I have been vaccinated for Covid. Now what?

Genes are Here 

“Which Gene is here? I only know one Jean,” he said. 

  

“No not that Jean and not the jeans that you wear. I mean the genes that make up your DNA.” They are here, I said. 

In the field of medicine, the 21st century is going to be gene therapy century. Genomics like other “omics”, is a field that deals with individual genes or the whole genome. Gene therapies are medical treatments that are based on abnormalities in the genes. These genetic defects can be inherited such as sickle cell disease or acquired such as cancer.  

Today, we are at the cusp of making many of such diseases, a thing of the past. 

Until the turn of the century, most of the medicines were made by chemists by mixing chemicals. Such a toxic brew of chemicals was first tested on live cells in a petri dish, then on little mice, followed by bigger animals. This is what is referred to as phase 1 trials. If the toxicity and benefits ratio was acceptable, the chemical was taken to phase 2 trials where it was tested on a small number of volunteer healthy humans. Phase 2 trials would then go on to large, multi-institutional, multi-national human phase 3 clinical trials before the drug would be approved by the FDA.  

Most of the new drugs are however not chemical agents. We now have biological therapies. These therapies also called biologics, are made from proteins, enzymes and other ingredients from living organisms. The advances in genomics has made it possible to create customized and personalized therapies. This is particularly the case where we can identify abnormalities or mutations in the genes.  

How many pharmaceutical companies are involved in developing Gene Therapies?  

I Googled and got 176,000,000 answers in 0.84 seconds.  

The top 4 answers of the Google were:  

• The 10 best gene therapy companies  
• Gene therapy on the rise 
• Global Gene Therapy Markets 

• Next Generation Therapeutics 

It is a fact that the era of medical diagnostics and therapeutics based on genomics is here and now. 

As of this week, the Food and Drug Administration (FDA) had 900 investigational drug applications for gene therapy products in the pipeline, waiting for approval. The FDA is scrambling to develop new policies to shape the modern structure for the development, testing and clinical applications of such therapies.  

There are many types of Biologic Therapies, Gene therapy being only one of them.  

I wonder if you have noticed that I am not referring to these as drugs but as treatments and therapies. Unlike the traditional drugs, these treatments involve a defined process which is custom made for individual genetic abnormality.   

Whereas 900 potential therapies are in the pipeline, FDA has approved 4 gene therapies since 2017. Pardon my pronunciation of these difficult generic names. I wonder if it is intentional to make them sound exotic and expensive. 

• tisagenlecteucel (Kymriah)  

• axicabtagene ciloleucel (Yescarta),  

• voretigene neparcovec-rzyl ( Luxturna) and  

• omanemsogene abeparvovec-xiol (Zolgensma).  

Should I dare mention the list price of these treatments: 

Kymriah  (CAR-T for cancer) $475,000    

Yescarta (CAR-T for cancer) $373,000 

Luxturna (inserting normal copy of RPE65 gene for inherited blindness) $ 425,000 

Zolgensma (inserting normal copy of SMA gene for spinal muscular dystrophy) $2.1 M 

Mind you that the list price mentioned above is for one dose or one treatment only. The costs to the individuals and the healthcare system and the ethical issues associated with their use is a topic of another discussion. 

While I am still trying to learn how to pronounce and memorize the names of the new treatments; the field of Gene Therapy is moving at warping speed.  

The Luxturna therapy, which is used for inherited blindness is an example. 

Inherited blindness is a rare disease caused by one defective gene in the retina.  

Is it possible to bring sight to these people by replacing the single defective gene with a normal one? The answer, most likely YES. 

This is how Luxturna therapy works:  We know that viruses have the ability to insert their own DNA into the nuclei of the human cells. Scientists have access to a host of harmless viruses. Some have been used for decades in medical research, A normal copy of the RPE65 gene is fused with the DNA of such carrier viruses called the vectors. These vectors are then directly injected into the eye. The carrier viruses, then move to the retina and insert their DNA with the copy of the healthy gene RPE65 into the retinal cells and possibly restoring the vision. Sounds like a miracle. 

But since it is a hit and trial process, it is possible that the carrier viruses may not deliver their payload accurately.  But that may change very soon. It seems that now we have a more efficient and perhaps a definitive method of inserting the gene at the right place in the retinal cells.  

As I sat down with a cup of coffee in my hand and opened the Wall Street Journal on Thursday (3/5/2020), a news item on page 2 caught my attention.  

“Gene Editing Used to treat blindness”.  

The report stated that a patient with inherited blindness was treated with at the Oregon Health and Science University in Portland. The researchers have succeeded in including the gene editing tool called CRISPR to these vectors (carrier viruses). The CRISPR tool is expected to cut out the abnormal gene and allow the vector to insert the normal gene at the precise location in the DNA.  

The report said that if it all works out, it will restore the vision of this person who has never seen the light of the day or the face of the mother.  

Lets wait and see.