U.S. FDA Approves Tecartus® for Adults With Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia
“Adults with ALL face a significantly poorer prognosis compared to children, and roughly half of all adults with B-ALL will relapse on currently available therapies,” said Bijal Shah, MD, ZUMA-3 investigator and medical oncologist, Moffitt Cancer Center, Tampa, Florida. “We now have a new meaningful advancement in treatment for these patients. A single infusion of Tecartus has demonstrated durable responses, suggesting the potential for long-term remission and a new approach to care.”

FDA Granted Breakthrough Therapy Designation for Repotrectinib Treatment in Patients with NTRK-Positive, TKI-Pretreated Advanced Solid Tumors
“We are excited to receive our second Breakthrough Therapy designation and seventh overall regulatory designation for repotrectinib,” said Athena Countouriotis, M.D., president and chief executive officer. “There remains an unmet medical need for NTRK-positive, TKI-pretreated advanced solid tumor patients where there are no targeted therapies currently approved. We look forward to presenting additional clinical data from our TRIDENT-1 study of repotrectinib during the AACR-NCI-EORTC conference later this week, including a late-breaker plenary presentation where early clinical data from the NTRK EXP-5 and EXP-6 cohorts will be discussed.”

N-803 IL-15 Receptor Agonist to Combine with Keytruda in 700-Site Lung-MAP Clinical Trial of a Chemo-Free Therapy
“While some patients with lung tumors have targetable genetics, the majority do not, and for them there are fewer treatment options,” said John Wrangle, M.D., one of the researchers at the Medical University of South Carolina who developed the study. “The Lung-MAP study aims to change that by combining different therapies such as Anktiva and Keytruda in an effort to discover highly effective and targeted therapies for these patients.”

PTC-AADC gene therapy shows holistic improvements in children with AADC deficiency
 PTC Therapeutics shows five-year results for its novel gene therapy, PTC-AADC, which leads to profound improvements in children with aromatic L-Amino acid decarboxylase (AADC) deficiency, a previously intractable, fatal and devastating rare disorder of the central nervous system. Children treated with PTC-AADC developed motor function and cognitive skills not previously seen, such as holding up their head, sitting or standing with support and communicating, and these persisted for up to 10 years. PTC-AADC is currently under review by the European Medicines Agency's Committee for Medicinal Products for Human Use. A Biologics License Application is expected to be submitted to the U.S. Food and Drug Administration by the end of 2021.

UCSF Researchers Report Initial Success With Neuromodulation for Treatment of Treatment-Resistant Depression
Researchers at the University of California, San Francisco (UCSF) have developed a first-of-its-kind neuromodulation treatment for treatment-resistant depression. The treatment makes use of a  small device implanted in the brain of a patient trained to issue electrical pulses to areas of the brain linked to the condition.  The effects of the neurostimulation showed near-immediate effects, according to the study published in Nature Medicine.
The use of neuromodulation for the treatment of other neurological disorders like anxiety, depression, Parkinson’s disease, and other conditions has seen only limited success. The, largely because many neurological illnesses present differently from person to person, especially for difficult-to-treat conditions like depression.
To overcome this limitation, the research team overcame this by personalizing the treatment based on the patient’s specific brain patterns of a patient – producing remarkable, near-immediate relief of the patient’s severe, treatment-resistant depression.
The team’s neuromodulation system dubbed as the “pacemaker for the brain,” includes a small device implanted in the patient’s brain that continuously monitors neurological activity and instructs two leads in separate areas of the brain to emit electrical pulses if abnormal activity is detected.
Not only did the treatment eliminate depression symptoms almost immediately, the improvements lasted for approximately 15 months, according to the researchers. That’s in sharp contrast to other deep brain stimulation devices, which typically offer some relief from depression symptoms after four to eight weeks after continuous stimulation begins.
Although they have shown the success of the neuromodulation treatment in one patient, the researchers plan to continue validating the technology as a treatment for severe depression, with plans to recruit around a dozen subjects for trials.

 

Zaki syndrome: the newly uncovered condition and its potential cure