Brian Shelton was diagnosed with Type 1 diabetes.
He would lose consciousness when his blood sugar dropped. He crashed his motorcycle. He passed out while delivering mail. His supervisor told him to retire after 25 years in the Postal Service. He was older.
Cindy Shelton took him to her home. She said she was afraid to leave him alone.
She spotted a call for people with Type 1 diabetes to participate in a clinical trial. The company was testing a treatment developed over decades by a scientist who promised to find a cure after his son and daughter were diagnosed with the disease.
Mr. Shelton was the first patient. He got an injection of stem cells on June 29 that were similar to the cells he had lacked.
His body now controls his blood sugar levels.
Mr. Shelton may be the first person to be cured of the disease with a new treatment that has experts daring to hope that help may be coming for many of the 1.5 million Americans suffering from Type 1 diabetes.
Mr. Shelton said it was a whole new life. It is like a miracle.
Diabetes experts urged caution, but they were astonished. 17 people with severe cases of Type 1 diabetes are being studied for five years. It is not intended to be a treatment for Type 2 diabetes.
Dr. Irl Hirsch, a diabetes expert at the University of Washington, said that they have been looking for something like this for decades. He wants to see the result replicated in many more people. He wants to know if the cells will last for a lifetime or if the treatment will have to be repeated.
He said it was an amazing result.
While offering the same caveat, Dr. Peter Butler, a diabetes expert at U.C.L.A., agreed with the research findings.
It is a remarkable result, according to Dr. Butler. It is comparable to the miracle of the first 100 years of the 20th century when the first type ofinsulin was available.
The 30-year quest of a Harvard University Biologist, Doug Melton, started it all.
Dr. Melton had never thought about diabetes until 1991, when his baby began to vomit and shake.
The doctor didn't know what it was because he was so sick. He and his wife rushed their baby to the hospital. Sam had a lot of sugar in his urine.
The islet cells of the pancreas are destroyed when the body's immune system attacks it. If patients don't get injections of insulin, they will die quickly from Type 1 diabetes. Everyone gets better.
It is a terrible disease, said Dr. Butler at U.C.L.A.
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The New York Times reported that a Harvard University doctor didn't think much about diabetes until his son started showing symptoms.
Diabetes is the leading cause of blindness in this country. It's the leading cause of failure of the kidneys. People with Type 1 diabetes are at risk of having their legs amputations if their blood sugar goes down during sleep. They have a higher chance of having a heart attack or stroke. The immune system is weakened by Covid-19.
The high cost ofinsulin has risen each year, adding to the burden of the disease.
The only cure that has ever worked is a transplant of islet cells from an organ donor. A shortage of organs is an impossibility for most people with the disease.
Dr. Ali Naji, a transplant surgeon at the University of Pennsylvania who pioneered islet cell transplants and is now a principal investigator for the trial that treated Mr. Shelton, said that even if we were in utopia, we would never have enough pancreases.
The care of an infant with the disease was frightening for Dr. Melton and Ms. O'Keefe. Ms. O'Keefe had to check Sam's blood sugar four times a day. She had to inject him with the drug. The baby was not sold the proper amount of the drug. His parents had to make it smaller.
If I am doing this, you have to figure out this disease, said Gail. When Emma was 14, she would develop the disease as well.
Dr. Melton abandoned frog development work in order to find a cure for diabetes. He looked at the potential of embryonic stem cells to become any cell in the body. He wanted to turn them into islet cells to treat patients.
The cells came from unused fertilized eggs from a fertility clinic. In August 2001, President George W. Bush stopped using federal money for human embryo research. The stem cell lab at Harvard had to be severed from everything else. He got private funding from the Howard Hughes Medical Institute, Harvard and philanthropists to set up a completely separate lab with an accountant who kept all its expenses separate, down to the light bulbs.
The lab of 15 or so people took 20 years to convert stem cells into islet cells.
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Mr. Shelton has diabetes treatment supplies. He said that his new drugs, which suppress his immune system, are less burdensome than the constant blood sugar monitoring.
The challenge was figuring out what sequence of chemical messages would turn stem cells into islet cells. The work involved unraveling normal pancreatic development, figuring out how islets are made in the pancreas and conducting endless experiments to steer embryonic stem cells to becoming islets. It was slow.
A small team of researchers, including a graduate student, were in the lab one night in the summer of 2014, doing one more experiment after years of futility.
She said they weren't very optimistic. They put a dye in the liquid where the stem cells were growing. If the cells made the drug, the liquid would turn blue.
Her husband had called to ask when she was coming home. She saw a faint blue hue that became darker and darker. She and the others were happy. They had made functioning islet cells from stem cells.
The lab had a party and a cake. They made bright blue wool caps with five circles of red, yellow, green, blue and purple representing the stages stem cells had to pass through to become islet cells. They had been stuck at green until they got purple.
The next step for Dr. Melton was to start a company so he could make a drug that could be marketed.
His company was founded in a mix of names.
One challenge was to figure out a way to grow islet cells in large quantities. It took five years.
The company's cells worked well in mice and rats, and they cured diabetes in rodents.
A large, well financed and experienced company with hundreds of employees was needed for a clinical trial in patients. Thousands of pages of documents were prepared and clinical trials were planned, but everything had to be done to the standards of the Food and Drug Administration.
There was a chance that it would happen. At a meeting at Massachusetts General Hospital in April, Dr. Melton ran into a former colleague, Dr. David Altshuler, who was the deputy director of the Broad Institute. Dr. Altshuler asked Dr. Melton what was new.
The doctor took out a small glass container with a purple pellet at the bottom.
He told Dr. Altshuler that the islet cells were made at Semma.
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Bill Sikes/AP
Human diseases have their biology understood. He was told by Dr. Altshuler that there might be an opportunity.
Semma was acquired by Vertex eight weeks later for $950 million. Dr. Sanna became an executive vice president.
The company will not make a price public until it is approved. It is likely to be expensive. Patients have been enraged by the high prices for drugs that are difficult and expensive to make.
The challenge was to make sure the production process worked and that the cells would be safe if injected into patients. Stem cells were turning into islet cells when employees monitored vessels of solutions containing biochemical signals.
The F.D.A. allowed a clinical trial to begin with Mr. Shelton as its initial patient.
Mr. Shelton has to take drugs to suppress his immune system. He says they cause him no side effects, and he finds them less risky than taking his blood sugars and takinginsulin. His body will reject the infused cells if he keeps taking them.
John Buse, a diabetes expert at the University of North Carolina who has no connection to Vertex, said the immunosuppression makes him pause. We need to carefully evaluate the trade-off between the burdens of diabetes and the potential side effects of immunosuppressive medications.
Dr. James Markmann, Mr. Shelton's surgeon at Mass General, said that Mr. Shelton's treatment called for careful follow-up and required starting with half the dose that would be used later in the trial. He said that no one expected the cells to function so well.
Dr. Markmann said the result was a real leap forward for the field.
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Mr. Shelton shed tears when he checked his blood sugar levels after his procedure.
The results were to be revealed to Dr. Melton last month. He didn't expect much.
He said he was prepared to give them a pep talk.
Dr. Melton was jittery during what felt like a moment of truth. He had spent decades on this project. The data was for real at the end of the presentation, and he had a huge smile on his face.
He went to dinner with Sam, Emma and Ms. O'Keefe. They were told the results when they sat down to eat.
There were a lot of tears and hugs.
The moment of truth came when Mr. Shelton left the hospital. He measured his blood sugar. It was perfect. They had a meal. His blood sugar was in the normal range.
Mr. Shelton cried when he saw the measurement.
I can only say thank you.
