Each year in our Annual Report we highlight some of the patients and scientists who represent diseases that are the focus of CIRM awards. This year one of our stories focuses on sickle cell disease. You can read that story here.
Nancy Rene spoke to our governing board about her grandson Joseph, whose sickle cell disease was diagnosed through California’s newborn screening program. Joseph had a stroke at nine months old and receives regular blood transfusions to prevent additional strokes and the severe pain that accompanies the disease.
Despite the leg brace he still wears due to the stroke damage, Rene told our governing board, “Being a little boy trumps having a stroke.” As she said this she showed a picture of Joseph, smile on his face, jumping on a bed.
But not all of her stories were so positive. She works with families of kids with sickle cell and spoke of discrimination in the schools and medical system. The genetic disease is most common in people of African descent.
CIRM board member Bertram Lubin is president of Children’s Hospital and Research Center of Oakland and has a 40 year interest in treating sickle cell disease. In his introduction he said CIRM’s $9 million disease team award to find a therapy for sickle cell disease could significantly help children and families with the disease:
"This demonstrates CIRM’s commitment to minority health, health disparities and social justice issues and I think when you look at the people in the state of California who voted for proposition 71 there is a substantial number of minorities who are committed to improving the health of people within their communities."Lubin went on to say that each year 150 children in California are diagnosed with sickle cell disease through the newborn screening program that identified Joseph’s disease. Altogether, he said there are 9,000 people in California with the disease. For each person, the yearly medical costs are between $10,000 to $15,000. Lubin said:
"This is going to create a major burden. Any approach that has the potential to change that not only has major economic benefit but has phenomenal quality of life benefits for the children and for the family."You can read about the CIRM disease team approach led by Donald Kohn, who is part of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, and about Joseph in our annual report story online.
This is the fourth annual report story we’ve posted. Here are the others:
- Research Progress: Locking out HIV
- Spotlights on Disease: Autism
- Research Progress: Mimicking the pancreas