By using this site, you agree to the Privacy Policy and Terms of Use.
Accept
Health Works CollectiveHealth Works CollectiveHealth Works Collective
  • Health
    • Mental Health
    Health
    Healthcare organizations are operating on slimmer profit margins than ever. One report in August showed that they are even lower than the beginning of the…
    Show More
    Top News
    What Are the Benefits of CBD?
    November 27, 2021
    How to Measure Adult Diapers- The Ultimate Guide to Picking the Right Size
    March 8, 2022
    medicine cabinet
    The Effect Of Finished Dosage Form Manufacturing In New Drugs
    July 5, 2022
    Latest News
    Biohazard Cleanup: The Importance of Proper Disposal and Containment
    December 2, 2023
    Medicare Helps Seniors, and People with Disabilities & ESRD
    November 27, 2023
    The Link Between Allergies and Sinusitis & Strategies for Relief
    November 27, 2023
    What Health Complications Can a Sunburn Cause?
    November 22, 2023
  • Policy and Law
    • Global Healthcare
    • Medical Ethics
    Policy and Law
    Get the latest updates about Insurance policies and Laws in the Healthcare industry for different geographical locations.
    Show More
    Top News
    Being Your Own Patient Advocate
    December 18, 2011
    Health Links for Senior Citizens
    January 11, 2012
    Restrict Flexible Spending Accounts
    August 25, 2017
    Latest News
    Automation in Pharmacovigilance: A Double-Edged Sword
    November 15, 2023
    What Does Science Say About Modern Health Practices?
    November 12, 2023
    Harnessing the Power of Email Marketing in Healthcare
    October 26, 2023
    10 Proven Strategies to Level Up Your Health Business
    October 25, 2023
  • Medical Innovations
  • News
  • Wellness
  • Tech
Search
© 2023 HealthWorks Collective. All Rights Reserved.
Reading: Is Genetic Therapy the Future for Treating Leukemia and Other Blood Disorders?
Share
Sign In
Notification Show More
Font ResizerAa
Health Works CollectiveHealth Works Collective
Font ResizerAa
Search
Have an existing account? Sign In
Follow US
  • About
  • Contact
  • Privacy
© 2023 HealthWorks Collective. All Rights Reserved.
Health Works Collective > Technology > Medical Innovations > Is Genetic Therapy the Future for Treating Leukemia and Other Blood Disorders?
Medical InnovationsSpecialtiesTechnology

Is Genetic Therapy the Future for Treating Leukemia and Other Blood Disorders?

Susan Scutti
Last updated: 2013/12/12 at 9:00 AM
Susan Scutti
Share
7 Min Read
genetic therapy for leukemia
SHARE

genetic therapy for leukemiaBlood disorders, including cancers like leukemia, often begin in the bone marrow where blood cells are produced, and from there the blood’s ability to properly perform some of its functions, such as fighting off infections, is thwarted.

Contents
Precision MedicinePresentation of Studies

genetic therapy for leukemiaBlood disorders, including cancers like leukemia, often begin in the bone marrow where blood cells are produced, and from there the blood’s ability to properly perform some of its functions, such as fighting off infections, is thwarted. Now, at the American Society of Hematology (ASH) annual meeting this past weekend, scientists have begun to offer evidence that experimental genetic therapies — precision medicines — may be effective and safe for treating blood disorder patients who fail to respond to standard treatments.

“It’s exciting to see these encouraging initial results with engineered immune cells, particularly such a durable response among patients who have very aggressive disease that has relapsed after standard treatments,” Dr. Laurence Cooper, of The University of Texas MD Anderson Cancer Center in Houston, stated in a press release. “With the right technology and laboratory expertise, the process of cell engineering is feasible for many patients.”

Precision Medicine

Precision medicine, also referred to as personalized medicine, is all about creating treatments tailored to an individual patient. This can be accomplished by first establishing a patient’s genetic profile and medical history, then examining those against existing clinical data to find appropriate disease targets, and finally engineering a cell therapy that can properly attack the origin of disease. Complicated? Wildly so, but this complex process represents the many and combined advancements in human genome studies, chemistry, technology, data-mining techniques, medicine — in short, all of science until now.

In particular, genetic therapies use DNA itself as a pharmaceutical agent. DNA is made up of nucleic acids, which are large biological molecules that may be catalyzed or activated by proteins. Commonly, genetic therapy will attempt to replace mutated genes with therapeutic genes introduced via a vector, which is able to penetrate a target cell and insert the genetic material into it. Once inside, the newly introduced DNA becomes expressed by the cell machinery, which in turn produces therapeutic proteins that target the disease — in short, a patient’s own re-engineered cells become soldiers in the war. Newer drugs that more precisely target pathways and proteins known to trigger CLL development, and leave normal cells unharmed, are proving to be more effective in promoting cancer cell death while safer for the patient.

In terms of blood disorders, stem cells contained within the bone marrow develop into three types of blood cells: red blood cells, white blood cells, or platelets. In blood cancers, the development process is interrupted by uncontrolled growth of an abnormal type of blood cell. In particular, chronic lymphocytic leukemia (CLL) occurs when abnormal white blood cells called lymphocytes accumulate in the blood, bone marrow, and lymph nodes, or other organs, causing these organs to enlarge. Although many patients with newly diagnosed blood disorders respond well to standard treatment, more than half of patients do not benefit from such therapies or experience a relapse after completing treatmentSeveral new, targeted therapies show promise to expand treatment options for CLL patients, as presented at the ASH conference.

Presentation of Studies

In cell therapy trials conducted at the Children’s Hospital of Philadelphia and the University of Pennsylvania, researchers used the chimeric antigen receptor (CAR) approach to manipulate the T cells — a type of lymphocyte — of 22 children and five adults with relapsed, treatment-resistant acute lymphocytic leukemia. After treatment with their own cells that had been re-engineered to seek, attack, and kill leukemic cells, 24 patients (19 children, five adults) achieved a complete response. One patient has remained in remission for a year and showed detectable engineered cells at 18 months post-infusion; unfortunately, six patients (five children, one adult) have since relapsed.

Another study concentrated on an experimental small molecule inhibitor designed to block the activity of a certain enzymes known as PI3K responsible for CLL cell signaling. Researchers enrolled 193 patients (average age 67), including 52 CLL patients with treatment–resistant or relapsed disease and 15 untreated CLL patients, in a Phase I trial testing dosage strengths from 8mg to 75mg. Researchers administered the new compound twice a day in 28–day cycles and observed clinical activity in relapsed or treatment–resistant patients at all doses studied. The experimental drug demonstrated activity in approximately half of the patients with a mutated form of a tumor suppressor gene (p53) that makes their cancer particularly hard to treat.

In one other trial conducted by the National Cancer Institute, 15 adult patients were treated with CAR-expressing T cells that targeted CD19, a protein on the surface of B cells (one type of white blood cell). All of the patients had advanced B cell malignancies, and eight had large B cell lymphomas. Working with a Los Angeles biotech firm, Kite Pharma Inc., the investigators infused all 15 patients with their own genetically-modified T cells following a chemotherapy conditioning regimen of cyclophosphamide and fludarabine. “They’ve had every treatment known to man,” study author Dr. James Kochenderfer of the National Cancer Institute, told CBS News.  Nevertheless, the results of the trial were extremely encouraging; six patients achieved complete remission and six achieved partial remission.

“Our data provide the first true glimpse of the potential of this approach in patients with aggressive lymphomas that, until this point, were virtually untreatable,” Kochenderfer stated in a press release. “This approach is still an early-stage experimental therapy, and we will continue our research to further improve the protocol and evaluate its value in additional patients with treatment-resistant disease.” Abstracts of these and many other studies appear on the ASH website.

(shutterstock)

TAGGED: blood disorders, leukemia

Sign Up For Daily Newsletter

Be keep up! Get the latest breaking news delivered straight to your inbox.
By signing up, you agree to our Terms of Use and acknowledge the data practices in our Privacy Policy. You may unsubscribe at any time.
Susan Scutti December 12, 2013 December 12, 2013
Share This Article
Facebook Twitter Copy Link Print
Share
Previous Article obamacare an covered california Obamacare and Reimbursement Rates to Providers
Next Article collaborative conflict in healthcare Collaborative Conflict

Stay Connected

1.5k Followers Like
4.5k Followers Follow
2.8k Followers Pin
136k Subscribers Subscribe

Latest News

weight loss
The Key Steps For Affective Weight Loss And Improving Health
Weight Loss Wellness December 6, 2023
Empowering Wellness: HBOT for Home Use Demystified
Empowering Wellness: HBOT for Home Use Demystified
Wellness December 5, 2023
Invasive Dentistry
Precision And Comfort: The Role Of Lasers In Minimally Invasive Dentistry
Dental health Specialties December 4, 2023
sublingual immunotherapy
Sublingual Immunotherapy: A Safe and Effective Alternative to Allergy Shots
Allergy Specialties December 2, 2023

You Might also Like

Invasive Dentistry
Dental healthSpecialties

Precision And Comfort: The Role Of Lasers In Minimally Invasive Dentistry

December 4, 2023
sublingual immunotherapy
AllergySpecialties

Sublingual Immunotherapy: A Safe and Effective Alternative to Allergy Shots

December 2, 2023
Medical Device Concept Development Paving the Way for Healthcare Innovations
Medical Devices

Medical Device Concept Development: Paving the Way for Healthcare Innovations

December 2, 2023
healthcare business website
Technology

Choosing the Right Domain Name for Your Healthcare Business

November 27, 2023
Subscribe

Subscribe to our newsletter to get our newest articles instantly!

Follow US
© 2008-2023 HealthWorks Collective. All Rights Reserved.
  • About
  • Contact
  • Privacy
Welcome Back!

Sign in to your account

Lost your password?