The U.S. Needs a Medical Second Amendment

11/22/2013 10:45

by Ray Blanco


In the United States, we recognize a person's right to self-defense -- the ability to defend your life when threatened with death or severe injury. We do not, however, recognize a person's right to defend their life by pursuing therapies of their choice when faced with a death-dealing disease for which there are no FDA-approved treatment options.

There might be a lifesaving therapy out there, but unless it has been approved or you are lucky enough to be enrolled in a clinical trial and not get the placebo, you are left essentially defenseless. There are few or no exceptions to this state of affairs.

I'm pretty sure you can see I'm no fan of the FDA. In an ideal world, it would function as a sort of master peer-review organization, affixing its stamp of approval on drug safety, but otherwise not impeding biotechnology companies like Sarepta Therapeutics (NASDAQ:SRPT) from being able to bring their lifesaving therapies to the market. Patients could decide for themselves under the guidance of their physicians, taking FDA recommendations into account. It's true that the FDA has kept some dangerous drugs off the market, but there is also a cost -- a huge cost-- in that it prevents therapies for patients with severe diseases from coming to market sooner.

Duchenne muscular dystrophy (DMD) is one of those diseases. Individuals with DMD have mutations of a gene coding for the dystrophin protein, which totally shuts down its production in muscle cells. Dystrophin, the largest protein in the body, is needed by these cells in order to stay healthy. DMD is one of the most severe forms of muscular dystrophy. It is also sex-linked, and only males get it. DMD patients can expect to lose their ability to walk around the time they reach puberty and not live long after that. Most of them never see their 30th birthday -- lungs can't breathe when the muscles driving them fail.

Sarepta has amassed 96 weeks’ worth of data on a dozen boys taking its breakthrough DMD drug, eteplirsen. Eteplirsen patches the defect in the dystrophin gene, allowing cells to produce the protein. The compound has shown remarkable safety, and better yet, the data we've seen to date show that the walking ability of the boys on the drug stabilizes. Normally, over a period that long, nearly two years, a steep reduction in the ability to walk would be expected. On a different measure, eteplirsen was also shown that it could cause muscle cells to produce dystrophin, as evidenced by muscle biopsies.

So here we have a drug that on two different measures -- molecular and functional -- is shown to work. Furthermore, patients have suffered no adverse effects from receiving it.

So why not allow a compound that has shown such great data to be brought to the market with the proviso that more studies will be performed?

Sarepta hoped, based on these data, that it could receive a provisional approval to market eteplirsen for DMD patients, subject to further studies after getting the go-ahead. That's not going to be the case. The FDA has told Sarepta it doesn't recommend the company apply for marketing approval at this time -- which amounts to a politely worded rejection.

So where does this leave Sarepta? Its drug has shown enormous promise. The company will pursue larger clinical trials to provide more data to support a marketing application. This, however, will mean Sarepta will have to wait two or three more years -- even if it collects data that show its compound is safe and beneficial and it efficiently conducts its trials.

There's a time component to money, and many investors sold their shares and moved on when faced with this news. Important, however, are the DMD patients. They will have to wait too. How many boys will become wheelchair-bound in the next couple of years who could have been spared thanks to eteplirsen?

Next-Gen Diagnostic Company Builds Case for Genomic Colon Test

Genomic Health (NASDAQ:GHDX) is busily expanding its cancer diagnostic franchise, originally built on the success of its breast cancer test. There is an important statistical aspect to Genomic's technology. The key is to identify the most predictive genes in a cancer type and then build a test that can use these genes to help a patient make a decision regarding his or her cancer treatment. To do this well, large studies in which an outcome can be compared with the particular genomic profile of a patient's tumor have been necessary.

Genomic has continued to pursue studies of its cancer diagnostics, even after marketing approval, in order to further validate its offerings. What's even better, however, is the fact that independent researchers have taken a keen interest in Genomic's diagnostic platform and are continuing to publish results supporting the company's claims.

That's the subject of Genomic's news release this week. The National Surgical Adjuvant Breast and Bowel Project (NSABP) has published an 892-patient study of Genomic's recently introduced colon cancer diagnostic, Oncotype DX Colon, in the Journal of Clinical Oncology. The results support the accuracy of Genomic's “recurrence score,” a genome-based measure of risk of disease recurrence. Furthermore, the study showed that Genomic's scoring was also predictive of disease-free and overall survival in patients.

Genomic is part of a wave of new diagnostic technology that is only just breaking into the industry. We've amassed a large number of new cancer therapies in the past few years, but without good diagnostics, we may be misusing, underusing or overusing them. Gene-based tests help make the best use of our therapeutics, helping patient health as well as reducing health care costs.

Genomic's colon diagnostic product is still relatively new, and its prostate test even more so. The company has already shown it can succeed with its original breast cancer assay. I expect it will do so in these new cancer types.

Ad lucrum per scientia (toward wealth through science)..



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