Blood Cancer Research & Developments
- Evergreen Chapter
- Mar 19
- 3 min read
Author: Arshdeep Kaur
Gene Therapy & CRISPR Applications:
To treat or prevent diseases, scientists have been using gene therapy as a way to alter genetic instructions within a cell. In blood cancers, gene therapy can be used to repair or replace mutations that cause cancer, enhance the immune system’s response skills to better recognize cancerous cells, help create new proteins in the body by introducing new genes, and can make cancer cells weaker when exposed to other treatment options.
A specific type of gene therapy is CRISPR, which is a gene editing tool that specifically alters faulty genes in DNA without triggering cancer development (insertional oncogenesis) and triggering unwanted immune responses from substances like drugs or therapeutic proteins (immunologic toxicity). CRISPR is a type of technology that repairs, introduces, or inactivates a gene. The goal for this method is that cancer cells will eventually die out by interrupting some process required for cancer survival. This is done by preventing blood flow to tumors, led by cancer cells and surrounding tissues, and blocking the intake of nutrients that are used to fuel the cancer’s growth. However, with this technology, several obstacles have risen, including “gene silencing”, which is when a newly inserted gene fails to turn on.
Personalized Medicine in Blood Cancer Treatment:
Personalized medicine is a treatment option that is specifically tailored to meet the needs of a patient. Personalized medicine mainly affects cancer cells and usually leaves healthy, normal cells alone. The targeted drugs used in personalized medicine work by blocking cancer cells from multiplying. These therapies are programmed to find and attack specific areas in cancer cells (dangerous proteins and enzymes), and can detect and block out messages that tell a cancer cell to grow.
Clinical Trials:
In order to test new medical treatments, scientists conduct research studies to evaluate their findings and explore more options. The purpose of blood cancer clinical trials is to improve and increase survival rates, life quality, and treatment options. Before a trial can be conducted, the new treatment often undergoes much testing in laboratories and on animals. If the early findings show promise that the treatment might be safe and effective, a monitored clinical trial will be conducted on various groups of people. Members of the medical team and doctors closely monitor the ‘test subjects’. There are many types of treatment offered through blood cancer clinical trials, such as, drug therapy, radiation therapy, immunotherapy, stem cell transplantation, supportive therapy, and more.
A cancer clinical trial is split up into four phases, each focusing on a specific aspect of the treatment. As trials are completed successfully and safely, it will move up to the next phase. During Phase 1, the treatment is tested on a small group of people to determine the appropriate dose, safety, and best way to manage the treatment. After that, Phase 2 takes place which is when the treatment is tested on a larger group of people to discover the effectiveness of the treatment. In Phase 3, the results of a “treatment group” are compared to a “control group”, making the trials randomized. Patients in the treatment group will receive the new study treatment. Patients in the control group receive the best, standard treatment. If the new treatment passes the third phase, the U.S. The Food and Drug Administration (FDA) will approve the treatment if it meets safety guidelines and is proven to be better than the pre-existing treatments or equally as effective as pre-existing treatments but with less harmful side effects. During Phase 4 (the last phase), final studies are conducted to gather extra information on efficiency.
Works Cited:
Dougherty, Beth. “How Is Gene Therapy Being Used to Treat Cancer and Other Diseases?” Dana-Farber Cancer Institute, 2 Dec. 2024,
Uddin, Fathema, et al. “CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future.” Frontiers in Oncology, vol. 10, Aug. 2020,
Gene Therapy Treatment | Dana-Farber Cancer Institute. 23 May 2019,
“Targeted Cancer Therapy | Targeted Drug Therapy for Cancer.” American Cancer Society,
Clinical Trials | How a Clinical Trial Works | LLS
Clinical Trials | What These Research Studies Are | LLS.
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