Medical

Molecule behind Huntington's disease found to be cancer-killing "super assassin"

Molecule behind Huntington's d...
The same molecule that causes Huntington's disease has been found to have remarkable cancer-killing properties
The same molecule that causes Huntington's disease has been found to have remarkable cancer-killing properties
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The same molecule that causes Huntington's disease has been found to have remarkable cancer-killing properties
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The same molecule that causes Huntington's disease has been found to have remarkable cancer-killing properties

For several years, scientists have noticed an unexplained reduced incidence of cancer in patients suffering from the devastating hereditary neurodegenerative condition Huntington's disease. Now a team at Northwestern Medicine has uncovered how the disease could be inadvertently killing cancer cells, and how this process could be harnessed for a new cancer treatment.

Huntington's disease is caused by a mutation in a gene called huntingtin. The mutation generates repeating RNA sequences and these repeating sequences, known as small interfering RNAs (siRNAs) are what slowly damage neural cells, causing the progressive neurodegeneration associated with the condition.

Previous research by the same Northwestern team discovered that siRNA molecules were amazing cancer-killing assassins that evolved in living organisms millions of years ago to fight cancer before the more complex adaptive immune system developed. This research inspired the team to investigate whether there were diseases involving similar RNA mechanisms that also correspond with lower rates of cancer.

"I thought maybe there is a situation where this kill switch is overactive in certain people, and where it could cause loss of tissues," says first author on the study Andrea Murmann. "These patients would not only have a disease with an RNA component, but they also had to have less cancer."

The repeating siRNA sequences found in Huntington's pathology were discovered to be very similar to the siRNA molecules identified in the team's earlier work. So the next step was to test whether this particular molecule, when delivered via nanoparticles to mice, actually worked to kill cancer cells. The results were remarkable. Tumor growth was significantly reduced in a huge variety of different cancer cell lines, including ovarian, breast, prostate, liver, brain, lung, skin and colon cancer cells.

"This molecule is a super assassin against all tumor cells," say senior author Marcus Peter. "We've never seen anything this powerful."

The next step for the researchers is to work on refining the nanoparticle delivery method so that the cancer-killing molecules can be more accurately delivered to a target tumor. This is obviously several years away from becoming an approved clinical treatment, and it is still unknown whether administering these damaging molecules to neurologically healthy humans would cause Huntington's-like symptoms.

The researchers suggest that Huntington's patients are exposed to these toxic RNA sequences for decades before the damage is great enough to cause clinically-observable symptoms, so this may lead to a viable short-term cancer treatment.

"We believe a short-term treatment cancer therapy for a few weeks might be possible, where we could treat a patient to kill the cancer cells without causing the neurological issues that Huntington's patients suffer from," says Marcus Peter.

The research was published in the journal EMBO Reports.

Source: Northwestern Medical

3 comments
Nik
I've lost count of the number of articles that I've read that announce, 'amazing cancer killing possibilities' that then disappear into obscurity. Treating cancer is a multi-billion$ industry, curing it is not. So this 'amazing cancer killing possibility' will just disappear like all the rest:-(
Ralf Biernacki
@Nik: There are three hurdles all these promising cancer treatments must clear before they are used. 1) After they have shown promise in "humanized" mouse trials, they must undergo human trials, which take a long time to approve and are several times more expensive than all previous research together. 2) To be approved, the new drug must meet exacting regulatory requirements. These bureaucratic procedures take years, and if only a few human test subjects develop mild side effects (and I do mean mild, e.g. diarrhea or a rash) the promising drug is disqualified. It may not make sense to you to disqualify a cancer drug because it sometimes causes a rash; but FDA regulations treat all drugs the same, whether they are meant to clear acne or save lives. It makes little sense for the producer to sink millions into a drug that is disqualified because two test subjects developed a headache. 3) After the new drug is released into the market, if unexpected side effects or drug interactions show up, the manufacturer is liable for astronomical damage payments, often way out of proportion to the actual harm. The lawyers, and regrettably the courts, seem to think: "Hey, these pharma companies are rich! Let's milk them for what they're worth!" For these reasons, most prospective drugs are simply shelved after mouse trials, and the companies look instead for previously tested and approved drugs that can be reused to treat other conditions. No pharma company, however rich, can afford to market drugs at a loss. No company of any kind could.
Rocky Stefano
@Ralf. Appreciate the comments but like Nik said, there's too much money involved in the current infrastructure around cancer treatments and research to let any real cure loose. The effects would destabilize the economy. Same with oil. It takes decades for the economies to shift and it doesn't matter what any of us wish would happen. Yes no pharma can afford to lose money but watch this video @ https://www.youtube.com/watch?v=FlK-PeCfezM . You tell me that given the opportunity, a family would rather watch their child die from leukemia rather than chance the new treatment in the video? I'll take the shot and I bet 99% of the other parents would as well. Let's drop this nonsense about mice to men trials.