By: Danielle Friend, Ph.D.
Gene Therapy Treatment
Gene Therapy Effective in Treating Rare Brain Disorder
A recent study has shown promising results using gene therapy to treat andrenoleukodystrophy (ALD), a genetic disorder that occurs in approximately 1 in 18,000 individuals and is caused by a mutation in a gene on the X chromosome. With ALD, individuals lose the myelin sheath that protects and allows brain cells to function properly. Symptoms of ALD begin between the ages of four and ten and include behavioral issues. Symptoms progress with individuals developing blindness, deafness, seizures, loss of muscle control, and dementia, ultimately leading to death. Until now the only treatment for ALD was a bone marrow transplantation that includes problems related to immune rejections and difficulty matching donors.
Researchers at Bluebird Bio have now tested a gene therapy approach in a trial including 17 children with ALD. Blood from the affected children was removed, the blood cells treated with a virus that expressed the normal ALD protein, and the blood was then reinfused into the patients. According to the report, within 6 months, 16 of the 17 patients symptoms had stabilized. Brains scans conducted 2 years after the reinfusion indicate that the myelin loss stopped progressing. Importantly, none of the patients have shown signs that the gene was inserted into an inappropriate location on the genome, causing serious side effects seen with other gene therapies.
Although these results have lead to a possible therapeutic for halting symptoms, the treatment is unable to reverse the damage to the myelin and thus reverse symptoms that have already manifested. However, the hope is that in the future gene therapy to treat ALD could be used as soon as patients are diagnosed, thus ideally preventing the progression and development of the more severe symptoms.
Bluebird Bio will soon be applying for approval from U.S. Food and Drug Administration (FDA), however a long road to treatment of patients in the United States could be ahead. While authorities in Europe have given approval for the use of gene therapy for rare disorders, the FDA has yet to approved a single gene therapy. This hesitancy from the FDA likely stems from previous side effects and the unfortunately death of Jesse Gelsinger resulting from gene therapy treatment. In addition to the development of an effect means for treating ALD, these gene therapy findings also provide hope for gene therapy techniques in safely treating other diseases in the future. (Jocelyn Kaiser, Science)
Rapid, Cost Efficient Diagnostic for Zika Coming Soon
Until recently the only means for determining if an individual was infected with Zika was to use a technique known as the Polymerase Chain Reaction (PCR) or antibody detection. Unfortunately the resources to perform these tests are unavailable in many areas. Furthermore, antibodies produced against Zika may cross react with those produced against other similar viruses, thus limiting the specificity of the results. For an example, an individual whom was infected with dengue, when tested using the antibody method, may also test positive for Zika despite never having been infected with Zika. Since the rise in Zika spread there has been an emphasis on the development of a diagnostic test that would allow individuals all over the country (and world) to easily and quickly be tested for the virus.
Last week, a consortium of researchers including scientists from Harvard, Massachusetts Institute of Technology, Cornell, Arizona State, and Boston University all led by Dr. James Collins, a synthetic biologist at Harvard announced that they may developed such a test. The new test, which can develop results in two to three hours is not only faster and potentially more accurate than current methods but is also much cheaper and the result can be easily read. The test is paper-based and consists of a series of yellow dots that change to the color purple when placed in contact with Zika virus RNA.
While the test has not yet been tested with human samples, it has successfully detected Zika virus in the blood of infected monkeys. Dr. Francis Collins, the director of the National Institutes of Health stated that they are now working with groups in Colombia and Brazil to test the diagnostic in the field. Additionally the researchers state that they hope the new test will not only reduce future spread of Zika but other possible diseases as well by paving the way for a rapid, low-cost diagnostics. (Donald McNeil Jr., New York Times)
Public Health Policy
Food and Drug Administration Begins Regulating E-Cigarettes
The first piece of legislation aimed at regulating e-cigarettes was issued last week when the Food and Drug Administration (FDA) made the sale of e-cigarettes illegal to those under the age of 18. According to the Center for Disease Control and Prevention, approximately 5.3 percent of middle school and 16 percent of high school students reported using e-cigarettes in the past 30 days, a statistic that has risen in the last couple of years.
While public health experts support the new regulations, they also call for the FDA to do much more, suggesting that increased regulation on advertising for e-cigarettes and the ban of e-cigarette flavors that appeal to children including bubble gum, chocolate, and cotton candy should be put in place. In contrast, the e-cigarette industry and others are concerned that the new regulations will make it harder for consumers to obtain less harmful alternatives to traditional cigarettes. In fact, David Levy, professor in the department of oncology at Georgetown Lombardi Comprehensive Cancer Center believes that there is strong evidence that e-cigarettes may help addicted individuals stop smoking and the new regulations may be prohibitive for individual who use traditional cigarettes from quitting.
In addition to banning sales for individuals under 18, the new regulations also call for e-cigarette manufactures whose products went on sale after February 15th 2007 to seek approval from the FDA before they begin selling their products. The FDA also states that the new approval requirement will allow the FDA to verify e-cigarette ingredients and evaluate possible health risks. The American Vaping Association stated in response “This is not regulation — it is prohibition,” further emphasizing that submitting an application for approval takes more than 1,700 hours and cost upwards of $1 million. Retailers are also banned from selling e-cigarettes in vending machines that minors have access to and from distributing free e-cigarette samples. The FDA also suggests that they may soon ban e-cigarette flavors that are specifically targeted to children.
No doubt this will not be the last we hear of this battle between the e-cigarette industry and the FDA, Nicopure Labs LLC, a manufacturer of e-cigarette products, announced Tuesday that it has filed a lawsuit in the federal district court in Washington, D.C., challenging the FDA’s regulations. (Laurie McGinley and Brady Dennis, Washington Post)
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