New Applications of Gene Editing to Cure Cancer

I've mentioned gene editing techniques quite a bit in the past 2 years, in particular referring to the development of the hugely successful CRISPR-Cas9 technique, but in the last few months, we've gone farther still.

Scientists have developed more techniques that are classified as gene editing, but not necessarily involving CRISPR itself.  In fact, I believe the CRISPR craze has re-energized laboratories and of course funders to take the plunge into the general area and develop even old techniques into viable treatments for truly serious diseases, including Cancer.

There has also been significant discussion around regulation of new techniques since we are talking about very new areas of genetic engineering that we have little experience with.  So DARPA, for one, has invested $65 million dollars into a program that should help prevent serious long term issues for people treated via gene editing techniques in the future.

The program has three main technical objectives: to develop processes that allow greater control of genome editing in living systems, to develop countermeasures that protect genome integrity in populations, and to investigate a way to remove engineered genes from living systems.

This is smart because companies in capitalistic systems can take advantage of the quick fix without caring about long term effects on people and on societies.  I never prescribe stopping or blocking research, but since science is moving at a breakneck speed, our regulatory bodies do need to be faster and even more proactive.

I talk more about the sociological impact of these technologies and ideas on regulation in the video below:

What's truly amazing about these techniques is that we're entering the realm of personalized treatments for the most part.  What is particular about cancer is that it is caused by mutated DNA.  Since the mutation can be located in different parts of the cell to cause the uncontrolled division of cells that is the disease, it means that each cancer is in fact somewhat different and unique to the individual and cancer type (both).

Thankfully, gene editing techniques means giving us the ability to target very specific gene sequences to dole out treatment that would work only for a certain individual.

For example, a lab in Boston's Dana-Farber Cancer Institute got very successful cure/remission results with patients by analyzing the patient's individual cancer neoantigens.  These are specific proteins on the surface of cancer cells that aren't present in healthy cells.  The lab reproduced the neoantigens outside of the patient, and then let the patient's immune system (T-Cells) attack the proteins once injected back into the patient along with immune system activators.  That way, the T-Cells would attack their cancer-specific neoantigens in the blood, but also attacking the cancer cells too since they are full of the mutated proteins.

Ok, this is not gene editing, but it was inspired by the idea of personalized treatment.  

Another lab had a similar idea, but instead of injecting the body with the neoantigens, they chose to extract some of the patient's T-Cells and edit their genetic code to better attack the patient's specific cancer type, and then re-inject the cells back.  This treatment saw an FDA panel approve of this treatment recently (though not FDA approved yet).  Credits for the treatment goes to the University of Pennsylvania and Novartis Corp.   

Both cancer treatments mentioned above had over 50% test patient recovery rate or better and did not cause any ill effect on the host bodies and no harmful chemicals were needed in the process either.

Gene therapy techniques like this one and others are still being tested on animals, ready for human trials for cancer and also for other terrible diseases like DMD (Duchenne Muscular Dystrophy).  We're quickly getting to very nice roads to broad treatments and cures of very nasty health problems, which is very encouraging!

And because labs everywhere have had a great time finding good ways to use gene therapy and associated techniques, it has also encouraged labs to go as far as using gene therapy to cure genetic diseases found at the embryonic level.  Yes, that means using gene editing techniques on embryos, potentially still in the womb, thus allowing the newborn baby to grow into an adult and have a healthy life without a potentially deadly genetic disease running around inside his DNA.

Pretty cool stuff and indeed, something that must be studied further and regulations in place to avoid the possible scenario of causing long term harm in trade for a short term good.

Cancer Treatments That Actually Show Promise

Just like everything else, diseases like cancer can be defeated through good scientific practice, intelligent clinical trials, and decent funding.

Understanding and perseverance can trump everything.

Lots of people have been saying that a Cure For Cancer has already been discovered a long time ago.

Truth is that is highly unlikely.

What is likely is that cures for some specific types of cancers have been discovered in the past, or are actually in use today.

What one must understand is that cancer is in fact a collection of diseases, and each person that has cancer has his own special little variation of it.  They can also appear in different tissues and organs which also adds a layer of complexity to it.

All cancers have some things in common however.  For instance, they are all caused by the modification of a cell's genetic code and they all cause the affected cells to divide abnormally, thus creating abnormal growths called tumors in areas of the body that shouldn't have such growth, and in the case of malignant tumors, those growths will eventually disrupt the natural function of the body, causing death at some point.

Not all cancers cause tumors however, such as leukemia, where cancer cells propagate in the bloodstream as individual cells,  more and more of them.  The end result is the same.

Benign tumors or cancer cause growths or abnormalities in the body but behave in such a way that in most cases, won't cause death.

Since the genetic code can be disrupted, cut, damaged or reduced in a great number of locations in such a way to cause cancer, there isn't any one pill, compound or treatment known today that can treat all cancers.

Gene editing, something that I've talked about a few times in my videos and blogs over the past 6 months, using the CRISPR-Cas9 complex, can potentially repair cells that are damaged, but that would require knowing the exact sequence that is damaged first... so that kind of treatment to cure cancer is way off right now.

The typical traditional treatments currently are either surgery or chemotherapy.

Surgery can work if the tumor is in only one spot and you can get most or all of the cells out with a scalpel.

Chemotherapy, the use of a cocktail of very nasty poisonous chemicals that will try to kill cancer cells more than it kills normal cells, is a treatment that can work on all types of tumors unless there is just too much cancer that cannot be surgically removed... In that case, the chemotherapy itself will likely kill the patient before the cancer cells are all destroyed...

Neither of these treatments are very nice and desirable, but that's all we have right now approved at hospitals.

Promising treatments and why

Luckily, a number of laboratories are working on very clever ways to treat different kinds of cancer.  Some of which have the potential to actually work on multiple types of cancer, whether they are hard localized tumors or free cells in the blood or lymph systems.

I talk about some of these in the following video:

Here they are summarized in written form for you readers out there.

Using other cells as delivery methods for medicine

In my opinion, the most promising general treatment available is to change how drugs are delivered to cancerous cells when they are localized in any form of tumor (so doesn't help with cancers like leukemia but any tumor-based cancer).

Labs have found and tested ways to deliver drugs to the locus of cancer cells and tumors.  The biggest advantage of these techniques is that the drugs can be deployed only within the tumor itself, where the chemicals can't affect normal cells.  This also means only a fraction of the normal dose of drugs would be required to kill the same amount of cancer cells.

The first uses a tumors unique characteristic of being hypoxic, which means they become areas where oxygen is lacking due to the larger amounts of oxygen consumed by these cells that keep reproducing.

The doctors could inject a patient with a malignant tumor with special bacteria that can move on their own, filled with pockets of possibly toxic cancer-killing chemicals, and the bacteria seeks and finds these hypoxic areas and stays there.  The bacteria of course are programmed to move towards and stay within those areas.  They also have a trigger that, when inside this area, they will release the medicine locally.  The bacteria are then absorbed by the body harmlessly.

The second uses a tumor's apparently natural tendency to attract our body's stem cells to them.  Scientists suspect this is a normal reaction to the body's attempt to heal where the cancer lies.  Well, labs are studying and have tested using a patient's stem cells to do the work of the bacteria stated above.

They take the patient's mature cells, change them into stem cells, fill them with the appropriate cancer-killing drugs, and then re-inject the cells into the patient's body.  The body then migrates the injected cells to the tumor, where the medicine is then released within the tumor.

This is exciting stuff because though currently, the thought is to use the same chemotherapy drugs in those delivery methods, it means we can use any other type of drug or compound delivered into the tumor once perfected, tailored to the type of cancer etc... with minimal consequence to the patient.

Using heat instead of chemicals to kill cancerous tumors

One lab has released information about how they are looking at using metallic magnetic nanoparticles instead of harsh chemicals to kill cancer cells.  The nanoparticles need to be absorbed by the cancer cells to do their work but using any of the techniques above, one could achieve such a goal (using programmed bacteria or reprogrammed adult cells).

So instead of using harsh drugs, if this thermal therapy works, we could deliver cancer-killing nanoparticles into tumors, leaving normal cells intact all the way through.  Cancer cells would be killed by heating too much versus other cells and the nanoparticles would be eliminated by the body over time afterwards without harming any other part of the body.

I think it is important to support innovative research like these versus supporting the pharmaceutical companies that make a fortune by perfecting new chemotherapy drugs that do a lot of harm to patient's bodies.  Funding is typically the most important hurdle of this type of research.

The second most important hurdle, is our willingness to try new approaches that show potential.