Hemoglobin is a protein in red blood cells that carries oxygen throughout the body. It is made of four protein chains, two of which change after birth. Mutations on those two chains can lead to sickle cell disease and ß thalassemia, conditions that can lead to significant morbidity, poor quality of life and early death. The only curative treatment has been a bone marrow transplant from a genetically matched sibling, but fewer than 20 per cent of patients have suitable donors.
A new treatment being developed by CRISPR Therapeutics, where Krista McKerracher (BSc Health Studies ’84) serves as vice-president and head of the hemoglobinopathy program, allows people to serve as their own donors and may be a new curative option for these diseases.
In her role at CRISPR, based in Massachusetts, just off MIT’s campus, McKerracher functions like a conductor for an orchestra of clinicians, scientists and project managers. With input from the team, she makes decisions about their study design and liaises with regulators and partner companies.
She is also a member of the corporate executive team, which is using CRISPR Cas9, a gene-editing technology developed from the way bacteria protect themselves from a virus in order to edit blood stem cells. A patient’s blood stem cells are collected, edited and then given back via a stem cell transplant. The editing process turns fetal hemoglobin on again by disabling the gene that turns it off when we are born. The objective is to make enough fetal hemoglobin post-transplant to overcome the inability to make good adult hemoglobin. The project is in the early days of human testing but holds the promise of making more patients a candidate for a curative transplant.
“What keeps me up at night is an obligation to minimize risk for the patients who are volunteering for investigational therapy,” says McKerracher. “I never forget that behind everything we do are people’s mothers, fathers, daughters and sons who are going through this experimental transplant with the hope that this horrible disease would be cured.”
Giving hope when there are no other options
Several years ago, McKerracher found herself on the other side of the system when she was diagnosed with cancer. She says of the cancer, which was treated surgically, “Having worked on cancer drugs for so long, and then getting in the system as a patient, reconnected me to how frightening it is to be in need of health care.”
Throughout her career at a wide variety of innovation-based pharmaceutical companies, especially those engaged in research, McKerracher has fought the public perception of Big Pharma as evil, and says, “Everyone I’ve worked with has been really conscientious.” She has worked on products for serious diseases where patients don’t have options – like those living with sickle cell disease and thalassemia. “We are in the business of giving people hope where they don’t have options,” she says.
"I never forget that behind everything we do are people’s mothers, fathers, daughters and sons."
McKerracher may use her work to this end, but she is also in the business of giving hope outside of it, selectively choosing charities and organizations to support with her time and money. She serves on the board of directors of the Valerie Fund, which supports families with kids with cancer or serious blood disease. “It’s hard enough when you’re diagnosed in your 50s, but it’s devastating for a family with a five-year-old with a brain tumour. I want to give back in that way.”
Waterloo’s Applied Health Sciences is another place McKerracher gives back, with regular donations to the program’s undesignated funds. “UWaterloo is a special place to me. It gave me an excellent foundation and I maintain a lot of friendships from my Waterloo days.”
She adds, “If I’m giving someone money, I trust that they are the best custodians of that money. I like keeping things open to possibility – I know there are a lot of good things happening at UWaterloo and a lot of innovation, and I’m happy to enable that.”
