Largest study of its kind finds Guardant 360 MSI highly concordant to tissue.
Large scale study shows Guardant360 effectively guides precision oncology treatment in metastatic breast cancer patients.
Liquid biopsy utility reinforced in early results from UK target program.
COTA and Guardant Health collaborate using real-=world data to reveal insights into metastatic colon cancer and prevalence of biomarker testing.
Colorado Center for Personalized Medicine Biobank begins returning participant PGx results.
Study shows only 40 percent of patients with metastatic colon cancer receive guideline-recommended biomarker testing.
I am writing this article using headlines from respectable journals of medicine and gene therapy scientific material, as a rebuttal of sorts to the articles written by so many laypeople and politicians who know so little about this industry and who seem to only find fault with it.
As a retired pharmacist with over 50 plus years in the profession, and business, of pharmacy, I consider myself more knowledgeable on this subject matter, and I think it is fair to say that the men and women who run these companies are not liars.
Higher profit margins are a necessity, not a luxury, as a source of funding for the “innovations” that some say are funded by the government, through the National Institutes of Health as well as private institutions.
The National Institutes of Health, a government-sponsored institution, in recent years has shifted its focus to DNA and gene therapy studies and basic research using these two tools
Biomarkers is another term you may hear more often now. It is defined as a molecule that indicates a normal or abnormal process taking place in your body and may be a sign of an underlying condition or disease.
Genes, DNA and proteins can serve as biomarkers. Cancer biomarkers can include proteins, gene mutations (changes in genes), DNA, and hormones.
Scientists working in the NIH and pharmaceutical companies are working in an area that is always new and very costly in its discovery and processing of drugs
Scott Gottlieb, former FDA commissioner, board member of Pfizer, and someone who is very knowledgeable on the subject of gene therapy and research on gene therapy cures, writing in the Wall Street Journal opinion page, talks of a “Victoria Gray of Mississippi, who recently became the first U.S. patient with a genetic disorder to be treated using the CrispR gene-editing technique.
Doctors used a novel drug to overwrite the function of a faulty gene, that gave rise to her sickle-cell disease
He says that over the decade, we will have gene-therapy cures for deadly inherited disorders such as muscular dystrophy, I am sure that many of the readers of the journal will remember the Labor Day telethons of Jerry Lewis to cure this horrible disease
Gene editing and regenerative medicine will be used to restore human functions lost to disease, including returning some sight to the blind. Gottlieb writes that “New and high-risk platforms like gene therapies are often targeted first to treat rare and serious conditions; after they are proven to work safely, they will be used to treat more common maladies such as heart disease.
“The Food and Drug Administration approved four gene therapies in only the past three years, with 800 similar kinds of products in various stages of development. An assessment of the current pipeline and historical rates of success in clinical trials suggest that by 2025, the FDA will be approving 10 to 20 gene therapy drugs a year. Progress is especially strong in oncology.”.
Gottlieb states that the cost of enrolling a single patient in a clinical trial for a gene-altering drug often costs between $500,000 and $700,000 and can reach as high as $1 million. That is for one patient in a clinical trial.
Gottlieb points out the important but very difficult process of giving “second to market” drugs the same regulatory benefits that” first to market drugs” receive since it would be difficult and possibly not ethical to treat a patient with a drug that may work when there is already one available that does work.
Companies today seek to discover what are frequently called “innovative” and that costs lots of money, time, and patience but without the first, there is no second or third.
The National Institutes of Health do use monies to do basic research that is taxpayer-funded. But only to a degree. Much more is needed than what is available from the NIH or other private organizations to discover and bring to market a new drug using gene therapy.
It is no sin to make a profit and to attack an industry that is now developing new types of drugs that actually cure disease states of very serious diseases. Years ago, many companies introduced drugs that were really just “me-too drugs” but those days are gone.
Most genetic diseases are quite rare and affect one person in every several thousand or million. Genetic disorders may be hereditary or non-hereditary meaning that they may be passed down from the parents’ genes.
Examples of genetic disorders are and include cystic fibrosis, sickle cell anemia, spinal muscular atrophy, and tay-sachs disease.
The headlines I quoted in the beginning of this article are there only to have you understand how different today’s drug companies and the scientists who work there are.
How we will pay for such curative gene therapies is another discussion for another day but really has nothing to do with the Sacklers, high drug prices we are experiencing now and higher drug costs.
Bert Drachtman
Great Neck
