicine.” They are also expensive, often patent-protected, marketed directly to doctors or in some cases patients instead of to medical laboratories, and very profitable. Genetic testing is already a US$5 billion business and growing by 25 percent annually.

Oncotype DX can tell whether a woman with a certain kind of breast tumor might not benefit from chemotherapy, therefore saving her the pain, cost, and futility of a treatment that isn’t going to work. A Genomics Health competitor told Pollack the company “had done a ‘fantastic’” job of validating its test using clinical trials and then of ‘packaging it in a Starbucks package at a high price.’”

Genomic Health and companies with similar business models perform all the testing in their own laboratories, rather than selling or licensing the tests to hospital and commercial labs. This could keep prices high and deter independent validation of the tests’ value over time. It could also take US Food and Drug Administration oversight out of the loop, since tests done by a single laboratory do not usually need regulatory approval, unlike test kits sold to hospitals and doctors. However, the FDA is apparently treating the tests as devices, to bring them within its purview; but critics say that this will stifle progress because few companies can afford to put their tests through the regulatory process.

Other tests on the market include:

  • XDx’s $3,000 blood test that analyzes whether a transplanted heart is being rejected. Myriad Genetics’ $3,000 test for the risk of developing breast cancer.
  • Monogram Biosciences’ up-to-$1,460 tests to determine the best drugs regimen for HIV/AIDS patients.
  • Exact Sciences’ $500 DNA test for colon cancer, which requires a stool sample and is apparently not selling well.

Insurers are not yet rushing to pay for the tests, though Genomic Health’s test has a foot in the door.


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