International Biopharmaceutical Association Publication

 

CANCER-FROM MOLECULAR BIOLOGY TO DRUG DEVELOPMENT

 

Sonal Gupta

sg52003@gmail.com

 

Abstract: Cancer is a condition in which control of cell division is lost and cells start to divide uncontrollably.  These cells attain the ability to detach from their organ of origin and invade other tissues, a process called metastasis.  Metastasis is the process which differentiates malignant tumors from benign tumors.  Finding a cure for cancer with minimal side-effects has been a huge challenge for scientists all over the world.  The biggest hurdle in finding a cure for cancer is the nature of disease which involves multiple levels of complexity.  The complex nature of disease begins with its causative agents.  Unlike other diseases, a single agent could not be linked with the cancer.  Factors leading to cancer may be as diverse like high-energy radiations, certain chemicals, biological agents like certain bacteria and virus, genetics makeup of the individual and even life-style.  But one common theme among all of these agents is their ability to alter genetic material of the cell.  There are more than 130 genes, mutation in which may lead to cancer. While most diseases affect a single organ-system of the body, cancer is not limited to only one organ-system.  Despite the complex nature of the disease, currently available treatments may significantly better the condition of the patient.  Present treatments include chemotherapy, surgery, biological therapy and radiation therapy etc.  These treatments are not very specific to cancer cells and affect normal cells too causing significant side-effects.  But enormous research is being done for the cancer treatment and the results are very promising. Better understanding of the molecular biology of cancer cells and advancement of techniques like micro-array, led to the discovery of more specific treatments which exclusively affect cancer cells without any serious harm to normal cells.  These new approaches include targeted therapies, gene therapy, angiogenesis inhibitors and cancer vaccines.  Some of these new treatments have already been approved by FDA while many are in pre-clinical or in clinical phase.

 

Cancer: Introduction

Cancer is one of the most dreaded diseases and numbers of its victims are next only to the cardiovascular disease. Approximately 1500 people die each day in the United States due to cancer. About 3,400 people are diagnosed with cancer each day in the U.S.  For a very long time cancer has been considered a synonym to death so trauma of cancer is not only physical but it put enormous amount of mental pressure too.

Scientific term for cancer is malignancy.  It is the condition in which control of cell-cycle is lost and cells start to divide uncontrollably.  These deadly cells may detach from their organ of origin and may spread to other organs too. This process of invading other organs is called metastasis and it is the most problematic quality of malignant cells while looking for a cure for malignancy.  Metastasis is the process which differentiates malignant tumors/malignancy from benign tumors, which are self-limited and do not invade other tissues and for most part are harmless.

Though cancer may attack any organ of the body at any age including fetus its risk increases with the age.  Organs which are more commonly affected by malignancy are liver, breast, colon, lining of stomach, kidney, cervix, prostate and bladder etc.

 

Causes of Cancer:

Invention of a cure for cancer has proved difficult because there is no single cause for cancer.  The risk-factors for cancer are as diverse as physical inactivity to hereditary.  The agents which are known to stimulate the process of cancer are called carcinogens.  Here is a list of certain risk-factor which increases the chance that a person will develop cancer:

  • Age:  Though cancer is not limited to only older people the chances of getting cancer is much higher in people over 65 year of age.
  • Tobacco:  Tobacco use is the most preventable cause of death of cancer.  Using tobacco products or regularly being around tobacco smoke increases the chance of getting cancer.  Smokers are more likely to develop cancer of lung, larynx, throat and esophagus.  People who use smokeless tobacco (snuff or chewing) are at increased risk of cancer of mouth.
  • Physical agents:  Ultraviolet radiations may cause DNA damage and thus stimulate the chances of getting cancer.  Sources of UV radiations are sun, sunlamp and tanning booths.  UV radiations cause early aging and skin damage that may lead to skin cancer.

Apart from UV radiations, other ionizing radiations like radon gas and X-ray may also increase chances of getting cancer. Radon is a radioactive gas that forms in soil and rocks and people working in mines may be exposed to that.  It increases chances of getting lung cancer.  Medical procedure like X-ray and radiation therapy are also a source of ionizing radiation but the chances of getting cancer are much lower in these type of radiations and benefits always significantly overweigh the small risk.

  • Chemical Agents:  Chemicals like benzene, benzidine, asbestos, cadmium, nickel, vinyl chloride may cause cell damage and lead to various kind of cancers.  The chances of getting cancer are highest for the people who come in contact with these chemicals on a regular basis such as at workplace.  
  • Biological Agents:  Certain viruses and bacteria may cause to develop certain cancers.  Here is a list of some viruses and bacteria which are known to be linked with certain cancers: 
  1. Human papillomaviruse (HPV) infection is the main cause of cervical cancer.  Most women who develop cervical cancer have been infected with a particular strain of HPV at some point of their life.
  2. Hepatitis B and hepatitis C viruses:  Chronic infection with hepatitis B or hepatitis C virus may lead to cancer of liver.
  3. Human T-cell lymphoma virus (HTLV-1): Infection with this virus may increase the chance of getting lymphoma and leukemia.
  4. Epstein-Barr virus (EBV):  Infection with EBV has been linked to an increased risk of getting lymphoma.
  5. Human herpes virus 8 (HHV8):  This virus is a risk factor for a rare type of cancer called Kaposi’s sarcoma.
  6. Helicobacter pylori:  This bacteria cause stomach ulcer.  Persistent infection may aggravate to develop stomach cancer and lymphoma in stomach lining.
  • Hormone Therapy:  Side effects of menopausal hormone therapy include increased chances of getting breast cancer.
  • Family history of cancer:  Some cancers like melanoma, cancers of breast, ovary, prostate and colon, have strong genetic link.  Most cancers develop because of mutation in genes.  A series of mutations are required to turn a healthy cell into a cancerous cell.  These mutations may be hereditary or acquired. If cancer is running in a family many mutations may be acquired genetically.  Due to already pre-disposed genes running in the family a few mutations or even a single mutation by a carcinogen may lead to the development of cancer.
  • Lifestyle:  People with poor eating habits, overweight and lack of sufficient physical activity may lead to increased chance of getting cancer. People whose diet is high in fat have an increased risk of cancers of the colon, uterus, and prostate. Lack of physical activity and being overweight are risk factors for cancers of the breast, colon, esophagus, kidney, and uterus 

Molecular biology of Cancer:

Though a single agent could not be linked to all types of cancer but all of these agents cause some sort of changes in genetic makeup. There are two important classes of genes which are critical in the development or suppression of cancer; these genes are called proto-oncogenes and tumor suppressor genes.  In an easy sense proto-oncogenes can be compared to gas-pedal of the car which causes cells to divide while tumor-suppressor genes can be compared to the break pedal of the car which controls when to stop the cell division.  In almost all types of cancers, a series of independent mutations are accumulated in a step-wise progression in several proto-oncogenes and tumor suppressor genes; this assembly of independent mutations is responsible for the transition from normal cell to cancerous cell.  This explains the reason why cancer is more prevalent in older people because cells in their body have had more time to accumulate mutations which are responsible for causing cancer.  Because of their critical role in the development of cancer, proto-oncogenes and suppressor genes will be discussed at a length here:

Oncogenes:  In normal cells proto-oncogenes genes code for the proteins that control cell division and regulate how often a cell divides and the degree to which it specializes.  Mutation in a proto-oncogene may cause it to become an oncogene (gene responsible for causing cancer).  More than hundred oncogenes have been recognized by now.  Unlike proto-oncogenes, oncogenes are permanently activated, as a result of which control of cell-cycle is lost and cell divides too quickly. 

Growth of a normal cell starts with a growth factor, which binds to a growth factor receptor located on the cell surface.  The signal from the receptor to the nucleus is sent via a signal transducer.  As a response to this signal a transcription factor is produced this initiates the cell to divide.  If any abnormality is detected during the cell division, cell is made to commit suicide by programmed death cell regulators.

On the basis of their action on the cell cycle, oncogenes have been divided into five different classes: growth factors, growth factor receptors, signal transducer, transcription factors and cell death regulators.  As oncogenes are permanently activated, cells divide continuously and even in the presence of any abnormality cells do not die as cell death regulators do not turn off.

Tumor suppressor genes:  These are genes which code for the proteins which make cell to slow down its division, repair DNA and tell cells when to die.  About thirty tumor suppressor genes have been discovered by now.  The main functional abnormality caused by the mutations in oncogenes and suppressor genes differs in their state of activation.  Mutations in oncogenes leading to the cancer make them permanently on while in tumor suppressor genes theses mutations cause them to permanently turn off. 

On the basis of their function tumor suppressor genes have been divided into three classes: genes controlling cell-division, genes that repair DNA and cell suicide genes.  As mutations in these genes make them permanently inactive cell-division continues uncontrollably, DNA does not get repaired in case of any damage and cells containing any abnormality do not undergo the process of cell death or apoptosis.

Apart from mutations in proto-oncogenes and suppressor genes, other changes like structural changes in genetic material and epigenetic changes may also lead to cancer.  Structural changes are chromosomal level changes in which a portion of the chromosome may undergo processes like duplication, translocation, inversion, gene-amplification and anupleoidy.  While epigenetic changes do not actually change the nucleotide sequence, the expression of the gene is altered by the addition of the methyl group in the nucleotide or by the addition of acetyl group on the histone protein making the expression of gene less and more active respectively.

 

Problems in finding a cure for Cancer:

Cancer is a behemoth of a challenge for the scientists all over the world.  More than 1.7 million papers have been published on cancer research but still no satisfactory cure without tons of side-effects could be found for all types of cancer, though a lot has been understood about the mechanism and biology of cancer.  And it helped for the development of various lines of treatments which can be used individually or in combination for the treatment of cancer.

Basic problems in finding a cure for cancer are multifold because of the complexity of cancer at all levels.  Unlike most other diseases the causative agent of cancer is not known.  A single agent could not be linked even to one type of cancer.  The other problem is the diversity of changes in genetic material which may lead to cancer.  There are about hundred oncogenes and about thirty suppressor genes in a cell.  Even a single nucleotide change in these genes may be a stimulant towards the cancer.  Normally cancer develops following a series of changes in the genes, so there are numerous possible combinations in which these changes may occur leading to cancer.  Another hurdle in finding a cure to cancer is the ability of cancerous cells to metastasize.  These cells may degrade binding proteins and invade other tissues and cause cancer to spread from one organ to other.  By the time cancer is diagnosed in the primary organ it has already spread to the secondary organs making condition even more complicated.     

 

 

Treatment of Cancer: Present approaches

Though a definite cure could not be found for cancer, there are methods of treatment which increase life expectancy as well as provide better quality of life.  The benefit depends on the stage of the cancer and the organ involved.

Here is a list of types of treatment available for cancer:

1.      Surgery: This is the first line of treatment for many solid tumors.  If diagnosed at an early stage, surgery alone may be sufficient to cure the cancer by removing all cancerous cells.  Depending on the size, location and type of cancer surgery is often used in combination with radiation or chemotherapy.

2.      Radiation therapy:  This therapy utilizes high energy radiations like x-rays to kill cancerous cells or shrink tumors.  Radiation therapy destroys cells in the area under treatment by damaging their genetic material.  Although radiation therapy damages both normal and cancerous cells, most normal cells are able to recover from the radiations.  This therapy is often used in combination with surgery or chemotherapy.

3.      Chemotherapy:  Chemotherapy is a type of treatment that uses drugs to kill quickly dividing cancer cells.  But at the same time it also kills quickly dividing normal cells like those present in the lining of mouth and intestine and those that cause hair to grow.  Damage to healthy cells is responsible for the side effects of chemotherapy.  Depending on the type of cancer and how advanced it is chemotherapy may cure, control or ease cancer symptoms.

4.      Biological therapy or Biological Response Modifier (BRM) therapy: BRMs are the compounds which are used to treat cancer by augmenting body’s immune system.  These compounds target immune system cells and cause increased production of cytokines and immunoglobulin.  These compounds too have side effects when used in high doses required for cancer treatment and their effectiveness depends on the type of cancer.

5.      Hormone therapy:  Although abnormal, cancer cells retain their ability to respond to hormonal signals.  The idea behind this therapy is to starve cancer cells of these hormone signals which otherwise stimulate these cells to divide.  Cutting off the hormonal supply has side effects on normal cells too.  Not all cancers respond to hormone therapy.  Some types of breast cancer, uterine cancer and prostate cancer are sensitive to hormone therapy.

6.      Antibody therapy: Monoclonal antibodies can be used to inhibit the growth of cancer cells by different approaches.  These antibodies perform their action either by stimulating cancer cell killing or blocking a growth receptor.  These antibodies may also be tagged with toxins which would eventually kill cancer cell.

7.      Alternative approaches: These approaches include ancient knowledge of Indian Yoga, Ayurveda and Chinese feng-shui etc.  Though modern science does not give an explanation for their mode of action, but some people claim to get benefit from these approaches.

 

Future Drug development in Cancer Treatment:

  1. Targeted therapy: The most important theme in the development of more specific cancer drugs is this that these drugs target only cancer-specific processes instead of processes common to all cells.  Because these drugs are not toxic to all cells, and because they only affect cancer cells, they are highly specific with minimal side effects. The specificity of current drugs has one drawback. Blocking a single pathway in a cancer cell may slow down the growth of cancer, but this often is not sufficient to kill it. Therefore, many specific cancer drugs are currently being used together with traditional chemotherapy. The combination of a highly specific cancer drug that is able to attack a tumor's weaknesses and standard chemotherapy deliver a powerful attack on the tumor.  And this strategy may prove to be an excellent means of treating cancer.
  2. Tumor vaccines:  Cancer vaccines are either intended to prevent occurring cancer or to treat existing cancer.  Therapeutic vaccines are designed to stimulate the immune system to recognize and kill cancer cells without harming normal cells. While prophylactic vaccines are administered in healthy individuals to stimulate the immune system to attack cancer-causing viruses and prevent viral infection.  Till now, two cancer vaccines have been approved by FDA to prevent viral infections that may lead to cancer: hepatitis B vaccine that prevents infection with hepatitis B virus which may cause liver cancer and Gardasilwhich prevents infection with two types of human papillomavirus that together account for about seventy percent of cervical cancer.
  3. Gene therapy:  This is an emerging field for cancer therapy.  More than half of the gene therapy clinical trials are for the treatment of cancer.  This therapy uses the concept of gene transfer into cancer cells.  These genes may aid in the treatment of cancer by following methods:

Immuno-modulation: Introduction of genes which enhance the immune response of the body.

Targeting genetic lesions in tumor cells:  Introduction of genes which code for the reverse sequence of faulty genes in tumors.  mRNA of the faulty gene and the gene introduced in gene therapy will form Watson-Crick base pair.  These double stranded mRNA can not be translated and will block the expression of specific genes responsible for the disease.

Suicide genes:  These genes are transferred into cancer cells and then a pro-drug (inactive form of a drug) is given to the patients.  This drug is converted into its active form only in the cells containing the transferred gene.  And these cells die because of the active form of the drug.

These are the most popular strategies with the gene therapy for the treatment of cancer, but other strategies are also in the pre-clinical or clinical phases. 

  1. Angiogenesis inhibitors:  Tumor can not grow or spread without the formation of new blood vessels (angiogenesis).  Scientists are looking for synthetic and natural chemicals to stop angiogenesis in tumor cells. Recently FDA has approved angiogenesis inhibitor bevacizumab (Avastin®) for its use in the treatment of colorectal cancer.
  2. Combination of genomics with other strategies:  Currently most available approaches for cancer treatment try to treat patients with the same kind of therapy.  But every individual is unique and as described above because of the complex nature of cancer, response of patients varies significantly for the same treatment.  Cancer of some patients may respond very positively while for others their cancer may remain unaffected for the same type of treatment.  This difference in responses is because of the unique genome of every individual.  This approach proposes the use of personalized treatment for each patient.  With the use of the advanced techniques like microarray, one can look through the genetic expression profile of an individual and it can be predicted if the person is likely to respond to one kind of treatment or not.  As most cancer treatments are very expensive this approach may help in reducing the cost of treatment and will save even more precious thing in cancer treatment that is the TIME. Doctors will not waste time in treating the patients with the treatments to which he/she is unlikely to respond. Not only in the treatment, this approach has, a lot of potential in the prognosis of cancer too.  By looking at the genetic profile of an individual susceptibility for cancer can be predicted.  By improving life-style and minimizing the exposure to risk factors chances of getting cancer in that individual may be reduced significantly. Though currently this approach is at the clinical trial phase and still lot of bench research is being done to identify more and more bio-markers, this approach has enormous potential in the treatment of cancer in near future.

 

 

 

 

 

References:

www.cancer.gov

www.cancerquest.org   

www.cancer.org  

www.krctraining.com

http://www.medschool.lsuhsc.edu/

http://patient.cancerconsultants.com

http://www.ias.ac.in/currsci/sep102001/555.pdf