The Last Generation – a mother’s hope for her daughter

Adrienne Shapiro

Earlier today Sangamo Biosciences announced it had reached a collaborative agreement with Biogen Idec, something that could help speed up development of a cure for sickle cell disease. That can’t come soon enough for Adrienne Shapiro, a Patient Advocate and mother of a daughter with sickle cell disease. In this guest blog Adrienne talks about what finding a cure would mean for her, for her daughter, and for everyone else battling this debilitating and deadly disorder. 

Finding this cure would mean that I am the last generation of mothers in my family to spend her adult life keeping her child alive. Watching my bright, beautiful, brave girl as this horrible painful disease ravages her body and the pain medicines impair her mind. Fighting for her while the weight of the psychological and social issues surrounding the treatment of Sickle Cell wears away at her spirit.

My grandmother told me the story of her eldest and favorite brother. He was smart, strong and bold. As a child he would sometimes complain of pain and would just get weak. Other times he was just a normal happy boy. At fifteen he went off to the College for Negro Boys to study education. He wanted to be a teacher. His health became a problem off and on, but he managed to do well with his studies. During his second year his friends found him dead in his bed. No one knew that he had Sickle Cell disease. My Great-Grandmother grieved over her first born son and said it was not natural for a mother to bury her child.

Moms (an aunt who raised me) had a son named Teddy. He was diagnosed with Sickle Cell shortly after birth. Everyone said it was something that skipped a generation. Teddy received a “bad blood transfusion” when he was three years old. It left him mentally and physically handicapped. Everyone told her to put him in a home and she said “He has a home. With us”. Teddy was the oldest child and as the other children were born each was tested for the disease. None of us had the disease and all were told we did not have the trait. Teddy lived to be nearly fifty years old. He always lived at home with Moms and Pops and had very few painful Sickle Cell crises. Moms was convinced that it was because she watched over him so carefully.

Last Generation
When my bright, beautiful girl was born she looked perfect. I did not worry, I had been tested and told that I did not have the trait and you needed two parents with the trait. Her father was Cuban and we didn’t even think Cubans could have Sickle Cell. She was diagnosed at nine months during newborn screening. We all said it could not be possible. We found out that the test we had been given was faulty and I did indeed have the trait. I said my daughter would live with me. She would have the best health care and education because she would be cured in her lifetime.

Stem cell research can make that happen. I will not have to repeat the experience of burying my first born as the generations before me. She can be cured and I can be the last generation of mothers in my family to live with the fear of losing her child to Sickle Cell disease.

Adrienne Shapiro

At the stem cell agency we are funding several research projects looking into developing treatments and even a cure for sickle cell disease.

Big deal raises big hopes for treating sickle cell disease and Beta-thalassemia

Sickle cell blood cells – targets for a new deal

2014 is getting off to a very good start for CIRM-funded companies. Today Sangamo BioSciences announced that it has reached agreement on a global collaboration with Biogen Idec, a move that could speed up work to develop cures for both sickle cell disease and Beta-thalassemia.

This follows hard-on-the-heels of a deal announced earlier this week between Capricor and Janssen Biotech, a division of Johnson & Johnson, for a stem cell treatment for heart attacks. That approach recently got approval to move into a Phase 2 clinical trial, which we are funding.

In this latest collaboration Biogen Idec will give Sangamo $20 million upfront with the possibility of an additional $300 million if the company’s research hits certain milestones in development, sales and royalties.

Last May Sangamo got a $6.3 million award from CIRM to develop a method of correcting faulty genes that lead to sickle cell disease and beta-thalassemia This approach involves removing bone marrow from people with these conditions, fixing the genetic defect in the blood-forming stem cells., then reintroducing the modified stem cells into the patient where they are expected to produce new healthy red blood cells.

Ellen Feigal, M.D., Senior VP for Research and Development at the stem cell agency said:

“This is great news. It’s great news for the companies involved, for the stem cell field in general and of course for patients whose lives could benefit from this collaboration. It’s also important because it shows how important the funding we provide is in helping companies like Sangamo get their research to a point where big pharmaceutical companies stand up and take notice, and invest.” 

In a news release announcing the collaboration Sangamo’s President and CEO, Edward Lanphier, said:

“This alliance is further validation of our ZFP platform as a transformative technology and accelerates our goal of developing a novel class of therapeutics which has the potential to revolutionize the treatment of genetic diseases.”

He was even nicer in an email to us saying:

“Thank you for ALL of your support over the past several years. We wouldn’t be where we are today without you.” 

The two big deals this week are likely to be hot topics for conversation at next week’s Alliance for Regenerative Medicine’s State of the Industry briefing in San Francisco. That’s a gathering for many of the key players in the regenerative medicine field that focuses on the recent advances in the field and the outlook for the coming year.

And so far, the coming year looks like being a pretty good one for the field.

kevin mccormack