BREAKTHROUGHS BLOG

June 04, 2017

Research Roundup: CRISPR concerns, using innovation to cure malaria, and MSF's TB trial

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PATH/Doune Porter

In this regular feature on Breakthroughs, we highlight some of the most interesting reads in global health research from the past week.

CRISPR-cas9, a revolutionary gene editing tool, has been hailed a breakthrough device for microbiologists and geneticists—but not without certain reservations. In a study recently published in Nature, researchers attempting to target and edit genetic blindness in mice analyzed nucleotides—the single-unit building blocks for DNA—in the edited and control mice in order to evaluate success of the tool. CRISPR successfully targeted the alleles—or mutated nucleotides—causing blindness in the mice, but the editing also caused 1,600 off-target, unpredicted mutations, including 100 “large changes” in the DNA structure. This is important because single-nucleotide mutations are associated with many diseases, such as sickle-cell anemia, Huntington's disease, cystic fibrosis, and more. The researchers stressed that more needs to be done to study off-target mutations before CRISPR can be considered risk-free, but that the outlook for the tool is still positive, as any scientific tool has side effects.

A man lies in a bed in a TB hospital in Kyiv, Ukraine. (PATH/Mike Wang)

The science behind creating an effective vaccine that is capable of completely eradicating a disease is complex and hard-won. The only vaccine that has been used to successfully eradicate a disease in humans is the smallpox vaccine. Edward Jenner used cowpox, a similar but much less dangerous bovine variation of the disease, to create a successful vaccine against smallpox—and now scientists are going back to that methodology in an attempt to eradicate malaria. Scientists plan to genetically modify a rodent-variation of the malaria parasite to then use on healthy human volunteers. The hope is that the variation will result in a protective response in humans. The first phase of trials will begin in 2018 and will involve studying initial protection against the disease, with future phases focusing on continued efficacy of the vaccine

Close-up of a healthcare workers hands as she inserts needle into a vaccine vial. (PATH/Satvir Malhotra)

Motivated by a lack of awareness and funding for tuberculosis (TB), Médecins Sans Frontières, or Doctors Without Borders, launched a clinical trial, entitled  TB PRACTECAL, aimed at finding a new, improved treatment for drug-resistant TB. The trial, which officially launched in January 2017, is testing an innovative combination of TB drugs that will only need to be taken for six months. Current treatments for drug-resistant TB are physically and emotionally arduous, painful, and only successful about 50 percent of the time, which is why shorted, improved treatment regimens are needed. The first set of trial results is expected to be released within two years with final results within four. If successful, the organization hopes the new treatment will be rolled out in countries around the world and mark an important step forward in combatting the global epidemic.

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