Scientists discover a drug that could target acute myeloid leukemia

Acute myeloid leukemia (AML) is a cancer of the myeloid line of blood cells, characterised by the rapid growth of abnormal white blood cells that build up in the bone marrow and interfere with the production of normal blood cells.

AML is the most common acute leukemia affecting adults, and its incidence increases with age. Mainstream AML treatments have remained unchanged for decades and fewer than one in three people survive the cancer.

But, now a study has found an unexpected new  drug target for acute myeloid leukemia (AML) that could open new avenues to develop effective treatments against this potentially lethal disease. The scientists used CRISPR-Cas9 gene-editing technology to screen cancer cells for vulnerable points.
They created mouse leukemia cells with mutations in the genes that may be targetted in human AML cells and systematically tested each gene, finding which were essential for AML survival.

The researchers ended up with 46 likely candidate genes, many of which produce proteins that could modify RNA. Amongst these, METTL3 was one of the genes with the strongest effect. They found that whilst it was essential for the survival of AML cells, it was not required for healthy blood cells, making it a good potential drug target.

Talking about the research, a  Professor said, "New treatments for AML are desperately needed and we have been looking for genes that would be good drug targets. We identified the methyl transferase enzyme METTL3 as a highly viable target against AML. Our study will inspire pharmaceutical efforts to find drugs that specifically inhibit METTL3 to treat AML."

For proteins to be produced in a cell, the DNA is transcribed into messenger RNA, which is then translated into the proteins that the cell needs. However, modifications to the RNA can control if a protein is produced.

This is a recently-discovered type of gene regulation called RNA editing. Having found a potential target in METTL3, the researchers investigated how it worked. They discovered that the protein produced by METTL3 bound to the beginning of 126 different genes, including several required for AML cell survival.

Then, as RNAs were produced, the METTL3 protein added methyl groups to their middle section, something which had not been previously observed.

The scientists found that these middle methyl groups increased the ability of the RNAs to be translated into proteins. They then showed that when METTL3 was inhibited, no methyl groups were added to the RNA. This prevented the production of their essential proteins so the AML cells started dying.

One of the first authors on the study noted, "This study uncovered an entirely new mechanism of gene regulation in AML that operates through modifications of RNA. We discovered that inhibiting the methyl transferase activity of METTL3 would stop the translation of a whole set of proteins that the leukemia needs. This mechanism shows that a drug to inhibit methylation could be effective against AML without affecting normal cells."

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Scientists grow retinal nerve cells which could help people blinded by glaucoma, MS

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Scientists have developed a new method to efficiently turn human stem cells into retinal nerve cells that transmit visual signals from the eye to the brain, an advance that could lead to treatments for people blinded by glaucoma and multiple sclerosis (MS).

Death and dysfunction of these cells, known as retinal ganglion cells, cause vision loss in conditions like glaucoma and MS. "Our work could lead not only to a better understanding of the biology of the optic nerve, but also to a cell-based human model that could be used to discover drugs that stop or treat blinding conditions," said study leader Donald Zack, from the Johns Hopkins University School of Medicine in US. "And, eventually it could lead to the development of cell transplant therapies that restore vision in patients with glaucoma and MS," said Zack.

The laboratory process entails genetically modifying a line of human embryonic stem cells to become fluorescent upon their differentiation to retinal ganglion cells, and then using that cell line for development of new differentiation methods and characterisation of the resulting cells. Using a genome editing laboratory tool called CRISPR-Cas9, the researchers inserted a fluorescent protein gene into the stem cells' DNA.

This red fluorescent protein POU4F2 would be expressed only if another gene named BRN3B was also expressed. BRN3B is expressed by mature retinal ganglion cells, so once a cell differentiated into a retinal ganglion cell, it would appear red under a microscope.

Next, they used a technique called fluorescence-activated cell sorting to separate out the newly differentiated retinal ganglion cells from a mixture of different cells into a highly purified cell population. The cells showed biological and physical properties seen in retinal ganglion cells produced naturally, said Zack. Researchers also found that adding a naturally occurring plant chemical called forskolin on the first day of the process helped improve the cells' efficiency of becoming retinal ganglion cells. 
"By the 30th day of culture, there were obvious clumps of fluorescent cells visible under the microscope," said lead author Valentin Sluch, a former Johns Hopkins student. "It seems we can now isolate the cells and study them in a pure culture, which is something that wasn't possible before," Sluch said. "We hope that these cells can eventually lead to new treatments for glaucoma and other forms of optic nerve disease," said Zack.

The study was published in the journal Scientific Reports.