More on the trial aiming to cure sickle cell

Summary

A groundbreaking medical treatment for sickle cell anemia is discussed in this interview, where Dr. Jon LaPook highlights the potential for a cure through gene therapy. Janelle Stephenson, a patient who underwent the treatment, shares her emotional journey, including the risks and sacrifices she faced, such as the loss of fertility due to chemotherapy. The conversation also addresses the scientific challenges and fears surrounding gene therapy, particularly its use of the HIV virus. This medical advancement illustrates how basic research, like HIV studies, can lead to life-changing treatments for various diseases, offering hope for future cures.

Takeaways

• 😀 A significant medical breakthrough has been made in the treatment of sickle cell anemia, showcasing the potential of gene editing to cure the disease.
• 😀 Dr. Jon LaPook, in his report on 60 Minutes, discusses a possible cure for sickle cell anemia through gene therapy.
• 😀 The gene therapy involves manipulating the patient's genes, potentially offering a cure for sickle cell disease, but there are still risks involved.
• 😀 Despite the promising results, Dr. LaPook remains cautious, emphasizing that a cure should not be declared prematurely, though it looks promising.
• 😀 The emotional impact of the breakthrough is deeply personal for Dr. LaPook, who recalls witnessing many deaths from sickle cell anemia in the 1970s and 1980s.
• 😀 Janelle Stephenson, a patient featured in the story, was given a life-changing treatment to potentially cure her sickle cell anemia, although it involved significant risks.
• 😀 Janelle has had to make sacrifices, including the inability to have children, as a result of the chemotherapy required for the gene therapy.
• 😀 The fear surrounding gene therapy is linked to the irreversible nature of altering the human genome, despite future technologies like gene 'kill switches' being explored.
• 😀 The gene therapy relies on using a weakened HIV virus to deliver the treatment, demonstrating how research into other diseases (like HIV) can aid in breakthroughs for others.
• 😀 Dr. LaPook emphasizes the importance of funding basic research, which can lead to unexpected discoveries and ultimately improve treatments for various diseases in the future.

Q & A

How significant is the medical breakthrough reported by Dr. Jon LaPook for sickle cell anemia?

The medical breakthrough is significant because it serves as a proof-of-concept, demonstrating that gene manipulation could potentially cure sickle cell anemia.

Is Dr. LaPook willing to say that the treatment is a cure for sickle cell anemia?

Dr. LaPook is cautious and hesitant to say it's a definitive cure, but he believes that the treatment has the potential to cure sickle cell disease.

What personal connection does Dr. LaPook have to sickle cell anemia?

Dr. LaPook has a personal connection because, during his early medical career from 1976 to 1986, he witnessed many people with sickle cell anemia die without effective treatment, which left him feeling helpless.

What role did Janelle Stephenson play in the story reported by Dr. LaPook?

Janelle Stephenson was the patient featured in the story who underwent the treatment. She had lived with sickle cell anemia her whole life and underwent a gene therapy that ultimately cured her.

Did Dr. LaPook know the outcome of Janelle's treatment before reporting on it?

No, Dr. LaPook did not know if the treatment would be successful. At the time of reporting, Janelle had not yet received the treatment, and there was uncertainty about the outcome.

What were some of the risks and side effects Janelle faced as part of her treatment?

Janelle faced the risk of permanent infertility as a side effect of the chemotherapy, which was part of the treatment. She had to give up the possibility of having children in the future.

How did Janelle feel about giving up the possibility of having children?

Janelle came to terms with the idea of not having children, acknowledging that it was a sacrifice for her health. She felt that it would be too painful to bring a child into the world with sickle cell anemia.

What concerns did Janelle have about the gene therapy used in her treatment?

Janelle was initially terrified because the treatment involved altering her genes using a weakened form of the HIV virus, a technology she associated with past fears from the HIV epidemic.

Why is gene therapy considered a risky approach in medical treatment?

Gene therapy is considered risky because it involves altering the human genome, and once a gene is inserted into a patient, it cannot be undone. There are also concerns about the potential for severe adverse effects, such as the risk of death, which have occurred in previous gene therapy trials.

How is the knowledge gained from HIV research contributing to gene therapy for sickle cell anemia?

Research into the HIV virus has played a crucial role in gene therapy development. Understanding how the HIV virus works has helped scientists use it as a vehicle for delivering therapeutic genes, which could potentially cure diseases like sickle cell anemia.