Precision Medicine and Challenging diseases such as Cancer

Precesion Medicine & Personalized Medicine 

 

 

                      

                        'One size does not fit all'

 

Demands to treat the individual rather than the average human and the advances in artificial intelligence (AI) is ushering in precision medicine or personalized medicine where tailoring predictive, preventive and treatment strategies to the individual is a priority.  Since protein expression is dynamic and changes in relation to disease onset, severity or response to therapy are difficult to understand, proteomics stands to play a pivotal role in characterizing a disease at protein level. 

 

Proteomic technologies have progressed over the last decade allowing in principle the comprehensive analysis of expressed proteins in time and space.  Until now, quantitative proteomics has been pin-pointing minor differences in the protein levels between normal and pathological samples. There is now an urgent need for sophisticated ‘enabling technologies’ to identify structural differences in proteins introduced by mutations or structural variations induced by post-translational modifications or protein truncation that are associated with a disease.  Additionally, comprehensive characterization of the small molecule metabolites in biological systems and biological applications of the Metabolome together with the Proteome in precision medicine of the patient, stands to revolutionize global health.

 

The complexity of the data generated has also been a stumbling block in understanding diseases because proteome analysis does not provide a simple ‘yes/no’ answer but rather requires deep interpretation. To this end, utilization of data and information from various ‘multi-omics’ studies including metabolomics, together with AI in the hands of skilled researchers, can have significant societal impact.

 

 

 CEEPC's careful balance between excellence and focus on societal needs holds the key to its success. It is evident from CEEPC’s progress that it is ready for the next decade of excitement and expectations of multifaceted proteomics in Central and Eastern Europe. Additionally, in the era of emerging personalized medicine where treatment selection for each patient is becoming individualized, CEEPC and proteomics is expected to play a significant role moving forward for the benefit of mankind.

 

Scientists recently mapped the human epigenome for the first time.  The epigenome is made up of chemicals and proteins that can attach to DNA and modify its function by turning our genes on and off.  An individual's lifestyle and environment factors like whether they smoke or what their diet looks like, can prompt sometimes deadly changes in their epigenome that can cause cancer.  Mapping the epigenome may help scientists understand how tumors develop and cancer spreads.

 

Current medical and research challenges include:

Cancer, Neurological, Metabolic, Cardio-vascular, etc..... 

 

Excitement:

 

CAR T-cell

 

                                              

   

 

Efforts are on-going with genetic engineering to help the body’s immune system target cancer cells using CAR (chimeric antigen receptor) T-cell therapy.  CAR-T cells are T-cells, immune cells of the body that are reprogrammed to identify specific surface signatures that define specific types of cancer cells.  It allows these immune agents to seek and destroy cancer cells with great accuracy and with fewer side effects than the traditional chemotherapy or radiation.  

* Tisagenlecleucel, marketed as Kymriah, is a treatment for B-cell acute lymphoblastic leukemia (ALL) and Diffuse large B-cell lymphoma (DLBCL), which uses the body's own T cells to fight cancer (adoptive cell transfer).  

* Kymriah is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine which works by delivering genes into the body.  Kymriah contains the active substance tisagenlecleucel (consisting of genetically modified white blood cells). 

Currently, it is being tried out for treating two types of blood cancer:

* B-cell acute lymphoblastic leukaemia (ALL), in children and young adults up to 25 years of age whose cancer did not respond to previous treatment, has come back two or more times, or has come back after a transplant of stem cells;

* Diffuse large B-cell lymphoma (DLBCL) in adults whose cancer has come back or did not respond after two or more previous treatments.

While in their infancy, these techniques show great promise for future therapies and are currently being tried out as shown above.

 

 

Gene Therapy

 

 

                                      

     

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow treatment of a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Several approaches are being tried out in gene therapy, including:

 a) Replacing a mutated gene that causes disease with a healthy copy of the gene.

 b) Inactivating, or “knocking out,” a mutated gene that is functioning improperly.

 c) Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to ensure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures.

 

Reality and Reasons 

 

 

                                         

 

'On-going' studies show the complexity of cancer and the need to understand the microenvironment and the inter-relationships of various triggers and signals !

 

 

                                                 

                                                 Cancer cell-cell  / cell - matrix interactions that remain elusive !

 

Protein as Drug treatment

In cancer cells, one protein that stands out on their surface is the B- cell maturation antigen and it is possible that the future of treating the most advanced blood cancer multiple myeloma may lie with this single protein on tumor cells.  This protein is now an appealing target to wipe out the cancer without affecting healthy tissue and three different initiatives include:

1)      Activating the body's own immune cells to identify and kill diseased cells;

2)     Using another protein that binds with the antigen to deliver a killer dose of chemotherapy;

3)     Using a dual-acting protein that will pull cancer-fighting immune cells into cancerous cells.

Protein as a drug, chemical molecules as drugs or combinations of with the above mentioned therapies may lead to a possible solution in the near future.  The chances of a success remain much lower than that of a failure !

 

 

.......what is the reality and why are we failing in finding an ideal solution to cancers !

CEEPC is focused on improving prevention, early detection, diagnosis, and treatment of cancer by enhancing the understanding of the molecular mechanisms of cancer.  Advancing proteome science and the understanding of proteogenomic inter-relationship together with metabolomics, may help to further the cause for all above scenarios mentioned.