Breakthroughs in Cancer: Immunotherapies and Personalized Treatments

  • On Friday, Dr. Ned Sharpless will end his nearly five-year tenure at the National Cancer Institute.
  • He told Insider he plans to stay involved in this “golden age” of biomedical research.
  • Sharpless shared three of the most exciting findings about the cancer he watches.

Friday will be the last day in office of Dr. Ned Sharpless, director of the National Cancer Institute.

After nearly five years on the job — including seven months as acting food and drug commissioner at the Food and Drug Administration — he said he’s leaving federal office to spend more time with his family and consider his next career steps.

“It was an exhilarating and exciting experience to be in the federal government,” Sharpless told Insider.

The position has also been exhausting during a pandemic and a tumultuous election cycle. “I haven’t been the best husband, wife and son,” he said.

Sharpless, 55, has no plans to retire straight away. While he catches his breath, he will keep an eye on the latest scientific advances. He said we are in the midst of a “golden age” of biomedical research and that President Joe Biden’s goal of reducing cancer death rates by at least 50% over the next 25 years was feasible.

“There’s really a lot going on in the field of cancer. It’s an inspiring time in terms of biomedical progress,” Sharpless said.

This month he attended the annual conference of the American Association for Cancer Research, one of the biggest oncology conferences of the year. There were three cutting-edge presentations that particularly excited him, he told Insider.

He thinks the next breakthroughs in the fight against cancer could be cell therapy for an aggressive childhood tumour, a personalized method of treating breast cancer and new drugs for an important genetic mutation.

A new treatment for a deadly childhood tumor

Each year, approximately 300 children in the United States are diagnosed with a rare and aggressive brain tumor called diffuse intrinsic pontine glioma, or DIPG. The prognosis for this cancer is grim – it cannot be treated with surgery and only 10% of patients survive for two years after diagnosis.

But new research, published in the medical journal Nature and presented at the American Association for Cancer Research conference, has found that a type of CAR-T-cell therapy – where immune cells are taken from a patient, genetically engineered to target certain cancer proteins, then infused back into the patient – has been successful in a small sample of patients.

The phase one clinical trial was conducted by Dr. Robbie Majzner, a pediatric oncologist, and his lab at Stanford University. Four patients received a low dose of the new immunotherapy and three showed clinical improvement in their symptoms.

“It’s the beginning. It’s therapy that requires more work, but it’s really encouraging and exciting,” Sharpless said.

A personalized approach for the treatment of metastatic breast cancer

Sharpless said he’s also looking forward to keeping tabs on the cutting-edge work in breast cancer immunotherapy by Dr. Steven Rosenberg, chief of surgery at the National Cancer Institute.

“He’s the father of immunotherapy, in some ways,” Sharpless said.

Rosenberg, who won this year’s Pezcoller Foundation-AACR International Award for Extraordinary Achievement in Cancer Research, is researching ways to boost a patient’s immune system to fight cancer cells. In an article published in February in the Journal of Clinical Oncology, Rosenberg and his collaborators said that more than half of the 42 women studied were able to mount an immune response against their aggressive breast cancer.

Rosenberg used an experimental therapy involving tumor-infiltrating lymphocytes — white blood cells that can identify and fight cancerous growths — on six of those patients, three of whom experienced shrinkage in their tumors.

More drugs to come for ‘elusive’ cancer mutation

In May, the FDA approved Amgen’s sotorasib, the first cancer drug that targets mutations in a gene called KRAS. Mutations in this gene cause cells to grow out of control and are commonly seen in patients with lung cancer.

The effect of KRAS mutations on cancer was discovered in the 1980s, but it is a difficult mutation to treat.

“We’ve been working on this goal since the very early days,” Sharpless said. “He proved elusive.”

But, Sharpless said, the approval for sotorasib was just “the first in a long line.” Other major pharmaceutical companies will soon release these KRAS inhibitor treatments, he said.

This month, Swiss pharmaceutical company Novartis announced promising early clinical data for its own KRAS inhibitor, which showed response in four of seven lung cancer patients.