New Lifesaving Therapies

Our sustained research investment is helping save and improve the lives of patients. This year the U.S. Food and Drug Administration (FDA) approved 18 treatment options for patients who urgently need them. What’s more, LLS played a role in virtually all of these advancements.

A Breakthrough in Cancer: CAR T-Cell Immunotherapy

This year we witnessed a historic victory for cancer patients when a revolutionary, innovative immunotherapy was approved, ushering in a new era in the treatment of cancer and other serious and life-threatening diseases.

The FDA approved the first CAR T-cell immunotherapy, a treatment in which a patient’s T-cells, the soldiers of the immune system, are genetically reprogrammed to find and kill cancer cells. LLS recognized the early promise of this approach. Over the past two decades, LLS provided $40 million in funding for more than 15 researchers and companies around the world to develop this revolutionary approach to treating cancer.

LLS’s long term investment paid off when two CAR T-cell immunotherapies were approved by the FDA this year:

  • Tisagenlecleucel (Kymriah™) was the first CAR T-cell immunotherapy approved for children and young adults up to 25 years of age who relapsed or did not respond to therapy for acute lymphoblastic leukemia (ALL).
  • Axicabtagene ciloleucel (Yescarta™) was approved for adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment.

This is an extremely promising development for patients who have otherwise run out of treatment options. It has also dramatically stimulated research and clinical development exploring the use of CAR-T for solid tumors.


My work with LLS began when I was an oncology nurse, and our inpatient unit put together a team for Light The Night (LTN). It was a great way to support my patients and get involved in our new community after moving.

Then, in 2011, the unthinkable happened. My two-year-old son, Austin, was diagnosed with leukemia. It hit us like a ton of bricks. I had been taking care of others with cancer, but never imagined that I’d have to do the same for my son. My mission became to educate others about blood cancers and to raise funds.

Because of breakthrough research funded by LLS, my son is here today. After we ran out of treatment options when he was five years old, we enrolled him in a clinical trial for CAR T-cell immunotherapy. A therapy LLS has funded for two decades. Today, he is cancer free.

Our family volunteers with LLS in many ways – we started Team Austin’s Courage for LTN and I participated in Man & Woman of the Year (MWOY). During my 2014 MWOY campaign, I was able to raise the most funds in 10 weeks while we were traveling back and forth to Philadelphia here Austin was being treated. Winning Woman of the Year was one of my greatest moments, and I’m proud that I could do this in honor of Austin.

Progress in the Fight to Beat AML (Acute Myeloid Leukemia)

After more than 40 years of little change in the standard of care for AML patients, an unprecedented four new treatments were approved this year. By funding the most innovative research, LLS played a role in the advancement of each of these therapies:

  • Midostaurin (Rydapt®) was the first targeted therapy approved to treat adults with AML. It is intended to be used along with chemotherapy to treat newly diagnosed adults who have a mutation in a gene called FLT3.
  • Enasidenib (Idhifa®) was the second targeted therapy approved. It is for adults with AML who have a mutation in a gene called IDH2.
  • CPX-351 (Vyxeos™) is an innovative combination of two standard chemotherapies to treat patients with two high-risk types of AML.
  • Gemtuzumab ozogamicin (Mylotarg™) is an antibody drug conjugate that was approved for adults and children whose cancer cells test positive for the antigen CD33.

After so many years of limited options we are finally building a treatment armamentarium to give AML patients more choices and a chance for better outcomes,” said Louis J. DeGennaro, LLS President and CEO.

LLS supported the research that formed the foundation for development of each of these new therapies. We continue to fund new ways to use the arsenal of AML therapies, either alone or in combination, as well as help identify patients who will benefit the most from these therapies.