Sunday, July 10, 2016

A Shot in the Arm for Obama’s Precision Medicine Initiative

.Precision medicine is a big idea. Tailoring drugs and therapies to a patient’s individual disease, lifestyle, environment, and genes could touch off a health-care revolution, or so the thinking goes. But first there is much we need to learn about what that all means to a person’s health. That’s why the Obama administration announced Wednesday evening that it is devoting $55 million this year to the creation of a public database containing detailed health information about a million or more volunteers. It’s also why it’s trying to figure out how to better regulate the fast-growing genetic testing market.
Precision medicine is not wihout high risk, as the molecules have powerful effects, and side effects that are potentially lethal.  Hence the need for clinical trials.
Called the Precision Medicine Cohort, the database will be the “largest, most ambitious research project of this sort ever undertaken,” said Francis Collins, director of the National Institutes of Health, during a call with reporters. It will contain medical records, sequenced genomes, blood and urine tests, and even data from mobile health tracking devices and applications. Collins stressed that the database will represent people from all races, ethnicities, and socioeconomic classes, and said it will track participants over many years.
In a separate but related project, also announced Wednesday, the U.S. Food and Drug Administration published draft guidance documentson how it might police the exploding field of genetic testing. The agency is concerned that a new generation of genetic tests could risk patient safety. The technology underlying these tests can quickly and inexpensively sequence an entire genome and identify millions of genetic abnormalities at a time. But interpreting the results is still a work in progress.
Many of the tests purport to tell patients whether they have or are at risk for certain diseases and even direct them toward targeted therapies. The whole area has been largely unregulated, but the FDA aims to use its regulatory power to assess how accurate and clinically useful the tests really are.
Advanced tests like these are “pivotal to the future promise of the Precision Medicine Initiative,” said Robert Califf, the FDA’s commissioner, but that promise is only “as good as the tests that guide diagnosis and treatment.” Califf wouldn’t say when the new regulations would be finalized, but the new information should clarify some of the regulatory uncertainty facing companies that develop such tests.
                                                                     (figure 1) retina of a patient with retinitis pigmentosa:

Patients with currently untreatable neurologic diseases may benefit from novel therapies such as Retinitis Pigmentosa, Schizophrenia and Parkinson's disease.

 (figure 2) Normal retina

VC companies are investing in companies such as  Retro-sense to develop novel methods using injectables directly into the eye.

The current clinical trial is now recruiting it's initial patient cohort. To learn more about the study, it is published at  
RST-001 is a gene therapy given as an injection into the eye and delivers a gene encoding a photo switch, channelrhodopsin-2, (optogenetics) to cells in the retina of the eye. When expressed, the channelrhodopsin-2 protein can depolarize in response to light thus generating a signal that is transmitted to the brain.
The study is composed of two parts. An initial dose-ranging study (part 1) is proposed whereby three dose levels of RST-001 will be studied in three separate groups of adult patients with advanced disease. This first part of the study is aimed at determining a single dose of the experimental agent which is safe and well tolerated, to further evaluate in a fourth group of patients. The second part of the study is aimed at obtaining additional safety data at the highest tolerated dose and providing important additional clinical data to guide the design of future efficacy studies.)

A Shot in the Arm for Obama’s Precision Medicine Initiative
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