Gene Therapy: Is This The Future of Medicine & Curing Diseases?

An Introduction To Gene Therapy

Model of a DNA strand being manipulated with pincers

Gene Therapy was first introduced in 1972 with a paper titled ‘Gene Therapy For Human Genetic Disease?’ published in Science by US composed by Theodore Friedmann and Richard Roblin. This outlined the potential for incorporating DNA sequences into a patient in hopes of curing genetic disorders. This introduction followed 18 years of research until on September 14, 1990, the first ever approved gene therapy produced was performed on a four-year-old girl named Ashanthi de Silva. She was born with severe combined immunodeficiency and the procedure was done by W. French Anderson and his colleagues at NIH. This was the first successful procedure that pathed the way for gene therapy as a legitimate technique used to treat and prevent diseases.

Gene therapy is an experimental technique that is designed to introduce genetic material to cells to compensate for abnormal genes or to create missing beneficial proteins. Though a gene that is injected directly into a cell does not function, a carrier called a vector was engineered to deliver the gene, either through injection or IV, by infecting the cell. This technique can allow doctors to treat different diseases and disorders by inserting a gene into the patient’s cells instead of using drugs or resorting to surgery. This can be done several ways such as replacing a mutated gene with a healthy version, inactivating a mutated gene, making the mutated gene more evident so their immune system can identify and attack it or introducing a new gene entirely to help combat disease.

Gene therapy has not had a smooth path in the scientific community. Many scholars doubted the legitimacy and potential of this technique and the doubts grew rapidly with the first death after a gene therapy trial in 1999. This incident eventually led to the closure of the University of Pennsylvania’s gene therapy program which was one of the largest in the world and launched an additional 69 individual investigations of other gene therapy trials. It took almost four years to even begin it’s rebound to the path of being a reputable treatment option for diseases and disorders with no other viable cures.

Diseases/Disorders That Could Be Treated by Gene Therapy

● Cancer
● Cystic Fibrosis
● Adenosine Deaminase Deficiency
● Heart Disease
● Diabetes
● Hemophilia
● AIDS
● Immunodeficiency Syndrome
● Leber Congenital Amaurosis
● Blood Disorders
● Neurological Disorders
● Musculoskeletal Disorders
● Retinal Disorders
● Oncological Disorders
● Inherited blindness

Model of a DNA strand on black background

A New Hope For Gene Therapy

Initially, gene therapy was reintroduced in China in 2003 and then in Russia in 2011. These two trials set the path for an incredible boom in gene therapy that the United States quickly followed. After the first initial wave of gene therapy approval between the years 2003 and 2012 in addition to the significant advancements made in the vector technologies, the pace of gene therapy trial approvals rapidly picked up. In fact, the FDA predicts that the approval rate will grow to 10-20 annually by 2025.

2017 was set as a groundbreaking year for gene therapy due to multiple successful trials. These trials include a boy from France who was cured of Sickle Cell Disease after receiving treatment from Bluebird Bio. In the same year, two treatments were approved by the FDA to fight rare types of cancer. In Germany, a boy with a bacterial infection and a connective tissue disorder was grafted new skin with gene therapy, scientists accomplished this by extracting cells from a part of the boy’s body that was not blistered yet. In December of 2017, the FDA approved the first gene therapy treatment called Luxturna that corrects the mutation that is responsible for a range of retinal diseases. In that same month, BioMarin, a company that is working with gene therapy, published early clinical trials showing that nine patients saw substantial blood-clotting proteins that are missing with hemophilia.

Even with the amazing strides accomplished in recent years, researchers still must overcome challenges such as finding better ways to deliver genes, avoiding disrupting other gene’s functions, and targeting specific cells. On a more positive note, gene therapy on average results in an increase of life expectancy by anywhere from 5 to 15 years. Gene therapies also gives individuals the option to opt out of invasive surgeries or daily disease management life plans and it typically takes around only 10 months to complete a trial. Incredibly, as of 2021, there are currently 21 FDA approved gene therapies:

1. ABECMA (idecabtagene vicleucel)
Celgene Corporation, a Bristol-Myers Squibb Company

2. ALLOCORD (HPC, Cord Blood)
SSM Cardinal Glennon Children’s Medical Center

3. BREYANZI
Juno Therapeutics, Inc., a Bristol-Myers Squibb Company

4. CLEVECORD (HPC Cord Blood)
Cleveland Cord Blood Center

5. Ducord, HPC Cord Blood
Duke University School of Medicine

6. GINTUIT (Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen)
Organogenesis Incorporated

7. HEMACORD (HPC, cord blood)
New York Blood Center

8. HPC, Cord Blood
Clinimmune Labs, University of Colorado Cord Blood Bank

9. HPC, Cord Blood – MD Anderson Cord Blood Bank
MD Anderson Cord Blood Bank

10. HPC, Cord Blood – LifeSouth
LifeSouth Community Blood Centers, Inc.

11. HPC, Cord Blood – Bloodworks
Bloodworks

12. IMLYGIC (talimogene laherparepvec)
BioVex, Inc., a subsidiary of Amgen Inc.

13. KYMRIAH (tisagenlecleucel)
Novartis Pharmaceuticals Corporation

14. LAVIV (Azficel-T)
Fibrocell Technologies

15. LUXTURNA
Spark Therapeutics, Inc.

16. MACI (Autologous Cultured Chondrocytes on a Porcine Collagen Membrane)
Vericel Corp.

17. PROVENGE (sipuleucel-T)
Dendreon Corp.

18. STRATAGRAFT
Stratatech Corporation

19. TECARTUS (brexucabtagene autoleucel)
Kite Pharma, Inc.

20. YESCARTA (axicabtagene ciloleucel)
Kite Pharma, Incorporated

21. ZOLGENSMA (onasemnogene abeparvovec-xioi)
AveXis, Inc.

While gene therapy is not yet considered a primary treatment option and still must overcome technical challenges, it is continually gaining momentum within the scientific community. The FDA itself is committed to helping the development of these innovative treatments. In addition, there are research and technical advancements happening regularly. As technology grows so do the possibilities to discover the best vector delivery systems, expand the ability to correct rare genetic disorders and cure currently incurable diseases. Gene therapy holds a promise to transform medicine and become a real medical reality offering a new hope to people worldwide.

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Research For Life - Understanding Whole Body Donor Consent

Hello, my name is Garland Shreves, CEO of Research For Life. I want to take a moment to discuss some very basic information with you regarding consent forms, in general, that you may encounter when considering to donate to a whole body donor organization.

First and foremost, you need to understand and read the consent form, also known as the authorization form or document of gift, so you know what you are consenting to.  Ask questions of the organization if you don’t understand something. 

 All states require, under the Uniform Anatomical Gift Act, that consent be granted by an authorized agent of the donor or self-authorization before death.

Each state defines who in the consenting class has the most authority to direct donation. Such as the medical power of attorney, spouse, children, grandchildren, siblings, etc. and differs slightly in each state. 

Consent may be given by means of a verbal recorded consent or by a written document of gift.

Research For Life uses a written consent form which can be found on our website.

Understand that the donor or authorizing agent is giving the body to an organization. Once given it belongs to the organization to use in accordance with the consent form.

In other words, the donor organization is free to use the donor provided it does so within the terms

of the consent, it may not use the donor in a manner not consented too.  

The consent may state how the body may be used.  Educational and/or research purposes or some other purpose may be stated or in the discretion of the donor organization.

Research For Life provides cadavers and/or anatomical specimens for education and research purposes and does not do ballistic testing.

The consent may state that the body will be used in whole or in parts. It may also state that the anatomical parts may be used domestically and or internationally.

And most consents will cover some basic things like consent to test the donor for diseases and order medical records to help best determine the medical suitability for the donation.

The consent may also touch on issues like for profit or nonprofit status and if the donor or anatomical specimens will be used by one or more or both types of entities.  Remember that regardless of an organizations tax status they all charge fees to end users who order anatomical specimens and offer those specimens to both for profit and nonprofit entities.

From the very start of the donation process costs to the donor organization begin. 24-hour answering service, transport team to respond 24/7, qualified trained staff paid a livable wage with benefits and retirement, electric, gas, phone, insurances, building payments, maintenance, medical director, and regulatory requirements, and cremation fees. And these are just some of the expenses that an organization may have to cover.

Another item you may see on most authorization forms is a release of liability, a hold harmless agreement, excluding misconduct of course.  

Research For Life states clearly it will not and donor or agent agrees that Research For Life will not be held responsible for acts of third parties in connection with the donation.

Another item that reduces a donor organizations liability is the Anatomical Gift Act prohibits criminal, civil or administrative actions provided there is no intentional misconduct on the part of the donor organization. In other words, if the donor organization acted in good faith it is immune and provided some protection from lawsuits.

Another important part on a consent form is the person signing the authorization attests (affirms) that they have the authority to direct the donation. The donor organization accepts the authorizing agent’s authority in good faith barring any information known to it at the time of donation that would contradict the authority of the person authorizing donation.    

Remember, should you decide to register, tell your family and friends about your decision.

Also, the donation authorization form is not valid until notarized or signed by two witnesses; one witness must be non-family or disinterested party. 

Consent forms contain other important information that you need to read and understand.

All documents of gift or authorizations can be cancelled prior to death.

I want to thank you for taking the time to watch this video and I hope it helped provide you with some basic information regarding whole body donation consent forms.  Thank you.