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Science Feature Articles</p>

When Genes Conspire to Cause Disease

English
Prof. Eytan Domany and Dr. Libi Hertzberg
 
It has been known for a while that schizophrenia has a strong genetic component: The proof comes from studies of identical twins. But when researchers look for genes associated with the disease, they are confronted with a profound muddle: Hundreds of genes appear to be involved, but upon closer inspection each only confers a slightly higher than normal risk of developing the disease. Recent findings arising from a unique collaboration between researchers at the Weizmann Institute of Science and Shalvata Mental Health Center in Israel, and the Mount Sinai School of Medicine in New York, suggest a way out of this mire.

Such disease-encoding genes are generally identified in so-called genome-wide association studies. The idea is to compare genomic sequences of thousands of subjects – patients as well as healthy people – and search for tiny differences of just one or two “letters” in the genetic sequences that make up the genes. If certain variations appear more frequently in those with a disease such as schizophrenia than in the healthy population, one can start asking whether the change in that particular letter is connected to the disease.

But with hundreds of somewhat feeble candidates, the data dissolve into “noise.” There is little way to tell if the switched letter is an alternate spelling or punctuation, or whether it will be like substituting “pear” for “peach” in a recipe – a slight but possibly significant alteration to the final dish. To further complicate things, many of the substituted letters in the genomes of people with schizophrenia show up in so-called non-coding regions – those that do not contain instructions for making proteins but, rather, regulate such things as protein levels. These sequences are not only less well studied and harder to identify than the ones in coding regions; their functions are difficult to observe in standard lab tests.
 
Unraveling this mystery presented a compelling challenge to Dr. Libi Hertzberg, who is no stranger to challenges. Hertzberg had been a master’s student under Prof. Eytan Domany, head of the computational biology group in the Institute’s Physics of Complex Systems Department. From there, she went on to Tel Aviv University to complete both an MD in the Sackler Faculty of Medicine and, concurrently, a PhD that was supervised, in part, by Domany. She is now at Shalvata Mental Health Center, as a resident in psychiatry, and, in addition to her demanding clinical work, has decided to research the basis of mental illness together with Domany.
Gene expression of schizophrenia-related genes. 1,028 genes with very similar expression profiles are shown, one to a row, and 480 samples are arrayed in the columns. Red-to-blue shows the strength of the gene's expression level, red being stronger
 
 
Domany and Hertzberg teamed up with Prof. Vahram Haroutunian of Mount Sinai. Domany, who holds the Weizmann Institute’s Henry J. Leir Professorial Chair, had met Haroutunian at an annual conference hosted for its researchers by the Leir Charitable Foundation, and the two realized that Haroutunian had a unique resource that could help solve the mystery: He has a database of information gleaned from post-mortem brains that have been donated to his lab, including those from schizophrenia patients. From these, he can test the levels of the messenger molecules – mRNA – that are produced from the various genes. In other words, scientists can use these data to understand how the genetic information translates into action in various brain cells.

Now the team had two very different sets of information – genes identified in the broad, genome-wide studies and the mRNA levels from the brain database – giving them a sort of “filter” that enabled them to identify the genetic sequences whose slight misspelling was not only associated with the disease but also exhibited interesting patterns of expression in the brain.
 
 
 
 

The team then began to analyze their narrowed-down list of genes: The approach Domany has developed over the years looks for the actions of groups of genes, rather than searching for the effects of a single gene, and this strategy worked well for the schizophrenia data. Using algorithms he and his team have developed to first identify paired correlations and from these, clusters, they ultimately identified a collection of around 19 genes that clearly stood out from the noise.

Now the question was: What does this group of genes do? That question is far from simple: there are hundreds of ways that these genes could interact and thousands of possible effects of their actions. Further computational analysis of the data revealed that the cluster of genes they had identified is associated with the functioning of the cells’ calcium channels. Nerve cells rely on these channels in their membranes to regulate the uptake of calcium ions, which excite the cells to action. Additional tests using information from the genome-wide studies and databases of protein interaction analyses supported their results.  

Hertzberg says that these findings give strong backing to the idea that calcium regulation plays a central role in schizophrenia, and adds that the genetic interactions they have revealed might present useful targets for drugs. Domany points out that the next step is to understand exactly how the regulation of calcium signaling goes awry in the disease – a step that will require much more research. But the scientists are hopeful that their results, in addition to pointing to a fruitful approach to understanding how genes contribute to neuropsychological disease might, in the future, lead to both better diagnostics and possible treatments for schizophrenia.

 

Prof. Eytan Domany’s research is supported by the Leir Charitable Foundations; and the Louis and Fannie Tolz Collaborative Research Project. Prof. Domany is the incumbent of the Henry J. Leir Professorial Chair.




 

 
Gene expression of schizophrenia-related genes. 1,028 genes with very similar expression profiles are shown, one to a row, and 480 samples are arrayed in the columns. Red-to-blue shows the strength of the gene's expression level, red being stronger
Life Sciences
English

Cold War against Cancer

English
Lea Eisenbach
 

Prof. Lea Eisenbach is a pioneer in the field of cryoimmunotherapy. As its name suggests, this approach combines two forms of treatment: cryosurgery, from the Greek cryo, for “icy cold,” and immunotherapy, which harnesses the tools of the immune system. The combination of these two accepted treatments of cancer results in an innovative approach that has produced promising results in the lab.


Eisenbach, of the Weizmann Institute’s Immunology Department, began to experiment with this method some ten years ago, when a physician interning in her lab suggested they look into a technique known as cryosurgery, which is not really surgery at all. Rather, a tumor is pierced a number of times with a needle previously frozen in liquid nitrogen, and the repeated freezing and thawing causes tumor cells to burst and die. This treatment, used increasingly in the past few decades to eliminate cancerous tumors of the prostate, liver, kidney and other organs, causes less damage to surrounding tissue than regular surgery. Yet another advantage: The destruction of tumor cells triggers inflammation, which activates the immune system, prompting it to fight the cancer. This activation, however, does not necessarily prevent the cancer from spreading to other organs. So the goal of cryoimmunotherapy is to enhance the effectiveness of the immune response in metastasis, when the cancer in on the move.

 


Several years ago, Eisenbach’s team performed cryosurgery on mice that had cancer metastases in the lungs, after which they injected these mice with dendritic cells – cells that detect infectious organisms, malignancy and other dangers, and activate the immune system accordingly. The mice that received the cellular injections remained disease-free for longer periods and lived longer than those treated with cryosurgery alone. But ultimately, only half of them survived.

 
dendritic cells
In the healthy lymph node, dendritic cells (green) form interconnected networks in which T cells (red) migrate. Image: lab of Dr. Guy Shakhar
In a recent study, research student Zoya Alteber and other members of Eisenbach’s team have managed to improve this survival rate. As reported in Cancer Immunology and Immunotherapy, they treated mice that had lung metastases in the same manner as before, with cryosurgery and injections of dendritic cells, but added an injection of molecules called CpG-ODNs, known to provoke a strong immune response. These molecules, present on the surfaces of bacteria and viruses, bind to receptors on dendritic cells, causing these cells to unleash a chain of biochemical signals that results in the release of various immune chemicals and in the activation of immune T killer cells, which fight malignancy.
 
Indeed, the scientists found that about a week after the treatment, the mice had increased levels of several immune chemicals called cytokines, as well as high levels of T killer cells, in their lymph nodes and elsewhere. But most important, mice that received the dual immune treatment had almost no lung metastases, and about three-quarters of them survived – a marked improvement over the results of the earlier study.

Moreover, when the scientists later injected the surviving mice with malignant cells, these cells were eliminated by the immune system, so they produced no tumors. T killer cells and other components of the immune system had evidently retained a memory of their previous antitumor response, protecting the mice against a cancer relapse.
 
Taking part in the study were Drs. Meir Azulay, Gal Cafri and Esther Tzehoval, as well as Ezra Vadai, all of Eisenbach’s lab.

Yeda Research & Development Co., the Weizmann Institute’s technology transfer arm, has filed a patent for the dual immune therapy to accompany cryosurgery. If developed further for use in humans, it may be employed in the future to treat various types of cancer while at the same time preventing metastases.
 
Prof. Lea Eisenbach’s research is supported by the Jeanne and Joseph Nissim Foundation for Life Sciences Research; the Victor Pastor Fund for Cellular Disease Research; the Pearl Welinsky Merlo Foundation Scientific Progress Research Fund; the Lewis Family Charitable Trust; and the estate of John Hunter. Prof. Eisenbach is the incumbent of the Georg F. Duckwitz Professorial Chair of Cancer Research.
Prof. Lea Eisenbach
Life Sciences
English

Working under Pressure

English

Several molecules must work together for proper joint lubrication. In this illustration, the lubricin molecules anchor the long hyaluronan chains to the cartilage collagen network; these hyaluronan chains in turn attach the phospholipid molecules in either single layers (green) or double layers (blue)

 

No man-made lubricant can even come near to meeting the daily demands we make of our joints. As we run or walk, the surfaces of the cartilage in our hip and knee joints slide over one another at high pressures. And they do so over and over, every day, for many decades. Two new studies in the group of Prof. Jacob Klein of the Weizmann Institute’s Materials and Interfaces Department, which appeared in Nature Communications, are helping to solve the puzzle of joint lubrication, and they may, in the future, point the way to treatments for problems with this lubrication, especially osteoarthritis. One in three people suffers from osteoarthritis – the most common form of arthritis – by age 65, and nearly one in two does so by age 85; while younger people often develop the disease after sporting or other accidents; and there is no cure. 

Part of the reason that the joint lubrication system had not been revealed until now, says Klein, is that it forms a very thin layer on the cartilage surface, and its properties are not understood. Moreover, of the many different molecular substances in the joints, several have been proposed to be the main lubrication molecule. The three main molecules that have been suggested for the role of lubricant are phospholipids, hyaluronan (a chain-like, polymer molecule) and lubricin (a protein). The problem is that in some five decades of tests and experiments, none of them alone has performed nearly as well as the body’s actual lubrication system.

 

(L-R) Drs. Nir Kampf and Ronit Goldberg, Prof. Jacob Klein, Dr. Jasmine Seror and Anastasia Gaisniskaya-Kipnis

Klein and postdoctoral fellow Jasmin Seror suggested a way out of this bind: What if all three molecules worked together to lubricate the joints? They devised an experiment using two of the molecules. Working together with Linyi Zhu,a visiting student from the Chinese Academy of Science, Dr. Ronit Goldberg, a senior intern in Klein’s group, and Prof. Anthony Day of the University of Manchester, they created a model lubricating system of phospholipids and hyaluronan placed between atomically-smooth test surfaces made of the mineral mica. They then applied a wide range of loads to the surfaces, mimicking the pressures in actual joints, to see how well the two layers could slide when pressed.

Klein and his group had been studying the first type, phospholipids, because they are an unusual kind of lubricant that relies on water – via so-called hydration lubrication. Phospholipid molecules have two parts: a water-loving head and a couple of long, water-repelling tails. The heads have both a positive and a negative charge, and so they strongly attract water molecules, H2O, which also carry the two types of charge: negative on the oxygen and positive on the hydrogen. The water molecules form a hydration shell around the lipid head; it is this shell that works like a microscopic “ball bearing” and provides the lubrication.

An atomic force microscopy image of mica surfaces coated with hyaluronan molecules to which phospholipids were added. The “bead-necklace” features seen in the image – two of which are indicated by red lines - reveal a lubricating structure consisting of hyaluronan and phospholipids similar to that depicted in the above illustrationThe results of the experiments showed that the combination of the two molecules, hyaluronan and phospholipids, worked remarkably well as lubricants – close to the performance of real joint lubrication. The scientists believe that each plays a different role in reducing friction between the joints’ surfaces. Klein explains: “Although you would not think it of a structure made of water, the hydration shell is quite strong – it is at once incompressible and yet fluid. In other words, it is an excellent lubricant. The long hyaluronan molecules act as a ‘sticky’ layer at the joints' surface, anchoring the phospholipids so that their hydrated heads are exposed on the outside face. This lubricates the sliding. We think that the third molecule – the lubricin – acts as a connector, attaching the hyaluronan molecules to the cartilage.”

 

Just the Right Resistance

In this model, the hydration shells on the phospholipid heads are what ultimately reduce the friction. How well do they work? One way to understand lubrication is to test the viscosity of the fluid substance – how resistant it is to flow. Anyone who has changed the motor oil in a car knows that the correct viscosity (the SAE numbers on the can) is important for keeping the motor running smoothly. The same is true of our joints. Not viscous enough, like plain water, and the substance would be squeezed out of the joint. Too viscous, like gelatin, and it would gum up the works. Hydration shells are a bit more complex, kept in place by the enclosed charges that attract the water molecules. But the question remained: Is their viscosity “just right,” so as to explain the lubrication?

To answer this, in the second study, Klein and his research group, including Liran Ma, Anastasia Gaisinskaya-Kipnis and Nir Kampf, investigated the viscosity of these watery shells. The researchers created simple hydration shells – small positive ions surrounded by water molecules. They then trapped these hydration shells between two atomically-smooth surfaces and measured their viscosity by compressing and sliding those surfaces at different velocities. Their results showed that a fluid made up of these shells is about 200 times as viscous as water – around that of motor oil SAE 30 or cold maple syrup. This viscosity, measured in trapped hydration shells, turns out to provide a good explanation for the observed reduction in friction in the previous study.

Taken together, says Klein, these experiments will lead to both a better understanding of the complex lubrication system that keeps our joints in condition and, hopefully, in the future, to treatments for osteoarthritis.

 

Prof. Jacob Klein's research is supported by the European Research Council; the Charles W. McCutchen Foundation; the Minerva Foundation; the Israel Science Foundation; and the ISF-NSFC joint research program. Prof. Klein is the incumbent of the Hermann Mark Professorial Chair of Polymer Physics.


 

 
Several molecules must work together for proper joint lubrication. In this illustration, the lubricin molecules anchor the long hyaluronan chains to the cartilage collagen network; these hyaluronan chains in turn attach the phospholipid molecules in either single layers (green) or double layers (blue)
Chemistry
English

Bursting with Activity

English
The heart gets all the glory in poetry and aphorisms, but it is the liver that plays a truly central role in orchestrating the entire body’s metabolism. In carrying out its many and varied tasks, the liver faces the tremendous challenge of constantly adjusting to changing conditions – for instance, keeping blood glucose at a steady level despite large mealtime fluctuations in the glucose supply. Weizmann Institute scientists have now revealed that genes in the liver operate in bursts rather than continuously – a pattern that may help optimize their activity, assisting the liver in coping with ongoing challenges. An in-depth understanding of this mechanism may shed new light on the way the liver, and perhaps other organs too, function in health and disease.
 
A similar mechanism had been earlier known to exist in bacteria: When it comes to bacterial genes, the initial stage of activity, the production of a messenger molecule called mRNA, often proceeds in bursts that vary randomly in length, resulting in widely varying mRNA levels in different bacterial cells. This pattern suggests a strategy that has been described as “bet-hedging”: The diversity in mRNA production ensures the survival of at least some of the bacteria, i.e., the ones for which mRNA levels happen to be best suited to the current circumstances.   
Dr. Shalev Itzkovitz
 
In the new study, reported in Molecular Cell, scientists led by Dr. Shalev Itzkovitz of the Molecular Cell Biology Department set out to explore the question: Do genes in the mammalian body resort to the same mechanism; that is, do they produce mRNA in bursts? The scientists used an innovative method developed in Itzkovitz’s lab that has, for the first time, made it possible to visualize individual mRNA molecules as they are being manufactured in intact mammalian tissue. The method combines advanced microscopy with computational approaches.   
 
Using this method, they showed that, just as in bacteria, genes in mouse liver tissue work in random bursts of varying length. The lifetimes of different mRNA molecules, it turns out, also vary; the mRNAs of some genes are longer-lasting than others. The combination of these two variables renders the control of liver gene activity extremely flexible. Thus an mRNA of a particular gene can be generated in long bursts; but if this mRNA itself is short-lived, stopping the bursts will rapidly put an end to the gene’s activity.
 
 
Activity of a glucose-manufacturing gene in mouse liver tissue, viewed under a fluorescence microscope. A high concentration of mRNA (red dots) reveals that this activity is highest near a blood vessel (PP) that bathes the tissue in oxygen-rich blood, essential for glucose manufacture
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
This flexibility can be crucial in performing the liver’s dynamic functions – for example, in regulating blood glucose. As part of ongoing maintenance, the liver takes up extra glucose when its levels are too high, then gradually releases it or synthesizes new glucose when levels drop. As glucose levels rise within minutes after a meal, the synthesis of new glucose must be able to stop instantly. The short-lived mRNA, which quickly disappears from the cell once gene activity is shut down, is perfectly suited to this end.
 
Indeed, the Weizmann study found that the mRNAs of two genes essential for glucose production are extremely short-lived. To compensate for their brief life span, they are produced in longer than average bursts, presumably to reduce the variability among cells caused by the bursts. On the other hand, other mRNAs, with a longer life span, are produced in shorter bursts.
Fluorescence microscope image of mouse liver cells. Cell membranes are in green; the cell nuclei (blue) contain different numbers of DNA strands, from the usual 2 to as many as 8
 
The scientists believe the bursty expression of genes could have evolved because it can protect the DNA from damage: Genes are physically more exposed when active, so by being active only at intervals, rather than permanently, they are less vulnerable to surrounding toxins. This feature is particularly important in an organ like the liver, which is involved in filtering out harmful substances.
 
The scientists also believe that the bursty activity may help explain a baffling feature of many liver cells: the presence of multiple copies of the genome, comprising four or eight DNA strands instead of the usual two. Their proposed explanation goes as follows: The bursts cause mRNA levels to fluctuate at random, but thanks to the extra DNA copies, each of which produces mRNA, this randomness is averaged out among cells. As a result, different liver cells end up producing a particular mRNA in a uniform manner. Indeed, in a “factory” such as the liver, where cells work together towards a common physiological goal, excessive variability among cells caused by such bursts could have been a disadvantage. 
 
Fluorescence microscope snapshots of mouse liver tissue revealing new mRNA, an indicator of gene activity (bright dots marked by white triangles). The lone new mRNA (left) indicates that its gene operates only in infrequent bursts; in contrast, the presence of numerous new mRNAs (right) suggests gene activity that proceeds in long, frequent bursts
 
 
The team that performed this research included Dr. Keren Bahar Halpern, Sivan Tanami, Shanie Landen, Michal Chapal, Liran Szlak, Anat Hutzler and Anna Nizhberg. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 

 
 
Further clarification of the bursty gene expression in the liver may help reveal such mechanisms of faulty liver function as defective glucose metabolism, which leads to diabetes. The scientists have also found indications that bursty gene expression may be found in organs other than the liver, a finding that opens new ways of investigating the control of gene activity in different tissues.
 
 
 
 
Activity of a glucose-manufacturing gene in mouse liver tissue, viewed under a fluorescence microscope. A high concentration of mRNA (red dots) reveals that this activity is highest near a blood vessel (PP) that bathes the tissue in oxygen-rich blood, essential for glucose manufacture
Life Sciences
English

Victory in Numbers

English
We tend to think of cancer as a single disease, but in fact, there are dozens of different cancers, each caused by a specific set of genetic defects. An international team headed by Weizmann Institute researchers has identified a previously unknown defect that occurs in certain breast cancer patients. Under license from Yeda Research and Deveolpment Co., the Weizmann Institute’s technology transfer arm, the pharmaceutical company Merck Serono has started developing a drug that in the future may help prevent metastasis – the spread of the cancer to other organs – in patients with this genetic aberration.

Defects of the type discovered in the new study stem from chromosome abnormalities that in some cases create extra copies of certain genes beyond the two normally present in each cell of the body. One such abnormally multiplied gene, most commonly known as HER2, was discovered more than two decades ago and has already become a target for a drug, Herceptin®, which successfully treats a certain proportion of breast cancer patients with too many HER2 copies.
 
(l-r) Prof. Marcelo Ehrlich, Dr. Silvia Carvalho, Dr. Haim Barr, Prof. Yosef Yarden and Dr. Nir Ben-Chetrit
 

 

 
In the new study, reported in Science Signaling, an international team of researchers headed by Prof. Yosef Yarden of the Weizmann Institute of Science, performed a computerized analysis of some 2,000 genomes of breast cancer patients. Their investigation turned up yet another gene, SYNJ2, which is present in too many copies in a certain number of breast cancers. The study was conducted in the Weizmann Institute’s Biological Regulation Department by Yarden’s graduate student Nir Ben-Chetrit, in collaboration with Yarden’s team member Dr. Silvia Carvalho, Profs. Tsvee Lapidot and Ronen Alon of Weizmann’s Immunology Department, Dr. Haim Barr of the Nancy & Stephen Grand Israel National Center for Personalized Medicine (G-INCPM) and Prof. Marcelo Ehrlich of Tel Aviv University, as well as other scientists and students from Israel and abroad.

The study revealed that women with extra copies of the SYNJ2 gene died sooner than the average for breast cancer patients in the sample, suggesting that excessive copies of this gene could be deadly. The function performed by SYNJ2 in the cell also points to its potential role in decreasing survival: It facilitates the migration of cells – a sometimes useful task, but one that also enables metastasis, the major cause of death from cancer. The SYNJ2 gene encodes an enzyme that operates on the side of the cell facing the direction of movement; this enzyme helps the cell to form extensions called podia that are essential for its migration, as well as enlisting other enzymes that drill a path through walls of arteries and veins, enabling the cancer cells to stream throughout the body.
 
A metastatic breast cancer cell under a fluorescent microscope. The SYNJ2 gene marks “signposts” for two substances (top left, green and red dots), which then enable the cell to drill holes (top right, black dots) in the extracellular matrix, in preparation for metastasis; when SYNJ2’s function is disrupted, one of these substances fails to get recruited and diffuses instead throughout the cell (bottom left, green fluorescence), so there are no signposts (bottom right) and thus the cell cannot drill into the matrix
 

 

When the scientists disabled SYNJ2 in breast cancer cells in a laboratory dish using genetic engineering, the cells’ movement was impeded because they failed to form podia. The researchers then implanted mice with different types of breast cancer cells – those that had a functioning copy of the SYNJ2 gene and those that did not. The cells with the functioning SYNJ2 produced faster-growing tumors and caused more metastases to the lymph nodes and lungs than the ones without the copy.

The next step was to find a prototype for a drug that could be applicable to human patients. With the help of advanced screening technology available at the G-INCPM, the researchers sifted through tens of thousands of small molecules, ultimately identifying one that effectively blocked SYNJ2’s activity. Moreover, it worked with a great deal of precision, targeting SYNJ2 without affecting sibling enzymes, suggesting that it would cause no major unwanted side effects.

This potential therapy, under consideration for further development by Merck Serono, will be aimed at women whose breast tumors have extra copies of SYNJ2 – about four percent of all breast cancer patients. This number may not sound like much, but considering that nearly 1.7 million new breast cancer cases are diagnosed around the world each year, over time the ability to treat this particular type might translate into millions of saved lives.
 

 
Prof. Ronen Alon’s research is supported by the M.D. Moross Institute for Cancer Research; Lord David Alliance, CBE; and Mr. and Mrs. William Glied, Canada. Prof. Alon is the incumbent of the Linda Jacobs Professorial Chair in Immune and Stem Cell Research.

Prof. Tsvee Lapidot’s research is supported by the Helen and Martin Kimmel Institute for Stem Cell Research, which he heads; the Leona M. and Harry B. Helmsley Charitable Trust; the Adelis Foundation; and the Dr. Beth Rom-Rymer Stem Cell Research Fund. Prof. Lapidot is the incumbent of the Edith Arnoff Stein Professorial Chair in Stem Cell Research.

Prof. Yosef Yarden’s research is supported by the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation; the Maurice and Vivienne Wohl Biology Endowment; the Louis and Fannie Tolz Collaborative Research Project; the European Research Council; and the Marvin Tanner Laboratory for Research on Cancer. Prof. Yarden is the incumbent of the Harold and Zelda Goldenberg Professorial Chair in Molecular Cell Biology.


 
 
 
 
A metastatic breast cancer cell under a fluorescent microscope.
Life Sciences
English

Seek and Destroy

English
(l-r) bottom: Tamar Gross, Prof. Lea Eisenbach, Dr. Esther Tzehoval and Zoya Alteber; middle row: David Bassan and Adi Sharbi-Yunger; top row: Lior Roitman, Mareike Grees and Adam Solomon
 
 

Prof. Lea Eisenbach of the Weizmann Institute’s Immunology Department wants to persuade the body’s immune system to fight cancer. The field of cancer immunotherapy – making use of the immune system’s weapons against malignancy – was selected the "Breakthrough of the Year" for 2013 by Science magazine. But much research is still needed to make these therapies safer and more effective.


The idea for immune-based therapy arose from the fact that the immune system constantly clears away cells that have become cancerous or are on their way to turn malignant. But obviously, since many people do get the disease, some cancer cells manage to evade the immune system’s defenses. Eisenbach is devising several strategies – so far tested only in laboratory animals – to outwit those cancer cells, enabling the immune system to seek out and destroy them.


As a first step, she and her team identified a number of proteins and protein fragments that in cancerous tumors are mutated or present in higher than normal amounts. In experiments in mice, these protein fragments were used as a vaccine that eliminated lung cancer metastases and other tumors, including those produced in mice by various human cancer cells.  
 


 
 
back immune cells
 

To further develop anti-cancer vaccines, Eisenbach’s team and their collaborators in the MIGAL Research Institute in Kiryat Shmona are teaching the immune system to detect evasive cancers with the help of dendritic cells, the system’s sentinels that normally alert it to the presence of viruses, bacteria and other dangers. The scientists achieve this goal by outfitting dendritic cells with the above-mentioned protein fragments fused with additional molecules that trigger an immune response. By making use of RNA molecules, which convey genetic information but are easier to manipulate than the genes themselves, they have been able to engineer several of the tumor’s genetic features into a dendritic cell, thus increasing the chance that the tumor will be detected.


The engineered dendritic cells can then serve as a vaccine: They migrate to lymph nodes, where they activate the immune system against cancer, priming it into attacking a tumor it previously did not recognize. In a recent review in the Annals of the New York Academy of Sciences, the scientists noted that studies in mice point to the potential efficacy of this approach against metastases of melanoma, particularly those that move to the lungs.

 
Brain immune cells
 
But the best way to fight cancer is to prevent it. In  another project, Eisenbach’s team is focusing on a family of genes called IFITM that encode a group of interferon-activated genes believed to play a protective role against inflammation of the colon, which, in turn, may contribute to cancer. In a study in mice, the scientists showed that in the absence of IFITM genes, the incidence of inflammation in the colon did indeed increase, and with it the risk of colon cancer. Because in humans IFITM genes can be present in different variants, it’s possible that people with certain versions that are relatively ineffective at warding off inflammation are more prone to colon cancer than those with optimally functioning IFITM genes. This research may in the future help develop markers for identifying people at increased risk of colon cancer.
 
 
Brain cells
 
In yet another avenue of research, Eisenbach has focused on a phenomenon called “split immunity” to investigate potential ways of treating glioblastoma, an extremely malignant brain tumor. In research conducted by Dr. Ilan Volovitz, then a student in Eisenbach’s lab, with departmental colleague Prof. Irun Cohen, she and her team found that glioblastoma cells produced highly malignant tumors in rats when implanted into their brains, but these same cells were eliminated by the immune system when implanted in the backs of the rats. When the glioblastoma cells were implanted in the brains of rats that had already rejected tumors in their backs, however, no tumors formed there: Apparently, the previous injections in the back had somehow primed immune T cells to effectively fight the brain tumors.
 
As reported in the Journal of Immunology, the researchers further discovered that existing tumors in the brains of rats could be effectively treated by injecting the animals with T cells drawn from other rats that had cleared these tumors in their backs. Dr. Volovitz and Prof. Zvi Ram, both of the Tel Aviv Sourasky Medical Center, are now continuing to develop this approach.


 

Light microscope images of mouse tissue samples (tissue cells are blue): When tumors are implanted in the back (left), they are infiltrated by immune cells (pink) and eliminated; tumors implanted in the brains of the mice (middle) are infiltrated by very few immune cells and continue to grow; normal brain tissue (right) contains no immune cells at all
 
 

Prof. Lea Eisenbach's research is supported by the Jeanne and Joseph Nissim Foundation for Life Sciences Research; the Victor Pastor Fund for Cellular Disease Research; the Pearl Welinsky Merlo Foundation Scientific Progress Research Fund; the Lewis Family Charitable Trust; and the estate of John Hunter. Prof. Eisenbach is the incumbent of the Georg F. Duckwitz Professorial Chair of Cancer Research.
 

 


 

 


 

 
 
(l-r) bottom: Tamar Gross, Prof. Lea Eisenbach, Dr. Esther Tzehoval and Zoya Alteber; middle row: David Bassan and Adi Sharbi-Yunger; top row: Lior Roitman, Mareike Grees and Adam Solomon
Life Sciences
English

Use It or Lose It

English
3-D computer model of a fruit fly larva muscle fiber with multiple nuclei

 
Everyone knows that exercise can build muscle; without exercise, muscles quickly lose their strength and volume. These facts have been common knowledge for so long, it might come as a surprise to find that very little is known about the molecular mechanisms behind them. How indeed does physical activity cause muscles to strengthen?
 
Prof. Talila Volk
 
Weizmann Institute scientists have now proposed an explanation. Research conducted by the team of Prof. Talila Volk in the Molecular Genetics Department suggests that the long, cylinder-shaped muscle cells – the muscle fibers – contain proteins that function as biological “mechanosensors.” These spring-like, elastic proteins are connected to the cellular supporting structure – the cytoskeleton – on the one side, and on the other side, to the cell’s nucleus. Muscle contractions exert mechanical pressure on the cytoskeleton, which, in turn, puts pressure on these sensor proteins, causing them to transmit a signal to the nucleus.

This signal presumably alters the internal architecture of the chromosomes within the nucleus, changing gene expression – that is, the activity of genes – in the affected portion of the DNA. As a result, certain genes become activated, prompting the release of proteins that make up the filaments responsible for the contraction of the muscle fiber. In addition to contraction, the proteins fortify existing filaments and help produce new ones, building up muscle mass.
 
Muscle fibers of a fruit fly larva viewed under a confocal microscope: A normal fiber has normally-shaped, properly distributed nuclei (A), whereas the nuclei of fibers with mutated MSP-300 or its interacting proteins Klar and Klaroid are distorted and distributed abnormally (B, C and D)
 
 
In this manner, exercise triggers a strengthening of the muscle. But because muscle-building proteins have a high turnover, the signal for their production must be periodically repeated. If, in the absence of exercise, the muscle fails to contract for a while, the mechanosensors no longer send their signals to the nucleus, ultimately leading to a loss of muscle mass.

Evidence for this explanation comes from a series of studies that Volk and her team – including research students Hadas Elhanany-Tamir and Miri Shnayder, and postdoctoral fellow Dr. Shuoshuo Wang – conducted in fruit fly larvae. As reported in the Journal of Cell Biology, the scientists have identified a fruit fly protein, called MSP-300, whose shape and mechanical properties make it perfectly suited to serving as a mechanosensor. MSP-300 forms a ring around the nucleus, with numerous radial extensions connected to the cytoskeleton. It has elastic properties, as would be required from a mechanosensor; but on the other hand, it operates in close cooperation with two other proteins that help create rigid scaffolding around the nucleus, protecting it from muscle contractions. By introducing mutations into MSP-300, the scientists showed that it is indeed essential for maintaining muscle mass. The effect of the mutations on the fly’s muscles was devastating: The muscle fibers thinned out and their nuclei were deformed and clumped together abnormally. As a result, the muscles didn’t work properly, so the larvae couldn’t crawl and adult flies couldn’t fly.
 
The MSP-300 protein, viewed here under a confocal microscope, forms a ring (red in the left image, green on the right) around the muscle cell nucleus, with extensions to the cytoskeleton
 

Because human muscle cells contain proteins equivalent to MSP-300, suggesting they may also function as mechanosensors, these findings can shed new light on the connection between physical activity and a healthy build-up of muscle in humans. A better understanding of this connection may in the future lead to improved ways to prevent muscle loss resulting from aging, forced inactivity as in paralysis, or such disorders as muscular dystrophy.

Prof. Talila Volk's research is supported by Erica A. Drake and Robert Drake. Prof. Volk is the incumbent of the Sir Ernst B. Chain Professorial Chair.
 

 
Muscle fibers of a fruit fly larva viewed under a confocal microscope: A normal fiber has normally-shaped, properly distributed nuclei (A), whereas the nuclei of fibers with mutated MSP-300 or its interacting proteins Klar and Klaroid are distorted and distributed abnormally (B, C and D)
Life Sciences
English

The Coin Toss Paradox

English

Alice and Bob are two fictional characters who love to play games with a mathematical bent. Here, for example, is one game they play: They toss coins and figure their odds of winning. What are their chances, for example, if Bob tosses a coin and Alice has to guess which side is up, and then Alice flips a coin and Bob has to guess, and they play as a team, so that both must be correct to win?

Prof. Irit Dinur
 
“There are many variations on this game,” says Prof. Irit Dinur of the Weizmann Institute's Computer Science and Applied Mathematics Department. “The different versions and their mathematical solutions give us insight into how information is shared in the real world, in computing, in different branches of mathematics and even in the world of quantum mechanics and quantum communication.” Dinur and her colleagues recently used one variation on this game to reveal how it might be played under “quantum” rules.These rules are very relevant to those who hope to use the strange properties of quantum mechanics to construct new ways of using information – for example in quantum communication, a technology that is already under development today.

In the coin-toss game, says Dinur, one might think that after both had flipped and guessed they would have a 25% chance of winning the game, since each had a 50% chance of guessing correctly. But there is a clever strategy that Bob and Alice can use: If each guesses that the other has flipped exactly the same as their own coin flip, they raise their odds to 50%.  

Now imagine Bob and Alice continue playing the game – they must guess correctly every time to win. Are there more clever tricks they can use to increase their chances of winning? The answer, sadly, is no. As they continue to flip coins, their odds decrease round by round: Mathematical calculations reveal that in all cases, as the game is repeated, the chance of winning quickly approaches zero.  
 

Changing the rules

When Dinur and her colleagues changed the rules yet again, entering the world of quantum mechanics, things become more complicated.

Alice and Bob’s information was now in the shape of quantum particles; to understand the game, one must understand a few basic principles of quantum mechanics. First, there is superposition: A quantum particle can be in more than one state at a time. But when it is observed or measured, it “collapses” into a single state. So the possible information held by each superimposed particle can be much greater than “yes” or “no.”

Alice and Bob’s particles were also entangled: When two particles are entangled in a quantum setup, they can be placed at a distance from each other, but their states remain in perfect sync, so that any change in the state of one results in an instantaneous change in the state of the other.

Both of these ideas were proposed in the early 20th century, and Einstein was famously opposed to the concept of entanglement, calling it “spooky action at a distance.” A paper he coauthored in 1935 presented the “EPR paradox,” which suggested that, because information cannot travel between the particles faster than the speed of light, there must either be some hidden variables controlling the process, or else the outcome is already “known” before the measurement is performed. Today, entanglement has been proven experimentally, and Einstein’s hidden variables did not pan out. But the paradox remains: How do two particles “share information,” coordinating their states with no time lag?  
 
Game plans
 
 
In the entanglement game, if Alice measures her superimposed quantum bit, collapsing it into a particular state, then Bob’s entangled particle must immediately assume a corresponding state. So entangling bits of information could be seen as cheating. Would Alice and Bob win every time, the results seemingly predetermined, or would the game still be subject to other, less spooky, laws of play? In other words, how would the conditions of the EPR paradox apply to the game?

Dinur and her colleagues showed, mathematically, that a game based on entangled bits of information will eventually follow the pattern of the other games: As the rounds are repeated, the chances of winning will drop significantly. Entanglement may give them some advantage in the beginning, as Bob now has a piece of information about Alice’s knowledge. But, like the coin toss, Alice’s information will still be random: Measuring will collapse her quantum system to a particular state, but she will not be able to control or predict what that state will be. Thus while the odds for each individual round will be better, the pattern will remain the same, moving toward zero as rounds are added to the game. In other words, entanglement would only be a partial cheat, at best, and the EPR paradox is not quite the paradox it seemed.  

If quantum communication becomes a reality, Alice and Bob will be able to use it to ensure encryption – for example, to detect any interference in messages sent from one to the other. Although the technology is still far in the future, it will rely on today’s mathematics to set the rules and the limits on its operation.  

 

 
Game plans
Math & Computer Science
English

It Ain’t Over Till It’s Over

English
More than half of the human body, by weight, is made up of water, yet scientists studying body chemistry have traditionally regarded water as a mere bystander – a neutral environment for various biochemical reactions. But that is starting to change: Current research is showing that water plays an active role in many bodily processes. In a new study reported in the Proceedings of the National Academy of Sciences (USA), researchers from the Weizmann Institute of Science, Ruhr University Bochum in Germany and Torrey Pines Institute for Molecular Studies in Florida reveal a surprising performance by water molecules – one that may open up new avenues for the faster and more effective design of drugs for cancer, autoimmune diseases and other disorders.
(l-r) Drs. Moran Grossman and Inna Solomonov, and Prof. Irit Sagi
        

 

 
 
The study focused on a biochemical reaction that routinely occurs in the lungs, liver, skin and most other body tissues. Enzymes carry out this reaction to dismantle collagen, the main component of the scaffolding known as the extracellular matrix, which provides structural support for cells. Such enzymatic dismantling is part of ongoing “maintenance” remodeling, in which the matrix is continuously broken down and rebuilt. Using a combination of three technologies – fluorescence and X-ray, and infrared terahertz spectroscopy – the team of Weizmann Institute’s Prof. Irit Sagi, together with teams headed by Profs. Martina Havenith and Gregg Fields, monitored the dismantling of collagen by enzymes in a laboratory dish.

In classical theory, enzymatic reactions are described by a curve: The reaction proceeds at a rate that increases at first and then levels off, continuing until the chemical broken down by the enzyme runs out. But in the new study, the scientists were amazed to discover that even after all the collagen was broken down, there were aftereffects of the reaction that persisted. Much like ripples that continue to spread after a stone is thrown in a pond, surrounding water molecules remained in motion, continuously altering their hydrogen bonds in response to the structural changes that had occurred on the surfaces of the enzyme and collagen during the reaction. The fact that these water dynamics lasted longer than the reaction itself may be an aftereffect that probably facilitates further chemical and biochemical processes in the tissue. In some of the experiments, the collagen was completely broken down within a second, whereas the water dynamics persisted for at least five times as long.
 

Water dynamics near an enzyme-collagen complex: The rates at which water molecules exchange hydrogen bonds with one another depend on the distance from the active site of the enzyme (gray) and on the type of collagen interacting with the enzyme; the different rates create a gradient of water motions, ranging from extremely fast (red) to very slow (blue and light-blue)

 

 

 

 

 

 

 

 

Moreover, the scientists found that the water dynamics differed depending on the type of collagen and the resulting products of the chemical reaction. This finding suggests a close connection between the water and the reaction.

This is a previously unknown biological function of water; it suggests that to obtain an in-depth understanding of enzymatic reactions in the human body, it is essential to clarify exactly how they are affected by the surrounding water molecules. Beyond clarifying the fundamentals of body chemistry, such an understanding may be crucial for the development of new drugs. For example, excessive collagen dismantling facilitates the spread of malignant cells in cancer and of inflammation-causing cells in certain autoimmune disorders. It might be possible to develop new drugs by harnessing the mechanistic insights derived from observing water-protein interactions. On a more general level, the design of a wide variety of drugs may be rendered more effective by incorporating the water-protein dynamics into computer-based drug design programs.

Prof. Sagi’s team in the Weizmann Institute’s Biological Regulation Department included Drs. Benjamin Born and Inna Solomonov. The German team, headed by Prof. Martina Havenith from the Department of Physical Chemistry, consisted of Dr. Moran Grossman, a former PhD student at the Weizmann Institute, Dr. Jessica Dielmann-Gessner and Dr. Valeria Conti Nibali. Also taking part in the study was Prof. Gregg Fields of the Torrey Pines Institute in Florida, USA.
 
Prof. Irit Sagi's research is supported by the Spencer Charitable Fund; the Leona M. and Harry B. Helmsley Charitable Trust; Michael and Rhoda Ambach; Cynthia Adelson, Canada; Dr. Mireille Steinberg, Canada; and the Leonard and Carol Berall Post Doctoral Fellowship. Prof. Sagi is the incumbent of the Maurizio Pontecorvo Professorial Chair.
 
Water dynamics near an enzyme-collagen complex: The rates at which water molecules exchange hydrogen bonds with one another depend on the distance from the active site of the enzyme (gray) and on the type of collagen interacting with the enzyme; the different rates create a gradient of water motions, ranging from extremely fast (red) to very slow (blue and light-blue)
Life Sciences
English

Cells with an Edge

English

Within the pancreas are little spherical structures called islets of Langerhans – a name that might conjure up images of cells lazing on a remote beach, sipping tropical drinks. That picture could not be further from the truth: The so-called beta cells, the major component of these islets, are some of the busiest, most connected cells in our bodies. They do sip, but it is the sugar in our bloodstream they taste; they then secrete insulin back into the bloodstream to regulate the metabolism of that sugar. And, according to new Weizmann Institute research, these cells submit to a very tight organization. The study, which appeared in Cell Reports, revealed, for the first time, an image of beta cells that are “edgy.” The details of how beta cells are shaped may change our understanding of how they function and lead to new insights into how glucose is regulated in the body.

 

Mouse islet of Langerhans; insulin-containing vesicles within beta cells are shown in white
 
Beta cells are those affected by diabetes: In type 1 diabetes they are attacked by an autoimmune response in the body, and in type 2 diabetes the body cells do not respond to the insulin they secrete, which eventually leads to their dysfunction. The beta cells are a minority in the pancreas, but the island-like arrangement within the “sea” of pancreatic cells enables them to function as an organ within an organ. A closer look at the arrangement of cells in an islet of Langerhans shows that they assemble in “rosette” patterns around veins running through the pancreas, with small arteries encircling the rosettes.
 
Prof. Ben-Zion Shilo, research student Erez Geron and Dr. Eyal Schejter of the Molecular Genetics Department wanted to zoom in a bit closer – to the molecular arrangements of these cells. Many had assumed that the cells were polar – that they concentrate some functions on one side and others on the opposite side. Polarity, according to this view, would enable the beta cell to compartmentalize its activities, possibly sensing on one side and secreting on the other. In addition, beta cells are related to other polar cells – for example, those that line the intestine. But no one had actually managed to observe clear hallmarks of polarity in beta cells.  

To investigate, Geron studied proteins on the cells’ outer membranes – where sensing and secretion take place. Isolating individual islets of Langerhans from a mouse pancreas, he inserted a fluorescent protein into several of their cells. The team then devised a technique that allowed them to visualize filamentous actin, a major component of the cellular cytoskeleton, just within a few individual cells of each islet, thus providing good contrast right at the cells’ contours. Since islets of Langerhans can be kept alive in the lab dish, the researchers were able to observe the shapes of beta cells at work.
 
 

                                                           Cell design on a line

Prof. Ben-Zion Shilo
 
What they saw surprised them: Rather than a polar arrangement, the actin in the membrane formed stiff, linear edges, like those on a cube, along the length of the cell, giving it the appearance of an angular tent with poles. But these lines were more than just support poles: Numerous thin protrusions extended from the cell along these lines, and membrane features congregated along them – signaling proteins, channels for letting in glucose, others for trading short messages encoded in calcium ions with neighboring cells, and even outlets for secreting insulin.

Why do these particular cells reject their polar heritage and go for edgy shapes? Why would they take all of their functions – sensing glucose, releasing insulin and intercellular communication – and align them together along these cell edges? Shilo says that they do not yet have all the answers, but he and his group have some definite clues. For example, they think that keeping the sensing and secreting machinery close together could increase efficiency and/or cut down on response time.

Further research will be needed to understand the exact function of the edges, but Shilo says the study points to an important role in cell-to-cell communication. Indeed, one of the major implications of these findings is that, when it comes to cell design, relations between neighboring beta cells are critical. It is communication between cells that creates the tent-like shape in the first place: Beta cells that are cut off from the others soon lose their edges. And if the “tent poles” help facilitate communication, then the protrusions along their length are likely to play a role in passing messages back and forth, possibly by helping align the membranes’ pores, which are just big enough to admit the calcium ions. “These findings suggest a mechanism for beta cells to function in unison,” says Shilo. “Coordinating their actions might help prevent random responses by single cells.”

Although the work was done on mouse pancreatic cells, there is already some evidence that human beta cells also have edges. These findings, says Shilo, shed light on the ways that insulin-producing beta cells function, and they could lead to new ways of thinking about the normal complex, coordinated activity of these cells, which play such a central role in our health.
 
Prof. Ben- Zion Shilo's research is supported by the M.D. Moross Institute for Cancer Research; the Carolito Stiftung; and the Mary Ralph Designated Philanthropic Fund. Prof. Shilo is the incumbent of the Hilda and Cecil Lewis Professorial Chair of Molecular Genetics.

 
 
Mouse islet of Langerhans; insulin-containing vesicles within beta cells are shown in white
Life Sciences
English

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