Patient Worthy’s One-Year-Checkup on Victoria Gray, the First Sickle Cell Disease Patient to Receive CRISPR in the US

 

 CRISPR is a gene-editing technique that has revolutionized the medical world with its approach to making precise changes in DNA. CRISPR found its match in 35-year-old Victoria Gray of Forest, Mississippi, who volunteered to be part of the most highly anticipated experiment in years. NPR received exclusive rights to report Victoria’s experience which began on July 2nd, 2019.

The Key Word is “Successful”

Victoria’s disease was diagnosed when she was three months old. Every year of her life since then she has endured sudden and excruciating pain. Victoria remembers having at least seven episodes each year with most involving hospitalization.

Now she can report that CRISPR has relieved her stabbing pains to a great extent, enabling her to be active in her children’s lives and look forward to a better future.

In June of this year, NPR contacted Victoria for an interview just to “check-in.” It appears that Victoria continues to improve. The scientists are cautiously optimistic. They feel that the treatment is safe and so far it is working as anticipated.

Surpassing Anticipated Results

The scientists were hoping that post-treatment there would be at least 20% of fetal hemoglobin in her system. Surprisingly, the levels of fetal hemoglobin have remained at about 46% with 99.7% of the fetal hemoglobin remaining in her red blood cells.

 Victoria’s First Flight

Victoria even felt well enough to fly out to see her husband, who has been deployed with the National Guard in Washington, D.C., for months. She had been afraid of hyperventilating on the plane, as this was her first flight. It was something she had always dreamed about, but she had previously been unable to fly.

Victoria’s doctor, Haydar Frangoul, M.D. at Nashville’s Research Institute, leads the study. Dr. Frangoul cautions that it is too soon to come to definitive conclusions. Victoria and the other participants must be followed for longer periods of time to determine if the treatment is successful.

About the First Ten Patients

Victoria was the first of ten patients who were recipients of the CRISPR technique as part of a research study. Two other participants, in addition to Victoria, have sickle cell disease. Seven patients have beta thalassemia, which is a similar blood disorder.

The latest results of the research study were discussed recently at the American Society for Hematology annual meeting. The patients have been followed between three to eighteen months and seem to be responding well. However, the intense chemotherapy that was administered prior to the CRISPR treatment did cause side effects. The treatment involved the infusion of billions of edited cells.

About the NEJM Paper

Victoria and another patient became the focus of a paper in the New England Journal of Medicine which was recently published online. It is the only peer-reviewed paper on the study to date.

Victoria and the other sickle cell participants in the study report that they have not experienced complications from the treatment. They were especially pleased to report that they have not had painful attacks, nor were they required to spend time in the hospital as they had in previous years.

In addition, Victoria was able to taper off the pain medication she relied on for years.

One of the two scientists who discovered CRISPR, Dr. Jennifer Doudna, commented on the study results. Dr. Doudna said that she is very excited to learn that patients are seemingly on their way to being cured.

Additionally, there are two other companies sponsoring research to treat nine new patients. Reports about results for this new research are currently unavailable.

Looking Forward

If Victoria’s condition continues to improve, she hopes to go back to nursing school. Her most passionate desire though is to just see her children grow up. She wants to see her children get married and be there for their weddings. She is hopeful that they will no longer have to worry about their mother dying.

And she wants to be a grandmama.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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